Poseida Therapeutics(PSTX) - 2023 Q4 - Annual Results

Executive Summary Poseida Therapeutics reported Q4 and FY2023 results, highlighting positive P-BCMA-ALLO1 Phase 1 data, P-CD19CD20-ALLO1 trial initiation, and gene therapy progress, with 2024 poised for key data readouts Summary of Q4 and FY2023 Highlights Poseida Therapeutics reported Q4 and FY2023 results, highlighting positive P-BCMA-ALLO1 Phase 1 data, P-CD19CD20-ALLO1 trial initiation, and gene therapy progress, with 2024 poised for key data readouts - Lead asset P-BCMA-ALLO1 Phase 1 clinical trial data presented at ASH 2023 demonstrated 82% ORR and a favorable emerging safety and reliability profile[1] - Initiated clinical trial of Phase 1 P-CD19CD20-ALLO1, the company's first allogeneic dual CAR-T program[1] - P-FVIII-101 gene therapy data presented at ASH, with a Gene Therapy R&D Day planned for April 17, 2024[1] - CEO Kristin Yarema stated that '2024 looks to be a breakout year for Poseida' with planned data readouts for three clinical-stage CAR-T programs[2] Recent Business Accomplishments (2023) Poseida Therapeutics achieved significant milestones in 2023, advancing its cell and gene therapy programs with positive clinical data and strategic partnerships Cell Therapy Program Updates In 2023, Poseida presented positive Phase 1 P-BCMA-ALLO1 data, continued P-MUC1C-ALLO1 enrollment, and advanced Roche-partnered hematologic malignancy programs, including P-CD19CD20-ALLO1 initiation - Presented positive data from the Phase 1 study of P-BCMA-ALLO1 in relapsed/refractory multiple myeloma (RRMM) at ASH in December 2023[3] - Continued enrollment in the Phase 1 clinical trial of P-MUC1C-ALLO1 for solid tumors, exploring various dosing approaches including higher lymphodepletion[5] - Advanced programs within hematologic malignancies in partnership with Roche, with expected acceleration of milestones resulting in a $30 million payment in Q1 2024[5] P-BCMA-ALLO1 (Multiple Myeloma) The P-BCMA-ALLO1 Phase 1 trial demonstrated high overall response rates and a favorable safety profile in heavily pretreated multiple myeloma patients - 82% Overall Response Rate (ORR) in heavily pretreated patients receiving adequate lymphodepletion, including sCRs and MRD-negative patients[5] - 100% ORR in patients not previously treated with a BCMA-targeted bispecific T-cell engaging antibody[5] - Favorable emerging safety and reliability profile, with low incidences of CRS and neurotoxicity observed (all ≤ Grade 2)[5] P-MUC1C-ALLO1 (Solid Tumors) Enrollment continued for P-MUC1C-ALLO1 in solid tumors, with exploration of higher lymphodepletion and cell doses based on allogeneic BCMA CAR-T program insights - Continued enrollment in Phase 1 clinical trial for solid tumors, exploring higher lymphodepletion, cell dose, and scheduling based on insights from allogeneic BCMA CAR-T program[5] - Assessing lymphodepleting conditioning regimens with cyclophosphamide doses exceeding 300 mg/m² in the MUC1C program, following observations of significantly elevated P-BCMA-ALLO1 levels with higher doses[5] Roche Partnership Programs Roche partnership programs advanced with an accelerated $30 million milestone payment, expanded P-BCMA-ALLO1 enrollment, and initiated P-CD19CD20-ALLO1 for B-cell malignancies - Expected acceleration of achieving milestones resulted in the receipt of a $30 million payment in the first quarter of 2024[5] - Expanded the protocol and continued to enroll patients in the Phase 1 study of P-BCMA-ALLO1, incorporating lymphodepletion learnings from ASH 2023[5] - Continued site activation activities and initiation of enrollment for P-CD19CD20-ALLO1, the company's first dual, allogeneic CAR-T for B-cell malignancies[5] Gene Therapy Program Updates Preclinical data for P-FVIII-101 supported its potential for Hemophilia A, demonstrating stable Factor VIII expression, re-dosing capabilities, and favorable tolerability with 52-week durability - Presented proof-of-principle data supporting P-FVIII-101 as a potential treatment option for Hemophilia A at ASH[4] - Data demonstrated stable Factor VIII (FVIII) transgene expression through genomic integration using the company's non-viral approach[4] - Highlighted re-dosing, or titrating to efficacy, along with favorable tolerability data, including 52-week durability data in an adult Hemophilia A mouse model[4] Operational Updates & Upcoming Events Poseida plans multiple data presentations and R&D Days in 2024, alongside strategic expansion into autoimmune disease and key leadership changes Anticipated Program Milestones Poseida anticipates multiple 2024 data presentations for its CAR-T programs, including P-BCMA-ALLO1 and P-MUC1C-ALLO1 at AACR, an interim P-CD19CD20-ALLO1 update, and advancing P-PSMA-ALLO1 into IND-enabling studies - P-BCMA-ALLO1: Data presentation planned at AACR on April 8, 2024, for patients refractory to initial BCMA targeting therapy, with an additional clinical update in H2 2024[10] - P-MUC1C-ALLO1: Initial clinical findings from Phase 1 trial to be reviewed at AACR on April 8, 2024, with a more comprehensive clinical update in H2 2024[10] - P-CD19CD20-ALLO1: Interim data update expected in H2 2024, following Phase 1 trial initiation in late 2023[10] - P-PSMA-ALLO1: Next allogeneic solid tumor program in prostate cancer expected to advance into IND-enabling studies in 2024[10] R&D Days Poseida plans two R&D Days in 2024: a gene therapy focused event on April 17, highlighting non-viral gene technologies, and a cell therapy focused event in the second half - Gene therapy specific R&D Day planned for April 17, 2024, featuring presentations on proprietary non-viral gene insertion and editing technologies, portfolio review, and new preclinical data[9] - Cell therapy focused R&D Day planned for the second half of 2024[9] Strategic Expansion into Autoimmune Disease Poseida is actively developing a strategy to apply its TSCM-rich CAR-T platform and proprietary technologies to autoimmune and inflammatory diseases, with updates to follow - The company believes its TSCM-rich CAR-T platform and associated proprietary technologies have strong potential to deliver new therapeutic approaches in autoimmune disease[7][11] - The company is actively developing a strategy in inflammation and autoimmune disease and will provide an update at an appropriate time[7][11] Leadership Updates Poseida announced key leadership changes, including Alexander Chapman as SVP, IR & Corporate Communications, Loren Wagner's promotion to COO, and Brent Warner's departure as President, Gene Therapy - Alexander Chapman joined Poseida on February 26, 2024, as Senior Vice President, Investor Relations & Corporate Communications[12] - Loren Wagner was promoted to Chief Operations Officer in February 2024[13] - Brent Warner, President, Gene Therapy, will depart Poseida effective April 1, 2024, to pursue an external career opportunity[14] Financial Results for Fourth Quarter and Full Year 2023 This section details Poseida's financial performance for Q4 and full year 2023, covering revenues, operating expenses, net loss, and cash position Revenues Q4 2023 revenues significantly increased due to a Roche milestone, while full-year 2023 revenues decreased primarily due to initial Roche license revenue recognized in 2022 Revenue Performance | Metric | Q4 2023 (Millions) | Q4 2022 (Millions) | YoY Change | FY 2023 (Millions) | FY 2022 (Millions) | YoY Change | | :---------------- | :----------------- | :----------------- | :--------- | :----------------- | :----------------- | :--------- | | Collaboration Revenue | $25.0 | $10.1 | +147.5% | $64.7 | $130.5 | -50.5% | - Q4 2023 revenue increase primarily due to a milestone achieved under the Roche collaboration and increased activity, offset by a decrease related to Takeda due to its collaboration termination[15] - Full-year 2023 revenue decrease primarily due to initial license revenue recognized from the Roche collaboration in Q3 2022, partially offset by research services revenue from Roche and Takeda (including $8.9 million of previously deferred revenue from Takeda termination)[16] Operating Expenses R&D expenses increased in Q4 and FY2023 due to preclinical and allogeneic clinical programs, partially offset by autologous program wind-down, while G&A expenses slightly decreased due to lower insurance costs Research and Development Expenses R&D expenses increased in Q4 and FY2023, driven by preclinical programs, increased headcount, and allogeneic clinical programs, partially offset by autologous program wind-down Research and Development Expenses | Metric | Q4 2023 (Millions) | Q4 2022 (Millions) | YoY Change | FY 2023 (Millions) | FY 2022 (Millions) | YoY Change | | :----------------------- | :----------------- | :----------------- | :--------- | :----------------- | :----------------- | :--------- | | Research and Development | $42.0 | $33.9 | +23.9% | $156.8 | $152.9 | +2.6% | - Increase primarily due to increased preclinical stage programs, research collaboration activity, personnel expenses (increased headcount), and clinical stage allogeneic programs[17][18] - Partially offset by a decrease in clinical stage autologous programs due to the wind-down of development activities[17][18] General and Administrative Expenses G&A expenses decreased in Q4 and FY2023 primarily due to lower insurance costs, with the full-year decrease partially offset by increased personnel costs General and Administrative Expenses | Metric | Q4 2023 (Millions) | Q4 2022 (Millions) | YoY Change | FY 2023 (Millions) | FY 2022 (Millions) | YoY Change | | :-------------------------- | :----------------- | :----------------- | :--------- | :----------------- | :----------------- | :--------- | | General and Administrative | $8.9 | $9.4 | -5.3% | $37.4 | $37.5 | -0.3% | - Decrease primarily due to lower insurance costs in both Q4 and full year 2023[19][20] - Full-year decrease was partially offset by an increase in personnel costs due to accelerated stock-based compensation expense related to the retirement of the company's former Executive Chairman in Q1 2023[20] Net Loss Poseida reported a reduced net loss in Q4 2023 but a significantly increased net loss for the full year, primarily driven by changes in revenue and operating expenses Net Loss and EPS | Metric | Q4 2023 (Millions) | Q4 2022 (Millions) | YoY Change | FY 2023 (Millions) | FY 2022 (Millions) | YoY Change | | :------- | :----------------- | :----------------- | :--------- | :----------------- | :----------------- | :--------- | | Net Loss | $(25.3) | $(33.3) | +23.9% | $(123.4) | $(64.0) | -92.8% | | Net Loss Per Share, Basic and Diluted | $(0.27) | $(0.39) | +30.8% | $(1.37) | $(0.89) | -53.9% | Weighted-Average Shares Outstanding | Metric | Q4 2023 | Q4 2022 | YoY Change | FY 2023 | FY 2022 | YoY Change | | :------------------------------------------------ | :---------- | :---------- | :--------- | :---------- | :---------- | :--------- | | Weighted-average number of shares outstanding, basic and diluted | 95,623,219 | 85,953,375 | +11.2% | 90,185,096 | 71,953,703 | +25.3% | Cash Position As of December 31, 2023, Poseida held $212.2 million in cash, projecting this, with anticipated Roche payments, to fund operations into H2 2025, with potential for further extension Cash Position | Metric | December 31, 2023 (Millions) | December 31, 2022 (Millions) | | :-------------------------------------- | :--------------------------- | :--------------------------- | | Cash, cash equivalents and short-term investments | $212.2 | $282.5 | - The company expects its cash, cash equivalents, and short-term investments, along with remaining near-term milestones and other payments from Roche, to fund operations into the second half of 2025[22] - Potential additional payments under the amended Roche collaboration and license agreement or additional business development could further extend cash runway[22] Company Information & Disclosures This section provides an overview of Poseida Therapeutics, its forward-looking statements, and detailed selected financial data About Poseida Therapeutics, Inc. Poseida Therapeutics is a clinical-stage biopharmaceutical company developing differentiated cell and gene therapies for cancer and rare diseases, leveraging proprietary genetic editing platforms and in-house GMP manufacturing - Poseida Therapeutics is a clinical-stage biopharmaceutical company advancing differentiated cell and gene therapies with the capacity to cure certain cancers and rare diseases[23] - 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[23] - Its approach is based on proprietary genetic editing platforms, including non-viral piggyBac® DNA Delivery System, Cas-CLOVER™ Site-Specific Gene Editing System, Booster Molecule, and nanoparticle/hybrid gene delivery technologies, along with in-house GMP cell therapy manufacturing[23] Forward-Looking Statements This section contains standard cautionary language regarding forward-looking statements, emphasizing that actual results may differ materially due to inherent biopharmaceutical industry risks and uncertainties - Statements regarding matters that are not historical facts are 'forward-looking statements' subject to risks and uncertainties[24][25] - Risks include reliance on third parties, uncertainties with development and regulatory approval, ability to retain key personnel, limited control over Roche's efforts, and other risks described in SEC filings[25] - The company undertakes no obligation to update such statements to reflect events or circumstances after the date they were made, except as required by law[25] Selected Financial Data This section provides detailed financial tables for the three and twelve months ended December 31, 2023 and 2022, covering Statements of Operations and Selected Balance Sheet Data Statements of Operations (in thousands) | | Three Months Ended Dec 31, 2023 | Three Months Ended Dec 31, 2022 | Twelve Months Ended Dec 31, 2023 | Twelve Months Ended Dec 31, 2022 | | :-------------------------- | :------------------------------ | :------------------------------ | :------------------------------- | :------------------------------- | | Collaboration revenue | $24,995 | $10,051 | $64,703 | $130,492 | | Total revenue | $24,995 | $10,051 | $64,703 | $130,492 | | 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) | | 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 (in thousands) | | December 31, 2023 | December 31, 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 |