Marvel Announces First of Two Final Data Sets from Rett Syndrome Study: MB-204 Significantly Outperforms Trofinetide

Rhea-AI Summary

Marvel Biosciences (TSXV: MRVL, OTCQB: MBCOF) has released partial final data from its preclinical Rett syndrome study comparing its lead compound MB-204 with Trofinetide, the only FDA and Health Canada approved treatment for the condition.

The study, conducted with the iBraiN Institute, demonstrated that MB-204 (10 mg/kg oral daily) significantly outperformed Trofinetide (100 mg/kg injected daily) across multiple endpoints in Mecp2 mice over a two-week treatment period:

• MB-204 reversed social behavioral deficiencies in nose and paw touching (p<0.0001 vs control)
• MB-204 reversed impairments in following behaviour (p<0.0001 vs control)
• Both treatments improved social preference behaviour in the three-chamber test
• MB-204 reversed spontaneous alternations and same arm returns in Y-maze test (p<0.0001 vs control)

Analysis of the carry-over effects is ongoing and will be released separately. The company plans to publish and present the data at an upcoming scientific conference.

Positive

• MB-204 demonstrated superior efficacy compared to FDA-approved Trofinetide across multiple endpoints
• MB-204 is administered orally once daily vs. Trofinetide's injectable administration
• Lower dosage requirement for MB-204 (10mg/kg) compared to Trofinetide (100mg/kg)
• Statistically significant improvements in multiple behavioral metrics (p<0.0001)

Negative

• Study results are preclinical (animal testing phase) - still far from market approval
• Complete data set not yet available
• Carry-over effects analysis still pending

Calgary, Alberta--(Newsfile Corp. - March 13, 2025) - Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF) and its wholly owned subsidiary, Marvel Biotechnology Inc. (collectively the "Company" or "Marvel"), is pleased to share part of the final data from its preclinical Rett syndrome study conducted in collaboration with the iBraiN Institute.

The study evaluated MB-204, Marvel's lead compound (10 mg/kg oral once daily), in comparison to Trofinetide (100 mg/kg injected once daily), the only FDA and Health Canada approved treatment for Rett syndrome. Mecp2 mice were treated for approximately two weeks with either compound, and multiple endpoints were assessed while on drug treatment. Analysis of the carry-over effect of MB-204 and Trofinetide is ongoing and will be released separately.

Key Data Highlights:

• MB-204 reversed social behavioural deficiencies as measured by the number and duration of nose touching and the number and duration of paw touching (p<0.0001 vs control). Trofinetide showed no improvement and MB-204 was significantly better than Trofinetide on the same endpoints (p<0.0001).
• MB-204 also reversed impairments in following behaviour (p<0.0001 vs control). Trofinetide showed no improvement and MB-204 was significantly better than Trofinetide on the same endpoint (p<0.001).
• Both MB-204 and Trofinetide reversed social preference behaviour measured by the three-chamber test (both p<0.0001 vs control). The three-chamber test is one of the most commonly used methods for evaluating social behavior in mouse models of autism spectrum disorder.
• MB-204 reversed the percentage of spontaneous alternations (%SPA) and the percentage of same arm returns (%SAR) in the Y-maze test (p<0.0001 vs control). Trofinetide showed no improvement and MB-204 was significantly better than Trofinetide on the same endpoints (p<0.0001). SPA typically measures spatial working memory and repetitive behaviours. SAR also reflects repetitive behaviour which often occurs in autism and obsessive-compulsive disorders models.

"We are very grateful to our collaborators for this excellent data, which clearly suggests MB-204 could be a very promising treatment for Rett Syndrome," said Chief Science Officer Dr. Mark Williams. "We also look forward to the remainder of the data, where we see how long the effect of MB-204 lasts after treatment has stopped."

The Company and its collaborators intend to publish and present the data at an upcoming scientific conference.

About Marvel Biosciences Corp.
Marvel Biosciences Corp., and its wholly owned subsidiary, Marvel Biotechnology Inc., is a Calgary-based pre-clinical stage pharmaceutical development biotechnology company. The Company is developing MB-204, a novel fluorinated derivative of the approved anti-Parkinson's drug Istradefylline, the only clinically approved adenosine A2a antagonist. A significant and growing body of scientific evidence suggests drugs that block the adenosine A2a receptor, such as MB-204, could be useful in treating other neurological diseases such as autism, depression and Alzheimer's Disease. The Company is actively investigating its potential in addressing other neurodevelopmental disorders, such as Rett Syndrome and Fragile X Syndrome, to expand its therapeutic reach.

Contact Information:

Marvel Biosciences Corp.
J. Roderick (Rod) Matheson, Chief Executive Officer or
Dr. Mark Williams, President and Chief Science Officer
Tel: 403 770 2469

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.

To view the source version of this press release, please visit https://www.newsfilecorp.com/release/244351

FAQ

How does Marvel's MB-204 compare to Trofinetide in treating Rett syndrome symptoms?

MB-204 significantly outperformed Trofinetide in reversing social behavioral deficiencies, following behavior, and Y-maze test metrics (p<0.0001), while both drugs showed improvement in social preference behavior.

What dosage of MB-204 was used in Marvel's Rett syndrome study (MBCOF)?

The study used 10 mg/kg oral once daily dosage of MB-204, compared to 100 mg/kg injected once daily of Trofinetide.

What specific behavioral improvements did MBCOF's MB-204 show in the Rett syndrome trial?

MB-204 improved nose touching, paw touching, following behavior, social preference, spontaneous alternations (%SPA), and same arm returns (%SAR) in test subjects.

When will Marvel Biosciences (MBCOF) release the complete Rett syndrome study results?

The analysis of carry-over effects is ongoing and will be released separately, with plans to present complete data at an upcoming scientific conference.