SELLAS Life Sciences Announces First Pediatric AML Patient Dosed in the Ongoing Phase 2 Trial of SLS009 r/r AML
SELLAS Life Sciences (NASDAQ: SLS) has announced the dosing of its first pediatric patient in the ongoing Phase 2 trial of SLS009 (tambiciclib) for relapsed/refractory acute myeloid leukemia (r/r AML). The trial evaluates SLS009, a highly selective CDK9 inhibitor, in combination with venetoclax and azacitidine at 45mg and 60mg dose levels. The company received FDA Rare Pediatric Disease Designation (RPDD) in July 2024, making it eligible for a Priority Review Voucher upon potential NDA approval, which could be worth approximately $100 million.
The Phase 2 trial targets a 20% response rate with a median survival of at least 3 months. The study includes additional cohorts for ASXL1-mutated AML patients and those with myelodysplasia-related molecular abnormalities.
SELLAS Life Sciences (NASDAQ: SLS) ha annunciato la somministrazione della prima dose a un paziente pediatrico nel corso della sperimentazione di Fase 2 in corso di SLS009 (tambiciclib) per la leucemia mieloide acuta recidivante/refrattaria (r/r AML). Lo studio valuta SLS009, un inibitore altamente selettivo di CDK9, in combinazione con venetoclax e azacitidina a dosaggi di 45 mg e 60 mg. A luglio 2024, l'azienda ha ottenuto la designazione FDA per malattie pediatriche rare (RPDD), che rende idonea la società a ricevere un Voucher di Revisione Prioritaria in caso di approvazione NDA, con un valore potenziale di circa 100 milioni di dollari.
La sperimentazione di Fase 2 punta a un tasso di risposta del 20% con una sopravvivenza mediana di almeno 3 mesi. Lo studio include inoltre coorti aggiuntive per pazienti con AML mutata ASXL1 e per quelli con anomalie molecolari correlate alla mielodisplasia.
SELLAS Life Sciences (NASDAQ: SLS) ha anunciado la administración de la primera dosis a un paciente pediátrico en el ensayo de Fase 2 en curso de SLS009 (tambiciclib) para leucemia mieloide aguda recidivante/refractaria (r/r AML). El ensayo evalúa SLS009, un inhibidor altamente selectivo de CDK9, en combinación con venetoclax y azacitidina en dosis de 45 mg y 60 mg. La compañía recibió en julio de 2024 la Designación de Enfermedad Pediátrica Rara de la FDA (RPDD), lo que la hace elegible para un Voucher de Revisión Prioritaria en caso de aprobación NDA, con un valor potencial aproximado de 100 millones de dólares.
El ensayo de Fase 2 busca un tasa de respuesta del 20% con una supervivencia media de al menos 3 meses. El estudio incluye cohortes adicionales para pacientes con AML mutada en ASXL1 y aquellos con anomalías moleculares relacionadas con mielodisplasia.
SELLAS Life Sciences (NASDAQ: SLS)는 재발/불응성 급성 골수성 백혈병(r/r AML)을 위한 SLS009(탐비시클립)의 진행 중인 2상 임상시험에서 첫 소아 환자에게 투여했다고 발표했습니다. 이 임상시험은 CDK9을 고도로 선택적으로 억제하는 SLS009를 베네토클락스와 아자시티딘과 함께 45mg 및 60mg 용량 수준에서 평가합니다. 회사는 2024년 7월에 FDA 희귀 소아 질환 지정(RPDD)을 받아 잠재적 NDA 승인 시 우선 심사 바우처를 받을 자격이 있으며, 이 바우처의 가치는 약 1억 달러에 달할 수 있습니다.
2상 임상시험은 20% 반응률과 최소 3개월의 중앙 생존 기간을 목표로 합니다. 연구에는 ASXL1 돌연변이 AML 환자와 골수형성이상증 관련 분자 이상을 가진 환자를 위한 추가 코호트도 포함됩니다.
SELLAS Life Sciences (NASDAQ : SLS) a annoncé la première administration à un patient pédiatrique dans l'essai de phase 2 en cours de SLS009 (tambiciclib) pour la leucémie myéloïde aiguë en rechute/réfractaire (r/r LMA). L'essai évalue SLS009, un inhibiteur hautement sélectif de CDK9, en combinaison avec le vénétoclax et l'azacitidine à des doses de 45 mg et 60 mg. La société a obtenu en juillet 2024 la désignation FDA pour maladie pédiatrique rare (RPDD), ce qui la rend éligible à un bon de révision prioritaire en cas d'approbation NDA, pouvant valoir environ 100 millions de dollars.
L'essai de phase 2 vise un taux de réponse de 20% avec une survie médiane d'au moins 3 mois. L'étude comprend également des cohortes supplémentaires pour les patients atteints de LMA mutée ASXL1 et ceux présentant des anomalies moléculaires liées à la myélodysplasie.
SELLAS Life Sciences (NASDAQ: SLS) hat die Dosierung des ersten pädiatrischen Patienten in der laufenden Phase-2-Studie von SLS009 (tambiciclib) bei rezidivierter/refraktärer akuter myeloischer Leukämie (r/r AML) bekannt gegeben. Die Studie bewertet SLS009, einen hochselektiven CDK9-Inhibitor, in Kombination mit Venetoclax und Azacitidin in Dosierungen von 45 mg und 60 mg. Das Unternehmen erhielt im Juli 2024 die FDA Rare Pediatric Disease Designation (RPDD), wodurch es für einen Priority Review Voucher bei möglicher NDA-Zulassung berechtigt ist, der einen Wert von etwa 100 Millionen US-Dollar haben könnte.
Die Phase-2-Studie zielt auf eine 20%ige Ansprechrate mit einer medianen Überlebenszeit von mindestens 3 Monaten ab. Die Studie umfasst zudem zusätzliche Kohorten für Patienten mit ASXL1-mutierter AML und solche mit myelodysplasiebezogenen molekularen Anomalien.
- FDA Rare Pediatric Disease Designation (RPDD) granted, potentially leading to a Priority Review Voucher worth ~$100M
- Expansion of trial to include pediatric patients demonstrates progress in development
- Multiple dosing cohorts and combinations being tested to optimize treatment
- Modest target response rate of only 20%
- Limited target median survival of just 3 months
Insights
SELLAS reaches milestone in pediatric AML trial with potential for valuable Priority Review Voucher if successful.
This dosing of the first pediatric ASXL1-mutated Acute Myeloid Leukemia (AML) patient marks a significant expansion of SELLAS's ongoing Phase 2 trial of SLS009 (tambiciclib). The CDK9 inhibitor is being evaluated in relapsed/refractory AML patients, a population with notoriously poor outcomes and limited treatment options.
The expansion into pediatric patients is particularly noteworthy as SELLAS received Rare Pediatric Disease Designation (RPDD) for this indication in July 2024. This designation provides a crucial regulatory advantage - if an eventual New Drug Application is approved, SELLAS would receive a Priority Review Voucher that could be sold for approximately
The ongoing Phase 2 trial employs a combination strategy of SLS009 with venetoclax and azacitidine, exploring two dose levels (45mg and 60mg). The 60mg cohort utilizes two different dosing schedules - either once weekly or 30mg twice weekly. This adaptive design suggests the company is carefully optimizing the dosing regimen.
The trial has modest efficacy targets - a
- First ASXL1 Pediatric Acute Myeloid Leukemia (AML) Patient Dosed at MD Anderson Cancer Center: Program Supported by Rare Pediatric Disease Designation (RPDD)
NEW YORK, May 15, 2025 (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 first pediatric AML patient has been dosed in the ongoing Phase 2 trial of SLS009 (tambiciclib), a highly selective CDK9 inhibitor, in relapsed/refractory acute myeloid leukemia (r/r AML).
“Building upon our promising Cohort 3 data, we are pleased to dose our first pediatric AML patient as part of the ongoing Phase 2 trial,” said Dragan Cicic, MD, Chief Development Officer of SELLAS. “This milestone reflects our commitment to addressing critical unmet needs in hematologic disorders as we develop treatments for the most difficult to treat patients, particularly pediatric patients, with very few available options, including multi-hit TP53 mutation, failure of azacitidine and venetoclax, failure of transplant, and almost all available high-intensity chemotherapies. With the Rare Pediatric Disease Designation already in place, we are hopeful that our work will bring meaningful progress and potential regulatory advantages as we continue to advance this important program.”
SELLAS was granted the FDA RPDD for the treatment of pediatric AML in July 2024. If, in the future, a New Drug Application (NDA) for SLS009 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
The Phase 2 clinical trial of SLS009 is an open-label, single-arm, multi-center study designed to evaluate the safety, tolerability, and efficacy of SLS009 in combination with venetoclax and azacitidine at two dose levels, 45 and 60 mg. In the 60 mg dose cohort, patients were treated at either a 60 mg dose once per week or a 30 mg dose two times per week. The trial was expanded to include two additional cohorts, one with ASXL1-mutated AML patients and one with patients with myelodysplasia-related molecular abnormalities other than ASXL1. The target response rate at the optimal dose level is
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 (tambiciclib) - 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 20, 2025 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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