Announced Achievement of Primary Endpoint and Additional Key Data from Low-Dose
Mavacamten Cohort of PIONEER-HCM Phase 2 Study in Symptomatic, Obstructive
Hypertrophic Cardiomyopathy
Company to Host Conference Call and Webcast Today at 4:30 p.m. ET (1:30 p.m. PT)
SOUTH SAN FRANCISCO, Calif., March 08, 2018 (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 fourth quarter and full year ended December 31, 2017. In addition, the company also announced positive results from the PIONEER-HCM Phase 2 proof-of-concept trial, including results from the low-dose cohort, in a press release issued this afternoon.
“Following an eventful and important year of progress, we are poised in 2018 to move closer to achieving our mission to change the lives of patients suffering from serious cardiovascular diseases,” said Tassos Gianakakos, Chief Executive Officer. “As we prepare to initiate our Phase 3 EXPLORER-HCM pivotal trial, the additional PIONEER-HCM study data announced today increase our confidence in mavacamten’s potential to help patients with oHCM. This enthusiasm carries over into our growing clinical pipeline, which also includes the Phase 2 study of mavacamten in non-obstructive HCM and the continued clinical study of MYK-491 in dilated cardiomyopathy patients.”
Full Year 2017 and Recent Clinical Program Highlights
Mavacamten for Hypertrophic Cardiomyopathy (HCM)
MYK-491 for Dilated Cardiomyopathy (DCM)
Corporate Updates
2018 Anticipated Milestones
Fourth Quarter and Full Year 2017 Financial Results
2018 Financial Guidance
Conference Call and Webcast
MyoKardia management will host a conference call and live audio webcast today, March 8, at 4:30 p.m. ET / 1:30 p.m. PT to review data from the Phase 2 PIONEER-HCM clinical trial, as well as fourth quarter and year end 2017 financial results. 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 8496928. 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 two of the 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 intended to reduce hypercontractility. Mavacamten is advancing into a pivotal Phase 3 clinical trial, known as EXPLORER-HCM in patients with symptomatic, obstructive HCM and a Phase 2 trial, the MAVERICK-HCM study, in patients with non-obstructive HCM. 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 1b study in DCM patients. 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, our use of data from the PIONEER-HCM trial to guide the starting dose and inform the dose-adjustments for our planned Phase 3 EXPLORER-HCM trial, the initiation of patient dosing in the Phase 3 EXPLORER-HCM trial, the trial design for EXPLORER-HCM, the release of complete results from the Company’s Phase 2 PIONEER-HCM trial, the commencement of a Phase 2 trial of mavacamten in nHCM patients, the open-label extension study for patients who participated in the Phase 2 PIONEER-HCM trial, the release of topline data from the Phase 1b clinical trial of MYK-491 in DCM patients and the initiation of the Company’s planned Phase 2 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 Annual Report on Form 10-K for the year ended December 31, 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.
Consolidated Statements of Operations and Comprehensive Loss
(In thousands, except share and per share amounts)
Year Ended December 31, | ||||||||||||
2017 | 2016 | 2015 | ||||||||||
Collaboration and license revenue | $ | 22,500 | $ | 39,199 | $ | 14,199 | ||||||
Operating expenses: | ||||||||||||
Research and development, net | 48,136 | 36,215 | 28,393 | |||||||||
General and administrative | 21,973 | 16,289 | 9,019 | |||||||||
Total operating expenses | 70,109 | 52,504 | 37,412 | |||||||||
Loss from operations | (47,609 | ) | (13,305 | ) | (23,213 | ) | ||||||
Interest and other income (loss), net | 1,657 | 153 | (47 | ) | ||||||||
Change in fair value of redeemable convertible preferred stock call option liability | — | — | 314 | |||||||||
Net loss | (45,952 | ) | (13,152 | ) | (22,946 | ) | ||||||
Other comprehensive (loss) income | (200 | ) | 8 | — | ||||||||
Comprehensive loss | (46,152 | ) | (13,144 | ) | (22,946 | ) | ||||||
Cumulative dividend relating to redeemable convertible preferred stock | — | — | (5,151 | ) | ||||||||
Accretion of redeemable convertible preferred stock to redemption value | — | — | (98 | ) | ||||||||
Net loss attributable to common stockholders | $ | (45,952 | ) | $ | (13,152 | ) | $ | (28,195 | ) | |||
Net loss per share attributable to common stockholders, basic and diluted | $ | (1.40 | ) | $ | (0.48 | ) | $ | (4.48 | ) | |||
Weighted average number of shares used to compute net loss per share attributable to common stockholders, basic and diluted | 32,832,514 | 27,475,792 | 6,292,800 | |||||||||
MYOKARDIA, INC.
Consolidated Balance Sheets
(In thousands, except share and per share amounts)
As of December 31, | ||||||||
2017 | 2016 | |||||||
Assets | ||||||||
Current assets | ||||||||
Cash and cash equivalents | $ | 224,571 | $ | 135,797 | ||||
Short-term investments | 31,933 | 4,072 | ||||||
Receivable from collaboration partner | 1,013 | 45,000 | ||||||
Prepaid expenses and other current assets | 1,876 | 1,394 | ||||||
Total current assets | 259,393 | 186,263 | ||||||
Property and equipment, net | 3,147 | 2,758 | ||||||
Long-term investments | 19,900 | 12,002 | ||||||
Other long-term assets | 368 | 283 | ||||||
Total assets | $ | 282,808 | $ | 201,306 | ||||
Liabilities and stockholders’ equity | ||||||||
Current liabilities | ||||||||
Accounts payable | $ | 2,301 | $ | 1,798 | ||||
Accrued liabilities | 11,639 | 8,690 | ||||||
Prepayment from collaboration partner | 4,432 | — | ||||||
Deferred revenue - current | 22,500 | 22,500 | ||||||
Total current liabilities | 40,872 | 32,988 | ||||||
Other long-term liabilities | 202 | 436 | ||||||
Deferred revenue - noncurrent | — | 22,500 | ||||||
Total liabilities | 41,074 | 55,924 | ||||||
Commitments and contingencies | ||||||||
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, 2017 and 2016, respectively; 35,812,791 and 31,428,998 shares, issued and outstanding at December 31, 2017 and 2016, respectively | 4 | 3 | ||||||
Additional paid-in capital | 365,719 | 223,208 | ||||||
Accumulated other comprehensive (loss) income | (192 | ) | 8 | |||||
Accumulated deficit | (123,797 | ) | (77,837 | ) | ||||
Total stockholders’ equity | 241,734 | 145,382 | ||||||
Total liabilities and stockholders’ equity | $ | 282,808 | $ | 201,306 |
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]
We use cookies to tailor your experience, measure site performance and present relevant offers and advertisements. By clicking ‘Accept’ or any content on this site, you agree that cookies can be placed on your browser. You can view our privacy policy to learn more.
If you would like to get more data, alerts and access to Real Vision videos, join us as an Insider Tracking Advantage Ultra member