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Huntington's Disease

11:02 a.m. March 24, 2021 - By Jaimy Lee
Ionis shares tumble 19% after Roche halts Phase 3 study for rare-disease drugShares of Ionis Pharmaceuticals Inc. were down 19.6% in premarket trading on Tuesday, the day after the company said Roche Holding AG decided to discontinue a late-stage clinical trial for a Huntington's disease treatment candidate. Roche licensed the investigational therapy, tominersen, from Ionis in December 2017. However, an independent data monitoring committee first made the recommendation to discontinue the study. "The program isn't necessarily dead, but it's unlikely to drive much value for the stock near term," Bernstein's Vincent Chen wrote in a note to investors on Tuesday. "This discontinuation doesn't mean that the program is dead. It depends on why it failed." Ionis' stock is up 33.7% over the past 12 months, while the broader S&P 500 has gained 70.9%.
10:03 a.m. Feb. 8, 2021 - By Jaimy Lee
UniQure's stock rallies 7% on positive safety data for Huntington's disease treatmentShares of UniQure gained 7.2% in trading on Monday after the company said an independent data safety monitoring board said there were no safety concerns in a Phase 2/3 clinical trial for its experimental Huntington's disease treatment. The board has reviewed six months of safety data for the first two patients enrolled in the randomized, double-blind study. UniQure said it expects to enroll six more patients who have been diagnosed with the rare neurodegenerative disease by mid-2021. The investigational gene therapy is called AMT-130. "While early, we remain bullish about uniQure's [Huntington's disease] gene therapy and continue to believe the program is nearly all upside from current levels," Raymond James analyst Danielle Brill wrote in a note to investors. Uniqure's stock is down 35.9% for the past year, while the broader S&P 500 has gained 16.5%.
4:40 a.m. Sept. 22, 2020 - By Jaimy Lee
Vaccinex's stock falls after investigational drug fails Huntington's disease studyShares of Vaccinex Inc. tumbled 61.6% in premarket trading on Tuesday after the preclinical biotechnology company said an experimental Huntington's disease treatment, pepinemab, did not meet the primary endpoints in a Phase 2 double-blind, placebo-controlled clinical trial. Earlier this month, Vaccinex had announced plans to study pepinemab in combination with Merck & Co. Inc.'s Keytruda as a possible treatment for advanced forms of metastatic head and neck squamous cell carcinoma. Vaccinex's stock is up 12.8% so far this year. The S&P 500 has gained 1.5%.
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