ACC: Bristol Myers drug helps patients avoid heart procedure amid FDA countdown

 If you’re a patient, avoiding a potentially invasive surgical procedure is a pretty obvious goal—especially if it involves the heart. Bristol Myers Squibb is presenting data this weekend that shows its targeted heart drug mavacamten helped reduce the risk of needing septal reduction therapy.

In the phase 3 VALOR study, mavacamten significantly reduced the need for the procedure in patients with severely symptomatic obstructive hypertrophic cardiomyopathy. In this disease, the wall of the heart thickens, sometimes progressing to the point where blood flow is obstructed.

At the end of the study’s 16 weeks, 82% of patients taking mavacamten had not proceeded with septal reduction therapy and no longer met the criteria for the procedure. In the placebo arm, just 23% patients had the same result. The results are being presented Saturday at the American College of Cardiology’s 71st Annual Scientific Session & Expo.

Septal reduction therapy can be done one of two ways. In the less invasive procedure, a catheter is threaded from the groin up to the heart to inject an alcohol solution to “cause a controlled heart attack,” according to Amy Sehnert, BMS VP and mavacamten clinical development team lead, speaking to Fierce Biotech. The solution kills the heart muscle in a very targeted way to reduce the thickened wall and form scar tissue.

But not all patients are eligible for that less invasive option. For those that are not, the procedure must be done via open-heart surgery to physically remove muscle from the wall in the lower chambers of the heart. Either way, the procedures come with risks.

“They're both very mechanical, they're obviously not treating the underlying cause of the disease or function,” Sehnert said.

Patient may also require a pacemaker or defibrillator post-surgery due to scarring. About 2,500 to 3,000 of these procedures are performed in the U.S. each year, she added.

“Alternative treatment options … appear to be very much desired by the patients themselves and many of them do continue on the study, which was designed to have continuation beyond Week 60,” Sehnert said.

Previous data collected on mavacamten have shown the therapy works well with traditional beta blocker therapies, improved heart function and oxygen consumption. The VALOR trial also has an open label extension.

The VALOR results are the latest to pile on to the record for mavacamten, the heart disease drug BMS picked up from the $13 billion buyout of MyoKardia in 2020. BMS is awaiting an FDA decision on the therapy based on earlier results from the phase 3 EXPLORER program. The FDA is due to make its decision by April 28, a date that slid from January to the spring as the agency worked out a Risk Evaluation and Mitigation Strategy (REMS).

The REMS program was always in the plan given mavacamten’s mechanism of action, according to Marie-Laure Papi, VP and cardiovascular development program lead at BMS. She said the FDA needed a bit more time to work on the program, and BMS continues to work with the agency to fine-tune the details.

“The optimal outcome of the label and the REMS and the approval for mavacamten is to make sure that we continue seeing the excellent benefit-risk profile that we see in the trial, and we think that having a well-defined REMS program will help us achieve that,” said Papi.

Sehnert said the data package BMS has collected so far “has been very satisfying as a clinical researcher,” given its consistency across multiple findings.

https://www.fiercebiotech.com/clinical-data/bristol-myers-heart-drug-helps-patients-avoid-heart-procedures-countdown-fda-decision