Vanda Pharmaceuticals Announces Presentation at 2025 AAN Annual Meeting

Rhea-AI Summary

Vanda Pharmaceuticals (VNDA) announced its participation at the American Academy of Neurology (AAN) Annual Meeting in San Diego from April 5-9, 2025. The company will present a poster on April 9 showcasing data for VCA-894A, a novel therapeutic customized for a specific genetic mutation in Charcot-Marie-Tooth disease type 2S (CMT2S).

The presentation will highlight how VCA-894A demonstrated significant improvements in neuromuscular function using Hesperos' Human-on-a-Chip® model with patient-derived cells. Key improvements include reduced muscle fatigue and enhanced synaptic transmission between motor neurons and skeletal muscle myotubes.

The FDA has granted VCA-894A an orphan designation, and it is expected to be administered to the specific patient for whom it was developed. The experimental platform represents advancement in precision medicine, potentially enabling development of genetically-tailored treatments for unmet medical needs.

Positive

• FDA granted orphan drug designation for VCA-894A
• Successful demonstration of therapeutic efficacy in patient-derived cell testing
• Near-term catalyst with imminent patient treatment

Negative

• Treatment to single patient with specific genetic mutation
• Early-stage development with unproven commercial viability

News Market Reaction

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Data tracked by StockTitan Argus on the day of publication.

WASHINGTON, April 7, 2025 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced participation at the American Academy of Neurology (AAN) Annual Meeting, to be held in San Diego, California from April 5 through April 9, 2025. 

The following will be presented:

April 9, 2025 

Presentation Title: "Translating IGHMBP2 Variants with a CMT2S Patient-specific Organ-on-a-chip Model: Personalized Medicine ASO-based Therapeutic Rescue"
Poster Presentation Session: P-12
Poster Number: 11-017
Presenter: Dr. Sandra Paulina Smieszek, Head of Genetics

This poster describes the data around a novel breakthrough therapeutic (VCA-894A) customized to a specific genetic mutation of a patient with Charcot-Marie-Tooth disease type 2S (CMT2S). The effects of VCA-894A were confirmed in Hesperos' Human-on-a-Chip^® neuromuscular junction model with patient-derived cells where VCA-894A demonstrated significant improvements in neuromuscular function, including reduced muscle fatigue and improved synaptic transmission between motor neurons and skeletal muscle myotubes. The use of a microphysiological system to evaluate the effects of a genetically tailored treatment represents a significant advancement in precision medicine and the use of human relevant methods of evaluation.

VCA-894A has been granted an orphan designation by the FDA and it is expected to soon be administered to the specific patient for whom it was developed. The experimental platform described in this poster has the potential to unlock the development of treatments that can address significantly unmet medical needs based on genetic understandings and precision medicine.

Vanda has previously published an article titled "Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2S."  In addition to the data published in the article, this poster will present some novel unpublished data on conduction velocity. 

For access to the full article, visit DOI: 10.1016/j.omtn.2025.102479.

For more information on potential ASO-based personalized treatment for CMT2S, please see Vanda's prior announcement, available here. 

For more information on the AAN Annual Meeting, please refer to www.aan.com. 

About Vanda Pharmaceuticals Inc.

Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit www.vandapharma.com and follow us on X @vandapharma.

Corporate Contact:
Kevin Moran
Senior Vice President, Chief Financial Officer and Treasurer 
Vanda Pharmaceuticals Inc.
202-734-3400
pr@vandapharma.com

Jim Golden / Jack Kelleher / Dan Moore
Collected Strategies
VANDA-CS@collectedstrategies.com

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SOURCE Vanda Pharmaceuticals Inc.

FAQ

What breakthrough did Vanda Pharmaceuticals (VNDA) announce for CMT2S treatment?

Vanda developed VCA-894A, a personalized therapeutic targeting a specific genetic mutation in CMT2S, showing significant improvements in neuromuscular function through Human-on-a-Chip® testing.

What regulatory status has VNDA's VCA-894A received from the FDA?

VCA-894A has received orphan drug designation from the FDA and is expected to be administered to the specific patient it was developed for.

When will Vanda Pharmaceuticals present their CMT2S research at AAN 2025?

Vanda will present their research on April 9, 2025, at the AAN Annual Meeting in San Diego, with poster number 11-017 in session P-12.

What improvements did VNDA's VCA-894A demonstrate in testing?

VCA-894A showed reduced muscle fatigue and improved synaptic transmission between motor neurons and skeletal muscle myotubes in patient-derived cell testing.