Shkreli started his hedge-fund career in 2000 at Cramer, Berkowitz. He is best known to biotech investors for a 2011 hostile takeover attempt of Amag Pharmaceuticals, done for the specific purpose of firing the company’s management and stopping a proposed merger with Allos Therapeutics. When the merger plans stopped, so did Shkreli. He also filed a petition with the FDA arguing that an inhaled insulin from the Mannkind, the biotech firm run by billionaire Al Mann, should not be approved. It was not. At the time, he was short Mannkind shares. He publicly criticized Pfizer‘s decision to replace chief executive Jeffrey Kindler with Ian Read, arguing the company needed a fresh perspective.
Citizens for Responsibility and Ethics In Washington, a Beltway group that targets special interests, has accused Shkreli of “trying to foist his medical views on the FDA.”
Shkreli tired of trying to reshape drug development from the outside, and found an opportunity in a rare disease called focal segmental glomerulosclerosis (FSGS), a leading cause of kidney failure that afflicts 50,000 people in the U.S. Most are diagnosed as children.
There was scientific reason to believe that a drug that combined the action of two different kinds of hypertension medicines could help slow the build-up of protein in the urine and the damage to the kidneys from the disease. Shkreli knew that Ligand Pharmaceuticals and Bristol-Myers Squibb had been working on such a drug and licensed it.
“Martin and Retrophin approached me, which is really quite unique,” says Howard Trachtman, the director of pediatric nephrology at NYU Langone Medical Center and the lead investigator of Retrophin’s planned trial. “I’m hoping they’ll be able to pull this off.
“It’s really remarkable that they’re willing to look at FSGS as their first test. But I can speak to personal experience with other drug companies where they have been gunshy about venturing into this disease. It’s hard to succeed and we need new ideas and I think that’s pretty impressive.”
In September Retrophin named Stephen Aselage, a marketing executive from BioMarin, as its chief executive officer. But Shkreli has instead retained the CEO. Aselage remains involved with the company, Shkreli says.
So far Retrophin has raised $4 million in a series A round. Shkreli says he can’t disclose how much the company has in the bank but says the company is largely funded on an “as-it-goes” basis.