Neurological
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.
Avidity hopes to complete enrollment in mid-2025 for an ongoing Phase III trial of its antibody-oligonucleotide conjugate in myotonic dystrophy type 1, which has no approved drug therapy.
Would-be partners for the Spanish firm’s vafidemstat will be closely following the news that regulators in the US are looking favorably on the LSD1 inhibitor’s potential as a therapy for the debilitating psychiatric illness.
At an investor meeting in London, the Swiss company sets out plans for more disciplined decision-making to help it launch a new wave of blockbuster drugs.
The charges stemmed from allegations of manipulation of Phase II data for the Alzheimer’s disease drug simufilam.
Emerging Company Profile: Danish group Vesper Bio is advancing an oral sortilin inhibitor in frontotemporal dementia, and is aiming to get to market in a similar timeframe to its much larger rivals.
BMS said Cobenfy’s annual list price of $22,500 is in line with other branded antipsychotics and sees the drug as a 2025 launch given late 2024 approval and ongoing reimbursement talks.