PaxMedica, Inc. today announced that it has presented additional data and analyses from its Phase 2 clinical trial evaluating PAX-101
- David Hough, MD, CMO of PaxMedica presented new trial data and analyses during the “Research Pipeline: New Findings on Diagnostics and Therapeutics” program
|
| [28-October-2021] |
| TARRYTOWN, N.Y., Oct. 28, 2021 /PRNewswire/ -- PaxMedica, Inc. (“PaxMedica” or the “Company”), a biopharmaceutical company developing medicines that help overcome the challenges of living with complex neurological conditions, today announced that it has presented additional data and analyses from its Phase 2 clinical trial evaluating PAX-101 (intravenous suramin), an investigational drug with a proposed novel mechanism of action, that the Company is developing as a potential treatment for the core and related symptoms of Autism Spectrum Disorder (ASD). This trial, using low doses of intravenous suramin once monthly, expanded on earlier published reports of the potential for suramin as a treatment for the core and related symptoms of ASD and lends support to the important role that purine mediated mechanisms may have in the treatment of ASD. “We are excited to share these clinical data and the results of our recent analyses with the academic and medical child and adolescent psychiatry community at AACAP,” said David Hough, MD, Chief Medical Officer of PaxMedica. “There are currently no FDA approved treatments for the core symptoms of ASD and these data have generated support from the company’s highly regarded scientific advisors to rapidly advance the development of PAX-101 for treatment of core and related symptoms of ASD in larger clinical studies in the US and elsewhere.” This phase 2 study was a randomized, double-blind, placebo-controlled proof of concept clinical trial that was designed to test the efficacy and safety of suramin in boys aged 4-17 years with moderate to severe ASD. The study was conducted at 6 sites in South Africa where suramin is an approved medicine. The protocol was approved by the South Africa Health Products Regulatory Authority and each site’s Institutional Review Board or Ethics Committee. Fifty-two boys, ages 4 – 15 years were enrolled in 3 treatment arms: suramin 10 mg/kg, suramin 20 mg/kg, and placebo and were dosed by intravenous infusion at baseline, week 4, and week 8. The final study visit was at week 14. Subjects were diagnosed with ASD by DSM-5 criteria and severity was measured by the Autism Diagnostic Observation Schedule. The Aberrant Behavior Checklist (ABC) Core (subscales 2, 3, and 5) was the primary endpoint and Clinical Global Impressions – Improvement Scale (CGI-I), was a secondary endpoint. Both were assessed using intent-to-treat change scores from baseline to endpoint using analysis of variance. The subjects were multiracial with a mean age of ~8 years with wide variability in the severity of ASD symptoms at baseline. Forty-four subjects completed the study with 8 early withdrawals [COVID-19: 5, serious adverse event (SAE): 1, other reasons: 2]. ABC Core mean ± SE of 10 mg showed a greater numeric improvement (-12.5 ± 3.18) vs. placebo (-8.9 ± 2.86) (p = 0.37) at Week 14. The 20 mg arm did not show improvement vs. placebo. In exploratory analyses of the ABC-Core, the 10 mg arm showed greater differences from placebo in younger subjects and those with less severe symptoms. CGI-I mean ± SE changes from baseline were 2.8 ± 0.30 (p=0.016) in the 10 mg/kg arm and 2.0 ± 0.28 (p=0.65) in the 20 mg/kg arm, vs. 1.7 ± 0.27 in placebo. Suramin was generally safe and well-tolerated over 14 weeks. There was one SAE, “status epilepticus,” in a subject (20 mg arm) with multiple risk factors (cerebral palsy, hydrocephalus, VP shunt) for seizure, which resolved without sequelae. In summary, this dose-ranging proof-of-concept study of intravenous low-dose suramin for treatment of core and associated symptoms of ASD showed positive results on the CGI-I with a novel treatment. The 10 mg/kg dose showed greater efficacy, tolerability, and safety than the 20 mg/kg dose. Limitations of the study include small sample size and exploratory analyses that require confirmation in a larger study. About PAX-101 (IV suramin) About Autism Spectrum Disorder (ASD) About PaxMedica, Inc. Forward-Looking Statements These statements may be identified by the use of forward-looking expressions, including, but not limited to, “expect,” “anticipate,” “intend,” “plan,” “believe,” “estimate,” “potential, “predict,” “project,” “should,” “would” and similar expressions and the negatives of those terms. These statements relate to future events and involve known and unknown risks, uncertainties, and other factors which may cause actual results, performance, or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Prospective investors are cautioned not to place undue reliance on such forward-looking statements, which speak only as of the date of this press release. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. For more information, contact: PaxMedica, Inc. Media Relations:
SOURCE PaxMedica |
View original content:
