Stock market today: S&P 500 climbs as health care, tech gain; Nvidia earnings loom
Investing.com -- Shares of Regulus Therapeutics (NASDAQ:RGLS) soared 23.3% today following the announcement of successful results from its Phase 1b multiple ascending dose (MAD) clinical trial of farabursen for the treatment of autosomal dominant polycystic kidney disease (ADPKD).
The biopharmaceutical company reported that the trial met its primary endpoints, showing a statistically significant increase in urinary PC1 and PC2 protein levels, which are inversely correlated with ADPKD severity, and a halt in the growth of total kidney volume (htTKV) over the four-month study period. The data suggests that farabursen may directly impact disease progression by limiting abnormal cyst growth.
The trial’s fourth cohort, consisting of 26 subjects, received a 300 mg dose of farabursen every other week for three months. This group demonstrated a mean htTKV growth rate of 0.05%, compared to a mean growth rate of 2.58% in placebo subjects. Exploratory analysis also indicated that farabursen-treated patients experienced a reduction in htTKV growth rate when compared to historical control groups from previous ADPKD trials.
Preston Klassen, M.D., President and Head of Research & Development at Regulus, expressed confidence in the optimal target exposure provided by the 300 mg dose and the potential for farabursen to halt kidney growth in ADPKD patients. The company is preparing to advance into a pivotal Phase 3 trial later this year.
CEO Jay Hagan thanked the patients, physicians, and support staff involved in the development of farabursen and highlighted the alignment with the FDA on the program’s plans and Phase 3 trial design, which may support Accelerated Approval.
Amidst this positive news, Wells Fargo (NYSE:WFC) analyst Yanan Zhu upgraded Regulus Therapeutics from Equal Weight to Overweight and raised the price target from $3.00 to $6.00, further buoying investor confidence in the stock.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.