Italfarmaco Announces Publication of Positive Long-term Data for Givinostat in Polycythemia Vera Patients in Blood Cancer Journal

• Four-year mean follow-up data demonstrate an overall response rate (ORR) of over 80% throughout the follow-up period
• Randomized, double-blind Phase 3 study to assess the efficacy and safety of GIVinostat versus hydroxyurea IN JAK2V617F-positive high-risk Polycythemia Vera patients (GIV-IN PV TRIAL) with over 100 participating international clinical sites to be initiated by mid-2021

MILAN--(BUSINESS WIRE)-- The Italfarmaco Group announced a publication in the Blood Cancer Journal outlining the first interim analysis results from follow-up of a mean of four years in 51 patients with polycythemia vera (PV), a type of hematological malignancy belonging to BCR-ABL1-negative chronic myeloproliferative neoplasms (MPNs). The results show a long-term benefit in PV patients treated with givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor. The publication covers the overall long-term data from patients that were a part of three Phase 1/2 studies, as well as from a compassionate use program. The data preliminarily support the long-term use of givinostat. The open access article is available via this link.

“These long-term results support the potential of givinostat as a disease-altering treatment in patients with PV who are currently confined to symptomatic care,” said Alessandro Rambaldi, M.D., Professor of Hematology at the University of Milan, Head, Hematology and Bone Marrow Transplant Unit at ASST Papa Giovanni XXIII in Bergamo, lead author and investigator of the trial. “The overall response rate, which continues to be persistent over the four-year mean follow-up, combined with the good safety profile, justifies the global pivotal Phase 3 study, which will start this year, to further evaluate givinostat and generate clinical evidence necessary to bring the drug to patients.”

“With over 100,000 polycythemia vera cases in the U.S. and considering that these patients have an increased risk of developing myelofibrosis or acute myeloid leukemia, in addition to the day-to-day complications caused by the disease, the data announced today provide PV patients with hope for a new treatment option,” added Srdan Verstovsek, M.D., Ph.D., Professor of Medicine, Chief, Section for MPNs at the Department of Leukemia and Director at the Clinical Research Center for MPNs at MD Anderson Cancer Center in Houston, Texas. “I value the opportunity to participate in the upcoming pivotal and global Phase 3 trial, which will be the first of its kind in PV.”

The results published in Blood Cancer Journal summarize the four-year mean follow-up of an open-label, long-term study (Clinicaltrials.gov: NCT01761968) that enrolled 51 patients with PV, who received clinical benefit from givinostat in the company’s previous three Phase 1/2 clinical trials and/or on compassionate use. These patients continued to receive givinostat at the last effective and tolerated dose. The follow-up analyses showed that givinostat had a good overall safety and tolerability profile. During the study, only five patients (10.0%) experienced Grade 3 Adverse Events (AE) and no treatment-related Grade 4 or Grade 5 AEs occurred, supporting the strong safety profile reported in previous analyses. Over 80% of the patients responded to treatment according to the ELN Response Criteria, i.e., the internationally recognized response criteria that evaluates the effect of treatment on haematology, spleen volume and symptoms in PV (Barosi G et al., Blood 2009). Such a response was maintained for the duration of the follow-up period.

A reduction in the mean Janus Kinase 2 (JAK2) allele burden, a gene believed to be key in the pathogenesis of PV, was also observed regularly at annual patient visits. Importantly, the majority of the PV patients continued treatment with givinostat after the conclusion of the study period as they were receiving relevant clinical benefit, supporting its long-term use.

Italfarmaco expects to initiate the global Phase 3 trial with givinostat in PV patients by mid-2021 with more than 100 participating sites in Europe, US, Canada and Asia.

About Polycythemia Vera
Polycythemia Vera is a rare blood disease characterized by an overproduction of red blood cells, white blood cells and platelets, which thickens the blood and increases the risk of blood clots, a major underlying cause of life-threatening conditions such as thrombosis, embolisms, heart attacks or strokes. In up to 95% of patients, the disease is associated with a mutation in the Janus Kinase 2 (JAK2) gene and disease-related symptoms include headaches, itching and microvascular symptoms. The current standard of care ranges from phlebotomy alone or in combination with low-dose aspirin, to drugs such as the cytoreductive hydroxycarbamide, interferon (ropeginterferon) or the JAK inhibitor (ruxolitinib). These reduce symptoms, but no treatments targeting the underlying disease mechanism are available.

About Givinostat
Givinostat is an investigational drug discovered through Italfarmaco’s internal research. It is being evaluated for safety and efficacy for the treatment of Duchenne- and Becker- Muscular Dystrophy and Polycythemia Vera. Givinostat inhibits histone deacetylases (HDACs), which are enzymes that prevent gene translation by changing the three-dimensional folding of DNA in the cell. Givinostat selectively targets the specific JAK2 mutation responsible for MPNs, thereby inhibiting cell growth and proliferation of hematopoietic cells.

About Italfarmaco Group
Italfarmaco is a specialty pharmaceutical company engaged in the discovery, development, manufacturing and marketing of branded prescription and nonprescription products in more than 60 countries on 5 continents. Italfarmaco’s research and development expertise is best demonstrated through its HDAC inhibitor development programs, addressing new therapeutic treatments of specialty and rare diseases. Through both marketed drugs and compounds in development, Italfarmaco is dedicated to serving patients whose needs remain largely unmet.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210318005370/en/