Actuate Therapeutics Receives EMA Orphan Medicinal Product Designation for Elraglusib for the Treatment of Pancreatic Cancer
Actuate Therapeutics (NASDAQ: ACTU) has received European Medicines Agency (EMA) Orphan Medicinal Product Designation for elraglusib in treating pancreatic ductal adenocarcinoma (PDAC). This designation follows a similar FDA Orphan Drug Designation and recent positive interim Phase 2 trial results.
The interim analysis of the Phase 2 trial showed that elraglusib, combined with gemcitabine/nab-paclitaxel (GnP), demonstrated statistically significant improvements in both 1-year survival rate (p=0.002) and median overall survival (hazard ratio 0.63, p=0.016) compared to GnP alone in metastatic PDAC patients.
The EMA designation, granted for conditions affecting fewer than 2 in 10,000 EU residents, provides benefits including potential 10-year market exclusivity, reduced regulatory fees, and centralized EU approval process. The company expects to report topline Phase 2 data in first half of 2025.
Actuate Therapeutics (NASDAQ: ACTU) ha ricevuto la Designazione di Medicinale Orfano dalla European Medicines Agency (EMA) per elraglusib nel trattamento dell'adenocarcinoma duttale pancreatico (PDAC). Questa designazione segue una simile Designazione di Farmaco Orfano da parte della FDA e i recenti risultati positivi dell'analisi intermedia della Fase 2.
L'analisi intermedia della Fase 2 ha mostrato che elraglusib, combinato con gemcitabina/nab-paclitaxel (GnP), ha dimostrato miglioramenti statisticamente significativi sia nel tasso di sopravvivenza a 1 anno (p=0.002) sia nella sopravvivenza complessiva mediana (rapporto di rischio 0.63, p=0.016) rispetto a GnP da solo nei pazienti con PDAC metastatico.
La designazione dell'EMA, concessa per condizioni che colpiscono meno di 2 persone ogni 10.000 residenti nell'UE, offre vantaggi tra cui una potenziale esclusività di mercato di 10 anni, riduzione delle tasse di regolamentazione e un processo di approvazione centralizzato nell'UE. L'azienda prevede di riportare i dati principali della Fase 2 nella prima metà del 2025.
Actuate Therapeutics (NASDAQ: ACTU) ha recibido la Designación de Medicamento Huérfano por parte de la Agencia Europea de Medicamentos (EMA) para elraglusib en el tratamiento del adenocarcinoma ductal pancreático (PDAC). Esta designación sigue a una similar Designación de Medicamento Huérfano de la FDA y a recientes resultados intermedios positivos del ensayo de Fase 2.
El análisis intermedio del ensayo de Fase 2 mostró que elraglusib, combinado con gemcitabina/nab-paclitaxel (GnP), demostró mejoras estadísticamente significativas tanto en la tasa de supervivencia a 1 año (p=0.002) como en la supervivencia general mediana (cociente de riesgos 0.63, p=0.016) en comparación con GnP solo en pacientes con PDAC metastásico.
La designación de la EMA, concedida para condiciones que afectan a menos de 2 de cada 10,000 residentes de la UE, ofrece beneficios que incluyen la exclusividad en el mercado por 10 años, reducción de tasas regulatorias y un proceso de aprobación centralizado en la UE. La empresa espera informar sobre los datos de la Fase 2 en la primera mitad de 2025.
Actuate Therapeutics (NASDAQ: ACTU)는 췌장관 선암(PDAC) 치료를 위해 elraglusib에 대해 유럽 의약품 감독청(EMA)에서 희귀의약품 지정을 받았습니다. 이 지명은 FDA의 유사한 희귀의약품 지정 및 최근 긍정적인 중간 2상 시험 결과에 이어 이루어진 것입니다.
2상 시험의 중간 분석에 따르면, elraglusib과 gemcitabine/nab-paclitaxel (GnP) 병용요법이 전이성 PDAC 환자에서 GnP 단독요법에 비해 통계적으로 유의미한 개선을 보여주었으며, 1년 생존율(p=0.002)과 중앙 전체 생존 기간(위험비 0.63, p=0.016) 모두에서 향상을 나타냈습니다.
EMA의 지명은 1만 명당 2명 이하의 EU 거주자에게 영향을 미치는 조건에 대해 부여되며, 10년의 시장 독점권, 규제 수수료 감소 및 중앙 집중화된 EU 승인 절차를 포함한 혜택을 제공합니다. 회사는 2025년 상반기에 2상 데이터의 주요 결과를 발표할 것으로 예상하고 있습니다.
Actuate Therapeutics (NASDAQ: ACTU) a reçu de l'Agence européenne des médicaments (EMA) la désignation de médicament orphelin pour elraglusib dans le traitement de l'adénocarcinome canalaire pancréatique (PDAC). Cette désignation fait suite à une désignation similaire de médicament orphelin par la FDA et à des résultats intermédiaires positifs récents d'un essai de Phase 2.
L'analyse intermédiaire de l'essai de Phase 2 a montré qu'elraglusib, en combinaison avec gemcitabine/nab-paclitaxel (GnP), a démontré des améliorations statistiquement significatives tant du taux de survie à 1 an (p=0.002) que de la survie globale médiane (rapport de risque 0.63, p=0.016) par rapport à GnP seul chez les patients atteints de PDAC métastatique.
La désignation de l'EMA, accordée pour des conditions touchant moins de 2 personnes sur 10 000 résidents de l'UE, offre des avantages tels qu'une exclusivité de marché potentielle de 10 ans, des frais réglementaires réduits et un processus d'approbation centralisé au sein de l'UE. L'entreprise s'attend à communiquer les données principales de la Phase 2 au cours de la première moitié de 2025.
Actuate Therapeutics (NASDAQ: ACTU) hat von der Europäischen Arzneimittelagentur (EMA) die Bezeichnung als Orphan Medicinal Product für elraglusib zur Behandlung von pankreatischem duktalem Adenokarzinom (PDAC) erhalten. Diese Bezeichnung folgt einer ähnlichen Orphan Drug Designation durch die FDA sowie den kürzlichen positiven interimistischen Ergebnissen der Phase-2-Studie.
Die interimistische Analyse der Phase-2-Studie zeigte, dass elraglusib in Kombination mit Gemcitabin/nab-Paclitaxel (GnP) statistisch signifikante Verbesserungen sowohl bei der Überlebensrate nach 1 Jahr (p=0.002) als auch beim medianen Gesamtüberleben (Hazard Ratio 0.63, p=0.016) im Vergleich zu GnP allein bei metastasierten PDAC-Patienten aufwies.
Die EMA-Bezeichnung, die für Zustände vergeben wird, die weniger als 2 von 10.000 EU-Bewohnern betreffen, bietet Vorteile wie eine potenzielle Marktexklusivität von 10 Jahren, reduzierte Zulassungsgebühren und einen zentralisierten Genehmigungsprozess in der EU. Das Unternehmen rechnet damit, dass in der ersten Hälfte von 2025 die Hauptdaten der Phase-2-Studie veröffentlicht werden.
- Received EMA Orphan Medicinal Product Designation for elraglusib
- Phase 2 trial showed statistically significant improvement in 1-year survival rate (p=0.002)
- Demonstrated significant increase in median overall survival (hazard ratio 0.63, p=0.016)
- Potential for 10-year market exclusivity in EU upon approval
- None.
Insights
The EMA's OMPD designation for elraglusib marks a important milestone in addressing pancreatic cancer treatment. The interim Phase 2 data is particularly compelling, showing a
The GSK-3β inhibition mechanism represents an innovative approach in pancreatic cancer treatment. When combined with the standard GnP regimen, elraglusib's performance suggests a potential paradigm shift in first-line mPDAC therapy. The dual regulatory recognition (FDA and EMA orphan designations) strengthens the drug's market position and development trajectory.
For context: pancreatic cancer has a 5-year survival rate below
This regulatory milestone substantially de-risks Actuate's lead asset and enhances its commercial prospects. The OMPD designation provides significant strategic advantages: reduced regulatory fees, streamlined EU approval process and critically, 10 years of market exclusivity. For a small-cap biotech (
The positive interim Phase 2 data, coupled with dual orphan designations, increases the probability of successful commercialization and potential partnership opportunities. The pancreatic cancer therapeutics market, projected to reach
CHICAGO and FORT WORTH, Texas, Jan. 07, 2025 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), 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-3β), today announced that the European Medicines Agency (EMA) has granted Orphan Medicinal Product Designation (OMPD) to elraglusib, a novel GSK-3β inhibitor, for the treatment of pancreatic ductal adenocarcinoma (PDAC).
“With limited treatment options available for patients with PDAC, the EMA Orphan Drug Designation and FDA Orphan Drug Designation granted to elraglusib for the same indication reflect elraglusib’s potential to address a critical need for people living with this aggressive disease with a notably poor prognosis. This regulatory milestone, along with the recently announced positive interim results from our Phase 2 trial of elraglusib in patients with mPDAC that showed a significant clinical benefit and anti-tumor activity, should expedite our efforts to advance elraglusib as a novel, potential treatment for mPDAC,” said Daniel Schmitt, President & Chief Executive Officer of Actuate. “We look forward to reporting topline data from our Phase 2 trial in 1H 2025 and working closely with the EMA and the FDA to accelerate elraglusib’s clinical development program in metastatic pancreatic cancer.”
Elraglusib is a novel GSK-3β inhibitor currently in a randomized Phase 2 trial (Actuate-1801 Part 3B study, NCT03678883) comparing the combination of elraglusib with gemcitabine/nab-paclitaxel (GnP) to GnP alone as a first line therapy in patients with metastatic pancreatic cancer (mPDAC). A recently reported analysis of interim Phase 2 data demonstrated that treatment with the elraglusib combination led to statistically significant increases in 1-year survival rate (p value of 0.002) and median overall survival (hazard ratio of 0.63, p value of 0.016) versus treatment with GnP alone.
Orphan Designation is granted to therapies intended for the treatment, prevention, or diagnosis of life-threatening or chronically debilitating diseases that affect no more than two in 10,000 people in the European Union (EU) and for which no satisfactory therapy is available. The treatment must also provide significant benefit to those affected by the condition. EMA orphan drug designation provides certain benefits, including the potential for 10 years of market exclusivity following regulatory approval in the EU, reduction in regulatory fees and a centralized EU approval process1.
1. European Medicines Agency, "Orphan Designation." https://www.ema.europa.eu/en/human-regulatory-overview/orphan-designation-overview
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’s lead investigational drug product, elraglusib (a novel GSK-3β inhibitor), targets molecular pathways in cancer that are involved in promoting tumor growth and resistance to conventional cancer drugs such as chemotherapy including several DNA Damage Response (DDR) pathways. Elraglusib is designed to act as a mediator of anti-tumor immunity through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and regulates multiple immune checkpoints and immune cell function. For additional information, please visit the Company’s website at http://www.actuatetherapeutics.com.
Forward-Looking Statements
This press release contains forward-looking statements about us, including our clinical trials and development plans, and our industry. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would,” 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 clinical and preclinical drug development involves a lengthy and expensive process with uncertain timelines and outcomes, results of prior preclinical studies and early clinical trials are not necessarily predictive of future results, 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; preliminary and unpublished data may be subject to change 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; 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; and our ability to realize any benefits or value associated with the Orphan Medicinal Product Designation (OMPD) granted to elraglusib by the EMA for the treatment of pancreatic ductal adenocarcinoma (PDAC). In addition, any forward-looking statements are qualified in their entirety by reference to the factors discussed under the heading “Item 1A. Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 filed with the SEC on November 13, 2024 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
mmoyer@lifesciadvisors.com
FAQ
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