Positive Data on Alexion's Soliris - Analyst Blog

Alexion Pharmaceuticals Inc. (ALXN) recently announced final data from a 26-week phase IIa trial investigating Soliris as a treatment for atypical hemolytic uremic syndrome (aHUS) in adolescent and adult patients who are resistant to plasma therapy at the American Society of Nephrology annual meeting.

Interim data from the study was earlier presented in the form of abstracts in October 2010. Soliris met its primary and secondary endpoints with statistical significance and clinical relevance. The trial met its primary endpoint of increased platelet counts from baseline, a measure of thrombotic microangiopathy process (TMA or blood clotting).

The secondary endpoints were TMA event-free status (defined as stable platelet counts, absence of plasma therapy and no new dialysis), improvement in kidney function and quality of life. 88% of patients achieved the key secondary endpoint of TMA-event-free status.

aHUS is a rare condition affecting the kidneys. The company hopes to be in a position to launch Soliris for adult/adolescent aHUS in 2012. Soliris is currently in a single-arm, phase II, multi-center study that is being conducted with pediatric patients for aHUS.

Soliris was approved by the FDA and the European Commission in 2007 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The drug is currently marketed in EU, US, Canada, Japan and some Latin American countries for PNH.

Soliris is also in different stages of development for indications like acute humoral kidney rejection (AHR), dense deposit disease or membranoproliferative glomerulonephristis (MPGN II), cold agglutinin disease (CAD), neuromyelitis optica, multifocal motor neuropathy, myasthenia gravis, and the dry form of age related macular degeneration (dry AMD).

Soliris has been granted orphan drug designation for the treatment of PNH, which means that Alexion has market exclusivity for the drug for seven years in the US and ten years in Europe. Soliris also has orphan protection in Japan until 2020.  The company also has orphan drug designation for the aHUS indication, both in the US and EU.

Our Recommendation   

Alexion currently has a Zacks #3 Rank, which translates into a short-term Hold rating. We are also Neutral on the stock in the long term. We believe that Soliris has strong growth potential from increased penetration in existing markets and contributions from new territories that will drive product sales for the company. Besides, approval for the aHUS indication could be a major growth driver for the company.

However, we remain concerned about the company's overt dependence on a single product for growth. Although Soliris is being evaluated in multiple additional indications, most of them are in the early to mid stages of development. Given the absence of any late-stage pipeline candidate, we are concerned about the company's long-term growth prospects.