OrphoMed Initiates U.S. Phase 2 Adaptive Design Study of ORP-101 for Treatment of IBS-D
—Interim Assessment Anticipated in Second Half of 2020—
January 7, 2020
SAN FRANCISCO, CA– OrphoMed, Inc., a clinical-stage biopharmaceutical company developing first-in-class dimer therapeutics, today announced initiation of its PREVAIL trial, a Phase 2 response-adaptive clinical study of ORP-101, a peripherally-acting buprenorphine dimer for the treatment of irritable bowel syndrome with diarrhea (IBS-D). The study is designed to assess safety and efficacy of ORP-101 compared to placebo and includes two pre-defined interim analyses, both of which are anticipated in the second half of 2020.
Principal Investigator Dr. Anthony Lembo stated, “ORP-101 has the potential to become an important therapy for the treatment of IBS-D. It could have a significant impact on patients’ health and quality of life.”
ORP-101 is a new chemical entity designed with intent to create a large stable molecule and to confer partial μ agonist and full κ-antagonist receptor pharmacology.
- ORP-101 has been shown in preclinical studies to not cross the blood-brain barrier.
- ORP-101 successfully completed Phase 1 and was granted FDA Fast Track designation in 2018. Regulatory path to NDA has been agreed with the FDA.
- ORP-101 Phase 2 currently enrolling, utilizing adaptive 3-month treatment design powered for Phase 3 predictive outcomes.
About the PREVAIL Trial
The PREVAIL study, now enrolling patients, 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.
The study includes two interim analyses at enrollment of 200 and 260 patients, respectively, conducted by a third party to evaluate (1) dose response and patient allocation using a response-adaptive randomization and (2) stopping accrual early for sufficient efficacy or futility. OrphoMed expects to conduct the interim analyses in the second half of 2020. Topline results are expected in the first half of 2021.
IBS-D is a multifactorial symptoms-based disorder marked by recurrent abdominal pain or discomfort and altered bowel function. It affects between 10 and 20 percent of people in the developed world, about one third of whom have IBS associated with diarrhea. IBS-D is more prevalent in women than men (14% vs. 9%). About 57% of patients experienced symptoms daily, 25% weekly and 14% monthly. The disease affects daily living, work and leisure. It costs about 40% more to take care of IBS patients than the population in general. In general, IBS patients account for 20% of secondary care burden of GI physician outpatient work. The FDA has recently stated there is a great medical need for new therapies for IBS.
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. For more information please visit www.orphomed.com.
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