Hemophilia

Data from novel proprietary neurotropic adeno-associated virus (AAV) delivery capsid, STAC-BBB, demonstrated industry-leading blood-brain barrier (BBB) penetration in non-human primates (NHPs) following intravenous administration, with capsid-enabled delivery of zinc finger payloads targeting prion disease and tauopathies resulting in potent and widespread repression of target genes. Chronic neuropathic pain and prion disease preclinical programs advance, with investigational new drug (IND) and clinical trial authorization (CTA) submissions expected in the fourth quarters of 2024 and 2025, respectively. Announced U.S. Food and Drug Administration (FDA) alignment on an abbreviated pathway to potential approval, and grant by European Medicines Agency (EMA) of priority medicines (PRIME) eligibility for isaralgagene civaparvovec in Fabry disease. Pfizer anticipates Biologics License Application (BLA) and marketing authorization application (MAA) submissions for Hemophilia A collaboration by early 2025 if the pivotal readout is supportive. RICHMOND, Calif.--(BUSINESS WIRE)-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline. “In 2023, Sangamo announced the prioritization of its pipeline programs that support our focus as a neurology-focused genomic medicine company,” said Sandy Macrae, Chief Executive Officer of Sangamo. “With the meaningful preclinical data announced today, we believe our ability to combine potent zinc finger epigenetic regulation payloads with exciting new industry-leading capsid delivery technology could unlock significant potential for the treatment of devastating neurological diseases, indications for which delivery of treatments to the central nervous system has historically proved challenging. In the near-term, we are also seeking to create value by partnering our Fabry disease program, for which we aligned with the FDA on a potentially abbreviated and more cost-effective timeline and received EMA PRIME eligibility. We look forward to advancing our pipeline into the clinic to develop therapies designed to target neurological diseases with high unmet medical needs.” Recent Business Highlights Prioritized Neurology Pipeline Sangamo is developing epigenetic regulation therapies to treat serious neurological diseases and novel proprietary AAV capsids designed to deliver our therapies to the intended neurological targets, including across the blood-brain barrier. Novel AAV Capsid Delivery Technology – Data demonstrated industry-leading BBB penetration and brain transduction in NHPs, with capsid-enabled delivery of zinc finger payloads targeting prion disease and tauopathies resulting in potent and widespread repression of target genes. Novel STAC-BBB capsid demonstrated robust penetration of BBB with 700-fold higher transgene expression in neurons compared to the benchmark capsid AAV9 and outperformed all other known published capsid variants evaluated in the study. STAC-BBB mediated robust expression of zinc finger cargo in neurons, with potent and widespread repression of prion and tau genes observed across key brain regions, demonstrating the potential for modification of disease progression in prion disease and various tauopathies. Visualization of gene expression in individual brain cells by RNAscope revealed highly potent repression of tau in neurons expressing the zinc finger cargo, across multiple brain regions. Capsid biodistribution pro optimal for the treatment of neurological diseases with AAV-based treatments, highlighted by the observed enrichment in the CNS and de-targeting from the liver, dorsal root ganglia (DRG) and other peripheral organs. STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues. We believe that STAC-BBB is manufacturable at commercial scale using standard cell culture and purification processes, is soluble using known excipients, and can be characterized using available analytics. Chronic Neuropathic Pain – Preclinical data demonstrated potent and specific repression of Nav1.7 expression without impacting other sodium channels and that zinc finger repressors (ZFRs) were well tolerated in NHPs; IND submission expected in fourth quarter of 2024. IND-enabling toxicology studies are nearing completion. An IND submission is expected in the fourth quarter of 2024. Prion Disease – Preclinical data demonstrated that AAV-delivered ZFRs significantly reduced expression of the prion protein in the brain, extended lifespan and limited formation of toxic prion aggregates in mice; CTA expected in the fourth quarter of 2025. CTA-enabling studies continue to advance. Prion-targeted zinc finger repressor, delivered via an intravenous administration of the STAC-BBB novel capsid, resulted in a dose-dependent repression of prion genes in NHPs. A CTA submission is expected in the fourth quarter of 2025. Tauopathies – Preclinical data demonstrated that AAV-delivered ZFRs significantly reduced expression of tau mRNA in brain of NHPs; intend to resume development of this program, leveraging STAC-BBB; IND submission expected as early as the fourth quarter of 2025. Intend to resume development of our previously paused tau program, leveraging the newly identified STAC-BBB capsid variant, subject to additional funding. Tau clinical-lead zinc finger repressor, delivered via an intravenous administration of the STAC-BBB novel capsid, resulted in a dose-dependent repression of tau genes in NHPs. Visualization of gene expression in individual brain cells by RNAscope revealed highly potent repression of tau in neurons expressing the zinc finger cargo across multiple brain regions. We expect the IND submission could occur as early as the fourth quarter of 2025. Other Programs Fabry Disease – Dosed total of 32 patients in Phase 1/2 STAAR study, with updated clinical data presented at 20th Annual WORLDSymposium showing sustained benefit and differentiated safety pro >; announced FDA alignment on abbreviated pathway to potential approval; actively seeking collaboration partner to advance asset toward potential registration and commercialization. Dosed seven additional patients in the dose expansion phase of the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, our wholly owned gene therapy product for the treatment of Fabry disease, for a total of 32 patients dosed to date. Presented updated clinical data at the 20th Annual WORLDSymposium in San Diego, CA in February 2024, showing that elevated levels of α-Gal A activity were sustained in all 24 patients evaluated as of the September 19, 2023 data cutoff date and accompanied by the reduction and/or long-term stabilization of lyso-Gb3 levels, with the largest reductions in plasma lyso-Gb3 seen in patients with the highest levels at baseline. All 13 patients who were withdrawn from Enzyme Replacement Therapy (ERT) remain off ERT as of March 12, 2024. In the 13 patients followed for 12-months or more after treatment, renal function remained stable, and significant improvements in overall disease severity, quality of life, and gastrointestinal symptoms compared to baseline were reported. Aligned with the FDA that data from a single, adequate, and well-controlled study may form the primary basis of approval of a BLA for isaralgagene civaparvovec, enabling a potentially abbreviated and more cost-effective pathway to BLA submission than originally anticipated. The study would enroll up to 25 patients, both male and female, without the need for a control arm. A head-to-head comparison with ERT is not part of the proposed study design deemed acceptable by the FDA. Granted PRIME eligibility from the EMA for isaralgagene civaparvovec, which aims to enhance support for the development of medicines that target an unmet medical need and is intended to optimize development plans and expedite review and approval processes. Granted Innovative Licensing and Access Pathway (ILAP) for isaralgagene civaparvovec from U.K. Medicines and Healthcare products Regulatory Agency (MHRA) which aims to accelerate time to market and facilitate access to medicines. Isaralgagene civaparvovec has already received Orphan Medicinal Product designation from the EMA as well as Orphan Drug, Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. Completed screening and enrollment in the Phase 1/2 STAAR study and expect to complete dosing of remaining patients in the first half of 2024. Deferring additional investments in planning for a potential registrational trial until a collaboration partnership or financing for this program is secured. Hemophilia A (Pfizer) – Pivotal data read-out in Phase 3 AFFINE trial expected in mid-2024; BLA and MAA submissions anticipated by early 2025 if pivotal readout is supportive. A pivotal readout is expected in mid-2024 in the Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia A. Pfizer anticipates BLA and MAA submissions by early 2025 if the pivotal readout is supportive. Presented updated data with Pfizer from the Phase 1/2 ALTA study of giroctocogene fitelparvovec in an oral presentation at the 65th American Society for Hematology Annual Meeting and Exposition in December 2023. Eligible to earn from Pfizer up to $220.0 million in milestone payments upon the achievement of certain regulatory and commercial milestones for giroctocogene fitelparvovec and product sales royalties of 14% - 20% if giroctocogene fitelparvovec is approved and commercialized, subject to certain reductions. CAR-Tregs – In alignment with previously announced strategic transformation, announced winddown of the Company’s French research and manufacturing operations and a corresponding reduction in workforce; continuing to seek a potential collaboration partner or external investment in the CAR-Treg cell therapy programs. Announced a winddown of Sangamo’s French operations and a corresponding reduction in workforce, including closure of Sangamo’s cell therapy manufacturing facility and research labs in Valbonne, France, which is expected to commence in April 2024 and be complete by end of year. We expect this restructuring to result in the elimination of all roles in France (approximately 93). Dosed two additional patients in the Phase 1/2 STEADFAST study evaluating TX200, our wholly owned autologous CAR-Treg cell therapy treating patients receiving an HLA-A2 mismatched kidney from a living donor, including the first patient in the new fourth, highest dose cohort. Expect to dose up to two additional patients in the fourth highest-dose level cohort by the end of the second quarter of 2024. Plan to continue seeking a potential collaboration partner or external investment in our autologous CAR-Treg cell therapy programs. Fourth Quarter and Full Year 2023 Financial Results Consolidated net loss for the fourth quarter ended December 31, 2023 was $60.3 million, or $0.34 per share, compared to a net loss of $52.0 million, or $0.32 per share, for the same period in 2022. For the year ended December 31, 2023, consolidated net loss was $257.8 million, or $1.48 per share, compared to consolidated net loss of $192.3 million, or $1.25 per share, for the year ended December 31, 2022. Revenues Revenues for the fourth quarter ended December 31, 2023 were $2.0 million, compared to $27.2 million for the same period in 2022. The decrease of $25.2 million in revenues was primarily attributed to a decrease of $17.5 million in revenue relating to our collaboration agreement with Kite, reflecting a reduction in level of our research and development services, and a decrease of $10.3 million in revenue relating to our collaboration agreement with Novartis, due to the impact of termination of the collaboration agreement. These decreases were partially offset by an increase of $2.6 million in revenues from other licensing agreements. Revenues were $176.2 million in 2023, compared to $111.3 million in 2022. The increase of $64.9 million in revenues in 2023 compared to 2022, was primarily attributed to an increase of $106.4 million in revenue relating to our collaboration agreement with Biogen, primarily due to the impact of termination of the collaboration agreement in June 2023, which resulted in an increase in the measure of proportional cumulative performance on this collaboration, an increase of $3.8 million in revenue relating to our license agreement with Sigma-Aldrich Corporation, and an increase of $3.6 million in revenues from other licensing agreements. These increases were partially offset by a decrease of $27.5 million in revenue relating to our collaboration agreement with Novartis due to the termination of the collaboration agreement in June 2023, a decrease of $18.1 million in revenue relating to our collaboration agreement with Kite due to a reduction in the estimated future level of our research and development services related to this collaboration, and a decrease of $3.3 million in revenue relating to our collaboration agreement with Sanofi due to the termination of the collaboration agreement in June 2022. GAAP and Non-GAAP Operating Expenses Three Months Ended Year Ended December 31, December 31, (In millions) 2023 2022 2023 2022 Research and development $ 50.7 $ 66.2 $ 234.0 $ 249.9 General and administrative 13.1 16.4 61.2 62.7 Impairment of goodwill and indefinite-lived intangible assets - - 89.5 - Impairment of long-lived assets 0.3 - 65.5 - Total operating expenses 64.1 82.6 450.2 312.6 Impairment of goodwill and indefinite-lived intangible assets - - (89.5 ) - Impairment of long-lived assets (0.3 ) - (65.5 ) - Depreciation and amortization (1.8 ) (3.3 ) (15.1 ) (12.1 ) Stock-based compensation expense (6.1 ) (8.3 ) (27.4 ) (31.7 ) Non-GAAP operating expenses $ 55.9 $ 71.0 $ 252.7 $ 268.8 Total operating expenses on a GAAP basis for the fourth quarter ended December 31, 2023 were $64.1 million compared to $82.6 million for the same period in 2022. Non-GAAP operating expenses, which exclude impairment charges, depreciation and amortization, and stock-based compensation expense, for the fourth quarter ended December 31, 2023 were $55.9 million, compared to $71.0 million for the same period in 2022. The decrease in total operating expenses on a GAAP and non-GAAP basis was primarily attributable to lower compensation and other personnel costs mainly due to a reduction in the bonus expense and lower headcount as a result of restructuring of operations and a corresponding reduction in workforce announced during the year, and a decrease in research and clinical expenses due to deferral and reprioritization of certain programs. These decreases were partially offset by restructuring charges related to the reductions in workforce announced in April and November 2023, and the France restructuring. The expense related to the France restructuring was recorded in the fourth quarter of 2023 as the payouts are based an ongoing post-employment benefit plan, and the payments were probable and could be estimated as of December 31, 2023. Total operating expenses on a GAAP basis in 2023 were $450.2 million compared to $312.6 million in 2022. Non-GAAP operating expenses, which exclude impairment charges, depreciation and amortization, and stock-based compensation expense, were $252.7 million in 2023 compared to $268.8 million in 2022. Operating expenses on a GAAP basis included non-cash charges relating to impairment of goodwill and indefinite-lived intangible assets of $89.5 million, and impairment of long-lived assets of $65.5 million, which were a result of continued decline in our stock price and related market capitalization and termination of our collaboration agreements with Biogen and Novartis. The decrease in total operating expenses on a non-GAAP basis was primarily attributable to a reduction in the bonus expense and lower headcount as a result of restructurings of operations and corresponding reductions in workforce announced during the year, and a decrease in preclinical and clinical expenses due to the termination of collaboration agreements with Biogen and Novartis and deferral and reprioritization of certain programs. These decreases were partially offset by restructuring charges related to the reductions in workforce announced in April and November 2023, and the France restructuring. Cash, Cash Equivalents and Marketable Securities Cash, cash equivalents and marketable securities as of December 31, 2023 were $81.0 million, compared to $307.5 million as of December 31, 2022. During the year ended December 31, 2023, we raised approximately $15.1 million in net proceeds under our at-the-market offering program. We believe that our available cash, cash equivalents and marketable securities as of December 31, 2023, in combination with potential future cost reductions, will be sufficient to fund our planned operations into the third quarter of 2024, excluding any additional capital raised. We are actively pursuing opportunities to raise additional capital. Financial Guidance for 2024 On a GAAP basis, we expect total operating expenses in the range of approximately $145 million to $165 million in 2024, which includes non-cash stock-based compensation expense and depreciation and amortization, subject to additional funding. We expect non-GAAP total operating expenses, excluding estimated non-cash stock-based compensation expense of approximately $13 million, and depreciation and amortization of approximately $7 million, in the range of approximately $125 million to $145 million in 2024, subject to additional funding. Upcoming Events Sangamo plans to participate in the following events: Investor Conferences RBC Capital Markets Global Healthcare Conference, May 14-15, 2024 Jefferies Global Healthcare Conference, June 5-6, 2024 H.C. Wainwright 5th Annual Neuro Perspectives Virtual Conference, June 27, 2024 Access links for available webcasts for these investor conferences will be available on the Sangamo website in the Investors and Media section under Events. Available materials will be found on the Sangamo website after the event under Presentations. Conference Call The Sangamo management team will hold a corporate call to further discuss program advancements and financial updates on Wednesday, March 13 at 4:30pm Eastern Time. Participants should register for, and access, the call using this link. While not required, it is recommended you join 10 minutes prior to the event start. Once registered, participants will be given the option to either dial into the call with the number and unique passcode provided or to use the dial-out option to connect their phone instantly. An updated corporate presentation is available in the Investors and Media section under Presentations. The link to access the live webcast can also be found on the Sangamo website in the Investors and Media section under Events. A replay will be available following the conference call, accessible at the same link. About Sangamo Therapeutics Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo’s zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders and Sangamo’s capsid discovery platform is expanding delivery beyond currently available intrathecal delivery capsids, including in the central nervous system. Sangamo’s pipeline also includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit and connect with us on LinkedIn and Twitter/X. Forward-Looking Statements This press release contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to: the therapeutic and commercial potential of Sangamo’s product candidates and its engineered capsids, including the ability of STAC-BBB to unlock potential for the treatment of various neurological diseases, the anticipated plans and timelines of Sangamo and its collaborators dosing patients in and conducting our ongoing and potential future clinical trials and presenting clinical data from our clinical trials, including expectations regarding the conclusion of dosing in our Phase 1/2 STAAR study, plans for patient dosing in the STEADFAST study, the anticipated advancement of Sangamo’s product candidates to late-stage development, including plans to seek a potential partner or additional financing to proceed with potential future Phase 3 trials of isaralgagene civaparvovec and the design and timing thereof, the timeline to present data from the Phase 3 AFFINE trial and to make BLA and MAA submissions for giroctocogene fitelparvovec, the potential to earn milestone payments and receive product sales royalties if giroctocogene fitelparvovec is approved and commercialized, expectations regarding advancement of Sangamo’s preclinical neurology programs, including announcement of data from, and anticipated IND and CTA submissions related to, such programs, plans to seek a partner for or investor in Sangamo’s CAR-Treg program, expectations concerning Sangamo’s announced winddown of our French research and manufacturing operations and a corresponding reduction in workforce and the expected charges and cost savings associated with such restructuring; future potential cost reductions, Sangamo’s expected cash runway, Sangamo’s 2024 financial guidance related to GAAP and non-GAAP total operating expenses, impairments and stock-based compensation, plans to participate in industry and investor conferences, efforts to secure additional funding, and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to Sangamo’s lack of capital resources to fully develop, obtain regulatory approval for and commercialize its product candidates, including the ability to secure the funding or partnerships required to advance its preclinical and clinical programs; Sangamo’s ability to execute its restructurings as currently contemplated; the actual charges associated with the restructurings being higher than anticipated or changes to the assumptions on which the estimated charges associated with the restructurings are based; Sangamo’s ability to achieve projected cost savings in connection with the restructurings and to further reduce operating expenses; unintended consequences from the restructuring that impact Sangamo’s business; Sangamo’s need for substantial additional funding to execute our operating plan and to continue to operate as a going concern, including the risk that Sangamo will be unable to obtain the funding necessary to advance its preclinical and clinical programs and to otherwise operate as a going concern, in which case Sangamo may be required to cease operations entirely, liquidate all or a portion of its assets and/or seek protection under the U.S. Bankruptcy Code; the uncertain and costly research and development process, including the risk that preclinical results may not be indicative of results in any future clinical trials; the effects of macroeconomic factors or financial challenges, including as a result of the ongoing overseas conflict, current or potential future bank failures, inflation and rising interest rates, on the global business environment, healthcare systems and business and operations of Sangamo and its collaborators, including the initiation and operation of clinical trials; the impacts of clinical trial delays, pauses and holds on clinical trial timelines and commercialization of product candidates; the uncertain timing and unpredictable nature of clinical trial results, including the risk that therapeutic effects in the Phase 3 AFFINE trial will not be durable in patients as well as the risk that the therapeutic effects observed in the latest preliminary clinical data from the Phase 1/2 STAAR study will not be durable in patients and that final clinical trial data from the study will not validate the safety and efficacy of isaralgagene civaparvovec, and that the patients withdrawn from ERT will remain off ERT; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; reliance on results of early clinical trials, which results are not necessarily predictive of future clinical trial results, including the results of any registrational trial of Sangamo’s product candidates; the potential for technological developments that obviate technologies used by Sangamo; Sangamo’s reliance on collaborators and its potential inability to secure additional collaborations, and Sangamo’s ability to achieve expected future operating results. All forward-looking statements about our future plans and expectations, including our financial guidance, are subject to our ability to secure adequate additional funding. There can be no assurance that Sangamo and its collaborators will be able to develop commercially viable products. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and its collaborators. These risks and uncertainties are described more fully in Sangamo’s Securities and Exchange Commission, or SEC, filings and reports, including in Sangamo’s Annual Report on Form 10-K for the year ended December 31, 2023, and subsequent filings and reports that Sangamo makes from time to time with the SEC. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law. Non-GAAP Financial Measures To supplement our financial results and guidance presented in accordance with GAAP, we present non-GAAP operating expenses, which excludes depreciation and amortization, stock-based compensation expense and impairment of goodwill, indefinite-lived intangible assets and long-lived assets from GAAP operating expenses. We believe that this non-GAAP financial measure, when considered together with our financial information prepared in accordance with GAAP, can enhance investors’ and analysts’ ability to meaningfully compare our results from period to period and to our forward-looking guidance, and to identify operating trends in our business. We have excluded depreciation and amortization, and stock-based compensation expense because they are non-cash expenses that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented, and we have excluded impairment of goodwill, indefinite-lived intangible assets and long-lived assets to facilitate a more meaningful evaluation of our current operating performance and comparisons to our operating performance in other periods. This non-GAAP financial measure is in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. We encourage investors to carefully consider our results under GAAP, as well as our supplemental non-GAAP financial information, to more fully understand our business. SELECTED CONSOLIDATED FINANCIAL DATA (unaudited; in thousands, except per share data) Statement of Operations Data: Three Months Ended Year Ended December 31, December 31, 2023 2022 2023 2022 Revenues $ 2,042 $ 27,230 $ 176,232 $ 111,299 Operating expenses: Research and development 50,706 66,179 234,057 249,898 General and administrative 13,099 16,443 61,167 62,682 Impairment of goodwill and indefinite-lived intangible assets - - 89,485 - Impairment of long-lived assets 296 - 65,528 - Total operating expenses 64,101 82,622 450,237 312,580 Loss from operations (62,059 ) (55,392 ) (274,005 ) (201,281 ) Interest and other income, net 1,491 3,678 11,102 9,432 Loss before income taxes (60,568 ) (51,714 ) (262,903 ) (191,849 ) Income tax (benefit) expense (272 ) 259 (5,072 ) 429 Net loss $ (60,296 ) $ (51,973 ) $ (257,831 ) $ (192,278 ) Basic and diluted net loss per share $ (0.34 ) $ (0.32 ) $ (1.48 ) $ (1.25 ) Shares used in computing basic and diluted net loss per share 177,619 164,717 174,444 154,345 Selected Balance Sheet Data: December 31, 2023 December 31, 2022 Cash, cash equivalents, and marketable securities $ 81,002 $ 307,477 Total assets $ 165,320 $ 562,509 Total stockholders' equity $ 82,887 $ 294,958

Lead asset P-BCMA-ALLO1 Phase 1 clinical trial data presented at ASH 2023 demonstrated 82% ORR and favorable emerging safety and reliability pro > 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 SAN DIEGO, March 7, 2024 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, today announced business updates and financial results for the fourth quarter and full year ended December 31, 2023. "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 pro 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 payment in the first quarter of 2024. 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. 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 U.S. leadership roles at Amgen, including Corporate Affairs, Value, Access & Pricing, and U.S. Business Operations. 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 $25.0 million for the fourth quarter ended December 31, 2023, compared to $10.1 million for the same period in 2022. The increase was primarily due to revenue earned as a result of a milestone achieved in the fourth quarter under the amendment to the collaboration and license agreement with Roche and increased activity under the collaboration, offset by a decrease in revenue related to Takeda due to the termination of its collaboration agreement in the third quarter of 2023. For the full year ended December 31, 2023, revenues were $64.7 million, compared to $130.5 million for the same period in 2022. The decrease was primarily due to initial license revenue recognized from the collaboration and license agreement with Roche, which became effective in the third quarter of 2022, offset by the revenue recognized related to the research services performed under the collaboration and license agreements with Roche and Takeda, including $8.9 million of previously deferred revenue recognized as a result of the termination of its collaboration agreement with Takeda in the third quarter of 2023. Research and Development Expenses Research and development expenses were $42.0 million for the fourth quarter ended December 31, 2023, compared to $33.9 million for the same period in 2022. The increase was primarily due to an increase in preclinical stage programs and other unallocated expenses due to an increase in research collaboration activity, an increase in personnel expenses as a result of increased headcount, and an increase in clinical stage allogeneic programs, partially offset by a decrease in clinical stage autologous programs driven by the wind-down of the Company's clinical development activities for such programs. For the full year ended December 31, 2023, research and development expenses were $156.8 million, compared to $152.9 million for the same period in 2022. The increase was primarily due to an increase in preclinical stage programs and other unallocated expenses due to an increase in research collaboration activity, an increase in personnel expenses as a result of increased headcount, an increase in clinical stage allogeneic programs, and an increase in internal costs related to facilities and other expenses, offset by a decrease in clinical stage autologous programs driven by the wind-down of the Company's clinical development activities for such programs. General and Administrative Expenses General and administrative expenses were $8.9 million for the fourth quarter ended December 31, 2023, compared to $9.4 million for the same period in 2022. The decrease was primarily due to lower insurance costs. For the full year ended December 31, 2023, general and administrative expenses were $37.4 million, compared to $37.5 million for the same period in 2022. The decrease was primarily due to lower insurance costs, offset by an increase in personnel costs due to an accelerated stock-based compensation expense in the first quarter of 2023 related to a one-time expense associated with the retirement of the Company's former Executive Chairman. Net Loss Net loss was $25.3 million and $123.4 million for the fourth quarter and full year ended December 31, 2023, respectively, compared to net loss of $33.3 million and $64.0 million for the fourth quarter and full year ended December 31, 2022, respectively. Cash Position As of December 31, 2023, the Company's cash, cash equivalents and short-term investments balance was $212.2 million. The Company expects that its cash, cash equivalents and short-term investments together with the remaining near-term milestones and other payments from Roche will be sufficient to fund operations into the second half of 2025. Potential additional payments under the amended Roche collaboration and license agreement and/or potential additional business development could further extend cash runway. 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 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 pro 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. Company Codes: NASDAQ-NMS:PSTX

SUMMIT, N.J., March 6, 2024 /PRNewswire/ -- CSL Seqirus, a global leader in the protection of public health, confirmed it is fully prepared to deliver its influenza vaccine portfolio for the 2024/25 U.S. season, based on the trivalent strains recommended this week by the U.S. Food and Drug Administration's (FDA) Vaccines and Related Biological Products Advisory Committee (VRBPAC). During the meeting, the committee confirmed the viral strain selection of influenza vaccines for the Northern Hemisphere 2024/25 season, which aligns with the February 2024 World Health Organization (WHO) annual recommendations and is unchanged from the Southern Hemisphere 2024 season.1 "CSL Seqirus applauds the VRBPAC's commitment to providing a clear, evidence-backed recommendation to the FDA so that the composition of seasonal influenza vaccines for the upcoming season will be formulated to match projected circulating strains, based on the WHO's ongoing surveillance," said Gregg Sylvester, Chief Health Officer and Head of Medical Affairs, CSL Seqirus. "In addition to implementing this change, we are committed to working with our partners in public health to reverse the alarming decline in immunization rates and vaccine confidence that we have seen over the past two flu seasons." The VRBPAC's recommendation follows the October 2023 VRBPAC meeting, where the committee strongly supported the WHO's recommendation to expeditiously remove the B/Yamagata influenza virus strain from quadrivalent influenza vaccines and transition to trivalent influenza vaccines for the 2024/25 season.2 The recommendation was grounded in data from the WHO's global influenza response and surveillance system, which detected a decline in B/Yamagata circulation before the onset of the COVID-19 pandemic and no further circulation of B/Yamagata lineage viruses since March of 2020.3 The strain selection for the 2024/25 influenza season reflects the removal of B/Yamagata and it will not be included in the vaccines manufactured and delivered by CSL Seqirus. CSL Seqirus worked closely with the FDA during 2023 to align on a path forward and is fully prepared to transition its complete portfolio of seasonal influenza vaccines in the U.S. market from quadrivalent to trivalent formulations for the 2024/25 influenza season. CSL Seqirus completed its transition plan for the 2024/2025 season by December 2023 and regulatory and manufacturing teams are on track to convert the full U.S. portfolio. As a result of this effort, CSL Seqirus received FDA approval on March 4, 2024 for all of its U.S. trivalent influenza vaccines. "This strain consistency across hemispheres will support both speed and efficiency, as CSL Seqirus has experience manufacturing these recommended strains," said Dave Ross, Vice President, North America Commercial Operations, CSL Seqirus. Across the world, CSL Seqirus is collaborating with regulatory bodies and public health authorities on an appropriate transition timeline for each country that aims to ensure a smooth transition, increase vaccine confidence and improve immunization rates. The undetectable circulation of B/Yamagata is a testament to the critical role of widely implemented influenza immunization programs, amongst other contributing factors such as the inherent characteristics and epidemiology of the B/Yamagata virus and the unique environment created by the COVID-19 pandemic.3 About VRBPAC The VRBPAC is the FDA's advisory committee of experts in the fields of immunology, molecular biology, rDNA, virology bacteriology, epidemiology or biostatistics, vaccine policy, vaccine safety science, federal immunization activities, vaccine development including translational and clinical evaluation programs, allergy, preventive medicine, infectious diseases, pediatrics, microbiology, and biochemistry.4 VRBPAC reviews and evaluates data concerning the safety, effectiveness, and appropriate use of vaccines and related biological products which are intended for use in the prevention, treatment, or diagnosis of human diseases, and, as required, any other products for which the FDA has regulatory responsibility.4 About Seasonal Influenza Influenza is a common, contagious seasonal respiratory disease that may cause severe illness and life-threatening complications in some people.5 Influenza can lead to clinical symptoms varying from mild to moderate respiratory illness to severe complications, hospitalization and in some cases, death. Because transmission of influenza viruses to others may occur one day before symptoms develop and up to 5 to 7 days after becoming sick, the disease can be easily transmitted to others.5 Preliminary estimates from the Centers for Disease Control and Prevention (CDC) report that during the 2023/24 influenza season, there have been an estimated 290,000-610,000 influenza-related hospitalizations in the U.S.6 The CDC recommends annual vaccination for individuals aged 6 months and older, who do not have any contraindications.7 Since it takes about two weeks after vaccination for antibodies to develop in the body that help protect against influenza virus infection, it is recommended that people get vaccinated before influenza begins spreading in their community.7 The CDC recommends that for most people who need only 1 dose of flu vaccine, vaccination should ideally happen in September or October.7 About CSL Seqirus CSL Seqirus is part of CSL (ASX: CSL). As one of the largest influenza vaccine providers in the world, CSL Seqirus is a major contributor to the prevention of influenza globally and a transcontinental partner in pandemic preparedness. With state-of-the-art production facilities in the U.S., the U.K. and Australia, and leading R&D capabilities, CSL Seqirus utilizes egg, cell and adjuvant technologies to offer a broad portfolio of differentiated influenza vaccines in more than 20 countries around the world. For more information about CSL Seqirus, visit CSL.com/CSLSeqirus. About CSL CSL (ASX: CSL) (USOTC: CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSL.com/Vita and follow us on Twitter.com/CSL. For more information about CSL, visit . Intended Audience This press release is issued from CSL Seqirus in Summit, New Jersey, USA and is intended to provide information about our global business. Please be aware that information relating to the approval status and labels of approved CSL Seqirus products may vary from country to country. Please consult your local regulatory authority on the approval status of CSL Seqirus products. Forward-Looking Statements This press release may contain forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. USA-CRP-24-0011 MEDIA CONTACT Tiffany Cody +1 (908) 370-1863 [email protected] REFERENCES 1 Food and Drug Administration (FDA). Center for Biologics Evaluation and Research. 184th Vaccines and Related Biological Products Advisory Committee. March 2024. Retrieved from: . Accessed March 2024. 2 FDA. Center for Biologics Evaluation and Research. 183rd Vaccines and Related Biological Products Advisory Committee . Challenges and Opportunities for Vaccine Strain Composition with the Reduced Public Health Threat from Influenza B/Yamagata Lineage Viruses. Slide 7. October 2023. Retrieved from: . Accessed March 2024. 3 Koutsakos, M., Wheatley, A. K., Laurie, K., Kent, S. J., & Rockman, S. (2021). Influenza lineage extinction during the COVID-19 pandemic? Nature Reviews Microbiology, 19(12), 741-742. . 4 FDA. Center for Biologics Evaluation and Research. Vaccines and Related Biological Products Advisory Committee . (2019). Retrieved from: . Accessed March 2024. 5 Centers for Disease Control and Prevention (CDC). (2022). Key Facts About Influenza (Flu). Retrieved from: . Accessed March 2024. 6 CDC. (2024). 2023-2024 U.S. Flu Season: Preliminary In-Season Burden Estimates. Retrieved from: . Accessed March 2024. 7 CDC. (2024). Who Needs a Flu Vaccine. Retrieved from: . Accessed March 2024. 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