Blood Cancer

December 19, 2016

AOP Orphan Pharmaceuticals and PharmaEssentia reported results of a phase III study of ropeginterferon alfa-2b versus HU in patients with Polycythemia Vera. PROUD-PV is a phase III, randomized, open-label, multicenter, controlled, parallel arm study and is part of the development program to support marketing authorizations of ropeginterferon alfa-2b (AOP2014/ P1101) in Europe and in the U.S. The pre-specified primary endpoint, which was a composite of Complete Hematologic Response (CHR) and spleen length normality, was confounded by the fact that the median spleen length was almost normal at baseline and the observed change was not clinically relevant (21.3% for ropeginterferon alfa-2b versus 27.6% for HU in the intent-to-treat-population, p=0.2233). At 12 months, Complete Hematologic Response (CHR) was achieved in a high proportion of patients and non-inferiority was demonstrated (43.1% for ropeginterferon alfa-2b versus 45.6% for HU in the intentto- treat-population, p=0.0028). Ropeginterferon alfa-2b showed significantly better tolerability than HU. Overall, 59.6% of the patients in the ropeginterferon alfa-2b arm experienced treatment-related adverse events compared to 75.6% of the patients treated with HU (p<0.05). There was no difference in adverse events of special interest concerning interferons (auto-immune, psychiatric) or concerning PV (cardiovascular disorders).

August 22, 2011

Bio-Path Holdings issued interim results from an ongoing phase I trial of BP-100-1.01 (Liposomal Grb-2) for hematological cancers. A total of 13 treatment experienced subjects received an intravenous dose of 5 mg/m2 twice a week for four weeks. Six of the 13 subjects were evaluable for response; the remaining seven failed to complete a full 28-day cycle due to disease progression. Liposomal Grb-2, at a dose of 5 mg/m2 was well tolerated. Lab parameters for blasts and bone marrow also demonstrated possible anti-leukemia activity. Two subjects had transient improvement and/or stable disease and received a total of five cycles each, representing five months on treatment with the drug. Two additional subjects had transient improvement on leukemia cutis lesions. In addition to the six evaluable subjects, a single subject with CML blast phase who had failed all available therapies showed a significant reduction in blasts from 81% to 4%.

April 18, 2005

Celgene reported positive interim results of a pair of parallel phase III trials of Revlimid (lenalidomide), for the treatment of multiple myeloma. Trial data have yielded strong evidence of superiority, with a median time to progression of at least 15 months for the US study and more than 11 months for the international study, vs. only 5 months for subjects receiving dexamethasone alone. This superiority was highly statistically significant (p<0.00001), and the duration and number of ongoing responses mean that the primary endpoint, which was to characterize the overall time to progression, cannot yet be calculated. Clinical responses were observed in 51.3% and 47.5% (respectively by trial) of subjects receiving combination therapy with the drug, vs. 22.9% and 18.4% of subjects receiving dexamethasone monotherapy (p=0.001). Both studies were randomized, double-blinded, placebo-controlled trials which enrolled relapsed or refractory multiple myeloma patients; these patients were randomized 1:1 to receive Revlimid plus dexamethasone (combination therapy) or dexamethasone alone. One trial enrolled 354 subjects across 47 US sites, while the other enrolled 351 subjects across 50 international sites. These ongoing trials are being conducted under a Special Protocol Assessment with the FDA, and will form the basis of both NDA and MAA submissions.

CureTech issued results of a phase I trial of their investigational monoclonal antibody CT-011, for the treatment of hematological malignancies. Trial data yielded positive safety indications, with no significant adverse events noted and a positive overall toxicity profile: no dose-limiting toxicities were observed, the most common adverse events were mild allergic reaction and low grade fever, and single-administration maximum tolerated dose was not reached. Preliminary evidence of clinical response was also observed in 5 subjects, with 1 partial response (including platelet transfusion independence) through 8 months, 2 incidences of stable disease through at least 7 months, and 2 minimal responses. This open-label escalating dose study enrolled 17 subjects with advanced refractory hematological malignancies at the Chaim Sheba Medical Center in Tel-Hashomer, Israel. Subjects received a single 5-hour intravenous infusion of escalating doses of the drug, and were followed for safety, tolerability, and evidence of disease progression.

Lorus Therapeutics has reported preliminary results of a phase II study of GTI-2040, their investigational ribonucleotide reductase R2 inhibitor, for the treatment of renal cell carcinoma. Results from the study yielded evidence of efficacy, with 52% of subjects experiencing disease stabilization or better, including 1 patient with a 23% reduction in tumor burden and disease stabilization through 10 months and 1 partial response, a 39% reduction in tumor burden and disease stabilization through 8 months. This open-label study enrolled 33 patients with advanced metastatic renal cell carcinoma across 7 US sites, who received combination therapy with GTI-2040 and capecitabine, an approved chemotherapeutic. The trial was co-sponsored by the National Cancer Institute.