A Randomized, Placebo-Controlled, Four-Period Crossover, Study to Evaluate the Effect of Volanesorsen on the QTc Interval Using a Therapeutic and Supra-Therapeutic Dose Compared With Placebo in Healthy Volunteers: a Thorough QT Study

The primary objective of this study is to assess the corrected QT interval (QTc) effect of volanesorsen (ISIS 304801) administered as a 300 mg subcutaneous (SC) therapeutic and a 300 mg intravenous (IV; 2-hour infusion) supra-therapeutic dose relative to placebo in healthy adult male and female subjects.

Start Date19 Sep 2016

Sponsor / Collaborator

Ionis Pharmaceuticals, Inc.

[+1]

100 Clinical Results associated with Volanesorsen

100 Translational Medicine associated with Volanesorsen

100 Patents (Medical) associated with Volanesorsen

Literatures (Medical) associated with Volanesorsen

01 Jul 2026·Journal of Comparative Effectiveness Research

Comparative efficacy and safety of olezarsen versus volanesorsen for familial chylomicronemia syndrome: a matching-adjusted indirect comparison

Article

Author: Hakimi, Zalmai ; Kassner, Ursula ; Soran, Handrean ; Serafini, Paul ; Wilson, Koo ; Alexander, Veronica J ; Pushkarna, Divya ; Falk, Aletta ; Stroes, Erik ; Rodrigues, Elisabete ; Vera-Llonch, Montserrat ; Kessler, Asia Sikora ; Schatz, Ulrike

Background: Familial chylomicronemia syndrome (FCS) is a rare genetic disorder characterized by severe hypertriglyceridemia causing recurrent acute pancreatitis (AP). Since FCS is a genetic deficiency in functional lipoprotein lipase, conventional triglyceride-lowering therapies are ineffective in this metabolic disorder. Apolipoprotein C-III (apoC-III) inhibitors, including volanesorsen and olezarsen, have emerged as targeted treatments for FCS. Aim: To compare the efficacy and safety of olezarsen 80 mg every four weeks (Q4W) versus volanesorsen 300 mg weekly (QW) in patients with FCS using an anchored matching-adjusted indirect comparison. Materials & methods: Individual patient data from the Balance trial of olezarsen (n = 45) were weighted to match baseline characteristics reported in the APPROACH trial of volanesorsen (n = 66). Outcomes included percent change in fasting triglycerides (TG) and apoC-III at 26 and 52 weeks, and risks of AP events and adverse events at 52 weeks. Results: At 52 weeks, mean differences in fasting TG and apoC-III were -27.4% (95% CI: -69.4, 14.5) and -21.3% (95% CI: -61.9, 19.3). Relative risks of AP, treatment-emergent adverse events, and serious adverse events were 0.23 (95% CI: 0.01, 5.02), 0.87 (95% CI: 0.66, 1.13) and 0.50 (95% CI: 0.08, 3.26) at week 52. The rate ratio for AP events per patient-year was 0.06 (95% CI: 0.003, 1.41). No comparisons were statistically significant. Conclusion: In this matching-adjusted indirect comparison, no statistically significant differences in outcomes were observed between olezarsen and volanesorsen in patients with FCS. These findings provide important comparative context in a setting where head-to-head evidence is unavailable.

01 Jun 2026·DIABETES OBESITY & METABOLISM

Efficacy and Safety of Apolipoprotein C‐ III Inhibitors in Hypertriglyceridemia: A Network Meta‐Analysis of Randomised Controlled Trials

Review

Author: Elazab, Ahmed ; Elbahloul, Mohammed A. ; Elgendy, Islam Y. ; Gamal, Aliaa ; Awad, Ayman K. ; Attia, Manar Khaled ; Elbaz Hassan, Moustafa M. ; Salah, Ahmed M. ; Elboraay, Toka ; Akoum, Atef ; Maihoub, Odai

ABSTRACT:

Background:

Hypertriglyceridemia is an independent risk factor for atherosclerotic cardiovascular disease (ASCVD). We aimed to evaluate the efficacy and safety of apolipoprotein C‐III (ApoC‐III) inhibitors in patients with hypertriglyceridemia.

Methods:

Electronic databases were systematically searched for randomised controlled trials (RCTs) comparing ApoC‐III inhibitors (Volanesorsen, Olezarsen and Plozasiran) with placebo. A frequentist network meta‐analysis was performed. Continuous outcomes were presented as mean differences (MDs), and dichotomous outcomes as risk ratios (RRs), both with 95% confidence intervals (CIs).

Results:

A total of 15 RCTs involving 3934 patients were included. All ApoC‐III inhibitors demonstrated significant reductions in TG, except for Volanesorsen 100 mg Q1W and Olezarsen 10 mg Q4W. Olezarsen 80 mg administered every 4 weeks had the highest SUCRA ranking for TG reduction (MD: −63.26; 95% CI: −70.04 to −56.47; p < 0.01). Moreover, ApoC‐III inhibitors were associated with a significant reduction in the incidence of pancreatitis (HR: 0.16, 95% CI: 0.07–0.33, p < 0.01). None of the agents significantly increased the risk of serious adverse events.

Conclusions:

ApoC‐III inhibitors significantly improved triglyceride levels and other lipid parameters and, most importantly, substantially reduced the risk of acute pancreatitis. Future trials are needed to evaluate their effects on cardiovascular outcomes.

04 May 2026·Expert Review of Endocrinology & Metabolism

The evaluation of plozasiran for the treatment of familial chylomicronemia syndrome

Review

Author: Hang, Shyann ; Hegele, Robert A.

INTRODUCTION:

Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive disorder marked by severe hypertriglyceridemia and characteristic clinical manifestations, particularly acute pancreatitis. Conventional triglyceride-lowering therapy is largely ineffective. Apolipoprotein (apo) C-III has emerged as a key therapeutic target to lower triglycerides in FCS.

AREAS COVERED:

This review compares FCS with more common multifactorial chylomicronemia. We searched PubMed for all English language literature focusing on the search terms 'chylomicronemia,' 'hypertriglyceridemia,' 'APOC3 inhibition,' 'plozasiran,' 'olezarsen,' and 'volanesorsen.' We outline traditional management strategies and their limited role in FCS and explore non-traditional therapies including orlistat, lomitapide, inhibitors of angiopoietin like protein 3 (ANGPTL3), and analogues of fibroblast growth factor 21 (FGF21). The primary focus is on RNA-based gene silencing therapeutics that target apo C-III, particularly the small interfering RNA plozasiran and the allele specific oligonucleotides volanesorsen and olezarsen, highlighting key differences in efficacy and tolerability.

EXPERT OPINION:

In a phase 3 trial of plozasiran, at 10 months, median placebo-adjusted reductions in apo C-III were approximately -90%, while TG levels were reduced up to -59%. Thus, plozasiran and alternative RNA-based therapeutics directed against APOC3 represent transformational therapies for patients with FCS and related phenotypes characterized by severe recalcitrant hypertriglyceridemia.

News (Medical) associated with Volanesorsen

03 Aug 2026

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results

– Robust Q2 performance with total revenue of $361 million, including $239 million of product revenue – – Full-year 2026 expected total revenue increased to $1.18 to $1.28 billion, with full-year product revenue guidance raised to $850 to $950 million – – Sephience™ (sepiapterin) Q2 2026 revenue of $151 million, with continued broad uptake and geographical expansion – – Strong cash position of $2.2 billion as of June 30, 2026 – WARREN, N.J., July 30, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ended June 30, 2026. "Our teams delivered another strong quarter enabling us to again raise 2026 product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "We also made a number of advances in our R&D pipeline, including identification of our next small molecule splicing development candidate, PTC303, targeting somatic expansion disorders including Huntington's disease and myotonic dystrophy. In addition, we initiated the Phase 1 study of PTC612, our differentiated NLRP3 small molecule." Key Corporate Updates Q2 2026 total revenue of $361 million, including $239 million of product revenue Sephience global launch continues with strong momentum Q2 2026 revenue of $151 million, with the majority from the US and increasing contributions internationally 1,647 patients on commercial therapy worldwide as of June 30, 2026 Positive results from votoplam PIVOT-HD long-term extension study reported and enrollment in global Phase 3 INVEST-HD study ongoing PIVOT-HD 24-month results demonstrated dose-dependent disease slowing on cUHDRS scale in Stage 2 participants, reaching 52% at 10 mg dose level Plan to engage with FDA in 2H 2026 to discuss PIVOT-HD 24-month results being finalized INVEST-HD study being conducted and funded by partner Novartis with target enrollment of approximately 770 individuals with early symptomatic disease INVEST-HD initiation triggered a $50 million milestone payment from Novartis to PTC in Q2 2026 Vatiquinone PROVE-FA study to support NDA resubmission for treatment of Friedreich's ataxia expected to initiate in Q3 2026 Global study will enroll approximately 120 individuals with Friedreich's ataxia age 7 to 21 Study includes open-label treatment arm with matched natural history control group PTC pipeline continues to advance PTC303 named as MSH3 splicing program development candidate targeting Huntington's disease and myotonic dystrophy with plan to enter clinic in 2027 Phase 1 study of PTC612, an oral NLRP3 inhibitor, initiated in Q2 2026 Phase 2a study of PTC844, a next-generation DHODH inhibitor, expected to initiate in Q3 2026 Hege Sollie-Zetlmayer has been appointed to PTC's Board of Directors. Ms. Sollie-Zetlmayer has over 30 years of experience in the biopharmaceutical and medical device industry as a global business operations and human resources leader. She most recently served as PTC's Chief Human Resources Officer prior to her retirement in June 2026. Second Quarter 2026 Financial Highlights Total net product revenue and royalty revenue was $309.9 million for the second quarter of 2026, compared to $175.9 million for the second quarter of 2025. Total net product revenue across the commercial portfolio was $238.8 million for the second quarter of 2026, compared to $118.3 million for the second quarter of 2025, representing over 100% increase. Sephience net product revenues were $151.3 million for the second quarter of 2026, representing 21% growth compared to the first quarter of 2026. Translarna™ (ataluren) net product revenues were $42.2 million for the second quarter of 2026, compared to $59.5 million for the second quarter of 2025. Emflaza® (deflazacort) net product revenues were $24.6 million for the second quarter of 2026, compared to $36.4 million for the second quarter of 2025, due to continued generic erosion. Roche reported Evrysdi® (risdiplam) year-to-date sales of approximately 968 CHF million, resulting in royalty revenue of $71.1 million to PTC for the second quarter of 2026, compared to $57.6 million to PTC for the second quarter of 2025. In the second quarter of 2026, PTC recorded a development milestone of $50.0 million from Novartis for the first patient dosed in the ongoing INVEST-HD Phase 3 study. This sales milestone was recorded as collaboration revenue. Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $99.2 million for the second quarter of 2026, compared to $113.0 million for the second quarter of 2025. Non-GAAP R&D expenses were $88.6 million for the second quarter of 2026, excluding $10.5 million in non-cash, stock-based compensation expense, compared to $104.0 million for the second quarter of 2025, excluding $9.0 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $80.6 million for the second quarter of 2026, compared to $85.3 million for the second quarter of 2025. Non-GAAP SG&A expenses were $67.9 million for the second quarter of 2026, excluding $12.7 million in non-cash, stock-based compensation expense, compared to $75.7 million for the second quarter of 2025, excluding $9.5 million in non-cash, stock-based compensation expense. Net income was $83.5 million for the second quarter of 2026, compared to net loss of $64.8 million for the second quarter of 2025. In June 2026, PTC issued $550.0 million of senior convertible notes due in 2031 at 0% coupon and a conversion price representing a 40% premium over the stock's closing price at the time of the deal. Concurrent with the transaction, PTC repurchased the majority of its 1.5% senior convertible notes due in September 2026. Cash, cash equivalents, and marketable securities were $2,229.1 million on June 30, 2026, compared to $1,945.4 million on December 31, 2025. Shares issued and outstanding as of June 30, 2026, were 83,327,286. PTC Updates Full-Year 2026 Financial Guidance Expected total revenue increased to $1.18 to $1.28 billion, with total product revenue guidance raised to $850 to $950 million from $750 to $850 million GAAP R&D and SG&A expense guidance remains $775 to $815 million Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million 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 accordance with 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 CHF: Confoederatio Helvetica Francs (Swiss francs) cUHDRS: Composite Unified Huntington's Disease Rating Scale DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: US Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease NDA: New Drug Application nmDMD: Nonsense mutation Duchenne muscular dystrophy R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder To access the live webcast, please visit the "Events & Presentations" page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Facebook and Instagram. For more information please contact: Investors: Ellen Cavaleri +1 (615) 618-8228 [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 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total revenue guidance and (ii) 2026 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, meetings with regulatory agencies, 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," "aim," 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; expectations with respect to Sephience, including commercialization and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the US on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, 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 vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; expectations regarding PTC's product candidates, including the timing of clinical trials and studies; 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 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 Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. 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. 21% more press release views with Request a Demo

Phase 1Financial StatementExecutive ChangePhase 2Phase 3

24 Jun 2026

·allsci.com

FDA approves Ionis’ Tryngolza as first drug proven to cut acute pancreatitis risk in hypertriglyceridemia

The US FDA has approved Tryngolza (olezarsen) — making it the first therapy demonstrated to reduce the risk of acute pancreatitis in adults with severe hypertriglyceridemia. The approval, granted to California-based Ionis Pharmaceuticals, addresses a gap that has persisted despite decades of available triglyceride-lowering agents: no prior approved medication had generated sufficient pancreatitis event data in clinical trials to establish a risk reduction claim. Tryngolza is indicated, used alongside dietary modification, to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia, defined as fasting triglyceride levels of at least 500 mg/dL. It is administered as a subcutaneous injection once monthly. The drug received both Priority Review and Breakthrough Therapy designations from the FDA, reflecting the agency’s assessment of its potential to address an unmet clinical need. Olezarsen is an antisense oligonucleotide that targets apolipoprotein C-III (APOC3) messenger RNA in the liver, reducing APOC3 protein levels and thereby enhancing triglyceride clearance from the bloodstream. APOC3 inhibits lipoprotein lipase, the primary enzyme responsible for triglyceride hydrolysis, making it a validated target in severe hypertriglyceridemia. Efficacy and safety data came from two randomized, double-blind, placebo-controlled Phase III trials — NCT05079919 and NCT05552326 — enrolling a combined 1,061 adults with severe hypertriglyceridemia. The average baseline triglyceride level across both trials was 1,116 mg/dL. The primary endpoint in each trial was percent change in fasting triglycerides from baseline to month 6. In the first trial, olezarsen reduced triglycerides by 63% at the 50 mg dose and 72% at the 80 mg dose versus placebo; the second trial reported reductions of 49% and 55%, respectively. An integrated analysis across both trials demonstrated a reduction in acute pancreatitis event rate in the pooled olezarsen group compared with placebo. The most common adverse reactions reported were injection site reactions and liver enzyme elevations. Prescribers are advised to consider liver enzyme monitoring before initiation or dose increases, with dose interruption or reduction recommended if elevations persist. Potential hypersensitivity reactions, including urticaria, facial swelling, and dyspnea, have also been reported. The approval is notable because existing approved triglyceride-lowering therapies — including fibrates, omega-3 fatty acids, and niacin — have not demonstrated a reduction in acute pancreatitis risk in controlled trials. Volanesorsen (Waylivra), an earlier antisense APOC3 inhibitor developed by Ionis and approved in the EU for familial chylomicronemia syndrome, required a risk management program due to thrombocytopenia; olezarsen’s differentiated safety profile positions it as a potentially broader-use option in the severe hypertriglyceridemia population. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Privacy Terms About Us Copyright © 2026. AllSci Corp. All rights reserved.

Clinical ResultPhase 3Drug ApprovalBreakthrough TherapyPriority Review

07 May 2026

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports First Quarter 2026 Financial Results

– First quarter 2026 total revenue of $273 million, including $226 million of product revenue, supporting full-year 2026 guidance raise – – Global Sephience™ (sepiapterin) launch momentum continues with first quarter 2026 revenue of $125 million, representing 36% quarter-over-quarter growth – – Positive topline results from 24-month interim analysis of PIVOT-HD extension study of votoplam, supporting ongoing global Phase 3 INVEST-HD study and potential regulatory interactions – – Open-label vatiquinone registration study to be initiated in Q3 2026 based on FDA feedback – WARREN, N.J., May 7, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ended March 31, 2026. "We are off to a strong start to 2026 with outstanding revenue performance this quarter that supports raising our full-year product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "The Sephience launch continues to be strong, with sustained momentum in the US and growing momentum internationally as more countries contribute to the global launch." Key Corporate Updates Q1 2026 total revenue of $273 million, with $226 million of product revenue Sephience global launch continues strong momentum Q1 2026 revenue of $125 million, including $112 million revenue in the US and $13 million revenue ex-US, up 36% from Q4 2025 1,244 total patients on commercial therapy worldwide as of March 31, 2026 Continued strong cadence of patient start forms in the US, with ~140 per month over past several months, surpassing 1,500 mark in the quarter First commercial sales in Japan in Q1 2026, with revenue expected from up to 30 countries by year-end 2026 Reported positive topline results in April 2026 from 24-month interim analysis of PIVOT-HD long-term extension study of votoplam, an oral small molecule splicing agent Dose-dependent benefit on cUHDRS in Stage 2 participants relative to natural history cohort, with 52% slowing of disease progression at 10 mg dose Continued favorable safety and tolerability with no treatment-related NfL spikes PTC and Novartis will complete data review and align on potential regulatory interactions Findings support ongoing global Phase 3 INVEST‑HD study of votoplam led by Novartis First patient dosed in Phase 3 INVEST-HD study, triggering $50 million milestone payment from Novartis to PTC in Q2 2026 Type C meeting with FDA held in April 2026 to discuss vatiquinone study to support NDA resubmission for Friedreich's ataxia program Based on meeting discussion and written feedback, PTC expects to initiate an open-label study using matched natural history control in Q3 2026 Primary endpoint will be change in mFARS from baseline to 24 months First Quarter 2026 Financial Highlights Total net product revenue and royalty revenue was $272.4 million for the first quarter of 2026, compared to $189.9 million for the first quarter of 2025. Total net product revenue across the commercial portfolio was $225.6 million for the first quarter of 2026, compared to $153.4 million for the first quarter of 2025, representing a 47% increase. Sephience net product revenues were $124.6 million for the first quarter of 2026, representing 36% growth compared to fourth quarter of 2025. Translarna™ (ataluren) net product revenues were $59.0 million for the first quarter of 2026, compared to $86.2 million for the first quarter of 2025. Translarna first quarter of 2026 revenue includes a large government purchase order from Brazil. Emflaza® (deflazacort) net product revenues were $21.5 million for the first quarter of 2026, compared to $47.8 million for the first quarter of 2025, due to continued generic erosion. Roche reported Evrysdi® (risdiplam) sales of approximately 464 CHF million, resulting in royalty revenue of $46.8 million to PTC for the first quarter of 2026, compared to $36.4 million to PTC for the first quarter of 2025. Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $100.9 million for the first quarter of 2026, compared to $109.0 million for the first quarter of 2025. Non-GAAP R&D expenses were $89.7 million for the first quarter of 2026, excluding $11.1 million in non-cash, stock-based compensation expense, compared to $100.3 million for the first quarter of 2025, excluding $8.7 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $86.2 million for the first quarter of 2026, compared to $81.0 million for the first quarter of 2025. Non-GAAP SG&A expenses were $73.9 million for the first quarter of 2026, excluding $12.3 million in non-cash, stock-based compensation expense, compared to $71.6 million for the first quarter of 2025, excluding $9.4 million in non-cash, stock-based compensation expense. Net loss was $2.8 million for the first quarter of 2026, compared to net income of $866.6 million for the first quarter of 2025. Cash, cash equivalents, and marketable securities were $1,892.5 million on March 31, 2026, compared to $1,945.4 million on December 31, 2025. Shares issued and outstanding as of March 31, 2026, were 82,882,024. PTC Updates Full-Year 2026 Financial Guidance Total product revenue guidance raised to $750 to $850 million, with expected total revenue of $1.08 to $1.18 billion GAAP R&D and SG&A expense guidance remains $775 to $815 million Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million 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 accordance with 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 CHF: Confoederatio Helvetica Francs (Swiss francs) cUHDRS: Composite Unified Huntington's Disease Rating Scale DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: US Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease mFARS: Modified Friedreich's Ataxia Rating Scale NDA: New Drug Application NfL: Neurofilament light chain 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 live webcast, please visit the "Events & Presentations" page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Facebook and Instagram. For more information please contact: Investors: Ellen Cavaleri +1 (615) 618-8228 [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 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total revenue guidance and (ii) 2026 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, meetings with regulatory agencies, 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," "aim," 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; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the US on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, 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 vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; 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 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 Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. 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. 21% more press release views with Request a Demo

Financial StatementPhase 3License out/inNDA

100 Deals associated with Volanesorsen

External Link

KEGG	Wiki	ATC	Drug Bank
D11648	Volanesorsen	J05AB	DB15067

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Familial chylomicronaemia syndrome	European Union	Akcea Therapeutics Ireland Ltd.	03 May 2019
Familial chylomicronaemia syndrome	Iceland	Akcea Therapeutics Ireland Ltd.	03 May 2019
Familial chylomicronaemia syndrome	Liechtenstein	Akcea Therapeutics Ireland Ltd.	03 May 2019
Familial chylomicronaemia syndrome	Norway	Akcea Therapeutics Ireland Ltd.	03 May 2019

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Lipodystrophy, Familial Partial	Phase 3	United States	Ionis Pharmaceuticals, Inc.	28 Dec 2015
Lipodystrophy, Familial Partial	Phase 3	Belgium	Ionis Pharmaceuticals, Inc.	28 Dec 2015
Lipodystrophy, Familial Partial	Phase 3	Brazil	Ionis Pharmaceuticals, Inc.	28 Dec 2015
Lipodystrophy, Familial Partial	Phase 3	Canada	Ionis Pharmaceuticals, Inc.	28 Dec 2015
Lipodystrophy, Familial Partial	Phase 3	Germany	Ionis Pharmaceuticals, Inc.	28 Dec 2015
Lipodystrophy, Familial Partial	Phase 3	Netherlands	Ionis Pharmaceuticals, Inc.	28 Dec 2015
Lipodystrophy, Familial Partial	Phase 3	Russia	Ionis Pharmaceuticals, Inc.	28 Dec 2015
Hyperlipoproteinemia Type I	Phase 3	United States	Ionis Pharmaceuticals, Inc.	23 Dec 2015
Hyperlipoproteinemia Type I	Phase 3	Brazil	Ionis Pharmaceuticals, Inc.	23 Dec 2015
Hyperlipoproteinemia Type I	Phase 3	Canada	Ionis Pharmaceuticals, Inc.	23 Dec 2015

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02300233 \| NCT02211209 \| NCT02527343 (COMPASS \| APPROACH \| BROADEN, Pubmed \| J Clin Lipidol)	Phase 2/3	Hypertriglyceridemia	-	Volanesorsen	tvbsmprgau(sowhrpdmrz) = itmhbnpnok jtegjprmbd (yiufretkqa, (-5.60 - -0.60))	Positive	01 May 2023
				Placebo	-
NCT02639286 (Pubmed \| J Clin Lipidol)	Not Applicable	Lipodystrophy	5	Volanesorsen 300 mg	slywiariek(stkoknfvxp) = pozhsecope zvdahdezvk (tiitpybblm ) View more	-	22 Sep 2022
				Placebo	slywiariek(stkoknfvxp) = pjzthksvbk zvdahdezvk (tiitpybblm ) View more
NCT02211209 (APPROACH, CTgov)	Phase 3	Familial chylomicronaemia syndrome	67	Placebo (Placebo)	tvjztvdimx(mlmoijywzd) = zunkqbdtbv vwcllujvya (vavyzmutud, gdnkflphyx - qqzydnapvc) View more	-	13 Apr 2022
				Volanesorsen (Volanesorsen)	tvjztvdimx(mlmoijywzd) = mwhlpxtqdz vwcllujvya (vavyzmutud, jjjqsjezlz - kmrcjunfys) View more
NCT02300233 (COMPASS, CTgov)	Phase 3	Hypertriglyceridemia	114	Placebo (Placebo)	toipflbsdr(gmqfqjjswn) = yzgftrcqhj rcdkjevijo (hucnenhcno, lcckralcka - vgnrqtdojh) View more	-	13 Apr 2022
				Volanesorsen (Volanesorsen 300 mg Weekly)	rurrtjynmu(ewjloosern) = tvmijiysqy isqnwrngog (saadtltdjx, 4.89) View more
NCT02527343 (BROADEN, CTgov)	Phase 2/3	Lipodystrophy, Familial Partial	40	placebo+volanesorsen (Randomized Treatment Period: Placebo)	aadlgifpap(fvwwmknbxi) = xdwkyysrec bwzrjzitlv (weviibfotv, tneqyumabe - hpnuvkglyu) View more	-	18 Oct 2021
				volanesorsen (Randomized Treatment Period: Volanesorsen)	aadlgifpap(fvwwmknbxi) = jqfbsdjvni bwzrjzitlv (weviibfotv, tfwqzawjfp - ohygbbbwfe) View more
NCT02658175 (ISIS 304801-CS7, CTgov)	Phase 3	Hyperlipoproteinemia Type I	68	Volanesorsen (Treatment-naïve Group)	ssvhnnalyj(kdfmgnvzil) = boomlbbehm zwnylmuqct (cqtfupxalx, 37.0) View more	-	26 Aug 2021
				Volanesorsen (CS6-Volanesorsen)	ssvhnnalyj(kdfmgnvzil) = lydirwfclo zwnylmuqct (cqtfupxalx, 34.8) View more
NCT02300233 (COMPASS, Pubmed \| Lancet Diabetes Endocrinol) Manual	Phase 3	Familial chylomicronaemia syndrome	114	volanesorsen	fzjuirjygr(cmofipupmi) = bwwnyxydtl akksazhdag (cmoigvohmu, -79.3 to -63.2) View more	Positive	01 May 2021
				Placebo	fzjuirjygr(cmofipupmi) = kwshmudwzz akksazhdag (cmoigvohmu, -13.9 to 12.2) View more
NCT02639286 (CTgov)	Phase 2	Lipodystrophy	5	Placebo (Placebo)	fygujhclmr(ibtyhawxkj) = ozacrtbvht rmclihjdax (mesrsggxny, 0.2) View more	-	20 Oct 2020
				ISIS 304801 (ISIS 304801)	fygujhclmr(ibtyhawxkj) = clqglfcdng rmclihjdax (mesrsggxny, 0.4) View more
NCT02211209 (Pubmed \| Br Dent J \| N Engl J Med) Manual	Phase 3	Familial chylomicronaemia syndrome	66	volanesorsen	khzniqwopq(ddadtjsrvl) = zfjhkacisu avybtepdkr (rnekevutaj ) View more	Positive	08 Aug 2019
				Placebo	khzniqwopq(ddadtjsrvl) = qtnvwohvxh avybtepdkr (rnekevutaj ) View more
NCT02527343 (BROADEN, GlobeNewswire) Manual	Phase 2/3	Lipodystrophy, Familial Partial	40	Volanesorsen	nbwevpmbga(wivfrogbut) = ppzcwvrarn hlcbywzwal (obzgvwzzfd ) View more	Positive	06 Aug 2019
				Placebo	nbwevpmbga(wivfrogbut) = srzafzklem hlcbywzwal (obzgvwzzfd ) View more

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