ASCO 2022: Surprising News in Cancer Research

The American Society of Clinical Oncology (ASCO) held its annual meeting this week, and it's a must-show event for anybody pursuing cancer therapies. Our mini-roundtable has been digging into some of the presentations, and we found a few companies announcing nice surprises, among them, Adaptive Biotechnologies (ADPT -2.33%), Legend Biotech (LGND -4.43%), and Arcellx (ACLX 0.08%).

Here's what these biotechs revealed this week.

Testing for minimum residual disease (MRD) in cancer

Taylor Carmichael (Adaptive Biotechnologies): Because the biotech industry is highly secretive, Adaptive Biotechnologies often can't talk about what companies are using its technology. At ASCO, however, the scientists (and investors) want to trust the data they're being shown, so a lot of researchers were noting that they used Adaptive's test for minimum residual disease (MRD) in blood cancers. That offers investors a peek at some of the researchers who are using Adaptive's solution.

Adaptive's test, clonoSEQ, is the first (and only) assay cleared by the Food and Drug Administration (FDA) to check for MRD in blood cancers like leukemia and myeloma. In blood cancers, if a treatment plan is working and the cancer is destroyed, doctors say the cancer is in complete remission (CR). But many of these patients often have sub-microscopic cancer cells that can't be detected with a microscope. Those cells could multiply, allowing the cancer to return in the future.

The clonoSEQ genetic test checks for MRD, giving the doctor a more complete picture of health. In fact, MRD status may ultimately predict clinical relapse. As one doctor put it, "The disconnection between CR and long-term efficacy suggests that persistent disease remains undetected, and measuring deeper responses is necessary to predict and improve long-term outcomes."

Nine abstracts at ASCO cited Adaptive's clonoSEQ test for blood cancers. The list includes Legend Biotech, Arcellx, Johnson & Johnson, Gilead, Roche, as well as universities and hospitals. (Of course, Adaptive was a participant in all of these trials.)

Poster abstract numbers from company press releases. Sponsors of clinical trials from author's research.

It's notable to see so many CAR-T trials using Adaptive's clonoSEQ assay. It is likely that in the future, more and more oncologists will check for MRD before announcing a blood cancer is in complete remission.  

CAR-T has immensely improved 

Patrick Bafuma (Arcellx and Legend Biotech): It was just five years ago that Gilead's Yescarta became the first CAR-T cell-based gene therapy approved for the treatment of certain blood cancers. Since then, the field has only gotten better. And two fledgling biotechs showcased eye-catching data for the treatment of multiple myeloma at ASCO's annual meeting. 

The global market for multiple myeloma treatments is estimated to be $18 billion annually, with an estimated $10 billion opportunity for CAR-T treatments. While early CAR-T therapies were plagued by neurotoxic side effects such as somnolence, confusion, and even seizures, new treatments have brought better outcomes. For example, Bristol-Myers Squibbs' and 2seventy bio's CAR-T treatment Abecma, has a less than 10% rate of severe neurotoxic events. Approved as a treatment for relapsed or refractory multiple myeloma after patients have tried four or more prior lines of therapy, Abecma also has an overall response rate of 73% (any positive response to treatment) with a 31% complete response rate (the disappearance of all signs of cancer in the body). And at ASCO, the candidates of two up-and-coming biotechs, Arcellx and Legend Biotech, posted even better results.

In a small phase 1 clinical trial, small-cap biotech Arcellx boasted a 100% overall response rate and a 70% complete response rate. Even more impressive, these results were in a difficult-to-treat population of patients who had already received three or more prior therapies. Likewise, Legend biotech showcased 28-month follow-up data for Carvykti, which like Abecma, is also approved as a fifth-line treatment. After more than two years, overall response rates remained at 98%, according to the data presented at ASCO for the $7 billion biotech's recently approved CAR-T. Fewer than 10% of patients had severe neurotoxic effects from either Legend's or Arcellx's treatments, which puts their products on par with Abecma in terms of side effects, but with better response rates.

With attractive safety profiles and overall response rates north of 90%, it appears that CAR-T solutions may present growing opportunities in the multiple myeloma market. If trials continue to produce positive results, these therapies are likely to be authorized for use as earlier lines of treatment.

While both companies received applause at ASCO, they offer different risk profiles for biotech investors seeking growth. Legend Biotech already has Carvykti on the market, but its nearly $7 billion market cap, is around eight times that of Arcellx. While the lead Legend enjoys may be difficult to overcome, it has to evenly split profits with Johnson & Johnson, whereas Arcellx remains unpartnered for now. And with its market cap in the neighborhood of $900 million, it could make an attractive acquisition target for a big pharma player. The good news is that investors don't necessarily need to choose between the two -- they could buy both as part of a basket approach.