Tolebrutinib shows 31% delay in disability progression in phase 3 study for non-relapsing secondary progressive multiple sclerosis
- Tolebrutinib delayed the time to onset of 6-month confirmed disability progression (CDP) by 31% compared to placebo in the HERCULES phase 3 study.
- Secondary endpoints showed nearly a two-fold increase in confirmed disability improvement with tolebrutinib (10%) compared to placebo (5%).
- Adverse events included liver enzyme elevations (>3xULN) in 4.1% of tolebrutinib-treated patients versus 1.6% in the placebo group.
- Global regulatory submissions for tolebrutinib are planned to begin in H2 2024.
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Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne muscular dystrophy
- Neu-REFIX Beta glucan has received Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the US FDA for Duchenne muscular dystrophy (DMD).
- These designations will facilitate the transition of pre-clinical and clinical studies from Japan and India to clinical trials in the USA.
- Neu-REFIX Beta 1,3-1,6 glucan has shown potential in reducing skeletal muscle inflammation, enhancing muscle regeneration, and improving plasma dystrophin levels in DMD patients.
- The designations offer benefits such as market exclusivity, access to grants, and fast-track progress for regulatory approval.
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Edgewise Therapeutics announces positive top-line data from Phase 1 and Phase 2 CIRRUS-HCM trials
- Edgewise Therapeutics reported positive top-line data for EDG-7500 from Phase 1 and Phase 2 CIRRUS-HCM trials.
- In Phase 1, EDG-7500 was well tolerated with no significant changes in vital signs, clinical chemistry, or LVEF.
- Phase 2 CIRRUS-HCM Part A showed a 67% mean reduction in resting LVOT pressure gradient and a 55% mean reduction in provokable LVOT-G.
- A 64% mean reduction in NT-proBNP was observed in the 200 mg cohort, indicating potential for treating diastolic dysfunction.
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Xspray Pharma announces positive FDA meeting and plans for Dasynoc NDA resubmission
- Xspray Pharma plans to resubmit its NDA for Dasynoc in Q4 2024, following a productive meeting with the FDA.
- The FDA recommended adjustments to Dasynoc's tablet strengths to reduce medication errors, requiring new batches to be produced.
- Xspray Pharma has initiated production of these new batches and will provide further clarification on the manufacturing process.
- A new PDUFA date will be assigned upon resubmission, with a final decision expected within two to six months.
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CD (Suzhou) Biopharma announces FDA clearance for phase I clinical trial of CD-001
- CD (Suzhou) Biopharma received FDA clearance for its IND application of CD-001.
- CD-001 is built on the proprietary Bispecific Fusion Protein (BsFP) platform.
- The therapy targets PD-1 positive CD8+ T cells using an anti-PD-1 antibody and engineered IL-21 mutant.
- Initial clinical data from the Phase I trial is expected in the upcoming months.
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Axcelead Drug Discovery Partners enters into master service agreement for drug discovery projects with Acadia Pharmaceuticals
- Axcelead DDP and Acadia Pharmaceuticals have signed a Master Service Agreement to support new drug discovery projects.
- The agreement aims to leverage Axcelead DDP's integrated drug discovery platform and extensive experience.
- A new drug discovery project has been launched under this agreement, with Axcelead DDP receiving an upfront fee, research funding, and milestone fees.
- Financial terms of the agreement and the new project have not been disclosed.
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Apellis receives negative CHMP opinion for pegcetacoplan for geographic atrophy in the EU following re-examination
- The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) confirmed its negative opinion on Apellis' marketing authorization application for pegcetacoplan.
- Pegcetacoplan is designed to treat geographic atrophy (GA) secondary to age-related macular degeneration.
- Despite support from the European retina community, the negative opinion leaves millions of Europeans without a treatment for this irreversible form of blindness.
- Apellis remains committed to expanding access to pegcetacoplan in the U.S. and other regions globally.
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Takeda to present additional clinical trial study data on TAK-861 for narcolepsy at Sleep Europe 2024
- Takeda will present additional data from Phase 2b trials and a long-term extension study of TAK-861 in narcolepsy type 1 (NT1) and type 2 (NT2) at Sleep Europe 2024.
- TAK-861 is an investigational oral orexin receptor 2 (OX2R) selective-agonist designed to address orexin deficiency in NT1.
- The presentations will include a podium presentation on cognitive impairment and several poster presentations on nocturnal sleep quality and sustained attention.
- Takeda has initiated the FirstLight Study, a global Phase 3 trial evaluating the efficacy and safety of TAK-861 in adults with NT1.
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