SAN FRANCISCO--(BUSINESS WIRE)--OrphoMed, Inc., a clinical-stage biopharmaceutical company developing first-in-class dimer therapeutics, announced today that its ORP-101 molecule successfully cleared its first interim futility analysis in a Phase 2 clinical trial for treatment of irritable bowel syndrome with diarrhea (IBS-D).
“We are pleased to have passed this important milestone for our lead compound, ORP-101, and for the company,” said Gary Phillips, M.D., President and Chief Executive Officer of OrphoMed. “We continue to enroll patients in this trial and await results of the next planned interim futility analysis as we evaluate strategic options in our continued effort to improve the lives of patients with IBS-D.”
OrphoMed reached the enrollment milestone despite the challenges resulting from the COVID-19 pandemic. Dr. Phillips noted the continued ability to recruit patients, including IBS-D patients without gallbladders, underscores the critical need for an IBS-D treatment that is both effective and safe in all patients. Approximately 30% of patients enrolled to date are post-cholecystectomy IBS-D patients.
The next major trial milestone will be a second interim analysis triggered upon total enrollment reaching 260 patients, expected in late Q1 2021. The company anticipates topline results from this Phase 2 trial in the second half of 2021.
ORP-101 is a new chemical entity designed to confer peripheral partial μ agonist and full κ receptor antagonist activity, enabling treatment of both IBS-D symptoms of dysmotility and pain without the risk of sphincter of Oddi spasm or pancreatitis.
- ORP-101 has been shown in preclinical studies to not cross the blood-brain barrier.
- ORP-101 successfully completed Phase 1 clinical trials and was granted FDA Fast Track designation in 2018. Regulatory path to NDA has been agreed with the FDA.
- ORP-101 Phase 2 clinical trial is currently enrolling, utilizing adaptive 3-month treatment design powered for Phase 3 predictive outcomes.
About the PREVAIL Trial
The PREVAIL trial, managed by PPD, a leading global contract research organization, is a multi-center, double-blind, placebo-controlled, response-adaptive randomization study to evaluate the safety and efficacy of ORP-101 in approximately 320 patients with IBS-D. Study participants will be randomized to receive one of two different doses of ORP-101 tablets or placebo for 12 weeks. The primary endpoint is percentage of patients who are composite responders, based on improvements from baseline in daily worst abdominal pain and daily stool consistency scores. Secondary endpoints include percentage of participants who are responders in the following assessments: daily worst abdominal pain, daily stool consistency, IBS global symptom, IBS adequate relief, modified composite response, daily abdominal discomfort, daily abdominal bloating, number of bowel movements per day, and number of bowel incontinence-free days. For more information about the study, please see www.orphomed.com.
About OrphoMed, Inc.
OrphoMed is a clinical-stage biopharmaceutical company focused on the development of novel first-in-class dimer therapeutics. Leveraging its proprietary dimer platform technology engineered by founder Nikhilesh Singh, Ph.D., OrphoMed is advancing ORP-101 and a pipeline of additional dimer conjugates. In May 2017, OrphoMed completed a $39 million Series A financing with New Enterprise Associates, Takeda Ventures, Pappas Capital and Relativity Healthcare. OrphoMed is currently evaluating all strategic options, including advancing the compound through Phase 3 and NDA submission. For more information, please visit www.orphomed.com.
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