Familial Adenomatous Polyposis (FAP)
Familial Adenomatous Polyposis is a genetic condition that increases the lifetime risk of colon cancer from ~20% to nearly 100%. The disease presents as hundreds to thousands of polyps growing in the colon and rectum, usually starting in an individual’s mid-teens. If untreated, the polyps are nearly 100% certain to develop into colon or rectal cancer by age 40. No approved pharmaceutical treatments exist for FAP; the current standard of care is colon and bowel resection to avoid progression to colon cancer. Approximately 50,000 FAP patients exist in the US. This relatively small patient population designates FAP as a rare disease by the FDA.
While several publications demonstrate promise for rapamycin in gastrointestinal diseases in humans, the high doses required to counteract the acid degradation in the stomach result in many side effects. However, eRapa™ protects the active ingredient from the low pH environment of the stomach. In pre-clinical studies, eRapa™-dosed mice showed impressive disease suppression and life spans 5x greater than untreated mice in a mouse model for FAP.
- Phase 2 efficacy trial fully enrolled; primary endpoint data available by January 2023
- Proven effect in pre-clinical mouse model
- Targeted delivery of the active pharmaceutical ingredient to the lower intestines
- $3M grant from the Cancer Prevention and Research Institute of Texas (CPRIT) awarded for Phase 2 trial
- $16.9M grant from the Cancer Prevention and Research Institute of Texas (CPRIT) awarded for intended future registrational study
- Orphan Drug Designation received in FAP; label expansion opportunities in future
- Expedited 505(b)(2) pathway greenlighted by FDA
- ClinicalTrials.gov Identifier: NCT04230499
Expanded Access Policy
Emtora Biosciences is a life science company developing a novel formulation of rapamycin, eRapa™, for use in preventing the onset and recurrence of cancer. Under the 21st Century Cures Act, the manufacturer or distributor of investigational drugs for the treatment of one or more serious diseases or conditions shall make available its policy on how it evaluates and responds to requests submitted under section 561(b) of the Federal Food, Drug, and Cosmetic Act for provision of such a drug. The following is Emtora’s policy for evaluating and responding to requests for individual patient access outside the clinical trial process to investigational drugs that are intended to treat serious diseases.
Prior to regulatory approval by the U.S. Food and Drug Administration (FDA) and foreign regulatory authorities, patients gain access to investigational treatments, such as eRapa, by participating in clinical trials. Expanded access, also called compassionate use, enables some patients with serious or life-threatening diseases, who would not have otherwise met the enrollment criteria for the clinical trials in progress, to gain access to investigational treatments. Unlike the use of an investigational new drug in a clinical trial setting, the primary purpose of expanded access is to use the investigational drug for patient treatment purposes, rather than to gather data on safety, tolerability and effectiveness.
Emtora believes that investigational drugs should be studied in patients as part of clinical trials designed to obtain data on safety and efficacy that may be used to support approval of the product and subsequent wider accessibility to patients. As such, Emtora currently does not offer an expanded access program and does not accept expanded access requests. Patient access to eRapa outside of a controlled clinical trial may interfere with the conduct of our ongoing and future clinical trials, and may also disrupt the progress of our development program, which would, in turn, delay access to many patients in need.
Accordingly, while clinical trials are ongoing, we believe participation in our clinical trials is the only safe and appropriate way to access eRapa.
If you have questions about Emtora’s expanded access policy, please contact Emtora at info@emtorabio.com. See this link for more information about Emtora’s trial in FAP. In accordance with the 21ST Century Cures Act, Emtora may revise this policy at any time.
