Neurological

The big pharma is tapping into Muna’s MiND-MAP platform to gain extra validation for its own early-stage work in Alzheimer’s and other neurodegenerative conditions.

China’s fresh approval of a sublingual form of Simcere's edaravone/dexborneol brain cytoprotective agent for acute ischemic stroke is paving the way for an international Phase III study in this indication.

Novartis will also pay up to $1.9bn for milestones and share US profits on the Phase II RNA-splicing candidate. PTC is discussing potential accelerated approval with the US FDA.

While most drug development in the multiple sclerosis space has focused just on relapse prevention, Immunic’s dual-acting therapy vidofludimus calcium also offers a novel approach for neurodegeneration, its CEO Daniel Vitt tells Scrip.

Deal Snapshot: The essential tremor space has not seen innovation in treatment for decades. Acadia believes Saniona's GABAA-α3 positive allosteric modulator could be the answer.

AL002 failed across the board in a mid-stage study, and Alector has trimmed its workforce as it refocuses on its other two assets.

Following ex-US approvals, PTC obtains a US OK for Kebilidi, branded Upstaza outside the US, as a gene therapy for ultra-rare AADC deficiency. The drug is administered via neurological surgery.

With two misses for emraclidine, AbbVie will hope to extract some value from the Cerevel acquisition from the Parkinson’s drug tavapadon, while BMS may get a big lead in the schizophrenia space.

The company, now the most advanced clinically in the psychedelic space after the rejection of Lykos's MDMA-based post-traumatic stress disorder drug, is cutting its workforce by a third and narrowing its research focus after shifting the timeline for its late-stage depression candidate.

As it posts third-quarter numbers that again reveal the decline of revenues from its multiple sclerosis portfolio, the US biotech major is entering into the hot area of targeted protein degradation to boost its immunology and neurology pipeline.

Aliada’s ALIA-1758 addresses the same target as Lilly’s Kisunla, but is paired with a blood-brain barrier transport technology that may offer high affinity and potential for lower dosing.

A Phase II failure with an old asset throws the company’s biomarker-driven strategy into doubt.

Vyalev, a 24-hour constant infusion of carbidopa and levodopa prodrugs, obtained US FDA approval and will launch at $119,000 per year.

Although the data are from a small bridging study, MeiraGTx plans to discuss Phase III options for AAV-GAD in Parkinson’s disease after showing efficacy on a disease rating scale and a quality-of-life measure.

Impressed by the data to date on bexicaserin for epileptic encephalopathies syndromes, the Danish drugmaker hopes the drug, through the acquisition of Longboard, will become a cornerstone of its new neuro-rare disease franchise.

The French major and partner Denali's attempts to validate RIPK1 as a promising target have once again foundered as oditrasertib comes up short in a multiple sclerosis trial months after a failure in amyotrophic lateral sclerosis.

While vatiquinone may have missed its primary endpoint in a Phase III trial, PTC is ready to file the drug for the debilitating, life-shortening disorder after showing that it slowed disease progression over 144 weeks.

Dalzanemdor missed its primary endpoint in a Phase II Parkinson’s study, Sage announced, following April’s Phase II miss in Alzheimer’s. Phase II data in Huntington’s are still expected this year.

Phase III success positions Scholar Rock’s selective myostatin inhibitor for regulatory filings in early 2025. Analysts see a blockbuster opportunity for additive therapy in spinal muscular atrophy.

CEO Chris Cargill talks to Scrip about Sosei Heptares’ new identity as Nxera, the ups and downs of being big pharma’s go-to small-molecule drug hunter and its move into commercialization in Japan.