AUSTIN, Texas, April 6, 2016 /PRNewswire/ -- Lumos Pharma, a biotechnology company focused on developing therapeutics for orphan diseases, announced the successful completion of a $34 million Series B financing. Lumos will use the proceeds for clinical trials and commercialization of its lead compound, LUM-001, for the treatment of Creatine Transporter Deficiency (CTD). Funding will also be used to further develop other therapeutics in the company's pipeline. Deerfield Management Company led the financing, and is joined by new investors Clarus Ventures and Roche Venture Fund, as well as existing investors New Enterprise Associates (NEA), Sante Ventures and Belgian pharmaceutical company, UCB.
LUM-001 is a disease-modifying therapeutic targeting CTD, the second leading cause of X-linked mental retardation in males after Fragile X Syndrome. Patients with this recently-discovered defect can synthesize creatine but cannot actively transport it across the blood-brain barrier. They have severe delays in expressive speech and mental development, and also experience behavioral abnormalities, epilepsy and seizures. Due to the clinical presentation, they are often misdiagnosed with autism. This disease is severely debilitating and patients require lifelong care.
"We are excited to partner with Lumos, a pioneer in the orphan disease space, to help address the significant unmet need for patients suffering from Creatine Transporter Deficiency. We believe the company has a strong team in place to develop this critical asset in a rigorous manner to reach patients and the market quickly," said Cameron Wheeler, a Principal at Deerfield Management.
"We are pleased to have the support of such a distinguished group of investors who share our enthusiasm about the opportunity to develop an effective therapeutic for Creatine Transporter Deficiency," noted Rick Hawkins, CEO of Lumos Pharma. "We believe we are uniquely qualified to tackle this challenge and develop additional therapies that will help patients with rare diseases."
Lumos is an awardee of the highly-selective National Institutes of Health Therapeutics of Rare and Neglected Diseases (TRND) program. In 2015, Lumos was awarded an investment from the Translation Fund of the Wellcome Trust.
LUM-001 has been granted orphan status in the US and is in preclinical development in partnership with scientists at the National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health, through its Therapeutic for Rare and Neglected Diseases program (TRND). This NCATS collaboration has helped advance the lead molecule LUM-001 as a clinical candidate by generating the data needed to file an investigational new drug application with the Food and Drug Administration.
About Lumos Pharma:
Lumos Pharma is focused on bringing novel therapies to patients afflicted with unmet medical needs in severe, rare, and genetic diseases. Lumos Pharma is led by an experienced management team with longstanding experience in the rare disease space. Lumos Pharma's lead compound is supported by the National Institutes of Health's Therapeutics for Rare and Neglected Diseases (TRND) program.