Show me the non-human
primate data. Robust evidence of
gene knockdown in monkeys is a key requirement to gain confidence in a company’s
liposomal delivery claims. Dicerna has
never done that and yet been able to pull off an IPO and make claims about
clinical development timelines without actually being in possession of a realistic
delivery technology.
In retrospect, it is not surprising that they eventually had
to come hat in hand to the liposomal delivery powerhouse, aka Tekmira- after
claiming that its liposomal delivery technology (EnCore) was superior to
Tekmira’s… After all, Dicerna is under
pressure to get its first proper development candidate in the clinic for Primary
Hyperoxaluria Type I (PH1), especially after Alnylam had publicly announcedtheir intention to go after PH1, too.
According to the agreement announced today, Dicerna will use
Tekmira’s 3rd generation LNP technology and manufacturing to get
DCR-PH1 into the clinic sometime in 2015.
In return, Tekmira will get $2.5M upfront, $22M in potential development
milestones and single-digit royalties- in other words, nothing to write home
about other than the satisfaction that they were proven right scientifically.
You may interject that Dicerna already has a clinical program
using EnCore technology, DCR-MYC for cancer. Unfortunately, there are many ways to curing
cancer in mice and I am yet to be convinced that they have been primarily the
result of RNAi mechanism of action.
In summary, a moral victory for Tekmira, a deal that made
sense for Dicerna and which could and should have happened a long time
ago. In a final twist of irony, Dicerna has
given Tekmira a back-handed compliment in issuing another press release today where
it announces that future development programs will be based on Dicerna’s
conjugate delivery technology.
Now that makes
actually sense for a Dicer-substrate-based company. Expect Alnylam to increase its saber-rattling vis-a-vis Dicerna.
.
Disclosure: Long
Dicerna, no position in Tekmira
4 comments:
Dirk
Could you share your investment thesis here
I'm looking as much at oligo Rx technical fundamentals as what I think the market will do going forward.
Thanks. You suspected they were having delivery problems. What sets them apart from ALNY going forward?
Also, long run do you see any non-exon skipping approaches to DMD?
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