TrueBinding receives FDA IND go ahead for Parkinson’s Phase 2A clinical trial
- TrueBinding has received FDA IND approval for TB006, a monoclonal antibody targeting Galectin-3, for Parkinson’s Disease.
- TB006 aims to neutralize Galectin-3, potentially modifying the disease by reducing neurotoxic oligomers and inflammation.
- The Phase 2A trial will be multi-center, placebo-controlled, enrolling 62 patients aged 50-80 with early to mild Parkinson’s.
- The trial's primary completion is expected by Q1 2026, focusing on safety and efficacy in patients with limited treatment options.
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Regenxbio initiates pivotal phase of Affinity Duchenne trial of RGX-202 gene therapy and reports positive functional data
- Regenxbio has started the pivotal phase of the Affinity Duchenne trial for RGX-202, enrolling ambulatory patients aged 1 and above.
- Phase I/II data shows RGX-202 recipients exceeding benchmarks for clinical outcomes with functional improvements at 12 and 9 months.
- The trial aims for a Biologics License Application (BLA) submission in 2026 using the accelerated approval pathway.
- No serious adverse events were reported, and the therapy showed a favorable safety profile.
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Neurogene updates on NGN-401 gene therapy trial for Rett syndrome
- Neurogene reported a serious adverse event (SAE) in a participant of the NGN-401 Phase 1/2 trial for Rett syndrome.
- The SAE involved a systemic hyperinflammatory syndrome in a high-dose cohort participant.
- The FDA reviewed safety data and allowed the trial to continue with a lower dose.
- Neurogene paused the high-dose cohort and will not enroll further participants at that dose.
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Contineum Therapeutics expands clinical development of PIPE-791 with FDA authorization
- Contineum Therapeutics received FDA authorization for its IND application for PIPE-791, targeting chronic pain in osteoarthritis and low back pain.
- PIPE-791 is a novel small molecule antagonist of the LPA1 receptor, aimed at providing a non-opioid treatment option.
- The Phase 1b study will be a randomized, double-blind, placebo-controlled trial, enrolling approximately 40 patients across five sites.
- Topline data from the study is anticipated in early 2026, with the trial expected to start in Q1 2025.
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Neurogene reports third quarter 2024 financial results and highlights recent updates
- Neurogene announced interim data from the NGN-401 clinical trial for Rett syndrome, showing improvements in participants dosed with 1E15 vg.
- The FDA has allowed the continuation of the Phase 1/2 trial using the 1E15 vg dose after a serious adverse event with the 3E15 vg dose.
- Enrollment for the NGN-101 gene therapy for CLN5 Batten disease is complete, but the program will not proceed due to lack of FDA alignment.
- Neurogene raised approximately $200 million through a private placement, extending its cash runway into the second half of 2027.
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