Replies to post #176963 on Biotech Values

[guerlain]

QURE - uniQure/Chiesi's hemophilia program is still in late stage pre-clinical development and won't move into clinic until 2H14, while Baxter/Chatham already has their program in clinic, thus it seems a weak argument to assume that uniqure has an advantage over Baxter that it will be ithe first to market...

The key differentiation will come from clinical data (both safety and efficacy), which we have none from both yet, so it's quite premature to assume any advantage at this point. Just typical sell-side spin IMHO.

[mcbio]

QURE - Baxter was a previously partner with Chatham Therapeutics on their Hemophilia treatments and had previously paid $25 million to Chatham related to the development of those treatments. I assume their relationship was a driving factor to the eventual purchase.

Took a closer look at QURE's pipeline (http://www.uniqure.com/pipeline/clinical-pipeline/ ) and they seem to have a lot more going on than just the hemophilia program. What do you make of the opportunity for the lead drug Glybera? It's apparently already approved in the EU under "exceptional circumstances" and it appears they are looking to broaden approval in EU and ultimately the U.S. Lipoprotein lipase deficiency is an orphan disease and unmet need according to the company. Then there are programs for Acute Intermittent Porphyria, Sanfilippo B Syndrome, and also Parkinson's. Seems like they have a lot going on.

[DewDiligence]

BAX encounters immunogenicity in phase-1/2 trial testing gene therapy for hemophilia-B:

http://finance.yahoo.com/news/baxter-provides-progress-gene-therapy-213000523.html

As of the end of 2014, a total of six patients in three dosing cohorts have been treated in the trial with evidence of a dose-related response. No patients have developed FIX inhibitors to date. In the two highest dose cohorts, FIX activity levels around 10 percent or above have been observed in two patients, who also experienced no bleeding events. One of these patients showed elevated levels of liver enzymes indicative of an immune response, which is being treated with oral corticosteroids, per protocol. Immune responses have been reported in previous studies with gene therapy technology. Additional patients are being screened…

The trial in question is listed at https://www.clinicaltrials.gov/ct2/show/NCT01687608 .