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

Company Product Description Indication Status
AB Science SA, of Paris AB-8939 Tubulin inhibitor Acute myeloid leukemia In vivo experiments in 3 patient-derived xenograft mouse models and cytarabine(Ara-C)-resistant mouse model suggested study drug, alone or with Ara-C, increased survival and reduced tumor growth vs. Ara-C alone; separate ex vivo and in vivo studies showed study drug had broad antiproliferative activity across AML subtypes and produced antiproliferative effect against blasts isolated from AML patients, with majority of IC50 values ranging from 1.4 nM to 1 µM
Abbvie Inc., of North Chicago Imbruvica (ibrutinib) BTK inhibitor Chronic lymphocytic leukemia Extended follow-up analysis of phase III E1912 study showed combination with rituximab vs. standard chemoimmunotherapy regimen (FCR) in previously untreated patients, ages 70 and younger, showed progression-free survival benefits were sustained in combination arm vs. FCR (p-0.009); overall survival continued to favor combo arm (p=0.009)
Abbvie Inc., of North Chicago Imbruvica (ibrutinib) BTK inhibitor Chronic lymphocytic leukemia Analysis with up to 6 years of follow-up from phase III Resonate and Resonate-2 studies in previously untreated and relapsed/refractory patients showed median progression-free survival not reached for first-line or the 1-2 prior lines groups, and median PFS was 40.1 months for the 3 or more prior lines group; greater proportion of patients treated in earlier lines remained progression-free or alive at 60 months (70% for first-line, 60% for 1-2 prior lines and  33% for 3 or more prior lines
Abbvie Inc., of North Chicago Imbruvica (ibrutinib) BTK inhibitor Chronic lymphocytic leukemia New data from phase II Captivate study in combination with Venclexta (venetoclax) in previously untreated patients showed those receiving up to 12 cycles of combination regimen achieved high rates of undetectable minimal residual disease in both peripheral blood (75% of patients) and in bone marrow (72% of patients)
Abbvie Inc., of North Chicago Imbruvica (ibrutinib) BTK inhibitor Relapsed/refractory mantle cell lymphoma Results from 7.5-year pooled analysis of phase II and III studies showed earlier treatment with monotherapy vs. later lines of therapy extended progression-free survival and increased likelihood of complete response; some patients achieved disappearance of any signs of disease
Agios Pharmaceuticals Inc., of Cambridge, Mass. Mitapivat (AG-348) Oral, small-molecule allosteric activator of wild-type and variety of mutated PKR enzymes Non-transfusion-dependent thalassemia  Data from 8 evaluable patients in phase II study showed 7 achieved hemoglobin increase of ≥1 g/dL; for responders, mean hemoglobin increase from baseline was 1.76 g/dL (range, 0.9–3.3 g/dL) during weeks 4-12
Agios Pharmaceuticals Inc., of Cambridge, Mass. Tibsovo (ivosidenib) IDH1 gene inhibitor Acute myeloid leukemia  As of Feb. 19 data cutoff, in 23 participants with newly diagnosed IDH1-mutant disease ineligible for intensive chemotherapy in ongoing phase I/II study in combination with azacitidine, complete response (CR) rate was 61% and CR + CR with partial hematologic recovery (CRh) rate was 70%; median duration of CR and CR+CRh were not reached; in those with CR, 10 of 14 (71%) had IDH1 mutation clearance in bone marrow mononuclear cells measured by BEAMing digital PCR; most with IDH1 mutation clearance showed measurable residual disease negativity by flow cytometry or next-gen sequencing
Agios Pharmaceuticals Inc., of Cambridge, Mass. Tibsovo (ivosidenib) IDH1 gene inhibitor Acute myeloid leukemia Genomic profiling of samples from phase I in IDH1-mutant relapsed/refractory AML found RTK pathway mutations NRAS and PTPN11 at baseline were associated with lower likelihood of clinical response to study drug as monotherapy in relapsed/refractory AML; those with JAK2 mutations were more likely to achieve response
Aleta Biotherapeutics Inc., of Natick, Mass. CD19-anti-CD20 bridging protein Monomeric CD19-ECD-anti-CD20 bridging protein CD19-negative relapse from CAR-CD19 T-cell treatment Preclinical results from in vitro study showed CAR19 T cells found and eliminated CD19-negative cells that escaped from CAR-CD19 T- cell treatment; in vivo, CAR-CD19 T cells, plus the injected Aleta bridging protein, controlled tumor cell growth, preventing escape from therapy, while CAR-CD19 T cells alone did not prevent tumor relapse
Alpine Immune Sciences Inc., of Seattle ALPN-101 Dual CD28/ICOS antagonist Graft-vs.-host disease Dose-dependent pharmacodynamic activity observed in phase I study in healthy volunteers, including inhibition of T-cell activation, assessed ex vivo based on inhibition of staphylococcal enterotoxin B-induced cytokine production, and inhibition of antibody responses, assessed following immunization with keyhole limpet hemocyanin
Alx Oncology, of Burlingame, Calif.  ALX-148 CD47 antagonist Non-Hodgkin lymphoma In phase I combination study with rituximab in 29 participants with relapsed/refractory NHL, 21 response-evaluable participants showed objective response rate  of 43% and median progression-free survival (mPFS) of 7.3 months; in those with aggressive NHL (n=14), ORR of 36% and mPFS of 3.1 months were observed; in indolent NHL (n=7) ORR of 57% was observed and mPFS was not reached; 2 participants achieved complete response, 1 of whom was refractory to prior rituximab therapy; in those with rituximab-refractory disease (n=9), ORR of 44% was observed; in initial response-evaluable patients with relapsed/refractory NHL (n=3) administered ALX-148 (15 mg/kg once weekly), ORR of 67% was reported and mPFS was not reached
Arqule Inc., of Burlington, Mass. ARQ-531 Dual BTK/TRK tyrosine kinase inhibitor B-cell non-Hodgkin lymphoma Final data from phase I study in 47 participants showed overall response rate of 89% (8/9 evaluable patients, 7/8 harboring BTK-C481S mutation) in relapsed/refractory chronic lymphocytic leukemia (CLL); 11 of 19 dosed at 65 mg once daily remain on study; ORR of 50% (3/6 evaluable) in Richter’s transformation was achieved at 65 mg once daily; 2 additional partial responses observed, including 1 in follicular lymphoma and 1 in diffuse large B-cell lymphoma; 5/5 evaluable CLL patients remain durable confirmed PRs through cycle 9 and remain on therapy; 2/3 of Richter’s patients who achieved PRs came off study after becoming eligible for CAR T therapy
the follicular lymphoma patient that achieved a PR has been on study for 120 weeks and remains a PR and on therapy
Astrazeneca plc, of Cambridge, U.K. Calquence (acalabrutinib) BTK inhibitor Chronic lymphocytic leukemia Interim analysis of phase III Elevate TN trial showed study drug, combined with obinutuzumab (Gazyva, Roche Holding AG) or as monotherapy, reduced progression-free survival by 90% and 80%, respectively, vs. chlorambucil + obinutuzumab at median follow-up of 28.3 months; in exploratory analysis, Calquence alone or in combination showed PFS improvements across most prespecified subgroups with high-risk disease characteristics
Aurora Bio Inc., of South San Francisco AU-R01 (124I-p5+14) Imaging PET peptide radiotracer Amyloidosis  Among first 18 participants with light chain amyloidosis (AL) dosed in phase I study, analysis of PET images indicated cardiac uptake in 83%, including 50% with no cardiac symptoms and normal cardiac biomarkers; kidney, spleen and liver uptake was observed in 67%, 42% and 42%, respectively; retention of radiotracer was observed in the nerves, ligaments and lungs in those with transthyretin amyloidosis (ATTR) and in the kidney, spleen, adrenal glands and liver in those with leukocyte cell-derived chemotaxin 2 amyloidosis; 100% of ATTR patients had cardiac uptake, including 50% with no cardiac symptoms and normal cardiac biomarkers and 1 participant with negative pyrophosphate scan confirmed via endomyocardial biopsy
Autolus Therapeutics plc, of London AUTO-1 CD19 CAR Acute lymphoblastic leukemia Updated phase I data in recurrent/refractory ALL for 15 evaluable patients showed 13 (87%) achieved minimal residual disease (MRD)-negative complete response at 1 month and all had ongoing CAR T-cell persistence at last follow-up; CD19-negative relapse occurred in 22% (2 of 15); in patients dosed with AUTO-1 manufactured in closed process, 9 of 9 (100%) achieved MRD-negative CR at 1 month and 6-month event-free survival, with overall survival at 100% in that cohort
Autolus Therapeutics plc, of London AUTO-1 CD19 CAR B-cell acute lymphoblastic leukemia Data from phase I Carpall trial in pediatric patients showed 19 of 21 (90%) achieved molecular complete remission at 1 month post infusion; CAR T cell expansion was detectable by flow in a number of patients up to 36 months; in cohort 2, 100% achieved molecular complete remission at 1 month post infusion; of 14 patients in cohort 1, overall survival at 6 months was 86% and at 12 months was 71%, while event-free survival was 71% and 54%, respectively; data from cohort 2 not yet evaluable
Autolus Therapeutics plc, of London AUTO-3 Bicistronic CAR targeting CD19 and CD22 Relapsed/refractory diffuse large B-cell lymphoma Data from phase I/II Alexander study showed 5 of 14 had compete response, with 4 of 5 CRs ongoing, the longest at 18 months
Autolus Therapeutics plc, of London AUTO-3 Bicistronic CD19 and CD22 CAR  Pediatric acute lymphoblastic leukemia Data from phase I/II Amelia study in relapsed/refractory patients showed among 10 CAR T-naïve patients, at median follow-up of 9.7 months, 9 of 10 (90%) achieved complete response and 8 of 10 (80%) achieved complete molecular remission by PCR; estimated overall survival at 12 months was 100%
Axcella Health Inc., of Cambridge, Mass. AXA-4010 Endogenous metabolic modulator Sickle cell disease Preclinical data showed plasma amino profiles of adults with SCD were different from those in control group, with 50% reduction in arginine and > 30% reduction in total essential amino acids; endothelial cell cultures treated with TNF-alpha and constituents of AXA-4010 saw reduction in markers of vascular adhesion, inflammation and cell migration vs. cell cultures treated with glutamine and TNF-alpha, which saw increase in adhesion, no change in inflammation and reduction in cell migration; compared to individual amino acids, AXA-4010 constituents showed better improvement in red blood cell deformability
Beigene Ltd., of Beijing Brukinsa (zanubrutinib) BTK inhibitor Chronic lymphocytic leukemia; small lymphocytic lymphoma Initial results from arm C in phase III Sequoia monotherapy trial, at Aug. 7 data cutoff with median follow-up of 10 months, showed overall response rate of 92.7% (101/109); partial response rate was 78.9% (86/109) and PR rate with lymphocytosis was 11.9% (13/109); complete response rate was 1.9% (2/109); 4 cases of disease progression occurred; 23.9% (26/109) experienced at least 1 serious adverse event (AE), including 1 fatal AE, pneumonia leading to sepsis and death, considered related to treatment drug
Beigene Ltd., of Beijing Brukinsa (zanubrutinib) BTK inhibitor Chronic lymphocytic leukemia; small lymphocytic lymphoma Updated results from global phase I/II trial showed Brukinsa was well-tolerated and active in relapsed/refractory (R/R) or treatment-naive (TN) CLL/SLL, irrespective of del(17p) status (n=123; 101 R/R, 22 TN); at data cutoff of May 8 with median follow-up of 29.5 months, overall response rate was 95.9% (118/123); partial response rate was 73.2% (90/123); PR rate with lymphocytosis was 6.5% (8/123); complete response rate was 16.3% (20/123), including 1 CR with incomplete bone marrow recovery; 2-year progression-free survival was 91% in R/R and 95% in TN disease
Beigene Ltd., of Beijing Brukinsa (zanubrutinib) BTK inhibitor Non-Hodgkin’s lymphoma In 54 participants with aggressive disease in phase Ib combination trial with anti-PD-1 antibody tislelizumab (Beigene), at data cutoff of Aug. 31 with median follow-up of 8.1 months, preliminary findings showed overall response rate of 37% (20/54), partial response rate of 20.4% (11/54), complete response rate of 16.7% (9/54) and stable disease rate of 9.3% (5/54)
Bergenbio ASA, of Bergen, Norway Bemcentinib (BGB-324) AXL inhibitor Acute myeloid leukemia Phase II combination trial with low-dose cytarabine in older adults showed long duration of response (> 9.9 mo, still maturing), with 50% complete response/complete response with incomplete hematologic recovery (CR/CRi)  in 6 evaluable newly diagnosed patients; 1 CR/CRi and 3 stable disease seen in 6 evaluable relapsed/refractory patients with > 2 previous therapies
Beyondspring Inc., of New York Plinabulin Guanine nucleotide exchange factor stimulator; tubulin receptor antagonist Chemo-induced neutropenia Data from phase II/III study 105 showed drug's ability to protect granulocyte-monocyte progenitor cells; additional data from study 105 suggested plinabulin protects common lymphoid progenitor cells
Bluebird Bio Inc., of Cambridge, Mass. Lentiglobin Gene therapy Sickle cell disease New data from ongoing phase I/II HGB-206 study showed of 17 patients in cohort C with longest follow-up at 21 months, none required regular red blood cell transfusions post treatment; in those with 6+ months of follow-up, median levels of gene therapy-derived anti-sickling hemoglobin, HbAT87Q, were at least 40% of total hemoglobin; total hemoglobin and HbAT87Q levels ranged from 9.3 – 15.2 g/dL and 2.7 – 9 g/dL, respectively, at last visit; among 9 patients with 6+ months follow-up who had 4 or more vaso-occlusive crises or acute chest syndrome in 2 years prior to treatment, there was 99% reduction in annualized rate of both
Blueprint Medicines Corp., of Cambridge, Mass. Avapritinib KIT tyrosine kinase inhibitor; PDGF receptor alpha antagonist Systemic mastocytosis In dose-finding part 1 of phase II Pioneer trial that enrolled 39 participants, at Nov. 12 cutoff date with median study time of 12 weeks, mean % change in serum tryptase was -37.72% for 25-mg dose, -54.08% for 50 mg and -56.16% for 100 mg vs. 7.05% for placebo at first post-baseline assessment and -48.24% for 25 mg, -66.67% for 50 mg and -61.83% for 100 mg vs. 0.39% for placebo at 12 weeks
Blueprint Medicines Corp., of Cambridge, Mass. Avapritinib KIT tyrosine kinase inhibitor; PDGF receptor alpha antagonist Systemic mastocytosis Top-line results from phase I Explorer trial, at data cutoff of Aug. 30 with median follow-up of 21 months, showed confirmed overall response rate, defined as complete remission with full or partial recovery of peripheral blood counts, partial remission or clinical improvement, of 77% in 48 evaluable participants; median duration of response and median overall survival not reached
Bristol-Myers Squibb Co., of New York Lisocabtagene maraleucel (liso-cel) CD19-directed CAR T-cell therapy Chronic lymphocytic leukemia; small lymphocytic lymphoma In phase I/II Transcend CLL 004 study, with 22 participants evaluable for efficacy at data cutoff, at median follow-up of 11 months overall response rate was 81.5% (18/22) with 45.5% (10/22) achieving complete response; in those who failed a BTK inhibitor and venetoclax (Venclexta, Abbvie Inc.), ORR was 89% (8/9), with 67% (6/9) achieving CR; by day 30 following treatment, 68% (15/22) achieved early objective response, with 10/12 responders at 6 months remaining progression-free after at least 9 months and 8 in response at 12 months or >; among 20 participants evaluable for minimal residual disease (MRD), most achieved undetectable MRD in blood (75%) and bone marrow (65%) by next-generation sequencing
Bristol-Myers Squibb Co., of New York Lisocabtagene maraleucel (liso-cel) CD19-directed CAR T-cell therapy Large B-cell non-Hodgkin's lymphoma In phase II Pilot study, with 13 participants receiving lymphodepletion followed by liso-cel at data cutoff, all 12 eligible for response evaluation achieved response, including 6 with complete response; 7 of 12 maintained response levels at 3 months following liso-cel infusion
Bristol-Myers Squibb Co., of Princeton, N.J. Lisocabtagene maraleucel  CD19-directed CAR T-cell therapy Relapsed/refractory large B-cell lymphoma Transcend NHL 001 study met primary endpoint; among evaluable patients, overall response rate was 73% (187/256), with 53% (136/256) achieving complete response; median duration of response was not reached at median follow-up of 12 months; median progression-free survival was 6.8 months and median overall survival was 21.1 months; median PFS and OS for patients who achieved CR was not reached, with 65% progression free and 85.5% alive at 12 months, respectively
Bristol-Myers Squibb Co., of Princeton, N.J., and Pfizer Inc., of New York Eliquis (apixaban) Factor Xa inhibitor Venous thromboembolism Real-world analyses vs. low-molecular-weight heparin (LMWH) in patients with VTE and active cancer showed Eliquis use associated with lower rates of major bleeding (MB) (p=0.003), clinically-relevant non-major (CRNM) bleeding (p=0.006) and recurrent VTE (p=0.001) vs. LMWH; Eliquis also associated with lower rate of recurrent VTE (p=0.007) and similar rates of major bleeding (p=0.051) and CRNM bleeding (p=0.145) compared to warfarin
Catalyst Biosciences Inc., of South San Francisco Marzeptacog alfa, activated (Marzaa) Factor VIIa agonist Hemophilia As rescue therapy in hemophilia A mouse model of severe bleeding, subcutaneously dosed therapy reduced bleeding and was comparable to intravenous Novoseven (eptacog alfa, activated, Novo Nordisk A/S); separate preclinical study showed Marzaa and Novoseven exhibited comparable  characteristics when spiked into hemophilia A plasma containing Hemlibra (emicizumab, Roche Holding AG) at clinically relevant concentrations, suggesting Marzaa expected to be safe in combination with Hemlibra
Cellectar Biosciences Inc., of Florham Park, N.J. CLR-131 Phospholipid ether-drug conjugate Multiple myeloma Fractionated dosing data in 19 patients with relapsed/refractory disease from phase I and phase II Clover-1 trials, at cutoff date of July 30, showed overall response rate across 3 dose cohorts was 31.3%, with 100% disease control rate; those receiving higher fractionated 37.5 mCi/m2 dose had 50% ORR with remaining 50% having minimal responses (> 25% reduction in surrogate efficacy marker)
Cellular Biomedicine Group Inc., of New York C-CAR088 Anti-B-cell maturation antigen CAR T-cell therapy Relapsed or refractory multiple myeloma Of 5 evaluable patients in ongoing phase I trial, all showed clinical improvement as early as 2 weeks post treatment; by 4 weeks, 1 patient achieved complete response, 3 patients reached very good partial response and 1 reached partial response; C-CAR088 proliferation and expansion in peripheral blood correlated with decrease of tumor burden
Celularity Inc., of Warren, N.J. PNK-007 Allogeneic, placental-derived CD19 CAR T cell-based product Multiple myeloma Phase I data showed single infusion was well-tolerated after autologous stem cell transplant for up to 1 year
Corvus Pharmaceuticals Inc., of Burlingame, Calif. CPI-818 ITK inhibitor T-cell lymphomas Phase I/Ib data for first 7 patients showed pharmacokinetics and occupancy studies in line with expectations; CPI-818 shown to bind covalently to ITK at low nanomolar concentrations without reacting with other kinases; in vitro data showed selective cytotoxicity to Sezary cells, while sparing normal T cells, in 3 subjects not enrolled in study; preclinical data in murine models of lymphoproliferative and autoimmune disease showed CPI-818 inhibited development of lymph node and spleen enlargement by preventing proliferation of abnormal T cells and treatment led to regression of lymphadenopathy and splenomegaly in animals with established disease
Cyclacel Pharmaceuticals Inc., of Berkeley Heights, N.J. CYC-065 Dual CDK2/CDK9 inhibitor Acute myeloid leukemia; myelodysplastic syndromes; chronic lymphocytic leukemia In phase I combination study (CYC065-03) with venetoclax (Venclexta, Abbvie Inc./Roche Holding AG), 3 of 9 participants with relapsed/refractory (R/R) AML/MDS who received doses from 64 mg/m2 to 150 mg/m2 achieved decreases in leukemia blast cells in peripheral blood as reported by investigators; in separate phase I combination study (CYC065-02), first 2 R/R CLL patients achieved shrinkage of enlarged lymph nodes by CT scan 
Eli Lilly and Co., of Indianapolis LOXO-305 Highly selective, non-covalent BTK inhibitor Chronic lymphocytic leukemia and mantle cell lymphoma Phase I/II data showed it delivered objective responses at all doses studied in patients who had received diverse prior therapies and who had exhibited varied molecular mechanisms of acquired resistance, including those with BTK resistance, BTK intolerance and BCL2 resistance
Epizyme Inc., of Cambridge, Mass. Tazemetostat Oral EZH2 Follicular lymphoma Mature data from ongoing phase II study testing monotherapy in patients, with or without EZH2 activating mutations, who have received at least 2 prior lines of systemic therapy demonstrated meaningful clinical activity as assessed by both investigators and an independent review committee; objective response rate of 69% for EZH2 mutation and 35% for wild-type EZH2; median duration of response was 11 months and 13 months, respectively, while median-progression-free survival was 14 months and 11 months, respectively; NDA submission in December 2019
Fate Therapeutics Inc., of San Diego FT-596 Multi-antigen targeting natural killer cell product derived from a clonal master engineered induced pluripotent stem cell line Lymphoma Demonstrated comparable antitumor activity to CAR19 T cells in vivo in humanized mouse model of lymphoma; combination with rituximab showed durable tumor clearance in vivo in preclinical lymphoma model; company plans to begin enrollment in clinical trial in early 2020
Forty Seven Inc., of Menlo Park, Calif. Magrolimab Monoclonal antibody against CD47 Myelodysplastic syndrome and acute myeloid leukemia Phase Ib data showed a 50% complete response rate and a 92% overall response rate in untreated patients with higher-risk MDS, as well as a 55% CR with complete blood count recovery rate and a 64% ORR rate in patients with untreated AML who are ineligible for induction chemotherapy; median duration of response is not yet reached in those on treatment for more than 6.4 and 8.8 months for MDS and AML, respectively; clinical program design may support accelerated approval in higher-risk MDS, with a BLA filing expected for the fourth quarter of 2021
Genentech, a unit of the Roche Group, of South San Francisco Mosunetuzumab CD20-CD3 T-cell engaging bispecific antibody Relapsed or refractory B-cell non-Hodgkin lymphoma Phase I/Ib GO29781 study, which includes people previously treated with CAR T-cell therapy, showed an objective response rate of 62.7% in slow-growing NHL and 37.1% in aggressive NHL; data demonstrated a complete response rate of 43.3% and 19.4%, respectively; in those who received prior CAR T-cell therapy, the ORR was 38.9%, and 22.2% achieved a CR
Genentech, a unit of the Roche Group, of South San Francisco CD20-TCB CD20-CD3 T-cell engaging bispecific antibody Relapsed or refractory B-cell non-Hodgkin's lymphoma Phase I/Ib NP30179 study of CD20-TCB in combination with Gazyva (obinutuzumab) showed an objective response rate of 54% and a complete response rate of 46%, including an ORR and CR of 66.7% in those with follicular lymphoma and an ORR of 50% and a CR of 40.9% in aggressive NHL
IMV Inc., of Dartmouth, Nova Scotia DPX-Survivac Immunotherapy consisting of survivin-based peptides formulated in IMV's DPX drug delivery platform Recurrent/refractory diffuse large B-cell lymphoma Phase II Spriel study data show that 7 of 9 (77.8%) evaluable subjects exhibited clinical benefit, including 3 complete responses and 2 partial responses; all 7 had reproducible survivin-specific T-cell responses and favorable safety profiles; top-line results and the launch of an IMV-sponsored study are planned in 2020
Innate Pharma SA, of Marseille, France Lumoxiti (moxetumomab pasudotox-tdfk) CD22-directed immunotoxin Relapsed/refractory hairy cell leukemia Long-term data from pivotal phase III study showed 36% (29/80) of patients achieved durable complete response at day 181, compared to primary analysis in which 30% durable CR rate was reported; there was a 61% probability that patients who achieved CR would maintain it after 5 years
Janssen Pharmaceutical Cos. of New Brunswick, N.J.-based Johnson & Johnson JNJ-4528 BCMA-directed CAR T-cell therapy Relapsed or refractory multiple myeloma Initial results from phase Ib/II Cartitude-1 study showed early and deep responses, with median of 5 prior treatment regimens (range 3-18), with 100% achieving a response at median 6-month follow-up; overall response rate included 69% of patients achieving complete response or better, 86% achieving very good partial response or better and 14% achieving partial response; 100% achieved early minimal residual disease-negative status at day 28 post infusion; at 6-month follow-up, 27 of 29 were progression-free
Jasper Therapeutics, of Menlo Park, Calif. JSP-191 (formerly AMG-191) Humanized antibody targeting CD117; conditioning agent to enable stem cell transplantation Severe combined immunodeficiency Phase I dose-escalation study data were encouraging and company plans to expand clinical development beyond SCID into clinical trials for acute myeloid leukemia, myelodysplastic syndrome and Fanconi anemia
Kite, a company of Foster City, Calif.-based Gilead Sciences Inc. Yescarta (axicabtagene ciloleucel) CAR T therapy targeting CD19 Refractory large B-cell lymphoma New data from Zuma-1 trial showed, at minimum follow-up of 3 years, 47% of patients were alive, and median overall survival was 25.8 months; updated results from separate Zuma-1 safety management study showed earlier steroid use appeared to decrease percentage of patients with grade ≥3 cytokine release syndrome (2%) and neurologic events (17%); objective response rate in cohort 4 was 73%, with 51% achieving complete response
Kura Oncology Inc., of San Diego Tipifarnib Inhibitor of farnesyl transferase  Angioimmunoblastic T-cell lymphoma  Further phase II data from 20 evaluable patients with relapsed/refractory AITL showed 5 achieved complete response and 5 achieved partial response, for objective response rate of 50% on evaluable basis and 38% on an intent-to-treat basis; 3 patients experienced disease stabilization; next-generation sequencing of 19 available biopsies showed 10 (53%) carried C336R/Q386E variants in the killer-cell immunoglobulin-like receptor 3DL2, an immune checkpoint receptor
Maat Pharma SA, of Lyon, France MaaT-013 Full-ecosystem microbiome restoration biotherapeutic Gastrointestinal-predominant acute graft-vs.-host disease Clinical data on compassionate use after allogeneic hematopoietic stem cell transplantation showed all 8 patients experienced at least partial response, with 3 achieving complete response, 2 with very good partial response and 3 with partial response
Magenta Therapeutics Inc., of Cambridge, Mass. MGTA-145 Stem cell mobilizer Hematopoietic stem cell transplant Data from healthy volunteers showed drug engages CXCR2 on neutrophils to mobilize CD34+ cells into peripheral blood with limited neutrophil activation; 5 of 6 subjects who received a single dose of MGTA-145 at 0.03 dose level and plerixafor mobilized more than 20 CD34+ cells/microliter, the clinically accepted threshold for successful mobilization, in a single day
Magenta Therapeutics, of Cambridge, Mass. CD117-ADC Targets CD117; antibody-drug conjugate Sickle cell disease and beta-thalassemia Preclinical results show a single dose of CD117-ADC achieves the same level of depletion as 4 doses of busulfan chemotherapy to enable successful engraftment and persistence of stem cells modified with the β-globin gene, which in its mutated form causes sickle cell disease and beta-thalassemia
Mustang Bio Inc., of New York MB-107 Lentiviral gene therapy X-linked severe combined immunodeficiency Phase I/II data presented by St. Jude's Children's Research Hospital and the NIH show MB-107 preceded by low-dose busulfan conditioning continues to be well-tolerated and results in development of functional immune system in newly diagnosed infants; enhanced transduction procedure is demonstrating improvements in older patients who received prior hematopoietic stem cell transplantation
Oncolytics Biotech Inc., of San Diego Pelareorep Intravenously delivered immuno-oncolytic virus Multiple myeloma Preclinical and clinical data demonstrate synergies between pelareorep and proteasome inhibitor carfilzomib through inflammation, apoptosis and tumor responses, supporting scientific rationale of the ongoing phase Ib trial; pelareorep treatment selectively infected multiple myeloma cells and not normal bone marrow cells
Onconova Therapeutics Inc., of Newtown, Pa. Rigosertib Small-molecule Ras mimetic Myelodysplastic syndromes At entry of the Inspire trial, 50 different mutations were identified at baseline prior to patients receiving rigosertib or physician's choice; phase II data of oral rigosertib combined with azacitidine as a first-line therapy produced an overall response rate of 90% and a complete response rate of 34%
Oncopeptides AB, of Stockholm Melflufen Peptide-drug conjugate Relapsed/refractory multiple myeloma Follow-up data from pivotal phase II Horizon study showed overall response rate of 29% and clinical benefit rate of 37% in patients with late-stage disease; NDA expected in first half of 2020
Oncopeptides AB, of Stockholm Melflufen Peptide-drug conjugate  Relapsed/refractory multiple myeloma Phase II Anchor (OP-104) triple-combination study data of melflufen and dexamethasone with daratumumab or bortezomib demonstrated positive efficacy; with daratumumab, the overall response rate was 76% with a median progression-free survival of 14.3 months; with bortezomib the ORR was 67%; both combinations were well-tolerated
Oncternal Therapeutics Inc., of San Diego Cirmtuzumab Antibody that binds to ROR1 Chronic lymphocytic leukemia and mantle cell lymphoma Phase I/II interim data of cirmtuzumab and ibrutinib show patients with CLL achieved overall best objective response rate of 85% (29 of 34) with progression-free survival of 100%; 1 patient achieved a complete response and 5 had stable disease; cirmtuzumab was well-tolerated; inhibition of ROR1 signaling was shown to reverse gene expression signatures associated with cancer-cell-stemness, oncogenic dedifferentiation and inflammation
Oryzon Genomics SA, of Madrid, Spain Iadademstat Small-molecule LSD1 inhibitor Acute myeloid leukemia New data from phase II Alice trial in elderly patients in combination with azacitidine show 6 of 8 (75%) achieving objective responses; of those, 2 were complete remission, 3 were complete remissions with incomplete hematologic recovery and 1 was partial remission; 2 of 5 patients (40%) receiving more than 3 cycles also have become transfusion independent 
Portola Pharmaceuticals Inc., of South San Francisco Cerdulatinib Oral SYK/JAK inhibitor Non-Hodgkin lymphoma subtypes Interim phase IIa data showed good tolerability and a 22% complete response in patients with relapsed/refractory peripheral T-cell lymphoma, as well as an 8% complete response in patients with cutaneous T-cell lymphoma; partial responses were seen in 15% and 35% of patients, respectively; there was a 52% overall response rate observed with angioimmunoblastic T-cell lymphoma
Precision Biosciences Inc., of Durham, N.C. PBCAR-0191 Targets the cancer cell surface protein CD19 Relapsed/refractory non-Hodgkin lymphoma and R/R B-cell precursor acute lymphoblastic leukemia Phase I data showed the NHL cohort achieved day 28-plus objective response rate of 66% (4 of 6) including 1 complete response and 3 partial responses; B-ALL cohort achieved ORR of 33% with 1 CR (1 of 3)
Principia Biopharma Inc., of South San Francisco PRN-1008 Oral, reversible, covalent inhibitor of BTK Immune thrombocytopenia Data from phase I/II trial in 31 highly treatment-resistant and refractory patients showed 39%, irrespective of dose and duration of treatment, achieved primary endpoint of ≥2 consecutive platelet counts of ≥50,000/µL, separated by at least 5 days, and increased by ≥20,000/µL from baseline, without requiring rescue medication; 45% achieved any 2 platelet counts ≥50,000/µL; most patients who achieved primary endpoint had a platelet count >30,000/µL by the first week of treatment
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. REGN-5458 BCMAxCD3 bispecific antibody Relapsed or refractory multiple myeloma Initial clinical data showed responses in 4 of 7 (57%) patients, including 3 of 4 (75%) in the 6-mg dose group
Roche Holding AG, of Basel, Switzerland Venclexta/Venclyxto (venetoclax) Binds and inhibit BCL-2 protein Chronic lymphocytic leukemia Updated data from pivotal phase III CLL14 study in combination with Gazyva (obinutuzumab) in previously untreated patients who had co-existing medical conditions showed, at median follow-up of more than 3 years, higher rates of minimal residual disease (MRD)-negativity in peripheral blood (76% vs. 35%; p<0.001) and bone marrow (57% vs. 17; p<0.001%) at the end of treatment vs. Gazyva/chlorambucil, respectively; MRD-negativity observed in 42% of those treated with combination who achieved complete response in peripheral blood vs. 14% for those in Gazyva/chlorambucil group (p<0.001); in bone marrow, MRD-negativity was observed in 34% vs. 11% (p<0.001)
Roche Holding AG, of Basel, Switzerland Venclexta/Venclyxto (venetoclax) Binds and inhibit BCL-2 protein Chronic lymphocytic leukemia 4-year follow-up data from pivotal phase III Murano study in combination with rituximab in relapsed/refractory patients showed reduced risk of disease progression or death by 81% vs. bendamustine/rituximab (p<0.0001); 4-year progression-free survival estimates were 57.3% vs. 4.6%, respectively
Rocket Pharmaceuticals Inc., of New York RP-L102 Lentiviral vector-based gene therapy Fanconi anemia Preliminary phase I results from 2 pediatric patients (ages 5 and 6) treated with Process B RP-L102 prior to development of severe bone marrow failure showed both exhibited early signs of engraftment based on peripheral blood, vector copy number and/or MMC-resistance; preliminary phenotypic correction also apparent in both
Rocket Pharmaceuticals Inc., of New York RP-L102 Lentiviral vector-based gene therapy Leukocyte adhesion deficiency-1 Preliminary phase I/II data from first pediatric patient show early evidence of safety and potential efficacy; early signs of engraftment with myeloid-lineage vector copy number levels of 1.5 at 3 months and CD18 expression of 45%, compared to pre-treatment CD18 expressions of <1%.; patient also displayed visible improvement of multiple disease-related skin lesions 
Sangamo Therapeutics Inc., of Brisbane, Calif., and Pfizer Inc., of New York SB-525 Gene therapy Severe hemophilia A Updated data from phase I/II Alta study showed SB-525 was generally well-tolerated and demonstrated sustained increased factor VIII levels through to 44 weeks, the extent of follow-up for longest treated patient in 3e13 vg/kg dose cohort
Sangamo Therapeutics Inc., of Richmond, Calif. ST-400 Ex vivo gene-edited cell therapy Transfusion-dependent beta-thalassemia First 3 patients in phase I/II Thales study showed for patient 1, on-target indels in the infused ST-400 product were 23%, and the CD34+ cell dose was 5.4 x 106 cells/kg; for patient 2, on-target indels in the ST-400 product were 73%, with a CD34+ cell dose of 3.9 x 106 cells/kg; for patient 3, on-target indels in the ST-400 product were 54%, with a CD34+ cell dose of 10.3 x 106 cells/kg
Seattle Genetics Inc., of Bothell, Wash. Adcetris (brentuximab vedotin) Antibody-drug conjugate directed to CD30 Front-line Hodgkin lymphoma Long-term follow-up analyses from phase II study in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.) in patients 60 and older showed 18 of 19 evaluable patients (95%) had objective response, including 13 (68%) with complete response and 5 (26%) with partial response; all response-evaluable patients experienced tumor reduction; median duration of response not yet reached 
Seattle Genetics Inc., of Bothell, Wash. Adcetris (brentuximab vedotin) Antibody-drug conjugate directed to CD30 Relapsed/refractory classical Hodgkin lymphoma 2-year follow-up from phase I/II study in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.) showed of 91 treated patients, 85% (77/91) had objective response, including 67% (61/91) with complete response, 16 with partial response and 6 with stable disease; of 91 treated patients, 67 received autologous stem cell transplant per protocol with no additional salvage therapy
Seattle Genetics Inc., of Bothell, Wash., and Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Adcetris (brentuximab vedotin) CD30-targeting antibody-drug conjugate Stage III or IV frontline classical Hodgkin lymphoma 4-year post-hoc exploratory analysis showed progression-free survival rate for patient receiving Adcetris plus adriamycin, vinblastine and dacarbazine was 81.7% vs. 75.1% in adriamycin, bleomycin, vinblastine and dacarbazine arm, a difference of 6.6%, representing a 31% reduction in the risk of progression or death; median follow-up time was 48.4 months
Seattle Genetics Inc., of Bothell, Wash., and Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Adcetris (brentuximab vedotin) CD30-targeting antibody-drug conjugate CD30+ peripheral T-cell lymphoma Analyses from Echelon-2 trial in subset of patients who underwent consolidative stem cell transplant showed combination with cyclophosphamide, doxorubicin, prednisone vs. cyclophosphamide, doxorubicin, vincristine, prednisone showed progression-free survival estimate favored the use of stem cell transplant; after a median follow-up time of 35.9 months, the 3-year PFS rate for the 38 patients who received a stem cell transplant was 76.1%, while after a median follow-up time of 41.6 months, the 3-year PFS rate for the 76 patients who did not receive a stem cell transplant was 53.3%
Sierra Oncology Inc., of Vancouver, British Columbia Momelotinib JAK1/2 and ACVR1 inhibitor Myelofibrosis New phase III analyses show patients had significantly decreased transfusion requirements vs. those treated with Jakafi (ruxolitinib, Incyte Corp.), including nearly 10-fold higher odds of receiving no transfusion during 24-week study period (p<0.0001); patients had significantly reduced chance of receiving 1 transfusion, 2 transfusion events or 3 transfusion events; mean cumulative number of red blood cell (RBC) units received for typical patient was about half of that for patients on Jakafi (p<0.0001); Kaplan-Meier time-to-first RBC unit transfused analysis indicated immediate and sustained treatment effect vs. ruxolitinib (p<0.0001) 
Syros Pharmaceuticals Inc., of Cambridge, Mass. Fetal hemoglobin repressor Nuclear factor IX Sickle cell disease Preclinical work discovered and validated nuclear factor I X, using company’s gene control platform, showing how gamma-globin gene, which leads to production of fetal hemoglobin, is controlled and points to new potential targets for therapeutic intervention in SCD
Takeda Pharmaceutical Co., of Osaka, Japan Ninlaro (ixazomib)  Oral proteasome inhibitor Relapsed/refractory systemic light-chain amyloidosis Additional data from phase III Troumaline-AL1 study in combination with dexamethasone, which reported not meeting first of 2 primary endpoints in June 2019, showed complete response rate of 26% vs. 18% in physician’s choice arm; other endpoint data showed median duration of hematologic response was 46.5 months vs. 20.2 months in physician’s choice; vital organ progression-free survival was 18 months and 11 months, respectively, and hematologic PFS was 20.1 months and 16.7 months, respectively; time to treatment failure was 10.1 months and 5.2 months, respectively
TG Therapeutics Inc., of New York Ublituximab (TG-1101) and umbralisib (TGR-1202) Glycoengineered anti-CD20 monoclonal antibody and dual inhibitor of PI3K delta and CK1 epsilon Relapsed/refractory chronic lymphocytic leukemia Triple-therapy data from phase I/II study in combination with venetoclax showed patients followed for at least 12 months had 100% objective response rate and all achieved minimal residual disease (MRD) negativity in peripheral blood; 7 of 9 patients also achieved MRD negativity in bone marrow
TG Therapeutics Inc., of New York TG-1701 Oral BTK inhibitor B-cell malignancies Phase I data show single-agent treatment produced partial responses at multiple dose levels across multiple B-cell diseases; 86% (6/7) of patients treated with 100 mg TG-1701 plus U2 have achieved a response
TG Therapeutics Inc., of New York Ublituximab (TG-1101) and umbralisib (TGR-1202) Glycoengineered monoclonal antibody targeting CD20 and dual PI3K delta and CK1 epsilon inhibitor Relapsed/refractory chronic lymphocytic leukemia Phase I/II data testing triple combination therapy with venetoclax (Roche Holding AG) showed overall response rate of 87% 20/23) after U2 induction period at cycle 3, prior to introduction of venetoclax, in relapsed/refractory CLL patients, including patients refractory to ibrutinib
Unum Therapeutics Inc., of Cambridge, Mass. ACTR-707 Antibody-coupled T-cell receptor engineered T-cell therapy Relapsed/refractory CD20+ non-Hodgkin lymphoma Ongoing phase I trial in combination with rituximab showed complete response achieved in 40% (8 of 20) of patients in cohorts 1-4; of 8 complete responders, 4 remained in completed response at 6 months follow-up, 2 remain in complete response but have not yet reached 6-month timepoint for evaluation and 2 progressed before 6 months
Unum Therapeutics Inc., of Cambridge, Mass. ACTR-087 Antibody-coupled T-cell receptor engineered T-cell therapy Relapsed/refractory CD20+ non-Hodgkin lymphoma Data from phase I ATTCK-20-2 trial in combination with rituximab generated complete responses lasting greater than 6 months in 20% (4 of 20) of evaluable patients
Verastem Inc., of Boston Copiktra (duvelisib) PI3K inhibitor Relapsed/refractory peripheral T-cell lymphoma Results from dose-optimization portion of phase II Primo study showed objective response rates of 35% and 54% in cohorts 1 and 2, respectively, with complete response rates of 25% and 31%, respectively 
Viracta Therapeutics Inc., of San Diego Nstat (nanatinostat) Oral HDAC inhibitor EBV-associated relapsed/refractory lymphomas Phase Ib/IIa data in combination with valganciclovir showed overall response rate in phase Ib portion of 56% (10/18), with 28% (5/18) complete response and clinical benefit rate of 78%; median duration of treatment for responders of 6.5 months; in HIV-negative patients, ORR was 67% (10/15), with a 33% (5/15) CR and a CBR of 93% (14/15)
Xencor Inc., of Monrovia, Calif. XmAb®13676 CD20 x CD3 bispecific antibody B-cell malignancies Data from ongoing phase I study showed XmAb13676 was generally well-tolerated, and a priming dose of 45 mcg/kg was chosen for continued dose escalation in part B for patients with non-Hodgkin lymphoma
Xenikos BV, of Nijmegen, the Netherlands T-Guard  2 toxin-conjugated monoclonal antibodies targeting CD3 and CD7 molecules on T cells and NK cells Steroid-refractory acute graft-vs.-host disease Data from expanded access program as second- or third-line treatment to 12 high-risk adults following allogeneic stem cell transplantation for a myeloid or lymphoid malignancy showed 75% achieved clinical response by day 28, with 5 of 9 achieving complete remission; 6-month and 1-year overall survival rate was 75% and 58%, respectively
Ziopharm Oncology Inc., of Boston T-cell therapies Sleeping Beauty-modified T cells expressing membrane bound IL-15 Tumors Preclinical data showed T cells can be genetically modified with Sleeping Beauty system to express TCR and mbIL-15; T cells expressing TCR and mbIL-15 exhibited superior antitumor effects vs. TCR-modified T cells without mbIL15; T cells expressing TCR and mbIL-15 persist in mouse model


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