PTC Therapeutics, Inc.

PTC Therapeutics Provides Corporate Update and Reports Third Quarter 2026 Financial Results

PTC Therapeutics reported Q3 2026 total revenue of $357.8 million, up roughly 70% year-over-year, with net product revenue more than doubling to $275.9 million from $131.0 million. The Sephience launch drove the quarter with $181.9 million in revenue (up 20% sequentially) and over 2,000 active patients worldwide; Evrysdi royalties added $81.7 million. Full-year 2026 guidance was raised: total revenue to $1.29-$1.32 billion and product revenue to $960-$990 million from $850-$950 million. GAAP net loss was $51.7 million (EPS -$0.62), driven by a $111.0 million acquired IPR&D charge from purchasing the ST-920 Fabry gene therapy from Sangamo, with a BLA submission expected in Q4 2026 and potential 2027 launch. Cash and marketable securities stood at $2.13 billion, and management said the company is on track to reach cash flow breakeven this year.

•3d ago

PTC Completes Acquisition of ST-920 Fabry Disease Gene Therapy

PTC Therapeutics completed its previously announced acquisition of ST-920 (isaralgagene civaparvovec) from Sangamo Therapeutics — a BLA-stage, one-time administered AAV gene therapy for Fabry disease. A rolling BLA submission to the FDA for accelerated approval is expected to be completed in Q4 2026. ST-920 carries FDA Orphan Drug, Fast Track, and RMAT designations, plus EMA Orphan/PRIME and UK ILAP designations. No deal terms are disclosed in this release. Clinical data showed durable alpha-galactosidase A production and significant Gb3 reduction, positioning ST-920 as a potential one-time alternative to chronic enzyme replacement therapy in Fabry disease.

•1mo ago

PTC Therapeutics Announces New Sephience™ (sepiapterin) Data Reinforcing Broad Patient Benefits Including Sustained Metabolic Control and Diet Liberalization as well as Consistent Favorable Safety Profile

PTC Therapeutics announced that 17 abstracts featuring Sephience (sepiapterin) data will be presented at the SSIEM Annual Symposium, demonstrating sustained metabolic control and significant diet liberalization for PKU patients. New analyses from the AMPLIPHY study indicate a 100% greater reduction in blood Phe for participants switching from sapropterin to Sephience, with meaningful reductions observed in high-baseline patients within 14 days. Real-world evidence supports the drug's use in adolescents by reducing dietary burden while maintaining metabolic control, with a consistent safety profile across all patient subgroups.

•2mo ago

PTC to Expand Rare Disease Portfolio with Acquisition of BLA-Stage ST-920 Fabry Disease Program

PTC Therapeutics was selected as the winning bidder to acquire ST-920, a BLA-stage gene therapy for Fabry disease, from Sangamo Therapeutics in a competitive bankruptcy auction. The transaction terms include $111 million upfront and up to $100 million in contingent milestone payments, with a rolling BLA submission for accelerated approval expected to be completed in Q4 2026. PTC plans to leverage its existing commercial infrastructure to launch the therapy in 2027, stating the acquisition will not impact its objective of reaching cash flow break-even in 2026.

•2mo ago

PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results

PTC Therapeutics delivered a strong Q2 with total revenue of $361 million and product revenue jumping over 100% year-over-year to $239 million, driving a swing to net income of $83.5 million. Management raised full-year 2026 total revenue guidance to $1.18 billion to $1.28 billion and increased product revenue expectations to $850 million to $950 million, citing continued momentum for Sephience. The balance sheet remains robust with $2.23 billion in cash, bolstered by a $50 million milestone from Novartis and a $550 million convertible note offering at a 40% premium conversion price.

•3mo ago

Hamilton Lane Board Elects Michael Schmertzler as New Independent Director

Hamilton Lane elected Michael Schmertzler as a new independent director to its Board, effective July 22, 2026, bringing the total number of independent directors to five. Schmertzler brings decades of experience in investment and corporate finance, including former roles at Credit Suisse First Boston and Morgan Stanley, and currently serves as Chairman of PTC Therapeutics. The firm currently manages approximately $1 trillion in assets under management and supervision, employing 785 professionals globally.

•3mo ago

PTC Therapeutics Announces Pricing of Convertible Notes Offering to Refinance 2026 Convertible Notes

PTC Therapeutics priced $500 million of 0% convertible senior notes due 2031 to refinance its 1.5% convertible notes maturing in 2026. The notes carry a conversion price of $107.48, a 40% premium to the June 15 closing price. The company expects net proceeds of approximately $486.8 million and plans to use about $329 million to repurchase the near-term 2026 notes. This transaction extends the debt maturity by five years and removes the 1.5% interest burden, strengthening the balance sheet.

•4mo ago

PTC Therapeutics Provides Corporate Update and Reports First Quarter 2026 Financial Results

PTC reported Q1 2026 total revenue of $272.6 million, with product revenue jumping 47% year-over-year to $225.6 million, driven by the global launch of Sephience. The company raised full-year 2026 product revenue guidance to $750-$850 million, citing strong momentum where Sephience revenue grew 36% quarter-over-quarter to $125 million. Pipeline progress included positive topline results from the PIVOT-HD extension study for votoplam and a $50 million milestone payment from partner Novartis following the first patient dosed in the Phase 3 INVEST-HD study. Management plans to initiate an open-label registration study for vatiquinone in Q3 2026 based on FDA feedback to support the NDA resubmission for Friedreich's ataxia.

•5mo ago

PTC Therapeutics Reports Positive Topline Results from Month 24 Interim Analysis of PIVOT-HD Extension Study of Votoplam

PTC Therapeutics reported positive topline results from the 24-month interim analysis of the PIVOT-HD extension study, showing a 52% slowing of disease progression at the 10 mg dose compared to a natural history cohort. Safety data remained favorable with no treatment-related neurofilament light chain protein increases observed at the 24-month mark for either dose level. Partner Novartis initiated the global Phase 3 INVEST-HD study, which plans to enroll approximately 770 individuals with early-stage Huntington's disease randomized 3:2 to receive Votoplam 10 mg or placebo.

•6mo ago