SELLAS Announces U.S. FDA Rare Pediatric Disease Designation (RPDD) Granted to Galinpepimut-S (GPS) for the Treatment of Pediatric Acute Myeloid Leukemia
SELLAS Life Sciences Group (NASDAQ: SLS) has received Rare Pediatric Disease Designation (RPDD) from the FDA for Galinpepimut-S (GPS) in treating pediatric acute myeloid leukemia (AML). GPS, an immunotherapeutic targeting Wilms Tumor-1, is currently in a Phase 3 REGAL trial for adult AML patients, with an interim analysis expected in Q4 2024.
The RPDD makes GPS eligible for a Priority Review Voucher (PRV) upon marketing approval, which can be transferred or sold, with recent valuations around $100 million. GPS has shown promise in clinical settings, particularly in younger patients. In a Phase 2 trial with adult AML patients, GPS demonstrated a median overall survival of 67.6 months across all ages, with even better outcomes in younger patients.
AML prognosis in refractory/relapsed pediatric patients remains poor, with a 5-year overall survival rate of 33% for all patients, and only 15.7% for those whose remission lasted less than 12 months. About 50% of children with pediatric AML relapse, highlighting the need for new treatment options.
SELLAS Life Sciences Group (NASDAQ: SLS) ha ricevuto la Designazione di Malattia Pediatrica Rara (RPDD) dalla FDA per il Galinpepimut-S (GPS) nel trattamento della leucemia mieloide acuta pediatrica (AML). GPS, un'immunoterapia che mira a Wilms Tumor-1, è attualmente in fase di trial REGAL di Fase 3 per pazienti adulti con AML, con un'analisi intermedia prevista per il quarto trimestre del 2024.
La RPDD rende GPS idonea per un Voucher di Revisione Prioritaria (PRV) al momento dell'approvazione al commercio, il quale può essere trasferito o venduto, con valutazioni recenti intorno ai 100 milioni di dollari. GPS ha mostrato promettenti risultati in contesti clinici, in particolare nei pazienti più giovani. In un trial di Fase 2 con pazienti adulti affetti da AML, GPS ha dimostrato una sopravvivenza globale mediana di 67,6 mesi tra tutte le età, con risultati ancora migliori nei pazienti più giovani.
La prognosi dell'AML nei pazienti pediatrici refrattari/recidivati rimane scarsa, con un tasso di sopravvivenza globale a 5 anni del 33% per tutti i pazienti, e solo del 15,7% per quelli la cui remissione è durata meno di 12 mesi. Circa il 50% dei bambini con AML pediatrica recidiva, evidenziando la necessità di nuove opzioni di trattamento.
SELLAS Life Sciences Group (NASDAQ: SLS) ha recibido la Designación de Enfermedad Pediátrica Rara (RPDD) de la FDA para Galinpepimut-S (GPS) en el tratamiento de leucemia mieloide aguda pediátrica (AML). GPS, una inmunoterapia que se dirige a Wilms Tumor-1, actualmente se encuentra en un ensayo REGAL de Fase 3 para pacientes adultos con AML, con un análisis interino esperado para el cuarto trimestre de 2024.
La RPDD hace que GPS sea elegible para un Vale de Revisión Prioritaria (PRV) tras la aprobación de comercialización, que puede ser transferido o vendido, con valoraciones recientes alrededor de 100 millones de dólares. GPS ha mostrado promesas en entornos clínicos, especialmente en pacientes más jóvenes. En un ensayo de Fase 2 con pacientes adultos de AML, GPS demostró una supervivencia general mediana de 67.6 meses entre todas las edades, con resultados aún mejores en pacientes más jóvenes.
El pronóstico de AML en pacientes pediátricos refractarios/recidivantes sigue siendo pobre, con una tasa de supervivencia global a 5 años del 33% para todos los pacientes, y solo del 15.7% para aquellos cuya remisión duró menos de 12 meses. Alrededor del 50% de los niños con AML pediátrica recaen, lo que resalta la necesidad de nuevas opciones de tratamiento.
SELLAS Life Sciences Group (NASDAQ: SLS)는 소아 급성 골수성 백혈병(AML) 치료를 위한 Galinpepimut-S (GPS)에 대해 FDA로부터 희귀 소아 질병 지정(RPDD)를 받았습니다. Wilms Tumor-1을 표적하는 면역 요법인 GPS는 현재 성인 AML 환자를 대상으로 하는 3상 REGAL 임상 시험 중에 있으며, 2024년 4분기에 중간 분석이 예정되어 있습니다.
RPDD는 GPS가 마케팅 승인 후 우선 검토 바우처(PRV)에 적격이 되도록 하며, 이 바우처는 양도하거나 판매할 수 있습니다. 최근 평가액은 약 1억 달러입니다. GPS는 특히 어린 환자들에서 임상 환경에서 가능성을 보였습니다. 성인 AML 환자를 대상으로 한 2상 시험에서, GPS는 모든 연령대에서 중간 전체 생존 기간이 67.6개월로 나타났으며, 특히 어린 환자들에서 더 나은 결과를 보였습니다.
재발/불응 소아 환자에서 AML의 예후는 여전히 불량하며, 모든 환자에 대해 5년 전체 생존율은 33%, 12개월 미만의 관해 기간을 가진 환자에 대해서는 15.7%에 불과합니다. 소아 AML 환자의 약 50%가 재발하며, 이는 새로운 치료 옵션의 필요성을 강조합니다.
SELLAS Life Sciences Group (NASDAQ: SLS) a reçu la Désignation de Maladie Pédiatrique Rare (RPDD) de la FDA pour le Galinpepimut-S (GPS) dans le traitement de la leucémie myéloïde aiguë pédiatrique (AML). GPS, une immunothérapie ciblant Wilms Tumor-1, est actuellement en essai REGAL de Phase 3 pour les patients adultes atteints d'AML, avec une analyse intermédiaire prévue pour le quatrième trimestre 2024.
La RPDD rend GPS éligible à un Bon de Révision Prioritaire (PRV) après approbation de la commercialisation, qui peut être transféré ou vendu, avec une valorisation récente autour de 100 millions de dollars. GPS a montré des résultats prometteurs dans des contextes cliniques, notamment chez les patients plus jeunes. Dans un essai de Phase 2 avec des patients adultes atteints d'AML, GPS a démontré une survie globale médiane de 67,6 mois tous âges confondus, avec des résultats encore meilleurs chez les plus jeunes patients.
Le pronostic de l'AML chez les patients pédiatriques réfractaires/récidivants reste mauvais, avec un taux de survie global à 5 ans de 33 % pour tous les patients, et seulement 15,7 % pour ceux dont la rémission a duré moins de 12 mois. Environ 50 % des enfants atteints d'AML pédiatrique rechutent, ce qui souligne la nécessité de nouvelles options de traitement.
SELLAS Life Sciences Group (NASDAQ: SLS) hat von der FDA die Rare Pediatric Disease Designation (RPDD) für Galinpepimut-S (GPS) zur Behandlung von pediatrischer akuter myeloischer Leukämie (AML) erhalten. GPS, eine immuntherapeutische Behandlung, die auf Wilms Tumor-1 abzielt, befindet sich derzeit in einer Phase-3 REGAL-Studie für erwachsene AML-Patienten, mit einer Zwischenanalyse, die für das vierte Quartal 2024 erwartet wird.
Die RPDD macht GPS für einen Prioritätsüberprüfungs-Gutschein (PRV) nach der Marktzulassung berechtigt, der übertragen oder verkauft werden kann, wobei die jüngsten Bewertungen bei etwa 100 Millionen Dollar liegen. GPS hat in klinischen Studien vielversprechende Ergebnisse gezeigt, insbesondere bei jüngeren Patienten. In einer Phase-2-Studie mit erwachsenen AML-Patienten zeigte GPS eine mediane Gesamtüberlebenszeit von 67,6 Monaten über alle Altersgruppen hinweg, mit noch besseren Ergebnissen bei jüngeren Patienten.
Die Prognose für AML bei refraktären/rückfälligen pädiatrischen Patienten bleibt schlecht, mit einer 5-Jahres-Gesamtüberlebensrate von 33 % für alle Patienten und nur 15,7 % für jene, deren Remission weniger als 12 Monate dauerte. Etwa 50 % der Kinder mit pädiatrischer AML fallen zurück, was die Notwendigkeit neuer Behandlungsoptionen unterstreicht.
- FDA granted Rare Pediatric Disease Designation (RPDD) for GPS in pediatric AML treatment
- Eligibility for Priority Review Voucher (PRV) upon marketing approval, valued at ~$100 million
- Phase 3 REGAL trial for GPS in adult AML patients ongoing, with interim analysis expected in Q4 2024
- Phase 2 trial showed median overall survival of 67.6 months across all ages for GPS in adult AML patients
- Better outcomes observed in younger patients in Phase 2 trial, suggesting potential efficacy in pediatric population
- GPS not yet approved for pediatric AML treatment
- Phase 3 trial results still pending, with interim analysis not expected until Q4 2024
- Current poor prognosis for pediatric AML patients, especially in relapsed/refractory cases
Insights
The FDA's Rare Pediatric Disease Designation (RPDD) for Galinpepimut-S (GPS) in pediatric AML is a significant development. This designation highlights the unmet medical need in pediatric AML treatment and positions GPS as a potential breakthrough therapy. The Phase 2 results in adult AML patients, especially the improved outcomes in younger patients, are particularly encouraging for pediatric applications.
Key points to consider:
- GPS's mechanism of action, targeting Wilms Tumor-1 (WT1), could be more effective in younger patients due to their more robust immune systems.
- The potential for a Priority Review Voucher (PRV), valued at approximately
$100 million , adds significant financial incentive for SELLAS. - The ongoing Phase 3 REGAL trial in adult AML, with interim analysis expected in Q4 2024, will be important for validating GPS's efficacy.
While promising, it's important to note that pediatric trials are still needed to confirm GPS's safety and efficacy in children specifically.
This RPDD grant for GPS in pediatric AML is a significant milestone for SELLAS, potentially accelerating the drug's path to market and enhancing its commercial value. Key financial implications include:
- Eligibility for a Priority Review Voucher (PRV) upon FDA approval, which could be sold for approximately
$100 million , a substantial sum compared to SELLAS's current market cap of about$80 million . - Potential for expedited review process, possibly leading to earlier market entry and revenue generation.
- Enhanced attractiveness to potential partners or acquirers, given the added value of the RPDD and potential PRV.
Investors should monitor the Phase 3 REGAL trial results closely, as positive outcomes could significantly impact SELLAS's stock price. However, it's important to remember that clinical success is not guaranteed and the company's small market cap suggests high risk alongside high potential reward.
- GPS Currently Investigated in Phase 3 REGAL Trial in Adult AML Patients – Interim Analysis Anticipated in Q4 2024 -
- RPDD Provides Eligibility for GPS to Receive a Priority Review Voucher (PRV) Upon Marketing Approval that can be Transferred/Sold to Other Parties –
- Recent Valuations for PRVs Remain Attractive (~
NEW YORK, Oct. 15, 2024 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS” or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to Galinpepimut-S (GPS), an immunotherapeutic targeting Wilms Tumor-1 (WT1), for the treatment of pediatric acute myeloid leukemia (AML).
“GPS has already demonstrated promise in clinical settings for AML, and we believe its potential could extend to pediatric patients,” said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. “Receiving RPDD from the FDA is another acknowledgment of the critical need for new treatment options for AML and our results in adult patients. In our Phase 2 trial in adult patients which included patients as young as 25, clinical benefits were significantly higher in younger patients, which was expected based on the mechanism of action of GPS that is mediated via the immune system that is generally better preserved in younger patients, and even more so in children. With both of our development candidates, GPS and SLS009, now granted RPDD for AML, this recognition further reinforces our commitment to delivering potential new therapies to children affected by this challenging condition.”
AML prognosis with currently available treatments in the refractory and/or relapsed pediatric patient population remains poor. In a representative study, the 5-year overall survival (OS) rate in relapsed pediatric AML was
In adult AML patients in first complete remission, GPS showed a median OS of 67.6 months across all ages with a favorable safety profile in an earlier Phase 2 study and induced T-lymphocytes response in both cytotoxic CD8+ cells and memory and helper CD4-+ cells with its innovative heteroclitic technology. In that study, outcomes were even better in younger patients in whom neither the median disease-free survival (DFS) nor OS was reached, i.e. among younger patients more than half of the patients were alive and leukemia-free for more than 5 years after treatment commenced.
Rare Pediatric Disease Designation is granted by the FDA for serious or life-threatening diseases that affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If, in the future, a New Drug Application (NDA) for GPS for the treatment of pediatric AML is approved by the FDA, SELLAS will be eligible to receive a Priority Review Voucher (PRV) that could be redeemed to receive a priority review for any subsequent marketing application. PRVs may be used by the sponsor or sold to another sponsor for their use and have recently sold for approximately
About SELLAS Life Sciences Group, Inc.
SELLAS is a late-stage clinical biopharmaceutical company focused on the development of novel therapeutics for a broad range of cancer indications. SELLAS’ lead product candidate, GPS, is licensed from Memorial Sloan Kettering Cancer Center and targets the WT1 protein, which is present in an array of tumor types. GPS has the potential as a monotherapy and combination with other therapies to address a broad spectrum of hematologic malignancies and solid tumor indications. The Company is also developing SLS009 (formerly GFH009) - potentially the first and best-in-class differentiated small molecule CDK9 inhibitor with reduced toxicity and increased potency compared to other CDK9 inhibitors. Data suggests that SLS009 demonstrated a high response rate in AML patients with unfavorable prognostic factors including ASXL1 mutation, commonly associated with poor prognosis in various myeloid diseases. For more information on SELLAS, please visit www.sellaslifesciences.com.
Forward-Looking Statements
This press release contains forward-looking statements. All statements other than statements of historical facts are “forward-looking statements,” including those relating to future events. In some cases, forward-looking statements can be identified by terminology such as “plan,” “expect,” “anticipate,” “may,” “might,” “will,” “should,” “project,” “believe,” “estimate,” “predict,” “potential,” “intend,” or “continue” and other words or terms of similar meaning. These statements include, without limitation, statements related to the GPS clinical development program, including the REGAL study and the timing of future milestones related thereto. These forward-looking statements are based on current plans, objectives, estimates, expectations, and intentions, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties with oncology product development and clinical success thereof, the uncertainty of regulatory approval, and other risks and uncertainties affecting SELLAS and its development programs as set forth under the caption “Risk Factors” in SELLAS’ Annual Report on Form 10-K filed on March 28, 2024 and in its other SEC filings. Other risks and uncertainties of which SELLAS is not currently aware may also affect SELLAS’ forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof. SELLAS undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations, or other circumstances that exist after the date as of which the forward-looking statements were made.
Investor Contact
Bruce Mackle
Managing Director
LifeSci Advisors, LLC
SELLAS@lifesciadvisors.com
FAQ
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How does GPS work in treating AML?
