PTC Therapeutics Swings to Profit as Sephience Sales Surge
PTC Therapeutics posted $361 million in second-quarter revenue, more than double the year-earlier total, and raised its full-year product-revenue guidance for the second consecutive quarter.
PTC Therapeutics (PTCT) reported second-quarter revenue of $361 million, more than double the $178.9 million posted a year earlier and up from $273 million in the first quarter. The rare-disease drugmaker also swung to net income of $83.5 million from a net loss of $64.8 million in the second quarter of 2024, aided by revenue growth and lower research and administrative spending.
The turnaround marks a reversal from late last year, when total revenue fell year-over-year in the fourth quarter of 2023 as a large one-time Novartis collaboration payment tied to a January 2024 licensing deal rolled off. That gap has since been filled by organic product-revenue growth, pushing the year-over-year comparison from a decline to more than a doubling by the second quarter.
Sephience, PTC's treatment for phenylketonuria, remained the primary growth driver, with revenue climbing to $151.3 million, up 21% from $124.6 million in the first quarter. That sequential pace has slowed from the 36% jump the drug posted between the fourth quarter of 2023 and the first quarter of 2024, even as the underlying patient base kept expanding — the number of patients on commercial Sephience therapy worldwide rose to 1,647 as of June 30, from 1,244 three months earlier and 946 at the end of 2023.
Total net product revenue reached $238.8 million, more than double the $118.3 million recorded a year earlier and up from $225.6 million in the first quarter, when year-over-year growth ran at 47%. The acceleration in that growth rate came even as two older products kept losing ground: Translarna revenue fell to $42.2 million from $59.5 million a year earlier, and Emflaza revenue dropped to $24.6 million from $36.4 million amid continued generic competition. Evrysdi royalty income, by contrast, kept climbing steadily, rising to $71.1 million from $57.6 million a year earlier.
The quarter's results also included a new $50 million milestone payment from Novartis, recognized as collaboration revenue after the first patient was dosed in the Phase 3 INVEST-HD trial. PTC had flagged the payment as pending in its first-quarter release; it converted into recognized revenue this quarter.
PTC raised its full-year 2024 revenue guidance to a range of $1.18 billion to $1.28 billion, with product-revenue guidance lifted to $850 million to $950 million from a prior range of $750 million to $850 million — the second straight quarterly increase to that outlook. GAAP research and development expenses declined to $99.2 million from $113.0 million a year earlier, continuing a downward trend from $100.9 million in the first quarter, while selling, general and administrative costs fell to $80.6 million from $85.3 million.
PTC ended the quarter with $2.23 billion in cash, cash equivalents and marketable securities, up from $1.95 billion at the end of 2023, after issuing $550 million of zero-coupon senior convertible notes in June. Proceeds from the offering were used in part to repurchase existing 1.5% notes due September 2026, leaving total debt at $591.0 million, more than double the $286.6 million on the books at year-end.