Pharma, Startups

Lumos Pharma raises $34M to treat inherited autism-like disorder

The round, led by Deerfield Management, will support clinical development of a drug for creatine transporter deficiency.

biotech investing JP Morgan 2016

Austin-based Lumos Pharma just closed out a $34 million Series B round, meant to fuel the development of its drug – LUM-001 – that treats an inherited disease called Creatine Transporter Deficiency. It’ll also build out its pipeline, with drug candidates LUM-002 and LUM-003 being developed for undisclosed indications. The round was led by Deerfield Management.

Commonly mistaken for autism, CTD is caused by a rare defect that prevents the transport of creatine across the blood-brain barrier. It can lead to behavioral problems, epilepsy and seizures. It most commonly presents in young boys and, notably, is the second leading cause of X-linked mental retardation in males after Fragile X syndrome, Lumos said.

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A Deep-dive Into Specialty Pharma

A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.

The disease is transmitted via the SLC6A8 gene, and sits solidly in the rare disease space. More than 150 individuals have been pinpointed with this specific disease, but it’s estimated to account for between 1 and 2 percent of males with intellectual deficiency, according to the NIH.

Lumos Pharma raised a $14 million Series A in February 2014, and has orphan designation. As Xconomy wrote at the time:

Lumos’s technology had been developed by researchers at the University of Cincinnati and was previously developed for use in cancer patients. But human trials didn’t provide the outcomes researchers were hoping to find, and so the technology was parked in a California company’s tech portfolio. Lumos was founded in 2011 and has a two-way license with the Cincinnati-based university to repurpose the compound as a therapy for CTD. Trials in mice have shown reversal in mental-retardation symptoms, Hawkins says.

If patients with creatine transportation disorders are given creatine, their cognitive symptoms improve. Lumos’ approach is to recreate a transport mechanism for creatine, Xconomy wrote in 2014. Today, LUM-001 is on the verge of entering Phase 1 trials.

[Image courtesy of Flickr user Pictures of Money]