Arimoclomol

– Takeda regains a majority of XOMA Royalty’s royalty interest in mezagitamab (TAK-079) –– XOMA Royalty will be entitled to payments based on a share of milestones and royalties associated with nine development-stage assets held within Takeda’s externalized assets portfolio – EMERYVILLE, Calif., Dec. 30, 2025 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (NASDAQ: XOMA), the biotech royalty aggregator, announced today it has amended its collaboration, originally established in 2006, with Takeda through a strategic royalty share transaction. Takeda’s royalty and milestone payment obligations to XOMA Royalty related to mezagitamab will be reduced, and XOMA Royalty will receive payments based on low to mid-single-digit royalties and milestones across a basket of nine development-stage assets that are held within Takeda’s externalized assets portfolio. “We continue to find ways to expand and diversify our royalty and milestone portfolio through creative transactions that are beneficial to both parties,” stated Brad Sitko, Chief Investment Officer of XOMA Royalty. “Takeda and XOMA Royalty have a long history of collaboration. By amending our collaboration, we are able to return a portion of our mezagitamab economics to Takeda, while also expanding and diversifying XOMA Royalty’s portfolio across several interesting early- and late-stage programs.” MezagitamabPrior to amending the collaboration, XOMA Royalty held a mid-single digit royalty and $16.25 million in potential milestones associated with mezagitamab. Going forward, XOMA Royalty will retain a low single-digit royalty entitlement on mezagitamab and up to $13.0 million in milestones. Development-Stage Assets from Takeda’s Externalized Assets Portfolio Osavampator Neurocrine Biosciences is developing osavampator, a potential first-in-class, investigational alpha-amino-3-hydroxy-5-methyl-4-isoxazole propionic acid (AMPA) positive allosteric modulator (PAM) for patients who have inadequate response to treatment for major depressive disorder (MDD). Volixibat Mirum Pharmaceuticals is developing volixibat, a minimally absorbed, orally administered investigational therapy designed to selectively inhibit ileal bile acid transporter (IBAT), for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC). OHB-607 and five early-stage Oak Hill Bio assets Oak Hill Bio and their partner are developing OHB-607, a recombinant human IGF-1/IGFBP-3 for the prevention of bronchopulmonary dysplasia in premature infants. Additional Oak Hill Bio assets that have the potential to generate royalties address other high unmet need or rare disease areas. REC-4881 Recursion Pharmaceuticals is developing REC-4881, an investigational MEK1/2 inhibitor for familial adenomatous polyposis, a rare tumor predisposition syndrome affecting approximately 50,000 people in the U.S., France, Germany, Italy, Spain, and the UK1. About XOMA Royalty CorporationXOMA 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 sellers receive non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. XOMA Royalty 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 XOMA Royalty and its portfolio, please visit www.xoma.com or follow XOMA Royalty Corporation on LinkedIn. XOMA Royalty Forward-Looking Statements/Explanatory NotesCertain 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 and milestone payments to XOMA Royalty and other developments related to mezagitamab, osavampator, volixibat, OHB-607 and the Oak Hill Bio assets, and REC-4881. 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-Q 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)], 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 Investor Contact:XOMA Royalty Media Contact:Juliane SnowdenKathy VincentXOMA Royalty CorporationKV Consulting & Management+1-646-438-9754kathy@kathyvincent.com juliane.snowden@xoma.com 1 https://ir.recursion.com/news-releases/news-release-details/preliminary-phase-1b2-data-rec-4881-familial-adenomatous

CELEBRATION, Fla., Dec. 29, 2025 (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 announced that the Company has executed an exclusive expanded access distribution agreement with Uniphar, an Ireland-based pharmaceutical services provider with a proven record of success in global warehousing, distribution and supply chain management. The agreement enables Niemann-Pick Disease Type C (NPC) patients to access MIPLYFFA® (arimoclomol), for reimbursed named patient supply in select territories outside of Europe. NPC is an ultra-rare, relentlessly progressive, genetic disorder that leads to premature mortality. MIPLYFFA, used in conjunction with miglustat, is the only treatment shown to halt disease progression by addressing the underlying pathology of NPC with improvement seen at the first evaluation at week 12, and durable effect for more than five years. “There remains a clear unmet need within the rare disease community and this distribution agreement enables us to further our mission by expanding access and supporting a greater number of patients and families living with NPC,” said Neil F. McFarlane, Zevra's President and Chief Executive Officer. “By leveraging Uniphar’s leadership and infrastructure, we have an opportunity to initially address a select patient population, while continuing to prioritize our U.S. commercial launch and prepare for a potential EU approval.” Commenting on the agreement, Brian O’Shaunnessy, Chief Commercial Officer at Uniphar, said, “Uniphar is proud to partner with Zevra to expand global access to MIPLYFFA. We believe our proven expertise in global distribution and supply chain management combined with Zevra's innovative approach to rare disease therapies will help make a life-changing difference to patients living with NPC.” MIPLYFFA is approved by the U.S. Food and Drug Administration and is commercially available in the U.S. A Marketing Authorisation Application for the evaluation of arimoclomol for the treatment of NPC has been validated and is under review by the European Medicines Agency (EMA). 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 more than 5 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 INFORMATIONHypersensitivity 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 Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. Expanded access programs are made available by Zevra Therapeutics, Inc. and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy, as published on its website. Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion. About Uniphar Uniphar is a trusted global partner to pharma, medtech and biotech, delivering solutions that connect medicines and technologies with patients worldwide. With over 57 years of experience and partnerships with 200+ multinational clients, Uniphar combines deep expertise, infrastructure and market insight to meet the evolving needs of the life sciences sector. Uniphar Pharma integrates Development, Clinical, Access, Medical, Commercial, Distribution and Global Sourcing to support the full product lifecycle – from early-stage research through to commercialization and beyond. Uniphar’s unified platform spans medical affairs, regulatory strategy, market access, patient engagement, commercial services, distribution and supply chain. With a team of 3,500+ across 180 countries, Uniphar bridges manufacturers, healthcare providers and patients, ensuring innovative therapies reach those who need them – driving value, access and better outcomes worldwide. From molecule to market and beyond. 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 the Company’s expanded access program. 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, 2024, filed on March 12, 2025, Quarterly Report on Form 10-Q for the three and nine months ended September 30, 2025, filed on November 5, 2025, as well as 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) 754-2545 nochsner@zevra.com Media Contact Julie Downs+1 (508) 246-3230 jdowns@zevra.com