F8

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

BioMarin Pharmaceutical has shared new seven-year follow-up results for its adeno-associated virus vector-based gene therapy, Roctavian (valoctocogene roxaparvovec-rvox), in adults with severe haemophilia A. Affecting approximately one in 10,000 people, haemophilia A is a genetic disorder caused by a lack of sufficient functioning factor VIII clotting protein. This can lead to spontaneous bleeds, as well as bleeding following injuries or surgery. Patients with the most severe form of the disease are usually treated with intravenous factor VIII infusions two to three times per week or a bispecific monoclonal antibody one to four times per month. BioMarin’s Roctavian, which is administered as a one-time infusion, delivers a functional gene that is designed to enable the body to produce factor VIII on its own without the need for continued haemophilia prophylaxis, relieving patients of their treatment burden. The results from the mid-stage, open-label study of the therapy, presented at this year’s European Association for Haemophilia and Allied Disorders Congress, showed that median factor VIII activity at year seven remained in the mild haemophilia range. The mean annualised bleeding rate for treated bleeds over the full follow-up period decreased by 96% from baseline for adults with severe haemophilia A in the 6x1013vg/kg dose cohort, the company said, adding that the majority of patients maintained haemostasis, with only two of seven returning to regular prophylaxis. Hank Fuchs, president of worldwide research and development at BioMarin, said: "People living with severe haemophilia A face a lifelong treatment burden, including frequent injections or infusions and a high risk of health complications like uncontrolled bleeding and irreversible joint damage, which may persist despite good adherence to prophylactic therapy. "We are pleased to present data showing the impact of one-time treatment with Roctavian over seven years following the infusion, underscoring the potential of gene therapy to make a meaningful and long-lasting impact for people living with severe haemophilia A.” Roctavian has already been granted conditional marketing authorisation by the European Commission and has been approved by the US Food and Drug Administration. The company announced last August that a patient in Germany with severe haemophilia A had been treated with Roctavian, which signified the first time that the therapy had been given commercially in Europe.

New Seven-Year Follow-Up Phase 2 Study Results Showed Durable Hemostatic Benefit for Adults with Severe Hemophilia A SAN RAFAEL, Calif., Feb. 6, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that new data on ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) will be presented at the 2024 European Association for Haemophilia and Allied Disorders (EAHAD) Congress, Feb. 6-9, 2024. Results from a Phase 2, open-label study of ROCTAVIAN showed that median factor VIII (FVIII) activity at year seven remained in the mild hemophilia range (10.3 IU/dL per chromogenic assay), and mean annualized bleeding rate (ABR) for treated bleeds over the full follow-up period decreased by 96% from baseline for adults with severe hemophilia A in the 6x1013 vg/kg dose cohort. The majority of participants maintained hemostasis, with only two of the seven participants in the study returning to regular prophylaxis, and no new safety signals emerged. These data will be presented as a poster presentation from 6:30-7:30 p.m. Central European Time (CET) on Wednesday, Feb. 7. "People living with severe hemophilia A face a lifelong treatment burden, including frequent injections or infusions and a high risk of health complications like uncontrolled bleeding and irreversible joint damage, which may persist despite good adherence to prophylactic therapy," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We are pleased to present data showing the impact of one-time treatment with ROCTAVIAN over seven years following the infusion, underscoring the potential of gene therapy to make a meaningful and long-lasting impact for people living with severe hemophilia A." Additional studies will be shared during oral presentations on Friday, Feb. 9 from 8:30-10:00 a.m. CET, including preliminary results from GENEr8-INH, a Phase 1/2 study evaluating the safety and efficacy of ROCTAVIAN in participants with active and prior FVIII inhibitors, as well as one-year results from GENEr8-3, a Phase 3b study evaluating the use of prophylactic corticosteroids with ROCTAVIAN treatment. BioMarin's key presentations at EAHAD include: Poster Presentations: Seven-year follow-up of valoctocogene roxaparvovec gene therapy for haemophilia A #PO124 Wednesday, Feb. 7, 2024, 6:30-7:30 p.m. CET Treatment preferences in people with severe haemophilia A: a discrete choice experiment in the United States #PO314 Wednesday, Feb. 7, 2024, 6:30-7:30 p.m. CET Health-related quality of life and long-term joint damage in people with severe haemophilia A in Brazil #PO144 Wednesday, Feb. 7, 2024, 6:30-7:30 p.m. CET Oral Presentations: Safety and efficacy of valoctocogene roxaparvovec in participants with active and prior FVIII inhibitors: preliminary results from GENEr8-INH, a phase 1/2 study #OR10 Friday, Feb. 9, 2024, 8:30-10:00 a.m. CET Safety and efficacy of valoctocogene roxaparvovec with prophylactic corticosteroids: 1-year GENEr8-3 results #OR02 Friday, Feb. 9, 2024, 8:30-10:00 a.m. CET Human liver biopsy analysis reveals lower RNA transcription may contribute to a decline in FVIII levels following AAV5-hFVIII-SQ gene therapy #OR06 Friday, Feb. 9, 2024, 8:30-10:00 a.m. CET About Hemophilia A Hemophilia A, also called factor VIII (FVIII) deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective FVIII, a clotting protein. Although it is passed down from parents to children, about one-third of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A. About ROCTAVIAN ROCTAVIAN is an adeno-associated virus vector-based gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to adeno-associated virus serotype 5 (AAV5), which is determined by a blood test. The one-time infusion works by delivering a functional gene that is designed to enable the body to produce FVIII on its own, reducing the need for ongoing prophylaxis. The European Commission (EC) granted conditional marketing authorization to ROCTAVIAN on August 24, 2022. The U.S. Food and Drug Administration (FDA) approved ROCTAVIAN on June 29, 2023. More information on testing to determine eligibility to receive ROCTAVIAN can be found at in the U.S. and in Germany. U.S. Important Safety Information ROCTAVIAN U.S. Important Safety Information Contraindications: Patients with active infections, either acute (such as acute respiratory infections or acute hepatitis) or uncontrolled chronic (such as chronic active hepatitis B). Patients with known significant hepatic fibrosis (stage 3 or 4 on the Batts-Ludwig scale or equivalent), or cirrhosis, and patients with known hypersensitivity to mannitol. Infusion-related reactions including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during and for at least 3 hours after ROCTAVIAN administration. Administer ROCTAVIAN in a setting where personnel and equipment are immediately available to treat infusion-related reactions. Discontinue infusion for anaphylaxis. Hepatotoxicity: The safety and effectiveness of ROCTAVIAN in patients with hepatic impairment has not been established. Perform liver health assessments prior to administration. The majority of patients treated with ROCTAVIAN experienced ALT elevations and required corticosteroids for ALT elevation. Assess patient's ability to receive corticosteroids and/or other immunosuppressive therapy that may be required for an extended period. Live vaccines should not be administered to patients while on immunosuppressive therapy. Monitor ALT weekly for at least 26 weeks and as clinically indicated, during corticosteroid therapy and institute corticosteroid treatment in response to ALT elevations as required. Continue to monitor ALT until it returns to baseline. Monitor factor VIII activity levels since ALT elevation may be accompanied by a decrease in factor VIII activity. One case of autoimmune hepatitis was reported during third year follow-up in a patient with history of hepatitis C and steatohepatitis. It is recommended that patients abstain from consuming alcohol for at least 1 year after administration and thereafter limit alcohol use. Concomitant medications may cause hepatotoxicity, decrease factor VIII activity, or change plasma corticosteroid levels which may impact liver enzyme elevation and/or factor VIII activity or decrease the efficacy of the corticosteroid regimen or increase their side effects. Closely monitor concomitant medication use including herbal products and nutritional supplements and consider alternative medications in case of potential drug interactions. Thromboembolic events: Factor VIII activity above ULN has been reported following ROCTAVIAN infusion. Thromboembolic events may occur in the setting of elevated factor VIII activity above ULN. Evaluate patients for risk of thrombosis including general cardiovascular risk factors before and after administration of ROCTAVIAN. Advise patients on their individual risk of thrombosis in relation to their factor VIII activity levels above ULN and consider prophylactic anticoagulation. Advise patients to seek immediate medical attention for signs or symptoms indicative of a thrombotic event. Factor VIII inhibitors and Monitoring for inhibitors: The safety and effectiveness of ROCTAVIAN in patients with prior or active factor VIII inhibitors have not been established. Patients with active factor VIII inhibitors should not take ROCTAVIAN. Following administration, monitor patients for factor VIII inhibitors (neutralizing antibodies to factor VIII). Test for factor VIII inhibitors especially if bleeding is not controlled, or plasma factor VIII activity levels decrease. Monitor Factor VIII using the same schedule for ALT monitoring. It may take several weeks after ROCTAVIAN infusion before ROCTAVIAN-derived factor VIII activity rises to a level sufficient for prevention of spontaneous bleeding episodes. Exogenous factor VIII or other hemostatic products may also be required in case of surgery, invasive procedures, trauma, or bleeds. Consider more frequent monitoring in patients with factor VIII activity levels ≤ 5 IU/dL and evidence of bleeding, taking into account the stability of factor VIII levels since the previous measurement. Factor VIII activity produced by ROCTAVIAN in human plasma is higher if measured with one-stage clotting assays compared to chromogenic substrate assays. When switching from hemostatic products prior to ROCTAVIAN treatment, physicians should refer to the relevant prescribing information to avoid the potential for factor VIII activity assay interference during the transition period. Malignancy: The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of hepatocellular carcinoma development. ROCTAVIAN can also insert into the DNA of other human body cells. Monitor patients with risk factors for hepatocellular carcinoma (e.g., hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, non-alcoholic steatohepatitis, advanced age) with regular liver ultrasound (e.g., annually) and alpha-fetoprotein testing for 5 years following ROCTAVIAN administration. In the event that any malignancy occurs after treatment with ROCTAVIAN, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100. Most Common Adverse Reactions: Most common adverse reactions (incidence ≥ 5%) were nausea, fatigue, headache, infusion-related reactions, vomiting, and abdominal pain. Most common laboratory abnormalities (incidence ≥ 10%) were ALT, AST, LDH, CPK, factor VIII activity levels, GGT and bilirubin > ULN. Patients also experienced adverse reactions from corticosteroid use. Isotretinoin, Efavirenz, and HIV Positive Patients: Isotretinoin is not recommended in patients who are benefiting from ROCTAVIAN. Efavirenz is not recommended in patients treated with ROCTAVIAN. Clinical studies of ROCTAVIAN did not include sufficient numbers of patients with HIV to determine whether the efficacy and safety differs compared to patients without HIV infection. Females and Males of Reproductive Potential: ROCTAVIAN is not intended for administration in women. There are no data on the use of ROCTAVIAN in pregnant women or regarding lactation. For 6 months after administration of ROCTAVIAN, men of reproductive potential and their female partners must prevent or postpone pregnancy using an effective form of contraception, and men must not donate semen. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to BioMarin at 1-866-906-6100. Please see the ROCTAVIAN full Prescribing Information for additional Important Safety Information. About BioMarin Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit . Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the data BioMarin plans to present at the 2024 European Association for Haemophilia and Allied Disorders (EAHAD) Congress, including the three oral and three poster presentations; the development of BioMarin's ROCTAVIAN program; the potential impact and benefits of ROCTAVIAN for people with severe hemophilia A; and the continued clinical development of ROCTAVIAN. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of ROCTAVIAN, including achieving adequate market share and reimbursement levels; whether ROCTAVIAN will have the impacts and benefits as anticipated; the results and timing of current and planned preclinical studies and clinical trials of ROCTAVIAN and the release of data from those trials, including continued monitoring of the participants in the clinical trials and post-approval studies; BioMarin's ability to successfully manufacture ROCTAVIAN for the clinical trials and commercially; the content and timing of decisions by the FDA, EU health authorities and other regulatory authorities regarding ROCTAVIAN; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc. ROCTAVIAN™ is a trademark of BioMarin Pharmaceutical Inc. in the United States. Company Codes: NASDAQ-NMS:BMRN