Welcome to another edition of "3 Things In Biotech You Should Learn Today," a daily digest dedicated to helping you parse through all the news emerging out of the world of biotechnology and pharmaceuticals.
Let's find out what's in store for today!
Merck (MRK) made huge waves with its approval for pembrolizumab in any tumor harboring deficiencies in DNA repair, resulting in microsatellite instability. That approval was built largely on the back of work done in colorectal cancer. And if you recall, Bristol-Myers Squibb (BMY) was actually one of the first to bat in the area of immune checkpoint inhibition for colorectal cancer.
If you want to go on a trip down memory lane, here's the article I wrote on that matter last June. So much has changed since then!
So it stands to reason that BMY has not stood still here. And recently we got word that the FDA has granted accelerated approval to nivolumab in relapsed colorectal cancer harboring microsatellite instability-high or mismatch repair deficient metastatic colorectal cancer.
Looking forward: BMY needs all the clawback it can get in 2017 to catch the staggering progress of MRK, and this helps in a pretty big way. Colorectal cancer continues to represent a major unmet need for patients, and by being not too far behind MRK in this space, BMY has a reasonable position to claim some of the market. Time will tell if it goes for and receives a similarly wide approval like MRK got for any tumors harboring these biomarkers.
The realm of acute myeloid leukemia (AML) research is rather grim, overall, as the disease remains a substantial unmet need for patients, especially those who relapse after or are not appropriate for allogeneic stem cell transplant. A lot of progress has been made in recent years with respect to delineating molecular subgroups of AML to help identify targeted therapies that can help patients.
The FLT3 inhibitors are an emerging illustration of the fruits of that labor, as now patients have access to a growing list of targeted therapies.
Another mutation that is important in AML is found on IDH2, which can confer either favorable or worse prognosis, depending on the location. But the big news out recently is that we now know this mutation is druggable. And Celgene Corporation (CELG) is the first beneficiary of this knowledge. It has announced that the FDA granted approval for an IDH inhibitor: enasidenib, branded Idhifa.
The approval is specifically for adult patients with relapsed/refractory AML and IDH2 mutation, as detected by the newly approved companion diagnostic test manufactured by Abbott Laboratories (ABT).
Looking forward: Drug approvals in the oncology space don't usually catch me off guard since it's my primary interest. But this one did! It's most likely due to the rather slow progress we've seen in AML research to date; however, over the last year or so, we are seeing some serious momentum building in this space. Hopefully, there will be an ibrutinib-level breakthrough coming for this disease in the next few years. And certainly this is big news for Celgene, as this represents the first approval in AML for the company and its strategic partner, Agios (AGIO).
Ibrutinib was a huge breakthrough for the management of various forms of non-Hodgkin lymphoma, and a lot of the jockeying in that space has used the agent as a reference point in terms of efficacy and market share.
So it might surprise you to find that AbbVie (ABBV) and Janssen (JNJ) were working to characterize the benefit of ibrutinib in graft-versus-host-disease (GVHD), which is a complication of hematopoietic stem cell transplantation.
GVHD is a very common, difficult-to-manage outcome of this potentially curative therapeutic approach to leukemia. In fact, it can be life threatening, and the treatment options have historically been suboptimal. Corticosteroids can help control GVHD, but once those fail? There's basically been very little doctors could use to help patients at that point.
But recently ABBV and JNJ announced that the FDA has approved ibrutinib and it has received approval for "chronic" GVHD that has grown unresponsive to at least one other treatment option. In the pivotal study for this approval, 67% of patients had resolution of their symptoms, and for many that included involvement of two or more organs.
Looking forward: Based on my recent Bellicum Pharmaceuticals (BLCM) article, you can rightly surmise that I am excited about the prospect of potentially being able to eliminate GVHD as a significant risk factor from the allo-SCT playbook. However, until that technology is mature (assuming it ever will be), we need new treatment options now. Ibrutinib represents one of the first major breakthroughs in quite some time, and patients should be able to benefit significantly from this approval.
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