Japan’s Healios K.K. is in discussions with Japan’s Pharmaceutical and Medical Devices Agenc about the regulatory path forward for its Multistem somatic stem cell products, for which it conducted phase II/III trials in ischemic stroke and a phase II trial in acute respiratory distress syndrome.
As Novartis AG works to streamline the company and finalized a long-considered plan to separate its Sandoz Inc. business by creating a standalone company, it temporarily stopped dosing in a study of branaplam for treating Huntington’s disease. Several findings from the phase IIb study suggested the presence of peripheral neuropathy in some participants. An independent data monitoring committee recommended the dosing halt but fell short of recommending the study be terminated. The VIBRANT-HD steering committee agreed with the committee’s recommendation.
Noninvasive electrical stimulation of the brain for 20 minutes per session over four days has been demonstrated to improve both working- and long-term memory for at least one month, in people ages 65 to 88.
Alterity Therapeutics Ltd. has begun phase II trials with its lead candidate, ATH-434, in multiple system atrophy, a rare and highly debilitating Parkinsonian disorder.
Central nervous system specialist Cerevance Inc. has announced a multiyear strategic research collaboration with Merck & Co. Inc. to identify novel targets for Alzheimer’s disease in a deal worth more than $1.1 billion, plus royalties. Boston-based Cerevance will receive $25 million up front with the $1.1 billion due when certain development and commercial milestones are achieved.
Following the readout of a phase II trial evaluating its COVID-19 oral antiviral Pentarlandir (SNB-01), Taiwan’s Syneurx International Corp. said it expects to launch a phase III test of the candidate in the next few months.
Shares in Karuna Therapeutics Inc. soared Aug. 8 as the biotech moved closer to a breakthrough for schizophrenia therapy after its potential first-in-class drug Karxt (xanomeline + trospium) met its goals in a closely watched phase III trial. Analysts said results from the Emergent-2 trial were at the top end of what was expected, lifting Karuna’s shares (NASDAQ:KRTX) 71.8% to a 52-week high of $241.19.
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.
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