papaji schreef op 30 juli 2016 09:45:
Pharming 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. Event
Pharming 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).Impact
This 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. Action
We 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.
above an extract from analyst roth coverage 28/july:www.pharming.com/wp-content/uploads/2...