ASH 2017 Highlights: Gilead, Novartis, Juno, Bluebird Bio And More

The curtains have come down on one of the most-watched annual biotech conferences. The 59th annual meeting of the American Society of Hematology was held Dec. 9-12 in Atlanta. 

This year's meeting was all about competition in the CART-T space. Immunotherapy — the fifth pillar of cancer treatment after surgery, radiation, chemotherapy and targeted therapies — has seen rapid evolution, with an approach called adoptive cell transfer, or ACT, gaining prominence.

A Three-Way Race For Lymphoma Treatment

CAR, or chimeric antigen receptor, T-cell therapy is a type of ACT where T cells responsible for immunity are removed from a patient and modified so  they express receptors targeting the patient's cancer cells. The cells are subsequently reintroduced into the patient.

Novartis AG (ADR) NVS got the FDA nod in August 2017 for Kymriah, its CAR-T cell version, for treating certain forms of acute lymphoblastic leukemia in children and adults. Kymriah was priced at $475,000 for a single infusion.

Yescarta, a CAR-T cell therapy developed by Kite Pharma Inc KITE, a unit of Gilead Sciences, Inc. GILD has also been approved for treating adult non-Hodgkin lymphoma. A single injection of Yescarta is priced at $373,000.

Juno Therapeutics Inc JUNO is also vying for a piece of the lucrative CAR T-cell therapy cake.

With competition heating up, this year's presentations in the CAR-T arena were looked upon with keen interest.

At ASH 2017, Gilead's Kite unit presented data showing that, at a median of 15.4 months after Yescarta infusion, 42 percent of previously treated non-Hodgkin's lymphoma patients continued to show response to the therapy, with 40 percent showing complete response.

This longevity in response is a sine-qua-non, given the high cost associated with each infusion.

Data from Novartis' Juliet trial showed an overall response rate, or ORR, of 53 percent and complete response, or CR, of 53 percent in patients using Kymriah. At six months, 30 percent showed CR, with a 74-percent relapse-free rate after the onset of response.

Juno presented new insights on clinical outcomes with its investigational CAR-T product candidate JCAR017 at the ASH. The additional data from a trial dubbed TRANSCEND showed 74 percent ORR and 68 percent CR, at three months. At six months, the CR was 50 percent.

JCAR017 has better safety profile in relapsed and refractory aggressive non-Hodgkin Lymphoma as compared to Kymriah and Yescarta, according to Raymond James analyst Reni Benjamin.

Following the ASH presentations, Novartis showed very little market reaction and Gilead gained about 2.5 percent, while Juno shares have fallen roughly 22 percent. Notwithstanding the fairly encouraging data, investors appear to be skeptical about Juno's competitive positioning.

See also: Attention Biotech Investors: Mark Your Calendar For These December PDUFA Dates

Multiple Myeloma Treatments In The Pipeline

Multiple myeloma is the cancer of plasma cells formed when a type of lymphocytes, namely B cells mature in response to infection. Efforts are on to target this form of cancer with cell therapy, whereby immune cells are engineered to attack cells with B-cell maturation antigen, or BCMA, protein.

At the ASH, bluebird bio Inc BLUE and partner Celgene Corporation CELG announced updated results from the ongoing multicenter Phase 1 study of bb2121 anti-BCMA CAR-T cell therapy in patients with late-stage relapsed/refractory multiple myeloma.

The results showed a 94 percent overall response rate in patients in active dose cohorts.

Cantor Fitzgerald's Elemer Piros said the data provided a strengthened profile for the therapy. Piros ascribes a "high" 60 percent probability of success for the program. Bluebird could share peak sales of $2.6 billion by 2026 with partner Celgene, he said. 

Bluebird also said a second-generation CAR-T therapy candidate called bb212117 has begun initial testing.

Novartis presented Phase 1 trial data for its CART-BCMA therapy, which evaluated 10 patients. The data confirmed the significant clinical efficacy of the therapy.

GlaxoSmithKline plc (ADR) GSK is another contender in the space. At ASH, the company presented positive results from a mid-stage study of its GSK2857916, a humanized anti-BCMA monoclonal antibody conjugated to the cytotoxic agent monomethyl auristatin-F.

The DREAMM-1 study showed that heavily pre-treated multiple myeloma patients demonstrated a 60-percent response rate and a median progression free survival, or PFS, of 7.9 months.

While bluebird bio shares jumped about 18 percent Monday in response to the ASH presentation, they have given back some of their gains since then. Partner Celgene gained about 6 percent over two sessions following the presentation. GlaxoSmithKline hasn't reacted much.

Hemophilia Treatments

Spark Therapeutics Inc ONCE presented preliminary data from a Phase 1/2 trial on SPK-8011, a treatment candidate for hemophilia A, which showed a 100-percent reduction in annualized bleeding rate, or ABR, beginning four weeks after the gene therapy, and a 98-percent reduction in annualized infusion rate, or AIR, in the first four participants.

BioMarin Pharmaceutical Inc. BMRN presented new results for gene therapy valoctocogene roxaparvovec. The new 1.5-year results showed that a 6e13 vg/kg dose achieved sustained factor VIII levels within the normal range in severe hemophilia A for most patients.

Spark's stock fell hard Monday as the levels of factor VIII, the missing clotting factor in hemophilia A patients, did not improve as much as with BioMarin's therapy.

Spark Therapeutics shares have slumped about 40 percent in the aftermath of the presentation, while BioMarin has added about 7 percent.

Other Key Presentations:

Global Blood Therapeutics Inc GBT presented the results of a Phase 2a study of once-daily oral therapy voxelotor used to treat adolescents with sickle cell disease. The results showed increased hemoglobin levels and increased measures of hemolysis at 16 weeks, with a median hemoglobin change of 1.1 g/dL, reduced symptoms at 16 weeks and favorable tolerability profile in adolescents.

The stock has lost about 16 percent since the presentation, apparently in reaction to the news that two of the seven patients involved in the study died.

Syros Pharmaceuticals Inc SYRS presented new preclinical data on SY-1365, its first-in-class selective cyclin dependent kinase 7, or CDK7, inhibitor currently in Phase 1 clinical trial in advanced solid tumors. The preclinical data showed anti-tumor in in-vitro and in-vivo models of blood cancers.

Phase II data of SY-1425 showed biological and clinical activity as a single agent in genomically defined AML and myelodysplastic syndrome, or MDS, although just one of 48 evaluable patients showed CR. The company now plans to test SY-1425 in combination with azacitidine, a standard chemotherapy drug, and Darzalex from Johnson & Johnson JNJ.

Syros plunged 32 percent Monday in reaction to the ASH presentation.

Related Link:

11 Biotech Stocks Moving From ASH 2017