Lumos Pharma

Lumos Pharma, based in Austin, Texas, is focused on developing novel therapies for patients with unmet medical needs in rare and neglected diseases. Lumos Pharma is a proud awardee of the National Institutes of Health's (NIH) Therapeutics for Rare and Neglected Diseases (TRND) program. The TRND program collaborates with biotechnology companies in the development of early stage technologies that show promise for the treatment of rare diseases. The TRND program provides in-kind scientific and financial support for the preclinical program to allow for the commencement of clinical trials in human subjects. Lumos Pharma's lead compound, LUM001, is a novel treatment for the rare disease, Creatine Transporter Deficiency (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 intellectual disability and developmental delay after Fragile X Syndrome. The inability to transport creatine can lead to intellectual disability, developmental delay,expressive speech deficits, autistic behaviors, and seizures. Many boys are misdiagnosed with autism due to the clinical presentation. 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. Additionally, Lumos Pharma has partnered with Key Opinion Leaders in the field in conducting a preclinical and clinical development program to evaluate the safety and efficacy of its promising drug candidate for the treatment of CTD.