Blood disease specialists and doctors will soon travel to New Orleans for the 64th annual meeting of the American Society of Hematology, a high-profile stage for some of their field's most consequential research.
They, along with the many investors and analysts who track emerging treatments for blood diseases, got a peek at what's to come with the release of hundreds of study abstracts in early November. These study summaries contain important clues to what will draw the most attention at the Dec. 10-13 meeting.
Notably, there will be updates on an emerging class of protein-based cancer drugs that can simultaneously latch onto targets on cancer and immune cells. There's significant competition between drugmakers like Roche, Regeneron, Johnson & Johnson, Pfizer and AbbVie, which are developing rival treatments for lymphoma and multiple myeloma.
While ASH is often a forum for the latest in blood cancer research, it's also historically been an important conference for gene therapy in blood diseases like beta thalassemia, sickle cell and hemophilia. This year is no different and will be buoyed by the recent approval of a Bluebird bio treatment.
Read on for three notable areas of focus at this year’s meeting.
Competitors crowd behind Roche’s new lymphoma drug …
Earlier this year, European regulators cleared a first-of-its-kind drug from Roche for a type of lymphoma. Sold as Lunsumio, the drug is a two-pronged antibody that latches onto CD20, a target on the B cells that proliferate in lymphoma, and CD3, a protein on tumor-fighting T cells. Its approval was the latest step forward for antibody drugs known as bispecifics, which have become a target for drugmakers and have produced multiple marketed treatments for cancers, eye diseases and hemophilia. U.S. regulators are set to make a decision on Lunsumio by late December.
Yet Lunsumio likely won’t be alone on the market for long. AbbVie and Genmab recently completed approval applications in the U.S. and Europe for a rival drug. And ASH abstracts posted Nov. 4 show the range of competitors following behind them.
Regeneron revealed results from a Phase 2 study of its drug odronextamab in two different types of lymphoma that analysts at the investment firm SVB Securities called “competitive” in both disease settings. Those results included a 53% response rate in diffuse large B-cell lymphoma and an 81% response rate in follicular lymphoma. Remission rates were 37% and 75%, respectively, after a median of roughly 17 months of follow-up. However, two patients with follicular lymphoma died from treatment-related adverse events.
In a small Phase 1 study of heavily pretreated patients, Johnson & Johnson’s plamotamab, licensed from the biotechnology company Xencor, led to a 47% response rate and 26% remission rate in DLBCL. All follicular lymphoma patients responded, and half were in remission as of data cutoff.
Multiple developers, including AbbVie and Roche, revealed data showing use of these drugs in combinations. Some regimens have advanced into Phase 3 testing. They're also being evaluated in rarer lymphoma types, like mantle cell, which “demonstrates the breadth of potential” for the drug class, SVB analysts wrote.
…and J&J’s first-of-its-kind multiple myeloma drug
The competition is equally strong among developers of dual-targeting antibodies for multiple myeloma.
A week ago, the Food and Drug Administration made J&J’s Tecvayli the first such treatment to be commercially available. But J&J’s drug has behind it a growing pipeline of treatments, including experimental medicines from Pfizer, Regeneron, AbbVie, Roche and Bristol Myers Squibb. They all aim to surpass Tecvayli, as well as provide a more convenient alternative to the personalized CAR-T therapies now available for very sick multiple myeloma patients.
Most of these drugs target a protein called BCMA that’s found on malignant cells on multiple myeloma. And most showed “generally favorable efficacy and tolerability characteristics,” SVB analysts wrote. But newer approaches are emerging too, some of which will be featured at ASH.
Pfizer and Regeneron are among those presenting updated study results. Pfizer’s drug elranatamab had a 61% response rate in a Phase 2 study and no instances of a common immune-related side effect that were graded as severe. Analysts at Evercore ISI flagged a severe side effect related to low potassium levels, however.
The response rates of patients who received higher doses of Regeneron’s linvoseltamab in a Phase 1/2 trial were higher than 75%, but treatment came with a variety of blood-related side effects. Regeneron is moving forward with a lower dose.
J&J and Roche will each present results from drugs directed at newer targets. Both have medicines aimed at a protein called GPRC5D that’s overexpressed in the bone marrow of myeloma patients. Both led to similar response rates of over 70%.
Roche has results from two studies of a second medicine targeting FcRH5, which is expressed on B cells and plasma cells.
AbbVie will present data from a myeloma drug it licensed from Harpoon Therapeutics in 2019. That drug is one of two antibodies for multiple myeloma the company will be highlighting at ASH. AbbVie acquired the other when it bought TeneoOne last year.
An approved gene therapy and an advancing pipeline
Each year for much of the past decade, ASH attendees have heard regular updates on Bluebird's progress developing a gene therapy for beta thalassemia.
This year, the latest data on the treatment will carry new significance, as in August the FDA granted the therapy a landmark approval for patients with severe forms of the blood disorder.
Abstracts released Nov. 4 show that research on the long-term outcomes of patients given the therapy in clinical testing will be presented, as will data on changes in treated individuals' quality of life. The results confirm the treatment's dramatic impact, with nearly all patients still free from the blood transfusions they previously needed on a regular basis. Patients surveyed post-treatment also reported improvements in their well-being.
Such research is likely to remain a staple of ASH conferences to come, as more gene therapies advance through testing and, possibly, reach the market. Also being presented are multi-year follow-up on hemophilia patients treated with a gene therapy being developed by Roche and its subsidiary Spark Therapeutics. There have been few updates on that treatment since Roche's buyout of Spark in 2019, so that presentation could attract interest.
Researchers associated with a half dozen medical institutions and Novartis' research labs, meanwhile, will present new data on a gene-edited stem cell-based treatment for severe sickle cell disease.