Liquid Biopsy · @Liquid_Biopsy
1st Dec 2015 from TwitLonger
chardan: $QURE Buy on the market's -17% reaction to old or expected
news
We reiterate our Buy rating on uniQure and advocate using
yesterday's share price weakness to buy into recent positives on
AMT-110 (Sanfilippo B) that were inadequately priced during the
recent biotech market turmoil. Per our 27 August research
("UniQure 2Q results provide visibility on key September to
December catalysts"), we removed US Glybera (lipoprotein lipase
deficiency) from our model after uniQure announced the FDA would
require more than one additional study to support a BLA filing for
Glybera in the US. Our decision was based on Glybera's overall
commercial profile, which we did not believe justified US investment.
The impact on our price target at the time was 7.5%, and with far
more important assets like AMT-060 (hemophilia B) and AMT-110
(Sanfilippo B) making progress, and having upcoming catalysts, we
affirmed our Buy rating on uniQure. Yesterday, uniQure reported
3Q15 results and provided an update on key programs, including the
disclosure that, as we expected, uniQure would not pursue regulatory
approval for Glybera in the US, confirming a positive for us, that
uniQure is pursuing the value-enhancing decision (i.e. not to pursue
US Glybera). We further note that a 1- to 4-week delay on the release
of preliminary AMT-060 hemophilia B data is immaterial to models.
The following are what we consider key incremental items from the
3Q2015 results release and conference call:
• AMT-060 (hemophilia B): In early January 2016 (previously due
by end-2015), uniQure will release preliminary, top-line data for 2
patients who have completed at least 12 weeks of treatment, with
full results from the first cohort of 5 patients at a scientific
conference in 1H2016.
•
- Our take: A delay of a few weeks is immaterial to our
thesis, or any reasonably-modeled hemophilia B view, and
we continue to expect a signal of factor IX expression at 12
weeks in each of the 2 patients shown in early 2016.
- Another take: We believe Dimension Therapeutics (Neutral,
PT$13) traded up 9% yesterday, as some in the market
wondered why uniQure is presenting data for only 2 patients
of 5 in the interim data. Our catalyst sheets for months have
consistently showed uniQure's expectations of "2-3 patients"
of data out of the 10 in the trial, and we note this approach
enhances the ability of QURE to present the data at a major
scientific conference. As such, we continue to prefer uniQure
over Dimension Therapeutics.
• AMT-110 (Sanfilippo B): uniQure is still working to complete
in-licensing of the recent positive phase I/II data from Institut
Pasteur and will follow the patients through the end of the
30-month study, which is expected around year-end 2016.
Our take: We continue to believe long-term durability of AMT-110's benefits in a deadly disease like Sanfilippo
B would represent a landmark result in gene therapy and in lysosomal storage diseases, as patients who
should have been heading towards a vegetative state by the age of 7 or so (and death in their teens) showed
age-dependent and almost normal brain development without atrophy at sequential MRIs.
• Glybera US: As above, the company has decided not to pursue U.S. regulatory approval of Glybera in lipoprotein
lipase deficiency and as a consequence reported a one-time, non-cash impairment of €11.6 million, which drove an
EPS of -€1.08, versus our expectation of -€0.60.
•
- Our take: Prior to this news, we modeled €0 million in 2025E revenues for Glybera US, meaning no impact to
our sales model and no impact to our valuation.