GSK's Linerixibat Achieves Phase 3 Primary Endpoint in PBC-Associated Cholestatic Pruritus, with 56% of Patients Showing Clinically Meaningful Itch Relief

70% of Patients Show Symptom Relief, Antibody Therapy Receives FDA Breakthrough Therapy Designation

Viridian Therapeutics Announces Veligrotug Granted Breakthrough Therapy Designation by U.S. FDAVeligrotug is an intravenous infusion antibody targeting insulin-like growth factor-1 receptor (IGF-1R)., currently in Phase 3 clinical trials for the treatment of Thyroid Eye Disease (TED). The company anticipates submitting a Biologics License Application (BLA) to the U.S. FDA in the second half of this year.

In the pivotal Phase 3 clinical trials THRIVE and THRIVE-2, veligrotug met all primary and secondary endpoints. Veligrotug demonstrated rapid onset of action in both studies, providing statistically significant and clinically meaningful improvements in diplopia relief and elimination. Previously announcedTHRIVE TrialThe results showed,At week 15, the proptosis response rate was 70% for patients treated with veligrotug and 64% for those receiving placebo-adjusted treatment (p<0.0001). Notably, 53% of patients experienced significant improvement in proptosis symptoms within 3 weeks, having received only one treatment at that point.

56% of Patients Experienced Significant Relief in Itch Symptoms, GSK's Small Molecule Drug Meets Primary Endpoint in Phase 3 Clinical Trial

GSK Announces Positive Results from Phase 3 GLISTEN Trial Evaluating Linerixibat in Adults with Cholestatic Pruritus and Primary Biliary Cholangitis (PBC)

GLISTEN Trial Meets Primary Endpoint, Results Show,Patients treated with linerixibat (n=119) showed significant improvement in pruritus within 24 weeks compared to the placebo group (n=119). The least squares mean difference between the two groups, assessed using the Worst Itch-Numerical Rating Scale (WI-NRS), was -0.72 (95% CI: -1.15, -0.28, p=0.001).The results show that linerixibat has the potential to alleviate a major symptom in PBC patients—refractory pruritus.

The study also achieved multiple key secondary endpoints, including improvement in itch score at Week 2 and improvement in itch-related sleep interference score within 24 weeks, with specific performance as follows:

Rapid onset of itch improvement was observed, with significant superiority over the placebo group by Week 2 (least squares mean difference: -0.71, 95% CI: -1.07, -0.34, p<0.001). The efficacy was sustained throughout the trial period. Over the 24-week treatment period, linerixibat significantly improved sleep disturbance caused by itching, a symptom that greatly impacts patients' quality of life, compared to placebo. By Week 24, 56% of patients in the linerixibat group achieved clinically meaningful improvement in itch scores (WI-NRS reduction ≥3 points), compared to 43% in the placebo group, with a between-group difference of 13% (95% CI: 0, 27).

Developing RNA-Targeted Therapies, Start-Up Completes $65 Million Series A Financing

HAYA Therapeutics Announces $65 Million Series A Financing to Advance Clinical Development of HTX-001, a Long Non-Coding RNA (lncRNA) Targeted Candidate Drug for Heart Failure, and Expand Its RNA-Guided Gene Regulation Platform

HAYA Therapeutics is a precision medicine company dedicated to developing programmable therapies that target regulatory non-coding RNA to reprogram pathological cell states in a variety of diseases, including cardiovascular disease, metabolic disorders, and cancer.The company is leveraging its innovative platform to deeply analyze disease cell states and the regulatory mechanisms of lncRNA involved. HAYA's lead project is HTX-001, which is being developed for the treatment of heart failure. HAYA is also developing a series of precision therapies targeting lncRNA for cell-specific treatments aimed at other tissue diseases. In September last year, HAYA reached an agreement worth up to 1 billion US dollars with Eli Lilly and Company.Multi-Year AgreementThe two companies will apply HAYA's RNA-guided gene regulation platform to support preclinical drug research and development efforts for obesity and related metabolic diseases.

References:

[1] HAYA Therapeutics Raises $65 Million in Series A Funding to Deliver Precision RNA-Guided Medicines for Chronic and Age-Related Diseases. Retrieved May 8, 2025, from https://www.hayatx.com/news-and-publications/haya-therapeutics-raises-65-million-in-series-a-funding-to-deliver-precision-rna-guided-medicines-for-chronic-and-age-related-diseases

[2] Viridian Therapeutics Receives FDA Breakthrough Therapy Designation for Veligrotug for the Treatment of Thyroid Eye Disease (TED). Retrieved May 8, 2025, from https://www.businesswire.com/news/home/20250507164030/en/Viridian-Therapeutics-Receives-FDA-Breakthrough-Therapy-Designation-for-Veligrotug-for-the-Treatment-of-Thyroid-Eye-Disease-TED

[3] GLISTEN phase III trial results show linerixibat significantly improves cholestatic pruritus (relentless itch) in primary biliary cholangitis (PBC). Retrieved May 8, 2025, from https://www.gsk.com/en-gb/media/press-releases/glisten-phase-iii-trial-results-show-linerixibat-significantly-improves-cholestatic-pruritus/