This week, in typical years, doctors and researchers specializing in blood diseases and cancers would be en route to San Diego, where the closely followed annual meeting of the American Society of Hematology is regularly held.
The conference this year, of course, is virtual, beamed to thousands of laptops around the world starting in earnest tomorrow and running through Tuesday. An online ASH won't lack high-impact research, though.
Results from key studies of cutting-edge therapies for beta-thalassemia, sickle cell disease and hemophilia are expected, as well as from trials testing new approaches to treating multiple myeloma, leukemia and lymphoma.
Here are four storylines we'll be covering:
Multiple myeloma cell therapies are nearing approval. Could antibody drugs steal their spotlight?
The race to find new treatments for advanced multiple myeloma — a persistent and deadly bone marrow cancer — by homing in on a protein known as B-cell maturation agent, or BCMA, gets more competitive each year.
The Food and Drug Administration approved the first BCMA-targeting agent earlier this year, an antibody drug from GlaxoSmithKline called Blenrep. This weekend at ASH will bring new results for many more, as several drugmakers are set to present results on a group of cell therapies and antibody-based drugs quickly progressing through human testing. Two separate Saturday sessions, in fact, are scheduled, a reflection of the many programs being developed.
Bristol Myers Squibb and Bluebird bio have long been in the lead with an autologous cell therapy, ide-cel, that could be approved by the FDA next year. Multiple delays have slowed its path to market, though, allowing several rival programs to gain ground.
Chief among them is a CAR-T therapy from Johnson & Johnson and Legend Bio, cilta-cel, that could also be under regulatory review next year. The two companies are expected to report new data from a trial known as CARTITUDE. Bristol Myers and Bluebird will have the latest on ide-cel, too. Results from CARSgen Therapeutics, Cellular Biomedicine Group and Poseida Therapeutics are also expected.
Off-the-shelf cell therapies, which are far less complex to administer than autologous treatments, are also progressing, though they remain early Initial Phase 1 results from a biotech called Allogene Therapeutics were mixed: more clarity on those data will come at a session on Saturday.
This year, a large slate of antibody-based drugs targeting BCMA are also being showcased. As with off-the-shelf cell therapies, these drugs would be easier to administer than autologous cell treatment. Early-stage results from Regeneron, Amgen, AstraZeneca and Johnson & Johnson are scheduled to be presented.
CRISPR gene editing takes center stage in beta-thalassemia, sickle cell
This weekend brings the latest update from a pioneering program meant to treat two chronic blood diseases using CRISPR gene editing technology. The treatment, from partners CRISPR Therapeutics and Vertex, has already shown promise in both diseases, with the first three patients — two with beta thalassemia, the other with sickle cell disease — responding to treatment.
The program's progress has highlighted the potential of the Nobel Prize winning technology, which has only recently moved into human testing after exciting scientists across the globe. And it is taking center stage at an ASH plenary session, the conference's main research showcase. A study abstract touts the treatment as a “potential functional cure” for both diseases.
At the presentation, CRISPR and Vertex will show whether the results from those three patients have held up, and if others in the study have benefited similarly. If so, that will put even more pressure on the gene therapy LentiGlobin, from Bluebird bio, which is approved in Europe but has been delayed in the U.S. multiple times. A U.S. approval filing is now expected in 2022.
The next test of an $8 billion cancer drug buyout
Nearly two years ago, Eli Lilly paid $8 billion to acquire Loxo Oncology, betting big on two targeted cancer drugs developed by the biotech.
The first, a treatment for certain lung and thyroid tumors, won approval earlier this year and is now sold as Retevmo. This weekend, researchers will share key data on the second, a blood cancer therapy that Lilly hopes can challenge medicines from AbbVie, AstraZeneca and Beigene.
Known as LOXO-305, the drug, like its would-be rivals, blocks a protein called Bruton's tyrosine kinase that plays an important role in the development of B cells, which are involved in several types of leukemias and lymphomas.
On Saturday, and then again on Monday, Lilly will present results from a Phase 1/2 study of LOXO-305 in patients with chronic lymphocytic leukemia or mantle cell lymphoma, as well as several related cancers. Data included in study abstracts, made public last month, showed encouraging rates of response among tested patients, many of whose cancer had already progressed following treatment with one of the commercially available BTK inhibitors.
LOXO-305 binds to BTK in a different fashion than those drugs and, Lilly argues, does so more selectively, potentially helping the therapy's safety and potency. The ASH results will be a major proving point for that hypothesis.
A gene therapy for hemophilia B nears the finish line
This year's ASH will include the most detailed look yet at a leading gene therapy for hemophilia B, the less common form of the rare bleeding disorder.
Hemophilia has long been a target for gene therapy research, as it's caused by mutations in one of the sex chromosomes. But while drugmakers have been able to develop more than a dozen effective medicines that keep the disease in check, they have yet to prove a one-time, permanent fix.
That's what UniQure, a Netherlands-based biotech, has been working toward for nearly a decade. Using technology from its precursor company, Amsterdam Molecular Therapeutics, the biotech is closest to bringing a hemophilia B gene therapy to market. Its most advanced therapy, named AMT-061, is being studied in a late-stage clinical study called HOPE-B, data from which will be presented Tuesday.
Already, UniQure has given a glimpse of the results, which ASH picked as a late-breaking abstract. Six months after receiving AMT-061, most of the 54 patients in the HOPE-B trial were no longer having bleeding events, and all but two of them were able to stop routine preventive treatment. Notably, the trial enrolled patients with moderately severe to severe hemophilia B, meaning they have very low levels of blood clotting protein and are at higher risk of experiencing uncontrolled bleeds.
The data did leave some questions, though. Fifteen of the patients still had bleeds after treatment with AMT-061. Some of those patients also required standard clotting replacement treatment — noteworthy, since one of the draws to gene therapy is that it's supposed to eliminate the need for these treatments.
Still, the results appear to have encouraged hemophilia doctors and UniQure investors. The biotech said it expects the trial to support an approval application to the Food and Drug Administration next year.
Spark Therapeutics, which is now a part of Roche, and partner Pfizer also have a hemophilia B gene therapy in late-stage testing.