MyoKardia and Fulcrum Therapeutics Announce Multi-Target Collaboration to Discover Novel Targeted Therapies for Genetic Cardiomyopathies

Rhea-AI Summary

MyoKardia and Fulcrum Therapeutics have formed a strategic collaboration to develop targeted therapies for genetic cardiomyopathies. MyoKardia will utilize Fulcrum's proprietary target discovery engine to identify therapeutics that address key genetic drivers. The agreement involves an upfront payment of $12.5 million, with potential milestone payments up to $302.5 million for the first product, and additional payments if more targets are pursued. MyoKardia aims to leverage its research capabilities to advance drug development and meet the needs of patients with cardiovascular diseases.

Positive

• Strategic collaboration to develop targeted therapies for genetic cardiomyopathies.
• Upfront payment of $12.5 million from MyoKardia to Fulcrum.
• Potential total milestone payments of up to $302.5 million for the first product developed.
• Access to Fulcrum’s proprietary target discovery engine enhances MyoKardia's research capabilities.

Negative

• None.

BRISBANE, Calif. and CAMBRIDGE, Mass., July 21, 2020 (GLOBE NEWSWIRE) -- MyoKardia, Inc. (Nasdaq: MYOK), a clinical-stage biopharmaceutical company discovering and developing targeted therapies for the treatment of serious cardiovascular diseases and Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced today that they have entered into a strategic collaboration and license agreement to discover, develop and commercialize novel targeted therapies for the treatment of genetic cardiomyopathies.

Under the agreement, MyoKardia will access Fulcrum’s unique, proprietary target discovery engine to identify therapeutics that control the expression of genes that are known to be underlying drivers of genetic cardiomyopathies. The collaboration focuses joint discovery efforts on certain undisclosed genetic targets. MyoKardia will be responsible for all development and commercialization activities for, and will have global rights to, any potential therapeutics identified through this collaboration.

Fulcrum will receive a payment of $12.5 million at the close of the transaction and may be eligible to receive research, development and commercial milestone payments and additional research reimbursement of up to $302.5 million for a first product to progress through development and commercialization. If MyoKardia chooses to develop and commercialize products directed to additional targets under the collaboration, Fulcrum may be eligible for up to $150.0 million in milestone payments. Fulcrum may also be eligible to receive tiered royalty payments in the mid-single-digit to low double-digit range on net sales for any products under the collaboration that are commercialized.

MyoKardia intends to select targets for further exploration under this collaboration informed by its integrated research and development engine, which includes capabilities in translational research, proprietary and novel disease models, clinical development, and patient engagement and identification. Potential diseases associated with such undisclosed targets are expected to share common characteristics with indications currently being pursued by MyoKardia: strong and genetically-validated mechanistic rationale, high unmet patient need, potentially efficient pathway to approval, and synergy with the commercial organization that MyoKardia is building.

“This partnership is a natural extension of MyoKardia’s investments over the last eight years in building a world-leading cardiovascular research, translational, clinical and commercial organization. We believe this collaboration will enable us to leverage our unique strengths to expand thoughtfully in identifying new therapeutic candidates for the potential treatment of heritable cardiomyopathies,” said Robert S. McDowell, Ph.D., MyoKardia’s Chief Scientific Officer. “We have been impressed by Fulcrum’s ability to discover new biology around genetic muscle disorders. By working together, we hope to further our mission to treat patients suffering from serious cardiovascular disease.”

“This collaboration highlights the broad applicability of our product engine to discover and develop new treatments in genetically defined rare diseases with high unmet need,” said Robert J. Gould, Ph.D., Fulcrum’s President and Chief Executive Officer.  “We are pleased to partner with MyoKardia, a leader in the field of precision cardiovascular medicine and look forward to leveraging their unique capabilities to rapidly advance potential treatments to serve patients in urgent need.”

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.

About Fulcrum Therapeutics
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and is advancing losmapimod to Phase 3 for the treatment of COVID-19. Fulcrum also anticipates a regulatory filing in the second half of 2020 with FTX-6058 for the treatment of sickle cell disease.

Please visit www.fulcrumtx.com.

MyoKardia Contacts Michelle Corral Executive Director, Corporate Communications and Investor Relations MyoKardia, Inc. 650-351-4690 ir@myokardia.com Hannah Deresiewicz (investors) Stern Investor Relations, Inc. 212-362-1200 hannah.deresiewicz@sternir.com Julie Normant (media) W2O 628-213-3754 jnormart@w2ogroup.com		Fulcrum Contacts Christi Waarich Director, Corporate Communications and Investor Relations Fulcrum Therapeutics, Inc. 617-651-8664 cwaarich@fulcrumtx.com Stephanie Ascher (investors) Stern Investor Relations, Inc. 212-362-1200 stephanie.ascher@sternir.com Kaitlin Gallagher (media) Berry & Company Public Relations 212-253-8881 kgallagher@berrypr.com

FAQ

What is the strategic collaboration between MYOK and Fulcrum Therapeutics?

MyoKardia has entered into a collaboration with Fulcrum Therapeutics to develop targeted therapies for genetic cardiomyopathies using Fulcrum's proprietary target discovery engine.

What financial terms were disclosed in the MYOK press release?

MyoKardia will pay Fulcrum $12.5 million upfront, with potential milestone payments up to $302.5 million for the first product and additional payments for further targets.

What are the goals of the MYOK and Fulcrum collaboration?

The collaboration aims to identify therapeutics addressing genetic cardiomyopathies, leveraging both companies' strengths in research and development.

How might this collaboration impact MYOK stock performance?

The strategic partnership may enhance MyoKardia’s product pipeline and market positioning in genetic cardiomyopathies, potentially positively affecting MYOK stock performance.