Pacritinib

Sobi's presentations highlight research across multiple rare diseases in haematology including PNH, ITP, HLH and myelofibrosis. STOCKHOLM, June 11, 2026 /PRNewswire/ -- Sobi® (STO: SOBI) today announced that new clinical and real-world data will be presented at the 2026 Congress of the European Hematology Association (EHA2026), taking place June 11–14 in Stockholm, Sweden. The presentations include data across paroxysmal nocturnal haemoglobinuria (PNH), immune thrombocytopenia (ITP), primary haemophagocytic lymphohistiocytosis (HLH), and myelofibrosis. The data include real-world evidence and post hoc analyses for Aspaveli® (pegcetacoplan), Doptelet® (avatrombopag), Gamifant® (emapalumab), and Vonjo® (pacritinib). "As EHA comes to Stockholm this year, we are pleased to present new clinical and real-world evidence spanning multiple rare diseases," said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs and Chief Medical Officer at Sobi. "These presentations reflect the breadth of our ongoing research across PNH, ITP, HLH and myelofibrosis, and continue to build understanding of treatment outcomes in areas of high unmet need." About Aspaveli®/Empaveli® (pegcetacoplan) Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Aspaveli/Empaveli is approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the United States, European Union, and other countries globally. It is the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older approved in the United States, European Union, and other countries globally. About the Sobi® and Apellis (now part of Biogen) collaboration Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan. Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About Doptelet® (avatrombopag) Doptelet® is an orally administered thrombopoietin receptor agonist (TPO-RA) that increases platelet count. It is approved in the United States, European Union and Japan for thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure, and for ITP in adults according to local labeling. In the United States, Doptelet® is also approved for pediatric patients 1 year and older with persistent or chronic ITP who have had an insufficient response to a previous treatment. About Gamifant (emapalumab) Gamifant is the only approved anti-interferon gamma (IFNy) monoclonal antibody. Gamifant works by binding to and neutralising interferon gamma (IFNy). When interferon gamma (IFNy) is secreted in an uncontrolled manner, hyperinflammation occurs within the body. Gamifant is indicated for administration through intravenous infusion over one hour. Gamifant is approved in the US for the treatment of adult and paediatric patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Gamifant is also approved in the US for the treatment of adult and paediatric patients with haemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still's disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS . About Vonjo (pacritinib) Vonjo is a kinase inhibitor that is indicated in the US for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 2 000 employees across Europe, North America, the Middle East, Asia and Australia. In 2025, revenue amounted to SEK 28 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. Visiting address Norra Stationsgatan 93A, Stockholm +46 8 697 20 00 [email protected] This information was brought to you by Cision . The following files are available for download: 21% more press release views with Request a Demo

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