PTC Therapeutics : Corporate Presentation – May 2026

Published: 2026-05-10 15:21:04 ET PTCT

Published on 05/10/2026 at 03:21 pm EDT

May 2026

US, EU & Japan approvals of Sephience

Strong start to Sephience global launch

Drive revenue and effectively manage OpEx

Advancement of early-stage R&D programs

$831M

Product Revenue $587M

* Revenue performance includes product and royalty revenue

$112M US

$13M ex-US

* As of March 31, 2026

Product Revenue1

Total Revenue2

$750 - 850M

28 - 45% YoY Growth

$1.08 - 1.18B

OpEx3

$680 - 720M

Product revenue excludes Evrysdi royalty and collaboration revenues; YoY based on $750-850M guidance.

Total revenue includes Evrysdi royalty and collaboration revenues.

OpEx is a Non-GAAP measure that includes only R&D and SG&A expenses for full year 2026, and excludes estimated non-cash, stock-based compensation expense of approximately $95M. GAAP R&D and SG&A expense for full year 2026 is anticipated to be betw een $775 and $815M.

Continue Sephience global launch momentum

Advance Phase 3 votoplam HD program

Advance early-stage

R&D programs

Move toward reaching cash flow breakeven

PKU Program

Katie, living w ith PKU

Precursor to BH4

Independent Chaperone Function

Sephience

SR*

7.8-Dihydrobiopterin (BH2)

DHFR**

BH4

Tetrahydrobiopterin (BH4)

Misfolded PAH Enzyme

Decreased Enzyme Activity

Chaperone Effect

Increased Enzyme Activity

Sephience significantly increases intracellular BH4 levels

Chaperone effect stabilizes enzyme, increases function, and enables treatment of BH4-non-responsive mutations

* SR, sepiapterin reductase (enzyme that converts Sephience to BH2)

** DHFR, dihydrof olate reductase (enzyme that converts BH2 to BH4) Lah, M. Advances in Therapy (In press)

Mean Absolute Change in Blood Phe from Baseline

100

Sephience BH4

-437.0 -256.6

-100

-200

-300

-400

70.3%

Greater reduction with Sephience

-500

p < 0.0001

-600

AMPLIPHY was a crossover study allowing for intra-subject

comparison of Sephience and BH4 treatment effect

Absolute least squares mean change in blood Phe concentration (µmol/L)

Meaningful diet liberalization

69% of subjects reached age-adjusted protein RDA

Improved cognitive function and mood

Improved concentration & slow thinking, and decreased irritability

Improved quality of life

Reduced disease impact on emotional, social, and familial wellbeing

Sephience Differentiated Profile Enables Penetration Into All Key Market Segments

On Treatment

Treatment Failures

Treatment Naive

T O TAL ADDRE S S ABL E M ARKE T

I N CL U DI NG AL L AG E S AN D S E V E RI T I E S

17K Patients in US

C ANAD A

UNI T E D S T AT E S

E URO P E

NO RT H AM E RI C A

E URO P E AS I A

L AT A M

Expect commercial patients in up to 30 countries by YE 2026

Votoplam

Huntington's Disease Program

A pril, living w ith HD

PTC

PIVOT-HD study met primary endpoint of blood HTT protein lowering at Week 12 with

durable dose-dependent lowering at Month 12

Evidence of dose-dependent disease slowing on cUHDRS in Stage 2 participants at

24 months

Continued evidence of favorable safety profile with no new AE signals identified for both dose levels and stages at 24 months

Mean (+/-SE) Change from Baseline of Composite UHDRS

-0.5

-1.0

-1.5

Baseline

Votoplam 10 mg

Mean difference in cUHDRS progression of 52% (10mg) and 28% (5mg)

at Month 24

Worsening

Votoplam 5 mg

NH Cohort

12 Month 24 Month

Treatment Group Mean (SE) Mean (SE)

Natural History (N=73) 5 mg (N=21)

10 mg (N=24)

-0.58 (0.15)

-0.42 (0.16)

-0.34 (0.21)

-1.20 (0.20)

-0.86 (0.24)

-0.57 (0.24)

Results based on observed data SE = Standard Error

INVEST-HD* Global Phase 3 Study Overview

Individuals with early symptomatic disease

3:2 randomization of votoplam 10mg: placebo

Target enrollment: ~770 participants in >30 countries

Primary endpoint: Change from baseline in cUHDRS

Treatment period up to 36 months

Interim analysis planned for efficacy and futility

*Study sponsored and funded by Novartis; NCT#: NCT07326709

Friedreich's Ataxia Program

Olivia, living w ith FA

72-week Placebo-Controlled MOVE-FA Trial* Long-term Extension Studies

Mean change from Baseline in mFARS Upright Stability score (+/- SE)

Vatiquinone Placebo

50% slowing (p<0.0001) of disease progression over 3 years ** in MOVE-FA longterm extension study

42%

Slowing p=0.021

4.8-point benefit (p<0.0001)

on mFARS** over 2 years in ambulatory and non-ambulatory adults

Weeks

* MOVE-FA did not meet its primary endpoint of statistically significant change in total mFARS at 72 w eeks

** Relative to matched FACOMS (Friedreich's Ataxia Clinical Outcome Measures Study) natural history cohort

Disclaimer

PTC Therapeutics Inc. published this content on May 10, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 10, 2026 at 19:20 UTC.