On August 5, 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 updates and financial results for the second quarter ended June 30, 2024 (Press release, Poseida Therapeutics, AUG 5, 2024, View Source [SID1234645343]).
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"Poseida has delivered strong progress across our pipeline of innovative, non-viral allogeneic cell therapy and genetic medicine programs so far in 2024, setting the stage for significant potential catalysts in the second half of the year," said Kristin Yarema, Ph.D., President and Chief Executive Officer of Poseida Therapeutics. "For CAR-T, this is highlighted by multiple clinical data readouts across our BCMA, CD19CD20 and MUC1-C programs before year-end, the ongoing advancement of our partnerships with Roche and Astellas, and progress on our strategic approach to leveraging our allogeneic platform for autoimmune disease. We also expect new data from our genetic medicine programs that will continue to support the path towards IND filings and potential business development opportunities."
Recent Accomplishments
Cell Therapy
Advanced Roche partnership, securing $45 million from milestone execution in the first half of 2024, with additional milestones anticipated in the second half of the year, including a payment related to the planned initiation of the Phase 1b portion of the P-BCMA-ALLO1 clinical trial. Poseida and Roche have aligned on the Phase 1b trial design, which incorporates process improvements and feedback from recently completed advisory board meetings with leading clinicians and the study is expected to commence shortly. Poseida will continue to have operational responsibility for the expanded Phase 1/1b trial, which will be funded by Roche.
Progressed the strategic research collaboration and license agreement with Astellas’ wholly owned subsidiary Xyphos Biosciences with the formal nomination of the first high-potential program target. Poseida received $50 million in an upfront payment during the second quarter of 2024 related to the initiation of the license agreement.
Genetic Medicine
Demonstrated ongoing leadership in development of non-viral approach to genetic medicines, supported by multiple data presentations at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting featuring Poseida’s lead genetic medicine approaches.
ASGCT data highlights:
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In P-KLKB1-101 for hereditary angioedema (HAE), interim preclinical data in a non-human-primate (NHP) model showed that the Cas-CLOVER nuclease formulation was well tolerated and yielded dose-dependent levels of editing in early read-out data.
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Studies in human cells and rodent models show high fidelity and high efficiency KLKB1 editing within a target range for correction of HAE. P-KLKB1-101 demonstrated a highly controlled dose-dependent reduction in kallikrein protein with human hepatocyte studies revealing minimal off-target editing. This was consistent with findings that Cas-CLOVER displays approximately 20-fold higher fidelity than Cas9, across multiple cell types and targets.
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In P-FVIII-101 for Hemophilia A, preclinical rodent data support advancing to NHP studies based on sustained FVIII expression at physiologically desired levels over 13 months from a single dose, along with data supporting significantly reduced immunogenicity (relative to a conventional lipid nanoparticle (LNP)) and the ability to fine tune FVIII expression levels via repeat dosing and/or Poseida’s proprietary modulator switch.
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Additional data describing the Company’s advancements in its proprietary LNP technology, intracellular targeting agents and nuclease fidelity.
In addition, the FDA recently granted an INTERACT meeting for P-FVIII-101 in September 2024, which will provide Poseida with early engagement and communication with FDA on the program, in order to support efficient development that is aligned with FDA standards. INTERACT meetings focus on innovative and emerging technologies covered by the FDA’s Center for Biologics Evaluation and Research (CBER).
Upcoming Milestones
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P-BCMA-ALLO1 in relapsed/refractory multiple myeloma (RRMM): new data anticipated for presentation at the International Myeloma Society 21st Annual Meeting, being held September 25-28, 2024, in Rio de Janeiro. Additional clinical updates are planned for the second half of 2024, subject to coordination with Roche.
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P-MUC1C-ALLO1 in solid tumors: clinical update planned for the second half of 2024.
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P-CD19CD20-ALLO1 in B-cell malignancies: interim data update anticipated in the second half of 2024, subject to coordination with Roche.
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P-KLKB1-101 for HAE and P-FVIII-101 for Hemophilia A: data updates anticipated in the fourth quarter 2024.
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 superior 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 the Company, including its earlier stage allogeneic CAR-T pipeline. Further details on the event will be disclosed at a later time.
Evaluating Opportunities in CAR-T Beyond Oncology
The Company believes its TSCM-rich CAR-T platform and associated proprietary technologies have strong potential to deliver new therapeutic approaches in autoimmune disease. The Company remains well positioned to potentially advance an allogeneic CAR-T strategy for autoimmune disease and is actively working to identify the optimal opportunity to leverage its existing programs and/or platforms in areas where it is best positioned to lead. The Company will provide an update later this year.
Financial Results for the Second Quarter 2024
Revenues
Revenues were $26.0 million for the three months ended June 30, 2024, compared to $20.0 million for the same period in 2023. The increase was primarily due to milestone recognition and an increase in reimbursed research and development expenses under the Roche Collaboration Agreement.
Revenues were $54.1 million for the six months ended June 30, 2024 compared to $30.4 million for the same period in 2023. The increase was 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.
Research and Development Expenses
Research and development expenses were $45.5 million for the three months ended June 30, 2024, and $88.5 million for the six months ended June 30, 2024 compared to $39.2 million and $77.2 million for the same periods in 2023, respectively. The increases were 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 $12.2 million for the three months ended June 30, 2024, compared to $8.7 million for the same period in 2023. The increase was primarily due to higher personnel expenses, mainly caused by an increase in stock-based compensation expense driven by a one-time expense associated with the succession plan in which the Company’s former CEO became the Executive Chairman in 2024.
General and administrative expenses were $22.0 million for the six months ended June 30, 2024, compared to $20.5 million for the same period in 2023. The increase was primarily due to higher legal fees related to patent expenses and the Astellas Collaboration Agreement, and higher personnel expenses, mainly caused by an increase in stock-based compensation expense driven by a one-time expense associated with the succession plan in which the Company’s former CEO became the Executive Chairman in 2024.
Net Loss
Net loss was $31.4 million and $55.6 million for the three and six months ended June 30, 2024, respectively, compared to net loss of $27.5 million and $66.3 million for the three and six months ended June 30, 2023, respectively.
Cash Position
As of June 30, 2024, the Company’s cash, cash equivalents and short-term investments balance was $237.8 million. This includes $95 million in milestone and upfront payments generated in the first half of 2024, consisting of a $50 million upfront payment from the Astellas collaboration and $45 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 the second half of 2025 and potential additional anticipated progress and payments under the Roche Collaboration Agreement and/or potential additional business development could further extend the cash runway.