Apazunersen

FDA Established April 23, 2026 as PDUFA Target Date for Review of Submission Seeking Approval for GTx-104 in the Treatment of Patients with aneurysmal Subarachnoid Hemorrhage (aSAH) Phase 3 STRIVE-ON Safety Trial Data Presented at 2025 Society of Vascular and Interventional Neurology Annual Meeting Company Continues Pre-Commercial Planning in Anticipation of Potential FDA Approval of NDA Submission for GTx-104 for the Treatment of Patients with aSAH PRINCETON, N.J., Feb. 12, 2026 (GLOBE NEWSWIRE) -- Grace Therapeutics, Inc. (Nasdaq: GRCE) (Grace Therapeutics or the Company), a late-stage, biopharma company advancing GTx-104, a clinical-stage, novel, injectable formulation of nimodipine being developed for IV infusion to address significant unmet medical needs in aSAH patients, today announced the financial results and business highlights for the quarter ended December 31, 2025. “During our third quarter of fiscal 2026 we continued to execute on our clinical and corporate goals, led by our pre-commercial planning in anticipation of potential FDA approval of our New Drug Application (NDA) for GTx-104 for the treatment of aSAH,” said Prashant Kohli, CEO of Grace Therapeutics. “Our NDA is supported by a robust data package, including positive results from our STRIVE-ON trial, which provided evidence of improved clinical outcomes in aSAH patients treated with GTx-104 as well as potential medical and pharmacoeconomic benefits of GTx-104 in the treatment of aSAH. We were pleased to see these data highlighted in a late breaking presentation at the Society of Vascular and Interventional Neurology annual meeting in November 2025. Our STRIVE-ON trial results continue to be well received by researchers, practitioners and industry leaders. We believe that if our NDA for GTx-104 is approved by the FDA, our strong U.S. and international patent estate will help to maximize the long-term market value of GTx-104 and correspondingly deliver value for our shareholders. The standard of care for aSAH has not seen meaningful innovation in nearly 40 years, and we believe that if GTx-104 is approved, our STRIVE-ON trial results point to a very promising role for GTx-104 in the treatment of these patients. We look forward to continuing to engage with the FDA during their review as they work toward the PDUFA target date of April 23, 2026.” Third Quarter 2026 and Recent Corporate Highlights During our third fiscal quarter we provided grant support for the development of a Continuing Medical Education (CME) program for healthcare professionals in medical specialties related to the care of aSAH patients. Content for the CME program was independently developed by leading providers of CME content and is intended to educate Health Care Professionals on the current challenges in treating aSAH patients. On November 21, 2025, the Company presented data from its pivotal Phase 3 STRIVE-ON Safety Trial of GTx-104 in aSAH as a late breaking trial at the Society of Vascular and Interventional Neurology annual meeting. The presentation titled Safety and Tolerability of GTX-104 (Nimodipine Injection for IV Infusion) Compared with Oral Nimodipine in Patients with Aneurysmal Subarachnoid Hemorrhage: a Prospective, Randomized Trial, was presented by Thomas P. Bleck, MD, Professor, Neurology (Neurocritical Care and Epilepsy/Clinical Neurophysiology) at Northwestern University Feinberg School of Medicine. On October 23, 2025, the Company announced that it had secured approximately $4.0 million in additional funding through exercises of common warrants that were previously issued in a private placement that the Company closed in September 2023. The Company issued 1,345,464 new shares of common stock at an exercise price of $3.003 per share. The remaining 1,190,927 common warrants issued in the 2023 private placement expired on October 21, 2025. Third Quarter 2026 Financial Results The Company reported a net loss of approximately $2.3 million, or $0.14 per share, for the three months ended December 31, 2025, a decrease of approximately $1.8 million from the net loss of $4.2 million, or $0.36 per share, for the three months ended December 31, 2024. The decrease in net loss was primarily due to a $1.7 million decrease in research and development expenses and a $1.1 million decrease in the change of fair value of derivative warrant liabilities, partially offset by increases in general administrative expenses of $0.5 million and a decrease in income tax benefit of $0.6 million. Total research and development expenses for the three months ended December 31, 2025, were $0.5 million, compared to $2.2 million for the three months ended December 31, 2024. The decrease of $1.7 million was primarily due to a $1.8 million decrease in research activities mainly due to completion of our GTx-104 pivotal Phase 3 STRIVE-ON safety clinical trial, partially offset by a $0.1 million increase in salaries and benefits due to merit increases. General and administrative expenses were $2.0 million for the three months ended December 31, 2025, an increase of $0.5 million from $1.5 million for the three months ended December 31, 2024. The increase was primarily a result of an increase in professional fees and other general and administrative expenses primarily due to costs for GTx-104 pre-commercial planning. Cash Runway As of December 31, 2025, cash and cash equivalents were $18.7 million, a net decrease of $3.4 million compared to cash and cash equivalents of $22.1 million at March 31, 2025. The private placement the Company completed in February 2025 included common warrants exercisable for shares of common stock (or pre-funded warrants in lieu thereof) at an exercise price of $3.395 per share. Each common warrant is immediately exercisable and will expire on the earlier of (i) the 60th day after the date the FDA approves the NDA for GTx-104 and (ii) September 25, 2028. Potential gross proceeds from the exercise of the February 2025 common warrants are $15.0 million. The Company plans to use its current cash and cash equivalents to further the regulatory review process for GTx-104, pre-commercial planning, commercial team buildout, and product launch if GTx-104 is approved, working capital and other general corporate purposes. The Company believes its existing cash and cash equivalents will be sufficient to sustain planned operations through at least 12 months from the date of this press release. About the STRIVE-ON Trial The STRIVE-ON trial ( NCT05995405 ) was a prospective, randomized open-label trial of GTx-104 compared with oral nimodipine in patients hospitalized with aSAH. 50 patients were administered GTx-104 and 52 patients received oral nimodipine. The primary endpoint was the number of patients with at least one episode of clinically significant hypotension reasonably considered to be caused by the drug, and additional secondary endpoints included safety, clinical, and pharmacoeconomic outcomes. The trial met its primary endpoint, with patients receiving GTx-104 observed to have a 19% reduction in at least one incidence of clinically significant hypotension compared to oral nimodipine (28% versus 35%). Other measures also favored GTx-104 or were comparable between GTx-104 and oral nimodipine, including: 54% patients on GTx-104 had relative dose intensity (RDI) of 95% or higher compared to only 8% on oral nimodipine, and 29% more patients on GTx-104 than on oral nimodipine had favorable functional outcomes at 90 days. In addition, there were fewer intensive care unit (ICU) readmissions, ICU days, and ventilator days for patients receiving GTx-104 versus oral nimodipine. Adverse events were comparable between the two arms and no new safety issues were identified with patients receiving GTx-104. All deaths in both arms of the trial were due to severity of the patient’s underlying disease. There were eight deaths on the GTx-104 arm compared to four deaths on the oral nimodipine arm. The survival status of one patient on the oral nimodipine arm was unknown. No deaths were determined to be related to GTx-104 or oral nimodipine. About aneurysmal Subarachnoid Hemorrhage (aSAH) aSAH is bleeding over the surface of the brain in the subarachnoid space between the brain and the skull, which contains blood vessels that supply the brain. A primary cause of such bleeding is the rupture of an aneurysm in the brain. The result is aSAH, a relatively uncommon type of stroke that accounts for about 5% of all strokes and an estimated 42,500 U.S. hospital treated patients. About the Grace Therapeutics Asset Portfolio GTx-104 is a clinical stage, novel, injectable formulation of nimodipine being developed for IV infusion in aSAH patients to address significant unmet medical needs. The unique nanoparticle technology of GTx-104 facilitates aqueous formulation of insoluble nimodipine for a standard peripheral IV infusion. GTx-104 provides a convenient IV delivery of nimodipine in the Intensive Care Unit potentially eliminating the need for nasogastric tube administration in unconscious or dysphagic patients. Intravenous delivery of GTx-104 also has the potential to lower food effects, drug-to-drug interactions, and eliminate potential dosing errors. Further, GTx-104 has the potential to better manage hypotension in aSAH patients. GTx-104 has been administered in over 200 patients and healthy volunteers and was well tolerated with significantly lower inter- and intra-subject pharmacokinetic variability compared to oral nimodipine. GTx-102 is a novel, concentrated oral-mucosal spray of betamethasone intended to improve neurological symptoms of Ataxia-Telangiectasia (A-T), for which there are currently no FDA-approved therapies. GTx-102 is a stable, concentrated oral spray formulation comprised of the gluco-corticosteroid betamethasone that, together with other excipients can be sprayed conveniently over the tongue of the A-T patient and is rapidly absorbed. The Company received written responses to its End of Phase 1 meeting in GTx-102 where the FDA made recommendations on the path toward an NDA. The FDA provided guidance on the design of a single pivotal efficacy and safety trial, including the neurological assessment scale for the primary endpoint, that could, with appropriate confirmatory evidence, support an NDA. The further development of GTx-102 has been deprioritized in favor of focusing on development of GTx-104. It is also possible that the Company may license or sell GTx-102. GTx-101 is a non-narcotic, topical bio-adhesive film-forming bupivacaine spray designed to ease the symptoms of patients suffering with postherpetic neuralgia (PHN). GTx-101 is administered via a metered-dose of bupivacaine spray and forms a thin bio-adhesive topical film on the surface of the patient’s skin, which enables a touch-free, non-greasy application. It also comes in convenient, portable 30 ml plastic bottles. Unlike oral gabapentin and lidocaine patches, which are used for the treatment of PHN, the Company believes that the biphasic delivery mechanism of GTx-101 has the potential for rapid onset of action and continuous pain relief for up to eight hours. No skin sensitivity was reported in a Phase 1 trial. The further development of GTx-101 has been deprioritized in favor of focusing on development of GTx-104. It is also possible that the Company may license or sell GTx-101. About Grace Therapeutics Grace Therapeutics, Inc. (Grace Therapeutics or the Company) is a late-stage biopharma company with drug candidates addressing rare and orphan diseases. Grace Therapeutics’ novel drug delivery technologies have the potential to improve the performance of currently marketed drugs by achieving faster onset of action, enhanced efficacy, reduced side effects, and more convenient drug delivery. Grace Therapeutic’s lead clinical assets have each been granted Orphan Drug Designation by the FDA, which provides seven years of marketing exclusivity post-launch in the United States if certain conditions are met at NDA approval, and additional intellectual property protection with over 40 granted and pending patents. Grace Therapeutics’ lead clinical asset, GTx-104, is an IV infusion targeting aneurysmal Subarachnoid Hemorrhage (aSAH), a rare and life-threatening medical emergency in which bleeding occurs over the surface of the brain in the subarachnoid space between the brain and skull. For more information, please visit: Forward-Looking Statements Statements in this press release that are not statements of historical or current fact constitute “forward-looking statements” within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and “forward-looking information” within the meaning of Canadian securities laws (collectively, “forward-looking statements”). Such forward-looking statements involve known and unknown risks, uncertainties, and other factors that could cause the actual results of Grace Therapeutics to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. In addition to statements which explicitly describe such risks and uncertainties, readers are urged to consider statements containing the terms “believes,” “belief,” “expects,” “intends,” “anticipates,” “estimates,” “potential,” “should,” “may,” “will,” “plans,” “continue,” “targeted” or other similar expressions to be uncertain and forward-looking. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. The forward-looking statements in this press release, including statements regarding, the Company’s cash runway and cash position, the future prospects of the Company’s GTx-104 drug candidate, the outcome of the Company’s NDA submission for GTx-104, GTx-104’s potential to bring enhanced treatment options to patients suffering from aSAH, GTx-104’s potential to be administered to improve the management of hypotension in patients with aSAH, the ability of GTx-104 to achieve a pharmacokinetic and safety pro to the oral form of nimodipine, GTx-104’s potential to achieve medical and pharmacoeconomic benefit, GTx-104’s commercial prospects, the future prospects of the Company’s GTx-102 drug candidate, GTx-102’s potential to provide clinical benefits to decrease symptoms associated with A-T, the timing and outcomes of a Phase 3 efficacy and safety trial for GTx-102, the timing of an NDA filing for GTx-102, the future prospects of the Company’s GTX-101 drug candidate, GTX-101’s potential to be administered to PHN patients to treat the severe nerve pain associated with the disease and any future patent and other intellectual property filings made by the Company for new developments, are based upon Grace Therapeutics’ current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including, without limitation: (i) the success and timing of regulatory submissions of the Phase 3 STRIVE-ON safety trial for GTx-104; (ii) regulatory requirements or developments and the outcome of the Company’s NDA submission for GTx-104; (iii) changes to regulatory pathways; (iv) our ability to protect our intellectual property for our drug candidates; and (v) legislative, regulatory, political and economic developments. The foregoing list of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors detailed in the “Special Note Regarding Forward-Looking Statements,” “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of the Company’s Annual Report on Form 10-K for the fiscal year ended March 31, 2025, the Company’s Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2025, the Company’s Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2025 and the Company’s Quarterly Report on Form 10-Q for the quarterly period ended December 31, 2025, to be filed with the Securities and Exchange Commission (“SEC”) and other documents that have been and will be filed by Grace Therapeutics from time to time with the SEC and Canadian securities regulators. All forward-looking statements contained in this press release speak only as of the date on which they were made. Grace Therapeutics undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by applicable securities laws. For more information, please contact: Grace Therapeutics Contact: Prashant Kohli Chief Executive Officer Tel: 609-322-1602 Email: info@gracetx.com Investor Relations: LifeSci Advisors Mike Moyer Managing Director Phone: 617-308-4306 Email: mmoyer@lifesciadvisors.com ---tables to follow--- GRACE THERAPEUTICS, INC. Condensed Consolidated Balance Sheets (Unaudited) December 31, 2025 March 31, 2025 (Expressed in thousands except share data) $ $ Assets Current assets: Cash and cash equivalents 18,672 22,133 Receivables 20 126 Prepaid expenses 519 453 Total current assets 19,211 22,712 Equipment, net 11 15 Intangible assets 41,128 41,128 Goodwill 8,138 8,138 Total assets 68,488 71,993 Liabilities and Stockholders’ equity Current liabilities: Trade and other payables 1,284 1,930 Total current liabilities 1,284 1,930 Derivative warrant liabilities - 1,141 Deferred tax liability 2,312 2,312 Total liabilities 3,596 5,383 Commitments and contingencies Stockholders’ equity: Preferred stock, $0.0001 par value per share; 10,000,000 authorized; none issued and outstanding as of December 31, 2025 and March 31, 2025 — — Common stock, $0.0001 par value per share; 100,000,000 authorized; 15,474,026 and 13,718,106 shares issued and outstanding as of December 31, 2025 and March 31, 2025, respectively 1 1 Additional paid-in capital 298,231 293,334 Accumulated other comprehensive loss (6,038 ) (6,038 ) Accumulated deficit (227,302 ) (220,687 ) Total stockholders’ equity 64,892 66,610 Total liabilities and stockholders’ equity 68,488 71,993 GRACE THERAPEUTICS, INC. Condensed Consolidated Statements of Loss and Comprehensive Loss (Unaudited) Three months ended Nine months ended December 31, 2025 December 31, 2024 December 31, 2025 December 31, 2024 (Expressed in thousands, except share and per share data) $ $ $ $ Operating expenses Research and development expenses, net of government assistance (462 ) (2,194 ) (1,985 ) (7,877 ) General and administrative expenses (1,987 ) (1,510 ) (6,082 ) (5,619 ) Loss from operating activities (2,449 ) (3,704 ) (8,067 ) (13,496 ) Foreign exchange gain (loss) 4 (16 ) 5 (11 ) Change in fair value of derivative warrant liabilities (40 ) (1,178 ) 900 578 Interest and other income, net 170 138 547 544 Total other income (loss), net 134 (1,056 ) 1,452 1,111 Loss before income tax recovery (2,315 ) (4,760 ) (6,615 ) (12,385 ) Income tax benefit — 605 — 2,181 Net loss and total comprehensive loss (2,315 ) (4,155 ) (6,615 ) (10,204 ) Basic and diluted loss per share (0.14 ) (0.36 ) (0.41 ) (0.89 ) Weighted-average number of shares outstanding 16,933,620 11,506,234 16,262,111 11,506,234

Preliminary 2025 total revenue of $672 million to $674 million, exceeding top end of guidance Preliminary cash and investments of approximately $735 million as of December 31, 2025 Anticipated 2026 catalysts include two potential approvals and pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome NOVATO, Calif., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today reported preliminary unaudited 2025 revenue results, cash and investments at year end 2025, and provided a corporate update. “In 2025, we delivered another year of significant revenue growth, with preliminary revenue exceeding the top end of guidance," said Emil Kakkis, chief executive officer and president of Ultragenyx. "The year ahead will be transformational for our company and the rare disease communities we serve, with two potential gene therapy approvals of first-ever treatments, and a pivotal data readout from our Phase 3 program in Angelman syndrome, setting the stage for our next phase of growth." Financial Update 2025 Preliminary Revenue (unaudited) Total revenue for 2025 is estimated to be $672 million to $674 million, which exceeds the top end of the guidance range, and represents approximately 20% growth versus 2024. Crysvita ® revenue for 2025 is estimated to be $480 million to $482 million, which exceeds the top end of the guidance range, and represents approximately 17% growth versus 2024. Dojolvi ® revenue for 2025 is estimated to be $95 million to $97 million, at the midpoint of the guidance range, and represents approximately 9% growth versus 2024. 2025 Ending Cash Position (unaudited) Cash and investments were approximately $735 million as of December 31, 2025. The 2025 revenues and cash position included in this release are preliminary and are therefore subject to adjustment. The preliminary revenue results are based on management’s initial analysis of operations for the year ended December 31, 2025. The Company expects to issue financial guidance for fiscal year 2026, including details on previously announced planned expense reductions, and full financial results for the fourth quarter and fiscal year 2025, in February 2026. 2026 Clinical and Regulatory Catalysts DTX401 (pariglasgene brecaparvovec) AAV gene therapy for glycogen storage disease type Ia (GSDIa): Biologics License Application (BLA) rolling submission completed in December 2025, with an anticipated PDUFA date in the third quarter of 2026. UX111 (rebisufligene etisparvovec) AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Resubmission of the BLA is on track for early in 2026 and will be followed by an up to 6-month review per FDA regulations. GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment of Angelman syndrome (AS): Data from the fully enrolled, pivotal, Phase 3 Aspire study in patients with a genetically confirmed diagnosis of UBE3A deletion is expected in the second half of 2026. The Phase 2/3 Aurora study is also underway in other genotypes and ages, with first patient dosed in October 2025. UX701 (rivunatpagene miziparvovec) AAV gene therapy for Wilson disease: Enrollment is complete for the fourth cohort in the ongoing, dose-finding stage of the pivotal Cyprus2+ study. Data are expected in the first half of 2026. Data from the Phase 3 Orbit and Cosmic studies of UX143 (setrusumab) in osteogenesis imperfecta, including data on bone mineral density, vertebral fractures, and patient reported outcomes on pain and physical function, will be presented at the J.P. Morgan Healthcare Conference. The company presentation is scheduled for Monday, January 12, 2026 at 10:30 AM PT. The live and archived webcast of the presentation will be accessible from the company’s website at . About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: . Forward-Looking Statements and Use of Digital Media Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, the components and timing of regulatory submissions and the timing of results from ongoing clinical studies are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the company’s clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercialization of Crysvita in certain major markets, including the U.S. and Canada, and for our commercial supply of Crysvita in those markets, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, , smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, our ability to successfully manage the expansion of our company, competition from other therapies or products, regulatory scrutiny of the company’s products and product candidates, the company’s limited experience as a company in operating its own manufacturing facility, market acceptance of our products, uncertainty related to insurance coverage and reimbursement, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidate. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 4, 2025, and its subsequent periodic reports filed with the SEC. In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website ( ) and LinkedIn website ( ). Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jess Rowlands media@ultragenyx.com