Mavacamten

Braveheart Bio and Hengrui Pharma on Monday reported new study data suggesting their partnered cardiac myosin inhibitor BHB-1893 (HRS-1893) may have potential beyond simply easing symptoms in hypertrophic cardiomyopathy (HCM).At the Heart Failure 2026 congress, the companies presented results from a Phase II study of 84 patients with symptomatic non-obstructive HCM, a form of the disease that affects about half of the HCM population. Patients treated with BHB-1893 improved across a number of measures, including symptoms, exercise capacity, cardiac biomarkers and echocardiographic measures.Specifically, in the high-dose arm, patients achieved a placebo-adjusted 5.5-point gain on the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS), while 52% saw at least a 20-point improvement, compared to 21% for placebo. Exercise capacity improved as well, with patients titrated to 60 mg twice daily posting a placebo-adjusted gain of 0.9 mL/kg/min in peak oxygen consumption (pVO2).Structural reverse remodellingThere were also signals of improved heart relaxation and structure among higher-dose recipients. The left atrial volume index (LAVI) fell by 7.1 mL/m² from baseline, versus essentially no change on placebo, while left ventricular mass (LVMI) decreased by 17.4 g/m² in the treatment arm compared to an increase of 3.2 g/m² for placebo. Heart wall thickness declined by 3.3 mm, versus 0.1 mm for placebo.Non-obstructive HCM lacks the left ventricular outflow tract (LVOT) gradient seen in obstructive disease; that's where a pressure difference is created when blood is trying to leave the heart by squeezing through a narrowed "obstructed" exit. In obstructive HCM, drugs can improve symptoms by relieving the blockage that is causing the gradient, making it harder to tell whether they are actually changing disease biology.However, in non-obstructive HCM, that specific confounder doesn't exist because there is no LVOT gradient to relieve."The structural reverse remodelling toward normal observed here is a compelling early signal that BHB-1893 may be modifying the underlying disease in non-obstructive HCM, in addition to helping manage the condition," said Anjali Owens, a member of Braveheart's clinical advisory board.Meanwhile, CEO Travis Murdoch said the placebo-controlled design boosts his confidence in the findings. "The fact that we didn't see movement in the placebo arm, but we saw…a dose-dependent movement, which in many cases was statistically significant, really gave us encouragement that this could continue to play out in a meaningful way in a larger pivotal study," he told FirstWord.Murdoch also pointed to the biomarker response that appeared "more rapid" and "deeper" than what has been seen previously. NT-proBNP, a marker associated with diastolic wall stress, fell roughly 68% to 69%, while cardiac troponin I, a marker of myocardial injury, declined 55% to 60%, both statistically significant against placebo.Braveheart and Hengrui said BHB-1893 was generally well tolerated, with no severe treatment-related adverse events and no discontinuations tied to the drug. As cardiac myosin inhibitors work by intentionally reducing the force of heart contraction, researchers also looked at the incidence of left ventricular ejection fraction (LVEF) <50%. Four treated patients (7%) experienced temporary declines in LVEF requiring dose reductions, though all recovered to above 50% after adjustment.Cytokinetics' recent successLast week, Cytokinetics reported that its drug Myqorzo (aficamten) became the first cardiac myosin inhibitor to succeed in a Phase III non-obstructive HCM trial, hitting dual endpoints in the ACACIA-HCM study (see – Spotlight On: Cytokinetics could double Myqorzo's reach with non-obstructive HCM win).That result contrasted with the setback suffered by Bristol Myers Squibb last year when Camzyos (mavacamten) failed to meet the main goals of the Phase III ODYSSEY-HCM study in the same indication.Both Myqorzo and Camzyos are already approved for obstructive HCM, with Camzyos generating growing sales since its 2022 launch, including hitting blockbuster status last year with revenues of around $1.1 billion, while Myqorzo won approvals in China and the US in late 2025 followed by Europe earlier this year.Pivotal study plansBraveheart is hoping BHB-1893 can be a differentiated entrant in the space. The drug was originally discovered by Hengrui, which out-licensed ex-China rights to Braveheart last year in a deal worth up to $1.1 billion. This past March, Braveheart and Hengrui unveiled Phase II data for BHB-1893 in obstructive HCM showing complete LVOT gradient response ranging from 50% to 86% with minimal changes in LVEF.Hegnrui is running a Phase III study of the drug in obstructive HCM in China that should readout towards the end of the year, according to ClinicalTrials.gov. Braveheart is in the middle of ramping up for pivotal studies as well, initially in the obstructive form of the disease slated for this year. It is also planning to have discussions with regulators about initiating a non-obstructive study, which they hope to begin this year as well.

Welcome back to another edition of Endpoints Weekly! Earnings season is in full swing, but we had plenty of original reporting and exclusives you won’t find anywhere else this week. Chief among them: Andrew Dunn’s excellent story Friday on why biopharma is struggling to recruit young AI talent . Spoiler — it’s not just the pay gap with companies like OpenAI, though that’s definitely part of it. We also hosted a Post-Hoc Live recapping what we’ve seen in earnings so far, where I joined Drew Armstrong, Elizabeth Cairns and Leerink senior research analyst David Risinger to dig into the biggest stories in pharma right now. Paid subscribers can click here to watch a recording of the conversation. And, on Friday afternoon, the Wall Street Journal reported that President Donald Trump is planning to fire FDA Commissioner Marty Makary — make sure to read about that here . Have a great weekend! — Max Gelman 🇨🇳 A new trend is shaking up how biotechs are built, Kyle LaHucik observes in this feature story . Instead of launching with eye-popping early-stage science or a new platform, there’s a surge of startups with small teams, large checks and one or two clinical-stage assets in-licensed from China. The so-called NewCo model has been around for years, but its use with assets connected to China appears to be increasing. Endpoints News identified at least 18 private biotechs that have followed this route since the beginning of 2025. Previously, most biotechs started with a piece of fundamental science, then worked for years to build a drug around it that could eventually be tested. But with the explosion of trial-ready assets from China, many are skipping those early steps. Some companies are focusing on a single drug, hoping to expand into multiple indications with the same asset. Others have sourced a clinical-stage asset or two from China to kickstart their pipelines and build a foundation for additional discovery work. The model represents a fundamental shift in how biotechs are formed and grow, Kyle writes . The dynamics can be traced back to a handful of factors, including a yearslong funding downturn, an explosion in target and modality crowding, a desire to get quicker returns, a flocking to diseases with large patient populations, and a massive growth spurt in competition from R&D hubs like Shanghai. You can read more here. 💰 Candid’s effort to prove T cell engagers’ potential in autoimmune diseases is getting picked up by one of Europe’s oldest pharma companies, Kyle LaHucik writes . UCB is paying $2 billion in cash to acquire Candid, with up to $200 million in milestones on the line. Candid had been planning to go public this summer via a reverse merger with Rallybio. But unsolicited interest from multiple companies “led toward a broader evaluation,” CEO Ken Song said. “I was a bit surprised,” Song told Endpoints . But he noted that there has been “an increasing appreciation broadly across the industry on the benefit of T cell engagers for B cell depletion in a variety of autoimmune diseases.” Song has been committed to testing the modality for autoimmune diseases, previously claiming that Candid would “prove or disprove” the approach “for the entire industry.” The deal marks UCB’s second biotech acquisition in as many months, in what has so far been a busy year for M&A. Drugmakers have signed at least 30 acquisitions so far this year, according to an Endpoints tally. 💩 Scientists trying to understand why cancer immunotherapy works startlingly well for some patients — and not others — have found a potential answer in one cancer survivor’s poop . Kanvas Biosciences studied a woman’s stool and identified roughly 50 strains of bacteria that, collectively, created just the right conditions for immunotherapy to cure her advanced colorectal cancer. Researchers also say they’ve figured out how to grow that microbial community in the lab and package them in pills, senior science correspondent Ryan Cross reports . Kanvas raised $48 million in a Series A to run a clinical trial later this year testing those pills. The money will also fund two other microbe-based therapies: one to help reduce colon inflammation caused by immunotherapy, and another, backed by the Gates Foundation, aimed at healing the guts of patients who can’t properly absorb nutrients. Read more from Ryan here . 🤝 GSK inked a $1 billion biobucks deal with Suzhou-based SiranBio, expanding its reach in the cardiometabolic space. GSK will pay $55 million upfront for the rights to SA030 — an siRNA in Phase 1 for obesity — outside of China, Hong Kong, Macau and Taiwan. SiranBio has been building up the drug candidate as a next-generation obesity treatment, with the potential to “achieve selective fat reduction without compromising muscle mass.” It goes after activin receptor-like kinase 7, or ALK7, which is the same target as one of Arrowhead Pharmaceuticals’ obesity drugs. But GSK sees potential beyond obesity. A GSK spokesperson told Endpoints News that the company doesn’t plan to develop the program “for the sole purpose of weight loss.” Rather, GSK said it will test the candidate in cardiometabolic disease patients with “chronic conditions that affect the lung, liver and kidney.” The deal also extends GSK’s dealmaking streak in China. Over the last couple of years, it has done deals with Hengrui, Hansoh and Chimagen. Read more here from Kyle LaHucik. 🫀 This week, Cytokinetics said its heart drug Myqorzo succeeded in a Phase 3 study, showing improvements in symptoms and exercise capacity in patients with the non-obstructive form of hypertrophic cardiomyopathy (nHCM). The drug already won approval for the obstructive form of the condition (oHCM) late last year. Myqorzo competes with Bristol Myers Squibb’s Camzyos in oHCM. And Cytokinetics’ new data would potentially open up a whole new market, in an indication where Camzyos notably failed in its pivotal trial last year. Both drugs work by inhibiting the heart muscle protein cardiac myosin. Read more about the pivotal win here . DON’T MISS: