FDA accepts Bristol Myers' pitch for former MyoKardia drug; Clovis' Rubraca aces advanced ovarian cancer study
The program at the centerpiece of Bristol Myers Squibb’s buyout of MyoKardia just got one step closer to gaining potential FDA approval.
BMS announced Friday that the agency has accepted its NDA pitch, scheduling a Jan. 28, 2022 PDUFA date for the experimental drug mavacamten. The company is shooting for an initial indication of symptomatic obstructive hypertrophic cardiomyopathy, or oHCM.
In the disease, a mutation in proteins forces the heart to exert more effort and squeeze more, blocking the outflow of blood and thickening muscle. Mavacamten is an oral allosteric modulator of cardiac myosin, intended to reduce contraction by the heart muscle.
But BMS is betting heavily that the program could turn into a potential blockbuster after ponying up more than $13 billion in their acquisition of MyoKardia. The price per share, $225, represented a 61% premium.
Friday’s NDA acceptance was based on a Phase III study designed to capture both symptoms and cardiac function. Reading out data in May 2020, mavacamten beat placebo with a p-value of p=0.0005, in addition to delivering stellar numbers on a slate of secondary endpoints. And later that year, MyoKardia reported that the benefits extended to all subgroups.
The acquisition last October represented a move away from oncology for BMS, where the company has struggled to grow its Opdivo franchise in the wake of continued Keytruda success from Merck. — Max Gelman
Clovis’ Rubraca hits its marks in PhIII ovarian cancer study
Clovis Oncology’s PARP inhibitor Rubraca hit its primary endpoint for progression-free survival over chemo in BRCA-mutated, advanced ovarian cancer patients who have received two or more prior lines of chemo, according to top-line data presented Friday at the virtual Society of Gynecologic Oncology meeting.
Patients treated with Rubraca in the ARIEL4 study posted a median PFS of 7.4 months compared with 5.7 months in the chemo arm. The study enrolled 349 women in Europe, Israel and North and South America.
“The ARIEL4 data add to the growing scientific understanding about the clinical utilization of Rubraca compared to chemotherapy, including platinum-based chemotherapy, for women diagnosed with BRCA mutation-positive advanced ovarian cancer,” Clovis CEO Patrick Mahaffy said in a statement.
In terms of safety, Rubraca posted similar side effect rates as in previous studies, Clovis said, with anemia the most commonly reported Grade 3 and higher event. — Kyle Blankenship
Sanofi wants out of gene therapy deal with Oxford BioMedica
Gene therapy player Bioverativ inked a $100 million deal for access to Oxford BioMedica’s lentiviruses to treat hemophilia back in 2018 — just a month after the Biogen spinoff was snatched by Sanofi for $11.6 billion. Now, Sanofi says it wants out.
Sanofi has given notice that it intends to terminate the collaboration with Oxford BioMedica, according to a statement.
Oxford BioMedica got $5 million upfront, and was eligible for milestones up to $100 million, along with royalties on sales of the hemophilia treatments Bioverativ was developing.
In 2019, Sanofi watched the market for one of those drugs — the hemophilia treatment Eloctate — shrivel in the face of Roche’s star, Hemlibra.
“It is a non-cash event of course but it is clear that the competition from Hemlibra was a surprise to everyone and it is indeed putting pressure,” Sanofi chief financial officer Jean-Baptiste de Chatillon told reporters, according to a report in Reuters. — Nicole DeFeudis