Data from mid-stage trials of two of the company’s antisense therapies for Huntington’s disease (HD) failed to demonstrate efficacy, even at higher dose levels. Furthermore, Serious Adverse Events related to treatment were reported for 7 of the 13 participants in the highest dose (32 mg) group with 6 participants consequently discontinuing treatment. These included disorientation, delirium, ataxia, slurred speech, amnesia, meningitis, fever, and vertigo.
Wave is now looking to its next-generation PN backbone chemistry (said to improve potency, exposure and durability) to advance yet another therapy for Huntington’s as well as candidate therapies for amyotrophic lateral sclerosis, frontotemporal dementia and Duchenne muscular dystrophy.
Wave’s trial failure in HD closely follows Roche’s announcement last week that it was stopping a Phase 3 trial of its Ionis Pharmaceuticals-partnered antisense drug tominersen in HD. As there were no new safety problems identified in the 120mg dose used in the trial, it is believed that Roche’s decision to terminate the program was driven by lack of efficacy.
As stated by PYC in its ICR Westwicke Conference presentation in January, RNA therapeutics have come of age, but their ongoing success is impeded by inefficient or toxic delivery inside cells. PYC believes that its cell-penetrating peptide delivery platform solves this problem.
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