KIGALI, Rwanda — Akagera Medicines, Inc., a clinical-stage biopharmaceutical company developing lipid-nanoparticle therapeutics and vaccines for infectious diseases that disproportionately affect the majority world, together with the TASK Clinical Research Center in South Africa, today announced positive results from the single-ascending-dose (SAD) portion of the first-in-human Phase 1 study of AKG-100, the company’s investigational long-acting intravenous (IV) therapeutic being developed to transform the treatment of tuberculosis including drug-resistant tuberculosis (DR-TB).
The SAD study evaluated single IV doses of AKG-100 across a range of dose levels in 40 healthy volunteers and 15 patients with pulmonary tuberculosis in a 14-day Early Bactericidal Activity (EBA) cohort. AKG-100 was well tolerated at every dose studied, up to and including the highest dose of 850 mg. The overall safety profile was favorable; no participants discontinued due to adverse events and most adverse events were mild and self-limiting.
AKG-100 demonstrated a pharmacokinetic profile consistent with a long-acting profile. Following a single dose, drug concentration was maintained above the pre-specified target threshold for at least 14 days following a single administration while the modeled concentration-time profiles support exposure being maintained over 28 days. These findings support the potential for dosing measured in weeks to months rather than every day. If confirmed in later studies, that profile would represent a significant departure from current treatment regimens, which require daily oral dosing for six months or longer and for which adherence over the full course remains one of the central challenges in tuberculosis care.
AKG-100 acts through a mechanism that is independent of rifampicin and isoniazid, the backbone of first-line tuberculosis treatment, and we have demonstrated with clinical isolates that it retains its full activity in multidrug-resistant strains.
In an exploratory, model-based analysis of the tuberculosis-patient EBA cohort, a single dose of AKG-100 on day 1 was associated with an estimated 3.7 log10 reduction in bacterial burden by day 14, corresponding to an approximately 99.98% bacterial killing. The analysis also demonstrated a substantial increase in time to culture positivity by Day 14 with a model predicted improvement of approximately 298 hours from baseline. While these are early findings and model-derived signals from single-dose cohorts, they compare favorably with historical early bactericidal activity benchmarks and support continued clinical evaluation.
On the strength of these results, the multiple-ascending-dose (MAD) portion of the Phase 1 program is now underway.
Dr. Sachin Marulkar, Chief Medical Officer, Akagera Medicines
“These first-in-human results are an important milestone for the patients who today face many months of daily treatment for tuberculosis. AKG-100 was well tolerated at every dose we studied, and its pharmacokinetic profile points toward dosing measured in months rather than days. If that profile holds through later studies, it could change what a course of DR-TB treatment looks like, particularly in the settings where the burden of this disease is greatest.”
Prof. Andreas Diacon, Founder and Chief Scientific Officer, the TASK Group
“Akagera’s approach to tuberculosis treatment is undoubtedly the most innovative TASK has explored in its 20-year history. Early results with AKG-100 show good tolerability and safety alongside strong bactericidal activity. If follow-up studies are successful, AKG-100 could revolutionize tuberculosis treatment. Beyond being a new antibiotic, AKG-100 has the potential to transform how patients and caregivers experience tuberculosis treatment. Long-acting agents can significantly reduce pill burden, shorten treatment duration, improve compliance, and combat the emergence of resistance.”
Dr. Daryl Drummond, Co-Founder and Chief Scientific Officer, Akagera Medicines
“AKG-100 is built on a lipid-nanoparticle delivery platform with an approved clinical precedent, and this study is the first human evidence that the platform can carry a tuberculosis therapeutic with the long-acting exposure for which it was engineered. Sustaining levels above our target threshold from a single dose, alongside a clean safety readout, is exactly the proof of concept we were looking for as we move into multiple-dose testing.”
Michael Fairbanks, Co-Founder and Executive Chairman, Akagera Medicines
“Tuberculosis is the oldest pandemic we know. It has stalked humankind for nine thousand years under various names: consumption, the white plague, “the captain of all the men of death.” It has killed more people than any disease in history, and it still kills more than a million every year. It falls hardest on the people who have the least say over their own medicines. AKAGERA Medicines is majority-owned by the people of Rwanda. The company produced the first human data on a long-acting protocol. We intend to prove that innovations created and owned by the people who bear the greatest burden is the best way to finally end the pandemic.”
About AKG-100
AKG-100 is Akagera Medicines’ lead therapeutic candidate, a long-acting IV treatment for tuberculosis delivered on the company’s proprietary lipid-nanoparticle platform. It is designed to provide sustained drug exposure from infrequent dosing and to remain active against strains susceptible or resistant to currently recommended therapy. As a first aim, AKG-100 could help address major barriers in MDR-TB treatment, including adherence challenges, treatment interruptions, and the complexity of current multidrug regimens.
About Akagera Medicines
Akagera Medicines, Inc. is a clinical-stage biopharmaceutical company developing messenger-RNA and lipid-nanoparticle therapeutics and vaccines for infectious diseases that disproportionately affect the Global South, including tuberculosis, nontuberculous mycobacterial disease, RSV, HIV, Lassa fever, and Malaria, influenzas well as Avian Flu and next generation Mumps vaccines. Founded in 2018 and majority-owned by the Rwanda Social Security Board on behalf of Rwanda’s 14 million citizens, the company has partnered with the European Investment Bank, CEPI, the NIH/VRC, and the Gates Foundation. It operates in Kigali, Rwanda, San Francisco, California, and Waltham, Massachusetts, and is building sovereign strategic, scientific, and trials capabilities in Africa. For more information please visit https://www.akageramedicines.com/.
About the TASK Group
TASK is a global clinical research organization dedicated to advancing healthcare through innovative and ethical clinical trials. Its origins lie in Cape Town, South Africa, where TASK has conducted many pioneering clinical evaluations of novel tuberculosis antibiotics, vaccines or diagnostics in the past. TASK is covering a variety of therapeutic area at clinical sites in South African and Europe.
Media Contact
Sachin Marulkar, MD, MPH, MBA
Email: [email protected]