Mavacamten is a potential first-in-class drug for treating obstructive hypertrophic cardiomyopathy (HCM), a chronic and progressive disease where the heart muscle becomes abnormally thick and obstructs blood flow. It affects up to 200,000 people across Europe and the US and there are currently no effective treatment options other than providing symptomatic relief.

“The acquisition of MyoKardia further strengthens our portfolio, pipeline and scientific capabilities and is expected to add a meaningful medium- and long-term growth driver,” said Giovanni Caforio, board chair and CEO of Bristol Myers Squibb.

Both companies’ boards of directors have approved the deal, which is expected to close by the end of this year.

Based on data from the Phase 3 Explorer-HCM trial, a New Drug Application (NDA) for mavacamten is expected to be submitted to the US Food and Drug Administration in the first quarter of 2021. Following the “clinically meaningful” results of the Explorer-HCM trial, analysts at Cantor Fitzgerald said they believed mavacamten could reach sales of $2 billion or more in obstructive HCM, and another $600 million or more from non-obstructive HCM, if approved.

Bristol-Myers Squibb said it expects to explore the full potential of mavacamten in additional indications, including non-obstructive HCM, as well as developing MyoKardia’s pipeline of novel compounds, including two clinical-stage therapeutics: danicamtiv (formerly MYK-491) – an oral small molecule, selective cardiac myosin activator – and MYK-224, which also specifically targets cardiac myosin.

In its cardiovascular portfolio, the US drugmaker already has leading oral anticoagulant brand Eliquis, made with partner Pfizer, as well as antiplatelet medication Plavix, which is manufactured in cooperation with Sanofi.

MyoKardia was previously in a development partnership with Sanofi on mavacamten, as well as danicamtiv and MYK-224, from 2014 through 2018, but the French pharma discontinued their agreement in the first half of 2019. In July 2019, MyoKardia reclaimed from Sanofi the US royalty rights to mavacamten and MYK-224 for $80 million, effectively severing all ties between them.

Author: Elaine Burridge, Freelance Journalist