10-Q – Quarterly report [Sections 13 or 15(d)]

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

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MBQ Pharma announces the First-in-Human Dose of MBQ-167 for Advanced Breast Cancer in a Phase 1 Clinical Trial in Puerto Rico with the dual targeted Rac/Cdc42 inhibitor

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.

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"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.

Ankyra Therapeutics Announces Clinical Trial Supply Agreement with Regeneron to Evaluate ANK-101 in Combination with Libtayo® (cemiplimab) in Patients with Cutaneous Squamous Cell Carcinoma

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]).

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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.

Legend Biotech Announces Exclusive, Global License Agreement for Certain CAR-T Therapies Targeting DLL3

On November 13, 2023 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, reported that Legend Biotech Ireland Limited, a wholly owned subsidiary of Legend Biotech, has entered into an exclusive, global license agreement (License Agreement) with Novartis Pharma AG for certain Legend Biotech chimeric antigen receptor T-cell (CAR-T) cell therapies targeting DLL3, including its autologous CAR-T cell therapy candidate, LB2102 (NCT05680922) (Press release, Legend Biotech, NOV 13, 2023, View Source [SID1234637584]). The License Agreement grants Novartis the exclusive worldwide rights to develop, manufacture and commercialize these cell therapies, and Novartis may apply its T-Charge platform to their manufacture.

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Legend Biotech is initiating clinical development of LB2102 for the treatment of extensive stage small cell lung cancer and large cell neuroendocrine carcinoma after the U.S. Food and Drug Administration (FDA) cleared its investigational new drug application in 2022. In 2023, the FDA granted the product candidate Orphan Drug Designation, a status conferred to drugs or biologics that are intended to treat, diagnose or prevent rare diseases and conditions.2,3

The Novartis T-Charge platform is a next-generation CAR-T cell therapy manufacturing platform designed to preserve T cell stemness and facilitate CAR-T cell expansion primarily in vivo. The T-Charge platform is designed to reduce the need for extensive culture time outside the body and results in T cells with greater proliferative potential, as well as fewer exhausted T cells.4 LB2102 would be the first application of T-Charge by Novartis to a cell therapy candidate targeting solid tumors.

"We believe LB2102 has an innovative CAR design and armor mechanism that increases its anti-tumor activity. The preclinical evidence shows that an autologous CAR-T could be a differentiated treatment option for patients with small cell lung cancer," said Guowei Fang, Chief Scientific Officer and Head of Business Development of Legend Biotech. "We are excited that a major pharmaceutical company with deep roots in oncology and cell therapy has chosen to further this product candidate in the clinic. We are delighted that a combination of our unique candidate design in LB2102 with the T-Charge platform may potentially offer transformative benefits to small cell lung cancer patients."

Under the License Agreement, Legend Biotech will conduct a Phase 1 clinical trial for LB2102 in the U.S. Novartis will conduct all other development for the licensed products.

Under the terms of the License Agreement, Legend Biotech will receive a $100 million upfront payment and will be eligible to receive up to $1.01 billion in clinical, regulatory and commercial milestone payments and tiered royalties. Closing of the transaction is subject to the parties’ receipt of any necessary consents or approvals, including the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.

Bayer Provides Update on Aliqopa® (copanlisib)

On November 13, 2023 Bayer reported that, following discussions with the U.S. Food and Drug Administration (FDA), it will work with the FDA on a voluntary withdrawal of the Aliqopa (copanlisib) U.S. New Drug Application for adult patients with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies (Press release, Bayer, NOV 13, 2023, View Source [SID1234637583]).

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Aliqopa was granted accelerated approval by the FDA in September 2017 based on CHRONOS-1, an open-label, single-arm Phase II study. The FDA required clinical benefit to be confirmed through the CHRONOS-4 study. In the study, the addition of Aliqopa to standard immunochemotherapy regimens did not meet the primary endpoint of progression-free survival benefit versus the standard immunochemotherapy control arm in patients with relapsed follicular lymphoma. Bayer intends to publish the results of CHRONOS-4 in a timely manner.

Bayer is exploring access options for patients currently receiving Aliqopa who have experienced a favorable response to treatment, whose treating physician supports continuing treatment with Aliqopa, and for whom there may be no suitable alternative treatments available. Patients currently being treated with Aliqopa should consult their healthcare provider. No new patients should be prescribed Aliqopa. For questions related to ongoing access, please contact Bayer Medical Communications at 1-888-84-Bayer.

About non-Hodgkin’s lymphoma
Non-Hodgkin’s Lymphoma (NHL) comprises a highly heterogeneous group of chronic diseases with poor prognosis. NHL is the most common hematologic malignancy and the tenth most common cancer worldwide.

About Copanlisib (Aliqopa)
Copanlisib is currently approved in the U.S., China and Taiwan as a monotherapy under the brand name Aliqopa for the treatment of adult patients with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies.