Poseida Therapeutics Provides Updates and Financial Results for the Fourth Quarter and Full Year 2023
- Positive Phase 1 clinical trial data for P-BCMA-ALLO1 with 82% ORR
- Initiation of Phase 1 trial for P-CD19CD20-ALLO1
- Revenue increase to $25.0 million in Q4 2023
- Net loss of $25.3 million for Q4 2023
- Cash balance of $212.2 million as of December 31, 2023
- Decrease in revenues for full year 2023 compared to 2022
- Increase in research and development expenses for Q4 and full year 2023
- General and administrative expenses remained relatively stable
- Net loss increased for full year 2023 compared to 2022
Insights
The 82% overall response rate (ORR) for P-BCMA-ALLO1 in the Phase 1 clinical trial is a significant finding, particularly for relapsed/refractory multiple myeloma (RRMM) patients who have limited treatment options. The safety profile, with all adverse events being ≤ Grade 2, suggests a potentially better tolerability compared to current therapies, which often have more severe side effects. The ability of TSCM-rich CAR-T cells to traffic to bone marrow and demonstrate persistence for at least 6 weeks could imply long-term efficacy, a crucial factor in treating RRMM.
The data indicating a 100% ORR in patients not previously treated with a BCMA-targeted bispecific T-cell engaging antibody could redefine treatment protocols for RRMM. This suggests that P-BCMA-ALLO1 may be more effective when used earlier in the treatment regimen. Moreover, the lack of need for bridging chemotherapy is notable, as it may reduce overall healthcare costs and improve patient quality of life during treatment. The exploration of higher lymphodepletion doses in the P-MUC1C-ALLO1 trial for solid tumors may lead to improved cell expansion and persistence, addressing a common challenge in CAR-T therapy for solid tumors.
The $30 million payment from Roche in Q1 2024, as a result of accelerated milestone achievements, is a positive financial indicator for Poseida Therapeutics. This collaboration, along with the reported revenues that increased significantly in Q4 2023 compared to the same period in 2022, reflects strong performance and potential for future revenue streams. The decrease in full-year revenues from 2023 compared to 2022 is attributed to the high initial license revenue in the previous year, which should be considered in the context of the company's overall growth trajectory. The cash position of $212.2 million and the projection that current funds will sustain operations into the second half of 2025 provide a stable financial outlook, potentially bolstered by additional payments from Roche or new business developments.
Lead asset P-BCMA-ALLO1 Phase 1 clinical trial data presented at ASH 2023 demonstrated
Initiated clinical trial of Phase 1 P-CD19CD20-ALLO1, the Company's first allogeneic dual CAR-T program
Allogeneic CAR-T data to be presented at AACR in April 2024 describing activity of P-BCMA-ALLO1 in BCMA-experienced patients and comparison of lymphodepletion needs in heme vs. solid tumor settings
P-FVIII-101 gene therapy data presented at ASH and Company to host Gene Therapy R&D Day April 17, 2024
"2024 looks to be a breakout year for Poseida as we build on the recent allogeneic BCMA clinical data presented at ASH, which demonstrated the potential of our high-TSCM allogeneic CAR-T therapies to offer effective, safe, and reliable treatment addressing unmet needs in multiple myeloma. We are able to achieve high levels of CAR-TSCM in our products because of our proprietary, nonviral technologies," said Kristin Yarema, Ph.D., President and Chief Executive Officer. "Over the coming months we are planning data readouts for each of our three clinical-stage CAR-T programs, all of which are manufactured at our own GMP facility and will discuss the latest progress of our gene therapy programs at our R&D Day in April."
Recent 2023 Accomplishments
Cell therapy
- Presented positive data from the Phase 1 study of P-BCMA-ALLO1 in relapsed/refractory multiple myeloma (RRMM) at ASH: In December 2023, the Company presented data from its Phase 1 study of P-BCMA-ALLO1 at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition. These early data support the Company's belief that TSCM-rich allogeneic CAR-Ts have the potential to offer promising product response rates, safety profiles, and rapid patient access which if confirmed could provide differentiation from currently available therapies, including autologous CAR-T therapies. The study used product from 6 different CAR-T lots produced from 6 different donors with data demonstrating:
82% ORR in deep clinical responses, including sCRs and MRD-negative patients from off-the-shelf, allogeneic BCMA-targeted CAR-T in heavily pretreated patients receiving adequate lymphodepletion.100% ORR in those patients who were not previously treated with a BCMA-targeted bispecific T-cell engaging antibody.- Favorable emerging safety and reliability profile, with all (
100% ) intent-to-treat patients receiving therapy with no use of bridging chemotherapy or other anti-myeloma bridging therapies and low incidences of CRS and neurotoxicity observed (all ≤ Grade 2). - Preliminary data suggest that allogeneic TSCM-rich CAR-T cells traffic to bone marrow, differentiate to cell-killing effector T cells and persist at least 6 weeks, supporting the hypothesis of cell persistence at tumor-relevant sites.
- 8 of 9 responding patients still in response at data cut off.
- Continued enrollment of P-MUC1C-ALLO1: The Company continued enrollment in the Phase 1 clinical trial of P-MUC1C-ALLO1 for solid tumors. Drawing from insights gained in allogeneic BCMA CAR-T, it is exploring various dosing approaches, including higher lymphodepletion, cell dose, and scheduling in the MUC1C program. Data from the allogeneic BCMA CAR-T program, presented at ASH, underscored the important role of cyclophosphamide conditioning dose in CAR-T cell expansion and persistence. Patients in arms receiving higher doses (500 mg/m2 and 1,000 mg/m2) demonstrated significantly elevated P-BCMA-ALLO1 levels compared to those in the 300 mg/m2 cohort. Consequently, the Company is assessing lymphodepleting conditioning regimens with doses exceeding 300 mg/m2 in the MUC1C program.
- Advanced programs within hematologic malignancies at all stages, in partnership with Roche, throughout 2023:
- Previously announced the expected acceleration of achieving milestones, resulting in the receipt of a
$30 million - Expanded the protocol and continued to enroll patients in the Phase 1 study of P-BCMA-ALLO1, incorporating lymphodepletion learnings from ASH 2023.
- Continued site activation activities and initiation of enrollment for P-CD19CD20-ALLO1, the Company's first dual, allogeneic CAR-T for B-cell malignancies.
- Advanced research programs optioned to Roche (P-BCMACD19-ALLO1 for multiple myeloma and P-CD70-ALLO1 for AML).
- Developed and deployed manufacturing platform improvements to all programs that delivered higher and more consistent levels of cell expansion.
- Previously announced the expected acceleration of achieving milestones, resulting in the receipt of a
Gene therapy
- Presented proof-of-principle data supporting P-FVIII-101 as a potential treatment option for Hemophilia A at ASH: New preclinical data presented at ASH demonstrated the capabilities of the Company's non-viral approach in providing stable Factor VIII (FVIII) transgene expression through genomic integration. Additionally, the data highlighted re-dosing, or titrating to efficacy, along with favorable tolerability data for this approach, including 52-week durability data in an adult Hemophilia A mouse model supporting sustained and robust expression.
Anticipated Milestones
- P-BCMA-ALLO1: The Company plans to present data at the American Association for Cancer Research (AACR) Annual Meeting in
San Diego . At AACR, the Company will present data on a subset of recently enrolled patients refractory to initial BCMA targeting therapy. The poster presentation, titled "Clinical Activity of P-BCMA-ALLO1, a B-cell Maturation Antigen (BCMA) Targeted Allogeneic Chimeric Antigen Receptor T-cell (CAR-T) Therapy, in Relapsed Refractory Multiple Myeloma (RRMM) Patients (pts) Following Progression on Prior BCMA Targeting Therapy," is anticipated to take place on April 8, 2024, 9:00 AM to 12:30 PM PT. Subject to coordination with Roche, the Company plans to provide an additional clinical update on this program at a scientific meeting in the second half of 2024. - P-MUC1C-ALLO1: The Company plans to review initial clinical findings from the Phase 1 clinical trial in solid tumors at AACR related to cell expansion and lymphodepletion regimens. The poster presentation, titled "Solid Tumor Patients Require Higher Cyclophosphamide Dose than Multiple Myeloma Patients to Achieve Adequate Lymphodepletion (LD) Necessary to Enable Allogeneic CAR-T Expansion," is anticipated to take place on April 8, 2024, 9:00 AM to 12:30 PM PT. In addition to the AACR presentation, the Company plans to present a more fulsome clinical update at an appropriate forum in the second half of 2024.
- P-CD19CD20-ALLO1: Following the initiation of the Phase 1 trial targeting B-cell malignancies in late 2023, the Company expects to provide an interim data update in the second half of 2024, subject to coordination with Roche.
- P-PSMA-ALLO1: In January 2024, Poseida announced that it expects to advance its next allogeneic solid tumor program, P-PSMA-ALLO1 in prostate cancer, into IND-enabling studies incorporating the progress and learnings of its previous autologous study and recent advancements in its allogeneic platform in 2024.
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 is actively developing a strategy in inflammation and autoimmune disease and will provide an update at an appropriate time.
Other Operational Updates and Upcoming Events
Poseida R&D Days
The Company plans to host two R&D Days in 2024 focusing on gene therapy and cell therapy respectively.
The Company plans to host its gene therapy specific R&D Day on April 17, 2024, which will feature presentations from gene therapy management as well as key opinion leaders. The event will highlight the Company's progress across its proprietary non-viral gene insertion and editing technologies, an update following a strategic review of its portfolio and new preclinical data across in vivo gene therapy programs.
The Company plans to host an R&D Day focusing on cell therapy in the second half of 2024.
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 is developing a strategy in inflammation and autoimmune disease and will provide an update at an appropriate time.
Leadership Updates
Alexander Chapman joined Poseida on February 26, 2024, as Senior Vice President, Investor Relations & Corporate Communications. He has over 20 years' experience spanning a broad range of strategic roles across the biotech sector. Prior to Poseida, he was the Head of Corporate Communications & Investor Relations at Atara Biotherapeutics, the first company in the world to receive regulatory approval for an allogeneic T-cell immunotherapy. Previously, Mr. Chapman held a series of global and
Loren Wagner was promoted to the role of Chief Operations Officer in February 2024. Mr. Wagner joined the Company in March 2021 and most recently served as Senior Vice President, Global Operations. He has more than 30 years of industry experience across a variety of manufacturing, quality, and engineering roles. Prior to the Company, Mr. Wagner was the Head of CAR-T Operations at Bristol Myers Squibb/Celgene. Prior to Celgene, he worked for 12 years at Allergan, where he was responsible for the production and distribution of investigational supplies globally.
Brent Warner, who has served as the Company's President, Gene Therapy, will depart Poseida to pursue an external career opportunity effective April 1, 2024.
Financial Results for the Fourth Quarter and Full Year 2023
Revenues
Revenues were
For the full year ended December 31, 2023, revenues were
Research and Development Expenses
Research and development expenses were
For the full year ended December 31, 2023, research and development expenses were
General and Administrative Expenses
General and administrative expenses were
For the full year ended December 31, 2023, general and administrative expenses were
Net Loss
Net loss was
Cash Position
As of December 31, 2023, the Company's cash, cash equivalents and short-term investments balance was
About Poseida Therapeutics, Inc.
Poseida Therapeutics is a clinical-stage biopharmaceutical company advancing differentiated cell and gene therapies with the capacity to cure certain cancers and rare diseases. The Company's pipeline includes allogeneic CAR-T cell therapy product candidates for both solid and liquid tumors as well as in vivo gene therapy product candidates that address patient populations with high unmet medical need. The Company's approach to cell and gene therapies is based on its proprietary genetic editing platforms, including its non-viral piggyBac® DNA Delivery System, Cas-CLOVER™ Site-Specific Gene Editing System, Booster Molecule, and nanoparticle and hybrid gene delivery technologies as well as in-house GMP cell therapy manufacturing. The Company has formed a global strategic collaboration with Roche to unlock the promise of cell therapies for patients with hematological malignancies. Learn more at www.poseida.com and connect with Poseida on X and LinkedIn.
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, expected plans with respect to clinical trials, including timing of regulatory submissions and approvals and clinical data updates; potential fees, milestones and other payments that the Company may receive pursuant to its collaboration agreement with Roche, including related timing; anticipated timelines and milestones with respect to the Company's development programs and manufacturing activities and capabilities; the potential capabilities and benefits of the Company's technology platforms and product candidates, including the efficacy and safety profile of such product candidates; the quote from Dr. Yarema; estimates of the Company's cash balance, expenses, capital requirements and any future revenue; the Company's ability to exploit and consummate additional business development opportunities, including with Roche, and any anticipated impact on the Company's cash balance and cash runway; the Company's ability to attract and/or retain new and existing collaborators with relevant expertise and its expectations regarding the potential benefits to be derived from any such collaborations; and the Company's plans and strategy with respect to developing its technologies and product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon the Company's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the Company's reliance on third parties for various aspects of its business; risks and uncertainties associated with development and regulatory approval of novel product candidates in the biopharmaceutical industry; the Company's ability to retain key scientific or management personnel; the fact that the Company will have limited control over the efforts and resources that Roche devotes to advancing development programs under its collaboration agreement and the Company may not receive the potential fees and payments under the collaboration agreement and the ability of Roche to early terminate the collaboration, such that the Company may not fully realize the benefits of the collaboration; and the other risks described in the Company's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
Poseida Therapeutics, Inc. Selected Financial Data (In thousands, except share and per share amounts)
STATEMENTS OF OPERATIONS | ||||||||||||||||
Three Months Ended | Twelve Months Ended | |||||||||||||||
2023 | 2022 | 2023 | 2022 | |||||||||||||
(Unaudited) | ||||||||||||||||
Revenues: | ||||||||||||||||
Collaboration revenue | $ | 24,995 | $ | 10,051 | $ | 64,703 | $ | 130,492 | ||||||||
Total revenue | 24,995 | 10,051 | 64,703 | 130,492 | ||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | 42,045 | 33,904 | 156,772 | 152,899 | ||||||||||||
General and administrative | 8,859 | 9,368 | 37,435 | 37,539 | ||||||||||||
Total operating expenses | 50,904 | 43,272 | 194,207 | 190,438 | ||||||||||||
Loss from operations | (25,909) | (33,221) | (129,504) | (59,946) | ||||||||||||
Other income (expense): | ||||||||||||||||
Interest expense | (2,267) | (1,975) | (8,671) | (6,370) | ||||||||||||
Other income, net | 2,827 | 2,170 | 14,852 | 2,858 | ||||||||||||
Net loss before income tax | (25,349) | (33,026) | (123,323) | (63,458) | ||||||||||||
Income tax expense | — | (292) | (107) | (544) | ||||||||||||
Net loss | $ | (25,349) | $ | (33,318) | $ | (123,430) | $ | (64,002) | ||||||||
Net loss per share, basic and diluted | $ | (0.27) | $ | (0.39) | $ | (1.37) | $ | (0.89) | ||||||||
Weighted-average number of shares outstanding, basic and diluted | 95,623,219 | 85,953,375 | 90,185,096 | 71,953,703 | ||||||||||||
SELECTED BALANCE SHEET DATA | ||||||||
December 31, | ||||||||
2023 | 2022 | |||||||
Cash, cash equivalents and short-term investments | $ | 212,202 | $ | 282,493 | ||||
Total assets | 273,885 | 351,837 | ||||||
Total liabilities | 170,184 | 164,242 | ||||||
Total stockholders' equity | 103,701 | 187,595 | ||||||
View original content to download multimedia:https://www.prnewswire.com/news-releases/poseida-therapeutics-provides-updates-and-financial-results-for-the-fourth-quarter-and-full-year-2023-302083497.html
SOURCE Poseida Therapeutics, Inc.
FAQ
What was the ORR in the Phase 1 trial for P-BCMA-ALLO1?
What is the ticker symbol for Poseida Therapeutics?
What was the net loss for Poseida Therapeutics in Q4 2023?
How much cash does Poseida Therapeutics have as of December 31, 2023?
What is the revenue for Poseida Therapeutics in Q4 2023?
What new program did Poseida Therapeutics initiate a Phase 1 trial for?
