RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Rare Pediatric Disease (United States), Priority Review (United States)

Structure/Sequence

Molecular FormulaC29H44O3

InChIKeyLNOVHERIIMJMDG-XZXLULOTSA-N

CAS Registry1213269-98-7

Clinical Trials associated with Vatiquinone

NCT05515536

/ Active, not recruitingPhase 3

Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia

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 Date12 Oct 2022

Sponsor / Collaborator

PTC Therapeutics, Inc.

JPRN-jRCT2031200224

/ Not yet recruitingPhase 3IIT

Clinical study on PTC743 in Patients with Leigh's Encephalomyelopathy (Continued Dosing Study)

Start Date22 Dec 2020

Sponsor / Collaborator-

100 Clinical Results associated with Vatiquinone

100 Translational Medicine associated with Vatiquinone

100 Patents (Medical) associated with Vatiquinone

Literatures (Medical) associated with Vatiquinone

01 Dec 2025·BIOPHARMACEUTICS & DRUG DISPOSITION

Enhanced Vatiquinone Bioavailability With Fatty Meals and Optimal Dosing Schedule

Article

Author: Golden, Lee ; Thoolen, Martin ; Lee, Lucy ; Kong, Ronald ; Giannousis, Peter ; Murase, Katsuyuki ; Kaushik, Diksha

ABSTRACT:

Vatiquinone, a 15‐lipoxygenase (LO) inhibitor, is an orally bioavailable small molecule being developed for the treatment of Friedreich's ataxia, a disorder characterized by high levels of oxidative stress and dysregulation of energy metabolism. This investigation discusses the results of the food effect and three times a day (TID) dosing schedule studies data. The food effect study showed that absorption is significantly enhanced when vatiquinone was administered with fatty meals, either solid or liquid. Mean vatiquinone area under the concentration‐time curve (AUC) values were 22‐fold and 3‐fold higher in subjects who consumed fatty meals and liquid meals, respectively, compared with subjects who fasted. With fatty meals, the intersubject variabilities appeared lower for AUC. The TID dosing study showed that the typical individual pharmacokinetic concentration profile exhibited 3 peaks on both Day 1 and after multiple dosing on Day 6. Vatiquinone exposures, AUC, and maximum concentration (C max ) appeared to increase from 200 to 400 mg in a dose‐proportional manner with moderate variabilities, ranging from approximately 35% to 65%. The mean Day 6 accumulation ratio (AR AUC ) suggested an accumulation ratio of 1.61 and 1.73 for 200 and 400 mg, respectively. The results of both studies support the recommendation of administering vatiquinone TID with fatty meals taken on a convenient dosing regimen.

01 Feb 2025·JOURNAL OF CLINICAL PHARMACOLOGY

Determination of Vatiquinone Drug‐Drug Interactions, as CYP450 Perpetrator and Victim, Using Physiologically Based Pharmacokinetic (PBPK) Modeling and Simulation

Article

Author: Kong, Ronald ; Lee, Lucy ; Okudaira, Noriko ; Murase, Katsuyuki ; Jones, Hannah M.

Abstract:

Vatiquinone, a 15‐lipoxygenase inhibitor, is in development for patients with Friedreich's ataxia. Physiologically based pharmacokinetic (PBPK) modeling addressed drug‐drug interaction gaps without additional studies. A PBPK model (Simcyp Simulator version 21, full model) was developed using parameters obtained from in vitro studies, in silico estimation and optimization, and two clinical studies. A venous blood dosing model best characterized vatiquinone lymphatic absorption. Apparent oral clearance (CL/F) was used to optimize intrinsic clearance (CLint). Intestinal availability (Fg) was estimated using the hybrid flow term (Qgut), unbound fraction in the enterocytes (fugut), and gut intrinsic metabolic clearance (CLuG,int). Renal clearance (CLR) was set to zero. Assuming an Fa of 1, CYP3A4 contribution (fmCYP3A4) was further optimized. The PBPK model was verified with two clinical studies and demonstrated that it adequately characterized vatiquinone PK. As a perpetrator, the model predicted no risk for vatiquinone to significantly alter the drug exposures of CYP3A4 and CYP1A2 substrates as evident bynegligible reduction in both midazolam and caffeine area under the curve (AUC)inf and Cmax. As a victim, the model predicted that vatiquinone exposures are weakly influenced by moderate CYP3A4 inhibitors and inducers. With fluconazole coadministration, vatiquinone AUCinf and Cmax increased by nearly 50% and 25%, respectively. With efavirenz coadministration, vatiquinone AUCinf and Cmax decreased by approximately 20% and 10%, respectively. Results suggested that vatiquinone does not significantly impact CYP3A4 and CYP1A2 substrates and that moderate CYP3A4 inhibitors and inducers weakly impact vatiquinone AUC.

01 Dec 2024·Clinical Pharmacology in Drug Development

Effect of Itraconazole, a CYP3A4 Inhibitor, and Rifampin, a CYP3A4 Inducer, on the Pharmacokinetics of Vatiquinone

Article

Author: Thoolen, Martin ; Golden, Lee ; Ma, Jiyuan ; Lee, Lucy ; Kong, Ronald ; Kaushik, Diksha

Abstract:

Vatiquinone is a small molecule inhibitor of 15‐lipoxygenase in development for patients with Friedreich's ataxia. The objective of this analysis was to determine the effect of a cytochrome P450 isoform 3A4 (CYP3A4) inhibitor and inducer on vatiquinone pharmacokinetics (PKs). The coadministration of 400 mg of vatiquinone with 200 mg of itraconazole (a CYP3A4 inhibitor) resulted in increased maximum observed concentration (Cmax) of vatiquinone and systemic exposure (AUC0‐inf) by approximately 3.5‐ and 2.9‐fold, respectively. The coadministration of 400 mg of vatiquinone with 600 mg of rifampin (a CYP3A4 inducer) resulted in decreased vatiquinone Cmax and AUC0‐inf by approximately 0.64‐ and 0.54‐fold, respectively. The terminal half‐life of vatiquinone was not affected by itraconazole or rifampin. These clinical study results confirm the in vitro reaction phenotyping data that shows that CYP3A4 plays an important role in vatiquinone metabolism. The result of this analysis together with phase 3 efficacy and safety data, population PK analysis, and the exposure‐response relationship will determine if the extent of vatiquinone changes in the presence of CYP3A4 inhibitors and inducers are considered clinically relevant.

News (Medical) associated with Vatiquinone

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

16 Feb 2026

·www.fiercepharma.com

PTC shuts down FDA approval bid for troubled Duchenne med Translarna

With the writing apparently on the wall, PTC Therapeutics has called off its latest bid for FDA approval of its Duchenne muscular dystrophy (DMD) drug Translarna. Late Thursday, PTC revealed that it withdrew its FDA application for Translarna in nonsense mutation DMD after receiving feedback on its filing from the regulator. "FDA shared that based on its review to date, the data in the [New Drug Application] submission are unlikely to meet the Agency's threshold of substantial evidence of effectiveness to support approval of Translarna," Matthew Klein, M.D., PTC's chief executive, explained in a statement. PTC is "disappointed that FDA approval cannot be achieved," Klein said, citing the decades of development work that have gone into the company's protein restoration therapy. In a letter to the U.S. DMD community, PTC cited unresolvable "differences in data interpretation" with the FDA, Pharmaphorum reports. Translarna has a checkered past with the FDA and, despite scoring a thumbs-up in Europe back in 2014, recently met a major marketing setback there, as well. Stateside, the FDA initially declined to review PTC's Translarna application in 2016, telling the company its application was incomplete. About a year and a half later, the FDA formally rejected the drug, citing the need for PTC to run an additional trial "at a minimum" to better validate Translarna's effectiveness. The FDA also flagged other "nonclinical and [chemistry, manufacturing and controls] matters" in the approval bid. PTC announced it would make another attempt at U.S. approval in early 2024, pointing to "recent feedback" from the FDA. Now, that effort is ending without success. The drug has also suffered recent regulatory setbacks in the EU and, despite some pushback from European officials, the European Medicines Agency's human medicines committee ultimately decided not to renew the drug's conditional marketing authorization in late 2024. The European Commission, which has final say on approvals in the bloc, made the same call last March. European regulators blessed the med with a conditional nod in 2014 on results from a phase 2b study that favored Translarna over placebo on a six-minute walk test after 48 weeks of treatment. But that study and a subsequent trial did not produce statistically significant results on the metric. Back in the U.S., PTC also faced FDA friction around its Friedreich's ataxia candidate vatiquinone last year when the regulator denied approval of the candidate over a lack of "substantial evidence of efficacy" in the company's filing. Translarna, which goes by the generic moniker ataluren, is a protein restoration therapy that is designed to allow the formation of a functioning protein in patients with genetic disorders caused by nonsense mutations. PTC describes that type of mutation as an alteration in the genetic code that prematurely halts the synthesis of an essential protein. In the case of DMD, that protein would be dystrophin. The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultDrug Approval

19 Jan 2026

·www.biospace.com

JPM26: Beam, Cabaletta, More Zero In on Regulatory Alignment After Tumultuous Year

iStock, wildpixel As biotechs faced investors at the J.P. Morgan Healthcare Conference this week, they emphasized agreement with the FDA on clinical trial design and regulatory pathways to approval. Atara, meanwhile, lamented the agency’s “complete reversal of position” after its therapy for a rare surgical complication was rejected. Last year, a clutch of biotechs experienced regulatory whiplash as previous FDA guidance was seemingly reversed, leading to rejections or delayed timelines. This trend continued into 2026, as Atara Biotherapeutics on Monday announced an FDA rejection it described as “a complete reversal of position.” Amid this regulatory uncertainty, several biotechs wanted to make it crystal clear during the J.P. Morgan Healthcare Conference this week that their alignment with the regulator was rock solid. Beam Therapeutics, Cabaletta Bio, PTC Therapeutics and BridgeBio all communicated alignment with the FDA on regulatory pathways or next steps for their programs, according to analyst notes dispersed throughout the week as companies provided updates at JPM26. H.C. Wainwright analysts highlighted regulatory alignment as a key theme throughout the meeting. “Regulatory alignment is compressing timelines and improving visibility to value inflections,” the firm wrote in a Wednesday morning note. Nevertheless, Atara’s Monday news demonstrates that biotech is not out of the woods of FDA uncertainty just yet. In its complete response letter, for the proposed use of Ebvallo in treating a serious post-transplant complication related to the Epstein-Barr virus, the FDA said Atara’s single-arm ALLELE study was “no longer considered to be adequate to provide evidence of effectiveness for accelerated approval,” according to Atara’s announcement. The company further noted that the agency’s “new position is contrary to the FDA’s prior guidance to Atara, the FDA’s alignment with Atara on the clinical trial data set, and the acceptance of the trial design as a single arm study as relevant for this patient population at BLA submission.” The chief executives of Replimune and Capricor shared similar sentiments after rejections last year. Speaking on a BioSpace webinar in November, BMO Capital Markets analyst Evan Seigerman spoke to this trend, specifically pointing to Replimune, which in July was hit with a surprise rejection for its advanced melanoma candidate RP1. “Today, they had one set of guidance from FDA with regard to clinical trial design, or how they could have a unique trial design . . . and then all of a sudden, there’s a 180,” Seigerman said of the company’s experience, “and it throws everything into a tailspin.” FDA FDA Reversals Send UniQure, Biohaven, Capricor, More Into a ‘Tailspin’ Since July, several biotechs have been forced to pivot as previous agreements with the FDA around evidence required for approval were reversed, a phenomenon that, according to experts, could portend a more restrictive regulator. November 24, 2025 · 7 min read · Heather McKenzie Beam, Cabaletta, PTC and BridgeBio are hoping for better luck. Here is how their regulatory pathways are lining up as they embark on 2026. Beaming Up to an Accelerated Pathway With AATD Alignment Beam Therapeutics, which is taking key steps this year to transition to a commercial-ready company, announced that it has aligned with the FDA on a potential accelerated approval pathway for BEAM-302 in alpha-1 antitrypsin deficiency (AATD). The investigational gene editor showed positive early results in a Phase I/II trial in March 2025, demonstrating the first-ever genetic correction in the rare disease, which is characterized by no or insufficient expression of the AAT protein. The accelerated pathway for BEAM-302 will be based on AAT biomarkers evaluated over 12 months, Beam said in a press release issued Jan. 11, two days before the company’s presentation at JPM26. The company plans to enroll around 50 additional patients who will be treated with the optimal biologic dose in an expansion of the Phase I/II study. “We believe this defined regulatory path for BEAM-302 significantly de-risks development,” Wainwright analysts wrote on Wednesday. Beam’s autologous cell therapy for sickle cell disease (SCD), risto-cel, is even closer to the regulatory finish line, with the company expecting to biologics license application by the end of 2026. Cabaletta Reaches Consensus With FDA on Registrational Lupus Cohorts Meanwhile, Cabaletta Bio has reached alignment with the FDA on new registrational cohort designs for its CD19 CAR T cell therapy rese-cel in Phase I/II trials in systemic lupus erythematosus (SLE) and lupus nephritis (LN). However, the plan involves the use of single-arm cohorts, which have been a point of contention in some of the FDA’s seemingly inconsistent guidance. Under the agreed-upon design, rese-cel will be evaluated in two independent, single-arm cohorts, one comprising patients with non-renal SLE and the other made up of patients with LN, according to Cabaletta’s Jan. 12 press release . Each cohort will include approximately 25 patients. Cabaletta is even further along with rese-cel in dermatomyositis (DM) and antisynthetase syndrome (ASyS), as the biotech launched these “FDA-aligned” registrational cohorts in December. “In our view, Cabaletta continues to execute on advancing rese-cel in multiple indications, as highlighted by the initiation” of this registrational trial, William Blair analysts said in a Monday note. And Cabaletta is expecting complete Phase I/II data from rese-cel in SLE, systemic sclerosis (SSc) and myasthenia gravis (MG) in the first half of 2026, according to its announcement. “While we believe the company has several opportunities in 2026 for value creation, the most critical will likely be potential regulatory alignment in SSc and MG and updated clinical data from its trials without preconditioning,” William Blair said on Monday. PTC Gives Positive Huntington’s Update After Mixed Phase II Reaction PTC Therapeutics had a mixed 2025 from a regulatory standpoint. In July, the rare disease–focused biotech won FDA approval for Sephience for children and adults with phenylketonuria (PKU). However, in August the FDA rejected the company’s Friedreich’s ataxia candidate, vatiquinone, saying in its CRL that “substantial evidence of efficacy was not demonstrated.” In a press release issued ahead of CEO Matthew Klein’s JPM presentation on Jan. 12, Klein lauded the approval and “strong start to the global launch” of Sephience. Earlier in the pipeline, PTC is developing votoplam for Huntington’s disease. On Monday, the company announced it had aligned with the FDA on design of a global Phase III trial, which it plans to initiate in the first half of this year. During the end-of-Phase II meeting, held during the fourth quarter last year, the FDA also confirmed its openness to a potential accelerated approval pathway for the asset, PTC said, given the significant unmet need in Huntington’s. Neuroscience Deep Dive: Huntington’s at a Crossroads Recent breakthroughs and three decades of progress in treating Huntington’s disease January 6, 2026 · 2 min read · Heather McKenzie PTC518, a small molecule that includes a premature stop codon in the huntingtin (HTT) mRNA, lowered blood HTT protein levels by 23% at the lowest dose in the Phase II PIVOT-HD trial, PTC reported in May 2025. Earlier-stage patients also saw functional improvements. While the trial met its primary endpoint, analysts were less than impressed. Willliam Blair at the time noted that patients with later-stage disease did not show any functional benefit on PTC518 and protein reduction in cerebrospinal fluid was less robust in these patients. In a note to investors on Monday, William Blair analysts said they “view alignment with the FDA on the trial design for votoplam in Hungtinton’s [sic] disease as positive, and look forward to the company sharing additional details regarding the trial’s design and what endpoints could be used to support accelerated approval.” BridgeBio’s Muscular Dystrophy Drug on Track for Traditional Approval Everything is coming up roses for BridgeBio, which delivered what Truist Securities in a Monday note called a “ surprise beat ” for transthyretin amyloid cardiomyopathy (ATTR-CM) drug Attruby in Q4, 2025. The $146 million in revenue, reported during the company’s JPM presentation on Jan. 12, represented a growth of 35% quarter-on-quarter, according to Truist. On the regulatory front, BridgeBio reported that two end-of-Phase II meetings with the FDA had taken place, for encaleret in chronic hypoparathyroidism and ribitol in limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). For ribitol, BridgeBio suggested that the FDA indicated alignment on pursuing traditional full approval in LGMD2I/R9 based on positive functional data at the trial’s interim analysis, according to Truist. The company plans to submit new drug applications for both assets to the FDA in the first half of 2026. .responsive-container { display: flex; flex-wrap: wrap; border: 1px solid #ededed; font-family: Helvetica, Arial, Sans-Serif; padding: 20px 20px 10px 20px; } .column-left { flex: 1; max-width: 45%; padding: 20px 20px 10px 20px; } .column-right { flex: 2; padding: 20px 20px 10px 20px; } @media (max-width: 768px) { .column-left, .column-right { max-width: 100%; flex: 100%; padding: 10px 0; } } Subscribe to ClinicaSpace! 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Clinical ResultPhase 3Phase 2Drug Approval

100 Deals associated with Vatiquinone

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D10407	-	-	DB11917

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Friedreich Ataxia	NDA/BLA	United States	PTC Therapeutics, Inc.	19 Feb 2025
Mitochondrial Diseases	Phase 3	Sweden	PTC Therapeutics, Inc.	08 Feb 2023
Autosomal Recessive Disorder	Phase 3	Germany	PTC Therapeutics, Inc.	14 Dec 2022
MELAS Syndrome	Phase 3	United States	PTC Therapeutics, Inc.	22 Jun 2022
MELAS Syndrome	Phase 3	Japan	PTC Therapeutics, Inc.	22 Jun 2022
MELAS Syndrome	Phase 3	France	PTC Therapeutics, Inc.	22 Jun 2022
MELAS Syndrome	Phase 3	Italy	PTC Therapeutics, Inc.	22 Jun 2022
MELAS Syndrome	Phase 3	Poland	PTC Therapeutics, Inc.	22 Jun 2022
MELAS Syndrome	Phase 3	Spain	PTC Therapeutics, Inc.	22 Jun 2022
MELAS Syndrome	Phase 3	United Kingdom	PTC Therapeutics, Inc.	22 Jun 2022

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05218655 (CTgov)	Phase 3	Mitochondrial Diseases \| Mitochondrial Encephalomyopathies \| MERRF Syndrome ... View more	101	Vatiquinone	ayadqtmlil = xprexupmsy gppqsdkqih (lrvmstkvww, ceslhaypxf - bgdmdfiton) View more	-	22 Dec 2025
NCT04577352 (MOVE-FA, MDA2025)	Phase 3	Friedreich Ataxia	143	Vatiquinone	yuuwcfpcav(wrkgkkljfe) = fvcphslrly mlpftbmsus (tvbacuafgw )	Positive	16 Mar 2025
				Vatiquinone	yuuwcfpcav(jwplkxwdeo) = vpuevdyrdh osledqfrpb (facicjttea ) View more
NCT04577352 (MOVE-FA, MDS2024)	Phase 3	Friedreich Ataxia	123	Vatiquinone	mzxcnzajss(jqhyewpxrd) = nigzghsude fyaonqfeqg (lmdabpsvmi ) View more	Positive	27 Sep 2024
				Placebo	-
NCT04577352 (MOVE-FA, MDS2024 \| MDA2024)	Phase 3	Friedreich Ataxia	143	Vatiquinone	etytyzixig(fiordnwlbi) = zvbcipaofl dhrmkmssoy (qdsmkksxjy )	Positive	27 Sep 2024
				Placebo	-
NCT04378075 (MIT-E, NEWS 1 \| NEWS 2)	Phase 2/3	Mitochondrial Diseases	68	Vatiquinone	kmwyffvmmr(ugratadzqg) = yucsujqcbj srolarlmhx (qryhfyhwbh )	Negative	29 Jun 2023
				安慰剂	kmwyffvmmr(ugratadzqg) = wudgfubyty srolarlmhx (qryhfyhwbh )
NCT04577352 (MOVE-FA, MDA 12023 \| MDA 22023 \| NEWS 1 \| NEWS 2)	Phase 2/3	Friedreich Ataxia	143	Vatiquinone	ojyivrfgoj(qjsmkjqhbp) = ieepkarast nbwtkvvsto (lfkouwapim )	Negative	23 May 2023
				安慰剂	ojyivrfgoj(qjsmkjqhbp) = unavowogdw nbwtkvvsto (lfkouwapim )
NCT01642056 (CTgov)	Phase 1/2	Mitochondrial Diseases \| Muscular Diseases	20	Placebo+EPI-743 (EPI-743, Then Placebo)	kmvqbyjivt(qbmwbqbigb) = cggpighhyo qvivomkhfo (qigketyghf, 2.8) View more	-	14 Apr 2021
				Placebo+EPI-743 (Placebo, Then EPI-743)	kmvqbyjivt(qbmwbqbigb) = ofhmpuhyea qvivomkhfo (qigketyghf, 3.7) View more
AAN 12016 \| AAN 22016	Phase 2	glutamine/glutamate levels	10	EPI-743 200 mg TID	jonrvvydxa(qiyxrubkns) = mncgmauhcl favzsupqlw (atmtrtplsv )	Positive	05 Apr 2016
				EPI-743 400 mg TID	jonrvvydxa(qiyxrubkns) = gejemuofbn favzsupqlw (atmtrtplsv )
AAN 12016 \| AAN 22016	Phase 2	-	10	EPI-743 200 mg TID	ecxnvdtkmp(kxkeprtnyl) = uvnygpqycj vfxiqagesn (osbljjqerp )	Positive	05 Apr 2016
				EPI-743 400 mg TID	ecxnvdtkmp(kxkeprtnyl) = kwbaannpxi vfxiqagesn (osbljjqerp )

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