3 Things In Biotech You Should Learn Today: August 24, 2017

by: Zach Hartman


Astellas moving forward with more and more trials in AML.

Ultragenyx suffers a setback in myopathy.

Cardiome's approach to atrial fluttering not good enough, per the FDA.

Welcome to another edition of "3 Things in Biotech You Should Learn Today," a daily digest dedicated to helping you keep pace with the rapid movements in biotech and pharmaceutical research!

What's in store for us today?

Astellas moving forward with more and more trials in AML

In the world of acute myeloid leukemia (AML), therapies directed against specific molecular subtypes have recently reached a fever pitch in development. One of the more important AML subtypes (as of right now) is FLT3-mutated.

Why? Because we're finally starting to see progress with selective FLT3 kinase inhibitors as therapy. Astellas (OTCPK:ALPMY) is at the front of a pioneering herd of developers with its own entry to this space: gilteritinib, which has already shown encouraging findings for FLT3-mutated AML.

Now, the company has announced the initiation of its fourth Phase 3 trial, MORPHO, which is assessing the benefit of gilteritinib maintenance therapy, following the achievement of a complete response from a hematopoietic stem cell transplant.

Looking forward: This should be a very important study for ALPMY, since Novartis (NVS) beat it to market. Gilteritinib's ability to block the receptor tyrosine kinase AXL may help to reduce the possibility of treatment resistance, as well, and the maintenance setting might be the best area to exercise this potential, assuming the drug is not too toxic. In my mind, the FLT3 mutation story shows significant similarities to the CML story, though I won't get my hopes up with acute leukemia, since these tend to be more aggressive diseases.

Ultragenyx suffers a setback in myopathy

Ultragenyx Pharmaceutical (RARE) is a midcap biotech stock dedicated to the discovery and development of treatments for rare, genetic disorders. It has a variety of agents in the pipeline for a range of disorders.

However, the recent news pertains to the use of aceneuramic acid in patients with GNE myopathy. RARE announced top-line results from the Phase 3 pivotal study for this agent, indicating that it did not meet its primary endpoint of improvement in upper extremity composite score compared with placebo.

RARE intimated that it will terminate this particular developmental program due to these unfavorable results.

Looking forward: Definitely not good news for RARE or for patients with GNE myopathy. On the bright side for the company, however, is the volume of other therapies in the pipeline. So, it seems likely that it will take a near-term thumping from this news, but good news in another area could go a long way toward reversing this setback.

Cardiome's approach to atrial fluttering not good enough, per the FDA

Cardiome Pharma (CRME) hopes to bring a European-approved infusional therapy called vernakalant for atrial fibrillation to the United States. After previous failure to launch in the United States, CRME has embraced a more international focus, seeking approvals in Europe and Canada and launching vernakalant.

So, with several years of accumulated evidence, CRME seemed poised to reapply to the United States regulators with its agent. Unfortunately, communication with the FDA indicated that the company does not have sufficient evidence to seek resubmission of its NDA at this time.

CRME continues to believe in its approach, however, and will keep working on accumulating data internationally.

Looking forward: We did not get much description in the company's press release, but it is indeed interesting that vernakalant has received international approval for the label that CRME was seeking. But the FDA did not deem these findings sufficient? Very unusual, as the standards for Europe and Canada don't typically differ so widely. I would expect that this setback will be manageable in the near term for CRME.

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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.

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