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Share Name | Share Symbol | Market | Type |
---|---|---|---|
Rocket Pharmaceuticals Inc | NASDAQ:RCKT | NASDAQ | Common Stock |
Price Change | % Change | Share Price | Bid Price | Offer Price | High Price | Low Price | Open Price | Shares Traded | Last Trade | |
---|---|---|---|---|---|---|---|---|---|---|
-0.31 | -1.35% | 22.68 | 21.88 | 24.50 | 23.21 | 22.36 | 22.63 | 536,257 | 01:00:00 |
Agreement reached with FDA on Phase 2 pivotal study design of RP-L301 for PKD to support accelerated approval; Initiating single-arm, 10-patient pivotal study with primary endpoint of ≥1.5 point Hgb improvement at 12 months
Initiated Phase 2 pivotal trial of RP-A501 for Danon Disease following FDA alignment
Initiated Phase 1 study of RP-A601 for PKP2-ACM
FDA accepted BLA with Priority Review for KRESLADITM (marnetegragene autotemcel) for severe LAD-I; PDUFA target action date is March 31, 2024
Product filings for RP-L102 for Fanconi Anemia anticipated in the first half of 2024 in the U.S. and Europe
Cash, cash equivalents and investments of approximately $437.2M; expected operational runway through 2025
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial results for the quarter ending September 30, 2023, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.
“I’m very pleased with Rocket’s results this quarter, as we realized significant regulatory milestones across our pipeline, including reaching FDA alignment on the study design for the 10-patient Phase 2 pivotal trial of RP-L301 for the treatment of severe PKD and securing the FDA’s acceptance of Rocket’s BLA for RP-L201, now named KRESLADITM, for the treatment of severe LAD-I,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “At the same time, we initiated the Phase 2 pivotal trial of RP-A501 for Danon Disease following FDA alignment and the Phase 1 study of RP-A601 for PKP2-ACM, which move us substantially closer to delivering potential one-time, curative therapies to patients facing these two devastating cardiac diseases.”
Dr. Shah continued, “As we bring 2023 to a close, we have made remarkable progress across the breadth of our AAV cardiovascular and LV hematology portfolios. Four programs have now reached pivotal studies, one of which is under review for approval, thanks to strong collaboration with health authorities and scientific partners, as we collectively seek to fulfill the high unmet needs of patients with rare and life-threatening diseases.”
Key Pipeline and Operational Updates
AAV Cardiovascular Portfolio
Danon Disease
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
LV Hematology Portfolio
Leukocyte Adhesion Deficiency-I (LAD-I)
Fanconi Anemia (FA)
Pyruvate Kinase Deficiency (PKD)
Operational Updates
Upcoming Investor Conferences
Third Quarter Financial Results
Financial Guidance
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral (LV) vector-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also is developing AAV-based gene therapy programs in PKP2-arrhythmogenic cardiomyopathy (ACM) and BAG3-associated dilated cardiomyopathy (DCM). For more information about Rocket, please visit www.rocketpharma.com.
Rocket Cautionary Statement Regarding Forward-Looking Statements
Various statements in this release concerning Rocket’s future expectations, plans and prospects, including without limitation, Rocket’s expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, Rocket’s plans for the advancement of its Danon Disease program, including its planned pivotal trial, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2022, filed February 28, 2023 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
Three Months Ended September 30, Nine Months Ended September 30,
2023
2022
2023
2022
Operating expenses: Research and development
$
46,844
$
43,383
$
144,598
$
115,533
General and administrative
18,585
15,105
51,782
39,728
Total operating expenses
65,429
58,488
196,380
155,261
Loss from operations
(65,429
)
(58,488
)
(196,380
)
(155,261
)
Interest expense
(469
)
(465
)
(1,405
)
(1,395
)
Interest and other income, net
1,720
1,353
4,474
2,644
Accretion of discount and amortization of premium on investments, net
2,279
(156
)
7,376
(1,128
)
Net loss$
(61,899
)
$
(57,756
)
$
(185,935
)
$
(155,140
)
Net loss per share - basic and diluted$
(0.75
)
$
(0.87
)
$
(2.30
)
$
(2.37
)
Weighted-average common shares outstanding - basic and diluted
82,636,120
66,215,535
80,865,658
65,406,844
September 30, 2023 December 31, 2022 Cash, cash equivalents, and investments
$
437,171
$
399,670
Total assets
598,762
551,807
Total liabilities
57,676
62,121
Total stockholders' equity
541,086
489,686
View source version on businesswire.com: https://www.businesswire.com/news/home/20231106135977/en/
Media Kevin Giordano media@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
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