On September 12, 2024 Vironexis Biotherapeutics, focused on transforming the future of cancer treatment by pioneering AAV-delivered T-cell immunotherapy, reported the company launched from stealth, unveiling its TransJoin AAV Gene Therapy Platform and a pipeline of more than ten product candidates for blood-based cancers, solid tumor metastasis prevention, and a cancer vaccine (Press release, Vironexis Biotherapeutics, SEP 12, 2024, View Source [SID1234646797]). Vironexis’s $26 million seed financing was led by Drive Capital and Future Ventures, with participation from Moonshots Capital and Capital Factory. The company has received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) for VNX-101, its first gene therapy product candidate, for the treatment of CD19+ acute lymphoblastic leukemia. Vironexis anticipates initiating patient enrollment of a Phase 1/2 trial of VNX-101 in the fourth quarter of 2024, which will mark the first-ever clinical trial of an AAV-delivered cancer immunotherapy. VNX-101 has received both Fast Track Designation and Rare Pediatric Disease Designation from the FDA.

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"We’re excited to launch Vironexis from stealth and reveal our noteworthy progress advancing AAV-delivered T-cell immunotherapy," said Samit Varma, co-founder and CEO of Vironexis. "Our novel technology builds on the power of T-cell immunotherapy while overcoming key shortcomings and challenges of existing approaches such as CAR-T and bispecific antibodies. We believe we have the opportunity to dramatically improve upon the safety, efficacy and durability of these drug classes, while streamlining manufacturing and significantly lessening the burden of treatment for patients. Our focus on execution has yielded an expansive pipeline and a clinic-ready lead program in just three years. We’re working as quickly as possible to transform the future of cancer treatments for patients."

TransJoin enables a patient’s body to express an engineered transgene that redirects T cells throughout the body to tumor cells. The proprietary technology requires only a single dose and provides a bridge that joins together T cells and tumor cells to promote long-term, continuous T cell-mediated tumor killing. The foundational technology for TransJoin was licensed from Nationwide Children’s Hospital. In 2022, research led by Timothy Cripe, M.D., Ph.D., Chief of the Division of Pediatric Hematology/Oncology/Bone and Marrow Transplant, describing the TransJoin technology was published in Science Advances [link here to paper]. Dr. Cripe is one of Vironexis’s co-founders, along with Mr. Varma, a seasoned biotechnology and gene therapy company entrepreneur, and Brian Kaspar, Ph.D., a notable gene therapy scientist-entrepreneur who founded AveXis, Inc. (acquired by Novartis) and pioneered the AAV gene therapy ZOLGENSMA.

"AAV is a proven delivery technology with multiple approvals since 2017. Recognizing the pivotal impact of AAV delivery for the treatment of rare diseases, we believed its unique ability to enable long-term, continuous expression of a therapeutic protein could be the missing link to overcome the myriad challenges associated with first-generation T-cell immunotherapies like CAR-T. We subsequently demonstrated the potential of this approach in the preclinical setting," said Dr. Cripe. "It’s thrilling to now be on the cusp of seeing how this technology translates in the clinical setting. Our ultimate goal is to help a vastly broader population of patients realize the tremendous benefits of T-cell immunotherapy."

Vironexis is in the process of obtaining pre-IND input from the FDA for its second program, VNX-202, as a treatment for the prevention of metastatic HER2+ cancer (including breast cancer and other tumor types), and plans to start dosing patients in a Phase 1/2 clinical trial in 2025. The company’s other current product candidates include treatments for BCMA/GPRC5D+ multiple myeloma, CD19/20+ B-cell lymphoma; treatments to prevent metastases in GD2+ neuroblastoma, HER2+ gastric cancer, PSMA+ and MSLN+ pancreatic cancer, B7H3+ ostersarcoma and GP350+ nasopharyngeal cancer; a cancer vaccine for GP350+ nasopharyngeal cancer; and a treatment for CD19+ systemic lupus erythematosus, which Vironexis plans to partner for further development.

Molly Bonakdarpour, Partner at Drive Capital commented, "As a portfolio investment, Vironexis offers an ideal blend of groundbreaking technology, impressive preclinical data, the potential for vast patient impact, and a founding team with deep, relevant expertise and a proven track record of company formation, strategic thinking, and successful execution. Vironexis’s notable productivity in a very short timeframe has been remarkable. We look forward to its upcoming transition to a clinical-stage company."

Steve Jurvetson, Co-Founder of Future Ventures, added, "We were drawn not only to the novelty of the TransJoin technology but also to its broad applicability, spanning treatments for blood-based cancers, solid tumor metastasis prevention, and cancer vaccines, as well as immune disorders. The versatility of this platform is truly standout, and the Vironexis team’s progress in rapidly building a pipeline that explores the expanse of these opportunities is extraordinary."

How TransJoin Works
Vironexis’s TransJoin technology is designed to enable the expression of a secreted T cell engager that binds the tumor cell on one side (changeable depending on the product candidate indication target) and T cells via CD3 on the other side. CD3 is a protein that helps promote T-cell recognition of and activation against cancer cells. Following a single, one-time intravenous infusion, the TransJoin technology instructs the liver to continuously secrete the bispecific protein into the bloodstream to redirect T cells to tumor cells. TransJoin provides a bridge that joins T cells and tumor cells, resulting in long-term, consistent serum levels of the therapy and, thus, long-term, consistent T-cell-mediated tumor cell killing. TransJoin’s extremely low-dose AAV delivery minimizes toxicity and adverse events.

On September 12, 2024 Oncopeptides AB, a biotech company focused on difficult-to-treat cancers, reported it has signed an exclusive license and supply agreement with SCBIO Inc., a Korean pharmaceutical company for the commercialization of Oncopeptides’ flagship drug Pepaxti in South Korea (Press release, Oncopeptides, SEP 12, 2024, View Source [SID1234646773]).

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Oncopeptides estimates the total potential deal value to be SEK 150-300 million (USD 15-30 million) until 2032 consisting of a fixed share of all sales of Pepaxti in the country, an upfront payment and several one-time payments after reaching certain milestones including the submission of a marketing authorization application and first commercial sales of Pepaxti. Should longer market exclusivity be granted, which is the ambition of the parties, market potential is larger. Based on the high unmet need of Pepaxti, Oncopeptides and SCBIO are aiming for an accelerated regulatory approval path with first sales potentially in 2026.

"Based on the strong interest among physicians, the high unmet medical need and the expanding market opportunity with a growing elderly population we believe that South Korea is an ideal steppingstone into our communicated endeavor to expand sales of Pepaxti outside EU", said Sofia Heigis, CEO of Oncopeptides. "In SCBIO, we have found a solid partner providing a good network within multiple myeloma."

Founded in 2021 and headquartered in Daejon, South Korea, SCBIO is a pharmaceutical company specialized within immune-oncology, focusing on improving wellbeing to all patients in need by smart and timely solutions for R&D, product selection, and launch strategies.

South Korea, a country with a population of just over 50 million, with both an aging population and a high average lifespan, has been identified as a good fit for Pepaxti as a maintained quality of life is particularly beneficial for elderly multiple myeloma patients. South Korea was also included in the development program of the drug and experts in the country have clinical experience from using Pepaxti.

This year, Oncopeptides has announced partnership agreements for the sale of Pepaxti on a so called named-patient basis in the Middle East and North Africa, Sub-Saharan Africa and Eurasia. The agreement with SCBIO marks the company´s first licensing agreement, where SCBIO will support Oncopeptides through multiple steps in the value chain, from regulatory approval to commercial sales. Oncopeptides continues to explore similar agreements for other markets including China and Japan.

For more information, please visit oncopeptides.com where questions and answers for investors also will be published.

On September 12, 2024 Turbine, the biological simulation company using AI to build a digital lab for predictive computational models of human cells and tissue, reported that Ono Pharmaceutical Co., Ltd. (Osaka, Japan, "Ono") has selected multiple targets identified using Turbine’s in silico Simulated Cell platform for further development (Press release, Turbine, SEP 12, 2024, View Source [SID1234646555]). Turbine will now lead in vitro validation of the identified targets in one of Ono’s priority cancer biology domains, in consultation with Ono.

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"Achieving this milestone so quickly speaks to the speed and power of our digital laboratory platform, which models foundational protein signaling logic to target disease and position therapeutic assets for clinical success," said Szabolcs Nagy, Turbine’s co-founder and Chief Executive Officer. "All within just a year, we built, trained, and tested the optimal avatars to identify targets within Ono’s priority domain, screened for all relevant perturbations, then filtered and translated millions of in silico experiments to find key hypotheses ready for validation in our wet lab. Our insights included causal factors for each target’s mechanism of action, which should simplify and speed up validation. We’re looking forward to working with our colleagues at Ono to advance this important work."

Under the terms of the collaboration, which was announced in October 2023, Turbine will receive a milestone payment for completing this phase of the research program. As Turbine leads in vitro validation studies of the selected targets in its state-of-the-art wet laboratory facilities, it will consult with Ono’s experts on plans and expected outcomes. Turbine will be eligible for additional payments related to the validation process, as well as for progress of drug development and commercialization by Ono.

"Ono is committed to innovating transformative therapies that improve outcomes for cancer patients, and identifying novel targets with robust scientific support is essential for achieving this goal," said Seishi Katsumata, Corporate Officer/Executive Director, Discovery & Research of Ono. "Turbine has provided multiple targets that have first-in-class potential, along with the biology-driven insights necessary to rapidly validate this potential. We’re excited to move these targets forward and expect that the collaboration will consequently lead to providing a new therapeutic option to cancer patients as soon as possible."

On September 12, 2024 ITM Isotope Technologies Munich SE (ITM), a leading radiopharmaceutical biotech company and Debiopharm (www.debiopharm.com), a Swiss-based, global biopharmaceutical company, aiming to establish tomorrow’s standard-of-care to cure cancer and infectious diseases, reported that the companies entered an agreement under which ITM gains the exclusive global license for the clinical and commercial development of the peptide-based, theranostic pair ITM-91/ITM-94D, formerly Debio 0228/0328, targeting the Carbonic Anhydrase IX (CA IX) surface protein (Press release, ITM Isotopen Technologien Munchen, SEP 12, 2024, View Source [SID1234646554]). CA IX plays a key role in the tumor microenvironment, promoting tumor growth, survival, invasion and metastasis. ITM-91 (Debio 0228) ([177Lu]Lu-DPI-4452) is a Lutetium-177-labeled radioligand therapeutic compound and ITM-94D (Debio 0328) ([68Ga]Ga-DPI-4452) is a Gallium-68-labeled imaging agent. The theranostic pair is being investigated in the phase 1/2 GaLuCi(NCT05706129) clinical trial in patients living with locally advanced ccRCC, PDAC and CRC. Further terms and financial details have not been disclosed.

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"Curing patients is our mission. Through this agreement with ITM, we hope to bring breakthrough solutions to patients living with hard-to-treat cancer types. We’re extremely pleased that the trial data generated to-date for our imaging radiotracer have provided high-quality images with high tumor uptake and excellent tumor-to-background ratios. Debio 0328’s outstanding potential as a stand-alone imaging agent has also boosted our confidence for the upcoming evaluation of Debio 0228, the therapeutic agent. As development continues, we are grateful to be able to rely on ITM’s radiopharmaceutical expertise to advance research for patients," said Bertrand Ducrey, CEO of Debiopharm.

Debiopharm is a drug development expert with an evolving radioligand therapy pipeline and strong pre-clinical and clinical competence in this field. Their unique business model allows Debiopharm to continuously bridge the gap between innovative discoveries and leading pharmaceutical companies for commercialization. As the globally leading manufacturer of n.c.a. Lutetium-177 and with a broad pipeline of radiopharmaceutical diagnostic and therapeutic candidates, ITM will use its production capabilities and clinical expertise to advance this theranostic pair.

"Patients with advanced renal cancer often have a long and difficult journey, with recurrence after surgery not infrequent and limited treatment options following immune-oncology or tyrosine kinase inhibitor therapy. I have high hopes for trials like GaLuCi will finally shift the odds defining both a new PET/CT scan and targeted treatment option," said Prof. Michael Hofman, Peter MacCallum Cancer of Melbourne and investigator of the Phase 1/2 GaLuCi study.

"Theranostic approaches are a very exciting treatment modality for patients with hard-to-treat malignancies due to their potential to target specific surface proteins, often regardless of tumor origin. This technique has been shown to improve outcomes for patients with advanced-staged prostate cancer and we are hoping to bring it to the forefront of treatment for kidney cancer," added Darren R. Feldman, MD, Medical Oncologist, Genitourinary Oncology Service at Memorial Sloan Kettering Cancer Center and investigator of the Phase 1/2 GaLuCi study.

About ITM-91/ ITM-94D (Debio 0228/0328)

ITM-91/ ITM-94D (Debio 0228/0328) is an investigational theranostic pair originally discovered by 3B Pharmaceuticals GmbH and now exclusively licensed to ITM. ITM-94D (Debio 0328) ([68Ga]Ga-DPI-4452) is a PET imaging agent that may be used independently and is designed to identify patients whose cancers overexpress CA IX. Once identified, these patients may be treated with the lutetium-labelled radioligand, ITM-91 (Debio 0228) ([177Lu]Lu-DPI-4452), which is designed to deliver targeted radiation to the tumor with the aim to destroy it from the inside.

About the GaLuCi trial

The GaLuCi trial is the first-in-human, multicenter, non-randomized phase 1/2 clinical trial assessing safety and tolerability, imaging characteristics, and the efficacy of the theranostic pair ITM-91/ ITM-94D (Debio 0228/0328) in patients with unresectable, locally advanced, or metastatic solid tumors. This theranostic trial is being carried out in three stages. The ongoing Part A is evaluating the safety and performance of the imaging agent in detecting CA IX-expressing solid tumors. Part B will assess escalating doses of the therapeutic agent, ITM-91 (Debio 0228) in patients, whose tumors show high uptake of imaging tracer. Finally, based on the recommended dose from Part B, Part C of the study will further assess the safety and preliminary efficacy of ITM-91 (Debio 0228) in ccRCC, PDAC and CRC.

On September 12, 2024 Molecular Targeting Technologies, Inc. (MTTI) reported that it will update findings on its proprietary topical near-infrared fluorescent dye, CypH-11 (Cmetglo), at the World Molecular Imaging Conference (WMIC) meeting in Montreal from September 9-13 and at the Peritoneal Surface Oncology Group International (PSOGI) meeting in Lyon from September 26-28, 2024 (Press release, Molecular Targeting Technologies, SEP 12, 2024, View Source [SID1234646553]).

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This convenient fast-acting technology* shows promise as an effective real-time tool for oncologists in fluorescence-guided surgery (FGS). This will allow clinicians to see small tumor residue and achieve a more complete resection of cancer in the abdominal cavity. We expect that Cmetglo may improve patient outcomes by avoiding unnecessary damage to normal tissue and increase progression-free survival of patients with peritoneal surface malignancies (PM).

Dr. Johnny Ong, MD, Associate Professor, Department of Sarcoma, Peritoneal and Rare Tumors, National Cancer Centre Singapore** commented: "One of the limitations of cytoreductive surgery (CRS) is the difficulty in distinguishing tumors from normal and scar tissues. Here, we performed ex vivo validation of patient tissues to evaluate the clinical utility of Cmetglo in detecting PM via topical administration. Preliminary analysis suggests that the best clinical utility of Cmetglo could be in patients with colorectal malignancy, with the possibility of expanding its use to other histological subtypes."

Dr. Brian D Gray, SVP Research and Development noted: "MTTI’s Cmetglo makes the invisible visible. Tumor margins and metastases glow under near-infrared light. It can be a valuable addition to the surgeon’s’ armamentarium to achieve maximal cytoreduction during FGS."

Dr. Seung Koo Lee, Assistant Professor of Cell Biology Research, in Radiology at Weill Cornell Medicine***, commented: "CypH-11 is a sprayable pH-responsive fluorogenic probe that exhibits minimal fluorescence at neutral pH; however, it fluoresces brightly in an acidic environment which is a universal signature of cancer cell proliferation. Its capability of detecting small-sized ovarian tumors was further demonstrated by the spray of CypH-11 in a disseminated high-grade serous ovarian cancer (HGSOC) model."

Chris Pak, President & CEO of MTTI commented: "This groundbreaking molecule builds on MTTI’s innovative legacy in targeted diagnostics and therapeutics. We’re pursuing its use in colorectal, ovarian, and brain cancers, adding value to patients, surgeons, and other stakeholders."