On September 16, 2019 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, reported its sponsored research at MD Anderson Cancer Center has resulted in the filing of a new patent on behalf of MD Anderson Cancer Center covering the combination of its immune-stimulating/transcriptional-modulator, WP1066, with well-known immune checkpoint inhibitors (Press release, Moleculin, SEP 16, 2019, View Source [SID1234539550]).

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Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

"We had previously announced preliminary preclinical data showing beneficial therapeutic effect from WP1066 when used in combination with PDL-1 and CTLA-4 immune checkpoint inhibitors in pancreatic cancer models," commented Walter Klemp, Moleculin’s Chairman and CEO. "We are pleased to report the filing of a patent on behalf of MD Anderson Cancer Center for this new discovery that opens new potentially effective approaches to utilize check point inhibitors for treatment of pancreatic cancer and other types cancers that are unresponsive to current immunotherapies. These studies were supported by our sponsored research agreement with MD Anderson Cancer Center. Pursuant to the terms of our Sponsored Research Agreement, we intend to add this to our already diverse list of technology licenses."

On September 16, 2019 Seattle Genetics, Inc. (Nasdaq:SGEN) and Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for the investigational agent enfortumab vedotin and granted Priority Review for the treatment of patients with locally advanced or metastatic urothelial cancer who have received a PD-1/L1 inhibitor and who have received a platinum-containing chemotherapy in the neoadjuvant/adjuvant, locally advanced or metastatic setting (Press release, Astellas, SEP 16, 2019, View Source [SID1234539549]). The filing is based on results from the first cohort of patients in the EV-201 pivotal phase 2 clinical trial that were presented as a late-breaking oral presentation at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in June 2019. Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of March 15, 2020. Enfortumab vedotin is a novel investigational antibody-drug conjugate (ADC) that targets Nectin-4, a protein that is highly expressed in urothelial cancers.1

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The FDA granted enfortumab vedotin Breakthrough Therapy designation in March 2018, for patients with locally advanced or metastatic urothelial cancer whose disease has progressed during or following checkpoint inhibitor therapy.

"The FDA’s filing of the application for enfortumab vedotin and granting of Priority Review is a significant milestone toward offering a new treatment to patients with advanced urothelial cancer who have a clear unmet need," said Roger Dansey, M.D., Chief Medical Officer at Seattle Genetics.

"If approved, enfortumab vedotin will likely play an important role in the treatment of advanced urothelial cancer, and we look forward to working with the FDA as the review process advances," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head at Astellas.

About the EV-201 Trial

EV-201 is an ongoing single-arm, pivotal phase 2 clinical trial of enfortumab vedotin in patients with locally advanced or metastatic urothelial cancer who have been previously treated with a PD-1/L1 inhibitor, including those who have also been treated with a platinum-containing chemotherapy (cohort 1) and those who have not received a platinum-containing chemotherapy and who are ineligible for cisplatin (cohort 2). In cohort 1, 128 patients were enrolled at multiple centers internationally.2 The primary endpoint is confirmed objective response rate per blinded independent central review. Secondary endpoints include assessments of duration of response, disease control rate, progression-free survival, overall survival, safety and tolerability. EV-201 continues to enroll patients in cohort 2.

More information about enfortumab vedotin clinical trials can be found at clinical trials.gov.

About Urothelial Cancer

Urothelial cancer is the most common type of bladder cancer (90 percent of cases). In 2018, more than 82,000 people were diagnosed with bladder cancer in the United States.3 Globally, approximately 549,000 people were diagnosed with bladder cancer last year, and there were approximately 200,000 deaths worldwide.4

About Enfortumab Vedotin

Enfortumab vedotin is an investigational ADC composed of an anti-Nectin-4 monoclonal antibody attached to a microtubule-disrupting agent, MMAE, using Seattle Genetics’ proprietary linker technology. Enfortumab vedotin targets Nectin-4, a cell adhesion molecule that is expressed on many solid tumors, and that has been identified as an ADC target by Astellas.

The safety and efficacy of enfortumab vedotin are under investigation and have not been established. There is no guarantee that the agent will receive regulatory approval or become commercially available for the uses being investigated.

On September 16, 2019 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3-stage biopharmaceutical company discovering and developing novel products to treat cancer, with a focus on myelodysplastic syndromes (MDS),reported that it will present at the RAS-Targeted Drug Discovery Summit being held September 17-19 in Boston, Massachusetts (Press release, Onconova, SEP 16, 2019, View Source [SID1234539548]). Dr. Steven Fruchtman, President & CEO, will be presenting. Also attending the conference will be Avi Oler, VP Corporate Development.

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ABOUT RAS AND RIGOSERTIB:

There is a high frequency of Ras mutations in cancer that leads to the belief that mutations of the Ras Pathway provide a proliferative advantage and thus is involved in the pathogenesis of cancer. As a result, targeting the Ras pathway has been the objective of scientific research for decades. As published in the journal Cell in 2016, and now under investigation in a pivotal Phase 3 Trial, rigosertib targets the mutated RAS pathway by its interaction with Ras effector proteins containing the Ras Binding Domain. The RAS-Targeted Drug Discovery Summit provides an opportunity to showcase the potential for rigosertib in MDS and in other RAS-driven cancers, such as KRAS-mutated lung cancer and colorectal cancer, and Ras- driven pediatric cancers as well. Onconova will review its INSPIRE Trial for which the Company anticipates reporting top-line data for second-line, higher-risk MDS patients in the first half of 2020 following full enrollment and 288 death events. Onconova’s representatives look forward to joining colleagues at the Summit to discuss advancements in rigosertib’s development and the progress the Company and others have made in targeting RAS.

DETAILS OF THE PRESENTATION:

Title: Rigosertib: Targeting the Ras+ Pathway and Clinical Trials in MDS and Beyond
Date/Time: Thursday, September 19th, 10:30 a.m.
Presenter: Steven M. Fruchtman, M.D.

On September 16, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for the treatment of cancer, reported that the first patient has been dosed in a Phase 1 clinical trial of KO-539, the Company’s first-in-class inhibitor of the menin-mixed lineage leukemia (menin-MLL) interaction, in patients with relapsed or refractory acute myeloid leukemia (AML) (Press release, Kura Oncology, SEP 16, 2019, View Source [SID1234539547]).

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"We believe KO-539 represents a differentiated approach to the treatment of patients with AML," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "Our preclinical data for KO-539 support the potential for potent, anti-tumor activity in multiple, genetically defined subsets such as tumors with MLL fusions and rearrangements and NPM1 mutations. With the initiation of this trial, Kura now has three wholly-owned, clinical-stage oncology assets, along with the financial resources to advance each program through important inflection points."

This Phase 1, open-label, dose-escalation study is designed to determine the maximum tolerated dose (MTD) of KO-539 in patients with relapsed or refractory AML. KO-539 will be administered as a once daily oral dose in 28-continuous-day cycles. Upon completion of the dose-escalation portion of the trial, expansion cohorts are planned to further asses the safety and activity of KO-539 in specific genetic subgroups, such as NPM1. Additional information about the Phase 1 trial of KO-539 can be found at ClinicalTrials.gov using the identifier NCT04067336.

In July 2019, the U.S. Food and Drug Administration granted Orphan Drug Designation to KO-539 for the treatment of AML, recognizing the potential for KO-539 to address a population of patients with high unmet need.

About KO-539

KO-539 is a potent and selective small molecule inhibitor of the menin-MLL protein-protein interaction. MLL-rearranged leukemias are characterized by chromosomal translocations of the KMT2A gene that are primarily found in patients with AML and acute lymphoblastic leukemia (ALL). These translocations form oncogenes encoding MLL fusion proteins, which play a causative role in the onset, development and progression of MLL-rearranged leukemias. The target genes of the MLL fusion proteins are also found to be overexpressed in a broader subset of AMLs characterized by oncogenic driver mutations in genes, such as NPM1. These mutations also appear to be dependent on the interaction between menin and MLL, suggesting that the menin-MLL complex is a central node in epigenetic dysregulation driven by distinct oncogenic driver mutations known to be important in AML and other hematologic malignancies.

In preclinical studies, KO-539 has demonstrated potent and selective inhibition of the proliferation of MLL-rearranged leukemia cell lines. Kura has also generated preclinical data showing robust and durable anti-tumor activity in multiple in vivo models of AML characterized by MLL-rearrangements or oncogenic driver mutations in genes, such as NPM1.

On September 16, 2019 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported Mr. Jarrod Longcor, chief business officer of Cellectar presented data from Cohort 6 of its Phase 1 dose escalation study of CLR 131 in relapsed or refractory multiple myeloma (R/R MM), in a late breaker poster at the 17th International Myeloma Workshop being held in Boston, MA from September 12-15, 2019 (Press release, Cellectar Biosciences, SEP 16, 2019, View Source [SID1234539546]).

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The poster, entitled: "CLR 131 Demonstrates High Rate of Activity in a Phase 1, Dose Escalation Study in Patients with Relapsed or Refractory Multiple Myeloma (RRMM)" highlights data from 4 subjects in Cohort 6 who received a fractionated dose of 37.5 mCi/m2. Subjects in this cohort achieved a 50% overall response rate, with two subjects achieving a partial response and two subjects achieving minimal responses (39% and 48% reduction in M protein). CLR 131 was deemed safe and tolerated in all subjects with cytopenias being the only reported treatment emergent adverse events of grade 3 or higher. The majority (75%) of the subjects had high risk cytogenetics where median bone marrow plasma cell involvement was 25%. Patients’ median age was 72.5 and averaged 5 prior systemic therapies, with one patient being dual class refractory, one being quad-refractory, and two being penta-refractory.

The Phase 1 study employs the International Myeloma Working Group (IMWG) criteria for measuring responses. The IMWG defines a partial response as a 50% reduction in the marker of disease and a minimal response as a 25% to 49.9% reduction.

"Cohort 6’s overall response rate of 50% with 100% disease control in highly chemo-refractory elderly patients highlights CLR 131’s potential as a first-in-class targeted radiotherapeutic for relapsed or refractory multiple myeloma. We saw an encouraging dose response compared to prior cohorts and CLR 131 continues to demonstrate a favorable safety profile," said James Caruso, president and CEO of Cellectar. "We have progressed to a higher 40 mCi/m2 fractionated dose Cohort 7, with data expected in Q4 2019. Additionally, based on the positive results from Cohort 6, we are now allowed to use the 37.5 mCi/m2 dosing level in our ongoing Phase 2 (CLOVER-1) study evaluating CLR 131 in patients with relapsed/refractory (R/R) B-cell malignancies and expect data from the Phase 2 trial in Q4 2019."

About the Phase 1 R/R MM Trial

The Phase 1 multicenter, open-label, dose-escalation study is designed to evaluate the safety and tolerability of CLR 131 administered as a 30-minute IV infusion, either as a single bolus dose or as two fractionated doses, in patients with R/R MM. All doses to date have been deemed safe and well tolerated by an independent Data Monitoring Committee (DMC). Based on the data and the recommendation of the DMC, the Company has initiated a Cohort 7 where patients will receive 40mCi/m2 fractionated dose of CLR 131.

Based upon the encouraging activity from the 37.5 mCi/m2 fractionated dose of CLR 131 in Cohort 6, the company is evaluating this dose level in a larger population in its Phase 2 (CLOVER-1) trial as well.

A copy of the poster presentation and materials can be accessed on the Posters and Publications section of the Cellectar website.

About CLR 131

CLR 131 is a small-molecule, cancer-targeting radiotherapeutic Phospholipid Drug ConjugateTM (PDC) designed to deliver cytotoxic radiation directly and selectively to cancer cells and cancer stem cells. CLR 131 is the company’s lead therapeutic PDC product candidate and is currently being evaluated in both Phase 2 and Phase 1 clinical studies. The FDA granted orphan drug designation for CLR 131 for the treatment of multiple myeloma as well as orphan drug and rare pediatric disease designations for CLR 131 for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. In addition to the ongoing Phase 1 dose-escalation study and the Phase 2 (CLOVER-1) trial, the company recently initiated a Phase 1 open-label, dose-escalating study in pediatric solid tumors and lymphoma to evaluate the safety and tolerability of a single intravenous administration of CLR 131 in up to 30 children and adolescents with cancers including neuroblastoma, sarcomas, lymphomas (including Hodgkin’s lymphoma) and malignant brain tumors.