Istradefylline

CALGARY, Alberta, Aug. 05, 2026 (GLOBE NEWSWIRE) -- Marvel Biosciences Corp. (TSXV: MRVL | OTC: MBCOF ), and its wholly-owned subsidiary, Marvel Biotechnology Inc. (collectively the “Company” or “Marvel”), a drug discovery company developing novel therapeutics for autism spectrum disorder and related conditions, is pleased to announce positive results from its preclinical Fragile X study conducted through the FRAXA Drug Validation Initiative in collaboration with the FRAXA Research Foundation. This announcement represents another significant milestone in the continued de-risking of Marvel Biosciences' lead drug candidate, MB-204. By demonstrating statistically significant improvements in both behavioural and cognitive function in a Fragile X mouse model , including durable carry-over effects after treatment ceased, MB-204 has now generated compelling efficacy data across three independent preclinical models of autism spectrum disorder (ASD) , including Rett syndrome (Mecp2), the Oprm1 model of ASD, and now Fragile X syndrome (Fmr1). Collectively, these results further strengthen the scientific foundation supporting MB-204's advancement toward clinical trials while expanding its potential commercial opportunity across multiple orphan neurological indications with significant unmet medical need. Fragile X syndrome (FXS) is the most common inherited cause of intellectual disability and one of the most common single-gene causes of autism spectrum disorder. It is caused by mutations in the FMR1 gene on the X chromosome. As males have only one X chromosome, they are typically more severely affected than females. Current epidemiological studies estimate that FXS occurs in approximately 1 in 7,000 males and 1 in 11,000 females in the general population and as such it qualifies as an orphan disease in both the United States and Europe. The study was conducted through the FRAXA Drug Validation Initiative (FRAXA-DVI), an independent program that evaluates promising therapeutic candidates for Fragile X syndrome. The study evaluated MB-204, Marvel’s lead compound, in low- and high-dose treatment groups administered orally once daily in FMR1 knockout (KO) mice. FMR1 mice were treated for approximately two weeks and subjected to a standard battery of model-specific behavioural and cognitive tests, then retested two weeks after treatment ceased to study the carry-over effect of the drug. Overall, MB-204 restored every behavioural and cognitive endpoint studied in Fragile X mice toward wild-type (normal) levels , including improvements in novel object recognition (cognition), open-field test (locomotor activity), self-grooming (stereotypy), nesting (hippocampal dependent activity of daily living), hyphoneophagia (anxiety). Key highlights included: ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); After Two Weeks On Treatment: MB-204 produced clear dose-dependent behavioural effects in the Fmr1 knockout mouse on every endpoint, with the high-dose group showing the strongest and most consistent efficacy. Animals receiving the high-dose treatment generated response patterns statistically consistent with wildtype-like performance (WT) in open-field test activity, self-grooming, nesting and hyponeophagia (p<0.0001 vs KO vehicle), although nesting remained different from WT. The low-dose group produced significant but non-WT-like effects in open field, self-grooming and hyponeophagia (p<0.0001, p<0.017 and p<0.0001 vs KO vehicle, respectively) but had no detectable effect on nesting. Both treatment groups restored novel-object discrimination (p<0.0001 for both doses), demonstrating significant improvement in recognition memory, a key measure of cognitive function. After Two Weeks Off Treatment (Carry-Over Effect): The high-dose treatment group retained significant effects relative to KO vehicle in open field (p=0.003) and self-grooming (p=0.0015), although these responses were attenuated and no longer WT-like. The high-dose group also retained significant novel-object discrimination when tested with a completely different object set (p=0.0052), supporting a persistent recognition-memory effect rather than simple memory for the original objects. Overall, results support robust on-treatment efficacy and a measurable, though reduced, post-washout effect in the high-dose treatment group, while the low-dose treatment was insufficient to produce a durable post-treatment benefit. “This is now the third ASD model and second independent group that has demonstrated the potential of MB-204 to restore behavioural and cognitive function to near normal levels in mice regardless of the genetic cause of the neurodevelopmental disorder” commented Dr. Mark Williams, CSO of Marvel Biosciences . “We also confirmed a carry-over effect in a second model, suggesting MB-204 has a very attractive target product profile. We are very grateful to FRAXA for this collaboration and look forward to advancing MB-204 to the clinic”. This latest preclinical success in Fragile X syndrome marks the third autism-related model in which MB-204 has demonstrated the ability to restore behavioural and cognitive function toward normal levels, providing further validation of the drug's mechanism of action and strengthening the Company’s core investment thesis. Combined with previously reported positive results in the Rett syndrome (Mecp2) and Oprm1 ASD models, these findings continue to build a compelling preclinical package supporting the advancement of MB-204 toward clinical trials. The Company has also completed preclinical evaluation of MB-204 in a Shank3 model of Phelan-McDermitt syndrome and is awaiting final statistical analysis of the results. Marvel expects to report those results in a future announcement. About Marvel Biosciences Corp. Marvel Biosciences Corp. through its wholly-owned subsidiary Marvel Biotechnology Inc., is a Calgary-based biotechnology company developing new treatments for neurological diseases and neurodevelopmental disorders. Our lead drug candidate, MB-204, is a novel fluorinated derivative version of Istradefylline, an approved Parkinson’s drug and the only adenosine A2A receptor blocker currently on the market. Research shows that blocking the A2A receptor may help treat conditions such as autism, depression, and Alzheimer’s disease. Marvel is also exploring MB-204’s potential in rare disorders like Rett syndrome and Fragile X syndrome, aiming to bring new options to patients with few effective treatments. Contact Information: Marvel Biosciences Corp. J. Roderick (Rod) Matheson, Chief Executive Officer Email: rod@marvelbiosciences.com Dr. Mark Williams, President and Chief Science Officer Email: mark@marvelbiosciences.com Tel: 403 770 2469 Website: | Twitter/X | LinkedIn Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSXV) accepts responsibility for the adequacy or accuracy of this press release. All information contained in this news release with respect to the Company and its subsidiary,(collectively, the "Parties") were supplied by Marvel, respectively, for inclusion herein and each parties' directors and officers have relied on each other for any information concerning such Party. This news release may contain forward-looking statements and other statements that are not historical facts. Forward-looking statements are often identified by terms such as "will", "may", "should", "anticipate", "expects" and similar expressions. All statements other than statements of historical fact, included in this release, including, without limitation, statements regarding the future plans and objectives of the Company are forward-looking statements that involve risks and uncertainties. There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those anticipated in such statements. Important factors that could cause actual results to differ materially from the expectations of the Company and include other risks detailed from time to time in the filings made by the Company under securities regulations. The reader is cautioned that assumptions used in the preparation of any forward-looking information may prove to be incorrect. Events or circumstances may cause actual results to differ materially from those predicted, as a result of numerous known and unknown risks, uncertainties, and other factors, many of which are beyond the control of the Company. As a result, the Company cannot guarantee that the above events on the terms will occur and within the time disclosed herein or at all. The reader is cautioned not to place undue reliance on any forward-looking information. Such information, although considered reasonable by management at the time of preparation, may prove to be incorrect and actual results may differ materially from those anticipated. Forward-looking statements contained in this news release are expressly qualified by this cautionary statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company will update or revise publicly any of the included forward-looking statements as expressly required by Canadian securities law.