Company (location)

Product

Description

Indication

Status

Date


AB Science SA (Paris)

Masitinib

Tyrosine kinase inhibitor

Severe systemic mastocytosis

Data analyses from its phase III trial showed a significant improvement over placebo, with a cumulative response of 18.7% vs. 7.4%, respectively (p=0.008); the primary endpoint was based on repeated measures methodology for rare diseases via the generalized estimating equation model; computing treatment effect according to cumulative response per patient confirmed the outcome on the primary endpoint: 26.7% vs. 12.8%, respectively (p=0.0212); computing treatment effect according to individual patient response (Pearson chi-square) was also significant for masitinib: 40.3% vs. 24.2%, respectively (p=0.0062); response per patient on all severe baseline symptoms for at least one visit was: 16.4% for masitinib vs. 1.6% for placebo (p=0.0062).

6/23/17

Abbvie Inc. (North Chicago)

Venclyxto

Oral B-cell lymphoma-2 inhibitor

Relapsed/refractory chronic lymphocytic leukemia and 17p deletion

Results from a pivotal phase II study showed it produced an overall response – the primary endpoint, defined as complete remissions (CR), complete remission with incomplete marrow recovery (Cri), partial remission and nodular partial remission (nPR) – in 77% of patients; CR+Cri occurred in 18 percent of patients, the secondary endpoint; for the exploratory endpoints, 53% achieved a partial remission, 6% achieved an nPR and 27% achieved blood minimal residual disease negativity, as measured by flow cytometry

6/26/17

Acceleron Pharma Inc. (Cambridge, Mass.)

Luspatercept

Modified activin receptor type IIB fusion protein

Beta-thalassemia

Preliminary results from an ongoing phase II trial showed it reduced red blood cell (RBC) transfusion burden by at least 33% in any of the 12-week treatment interval as compared to baseline in 69% of the 32 patients; at the fixed periods for weeks 13 to 24 and weeks 37 to 48 compared to the baseline, 50% and 46% of patients achieved at least a 33% reduction in RBC transfusion burden respectively, and, of the 31 non-transfusion-dependent beta-thalassemia patients, 71% achieved a clinically meaningful increase in hemoglobin of at least 1.0 g/dL compared to baseline after treatment with luspatercept

6/26/17

Acceleron Pharma Inc. (Cambridge, Mass.)

Luspatercept

Modified activin receptor type IIB fusion protein

Lower-risk myelodysplastic syndromes

Preliminary results from its ongoing phase II studies showed it produced 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 in 50% of patients; additionally, 38% of luspatercept-treated patients with RBC units greater than or equal to two at baseline achieved RBC transfusion independence for at least eight weeks

6/26/17

Agios Pharmaceuticals Inc. (Cambridge, Mass.)

AG-348

Pyruvate kinase-R activator

Pyruvate kinase deficiency

Updated data from its DRIVE PK phase II trial showed AG-348 increased the maximum Hb from baseline by more than 1.0 g/dL in 48% of all 52 treated patients and 57% of the 42 patients with at least one missense mutation

6/27/17

Agios Pharmaceuticals Inc. (Cambridge, Mass.)

Idhifa

Enasidenib

Relapsed or refractory acute myeloid leukemia (R/R AML)

Data from its ongoing phase I/II dose-escalation and expansion study evaluating Idhifa in patients who have an isocitrate dehydrogenase-2 mutation (IDH2), which Idhifa inhibits, showed it produced an overall response rate of 37%, including a complete response rate of 20.1% in 214 patients

6/27/17

Akari Therapeutics plc (London)

Coversin

Second-generation complement inhibitor

Paroxysmal nocturnal hemoglobinuria

Data for the first four patients in its ongoing phase II trial showed that none of the patients taking Coversin required transfusions during the 90-day trial, compared to three of the four patients during the three months preceding the trial

6/26/17

AOP Orphan Pharmaceuticals AG (Vienna) and Pharmaessentia Corp. (Taipei, Taiwan)

Ropeginter-feron alfa-2b

Long-acting, mono-pegylated proline interferon

Polycythemia vera

Data from three clinical trials showed that in one study, after four years of treatment, a majority of the 29 patients showed a sustained reduction of the mutant JAK2V617F allelic burden to below 10%, demonstrating the disease modifying capability of Ropeginterferon alfa-2b treatment; another study demonstrated the ability of Ropeginterferon alfa-2b to be self-injected via a pen injection at home in 36 patients; in a third study, PROUD-PV, it decreased mutated hematopoietic bone marrow progenitor cells by a median of 64% compared to 25% for the control group treated with hydroxyurea

6/27/17

Bellicum Pharmaceuticals Inc. (Houston)

BPX-501

Adjunct T-cell therapy comprised of genetically modified donor T cells incorporating Bellicum's CaspaCIDe safety switch

Acute leukemias

Data from the phase I/II BP-004 trial of BPX-501 and rimiducid in a cohort of 47 pediatric patients who lack a matched donor showed the cumulative incidence of grade two-four acute graft-vs.-host disease (GvHD) was 13% six months after treatment with BPX-501 following an alpha/beta T-cell depleted haploidentical hematopoietic stem cell transplant; by one year of follow-up, the cumulative incidence of chronic GvHD was 3%

6/26/17

Bluebird Bio Inc. (Cambridge, Mass.)

Lentiglobin

Gene therapy product candidate

Transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD)

Data from the ongoing phase I/II HGB-205 study showed all four patients with TDT remain free of transfusions and have stable production of HbAT87Q, the gene transferred during the therapy, through 3.5 years; the first SCD patient treated is producing 50% HbAT87Q, above the approximately 30% anti-sickling hemoglobin level predicted to have potential clinical impact on the disease, but had a vaso-occlusive crisis and subsequent hospitalization approximately 30 months after treatment; data on two additional SCD patients were reported with 20% and 15% HbAT87Q expression 6.1 months and 3.4 months after treatment respectively

6/26/17

Bluebird Bio Inc. (Cambridge, Mass.)

Lentiglobin

Gene therapy product candidate

Transfusion-dependent beta-thalassemia in patients with non-β0/β0 genotypes

Data from its phase III Northstar-2 (HGB-207) study showed the new manufacturing protocol produced a median DP vector copy number (VCN) of 3.0 with a range between 2.4 and 4.0 in the first six patients manufactured, compared to a median DP VCN of 0.7 with a range between 0.3 and 1.5 in the original Northstar study

6/26/17

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

Empliciti

Elotuzumab

Relapsed/refractory multiple myeloma

Four-year follow-up data from the phase III ELOQUENT-2 study testing Empliciti combined with lenalidomide/dexamethasone (Ld) showed that, four years after the start of treatment, Empliciti+Ld maintained a reduction in the risk of disease progression or death of 29% compared to Ld; at that time point, the progression-free survival rate for Empliciti+Ld was 21% compared to 14% for Ld

6/27/17

Global Blood Therapeutics Inc. (South San Francisco)

GBT440

A hemoglobin modifier

Sickle cell disease

Preliminary results from the single-dose adolescent (12 to 17) cohort of the ongoing HOPE-KIDS 1 phase IIa study showed that the pharmacokinetics of GBT440 are similar in adolescents and adults; based on these results, the company is expanding the trial to evaluate GBT440 administered at doses of 900 mg and 1,500 mg per day, consistent with the doses currently being administered in the phase III HOPE Study

6/26/17

Immunogen Inc. (Waltham, Mass.)

IMGN779

Anti-CD33 antibody drug conjugate

Relapsed or refractory adult acute myeloid leukemia

Data from a dose-escalating phase I trial testing IMGN779 in patients whose tumors express CD33 showed it produced anti-leukemia activity at dose levels six and seven in patients who failed intensive frontline therapy

6/27/17

Karyopharm Therapeutics Inc. (Newton, Mass.)

Selinexor (KPT-330)

Selective Inhibitor of Nuclear Export (SINE) compound that inhibits the nuclear export protein XPO1

Relapsed or refractory diffuse large B-cell lymphoma

An update on its ongoing phase IIb Selinexor Against Diffuse Aggressive Lymphoma (SADAL) study showed it produced an overall response rate (ORR) of 33.3% in the 63 patients in the interim analysis cohort; ORR of the 32 patients who received the 60-mg dose (34.4%) was similar to the 31 patients receiving 100 mg (32.2%)

6/26/17

Novartis AG (Basel, Switzerland)

CTL019

Tisagenlecleucel; chimeric antigen receptor T cell therapy

Relapsed/refractory B-cell acute lymphoblastic leukemia

Follow-up data from its pivotal phase II ELIANA clinical trial in pediatric and young adult patients showed it produced a complete remission (CR) or CR with incomplete blood count recovery within three months of infusion in 83% of the treated patients; the relapse-free probability was 75% at six months and 64% at 12 months among responders and the probability of survival was 89% at six months and 79% at 12 months

6/26/17

Novartis AG (Basel, Switzerland)

Tasigna

Nilotinib

Philadelphia chromosome-positive chronic myeloid leukemia

Data from the ENESTfreedom and ENESTop phase II trials testing Tasigna showed that more than 90% of patients in the chronic phase who stopped Tasigna and were in Treatment-free Remission (TFR) at 48 weeks remained in TFR at 96 weeks

6/26/17

Onconova Therapeutics Inc. (Newtown, Pa.)

Rigosertib

A dual P13 kinase and pololike kinase inhibitor

Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS)

Data from a trial testing oral rigosertib with azacitidine showed the eight AML patients who were evaluable for response had an overall response rate (ORR) of 37.5%; among 33 evaluable MDS patients, the ORR was 76% including 24% complete remission and 30% concurrent marrow CR and hematologic improvement

6/27/17

Stemline Therapeutics Inc. (New York)

SL-401

Targeted therapy directed to the interleukin-3 receptor

Blastic plasmacytoid dendritic cell neoplasm

Updated data from stages one and two of its ongoing pivotal phase II clinical trial testing showed it produced an overall response rate of 84% in the 32 patients, with a complete response rate of 59%, by investigator-assessment

6/26/17

Sunesis Pharmaceuticals Inc. (South San Francisco)

Qinprezo

Vosaroxin; quinolone derivative that intercalates DNA and inhibits topoisomerase II

Myelodysplastic syndrome

Data from a phase I cohort expansion trial of Qinprezo plus Vidaza (azacitidine, Celgene Corp.) showed that, of the 32 patients that completed at least one cycle and were evaluable for response, eight (25%) had a complete response and five (15.6%) had a complete remission

6/26/17

Takeda Pharmaceutical Co. Ltd. (Osaka, Japan)

Ninlaro

Ixazomib

Newly diagnosed multiple myeloma

Data from two phase I/II trials showed that, in the trial of weekly dosing, Ninlaro plus Revlimid (lenalidomide, Celgene Corp.) and dexamethasone produced a confirmed overall response rate (ORR) of 80%; in a second trial testing twice weekly dosing of the same agents, the ORR was 92%; during the maintenance phase of the two trials testing Ninlaro alone, 32% of patients treated with weekly dosing and 22% of patients with twice-weekly dosing improved their responses during maintenance

6/26/17

TG Therapeutics Inc. (New York)

TGR-1202 and TG-1101

Umbralisib, next-generation PI3K delta inhibitor, and ublituximab, glycoengineered anti-CD20 monoclonal antibody

Chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL) and non-Hodgkin lymphoma (NHL)

Data from the combination testing TGR-1202, TG-1101 and Imbruvica (ibrutinib, Abbvie Inc. and Johnson & Johnson) showed clinical activity observed at all dose levels, with 36 of 38 patients evaluable for efficacy; data showed a 100% (19 of 19) overall response rate, including a 32% complete response rate, in patients with CLL/SLL; in NHL, response rates were 100% (two of two) in marginal zone lymphoma patients; 100% (four of four) in mantle cell lymphoma patients; 80% (four of five) in follicular lymphoma; and 17% in patients with diffuse large B-cell lymphoma

6/27/17

Verastem Inc. (Boston)

Duvelisib

Dual inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma

Non-Hodgkin lymphoma

Long-term follow-up data from the Dynamo study showed it met its primary endpoint of overall response rate (ORR; p=0.0001) at the final analysis

6/27/17


Notes

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

For more information about individual companies and/or products, see Cortellis.