The U.S. FDA approved Corcept Therapeutics Inc.’s oral, selective glucocorticoid receptor antagonist, Lifyorli (relacorilant), nearly four months ahead of schedule for adults with platinum-resistant epithelial ovarian, fallopian tube or primary peritoneal cancer. A short time after the agency approved Lifyorli, it cleared Denali Therapeutics Inc.’s Avlayah (tividenofusp alfa) under the accelerated approval pathway for mucopolysaccharidosis II, also called Hunter syndrome, ahead of the April 5 PDUFA date.
Christmas came early for a number of biopharma companies this year as eight companies collectively raised $3.24 billion in public offerings. Both Structure Therapeutics Inc. and Terns Pharmaceuticals Inc. announced upsized offerings of $650 million each, and Kymera Therapeutics Inc. raised $602 million, placing all three in the top 10 follow-on offerings of the year.
One of the major obstacles to treating neurological disorders such as Alzheimer’s disease is the blood-brain barrier: drugs that are injected into the circulation usually do not enter the brain effectively. Researchers at Denali Therapeutics Inc., Biogen Inc. and the University of Minnesota have devised a vehicle for transporting antibodies against amyloid-β that can bypass the blood-brain barrier by binding to the transferrin receptor, which is expressed much more abundantly in capillaries than in arteries.
Denali Therapeutics Inc. has divulged NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of glaucoma, spinal cord Injury, multiple sclerosis, Niemann-Pick disease, stroke, Alzheimer’s disease, amyotrophic lateral sclerosis and diabetic neuropathy, among others.
Though down nearly 6% in January, the BioWorld Drug Developers Index (BDDI) rebounded in February, finishing the month with a 4.41% increase and outperforming both the Nasdaq Biotechnology Index (NBI; up 1.33%) and the Dow Jones Industrial Average (DJIA; up 3.47%). In 2023, BDDI concluded the year down 11.35%, trailing behind the NBI and DJIA.
Getting a drug successfully through clinical development in amyotrophic lateral sclerosis (ALS) continues to be an elusive task, with Denali Therapeutics Inc. and partner Sanofi SA reporting the latest failure in the progressive neurodegenerative disease.
Denali Therapeutics Inc. has divulged NLRP3 inflammasome inhibitors reported to be useful for the treatment of Alzheimer's disease, atherosclerosis, asthma, nonalcoholic fatty liver disease (NAFLD), multiple sclerosis (MS), experimental autoimmune encephalitis, type 1 diabetes and rheumatoid arthritis, among others.
Denali Therapeutics Inc. has patented new NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of glaucoma, spinal cord Injury, leukoencephalopathy, mitochondrial disease, multiple sclerosis, Niemann-Pick disease, spinal muscular atrophy and stroke, among others.
For the second time in a week, measures of serum neurofilament light chain (NfL) took center stage, this time as Denali Therapeutics Inc. unveiled interim data from the open-label, single-arm phase I/II study testing DNL-310 in children with mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.
Mucopolysaccharidosis type IIIA (MPS IIIA) is a genetic disorder where mutations in SGSH lead to the accumulation of heparan sulfate (HS) and lysosomal dysfunction that translate into developmental delay and cognition decline in humans. To date, there is no cure for MPS IIIA and that is why finding new strategies is an urgent need.
