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`Dec 21, 2012
`
`(cid:67) Previous Release | Next Release (cid:65) (cid:94) (cid:44)
`FDA Approves Gattex®
`(teduglutide [rDNA Origin]) for
`Injection for the Treatment of
`Adult Short Bowel Syndrome
`-- First major long-term treatment advance
`for adult SBS in nearly 40 years --
`-- U.S. launch in first quarter of 2013 --
`-- NPS Pharmaceuticals to host conference
`call on January 2, 2013 at 5:00 PM ET --
`
`BEDMINSTER, N.J., Dec. 21, 2012 /PRNewswire/ -- NPS
`Pharmaceuticals, Inc. (NASDAQ: NPSP), a biopharmaceutical company
`pioneering and delivering therapies that transform the lives of patients
`with rare diseases worldwide, today announced that the U.S. Food and
`Drug Administration (FDA) has approved Gattex® (Teduglutide [rDNA
`origin]) for Injection, for subcutaneous use for the treatment of adult
`patients with short bowel syndrome (SBS) who are dependent on
`parenteral support. Gattex will be available in the first quarter of 2013.
`For full prescribing information, please visit www.Gattex.com.
`
`To view the multimedia assets associated with this release, please click:
`http://www.multivu.com/mnr/59518-nps-pharmaceuticals-fda-approval-
`gattex-adult-short-bowel-syndrome-sbs
`
`"Gattex is a ground-breaking therapy that has been evaluated in the
`largest clinical program to date in short bowel syndrome," said Francois
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`CFAD Exhibit 1064
`CFAD v. NPS
`IPR2015-01093
`
`

`
`Nader, MD, president and chief executive officer of NPS
`Pharmaceuticals. "We are very excited about the opportunity to help
`SBS patients by offering this first-in-class therapy. The approval of
`Gattex is a crowning achievement for our company and the catalyst for
`our transformation into a premier orphan drug business. We are
`prepared for a successful launch based on productive interactions with
`payers, methodical patient identification, and the strategic development
`of our field-based commercial infrastructure. We have also launched
`NPS Advantage, our free support program that includes, care
`coordinators who will work closely with patients to help them
`understand the clinical characteristics of Gattex and navigate the
`reimbursement landscape."
`
`SBS is a serious, complex disorder in which the body is unable to absorb
`enough nutrients and fluids through the gastrointestinal tract to sustain
`life. SBS patients are commonly infused with PN/IV five to seven nights
`per week for up to 10 to 12 hours at a time, and in extreme cases,
`some patients could receive PN/IV for 24 hours a day. Long-term use of
`PN/ IV fluids can be associated with life-threatening complications such
`as liver damage, serious bloodstream infections, and blood clots.
`
`Gattex is the first major long-term treatment advance for SBS in nearly
`40 years. The unique mechanism of action of Gattex enhances
`gastrointestinal absorption. In Phase 3 studies, significant reductions in
`PN/IV volume and infusion days per week were achieved from pre-
`treatment baseline. In addition, some patients achieved independence
`from PN/IV support.
`
`"In addition to serious medical complications, patients with short bowel
`syndrome can have socially-restricted lives. Long infusion periods often
`disrupt sleep for patients. This is coupled with constant concern about
`using restrooms as many patients will need to use the bathroom up to
`25 times a day or having an accident with unpredictable diarrhea. Other
`patients that have an ostomy bag have a fear of an ostomy bag
`leakage. These factors leave many patients unable to socialize or work,"
`said Ken Fujioka, MD, Nutrition and Metabolic Research Center, Scripps
`Clinic, Del Mar. "Considering Gattex has been shown to significantly
`reduce or in some cases even eliminate the requirement for parenteral
`support, it may become a cornerstone therapy in the management of
`short bowel syndrome."
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`SBS typically occurs when a large portion of the intestine has been
`removed by surgery caused by disease or injury. In rare cases, it is
`congenital. Common symptoms of SBS can include diarrhea,
`dehydration, malnutrition, and weight loss, which are closely related to
`the functional issues of the bowel. During a typical day, SBS patients
`will move their bowels up to 10 times, or change their ostomy bag,
`which collects their urine output, three to four times. In extreme cases,
`patients may move their bowels as many as 25 times each day.
`
`"Patients with short bowel syndrome/intestinal failure need a
`comprehensive program to receive optimal care so that their quality of
`life is maintained to its fullest extent. The Oley Foundation encourages
`research and new development of drugs, products and services that
`improve health and well-being," said Joan Bishop, executive director,
`The Oley Foundation. "We are pleased that NPS Pharmaceuticals has
`engaged in this research and has set upon a path that serves to bring
`hope that it will reduce the challenges of short bowel
`syndrome/intestinal failure and improve the quality of life of many of
`our members and their families."
`
`NPS Advantage™
`
`To assist patients and healthcare professionals in facilitating care with
`Gattex, NPS has launched a free support program called NPS
`Advantage™. This program is designed to help navigate all aspects of
`care, help with insurance authorizations and appeals, answer questions
`about Gattex and its use, and locate resources for patients that connect
`them to care.
`
`A key feature of NPS Advantage is the involvement of experienced care
`coordinators, who provide comprehensive support with a single point of
`contact. These NPS professionals will work with Gattex patients to
`confirm authorizations and benefit approvals for Gattex and help resolve
`health insurance issues. They will also support healthcare professionals
`to streamline the reimbursement process for Gattex and help their
`patients obtain appropriate medical care. For more information, please
`visit http://www.npsadvantage.com.
`
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`Conference Call Information
`
`NPS will host a conference call to discuss its commercialization plan for
`Gattex, including the cost of therapy, on Wednesday, January 2, 2013
`at 5:00 p.m. Eastern Time. To participate in the conference call, dial
`(800) 706-7748 and use pass code 18085144. International callers
`may dial (617) 614-3473, using the same pass code. In addition, a live
`audio of the conference call will be available over the Internet.
`Interested parties can access the event through the NPS website,
`http://www.npsp.com.
`
`For those unable to participate in the live call, a replay will be available
`at (888) 286-8010, with pass code 79197503, until midnight Eastern
`Time, January 16, 2013. International callers may access the replay by
`dialing (617) 801-6888, using the same pass code. The webcast will
`also be available through the NPS website for the same period.
`
`About Short Bowel Syndrome
`
`Short bowel syndrome (SBS) is a highly disabling condition that can
`impair a patient's quality of life and lead to serious life-threatening
`complications. SBS typically arises after extensive resection of the bowel
`due to Crohn's disease, ischemia or other conditions. SBS patients often
`suffer from malnutrition, severe diarrhea, dehydration, fatigue,
`osteopenia, and weight loss due to the reduced intestinal capacity to
`absorb nutrients, water and electrolytes. The usual treatment for SBS is
`nutritional support, including parenteral nutrition (PN) and/or
`intravenous (IV) fluids to supplement and stabilize nutritional needs.
`
`Although PN can provide nutritional support for SBS patients, it does not
`improve the body's own ability to absorb nutrients. PN is associated with
`serious complications, such as infections, blood clots or liver damage,
`and the risks increase the longer patients are on PN. Patients on PN
`often experience poor quality of life with difficulty sleeping, and frequent
`urination, and patients receiving chronic PN often experience a loss of
`independence.
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`Gattex Clinical Trials
`
`NPS' clinical development program for Gattex is the largest and most
`comprehensive conducted in SBS patients to date, consisting of 15
`clinical studies. Across all clinical studies, 566 subjects were exposed to
`at least one dose of Gattex, of whom 134 had SBS and were treated
`with 0.05 mg/kg/day Gattex. The FDA's approval of Gattex was based
`on an international, 24-week, double-blind, placebo-controlled, pivotal
`Phase 3 trial, known as STEPS. The primary endpoint of STEPS was
`defined as a 20 percent or greater PN/IV volume reduction
`demonstrated at week 20 and sustained at week 24. The study's
`secondary endpoints included reductions in PN/IV volume and additional
`days off therapy. Key findings from the STEPS trial include:
`
`• In an intent-to-treat analysis at weeks 20 and 24, 63 percent of
`patients treated with Gattex achieved at least a 20 percent
`reduction in weekly PN/IV volume when compared to baseline,
`versus 30 percent for placebo (p=0.002).
`• After 24 weeks of treatment, PN volume declined by 32 percent
`(4.4 L/wk) in Gattex-treated patients, versus 21 percent (2.3
`L/wk) in the placebo group (p<0.001).
`• After 24 weeks of treatment, 54 percent of Gattex-treated patients
`were able to reduce the number of infusion days per week by one
`or more days, compared to 23 percent of those treated with
`placebo (p=0.005).
`
`The most common adverse reactions ((cid:149)10 percent) across all studies
`with Gattex are abdominal pain, injection site reactions, nausea,
`headaches, abdominal distension, upper respiratory tract infection. In
`addition, vomiting and fluid overload were reported in the Phase 3 SBS
`studies at rates (cid:149) 10 percent.
`
`For full prescribing information, please visit www.Gattex.com.
`
`About Gattex® (teduglutide [rDNA origin]) for Injection
`
`Gattex® (teduglutide [rDNA origin]) for Injection, for subcutaneous use
`is a novel, recombinant analog of human glucagon-like peptide 2, a
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`protein involved in the rehabilitation of the intestinal lining. Gattex®
`0.05 mg/kg/d (teduglutide [rDNA origin] for Injection) is indicated for
`the treatment of adult patients with short bowel syndrome (SBS) who
`are dependent on parenteral support. Significant reductions in mean
`PN/IV infusion volume from baseline to end of treatment were seen in
`the Phase 3 studies of Gattex. In addition, some patients were able to
`achieve independence from PN/IV support during these trials. The most
`common side effects of Gattex include stomach area (abdomen) pain or
`swelling, skin reaction where the injection was given, nausea, headache,
`cold or flu like symptoms, vomiting, and holding too much fluid in the
`body (swelling of face, ankles, hands or feet).
`
`Gattex has received orphan drug designation for the treatment of SBS
`from the European Medicines Agency (EMA) and the FDA.
`
`In 2007, NPS granted Takeda GmbH, the rights to develop and
`commercialize teduglutide outside the United States, Canada, Mexico
`and Israel. NPS retains all rights to teduglutide in North America. The
`European Commission granted European market authorization on
`August 30, 2012 for the medicinal product teduglutide (trade name in
`Europe: Revestive®) as a once-daily treatment for adult patients with
`short bowel syndrome.
`
`Teduglutide was discovered by Daniel J. Drucker, MD, currently a senior
`scientist based in the Samuel Lunenfeld Research Institute at Mt. Sinai
`Hospital, University of Toronto, in Toronto, Canada. NPS has an
`exclusive license agreement with Dr. Drucker for teduglutide and its
`therapeutic uses.
`
`Important Safety Information (ISI)
`
`Gattex has been associated with serious risks including:
`
`• Neoplastic growth. There is a risk for acceleration of neoplastic
`growth. Colonoscopy of the entire colon with removal of polyps
`must be done before initiating treatment with Gattex and is
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`recommended after 1 year. Subsequent colonoscopies should be
`done as needed, but no less frequently than every 5 years. In case
`of intestinal malignancy discontinue Gattex. The clinical decision
`to continue Gattex in patients with active non-gastrointestinal
`malignancy should be made based on risk and benefit
`considerations.
`• Intestinal obstruction. In patients who develop obstruction, Gattex
`should be temporarily discontinued pending further clinical
`evaluation and management.
`• Biliary and pancreatic disease. Patients should undergo laboratory
`assessment (bilirubin, alkaline phosphatase, lipase, amylase)
`before starting Gattex. Subsequent laboratory tests should be
`done every 6 months. If clinically meaningful changes are seen,
`further evaluation is recommended including imaging, and
`continued treatment with Gattex should be reassessed.
`• Fluid overload. There is a potential for fluid overload while on
`Gattex. If fluid overload occurs, especially in patients with
`cardiovascular disease, parenteral support should be appropriately
`adjusted, and Gattex treatment reassessed.
`
`Prescribers should select the appropriate patients to receive Gattex in
`accordance with the approved prescribing information, discuss the
`benefits and risks of Gattex with patients, and monitor patients as
`specified in the approved prescribing information and report adverse
`events to NPS' Gattex information line at 1-855-5GATTEX (1-855-542-
`8839) or event/product complaint line at 1-855-215-5550.
`
`About NPS Pharmaceuticals
`
`NPS Pharmaceuticals is a biopharmaceutical company focused on
`bringing orphan products to patients with rare disorders and few, if any,
`therapeutic options. The company's lead product, Gattex® 0.05 mg/kg/d
`(Teduglutide [rDNA origin]) for Injection is FDA-approved for the
`treatment of adult patients with short bowel syndrome (SBS) who are
`dependent on parenteral support. NPS is also developing Natpara®
`(rhPTH[1-84]) for the treatment of adult hypoparathyroidism and
`expects to submit its Biologic License Application (BLA) to the FDA in
`mid-2013.
`
`NPS's earlier stage pipeline includes two calcilytic compounds, NPSP790
`and NPSP795, with potential application in rare disorders involving
`increased calcium receptor activity, such as autosomal dominant
`hypocalcemia with hypercalciuria (ADHH). NPS complements its
`proprietary programs with a royalty-based portfolio of products and
`
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`product candidates that includes agreements with Amgen,
`GlaxoSmithKline, Janssen Pharmaceuticals, Kyowa Hakko Kirin, and
`Takeda GmbH.
`
`"NPS," "NPS Pharmaceuticals," "Gattex," and "Natpara" are the
`company's trademarks. All other trademarks, trade names or service
`marks appearing in this press release are the property of their
`respective owners.
`
`Statements made in this press release, which are not historical in
`nature, constitute forward-looking statements for purposes of the safe
`harbor provided by the Private Securities Litigation Reform Act of 1995.
`These statements are based on the company's current expectations and
`beliefs and are subject to a number of factors and uncertainties that
`could cause actual results to differ materially from those described in
`the forward-looking statements. Forward looking statements include,
`but are not limited to, statements concerning our future commercial
`performance. Risks associated to the company's business include, but
`are not limited to, the risks associated with any failure by the company
`to successfully commercialize Gattex, including the risk that physicians
`and patients may not see the advantages of Gattex and may therefore
`be reluctant to utilize the product, the risk that private and public
`payers may be reluctant to cover or provide reimbursement for Gattex,
`as well as other risk factors described in the company's periodic filings
`with the U.S. Securities and Exchange Commission, including its Annual
`Report on Form 10-K and Form 10-Qs. All information in this press
`release is as of the date of this release and NPS undertakes no duty to
`update this information, whether as a result of new information, future
`events or otherwise.
`
`SOURCE NPS Pharmaceuticals
`
`Susan M. Mesco, NPS Pharmaceuticals, Inc., +1-908-450-5516,
`smesco@npsp.com, www.npsp.com
`
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