On December 4, 2023 Cancer Research UK, one of the world’s largest charitable funders of cancer research, its innovation unit, Cancer Research Horizons, and Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported a collaboration to explore the sharing of technologies, data, and insights to advance the development and precision of cancer detection and treatment (Press release, Cancer Research UK, DEC 4, 2023, View Source [SID1234638145]).

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The agreement will enable the parties to discuss opportunities for collaboration to support the charity’s research and clinical development activities, focusing primarily on its clinical trials run by its Centre for Drug Development. This includes the DETERMINE trial – the first UK national precision medicine trial in rare cancers, and other Cancer Research UK and Cancer Research Horizons research collaborations.

Additional potential collaboration activities may include sharing data and samples from the Cancer Research UK-funded research network to further develop and validate Guardant’s technologies, as well as explore other projects to accelerate the discovery and development of cancer drugs and diagnostics.

"Partnering with Cancer Research UK grants us with a unique opportunity to further validate Guardant’s unique technologies, including our proprietary InfinityTM platform, while providing the support needed for those with rare cancers and others who could greatly benefit from precision oncology," said Helmy Eltoukhy, co-CEO of Guardant Health. "We’re looking forward to learning more throughout this partnership to provide an even higher-level of quality in our tools."

Through this partnership, Guardant Health will further expand its partnership with one of the world’s leading cancer charities, Cancer Research UK, combining leading edge technology in biotechnology with an expansive reach of more than 200 clinical trials and studies in over a million people in the UK.

"Partnering with Guardant Health brings exciting potential for us to accelerate our work by applying best-in-class data-driven insights and technologies to our detection and treatment studies. Our Centre for Drug Development has an excellent track record in bringing novel therapeutics to first-in-human trials and we look forward to seeing the positive impact this collaboration will have on the Centre’s work and beyond," said Tony Hickson, Chief Business Officer for Cancer Research UK and Cancer Research Horizons.

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.