GO is a condition suffered by a significant number of Graves' Disease patients in which inflammation and a build up of tissue behind the eye cause the eyeball to protrude from the socket. This changes the patient's appearance and results in significant morbidity; including corneal ulceration, pain, optic neuropathy and double vision among other serious effects. The work of Terry Smith, MD and Ray Douglas, MD (University of Michigan), Principal Investigators for this study, and data from a growing number of other GO researchers, suggests that the IGF-1 receptor in orbital tissue plays an important role in regulating the autoimmune response that underlies GO.
The study is a double-masked, randomized, placebocontrolled trial intended to assess the efficacy and safety of RV001 for the treatment of patients with moderate to severe GO. Patients in the study receive 8 infusions of either saline or RV001 over a period of 24 weeks, after which the primary efficacy assessment is made. RVDC expects that this portion of the study will be completed early in the second quarter of 2016 and that the top-line results will be available shortly thereafter.
“This trial of RV001 sponsored by RVDC represents the largest and one of few placebo controlled studies ever conducted in GO,” said Guido Magni, Chief Medical Officer of RVDC. “It should enable us to test the hypothesis that this condition is regulated by autoantibodies targeting the IGF-1 and TSH receptors and that the impact of these can be attenuated by RV001, as the work of Drs. Smith and Douglas would suggest. More importantly, a positive result would represent a major step forward in the prospects for an efficacious medical treatment for these patients where none exists today.” Recognizing the importance of this condition and the lack of treatment options for patients, the FDA recently granted Fast Track designation for RV001 for GO. The
drug had previously been granted Orphan Designation by the Agency. RVDC anticipates filing an Orphan Drug Application in the EU in Q3 of this year and an application for Breakthrough Designation in the US in Q2 of 2016 if merited by the trial's outcome.
About River Vision Development Corp.
River Vision Development Corp. was founded by Narrow River Management, LP (Narrow River) for the sole purpose of developing teprotumumab for GO and other potential indications. The Company is managed under contract with Narrow River, which provides leadership expertise for special purpose drug development companies. The Company's investors include SROne, Lundbeckfond Ventures and Narrow River Management, LP.