WASHINGTON, Dec. 20,
2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc.
(Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug
Administration (FDA) has granted Orphan Drug Designation for
VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2
inhibitor for the treatment of polycythemia vera (PV), a form of a
rare hematologic malignancy that is estimated to affect 1 in 2000
Americans.1
PV is a chronic myeloproliferative disorder characterized by
aberrant hematopoiesis of myeloid lineage with exuberant red cell
production and increased release of pro-inflammatory cytokines.
More than 95% of PV patients harbor the JAK2 V617F gain-of-function
mutation leading to aberrant JAK2 production.2
Inhibiting JAK2 acts to suppress hematopoiesis, consequently
reducing red blood cell, neutrophil, platelet, and lymphocyte
production. JAK2 inhibitors have been shown to be efficacious in
treating various JAK-dependent hematologic malignancies, including
the treatment of PV. By selective reduction of JAK2 levels, the ASO
VGT-1849A has the potential to reduce JAK2V617F-driven pathogenic
signaling, ultimately suppressing the malignant proliferation and
survival of hematopoietic cells.
Currently available small molecule inhibitors targeting the JAK2
protein kinase, such as Jakafi®, Inrebic®,
Ojjaara®, and Vonjo®, lack sole selectivity
for the target protein, which can result in off target effects. The
adverse side effects that may occur from JAK inhibition emphasize
the importance of selectively targeting JAK2 while avoiding
inhibition of other JAK family members. By specifically targeting
JAK2, Vanda seeks to reduce the risk of infection and toxic effects
that are seen with inhibitors also blocking JAK1, JAK3, TYK2, or
other kinases outside of the JAK family.
If approved, VGT-1849A could offer targeted efficacy with an
improved safety profile and convenient dosing.
"This orphan designation for VGT-1849A is an important milestone
in precision medicine-based therapeutics in the space of
hematological malignancies. This milestone marks the second
precision medicine therapeutic for Vanda following the development
of VCA-894A for Charcot-Marie-Tooth3 that is expected to
begin clinical testing in the coming months," said Mihael H. Polymeropoulos, M.D., Vanda's
President, CEO and Chairman of the Board.
VGT-1849A is a novel ASO treatment candidate for PV and other
JAK2-driven hematologic malignancies. By selectively targeting
JAK2, VGT-1849A reduces downstream signaling and
JAK2V617F-driven autonomous cell proliferation, without
any off-target kinase effects. The ability of VGT-1849A to reduce
JAK2 activity may alleviate the disease burden that patients with
PV face with a favorable safety profile, resulting in a higher
quality of life for patients.
Orphan Drug Designation is granted by the FDA to investigational
therapies addressing rare medical conditions and provides benefits
to drug developers.
References:
- Grunwald, M. R.; Stein, B. L.; Boccia, R. V.; Oh, S. T.;
Paranagama, D.; Parasuraman, S.; Colucci, P.; Mesa, R. Clinical and
Disease Characteristics From REVEAL at Time of Enrollment
(Baseline): Prospective Observational Study of Patients With
Polycythemia Vera in the United
States. Clin Lymphoma Myeloma Leuk 2018, 18 (12),
788-795.e2. https://doi.org/10.1016/j.clml.2018.08.009.
- P. Gou, W. Zhang, and S. Giraudier, "Insights into the
Potential Mechanisms of JAK2V617F Somatic Mutation Contributing
Distinct Phenotypes in Myeloproliferative Neoplasms,"
Myeloproliferative Neoplasms. Int. J. Mol. Sci, vol. 2022,
p. 1013, 2022, https://doi.org/10.3390/ijms23031013
- S. Smieszek, C. Tyner, A. Kaden, C. Johnson, C.
Polymeropoulos, G. Birznieks, M. Polymeropoulos. Potential
treatment for CMT2S caused by IGHMBP2 cryptic splice variant, with
ASO based therapeutic [abstract]. Mov Disord. 2023; 38
(suppl 1).
https://www.mdsabstracts.org/abstract/potential-treatment-for-cmt2s-caused-by-ighmbp2-cryptic-splice-variant-with-aso-based-therapeutic/.
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 VGT-1849A
VGT-1849A is an antisense oligonucleotide (ASO) that selectively
targets JAK2, reducing increased activity of JAK2 that may cause
hematologic malignancies. ASOs have broad applicability in
addressing a number of disorders caused by genetic variants.
CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
Various statements in this press release, including, but not
limited to statements regarding the estimated prevalence of PV, the
potential therapeutic effects of VGT-1849A, the timing of the
initiation of clinical testing of VCA-894A and the potential
benefits of VGT-1849A, 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 PV cases, the ability of VGT-1849A
to safely and effectively treat PV, Vanda's ability to begin
clinical testing of VCA-894A during the specified timeframe and
Vanda's ability to successfully complete the clinical development
of, and obtain regulatory approval for, VGT-1849A in the treatment
of PV. There can be no assurance that the actual 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. Therefore, no assurance can
be given that the outcomes stated in such forward-looking
statements and estimates will be achieved. 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 Annual Report on Form
10-K for the fiscal year ended December 31,
2023, 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
Jim Golden / Jack Kelleher / Dan
Moore
Collected Strategies
VANDA-CS@collectedstrategies.com
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SOURCE Vanda Pharmaceuticals Inc.
