Homerun success of Novo Nordisk A/S’ semaglutide, which recently became the U.S.’s biggest blockbuster drug, is serving as an “inflection point” for obesity therapeutics and fueling the drive for new and improved therapies, speakers said at Bio Korea 2024 on May 8. Peter Neubeck of Kurma Partners SA highlighted that the obesity market is highly “unusual,” primarily because people are willing to pay out of pocket for therapies just to lose weight. But until the success of semaglutide, repeated failure in the obesity therapeutics space had driven investors away. The meteoric rise of semaglutide, which surpassed Abbvie Inc.’s Humira (adalimumab) and all its biosimilars in U.S. prescription drug expenditure in 2023, has swung the pendulum in the other direction, effectively making obesity an “overhyped” area where “everyone wants to get in.”

Pfizer reports boy’s death in phase I DMD gene therapy study

A boy participating in the phase II Daylight study of Duchenne muscular dystrophy (DMD) “has passed away suddenly,” according to Pfizer Inc. The participant received fordadistrogene movaparvovec, a mini-dystrophin gene therapy, in early 2023. The fatal serious adverse event was reported May 3 as a cardiac arrest, Pfizer told BioWorld. Participants in the Daylight study are boys ages 2- and 3-years-old. Pfizer said it does not yet have the complete information it needs to understand the incident. The information was posted in a letter dated May 7 from Pfizer on the Parent Project Muscular Dystrophy website. In December 2021, also in a letter to the DMD community, Pfizer reported a death in the non-ambulatory cohort of a phase Ib study of the gene therapy in DMD. At the time, the FDA placed the IND application on clinical hold.

US FDA adcoms turn the corner on pandemic

Right on cue, the U.S. FDA’s Center for Drug Evaluation and Research (CDER) is scheduling its first in-person advisory committee meetings since the COVID-19 pandemic. The day after CDER Director Patrizia Cavazzoni said the center was preparing to go back to in-person adcoms, CDER announced that two in-person adcoms have been scheduled for June. The Psychopharmacologic Drugs Advisory Committee will convene in person June 4 to discuss Lykos Therapeutics Inc.’s NDA for midomafetamine capsules as a treatment of post-traumatic stress disorder. Then on June 10, the Peripheral and Central Nervous System Drugs Advisory Committee will meet to discuss Eli Lilly and Co.’s Alzheimer’s candidate, donanemab.

Flat sales of Vowst sink Seres stock; launch changes underway

Shares of Seres Therapeutics Inc. (NASDAQ:MCRB) were trading at 77 cents, down 34 cents, or 30%, after the Cambridge, Mass.-based firm provided an update on the launch of Vowst, the oral microbiome therapy formerly known as SER-109. Vowst was approved by the U.S. FDA about a year ago for prevention of recurrent Clostridium difficile infection. Sales were roughly flat quarter over quarter, Seres said. In July 2021, Seres signed an agreement with Nestlé Health Science SA, of Lutry, Switzerland, to jointly commercialize Vowst in the U.S. and potentially Canada. During a conference call, Seres updated investors on changes made by Nestle.

India launches Immunoact’s homegrown NexCAR19 for cancer

India’s first indigenous CAR T therapy is expected to cost around $50,000, nearly one-tenth of the price of top-selling CAR Ts in the U.S. India President Droupadi Murmu officially launched Immunoadoptive Cell Therapy’s (Immunoact) NexCAR19 (actalycabtagene autoleucel), a CD19-targeted CAR T, and dedicated it to the nation in April 2024. Cleared by India’s Central Drugs Standard Organization in October 2023, Immunoact’s NexCAR19 is indicated for treatment of relapsed/refractory B-cell lymphomas and leukemia. 

BioWorld Insider Podcast – Back to fundamentals: The latest numbers point to better times

BioWorld Managing Editor Karen Carey joins the podcast to talk about the numbers from the first quarter of 2024, along with a look back at some 2023 deals and indicators that signal better times are on the way. Financings for the quarter were better than expected, sporting some of the best numbers of the past 13 years. It’s part of a larger trend, Carey says, of investors being a lot pickier about where they put their money and demanding better data. The result is a strengthened market and a better outlook.

BioWorld reader feedback needed: Cyberattack impact survey

The recent cyberattack on Change Healthcare, a part of the Unitedhealth Group, crippled reimbursement claims processing for thousands of providers for several weeks and potentially exposed troves of patient data. Congressional committees are investigating the attack, its scope, and Unitedhealth’s response. Are there downstream impacts that are being overlooked in the biopharmaceutical and/or medical technology sectors? Please take a few minutes to fill out this short survey to help BioWorld understand how your company is responding to these threats. You can click through to the questions here.

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