The biotech sector is closing the year with a flurry of clinical trial updates. Immutep, PYC Therapeutics, and Percheron Therapeutics have recently shared developments regarding their respective programs.
Immutep's IMP-761 Shows Favorable Safety Profile
Immutep (ASX:IMM) announced positive Phase I results for IMP-761, its LAG-3 (lymphocyte-activation gene-3) agonist. The first-in-human trial showed no significant safety or toxicity issues. IMP-761 aims to enhance LAG-3's function in suppressing the production of rogue T-cells, which contribute to autoimmune diseases. This mechanism differs from other immunotherapy agents that act as 'accelerators' of the immune system. The drug is being developed as a potential treatment for autoimmune diseases such as rheumatoid arthritis, type-1 diabetes, and multiple sclerosis.
The placebo-controlled, double-blind study, conducted in the Netherlands, is evaluating IMP-761 in up to 49 healthy participants. "Given that IMP-761 is potentially addressing the root cause of many different autoimmune diseases, we are eager to see this study generating more data," said Dr. Frédéric Triebel, Immutep's chief scientific officer.
Percheron Therapeutics Focuses on Duchenne Muscular Dystrophy
Percheron Therapeutics (ASX:PER) is set to release top-line results from its Phase 2b trial of ATL-1102 for Duchenne muscular dystrophy. Duchenne muscular dystrophy is a genetic condition affecting approximately 1 in 10,000 males, resulting from a gene mutation that impairs the production of the muscle protein dystrophin. This leads to muscle damage, chronic inflammation, and progressive loss of function.
The Phase 2b trial enrolled 48 wheelchair-bound boys across 16 hospitals in five countries. ATL-1102 is designed to exert an immune-modulatory effect, potentially offering therapeutic benefits in various inflammatory diseases.
PYC Therapeutics Advances Program for Phelan-McDermid Syndrome
PYC Therapeutics (ASX:PYC) recently updated investors on its program targeting Phelan-McDermid syndrome, a rare genetic disorder characterized by delayed development, intellectual disability, and speech impairment. Pre-clinical animal studies of PYC-002 have shown promise in addressing the underlying cause of the syndrome. Human trials are anticipated to begin in 2026.
Neuren Pharmaceuticals (ASX:NEU), which has a market cap of $1.6 billion, has also been active in this space, having completed a Phase II trial for Phelan-McDermid syndrome and is currently in discussions with the US Food & Drug Administration regarding the next steps.