Arimoclomol

The EMA's drug advisory panel backed a dozen medicines for approval at its July meeting, including Johnson & Johnson's IL-23 antagonist Icotyde (icotrokinra) for plaque psoriasis, Roche's ocular implant Susvimo (rabinizumab) for wet age-related macular degeneration (wAMD), and several cholesterol-lowering therapies.Among the more closely watched decisions, the Committee for Medicinal Products for Human Use (CHMP) recommended Icotyde for adults and adolescents aged 12 years and older with moderate-to-severe plaque psoriasis. If approved by the European Commission, Icotyde would be the first oral medicine targeting the Il-23 receptor available in the region, and expand a class that is dominated by injectables at the moment.The drug was approved in the US in March, with J&J recently saying its product is off to a strong commercial start. For related analysis, see Physician Views Results: J&J's Icotyde may have an edge on Takeda's TYK2 hopeful and Spotlight On: As Stelara fades, J&J's IL-23 tag team steps up.The CHMP recommendation was supported by four Phase III trials involving roughly 2500 patients. Across three studies, between 50% and 57% of patients achieved at least a 90% reduction in Psoriasis Area and Severity Index (PASI 90) scores after 16 weeks, versus between 1% and 4% of placebo recipients. Additionally, 65% to 71% of those given Icotyde achieved clear or almost clear skin, compared with 8% to 11% for placebo.Twice-yearly refillsMeanwhile, the Susvimo recommendation covers its use in adults with wAMD whose disease has stabilised following intravitreal anti-VEGF therapy. The treatment combines ranibizumab, which blocks VEGF, with a surgically implanted refillable ocular device designed to continuously release the drug.The implant is refilled every six months and would reduce the burden of monthly intravitreal injections that have been the standard for ranibizumab-treated patients since the drug was first approved in Europe nearly 20 years ago. If ultimately cleared by the European Commission, Susvimo would become the first implant of its kind approved in the EU.The recommendation was based on a Phase III study of 415 patients showing that Susvimo maintained visual and anatomical outcomes comparable to monthly ranibizumab injections. Common side effects included iritis, conjunctival bleb leak, cataract, vitreous floaters, conjunctival haemorrhage, conjunctival hyperaemia, eye pain and headache.Originally approved in the US for wAMD in 2021, Susvimo has since had its label expanded there to include diabetic macular oedema and diabetic retinopathy.Trio of anti-cholesterol drugsCHMP also backed approval of several cholesterol treatments for adults, including NewAmsterdam Pharma and Menarini's Ubeslo, a once-daily oral non-statin therapy containing low-dose CETP inhibitor obicetrapib, as well as Evlarco, a fixed-dose combination containing obicetrapib plus ezetimibe.Those positive opinions recommend the drugs for patients with primary hypercholesterolemia, both heterozygous familial and non-familial or mixed dyslipidemia based on Phase III data from the BROADWAY, BROOKLYN and TANDEM trials, which demonstrated significant LDL-C reductions reaching up to 40% with Ubeslo monotherapy and about 50% with Evlarco, when compared to placebo (see – KOL Views Q&A: NewAmsterdam's obicetrapib will elbow into LDL space pending two unknowns, says leading cardiologist).CHMP also adopted a positive opinion for LIB Therapeutics' Lyrokaul (lerodalcibep), an injection that patients can self-administer monthly. The third-generation PCSK9 inhibitor was approved by the FDA in December as Lerochol, based on data from the Phase III LIBerate programme showing sustained LDL-C reductions of 60% or more in patients with or at high risk of cardiovascular disease, and 50% or more in those with heterozygous familial hypercholesterolemia who have more severe LDL-C elevations.A first for cALDAmong medicines for rare diseases, CHMP recommended approval under exceptional circumstances for Minoryx Therapeutics and Neuraxpharm's Nezglyal (leriglitazone) to treat cerebral adrenoleukodystrophy (cALD) in patients aged two to 12 years with gadolinium-negative brain lesions.The opinion was based on the Phase II/III NEXUS study, where Nezglyal achieved the primary endpoint of clinical and radiological arrested disease at week 96 or at a visit prior to haematopoietic stem cell transplant, as well as additional real-world evidence from compassionate-use programmes.If regulators give the green light, expected in September, Nezglyal would become the first pharmacological treatment for cALD in the EU.Rare disease snubThe committee also issued negative opinions for three applications, including for Zevra Therapeutics' Meplyffa (arimoclomol citrate), which the company had been seeking approval for to treat Niemann-Pick disease type C in patients aged two years and older in combination with miglustat. "Due to the way the data had been handled and the results analysed, there were uncertainties about the reliability and robustness of the results. Moreover, no benefit was seen in terms of ambulation and cognition," the EMA said.The agency noted that data for a subgroup of patients who were also taking miglustat showed a treatment effect favouring Meplyffa, though this "was not considered robust since effectiveness had not been demonstrated in the overall population."Zevra, whose shares tumbled nearly 24% on Friday, said it is going to request a re-examination of the CHMP opinion.The oral heat shock protein amplifier was approved by the FDA in 2024 under the brand Miplyffa, after a bumpy regulatory journey. That decision was based on data from a 50-patient, placebo-controlled Phase II/III study where patients who received thrice-daily Miplyffa had slower disease progression than those on placebo, as measured by the 4-domain NPC Clinical Severity Scale (R4DNPCCSS).

Acquisition adds seven commercial products including VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), and MIPLYFFA® (arimoclomol), and more than 100 development and commercial-stage assets, creating long-term opportunities to drive growth Ligand to provide updated 5-year outlook during Investor Day on December 8, 2026 JUPITER, Fla., July 14, 2026 (GLOBE NEWSWIRE) -- Ligand Pharmaceuticals Incorporated (Nasdaq: LGND) today announced it has completed its acquisition of XOMA Royalty Corporation (“XOMA Royalty”), a biotechnology royalty aggregator, for $39.00 per share of common stock in cash, for a total equity value of approximately $739 million. XOMA Royalty stockholders also received one non-transferable Contingent Value Right (“CVR”) per share entitling the holder to receive a portion of 75% of the net proceeds that may result from certain pending litigation at XOMA Royalty. “At Ligand, we are focused on building a diversified portfolio of high-value royalty assets tied to innovative medicines that have the potential to improve patient outcomes around the world,” said Todd Davis, CEO of Ligand. “Over the past several years, we have executed a disciplined strategy to expand, diversify and strengthen our royalty portfolio, and the acquisition of XOMA Royalty meaningfully advances that vision. This transaction adds exposure to high-quality commercial and development-stage assets and enhances our ability to generate durable, long-term shareholder value, marking an important milestone in our evolution as a leading biopharmaceutical royalty aggregator.” This acquisition strengthens Ligand’s royalty portfolio by adding seven commercial products, including Roche’s VABYSMO® (faricimab-svoa), Servier’s OJEMDA™ (tovorafenib), and Zevra Therapeutics’ MIPLYFFA® (arimoclomol). Additionally, it includes 14 late-stage development programs, featuring Takeda’s mezagitamab and certain assets from Takeda’s externalized asset portfolio, such as osavampator, volixibat, and OHB-607, along with more than 100 assets in various stages of development. As a result, Ligand’s portfolio has more than doubled in size, now comprising over 200 commercial, clinical, and preclinical stage royalty assets. The closing of the transaction met Ligand’s original timeline expectations. The transaction is expected to be immediately accretive and to add approximately $0.50 and $1.50 per share to Ligand’s projected 2026 and 2027 adjusted earnings per share 1 , respectively. Ligand will provide an updated 5-year outlook during the Company’s Investor Day on December 8, 2026. In connection with the completion of the transaction, XOMA Royalty common stock ceased trading on The Nasdaq Global Market. Advisors Stifel served as lead financial advisor, Citi served as financial advisor, Paul Hastings LLP served as legal advisor, and Collected Strategies served as strategic communications advisor to Ligand. Leerink Partners served as lead financial advisor, H.C. Wainwright & Co. served as financial advisor, and Gibson, Dunn & Crutcher LLP served as legal advisor to XOMA Royalty. About Ligand Ligand is a leading royalty aggregator, partnering with biopharmaceutical companies to finance and advance late-stage clinical development programs. Ligand owns and manages one of the largest and most diversified portfolios of biopharmaceutical royalties in the industry, with economic interests in more than 200 development and commercial-stage assets. Ligand funds high-value programs in exchange for long-term economic interests, aligning capital with clinical and commercial success. Ligand’s royalty portfolio is designed to deliver consistent and predictable revenue streams across a broad range of therapeutic assets. Ligand also licenses its proprietary technologies, Captisol® and NITRICIL™, to support drug development and formulation across its global partner network. For more information, visit or follow Ligand on X and LinkedIn . Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including information about, among other topics, Ligand’s acquisition of XOMA Royalty, Ligand’s and XOMA Royalty’s products pipeline and the substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, risks related to the ability to realize the anticipated benefits of the acquisition, including the possibility that the expected benefits from the acquisition will not be realized or will not be realized within the expected time period; the risk that the businesses will not be integrated successfully; the risk that XOMA Royalty stockholders may receive no payment with respect to the CVRs; disruption from the transaction making it more difficult to maintain business and operational relationships, including the ability to attract and retain highly qualified management and other clinical and scientific personnel; negative effects of this announcement or the completion of the acquisition on the market price of Ligand’s common stock and/or operating results; significant transaction costs; unknown liabilities; the risk of litigation and/or regulatory actions related to the acquisition of XOMA Royalty’s business; other business effects and uncertainties, including the effects of industry, market, business, economic, political or regulatory conditions; future exchange and interest rates; risks and uncertainties related to issued or future executive orders or other new, or changes in, laws, regulations or policy; changes in tax and other laws, regulations, rates and policies; the uncertainties inherent in business and financial planning, including, without limitation, risks related to Ligand’s business and prospects, adverse developments in Ligand’s markets, or adverse developments in the U.S. or global capital markets, credit markets, regulatory environment, tariffs and other trade policies or economies generally; future business combinations or disposals; uncertainties regarding the commercial success of commercialized and/or pipeline products; risks associated with drug development; Ligand’s reliance on collaborative partners for milestone payments, royalties, materials revenue, contract payments and other revenue projections, which may not be received; the uncertainties inherent in research and development, including the ability of Ligand’s partners to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with initial, preliminary or interim data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from the clinical trials conducted by Ligand’s partners; whether and when drug applications may be filed in any jurisdictions for pipeline products for any potential indications by Ligand’s partners; whether and when any such applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy and, if approved, whether any such products will be commercially successful; and decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of such products. You should carefully consider the foregoing factors and the other risks and uncertainties that affect the businesses of Ligand and XOMA Royalty described in the “Risk Factors” and “Cautionary Note Regarding Forward-Looking Statements” (in the case of Ligand) and “Forward Looking Statements” (in the case of XOMA Royalty) sections of their respective Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q and other documents filed by either of them from time to time with the U.S. Securities and Exchange Commission (the “SEC”), all of which are available at . These filings identify and address other important risks and uncertainties that could cause actual events and results to differ materially from those contained in the forward-looking statements. Forward-looking statements speak only as of the date they are made. Readers are cautioned not to put undue reliance on forward-looking statements, and Ligand assumes no obligation to, and does not intend to, update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise, unless required by law. Ligand gives no assurance that it will achieve its expectations. Contacts Investors: Melanie Herman investors@ligand.com (858) 550-7761 Media: Nick Lamplough/Jude Gorman Ligand-CS@collectedstrategies.com (917) 885-1013 1 The financial outlook, expectations and other forward-looking statements provided by Ligand for 2026 and beyond reflect Ligand's judgment based on the information available at the time of this release. Please see the Forward-looking Statements section in this release for factors that may impact Ligand's ability to meet expectations. In reliance upon Item 10(e)(1)(i)(B) of Regulation S-K, reconciliations of forward-looking core adjusted earnings per diluted share for 2027 is not provided because of the unreasonable effort associated with providing such reconciliations due to the variability in the occurrence and the amounts of certain components thereof. For the same reasons, we are unable to address the significance of the unavailable information, which could be material to future results.