Being busy and awesome
This morning, we announced strong results for Q4 and the full year 2012, reflecting successful execution on our mission to develop and deliver life-transforming therapies for patients with disorders that are severe, life-threatening, and also ultra-rare. Over the past year, we have significantly grown our commercial operations in existing and new countries, and accelerated our pipeline programs with five highly innovative therapeutic candidates, including eculizumab (Soliris®), in nine severe and ultra-rare disorders. During Q4 2012, we made strong progress across our three primary strategic growth initiatives:
First, our global rollout in PNH proceeded during Q4 with a steady addition of patients commencing Soliris therapy. In 2013, deeper penetration in existing and new countries positions us to continue to strongly grow our PNH operations.
· Second, in Q4 we continued to observe a steady addition of new patients in our aHUS launch in the US. In 2013, we look forward to serving more patients with aHUS in the US, as well as beginning to serve more patients in a growing number of countries in Western Europe.
· Third, during Q4 we continued to advance our lead development programs in severe and ultra-rare disorders with eculizumab (Soliris®) and 4 additional highly innovative therapeutics. Importantly, as we prepare for continued strong growth over the course of many years, each of our 9 lead programs is expected to reach one or more significant milestones in 2013, including registration trial commencements, registration dossier submissions, and key data publications.
I would like to highlight some key points within each of these initiatives.
Steady Growth in PNH and aHUS: Continuing to Serve More Patients Worldwide
In PNH during Q4, a significant number of new patients again commenced Soliris therapy in our core territories of the U.S., Western Europe and Japan. Importantly, and as in past quarters, the majority of PNH patients newly started on Soliris during Q4 were also newly diagnosed, reflecting the ongoing positive impact that we are having on patient care through our disease awareness programs and diagnostic initiatives. In a growing number of countries, we are helping physicians to better understand the progressive, life-threatening complications of PNH and the transformative clinical benefits of Soliris.
Also in 2012, we continued to expand our PNH operations in additional large countries, including Turkey, Brazil and Russia. More than six years after the PNH launch, our mission is as important - and urgent - as ever, because on a global basis, the majority of patients with PNH have not yet received an accurate diagnosis, let alone started appropriate treatment.
In aHUS, we had significant success in the first full year of our launch in the US during 2012. In 2013, we expect to serve more patients with aHUS in the US while completing the reimbursement processes in the larger Western European Countries, enabling us to launch in these additional major markets later in the year. Looking beyond the US and Europe, we announced on this morning’s call that we have recently submitted our sBLA for aHUS in Japan.
Leveraging Multiple Indications Across Our Expanded Global Presence
In 2012, we had a full year of both PNH and aHUS operations in the US - resulting in a growth rate of 52%. We believe that - over time, as our global introductions in PNH and aHUS continue - we will have the opportunity to serve a similar or greater number of aHUS patients compared to PNH across the 50 countries in which we now serve patients. And as these equally large PNH and aHUS opportunities are developed over the course of many years, we expect, likewise, to introduce new indications for Soliris, and new products, which will be launched on the same global platform.
The Most Robust Pipeline in Our History
We enter 2013 with the most robust and focused pipeline in the Company’s history, with five highly innovative therapeutic candidates at various stages of development across nine severe and life-threatening disorders that are also ultra-rare. Our R&D team will continue to drive our programs forward with urgency as we plan to achieve key milestones across our pipeline in 2013.
Eculizumab programs in severe and ultra-rare complement-mediated disorders
Neurology - We are highly encouraged by the early results and the large opportunity to serve patients with severe and relapsing neuromyelitis optica, or NMO. Following the presentation of positive data from an investigator initiated study during Q4, we have recently met with regulators in both the US and Europe to discuss plans for a study in patients with severe relapsing NMO. Based on these discussions, we are now accelerating this program and expect to conduct a single multinational registration trial commencing in the next few months. Also in neurology, we are scheduled to meet with regulators to discuss our plans for a larger, prospective, controlled trial in patients with severe myasthenia gravis, with a goal of initiating our company-sponsored MG registration program in the second half of 2013.
STEC-HUS - During Q4, we were encouraged by the presentation of positive final data from our single arm eculizumab STEC-HUS trial Germany. These data will now be significantly augmented with the addition of control data from an epidemiology study including patients who did not receive eculizumab during the crisis. We expect to file an application for regulatory approval in STEC-HUS later in 2013.
Kidney Transplant (Acute Humoral Rejection) - In addition to our living-donor and deceased-donor studies in acute humoral rejection in patients undergoing kidney transplant, there is now underway a 3rd clinical study in transplant patients at elevated risk for delayed graft function, or DGF. We expect enrollment to be complete in the deceased donor and living donor trials this year, and for data from the investigator-initiated DGF study to be presented later this year. We anticipate to accelerate our development in DGF with an Alexion sponsored multinational DGF registration program.
Programs with innovative therapeutics beyond eculizumab for patients with severe, ultra-rare and life-threatening disorders
In addition to eculizumab, we have four additional candidates with the potential to provide breakthrough innovations to patients with severe ultra-rare disorders who currently lack effective treatments.
Asfotase Alfa - We have completed the process development work for the current asfotase alfa manufacturing program. In 2013, we expect to complete the natural history study in infants, which will supplement the existing, open-label infant trials in the first half of the year. In addition, we plan to initiate a placebo-controlled study in juveniles with HPP in the next few months. We will be discussing the registration process for pediatric patients with regulatory authorities, with an eye toward a pediatric filing in the US and EU to follow. Also, following recent discussions with the PMDA in Japan, we have now initiated enrollment in our clinical trial for asfotase alfa in Japan, after which we anticipate filing a registration dossier in Japan.
cPMP replacement therapy - also in our metabolic disease area, is an investigational candidate for the treatment of patients with Molybdenum Cofactor Deficiency Type A - a severe, ultra-rare and genetic metabolic disorder that is fatal in newborns. In the next several months, we expect to meet with regulators to discuss plans for clinical studies, and to complete GMP manufacturing runs of cPMP in order to initiate clinical studies.
ALXN1102 and ALXN1103 - are two forms (intravenous and subcutaneous) of our novel inhibitor of the alternative complement pathway. We are on track to complete these Phase I studies in the next several months, and to meet with regulators to discuss trial data and the development program later in the year.
ALXN1007 - we have recently completed dosing in our 56 patient, single-dose, intravenous Phase I clinical study. Our next step in this program will be to discuss with regulators the initiation of a multi-dose, Phase I study, after which we will explore development in multiple severe, life-threatening, and ultra-rare conditions.
2012 Financial Results
For the full year of 2012, we recorded sales of 1.134 Billion, an increase of 45% compared to 2012. By maintaining fiscal discipline as we have grown our global operations, we achieved a 60% increase in non-GAAP net income to $425.2 million in 2012. I invite you to read our Q4 and full year 2012 press release, and listen to a replay of the Q4 and full year 2012 conference call, both of which can be accessed on our corporate web site at www.alexionpharma.com.
Our Steadfast Focus on Serving Patients
As we enter 2013, we have a great deal to be proud of as a company dedicated to helping patients and families who currently live with no treatments, and no hope, for the severe and ultra-rare disorders from which they suffer. 2012 was a great year for our company, and we expect 2013 to be still greater. As always, it is your hard work and dedication that make our accomplishments possible. On behalf of the entire management team, I’d like to thank you for all you do, each day, on behalf of patients around the world.
First, our global rollout in PNH proceeded during Q4 with a steady addition of patients commencing Soliris therapy. In 2013, deeper penetration in existing and new countries positions us to continue to strongly grow our PNH operations.
· Second, in Q4 we continued to observe a steady addition of new patients in our aHUS launch in the US. In 2013, we look forward to serving more patients with aHUS in the US, as well as beginning to serve more patients in a growing number of countries in Western Europe.
· Third, during Q4 we continued to advance our lead development programs in severe and ultra-rare disorders with eculizumab (Soliris®) and 4 additional highly innovative therapeutics. Importantly, as we prepare for continued strong growth over the course of many years, each of our 9 lead programs is expected to reach one or more significant milestones in 2013, including registration trial commencements, registration dossier submissions, and key data publications.
I would like to highlight some key points within each of these initiatives.
Steady Growth in PNH and aHUS: Continuing to Serve More Patients Worldwide
In PNH during Q4, a significant number of new patients again commenced Soliris therapy in our core territories of the U.S., Western Europe and Japan. Importantly, and as in past quarters, the majority of PNH patients newly started on Soliris during Q4 were also newly diagnosed, reflecting the ongoing positive impact that we are having on patient care through our disease awareness programs and diagnostic initiatives. In a growing number of countries, we are helping physicians to better understand the progressive, life-threatening complications of PNH and the transformative clinical benefits of Soliris.
Also in 2012, we continued to expand our PNH operations in additional large countries, including Turkey, Brazil and Russia. More than six years after the PNH launch, our mission is as important - and urgent - as ever, because on a global basis, the majority of patients with PNH have not yet received an accurate diagnosis, let alone started appropriate treatment.
In aHUS, we had significant success in the first full year of our launch in the US during 2012. In 2013, we expect to serve more patients with aHUS in the US while completing the reimbursement processes in the larger Western European Countries, enabling us to launch in these additional major markets later in the year. Looking beyond the US and Europe, we announced on this morning’s call that we have recently submitted our sBLA for aHUS in Japan.
Leveraging Multiple Indications Across Our Expanded Global Presence
In 2012, we had a full year of both PNH and aHUS operations in the US - resulting in a growth rate of 52%. We believe that - over time, as our global introductions in PNH and aHUS continue - we will have the opportunity to serve a similar or greater number of aHUS patients compared to PNH across the 50 countries in which we now serve patients. And as these equally large PNH and aHUS opportunities are developed over the course of many years, we expect, likewise, to introduce new indications for Soliris, and new products, which will be launched on the same global platform.
The Most Robust Pipeline in Our History
We enter 2013 with the most robust and focused pipeline in the Company’s history, with five highly innovative therapeutic candidates at various stages of development across nine severe and life-threatening disorders that are also ultra-rare. Our R&D team will continue to drive our programs forward with urgency as we plan to achieve key milestones across our pipeline in 2013.
Eculizumab programs in severe and ultra-rare complement-mediated disorders
Neurology - We are highly encouraged by the early results and the large opportunity to serve patients with severe and relapsing neuromyelitis optica, or NMO. Following the presentation of positive data from an investigator initiated study during Q4, we have recently met with regulators in both the US and Europe to discuss plans for a study in patients with severe relapsing NMO. Based on these discussions, we are now accelerating this program and expect to conduct a single multinational registration trial commencing in the next few months. Also in neurology, we are scheduled to meet with regulators to discuss our plans for a larger, prospective, controlled trial in patients with severe myasthenia gravis, with a goal of initiating our company-sponsored MG registration program in the second half of 2013.
STEC-HUS - During Q4, we were encouraged by the presentation of positive final data from our single arm eculizumab STEC-HUS trial Germany. These data will now be significantly augmented with the addition of control data from an epidemiology study including patients who did not receive eculizumab during the crisis. We expect to file an application for regulatory approval in STEC-HUS later in 2013.
Kidney Transplant (Acute Humoral Rejection) - In addition to our living-donor and deceased-donor studies in acute humoral rejection in patients undergoing kidney transplant, there is now underway a 3rd clinical study in transplant patients at elevated risk for delayed graft function, or DGF. We expect enrollment to be complete in the deceased donor and living donor trials this year, and for data from the investigator-initiated DGF study to be presented later this year. We anticipate to accelerate our development in DGF with an Alexion sponsored multinational DGF registration program.
Programs with innovative therapeutics beyond eculizumab for patients with severe, ultra-rare and life-threatening disorders
In addition to eculizumab, we have four additional candidates with the potential to provide breakthrough innovations to patients with severe ultra-rare disorders who currently lack effective treatments.
Asfotase Alfa - We have completed the process development work for the current asfotase alfa manufacturing program. In 2013, we expect to complete the natural history study in infants, which will supplement the existing, open-label infant trials in the first half of the year. In addition, we plan to initiate a placebo-controlled study in juveniles with HPP in the next few months. We will be discussing the registration process for pediatric patients with regulatory authorities, with an eye toward a pediatric filing in the US and EU to follow. Also, following recent discussions with the PMDA in Japan, we have now initiated enrollment in our clinical trial for asfotase alfa in Japan, after which we anticipate filing a registration dossier in Japan.
cPMP replacement therapy - also in our metabolic disease area, is an investigational candidate for the treatment of patients with Molybdenum Cofactor Deficiency Type A - a severe, ultra-rare and genetic metabolic disorder that is fatal in newborns. In the next several months, we expect to meet with regulators to discuss plans for clinical studies, and to complete GMP manufacturing runs of cPMP in order to initiate clinical studies.
ALXN1102 and ALXN1103 - are two forms (intravenous and subcutaneous) of our novel inhibitor of the alternative complement pathway. We are on track to complete these Phase I studies in the next several months, and to meet with regulators to discuss trial data and the development program later in the year.
ALXN1007 - we have recently completed dosing in our 56 patient, single-dose, intravenous Phase I clinical study. Our next step in this program will be to discuss with regulators the initiation of a multi-dose, Phase I study, after which we will explore development in multiple severe, life-threatening, and ultra-rare conditions.
2012 Financial Results
For the full year of 2012, we recorded sales of 1.134 Billion, an increase of 45% compared to 2012. By maintaining fiscal discipline as we have grown our global operations, we achieved a 60% increase in non-GAAP net income to $425.2 million in 2012. I invite you to read our Q4 and full year 2012 press release, and listen to a replay of the Q4 and full year 2012 conference call, both of which can be accessed on our corporate web site at www.alexionpharma.com.
Our Steadfast Focus on Serving Patients
As we enter 2013, we have a great deal to be proud of as a company dedicated to helping patients and families who currently live with no treatments, and no hope, for the severe and ultra-rare disorders from which they suffer. 2012 was a great year for our company, and we expect 2013 to be still greater. As always, it is your hard work and dedication that make our accomplishments possible. On behalf of the entire management team, I’d like to thank you for all you do, each day, on behalf of patients around the world.