Monday, December 15, 2014

Dicerna About to Disclose Status of GalNAc-Conjugated Dicer Substrates

Dicerna is about to hold its annual R&D day tonight and, for the first time, reveal the status of their version of GalNAc-RNAi trigger conjugates.  To wit, when Dicerna licensed LNP technology from Tekmira for their primary hyperoxaluria development candidate, it concurrently announced that future development candidates will be based on GalNAc conjugate technology.

For stock market investors, the big question is whether the data suggest sufficient maturity and competitiveness compared to the more famous GalNAc-siRNAs by Alnylam.  If Dicerna can show tonight robust and prolonged knockdowns in non-human primates, e.g. in the form of an ~80% PCSK9 knockdown after 30 days with less than 10mg/kg, Dicerna could be viewed as a great Alnylam catch-up investment.  This is because such maturity would allow Dicerna, which sports a market cap just 1/30 that of Alnylam, to rapidly expand its liver-directed pipeline.

Scientifically, this should be possible although the requirement for prior Dicer processing of Dicerna’s, but not Alnylam’s RNAi triggers could impose limitations as to the extent of nucleic acid modifications.  It is a high degree of stability and consequently modifications that has been key to Alnylam’s recent technology progress.


On the other hand, an unanticipated upside would be if Dicerna could reveal advantages over Alnylam’s technology that are a direct reflection of it using a slightly longer RNAi trigger. 

Disclosure: long DRNA.

5 comments:

Anonymous said...

Rossi was the founder of DRNA at the time he was on the SAB of Benitec. The question to ask is how much of Benitec know how and technology is incorporated in to what DRNA do?

If substantial, then Dirk is unwittingly giving BLT kudos by talking up DRNA's book.

Voyager may yet have to pay that licence to little ol' Benny if Kay et. al. want to sit at the same table as DRNA, BLUE etc.

Anonymous said...

BLT were due to receive data on their HCV trial over the Xmas break.

On Xmas Eve, they apparently filed notice of something. If the post is to be believed it could be a takeover bid and no short selling is allowed.

It could easily be the HCV data set, FDA authority to continue or heavens forbid, authority to fast track to market or a combination thereof.

Whatever it is, the market response will be interesting.

Anonymous said...

Oh please.... Get your silly facts right. The six weeks was only up on Xmas Day. The data is to be assessed and reported on in due course. IE; at LEAST a week, probably two. Notice was received late on the 23rd. Read announcements and you will find the data and dosing will be announced simultaneously. No wonder Benitec get a ribbing here..... Holders ramping away without due diligence. There is no new notice.

Anonymous said...

Every mention of Benitec is the beginning of amateur hour.

There is not a single thing they can get right in their communications and most of the board have already quit and moved on without saying as much.

But my question is, if the sophisticated North American investors like R.A. Capital are in control of the company then why is it every erroneous document has Yuk-Ching Chung as its author? Regardless of whether or not it has been signed off by the secretary, the CEO or contracted PR company.

Add to that, even the CSIRO have yielded on holding its equity stake. Soon, post takeover I expect, they will relinquish IP prosecution rights as well.

I guess this is why Voyager can operate with so much confidence without taking a licence.

Anonymous said...

Plastic .... err, Rubra, you just can't let BLT go can you? Benitec's press releases have exactly what to do with the science. ddRNAi is the most elegant platform in gene therapy. Hopefully they will silence you as well.

By Dirk Haussecker. All rights reserved.

Disclaimer: This blog is not intended for distribution to or use by any person or entity who is a citizen or resident of, or located in any locality, state, country or other jurisdiction where such distribution, publication, availability or use would be contrary to law or regulation or which would subject the author or any of his collaborators and contributors to any registration or licensing requirement within such jurisdiction. This blog expresses only my opinions, they may be flawed and are for entertainment purposes only. Opinions expressed are a direct result of information which may or may not be accurate, and I do not assume any responsibility for material errors or to provide updates should circumstances change. Opinions expressed in this blog may have been disseminated before to others. This blog should not be taken as investment, legal or tax advice. The investments referred to herein may not be suitable for you. Investments particularly in the field of RNAi Therapeutics and biotechnology carry a high risk of total loss. You, the reader must make your own investment decisions in consultation with your professional advisors in light of your specific circumstances. I reserve the right to buy, sell, or short any security including those that may or may not be discussed on my blog.