Replies to post #37 on Alexion Pharmaceuticals, Inc. (ALXN)

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Alexion Cites Positive Soliris Data

Zacks Equity Research, On Wednesday June 15, 2011, 3:15 pm EDT

Alexion Pharmaceuticals Inc. (NasdaqGS: ALXN - News) recently presented final data from two 26-week studies evaluating its sole marketed drug, Soliris, for an additional indication. The studies evaluated Soliris for treating adults and adolescents suffering from atypical hemolytic uremic syndrome (aHUS). Both the studies met the primary and secondary endpoints, consistent with the previously announced data. The results were presented at the European Hematology Association (EHA) Congress.

The first study tested patients on plasma exchange/ infusion, later followed by Soliris. The second study was conducted on patients who were intolerant to plasma exchange/ infusion. These patients were given Soliris. The data revealed that Soliris has a significant impact on reducing TMA which is the main reason behind life-threatening events in aHUS patients. This reduction in TMA helps in significant improvements in kidney function and reduces the need for interventions like dialysis for these patients, leading to a better quality of life.

Alexion also presented data from a study investigating the use of Soliris in pediatric patients with aHUS at the EHA Congress. The data revealed that Soliris significantly reduced TMA in pediatric patients too.

We note that Soliris is already available in the US for treating patients suffering from paroxysmal nocturnal hemoglobinuria (PNH), a rare genetic blood disorder. Besides the US, Soliris is approved for PNH in the EU, Japan and many other countries. However, Alexion’s efforts to expand the drug’s label are encouraging since the sole dependence on Soliris, only for PNH, is risky.

Only in this month, the FDA granted priority review status to the supplemental Biologics License Application (sBLA) filed by Alexion in April 2011 for the aHUS indication. If approved, Alexion could launch Soliris for aHUS in the US in the fourth quarter of 2011.

Soliris is under review in the European Union for the same indication.

We note that the market for aHUS holds immense potential as patients suffering from the disorder have limited treatment options. Approximately 60% of patients with aHUS require a kidney transplant or a dialysis. Those who receive a kidney transplant continue facing complications and, in more than 90% of these patients, the donor kidney fails. We are hopeful that Soliris can leverage the unmet demand for the product thus gaining market share.

Alexion Pharma is also studying Soliris in patients undergoing kidney transplantation, having a high risk of organ rejection. The condition is referred to as acute humoral rejection (AHR).

Our Recommendation

Currently, we have a Neutral stance on Alexion. The stock carries a Zacks #3 Rank (short-term Hold recommendation).

We believe that Alexion’s future is tied to Soliris. Approval of the drug in additional markets should help drive sales in the coming quarters. Alexion’s efforts to expand the label of Soliris are also encouraging. We, however, remain concerned about Alexion’s dependence on a single product for growth. Given the absence of any late-stage pipeline candidate, we are cautious about the company’s long-term growth prospects.

We prefer to remain on the sidelines until more visibility is obtained regarding the pipeline development at Alexion and retain our Neutral stance on the stock.

ALEXION PHARMACEUTICALS INC (ALXN): Read the Full Research Report

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Researchers Present Final Data from Phase 2 Studies of Soliris® (eculizumab) in Patients with aHUS

Clinical Data from Retrospective Study in Pediatric Patients with aHUS Also Presented at EHA Congress

Press Release Source: Alexion Pharmaceuticals, Inc. On Saturday June 11, 2011, 12:46 pm EDT

CHESHIRE, Conn.--(BUSINESS WIRE)-- Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN - News) today announced the presentation of final data from the two Phase 2 studies of Soliris® (eculizumab) as a treatment for patients with atypical hemolytic uremic syndrome (aHUS): (i) a study in patients who were resistant to plasma exchange/infusion and received eculizumab and (ii) a study in patients who were receiving chronic plasma exchange/infusion followed by late intervention with eculizumab. Consistent with previously announced data, both studies met their primary endpoints and key secondary endpoints with high levels of statistical and clinical significance. Separately, researchers presented for the first time findings from a retrospective clinical study of eculizumab in pediatric patients with aHUS. These three studies were presented at the 16th Congress of the European Hematology Association (EHA) in London.

aHUS is an ultra-rare, genetic, life-long disease in which chronic uncontrolled complement activation causes blood clots in small blood vessels throughout the body (thrombotic microangiopathy, or TMA), leading patients to suffer kidney failure, stroke, heart attack and death.1,2,3 Approximately 60% of patients with aHUS require dialysis, undergo a kidney transplant, or die within one year of diagnosis.4 Alexion has filed marketing applications with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for eculizumab, a first-in-class terminal complement inhibitor, as a treatment for patients with aHUS.

“Final data from these pivotal clinical trials suggest that complement inhibition therapy with eculizumab could potentially alter the course of aHUS,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “By considerably reducing TMA, investigators have demonstrated that eculizumab improved kidney function, reduced the need for interventions such as dialysis, and substantially improved quality of life in the studied patients. We continue to work closely with regulatory authorities and the aHUS community with the goal of offering a safe and effective treatment option for patients suffering with this ultra-rare, devastating, and life-threatening disease.”

Patients Resistant to Plasma Exchange/Infusion

In a poster session today, researchers presented final data from a Phase 2 study of eculizumab in 17 adult and adolescent patients who were resistant or intolerant to plasma exchange/infusion and received eculizumab.7 Fifteen patients received eculizumab therapy for 26 weeks; two patients did not complete the study but were included in the analysis. Of note, patients in this study had a median duration of aHUS from diagnosis to screening of 10 months (range: 1–236).

The study met its primary endpoint and key secondary endpoints with high levels of statistical and clinical significance, and findings were consistent with data reported at the 2010 American Society of Nephrology annual meeting. For the primary endpoint, platelet count increased from baseline through week 26 by a point estimate 73×109/L (p=0.0001). Platelet count was normalized in 13 out of 15 patients (87%) who had abnormal platelets at baseline; 100% of patients who were analyzed per protocol achieved normal platelet count.

With regard to secondary endpoints, 15 patients (88% of the total enrollment and 100% of those analyzed per protocol) achieved TMA event-free status, defined as at least 12 consecutive weeks of stable or increasing platelet counts, absence of plasma exchange/infusion, and no new dialysis. Median TMA intervention (plasma exchange/infusion/dialysis) rate decreased from 0.88 events per patient per day to 0 (p<0.0001). Four out of 5 dialysis patients became dialysis-free, and 10 patients (59%; 95% CI 33-82) had sustained improvement in chronic kidney disease by at least one stage. Eight out of 17 patients (47%; 95% CI 23-82) had increased renal function by at least 15 mL/min/1.73 m2. Improvements in health-related quality of life (HRQoL) were highly statistically significant, with 80% of patients experiencing a clinically meaningful benefit. The mean change in HRQoL from baseline to week 26 of eculizumab treatment was 0.29±0.28 (p<0.002). Eculizumab was well tolerated in the study. The most frequently reported adverse events were headache, anemia and diarrhea (generally mild to moderate in severity). An extension trial continues.

Patients Receiving Chronic Plasma Exchange/Infusion

In another poster presentation today, researchers presented final data from a Phase 2 study of Soliris in aHUS patients who were receiving chronic plasma exchange/infusion.5 In this 26-week study, 20 patients continued to receive plasma exchange/infusion during an 8-week observation period, and then discontinued plasma exchange/infusion and commenced eculizumab treatment. Of note, patients in this study received late intervention with eculizumab: they had a median duration of aHUS from diagnosis to screening of 48 months (range: 0.66–286), with a long duration of renal insufficiency. In the study, 80% of patients achieved TMA event-free status, the primary endpoint, defined as at least 12 consecutive weeks of stable platelet count, absence of plasma exchange/infusion, and no new dialysis.

Key secondary endpoints were also achieved with high clinical and statistical significance. Platelet count normalization was achieved in 18 out of 20 patients (90%); all patients who had normal platelet count at baseline (17 out of 20) maintained that level after discontinuation of plasma exchange/infusion and commencement of eculizumab treatment. TMA intervention rate (number of plasma exchange/infusion and new dialysis events per patient per day) decreased from a median of 0.23 events per patient per day to zero events (p<0.0001). Seven out of 20 patients (35%, 95% CI 15 – 59) experienced a sustained improvement of at least one stage of chronic kidney disease (CKD). During treatment with eculizumab, all patients (100%) discontinued plasma exchange/infusion and did not require new dialysis.

In addition, treatment with eculizumab significantly improved quality of life, with 73% of patients achieving a clinically meaningful benefit. Mean change from baseline to week 26 was 0.11±0.19 (p<0.002). Eculizumab was well tolerated by patients in the study. The most frequently reported adverse events were diarrhea, headache, hypertension and nausea (generally mild to moderate in severity).

“The data presented today demonstrate again that eculizumab has a profound impact on TMA — the underlying cause of life-threatening events in patients with aHUS — which leads to permanent discontinuation of plasma exchange, significant improvements in kidney function and other critical measures in these patients,” said Chantal Loirat, M.D., Pediatric Nephrology Department, Hôpital Robert Debre, Paris.

Pediatric Patients

In a poster session on June 10, 2011, researchers presented data for the first time from a retrospective clinical study of 15 pediatric patients with aHUS who received at least one dose of eculizumab outside of prospective clinical trials between 2007 and 2009.6 All patients were under the age of 12 (under 2 years [n=5], 2 to 4 years [n=3], 5 to 11 years [n=7]).

The analysis showed that eculizumab treatment significantly reduced TMA in pediatric patients with aHUS, as platelet counts were normalized in 93% (14/15) and 73% (11/15) of patients achieved TMA-event free status. Importantly, 53% of patients had improved renal function with eculizumab treatment. In addition, four of six patients with dialysis in the 30 days prior to eculizumab did not require dialysis during eculizumab treatment. Eculizumab efficacy was similar across all pediatric age groups. The most frequently reported adverse events were fever, upper respiratory tract infection, diarrhea and cough. The results in this pediatric population are consistent with those from controlled trials in adult and adolescent patients in demonstrating that eculizumab controlled TMA, improved kidney function and reduced the need for plasma exchange/infusion.

Separately, Alexion is currently enrolling patients in a Phase 2, open-label, single-arm, multi-center study of Soliris in pediatric patients with aHUS in the United States, European Union and Canada. Information about the trial is available at www.clinicaltrials.gov, Identifier Number NCT01193348, or by contacting Alexion at [e-mail].