Novartis Pharma AG: Novartis to feature new PNH and CML scientific data from broad hematology portfolio at European Hematology Association Annual Meeting

• Updated iptacopan data confirm hemolysis control in paroxysmal nocturnal hemoglobinuria (PNH) patients from Phase III APPLY-PNH and APPOINT-PNH studies

• Head-to-head data from Phase III ASCEMBL trial reaffirm Scemblix superiority over Bosulif* in patients with chronic myeloid leukemia (CML) in chronic phase, previously treated with two or more tyrosine kinase inhibitors

• Preliminary global results from large-scale Survey on Unmet Needs in CML disrupt notion that CML is solved and underscore importance of the patient voice in treatment decisions that balance quality of life, efficacy and tolerability goals

Basel, May 11, 2023 - Novartis will present new data across its hematology portfolio at the upcoming European Hematology Association (EHA) 2023 Hybrid Congress, with 40 accepted abstracts, including updated results from pivotal trials for iptacopan and Scemblix^®.

"Further scientific advancement is crucial to fulfilling unmet treatment needs for patients living and dealing with daily challenges from devastating cancers and blood disorders," said Jeff Legos, Executive Vice President, Global Head of Oncology and Hematology Development at Novartis. "Set against the backdrop of our established hematology legacy, we look forward to presenting updated trial results in key areas such as CML and PNH, at EHA."

Key highlights of data accepted by EHA include:

Medicine?/ Therapeutic area	Abstract Title?	Abstract Number/ Presentation Details??
Iptacopan (LNP023)	Oral Iptacopan Monotherapy Increases Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell Clone Size via Control of Intra- and Extravascular Hemolysis in Anti-C5-Treated PNH Patients With Anemia	Abstract S182 Oral Presentation Friday, June 9, 3:15 - 3:30 PM CEST
Iptacopan (LNP023)	Substantial Increases in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell Clone Size With Oral Iptacopan Monotherapy Confirms Control of Hemolysis in Complement Inhibitor-Naïve PNH Patients	Abstract P774 Poster Presentation Friday, June 9, 6:00 - 7:00 PM CEST
Scemblix® (asciminib)	Rapid and Deep Responses With Asciminib in Patients (Pts) with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) After =2 Prior Tyrosine Kinase Inhibitors (TKIs) in the Phase 3 ASCEMBL Study	Abstract P665 Poster Presentation Friday, June 9, 6:00 - 7:00 PM CEST
Jakavi® (ruxolitinib)	Ruxolitinib in Pediatric Patients With Treatment-Naive or Steroid Refractory Chronic Graft versus Host Disease: Primary Findings From the Phase II REACH 5 Study	Abstract S245 Oral Presentation Saturday, June 10, 11:30 AM - 12:45 PM CEST
Paroxysmal nocturnal hemoglobinuria	Hospitalization in Patients With Paroxysmal Nocturnal Hemoglobinuria: A Retrospective Analysis of Observational Study Data From the United States	Abstract P796 Poster Presentation Friday, June 9, 6:00 - 7:00 PM CEST
Chronic myeloid leukemia	Chronic Myeloid Leukemia Survey on Unmet Needs (CML SUN): Balancing Tolerability and Efficacy Goals of Patients and Physicians Through Shared Treatment Decision-making	Abstract P668 Poster Presentation Friday, June 9, 6:00 - 7:00 PM CEST
Immune thrombocytopenia	Patient (PT) and Physician (MD) Perceptions of the Burden of Immune Thrombocytopenia (ITP) and its Management: Results From the ITP World Impact Survey (I-WISH) 2.0	Abstract P1589 Poster Presentation Friday, June 9, 6:00 - 7:00 PM CEST

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*BOSULIF^® (bosutinib) is a registered trademark of Pfizer.

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