Celon Pharma Reports Phase II Trial Results for CPL' 116 in Rheumatoid Arthritis Treatment

On June 17, 2024, Celon Pharma S.A. (CLN.WA) declared the successful conclusion of a Phase 2 clinical trial of CPL'116, a JAK/ROCK dual inhibitor, for treating Rheumatoid Arthritis (RA). The study met its primary endpoint, demonstrating statistically significant results.

Conducted as a multicentre, randomized, and double-blind trial, the study included over 100 patients who exhibited insufficient response to methotrexate. Participants received CPL'116 over 12 weeks in three doses: 60 mg, 120 mg, and 240 mg twice daily, in addition to methotrexate. The trial was placebo-controlled, with the main objective being to evaluate the dose-dependent response. The primary endpoint was assessed based on changes in the Disease Activity Score-28 for RA with CRP (DAS28-CRP) from baseline at week 12. Secondary endpoints included efficacy measurements using various scales, remission rates, and safety of the drug.

Results indicated that CPL'116 improved patient conditions in a dose-dependent manner, as reflected in the DAS28-CRP scores. By week 12, the changes from baseline for the 60 mg, 120 mg, and 240 mg doses were 1.702, 2.032, and 2.361, respectively, compared to 1.668 for the placebo. The change in DAS28-CRP compared to placebo (Least Square Mean Difference) was 0.145 (p=0.67) for 60 mg, 0.564 (p=0.10) for 120 mg, and 0.887 (p=0.01) for 240 mg. Thus, the primary endpoint was achieved with statistical significance.

The 240 mg dose displayed a rapid response, with significant improvements over placebo in DAS28-CRP scores by week 4. This dose also showed statistically significant differences in most secondary endpoints and maintained a high remission rate throughout the trial, defined as DAS28-CRP<2.6. The 120 mg dose was moderately effective, showing significant differences in some measurements compared to placebo.

The overall tolerability of CPL'116 was favorable, with no unexpected adverse effects beyond those typical of the drug class.

In the forthcoming weeks, Celon Pharma plans to present detailed pharmacokinetic and pharmacodynamic analyses, as well as comprehensive safety evaluations of this trial. The successful outcomes support further clinical development of CPL'116 as the first dual JAK/ROCK inhibitor for a variety of autoimmune diseases, especially those involving inflammatory and fibrotic components, such as idiopathic pulmonary fibrosis (IPF) and rheumatoid arthritis-associated interstitial lung disease (RA-ILD).

Celon Pharma, founded in 2002 by Maciej Wieczorek, PhD, is an integrated biopharmaceutical firm focused on designing, developing, manufacturing, and distributing pharmaceutical products. With approximately 500 employees, including around 160 scientists, half of whom hold or are pursuing PhDs, the company has a robust pipeline featuring over a dozen innovative drug development projects targeting oncology, neuro-psychiatry, autoimmunity, and metabolism. Celon Pharma operates its own research and development laboratories and a modern manufacturing facility for producing dry pharmaceutical forms. The company has also been a pioneer in inhalation drug technology since 2007 and has been listed on the Warsaw Stock Exchange since 2016.