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Share Name | Share Symbol | Market | Type |
---|---|---|---|
Regulus Therapeutics Inc | NASDAQ:RGLS | NASDAQ | Common Stock |
Price Change | % Change | Share Price | Bid Price | Offer Price | High Price | Low Price | Open Price | Shares Traded | Last Trade | |
---|---|---|---|---|---|---|---|---|---|---|
-0.07 | -2.60% | 2.62 | 0.77 | 3.06 | 2.83 | 2.64 | 2.83 | 406,153 | 05:00:06 |
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FORM 10-Q
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x
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QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
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¨
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TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
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Regulus Therapeutics Inc.
(Exact name of registrant as specified in its charter)
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Delaware
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26-4738379
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(State or Other Jurisdiction of
Incorporation or Organization)
|
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(I.R.S. Employer
Identification No.)
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10614 Science Center Drive
San Diego, CA
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92121
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(Address of Principal Executive Offices)
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(Zip Code)
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Large accelerated filer
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¨
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Accelerated filer
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x
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Non-accelerated filer
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¨
(Do not check if a smaller reporting company)
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Smaller reporting company
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¨
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PART I. FINANCIAL INFORMATION
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PART II. OTHER INFORMATION
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ITEM 1.
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FINANCIAL STATEMENTS
|
|
June 30,
2016 |
|
December 31,
2015 |
||||
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(Unaudited)
|
|
|
||||
Assets
|
|
|
|
||||
Current assets:
|
|
|
|
||||
Cash and cash equivalents
|
$
|
27,040
|
|
|
$
|
15,960
|
|
Short-term investments
|
80,502
|
|
|
98,103
|
|
||
Restricted cash
|
462
|
|
|
1,256
|
|
||
Prepaid expenses
|
7,565
|
|
|
8,159
|
|
||
Contract and other receivables
|
143
|
|
|
10,021
|
|
||
Other current assets
|
522
|
|
|
759
|
|
||
Total current assets
|
116,234
|
|
|
134,258
|
|
||
Property and equipment, net
|
12,322
|
|
|
5,400
|
|
||
Intangibles, net
|
1,062
|
|
|
1,081
|
|
||
Other assets
|
310
|
|
|
344
|
|
||
Total assets
|
$
|
129,928
|
|
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$
|
141,083
|
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Liabilities and stockholders’ equity
|
|
|
|
||||
Current liabilities:
|
|
|
|
||||
Accounts payable
|
$
|
3,504
|
|
|
$
|
2,717
|
|
Accrued liabilities
|
5,472
|
|
|
6,329
|
|
||
Accrued compensation
|
1,863
|
|
|
2,392
|
|
||
Current portion of deferred revenue
|
258
|
|
|
1,194
|
|
||
Total current liabilities
|
11,097
|
|
|
12,632
|
|
||
Term loan, less debt issuance costs
|
19,786
|
|
|
—
|
|
||
Deferred revenue, less current portion
|
2,029
|
|
|
2,065
|
|
||
Other long-term liabilities
|
8,570
|
|
|
2,308
|
|
||
Total liabilities
|
41,482
|
|
|
17,005
|
|
||
Commitments and Contingencies
|
|
|
|
||||
Stockholders’ equity:
|
|
|
|
||||
Common stock, $0.001 par value; 200,000,000 shares authorized, 52,787,756
and 52,669,266 shares issued and outstanding at June 30, 2016 (unaudited) and December 31, 2015, respectively
|
53
|
|
|
53
|
|
||
Additional paid-in capital
|
322,287
|
|
|
315,673
|
|
||
Accumulated other comprehensive loss
|
(83
|
)
|
|
(133
|
)
|
||
Accumulated deficit
|
(233,811
|
)
|
|
(191,515
|
)
|
||
Total stockholders’ equity
|
88,446
|
|
|
124,078
|
|
||
Total liabilities and stockholders’ equity
|
$
|
129,928
|
|
|
$
|
141,083
|
|
|
Three months ended
June 30, |
|
Six months ended
June 30, |
||||||||||||
|
2016
|
|
2015
|
|
2016
|
|
2015
|
||||||||
|
(Unaudited)
|
||||||||||||||
Revenues:
|
|
|
|
|
|
|
|
||||||||
Revenue under strategic alliances and collaborations
|
$
|
483
|
|
|
$
|
3,834
|
|
|
$
|
972
|
|
|
$
|
8,034
|
|
Total revenues
|
483
|
|
|
3,834
|
|
|
972
|
|
|
8,034
|
|
||||
Operating expenses:
|
|
|
|
|
|
|
|
||||||||
Research and development
|
18,007
|
|
|
19,201
|
|
|
34,772
|
|
|
32,628
|
|
||||
General and administrative
|
3,664
|
|
|
5,814
|
|
|
8,767
|
|
|
9,458
|
|
||||
Total operating expenses
|
21,671
|
|
|
25,015
|
|
|
43,539
|
|
|
42,086
|
|
||||
Loss from operations
|
(21,188
|
)
|
|
(21,181
|
)
|
|
(42,567
|
)
|
|
(34,052
|
)
|
||||
Other income (expense):
|
|
|
|
|
|
|
|
||||||||
Interest and other income
|
180
|
|
|
151
|
|
|
372
|
|
|
351
|
|
||||
Interest and other expense
|
(90
|
)
|
|
(7
|
)
|
|
(114
|
)
|
|
(16
|
)
|
||||
Loss from valuation of convertible note payable
|
—
|
|
|
—
|
|
|
—
|
|
|
(1,811
|
)
|
||||
Loss before income taxes
|
(21,098
|
)
|
|
(21,037
|
)
|
|
(42,309
|
)
|
|
(35,528
|
)
|
||||
Income tax benefit
|
8
|
|
|
2
|
|
|
13
|
|
|
6
|
|
||||
Net loss
|
$
|
(21,090
|
)
|
|
$
|
(21,035
|
)
|
|
$
|
(42,296
|
)
|
|
$
|
(35,522
|
)
|
Other comprehensive loss:
|
|
|
|
|
|
|
|
||||||||
Unrealized gain (loss) on short-term investments, net
|
9
|
|
|
(2
|
)
|
|
50
|
|
|
55
|
|
||||
Comprehensive loss
|
$
|
(21,081
|
)
|
|
$
|
(21,037
|
)
|
|
$
|
(42,246
|
)
|
|
$
|
(35,467
|
)
|
Net loss per share, basic and diluted
|
$
|
(0.40
|
)
|
|
$
|
(0.41
|
)
|
|
$
|
(0.80
|
)
|
|
$
|
(0.70
|
)
|
Weighted average shares used to compute basic and diluted net loss per share
|
52,782,643
|
|
|
51,073,488
|
|
|
52,746,657
|
|
|
50,575,096
|
|
|
Six months ended
June 30, |
||||||
|
2016
|
|
2015
|
||||
|
(Unaudited)
|
||||||
Operating activities
|
|
|
|
||||
Net loss
|
$
|
(42,296
|
)
|
|
$
|
(35,522
|
)
|
Adjustments to reconcile net loss to net cash used in operating activities
|
|
|
|
||||
Depreciation and amortization expense
|
928
|
|
|
798
|
|
||
Loss from valuation of convertible note payable
|
—
|
|
|
1,811
|
|
||
Stock-based compensation
|
5,986
|
|
|
8,972
|
|
||
Amortization of premium on investments, net
|
359
|
|
|
801
|
|
||
Other
|
45
|
|
|
56
|
|
||
Change in operating assets and liabilities:
|
|
|
|
||||
Contracts and other receivables
|
9,878
|
|
|
(50
|
)
|
||
Prepaid expenses
|
594
|
|
|
826
|
|
||
Other assets
|
270
|
|
|
20
|
|
||
Accounts payable
|
787
|
|
|
130
|
|
||
Accrued liabilities
|
(1,063
|
)
|
|
2,459
|
|
||
Accrued compensation
|
(529
|
)
|
|
167
|
|
||
Deferred revenue
|
(972
|
)
|
|
(1,323
|
)
|
||
Deferred rent and other liabilities
|
(262
|
)
|
|
(160
|
)
|
||
Net cash used in operating activities
|
(26,275
|
)
|
|
(21,015
|
)
|
||
Investing activities
|
|
|
|
||||
Purchases of short-term investments
|
(30,231
|
)
|
|
(46,281
|
)
|
||
Sales and maturities of short-term investments
|
47,522
|
|
|
49,645
|
|
||
Purchases of property and equipment
|
(266
|
)
|
|
(410
|
)
|
||
Acquisition of intangibles
|
(34
|
)
|
|
(19
|
)
|
||
Net cash provided by investing activities
|
16,991
|
|
|
2,935
|
|
||
Financing activities
|
|
|
|
||||
Proceeds from borrowing under term loan, net
|
19,819
|
|
|
—
|
|
||
Proceeds from issuance of common stock, net
|
363
|
|
|
257
|
|
||
Proceeds from exercise of common stock options
|
265
|
|
|
1,655
|
|
||
Principal payments on other long-term obligations
|
(83
|
)
|
|
(75
|
)
|
||
Net cash provided by financing activities
|
20,364
|
|
|
1,837
|
|
||
Net increase (decrease) in cash and cash equivalents
|
11,080
|
|
|
(16,243
|
)
|
||
Cash and cash equivalents at beginning of period
|
15,960
|
|
|
37,327
|
|
||
Cash and cash equivalents at end of period
|
$
|
27,040
|
|
|
$
|
21,084
|
|
Supplemental disclosure of cash flow information
|
|
|
|
||||
Net changes in restricted cash
|
$
|
(794
|
)
|
|
$
|
—
|
|
Interest paid
|
$
|
(54
|
)
|
|
$
|
(15
|
)
|
Income taxes paid
|
$
|
(1
|
)
|
|
$
|
(1
|
)
|
Supplemental disclosure of non-cash investing and financing activities
|
|
|
|
||||
Allowance for tenant improvements
|
$
|
6,545
|
|
|
$
|
—
|
|
Amounts accrued for property and equipment
|
$
|
221
|
|
|
$
|
90
|
|
Amounts accrued for patent expenditures
|
$
|
7
|
|
|
$
|
—
|
|
Unpaid debt issuance costs
|
$
|
38
|
|
|
$
|
—
|
|
|
Maturity
(in years)
|
|
Amortized
cost
|
|
Unrealized
|
|
Estimated
fair value
|
||||||||||
Gains
|
|
Losses
|
|
||||||||||||||
As of June 30, 2016
|
|
|
|
|
|
|
|
|
|
||||||||
Corporate debt securities
|
2 or less
|
|
$
|
65,725
|
|
|
$
|
8
|
|
|
$
|
(26
|
)
|
|
$
|
65,707
|
|
Certificates of deposit
|
2 or less
|
|
9,800
|
|
|
—
|
|
|
—
|
|
|
9,800
|
|
||||
Commercial paper
|
1 or less
|
|
4,983
|
|
|
12
|
|
|
—
|
|
|
4,995
|
|
||||
Total
|
|
|
$
|
80,508
|
|
|
$
|
20
|
|
|
$
|
(26
|
)
|
|
$
|
80,502
|
|
|
Maturity
(in years)
|
|
Amortized
cost
|
|
Unrealized
|
|
Estimated
fair value
|
||||||||||
Gains
|
|
Losses
|
|
||||||||||||||
As of December 31, 2015
|
|
|
|
|
|
|
|
|
|
||||||||
Corporate debt securities
|
2 or less
|
|
$
|
81,054
|
|
|
$
|
16
|
|
|
$
|
(103
|
)
|
|
$
|
80,967
|
|
Certificates of deposit
|
2 or less
|
|
13,640
|
|
|
—
|
|
|
—
|
|
|
13,640
|
|
||||
Commercial paper
|
1 or less
|
|
3,490
|
|
|
6
|
|
|
—
|
|
|
3,496
|
|
||||
Total
|
|
|
$
|
98,184
|
|
|
$
|
22
|
|
|
$
|
(103
|
)
|
|
$
|
98,103
|
|
•
|
Level 1 includes financial instruments for which quoted market prices for identical instruments are available in active markets.
|
•
|
Level 2 includes financial instruments for which there are inputs other than quoted prices included within Level 1 that are observable for the instrument such as quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets with insufficient volume or infrequent transactions (less active markets) or model-driven valuations in which significant inputs are observable or can be derived principally from, or corroborated by, observable market data.
|
•
|
Level 3 includes financial instruments for which fair value is derived from valuation techniques in which one or more significant inputs are unobservable, including management’s own assumptions.
|
|
Fair value as of June 30, 2016
|
||||||||||||||
|
Total
|
|
Level 1
|
|
Level 2
|
|
Level 3
|
||||||||
Assets:
|
|
|
|
|
|
|
|
||||||||
Cash equivalents
|
$
|
17,443
|
|
|
$
|
17,443
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Corporate debt securities
|
65,707
|
|
|
—
|
|
|
65,707
|
|
|
—
|
|
||||
Certificates of deposit
|
9,800
|
|
|
—
|
|
|
9,800
|
|
|
—
|
|
||||
Commercial paper
|
4,995
|
|
|
—
|
|
|
4,995
|
|
|
—
|
|
||||
|
$
|
97,945
|
|
|
$
|
17,443
|
|
|
$
|
80,502
|
|
|
$
|
—
|
|
|
Fair value as of December 31, 2015
|
||||||||||||||
|
Total
|
|
Level 1
|
|
Level 2
|
|
Level 3
|
||||||||
Assets:
|
|
|
|
|
|
|
|
||||||||
Cash equivalents
|
$
|
15,152
|
|
|
$
|
15,152
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Corporate debt securities
|
80,967
|
|
|
—
|
|
|
80,967
|
|
|
—
|
|
||||
Certificates of deposit
|
13,640
|
|
|
—
|
|
|
13,640
|
|
|
—
|
|
||||
Commercial paper
|
3,496
|
|
|
—
|
|
|
3,496
|
|
|
—
|
|
||||
|
$
|
113,255
|
|
|
$
|
15,152
|
|
|
$
|
98,103
|
|
|
$
|
—
|
|
2016
|
$
|
—
|
|
2017
|
—
|
|
|
2018
|
5,000
|
|
|
2019
|
10,000
|
|
|
2020
|
5,000
|
|
|
|
$
|
20,000
|
|
Common stock options outstanding
|
6,937,798
|
|
Common stock available for future grant under 2012 Equity Incentive Plan
|
2,164,263
|
|
Common stock available for future grant under 2015 Inducement Plan
|
581,806
|
|
Employee Stock Purchase Plan
|
1,697,431
|
|
Total common shares reserved for future issuance
|
11,381,298
|
|
|
Number of
options
|
|
Weighted
average
exercise
price
|
|||
Options outstanding at December 31, 2015
|
5,126
|
|
|
$
|
8.91
|
|
Granted
|
2,375
|
|
|
$
|
6.62
|
|
Exercised
|
(56
|
)
|
|
$
|
4.76
|
|
Canceled/forfeited/expired
|
(507
|
)
|
|
$
|
10.47
|
|
Options outstanding at June 30, 2016
|
6,938
|
|
|
$
|
8.04
|
|
|
Three months ended
June 30, |
|
Six months ended
June 30, |
||||||||
|
2016
|
|
2015
|
|
2016
|
|
2015
|
||||
Stock options
|
|
|
|
|
|
|
|
||||
Risk-free interest rate
|
1.5
|
%
|
|
1.8
|
%
|
|
1.4
|
%
|
|
1.8
|
%
|
Volatility
|
79.1
|
%
|
|
79.1
|
%
|
|
79.7
|
%
|
|
78.8
|
%
|
Dividend yield
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
Expected term (years)
|
5.8
|
|
|
6.1
|
|
|
5.9
|
|
|
6.1
|
|
Performance stock options
|
|
|
|
||||||||
Risk-free interest rate
|
0.4
|
%
|
|
—
|
|
|
1.4
|
%
|
|
1.8
|
%
|
Volatility
|
78.5
|
%
|
|
—
|
|
|
79.3
|
%
|
|
76.4
|
%
|
Dividend yield
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
Expected term (years)
|
5.5
|
|
|
0
|
|
|
6.0
|
|
|
6.0
|
|
Employee stock purchase plan shares
|
|
|
|
||||||||
Risk-free interest rate
|
0.5
|
%
|
|
0.1
|
%
|
|
0.4
|
%
|
|
0.1
|
%
|
Volatility
|
82.6
|
%
|
|
76.9
|
%
|
|
81.0
|
%
|
|
74.9
|
%
|
Dividend yield
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
Expected term (years)
|
0.5
|
|
|
0.5
|
|
|
0.5
|
|
|
0.5
|
|
|
Three months ended
June 30, |
|
Six months ended
June 30, |
||||||||||||
|
2016
|
|
2015
|
|
2016
|
|
2015
|
||||||||
Research and development
|
$
|
1,299
|
|
|
$
|
3,496
|
|
|
$
|
2,731
|
|
|
$
|
5,452
|
|
General and administrative
|
939
|
|
|
2,373
|
|
|
3,255
|
|
|
3,520
|
|
||||
Total
|
$
|
2,238
|
|
|
$
|
5,869
|
|
|
$
|
5,986
|
|
|
$
|
8,972
|
|
|
Three months ended
June 30, |
|
Six months ended
June 30, |
||||||||||||
|
2016
|
|
2015
|
|
2016
|
|
2015
|
||||||||
AstraZeneca
|
$
|
465
|
|
|
$
|
3,066
|
|
|
$
|
936
|
|
|
$
|
6,648
|
|
Sanofi
|
18
|
|
|
18
|
|
|
36
|
|
|
36
|
|
||||
Biogen
|
—
|
|
|
750
|
|
|
—
|
|
|
1,350
|
|
||||
Total
|
$
|
483
|
|
|
$
|
3,834
|
|
|
$
|
972
|
|
|
$
|
8,034
|
|
•
|
the initiation, cost, timing, progress and results of, and our expected ability to undertake certain activities and accomplish certain goals with respect to our research and development activities, preclinical studies and clinical trials;
|
•
|
our ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations, and/or warnings in the label of an approved product candidate;
|
•
|
our ability to obtain funding for our operations;
|
•
|
our plans to research, develop and commercialize our product candidates;
|
•
|
our strategic alliance partners’ election to pursue development and commercialization of any programs or product candidates that are subject to our collaboration and license agreements with such partners;
|
•
|
our ability to attract collaborators with relevant development, regulatory and commercialization expertise;
|
•
|
future activities to be undertaken by our strategic alliance partners, collaborators and other third parties;
|
•
|
our ability to obtain and maintain intellectual property protection for our product candidates;
|
•
|
the size and growth potential of the markets for our product candidates, and our ability to serve those markets;
|
•
|
our ability to successfully commercialize, and our expectations regarding future therapeutic and commercial potential with respect to our product candidates;
|
•
|
the rate and degree of market acceptance of our product candidates;
|
•
|
our ability to develop sales and marketing capabilities, whether alone or with potential future collaborators;
|
•
|
regulatory developments in the United States and foreign countries;
|
•
|
the performance of our third-party suppliers and manufacturers;
|
•
|
the success of competing therapies that are or become available;
|
•
|
the loss of key scientific or management personnel;
|
•
|
our ability to successfully secure and deploy capital;
|
•
|
our ability to satisfy our debt obligations;
|
•
|
our expectations regarding the time during which we will be an emerging growth company under the Jumpstart Our Business Startups Act of 2012, or the JOBS Act;
|
•
|
the accuracy of our estimates regarding future expenses, future revenues, capital requirements and need for additional financing; and
|
•
|
the risks and other forward-looking statements described under the caption “Risk Factors” under Part II, Item 1A of this quarterly report on Form 10-Q.
|
•
|
micro
RNAs, until recently, have not been a focus of pharmaceutical research;
|
•
|
micro
RNAs play a critical role in regulating biological pathways by controlling the translation of many target genes;
|
•
|
micro
RNA therapeutics target entire disease pathways which may result in more effective treatment of complex multi-factorial diseases; and
|
•
|
micro
RNA therapeutics may be synergistic with other therapies because of their different mechanism of action.
|
•
|
In August 2015, we initiated a Phase II study investigating RG-101 designed to evaluate a shortened, four-week treatment regimen containing a subcutaneous administration of 2 mg/kg of RG-101 at Day 1 and Day 29, in combination with oral direct-acting antiviral agents Harvoni®, Olysio®, and Daklinza® for 28 days. In June 2016, we announced positive top-line results from the primary endpoint analysis of this clinical study:
|
Time Since Treatment Completion
|
RG-101 + Harvoni®
|
RG-101 + Olysio®
|
RG-101 + Daklinza™
|
Week 12
|
27/27 (100%)
|
26/27 (96.3%)
|
22/24 (91.7%)*
|
Week 16
|
21/21 (100%)
|
19/20 (95.0%)
|
20/22 (90.9%)
|
Week 20
|
14/14 (100%)
|
13/15 (86.7%)
|
13/13 (100%)
|
Week 24
|
10/10 (100%)
|
8/10 (80.0%)
|
8/9 (88.9%)
|
•
|
In November 2015, we entered into a clinical trial collaboration and formulation agreement with GSK LLC. In Q1 2016, we initiated a Phase II study evaluating the potential to achieve sustained viral responses post treatment with a single subcutaneous administration of RG-101 in combination with daily oral administrations of GSK2878175, a non-nucleoside NS5B polymerase inhibitor, for up to 12 weeks in treatment-naïve patients chronically infected with HCV genotypes 1 and 3. Concurrently, GSK had initiated the development of a long-acting parenteral formulation for injection, or LAP, of GSK2878175 which could improve patient compliance through reduced dosing intervals and potentially extend opportunities for HCV therapeutic intervention. We expect to report interim safety and efficacy data from the GSK Phase II study before the end of 2016.
|
•
|
In May 2016, we expanded the clinical trial collaboration agreement with GSK to conduct a multi-centered, randomized, dose-ranging Phase II study evaluating the combination of RG-101 and GSK’s LAP formulation of GSK2878175 as a potential single-visit cure in patients chronically infected with HCV. This study will be conducted outside the United States and is planned to begin in the fourth quarter of 2016. Based on predicted enrollment rates, we expect interim results from this expanded collaboration to be available in the second half of 2017, enabling a potential initiation of a pivotal study in late 2017. As with the initial collaboration, both parties will contribute to the costs associated with the study. Neither we nor GSK has any further obligations or commitments to each other beyond this expanded clinical collaboration agreement.
|
•
|
In January 2016, we initiated a multi-center, open label, non-randomized Phase I study to compare the safety, tolerability, pharmacokinetics, and pharmacodynamics of 2 mg/kg of RG-101 in subjects with severe renal insufficiency or end-stage renal disease (ESRD) to healthy control subjects, and further explore RG-101 in hepatitis C infected subjects with severe renal insufficiency or ESRD. The Phase I study has three treatment arms (n=24): (i) healthy volunteers (n=8); (ii) patients with severe renal impairment or ESRD (n=8); and (iii) HCV patients with severe renal impairment or ESRD (n=8). Enrollment was completed in the second quarter of 2016, and we anticipate reporting additional safety and efficacy data from the HCV/severe renal impairment or ESRD arm in the second half of 2016.
|
•
|
In June 2016, we received verbal notice from the U.S. Food and Drug Administration, or FDA, that our IND for RG-101 for the treatment of chronic HCV infection has been placed on clinical hold. The FDA initiated the clinical
|
•
|
employee-related expenses, including salaries, benefits, travel and stock-based compensation expense;
|
•
|
external research and development expenses incurred under arrangements with third parties, such as contract research organizations, or CROs, contract manufacturing organizations, or CMOs, other clinical trial related vendors, consultants and our scientific advisors;
|
•
|
license fees; and
|
•
|
facilities, depreciation and other allocated expenses, which include direct and allocated expenses for rent and maintenance of facilities, depreciation of leasehold improvements and equipment, and laboratory and other supplies.
|
|
Three months ended
June 30, |
|
Six months ended
June 30, |
||||||||||||
|
2016
|
|
2015
|
|
2016
|
|
2015
|
||||||||
Revenue under strategic alliances and collaborations
|
$
|
483
|
|
|
$
|
3,834
|
|
|
$
|
972
|
|
|
$
|
8,034
|
|
Research and development expenses
|
18,007
|
|
|
19,201
|
|
|
34,772
|
|
|
32,628
|
|
||||
General and administrative expenses
|
3,664
|
|
|
5,814
|
|
|
8,767
|
|
|
9,458
|
|
||||
Loss from valuation of convertible note payable
|
—
|
|
|
—
|
|
|
—
|
|
|
(1,811
|
)
|
|
Three months ended
June 30, |
|
Six months ended
June 30, |
||||||||||||
|
2016
|
|
2015
|
|
2016
|
|
2015
|
||||||||
AstraZeneca
|
$
|
465
|
|
|
$
|
3,066
|
|
|
$
|
936
|
|
|
$
|
6,648
|
|
Sanofi
|
18
|
|
|
18
|
|
|
36
|
|
|
36
|
|
||||
Biogen
|
—
|
|
|
750
|
|
|
—
|
|
|
1,350
|
|
||||
Total revenues under strategic alliances and collaborations
|
$
|
483
|
|
|
$
|
3,834
|
|
|
$
|
972
|
|
|
$
|
8,034
|
|
|
Six months ended
June 30, |
||||||
|
2016
|
|
2015
|
||||
|
(unaudited)
|
||||||
Net cash (used in) provided by:
|
|
|
|
||||
Operating activities
|
$
|
(26,275
|
)
|
|
$
|
(21,015
|
)
|
Investing activities
|
16,991
|
|
|
2,935
|
|
||
Financing activities
|
20,364
|
|
|
1,837
|
|
||
Total
|
$
|
11,080
|
|
|
$
|
(16,243
|
)
|
•
|
identifying and validating new
micro
RNAs as therapeutic targets;
|
•
|
completing our research and preclinical development of product candidates;
|
•
|
initiating and completing clinical trials for product candidates;
|
•
|
seeking and obtaining marketing approvals for product candidates that successfully complete clinical trials;
|
•
|
establishing and maintaining supply and manufacturing relationships with third parties;
|
•
|
launching and commercializing product candidates for which we obtain marketing approval, with an alliance partner or, if launched independently, successfully establishing a sales force, marketing and distribution infrastructure;
|
•
|
maintaining, protecting and expanding our intellectual property portfolio; and
|
•
|
attracting, hiring and retaining qualified personnel.
|
•
|
significantly delay, scale back or discontinue the development or commercialization of any future product candidates;
|
•
|
seek strategic alliances for research and development programs at an earlier stage than otherwise would be desirable or on terms that are less favorable than might otherwise be available; or
|
•
|
relinquish or license on unfavorable terms, our rights to technologies or any future product candidates that we otherwise would seek to develop or commercialize ourselves.
|
•
|
dispose of assets;
|
•
|
complete mergers or acquisitions;
|
•
|
incur indebtedness;
|
•
|
encumber assets;
|
•
|
pay dividends or make other distributions to holders of our capital stock;
|
•
|
make specified investments; and
|
•
|
engage in transactions with our affiliates.
|
•
|
our research methodology or that of our strategic alliance partners may be unsuccessful in identifying potential product candidates;
|
•
|
potential product candidates may be shown to have harmful side effects or may have other characteristics that may make the products unmarketable or unlikely to receive marketing approval; or
|
•
|
our strategic alliance partners may change their development profiles for potential product candidates or abandon a therapeutic area.
|
•
|
successful completion of preclinical studies and clinical trials;
|
•
|
receipt of marketing approvals from applicable regulatory authorities;
|
•
|
obtaining and maintaining patent and trade secret protection for future product candidates;
|
•
|
establishing and maintaining manufacturing relationships with third parties or establishing our own manufacturing capability; and
|
•
|
successfully commercializing our products, if and when approved, whether alone or in collaboration with others.
|
•
|
delays in reaching an agreement with the FDA or other regulatory authorities on final trial design;
|
•
|
imposition of a clinical hold following an inspection of our clinical trial operations or trial sites by the FDA or other regulatory authorities;
|
•
|
delays in reaching agreement on acceptable terms with prospective CROs and clinical trial sites;
|
•
|
our inability to adhere to clinical trial requirements directly or with third parties such as CROs;
|
•
|
delays in obtaining required institutional review board approval at each clinical trial site;
|
•
|
delays in recruiting suitable patients to participate in a trial;
|
•
|
delays in the testing, validation, manufacturing and delivery of the product candidates to the clinical sites;
|
•
|
delays in having patients complete participation in a trial or return for post-treatment follow-up;
|
•
|
delays caused by patients dropping out of a trial due to product side effects or disease progression;
|
•
|
clinical sites dropping out of a trial to the detriment of enrollment;
|
•
|
time required to add new clinical sites; or
|
•
|
delays by our contract manufacturers to produce and deliver sufficient supply of clinical trial materials.
|
•
|
be delayed in obtaining marketing approval for our future product candidates;
|
•
|
not obtain marketing approval at all;
|
•
|
obtain approval for indications or patient populations that are not as broad as originally intended or desired;
|
•
|
obtain approval with labeling that includes significant use or distribution restrictions or safety warnings;
|
•
|
be subject to additional post-marketing testing requirements; or
|
•
|
have the product removed from the market after obtaining marketing approval.
|
•
|
regulatory authorities may withdraw their approval of the product or impose restrictions on its distribution in the form of a modified risk evaluation and mitigation strategy;
|
•
|
regulatory authorities may require the addition of labeling statements, such as warnings or contraindications;
|
•
|
we may be required to change the way the product is administered or conduct additional clinical trials;
|
•
|
we could be sued and held liable for harm caused to patients; or
|
•
|
our reputation may suffer.
|
•
|
issue a warning letter asserting that we are in violation of the law;
|
•
|
seek an injunction or impose civil or criminal penalties or monetary fines;
|
•
|
suspend or withdraw regulatory approval;
|
•
|
suspend any ongoing clinical trials;
|
•
|
refuse to approve a pending NDA or supplements to an NDA submitted by us;
|
•
|
seize product; or
|
•
|
refuse to allow us to enter into supply contracts, including government contracts.
|
•
|
an alliance partner may shift its priorities and resources away from our programs due to a change in business strategies, or a merger, acquisition, sale or downsizing of its company or business unit;
|
•
|
an alliance partner may cease development in therapeutic areas which are the subject of our strategic alliances;
|
•
|
an alliance partner may change the success criteria for a particular program or potential product candidate thereby delaying or ceasing development of such program or candidate;
|
•
|
a significant delay in initiation of certain development activities by an alliance partner will also delay payment of milestones tied to such activities, thereby impacting our ability to fund our own activities;
|
•
|
an alliance partner could develop a product that competes, either directly or indirectly, with an alliance product;
|
•
|
an alliance partner with commercialization obligations may not commit sufficient financial or human resources to the marketing, distribution or sale of a product;
|
•
|
an alliance partner with manufacturing responsibilities may encounter regulatory, resource or quality issues and be unable to meet demand requirements;
|
•
|
an alliance partner may exercise its rights under the agreement to terminate a strategic alliance;
|
•
|
a dispute may arise between us and an alliance partner concerning the research, development or commercialization of a program or product candidate resulting in a delay in milestones, royalty payments or termination of a program and possibly resulting in costly litigation or arbitration which may divert management attention and resources; and
|
•
|
an alliance partner may use our proprietary information or intellectual property in such a way as to invite litigation from a third party or fail to maintain or prosecute intellectual property rights such that our rights in such property are jeopardized.
|
•
|
in the case of Sanofi, under certain circumstances, we may owe Sanofi royalties with respect to product candidates covered by our agreement with Sanofi that we elect to continue to commercialize, depending upon the stage of development at which such product commercialization rights reverted back to us, or additional payments if we license such product candidates to third parties;
|
•
|
the development of our product candidates subject to the AstraZeneca agreement or the Sanofi agreement, as applicable, may be terminated or significantly delayed;
|
•
|
our cash expenditures could increase significantly if it is necessary for us to hire additional employees and allocate scarce resources to the development and commercialization of product candidates that were previously funded, or expected to be funded, by AstraZeneca or Sanofi, as applicable;
|
•
|
we would bear all of the risks and costs related to the further development and commercialization of product candidates that were previously the subject of the AstraZeneca agreement or the Sanofi agreement, as applicable, including the reimbursement of third parties; and
|
•
|
in order to fund further development and commercialization, we may need to seek out and establish alternative strategic alliances with third-party partners; this may not be possible, or we may not be able to do so on terms which are acceptable to us, in which case it may be necessary for us to limit the size or scope of one or more of our programs or increase our expenditures and seek additional funding by other means.
|
•
|
the inability to meet any product specifications and quality requirements consistently;
|
•
|
a delay or inability to procure or expand sufficient manufacturing capacity;
|
•
|
manufacturing and product quality issues related to scale-up of manufacturing;
|
•
|
costs and validation of new equipment and facilities required for scale-up;
|
•
|
a failure to comply with cGMP and similar foreign standards;
|
•
|
the inability to negotiate manufacturing or supply agreements with third parties under commercially reasonable terms;
|
•
|
termination or nonrenewal of manufacturing agreements with third parties in a manner or at a time that is costly or damaging to us;
|
•
|
the reliance on a limited number of sources, and in some cases, single sources for raw materials, such that if we are unable to secure a sufficient supply of these product components, we will be unable to manufacture and sell future product candidates in a timely fashion, in sufficient quantities or under acceptable terms;
|
•
|
the lack of qualified backup suppliers for any raw materials that are currently purchased from a single source supplier;
|
•
|
operations of our third-party manufacturers or suppliers could be disrupted by conditions unrelated to our business or operations, including the bankruptcy of the manufacturer or supplier;
|
•
|
carrier disruptions or increased costs that are beyond our control; and
|
•
|
the failure to deliver products under specified storage conditions and in a timely manner.
|
•
|
discover and develop therapeutics that are superior to other products in the market;
|
•
|
attract qualified scientific, product development and commercial personnel;
|
•
|
obtain patent and/or other proprietary protection for our
micro
RNA product platform and future product candidates;
|
•
|
obtain required regulatory approvals; and
|
•
|
successfully collaborate with pharmaceutical companies in the discovery, development and commercialization of new therapeutics.
|
•
|
demonstration of clinical safety and efficacy compared to other products;
|
•
|
the relative convenience, ease of administration and acceptance by physicians, patients and healthcare payors;
|
•
|
the prevalence and severity of any AEs;
|
•
|
limitations or warnings contained in the FDA-approved label for such products;
|
•
|
availability of alternative treatments;
|
•
|
pricing and cost-effectiveness;
|
•
|
the effectiveness of our or any collaborators’ sales and marketing strategies;
|
•
|
our ability to obtain hospital formulary approval;
|
•
|
our ability to obtain and maintain sufficient third party coverage or adequate reimbursement; and
|
•
|
the willingness of patients to pay out-of-pocket in the absence of third party coverage.
|
•
|
different regulatory requirements for drug approvals in foreign countries;
|
•
|
reduced protection for intellectual property rights;
|
•
|
unexpected changes in tariffs, trade barriers and regulatory requirements;
|
•
|
economic weakness, including inflation, or political instability in particular foreign economies and markets;
|
•
|
compliance with tax, employment, immigration and labor laws for employees living or traveling abroad;
|
•
|
foreign taxes, including withholding of payroll taxes;
|
•
|
foreign currency fluctuations, which could result in increased operating expenses and reduced revenues, and other obligations incident to doing business in another country;
|
•
|
workforce uncertainty in countries where labor unrest is more common than in the United States;
|
•
|
production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and
|
•
|
business interruptions resulting from geopolitical actions, including war and terrorism, or natural disasters including earthquakes, typhoons, floods and fires.
|
•
|
the federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, receiving, offering or paying remuneration, directly or indirectly, to induce, or in return for, either the referral of an individual, or the purchase or recommendation of an item or service for which payment may be made under a federal healthcare program, such as the Medicare and Medicaid programs;
|
•
|
federal civil and criminal false claims laws and civil monetary penalty laws, including the civil False Claims Act, which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment to the federal government, including Medicare or Medicaid, that are false or fraudulent;
|
•
|
the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which created additional federal criminal statutes that prohibit, among other things, executing a scheme to defraud any healthcare benefit program and making false statements relating to healthcare matters;
|
•
|
HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009, or HITECH, and their implementing regulations, which imposes certain requirements on certain types of individuals and entities relating to the privacy, security and transmission of individually identifiable health information;
|
•
|
the federal Physician Payments Sunshine Act, which 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 annually to the Centers for Medicare & Medicaid Services, or CMS, information related to payments or other transfers of value made to physicians, and further requires applicable manufacturers and applicable group purchasing organizations to report annually to CMS ownership and investment interests held by physicians and their immediate family members; and
|
•
|
state and foreign law equivalents of each of the above federal laws, such as: anti-kickback and false claims laws which may apply to items or services reimbursed by any third party payor, including commercial insurers;state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government; state laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures; and state and foreign laws governing the privacy and security of health information in certain circumstances, many of which differ from each other in significant ways and may not have the same effect, thus complicating compliance efforts.
|
•
|
an annual, 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;
|
•
|
an increase in the statutory minimum rebates a manufacturer must pay under the Medicaid Drug Rebate Program to 23.1% and 13.0% of the average manufacturer price for branded and generic drugs, respectively;
|
•
|
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;
|
•
|
extension of a manufacturer’s Medicaid rebate liability to covered drugs dispensed to individuals who are enrolled in Medicaid managed care organizations;
|
•
|
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;
|
•
|
a new Medicare Part D coverage gap discount program, in which manufacturers must agree to offer 50% point-of-sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for a manufacturer’s outpatient drugs to be covered under Medicare Part D;
|
•
|
expansion of the entities eligible for discounts under the Public Health Service pharmaceutical pricing program; and
|
•
|
a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research.
|
•
|
impairment of our business reputation;
|
•
|
withdrawal of clinical trial participants;
|
•
|
costs due to related litigation;
|
•
|
distraction of management’s attention from our primary business;
|
•
|
substantial monetary awards to patients or other claimants;
|
•
|
the inability to commercialize our product candidates; and
|
•
|
decreased demand for our product candidates, if approved for commercial sale.
|
•
|
adverse results or delays in preclinical studies or clinical trials;
|
•
|
inability to obtain additional funding;
|
•
|
any delay in filing an IND or NDA for any of our product candidates and any adverse development or perceived adverse development with respect to the FDA’s review of that IND or NDA;
|
•
|
failure to maintain our existing strategic alliances or enter into new alliances;
|
•
|
failure of our strategic alliance partners to elect to develop and commercialize product candidates under our alliance agreements or the termination of any programs under our alliance agreements;
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failure by us or our licensors and strategic alliance partners to prosecute, maintain or enforce our intellectual property rights;
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failure to successfully develop and commercialize our product candidates;
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changes in laws or regulations applicable to our preclinical and clinical development activities, product candidates or future products;
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inability to obtain adequate product supply for our product candidates or the inability to do so at acceptable prices;
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adverse regulatory decisions;
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introduction of new products, services or technologies by our competitors;
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failure to meet or exceed financial projections we may provide to the public;
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failure to meet or exceed the estimates and projections of the investment community;
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the perception of the pharmaceutical industry by the public, legislatures, regulators and the investment community;
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announcements of significant acquisitions, strategic partnerships, joint ventures or capital commitments by us, our strategic alliance partners or our competitors;
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disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain patent protection for our technologies;
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additions or departures of key scientific or management personnel;
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significant lawsuits, including patent or stockholder litigation;
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changes in the market valuations of similar companies;
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sales of our common stock by us or our stockholders in the future; and
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trading volume of our common stock.
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authorizing the issuance of “blank check” preferred stock, the terms of which may be established and shares of which may be issued without stockholder approval;
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prohibiting stockholder action by written consent, thereby requiring all stockholder actions to be taken at a meeting of our stockholders;
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eliminating the ability of stockholders to call a special meeting of stockholders;
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establishing the state of Delaware as the sole forum for certain legal actions against the Company, its officers and directors; and
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•
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establishing advance notice requirements for nominations for election to the board of directors or for proposing matters that can be acted upon at stockholder meetings.
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Regulus Therapeutics Inc.
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||
Date: August 2, 2016
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By:
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/s/ Paul C. Grint
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Paul C. Grint, M.D.
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President and Chief Executive Officer
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(Principal Executive Officer)
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Date: August 2, 2016
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By:
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/s/ Joseph P. Hagan
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Joseph P. Hagan
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Chief Operating Officer
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(Principal Financial and Accounting Officer)
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*
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These certifications are being furnished solely to accompany this quarterly report pursuant to 18 U.S.C. Section 1350, and are not being filed for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, and are not to be incorporated by reference into any filing of the Registrant, whether made before or after the date hereof, regardless of any general incorporation language in such filing.
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