- Phase 2a publication entitled “Combined Regulatory T-Lymphocyte and IL-2 Treatment is Safe, Tolerable, and Biologically Active for One Year in Persons with Amyotrophic Lateral Sclerosis” has been published in peer-reviewed journal Neurology, Neuroimmunology & Neuroinflammation
- COYA 101 was well tolerated and increased Treg suppressive function
- The majority (6 out of 8) of patients either slowed or stopped progression (mean change of -2.7 points on ALSFRS-R score) during the open label extension over a 24-week period while 2 out of 8 patients did not benefit from treatment
- Assessment of biomarkers of inflammation and oxidative stress over the course of the study correlated with ALS disease severity and may serve to prospectively evaluate and/or monitor treatment response
HOUSTON, Sept. 01, 2022 (GLOBE NEWSWIRE) —(“Coya”), a clinical-stage biotechnology company developing multiple approaches that aim to enhance regulatory T cells (Tregs) function, including allogeneic Treg-derived exosome therapeutics and novel biologics, today announced the publication of the Phase 2a clinical trial data for its first-in-class Treg cell therapy product candidate, COYA 101. Coya has an exclusive worldwide license from Houston Methodist Hospital to develop, manufacture, and commercialize COYA 101.
The article entitled “Combined Regulatory T-Lymphocyte and IL-2 Treatment is Safe, Tolerable, and Biologically Active for One Year in Persons with Amyotrophic Lateral Sclerosis” has been published online in the peer-reviewed journal Neurology, Neuroimmunology, & Neuroinflammation.
Subjects enrolled in the Phase 2a trial were initially randomized into a double-blind portion to receive active Treg cells or matching placebo every four weeks over 24 weeks. After completion of the 24-week double-blind (DB) portion, subjects entered an open-label extension (OLE) during which all patients received active Treg cells every four weeks for additional 24 weeks. The DB portion of the trial was curtailed due to COVID-19-related restrictions, and the limited number of patients enrolled made statistical analysis not feasible. However, the eight patients included in the OLE completed the 24-week study, and 75% (n=6) of them exhibited slow to no progression as measured by the ALSFRS-R (mean decline of -2.7 points). A sub-analysis of these six patients showed that four of them (50% of the intention-to-treat [ITT] patient population, n=8) exhibited no decline over the 24-week treatment period. The two remaining patients in the OLE did not experience a treatment benefit. The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) is a validated rating instrument that gauges the progression of disability in patients with amyotrophic lateral sclerosis (ALS) on a 48-point scale.
In addition to the benefit experienced by the majority of patients in clinical function, Treg suppressive function was higher following infusions, compared to baseline values at screening. An unbiased proteomics assay of the OLE specimens documented that the two rapidly progressing patients had markedly higher levels of biomarkers of inflammation (IL-17F and IL-17C) and oxidative stress (OLR1 and ox-LDL) compared to the other six patients who exhibited amelioration or halting of disease progression, representing important findings that may provide new tools to identify the most suitable ALS patients for the next well-powered and well controlled trial.
Overall, treatment with COYA 101 was well tolerated. No deaths were reported and no patient receiving Treg infusions were discontinued from the study.
Howard Berman, Ph.D., Chief Executive Officer of Coya, stated, “We believe this Phase 2a study, with the limitations of a small sample size, supports that COYA 101 was well tolerated and biologically active in ALS patients and may slow or halt disease progression in the right patient population. We believe the findings that high levels of inflammation and oxidative stress may limit the effectiveness of Tregs, resulting in shorter cell half-lives and impacting their downstream suppressive anti-inflammatory effects, may help to better enhance the signal by prospectively identifying the patients with the higher likelihood to respond,” concluded Dr. Berman.
“We are encouraged by the results from this study, which are consistent with the positive outcomes of the previous Phase 1 trial in ALS patients. We believe these results are relevant, as they provide information we need to design our next double-blind controlled study to assess the efficacy and safety of COYA 101,” added Adrian Hepner, M.D, Ph.D., President and Chief Medical Officer of Coya.
Stanley H. Appel, M.D., the Chair of the Scientific Advisory Board of Coya, stated, “Although the double-blind portion of the Phase 2a study was compromised by COVID-19, we believe the Open Label Extension supports the findings of our Phase 1 study that COYA 101 infusions are well tolerated, may enhance suppressive functions and may have the potential to slow disease progression.”
The online publication can be accessed at
About Coya Therapeutics, Inc.
Headquartered in Houston, TX, Coya Therapeutics, Inc. (Coya) is a clinical-stage biotechnology company developing approaches utilizing Treg modifying therapeutics to target systemic and neuro inflammation. Coya has pioneered the ability to produce Tregs with a highly suppressive phenotype (“Super Tregs”) from a patient’s own dysfunctional Tregs. “Super Tregs” confer their properties through reproducible upregulated proteins in the expanded/post cryopreserved condition that allow for an off-the-shelf like approach for serial infusion. Coya is also developing exosome therapies derived from “Super Tregs” for allogeneic applications, as well as a biologic that works to upregulate Treg function in vivo. Coya is focused on the advancement of disease modifying approaches to address the significant unmet medical needs of patients with Amyotrophic Lateral Sclerosis (ALS), Frontal Temporal Dementia (FTD), Scleroderma, and Hepatic Inflammation and Fibrosis. For more information, please visit.