Every 8-K that PTC Therapeutics, Inc. (PTCT) has filed with the SEC in the last 24 months is listed below, newest first, and each one links through to the document itself with the summary and the scores our analysis gives it.
A 8-K covers material events a company has to report between its quarterly reports, so if you follow PTCT and want that one kind of document rather than the whole filing history, this is the page to keep. The company's other filings, of every form, are on the full PTCT filings page.
PTC Therapeutics, Inc. (PTCT) announced completion of its acquisition of ST-920, a BLA-stage, one-time administered AAV gene therapy product candidate for Fabry disease, from Sangamo Therapeutics. PTC plans to complete a rolling biologics license application submission to the U.S. Food and Drug Administration for accelerated approval of ST-920 in the fourth quarter of 2026.
The planned BLA is based on evidence of meaningful favorable clinical effect on renal function and a safety and tolerability profile over 52 weeks in the Phase 1/2 STAAR study. PTC also expects to pursue regulatory approval of ST-920 outside the United States. ST-920 (isaralgagene civaparvovec) has received multiple regulatory designations, including Orphan Drug, Fast Track and RMAT from the FDA, Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency, and access via the U.K. Innovative Licensing and Access Pathway.
PTC Therapeutics, Inc. (PTCT) agreed to acquire from Sangamo Therapeutics all assets primarily related to ST-920, a one-time AAV gene therapy product candidate for Fabry disease, and assume certain specified liabilities. The consideration includes an upfront cash payment of $111 million plus up to $100 million in contingent FDA-approval milestones.
The milestones comprise $80 million upon FDA accelerated approval of a BLA for ST-920 based on Sangamo’s STAAR Studies and $20 million upon full FDA approval based on the same studies. The transaction follows PTCT’s selection as winning bidder for ST-920 in Sangamo’s Chapter 11 bankruptcy auction.
Closing is subject to customary conditions, including a Delaware Bankruptcy Court order approving the acquisition, expiration or termination of the Hart-Scott-Rodino waiting period, and each party’s representations and covenants being satisfied. Either party may terminate if closing has not occurred by October 15, 2026. A Bankruptcy Court hearing to consider approval is scheduled for September 10, 2026.
PTC Therapeutics, Inc. plans to acquire ST-920, a BLA-stage, one-time administered AAV gene therapy for Fabry disease, from Sangamo Therapeutics after being selected as winning bidder in a competitive bankruptcy auction. Terms include a $111 million upfront payment and up to $100 million in contingent milestones tied to U.S. FDA approvals.
The transaction is subject to definitive documentation, bankruptcy court approval, antitrust review and other customary closing conditions, and is expected to close in late third or early fourth quarter 2026. A rolling BLA submission for accelerated approval is expected to be completed in the fourth quarter of 2026, with a potential commercial launch in 2027 and additional regulatory filings planned outside the United States.
PTC Therapeutics reported strong second-quarter 2026 results, with total revenue of $361 million, including $239 million of product revenue. Sephience generated $151 million of Q2 2026 revenue and had 1,647 patients on commercial therapy worldwide as of June 30, 2026. Total net product and royalty revenue was $309.9 million versus $175.9 million a year earlier, and net income attributable to common stockholders was $83.5 million, compared with a $64.8 million loss in Q2 2025; diluted EPS was $0.92.
The company raised full-year 2026 guidance, targeting total revenue of $1.18 to $1.28 billion and product revenue of $850 to $950 million. Projected 2026 GAAP R&D and SG&A expense is $775 to $815 million, with non-GAAP expected at $680 to $720 million. Cash and marketable securities were about $2.2 billion as of June 30, 2026, with total debt of $590.97 million and a $2.32 billion liability for sale of future royalties.
Pipeline updates included positive 24‑month PIVOT‑HD data for votoplam showing 52% dose‑dependent disease slowing on the cUHDRS at the 10 mg dose in Stage 2 participants, and a Novartis-funded Phase 3 INVEST‑HD trial (~770 patients) that triggered a $50 million milestone payment. PTC named PTC303 as its next MSH3 splicing candidate, initiated Phase 1 for PTC612 and plans a Phase 2a study of PTC844 in Q3 2026. The company also appointed Hege Sollie‑Zetlmayer to its board as a Class III director, granting equity awards under its director compensation policy.
PTC Therapeutics, Inc. completed a private offering of $550,000,000 aggregate principal amount of 0.0% Convertible Senior Notes due 2031. The notes carry no regular interest, may only bear special interest in limited circumstances, and mature on June 15, 2031 unless earlier converted, redeemed or repurchased.
The notes are initially convertible at 9.3042 shares of common stock per $1,000 principal amount, equivalent to a conversion price of approximately $107.48 per share. PTC estimates net proceeds of about $535.5 million and is using approximately $328.8 million to repurchase for cash $222.0 million principal of its 1.50% Convertible Senior Notes due 2026, leaving $55.5 million of those existing notes outstanding. The new notes are senior unsecured obligations with customary conversion, redemption, repurchase and default provisions.
PTC Therapeutics has priced a private Offering of $500.0 million aggregate principal amount of 0.00% Convertible Senior Notes due 2031, with an option for initial purchasers to buy an additional $50.0 million. PTC estimates net proceeds of about $486.8 million (or $535.5 million if the option is fully exercised).
The company expects to use approximately $328.8 million of the proceeds to repurchase for cash $222.0 million principal amount of its 1.5% Convertible Senior Notes due 2026, and to use the remainder for general corporate purposes, including potential additional 2026 note repurchases and repayment at maturity. The Notes mature on June 15, 2031, carry no coupon, and are convertible at an initial rate of 9.3042 shares per $1,000, implying a conversion price of about $107.48 per share, a roughly 40% premium to the $76.77 closing share price on June 15, 2026.
PTC Therapeutics reports that board member Alethia Young resigned from its board of directors effective June 8, 2026. She stated that the decision reflects the need to focus on other work commitments and was not due to any disagreement with the company.
On the same date, Young entered into a Consulting Agreement under which she will continue as an advisor to PTC Therapeutics through June 8, 2027. For these advisory services, she will receive a monthly consulting fee of $4,166.67, as detailed in Exhibit 10.1 to the report.
PTC Therapeutics, Inc. reported the results of its annual stockholder meeting held on June 2, 2026. As of the April 10, 2026 record date, 82,911,783 common shares were outstanding, with each share entitled to one vote.
Stockholders elected four Class I directors to serve until the 2029 annual meeting: Jessica Chutter, Matthew B. Klein, M.D., M.S., F.A.C.S., Stephanie S. Okey, M.S., and Jerome B. Zeldis, M.D., Ph.D., each receiving over 68.9 million votes “For.”
Ernst & Young LLP was ratified as independent registered public accounting firm for the year ending December 31, 2026, with 76,170,292 votes “For.” A non-binding advisory vote on named executive officer compensation was also approved, receiving 71,166,204 votes “For.”
PTC Therapeutics, Inc. reported first quarter 2026 total revenue of $272.6 million, including $225.6 million in net product revenue and $46.8 million in royalty revenue. Management said this strong start supports raising full-year 2026 product revenue guidance.
Global Sephience sepiapterin revenue reached $125 million in the quarter, reflecting 36% quarter-over-quarter growth. The company reported a small net loss of $2.8 million, or $(0.03) per basic and diluted share, compared with a large prior-year profit that included substantial collaboration revenue.
PTC highlighted positive topline results from a 24‑month interim analysis of the PIVOT‑HD votoplam extension study and continued work on the global Phase 3 INVEST‑HD study. An open-label vatiquinone registration study is planned to start in Q3 2026. Cash, cash equivalents and marketable securities were $1.89 billion as of March 31, 2026.
PTC Therapeutics reported positive 24‑month interim results from its PIVOT‑HD long‑term extension study of votoplam in Huntington’s disease. Stage 2 patients showed dose‑dependent slowing of disease progression on the cUHDRS scale versus a matched natural history cohort, with 52% slowing at the 10 mg dose and 28% at 5 mg.
Safety after 24 months remained consistent with the previously favorable profile, with no treatment‑related increases in neurofilament light chain and mean levels staying below baseline, unlike typical HD progression. Novartis has initiated the global placebo‑controlled Phase 3 INVEST‑HD study of votoplam in approximately 770 early‑stage HD patients, further advancing the program.
PTC Therapeutics has appointed veteran biotechnology investment banker Jessica Chutter to its board of directors, effective March 24, 2026, filling a vacancy. She will serve as a Class I director with a term ending at the 2026 annual meeting of stockholders.
Chutter recently retired from Morgan Stanley after more than 40 years in healthcare investment banking and has advised on about $80 billion of capital raising and $85 billion of strategic transactions. As compensation, she will receive a $50,000 annual cash retainer plus initial and pro-rated 2026 equity grants totaling 6,371 stock options and 7,333 restricted stock units, vesting over multi-year schedules.
PTC Therapeutics reported strong fourth quarter and full-year 2025 results and provided 2026 guidance. Full-year 2025 product and royalty revenue was $831M, exceeding guidance, helped by Sephience, which generated $92M in fourth-quarter revenue and $111M for 2025 after its 2H 2025 launch.
For 2025, total revenues were $1,730,655,000, more than doubling from $806,780,000 in 2024, based on figures presented in thousands. Net income attributable to common stockholders was $682.6M, reversing a $363.3M loss in 2024, driven largely by $998.4M of collaboration and license revenue and despite a $98.6M negative adjustment to Translarna France sales allowances.
At December 31, 2025, cash, cash equivalents and marketable securities were $1.95B, total debt was $286.6M, and total liability for sale of future royalties was $2.31B, with stockholders’ deficit at $205.3M. For full-year 2026, projected GAAP R&D and SG&A expense is $775–$815M, and projected non-GAAP R&D and SG&A is $680–$720M after excluding $95M of expected stock-based compensation.
PTC Therapeutics has withdrawn its New Drug Application resubmission for Translarna (ataluren) as a treatment for nonsense mutation Duchenne muscular dystrophy after U.S. FDA feedback on the review. FDA indicated the data are unlikely to meet its standard of substantial evidence of effectiveness needed for approval.
CEO Matthew B. Klein said the company is disappointed that FDA approval for Translarna in the U.S. “cannot be achieved” despite more than two decades of development work. The update underscores the challenges of securing U.S. approval for this rare disease therapy, even as PTC continues broader work on treatments for genetic disorders.
PTC Therapeutics released preliminary, unaudited 2025 figures and issued 2026 guidance. For the year ending December 31, 2025, it expects to report total net product and royalty revenue of approximately $823.4 million, including total net product revenue of about $587.8 million and net product revenue for Sephience™ (sepiapterin) of roughly $112.1 million. Final results will follow completion of the year-end audit.
For 2026, the company anticipates full-year total product revenues of $700 million to $800 million, excluding Evrysdi® royalty and collaboration revenue. It projects GAAP R&D and SG&A expenses of $775 million to $815 million and non-GAAP R&D and SG&A expenses of $680 million to $720 million, excluding an estimated $95 million of non-cash stock-based compensation. PTC plans to discuss these figures and 2026 milestones at the J.P. Morgan Healthcare Conference and then enter an investor quiet period.
PTC Therapeutics filed an 8-K announcing financial results for the quarter ended September 30, 2025. The full details are provided in a press release furnished as Exhibit 99.1.
The company will host a conference call on November 4, 2025 at 4:30 p.m. Eastern to review the results and other updates. Items 2.02 and 7.01, and Exhibit 99.1, are furnished and not deemed filed under Section 18 of the Exchange Act.
PTC Therapeutics, Inc. reported that it received a Complete Response Letter from the U.S. Food and Drug Administration regarding its New Drug Application for vatiquinone. This application sought approval of vatiquinone to treat children and adults living with Friedreich’s ataxia, a rare neurodegenerative disease. The company disclosed this development through a press release furnished as an exhibit to this current report.
The Complete Response Letter means the FDA has completed its review of the application but did not approve vatiquinone in its current form, and additional steps would be required before any potential approval. No financial results or updated guidance are discussed in this report.
On 5 Aug 2025, PTC Therapeutics (PTCT) entered a Rights Satisfaction Agreement with holders of roughly 90% of former Censa Pharmaceuticals equity. The deal cancels the royalty-style Net Sales Payments—8% on annual sepiapterin sales ≤ $250 M, 10% on sales > $250 M ≤ $500 M, and 12% on sales > $500 M—originally embedded in the 2020 Censa merger.
In exchange, PTC paid an up-front cash consideration of ≈ $225 M at closing and agreed to up to five additional milestone payments of ≈ $90 M each (aggregate ≈ $450 M) tied to cumulative global sepiapterin net-sales thresholds of $3 B, $5 B, $7 B, $9 B and $11 B achieved within rolling periods of 3–11 consecutive years. Any former Censa holder that later joins the agreement will receive its pro-rata share of the up-front and any future milestones.
The arrangement only affects the specified Net Sales Payments; all other contingent obligations under the original merger agreement remain intact. The agreement contained customary reps, warranties and covenants and closed simultaneously with signing.