Vanda Pharmaceuticals Announces First Patient Dosed in a Trial Evaluating VCA-894A

Vanda Pharmaceuticals Announces First Patient Dosed in a Trial Evaluating VCA-894A

By, Admin 11 June 2025

Overview

Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the first dose in the first-in-human clinical trial to evaluate the safety and tolerability of VCA-894A, an antisense oligonucleotide (ASO) therapeutic, for a patient with a rare variant in the IGHMBP2 gene causing Charcot-Marie-Tooth disease Type 2S (CMT2S).

The VCA-894A & CMT2S

  • VCA-894A is being developed for a patient who was first diagnosed at the age of 5 with a rare subtype of CMT disease known as CMT2S.
  • CMT2S is an inherited neuromuscular disorder that progressively leads to muscle weakness and loss of motor function, and has an estimated prevalence of less than 1 in 1,000,000 worldwide.
  • The severity and clinical presentations of CMT2S are influenced by the diverse genetic variants associated with CMT disease.

Mechanism of working

  • VCA-894A targets a patient-specific IGHMBP2 variant and has been previously shown to restore expression levels of the IGHMBP2 gene in an ""organ-on-a-chip"" NMJ (neuromuscular junction) system derived from the patient's cells, highlighting the exciting potential of this approach for personalized therapeutics for rare diseases. 
  • This platform has the potential to unlock the development of treatments that can address significantly unmet medical needs based on identified causative genetic variants.

About rare diseases

  • Rare diseases collectively affect over 300 million people globally. 
  • Individualized treatments for such rare genetic disorders using nucleic acid-based platforms carry unprecedented potential to restore function where expression of crucial genes has been aberrant.

References:

  • Smieszek, S. et al. Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2S. Mol Ther Nucleic Acids 36, 102479 (2025).
  • Charcot-Marie-Tooth disease type 2S. Orpha.net (2025). https://www.orpha.net/en/disease/detail/443073

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.

About VCA-894A

  • VCA-894A is a 2'-O-methoxyethyl (MOE) phosphorothioate oligonucleotide sodium salt.
  • VCA-894A specifically targets a cryptic splice site variant within IGHMBP2, which causes CMT2S. 
  • ASOs may have broad applicability in addressing a number of disorders, from nervous system treatments to systemic treatments.

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

The press release contains forward-looking statements, subject to risks and uncertainties.

These statements include:

  • Estimated prevalence of CMT2S.
  • Development of individualized treatments.
  • Timing for administering VCA-894A to the intended patient.

Actual results may differ due to multiple factors, including:

  • Accuracy of CMT2S diagnosis reporting.
  • Success of Vanda's experimental precision medicine platform.
  • Timely administration of VCA-894A as planned.
  • No guarantees exist that anticipated results will occur as expected.

Forward-looking statements must be evaluated alongside market risks outlined in:

  • Vanda’s Annual Report on Form 10-K.
  • Quarterly Reports on Form 10-Q & SEC filings (www.sec.gov).
  • Vanda cautions investors not to rely too heavily on forward-looking statements.
  • The company is not obligated to update statements publicly, except as required by law.

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