Risdiplam

– Strong revenue performance, supporting increase in full-year revenue guidance to $750-800 million – – Regulatory filings for sepiapterin, Translarna™ and AADC gene therapy under review by FDA – – On track to achieve remaining 2024 clinical and regulatory milestones, including NDA submission for vatiquinone – WARREN, N.J., Nov. 7, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the third quarter ending September 30, 2024. "I am proud of our team's strong execution," said Matthew Klein, M.D., Chief Executive Officer. "We continue to achieve excellent revenue performance allowing us to raise full-year revenue guidance. In addition, we have submitted three approval applications to FDA so far this year, all of which have been accepted for review, and plan a fourth submission for vatiquinone for Friedreich ataxia in December. We are on our way to accomplishing all of our 2024 objectives and are in a strong financial position to execute on our long-term strategic plans." Key Corporate Updates: Third quarter 2024 total revenue of $197 million Third quarter 2024 revenue for the DMD franchise was $124 million Translarna™ (ataluren) net product revenue was $72 million, driven by continued access for patients in existing geographies, continued geographic expansion, high compliance and adherence, and dose adjustments. Emflaza® (deflazacort) net product revenue was $52 million, driven by new patient starts, continued brand loyalty, high compliance and adherence, and dose adjustments. Key Clinical and Regulatory Milestones: PTC submitted an NDA to the FDA for sepiapterin for the treatment of PKU. The file was accepted for review, and the regulatory action date is set for July 29, 2025. PTC resubmitted the NDA for Translarna for the treatment of nmDMD; the file was accepted, and a regulatory action date was not provided. The BLA for PTC's gene therapy for AADC deficiency remains on schedule for a regulatory action date of November 13, 2024. PTC received Fast Track Designation for the PTC518 Huntington's disease program. PTC plans to submit an NDA for vatiquinone for the treatment of Friedreich ataxia in December 2024. PTC expects to share topline data for the CardinALS trial of utreloxastat for the treatment of ALS in the fourth quarter of 2024. Third Quarter 2024 Financial Highlights: Total revenues were $196.8 million for the third quarter of 2024, compared to $196.6 million for the third quarter of 2023. Total revenue includes net product revenue across the commercial portfolio of $135.4 million for the third quarter of 2024, compared to $144.0 million for the third quarter of 2023. Total revenue also includes royalty and manufacturing revenue of $61.4 million in the third quarter of 2024, compared to $52.5 million for the third quarter of 2023. Translarna net product revenues were $72.3 million for the third quarter of 2024, compared to $69.0 million for the third quarter of 2023. Emflaza net product revenues were $51.9 million for the third quarter of 2024, compared to $67.4 million for the third quarter of 2023. Roche reported Evrysdi® 2024 year-to-date sales of approximately CHF 1.2 billion, resulting in royalty revenue of $61.4 million to PTC for the third quarter of 2024, compared to $50.2 million for the third quarter of 2023. Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $161.4 million for the third quarter of 2024, compared to $164.2 million for the third quarter of 2023. The decrease in research and development expenses reflects strategic portfolio prioritization, as PTC continues to focus its resources on its differentiated, high-potential R&D programs. Included in third quarter 2024 R&D expense is $50.0 million in regulatory success-based milestones related to the Censa acquisition. Non-GAAP R&D expenses were $152.0 million for the third quarter of 2024, excluding $9.4 million in non-cash, stock-based compensation expense, compared to $150.2 million for the third quarter of 2023, excluding $14.0 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $73.5 million for the third quarter of 2024, compared to $80.9 million for the third quarter of 2023. The decrease in selling, general and administrative expenses reflects lower employee costs as a result of the reduction in workforce in 2023. Non-GAAP SG&A expenses were $63.1 million for the third quarter of 2024, excluding $10.3 million in non-cash, stock-based compensation expense, compared to $67.9 million for the third quarter of 2023, excluding $13.0 million in non-cash, stock-based compensation expense. Change in the fair value of deferred and contingent consideration was a loss of $0.7 million for the third quarter of 2024, compared to a loss of $1.5 million for the third quarter of 2023. Tangible asset impairment and losses (gains) on transactions, net, was a loss of $1.8 million for the third quarter of 2024, compared to $0.0 million for the third quarter of 2023. These results were primarily related to fixed asset impairments. Net loss was $106.7 million for the third quarter of 2024, compared to net loss of $133.0 million for the third quarter of 2023. Cash, cash equivalents, and marketable securities was $1,013.4 million on September 30, 2024, compared to $876.7 million on December 31, 2023. Shares issued and outstanding as of September 30, 2024, were 76,952,124. PTC Updates Full Year 2024 Financial Guidance: PTC anticipates total revenues for full year 2024 to be between $750 million and $800 million. PTC anticipates GAAP R&D and SG&A expenses for full year 2024 to be between $740 million and $835 million, including expected R&D expense milestone payments of up to $65 million. PTC anticipates non-GAAP R&D and SG&A expenses for full year 2024 to be between $660 million and $755 million, including expected R&D expense milestone payments of up to $65 million and excluding estimated non-cash, stock-based compensation expense of $80 million. PTC anticipates up to $90 million of payments for full year 2024 upon achievement of potential regulatory success-based milestones from previous acquisitions, of which up to $65 million will be recorded as R&D operating expenses. As of September 30, 2024, $85 million in milestones have been achieved, of which $65 million has been recorded as R&D operating expenses and $20 million as contingent consideration payable. Non-GAAP Financial Measures: In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense 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 l-Amino Acid Decarboxylase ALS: Amyotrophic Lateral Sclerosis BLA: Biologics License Application CHF: Confoederatio Helvetica Francs (Swiss francs) DMD: Duchenne Muscular Dystrophy FA: Friedreich Ataxia FDA: U.S. Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's Disease NDA: New Drug Application nmDMD: Nonsense Mutation Duchenne Muscular Dystrophy PKU: Phenylketonuria R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder: To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at . A replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days following the call. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit us at and follow us on Facebook, X, and LinkedIn. For More Information: Investors: Ellen Cavaleri +1 (615) 618-6228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: 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 "PTC Updates Full Year 2024 Financial Guidance", including with respect to (i) 2024 total revenue guidance, (ii) 2024 GAAP and non-GAAP R&D and SG&A expense guidance and (iii) 2024 acquisition related milestone payment 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; the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce; 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 Upstaza, including any regulatory submissions and potential approvals, commercialization, manufacturing capabilities, the potential achievement of development, regulatory and 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, the potential achievement of development, 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; the timing of and actual expenses incurred in connection with the discontinuation of PTC's preclinical and early research programs in gene therapy and reductions in workforce, which may be in different periods and may be materially higher than estimated; the savings that may result from the discontinuation of PTC's strategic pipeline prioritization and reductions in workforce, which may be materially less than expected; 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, Upstaza, Evrysdi, Tegsedi, Waylivra or sepiapterin. 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. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Group sales grew by 6%1 at constant exchange rates (CER) (2% in CHF) in the first nine months, driven by the high demand for both our medicines and diagnostics; excluding COVID-19-related products, sales increased by 8%In the third quarter, Group sales rose by 9% (6% in CHF), as they did in the second quarter Pharmaceuticals Division sales rose by 7% in the first nine months; the strong growth of 9% in the base business2 was driven by continued high demand for our newer medicines to treat severe diseases; Vabysmo (serious eye diseases), Phesgo (breast cancer) and Ocrevus (multiple sclerosis) were major growth driversDiagnostics Division sales increased by 5% in the first nine months, while the base business2 grew by 8% due to higher demand for immunodiagnostic, pathology and molecular solutionsHighlights: US approval for Itovebi (inavolisib) for breast cancer, Ocrevus Zunovo subcutaneous injection for multiple sclerosis and Tecentriq Hybreza subcutaneous formulation for various types of cancerEU approval for Vabysmo for retinal vein occlusion (RVO), a serious eye disease, and PiaSky for paroxysmal nocturnal haemoglobinuria (PNH), a rare life-threatening blood conditionPositive phase III data for Gazyva/Gazyvaro (lupus nephritis, a kidney disease), Xofluza (influenza) and Tecentriq (lung cancer). New positive phase II data for fenebrutinib (multiple sclerosis), and new positive long-term data for Evrysdi (spinal muscular atrophy)Acquired AntlerA Therapeutics for a novel target in ophthalmology, and signed agreement for the acquisition of two next-generation CDK inhibitor drugs targeting breast cancer from Regor PharmaceuticalsClosing of acquisition of LumiraDx’s point-of-care technology to expand access to diagnostic testing in primary care and low- and middle-income countriesLaunch of the cobas Respiratory flex test, the first to use our new Temperature-Activated Generation of Signal (TAGS) technologyWHO endorsement for CINtec PLUS testing for cervical cancer prevention Outlook for 2024 confirmed Roche CEO Thomas Schinecker: “Our strong growth momentum continued in the third quarter, reflecting the high demand for our innovative medicines and diagnostic solutions and their positive impact on patients’ lives around the world. We made significant progress in our pharmaceuticals portfolio in the last quarter with five important regulatory approvals for our medicines, three positive phase III read-outs, and two acquisitions to strengthen our oncology and ophthalmology pipelines. Itovebi (inavolisib) recently received US approval based on clinical data demonstrating a reduction of more than 50% in the risk of death or worsening disease for people suffering from a form of advanced, hard-to-treat breast cancer. In addition, we had positive phase III results for Gazyva/Gazyvaro in lupus nephritis, a potentially life-threatening kidney disease for which limited treatment options are available today. We confirm our outlook for 2024.” Sales CHF millions As % of sales % change January‒September 2024 2023 2024 2023 At CER In CHF Group 44,984 44,053 100.0 100.0 6 2 Pharmaceuticals Division 34,257 33,372 76.2 75.8 7 3 United States 18,166 17,430 40.4 39.6 7 4 Europe 6,613 6,259 14.7 14.2 7 6 Japan 2,083 2,937 4.6 6.7 -21 -29 International* 7,395 6,746 16.5 15.3 19 10 Diagnostics Division 10,727 10,681 23.8 24.2 5 0 All figures shown in the table were restated to reflect the shift of the Foundation Medicine (FMI) business from the Pharmaceuticals Division to the Diagnostics Division. *Asia-Pacific, CEETRIS (Central Eastern Europe, Türkiye, Russia and Indian subcontinent), Latin America, Middle East, Africa, Canada, others Outlook for 2024 confirmed Roche expects an increase in Group sales in the mid single digit range (CER). Core earnings per share are targeted to grow in the high single digit range (CER), excluding the impact from the resolution of tax disputes in 2023. Roche expects to further increase its dividend in Swiss francs. Group salesIn the first nine months of 2024, Group sales increased by 6% at CER (2% in CHF) to CHF 45.0 billion as strong demand for our novel medicines as well as diagnostic products including immunodiagnostic, pathology and molecular solutions more than offset the anticipated decline in COVID-19-related sales and the impact of biosimilar/generic erosion. The appreciation of the Swiss franc against most currencies had an adverse impact on the sales reported in Swiss francs compared to constant exchange rates. The Pharmaceuticals Division sales increased by 7% to CHF 34.3 billion, while the base business (excluding COVID-19) grew by 9%, driven primarily by higher sales of Vabysmo (severe eye diseases), Phesgo (breast cancer), Ocrevus (multiple sclerosis), Hemlibra (haemophilia) and Polivy (blood cancer). These five medicines together generated total sales of CHF 13.2 billion, an increase of CHF 2.7 billion (CER) from the first nine months of 2023. The eye medicine Vabysmo, launched in early 2022, continues to be a major growth driver, generating sales of CHF 2.8 billion on growing demand in all regions. Sales of Avastin (various types of cancer), Herceptin (breast and gastric cancer) and MabThera/Rituxan (blood cancer, rheumatoid arthritis) decreased by a combined CHF 0.5 billion as the impact of biosimilar competition slowed further. Sales of the COVID-19 medicine Ronapreve were negligible compared with CHF 0.5 billion in the first nine months of 2023. In the United States, sales grew by 7% as strong sales of Vabysmo, Ocrevus, Polivy and Xolair (food allergies) were partially offset by the continued decline in sales of medicines for which patent protection has expired. Vabysmo achieved CHF 2.1 billion in sales, showing a high uptake in both new patients and patients switching from other medications. In Europe, sales rose by 7%, driven by demand for Vabysmo as well as by the continued uptake of Phesgo, Ocrevus, Evrysdi (spinal muscular atrophy) and Hemlibra. This was partially offset by lower sales of medicines for which patent protection has expired and of Perjeta (breast cancer) due to the ongoing conversion of patients to Phesgo. Sales in Japan were down 21%, mainly due to the base effect of the supply of Ronapreve (COVID-19) to the government in the first quarter of 2023. Excluding this effect, sales in Japan were 3% lower as strong demand for Phesgo and Vabysmo was more than offset by the impact of government price cuts and lower sales of medicines for which patent protection has expired. Sales in the International region surged by 19%, led by demand for Perjeta, Hemlibra, Tecentriq (cancer immunotherapy), Phesgo and Ocrevus as well as the launch of Elevydis (gene therapy, Duchenne muscular dystrophy). Sales in China increased by 8%, driven by Xofluza, Perjeta, Polivy and Avastin. The Diagnostics Division sales increased by 5% to CHF 10.7 billion, while the base business (excluding COVID-19) grew by 8%. Immunodiagnostic products, which include cardiac, oncology and thyroid tests, were the main growth drivers (10%). Additional growth came from pathology and molecular solutions. Sales of COVID-19 tests were CHF 0.1 billion in the first nine months of 2024 compared with CHF 0.4 billion in the corresponding period last year. Sales growth was reported across regions, with the Europe, Middle East and Africa (EMEA) region growing by 5%, North America by 6%, Asia-Pacific by 2% and Latin America by 18%. Pharmaceuticals: key developments Compound Milestone Regulatory Itovebi (inavolisib)Breast cancer FDA approves Itovebi, a targeted treatment for advanced hormone receptor (HR)-positive, HER2-negative breast cancer with a PIK3CA mutation Approval is based on phase III INAVO120 results, showing the regimen based on Itovebi (inavolisib) more than doubled progression-free survival compared with palbociclib and fulvestrant alone in the first-line settingThis approval helps address an urgent unmet need in breast cancer for people with a mutated PIK3CA gene, one of the most commonly mutated genes in HR-positive disease and which is associated with poor prognosisItovebi is Roche’s first targeted therapy approved for people with HR-positive disease, the most prevalent breast cancer subtype, marking an important step in our ambition to continue bringing innovative medicines to more people with breast cancer More information: Media Release, 11 October 2024 Ocrevus ZunovoMultiple sclerosis FDA approves Ocrevus Zunovo as the first and only twice-a-year 10-minute subcutaneous injection for people with relapsing and progressive multiple sclerosis Ocrevus Zunovo has the potential to expand treatment options to centres without intravenous (IV) infrastructure or with IV constraints, like at a doctor’s officeThis approval is backed by a decade of proven safety and efficacy data of Ocrevus IV, with over 350,000 people treated globallyOcrevus Zunovo offers people with multiple sclerosis more options to access treatment based on their individual needs More information: Media Release, 16 September 2024 Tecentriq Hybreza Various types of cancer FDA approves Tecentriq Hybreza, the first and only subcutaneous anti-PD-(L)1 cancer immunotherapy Tecentriq Hybreza provides patients and physicians with greater flexibility of treatment options while showing safety and efficacy consistent with intravenous (IV) TecentriqNew subcutaneous option reduces treatment time to approximately seven minutes, compared with 30‒60 minutes for IV infusion More information: Media Release, 13 September 2024 PiaSkyRare blood disease PiaSky approved in the EU as the first monthly subcutaneous treatment for people with paroxysmal nocturnal haemoglobinuria (PNH) With the option of self-administration, PiaSky (crovalimab) has the potential to reduce treatment burden for people with PNH and their caregivers in EuropeApproval is based on COMMODORE 2, where subcutaneous (SC) PiaSky once a month was equivalent to intravenous eculizumab every two weeksPiaSky advances C5 inhibition through innovative recycling technology, which enables its monthly SC administration More information: Media Release, 27 August 2024 Vabysmo Severe eye diseases European Commission approves Vabysmo for treatment of retinal vein occlusion (RVO) Approval is based on data from two phase III studies in branch and central retinal vein occlusion (RVO) showing early and sustained vision improvements non-inferior to aflibercept, and robust retinal drying with VabysmoAdditional submitted data shows that up to 60% of people receiving Vabysmo were able to extend treatment intervals to three or four monthsVabysmo is already approved in several countries, including the US and Japan, for RVO and in nearly 100 countries for people with neovascular or ‘wet’ age-related macular degeneration (nAMD) and diabetic macular edema (DME) More information: Media Release, 30 July 2024 Phase III, pivotal and other key readouts EvrysdiSpinal muscular atrophy Majority of children with spinal muscular atrophy (SMA) treated with Evrysdi are able to sit, stand and walk independently, two-year data demonstrate Positive data confirm Evrysdi efficacy and safety in children first treated pre-symptomatically before six weeks of age, with most achieving motor milestones similar to children without SMAAll children were able to swallow and feed orally, with none requiring permanent ventilationEvrysdi is the only non-invasive SMA therapy and is approved in over 100 countries, with more than 16,000 people with SMA treated globally More information: Media Release, 14 October 2024 Gazyva/GazyvaroKidney disease Positive phase III results for Gazyva/Gazyvaro show superiority to standard therapy alone in people with lupus nephritis The REGENCY study met its primary endpoint, demonstrating statistically significant and clinically meaningful treatment benefits in people with active lupus nephritisGazyva/Gazyvaro is designed to target an underlying cause of lupus nephritis, aiming to prevent or delay progression to end-stage kidney diseaseLupus nephritis is a potentially life-threatening manifestation of an autoimmune disease affecting 1.7 million people worldwide, primarily women; up to one-third of people on current treatments will progress to end-stage kidney disease within 10 years More information: Media Release, 26 September 2024 Xofluza Influenza Positive phase III results show Xofluza significantly reduces the transmission of influenza viruses Data from the CENTERSTONE study shows single-dose Xofluza reduces transmission of influenza from an infected person to household membersThis is the first time that any antiviral used in the treatment of a respiratory viral illness has demonstrated a transmission reduction benefit in a global phase III studyReducing the spread of infection in the household could help limit transmission within communities and societies, easing the burden of both seasonal and pandemic influenza on healthcare systems More information: Media Release, 19 September 2024 Fenebrutinib Multiple sclerosis Fenebrutinib demonstrated near-complete suppression of disease activity and disability progression for up to 48 weeks in patients with relapsing multiple sclerosis New phase II data show vast majority of patients experiencing no relapses or disability progressionFenebrutinib suppressed acute and chronic MRI lesionsFenebrutinib’s safety profile was consistent with previous and ongoing clinical trials across multiple diseases including more than 2,700 people to date More information: Media Release, 4 September 2024 Susvimo Severe eye disease New data for Susvimo demonstrates sustained efficacy in two serious diabetic eye conditions Two-year phase III data presented at ASRS 2024 show Susvimo’s potential as an alternative to eye injections to treat diabetic macular edema (DME) and diabetic retinopathy (DR)Safety data were consistent with the known safety profile for Susvimo in people with DME and DRAdditionally, the US FDA has accepted the filing application for Susvimo in DME and DR based on one-year Pagoda and Pavilion study data More information: Media Release, 18 July 2024 Other Pharma Research and Early Development Center Roche opens Pharma Research and Early Development Center in Basel to accelerate scientific innovation Switzerland’s most innovative research and development centre underscores Roche’s long-term investment in scientific advancement to meet patient needsThe new centre will simplify and increase collaboration, thereby accelerating scientific innovation More information: Media Release, 10 September 2024 Pharmaceuticals sales Sales CHF millions As % of sales % change January–September 2024 2023 2024 2023 At CER In CHF Pharmaceuticals Division 34,257 33,372 100.0 100.0 7 3 United States 18,166 17,430 53.0 52.2 7 4 Europe 6,613 6,259 19.3 18.8 7 6 Japan 2,083 2,937 6.1 8.8 -21 -29 International* 7,395 6,746 21.6 20.2 19 10 All figures shown in the table were restated to reflect the shift of the Foundation Medicine (FMI) business from the Pharmaceuticals Division to the Diagnostics Division. * Asia-Pacific, CEETRIS (Central Eastern Europe, Türkiye, Russia and Indian subcontinent), Latin America, Middle East, Africa, Canada, others Top-selling medicines Total United States Europe Japan International CHF m % CHF m % CHF m % CHF m % CHF m % Ocrevus Multiple sclerosis 5,056 9 3,640 7 961 11 - - 455 26 Hemlibra Haemophilia A 3,280 10 1,906 5 690 10 259 4 425 42 Vabysmo Eye diseases (nAMD, DME, RVO) 2,816 79 2,146 67 454 148 86 37 130 256 Perjeta3 Breast cancer 2,809 -1 1,029 -4 502 -17 92 -36 1,186 17 Tecentriq Cancer immunotherapy 2,703 1 1,325 -8 649 5 277 -1 452 32 Actemra/RoActemra3 RA, COVID-19 1,948 5 949 11 508 -11 225 9 266 17 Xolair3 Asthma 1,737 11 1,737 11 - - - - - - Kadcyla3 Breast cancer 1,494 6 574 4 428 -2 71 4 421 22 Evrysdi Spinal muscular atrophy 1,246 21 429 15 435 18 66 10 316 35 Phesgo Breast cancer 1,244 58 404 29 543 44 88 - 209 104 Alecensa Lung cancer 1,151 7 372 12 217 1 144 3 418 8 Herceptin3 Breast and gastric cancer 1,063 -11 201 -20 227 -15 11 -48 624 -5 MabThera/Rituxan3 Blood cancer, RA 1,023 -16 615 -17 109 -21 12 -26 287 -10 Avastin 3 Various cancer types 943 -17 289 -20 63 -17 149 -33 442 -8 Activase/TNKase3 Cardiac diseases 895 2 850 1 - - - - 45 5 Polivy Blood cancer 817 41 410 83 142 6 143 -4 122 79 Gazyva/Gazyvaro3 Blood cancer 670 13 333 15 185 8 21 -16 131 21 Pulmozyme3 Cystic fibrosis 329 0 213 -5 55 -3 1 17 60 23 Mircera3 Anaemia related to kidney disease 304 -1 - - 31 -6 28 -23 245 4 CellCept3 Immunosuppressant 283 1 17 -21 81 -13 27 -9 158 17 DME: diabetic macular edema / nAMD: neovascular or ‘wet’ age-related macular degeneration / RVO: retinal vein occlusion / RA: rheumatoid arthritis Diagnostics: key developments Product Milestone VENTANA CLDN18 assay Gastric or gastro-oesophageal junction cancer Roche obtains CE certification for the first companion diagnostic to identify patients with gastric and gastro-oesophageal junction cancer eligible for targeted treatment with VYLOY The new VENTANA CLDN18 (43-14A) RxDx assay helps fulfil an unmet medical need by enabling clinicians to identify patients with gastric or gastro-oesophageal junction (GOJ) cancer who may benefit from a targeted treatment optionCLDN18.2 is an emerging biomarker in gastric and GOJ cancers and helps predict the likelihood of response to targeted therapyAs the leader in companion diagnostics, Roche continues to build on its commitment to improve personalised healthcare to enable better patient outcomes More information: Media Release, 10 October 2024 cobas Respiratory flex testRespiratory illnesses Roche launches the first test to use its breakthrough TAGS technology for high-throughput, simultaneous detection of 12 respiratory viruses The new Temperature-Activated Generation of Signal (TAGS) technology enables up to 15 targets to be detected simultaneously in a single patient sample on the high-throughput molecular diagnostic analysers cobas 5800, 6800 and 8800TAGS has the potential to revolutionise testing for other infectious diseases in the future, by bringing high-throughput customised syndromic panel testing to the routine clinical laboratoryThe first TAGS-based test to be made available, the cobas Respiratory flex, offers fast, efficient detection of up to 12 of the most common respiratory viruses with the flexibility for targeted testing, expediting accurate diagnosis, optimising antimicrobial use and saving time in the lab More information: Media Release, 24 September 2024 CINtec PLUS Cervical cancer WHO endorses dual-stain cytology (CINtec PLUS) testing in its cervical cancer prevention guidelines, advancing patient care and underlining Roche’s role in pioneering cervical cancer solutions CINtec PLUS Cytology is the only FDA-approved and CE-marked dual-stain test to triage human papillomavirus (HPV)-positive cervical cancer screening test resultsDual-stain biomarkers aid in detection of cervical precancer and may reduce the number of women who undergo unnecessary colposcopy procedures while allowing earlier intervention for those who are at higher risk of developing cervical cancerThis recognition follows the American Society for Colposcopy and Cervical Pathology (ASCCP)’s recent inclusion of dual-stain testing in cervical cancer screening guidelines, as well as other WHO prequalifications of Roche’s cobas HPV test More information: Media Release, 23 September 2024 cobas MPXV test, LightMix Mpox Roche responds to WHO’s declaration of a global health emergency due to the ongoing mpox outbreak Roche is committed to supporting all those working to overcome the mpox outbreak by providing access to high-quality polymerase chain reaction (PCR) testingRoche confirms that its cobas MPXV test and the LightMix research-use-only kits detect the latest mpox virus variantsWe are actively working to enhance laboratory testing capacity for mpox worldwide More information: Media Release, 20 August 2024 LumiraDx Roche closes acquisition of LumiraDx’s point-of-care technology to expand access to diagnostic testing in primary care The acquisition of LumiraDx’s point-of-care technology received all required antitrust and regulatory clearancesLumiraDx’s transformative point-of-care solution will complement Roche’s diagnostics portfolio across clinical chemistry, immunochemistry, coagulation and molecular, and across multiple disease areasBy integrating LumiraDx, Roche will further its ambition to deliver decentralised solutions that expand global access to testing in primary care settings worldwide More information: Media Release, 29 July 2024 Diagnostics sales Sales CHF millions As % of sales % change January–September 2024 2023 2024 2023 At CER In CHF Diagnostics Division 10,727 10,681 100.0 100.0 5 0 Customer Areas4 Core Lab 6,052 5,836 56.4 54.6 9 4 Molecular Lab5 1,903 1,897 17.7 17.8 4 0 Near Patient Care6 1,616 1,902 15.1 17.8 -10 -15 Pathology Lab 1,156 1,046 10.8 9.8 15 11 Regions Europe, Middle East and Africa 3,589 3,569 33.5 33.4 5 1 North America5 3,222 3,103 30.0 29.1 6 4 Asia-Pacific 3,146 3,263 29.3 30.5 2 -4 Latin America 770 746 7.2 7.0 18 3 More information on Roche sales in the first nine months of 2024: Q3 2024 presentationAppendix with tables About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in diagnostics and pharmaceuticals with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com. All trademarks used or mentioned in this release are protected by law. References [1] Unless otherwise stated, all growth rates and comparisons to the previous year in this document are at constant exchange rates (CER: average rates 2023) and all total figures quoted are reported in CHF. [2] Pharmaceuticals Division base business: excluding COVID-19 medicine Ronapreve. Diagnostics Division base business: excluding COVID-19-related products. [3] Products launched before 2015. [4] Core Lab: diagnostics solutions in the areas of immunoassays, clinical chemistry and CustomBiotech. Molecular Lab: diagnostics solutions for pathogen detection and monitoring, donor screening, sexual health and genomics, genomic tumour profiling. Near Patient Care: diagnostics solutions in emergency rooms, medical practices and directly with patients, including integrated personalised diabetes management. Pathology Lab: diagnostics solutions for tissue biopsies and companion diagnostics. [5] Sales in the Molecular Lab customer area include sales from the Foundation Medicine business, which moved under the responsibility of the Diagnostics Division from the Pharmaceuticals Division effective 1 January 2024. The comparative information for 2023 has been restated accordingly. [6] Sales in the new Near Patient Care customer area include sales from Diabetes Care and the Point of Care business, both previously shown as separate customer areas. The comparative information for 2023 has been restated accordingly. Cautionary statement regarding forward-looking statements This document contains certain forward-looking statements. These forward-looking statements may be identified by words such as ‘believes’, ‘expects’, ‘anticipates’, ‘projects’, ‘intends’, ‘should’, ‘seeks’, ‘estimates’, ‘future’ or similar expressions or by discussion of, among other things, strategy, goals, plans or intentions. Various factors may cause actual results to differ materially in the future from those reflected in forward-looking statements contained in this document, such as: (1) pricing and product initiatives of competitors; (2) legislative and regulatory developments and economic conditions; (3) delay or inability in obtaining regulatory approvals or bringing products to market; (4) fluctuations in currency exchange rates and general financial market conditions; (5) uncertainties in the discovery, development or marketing of new products or new uses of existing products, including without limitation negative results of clinical trials or research projects, unexpected side effects of pipeline or marketed products; (6) increased government pricing pressures; (7) interruptions in production; (8) loss of or inability to obtain adequate protection for intellectual property rights; (9) litigation; (10) loss of key executives or other employees; and (11) adverse publicity and news coverage. The statement regarding earnings per share growth is not a profit forecast and should not be interpreted to mean that Roche’s earnings or earnings per share for this or any subsequent period will necessarily match or exceed the historical published earnings or earnings per share of Roche. Roche Global Media Relations Phone: +41 61 688 8888 / e-mail: media.relations@roche.com Hans Trees, PhD Phone: +41 79 407 72 58 Sileia Urech Phone: +41 79 935 81 48 Nathalie Altermatt Phone: +41 79 771 05 25 Lorena Corfas Phone: +41 79 568 24 95 Simon Goldsborough Phone: +44 797 32 72 915 Karsten Kleine Phone: +41 79 461 86 83 Nina Mählitz Phone: +41 79 327 54 74 Kirti Pandey Phone: +49 172 6367262 Yvette Petillon Phone: +41 79 961 92 50 Dr Rebekka Schnell Phone: +41 79 205 27 03 Roche Investor Relations Dr Bruno Eschli Phone: +41 61 68-75284 e-mail: bruno.eschli@roche.com Dr Sabine Borngräber Phone: +41 61 68-88027 e-mail: sabine.borngraeber@roche.com Dr Birgit Masjost Phone: +41 61 68-84814 e-mail: birgit.masjost@roche.com Investor Relations North America Loren Kalm Phone: +1 650 225 3217 e-mail: kalm.loren@gene.com Attachments 23102024_Roche Q32023 appendix tables 23102024_Roche Media Release Q32024_EN