On November 7, 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 updates and financial results for the third quarter ended September 30, 2024 (Press release, Poseida Therapeutics, NOV 7, 2024, View Source [SID1234647944]).
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"Poseida continues to make excellent progress across all of our key initiatives, highlighted by compelling data presentations from our pipeline of innovative, non-viral allogeneic cell therapy and genetic medicine programs, the expansion and advancement of our collaborations with Roche and Astellas, and our science-backed strategy to apply our platform to the large and growing opportunity for CAR-T in autoimmune diseases," said Kristin Yarema, Ph.D., President and Chief Executive Officer of Poseida Therapeutics. "As a result, we have generated $130 million in non-dilutive, partnership related milestones and payments so far this year, along with $49 million earned through R&D expense reimbursements. This has resulted in Poseida being cash flow positive for the first nine months of the year and extended our cash runway, with additional upside potential from continued execution, clinical progress and business development. We look forward to sharing updates on our CAR-T programs at our upcoming Cell Therapy R&D Day and future medical meetings."
Recent Accomplishments
Cell Therapy
Expanded strategic global collaboration with Roche, including ongoing pipeline progress and the nomination of a new dual CAR-T development candidate.
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The new candidate is an allogeneic, dual CAR-T therapy targeting known antigens expressed in hematologic malignancies, including multiple myeloma. Poseida and Roche now have three programs under their collaboration, including P-BCMA-ALLO1, an allogeneic CAR-T therapy in Phase 1/1b development for multiple myeloma, and P-CD19CD20-ALLO1, an allogeneic dual CAR-T candidate in Phase 1 development for B-cell malignancies. Roche has the option to nominate additional development candidates in the future.
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P-BCMA-ALLO1 continues to be positioned as a leading clinical-stage allogeneic CAR-T therapy with a compelling and differentiated profile: interim Phase 1 data presented at the International Myeloma Society (IMS) annual meeting in September showed a 91% overall response rate (ORR) in an optimized lymphodepletion arm, including a 100% ORR in B-cell maturation antigen (BCMA)-naïve patients, and an 86% ORR in those who had received at least one prior BCMA- and/or G protein-coupled receptor class C group 5 member D (GPRC5D)-targeting treatment modality, along with differentiated safety results with no dose-limiting toxicities, low rates of cytokine release syndrome (CRS) and immune effector cell neurotoxicity syndrome (ICANS) all Grade 2 or less and no graft vs. host disease or Parkinsonism. No anti-myeloma bridging therapy or prophylaxis with steroids or tocilizumab, no invasive apheresis or manufacturing wait with average time from treatment decision to clinical response of only 3.5 weeks1 (median time to response of 16 days post initial P-BCMA-ALLO1 therapy). The patients in this study had more advanced disease than the myeloma patients studied in clinical trials of approved autologous CAR-T therapies2, and in the intent-to-treat population, 100% of patients were infused with P-BCMA-ALLO1.
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Poseida’s conference call to discuss IMS data featured a fireside chat with leading myeloma experts that highlighted key aspects of the data and provided context on current treatment approaches. The experts highlighted that off-the-shelf availability allows patients to start therapy fast, without need for bridging therapy commonly required by autologous CAR-T, and in some cases, offers a solution for patients who are ineligible for autologous treatments; rapid clinical responses; attractive safety profile; ability to treat patients on an outpatient basis; and ability to treat BCMA-exposed patients as key benefits observed in the trial.
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P-BCMA-ALLO1 development continues, with ongoing patient enrollment in the recently initiated Phase 1/1b trial that is using the same lymphodepletion regimen as the optimized lymphodepletion arm described above, including two different cell doses. During the third quarter, P-BCMA-ALLO1 was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) to treat relapsed/refractory multiple myeloma after three or more prior lines of therapies including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody.
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Poseida has secured $80 million from Roche collaboration milestones to-date in 2024, including a milestone payment received in the third quarter related to the initiation of the P-BCMA-ALLO1 Phase 1b clinical trial, and an additional payment for the nomination of the new development candidate in October.
Progressed the strategic research collaboration and license agreement with Astellas’ wholly owned subsidiary Xyphos Biosciences with the formal nomination of the second high potential program target. Both program targets nominated under the collaboration are well-known and validated solid tumor targets.
Accelerating highly differentiated dual targeting allogeneic CAR-T for autoimmune disease with P-BCMACD19-ALLO1 as first pipeline candidate to address this significant market opportunity. P-BCMACD19-ALLO1 is an allogeneic dual CAR-T candidate currently in IND-enabling studies. We believe targeting BCMA and CD19 could provide the potential to enable more complete immune cell depletion than CAR-Ts targeting only one of the antigens, and that targeting BCMA specifically could provide the potential to deplete autoantibodies from plasma cells, which are believed to be a key driver in many autoimmune diseases and not addressed by targeting CD19 alone. Emerging data from an autologous dual CAR-T targeting BCMA and CD19 has thus far substantiated this dual targeting approach. P-BCMACD19-ALLO1 is also positioned to provide the access benefits of an allogeneic product and a potentially attractive safety profile derived from Poseida’s non-viral TSCM approach and other features unique to the Company’s CAR-T platform, such as its proprietary safety switch.
Strengthened Poseida’s innovation profile and emerging leadership in allogeneic CAR-T with new data at the Society of Hematologic Oncology (SOHO) Twelfth Annual Meeting in September and the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 39th Annual Meeting & Pre-Conference Programs in November.
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A new case study demonstrating the reactivation of an autologous Poseida CAR-T therapy with a T-cell engager in a patient with relapsed multiple myeloma was presented at SOHO. The patient attained and remains in stringent complete response over 10 months after CAR-T reactivation. This case highlights the potential of Poseida’s TSCM-based CAR-T therapies to deliver a strong anti-myeloma response with long-term remission and CAR-T cell persistence. The Company believes this is the first time that a T-cell engager has been seen to reactivate a CAR-T therapy.
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New preclinical data highlighting the potential of Poseida’s platform to design and manufacture CAR-TCR-T cells that are rich in stem cell memory T cell (TSCM) and central memory T cells (TCM) for potential use in solid tumors will be presented at SITC (Free SITC Whitepaper) on November 9. In solid tumors, multi-antigens are believed to be necessary for efficacy, and CAR+TCR-T cells can recognize and kill single and double antigen target cells and show the potential to synergize with T-cell engagers. Key highlights from the SITC (Free SITC Whitepaper) presentation include:
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CAR+TCR-T cells were shown to control single and double antigen-positive tumor growth in vivo, with sustained persistence.
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T-cell engager was used to re-activate and re-direct engrafted CAR+TCR-T cells to control a secondary tumor challenge expressing different antigens echoing the patient case study presented at SOHO and suggesting an approach to address heterogeneous tumors and/or tumors whose composition evolves over time.
Continued to enroll patients in the Phase 1 clinical trial of P-CD19CD20-ALLO1. In light of the competitive environment for therapies targeting CD19 and CD20, Poseida and Roche anticipate providing initial clinical data from the trial in 2025 once a more complete dataset of the program is available.Genetic Medicines
Demonstrated ongoing leadership in development of non-viral approaches to genetic medicines, supported by data presentation at the American College of Allergy, Asthma & Immunology (ACAAI) 2024 Scientific Meeting featuring P-KLKB1-101, a non-viral, liver-directed genetic medicine that uses the Company’s Cas-CLOVER Site-Specific Gene Editing System. The data showed high-fidelity gene editing at KLKB1 for the targeted correction of hereditary angioedema (HAE), the ability for controlled dose response, favorable tolerability and liver editing approaching the desired therapeutic range. The data reinforce the potential of Cas-CLOVER to be a unique and attractive gene editing tool for a variety of diseases, starting with HAE and KLKB1 editing.
In addition, Poseida had a successful INTERACT meeting with the U.S. FDA for P-FVIII-101 in September 2024. The meeting provided Poseida with early engagement and communication with FDA on the program, in order to support efficient development designed to be aligned with FDA standards. INTERACT meetings focus on innovative and emerging technologies covered by the FDA’s Center for Biologics Evaluation and Research (CBER).
Other Operational Updates and Upcoming Events
Manufacturing Updates
The Company continues to advance its platform process and analytical capabilities for allogeneic cell therapy manufacturing. Recent analytical enhancements have enabled more precise evaluation of prospective donors as well as providing improved characterization of drug product attributes.
Cell Therapy R&D Day
Poseida will host a cell therapy-focused R&D Day on November 14, 2024, to highlight progress and further opportunities 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.
Financial Results for the Third Quarter 2024
Revenues
Revenues were $71.7 million for the three months ended September 30, 2024, and $125.9 million for the nine months ended September 30, 2024, compared to $9.4 million and $39.7 million for the same periods in 2023, respectively. The increases were primarily due to milestone recognition and an increase in reimbursed research and development expenses under the Roche Collaboration Agreement, and revenue recognized from the Astellas Strategic Agreements and Astellas Collaboration Agreement.
Research and Development Expenses
Research and development expenses were $41.9 million for the three months ended September 30, 2024, compared to $37.5 million for the same period in 2023. The increase was primarily due to an increase in allogeneic clinical stage programs, driven mainly by an increase in overall enrollment of the Company’s allogeneic programs, and by an increase in preclinical stage programs and other unallocated expenses.
Research and development expenses were $130.4 million for the nine months ended September 30, 2024, compared to $114.7 million for the same period in 2023. The increase was primarily due to an increase in allogeneic clinical stage programs, driven mainly by an increase in overall enrollment of the Company’s allogeneic programs and the initiation of its third allogeneic clinical trial, P-CD19CD20-ALLO1, and by an increase in preclinical stage programs and other unallocated expenses.
General and Administrative Expenses
General and administrative expenses were $10.1 million for the three months ended September 30, 2024, and $32.1 million for the nine months ended September 30, 2024, compared to $8.1 million and $28.6 million for the same periods in 2023, respectively. The increases were primarily due to increased legal and professional fees as a result of higher patent-related and other consulting costs, and higher personnel expenses.
Net Income (Loss)
Net income was $20.2 million for the three months ended September 30, 2024, and net loss was $35.4 million for the nine months ended September 30, 2024, compared to net loss of $31.8 million and $98.1 million for the three and nine months ended September 30, 2023, respectively.
Cash Position
As of September 30, 2024, the Company’s cash, cash equivalents and short-term investments balance was $230.9 million. This includes $115 million in milestone and upfront payments generated in the first nine months of 2024, consisting of a $50 million upfront payment from the Astellas collaboration and $65 million from continued execution in the Company’s CAR-T partnership with Roche. The Company expects that its cash, cash equivalents and short-term investments together with these and 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.