Company |
Product |
Description |
Indication |
Status |
Date |
American Society of Hematology | |||||
Abbvie Inc. (North Chicago, Ill.) |
Imbruvica |
Ibrutinib; Bruton's tyrosine kinase inhibitor |
Chronic lymphocytic leukemia or small lymphocytic lymphoma |
Phase Ib/II extension study data showed that 89% achieved complete or partial response at a five-year analysis, and 29% who received it as their first treatment achieved a complete response, and patients lived without disease progression longer when treatment was started earlier |
12/6/16 |
Abbvie Inc. (North Chicago, Ill.) |
Imbruvica |
Ibrutinib; Bruton's tyrosine kinase inhibitor |
Relapsed or refractory diffuse large B-cell lymphoma and follicular lymphoma (FL) |
Phase Ib data showed that as a combination therapy with rituximab and lenalidomide, it demonstrated the highest response rate in patients with the worst prognosis subtype and in patients with transformed disease; phase II data showed the combination of ibrutinib and rituximab produced, at a median time on study of 22 months, an overall response rate of 85%, with 35% achieving a complete response |
12/6/16 |
Abbvie Inc. (North Chicago, Ill.) |
Imbruvica |
Ibrutinib; Bruton's tyrosine kinase inhibitor |
Chronic graft-vs.-host disease |
Phase II data showed an overall response rate of 67% |
12/7/16 |
Acceleron Pharma Inc. (Cambridge, Mass.) and Celgene Corp. (Summit, N.J.) |
Luspatercept |
Protein therapeutic targeting molecules in the TGF-beta superfamily |
Lower risk myelodysplastic syndromes |
Phase II results demonstrated encouraging rates of transfusion independent and International Working Group Hematologic Improvement – Erythroid (IWG HI-E) response criteria |
12/6/16 |
Acceleron Pharma Inc. (Cambridge, Mass.) and Celgene Corp. (Summit, N.J.) |
Luspatercept |
Protein therapeutic targeting molecules in the TGF-beta superfamily |
Beta-thalassemia |
Data showed 81% (25 of 31) of transfusion dependent (TD) beta-thalassemia patients in the base study treated with luspatercept and 96% of those in the long-term extension study showed a red blood cell transfusion reduction ≥ 20% over any 12 weeks of the study period compared to 12 weeks pre-treatment; response rates were 71% and 83%, respectively |
12/7/16 |
Acetylon Pharmaceuticals Inc. (Boston) |
Citarinostat |
ACY-241; selective HDAC6 inhibitor |
Relapsed or relapsed-and-refractory multiple myeloma |
Early phase Ia/Ib results from citarinostat in combination with Pomalyst and dexamethasone showed that in 56 evaluable patients with a four-month median follow-up, the confirmed overall response rate was 46%, with a clinical benefit rate of 59% and disease control rate of 91%; the median duration of response was 9.2 months and median progression-free survival was 6.5 months |
12/6/16 |
Actinium Pharmaceuticals Inc. (New York) |
Actimab-A |
N/A |
Acute myeloid leukemia |
The analysis showed that 42% (8 of 19) of patients with blasts counts below 200/µL responded to Actimab-A while no patients with blast counts above 200/µL responded |
12/7/16 |
Agios Pharmaceuticals Inc. (Cambridge, Mass.) |
AG-120 |
A first-in-class oral, selective, potent inhibitor of mutant isocitrate dehydrogenase-1 |
IDH1 mutant positive hematologic malignancies |
Data from the completed dose-escalation portion of its phase I trial continued to show favorable safety and durable clinical activity; the overall response rate (ORR) was 38% (30 of 78) and complete remission rate (CRR) was 18% (14 of 78) were observed first-in-class, oral, selective, potent inhibitor of mutant isocitrate dehydrogenase-1 |
12/7/16 |
Agios Pharmaceuticals Inc. (Cambridge, Mass.) |
AG-348 |
PKR activator |
Pyruvate kinase deficiency |
New data showed it is well tolerated and demonstrates dose-dependent changes in ATP and 2,3-DPG blood levels in normal healthy volunteers consistent with PKR enzyme activation |
12/6/16 |
Akari Therapeutics (New York and London) |
Coversin |
Recombinant small protein |
Paroxysmal nocturnal hemoglobinuria |
Data from additional cohorts (15 mg and 22.5 mg) of the ongoing phase Ib trial showed a dose effect and demonstrated that the 22.5 mg maintenance dose also supports once daily dosing, as the 30-mg dose demonstrated earlier; data from the 22.5-mg once daily maintenance cohort demonstrated that subcutaneous coversin achieved complete complement inhibition within the first day, and demonstrated complete complement inhibition at the end of dosing on day seven |
12/6/16 |
Alexion Pharmaceuticals Inc. (New Haven, Conn.) |
ALXN1210 |
Longer-acting anti-C5 antibody |
Paroxysmal nocturnal hemoglobinuria |
Phase I/II data showed rapid and sustained reductions in lactate dehydrogenase in patients treated with once-monthly dosing; an interim analysis of 13 patients showed reductions in LDH at week one, and they were sustained over the study analysis period of up to 24 weeks |
12/6/16 |
Alnylam Pharmaceuticals Inc. (Cambridge, Mass.) |
Fitusiran |
RNAi therapeutic |
Hemophilia with inhibitors |
Phase I data showed once-monthly, subcutaneous dosing achieved a median annualized bleeding rate of zero; it was generally well tolerated with no thromboembolic events, including with co-administration of bypassing agents |
12/6/16 |
Alnylam Pharmaceuticals Inc. (Cambridge, Mass.) |
Fitusiran |
RNAi therapeutic |
Hemophilia A or B without inhibitors |
Phase II open-label extension data showed once-monthly, subcutaneous fitusiran achieved a median annualized bleeding rate of 1.0, with median observation period of 5.7 months |
12/6/16 |
Alnylam Pharmaceuticals Inc. (Cambridge, Mass.) |
Givosiran |
ALN-AS1; RNAi therapeutic targeting aminolevulinic acid synthase 1 |
Acute hepatic porphyrias |
Initial results from its phase I study showed robust and durable lowering of aminolevulinic acid (ALA) and porphobilinogen (PBG); in the first unblinded treatment cohort, givosiran demonstrated initial evidence for clinical activity in AIP patients with meaningful reductions in the number and frequency of porphyria attacks |
12/6/16 |
Alopexx Oncology LLC (Concord, Mass.) |
DI-Leu16-IL12 |
An immunocytokine that is a fusion of an anti-CD20 antibody and the cytokine interleukin-2 |
Relapsed/refractory non-Hodgkin lymphoma |
Disclosed data from an ongoing phase I/II trial, which showed 12 of 13 patients receiving two cycles of therapy had tumor regression or stabilization including a complete response (no detectable tumor) in one patient after two cycles of therapy |
12/7/16 |
AOP Orphan Pharmaceuticals AG (Vienna) and Pharmaessentia Corp. (Taipei, Taiwan) |
Ropeginter-feron alfa-2b |
Long-acting, mono-pegylated proline interferon |
Polycythemia vera (PV) |
Results from the PROUD-PV study, a phase III study to assess the efficacy and safety of ropeginterferon alfa-2b versus hydroxyurea (HU) showed that at 12 months, complete hematologic response was achieved in a high proportion of patients and non-inferiority was demonstrated (43.1% for ropeginterferon alfa-2b vs. 45.6% for HU in the intent-to-treat-population, p=0.0028) |
12/6/16 |
Apogenix GmbH (Heidelberg, Germany) |
Asunercept |
APG101; fusion protein consisting of the extracellular domain of human CD95-receptor and the Fc domain of a human IgG1 antibody |
Myelodysplastic syndrome |
Final phase I data showed eight of the 20 patients (40%) showed a marked reduction of transfusion frequency for six months (end of observation period); it was generally well tolerated with no reported grade 3 or higher related adverse events |
12/6/16 |
Argen-X BV (Breda, the Netherlands) |
ARGX-113 |
First-in-class antibody fragment |
Myasthenia gravis and immune thrombocytopenia |
Phase I data justified its dose selection for upcoming phase II trials; the company anticipates starting the first phase II study in MG before the end of the year, while the ITP study is expected to start in the first quarter of 2017. The company also presented further efficacy and safety data from an ongoing phase Ib study in relapsed/refractory T-cell lymphoma patients who failed multiple lines of therapy. Five out of 10 patients have shown encouraging signs of clinical activity, including three partial responses and two cases of stable disease |
12/5/16 |
Argen-X BV (Breda, the Netherlands) |
ARGX-113 |
First-in-class antibody fragment |
Relapsed/refractory T-cell lymphoma |
Phase Ib data showed five out of 10 patients have shown encouraging signs of clinical activity, including three partial responses and two cases of stable disease |
12/5/16 |
Ariad Pharmaceuticals Inc. (Cambridge, Mass.) |
Iclusig |
Ponatinib; BCR-ABL inhibitor |
Chronic myeloid leukemia |
A study of chronic-phase CML patients with the T315I mutation demonstrated a 72% probability of overall survival at 4.5 years among these patients who, prior to Iclusig, had no approved targeted treatment options and had a median survival of less than two years |
12/7/16 |
Astrazeneca plc (London) and Acerta Pharma |
Acalabrutinib |
Bruton tyrosine kinase (BTK) |
Difficult-to-treat forms of chronic lymphocytic leukemia |
Preliminary results from the phase I/II ACE-CL-001 clinical trial showed that in a population with difficult-to-treat disease and limited treatment options, a 79% overall response rate was achieved with acalabrutinib; the median progression free survival has not yet been reached, with 81% of responding patients achieving a duration of response ≥12 months on acalabrutinib treatment, which may allow for continuation of BTK inhibitor therapy |
12/6/16 |
Atara Biotherapeutics Inc. (South San Francisco) |
CMV-CTL |
Allogeneic T-cell product candidate |
Cytomegalovirus |
Released phase II results, which describe the efficacy and safety of CMV-CTL in the treatment of 15 patients with documented cytomegalovirus mutations conferring resistance to anti-viral therapies |
12/6/16 |
Beigene Ltd. (Waltham, Mass.) |
BGB-3111 |
Bruton tyrosine kinase (BTK) inhibitor |
Chronic lymphocytic leukemia (CLL) and small lymphocytic leukemia (SLL) |
Preliminary phase I data demonstrate that it is well-tolerated and highly active in CLL/SLL, with an overall response rate of 96%, and no cases of disease progression, at a median follow-up time of 8.6 months |
12/6/16 |
Bellicum Pharmaceuticals Inc. (Houston) |
BPX-501 modified T cells |
Donor T cells transduced with iC9 suicide gene |
Graft vs. host disease (GvHD) |
Results from the BP-004 clinical trial show that all 35 children treated are alive, free from GvHD and cured of their underlying disease; the results show that the inclusion of BPX-501 T cells may make haplo-HSCT a first-line option for children with PIDs who lack a suitable, HLA-matched donor |
12/6/16 |
Bergenbio AS (Bergen, Norway) |
BGB324 |
Axl inhibitor |
Acute myeloid leukemia |
Clinical and biological data from a phase I trial showed treatment was generally well-tolerated and steady-state levels of BGB324 were reached between three and six days after initiation of treatment |
12/6/16 |
Beyondspring Pharmaceuticals Inc. (New York) |
Plinabulin |
Small molecule agent with immune enhancing mechanism, tumor vasculature targeting and activating JNK pathway to induce cancer cell apoptosis |
Chemotherapy-induced neutropenia |
Phase II results showed that, given by intravenous infusion one hour after chemotherapy, it has the potential to be a safe, cost-effective and convenient alternative to granulocyte-colony stimulating factor with much less bone pain and a more favorable safety profile |
12/20/16 |
Biogen Inc. (Cambridge, Mass.) and Swedish Orphan Biovitrum AB (Sobi; Stockholm) |
Eloctate and Alprolix |
antihemophilic factor (recombinant), Fc fusion protein, and coagulation factor IX (recombinant), Fc fusion protein |
Hemophilia B |
Updated longitudinal safety and efficacy findings from phase III and extension studies showed low target joint annual bleeding rates and effective target joint resolution (≤2 spontaneous bleeding episodes over one year) in pediatric, adolescent and adult patients on long-term prophylaxis with Eloctate; an 18% improvement in hemophilia-related quality of life measures was seen in adolescents and adults who experienced target joint resolution with prophylactic treatment with Eloctate, compared to baseline measurements at phase III study entry; the greatest impact (≥20%) occurred in areas such as physical health, sports and leisure, and work and school |
12/6/16 |
Biolinerx Ltd. (Tel Aviv, Israel) |
BL-8040 |
Short peptide |
Acute myeloid leukemia (AML) |
Said final correlative results from its phase IIa trial of 45 patients with AML showed the composite complete remission rate, including both complete remission and complete remission with incomplete blood count recovery, was 38% in those receiving BL-8040 dose ≥1.0 mg/kg (n=39) |
12/6/16 |
Bio-Path Holdings Inc. (Houston) |
BP1001 |
Liposomal Grb2 antisense |
Chronic myelogenous leukemia |
Showed that BP1001 decreased the proliferation of Gleevec (imatinib, Novartis AG)-resistant CML cells in a dose-dependent manner; two CML patients, who had T315I mutation, showed significant reductions in circulating blasts during treatment |
12/7/16 |
Biothera Pharmaceuticals Inc. (Eagan, Minn.) |
Imprime PGG |
Pathogen-associated molecular patterning molecule designed to increase patient responses to Keytruda |
Head and neck squamous cell cancer |
Disclosed an expansion of its research collaboration with Merck & Co. Inc., of Kenilworth, N.J., to include a phase II study investigating Imprime PGG in combination with Merck's Keytruda (pembrolizumab); the goal of the new study is to determine the potential of Imprime PGG |
12/6/16 |
Bluebird Bio Inc. (Cambridge, Mass.) |
Lentiglobin |
Lentiviral-based gene therapy |
Transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD) |
Data from the ongoing open-label, single-center phase I/II HGB-205 study showed patients with TDT have 11.6 to 33.5 months of follow-up while the patient with SCD has 22.9 months of follow-up; as of the Sept. 9 data cut-off, three patients with TDT and β0/βE genotype remained free of transfusions since shortly after receiving Lentiglobin treatment, including one patient free of transfusions for 33.1 months with total hemoglobin of 10.9 g/dL – of which 7.7 g/dL was HbAT87Q – and one free of transfusions for 29.9 months with total hemoglobin of 13.5 g/dL; the first patient was enrolled in the global, multicenter Northstar-2 (HGB-207) phase III study |
12/6/16 |
Blueprint Medicines Corp. (Cambridge, Mass.) |
BLU-285 |
Selective inhibitor of the D816V mutant, KIT |
Advanced systemic mastocytosis (SM) |
Data from an ongoing phase I trial showed, in the dose escalation portion of the trial, a favorable pharmacokinetic profile with a half-life that supports once-daily dosing; the agent was well-tolerated at all doses, and no patients discontinued treatment as a result of an adverse event (AE), and no grade 4 or worse treatment-related AEs were reported; AEs that occurred in two or more patients included fatigue, anemia and alkaline phosphatase elevation |
12/6/16 |
Boehringer Ingelheim Pharmaceuticals Inc. (Ridgefield, Conn.) |
Pradaxa |
Dabigatran etexilate mesylate |
Atrial fibrillation (AF) |
Results from an analysis of the GLORIA-AF Registry Program showed that newly diagnosed non-valvular AF patients treated with Pradaxa had a 76.6% probability of remaining on treatment at one year and a 69.2% probability at two years; of patients who permanently discontinued treatment with Pradaxa, half (418/828) had switched to another oral anticoagulant by the second year of follow-up |
12/6/16 |
Boston Biomedical Inc. (Cambridge, Mass.) |
DSP-7888 |
Cancer peptide vaccine |
Myelodysplastic syndrome |
Data from a phase I/II trial showed it was well tolerated in MDS patients, and CTL induction and delayed type hypersensitivity (DTH) were detected; of 12 patients enrolled in the study, eight showed stable disease, including three with hematological improvements, for a disease control rate of 66.7% |
12/7/16 |
Bristol-Myers Squibb Co. (New York) |
Opdivo |
Nivolumab |
Non-small cell lung cancer |
Updated phase Ib findings evaluating its Opdivo monotherapy, or in combination with its Yervoy (ipilimumab), showed that in the pooled combination cohorts, the median progression-free survival in patients with PD-L1 expression ≥1% (n=46) was 12.7 months (95% CI: 7.8, 23.0) and was not reached in patients with PD-L1 expression >50% (n=13; 95% CI: 7.8, NR); for patients with ≥50% PD-L1 expression (n=13), the one-year overall survival rate was 100% in the pooled combination cohorts; the confirmed objective response rates in all treated patients (n=77) was 43%, nearly double the response rate reported with Opdivo monotherapy (23%; n=52), with six patients (8%) achieving a complete response, three of which were in patients with PD-L1 expression <1% |
12/6/16 |
Celgene Corp. (Summit, N.J.) |
CC-846 |
A DNA methyltransferase inhibitor |
Myelodysplastic syndromes, chronic myelomonocytic leukemia and acute myeloid leukemia |
Results from an analysis of three phase I/II studies showed the overall response rate was 38% |
12/7/16 |
Celgene Corp. (Summit, N.J.) |
Revlimid |
Lenalidomide |
Multiple myeloma |
The Myeloma XI trial, a U.K.-based, large, randomized, open-label phase III study, showed that the primary endpoint of progression-free survival was 36 months for the 857 patients receiving lenalidomide and 18 months for the 694 patients assigned to an observation arm (p< 0.0001); in the phase III BMT CTN 0702 StaMINA trial, stem cell transplant-eligible patients were randomized following transplant between three arms to receive either four cycles of lenalidomide-bortezomib-dexamethasone (RVD) consolidation therapy, tandem melphalan 200mg/m2 autologous stem cell transplant consolidation, or no consolidation.; all arms included lenalidomide maintenance; at a median follow-up of 38 months, all three arms demonstrated comparable progression-free survival and overall survival |
12/8/16 |
Cellectar Biosciences Inc. (Madison, Wis.) |
CLR 131 |
PDC radiotherapeutic |
Relapsed or refractory multiple myeloma |
Phase I data showed all eight evaluable patients (of 10 enrolled) achieved a minimum of stable disease |
12/7/16 |
Celyad SA (Mont-Saint-Guibert, Belgium) |
NKR-2 |
CAR-T candidate that uses NKG2D |
Acute myeloid leukemia and multiple myeloma |
Data from the phase I trial showed the drug was safe and well tolerated at the highest dose level tested to date (3x107), including no cases of cytokine release syndrome, cell-related neurotoxicity, auto-immunity or CAR-T related death |
12/7/16 |
Cornerstone Pharmaceuticals Inc. (Cranbury, N.J.) |
CPI-613 |
N/A |
Acute myeloid leukemia and T-cell non-Hodgkin lymphoma |
An open-label phase I dose escalation trial of CPI-613 administered intravenously in combination with high-dose cytarabine (HiDAC) and mitoxantrone showed an overall response rate (ORR) of 50%, including 26 patients in complete remission (CR) and five in CR with incomplete blood count recovery (Cri); median survival was 6.7 months |
12/7/16 |
CSL Behring (King of Prussia, Pa.) |
Idelvion |
Coagulation factor IX (recombinant), albumin fusion protein |
Hemophilia B |
Results from its phase III development program showed that adult patients who achieved sustained factor IX activity levels above 5 or 10% had approximately 80% lower risk of bleeding events over one year compared with those who had factor IX activity levels below these thresholds; patients who took Idelvion for one year and maintained cumulative factor IX trough levels above 5% and above 10% were predicted to reduce their bleeding risk by more than 80%, compared with patients having lower trough levels of factor IX activity |
12/6/16 |
CTI Biopharma Corp. (Seattle) |
Pacritinib |
Oral multikinase inhibitor |
High risk, thrombocytopenic myelofibrosis |
Data from PERSIST-2, a randomized phase III trial comparing pacritinib with physician-specified best available therapy (BAT) show that in myelofibrosis patients a statistically significant response rate in spleen volume reduction (SVR) with pacritinib therapy was observed compared to BAT that included use of the approved JAK1/JAK2 inhibitor ruxolitinib (p=0.001); the co-primary endpoint of reduction of total symptom score (TSS) was not achieved (p=0.079) but trended toward improvement in TSS |
12/7/16 |
Daiichi Sankyo Co. Ltd. (Tokyo) |
DS-3032 |
Oral selective MDM2 inhibitor |
Hematological malignancies including relapsed/refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS) |
Preliminary data from the dose escalation part of the phase I study found that the maximum tolerated dose of DS-3032 was determined to be 160 mg once daily for 21 days in a 28 day cycle based on results from 37 patients who received at least one dose of DS-3032; complete remission was seen in two patients with relapsed/refractory AML receiving 120 mg and 160 mg of DS-3032 with a duration of approximately four months and 13 months, respectively; one patient with high-risk MDS receiving the 120 mg dose of DS-3032 achieved marrow complete remission with platelet improvement for four months |
12/6/16 |
Dynavax Technologies Corp. (Berkeley, Calif.) |
SD-101 |
Toll-like receptor 9 agonist CpG-C class oligodeoxynucleotide |
Low-grade lymphoma |
Reported data on 28 evaluable patients from the dose escalation and expansion phase of a phase I/II trial; findings showed a combination of intratumoral SD-101 and low-dose irradiation resulted in tumor regression in untreated tumor sites as well as in the treated tumors; three patients had a partial response and one had a complete response; the most common treatment-related treatment emergent adverse events in the expansion phase were flu-like symptoms, consistent with the engagement of TLR9 and the induction of interferon-alpha; increases in CD8+ cells were observed in the injected tumor and correlated with increased abscopal tumor shrinkage |
12/6/16 |
Gamida Cell Ltd. (Jerusalem) |
Nicord |
Ex vivo expanded UCB progenitor cells with nicotinamide |
Sickle cell disease |
Phase I/II study provided clinical proof of concept that the therapy, which was transplanted with an un-manipulated unit of umbilical cord blood (UCB), may enable rapid engraftment in SCD patients transplanted after myeloablative conditioning; nine patients, ages 3 to 17, who were transplanted with Nicord and an un-manipulated unit of cord blood engrafted |
12/7/16 |
Global Blood Therapeutics Inc. (South San Francisco) |
GBT440 |
Oral, once-daily therapy |
Sickle cell disease (SCD) |
Results from its ongoing phase I/II study demonstrated that all 41 patients who received GBT440 for up to six months showed a profound and durable reduction in hemolysis; results from Part C of the trial showed that the 13 GBT440-treated patients demonstrated a clinically significant increase in hemoglobin (greater than 1 g/dL increase) compared with 14 placebo patients (46% vs. 0%; p=0.006); patients treated with GBT440 also had sustained reduction in irreversibly sickled cells compared with placebo treated patients (-76.6% vs. +9.7%; p<0.001); GBT440 was well tolerated during up to six months of dosing; the most common treatment-related adverse events were grade 1/2 headache and gastrointestinal disorders and occurred in similar rates in the placebo and GBT440 arms |
12/6/16 |
Glycomimetics Inc. (Rockville, Md.) |
GMI-1271 |
E-selectin antagonist |
Acute myeloid leukemia |
Results of its phase I/II clinical trial continued to show high rates of remission and favorable tolerability; GMI-1271 showed significant progress in the phase I/II clinical trial, in which clinicians are studying use of the drug candidate along with chemotherapy; for a total of 33 study participants with relapsed or refractory disease in one arm of the trial, the complete response (CR) rate was 45%; for 11 newly diagnosed study participants 60 or more years of age in the second arm of the trial, the CR rate was 73% |
12/6/16 |
Helsinn Group (Lugano, Switzerland) and MEI Pharma Inc. (San Diego) |
Pracinostat |
Oral histone deacetylase inhibitor |
Acute myeloid leukemia (AML) |
Final results from an open-label phase II study of pracinostat in combination with Vidaza (azacitidine, Celgene Corp.) in elderly patients with AML who were not eligible for induction chemotherapy showed the median overall survival was 19.1 (95% CI: 10.7-26.5) months, with a one-year survival of 62% and a complete response rate of 42%; the combination of pracinostat and azacitidine had no unexpected toxicities; the most common grade 3/4 treatment-emergent adverse events, reported in >10% of all patients, included thrombocytopenia, febrile neutropenia, neutropenia, fatigue and anemia |
12/6/16 |
Incyte Corp. (Wilmington, Del.) |
Jakafi |
Ruxolitinib; JAK1/JAK2 inhibitor |
High-risk myelofibrosis |
Reported an exploratory pooled data analysis from the five-year follow-up of the randomized phase III COMFORT-I and COMFORT-II trials; data suggested that earlier treatment may result in an improved survival advantage for patients compared to best available therapy (BAT) or placebo; median overall survival for ruxolitinib was 5.3 years compared with 3.8 years for the control group |
12/6/16 |
Innate Pharma SA (Marseille, France) |
IPH4102 |
Humanized anti-KIR3DL2 monoclonal antibody |
Relapsed/refractory cutaneous T-cell lymphomas |
Preliminary findings from a multicenter phase I study showed it was well tolerated, yielding a preliminary global objective response rate of 38% in the evaluable population across all dosage levels; explorative assessments showed clinical improvement in skin occurred in conjunction with decreases in malignant cells and normalization of immune parameters in the tumor microenvironment |
12/6/16 |
Innate Pharma SA (Marseille, France) |
Lirilumab |
Fully human monoclonal antibody that blocks inhibitory killer-cell immunoglobulin-like receptors expressed predominantly on natural killer cells |
Relapsed acute myeloid lymphoma (AML) |
Full doses in combination with azacytidine were tolerated in a heavily pretreated patient population in a phase Ib/II study; no dose-limiting toxicities were observed; preliminary efficacy data for 25 evaluable patients showed a response rate of 20%, including two patients who achieved a complete response (CR) or a CR with insufficient count recovery |
12/6/16 |
Janssen Research & Development LLC (Raritan, N.J.; unit of Johnson & Johnson) |
Darzalex |
Daratumumab |
Multiple myeloma |
Daratumumab in combination with lenalidomide and dexamethasone reduced the risk of disease progression or death by 64%, compared to lenalidomide and dexamethasone alone, in patients who received one to three prior lines of therapy; p<0.0001 |
12/7/16 |
Janssen Research & Development LLC (Raritan, N.J.; unit of Johnson & Johnson) |
Imbruvica |
Ibrutinib; Bruton's tyrosine kinase inhibitor |
Chronic lymphocytic leukemia/small lymphocytic lymphoma |
Updated phase Ib/II data showed an overall response rate (ORR) of 89%, including patients with genetic mutations associated with poor outcomes, and a complete response was observed in 29% of patients treated in the first-line setting; progression-free survival (PFS) was improved with earlier initiation of therapy across treatment-naïve (TN) and relapsed/refractory (r/r) patients; overall survival (OS) at five years was 92% for TN patients and 57% for r/r patients, with a PFS rate of 92% and 43%, respectively; the most frequent AEs were hypertension (26%), pneumonia (22%), neutropenia (17%) and atrial fibrillation (9%) |
12/6/16 |
Juno Therapeutics Inc. (Seattle) |
JCAR014 |
Targets CD19 |
Chronic lymphocytic leukemia (CLL) |
Early phase I data in patients with CLL who failed treatment with ibrutinib (Imbruvica, Janssen Biotech Inc./Pharmacyclics LLC) showed that of patients received lymphodepletion with either fludarabine/cyclophosphamide (flu/cy) (N=21) or non-flu/cy (N=3) prior to infusion of JCAR014, two of 24 (8%) developed grade 3-5 severe cytokine release syndrome (sCRS) and 6 of 24 (25%) developed grade 3-5 severe neurotoxicity; of 17 efficacy-evaluable patients with bone marrow disease who were treated with flu/cy and the two lowest doses of JCAR014, 15/17 (88%) had a complete marrow response by flow cytometry; in patients with PET-avid disease at baseline and treated with flu/cy and the two lowest doses of JCAR014, 8 of 11 (73%) had a partial response (PR) or complete response (CR) at four weeks, with 7 of 11 (64%) having a CR |
12/6/16 |
Juno Therapeutics Inc. (Seattle) |
JCAR017 |
Chimeric antigen receptor T-cell therapy |
Relapsed or refractory CD19-positive acute lymphoblastic leukemia |
40 out of 43 evaluable pediatric and young adult patients experienced a minimal residual disease (MRD)-negative complete remission; all patients who received preconditioning with fludarabine/cyclophosphamide lymphodepletion demonstrated an overall response to the therapy; the estimated 12-month event-free survival is 50.8% and overall survival is 69.5%; severe cytokine release syndrome was observed in 23% of the patients |
12/8/16 |
Karyopharm Therapeutics Inc. (Newton, Mass.) |
KPT-330 |
Selinexor |
Multiple myeloma |
Results from the phase Ib dose escalation portion of its ongoing STOMP study showed high response rates when selinexor was combined with the proteasome inhibitor bortezomib (Velcade, Takeda Oncology Co.), including in patients with multiple myeloma previously refractory to proteasome inhibitors |
12/7/16 |
Karyopharm Therapeutics Inc. (Newton, Mass.) |
Selinexor |
KPT-330; oral selective inhibitor of nuclear export |
Multiple myeloma (MM) |
Said phase IIb STORM study data showed that among 78 evaluable patients who had a median of seven prior treatment regimens, the overall response rate was 21%; among 48 in the quad-refractory group, the ORR was 21%; clinical benefit rate of selinexor was 32% and median overall survival was 9.3 months for all patients |
12/6/16 |
Karyopharm Therapeutics Inc. (Newton, Mass.) |
Selinexor |
KPT-330; oral selective inhibitor of nuclear export |
Acute myeloid leukemia (AML) |
Phase II SAIL data, in which deep responses to selinexor allowed patients with heavily pretreated AML to proceed onto stem cell transplantation or donor lymphocyte transfusion, showed that among 42 evaluable patients, the overall response rate was 55% and included 10 complete remissions and 10 achieving complete remission with incomplete blood count recovery, suggesting it is an effective treatment option when used in combination with Ara-C and idarubicin |
12/6/16 |
Kiadis Pharma NV (Amsterdam) |
ATIR101 |
Adjuvant infusion |
Leukemia |
One-year data from the single dose phase II trial showed no patients developed grade III-IV graft vs. host disease (GvHD) upon infusion of ATIR101; three cases of grade II acute GvHD and one case of chronic GvHD was reported after infusion; two patients developed disease relapse within the first 12 months after HSCT; no mortality was observed within the first 100 days post-HSCT; nine patients died within the first year after transplantation, resulting in a one-year overall survival of 61% |
12/7/16 |
Kite Pharma Inc. (Santa Monica) |
KTE-C19 |
Therapy in which a patient's T cells are engineered to express a chimeric antigen receptor to target the antigen CD19 |
Adult and pediatric relapsed and refractory acute lymphoblastic leukemia |
82% of patients achieved complete remission in a preliminary analysis of its phase I ZUMA-3 and ZUMA-4 trials; all of the responders tested negative for minimal residual disease, which is known to correlate with risk of disease relapse; five of 13 patients had grade 3 or higher cytokine release syndrome, and one patients in ZUMA-3 died from KTE-C19-related CRS |
12/6/16 |
Mateon Therapeutics Inc. (South San Francisco) |
OXi4503 |
Vascular disrupting agent |
Acute myeloid leukemia or myelodysplastic syndrome |
Data from its ongoing phase Ib OX1222 study of OXi4503, in combination with cytarabine, showed two of 10 patients achieved a complete remission (CR) on treatment and currently remain in CR without further treatment, one at six months and the other at three months; one of six responded in the 3.75 mg/m2 dose cohort, and one patient of four responded in the 4.68 mg/m2 dose cohort |
12/7/16 |
Merck & Co. Inc. (Kenilworth, N.J.) |
Keytruda |
Pembrolizumab); anti-PD-1 therapy |
Relapsed or refractory primary mediastinal large B-cell lymphoma (PMBCL) |
Demonstrated an overall response rate of 41% with follow-up of up to 27 months in patients with PMBCL who were ineligible for or failed to respond to autologous stem-cell transplantation; 86% of responses were ongoing at the time of analysis |
12/6/16 |
Merck & Co. Inc. (Kenilworth, N.J.) |
Keytruda |
Pembrolizumab); anti-PD-1 therapy |
Relapsed or refractory classical Hodgkin lymphoma |
In the KEYNOTE-087 and KEYNOTE-013 trials, Keytruda demonstrated overall response rates (ORRs) of 69% and 58%, respectively; KEYNOTE-013, which had a median follow up of 29 months, showed responses of 12 months or greater in 70% of patients who responded to therapy; in KEYNOTE-087, across all 210 enrolled patients, the complete remission rate was 22.4% (n=47; 95% CI, 16.9-28.6); across all cohorts, 93% of patients experienced a decrease in tumor burden (n=192) |
12/7/16 |
Morphosys AG (Munich, Germany) |
MOR208 |
Fc-modified antibody targeting CD19 |
Advanced B-cell malignancies |
Updated safety and efficacy data from two ongoing phase II studies showed that at the last cutoff date of June 3, three patients with DLBCL and six with iNHL were in remission and on study treatment, with the longest responses in both subgroups ongoing for more than 26 months; of these nine patients, seven showed complete responses and two experienced partial responses; the median duration of response was 20.1 months for DLBCL and not yet reached for iNHL; the overall response rate was 36% in the DLBCL subgroup and 33% in iNHL patients |
12/7/16 |
Nordic Nanovector ASA (Oslo, Norway) |
Betalutin |
177Lu-satetraxetan-lilotomab |
Non-Hodgkin lymphoma |
Updated phase I/II data confirm the efficacy and safety of Betalutin in relapsed NHL patients who have failed multiple prior regimens; in 35 evaluable patients, the overall response rate was 63%, with 29% complete responses; of 21 evaluable patients who received the 15 MBq/kg dose with 40 mg/m2 lilotomab pre-dosing had an ORR of 62% and a CR of 38%; of these, the 16 enrolled in the phase II expansion of Arm 1 had an ORR of 69% and a CR of 38% |
12/6/16 |
Novartis AG (Basel, Switzerland) |
CTL019 |
Chimeric antigen receptor T-cell therapy |
B-cell acute lymphoblastic leukemia |
Said in the global phase II ELIANA study it demonstrated complete remission or complete remission with incomplete blood count recovery at three months post infusion in 82% (41 of 50) of patients; the estimated relapse-free rate among responders was 60% |
12/6/16 |
Novartis AG (Basel, Switzerland) |
SEG101 |
Crizanlizumab; anti-P-selectin antibody |
Sickle cell-related pain |
Phase II SUSTAIN study data showed it reduced the median annual rate of sickle cell-related pain crises by 45.3% compared to placebo (p=0.010) in the high-dose group (5.0 mg/kg) in patients with or without hydroxyurea therapy; in the low-dose group (2.5 mg/kg), the annual rate of SCPC was reduced by 32.6% vs. placebo (p=0.180) |
12/6/16 |
Novo Nordisk (Bagsvaerd, Denmark) |
NovoSeven |
Portable room-temperature stable recombinant activated factor VIIa |
Hemophilia A or B with inhibitors |
Said it resolved 96.5% of bleeds when initiated within one hour after onset of bleeding, demonstrating efficacy of early treatment |
12/6/16 |
Oncoceutics Inc. (Philadelphia) |
ONC201 and ONC212 |
Imipridone class of small-molecule chemical compounds |
Acute leukemias |
Three abstracts related to ONC201 describe preliminary results from an ongoing phase I/II trial in acute leukemias, the demonstration of synergy between ONC201 and cytarabine in preclinical models, and the downstream anticancer effects of dopamine receptor 2 antagonism in multiple myeloma; a fourth abstract highlights ONC212, which induced p53-independent apoptosis, in synergy with ABT-199 (venetoclax) |
12/6/16 |
Oncolytics Biotech Inc. (Calgary, Alberta) |
Reolysin |
Oncolytic virus |
Myeloma |
Initial findings from the two-stage, open-label REO 019 phase Ib trial evaluating tolerability and confirming that the addition of Reolysin to bortezomib (Velcade, Takeda Oncology Co.) increases endoplasmic reticulum stress and death of myeloma cells and to document pharmacodynamic effects; a maximum tolerated dose was not defined in the first two cohorts |
12/7/16 |
Onconova Therapeutics Inc. (Newtown, Pa.) |
Rigosertib |
Oral therapy designed to inhibit P13K and pololike kinase |
First-line higher risk myelodysplastic syndromes |
Said oral rigosertib and azacitidine demonstrated a 35% complete remission rate in a phase II trial; the rate is higher and responses occur more rapidly and durably with the combination compared to historic single-agent azacitidine |
12/6/16 |
Oryzon Genomics (Barcelona, Spain) |
RG6016 |
A potent and selective inhibitor of lysine-specific histone demethylase-1 |
Relapsed or refractory acute leukemia |
Top-line results from its global phase I trial showed it met its primary and secondary endpoints, with data supportive of anti-leukemia activity including observations of morphologic differentiation, changes of gene expression patterns consistent with blast differentiation, and effects on blast counts in blood and bone marrow (BM) |
12/7/16 |
Pfizer Inc. (New York) |
Glasdegib |
Oral, smoothened (SMO) inhibitor |
Acute myeloid leukemia or high-risk myelodysplastic syndrome |
Phase II data showed its addition to low-dose cytarabine (LDAC) significantly increased overall survival when compared to LDAC alone in patients who were ineligible for intensive chemotherapy (p=0.0003); median overall survival for glasdegib plus LDAC was 8.8 months, compared to 4.9 months for those taking LDAC only |
12/6/16 |
Roche Holding AG (Basel, Switzerland) |
Gazyva |
Obinutuzumab |
Follicular lymphoma |
Improved progression-free survival (PFS) by 34% compared to Rituxan (p=0.0012), but lacked an overall survival benefit (p=0.21), in the GALLIUM study |
12/6/16 |
Seattle Genetics Inc. (Bothell, Wash.) |
Adcetris |
Brentuximab vedotin; antibody-drug conjugate directed to CD30 |
High-risk diffuse large B-cell lymphoma |
Reported that in a phase II study it demonstrated an objective response rate of 83%, including 69% complete remissions, when used in combination with rituximab (Rituxan), or with cyclophosphamide, doxorubicin, vincristine and prednisone (RCHOP); it showed an objective response rate of 91%, including 82% complete remissions, when used in combination with RCHP (removing the vincristine); the most common adverse events in were fatigue, peripheral sensory neuropathy, diarrhea, nausea, alopecia and constipation |
12/6/16 |
Seattle Genetics Inc. (Bothell, Wash.) |
Vadastuximab talirine (33A) |
A CD33-directed antibody-drug conjugate |
Acute myeloid leukemia |
Results from 42 newly diagnosed AML patients treated in the ongoing phase Ib trial of 33A in combination with the 7+3 induction therapy showed that half (21) went on to receive an allogeneic stem cell transplant; unlike Mylotarg, which has been associated with a risk of liver toxicity, only one patient in the 33A study showed an LFT elevation of grade 3 or higher; a total of 76% of patients achieved complete remission, and most of those - 60% - hit CR with marrow recovery |
12/6/16 |
Seattle Genetics Inc. (Bothell, Wash.) and Bristol-Myers Squibb Co. (New York) |
Adcetris |
Brentuximab vedotin; antibody-drug conjugate directed to CD30 |
Relapsed or refractory Hodgkin lymphoma |
Data from an ongoing phase I/II clinical trial showed that 26 patients (90%) had an objective response, including 18 patients (62%) with a complete metabolic response and eight patients (28%) with a partial metabolic response |
12/7/16 |
Seattle Genetics Inc. (Bothell, Wash.) and Takeda Pharmaceutical Co. Ltd. (Osaka, Japan) |
Adcetris |
Brentuximab vedotin; antibody-drug conjugate directed to CD30 |
CD30-expressing cutaneous T-cell lymphoma |
Reported phase III data showing it met the primary endpoint of achieving a highly statistically significant improvement in the rate of objective response lasting at least four months (56.3% vs. 12.5%); median progression-free survival was 16.7 months vs. 3.5 months |
12/6/16 |
Spark Therapeutics Inc. (Philadelphia) |
SPK-9001 |
Gene therapy |
Hemophilia B |
All nine patients had achieved stable expression levels of Factor IX that were at least 12% of normal |
12/6/16 |
Spectrum Pharmaceuticals Inc. (Henderson, Nev.) |
Pralatrexate |
N/A |
Relapsed/refractory peripheral T-cell lymphoma |
Data from a case match control analysis of the PROPEL study revealed a survival advantage; 80 patients out of 109 were successfully matched 1:1 with the control population; a highly significant difference in the overall survival between the control population and the pralatrexate group was observed; the overall survival in the control population was 4.04 months (95% CI 2.60, 6.01) which was consistent with historical controls, while the median OS in for pralatrexate treated cohort was 14.78 months (95% CI 10.61-22.31) |
12/7/16 |
Stemline Therapeutics Inc. (New York) |
SL-401 |
Targeted therapy directed to the interleukin-3 receptor |
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) |
Results from the ongoing SL-401 phase II trial demonstrated that SL-401 produced a 100% overall response rate (ORR), including an 81% complete response rate in first-line BPDCN patients treated at the recommended dose of 12 ug/kg/day and a 95% ORR in first-line patients treated at the recommended dose or lower; in relapsed/refractory patients, the ORR was 69% |
12/6/16 |
Sunesis Pharmaceuticals Inc. (South San Francisco) |
SNS-062 |
Oral non-covalent reversible BTK inhibitor |
B-cell malignancies |
Phase Ia data show a favorable safety, pharmacokinetic and pharmacodynamics profile; SNS-062 demonstrated rapid, profound (~100%) and prolonged inhibition of BTK at all dose levels and support investigation of a twice-daily dosing regimen in B-cell malignancies with or without an acquired BTK resistance mutation |
12/6/16 |
Sunesis Pharmaceuticals Inc. (South San Francisco) |
Vosaroxin/cytarabine |
N/A |
Relapsed/refractory acute myeloid leukemia |
Updated results from the phase III VALOR trial examining overall survival in 711 patients age 60 years and older found, during a primary analysis in October 2014, that overall survival was significantly improved with vosaroxin/cytarabine versus placebo/cytarabine (7.1 months vs 5.0 months, HR=0.75, p=0.0030), with a complete remission (CR) rate of (31.9% vs 13.8%, p < 0.0001); results demonstrate that, after a median of 39.9 months of follow-up, OS for patients age 60 years and older remains significantly improved for the vosaroxin/cytarabine arm compared to the placebo/cytarabine arm), with survival curves remaining separated through 48 months |
12/7/16 |
TG Therapeutics Inc. (New York) |
TGR-1202 and TG-1101 |
A once-daily P13K delta inhibitor and ublituximab, a glycoengineered anti-CD20 monoclonal antibody |
Relapsed or refractory diffuse large B-cell lymphoma or follicular lymphoma |
Data from three combination studies showed the triple combination of TG-1101, TGR-1202 and bendamustine appeared to be well tolerated with no discontinuations for a treatment related adverse events; a 71% overall response rate (ORR) was noted, including a 43% complete response (CR) rate observed in patients with relapsed or refractory DLBCL; a phase I trial of TGR-1202 in combination with brentuximab vedotin, in patients with relapsed/refractory Hodgkin's lymphoma revealed a 60% ORR, including a 40% CR rate observed across brentuximab vedotin refractory patients |
12/7/16 |
Trillium Therapeutics Inc. (Mississauga, Ontario) |
TTI-621 |
Fusion protein that consists of the CD47-binding domain of human SIRPa linked to the Fc region of a human immunoglobulin |
Relapsed or refractory hematologic malignancies |
Data showed decreased tumor volume and/or reduced metabolic activity over extended intervals of continued dosing in several patients; phase Ia/Ib data in demonstrated that an optimal dose of 0.2 mg/kg/week was well tolerated and associated with predictable, transient thrombocytopenia |
12/6/16 |
True North Therapeutics Inc. (South San Francisco) |
TNT009 |
Monoclonal antibody that selectively inhibits the classical complement pathway by targeting C1s |
Cold agluttinin disease (CAD) |
Said phase Ib data support it as a treatment, given the normalization of hemoglobin levels seen in severely anemic patients; all five CAD patients without an active malignancy had a sustained response with improvements in hemoglobin of approximately 4-5 g/dL; patients that previously required transfusions became transfusion-free during treatment with TNT009, which was safe and well-tolerated with no serious or severe adverse events |
12/6/16 |
Uniqure NV (Lexington, Mass. and Amsterdam, the Netherlands) |
AMT-060 |
Gene therapy |
Severe hemophilia B |
Phase I/II data showed all four patients that previously required chronic replacement therapy were able to discontinue Factor IX infusions, and one spontaneous bleed was reported following the discontinuation; all five patients in the low-dose cohort continue to maintain robust, constant and clinically meaningful levels of FIX activity for up to 52 weeks post treatment; an update from its second, higher dose cohort, showed gene expression of Factor IX of 7% of normal |
12/6/16 |
Verastem Inc. (Boston) |
Duvelisib |
Oral, dual inhibitor of phosphoinositide-3-kinase-delta and P13K-gamma |
Multiple hematologic cancers |
Results from the DYNAMO study, a phase II clinical trial yielded an overall response rate of 46%; among disease subgroups, the ORR was 41% in follicular lymphoma (n=83), 68% in small lymphocytic lymphoma (n=28), and 33% in marginal zone lymphoma (n=18); median duration of response among all patients was 9.9 months; notably, 83% of patients had reductions in the size of their target lymph nodes |
12/7/16 |
San Antonio Breast Cancer Symposium | |||||
Astrazeneca plc (London) |
Faslodex |
Fulvestrant |
Locally advanced or metastatic breast cancer |
Results from a pre-specified subgroup analysis of the phase III FALCON trial showed a decreased risk of progression by 41% in women whose disease has not spread to organs within the chest or abdomen when compared with Arimidex |
12/9/16 |
Corcept Therapeutics Inc. (Menlo Park, Calif.) |
Korlym |
Mifepristone |
Triple-negative breast cancer with tumors expressing GR |
Results of an open-label study showed the addition of mifepristone enhances the effect of Halaven (eribulin, Eisai Co. Ltd.); four patients in the study exhibited a partial response, defined as a 30% or greater reduction in tumor size, eight had stable disease and 11 had progressive disease |
12/13/16 |
Eisai Co. Ltd. (Tokyo) |
Halaven and Keytruda |
Eribulin and pembrolizumab |
Metastatic triple negative breast cancer |
Interim phase Ib/II data showed an overall response rate of 33.3%; one patient showed a complete response and 12 patients showed a partial response; the most common treatment-emergent adverse events for the combination regimen were fatigue, nausea, peripheral neuropathy, neutropenia and alopecia |
12/13/16 |
Eli lilly and Co. (Indianapolis) |
Abemaciclib |
A cyclin-dependent kinase 4 and CDK 6 inhibitor |
Breast cancer |
The neoMONARCH study of abemaciclib met its primary endpoint of reducing expression of Ki67, a biomarker of cell proliferation, after two weeks of treatment |
12/9/16 |
Puma Biotechnology Inc. (Los Angeles) |
PB272 |
Neratinib |
Grade 3 diarrhea |
Interim results from a phase II trial of PB272 showed the grade 3 diarrhea rate was 25% and for the 107 patients who received the modified loperamide dosing regimen the grade 3 diarrhea rate was 29% |
12/9/16 |
Radius Health Inc. (Waltham, Mass.) |
RAD1901 |
An oral selective estrogen receptor degrader |
Estrogen receptor positive breast cancer |
Data from two ongoing phase I studies showed RAD1901 was well-tolerated with the most common adverse events being low grade nausea and dyspepsia |
12/9/16 |
Seattle Genetics Inc. (Bothell, Wash.) |
SGN-LIV1A |
Antibody-drug conjugate |
Triple-negative metastatic breast cancer |
An interim analysis of its ongoing phase I trial showed that, out of 30 of 47 efficacy-evaluable patients, 37% achieved a partial response (PR); the disease control rate was 67% and the clinical benefit rate was 47%; at the time of the interim data analysis, the estimated median progression-free survival was 12 weeks with seven patients remaining on treatment; the most common adverse events included fatigue, nausea, alopecia, decreased appetite and constipation |
12/13/16 |
Notes Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. The date indicated refers to the BioWorld Today issue in which the news item can be found. For more information about individual companies and/or products, see Thomson Reuters Cortellis. |