Sphincter of Oddi Dysfunction

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 passed a second interim futility analysis triggered by enrollment of 260 patients in its Phase 2 clinical trial for treatment of irritable bowel syndrome with diarrhea (IBS-D). “We are very pleased to have successfully cleared this important milestone in the development of ORP-101,” said Gary Phillips, M.D., President and Chief Executive Officer of OrphoMed. “We will now work diligently to complete the trial and to advance a promising new treatment for people suffering from IBS-D.” Dr. Phillips also noted that approximately 25% of IBS-D patients enrolled to date are post-cholecystectomy patients. OrphoMed anticipates topline results from this Phase 2 trial in the second half of 2021. About ORP-101 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 ORP-101 Phase 2 Trial The trial 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 . About OrphoMed, Inc. OrphoMed is a clinical-stage biopharmaceutical company focused on the development of innovative and de-risked 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 . Forward-Looking Statements This press release contains “forward-looking statements,” which reflect our current views and expectations with respect to future outcomes or events. When used in this press release, the words “could,” “believe,” “anticipate,” “intend,” “estimate,” “expect,” “project” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain such identifying words. Because these statements are based on current estimates and assumptions, they are inherently subject to risks that could cause the actual outcomes to differ materially from what we currently expect. These forward-looking statements are subject to numerous risks and uncertainties, including, among others, the facts that we are substantially dependent on our ability to successfully develop and commercialize our products; the commercial adoption of our products and any other product candidates we develop will depend on the degree of their market acceptance; we have only limited assets and will need to raise additional capital before we can expect to generate revenue or become profitable; and we have never generated any revenue and may never be profitable. Forward-looking statements in this presentation apply only as of the date made, and we undertake no obligation to update or revise any forward-looking statements to reflect subsequent events or circumstances. View source version on businesswire.com:

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. About ORP-101 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 . 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 . Forward-Looking Statements This press release contains “forward-looking statements,” which reflect our current views and expectations with respect to future outcomes or events. When used in this press release, the words “could,” “believe,” “anticipate,” “intend,” “estimate,” “expect,” “project” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain such identifying words. Because these statements are based on current estimates and assumptions, they are inherently subject to risks that could cause the actual outcomes to differ materially from what we currently expect. These forward-looking statements are subject to numerous risks and uncertainties, including, among others, the facts that we are substantially dependent on our ability to successfully develop and commercialize our products; the commercial adoption of our products and any other product candidates we develop will depend on the degree of their market acceptance; we have only limited assets and will need to raise additional capital before we can expect to generate revenue or become profitable; and we have never generated any revenue and may never be profitable. Forward-looking statements in this presentation apply only as of the date made, and we undertake no obligation to update or revise any forward-looking statements to reflect subsequent events or circumstances.