Arrowhead Pharmaceuticals FY Conference Summary Company Overview - **Company**: Arrowhead Pharmaceuticals (NasdaqGS:ARWR) - **Event**: FY Conference held on December 03, 2025 Key Industry Insights - **Market Opportunity**: Arrowhead's first drug, Plozasiran, has been approved, targeting familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG) [3][4] - **Patient Need**: Approximately 10,000 patients with FCS require treatment to prevent severe pancreatitis, which can be fatal [4] - **Phase Three Data**: Plozasiran demonstrated an 80% reduction in triglycerides and a numerical risk reduction in pancreatitis [5] Competitive Landscape - **Pricing Strategy**: Plozasiran is priced lower than competitors like Tryngolza, which is priced at $595,000 per year per patient. Arrowhead aims to establish value in the SHTG market by initially targeting high-risk patients [6][7] - **Health Economics**: The cost of treating pancreatitis can exceed $60,000 per event, making Plozasiran's pricing compelling from both economic and human perspectives [8][9] Pipeline Developments - **New Candidates**: Arrowhead is developing a dual dimer targeting PCSK9 and ApoC3, which aims to lower LDL and remnant cholesterol [11][12] - **Obesity Programs**: Two programs, ARO-INHBE and ARO-ALK7, are in phase 1/2 studies, focusing on increasing lipolysis without affecting appetite [15][16] - **CNS Delivery**: ARO-MAPT targets tau tangles in Alzheimer's and tauopathies, with expectations for significant knockdown in cerebrospinal fluid (CSF) [22][24] Financial Position - **Cash Reserves**: Ended the fiscal year with $782 million, with additional capital from Sarepta and Novartis bringing total cash close to $1.2 billion [32] - **Funding Strategy**: The capital allows Arrowhead to advance core programs and aims for profitability by 2028, while reducing reliance on capital markets [32][33] Partnerships - **Sarepta Collaboration**: A significant partnership with Sarepta, valued at $800 million upfront and potential $10 billion in milestones, focuses on skeletal muscle programs [26][28] - **Novartis Partnership**: A $200 million upfront deal with Novartis for a preclinical Parkinson's disease drug, with potential for $2 billion in milestone payments [30][31] Conclusion - Arrowhead Pharmaceuticals is positioned for growth with a strong pipeline, strategic pricing, and significant partnerships, aiming to address critical health needs in cardiometabolic diseases and CNS disorders while maintaining a robust financial outlook.

Market Performance - The Dow is leading among large cap indexes with an increase of 0.66%, followed by the S&P 500 at 0.44% and Nasdaq at 0.42% [2] - Approximately 60% of U.S. stocks are rising today, indicating a positive market sentiment [3] Top Performers - Falcon's Beyond Global has surged by 15.3%, competing closely with Endeavour Silver Corp, which is up by 15.2%. Falcon's rise is attributed to recent earnings and a partnership with CD PROJEKT RED [3] - Endeavour Silver Corp's increase is linked to a 3% rise in silver prices [3] Underperformers - Jyong Biotech has dropped by 9.3%, and Arrowhead Pharmaceuticals has decreased by 8.4%, both showing significant declines without specific news driving the downturn [4] Opening Market Trends - The Russell 2000 index opened with a gain of 0.82%, leading the market, while the Nasdaq, S&P 500, and Dow also showed positive movements [5] - The 10Y Treasury yield increased by 1.3 basis points to 4.011%, indicating a slight rise in interest rates [5] - Silver futures rose by 3.69% to $55.59, while gold increased by 0.75% to $4,233. Brent Crude Oil saw a minor decline of 0.25% to $62.71 [5] Federal Reserve Leadership - Kevin Hassett is emerging as the frontrunner for the next Fed Chair, advocating for faster rate cuts to stimulate economic growth, which may challenge traditional views on Fed independence [6]

Overview of Arrowhead Pharmaceuticals - Arrowhead Pharmaceuticals has made significant recent progress and accomplishments, particularly highlighted by the update on the Sarepta partnership [2] Future Expectations - Investors should anticipate further developments from Arrowhead as the company approaches the end of the year and into 2026 [2]

Core Insights - Arrowhead Pharmaceuticals has achieved its first FDA approval for Redemplo, a treatment for familial chylomicronemia syndrome (FCS), marking a significant milestone for the company [3] - Following the earnings release, Arrowhead's stock surged by over 23%, reflecting investor optimism [1][5] - The company's revenue for fiscal 2025 reached over $829 million, a substantial increase from $3.6 million in 2024, driven by licensing and collaboration funds [2] Financial Performance - Arrowhead reported a narrowed attributable net loss of $1.6 million for the fiscal year, compared to a loss of $599 million the previous year [3] - The company's gross margin stands at an impressive 96.06% [4] - The market capitalization of Arrowhead Pharmaceuticals is currently $6 billion [4] Market Activity - The stock price of Arrowhead Pharmaceuticals increased to $10.92, with a daily range between $45.09 and $59.15 [4] - The trading volume was reported at 7.1 million, significantly higher than the average volume of 2.3 million [4] Future Prospects - The approval of Redemplo is expected to create a new revenue stream for Arrowhead, enhancing its financial outlook [5] - Ongoing collaborations are anticipated to continue generating funds as the company prepares for the commercialization of Redemplo [5]

Financial Data and Key Metrics Changes - The net loss for fiscal year 2025 was $2 million, a significant improvement from a net loss of approximately $599 million in fiscal year 2024 [33] - Revenue for fiscal year 2025 totaled $829 million, driven primarily by licensing and collaboration agreements, with $697 million from Sarepta Therapeutics [33][34] - Total operating expenses for fiscal year 2025 were approximately $731 million, an increase from $605 million in fiscal 2024, primarily due to higher R&D and SG&A costs [35] Business Line Data and Key Metrics Changes - The FDA approved Redemplo, Arrowhead's first FDA-approved medicine, marking a major milestone for the company as it transitions into the commercial stage [7][8] - Redemplo is indicated for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS) and can be self-administered at home [19][24] - The One Redemplo pricing model is set at $60,000 per patient per year, aimed at providing consistent pricing across indications [11][25] Market Data and Key Metrics Changes - There are an estimated 6,500 people in the U.S. living with genetic or clinical FCS, with a prescriber base comprising specialist physicians [25] - The broader patient population at risk of acute pancreatitis includes approximately 750,000 patients with persistent chylomicronemia [10] Company Strategy and Development Direction - Arrowhead is focusing on the cardiometabolic space, with ongoing development programs for Zodasiran and ArrowDiamond PA [11][14] - The company aims to have 20 individual drug candidates in clinical trials by the end of 2025, with a balanced pipeline of wholly owned and partnered programs [41] - Arrowhead is also expanding its clinical pipeline in CNS with ArrowMapT, targeting tauopathies including Alzheimer's disease [15] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the early feedback from healthcare professionals and payers regarding Redemplo, highlighting its significant and sustained triglyceride reduction [24] - The company anticipates that commercial sales of Redemplo will not have a substantial impact on financial statements in fiscal year 2026, but believes its cash runway is sufficient to extend into fiscal year 2028 [39] Other Important Information - Arrowhead received a $200 million upfront payment from Novartis for a licensing agreement and is eligible for up to $2 billion in future milestone payments [18][38] - The company earned a $200 million milestone payment from Sarepta following progress in the ARO-DM1 program [17][38] Q&A Session Summary Question: What is the plan to show benefit in terms of acute pancreatitis for Plozasiran? - Management indicated that shots at three and four were powered on triglyceride reduction, with the capability to pool data for evaluating pancreatitis [45] Question: Can you provide more details on the upcoming data for obesity programs? - Management confirmed that initial data from the ARO-INHBE program will be shared in early January, with more comprehensive data expected later in 2026 [51] Question: What are the estimates on acute pancreatitis events accrual based on patient baseline characteristics? - Management noted that it is challenging to predict events due to the use of modified Atlanta criteria, but they expect a good chance of having the necessary number of events [56]

Financial Data and Key Metrics Changes - The net loss for fiscal year 2025 was $2 million, a significant improvement from a net loss of approximately $599 million in fiscal year 2024, resulting in a loss of $0.01 per share compared to a loss of $5 per share [32] - Revenue for fiscal year 2025 totaled $829 million, driven primarily by licensing and collaboration agreements, with $697 million from Sarepta Therapeutics [32][33] - Total operating expenses increased to approximately $731 million in fiscal year 2025 from $605 million in fiscal 2024, primarily due to higher R&D and SG&A costs [35] Business Line Data and Key Metrics Changes - The FDA approval of Redemplo marked a major milestone, being the first FDA-approved siRNA medicine for familial chylomicronemia syndrome (FCS) [7][8] - Redemplo achieved an unprecedented reduction in triglycerides by approximately 80% from baseline as early as one month, with 50% of patients at the 25-mg dose achieving triglyceride levels below 500 mg per deciliter [24] - The company has made progress with Zodasiran, initiating the Yosemite phase III clinical trial for homozygous familial hypercholesterolemia (HoFH) [11][12] Market Data and Key Metrics Changes - There are an estimated 6,500 people in the U.S. living with genetic or clinical FCS, and approximately 750,000 patients with persistent chylomicronemia at high risk for acute pancreatitis [10][25] - The One Redemplo pricing model is set at $60,000 annually, aimed at providing consistent pricing across current and future indications [25] Company Strategy and Development Direction - The company is focusing on the cardiometabolic space, with ongoing development programs for Zodasiran and ArrowDiamond PA, targeting large populations without proper treatment options [14][15] - Arrowhead aims to have 20 individual drug candidates in clinical trials by the end of 2025, with a balanced pipeline of wholly owned and partnered programs [41] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the commercial launch of Redemplo and the positive feedback received from healthcare professionals and patient societies [24][25] - The company anticipates that the commercial sales of Redemplo will not have a substantial impact on financial statements in fiscal year 2026, but believes its cash runway is sufficient to extend into fiscal year 2028 [39] Other Important Information - Arrowhead received a $200 million upfront payment from Novartis for a licensing agreement and is eligible for up to $2 billion in future milestone payments [18][37] - The company has expanded its clinical pipeline in CNS with AROMAPT, targeting tauopathies including Alzheimer's disease [15] Q&A Session Summary Question: Can you discuss the plan to show benefit in terms of acute pancreatitis for Plozasiran? - Management indicated that shots at three and four were powered for triglyceride reduction, with a reasonable chance to show a difference in acute pancreatitis, but emphasized that shots at five are specifically designed to demonstrate benefit in acute pancreatitis [45][46] Question: Can you provide more details on the upcoming data for obesity programs? - Management confirmed that initial data from the AROINHBE program will be shared in early January, with a focus on safety and biomarker data, while ARO-ALK7 will provide limited data focused on monotherapy safety [50][52] Question: What are the estimates on acute pancreatitis events accrual based on patient baseline characteristics? - Management noted that it is difficult to estimate the number of events due to the new criteria being used, but they expect to have a sufficient number of events based on the percentage of patients with a history of pancreatitis enrolled in the study [56] Question: Can you discuss the differentiation of the ArrowMapT program from J&J's failed anti-tau antibody? - Management highlighted that ArrowMapT targets intracellular tau, potentially reducing both intracellular and extracellular tau levels, unlike J&J's approach which primarily targets extracellular tau [62] Question: What is the visibility on launching a CBOT study? - Management indicated that they expect to have a clearer idea by summer regarding the data from ongoing studies, which will inform the timeline for launching pivotal studies [65]

Financial Data and Key Metrics Changes - The net loss for fiscal year 2025 was $2 million, a significant improvement from a net loss of approximately $599 million in fiscal year 2024, resulting in a loss of $0.01 per share compared to a loss of $5 per share [32] - Revenue for fiscal year 2025 totaled $829 million, driven primarily by licensing and collaboration agreements, with $697 million from Sarepta Therapeutics [32][33] - Total operating expenses for fiscal year 2025 were approximately $731 million, an increase from $605 million in fiscal 2024, primarily due to higher R&D and SG&A costs [33][34] - Cash and investments totaled $919 million as of September 30, 2025, up from $681 million a year earlier, reflecting strong cash flow from licensing agreements [35][36] Business Line Data and Key Metrics Changes - The FDA approved Redemplo, Arrowhead's first FDA-approved medicine, marking a major milestone as it transitions into the commercial stage [6][7] - Redemplo is indicated for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS) and can be self-administered at home [19][20] - The One Redemplo pricing model is set at $60,000 annually, aimed at providing consistent pricing across indications [11][25] Market Data and Key Metrics Changes - There are an estimated 6,500 people in the U.S. living with genetic or clinical FCS, with a prescriber base comprising specialists such as lipidologists and endocrinologists [25] - The broader patient population at risk of acute pancreatitis includes approximately 750,000 patients with persistent chylomicronemia [10] Company Strategy and Development Direction - The company is focusing on the cardiometabolic space, with ongoing clinical trials for Zodasiran and ArrowDiamond PA, targeting conditions like homozygous familial hypercholesterolemia and atherosclerotic cardiovascular disease [12][13] - Arrowhead aims to have 20 individual drug candidates in clinical trials by the end of 2025, with a balanced pipeline of wholly owned and partnered programs [39] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the commercial launch of Redemplo and the encouraging feedback from the FCS community [39] - The company does not anticipate substantial financial impact from Redemplo sales in fiscal year 2026 but believes its cash runway is sufficient to support operations into fiscal year 2028 [38] Other Important Information - Arrowhead received a $200 million upfront payment from Novartis for a licensing agreement and is eligible for up to $2 billion in future milestone payments [36][37] - The company has made significant progress in business development, including a $200 million milestone payment from Sarepta for ARO-DM1 [17][38] Q&A Session Summary Question: What is the plan to show benefit in terms of acute pancreatitis for Plozasiran? - Management indicated that while shots at three and four were powered for triglyceride reduction, there is reasonable power to see a difference in acute pancreatitis, with a specific design for SHASTA-5 to demonstrate benefit [42][44][45] Question: Can you provide details on the upcoming data for obesity programs? - Management confirmed that initial data from the ARO-INHBE program will be shared in early January, with a focus on safety and biomarker data [48][50] Question: What are the estimates on acute pancreatitis events accrual based on patient baseline characteristics? - Management noted that it is challenging to predict due to the adoption of modified Atlanta criteria for adjudication, but they expect a sufficient number of events based on patient history [53][54] Question: Can you discuss the differentiation of ArrowMapT from J&J's failed anti-tau antibody? - Management highlighted that ArrowMapT targets intracellular tau, potentially reducing both intracellular and extracellular tau levels, unlike J&J's approach [57][59] Question: What is the visibility on launching a CBOT study? - Management stated that they expect to have a clearer idea by summer 2026 based on data from ongoing studies, with plans to move into pivotal studies if results are favorable [62][63]

Drug Development and Clinical Trials - The Company has 18 drug candidates in clinical trials, ranging from Phase 1 to Phase 3, with a robust discovery pipeline capable of generating multiple new clinical candidates each year[19] - REDEMPLO (plozasiran) was approved by the FDA in 2025 to reduce triglycerides in adults with Familial Chylomicronemia Syndrome (FCS), marking the Company's first commercial launch[20] - The Company plans to file a supplemental NDA for severe hypertriglyceridemia (sHTG) in 2026, pending successful completion of Phase 3 studies[20] - ARO-DIMER-PA is a dual functional RNAi molecule targeting PCSK9 and APOC3, representing a significant advancement in RNAi therapeutics[48] - The Company is investigating ARO-MAPT, designed to silence the MAPT gene, in a Phase 1/2a clinical trial for Alzheimer's and tauopathies[57] - ARO-CFB is being developed as a potential treatment for complement-mediated kidney diseases, currently in a Phase 1/2a clinical trial[61] - GSK is conducting Phase 2b clinical trials for GSK-4532990 targeting metabolic-dysfunction associated steatohepatitis (MASH) and alcohol-related liver disease (ALD)[65] - GSK received a worldwide exclusive license for daplusiran/tomligisiran (GSK5637608) to develop a potential therapy for chronic hepatitis B virus infection, with a Phase 2 study currently being initiated[66] - Takeda and the Company co-develop the fazirsiran program for liver disease associated with alpha-1 antitrypsin deficiency, with a 50/50 profit-sharing structure in the U.S.[67] - Fazirsiran aims to reduce the production of the mutant Z-AAT protein to prevent liver injury and fibrosis in patients with alpha-1 antitrypsin deficiency[70] - Amgen is responsible for the clinical development of olpasiran, designed to reduce elevated lipoprotein(a), a risk factor for atherosclerotic cardiovascular disease[73] Regulatory and Compliance - The FDA requires a user fee of approximately $4.682 million for NDA submissions requiring clinical data for fiscal year 2026[106] - The FDA's review process for NDAs typically aims for completion within ten months, with priority reviews targeted at six months[108] - The Company must comply with cGMP requirements to ensure consistent production of its products before the FDA approves an NDA[109] - The FDA may require a REMS plan to mitigate serious risks associated with a drug, which can affect market potential and profitability[110] - The FDA may impose conditions on approved products, including post-approval studies and distribution restrictions, which can materially affect market dynamics[113] - The FDA may not approve any other application to market the same drug for the same indication for seven years, except in very limited circumstances[116] - The FDA's interpretation of orphan drug exclusivity may change, potentially limiting the drugs that can receive such exclusivity[117] - Fast Track Designation allows for priority review and rolling review for drugs addressing serious conditions with unmet medical needs[118] - The FDA may approve a drug under the accelerated approval program if it provides a meaningful advantage over existing therapies and demonstrates an effect on a surrogate endpoint[119] - The Breakthrough Therapy designation provides more intensive guidance and expedited review for drugs showing substantial improvement over existing therapies[120] - Post-approval requirements include ongoing regulation by the FDA, including recordkeeping and reporting of adverse experiences[121] - Drug manufacturers must register with the FDA and are subject to periodic inspections to ensure compliance with cGMP requirements[122] - The FDA may withdraw approval if compliance with regulatory requirements is not maintained or if new problems are discovered post-market[123] Intellectual Property and Licensing - The Company controls approximately 643 issued patents and has around 833 pending patent applications worldwide, covering various RNAi technologies[85] - The Company may need to obtain additional patent licenses prior to commercialization of its candidates due to the complex patent landscape in RNAi and drug delivery[89] - The Company acquired RNAi assets and rights from Novartis, including patents related to RNAi therapeutics and delivery technologies[90] - The acquisition from Roche provided the Company with licenses to patents related to modifications of double-stranded oligonucleotides, enhancing its operational freedom in RNAi-trigger formats[91][92] - The Company holds a worldwide, royalty-bearing, exclusive license under the Alnylam-Novartis License to research, develop, and commercialize 30 undisclosed gene targets[90] Market Exclusivity and Orphan Drug Designation - Orphan drug designation can provide the Company with incentives such as grant funding and tax advantages for drugs intended to treat rare diseases[114] - The company is subject to a 10-year market exclusivity for orphan medicinal products, which can be reduced to six years if criteria are not met[165] - Orphan drug designation provides up to ten years of market exclusivity in the EU, contingent on maintaining the designation at the time of marketing authorization review[163] Manufacturing and Workforce - The Company expanded its footprint with a new manufacturing and laboratory facility in Verona, Wisconsin during Q1 fiscal 2025[208] - The new state-of-the-art GMP manufacturing facility in Verona includes over 251,000 total square feet of laboratories and office space[211] - As of September 30, 2025, the Company employed 711 full-time employees across four facilities in the U.S.[212] - The Company continues to expand its workforce to increase in-house manufacturing capacity and enhance clinical and preclinical research expertise[213] - The Company offers a total compensation package targeting the 50 to 75 percentile of the market, including base salary, cash bonuses, and equity compensation[214] - The Company supports employee development through individualized plans, mentoring, and tuition reimbursement[214] - The Company is committed to fostering an inclusive environment with ongoing programs for awareness and engagement[215] Data Privacy and Security - The company must comply with various data privacy and security laws, including HIPAA and state privacy laws, which impose significant obligations regarding personal information[185] - Failure to comply with data privacy laws can result in substantial civil and criminal penalties, posing financial and reputational risks to the company[188] - The GDPR and UK GDPR impose fines of up to €20 million or 4% of annual global revenues for noncompliance, whichever is greater[191] - The complexity of data protection regulations in the EEA is heightened by varying enforcement and additional national regulations[191] - The transfer of personal data outside the EEA requires appropriate safeguards, such as standard contractual clauses[193] Pricing and Reimbursement - The pricing and reimbursement environment in the EU allows member states to control prices and reimbursement levels, which may affect market access for the company's products[177] - Health technology assessments (HTA) are increasingly influencing pricing and reimbursement decisions in EU member states, focusing on clinical efficacy, safety, and cost-effectiveness[179] - The company may be required to conduct cost-effectiveness studies to obtain favorable pricing and reimbursement conditions in certain EU member states[182] - Orphan pharmaceutical products may be exempted from providing certain clinical and economic data for pricing/reimbursement approval in some EU member states[183] Regulatory Changes in the EU - The UK government enacted the Medicines and Medical Devices Act 2021 to update regulatory frameworks for human medicines and clinical trials, ensuring public health is safeguarded[141] - The centralized procedure for Marketing Authorization (MA) in the EU has a standard evaluation timeframe of 210 days, which can be extended due to additional information requests[152] - New Chemical Entities (NCE) approved in the EU qualify for eight years of data exclusivity and ten years of marketing exclusivity, extendable to eleven years under certain conditions[157] - The European Medicines Agency (EMA) provides scientific advice for drug development, with fees significantly reduced for orphan medicines[149] - The Medical Devices (Amendment) (Great Britain) Regulations 2023 allow CE marked devices to be sold in the UK until 2028 or 2030, depending on the device type[144] - The Medicines and Healthcare products Regulatory Agency (MHRA) plans to implement new 'Pre-Market Regulations' in 2026, extending the sunset period for key EU-derived regulations[144] - The EU Clinical Trials Regulation (CTR) became applicable on January 31, 2022, streamlining the approval process for clinical trials across member states[145] - The current proposal for reforming European pharmaceutical legislation includes shortening data exclusivity from eight to six years, with incentives for developing new antibiotics[161]

Financial Performance - Arrowhead Pharmaceuticals reported a revenue of $829.448 million for the fiscal year 2025, a significant increase from $3.551 million in 2024[10] - The company achieved an operating income of $98.346 million in 2025, compared to an operating loss of $601.080 million in 2024[10] - Arrowhead's net income attributable to the company was a loss of $1.631 million in 2025, an improvement from a loss of $599.493 million in the previous year[10] - The company has $919.366 million in total cash resources as of September 30, 2025, compared to $680.961 million in 2024[10] - Arrowhead's total assets increased to $1.385 billion in 2025, up from $1.140 billion in 2024[10] Regulatory Approvals and Clinical Trials - The FDA approved REDEMPLO (plozasiran), the first FDA-approved siRNA medicine for familial chylomicronemia syndrome (FCS), which can reduce triglycerides by 80% from baseline[6][11] - The company filed for regulatory clearance to initiate Phase 1/2a clinical trials for ARO-DIMER-PA and ARO-MAPT, targeting atherosclerotic cardiovascular disease and tauopathies, respectively[7] Partnerships and Licensing Agreements - Arrowhead received a $200 million upfront payment from Novartis as part of a global licensing agreement for ARO-SNCA, with potential milestone payments of up to $2 billion[7] - Arrowhead earned $300 million in milestone payments from Sarepta Therapeutics related to the ARO-DM1 clinical study[7] - The company signed an asset purchase agreement with Sanofi, receiving an upfront payment of $130 million and potential milestone payments of up to $265 million for plozasiran in Greater China[8]

Core Insights - Sarepta Therapeutics' shares increased by 7% following the announcement of progress in its phase I/II study for SRP-1003, a therapeutic for type 1 myotonic dystrophy (DM1) [1][8] Study Progress - The company has completed two cohorts of the study and has enrolled patients in a third cohort, with a positive review from a drug safety committee allowing for the advancement of additional dosing cohorts [2] - Currently, patients are being dosed in a fourth cohort, with plans to start the fifth cohort in Q1 2026, and initial readout from the study is expected early next year [2] Stock Performance - Following the announcement, investor sentiment improved, especially in light of safety concerns surrounding other investigational DM1 therapies from competitors [3] - The stock has faced significant challenges this year, dropping nearly 85% compared to the industry’s 17% growth [6] Milestone Payments - The progress in the study triggered a $200 million milestone payment to Arrowhead Pharmaceuticals, part of a multi-billion-dollar licensing deal for SRP-1003 [7][8] - This is the second milestone payment in 2023, with the first being $100 million in July for reaching enrollment targets [9] Additional Acquisitions - Besides SRP-1003, Sarepta has acquired exclusive rights to three other clinical-stage programs from Arrowhead, which are in separate phase I/II studies [10] - Initial data for the facioscapulohumeral muscular dystrophy (FSHD) program is anticipated in early 2026 [10]