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.

As Reported by Patrick Crutcher

Repligen Corporation (NASDAQ: RGEN) is another attractive biotech that has revenues, cash and pending data in early 2011. with pending Phase 2 and Phase 3 data, Repligen seems set for a transformative 2011.

Specifically, they have 2 important clinical catalysts in Q1 2011: Phase 3 data for RG1068 (pancreatic imaging agent) and Phase 2b data for their bipolar drug (RG2417). Both of these catalysts have the potential to take RGEN to new levels in 2011.

Phase 2a results demonstrated a statistically significant reduction in the symptoms of depression in patients receiving RG2417 (vs. placebo) on the MADRS and on the CGI-BP-C scale over the 6-week course of the study. RG2417 was safe and well tolerated. Repligen has exclusively licensed this patent from McLean Hospital, the largest psychiatric facility of Harvard Medical School. RGEN intends on seeking a partner, since this drug has a potential $2 billion market. Positive results from this trial could result in significant appreciation of their value. Enrollment is complete in this trial and most clinical work should be done by the end of the year. Results are expected in early Q1 2011.another catalyst to look forward to in Q1 2011 is the re-analysis of images from their Phase 3 results RG1068 in Pancreatic Imaging. RG1068 is a synthetic version of human secretin, a natural gastrointestinal hormone involved in the process of digestion. RG1068 was granted orphan drug status and fast track designation by the FDA. The use of RG1068 in combination with a non-invasive procedure such as MRI can improve the detection of abnormalities and increase the diagnostic quality of the MRI image of the pancreas. last year, they reported results from a large, multicenter study that showed significant advantages of using RG1068 with MRI over endoscopy(ERCP) alone, however, the study did not show statistical significance. this was in large part because of errors made by the contract research organization(CRO) during the analysis of trial data, namely, deviating from the trial protocol. Fortunately for Repligen, the FDA and EMA recognized these issues and agreed to have the Phase 3 re-read of the data.