A Phase 3, Randomized, Crossover, Open-Label, Active-Controlled Study of Sepiapterin versus Sapropterin in Participants With Phenylketonuria ≥2 years of Age

Start Date10 May 2024

Sponsor / Collaborator

PTC Therapeutics, Inc.

NCT06302348

/ RecruitingPhase 3

A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin

The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.

Start Date04 Mar 2024

Sponsor / Collaborator

PTC Therapeutics, Inc.

100 Clinical Results associated with Sepiapterin

100 Translational Medicine associated with Sepiapterin

100 Patents (Medical) associated with Sepiapterin

463

Literatures (Medical) associated with Sepiapterin

01 May 2026·METABOLISM-CLINICAL AND EXPERIMENTAL

Efficacy and safety of sepiapterin versus sapropterin in patients with phenylketonuria: Results from the Phase 3, randomized, crossover, open-label, active-controlled AMPLIPHY trial

Article

Author: Peters, Heidi ; van Spronsen, Francjan ; Hughes, Catalina ; Bélanger-Quintana, Amaya ; Potter, Murray ; Ingalls, Kim ; Maillot, François ; Smith, Neil ; Tchan, Michel ; Burlina, Alberto ; Inwood, Anita ; Tyčová, Renáta ; Grošelj, Urh ; Vijay, Suresh ; Chakrapani, Anupam ; Rutsch, Frank ; Venegas-Moreno, Eva Maria ; Guilder, Laura ; Giżewska, Maria ; Fjellbirkeland, Olivia ; Zhao, Zhenming ; Muntau, Ania C ; Arnoux, Jean-Baptiste

AIM:

AMPLIPHY is the first Phase 3 study comparing sepiapterin versus sapropterin in children and adults with phenylketonuria (PKU).

METHODS:

AMPLIPHY was an international, Phase 3, two-part, open-label study in participants with PKU aged ≥2 years. Participants responsive to sepiapterin (60 mg/kg/day) in Part 1 (≥20% reduction in blood phenylalanine [Phe]) entered Part 2, a crossover treatment period, and were randomized 1:1 to alternative treatment sequences of sepiapterin (60 mg/kg/day, licensed dosage) and sapropterin (20 mg/kg/day, maximum licensed dosage) for 4 weeks each, with a 14-day washout between treatments. The primary endpoint was mean change in blood Phe from baseline to Weeks 3-4 of each treatment period (Part 2).

RESULTS:

Of 82 participants enrolled, 67 (81.7%) and 62 (75.6%) had reductions in blood Phe ≥20% and ≥30%, respectively, in Part 1. Sixty-two participants were randomized in Part 2 (mean [SD] age, 15.8 [10.8] years). In the primary analysis set (≥30% reduction in blood Phe in Part 1, n = 58), mean (SD) baseline blood Phe before sepiapterin and sapropterin treatment was 725.8 (302.1) and 790.4 (370.0) μmol/L, respectively. Least-squares mean (SE) reduction in blood Phe from baseline was -437.0 (28.0) and -256.6 (28.2) μmol/L, respectively, representing a least-squares mean difference of -180.4 μmol/L (95% CI: -229.5, -131.4; p < 0.0001) and a relative 70% greater reduction with sepiapterin versus sapropterin. Both treatments were well tolerated, with safety profiles consistent with previous reports.

CONCLUSIONS:

Sepiapterin was superior to the highest approved dose of sapropterin in lowering blood Phe. No new safety signals were observed. The trial was registered in the UK Clinical Study Registry, ISRCTN, on January 29, 2024 (ID number, ISRCTN79102999; https://www.isrctn.com/ISRCTN79102999).

01 Apr 2026·GENETICS IN MEDICINE

Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study

Article

Author: Thomas, Janet A ; Vijay, Suresh ; Giżewska, Maria ; Fjellbirkeland, Olivia ; Gao, Lan ; Lund, Allan ; Mungan, Halise ; Guimas, Arlindo ; Ingalls, Kimberly ; Sacharow, Stephanie ; Burlina, Alberto ; Canda, Ebru ; Peters, Heidi ; Rutsch, Frank ; Opladen, Thomas ; Minami, Maria ; Hughes, Catalina ; Plana, Jaume Campistol ; Oliveira, Anabela ; Hamazaki, Takashi ; Margvelashvili, Lali ; van Spronsen, Francjan ; Vockley, Jerry ; Smith, Neil ; Tchan, Michel ; Muntau, Ania C ; Manti, Filippo ; Bélanger-Quintana, Amaya ; Bratkovic, Drago ; Chakrapani, Anupam ; Kiykim, Ertuğrul ; Inwood, Anita ; Liu, Emelline ; Longo, Nicola ; Larkin, Alex ; Yıldız, Yılmaz ; Ito, Tetsuya ; MacDonald, Anita ; Lah, Melissa ; Ezgü, Fatih ; Schwartz, Ida Vanessa D ; Janeiro, Patricia ; Zori, Roberto ; Ishige, Mika ; Agladze, Dodo ; Guerra, Ixiu-Cabrales ; Breilyn, Margo Sheck ; Guilder, Laura ; Khan, Aneal

PURPOSE:

To report interim results from the ongoing, open-label, phase 3 APHENITY Extension Study (NCT05166161), evaluating long-term treatment with sepiapterin in patients with phenylketonuria.

METHODS:

Participants received an age-based dose of oral sepiapterin daily; those with mean blood phenylalanine (Phe) levels <360 μmol/L (<5.95 mg/dL) after 2 weeks underwent a 26-week dietary Phe tolerance assessment, wherein dietary Phe intake was adjusted and blood Phe levels monitored. Other participants continued treatment with optional diet liberalization. Primary endpoints included change from baseline to week 26 in dietary Phe intake and treatment-emergent adverse events (TEAEs).

RESULTS:

As of September 2, 2024, 169 participants received sepiapterin (median [minimum, maximum] age: 14.0 [0.2, 55.0] years, median exposure: 72.9 weeks); 102 participants underwent dietary Phe tolerance assessments. Mean (SD) dietary Phe intake increased from 27.6 (18.0) mg/kg/day at baseline to 62.5 (41.5) mg/kg/day at week 26 (least-squares mean change [SE]: 36.4 [2.8] mg/kg/day from baseline) (P < .0001 from post hoc analysis). The incidence of treatment-related TEAEs was 29.0%; 3 participants (1.8%) discontinued treatment owing to treatment-related TEAEs. There were no treatment-related serious TEAEs or deaths.

CONCLUSION:

Interim results support the long-term safety of sepiapterin and demonstrate the potential for diet liberalization in adults and children with phenylketonuria.

CLINICALTRIALS:

gov/study/NCT05166161; date of registration, December 8, 2021).

GOV IDENTIFIER:

NCT05166161 (https://www.

01 Mar 2026·Integrative Zoology

Exploring Dietary Composition in an Invasive Apple Snail from Different Habitats Combining with Intestinal Microbiota and Metabolomics

Article

Author: Li, Xuexia ; Chen, Lian ; Li, Shuxian ; He, Xinni ; Wang, Yue ; Xue, Shaoshuai ; Qian, Zijin ; Li, Hong ; Shen, Wenjia ; Jing, Muzi

ABSTRACT:

Pomacea canaliculata is recognized as a globally invasive aquatic species. Analyses of intestinal microbiota, dietary composition, and metabolism of invasive species can enhance our understanding of their feeding strategies and physiological adaptation strategies to the environment. Intestinal content samples were collected from P. canaliculata inhabiting three distinct environments including a pond, a river, and a ditch. These samples were subjected to 16S rRNA gene sequencing analysis and multiple metabarcoding analyses, including eukaryotic 18S rRNA, mitochondrial cytochrome c oxidase I (COI), and chloroplast rbcL genes. In addition, metabolomics analysis was conducted on the intestinal content samples to investigate metabolic change. The highest dietary diversity in P. canaliculata was observed in the ditch, and females exhibited a higher dietary diversity than males in the pond. The 18S rRNA gene has a high potential for identifying the dietary components of omnivorous species. The intestinal microbiota of P. canaliculata from different habitats displayed significant variations, attributed to differences in food resources and other environmental factors. Bacteria in the aquatic environment had minimal impact on the intestinal microbiota of P. canaliculata . Overall, P. canaliculata exhibited adaptive changes in physiological characteristics across different habitats, including alterations in diet, which, in turn, influence microbiota and metabolic pathways such as amino acid biosynthesis in the intestine. The present study investigated the physiological mechanisms that enable P. canaliculata to adapt to diverse habitats, considering various factors including diet, which is important for comprehending its invasive potential and the subsequent threats it poses to aquatic ecosystems.

News (Medical) associated with Sepiapterin

19 Feb 2026

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2025 Financial Results

– Full-year 2025 product and royalty revenue of $831M, exceeding guidance – – Strong Sephience™ (sepiapterin) uptake since 2H 2025 launch with fourth quarter and 2025 revenue of $92M and $111M, respectively – – Cash of $1.95B as of December 31, 2025 – WARREN, N.J., Feb. 19, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2025. "We delivered another strong quarter and finish to 2025, building on the successful global launch of Sephience," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "With our robust commercial engine, innovative R&D programs, and strong financial position, we look forward to continued success as we approach cash flow breakeven." Key Corporate Highlights Full-year 2025 product and royalty revenue of $831 million, exceeding guidance Global launch of Sephience off to strong start Q4 2025 revenue of $92 million, including $81 million revenue in the US and $11 million revenue ex-US Sephience total net revenue of $111 million in 2025 since launch 946 total patients on commercial therapy worldwide as of December 31, 2025 1,134 patient start forms received in the US as of December 31, 2025 Approval in Japan in December 2025; approval in Brazil in February 2026 Sephience global footprint expected to increase to 20 to 30 countries by end of 2026 In December 2025, PTC sold the remainder of its Evrysdi® (risdiplam) royalty to Royalty Pharma for $240 million upfront and up to $60 million in sales-based milestones; PTC maintains the right to receive a $150 million milestone from Roche based on single-year Evrysdi sales of $2.5 billion End-of-Phase 2 meeting with FDA held in Q4 2025 to discuss the votoplam Huntington's disease (HD) program Alignment reached on design of global Phase 3 trial, INVEST-HD, which is planned to initiate in 1H 2026 FDA confirmed openness for potential Accelerated Approval pathway given significant unmet need Type C meeting with FDA held in December 2025 to discuss the vatiquinone Friedreich's ataxia program; FDA indicated that an additional study would be necessary to support NDA resubmission and meeting minutes stated that this could be an open-label study with a natural history control group Fourth Quarter and Full Year 2025 Financial Highlights Total revenues were $164.7 million for the fourth quarter of 2025, compared to $213.2 million for the fourth quarter of 2024. Total revenues were $1,730.7 million for full year 2025, compared to $806.8 million for full year 2024. Included in total revenues is collaboration and license revenue of $998.4 million for the full year 2025, related to the votoplam license and collaboration agreement with Novartis, which closed in January 2025. Total net product revenues were $184.0 million for the fourth quarter of 2025, compared to $150.1 million for the fourth quarter of 2024. Total net product revenues were $586.7 million for full year 2025, compared to $582.1 million for full year 2024. Translarna™ (ataluren) net product revenues were $39.0 million for the fourth quarter of 2025, compared to $89.1 million for the fourth quarter of 2024. Translarna net product revenues were $235.3 million for full year 2025, compared to $321.1 million for full year 2024. Emflaza® (deflazacort) net product revenues were $27.1 million for the fourth quarter of 2025, compared to $50.5 million for the fourth quarter of 2024. Emflaza net product revenues were $146.4 million for full year 2025, compared to $207.2 million for full year 2024. Roche reported Evrysdi full year 2025 sales of approximately 1,757 CHF million, resulting in royalty revenue of $244.2 million to PTC for full year 2025, compared to $203.9 million to PTC for full year 2024. Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $133.1 million for the fourth quarter of 2025, compared to $124.8 million for the fourth quarter of 2024. GAAP R&D expenses were $455.2 million for full year 2025, compared to $534.5 million for full year 2024. Non-GAAP R&D expenses were $124.3 million for the fourth quarter of 2025, excluding $8.8 million in non-cash, stock-based compensation expense, compared to $116.0 million for the fourth quarter of 2024, excluding $8.8 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $419.6 million for full year 2025, excluding $35.7 million in non-cash, stock-based compensation expense, compared to $497.9 million for full year 2024, excluding $36.6 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $96.9 million for the fourth quarter of 2025, compared to $84.7 million for the fourth quarter of 2024. GAAP SG&A expenses were $347.1 million for full year 2025, compared to $300.9 million for full year 2024. Non-GAAP SG&A expenses were $87.2 million for the fourth quarter of 2025, excluding $9.7 million in non-cash, stock-based compensation expense, compared to $76.3 million for the fourth quarter of 2024, excluding $8.4 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $308.3 million for full year 2025, excluding $38.9 million in non-cash, stock-based compensation expense, compared to $262.9 million for full year 2024, excluding $38.0 million in non-cash, stock-based compensation expense. Net loss was $135.0 million for the fourth quarter of 2025, compared to net loss of $65.9 million for the fourth quarter of 2024. Net income was $682.6 million for full year 2025, compared to net loss of $363.3 million for full year 2024. Cash, cash equivalents, and marketable securities were $1,945.4 million on December 31, 2025, compared to $1,139.7 million on December 31, 2024. Shares issued and outstanding as of December 31, 2025, were 81,474,366. Full Year 2026 Financial Guidance Total product revenue of $700 to $800 million, representing a 19 to 36% increase from 2025, with the majority from Sephience GAAP R&D and SG&A expense of $775 to $815 million Non-GAAP R&D and SG&A expense of $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) DMD: Duchenne muscular dystrophy FA: Friedreich's 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. The webcast conference call can be accessed on the Investors section of the PTC website at . A replay of the call will be available after completion of the call and will be archived on the company's website. 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, Instagram and Facebook. 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 "Full Year 2026 Financial Guidance", including with respect to (i) 2026 total product 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 U.S. 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/inDrug ApprovalNDA

13 Feb 2026

·endpoints.news

PTC Therapeutics withdraws US filing for troubled Duchenne treatment

PTC Therapeutics has pulled a US application for its Duchenne muscular dystrophy treatment, adding to a long history of regulatory setbacks for the protein restoration therapy. The biotech on Thursday said it withdrew its NDA resubmission for Translarna after the FDA said the data were unlikely to meet its bar for effectiveness. PTC added it was “disappointed that FDA approval cannot be achieved,” but didn’t share what it would do next. TD Cowen analysts wrote the same day that, while the announcement is “unfortunate,” Translarna’s chances of US approval were low anyway, given its history and mixed clinical results. The drug was hit with two refusal-to-file letters from the FDA in 2011 and 2016 and received a complete response letter for its first US filing in 2017. In Europe, the EMA conditionally approved Translarna in 2014, but the Committee for Medicinal Products for Human Use, or CHMP, has since recommended against renewing the authorization on four separate occasions , mostly recently in October 2024. Translarna is designed to trigger functional dystrophin protein in Duchenne patients with a nonsense mutation, according to PTC. Following the conditional approval for Translarna in Europe, PTC conducted two studies to confirm the drug’s efficacy. The first trial failed to achieve that goal, although the results suggested that a subgroup of patients with progressive decline in walking ability benefited more than others. The second study focused on that subgroup, but didn’t hit statistical significance. Translarna is fully approved in Brazil and Russia. Although the drug’s marketing authorization in Europe was revoked last year, it remains available in some European countries. TD Cowen analysts estimate the drug will reach $160 million in sales this year. The bigger focus for PTC is the ongoing launch of Sephience, a treatment for another rare genetic disorder called phenylketonuria (PKU), which secured US approval in July last year.

Drug ApprovalAccelerated ApprovalNDA

22 Dec 2025

·www.prnewswire.com

PTC Therapeutics Announces Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU) in Japan

– Indication includes all ages and the full spectrum of disease severity – – First Japan product approval for PTC – WARREN, N.J., Dec. 22, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that Sephience™ (sepiapterin) has been approved by the Japanese Ministry of Health, Labor and Welfare (MHLW) for the treatment of children and adults living with phenylketonuria (PKU). The label includes individuals of all ages and the full spectrum of disease severity. "The approval of Sephience in Japan is an important milestone for Japanese patients living with PKU as well as for PTC," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "I am proud that we have been able to achieve approvals for Sephience in the EU, US and Japan within less than six months, allowing for a true global launch. Our team in Japan is fully built and excited to bring Sephience to any individual who may benefit." PTC will now engage in pricing discussions which are expected to conclude in Q1 2026, with launch occurring shortly thereafter. About Sephience™ (sepiapterin) Sephience™ is indicated for the treatment of adult and pediatric patients with phenylketonuria (PKU). Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Through its mechanism of action, Sephience is able to effectively reduce blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients. Sephience is approved in the European Union / European Economic Area region, the United States and Japan. About Phenylketonuria Phenylketonuria (PKU) is a rare, inherited metabolic disease, characterized by the body's inability to break down an essential amino acid called phenylalanine (Phe) and which can result in neurological and other symptoms. If left untreated or poorly managed, Phe can build up to harmful levels in the body. This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems. Newborns with PKU initially do not have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of Phe in the first few years of life is irreversible. Diagnosis of PKU usually takes place during newborn screening programs. There are an estimated 58,000 people living with PKU globally. 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. For More Information: Investors: Ellen Cavaleri +1 (615) 618-6228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statement This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including 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; expectations with respect to Sephience, including timing of commercialization; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization and the potential achievement of regulatory and sales milestones and contingent payments that PTC may be obligated to make; 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; 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 Sephience. 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

Drug Approval

100 Deals associated with Sepiapterin

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KEGG	Wiki	ATC	Drug Bank
-	Sepiapterin	-	DB16326

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Phenylketonurias	European Union	PTC Therapeutics, Inc.	24 Jun 2025
Phenylketonurias	Iceland	PTC Therapeutics, Inc.	24 Jun 2025
Phenylketonurias	Liechtenstein	PTC Therapeutics, Inc.	24 Jun 2025
Phenylketonurias	Norway	PTC Therapeutics, Inc.	24 Jun 2025
Hyperphenylalaninemia	European Union	PTC Therapeutics International Ltd.	19 Jun 2025
Hyperphenylalaninemia	Iceland	PTC Therapeutics International Ltd.	19 Jun 2025
Hyperphenylalaninemia	Liechtenstein	PTC Therapeutics International Ltd.	19 Jun 2025
Hyperphenylalaninemia	Norway	PTC Therapeutics International Ltd.	19 Jun 2025

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Diabetic Gastroparesis	Phase 2	United States	PTC Therapeutics, Inc.	27 Feb 2019
Gastroparesis	Phase 2	United States	PTC Therapeutics, Inc.	27 Feb 2019
Tetrahydrobiopterin Deficiency	Phase 2	United States	PTC Therapeutics, Inc.	03 Jan 2019

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05099640 (FDA_CDER) Manual	Phase 3	Hyperphenylalaninemia \| Phenylketonurias	98	SEPHIENCE	gamjokeacf(dspujckasb) = ccsldmsmqi vhmhckbpyf (mqhrogcojg, 24.1) View more	Positive	28 Jul 2025
				Placebo	gamjokeacf(dspujckasb) = uystrjankl vhmhckbpyf (mqhrogcojg, 24.2) View more
NCT05099640 (APHENITY, Literature) Manual	Phase 3	Phenylketonurias	157	Placebo (part 2)	krdzohubwi(iqnumjuoyu) = hwdmmxhhmv khhwfcguic (ttuemhlxir, 29)	Positive	05 Oct 2024
				Placebo	zvvrtotmbs(qgexpvvugb) = cimfgeahmz nktsztddnp (dgxzaibcxd ) View more
NCT05099640 (APHENITY, CTgov)	Phase 3	Phenylketonurias	157	Placebo	xilmwiyrdc(arlyaqwuqc) = ytpckzvcje rwjxeivqzq (tzhclrtlbl, 24.223) View more	-	10 Jan 2024
NCT03519711 (CTgov)	Phase 1/2	Tetrahydrobiopterin Deficiency \| Hyperphenylalaninemia	8	PTC923 (Cohort 1: PTC923 2.5 mg/kg/Day)	pjlqwxuadr = hgdhopsfab smkaskmhkf (riathrefpa, htchsrxtkv - gcxluocqqn) View more	-	14 Nov 2023
				PTC923 (Cohort 1: PTC923 10 mg/kg/Day)	pjlqwxuadr = soqmaqdnxk smkaskmhkf (riathrefpa, bejsmlxhyv - cadzkfhqgc) View more
NCT03712124 (CTgov)	Phase 2	Gastroparesis \| Diabetic Gastroparesis	21	CNSA-001 (CNSA-001)	sdoxospnew(fygfqoocoi) = pgkkijdshr vmiksikwef (ecjbembcvf, 124.52) View more	-	05 Jan 2022
				Placebo (Placebo)	sdoxospnew(fygfqoocoi) = tlcqtrhwfh vmiksikwef (ecjbembcvf, 99.06) View more
NCT03712124 (Pubmed) Manual	Phase 2	Diabetic Gastroparesis	-	PTC923	briwhupzfn(bqabnowxgp): difference = 98 (95% CI, 36 - 161), P-Value = 0.0042	Positive	01 Sep 2021
				Placebo

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