Piper: $QURE note 4/13 Last week, unfortunately timed with its secondary offering, QURE was notified by the CHMP that a negative assessment on Glybera is being submitted to the CAT
(Committee for advanced Therapy Medicinal Products) which theoretically places the
drug's EU approval at risk. The CHMP held the same negative view the first time Glybera
was approved, so we are skeptical it will have any impact this time. Even if it does, QURE
has become far less reliant on EU Glybera prospects as the hemophilia B gene therapy
program has advanced to the clinic and it recently signed a transformative partnership
with BMY. We reiterate our $40 PT.
• More of the same? After review of the 6-year follow-up data for Glybera, the CHMP
rapporteur has concluded the drug has not demonstrated robust efficacy and as such
the benefit-risk balance is negative. This is the same conclusion the CHMP had prior
to Glybera approval, which required 4 CAT committee meetings to assess. After an
initial CAT negative vote, the subsequent ones were all positive because it was decided
that the drug's effect on post-prandial chylomicrons was much more relevant for
predicting pancreatitis risk than the drug's overall effect on triglycerides; a perspective
we share.
• What's next: The rapporteur's report will be submitted to the CAT for further
assessment. The analysis is likely to be considered during an April 21-23 meeting;
following this meeting there may be additional correspondence with the company to
clarify any outstanding questions. A final decision may take up to a few months. We'd
be surprised if the 6 year update led to any different decision by the CAT this time
around.
• Worst case: If the CAT reversed its prior views and decided to pull Glybera from the
market, it would have a modest impact on our valuation (<25% impact) since we
assume only modest contribution given a slow launch and royalty partially offset by
payments owed. At this year's Phacilitate conference we had an opportunity to speak
with one of the CHMP members who had been opposed to Glybera's approval- she
was quite clear that this was not because it was a gene therapy product but because
she didn't believe the drug had clear efficacy. She fully expected other gene therapy
products will be approved on the basis of a larger demonstrated effect size. As such,
we do not believe there should be any negative readthroughs even in the unlikely
circumstance that Glybera is withdrawn.
Ook via link vorige post.