Abbvie Inc. (North Chicago)

Imbruvica

Ibrutinib

Relapsed/refractory mantle cell lymphoma

Pooled analysis results in Bruton's tyrosine kinase inhibition found that at three years 45% of patients were able to achieve overall survival (OS) and 26% had disease progression-free survival (PFS); analysis also found that patients who were treated with it earlier, after their first relapse/progression, experienced higher rates of OS and PFS

12/12/17

Ablynx NV (Ghent, Belgium)

Caplacizumab

Anti-von Willebrand factor Nanobody

Acquired thrombotic thrombocytopenic purpura

Phase III HERCULES data showed treatment with it resulted in a 38% reduction in the number of days of plasma exchange compared to placebo and a 41% reduction in the volume of plasma used; it also reduced the number of days in the intensive care unit by 65% compared to placebo and reduced the number of days in the hospital by 31%

12/13/17

Acceleron Pharma Inc. (Cambridge, Mass.)

Luspatercept

Modified activin receptor type IIB fusion protein

Lower-risk myelodysplastic syndromes

Phase II data showed that of the 99 patients treated with therapeutic dose levels, 53% achieved a clinically meaningful erythroid response of an increase in hemoglobin or reduction in red blood cell (RBC) transfusion burden as per the International Working Group's Hematologic Improvement Erythroid response criteria; of the 67 patients with an RBC transfusion burden at baseline of at least two units per eight weeks, 43% achieved RBC transfusion independence for at least eight weeks

12/12/17

Actinium Pharmaceuticals Inc. (New York)

Actimab-A

Anti-CD33 antibody lintuzumab labeled with the alpha-emitting isotope actinium-225

Acute myeloid leukemia

Phase II trial testing in patients newly diagnosed who are over the age of 60 and not able to tolerate induction chemotherapy showed treatment produced an overall response rate (ORR) of 69% and reduced bone marrow blasts by 98%

12/12/17

Actinium Pharmaceuticals Inc. (New York)

Actimab-M

Anti-CD33 monoclonal antibody lintuzumab coupled to an alpha-emitting radioisotope Actinium-225

Multiple myeloma

Results from a study of 865 samples from U.S. patients showed that 25% had CD33 expression and support the rationale for the company's phase I trial

12/13/17

Adaptimmune Therapeutics plc (Oxford, U.K.)

NY ESO SPEAR

T-cell therapy

Multiple myeloma

Data from its pilot study showed: as of the Aug. 16 cut-off, 11 of 25 patients were still alive with three of the patients remaining disease-free at three, 4.5 and five years post-treatment; the median duration of response was more than 12 months with a median predicted overall survival of approximately three years; the overall response rate at one year was 44%

12/13/17

Affimed NV (Heidelberg, Germany)

AFM-13

Tetravalent, bispecific NK cell engager

Relapsed or refractory Hodgkin lymphoma

Phase Ib data showed that, in combination with Keytruda, it was well-tolerated as salvage therapy after failure of standard therapies with a three-month overall response rate (ORR) of 83% which compares favorably to historical ORR of pembrolizumab alone in a similar patient population (58% to 63%)

12/12/17

Agios Pharmaceuticals Inc. (Cambridge, Mass.)

AG-348

Pyruvate kinase-R activator

Pyruvate kinase deficiency

Phase II DRIVE PK trial data showed treatment with it produced a maximum hemoglobin (Hb) increase from baseline of more than 1 g/dL during the six-month treatment in 50% of the patients; of the 42 patients enrolled with at least one missense mutation, 60% had a maximum Hb increase from baseline of at least 1 g/dL

12/12/17

Agios Pharmaceuticals Inc. (Cambridge, Mass.)

AG-120 and Idhifa

Ivosidenib and enasidenib

Newly diagnosed acute myeloid leukemia (AML) and an isocitrate dehydrogenase (IDH)1 or IDH2 mutation

Data from two studies demonstrate the potential benefit of IDHm inhibitors in the front-line setting for patients with AML; specifically, the phase I front-line combination trials showed that both are well tolerated when combined with standard induction chemotherapy or azacitidine, and both trials demonstrated early encouraging signs of efficacy

12/13/17

Alexion Pharmaceuticals Inc. (New Haven, Conn.)

ALXN-1201

Long-acting C5 complement inhibitor

Paroxysmal nocturnal hemoglobinuria

Data from two phase Ib/II studies showed that treatment for up to eight months resulted in rapid and sustained reduction of plasma lactate dehydrogenase (LDH) levels, a direct marker of hemolysis, with reductions in mean LDH levels from baseline ranging from 73% to 88%

12/12/17

Amgen Inc. (Thousand Oaks, Calif.)

Kyprolis

Carfilzomib

Relapsed or refractory multiple myeloma

The phase III Aspire trial met the key secondary endpoint of overall survival, demonstrating that the addition of Kyprolis to Revlimid and dexamethasone (KRd) reduced the risk of death by 21% vs. lenalidomide and dexamethasone alone (Rd) and extended survival by 7.9 months (median OS 48.3 months for KRd vs. 40.4 months for Rd, HR=0.79, 95% CI, 0.67 – 0.95; p=0.0045)

12/13/17

Astellas Pharma Inc. (Tokyo)

Gilteritinib

FLT3 inhibitor

Newly diagnosed acute myeloid leukemia

Phase I findings from the dose-escalation cohort (n=17) showed that two subjects in the 40-mg/day cohort receiving it on days one through 14 experienced dose-limiting toxicities; maximum tolerated dose was not reached; 120 mg/day was chosen as the recommended expansion dose; in FLT3mut-positive and FLT3 wild-type subjects, end-of-treatment composite complete response rates were 100% and 60.9%, respectively

12/12/17

Astrazeneca plc (Cambridge, U.K.) and subsidiary Acerta Pharma LLC (Wilmington, Del.)

Calquence

Acalabrutinib

Mantle cell lymphoma

Phase II ACE-LY-004 data showed the overall response rate to the therapy was consistent across multiple subgroups, including age, tumor burden and number or type of prior treatments; the secondary endpoint of median duration of response had not yet been reached at 15.2 months median follow-up

12/12/17

Atara Biotherapeutics Inc. (South San Francisco)

Tabelecleucel

Immuno-therapy

Epstein-Barr virus (EBV)-associated cancers

Interim results from a multicenter expanded access protocol study showed: in six patients with Rituxan-refractory EBV-associated post-transplant lymphoproliferative disorder (PTLD) following solid organ transplant (SOT), the objective response rate (ORR) was 83%; in five patients with rituximab-refractory EBV+PTLD following allogeneic hematopoietic cell transplant (HCT), an ORR of 80% was observed; estimated one-year overall survival for the 12 treated patients with EBV+PTLD following HCT or SOT, was 90.9%

12/12/17

Beigene Ltd. (Cambridge, Mass. and Beijing)

Zanubrutinib

BTK inhibitor

Non-Hodgkin's lymphoma (NHL) subtypes

Preliminary phase Ib data suggest that it was generally well-tolerated and had antitumor activity across NHL subtypes; in patients with follicular lymphoma, the overall response rate (ORR) was 41% with complete responses (CRs) in 18% and partial responses (PRs) in 24% of patients, stable disease (SD) was observed in 41% of patients and progressive disease (PD) was observed in one patient; in patients with marginal zone lymphoma, the ORR was 78% with no CR, PRs in 78% of patients, SD was observed in 22% of patients and no PD was observed.

12/12/17

Beigene Ltd. (Cambridge, Mass. and Beijing)

Zanubrutinib

BTK inhibitor

Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and follicular lymphoma (FL)

Testing it in combination with Gazyva showed: in treatment-naive (TN) CLL/SLL patients, after a median follow-up of 11.4 months (6 months to 17.3 months), the overall response rate (ORR) was 95% with complete responses (CRs) in 35% and partial responses (PRs) in 60% of patients; in relapsed or refractory (R/R) CLL/SLL patients, at a median follow-up time of 12.7 months, the ORR was 92% with CRs in 20% and PRs in 72% of patients; in R/R FL patients, at a median follow-up time of 12.1 months, the ORR was 76% with CRs in 38% and PRs in 38% of patients; ORR in high-risk CLL/SLL patients with del17p/p53 mutation, del11q mutation, and unmutated IGHV were 83%, 100%, and 95%, respectively

12/12/17

Bellicum Pharmaceuticals Inc. (Houston)

BPX-501

Adjunct T-cell therapy

Inherited blood disorders and hematologic malignancies

Patients treated with it following an alpha/beta T-cell and CD19+ B-cell-depleted haploidentical hematopoietic stem cell transplant (haplo-HSCT) experienced both persistence and expansion of the cells, contributing to improved immune recovery vs. historical controls; peak expansion of the cells is reached at nine months after infusion, and BPX-501 cells are consistently detected after two years

12/12/17

Bergenbio ASA (Bergen, Norway)

BGB-324

AXL inhibitor

Relapsed/refractory (R/R) acute myeloid leukemia (AML) or myeloid dysplastic syndrome (MDS)

Out of 35 patients with R/R AML or MDS who received it in a phase II study, two achieved complete responses with incomplete hematologic recovery and five achieved partial responses; seven reported disease stabilization for more than four months; four remained on study at the time of data cut-off; three novel predictive biomarker candidates that correlated significantly with clinical benefit were detected in blood, bone marrow plasma or bone marrow cell samples from patients

12/12/17

Bristol-Myers Squibb Co. (Princeton, N.J.)

Sprycel

Dasatinib

Newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL)

Data from a phase II study in pediatric patients demonstrated an event-free survival rate, the study's primary endpoint, of 65.5% (95% CI: 57.7 to 73.7); the overall survival rate was 91.5% (95% CI: 84.2 to 95.5) at three years; Sprycel and chemotherapy were generally well tolerated in pediatric Ph+ ALL patients and all treated patients achieved complete remission

12/12/17

Bristol-Myers Squibb Co. (Princeton, N.J.)

Orencia

Abatacept

Graft-vs.-host disease (GVHD)

Phase II data showed when added to the standard drug regimen it reduced the occurrence of acute, grade III-IV GVHD from 32% to 3% in pediatric and adult patients who underwent mismatched unrelated donor stem cell transplants to treat advanced cancer and other blood disorders; as a result, patients receiving the post-transplant regimen with abatacept experienced improved disease-free and overall survival compared to those who did not

12/12/17

Caelum Biosciences Inc. (New York, unit of Fortress Biotech Inc.)

CAEL-101

mAb 11-1F4

Relapsed and refractory amyloid light chain amyloidosis

Phase Ia/Ib trial investigators determined the study achieved its primary objective of establishing maximum tolerated dose of up to 500 mg/m2; investigators also presented organ response rates in phase Ia and Ib, with a 63% overall organ response rate, 67% overall cardiac response rate and 50% overall renal response rate; early organ response was demonstrated in a high-mortality population

12/12/17

Catalyst Biosciences Inc. (South San Francisco)

CB-2679d/ISU-304

Prophylactic factor IX variant

Severe hemophilia B

Interim phase I/II data showed that subcutaneous delivery significantly increases the factor IX activity half-life to 98.7 hours; cohort one results showed that I.V. CB-2679d is about 22 times more potent and has a significantly longer half-life and mean residence time than Benefix (36 hours vs. 25 hours); cohorts two and three results showed that subcutaneous delivery of CB-2679d had a bioavailability of 18.5% and significantly increases the factor IX activity half-life to 98.7 hours; no serious adverse events were observed

12/12/17

Cyclacel Pharmaceuticals Inc. (Berkeley Heights, N.J.)

Sapacitabine

Orally-available nucleoside analogue

Newly diagnosed acute myeloid leukemia

The phase III SEAMLESS trial failed to meet its primary endpoint of increasing overall survival, but a prespecified subgroup analysis showed increased overall survival in patients with less than 10,000 white blood cells with a median of eight months for decitabine-sapacitabine compared to 5.8 months for decitabine alone, although the difference wasn't statistically significant; in that subgroup, there was a statistically significant increase in complete remission (CR) rate of 17% for the decitabine-sapacitabine arm vs. 11% for decitabine alone; decitabine-sapacitabine also produced a statistically significant improvement in CR rate in patients who had prior antecedent hematologic disorder with a CR rate of 16.7% compared to 5.75% for patients taking decitabine alone, although again the combination didn't produce a statistically significant increase in overall survival in those patients

12/13/17

Daiichi Sankyo Co. Ltd. (Tokyo)

DS-Savaysa/Lixiana

Edoxaban

Venous thromboembolism (VTE) associated with primarily active cancer

Results from the Hokusai-VTE CANCER study showed it was deemed noninferior to dalteparin for the composite outcome of first recurrent VTE or ISTH-defined major bleeding during the 12-month study period with a rate of 12.8% for those treated with it compared to 13.5% for the dalteparin group; the trial also met its secondary endpoint of event-free survivalat 12 months with rates of 55% and 56.5% for patients taking edoxaban and dalteparin, respectively

12/13/17

Daiichi Sankyo Co. Ltd. (Tokyo)

DS-3201

EZH1/2 dual inhibitor

Relapsed or refractory non-Hodgkin lymphomas

Phase I data showed it produced an overall response rate (ORR) of 58.8% in the 17 evaluable patients, including one complete remission and nine partial remissions; in the subset of 11 evaluable patients with B-cell lymphomas, the ORR was 45.5% while the ORR in the six patients with T-cell lymphomas was 83.3%

12/13/17

Effector Therapeutics Inc. (San Diego)

eFT-508

Selective translation regulator

Lymphoma

Phase I/II data showed that out of 10 patients treated and evaluable for efficacy, one patient with non-germinal center B-cell like diffuse large B-cell lymphoma experienced a partial response and remains on therapy, while four additional patients experienced stable disease, including a patient with classical Hodgkin lymphoma who had a marked reduction in tumor volume and also remains on therapy

12/12/17

Gamida Cell Ltd. (Jerusalem)

Nicord

Universal bone marrow transplant solution derived from cord blood stem cells

High-risk hematologic malignancies

Phase I/II data showed the median time to neutrophil engraftment was 11 days while the median time to platelet engraftment was 34 days; matched patients from a database of patients treated with unmanipulated cord blood transplantation had a median time to neutrophil engraftment of 21 days and a median time to platelet engraftment of 46 days

12/13/17

Genentech Inc. (unit of Roche Holding AG; Basel, Switzerland)

Polatuzumab vedoti

Anti-CD79b antibody-drug conjugate

Relapsed or refractory diffuse large B-cell lymphoma

The phase II GO29365 study met its primary endpoint, demonstrating that the addition of polatuzumab vedotin to bendamustine plus Rituxan (BR) increased complete response rates from 15% to 40% (p=0.012) at the end of treatment, as measured by positron emission tomography and assessed by an independent review committee; no unexpected safety signals were observed with the addition of polatuzumab vedotin to BR

12/12/17

Geron Corp. (Menlo Park, Calif.)

Imetelstat

Telomerase inhibitor

Lower-risk myelodysplastic syndromes

IMerge phase II/III data showed 38% achieved RBC transfusion independence (TI) of at least eight weeks with a mean relative reduction in transfusion burden from baseline of 64%; in the 13 patients who were naive to Revlimid and HMAs and who lacked del(5q), treatment with it resulted in 54% of patients achieving RBC TI of at least eight weeks with a mean relative reduction in transfusion burden from baseline of 71%

12/13/17

Global Blood Therapeutics Inc. (South San Francisco)

Voxelotor

Hemoglobin modifier

Sickle cell disease (SCD)

Phase IIa Hope-Kids 1 data showed increased hemoglobin levels and improved clinical measures of hemolysis at 16 weeks, as evaluated by changes from baseline in hemoglobin, percent of reticulocytes and percent of unconjugated bilirubin; results are consistent with results previously seen in adults, even though nearly all of the adolescent patients were also receiving hydroxyurea; 55% of patients achieved a hemoglobin response >1 g/dL with a median hemoglobin change of 1.1 g/dL

12/12/17

Glycomimetics Inc. (Rockville, Md.)

GMI-1271

Small molecule E-Selectin antagonist

Relapsed/refractory (r/r) acute myeloid leukemia (AML)

Phase I/II trial showed: for the 54 r/r AML patients treated at the phase II dose, the clinical remission rate was 43% with a median duration of response of 11.1 months; the median overall survival for the r/r AML patients was of 9.4 months; for the 25 older patients with newly diagnosed AML, the clinical remission rate was 68% with a median duration of 14.8 months and a median event-free survival of 11.3 months; in those patients the median overall survival was 15.8 months

12/13/17

Immune Design Inc. (Seattle)

G-100

Intratumoral Toll-like receptor 4 agonist

Follicular non-Hodgkin's lymphoma

Phase II data showed the monotherapy and pembrolizumab combination resulted in a 39% objective response rate (ORR), with a 57% ORR in those patients who expressed a potential predictive biomarker

12/12/17

Immunogen Inc. (Waltham, Mass.)

IMGN-779

CD33-targeting antibody-drug conjugate

Relapsed or refractory adult acute myeloid leukemia (AML)

Phase I data showed that it was well-tolerated with no dose-limiting toxicities observed across nine dose levels administered once every two weeks and one dose level administered once a week; antileukemia activity was seen at doses ≥0.39 mg/kg in both schedules in patients with poor prognostic features; the maximum tolerated dose has not been reached and dose escalation continues

12/12/17

Impact Biomedicines Inc. (San Diego)

Fedratinib

Selective oral small-molecule JAK2 inhibitor

Myelofibrosis

A case review showed, of the eight patients suspected to have Wernicke encephalopathy (WE), thiamine levels and MRI results only supported diagnosis of WE in one patient, who had more than 10% weight loss and had preceding protracted nausea and vomiting before entering the trial, which the company believes was a contributing factor to malnutrition and thiamine deficiency.

12/13/17

Incyte Corp. (Wilmington, Del.)

Jakafi

Ruxolitinib

Polycythemia vera (PV) resistant to or intolerant of hydroxyurea

Four-year data from the phase III RESPONSE study showed a durable primary response and the overall safety profile remained consistent with previously reported 80-week RESPONSE data; in both the Jakafi arm and crossover population, about 30% of patients completed study treatment and 37% of those patients were still receiving treatment

12/12/17

Italfarmaco Group (Milan)

Givinostat

HDAC inhibitor

Polycythemia vera

It was well-tolerated at 100 mg twice daily in a phase Ib/II study and most adverse events were mild to moderate in severity; overall response rate was more than 80%; in a larger study, 80% of patients maintained at least a partial response for more than four years, and none reported microvascular symptoms or headaches during that period; pruritus was absent in 67% of those patients, and the overall incidence of thrombosis (1.13% of patients per year) was reduced in comparison to the expected incidence rate of 3% of patients per year; treatment reduced the levels of mutated JAK2 by 25% in more than 40% of the patients

12/12/17

Janssen-Cilag International NV (Beerse, Belgium; unit of Johnson & Johnson)

Darzalex

Daratumumab

Multiple myeloma

Data from the phase Ib PAVO trial testing the subcutaneous delivery of Darzalex co-formulated with recombinant human hyaluronidase enzyme (daratumumab-SC) in patients with relapsed or refractory multiple myeloma showed the treatment was generally well-tolerated with a 12% rate of infusion-related reactions; data from the phase II CENTAURUS trial testing three dosing schedules for daratumumab monotherapy in patients with intermediate or high-risk smoldering multiple myeloma showed the estimated 12-month progression-free survival rates were 95%, 88% and 81% in the long-intense, intermediate and short dosing schedules, respectively

12/13/17

Juno Therapeutics Inc. (Seattle)

JCAR-017

Lisocabtagene maraleucel

Diffuse large B-cell lymphoma

TRANSCEND study results showed that baseline patient characteristics, including high tumor burden and markers of inflammation, were associated with high CAR T-cell expansion and an approximately eightfold increased risk for cytokine release syndrome and neurotoxicity; baseline markers of inflammation were associated with more durable responses; with respect to tumor burden the association was less pronounced; another analysis showed that JCAR-017 CD4 and CD8 CAR T cells infiltrated tumors post-treatment; CAR T-cell infiltration trended higher in patients who achieved a response; at disease progression, CAR T cells were rare or absent in tumor tissue despite the presence of CD19 and persistence of peripheral blood CAR T cells in most patients; pathways such as PD-L1 and IDO were up-regulated in different patients

12/12/17

Juno Therapeutics Inc. (Seattle) and Celgene Corp. (Summit, N.J.)

JCAR-017

Lisocabtagene maraleucel

Relapsed or refractory aggressive B-cell non-Hodgkin lymphoma

Based on a cut-off date of Oct. 9, at dose level two (DL2 = 100 million cells), the Phase I TRANSCEND data showed a three-month overall response rate of 74% (14/19) and a three-month complete response (CR) rate of 68% (13/19; of patients that reached six months of follow-up, 50% (7/14) were in CR; across doses, 80% (16/20) of patients with CR at three months remained in CR at six months, and 92% (11/12) of patients in response at six months remained in response as of data cut-off; across doses, median duration of response (DOR) was 9.2 months and median durability of CR was not reached

12/12/17

Kadmon Holdings Inc. (New York)

KD-025

Rho-associated coiled-coil kinase 2 inhibitor

Chronic graft-vs.-host disease

Findings from an ongoing phase II trial demonstrated that it was well-tolerated and resulted in clinically meaningful responses; data from the second cohort of the trial (KD-025 200 mg BID; n=16) showed an overall response rate (ORR) of 63% as of data cut-off Nov. 20; updated data from cohort one (KD-025 200 mg QD; n=17) showed an ORR of 65%; responses were rapid with 71% of patients across cohorts one and two achieving a response by the first assessment after eight weeks of treatment; responses also were seen across all affected organs, including complete responses in upper and lower gastrointestinal tract, mouth, skin, joints, esophagus, eyes and liver

12/12/17

Kite Pharma Inc. (Santa Monica, Calif., a Gilead Co.)

KTE-C19

CD19 CAR T-cell therapy

Relapsed or refractory acute lymphoblastic leukemia (ALL)

Phase I/II ZUMA-3 data showed with a minimum of eight weeks of follow-up 71% of ALL patients (n=17/24) who received a single infusion achieved complete tumor remission (complete remission [CR] or CR with incomplete hematological recovery); data also showed 100% of responders had no detectable minimal residual disease, including those with high tumor burden and high-risk genetic abnormalities

12/13/17

Kura Oncology Inc. (San Diego)

Tipifarnib

Selective inhibitor of farnesyl transferase

Bone marrow cancers

Findings support the development of it, including the identification of CXCR4/CXCR2 expression ratio and bone marrow homing of myeloid cells as potential biomarkers of tipifarnib activity across the bone marrow cancers, myelodysplastic syndromes, acute myeloid leukemia and chronic myelomonocytic leukemia, further showing that the CXCL12/CXCR4 pathway is a potential therapeutic target of farnesyl transferase inhibitors

12/12/17

Kyowa Hakko Kirin Co. Ltd. (Tokyo)

KW-0761

Mogamuliz-umab

Cutaneous T-cell lymphoma

Phase III MAVORIC data showed that patients who received it had significantly better progression-free survival (PFS) vs. vorinostat, with a median PFS of 7.7 months vs. 3.1 months (p < 0.0001); global overall response rate was 28% vs. 4.8% for mogamulizumab and vorinostat, respectively, and patient-reported outcomes, as measured by the Skindex-29 and FACT-G, showed significantly greater symptom reduction and improved functional status in favor of mogamulizumab vs. vorinostat (p<0.05)

12/12/17

Macrogenics Inc. (Rockville, Md.)

Flotetuz-umab

CD123 x CD3 bispecific DART protein

Relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome

Phase I data showed it continues to demonstrate acceptable tolerability; of the eight evaluable patients in the dose-expansion cohort who received a lead-in dose followed by 500 ng/kg/day via continuous I.V. infusion, six patients (75%) have evidence of antileukemic activity, with three of those patients experiencing an objective response; the duration of response for the eight patients ranged from one month to 5.8 months, with five of those responses still ongoing as of the Nov. 30 cut-off

12/12/17

Magenta Therapeutics Inc. (Cambridge, Mass.)

MGTA-456

Single umbilical cord blood unit expanded with an aryl hydrocarbon receptor antagonist

Hematologic malignancies

Phase II data showed that the median increase in the number of CD34-positive cells was 327-fold; transplanted cells successfully engrafted in 18 of 18 patients, with rapid time to engraftment and immune recovery, regardless of conditioning intensity, compared to historical controls; complete chimerism for patients treated with it was rapid, with no late graft failures; low rates of graft-vs.-host disease and disease relapse also were observed with MGTA-456 vs. historical cohorts

12/12/17

Miragen Therapeutics Inc. (Boulder, Colo.)

MRG-106

RNA candidate

Mycosis fungoides form of cutaneous T-cell lymphoma

Phase I data showed that nine of 14 patients (64%) treated for more than one month showed a 50% or greater improvement in total skin disease as measured by the maximal change in each patient's modified Severity Weighted Assessment Tool (mSWAT) score; seven patients had maintained a durable response for four months or longer

12/13/17

Mustang Bio Inc. (New York)

MB-102

CD123 CAR T therapy

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) and acute myeloid leukemia (AML)

Phase I data showed it is safe and well-tolerated and achieved the first-ever complete response (CR) from a CAR T therapy in BPDCN as well as a CR in AML; one of the two patients treated at dose level one (50M CAR+ T) achieved a morphologic leukemic-free state at day 28 post-infusion; two patients receiving dose level two (200M CAR+ T) had CRs at day 28 (one CR and one CR with incomplete blood count recovery); infusions of up to 200M CAR T cells were safe, with no graft-vs.-host disease, myeloablative effects, neurologic toxicity or dose-limiting toxicities

12/13/17

Nordic Nanovector ASA (Oslo, Norway)

Betalutin

177Lu-satetrax-etan-lilotomab

Relapsed/refractory indolent non-Hodgkin lymphoma (iNHL)

Phase I/II data showed that single-agent treatment was effective and well-tolerated; 90% of patients (n=59) had a reduction in tumor size, and the overall response rate was 60%, with a complete response rate of 24% for all evaluable iNHL patients

12/12/17

Novartis AG (Basel, Switzerland)

Crizanliz-umab

Humanized anti-P-selectin monoclonal antibody

Sickle cell disease

Analysis of the phase II SUSTAIN study showed that it delayed time to first sickle cell pain crisis (SCPC) in patients vs. placebo in key subgroups of adults with sickle cell disease; the analysis showed 5 mg/kg per month increased the estimated median time to first SCPC vs. placebo by about twofold or more in all groups; in patients with the HbSS genotype, there was a 3.7-fold increase in estimated median time to first SCPC in those taking it vs. placebo, while in patients taking hydroxyurea, the time to first on-study SCPC was longer with it vs. placebo, suggesting its potential as an additive therapy

12/12/17

Onconova Therapeutics Inc. (Newtown, Pa.)

Rigosertib

RAS-targeting small-molecule

Myelodysplastic syndromes

Phase II data showed that out of 62 evaluable patients receiving it, 20 patients (32%) achieved transfusion independence (TI) lasting eight to more than 85 weeks, with a median of 18 weeks; the highest rate of TI (44%) was observed in the 560 mg BID intermittent cohort: 15 of 34 eligible patients achieved TI lasting eight to 85-plus weeks, with a median of 18 weeks; 93% of those 15 patients received it with continued erythropoiesis-stimulating agent

12/13/17

Pharmaessentia USA (Waltham, Mass., subsidiary of Pharmaessentia Corp.)

Ropeginter-feron alfa-2b

Long-acting, monopegylated proline interferon

Polycythemia vera

Follow-up results of the CONTINUATION-PV trial showed at 24 months treatment with it achieved a significantly higher complete hematologic response (CHR) of 70.5%, compared to CHR of 49.3% with hydroxyurea (HU) or best available treatment (BAT) (p=0.0101); durable clinical and hematological response rates increased steadily in treated patients over the two-year treatment period, in contrast to HU/BAT; the composite endpoint, CHR including disease symptom improvement, also was higher in treated patients vs. HU/BAT at 24 months (49.5% vs. 36.6%; p=0.1183)

12/12/17

Pharmamar SA (Madrid, Spain)

Plitidepsin

Targets the eEF1A2 protein

Relapsed or relapsed and refractory multiple myeloma

ADMYRE phase III data showed: in the group of patients who received it with dexamethasone (n=171), a 3.8-month progression-free survival (PFS) was observed in comparison to 1.9 months on dexamethasone alone; PFS was 2.6 months for the combination vs. 1.7 months for the dexamethasone monotherapy arm; it showed a statistically significant reduction in the risk of progression over the comparator, meeting the primary endpoint; a statistically significantly superior increase in overall survival in the combination arm was observed at 11.6 months for it and 6.4 months for the comparator

12/12/17

Spark Therapeutics Inc. (Philadelphia) and Pfizer Inc. (New York)

SPK-9001

Gene therapy

Hemophilia B

All 11 participants in the ongoing phase I/II trial had discontinued routine infusions of factor IX concentrates and shown sustained steady-state factor IX activity levels with no serious adverse events, thrombotic events or factor IX inhibitors observed; the overall annualized bleeding rate was reduced by 97% to a mean of 0.3 (0.5) annual bleeds, compared to a mean of 10.5 bleeds annually before SPK-9001 administration; overall annualized infusion rate was reduced 99% to a mean of 0.8 (1.7) annual infusions, compared to a mean of 62.5 infusions per year before SPK-9001 administration

12/12/17

TG Therapeutics Inc. (New York)

TGR-1202

PI3K delta inhibitor

Relapsed or refractory hematologic malignancies

Data pooled from five completed or ongoing phase I or phase II studies showed of the 347 patients treated, the overall response rate (ORR) was 85% for single-agent treatment in relapsed/refractory chronic lymphocytic leukemia (CLL) patients and 52% in relapsed/refractory follicular lymphoma patients; data from a phase II study testing it in patients with chronic CLL who are intolerant to prior BTK or PI3K inhibitor therapy showed a 94% (30 of 32 patients) progression-free response; data from a phase I/II study testing TG-1101 (ublituximab), TGR-1202 and Keytruda in patients with relapsed/refractory CLL showed an ORR of 78% (seven of nine patients), with a 75% ORR (three of four) in patients refractory to BTK inhibitor therapy

12/12/17

Trillium Therapeutics Inc. (Toronto)

TTI-621

CD47-blocking agent

Relapsed/refractory mycosis fungoides, Sezary syndrome and advanced, relapsed/refractory diffuse large B-cell lymphoma

Phase Ia/Ib data showed that a single direct intratumoral injection induced antitumor activity; a rapid reduction in CAILS scores, which measures local lesion responses, was observed in nine out of 10 mycosis fungoides patients and a reduction in circulating leukemic Sézary cells was observed in three out of three patients; it induced objective responses in patients with advanced, relapsed/refractory diffuse large B-cell lymphoma; in combination with Rituxan, treatment resulted in objective responses in five out of 18 evaluable patients with heavily pre-treated disease

12/12/17

Uniqure NV (Amsterdam, the Netherlands)

AMT-060

Gene therapy

Severe hemophilia B

Eighteen-month follow-up phase I/II data from the second-dose cohort continue to show stable factor IX (FIX) activity with substantial improvement in disease state in all five patients, including the discontinuation of routine prophylactic FIX infusions in all patients that previously required chronic replacement therapy; the annualized spontaneous bleeding rate for the second dose cohort declined 89% to a mean of 0.3 bleeds after gene transfer; in the last year of follow-up, no patient in the second cohort has reported any spontaneous bleeds

12/12/17

Verastem Inc. (Boston)

Duvelisib

Oral dual inhibitor of PI3K-delta and PI3K-gamma

Relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL)

The phase III DUO study met its primary endpoint with the monotherapy achieving a statistically significant improvement in median progression-free survival (mPFS) compared to ofatumumab per a blinded independent review committee (IRC) using iwCLL or revised IWG Response Criteria (modified iwCLL/IWG; 13.3 months vs. 9.9 months, respectively; HR=0.52; p<0.0001), representing a 48% reduction in the risk of progression or death; similar efficacy of duvelisib was observed regardless of whether patients had 17p deletion; the primary outcome of mPFS via IRC review in the del[17p] subpopulation significantly favored duvelisib over ofatumumab (12.7 months vs. nine months, respectively; HR=0.41; p=0.0011), representing a 59% reduction in the risk of progression or death; per investigator assessment, duvelisib demonstrated a mPFS of 17.6 months, compared to 9.7 months for ofatumumab (HR=0.40, p<0.0001)

12/12/17

Verastem Inc. (Boston)

Duvelisib

Oral PI3K delta/gamma inhibitor

Relapsed or refractory T-cell lymphomas

Phase I data showed in arm A, which combined it with Velcade, 15 patients were evaluable for efficacy, with nine showing responses (four complete responses and five partial responses) for an overall response rate (ORR) of 60%; in arm B, which tested it with Istodax, 17 were evaluable for efficacy, with six responding (three complete responses and three partial responses) for an ORR of 35%

12/13/17

X4 Pharmaceuticals Inc. (Cambridge, Mass.)

X4P-001-RD

CXCR4 inhibitor

WHIM syndrome

Preliminary data from the phase II portion of an ongoing phase II/III study demonstrated promising activity with dose-dependent increases in neutrophil and lymphocyte counts; all patients enrolled in the study to date had meaningful increases in the levels of circulating white blood cells with daily oral administration of it, and it was considered to be safe and well-tolerated

12/12/17

Notes

The date indicated refers to the BioWorld issue in which the news item can be found.

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