Company
(location)

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

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