Kynexis announces positive topline results from phase 1 study of KYN-5356
- KYN-5356, a first-in-class small molecule inhibitor of KAT-II, was safe and well-tolerated in a phase 1 study.
- The study showed evidence of target engagement and suggestive cognitive improvement in healthy volunteers.
- KYN-5356 demonstrated excellent pharmacokinetic properties and significant brain penetration.
- A clinical trial in patients with cognitive impairment associated with schizophrenia is anticipated to begin in 2025.
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Annexon announces positive topline results from real-world evidence study comparing ANX005 treatment to IVIg or plasma exchange in a matched patient cohort for the treatment of Guillain-Barré syndrome
- Annexon reported positive results from a real-world evidence study comparing ANX005 to IVIg or plasma exchange for Guillain-Barré syndrome (GBS).
- The study involved 79 patients treated with ANX005, matched 1:1 with patients from the IGOS registry based on disease severity.
- ANX005 showed faster and greater improvement in muscle strength and disability, with fewer patients requiring mechanical ventilation.
- Annexon plans to submit a U.S. Biologics License Application for ANX005 in the first half of 2025.
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Tiziana Life Sciences doses first Alzheimer's patient with intranasal foralumab
- Tiziana Life Sciences has dosed the first patient with moderate Alzheimer's disease using intranasal foralumab at Brigham and Women’s Hospital.
- Foralumab is a fully human, anti-CD3 monoclonal antibody aimed at reducing brain microglial inflammation by stimulating T regulatory cells.
- The study is part of a broader development program, supported by a $4 million NIH grant, to explore foralumab's potential in treating Alzheimer's.
- Future plans include expanding the study to patients with mild Alzheimer's disease.
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Atsena Therapeutics completes dosing in phase I/II trial for ATSN-201
- Atsena Therapeutics has completed dosing in Part A of the LIGHTHOUSE study, a Phase I/II trial for ATSN-201, targeting X-linked retinoschisis (XLRS).
- The trial evaluates the safety and efficacy of ATSN-201, using the novel AAV.SPR capsid for gene therapy.
- Nine adults were treated with three dose levels, showing structural and functional benefits without serious adverse events.
- The study continues with ongoing enrollment, and ATSN-201 has received Orphan Drug and Rare Pediatric Disease designations from the FDA.
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Contineum Therapeutics initiates patient dosing in Phase 1b PET trial of PIPE-791
- Contineum Therapeutics has started dosing the first cohort in the Phase 1b PET trial of PIPE-791.
- PIPE-791 is a brain-penetrant small molecule antagonist targeting the LPA1 receptor.
- The trial will assess pharmacokinetics and receptor occupancy in healthy volunteers, IPF, and PrMS patients.
- Topline data from the trial is expected in the second quarter of 2025.
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Corcept’s phase 3 study of relacorilant shows durable improvements in hypercortisolism
- Corcept Therapeutics presented results from a Phase 3 long-term extension study of relacorilant for endogenous hypercortisolism.
- The study involved 116 patients and demonstrated significant cardiometabolic improvements, including reductions in blood pressure.
- Relacorilant was well-tolerated over a treatment duration of up to six years.
- These results will support the new drug application (NDA) for relacorilant, planned for submission this month.
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Tenaya Therapeutics reports promising early data from MyPEAK-1 Phase 1b/2 clinical trial of TN-201 for treatment of MYBPC3-associated hypertrophic cardiomyopathy
- The MyPEAK-1 trial is evaluating TN-201 gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM).
- Preliminary data from the first cohort showed TN-201 was well tolerated with increasing RNA and protein levels.
- No cardiac toxicities were observed, though some patients experienced manageable liver enzyme elevations.
- Further data from Cohort 1 and higher dose Cohort 2 are anticipated in 2025.
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