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.

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.

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.

On November 13, 2023 Medivir AB (Nasdaq Stockholm: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, reported that clinical data from the ongoing phase 1b/2a study of in fostroxacitabine bralpamide (fostrox) in combination with Lenvima (lenvatinib) in hepatocellular carcinoma (HCC), will be presented at the ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium, January 18-20, 2024 in San Francisco, USA (Press release, Medivir, NOV 13, 2023, View Source;lenvima-in-hcc-and-shares-positive-response-from-type-d-meeting-with-fda-301985850.html [SID1234637582]).

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The abstract, titled "First safety and efficacy data from phase Ib/IIa study of fostroxacitabine bralpamide (fostrox, MIV-818) in combination with lenvatinib in patients with hepatocellular carcinoma (HCC)" will be presented by Dr. Maria Reig, Director of the Barcelona Clinic Liver Cancer (BCLC) and the Liver Oncology Unit at the Hospital Clinic of Barcelona in Spain. The presentation will include updated safety and independently reviewed efficacy data regarding the clinical benefit of fostrox in combination with Lenvima, a tyrosine kinase inhibitor.

Medivir furthermore announces that interactions with the FDA regarding fostrox’s clinical development plan have intensified with a first Type D meeting with a positive response regarding critical elements of the design for the planned phase 2b study. The finalization of the study design will take place in connection with an upcoming Type C meeting to enable study start in 2024. The goal of the upcoming study, as previously communicated, is to apply for accelerated approval.

– "In this phase 1b/2a study, the fostrox + Lenvima combination has shown a good safety profile and promising tumor control in second-line HCC, and we confidently look forward to presenting these data at ASCO (Free ASCO Whitepaper)-GI. Despite some success with first-line immunotherapy, patients with HCC have a poor prognosis and effective second-line treatment options are lacking. The data from this study, combined with the great medical need, opens the possibility of an accelerated approval path, which is why we are now planning for a pivotal, randomized phase 2b study. The FDA’s response at our Type D meeting was a positive step towards our ambition to give this vulnerable patient group access to fostrox as a new treatment option.", says Pia Baumann, CMO at Medivir.

On November 13, 2023 Intensity Therapeutics, Inc. (Nasdaq: INTS), a late-stage biotechnology company that applies novel engineered chemistry to discover and develop proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, reported financial results for the third quarter ended September 30, 2023, and provided a corporate update (Press release, Intensity Therapeutics, NOV 13, 2023, View Source [SID1234637581]).

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"Metastatic soft tissue sarcoma continues to plague cancer patients who have poor survival outcomes and insufficient therapeutic options. The positive overall survival and disease control rate data from our Phase 1/2 clinical trial of INT230-6 positions our lead candidate as a potential much needed reprieve, keeping the drug inside the tumor while sparing the body of toxicity. We expect to file an IND for a Phase 3 study of INT230-6 in soft tissue sarcoma by the end of 2023 and look forward to progressing this study," said Lewis H. Bender, President and Chief Executive Officer of Intensity.

Mr. Bender added: "To continue the momentum built from our successful upsized IPO in July, we also look forward to reporting additional results from our Phase 2 INVINCIBLE study in presurgical breast cancer at a medical meeting and finalizing the study design and protocol for a Phase 2/3 program in presurgical breast cancer before year end. I am pleased with our pace of progress to advance the clinical development of INT230-6 and am strongly encouraged by data to date, which reinforces the potential of INT230-6 to shift the treatment paradigm of cancer."

Recent Company Highlights

Presented Positive INT230-6 Data in Patients with Refractory Soft Tissue Sarcoma at the Connective Tissue Oncology Society (CTOS). In November, the Company presented positive data from its ongoing Phase 1/2 clinical trial of INT230-6 at the CTOS annual meeting. The data presented demonstrated a strong disease control rate of 93% for subjects in the monotherapy arm while also extending survival in subjects by nearly 15 months when compared to a synthetic control group. INT230-6 was found to be generally safe and well tolerated with the majority of treatment-emergent adverse events being grade 1 or 2.
Received Orphan Drug Designation for Components of INT230-6 for the Treatment of Soft Tissue Sarcoma. In September, Intensity was granted orphan drug designation (ODD) by the US Food and Drug Administration for three active moieties comprising its lead candidate INT230-6: cisplatin, vinblastine sulfate, and the diffusion enhancer SHAO-FA (8-((2-hydroxybenzoyl) amino)octanoate).
Completed Upsized Initial Public Offering (IPO) with Full Exercise of the Over-Allotment Option. In July, Intensity announced that it had closed its IPO with the full exercise of its over-allotment option. Intensity received a total of $20.2 million in net proceeds from the transaction, providing sufficient cash and cash equivalents to fund operations into the second half of 2025.
Anticipated Near-Term Milestones

Report additional results from the Phase 2 INVINCIBLE study in presurgical breast cancer at a medical meeting in 4Q 2023
File an Investigational New Drug (IND) application for a Phase 3 study of INT230-6 in soft tissue sarcoma in 4Q 2023
Finalize the study design for a Phase 2/3 program in presurgical breast cancer in 4Q 2023
Third Quarter 2023 Financial Highlights

Research and Development (R&D) Expenses were $1.4 million for the three months ended September 30, 2023, as compared to $1.2 million for the same period last year. The increase is due to ongoing Phase 3 IT-03 in sarcoma and phase 2/3 IT-04 in presurgical breast cancer, which will continue to incur planning, multiple regulatory filing, manufacturing, study initiation and trial preparation costs in 2023.

General and Administrative (G&A) Expenses were $1.1 million for the three months ended September 30, 2023, as compared to $0.6 million for the same period in 2022. The increase is primarily due to the costs of operating as a public company. The accounting services and legal costs related to the IPO in 2023 were charged directly to the equity section of the balance sheet as a reduction of additional paid in capital.

Interest Expense for the three months ended September 30, 2023, were $0 as compared to $15,123 for the three months ended September 30, 2022. The decrease is due to the convertible notes and accrued interest being converted to common stock at the time of the IPO.

Net Operating Loss for the third quarter ended September 30, 2023, was $2.3 million as compared to $1.8 million for the three months ended September 30, 2022.

Cash, Cash Equivalents and Marketable Securities as of September 30, 2023, were approximately $15.6 million. The Company expects to have sufficient cash to fund current operations into the second half of 2025.

About INT230-6
INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug is composed of two proven, potent anti-cancer agents, cisplatin and vinblastine, and a penetration enhancer molecule (SHAO) that helps disperse potent cytotoxic drugs throughout tumors for diffusion into cancer cells. These agents remain in the tumor resulting in a favorable safety profile. In addition to local disease control, direct killing of the tumor by INT230-6 releases a bolus of neoantigens specific to the patient’s malignancy, leading to engagement of the immune system and systemic anti-tumor effects. Importantly, these effects are mediated without immunosuppression that so often occurs with systemic chemotherapy.