Marvel Biosciences Corp.

Marvel Biosciences is pleased to share additional data from its ongoing preclinical Rett syndrome study conducted in collaboration with the iBraiN Institute. Marvel also announces plans to engage with the FDA to discuss these promising pilot results. This study assessed MB204, our lead compound, alongside Trofinetide, the only FDA- and Health Canada-approved treatment for Rett syndrome. Key Data Highlights: - MB204 demonstrated strong carry-over effects on multiple social behaviour endpoints, persisting for 21 days post-treatment. This new data suggests a durable post-treatment and potential neuromodulator effect. - In contrast, Trofinetide showed minimal carry-over effects, lasting only 14 days. Building on these results in two models of autism, Marvel intends to begin the process with the FDA of obtaining Orphan and/or rare disease designation for MB204 in Rett Syndrome. Such designation could provide market exclusivity, tax incentives, and priority review waivers. “MB204 continues to exceed our expectations in pre-clinical studies,” said Dr. Mark Williams, Chief Science Officer of Marvel Biosciences. “MB204’s long-last effects are particularly exciting and encouraging.” "Alongside this program, we are advancing preclinical studies in Fragile X syndrome and recently secured a major grant to support preclinical research in Alzheimer’s disease," added Rod Matheson, CEO of Marvel Biosciences. "Achieving orphan or rare disease designation for MB204 could also add significant value to our lead asset." Marvel looks forward to sharing the full results of this study in Q1 2025.

Exciting News from Marvel Biosciences! We are thrilled to announce that Marvel Biosciences has been awarded an Alberta Innovates AICE Validates grant, securing $300,000 to advance the preclinical validation of MB204 for Alzheimer’s disease. Alzheimer’s impacts nearly 50,000 Albertans, yet current treatments only manage symptoms. MB204, our novel adenosine A2A receptor antagonist, is designed to target the disease’s underlying pathology, offering hope for better outcomes. This funding will enable us to conduct an important long-term preclinical study, moving MB204 closer to Phase 1 clinical trials. We are proud to lead innovation in Alberta and tackle this important healthcare challenge. Follow us to keep up to date with the latest news! #Biotech #Alzheimers #Innovation #MB204

Marvel Bioscience's CSO, Dr. Mark Williams, CSO will present live at the Life Sciences Investor Forum hosted by https://lnkd.in/e9k8BwT DATE: November 14th TIME: 10:30 AM ET LINK: https://bit.ly/3NuUDa4 Available for 1x1 meetings: November 14 and 15 This will be a live, interactive online event where investors are invited to ask the company questions in real-time. If attendees are not able to join the event live on the day of the conference, an archived webcast will also be made available after the event. It is recommended that online investors pre-register and run the online system check to expedite participation and receive event updates. Learn more about the event at https://lnkd.in/eAQa2uQ. Recent Company Highlights: ·  Marvel Biosciences Shares Positive Results of MB204 in the Oprm1 Model of Autism: https://lnkd.in/dHyPfbs8 ·  Marvel Biosciences Announces MB204 Outperforms Approved Drug Trofinetide in a Preclinical Model of Rett syndrome: https://lnkd.in/gkTkunae Marvel Biosciences Corp., and its wholly owned subsidiary, Marvel Biotechnology Inc., is a Calgary-based pre-clinical stage pharmaceutical development biotechnology company that utilizes a “drug redevelopment” approach to drug development. Historically, when a new class of drug is developed, it is optimized for a particular target, but typically only approved for a specific disease. Often, a new disease is identified which involves the same target, however, pending the remaining patent life, the originally approved drug may not have sufficient time left for it to be commercially viable to be developed for the new disease indication. Marvel develops new synthetic chemical derivatives of the original approved drug for the new disease indication. Patent protection is sought, as the new potential asset is developed by the Company. The Company believes the business model results in significantly less risk, cost and time to develop its assets compared to traditional biotechnology companies. Marvel Biotechnology Inc. has currently developed several new chemical entities, using synthetic chemical derivatives of known, off-patent drugs, that inhibit the A2a adenosine receptor with application to neurological diseases (depression & anxiety, Alzheimer’s, ADHD), and the non-neurological conditions of cancer and non-alcoholic steatohepatitis. Marvel is also exploring additional undisclosed targets to expand its asset pipeline. Press Release: https://lnkd.in/gUexskrk

Marvel Biosciences released promising interim results from its preclinical study on MB204 as a potential new treatment for Rett syndrome. The study, conducted by experts at the iBraiN Institute in Tours (France), demonstrated that chronic oral dosing of MB204 restored social interaction behaviors to near-normal levels in a preclinical model of Rett syndrome. These notable improvements in cognitive function could signify a pivotal shift in therapy. Dr. Julie Le Merrer and Dr. Jerome Becker investigated the effects of 14 days of MB204 dosing on socialization, behavior, and cognition in the Rett syndrome model (Mecp2 knockout mouse). The approved drug Trofinetide was included as a positive control. Preliminary data highlights are: Key highlights include: Restoration of Social Behaviors: MB204 treatment (10 mg/kg oral dosing once daily) reversed most social and behavioral deficits after 2 weeks. MB204 outperformed Trofinetide (100 mg/kg via intraperitoneal injection once daily) in most studied endpoints. Lasting Effects: There was a distinct carry-over effect (lasting drug effect after dosing stopped) for MB204 after 7 days on most direct social behavior endpoints, which was not observed for Trofinetide. The carry-over effect continued for at least 14 days after cessation of MB204 dosing for select endpoints. “This is the second preclinical model where we have seen a profound effect of MB204 on restoring many social behaviors”, commented Marvel CSO Dr. Mark Williams. “We included the approved drug Trofinetide as a positive control in the study, but to dose it to clinically equivalent levels, Trofinetide had to be injected. Although early, the data suggests that MB204 can improve social and behavioral functions more effectively with a much lower dose than Trofinetide. We are very interested in the carry-over effect and suspect the drug could be inducing synaptic plasticity in brain structures. We intend to complete the balance of the study and publish the data.” “With the recent approval of Trofinetide by the FDA and Health Canada by Acadia Pharmaceuticals, there is renewed hope for people with Rett syndrome,” said Marvel CEO Rod Matheson. “We believe these data with MB204 are very compelling and could result in even better therapies for individuals with Rett syndrome.”  Press release for Mecp2 study: https://lnkd.in/gkTkunae Press release for Oprm1 study: https://lnkd.in/dHyPfbs8 Press release for Fmr1 study: https://lnkd.in/dgAHPXxE Press release for Acute Tau Phosphorylation Study: https://lnkd.in/gQxKxZkv #Biotech #Rettsyndromresearch #Pharma #Innovation #Healthcare #MB204 #MarvelBiosciences

We are delighted to share that our lead compound, MB204, has shown incredible results in a recent study on autism led by Drs. Le Merrer and Becker at the iBraiN Institute in Tours France. In the Oprm1 mouse model, commonly used to study social behavior deficits, MB204 significantly restored social interactions—almost like flipping a switch! In fact, just one hour after a single dose, social behaviors like nose and paw contacts were back to normal and, in some cases, even better than in non-autistic mice. Key Highlights: - A high dose of MB204 (2.5 mg/kg) significantly restored social behavior in all measured endpoints (p<0.0001). Improvements were seen in social interaction behaviors, including the number and duration of nose contacts, paw contacts, and time spent self-grooming, among others. - A lower dose of MB204 (1 mg/kg) also reversed nearly all social behavior deficits, showing a clear connection between dose and response. - The higher dose of MB204 not only improved/restored social interactions in Oprm1(autistic) mice but even outperformed the social interactions of normal (non-autistic) mice on several endpoints, demonstrating MB204’s potent pro-social effect. “These results are very encouraging,” said Drs. Le Merrer and Becker, “MB204, at both doses, nearly reversed all the social deficits/endpoints in our autism model, and in some cases, the Oprm1 animals appeared more sociable than normal animals. We have previously tested Istradefylline (an off-patent approved drug similar to MB204), in multiple models of autism such as Oprm1, Mecp2, and Fmr1. Still, we have not seen this level of improvement or the hyper-social effect before”. Currently, Drs. Le Merrer and Becker are now testing MB204 in another chronic Rett syndrome model (Mecp2) and are looking forward to seeing how it performs ‘head-to-head’ against the approved drug Trofinetide. “We are very pleased with these findings for MB204 and intend to publish them in a peer-reviewed journal”, said Marvel’s Chief Science Officer, Dr. Mark Williams. “MB204 is currently being tested in two very clinically relevant autism spectrum disorder models of Rett syndrome (Mecp2) and Fragile X syndrome (Fmr1); the latter is in collaboration with the FRAXA Research Foundation, and we look forward to reviewing the results”. Link to full press release: https://lnkd.in/dHyPfbs8 Link to release on Mecp2 study: https://lnkd.in/gMXEkzcQ Link to release on Fmr1 study: https://lnkd.in/dgAHPXxE #Biotech #AutismResearch #Pharma #Innovation #Healthcare #MB204 #MarvelBiosciences

Marvel Biosciences Corp. is excited to announce its collaboration with the FRAXA Research Foundation to complete pre-clinical research on its lead asset #MB204 for #FragileXSyndrome (#FXS). Autism and FXS are closely related, often sharing clinical symptoms (e.g., developmental delays, challenges in social interactions, repetitive behaviors, and sensory sensitivities) and genetic mutations (e.g., FMR1). FXS is a one of the most common causes of inherited intellectual disability affecting approximately 1 in 4000 males and 1 in 8000 females. These genetic and clinical ties suggest that treatments for FXS are likely to help people with autism, Alzheimer’s and other brain (neurological) conditions. The FRAXA Research Foundation is a not-for profit organization that promotes awareness of Fragile X and supports independent pre-clinical testing of potential therapeutics through a battery of standardized tests in mouse models of FXS. FRAXA intends to test Marvel’s lead asset MB204, an adenosine A2a receptor (A2aR) antagonist through its internal screening program this year. FRAXA previously identified BPN14770, which is currently in Phase 3 trials for Fragile X, initially developed by Tetra Therapeutics and later acquired by Shionogi. Of note, there is currently no approved drug to treat FXS.  “We are very interested in testing MB204 in our independent laboratories for two primary reasons”, said Dr. Mike Tranfaglia, the Medical Director and co-founder of FRAXA. ”First, MB204 is a novel fluorinated derivative of the approved anti-Parkinson’s Disease drug Istradefylline, the latter having already been shown to be active in Fmr1 mouse models of autism. Second, MB204 has completed its pre-clinical toxicology studies and cGMP manufacturing and is at the ready for clinical testing”. “We are very grateful to be able to collaborate with FRAXA to test MB204 in their mouse models of FXS”, commented Dr. Mark Williams, Chief Scientific Officer of Marvel Biosciences. “We believe MB204 is a novel potential treatment for FXS and other forms of autism spectrum disorder.” Full release here: https://lnkd.in/d89_XfQr #MarvelBiosciences #FRAXA #MB204 #FragileXSyndrome #FXS #Autism #Alzheimers #NeurologicalResearch #ClinicalTrials #DrugDevelopment #Biotech #Pharmaceuticals #RareDiseases #OrphanDisease #GeneticDisorders #MedicalResearch

Marvel Biosciences is excited to extend our collaboration with lead investigators Dr. Julie Le Merrer and Dr. Jerome Becker at the iBraiN Institute in Tours, France. Building on our promising results using MB204 in an autism model earlier this year (https://lnkd.in/gtNpnP7D, https://lnkd.in/gADkYWDk), we now aim to test our lead asset in a mouse model of Rett syndrome (Mecp2). Rett syndrome and autism share several genetic, clinical, and neurodevelopmental features, leading to a complex overlap between the conditions. Rett syndrome leads to severe impairments, including speaking, walking, eating, and breathing. "Based on our success of MB204 in rapidly improving social behaviors in a preclinical model of autism after a single oral dose, we are very interested in testing the chronic effect of MB204 in a head-to-head study against the only approved drug for Rett Syndrome, Trofinetide, in the Mecp2 preclinical model", commented Dr. Le Merrer. Trofinetide (Daybue®) was approved to treat Rett syndrome in 2023. "The extension of our collaboration with the iBraiN Institute is an exciting and significant step towards Marvel's mission to develop effective treatments for complex neurological disorders," explained Rod Matheson, Chief Executive Officer. “Marvel is hopeful that our ongoing research will yield impactful insights and a potential therapeutic option for Rett syndrome”. Full release here: https://lnkd.in/gMXEkzcQ #MarvelBiosciences #Biotechnology #Pharmaceuticals #RettSyndrome #AutismResearch #NeurologicalDisorders #iBraiNInstitute #MB204 #OrphanDrugs #ClinicalTrials #GeneticResearch #NeurodevelopmentalDisorders #AdenosineReceptor #Trofinetide #Collaboration #InnovativeTherapies #HealthcareInnovation #RareDiseases #PharmaPartnerships #MedicalResearch

Marvel would like to congratulate Dr. Gomez-Murcia, our scientific advisory board member Dr. Blum, and colleagues on their recently published paper “Neuronal A2A receptor exacerbates synapse loss and memory deficits in APP/PS1 mice”.  In this study, published in the leading neurological journal “Brain,” Dr. Blum and colleagues reported that early increases of the adenosine A2A receptor (A2aRs) in brain cells (neurons) could lead to the development of memory loss in mouse models of Alzheimer’s. What was notable in this study was that the memory deficits and behavior changes in transgenic (APP/PS1) mice were linked to Tau phosphorylation (specifically at the AT8 site) rather than amyloid plaques (the conventional Alzheimer’s target). “The data seems to back up the expression that in Alzheimer’s Disease (AD), beta amyloid is the trigger and Tau is the bullet” said Dr. Mark Williams, CSO of Marvel. In recent news, Marvel reported that its lead asset MB204, an A2aR antagonist (which acts against the receptor), decreased Tau phosphorylation (including the AT8 site) in an acute oral study. The mechanism(s) by which MB204 reduces Tau phosphorylation are currently being investigated with Professor Emmanuel Planel, a world leading expert on Tau pathology. Overall, the body of evidence suggests that orally available A2aR antagonists such as MB204 could be a valuable new approach to reducing Tau pathology and treating Alzheimer’s cognitive deficits. Link to Paper: Link to Paper: https://lnkd.in/ggE_T3H5 Link to Marvel Biosciences Website: https://lnkd.in/gmqT7YAY #AlzheimersDisease #Alzheimers #AlzheimersResearch #Neuroscience #A2AReceptor #TauPathology #BrainHealth #CognitiveDeficits #Biotechnology #Pharmaceuticals #Innovation #CanadianInvestors #LifeSciences #ClinicalResearch #ScientificDiscovery #Neurodegeneration #InvestmentOpportunity

Marvel Biosciences reported promising interim results from its acute study on MB-204 conducted by Professor Emmanuel Planel of Laval University, a world expert on Tau pathology. Tau phosphorylation is when phosphates attach to a Tau protein in the brain. When Tau proteins become overly phosphorylated, they aggregate and form "tangles" in neurons. The study demonstrated that short-term oral dosing of MB-204 reduces Tau phosphorylation at critical positions in Tau. Abnormal Tau phosphorylation is a crucial feature of cognitive impairment and neurodegenerative diseases like Alzheimer's disease. Using a mouse model of Tau hyperphosphorylation pioneered by Dr. Planel, a 2.5 mg/kg oral dose of MB-204 significantly reduced Tau phosphorylation at multiple epitopes in the cortex of mice, including the AT8 site on Tau. The AT8 site is exciting because it is used clinically to assess the extent of Tau tangles (Braak staging) and is well correlated with the degree of cognitive impairment. "These are very promising preliminary results with MB-204," commented Dr. Planel, "considering that hyperphosphorylated Tau is present not only in Alzheimer's but also in other neurodegenerative diseases such as Parkinson's, Chronic Traumatic Encephalopathy, FTD, CBD, PSP etc., it has the potential to be effective for multiple pathologies." "We are grateful for the opportunity to conduct this study with Dr. Planel." commented Dr. Mark Williams, CSO of Marvel Biosciences. "The A2a receptor has emerged as a target for depression, autism and Alzheimer's disease. We believe the accumulating data on MB-204 in each of these models suggests a promising product profile and look forward to starting clinical testing shortly." Check our website for the latest news updates and announcements: https://lnkd.in/gQxKxZkv #MarvelBiosciences #TauResearch #AlzheimersInnovation #NeuroscienceBreakthrough #MB204Study #TauProteinResearch #NeurodegenerativeDisease #BrainHealth #AlzheimersResearch #ScientificCollaboration #BiomedicalAdvances #TauPathogenesis #ClinicalTrials #PharmaceuticalResearch

Marvel Biosciences Corp. is delighted to report promising interim results from our collaborative autism study conducted by the iBrain Insitute in Tours, France. A single oral dose of MB-204 restored social interaction behaviors in autism model mice, signaling a potential game-changer in therapy. Collaborating with Dr. Julie Le Merrer and Dr. Jerome Becker, the study results exceeded expectations, showing Marvel's compound MB-204's capacity to normalize social deficits and enhance behaviors. "The interim data we've seen is not just promising; it could offer a totally novel approach to treating autism,” said Dr Julie Le Merrer. “The capacity of MB-204 to normalize behavior in animal models of autism supports our theory about the A2a receptor's influence on social interactivity. We're eager to validate these results with further testing and robust statistical analysis, which is already underway and expected to conclude in the coming months." Full news release here: https://lnkd.in/gtNpnP7D #AutismResearch #MB204 #biotech #canadianbiotech #canadianbusiness #alzheimers