San Antonio—Emtora Biosciences, a San Antonio biotech startup formerly known as Rapamycin Holdings, is planning to test its drug, eRapa, as a treatment for a rare genetic disease known as Familial Adenomatous Polyposis (FAP).
Emtora plans to begin a Phase 2a trial in late 2019 for the condition, which affects 60,000 people in the United States, the company said in a news release. For those with the condition, which is typically inherited, hundreds of polyps begin forming in the colon and rectum of a patient in his or her teenage years. Those growths are benign when they form, but can become cancerous as patients age, typically by their 40s or 50s, according to the Mayo Clinic. The reported incidence is somewhere between one in 7,000 to one in 22,000 people, according to the US National Library of Medicine.
Emtora plans to test the drug’s ability to reduce or prevent the formation of polyps in patients (focusing on the size and number of polyps) during the Phase 2a trial, CEO Carole Spangler Vaughn wrote in an email to Xconomy. It has preliminary results from a preclinical study in mice, during which the formation of polyps diminished with an increased dose of the drug, the company says. Emtora’s eRapa has received an orphan drug designation from the FDA, the company said in the April 30 news release, which means it could benefit from certain incentives if it gains regulatory approval—though that possible outcome is still quite far away. There currently isn’t a therapeutic treatment, the company said. The current standard of care is to cut away parts of the colon or bowels, which reduces a patient’s quality of life, Vaughn wrote.
Emtora changed its name from Rapamycin Holdings in February to tie the company’s branding to the mechanism of action that eRapa targets, known in short as mTOR, a protein kinase that has been observed to regulate cell proliferation and growth. The company hired Vaughn to take over as CEO for Dan Hargrove, who had spent about 18 months in the chief executive role. Emtora was founded in 2012 by a group of local scientists and entrepreneurs.
Fundraising will be the company’s next step. It is preparing for a Series B funding round and hopes to raise $7 million, though it may reduce that amount if Emtora receives a $3 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT), for which it recently applied, Vaughn wrote to Xconomy. Emtora recently sold $2 million in convertible notes, Vaughn said in a news release. It has raised more than $8.5 million in total funding to date, Vaughn wrote in the e-mail.
Emtora’s eRapa is a new formulation of a drug called rapamycin, which was initially developed as an immunosuppressant to prevent organ transplant rejection. While at a high dose the drug can suppress the body’s immune system, the company believes that a low dose might rejuvenate it. Emtora began a Phase 1b trial last year of eRapa’s use in prostate cancer.