Neurological
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.
Having earlier shown efficacy as adjunctive therapy, AbbVie is waiting for data from a third Phase III trial before determining filing plans for the D1/D5 receptor agonist.
IntraBio and Zevra will go head-to-head in the treatment of Niemann-Pick disease type C, with US FDA approvals of Aqneursa and Miplyffa, respectively, less than a week apart.
The FDA is scheduled to pronounce on Bristol Myers Squibb’s potential blockbuster on 26 September. But might forthcoming compounds be more effective?
Miplyffa (arimoclomol) was approved for Niemann-Pick disease type C when administered with another high-priced drug used off label as standard of care for about 80% of NPC patients.
The company announced data from a real-world evidence study that compared a three-year analysis of existing Phase III data with an external control and plans to file with the FDA in the fourth quarter.
Delaying disease progression in non-relapsing secondary multiple sclerosis may get the BTK inhibitor approved, despite previous pivotal trial failures in relapsing MS.
The Italian firm has received "several indications of interest" in evenamide, which is being prepped to go into Phase III for treatment-resistant schizophrenia.