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ASH Annual Meeting: Drugmakers Battle for Multiple Myeloma

By Brian Orelli, PhD - Dec 13, 2020 at 11:44AM

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Targeting BCMA is all the rage, but who will come out on top?

In this video from Motley Fool Live, recorded on Dec. 7, Corinne Cardina, bureau chief of healthcare and cannabis, and Fool.com contributor Brian Orelli review data from the American Society of Hematology (ASH) annual meeting on multiple myeloma drugs targeting B-cell maturation antigen (BCMA). Companies are targeting BCMA with both chimeric antigen receptor (CAR) T-cell therapy and bispecific antibodies. Both types of treatment seek to bring T-cells in proximity to the cancer cells for the T-cells to kill them. It's a hot space that investors should keep an eye on as there's no clear winner yet.

Corinne Cardina: There was quite a bit of data related to the BCMA target at the ASH conference. What are some of the highlights?

Brian Orelli: Yes. Maybe we should just start from most advanced to least advanced. bluebird bio ( BLUE -0.60% ) and Bristol-Myers Squibb ( BMY 0.53% ), who got the drug from the acquisition of Celgene -- their most advanced product is ide-cel. Again, that's one of those drugs that starts in "ide" and has "cell" in the second word. It's under FDA review right now. I think we talked about it last week. It had gotten a delay, they're waiting for their manufacturing plant to be inspected. They had a pivotal study in 128 patients who had failed three other drugs and they presented data that showed at least 65% responded, and at least 20% had complete responses.

A complete response then after a few years is considered a cure. An immediate complete response isn't necessarily a cure. You could still fail the drug and your cancer could come back, because you can't necessarily measure the small amount of cancer cells that might be remaining. But definitely, if you get a complete response after a few years, the doctors say you're cured.

Johnson & Johnson ( JNJ 0.26% ) and Legend Biotech ( LEGN 9.76% ), that's cilta-cel, it's in a phase 1b/2 clinical trial in 97 patients. They had a really high response rate of 97% with 67% complete responses. It definitely looks better than ide-cel, but it's hard to compare these trials head-to-head because they're not necessarily enrolling its vaccine patients. Depending on how sick the patients are, that will determine how many patients are likely to have responded. If you're on your sixth or seventh drug and you responded and failed, or not responded at all to their earlier drugs, then obviously, you're less likely to respond than if it's your first or second drug. First line or second line, in terms of the drug-treatments paradigm.

Bluebird and Bristol-Myers Squibb have a second drug. It's still got a code-name, bb21217. It's a second-generation CAR-T; 68% response rate and 29% complete responses. Not that much better than ide-cell. It doesn't appear that it's that much better than ide-cell. We'll have to wait and see as the data matures and they get more data. It doesn't look very promising when the second-generation is that much better.

Then onto the bispecifics, these are pretty early stage but Johnson & Johnson's phase 1 had 22 patients treated at the dose they're planning on taking into phase 2. They had 73% response with 23% complete responses. Then Regeneron's ( REGN 1.61% ) bi-specific, again, phase 1, they had 63% responded at the highest dose level. They had no complete responses, but it looks like it just might be not enough time. I think they were only out for a couple of months. They had at the lower doses -- so they are currently on dose level 6 -- at dose levels 1 through 3, they had 21% complete responses and 18% at doses 4 and 5. I think they'll probably end up getting some complete responses, so maybe somewhat equivalent to Johnson & Johnson, so we're looking at 73% responded for Johnson & Johnson versus 63% for Regeneron. I didn't catch whether the Regeneron is going to go up higher than the 63%.

Cardina: Excellent. Just to make sense of all these different mechanisms, do you think that there is room on the market for CAR-T and bi-specific antibodies to be successful? Is this a winner-take-all market? What do you think about the potential for multiple different mechanisms in the BCMA target area?

Orelli: I think it's likely that there's room for more than one. The CAR-Ts are definitely harder on the patients because they have to have their blood cells taken out and then they go manufacture them for three weeks. Then they put them back in. There can be a lot of development there. A lot of decline in patients there. If CAR Ts are better than the bispecifics, there's still room for the biospecifics to have a decent amount of sales because there will be patients where the doctor is worried about the patient lasting those three weeks and not declining so the CAR-T isn't going to help them. So I think that you'll see the more sick patients get the bispecifics and less sick patients get the CAR-Ts even if CAR-Ts are better than biospecifics in the end.

Cardina: Great, very helpful. Last question on this section. From an investment standpoint, are any of these stocks in the BCMA target market particularly compelling to you?

Orelli: Again, we got Bluebird in here again, Johnson & Johnson, obviously a very large company. I don't know much about Legend. I think they're a China-based company, but I haven't really studied them that much. Then Regeneron, big biotech with a lot of drugs and antibodies, so definitely not buying them specifically for the BCMA space, but it's definitely a solid company.

This article represents the opinion of the writer, who may disagree with the “official” recommendation position of a Motley Fool premium advisory service. We’re motley! Questioning an investing thesis – even one of our own – helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

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Stocks Mentioned

bluebird bio, Inc. Stock Quote
bluebird bio, Inc.
BLUE
$9.95 (-0.60%) $0.06
Johnson & Johnson Stock Quote
Johnson & Johnson
JNJ
$163.36 (0.26%) $0.42
Bristol Myers Squibb Company Stock Quote
Bristol Myers Squibb Company
BMY
$57.13 (0.53%) $0.30
Regeneron Pharmaceuticals, Inc. Stock Quote
Regeneron Pharmaceuticals, Inc.
REGN
$635.09 (1.61%) $10.09
Legend Biotech Corporation Stock Quote
Legend Biotech Corporation
LEGN
$49.39 (9.76%) $4.39

*Average returns of all recommendations since inception. Cost basis and return based on previous market day close.

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