Alexion Pharmaceuticals, Inc (ALXN)
FDA Approval of Soliris as a Treatment for Patients with aHUS Call Transcript
September 26, 2011, 10:00 am ET
Leonard Bell - CEO
Tom Dubin - SVP, Chief Legal Officer
David Hallal - SVP, Global Commercial Operations
Vikas Sinha - SVP, CFO
Salveen Richter - Collins Stewart
Sapna Srivastava - Goldman Sachs
Eric Schmidt - Cowen and Company
Geoff Porges - Bernstein
Robin Karnauskas - Deutsche Bank
Matthew Luchini - Piper Jaffray
[Andrew] - UBS
Jonathan Eckard - Leerink Swan
Marko Kozul - ThinkEquity
[Brian Kwon] - Citigroup
Previous Statements by ALXN
» Alexion Pharmaceuticals CEO Discusses Q2 2011 Results - Earnings Call Transcript
» Alexion Pharmaceuticals Q4 2008 Earnings Call Transcript
» Alexion Pharmaceuticals Inc. Q3 2008 Earnings Conference Call Transcript
Thank you, Operator. Good morning and thank you for joining us on today's call to discuss the FDA approval of Soliris as a treatment for patients with aHUS. I am joined today by David Hallal, Senior Vice President, Global Commercial Operations, and Vikas Sinha, Senior Vice President and Chief Financial Officer.
Also with us today are Stephen Squinto, Executive Vice President and Head of R&D, and Tom Dubin, Senior Vice President and Chief Legal Officer. We are also extremely pleased to welcome our Alexion team working around the world.
I will begin today's call by discussing our broad label for all patients with aHUS. David will then outline our plan to provide Soliris to the first US patients with aHUS as we continue to serve more patients with PNH.
Finally, Vikas will discuss some key financial matters, including Friday's upward revision guidance resulting from our improved outlook in PNH.
Before we begin, Tom will apprise you of our potential to make forward-looking statements. Tom?
Thanks, Lenny. During this call, we may make forward-looking statements such as medical benefits of Soliris, commercial potential and plans for the commercial launch of Soliris and aHUS, plans for further clinical trials of Soliris and aHUS and operations, reimbursement, price approval and funding processes in different territories.
Forward-looking statements are subject to factors and may cause our results and plans to differ from those expected including decisions of regulatory authorities regarding approval or limitations on the marketing of Soliris for various indications; the possibility that results of clinical trials are not predictive of the safety and efficacy of Soliris in broader patient populations in the disease study or in other diseases; the risk that third parties won't agree to license any necessary intellectual property to us on reasonable terms or at all; the possibility that initial results of commercialization are not predictive of future results; the risk that third-party pairs will not continue to reimburse for the use of Soliris at acceptable rates or at all; and a variety of other risks set forth from time to time in our filings with the SEC, including our 10-Q for the quarter ended June 30, 2011.
We do not intend to update any of these forward-looking statements after this call except when the duty arises under law.
Before beginning this call, I want to point out that we will be limiting today's question-and-answer period to the subject of our aHUS approval in the US and positive opinions in the [CHMD].
A more complete update of Alexion's operations, research pipeline and financial performance will be provided in our third-quarter conference call, which we expect to hold in the second half of October. Thank you. Lenny?
Thanks, Tom. Friday was truly a historic day for patients and families suffering with atypical hemolytic uremic syndrome, or aHUS. The FDA approval of Soliris as the first treatment for patients with aHUS brings life-transforming hope that children and adults with this severe, debilitating and life-threatening ultra rare disorder.
With the approval of Soliris with a broad label, this unprecedented hope is now extended to patients with aHUS of all ages and all clinical profiles in the United States.
The FDA approval of a second indication for Soliris opens a new era for patients with aHUS. The approval is likewise a milestone in Alexion's mission to provide innovative and life-transforming therapies to patients with severe and ultra rare disorders.
As we continue to serve more patients with PNH in more countries, we are now also fully prepared to begin making Soliris immediately available to aHUS patients in the United States.
On Friday, we are also very pleased to announce the positive opinion by the [CHMP] recommending the granting of a marketing authorization for Soliris as a treatment for children and adults with aHUS in Europe, a key milestone for patient access.
Approval by the European Commission is expected in approximately two months. Following country-by-country reimbursement processes, this would enable us to start serving patients in the first major European countries in the first half of 2012 with additional major European countries commencing through mid-2013.
Turning back to the United States, we have received a broad and strong label that will help us accomplish our key goals on behalf of patients with aHUS. The label supports building a common understanding among physicians that aHUS is a disorder of uncontrolled complement activation and identifies the dramatic clinical benefits of terminal complement inhibition with Soliris in patients with this ultra rare and life-threatening disorder.
Most importantly, our strong label will support broad and unrestricted access to Soliris for all patients with aHUS in the United States. Soliris is indicated for the treatment of patients with aHUS to inhibit complement-mediated thrombotic microangiopathy, for TMA, thus the new product indication statement clearly identifies the disease mechanism of aHUS and ties it directly to the therapeutic action of Soliris.