Moderna Inc. gained U.S. FDA approval on May 31, nearly three weeks past its original PDUFA date, for its respiratory syncytial virus (RSV) vaccine Mresvia (mRNA-1345), which had both breakthrough therapy and fast track designations in the U.S. It is the third approved RSV vaccine, but the first that utilizes mRNA technology. It is also the first approval of an mRNA vaccine for something other than COVID-19. The approval is to protect those 60 and older from lower respiratory tract disease caused by RSV infection. For Cambridge, Mass.-based Moderna, Mresvia is its second approved product, following the January 2022 clearance for its COVID-19 mRNA vaccine, Spikevax (elasomeran).

Ultragenyx gene therapy hits phase III goal in rare disease GSD1

Ultragenyx Pharmaceutical Inc. anticipates a meeting later this year with the U.S. FDA to discuss a BLA filing for DTX-401 as the first potential gene therapy for glycogen storage disease type 1a (GSD1a) after the phase III study hit the primary and two key secondary endpoints. GSD1a, an inherited and life-threatening disease that patients currently manage only by consuming large daily doses of cornstarch, affects roughly 6,000 patients worldwide and is caused by a defective gene coding for the G6Pase-alpha enzyme, resulting in the inability to regulate blood sugar. In the Glucogene study, treatment with DTX-401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake vs. placebo at week 48, with Ultragenyx executives pointing to longer-duration data from an earlier open-label trial suggesting patients eventually might be able to wean off cornstarch altogether.

Akeso’s ivonescimab bests Keytruda in PD-L1-positive NSCLC trial

Although details are scant, Akeso Pharmaceuticals Inc. reported that its PD-1/VEGF bispecific antibody, ivonescimab (AK-112), demonstrated statistically significant superiority as a first-line therapy for PD-L1-positive non-small-cell lung cancer (NSCLC) compared to Merck and Co. Inc.’s Keytruda (pembrolizumab). In the phase III head-to-head monotherapy trial, ivonescimab reached the primary endpoint of progression-free survival (PFS) superiority and demonstrated clinically meaningful superiority compared to pembrolizumab. The PFS benefit was demonstrated across all clinical subgroups, including those with PD-L1 low expression, PD-L1 high expression, squamous and non-squamous histologies, as well as other high-risk patients.

ASCO roars to life in Chicago as CEOs talk innovation

The American Society of Clinical Oncology (ASCO) meeting opened this afternoon at the McCormick Place convention center in Chicago with after-lunchtime sessions on breast cancer, melanoma, sarcoma and advancements on adjuvant cancer vaccines. The first full day of the massive annual conference begins early Saturday morning. As ASCO revved up Friday, the CEOs of Merck & Co., Gilead Sciences Inc. and Eli Lilly and Co. spoke about the future of oncological innovation, the impact of the Inflation Reduction Act and why they feel it is important to alter the 340B Drug Pricing Program. The CEO talk was sponsored by the Pharmaceutical Research and Manufacturers Association. ASCO continues through Tuesday, June 4.

Newco news: Sixpeaks launches to advance its dual activin IIA/B antibody

Obesity specialist Sixpeaks Bio AG emerged from stealth with $110 million in funding and an option to be acquired by Astrazeneca plc within the next two years, subject to filing an IND for the lead product. Of the $110 million, $30 million is a series A round, with the balance to come from Astrazeneca, which will provide nondilutive finance of up to $80 million. The $30 million series A was led by Versant Ventures, which founded Sixpeaks in 2022 to work on weight management drugs targeting activin receptors, with the aim of avoiding the muscle loss that occurs with glucagon-like peptide-1 agonists.

US Department of Defense leads in number of grants and largest nonprofit deal

In 2024, the value of biopharma nonprofit deals is tracking slightly higher than the previous year, but still significantly lower than the value seen in prior years. Meanwhile, grants to biopharma firms have dropped by more than 50% compared to last year’s monthly average. The U.S. Department of Defense emerged as the leading grant issuer to biopharma firms, and also saw the highest-value nonprofit deal in the sector.

Dissecting post-traumatic stress disorder and depression

Scientists from the PsychENCODE Consortium have analyzed the brain transcriptome in a coordinated series of studies to map all the cell types, genes, epigenetic factors, and molecular pathways involved in different psychiatric disorders. After a first set of projects based on bulk analysis, the second phase of this project included 14 simultaneous publications that revealed the cellular atlas of post-traumatic stress disorder and major depressive disorder, among others.

BioWorld Insider Podcast: ADCs and radiotherapies at the forefront of this year’s ASCO conference

Chris Barden, a co-managing partner at MPM Bioimpact who manages the firm’s Bioimpact Equities and Oncology Impact funds, shares her insights into the American Society of Clinical Oncology (ASCO) conference in Chicago. She talks about the major trends in oncology, including development of antibody drug conjugates, which is currently the hottest area in cancer treatment. Another highlight she expects from ASCO will be development in radiotherapy treatments, particularly in prostate cancer. Bardon provides a guide for attendees to use in setting their schedules for the year’s biggest cancer conference and what to watch for in the future.

Also in the news

AB Science, Adaptimmune, Aim Immunotech, Anocca, Aptose, Bridgebio, Calidi, Candel, Celcuity, Cinrx, Crinetics, Cyclo, Day One, Elevar, Escient, Galapagos, Incyte, Inhibrx, Invivyd, Inxmed, NH Theraguix, OS Therapies, Purminds, Sanofi, Shinobi, Soterios, Tetra, Teva, Verve, Xeris, Ziphius Vaccines