Vanda Pharmaceuticals Announces Orphan Drug Designation Granted for VGT-1849A, a Novel and Selective Antisense Oligonucleotide Candidate for the Treatment of Polycythemia Vera
Vanda Pharmaceuticals (VNDA) has received FDA Orphan Drug Designation for VGT-1849A, a selective antisense oligonucleotide-based JAK2 inhibitor designed to treat polycythemia vera (PV). PV is a rare blood cancer affecting approximately 1 in 2000 Americans, characterized by excessive red blood cell production.
VGT-1849A specifically targets JAK2, potentially offering advantages over current treatments like Jakafi®, Inrebic®, Ojjaara®, and Vonjo® which lack selective targeting. This specificity could reduce infection risks and toxic effects associated with broader JAK inhibition. The treatment represents Vanda's second precision medicine therapeutic, following VCA-894A for Charcot-Marie-Tooth disease.
Vanda Pharmaceuticals (VNDA) ha ricevuto la Designazione di Farmaco Orfano dalla FDA per VGT-1849A, un inibitore JAK2 basato su oligonucleotidi antisenso selettivi, progettato per trattare la policitemia vera (PV). La PV è un raro cancro del sangue che colpisce circa 1 persona ogni 2000 americani, caratterizzata da una produzione eccessiva di globuli rossi.
VGT-1849A colpisce specificamente JAK2, offrendo potenzialmente vantaggi rispetto ai trattamenti attuali come Jakafi®, Inrebic®, Ojjaara® e Vonjo®, che non mirano in modo selettivo. Questa specificità potrebbe ridurre i rischi di infezione e gli effetti tossici associati a un'inibizione più ampia di JAK. Il trattamento rappresenta il secondo farmaco di precisione sviluppato da Vanda, dopo VCA-894A per la malattia di Charcot-Marie-Tooth.
Vanda Pharmaceuticals (VNDA) ha recibido la Designación de Medicamento Huérfano de la FDA para VGT-1849A, un inhibidor de JAK2 basado en oligonucleótidos antisentido diseñado para tratar la policitemia vera (PV). La PV es un raro cáncer de sangre que afecta aproximadamente a 1 de cada 2000 estadounidenses, caracterizada por una producción excesiva de glóbulos rojos.
VGT-1849A se dirige específicamente a JAK2, ofreciendo potencialmente ventajas sobre los tratamientos actuales como Jakafi®, Inrebic®, Ojjaara® y Vonjo®, que carecen de un enfoque selectivo. Esta especificidad podría reducir los riesgos de infección y los efectos tóxicos asociados con una inhibición más amplia de JAK. El tratamiento representa la segunda terapia de medicina de precisión de Vanda, después de VCA-894A para la enfermedad de Charcot-Marie-Tooth.
반다 제약 (VNDA)는 VGT-1849A에 대해 FDA의 희귀 의약품 지정을 받았습니다. 이는 다발성 적혈구증가증(PV)을 치료하기 위해 설계된 선택적 안티센스 올리고뉴클레오타이드 기반의 JAK2 억제제입니다. PV는 약 2000명 중 1명에게 영향을 미치는 희귀 혈액암으로, 적혈구 과다 생산이 특징입니다.
VGT-1849A는 JAK2를 특정적으로 타겟팅하여 Jakafi®, Inrebic®, Ojjaara®, Vonjo®와 같은 현재 치료법에 비해 장점을 제공할 수 있습니다. 이러한 선택성은 광범위한 JAK 억제와 관련된 감염 위험 및 독성 효과를 줄일 수 있습니다. 이 치료법은 Charcot-Marie-Tooth병을 위한 VCA-894A에 이어 반다의 두 번째 정밀의학 치료제를 나타냅니다.
Vanda Pharmaceuticals (VNDA) a reçu la désignation de médicament orphelin de la FDA pour VGT-1849A, un inhibiteur JAK2 basé sur des oligonucléotides antisens, conçu pour traiter la polycythémie vraie (PV). La PV est un cancer du sang rare qui touche environ 1 personne sur 2000 américains, caractérisé par une production excessive de globules rouges.
VGT-1849A cible spécifiquement JAK2, offrant potentiellement des avantages par rapport aux traitements actuels tels que Jakafi®, Inrebic®, Ojjaara® et Vonjo®, qui manquent de ciblage sélectif. Cette spécificité pourrait réduire les risques d'infection et les effets toxiques associés à une inhibition plus large de JAK. Ce traitement représente le deuxième médicament de précision de Vanda, après VCA-894A pour la maladie de Charcot-Marie-Tooth.
Vanda Pharmaceuticals (VNDA) hat von der FDA die Orphan Drug Designation für VGT-1849A erhalten, einen selektiven auf Antisense-Oligonukleotiden basierenden JAK2-Inhibitor, der zur Behandlung der Polycythaemia vera (PV) entwickelt wurde. PV ist eine seltene Blutkrebserkrankung, die etwa 1 von 2000 Amerikanern betrifft und durch übermäßige Produktion roter Blutkörperchen gekennzeichnet ist.
VGT-1849A zielt spezifisch auf JAK2 ab und könnte potenziell Vorteile gegenüber den aktuellen Behandlungen wie Jakafi®, Inrebic®, Ojjaara® und Vonjo® bieten, die kein selektives Targeting aufweisen. Diese Spezifität könnte das Risiko von Infektionen und toxischen Effekten, die mit einer breiteren JAK-Hemmung verbunden sind, verringern. Die Behandlung stellt Vandas zweite Therapie der präzisen Medizin dar, nach VCA-894A für die Charcot-Marie-Tooth-Krankheit.
- Received FDA Orphan Drug Designation for VGT-1849A
- Potential competitive advantage through selective JAK2 targeting compared to existing treatments
- Addresses rare disease market with 1 in 2000 Americans affected
- Product still in early development phase
- No clinical trial data available yet
- Faces competition from established treatments (Jakafi®, Inrebic®, Ojjaara®, Vonjo®)
Insights
The FDA's Orphan Drug Designation for VGT-1849A represents a strategic advancement in Vanda's pipeline. This antisense oligonucleotide targeting JAK2 shows potential advantages over existing treatments like Jakafi, Inrebic and Ojjaara through its selective mechanism. The key differentiator is VGT-1849A's specificity for JAK2, which could translate to fewer off-target effects and improved safety profiles compared to current JAK inhibitors.
The market opportunity is notable, with polycythemia vera affecting approximately 165,000 Americans (1 in 2,000). Current JAK inhibitors generate substantial revenue - Jakafi alone reported
The designation provides regulatory benefits including tax credits for clinical trials and potential market exclusivity, enhancing the commercial viability of VGT-1849A's development program. This represents Vanda's second precision medicine therapeutic, indicating an expanding specialized pipeline.
The molecular targeting strategy of VGT-1849A addresses a critical medical need in PV treatment. The 95% prevalence of JAK2 V617F mutations in PV patients provides a clear rationale for this targeted approach. The antisense oligonucleotide technology offers precise genetic intervention, potentially reducing the systemic effects seen with traditional kinase inhibitors.
The therapeutic concept is particularly compelling because it targets the disease's root cause - aberrant JAK2 signaling - rather than just managing symptoms. For non-technical readers, think of it as precision-guided therapy that specifically targets the problematic protein while leaving related proteins unaffected, similar to using a sniper rifle instead of a shotgun.
This approach could be especially valuable for patients who experience adverse effects from current treatments or require long-term therapy, potentially offering a better balance of efficacy and tolerability.
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
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
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.
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FAQ
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