A twisted, uncertain path has led to a U.S. FDA approval for Biogen Inc.’s Qalsody (tofersen), the first drug targeting a genetic cause of amyotrophic lateral sclerosis (ALS). Qalsody is for ALS associated with a mutation in the superoxide dismutase 1 (SOD1) gene. Patients with SOD-1 mutations account for 2% of ALS cases. Read More
The long-anticipated top-line phase IIa study results for Morphic Therapeutic Inc.’s oral alpha 4 beta 7 integrin inhibitor, MORF-057, have surpassed even the company’s own expectations, with a significant decline in disease activity seen in moderate to severe ulcerative colitis (UC) patients combined with a safety profile consistent with phase I findings. Read More
As it requested, the U.S. FDA got an earful April 25 as people with long COVID and their caregivers shared their experiences with the condition that has no approved, or even authorized, treatment and is not that well understood, given its range of symptoms that vary from person to person. Read More
Dupuytren’s disease is often referred to as “the most common crippling hand condition that people have never heard of,” but Kurt Harrington is on a mission to change that. A seasoned biotech and pharma consultant, Harrington has had the condition himself for over a decade and, acutely aware of the lack of available treatment options, has founded Ventoux Biosciences Inc. to bring additional therapies forward. Read More
Swiss biotech firms raised CHF1.3 billion (US$1.5 billion) in equity and debt financing in 2022, a fall of 60% from the previous year’s total of over CHF3.3 billion. But it’s still ahead of historic pre-pandemic levels of funding. Read More
Xian Janssen Pharmaceutical Ltd. obtained marketing approval in China for Spravato (esketamine hydrochloride nasal spray) in combination with an oral antidepressant to treat depressive symptoms in adults with major depressive disorder with acute suicidal ideation or behavior. Unlike existing antidepressants on the market, which can take weeks to take effect, the newly approved nasal spray shows effects right away. Read More
Synonymous or silent mutations do not change the sequence of the protein that they encode. With some exceptions, they do not trigger any effect. Last year, however, a study by researchers from the University of Michigan tried to refute this concept after finding that they altered the protein function. But breaking dogmas can have answers. A group of scientists from various institutions has found that this work could have a method error. Read More
It used to be a lot easier to find partners. After two years of strong dealmaking, the volume and value of partnerships slid in the first quarter of 2023. M&As also are down compared to prior years. But there are some reasons to be cheerful. Looking at first quarter numbers and surveying the big picture on this BioWorld Insider podcast are Karen Carey, BioWorld’s managing editor and senior data analyst, along with Tim Shannon, a general partner at Canaan. Carey just wrote about biopharma deals and completed M&As in the first quarter. Shannon, who’s been with Canaan since 2009, has a new fund sporting $850 million in new capital, including an oversubscribed $650 million fund for seed and series A financings. They both offer their insights into the first quarter’s numbers and what may lie ahead. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Apellis, Applied, Astrazeneca, Belief, Citius, Daewoong, Faron, Foghorn, Ideaya, I-Mab, In8bio, Ionis, Krystal, Medigene, Neurona, Newamsterdam, Oncoinvent, Pharma Two B, Unity. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Abbvie, Alx, Arca, Assertio, Biomarin, Enanta, Guardant, I-Mab, Mina, Novo Nordisk, Nuvox, Sanofi, Spectrum, Stada, Tracon, Vyant, Wren, Xbrane. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Ani, Avidity, Bluebird, Bridge, Kancera, Lantheus, Maat, Moderna, Sanofi, Soligenix, Xbrane. Read More
