Vertex achieves 2 main goals of late-stage trial for rheumatoid arthritis

by Symptom Advice on September 16, 2011

6th Sep 2011, 1:15 pm by Brad Lemaire

Vertex Pharmaceuticals (NASDAQ:VRTX) reported Tuesday its experimental drug VX-509 for the treatment of rheumatoid arthritis met its two primary endpoints during a late-stage, 12-week trial.

According to results, VX-509 resulted in “substantial and statistically significant” improvements in rheumatoid arthritis activity, compared to a placebo during the phase 2a trial.

Rheumatoid arthritis is a chronic inflammatory disease that affects about 1.5 million adults in the U.S. the disease causes destruction of joint cartilage and erosion of adjacent bone resulting in deformity, loss of function, disability and the need for joint replacement.

About 80% of people with the disease become disabled within 20 years of diagnosis.

The two main goals of the study were a statistically significant improvement in the proportion of people who achieved at least a 20% improvement in the signs and symptoms of the disease, as well a similar improvement from baseline in a certain score measuring disease activity.

The company said it plans to execute a six month phase 2b study based on the results.

“These early results are encouraging and provide strong support that the selective targeting of JAK3 by VX-509 may represent a new approach to the treatment of rheumatoid arthritis and other autoimmune and inflammatory diseases,” Vertex’s chief scientific officer, Peter Mueller, said in a press release.

The VX-509 drug is taken orally, and is specifically designed to work by blocking a family of enzymes known as Janus Kinases, or JAK, known to cause chronic inflammation, and in the case of rheumatoid arthritis, irreversible damage to cartilage and bones.

The  204 person, placebo-controlled study evaluated four dose levels of VX-509, which were given twice daily for 12 weeks to RA patients, with about 40 clinical trial sites taking part in the trial in the US and Europe.

Data from the 150-milligram dose showed 66% of patients achieved a 20% improvement, while the placebo showed a response of 29%. the company also tested doses of 25-mg, 50-mg and 100-milligrams.

The study also evaluated patients who experienced clinical remission, defined as a patient with a Disease Activity Score (DAS28) of less than 2.6, at week 12. about 35% and 37% of people achieved DAS28 remission in the 100 mg and 150 mg treatment groups, respectively, compared to 7% of people in the placebo arm, the company said.

The next phase 2b trial for the drug is expected to test once-daily and twice-daily doses of VX-509 in combination with methotrexate, a commonly prescribed anti-rheumatic drug for rheumatoid arthritis.

Vertex develops and commercializes small molecule drugs for the treatment of diseases. It is engaged in phase-one clinical trials for a host of drug candidates, which include drugs for the treatment of cystic fibrosis and influenza, epilepsy and other life-threatening diseases.

The Cambridge, Massachusetts-based pharmaceutical company’s shares rose 16 cents, or 0.36%, to trade at $44.75 Tuesday afternoon on the Nasdaq.

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