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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): May 5, 2025

PTC THERAPEUTICS, INC.

(Exact Name of Company as Specified in Charter)

Delaware

    

001-35969

    

04-3416587

(State or Other Jurisdiction

(Commission

(IRS Employer

of Incorporation)

File Number)

Identification No.)

500 Warren Corporate Center Drive

    

Warren, NJ

07059

(Address of Principal Executive Offices)

(Zip Code)

Registrant’s telephone number, including area code: (908) 222-7000

Not applicable

(Former Name or Former Address, if Changed Since Last Report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

    

Trading Symbol(s)

    

Name of each exchange on which registered

Common Stock, $0.001 par value per share

PTCT

Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.

Item 7.01. Regulation FD Disclosure.

PTC Therapeutics, Inc. (the “Company”) will host a conference call on May 5, 2025 at 8:00 a.m. Eastern Time. During this conference call, the Company expects to discuss results from its Phase 2 study of PTC518 for the treatment of Huntington’s disease. Directions on how to access the conference call and a summary of the results are included in the press release furnished as Exhibit 99.1 hereto. A copy of the slide deck that will be presented during the conference call is furnished as Exhibit 99.2 hereto.

The information in this Item 7.01 of this Current Report on Form 8-K (this “Report”), including Exhibits 99.1 and 99.2, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing. All website addresses given in this Report or incorporated herein by reference are for information only and are not intended to be an active link or to incorporate any website information into this Report.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.

    

Description

99.1

Press Release, dated May 5, 2025 issued by PTC Therapeutics, Inc.

99.2

Corporate Presentation — PIVOT-HD Results

104

The cover page from this Current Report on Form 8-K, formatted in Inline XBRL

Signature

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this Report to be signed on its behalf by the undersigned hereunto duly authorized.

PTC Therapeutics, Inc.

Date: May 5, 2025

By:

/s/ Pierre Gravier

Name:

Pierre Gravier

Title:

Chief Financial Officer

Exhibit 99.1

PTC518 PIVOT-HD Study Achieves Primary Endpoint

- Study met primary endpoint with dose-dependent blood HTT protein lowering at Week 12 -

- Favorable dose-dependent trends across clinical scales in Stage 2 patients at Month 12 -

- Signals of dose-dependent clinical benefit relative to matched natural history cohort as well as dose-dependent lowering of NfL in Stage 2 patients at Month 24 -

- Continued favorable safety and tolerability profile with no treatment-related NfL spikes -

- PTC will host a conference call on May 5, 2025, at 8:00 am ET -

WARREN, N.J., May 5, 2025 - PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington’s disease (HD) patients. The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (p<0.0001) at Week 12 and favorable safety and tolerability. In addition, the 12-month data from the Stage 2 patients are consistent with the previously reported dose-dependent lowering of HTT protein and dose-dependent trends across clinical scales.

“These PIVOT-HD results confirm that PTC518 lowers Huntingtin protein and shows early signals of clinical benefit with a favorable safety profile," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. “In addition, at 24 months, we observed favorable dose-dependent trends on the cUHDRS and the TFC and SDMT subscales relative to natural history as well as dose-dependent lowering of neurofilament light chain protein. We look forward to discussions on the next development and regulatory steps including the potential for accelerated approval as we work to potentially bring the first disease-modifying therapy to those affected by Huntington’s disease.”

Results from the full 12-month cohort demonstrate dose-dependent lowering in blood HTT levels, with 23% at the 5mg dose level for both Stage 2 and 3 patients and 39% and 36% at the 10mg dose level for Stage 2 and 3 patients, respectively. For Stage 2 patients, there were dose-dependent trends of benefit on clinical scales including the Composite Unified Huntington's Disease Rating Scale (cUHDRS) and Total Motor Score (TMS) subscale. For Stage 3 patients, there were trends favoring the 5mg dose group relative to placebo, but not the 10mg dose group, suggesting that treatment effect may differ in Stage 3 patients relative to Stage 2 patients.

For all dose levels and disease stages, PTC518 showed a favorable safety and tolerability profile with no treatment-related serious adverse events or neurofilament light chain protein (NfL) spikes.


In addition, 24-month treatment data from the patients on whom data were shared last year (N=21) demonstrate signals of dose-dependent trends on the cUHDRS, Total Function Capacity (TFC) and Symbol Digit Modalities Test (SDMT) subscales when compared to a propensity matched natural history cohort from the ENROLL-HD Registry. At Month 24, there was also dose-dependent lowering of plasma NfL from baseline of -8.9% (nominal p=0.12) for the 5mg dose level and -14% (nominal p=0.03) for 10mg dose level.

Conference Call and Webcast Details:

PTC will hold a conference call at 8:00 am ET today to discuss this news. To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at https://ir.ptcbio.com/events-presentations. A replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days following the call.

About PIVOT-HD

PIVOT-HD was designed as a 12-month placebo-controlled trial to assess pharmacodynamic effect and safety of PTC518 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 PTC518. 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.

Presentation of study results is expected at a scientific meeting later in the year.

About PTC518

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. PTC518 was discovered from PTC's validated splicing platform, following the successful discovery and development of Evrysdi® (risdiplam) for spinal muscular atrophy (SMA).


About Huntington's Disease
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.

About PTC Therapeutics, Inc.
PTC is a global biopharmaceutical company that discovers, develops and commercializes clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to innovate new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. To learn more about PTC, please visit www.ptcbio.com and follow on Facebook, X, and LinkedIn.

For More Information:

Investors:
Ellen Cavaleri

+1 (615) 618-6228
ecavaleri@ptcbio.com

Media:
Jeanine Clemente
+1 (908) 912-9406
jclemente@ptcbio.com 

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 press release, other than statements of historic fact, are forward-looking statements, including statements with respect to the future expectations, plans and prospects for PTC, PTC's strategy, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses and other matters, 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 PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's most recent 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.

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.

References:

1.World Health Organization, 2020. 8A01.10 Huntington disease. Available at: https://icd.who.int/browse10/2019/en#/G10. Accessed October 2021.
2.Gatto EM, González Rojas N, Persi G, et al. Clin Parkinsonism Rel Disord 2020;3:100056.
3.Tabrizi SJ, Flower MD, Ross CA, et al. Nat Rev Neurol 2020;16(10):529–546.
4.Roos RAC. Orphanet J Rare Dis 2010; 5:40.
5.Kirkwood SC, Su JL, Conneally P, et al. Arch Neurol 2001;58(2):273–278.


Exhibit 99.2

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PTC Therapeutics PIVOT-HD Phase 2 Readout 1 Matthew B. Klein, MD CEO May 2025 PIVOT-HD Topline Results

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PTC Therapeutics PIVOT-HD Phase 2 Readout Forward Looking Statements 2 This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements with respect to the future expectations, plans and prospects for PTC, PTC's strategy, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses and other matters, 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 PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's most recent 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. The forward-looking statements contained herein represent PTC's views only as of the date of this presentation 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 presentation except as required by law.

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PTC Therapeutics PIVOT-HD Phase 2 Readout PIVOT-HD Study Design 3 *Placebo subjects randomized 1:1 to 5 mg or 10 mg for OLE and all subjects remain blinded to initial treatment assignment Primary Endpoint: Reduction in Blood HTT protein (Week 12) 12 Weeks Placebo-Controlled PTC518 5 mg PTC518 10 mg 12 Months Placebo 48 Months Open-Label Extension Study* PTC518 5 mg PTC518 10 mg PTC518 5 mg PTC518 10 mg Screening R

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PTC Therapeutics PIVOT-HD Phase 2 Readout Dose-Dependent mHTT Lowering and Safety Confirmed with Signals of Longer-term Dose-Dependent Biomarker and Clinical Effect 4 Continued favorable safety and tolerability profile with no treatment-related NfL spikes Favorable and dose-dependent trends on clinical scales at Month 12 in Stage 2 subjects Long-term positive clinical trends at Month 24 relative to natural history and dose-dependent NfL lowering Study met primary endpoint of blood HTT protein lowering at Week 12 with durable dose-dependent lowering at Month 12

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PTC Therapeutics PIVOT-HD Phase 2 Readout 5 12-Month Topline Results

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PTC Therapeutics PIVOT-HD Phase 2 Readout Baseline Characteristics of Enrolled Subjects * Includes 2 participants initially randomized to Stage 2 but reassigned to Stage 3 based on TFC 6 Baseline Characteristic Stage 2 Stage 3 Placebo (N=29) PTC518 5 mg (N=27) PTC518 10 mg (N=25) Placebo (N=24) PTC518 5 mg (N=25) PTC518 10 mg (N=29)* Age (years) mean 45.4 45.5 46.8 52.4 53.1 48.7 Gender, n (%) Male Female 19 (65.5%) 10 (34.5%) 12 (44.4%) 15 (55.6%) 12 (48.0%) 13 (52.0%) 14 (58.3%) 10 (41.7%) 14 (56.0%) 11 (44.0%) 17 (58.6%) 12 (41.4%) CAG length Mean (SD) Min – Max 44.3 (2.45) 41 – 49 44.1 (2.03) 41 – 49 43.7 (2.61) 41 – 50 43.2 (2.17) 40 – 48 43.1 (2.65) 40 – 50 44.1 (2.97) 40 – 50 TFC (Total Functional Capacity) Score Mean 13.0 13.0 13.0 11.6 11.6 11.9

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PTC Therapeutics PIVOT-HD Phase 2 Readout PTC518 Treatment Resulted in Dose-Dependent Blood HTT Lowering 7 Biomarker Month 12 Mean (SD) Treatment Effect PTC518 5 mg PTC518 10 mg mHTT Blood (95% CI) -23.3% (-33.4, -13.2) -36.1% (-45.9, -26.4) Biomarker Month 12 Mean (SD) Treatment Effect PTC518 5 mg PTC518 10 mg mHTT Blood (95% CI) -23.4% (-34.4, -12.4) -39.1% (-49.4,-28.7) Primary Endpoint of Blood HTT Lowering at Week 12 Achieved (p<0.0001) and Maintained at Month 12 (p<0.0001) mHTT CSF (95% CI) -20.8% (-47.4, 5.7) -22.3% (-48.3, 3.6) mHTT CSF (95% CI) -23.2% (-51.4, 4.9) -26.3% (-53.7, 1.1) Stage 2 Stage 3

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PTC Therapeutics PIVOT-HD Phase 2 Readout Favorable Trends of NfL Lowering in Stage 2 Subjects and no Treatment-Related Spikes 8 Stage 3 Mean plasma NfL % change from baseline to Month 12 Stage 2 Mean plasma NfL % change from baseline to Month 12 -15 -10 -5 0 5 10 15 Placebo PTC518 5 mg PTC518 10 mg 4.3% -1.3 % 4.7 % -15 -10 -5 0 5 10 15 Placebo PTC518 5 mg PTC518 10 mg 0.05 % -6.5 % -2.8 % CSF NfL (pg/mL) Treated Subject NfL Trajectories (Stage 2 and 3) 25000 20000 15000 10000 5000 0 Baseline Month 3 Month 6 Month 9 Month 12

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PTC Therapeutics PIVOT-HD Phase 2 Readout Caudate Brain Volume Changes Across Treatment Groups 9 Based on exploratory analysis Stage 2 Stage 3 0 -5 -10 -15 -20 Placebo PTC518 5 mg PTC518 10 mg Mean (SD) % change from baseline to Month 12 -4.6 % -4.4 % -4.9 % Placebo PTC518 5 mg PTC518 10 mg 0 -5 -10 -15 -20 Mean (SD) % change from baseline to Month 12 -7.2 % -4.4 % -6.9 %

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PTC Therapeutics PIVOT-HD Phase 2 Readout Early Signals of Favorable Effect on Clinical Scales in Stage 2 Subjects 10 Clinical Scale Mean (SD) Change Baseline to Month 12 Placebo (N=29) PTC518 5 mg (N=27) PTC518 10 mg (N=25) cUHDRS -0.49 (1.25) -0.41 (0.70) -0.27 (0.99) TFC -0.3 (0.78) -0.2 (0.49) -0.5 (0.59) TMS 3.3 (5.45) 1.5 (4.10) 0.7 (6.39) SDMT 0.0 (5.39) -1.1 (4.57) 1.1 (4.79) SWR -1.8 (13.16) -2.4 (7.97) -1.7 (8.76) Mean (SD) Change Baseline to Month 12 Placebo (N=24) PTC518 5 mg (N=25) PTC518 10 mg (N=29) -0.82 (1.26) -0.78 (1.40) -1.43 (1.32) -0.5 (0.95) -0.4 (1.24) -1.2 (1.56) 1.9 (7.01) 3.6 (5.90) 5.5 (7.27) -1.7 (5.99) -0.7 (6.52) -2.6 (3.75) -5.3 (12.42) -5.6 (10.20) -3.8 (11.97) Lower scores on cUHDRS, TFC, SDMT and SWR reflect worsening disease; higher scores on TMS reflect worsening disease Stage 2 Stage 3

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PTC Therapeutics PIVOT-HD Phase 2 Readout PTC518 Treatment Showed Favorable Safety and Tolerability Profile at Month 12 11 Most common adverse events were nasopharyngitis, influenza, headache and falls Similar adverse event profile across all treatment groups and disease stages, including placebo PTC518 was well tolerated with no treatment-related SAEs nor dose-limiting toxicities

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PTC Therapeutics PIVOT-HD Phase 2 Readout 12 AE Category Stage 2 Stage 3 Placebo (N=29) PTC518 5 mg (N=27) PTC518 10 mg (N=25) Placebo (N=24) PTC518 5 mg (N=25) PTC518 10 mg (N=29) TEAEs, n (%) 26 (89.7) 22 (81.5) 22 (88.0) 20 (83.3) 21 (84.0) 27 (93.1) TESAEs,* n (%) 3 (10.3) 1 (3.7) 1 (4.0) 1 (4.2) 0 1 (3.4) TEAEs resulting in death, n (%) 0 1 (3.7) 0 1 (4.2) 0 0 TEAEs leading to treatment discontinuation, n (%) 0 0 0 0 1 (4.0) 2 (6.9) TEAEs by maximum severity, n (%) Grade 1 Grade 2 Grade 3 Grade 4 Grade 5 15 (51.7) 8 (27.6) 2 (6.9) 1 (3.4) 0 10 (37.0) 10 (37.0) 1 (3.7) 0 1 (3.7) 8 (32.0) 12 (48.0) 2 (8.0) 0 0 12 (50.0) 7 (29.2) 0 0 1 (4.2) 13 (52.0) 8 (32.0) 0 0 0 12 (41.4) 12 (41.4) 3 (10.3) 0 0 * Only treatment-related SAE was in Stage 2 placebo subject PTC518 Treatment Showed Favorable Safety and Tolerability Profile at Month 12

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PTC Therapeutics PIVOT-HD Phase 2 Readout Month 12 Results Confirm Dose-Dependent mHTT Reduction, Safety and Early Trends of Favorable Clinical Effect Dose-dependent and durable lowering of HTT protein in blood Favorable safety profile with no treatment-related NfL spikes Early trends of favorable effect on clinical scales for Stage 2 subjects 13

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PTC Therapeutics PIVOT-HD Phase 2 Readout 14 24-Month Interim Results

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PTC Therapeutics PIVOT-HD Phase 2 Readout Baseline Characteristics of Subjects Included in 24-Month Interim Results (Stage 2) 15 Baseline Characteristic PTC518 5 mg (N=10) PTC518 10 mg (N=11) Age (years) Mean 44.7 46.8 Gender, n (%) Male Female 5 (50.0) 5 (50.0) 6 (54.5) 5 (45.5) CAG length Mean (SD) Min – Max 44.5 (2.07) 42 – 49 43.7 (2.41) 42 – 50 TFC (Total Functional Capacity) Score Mean 13.0 13.0

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PTC Therapeutics PIVOT-HD Phase 2 Readout Natural History Comparative Analysis Utilizing ENROLL-HD Disease Registry 16 • Age ≥ 25 years • CAG between 40 and 50 • TFC score of 13 • IS score of 100 • PINHD score between 0.18 to 4.93 • Propensity score weighting analysis applied to compare PTC518 treated subjects to Natural History cohort ENROLL-HD matched to enrollment criteria of PIVOT-HD • Propensity scores calculated with seven prognostic factors 1,045 Subjects Identified

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PTC Therapeutics PIVOT-HD Phase 2 Readout -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 Favorable Effect on cUHDRS at Month 24 Relative to Propensity-Matched Natural History Nominal p values from ad hoc natural history 17 comparator analysis -0.85 -0.80 PTC518 5 mg PTC518 10 mg cUHDRS Change from Baseline at Month 24 Worsening TMS trend at Month 12 not maintained at Month 24 PTC518 5 mg PTC518 10 mg -1 -0.8 -0.6 -0.4 -0.2 0 -0.53 -0.44 Natural History Control -0.74 TFC Change from Baseline at Month 24 Worsening p = 0.01 p < 0.001 PTC518 5 mg PTC518 10 mg -2.5 -2 -1.5 -1 -0.5 0 0.5 1 1.5 -0.96 0.73 Natural History Control -1.82 SDMT Change from Baseline at Month 24Worsening p = 0.05 p < 0.001 p = 0.05 p = 0.01 Natural History Control -1.1

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PTC Therapeutics PIVOT-HD Phase 2 Readout -25% -20% -15% -10% -5% 0% 5% 10% 15% Dose-Dependent NfL Lowering From Baseline At Month 24 * Change relative to subject baseline 18 % Change from Baseline in Mean plasma NfL -13.9% -9.0% PTC518 5 mg PTC518 10 mg p = 0.12* p = 0.03* Worsening Plasma NfL increases ~12% per year based on natural history data** **Parkin et al. Lancet 2024

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PTC Therapeutics PIVOT-HD Phase 2 Readout PTC518 Treatment Continues to Show Favorable Safety and Tolerability Profile at Month 24 19 Adverse event profile remains consistent through Month 24 No reported treatment-related SAEs nor NfL spikes at Month 24 PTC518 was well tolerated, with no dose-limiting toxicities

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PTC Therapeutics PIVOT-HD Phase 2 Readout Favorable Trends of Slowing of Progression and NfL Lowering at Month 24 20 Dose-dependent clinical trends of disease slowing relative to matched natural history at Month 24 Continued favorable safety and tolerability profile Dose-dependent NfL lowering at Month 24

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PTC Therapeutics PIVOT-HD Phase 2 Readout 21 Primary endpoint reached with durable mHTT lowering, safety and signals of clinical effect in Stage 2 patients Summary and Next Steps Complete data analyses and plan for development and regulatory discussions Dose-dependent trends on clinical scales and NfL lowering at Month 24

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PTC Therapeutics PIVOT-HD Phase 2 Readout

v3.25.1
Document and Entity Information
May 05, 2025
Document and Entity Information [Abstract]  
Document Type 8-K
Document Period End Date May 05, 2025
Entity File Number 001-35969
Entity Registrant Name PTC THERAPEUTICS, INC.
Entity Incorporation, State or Country Code DE
Entity Tax Identification Number 04-3416587
Entity Address, Address Line One 500 Warren Corporate Center Drive
Entity Address, City or Town Warren
Entity Address, State or Province NJ
Entity Address, Postal Zip Code 07059
City Area Code 908
Local Phone Number 222-7000
Written Communications false
Soliciting Material false
Pre-commencement Tender Offer false
Pre-commencement Issuer Tender Offer false
Title of 12(b) Security Common Stock, $0.001 par value per share
Trading Symbol PTCT
Security Exchange Name NASDAQ
Entity Emerging Growth Company false
Entity Central Index Key 0001070081
Amendment Flag false

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