PTC Therapeutics (PTCT)
Develops rare-disease medicines spanning PKU, DMD, gene therapy, and RNA-splicing programs.
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Sells medicines for PKU, DMD, AADC deficiency, and other rare disorders across the United States and international markets, while developing RNA-splicing and inflammation programs. Revenue combines product sales with collaboration and royalty income, with regulatory approvals, reimbursement, and partners shaping the business.
Develops and commercializes medicines for rare neurological and metabolic disorders. The marketed portfolio spans Sephience for phenylketonuria, Translarna and Emflaza for Duchenne muscular dystrophy, Upstaza/Kebilidi gene therapy for AADC deficiency, and Latin American rights to Tegsedi and Waylivra. Sales reach the United States, Europe, Japan, Latin America, and other international markets.
Builds candidates through RNA-splicing and inflammation-and-ferroptosis platforms. Votoplam for Huntington’s disease is being advanced in a Novartis-funded late-stage study, supporting collaboration economics, while earlier programs target neurological, hematologic, and inflammatory diseases.
Commercial execution pairs global launches with country-level pricing and reimbursement work. Operations are managed as a single segment and depend on product authorizations, external manufacturers and distributors, and collaborators that develop or commercialize partnered assets.
Company facts
Target rangeAs of Sep 22, 2026
Latest analyst notes
| Date | Firm | Action | Prior | Current |
|---|---|---|---|---|
| Sep 22, 2026 | Cantor Fitzgerald | Reiterate | Rating: —Target: $130 | Target: $137 |
| Sep 14, 2026 | Leerink Partners | Target | Target: — | Target: $98 |
| Aug 14, 2026 | Barclays | Reiterate | Rating: —Target: $120 | Target: $122 |
| Aug 13, 2026 | Wells Fargo | Reiterate | Rating: —Target: $106 | Target: $109 |
| Jul 31, 2026 | Morgan Stanley | Reiterate | Rating: —Target: $94 | Target: $104 |
EPSReportedEstimate
RevenueReportedEstimate
Balance sheet, Jun 30, 2026Cash & short-term investments $2.23B · Total debt $696.5M · Total assets $3.25B · Shareholders' equity −$165.4M
- Revenue
- $360.5M
- Operating income
- $149.0M
- Net income
- $83.5M
No dividends on record.
| Institutions | 121.41% | 101.33M shares | |
|---|---|---|---|
| 392 holders · as of Jun 30, 2026 | |||
| Insiders | 6.55% | 5.47M shares | |
| 19 holders · as of Sep 18, 2026 | |||
| Strategic holders | 8.62% | 7.19M shares | |
| 1 holder · as of May 15, 2026 | |||
| Adds to | 136.58% | ||
83.46M company shares as of Sep 22, 2026 · institutions as of Jun 30, 2026 · holders as of their latest filing
These groups come from separate filings that can overlap, so they add to 136.58%; nothing is left for Other.
Top shareholders13F · 13D/G · Form 4
| Holder | Stake | Shares | Date |
|---|---|---|---|
BlackRock, Inc.Institution | 16.73% | 13.96M | as of Jun 30, 2026 |
RTW Investments, LPInstitution | 9.28% | 7.75M | as of Jun 30, 2026 |
Jupiter Topco LLCInstitution | 8.67% | 7.24M | as of Jun 30, 2026 |
Janus Henderson Group PLCStrategic | 8.62% | 7.19M | filed May 15, 2026 |
Vanguard Portfolio Management LLCInstitution | 5.90% | 4.93M | as of Jun 30, 2026 |
Insider tradesForm 4
| Date | Insider | Role | Shares | Avg price |
|---|---|---|---|---|
| Sep 17, 2026 | Michael Schmertzler | director | −10,000 | $67.85 |
| Sep 16, 2026 | Michael Schmertzler | director | −10,000 | $67.12 |
| Sep 10, 2026 | Michael Schmertzler | director | −10,000 | $67.34 |
| Sep 9, 2026 | Michael Schmertzler | director | −10,000 | $67.84 |
| Aug 27, 2026 | Michael Schmertzler | director | −12,500 | $71.58 |
- BioMarin
Direct PKU rival through PALYNZIQ and KUVAN
- Sarepta Therapeutics
Core DMD genetic-medicine competitor
- Biogen
FA and SMA overlap through SKYCLARYS and SPINRAZA
CompareAfter hours
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| PTC Therapeutics | $65.96 | +0.02% | $5.45B | — | 53.3 | 5.4 | +101.6% |
| BioMarin | $63.86 | +0.77% | $12.38B | 164.3 | 10.3 | 3.6 | +20.0% |
| Sarepta Therapeutics | $20.86 | +0.19% | $2.13B | — | 36.2 | 1.1 | −34.3% |
| Biogen | $225.38 | −0.01% | $32.37B | 38.8 | 17.0 | 3.3 | +3.4% |
| Median | 101.5 | 17.0 | 3.3 | +3.4% |