3 Things In Biotech You Should Learn Today: May 3, 2017

by: Zach Hartman


Mixed findings for Neurotrope's phase 2 Alzheimer's drug.

bluebird bio teams up with GSK to move the dial on gene therapy.

Corvus and Roche in the driver's seat for immunotherapy combination.

Welcome to another edition of "3 Things in Biotech You Should Learn Today," a daily digest covering the world of biotechnology and pharma news. The goal of this publication is to help you keep pace with the whirlwind of new results and developments in this industry.

First, an announcement: due to a bit of a timing snafu, you may have missed the publication of yesterday's edition, which covered several high-profile events like the approval of durvalumab. So go ahead and check that out if you didn't get a chance to see it!

Without further ado...

Mixed findings for Neurotrope's phase 2 Alzheimer's drug

Neurotrope, Inc. (NASDAQ:NTRP) is a microcap pharma company that is in the business of drug development for Alzheimer's disease, which remains one of the major significant unmet neurologic disorders today. Its lead agent, bryostatin, is a modulator of protein kinase C, an important regulator of memory function.

As such, NTRP hoped that an activator of this molecule could help stave off or rescue memory loss due to neurodegeneration from Alzheimer's disease. And the company was engaged in the phase 2 202 study, which assessed bryostatin against placebo in terms of cognition defects.

Top-line results from the 202 study showed an apparent improvement over placebo in the degradation of cognition. However, this result failed to meet statistical significance, and the company did not report data for its 40 microgram dosing cohort, suggesting no clear dose response.

Looking forward: Studies of neurodegeneration can be difficult to interpret since they are subject to certain biases because of the testing involved in assessing cognitive decline. The fact that bryostatin appeared to improve cognition (somewhat) while patients in the placebo arm appeared to decline in cognition (somewhat) is a promising early sign to come out of this relatively short trial. It remains to be seen what kind of long-term effects bryostatin could have on cognition. The failure to achieve statistical significance is not an encouraging sign, however. So this presents a high-risk situation for shareholders and would-be investors.

bluebird bio teams up with GSK and NVS to move the dial on gene therapy

bluebird bio (NASDAQ:BLUE) is a biotech on the forefront of gene-based therapies for cancer and rare genetic disorders. With its hands in treatments for thalassemias, cancer, and other diseases, it stands to make a huge splash in the world, should its early promise be realized in clinical trials.

Recently, BLUE announced a license agreement with GlaxoSmithKline (NYSE:GSK) to develop its lentiviral vector-based treatment for Wiskott-Aldrich syndrome and cerebral adrenoleukodystrophy, two rare but serious genetic disorders. It has also licensed its lentiviral platform for modifying T cells to Novartis (NYSE:NVS) which is currently using the tech to push its late-stage development of CTL019, the CD19-directed chimeric antigen T cells, in acute lymphoblastic leukemia.

Looking forward: BLUE is a company with significant cash reserves for a non-marketing biotech. These agreements put into place further acquisition of resources, potentially allowing it to catch onto the tail of an emerging revolution in cancer immunotherapy, which I have detailed in a previous article. The terms of these license agreements have not yet been divulged, but it stands to reason that BLUE was able to capture significant value for its tech. The company disclosed that it would receive an upfront payment, milestones, and royalties.

Corvus and Roche in the driver's seat for immunotherapy combination

Corvus (NASDAQ:CRVS) is a small, clinical-stage biotech developing agents for use in oncology. Its lead candidate, CPI-444, blocks the adenosine 2A receptor, which normally helps tumors block the activation of the immune system nearby. In a recent presentation of early-phase clinical trial data, combination of CPI-444 and Roche's (OTCQX:RHHBY) atezolizumab appeared to provide some measure of disease control in a range of solid tumor types.

The market did not react to this news favorably, however, as the valuation plummeted 50% on that news.

Despite that, CRVS has announced that it is expanding its relationship with Roche, with a phase 1b/2 trial assessing CPI-444 plus atezolizumab in patients with relapsed/refractory non-small cell lung cancer.

Looking forward: I'm not familiar with the expectations of CRVS's phase 1 results to quite grasp why they fell so much during the trading session on reasonably favorable data. My skepticism of the negative mindset here is supported by Roche's willingness to continue working with CRVS when there are many, many candidates for combination immunotherapy it could be exploring. It will be some time before we see the fruits of this collaboration, but it is a sign that bodes well for an injured small-cap pharma company that needs to continue funding operations on the back of promise and not products (at least not yet).


Thank you for tuning into another edition of the digest. Once again, if you happened to miss out on yesterday's edition, I encourage you to check it out. Furthermore, if you enjoy these types of articles, please consider becoming a follower of mine on Seeking Alpha. This will allow you to receive real-time updates when my articles are released.

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.

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