Interius BioTherapeutics Receives HREC Approval and CTN Clearance from the TGA to Commence a Phase 1 Clinical Trial for Its First-in-Class In Vivo CAR Therapeutic for B Cell Malignancies

On July 9, 2024 Interius BioTherapeutics, a leading developer of in vivo cell-specific gene medicines, reported that it has been granted Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance by the Australian Therapeutic Goods Administration (TGA) to commence a first-in-human Phase 1 clinical trial of INT2104, its lead in vivo CAR candidate for treatment of B-cell malignancies (Press release, Interius BioTherapeutics, JUL 9, 2024, View Source [SID1234644747]).

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"Receiving HREC approval and CTN clearance for our first clinical trial is a significant milestone for Interius. We are very pleased that the regulators have approved the start of our first-in-human clinical trial for INT2104. The approval allows us to enroll patients in our first clinical study and recognizes the potential of our novel in vivo targeted gene therapy candidate, INT2104, to address an unmet medical need for patients with B cell malignancies," said Interius President and Chief Executive Officer Phil Johnson, M.D. "We look forward to continuing to work closely with the TGA and other regulators in the future to bring this innovative therapy to patients as quickly as possible."

The CTN clearance was granted based on HREC approval in Australia which included extensive review of Interius’s preclinical data and study protocol. Interius intends to begin the trial in the fourth quarter of 2024 and is well positioned to deliver key program milestones as early as the first quarter of 2025.

About the INT2104 Clinical Program
Interius’s Phase 1 trial (INVISE, Injectable Vectors for In Situ Engineering) will evaluate the safety of a single INT2104 infusion in adults with refractory/relapsing B cell malignancies. The Phase 1 study is a global, two-part, multicenter, open-label, single dose design with a dose escalation portion designed to inform the dose of INT2104 to be used in the dose confirmation part of the trial and future studies.

About INT2104
INT2104 is a wholly-owned investigational gene therapy candidate, which specifically targets CD7-positive T and NK cells and delivers a CAR transgene to create effector CAR-T and CAR-NK cells in vivo. The CAR cells target CD20-positive B cells for the treatment of B cell malignancies. Unlike ex vivo CAR-T therapies, INT2104 is an off-the-shelf, single dose treatment, administered systemically through intravenous infusion without the need for lymphodepletion or for any special equipment or training.

Elevar Therapeutics Reports Plans for Near-Term Resubmission of NDA for First-Line Treatment Option for Unresectable Hepatocellular Carcinoma Following Type A FDA Meeting

On July 9, 2024 Elevar Therapeutics, Inc. (Elevar), a majority-owned subsidiary of HLB Co., Ltd., reported near-term plans for resubmission of the NDA for rivoceranib in combination with camrelizumab as a first-line treatment option for Unresectable Hepatocellular Carcinoma (uHCC) after a Type A meeting with the FDA on July 2 (Press release, Elevar Therapeutics, JUL 9, 2024, View Source [SID1234644744]). The meeting was requested by Jiangsu Hengrui Pharmaceuticals (Hengrui Pharma) to address items cited in the May 16 FDA Complete Response Letters (CRLs) sent to Hengrui Pharma and Elevar. The original NDA was submitted by Elevar in May 2023.

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The CRL cited GMP deficiencies at the Hengrui Pharma facility where camrelizumab is manufactured and incomplete Bioresearch Monitoring (BIMO) clinical inspections due to FDA travel restrictions. The FDA did not indicate any issues related to clinical data or with the manufacturing site for rivoceranib.

Prior to the Type A meeting, the FDA had accepted Hengrui Pharma’s written responses to GMP deficiencies. During the meeting, the FDA confirmed resubmission can occur without delay. The FDA also confirmed BIMO inspections due to FDA travel restrictions may occur after resubmission.

"The most critical outcome from our discussion with the FDA is that resubmission of Elevar’s NDA can occur without further remediation at the Hengrui manufacturing site," commented Dr. Saeho Chong, Elevar chief executive officer. "Elevar left the meeting very motivated and with a clear path forward for resubmission, so patients and providers can soon have access to this novel combination therapy for uHCC where there continues to be a high unmet need. The resubmission will include the CARES-310 landmark analysis recently presented at ASCO (Free ASCO Whitepaper), demonstrating the longest median overall survival (mOS) of 23.8 months for any treatment in a global Phase 3 trial for patients with uHCC."

About Hepatocellular Carcinoma (HCC) HCC is the most common type of primary liver cancer. It most frequently develops in people with chronic underlying liver inflammation which may be from viral and non-viral causes. HCC typically has a poor prognosis with limited treatment options and continues to be a diagnosis with an ongoing urgent medical need.

About CARES-310 CARES-310 (NCT03764293) was a randomized, open-label, international Phase 3 study, which included 543 patients with unresectable or metastatic HCC who had not received prior systemic therapy. Patients were randomized 1:1 to receive the combination of camrelizumab + rivoceranib or sorafenib (400 mg orally twice daily), a standard-of-care first-line multi-kinase inhibitor treatment for uHCC. Camrelizumab was administered intravenously (190 mg) every two weeks and rivoceranib was administered orally (250 mg) once daily. The study was conducted at 95 study sites across 13 countries/regions, in which systemic treatment for uHCC independent of etiology, rivoceranib plus camrelizumab demonstrated statistically significant and clinically meaningful prolonged overall survival and progression-free survival and improved overall response rate versus sorafenib. The co-primary endpoints were overall survival and progression-free survival. Secondary endpoints included objective response rate and duration of response.

About Camrelizumab Camrelizumab (SHR-1210) is a humanized monoclonal antibody that binds to the programmed death-1 (PD-1) receptor. Blockade of the PD-1/PD-L1 signaling pathway is a therapeutic strategy showing success in a wide variety of solid and hematological cancers. Camrelizumab is developed by Hengrui Pharma and has been studied in more than 5,000 patients. Currently, 50 clinical trials are underway in a broad range of tumors (including liver cancer, lung cancer, gastric cancer, and breast cancer, etc.) and treatment settings. Camrelizumab, under the brand name AiRuiKa, is currently approved for eight indications in China, including monotherapy for the treatment of HCC (second-line), in combination with rivoceranib as a treatment for uHCC (first-line), relapsed/refractory classic Hodgkin’s lymphoma (third-line), esophageal squamous cell carcinoma (second-line) and nasopharyngeal carcinoma (third-line or further) and in combination with chemotherapy for the treatment of non-small cell lung cancer (non-squamous and squamous), esophageal squamous cell carcinoma and nasopharyngeal carcinoma in the first-line setting. The U.S. Food and Drug Administration granted Orphan Drug Designation to camrelizumab for advanced HCC in April 2021.

In October 2023, Elevar licensed camrelizumab, an anti-PD-1 antibody, for commercialization from Jiangsu Hengrui Pharmaceuticals Co., Ltd. (Hengrui Pharma) worldwide excluding Greater China and Korea.

About Rivoceranib Rivoceranib, a small-molecule tyrosine kinase inhibitor (TKI), is a highly potent inhibitor of vascular endothelial growth factor receptor 2 (VEGFR-2), a primary pathway for tumor angiogenesis. VEGFR-2 inhibition is a clinically validated approach to limit tumor growth and disease progression. Rivoceranib is currently being studied as a monotherapy and in combination with chemotherapy and immunotherapy in various solid tumor indications. Ongoing clinical studies include uHCC (in combination with camrelizumab), gastric cancer (as a monotherapy and in combination with paclitaxel), adenoid cystic carcinoma (as a monotherapy) and colorectal cancer (in combination with Lonsurf). Rivoceranib was the first TKI approved in gastric cancer in China (November 2014). It is also approved in China in combination with camrelizumab as a first-line treatment for uHCC (January 2023). The drug has been studied in more than 6,000 patients worldwide and was well tolerated in clinical trials with a comparable safety profile to other TKIs and VEGF inhibitors. Orphan drug designations have been granted in gastric cancer (U.S., EU and South Korea), in adenoid cystic carcinoma (U.S.) and in uHCC (U.S.). Elevar Therapeutics, Inc. holds the global rights (excluding China) to rivoceranib and has partnered for its development and marketing with HLB-LS in South Korea. Rivoceranib, under the name apatinib, is also approved in China for advanced gastric cancer and in second-line advanced HCC by the Chinese -territory license-holder, Jiangsu Hengrui Pharmaceuticals Company Ltd., (Hengrui Pharma), under the brand name Aitan.

About Elevar Therapeutics Elevar Therapeutics, Inc. is a fully integrated biopharmaceutical company built on the promise of elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options. With expertise rooted in oncology, Elevar is focused on identifying and developing promising medicines for complex yet under-treated health conditions. Elevar’s lead proprietary drug candidate is rivoceranib. Elevar is headquartered in New Jersey, with offices in South Korea. Additional information is available at ElevarTherapeutics.com.

Curium Submits New Drug Application for Lutetium Lu 177 Dotatate Injection

On July 9, 2024 Curium, a world leader in nuclear medicine, reported that it has submitted its 505(b)(2) New Drug Application for Lutetium Lu 177 Dotatate Injection, which, if approved by the U.S. Food and Drug Administration (FDA), would be intended for the treatment of somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETS) (Press release, Curium, JUL 9, 2024, View Source [SID1234644743]). Curium’s submission represents a major step in its strategy to become a key player in the promising nuclear medicine therapeutic field.

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Curium has been developing its formulation over the past several years and has ensured that its formulation avoided infringement of any Orange Book listed patents in order to expedite its path to market. Curium is excited to embark on the next phase of its journey toward delivering cancer therapies to the market as part of its mission to redefine the experience of cancer through its trusted legacy in nuclear medicine.

Curium will work closely with the FDA through the review and approval process. Curium looks forward to potential approval of its Lutetium Lu 177 Dotatate formulation so that more patients suffering from GEP-NETS may have access to the potential benefits of radioligand therapy.

Michael Patterson, Curium North America CEO commented: "Curium’s 505(b)(2) new drug application for Lutetium Lu 177 Dotatate Injection marks another important milestone for Curium in its evolution to bring new tools for patients and healthcare providers to both diagnose and now potentially treat disease. Nuclear medicine-based cancer therapies must continue to be more widely and reliably available for patients and their caregivers."

Curium will provide further updates as it proceeds with its application through the FDA’s review process.

C4 Therapeutics to Participate in the Virtual UBS Targeted Protein Degradation Day

On July 08, 2024 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported that C4T management will participate in a fireside chat at the UBS Targeted Protein Degradation (TPD) Day taking place virtually on July 15, 2024 (Press release, C4 Therapeutics, JUL 8, 2024, View Source [SID1234644707]).

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Epsilogen announces CTA approval for Phase Ib trial of MOv18 IgE in platinum-resistant ovarian cancer

On July 8, 2024 Epsilogen, a global leader in the development of immunoglobulin E (IgE) antibodies to treat cancer, reported that the Clinical Trial Application for the Phase Ib trial of MOv18 IgE has been approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA) (Press release, Epsilogen, JUL 8, 2024, View Source [SID1234644727]).

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The Phase Ib study is expected to initiate later in 2024 and will evaluate the efficacy of MOv18 IgE in patients with platinum-resistant ovarian cancer (PROC).

Dr Tim Wilson, Chief Executive Officer of Epsilogen, said: "This CTA is another significant milestone for Epsilogen and the clinical development of MOv18 IgE. We look forward to progressing MOv18 IgE into a Phase Ib efficacy study later this year as we continue to demonstrate the potential of IgE antibodies as a new, differentiated class of cancer treatments."

About MOv18 IgE

MOv18 IgE is an immunoglobulin E (IgE) antibody targeting the folate receptor alpha (FR alpha) antigen. FR alpha is present on a variety of cancers including ovarian, endometrial, lung and triple negative breast cancer. Epsilogen has successfully completed a Phase I safety study of MOv18 IgE in PROC patients. The results of the study, published in Nature Communications, found MOv18 IgE to be safe and well tolerated, with evidence of anti-tumour activity observed. Epsilogen, alongside its partner Lonza, also announced the successful completion of large-scale Good Manufacturing Practice (GMP) manufacturing of MOv18 IgE earlier this year.

About the Phase Ib study

The Phase Ib study is designed to confirm the safety and tolerability of MOv18 IgE and demonstrate efficacy in PROC. Following the dose escalation, an expansion cohort will be recruited to make a preliminary assessment of the anti-tumour activity of MOv18 IgE at a selected dose. In addition, delay to disease progression will be assessed along with a number of translational elements to generate further understanding of MOv18 IgE in the study population.