Stichting Centre for Human Drug Research (CHDR)

NMD Pharma's NMD670 Enters Phase 2 Trial for Charcot-Marie-Tooth Disease

6 months ago

• NMD Pharma initiates a Phase 2 clinical trial (SYNAPSE-CMT) to evaluate NMD670 in patients with Charcot-Marie-Tooth (CMT) disease types 1 and 2. • The SYNAPSE-CMT trial is a randomized, double-blind, placebo-controlled study involving 80 adults, assessing NMD670's efficacy, safety, and tolerability over 21 days. • NMD670, a novel small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1, aims to improve muscle function by enhancing muscle excitability. • Previous Phase 1 data showed NMD670 to be generally safe and well-tolerated, supporting its further development for neuromuscular diseases.

VQ-101 Shows Promise in Phase 1 Trial for GBA-Parkinson's Disease

6 months ago

• VQ-101, an oral GCase activator, demonstrated over 75% target engagement in healthy volunteers in a Phase 1a trial, exceeding the initial goal of 50% activation. • The Phase 1a trial showed VQ-101 achieved significant cerebrospinal fluid levels, indicating full central nervous system penetration, with mild to moderate adverse events. • Vanqua Bio plans to advance VQ-101 to a Phase 1b trial in Parkinson's disease patients, with data expected in 2025, to assess its impact on alpha-synuclein accumulation. • Vanqua Bio appointed Maurizio Facheris as CMO to advance VQ-101 through clinical development, leveraging his experience with Parkinson's disease therapies.

Immutep's IMP761 Phase I Trial Advances to Dose Escalation After Demonstrating Initial Safety

7 months ago

• Immutep's Phase I study of IMP761, a novel LAG-3 immunotherapy, has completed the single-dose portion with no reported safety concerns, allowing progression to dose escalation. • The trial, conducted by CHDR in the Netherlands, will now evaluate single IMP761 doses ranging from 0.03 mg/kg to 0.90 mg/kg in healthy volunteers. • Subsequent phases will assess multiple ascending doses and further evaluate pharmacokinetic/pharmacodynamic (PK/PD) relationships using a KLH challenge model. • Initial safety data is expected by year-end, with PK/PD assessments anticipated in the first half of 2025, offering insights into IMP761's activity.

Vanqua Bio's VQ-101 Shows Promise in Parkinson's Disease with Phase Ib Trial Initiation

7 months ago

• Vanqua Bio's VQ-101, an oral small molecule, demonstrated over 75% activation of GCase in healthy volunteers, surpassing the target engagement goal in a Phase Ia trial. • The Phase Ia trial showed VQ-101 to be well-tolerated, with no dose-limiting or serious adverse events, and achieved significant CNS penetration. • A Phase Ib trial will investigate multiple doses of VQ-101 in Parkinson's disease patients, with or without GBA mutations, with topline data expected in 2025. • Preclinical data suggests VQ-101 has the potential to halt alpha-synuclein accumulation, potentially slowing or stopping Parkinson's disease progression.

Mindstate's AI-Designed Psychedelic MSD-001 Receives FDA and EMA Nod for Human Trials

9 months ago

• Mindstate Design Labs has received FDA and EMA approval to begin Phase 1 human trials for its AI-designed psychedelic compound, MSD-001. • MSD-001 is designed to selectively target serotonin receptors, offering a more controlled and predictable psychoactive effect compared to traditional psychedelics. • The Phase 1 trials will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of MSD-001 in healthy participants. • Mindstate's Osmanthus AI platform synthesizes extensive drug reports and biochemical data to design targeted states of consciousness, potentially expanding therapeutic applications.