On February 19, 2026 PTC Therapeutics, Inc., (NASDAQ: PTCT) reported a corporate update and financial results for the fourth quarter and full year ending December 31, 2025.

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"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."

Key Corporate Highlights

Full-year 2025 product and royalty revenue of $831 million, exceeding guidance
Global launch of Sephience off to strong start
Q4 2025 revenue of $92 million, including $81 million revenue in the US and $11 million revenue ex-US
Sephience total net revenue of $111 million in 2025 since launch
946 total patients on commercial therapy worldwide as of December 31, 2025
1,134 patient start forms received in the US as of December 31, 2025
Approval in Japan in December 2025; approval in Brazil in February 2026
Sephience global footprint expected to increase to 20 to 30 countries by end of 2026
In December 2025, PTC sold the remainder of its Evrysdi (risdiplam) royalty to Royalty Pharma for $240 million upfront and up to $60 million in sales-based milestones; PTC maintains the right to receive a $150 million milestone from Roche based on single-year Evrysdi sales of $2.5 billion
End-of-Phase 2 meeting with FDA held in Q4 2025 to discuss the votoplam Huntington’s disease (HD) program
Alignment reached on design of global Phase 3 trial, INVEST-HD, which is planned to initiate in 1H 2026
FDA confirmed openness for potential Accelerated Approval pathway given significant unmet need
Type C meeting with FDA held in December 2025 to discuss the vatiquinone Friedreich’s ataxia program; FDA indicated that an additional study would be necessary to support NDA resubmission and meeting minutes stated that this could be an open-label study with a natural history control group
Fourth Quarter and Full Year 2025 Financial Highlights

Total revenues were $164.7 million for the fourth quarter of 2025, compared to $213.2 million for the fourth quarter of 2024. Total revenues were $1,730.7 million for full year 2025, compared to $806.8 million for full year 2024. Included in total revenues is collaboration and license revenue of $998.4 million for the full year 2025, related to the votoplam license and collaboration agreement with Novartis, which closed in January 2025.
Total net product revenues were $184.0 million for the fourth quarter of 2025, compared to $150.1 million for the fourth quarter of 2024. Total net product revenues were $586.7 million for full year 2025, compared to $582.1 million for full year 2024.
Translarna (ataluren) net product revenues were $39.0 million for the fourth quarter of 2025, compared to $89.1 million for the fourth quarter of 2024. Translarna net product revenues were $235.3 million for full year 2025, compared to $321.1 million for full year 2024.
Emflaza (deflazacort) net product revenues were $27.1 million for the fourth quarter of 2025, compared to $50.5 million for the fourth quarter of 2024. Emflaza net product revenues were $146.4 million for full year 2025, compared to $207.2 million for full year 2024.
Roche reported Evrysdi full year 2025 sales of approximately 1,757 CHF million, resulting in royalty revenue of $244.2 million to PTC for full year 2025, compared to $203.9 million to PTC for full year 2024.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $133.1 million for the fourth quarter of 2025, compared to $124.8 million for the fourth quarter of 2024. GAAP R&D expenses were $455.2 million for full year 2025, compared to $534.5 million for full year 2024.
Non-GAAP R&D expenses were $124.3 million for the fourth quarter of 2025, excluding $8.8 million in non-cash, stock-based compensation expense, compared to $116.0 million for the fourth quarter of 2024, excluding $8.8 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $419.6 million for full year 2025, excluding $35.7 million in non-cash, stock-based compensation expense, compared to $497.9 million for full year 2024, excluding $36.6 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $96.9 million for the fourth quarter of 2025, compared to $84.7 million for the fourth quarter of 2024. GAAP SG&A expenses were $347.1 million for full year 2025, compared to $300.9 million for full year 2024.
Non-GAAP SG&A expenses were $87.2 million for the fourth quarter of 2025, excluding $9.7 million in non-cash, stock-based compensation expense, compared to $76.3 million for the fourth quarter of 2024, excluding $8.4 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $308.3 million for full year 2025, excluding $38.9 million in non-cash, stock-based compensation expense, compared to $262.9 million for full year 2024, excluding $38.0 million in non-cash, stock-based compensation expense.
Net loss was $135.0 million for the fourth quarter of 2025, compared to net loss of $65.9 million for the fourth quarter of 2024. Net income was $682.6 million for full year 2025, compared to net loss of $363.3 million for full year 2024.
Cash, cash equivalents, and marketable securities were $1,945.4 million on December 31, 2025, compared to $1,139.7 million on December 31, 2024.
Shares issued and outstanding as of December 31, 2025, were 81,474,366.
Full Year 2026 Financial Guidance

Total product revenue of $700 to $800 million, representing a 19 to 36% increase from 2025, with the majority from Sephience
GAAP R&D and SG&A expense of $775 to $815 million
Non-GAAP R&D and SG&A expense of $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million

(Press release, PTC Therapeutics, FEB 19, 2026, View Source [SID1234662790])

On February 19, 2026 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported that management will participate in the following investor conferences:

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Oppenheimer 36th Annual Healthcare Life Sciences Conference. A fireside chat will take place virtually on Thursday, February 26 at 2:40 pm ET.
TD Cowen 46th Annual Health Care Conference. A fireside chat will take place on Tuesday, March 3 at 2:30 pm ET in Boston.
A live webcast of the events can be accessed by visiting the Events and Presentations section of the Company’s website: View Source The webcasts will be archived for a limited time following the presentation.

(Press release, Protara Therapeutics, FEB 19, 2026, View Source [SID1234662789])

On February 19, 2026 Olema Pharmaceuticals, Inc. ("Olema", or "Olema Oncology", Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of targeted therapies for breast cancer and beyond, reported that Sean P. Bohen, M.D., Ph.D., President and Chief Executive Officer, will present at the TD Cowen 46th Annual Health Care Conference on Tuesday, March 3, 2026 at 2:30 p.m. ET.

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A live webcast and recording of this presentation will be available, as permitted by the event host, in the Events and Presentations section of Olema’s investor relations website at ir.olema.com.

(Press release, Olema Oncology, FEB 19, 2026, View Source [SID1234662788])

On February 19, 2026 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), reported it has entered into agreements with certain holders of its existing warrants for the immediate exercise of certain outstanding warrants to purchase up to an aggregate of 2,122,652 shares of common stock of the Company at an exercise price of $3.90 per share. The issuance or resale of the shares of common stock issuable upon exercise of the outstanding warrants has been registered pursuant to effective registration statements. The aggregate gross proceeds from the exercise of the existing warrants is expected to total approximately $8.3 million, before deducting financial advisory fees.

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Roth Capital Partners is acting as the Company’s financial advisor for this transaction.

In consideration for the immediate exercise of the warrants for cash, the Company will issue new unregistered warrants to purchase shares of common stock. The new warrants will be exercisable for an aggregate of up to 6,367,956 shares of common stock, at an exercise price equal to the lesser of $3.90 per share or the lowest volume weighted average price of the Company’s common stock on any trading day during the next five trading days, will be exercisable upon shareholder approval and for a term of five years from the date of shareholder approval.

The transaction is expected to close on or about February 20, 2026, subject to satisfaction of customary closing conditions. The Company intends to use the net proceeds from the offering for working capital and general corporate purposes.

The new warrants described above were offered in a private placement pursuant to an applicable exemption from the registration requirements of the Securities Act of 1933, as amended (the "1933 Act") and, along with the shares of common stock issuable upon their exercise, have not been registered under the 1933 Act, and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission ("SEC") or an applicable exemption from such registration requirements. The Company has agreed to file a registration statement with the SEC covering the resale of the shares of common stock issuable upon exercise of the new warrants.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

(Press release, Moleculin, FEB 19, 2026, View Source [SID1234662787])

On February 19, 2026 MannKind Corporation (Nasdaq: MNKD), reported Chief Executive Officer Michael Castagna, PharmD is scheduled to present and share business updates during the Oppenheimer 36th Annual Healthcare Life Sciences Conference on Thursday, February 26, 2026, at 11:20 a.m. ET

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A link to the webcast of the presentation will be available on MannKind Corporation’s website at: View Source Recorded versions will also be available on the website for approximately 90 days following the conference.

(Press release, Mannkind, FEB 19, 2026, View Source [SID1234662786])