3 Things In Biotech You Should Learn Today: August 9, 2017

by: Zach Hartman

AstraZeneca unveils promising data in Parkinson's disease.

Bayer scores broader approval in Europe for its GI cancer drug.

Kite moves to expand trials to Europe in support of its marketing application.

Welcome to another edition of "3 Things in Biotech You Should Learn Today," a daily digest dedicated to helping you keep up with the recent news in biotech and pharmaceutical sciences!

So what's in store for us today?

AstraZeneca unveils promising data in Parkinson's disease

Parkinson's disease continues to be a major struggle for modern healthcare, since the best hope we have is symptomatic relief using drugs targeting neurologic pathways. Unfortunately, many of these treatments eventually stop working for patients, and they find themselves in need of other therapeutic options.

One such option may come from the world of diabetes. AstraZeneca (AZN) has had the GLP-1 agonist exenatide marketed for Type II diabetes since 2012. And in this indication they've been able to grow the sales of this agent into the hundreds of millions of dollars.

Recently, AZN published a paper in The Lancet detailing the experimental use of exenatide in patients with Parkinson's disease. This phase 2, placebo-controlled study was designed to assess the loss of motor function after a total of 60 weeks.

Patients in the active therapy group gained 1 point in the MDS-UPDRS Motor Subscale, compared with a loss of 2.1 points in the placebo group. This was determined to be a statistically significant improvement in outcomes for patients, with relatively minimal risk of serious adverse events.

These results suggest that patients can achieve some measure of disease control that persists for at least 12 weeks following cessation of therapy.

Looking forward: Certainly, any novel treatment that can reduce the impact of symptoms for patients is a welcome addition. These are still relatively early days for exenatide in this setting, as the effect will need to be validated in a larger study. But it bodes well for AZN’s new entry into the space.

Bayer scores broader approval in Europe for its GI cancer drug

In the space of gastrointestinal cancers, Bayer (OTCPK:BAYZF, OTCPK:BAYRY) has had in its pocket the multitargeted kinase inhibitor regorafenib (branded Stivarga) for several years, having first been approved in metastatic colorectal cancer. Back in April, the FDA approved this agent for liver cancer, as well, in patients who have progressed on first-line sorafenib.

Now, we can move that needle just a bit further, as BAYZF announced that they've received supplemental approval for regorafenib in Europe. Thus begins a worldwide focus for the company in liver cancer.

Importantly, regorafenib was the first targeted therapy to show benefit for patients with liver cancer following progression on sorafenib, so this has been a tough area, and more treatment options are welcome.

Looking forward: This is certainly an important notch in the belt for a drug that's been in development for quite a long time. As BAYZF moves to get approvals in Asia, this agent's importance may increase, since the phase 3 RESORCE trial appeared to identify Asian patients as receiving somewhat outsized benefit from regorafenib, presumably due to the more common etiologies of liver cancer in those countries compared with the West.

Kite moves to expand trials to Europe in support of its marketing application

A few days ago, we talked about how Kite Pharma (KITE) was able to get the jump on Novartis (NVS) by becoming the first company to file a marketing authorization application in Europe for CAR-T cell therapy. Though NVS and KITE are not looking for approval in the same space, it's at least a symbolic victory for the smaller pharma company.

Now, KITE have announced that the safety expansion cohort of ZUMA-1 is now in progress in Europe. This study is enrolling adult patients with relapsed/refractory diffuse large B cell lymphoma (DLBCL) to undergo anti-CD19 CAR-T cell therapy. Other, less common, forms of B cell lymphoma are also included.

Looking forward: This study should provide the EMA a clue as to whether KITE can land this technique on the multinational scale, so the expansion study could be more important than usual for drug development. CAR-T cell is complex, to say the least, as it requires extensive patient time and processing of the cells. If KITE can pull off similar results in Europe as what they've seen in the US, I don't see how they fail to get approval.


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