Androclus Therapeutics Reports Completion Of A Phase 2 Clinical Trial Of AT-001 (DnaJP1) In Patients With Rheumatoid Arthritis.

SAN DIEGO & MILAN, Italy -- Androclus Therapeutics announced the completion of a Phase II clinical trial of AT-001 (dnaJP1), a novel orally administered therapeutic candidate for the treatment of rheumatoid arthritis (RA).

The double-blind, placebo-controlled trial was conducted by Salvatore Albani, M.D., Ph.D., Professor of Medicine and Pediatrics at the University of California, San Diego (UCSD) School of Medicine. The trial enrolled 160 patients with RA in 11 centers nationwide. Patients in the AT-001 arm of the trial received 25 mg of AT-001 daily by mouth for six months.

AT-001 was found to be safe and well tolerated. When compared with a placebo, patients treated with AT-001 experienced an improvement in the signs and symptoms of RA such as swollen joints, tenderness, pain and mobility. Primary efficacy endpoint, of the area under the curve (AUC) of ACR20 response rates at Days 112, 140 and 168 reached a p value of 0.099. A post-hoc analysis demonstrated statistical significance for AUC of ACR20 response rates at Days 112, 140, 168 and 196 (p=0.039).

The improvement in the signs and symptoms of RA was particularly significant at the follow-up visit on Day 196, indicating a lasting effect of the drug. Intention-to-treat ACR response rates on Day 196 for patients treated with AT-001 were ACR20 in the 35-45% range (p=0.004 vs. placebo), ACR50 in the 20-30% range (p=0.04 vs. placebo), ACR70 in the 10-15% range (p=0.1 vs. placebo). Of the patients treated with AT-001 who completed the trial, Day 196 responses were ACR20 in the 50-55% range, ACR50 in the 30-40% range, ACR70 in the 15-20% range.

The immunological analysis demonstrated an immune deviation of a T cell function in patients treated with AT-001, from inflammatory to regulatory. Peptide-induced T cell production of TNF-alpha between Day 0 and Day 168 decreased significantly in the AT-001 arm (p=0.003) but not in the placebo arm. Similarly, there was a strong trend of increased production of IL-10 in the AT-001 arm (p=0.11) but not in the placebo arm.

"In essence, we re-educated the T cells to be tolerant of the dnaJP1 amino acid sequence, which would usually trigger inflammation in rheumatoid arthritis patients," Prof. Albani said. "Our drug leaves the patient's natural immune responses intact. This differs profoundly from what is currently available to patients."

"We are encouraged by the results of this Phase II trial, as a validation of Androclus Therapeutics technology platform, epitope-specific immunotherapy. We look forward to further developing the technology and to bringing novel, selective, orally-administered immunomodulatory drugs to millions of patients suffering from autoimmune diseases such as RA, multiple sclerosis and inflammatory bowel disease," said Dr. Igor Bilinsky, chief executive officer of Androclus.

The technology platform was licensed from UCSD to Androclus through the UCSD technology transfer office. The Phase II clinical trial was supported by the National Institutes of Health.

About RA

RA is a chronic, progressive, debilitating autoimmune inflammatory disease affecting approximately one percent of the U.S. population, or more than 2 million people. The disease strikes joints, such as the hands, feet or knees and can also have systemic effects. It is an incurable disease, with most therapies focusing on symptom relief and on delaying disease progression.

About AT-001

AT-001 (dnaJP1) is a short synthetic peptide derived from a heat shock protein dnaJ. The mechanism of action relies on selectively inducing an immune deviation of a T cell function from inflammatory to regulatory, thus inhibiting disease-related inflammation without affecting patient's immunity to infectious diseases or cancer. AT-001 was discovered at UCSD by Salvatore Albani, M.D., Ph.D., Professor of Medicine and Pediatrics and coworkers. Androclus signed an agreement with a large biotech for further development and commercialization of AT-001.

About Androclus Therapeutics

Androclus Therapeutics is a private clinical stage biotechnology company discovering and developing novel therapeutics for the treatment of autoimmune and inflammatory diseases, including rheumatoid arthritis, multiple sclerosis and inflammatory bowel disease.

Compared to existing biologics for the treatment of autoimmune diseases, Androclus' drug candidates are expected to offer increased selectivity in targeting autoimmune inflammation, improved tolerability and safety profile, and a more convenient oral route of administration. For more information, visit Androclus' website at www.androclus.com.