August 11, 2020

MyoKardia and LianBio Form Strategic Partnership to Develop and Commercialize Mavacamten Together in Greater China

Near-Term Focus on Pursuing Approval of Mavacamten for Obstructive HCM

Innovative Model Focused on Long-Term Value Creation: MyoKardia Receives Equity Position in Lian Cardiovascular and Tassos Gianakakos, MyoKardia’s CEO, to Join LianBio Board of Directors

Brisbane, Calif – August 11, 2020 – MyoKardia, Inc. (Nasdaq: MYOK) and LianBio announced today that they have entered into a strategic collaboration to develop and commercialize mavacamten in China and other Asian territories. Mavacamten is MyoKardia’s lead therapeutic candidate being developed for the treatment of patients with hypertrophic cardiomyopathy (HCM), as well as in a targeted population of patients with heart failure with preserved ejection fraction (HFpEF). The partnership will initially pursue a registration strategy for mavacamten in China for obstructive HCM, with plans for additional indications to follow, in keeping with MyoKardia’s development strategy.

LianBio was recently launched by Perceptive Advisors to partner with leading biopharmaceutical companies, such as MyoKardia, to accelerate the availability of innovative medicines addressing diseases where the unmet need is significant across China. Led by a seasoned executive team with deep regional experience, including Bing Li, CEO and Debra Yu, President and CBO, LianBio’s initial portfolio represents a diverse array of late-stage, transformative therapeutics for development and commercialization in China and major Asian markets.

“Hypertrophic cardiomyopathy is estimated to affect more than one million people in China alone, yet there are limited treatment options and none that target the pathophysiology of disease,” said Bing Li, LianBio’s Chief Executive Officer. “Mavacamten’s mechanism is directed to the hypercontractility that drives HCM and we have been impressed by its consistent and encouraging clinical data.”

Mavacamten will serve to anchor the portfolio for LianBio’s cardiorenal-focused subsidiary, Lian Cardiovascular. Lian Cardiovascular has assembled a strong team across development, regulatory and other key functions, including Jing Ji, who previously served as Vice President and Head of Cardiovascular, Renal and Metabolic TA for AstraZeneca China. The partnership is structured to enable MyoKardia to retain certain key decision-making input over the mavacamten program in the collaboration territory as the two companies bring their respective expertise to bear to the collaboration and share learnings from each other’s respective markets . MyoKardia will receive an equity stake in Lian Cardiovascular and Tassos Gianakakos, MyoKardia’s Chief Executive Officer, will be appointed to LianBio’s Board of Directors.

“The partnership with LianBio is consistent with MyoKardia’s strategy of adding capabilities to extend our efforts to bring precision cardiovascular therapeutics to patients in need while maintaining significant influence on both our global brand and economic rights,” said Jake Bauer, MyoKardia’s Chief Business Officer. “The LianBio leadership team brings an in-depth understanding of local market dynamics and a successful track record for regional drug development, as well as a shared commitment to accelerating the availability of mavacamten in China and other territories. This combination of proven know-how and impassioned drive to bring innovative targeted treatments to patients make them an ideal partner.”

“We have structured LianBio with the intent of greatly expanding our partner’s ability to succeed as we work together to bring exciting new therapeutics to patients in need,” added Konstantin Poukalov, LianBio’s Executive Chairman and Managing Director of Perceptive Advisors. “The collaboration with MyoKardia to bring a first-in-class targeted therapeutic with the potential to be a game-changer for cardiovascular patients, such as mavacamten, to patients in China exemplifies our aim for Lian Cardiovascular.”

In addition to its equity position, under the terms of the agreement, MyoKardia will a receive a payment of $40 million at closing, with additional committed payments within the first six months of the partnership. MyoKardia may be eligible to receive regulatory and sales milestone payments of up to $147.5 million, as well as tiered double-digit royalties on the sale of mavacamten in the territories outlined as part of the collaboration. LianBio will fund all development and commercial expenses in the collaboration territory.

About Mavacamten (MYK-461)
MyoKardia is developing mavacamten, a first-in-class, oral, allosteric modulator of cardiac myosin, for the treatment of conditions in which excessive cardiac contractility and impaired diastolic filling of the heart are the underlying cause. Mavacamten reduces cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance. In clinical and preclinical studies, mavacamten has consistently reduced biomarkers of cardiac wall stress, lessened excessive cardiac contractility and increased diastolic compliance.

Mavacamten is initially being developed for the treatment of hypertrophic cardiomyopathy (HCM) and has received breakthrough therapy and orphan drug designations for symptomatic, obstructive HCM. MyoKardia plans to submit a New Drug Application (NDA) for U.S. regulatory approval in this indication in the first quarter of 2021. Based on its mechanism of action and evidence of therapeutic activity, mavacamten is also being studied in the clinic for the treatment of symptomatic non-obstructive HCM, and the company plans to conduct future studies among a targeted population of patients with heart failure with preserved ejection fraction (HFpEF).

About LianBio
LianBio’s mission is to catalyze the development and accelerate availability of paradigm-shifting medicines to patients in China and major Asian markets through partnerships that provide access to the best science-driven therapeutic discoveries. LianBio collaborates with world-class partners across a diverse array of therapeutic and geographic areas to build out a pipeline based on disease relevance and the ability to impact patients with transformative mechanisms and precision-based therapeutics. For more information, please visit

About MyoKardia
MyoKardia is a clinical-stage biopharmaceutical company discovering and developing targeted therapies for the treatment of serious cardiovascular diseases. The company is pioneering a precision medicine approach to its discovery and development efforts by (1) understanding the biomechanical underpinnings of disease; (2) targeting the proteins that modulate a given condition; (3) identifying patient populations with shared disease characteristics; and (4) applying learnings from research and clinical studies to inform and guide pipeline growth and product advancement. MyoKardia’s initial focus is on small molecule therapeutics aimed at the proteins of the heart that modulate cardiac muscle contraction to address diseases driven by excessive contraction, impaired relaxation, or insufficient contraction. Among its discoveries are three clinical-stage therapeutics: mavacamten (formerly MYK-461); danicamtiv (formerly MYK-491) and MYK-224.

MyoKardia’s mission is to change the world for people with serious cardiovascular disease through bold and innovative science.

MyoKardia Forward-Looking Statement
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 mavacamten’s successful development for the treatment of patients with HCM and, in some instances, HFpEF, as well as non-obstructive HCM, our registration strategy for mavacamten in China for obstructive HCM, our plans for additional indications as well as the likelihood of adding capabilities to bring precision cardiovascular therapeutics to patients in need, or our timing regarding NDA submission 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, 2020, 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.

Michelle Corral
Executive Director, Corporate Communications and Investor Relations
MyoKardia, Inc.

Hannah Deresiewicz (investors)
Stern Investor Relations, Inc.

Julie Normant (media)

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