PTC Therapeutics Inc

PTC Therapeutics has completed its previously announced agreement with Sangamo Therapeutics to acquire ST-920, a BLA-stage, one-time administered AAV gene therapy for Fabry disease. A rolling BLA submission to the FDA for accelerated approval of ST-920 is expected to be completed in the fourth quarter of 2026. Chief Executive Officer Matthew B. Klein said the company looks forward to completing the submission and potentially bringing a one-time administered, safe and effective durable disease treatment to the Fabry community. ST-920, also known as isaralgagene civaparvovec, has received Orphan Drug, Fast Track, and RMAT designations from the FDA, along with Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and Innovative Licensing and Access Pathway from the U.K. Medicines and Healthcare products Regulatory Agency. In clinical studies, the therapy enabled long-term production of the deficient alpha-galactosidase A enzyme and significant reduction in globotriaosylceramide levels.

PTC Therapeutics has agreed to acquire ST-920, a BLA-stage one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics in a $111 million upfront deal plus milestone payments. ST-920 is designed to deliver a functional copy of the GLA gene using an adeno-associated viral vector, and in the Phase 1/2 STAAR study patients showed sustained enzyme activity for up to 4.5 years alongside improvements in renal function, cardiac outcomes, and quality of life. The BLA submission for accelerated approval is based on the intermediate clinical endpoint of annualized eGFR at Week 52, with nonclinical and clinical modules already submitted and the CMC package expected in Q4 2026, targeting a 2027 launch. The therapy has FDA RMAT, Orphan Drug and Fast Track designations, and CEO Matthew B. Klein said the acquisition advances the company's strategy of leveraging its rare disease commercial infrastructure to accelerate near-term revenue growth. The acquisition is expected to close in Q3 2026, strengthening PTC's pipeline in rare genetic disorders.

PTC Therapeutics reported record quarterly total revenue of $361 million for the second quarter of 2026, driven by strong product sales and royalty income, and raised its full-year 2026 product revenue guidance to between $850 million and $950 million. Product revenue reached $239 million, more than doubling from $118 million in the same quarter last year, while total revenue guidance for the year was increased to a range of $1.18 billion to $1.28 billion. The company's phenylketonuria drug Sephience generated $151 million in global net product revenue, a 21 percent increase from the previous quarter, with 1,647 commercial patients on therapy worldwide as of June 30, 2026. The Duchenne muscular dystrophy franchise contributed $67 million, Translarna net product revenue was $42 million including a Russian government purchase order, Emflaza net product revenue was $25 million, and Evrysdi royalty revenue was $71 million based on Roche's global revenue of approximately $628 million. PTC Therapeutics ended the quarter with $2.23 billion in cash, equivalents, and marketable securities, and management expressed confidence in the potential for cash flow breakeven during 2026.

PTC Therapeutics reported second-quarter earnings of $0.92 per share, beating the Zacks Consensus Estimate of a loss of $0.17 per share by a surprise of 641.18%. Revenue came in at $360.52 million, surpassing the consensus estimate by 22.24% and up from $178.88 million a year ago. The company has topped consensus EPS estimates three times over the last four quarters. Shares have lost about 1.9% year to date, underperforming the S&P 500's gain of 6.9%. The current Zacks Rank for the stock is #3 (Hold).

PTC Therapeutics is gaining momentum behind its newly launched PKU therapy Sephience, which generated $125 million in first-quarter 2026 product revenues, a 36% sequential increase. The drug is now approved in the U.S., Europe, Japan and other markets, with management expecting availability in nearly 30 countries by year-end. As of March 31, 2026, 1,244 patients were on therapy worldwide, and the U.S. was adding roughly 140 new patient start forms per month. Beyond Sephience, PTC's commercial portfolio includes the DMD franchise Emflaza and Translarna, the gene therapy Upstaza/Kebilidi, and royalty revenues from Roche's Evrysdi. The pipeline features votoplam for Huntington's disease, which reported positive phase II extension data and triggered a $50 million milestone payment from Novartis upon phase III initiation, and vatiquinone for Friedreich's ataxia, with a registration-enabling study planned for the third quarter of 2026. PTC shares have risen 17.9% year to date, outpacing the industry's 6.3% gain, and the stock carries a Zacks Rank #1 (Strong Buy).

The biotech sector is quietly emerging as a new market leader while the AI-driven semiconductor rally shows signs of exhaustion, according to Zacks Investment Research. The Health Care Sector ETF XLV hit record highs this month, and the Biotech ETF XBI is approaching levels not seen since 2021, fueled by a stronger drug development cycle and defensive rotation. Among standout names, Eli Lilly remains the clear leader, while mid-cap players like Exelixis, PTC Therapeutics, Fortrea Holdings, and Illumina are gaining traction. PTC Therapeutics surged roughly 75% since its drug Sephience was approved in July 2025, with first-quarter revenue reaching $273 million, and was upgraded to a Zacks Rank #1 (Strong Buy) on July 8. Fortrea Holdings, a contract research organization, rebounded from around $4 to the mid-teens on backlog growth and cost cuts, and was added to the Zacks Rank #1 list on July 7. Exelixis offers a blend of profitability and growth at roughly 16x forward earnings, while Illumina, the genomics leader, has recovered about 46% year to date after an 80% tumble.