FDA clears startup's virtual trial for drug against multiple sclerosis

Transparency Life Sciences picked up a win for its bet on open innovation in transforming drug development. The FDA cleared the developer's IND application to study a generic hypertension drug for a new potential use in patients with multiple sclerosis, after the startup tapped crowdsourced input from experts and patients on aspects of the clinical trial.

The study not only used New York-based Transparency's web-based Protocol Builder software to gather ideas on the design of the Phase II effort, but the trial will also be partially virtual. After patients' initial visits to trial sites, the planned 12-month study is expected to use telemonitoring technology from Advanced Monitored Caregiving and other partners to track participants until their final checkups, COO Marc Foster explained to FierceBiotechIT.

With these outside-the-box strategies, Transparency aims to drive down the cost of clinical trials by at least 50%. This is a big goal and one not easily achieved industry-wide, and at first blush, the startup's bold idea might draw some healthy skepticism. As published in a Nature Reviews Drug Discovery article in March, the number of FDA approvals per billion dollars in research money spent has been steadily declining over the past 60 years or so. The authors dubbed this "Eroom's Law," which is Moore's Law spelled backwards. Transparency's Foster believes there is hope for reversing the troubling trend.

"I think it's doable. It takes a fresh approach. It takes a bold approach," Foster said in a phone interview. "Just tinkering with the status quo is not working, and it's really resulted in an unsustainable clinical development model that has translated into escalating costs." 

How does Transparency expect to drastically reduce the cost of clinical research? For starters, the company is crowdsourcing patients and healthcare experts to augment its reliance on internal thought leaders to design trial protocols. Its hope is to show that this is an efficient and effective approach to protocol design, which can take substantial time and money to complete.

In the MS study, the company wants to trial a new use of a widely prescribed generic heart drug in the ACE inhibitor lisinopril, which has a well-established safety profile that allows the startup to begin its program with a midstage study. (A company co-founder documented the generic drug's benefits for MS in animal studies, resulting in a new method of use patent filing.) Foster also aims to get discounted supplies of the drug from generics companies. Overall, he sees an opportunity to pick up drugs that are ready for Phase II from around the industry, using the company's open innovation approach to reduce development costs of the molecules, with plans to license successful candidates to partners after midstage studies. The partners would foot the hefty bills for Phase III trials.

The company has found a lead investigator at Stanford who treats many MS patients, and Foster sees an opportunity to cut recruitment costs by enrolling all the estimated 150 to 180 study participants from the San Francisco Bay Area. Recruitment is notoriously expensive, and drawing from the investigator's large pool of patients for the study could significantly reduce that expense.

The next bucket of savings is expected to come from reduced clinical monitoring costs because participants will self-report their status from their homes with mobile devices for most of the study, reducing the cost of traveling to clinics with high costs of doing business, Foster says. Transparency is considering a GPS-enabled system to provide remote monitoring of MS patients' movements, as their neurological disorder causes progressive disability.

Transparency is taking a hybrid approach to virtual clinical development that might just work. We'll keep track of its progress.

- here's the release