NEW YORK (August 21, 2024) – In July 2024, a Phase 2 proof-of-concept study was initiated to explore the use of telacebec (Q203) to treat Buruli ulcer. If successful, a single drug could cut treatment duration by more than half. In partnership with TB Alliance and with support from Qurient Co. Ltd, Professor Daniel O’Brien – a prominent clinician-researcher and expert in Buruli ulcer – is leading the study at Barwon Health and the Royal Melbourne Hospital in Australia. The trial will enroll approximately 40 adult patients with Buruli ulcer who will receive 28 days of telacebec (300mg) once daily, followed by a year of observation. Preliminary results are expected in 2025.
Speaking to the promise of the trial, Eugene Sun MD, Senior Vice President of Research and Development for the TB Alliance remarked, “Telacebec is emerging as a promising and versatile compound. We’ve seen excellent activity using telacebec for TB, but the activity observed against Buruli ulcer has been especially exciting.” Describing the potential impact, Dr. Sun noted, “The tragedy of a disease like Buruli ulcer is the disfiguring and deeply stigmatizing effect. I’m hopeful that we can translate the positive preclinical work into life-altering results for patients.”
Found primarily in West Africa and Australia, Buruli ulcer is a rare bacterial infection of the skin and soft tissue caused by Mycobacterium ulcerans. Starting as painless swelling of affected areas, it often leads to the development of disfiguring ulcers and skin loss. Currently, Buruli ulcer requires up to eight weeks of treatment with a combination of dual antibiotic therapy that has a high incidence of side effects. The ulcers caused by the disease can often take months to heal even after use of antibiotics.
Telacebec is a compound discovered by Qurient Co. Ltd. of the Republic of Korea and licensed to TB Alliance in the field of TB and other mycobacterial diseases outside of the Republic of Korea, the Russian Federation and the Commonwealth of Independent States. Telacebec inhibits the mycobacterial cytochrome bc1 complex in the cellular respiration pathway, resulting in the depletion of ATP in M. tuberculosis, M. leprae and M. ulcerans, which are causative agents of tuberculosis, leprosy and Buruli ulcer, respectively. Depletion of ATP leads to the death of these bacteria. Extensive preclinical studies have been performed characterizing the potential efficacy and safety of telacebec for these mycobacterial diseases. Telacebec has received Orphan Drug Designation and Fast Track Designation from the U.S. FDA.
This is TB Alliance’s first significant involvement in a clinical trial of a drug candidate to treat a disease beyond TB and is the result of an effort in recent years to leverage the organization’s TB drug pipeline to address diseases when there is scientific opportunity and medical need.
Articulating the importance and compatibility of this work within the organization’s mission, TB Alliance President and CEO, Mel Spigelman MD noted, “When we see the potential for compounds in our pipeline to address other diseases with great unmet need, we don’t hesitate to bring our science to bear on finding solutions. We’re hopeful that our work in related fields will lead to new and improved cures for diseases that have long been neglected.”
For 25 years, TB Alliance has harnessed innovative science to advance better, faster-acting and affordable TB drugs. Learn more about the organization that manages the largest pipeline of TB drugs in history to eliminate the deadliest infectious disease on the planet at tballiance.org.