8:21 PM
Dec 08, 2017
 |  BioCentury  |  Product Development

Multimodal meeting

ASH 2017 by the numbers: cell and gene therapies making inroads

Editor's Note: This article was updated on Sep 14, 2018 at 6:40 PM PDT

This year’s clinical abstracts at the American Society of Hematology meeting hint at how newer treatment modalities -- cell therapies in cancer and gene therapies in hematology -- will branch out into new indications and fit into evolving treatment landscapes.

In advance of the conference, which runs Dec. 9-12 in Atlanta, BioCentury performed a series of manual and algorithm-guided analyses on 4,429 abstracts and identified 2,027 that concerned clinical studies of therapeutics, diagnostic or prognostic biomarkers.

Leukemias dominated the cancer abstracts this year -- among all the blood cancers mentioned in the abstracts, just over half were leukemias, while 26% were lymphomas and the remaining 23% were either myelodysplastic syndrome (MDS) or multiple myeloma (MM).

Like last year, acute myelogenous leukemia (AML) was the cancer subtype most often mentioned in the clinical abstracts, a trend that shows no signs of slowing. AML made up 28% of indications mentioned among preclinical blood cancer abstracts (see “ASH 2017: Top Indications”).

In BioCentury Innovation’s analysis of preclinical abstracts, AML also was the indication mentioned most often, and also had the greatest share of emerging targets, holding out the possibility of more novel clinical programs to come. BioCentury Innovations, which published its analysis on Dec. 7, defined emerging targets as proteins or genes mentioned in one or two ASH abstracts in 2016 and at least three abstracts in 2017, or in no abstracts in 2016 and at least four in 2017.

Acute lymphoblastic leukemia (ALL) was the second most common leukemia mentioned, thanks in part to a newly approved therapy, and in part to a bevy of late-stage therapies that feature new modalities beyond small molecules and mAbs.

Eight abstracts discussed treatment with Besponsa inotuzumab ozogamicin, Pfizer Inc.’s antibody-drug conjugate (ADC) that was approved for the indication in August. At least 24 described clinical use of chimeric antigen receptor (CAR) T cell therapies targeting CD19 or CD22.

Figure: ASH 2017: Top indications

Within cancer, acute myelogenous leukemia (AML) once again dominated abstracts published ahead of the 2017 American Society of Hematology conference in Atlanta, appearing in more than 400 clinical abstracts. References to AML, which was the top cancer indication in 2016, made up over 40% of the clinical mentions of leukemias, and occurred nearly twice as often as the next most commonly cited cancer indication, multiple myeloma (MM).

The two most commonly cited hematology indications -- neutropenia and thrombocytopenia -- were most often mentioned as adverse events or exclusion criteria, rather than as subjects of new drug development. Sickle cell disease (SCD) was the most commonly mentioned subject of new drug development, featuring multiple new targets and therapeutic modalities.

Out of over 4,000 abstracts posted in advance of the conference, BioCentury identified about 2,000 pertaining to clinical research. BioCentury assigned abstracts to one or more indications based on mentions of the indications in the abstract text, independent of context. Source: ASH abstracts as of Nov. 1

Sickle cell stands out

In hematologic diseases outside of cancer, while neutropenia and thrombocytopenia were mentioned most often, they were not typically the subject of new drug development. Rather, sickle cell disease was the indication most often described as the focus of drug development programs.

The sickle cell abstracts gave early clinical readouts for a cluster of products with disparate mechanisms of action.

Pfizer’s PF-04447943, a phosphodiesterase-9A (PDE-9A) inhibitor, will have Phase Ib data in sickle cell. This is the first study of the compound in sickle cell, although Pfizer conducted several Phase I studies in healthy volunteers as part of an Alzheimer’s disease program that failed.

Efficacy data also will be reported for Novartis AG’s crizanlizumab, which targets P selectin (SELP; CD62P); Global Blood Therapeutics Inc.’s allosteric hemoglobin modifier voxelotor (GBT-440); Prolong Pharmaceuticals LLC’s Sanguinate, a pegylated bovine carboxyhemoglobin; and bluebird bio Inc.’s LentiGlobin BB305, a lentiviral vector delivering the human beta globin gene.

Many of these new products could be combined with Emmaus Life Sciences Inc.’s Endari L-glutamine, which in July became the first product approved for sickle cell disease in nearly 20 years. Endari is thought to reduce sickling, adhesion and the oxidative stress that can trigger vaso-occlusive crisis.

Crizanlizumab, voxelotor and LentiGlobin also featured in beta thalassemia clinical abstracts. These included long-term follow-up data for LentiGlobin. Among 18 patients with one to three years of follow up, 10 were able to discontinue transfusions, and rest had significantly reduced annual transfusions.

New targets and compounds

The clinical abstracts are rife with mentions of familiar targets -- as expected given the large number of presentations on marketed and late-stage products. But BioCentury identified 11 targets among the clinical abstracts this year that were not represented among ASH presentations last year...

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