The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.

Start Date08 Dec 2022

Sponsor / Collaborator

PTC Therapeutics, Inc.

NCT05485987 / CompletedPhase 2

An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age

The primary objective of the study is to assess the pharmacokinetics (PK) and safety of vatiquinone administered in participants with Friedreich ataxia (FA) younger than 7 years.

Start Date13 Oct 2022

Sponsor / Collaborator

PTC Therapeutics, Inc.

NCT05218655 / Enrolling by invitationPhase 3

An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease

The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical study or treatment plan.
The study will continue until vatiquinone becomes commercially available or the program is terminated.

Start Date21 Jun 2022

Sponsor / Collaborator

PTC Therapeutics, Inc.

100 Clinical Results associated with Vatiquinone

100 Translational Medicine associated with Vatiquinone

100 Patents (Medical) associated with Vatiquinone

Literatures (Medical) associated with Vatiquinone

01 May 2023·Xenobiotica; the fate of foreign compounds in biological systems

Absorption, distribution, metabolism and excretion of ¹⁴C-vatiquinone in rats, dogs, and human subjects.

Article

Author: Ye, Qing ; Ma, Jiyuan ; Lee, Lucy ; Yao, Bert ; Thoolen, Martin ; Kong, Ronald ; Klein, Matthew ; Golden, Lee ; Giannousis, Peter

Vatiquinone is a potent inhibitor of 15-lipoxygenase and is in clinical development for the treatment of mitochondrial diseases and other disorders characterised by high levels of oxidative stress and dysregulation of energy metabolism.In rats, ¹⁴C-vatiquinone-derived radioactivity was quickly and widely distributed throughout the body and cleared from most tissues by 24 h post-dose following a single oral dose of ¹⁴C-vatiquinone.Following oral administration, 94% of dose was recovered within seven days in rats, approximately 61% of dose was recovered within seven days in dogs and approximately 93% of dose was recovered within nine days in human subjects (IND 119220). Faecal excretion was the major route (>56% dose) in all species; urinary excretion was minimal in rats and dogs (<3% dose) but was higher in humans (∼ 22% dose).Following oral administration, vatiquinone was the dominant circulating component in rats and dogs but was minor in human subjects. There were no plasma metabolites that were more than 10% of total drug related exposures in all species.Following oral administration, vatiquinone was not detectable in urine but was the most prominent component in faeces in rats, dogs, and humans.

01 Mar 2023·Clinical pharmacology in drug development

Clinical Drug‐Drug Interaction Between Vatiquinone, a 15‐Lipoxygenase Inhibitor, and Rosuvastatin, a Breast Cancer Resistance Protein Substrate

Article

Author: Ma, Jiyuan ; Thoolen, Martin ; Lee, Lucy ; Murase, Katsuyuki

Abstract:

Vatiquinone is a small‐molecule inhibitor of 15‐lipoxygenase in phase 3 development for patients with mitochondrial disease and Friedreich ataxia. The objective of this analysis was to determine the effect of vatiquinone on the pharmacokinetic profile of rosuvastatin, a breast cancer resistance protein substrate. In vitro investigations demonstrated potential inhibition of BCRP by vatiquinone (half maximal inhibitory concentration, 3.8 µM). An open‐label, fixed‐sequence drug‐drug interaction study in healthy volunteers was conducted to determine the clinical relevance of this finding. Subjects received a single dose of 20‐mg rosuvastatin followed by a 7‐day washout. On days 8 through 14, subjects received 400 mg of vatiquinone 3 times daily. On day 12, subjects concomitantly received a single dose of 20‐mg rosuvastatin. The geometric mean ratio for maximum plasma concentration was 77.8%; however, the rosuvastatin disposition phase appeared unaffected. The geometric mean ratios for the area under the plasma concentration–time curve from time 0 to time t and from time 0 to infinity were 103.2% and 99.9%, respectively. Mean rosuvastatin apparent elimination half‐life was similar between treatment groups. These results demonstrate that vatiquinone has no clinically relevant effect on the pharmacokinetics of rosuvastatin.

01 Nov 2022·European journal of clinical pharmacology

Evaluation of vatiquinone drug-drug interaction potential in vitro and in a phase 1 clinical study with tolbutamide, a CYP2C9 substrate, and omeprazole, a CYP2C19 substrate, in healthy subjects

Article

Author: Barrett, Rosemary ; Ma, Jiyuan ; Lee, Lucy ; Thoolen, Martin ; Murase, Katsuyuki

PURPOSE:

In this study, the drug-drug interaction potential of vatiquinone with cytochrome P450 (CYP) substrates was investigated in both in vitro and clinical studies.

METHODS:

The inhibitory potential of vatiquinone on the activity of CYPs 1A2, 2B6, 2C8, 2C9, 2C19, 2D6, and 3A4/5 was assessed in vitro. In an open-label, drug-drug interaction study in 18 healthy human subjects, a single oral dose of 500 mg tolbutamide and 40 mg omeprazole was administered on day 1, followed by a washout of 7 days. Multiple oral doses of 400 mg vatiquinone (three times a day [TID]) were administered from day 8 to day 13 with coadministration of a single oral dose of 500 mg tolbutamide and 40 mg omeprazole on day 12.

RESULTS:

In vitro, vatiquinone inhibited CYP2C9 (IC₅₀ = 3.7 µM) and CYP2C19 (IC₅₀ = 5.4 µM). In the clinical study, coadministration of vatiquinone did not affect the pharmacokinetic (PK) profile of tolbutamide and omeprazole. The 90% confidence intervals (CIs) of geometric least-square mean ratios for maximum plasma concentration (C_max), areas under the plasma concentration-time curve (AUC_0-t), and AUC₀_-inf of tolbutamide and omeprazole were entirely contained within the 80 to 125% no effect limit, except a minor excursion observed for C_max of omeprazole (geometric mean ratio [GMR], 94.09; 90% CI, 78.70-112.50). Vatiquinone was generally well tolerated, and no clinically significant findings were reported.

CONCLUSION:

The in vitro and clinical studies demonstrated vatiquinone has a low potential to affect the pharmacokinetics of concomitantly administered medications that are metabolized by CYP enzymes.

News (Medical) associated with Vatiquinone

25 Apr 2024

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports First Quarter 2024 Financial Results

– Strong revenue performance across product portfolio – – Submitted MAA for sepiapterin and BLA for Upstaza – – On target to achieve remaining 2024 clinical and regulatory milestones, including global submissions for sepiapterin – WARREN, N.J., April 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ending March 31, 2024. "We are off to a strong start in 2024, with outstanding commercial performance and achievement of all planned clinical and regulatory milestones for the first quarter," said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "We remain on track to achieve the many planned 2024 clinical and regulatory milestones, including global regulatory submissions for sepiapterin." Key Corporate Updates: First quarter 2024 total revenue of $210 million First quarter 2024 revenue for the DMD franchise was $161 million Translarna™ (ataluren) net product revenue was $104 million, driven by new patients in existing geographies and continued geographic expansion. Emflaza® (deflazacort) net product revenue was $57 million, resulting from new patient starts and high compliance. Key Clinical and Regulatory Milestones: PTC submitted an MAA to the EMA for sepiapterin for the treatment of PKU in March 2024. The company expects to submit an NDA to the FDA for sepiapterin no later than the third quarter of 2024 and to complete regulatory submissions in Japan and Brazil in 2024. PTC submitted a BLA to the FDA for Upstaza™ for the treatment of AADC deficiency in March 2024. Based on FDA feedback, PTC plans to resubmit the NDA for Translarna for the treatment of nmDMD in mid-2024. Based on FDA feedback, PTC plans to submit an NDA for vatiquinone for the treatment of FA in late 2024. The interim data update for the PIVOT-HD trial of PTC518 for HD patients remains on schedule for the second quarter of 2024. This update will include 12-month data on the initial group of subjects for whom data were reported in June 2023, as well as 12-week data on a larger number of stage 2 and stage 3 HD patients. PTC expects to report topline data for the CardinALS trial of utreloxastat for ALS in the fourth quarter of 2024. First Quarter 2024 Financial Highlights: Total revenues were $210.1 million for the first quarter of 2024, compared to $220.4 million for the first quarter of 2023. Total revenue includes net product revenue across the commercial portfolio of $177.6 million for the first quarter of 2024, compared to $187.6 million for the first quarter of 2023. Total revenue also includes collaboration, royalty, and manufacturing revenue of $32.5 million in the first quarter of 2024, compared to $32.8 million for the first quarter of 2023. Translarna net product revenues were $103.6 million for the first quarter of 2024, compared to $115.1 million for the first quarter of 2023, driven by new patients in existing geographies and continued geographic expansion. The decrease was due to the timing of bulk patient orders. Emflaza net product revenues were $57.5 million for the first quarter of 2024, compared to $54.6 million for the first quarter of 2023. These results were driven by new patient starts and high compliance. Roche reported Evrysdi® 2024 year-to-date sales of approximately $400 million, resulting in royalty revenue of $31.2 million to PTC for the first quarter of 2024, as compared to $30.8 million for the first quarter of 2023. Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $116.1 million for the first quarter of 2024, compared to $195.1 million for the first quarter of 2023. The decrease in research and development expenses reflects strategic portfolio prioritization as the Company continues to focus its resources on its differentiated, high-potential R&D programs. Non-GAAP R&D expenses were $107.2 million for the first quarter of 2024, excluding $9.0 million in non-cash, stock-based compensation expense, compared to $179.8 million for the first quarter of 2023, excluding $15.3 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $73.3 million for the first quarter of 2024, compared to $86.9 million for the first 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 $63.9 million for the first quarter of 2024, excluding $9.4 million in non-cash, stock-based compensation expense, compared to $73.4 million for the first quarter of 2023, excluding $13.5 million in non-cash, stock-based compensation expense. Change in the fair value of deferred and contingent consideration was a gain of $0.1 million for the first quarter of 2024, compared to a loss of $2.4 million for the first quarter of 2023. The net loss was $91.6 million for the first quarter of 2024, compared to a net loss of $139.0 million for the first quarter of 2023. Cash, cash equivalents, and marketable securities was $884.8 million on March 31, 2024, compared to $876.7 million on December 31, 2023. Shares issued and outstanding as of March 31, 2024, were 76,653,960. PTC Reaffirms Full Year 2024 Financial Guidance: PTC anticipates total revenues for full year 2024 to be between $600 million and $680 million. PTC anticipates GAAP R&D and SG&A expense for full year 2024 to be between $740 and $835 million. PTC anticipates Non-GAAP R&D and SG&A expense for full year 2024 to be between $660 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 expense. 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 EMA: European Medicines Agency FA: Friedreich Ataxia FDA: U.S. Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's Disease MAA: Marketing Authorization Application 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 patients 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, on Twitter at @PTCBio, and on LinkedIn. For More Information: Investors: Kylie O'Keefe +1 (908) 300-0691 [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 Reaffirms Full Year 2024 Revenue 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; 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 our 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 the commercialization of Evrysdi under our 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; the potential financial impact and benefits of PTC's leased biologics manufacturing facility; PTC's ability to satisfy its obligations under the terms of its lease agreements, including for its leased biologics manufacturing facility; 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 Quarterly Report on Form 10-Q and 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 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.

Financial StatementNDA

01 Mar 2024

·www.fiercebiotech.com

After pair of failures, PTC finds a path through FDA jungle for Friedreich ataxia med

After the FA failure, announced in May 2023, PTC cut staff and conducted a pipeline prioritization. Then in June, vatiquinone failed again in MDAS, causing PTC to move on from the indication. PTC Therapeutics has managed to slash through the regulatory jungle to find a path forward for Friedreich ataxia med vatiquinone after a pair of failures last year left the biotech reeling. In a fourth-quarter earnings update Thursday afternoon, PTC executives briefed investors on the results of a type C meeting with the FDA where they pled their case for using a subscale called upright stability to show efficacy using data already collected in the phase 3 MOVE-FA trial. FA is a rare degenerative disease that causes damage to the spinal cord, peripheral nerves and cerebellum region of the brain. The condition develops in children and teens and gets worse over time, leading to awkward movements and a loss of sensation. PTC’s candidate for the disorder failed to move the needle on the modified Friedreich Ataxia Rating Scale (mFARS), which measures disease progression. The therapy had little impact on the lower and upper limbs, which led to the failure. Despite the primary endpoint miss, PTC saw a glimmer of efficacy in the data, pointing to the upright stability subscales. That’s what executives took to the FDA meeting earlier this year, arguing that this data could be used for a new drug application. CEO Matthew Klein, M.D., explained that since the MOVE-FA study started, there has been an advancement in the understanding of FA and the importance of the upright stability on an ambulant adolescent population. Even an FDA-funded study has shown that, so PTC took the agency’s own evidence to the meetings, he noted. “We demonstrated significant benefits on the only thing you possibly could show significant benefit on disease progression, and that's the upright stability scale,” Klein said. Discussions are continuing on the NDA, as well as whether the FDA will accept the application for a regular approval or on an accelerated basis. But after a bleak year and the double failures of vatiquinone in FA as well as a rare seizure disorder called mitochondrial disease-associated seizures (MDAS), PTC now has a path forward. “What we're most excited about, obviously, is being able to have the potential to submit an NDA based on the MOVE-FA study and what is still a significant unmet need for pediatric and adolescent Friedreich ataxia patients,” Klein said. PTC is expecting to file the NDA for vatiquinone in late 2024. Klein said an open-label trial is currently ongoing, which will be used to support the package and could also provide confirmatory evidence if the FDA agrees to the accelerated review. For that type of approval, companies can get an advanced go-ahead to market a drug based on biomarker data but must later provide confirmatory evidence to show benefit. After the FA failure, announced in May 2023, PTC cut staff and conducted a pipeline prioritization. Then in June, vatiquinone failed again in MDAS, causing PTC to move on from the indication. Beyond vatiquinone, PTC has a number of approved products and is planning to file an NDA for sepiapterin in phenylketonuria by the end of the third quarter this year. Enrollment is also underway for the phase 2 PIVOT-HD trial of PTC518 in Huntington's disease. Twelve-month interim data from that trial is expected in the second quarter.

NDAPhase 2Phase 3

08 Jan 2024

·www.biospace.com

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 42nd Annual J.P. Morgan Healthcare Conference

– Unaudited 2023 total revenue of $946 million, representing 35% year-over-year growth – – Regulatory filings in the EU and US for sepiapterin in PKU, a potential $1 billion global commercial opportunity, remain on track for 2024 – – Multiple study readouts planned for 2024, including 12-month interim data from the PIVOT-HD study of PTC518 in HD patients – SOUTH PLAINFIELD, N.J., Jan. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update on its commercial progress and R&D pipeline at the 42nd Annual J.P. Morgan Healthcare Conference today, Monday, January 8, at 10:30am EST/7:30am PST. Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2023 accomplishments and highlight potential 2024 value-creating milestones. Preliminary 2023 unaudited financial results and 2024 financial guidance will also be provided. The presentation is being webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at . "2023 was a transformational year for PTC as we made necessary and important changes to become a leaner, more focused and financially stronger company positioned for future success," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "We look forward to a successful 2024, with anticipated key global regulatory submissions for our PKU program and multiple data readouts, including from our PTC518 HD program." Key 2023 Corporate Highlights: Unaudited net product revenue of $661 million in 2023 representing 23% year-over-year growth. Strong year-over-year growth for the Duchenne muscular dystrophy (DMD) franchise, with unaudited net product revenue of $355 million for Translarna™ (ataluren) and $255 million for Emflaza® (deflazacort) in 2023. Translarna growth was driven by new patients in existing geographies and continued geographic expansion. Emflaza growth was due to continued new prescriptions, high compliance, and more favorable access. PTC announced positive readouts from key programs in its clinical pipeline in 2023: Highly statistically significant and clinically meaningful results in the Phase 3 registration-directed APHENITY clinical trial of sepiapterin in adult and pediatric patients with PKU. Achievement of all key objectives of the readout of 12-week interim data in the PIVOT-HD study of PTC518 in HD. PTC announced strategic portfolio prioritizations, which resulted in reductions in both operating expenses and headcount of approximately 25% and 30%, respectively. In October, PTC finalized a royalty agreement, in which Royalty Pharma acquired additional royalties of Evrysdi for $1.0 billion upfront. The agreement included options for PTC to sell the remainder of its royalties of Evrysdi for up to $500 million or for Royalty Pharma to acquire half of such retained royalties for up to $250 million at a later date, less royalties received by PTC. PTC maintains all economics associated with up to $250 million in the remaining commercial sales milestones associated with Evrysdi global net sales. The proceeds from the financing were used to retire all outstanding debt obligations with Blackstone Life Sciences. 2024 Potential Key Clinical and Regulatory Events: Submission of a MAA to the EMA for sepiapterin for the treatment of PKU is expected in the first quarter. Submission of an NDA to the FDA for sepiapterin for the treatment of PKU is expected no later than the third quarter. Results from 12-month interim data from the PIVOT-HD trial of PTC518 in HD patients are expected in the second quarter. Submission of a BLA to the FDA for Upstaza for the treatment of AADC deficiency is expected in the first quarter. Topline results from the CardinALS trial of utreloxastat in ALS are expected in the fourth quarter. PTC expects the CHMP opinion from the re-examination procedure of the negative opinion on the Translarna conditional marketing authorization renewal in late January 2024, with ratification of that opinion by the European Commission 67 days later. FDA meeting for Translarna to align on the specific contents of a potential NDA resubmission is scheduled for the first quarter. FDA meeting for vatiquinone to discuss how the MOVE-FA data along with additional clinical and preclinical data could support an NDA submission in FA is scheduled for the first quarter. Scientific advice feedback from the EMA on a potential submission of vatiquinone for conditional marketing authorization for Friedreich ataxia is expected in the first quarter. Unaudited 2023 Financial Results: Total unaudited net revenue for full-year 2023 was approximately $946 million. Total unaudited net product revenue for full-year 2023 was approximately $661 million. DMD franchise unaudited revenue for full-year 2023 was approximately $610 million, including unaudited net product revenue for Translarna of approximately $355 million and for Emflaza of approximately $255 million. PTC expects to report approximately $278 million for full-year 2023 collaboration and royalty revenue associated with Evrysdi. PTC is currently in the process of finalizing its financial results for the 2023 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2023, subject to the completion of PTC's financial closing procedures. Evrysdi royalty revenue estimates are based on internal estimates. 2024 Financial Guidance: PTC anticipates total revenues for the full-year 2024 to be between $600 million and $850 million. PTC anticipates GAAP R&D and SG&A expense for the full-year 2024 to be between $740 and $835 million. PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2024 to be between $660 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 the 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 expense. 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 ALS: Amyotrophic Lateral Sclerosis BLA: Biologics License Application CHMP: Committee for Medicinal Products for Human Use DMD: Duchenne Muscular Dystrophy EMA: European Medicines Agency FDA: Food and Drug Administration MAA: Marketing Authorization Application NDA: New Drug Application PKU: Phenylketonuria R&D: Research and Development SG&A: Selling, General and Administrative SMA: Spinal Muscular Atrophy About PTC Therapeutics, Inc. PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients 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. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at and follow us on Instagram, Facebook, Twitter, and LinkedIn. For More Information: Investors Kylie O'Keefe +1 (908) 300-0691 kokeefe@ptcbio.com Media Jeanine Clemente +1 (908) 912-9406 jclemente@ptcbio.com 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 "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 Medicines Agency (EMA) determines in the re-examination process that the benefit-risk balance of Translarna authorization supports renewal of such authorization; 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, which was a specific obligation to continued marketing authorization in the EEA, to support a renewal of the conditional marketing authorization for Translarna for the treatment of nmDMD in the EEA; PTC's ability to utilize results from Study 041 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 our 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 the commercialization of Evrysdi under our 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; the potential financial impact and benefits of PTC's leased biologics manufacturing facility; PTC's ability to satisfy its obligations under the terms of its lease agreements, including for its leased biologics manufacturing facility; 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 Quarterly Report on Form 10-Q and 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 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. Company Codes: NASDAQ-NMS:PTCT

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Friedreich Ataxia	Phase 3	US	PTC Therapeutics, Inc.	08 Dec 2022
Friedreich Ataxia	Phase 3	AU	PTC Therapeutics, Inc.	08 Dec 2022
Friedreich Ataxia	Phase 3	BR	PTC Therapeutics, Inc.	08 Dec 2022
Friedreich Ataxia	Phase 3	CA	PTC Therapeutics, Inc.	08 Dec 2022
Friedreich Ataxia	Phase 3	FR	PTC Therapeutics, Inc.	08 Dec 2022
Friedreich Ataxia	Phase 3	DE	PTC Therapeutics, Inc.	08 Dec 2022
Friedreich Ataxia	Phase 3	IT	PTC Therapeutics, Inc.	08 Dec 2022
Friedreich Ataxia	Phase 3	NZ	PTC Therapeutics, Inc.	08 Dec 2022
Friedreich Ataxia	Phase 3	ES	PTC Therapeutics, Inc.	08 Dec 2022
Mitochondrial Diseases	Phase 3	JP	PTC Therapeutics, Inc.	21 Jun 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01642056 (CTgov)	Phase 1/2	Mitochondrial Diseases \| Muscular Diseases	20	Placebo+EPI-743 (EPI-743, Then Placebo)	svzopqtrkc(knbivfxscy) = ndssnozkdm fajjsgivgq (lgvmfddxcp, ghuudvkqqj - cdedsdclzq) View more	-	14 Apr 2021
				Placebo+EPI-743 (Placebo, Then EPI-743)	svzopqtrkc(knbivfxscy) = rvpghlfnjm fajjsgivgq (lgvmfddxcp, yujlsuypdq - ylshkolsjh) View more
EPI-743 (ARVO2016)	Not Applicable	Optic Atrophy, Hereditary, Leber	6	EPI-743 (alpha-tocotrienol quinone)	vmhtzbyzlm(izlgxlizip) = HVF improved (>5 dB) in one chronic and both acute patients, while three chronic patients remained stable. HVF remained stable after the withdrawal of therapy in those patients who chose to withdraw from the study. ojvwvzyxes (qrjvnvyznj ) View more	Positive	01 May 2016
AAN2016	Phase 2	-	10	EPI-743 200 mg TID	iopipvsmcb(nfbnsmjouf) = otwhrcchem ynlltrcwjy (nmddiqshxa )	Positive	05 Apr 2016
				EPI-743 400 mg TID	iopipvsmcb(nfbnsmjouf) = kfazpaugrd ynlltrcwjy (nmddiqshxa )
P5.388 (AAN2016)	Not Applicable	Friedreich Ataxia	3	EPI-743 400mg PO TID	uoihqpsokj(suqpbczbzx) = No serious adverse events, including hematologic or cardiac, were observed dqiyxatmcm (lmylwsmxri )	Positive	05 Apr 2016
I1.012 (AAN2016)	Phase 2	glutamine/glutamate levels	10	EPI-743 200 mg TID	wbwwpfvzll(oorrrlbfnr) = ilfhtkodyr ikzajwyehu (uziypazkye )	Positive	05 Apr 2016
				EPI-743 400 mg TID	wbwwpfvzll(oorrrlbfnr) = vocyfclboq ikzajwyehu (uziypazkye )
EPI-743 (ARVO2013)	Not Applicable	Optic Atrophy, Hereditary, Leber Neuron specific enolase (NSE)	12	LHON patients receiving EPI-743	tbbqcctjka(ukqkbbmpxb) = fvmnesnnow xladvsffzk (hvpudmbzrr, 6.21 - 12.64)	Positive	01 Jun 2013
				EPI-743	tbbqcctjka(ukqkbbmpxb) = rfproavbyy xladvsffzk (hvpudmbzrr )

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