The following data were presented at the American Society of Hematology meeting in Orlando, Fla.

• Acetylon Pharmaceuticals Inc., of Boston, reported favorable results of preclinical safety and efficacy testing of its oral selective HDAC6 inhibitor drug candidate, ACY-1215, in multiple myeloma. Studies showed that ACY-1215, when administered either as a single agent or in combination with the proteasome inhibitor drug Velcade (bortezomib, Millennium/Takeda), demonstrated effectiveness in two in vivo disease models of multiple myeloma as well as against drug-resistant multiple myeloma patient cells.

• Aeterna Zentaris Inc., of Quebec City, and Keryx Biopharmaceuticals Inc., of New York, reported data from two Phase II trials showing that perifosine achieved objective response and a high rate of stable disease as a single-agent therapy in chronic lymphocytic leukemia, and in combination with Nexavar (sorafenib, Onyx Pharmaceuticals Inc. and Bayer AG) in relapsed or refractory Hodgkin's lymphoma. The drug currently is in Phase III trials in multiple myeloma and advanced colorectal cancer.

• Alexion Pharmaceuticals Inc., of Cheshire, Conn., presented interim results from the first Phase I/II trial in adult patients with advanced stage B-cell chronic lymphocytic leukemia or multiple myeloma testing samalizumab, the company's humanized monoclonal antibody. Data indicated that samalizumab was well tolerated at all doses studied, exhibited a dose-dependent biological and pharmacokinetic response and exhibited initial evidence of antitumor activity.

• Allos Therapeutics Inc., of Westminster, Colo., reported that new analyses of data from the company's Phase II PROPEL trial of Folotyn (pralatrexate injection) in patients with relapsed or refractory peripheral T-cell lymphoma demonstrated the ability of Folotyn to achieve responses in patients in the second-line treatment setting immediately following treatment with cyclophosphamide/doxorubicin/vincristine/prednisone (CHOP), and indicated that Folotyn may be an effective treatment option in patients who have progressed after aggressive chemotherapy regimens such as ifosfamide, carboplatin and etoposide. Separately, the firm presented updated results from a Phase I dose-finding study of Folotyn which the company said supports the selection of 15 mg/m2 weekly for three weeks out of a four-week cycle as the optimal starting dose and schedule for further evaluation in patients with relapsed or refractory cutaneous T-cell lymphoma. (See BioWorld Today, July 29, 2010.)

• Amgen Inc., of Thousand Oaks, Calif., reported final results from a five-year, open-label extension study of the long-term safety and efficacy of Nplate (romiplostim) in adult chronic immune thrombocytopenic purpura. The study found that platelet cells were safely maintained between 50,000 and 200,000 per microliter in a majority of patients for up to 277 weeks. Ninety-five percent of the 292 patients in the study achieved 50,000 or more platelets per microliter. The median treatment duration was 78 weeks.

• Array BioPharma Inc., of Boulder, Colo., presented positive Phase I data for its kinesin spindle protein inhibitor, ARRY-520, that indicated that it was well tolerated and showed encouraging preliminary results in the treatment of multiple myeloma. ARRY-520 also is being investigated as a single-agent in a Phase II trial and in a Phase Ib combination trial with Velcade (bortezomib, Millennium/Takeda) plus dexamethasone in patients with relapsed and refractory multiple myeloma.

• Baxter Healthcare Corp., of Deerfield, Ill., presented interim data from a Phase I study that suggested recombinant von Willebrand factor (rVWF) may be safe and well tolerated in patients with Type III and severe Type I von Willebrand disease. The interim data from 22 patients in the 32-patient study also suggested that rVWF has a pharmacokinetic profile that is comparable to plasma-derived von Willebrand factor, the current standard for treatment for patients with the disease.

• BioCryst Pharmaceuticals Inc., of Birmingham, Ala., presented data that confirmed clinical activity of its drug forodesine in chronic lymphocytic leukemia. In the Phase II open-label, multicenter trial, six of 23 patients demonstrated a partial response to forodesine 200 mg twice daily for 28 days. The drug is a purine nucleoside phosphorylase inhibitor.

• Biovest International Inc., of Tampa, Fla., presented an updated Phase III data analysis suggesting that improved disease-free survival rates following administration with BiovaxID, its personalized vaccine for non-Hodgkin's lymphoma, depend on an integral tumor protein fragment administered as part of each patient's vaccine. In lymphoma, that protein fragment previously was believed to be unimportant to vaccine response. The new analysis further suggested that patients receiving a vaccine with a specific isotype (IgM) experienced a dramatic disease-free survival benefit, with IgM-positive patients treated with BiovaxID remaining tumor free nearly twice as long vs. IgM-positive control (52.9 months vs. 28.7 months median time to relapse).

• Bristol-Myers Squibb Co., of New York, and Otsuka Pharmaceutical Co. Ltd., of Tokyo, reported that an 18-month follow up from its Phase III trial of Sprycel (dasatinib) 100 mg once daily vs. Gleevec (imatinib, Novartis AG) 400 mg once daily for chronic myeloid leukemia (CML) was consistent with data at 12 months that showed Sprycel produced higher and faster rates of complete cytogenetic response. The trial was carried out as a first-line treatment in adults with Philadelphia chromosome-positive chronic phase CML.

• Calistoga Pharmaceuticals Inc., of Seattle, reported that updated data from an ongoing Phase I single-agent trial and the first data from a combination Phase I trial of CAL-101, the company's oral, delta-isoform-selective PI3K inhibitor, demonstrated the clinical potential of CAL-101 as a single agent and in combination with other standard-of-care therapies in hematologic malignancies that have relapsed or are refractory to existing treatments. In addition, pharmacokinetic data and preclinical data characterizing the pharmacological properties of CAL-101 were presented.

• Cell Therapeutics Inc., of Seattle, reported that pixantrone showed continuous improvement in primary and secondary endpoints in a Phase III trial. Complete response was 24 percent in the pixantrone arm vs. 7 percent in the comparator arm. The results of the trial are included in the company's marketing authorization application under review by the European Medicines Agency for use in relapsed or refractory aggressive non-Hodgkin's lymphoma.

• ChemGenex Pharmaceuticals Ltd., of Melbourne, Australia, presented updated clinical data showing that Omapro (omacetaxine mepesuccinate) produced durable hematologic and cytogenetic responses in a significant proportion of chronic phase chronic myeloid leukemia (CML) patients who had failed previous attempts to control their disease with two or three FDA-approved tyrosine kinase inhibitors. Data were presented from 61 evaluable CML patients in chronic phase (defined as those who had been adjudicated by an independent data monitoring committee and had a bone marrow report available for cytogenetic assessment) and from the complete group of 85 chronic phase CML patients analyzed on an intent-to-treat basis. ChemGenex plans to file a new drug application for Omapro for the treatment of CML patients who have failed two or more approved TKIs. (See BioWorld Today, July 15, 2010.)

• Compugen Ltd., of Tel Aviv, Israel, said results from recently completed studies by the Institute for Myeloma & Bone Cancer Research demonstrated the therapeutic potential of CGEN-928 as a drug target for treating multiple myeloma through mAb therapy. The company said that CGEN-928, a previously uncharacterized protein, was shown earlier by Compugen to be broadly expressed in multiple myeloma tumor cells.

• Cyclacel Pharmaceuticals Inc., of Berkeley Heights, N.J., reported preclinical data for CYC065 showing that CYC065 is cytotoxic to myeloma cell lines and CD138+ myeloma cells at submicromolar concentrations. The company is preparing clinical studies for the drug. In a separate presentation, Cyclacel reported one-year survival data from its Phase II trial of sapacitabine for myelodysplastic syndromes (MDS) refractory to hypomethylating agents. The study enrolled 61 patients older than 60 with MDS refractory to hypomethylating agents and randomly assigned them to 200 mg twice daily, 300 mg twice daily or 400 mg twice daily. Twenty-nine percent of patients achieved the primary endpoint of 1-year survival.

• Emergent BioSolutions Inc., of Rockville, Md., presented results from a Phase I dose-escalation study of TRU-016 (Protocol 16007) that showed TRU-016 demonstrated favorable response rates and was generally well tolerated in patients with chronic lymphocytic leukemia. TRU-016 is the company's humanized anti-CD37 small modular immunopharmaceutical candidate in development with Abbott, of Abbott Park, Ill., for the treatment of B-cell malignancies such as CLL and non-Hodgkin's lymphoma.

• Geron Corp., of Menlo Park, Calif., presented final data from its Phase II trial of GRNVAC1, an autologous dendritic cell vaccine targeting telomerase, in patients with acute myelogenous leukemia that suggested targeting telomerase through a dendritic cell-based vaccine approach may prolong remission duration in some patients with high-risk AML. The multicenter, open-label trial was designed to evaluate the feasibility of GRNVAC1 manufacture and the safety and tolerability of the vaccination regimen in patients with AML who were in complete clinical remission.

• Idera Pharmaceuticals Inc., of Cambridge, Mass., presented preclinical data showing that IMO-4200, a dual agonist of Toll-like receptor 7 and TLR8, in combination with anti-CD20 antibody Rituxan (rituximab, Biogen Idec Inc., and Genentech Inc./Roche AG) resulted in improved antitumor activity and greater antibody-dependent cell cytotoxicty in a mouse model of lymphoma. In combination with proteasome inhibitor Velcade (bortezomib, Millennium/Takeda), the drug resulted in increased survival compared to treatment with either agent alone.

• ImmunoGen Inc., of Waltham, Mass., reported positive initial data from a Phase I trial with the company's IMGN901 anticancer compound when used as part of a standard combination regimen to treat relapsed multiple myeloma. IMGN901 is designed to target and kill cancer cells that express the CD56 antigen. The company said encouraging updated data also were reported for the compound when used as a monotherapy in patients with extensively pretreated multiple myeloma.

• Immunomedics Inc., of Morris Plains, N.J., reported that combination treatment with milatuzumab, a humanized anti-CD74 antibody in clinical testing, and veltuzumab, a humanized anti-CD20 antibody, resulted in therapeutic responses in heavily pretreated and Rituxan (Genentech Inc./Roche AG and Biogen Idec Inc.)-refractory non-Hodgkin's lymphoma patients. Results on other milatuzumab-based combination treatments also were presented at the meeting in two separate preclinical studies. Separately, the firm presented data from a Phase II study of fractionated radioimmunotherapy with Y-90 epratuzumab in aggressive lymphoma. The company reported that consolidation treatment with fractionated yttrium-90 (Y-90) labeled epratuzumab, after induction therapy with rituximab and CHOP chemotherapy, improved remission status of adult patients with diffuse large B-cell lymphoma, a form of aggressive lymphoma. Preclinical results of IMMU-114 for prevention and treatment of graft-vs.-host disease also were reported.

• Incyte Corp., of Wilmington, Del., presented positive long-term results from an ongoing open-label Phase II trial for INCB18424, a selective, oral JAK1 and JAK2 inhibitor, in patients with advanced polycythemia vera (PV) and essential thrombocythemia. The data showed long-term clinical activity, including reduction in spleen size, phlebotomy independence (in PV patients) and improvement in blood counts lasting up to 27 months. The company said it anticipates top-line data from a U.S. Phase III study (COMFORT-I) in myelofibrosis this month. (See BioWorld Today, Sept, 14, 2010.)

• Ligand Pharmaceuticals Inc., of San Diego, reported data from a preclinical study on the erythropoietin receptor agonist LG5640. The company said it tested the effect of LG5640 on EPO-dependent cell lines and in cultures of CD34-positive human bone marrow cells, a well-established model for the effects of EPO on erythroid progenitor cells and their maturation into red blood cells. Ligand said its findings suggested the potential for those small-molecule EPOR agonists to provide additional benefit in the treatment of anemia with improved safety, tolerability and patient acceptance due to the convenience of oral administration and the lack of excessive erythropoietic stimulation that may contribute to the adverse effects of the current injectable ESAs.

• MethylGene Inc., of Montreal, reported that in a Phase II trial in 43 patients with relapsed or refractory Hodgkin lymphoma, dosing with mocetinostat (MGCD0103) resulted in complete response for two patients, partial response for 12 patients and durable stable disease for at least six months for one patient. Mocetinostat is an isoform-selective HDAC inhibitor.

• Micromet Inc., of Bethesda, Md., reported that an analysis of long-term efficacy data from a Phase II trial of the company's lead product candidate blinatumomab (MT103) in adult patients with minimal residual disease-positive acute lymphoblastic leukemia (ALL) demonstrated that blinatumomab produced prolonged remissions in patients with ALL. As of November, the hematologic disease-free survival was 60 percent, with a follow-up of up to 27.5 months. Separately, the firm presented updated results from an ongoing Phase I trial of blinatumomab in patients with relapsed non-Hodgkin's lymphoma, suggesting that blinatumomab continues to produce a high response rate and duration of response in a number of different NHL subtypes.

• Millennium Pharmaceuticals Inc., of Cambridge, Mass., a unit of Takeda Pharmaceutical Co. Ltd. and partner Janssen-Cilag, of Beerse Belgium, a subsidiary of Johnson & Johnson, reported results from a 222-patient study comparing subcutaneous vs. intravenous administration of Velcade (bortezomib) showing that the efficacy in patients with relapsed multiple myeloma was similar in both administrations, with one-year overall survival of 72.6 percent in the subcutaneous group and 76.7 percent in the I.V. group.

• Novartis Pharmaceutical Corp., of East Hanover, N.J., part of Novartis AG, reported pivotal Phase II data showing that LBH589 (panobinostat) demonstrated substantial disease control and tumor reduction in extensively pretreated Hodgkin lymphoma patients who had relapsed or had become refractory after an autologous stem cell transplant. Eight-two percent of patients achieved disease control and 74 percent achieved tumor reduction at a median follow-up of 9.6 months. Partial and complete responses were seen in 27 percent of patients, with a median duration of response of 6.9 months. Separately, Novartis reported new data from a Phase III trial of Tasigna (nilotinib) for chronic myeloid leukemia (CML). The data, collected at 24 months of treatment of patients with newly diagnosed Philadelphia chromosome-positive CML, showed that patients treated with 300 mg Tasigna twice daily had a lower incidence of progression to accelerated phase and blast crisis, compared to Glivec 400 mg once daily. The Tasigna group also had a lower incidence of suboptimal response and treatment failure.

• Onconova Therapeutics Inc., of Newtown, Pa., reported data from 48 higher risk patients with myelodysplastic syndromes or acute myeloid leukemia who failed or became resistant to previous drug treatments, showing that they had increases in overall survival when treated with small-molecule drug Estybon (ON 01910.Na). Those increases correlated with bone marrow blast responses. The most dramatic improvement in overall survival was seen in 19 patients who also showed either bone marrow complete response or an initial decrease of more than 50 percent in blast cells. Onconova separately reported preclinical data showing that cyclin D1 inhibitor ON 013105 was active in animal models.

• Pfizer Inc., of New York, reported that patients with chronic myeloid leukemia treated with its investigational compound bosutinib experienced major molecular response at a higher rate than those treated with Gleevec (imatinib, Novartis AG) in a Phase III study. The study did not meet its primary endpoint of superior complete cytogenic response at one year vs. imatinib. Patients on bosutinib experienced more adverse events leading to discontinuation than did those on imatinib.

• Pharmacyclics Inc., of Sunnyvale, Calif., presented data from three chronic lymphocytic leukemia (CLL) studies describing the Btk-selective inhibitor PCI-32765. Data from two preclinical studies together suggested a model whereby PCI-32765 directly affects CLL cells by inducing apoptosis and also blocks the ability of CLL cells to migrate to and adhere to lymph nodes. Clinical data from a pooled Phase Ia and Ib analysis of 54 patients with CLL or small lymphocytic lymphoma treated with PCI-32765 showed it was well tolerated by patients.

• Roche AG, of Nutley, N.J., reported Phase II data of monoclonal antibody GA101 for relapsed and refractory non-hodgkin's lymphoma, showing that a third of patients who had tried three previous therapies responded, and 25 percent of patients who had failed Rituxan/MabThera responded. In a second Phase II trial of patients with refractory disease, 55 percent responded to GA101 with a median progression-free survival of 11.3 months.

• S*BIO Pte. Ltd., of Singapore, said data from Phase I and II studies for its JAK2 inhibitor SB1518 indicated clinical efficacy and good tolerability for the treatment of patients with symptomatic myelofibrosis and enlarged spleens. SB1518 also was well tolerated in patients with relapsed/refractory lymphoma. S*BIO and Onyx Pharmaceuticals, Inc., of Emeryville, Calif., have a development collaboration and option and license commercialization agreement for the JAK2 inhibitors, SB1518 and SB1578, also known as ONX 0803 and ONX 0805, respectively. (See BioWorld Today, Jan. 8, 2009.)

• Spectrum Pharmaceuticals Inc., of Irvine, Calif., said median follow-up data from a Phase III trial in first-line indolent follicular non-Hodgkin's lymphoma showed that patients treated with a single-dose infusion of Zevalin (ibritumomab tiuxetan) after partial or complete response to first-line chemotherapy had a nearly three-year advantage in median progression-free survival compared to patients treated with either chemotherapy alone or chemotherapy plus Rituxan (rituximab, Genentech Inc./Roche AG and Biogen Idec Inc.) (49 months vs. 14 months). At the 66.2-month analysis, five-year PFS was 47 percent in the Zevalin group and 29 percent in the control group (p < 0.0001).

• Telik Inc., of Palo Alto, Calif., reported that its candidate Telintra (ezatiostat hydrochloride) showed positive results in a Phase II trial in patients with myelodysplastic syndromes. The study enrolled 87 patients at 22 sites divided into groups receiving 3 g daily for two weeks with one week of rest and 2 g daily for three weeks with one week of rest. Treatment continued for six months, and those receiving benefit continued for an additional six months. There was a significant response rate among various patient groups broken down by treatment history.

• YM BioSciences Inc., of Mississauga, Ontario, reported interim data from the first 60 patients enrolled in its Phase I/II myelofibrosis study of JAK1/JAK2 inhibitor CYT387, which showed an overall response rate of 62 percent. Of the 42 patients evaluable for anemia response, 21 subjects (50 percent) had achieved clinical improvement as per the Myeloproliferative Neoplasms Research and Treatment criteria, while 25 of 53 (47 percent) evaluable patients who had splenomegaly at baseline achieved a minimum 50 percent decrease in palpable spleen size. CYT387 was well tolerated, with no Grade 4 non-hematological toxicities observed. Shares of YM (AMEX:YMI) gained 11 percent, or 22 cents, to close Monday at $2.22.

• Ziopharm Oncology Inc., of New York, presented new preclinical data on the mechanism of its organic arsenic darinaparsin (ZIO-101) in various lymphoma models that demonstrated that darinaparsin inhibited cell growth and induced apoptosis through the AKT and MEK/ERK pathways and all cell lines at 1-3mcM. AKT and MEK/ERK-based pathways are known to play a key role in multiple cellular processes, including cell proliferation, apoptosis, transcription and cell migration, the company said, adding that at 2mcM (48 hours), darinaparsin induced approximately 80 percent apoptosis in each of the four TCL lines, while 3mcM resulted in 65 percent apoptosis in L428 cells.