Moleculin Announces First Patients Treated in Phase 2 Clinical Trial of STAT3 Inhibitor in Combination with Radiation for the Treatment of Glioblastoma
Moleculin Biotech (Nasdaq: MBRX) has announced the start of an investigator-initiated Phase 2 clinical trial evaluating WP1066 in combination with radiation therapy for the treatment of glioblastoma. The study, conducted at Northwestern University, is funded by the NIH and BrainUp®. WP1066 is Moleculin's Immune/Transcription Modulator designed to stimulate immune response to tumors by inhibiting regulatory T cells and oncogenic transcription factors.
The trial aims to address the unmet need in glioblastoma treatment, with four subjects already active and expectations to fill the safety lead-in group of six subjects soon. Glioblastoma is an aggressive brain tumor with a median survival of only 15 months. Moleculin has received Orphan Drug Designation for WP1066 for brain tumors and Rare Pediatric Disease designation for three other pediatric indications.
Moleculin Biotech (Nasdaq: MBRX) ha annunciato l'inizio di un trial clinico di Fase 2 iniziato da un investigatore per valutare WP1066 in combinazione con la radioterapia per il trattamento del glioblastoma. Lo studio, condotto presso la Northwestern University, è finanziato dal NIH e da BrainUp®. WP1066 è il Modulator Immunitario/di Trascrizione di Moleculin progettato per stimolare la risposta immunitaria ai tumori inibendo le cellule T regolatorie e i fattori di trascrizione oncogeni.
Il trial mira a soddisfare il fabbisogno insoddisfatto nel trattamento del glioblastoma, con già quattro soggetti attivi e la previsione di completare presto il gruppo di sicurezza di sei soggetti. Il glioblastoma è un tumore del cervello aggressivo con una sopravvivenza media di soli 15 mesi. Moleculin ha ricevuto la Designazione di Farmaco Orfano per WP1066 per i tumori cerebrali e la designazione di Malattia Pediatrica Rara per altre tre indicazioni pediatriche.
Moleculin Biotech (Nasdaq: MBRX) ha anunciado el inicio de un ensayo clínico de Fase 2 iniciado por un investigador que evalúa WP1066 en combinación con terapia de radiación para el tratamiento del glioblastoma. El estudio, que se lleva a cabo en la Universidad Northwestern, está financiado por el NIH y BrainUp®. WP1066 es el Modulador Inmunológico/Transcripcional de Moleculin diseñado para estimular la respuesta inmune a los tumores inhibiendo las células T reguladoras y los factores de transcripción oncogénicos.
El ensayo busca abordar la necesidad no satisfecha en el tratamiento del glioblastoma, con cuatro sujetos ya activos y expectativas de completar pronto el grupo de seguridad de seis sujetos. El glioblastoma es un tumor cerebral agresivo con una supervivencia media de solo 15 meses. Moleculin ha recibido la Designación de Medicamento Huérfano para WP1066 para tumores cerebrales y la designación de Enfermedad Pediátrica Rara para tres otras indicaciones pediátricas.
Moleculin Biotech (Nasdaq: MBRX)은 임상시험자 주도 제2상 클리니컬 트라이얼의 시작을 발표하며, WP1066를 방사선 치료와 병행하여 교모세포종 치료에 대해 평가합니다. 이 연구는 노스웨스턴 대학교에서 진행되고 있으며, NIH와 BrainUp®의 자금을 지원받고 있습니다. WP1066는 종양에 대한 면역 반응을 자극하기 위해 조절 T 세포와 종양 유전자 전사 인자를 억제하는 Moleculin의 면역 및 전사 조절제입니다.
이 시험은 교모세포종 치료에 있어 충족되지 않은 필요를 해결하는 것을 목표로 하며, 현재 네 명의 대상이 이미 활동 중이고, 곧 여섯 명의 대상이 포함된 안전성 선행 그룹을 채울 것으로 예상하고 있습니다. 교모세포종은 평균 생존 기간이 겨우 15개월인 공격적인 뇌 종양입니다. Moleculin은 WP1066에 대해 뇌 종양에 대한 고아 의약품 지정을, 다른 세 가지 소아 적응증에 대해 희귀 소아 질환 지정을 받았습니다.
Moleculin Biotech (Nasdaq: MBRX) a annoncé le lancement d'un essai clinique de phase 2 initié par un enquêteur évaluant WP1066 en association avec la radiothérapie pour le traitement du glioblastome. L'étude, réalisée à l'Université Northwestern, est financée par le NIH et BrainUp®. WP1066 est le modulateur immunitaire/de transcription de Moleculin conçu pour stimuler la réponse immunitaire aux tumeurs en inhibant les cellules T régulatrices et les facteurs de transcription oncogéniques.
L'essai vise à répondre au besoin non satisfait dans le traitement du glioblastome, avec déjà quatre sujets actifs et des attentes de remplir prochainement le groupe d'initiation de sécurité de six sujets. Le glioblastome est une tumeur cérébrale agressive avec une survie médiane de seulement 15 mois. Moleculin a reçu la désignation de médicament orphan pour WP1066 pour les tumeurs cérébrales et la désignation de maladie pédiatrique rare pour trois autres indications pédiatriques.
Moleculin Biotech (Nasdaq: MBRX) hat den Beginn einer vom Prüfer initiierten Phase-2-Studie bekannt gegeben, die WP1066 in Kombination mit Strahlentherapie zur Behandlung von Gliosarkomen bewertet. Die Studie, die an der Northwestern University durchgeführt wird, wird vom NIH und von BrainUp® finanziert. WP1066 ist Moleculins Immun-Transkriptionsmodulator, der entwickelt wurde, um die Immunantwort auf Tumoren zu stimulieren, indem regulatorische T-Zellen und onkogene Transkriptionsfaktoren gehemmt werden.
Die Studie zielt darauf ab, den ungedeckten Bedarf in der Glioblastom-Behandlung anzugehen, wobei bereits vier Probanden aktiv sind und schnell die Sicherheitsgruppe von sechs Probanden gefüllt werden soll. Glioblastom ist ein aggressiver Hirntumor mit einer durchschnittlichen Überlebenszeit von nur 15 Monaten. Moleculin hat für WP1066 eine Orphan Drug Designation für Gehirntumoren sowie eine Rare Pediatric Disease Designation für drei weitere pädiatrische Indikationen erhalten.
- Initiation of Phase 2 clinical trial for glioblastoma treatment
- Rapid patient recruitment with four subjects already active
- Funding support from NIH and BrainUp®
- Orphan Drug Designation received for WP1066 in brain tumors
- Rare Pediatric Disease designation for three other pediatric indications
- None.
Insights
The initiation of this Phase 2 trial for WP1066 in combination with radiation therapy for glioblastoma is a significant development. The study's focus on IDH wild-type, MGMT-unmethylated glioblastoma patients targets a particularly challenging subgroup with treatment options. The rapid recruitment of four subjects within months suggests high interest and unmet need in this area.
The trial's primary outcome measure of progression-free survival is a clinically relevant endpoint. The secondary measure of tumor microenvironment analysis could provide valuable insights into WP1066's mechanism of action. The combination approach of STAT3 inhibition with radiation therapy is innovative, potentially enhancing both direct tumor killing and immune system activation.
However, it's important to note that this is an early-stage trial and success is not guaranteed. The safety lead-in group will be important in determining the tolerability of this combination approach before broader enrollment.
This Phase 2 trial initiation is a positive development for Moleculin Biotech (NASDAQ: MBRX). The collaboration with Northwestern University and funding from the NIH and BrainUp® significantly reduces the company's financial burden for this study. This external support validates the potential of WP1066 and could attract further investor interest.
The Orphan Drug Designation for brain tumors and Rare Pediatric Disease designations for three other indications provide potential regulatory benefits and market exclusivity if WP1066 reaches approval. The glioblastoma market, while relatively small with an incidence rate of
Investors should monitor the safety lead-in results closely, as positive outcomes could significantly impact Moleculin's stock price. However, as a Phase 2 trial, substantial revenue impact is still years away and the company will likely need additional funding for future development stages.
– Investigator-initiated Phase 2 study is being conducted at Northwestern University in cooperation with Moleculin
– Funding for study provided by NIH and BrainUp®
– Trial combines WP1066 and radiation, which demonstrated both significant therapeutic response and immune memory in glioblastoma animal models
The NU 21C06 trial is a Phase 2, open-label, multi-arm trial of radiation therapy in combination with WP1066 in newly diagnosed IDH (isocitrate dehydrogenase) wild-type, MGMT-unmethylated glioblastoma patients. The primary outcome measure for the study is progression-free survival and secondary outcome measures include tumor microenvironment analysis.
Dr. Priya Kumthekar, Associate Professor at Northwestern University and the Co-Investigator for the study commented, "The start of patient dosing is a significant step towards addressing the unmet need in the treatment of glioblastoma. We were very encouraged by the preclinical data that demonstrated by WP1066 in combination with radiation therapy increased survival and induced anti-tumor immune responses and are excited to finally be treating patients with this approach."
Dr. Amy Heimberger, Co-Investigator and Vice Chair for Research, Department of Neurological Surgery for Northwestern University added, "The significance of the unmet need can be seen in the pace of recruitment for this trial. Within the first few months of opening the trial, we have four subjects active in the study and expect to fill the safety lead-in group of six subjects soon. If treatment is well tolerated in these subjects, we will continue enrollment for the balance of the trial."
WP1066 is Moleculin's flagship Immune/Transcription Modulator designed to stimulate the immune response to tumors by inhibiting the errant activity of regulatory T cells while also inhibiting key oncogenic transcription factors, including p-STAT3 (phosphorylated signal transducer and activator of transcription 3), c-Myc (a cellular signal transducer named after a homologous avian virus called Myelocytomatosis) and HIF-1α (hypoxia-inducible factor 1α). These transcription factors are widely sought targets because of their role in cancer cell survival and proliferation, angiogenesis (coopting vasculature for blood supply), invasion, metastasis, and inflammation associated with tumors.
"The NU 21C06 study is a critical next step in the development of STAT3-targeting technologies. STAT3 has long been a sought-after but elusive cell-signaling target in cancer therapy but the work we are doing in collaboration with Northwestern University supported by the NIH and BrainUp® could change all that. We believe that based on the data demonstrated to date, WP1066 has the potential for significant anti-tumor activity in a wide range of hard-to-treat cancers," added Mr. Walter Klemp, Chairman and Chief Executive Officer of Moleculin.
Glioblastoma is a common type of tumor originating in the brain. The average annual age-adjusted incidence rate of glioblastoma is 3.19 per 100,000 persons in
Moleculin has received Orphan Drug Designation for WP1066 for the treatment of brain tumors, as well as Rare Pediatric Disease designation for three other pediatric indications. For more information about the NU 21C06 Phase 2 study, visit clinicaltrials.gov and reference identifier NCT05879250.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company advancing a pipeline of therapeutic candidates addressing hard-to-treat tumors and viruses. The Company's lead program, Annamycin, is a next-generation anthracycline designed to avoid multidrug resistance mechanisms and to eliminate the cardiotoxicity common with currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases.
The Company is initiating the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial (MB-108), a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC, for the treatment of relapsed or refractory acute myeloid leukemia. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, the Company believes it has substantially de-risked the development pathway towards a potential approval for Annamycin for the treatment of AML. This study is subject to appropriate future filings with potential additional feedback from the FDA and their foreign equivalents.
Additionally, the Company is developing WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic and other cancers. Moleculin is also engaged in the development of a portfolio of antimetabolites, including WP1122 for the potential treatment of viruses, as well as certain cancer indications.
For more information about the Company, please visit www.moleculin.com and connect on X, LinkedIn and Facebook.
Forward-Looking Statements
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin has attempted to identify forward-looking statements by terminology including 'believes,' 'estimates,' 'anticipates,' 'expects,' 'plans,' 'projects,' 'intends,' 'potential,' 'may,' 'could,' 'might,' 'will,' 'should,' 'approximately' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These forward-looking statements, include, but are not limited to, the ability for the study to fill the safety lead-in group of six subjects on a timely basis and whether the preclinical results will be demonstrated in the Phase 2 trial. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. "Risk Factors" in our most recently filed Form 10-K filed with the Securities and Exchange Commission (SEC) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.
Investor Contact:
JTC Team, LLC
Jenene Thomas
(833) 475-8247
MBRX@jtcir.com
1 Thakkar J, Dolecek TA, Horbinski C, Ostrom QT, Lightner DD, Barnholtz-Sloan JS, et al. Epidemiologic and molecular prognostic review of glioblastoma. Cancer Epidemiol. Biomarkers Rev. 2014;23(10):1985-96.
2 Koshy M, Villano JL, Dolecek TA, Howard A, Mahmood U, Chmura SJ, et al. Improved survival time trends of glioblastoma using the SEER 17 population-based registries. J Neuro Oncol. 2012;107(1):207-12
3 Ostrom QT, Gittleman H, Farah P, Ondracek A, Chen Y, Wolinsky Y, et al. CBTRUS statistical report: Primary brain and central nervous system tumors diagnosed in
4 De Vleeschouwer S, editor.
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SOURCE Moleculin Biotech, Inc.
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