On December 4, 2023 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, reported that the U.S. Food and Drug Administration (FDA) has granted Investigational New Drug (IND) clearance for CT071, an autologous CAR T-cell therapy candidate targeting G protein-coupled receptor class C group 5 member D (GPRC5D), for the treatment of patients with relapsed/refractory multiple myeloma (MM) or relapsed/refractory primary plasma cell leukemia (PCL) (Press release, Carsgen Therapeutics, DEC 4, 2023, View Source [SID1234638144]).

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GPRC5D is emerging as an important target in the landscape of MM, a common but incurable hematologic malignancy characterized by the uncontrolled proliferation of plasma cells. The notable overexpression of GPRC5D on the surface of malignant plasma cells, coupled with its limited presence on normal tissues, makes GPRC5D an ideal candidate for the treatment of MM and PCL. CT071 incorporates a fully-human single-chain variable fragment (scFV) developed by CARsgen, specifically designed to target GPRC5D.

CT071 is manufactured with CARsgen’s proprietary CARcelerateTM platform, which shortens the manufacturing time to less than 2 days and therefore yields younger, healthier, and possibly more potent CAR T cells compared to conventional manufacturing. The improved manufacturing efficiency also enhances the supply capacity, reduces the manufacturing costs, and expedites the availability of the product to the patients.

An investigator-initiated trial (IIT) is already under way in China to assess the safety and efficacy of CT071 in treating relapsed/refractory MM or PCL (NCT05838131). Preliminary clinical data from the IIT shows an acceptable safety profile with preliminary efficacy.

About CT071

CT071 is a CAR T-cell therapy candidate developed utilizing proprietary CARcelerateTM platform of CARsgen targeting GPRC5D for the treatment of relapsed/refractory MM or relapsed/refractory PCL. An IIT (NCT05838131) is ongoing in China to evaluate the safety and efficacy of CT071 for the treatment of relapsed/refractory multiple myeloma or plasma cell leukemia.

About CARcelerateTM

CARcelerateTM is a proprietary platform CARsgen developed that can shorten the manufacturing time for the CAR T cells to less than 2 days, as compared to the conventional CAR T manufacturing process. The CARcelerateTM platform produces CAR T cells that are younger, healthier, are more likely to remain in a ‘naïve’ state and less likely to be exhausted; as such, these CAR T cells from the CARcelerateTM platform are expected to exhibit more potent tumor killing activity.

On December 4, 2023 Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) reported that the first patient has been dosed in a named patient (early access) program in the Netherlands for its investigational positron emission tomography (PET) imaging agent TLX250-CDx (89Zr-DFO-girentuximab) in clear cell renal cell carcinoma (ccRCC), the most common and aggressive form of kidney cancer (Press release, Telix Pharmaceuticals, DEC 4, 2023, View Source [SID1234638143]).

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Through this named patient program in the Netherlands (‘Leveren op Artsenverklaring’), physicians may seek individual access to TLX250-CDx for use in PET characterisation of renal masses as ccRCC. The first patient was dosed at Radboud University Medical Centre in Nijmegen, Netherlands.

The program follows the completion of Telix’s successful global Phase III ZIRCON study (Zirconium in Renal Cancer Oncology, ClinicalTrials.gov Identifier: NCT03849118), which reported positive results in November 2022, meeting all co-primary and secondary endpoints.1

Professor Dr. Peter Mulders from Radboud University Medical Centre (Netherlands), a Principal Investigator on Telix’s completed ZIRCON trial commented, "Continued access to this investigational imaging agent is critically important. The detection of ccRCC in the early stages of disease can often be challenging, and reliant on invasive biopsy and nephrectomy (kidney removal). It is therefore extremely good news that TLX250-CDx, which offers a non-invasive option – or ‘molecular biopsy’ – is now available in the Netherlands on a named patient basis."

Frédéric Fantino, Telix Medical Director for the Europe, Middle East and Africa (EMEA) region, added, "The first patient dosed in this European named patient program is a significant step for Telix to support continued unmet patient need and educate key opinion leaders on the potential of TLX250-CDx to change standard of care in the diagnosis and management of ccRCC. The ZIRCON study has shown this imaging agent to be highly sensitive and specific in the detection of ccRCC, where existing imaging techniques are sometimes inconclusive."

In the Netherlands, the use of a medicinal product in an individual patient prior to marketing authorisation and outside the context of a clinical trial is permitted in exceptional circumstances. Individual requests must be initiated by the treating physician and are evaluated by the Dutch Health Inspectorate (IGJ).

Telix is progressing towards a Biologics License Application (BLA) submission for TLX250-CDx with the United States Food and Drug Administration (FDA) and other equivalent applications with regulatory agencies in key commercial jurisdictions.

Physicians in Europe who may have eligible patients can email [email protected] for further information about TLX250-CDx named patient access.

For more information about ongoing clinical trials of TLX250-CDx, please visit View Source

Telix’s Policy on Offering Compassionate Use to Investigational Medicines can be downloaded at the following link.

On December 4, 2023 K36 Therapeutics, Inc. ("K36"), a clinical-stage biotech company focused on developing its first-in-class MMSET inhibitor KTX-1001 for t(4;14) multiple myeloma, reported that its abstract evaluating KTX-1001 in relapsed and refractory multiple myeloma has been accepted for presentation at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, which will be held December 9-12, 2023, in San Diego, California, and online (Press release, K36 Therapeutics, DEC 4, 2023, View Source [SID1234638142]). Dr. Jesús G. Berdeja, the Director of Multiple Myeloma Research at Tennessee Oncology, will deliver the presentation as part of the Trials in Progress session.

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"I am delighted to be presenting the trial progress of KTX-1001 in high-risk subsets like translocation t(4;14)," stated Dr. Berdeja. "There still remains an unmet need for new personalized oral therapies to treat multiple myeloma and novel precision therapeutics like KTX-1001 to address the challenges seen in high-risk patients with translocation t(4;14)."

"We are seeing significant interest and momentum in our Phase 1 trial, and I would like to thank the patients, investigators and their staff for their commitment to generate important data from our trial," said Terry Connolly, Ph.D., Chief Executive Officer of K36. "KTX-1001 has demonstrated encouraging preclinical activity and a favorable tolerability profile establishing the potential to create a breakthrough therapy for multiple myeloma patients who have exhausted available standard of care treatments."

Presentation Details:

Title: Trial in Progress: A Phase 1 Study of KTX-1001, an Oral, First-in-Class, Selective MMSET Inhibitor in Patients with Relapsed and Refractory Multiple Myeloma

Poster Number: 3391
Session Name: 653. Multiple Myeloma: Prospective Therapeutic Trials: Poster II
Date: Sunday, December 10, 2023
Time: 6:00 PM – 8:00 PM PT
Location: San Diego Convention Center, Halls G-H

Full abstracts can be found at the ASH (Free ASH Whitepaper) Annual Meeting website at www.Hematology.org.

About KTX-1001
KTX-1001 is a novel, first-in-class, potent, and selective methyltransferase inhibitor of the catalytic activity of lysine H3K36. It is an orally administered small molecule being developed initially for the treatment of relapsed and refractory multiple myeloma, with a focus on patients with the t(4;14) translocation. This inhibitor offers a promising avenue for addressing this challenging high risk patient population.

About the KTX-1001 Phase 1 Clinical Trial
The Phase 1 clinical trial is a single-arm, open-label study in subjects with relapsed and refractory multiple myeloma. It is a multi-part clinical trial with dose escalation followed by an expansion cohort in patients with the genetic translocation t(4;14) to evaluate the safety, tolerability, and preliminary efficacy of different doses of KTX-1001. For more information and participating centers visit NCT05651932.

On December 4, 2023 Abbisko Therapeutics reported that it has entered into a licensing agreement with Merck, a leading science and technology company headquartered in Darmstadt, Germany (Press release, Abbisko Therapeutics, DEC 4, 2023, View Source [SID1234638141]). Under the terms of the agreement, Merck will be granted an exclusive license to commercialize products comprising or containing pimicotinib (ABSK021) for all indications in Chinese mainland, Hong Kong, Macau and Taiwan (the "Licensed Territory"), and Abbisko Therapeutics will retain the exclusive rights to develop pimicotinib within the Licensed Territory. Abbisko Therapeutics has also granted Merck an exclusive option for global commercial rights of pimicotinib, subject to the terms and conditions as agreed between the parties (the "Global Commercialization Option"). In addition, Merck has the option to co-develop pimicotinib in additional indications under certain conditions.

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Pursuant to the License Agreement, Abbisko Therapeutics will receive a one-time, non-refundable down payment of US$ 70 million. In the event that Merck exercises the Global Commercialization Option, Merck will pay Abbisko Therapeutics an additional option exercise fee. The aggregate amount of upfront payment, exercising payment, and development and commercialization milestone adds up to US$ 605.5 million, plus a double-digit percentage (%) royalty on actual annual net sales.

Dr. Xu Yao-chang, Chairman of Abbisko Therapeutics, said that "The collaboration with Merck is an important milestone in advancing the global commercialization process of pimicotinib, and provides a new model for the commercialization path of the company’s pipeline in the future. We are pleased to collaborate with a leading multinational pharmaceutical company, jointly accelerating the global approval and commercialization pace of pimicotinib, and striving to bring new treatment options to patients as soon as possible."

"We have the opportunity through our partnership with Abbisko to deliver a first-in-class treatment for a critically underserved patient population in China and potentially beyond," said Andrew Paterson, Chief Marketing Officer for the Healthcare business sector of Merck. "Pimicotinib provides an opportunity to address a significant unmet medical need and for us to expand our commercial footprint in oncology in China, the second largest pharmaceutical market in the world."

About Pimicotinib (ABSK021)

Pimicotinib is a novel, orally available, highly selective, and potent small molecule CSF-1R inhibitor, independently developed by Abbisko Therapeutics. It has been granted the breakthrough therapy designation (BTD) and Priority Medicine (PRIME) designation by China NMPA, U.S.FDA, and EMA for the treatment of TGCT patients that are not amenable to surgery. The study is the first global Phase III clinical trial of TGCT conducted simultaneously in China, the U.S., Canada and Europe.

Upon 1-year follow-up, striking improvement in efficacy has been observed with pimicotinib treatment and results were presented at CTOS in November of 2023, with an ORR of 87.5% (28/32, including 3 CR) in the 50 mg QD cohort. The Phase I dose-escalation trial for pimicotinib has been completed in the United States previously.

There is currently no approved drug available in China for TGCT patients, and only one drug has been approved in the U.S.. However, it is only available through the Risk Evaluation and Mitigation Strategy (REMS) Program which is a restricted procedure due to the potential liver injuries it may cause. There are unmet medical needs of TGCT patients in China, the U.S., and Europe.

Abbisko Therapeutics is also actively exploring the clinical potential of pimicotinib in many types of solid tumors, and has obtained approval from NMPA to conduct a Phase II clinical study in chronic graft-versus-host disease and a Phase II clinical study in advanced pancreatic cancer.

As of the date of this announcement, no highly selective CSF-1R inhibitor has been approved in China.

On December 4, 2023 Immunitas Therapeutics ("Immunitas"), a clinical stage precision immunotherapy company committed to discovering and developing novel, differentiated therapeutics for patients with cancer, reported the appointment of James Wooldridge, M.D., as Chief Medical Officer (CMO) (Press release, Immunitas Therapeutics, DEC 4, 2023, View Source [SID1234638140]). The company also announced it will present new data supporting continued clinical development of its lead program IMT-009 for solid tumors and hematological malignancies at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH 2023).

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In the CMO role, Dr. Wooldridge will leverage his extensive oncology therapeutic development experience to support clinical advancement strategy for Immunitas’ pipeline of highly differentiated, antibody-based immuno-oncology assets, including lead candidate IMT-009. Dr. Wooldridge has served as Acting CMO since June 2023 and has been an advisor to the Company since October 2022.

"We are pleased to announce Jim’s appointment as our Chief Medical Officer as we continue progressing toward our goal of providing groundbreaking therapeutic solutions for patients with cancer," said Amanda Wagner, Chief Executive Officer of Immunitas. "Over the past year advising Immunitas he has provided invaluable expertise, drawing from his extensive breadth of experience in immuno-oncology development to support advancement of our evolving pipeline. We are delighted to have him on board as our CMO as we continue clinical development of IMT-009 and progress our portfolio of highly differentiated immuno-oncology biologics."

Dr. Wooldridge has more than 20 years of experience in clinical oncology research and drug development spanning the biotechnology, pharmaceutical, and academic sectors. He previously served as the Chief Medical Officer at Checkmate Pharmaceuticals leading up to the company’s acquisition by Regeneron. Additionally, he held the role of Chief Medical Officer at Aeglea BioTherapeutics, where he oversaw development of enzyme-based treatments for rare genetic diseases and cancer.

"I am excited to continue working with this team to further advance the Immunitas pipeline. Our lead program, IMT-009, targets the CD161/CLEC2D axis and is currently dosing in the clinic with key data expected in 2024. The talented Immunitas team has built on our unique expertise in this area to progress a second compelling program for tumors that have traditionally not responded to immune-therapy, and the company’s approach to target discovery is deeply rooted in human biology," said Dr. Wooldridge. "I’m particularly excited by Immunitas’ strategy to develop novel therapeutics using a biomarker-driven approach early in development, and I greatly look forward to continuing my work with this team to bring new therapies to patients."

The data to be presented at ASH (Free ASH Whitepaper) 2023 further supports the therapeutic potential of IMT-009 in hematological malignancies.

IMT-009 is a monoclonal, Fc silent, fully human Immunoglobulin G1 (IgG1) antibody that binds to CD161 with high affinity, selectively blocking interactions with its ligand CLEC2D. Previously reported preclinical data confirm that blocking CD161 with IMT-009 restores activation of effector functions of both CD161+ T and NK cells, enhancing cytotoxicity toward target tumor cells. IMT-009 is currently being evaluated in a Phase 1/2a clinical trial for use as monotherapy and combination treatment in both solid tumors and heme malignancies (NCT05565417).

"The new data to be shared at ASH (Free ASH Whitepaper) 2023 adds to the growing body of evidence supporting IMT-009’s potential as a differentiated treatment approach for hematologic malignancies, in addition to the strong data we have generated in solid tumors. Together with data presented at SITC (Free SITC Whitepaper) 2023 that demonstrated proof-of-concept for our second program, a myeloid and B cell modulating anti CLEC2D-Toll-like receptor 9 (TLR9) agonist conjugate, these data provide further compelling support for our approach targeting the CLEC2D/CD161 axis as a novel ligand-receptor pathway for immunotherapeutic intervention," said Amanda Wagner, Chief Executive Officer of Immunitas.

Presentation Details for ASH (Free ASH Whitepaper) 2023:
Title: Anti CD161 antibody IMT-009 is a novel immunotherapeutic agent that effectively blocks the inhibitory CLEC2D/CD161 axis in CLEC2D+ B Cell hematological malignancies reinvigorating T and NK cell function leading to anti-tumor benefit
Abstract Number: 2815
Date/Time: Sunday, December 10, 2023, 6:00 – 8:00 pm PT

About IMT-009
IMT-009 is a fully human, Fc-attenuated IgG1 monoclonal antibody that binds to CD161 and blocks its interaction with its ligand, CLEC2D. Preclinical data confirm that CD161 blockade with IMT-009 results in enhanced anti-tumor activity. IMT-009 is under evaluation in a Phase 1/2a clinical trial for use as a monotherapy and combination treatment for solid tumor and hematological malignancies. The Phase 1 study is designed to evaluate the safety, tolerability, pharmacodynamic biomarkers, and preliminary efficacy of IMT-009 as well as identify the Recommended Phase 2 Dose (RP2D). The trial will then transition into Phase 2 with multiple expansion cohorts to assess the safety and efficacy of IMT-009 alone or in combination with another antineoplastic agent.