3 Things In Biotech, May 2: Big CARs, Big Kar Lands, Amgen Advances

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Welcome to another edition of “3 Things In Biotech You Should Learn Today,” a daily digest dedicated to helping you keep pace with the fast-moving world of pharmaceutical and biotechnology research.

Novartis gets its second CAR-T cell therapy approval

Company: Novartis (NVS)

Therapy: Tisagenlecleucel

Disease: Diffuse large B cell lymphoma (DLBCL)

News: NVS announced that the FDA has granted approval to its CD19 CAR-T cell therapy tisagenlecleucel for relapsed/refractory DLBCL. This adds the second approval for a technique that was approved for relapsed/refractory ALL last year. This approval was based on the pivotal JULIET study, which showed favorable response rates with long durations of response.

Looking forward: And the streak continues here in the states. Now this puts it in direct competition with Gilead (NASDAQ:GILD) for the first time, as it previously received approval in a similar setting. At this point, there doesn’t seem to be any indication that the techniques are much different in terms of efficacy or safety. Perhaps projected price? But Gilead’s option is estimated at about $100,000 cheaper, so I’m very curious to see how it intends to compete.

Very important news for NVS, and now it kicks off the first brush war in CAR-T cell therapy.

Karyopharm is the talk of the town with its myeloma news

Company: Karyopharm (KPTI)

Therapy: Selinexor

Disease: Relapsed multiple myeloma

News: KPTI announced positive top-line data from its phase 2b STORM study, which is assessing its nuclear exportin inhibitor selinexor in heavily pretreated multiple myeloma. Selinexor has achieved an overall response rate of 25.4%, with median duration of response of 4.4 months. The therapy remained well tolerated, with no surprising toxicities observed. KPTI guided that it intends to submit a new drug application based on these findings.

Looking forward: You might feel that a 25.4% response rate is a little underwhelming, particularly in multiple myeloma, where there are now so many effective treatment options. But it’s important to remember that these patients are heavily pretreated, which at this time is typically taken to mean anywhere from 3 to 6 prior lines of therapy. And this is a pretty dire setting of therapy, so a new option with a remarkable mechanism of action. I think it’s the beginning of big things for KPTI.

You should definitely have this company on your watchlist if it isn’t already.

Amgen also brings out good myeloma news

Company: Amgen (AMGN)

Therapy: Carfilzomib

Disease: Multiple myeloma

News: AMGN announced that the CHMP has granted a positive opinion on the label extension for carfilzomib to include the final overall survival results from ASPIRE, which demonstrated improved survival when adding the agent to lenalidomide-dexamethasone. This provides further validation among the national regulators with respect to overall survival.

Looking forward: This is an important extension of what clinicians and pharmacists will see in Europe. And this is a congested treatment space where several treatment options are available. AMGN needs to keep on the attack to prove that carfilzomib is the best treatment that can be given.

Iterative event, but good news for AMGN.

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