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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.
AbbVie continues its slow coup of CLL treatment
Company: AbbVie (ABBV)
Disease: Chronic lymphocytic leukemia (CLL)
News: ABBV announced that the FDA has granted approval to their Bcl-2 inhibitor venetoclax in combination with rituximab for the treatment of CLL in patients who have relapsed on one prior line of therapy. This approval was based on the much publicized and ballyhooed MURANO study results, which showed that venetoclax-rituximab is superior to bendamustine-rituximab, a second-line treatment option that's been in standard use for years now.
ABBV's CSO provided a nice comment on the approval:
VENCLEXTA now gives indicated patients a new opportunity to significantly reduce the risk of their disease progressing, compared to a current standard of care. This combination provides previously treated CLL or SLL patients with a chemotherapy-free, fixed duration treatment allowing patients the ability to stop treatment after approximately two years
Looking forward: Chemotherapy-free...that's a big selling point for this regimen, aside from the obvious improvement in outcomes for patients from the MURANO study. This combo has had success spelled out for it since mid-2017 when we found out it was positive. And I call this a coup because it is becoming clear that venetoclax is a pretty unstoppable drug in CLL therapy. It's still going to take time to realize just how far it can go, but so far this drug has crushed every challenge thrown in its way. Of course, CLL has been a fertile land for new innovations in treatment, so it's not as pressing a need as some other forms of cancer. But it's still very refreshing to see medicine steamrolling a tumor. Obviously, it's also great news for ABBV, who now stand to take control of the entire game in the CLL standard of care.
Alnylam looks forward to a busy close to 2018
Company: Alnylam Pharmaceuticals (ALNY)
Disease: Primary hyperoxaluria type 1 (PH1)
News: ALNY presented updated findings from their phase 1/2 study investigating the interfering RNA drug lumasiran in patients with PH1. This gene therapy targets mutant glycolate oxidase to correct the excessive production of calcium oxalate, which increases the risk for developing severe kidney stones. The updated report showed a mean maximal reduction in oxalate levels of 64%, and all patients in the study reported reductions to the point where they are no longer at high risk for developing end-stage renal disease. Nine of the patients who were followed for 85 days continued to experience a 63% mean reduction in urinary oxalate, highlighting the durability of response to this therapy.
Looking forward: Very compelling results coming from ALNY again, continuing the successful venture into interfering RNA tech. I'm still amazed that this has made its way to clinic, since it was a tool of choice in my basic research experience, but one many of us couldn't really fathom ever making a genuine impact on medicine. Needless to say, I'm happy to have been ignorant, and it looks like ALNY is on the precipice of another winner in the vein of patisiran.
Roche looks to shore up its hemophilia game in the wake of gene therapy competition
Disease: Hemophilia A
News: RHHBY announced that the FDA has accepted their supplemental application for approval of emicizumab with priority review designation. This puts the action date from the FDA at October 4, 2018 for a decision. The application seeks to expand the label for emicizumab beyond patients who have developed inhibitory antibodies to Factor VIII treatment. Based on findings from the phase 3 HAVEN study, RHHBY believes this drug can be an effective treatment option for patients who have not yet developed inhibitors, as well.
Looking forward: We're in the midst of a big shakeup in the hemophilia world, with companies like Spark Therapeutics (ONCE) making waves in gene therapy that could effectively cure the disease. News like this just a few years ago would have been a huge deal for hemophilia treatment, but now it leaves me wondering just how much impact this drug can have. Still, it is very likely that there will be plenty of patients who do not respond to gene therapy. Moreover, gene therapy doesn't necessarily mean anything for patients who end up developing inhibitory antibodies. So there can still be some legs for a new best-in-class hemophilia intervention.
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