61st American Society of Hematology annual meeting in Orlando, Fla. – Dec. 7-10, 2019

Company Product Description Indication Status
Achillion Pharmaceuticals Inc., of Blue Bell, Pa. Danicopan (ACH-4471) Oral, small-molecule factor D inhibitor Paroxysmal nocturnal hemoglobinuria Top-line data showed dose-finding phase II study in combination with eculizumab in patients who have inadequate response to C5 monotherapy met primary endpoint, showing mean increase of 2.4 g/dL at 24 weeks; treatment also resulted in significant reduction in blood transfusions, with 10 patients receiving 34 infusions (58 units) in 6 months prior to screening and 1 patient receiving 1 transfusion (2 units) during the 24-week trial 
Actinium Pharmaceuticals Inc., of New York Actimab-A Antibody radiation conjugate that delivers alpha-emitting radioisotope Actinium-225 via the antibody lintuzumab to cells that express CD33 Relapsed or refractory acute myeloid leukemia Phase I combination trial of Actimab-A with the salvage chemotherapy regimen CLAG-M (cladribine, cytarabine and filgrastim with mitoxantrone) showed an 86% overall response rate, which is 56% higher than what was observed with CLAG-M and MEC (etoposide, cytarabine and mitoxantrone hydrochloride) and more than double that of CLAG in a similar patient population; 71% of patients (5 of 7) achieved negative minimal residual disease status in the second dose cohort
ADC Therapeutics SA, of Lausanne, Switzerland ADCT-402 (loncastuximab tesirine) Antibody-drug conjugate composed of humanized monoclonal antibody directed against human CD19 Relapsed or refractory diffuse large B-cell lymphoma Interim efficacy and safety data on 52 patients in the ongoing pivotal 145-patient phase II trial showed the overall response rate was 46.2%, including 19.2% complete responses and 26.9% partial responses; stable disease was attained in 19.2% of patients and toxicity was manageable; company plans a BLA submission for the second half of 2020
Amgen Inc., of Thousand Oaks, Calif. Kyprolis (carfilzomib) Blocks proteasomes Relapsed or refractory multiple myeloma Phase III Candor study of Kyprolis in combination with dexamethasone and Darzalex (daratumumab) (KdD arm) compared to Kyprolis and dexamethasone alone (Kd arm) showed it met the primary endpoint of progression-free survival, resulting in a 37% reduction in the risk of disease progression or death; median PFS was not reached for the KdD arm vs. 15.8 months for the Kd arm
Amphivena Therapeutics Inc., of South San Francisco AMV-564 Bivalent T-cell engager Relapsed/refractory acute myeloid leukemia Phase I data provide early evidence of safety and clinical activity and T-cell activation, as well as evidence that it increases in bone marrow T cells and has antileukemic blast activity; of 38 patients, 5 had complete or partial responses, and it was shown to be safe and well-tolerated with no dose-limiting toxicities up to 450 mcg/day
Aprea Therapeutics Inc., of Boston APR-246 Small molecule designed to reactivate mutant and inactivated p53 protein TP53-mutated myelodysplastic syndromes and acute myeloid leukemia Data from French phase Ib/II trial in combination with azacitidine showed, as of data cutoff, overall response rate in 24 evaluable MDS patients was 74%, with 66% complete remission rate; with median duration of follow-up of 6.4 months, median overall survival for all enrolled patients not yet reached; data from U.S. phase Ib/II trial showed, as of data cutoff, ORR in 33 evaluable MDS patients was 88%, with 61% CR rate; with median duration of follow-up of 10.8 months, median duration of response was 8.4 months and median duration of CR was 7.3 months; median OS for all enrolled patients was 10.8 months
Aptose Biosciences Inc., of San Diego CG-806 FLT3/BTK inhibitor Chronic lymphocytic leukemia Preclinical data comparing CG-806 to ibrutinib showed drug broadly inhibits B-cell receptor signaling in CLL cells, resulting in CLL cell apoptosis and reduced proliferation; CG-806 showed to be more potent than ibrutinib to induce apoptosis of MEC1 CLL cells
Aptose Biosciences Inc., of San Diego CG-806 FLT3/BTK inhibitor Mantle cell lymphomas Preclinical data showed superior antilymphoma effects vs. ibrutinib, exerting potent cell growth inhibitory effects in ibrutinib-resistant MCL cells; CG-806 suppresses phosphor-BTK, -Stat3, -AKT, -ERK, -Src, NF-kB and anti-apoptotic protein Mcl1, while up-regulating p53; also increases autophagy in MCL cells and up-regulates CXCR4/E-selectin levels in MCL cells
Astex Pharmaceuticals Inc., of Pleasanton, Calif. ASTX-727 Fixed-dose combination of cedazuridine and decitabine Intermediate and high-risk myelodysplastic syndromes Top-line data from the Ascertain phase III trial show it achieved its primary objective of decitabine systemic exposure equivalence between oral ASTX-727 and intravenous decitabine with an oral/I.V. ratio of about 99%; Astex plans to file NDA by the end of 2019
Atara Biotherapeutics Inc., of South San Francisco Tab-cel (tabelecleucel) Allogeneic T-cell immunotherapy Epstein-Barr virus-associated post-transplant lymphoproliferative disease  Long-term results from expanded access protocol study showed high overall response rate, short time to response and favorable estimated long-term overall survival rates in patients with EBV+ PTLD following hematopoietic cell transplant (HCT) or solid organ transplant (SOT) who have failed rituximab-based therapy; median time to response of 1 month in both patient cohorts; in responders, 2-year estimated OS rate was 86% for HCT and 100% for SOT
Beyondspring Inc., of New York Plinabulin Marine-derived small molecule that sequesters tubulin heterodimers Chemotherapy-induced neutropenia Phase III Study 106 trial design provides rationale for combining plinabulin with pegfilgrastim (Neulasta) due to their differing mechanisms of action for preventing CIN
Bluebird Bio Inc., of Cambridge, Mass.   Lentiglobin Gene therapy Beta-thalassemia Clinical data support potential benefits and a consistent safety profile across a broad range of transfusion-dependent β-thalassemia genotypes and patient populations, including pediatric patients, with the longest duration of follow-up beyond 5 years; patients have achieved and maintained transfusion independence, improvements in multiple markers of bone marrow red blood cell production and reductions in iron overload
Bluebird Bio Inc., of Cambridge, Mass., and Bristol-Myers Squibb, of Princeton, N.J. Bb-2217 BCMA-targeted CAR T-cell therapy Relapsed/refractory multiple myeloma Phase I safety and efficacy results show a median duration of response of 11.1 months; CAR T persistence was observed in 8 of 10 evaluable responders at month 6, and 2 of 2 evaluable responders at month 18; adverse events were consistent with known toxicities of CAR T therapies
Bristol-Myers Squibb Co., of Princeton, N.J.  CC-486 Oral hypomethylating agent  Acute myeloid leukemia Data from the Quazar AML-001 phase III trial showed a significant improvement in overall survival compared with placebo (24.7 months vs. 14.8 months, p=0.0009) in front-line patients; CC-486 had a manageable safety profile
Bristol-Myers Squibb Co., of Princeton, N.J., and Acceleron Pharma, of Cambridge, Mass. Reblozyl (luspatercept-aamt) Erythroid maturation agent Myelofibrosis-associated anemia Phase II data show that 14% (cohort 1) and 21% (cohort 3A) of anemia-only patients met the primary endpoint of hemoglobin increase of at least 1.5 g/dL from baseline for at least 12 consecutive weeks at every assessment within the first 24 weeks on the study, in the absence of any red blood cell transfusions
Bristol-Myers Squibb Co., of Princeton, N.J., and Acceleron Pharma, of Cambridge, Mass. Reblozyl (luspatercept-aamt) Erythroid maturation agent Myelodysplastic syndromes Pivotal phase III Medalist study data showed that 47.7% of treated patients and 15.8% of placebo patients achieved at least 1 episode of red blood cell transfusion independence lasting at least 8 weeks at any point in the study
Chimerix Inc., of Durham, N.C. Dstat (formerly CX-01) Dociparstat sodium Refractory myelodysplastic syndrome and acute myeloid leukemia Phase II data show that of 15 evaluable patients that received a median of 3 cycles of Dstat and azacitidine, there was 1 complete remission and 3 bone marrow complete remissions, 9 stable disease and 2 progressive disease for an overall response rate of 27%; the median overall survival was 221 days and not significantly different between AML (221 days) and MDS (248 days)
Constellation Pharmaceuticals Inc., of Cambridge, Mass. CPI-0610 BET protein inhibitor  Myelofibrosis Data from phase II Manifest trial in combination with Jakafi (ruxolitinib, Incyte Corp.) in JAK inhibitor-naïve patients showed 12 of 15 in first-line treatment arm achieved at least at 35% spleen volume response at 12 weeks
Cstone Pharmaceuticals, of Suzhou, China CS-1001 Monoclonal antibody directed against PD-L1 Relapsed or refractory extranodal natural killer T-cell lymphoma Phase II CS1001-201 data show that 13 (40.6%) of 32 enrolled patients remained on treatment, and 19 (59.4%) discontinued treatment due to radiographic disease progression, adverse events and nonradiographic symptomatic progression; 33.3% achieved a complete response and 3 achieved a partial response for an overall response rate of 43.3%
CTI Biopharma Corp., of Seattle Pacritinib Oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R Thrombocytopenic myelofibrosis PAC203 data demonstrate pacritinib 200 mg twice-daily is well-tolerated with clinical benefit in the highest-risk patient population; mutational analyses of phase II PAC203 patients demonstrate benefit in patients with high mutational risk and low JAK2 allele burden; Pacifica phase III trial is underway
Editas Medicine Inc., of Cambridge, Mass. EDIT-301 Gene editing medicine that uses CRISPR/Cpf1 (formerly Cas12a) Sickle cell disease and beta-thalassemia In vivo proof-of-concept data show HbF levels in human red blood cells were increased by about 50 percentage points above background at 16 weeks post-engraftment with pancellular distribution and no lineage skewing; the elevated levels were observed after editing with Cas12a
Forma Therapeutics Inc., of Watertown, Mass. FT-4202 Selective red blood cell pyruvate kinase-R activator Sickle cell disease Phase I data in healthy volunteers showed linear and time-independent pharmacokinetics demonstrating proof of mechanism based on mean maximum % of change in key pharmacodynamic measures from baseline, achieved within 24 hours of single dose and sustained for the multiple ascending-dose 14-day dose period; showed maximal 2,3-DPG response at doses ≥ 150 mg twice daily or 400 mg once daily in HV RBCs
Forma Therapeutics Inc., of Watertown, Mass. Olutasidenib Next-generation inhibitor of mutated IDH1 IDH1m acute myeloid leukemia and IDH1m myelodysplastic syndrome Phase I/II results from ongoing study showed drug was well-tolerated as monotherapy and in combination with azacitidine; demonstrated clinical activity in a high-risk phase I AML population and induced IDH1 mutation clearance in percentage of patients with TN and R/R AML regardless of IWG response
Forty Seven Inc., of Menlo Park, Calif. FSI-174 and magrolimab Anti-cKIT antibody and anti-CD47 antibody Conditioning regimen for stem cell transplantation Preclinical data in nonhuman primates showed all-antibody conditioning regimen significantly depleted hematopoietic stem cells from bone marrow, with no dose-limiting toxicities
Gamida Cell Ltd., of Boston GDA-201 Natural killer cell-based cancer immunotherapy Non-Hodgkin lymphoma and multiple myeloma Phase I data show GDA-201 in combination with monoclonal antibodies was generally well-tolerated and demonstrated early evidence of clinical activity in heavily pretreated patients, including 5 complete responses and 1 partial response observed among 9 patients with NHL, and 1 complete response and 5 stable disease patients were seen in the MM group; company plans to start a phase I/II multidose study in patients with NHL in 2020
Gracell Biotechnologies Co. Ltd., of Suzhou, China Fastcar-19 Genetically modifies a patient's T cells to express CD19-specific CAR B-cell acute lymphoblastic leukemia Treatment efficacy showed 34 (97.1%) patients achieved a complete remission with or without complete blood count recovery on day 28; 32 (91.4%) achieved minimum residual disease-negative complete remission; all 37 patients tolerated the single infusion at different dose levels with no dose-limiting toxicities observed
Gracell Biotechnologies Co. Ltd., of Suzhou, China Dual CAR-19-22 GC022, targets CD19 and CD22 B-cell acute lymphoblastic leukemia Treatment efficacy in 20 patients with a 28-day follow-up showed 4 in the low-dose group reported no response and 15 of 16 (93.8%) in the mid- and high-dose groups achieved complete remission
Gracell Biotechnologies Co. Ltd., of Suzhou, China Dual CAR-BCMA-19 GC012, targets BCMA and CD19 Multiple myeloma Demonstrated it was effective in eliminating MM tumor cells both in vitro and in vivo; the first-in-human study showed a good safety profile
Immunogen Inc., of Waltham, Mass. IMGN-632 CD123-targeting antibody-drug conjugate Relapsed/refractory acute myeloid leukemia Phase I data show it displays a well-tolerated safety profile and activity at doses up to and including 0.09 mg/kg per cycle; 38 (54%) of evaluable patients had a reduction in bone marrow blasts and 13 (18%) achieved an objective response, including 2 complete remissions and 10 with incomplete recovery and 1 morphologic leukemia-free state in heavily pretreated patients; 92% had failed prior intensive therapies
Innovent Biologics Inc., of Suzhou, China IBI-326 Fully human BCMA-targeting CAR T cells Relapsed/refractory multiple myeloma With 17 evaluable patients, the objective response rate was 100% in the investigator-initiated trial; 70.6% of patients achieved a best response of stringent complete or complete response, and 88.2% achieved a best response of very good partial response or better
Janssen Pharmaceutical, unit of Johnson & Johnson, of New Brunswick, N.J. Darzalex (daratumumab) CD38-directed antibody Multiple myeloma Phase III Alcyone study showed the addition of Darzalex to bortezomib, melphalan and prednisone (D-VMP) improved the overall survival rate (75% vs. 62%) in newly diagnosed, transplant-ineligible patients, with a 40% reduction in the risk of death compared to VMP alone; the addition of Darzalex resulted in higher rates of minimal residual disease negativity
Kezar Life Sciences Inc., of South San Francisco KZR-261 Sec61 translocon inhibitor Tumors Preclinical data demonstrated high degrees of potency against large number of therapeutically relevant oncology and immuno-oncology targets that are Sec61 client proteins, translating into broad antitumor activity; Sec61 inhibitors showed efficacy in vitro and in vivo against multiple hematologic tumor types without inducing cell death in normal cells or significant toxicity in animals
Kite, a unit of Gilead Sciences Inc., of Foster City, Calif. KTE-X19 CD19 CAR T-cell therapy Relapsed/refractory mangle cell lymphoma Results from phase II Zuma-2 trial showed, after single infusion, the best objective response via independent radiologic central review was 93%, with 67% having achieved complete response; 12-month estimates of progression-free survival and overall survival were 61% and 83%, respectively
Kite, a unit of Gilead Sciences Inc., of Foster City, Calif. Yescarta (axicabtagene ciloleucel)  CD19-targeting CAR T therapy Relapsed or refractory large B-cell lymphoma Findings from ongoing postmarketing study showed efficacy and safety of Yescarta comparable to that observed in ZUMA-1 trial, despite a larger proportion of older, more difficult-to-treat patients in the real-world setting
Kymera Therapeutics Inc., of Cambridge, Mass. KYM-003 Selective degrader of STAT3 Hematologic malignancies  Preclinical data showed activity across multiple hematologic malignancies, including ALK-positive anaplastic large cell lymphoma, acute myelogenous leukemia and diffuse large B-cell lymphoma; treatment resulted in rapid, potent and highly selective STAT3 degradation with similar activity against both mutant and wild-type STAT3, and sustained degradation of 90% or greater led to apoptosis induction and cancer cell death within 48 hours in vitro and in vivo
Macrogenics Inc., of Rockville, Md. Flotetuzumab Bispecific CD123 x CD3 DART molecule Primary induction failure and early relapsed acute myeloid leukemia Updated phase I/II data showed response rate of 30% in intent-to-treat population, including complete remission of 16.6%
Molecular Partners AG, of Zurich, Switzerland MP-0250 Trispecific multi-DARPin candidate neutralizing VEGF-A and HGF  Relapsed/refractory multiple myeloma Updated data from phase II study in combination with bortezomib and dexamethasone in patients previously exposed  to proteasome inhibitors and immunomodulatory drugs showed 1 of 20 patients achieved complete response, 3 achieved very good partial response and 5 achieved partial response, for an overall response rate of 45%
Molecular Templates Inc., of Austin, Texas MT-3724 CD20-targeted immunotoxin Relapsed/refractory diffuse large B-cell lymphoma Of 13 serum rituximab-negative DLBCL or mixed DLBCL/FL subjects, 5 responded (38% objective response rate) across range of 5 to 100 μg/kg doses; of the 5 responses, 2 were complete responses and 3 were partial responses; 3 had stable disease (including 2 patients with 49% and 47% tumor reductions), and 5 patients had progressive disease; of 5 patients receiving maximum tolerated dose of 50 μg/kg, 3 responded (2 CRs, 1 PR)
Nektar Therapeutics Inc., of San Francisco NKTR-255 IL-15 agonist Multiple myeloma and non-Hodgkin lymphoma  Preclinical data showed drug enhanced number and function of NK and CD8+ effector memory T-cell populations in peripheral blood and bone marrow from MM patients and increased expression of activating receptors found on those NK cells; drug also prevented tumor growth and increased survival of CAR T cells when added to a CD19-targeted CAR T-cell regimen in models of B-cell lymphoma 
Novartis AG, of Basel, Switzerland Kymriah tisagenlecleucel) CAR T therapy Relapsed/refractory diffuse large B-cell lymphoma and r/r B-cell acute lymphoblastic leukemia 2 analyses of real-world experience showed overall response rate of 58% for 80 patients in Juliet trial in DLBCL, including 40% who achieved complete response; among 146 children and young adults in Eliana r/r ALL trial, complete response was 85%
Portola Pharmaceuticals Inc., of South San Francisco Cerdulatinib Oral SYK/JAK inhibitor Relapsed/refractory follicular lymphoma Data from 42 patients in cerdulatinib-only cohort showed overall response rate of 48%; 7 patients (17%) achieved complete response, 13 (31%) achieved partial response and 10 (24%) achieved stable disease; among 21 patients in cerdulatinib and rituximab combination cohort, the ORR was 76%; 5 patients (24%) achieved CR, 11 (52%) achieved PR and 5 (24%) achieved SD; of 11 patients in the combination cohort who have been on 1 to 3 prior therapies, ORR was 91%, with a CR rate of 36%
Sanofi SA, of Paris Sutimlimab  Monoclonal antibody targeting C1s Primary cold agglutinin disease In the phase III Cardinal study, 54% of 13 patients met the composite endpoint criteria, with 62.5% of 15 patients achieving a hemoglobin ≥ 12 g/dL or an increase of at least 2 g/dL and 71% of 17 patients remaining transfusion-free after week 5
Springworks Therapeutics Inc., of Stamford, Conn., and Glaxosmithkline plc, of London Nirogacestat (PF-03084014) Gamma secretase inhibitor Multiple myeloma and other lymphomas GSK presented preclinical data in combination with its anti-BCMA antibody-drug conjugate, belantamab mafodotin, showing treatment of BCMA-expressing cancer cell lines with nirogacestat led to significantly increased levels of cell surface expression of BCMA and corresponding decreases in shedding of BCMA, as measured by levels of soluble BCMA; the combination resulted in synergistic increases in cancer cell killing vs. belantamab mafodotin alone, with an up to ~3,000-fold improvement in cytotoxicity
Stemline Therapeutics Inc., of New York Elzonris (tagraxofusp) CD123-directed cytotoxin Intermediate or high-risk relapsed/refractory myelofibrosis Phase I/II data showed 45% (9/20) of patients had symptom burden reduction, including 3 with symptom response per IWG-MRT 2013 MF response criteria; 53% (8/15) of patients with baseline splenomegaly >= 5cm experienced spleen size reduction; 20% (3/15) had reduction >35% (specifically 100%, 47% and 46% reductions)
Stemline Therapeutics Inc., of New York Elzonris (tagraxofusp) CD123-directed cytotoxin Relapsed/refractory multiple myeloma Phase I/II data showed combination with pomalidomide and dexamethasone produced partial responses in 56% (5/9) of patients; those patients notably had decreases in their plasmacytoid dendritic cells, a cell type implicated in myeloma growth and aggressiveness, and cell of origin of blastic plasmacytoid dendritic cell neoplasm
Synimmune GmbH, of Tubingen, Germany Flysyn Fc-optimized antibody Acute myeloid leukemia Interim phase I data of 21 patients treated in 5 cohorts showed 7 (33%) achieved a minimal residual disease response and 1 patient achieved complete molecular remission for more than 1 year
Trovagene Inc., of San Diego Onvansertib Oral adenosine triphosphate competitive inhibitor of serine/threonine polo-like-kinase 1 enzyme Relapsed/refractory acute myeloid leukemia Phase Ib data showed 6 (17%) patients had complete response; 9 (25%) had an objective response across LDAC and decitabine arms and doses
UCB SA, of Brussels, Belgium Rozanolixizumab Subcutaneous antibody binding to FcRn Primary immune thrombocytopenia Phase II data showed clinically relevant improvements (i.e., reaching ≥50x109/L) in platelet count and decreases in immunoglobin G levels observed across all dose groups, with higher response rate (55%–67% in 1 x 15-mg/kg and 1 x 20-mg/kg dose groups vs. 36%–45% in 5 x 4-mg/kg, 3 x 7-mg/kg and 2 x 10-mg/kg dose groups) and shorter time to response achieved by the 1 x 15-mg/kg and 1 x 20-mg/kg doses
Windmil Therapeutics Inc., of Baltimore CAR-MILs Chimeric antigen receptor-engineered MILs Multiple myeloma Preclinical data showed CAR-MILs possess superior antitumor immunity vs. traditional CAR T cells engineered using peripheral blood lymphocytes; in vitro studies showed superior killing of tumor target cells; CAR-MILs also showed greater degree of cytokine and chemokine secretion consistent with a more polyfunctional cytokine profile


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