Actuate Therapeutics Advances Clinical Program in Ewing Sarcoma After Positive Phase 1 Trial Demonstrates Complete and Partial Responses in Difficult-to-Treat Pediatric Sarcomas
Actuate Therapeutics (NASDAQ:ACTU) has announced positive results from its Phase 1 trial evaluating elraglusib in pediatric patients with refractory malignancies. The study demonstrated remarkable outcomes in Ewing Sarcoma (EWS), with 2 out of 10 patients achieving complete responses and two additional patients showing stable disease.
The trial also reported a partial response in a patient with desmoplastic small-round-cell tumor (DSRCT). Based on these encouraging results, Actuate plans to advance to a Phase 2 trial in children, adolescents, and adults with relapsed/refractory EWS, expected to initiate in 2026, subject to funding.
Elraglusib, which targets glycogen synthase kinase-3 beta (GSK-3尾), could potentially become a first-in-class therapy for EWS, where no approved targeted therapies currently exist.
Actuate Therapeutics (NASDAQ:ACTU) ha annunciato risultati positivi dal suo trial di Fase 1 che valuta elraglusib in pazienti pediatrici con malignit脿 refrattarie. Lo studio ha mostrato risultati notevoli nel sarcoma di Ewing (EWS), con 2 pazienti su 10 che hanno ottenuto risposte complete e altri due pazienti con malattia stabile.
Il trial ha inoltre riportato una risposta parziale in un paziente con tumore a piccole cellule rotonde desmoplastico (DSRCT). Sulla base di questi risultati incoraggianti, Actuate prevede di passare a un trial di Fase 2 su bambini, adolescenti e adulti con EWS recidivante/refrattario, con inizio previsto nel 2026, soggetto a finanziamenti.
Elraglusib, che agisce sul glicogeno sintasi chinasi-3 beta (GSK-3尾), potrebbe diventare una terapia innovativa per l鈥橢WS, dove attualmente non esistono terapie mirate approvate.
Actuate Therapeutics (NASDAQ:ACTU) ha anunciado resultados positivos de su ensayo de Fase 1 que eval煤a elraglusib en pacientes pedi谩tricos con malignidades refractarias. El estudio mostr贸 resultados notables en sarcoma de Ewing (EWS), con 2 de 10 pacientes logrando respuestas completas y dos pacientes adicionales con enfermedad estable.
El ensayo tambi茅n report贸 una respuesta parcial en un paciente con tumor desmopl谩sico de c茅lulas peque帽as redondas (DSRCT). Bas谩ndose en estos alentadores resultados, Actuate planea avanzar a un ensayo de Fase 2 en ni帽os, adolescentes y adultos con EWS en reca铆da/refractario, previsto para iniciar en 2026, sujeto a financiamiento.
Elraglusib, que act煤a sobre la quinasa 3 beta de la s铆ntesis de gluc贸geno (GSK-3尾), podr铆a convertirse en una terapia pionera para el EWS, donde actualmente no existen terapias dirigidas aprobadas.
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霕愴暅, 鞁滍棙鞐愳劀電� 靹湢氇劯韽� 靻岉槙 鞗愴槙 靹疙彫 膦呾枒(DSRCT) 頇橃瀽 1氇呾棎靹� 攵�攵� 氚橃潙霃� 氤搓碃霅橃棃鞀惦媹雼�. 鞚措煬頃� 瓿犽鞝侅澑 瓴瓣臣毳� 氚旐儠鞙茧 Actuate電� 2026雲� 鞛愱笀 歆�鞗� 鞐秬鞐� 霐半澕 鞛皽/雮滌箻靹� EWS毳� 臧�歆� 鞏措Π鞚�, 觳唽雲� 氚� 靹膘澑鞚� 雽�靸侅溂搿� 頃橂姅 2靸� 鞁滍棙鞚� 瓿勴殟頃橁碃 鞛堨姷雼堧嫟.
elraglusib鞚� 旮�毽綌瓴� 頃╈劚 須唽 韨る倶鞝�-3 氩犿儉(GSK-3尾)毳� 響滌爜鞙茧 頃橂┌, 順勳灛 鞀轨澑霅� 響滌爜 旃橂鞝滉皜 鞐嗠姅 EWS鞐� 雽�頃� 斓滌磮鞚� 順侅嫚 旃橂氩�鞚� 霅� 臧�電レ劚鞚� 鞛堨姷雼堧嫟.
Actuate Therapeutics (NASDAQ:ACTU) a annonc茅 des r茅sultats positifs issus de son essai de Phase 1 茅valuant l鈥檈lraglusib chez des patients p茅diatriques atteints de tumeurs malignes r茅fractaires. L鈥櫭﹖ude a d茅montr茅 des r茅sultats remarquables dans le sarcome d鈥橢wing (EWS), avec 2 patients sur 10 ayant obtenu des r茅ponses compl猫tes et deux autres pr茅sentant une maladie stable.
L鈥檈ssai a 茅galement rapport茅 une r茅ponse partielle chez un patient atteint de tumeur 脿 petites cellules rondes desmoplastique (DSRCT). Sur la base de ces r茅sultats encourageants, Actuate pr茅voit de lancer un essai de Phase 2 chez les enfants, adolescents et adultes atteints d鈥橢WS en rechute/r茅fractaire, pr茅vu pour d茅buter en 2026, sous r茅serve de financement.
Elraglusib, qui cible la glycog猫ne synthase kinase-3 b锚ta (GSK-3尾), pourrait devenir une th茅rapie innovante pour l鈥橢WS, maladie pour laquelle aucune th茅rapie cibl茅e approuv茅e n鈥檈xiste actuellement.
Actuate Therapeutics (NASDAQ:ACTU) hat positive Ergebnisse aus seiner Phase-1-Studie zur Bewertung von Elraglusib bei p盲diatrischen Patienten mit refrakt盲ren Malignit盲ten bekannt gegeben. Die Studie zeigte bemerkenswerte Ergebnisse beim Ewing-Sarkom (EWS), wobei 2 von 10 Patienten vollst盲ndige Remissionen erzielten und zwei weitere Patienten eine stabile Erkrankung zeigten.
Die Studie berichtete au脽erdem 眉ber eine partielle Remission bei einem Patienten mit desmoplastischem kleinrundigem Zelltumor (DSRCT). Basierend auf diesen ermutigenden Ergebnissen plant Actuate einen Phase-2-Studie bei Kindern, Jugendlichen und Erwachsenen mit rezidiviertem/refrakt盲rem EWS, die voraussichtlich 2026 beginnen soll, vorbehaltlich der Finanzierung.
Elraglusib, das die Glykogensynthase-Kinase-3 Beta (GSK-3尾) hemmt, k枚nnte eine erstmalige Therapieklasse f眉r EWS werden, f眉r die derzeit keine zugelassenen zielgerichteten Therapien existieren.
- Complete responses achieved in 2 out of 10 refractory Ewing Sarcoma patients
- Additional 2 patients showed durable stable disease
- Partial response observed in DSRCT patient
- Potential first-in-class therapy in an indication with no approved targeted treatments
- Small sample size of only 10 patients in the trial
- Phase 2 trial initiation dependent on funding availability
- Phase 2 trial not starting until 2026
Insights
Actuate's elraglusib shows remarkable complete responses in refractory Ewing Sarcoma, positioning it as potential breakthrough therapy where options are scarce.
The Phase 1 results for Actuate's elraglusib represent a potentially significant breakthrough in treating refractory Ewing Sarcoma (EWS), one of the most challenging pediatric cancers. The observation of complete responses in 2 out of 10 heavily pre-treated EWS patients is exceptional, as this aggressive bone and soft tissue cancer typically shows dismal outcomes after relapse. Additionally, 2 patients achieved durable stable disease, and there was a partial response in a patient with desmoplastic small-round-cell tumor (DSRCT), another rare and aggressive sarcoma.
What makes these results particularly compelling is the mechanism of action - elraglusib targets glycogen synthase kinase-3 beta (GSK-3尾), representing a novel approach in sarcoma treatment. Currently, patients with relapsed/refractory EWS face extremely limited options with poor survival rates, typically less than 20% at 5 years. The complete responses observed here are exceedingly rare in the refractory setting where patients have already failed multiple lines of therapy.
The combination of elraglusib with topotecan/cyclophosphamide appears to enhance efficacy beyond what we typically see with conventional chemotherapy alone. While the sample size of 10 EWS patients is small, even this limited data suggests meaningful clinical activity worthy of the planned Phase 2 expansion. The company's strategy to include pediatric, adolescent, and adult patients in the upcoming trial is appropriate given EWS affects primarily children and young adults but can occur across age groups.
This represents a potential first-in-class targeted therapy in an indication that desperately needs new approaches. However, the planned 2026 trial initiation timeline, contingent on funding, suggests commercialization remains years away, assuming success in later-stage trials.
- Announces Completion of Phase 1 Study of Elraglusib in Pediatric Patients
- Prolonged and Durable Complete Responses (CRs) Observed in Two of Ten Refractory Ewing Sarcoma Patients
- Two Additional Patients Had Durable Stable Disease in Notoriously Difficult-to-Treat Cancer
- Confirmed Partial Response (PR) in a patient with a desmoplastic small-round-cell tumor (DSRCT)
- Company Initiating Planning of Phase 2 Trial of Elraglusib in Children, Adolescents, and Adults with Refractory/Resistant Ewing Sarcoma
CHICAGO and FORT WORTH, Texas, July 17, 2025 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) (鈥淎ctuate鈥� or the 鈥淐ompany鈥�), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3尾), today announced the end of the Phase 1 portion of its clinical study evaluating elraglusib monotherapy or in combination with irinotecan, irinotecan plus temozolomide, or with cyclophosphamide plus topotecan in pediatric patients with refractory malignancies (Actuate-1902). Following encouraging signals of activity, particularly in treatment-refractory Ewing Sarcoma (EWS), a small round cell sarcoma that forms in soft tissue and bone, the Company will seek to advance the clinical development program towards a Phase 2 study in children, adolescents, and adults with relapsed/refractory EWS.
鈥淩efractory and resistant EWS is one of the most aggressive and underserved cancers affecting children and young adults with very low survival rates,鈥� said Dr. Andrew Mazar, Actuate鈥檚 Scientific Co-founder and Chief Operating Officer. 鈥淭o observe complete responses in this setting is highly unexpected, and these exceptional early results position elraglusib as a potential first-in-class therapy option in an indication where no approved targeted therapies currently exist.鈥�
The Phase 1/2 Trial (), also referred to as Actuate-1902, was an open-label, multicenter study evaluating the safety and efficacy of elraglusib in pediatric patients with relapsed/refractory malignancies, including EWS and other pediatric sarcomas. To date, the study has enrolled ten (10) patients with relapsed/refractory EWS (>1 remission) treated with the combination of elraglusib and topotecan/cyclophosphamide.
鈥淲e are highly encouraged by the responses observed in refractory and resistant EWS, a disease where treatment options are extremely limited beyond frontline chemotherapy,鈥� said Daniel Schmitt, President & Chief Executive Officer of Actuate. 鈥淲ith these promising clinical signals in hand, we are advancing our development plans focused on EWS by pursuing a Phase 2 trial and plan to meet with regulators to discuss potential registration path in this indication.鈥�
Of the ten (10) EWS patients enrolled, two (2) patients achieved complete responses by CT and/or complete metabolic responses (CR/CMR), while two (2) had confirmed stable disease. A partial response (PR) was also observed in one patient with a desmoplastic small-round-cell tumor (DSRCT), a small round cell sarcoma that forms in soft tissue. While the sample size specific to Ewing Sarcoma in the 1902 study was small, the responses are viewed as positive evidence of anti-tumor activity in this difficult-to-treat indication, and Actuate plans to further investigate elraglusib鈥檚 ability to positively change patients鈥� potential for successful treatment in a Phase 2 trial in pediatric, adolescent, and adult patients with relapsed/refractory Ewing Sarcoma. The Company is collaborating with key opinion leaders and consortiums with interest in EWS to ensure the design of the upcoming study aligns with both patient needs and regulatory expectations. The Company expects to initiate the trial in 2026, subject to available funding.
About Ewing Sarcoma
Ewing sarcoma (EWS) is a highly metastatic form of sarcoma with a peak incidence at the age of 15, that ranks as the second most prevalent primary malignant tumor of childhood and adolescence. Approximately
About Actuate Therapeutics, Inc.
Actuate is a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers. Actuate鈥檚 lead investigational drug, elraglusib (a novel GSK-3尾聽inhibitor), targets molecular pathways in cancer that are involved in promoting tumor growth and resistance to conventional cancer drugs such as chemotherapy through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and DNA Damage Response (DDR). Elraglusib may also mediate anti-tumor immunity through the regulation of multiple immune checkpoints and immune cell function. For additional information, please visit the Company鈥檚 website at http://www.actuatetherapeutics.com.
Forward-Looking Statements
This press release contains forward-looking statements about us, including our and other parties鈥� clinical trials and development plans, and our industry. The words 鈥渁nticipate,鈥� 鈥渂elieve,鈥� 鈥渃ontinue,鈥� 鈥渃ould,鈥� 鈥渆stimate,鈥� 鈥渆xpect,鈥� 鈥渋ntend,鈥� 鈥渕ay,鈥� 鈥渕ight,鈥� 鈥渙ngoing,鈥� 鈥減lan,鈥� 鈥減otential,鈥� 鈥減redict,鈥� 鈥減roject,鈥� 鈥渟hould,鈥� 鈥渢arget,鈥� 鈥渨ill,鈥� 鈥渨ould,鈥� or the negative of these terms or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements, other than statements related to present facts or current conditions or of historical facts, contained in this press release are forward-looking statements. Accordingly, these statements involve estimates, assumptions, substantial risks and uncertainties which could cause actual results to differ materially from those expressed in them, including but not limited to that preliminary and unpublished data may be subject to change and further interpretation following the availability of more data or following a more comprehensive review of the data and should not be relied upon as a final analysis; clinical and preclinical drug development involves a lengthy and expensive process with uncertain timelines and outcomes, results of prior preclinical studies, early clinical trials and sub-group studies are not necessarily predictive of future results and may not correlate with improved responses, and elraglusib may not achieve positive clinical results or favorable preclinical results or receive regulatory approval on a timely basis, if at all; that we may not successfully enroll additional patients or establish or advance plans for further development, including through conversations with the FDA or EMA and the standards such bodies may impose for such development; that elraglusib could be associated with side effects, adverse events or other properties or safety risks, which could delay or preclude regulatory approval, cause us to suspend or discontinue clinical trials or result in other negative consequences; our reliance on third parties to conduct our non-clinical studies and our clinical trials; our reliance on third-party licensors and ability to preserve and protect our intellectual property rights; that we face significant competition from other biotechnology and pharmaceutical companies; our ability to fund development activities, including because our financial condition raises substantial doubt as to our ability to continue as a going concern and we require additional capital to finance our operations for fiscal year 2025, and a failure to obtain this necessary capital in the near term on acceptable terms, or at all, could force us to delay, limit, reduce or terminate our development programs, commercialization efforts or other operations. In addition, any forward-looking statements are qualified in their entirety by reference to the factors discussed under the heading 鈥淚tem 1A. Risk Factors鈥� in our Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 13, 2025, and our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the SEC on May 15, 2025, and other filings with the SEC. Because the risk factors referred to above could cause actual results or outcomes to differ materially from those expressed in any forward-looking statements made by us or on our behalf, you should not place undue reliance on any forward-looking statements. Further, any forward-looking statement speaks only as of the date on which it is made. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Unless legally required, we do not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date of this press release or to reflect the occurrence of unanticipated events.
Investor Contact
Mike Moyer
Managing Director
LifeSci Advisors, LLC
[email protected]
Media Contact
Ignacio Guerrero-Ros, Ph.D., or David Schull
Russo Partners, LLC
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FAQ
What were the key results of Actuate Therapeutics' (ACTU) Phase 1 trial in Ewing Sarcoma?
When will Actuate Therapeutics (ACTU) begin its Phase 2 trial for elraglusib in Ewing Sarcoma?
What makes elraglusib unique for treating Ewing Sarcoma?
How many patients were treated in ACTU's Phase 1 trial for Ewing Sarcoma?
What is the current treatment landscape for Ewing Sarcoma that ACTU is addressing?
