On October 17, 2024 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage allogeneic cell therapy and genetic medicines company advancing differentiated non-viral treatments for patients with cancer, autoimmune, and rare diseases, reported the nomination of a new development candidate under its collaboration with Roche (Press release, Poseida Therapeutics, OCT 17, 2024, View Source [SID1234647253]). The nomination triggered a $15 million milestone payment from Roche to Poseida.

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The new candidate is an allogeneic, dual CAR-T therapy targeting known antigens expressed in hematologic malignancies, including multiple myeloma. The large capacity of Poseida’s non-viral transposon-based DNA delivery system enables the insertion of genes for two full length chimeric antigen receptors (CARs) into T stem cell memory cells (TSCM).

"The nomination of a new development candidate builds on our collaboration with Roche and highlights the unique potential of our proprietary non-viral genetic engineering toolkit to create differentiated, TSCM-rich allogeneic CAR-T therapies targeting one or more antigens," said Kristin Yarema, Ph.D., president and chief executive officer of Poseida Therapeutics. "Multiple myeloma is a common and incurable blood cancer with significant room for potent, safe and accessible novel agents to expand use across lines of therapy and sites of care. With compelling preclinical data supporting the target combination of this dual CAR-T, we look forward to advancing this program towards the clinic as a part of the collaboration. We also look forward to providing updates on our CAR-T programs and earlier-stage pipeline at Poseida’s upcoming Cell Therapy R&D Day."

Poseida and Roche now have three programs under their collaboration, which was established in August 2022 to develop allogeneic CAR-T therapies directed to hematologic malignancies. The lead collaboration program, P-BCMA-ALLO1, is an allogeneic CAR-T therapy targeting BCMA that has received Regenerative Medicine Advanced Therapy (RMAT) designation for adult patients with relapsed/refractory multiple myeloma after three or more prior lines of therapies. Poseida is currently enrolling patients in a Phase 1b portion of the clinical trial. The second program, P-CD19CD20-ALLO1, is an allogeneic dual CAR-T candidate in Phase 1 development for B-cell malignancies.

Dr. Yarema added, "Based on P-BCMA-ALLO1’s promising differentiated safety and efficacy results established with the recent interim Phase 1 data presented at the IMS Annual Meeting in September and in collaboration with Roche, we look forward to continued patient enrollment in the Phase 1b trial. The new allogeneic dual CAR-T therapy candidate announced today will leverage the same proprietary Poseida GMP manufacturing platform that was used to create and advance P-BCMA-ALLO1."

Poseida Cell Therapy R&D Day
The Company plans to host its Cell Therapy R&D Day on November 14th, 2024, featuring presentations from management and top scientists. The event will highlight the Company’s progress across its clinical-stage and earlier-stage pipeline of differentiated allogeneic CAR-T therapies in oncology and autoimmune disease.

The virtual event and access to the live webcast will be available through the following registration link: View Source Registration for this virtual event and access to a replay of the live webcast will also be available on the Investors & Media section of www.poseida.com. A replay of the webcast will be available for approximately 90 days following the presentation.

Cash Position
As of June 30, 2024, the Company’s cash, cash equivalents and short-term investments balance was $237.8 million. With the $15 million milestone from Roche’s nomination of a new development candidate, the Company expects that its cash, cash equivalents and short-term investments together with other remaining near-term milestones and other payments from Roche will be sufficient to fund operations into early 2026. Potential additional anticipated progress and payments under the Roche Collaboration Agreement and/or potential additional business development could further extend the cash runway.

On October 17, 2024 Factor Bioscience Inc. ("Factor") and Eterna Therapeutics Inc. ("Eterna") reported an exclusive license and collaboration agreement aimed at accelerating the development of advanced cell therapy candidates for oncology, rare diseases, and autoimmune disorders (Press release, Factor Bioscience, OCT 17, 2024, View Source [SID1234647252]). Under the terms of the agreement, Eterna has secured a worldwide, exclusive, non-transferable, royalty-bearing license, with the right to grant sublicenses, to develop and market certain induced pluripotent stem cell (iPSC)-based cell therapy products (in particular iPSC derived mesenchymal stem cells (iMSC) that are engineered to express certain cytokines) utilizing Factor’s cell reprogramming and gene-editing technologies, patents, and know-how. The agreement positions both companies with the potential to accelerate the development of advanced cell therapies in the oncology, rare disease, and autoimmune fields.

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"We are excited to collaborate with Factor to unlock the full potential of these transformative cell therapies," said Sanjeev Luther, Eterna’s President & CEO. "Our focus at Eterna has always been on developing transformative treatments for patients with difficult-to-treat diseases, and we believe this collaboration agreement increases our potential to rapidly advance therapies that target solid tumors, rare diseases, and autoimmune disorders."

Factor and Eterna will collaborate on data generation that seeks to demonstrate efficacy of the licensed drug candidates for development towards IND by Eterna directly or with third-party sublicensees. As part of the agreement, Factor is entitled to receive milestone payments per product candidate, as well as post-commercialization royalties.

"We are thrilled to support Eterna’s pipeline of advanced cell therapy product candidates," said Matt Angel, Ph.D., CEO of Factor. "Eterna’s team has deep translational and development expertise, making them the ideal partner to develop these products. Together, we are taking an important step toward making these therapies available to patients in need."

On October 17, 2024 Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class medicines for hematologic malignancies and solid tumors, reported that the South Korean Ministry of Food and Drug Safety (MFDS) has approved a supplemental New Drug Application (sNDA) for XPOVIO (selinexor) in combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma (MM) who have received at least one prior therapy (Press release, Antengene, OCT 17, 2024, View Source [SID1234647251]).

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Prior to the recent approval, XPOVIO has been approved for two indications in South Korea that are: in combination with dexamethasone for the treatment of adult patients with relapsed or refractory MM (R/R MM); and as a monotherapy for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL). In July 2024, XPOVIO was included into the reimbursement drug list in South Korea, thus became the first XPO1 inhibitor approved for public insurance coverage in the country.

With a novel mechanism of action, XPOVIO is the world’s first approved orally-available, selective XPO1 inhibitor, which has already been approved in nine countries and regions in APAC and included in the national insurance schemes in South Korea, the mainland of China, Australia, and Singapore. This recent approval for XPOVIO in South Korea will bring another innovative therapy to the clinical management of MM patients in South Korea, benefiting countless patients and families.

While bringing XPOVIO to more APAC markets, Antengene is also striving to expand the indications of XPOVIO. Leveraging the drug’s novel mechanism of action, Antengene is currently developing multiple combination regimens of XPOVIO for the treatment of various indications including myelofibrosis (MF), and endometrial cancer.

About XPOVIO (selinexor)

XPOVIO is the world’s first approved orally-available, selective inhibitor of the nuclear export protein XPO1. It offers a novel mechanism of action, synergistic effects in combination regimens, fast onset of action, and durable responses.

By blocking the nuclear export protein XPO1, XPOVIO can promote the intranuclear accumulation and activation of tumor suppressor proteins and growth regulating proteins, and down-regulate the levels of multiple oncogenic proteins. XPOVIO delivers its antitumor effects through three mechanistic pathways: 1) exerting antitumor effects by inducing the intranuclear accumulation of tumor suppressor proteins; 2) reducing the level of oncogenic proteins in the cytoplasm by inducing the intranuclear accumulation of oncogenic mRNAs; 3) restoring hormone sensitivity by activating the glucocorticoid receptors (GR) pathway. To utilize its unique mechanism of actions, XPOVIO is being evaluated for use in multiple combination regimens in a range of indications. At present, Antengene is conducting multiple clinical studies of XPOVIO in the mainland of China for the treatment of relapsed/refractory hematologic malignancies and solid tumors (3 of these studies are being jointly conducted by Antengene and Karyopharm Therapeutics Inc. [Nasdaq:KPTI]).

On October 17, 2024 Medigene AG (Medigene or the "Company", FSE: MDG1, Prime Standard), an oncology platform company focused on the research and development of T cell receptor (TCR)-guided therapies for the treatment of cancer, reported a universal detection system for 3S (specific, sensitive, and safe) recombinant TCRs (rTCRs), UniTope & TraCR, at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) to be held October 16-17, 2024 in Philadelphia, PA in the US (Press release, MediGene, OCT 17, 2024, View Source [SID1234647250]).

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The presented poster "Seamless Integration of a Universal Epitope into Recombinant TCRs for Tagging and Tracking of TCR-T Cells Expressing 3S TCRs" will be made available after the conference on Medigene’s website: View Source

"We incorporate innovative technologies into our End-to-End Platform, including UniTope and TraCR, a universal detection system for any rTCR across multiple modalities. These two tools may ensure precise tracking and verification of recombinant TCRs contributing to safer and more effective dosing strategies in the development and administration of TCR-guided therapies," said Dolores Schendel, CSO at Medigene. "Following the promising data previously presented at this year’s annual ESMO (Free ESMO Whitepaper) meeting for UniTope-modified rTCRs targeting NY-ESO-1/LAGE-1a, we are excited to now share our initial in vitro data for UniTope-modified rTCRs mKRAS G12V. The UniTope tag, when directly integrated, ensures that a unique identifier is always expressed in rTCR sequences. This represents a major improvement over existing techniques for detecting rTCRs in TCR-guided therapies. The use of UniTope and TraCR technologies will significantly enhance our ability to streamline quality control processes and generate highly accurate data for establishing the ideal drug dosage leading to significant cost and time reductions throughout development."

The poster showcased the Company’s newly developed universal TCR tagging and tracking technology, UniTope & TraCR. Through bioinformatic analysis of T cell receptor beta variable sequences, a six-amino-acid peptide, UniTope, was identified, that is absent in natural TCR beta chains and exhibits low immunogenicity. Further, the UniTope sequence allowed each rTCR to be tagged, adaptable for multi-parameter flow cytometry and ideal for standardized tracking and enrichment of rTCR-T cells with a design that’s easy to implement in complex gene transfer systems. Alongside this, an antibody (TraCR) was also designed to specifically recognize and bind to this unique peptide sequence, enabling precise tracking of the tagged TCRs.

In vitro data demonstrated that insertion of the UniTope tag to rTCR-T cells targeting mKRAS G12V did not alter the expression or functionality of the rTCR. The tagged rTCRs maintained their normal biological activity, indicating that UniTope integration has no negative impact on functionality of the rTCR.

Additionally, safety assessments verified that UniTope-modified rTCRs exhibited the same high safety profile as their unmodified counterparts. These modified rTCRs did not show any unintended recognition or cytotoxic activity against 16 different types of healthy cells tested, ensuring that addition of the UniTope tag does not compromise safety.

Finally, UniTope-modified rTCRs demonstrated equivalent specificity, sensitivity, and safety characteristics compared to unmodified rTCRs. making them highly versatile for use across various therapeutic modalities, including T cell receptor engineered T cell therapies, TCR-guided T cell engagers, and TCR-NK cell therapies.

On October 17, 2024 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing innovative immuno-oncology technologies addressing hard to treat oncology indications, reported it has entered into a Materials Transfer Agreement with Tokyo Medical University to advance the development of its systemic DNase program (Press release, Xenetic Biosciences, OCT 17, 2024, View Source [SID1234647249]).

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Under the terms of the agreement, Professor Takuro Nakamura of the Department of Experimental Pathology, Institute of Medical Science at Tokyo Medical University will lead the research program evaluating the effects of human recombinant DNase I (rhDNase I) when given in combination with chemotherapy in a proprietary immunocompetent preclinical mouse model of Ewing sarcoma. Professor Takuro Nakamura’s proprietary immunocompetent Ewing sarcoma model encompasses the biological characteristics, morphology and gene expression profiles of human Ewing sarcoma and has demonstrated translational relevance.

Ewing sarcoma is an aggressive orphan pediatric cancer that grows in bones or soft tissues, accounting for between 2 to 3 percent of all childhood cancers. There is a lack of effective treatment options for children with recurrent and metastatic disease where the five-year survival rate is only 20 to 30 percent for patients that have relapsed.

Clinical studies conducted at Tel Aviv Medical Center between 2010 and 2021 [1] showed that the formation of neutrophil extracellular traps (NETs) in the tumor microenvironment of Ewing sarcoma is an independent prognostic factor, with a clear association between NETs burden and poor prognosis. According to research from these clinical studies, elevated levels of NETs at diagnosis predicted a poor response to neoadjuvant chemotherapy, relapse, and death from the disease.

Xenetic’s proprietary recombinant DNase I is an enzyme that digests NETs in tumor microenvironment. The preclinical studies are designed to evaluate the efficacy of DNase to reduce NETs burden and to increase the efficacy of chemotherapy given in an adjuvant setting.

James Parslow, Interim Chief Executive Officer and Chief Financial Officer of the Company stated, "As part of our overall development strategy, we aim to leverage relationships like the one established with Tokyo Medical University. Our commitment to the DNase program remains steadfast, and we are pleased to enter into this agreement to further expand our growing body of data."

About DNase-Based Oncology Platform

Xenetic’s DNase-based oncology platform is designed to target NETs, which are weblike structures composed of extracellular chromatin coated with histones and other proteins. In cancer, NETs are expelled by activated neutrophils into the tumor microenvironment and blood, thereby promoting cancer spread and local and systemic immunosuppression. Reduction of NETs burden via application of Xenetic’s proprietary recombinant human DNase I has been shown to improve efficacy of immunotherapy, adoptive cell therapy and chemotherapy in preclinical animal models.