Some of the biggest news and trends captured in PharmaVoice’s newsletter this week. Sign up here to receive the newsletter daily.
Moderna’s cancer vaccine clears phase 3 test
Moderna and Merck’s personalized mRNA cancer vaccine has passed its biggest test yet. The partners said Wednesday that intismeran autogene, paired with Keytruda, met both primary endpoints in a phase 3 melanoma study — the first positive phase 3 result for an mRNA cancer vaccine.
INTerpath-001 enrolled 1,137 people with surgically removed, high-risk stage IIB through IV melanoma. The combination improved recurrence-free survival and distant metastasis-free survival compared with Keytruda alone, and no new safety signals emerged, the companies said. The magnitude of the benefit and overall survival data remain undisclosed.
The readout gives Moderna another potential path beyond its COVID-19 vaccine franchise. Earlier this week, PharmaVoice examined the commercial and regulatory hurdles facing mFlusiva, the company’s newly approved flu shot and the first mRNA flu vaccine cleared in the U.S.
Ultragenyx lands its first gene therapy approval
Ultragenyx notched its first gene therapy approval Wednesday when the FDA granted accelerated approval to Genglycos, the first approved treatment for glycogen storage disease type Ia, also known as Von Gierke disease.
The one-time AAV8 gene therapy is cleared for patients 8 years and older to reduce daily cornstarch intake alongside nutritional management. GSDIa prevents the body from properly releasing stored glucose, leaving patients vulnerable to dangerous drops in blood sugar and reliant on frequent meals and around-the-clock cornstarch.
The FDA based its decision on reduced cornstarch use, a surrogate endpoint, and Ultragenyx must still confirm the therapy’s clinical benefit. That evidence question also surfaced in PharmaVoice’s look this week at the scientific papers industry leaders keep sharing, including research on surrogate endpoints and clinical trial design.
Novo tests how low oral Wegovy can go
Novo Nordisk has begun a 60-week phase 3 study to find the lowest effective maintenance dose of its oral Wegovy formulation, Reuters reported. OASIS-5 will enroll 450 adults and test two undisclosed lower doses of oral semaglutide against placebo. The currently approved long-term dose is 25 mg, and the study is expected to run through 2028.
The move could give Novo more flexibility as it battles Eli Lilly in an increasingly segmented obesity market, where cost, tolerability and patient preferences matter alongside efficacy. PharmaVoice reported this week that affordability and prescriber perceptions could help determine how the rivals divide the market.
