This is a Phase Ib/II 2 study investigating the safety and efficacy of revumenib in two cohorts of participants with myelofibrosis. COHORT-1 will investigate the safety of revumenib as monotherapy in participants with myelofibrosis previously treated with a JAK inhibitor. Following confirmation of safety in COHORT-1, the study will proceed with enrollment in COHORT-2, which will evaluate the efficacy and safety of revumenib in combination with a JAK inhibitor.

Start Date01 Dec 2026

Sponsor / Collaborator

Icahn School of Medicine at Mount Sinai

[+2]

NCT05883904

/ Unknown statusNot ApplicableIIT

Observational Study Protocol REALFed - Real World Evidence of Fedratinib Effectiveness in Myelofibrosis

This is a multicenter prospective and retrospective observational clinical study in patients with primary or post polycythemia vera or post essential thrombocythemia myelofibrosis to test the efficacy of fedratinib in the rea world. Participants will be managed according to the clinical practice of the participating Center. All Centers will be Italian Hematology Units belonging to the GIMEMA Organization in Italy.

Start Date29 Jan 2024

Sponsor / Collaborator

Gruppo Italiano Malattie EMatologiche dell'Adulto

NCT06073847

/ RecruitingNot Applicable

Inrebic® (Fedratinib) Post-Marketing Surveillance in Korean Patients With Myelofibrosis

The purpose of this study is to assess the real-world safety of fedratinib for the treatment of adult participants with primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (post-PV MF), or post essential thrombocythemia myelofibrosis (post-ET MF) who were previously treated with ruxolitinib. Participants will represent the overall patient population with PMF, post-PV MF or post-ET MF who lost adequate response to and/or are intolerant to ruxolitinib. Inadequate response definitions will follow Ministry of Food and Drug Safety-approved label and reimbursement criteria of the Health Insurance Review & Assessment Service.

Start Date13 Jun 2023

Sponsor / Collaborator

Bristol Myers Squibb Co.

100 Clinical Results associated with Fedratinib Hydrochloride

100 Translational Medicine associated with Fedratinib Hydrochloride

100 Patents (Medical) associated with Fedratinib Hydrochloride

497

Literatures (Medical) associated with Fedratinib Hydrochloride

01 Dec 2026·Pharmaceutical Science Advances

Next-generation Janus kinase inhibitors: Integrating synthetic innovation, structural biology, and computational design for precision drug discovery

Review

Author: Cheriyan, Binoy Varghese ; Philiph, Sowmiya ; Karunakar, Karthik K ; Shanmugam, Rajesh Kumar ; Sree, Josme

Janus kinase (JAK) dysregulation plays a central role in the pathogenesis of inflammatory, autoimmune, and malignant disorders, making the JAK family an essential therapeutic target across multiple disease domains. Over the past two decades, the field has progressed from the identification of early JAK2 inhibitors to the approval of several first-generation agents, including ruxolitinib, tofacitinib, baricitinib, and fedratinib, which validated the clinical feasibility of JAK blockade. However, limitations related to safety, isoform selectivity, long-term tolerability, and off-target kinase interactions continue to restrict their broader application and highlight the need for next-generation molecules. In this review, we provide a comprehensive and strategic assessment of the molecular features underpinning JAK2 and JAK3 selectivity, including signaling features directly relevant to inhibitor design, mutational landscapes, and structural determinants such as the uniquely targetable Cys909 residue in JAK3. Although the JAK family comprises four kinases, this review intentionally focuses on JAK2 and JAK3, where structural divergence, disease relevance, and emerging selectivity strategies provide the strongest opportunities for next-generation precision inhibitor design. We integrate recent advances in synthetic chemistry, including hinge-binding optimization, heterocyclic diversification, multicomponent reactions, and scaffold-hopping strategies, with computational methodologies such as molecular docking, molecular dynamics simulations, QM/MM calculations, and machine-learning-based predictive modelling. Together, these multidisciplinary approaches have accelerated hit discovery, refined selectivity, and improved the pharmacokinetic and safety profiles of emerging JAK inhibitors. By consolidating progress across medicinal chemistry, structural biology, and computational design, this review outlines key opportunities and remaining challenges in developing next-generation JAK inhibitors with enhanced precision and therapeutic value for oncology, immunology, and chronic inflammatory diseases.

13 Aug 2026·JOURNAL OF MEDICINAL CHEMISTRY

Mapping Non-Homologous Pocket Compatibilities to Identify Hidden Drug-Target Relationships: A Pocket Hopping Framework

Article

Author: Zhang, Sulin ; Zeng, Chuanlong ; Yang, Ruirui ; Zhang, Yingying ; Song, Ying ; Li, Xutong ; Yang, Tianbiao ; Niu, Buying ; Lin, Jiusheng ; Tong, Xiaochu ; Zhang, Keke ; Zheng, Mingyue

Abstract:

Predicting small molecule–protein interactions across nonhomologous proteins remains challenging because shared ligand recognition is often not evident from sequence, fold, or pocket similarity. Here, we introduce pocket hopping, a machine-learning framework that learns residue-level interaction patterns from coligand binding pockets and infers compatibility between nonhomologous pockets for similar chemotypes. Using shared ligands as supervision rather than explicit geometric alignment, pocket hopping identifies pocket relationships that are not readily captured by conventional chemical-, sequence-, or structure-based comparisons. In two case studies, pocket hopping demonstrates broad utility in drug discovery by enabling de novo hit identification and mechanistic interpretation, identifying fedratinib and its analogues as helicase WRN inhibitors. The model also identified the clinical-stage HDAC inhibitor abexinostat as a direct ENPP1 binder and inhibitor, and cellular assays showed enhanced cGAMP–STING signaling under cGAMP stimulation. Together, these results indicate that pocket-level compatibility can complement existing approaches for target identification, hit discovery, and polypharmacology analysis.

01 Jul 2026·JOURNAL OF PARENTERAL AND ENTERAL NUTRITION

Adverse events associated with drug‐related thiamine deficiency: A pharmacovigilance study

Article

Author: Wang, Wenhua ; Zhou, Xin ; Ma, Zengguang ; Du, Xiaolang

Abstract:

Background:

Thiamine deficiency can cause severe complications, including beriberi, Wernicke's encephalopathy, and Korsakoff syndrome, potentially leading to irreversible neurological damage or death. While causes such as chronic alcohol use disorder are established, drug‐induced thiamine deficiency represents a potential risk that is often overlooked. However, systematic data on this association remain limited.

Methods:

We utilized relevant preferred terms from the Medical Dictionary for Regulatory Activities to identify adverse events in the United States Food and Drug Administration Adverse Event Reporting System from 2004 through 2024. Signal detection was performed using four algorithms: the reporting odds ratio, the proportional reporting ratio, the information component, and the empirical Bayes geometric mean.

Results:

We identified 1,046 cases of thiamine deficiency, showing an increasing trend. Hospitalization was reported in 46.0% of cases. The median patient age was 55.0 years (interquartile range: 41.0–68.0), and 42.5% were female. Healthcare professionals submitted 81.5% of reports, and the United States contributed the largest share (27.8%). The five agents most frequently reported were furosemide, metformin, fedratinib, metronidazole, and fluorouracil. Overall, 38 drugs demonstrated consistently positive safety signals across all four methods. Additional drugs exhibiting notable signals included arsenic trioxide, torasemide, rabeprazole, and hydrochlorothiazide.

Conclusion:

This pharmacovigilance analysis identifies safety signals linking specific medications with thiamine deficiency, potentially mediated by mechanisms such as transport inhibition and metabolic interference. These findings highlight the importance of clinical vigilance and the consideration of thiamine monitoring in high‐risk patients to mitigate the risk of neurological complications.

News (Medical) associated with Fedratinib Hydrochloride

24 Mar 2026

·allsci.com

Karyopharm’s sNDA for Xpovio in myelofibrosis uncertain after mixed Phase III data

Karyopharm Therapeutics (Nasdaq: KPTI) reported topline results from its Phase III SENTRY trial in myelofibrosis, showing that Xpovio (selinexor) combined with ruxolitinib nearly doubled the rate of spleen volume reduction compared to ruxolitinib alone, though the combination failed to separate from the comparator on symptom improvement. The mixed outcome on co-primary endpoints sets up a complex regulatory discussion for the Newton, Massachusetts-based company, which plans to meet with the FDA to explore a potential supplemental new drug application. Trial specifics The SENTRY trial (XPORT-MF-034; NCT04562389) is a randomized, double-blind, placebo-controlled Phase III study that enrolled 353 JAK inhibitor–naïve myelofibrosis patients with platelet counts above 100 × 10⁹/L. Patients were randomized 2:1 to receive 60 mg selinexor once weekly plus ruxolitinib or placebo plus ruxolitinib. The trial had two co-primary endpoints: spleen volume reduction of 35% or more (SVR35) at week 24, and mean change in absolute total symptom score (Abs-TSS) over 24 weeks relative to baseline. Data are reported as of a February 20, 2026 cut-off. On the first co-primary endpoint, 50% of patients in the combination arm achieved SVR35 at week 24 compared to 28% with ruxolitinib alone (one-sided p<0.0001). Spleen responses appeared early, with 49% of combination-arm patients reaching SVR35 by week 12 versus 20% in the control arm, and were sustained at week 36 (47% versus 23%). On the second co-primary endpoint, however, the trial did not reach statistical significance: patients receiving selinexor plus ruxolitinib reported a 9.89-point improvement in Abs-TSS versus a 10.86-point improvement with ruxolitinib alone. Both arms achieved similar symptom relief relative to baseline, meaning the addition of selinexor did not confer a detectable symptom benefit over ruxolitinib monotherapy. An exploratory overall survival analysis yielded a hazard ratio of 0.43 (95% CI [0.19, 1.00]; nominal one-sided p=0.0222), suggesting a reduction in risk of death with the combination. The company noted these data are immature and intends to continue follow-up. A pre-specified exploratory endpoint examining variant allele frequency (VAF) reduction showed 32% of combination-arm patients achieving 20% or greater VAF reduction for driver mutations (JAK2, MPL, CALR) at week 24, compared to 24% with ruxolitinib alone (n=261). Other secondary endpoints, including progression-free survival, hemoglobin stabilization, and bone marrow fibrosis improvement, showed no meaningful difference between arms at the data cut-off. The safety profile was consistent with the known effects of both agents. Development context Selinexor is a first-in-class oral inhibitor of exportin 1 (XPO1), a nuclear transport protein responsible for shuttling tumor suppressor proteins and growth regulators out of the nucleus. By blocking XPO1, selinexor forces retention of these proteins in the nucleus, restoring their function and inducing cell death in malignant cells. This mechanism is distinct from JAK pathway inhibition, and the rationale for combining selinexor with ruxolitinib rests on targeting complementary biological pathways in myelofibrosis. The molecule was first approved in 2019 for relapsed/refractory multiple myeloma, and in 2020 for relapsed or refractory diffuse large B-cell lymphoma (DLBCL). The spleen volume reduction rate of 50% at week 24 in the combination arm compares to historical SVR35 rates for ruxolitinib monotherapy observed in the COMFORT trials, though the 28% rate in SENTRY’s control arm fell below those benchmarks. Cross-trial comparisons are limited by differences in study design, patient selection, and duration. Within the SENTRY trial itself, the internal comparison is more interpretable, and the near-doubling of SVR35 at week 12 (49% versus 20%) suggests the combination accelerates spleen response. Whether this translates into durable clinical benefit remains to be established as survival and other endpoints mature. The myelofibrosis treatment landscape remains anchored by JAK inhibitors. Ruxolitinib, approved by the FDA in 2011, is the standard frontline therapy. Fedratinib, pacritinib, and momelotinib have since received approval, each addressing specific patient subpopulations defined by platelet counts or anemia burden. Definitive randomized frontline OS benefit remains difficult to establish in MF, particularly given crossover in prior studies. That means the immature OS signal from SENTRY is a point of interest, though the wide confidence interval spanning 1.00 warrants caution. The field has seen several combination strategies struggle to show clear added benefit over JAK inhibitor monotherapy The regulatory path forward for Karyopharm is uncertain. Meeting one co-primary endpoint while missing the other creates ambiguity, and the FDA’s willingness to consider an sNDA based on SVR35 alone, alongside immature survival data, will depend on the agency’s assessment of the totality of evidence. The company stated it plans to present additional data at an upcoming medical meeting and submit a manuscript for peer-reviewed publication, with potential compendia inclusion anticipated in the second half of 2026. Leave a Reply Cancel reply Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

Clinical ResultPhase 3Drug Approval

04 Mar 2026

·allsci.com

Sanofi acquires global rights to rovadicitinib from Sino Biopharmaceutical in up to USD 1.53b deal

Chia Tai Tianqing Pharmaceutical Group Co., Ltd (CTTQ), a subsidiary of Hong Kong-listed Sino Biopharmaceutical Limited (HKEX: 1177), entered into an exclusive license agreement with French giant Sanofi S.A. (Euronext: SAN). The deal confers global development, manufacturing, and commercialization rights to Sanofi in relation to Chia Tai’s rovadicitinib, a first-in-class oral small-molecule JAK/ROCK dual-target inhibitor, a molecule already approved and marketed in China under the brand name Anxu. The deal carries a total potential value of up to USD 1.53 billion, with Sino Bio eligible to receive an upfront payment of USD 135 million, plus development, regulatory, and sales milestone payments of up to USD 1.395 billion. Sanofi will also pay up to double-digit tiered royalties on annual net sales. Deal context Rovadicitinib is a dual inhibitor of the JAK/STAT and ROCK signaling pathways. By targeting JAK, the molecule suppresses inflammatory cytokine signaling from myeloid cells. Concurrent ROCK inhibition modulates STAT3/STAT5 phosphorylation, downregulating overactivated Th17 cells and enhancing regulatory T cell function to restore immune homeostasis. This dual mechanism produces synergistic anti-inflammatory and anti-fibrotic effects. Chi Tai secured a first approval for rovadicitinib in China in February 2026, indicated as a first-line treatment for adult patients with intermediate-2 or high-risk primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF). In chronic graft-versus-host disease (cGVHD), the molecule has advanced to Phase III in China, where it received Breakthrough Therapy Designation from the CDE in August 2025. In the US, rovadicitinib has been cleared to conduct Phase II clinical studies in cGVHD. Phase Ib/IIa data published in Blood demonstrated superior 12-month failure-free survival and enhanced responses in fibrosis-dominated organs compared to other approved therapies, along with potential to overcome ruxolitinib resistance. For Sanofi, the deal is the latest in a string of in-licensing from Chinese innovators, including an August 2022 collaboration with Innovent Biologics on oncology assets and a March 2022 agreement with Adagene valued at up to approximately USD 2.5 billion for masked antibody technology. Sanofi also acquired Kadmon Holdings in 2021, gaining belumosudil (Rezurock), an approved selective ROCK2 inhibitor for cGVHD. The addition of rovadicitinib, a JAK/ROCK inhibitor, complements belumosudil’s ROCK-only mechanism in the same indication. The rovadicitinib JAK ROCK inhibitor enters a competitive landscape shaped by several approved agents. In myelofibrosis, Incyte’s ruxolitinib (Jakafi), a JAK1/JAK2 inhibitor licensed to Novartis for ex-US rights under a 2009 deal, remains the standard of care. Bristol Myers Squibb markets fedratinib (Inrebic), a JAK2 inhibitor, and CTI BioPharma’s pacritinib, acquired by SOBI in 2023, targets JAK2/FLT3 in patients with thrombocytopenia. In cGVHD specifically, Sanofi’s own belumosudil and Incyte’s ruxolitinib hold approvals. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

Drug ApprovalLicense out/inPhase 2Breakthrough TherapyAcquisition

17 Feb 2026

·www.biospace.com

RECORDATI REPORTS STRONG PRELIMINARY FULL YEAR 2025 RESULTS: REVENUE +11.8%, EBITDA(1) +14.5%, ADJUSTED NET INCOME(2) +14.5%

Consolidated net revenue of € 2,618.4 million for full year 2025, +11.8% or +8.3% on a like-for-like basis (3) at constant exchange rates (CER) EBITDA (1) of € 991.1 million, +14.5%, margin on net revenue of 37.8% Adjusted net income (2) of € 651.1 million, +14.5%, margin on net revenue of 24.9% Net income of € 443.6 million, +6.5% Free cash flow (4) of € 558.8 million, +€ 23.7 million reflecting strong EBITDA partially offset by U.S. inventory build-up, higher interests and income tax paid Net debt (5) at € 2,037.3 million, just below 2.1x EBITDA FY 2026 financial targets: Net revenue € 2,730-€2,800 million with FX headwind of ~-3.5% ; EBITDA (1) € 995-€1,030 million, margin +/- 36.5% ; Adjusted net income (2) € 655-€ 685 million, margin +/- 24.0% In January 2026, the Group received a B‑ rating with ‘Prime’ status from ISS ESG, recognizing its leading sustainability performance within the industry Milan, February 17, 2026 – The Board of Directors of Recordati S.p.A. has reviewed and approved the preliminary consolidated financial statements for 2025. The Group’s final consolidated annual financial statements for 2025 will be submitted to the Board of Directors for approval on March 19, 2026. Rob Koremans, Chief Executive Officer of Recordati, commented: “2025 was another year of solid progress across the business, reflecting the strength of our execution. We delivered once again on our financial targets despite a challenging macroenvironment, including increased FX headwinds. During the year, we further strengthened our portfolio through strategic partnerships in both Rare Diseases and Specialty and Primary Care. There is excellent momentum in Rare Diseases, which continues to be a key driver of growth and value creation for the Group. We are excited by Isturisa’s opportunity to address the broader Cushing’s syndrome market, with uptake accelerating in the U.S. With such a strong foundation in place, we expect 2026 to be another year of disciplined execution as we continue to deliver on our strategic objectives, maintain sector-leading margins and create sustainable value for all our stakeholders.” Financial highlights Consolidated net revenue for full year 2025 was € 2,618.4 million, up 11.8% versus full year 2024 or 8.3% on a like-for-like (3) basis at CER, driven by solid contribution from both Specialty & Primary Care and Rare Diseases. The adverse FX impact for full year 2025 was € 64.2 million (-2.7%). Specialty & Primary Care revenue was € 1,477.9 million for full year 2025, up 2.0% or 3.8% on a like-for-like basis (3) at CER (+1.6% excluding Türkiye). This reflects continued positive performance of all core therapeutic areas (promoted product evolution index of 105), despite a slight slowdown in relevant market growth. In particular, the Urology and Cardiovascular franchises grew 2.5% and 2.8%, respectively, while the Gastrointestinal franchise grew 9.9%, driven by the strong in-market performance of several products in the portfolio, both prescription and OTC. Rare Diseases revenue was € 1,081.4 million for full year 2025, up 29.7% as compared to full year 2024, or 16.6% on a like-for-like (3) basis at CER, driven by strong volume growth across all three franchises. The Endocrinology franchise achieved net revenue of € 394.1 million, an increase of 22.5%, reflecting an acceleration of new patient uptake of Isturisa ® in the U.S. in the second half of 2025 with approximately 1,400 net active patients at the end of the year, as well as double-digit growth of Signifor ® . The Hema-Oncology franchise achieved net revenue of € 414.9 million, growing by 63.8%, reflecting the contribution of Enjaymo ® of € 146.3 million (+26.7% vs full year 2024 proforma (6) ), and driven by strong growth of Sylvant ® and Qarziba ® . The Metabolic franchise achieved net revenue of € 272.5 million, sustaining mid-single digit growth of 5.2%, driven by Carbaglu ® and Panhematin ® . Adjusted operating income (7) was € 774.9 million for full year 2025, up 13.2% over full year 2024, and 29.6% of net revenue versus 29.2% in the previous year. Operating income was € 670.8 million for full year 2025, up 5.0% over full year 2024, absorbing gross margin-related non-cash charges of € 66.8 million as compared to € 37.5 million for full year 2024, arising from the unwind of the fair value step up of acquired Rare Diseases inventory including € 62.5 million for Enjaymo ® . Non-recurring costs were € 37.3 million for full year 2025, versus € 8.0 million for full year 2024. These costs reflect primarily the continued optimization of the Specialty and Primary Care commercial organization, mainly in Italy and Spain. The non‑recurring costs also include a one‑off provision of €12.8 million (8) related to the settlement of a litigation case with AIFA concerning prior years payback for Urorec ® . Additionally, non‑recurring items include the impact of the ongoing voluntary liquidation of the Rare Diseases subsidiary in China, following the rejection of the National Reimbursement Drug List approval for Isturisa ® . The availability of Qarziba ® and Sylvant ® in the territory continues to be provided through a local distributor. EBITDA (1) was € 991.1 million for full year 2025, up 14.5% compared to full year 2024, with margin on net revenue of 37.8%. The improvement over the prior year was driven by a positive business mix and strong operating performance across both business units, despite the significant foreign exchange headwinds and higher investments to support the U.S. launch of the expanded Isturisa ® label, the continued development of Enjaymo ® and ongoing geographic expansion in Rare Diseases. Financial expenses were € 89.5 million for full year 2025, down by € 2.1 million as compared to full year 2024. New loans obtained in 2024, related to the acquisition of Enjaymo ® , and in 2025 led to an increase in interest expenses of € 17.4 million. Net exchange gains over the period were € 15.0 million (mainly unrealized and driven by the devaluation of the U.S. dollar), against net FX losses of € 9.3 million in FY 2024. This was partly offset by € 5.3 million of net monetary losses from hyperinflation accounting (compared to a loss of € 6.7 million in full year 2024) mainly driven by the net effect of the revaluation of Turkish balance sheet items. Adjusted Net Income was € 651.1 million, 24.9% of revenue, up by 14.5% compared to full year 2024. This growth reflects improvements in adjusted operating income as well as lower financial expenses partially offset by higher income taxes. Net income was € 443.6 million, 16.9% of revenue, increasing by 6.5% versus full year 2024, mainly driven by the higher operating income and lower financial expenses. Free cash flow (4) was € 558.8 million for full year 2025, an increase of € 23.7 million versus full year 2024, with strong EBITDA partially offset by higher working capital absorption (mainly driven by higher U.S. inventory levels), higher interests and income tax paid. Net debt (5) as of December 31, 2025 was € 2,037.3 million, or leverage of just below 2.1x EBITDA, compared to net debt of € 2,154.3 million on December 31, 2024, following dividend payments of € 267.6 million, treasury shares purchased for € 112.5 million (net of proceeds from exercising stock options), the upfront payment for Vazkepa ® rights of USD 25 million and the upfront payment for Inrebic ® rights of USD 11 million. Shareholders’ equity was € 1,919.8 million. Pipeline Update On April 15, 2025, the U.S. Food and Drug Administration (FDA) approved the supplemental new drug application (sNDA) for Isturisa ® (osilodrostat) for the treatment of endogenous hypercortisolemia in adults with Cushing’s syndrome for whom surgery is not an option or has not been curative. This was an expansion of the previous indication for the treatment of patients with Cushing’s disease, which is a sub-type of Cushing’s syndrome. The Isturisa ® indication expansion was supported by the extensive Isturisa ® clinical development program, which included over 350 patients. In addition, during the second quarter of 2025, Isturisa ® was granted regulatory approval in both Canada and Russia. A Phase IV study to assess the efficacy and safety of osilodrostat in adults with mild hypercortisolemia and uncontrolled hypertension (HTN) due to Cushing’s syndrome is expected to start in 2026. During the second quarter of 2025, an investigator-sponsored clinical trial (IST) was initiated to investigate the safety, dose and early signs of effect for dinutuximab beta (Qarziba ® ) in combination with chemotherapy for the treatment of patients with GD2-positive Ewing sarcoma. On July 28, 2025, the European Commission issued a positive decision and granted marketing authorization, under exceptional circumstances, for Maapliv ® , a solution of amino acids intended for the treatment of maple syrup urine disease (MSUD) presenting with an acute decompensation episode in patients from birth who are not eligible for an oral and enteral branched-chain amino acids (BCAA)-free formulation. The Company completed enrollment of the pasireotide Phase 2 trial for the treatment of post-bariatric hypoglycemia in August 2025. Top-line results are expected in the second quarter of 2026. Following the meeting with the U.S. Food and Drug Administration (FDA) in early September, a potential U.S. biologics license application (BLA) pathway was established with the FDA for Qarziba ® requiring an additional set of clinical data from the ongoing BEACON-2 investigator-sponsored trial. Results of the interim analysis are expected in the first half of 2028 and are expected to form the basis, together with existing clinical data, for a potential regulatory filing. On January 5, 2026, the UK Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorization for Eligard ® for the treatment of hormone dependent advanced prostate cancer and for the treatment of high-risk localized and locally advanced hormone dependent prostate cancer in combination with radiotherapy. The other lifecycle management programs are progressing in line with plans. Corporate Development On June 24, 2025, Recordati announced a licensing and supply agreement with Amarin to commercialize the marketed cardiovascular medicine, Vazkepa ® (icosapent ethyl) across 59 countries, focused in Europe. Vazkepa ® is indicated to reduce the risk of cardiovascular events in statin-treated adult patients at high cardiovascular risk with elevated triglycerides and either established cardiovascular disease or diabetes with at least one other cardiovascular risk factor. Vazkepa ® is expected to achieve over € 40 million in revenues in 2027 and to be EBITDA positive from 2026. Under the terms of the agreement, Recordati paid Amarin an upfront cash payment of USD 25 million. On December 17, 2025, Recordati announced the exclusive license agreement with Impact Biomedicines, Inc., a Bristol Myers Squibb subsidiary, and the related supply agreement with Celgene Logistics Sàrl to commercialize Inrebic ® (fedratinib dihydrochloride monohydrate) in Japan. Impact Biomedicines, Inc. will retain exclusive rights to develop and commercialize Inrebic® in the rest of the world. Inrebic ® is an oral kinase inhibitor with activity against wild-type and mutationally activated JAK2 to suppress the pathological features of myelofibrosis patients. Inrebic ® received regulatory approval from the Ministry of Health, Labour and Welfare (MHLW) in Japan in June 2025 for the treatment of myelofibrosis and is expected to launch in mid 2026. Under the terms of the agreement, Recordati paid Impact Biomedicines, Inc. an upfront payment of USD 11 million. On January 29, 2026, Recordati announced a collaboration and license agreement with Moderna to develop and commercialize worldwide mRNA-3927, an investigational product for the treatment of propionic acidemia (PA). Under the terms of the agreement, Moderna will continue to lead the development of mRNA-3927, in collaboration with Recordati, and if approved, Recordati will lead global commercialization. mRNA-3927 is a post proof-of-concept, investigational product aimed to restore propionyl-CoA carboxylase (PCC) enzyme activity in patients with propionic acidemia. If approved, this could be the first disease-modifying treatment option on the market for this severe disease. mRNA-3927 is currently being evaluated in a potential registrational clinical study. The target patient enrollment has been reached, with a potential data readout expected by the end of 2026. Under the terms of the agreement, Recordati will pay Moderna an upfront payment of USD 50 million and up to an additional USD 110 million in near-term development and regulatory milestones. Moderna is also eligible to receive commercial and sales milestones, as well as tiered royalties on annual net sales. Recordati does not expect any significant impact on its EBITDA prior to a potential launch. Business outlook The financial targets for full year 2026 are as follows: Net revenue between € 2,730 and € 2,800 million with FX headwind of ~-3.5% EBITDA (1) between € 995 and € 1,030 million; margin of +/- 36.5% Adjusted net income (2) between € 655 and € 685 million; margin of +/- 24.0% The full year 2027 targets (9) remain unchanged, with strong organic growth complemented by bolt-on BD and M&A. (1) Net income before income taxes, financial income and expenses, depreciation, amortization and write-downs of property, plant and equipment, intangible assets and goodwill, non-recurring items and non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS (2) Net income excluding amortization and write-downs of intangible assets (except software) and goodwill, non-recurring items, non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3, monetary net gains/losses from hyperinflation (IAS 29), net of tax effects. (3) Proforma growth calculated excluding revenue of Vazkepa® for FY 2025 (Specialty & Primary Care) and Enjaymo® for both FY 2025 and FY 2024 (Rare Diseases) (4) Total cash flow excluding financing items, milestones, dividends, purchases of treasury shares net of proceeds from exercise of stock options. (5) Cash and cash equivalents, less bank debts and loans, which include the measurement at fair value of hedging derivatives. (6) Comparing FY 2025 revenue (which considers also the margin retained by Sanofi’s on in market sales for those countries where it was still holding the MA) with proforma FY 2024 revenue also including sales totally realized by Sanofi. (7) Net income before income taxes, financial income and expenses and non-recurring items, non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3. (8) The provision has been revised since September to reflect the terms of the final settlement agreement with AIFA (9) FY 2027 targets: Net Revenue €3,000 - €3,200 million, EBITDA €1,140 - €1,225 million, Adjusted Net Income €770- €820 million, excluding potential impact from tariffs and/or most favored nation pricing policies in the U.S. Conference Call Recordati will host a conference call on February 18 th , at 2:00 p.m. CET ( 1:00 p.m. GMT ) to present the results for full year 2025. Please find the pre-registration link here with all the dial-in details and a calendar invitation to follow. Alternatively, if not pre-registered, the dial-in numbers for the conference call are: Italy + 39 02 802 09 11, toll free 800 231 525 UK + 44 1 212818004, toll free (44) 0 800 0156371 USA +1 718 7058796, toll free (1) 1 855 2656958 France +33 1 70918704 Germany +49 6917415712 Participants are invited to dial in 10 minutes before the start of the conference call. If operator assistance is required to connect, please dial *0. The slides that will be referenced during the call will be available at under Investors/Company Presentations. Recordati is an international pharmaceutical group listed on the Italian Stock Exchange (XMIL: REC), with roots dating back to a family-run pharmacy in Northern Italy in the 1920s. We are uniquely structured to provide treatments across specialty and primary care, and rare diseases. Our fully integrated operations span clinical development, chemical and finished product manufacturing, commercialization and licensing. We operate in approximately 150 countries across EMEA, the Americas and APAC with over 4,500 employees. We believe that health is a fundamental right, not a privilege. Today, our purpose of “unlocking the full potential of life” aims at empowering individuals to live life to the fullest, whether addressing common health challenges or the rarest. Investor Relations Eugenia Litz eugenia.litz@recordati.com Gianluca Saletta saletta.g@recordati.it Media Relations Laura Conti conti.l@recordati.it Rebecca Kerr rebecca.kerr@recordati.com This document contains forward-looking statements relating to future events and future operating, economic and financial results of the Recordati group. By their nature, forward-looking statements involve risk and uncertainty because they depend on the occurrence of future events and circumstances. Actual results may therefore differ materially from forecasts for a variety of reasons, most of which are beyond the Recordati group ’ s control. The information on the pharmaceutical specialties and other products of the Recordati group contained in this document is intended solely as information on the activities of the Recordati Group, and, as such, it is not intended as a medical scientific indication or recommendation, or as advertising . DECLARATION BY THE FINANCIAL REPORTING OFFICER The Financial Reporting Officer, Niccolò Giovannini, declares, pursuant to paragraph 2 of Article 154- bis of the Consolidated Law on Finance, that the accounting information contained in this press release corresponds to the Company’s documentation, books and accounting records. RECORDATI INDUSTRIA CHIMICA E FARMACEUTICA S.p.A. Registered Office Via Matteo Civitali, 1 20148 Milano, Italy Tel. +39 02 487871 Fax +39 02 40073747 Share Capital € 26.140.644,50 fully paid-up Milano, Monza, Brianza and Lodi Comp. Reg. No. 00748210150 Tax Code/VAT No. 00748210150 Milano R.E.A. No. 401832 Company subject to the Management and Coordination Activity of Rossini Luxembourg S.àr.l

License out/inDrug ApprovalPhase 2Financial Statement

100 Deals associated with Fedratinib Hydrochloride

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KEGG	Wiki	ATC	Drug Bank
-	Fedratinib Hydrochloride	L01XE57	DB12500

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Post-essential thrombocythemia myelofibrosis	European Union	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-essential thrombocythemia myelofibrosis	Iceland	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-essential thrombocythemia myelofibrosis	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-essential thrombocythemia myelofibrosis	Norway	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-polycythemia vera myelofibrosis	European Union	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-polycythemia vera myelofibrosis	Iceland	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-polycythemia vera myelofibrosis	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-polycythemia vera myelofibrosis	Norway	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Primary Myelofibrosis	European Union	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Primary Myelofibrosis	Iceland	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Primary Myelofibrosis	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Primary Myelofibrosis	Norway	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Splenomegaly	European Union	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Splenomegaly	Iceland	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Splenomegaly	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Splenomegaly	Norway	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Myelofibrosis	United States	Bristol Myers Squibb Co.	16 Aug 2019
Polycythemia Vera	United States	Bristol Myers Squibb Co.	16 Aug 2019
Thrombocythemia, Essential	United States	Bristol Myers Squibb Co.	16 Aug 2019

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hematologic Neoplasms	Phase 3	United States	Bristol Myers Squibb Co.	31 Dec 2011
Hematologic Neoplasms	Phase 3	Australia	Bristol Myers Squibb Co.	31 Dec 2011
Hematologic Neoplasms	Phase 3	Austria	Bristol Myers Squibb Co.	31 Dec 2011
Hematologic Neoplasms	Phase 3	Belgium	Bristol Myers Squibb Co.	31 Dec 2011
Hematologic Neoplasms	Phase 3	Brazil	Bristol Myers Squibb Co.	31 Dec 2011
Hematologic Neoplasms	Phase 3	Canada	Bristol Myers Squibb Co.	31 Dec 2011
Hematologic Neoplasms	Phase 3	France	Bristol Myers Squibb Co.	31 Dec 2011
Hematologic Neoplasms	Phase 3	Germany	Bristol Myers Squibb Co.	31 Dec 2011
Hematologic Neoplasms	Phase 3	Hungary	Bristol Myers Squibb Co.	31 Dec 2011
Hematologic Neoplasms	Phase 3	Ireland	Bristol Myers Squibb Co.	31 Dec 2011

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01437787 (JAKARTA, CTgov)	Phase 3	Mycosis Fungoides \| Post-essential thrombocythemia myelofibrosis \| Hematologic Neoplasms ... View more	289	SAR302503 (SAR302503 400 mg)	tiwwkjship = ilaojrviia lyeftrhusz (zfbdknhvqq, arsofvtcfv - xmdahkwoqt) View more	-	11 Aug 2026
NCT01437787 (JAKARTA, CTgov)	Phase 3		289	SAR302503 (SAR302503 500 mg)	tiwwkjship = xczvppwlps lyeftrhusz (zfbdknhvqq, wlfrsjmotq - eoxzmznogq) View more	-	11 Aug 2026
NCT01523171 (JAKARTA2 \| JAKARTA-2, CTgov)	Phase 2	Mycosis Fungoides \| Post-essential thrombocythemia myelofibrosis \| Hematologic Neoplasms ... View more	97	Fedratinib	qkxnyagdat = pwgcntmcao hqgaidibau (lqfignvfmp, mprsrdqaid - uvzeyfajqm) View more	-	16 Jul 2026
NCT05177211 (ASCO2026)	Phase 2	Leukemia, Neutrophilic, Chronic \| Myeloproliferative Disorders	25	Fedratinib 400 mg	znedjldyal(pqxxmfrkee) = iqakfanvyv nxdkhayann (xlbdrxldgf ) View more	Positive	29 May 2026
FRACTION (EHA2026)	Phase 2	Myelofibrosis	30	Fedratinib + Nivolumab	xncqleotkr(syrilwbzul) = occurring in >25% of pts were dyspnea (57%), diarrhea (43%), anemia (40%), nausea (40%), fatigue (37%), edema limbs (27%) and creatinine increase (27%). jcbpoxuxst (sukpnncuwf ) View more	Positive	12 May 2026
NCT05177211 (CTgov)	Phase 2	Myelodysplastic-Myeloproliferative Diseases \| Leukemia, Neutrophilic, Chronic \| BCR-ABL Negative Atypical Chronic Myeloid Leukemia ... View more	25	Fedratinib Pill	psfqbwyzxm = rzeinjlvvg uvxtyaibar (mrsoifixxv, zbqbjcukto - evpgnhmzbz) View more	-	17 Apr 2026
ISRCTN88102629 (FEDORA, ASH2025)	Phase 2	Myelofibrosis JAK2V617F positive	34	Fedratinib 400mg + Ropeginterferon Alfa-2b	wzjbuhtdqp(rwgjvbnznd) = sfgrniddxw iareixmwlz (vpbvuezvjk ) View more	Positive	06 Dec 2025
FRACTION (ASH2025)	Phase 2	Myelofibrosis	30	FedratinibNivolumabb + FedratinibNivolumab	vuoofvurzd(gbgiqlgcrq) = xrtomrpkpi vsiqaljwsz (eqjhpwqglv ) View more	Positive	06 Dec 2025
ASCO2025	Not Applicable	Myelofibrosis JAK2 \| FLT3	960	Fedratinib after Ruxolitinib	rwokwndsyi(ohpxdefmwg) = 31.2% (C.I. 0.27-0.36, p < 0.001) tiluxpccly (wbybdjajoo ) View more	Positive	30 May 2025
ASCO2025	Not Applicable	Myelofibrosis JAK2 \| FLT3	960	Ruxolitinib alone		Positive	30 May 2025
NCT03952039 (FREEDOM2, Pubmed \| Lancet Haematol) Manual	Phase 3	Myelofibrosis Second line	201	Fedratinib 400 mg per day	gktlauamtb(oyytysultp) = rmptxqvabd pifshymimj (dslpyjcymb ) View more	Positive	01 Sep 2024
NCT03952039 (FREEDOM2, Pubmed \| Lancet Haematol) Manual	Phase 3	Myelofibrosis Second line	201	Best Available Therapy (BAT)	gktlauamtb(oyytysultp) = xuwfkzlkhl pifshymimj (dslpyjcymb ) View more	Positive	01 Sep 2024
NCT03755518 (FREEDOM, Pubmed \| Leuk Lymphoma)	Phase 3	Myelofibrosis	38	Fedratinib	uvzdljjcel(cspywnfhfm) = lallzersco hdilakregs (vtnyjfvdrs, 12.5 - 43.3) View more	Positive	28 Jul 2024

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