Data presented at the European Hematology Association's Annual Congress – June 11-21

Company Product Description Indication Status
ADC Therapeutics SA, of Lausanne, Switzerland Loncastuximab tesirine (formerly ADCT-402) Antibody-drug conjugate targeting CD19 Relapsed or refractory diffuse large B-cell lymphoma after at least 2 lines of systemic therapy In the phase II Lotis 2 study, overall response rate was 48.3% and complete response rate was 24.1% in 145 patients; median duration of response was 10.25 months
ADC Therapeutics SA, of Lausanne, Switzerland Loncastuximab tesirine (formerly ADCT-402) Antibody-drug conjugate targeting CD19 Relapsed or refractory diffuse large B-cell lymphoma or mantle cell lymphoma  In the phase I/II Lotis 3 study, at the recommended phase II dose, loncastuximab tesirine plus ibrutinib produced an overall response rate of 75% and a complete response rate of 58.3%
Agios Pharmaceuticals Inc., of Cambridge, Mass. Mitapivat  PKR activator Non-transfusion-dependent alpha- and beta-thalassemia In the phase II study, 92.3% of the 13 evaluable patients met the primary endpoint of at least a 1 g/dL increase in Hb concentration from baseline at 1 or more assessments between week 4 and week 12; mean time to Hb increase of more than 1 g/dL among the Hb responders was 3.1 weeks; mean Hb change from baseline was 1.34 g/dL over weeks 4-12
Alx Oncology Holding Inc., of Burlingame, Calif. ALX-148   CD47 inhibitor Relapsed or refractory non-Hodgkin lymphoma In the phase I study, 10 mg/kg ALX-148 plus rituximab produced an objective response rate of 40.9% and median progression-free survival of 7.4 months in 22 patients; ORR was 33.3% in 15 aggressive patients and 57.1% in 7 indolent patients; in 11 patients treated with 15 mg/kg ALX-148 plus rituximab, ORR was 54.6% and median PFS wasn't reached; ORR was 42.9% in 7 aggressive patients and 75% in 4 indolent patients
Apellis Pharmaceuticals Inc., of Waltham, Mass. Pegcetacoplan C3 inhibitor Paroxysmal nocturnal hemoglobinuria In the phase III Pegasus study, pegcetacoplan produced an adjusted mean improvement of 3.8 g/dL of hemoglobin at week 16, 53% higher than the eculizumab (p<0.0001); in patients with low or no transfusion requirements, pegcetacoplan produced an adjusted mean hemoglobin increase of 2.97 g/dL vs. a 0.01 g/dL decrease for eculizumab; in patients with high transfusion requirements, pegcetacoplan increased adjusted mean hemoglobin by 2.11 g/dL vs. a 4.02 decline for eculizumab
Aprea Therapeutics Inc., of Boston APR-246 (eprenetapopt) Inhibitor of thioredoxin reductase TP53 mutant myelodysplastic syndromes and acute myeloid leukemia In the phase Ib/II study, APR-246 plus azacitidine produced an overall response rate of 75%, with a 57% complete remission in 28 evaluable MDS patients; median overall survival was 12.1 months for 34 MDS patients and 12.1 months for all 52 enrolled patients
Aptose Biosciences Inc., of San Diego CG-806  FLT3/BTK cluster selective kinase inhibitor Relapsed or refractory chronic lymphocytic leukemia, small lymphocytic lymphoma or non-Hodgkin lymphoma  In the phase Ia/Ib study, CG-806 produced lymphocytosis in 2 chronic lymphocytic leukemia patients and completely inhibited phospho-BTK and multiple oncogenic survival pathways in all patients receiving 300 mg or more twice daily
Astrazeneca plc., of Cambridge, U.K. Calquence (acalabrutinib) BTK inhibitor Chronic lymphocytic leukemia In the phase II ACE-CL-001 study, Calquence produced an overall response rate of 97% in 99 patients; in the phase III Ascend study, Calquence produced an ORR of 80% compared to 84% for idelalisib or bendamustine; median progression-free survival was not reached for Calquence compared to 16.8 months for idelalisib or bendamustine
Autolus Therapeutics Ltd., of London AUTO-1 CD19 CAR T cell Relapsed/refractory acute lymphocytic leukemia Of the 19 patients in the phase I study, 84% achieved MRD-negative complete response; 2 of those patients received a transplant while in remission and CD19-negative relapse occurred in 16% of patients
Beigene Ltd., of Beijing Zanubrutinib  BTK inhibitor Relapsed/refractory marginal zone lymphoma In the phase I/II study, overall response rate was 80%, including 15% complete responses and 65% partial responses; 24-month progression-free survival rate was 59.4%; 24-month overall survival rate was 83.2%
Bluebird Bio Inc., of Cambridge, Mass. Lentiglobin Gene therapy expressing hemoglobin Sickle cell disease In group C of the phase 1/2 HGB-206 study, total hemoglobin ranged from 9.6 – 16.2 g/dL and HbAT87Q levels ranged from 2.7 – 9.4 g/dL at the last reported visit for 16 patients with at least 6 months of follow-up; mean reduction in annualized rate of vaso-occlusive crises (VOCs) and acute chest syndrome (ACS) was 99.5% among 14 patients with at least 6 months of follow-up and a history of VOCs or ACS
Constellation Pharmaceuticals Inc., of Cambridge, Mass. CPI-0610 BET inhibitor Myelofibrosis  In the phase II Manifest study, 73% of 51 JAK-inhibitor-naïve patients treated with CPI-0610 plus ruxolitinib achieved a 35% reduction in spleen volume (SVR35) at 12 weeks and had a median spleen volume reduction of 51%; at 24 weeks 63% of 30 patients achieved SVR35 and had a median spleen volume reduction of 53%; 21% of 14 transfusion-dependent (TD) patients who were JAK-inhibitor-experienced or -ineligible treated with CPI-0610 converted to transfusion independence (TI); 34% of 32 ruxolitinib-experienced patients treated with CPI-0610 plus ruxolitinib converted from TD to TI
Crispr Therapeutics AG, of Zug, Switzerland, and Vertex Pharmaceuticals Inc., of Boston CTX-001  Autologous, gene-edited hematopoietic stem cell therapy  Transfusion-dependent beta-thalassemia  In the phase I/II Climb-111 study, 1 patient was transfusion independent and had total hemoglobin levels of 14.2 g/dL, fetal hemoglobin of 13.5 g/dL and F-cells (erythrocytes expressing fetal hemoglobin) of 100% 15 months after CTX-001 infusion; a second patient was transfusion independent and had hemoglobin levels of 12.5 g/dL, fetal hemoglobin of 12.2 g/dL and F-cells of 99.4% 5 months after CTX-001 infusion
Crispr Therapeutics AG, of Zug, Switzerland, and Vertex Pharmaceuticals Inc., of Boston CTX-001  Autologous, gene-edited hematopoietic stem cell therapy  Sickle cell disease In the phase I/II Climb-121 study, at 9 months after CTX-001 infusion, the first treated patient was free of vaso-occlusive crises, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%
Editas Medicine Inc., Cambridge, Mass. EDIT-301  Autologous, gene-edited hematopoietic stem cell therapy  Sickle cell disease Editing occurred in 90% of sickle cell disease patient donors; derived red blood cells had more than 50% HbF expression; cells had a 4-fold decrease in sickling when subjected to reduced oxygen levels compared to unedited control cells; in mice, HbF expression was increased by about 50% in the red blood cells and about 90% of the cells were HbF-positive
Forma Therapeutics Inc., of Watertown, Mass. FT-4202 Pyruvate kinase-R activator Sickle cell disease A single dose of the drug in the phase I study improved hemoglobin, red blood cell and reticulocyte counts
Imago Biosciences Inc., of San Francisco Bomedemstat (IMG-7289) Lysine-specific demethylase 1 inhibitor Advanced myelofibrosis In the phase II study, at 24 weeks, 83% of patients treated with bomedemstat had spleen volume reductions and 86% demonstrated reductions in Total Symptom Scores; 70% of patients had stable or improved hemoglobin; 71% of patients had a stable or improved BM fibrosis score; >90% of patients with elevated circulating inflammatory cytokines showed significant reductions
Imara Inc., of Boston IMR-687 PDE9 inhibitor Sickle cell disease In the phase IIa study, 100 mg/200 mg IMR-687 produced a relative increase in F-cell percentage of 18.1%, representing a mean increase from baseline through week 24 of about 155% (p=0.022); HbF percentage increased 1.7% from baseline through week 24, representing a mean increase of approximately 38%
Geron Corp., of Menlo Park, Calif. Imetelstat  Telomerase inhibitor Myelodysplastic syndromes In the phase II/III Imerge study, at week 8, 42% of patients were transfusion independent (TI) and 68% had hematologic improvement-erythroid (HI-E); median duration of TI was 20 months and median duration of HI-E was 21 months; 29% of patients transfusion-free for more than 1 year
Keros Therapeutics Inc., of Lexington, Mass. KER-050 Engineered ligand trap comprising modified ligand-binding domain of TGF-beta receptor  Cytopenias Phase I results in healthy subjects showed rapid, robust, sustained and dose-dependent increases in hemoglobin, reticulocytes and red blood cells, in addition to clinically meaningful increases in platelets; dose-dependent increase in proportion of subjects with a hemoglobin increase of ≥1.5 g/dL was observed; mean half-life of KER-050 observed in 0.5-, 1.5- and 4.5-mg/kg doses in part 1 and after the first 0.75-mg/kg dose in part 2 ranged from 9.7 to 11.9 days
Keros Therapeutics Inc., of Lexington, Mass. KER-050 Engineered ligand trap comprising modified ligand-binding domain of TGF-beta receptor  Cytopenias Preclinical data from multiple animal models showed treatment resulted in robust and dose-dependent increases in red blood cell (RBC) number, hemoglobin, hematocrit and reticulocyte number in male and female cynomolgus monkeys dosed subcutaneously at doses of 3 mg, 10 mg or 50 mg/kg for 3 months compared to vehicle-treated cohorts; in healthy mice dosed with 10 mg/kg of RKER-050 (a research form of KER-050), rapid increases in circulating reticulocytes, RBCs, hemoglobin and hematocrit were observed vs. vehicle-treated mice
Keros Therapeutics Inc., of Lexington, Mass. KTI-2338 ALK inhibitor Iron-refractory iron deficiency anemia  Preclinical data showed daily treatment in mice with established disease resulted in increases in hemoglobin, hematocrit, red blood cells and serum iron with a concomitant decrease in hepcidin   
Kiadis NV, of Amsterdam FC21-NK cells Hyperfunctional NK cells High-risk hematological malignancies, relapsed or refractory acute myeloid leukemia and high-grade gliomas Data from phase I/II studies show allogeneic FC21-NK cells can persist and proliferate in vivo up to five weeks without the support of exogenous cytokines
Kiadis NV, of Amsterdam FC21-NK cells Hyperfunctional NK cells Relapsed/refractory acute myeloid leukemia Phase I data show 2 of 3 patients with serious concomitant infections demonstrated complete responses lasting 151 and 269 days after receiving FC21-NK infusions; both patients had complete resolution of infection and pulmonary symptoms, while the third experienced significant clinical improvement with almost complete resolution of cholangitis
Omeros Corp., of Seattle Narsoplimab (OMS-721) MASP-2-targeting monoclonal antibody Hematopoietic stem cell transplant-associated thrombotic microangiopathy Data from pivotal study showed 54% complete response rate in all patients and 65% complete response rate in patients receiving at least 4 weeks of treatment; 100-day survival rate was 68% among all patients, with 83% for patients receiving at least 4 weeks of treatment and 93% among complete responders; statistically significant improvements from baseline in platelet count (p=0.001), lactate dehydrogenase (p=0.008) and haptoglobin (p<0.001)
Oryzon Genomics SA, of Madrid, Spain Iadademstat (ORY-1001) Small oral molecule inhibiting epigenetic enzyme LSD1 Acute myeloid leukemia Data from ongoing phase II Alice trial in combination with azacitidine in elderly patients showed objective response rate of 77% (10 of 13 evaluable patients); of those, 60% were complete remissions (6 CR/CRi) and 40% partial remissions (4 PR); mean time to response was 37 days in those patients who responded; longest remission at data cutoff was 488 days
Principia Biopharma Inc., of South San Francisco Rilzabrutinib BTK inhibitor Immune thrombocytopenia Data from ongoing phase I/II trial in 47 heavily pretreated patients showed 50% who started at 400 mg twice daily and had at least 12 weeks of treatment (n=26) achieved the primary endpoint, defined as proportion of patients able to achieve 2 or more consecutive platelet counts, separated by at least 5 days, of ≥ 50,000/μL and an increase of platelet count of ≥20,000/μL from baseline, without use of rescue medication; in overall patient population, the primary endpoint was met by 43%, irrespective of dose and duration of treatment
Sierra Oncology Inc., of Vancouver, British Columbia Momelotinib JAK1/2 and ACVR1 inhibitor Myelofibrosis Long-term data from more than 820 patients from phase II and III studies continue to show rapid and sustained increase in hemoglobin levels vs. decrease in hemoglobin for patients on ruxolitinib (Jakafi); patients on momelotinib also had significantly higher mean platelet counts vs. those on ruxolitinib
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Ninlaro (ixazomib) Proteasome inhibitor Multiple myeloma Results from phase III Tourmaline-MM4 study testing drug as first-line maintenance therapy in adults not treated with stem cell transplantation showed statistically significant and clinically meaningful improvement in progression-free survival vs. placebo (p<0.001), corresponding to 34% reduction in risk of progression or death; median PFS was 17.4 months for Ninlaro vs. 9.4 months for placebo; secondary endpoint of overall survival not yet mature
TG Therapeutics Inc., of New York TG-1701 BTK inhibitor Relapsed/refractory chronic lymphocytic leukemia and lymphomas Phase I data showed monotherapy dose-escalation cohort resulted in partial responses at all dose levels evaluated (100 mg to 400 mg once daily) in CLL, mantle cell lymphoma (MCL), Waldenström’s macroglobulinemia (WM) and small lymphocytic lymphoma; in monotherapy dose-expansion cohort, 25 patients were evaluable for efficacy, with 92% overall response rate in CLL patients, 33% ORR in MCL and 86% ORR in WM; in combination with umbralisib and ublituximab, encouraging clinical activity, with 77% ORR across all disease types, including complete responses in 3 patients
TG Therapeutics Inc., of New York Umbralisib Dual inhibitor of PI3K delta and CK1 epsilon Relapsed/refractory chronic lymphocytic leukemia and mantle cell lymphoma Phase I/Ib study in combination with ibrutinib showed overall response rate in CLL was 95%, including 29% complete response rate; 4-year progression-free survival and overall survival were 78% and 90%, respectively; in MCL, ORR was 71%, with 24% CR rate, and median PFS and OS were 10.8 and 30.7 months, respectively
Tolero Pharmaceuticals Inc., of Salt Lake City Alvocidib CDK9 inhibitor Acute myeloid leukemia Data from phase I Zella 101 trial in newly diagnosed AML showed alvocidib followed by cytarabine and daunorubicin induction therapy showed encouraging clinical activity and tolerable safety profile; 71% (22 of 31) of evaluable patients achieved complete remission, with an overall response rate of 77% (24 of 31); an exploratory cohort found 89% (8 of 9) achieved measurable residual disease negativity; at median of 9.2 months follow-up, overall survival not reached, with 62% of patients alive at data cutoff
X4 Pharmaceuticals Inc., of Cambridge, Mass. Mavorixafor Small-molecule antagonist of chemokine receptor CXCR4 WHIM (warts, hypogammaglobulinemia, infections and myelokathexis) syndrome Data from ongoing phase II trial showed sustained, dose-dependent increases in white blood cells, absolute neutrophil count (ANC) and absolute lymphocyte count; higher doses were shown to increase the TATANC at least 4.5-fold vs. TATANC at lower doses; decreased yearly infection rate from 4.63 events in 12 months prior to trial to 2.27 events at higher doses; patients with cutaneous warts on hands and/or feet at baseline achieved an average 75% reduction in number of warts

Notes

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