3 Things In Biotech You Should Learn Today: April 20, 2017

by: Zach Hartman


Regeneron's partner granted European patent on CRISPR/Cas9 for amyloidosis.

NovoCure presenting compliance data for Optune at AAN.

CytRx hangs on with a meeting at the FDA.

Welcome to another installment of "3 Things in Biotech You Should Learn Today." In this episode, we're going to consider the news of a few "out there" technologies, namely CRISPR gene editing and tumor-treating fields. Then, we'll come back to something a little closer to conventional.

Without further ado...

Regeneron's partner granted European patent on CRISPR/Cas9 for amyloidosis

Regeneron (NASDAQ:REGN) is quietly stepping into the spotlight on development of the CRISPR/Cas9 gene editing technique, as its partner Intellia Therapeutics (NASDAQ:NTLA) was granted a patent from the European Patent Office for its platform, which is currently being explored in preclinical studies to manage familial amyloidosis, a devastating condition caused by massive overproduction of transthyretin.

Earlier this year, REGN and NTLA published findings showing the ability for this CRISPR platform to reduce the expression of transthyretin in mice livers by 97%, which was sustained for at least four months in these animals.

Looking forward: This is obviously very, very early days for this treatment strategy, but I think we're all rather hungry to hear news of how well the CRISPR platform might work. REGN's continuing development in this area signifies that it is building confidence and continues to work. These are also impressive preclinical findings for amyloidosis, which currently has no cure and only variably effective symptomatic treatment options.

NovoCure presenting at AAN; more and more on Optune

NovoCure (NASDAQ:NVCR) is marketing its Optune technology, which is a wearable device that is designed to provide lethal doses of electric fields to tumors, a so-called "tumor-treating field" technique. This has been shown to deliver similar, if not better, efficacy compared with chemotherapy in glioblastoma, the most common form of primary brain cancer.

NVCR has announced that it will present new findings regarding tumor-treating fields at the American Academy of Neurology meeting in several poster sessions. A few of these are related to technical aspects of Optune, such as how tissue capacitance may predict benefit from the technique.

Another study assessing compliance with Optune will show that patients with longer overall exposure (cutoff at 2,885 hours) have better overall survival (under versus over: 8.8 months versus 17.2 months), representing a significant association between total exposure and benefit.

Looking forward: These findings point to a significant trend for doctors involved in glioblastoma. Obviously, taking your medicine is important for cancer therapy, but being able to point to solid data to explain the benefit helps doctors realign their priorities and teach their patients about compliance. This is especially important, considering that you're asking the patient to wear a backpack and headgear for several hours a day, every day. Putting rubber to the asphalt can give patients extra motivation.

CytRx hangs on with a meeting at the FDA

CytRx (NASDAQ:CYTR) has been involved in the translation of a new chemotherapeutic agent, aldoxorubicin, to the management of soft-tissue sarcomas, a group of tumors arising at multiple sites in the body that present a huge challenge for the medical community.

In a press release, CYTR announced that it had met with the FDA in a discussion of how it may progress through the drug approval process. The FDA assured CYTR that it will be able to use the same approval pathway used by similar chemotherapy agents in the past (such as Abraxane, an albumin-bound form of paclitaxel).

This keeps the company on course to submit an NDA to the FDA for aldoxorubicin later in 2017.

Looking forward: Aldoxorubicin has shown significant promise compared with other chemotherapeutic agents in soft-tissue sarcoma. Considering the high unmet need status of these tumors, it is unsurprising that the FDA is willing to work with the company to collaborate on the best path forward for an encouraging new version of chemo.

However, there is still major risk potential with a small-cap like CYTR, especially one that has had such a bumpy ride. Twice in 2016, it diluted shareholders substantially. Moreover, in July 2016 the company announced some not-so-pleasant news surrounding aldoxorubicin. Results of its pivotal study did not show significant improvement in PFS compared with investigator's choice chemotherapy, though disease control rate eked out a significant improvement. Whether this latest news from the FDA means that the company has since seen an improvement (nearly half of the results were censored as of July 2016 because of a clinical hold) remains to be seen. So there is quite a bit of risk investors take on, and I redouble my plea that you consider all relevant factors before considering an investment in a microcap.


Thank you for checking out another edition of this digest. If you would like to continue receiving updates from the world of biotech, please consider becoming a follower of mine on Seeking Alpha. This will allow you to receive email updates whenever the new edition comes out. And also, feel free to leave a comment in the section below!

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

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.

Editor's Note: This article covers one or more stocks trading at less than $1 per share and/or with less than a $100 million market cap. Please be aware of the risks associated with these stocks.