Beset by controversy, Turing unveils its first in-house drug candidate

Martin Shkreli

Turing Pharmaceuticals' move to jack up the price of an old drug has made it world-famous, emblematic of either the industry's moral bankruptcy or the triumph of creative entrepreneurialism, depending on whom you ask. Defending the Daraprim decision, Turing has again and again said its eventual profits will fund research into novel treatments for serious diseases, and now the world is watching to see whether the company and its brash CEO follow through.

Turing has now revealed its first post-Daraprim R&D project: an early-stage therapy for rare and severe brain disorders. Called TUR-004, Turing's candidate is designed to serve as an add-on treatment for easing seizures tied to epileptic encephalopathies, a group of syndromes that crop up in early childhood and are often fatal.

Turing said it submitted an investigational new drug application for TUR-004 to the FDA last month, completing the first step on the path to clinical study. If and when the FDA clears that application, Turing will start a placebo-controlled Phase I study to determine the safety and activity of TUR-004 in healthy patients, the company said.

A biotech filing an IND is not generally a notable occurrence, but Turing is not like other biotech companies. Led by CEO Martin Shkreli, Turing has been the subject of unwavering scrutiny and criticism since acquiring the 62-year-old toxoplasmosis treatment Daraprim and raising its price about 50-fold. Shkreli claims that the resulting revenue will enable drug development that would otherwise have gone undone, in toxoplasmosis and other diseases.

His seriousness, like his ethics, has been widely questioned. And all of Turing's baggage puts an uncommon amount of attention on a very early-stage drug program, as Shkreli's critics look for signs that the company is a profiteering scam.

In the meantime, Turing says it's piecing together a wide pipeline of novel therapies, and "TUR-004 is the first of multiple investigational new drug applications we expect to file in the next 12 months as a result of our focused research and development programs," Shkreli said in a statement. The company has also disclosed an early-stage ketamine product designed to treat mood disorders but has otherwise kept details on its pipeline under wraps.

- read the statement