WASHINGTON, Jan. 23,
2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc.
(Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug
Administration (FDA) has approved the Investigational New Drug
(IND) application to evaluate VCA-894A for the treatment of a
patient with Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S),
caused by cryptic splice site variants within the IGHMBP2 gene.
CMT2S is a rare subtype of Charcot-Marie-Tooth disease (CMT), an
inherited peripheral neuropathy for which there is no available
treatment. CMT2S is characterized by slowly progressive distal
muscle weakness and atrophy, affecting the upper and lower limbs in
a child's first decade of life, leaving patients with decreased
reflexes and sensory impairment1,2 The estimated overall
prevalence of CMT is 1 in 2,500 individuals, with varying clinical
features dependent on the various genetic variants of
CMT.1,2 The prevalence of the CMT2S variant is estimated
to be less than 1 in 1,000,000 worldwide.3
"This is an important milestone in the pursuit of personalized
medicine, which has the potential to enable the development of
treatments tailored to one's genetic variants, in this case
specifically for a patient with CMT2S causing genetic mutations,"
said Mihael H. Polymeropoulos, M.D., Vanda's President, CEO
and Chairman of the Board.
VCA-894A is a novel antisense oligonucleotide (ASO) with a
mechanism of action that specifically targets a cryptic splice site
variant within immunoglobulin mu-binding protein 2 (IGHMBP2).
Mutations within IGHMBP2 play a pivotal role in the manifestation
of CMT2S, likely due to alpha-motor neuron loss, and consequently
peripheral nervous system deterioration.4 ASOs have the
capacity to modulate gene expression, allowing for the personalized
treatment of rare diseases. Delivery of ASOs to the central nervous
system has been previously successful in several ASO programs, with
a broad applicability in addressing a number of neurodegenerative
and neuromuscular disorders.5
References
- Cottenie, E., et al. (2014). Truncating and missense mutations
in IGHMBP2 cause Charcot-Marie Tooth disease type 2. American
journal of human genetics, 95(5), 590–601.
https://doi.org/10.1016/j.ajhg.2014.10.002
- Nam, S. H., & Choi, B-O. (2019). Clinical and genetic
aspects of Charcot-Marie-Tooth disease subtypes." Precision and
Future Medicine, 3(2), 43–68.
https://doi.org/10.23838/pfm.2018.00163
- (2023). Charcot-Marie-Tooth disease type 2S. Orpha.net.
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=443073
- Rzepnikowska, W., & Kochański, A. (2021). Models for
IGHMBP2-associated diseases: an overview and a roadmap for the
future. Neuromuscular disorders: NMD, 31(12), 1266–1278.
https://doi.org/10.1016/j.nmd.2021.08.001
- Crooke, S. T., Liang, X. H., Baker, B. F., & Crooke, R. M.
(2021). Antisense technology: A review. The Journal of
biological chemistry, 296, 100416.
https://doi.org/10.1016/j.jbc.2021.100416
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 Twitter @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
Various statements in this press release, including, but not
limited to statements regarding the estimated prevalence of CMT and
CMT2S and the development of individualized treatments, are
"forward-looking statements" under the securities laws.
Forward-looking statements are based upon current expectations that
involve risks, changes in circumstances, assumptions and
uncertainties. Important factors that could cause actual results to
differ materially from those reflected in Vanda's forward-looking
statements include, among others, the accuracy of the reporting and
diagnosis of CMT and CMT2S cases and the ability to successfully
complete the clinical development of, and obtain regulatory
approval for, VCA-894A in the treatment of CMT2S. Therefore, no
assurance can be given that the results or developments anticipated
by Vanda will be realized or, even if substantially realized, that
they will have the expected consequences to, or effects on, Vanda.
Forward-looking statements in this press release should be
evaluated together with the various risks and uncertainties that
affect Vanda's business and market, particularly those identified
in the "Cautionary Note Regarding Forward-Looking Statements",
"Risk Factors" and "Management's Discussion and Analysis of
Financial Condition and Results of Operations" sections of Vanda's
most recent Annual Report on Form 10-K, as updated by Vanda's
subsequent Quarterly Reports on Form 10-Q, Current Reports on Form
8-K and other filings with the U.S. Securities and Exchange
Commission, which are available at www.sec.gov.
All written and verbal forward-looking statements attributable
to Vanda or any person acting on its behalf are expressly qualified
in their entirety by the cautionary statements contained or
referred to herein. Vanda cautions investors not to rely too
heavily on the forward-looking statements Vanda makes or that are
made on its behalf. The information in this press release is
provided only as of the date of this press release, and Vanda
undertakes no obligation, and specifically declines any obligation,
to update or revise publicly any forward-looking statements,
whether as a result of new information, future events or otherwise,
except as required by law.
Corporate Contact:
Kevin
Moran
Senior Vice President, Chief Financial Officer and Treasurer
Vanda Pharmaceuticals Inc.
202-734-3400
pr@vandapharma.com
View original content to download
multimedia:https://www.prnewswire.com/news-releases/vanda-pharmaceuticals-receives-fda-approval-to-proceed-with-investigational-new-drug-vca-894a-a-novel-antisense-oligonucleotide-candidate-for-the-treatment-of-charcot-marie-tooth-disease-type-2s-302042063.html
SOURCE Vanda Pharmaceuticals Inc.