For the U.S. FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, the medical need and the effectiveness of Eli Lilly and Co.’s Alzheimer’s candidate, donanemab, outweighs the safety concerns and lack of data for underrepresented groups and special needs patients. The panel voted unanimously, 11-0, June 10 that the available data show donanemab is effective in treating Alzheimer’s in the population enrolled in Lilly’s clinical trials and that the benefits of the amyloid-targeting monoclonal antibody outweigh the risks in the study population of patients with mild cognitive impairment and mild dementia.
Phase II-stage Rapport Therapeutics Inc. began trading on Nasdaq June 7 under the ticker RAPP after pricing its IPO of 8 million shares at $17 each to raise $136 million, gaining $3.80, or 22.4%, to close its first day at $20.80. With offices in Boston and San Diego, Rapport is developing drugs for central nervous system (CNS) disorders. The IPO is expected to close June 10.
Washington-based Vanda Pharmaceuticals Inc. has faced a challenging few years involving a federal lawsuit against the U.S. FDA, patent infringement litigation, increasing generic competition and dwindling sales, as well as a complete response letter nixing plans to expand its melatonin receptor agonist Hetlioz (tasimelteon) into insomnia, yet it has recently received a higher, unsolicited acquisition offer of $466 million from a second company, Cycle Pharmaceuticals Ltd.
Although the U.S. FDA unexpectedly sprang the news on Eli Lilly and Co. that it would hold an advisory committee meeting on the BLA for the company’s Alzheimer’s disease drug, donanemab, the agency’s briefing document for the June 10 meeting doesn’t appear to hold any surprises.
The discussion that preceded the June 4 U.S. FDA advisory committee vote against the approval of Lykos Therapeutics Inc.’s midomafetamine as a treatment for post-traumatic stress disorder could shine some light on the way forward for other sponsors developing psychedelics for approved medical use.
Although several members of the U.S. FDA’s Psychopharmacologic Drugs Advisory Committee said they thought Lykos Therapeutics Inc.’s midomafetamine (MDMA), used in combination with psychotherapy, is a promising treatment for post-traumatic stress syndrome, they were not ready to endorse its approval.
With its hands full developing QRL-101 and QRL-201, both in clinical trials for amyotrophic lateral sclerosis with plans to expand into other neurodegenerative diseases such as frontotemporal dementia, Quralis Corp. has decided to out-license its preclinical ALS and FTD drug, QRL-204.
Unblinding, zealous therapists, severity of harms, abuse potential and actual benefit could all be part of the conversation June 4 when the U.S. FDA’s Psychopharmacologic Drugs Advisory Committee meets in person for the first time since the COVID-19 lockdowns to consider the use of a psychedelic drug, guided by psychotherapy, to treat post-traumatic stress disorder, or PTSD.
Scientists from the PsychENCODE Consortium have analyzed the brain transcriptome in a coordinated series of studies to map all the cell types, genes, epigenetic factors, and molecular pathways involved in different psychiatric disorders. After a first set of projects based on bulk analysis, the second phase of this project included 14 simultaneous publications that revealed the cellular atlas of post-traumatic stress disorder and major depressive disorder, among others.
Understanding psychiatric disorders at a cellular and molecular level could provide a different perspective to design diagnostic and therapeutic tools searching for the origin of these disorders and the alterations they cause. Fourteen simultaneous studies from the PsychENCODE Consortium have delved into the cellular atlases of human neurodevelopment, reporting the broadest view of neuropsychiatry to date.
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