Marvel Announces the Second of Two Final Data Sets from Rett Syndrome Study: MB-204 Significantly Outperforms Trofinetide Following Cessation of Drug Treatment

Rhea-AI Summary

Marvel Biosciences has announced significant results from its preclinical Rett syndrome study comparing MB-204 to Trofinetide. The study revealed that MB-204, administered at 10 mg/kg orally once daily, demonstrated superior and longer-lasting effects compared to Trofinetide (100 mg/kg injected).

Key findings show MB-204 maintained effectiveness for up to three weeks after treatment cessation, significantly outperforming Trofinetide in reversing social behavioral deficiencies. The drug showed remarkable results in multiple behavioral tests, including nose contacts, paw contacts, and following episodes (p<0.0001 vs control).

The research, conducted at the iBraiN Institute, will be presented at the International Rett Syndrome Foundation Scientific Meeting in Boston, June 2025. Scientists noted MB-204's potential as a novel treatment for both Rett syndrome and autism, highlighting its sustained effectiveness without reduction after chronic dosing.

Positive

• MB-204 significantly outperformed FDA-approved Trofinetide in Rett syndrome treatment
• MB-204 showed sustained efficacy for 3 weeks after treatment cessation while Trofinetide showed no carry-over effect
• MB-204 demonstrated effectiveness at lower dosage (10mg/kg) compared to Trofinetide (100mg/kg)
• MB-204 reversed almost all social deficits in the test model with statistical significance (p<0.0001)
• Drug maintained effectiveness after chronic dosing, indicating no tolerance development

Negative

• None.

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Calgary, Alberta--(Newsfile Corp. - April 30, 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 second final data set from its preclinical Rett syndrome study conducted in collaboration with Dr. Julie Le Merrer and Dr. Jerome Becker at 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 i.p. 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 then treatment ceased. Animals were monitored for up to three weeks post-treatment to study the carry-over effect of the drug.

Key Data Highlights:

• Week 1: MB-204 continued to reverse social behavioural deficiencies as measured by the number and duration of nose contacts, the number and duration of paw contacts, the number of following episodes and in the 3 Chamber Test (p<0.0001 vs control). Trofinetide only showed an improvement on following episode (p<0.05 vs control). MB-204 was significantly better than Trofinetide on the same endpoints (p<0.0001 except following episodes p<0.01). The 3 Chamber Test was conducted on day 10 post treatment.
• Week 2: MB-204 continued to maintain its positive effect on the number and duration of nose contacts, in addition to a continued effect on following episodes (p<0.0001 vs control), and outperformed Trofinetide (p<0.0001 and p<0.05 on the same respective endpoints). Trofinetide showed no discernible carry-over effect.
• Week 3: MB-204 continued to maintain its positive effect on nose contacts and following episodes (p<0.0001 vs control). The Trofinetide was not studied owing to the lack of any discernible carry-over effect in the previous week.

"These results are very encouraging," said Drs. Le Merrer and Becker. "MB-204 nearly reversed all the social deficits in the Mecp2 model while under treatment as we previously saw after a single dose in the Oprm1 mouse model of autism. This study suggests the drug does not reduce its effectiveness after chronic dosing, and we were pleasantly surprised by the continued carry-over effect seen at week 3 which suggests a profound sustained effect of MB-204. We look forward to seeing MB-204 advance as a novel treatment for Rett syndrome and autism in general".

The Company and its collaborators intend to present the data as an oral presentation at the International Rett Syndrome Foundation (IRSF) Scientific Meeting, taking place in Boston, June 9-11th, 2025.

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/250025

FAQ

What are the key results of Marvel's MB-204 vs Trofinetide for Rett syndrome treatment in April 2025?

Marvel's MB-204 significantly outperformed Trofinetide by showing continued effectiveness up to 3 weeks after treatment stopped. MB-204 reversed social behavioral deficiencies with p<0.0001 vs control, while Trofinetide showed limited improvement and no carry-over effects after treatment cessation.

How long did the carry-over effects of MBCOF's MB-204 last in the Rett syndrome study?

MB-204's carry-over effects lasted at least 3 weeks after treatment cessation, maintaining positive effects on nose contacts and following episodes with p<0.0001 vs control, demonstrating a sustained therapeutic impact.

What dosage of MB-204 was used in Marvel's Rett syndrome study compared to Trofinetide?

Marvel's MB-204 was administered at 10 mg/kg orally once daily, while Trofinetide was given at 100 mg/kg injected i.p. once daily for approximately two weeks.

When will Marvel (MBCOF) present the Rett syndrome study results?

Marvel will present the MB-204 study data at the International Rett Syndrome Foundation Scientific Meeting in Boston, June 9-11th, 2025.

What social behaviors did Marvel's MB-204 improve in the Rett syndrome study?

MB-204 improved multiple social behaviors including the number and duration of nose contacts, paw contacts, following episodes, and performance in the 3 Chamber Test, with significant results (p<0.0001 vs control).