Merck & Co. Inc. detailed positive data from the phase IIb/III trial known as MK-1654-004 with clesrovimab, an investigational prophylactic monoclonal antibody designed to protect infants from respiratory syncytial virus (RSV) disease during their first RSV season. The results, plus interim findings from the ongoing phase III experiment dubbed MK-1654-007 were offered by Rahway, N.J.-based Merck during IDWeek 2024 in Los Angeles.

By the numbers: US leads charge of cell and gene therapies

U.S. biotechs and regulators ushered in the era of gene therapy in 2023, experts at Bio Japan said, but medical reform is needed to pave the way for the “year of cell therapy” in 2024 and implement wider access to ultra-expensive cell and gene therapies (CGT). CGT falls into the branch of regenerative medicine, referring to the use of human or animal-based cells or tissue to “regenerate” aging or dying cells in the body. Gene therapy involves the use, edit or transfer of genetic material to treat genetic conditions, like hemophilia, as well as acquired diseases like leukemia. Cell therapy involves the transplant of cell-based material. CAR T-cell therapies are a well-known example, which leverage genetically altered immune (T-)cells to target and destroy cancer cells.

Verastem preps an NDA as the stock struggles

With fresh phase II ovarian cancer data in hand, Verastem Oncology Inc. said it will file an NDA by the end of the month for its avutometinib and defactinib combination treatment. The NDA will be for adults who have recurrent KRAS mutant recurrent low-grade serous ovarian cancer (LGSOC). LGSOC is a rare, slow-growing and chronic type of ovarian tumor that accounts for 10% of serous carcinomas. The FDA advised against pursuing the recurrent KRAS wild type indication. Verastem stock (NASDAQ:VSTM) suffered a 20% drop on Oct. 17, when the data and regulatory direction was released. At midday on Oct. 18, shares had settled somewhat at $2.59 each, a drop of 2.6%.

32 NMEs gain US FDA clearance through Q3, approvals up over 2023

The U.S. FDA approved 23 drugs in September, up from 22 in August and 17 in July, bringing the 2024 monthly average to nearly 20. This surpasses last year's 16 per month, 12.5 in 2022, and 17 per month in both 2021 and 2020.

Study paves way for therapy, easier diagnosis in endometriosis

According to World Health Organization data, endometriosis affects about 10% of reproductive-age females globally. That already makes endometriosis a wildly underresearched and underfunded disease in relation to its prevalence. Plus, Rama Kommagani thinks even 10% is an underestimation. “Diagnosis is very underreported, particularly in low- and middle-income countries,” Kommagani, who is an associate professor of pathology at Baylor College of Medicine, told BioWorld. There are several reasons for this. Endometriosis is complicated to diagnose. It has overlapping symptoms and is often confused with irritable bowel syndrome (IBS) or menstrual disorders. Symptoms can also vary between patients, possibly because the disorder comes in three distinct forms. Endometriosis characterized by ovarian, peritoneal and deep infiltrating lesions may all be separate diseases.

The BioWorld Insider podcast: A quantum leap into the future of drug development

In the newest BioWorld Insider podcast, Victoria Lipinska, the America's lead for Quantum Innovation Centers at IBM Quantum, talks about the future of drug development using quantum computing. “The new technology is a completely different branch of computing as opposed to what we know right now, and it's meant to complement what we know, not to really replace it,” she said. Quantum computing could lead to more efficient drug discovery by identifying promising compounds faster, understanding their effects at the molecular level, and then reducing the need for costly or time-consuming lab experiments. Lipinska is one of the more than 100 experts who will evaluate the future of health care at the upcoming 2024 Biofuture conference. Each year, a group of trailblazers, disruptors and forward-thinking executives converge to evaluate and forecast the future of health care. This year, BioWorld is a sponsor of the Oct. 28-30 event in New York. If you attend, you'll have the chance to hear panels and join workshops and fireside chats with key opinion leaders like Lipinska.

Also in the news

Abbvie, Acadia, Alzamend Neuro, Anavex, Athos, Avacta, Avadel, Biodexa, Capricor, Cerus, Concentra, CSL Vifor, Curanex, Curevo Vaccine, Eisai, Eterna, Factor, Glaukos, Innovent, Intercept, Kezar, Lexicon, Limmatech, Marinus, Neuvivo, Noema, Orano, Photys, Poseida, Sage, Sanofi, Terray, Theratechnologies, Travere, Valneva, Verastem, Vianautis, Viatris, Xortx