FDA approves drug with new mechanism of action for treatment of schizophrenia
- The FDA approved Cobenfy (xanomeline and trospium chloride) for treating schizophrenia in adults.
- Cobenfy is the first antipsychotic targeting cholinergic receptors instead of dopamine receptors.
- Effectiveness was evaluated in two 5-week, randomized, double-blind, placebo-controlled studies.
- Common side effects include nausea, constipation, vomiting, and increased heart rate.
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PureTech-invented KarXT receives FDA approval for the treatment of schizophrenia in adults
- KarXT (xanomeline and trospium chloride), invented by PureTech, has received FDA approval for treating schizophrenia in adults.
- The approval triggers milestone payments totaling $29 million to PureTech under agreements with Royalty Pharma and Karuna Therapeutics.
- PureTech is entitled to potential future payments and approximately 2% royalties on net annual sales over $2 billion.
- KarXT, now marketed as Cobenfy, will be under the stewardship of Bristol Myers Squibb following their acquisition of Karuna Therapeutics.
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Longboard Pharmaceuticals initiates phase 3 DEEp SEA study evaluating bexicaserin in Dravet syndrome
- Longboard Pharmaceuticals has initiated the global Phase 3 DEEp SEA Study for bexicaserin in Dravet syndrome.
- The study will evaluate the efficacy and safety of bexicaserin in ~160 participants aged 2-65 years.
- Participants will undergo a 5-week screening, 3-week dose titration, and 12-week maintenance period.
- Eligible participants can enroll in a 52-week open-label extension study.
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Cocrystal Pharma advances oral pan-viral protease inhibitor CDI-988 into phase 1 multiple-ascending dose cohorts
- Cocrystal Pharma has begun dosing in the multiple-ascending dose (MAD) portion of the Phase 1 study for CDI-988.
- CDI-988 is a broad-spectrum, oral pan-viral protease inhibitor targeting noroviruses and coronaviruses.
- The Phase 1 study is randomized, double-blind, and conducted in Australia, focusing on safety, tolerability, and pharmacokinetics.
- Topline results from the MAD portion are expected in late 2024 or early 2025.
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ReCode Therapeutics doses first patient in phase 1b clinical study of RCT2100 for the treatment of cystic fibrosis
- ReCode Therapeutics has dosed the first patient in a Phase 1b study of RCT2100, an investigational inhaled mRNA therapy for cystic fibrosis (CF).
- RCT2100 aims to deliver CFTR mRNA directly to lung cells, potentially restoring CFTR protein function and addressing the root cause of CF.
- The study is currently enrolling in the Netherlands, with plans to expand to France, the U.S., and the UK.
- ReCode received global regulatory authorization to proceed with the study in the U.S., UK, and Europe.
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AIRAmed and Memory Treatment Centers launch Alzheimer's research partnership
- AIRAmed and Memory Treatment Centers (MTC) have partnered to study Alzheimer's disease in cognitively impaired individuals undergoing anti-amyloid therapy.
- The collaboration will utilize AIRAmed's AI-software, AIRAscore, to track changes in regional brain volume through serial MRIs.
- The study aims to provide enhanced tools for diagnosis and treatment, sharing HIPAA-compliant, real-world data with the global neurologic community.
- The partnership seeks to improve patient care, treatment strategies, and patient outcomes in the field of neurodegenerative diseases.
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Septerna initiates phase 1 trial for SEP-786 in hypoparathyroidism
- Septerna has started dosing participants in a Phase 1 clinical trial for SEP-786, an oral small molecule PTH1R agonist.
- The trial includes single-ascending dose (SAD) and multiple-ascending dose (MAD) phases to assess safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD).
- SEP-786 aims to provide a convenient, disease-modifying treatment for hypoparathyroidism, potentially normalizing serum calcium levels.
- The trial will enroll up to 180 healthy adult participants, with dosing already underway in the SAD portion.
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