CNS

France’s Provepharm Life Solutions, which finds new uses for known mature molecules, in particular antidotes and surgical dyes, by improving their efficacy and maximizing their therapeutic potential, has fresh funds to expand its portfolio.

After discussions with the US FDA, the company determined it can file AMX0035 based on Phase II/III results and interim Phase III data. Amylyx is sticking with its go-it-alone strategy for global commercialization.

Belén Garijo, the German group's CEO, has told analysts she considers targeted smaller to medium-sized acquisitions to be more likely than major transformational deals but said that "we are always open to something bolder."

CEO Michel Vounatsos and US president Alisha Alaimo outlined plans to overcome the Alzheimer’s drug’s launch challenges and hinted at lower sales expectations at a Morgan Stanley investor event.

The company, which has treatments in the clinic for CLN2 Batten disease and APOE4-associated Alzheimer’s disease, is developing AAV-mediated gene therapies designed for safer outcomes.

Emerging Company Profile: Monument Therapeutics’ novel approach to CNS drug development focuses on repurposing existing drugs with the aid of digital biomarkers.   

Deal Snapshot: Roche gene therapy subsidiary Spark gets option and will partner with start-up NeuExcell on a Huntington’s gene therapy that researchers say may regenerate functional neurons.

The diuretic bumetanide had shown potential to improve the core symptoms of children and adolescents with autism in small studies but has failed to demonstrate any sign of effectiveness in two late-stage trials.

Further promising compounds will be explored after Bristol Myers Squibb exercised its option to enter an exclusive license for Evotec’s investigational EVT8683, generated by the German group’s iPSC platform as a potential treatment for neurodegenerative conditions.

Semorinemab met only the cognition co-primary endpoint in the Phase II LAURIET trial in mild-to-moderate Alzheimer’s patients and missed all secondary endpoints, but some firsts were achieved in the top-line readout.

Korea's GNT Pharma is set to take its first-in-class, multi-targeted stroke therapy Nelonemdaz into Phase III, raising hopes for a new treatment that could sharply reduce disabilities and death in stroke patients. 

Cassava’s claims that its drug reversed cognitive decline in Alzheimer’s patients are seen as "too good to be true" by skeptics. A statement by former lab services partner Quanterix has further undermined investor confidence.