Innovations Advanced by M&A: A First-In-Class Treatment for Hypertrophic Cardiomyopathy

Oct 2, 2024 | Blog Post, Innovations Advanced by M&A

This blog is another installment in a series, Innovations Advanced by M&A, that underscores the importance of mergers and acquisitions in ensuring a competitive and vibrant biopharmaceutical landscape for patients. You can find previous blogs in this series here.

Mergers and acquisitions (M&A) are a vital mechanism for transitioning treatments currently in development to approved medicines available to the patients who need them the most. In one such example, the synergies from a life science acquisition brought a revolutionary therapy for hypertrophic cardiomyopathy (HCM) to patients.

In 2013, a group of scientists, including Drs. Christine and Jonathan Seidman – a husband-and-wife pair who discovered the genetic cause of HCM – founded MyoKardia. Along with their partners, Dr. Leslie Lewand and Dr. James Spudich, they began investigating cutting-edge genetically targeted therapies for cardiac disorders.

HCM is the most common genetic cardiac disorder, affecting as many as 1 in 500 people. It causes a thickening of the heart walls, which can obstruct the flow of blood from the heart to the rest of the body. HCM can cause chest pain, abnormal heart rhythms, fainting, shortness of breath and swelling of the legs and feet. It is also the leading cause of sudden cardiac death in young athletes, particularly Black athletes.

Mavacamten quickly emerged as a lead candidate for the treatment of obstructive HCM within MyoKardia’s pipeline. In 2019, MyoKardia sought to collaborate with Bristol Myers Squibb (BMS) to connect its innovative pipeline with the global reach and capabilities of a larger company, and was acquired by BMS shortly thereafter.

“Bristol Myers Squibb shares our vision for transforming the treatment of cardiovascular disease…Together, our complementary strengths and expanded resources and reach will further accelerate the pace at which we can discover, develop and commercialize our novel medicines for the benefit of people suffering from cardiovascular disease around the world.”

– Tassos Gianakakos, former Chief Executive Officer, MyoKardia

The acquisition brought together MyoKardia’s highly specialized scientific knowledge in the field of cardiovascular genetics and the substantial infrastructure and expertise of BMS in the global distribution of cardiovascular therapies. In April of 2022, the combined company received FDA approval for mavacamten (branded as Camzyos) to treat obstructive HCM.

“Our companies share a commitment to innovation and bold science, and our respective strengths will help us realize the value inherent in this portfolio. We have long admired MyoKardia and what they have done to revolutionize cardiovascular treatments through a precision medicine approach. We look forward to welcoming their talented team to our company.”

– Giovanni Caforio, former Chief Executive Officer, Bristol Myers Squibb

Once approved, mavacamten became the first therapy to target the root cause of HCM, and the first new medicine approved to treat genetic cardiac diseases in over a decade. The potential benefits of mavacamten are also being explored for other serious cardiovascular diseases through ongoing clinical trials, including for heart failure.

M&A represents a vital bridge for life sciences companies to be able to advance new innovations to patients and address critical unmet medical needs – a reality that policymakers must recognize and preserve.