On September 5, 2024 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a clinical-stage precision oncology company focused on the treatment and prevention of virus-associated cancers that impact patients worldwide, reported that Mark Rothera, President and Chief Executive Officer, will present at the H.C. Wainwright 26th Annual Global Investment Conference taking place in New York City on Wednesday, September 11, 2024, at 8:30 a.m. EDT (Press release, Viracta Therapeutics, SEP 5, 2024, View Source [SID1234646378]).

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A live webcast of the presentation will be available on the Investors section of the Viracta website under "Events and Presentations" and archived for 90 days.

On September 5, 2024 Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, reported new clinical data from its ongoing Phase 1/2 VBP101 study of patients with relapsed/refractory AML receiving trem-cel followed by MylotargTM (Press release, Vor BioPharma, SEP 5, 2024, View Source [SID1234646379]). The data demonstrated reliable engraftment, shielding from Mylotarg on-target toxicity, a broadened Mylotarg therapeutic window, and early evidence of patient benefit.

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"We are encouraged by this data and the potential benefit that trem-cel in combination with Mylotarg may offer to patients in a disease that has extremely poor outcomes even after transplant," said Dr. Eyal Attar, Vor Bio’s Chief Medical Officer. "With this data, we plan to explore a registrational trial while we continue to pursue other synergistic opportunities for Vor Bio’s platform such as VCAR33ALLO and VADC45."

The data released today included 18 patients treated with trem-cel of which ten had received Mylotarg as of the data cut-off date of July 19, 2024. The data demonstrated:

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Reliable engraftment, with 100% of patients achieving primary neutrophil engraftment (median 9 days) and robust platelet recovery (median 16.5 days). High CD33 editing efficiency (median 89%, range 71-94%) and full myeloid chimerism at Day 28.

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Shielding of the blood system, with maintained neutrophil and platelet counts across multiple Mylotarg doses of 0.5, 1, and 2 mg/m2.

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Broadened therapeutic index for Mylotarg with drug exposure represented by AUC which is related to efficacy, consistent with labeled Mylotarg doses, and with maximal concentrations, measured by Cmax and related to veno-occlusive disease, well below known toxic range.

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Early evidence suggesting patient benefit as measured by relapse-free survival when compared to published high-risk AML comparators1.

"All the hope I had in the safety of this approach has been supported by the data from this trial thus far," said Guenther Koehne, MD, PhD, an investigator on the VBP101 study and Deputy Director and Chief of Blood & Marrow Transplant and Hematologic Oncology at Miami Cancer Institute of Baptist Health South Florida, "I look forward to treating my next patients at high risk of relapse on this trial as their outcomes are otherwise limited with standard transplants."

Vor Bio plans to approach the U.S. Food & Drug Administration to discuss a pivotal trial design for trem-cel + Mylotarg by around year end.

Continued progress with VCAR33ALLO

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VCAR33ALLO represents another potentially significant synergistic treatment option after trem-cel.

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The VBP301 study continues enrolling patients with initial focus on relapsed/refractory AML post-transplant.

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Vor Bio is encouraged by in vivo CAR-T expansion data from three patients treated to date, all at the lowest dose of 1 x 106 CAR+ cells/kg.

Vor Bio announced today, a new preclinical asset, VADC45, which has a number of potential opportunities in oncology, gene therapy, and autoimmune disorders.

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VADC45 is an ADC that targets the CD45 protein. CD45 is a well-validated target for a wide variety of blood cancers with clinical proof of concept. The linker-payload used in VADC45 is also clinically validated.

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VADC45 has the potential to treat a number of diseases, including treatment of hematologic malignancies, as a targeted conditioning agent for gene therapies such as for sickle cell disease, holistic immune reset for autoimmune disorders, and for Vor Bio’s approach of combining this asset with epitope modification of CD45 to shield healthy stem cells.

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Vor Bio already has robust preclinical data for VADC45 and is progressing IND-enabling studies to enable future Phase 1 studies.

On September 5, 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 that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for its lead program MDG1015 for the treatment of advanced gastric cancer, ovarian cancer, myxoid/round cell liposarcoma and synovial sarcoma in the phase 1 clinical trial (EPITOME1015-I) (Press release, MediGene, SEP 5, 2024, View Source [SID1234646380]).

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EPITOME1015-I consists of a dose escalation followed by an expansion segment and aims to assess safety, feasibility and preliminary efficacy of MDG1015 in multiple solid tumor indications.

"We are very excited to reach this milestone with our lead program MDG1015 which reinforces our ambition to become a leading company with a range of different TCR-guided therapies for patients suffering from multiple advanced solid tumor types," said Selwyn Ho, CEO of Medigene AG. "In preclinical studies, MDG1015 has shown strongly enhanced and persistent T cell-driven anti-tumor activity and the ability to mitigate the effects of PD-L1, one of the major immunosuppressive signals present in the tumor microenvironment of solid cancers which hinder the effectiveness of TCR-T therapies. This very first FDA clearance of an IND Application for a Medigene TCR-T therapy represents a pivotal achievement, and we look forward to commencing our MDG1015 phase 1 study EPITOME1015-I targeting multiple solid tumors, subject to additional financing."

MDG1015 is a first-in-class, third generation T cell receptor engineered T cell (TCR-T) therapy targeting the cancer-testis antigen New York esophageal squamous cell carcinoma 1 / L Antigen Family Member-1a (NY-ESO-1/LAGE-1a) with a natural and optimal affinity 3S (specific, sensitive and safe) TCR and human leukocyte antigen (HLA)-A*02. The TCR-T cells are further armored and enhanced by the addition of the proprietary PD1-41BB costimulatory switch protein (CSP) technology and has demonstrated significantly enhanced anti-tumor activities against tumor cells expressing varying levels of PD-L1, one of the most immunosuppressive signals emanating from the solid tumor microenvironment. Importantly, compared to first generation TCR-T therapies, MDG1015 will be manufactured with a short, 6-day cell expansion period, leading to younger, fitter cells, with the potential for a markedly reduced number of cells required during dosing and a shorter vein-to-vein time for patients of approximately 20 days. This has also resulted in a drug product with an almost pure CD8+ population and with a very high proportion of cells with stemness-like qualities (~95%) that could lead to improved durability of response, greater efficacy and reduced adverse events.

To complement the IND approval, a Clinical Trial Application (CTA) submission for MDG1015 to the European Medicines Agency (EMA) is on track for the fourth quarter of 2024. Pending additional financing, the Company plans to initiate the phase 1 clinical trial EPITOME‑1015-I, which consists of a dose escalation followed by an expansion segment, by the end of 2024. Based on this timeline, the Company expects to be able to present early data from the dose escalation phase towards the end of 2025.

On September 4, 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 and rare diseases, reported new data from a case study of a patient with relapsed multiple myeloma treated in a clinical trial of P-BCMA-101, the Company’s original investigational T stem cell memory (TSCM)-rich BCMA targeting autologous CAR-T cell therapy (Press release, Poseida Therapeutics, SEP 4, 2024, View Source [SID1234646349]). The data were presented in an oral session at the Society of Hematologic Oncology (SOHO) Twelfth Annual Meeting in Houston.

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"This case study demonstrates the remarkable potential of T stem cell memory-based therapies, providing a strong anti-myeloma response with a long-term remission and notably CAR-T cell persistence," said Thomas G. Martin, M.D., Clinical Professor of Medicine, Adult Leukemia and Bone Marrow Transplantation Program and Director of Hematology, Blood and Marrow Transplantation and Cellular Therapy at UCSF, and co-leader of the Cancer Immunology & Immunotherapy Program at the UCSF Helen Diller Family Comprehensive Cancer Center. "Most notably, we believe this is the first time that a T-cell engager has been seen to reactivate a CAR-T therapy, and the evidence suggests that this reactivation drove a second wave of CAR-T cell proliferation that led to another complete response three years after the initial successful CAR-T treatment. This patient is now off all anti-myeloma treatments and living in remission for more than nine months following 1 week of TCE therapy, a truly amazing outcome."

"These patient clinical data demonstrate the power of TSCM CAR-T cells, which are a core element of all our investigational next-generation, off-the-shelf allogeneic CAR-T cell therapies," said Syed Rizvi, M.D., Chief Medical Officer of Poseida Therapeutics. "P-BCMA-101 demonstrated durable persistence due to the high TSCM content in the final product. This long-term persistence and engraftment led to activation by the TCE several years after initial CAR-T therapy. The high TSCM content and durable persistence is a unique feature of all autologous and allogeneic Poseida CAR-T."

Background Information and Oral Presentation Highlights
Patients with relapsed/refractory multiple myeloma who receive BCMA-directed CAR-T therapy can achieve deep and durable remissions, but most patients relapse. Detection of CAR-positive cells wanes rapidly over the first six months, and significant re-expansion of CAR-T cells has not been demonstrated previously in the clinical setting.

In this case study, a 57-year-old female patient with relapsed multiple myeloma received P-BCMA-101, an investigational TSCM-rich autologous CAR-T therapy. TSCM cells are a subset of T cells that have unique properties: They are long-lived, multi-potent and self-replicating and can engraft and create differentiated cells.

Two months after receiving treatment, the patient achieved a partial response that deepened into a stringent complete response and remained in remission for nearly 2 years (22.5 months). More than three years after receiving P-BCMA-101, she relapsed and was treated with one cycle of talquetamab, a T-cell engaging bispecific antibody that targets CD3 and GPRC5D. Upon receiving talquetamab, the patient developed a brisk lymphocytosis. Evaluation of peripheral blood revealed high levels of P-BCMA-101 CAR-T cells. Thorough molecular analysis revealed that the lymphocytosis was benign and reactive. The patient achieved complete remission with slow resolution of lymphocytosis. The patient continues to be in sCR and off all therapy more than nine months after receiving the last and only full dose of the T-cell engaging therapy.

Poseida’s lead investigational allogeneic CAR-T program, P-BCMA-ALLO1, is currently being evaluated in patients with relapsed/refractory multiple myeloma. The Company will report new clinical data at the International Myeloma Society 21st Annual Meeting, which is being held in Rio de Janeiro from September 25-28, 2024. Additional P-BCMA-ALLO1 clinical updates are planned for the second half of 2024, subject to coordination with Roche, which has a strategic collaboration with Poseida covering multiple investigational allogeneic CAR-T therapies targeting blood cancers, including P-BCMA-ALLO1.

In November 2022, Poseida made the strategic decision to transition its cell therapy focus from an autologous to an allogeneic approach. The Company believes the future of cell therapy and its ability to offer new treatment options lies in an allogeneic approach in which T cells are derived from healthy donors rather than from the patients themselves. Poseida has applied learnings from its autologous programs to support the development of its allogeneic pipeline.

About P-BCMA-ALLO1
P-BCMA-ALLO1 is an investigational allogeneic CAR-T therapy licensed to Roche that targets B-cell maturation antigen (BCMA) for the treatment of patients with relapsed/refractory multiple myeloma. This allogeneic program includes a VH-based binder that targets BCMA. Phase 1 clinical data presented at ASH (Free ASH Whitepaper) 2023 supports the Company’s belief that TSCM-rich allogeneic CAR-Ts have the potential to offer an effective, safe and reliable treatment addressing unmet needs in multiple myeloma. The U.S. Food and Drug Administration granted Orphan Drug Designation to P-BCMA-ALLO1 for the treatment of multiple myeloma. Additional information about the Phase 1 study is available at www.clinicaltrials.gov (NCT04960579).

On September 4, 2024 Fortress Biotech, Inc. (Nasdaq: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue, reported that Lindsay A. Rosenwald, M.D., Chairman, President and Chief Executive Officer, will present a corporate overview at the H.C. Wainwright 26th Annual Global Investment Conference (Press release, Fortress Biotech, SEP 4, 2024, View Source [SID1234646350]). The presentation will be available for on-demand viewing by conference attendees starting on September 9, 2024, at 7:00 a.m. ET. The Company will also attend one-on-one meetings during the conference.

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