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.
