RPh201 development in US supported by Fast Track Designation by US Food and Drug Administration
NESS-ZIONA, Israel, Aug. 2, 2018 /PRNewswire/ -- Regenera Pharma, a biopharmaceutical company developing novel therapies for neurodegenerative diseases, today announced the enrollment of the first patient in a United States (US) Phase 3 study of RPh201 in NAION (nonarteritic anterior ischemic optic neuropathy, a type of stroke of the optic nerve), supported by Fast Track designation from the US Food and Drug Administration. In addition, Regenera is dosing patients in a Phase 2a clinical trial of RPh201 in Alzheimer's disease in Canada.
"Despite their growing prevalence in the aging population, neurodegenerative disorders remain largely untreated and patients are left with few – if any – treatment options today," said Jordan Rubinson, Chief Executive Officer of Regenera. "Based on our clinical and extensive preclinical data obtained to date, we believe that RPh201 has the potential to deliver meaningful benefit in multiple neurological disorders. Our data sets have been met with enthusiasm by investigators and regulatory agencies in the United States and Canada, as demonstrated by the US FDA granting RPh201 Fast Track designation, and we are excited to move efficiently through these two clinical studies."
The multicenter, double-blind, placebo-controlled Phase 3 trial of RPh201 will enroll 234 patients diagnosed with NAION at least one year prior to enrollment into the study. Patients will be randomized and receive twice-weekly injections of RPh201 or placebo over a six-month period. The primary endpoint of the study is the proportion of patients with visual acuity improving by 3 lines or more from baseline to Week 26. The study will be conducted in 12 leading centers across the United States, with the final readout expected in Q4 2019.
The Phase 3 study is based on the encouraging results from a Phase 2 study of RPh201 in NAION that was not powered for statistical significance. In the Phase 2 study, 36 percent of the group treated with RPh201 had vision improved by 3 lines or more, as compared to 12 percent in the placebo group.
"Several thousand people have irreversible visual loss from NAION each year in the United States, and the likelihood of improvement after the first 6 months is very low," said Dr. Leonard Levin of McGill University, and chair of the Phase 3 trial. "This new study will help determine the potential for RPh201 to become the first drug shown to restore vision in these patients."
In addition to the NAION study, Regenera is conducting a Phase 2a study in Canada of RPh201 in patients with moderate Alzheimer-related dementia. This multicenter, double-blind, placebo-controlled study is expected to enroll between 45 and 81 patients. Patients will be randomized 2:1 (drug to placebo) and will receive twice weekly subcutaneous injections over a six-month period. The study will measure cognitive changes from baseline to 6 months. Study participants are currently being dosed at five centers in Ontario and Quebec, with the readout expected by Q2 2019.
Regenera's clinical trials are funded by a 2017 Series B led by aMoon I, a leading Israeli life sciences fund. Regenera has received the first $10 million of the $20 million round, and funds are expected to support Regenera through completion of both clinical programs and additional preclinical studies in stroke and cognition.
"Our involvement with Regenera has given us recognition of the tremendous opportunity created by RPh201 in a wide array of neurological conditions, based on data obtained to date," said Dr. Yair Schindel M.D., Co-Founder & Managing Partner at aMoon. "Our continued investment is based on our steady confidence in Regenera's capital-efficient approach in obtaining further validation and approval in NAION, while evaluating potential in Alzheimer's disease, before moving into additional neurodegenerative indications."
RPh201 was discovered by Dr. Zadik Hazan and is a well-defined extract from gum mastic, which is purified, formulated and stabilized in a proprietary process. Regenera has broad, global intellectual property protection for RPh201 related to formulation, composition of matter and use.
aMoon is a leading Israeli life sciences investment fund founded by Marius Nacht and Dr. Yair Schindel. The team is passionate about building successful businesses and helping Israeli biopharma, digital health and medical technology companies realize their potential. It partners with management teams dedicated to accelerating cures for the world's most life-threatening conditions and to providing solutions for global healthcare's biggest cost drivers.