Zhejiang Jingxin Pharmaceutical Co. Ltd. has in-licensed JBPOS-0101, a class I new drug for epilepsy, from Bio-Pharm Solutions Co. Ltd. for mainland China, Hong Kong, and Macau. The partnership could bring South Korea’s Bio-Pharm more than $40 million, including an up-front payment of $5 million, milestone payments of up to $35 million, and potential royalties on future sales.
LONDON – A significant body of research indicates inhibition of the mitochondrial permeability transition pore (PTP) would reduce neuroinflammation and protect neurons, but the difficulty of finding inhibitors that cross the blood-brain barrier to regulate the pore has left the therapeutic potential largely untapped.
DUBLIN – AC Immune SA and partner Genentech reported Aug. 31 that their Tau-targeting antibody, semorinemab, brought about a dramatic 43.6% reduction in cognitive decline vs. baseline in a phase II trial in patients with mild to moderate Alzheimer’s disease. The effect was statistically significant (p<0.0025) and is clinically meaningful. Indeed, it represents the biggest single treatment effect ever reported in a clinical trial in this population of Alzheimer’s patients and sets the stage for an extensive phase III program that could set the agenda for Alzheimer’s research for the foreseeable future.
Researchers from Denali Therapeutics Inc. have identified new functional links between progranulin, lysosomal function, and a subtype of frontotemporal dementia caused by progranulin deficiency (FTD-GRN) that suggest progranulin-mediated FTD could be conceptualized as a lysosomal storage disorder (LSD). They also showed that delivery of their experimental therapeutic PTV:PGRN, also known as DNL-593, reduced cell damage and symptoms of FTD in cell and animal models.
Following new positive top-line results from its pivotal phase III study of IPX-203, Amneal Pharmaceuticals Inc. said an NDA submission for the Parkinson’s disease therapy (PD) is now planned for the middle of 2022.
In one of the biggest collaboration deals of the year, Shape Therapeutics Inc. entered a collaboration and license agreement with Roche Holding AG to develop gene therapies for targets in areas that include Alzheimer’s disease, Parkinson’s disease and rare diseases. Seattle-based Shape is eligible to receive an initial payment, development, regulatory and sales milestone payments that could exceed $3 billion in aggregate value.
A month after Astrazeneca plc’s $39 billion takeover of Alexion Pharmaceuticals Inc. was finalized, the merged company has hit its first speed bump after the new unit discontinued a phase III rare disease trial. Alexion, of Boston, said it axed the 382-patient CHAMPION-ALS trial of its long-acting C5 complement inhibitor Ultomiris (ravulizumab) in amyotrophic lateral sclerosis (ALS).
For most people, neither polyglutamine disorders nor neuromuscular disorders are likely to be among the things they associate with androgen receptor (AR) dysfunction. But the three are indeed linked. And researchers have reported new insights into the nature of those links that could lead to a treatment for spinal and bulbar muscular atrophy, and possibly other disorders linked to AR signaling dysfunction.
LONDON – Persica Ltd. is poised to take a new pharmaceutical approach to curing chronic lower back pain, after delivering positive data in a phase Ib study of an injectable antibiotic. The development rests on research showing that, as with stomach ulcers, chronic lower back pain is caused by localized bacterial infection.
Vigil Neuroscience Inc. CEO Ivana Magovčević-Liebisch said the just-raised $90 million in series B money will let the company reach “a lot of value-generating milestones,” having been “moving extremely fast” in the year since it was founded.