Startup biotech charges into PhII with $23M bankroll for rare disease drugs

A little more than a year ago, the startup crew at Lumena Pharmaceuticals was based in North Carolina and working on a new drug for diabetics. But things have changed. The company moved to San Diego, refocused its lead program on a rare liver disease and today announced that it had rounded up $23 million in Series A cash from a trio of venture groups.

"That takes us through the end of 2014," says CEO Mike Grey, who preferred to have Lumena's office close to home on the West Coast, where he has helmed several biotechs over the years, including SGX. By that time the company will have had a chance to analyze mid-stage data from a trio of clinical studies of LUM001, which was picked up after Pfizer ($PFE) shelved the program following its acquisition of Pharmacia. Pappas Ventures, which started the company, contributed to the round along with RiverVest Venture Partners and Alta Partners.

LUM001 was originally a cholesterol drug, says Grey. But after some extensive clinical studies with 1,400 patients, the drug had a clear safety profile and ho-hum efficacy compared to statins on the market, causing investigators to lose interest. But LUM001 also could put a brake on bile, which recirculates into the liver. 

Adult and pediatric patients with cholestatic liver disease suffer from an excess of bile in the liver, triggering a powerful itch that can leave patients bloodied from persistent scratching. Currently patients are treated with a surgical procedure, leaving them with an opening in the abdomen to drain off bile into an ostomy bag. Grey believes a pharmacological alternative like LUM001 would be much preferred.

Because of the earlier testing, Grey says Lumena can skip straight into Phase II in search of solid proof-of-concept data for the rare disease. And once the trials are completed in the second half of next year, a successful outcome could leave them on the threshold of a partnership or set them up to go into Phase III after raising more cash.

Lumena has also acquired a follow-up drug for cholestatic liver disease from Sanofi ($SNY) and a Phase I study will be wrapped later in the year.

In addition to Grey, there are 6 other members of the team, including Alejandro Dorenbaum, the chief medical officer, who completed stints at Genentech and BioMarin ($BMRN), gaining experience in developing drugs for orphan indications.

- here's the press release

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