3 Things In Biotech You Should Learn Today: November 2, 2017
Summary
- Agios Pharmaceuticals looks toward first IDH1 approval in AML treatment.
- Juno looks not to be forgotten in the CAR-T cell race.
- bluebird also headlines in non-malignant heme disorders at ASH.
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.
Agios Pharmaceuticals looks toward first IDH1 approval in AML treatment
Company: Agios Pharmaceuticals (AGIO)
Therapy: Ivosidenib, an IDH1 inhibitor
Disease: Acute myeloid leukemia (AML)
News: AGIO will present new findings from a clinical trial investigating the IDH1 inhibitor ivosidenib in IDH-mutant AML at ASH 2017 in Atlanta in December. The most advanced of these clinical studies will be presented in abstract 639, which covers a phase 1b/2 study of IDH inhibitors (ivosidenib or the recently approved enasidenib) in combination with azacitidine. AGIO reports favorable rates of blast cell control in a patient population whose average age is 81 years old.
Looking forward: These early stage studies might give you pause, but it's important to remember that enasidenib came out of nowhere and got approval based on promising early-stage clinical data. Two other abstracts highlight the favorable tolerability of ivosidenib monotherapy and in combination with induction chemotherapy for AML. So it looks like there is a strong chance for AGIO to build a small empire of IDH inhibitors on the backs of enasidenib and ivosidenib.
Juno looks not to be forgotten in the CAR-T cell race
Company: Juno Therapeutics (JUNO)
Therapy: JCAR017, a CD19-directed CAR-T cell therapy
Disease: Diffuse large B-cell lymphoma (DLBCL)
News: JUNO announced that it would also highlight new efficacy data at ASH 2017. The TRANSCEND study is featured most heavily, with primary data presented in abstract 581. In the 68 evaluable patients, 75% achieved a response and 56% of the patients achieved a complete response as their best overall response. In patients strictly meeting the inclusion criteria for TRANSCEND, the median overall survival had not been reached at the time of analysis.
Looking forward: In the flurry of activity surrounding CAR-T cell therapy in the latter half of 2017, it's easy to lose JUNO in the shuffle. With this, it's making an aggressive data-driven move to prove that it is a viable player in the space, as well. And the complete response rates appear to compare well to those we've seen in patients undergoing KITE's CAR-T cell approach. So it looks like DLBCL could very well be the first battleground in the marketplace among a number of competitors, after all this hullabaloo surrounding acute lymphoblastic leukemia, which Novartis (NVS) currently owns in toto as far as CAR-T cell therapy goes.
bluebird also headlines in non-malignant heme disorders at ASH
Company: bluebird bio (BLUE)
Therapy: LentiGlobin
Disease: Sickle-cell anemia and beta-thalassemia
News: BLUE announced the acceptance of 11 abstracts at ASH 2017 focusing on the clinical data supporting LentiGlobin in genetic heme disorders. This includes updated results in patients with severe sickle cell disease, including a small group of patients enrolled after a protocol adjustment. In this group, vector copy number appeared higher than the first group of patients, and patients in group A experienced favorable rates of veno-occlusive events. In abstract 360, three-year follow-up of patients with beta-thalassemia receiving LentiGlobin showed continual control of transfusion dependence, with some patients not needing a blood transfusion for over a year.
Looking forward: These findings paint a highly encouraging, though perhaps numbingly slow, development in these congenital diseases. Current standards of care for sickle cell disease and beta-thalassemia are poor, and BLUE looks to be capitalizing effectively on a fresh wave of effective gene therapies, finally bringing hype to life. Could this be a good early signal of a strong 2018 for the company?
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