Mavacamten

Cytokinetics said late Thursday that the PDUFA date for its heart disease therapy aficamten would be delayed by three months to Dec. 26. The lag would allow the FDA to review a major change to its marketing application, namely the submission of a drug safety program or REMS. The major surprise here is that the company did not include a REMS in its original NDA filing. Stifel analysts described the omission as “not at all the Street’s expectation.” Previously, Cytokinetics’ management had said it would propose a REMS package when it submitted the NDA, they wrote on a Thursday note. Cytokinetics’ stock $CYTK was down by as much as 12% premarket Friday as investors digested the arguably unnecessary delay. The company appeared to suggest that the FDA was OK with the initial lack of a REMS, saying that it previously had pre-NDA discussions with the agency that led to the REMS-free filing, which the FDA accepted. The agency subsequently demanded a safety program “based on the inherent characteristics of aficamten,” though no extra clinical trials are needed. The PDUFA delay comes as the biopharma sector is keeping a close eye on the FDA, concerned that cuts could impact approval timelines . Aficamten is intended to treat the rare heart muscle condition obstructive hypertrophic cardiomyopathy, and in its pivotal trial, the cardiac myosin inhibitor beat placebo at improving oxygen uptake as patients exercised. Importantly, on a cross-trial basis, aficamten looked better than the existing medication for the disease, Bristol Myers Squibb’s Camzyos. Bristol Myers included a REMS program in its original NDA filing for Camzyos. That drug too had its approval date pushed back, from January to April 2022, to allow the FDA to assess updates to the proposed safety measures. In mid-April, the label of BMS’ drug was updated to lessen the REMS requirements, cutting the frequency of heart echo monitoring from every three to every six months, and lifting a ban on patients taking certain other drugs. Cytokinetics believes that aficamten will end up with differentiated REMS requirements to Camzyos, with even fewer restrictions.

BMS chief Chris Boerner figured it's necessary to put its recent clinical setbacks with Cobenfy and Camzyos "in proper context." On the heels of a clinical setback for Bristol Myers Squibb’s schizophrenia newcomer Cobenfy, the New Jersey pharma is serving up mixed results for the first three months of the year.Still, even as generics eat into sales of the company’s legacy medicines, BMS’ roster of recent launches continues to impress, and the drugmaker remains confident that Cobenfy can come out ahead of its recent defeat.While the Cobenfy stumble and another recent setback for Bristol’s cardiomyopathy drug Camzyos were “not the results we had hoped for,” there is a need to “put the results in proper context,” BMS chief Chris Boerner, Ph.D., said on a call with analysts Thursday.Although Cobenfy—which was approved as a monotherapy in September—failed to chart superior improvement over placebo in a phase 3 study testing the drug as an adjunctive treatment to atypicals in patients with inadequately controlled schizophrenia, the data remain “encouraging,” Boerner argued.In particular, the results from the late-stage Arise trial demonstrate a “noteworthy improvement” for most schizophrenia patients and reinforce the drug’s tolerable safety profile, the CEO explained.The study results—released earlier this week—did reflect a numerical improvement for the BMS drug, which was the centerpiece in the company's $14 billion acquisition of Karuna Therapeutics.“It’s important to remember there are no currently approved adjunctive therapies for schizophrenia,” Boerner added on the company’s call. “Our commercial strategy remains focused on monotherapy, which accounts for 70% to 80% of the market,” he said, noting that BMS aims to make Cobenfy “the foundational treatment” in the disease.BMS will continue to push Cobenfy toward new indications, too, and it's currently awaiting phase 3 data in Alzheimer’s disease psychosis.The company also plans to kick off a suite of “difficult studies” this year, including seven phase 3 trials for Cobenfy across three indications: Alzheimer’s agitation, Alzheimer’s cognition impairment and bipolar I.Those studies are expected to be underway by the middle of 2025, Boerner noted. On the commercial front, monotherapy remains “the most significant commercial opportunity” for Cobenfy, Bristol’s chief commercialization officer, Adam Lenkowsky, said on the call.BMS’ strategy for the drug—which reeled in $27 million in 2025 first quarter sales—involves progressively shifting Cobenfy into earlier lines of schizophrenia treatment, which is already playing out in prescription patterns, Lenkowsky pointed out.“Roughly 40% to 50% of the Cobenfy [being prescribed] today is now in second line, third line,” Lenkowsky said. “And so, physicians have also told us, both in research and through advisory boards, that missing the endpoint of the study would have no impact on model therapy usage or their willingness to use Cobenfy, particularly when you look at the safety of the study results.”So far, Cobenfy is enjoying a “nice start,” analysts at William Blair wrote in a note to clients Thursday. Analysts at Edward Jones concurred, noting that sales of the schizophrenia med “appear to be off to a good start, which is good to see” considering the recent setback in the clinic. The BMS execs gave their Cobenfy status report as the company posted $11.2 billion in first-quarter revenues for 2025, down 6% year over year. Breaking things down further, Bristol’s U.S. revenues fell 7%, while international sales dropped 2% during the quarter.Most of that decline can be attributed to generic competition facing legacy BMS drugs like Revlimid, Pomalyst, Sprycel and Abraxane, the company said in an earnings release. At the same time, however, BMS’ cadre of newer therapies—which includes Opdivo, Breyanzi, Reblozyl, Camzyos and now, Cobenfy—grew sales 16% to $5.6 billion for the quarter.Despite the revenue downturn, BMS is raising its guidance from roughly $45.5 billion in projected 2025 sales to a range of $45.8 billion to $46.8 billion. Bristol attributed the forecast upgrade to strong performance from its up-and-comer meds, plus “better-than-expected” sales from its legacy portfolio in Q1. On that surprise showing from BMS’ legacy medicines, the analyst team at William Blair noted that Revlimid’s first-quarter sales haul of $936 million beat their expectations, signaling that generic erosion for the drug has proven “lumpy and variable.”Meanwhile, BMS factored in the impact of retaliatory tariffs from China in its updated forecast, though the new guidance doesn’t account for any potential pharmaceutical tariffs, the William Blair team noted.