On January 13, 2025 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 KJ-C2219, an allogeneic CAR T-cell therapy targeting CD19/CD20, has administered the first dose to a patient in an investigator-initiated trial (IIT) (Press release, Carsgen Therapeutics, JAN 13, 2025, View Source [SID1234649685]).

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KJ-C2219 is developed based on CARsgen’s THANK-u Plus platform and is designed for the treatment of hematologic malignancies and autoimmune diseases. An investigator-initiated trial is ongoing in China to evaluate KJ-C2219 for the treatment of relapsed/refractory B-cell non-Hodgkin lymphoma (R/R B-NHL).

About THANK-u Plus

CARsgen has developed the THANK-u Plus platform as an enhanced version of its proprietary THANK-uCAR allogeneic CAR-T technology to address the potential impact of NKG2A expression levels on therapeutic efficacy. THANK-u Plus demonstrates sustained expansion regardless of varying NKG2A expression levels on NK cells and exhibits significantly improved expansion compared to THANK-uCAR. Preclinical studies show that THANK-u Plus delivers superior antitumor efficacy in the presence of NK cells compared to THANK-uCAR. Allogeneic BCMA or dual-targeting CD19/CD20 CAR-T cells developed using this platform exhibit robust antitumor activity in the presence of NK cells, indicating that THANK-u Plus has broad potential for developing diverse allogeneic CAR-T therapies.

On January 13, 2025 SciTech Development, a clinical-stage pharmaceutical company specializing in oncology, reported the initiation of the next stage of its clinical trial for their lead drug candidate, ST-001 nanoFenretinide (ST-001) (Press release, SciTech Development, JAN 13, 2025, View Source [SID1234649684]). This milestone follows the successful completion of the accelerated safety and dose-escalation stage, where ST-001 demonstrated exceptional safety, tolerability, and pharmacokinetic performance.

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Advancing the Phase 1a Trial with Clear Clinical Objectives

The continuation of the ST-001 clinical trial aims to achieve several critical clinical objectives:

Confirm that ST-001 achieves previously reported clinical response rates.
Determine the pharmacological profile of intravenous fenretinide delivered using SciTech’s novel nanoparticle delivery system, designed for drugs with poor water solubility.
Determine the recommended treatment dose (RTD) to evaluate disease activity and response rates in the follow-on Phase 1b trial and the planned trial in Small Cell Lung Cancer.
"The success of our accelerated Phase 1a trial marks a pivotal milestone, driving ST-001 into the next critical stage of development," said Earle Holsapple, CEO of SciTech Development. "We are thrilled about the potential of ST-001 nanoFenretinide and its promise to transform treatment outcomes for patients battling cancers such as T-cell Non-Hodgkin Lymphoma."

About ST-001 nanoFenretinide

ST-001 nanoFenretinide is a patented nanoparticle IV formulation that combines the drug fenretinide with biocompatible phospholipids. This groundbreaking new investigational drug overcomes bioavailability challenges, enhances therapeutic efficacy, and minimizes toxicity in cancer patients. Powered by SciTech’s advanced delivery platform, ST-001 enables rapid delivery of effective doses of fenretinide to cancer cells, marking a breakthrough in hard-to-treat cancers.

Encouraging Early Results in Phase 1a Accelerated Trial

During the accelerated stage of the trial, ST-001 achieved significant milestones:

Patient Enrollment is Complete for the accelerated stage of the clinical trial marking a significant accomplishment for patients with this rare disease.
Therapeutic Responses Observed: Patients demonstrated partial responses and stable disease, marking an important step for trial advancement.
Excellent Tolerability for ST-001 across all dose levels to date.
Outstanding Pharmacokinetics: The nanoparticle delivery platform overcame longstanding bioavailability challenges, ensuring efficient delivery of fenretinide to tissues.
Future Direction for ST-001 nanoFenretinide

Building on the success of the accelerated Phase 1a stage, the trial begins detailed evaluation of clinical activity and response rates in T-cell lymphomas. The trial is actively underway at nine renowned medical centers across the country. Click here to learn more about the locations and patient trial criteria. Additionally, a separate trial targeting Small Cell Lung Cancer utilizing ST-001 is scheduled to begin in Q2 2025.

On January 13, 2025 Rappta Therapeutics ("Rappta"), focused on developing first-in-class anti-cancer drugs activating protein phosphatase 2A (PP2A), reported an exclusive global license agreement with SpringWorks Therapeutics ("SpringWorks") for RPT04402, a first-in-class molecular glue of specific Protein Phosphatase 2A (PP2A) complexes (Press release, Rappta Therapeutics, JAN 13, 2025, View Source [SID1234649683]).

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Rappta’s PP2A-reactivating technologies developed using its proprietary high resolution structural data have the potential to create a new class of anti-cancer drugs for treating a broad range of human cancers. PP2A is a critical enzyme in regulating protein de-phosphorylation and its reactivation is fundamental for tumor suppression, but it has been historically hard to target.

PP2A mutations are oncogenic drivers in molecularly defined subsets of uterine cancer and represent a targetable subset of patients with a high unmet clinical need. In pre-clinical models of PP2A mutant uterine cancer, RPT0402 achieved rapid, deep and durable tumor regressions at as monotherapy.

Under the exclusive license agreement, SpringWorks Therapeutics will be responsible for global development and commercialization of RPT04402. SpringWorks has paid Rappta $13 million upfront, and Rappta is also eligible to receive further clinical, regulatory and commercial milestone payments, and tiered single-digit royalties on net sales. SpringWorks expects to file an Investigational New Drug (IND) application for RPT04402 by the end of 2025.

Sunjeet Sawhney, Chief Executive Officer of Rappta Therapeutics, commented: "Rappta is the only company to have successfully targeted PP2A, a notoriously difficult and undruggable target. Data we generated demonstrated the potential of RPT04402 for treating large, underserved patient populations. SpringWorks, a leader in the targeted oncology space, has the expertise and knowledge to accelerate the further development of our first in class asset. I would like to thank our team and investors who have supported our journey and we look forward to following the progress made in this area"

Goutham Narla, Chief Scientific Officer of Rappta Therapeutics and Professor of Internal Medicine and Human Genetics at the University of Michigan, said: "Our team at Rappta Therapeutics has been able to leverage our proprietary structural data to develop a first-in-class series of molecular glues to the previously undruggable tumor suppressor PP2A. We are excited to be able to work with SpringWorks to potentially translate this approach to the clinic with the hope that this approach will have broad applications for the treatment of human cancers."

Rappta was founded by Goutham Narla & Mikko Mannerkoski, back in 2019 with funding from Novartis Venture Fund ("NVF"), Novo Holdings, Advent Life Sciences and a family office in Series A financing alongside other non-dilutive funding from Business Finland.

On January 13, 2025 Simcere Zaiming, a subsidiary of Simcere Pharmaceutical Group Ltd (HKEX: 2096) and AbbVie Inc. (NYSE: ABBV) reported an option to license agreement to develop SIM0500, an investigational new drug candidate (Press release, Jiangsu Simcere Pharmaceutical Company, JAN 13, 2025, View Source [SID1234649682]). SIM0500 is currently in Phase 1 clinical trials in patients with relapsed or refractory multiple myeloma (MM), in both China and the U.S.

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SIM0500 is a humanized trispecific antibody that targets GPRC5D, BCMA, and CD3, developed independently by Simcere Zaiming using their T-cell engager polyspecific antibody technology platform. This molecule features a low affinity/high target-activating CD3 engaging arm and binding sites for the two tumor antigens: G-Protein-coupled receptor class 5 member D (GPRC5D) and B-cell maturation antigen (BCMA). SIM0500 has shown strong T cell cytotoxicity against multiple myeloma (MM) cells by leveraging a combination of various antitumor effects.

"As a leader in hematologic malignancies, AbbVie is committed to advancing innovative treatments for complex cancers like multiple myeloma through our relentless R&D efforts and collaborations," said Mariana Cota Stirner, M.D., Ph.D., vice president, therapeutic area head for hematology, AbbVie. "We look forward to partnering with Simcere Zaiming, to advance this novel trispecific antibody, which has the potential to help address significant unmet medical needs for people living with multiple myeloma"

"SIM0500 is developed via Simcere Zaiming’s proprietary T-cell engager platform," said Renhong Tang, PhD, CEO of Simcere Zaiming. "We are excited to partner with AbbVie on this novel drug candidate and look forward to working together to advance the clinical development of SIM0500. "

Simcere Zaiming will receive an upfront payment from AbbVie and is eligible to receive option fees and milestone payments of up to $1.055B, as well as tiered royalties on net sales outside of the Greater China territory. AbbVie is eligible to receive tiered royalties on net sales in the Greater China territory

On January 13, 2025 Rise Therapeutics, a biotechnology company engaged in developing novel oral Immunotherapeutics, reported that the U.S. Food and Drug Administration (FDA) has accepted its investigational new drug (IND) application to proceed with a cancer Phase 1 clinical trial for its program candidate, R-5780 (Press release, Rise Therapeutics, JAN 13, 2025, View Source [SID1234649681]). This is Rise Therapeutics’ fourth clinical program to enter patient clinical testing. For separate products, other clinical studies are ongoing for the treatment of ulcerative colitis, rheumatoid arthritis, and type 1 diabetes.

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R-5780 is an orally delivered immune oncology drug candidate being developed for the treatment of cancer. R-5780 has demonstrated a pronounced ability to engage immune pathways that enhance the effectiveness immune checkpoint inhibitor therapy. This drug candidate builds upon the growing understanding of how certain gut-regulated immune pathways can help promote efficacy of immune oncology drugs. R-5780 is a precision-directed synthetic biology medicine that engages selective immune pathways that can support a robust anti-tumor T cell response. This revolutionary immune oncology drug candidate is one of the first of its kind in the context of mechanism of action and potential for success in the context of cancer treatment.

"The FDA’s clearance for R-5780 is a testament to the innovation and dedication of our team at Rise Therapeutics driving novel immunotherapies forward into human proof-of-concept" states Christian Furlan Freguia, Senior Vice President of Research at Rise Therapeutics. "R-5780 represents a pioneering approach that leverages the power of gut-regulated immune pathways to enhance the effectiveness of immune checkpoint inhibitors… We believe R-5780 has the potential to expand responses in patients that become refractory to immune checkpoint inhibitors and increase responsiveness in tumors initially unresponsive to these medications".

The Phase I clinical trial (NCT06398418) will be a multi-dose clinical trial assessing the safety, drug exposure, and clinical activity of R-5780 in patients with cancer. The study will enroll up to 33 participants.