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4SC AG. (11/7/11). "Press Release: 4SC Presents Encouraging Final Results of Vidofludimus Phase IIb COMPONENT Study in Rheumatoid Arthritis at ACR Meeting in Chicago". Planegg-Martinsried.

Region Region Chicago, IL
  Country United States (USA)
Organisation Organisation 4SC AG (FSE: VSC)
  Group 4SC (Group)
Products Product vidofludimus (4SC-101)
  Product 2 methotrexate (INN)
Person Person Dauer, Ulrich (Probiodrug 201805– CEO before Omeicos + Activaero + 4SC + Tripos)

– Observed anti-inflammatory activity and very clean safety profile of Vidofludimus confirm broad further potential in autoimmune diseases including IBD –

4SC AG (Frankfurt, Prime Standard: VSC), a discovery and development company of targeted small molecule drugs for autoimmune diseases and cancer, has presented final results from its clinical Phase IIb COMPONENT study with 4SC's anti-inflammatory compound vidofludimus in rheumatoid arthritis (RA) at the annual scientific meeting of the American College of Rheumatology (ACR) in Chicago, USA. The final findings of this study, in particular the observed decrease of objective inflammation parameters, reveal substantial anti-inflammatory activity of vidofludimus in RA in combination with a very good safety profile, thus strongly confirming the drug's further potential also in other autoimmune diseases including inflammatory bowel disease (IBD).

The international, multi-centre, randomised, placebo-controlled study evaluated the efficacy of vidofludimus in combination with methotrexate (MTX) (treatment group), compared to MTX alone (placebo group), in 241 RA patients. As previously announced in the study's topline results on 8 June 2011, vidofludimus improved all ACR scores (ACR20, ACR50, ACR70), which are internationally accepted clinical standards to measure the efficacy of a drug in RA, but - with an improvement of 50.8% in the treatment group vs. 44.8% in the placebo group - missed the study's primary efficacy endpoint of improving ACR20 with statistical significance.

New findings in final analysis: objective inflammation values substantially improved

Final evaluation of the full data set of the COMPONENT study now revealed that vidofludimus showed a substantial anti-inflammatory activity in RA patients. This is in particular supported by decreases of objective inflammatory parameters comprising the biomarkers C-reactive protein (CRP) and erythrocyte sedimentation rate (ESR) in the vidofludimus treatment arm compared to placebo. Group comparison of mean CRP change at week 13 compared to baseline was a reduction of 1.82 mg/l for vidofludimus whereas in contrast an increase of 1.38 mg/l was recorded for placebo. Group comparison of mean ESR change at week 13 compared to baseline was a reduction of 3.98 mm/h for vidofludimus while an increase of 3.18 mm/h was detected for placebo. These results strongly confirm the drug's broad potential in autoimmune diseases including in particular IBD, an indication in which vidofludimus generated very positive results in a Phase IIa clinical trial (the ENTRANCE study).

The general anti-inflammatory activity of vidofludimus in the COMPONENT study is supported further by statistically significant differences in various efficacy endpoints (ACR20/DAS28) at specific time points between treatment and placebo group. Thus, ACR20 response improvement of the 35 mg vidofludimus group compared to placebo was statistically significant (p<0.05) at week 8 (46.7% vs. 31.9%). Time to ACR20 response was significantly (p<0.05) shorter in the vidofludimus group compared to placebo (median 56 days vs. 92 days). In addition, the rate of DAS28 (CRP), another secondary efficacy endpoint, was significantly (p<0.05) higher in the vidofludimus group compared to placebo at week 4 (55.0% vs. 41.3%). However, the difference was not statistically significant at week 13 (60.9% vs. 56.9%). Further results show that mean changes of RA-specific parameters which are mainly based on physicians' and patients' subjective assessments (including pain intensity, patients' and physicians' assessment of disease activity, tender and swollen joint counts, and HAQ-DI) were similar in both groups at week 13, although all ACR/DAS scores were numerically higher in the vidofludimus group compared to placebo and even significantly superior at specific time points. In addition, a statistically significant negative influence of MTX dosing on vidofludimus plasma levels was observed; this might have been a reason why the full anti-inflammatory activity of vidofludimus could not be fully shown in this study since patients demonstrated to have increased ACR20 response with increasing vidofludimus plasma levels.

Confirmation of clean safety and tolerability profile of vidofludimus

The final safety analysis confirmed the very good safety profile of vidofludimus, as no difference in the adverse event profile between the treatment with vidofludimus and placebo was observed. In particular, there were no relevant increases of infections, diarrhea, neutropenia, anemia, hypertension, cholesterol or liver enzyme levels in the vidofludimus group. Only one serious adverse event was reported in the vidofludimus group which was judged as not being related to vidofludimus. These positive safety results are consistent with previous clinical study results with vidofludimus in RA and IBD patients.

"These final results of our Phase IIb COMPONENT study are very supportive to the understanding and confirmation of the anti-inflammatory activity of vidofludimus in RA patients on methotrexate background therapy and we will certainly continue to discuss these data in more detail with potential pharmaceutical partners interested in further RA development," commented Dr Ulrich Dauer, CEO of 4SC AG. "In addition these findings, in particular the substantial efficacy in decreasing objective inflammation parameters and the very clean safety profile of vidofludimus, demonstrate the compound's broad potential as a therapeutic for various autoimmune diseases. We are particularly optimistic for our future development programme in inflammatory bowel disease (IBD) where vidofludimus has already shown encouraging efficacy in a phase II clinical trial. We are currently validating our development plan in IBD including dose finding studies also with higher doses of vidofludimus with regulatory agencies and potential partners to prepare the next development steps in this commercially attractive indication with high medical need."

The scientific poster with the detailed study results presented at the ACR meeting can be downloaded from 4SC's website at


Details of the Presentation:

Presentation #397, Title: "Efficacy, Safety and Pharmacokinetics of Vidofludimus, a Novel Oral Immunomodulator, in Patients with Active Rheumatoid Arthritis on Methotrexate Background Therapy: The COMPONENT Study"
Session date and time: Sunday, November 06, 2011, 10:15 AM (CST)
Session title: Rheumatoid Arthritis Treatment - Small Molecules, Biologics, Therapy I
Session type: Poster Session
Presenter: Stanislaw Sierakowski1, Bruno Dietrich2, Bernd Hentsch2 and Aldo Ammendola2
1Medical University Bialystok, Bialystok, Poland, 24SC AG, Planegg-Martinsried, Germany

About the COMPONENT study

The COMPONENT study is a randomised, double-blind, placebo-controlled, multi-centre, international Phase IIb study evaluating the efficacy of vidofludimus with methotrexate, compared to methotrexate alone, in rheumatoid arthritis (RA) patients. The primary endpoint of this study is the estimation of ACR20 at week 13, secondary endpoints are ACR50, ACR70, DAS28, safety parameters and pharmacokinetics. The trial enrolled 241 patients in two study arms across 28 sites in Poland, Romania, Bulgaria and Czech Republic. In the first study arm patients received 35 mg of vidofludimus given orally once-daily plus MTX, in the second study arm patients received placebo plus methotrexate. The study duration was 13 weeks and eligible patients must have had active RA, have received weekly doses of MTX (10-25 mg/week) for a minimum of 3 months prior to Day 1 dosing, and have received a stable MTX dose for at least 6 weeks prior to Day 1 dosing. More information about the COMPONENT study can be found on (Identifier NCT01010581).

About Vidofludimus

Vidofludimus is a novel, orally administered small molecule for the treatment of autoimmune disorders such as rheumatoid arthritis and inflammatory bowel disease. The therapeutic efficacy of vidofludimus is based on a dual principle. Vidofludimus inhibits the expression of selected pro-inflammatory cytokines, including interleukin-17 (IL-17A and IL-17F) and IFN-gamma that have crucial pathogenic roles in a variety of autoimmune diseases. Vidofludimus also inhibits dihydroorotate dehydrogenase (DHODH), a key enzyme of the pyrimidine biosynthesis, thereby halting the proliferation of activated T and B cells which are involved in the pathology of autoimmune disorders. Vidofludimus has completed a successful Phase IIa trial in inflammatory bowel disease. In addition, various preclinical models demonstrate the application options of vidofludimus in further autoimmune indications such as lupus, psoriasis, multiple sclerosis and transplant rejection.

About 4SC

4SC (ISIN DE0005753818) discovers and develops targeted small-molecule drugs for the treatment of diseases with a high unmet medical need in various autoimmune and cancer indications. These drugs are intended to provide patients with innovative treatment options that are more tolerable and efficacious than existing therapies, and provide a better quality of life. The company's balanced pipeline comprises promising products that are in various stages of clinical development. 4SC's aim is to generate future growth and enhance its enterprise value by entering into partnerships with leading pharmaceutical companies. Founded in 1997, 4SC currently has 94 employees and has been listed on the Prime Standard of the Frankfurt Stock Exchange since December 2005.

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Record changed: 2017-04-02


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