PTC Therapeutics reported fourth‑quarter 2025 revenue of $164.7 million, a 23.5% decline from the $213.2 million earned in Q3 2025 and a 45.9% shortfall versus the consensus estimate of $304.7 million. Net loss for the quarter widened to $134.9 million, and earnings per share fell to $‑1.67, missing the consensus estimate of $‑0.21 by $‑1.46, a miss of 700 %. The miss reflects a combination of lower product sales, particularly in the legacy Duchenne muscular dystrophy (DMD) franchise, and higher operating expenses associated with the ramp‑up of the new Sephience therapy.
Sephience, launched in the second half of 2025, contributed $92 million to Q4 revenue and $111 million for the full year, underscoring its role as the company’s primary growth engine. In contrast, revenue from the legacy DMD franchise fell sharply as sales of Translarna and Emflaza declined, contributing to the overall revenue shortfall and the widened net loss.
Cash and cash equivalents stood at $1.95 billion as of December 31 2025, bolstered by the $240 million upfront payment and up to $60 million in milestones from the sale of the remaining Evrysdi royalty to Royalty Pharma in December 2025. The strong liquidity position supports ongoing operations and future growth initiatives.
For 2026, PTC reaffirmed revenue guidance of $700 million to $800 million, a range that falls short of analyst consensus estimates of $959.6 million to $990.3 million. Management indicated that the guidance reflects a cautious outlook amid regulatory uncertainty surrounding the Translarna NDA withdrawal and the need to invest in the next‑generation votoplam program for Huntington’s disease.
Investors reacted negatively to the earnings miss and conservative guidance, reflecting concerns about the company’s ability to meet analyst expectations and the impact of the legacy franchise decline.
"We delivered another strong quarter and finish to 2025, building on the successful global launch of Sephience," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "With our robust commercial engine, innovative R&D programs, and strong financial position, we look forward to continued success as we approach cash flow breakeven."
The company withdrew the New Drug Application resubmission for Translarna, adding regulatory uncertainty to the legacy DMD portfolio. In addition, the Vatiquinone program faces a required additional study before the next NDA resubmission, further complicating the company’s pipeline timeline.
PTC is advancing its votoplam program, with a Phase 3 trial slated to begin in the first half of 2026. The company’s focus on scaling Sephience and investing in the splicing‑platform pipeline signals a strategic shift toward high‑margin, high‑growth therapies.
Overall, the earnings release highlights a mixed picture: a strong launch of Sephience and solid cash reserves counterbalance a significant revenue miss, EPS shortfall, and a conservative 2026 outlook. The company’s ability to navigate the decline of its legacy franchise while accelerating new‑product development will be critical to its long‑term trajectory.
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