11 tissue, blood, and cerebrospinal fluid biomarkers reflecting the biological cascade of neurodegeneration and neuroinflammation in PD respond in concert to risvodetinib treatment in patients with early untreated Parkinson’s disease
ATLANTA and BOSTON, Aug. 04, 2026 (GLOBE NEWSWIRE) -- ABLi Therapeutics (“ABLi”), a biotechnology company developing therapeutics to address diseases that arise from activation of Abelson Tyrosine Kinases (c-Abl kinases), announces today that it has completed an exhaustive biomarker study encompassing 7300 individual measures from up to 80 participants in the Phase 2 201 Trial, to evaluate the impact of risvodetinib treatment on human Parkinson’s disease.
“The analysis of tissue, blood and spinal fluid measures from participants in the 201 Trial has revealed surprising information, both about the underlying processes of disease and how patients respond to risvodetinib treatment. The outcomes indicate that the biological cascade of disease is reflected by these biomarkers and is substantially reversed in 12-weeks of once daily treatment with risvodetinib, including substantial reduction in phosphorylated alpha-synuclein, believed to be the causative agent of human PD. In our view, this is how ‘disease-modification should look,” Dr. Milton Werner, ABLi’s Chairman and Chief Executive.
ABLi was launched in May 2025 after drug-related reductions in alpha-synuclein aggregates, or ‘plaques’, in skin biopsy samples was demonstrated in 36 participants from the 201 Trial. On launch, ABLi undertook an ambitious effort to evaluate the effect of risvodetinib treatment on additional markers related to the pathological cascade, adding longitudinal blood (80 participants) and CSF (6 participants) measures from samples collected at baseline and at the end of the 12-week trial. In total, 11 markers were analyzed that report on processes of neuronal degeneration, mitochondrial health and neuroinflammation.
Analysis of these 11 biomarkers demonstrated that:
- 50, 100 and 200 mg once daily risvodetinib inhibited the target c-Abl kinase;
- 100 and 200 mg once daily risvodetinib substantially reduced phosphorylated alpha-synuclein biomarkers in the spinal fluid and all three doses substantially reduced phosphorylated alpha-synuclein levels in blood, suggesting the 100 and 200 mg doses should be further evaluated in the upcoming BASE, ABILITY and CAMPD trials;
- 50, 100 and 200 mg once daily risvodetinib suppressed neuroinflammation, measured by suppression of NLRP3 and the pro-inflammatory cytokines IL-1beta and IL-18, to levels below baseline, implying that neuroinflammation was suppressed by once daily risvodetinib treatment.
Taken together, these data demonstrate a consistent effect of risvodetinib on the cascade of cellular events that lead to Parkinson’s disease pathology in the central and peripheral nervous systems. ABLi is exploring diagnostic and prognostic applications of these biomarkers to inform its late stage CAMPD trial, which will also examine correlations between these markers of disease biology and clinical outcomes. These and other new data are the subject of an upcoming meeting with FDA in August 2026.
About Risvodetinib (ABLi-148009)
Risvodetinib is a potent, selective small-molecule inhibitor of the non-receptor c-Abl kinases, designed for once-daily oral use that targets the underlying biological mechanisms driving Parkinson’s disease initiation and progression. Risvodetinib is believed to be a disease-modifying therapy that halts disease progression and reverses the functional loss arising from Parkinson’s disease inside and outside of the brain. All marketed therapeutic approaches to treat Parkinson’s help manage the symptoms of the disease, but there are currently no available treatments to slow or stop the disease’s relentless progression. Recently, risvodetinib was the first monotherapy shown to improve patient quality of life in a randomized, placebo-controlled clinical trial (NCT NCT05424276) and simultaneously reduced the underlying synuclein aggregate pathology in untreated Parkinson’s disease. Risvodetinib currently has intellectual property protection beyond 2036.
About ABLi Therapeutics
ABLi Therapeutics (“ABLi”) applies innovative medicinal chemistry and a deep understanding of disease biology to develop small molecule therapeutics that target the cause of diseases that arise from activation or dysfunction of the Abelson Tyrosine Kinases (c-Abl). Leveraging its expertise in drug design, ABLi utilizes clinically validated data of kinase inhibitors to design and develop novel product candidates with enhanced penetration into the brain, greater potency and target selectivity, and improved safety to treat diseases in which Abl kinase activation or dysfunction is implicated. The Company’s primary focus is on developing therapeutics for the treatment of neurodegenerative diseases like Parkinson’s disease and the Parkinson’s-related neurodegenerative diseases Multiple System Atrophy and Dementia with Lewy Body that are all associated with Abl kinase activation or dysfunction. For more information visit www.ablitherapeutics.com or follow us on LinkedIn, Facebook or Instagram.
Contacts:
For ABLi Therapeutics
info@ablitherapeutics.com
Investor/Media
Mike Moyer
Managing Director – LifeSci Advisors
mmoyer@lifesciadvisors.com
