jurpsy schreef op 28 juli 2016 10:13:
PHGUF:
Be Afraid Cinryze, Be Very Afraid;Prophylaxis Study Nails ItPharming put up a major win, in our belief, with statistically significant data
from the Phase II study using Ruconest in the HAE prophylaxis setting. The
company will now look to the FDA/EMA for guidance on the path to label
expansion beyond the acute setting. We maintain our Buy rating and $1.84
target.
EventPharming announced the Ruconest Phase II prophylaxis study met its primary
endpoint in significantly reducing HAE attacks. This study was a randomized,
double-blind, placebo-controlled study, which enrolled 32 patients with HAE
with at least four attacks per month. The primary endpoint was met in
statistically significant fashion (p<0.0001) and was a >50% reduction in the
number of attacks compared to placebo over 28 days (details below). Twice
weekly and once weekly dosing were compared to placebo. Recall that
Ruconest is partnered 50/50 with U.S. partner Valeant (VRX-NC).
ImpactThis is a major win for Pharming and Ruconest, in our belief. The clinical
data are strong and we believe that the data point to potential superiority
over Shire's (SHPG-NC) on two fronts: 1) the patient population in the
Ruconest study appeared more severe with at least four attacks per month at
baseline compared with Cinryze's two (we note though that the Cinryze study
was a 12-week study) and 2) safety should represent the key differentiator
as a recombinant protein compared to plasma based Cinryze. The risks
around plasma based products continue, including thromboembolic events
and bloodborne infections. Pharming and Valeant will now engage the FDA
and EMA to discuss the path forward for Ruconest in the prophylaxis setting
as the company continues to "block and tackle" in building its revenue and
geographies in the acute setting. From a perception standpoint, we believe
investors still have some trepidations, not on the strength of the data/product,
but rather on the ongoing trials and tribulations at Valeant.
ActionWe maintain our Buy rating and $1.84 price target. The company's strategy to
expand geographies through collaborations and to develop therapies for rare
diseases should bear fruit over the long term, in our opinion. Given the pricing
power of orphan drugs and the expanding markets in these indications due to
better diagnoses, we believe that Pharming is well-positioned for commercial
success.