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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

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Vanda Pharmaceuticals Inc. announced that the FDA has approved the Investigational New Drug application to evaluate VCA-894A for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S. This rare subtype of CMT has no available treatment, and the estimated prevalence of the CMT2S variant is less than 1 in 1,000,000 worldwide. VCA-894A is a novel antisense oligonucleotide that specifically targets a cryptic splice site variant within the IGHMBP2 gene, which plays a pivotal role in the manifestation of CMT2S. The treatment aims to enable personalized medicine and modulate gene expression for rare diseases.
Positive
  • FDA approval for the Investigational New Drug application
  • VCA-894A specifically targets a cryptic splice site variant within the IGHMBP2 gene
  • Potential for personalized medicine and gene expression modulation for rare diseases
Negative
  • None.

The FDA's approval of the Investigational New Drug (IND) application for VCA-894A signifies a potential breakthrough in the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), a rare genetic disorder. As a Medical Research Analyst, the focus is on the scientific and therapeutic implications of this development. Antisense oligonucleotides (ASOs) represent a cutting-edge approach in gene therapy, offering targeted interventions for genetic disorders by modulating gene expression. The specificity of VCA-894A in addressing the cryptic splice site variants within the IGHMBP2 gene could pave the way for innovative treatments for other similarly rare and genetically complex diseases.

From a research perspective, the success of this IND application could stimulate further investment in personalized medicine and genetic therapies. The rarity of CMT2S, with a prevalence of less than 1 in 1,000,000, underscores the importance of developing treatments that can cater to even the most uncommon of conditions. The long-term impact of such advancements could lead to substantial improvements in quality of life for affected individuals and potentially reduce the overall healthcare burden associated with managing untreatable genetic disorders.

The approval of Vanda Pharmaceuticals' IND for VCA-894A by the FDA is a significant milestone with potential financial implications for the company and its investors. As a Biotech Financial Analyst, evaluating the economic viability of investing in treatments for rare diseases such as CMT2S is crucial. While the market for a CMT2S-specific treatment is inherently small, the development of such drugs can benefit from regulatory incentives like orphan drug designation, which can include tax credits, grant funding and extended market exclusivity.

Investors should monitor the progress of VCA-894A's clinical trials for efficacy and safety, which will be critical determinants of the drug's commercial potential. Additionally, success in these trials could enhance Vanda's reputation in the field of rare diseases and potentially lead to partnerships or acquisitions. However, the high costs associated with developing treatments for rare diseases and the uncertainty of market acceptance must be weighed against the potential long-term returns.

The advancement of antisense oligonucleotide therapies, as exemplified by Vanda Pharmaceuticals' VCA-894A, has broader implications for the genetic therapies market. As a Genetic Therapies Market Analyst, it is important to assess the growth trajectory of this sector. The FDA's approval for the IND reflects a growing acceptance of genetic therapies as viable treatment options, which is likely to encourage further research and development in this space.

Market trends indicate an increasing demand for personalized medicine and the success of ASOs in treating neurodegenerative and neuromuscular disorders could lead to expanded applications for a range of genetic conditions. This could result in a significant market expansion for ASOs and similar technologies. Stakeholders should be aware of the competitive landscape, as well as the regulatory and reimbursement challenges that come with introducing novel therapies. The potential for ASOs to address unmet medical needs in rare diseases could drive investment and innovation, reshaping the future of genetic therapies.

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

  1. 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  
  2. 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 
  3. (2023). Charcot-Marie-Tooth disease type 2S. Orpha.net. https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=443073  
  4. 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  
  5. 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

 

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SOURCE Vanda Pharmaceuticals Inc.

The FDA approved the Investigational New Drug application to evaluate VCA-894A for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S.

The estimated prevalence of the CMT2S variant is less than 1 in 1,000,000 worldwide.

VCA-894A is a novel antisense oligonucleotide that specifically targets a cryptic splice site variant within the IGHMBP2 gene.

Mihael H. Polymeropoulos, M.D. is the President, CEO, and Chairman of the Board of Vanda Pharmaceuticals Inc.
Vanda Pharmaceuticals Inc

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vanda is a specialty pharmaceutical company focused on the development and commercialization of novel therapies to address high unmet medical needs and improve the lives of patients.