San Antonio — Rapamycin Holdings has a new CEO, is raising a $3.5 million Series B round of funding, and plans to submit an investigational new drug filing to the FDA this year. If all goes well, the small biotech may start its first in-human clinical trials as early as later this year.
“The company is at an inflection point,” says Dan Hargrove, the company’s new CEO, who was hired in July.
That’s why Hargrove, an attorney by training, says he was asked to take over. He is coming from a clinical research organization he co-founded in San Antonio, Cancer Insight, which aids drug developers with regulatory affairs in addition to clinical trial work. Rapamycin was a client.
Former CEO and Rapamycin co-founder Randy Goldsmith is remaining with the company as a member of its board of directors.
Now, the company is waiting for additional data on how well its drug lasts on the shelf before it applies to the FDA for the go-ahead to start clinical trials. If granted approval to start a Phase 1 study, the plan is to enroll 40 prostate cancer patients in a trial testing four different doses of Rapamycin’s drug, known as eRapa.
Hargrove expects a $3.5 million Series B funding, which it launched last week, will support the company through a trial that may last as long as 21 months. It may simultaneously run a Phase 2a study, the company said in a news release from July. Rapamycin recently raised a $2 million convertible note from local investors.
The company takes its name from the decades-old drug rapamycin, a natural product discovered 44 years ago that has been best known as the Pfizer (NYSE: PFE) immunosuppressant drug sirolimus (Rapamune), which is used for organ transplants. That drug has since gained an additional FDA approval in a rare lung disease. There are other derivatives (or analogues) of rapamycin that are structurally similar but not the same. That’s true for Rapamycin Holdings’ formulation, eRapa (which stands for encapsulated rapamycin), which has been made with a coating that would allow the drug to pass through the stomach before being released, chief science officer Dana Vaughn told Xconomy last year.
Rapamycin Holdings is targeting prostate cancer with its first clinical trial in part because it is within the expertise of its principal investigator, Ian Thompson. Thompson is a former administrator and researcher at the University of Texas Health Science Center at San Antonio, who recently left for a position at the nearby Santa Rosa Hospital. The goal of the trial will be to show the drug has no toxicity. Michael Liss, another urologist at the university, will also work on the trial.
The company’s drug aims to be a buzzkill for overactive cells, such as cancer cells, which have been ramped up to divide and multiply at a faster rate than normal cells, Vaughn told Xconomy. The drug is meant to inhibit a signaling molecule that tells the cancer to multiply.
The secondary goal of the investigation is testing whether the drug may have an immunological affect on the cancer. That is close to the heart of Rapamycin’s chief medical officer, George Peoples, a long-time cancer surgeon and researcher who has developed multiple experimental cancer vaccines. Peoples is the other co-founder of Cancer Insight, the contract researcher that Hargrove came from, and he will also work on the clinical trial.
Rapamycin’s drug might have applications beyond cancer. Mouse studies suggested the drug could also affect cognitive abilities. Based on that data, the FDA recommended in its meetings with Rapamycin that company also study whether the drug improves patients’ cognition, Hargrove says.
The American Cancer Society estimates that more than 161,000 new cases of prostate cancer will be diagnosed this year. Approximately 26,700 patients will die from the disease. The standard of care for early prostate cancer cases is watching to see if the cancer progresses—known as active surveillance, he says. Surgery or radiation therapy can have unpleasant consequences, such as incontinence and sexual dysfunction, he says.
Thompson was interested in Rapamycin because he had been seeking a drug to treat patients with early stages of the cancer, Hargrove says.
Notably, Rapamycin already sells its formulation to academic research institutions in the U.S. and elsewhere, which use it as part of investigations into a variety age-related conditions and diseases in animals, Hargrove says. The company brings in about $250,000 in annual revenue from that, which he says he expects will grow.