Beta-site APP-cleaving enzyme 1 (BACE1) inhibitors have a long history of failure in patients with Alzheimer’s disease. Clinical development of verubecestat, elenbecestat, lanabecestat, umibecestat, atabecestat and LY-3202626 were all discontinued. The drugs largely showed that inhibiting BACE1 reduced amyloid beta (AB) in both cerebrospinal fluid and plasma, reduced AB plaques on PET scans and reduced phosphorylated tau. Unfortunately, patients had early cognitive worsening, and there were signs of lowering of brain volume and increases in psychiatric adverse events.
During a presentation at the Alzheimer's Association International Conference 2022, researchers from the Genetic Frontotemporal Dementia (FTD) Initiative consortium presented data from a study following nearly 1,300 patients with FTD caused by a genetic mutation, their presymptomatic family members who have the inherited mutation and unaffected family members to serve as controls. Researchers have used data from the study, which has been enrolling patients for over 10 years, to develop biomarkers that can be used to assess progression of FTD in interventional clinical trials.
Sequence analysis of 131 human brains has revealed the mutagenesis processes that take place throughout life, from development to senescence. In a new study published in the July 29, 2022, issue of Science, the authors described how high rates of brain somatic mutations (what they call hypermutability) correlated with age.
Top-line results from Vistagen Therapeutics Inc.’s Palisade-1 phase III study of PH-94B for treating social anxiety disorder showed the therapy fell shy of the primary endpoint. The South San Francisco-based company’s stock (NASDAQ:VTGN) fell dramatically on the results July 22 as shares closed 86% lower at 15 cents each.
As neurological disease stocks begin to move in the right direction, and a potential U.S. FDA approval nears for a major depressive disease drug, shares are still down by 11.68% this year. BioWorld’s Neurological Diseases Index is following a similar path as the Nasdaq Biotechnology Index (NBI), which hit bottom at the end of May and has begun to climb since then. Still, NBI is down 15.92% this year, while the Dow Jones Industrial Average is down 12.41%.
The U.K.’s competition watchdog has found that a subsidiary of Pfizer Inc. and a generics firm jacked up the price of a life-saving epilepsy drug by up to 2,600%, fining them a total of £70 million (US$83.72 million). The Competition and Markets Authority’s decision is part of a long-running dispute against subsidiary Pfizer Ltd. and the generics firm Flynn Pharma Ltd.
In a sense, memories are useless without being linked to feelings. Without knowing whether a memory is good or bad, there is no way to seek out good experiences, and avoid bad ones. Now, investigators at the Salk Institute have identified neurotensin as a critical molecule for the assignment of such emotional valence.
Japan’s Kyowa Kirin Co. Ltd. has stopped development of its adenosine A2a receptor antagonist, KW-6356, in Parkinson’s disease. Discovered internally by Kyowa Kirin, KW-6356 was studied in phase II trials in Japan for Parkinson’s disease, with results showing it was “potentially effective in relieving motor and non-motor symptoms both as a monotherapy and in combination with levodopa-containing therapy,” Kyowa said. However, development is now being discontinued after conducting an evaluation of the global regulatory landscape, development hurdles and potential timelines for market entry.
New interim data from Ultragenyx Pharmaceutical Inc. and Genetx Biotherapeutics LLC’s phase I/II study of GTX-102 in Angelman’s syndrome didn’t do much to bolster investor confidence, as Ultragenyx shares sagged considerably July 19. The Novato, Calif.-based company’s stock (NADAQ:RARE) closed 13% downward at $52.89 per share. That is nearly half the price shares fetched in late August. At $102.40 per share, that was the stock’s highest value in the past 12 months.
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