Parkinson’s disease (PD) is one of the most common neurodegenerative diseases, and the role of ?-synuclein accumulation and the subsequent death of dopaminergic neurons in the midbrain have long been recognized as key steps in the disease. Progress in understanding genetic risk factors, meanwhile, has uncovered multiple genetic risk factors. Even though aging is the single biggest risk factor for PD, there are versions of the disorder that affect children.
The new peroxisome proliferator-activated receptor gamma (PPARgamma) agonist leriglitazone (hydroxypioglizatone; Minoryx Therapeutics), has a better profile for treating neurodegenerative diseases, such as X-linked adrenoleukodystrophy (X-ALD), than similar drugs, including pioglitazone (Actos; Takeda).
LONDON – Cumulus Neuroscience Ltd. has raised £6 million (US$8.3 million) to advance development of its home use wearable headset for tracking response to treatment in clinical trials in psychiatric disorders and neurodegenerative diseases, including Alzheimer’s.
Capsida Biotherapeutics Inc., a gene therapy startup focused on advanced capsid engineering to generate tissue-selective vectors, emerged from stealth with $50 million in series A funding and another $90 million in cash from a strategic collaboration and option agreement in neurodegenerative disease with Abbvie Inc.
Merck & Co. Inc. is bringing in Amathus Therapeutics Inc. to collaborate on developing small molecules to treat neurodegenerative disease. The deal is one of several in the past 18 months that has expanded Merck’s presence in the space.
DUBLIN – Mina Therapeutics Ltd. is extending its small activating RNA (saRNA) technology into the central nervous system through a partnership with Les Laboratoires Servier SAS that could be worth as much as €220 million (US$266.5 million) in up-front and milestone payments.
Prevail Therapeutics Inc. won't be prevailing on its own. Eli Lilly and Co. is acquiring the gene therapy company for $880 million up front plus an earn-out of up to $160 million if Lilly can gain regulatory approval for at least one of Prevail's drugs.
In the past 10 years, the advances in understanding the etiology of neurodegenerative diseases have been dramatic. “The development of novel biomarkers and other tools as well are key in aiding diagnostic potential and the ability to track disease progression have been phenomenal,” Isaac Veinbergs, CEO of newly created Libra Therapeutics Inc., told BioWorld.
LONDON – With results of its first phase I trial imminent, inflammasome specialist Nodthera Ltd. has raised $55 million in a series B round, providing funding for phase II proof-of-concept studies and to advance a second, brain-penetrant, compound to the clinic.
Merck & Co. Inc. acquired Calporta Therapeutics Inc. for an up-front payment and milestone payments that could total $576 million. Calporta is a build-to-buy spinout of La Jolla, Calif.-based COI Pharmaceuticals Inc. Calporta develops selective small-molecule agonists to TRPML1, foremost in the mammalian mucolipin TRP channel subfamily.
