Austin's Lumos Pharma raises $34 million to fight rare genetic disease

Posted April 6th, 2016

Story highlights
  • Investment will allow Lumos Pharma to begin clinical trials

An Austin biotech company that's developing a new therapy for a rare genetic disease has raised $34 million to launch clinical tests.

Lumos Pharma, founded in 2011, will use the money to test and commercialize a therapeutic treatment for those suffering from Creatine Transporter Deficiency, or CTD.

CTD is a rare disorder characterized by severe cognitive impairment and seizures.

The disorder is estimated to affect about 50,000 boys in the United States and is thought to be the second-most common cause of X chromosome-linked developmental disability after Fragile X syndrome. Many boys are misdiagnosed with autism due to CTD's symptoms. 

Lumos received the funding from new investors Deerfield Management Co., Clarus Ventures and Roche Venture, as well as existing backers New Enterprise Associates, Sante Ventures and Belgian pharmaceutical company UCB.

The funding allows Lumos Pharma to move forward with its first phase of clinical trials in May, said Rick Hawkins, founder and CEO.

"These boys have severe developmental delays, no language development, often have seizures and may end up institutionalized," Hawkins said. "Currently there is no treatment, and we're hopeful that we're going to have a highly effective drug."

The deal is the second recent investment in an Austin-based biotech company. Savara Pharmaceuticals completed a $20 million financing in March as it prepares to launch new clinical trials of its inhalable drug for cystic fibrosis patients.

Meanwhile, Austin-based biomedical company Aeglea BioTherapeutics has the terms for a pending initial public offering of stock that could raise up to $72 million. Aeglea develops drugs to fight cancer.

Lumos' lead compound, LUM-001, has been granted orphan status by the U.S. Food and Drug Administration. That qualifies the company for a variety of incentives designed to speed the development of the drug. LUM-001 development is supported by a subsidiary of the National Institutes of Health, and researchers from that organization are collaborating with Lumos on the story and development of the treatment.

A year ago, Lumos received an award of up to $5.5 million from the Translation  Fund of the Wellcome Trust, a biomedical research charity based in London. Prior to that, the company raised $14 million from Sante Ventures and New Enterprise Associates.

The 10-person company is partnering with scientists at the National Center for Advancing Translational Sciences during pre-clinical development. 

Hawkins said that collaboration has generated the data needed to file an investigational new drug application for LUM-001 with the U.S. Food and Drug Administration.

In addition to developing the LUM-001 compound, Lumos will use the new funding to build an orphan diseases portfolio for the treatment of rare, debilitating genetic diseases.