Corestemchemon Inc. is planning to file a BLA for Neuronata-R (lenzumestrocel) by the end of 2025 to gain accelerated approval from the U.S. FDA, company officials confirmed to BioWorld during a June 2 interview. Neuronata-R is an autologous bone marrow-derived mesenchymal stem cell therapy that first gained approval in South Korea in 2014 to delay the progression of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder also known as Lou Gehrig’s disease.
As it advances its nonopioid analgesic ATX-101 breakthrough therapy through a phase IIb registration trial, Allay Therapeutics secured $57.5 million in a series D round, which included an investment from the company’s Japanese partner.
Aribio Co. Ltd. signed a $600 million license deal with Acino International AG, an Arcera Life Sciences subsidiary, granting the latter commercial rights to its oral Alzheimer’s disease therapy, AR-1001, in select countries including the Middle East.
Annji Pharmaceutical Co. Ltd.’s rosolutamide (AJ-201, JM-17) achieved positive results in a phase I/II trial in adults with spinal and bulbar muscular atrophy, also known as Kennedy’s disease.
Following a complete response letter last year, Satsuma Pharmaceuticals Inc. received U.S. FDA approval of dihydroergotamine nasal powder to treat acute migraine with or without aura. Branded Atzumi, it is the first product that uses Satsuma’s SMART (Simple MucoAdhesive Release Technology) platform that combines an advanced powder and device technology aimed at making delivery more simple.
Genes associated with lysosomal dysfunction increase the risk of Parkinson’s disease (PD), according to a study led by scientists at Northwestern University. The discovery also explains why some people who carry a pathogenic variant of the GBA1 gene develop PD or dementia with Lewy bodies (DLB) and others do not. The key lies in the Commander complex, involved in the transport of proteins to this organelle. This discovery raises the need for combinatorial therapies that act on more than one pathway for this type of neurodegenerative disorder.
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.
Tryptamine Therapeutics Ltd. is gearing up to enter the clinic with lead compound TRP-8803, an intravenous-infused psilocybin therapy, in patients with binge eating disorder in conjunction with psychotherapy.
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.
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.