Alzheon Inc.’s oral treatment for people in the early stages of Alzheimer’s disease missed its phase III primary endpoint, adding yet another therapy to a long list by many developers that can’t beat dementia. The study also received grant money, which is in increasingly short supply.
The current treatment strategies for neurodegenerative diseases focus on targeting Aβ in Alzheimer’s disease (AD), α-synuclein aggregates in Parkinson’s disease (PD) and anti-tau therapies, which are primarily used in AD but are also being explored for PD. At the 2025 International Conference of Alzheimer’s & Parkinson’s Disease and Related Neurological Disorders, Aditya Iyer, senior RD scientist from Amyl Therapeutics Srl, presented data on an option which could potentially serve as a pan-amyloid therapeutic.
As developers continue to search for better amyotrophic lateral sclerosis (ALS) therapies, Neurosense Therapeutics Ltd. turned up some hopeful findings from its phase IIb Paradigm trial with PrimeC. The drug, a combination therapy (ciprofloxacin and celecoxib) designed to target multiple ALS pathways, is having salutary effects on microRNA modulation (miRNA), Neurosense said, with the study showing a “profound and consistent” downregulation of 161 mature miRNAs across all time points in the double-blind period of the experiment.
Remegen Co. Ltd. emerged as a surprise challenger in the generalized myasthenia gravis space, unveiling positive phase III data of its China-approved lupus drug, telitacicept (RCT-18; Tai’ai), in the rare autoimmune neuromuscular disorder at the 2025 American Academy of Neurology conference.
China approved 48 first-in-class innovative drugs, as well as a significant number of medications for pediatric and rare diseases, thanks to measures aimed at enhancing review efficiency and accelerating patient access to novel therapies, according to a report released by China’s National Medical Products Administration.
ABL Bio Inc. announced April 7 that it sealed a potential £2.075 billion (US$2.65 billion) license deal with GSK plc, granting GSK global rights to use ABL’s blood-brain barrier (BBB) penetrating bispecific antibody platform, Grabody-B, to develop multiple programs in the neurodegenerative disease arena. Under the terms signed April 5, ABL agreed to transfer Grabody-B-related technology and know-how to GSK, upon which GSK will assume responsibility for preclinical and clinical development, manufacturing and commercialization.
Neurona Therapeutics Inc. has completed an upsized, oversubscribed $102 million financing round, the third company to do so in a month that’s only four days old. The privately held company’s lead asset is NRTX-1001, a regenerative cell therapy derived from human pluripotent stem cells.
Neurodegenerative pathologies, once primarily associated with protein alterations, should be revisited in the context of lipidopathies, researchers argued at the 2025 International Conference of Alzheimer’s & Parkinson’s Disease and Related Neurological Disorders (AD/PD 2025).
Sangamo Therapeutics Inc. is adding a much-needed $18 million up-front payment in a neurology-focused deal with Eli Lilly and Co. that could bring up to an additional $1.4 billion. In return, Lilly gets access to Sangamo’s neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, which has shown early promise in penetrating the blood-brain barrier penetration, for one initial target with the right to add up to four more.
Sumitomo Pharma Co. Ltd. announced that it will sell off two more of its subsidiaries, Sumitomo Pharma (China) Co. Ltd. and Sumitomo Pharma Asia Pacific Pte. Ltd. (and their subsidiaries), to Marubeni Global Pharma Corp. April 1, as the Japanese pharma continues restructuring efforts from last year.
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