Topline Data from Phase 2 PIONEER-HCM Trial of MYK-461 in Symptomatic Obstructive Hypertrophic Cardiomyopathy Expected in Third Quarter of 2017
MYK-491 Dilated Cardiomyopathy Candidate Phase 1 Study of Healthy Volunteers Under Way; Topline Data Expected in Third Quarter of 2017
Sanofi Payment Received for Global Cardiomyopathy Research Collaboration
Strong Cash Position to Further Advance Programs
SOUTH SAN FRANCISCO, Calif., March 13, 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 business highlights and financial results for the fourth quarter and year ended December 31, 2016.
“The year 2016 saw MyoKardia deepen our leadership, expand our scientific platform and advance our growing clinical pipeline,” said Tassos Gianakakos, chief executive officer. “We enter 2017 with two programs in the clinic aimed at the two most common heritable cardiomyopathies, HCM and DCM, and sufficient cash to fund operations into 2019. Both programs have data reporting out in the third quarter of this year that we hope will bring us one step closer to helping HCM and DCM patients.”
During 2016, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for MYK-461 for the treatment of symptomatic, obstructive hypertrophic cardiomyopathy (oHCM). Topline data from the PIONEER-HCM trial of MYK-461, and the Company’s Phase 1 study of dilated cardiomyopathy (DCM) candidate MYK-491, are expected in the third quarter of 2017.
“We are proud of the progress MyoKardia made in 2016 in reaching clinical milestones and broadening our pipeline of novel therapies that target the underlying biomechanical defects of the heart muscle,” said Marc Semigran, M.D., chief medical officer. “As these programs advance in the clinic, we continue to execute on our mission to change the world for patients with serious cardiovascular disease through bold and innovative science.”
Upcoming Clinical Milestones
Development and Product Pipeline
HCM Program
DCM Program
Leadership and Business Momentum
Research
Cardiomyopathy Community and Patient Outreach
Fourth Quarter and 2016 Financial Results
About MYK-461
MYK-461 is an oral small molecule designed to reduce left ventricular contractility by allosterically modulating the function of cardiac myosin, the motor protein that drives heart muscle contraction. MyoKardia has evaluated MYK-461 in three Phase 1 clinical trials, primarily designed to evaluate safety and tolerability of oral doses of MYK-461, as well as provide pharmacokinetic and pharmacodynamic data. In April 2016, the U.S. FDA granted Orphan Drug Designation for MYK-461 for the treatment of symptomatic oHCM, a subset of HCM.
MyoKardia is currently studying MYK-461 in PIONEER-HCM, a Phase 2 open-label single-arm study to evaluate safety, tolerability and efficacy of MYK-461 in patients with symptomatic oHCM. The primary endpoint of PIONEER-HCM is the level of reduction in post-exercise left ventricular outflow tract (LVOT) gradient over 12 weeks of drug treatment. PIONEER-HCM will also explore the relationship between reduction in contractility and LVOT gradient, endpoints measuring functional capacity (i.e., exercise) and clinical symptoms in addition to gathering safety and tolerability data on MYK-461 in an outpatient setting.
About MYK-491
The oral small molecule MYK-491 is an allosteric modulator of myosin designed to increase cardiac contractility in a DCM heart. Like MYK-461, the Company’s candidate for hypertrophic cardiomyopathy, MYK-491 targets the underlying biomechanical defects of the heart muscle. Based on preclinical research across multiple animal models, MYK-491 may hold potential for controlled increases in the heart’s contractility with minimal impact on diastole or relaxation. MyoKardia initiated a Phase 1 study of MYK-491 in healthy volunteers in early 2017, with topline results expected in the third quarter of 2017.
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, or HCM, and dilated cardiomyopathy, or DCM. MyoKardia’s most advanced product candidate, MYK-461, is an oral small molecule designed to reduce excessive cardiac muscle contractility leading to HCM and has been evaluated in three Phase 1 clinical trials. MyoKardia is currently studying MYK-461 in the Phase 2 PIONEER-HCM trial in symptomatic, obstructive HCM (oHCM), a subset of HCM for which the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation. MYK-491, the second clinical candidate generated by MyoKardia’s product engine, is designed to increase the overall force 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; topline data is expected in the third quarter of 2017. A cornerstone of the MyoKardia platform is the Sarcomeric Human Cardiomyopathy Registry, or 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 believes that SHaRe, currently consisting of data from approximately 10,000 individuals, is the world’s largest registry of patients with heritable cardiomyopathies. MyoKardia’s mission is to change the world for patients with serious cardiovascular disease through bold and innovative science. For more information, please visit www.myokardia.com.
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 MYK-461 and MYK-491, the Company's ability to advance MYK-461 in the PIONEER-HCM trial and MYK-491 in its Phase 1 trial in healthy volunteers and to generate data from these trials, the Company’s ability to initiate additional clinical trials of MYK-461 in symptomatic oHCM and non-obstructive HCM and the timing of these events, as well as the requirements for registration of the Company’s product candidates, the Company’s ability to successfully maintain its collaboration with Sanofi and the Company’s anticipated expenditures and cash runway, 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2016, 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. Consolidated Statements of Operations and Comprehensive Income (Loss) (In thousands, except share and per share amounts) (Unaudited) | ||||||||||||||||
Three Months Ended December 31, | Year Ended December 31, | |||||||||||||||
2016 | 2015 | 2016 | 2015 | |||||||||||||
Collaboration and license revenue | $ | 28,550 | $ | 3,550 | $ | 39,199 | $ | 14,199 | ||||||||
Operating expenses: | ||||||||||||||||
Research and development | 10,023 | 8,376 | 36,215 | 28,393 | ||||||||||||
General and administrative | 4,342 | 3,058 | 16,289 | 9,019 | ||||||||||||
Total operating expenses | 14,365 | 11,434 | 52,504 | 37,412 | ||||||||||||
Loss from operations | 14,185 | (7,884 | ) | (13,305 | ) | (23,213 | ) | |||||||||
Interest and other income, net | 74 | (25 | ) | 153 | (47 | ) | ||||||||||
Change in fair value of redeemable convertible preferred stock call option liability | — | — | — | 314 | ||||||||||||
Net loss | 14,259 | (7,909 | ) | (13,152 | ) | (22,946 | ) | |||||||||
Other comprehensive income | 8 | — | 8 | — | ||||||||||||
Comprehensive loss | 14,267 | (7,909 | ) | (13,144 | ) | (22,946 | ) | |||||||||
Cumulative dividend relating to redeemable convertible preferred stock | — | (621 | ) | — | (5,151 | ) | ||||||||||
Accretion of redeemable convertible preferred stock to redemption value | — | (10 | ) | — | (98 | ) | ||||||||||
Net loss attributable to common stockholders | $ | 14,259 | $ | (8,540 | ) | $ | (13,152 | ) | $ | (28,195 | ) | |||||
Net loss per share attributable to common stockholders, basic | $ | 0.46 | $ | (0.48 | ) | $ | (0.48 | ) | $ | (4.48 | ) | |||||
Weighted average number of shares used to compute net loss per share attributable to common stockholders, basic | 30,878,973 | 17,702,564 | 27,475,792 | 6,292,800 | ||||||||||||
Net loss per share attributable to common stockholders, diluted | $ | 0.44 | $ | (0.48 | ) | $ | (0.48 | ) | $ | (4.48 | ) | |||||
Weighted average number of shares used to compute net loss per share attributable to common stockholders, diluted | 32,228,172 | 17,702,564 | 27,475,792 | 6,292,800 |
MYOKARDIA, INC. Consolidated Balance Sheets (In thousands, except share and per share amounts) (Unaudited) | ||||||||
As of December 31, | ||||||||
2016 | 2015 | |||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 135,797 | $ | 112,265 | ||||
Short-term investments | 4,072 | — | ||||||
Receivable from collaboration partner | 45,000 | — | ||||||
Prepaid expenses and other current assets | 1,394 | 1,282 | ||||||
Total current assets | 186,263 | 113,547 | ||||||
Property and equipment, net | 2,758 | 2,744 | ||||||
Long-term investments | 12,002 | — | ||||||
Other long term assets | 283 | 289 | ||||||
Total assets | $ | 201,306 | $ | 116,580 | ||||
Liabilities and stockholders’ equity | ||||||||
Current liabilities | ||||||||
Accounts payable | $ | 1,798 | $ | 2,143 | ||||
Accrued liabilities | 8,690 | 5,633 | ||||||
Deferred revenue - current | 22,500 | 14,199 | ||||||
Total current liabilities | 32,988 | 21,975 | ||||||
Other long-term liabilities | 436 | 732 | ||||||
Deferred revenue - noncurrent | 22,500 | — | ||||||
Total liabilities | 55,924 | 22,707 | ||||||
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 December 31, 2016 and 2015, respectively; 31,428,998 and 27,053,156 shares, issued and outstanding at December 31, 2016 and 2015, respectively | 3 | 3 | ||||||
Additional paid-in capital | 223,208 | 158,555 | ||||||
Accumulated other comprehensive income | 8 | — | ||||||
Accumulated deficit | (77,837 | ) | (64,685 | ) | ||||
Total stockholders’ equity | 145,382 | 93,873 | ||||||
Total liabilities and stockholders’ equity | $ | 201,306 | $ | 116,580 |