It’s the season for reevaluation as companies weed out programs that don’t offer much promise. At the head of the line is a Sanofi’s SA’s once-potential multiple sclerosis and myasthenia gravis (MG) blockbuster, tolebrutinib. A partial clinical hold the phase III study is part of the reasoning for stopping its development. But so is competition, the company said. Astrazeneca plc already has Ultomiris (ravulizumab) and Soliris (eculizumab) approved for treating MG. Tolebrutinib came to Sanofi in 2020 with a hefty price tag, a $3.68 billion acquisition of its developer, Principia Biopharma Inc. Roche Holding AG also decided to drop two phase III programs, gantenerumab for Alzheimer’s disease and ipatasertib in prostate cancer. Other companies eliminating development programs include Gilead Sciences Inc., Merck & Co. and Eli Lilly and Co.
Biopharma financings hit $3.9B in January, down by 22% from last year
Starting out the year slow, biopharma financings are about 22% below where they were at the end of January 2022, and they are well behind the first month of the previous two years as well, suggesting company executives may need to continue to prioritize costly programs as resources are depleted – at least for the near future. So far in 2023, the industry has raised $4.26 billion through 94 financings, including $3.91 billion collected through 88 transactions in the month of January. The amount raised in January compares with $5 billion during the same month in 2022, $10.8 billion in 2021 and $6.95 billion in 2020. This year is slightly ahead of the $3.9 billion raised in January of 2019, but significantly down from 2018’s $7.78 billion. If financings continue the current pace, 2023 is on target to raise only about $47 billion this year, which is below seven of the last eight years.
Biohaven, Roche, Scholar Rock programs forge ahead in SMA
As research findings continue to spill out related to spinal muscular atrophy (SMA), a number of drug developers are pursuing new therapies for the indication. Names such as Biohaven Inc., Roche Holding AG and Scholar Rock Inc. are among those with programs in the works. Roche’s Evrysdi (risdiplam) was first approved by the U.S. FDA for SMA in August 2020.
Newco news: Haya targets lncRNA for tissue-specific fibrosis drugs
Could long non-coding RNAs be the key to developing organ-specific antifibrotic drugs that only mediate their effects in disease-related contexts? That’s the intriguing hypothesis that Haya Therapeutics SA has set out to explore, and its lead program, in heart failure caused by non-obstructive hypertrophic cardiomyopathy, is now in IND-enabling studies. A first clinical trial is pencilled in for late 2024 or early 2025.
Newco news: Turbine’s cell simulation platform to probe unanswered questions in oncology
Turbine Ltd. began the new year with a partnership with Cancer Research Horizons, the innovation arm of Cancer Research UK, which will put its Simulatedcell computational biology platform to work on the vexed question of how best to position CDC7 inhibitors in cancer. The company has developed an artificial intelligence platform that is intended to guide multiple steps during the drug development process, from target identification and validation through to drug discovery and drug positioning. It allows researchers to run simulated experiments, the results of which can inform the design of wet-lab experiments that may not otherwise have been conceived.
The BioWorld Insider Podcast: Rethinking obesity – Fitness may be more directly linked to health than weight
After many years of research and development, drug-induced weight loss has matured into a viable therapeutic option. But do the drugs directly help people become healthier or is it the weight loss? There are longstanding concerns that the health effects of weight might be overrated. And new studies show fitness improves health outside of weight. So, can a person still be considered healthy despite being over a “normal” weight? How will future medical science consider what is called the obesity epidemic? In this edition of the BioWorld Insider podcast, BioWorld Science Managing Editor Anette Breindl, discusses her new analysis of multiple studies related to weight loss, metabolic health and fitness which in many ways goes against the mainstream health care state of mind about obesity.
Also in the news
Agomab, Ambrx, Amylyx, Anaveon, Anavex, Arrowhead, Artelo, Astrazeneca, Aulos, Avalo, Biophytis, Cellectis, Clearside, Engene, Ensysce, Exscientia, First Wave, Inovio, Invivyd, Kiora, Magenta, Moonlake, Nrx, Oligomerix, Paxmedica, Pharmather, Pherin, Pliant, Provention, Roivant, Structure, Tenax, Treadwell, Vaxxinity, Vistagen, Y-Mabs