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Share Name | Share Symbol | Market | Type |
---|---|---|---|
Rexahn Pharmaceuticals Inc | NASDAQ:REXN | NASDAQ | Common Stock |
Price Change | % Change | Share Price | Bid Price | Offer Price | High Price | Low Price | Open Price | Shares Traded | Last Trade | |
---|---|---|---|---|---|---|---|---|---|---|
0.00 | 0.00% | 1.81 | 1.75 | 1.83 | 0 | 01:00:00 |
☒ |
Annual report pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934.
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☐ |
Transition report pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934.
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Delaware
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11-3516358
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(State or other jurisdiction of incorporation or organization)
37000 Grand River Avenue, Suite 120
Farmington Hills, MI
(Address of principal executive offices) |
(I.R.S. Employer Identification No.)
48335
(Zip Code)
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Title of each class
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Trading Symbol
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Name of each exchange on which registered
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Common Stock, $0.0001 par value per share
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OCUP
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The Nasdaq Stock Market LLC
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Large accelerated filer ☐
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Accelerated filer ☐
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Non-accelerated filer ☒
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Smaller reporting company ☒
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Emerging growth company ☐
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PART I
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6
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ITEM 1.
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6 |
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ITEM 1A.
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61 |
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ITEM 1B.
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103 |
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ITEM 2.
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103 |
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ITEM 3.
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103 |
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ITEM 4.
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103 |
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PART II
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104 |
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ITEM 5.
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104 |
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ITEM 6.
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104 |
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ITEM 7.
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105 |
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ITEM 7A.
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119 |
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ITEM 8.
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119 |
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ITEM 9.
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119 |
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ITEM 9A.
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119 |
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ITEM 9B.
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120 |
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ITEM 9C.
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120 |
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PART III
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121 |
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ITEM 10.
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121 |
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ITEM 11.
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121 |
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ITEM 12.
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121 |
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ITEM 13.
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121 |
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ITEM 14.
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121 |
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PART IV
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122 |
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ITEM 15.
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122 |
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ITEM 16.
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125 |
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154 |
• |
We currently depend entirely on the success of Nyxol and APX3330, our only product candidates. We may never complete clinical development of, receive marketing approval for, or successfully commercialize,
Nyxol alone or as adjunctive therapy with low dose pilocarpine (LDP), APX3330, or other product candidates we may pursue in the future for any indication.
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• |
Viatris has exclusive global rights to commercialize our Nyxol products in key global markets. Viatris’ failure to timely develop or commercialize these products would have a material adverse effect on our
business and operating results.
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• |
The results of previous clinical trials may not be predictive of future results, and the results of our current and planned clinical trials may not satisfy the requirements of the FDA or non-U.S. regulatory
authorities.
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• |
Changes in regulatory requirements or FDA guidance, or unanticipated events during our clinical trials, may result in changes to clinical trial protocols or additional clinical trial requirements, which
could result in increased costs to us or delays in its development timeline.
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• |
We expect to incur losses for the foreseeable future and may never achieve or maintain profitability.
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• |
Adverse global economic conditions could have a negative effect on our business results of operations and financial condition and liquidity.
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• |
Adverse developments affecting the financial services industry could negatively affect our current and projected business operations, financial condition and results of operations.
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• |
Raising additional capital may cause dilution to our stockholders, restrict our operations, or require us to relinquish rights to our technologies or product candidates.
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• |
Even if we receive marketing approval for our product candidates in the United States, we may never receive regulatory approval to market such product candidates outside of the United States.
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• |
Our employees or our representatives may engage in misconduct or other improper activities, including violating applicable regulatory standards and requirements or engaging in insider trading, which could
significantly harm our business.
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• |
We face substantial competition, which may result in others discovering, developing, or commercializing products before or more successfully than we do.
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• |
We lack experience in commercializing products, which may have an adverse effect on our business.
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• |
If we are unable to establish sales and marketing capabilities or enter into agreements with third parties to sell, market, and distribute APX3330, if
approved, we may not be successful in commercializing APX3330 if and when it is approved.
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• |
Product liability lawsuits against us, or our suppliers and manufacturers, could cause us to incur substantial liabilities and could limit commercialization of any product candidate that we may develop.
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• |
We are unable to control all aspects of our clinical trials due to our reliance on clinical research organizations (“CROs”), contract development and manufacturing organizations (“CDMOs”) and other third
parties that assist us in conducting clinical trials.
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• |
We are unable to control the supply, manufacture and testing of bulk drug substances and the formulation, testing and packaging of preclinical and clinical drug supplies of our product candidates, and will be unable to control these
elements at the commercial stage, due to our reliance on third party manufacturers and analytical facilities.
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• |
If we are not able to establish new collaborations for APX3330 on commercially reasonable terms, we may have to alter our development, manufacturing, and commercialization plans.
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• |
If we are unable to obtain and maintain sufficient patent protection for our product candidates, our competitors could develop and commercialize products or technology similar or identical to those of us,
which would adversely affect our ability to successfully commercialize any product candidates we may develop, our business, results of operations, financial condition and prospects.
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• |
If we do not obtain protection under the Hatch-Waxman Act and similar foreign legislation by extending the patent terms and obtaining data exclusivity for our product candidate, our business may be
materially harmed.
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• |
We may not be able to protect or practice our intellectual property rights throughout the world.
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• |
Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment, and other requirements imposed by governmental agencies, and our patent
protection could be reduced or eliminated for noncompliance with these requirements.
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• |
We depend on intellectual property sublicensed from Apexian Pharmaceuticals, Inc. (“Apexian”) for our APX3330 product candidate under development and our additional pipeline candidates, and the termination
of, or reduction or loss of rights under, this sublicense would harm our business.
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• |
We are dependent on our key personnel, and if we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy.
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• |
We will need to develop and expand our company and may encounter difficulties in managing this development and expansion, which could disrupt our operations.
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• |
Our insurance policies are expensive and protect only from some business risk, which leaves us exposed to significant uninsured liabilities.
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• |
Environmental, social, and governance matter and any related reporting obligations may impact our business.
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• |
If we fail to comply with the continued listing standards of the Nasdaq Capital Market, our common stock could be delisted. If it is delisted, our common stock and the liquidity of our common stock would be
impacted.
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• |
The market price of our common stock may fluctuate significantly.
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• |
We may be subject to securities litigation, which is expensive and could divert management attention.
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• |
Advance the clinical development of Nyxol and APX3330. Ocuphire entered into the Nyxol License Agreement in November 2022, pursuant to which Viatris has exclusive rights to develop and commercialize Nyxol. Pursuant to the Nyxol License Agreement, Ocuphire continues to conduct
development activities in the United States in partnership with Viatris, and is reimbursed by Viatris for such budgeted development activities. Ocuphire submitted a United States NDA for Nyxol for RM with a PDUFA goal date of
September 28, 2023, and is advancing Phase 3 trials for presbyopia and DLD. Viatris has exclusive rights to pursue development and undertake commercialization
efforts for Nyxol outside of the United States. Ocuphire plans an EOP2 meeting with the FDA to advance Phase 3 trials for APX3330 in DR.
|
• |
Target Nyxol and APX3330 for large ophthalmic indications. Ocuphire believes Nyxol has therapeutic potential to improve vision performance in RM, presbyopia and DLD.
Ocuphire also believes AXP3330 has potential to prevent or delay the progression of disease in patients with DR, DME, and other retinal diseases, while potentially reducing the burden of intravitreal injections.
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• |
Maintain and expand its intellectual property portfolio.
Ocuphire has out-licensed the global patent rights to Nyxol with respect to its formulation, combinations, and use in multiple indications to Viatris. Ocuphire owns
an exclusive worldwide sublicense for the Ref-1 Inhibitor program, including its product candidate APX3330, for all its ophthalmic and diabetic indications, and compositions and methods of use for Ref-1 pipeline candidates, including
APX2009 and APX2014. Ocuphire continues to explore additional opportunities to expand and extend this intellectual property protection, both in the U.S. and in other jurisdictions.
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• |
Maximize the global commercial value of Nyxol and APX3330. If cleared to market by the FDA, Nyxol will be commercialized by the Viatris Eye
Care Division in the U.S. and major non-U.S. markets pursuant to the Nyxol License Agreement. Ocuphire plans to seek one or more partners to commercialize APX3330 both in and outside of the United States.
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• |
Evaluate in-licensing and acquisition opportunities. Ocuphire’s team is well qualified to identify and in-license or acquire clinical-stage ophthalmological assets
and continually evaluates opportunities to expand and diversify its pipeline.
|
• |
Reduction in pupil diameter with durable effects. In multiple Phase 2 and Phase 3 trials Nyxol reduced pupil diameter by approximately 1 – 1.5 mm in both mesopic
(dim) and photopic (bright) conditions, with such reductions sustained over 24 hours.
|
• |
Improvement in distance corrected near visual acuity. When studied in patients with presbyopia in Phase 2 trials, Nyxol alone and in combination with LDP showed
statistically significant improvement in distance-corrected near visual acuity with ≥3 lines gain from baseline. Nyxol provides an optimal pupil size of 2 mm – 3 mm.
|
• |
Improvement in low contrast visual acuity. When studied in patients with DLD in multiple Phase 2 trials, Nyxol showed statistically significant improvement in low
contrast mesopic best-corrected distance visual acuity at ≥1 and ≥2 lines, with a trend at ≥3 lines on a standard visual chart.
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• |
Favorable tolerability profile. To date, Nyxol has been observed to be well-tolerated, with unchanged or decreased intraocular pressure in the 12 completed Phase 1,
Phase 2 and Phase 3 clinical trials conducted. Nyxol produces a transient, mild hyperemia effect that disappears within several hours or immediately upon application of anti-redness eye drops. Nyxol is also observed to have no systemic
effects such as changes in blood pressure or heart rate.
|
• |
Designed to be a convenient, once-daily eye drop or tunable combination option. Nyxol is being evaluated for chronic use as a once-daily administration before
bedtime. Nyxol has been shown in multiple Phase 2 trials and Phase 3 trials to have a durable effect of over 24 hours, which could encourage patient compliance. Use of LDP eye drops as an adjunct to Nyxol may offer the benefit of
tunability to presbyopia patients based on their vision and lifestyle needs.
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• |
Stable, cost-effective ophthalmic formulation. Nyxol is a single-use, preservative-free, proprietary eye drop formulation with stability suitable to support potential
commercialization. Its active pharmaceutical ingredient, phentolamine mesylate USP grade, is a small molecule with advantages of standardized, scalable, and lower-cost manufacturing processes.
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• |
RM, the reversal of pharmacologically induced dilation of the pupils, where dilation leads to increased sensitivity to light and an inability to focus, making it
difficult to read, work, and drive. RM is a single-use indication for which no approved therapy is commercially available at present.
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• |
Presbyopia, a condition in which the eye’s lens loses elasticity, affecting its ability to focus on near objects. Presbyopia typically occurs after age 40 and most
patients use reading glasses in order to read or see objects close to them. VuityTM, approved in October 2021, is the only eye drop currently marketed for the treatment of presbyopia.
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• |
DLD, a condition in which peripheral imperfections (aberrations) of the cornea scatter light when the pupil opens wide in dim light. Patients with DLD experience
glare, halos, starbursts, and decreased contrast sensitivity. DLD is a new indication with no approved therapies.
|
• |
Potential to be the first oral therapy. Compared to frequent intravitreal anti-VEGF injections, associated with ocular complications, once or twice a day oral
administration of APX3330 could be a convenient, new preventative therapeutic option or adjunctive treatment option for large number of patients with retinal diseases, if approved.
|
• |
Upstream target implicated in two validated pathways. APX3330 is designed to lead to inhibition of two validated cell signaling pathways (angiogenesis and
inflammation) known to cause various retinal diseases. Moreover, the APX3330 mechanism of action is distinct in working upstream of the current anti-VEGF therapies, suggesting that it could complement anti-VEGF therapies and potentially
reduce frequency of doctor visits and intravitreal injections.
|
• |
Favorable tolerability profile. In 12 completed Phase 1 and Phase 2 clinical trials, APX3330 was well-tolerated. The AEs were mostly infrequent and mild with
transient pruritis being the most common. No systemic effects such as changes in blood pressure or heart rate were seen, and no toxicities related to neurological, cardiovascular, renal, pulmonary, or gastrointestinal organs were
observed.
|
• | Potential benefit of systemic administration. As a systemic agent, APX3330 can be expected to treat bilateral binocular (both eyes) retinal vascular disease. |
• | Stable, cost-effective oral tablet. APX3330 is formulated as an oral tablet with favorable stability characteristics, and its active pharmaceutical ingredient is a small molecule with the advantages of standardized, scalable, and lower-cost manufacturing processes. |
• |
DR, the leading cause of vision loss in adults aged 20–74 years, which results from chronic elevations of glucose in the blood that leads to cell damage in the
retina. Retinal key opinion leaders’ feedback suggests that slowing of DR progression with an oral agent would be a useful treatment in patients with background DR and good visual function.
|
• |
DME, one of the most common complications of DR, in which vascular leakage causes swelling of the retinal macula and a loss of visual acuity.
|
• |
wAMD, a chronic eye disorder that causes visual distortions in the central part of one’s vision, in which abnormal blood vessels leak fluid or blood into the macula,
the part of the eye that is critical for central and color vision.
|
• |
GA, an advanced form of age-related macular degeneration (AMD) that leads to progressive and irreversible vision loss.
|
• |
Non-proliferative DR, or NPDR. NPDR is an earlier, more typical stage of DR and can progress into more severe forms of DR over time if untreated and if exposure to
elevated blood sugar levels persists.
|
• |
Proliferative DR, or PDR. PDR is a more advanced stage of DR than NPDR. It is characterized by retinal neovascularization and, if left untreated, leads to permanent
damage and blindness.
|
Trial
Name
(IND
Number)
|
Patient /
Indication
|
Phase
|
Trial Objectives
|
Doses
|
Number of
Patients^
|
Dosing
|
Key
Endpoints
|
|||||||
NYX-001
(67-288)
|
Healthy Volunteers
|
1
|
Double-masked, randomized, single dose, 3-arm controlled, parallel trial to determine the efficacy and safety of phentolamine mesylate
|
0.2%
PMOS
|
Nyxol*=15, Visine=15,
Visine + Nyxol*=15
Total = 45
|
Single-dose
|
Safety and Efficacy (PD)
|
|||||||
NYX-002^
(67-288)
|
Healthy Volunteers
|
1
|
Double-masked, randomized, placebo-controlled, single-dose, incomplete block, 3-period crossover, dose escalation trial evaluating the tolerability and efficacy of
phentolamine mesylate
|
0.2%, 0.4%, 0.8%
PMOS
|
Nyxol*=16 Placebo=12
Total = 16
|
Single-dose
|
Safety and Efficacy (PD, VA)
|
OP-NYX-004^
(73-987)
|
Night Vision Disturbances Patients
|
1 / 2
|
Double-masked, randomized, placebo-controlled, single-dose, incomplete block 3-period crossover, dose escalation trial to determine the efficacy and safety of
phentolamine mesylate
|
0.2%, 0.4%, 0.8%
PMOS
|
Nyxol*=16 Placebo=12
Total = 16
|
Single-dose
|
Safety and Efficacy
|
|||||||
OP-NYX-SNV
(70-736)
|
Severe Night Vision Disturbances Patients
|
2
|
Double-masked, randomized, placebo-controlled, single-dose trial to assess the efficacy and safety of phentolamine mesylate ophthalmic solution
|
1.0%
PMOS
|
Nyxol*=16, Placebo=8
Total = 24
|
Single-dose
|
Safety and Efficacy (PD, LCVA, CS, WA)
|
|||||||
OP-NYX-01a2
(70-499)
|
Severe Night Vision Disturbances Patients
|
2
|
Double-masked, randomized, placebo-controlled, single-dose, 3-arm trial to assess the efficacy and safety of Nyxol
|
0.5%, 1.0%
PMOS
|
Nyxol=40 Placebo=20
Total = 60
|
Multiple doses (15-28 days)
|
Safety and Efficacy (PD, LCVA, CS)
|
|||||||
OPI-NYXG-201
(ORION-1)
(70-499)
|
Glaucoma and Ocular Hypertension, Elderly Patients
|
2b
|
Double-masked, randomized, placebo-controlled, multiple-dose, multi-center trial to assess the efficacy and safety of Nyxol
|
1.0%
PMOS
|
Nyxol=19 Placebo=20
Total = 39
|
Multiple doses (14 days)
|
Safety and Efficacy (IOP, PD, near VA, VA)
|
|||||||
OPI-
NYXRM-201
(MIRA-1)
(70-499)
|
Healthy Patients/ Reversal of Mydriasis
|
2b
|
Double-masked, randomized, placebo-controlled, crossover, single-dose, multi-center trial to assess the efficacy and safety of Nyxol in reducing pharmacologically
induced mydriasis
|
1.0%
PMOS
|
Nyxol=31 Placebo=32
Total = 32
|
Single-dose
|
Safety and Efficacy (PD, Accommodation, VA)
|
|||||||
OPI-
NYXRM-301
(MIRA-2)
(70-499)
|
Healthy Patients/ Reversal of Mydriasis (including 12–17 years-old)
|
3
|
Double-masked, randomized, placebo-controlled, single-dose, multi-center trial to assess the efficacy and safety of Nyxol in reducing pharmacologically induced
mydriasis
|
0.75%
POS
|
Nyxol=94
Placebo=91
Total = 185
|
Single-dose
|
Safety and Efficacy (PD, Accommodation, VA)
|
OPI-NYXRM-302 (MIRA-3)
(70-499)
|
Healthy Patients/ Reversal of Mydriasis (including 12-17-years old)
|
Double-masked, randomized, placebo-controlled, single-dose, multi-center trial to assess the efficacy and safety of Nyxol in reducing pharmacologically induced mydriasis
|
0.75%
POS
|
POS=214
Placebo=124
Total = 368
|
Single-dose
|
Safety and Efficacy (PD, Accommodation, VA)
|
||||||||
OPI-NYXRMP-303 (MIRA-4)
(70-499)
|
Healthy Patients/ Reversal of Mydriasis (between ages of 3-and 11)
|
Randomized, Parallel-Arm, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Nyxol (0.75% Phentolamine Ophthalmic Solution) to Reverse Pharmacologically Induced
Mydriasis in Healthy Pediatric Subjects
|
0.75%
POS
|
POS=11
Placebo-12
Total=23
|
Single-dose
|
Safety and Efficacy (PD, VA)
|
||||||||
OPI-NYXP-201
(VEGA-1)
(70-499)
|
Presbyopia patients (ages of 40 and 64)
|
2
|
Randomized, Placebo-Controlled, Double-Masked Study of the Safety and Efficacy of Nyxol (0.75% Phentolamine Ophthalmic Solution) with Low-Dose (0.4%) Pilocarpine Eye Drops in Subjects with
Presbyopia
|
0.75%
POS
|
Nyxol +LDP
= 44
Placebo alone = 45
Nyxol alone
= 30
Placebo +LDP = 31
|
Multiple doses (4-5 days), Single dose of LDP
|
Safety and Efficacy (DCNVA, VA, PD)
|
|||||||
OPI-NYXDLD-301
(LYNX-1)
(70-499)
|
Night Vision Disturbances in adults
|
Randomized, Placebo-Controlled, Double-Masked Study of the Safety and Efficacy of Nyxol (0.75% Phentolamine Ophthalmic Solution) in Subjects with Dim Light Vision Disturbances
|
0.75%
POS
|
POS=72
Placebo=73
Total =145
|
Multiple doses
(14 days)
|
Safety and Efficacy (mLCVA, VA, PD)
|
Trial Number /
Name
|
Patient /
Indication
|
Phase
|
Trial Objectives
|
Doses
|
Number
of
Patients^
|
APX3330 Total Exposure Days
|
Dosing
|
Key Endpoints
|
||||||||
APX_CLN_0001
(Eisai)
Completed
|
Healthy Volunteers
|
1
|
Single-dose placebo-controlled trial of APX3330 to investigate safety and pharmacokinetics
|
10 mg 30 mg 60 mg 120 mg 180 mg 240 mg
|
APX3330 = 12 Placebo = 6
|
36 days
|
Single dose
|
Plasma Concentration of total quinone forms, safety
|
||||||||
APX_CLN_0002
(Eisai)
Completed
|
Healthy Volunteers
|
1
|
Repeat-dose placebo-controlled trial to investigate safety and pharmacokinetics
|
120 mg QD 120 mg BID
|
APX3330 = 12 Placebo = 6
|
96 days
|
8 days
|
Plasma Concentration of APX3330, safety
|
APX_CLN_0003
(Eisai)
Completed
|
Healthy Volunteers
|
1
|
Repeat-dose trial to determine effects of food on pharmacokinetics
|
240 mg
|
APX3330 = 6
|
84 days
|
1 week
|
Plasma Concentration of APX3330, safety
|
||||||||
APX_CLN_0004
(Eisai)
Completed
|
Healthy Volunteers
|
1
|
Single-dose trial to determine the effects of meals on pharmacokinetics
|
120 mg
|
APX3330 = 6
|
6 days
|
Single dose
|
Plasma Concentration of APX3330, Safety
|
||||||||
APX_CLN_0005
(Eisai)
Completed
|
Chronic Hepatitis B Patients
|
2
|
Dose-escalation trial to investigate safety, efficacy and tolerability
|
20 mg 60 mg 120 mg 240 mg
|
APX3330 = 40
|
3360 days
|
12 weeks
|
Safety
|
||||||||
APX_CLN_0006
(Eisai)
Completed
|
Chronic Hepatitis C Patients
|
2
|
Dose-escalation trial to investigate safety, efficacy and tolerability
|
20 mg 60 mg 120 mg 240 mg
|
APX3330 = 51
|
4284 days
|
12 weeks
|
Safety
|
APX_CLN_0007
(Eisai)
Completed
|
Chronic Hepatitis C Patients
|
2
|
Double-masked, placebo-controlled trial to investigate safety, efficacy and tolerability
|
120 mg 240 mg
|
APX3330 = 128
Placebo = 68
|
10,752 days
|
Placebo = 82 days
APX3330 120 mg = 79 days
240 mg = 78 days
|
Rate of change in GPT level, improvement in liver function, general performance
|
||||||||
APX_CLN_0008^
(Eisai)
Completed
|
Healthy Patients
|
1
|
Single-blind, single-dose, 3-step trial to investigate safety and pharmacokinetics of higher doses
|
300 mg 420 mg 600 mg
|
APX3330 = 18
|
54 days
|
Single dose
|
Plasma Concentration of APX3330, safety
|
||||||||
APX_CLN_0009
(Eisai)
Completed
|
Advanced Liver Cirrhosis Patients
|
2
|
Repeated-dose trial to investigate safety, efficacy and tolerability
|
120 mg
|
APX3330 = 30
|
420 days
|
2 weeks
|
Liver function, patient functional status, tolerability
|
||||||||
APX_CLN_0010
(Eisai)
Completed
|
Advanced Liver Cirrhosis Patients
|
2
|
Repeated-dose trial to investigate safety, efficacy and tolerability
|
120 mg
|
APX3330 = 18
|
504 days
|
4 weeks
|
Liver function, patient functional status, tolerability
|
||||||||
APX_CLN_0011
(Apexian)
Completed
|
Advanced Solid Tumor Patients
|
1
|
Multicenter, open-label, dose-escalation to investigate safety, efficacy, pharmacokinetics, and recommended Phase 2 dose
|
240 mg 360 mg 480 mg 600 mg 720 mg
|
APX3330 = 19
|
2354 days
|
21-day cycles until disease progression or study withdrawal
|
Tumor response, safety, PK, target engagement
|
||||||||
ZETA-1
(Ocuphire)
Completed
|
Diabetic Retinopathy and DME
|
2
|
Double-masked, randomized, placebo-controlled, multi-center trial
|
600mg
|
APX3330 = 51
Placebo =52
|
>7,900
|
600mg daily for 24 weeks
|
≥ 2 step improvement
on the DRSS score at week 24, binocular ≥3 step worsening or improvement and safety
|
• |
Lucentis® (ranibizumab) and Avastin® (bevacizumab), which are anti-VEGF monoclonal antibody intravitreal injections,
developed by Genentech, Inc and Roche AG.
|
• |
EYLEA® (aflibercept), a VEGF inhibitor intravitreal
injection, developed by Regeneron Pharmaceuticals.
|
• |
Vabysmo® (Faricimab), a bispecific monoclonal antibody targeting VEGF-A and Ang-Tie2 pathway developed by Genentech, Inc and
Roche AG.
|
• |
Beovu® (Brolucizumab), an anti-VEGF monoclonal antibody intravitreal injection, developed by Novartis AG.
|
• |
MACUGEN® (pegaptanib sodium injection), a selective inhibitor of VEGF-165, developed by Bausch + Lomb.
|
• |
Ozurdex® (dexamethasone), a corticosteroid IVT implant, developed by Allergan plc.
|
• |
Iluvien (fluocinolone acetonide), a corticosteroid IVT implant, developed by Alimera Sciences, Inc.
|
• |
Abicipar, an anti-VEGF intravitreal injection with a long duration of action, developed by Allergan plc and Molecular Partners.
|
• |
KSI-301, an anti-VEGF antibody intravitreal injection coupled with a biopolymer that is intended to increase the time between injections, developed by Kodiak Sciences.
|
• |
OPT-302, an intravitreal injection which binds to multiple types of VEGF receptors that could be used with other anti-VEGF agents, developed by Opthea Limited.
|
• |
ALG-1001, an integrin peptide therapy intravitreal injection that is being evaluated as a sequential or in-combination therapy with bevacizumab in patients with DME, developed by Allegro Ophthalmics, LLC.
|
• |
RG-7774, an orally administered selective CB2 (Cannabinoid 2) receptor agonist that is being evaluated in patients with moderately severe to severe non-proliferative diabetic retinopathy, developed by
Hoffmann-LA Roche, AG.
|
• |
RZ402, an oral small molecule selective and potent plasma kallikrein inhibitor (PKI) for the chronic treatment of diabetic macular edema (DME), developed by Rezolute, Inc.
|
• |
Xiflam™, an oral small molecule drug for the treatment of dry form of Age-Related Macular Degeneration (AMD), Geographic Atrophy (GA), Diabetic Retinopathy (DR) manifesting Diabetic Macular Edema (DME),
developed by InflammX.
|
• |
AKST4290, an oral small molecule CCR3 Eotaxin inhibitor for the treatment of diabetic retinopathy (terminated in 2022) and wet AMD by Alkahest.
|
• |
BAY1101042, an oral guanylate cycles activator for the treatment of diabetic retinopathy developed by Bayer.
|
• |
OPL-0401, an oral ROCK 1/2 inhibitor for the treatment of diabetic retinopathy, developed by Valo Health.
|
• |
BI 1467335, an oral AOC3 inhibitor for the treatment of diabetic retinopathy, developed by Boehringer Ingelheim.
|
• |
LY333531, an oral Protein Kinase C inhibitor for the treatment of diabetic retinopathy was terminated by Eli Lilly in 2006.
|
• |
CSF-1, with low dose pilocarpine and a secondary agent (lubricant), developed by Orasis Pharmaceuticals Ltd. (PDUFA date is October 22, 2023)
|
• |
LNZ100 and LNZ101, with aceclidine (another miotic agent) and brimonidine, developed by Lenz Therapeutics.
|
• |
MicroLine®, which is a micro-dose delivery of pilocarpine using proprietary device developed by Eyenovia, Inc.
|
• |
KT-101, which uses pilocarpine in the AcuStream delivery system, developed by Kedalion Therapeutics, Inc.
|
• |
BrimocholTM, with brimonidine and carbachol (both are miotic agents), developed by Visus Therapeutics, Inc.
|
• |
UNR844, which uses a mechanism that involves softening the lens to increase near visual acuity, developed by Novartis AG (originally Encore Vision, Inc.). This development program has been terminated.
|
• |
completion of preclinical laboratory tests, animal studies and formulation studies in compliance, as applicable, with the Animal Welfare Act and FDA’s good laboratory practice, or GLP, regulations;
|
• |
submission to the FDA of an IND, which must take effect before human clinical trials may begin;
|
• |
approval by an independent institutional review board, or IRB, representing each clinical site before each clinical trial may be initiated;
|
• |
performance of adequate and well-controlled human clinical trials in accordance with good clinical practices, or GCP, and other applicable regulations to establish the safety and efficacy of the proposed drug product for each proposed
indication;
|
• |
manufacturing, packaging, labelling, and distribution of drug substances and drug products consistent with the FDA’s Good Manufacturing Practice (GMP) regulations which are utilized in the GLP non-clinical and GCP clinical studies to
investigate the drug candidate;
|
• |
development of product label, package inserts, and prescriber information that is intended to be used and included with the commercial product;
|
• |
preparation and submission to the FDA of an NDA;
|
• |
review of the product by an FDA advisory committee, where appropriate or if applicable;
|
• |
satisfactory completion of one or more FDA inspections of the manufacturing facility or facilities at which the product, or components thereof, are produced to assess compliance with current Good Manufacturing Practices, or cGMP,
requirements and to assure that the facilities, methods and controls are adequate to preserve the product’s identity, strength, quality and purity;
|
• |
satisfactory completion of FDA audits of clinical trial sites to assure compliance with GCPs and the integrity of the clinical data;
|
• |
payment of user fees and securing FDA approval of the NDA; and
|
• |
compliance with any post-approval requirements, including Risk Evaluation and Mitigation Strategies, or REMS, and post-approval studies required by the FDA.
|
• |
Phase 1. The drug is initially introduced into healthy human patients or, in certain indications such as cancer, patients with the target disease or condition and tested for safety, dosage
tolerance, absorption, metabolism, distribution, excretion and, if possible, to gain an early indication of its effectiveness and to determine optimal dosage.
|
• |
Phase 2. The drug is administered to a limited patient population to identify possible adverse effects and safety risks, to preliminarily evaluate the efficacy of the product for specific
targeted diseases and to determine dosage tolerance and optimal dosage.
|
• |
Phase 3. The drug is administered to an expanded patient population, generally at geographically dispersed clinical trial sites, in well-controlled clinical trials to generate enough data to
statistically evaluate the efficacy and safety of the product for approval, to establish the overall risk-benefit profile of the product and to provide adequate information for the labeling of the product.
|
• |
restrictions on the marketing or manufacturing of the product, complete withdrawal of the product from the market or product recalls;
|
• |
fines, warning letters or holds on post-approval clinical trials;
|
• |
refusal of the FDA to approve pending NDAs or supplements to approved NDAs, or suspension or revocation of product license approvals;
|
• |
product seizure or detention, or refusal to permit the import or export of products; or
|
• |
injunctions or the imposition of civil or criminal penalties.
|
• |
A special, nondeductible fee on any entity that manufactures or imports specified branded prescription drugs and biologic agents, apportioned among these entities according to their market share in certain government healthcare
programs, although this fee would not apply to sales of certain products approved exclusively for orphan indications;
|
• |
Expansion of eligibility criteria for Medicaid programs by, among other things, allowing states to offer Medicaid coverage to certain individuals with income at or below 133% of the federal poverty level, thereby potentially increasing
a manufacturer’s Medicaid rebate liability;
|
• |
Expanded manufacturers’ rebate liability under the Medicaid Drug Rebate Program by increasing the minimum rebate for both branded and generic drugs and revising the definition of “average manufacturer price,” or AMP, for calculating
and reporting Medicaid drug rebates on outpatient prescription drug prices and extending rebate liability to prescriptions for individuals enrolled in Medicare Advantage plans; addressed a new methodology by which rebates owed by
manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected;
|
• |
Expanded the types of entities eligible for the 340B drug discount program;
|
• |
Established the Medicare Part D coverage gap discount program by requiring manufacturers to provide a 50% point-of-sale-discount off the negotiated price of applicable brand drugs to eligible beneficiaries during their coverage gap
period as a condition for the manufacturers’ outpatient drugs to be covered under Medicare Part D;
|
• |
A new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research; and
|
• |
Established the Center for Medicare and Medicaid Innovation within CMS to test innovative payment and service delivery models to lower Medicare and Medicaid spending, potentially including prescription drug spending.
|
• |
The federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce
or reward either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made, in whole or in part, under a federal healthcare program such as Medicare and Medicaid;
|
• |
The federal civil and criminal false claims laws, including the civil False Claims Act, and civil monetary penalties laws, which prohibit individuals or entities from, among other things, knowingly presenting, or causing to be
presented, to the federal government, claims for payment that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government;
|
• |
The federal Physician Payments Sunshine Act (part of the ACA), commonly known as the Sunshine Act or Open Payments, requires certain manufacturers of drugs, devices, biologics and medical supplies to disclose certain payments and
financial relationships with healthcare providers directly to the Centers for Medicare & Medicaid Services (CMS). Failure to comply with these laws can result in significant civil monetary penalties;
|
• |
The federal Health Insurance Portability and Accountability Act of 1996, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (collectively, "HIPAA"). HIPAA mandates, among other things, the
adoption of uniform standards for the electronic exchange of information in common health care transactions as well as standards relating to the privacy and security of individually identifiable health information. These standards require
the adoption of administrative, physical and technical safeguards to protect such information. In addition, many states have enacted comparable laws addressing the privacy and security of health information, some of which are more
stringent than HIPAA. Failure to comply with these laws can result in the imposition of significant civil and criminal penalties;
|
• |
Other analogous state and foreign laws and regulations, such as state anti-kickback and false claims laws, which may apply to healthcare related items or services that are reimbursed by non-governmental third-party payors, including
private insurers.
|
• |
Viatris may not be able to obtain from us or manufacture our products in a timely or cost-effective manner;
|
• |
Viatris may not timely perform its obligations under the Nyxol License Agreement;
|
• |
Viatris may fail to effectively commercialize our products;
|
• |
Viatris may not be able to sublicense Nyxol to one or more suitable parties outside
the United States; or
|
• |
Contractual disputes or other disagreements between us and Viatris, including those
regarding the development, manufacture, sub licensure and commercialization of our products, interpretation of the Nyxol License Agreement, and ownership of proprietary rights. Viatris may select a new development partner for Nyxol in
the U.S. upon 90 days’ notice to Ocuphire.
|
• |
the data collected from preclinical studies and clinical trials of our product candidates may not be sufficient to support the submission or acceptance of an NDA for one or more indications;
|
• |
we may not be able to demonstrate to the satisfaction of the FDA that our product candidates are safe and effective for any indication;
|
• |
the results of clinical trials may not meet the level of statistical significance or clinical significance required by the FDA for approval;
|
• |
the FDA may disagree with the number, design, size, conduct, or implementation of our clinical trials;
|
• |
the FDA may not find the data from preclinical studies and clinical trials sufficient to demonstrate that our product candidates’ clinical and other benefits outweigh the safety risks;
|
• |
the FDA may disagree with our interpretation of data from preclinical studies or clinical trials;
|
• |
the FDA may not accept data generated at our clinical trial sites;
|
• |
the FDA may have difficulties scheduling an advisory committee meeting in a timely manner or the advisory committee may recommend against approval of our application or may recommend that the FDA require, as a condition of approval,
additional preclinical studies or clinical trials, limitations on approved labeling or distribution and use restrictions;
|
• |
the FDA may require development of a Risk Evaluation and Mitigation Strategy (REMS) as a condition of approval;
|
• |
the FDA may identify deficiencies in the manufacturing processes or facilities of third-party manufacturers with which we entered or enter into agreements for clinical and commercial supplies; or
|
• |
the FDA may change its approval policies or adopt new regulations.
|
• |
regulators or IRBs may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site including due to the ongoing COVID-19 pandemic or other public health emergency;
|
• |
government or regulatory delays and changes in regulatory requirements, policy and guidelines may require us to perform additional clinical trials or use substantial additional resources to obtain regulatory approval;
|
• |
we may have delays in reaching or fail to reach agreement on acceptable clinical trial contracts or clinical trial protocols with prospective trial sites;
|
• |
clinical trials may produce negative or inconclusive results, and we may decide, or regulators may require it, to conduct additional clinical trials or abandon product development programs;
|
• |
the number of patients required for clinical trials may be larger, enrollment in these clinical trials may be slower or participants may drop out of these clinical trials at a higher rate than we anticipate;
|
• |
our third-party contractors may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all;
|
• |
our patients or medical investigators may be unwilling to follow our clinical trial protocols;
|
• |
we might have to suspend or terminate clinical trials for various reasons, including a finding that the participants are being exposed to unacceptable health risks;
|
• |
the cost of clinical trials may be greater than we anticipate;
|
• |
the supply or quality of any product candidate or other materials necessary to conduct clinical trials may be insufficient or inadequate;
|
• |
the product candidate may have undesirable side effects or other unexpected characteristics, causing us or our investigators, regulators or IRBs to suspend or terminate the trials;
|
• |
clinical trials may be delayed or terminated ; and
|
• |
federal agencies may, due to reduced manpower or diverted resources, require more time to review clinical trial protocols and INDs.
|
• |
severity of the disease under investigation;
|
• |
availability and efficacy of medications already approved for the disease under investigation;
|
• |
eligibility criteria and visit schedule for the trial in question;
|
• |
competition for eligible patients with other companies conducting clinical trials for product candidates seeking to treat the same indication or patient population;
|
• |
our payments for conducting clinical trials;
|
• |
perceived risks and benefits of the product candidate under study;
|
• |
efforts to facilitate timely enrollment in clinical trials;
|
• |
patient referral practices of physicians;
|
• |
the ability to monitor patients adequately during and after treatment;
|
• |
proximity and availability of clinical trial sites for prospective patients; and
|
• |
regulatory authorities may withdraw their approval of the product;
|
• |
we may be required to recall the product, change the way this product is administered, conduct additional clinical trials, or change the labeling or distribution of the product (including REMS);
|
• |
additional restrictions may be imposed on the marketing of, or the manufacturing processes for, the product;
|
• |
we may be subject to fines, injunctions, or the imposition of civil or criminal penalties;
|
• |
we could be sued and held liable for harm caused to patients;
|
• |
the product may be rendered less competitive and sales may decrease; or
|
• |
our reputation may suffer generally among both clinicians and patients.
|
• |
obtain favorable results from and complete the clinical development of APX3330 for their planned indications, including successful completion of additional clinical trials for these indications;
|
• |
submit applications to regulatory authorities for both product candidates and receive timely marketing approvals in the United States and foreign countries;
|
• |
establish and maintain commercially viable supply and manufacturing relationships with third parties that can provide adequate, in both amount and quality, products and services to support clinical development and meet the market
demand for product candidates that we develop, if approved;
|
• |
establish sales and marketing capabilities to effectively market and sell our product candidates in the United States or other markets, either alone or with a pharmaceutical partner;
|
• |
address any competing products and technological and market developments;
|
• |
obtain coverage and adequate reimbursement for customers and patients from government and third-party payors for product candidates that we develop; and
|
• |
achieve market acceptance of our product candidates.
|
• |
Delayed access to deposits or other financial assets or the uninsured loss of deposits or other financial assets;
|
• |
Loss of access to revolving existing credit facilities or other working capital sources and/or the inability to refund, roll over or extend the maturity of, or enter into new credit facilities or other
working capital resources;
|
• |
Potential or actual breach of contractual obligations that require us to maintain letters or credit or other credit support arrangements; or
|
• |
Termination of cash management arrangements and/or delays in accessing or actual loss of funds subject to cash management arrangements.
|
• |
the scope, size, rate of progress, results, and costs of researching and developing our product candidates, and initiating and completing our preclinical studies and clinical trials;
|
• |
the cost, timing and outcome of our efforts to obtain marketing approval for our product candidates in the United States and other countries, including to fund the preparation and filing of NDAs with the FDA for our product candidates
and to satisfy related FDA requirements and regulatory requirements in other countries;
|
• |
the number and characteristics of any additional product candidates we develop or acquire, if any;
|
• |
our ability to establish and maintain collaborations on favorable terms, if at all;
|
• |
the amount of revenue, if any, from commercial sales, should our product candidates receive marketing approval;
|
• |
the costs associated with commercializing our product candidates, if we receive marketing approval, including the cost and timing of developing sales and marketing capabilities or entering into strategic collaborations to market and
sell our product candidates;
|
• |
the cost of manufacturing our product candidates or products we successfully commercialize; and
|
• |
the costs associated with general corporate activities, such as the cost of filing, prosecuting and enforcing patent claims and making regulatory filings.
|
• |
litigation involving patients taking our drugs;
|
• |
restrictions on such drugs, manufacturers, or manufacturing processes;
|
• |
restrictions on the labeling or marketing of a drug;
|
• |
restrictions on drug distribution or use;
|
• |
requirements to conduct post-marketing studies or clinical trials;
|
• |
warning letters or untitled letters;
|
• |
withdrawal of the drugs from the market;
|
• |
refusal to approve pending applications or supplements to approved applications that we submit;
|
• |
product recall or public notification or medical product safety alerts to healthcare professionals;
|
• |
fines, restitution, or disgorgement of profits or revenues;
|
• |
suspension or withdrawal of marketing approvals;
|
• |
damage to relationships with any potential collaborators;
|
• |
unfavorable press coverage and damage to our reputation;
|
• |
refusal to permit the import or export of drugs;
|
• |
product seizure; or
|
• |
injunctions or the imposition of civil or criminal penalties.
|
• |
the federal Anti-Kickback Statute prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or
reward, or in return for, either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under a federal healthcare program such as Medicare and Medicaid;
|
• |
the federal false claims and civil monetary penalties laws, including the civil False Claims Act, impose criminal and civil penalties, including civil whistleblower or qui tam actions, against individuals or entities for knowingly
presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulent or making a false statement to avoid, decrease, or conceal an obligation to pay money to the federal government;
|
• |
HIPAA imposes criminal and civil liability for, among other things, executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters;
|
• |
HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, and its implementing regulations, also imposes obligations, including mandatory contractual terms, on certain people and entities with respect
to safeguarding the privacy, security, and transmission of individually identifiable health information;
|
• |
the federal Physician Payments Sunshine Act under the Affordable Care Act requires certain manufacturers of drugs, devices, biologics, and medical supplies for which payment is available under Medicare, Medicaid, or the Children’s
Health Insurance Program, with specific exceptions, to report specially to the Centers for Medicare & Medicaid Services within the U.S. Department of Health and Human Services information related to physician payments and other
transfers of value and physician ownership and investment interests; and
|
• |
analogous state and foreign laws and regulations, such as state anti-kickback and false claims laws, may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental
third-party payors, including private insurers, and some state laws require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the
federal government, in addition to requiring drug manufacturers to report information related to payments to physicians and other healthcare providers or marketing expenditures. Certain state and foreign laws also govern the privacy and
security of health information in ways that differ from each other and often are not preempted by HIPAA, thus complicating compliance efforts.
|
• |
comply with the regulations of the FDA and applicable non-U.S. regulators;
|
• |
provide accurate information to the FDA and applicable non-U.S. regulators;
|
• |
comply with healthcare fraud and abuse laws and regulations in the United States and abroad;
|
• |
report financial information or data accurately; or
|
• |
disclose unauthorized activities to us.
|
• |
Lucentis® (ranibizumab) and Avastin® (bevacizumab), which are anti-VEGF monoclonal antibody intravitreal injections, developed by Genentech, Inc and
Roche AG.
|
• |
EYLEA® (aflibercept), a VEGF inhibitor intravitreal injection, developed by Regeneron Pharmaceuticals.
|
• |
Vabysmo® (Faricimab), a bispecific monoclonal antibody targeting VEGF-A and Ang-Tie2 pathway developed by Genentech, Inc and Roche AG.
|
• |
Beovu® (Brolucizumab), an anti-VEGF monoclonal antibody intravitreal injection, developed by Novartis AG.
|
• |
MACUGEN® (pegaptanib sodium injection), a selective inhibitor of VEGF-165, developed by Bausch + Lomb.
|
• |
Ozurdex® (dexamethasone), a corticosteroid IVT implant, developed by Allergan plc.
|
• |
Iluvien (fluocinolone acetonide), a corticosteroid IVT implant, developed by Alimera Sciences, Inc
|
• |
Abicipar, an anti-VEGF intravitreal injection with a long duration of action, developed by Allergan plc and Molecular Partners.
|
• |
KSI-301, an anti-VEGF antibody intravitreal injection coupled with a biopolymer that is intended to increase the time between injections, developed by Kodiak Sciences.
|
• |
OPT-302, an intravitreal injection which binds to multiple types of VEGF receptors that could be used with other anti-VEGF agents, developed by Opthea Limited.
|
• |
ALG-1001, an integrin peptide therapy intravitreal injection that is being evaluated as a sequential or in-combination therapy with bevacizumab in patients with DME, developed by Allegro Ophthalmics, LLC.
|
• |
RG-7774, an orally administered selective CB2 (Cannabinoid 2) receptor agonist that is being evaluated in patients with moderately severe to severe non-proliferative diabetic retinopathy, developed by Hoffmann-LA Roche, AG.
|
• |
RZ402, a small molecule selective and potent plasma kallikrein inhibitor (PKI) for the chronic treatment of diabetic macular edema (DME), developed by Rezolute, Inc.
|
• |
Xiflam™, an oral small molecule drug for the treatment of dry form of Age-Related Macular Degeneration (AMD), Geographic Atrophy (GA), Diabetic Retinopathy (DR) manifesting Diabetic Macular Edema (DME), developed by InflammX.
|
• |
AKST4290, an oral small molecule CCR3 Eotaxin inhibitor for the treatment of diabetic retinopathy and wet AMD.
|
• |
BAY1101042, an oral guanylate cycles activator for the treatment of diabetic retinopathy.
|
• |
CSF-1, with low dose pilocarpine and a secondary agent (lubricant), developed by Orasis Pharmaceuticals Ltd.
|
• |
LNZ100 and LNZ101, with aceclidine (another miotic agent), developed by Lenz Therapeutics.
|
• |
MicroLine®, which is a micro-dose delivery of pilocarpine using proprietary device developed by Eyenovia, Inc.
|
• |
KT-101, which uses pilocarpine in the AcuStream delivery system, developed by Kedalion Therapeutics, Inc.
|
• |
BrimocholTM, with brimonidine and carbachol (both are miotic agents), developed by Visus Therapeutics, Inc.
|
• |
UNR844, which uses a mechanism that involves softening the lens to increase near visual acuity, developed by Novartis AG (originally Encore Vision, Inc.).
|
• |
the inability to recruit and retain adequate numbers of effective sales and marketing personnel or enter into distribution agreements with third parties;
|
• |
the inability of sales personnel to obtain access to physicians or persuade adequate numbers of physicians to prescribe our product candidate;
|
• |
the lack of complementary products to be offered by sales personnel, which may put us at a competitive disadvantage relative to companies with more extensive product lines;
|
• |
unforeseen costs and expenses associated with creating an independent sales and marketing organization; and
|
• |
the inability to obtain sufficient coverage and reimbursement from third-party payors and governmental agencies.
|
• |
If we enter into arrangements with third parties to perform sales, marketing, and distribution services, our product revenues or the profitability of these product revenues to us are likely to be lower than if we were to market and
sell a product that we developed ourselves. In addition, we may not be successful in entering into arrangements with third parties to sell and market any product candidate or may be unable to do so on terms that are favorable to us. We
likely will have little control over such third parties, and any of them may fail to devote the necessary resources and attention to sell and market a drug effectively. If we do not establish sales and marketing capabilities successfully,
either on our own or in collaboration with third parties, we will not be successful in commercializing our product candidates.
|
• |
efficacy and potential advantages compared to alternative treatments;
|
• |
the ability to offer our product for sale at competitive prices;
|
• |
the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies;
|
• |
any restrictions on the use of our product together with other medications;
|
• |
interactions of our product with other medicines patients are taking;
|
• |
inability of certain types of patients to take our product;
|
• |
demonstrated ability to treat patients and, if required by any applicable regulatory authority in connection with the approval for target indications as compared with other available therapies;
|
• |
the relative convenience and ease of administration as compared with other treatments available for approved indications;
|
• |
the prevalence and severity of any adverse side effects;
|
• |
limitations or warnings contained in the labeling approved by the FDA;
|
• |
availability of alternative treatments already approved or expected to be commercially launched in the near future;
|
• |
the effectiveness of our sales and marketing strategies;
|
• |
our ability to increase awareness through marketing efforts;
|
• |
guidelines and recommendations of organizations involved in research, treatment and prevention of various diseases that may advocate for alternative therapies;
|
• |
our ability to obtain sufficient third-party coverage and adequate reimbursement;
|
• |
the willingness of patients to pay out-of-pocket in the absence of third-party coverage; and
|
• |
physicians or patients may be reluctant to switch from existing therapies even if potentially more effective, safe or convenient.
|
• |
decreased demand for any product candidate that we are developing;
|
• |
injury to our reputation and significant negative media attention;
|
• |
withdrawal of clinical trial participants;
|
• |
increased FDA warnings on product labels;
|
• |
significant costs to defend the related litigation;
|
• |
substantial monetary awards to trial participants or patients;
|
• |
distraction of management’s attention from our primary business;
|
• |
loss of revenue; and
|
• |
the inability to commercialize any product candidate that we may develop.
|
• |
have staffing difficulties;
|
• |
fail to comply with contractual obligations;
|
• |
experience regulatory compliance issues;
|
• |
undergo changes in ownership or management;
|
• |
undergo changes in priorities or become financially distressed; or
|
• |
form relationships with other entities, some of which may be our competitors.
|
• |
collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations;
|
• |
collaborators may not perform their obligations as expected;
|
• |
collaborators may not pursue development and commercialization or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborator’s strategic focus or available
funding, or external factors such as an acquisition that diverts resources or creates competing priorities;
|
• |
collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials, or require a new formulation of a product
candidate for clinical testing;
|
• |
collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our product candidate if the collaborators believe that competitive products are more likely to be successfully
developed or can be commercialized under terms that are more attractive than ours;
|
• |
a collaborator with marketing and distribution rights to one or more product candidates may not commit sufficient resources to the marketing or distribution of any such product candidate;
|
• |
collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our proprietary information or expose
us to litigation;
|
• |
collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability;
|
• |
disputes may arise between us and collaborators that result in the delay or termination of research, development, or commercialization of our product candidates, or in litigation or arbitration that diverts management attention and
resources;
|
• |
we may lose certain valuable rights under circumstances identified in our collaborations, including if we undergo a change of control;
|
• |
collaborations may be terminated and such terminations may create a need for additional capital to pursue further development or commercialization of the applicable product candidates;
|
• |
collaborators may learn about our discoveries and use this knowledge to compete with us in the future;
|
• |
the results of collaborators’ preclinical or clinical studies could harm or impair other development programs;
|
• |
there may be conflicts between different collaborators that could negatively affect those collaborations and potentially others;
|
• |
the number and nature of our collaborations could adversely affect our attractiveness to potential future collaborators or acquirers;
|
• |
collaboration agreements may not lead to development or commercialization of our product candidate in the most efficient manner or at all. If a present or future collaborator of us were to be involved in a business combination, the
continued pursuit and emphasis on our product development or commercialization program under such collaboration could be delayed, diminished, or terminated; and
|
• |
collaborators may be unable to obtain the necessary marketing approvals.
|
• |
increased operating expenses and cash requirements;
|
• |
the assumption of indebtedness or contingent liabilities;
|
• |
the issuance of our equity securities which would result in dilution to our stockholders;
|
• |
assimilation of operations, intellectual property, products and product candidates of an acquired company, including difficulties associated with integrating new personnel;
|
• |
the diversion of management’s attention from our existing product candidates and initiatives in pursuing such an acquisition or strategic partnership;
|
• |
retention of key employees, the loss of key personnel, and uncertainties in our ability to maintain key business relationships;
|
• |
risks and uncertainties associated with the other party to such a transaction, including the prospects of that party and their existing products or product candidates and regulatory approvals; and
|
• |
our inability to generate revenue from acquired intellectual property, technology and/or products sufficient to meet our objectives or even to offset the associated transaction and maintenance costs.
|
• |
any of our patents, or any of our pending patent applications, if issued, will include claims having a scope sufficient to protect our product candidates;
|
• |
any of our pending patent applications will result in issued patents;
|
• |
we will be able to successfully commercialize our product candidates, if approved, before our relevant patents expire;
|
• |
we were the first to make the inventions covered by each of our patents and pending patent applications;
|
• |
we were the first to file patent applications for these inventions;
|
• |
others will not develop similar or alternative technologies that do not infringe our patents;
|
• |
any of our patents will be valid and enforceable;
|
• |
any patents issued to us will provide a basis for an exclusive market for our commercially viable products, will provide us with any competitive advantages or will not be challenged by third parties;
|
• |
we will develop additional proprietary technologies or product candidates that are separately patentable; or
|
• |
our commercial activities or products will not infringe upon the patents of others.
|
• |
the scope of rights granted under the license agreement and other interpretation-related issues;
|
• |
the extent to which our product candidates, technology and processes infringe on intellectual property of the licensor that is not subject to the licensing agreement;
|
• |
the sublicensing of patent and other rights under our collaborative development relationships;
|
• |
our diligence obligations under the license agreement and what activities satisfy those diligence obligations;
|
• |
the inventorship and ownership of inventions and know-how resulting from the joint creation or use of intellectual property; and
|
• |
the priority of invention of patented technology.
|
• |
compliance with differing or unexpected regulatory requirements for our product candidates;
|
• |
different medical practices and customs affecting acceptance of our product candidates, if approved, or any other approved product in the marketplace;
|
• |
language barriers;
|
• |
the interpretation of contractual provisions governed by foreign law in the event of a contract dispute;
|
• |
difficulties in staffing and managing foreign operations, and an inability to control commercial or other activities where it is relying on third parties;
|
• |
workforce uncertainty in countries where labor unrest is more common than in the United States;
|
• |
potential liability under the Foreign Corrupt Practice Act of 1977 or comparable foreign regulations;
|
• |
production shortages resulting from any events affecting raw material supply or manufacturing capability abroad;
|
• |
foreign government taxes, regulations, and permit requirements;
|
• |
U.S. and foreign government tariffs, trade restrictions, price and exchange controls, and other regulatory requirements;
|
• |
economic weakness, including inflation, natural disasters, war, events of terrorism, or political instability in particular foreign countries;
|
• |
fluctuations in currency exchange rates, which could result in increased operating expenses and reduced revenues;
|
• |
compliance with tax, employment, immigration, and labor laws, regulations, and restrictions for employees living or traveling abroad;
|
• |
changes in diplomatic and trade relationships; and
|
• |
challenges in enforcing our contractual and intellectual property rights, especially in those foreign countries that do not respect and protect intellectual property rights to the same extent as the United States.
|
• |
the announcement of new products or product enhancements by us or our competitors;
|
• |
changes in our relationships with our licensors or other strategic partners;
|
• |
developments concerning intellectual property rights and regulatory approvals;
|
• |
variations in ours and our competitors’ results of operations;
|
• |
substantial sales of shares of our common stock due to the release of lock-up agreements;
|
• |
the announcement of clinical trial results;
|
• |
the announcement of potentially dilutive financings;
|
• |
changes in earnings estimates or recommendations by securities analysts;
|
• |
changes in the structure of healthcare payment systems; and
|
• |
developments and market conditions in the pharmaceutical and biotechnology industries, including due to the COVID-19 pandemic.
|
ITEM 5.
|
MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES
|
•
|
continue clinical trials for Nyxol, APX3330 and for any other product candidate in our future pipeline;
|
•
|
continue preclinical studies for Nyxol, APX3330 and for any other product candidate in our future pipeline;
|
•
|
develop additional product candidates that we identify, in-license or acquire;
|
•
|
seek regulatory approvals for any product candidates that successfully complete clinical trials;
|
•
|
contract to manufacture our product candidates;
|
•
|
maintain, expand and protect our intellectual property portfolio;
|
•
|
hire additional staff, including clinical, scientific, operational and financial personnel, to execute our business plan;
|
•
|
add operational, financial and management information systems and personnel, including personnel to support our product development and potential future commercialization efforts;
|
•
|
continue to operate as a public company; and
|
•
|
establish on our own or with partners, a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain regulatory approval;
|
• |
per patient trial costs;
|
• |
the number of patients that participate in the trials;
|
• |
the number of sites included in the trials;
|
• |
the countries in which the trials are conducted;
|
• |
the length of time required to enroll eligible patients;
|
• |
the number of doses that patients receive;
|
• |
the drop-out or discontinuation rates of patients;
|
• |
potential additional safety monitoring or other studies requested by regulatory agencies;
|
• |
the duration of patient follow-up;
|
• |
the phase of development of the product candidate;
|
• |
arrangements with contract research organizations and other service providers; and
|
• |
the efficacy and safety profile of the product candidates.
|
For the Year Ended
December 31,
|
||||||||||||
2022
|
2021
|
Change
|
||||||||||
License and collaborations revenue
|
$
|
39,850
|
$
|
589
|
$
|
39,261
|
||||||
Operating expenses:
|
||||||||||||
General and administrative
|
7,269
|
8,121
|
(852
|
)
|
||||||||
Research and development
|
14,355
|
15,173
|
(818
|
)
|
||||||||
Total operating expenses
|
21,624
|
23,294
|
(1,670
|
)
|
||||||||
Income (loss) from operations
|
18,226
|
(22,705
|
)
|
40,931
|
||||||||
Interest expense
|
(9
|
)
|
(2
|
)
|
(7
|
)
|
||||||
Fair value change in warrant liabilities
|
—
|
(33,829
|
)
|
33,829
|
||||||||
Other expense, net
|
(14
|
)
|
(157
|
)
|
143
|
|||||||
Income (loss) before income taxes
|
18,203
|
(56,693
|
)
|
74,896
|
||||||||
Provision for income taxes
|
(315
|
)
|
—
|
(315
|
)
|
|||||||
Net income (loss)
|
$
|
17,888
|
$
|
(56,693
|
)
|
$
|
74,581
|
For the Year Ended
December 31,
|
||||||||
2022
|
2021
|
|||||||
Net cash provided by (used in) operating activities
|
$
|
14,314
|
$
|
(19,370
|
)
|
|||
Net cash used in investing activities
|
—
|
(100
|
)
|
|||||
Net cash provided by financing activities
|
3,786
|
27,605
|
||||||
Net increase in cash and cash equivalents
|
$
|
18,100
|
$
|
8,135
|
ITEM 12.
|
SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER MATTERS
|
ITEM 15. |
EXHIBITS AND FINANCIAL STATEMENT SCHEDULES
|
(a)
|
Financial Statements: The financial statements filed as part of this report are listed in Part II, Item 8.
|
(b)
|
Financial Statement Schedules: The schedules are either not applicable or the required information is presented in the consolidated financial statements or notes thereto.
|
(c)
|
Exhibits: The following exhibits are incorporated by reference or filed as part of this Annual Report on Form 10-K:
|
EXHIBIT
NUMBER
|
|
DESCRIPTION OF DOCUMENT
|
|
Agreement and Plan of Merger, dated as of June 17, 2020, by and among the Registrant, Razor Merger Sub, Inc. and Ocuphire Pharma, Inc. (incorporated by reference to Exhibit 2.1 to the Registrant’s Current
Report on Form 8-K, filed on June 19, 2020).
|
|
|
First Amendment to Agreement and Plan of Merger and Reorganization, dated as of June 29, 2020, by and among Rexahn, Merger Sub and Ocuphire (incorporated by reference to Exhibit 2.1 to the Registrant’s
Current Report on Form 8-K , filed on July 1, 2020).
|
|
|
Amended and Restated Certificate of Incorporation of the Registrant (incorporated by reference to Appendix G to the Registrant’s Definitive Proxy Statement on Schedule 14A, filed on April 29, 2005).
|
|
|
Certificate of Amendment of Amended and Restated Certificate of Incorporation of the Registrant (incorporated by reference to Exhibit 3.1 to the Registrant’s Current Report on Form 8-K, filed on May 5,
2017).
|
|
|
Certificate of Amendment of Amended and Restated Certificate of Incorporation of the Registrant (incorporated by reference to Exhibit 3.1 to the Registrant’s Current Report on Form 8-K, filed on August 30,
2018).
|
|
|
Certificate of Amendment of Amended and Restated Certificate of Incorporation of the Registrant (incorporated by reference to Exhibit 3.1 to the Registrant’s Current Report on Form 8-K, filed on April 12,
2019).
|
|
|
Certificate of Amendment of Amended and Restated Certificate of Incorporation (incorporated by reference to Exhibit 3.1 to the Registrant’s Current Report on Form 8-K, filed on November 6, 2020).
|
|
|
Certificate of Amendment of Amended and Restated Certificate of Incorporation (incorporated by reference to Exhibit 3.2 to the Registrant’s Current Report on Form 8-K, filed on November 6, 2020).
|
|
|
Second Amended and Restated Bylaws of the Registrant (incorporated by reference to Exhibit 3.3 to the Registrant’s Current Report on Form 8-K, filed on November 6, 2020).
|
|
Amendment to Second Amended and Restated Bylaws of Ocuphire Pharma, Inc. (incorporated by reference to Exhibit 3.1 to the Registrant’s Current Report on Form 8-K, filed on June 10, 2022).
|
||
Second Amendment to Second Amended and Restated Bylaws of Ocuphire Pharma, Inc. (incorporated by reference to Exhibit 3.1 to the Registrant’s Current Report on Form 8-K, filed on June 17, 2022).
|
||
|
Specimen Certificate for the Registrant’s Common Stock, par value $.0001 per share (incorporated by reference to Exhibit 4.3 to the Registrant’s Registration Statement on Form S-8 (File No. 333-129294),
filed on October 28, 2005).
|
|
|
Form of Common Stock Purchase Warrant (incorporated by reference to Exhibit 4.1 to the Registrant’s Current Report on Form 8-K, filed on October 13, 2017).
|
|
|
Form of Common Stock Purchase Warrant (incorporated by reference to Exhibit 4.1 to the Registrant’s Current Report on Form 8-K, filed on October 19, 2018).
|
|
Form of Common Stock Purchase Warrant (incorporated by reference to Exhibit 4.1 to the Registrant’s Current Report on Form 8-K, filed on January 25, 2019).
|
|
|
Form of Series A/B Warrants (incorporated by reference to Exhibit 4.1 to the Registrant’s Current Report on Form 8-K, filed on July 1, 2020).
|
|
|
Description of Securities (incorporated by reference to Exhibit 4.11 to the Registrant’s Annual Report on Form 10-K, filed on March 11, 2021).
|
|
Form of Warrant to purchase shares of common stock (incorporated by reference to Exhibit 4.1 to the Registrant’s Current Report on Form 8-K/A, filed on June 7, 2021).
|
||
|
Amended and Restated Employment Agreement by and among the Company and Mina Sooch, effective as of November 5, 2020 (incorporated by reference to Exhibit 10.27 to the Registrant’s Registration Statement on
Form S-4 (File No. 333-239702), filed on September 30, 2020).
|
|
First Amendment to the Amended and Restated Employment Agreement by and among the Company and Mina Sooch, effective as of March 26, 2023.
|
||
|
Amended and Restated Employment Agreement by and among the Company and Bernhard Hoffmann, effective as of November 5, 2020 (incorporated by reference to Exhibit 10.29 to the Registrant’s Registration
Statement on Form S-4 (File No. 333-239702), filed on September 30, 2020).
|
|
First Amendment to the Amended and Restated Employment Agreement by and among the Company and Bernhard Hoffmann, effective as of March 26,
2023.
|
||
|
Form of Indemnification Agreement (incorporated by reference to Exhibit 10.30 to the Registrant’s Registration Statement on Form S-4 (File No. 333-239702), filed on September 30, 2020).
|
|
|
Sublicense Agreement, dated as of January 21, 2020, by and between Ocuphire Pharma, Inc. and Apexian Pharmaceuticals, Inc (incorporated by reference to Exhibit 10.31 to the Registrant’s Registration
Statement on Form S-4 (File No. 333-239702), filed on September 30, 2020).
|
|
|
First Amendment to Sublicense Agreement, dated as of June 4, 2020, by and between Apexian Pharmaceuticals, Inc. and Ocuphire Pharma, Inc (incorporated by reference to Exhibit 10.32 to the Registrant’s
Registration Statement on Form S-4 (File No. 333-239702), filed on September 30, 2020).
|
|
|
Lease Agreement, dated as of May 19, 2019, by and between Ocuphire Pharma, Inc. and Duke & Duke, LP (incorporated by reference to Exhibit 10.33 to the Registrant’s Registration Statement on Form S-4
(File No. 333-239702), filed on September 30, 2020).
|
|
|
First Amendment to Lease Agreement, dated as of October 29, 2019, by and between Ocuphire Pharma, Inc. and Duke & Duke, LP (incorporated by reference to Exhibit 10.34 to the Registrant’s Registration
Statement on Form S-4 (File No. 333-239702), filed on September 30, 2020).
|
|
Second Lease Amendment, dated as of November 17, 2020, by and between the Company and Duke & Duke (incorporated by reference to Exhibit 10.43 to the Registrant’s Annual Report on Form 10-K, filed on
March 11, 2021).
|
||
Third Lease Amendment, dated as of September 9, 2021, by and between the Company and Duke & Duke (incorporated by reference to Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q, filed on
November 12, 2021).
|
||
Fourth Lease Amendment, dated as of October 17, 2022, by and between the Company and Duke & Duke (incorporated by reference to Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q, filed on
November 4, 2022).
|
||
|
Ocuphire Pharma, Inc. 2018 Equity Incentive Plan, dated as of April 9, 2018 (incorporated by reference to Exhibit 10.35 to the Registrant’s Registration Statement on Form S-4 (File No. 333-239702), filed on
September 30, 2020).
|
|
|
First Amendment to 2018 Equity Incentive Plan, dated as of December 23, 2019 (incorporated by reference to Exhibit 10.36 to the Registrant’s Registration Statement on Form S-4 (File No. 333-239702), filed
on September 30, 2020).
|
Form of Option Agreement issuable under the Ocuphire Pharma, Inc. 2018 Equity Incentive Plan (incorporated by reference to Exhibit 10.37 to the Registrant’s Registration Statement on Form S-4 (File No.
333-239702), filed on September 30, 2020).
|
||
|
Ocuphire Pharma, Inc. 2020 Equity Incentive Plan (incorporated by reference to Exhibit 10.38 to the Registrant’s Registration Statement on Form S-4 (File No. 333-239702), filed on September 30, 2020).
|
|
Form of Restricted Stock Unit Grant Notice issued under the Ocuphire Pharma, Inc. 2020 Equity Incentive Plan.
|
||
Form of Stock Option Grant Notice issued under the Ocuphire Pharma, Inc. 2020 Equity Incentive Plan.
|
||
|
Contingent Value Rights Agreement, dated as of November 5, 2020, by and among the Company, Shareholder Representative Services LLC and the Olde Monmouth Stock Transfer Co., Inc. (incorporated by reference
to Exhibit 10.4 to the Registrant’s Current Report on Form 8-K, filed on November 6, 2020).
|
|
|
Ocuphire Pharma, Inc. 2021 Inducement Plan (incorporated by reference to Exhibit 10.41 to the Registrant’s Annual Report on Form 10-K, filed on March 11, 2021)
|
|
Form of Stock Option Grant Notice issued under the Ocuphire Pharma, Inc. 2021 Inducement Plan
|
||
|
Employment Agreement dated November 11, 2020, by and between the Company and Amy Rabourn (incorporated by reference to Exhibit 10.42 to the Registrant’s Annual Report on Form 10-K, filed on March 11, 2021)
|
|
First Amendment to the Employment Agreement by and among the Company and Amy Rabourn, effective as of March 26, 2023.
|
||
Capital on Demand™ Sales Agreement, dated March 11, 2021 between the Company and JonesTrading Institutional Services LLC (incorporated by reference to Exhibit 1.1 to the Registrant’s Current Report on Form
8-K, filed on March 11, 2021).
|
||
Form of Securities Purchase Agreement, dated as of June 4, 2021, by and among Ocuphire Pharma, Inc. and the purchasers identified on the signature pages thereto (incorporated by reference to Exhibit 10.1 to
the Registrant’s Current Report on Form 8-K/A, filed on June 7, 2021).
|
||
Processa License Agreement dated June 16, 2021 (incorporated by reference to Exhibit 10.1 to the Registrant’s Current Report on Form 8-K, filed on June 23, 2021).
|
||
Famy License and Collaboration Agreement dated November 6, 2022 (incorporated by reference to Exhibit 10.1 to the Registrant’s Current Report on Form 8-K, filed on November 7, 2022).
|
||
Consulting Agreement dated April 8, 2022, by and between the Company and Jay Pepose (incorporated by reference to Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q, filed on May 13, 2022).
|
||
First Amendment to the Consulting Agreement dated September 19, 2022, by and between the Company and Jay Pepose.
|
||
Second Amendment to the Consulting Agreement dated December 1, 2022, by and between the Company and Jay Pepose.
|
||
Amended and Restated Non-Employee Director Compensation Policy dated July 1, 2022 (incorporated by reference to Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q, filed on August 12, 2022).
|
||
|
Subsidiaries of the Registrant.
|
|
|
Consent of Ernst & Young, LLP.
|
|
Certification of Principal Executive Officer pursuant to Rules 13a-14(a) and 15d-14(a) promulgated under the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of
2002.
|
|
|
Certification of Principal Financial Officer pursuant to Rules 13a-14(a) and 15d-14(a) promulgated under the Securities Exchange Act of 1934, as adopted pursuant to section 302 of the Sarbanes-Oxley Act of
2002.
|
|
|
Certification of Principal Executive Officer and Principal Financial Officer pursuant to Rules 13a-14(b) and 15d-14(b) promulgated under the Securities Exchange Act of 1934 and 18 U.S.C. Section 1350, as
adopted pursuant to section 906 of The Sarbanes-Oxley Act of 2002.
|
|
101.INS
|
Inline XBRL Instance Document (the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document).
|
|
101.SCH
|
Inline XBRL Taxonomy Extension Schema Document
|
|
101.CAL
|
Inline XBRL Taxonomy Extension Calculation Linkbase Document
|
|
101.DEF
|
Inline XBRL Taxonomy Extension Definition Linkbase Document
|
|
101.LAB
|
Inline XBRL Taxonomy Extension Label Linkbase Document
|
|
101.PRE
|
Inline XBRL Taxonomy Extension Presentation Linkbase Document
|
|
104
|
Cover Page Interactive Data File (formatted as inline XBRL and contained in Exhibit 101)
|
* |
Indicates management contract or compensatory plan.
|
+ |
Certain schedules and exhibits have been omitted pursuant to Item 601(b)(2) of Regulation S-K. A copy of any omitted schedule and/or exhibit will be furnished to the SEC upon request.
|
Measurement of License and Collaboration revenue
|
|
Description of the Matter
|
As discussed in Notes 1 and 10 to the consolidated financial statements, the Company entered into a license and collaboration agreement on November 6, 2022. The agreement was assessed under the recognition and measurement rules of
Accounting Standards Codification 606, Revenue from Contracts with Customers. The Company identified two performance obligations within the arrangement: (i) a license to intellectual property and (ii) research and development services.
The license and collaboration agreement transaction price was allocated between the performance obligations based on their relative standalone selling prices, which were estimated using a discounted royalty cash flow approach and an
expected cost plus margin approach. For the year ended December 31, 2022, the Company recognized $39.8 million of revenue related to this agreement.
Auditing the Company’s accounting for the license and collaboration agreement was complex due to the judgment used by management to determine the license of
intellectual property’s relative standalone selling price valuation assumptions. These assumptions include, but were not limited to discount rates, future royalty cash flows and development milestones. These assumptions are
forward-looking and could be affected by future economic and market conditions.
|
How we Addressed the
Matter in Our Audit
|
To test the measurement of license and collaboration revenue, our audit procedures included, among others, inspecting the license and collaboration agreement, assessing the completeness and accuracy of the Company’s technical
accounting analyses, evaluating the selection of relative standalone selling price valuation methodology and assumptions, and testing the underlying data. For example, we compared the assumption of anticipated future royalty cash flows
to current industry, market and economic trends. Our audit procedures also included the involvement of valuation specialist resources to assist in evaluating management’s valuation methodology and relative standalone selling price
calculation.
|
As of December 31,
|
||||||||
2022
|
2021
|
|||||||
Assets
|
||||||||
Current assets:
|
||||||||
Cash and cash equivalents
|
$
|
42,634
|
$
|
24,534
|
||||
Accounts receivable (Note 10)
|
1,298 | — | ||||||
Contract asset (Note 10)
|
3,552 | — | ||||||
Prepaids and other current assets
|
1,453
|
1,314
|
||||||
Short-term investments
|
49
|
219
|
||||||
Total current assets
|
48,986
|
26,067
|
||||||
Property and equipment, net
|
6
|
10
|
||||||
Total assets
|
$
|
48,992
|
$
|
26,077
|
||||
Liabilities and stockholders’ equity
|
||||||||
Current liabilities:
|
||||||||
Accounts payable
|
$
|
1,069
|
$
|
1,584
|
||||
Accrued expenses
|
1,684
|
1,733
|
||||||
Short-term loan
|
—
|
538
|
||||||
Total current liabilities
|
2,753
|
3,855
|
||||||
Warrant liabilities
|
—
|
—
|
||||||
Total liabilities
|
2,753
|
3,855
|
||||||
Commitments and contingencies (Note 4 and Note 8)
|
||||||||
Stockholders’ equity
|
||||||||
Preferred stock, par value $0.0001; 10,000,000 shares authorized as of December 31, 2022 and 2021; no shares issued and outstanding at December 31, 2022
and 2021.
|
—
|
—
|
||||||
Common stock, par value $0.0001; 75,000,000 shares authorized as of December 31, 2022 and 2021; 20,861,315 and 18,845,828 shares issued
and outstanding at December 31, 2022 and 2021, respectively.
|
2
|
2
|
||||||
Additional paid-in capital
|
117,717
|
111,588
|
||||||
Accumulated deficit
|
(71,480
|
)
|
(89,368
|
)
|
||||
Total stockholders’ equity
|
46,239
|
22,222
|
||||||
Total liabilities and stockholders’ equity
|
$
|
48,992
|
$
|
26,077
|
For the Year Ended
December 31,
|
||||||||
2022
|
2021
|
|||||||
License and collaborations revenue |
$ | 39,850 | $ | 589 | ||||
Operating expenses: | ||||||||
General and administrative
|
7,269
|
8,121
|
||||||
Research and development
|
14,355
|
15,173
|
||||||
Total operating expenses
|
21,624
|
23,294
|
||||||
Income (loss) from operations
|
18,226
|
(22,705
|
)
|
|||||
Interest expense
|
(9
|
)
|
(2
|
)
|
||||
Fair value change in warrant liabilities
|
—
|
(33,829
|
)
|
|||||
Other expense, net
|
(14
|
)
|
(157
|
)
|
||||
Income (loss) before income taxes
|
18,203
|
(56,693
|
)
|
|||||
Provision for income taxes
|
(315
|
)
|
—
|
|||||
Net income (loss)
|
17,888
|
(56,693
|
)
|
|||||
Other comprehensive income (loss), net of tax
|
—
|
—
|
||||||
Comprehensive income (loss)
|
$
|
17,888
|
$
|
(56,693
|
)
|
|||
Net income (loss) per share (Note 11):
|
||||||||
Basic
|
$
|
0.90
|
$
|
(3.82
|
)
|
|||
Diluted |
$ | 0.87 | $ | (3.82 | ) | |||
Number of shares used in per share calculations:
|
||||||||
Basic
|
19,931,080
|
14,852,745
|
||||||
Diluted |
20,597,212 | 14,852,745 |
Common Stock
|
Additional
Paid–In
|
Accumulated
|
Total
Stockholders’
|
|||||||||||||||||
Shares
|
Amount
|
Capital
|
Deficit
|
Equity
(Deficit)
|
||||||||||||||||
Balance at December 31, 2020
|
10,882,495
|
$ |
1
|
$ |
19,207
|
$ |
(32,675
|
)
|
$ |
(13,467
|
)
|
|||||||||
Reclassification of Series A warrant liability to equity |
— | — | 61,793 | — | 61,793 | |||||||||||||||
Issuance of common stock and warrants in connection with registered direct offering
|
3,076,923 | 1 | 14,999 | — | 15,000 | |||||||||||||||
Issuance of common stock in connection with the at-the-market program
|
2,778,890 | — | 13,491 | — | 13,491 | |||||||||||||||
Issuance of common stock in connection with settlement with investors
|
350,000 | — | 1,614 | — | 1,614 | |||||||||||||||
Issuance costs |
— | — | (1,517 | ) | — | (1,517 | ) | |||||||||||||
Exercise of Series B warrants |
1,629,634 | — | — | — | — | |||||||||||||||
Stock-based compensation |
54,444 | — | 1,914 | — | 1,914 | |||||||||||||||
Exercise of stock options |
73,442 | — | 87 | — | 87 | |||||||||||||||
Net and comprehensive loss |
— | — | — | (56,693 | ) | (56,693 | ) | |||||||||||||
Balance at December 31, 2021 | 18,845,828 | 2 | 111,588 | (89,368 | ) | 22,222 | ||||||||||||||
Issuance of common stock in connection with the at-the-market program
|
1,848,980 | — | 4,428 | — | 4,428 | |||||||||||||||
Issuance costs
|
— | — | (133 | ) | — | (133 | ) | |||||||||||||
Exercise of Series B warrants
|
60,832 | — | — | — | — | |||||||||||||||
Stock-based compensation
|
81,366 | — | 1,807 | — | 1,807 | |||||||||||||||
Exercise of stock options
|
24,309 | — | 27 | — | 27 | |||||||||||||||
Net and comprehensive income
|
— | — | — | 17,888 | 17,888 | |||||||||||||||
Balance at December 31, 2022
|
20,861,315 | $ | 2 | $ | 117,717 | $ | (71,480 | ) | $ | 46,239 |
For the Year Ended
December 31,
|
||||||||
2022
|
2021
|
|||||||
Operating activities
|
||||||||
Net income (loss)
|
$
|
17,888
|
$
|
(56,693
|
)
|
|||
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities:
|
||||||||
Stock-based compensation
|
1,807
|
1,914
|
||||||
Depreciation
|
4
|
4
|
||||||
Fair value change in warrant liabilities
|
—
|
33,829
|
||||||
Non-cash share settlement with investors
|
— | 1,614 | ||||||
Receipt of investments related to license agreement
|
— | (289 | ) | |||||
Unrealized loss from short-term investments
|
170 | 70 | ||||||
Change in assets and liabilities:
|
||||||||
Accounts receivable
|
(1,298 | ) | — | |||||
Contract asset
|
(3,552 | ) | — | |||||
Prepaid expenses and other assets
|
(139
|
)
|
(45
|
)
|
||||
Accounts payable
|
(515
|
)
|
381
|
|||||
Accrued expenses
|
(51
|
)
|
(155
|
)
|
||||
Net cash provided by (used in) operating activities
|
14,314
|
(19,370
|
)
|
|||||
Investing activities
|
||||||||
Transaction costs in connection with asset acquisition
|
—
|
(100
|
)
|
|||||
Net cash used in investing activities
|
—
|
(100
|
)
|
|||||
Financing activities
|
||||||||
Proceeds from issuance of common stock |
4,428 | 28,491 | ||||||
Issuance costs attributed to common stock
|
(131
|
)
|
(1,511
|
)
|
||||
Proceeds from short-term loan |
— | 646 | ||||||
Payments made on short-term loan principal |
(538 | ) | (108 | ) | ||||
Exercise of stock options and Series B warrants
|
27
|
87
|
||||||
Net cash provided by financing activities
|
3,786
|
27,605
|
||||||
Net increase in cash and cash equivalents
|
18,100
|
8,135
|
||||||
Cash and cash equivalents at beginning of period
|
24,534
|
16,399
|
||||||
Cash and cash equivalents at end of period
|
$
|
42,634
|
$
|
24,534
|
||||
Supplemental disclosure of cash flow information:
|
||||||||
Cash paid for income taxes
|
$
|
—
|
$
|
—
|
||||
Cash paid for interest
|
$
|
9
|
$
|
2
|
||||
Supplemental non-cash financing transactions:
|
||||||||
Non-cash reclassification of Series A warrant liability to equity |
$ | — | $ | 61,793 | ||||
Unpaid issuance costs
|
$
|
2
|
$
|
6
|
1. |
Company Description and Summary of Significant Accounting Policies
|
• |
Level 1 inputs: Unadjusted quoted prices for identical assets or liabilities in active markets;
|
• |
Level 2 inputs: Quoted prices for similar assets and liabilities in active markets, quoted prices in markets that are not active, or inputs which are observable, whether directly or indirectly, for substantially the full term of the
asset or liability; and
|
• |
Level 3 inputs: Unobservable inputs that reflect the Company’s own assumptions about the assumptions market participants would use in pricing the asset or liability in which there is little, if any, market activity for the asset or
liability at the measurement date.
|
As of December 31, 2022
|
||||||||||||||||
Description
|
Total
|
Level 1
|
Level 2
|
Level 3
|
||||||||||||
Assets:
|
||||||||||||||||
Short-term investments |
$
|
49
|
$
|
49
|
$
|
—
|
$
|
—
|
||||||||
Total assets at fair value
|
$
|
49
|
$
|
49
|
$
|
—
|
$
|
—
|
As of December 31, 2021
|
||||||||||||||||
Description
|
Total
|
Level 1
|
Level 2
|
Level 3
|
||||||||||||
Assets:
|
||||||||||||||||
Short-term investments
|
$
|
219
|
$
|
219
|
$
|
—
|
$
|
—
|
||||||||
Total assets at fair value
|
$
|
219
|
$
|
219
|
$
|
—
|
$
|
—
|
|
2022
|
2021
|
||||||
Short-term investments
|
||||||||
Balance as of beginning of period
|
$
|
219
|
$
|
—
|
||||
Receipt of investments related to license agreement
|
—
|
289
|
||||||
Unrealized loss
|
(170
|
)
|
(70
|
)
|
||||
Balance as of end of period
|
$
|
49
|
$
|
219
|
|
2021 |
|||
Warrant liabilities
|
||||
Balance as of beginning of period
|
$
|
27,964
|
||
Change in fair value of warrant liabilities
|
33,829
|
|||
Reclassification of warrants from liability to equity
|
(61,793
|
)
|
||
Balance as of end of period
|
$
|
—
|
2. |
Merger
|
• |
90% of all payments received by Rexahn or its affiliates during such
CVR Payment Period from or on behalf of BioSense Global LLC (“BioSense”) pursuant to that certain License and Assignment Agreement, dated as of February 25, 2019, by and between BioSense and Rexahn, as amended by Amendment No. 1,
dated August 24, 2019, and as further amended by Amendment No. 2, dated March 10, 2020, minus certain permitted deductions;
|
• |
90% of all payments received by Rexahn or its affiliates during such
CVR Payment Period from or on behalf of Zhejiang HaiChang Biotechnology Co., Ltd. (“HaiChang”) pursuant to that certain Exclusive License Agreement, dated as of February 8, 2020, by and between HaiChang and Rexahn, minus certain
permitted deductions; and
|
• |
75% of the sum of (i) all cash consideration paid by a third party to
Rexahn or its affiliates during the applicable CVR Payment Period in connection with the grant, sale or transfer of rights to Rexahn’s pre-closing intellectual property (other than a grant, sale or transfer of rights involving a sale
or disposition of the post-Merger combined company) that is entered into during the 10-year period after the Closing
(“Parent IP Deal”), plus (ii) with respect to any non-cash consideration received by Rexahn or its affiliates from a third party during the applicable CVR Payment Period in connection with any Parent IP Deal, all amounts received by
Rexahn and its affiliates for such non-cash consideration at the time such non-cash consideration is monetized by Rexahn or its affiliates, minus (iii) certain permitted deductions.
|
3. |
Pre-Merger Financing
|
4. |
Commitments and Contingencies
|
5. |
Supplemental Balance Sheet Information
|
December 31,
|
||||||||
2022
|
2021
|
|||||||
Prepaids
|
$
|
1,373
|
$
|
1,243
|
||||
Other
|
80
|
71
|
||||||
Total prepaids and other assets
|
$
|
1,453
|
$
|
1,314
|
December 31,
|
||||||||
2022
|
2021
|
|||||||
Equipment
|
$
|
20
|
$
|
20
|
||||
Furniture
|
5
|
5
|
||||||
Total property and equipment
|
25
|
25
|
||||||
Less accumulated depreciation
|
(19
|
)
|
(15
|
)
|
||||
Property and equipment, net
|
$
|
6
|
$
|
10
|
Accrued expenses consist of the following (in thousands):
|
December 31,
|
|||||||
2022
|
2021
|
|||||||
Income taxes
|
$ | 315 | $ | — | ||||
Payroll
|
782
|
488
|
||||||
Professional services
|
208
|
84
|
||||||
R&D services and supplies
|
212
|
1,081
|
||||||
Other
|
167
|
80
|
||||||
Total
|
$
|
1,684
|
$
|
1,733
|
6. |
Related Party Transactions
|
7. |
Stock-based Compensation
|
December 31,
|
||||||||
2022
|
2021
|
|||||||
General and administrative
|
$
|
1,060
|
$
|
1,116
|
||||
Research and development
|
747
|
798
|
||||||
Total stock-based compensation
|
$
|
1,807
|
$
|
1,914
|
Number of
Options
|
Weighted
Average
Exercise
Price
|
Weighted Average
Remaining
Contractual
Term (years)
|
Aggregate
Intrinsic
Value(1)
(in thousands)
|
|||||||||||||
Outstanding at December 31, 2020
|
1,784,198
|
$
|
2.17
|
8.87
|
$
|
7,744
|
||||||||||
Granted
|
420,300
|
$
|
5.72
|
—
|
—
|
|||||||||||
Exercised
|
(73,442
|
)
|
$
|
—
|
—
|
—
|
||||||||||
Forfeited/Cancelled
|
(34,220
|
)
|
$
|
—
|
—
|
—
|
||||||||||
Outstanding at December 31, 2021
|
2,096,836
|
$
|
2.97
|
8.20
|
$
|
2,795
|
||||||||||
Granted
|
893,305
|
$
|
2.64
|
|||||||||||||
Exercised
|
(24,309
|
)
|
$
|
1.09
|
||||||||||||
Forfeited/Cancelled
|
(29,788
|
)
|
$
|
6.21
|
||||||||||||
Outstanding at December 31, 2022
|
2,936,044
|
$
|
2.87
|
7.82
|
$
|
3,314
|
||||||||||
Vested and expected to vest at December 31, 2022 | 2,936,044 | $ | 2.87 | 7.82 | $ | 3,314 | ||||||||||
Vested and exercisable at December 31, 2022
|
1,723,792
|
$
|
2.56
|
7.07
|
$
|
1,680
|
(1) |
The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and
the fair value of our common stock as of December 31, 2022 and 2021 of $3.53 and $3.73 per share, respectively.
|
2022
|
2021
|
|||||||
Expected stock price volatility
|
97.4
|
%
|
98.1
|
%
|
||||
Expected life of options (years)
|
5.8
|
5.8
|
||||||
Expected dividend yield
|
0
|
%
|
0
|
%
|
||||
Risk free interest rate
|
2.3
|
%
|
0.9
|
%
|
Number of
Shares
|
||||
Non-vested at December 31, 2020
|
40,000
|
|||
Granted
|
—
|
|||
Vested
|
(40,000
|
)
|
||
Non-vested at December 31, 2021
|
—
|
8. |
Apexian Sublicense Agreement
|
9. |
Stockholder Equity
|
Contract Asset
|
||||
Balance as of December 31, 2021
|
$
|
—
|
||
Revenue recognized – license transfer |
39,519 | |||
Execution of Nyxol License Agreement and one-time non-refundable payment
|
(35,000 | ) | ||
Revenue recognized – research and development services
|
331 | |||
Reclassification to accounts receivable related to costs billed under the Nyxol License Agreement
|
(1,298 | ) | ||
Balance as of December 31, 2022
|
$ | 3,552 |
11. |
Net income (loss) per share
|
2022 | 2021 |
|||||||
Denominator (weighted average shares)
|
|
|
||||||
Basic common shares outstanding
|
|
19,931,080
|
|
14,852,745
|
||||
Dilutive stock options
|
589,165
|
—
|
||||||
Dilutive warrants
|
76,967
|
—
|
||||||
Diluted common shares outstanding
|
20,597,212
|
14,852,745
|
2022
|
2021
|
|||||||
Series A, Series B and RDO warrants
|
7,145,201
|
7,282,999 | ||||||
Stock options
|
2,346,879
|
2,096,836
|
||||||
Restricted stock awards including pending issuances of stock for services
|
—
|
6,970
|
||||||
Former Rexahn warrants
|
60,713
|
66,538 | ||||||
Former Rexahn options
|
—
|
82 |
12. |
Income Taxes
|
2022
|
2021
|
|||||||
Income tax (benefit) provision at federal statutory rate | 21.0 |
% |
(21.0 |
)% |
||||
Valuation allowance
|
(21.4 |
) |
11.9 |
|||||
State income tax, net of federal benefit
|
4.9 |
(4.8 |
) |
|||||
Warrants
|
— |
15.3 |
||||||
Stock options
|
0.4 |
(0.1 |
) |
|||||
Research and development
|
(3.1 |
) |
(1.1 |
) |
||||
Other
|
(0.1 |
) |
(0.2 |
) |
||||
Effective tax rate
|
1.7 |
% |
— |
% |
2022 |
2021 |
|||||||
Income (loss) before income taxes:
|
$ | 18,203 | $ | (56,693 | ) | |||
Current:
|
||||||||
Federal
|
$ | 279 | $ | — | ||||
State
|
36 | — | ||||||
Total current tax provision (benefit)
|
315 | — | ||||||
Deferred:
|
||||||||
Federal
|
— | — | ||||||
State
|
— | — | ||||||
Total tax provision (benefit)
|
$ | 315 | $ | — |
2022
|
2021
|
|||||||
Deferred tax assets:
|
||||||||
Federal and state operating loss carryforwards | $ | 13,087 | $ | 19,244 | ||||
Acquired intangibles
|
547 |
547
|
||||||
Deferral of research and development costs |
2,820 | — | ||||||
Organizational costs
|
7 |
7
|
||||||
Other | 62 | 18 | ||||||
Stock-based compensation
|
1,152 |
811
|
||||||
Research and development credit carryforward
|
731 |
1,035
|
||||||
Subtotal
|
18,406 |
21,662
|
||||||
Valuation allowance
|
(17,770
|
)
|
(21,662
|
)
|
||||
Total deferred tax assets, net of valuation allowance
|
636
|
—
|
||||||
Deferred tax liabilities:
|
||||||||
Deferred revenue |
(636 | ) | — | |||||
Total deferred tax liabilities
|
(636
|
)
|
—
|
|||||
Net deferred tax assets
|
$
|
—
|
$
|
—
|
13. |
Deferred Compensation Plan
|
14. |
Subsequent Events
|
OCUPHIRE PHARMA, INC.
|
||
Dated: March 30, 2023
|
By:
|
/s/ Mina Sooch
|
Mina Sooch
|
||
President, Chief Executive Officer and Director
|
By
|
/s/ Mina Sooch
|
Date: March 30, 2023
|
|
Mina Sooch
|
|||
President, Chief Executive Officer and Director
|
|||
By
|
/s/ Amy Rabourn
|
Date: March 30, 2023
|
|
Amy Rabourn
|
|||
Senior Vice President of Finance
|
|||
By
|
/s/ Sean Ainsworth
|
Date: March 30, 2023
|
|
Sean Ainsworth
|
|||
Director
|
|||
By
|
/s/ James S. Manuso
|
Date: March 30, 2023
|
|
James S. Manuso
|
|||
Director
|
|||
By
|
/s/ Cam Gallagher
|
Date: March 30, 2023
|
|
Cam Gallagher
|
|||
Director
|
|||
By
|
/s/ Jay Pepose
|
Date: March 30, 2023
|
|
Jay Pepose
|
|||
Director
|
|||
By
|
/s/ Richard J. Rodgers
|
Date: March 30, 2023
|
|
Richard J. Rodgers
|
|||
Director
|
|||
By
|
/s/ Susan K. Benton
|
Date: March 30, 2023
|
|
Susan K. Benton
|
|||
Director
|
1 Year Rexahn Pharmaceuticals Chart |
1 Month Rexahn Pharmaceuticals Chart |
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