In deeply disappointing news for the Huntington’s disease community and beyond, Roche announced on July 9 that it stopped two drug development programs because of lack of efficacy in slowing the progression of the disease.
Tominersen, a gene silencing drug developed by Ionis Pharmaceuticals, Inc., envisioned as a “laser-guided missile” against HD, in 2021 demonstrated lack of efficacy in a first worldwide trial run by Roche.
Roche, however, had believed that tominersen might show at least some efficacy in people at earlier stages of Huntington’s. So it launched a less ambitious second trial in January 2023.
In a statement to the HD community, after completing the 16-month treatment period of all trial participants and analyzing the data, Roche stated that “there was no meaningful impact on clinical efficacy for the study participants receiving tominersen, compared to those on placebo.”
Roche also said it had halted a preliminary Phase I clinical trial of the HD gene silencing drug RG6496, which had enrolled just three volunteers so far, “because we can no longer offer participants the possibility of long-term treatment” based on data from new animal studies.
“We have been humbled and inspired by the 1,500+ HD families and broader community who contributed to both programs – tominersen since clinical studies began in 2015 with our partner Ionis Pharmaceuticals, and RG6496 more recently,” the Roche statement noted. “These contributions changed the history of HD drug development – proving the protein that causes HD could be lowered in humans, shaping new research and approaches, which will undoubtedly lead to future breakthroughs.”
The setback ‘stings’
HD family members expressed sadness about the announcement from Roche.
In a Facebook posting, Help4HD International advocate Lauren Holder, like me an HD gene carrier, said the news was “disappointing.”
“This was the clinical trial I was participating in,” Jessica Robbins, who has HD, wrote on Facebook, granting me permission to quote her. “Unfortunately, it has come to an end.”
Jessica had dedicated two years to the tominersen trial.
“I’m not going to lie – am incredibly disappointed and heartbroken,” she wrote. “A setback like this stings, but I don’t regret a single day of it. Even though this door closed, the data from our trial will still help research in the long run. I am proud to have played a part in this fight against HD.”
I, too, had hoped to take tominersen, having tracked its development since 2008.
Scores of other initiatives
Drug development does take years, even decades, as tominersen’s story illustrates.
I had found the second trial of tominersen far less compelling, though necessary for the field to advance, because the drug would have been able to help only a portion of HD patients. So the disappointment for me does not match what I felt with the community in 2021.
I do remain optimistic that effective therapies will be found – if not for me, at least for those in the next generation, like my nephew Greg Noble, also an HD gene carrier.
The HD community has especially focused on uniQure’s AMT-130, a gene therapy that, for the first time, slowed the progression of HD. uniQure and the community have worked tirelessly to overcome the regulatory roadblocks placed by the Trump administration.
Roche, too, has not given up on HD science and is working on its own gene therapy.
PTC Therapeutics and Novartis have partnered on a clinical trial program using a huntingtin splicer modulator. Skyhawk Theapeutics’ HD program has also shown promise, and the controversial pridopidine, under study by Prilenia, will undergo a new Phase III trial to determine whether the drug can slow progression.
Scores of companies continue to work on HD, as do academic labs around the world.
I do feel hope!
(Disclosure: I hold a symbolic amount of Ionis shares.)
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