RARE Daily

MyoKardia and Fulcrum Therapeutics Enter Collaboration to Discover Targeted Therapies for Genetic Cardiomyopathies

July 21, 2020

Rare Daily Staff

Photo: Robert McDowell, MyoKardia’s chief scientific officer

MyoKardia has entered into a strategic collaboration and license agreement with Fulcrum Therapeutics 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 said it will select targets that share common characteristics with indications it is already pursuing. This includes ones that have strong and genetically validated mechanistic rationale, address high unmet patient need, have a potentially efficient pathway to approval, and provide a 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,” said Robert McDowell, MyoKardia’s chief scientific officer. “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.”  

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