By continuing to use our site, you agree to our Terms of Use and Privacy Policy. You can learn more about how we use cookies by reviewing our Privacy Policy.

I Agree

Lumos Pharma Receives Wellcome Trust Award for Advancing Potental Treatment of Creatine Transporter Deficiency (CTD)

AUSTIN, Texas – May 4th, 2015 – Lumos Pharma, Inc. (Austin, Texas) announced today that it has received an award of up to $5.5 million from the Translation Fund of the Wellcome Trust.

CEO of Lumos Pharma, Richard Hawkins, stated “We are very pleased Wellcome Trust has selected Lumos for an award and we are privileged to have an opportunity to work with them. This award will significantly advance our efforts to develop a much needed therapeutic for CTD.”

CTD is a relatively new and rare disorder, initially described in 2001, and is thought to be the second most common cause of X- linked developmental disability after Fragile X Syndrome. Boys generally present with developmental disability, most profoundly affecting speech, having a seizure disorder, and behavioral issues. Many boys are misdiagnosed with autism due to the clinical presentation.

“Rick and his team are singularly committed to developing a safe and effective treatment for this rare developmental disorder which currently has no other treatment alternatives. I am pleased to have the Wellcome Trust join the company’s investor syndicate, and I appreciate their recognition of the team’s excellent work to bring this important new medicine to market,” said Kevin M. Lalande, Managing Director of Santè Ventures.

LUM001 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. This NCATS collaboration has helped advance the lead molecule LUM001 as a clinical candidate by generating the data needed to file an investigational new drug application with the Food and Drug Administration.

The Wellcome Trust’s funding will further assist Lumos Pharma in conducting a preclinical and clinical development program to evaluate the safety and efficacy of its promising drug candidate for the treatment of CTD.


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 program. Lumos Pharma is located in Austin, Texas. Please visit the company’s website at:


The Wellcome Trust is a global charitable foundation dedicated to improving health. We provide more than £700 million a year to support bright minds in science, the humanities and the social sciences, as well as education, public engagement and the application of research to medicine. Please visit the trust’s website.