On November 13, 2023 Oncopeptides, a biotech company focused on difficult-to-treat cancers, reported that long-term outcomes from its Phase 3 OCEAN study has been accepted as a poster and will be presented at the 65th annual American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting and Exposition (Press release, Oncopeptides, NOV 13, 2023, View Source [SID1234646781]). The conference takes place in San Diego between December 9-12.

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!

ASH is the world’s largest professional hematologic society serving both clinicians and scientists around the world who are working to conquer blood diseases.

The phase 3 OCEAN study is a global, randomized, head-to-head, open-label study, evaluating the efficacy and safety of melflufen and dexamethasone, versus pomalidomide and dexamethasone in patients with relapsed refractory multiple myeloma who have received 2-4 prior therapies.

"This long-term follow-up of OCEAN confirms the favorable safety and overall survival outcomes of melflufen + dexametason in patients that have not progressed within 36 months after a stem cell transplantation and supports its use in patients with relapsed, refractory multiple myeloma," says Fredrik Schjesvold, MD, Head of Oslo Myeloma Center, Oslo University Hospital. "We are happy to have been selected to present our findings at ASH (Free ASH Whitepaper) and look forward to the opportunity."

Find more details about the abstract and presentation below. The abstract including key data has been published and is available through this link.
For more information about ASH (Free ASH Whitepaper) Annual Meeting and Exposition, click here.

Title Long-Term Outcomes from the Phase 3 OCEAN (OP-103) Study: Melflufen and Dexamethasone (Dex) Versus Pomalidomide (Pom) and Dex in Relapsed Refractory Multiple Myeloma (RRMM)
Publication Number 2018
Presenting author Fredrik Schjesvold, MD, Head of Oslo Myeloma Center, Oslo University Hospital
Program Oral and Poster Abstracts
Session 653. Multiple Myeloma: Prospective Therapeutic Trials: Poster I

On November 13, 2023 Rezolute, Inc. (Nasdaq: RZLT), a clinical-stage biopharmaceutical company committed to developing novel, transformative therapies for serious metabolic and rare diseases, reported its financial results for the first quarter of fiscal 2024 ended September 30, 2023 (Press release, Rezolute, NOV 13, 2023, View Source [SID1234639828]).

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 are on track to commence our Phase 3 study for RZ358 to treat congenital hyperinsulinism prior to year-end and are delighted to have recently obtained PRIME eligibility from the European Medicines Agency for this indication," said Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute. "We also anticipate completing enrollment this quarter for our ongoing Phase 2 study of RZ402 for the treatment of diabetic macular edema and plan to provide an update on the study prior to year end."

Clinical Highlights

· RZ358, a monoclonal antibody for the treatment of hyperinsulinism
o Received EMA priority medicines (PRIME) designation for the treatment of congenital hyperinsulinism (cHI) from the European Medicines Agency
o Plan to initiate sunRIZE, a pivotal Phase 3 clinical study in patients with cHI, in Europe and other geographies outside the US in the fourth quarter 2023
o Continuing the administration of RZ358 in the US with FDA approval under a compassionate use program to treat patients with tumor associated hyperinsulinism, including for a patient with refractory hypoglycemia due to metastatic insulinoma who has remained on RZ358 for nearly a year

· RZ402, an oral plasma kallikrein inhibitor in Phase 2 to treat diabetic macular edema (DME)
o Multi-center, randomized, double-masked, placebo-controlled, parallel-arm study ongoing to evaluate the safety, efficacy, and pharmacokinetics of RZ402 administered as a monotherapy over a 12-week treatment period in participants with DME who are naïve to or have received limited anti-VEGF injections
o Study is in the latter stages of patient recruitment and an update on the study will be provided prior to year end

First Quarter Fiscal 2024 Financial Results

· Cash, cash equivalents and investments in marketable debt securities totaled $106.9 million as of September 30, 2023, compared to $118.4 million as of June 30, 2023

· Research and development expenses were $12.2 million for the first quarter of fiscal 2024, compared to $7.7 million for the same period in fiscal 2023, with the increase primarily attributable to increased expenditures in clinical trial activities, manufacturing costs and higher personnel-related expenses, which included employee compensation and stock-based compensation

· General and administrative expenses were $3.7 million for the first quarter of fiscal 2024, compared to $2.5 million for the same period in fiscal 2023, with the increase primarily attributable to higher personnel-related expenses, including employee compensation and stock-based compensation

· Net loss was $14.5 million for the first quarter of fiscal 2024, compared to $9.8 million for the same period in fiscal 2023

On November 13, 2023 Kronos Bio reported its quarterly results (Filing, 3 mnth, SEP 30, Kronos Bio, 2023, NOV 13, 2023, View Source [SID1234637958]).

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!

On November 13, 2023 MBQ Pharma Inc., a pioneering clinical-stage biopharmaceutical company dedicated to advancing innovative treatments for solid tumor cancers, reported a significant milestone in its journey (Press release, MBQ Pharma, NOV 13, 2023, View Source [SID1234637903]). We are proud to announce that we have dosed the first participant in our Phase 1 clinical trial of MBQ-167. MBQ-167 is the first-in-class drug as a dual inhibitor designed to target two GTPase proteins: Rac and Cdc42. Overexpression of these proteins in cancer cells are considered the primary drivers of solid tumor cancer spread and of cancer cells developing resistance to treatment. MBQ Pharma is extremely proud that MBQ-167 was discovered at the University of Puerto Rico by innovative scientists and that MBQ Pharma has successfully initiated this trial for patients who need additional options after all possible standard cancer therapies have been attempted. This exciting development marks a decisive step forward in the fight against Advanced Breast Cancer (ABC). MBQ Pharma is grateful to the volunteer participants and referring oncologists.

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 are thrilled to have provided this first dose to our first participant in this important study," said Dr. José F. Rodríguez-Orengo, CEO of MBQ Pharma Inc. "We want to recognize and thank the patient volunteers who are suffering from this horrible disease, their family members and caregivers that support them. Additionally, I want to thank our team members and collaborators who have worked tirelessly to bring this innovative drug to eligible patients enduring a hard battle against ABC. We are committed to advancing MBQ-167 into the clinic with the hope of delivering a new safe and effective treatment option for patients with Advanced Breast Cancer who have failed all available standard of care therapies."

This Phase 1 clinical trial is an open-label, dose-escalation study aimed at establishing the maximum tolerated dose (MTD) of MBQ-167 in patients with ABC. MBQ-167 will be administered orally twice daily for a continuous period of 21 days. Eligible participants may continue to take MBQ-167 twice a day until stopping the drug due to disease progression or other reasons. The trial is being conducted by investigators at FDI Clinical Research in San Juan, PR. You can find further details about the Phase 1 trial of MBQ-167 and contact information for FDICR by visiting ClinicalTrials.gov and using the identifier NCT06075810.

In November 2022, the Congressional Directed Medical Research Program (CDMRP) administered by the US Department of Defense awarded a breakthrough multimillion-dollar grant to MBQ Pharma to initiate the clinical phase of this promising product, aimed at improving patient care for participants with advanced cancer involving metastatic disease. The grant’s support enables the rigorous testing and evaluation within this First-in-Human trial, bringing the innovative solution closer to becoming a reality for those in need.

About MBQ-167
MBQ-167 represents a highly potent and selective small molecule inhibitor, specifically targeting GTPases Rac and Cdc42. We intend to demonstrate in this clinical trial that the inhibition of Rac and Cdc42 GTPases achieved by MBQ-167 can have a profound impact on cancer cells in humans by impeding the proliferation, migration, and invasion of these cells and effectively reducing or preventing their spread to other organs. Preclinical data have demonstrated that this inhibition not only curtails new metastasis but can also have a remarkable inhibition of tumor growth (90%).
Notably, in participants with Advanced Breast Cancer (ABC) and in patients who suffer with many other common cancers such as Lung, Ovarian, Melanoma, Bladder and Pancreatic, the overexpression of Rac and Cdc42 in tumor cells is associated with elevated mortality rates, primarily due to an increased tendency for metastasis.

Additional preclinical investigations have showcased the remarkable effectiveness of MBQ-167, as it exhibited potent and highly selective inhibition of the proliferation of various breast cancer cell lines, encompassing both HER2+ and TNBC (Triple-Negative Breast Cancer) subtypes and in a Pancreatic cancer cell line. MBQ Pharma’s extensive preclinical data reveal, not only the robustness of this inhibition, but also its capacity to deliver enduring antitumor effects with minimal associated toxicity. These findings underscore the promising potential of MBQ-167 as an alternative therapeutic option both as a single-agent and in combination therapy for a broad spectrum of breast cancer patients and potentially many other highly metastatic cancer types.

On November 13, 2023 Ankyra Therapeutics, a clinical stage biotechnology company developing a new form of local immunotherapy termed "anchored immunotherapy," reported a clinical trial supply agreement with Regeneron to evaluate ANK-101 in combination with Regeneron’s anti-PD-1 therapy, Libtayo (cemiplimab) (Press release, Ankyra Therapeutics, NOV 13, 2023, View Source [SID1234637585]).

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 Ankyra platform uses an inert aluminum hydroxide scaffolding to link to bioactive immuno-oncology agents. Preclinical studies of ANK-101, a functional interleukin-12 (IL-12) cytokine, have demonstrated retention within the tumor microenvironment for up to 28 days with limited systemic diffusion or toxicity. Significant monotherapy anti-tumor activity has been seen in multiple murine tumor models and in a Phase I clinical trial of canine melanoma. Further studies have shown that ANK-101 drives expression of local PD-L1 and pre-clinical combination studies with PD-1 blockade demonstrated improved therapeutic activity in PD-1-refractory tumor models.

"We are excited to expand our clinical collaborations to evaluate the combination of ANK-101 with cemiplimab in cutaneous squamous cell carcinoma (CSCC)," said Dr. Joe Elassal, Chief Medical Officer at Ankyra. "Cemiplimab has been a major step forward for patients with advanced CSCC and we believe that this is a tumor where combination therapeutic effects are highly likely."

The combination study will proceed following Ankyra’s first-in-human phase I clinical trial, which is starting in the first quarter of 2024.

Dr. Howard L. Kaufman, President and Chief Executive Officer at Ankyra added, "We are especially pleased to be working with Regeneron, leaders in the CSCC space, and look forward to building a strong relationship with Regeneron to improve the lives of patients with this common form of skin cancer."

Libtayo is a registered trademark of Regeneron in Tarrytown, NY.

About ANK-101

ANK-101 is an intratumoral drug complex composed of interleukin-12 (IL-12) linked to aluminum hydroxide. ANK-101 allows local delivery of functional IL-12 to the tumor microenvironment where it remains biologically active for several weeks but does not diffuse into the systemic circulation thereby avoiding systemic toxicity. Treatment with ANK-101 in animal models has been associated with recruitment and retention of tumor-specific CD8+ T cells, NK cells and M1 macrophages activating innate and adaptive anti-tumor immunity. ANK-101 is being evaluated for the treatment of advanced solid tumors alone and in combination with anti-PD-1 agents.