Huntington disease 2 potential ones trails stop for negative outcomes:
"""Tough news! Roche announced that it halted dosing in its historic Phase 3 Huntington’s clinical trial of its gene silencing drug tominersen, GENERATION HD1, because of unfavorable efficacy data, as seen by an independent review committee. “The committee recently met for a pre-planned review of the latest safety and efficacy data from GENERATION HD1 and made a recommendation about the investigational therapy’s potential benefit/risk profile,” wrote David West, Roche’s senior director for Global Patient Partnership. “Based on the committee’s recommendation, we will permanently stop dosing with tominersen and placebo in the GENERATION HD1 study.” This means that the drug demonstrated an “unfavorable efficacy trend,” an official of U.S. Roche’s subsidiary Genentech said. “This is brutal and I am absolutely devastated for our patients and families,” said Dr. Jody Corey-Bloom, the director of the HDSA Center of Excellence at UC San Diego. HDSA Chief Scientific Officer George Yohrling, Ph.D., called the news “devastating.” “HD families around the world had their hopes held high that this experimental drug could one day soon become an effective therapy for HD,” Dr. Yohrling stated. “While this is clearly not the news we wanted to hear, I am confident that in the coming weeks the Generation HD1 data will help the scientific community understand why tominersen did not meet its desired outcome.” The stop to the Roche trial underscores the fact that an effective treatment still eludes not only HD scientists, but also researchers of other neurological conditions. The conclusions about GENERATION HD1 – and possible next steps by Roche – will depend on the analysis of the independent reviewers’ explanations and all of the massive data from the Phase 3 trial. Fortunately, the HD research community has deliberately diversified the approaches to treating HD. Thus, companies like Triplet Therapeutics, Inc.??"
