Amgen and Novo Nordisk Advance Obesity Drug Development, While GenAssist Pioneers Gene Editing for Duchenne

In a flurry of recent developments, pharmaceutical giants and biotech startups are making significant strides in obesity treatment and rare disease therapies. Amgen is gearing up for pivotal trials of its novel obesity drug, while Novo Nordisk introduces a discounted program for its popular weight-loss medication. Meanwhile, GenAssist receives clearance for groundbreaking gene editing trials in Duchenne muscular dystrophy.

Amgen Prepares for Phase 3 Trials of MariTide

Amgen is set to launch two Phase 3 trials for its experimental obesity drug, maridebart cafraglutide, also known as MariTide. The trials, registered on a federal database, aim to enroll approximately 4,500 participants who are overweight or obese. One of the studies will specifically focus on individuals with Type 2 diabetes.

The trials will evaluate three different dose levels of MariTide against a placebo, measuring weight-loss effects after 72 weeks of treatment. Industry observers are closely watching MariTide's development due to its potential for monthly administration, which could provide a significant advantage over weekly treatments like Novo Nordisk's Wegovy and Eli Lilly's Zepbound.

Novo Nordisk Launches Discounted Wegovy Program

In response to the growing demand for obesity treatments, Novo Nordisk has established an online pharmacy called NovoCare Pharmacy. This initiative offers Wegovy at a discounted price of $499 per month for cash-paying patients who have been prescribed the drug but lack insurance coverage or obesity drug benefits in their health plans.

The move follows a similar program by Eli Lilly for Zepbound, which offers monthly prices ranging from $349 to $499 depending on the dose. Novo Nordisk positions its program as an alternative to "fake or illegitimate compounded" versions of Wegovy, addressing concerns about counterfeit medications in the rapidly expanding obesity drug market.

GenAssist Pioneers Base Editing for Duchenne Muscular Dystrophy

GenAssist, a Suzhou-based biotechnology company, has received FDA clearance to begin U.S. testing of Gen6050x, a first-of-its-kind gene editing treatment for Duchenne muscular dystrophy. The therapy employs base editing technology to potentially restore dystrophin protein expression permanently.

Specifically designed for Duchenne patients amenable to exon 50 skipping, Gen6050x has already been administered to two patients in a Chinese study. This advancement marks a significant milestone in the application of gene editing techniques for treating genetic disorders.

Industry Leadership Changes and Clinical Trial Updates

In other industry news, Mikael Dolsten, former top scientist and R&D head at Pfizer, has been appointed to lead the board of directors at Orbis Medicines. The biotech startup, which recently raised nearly $100 million in Series A funding, is developing oral peptide drugs that aim to combine the strengths of biologics and small molecules.

Additionally, BioNTech has reported a hold on its Phase 1/2 trial of an RNA-based malaria vaccine by the FDA. The company is working to address the agency's requests and evaluate next steps for the study, which had enrolled approximately 180 malaria-naive adults to assess the shot's immunogenicity and safety.