CHMP Adopted Positive Opinion for ELZONRIS (tagraxofusp), for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

The Menarini Group, a privately held Italian pharmaceutical and diagnostics company, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion on the approval of ELZONRIS (tagraxofusp) as monotherapy for the first-line treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an aggressive hematologic malignancy with dismal outcomes. The positive opinion from the CHMP was based on the largest prospective clinical trial ever conducted in patients with treatment-naïve or previously-treated BPDCN.

The CHMP opinion will now be reviewed by the European Commission, which has the authority to grant marketing authorization for human medicines throughout the European Union (EU). If approved, ELZONRIS will be the first and only treatment for BPDCN, and the first approved CD123-targeted therapy, available across the EU, to address this high unmet medical need.

"Today's CHMP positive opinion is an important milestone for European patients suffering from BPDCN, and demonstrates our strong commitment to improve the lives of people affected by difficult-to-treat cancers", commented Elcin Barker Ergun, CEO of the Menarini Group. "BPDCN is a rare and aggressive disease with currently no approved therapeutic options across the EU. For this reason, we are particularly thrilled to be one step closer to making ELZONRIS available for BPDCN patients".

ELZONRIS is a targeted therapy directed to CD123, first commercially launched in the United States by Stemline Therapeutics, now part of the Menarini Group. Under the terms of the acquisition, Stemline shareholders received one non-tradeable Contingent Value Right (CVR) entitling each holder to $1.00 per share in cash upon completion of the first sale of ELZONRIS in any EU5 country after European Commission approval.

ELZONRIS was approved by the U.S. Food and Drug Administration in 2018, where it is currently available for the treatment of BPDCN in adult and pediatric patients, two years or older.

About ELZONRIS®

ELZONRIS® (tagraxofusp), a targeted therapy directed to CD123, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with BPDCN. For full prescribing information in the U.S., visit www.ELZONRIS.com.

ELZONRIS is also being evaluated in additional clinical trials in other CD123+ indications, including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), acute myeloid leukemia (AML), and others are planned, including a CD123+ all-comers trial.

About BPDCN

BPDCN, formerly blastic NK-cell lymphoma, is an aggressive hematologic malignancy, often with cutaneous manifestations, with historically poor outcomes. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. The World Health Organization (WHO) termed this disease "BPDCN" in 2008; previous names included blastic NK cell lymphoma and agranular CD4+/CD56+ hematodermic neoplasm.

About CD123

CD123 is a cell surface target expressed on a wide range of malignancies including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin's lymphoma (HL), and certain Non-Hodgkin's lymphomas (NHL). In addition, CD123+ cells have been detected in the tumor microenvironment of several solid tumors as well as in certain autoimmune disorders including cutaneous lupus and scleroderma.

About the Menarini Group

Menarini Group is a leading international pharmaceutical company with a presence in 140 countries, including a direct presence in over 70 countries. Its global platform extends throughout Europe, U.S., Central America, Africa, the Middle East and Asia Pacific, and generates over $4.2 billion in annual sales. Menarini is committed to oncology, with an already commercialized product in the US and several new investigational drugs in development for the treatment of a variety of tumors. For over 130 years, Menarini has also been investing in the development, production and distribution of pharmaceuticals to serve patients and physicians around the world with a full portfolio of products covering a number of different therapeutic areas.