On May 6, 2025 PTC Therapeutics, Inc., (NASDAQ: PTCT) reported a corporate update and financial results for the first quarter ended March 31, 2025 (Press release, PTC Therapeutics, MAY 6, 2025, View Source [SID1234652592]).
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"Following a year of outstanding execution across every part of the Company, we have built on this positive momentum with solid revenue performance in the first quarter, allowing us to narrow our full-year revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "Our strong cash balance supports all of our planned commercial and R&D activities and provides the ability to reach cashflow breakeven without raising additional capital. The positive CHMP opinion for Sephience kickstarts our anticipated global launch for what we see as a significant revenue opportunity."
Key Corporate Updates:
First quarter 2025 total net product and royalty revenue of $190 million.
First quarter 2025 revenue for the DMD franchise of $134 million, including net product revenue for Translarna of $86 million and for Emflaza of $48 million.
Key Clinical and Regulatory Milestones:
Sephience
Positive CHMP opinion on Sephience MAA for adult and pediatric PKU patients received on April 25, 2025; the opinion includes a broad label inclusive of all ages and disease severities. PTC expects the EC to adopt the opinion in approximately two months
NDA currently under review by the FDA, with a target regulatory action date of July 29, 2025
Japan regulatory submission under review and a decision is expected in Q4 2025
Vatiquinone
NDA for pediatric and adult patients with Friedreich’s ataxia accepted and granted Priority Review by the FDA, with a target regulatory action date of August 19, 2025
Translarna
NDA currently under review by the FDA
PTC518
Phase 2 PIVOT-HD study results announced on May 5, 2025:
Met primary endpoint of dose-dependent blood HTT lowering at Week 12
Dose-dependent trends of clinical benefit in Stage 2 patients at Month 12
Signals of dose-dependent clinical benefit relative to matched natural history cohort and dose-dependent lowering of NfL in Stage 2 subjects at Month 24
Continued favorable safety and tolerability with no treatment-related NfL spikes
Plans to complete additional analyses and discuss next development and regulatory steps, including potential for accelerated approval
First Quarter 2025 Financial Highlights:
Total net product and royalty revenue was $189.9 million for the first quarter of 2025, compared to $208.8 million for the first quarter of 2024.
Total revenue includes net product revenue across the commercial portfolio of $153.4 million for the first quarter of 2025, compared to $177.6 million for the first quarter of 2024. Total revenue also includes royalty, collaboration and license, and manufacturing revenue of $1,022.7 million for the first quarter of 2025, compared to $32.5 million for the first quarter of 2024. The first quarter of 2025 collaboration and license revenue includes $986.2 million, related to the PTC518 license and collaboration agreement with Novartis, which closed in January 2025.
Translarna net product revenues were $86.2 million for the first quarter of 2025, compared to $103.6 million for the first quarter of 2024.
Emflaza net product revenues were $47.8 million for the first quarter of 2025, compared to $57.5 million for the first quarter of 2024.
Roche reported Evrysdi full year 2025 sales of approximately 420 CHF million, resulting in royalty revenue of $36.4 million to PTC for first quarter 2025, as compared to $31.2 million for first quarter 2024.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $109.0 million for the first quarter of 2025, compared to $116.1 million for the first quarter of 2024.
Non-GAAP R&D expenses were $100.3 million for the first quarter of 2025, excluding $8.7 million in non-cash, stock-based compensation expense, compared to $107.2 million for the first quarter of 2024, excluding $9.0 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $81.0 million for the first quarter of 2025, compared to $73.3 million for the first quarter of 2024.
Non-GAAP SG&A expenses were $71.6 million for the first quarter of 2025, excluding $9.4 million in non-cash, stock-based compensation expense, compared to $63.9 million for the first quarter of 2024, excluding $9.4 million in non-cash, stock-based compensation expense.
Net income was $866.6 million for the first quarter of 2025, compared to net loss of $91.6 million for the first quarter of 2024.
Cash, cash equivalents, and marketable securities were $2,027.2 million as of March 31, 2025, compared to $1,139.7 million as of December 31, 2024.
Shares issued and outstanding as of March 31, 2025 were 79,225,276.
PTC Updates Full-Year 2025 Financial Guidance:
PTC now anticipates full-year 2025 revenue to be between $650 million and $800 million, which includes in-line products, potential new product launches, and royalty revenue from Evrysdi.
PTC anticipates full-year 2025 GAAP R&D and SG&A expense to be between $805 and $835 million.
PTC anticipates full-year 2025 non-GAAP R&D and SG&A expense to be between $730 and $760 million, excluding estimated non-cash, stock-based compensation expense of $75 million.