RegulationPriority Review (United States), Breakthrough Therapy (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Fast Track (United States)

Structure/Sequence

Molecular FormulaC18H24ClN3O7

InChIKeyOHUSJUJCPWMZKR-FEGZNKODSA-N

CAS Registry289893-26-1

Clinical Trials associated with Arimoclomol

NL-OMON49378

/ CompletedNot Applicable

Interventional, randomised, partial double-blind, placebo- and positive controlled, multiple-dose, 4-way-cross-over trial Investigating the effect of arimoclomol on cardiac repolarization in healthy men. - CS0344 Orphazyme TQT

Start Date02 Jun 2020

Sponsor / Collaborator-

NCT03836716

/ TerminatedPhase 3

Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial

A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial.

Start Date19 Sep 2019

Sponsor / Collaborator-

NCT04049097

/ TerminatedPhase 3

An Open-label, Non-randomized Trial to Investigate the Efficacy and Safety of Early Versus Delayed Start of Arimoclomol in Patients With Sporadic Inclusion Body Myositis Who Have Completed the IBM4809 Trial

A multicenter, nonrandomized, open-label, uncontrolled clinical extension trial designed to compare the efficacy and safety of early versus delayed start of arimoclomol in the treatment of Inclusion Body Myositis (IBM)

Start Date20 May 2019

Sponsor / Collaborator

University College London

[+1]

100 Clinical Results associated with Arimoclomol

100 Translational Medicine associated with Arimoclomol

100 Patents (Medical) associated with Arimoclomol

Literatures (Medical) associated with Arimoclomol

01 Jun 2026·CURRENT THERAPEUTIC RESEARCH-CLINICAL AND EXPERIMENTAL

Understanding Pharmacokinetic-Drug Interactions With Drugs Approved by the US Food and Drug Administration in 2024 to Better Manage the Risk of Drug Interactions With Concomitant Medications: A Review of Clinical Data From New Drug Applications

Review

Author: Owens, Katie ; Argon, Sophie ; Yu, Jingjing ; Ragueneau-Majlessi, Isabelle ; Wang, Yan

Objective:

This analysis aimed to provide a mechanistic understanding and an evaluation of the clinical relevance of pharmacokinetic drug-drug interactions (DDIs) associated with drugs approved by the Food and Drug Administration in 2024.

Methods:

Drug metabolism, transport, and absorption-based DDI data available in New Drug Applications (NDAs) of small molecular drugs approved (n = 34) was analyzed. The mechanism and clinical outcome of these interactions were characterized based on in vitro, in silico, and clinical data.

Results:

As objects, 11 drugs were identified as clinical substrates. Of these, 7 drugs were substrates of CYP3A, 3 of CYP2C9, one of CYP1A2, and one of CYP2C8, including the sensitive substrates vanzacaftor (CYP3A) and vorasidenib (CYP1A2). As precipitants, 6 drugs (acoramidis, cefepime/enmetazobactam, givinostat, lazertinib, mavorixafor, and resmetirom) were clinical inhibitors of CYP enzymes (2C8, 2C9, 2D6, 2E1, and 3A), with mavorixafor being a CYP2D6 strong inhibitor. Two drugs (elafibranor and tovorafenib) showed weak induction of CYP3A. Regarding transporter data, 3 drugs were substrates of transporters, including seladelpar (BCRP and OAT3), sulopenem (OAT3), and vadadustat (OAT1/3), and 8 drugs (arimoclomol, danicopan, givinostat, lazertinib, mavorixafor, resmetirom, vadadustat, and vazacaftor/tezacaftor/deutivacaftor) were inhibitors of transporters. All clinical DDIs with AUC changes ≥ 2-fold triggered labeling recommendations. Several DDIs with an AUC change <2 also had labeling recommendations, pertaining most often to the concomitant use of drugs with a narrow therapeutic index.

Conclusions:

Mechanistic DDI insights from newly approved therapies can be extrapolated to inform the management of commonly co-administered drugs, supporting a safer and more effective use of new drug products in the context of polypharmacy.

07 Feb 2026·BRAIN

Targeting lipid droplets in FUS-linked amyotrophic lateral sclerosis mitigates neuronal and astrocytic lipotoxicity

Article

Author: Troakes, Claire ; Sephton, Chantelle F ; Serrano, Jeanne ; Parham, Elahe ; Marcadet, Laetitia ; Li, Liang ; Khademullah, C Sahara ; Desmeules, Antoine ; Kennedy, Jaimee ; Durham, Heather D ; Dutchak, Paul A ; Soliz, Jorge ; Pelaez, Mari Carmen ; Vance, Caroline ; Cheng, Zhan

Abstract:

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by the progressive loss of motor neurons, muscle atrophy and systemic energy imbalance. Increasing evidence suggests a metabolic shift in ALS from glucose metabolism toward fatty acid utilization; however, the downstream consequences of this reprogramming on disease progression and neuropathology remain poorly defined.We investigated neurometabolic changes in ALS using in vitro and in vivo models of familial ALS expressing the human fused in sarcoma variant R521G (hFUSR521G), along with post-mortem spinal cord tissue from ALS-FUS cases. A combination of unbiased quantitative metabolomic profiling, immunolabelling, and biochemical and molecular approaches were employed.Mass spectrometry of cortical tissue from hFUSR521G mice and littermates revealed a significant increase in acylcarnitine moieties, key substrates used in mitochondrial β-oxidation and cellular energy production. Complementary cytohistological analyses in hFUSR521G mice demonstrated increased lipid droplets (LDs) and peroxidized lipids in both neurons and astrocytes, consistent with our post-mortem findings in spinal cords of individuals carrying FUS R495X or K510E mutations. Arimoclomol, previously shown to ameliorate behavioural phenotypes in this ALS mouse model, was found to enhance lipid metabolism and reduce lipotoxicity in hFUSR521G mice and in cultured neurons and astrocytes expressing FUS R521G. Mechanistically, arimoclomol enhanced LD-mitochondrial contacts and stimulated mitochondrial β-oxidation-dependent lipid catabolism under both basal and pro-inflammatory conditions. This effect was abrogated by etomoxir, an irreversible inhibitor of carnitine palmitoyltransferase I (CPT1), the rate-limiting enzyme of the carnitine shuttle, highlighting a CPT1-dependent mechanism for lipid mobilization.Together, these findings reveal a previously unrecognized role for mitochondrial lipid metabolism in ALS pathogenesis and identify a therapeutic pathway for mitigating the cytotoxic consequences of lipid and acylcarnitine accumulation in FUS-associated ALS.

01 Nov 2025·NEUROLOGICAL SCIENCES

Dose-dependent effects of Arimoclomol in ALS: insights from a network meta-analysis

Letter

Author: Nb, Karthik ; Sharma, Arkansh

199

News (Medical) associated with Arimoclomol

18 Mar 2026

·www.biospace.com

XOMA Royalty Reports 2025 Financial Results and Highlights Recent Business Achievements

Portfolio receipts : • Achieved over $50 million of cash receipts, including $33.6 million in royalties and $16.9 million milestones, in full year 2025 • Total receipts increased 9% with royalties up 68% versus full year 2024 Business development : Added 22 assets to portfolio, including five programs in Phase 2 or Phase 3 development Stock buyback program : Repurchased and retired 648,048 shares for an aggregate of $16.0 million Company acquisitions : Completed seven acquisitions, accumulating $11.7 million of non-dilutive capital 1 , economic interests of approximately 25% in up to $1.1 billion of milestones and low to mid-single digit royalties from eight partnered programs Key 2026 pipeline events : • Phase 2b data from volixibat in PSC in Q2 and Phase 3 data from ersodetug in tumor HI in 2H • Potential for EMA decisions on OJEMDA™ and MIPLYFFA™ marketing authorization applications • Regulatory updates related to ersodetug in congenital HI and seralutinib in PAH Webcast at 8:00 am Eastern Time today EMERYVILLE, Calif., March 18, 2026 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (NASDAQ: XOMA), the biotech royalty aggregator, reported its 2025 fourth quarter and full year financial results and highlighted recent actions that have the potential to deliver additional shareholder value. “We continue to search for innovative ways to drive enhanced optionality in the XOMA portfolio, with the addition of 22 assets and two platform technologies over the past year,” stated Owen Hughes, Chief Executive Officer of XOMA Royalty. “With multiple commercial assets delivering growing royalty receipts, we achieved positive cash flow from operations and were able to return $16 million of capital through a share buyback in 2025. Looking ahead, with 14 programs in registrational studies, we anticipate a number of catalysts over the ensuing years, including several regulatory updates and late-stage clinical readouts in 2026, which, if positive, will further diversify our commercial royalty streams and drive growing free cash flow in 2027 and beyond.” Portfolio Updates Day One OJEMDA New Drug Application filing in Japan triggered $2 million milestone in 4Q25 OJEMDA FY 2026 revenue guidance of $225 – $250 million 2 In February 2026, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending the conditional marketing authorization of OJEMDA 3 In March 2026, Day One and Servier announced that they have entered into a definitive agreement for Servier to acquire Day One for $21.50 per share in cash, representing a total equity value of approximately $2.5 billion 4 Zevra Therapeutics A Marketing Authorization Application for the evaluation of arimoclomol (MIPLYFFA) for the treatment of NPC is under review by the EMA 5 Rezolute In December 2025, Rezolute announced that the Phase 3 clinical study of ersodetug for the treatment of congenital hyperinsulinism (“HI”) demonstrated reductions from baseline in hypoglycemia events by self-monitored blood glucose at both ersodetug dose levels, but the reductions were not statistically significant compared to placebo, due to a pronounced study effect 6 Rezolute will meet with FDA under its Breakthrough Therapy Designation in the first quarter of 2026 to determine next steps for the program 6 Rezolute anticipates topline results of upLIFT, a Phase 3, single-arm, open-label study in participants with tumor HI, in the second half of 2026 6 Gossamer Bio & Chiesi In February 2026, Gossamer Bio announced topline results from the Phase 3 PROSERA clinical trial evaluating seralutinib for the treatment of PAH 7 Seralutinib demonstrated a placebo-adjusted improvement in Six-Minute Walk Distance (6MWD) of +13.3 meters at Week 24 (p = 0.0320), missing the prespecified alpha threshold of 0.025 7 Gossamer plans to meet with the U.S. FDA to discuss the path forward 7 Volixibat Volixibat VISTAS study in primary sclerosing cholangitis (PSC) topline data expected in Q2 2026 8 Volixibat VANTAGE study in primary biliary cholangitis (PBC) expected to complete enrollment in H2 2026 8 Business Development Activity Takeda Strategic Royalty Share Transaction In December 2025, XOMA amended its collaboration with Takeda XOMA will receive low to mid-single-digit royalties and up to $852.6 million in potential milestones across nine development-stage assets, including osavampator, which is being evaluated in Phase 3 studies for major depressive disorder; volixibat, which is being evaluated in PSC and PBC; OHB-607, which Oak Hill Bio Ltd and its partner are developing for the prevention of bronchopulmonary dysplasia in extremely premature infants; REC-4881, which is in Phase 2 development for familial adenomatous polyposis; and five early-stage Oak Hill Bio assets Prior to amending the collaboration, XOMA held a mid-single digit royalty and $16.25 million in potential milestones associated with mezagitamab Following the transaction, XOMA will retain a low single-digit royalty entitlement on mezagitamab and up to $13.0 million in milestones Company Acquisitions Completed or served as the structuring agent in the acquisition of seven companies since the beginning of 2025 Accumulated non-dilutive capital of $11.7 million, net of transaction expenses Obtained economic interests of approximately 25% in up to $1.1 billion of potential milestone payments and low to mid-single-digit royalties from eight partnered assets Eligible for 25-70% of proceeds related to any future out license or sale of legacy assets or platform technology from these companies, including the ctLNP delivery platform from Generation Bio Fourth Quarter and Full-Year 2025 Financial Results In the fourth quarter of 2025, XOMA Royalty received $3.2 million in cash receipts from royalties and commercial payments and $3.3 million in milestone payments and paid $1.4 million in dividends on the XOMA Royalty Perpetual Preferred stocks. For the full year of 2025, XOMA Royalty received $50.5 million in cash receipts, including $33.6 million in royalties and commercial payments and $16.9 million in milestone payments and fees. During 2025, XOMA Royalty deployed $25.0 million to acquire additional assets for its royalty and milestone portfolio, repurchased 648,048 shares of its common stock for a cost of $16.0 million, and paid $5.5 million in dividends on the XOMA Royalty Perpetual Preferred stocks. Income and Revenue : Income and revenues for the three months ended December 31, 2025 and 2024, were $13.8 million and $8.7 million, respectively. Income and revenues for the years ended December 31, 2025 and 2024, were $52.1 million and $28.5 million, respectively. The increase in both periods was primarily driven by increased income related to VABYSMO® (faricimab-svoa) and OJEMDA™ (tovorafenib) and milestone payments received from Rezolute and Takeda. General and Administrative (G&A) Expenses : G&A expenses for the three months ended December 31, 2025 and 2024, were $10.4 million and $7.0 million, respectively. G&A expenses for the years ended December 31, 2025 and 2024, were $36.1 million and $34.5 million, respectively. The increase of $1.6 million in 2025 was primarily due to an increase in business development and deal-related costs of $3.7 million and an increase in lease costs of $1.0 million primarily related to the HilleVax acquisition partially offset by $3.6 million in costs related to exit packages for Kinnate senior leadership in 2024. G&A expenses for the year ended December 31, 2025, also include an increase of approximately $1.1 million associated with ongoing litigation initiated by XOMA Royalty against Janssen Biotech, Inc., asserting claims for breach of contract and unjust enrichment arising from Janssen's unauthorized use of XOMA’s intellectual property in the commercialization of TREMFYA (guselkumab). XOMA Royalty expects to continue to incur legal fees and other professional service costs associated with pursuing this litigation. Litigation is inherently uncertain, and there can be no assurance regarding the outcome of the matter or the timing or amount of any potential recovery. XOMA Royalty’s G&A expenses for the three months ended December 31, 2025 and 2024, included non-cash stock-based compensation expenses of $3.9 million and $2.2 million, respectively, and $9.3 million and $10.3 million for the full years of 2025 and 2024, respectively. Interest Expense : Interest expense for the three months ended December 31, 2025 and 2024, was $3.0 million and $3.4 million, respectively. Interest expense for the twelve months ended December 31, 2025 and 2024, were $13.0 million and $13.8 million, respectively. Interest expense relates to the Blue Owl Loan established in December 2023. Net Income (Loss) : XOMA Royalty reported net income of $6.1 million and $31.7 million for the three months and year ended December 31, 2025, as compared to net losses of $4.0 million and $13.8 million in the corresponding periods of 2024. Cash Position : On December 31, 2025, XOMA Royalty had cash and cash equivalents of $133.7 million, including $50.8 million in restricted cash. The restricted cash balance included $42.3 million related to the assumed HilleVax lease and $2.2 million related to the Blue Owl Loan. Cash and cash equivalents of $106.4 million as of December 31, 2024, included $4.8 million in restricted cash related to the Blue Owl Loan. Webcast The Company will host a webcast on March 18, 2026, at 8:00 am Eastern Time to discuss the results and provide a business update. The webcast will be accessible on the “News & Events” page in the Investors section of XOMA Royalty’s website ( ). A replay of the webcast will be available for 30 days following the live event. About XOMA Royalty Corporation XOMA Royalty is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA Royalty acquires the potential future economics associated with pre-commercial and commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA Royalty acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The Company has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about the Company and its portfolio, please visit or follow XOMA Royalty Corporation on LinkedIn . Forward-Looking Statements/Explanatory Notes Certain statements contained in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, including statements regarding the timing and amount of potential commercial payments to XOMA Royalty, expectations around future royalty cash flows covering XOMA Royalty’s core operating expenses (the inflection point) and other developments related to VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], DSUVIA® (sufentanil sublingual tablet), and DARE to PLAY™ Sildenafil Cream and Sildenafil Cream, 3.6%; the potential occurrences and timing of the events listed under “ Key 2026 Pipeline Events”; expectations regarding the inflection point in XOMA Royalty’s business model of breakeven operating cash flows; the anticipated timings of regulatory filings and approvals related to assets in XOMA Royalty’s portfolio; and the potential of XOMA Royalty’s portfolio of partnered programs and licensed technologies generating substantial milestone and royalty proceeds over time. In some cases, you can identify such forward-looking statements by terminology such as “anticipate,” “intend,” “believe,” “estimate,” “plan,” “seek,” “project,” “expect,” “may,” “will”, “would,” “could” or “should,” the negative of these terms or similar expressions. These forward-looking statements are not a guarantee of XOMA Royalty’s performance, and you should not place undue reliance on such statements. These statements are based on assumptions that may not prove accurate, and actual results could differ materially from those anticipated due to certain risks inherent in the biotechnology industry, including those related to the fact that our product candidates subject to out-license agreements are still being developed, and our licensees may require substantial funds to continue development which may not be available; we do not know whether there will be, or will continue to be, a viable market for the products in which we have an ownership or royalty interest; and if the therapeutic product candidates to which we have a royalty interest do not receive regulatory approval, our third-party licensees will not be able to market them. Other potential risks to XOMA Royalty meeting these expectations are described in more detail in XOMA Royalty's most recent filing on Form 10-K and in other filings with the Securities and Exchange Commission. Consider such risks carefully when considering XOMA Royalty's prospects. Any forward-looking statement in this press release represents XOMA Royalty's beliefs and assumptions only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. XOMA Royalty disclaims any obligation to update any forward-looking statement, except as required by applicable law. EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA Royalty’s milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], DARE to PLAY™ (Sildenafil Cream), and DSUVIA® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. XOMA ROYALTY CORPORATION CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except per share amounts) Three Months Ended December 31, Year Ended December 31, 2025 2024 2025 2024 Income and revenues: Income from purchased receivables under the EIR method $ 7,706 $ 5,081 $ 26,745 $ 15,066 Income from purchased receivables under the cost recovery method 4,619 1,291 13,744 3,201 Revenue from contracts with customers 1,100 600 10,350 6,650 Revenue recognized under units-of-revenue method 332 1,742 1,310 3,570 Total income and revenues 13,757 8,714 52,149 28,487 Operating expenses: Research and development 281 864 1,712 2,875 General and administrative 10,410 6,993 36,092 34,478 Credit losses on purchased receivables — 7,904 — 30,904 Amortization of intangible assets 884 206 2,961 206 Total operating expenses 11,575 15,967 40,765 68,463 Income (loss) from operations 2,182 (7,253 ) 11,384 (39,976 ) Other income (expense), net: Gains on acquisitions 3,220 — 21,224 19,316 Change in fair value of embedded derivative related to RPA — — — 8,100 Interest expense (3,027 ) (3,394 ) (13,031 ) (13,840 ) Other income, net 3,782 1,021 12,238 6,921 Net income (loss) before tax 6,157 (9,626 ) 31,815 (19,479 ) Income tax (expense) benefit (54 ) 5,658 (103 ) 5,658 Net income (loss) $ 6,103 $ (3,968 ) $ 31,712 $ (13,821 ) Net income (loss) available to (attributable to) common stockholders, basic $ 3,319 $ (5,336 ) $ 18,516 $ (19,293 ) Basic net income (loss) per share available to (attributable to) common stockholders $ 0.27 $ (0.45 ) $ 1.53 $ (1.65 ) Weighted average shares used in computing basic net income (loss) per share available to (attributable to) common stockholders 12,208 11,868 12,081 11,701 Net income (loss) available to (attributable to) common stockholders, diluted $ 4,679 $ (5,336 ) $ 26,184 $ (19,293 ) Diluted net income (loss) per share available to (attributable to) common stockholders $ 0.26 $ (0.45 ) $ 1.46 $ (1.65 ) Weighted average shares used in computing diluted net income (loss) per share available to (attributable to) common stockholders 18,095 11,868 17,982 11,701 XOMA ROYALTY CORPORATION CONSOLIDATED BALANCE SHEETS (in thousands, except share and per share amounts) December 31, December 31, 2025 2024 ASSETS Current assets: Cash and cash equivalents $ 82,908 $ 101,654 Short-term restricted cash 5,441 1,330 Investment in equity securities 382 3,529 Trade and other receivables, net 4,896 1,839 Short-term royalty and commercial payment receivables under the EIR method 22,780 14,763 Short-term royalty and commercial payment receivables under the cost recovery method - 413 Prepaid expenses and other current assets 852 2,076 Total current assets 117,259 125,604 Long-term restricted cash 45,361 3,432 Property and equipment, net 21 32 Operating lease right-of-use assets 256 319 Long-term royalty and commercial payment receivables under the EIR method 4,433 4,970 Long-term royalty and commercial payment receivables under the cost recovery method 55,888 55,936 Exarafenib milestone asset 3,600 3,214 Investment in warrants 697 — Intangible assets, net 44,756 25,909 Other assets - long term 427 1,861 Total assets $ 272,698 $ 221,277 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable $ 2,208 $ 1,053 Accrued and other liabilities 9,885 5,752 Contingent consideration under RPAs, AAAs, and CPPAs - 3,000 Operating lease liabilities 2,464 446 Unearned revenue recognized under units-of-revenue method 1,268 1,361 Preferred stock dividend accrual 1,424 1,368 Current portion of long-term debt 12,526 11,394 Contingent value rights liabilities - current portion 5,045 - Total current liabilities 34,820 24,374 Unearned revenue recognized under units-of-revenue method – long-term 3,193 4,410 Exarafenib milestone contingent consideration 3,600 3,214 Long-term operating lease liabilities 20,114 483 Long-term debt 96,451 106,875 Contingent value rights liabilities - long-term 10,457 - Deferred tax liability 103 - Total liabilities 168,738 139,356 Convertible preferred stock, $0.05 par value, 5,003 shares authorized, issued and outstanding as of December 31, 2025 and December 31, 2024 20,019 20,019 Stockholders’ equity: 8.625% Series A cumulative, perpetual preferred stock, $0.05 par value, 984,000 shares authorized, issued and outstanding as of December 31, 2025 and December 31, 2024 49 49 8.375% Series B cumulative, perpetual preferred stock, $0.05 par value, 3,600 shares authorized, 1,760.5 and 1,600 shares issued and outstanding as of December 31, 2025 and December 31, 2024, respectively — — Common stock, $0.0075 par value, 277,333,332 shares authorized, 11,858,955 and 11,952,377 shares issued and outstanding as of December 31, 2025 and December 31, 2024, respectively 89 90 Additional paid-in capital 1,305,200 1,298,747 Accumulated other comprehensive income 53 73 Accumulated deficit (1,221,450 ) (1,237,057 ) Total stockholders’ equity 83,941 61,902 Total liabilities, convertible preferred stock and stockholders’ equity $ 272,698 $ 221,277 XOMA ROYALTY CORPORATION CONSOLIDATED STATEMENTS OF CASH FLOWS (in thousands) Year Ended December 31, 2025 2024 Cash flows from operating activities: Net income (loss) $ 31,712 $ (13,821 ) Adjustments to reconcile net loss to net cash used in operating activities: Income from purchased receivables under the EIR method (5,925 ) (15,066 ) Stock-based compensation expense 9,273 10,312 Gains on acquisitions (21,224 ) (19,316 ) Credit losses on purchased receivables — 30,904 Gain on sale of equity securities (3,663 ) — Income tax expense (benefit) 103 (5,658 ) Common stock contribution to 401(k) 141 118 Amortization of intangible assets 2,961 206 Depreciation 11 10 Accretion of long-term debt discount and debt issuance costs 1,385 1,350 Non-cash lease expense 64 60 Change in fair value of equity securities (90 ) (131 ) Change in fair value of available-for-sale debt securities classified as cash equivalents (20 ) 73 Change in fair value of derivatives (93 ) — CVR liability working capital adjustment (394 ) — Changes in assets and liabilities: Trade and other receivables, net (2,426 ) (835 ) Prepaid expenses and other assets 3,839 302 Accounts payable and accrued liabilities (10,597 ) 1,598 Operating lease liabilities (876 ) (284 ) Unearned revenue recognized under units-of-revenue method (1,310 ) (3,570 ) Net cash provided by (used in) operating activities 2,871 (13,748 ) Cash flows from investing activities: Net cash acquired in Kinnate acquisition — 18,926 Net cash acquired in Turnstone acquisition 3,850 — Net cash and restricted cash acquired in HilleVax acquisition 46,384 — Net cash, cash equivalents, and restricted cash acquired in LAVA acquisition 15,263 — Net cash and cash equivalents acquired in Mural acquisition 4,464 — Payments of consideration under RPAs, AAAs, and CPPAs (8,000 ) (53,000 ) Receipts under RPAs, AAAs, and CPPAs 3,300 29,248 Net payment for IP acquired under the Pulmokine Acquisition — (20,176 ) Payment for BioInvent contract-based intangible asset (20,725 ) — Payment of contingent consideration related to Kinnate IP asset (550 ) — Purchase of property and equipment — (20 ) Purchase of equity securities (99 ) (3,237 ) Sale of equity securities 6,999 — Payment to issue short-term loan to Xeno (5,877 ) — Receipt from short-term loan repayment by Xeno 5,877 — Net cash provided by (used in) investing activities 50,886 (28,259 ) Cash flows from financing activities: Proceeds from issuance of common stock 323 — Proceeds from issuance of preferred stock 4,019 — Payments of preferred and common stock issuance and financing costs (672 ) — Principal payments – debt (10,598 ) (6,902 ) Debt issuance costs and loan fees paid in connection with long-term debt (80 ) (740 ) Payment of preferred stock dividends (5,472 ) (5,472 ) Repurchases of common stock (16,043 ) (13 ) Proceeds from exercise of options and other share-based compensation 5,046 5,214 Taxes paid related to net share settlement of equity awards (2,986 ) (3,214 ) Net cash used in financing activities (26,463 ) (11,127 ) Net increase (decrease) in cash, cash equivalents, and restricted cash 27,294 (53,134 ) Cash, cash equivalents, and restricted cash as of the beginning of the period 106,416 159,550 Cash, cash equivalents, and restricted cash as of the end of the period $ 133,710 $ 106,416 Supplemental Cash Flow Information: Cash paid for interest $ 11,906 $ 9,985 Cash paid for taxes $ 277 $ — Non-cash investing and financing activities: Accrual of contingent value rights liability in the Turnstone acquisition $ 1,110 $ — Accrual of contingent value rights liability in the HilleVax acquisition $ 5,673 $ — Accrual of contingent value rights liability in the LAVA acquisition $ 9,114 $ — Right-of-use assets obtained in exchange for operating lease liabilities in the HilleVax acquisition $ 22,525 $ — Relative fair value basis reduction of right-of-use assets in the HilleVax acquisition $ (22,525 ) $ — Transaction costs in connection with Mural acquisition included in accrued expenses $ 320 $ — Excise tax accrual due to stock repurchases $ 68 $ — Reclassification of equity classified awards to liabilities $ (739 ) $ — Reclassification of deferred issuance cost to equity $ 578 $ — Preferred stock dividend accrual $ 1,424 $ 1,368 Estimated fair value of the Exarafenib milestone asset $ — $ 2,922 Estimated fair value of the Exarafenib milestone contingent consideration $ — $ (2,922 ) Right-of-use assets obtained in exchange for operating lease liabilities in the Kinnate acquisition $ — $ 824 Relative fair value basis reduction of rights-of-use assets in the Kinnate acquisition $ — $ (824 ) Accrual of contingent consideration under the Affitech CPPA $ — $ 3,000 Accrual of contingent consideration under the LadRx AAA $ — $ 1,000 Investor contact: Media contact: Juliane Snowden Kathy Vincent XOMA Royalty Corporation KV Consulting & Management +1-646-438-9754 kathy@kathyvincent.com juliane.snowden@xoma.com 1 This amount includes structuring agent fees associated with Repare Therapeutics and ESSA Pharmaceuticals. 2 3 4 5 6 7 8

Phase 3Clinical ResultFinancial StatementBreakthrough TherapyPhase 2

18 Mar 2026

·www.biospace.com

XOMA Royalty Declares Quarterly Preferred Stock Dividends - March 18, 2026

EMERYVILLE, Calif., March 18, 2026 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (Nasdaq: XOMA) today announced its Board of Directors has authorized the following cash dividends to holders of XOMA Royalty’s Series A and Series B Cumulative Preferred Stock: Holders of the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) shall receive a cash dividend equal to $0.53906 per share. Holders of depositary shares, each representing 1/1000 of a share of XOMA Royalty’s 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO), shall receive a cash dividend equal to $0.52344 per depositary share. The preferred dividends will be paid on or about April 15, 2026, to respective holders of record at the close of business on April 2, 2026. About XOMA Royalty Corporation XOMA Royalty is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA Royalty acquires the potential future economics associated with pre-commercial and commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA Royalty acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The Company has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about the Company and its portfolio, please visit or follow XOMA Royalty Corporation on LinkedIn . EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA Royalty’s milestone and royalty portfolio are VABYSMO ® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY ® [coagulation factor IX (recombinant)], DARE to PLAY™ (Sildenafil Cream), and DSUVIA ® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. Investor contact: Media contact: Juliane Snowden Kathy Vincent XOMA Royalty Corporation KV Consulting & Management +1-646-438-9754 +1-310-403-8951 juliane.snowden@xoma.com kathy@kathyvincent.com

09 Mar 2026

·investors.zevra.com

Zevra Reports Fourth Quarter and Full Year 2025 Financial Results

Q4 net revenue of $34.1 million, representing 31% growth quarter-over-quarter FY 2025 net revenue of $106.5 million, driven by growth in MIPLYFFA® net revenue to $87.4 million 2025 EPS of $1.40 basic and $1.35 diluted Company to host conference call and webcast TODAY, March 9, 2026, at 4:30 p.m. ET BOSTON, March 09, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today reported its financial results for the fourth quarter and full year ended December 31, 2025. “MIPLYFFA is making a meaningful difference for patients with Niemann-Pick disease type C, and its strong, accelerating performance reinforces our confidence in its long-term potential,” said Neil F. McFarlane, Zevra's President and Chief Executive Officer. “We have officially relocated our global corporate headquarters to Boston, enhancing our access to a deep and highly specialized talent pool. Looking ahead, we are focused on executing against multiple near-term growth opportunities in 2026, and believe we are well-positioned to execute on our strategic priorities to create meaningful value for the rare disease community and our shareholders.” MIPLYFFA® (arimoclomol) Highlights U.S.: Received 24 MIPLYFFA prescription enrollment forms for Niemann-Pick disease type C (NPC) during Q4 2025, bringing the total to 52 in 2025 and 161 since product launch. Market access has reached 68% of covered lives, supporting broad access to MIPLYFFA. EU: A Marketing Authorisation Application for the evaluation of arimoclomol for the treatment of NPC is under review by the European Medicines Agency (EMA). Arimoclomol has been designated an Orphan Medicinal Product by the EMA. The review process is progressing as expected, and at year-end, the EMA sent their 120-day list of questions, which the Company is prepared to respond to within the regulatory 90-day clock stop period. Global Expanded Access Program (EAP): In Q4 2025, the Company executed a distribution agreement to broaden access to arimoclomol to select territories beyond Europe. As of December 31, 2025, 113 patients were enrolled in the global EAP. Pipeline and Innovation Highlights Enrolled eight patients in the event-driven Phase 3 DiSCOVER trial for the treatment of Vascular Ehlers-Danlos Syndrome during Q4 2025, bringing the total number of enrolled patients at year-end to 52, with a total of one confirmed event. The Company has recently engaged the Food and Drug Administration (FDA) in a Type C meeting to discuss regulatory options to accelerate the development program. Publication Highlights Four posters were presented at the 22nd Annual WORLD Symposium™ including new data for MIPLYFFA. Four-year real-world data from the U.S. EAP demonstrated that it was well tolerated and stabilized disease progression in the overall cohort, and new data for the subgroup of adults in the EAP showed consistent results. In the post hoc efficacy analysis of the randomized, placebo-controlled study, there was a statistically significant slowing of disease progression compared to placebo as early as three months after treatment initiation, with sustained and increasing benefit through 12 months, highlighting early onset of clinical effect in patients with NPC. Abstract titled “Analysis of NPC1 Genotypes: Findings from the U.S. Arimoclomol Expanded Access Program for Niemann-Pick Type C” (#P214) was accepted for poster presentation at the Annual Clinical Genetics Meeting on Friday, March 13, 2026. Four-year real-world data from the U.S. EAP demonstrated that it was well tolerated and stabilized disease progression in the overall cohort, and new data for the subgroup of adults in the EAP showed consistent results. In the post hoc efficacy analysis of the randomized, placebo-controlled study, there was a statistically significant slowing of disease progression compared to placebo as early as three months after treatment initiation, with sustained and increasing benefit through 12 months, highlighting early onset of clinical effect in patients with NPC. FY 2025 Financial Highlights Revenue, Net: $106.5 million, which includes $87.4 million of MIPLYFFA net revenue, $0.8 million of OLPRUVA net revenue, $13.0 million in net reimbursements from our EAP, and $5.0 million in royalties and other reimbursements under the AZSTARYS® license agreement. For full year 2024, net revenue was $23.6 million, which was primarily driven by $10.1 million in MIPLYFFA net revenue, $0.1 million in OLPRUVA net revenue, $9.1 million in net reimbursements from our EAP, and $4.3 million in royalties and other reimbursements under the AZSTARYS® license agreement. Cost of Product Revenue: $16.5 million, excluding non-cash intangible asset amortization. Cost of product revenue for the year ended December 31, 2024 was $7.4 million. Operating Expenses: $90.4 million, which includes non-cash compensation expense of $12.6 million. Total operating expenses for the year ended December 31, 2024 were $97.0 million. R&D expense was $12.7 million, which was a decrease of $29.4 million compared to $42.1 million for FY 2024 due primarily to a decrease in personnel-related costs, combined with a decrease in third-party costs. SG&A expense was $77.6 million, which was an increase of $22.7 million compared to $54.9 million for FY 2024 due primarily to an increase in third-party costs related to MIPLYFFA, combined with an increase in personnel-related costs, professional fees, and other expenses associated with our commercial, medical and launch activities. Net Income (Loss): Net income of $83.2 million, or $1.40 per basic and $1.35 per diluted share for 2025, compared to a net loss of $(105.5) million, or $(2.28) per basic and diluted share for 2024. 2025 net income includes non-cash fair value adjustment of $2.2 million, non-cash stock-based compensation expense of $12.6 million, and non-cash intangible asset amortization expense of $3.9 million. Cash Position: Cash, cash equivalents and securities were $238.9 million as of December 31, 2025. Based on the Company’s current operating forecast, the Company believes it has sufficient resources and financial flexibility to execute on its strategic priorities independent from the capital markets. Common and Fully Diluted Shares O/S: As of December 31, 2025, total shares of common stock outstanding were 56,854,781, and fully diluted common shares were 67,939,395, which included 7,060,457 issuable from outstanding awards under equity incentive plans, and 4,024,157 shares issuable upon exercise of warrants. R&D expense was $12.7 million, which was a decrease of $29.4 million compared to $42.1 million for FY 2024 due primarily to a decrease in personnel-related costs, combined with a decrease in third-party costs. SG&A expense was $77.6 million, which was an increase of $22.7 million compared to $54.9 million for FY 2024 due primarily to an increase in third-party costs related to MIPLYFFA, combined with an increase in personnel-related costs, professional fees, and other expenses associated with our commercial, medical and launch activities. Q4 2025 Financial Highlights Revenue, Net: $34.1 million for Q4 2025, which includes $26.4 million of MIPLYFFA net revenue, $0.4 million of OLPRUVA net revenue, $5.6 million in net reimbursements from the EAP, and $1.8 million in royalties and other reimbursements under the AZSTARYS® license agreement. For Q4 2024, total net revenue was $12.0 million, which includes $10.1 million of MIPLYFFA net revenue, $0.1 million of OLPRUVA net revenue, $1.1 million in net reimbursements from the EAP, and $0.7 million in royalties and other reimbursements under the AZSTARYS® license agreement. Cost of Product Revenue: $1.5 million for Q4 2025, excluding non-cash intangible asset amortization. Cost of product revenue for Q4 2024 was $1.4 million. Operating Expenses: $23.0 million for Q4 2025, which includes non-cash stock compensation of $4.3 million. Total operating expenses for Q4 2024 were $24.5 million. R&D expense was $2.6 million for Q4 2025, which was a decrease of $5.8 million compared to $8.4 million for Q4 2024 due primarily to a decrease in personnel-related costs, combined with a decrease in third-party costs. SG&A expense was $20.4 million for Q4 2025, which was an increase of $4.3 million compared to $16.1 million for Q4 2024, due primarily to an increase in third-party costs incurred, mainly due to higher spend on commercial products, combined with an increase in personnel-related costs. Net Income (Loss): Net income of $12.2 million, or $0.20 per basic and $0.19 per diluted share for Q4 2025, compared to a net loss of $(35.7) million, or $(0.67) per basic and diluted share for Q4 2024. R&D expense was $2.6 million for Q4 2025, which was a decrease of $5.8 million compared to $8.4 million for Q4 2024 due primarily to a decrease in personnel-related costs, combined with a decrease in third-party costs. SG&A expense was $20.4 million for Q4 2025, which was an increase of $4.3 million compared to $16.1 million for Q4 2024, due primarily to an increase in third-party costs incurred, mainly due to higher spend on commercial products, combined with an increase in personnel-related costs. Conference Call Information Zevra will host a conference call and audio webcast TODAY at 4:30 p.m. ET to discuss its corporate update and financial results for the fourth quarter and full year 2025. A link to the audio webcast is accessible on the “Events & Presentations” page in the Investor Relations section of the Zevra's website at investors.zevra.com. A replay of the webcast will be available for 90 days beginning at approximately 5:30 p.m. ET on March 9, 2026. Additionally, interested participants and investors may access the conference call by dialing either: (800) 579-2543 (United States) +1 (785) 424-1789 (International) Conference ID: ZVRAQ425 About MIPLYFFA® (arimoclomol) MIPLYFFA (arimoclomol) is Zevra’s approved therapy for the treatment of Niemann-Pick disease type C (NPC). Approved by the U.S. Food and Drug Administration on Sep. 20, 2024, MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal phase 3 trial, MIPLYFFA halted disease progression compared to placebo over the one-year duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. The extensive data generated for MIPLYFFA has shown long-term, meaningful clinical outcomes with 5 and in some patients 7 years of patient experience across more than 270 NPC patients worldwide through a Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, which is the most expansive clinical development program in NPC to date. Zevra has submitted a Marketing Authorization Application to the European Medicines Agency for the evaluation of arimoclomol for the treatment of Niemann-Pick disease type C. INDICATIONS AND USAGE MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions: Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve. Embryofetal Toxicity: MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential. Increased Creatinine without Affecting Glomerular Function: Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function. During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation. The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight. Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema. To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1 800-FDA-1088 or www.fda.gov/medwatch. Drug Interaction(s): Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate. Use in Females and Males of Reproductive Potential: Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights. Renal Impairment: The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to <50 mL/minute is lower than the recommended dosage (less frequent dosing) in patients with normal renal function. MIPLYFFA capsules for oral use are available in the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg. About OLPRUVA® OLPRUVA (sodium phenylbutyrate) is Zevra’s approved treatment for the treatment of certain UCDs. OLPRUVA (sodium phenylbutyrate) for oral suspension is a prescription medicine used along with certain therapies, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with UCDs, involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). OLPRUVA is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment. For more information, please visit www.OLPRUVA.com. Important Safety Information Certain medicines may increase the level of ammonia in your blood or cause serious side effects when taken during treatment with OLPRUVA. Tell your doctor about all the medicines you or your child take, especially if you or your child take corticosteroids, valproic acid, haloperidol, and/or probenecid. OLPRUVA can cause serious side effects, including: 1) nervous system problems (neurotoxicity). Symptoms include sleepiness, tiredness, lightheadedness, vomiting, nausea, headache, confusion, 2) low potassium levels in your blood (hypokalemia) and 3) conditions related to swelling (edema). OLPRUVA contains salt (sodium), which can cause swelling from salt and water retention. Tell your doctor right away if you or your child get any of these symptoms. Your doctor may do certain blood tests to check for side effects during treatment with OLPRUVA. If you have certain medical conditions such as heart, liver or kidney problems, are pregnant/planning to get pregnant or breast-feeding, your doctor will decide if OLPRUVA is right for you. The most common side effects of OLPRUVA include absent or irregular menstrual periods, decreased appetite, body odor, bad taste or avoiding foods you ate prior to getting sick (taste aversion). These are not all of the possible side effects of OLPRUVA. Call your doctor for medical advice about side effects. You may report side effects to U.S. FDA at 1-800-FDA-1088. About Celiprolol Celiprolol is Zevra’s investigational clinical candidate for the treatment of Vascular Ehlers-Danlos Syndrome (VEDS). Celiprolol has been granted Orphan Drug and Breakthrough Therapy designations by the U.S. FDA. Zevra recently restarted enrollment in the DiSCOVER trial, a Phase 3 trial being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. FDA. Celiprolol’s mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with rare diseases. The Company is focused on broadening access through geographic expansion opportunities, progressing its pipeline toward key milestones, and delivering meaningful therapeutics. The commercialization of its lead product, marketed in the U.S. for Niemann-Pick disease type C (NPC), a rare, progressive neurodegenerative disease, provides a strong corporate foundation and validates its ability to advance therapies from development to market. Zevra's vision is realized through disciplined execution of its strategic plan and core values — patient centricity, integrity, accountability, innovation, and courage — which guide its efforts to deliver long-term value. For more information, please visit www.zevra.com or follow us on X and LinkedIn. Cautionary Note Concerning Forward-Looking Statements This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding our growth; the U.S. launch and potential global expansion of MIPLYFFA®; submissions to, review by, and discussions with the EMA regarding arimoclomol; promise and potential impact of our preclinical or clinical trial data; the initiation, timing and results of any clinical trials or readouts; the potential benefits of any of our products or product candidates for any specific disease or at any dosage; future research and development activities; our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company; our financial position, including our cash balance and anticipated cash runway; and the timing of any of the foregoing. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the “Risk Factors” section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2025, to be filed with the Securities and Exchange Commission, and Zevra's other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Investor Contact Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com Media Contact Julie Downs+1 (508) 246-3230 jdowns@zevra.com

Phase 3Clinical ResultBreakthrough TherapyDrug ApprovalOrphan Drug

100 Deals associated with Arimoclomol

External Link

KEGG	Wiki	ATC	Drug Bank
-	Arimoclomol	-	DB05025

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Niemann-Pick Disease, Type C	United States	Zevra Therapeutics, Inc.	20 Sep 2024
Niemann-Pick Disease, Type C	United States	Zevra Denmark as	20 Sep 2024

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Myositis, Inclusion Body	Phase 3	United States	University College London	20 May 2019
Myositis, Inclusion Body	Phase 3	United Kingdom	University College London	20 May 2019
Amyotrophic Lateral Sclerosis	Phase 3	United States	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Belgium	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Canada	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	France	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Germany	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Italy	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Netherlands	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Poland	Zevra Denmark as	31 Jul 2018

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02612129 (NPC-002, Pubmed) Manual	Phase 2/3	Niemann-Pick Disease, Type C	50	arimoclomol (DB phase)	-	Positive	01 Aug 2025
				arimoclomol (OLE phase)	ngdivmgjnv(riifgdeyut) = vrirarowvr xgerlriqyh (kbeisecifb, 4.8) View more
NCT02612129 (FDA_CDER) Manual	Phase 3	Niemann-Pick Disease, Type C	50	MIPLYFFA with miglustatMIPLYFFA with miglustat	fioracuhaw(gksofzfaow) = xlddaldlfl hgilxvmglj (ufrqamklql, 1)	Positive	20 Sep 2024
				Placebo with miglustat	fioracuhaw(gksofzfaow) = uhcpxqzput hgilxvmglj (ufrqamklql, 3.4)
NCT04049097 (CTgov)	Phase 3	Myositis, Inclusion Body	121	Arimoclomol	qthixascrl(jweqiztluq) = ukanifmtxp ovhrtqvhaw (cadwaywqiz, 2.60) View more	-	15 Sep 2023
NCT03491462 (ORARIALS-01, CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	245	Arimoclomol (Arimoclomol (up to 76 Weeks))	lkhgingbfw(pbodufzpnb) = tjejbdiwke juyivzuaat (cycqzvupvn, 0.291) View more	-	24 Aug 2023
				Placebo (Placebo (up to 76 Weeks))	lkhgingbfw(pbodufzpnb) = yyahklmfpz juyivzuaat (cycqzvupvn, 0.283) View more
NCT03836716 (CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	120	Arimoclomol	iukrvegfcv = molrogxadl azpzauopxy (tylkahdaqp, zrxssbppbk - hppmpvsinz) View more	-	24 Aug 2023
NCT02612129 (NPC-002, CTgov)	Phase 2/3	Niemann-Pick Disease, Type C	50	arimoclomol (Arimoclomol (12-month Double-blind Phase))	uttybhzriu(tbozppgeln) = zehhvqcxrm wimeshlcaw (fxidhjjrhe, nrzqfzwjza - lhazjhmvcu) View more	-	09 Jun 2023
				Placebo (Placebo (12-month Double-blind Phase))	uttybhzriu(tbozppgeln) = dztarlfntv wimeshlcaw (fxidhjjrhe, mucplmlxbf - qltblrzlxo) View more
NCT02753530 (NCT02753530, CTgov)	Phase 2	Myositis, Inclusion Body	152	Arimoclomol (Arimoclomol (20 Months))	dckcfsvgtd(jfjybstmcu) = cvrdrzodqh uostjdatta (ylwawwkgvh, ogomjasaja - fmpmqapnab) View more	-	10 May 2023
				Placebo (Placebo (20 Months))	dckcfsvgtd(jfjybstmcu) = ywqxzgpdrp uostjdatta (ylwawwkgvh, nzddfggzew - ryixmfzssb) View more
NCT02753530 (ACR2021) Manual	Phase 2	Myositis, Inclusion Body	152	Arimoclomol	ieggjxuioe(hbmmuggfuh) = vqfhuimrzq ltqlvlqizg (kzelsmobca ) View more	Negative	01 Sep 2021
				Placebo	ieggjxuioe(hbmmuggfuh) = ngjffsuszo ltqlvlqizg (kzelsmobca ) View more
NCT03491462 (ORARIALS-01, GlobeNewswire) Manual	Phase 3	Amyotrophic Lateral Sclerosis	245	arimoclomol 248 mg	rcqpgjneld(fruketulfv) = did not meet its primary endpoints pkdsadvbby (ohkthcvsdc ) View more	Negative	07 May 2021
				Placebo
NCT00706147 (Pubmed \| Ann Neurol \| Neurology) Manual	Phase 2/3	Amyotrophic Lateral Sclerosis	36	arimoclomol	aeptyhfogm(lnqzunnijx) = lkbyeneilq meokaxqgzz (wkldourqtg, -0.63 to 1.63) View more	Positive	13 Feb 2018
				Placebo	-

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis

Arimoclomol

Overview

Basic Info

Structure/Sequence

Related

External Link

R&D Status

Clinical Result

Translational Medicine

Deal

Core Patent

Clinical Trial

Approval

Regulation