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Emtora Nets $16.9M Grant to Advance Drug for Genetic Condition That Increases Colon Cancer Risk

NEW YORK – Emtora Biosciences said on Friday that it has received a $16.9 million grant from the Cancer Prevention & Research Institute of Texas (CPRIT) to advance its investigational treatment for familial adenomatous polyposis (FAP), a genetic condition that commonly precedes gastrointestinal cancers if left untreated.

FAP is caused by mutations in the autosomal dominant APC gene and predisposes patients to developing hundreds of thousands of polyps that can later progress to colorectal cancer. As of now, the condition has no treatments other than prophylactically removing patients' colons and rectums.

Emtora will use the grant from CPRIT to develop a therapy for FAP — dubbed eRapa — involving a capsule of rapamycin, an mTOR inhibitor that is sold under a variety of brand names for non-cancer conditions, including preventing organ transplant rejection.

San Antonio-based Emtora previously received a $3 million grant from CPRIT in 2019, which it is currently using to complete a Phase II clinical trial of eRapa in 30 patients with FAP. The firm will share data in early 2023 on the trial's primary endpoints including tolerability, recommended dose, and change in polyp burden. Subsequently, the firm plans to use the new CPRIT grant to complete a registrational Phase III trial of the treatment and submit the data to the US Food and Drug Administration via an abbreviated new drug application.

The $16.9 million award from CPRIT also includes a matching element, in which external sources will match 50 percent of funds. Emtora also is hoping to raise additional funds and is exploring partnering options.

Beyond the FAP trial, Emtora is conducting a Phase II study of eRapa in patients with bladder cancer, which it expects to read out in 2024.