PTCT Stock — PTC Therapeutics Inc.: Cash, EV, Pipeline

What they do

We are a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. We are advancing a robust and diversified pipeline of transformative medicines as part of our mission to provide access to best-in-class treatments for patients with unmet medical needs.

Source: 10-K FY2025 via SEC EDGAR

Share price

$63.50

Market cap

$5,304.5M

Cash

$2,127.9M

Enterprise value

$6,073.9M

Balance sheet

Cash	$2,127.9M
Debt	$591.7M
Royalty-sale liability	$2,305.6M
Net cash	$1,536.2M
Enterprise value	$6,073.9M
Basic shares	83.5M shares
Fully diluted shares	98.8M shares
Fully diluted market cap	$6,276.4M
Cash per share	$25.47
Quarterly burn	+$129.1M generated

Cash figures as of 2026-09-30. Database snapshot: 2026-11-13.

Royalty-sale liability as of 2026-09-30 (10-Q); included in enterprise value.

Share structure

Common

83.5M

Pre-funded warrants

0.0M

Options

6.4M

RSUs

3.5M

Warrants

0.0M

Converts

5.5M

Conv preferred

0.0M

ESPP reserve

—

FDSO

98.8M

Cap table as of 2025-12-31.

Price history

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Next catalyst

PTC844 · Nov 11 - Dec 23 in 12 mo · Phase 2 · Rheumatoid Arthritis

Upcoming catalysts

Expected	Drug	Phase	Indication	Mgmt guide
Nov 11 - Dec 23 in 12 mo	PTC844	Phase 2	Rheumatoid Arthritis	—
Feb 11 - Mar 24 in 15 mo	Vatiquinone	Phase 3	Friedreich Ataxia	—
Apr 26 - Jun 7 in 30 mo	Vatiquinone	Phase 3	Friedreich's Ataxia	—
Apr 11 - May 23 in 54 mo	Sepiapterin	Phase 3	Phenylketonuria	—

Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.

Full readout calendar →

Pipeline assets

Sephience

Approved · Active

Aliases sepiapterin

Target phenylalanine hydroxylase (PAH)

Indications

Phenylketonuria (PKU) (Approved)

Status note Approved EC Jun 2025 (EEA), FDA Jul 2025 (US, age 1 month+), Japan Dec 2025, Brazil Feb 2026, plus additional geographies.

Translarna

Stage not disclosed · Active

Aliases ataluren

Indications

Nonsense mutation Duchenne muscular dystrophy (nmDMD) (Stage not disclosed)

Status note EC adopted CHMP negative opinion Mar 2025 (conditional MA not renewed; continued use possible via EU Directive 2001/83 Articles 117(3)/5(1)). US NDA resubmission withdrawn Feb 2026 after FDA feedback; no further US development planned. Authorizations remain in some geographies outside the EEA.

Emflaza

Approved · Active

Aliases deflazacort

Indications

Duchenne muscular dystrophy (Approved)

Status note Approved in US for DMD patients 2 years and older; lost US market exclusivity under the Orphan Drug Act.

Upstaza

Approved · Active Modality: gene therapy

Aliases eladocagene exuparvovec, Kebilidi

Target AADC (dopa decarboxylase gene)

Indications

Aromatic L-amino acid decarboxylase (AADC) deficiency (Approved)

Status note Approved EC Jul 2022 (EEA, 18 months+), UK Nov 2022, FDA Nov 2024 (marketed as Kebilidi in the US).

Evrysdi

Approved · Active

Aliases risdiplam

Target SMN2 (splicing modifier)

Partnerships Roche + SMA Foundation: SMA License Agreement (2011); PTC receives milestones and royalties.

Indications

Spinal muscular atrophy (SMA) (Approved)

Status note Approved FDA Aug 2020, EC Mar 2021, 100+ countries; label expanded to infants under 2 months (FDA May 2022, EC Aug 2023).

Votoplam

Phase 3 · Active Modality: splicing modifier

Aliases PTC518

Target huntingtin (HTT)

Partnerships Novartis: License and Collaboration Agreement (closed Jan 2025, $1.0B upfront); $50M milestone on Phase 3 initiation; development/regulatory/sales milestones + profit sharing + royalties.

Indications

Huntington's disease (Phase 3)

Catalysts

— H2 2026

Status note HD splicing-platform program. Novartis license closed Jan 2025 ($1.0B upfront). Global Phase 3 commenced Apr 2026 (~770 early symptomatic patients; interim analysis planned).

Vatiquinone

Stage not disclosed · Active Modality: small molecule

Target 15-lipoxygenase

Indications

Friedreich's ataxia (Stage not disclosed)

Catalysts

— Q3 2026

Status note Registration-directed Phase 3 MOVE-FA did not meet its primary endpoint (May 2023). NDA submitted Dec 2024, accepted Feb 2025, CRL Aug 2025. FDA suggested additional study; PROVE-FA open-label study (~120 patients, ages 7-21) planned Q3 2026.

PTC612

Phase 1 · Active Modality: small molecule (oral)

Target NLRP3

Status note Oral NLRP3 inhibitor; Phase 1 in healthy volunteers initiated Q2 2026.

PTC844

Stage not disclosed · Active Modality: small molecule (oral)

Target DHODH

Indications

Rheumatoid Arthritis (Phase 2)

Catalysts

— Q3 2026

Status note Next-generation DHODH inhibitor; Phase 2a planned.

Tegsedi

Approved · Active

Aliases inotersen

Target TTR

Partnerships Ionis/Akcea: PTC holds commercialization rights in Latin America and the Caribbean.

Indications

Hereditary transthyretin amyloidosis polyneuropathy (Approved)

Status note Approved US, EU, Brazil for stage 1/2 polyneuropathy in hATTR amyloidosis.

Waylivra

Approved · Active

Aliases volanesorsen

Partnerships Ionis/Akcea: PTC holds commercialization rights in Latin America and the Caribbean.

Indications

Familial chylomicronemia syndrome (Approved)
Familial partial lipodystrophy (Approved)

Status note Approved Brazil (Aug 2021, first FCS treatment; Dec 2022 for familial partial lipodystrophy) and EU for FCS.

Per-asset detail extracted from the 10-Q filed 2026-07-30. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.

No lead program on file for this company.

Active clinical trials

Trial	Phase	Status	Role	Sponsor	BP	Condition(s)	Mgmt guide
NCT05515536	Phase 3	Active, not recruiting	Sponsor	PTC Therapeutics	—	Friedreich Ataxia	—
NCT07681713	Phase 3	Not yet recruiting	Sponsor	PTC Therapeutics	—	Friedreich's Ataxia	—
NCT06302348	Phase 3	Recruiting	Sponsor	PTC Therapeutics	—	Phenylketonuria	—
NCT07779616	Phase 2	Not yet recruiting	Sponsor	PTC Therapeutics	—	Rheumatoid Arthritis	—
NCT04903288	Phase 2	Active, not recruiting	Sponsor	PTC Therapeutics	—	AADC Deficiency	—

Sponsor-matched active trials from ClinicalTrials.gov (5 as sponsor, 0 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.

View all 5 active trials for PTC Therapeutics on ClinicalTrials.gov

Specialist fund holders

Tracked specialist funds: ADAR1 Capital Management, Aisling Capital, Ally Bridge Group, Avoro Capital Advisors, BVF Partners, Baker Bros. Advisors, Boxer Capital, Braidwell LP, Casdin Capital, Cormorant Asset Management, DAFNA Capital, Deerfield Management, EcoR1 Capital, Frazier Life Sciences, Great Point Partners, Ikarian Capital, Logos Global Management, OrbiMed Advisors, Perceptive Advisors, RA Capital Management, RTW Investments, Sphera Biotech, Stonepine Capital, Tang Capital Partners, Vivo Capital.

Fund	Shares	Q/Q change	% of portfolio	Filing
RTW Investments	7,746,415	no change	5.39%	2026-08-14
Tang Capital Partners	1,500,000	no change	5.56%	2026-08-14
Boxer Capital	200,000	no change	1.23%	2026-08-14
OrbiMed Advisors	49,100	no change	0.08%	2026-08-14

Specialist-fund positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.

Latest news

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PTCT — PTC Therapeutics Inc. ☆

What they do

Balance sheet

Share structure

Price history

Next catalyst

Upcoming catalysts

Pipeline assets

Active clinical trials

Specialist fund holders

Latest news

PTCT — PTC Therapeutics Inc.