Factor Bioscience to Deliver Six Presentations at the International Society for Stem Cell Research (ISSCR) 2024 Annual Meeting

On July 9, 2024 Factor Bioscience Inc., a Cambridge-based biotechnology company focused on developing mRNA and cell-engineering technologies, reported its participation in the International Society for Stem Cell Research (ISSCR) 2024 Annual Meeting to be held in Hamburg, Germany from July 10-13, 2024 (Press release, Factor Bioscience, JUL 9, 2024, View Source [SID1234644763]). Factor will deliver six presentations covering the latest preclinical data from Factor’s cell engineering platforms.

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"We are excited to showcase our recent progress on developing next-generation therapies based on cutting-edge stem cell science at ISSCR 2024," said Dr. Matt Angel, Co-Founder, Chairman and CEO of Factor. "The work that we will be presenting this week represents more than a decade of focused effort. We are committed to developing these new medicines to enable a brighter future for patients and their families."

Dr. Kyle Garland, Factor’s Director of Translational Science, added, "Our six presentations at ISSCR 2024 will cover several novel and unique stem cell technologies, including iPSC-derived macrophages engineered with mRNA to enhance T cell cytotoxicity to solid tumor cells. We are excited to share these and other advances in Hamburg over the next few days."

Details of the presentations are below:

"Engineered iPSC-Derived Macrophages Evade Host-Versus-Graft Alloreactivity and Enhance T Cell Cytotoxicity to Triple Negative Breast and Ovarian Cancer Cells In Vitro." -to be presented by Ian Hay on July 12 from 1:30-3:00 pm CEST, PSC-Based Cell Therapies Session (Oral Presentation), Hall 3 – Entrance Level.

"B2M-KO iMSCs Better Suppress T Cell Proliferation by Upregulating IDO1 in Response to Proinflammatory Signals." -to be presented by Raven Dance Hinkel on July 10 from 5:45-6:45 pm CEST, Clinical Applications (CA) Session I: (Poster Presentation #165), Hall H – Entrance Level.

"Donor-Sequence Optimization Enables Targeted Insertion of Complex Stealthing Constructs in mRNA-Reprogrammed Induced Pluripotent Stem Cells." -to be presented by Elizabeth Belcher on July 10 from 6:45-7:45 pm CEST, Clinical Applications (CA) Session I: (Poster Presentation #228), Hall H – Entrance Level.

"Novel Polyvalent Ionizable Lipids Enable Targeted Delivery of mRNA to Immune Cells and iPSC-derived MSCs." -to be presented by Ariadna Lubinus on July 11 from 3:45-4:45 pm CEST, New Technologies (NT) Session II: (Poster Presentation #509), Hall H – Entrance Level.

"Novel Regulatory Sequences Drive Persistent Transgene Expression During Directed Differentiation of iPSCs to Lymphocytes and Macrophages." -to be presented by Claire Aibel on July 11 from 4:45-5:45 pm CEST, New Technologies (NT) Session II: (Poster Presentation #368), Hall H – Entrance Level.

"Reporter-Free Generation of iPSC-derived Tissue-Specific Cells Engineered for the Stable Expression of Immunomodulatory Proteins." -to be presented by Taeyun Kim on July 11 from 4:45-5:45 pm CEST, New Technologies (NT) Session II: (Poster Presentation #394), Hall H – Entrance Level.
For more information about the International Society for Stem Cell Research (ISSCR) 2024 Annual Meeting, visit www.isscr2024.org.

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.

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.

Termination of a Material Definitive Agreement

On July 9, 2024, Bicycle Therapeutics plc (the "Company") reported that it has repaid in its entirety and voluntarily terminated its loan and security agreement, dated September 30, 2020 (as amended from time to time, the "Loan Agreement"), by and among the Company, certain of its subsidiaries and Hercules Capital, Inc. ("Hercules") (Filing, 8-K, Bicycle Therapeutics, JUL 9, 2024, View Source [SID1234644767]). The Loan Agreement provided for term loans in an aggregate principal amount of up to $75.0 million (the "Term Loans"), of which $30.0 million was outstanding and which bore interest at an annual rate equal to the prime rate as reported in the Wall Street Journal plus 4.55%, with a minimum annual rate of at least 8.05%, capped at a rate no greater than 9.05%.

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The Term Loans were scheduled to mature on July 1, 2025. The Company elected to repay all amounts outstanding, including accrued and unpaid interest, an end-of-term charge of $1.5 million and a prepayment charge of $0.3 million, for a total aggregate payment of $31.9 million, using cash on hand. As collateral for the obligations under the Loan Agreement, the Company granted to Hercules a senior security interest in all of Company’s right, title and interest in, to and under substantially all of Company’s personal property and other assets, other than its intellectual property, and, upon the termination of the Loan Agreement, all security interests granted to the secured parties thereunder were terminated and released.

The Loan Agreement also included customary affirmative and restrictive covenants, representations and warranties and events of default, as more fully set forth in the Loan Agreement.

Entry into a Material Definitive Agreement

On July 9, 2024, bluebird bio, Inc. (the "Company") reported to have entered into an amendment (the "Second Amendment") to its Loan and Security Agreement (the "LSA"), dated as of March 15, 2024, as amended on April 30, 2024, by and among the Company, the several banks and other financial institutions or entities party thereto, as lenders (collectively, the "Lenders"), and Hercules Capital, Inc., as administrative agent and collateral agent (the "Agent") (Filing, 8-K, bluebird bio, JUL 9, 2024, View Source [SID1234644788]).

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Pursuant to the Second Amendment, the Company, the Agent and the Lenders agreed to, among other things: (i) extension of the period during which the Company will provide revised monthly financial reporting metrics to the Lenders; and (ii) extension of the deadlines by which the Company must provide to the Lenders financial statements for the year ended December 31, 2023 and for each of the quarters ended March 31, 2024 and June 30, 2024. Further, the Second Amendment provides that the Company’s late delivery and filing of its Form 10-K for the year ended December 31, 2023 and Forms 10-Q for the quarters ended March 31, 2024 and June 30, 2024 shall not be deemed a violation of the Company’s covenant to maintain compliance with applicable law, so long as such documents are filed by the extended deadlines.

The foregoing description of the Second Amendment does not purport to be complete and is qualified in its entirety by the full text of the Second Amendment, a copy of which is filed as Exhibit 10.1 to this Current Report on Form 8-K and incorporated herein by reference.