BRIDGEWATER, N.J., Nov. 13, 2012 /PRNewswire/ -- Savient Pharmaceuticals, Inc. (NASDAQ: SVNT) today announced results from new post-hoc analyses of KRYSTEXXA® (pegloticase) pivotal Phase III and open-label extension trials that reinforced the health-related quality of life (HRQOL) benefits of KRYSTEXXA treatment in adult patients with refractory chronic gout (RCG), including tophus reduction outcomes. New data also demonstrated that guidance on measuring serum uric acid (sUA) levels as a biomarker of therapeutic response and infusion reaction (IR) risk significantly reduced the rate of IRs in the post-approval setting, as compared to rates seen during randomized clinical trials. These data will be presented this week at the American College of Rheumatology/Associate Rheumatology Health Professional (ACR/ARHP) Annual Meeting this week, November 9-14, 2012.
An analysis examining relative risk of IRs with KRYSTEXXA during a defined post-approval period (September 2010 September 2012) supported the effectiveness of the clinical guidance contained in the current U.S. label designed to identify patients at potentially higher risk for IRs by monitoring sUA response and discontinuing treatment if levels increase to above 6 mg/dL, particularly when two consecutive levels above 6 mg/dL are observed. The analysis showed that there was an approximate two-thirds reduction in the incidence of IRs in the post-approval period compared to the rate seen in the randomized clinical trials where there was no clinical guidance in effect.
In another analysis, researchers observed sustained patient-reported HRQOL improvements among participants identified as responders during their first six months of treatment in the six-month, randomized, placebo-controlled Phase III trials for an additional two-and-a-half years when treated with KRYSTEXXA 8 mg every two weeks during open-label extension study. These outcomes were measured through widely-utilized assessment tools, including Medical Outcomes Study Short Form-36 (SF-36) physical component summary scores and the Health Assessment Questionnaire-Disability Index (HAQ-DI).
"This is the first therapy for chronic gout that has shown sustained improvement in the health-related quality of life. Further knowledge of how KRYSTEXXA can improve long-term health-related quality of life outcomes for responsive RCG patients helps us to better understand the treatment's utility for these severe gout patients," said Dinesh Khanna, M.D., M.Sc., Associate Professor, Department of Internal Medicine at The University of Michigan.
For patients with RCG, tophus burden is a serious factor in the condition's impact on long-term functionality. Tophi form when uric acid collects and crystallizes in and around the joints. Over time, if not treated appropriately, tophi can lead to the disabling of joints, immobility and bone destruction. Another analysis characterized the reduction in tophus burden experienced by RCG patients treated with KRYSTEXXA. The study examined patients' tophi response to KRYSTEXXA from baseline of Phase III trials through the conclusion of the open-label extension trial. Of the 212 patients with RCG enrolled in the Phase III trials, 155 (73 percent) had tophi at baseline. A complete response to therapy was defined as a 100 percent decrease in the area of the target tophus and the absence of any new or enlarging tophi. Complete response of at least one tophus was significantly more frequent among patients receiving KRYSTEXXA at 13 weeks of treatment and at all subsequent times of measurement in the pivotal trials, with 40 percent of patients achieving complete response after six months as compared to seven percent treated with placebo (p=0.002). After one year in the open-label extension study, complete response was achieved for 74 percent of patients receiving KRYSTEXXA. Despite receiving no urate-lowering therapy during six months of enrollment in the Phase III study, approximately 50 percent of previously placebo-treated patients achieved tophus complete response within six months of treatment with KRYSTEXXA 8 mg every two weeks in the open-label extension study.
"Savient is committed to supporting research that will further understanding surrounding KRYSTEXXA, RCG and how these patients' treatment experiences and ultimately their quality of life may be improved," said Kenneth M. Bahrt, M.D., Senior Vice President and Chief Medical Officer of Savient. "These analyses reinforce our confidence in KRYSTEXXA and its ability to fulfill a significant unmet need for many patients who face the debilitating effects of RCG on a daily basis."
Full text ACR/ARHP abstracts on these studies can be found via the following links:
KRYSTEXXA®(pegloticase) is a PEGylated uric acid specific enzyme for administration by intravenous infusion for the treatment of refractory chronic gout (RCG) in adult patients. KRYSTEXXAbecame commercially available in the U.S. by prescription on December 1, 2010 and is the only U.S. Food and Drug Administration approved product specifically indicated for the treatment of RCG. KRYSTEXXA is not recommended for the treatment of asymptomatic hyperuricemia.
For more information about KRYSTEXXA,please visit: http://www.KRYSTEXXA.com.
IMPORTANT SAFETY INFORMATION ABOUT TREATMENT WITH KRYSTEXXA®
KRYSTEXXA is not indicated for the treatment of asymptomatic hyperuricemia. Patients who are at risk of having a condition known as G6PD deficiency should be screened by their physician prior to starting therapy with KRYSTEXXA.
Discontinue oral urate-lowering therapies before instituting KRYSTEXXA and do not institute oral urate-lowering therapy while the patient is on KRYSTEXXA therapy.
Possible side effects of KRYSTEXXA include:
- Anaphylaxis which occurred in some patients treated with KRYSTEXXA. KRYSTEXXA should be administered in healthcare settings and by healthcare providers prepared to manage anaphylaxis. Patients should be pre-medicated with antihistamines and corticosteroids. Patients should be closely monitored for an appropriate period of time for anaphylaxis after administration of KRYSTEXXA.
- Infusion reactions which occurred in some patients treated with KRYSTEXXA. The risk of an infusion reaction is higher in patients who have lost therapeutic response. Because the risk of infusion reactions is higher in patients who lose therapeutic response to KRYSTEXXA, monitor serum uric acid before each infusion and consider discontinuing treatment if levels rise above 6mg/dL, particularly when two consecutive levels above 6 mg/dL are observed.
- As with other urate-lowering therapies, an increase in gout flares was seen in some patients treated with KRYSTEXXA. Gout flare prophylaxis with a non-steroidal anti-inflammatory drug (NSAID) or colchicine is recommended starting at least 1 week before initiation of KRYSTEXXA therapy and lasting at least 6 months, unless medically contraindicated or not tolerated.
KRYSTEXXA has not been formally studied in patients with congestive heart failure, but some patients in clinical trials experienced exacerbation. Exercise caution when using KRYSTEXXA in patients who have congestive heart failure and monitor patients closely following infusion. Patients receiving re-treatment may be at increased risk for anaphylaxis and infusion reactions and should be monitored carefully.
The most commonly reported serious adverse reactions are anaphylaxis, infusion reactions and gout flares. Most common adverse reactions: gout flares (77%), infusion reactions (26%), nausea (12%), contusion or ecchymosis (11%), nasopharyngitis (7%), constipation (6%), chest pain (6%), anaphylaxis (5%), and vomiting (5%).
Please see the Full Prescribing Information and Medication Guide at http://www.krystexxa.com/.
ABOUT REFRACTORY CHRONIC GOUT
Gout is a painful, debilitating form of arthritis and affects approximately eight million people in the U.S. alone. A significant sub-population of gout patients, approximately 120,000, are burdened with a difficult-to-treat form of the condition, known as refractory chronic gout (RCG). Symptoms of gout are caused by the body's response to the presence of uric acid crystals in the joints and surrounding tissue, which form when uric acid levels in the blood are elevated (a condition called hyperuricemia). The longer hyperuricemia persists, the higher the risk of developing gout. Symptoms of gout may include painful flares, pain or swelling in the joints (known as "gouty arthritis") or deposits of uric acid crystals under the skin, called "tophi." In cases of RCG, these symptoms may have a major influence on patient health-related quality of life due to the frequency and severity of episodes, the recurrent pain and the disfigurement associated with this condition. Although most cases of gout can be controlled with conventional urate-lowering therapy, when uric acid levels remain high and symptoms persist despite treatment efforts, chronic gout may be defined as refractory.
ABOUT SAVIENT PHARMACEUTICALS, INC.
Savient Pharmaceuticals, Inc. is a specialty biopharmaceutical company focused on developing and commercializing KRYSTEXXA® (pegloticase) for the treatment of chronic gout in adult patients refractory to conventional therapy. Savient has exclusively licensed worldwide rights to the technology related to KRYSTEXXA and its uses from Duke University ("Duke") and Mountain View Pharmaceuticals, Inc. ("MVP"). Duke developed the recombinant uricase enzyme and MVP developed the PEGylation technology used in the manufacture of KRYSTEXXA. MVP and Duke have been granted U.S. and foreign patents disclosing and claiming the licensed technology and, in addition, Savient owns or co-owns U.S. and foreign patents and patent applications, which collectively form a broad portfolio of patents covering the composition, manufacture and methods of use and administration of KRYSTEXXA. Savient also supplies Oxandrin® (oxandrolone tablets, USP) CIII in the U.S. For more information, please visit the Company's website at www.savient.com.
All statements other than statements of historical facts included in this press release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and Section 21E of the Securities Exchange Act of 1934, as amended, and are subject to certain risks, trends and uncertainties that could cause actual results and achievements to differ materially from those expressed in such statements. These risks, trends and uncertainties are in some instances beyond our control. Words such as "anticipate," "believe," "estimate," "expect," "intend," "plan," "will" and other similar expressions identify forward-looking statements, although not all forward-looking statements contain these identifying words. In particular, any statements regarding the safety and efficacy of KRYSTEXXA®, market demand and our ability to gain market acceptance for KRYSTEXXA among physicians, patients, health care payors and others in the medical community; our ability to execute on our plans for the expansion of clinical utility for KRYSTEXXA; our market expansion plans for KRYSTEXXA outside the United States, including our Marketing Authorization Application which is pending before the European Medicine Agency, our ability to service our outstanding debt obligations, our financing needs and liquidity, market acceptance of reimbursement risks with third party payors, and our view of the market size for KRYSTEXXA in the US and ex-US and our view of our penetration of this market are forward-looking statements. These forward-looking statements involve substantial risks and uncertainties and are based on our assessment and interpretation of the currently available data and information, current expectations, assumptions, estimates and projections about our business and the biopharmaceutical and specialty pharmaceutical industries in which we operate. Other important factors that may affect our business are set forth more fully in our reports filed with the Securities and Exchange Commission, to which investors are referred for further information. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements, which speak only as of the date of publication of this press release. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements that we make. Our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, dispositions, joint ventures or investments that we may make. We do not have a policy of updating or revising forward-looking statements and, except as required by law, assume no obligation to update any forward-looking statements.
SOURCE Savient Pharmaceuticals, Inc.