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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

On September 16, 2019 ImmunoPrecise Antibodies reported that it will participate in the 17th Annual Discovery on Target (DOT) being held Boston, MA September 16-19 (Press release, ImmunoPrecise Antibodies, SEP 16, 2019, View Source [SID1234539545]). With over 1,300 attendees and a variety of keynote speakers, training seminars, short courses, roundtables, and networking functions, DOT is said to be the industry’s preeminent event on novel drug target and technologies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Jennifer Bath, Ph.D., and Barry Duplantis, Ph.D., will be available at booth 308 to discuss with attendees how ImmunoPrecise Antibodies’ broad range of products and services, including our B cell Select antibody discovery platform, can streamline and accelerate their therapeutic antibody development efforts. Attendees can schedule a meeting with Jennifer or Barry by emailing Barry at [email protected] or by using the Brella networking app.

On September 16, 2019 Context Therapeutics, a clinical-stage biopharmaceutical company dedicated to advancing medicines for hormone driven cancers, reported a clinical collaboration with SOLTI, a leading collaborative and academic group in Spain dedicated to clinical and translational research in breast cancer (Press release, Context Therapeutics, SEP 16, 2019, View Source [SID1234539544]). This clinical collaboration, being supported by Context and sponsored by SOLTI, is a window of opportunity study (WOO) in the neoadjuvant setting for breast cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

During the study, patients participating in the ONAWA (ONApristone Window Assessment) trial will receive oral doses of the progesterone receptor antagonist, Apristor (onapristone ER). Patients will be biopsied on day one, followed immediately by treatment and a final biopsy after three weeks, on the day of their full lumpectomy or mastectomy. Data generated from this study is intended to confirm the Recommended Phase 2 Dose (RP2D) of Apristor and to provide comprehensive biomarker data to further validate that the antitumor activity of Apristor is driven through downregulation of progesterone receptor and associated signaling pathways. This data will support Context’s ongoing and planned Phase 2 studies in breast, ovarian, and endometrial cancers.

"We are thrilled to enter into this collaboration with SOLTI and facilitate this window of opportunity study. We expect that this study will provide additional biomarker data to support our planned Phase 2 studies in progesterone receptor positive (PR+) female cancers," said Martin Lehr, CEO of Context Therapeutics.

The study, conducted within SOLTI’s network, has two Principal Investigators, both members of SOLTI Governing Board: Dr. Meritxell Bellet, Senior Consultant at Breast Cancer Unit at Vall d’Hebron University Hospital and Dr. Cristina Saura, Head of Breast Cancer Unit at Vall d’Hebron University Hospital. "The majority of breast cancer patients have an hormone-dependent disease. The hormones estrogen and progesterone drive breast cancer progression in those patients, but antiestrogens are the only antihormonal therapy available to clinicians. As such, antiestrogen resistance is now a major clinical challenge," said Dr. Bellet. "We believe that a progesterone receptor antagonist has the potential to address antiestrogen resistance and/or potentiate antiestrogen activity, which we believe will lead to better outcomes for patients. This window of opportunity provides us with the first clinical opportunity to delve deep into how progesterone receptor antagonists modulate the breast cancer tumor and its microenvironment."

About Hormone Driven Breast Cancer
Hormone receptor positive (HR+) breast cancer is the most common form of breast cancer and accounts for more than 70% of all breast cancers. HR+ cancer is usually treated with antiestrogen therapies first that help stop tumor growth. For many patients, antiestrogen therapy becomes ineffective over time and the cancer becomes resistant to antiestrogen therapy. In this recurrent setting, progesterone receptor has emerged as a prominent resistance mechanism. It is estimated that there are over 750,000 patients with recurrent disease worldwide.

About Window of Opportunity Trials
Window of opportunity (WoO) trials take advantage of natural scheduling delays that occur between initial consultation and surgery. This allows learning about anticancer activity of the intervention in a patient who has not been exposed to previous therapies. Such studies can better define biological effects of the therapy and outline the target patient population for the following studies. In turn, development and identification of promising drugs could speed up. WoO trials are usually short, in the order of 2–6 weeks of treatment, making it difficult to achieve clinical endpoints such as objective response. The endpoint of WoO trial is usually biomarker modulation. The collection of before and after treatment tumor biopsies allows determination of extent of target inhibition, cell proliferation and apoptosis, and identification of other biomarkers.

About Apristor
Apristor (onapristone extended release) is a potent and specific antagonist of the progesterone receptor that is orally administered. Currently, there are no approved therapies that selectively target PR+ cancers. Preliminary preclinical and clinical data suggest that Apristor has anticancer activity by inhibiting the binding of progesterone receptor to chromatin, downregulating cancer stem cell mobilization, and blocking immune evasion. Apristor is an investigational drug that has not been approved for marketing by any regulatory authority.