Regeneron and Johnson & Johnson Report Breakthrough Phase 3 Results for First-in-Class Therapies in Ultra-Rare Disease and Psoriasis

Potential First-in-Class! Reduces Disease Marker by Nearly 100%, Regeneron's Antibody NDA Submission Imminent

Regeneron Pharmaceuticals today announced that its investigational therapy garetosmab for the treatment of adults with fibrodysplasia ossificans progressiva (FOP) met the primary endpoint in the Phase 3 OPTIMA study. The therapy significantly reduced the number and volume of new heterotopic ossification (HO) lesions. The company plans to submit a related regulatory application to the U.S. FDA by the end of 2025.According to the press release, garetosmab significantly reduced both the number and volume of HO lesions in adult FOP populations.The FirstTreatment Plan.

Garetosmab is a monoclonal antibody targeting Activin A., which is considered the key protein for HO lesion formation in FOP patients. FOP is a disease where muscles, tendons, and ligaments are progressively replaced by bone tissue, eventually leading to the loss of mobility in patients. Study results showed that the primary endpoint was met at 56 weeks: a ≥90% reduction in new HO lesions.Two dosing regimens (3 mg/kg and 10 mg/kg) reduced the number of new HO lesions by 94% and 90% respectively compared to placebo, with an overall reduction in new HO lesion volume exceeding 99%.Based on the above efficacy and safety results, the Independent Data Monitoring Committee (IDMC) recommended that patients in the placebo group be transferred to garetosmab treatment as soon as possible.

Show Lasting Efficacy! Johnson & Johnson Announces Positive Phase 3 Results for Oral Peptide

Today, Johnson & Johnson announced its investigational oral peptide therapy icotrokinra for the treatment of moderate to severe plaque psoriasis (PsO) Phase 3 study efficacy and safety results. Analysis of the ICONIC-ADVANCE 1 and 2 Phase 3 studies shows,Icotrokinra Achieves Two Co-Primary Endpoints at Week 16; Demonstrates Superiority in Skin Clearance Compared to Approved Therapies at Multiple Time Points, Including Weeks 16 and 24.In terms of safety, no new safety signals were identified. The incidence of adverse events with icotrokinra was comparable to placebo and numerically lower than the approved drug by week 24.

In the ICONIC-LEAD withdrawal/re-treatment study, icotrokinra maintained sustained skin clearance through week 52 with a favorable safety profile and no new safety signals observed. At week 24, adult responders who achieved 90% improvement in the Psoriasis Area and Severity Index (PASI 90) were re-randomized; results showed,The proportion of patients maintaining PASI 90 in the icotrokinra group was significantly higher than that in the placebo group at Week 52 (84% vs 21%; p<0.001).In addition,In the adolescent population, 86% of those who continued receiving icotrokinra for 52 weeks by Week 52 achieved PASI 90, while 77% of those who switched from placebo to icotrokinra at Week 16 achieved PASI 90.

Icotrokinra is a potential "first-in-class" targeted oral peptide that can selectively block the IL-23 receptor (IL-23R).IL-23 plays a key role in the pathogenic T-cell activation in moderate to severe plaque PsO and is fundamental to the inflammatory response in PsO and other dermatological, rheumatological, and IL-23-mediated gastrointestinal conditions. Icotrokinra can bind to IL-23R with single-digit picomolar affinity and exerts potent selective inhibition of IL-23 signaling in human T cells.

Effectively Reduces Pathogenic Factor Levels! Positive Results of Small Molecule Trials Announced

Today, Skyhawk Therapeutics announced positive results from the first interim analysis of patient cohorts in Part C of the Phase 1 clinical trial for its investigational drug SKY-0515 in the treatment of Huntington's disease (HD). SKY-0515 is an orally administered small-molecule RNA splicing modulator developed based on the company’s proprietary RNA splicing platform, SKYSTAR. It aims to simultaneously reduce levels of huntingtin protein (HTT) and PMS1 protein, with PMS1 considered one of the key drivers of somatic CAG repeat expansion and HD pathogenesis.

The interim analysis showed that on Day 84,Patients treated with SKY-0515 showed a dose-dependent reduction in mutant huntingtin protein (mHTT) in the blood, with a 62% decrease in the 9 mg dose group.; SKY-0515 was well tolerated overall at the two evaluated dosing levels. Further results indicate that the drug can reduce in a dose-dependent manner.PMS1 mRNA, and demonstrated good central nervous system penetration. No new safety signals were observed, and the overall data support the continued clinical development of the project; the phase 2/3 trial of this therapy is currently underway.

References:

[1] Regeneron Announces Positive Phase 3 Trial in Adults with Ultra-Rare Genetic Disorder Fibrodysplasia Ossificans Progressiva (FOP), Demonstrating that Garetosmab Prevents Greater than 99% of Abnormal Bone Formation. Retrieved September 17, 2025 from https://www.globenewswire.com/news-release/2025/09/17/3151506/0/en/Regeneron-Announces-Positive-Phase-3-Trial-in-Adults-with-Ultra-Rare-Genetic-Disorder-Fibrodysplasia-Ossificans-Progressiva-FOP-Demonstrating-that-Garetosmab-Prevents-Greater-than-.html

[2] Icotrokinra shows superiority to deucravacitinib in first reported head-to-head trials reinforcing promise of novel targeted oral peptide for treatment of plaque psoriasis. Retrieved September 17, 2025 from https://www.prnewswire.com/news-releases/icotrokinra-shows-superiority-to-deucravacitinib-in-first-reported-head-to-head-trials-reinforcing-promise-of-novel-targeted-oral-peptide-for-treatment-of-plaque-psoriasis-302557781.html

[3] Skyhawk Therapeutics Announces Positive First Interim Results in Patients from its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington's Disease. Retrieved September 17, 2025 from https://www.prnewswire.co.uk/news-releases/skyhawk-therapeutics-announces-positive-first-interim-results-in-patients-from-its-phase-1-clinical-trial-of-sky-0515-as-a-treatment-for-huntingtons-disease-302559094.html