Mavacamten

Today, a brief rundown of news from Sanofi and Novo Nordisk, as well as updates from The Lasker Foundation, Edgewise Therapeutics and Vanda Pharmaceuticals that you may have missed.Novo Nordisk shares fell 6% in Friday trading today after reporting mixed study resultsfrom a Phase 2 trial of a weight-loss drug it acquired in a deal for startup Inversago Pharma last year. According to Novo, people who received the lowest dose of the drug, a new type of medicine blocking the protein receptor CB1, lost an average of just over 7 kilograms of weight after 16 weeks. Higher doses resulted in limited additional weight loss, while drug recipients reported gastrointestinal as well as neuropsychiatric side effects. Novo is planning a larger and longer mid-stage study of the drug, monlunabant, to find the optimal dosing to balance safety and efficacy, said Martin Holst Lange, its executive vice president and head of development, in a statement. Jonathan GardnerSanofi on Friday presented study results the company has claimed could support approval of its experimental drug tolebrutinib in a form of multiple sclerosis. The data showed tolebrutinib helped delay the time to onset of confirmed disability progression by 31% compared to a placebo in people with non-relapsing secondary progressive MS, while also inducing a spike in liver enzymes that Sanofi said can be managed by monitoring patients. Overall, the findings should enable global filings and approvals given [the] current unmet need in that form of MS, wrote Jefferies analyst Peter Welford. Ben FidlerThree scientists whose research led to a new group of widely popular weight-loss drugs have won a Lasker Award, an annual prize given for discoveries that advance medicine and public health. Joel Habener of Massachusetts General Hospital, Svetlana Mosjov of The Rockefeller University and Lotte Bjerre Knudsen of Novo Nordisk were each handed awards for their roles researching and developing the GLP-1 medicines now used to treat diabetes and obesity. The three have introduced a new era of weight management in which GLP-1 based pharmaceuticals promise to dramatically enhance health, the Lasker Foundation said in a Thursday statement. Ben FidlerShares of Edgewise Therapeutics surged nearly 50% after the company on Thursday revealed early study results for a drug its developing for a form of heart failure. Though only from a small number of healthy volunteers in a Phase 1 trial and people with hypertrophic obstructive cardiomyopathy in a mid-stage study, the data clearly differentiates Edgewises drug from Bristol Myers Squibbs Camzyos and Cytokinetics aficamten, wrote Leerink Partners analyst Joseph Schwartz. Edgewise dosed the first participants in the next part of its Phase 2 study and expects to report findings next year.Ben FidlerThe Food and Drug Administration on Thursday declined to approvea Vanda Pharmaceuticalsdrug called tradipitantfor symptoms of gastroparesis. Vanda claimed the agency generally disregarded the evidence from two placebo-controlled trials and requested additional studies with a design and duration that are “inconsistent with the advice of key experts“ in the field. The company still plans to pursue an approval in gastroparesis, and intends to file a separate application for use preventing vomiting and motion sickness. Delilah AlvaradoThe House Energy and Commerce Committee voted unanimously Wednesday to support legislation extending the pediatric rare disease voucher program for another six years, to 2030. The program, which was due to expire on Sept. 30, grants companies that win approvals of medicines for rare pediatric diseases a voucher that speeds up drug reviews and can be sold for cash. As of the end of April,the FDA had awarded 53 vouchers since the programs inception in 2012, according to the National Organization for Rare Disorders. Jonathan Gardner '

Edgewise Therapeutics has unveiled early safety data for its rare heart disease drug, with results suggesting it could do away with a black box warning seen with Bristol Myers Squibb’s Camzyos in obstructive hypertrophic cardiomyopathy (HCM). Edgewise’s candidate, dubbed EDG-7500, also shows signs of having a relatively better side effect profile than Cytokinetics’ aficamten in HCM. On Thursday morning, Edgewise said that in two trials that separately tested the drug in HCM patients and healthy volunteers, none of the participants saw a drop in left ventricular ejection fraction (LVEF) below 50% across a “broad range” of doses. LVEF refers to how much blood is being pumped out of the heart’s left ventricle. Ejection fraction below 40% means that the heart isn’t pumping enough blood and can be a sign of heart failure. Ahead of Edgewise’s data release, Leerink analysts wrote Wednesday that if the results suggest a better risk-benefit profile than Camzyos, Edgewise could win approval without a risk evaluation and mitigation strategy (REMS) and with a more simplified dosing regimen, which would enable uptake among a greater number of HCM patients. Investors had an unintentional preview of Thursday’s results. An abstract for the Heart Failure Society of America’s annual meeting showcasing EDG-7500 was briefly published early on Tuesday on the conference website and distributed on social media. Nonetheless, the company’s share price $EWTX was up 45% on Thursday. But Edgewise is behind in the HCM race. Camzyos was approved in 2022 and it collected $231 million in sales last year. Cytokinetics is set to put forward its candidate to the FDA soon with an eye to launching its drug next year. Edgewise’s safety data are from a Phase 1 trial with 72 healthy volunteers and part one of a Phase 2 trial in HCM, which enrolled 11 patients with obstructive HCM. Camzyos carries a black box warning that cautions a drop in LVEF. In the Phase 3 EXPLORER-HCM trial, nine Camzyos patients had to pause dosing because of the side effect, although eight of them later resumed treatment at a lower dose after recovery. Leerink analysts said Thursday that Edgewise’s data also appear to be differentiated from aficamten. There were instances of LVEF drops below 50% in early studies of Cytokinetics’ drug, while new data show Edgewise’s candidate doesn’t, they added. Patients dosed with aficamten in a registrational trial had an average 4.8% drop in LVEF from baseline compared with placebo but with no associated dose interruptions. Edgewise CEO Kevin Koch told Endpoints News in an email that cardiac myosin inhibitors such as Camzyos and aficamten may be on-off switches for myosin, while EDG-7500, which is a cardiac sarcomere modulator, is more about controlling the rate of interaction. With the company’s more controlled approach, there may be fewer side effects, he added. As for next steps, Edgewise has started the next part of its Phase 2 trial, which is testing EDG-7500 in obstructive and non-obstructive HCM patients with data expected early next year. In May, Cytokinetics CEO Robert Blum told Endpoints that aficamten could be suitable for a “broader array” of obstructive HCM patients compared with Camzyos. Edgewise said in the presentation it is “well-capitalized to execute important milestones,” with $512 million in cash, equivalents and marketable securities as of the end of June. This should support the company’s activities through 2027.