69366JAB7 Filings — Ptc Therapeutics, Inc. - FilingSpy
69366JAB7
Ptc Therapeutics, Inc.
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A biopharmaceutical company that develops treatments for rare diseases, with medicines including Sephience for PKU, Translarna and Emflaza for Duchenne muscular dystrophy, Evrysdi for spinal muscular atrophy, and the gene therapy Upstaza/Kebilidi. Founded in 1998 by RNA researcher Stuart Peltz, the company's name comes from "post-transcriptional control," the field of RNA biology it was built around.
Q2 2026 revenue rose 101.5% to $360.5M on Sephience launch and a Novartis milestone
Sephience's first launch year drove product past pre-launch levels. Revenue rose 101.5% to $360.5M and was $0.92 as $151.3M in Sephience sales and a $50.0M Novartis milestone offset declines elsewhere. The company is profitable again, but a $287.5M note matures in September.
Key takeaways
Net product rose over 100% to $238.8M, driven by $151.3M in Sephience sales in its first launch year, partially offset by Emflaza declining to $11.7M on generic competition and Translarna falling to $17.3M after the EC's negative CHMP opinion.
Collaboration and license increased to $50.6M from $2.9M, primarily from a $50.0M milestone paid by Novartis for initiating the first Phase 3 trial of votoplam.
grew 23% to $71.1M on higher Evrysdi sales, the one growing recurring royalty line this quarter.
Section summaries
Management's Discussion and Analysis
Total revenue more than doubled to $361M driven by Sephience launch and higher Evrysdi royalties, while R&D and SG&A declined.
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Net product rose over 100% to $238.8M, driven by $151.3M in Sephience sales in its first launch year, partially offset by declines in Emflaza ($11.7M) and Translarna ($17.3M) due to generic competition and the EC's negative CHMP opinion, respectively.
was $149.0M versus a $40.2M loss in Q2 2025, and was $0.92 versus a $0.83 loss, as R&D fell 12% to $99.2M and SG&A fell 5% to $80.6M.
The company issued $550M in 0% due 2031 and used $328.8M to part of its 2026 convertible notes; cash and equivalents ended at $1,049.9M, up 1.6% .
Cost of product sales rose 74% to $19.9M and more than doubled to $11.8M, both driven by the Sephience launch and related milestones.
What changed
The FY2025 watch item on the $287.5M convertible senior notes maturing September 2026 remains open; the company issued $550M new notes and repurchased a portion of the 2026 notes, leaving the maturity to be fully resolved at due date.
Emflaza continued to decline from generic competition, reaching $11.7M this quarter versus $47.8M in Q1 2025 and a 23% Q2 2025 drop — the decline has not stabilized.
Sephience sales rose to $151.3M from $124.6M in Q1 2026, showing a launch ramp in its first two quarters.
Translarna fell to $17.3M this quarter from the EC withdrawal, after a 15% Q2 2025 drop and a 17% Q1 2026 decline — the EU loss continues to cut the line.
Evrysdi rose 23% to $71.1M, continuing the recurring growth seen in prior quarters (8% Q2 2025, 15% Q3 2025, 29% Q1 2026) before the remaining interest was sold to Royalty Pharma.
Q1 2026 had swung to a $2.8M loss after the prior-year Novartis payment dropped out; Q2 2026 returned to profit on Sephience and the Novartis milestone, reversing that dip.
What to watch
September 2026 maturity of the $287.5M convertible senior notes and whether cash or new financing covers the remaining principal after the $328.8M .
Q3 2026 Sephience sales trajectory after $151.3M in Q2 to gauge launch ramp.
Q3 2026 Emflaza to see if the decline to $11.7M stabilizes against generic competition.
Q3 2026 Translarna in remaining EU countries after the $17.3M Q2 figure from the EC withdrawal.
Collaboration and license increased to $50.6M from $2.9M, primarily due to a $50.0M milestone from Novartis for initiating the first Phase 3 trial of votoplam.
Royalty grew 23% to $71.1M on higher Evrysdi sales, while cost of product sales rose 74% to $19.9M and of acquired intangibles more than doubled to $11.8M, both driven by the Sephience launch and related milestones.
R&D expense decreased 12% to $99.2M due to lower development program spend, and SG&A decreased 5% to $80.6M primarily from reduced prelaunch activities for Sephience.
Cash provided by operations was $70.1M for the first half of 2026, down from $811.8M in the prior-year period which included a $1.0B upfront payment from Novartis; the company issued $550M in 0% convertible notes due 2031 and used $328.8M to a portion of its 2026 convertible notes.
Quantitative and Qualitative Disclosures About Market Risk
During the period ended June 30, 2026, there were no material changes in our market risk or how our market risk is managed, compared to those disclosed under the heading “Quantitative and Qualitative Disclosures about Market Risk” in our 2025 Annual Report. 54 Table of Contents
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During the period ended June 30, 2026, there were no material changes in our market risk or how our market risk is managed, compared to those disclosed under the heading “Quantitative and Qualitative Disclosures about Market Risk” in our 2025 Annual Report.
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Table of Contents
From time to time in the ordinary course of our business, we are subject to claims, legal proceedings and disputes, including as a result of patients seeking to participate in our clinical trials or otherwise gain access to our product candidates. We are not currently aware of a…
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From time to time in the ordinary course of our business, we are subject to claims, legal proceedings and disputes, including as a result of patients seeking to participate in our clinical trials or otherwise gain access to our product candidates. We are not currently aware of any material legal proceedings to which we are a party or of which any of our property is subject.
We have set forth in Item 1A to our Annual Report on Form 10-K for the year ended December 31, 2025, risk factors relating to our business, our industry, our structure and our common stock. Readers of this Quarterly Report on Form 10-Q are referred to such Item 1A for a more com…
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We have set forth in Item 1A to our Annual Report on Form 10-K for the year ended December 31, 2025, risk factors relating to our business, our industry, our structure and our common stock. Readers of this Quarterly Report on Form 10-Q are referred to such Item 1A for a more complete understanding of risks concerning us.