Idorsia’s Fabry disease therapy “did exactly what we were expecting,” according to one executive—except relieve neuropathic pain.
That means lucerastat failed in a phase 3 clinical trial in adult patients with Fabry disease, a rare metabolic disorder that causes episodes of severe burning pain and can eventually lead to kidney failure or cardiac disease.
The trial, called MODIFY, was looking at the efficacy and safety of the therapy, with a specific goal of improving neuropathic pain during six months of treatment, as measured by patients. The study enrolled around 100 patients to try and relieve the burning pain that is a hallmark of the disease and one of the most difficult symptoms reported by patients.
Guy Braunstein, M.D., who serves as Idorsia’s head of global clinical development, said the drug succeeded in reducing plasma Gb3, which is a known biomarker for the disease. This confirmed the pharmacological activity of lucerastat, but unfortunately, the goal was to reduce pain.
Idorsia is not quite ready to give up on lucerastat and will await interim results of an open-label extension study before determining next steps for the development program.
“Taking into account the quality of the study, the volume of data we have collected, and some observations made in the six-month double-blind placebo-controlled treatment period, we need to wait for the results of the interim analysis of the open-label phase before making a decision,” said CEO Jean-Paul Clozel, M.D. “I expect to be in a position to share our future direction before the end of year.”
Previous studies of the drug have shown a rapid effect on plasma Gb3 levels and tolerability even at high doses. Idorsia was hoping that lucerastat could become a treatment for all different types of Fabry disease, regardless of a patient's genetic mutation type.
Idorsia arrived on the biotech scene with a splash in 2017, by way of a spin out from Actelion, which was bought by Johnson & Johnson. The Swiss company launched with $1 billion in cash and a handful of clinical stage drug candidates—plus a potentially lucrative development deal with J&J for the blood pressure medication aprocitentan.
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Back in 2020, Idorsia had success with a phase 3 insomnia med that’s being developed as a rival to Merck’s Belsomra. That news sent the company’s shares surging double digits.
The latest Fabry news saw the shares dip 6.5% to $20.14 on the Swiss Stock Exchange as of 8:38 a.m. ET.