Date: 2012-08-22
Type of information:
phase: 2
Announcement: publication of phase 2 data in Blood
The article is entitled “Rozrolimupab, A Mixture of 25 Recombinant Human Monoclonal RhD Antibodies, in the treatment of Primary Immune Thrombocytopenia”. It has been published online before print August 20, 2012 (doi: 10.1182/blood-2012-06-438804 - http://bloodjournal.hematologylibrary.org/content/early/recent).
Company: Symphogen (Denmark)
Product: rozrolimupab (SYM001)
Action mechanism: Rozrolimupab (SYM001) is a fully human recombinant anti-RhD monoclonal antibody (mAb) mixture, comprised of 25 mAbs binding to RhD. Rozrolimupab is produced by a single-batch manufacturing strategy designed to capture the natural diversity of the human antibody response to RhD as a modern counterpart to the plasma-derived anti-RhD immunoglobulins currently used in the treatment of ITP.
Disease: immune thrombocytopenia purpura (ITP)
Therapeutic area: Hematologic diseases - Autoimmune diseases
Country:
Trial
details: The Phase 2 study was an open-label, multi-center clinical trial evaluating the efficacy, safety, and tolerability of rozrolimupab (SYM001) in adult, RhD positive, non-splenectomized ITP patients.
The primary end point of the study was defined as response at day 7 from rozrolimupab administration. In the study, 61 patients were treated with single doses from 75 µg/kg to 300 µg/kg. Rozrolimupab was administered as single infusions of 15-20 minutes duration. Response was defined as platelet counts of >30 x 10 9/L and an increase of >20 x 109/L from baseline.
Latest
news: * On August 22, 2012, Symphogen has announced that the hematology journal Blood has published a full-length article describing final Phase 2 data for Symphogen’s rozrolimupab, a novel human recombinant mixture of 25 antibodies which all are manufactured simultaneously from a single batch.
The data demonstrates rozrolimupab’s favorable safety profile and its induction of a rapid increase in platelet counts in patients with Primary Immune Thrombocytopenia Purpura (ITP). The trial demonstrated that at 300µg/kg, 8 of 13 (62%) of patients responded at day 7. Already within 5 to 8 hours after rozrolimupab administration, 23% of the patients achieved platelet responses (> 30×109/L and increase in platelet count by > 20×109/L from baseline). Median time to response was 59 hours (approximately 2.5 days) and the median duration of response was 14 days.
The most common adverse events observed, were headache (20%), mostly mild or moderate, pyrexia (13%), chills (10%), and fatigue (8%). Four serious adverse events considered related to study drug were reported: decreased hemoglobin, extravascular hemolysis/dizziness and two cases of transient rise in D-dimer values without clinical symptoms.
According to professor Robak, “These Phase 2 results suggest an efficacy and safety profile similar to that seen with plasma derived immunoglobulin products. It seems promising that rozrolimupab rapidly yields platelet responses. This unique recombinant human monoclonal antibody mixture, rozrolimupab can be produced indefinitely and may represent a novel and convenient replacement for blood-derived immunoglobulins with more limited supply.”
Last December, Symphogen has presented final Phase 2 data at the 53rd Annual Meeting of the American Society of Hematology (ASH) in San Diego (See below)
* On December 12, 2011, Symphogen has presented final Phase 2 data demonstrating that its recombinant polyclonal antibody drug candidate rozrolimupab exhibited a favorable safety profile and induced a rapid increase in blood platelets in patients with Immune Thrombocytopenia Purpura (ITP). The trial demonstrated that at 300µg/kg, the best dose, 8 of 13 (62%) of patients responded at day 7. Median time to response was 59 hours (approximately 2.5 days) and the median duration of response was 14 days.
The most common adverse events, mostly mild or moderate, were headache (18%), pyrexia (13%), chills (8%), and fatigue (8%). Four serious adverse events considered related to study drug were reported: decreased hemoglobin, extravascular hemolysis/dizziness and two cases of transient rise in D-dimer values without clinical symptoms.
These results have been presented at the 53rd Annual Meeting of the American Society of Hematology (ASH) in San Diego CA in the oral session: « Disorders of Platelet Number or Function ». Professor Tadeusz Robak of the University of Lodz, Poland, (the lead investigator on the study), also presented a detailed analysis of the study’s findings in a talk entitled, « Final Results From a Phase II Trial with the First-in-Class Recombinant Polyclonal Antibody Product Rozrolimupab in Primary Immune Thrombocytopenia (ITP) ». According to Pr. Roback, « The Phase 2 results suggest a preliminary efficacy profile quite similar to that seen with plasma derived immunoglobulin products. The fully human recombinant nature of rozrolimupab makes it a promising, convenient replacement for older blood-derived immunoglobulins which while effective, carry the potential risk of disease transmission and have often been in short supply. »
