WP1122 / Moleculin, CNS Pharma, WPD Pharma - LARVOL DELTA

Moleculin Announces Positive Interim Data in Annamycin Clinical Trials: MB-106 AML Trial Complete Response (CR) Rate of 38% and MB-107 STS Lung Mets Trial Median Phase 1B Extended Overall Survival of 11 Months (PRNewswire) - "WP1066: Moleculin is in ongoing discussions with multiple academic institutions in separate programs evaluating WP1066 for the treatment of glioblastomas and/or pediatric brain tumors. The Company expects to finalize agreements with Northwestern University and FDA filings in the early 2024 (Clinicaltrials.gov ID: NCT05879250)...The Company supplied drug product to an externally funded pediatric brain tumor trial with WP1066 up to its conclusion in February 2023 and expects additional externally funded clinically trials for WP1066 (in combination with radiation) in 2023 in the U.S. and, possibly, in Southeast Asia...Seek external funding opportunities for an investigator-initiated clinical trial in adult and pediatric cancer patients in 2023; Announce progress regarding an IV formulation by the end of 2023 or in early 2024....Identify investigators interested in initiating a clinical trial to study the safety, pharmacokinetics and efficacy of oral WP1122 in adult patients with GBM."

FDA event • Licensing / partnership • New trial • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

d-Glucose- and d-mannose-based antimetabolites. Part 4: Facile synthesis of mono- and di-acetates of 2-deoxy-d-glucose prodrugs as potentially useful antimetabolites. (PubMed, Carbohydr Res) - "2-Deoxy-d-glucose (2-DG), a compound known to interfere with d-glucose and d-mannose metabolism, has been tested as a potential anticancer and antiviral agent. We describe novel facile and scalable syntheses of seven selectively acetylated water-soluble derivatives of 2-DG and present a detailed H and C NMR analysis of all final products. X-ray diffraction analysis has been performed for compound WP1122 that was selected for detailed preclinical and subsequent clinical evaluation as potential anticancer or antiviral agent."

Journal • Oncology

Study to Assess the Safety and Pharmacokinetics of WP1122 in Healthy Volunteers (clinicaltrials.gov) - P1a | N=53 | Completed | Sponsor: Moleculin Biotech, Inc. | Active, not recruiting ➔ Completed | N=80 ➔ 53

Enrollment change • Trial completion

Moleculin Granted FDA Fast Track Designation of WP1122 for the Treatment of Glioblastoma Multiforme (PRNewswire) - "Moleculin Biotech...announced that the U.S. Food and Drug Administration ('FDA') has granted Fast Track designation of WP1122 for the treatment of Glioblastoma Multiforme ('GBM'). The FDA's Fast Track designation is intended to potentially facilitate the development and expedite the review of novel therapies to treat serious conditions for which there is unmet medical need. With the Fast Track designation, Moleculin is potentially eligible for more frequent regulatory meetings and communications with the FDA."

Fast track designation • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

Study to Assess the Safety and Pharmacokinetics of WP1122 in Healthy Volunteers (clinicaltrials.gov) - P1a | N=80 | Active, not recruiting | Sponsor: Moleculin Biotech, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed

Moleculin Receives FDA Orphan Drug Designation of WP1122 for the Treatment of Glioblastoma Multiforme (PRNewswire) - "Moleculin Biotech...announced that the U.S. Food and Drug Administration ('FDA') has granted Orphan Drug Designation of WP1122 for the treatment of Glioblastoma Multiforme ('GBM')....'Given the progress of our Phase 1 clinical trial in healthy volunteers, the strong preclinical data supporting GBM as one of many potential indications and the recent clearance by the FDA of IND status for WP1122 in GBM, we believe this designation further supports the potential of WP1122 and is another step forward in further validating our deep pipeline'."

Orphan drug • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

Study to Assess the Safety and Pharmacokinetics of WP1122 in Healthy Volunteers (clinicaltrials.gov) - P1a | N=80 | Recruiting | Sponsor: Moleculin Biotech, Inc. | Trial completion date: Jul 2022 ➔ Oct 2022 | Trial primary completion date: Jul 2022 ➔ Oct 2022 | Not yet recruiting ➔ Recruiting

Enrollment open • Trial completion date • Trial primary completion date

WP1122: Clinical Trial of Efficacy and Safety of Oral Drug in Adult Patients With COVID-19 (clinicaltrials.gov) - P1/2 | N=75 | Not yet recruiting | Sponsor: Andrei Carvalho Sposito

New P1/2 trial • Acute Respiratory Distress Syndrome • Infectious Disease • Novel Coronavirus Disease • Pulmonary Disease • Respiratory Diseases • IL6

Study to Assess the Safety and Pharmacokinetics of WP1122 in Healthy Volunteers (clinicaltrials.gov) - P1a | N=80 | Not yet recruiting | Sponsor: Moleculin Biotech, Inc. | Initiation date: Jan 2022 ➔ Apr 2022

Trial initiation date

Study to Assess the Safety and Pharmacokinetics of WP1122 in Healthy Volunteers (clinicaltrials.gov) - P1a; N=80; Not yet recruiting; Sponsor: Moleculin Biotech, Inc.

Clinical • New P1 trial

Synergistic Anticancer Effect of Glycolysis and Histone Deacetylases Inhibitors in a Glioblastoma Model. (PubMed, Biomedicines) - "Over the last decade, we have seen tremendous progress in research on 2-deoxy-D-glucose (2-DG) and its analogs...Novel 2-DG analogs such as WP1122 and others have revived the old concept of glycolysis inhibition as an effective anticancer strategy...We focused our efforts on the development of new combinations of anticancer agents coupled with 2-DG and its derivatives, targeting glioblastoma, which is in desperate need of novel approaches and therapeutic options and is particularly suited to glycolysis inhibition, due to its reliance on aerobic glycolysis. Herein, we present evidence that a combined treatment of 2-DG analogs and modulation of histone deacetylases (HDAC) activity via HDAC inhibitors (sodium butyrate and sodium valproate) exerts synergistic cytotoxic effects in glioblastoma U-87 and U-251 cells and represents a promising therapeutic strategy."

Epigenetic controller • Journal • Brain Cancer • Glioblastoma • Oncology • Solid Tumor

Moleculin Receives FDA Allowance to Begin a Phase 1 Study of WP1122 for the Treatment of Glioblastoma Multiforme (PRNewswire) - "Moleculin Biotech, Inc...announced that the US Food and Drug Administration (FDA) is allowing the Company's Investigational New Drug (IND) application to study WP1122 for the treatment of Glioblastoma Multiforme (GBM) to go forward. With this IND now cleared, Moleculin plans to initiate a Phase 1 open label, single arm, dose escalation study of the safety, pharmacokinetics and efficacy of oral WP1122 in adult patients with GBM....Consistent with its strategy of leveraging external funding for many of its clinical trials, Moleculin intends to seek opportunities for an investigator-initiated clinical trial of WP1122 in cancer patients in 2022."

IND • New P1 trial • Glioblastoma • Oncology

Moleculin Reports First Quarter 2021 Financial Results and Provides Programs Update (PRNewswire) - “First-in-class p-STAT3 inhibitors - WP1066 and WP1220 - Upcoming Milestones - 2H 2021: Facilitate Phase 1/2 study of WP1066 + radiation for the treatment of GBM. 1H 2022: Facilitate launch of Phase 2 study of WP1066 for the treatment of pediatric brain tumors, including DIPG. Actively seeking collaboration with a strategic partner in the near term for external funding for the continued development of WP1220 in a Phase 2 clinical trial as a topical therapy for CTCL…2H 2021: File an IND in the U.S. for the treatment of certain cancers such as GBM and pancreatic cancer, with WP1122.”

IND • Licensing / partnership • New P2 trial • Trial status • Cutaneous T-cell Lymphoma • Gastrointestinal Cancer • Glioblastoma • Hematological Malignancies • Non-Hodgkin’s Lymphoma • Oncology • Pancreatic Cancer

Moleculin Engages IQVIA to Manage Potential COVID-19 Clinical Trial (PRNewswire) - "Moleculin Biotech, Inc....announced the engagement of IQVIA Biotech, a contract research organization (CRO) to manage the Company's effort to begin potential clinical trials of WP1122 for the treatment of COVID-19....'We believe all of the necessary preclinical safety testing has now been completed to qualify for the equivalent of an IND outside the US. Over the last quarter, we completed our pre-clinical data'...'In addition, considering that the active ingredient in WP1122 is 2-deoxy-D-glucose (2-DG) and that 2-DG has now shown efficacy in a Phase 2 clinical trial conducted by an unrelated drug developer outside of the US, we believe that a sufficient efficacy rationale for WP1122 already exists to begin clinical trials.'"

Licensing / partnership • Infectious Disease • Novel Coronavirus Disease

WPD Pharmaceuticals Announces Amended Sublicense Agreement with Moleculin Biotech for WP1066, WP1122 and Annamycin Drug Candidates (Yahoo Finance) - “WPD Pharmaceuticals…announce that is has entered into an amended and restated sublicense agreement with Moleculin Biotech…In consideration of the sublicense, WPD agreed that it must use commercially reasonable efforts to develop and commercialize Products in the Territories. The term 'commercially reasonable efforts' has been amended to mean expenditure by WPD of at least USD$2,500,000 during the first 4 years of the agreement on the research, development and commercialization of Products and at least USD$1,000,000 in each of the 4 years thereafter. WPD also will pay to Moleculin a royalty on Products sold.”

Licensing / partnership • Oncology

Moleculin Biotech, Inc. Reports Financial Results for the Year Ended December 31, 2020 (PRNewswire) - “WP1122, WP1096 and WP1097 Portfolio…Working to initiate a Phase 1a/1b clinical trial in COVID-19 or a physician-sponsored clinical trial for a cancer indication, or both in 2021. Anticipated 2021 Milestones…Potential for 8 clinical trials in 2021, including 3 to be conducted by Moleculin and 5 primarily externally funded and conducted trials; External funding will be relied upon to the extent it is available.”

Clinical • New P1 trial • Brain Cancer • Glioblastoma • Oncology

Moleculin Announces Agreement to Produce WP1122 for Expanded Development of Potential COVID-19 and Oncology Drug Candidate (PRNewswire) - "Moleculin Biotech, Inc....today announced that it has entered into an agreement with Sterling Pharma USA LLC for US production of WP1122 to support its expanded development efforts in preparation for submitting a request to the US Food and Drug Administration ('FDA') for Investigational New Drug ('IND') status for WP1122 for the potential treatment of COVID-19."

IND • Licensing / partnership • Infectious Disease • Novel Coronavirus Disease

CNS provides update on development agreement for WP1122 (PRNewswire) - "CNS Pharmaceuticals...provided an update on the development of WP1122. As previously announced, CNS entered into an agreement with WPD Pharmaceuticals for the development of several preclinical drug candidates for antiviral indications, including WP1122, in certain territories. WPD Pharmaceuticals previously licensed rights to WP1122, from Moleculin Biotech, Inc. (Moleculin) for certain territories....Moleculin further stated that it may be able to submit its IND application in the second half of this year."

IND • Licensing / partnership • Oncology

Moleculin provides update on coronavirus drug development (PRNewswire) - "Moleculin Biotech...announced that the US Food and Drug Administration ('FDA') has granted the Company's request for a Pre-IND Meeting to provide guidance regarding the Company's plan to study its drug candidate, WP1122, in a clinical trial for patients with COVID-19 (the disease caused by the SARS-CoV-2 coronavirus). In addition, Moleculin provided a general update on its WP1122 development efforts....Given the toxicology testing plan discussed below, we estimate that we should be able to submit our IND application in the second half of this year. More specific timing will depend upon the guidance we receive from our Pre-IND Meeting with the FDA."

FDA event • IND • Infectious Disease • Novel Coronavirus Disease

"$MBRX Announces Active Compound in WP1122 Reduces #Coronavirus Replication In Vitro by 100%" (@BioStocks)

Preclinical

"Moculin Biotech WP1122" (@WhereTruthRulz)

Moleculin announces patent filing to cover new coronavirus drug candidate (PRNewswire) - “Moleculin Biotech, Inc...announced that a new patent application has been filed covering the use of WP1122 and its analogs as therapies to limit the ability of coronavirus and other viruses to replicate. The patent application covers joint discoveries which came as a result of an ongoing sponsored research agreement."

Patent

Moleculin signs agreement with UTMB to test WP1122 on a range of viruses, including coronavirus (PRNewswire) - “Moleculin Biotech, Inc…announced that it has entered into an agreement with the University of Texas Medical Branch at Galveston (UTMB) to conduct research on Moleculin's patented portfolio of molecular inhibitors, including drug candidate, WP1122, for antiviral properties against a range of viruses, including Coronavirus….Under the agreement, Moleculin will supply the lead drug candidate, WP1122, and related inhibitors, as well as technical support and UTMB will begin testing these candidates against various viral disease models, including COVID-19, in connection with the UTMB Center for Biodefense and Emerging Infectious Diseases.”

Licensing / partnership

Hyperpolarized Pyruvate MR Spectroscopy Depicts Glycolytic Inhibition in a Mouse Model of Glioma. (PubMed, Radiology) - "Large contrast agent-enhanced tumors were shown in mice with glioma at T2-weighted and T1-weighted postcontrast MRI by postimplantation day 40. After treatment with WP1122, a decrease in lactate was observed in mice with glioma (baseline and treatment mean k, 0.027 and 0.018 sec, respectively, P = .01; baseline and posttreatment mean nLac, 0.28 and 0.22, respectively, P = .01) whereas no significant decrease was observed in healthy control mice (baseline and posttreatment mean k, 0.011 and 0.017 sec, respectively, P = .91; baseline and posttreatment mean nLac, 0.16 and 0.21, respectively, P = .84).ConclusionHyperpolarized carbon 13 measurements of pyruvate metabolism can provide rapid feedback for monitoring treatment response in glioma"

Journal • Preclinical

Newly added product - Preclinical, Oncology, Pancreatic Cancer

Pipeline update