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.
Over 80,000 new cases of bladder cancer are diagnosed every year, making it the sixth most common cancer in the US. Approximately 70% of new cases are classified as non-muscle invasive, where cancer is found in the tissue that lines the inner surface of the bladder. Bladder cancer is more common in men than women; approximately 75% of new cases occur in males.
The standard treatment for NMIBC is endoscopic biopsy of the tumor followed by instillation of chemotherapeutic agents or immunotherapeutic bacillus Calmette-Guérin (BCG) directly into the bladder. Although these agents are helpful in preventing or delaying disease relapse, recurrence rates remain high (up to 60%). Because of the need for lifelong monitoring and repeated procedures, it is estimated that bladder cancer has the highest cost per patient from diagnosis to death. We propose mTOR inhibition for secondary prevention of NMIBC using eRapa™, aiming to reduce disease relapse.
Prostate cancer is the most common cancer in men. Currently ~3M men are living with prostate cancer in the US and prostate cancer deaths exceed 29,000 in the US annually. Around 165,000 men in the US are diagnosed with prostate cancer each year. Approximately 80% of newly diagnosed cases are early stage, and men with early stage cancers (Gleason <7) under active surveillance have no effective therapies to slow or prevent cancer progression. A majority of men elect premature surgical and/or radiation treatment regardless of the disease severity. Up to 90% of treated patients experience erectile dysfunction, impotency, bowel distress, and other side effects. We postulate that treating low grade prostate cancer patients with low doses of eRapa will prevent the progression to prostate cancer.
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 email@example.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.