Moleculin Receives IND Clearance to Conduct Phase 1 Study of WP1066 for the Treatment of Recurrent Malignant Glioma
21 Apr 2022
GliomaWP-1066Orphan DrugCollaborate
Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, today announced that the US Food and Drug Administration (FDA) is allowing the Company's Investigational New Drug (IND) application to study WP1066 for the treatment of recurrent malignant glioma. With this IND now cleared, Moleculin plans to evaluate strategic partnerships and collaborations to conduct a Phase 1 open label, single arm, dose escalation study of the safety, pharmacokinetics and efficacy of oral WP1066 in adult patients with recurrent malignant glioma.
WP1066 is the Company's flagship Immune/Transcription Modulator designed to stimulate the immune response to tumors by inhibiting the errant activity of Regulatory T-Cells (TRegs) 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 that are believed to contribute to an increase in cell survival and proliferation, and the angiogenesis (coopting vasculature for blood supply), invasion, metastasis and inflammation associated with tumors. They may also play a role in the inability of immune checkpoint inhibitors to affect more resistant tumors.
"WP1066 has demonstrated significant anti-tumor activity in a wide range of tumor cell lines and increased survival in a number of animal models to-date. Additionally, the preliminary results demonstrated in the ongoing trial of WP1066 for pediatric brain tumors bolster our confidence and this IND clearance provides further momentum for its continued research and development," commented Walter Klemp, Chairman and CEO of Moleculin. "We expect the clearance of this IND to further support the ongoing pediatric studies being conducted by the team at Emory University, and we are evaluating the potential for additional externally funded investigator-initiated studies."
WP1066 is currently being evaluated in collaboration with Emory University for the treatment of pediatric brain tumors, including Diffuse Interstitial Pontine Glioma (DIPG). The Emory trial for pediatric brain tumors has now treated three subjects in the first two cohorts of the Phase 1 dose escalation portion of physician-sponsored clinical trial. In that trial, one of the subjects in the first cohort with DIPG showed an apparent response to the treatment with both clinical improvement and radiologic reduction of tumor size. The Company cautions that this is preliminary data, and no conclusions should be drawn from this single event. One subject has been treated in the third cohort at the dose level of 8mg/kg. Two more subjects will be treated at this dose level. Emory University has amended its protocol to allow dosing at 16 mg/kg after these two additional subjects have been dosed, and the third cohort dosing has been deemed safe.
The Company 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. Additionally, WP1066 + radiation is being evaluated, pre-clinically, in the treatment of Glioblastoma Multiforme (GBM).
Glioma is a common type of tumor originating in the brain. About 33% of all brain tumors are gliomas, which originate in the glial cells that surround and support neurons in the brain, including astrocytes, oligodendrocytes and ependymal cells. GBM is the most aggressive malignant primary brain tumor and remains as an incurable tumor with a median survival of only 15 months. It is the most common malignant primary brain tumor making up 54% of all gliomas and 16% of all primary brain tumors, and despite advancements, survival rates for patients with GBM have shown no notable improvement in population statistics in the last three decades. The average annual age-adjusted incidence rate of GBM is 3.19 per 100,000 persons in the United States.
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