In yet another example of industry’s growing interest in orphan drugs, a small Texas startup secured an impressive Series A to ramp up its work on a treatment for a rare disease called CTD.
Life science investors Sante Ventures and New Enterprise Associates co-led the $14 million Series A for Lumos Pharma, which is repurposing a small molecule drug originally studied for use in solid tumors.
It thinks the molecule, now called LUM-001, may be able to alter the underlying cause of creatine transporter deficiency, an x-linked genetic defect that results in an absence of an essential component for the brain’s energy. Classified as an autism spectrum disorder, CTD can cause developmental delay, speech defects and behavior problems in children. It’s thought to be the second-largest cause of autism behind Fragile-X syndrome, according to Lumos.
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But the candidate is still in preclinical studies. Lumos says it will use the new funding to support preclinical and clinical development of the drug, which has already received orphan designation from the FDA. It’s working with researchers at the University of Cincinnati and the NIH’s Therapeutics for Rare and Neglected Diseases program on those studies.
The investment isn’t a surprising move for NEA, which has invested in a number of orphan drug efforts including Edimer Pharmaceuticals and an accelerator called Cydan LLC. Prosensa Holdings, a portfolio company that’s developing drugs for orphan neuromuscular disorders, went public last year.
Because of the small patient populations poised to benefit from orphan drugs, coupled with the high cost of drug development, the commercial side of healthcare hasn’t always been so interested in them. But as the pharmaceutical industry is slowly shifting away from blockbuster drugs and toward personalized medicine, and the FDA has put incentives in place, that’s changed.
Shire, for example, paid $4.2 billion to acquire ViroPharma‘s pipeline of drugs for rare diseases last year.
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The Pharmaceutical Research and Manufacturers of America claim there are more than 450 new medicines for rare diseases in clinical-stage development or undergoing FDA review, including 85 for genetic disorders.
