3 Things, November 1, 2017: Geron On Fast Track, AstraZeneca's New Approval, Novartis Tackles DLBCL

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Includes: AZN, GERN, GILD, NVS, PCYC
by: Zach Hartman
Summary

Geron Corp. snags a fast-track designation in MDS.

AstraZeneca gets a first approval for its Btk inhibitor.

Novartis looks to score its second approval for its CAR-T cell therapy.

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.

Geron Corp. snags a fast-track designation in MDS

Company: Geron Corp. (NASDAQ:GERN)

Therapy: Imetelstat, an inhibitor of telomerase

Disease: Myelodysplastic syndromes

News: GERN announced that its flagship agent, imetelstat, has received fast-track designation from the FDA in low-risk myelodysplastic syndrome. This entitles GERN to several potential perks that could speed up development of imetelstat in this context, including priority review and possibly accelerated approval.

Looking forward: Many shareholders are surprised that myelofibrosis is not getting a lot of play here, but it's important to remember that MDS is a pretty big area of unmet need, including the low-risk setting. People who can't get their need for blood transfusions under control are at risk for transformation to full-blown leukemia, and many patients do not achieve this control with standard agents (explained in more detail in my article about the potential competition GERN stands to face in MDS). If this is a sign that the IMerge study is going well, I'm sure investors will be delighted to hear it, as GERN has been quiet for a long time.

AstraZeneca gets a first approval for its Btk inhibitor

Company: AstraZeneca (NYSE:AZN)

Therapy: Acalabrutinib, a Btk inhibitor

Disease: Mantle cell lymphoma (MCL)

News: AZN announced that the FDA has granted accelerated approval to acalabrutinib for patients with MCL who have received at least one other therapy. As with many accelerated approvals in oncology, this is based on response rates (80% overall response observed in the ACE-LY-004 trial).

Looking forward: A first approval for the world's second Btk inhibitor. This is definitely a win for AZN, as this places it as an option that is one of the best current classes of therapy for MCL. Ibrutinib has been a major blockbuster for Pharmacyclics (NASDAQ:PCYC), so a break into the leukemia space for AZN represents a major win, and it finally brings full circle a thesis I had about this agent last year, although I assumed at the time that CLL would be a better fit as a first approval for acalabrutinib, based on clinical data at the time.

Novartis looks to score its second approval for its CAR-T cell therapy

Company: Novartis (NYSE:NVS)

Therapy: Tisagenlecleucel and anti-CD19 CAR-T cell therapy

Disease: Diffuse large B-cell lymphoma (DLBCL)

News: NVS has submitted a supplemental new drug application for tisagenlecleucel, which is currently approved for treatment of patients with relapsed ALL, for approval in patients with relapsed/refractory DLBCL who are ineligible for stem cell transplant. This submission is based on findings from the JULIET study, which will be presented at this year's ASH meeting.

Looking forward: Hot on the coattails of its own approval, as well as the approval in DLBCL that Kite Pharma/Gilead (NASDAQ:GILD) just received, here's NVS again. I have to admit I am a bit surprised with how rapidly the company has translated this CAR-T cell platform into other B-cell-related diseases. I had this rosy notion that the big players in the CAR-T cell space would be happy in their own corners for a moment, but that isn't business! So now we can expect some serious competition between NVS and GILD in the relatively near future, assuming tisagenlecleucel gets approval.

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Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.