Lumos Pharma spent much of its $14 million A round on laying the preclinical/IND foundation for its lead rare disease program. And now the Austin-based company has $34 million more to mount a clinical stage program over the next two years that could blaze a path directly to an NDA.
Lumos is tackling a tough challenge: Rare cases of X-linked creatine transporter deficiency, a type of mental retardation caused by a genetic mutation that disrupts the transporter protein required to ship creatine through the blood-brain barrier and cell membranes. Their LUM-001 is designed to accomplish the task.
The startup strategy at Lumos, which is staffed by a virtual team of 10, is to take advantage of the clinical and regulatory shortcuts open to an orphan-oriented biotech so they can blitz through a small safety study--with a subset of patients to look at efficacy--and then straight into a small pivotal study on the way to an approval, if everything works according to plan.
"The orphan space offers some pretty interesting regulatory advantages, and we're going to take advantage of every one that's available," says CEO Rick Hawkins, singling out a shot at a breakthrough drug designation as well as orphan trial designs that can deliver big results from small patient groups.
Hawkins has an A team of investors willing to fund the work. Deerfield Management led the latest financing, joined by new investors Clarus Ventures and Roche Venture Fund, as well as existing investors New Enterprise Associates, Sante Ventures and the Belgian pharmaceutical company UCB.
The star of the show right now is LUM-001 for CTD, but Hawkins has another program taking shape now that can move toward clinical testing as well. That therapy is centered on lymphangioleiomyomatosis, a rare lung disease that usually strikes women during their child-bearing years.
Beyond that, Hawkins is looking at in-licensing more programs that can be used to build a rare-disease pipeline. Hawkins has the kind of international background that makes it relatively easy to run a virtual biotech in Austin. He earlier ran Pharmaco, a CRO that merged with PPD, and then operated a biotech research management company while advising startups. That kind of background provided plenty of opportunity to link up with development specialists who could do much of the work for the biotech under contract.
- here's the release