A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm.
HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.

Start Date24 Mar 2026

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT06254482

/ Active, not recruitingPhase 2

A Phase 2b, Double-Blind, Randomized Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease

The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of votoplam in participants with HD.

Start Date25 Aug 2023

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT05358717

/ CompletedPhase 2

A Phase 2A, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's Disease

The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.

Start Date03 Jun 2022

Sponsor / Collaborator

PTC Therapeutics, Inc.

100 Clinical Results associated with Votoplam

100 Translational Medicine associated with Votoplam

100 Patents (Medical) associated with Votoplam

Literatures (Medical) associated with Votoplam

01 Mar 2026·Clinical Pharmacology in Drug Development

Effects of High‐ and Low‐Fat Meals on the Bioavailability and Pharmacokinetics of Votoplam, a HTT Gene Splicing Modifier

Article

Author: Golden, Lee ; Kong, Ronald ; Lee, Lucy ; Beers, Brian ; Reddy, Nageswara ; Richards, Amy‐Lee

Abstract:

Votoplam is a novel, orally bioavailable, small molecule HTT gene splicing modifier that is being developed for the treatment of Huntington's disease. This was a single dose, open‐label, two‐period, crossover food effect study that evaluated the effect of high‐ and low‐fat meals on 20 mg votoplam in healthy participants. There was a washout of 21 days between the two periods. Twenty‐six participants completed the study. There were minimal changes in the bioavailability and pharmacokinetics of votoplam following administration of a single dose of 20 mg votoplam when taken with low‐fat and high‐fat meals relative to fasted condition. The mean C max , AUC 0–last , and AUC 0–inf for votoplam following administration of a single dose of 20 mg votoplam were 1.4‐fold, 1.2‐fold, and 1.1‐fold higher, respectively, in high‐fat fed conditions, and were 1.3‐fold, 1.1‐fold, and 1.1‐fold higher, respectively, in the low‐fat fed conditions, when compared to fasted conditions. There were no relevant safety findings in any of the treatment groups. Votoplam can be administered with or without food in patients.

01 Mar 2025·SYNLETT

Synthesis, Characterization, and Biological Applications of Tetramethylpiperidinyl Triazolopyridazine Derivatives

Author: Bharathkumar, Hanumantharayappa ; Gundekari, Sreedhar ; Keesara, Srinivasa Reddy ; Varkolu, Mohan ; Bolla, Ratnasekhar ; Andugulapati, Sai Balaji ; Gundekari, Sreedhar

Abstract:

We have synthesized a series of tetramethylpiperidinyl triazolopyridazine derivatives and screened the molecules for their biological activity against a cancer (NCI-H460) cell line. Among the tested molecules, 2-[3-(2,2,6,6-tetramethylpiperidin-4-yl)-3H-[1,2,3]triazolo[4,5-c]pyridazin-6-yl]-5-(2H-1,2,3-triazol-2-yl)phenol significantly inhibited cell growth with an IC50 value of 5.2 μM. We hope that this study will help in the development of better candidates for the treatment of lung cancer.

01 Dec 2024·BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Pharmacokinetics and pharmacodynamics of PTC518, an oral huntingtin lowering splicing modifier: A first‐in‐human study

Article

Author: Bredlau, Amy‐Lee ; Bhattacharyya, Anuradha ; Beers, Brian ; Kristensen, Allan ; Kong, Ronald ; Grant, Richard ; Gao, Lan ; Kaushik, Diksha ; Abd‐Elaziz, Khalid ; Golden, Lee

Abstract:

Aims:

PTC518 is an orally administered, centrally and peripherally distributed huntingtin (HTT) pre‐mRNA splicing modifier being developed for the treatment of Huntington's disease (HD) for which there is a high unmet medical need as there are currently no approved disease‐modifying treatments. This first‐in‐human study investigated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of PTC518 in healthy volunteers.

Methods:

This phase 1, single‐centre, randomized study in 77 healthy male and female volunteers evaluated the safety and tolerability and PK of PTC518 following single ascending doses and multiple ascending doses, PD as assessed by HTT mRNA and HTT protein levels after single and multiple doses, and food effects.

Results:

PTC518 demonstrated a favourable safety profile. The majority of treatment‐emergent adverse events were mild and transient. PTC518 Tmax was reached at 6–7 h and the terminal T1/2 was 54.0–75.3 h following a single oral dose. Exposure increased with dose though less than dose proportionally. The PTC518 concentrations in cerebrospinal fluid were approximately 2.6‐fold higher than the unbound free‐drug concentrations in plasma. A significant dose‐dependent reduction of up to approximately 60% in HTT mRNA and a significant dose‐dependent, time‐dependent and sustained reduction in HTT protein levels of up to 35% were observed after PTC518 treatment.

Conclusions:

PTC518 was well tolerated, and proof of mechanism of this novel splicing modifier was demonstrated by the dose‐dependent decrease in systemic HTT mRNA and HTT protein levels. Results from this first‐in‐human study support further studies in patients with HD and demonstrate the potential for PTC518 as a breakthrough treatment for HD.

News (Medical) associated with Votoplam

19 hours ago

·www.prnewswire.com

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

– First quarter 2026 total revenue of $273 million, including $226 million of product revenue, supporting full-year 2026 guidance raise – – Global Sephience™ (sepiapterin) launch momentum continues with first quarter 2026 revenue of $125 million, representing 36% quarter-over-quarter growth – – Positive topline results from 24-month interim analysis of PIVOT-HD extension study of votoplam, supporting ongoing global Phase 3 INVEST-HD study and potential regulatory interactions – – Open-label vatiquinone registration study to be initiated in Q3 2026 based on FDA feedback – WARREN, N.J., May 7, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ended March 31, 2026. "We are off to a strong start to 2026 with outstanding revenue performance this quarter that supports raising our full-year product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "The Sephience launch continues to be strong, with sustained momentum in the US and growing momentum internationally as more countries contribute to the global launch." Key Corporate Updates Q1 2026 total revenue of $273 million, with $226 million of product revenue Sephience global launch continues strong momentum Q1 2026 revenue of $125 million, including $112 million revenue in the US and $13 million revenue ex-US, up 36% from Q4 2025 1,244 total patients on commercial therapy worldwide as of March 31, 2026 Continued strong cadence of patient start forms in the US, with ~140 per month over past several months, surpassing 1,500 mark in the quarter First commercial sales in Japan in Q1 2026, with revenue expected from up to 30 countries by year-end 2026 Reported positive topline results in April 2026 from 24-month interim analysis of PIVOT-HD long-term extension study of votoplam, an oral small molecule splicing agent Dose-dependent benefit on cUHDRS in Stage 2 participants relative to natural history cohort, with 52% slowing of disease progression at 10 mg dose Continued favorable safety and tolerability with no treatment-related NfL spikes PTC and Novartis will complete data review and align on potential regulatory interactions Findings support ongoing global Phase 3 INVEST‑HD study of votoplam led by Novartis First patient dosed in Phase 3 INVEST-HD study, triggering $50 million milestone payment from Novartis to PTC in Q2 2026 Type C meeting with FDA held in April 2026 to discuss vatiquinone study to support NDA resubmission for Friedreich's ataxia program Based on meeting discussion and written feedback, PTC expects to initiate an open-label study using matched natural history control in Q3 2026 Primary endpoint will be change in mFARS from baseline to 24 months First Quarter 2026 Financial Highlights Total net product revenue and royalty revenue was $272.4 million for the first quarter of 2026, compared to $189.9 million for the first quarter of 2025. Total net product revenue across the commercial portfolio was $225.6 million for the first quarter of 2026, compared to $153.4 million for the first quarter of 2025, representing a 47% increase. Sephience net product revenues were $124.6 million for the first quarter of 2026, representing 36% growth compared to fourth quarter of 2025. Translarna™ (ataluren) net product revenues were $59.0 million for the first quarter of 2026, compared to $86.2 million for the first quarter of 2025. Translarna first quarter of 2026 revenue includes a large government purchase order from Brazil. Emflaza® (deflazacort) net product revenues were $21.5 million for the first quarter of 2026, compared to $47.8 million for the first quarter of 2025, due to continued generic erosion. Roche reported Evrysdi® (risdiplam) sales of approximately 464 CHF million, resulting in royalty revenue of $46.8 million to PTC for the first quarter of 2026, compared to $36.4 million to PTC for the first quarter of 2025. Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $100.9 million for the first quarter of 2026, compared to $109.0 million for the first quarter of 2025. Non-GAAP R&D expenses were $89.7 million for the first quarter of 2026, excluding $11.1 million in non-cash, stock-based compensation expense, compared to $100.3 million for the first quarter of 2025, excluding $8.7 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $86.2 million for the first quarter of 2026, compared to $81.0 million for the first quarter of 2025. Non-GAAP SG&A expenses were $73.9 million for the first quarter of 2026, excluding $12.3 million in non-cash, stock-based compensation expense, compared to $71.6 million for the first quarter of 2025, excluding $9.4 million in non-cash, stock-based compensation expense. Net loss was $2.8 million for the first quarter of 2026, compared to net income of $866.6 million for the first quarter of 2025. Cash, cash equivalents, and marketable securities were $1,892.5 million on March 31, 2026, compared to $1,945.4 million on December 31, 2025. Shares issued and outstanding as of March 31, 2026, were 82,882,024. PTC Updates Full-Year 2026 Financial Guidance Total product revenue guidance raised to $750 to $850 million, with expected total revenue of $1.08 to $1.18 billion GAAP R&D and SG&A expense guidance remains $775 to $815 million Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million Non-GAAP Financial Measures In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms CHF: Confoederatio Helvetica Francs (Swiss francs) cUHDRS: Composite Unified Huntington's Disease Rating Scale DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: US Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease mFARS: Modified Friedreich's Ataxia Rating Scale NDA: New Drug Application NfL: Neurofilament light chain nmDMD: Nonsense mutation Duchenne muscular dystrophy PKU: Phenylketonuria R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder To access the live webcast, please visit the "Events & Presentations" page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Facebook and Instagram. For more information please contact: Investors: Ellen Cavaleri +1 (615) 618-8228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "PTC Updates Full-Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the US on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo

Financial StatementPhase 3License out/inNDA

01 May 2026

·allsci.com

Novartis Q1’26 Earnings Call: Remibrutinib’s MS readout key near-term growth determinant

During its Q1 2026 earnings conference call, held April 28, 2026, Novartis framed remibrutinib’s summer MS readouts as the most important near-term swing factor for potential upside to its 2030 growth outlook, while using Q1 to defend Pluvicto’s peak-sales case after withdrawing a European pre-taxane filing and to temper expectations around unresolved accelerated-approval discussions for votoplam. Remibrutinib (Rhapsido) — MS and hidradenitis suppurativa (HS) as franchise-defining catalysts: CEO Vasant Narasimhan said a superior-to-anti-CD20 result in the two replicate Phase III relapsing MS trials, expected this summer, would represent a “massive” opportunity, while a non-inferior result would still position HS as a comparably sized indication. The HS Phase III program enrolled ahead of schedule and was accelerated into H2 2026, with management describing an oral-first positioning strategy ahead of biologics if data support it. Avidity-derived neuromuscular assets — first-to-market framing across DM1 and FSHD: Following the close of the Avidity acquisition, Novartis said del-desiran’s Phase III HARBOR study in myotonic dystrophy type 1 (DM1) is on track for an H2 2026 readout, and that del-zota for Duchenne muscular dystrophy (DMD) exon 44 skipping is tracking toward an NDA submission in H1 2026 pending chemistry, manufacturing, and controls (CMC) resolution. Management explicitly framed both assets as first-to-market opportunities and said the Antibody Oligonucleotide (AOC) modality acquired through Avidity would be applied across internal pipeline targets beyond the neuromuscular franchise. Pluvicto — EMA pre-taxane filing withdrawn, European path rerouted: Novartis withdrew its European pre-taxane metastatic castration-resistant prostate cancer (mCRPC) submission after failing to resolve the European Medicines Agency’s (EMA) objections to the comparator arm design, and said it would now wait for overall survival data from the hormone-sensitive prostate cancer (HSPC) setting before pursuing a new European filing. Management said the withdrawal had no impact on the $5 billion-plus peak sales forecast, noting that Japan and China retained access to both the PSMAfore and PSMA VISION populations. Remibrutinib HS efficacy bar — Phase II data difficult to interpret: Analyst Sachin Jain of Bank of America pressed management on the target efficacy profile for remibrutinib in HS relative to existing biologics, noting that the Phase II data featured an inverse dose response and low placebo-adjusted response rates. Narasimhan acknowledged the heterogeneity of the HS population and said the positioning relative to biologics would depend on the Phase III data profile, declining to specify a target efficacy threshold. Votoplam — Phase III dose confirmed but FDA engagement unresolved: Analyst Thibault Boutherin of Morgan Stanley asked whether new data from the open-label extension had increased confidence in an accelerated approval pathway. Management confirmed the 10-milligram dose for the pivotal Phase III study based on in-house data but said discussions with partner PTC Therapeutics on the optimal FDA interaction strategy, including the potential for accelerated approval, remained incomplete, with no further timeline provided. Summer 2026 — Remibrutinib Phase III MS dual readout: The two replicate Phase III studies in relapsing MS are expected to read out this summer. Management confirmed blinded safety data show no drug-induced liver injury or alanine transaminase (ALT) elevations attributable to remibrutinib, but said no visibility into annualized relapse rate data was available ahead of the readout. Narasimhan said a positive MS outcome was the single largest potential driver of upside to the company’s guided 5% to 6% growth outlook to 2030. Early H2 2026 — Pelacarsen HORIZON cardiovascular outcomes readout: The HORIZON study is powered for a 20% relative risk reduction in patients with lipoprotein(a) [Lp(a)] of 70 milligrams per deciliter or higher, and a 25% relative risk reduction in the 90-milligram-per-deciliter subgroup, with a median enrolled Lp(a) level of 108 milligrams per deciliter. Management said the follow-on once-yearly siRNA asset DII-235 is already Phase III-ready, signaling a deliberate two-asset Lp(a) franchise build regardless of the primary trial outcome, and noted that the American College of Cardiology and American Heart Association have now recommended universal Lp(a) testing once per lifetime in the United States. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

30 Apr 2026

·www.prnewswire.com

PTC Therapeutics to Report Results from PIVOT-HD Long Term Extension Study

April 28, 2026 -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call today, April 28 at 4:30 p.m. ET to share results from the 24-month interim analysis of the PIVOT-HD long-term extension study of votoplam. PTC's partner Novartis stated on its quarterly earnings call today that long-term extension data are supportive of the now-initiated Novartis Phase 3 INVEST-HD study and Novartis and PTC will assess potential best next steps for the program including further actions with FDA. To access the live webcast, please visit the "Events & Presentations" page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details. PIVOT-HD was designed as a 12-month placebo-controlled trial to assess pharmacodynamic effect and safety of votoplam at two dose levels--5mg and 10mg, relative to placebo. Initially, the study included only Stage 2 patients. A Stage 3 cohort of similar size was subsequently added to help identify the best study population for future studies. The primary endpoints of PIVOT-HD were total blood Huntingtin (HTT) lowering at 12 weeks and safety events. Secondary endpoints included 12-month blood HTT levels, and other blood-and central nervous system (CNS) biomarkers as well as changes in Composite Unified Huntington's Disease Rating Scale (cUHDRS). Following 12 months, patients were eligible to enroll in a long-term extension study in which all subjects would receive votoplam. Those originally randomized to 5mg and 10mg would continue at that dose level; those initially randomized to placebo would be randomized 1:1 to 5mg or 10mg. All subjects and investigators remain blinded to initial treatment assignment. Votoplam (formerly PTC518) is a small molecule splicing modifier that acts via a unique mechanism to promote the inclusion of a novel pseudoexon containing a premature termination codon, thus triggering Huntingtin (HTT) mRNA degradation and subsequent reduction in HTT protein levels. Votoplam was discovered from PTC's validated splicing platform, following the successful discovery and development of Evrysdi® (risdiplam) for spinal muscular atrophy (SMA). Votoplam was partnered with Novartis in December 2024. Following the completion of the PIVOT-HD clinical trial, Novartis assumed responsibility for votoplam's development, manufacturing and commercialization. Huntington's disease (HD) is a fatal, hereditary, genetic disorder of the central nervous system.1 It is caused by a defective gene. This gene produces a protein, called Huntingtin (HTT), which is involved in the functioning of the nerve cells in the brain (neurons). When the gene is defective, it produces an abnormal (or mutated) HTT protein that is toxic and causes neuron damage and neuron death.2 HD usually presents in people who are in their 30s or 40s. Symptoms can present earlier in life, and this is called Juvenile HD.2,3 There are also cases of infantile HD, when symptoms develop in children who are younger than 10 years old.2 While symptoms vary from person to person, the disease primarily affects the brain and results in abnormal movements, difficulties with speech, swallowing and walking, as well as a number of other symptoms including behavioral, cognitive and motor symptoms.4,5 While there are therapies approved for specific disease symptoms, currently, there is no cure for HD and there are no approved drugs that delay the onset or slow disease progression. 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. References: World Health Organization, 2020. 8A01.10 Huntington disease. Available at: https://icd.who.int/browse10/2019/en#/G10. Accessed October 2021. Gatto EM, González Rojas N, Persi G, et al. Clin Parkinsonism Rel Disord 2020;3:100056. Tabrizi SJ, Flower MD, Ross CA, et al. Nat Rev Neurol 2020;16(10):529–546. Roos RAC. Orphanet J Rare Dis 2010; 5:40. Kirkwood SC, Su JL, Conneally P, et al. Arch Neurol 2001;58(2):273–278. The content above comes from the network. if any infringement, please contact us to modify.

100 Deals associated with Votoplam

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Huntington Disease	Phase 3	United States	Novartis Pharmaceuticals Canada, Inc.	24 Mar 2026
Huntington Disease	Phase 3	Canada	Novartis Pharmaceuticals Canada, Inc.	24 Mar 2026
Huntington Disease	Phase 3	United Kingdom	Novartis Pharmaceuticals Canada, Inc.	24 Mar 2026

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05358717 (PIVOT-HD, CTgov)	Phase 2	Huntington Disease	159	Placebo (Placebo)	hdboypjkey = xgptashluz lydhcyjtdb (mgfygwyfch, chfbkypvrg - nbasncrefd) View more	-	13 Jan 2026
				PTC518 (PTC518 5 mg)	hdboypjkey = inxysfafsg lydhcyjtdb (mgfygwyfch, aqxwtsvyvm - kqthdtibid) View more
NCT05358717 (PIVOT-HD, PRNewswire \| Corporate Publications) Manual	Phase 2	Huntington Disease	159	PTC518 (votoplam) 5mg dose (Stage 2 Huntington's disease)	czvpnmyzig(bigqmcytyn) = nsjtuhlqnp nccadvxqgl (rekcghivdn, -34.4 to -12.4) Met View more	Positive	05 May 2025
				PTC518 (votoplam) 10mg dose (Stage 2 Huntington's disease)	czvpnmyzig(bigqmcytyn) = myagqkssvx nccadvxqgl (rekcghivdn, -49.4 to -28.7) Met View more
Pubmed Manual	Phase 1	-	77	PTC518	occbzrjcyw(gzoumyfvfk) = scfsdualbq hrtzlabrwx (ijxleuberb ) View more	Positive	18 Aug 2024
NCT05358717 (PIVOT-HD, Company_Website) Manual	Phase 2	Huntington Disease	-	PTC518 5mg	nnjtjfrtkl(tnljtvbdny) = zzjmkstcxe bwyfstbmwo (iisxsgsoak ) View more	Positive	20 Jun 2024
				PTC518 10mg	nnjtjfrtkl(tnljtvbdny) = xjxlpzogwm bwyfstbmwo (iisxsgsoak ) View more

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