Received FDA Feedback for Mavacamten Registration Program in Symptomatic, Obstructive Hypertrophic Cardiomyopathy
MyoKardia to Initiate EXPLORER-HCM as Pivotal Trial in Second Quarter 2018
Company to Host Conference Call and Webcast Today at 4:30 p.m. ET (1:30 p.m. PT)
SOUTH SAN FRANCISCO, Calif., Nov. 02, 2017 (GLOBE NEWSWIRE) -- MyoKardia, Inc. (Nasdaq:MYOK), a clinical-stage biopharmaceutical company pioneering a precision medicine approach for the treatment of heritable cardiovascular diseases, today reported financial results for the third quarter ended September 30, 2017.
MyoKardia also provided an update on the company’s End-of-Phase 2 meeting with the U.S. Food and Drug Administration’s (FDA) Division of Cardiovascular and Renal Products. The meeting focused on MyoKardia’s proposed registration program for mavacamten (formerly MYK-461) in symptomatic, obstructive hypertrophic cardiomyopathy (oHCM). MyoKardia is incorporating guidance from the FDA on study design elements and plans to proceed with the pivotal Phase 3 trial, EXPLORER-HCM, and a long-term extension study, as the key remaining studies in the mavacamten registration program. The company anticipates dosing the first patient in EXPLORER-HCM in the second quarter of 2018. Between now and study start, the company plans to review the final trial design with the FDA.
“It has been a remarkable quarter for MyoKardia, highlighted by the PIONEER-HCM Phase 2 results and by our meeting with the FDA further clarifying our path to registration for mavacamten,” said Tassos Gianakakos, Chief Executive Officer. “The progress we’ve made across our clinical pipeline has generated important disease insights that we believe strengthen our research platform. In the months to come, we expect to report important progress in our MYK-491 clinical program for dilated cardiomyopathy, present mavacamten data at the 2017 American Heart Association annual meeting and initiate a Phase 2 study of mavacamten in its second indication, non-obstructed HCM.”
Clinical Updates and Highlights
Mavacamten for Hypertrophic Cardiomyopathy (HCM)
MYK-491 for Dilated Cardiomyopathy (DCM)
Corporate Updates and Highlights
Third Quarter and Year-to-Date 2017 Financial Results
Based on its current operating plans, the Company expects that its cash, cash equivalents and investments as of September 30, 2017, together with anticipated payments from Sanofi under its collaboration agreement, will enable the Company to fund its anticipated operating expenses and capital expenditure requirements at least into 2020.
Conference Call and Webcast
MyoKardia will host a conference call and live audio webcast on Thursday, November 2, 2017 at 4:30 p.m. ET / 1:30 p.m. PT. The call may be accessed by phone by calling (844) 494-0193 from the U.S. and Canada or (508) 637-5584 internationally and using the conference ID 7295509. The webcast may be accessed live on the Investor Relations section of the Company's website at http://investors.myokardia.com. A replay of the webcast will be available on the MyoKardia website for 90 days following the call.
About MyoKardia
MyoKardia is a clinical-stage biopharmaceutical company pioneering a precision medicine approach to discover, develop and commercialize targeted therapies for the treatment of serious and rare cardiovascular diseases. MyoKardia’s initial focus is on the treatment of heritable cardiomyopathies, a group of rare, genetically-driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction. MyoKardia has used its precision medicine platform to generate a pipeline of therapeutic programs for the chronic treatment of the two most prevalent forms of heritable cardiomyopathy—hypertrophic cardiomyopathy (HCM), and dilated cardiomyopathy (DCM). MyoKardia’s most advanced product candidate is mavacamten (formerly MYK-461), a novel, oral, allosteric modulator of cardiac myosin that has been shown to reduce hypercontractility in early clinical studies and is currently being studied in the Phase 2 PIONEER-HCM clinical trial. MYK-491, MyoKardia’s second product candidate, is designed to increase the overall extent of the heart’s contraction in DCM patients by increasing cardiac contractility. MyoKardia is currently evaluating MYK-491 in a Phase 1 study in healthy volunteers. A cornerstone of the MyoKardia platform is the Sarcomeric Human Cardiomyopathy Registry (SHaRe), a multi-center, international repository of clinical and laboratory data on individuals and families with genetic heart disease, which MyoKardia helped form in 2014. MyoKardia’s mission is to change the world for patients with serious cardiovascular disease through bold and innovative science.
Forward-Looking Statements
Statements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are usually identified by the use of words such as "anticipates," "believes," "estimates," "expects," "intends," "may," "plans," "projects," "seeks," "should," "will," and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements regarding the clinical and therapeutic potential of mavacamten and MYK-491, the commencement of enrollment and patient dosing in the EXPLORER-HCM trial, the trial design for EXPLORER-HCM, the release of topline data from the Company’s lower-dose cohort in the PIONEER-HCM trial, the commencement of a Phase 2 trial of mavacamten in nHCM patients, the release of topline data from the Phase 1 single ascending dose trial of MYK-491 in healthy volunteers and the initiation of the Company’s planned Phase 1b single ascending dose trial of MYK-491 in DCM patients, as well as the timing of these events, and the Company’s expected cash runway and ability to receive additional payments from its collaboration agreement with Sanofi, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, risks associated with the development and regulation of our product candidates, as well as those set forth in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2017, and our other filings with the SEC. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
MYOKARDIA, INC.
Condensed Consolidated Balance Sheets
(In thousands, except share and per share amounts)
(Unaudited)
September 30, 2017 | December 31, 2016 | |||||||
Assets | ||||||||
Current assets | ||||||||
Cash and cash equivalents | $ | 227,184 | $ | 135,797 | ||||
Short-term investments | 19,993 | 4,072 | ||||||
Receivable from collaboration partner | — | 45,000 | ||||||
Prepaid expenses and other current assets | 1,545 | 1,394 | ||||||
Total current assets | 248,722 | 186,263 | ||||||
Property and equipment, net | 2,633 | 2,758 | ||||||
Long-term investments | 36,003 | 12,002 | ||||||
Other long-term assets | 432 | 283 | ||||||
Total assets | $ | 287,790 | $ | 201,306 | ||||
Liabilities and stockholders’ equity | ||||||||
Current liabilities | ||||||||
Accounts payable | $ | 2,030 | $ | 1,798 | ||||
Accrued liabilities | 10,634 | 8,690 | ||||||
Deferred revenue - current | 22,500 | 22,500 | ||||||
Total current liabilities | 35,164 | 32,988 | ||||||
Other long-term liabilities | 246 | 436 | ||||||
Deferred revenue - noncurrent | 5,625 | 22,500 | ||||||
Total liabilities | 41,035 | 55,924 | ||||||
Commitments and contingencies (Note 6) | ||||||||
Stockholders’ equity | ||||||||
Preferred stock, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding | — | — | ||||||
Common stock, $0.0001 par value, 150,000,000 and 150,000,000 shares authorized at September 30, 2017 and December 31, 2016, respectively; 35,733,002 and 31,428,998 shares issued and outstanding at September 30, 2017 and December 31, 2016, respectively | 4 | 3 | ||||||
Additional paid-in capital | 363,205 | 223,208 | ||||||
Accumulated other comprehensive (loss) income | (52 | ) | 8 | |||||
Accumulated deficit | (116,402 | ) | (77,837 | ) | ||||
Total stockholders’ equity | 246,755 | 145,382 | ||||||
Total liabilities and stockholders’ equity | $ | 287,790 | $ | 201,306 |
The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.
MYOKARDIA, INC.
Condensed Consolidated Statements of Operations and Comprehensive Loss
(In thousands, except share and per share amounts)
(Unaudited)
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
2017 | 2016 | 2017 | 2016 | |||||||||||||
Collaboration and license revenue | $ | 5,625 | $ | 3,550 | $ | 16,875 | $ | 10,649 | ||||||||
Operating expenses: | ||||||||||||||||
Research and development | 14,361 | 8,783 | 39,967 | 26,192 | ||||||||||||
General and administrative | 5,884 | 4,031 | 16,442 | 11,947 | ||||||||||||
Total operating expenses | 20,245 | 12,814 | 56,409 | 38,139 | ||||||||||||
Loss from operations | (14,620 | ) | (9,264 | ) | (39,534 | ) | (27,490 | ) | ||||||||
Interest and other income, net | 447 | 33 | 977 | 79 | ||||||||||||
Net loss | (14,173 | ) | (9,231 | ) | (38,557 | ) | (27,411 | ) | ||||||||
Other comprehensive loss | 2 | — | 60 | — | ||||||||||||
Comprehensive loss | (14,171 | ) | (9,231 | ) | (38,497 | ) | (27,411 | ) | ||||||||
Net loss attributable to common stockholders | $ | (14,173 | ) | $ | (9,231 | ) | $ | (38,557 | ) | $ | (27,411 | ) | ||||
Net loss per share attributable to common stockholders, basic and diluted | $ | (0.42 | ) | $ | (0.35 | ) | $ | (1.21 | ) | $ | (1.04 | ) | ||||
Weighted average number of shares used to compute net loss per share attributable to common stockholders, basic and diluted | 33,525,567 | 26,470,298 | 31,951,631 | 26,331,852 |
The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.
Contacts:
Michelle Corral
Corporate Communications & Investor Relations
MyoKardia, Inc.
650-351-4690
[email protected]
Beth DelGiacco (Investors)
Stern Investor Relations, Inc.
212-362-1200
[email protected]
Steven Cooper (Media)
Edelman
415-486-3264
[email protected]
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