Biotech Stock Roundup: ASH Data In Focus, Amgen Gets FDA Nod To Remicade Biosimilar

This week, the focus was on data presented at the annual conference of the American Society of Hematology ("ASH") from Dec 7-10. Apart from this, the regular pipeline updates were also significant to the biotech sector.

Recap of the Week’s Most Important Stories:

ASH Roundup: Several companies were at ASH with data on approved and pipeline drugs.

Kite, a Gilead Sciences, Inc. (NASDAQ:GILD) company, announced long-term data from the ZUMA-1 study on CAR T cell therapy, Yescarta (axicabtagene ciloleucel), in adult patients with refractory large B-cell lymphoma at the 61st ASH Annual Meeting & Exposition in Orlando.

This included updated overall survival (OS) data from the phase II study after three years, following a single infusion of Yescarta as well as an analysis from a separate safety management cohort of patients receiving early steroid intervention for cytokine release syndrome (CRS) and neurologic events. With a minimum follow-up of three years after a single infusion of Yescarta (median follow-up of 39.1 months), approximately 47% of patients with refractory large B-cell lymphoma in ZUMA-1 phase II cohorts were alive, while the median OS was 25.8 months.

The company also announced primary results from ZUMA-2, a global, multicenter, single-arm, open-label phase 2 study of KTE-X19, which is an investigational CD19 CAR T cell therapy, in adult patients with relapsed or refractory mantle cell lymphoma (MCL). In the study, 93% of patients treated with investigational KTE-X19 achieved response.

bluebird bio, Inc. (NASDAQ:BLUE) and partner Bristol-Myers Squibb Company (NYSE:BMY) announced positive top-line results from a phase II study on their lead investigational BCMA-targeted CAR T-cell therapy candidate, idecabtagene vicleucel (ide-cel; bb2121). The phase II KarMMa study evaluated the efficacy and safety of the candidate in patients with relapsed and refractory multiple myeloma. The study met its primary endpoint and key secondary endpoint demonstrating deep and durable responses in a heavily pre-treated multiple myeloma patient population.

Shares of Fate Therapeutics, Inc. (NASDAQ:FATE) surged significantly after it reported positive data on experimental cancer candidates. The company announced initial clinical data on its candidates — FT516 and FT500. Both are off-the-shelf, induced pluripotent stem cell (iPSC)-derived natural killer (NK) cell product candidates. The initial findings show the absence of morphologic evidence of leukemia (at day 42) along with complete neutrophil recovery in the first patient treated with FT516 monotherapy for AML following the first dosing cycle. The company also announced new in vivo preclinical data for FT596, its off-the-shelf, multi-antigen targeting NK cell product candidate derived from a clonal master engineered iPSC line.

FT596 as a monotherapy demonstrated comparable anti-tumor activity to CAR19 T Cells in vivo in the humanized mouse model of lymphoma.

Amgen, Inc. (NASDAQ:AMGN) announced additional results from the primary analysis of the phase III CANDOR study, evaluating Kyprolis (carfilzomib) in combination with dexamethasone and Darzalex (daratumumab) (KdD) compared to Kyprolis and dexamethasone alone (Kd) in patients with relapsed or refractory multiple myeloma. Treatment with KdD resulted in a significant progression-free survival (PFS) benefit.

Amgen Gets FDA Nod for Remicade Biosimilar: Amgen announced that the FDA has approved its biosimilar version of Remicade (infliximab). The biosimilar will be marketed by the trade name of Avsola (ABP 710) for all approved indications of Remicade, which includes a range of autoimmune disorders. ABP 710 is an anti-TNF monoclonal antibody. However, Amgen did not mention when it plans to launch the same.

The approval was based, partly, on data from a late-stage study, which evaluated ABP 710 versus branded Remicade for the treatment of moderate-to-severe rheumatoid arthritis (RA). Data from the phase III study showed that ABP 710 was non-inferior compared to Remicade based on its primary endpoint, which was 20% improvement in American College of Rheumatology core set measurements (ACR20) at week 22.

Pipeline Updates From Regeneron: Regeneron (NASDAQ:REGN) announced top-line data from a mid-stage study on pipeline candidate, pozelimab (REGN3918), for the treatment of rare blood disorder — paroxysmal nocturnal hemoglobinuria. The results showed that pozelimab reduced the abnormal destruction of red blood cells (known as "hemolysis), with patients in the initial cohort achieving normal levels of a blood biomarker of elevated hemolysis called lactate dehydrogenase (LDH). Patients in the trial initially received a 30-mg/kg IV loading dose of pozelimab, followed by weekly 800-mg subcutaneous injections. All six patients in the initial treatment cohort treated with pozelimab experienced rapid and sustained reductions in LDH up to week 8.

Regeneron also announced initial data for REGN5458, a BCMAxCD3 bispecific antibody, in patients with relapsed or refractory (R/R) multiple myeloma — the second most common blood cancer. Results from the first two dose groups (3 mg and 6 mg weekly doses) were presented at the ASH Annual Meeting. Patients had a median of seven lines of prior systemic therapy, and all had failed in the CD38 antibody treatment arm. Responses were observed in four out of seven (57%) patients, including three out of four (75%) in the 6 mg dose group. In the 6 mg dose group, two patients (50%) were also minimal residual disease (MRD) negative, which means no cancer cells were detectable in their bone marrow. Two patients achieved the high bar of MRD negativity, and another patient attained a very good partial response despite entering the trial with difficult-to-treat plasmacytomas outside of the bone marrow.

Vertex’s Kalydeco Label Expanded in Europe: Vertex Pharmaceuticals (NASDAQ:VRTX) announced that the European Commission granted approval to the label extension for Kalydeco (ivacaftor) to include the treatment of infants with cystic fibrosis (CF) aged 6 months to less than 12 months, weighing 5 kg and more 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 label update was based on data from the ongoing, phase III, open-label safety study (ARRIVAL) of children with CF, who are less than 24 months of age and have a CFTR gating mutation. The study showed a safety profile similar to that observed in previous studies of older children and adults, and improvements in sweat chloride, a secondary endpoint.

Vertex currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Sage Plunges on Study Failure: Shares of Sage Therapeutics (NASDAQ:SAGE) plummeted after it announced that a phase III study on SAGE-217 for the treatment of major depressive disorder (MDD) failed to meet the primary endpoint. The MOUNTAIN study evaluated the efficacy, safety and pharmacokinetics of SAGE-217 in adult patients diagnosed with MDD. The phase III study did not meet its primary endpoint of a statistically significant reduction from baseline compared to placebo in the 17-item Hamilton Rating Scale for Depression (HAM-D) total score at Day 15. SAGE-217 30 mg, given once-daily as an oral treatment, was associated with a mean reduction of 12.6 in HAM-D total score compared to 11.2 for placebo.

Performance

Medical - Biomedical and Genetics Industry 5YR % Return

Medical - Biomedical and Genetics Industry 5YR % Return

The Nasdaq Biotechnology index inched up 0.81% in the last five trading sessions. Among the biotech giants, Biogen (NASDAQ:BIIB) gained 2.24% in the period. Over the past six months, shares of Amgen have gained 35.02%. (See the last biotech stock roundup here (See the last biotech stock roundup here: Biotech Stock Roundup: Pipeline Updates From BIIB, AXSM, CARA, Collaborations & More)

What's Next in Biotech?

Stay tuned for more pipeline updates.

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