Shire to Lead Development Advancement of RP Therapy

October 27, 2014

The development program for a new drug with the potential to treat a common form of autosomal dominant retinitis pigmentosa (adRP) has received a boost with Shire’s acquisition of BIKAM Pharmaceuticals, the original developer of the experimental treatment.

“Shire’s leadership in global specialty pharmaceuticals and the orphan disease market, along with its strong clinical development expertise and resources, will enhance the potential for the treatment’s advancement into and through human studies,” says Patricia Zilliox, Ph.D., chief drug development officer at the Foundation Fighting Blindness Clinical Research Institute (FFB CRI). “The acquisition provides a better opportunity to move the therapy through the clinical and regulatory process in order to determine if it is safe and effective for the people who desperately need treatment.”

Known as a pharmacological chaperone, the new compound is hoped to help preserve photoreceptor cell function, and potentially vision, in people with adRP caused by Class II mutations in the gene rhodopsin (RHO). Researchers believe the drug may work by binding to the defective, misfolded protein, enabling normal folding, transport, and function within the patient’s rod photoreceptor cell. Without treatment, the proteins aggregate and fail to translocate to the plasma membrane of the rod photoreceptor cells, their normal site of action, which is believed to lead to progressive photoreceptor degeneration and eventual vision loss.

 “We are very excited about the strong expertise and resources that are now behind this exciting therapy,” says William T. Schmidt, chief executive officer at FFB. “Moving treatments into human studies always presents significant business and regulatory challenges, and with Shire’s leadership and know how, this potential treatment has an improved chance to make it into the marketplace. We look forward to continuing our supportive role.”