Theris Medical Launches Patient Enrollment for “Orphan Drug” Clinical Trial to Help African-Americans Suffering from Painful Sickle Cell Ulcers
We navigated a pandemic to enroll our first patient in a clinical trial to treat a very underserved patient group unaddressed by Big Pharma
It’s Sickle Cell Awareness Month, and we couldn’t be more proud to announce the enrollment of the first patient in our Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment (D-SCOUT) clinical trial (NCT 04058197). The trial will investigate the company’s Deferoxamine Intradermal Drug Patch (DIDP) for the topical treatment of Sickle Cell Leg Ulcers (SCUs) to support our Orphan Drug Designation awarded in March of 2019.
The Food and Drug Administration’s Orphan Drug Designation Program provides orphan status to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S.
Sickle cell disease affects about 100,000, mostly African-American people in the U.S., according to the Center for Disease Control. African-Americans remain the least healthy ethnic group in the U.S. and still face profound disparities in health outcomes and access to quality health care, according to information published by the National Institutes of Health.
University of Alabama, Birmingham Enrolls First Patient
Multiple clinical sites across the U.S. will treat up to 48 patients to provide the initial safety data as part of the 12-week Phase I/IIa D-SCOUT study. Patients are initially evaluated in a 4 week run-in period and followed another 4 weeks after wound closure or the final treatment.
The first patient enrolled in the trial is under the care of the University of Alabama, Birmingham’s Adult Sickle Cell Program (ASCP), and Lifespan Comprehensive Sickle Cell Center (LCSCC).
“Chronic sickle cell leg ulcers greatly impact the patient’s quality of life and lead to significant pain, but they’re a rare complication and not well studied or evaluated,” explains Dr. Julie Kanter, director of the ASCP and co-director of the LCSCC. “A new potential therapy that could specifically target this complication and improve outcomes for affected individuals is very exciting.”
Novel Approach Based on a Proven Drug
Deferoxamine (DFO) is currently used to treat iron overload in sickle cell patients, but Theris’ approach is the first-ever use of the drug in a patch for topical treatment of sickle cell leg ulcers.
“We have limited options for treating SCUs, none of them effective,” adds Dr. Dag Shapshak, co-medical director of UAB’s wound clinic who is treating the SCU patients. “The successful outcome of the DIDP’s topical treatment of SCUs could have a significant impact on the treatment of these painful wounds.”
“This trial is a significant clinical program achievement for a very underserved population,” said Dr. Geoff Gurtner, CEO of Theris Medical and director of Stanford Hospital’s Advanced Wound Care Clinic. “DIDP is the culmination of over a decade of research in understanding how iron chelation can be effective in the treatment of iron-based wounds such as SCUs. If successful, it will be the first small molecule approved for use in treating wounds and open the door for further drug product innovation.”
The DIDP technology was developed at TauTona from published Stanford research and licensed intellectual property. TauTona recently established wholly-owned Theris Medical Incorporated to commercialize TauTona’s DIDP technology and develop additional wound therapies.
“Starting this study represents a notable milestone for TauTona, as this is our first biopharmaceutical company,” Joe Rimsa, TauTona’s COO. “Our fund structure facilitates quick, nimble, and cost-effective development of technology such as DIDP, and transitioning the technology to the clinic as demonstrated with the initiation of the D-SCOUT trial. Most importantly, we believe the DIDP program has the potential to provide relief to sickle cell patients who suffer from such debilitating wounds and pain.”
For information about enrolling in the clinical trial, visit: https://therismedical.com/clinicals/
