SAN DIEGO — At the American Society of Hematology’s annual meeting, pharmaceutical branding is inescapable.
Johnson & Johnson’s and GSK’s new logos are splashed across the side of phone charging stations. Sanofi, among others, let attendees know via hand sanitizer stands that it was proud to keep those fingers and palms germ-free.
A few, AbbVie among them, have taken a more mobile approach. The company’s navy blue logo papers the back of honking pedicabs, with drivers wearing a logoed T-shirt to match. And, in a sign of the times, the Imbruvica maker is also sponsoring a chatbot named Red, who can answer questions about shuttle buses.
The biotechnology sector may be in a bleak midwinter, but, from the looks of advertising budgets, pharma companies have plenty of cash to spare for ASH this year. Most sponsorships were sold by the start of the convention, with every $2,500 in spending giving exhibitors “priority points” toward picking their space for next year’s convention, according to a media kit.
Attendees could get in on the spending themselves, too. Among the offerings at the “fASHion” booth in the convention center were neckties ($20) and scarves ($22) patterned with red blood cells, baby onesies ($15) emblazoned with the slogan “Future Hematologist” and white pet bandanas ($10) bearing the ASH logo, paw prints and bones.
For those who could wrest their attention from all the advertising and pharma-proffered espresso, though, ASH continued to offer reams of important research Sunday and Monday.
Diversity in myeloma trials
How should researchers and drugmakers incorporate race into clinical trial design?
Studies often have specific patient eligibility criteria to test how an experimental treatment performs in different populations. But researchers don’t always prioritize demographic markers such as race and socioeconomic status in trial recruitment.
When the Food and Drug Administration looked at 19 multiple myeloma drug trials conducted between 2006 and 2019, they found that Black, Latino and Native American people were underrepresented in clinical studies compared to the demographics of the general patient population.
Per FDA data presented Sunday at ASH, Black people made up just 4% of multiple myeloma trial participants over that time frame. By contrast, 20% of multiple myeloma patients in the U.S. are Black, according to the International Myeloma Foundation.
“This is a significant underrepresentation of populations that have the disease and really underscores that we really don't have a lot of information about the safety and efficacy of these specific products in patients that will ultimately receive them,” said Nicole Gormley, the FDA's associate director for oncology endpoint development.
A type of blood cancer formed in plasma cells, multiple myeloma fills the bone marrow with malignant clones, crowding out normal cells and damaging bones and other organs.
A wide range of treatments exist for myeloma, but patients often cycle through many different regimens. Newer drugs to recently reach market include antibodies such as Johnson & Johnson’s Tecvayli and Pfizer’s Elrexfio as well as CAR-T therapies like Bristol Myers Squibb and 2Seventy Bio’s Abecma and J&J and Legend Biotech’s Carvykti.
Improvements in the standard of care have quadrupled survival outcomes for multiple myeloma patients, said Saad Usmani, chief of myeloma service at Memorial Sloan Kettering Cancer Center.
Still, patients of color face bias and delay in referrals to clinical trials of myeloma treatments, as do people living in parts of the U.S. where there are no cell therapy centers offering CAR-T, Usmani said.
Federal reforms could help broaden who enrolls in clinical studies. In 2022, Congress passed the Food and Drug Omnibus Reform Act, which requires drug sponsors to submit plans outlining how they plan to diversify trial enrollment for Phase 3 or post-approval studies. This year, the FDA drafted guidance for cases when data from specific populations hasn’t been obtained prior to a drug approval.
Editas’ high bar
CRISPR gene editing company Editas Medicine has positive things to say about the FDA’s approval Friday of two gene therapies for sickle cell disease.
“To see both approvals go smoothly is a good sign for all of us in this field,” said Caren Deardorf, the biotech company’s chief commercial and strategy officer.
But it’s also true that the approvals set a high bar that Editas, which is developing its own genetic therapy for sickle cell, now has to clear. The company came to ASH with updated results from 17 patients treated with its therapy, which is being developed for the blood condition beta thalassemia as well.
Among the 11 with sickle cell, none have had a sickle cell pain crisis since receiving Editas’ therapy, called reni-cel and similar in construction to Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy. Those who have been followed for at least five months post treatment have levels of hemoglobin — the oxygen-carrying protein that’s damaged in sickle cell — in the normal range.
Baisong Mei, the company’s chief medical officer, said the data are encouraging so far, but acknowledges that it’s on Editas to prove its approach can be superior.
Reni-cel differs from Casgevy in two key respects. To create reni-cel, Editas uses CRISPR to edit a different part of stem cell DNA than Casgevy does, a tweak it claims leads to better red blood cell production once those modified cells are reinfused into a patient. The company also uses a different form of CRISPR than Vertex — AsCas12a rather than the standard cas9 enzyme.
Those differences, Editas says, could lead to higher hemoglobin levels and better anemia protection. “There are data that say, actually, every gram per deciliter [of hemoglobin] increase can be helpful,” said Mei.
But it may be hard for the company to prove it’s better than either Casgevy or Bluebird bio’s Lyfgenia, which will be on the market for some time before Editas can even submit for approval.
Editas, which formed around the same time as Vertex partner CRISPR Therapeutics, is well behind because it originally focused on gene editing for eye diseases, a strategy it has since scrapped. The company has also hit clinical delays and endured significant executive turnover.
It plans to enroll 40 patients in its Phase 1/2 sickle cell study, which will assess the proportion of patients who achieve complete resolution of pain crises. Updated data are expected by the middle and end of next year, Editas said Monday.
Sticker shock workarounds
The list of million-dollar medicines got two new additions last week with the sickle cell therapies Casgevy and Lyfgenia, which their makers priced at $2.2 million and $3.1 million, respectively.
While expected, the high cost raised questions about insurance coverage, particularly for the many sickle cell patients in the U.S. who are on Medicaid.
“New potential curative treatments are a tremendous step forward,” said Punam Malik, director of a sickle cell center at Cincinnati Children’s Hospital, during a session at ASH on Monday. “But they have to be accessible.”
Malik, who’s worked with drugmakers before, acknowledged that these therapies require a large investment to develop and bring to market. Still, she argued there are ways to bring the costs down over time, ticking off strategies like improved techniques for purifying the stem cells used to create the drugs or reducing the time patients spend in hospital.
But the best way, Malik said, is to continue research on “in vivo” therapies that don’t require the laborious process of extracting, shipping and reinfusing a patient’s stem cells.
Malik shared the stage with Nicole Verdun, director of the FDA office that reviewed Casgevy and Lyfgenia. While the FDA doesn’t consider costs in its approval decision, Verdun said the industry “needs to partner to make sure that we have equity and access after these therapies are approved.”