Volanesorsen

- First independent launch underway following TRYNGOLZATM approval - - Second independent launch on track with donidalorsen PDUFA - - WAINUATM launch delivering accelerating sequential growth - - Ionis exceeds 2024 financial guidance and provides full year 2025 guidance - CARLSBAD, Calif. --(BUSINESS WIRE)--Feb. 19, 2025-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results for the fourth quarter and full year ended December 31, 2024 . “With the recent launch of our first independent medicine, TRYNGOLZA for familial chylomicronemia syndrome, Ionis has begun a new chapter as a fully integrated commercial-stage biotechnology company,” said Brett P. Monia , Ph.D., chief executive officer, Ionis. “Over the next three years, we expect three more independent launches, including donidalorsen later this year for hereditary angioedema and olezarsen for severe hypertriglyceridemia in 2026, pending Phase 3 results in the second half of this year. Additionally, our partners are on track to launch four Ionis-discovered medicines over the same time period, including several that address broad patient populations. Ionis continues to advance our next wave of potentially transformational wholly owned medicines, including ION582 for Angelman syndrome, which is on track to start Phase 3 development in the first half of this year. Our recent achievements, combined with our strong commercial execution and advancing pipeline, position Ionis to deliver increasing value for all our stakeholders.” Fourth Quarter and Full Year 2024 Summary Financial Results(1): Three months ended December 31 , Year ended December 31 , 2024 2023 2024 2023 (amounts in millions) Total revenue $ 227 $ 325 $ 705 $ 788 Operating expenses $ 337 $ 331 $ 1,180 $ 1,141 Operating expenses on a non-GAAP basis $ 301 $ 305 $ 1,050 $ 1,035 Loss from operations $ (110 ) $ (6 ) $ (475 ) $ (353 ) Income (Loss) from operations on a non-GAAP basis $ (74 ) $ 20 $ (345 ) $ (247 ) (1) Reconciliation of GAAP to non-GAAP basis contained later in this release. Financial Highlights Revenue for the year ended December 31, 2024 substantially exceeded guidance as Ionis continued to generate revenue from diverse sources. Ionis added new sources of revenue in 2024 with the launch of WAINUA and TRYNGOLZA in the U.S. in late January and late December, respectively Operating expenses for the year ended December 31, 2024 were in line with expectations with increased expenses from commercialization efforts for WAINUA, TRYNGOLZA and donidalorsen Cash, cash equivalents and short-term investments of $2.3 billion as of December 31, 2024 exceeded revised guidance and enable continued investments to support ongoing and upcoming planned launches and advancing wholly owned medicines in development Recent Highlights- Marketed Medicines TRYNGOLZATM (olezarsen) launch underway following approval on December 19, 2024 in the U.S as first-ever treatment for adults living with familial chylomicronemia syndrome (FCS) as an adjunct to diet WAINUATM (eplontersen) (WAINZUA in EU) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) achieved multiple commercial and regulatory milestones: Generated sales of $85 million resulting in royalty revenue of $20 million in the year ended December 31, 2024 . Substantial sequential growth throughout 2024 as launch progressed, including an 84% increase in product sales in the fourth quarter, compared to the third quarter Launch underway in numerous countries, including the UK , following approval by the Medicines and Healthcare products Regulatory Agency (MHRA) with an accelerated National Institute for Health and Care Excellence (NICE) recommendation Received positive Committee for Medicinal Products for Human Use (CHMP) opinion from European Medicines Agency (EMA) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of $1.6 billion resulting in royalty revenue of $216 million in year ended December 31, 2024 . Product sales increased 2% in the fourth quarter of 2024, compared to the same period in 2023 Higher dose nusinersen under regulatory review in U.S. (PDUFA date of September 22, 2025 ) and EU QALSODY® (tofersen) for the treatment of SOD1-ALS generated global sales of $32 million resulting in royalty revenue of $4 million in the year ended December 31, 2024 . Product sales grew sequentially throughout 2024 Granted marketing approval in China and Japan Generated sales of $85 million resulting in royalty revenue of $20 million in the year ended December 31, 2024 . Substantial sequential growth throughout 2024 as launch progressed, including an 84% increase in product sales in the fourth quarter, compared to the third quarter Launch underway in numerous countries, including the UK , following approval by the Medicines and Healthcare products Regulatory Agency (MHRA) with an accelerated National Institute for Health and Care Excellence (NICE) recommendation Received positive Committee for Medicinal Products for Human Use (CHMP) opinion from European Medicines Agency (EMA) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy Higher dose nusinersen under regulatory review in U.S. (PDUFA date of September 22, 2025 ) and EU Granted marketing approval in China and Japan Recent Highlights- Late-Stage Pipeline Olezarsen on track for Phase 3 data in patients with severe hypertriglyceridemia (sHTG) in H2:2025, positioning it to potentially treat this second more prevalent patient population with urgent unmet need Olezarsen and donidalorsen Canadian commercialization rights licensed to Theratechnologies Donidalorsen on track for potential launch this year as the first RNA-targeted prophylactic treatment for people with hereditary angioedema (HAE): Under review in U.S. (PDUFA date of August 21, 2025 ) and EU Presented positive Phase 2 open label extension (OLE) study data demonstrating an overall sustained mean reduction in HAE attack rates of 96% in patients treated up to three years with every four weeks or every eight weeks dosing ION582 on track to initiate Phase 3 development in Angelman syndrome (AS) in H1:2025 Ulefnersen global commercialization rights licensed to Otsuka; Phase 3 development ongoing for the treatment of patients with FUS-ALS Under review in U.S. (PDUFA date of August 21, 2025 ) and EU Presented positive Phase 2 open label extension (OLE) study data demonstrating an overall sustained mean reduction in HAE attack rates of 96% in patients treated up to three years with every four weeks or every eight weeks dosing Fourth Quarter, Full Year 2024 Financial Results and 2025 Financial Guidance “In 2024, we exceeded our revenue guidance due to our continued pipeline and technology successes, which drove a smaller than anticipated operating loss. Importantly, we added two new sources of commercial revenue with TRYNGOLZA product revenue from Ionis’ first independent launch and WAINUA royalties,” said Elizabeth L. Hougen , chief financial officer, Ionis. “In 2025, we will continue to invest in go-to-market activities for TRYNGOLZA for FCS and scale our resources to support our next planned launches, including donidalorsen for hereditary angioedema later this year. At the same time, we are investing in our next wave of medicines, including Phase 3 development and pre-commercialization activities for ION582 for Angelman syndrome. These important investments position Ionis to deliver substantial and growing product revenue; and when combined with increasing royalty revenue from anticipated partner launches, provides us with a clear path to achieve sustained positive cash flow.” Revenue Ionis’ revenue was comprised of the following: Three months ended Year ended December 31 , December 31 , 2024 2023 2024 2023 Revenue: (amounts in millions) Commercial revenue: SPINRAZA royalties $ 64 $ 62 $ 216 $ 240 WAINUA royalties 10 - 20 - Other commercial revenue: TEGSEDI and WAYLIVRA revenue, net 8 9 34 35 Other revenue 4 8 23 34 Total commercial revenue 86 79 293 309 Research and development revenue: Amortization from upfront payments 27 76 132 125 Milestone payments 30 11 106 101 License fees 34 92 71 117 Other services 6 - 24 10 Collaborative agreement revenue 97 179 333 353 WAINUA joint development revenue 44 67 79 126 Total research and development revenue 141 246 412 479 Total revenue $ 227 $ 325 $ 705 $ 788 Commercial revenue for the year ended December 31, 2024 included new sources of commercial revenue with the launch of WAINUA in the U.S. in late January 2024 and the launch of TRYNGOLZA in the U.S. in late December 2024 . SPINRAZA product sales in the U.S. increased slightly in 2024 compared to 2023. SPINRAZA product sales outside of the U.S. were impacted from an annual order from a single country that did not recur in 2024. R&D revenue decreased for the year ended December 31, 2024 compared to 2023 primarily due to the decrease in WAINUA joint development revenue as development activities relating to ATTRv-PN wound down with the launch of WAINUA. Operating Expenses Ionis’ operating expenses increased slightly for the year ended December 31, 2024 compared to 2023. SG&A expenses increased year over year primarily due to the launches of WAINUA and TRYNGOLZA, and advancing launch preparation activities for donidalorsen. R&D expenses were flat year over year. Balance Sheet As of December 31, 2024 , Ionis’ cash, cash equivalents and short-term investments were $2.3 billion , consistent with December 31, 2023 . In September 2024 , Ionis generated gross proceeds of $500 million from a public offering of its common stock. Ionis’ working capital increased as of December 31, 2024 , compared to December 31, 2023 primarily from the Company’s lower current liabilities as a result of lower deferred contract revenue. 2025 Financial Guidance The Company’s 2025 financial guidance reflects its evolution to a fully integrated commercial-stage biotechnology company independently launching its first medicine, TRYNGOLZA, and advancing commercialization efforts for multiple additional upcoming planned launches. As a result, the Company expects to earn substantial revenue from numerous diverse sources, with a shift toward increasing commercial revenue. Additionally, the Company expects a modest increase in its non-GAAP operating expenses in line with its plan to invest in the Company’s independent launches and advance its wholly owned pipeline of innovative medicines. The Company expects that these investments will enable Ionis to deliver accelerating value. Full Year 2025 Guidance Revenue > $600 million Operating loss on a non-GAAP basis < $495 million Cash, cash equivalents and short-term investments ~$1.7 billion Webcast Management will host a conference call and webcast to discuss Ionis’ fourth quarter and full year 2024 results at 11:30 a.m. Eastern time on Wednesday, February 19, 2025 . Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address. To access the Company’s fourth quarter and full year 2024 earnings slides click here. For more information about SPINRAZA and QALSODY, visit https://www.spinraza.com/ and https://www.qalsody.com/, respectively. QALSODY is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval may be contingent upon verification of clinical benefit in confirmatory trial(s). INDICATION for TRYNGOLZA™ (olezarsen) TRYNGOLZA™ (olezarsen) was approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur. ADVERSE REACTIONS The most common adverse reactions (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased platelet count and arthralgia. Please see full Prescribing Information for TRYNGOLZA. INDICATION for WAINUA™ (eplontersen) WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. IMPORTANT SAFETY INFORMATION for WAINUA™ (eplontersen) WARNINGS AND PRECAUTIONS Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur. ADVERSE REACTIONS Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%). Please see link to Full Prescribing Information for WAINUA. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram. Ionis’ Forward-looking Statement This press release includes forward-looking statements regarding Ionis’ business, financial guidance and the therapeutic and commercial potential of our commercial medicines, additional medicines in development and technologies. Any statement describing Ionis’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis’ forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2023 , and most recent Form 10-Q, which are on file with the Securities and Exchange Commission . Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, “Ionis,” “Company,” “we,” “our” and “us” all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals ® is a registered trademark of Ionis Pharmaceuticals, Inc. TRYNGOLZATM is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics ® is a registered trademark of Akcea Therapeutics, Inc. TEGSEDI® is a registered trademark of Akcea Therapeutics, Inc. WAYLIVRA® is a registered trademark of Akcea Therapeutics, Inc. SPINRAZA® and QALSODY® are registered trademarks of Biogen. WAINUATM is a registered trademark of the AstraZeneca group of companies. IONIS PHARMACEUTICALS, INC. SELECTED FINANCIAL INFORMATION Condensed Consolidated Statements of Operations (In Millions, Except Per Share Data) Three months ended Year ended December 31 , December 31 , 2024 2023 2024 2023 (unaudited) Revenue: Commercial revenue: SPINRAZA royalties $ 64 $ 62 $ 216 $ 240 WAINUA royalties 10 - 20 - Other commercial revenue 12 17 57 69 Total commercial revenue 86 79 293 309 Research and development revenue: Collaborative agreement revenue 97 179 333 353 WAINUA joint development revenue 44 67 79 126 Total research and development revenue 141 246 412 479 Total revenue 227 325 705 788 Expenses: Cost of sales 4 3 11 9 Research, development and patent 245 257 902 900 Selling, general and administrative 88 71 267 232 Total operating expenses 337 331 1,180 1,141 Loss from operations (110 ) (6 ) (475 ) (353 ) Other income (expense): Interest expense related to the sale of future royalties (19 ) (18 ) (73 ) (69 ) Other income, net 22 21 88 88 Loss before income tax benefit (expense) (107 ) (3 ) (460 ) (334 ) Income tax benefit (expense) 3 (6 ) 6 (32 ) Net loss $ (104 ) $ (9 ) $ (454 ) $ (366 ) Basic and diluted net loss per share $ (0.66 ) $ (0.06 ) $ (3.04 ) $ (2.56 ) Shares used in computing basic and diluted net loss per share 158 144 150 143 IONIS PHARMACEUTICALS, INC. Reconciliation of GAAP to Non-GAAP Basis: Condensed Consolidated Operating Expenses, Loss From Operations, and Net Loss (In Millions) Three months ended December 31 , Year ended December 31 , 2024 2023 2024 2023 (unaudited) As reported research, development and patent expenses according to GAAP $ 245 $ 257 $ 902 $ 900 Excluding compensation expense related to equity awards (25 ) (20 ) (92 ) (78 ) Non-GAAP research, development and patent expenses $ 220 $ 237 $ 810 $ 822 As reported selling, general and administrative expenses according to GAAP $ 88 $ 71 $ 267 $ 232 Excluding compensation expense related to equity awards (11 ) (6 ) (37 ) (27 ) Non-GAAP selling, general and administrative expenses $ 77 $ 65 $ 230 $ 205 As reported operating expenses according to GAAP $ 337 $ 331 $ 1,180 $ 1,141 Excluding compensation expense related to equity awards (36 ) (26 ) (130 ) (106 ) Non-GAAP operating expenses $ 301 $ 305 $ 1,050 $ 1,035 As reported loss from operations according to GAAP $ (110 ) $ (6 ) $ (475 ) $ (353 ) Excluding compensation expense related to equity awards (36 ) (26 ) (130 ) (106 ) Non-GAAP loss from operations $ (74 ) $ 20 $ (345 ) $ (247 ) As reported net loss according to GAAP $ (104 ) $ (9 ) $ (454 ) $ (366 ) Excluding compensation expense related to equity awards and related tax effects (36 ) (26 ) (130 ) (106 ) Non-GAAP net loss $ (68 ) $ 17 $ (324 ) $ (260 ) Reconciliation of GAAP to Non-GAAP Basis As illustrated in the Selected Financial Information in this press release, non-GAAP operating expenses, non-GAAP loss from operations, and non-GAAP net loss were adjusted from GAAP to exclude compensation expense related to equity awards and the related tax effects. Compensation expense related to equity awards are non-cash. These measures are provided as supplementary information and are not a substitute for financial measures calculated in accordance with GAAP. Ionis reports these non-GAAP results to better enable financial statement users to assess and compare its historical performance and project its future operating results and cash flows. Further, the presentation of Ionis’ non-GAAP results is consistent with how Ionis’ management internally evaluates the performance of its operations. IONIS PHARMACEUTICALS, INC. Condensed Consolidated Balance Sheets (In Millions) December 31 , December 31 , 2024 2023 (unaudited) Assets: Cash, cash equivalents and short-term investments $ 2,298 $ 2,331 Contracts receivable 92 98 Other current assets 230 213 Property, plant and equipment, net 94 71 Right-of-use assets 162 172 Other assets 127 105 Total assets $ 3,003 $ 2,990 Liabilities and stockholders’ equity: Current portion of deferred contract revenue $ 79 $ 151 0.125% convertible senior notes, net – short-term - 44 Other current liabilities 229 253 1.75% convertible senior notes, net 565 562 0% convertible senior notes, net 629 625 Liability related to sale of future royalties, net 542 514 Long-term lease liabilities 162 171 Long-term obligations, less current portion 52 42 Long-term deferred contract revenue 157 241 Total stockholders’ equity 588 387 Total liabilities and stockholders’ equity $ 3,003 $ 2,990 Key 2025 and 2026 Value Driving Events(1) New Product Launches Program Indication 2025 2026 Donidalorsen ( U.S. ) HAE • TRYNGOLZA ( U.S. ) FCS Achieved WAINZUA (EU) ATTRv-PN • Olezarsen ( U.S. ) sHTG • Zilganersen ( U.S. ) Alexander disease • Regulatory Actions Program Indication Regulatory Action 2025 2026 Donidalorsen HAE U.S. approval decision • EU approval decision • TRYNGOLZA FCS EU approval decision • Olezarsen sHTG U.S. submission • U.S. approval decision • Zilganersen Alexander disease U.S. submission • U.S. approval decision • Nusinersen (higher dose) SMA U.S. and EU submissions Achieved U.S. approval decision • WAINZUA ATTRv-PN EU approval decision • Pelacarsen Lp(a)- CVD U.S. submission • Bepirovirsen HBV Regulatory submission(s) • Regulatory decision(s) • Key Phase 3 Clinical Events Program Indication Event 2025 2026 Olezarsen sHTG CORE, CORE2 and Essence data • Zilganersen Alexander disease Phase 3 data • ION582 Angelman syndrome Phase 3 study start • Phase 3 enrollment completion • Pelacarsen Lp(a)-CVD HORIZON data • Bepirovirsen HBV B-Well data • Eplontersen ATTR-CM CARDIO-TTRansform data • Sefaxersen IgAN IMAGINATION data • Ulefnersen FUS-ALS Phase 3 data • (1) Timing expectations based on current assumptions and subject to change. • View source version on businesswire.com: https://www.businesswire.com/news/home/20250219347982/en/ Ionis Investor Contact: D. Wade Walke , Ph.D. IR@ionis.com 760-603-2331 Ionis Media Contact: Hayley Soffer media@ionis.com 760-603-4679 Source: Ionis Pharmaceuticals, Inc.

– Unaudited 2024 total revenue of approximately $814 million, exceeding guidance – – Four approval applications submitted to FDA in 2024 – – Global launch preparations underway for sepiapterin for PKU, a $1 billion market opportunity; CHMP opinion expected Q2 2025, U.S. approval decision in July 2025 – – License and collaboration agreement with Novartis for PTC518 program closed – – PIVOT-HD data readout for PTC518 expected Q2 2025 – WARREN, N.J., Jan. 13, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today provided an update on the Company's progress and its outlook for 2025. Matthew B. Klein, M.D., Chief Executive Officer of PTC, will discuss these updates at the 43rd Annual J.P. Morgan Healthcare Conference today at 11:15 a.m. PST / 2:15 p.m. EST. The presentation will be webcast live on the Events and Presentations page under the Investors section of PTC Therapeutics' website at and will be archived for 30 days following the presentation. "I am very proud of our team's performance across every part of the company in 2024," Dr. Klein said. "We met every one of our planned clinical and regulatory milestones on schedule. Commercially, we outperformed guidance. And we begin 2025 with a strong cash balance, ready to build on our successes. PTC, now stronger and more innovative than ever, will continue to work to bring transformative therapies to the patient communities we serve in 2025 and beyond." Key 2024 Corporate Highlights: Unaudited net product revenue of approximately $814 million, exceeding guidance. Strong performance was driven by in-line products, including the DMD franchise with unaudited net product revenue of approximately $340 million for Translarna™ (ataluren) and approximately $207 million for Emflaza® (deflazacort) in 2024. Cash balance of approximately $1.1 billion as of December 31, 2024, with an additional $1.0 billion in upfront proceeds from PTC518 collaboration agreement with Novartis following closing. PTC submitted four regulatory approval applications to FDA: Kebilidi™ (eladocagene exuparvovec-tneq) gene therapy for AADC deficiency, approved in November 2024. Sepiapterin for children and adults with PKU, accepted with an FDA action date of July 29, 2025. Vatiquinone for children and adults with FA, with filing acceptance decision anticipated Q1 2025. Translarna for nmDMD, accepted with no action date provided. PTC signed a global license and collaboration agreement with Novartis for the research, development and commercialization of PTC518 for HD, which has now closed. Key aspects of the transaction include the following: PTC to receive $1.0 billion in upfront proceeds following closing. PTC is eligible to receive up to $1.9 billion in development, regulatory and sales milestones. PTC to receive 40% profit share on U.S. sales, and double-digit tiered royalties on ex-U.S. sales. Novartis will assume global development, manufacturing and commercial responsibilities following the completion of the placebo-controlled portion of PIVOT-HD, which is expected in 1H 2025. PTC received $150 million from the sale of the Rare Pediatric Disease PRV it received with FDA approval of Kebilidi. In December 2024, PTC had a Type C meeting with FDA to discuss the potential for HTT lowering to serve as a surrogate endpoint to support accelerated approval for PTC518 for HD. The Agency was aligned with the scientific rationale for HTT lowering as a potential surrogate endpoint and asked that PTC provide additional clinical data, such as those being collected in PIVOT-HD, to show associations between HTT lowering and changes in clinical outcome measures. 2025 Potential Key Clinical and Regulatory Events: CHMP opinion on sepiapterin MAA expected in Q2 2025. FDA approval decision on sepiapterin NDA expected July 29, 2025. Results from the PIVOT-HD Phase 2 study of PTC518 expected in Q2 2025. NDA acceptance of vatiquinone expected in Q1 2025, with potential regulatory approval in 2H 2025. Unaudited 2024 Financial Results: Total unaudited net revenue for full-year 2024 was approximately $814 million. Total unaudited net product revenue for full-year 2024 was approximately $601 million. DMD franchise unaudited revenue for full-year 2024 was approximately $547 million, including unaudited net product revenue for Translarna of approximately $340 million and for Emflaza of approximately $207 million. PTC expects to report approximately $211 million of full-year 2024 royalty revenue associated with Evrysdi®. PTC is finalizing its financial results for the 2024 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2024, subject to the completion of PTC's financial closing procedures. Evrysdi royalty revenue estimates are based on internal estimates. 2025 Financial Guidance: PTC anticipates total revenues for the full-year 2025 to be between $600 million and $800 million, including in-line products, potential new product launches and royalty revenue from Evrysdi. PTC anticipates GAAP R&D and SG&A expense for the full-year 2025 to be between $805 million and $835 million. PTC anticipates Non-GAAP R&D and SG&A expense for the full-year 2025 to be between $730 million and $760 million, excluding estimated non-cash, stock-based compensation expense of $75 million. Non-GAAP Financial Measures: In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms: AADC: Aromatic I-Amino Acid Decarboxylase CHMP: Committee for Medicinal Products for Human Use DMD: Duchenne Muscular Dystrophy FA: Friedreich's ataxia FDA: Food and Drug Administration HD: Huntington's Disease MAA: Marketing Authorization Application NDA: New Drug Application nmDMD: nonsense mutation Duchenne Muscular Dystrophy PKU: Phenylketonuria PRV: Priority Review Voucher R&D: Research and Development SG&A: Selling, General and Administrative About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company that discovers, develops and commercializes clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. To learn more about PTC, please visit us at and follow us on Facebook, Instagram, LinkedIn and X. For More Information: Investors: Ellen Cavaleri +1 (615) 618-6228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statement This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "2025 Financial Guidance", including with respect to (i) 2025 total revenue guidance and (ii) 2025 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia, the European Economic Area (EEA) and other regions, including whether the European Commission adopts the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) for the conditional marketing authorization for Translarna in the EEA, or PTC's ability to identify other potential mechanisms by which it may provide Translarna to nmDMD patients in the EEA; PTC's ability to use the clinical data from its international drug registry study and real-world evidence concerning Translarna's benefits to support a continued marketing authorization for Translarna for the treatment of nmDMD in the EEA; PTC's ability to use the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from its trials in Translarna; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation including its right to receive any upfront payment, development, regulatory and sales milestones, profit sharing and royalty payments from Novartis; expectations with respect to Kebilidi and Upstaza, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to sepiapterin, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of regulatory and sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna, Emflaza, Kebilidi, Upstaza, Evrysdi, Tegsedi, Waylivra, sepiapterin or vatiqunone. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 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