This week, focus was on data presented at the annual conference of the American Society of Hematology ("ASH") from Dec 1-4. Apart from this, the regular pipeline updates were also in focus in the biotech sector.
Recap of the Week’s Top Stories:
ASH Roundup: Several companies were at ASH with data on approved and pipeline drugs.
Amgen, Inc. (AMGN - Free Report) announced the first clinical data from the early-stage studies on two of its investigational novel bispecific T cell engager (BiTE) immunotherapies — AMG 420 and AMG 330. Two separate phase I studies evaluated AMG 420, which targets B-cell maturation antigen (BCMA) and AMG 330, which targets CD33 in heavily pre-treated patients with multiple myeloma and acute myeloid leukemia (AML). Data from the studies showed that both AMG 420 and AMG 330 demonstrated anti-tumor activity and early evidence of tolerability in patients, with relapsed and/or refractory multiple myeloma and relapsed or refractory AML, respectively.
Kite, a Gilead (GILD - Free Report) company, announced updated results from a phase I/II study, ZUMA-3 on experimental candidate, KTE-X19, which is a CD19 chimeric antigen receptor T (CAR T) cell therapy being evaluated in adult patients, with relapsed or refractory acute lymphoblastic leukemia (ALL). ZUMA-3 is an ongoing multicenter, registrational phase I/II study in adult patients (≥18) with ALL. In the study, 69% of evaluable patients (n=25/36) achieved complete tumor remission, defined as complete remission (CR) or CR with incomplete hematological recovery (CRi), with a median follow-up of 15.1 months (range 3.7-28.6 months) following a single infusion of KTE-X19. The rate of undetectable minimal residual disease (MRD) in patients, who achieved complete tumor remission was 100%. Based on these data, Gilead initiated a phase II study, evaluating KTE-X19 in a larger set of adult patients with ALL.
Celgene (CELG - Free Report) too presented data from several studies at the ASH.
Celgene along with its partner Acceleron Pharma Inc. XLRN, announced results from a phase III study (BELIEVE) evaluating their pipeline candidate luspatercept for the treatment of adults with beta-thalassemia-associated anemia, who require regular red blood cell (RBC) transfusions. The study met the primary endpoint of erythroid response, demonstrating that luspatercept may help patients reduce their dependence on red blood cell transfusions. Both companies also presented data from phase III MEDALIST study evaluating the efficacy and safety of luspatercept to treat patients with ring sideroblast (RS+) myelodysplastic syndromes (MDS)-associated anemia, who require RBC transfusions and had failed, were intolerant to or ineligible for erythropoietin therapy. The study data demonstrated that treatment with luspatercept led to statistically significant increased red blood cell transfusion independence in the above-mentioned patients compared to placebo.
Celgene also announced results of the phase III AUGMENT study, which showed that Revlimid (lenalidomide) in combination with Rituxan led to superior progression-free survival (PFS) in patients with relapsed/refractory indolent lymphoma compared to patients who received rituximab plus placebo (R-placebo). Celgene and its partner bluebird bio, Inc. (BLUE - Free Report) announced initial data from the ongoing phase I study of bb21217 (CRB-402), an investigational next-generation anti-BCMA CAR T cell therapy, being studied in patients with relapsed/refractory multiple myeloma. Of the 12 heavily pre-treated patients who received bb21217, 83% (n=10) achieved an objective clinical response. The early safety results depicted manageable findings and were consistent with known toxicities of CAR T therapies. Celgene announced encouraging initial data from the dose-escalation part of an ongoing phase I/II study of lisocabtagenemaraleucel (JCAR017), in patients with relapsed/refractory chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), including those with cytogenetic features of high-risk disease, who were previously treated with ibrutinib.
Gilead’s Drugs Get Approval in China: Gilead Sciences, Inc. announced that the National Medical Products Administration (NMPA) has approved hepatitis C virus (HCV) drug Harvoni in China in adults and adolescents aged 12 to 18 years. The approval was supported by an open-label, phase IIIb study, which was conducted at 18 study centers in mainland China between May 2016 and July 2017. The NMPA also approved Descovy (emtricitabine 200 mg/tenofovir alafenamide 10 mg and emtricitabine 200 mg/tenofovir alafenamide 25 mg, F/TAF), a fixed-dose combination for the treatment of HIV. The drug has been approved in combination with other antiretroviral agents for the treatment of HIV-1 in adults and adolescents (aged 12 years and older with body weight at least 35 kg).
Amgen’s Osteoporosis Drug Under Review: Amgen and partner UCB announced the FDA Bone, Reproductive and Urologic Drugs Advisory Committee (BRUDAC) will review data supporting the Biologics License Application (BLA) for osteoporosis drug, Evenity, at a meeting on Jan 16, 2019. The drug is being evaluated for the treatment of osteoporosis in postmenopausal women at high risk for fracture. The Evenity clinical program comprises three pivotal phase III studies, namely FRAME, ARCH and BRIDGE. The FRAME study evaluated Evenity on postmenopausal women with osteoporosis. The ARCH study assessed the candidate on postmenopausal women suffering osteoporosis and who are at increased risk of fracture while the BRIDGE study evaluated Evenity on men with osteoporosis. The first BLA seeking approval for Evenity regarding the same disease in the United States was issued a complete response letter (CRL) from the FDA in July 2017due to a cardiovascular side effect observed in the ARCH study. However, in July 2018, Amgen along with UCB refiled the BLA for Evenity to the FDA. The second BLA now includes data from both ARCH and BRIDGE studies.
Vertex’s Kalydeco Approved in Europe: Vertex Pharmaceuticals Incorporated (VRTX - Free Report) announced that the European Commission has granted approval to the label extension of cystic fibrosis (CF) drug Kalydeco. The drug has been approved to include the treatment of CF patients aging between 12 and 24 months, who have at least one of the following nine mutations in their cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. The drug is already approved in Europe for the treatment of CF in patients aged two years and older, who have one of the nine following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. The label update was based on encouraging data from the ongoing phase III open-label safety study (ARRIVAL) that demonstrated a safety profile consistent with that observed in previous studies of older children and adults, and improvements in sweat chloride, a key secondary efficacy endpoint.
The NASDAQ Biotechnology index gained 1.76% in the last five trading sessions. Among the major biotech stocks, Regeneron gained 5.43% in the last five trading sessions. Over the past six months, shares of Regeneron have rallied 19.45%, while Celgene has dropped 7.86%. (See the last biotech stock roundup here: Biotech Stock Roundup: Pipeline Updates From CELG & VRTX, ZFGN Plunges on Hold News)
What's Next in Biotech?
Stay tuned for more regulatory and pipeline updates.
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