Takeda Pharmaceutical Company Limited

Takeda Receives Positive CHMP Opinion for Teduglutide (Revestive®) for Patients with Short Bowel Syndrome

Osaka, Japan And Bedminster, New Jersey (ots/PRNewswire) -

        - European CHMP adopts positive opinion for teduglutide (Revestive(R)) for
          patients with short bowel syndrome (SBS)
        - SBS is a rare and highly disabling condition that impacts patients' quality of
          life and can lead to serious life-threatening complications
        - Teduglutide has received orphan drug designation for the treatment of SBS from
          the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) 

Takeda Pharmaceutical Company Limited and NPS Pharmaceuticals, Inc. , jointly announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product teduglutide (tradename in Europe: Revestive(R)) as a once-daily treatment for adult patients with short bowel syndrome (SBS). The marketing authorisation application was submitted in March 2011.

SBS is a rare and debilitating disease characterised by the body's severely impaired ability to absorb nutrients and fluids through the gastrointestinal tract in people who have had a significant portion of their small intestine removed. SBS typically arises after extensive surgical resection of the bowel due to Crohn's disease, ischemia or other conditions. Many patients with SBS depend on chronic parenteral nutrition (PN) and/or intravenous (IV) fluids to survive. There are currently no therapies approved for the treatment of SBS in Europe.

Teduglutide (Revestive(R)) is a novel, recombinant analogue of human glucagon-like peptide 2 (GLP-2), a naturally occurring protein involved in the rehabilitation of the intestinal lining. Teduglutide has received orphan drug designation for the treatment of SBS from the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA).

"Short bowel syndrome patients suffer from malnutrition and diarrhoea, and often parenteral nutrition is necessary to maintain life," said Professor Palle Bekker Jeppesen, M.D., Ph.D, Department of Medical Gastroenterology, Rigshospitalet, University Hospital of Copenhagen, Denmark. "Revestive is a new, unique and important treatment option for our patients and is adding important value to the limited treatment armamentarium."

"We welcome the positive opinion from the CHMP for teduglutide. This is good news for patients with SBS," said Trevor Smith, Head of Commercial Operations, Europe & Canada, of Takeda.

"We are pleased with the Committee's recommendation, which brings us closer to our goal of making teduglutide available in Europe for patients with short bowel syndrome," said Francois Nader, MD, President and Chief Executive Officer of NPS Pharmaceuticals. "Teduglutide represents an important treatment advance that could significantly reduce or even eliminate parenteral nutrition support for patients with short bowel syndrome. We congratulate our partner Takeda on receiving this positive opinion and look forward to supporting their efforts to bring this much-needed therapy to patients."

The CHMP opinion was based upon data from STEPS, the pivotal phase-3 double-blind, placebo-controlled study in patients with SBS, who required parenteral nutrition; 43-patients were randomised to a subcutaneous 0.05-mg/kg/day dose of teduglutide and 43-patients to placebo for up to 24-weeks.[1]

The proportion of teduglutide treated subjects achieving a 20% to 100% reduction of parenteral nutrition at Week-20 and 24 was statistically significantly different from placebo, (63% versus 30%, p=0.002). Treatment with teduglutide resulted in a 4.4-l/week reduction in parenteral nutrition requirements versus 2.3-l/week for placebo at 24-weeks (p<0.001). 21 patients treated with teduglutide (54%) versus nine on placebo (23%) achieved at least a one day reduction in parenteral nutrition administration (p=0.005) Teduglutide was well-tolerated in the doses, frequency, and duration of treatment used in this study.[1]

In another phase-3 double-blind, placebo-controlled study in patients with SBS, who required parenteral nutrition, patients subcutaneously received a 0.05-mg/kg/day dose (n-=-35), a 0.10-mg/kg/day dose (n-=-32) of teduglutide or placebo (n-=-16) for up to 24-weeks.[2]

The primary efficacy analysis of the study results showed no statistically significant difference between the group on teduglutide 0.10-mg/kg/day and the placebo group, while the proportion of subjects receiving the recommended teduglutide dose of 0.05-mg/kg/day achieving at least a 20% reduction of parenteral nutrition at Week-20 and-24 was statistically significantly different versus placebo (46% versus 6%, p<0.01). Treatment with teduglutide resulted in a 2.5-l/week reduction in parenteral nutrition requirements versus 0.9-l/week for placebo at 24-weeks (p=0.08). Teduglutide was well tolerated at the 0.05 mg/kg daily dose for the duration of treatment of adult SBS subjects.[2]

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 diarrhoea, dehydration, fatigue, osteopenia, and weight loss due to the reduced intestinal capacity to absorb nutrients, water, and electrolytes. The usual treatment for short bowel syndrome 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 short bowel syndrome patients, it does not improve the body's own ability to absorb nutrients. PN is also 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 a poor quality of life with difficulty sleeping, frequent urination and loss of independence.

About Teduglutide (Revestive(R))

Teduglutide is a novel, recombinant analogue of human glucagon-like peptide 2, a protein involved in the rehabilitation of the intestinal lining. It has been developed to reduce dependence on parenteral nutrition (PN) in adult patients with short bowel syndrome (SBS). Two phase III studies of teduglutide demonstrated a favorable safety profile and significant reductions in mean PN volume from baseline to end of treatment. In addition some patients were able to be weaned off PN and continue their life without parenteral support

Teduglutide has received orphan drug designation for the treatment of SBS from the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA).

In 2007, NPS Pharmaceuticals, Inc. , a specialty pharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, granted Nycomed the rights to develop and commercialize teduglutide outside the United States, Canada and Mexico and Israel. NPS retains all rights to teduglutide in North America. NPS submitted a new drug application for teduglutide to the U.S. Food and Drug Administration (FDA) in November 2011. Teduglutide is known as Gattex(R) in the U.S.

References

        1) Publication of STEPS is in preparation. First results were report at the
          AGA 2011 in an abstract: Jeppesen PB, Pertkiewicz M, Seidner DL, O'Keefe S, Heinze H,
          Joelsson B: Teduglutide, a novel analogue of Glucagon-like Peptide 2 (GLP-2), is
          effective and safe in reducing parenteral support volume in short bowel
          syndrome-intestinal failure subjects: Results from a 24-week, placebo-controlled phase
          3 trial (STEPS), Gastroenterology 2011; 140 (5), Supplement 1, S146
        2) Jeppesen PB, Gilroy R, Pertkiewicz M, Allard JP, Messing B, O'Keefe SJ.
          Randomised placebo-controlled trial of teduglutide in reducing parenteral nutrition
          and/or intravenous fluid requirements in patients with short bowel syndrome. Gut. 2011
          Jul;60(7):902-14. Epub 2011 Feb 11. 

About Takeda Pharmaceuticals Company Limited

Located in Osaka, Japan, Takeda is a research-based global company with its main focus on pharmaceuticals. As the largest pharmaceutical company in Japan and one of the global leaders of the industry, Takeda is committed to strive towards better health for patients worldwide through leading innovation in medicine. Additional information about Takeda is available through its corporate website, http://www.takeda.com.

Additional information about Takeda is available through its corporate website, http://www.takeda.com.

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. NPS is advancing two late-stage registration programs. A New Drug Application is undergoing FDA review for Gattex(R) (teduglutide) as a treatment for adult short bowel syndrome (SBS) and a Phase 3 registration study has been completed for Natpara(TM) (recombinant human parathyroid hormone (rhPTH [1-84]) in adult hypoparathyroidism. NPS' 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 product candidates that includes agreements with Amgen, GlaxoSmithKline, Janssen Pharmaceuticals, Kyowa Hakko Kirin, and Nycomed (acquired by Takeda Pharmaceutical Company Limited).

Forward-looking statements

This press release contains forward-looking statements. Forward-looking statements include statements regarding Takeda's and NPS' plans, outlook, strategies, results for the future, and other statements that are not descriptions of historical facts. Forward-looking statements may be identified by the use of forward-looking words such as "may," "believe," "will," "expect," "project," "estimate," "should," "anticipate," "plan," "assume," "continue," "seek," "pro forma," "potential," "target," "forecast," "guidance," "outlook" or "intend" or other similar words or expressions of the negative thereof. Forward-looking statements are based on estimates and assumptions made by management that are believed to be reasonable, though they are inherently uncertain and difficult to predict. Investors are cautioned not to unduly rely on such forward-looking statements.

Forward-looking statements involve risks and uncertainties that could cause actual results or experience to differ materially from that expressed or implied by the forward-looking statements. Some of these risks and uncertainties include, but are not limited to, (1) the economic circumstances surrounding Takeda's and NPS' business, including general economic conditions in Japan, the United States and worldwide; (2) competitive pressures and developments; (3) applicable laws and regulations; (4) the success or failure of product development programs; (5) actions of regulatory authorities and the timing thereof; (6) changes in exchange rates; (7) claims or concerns regarding the safety or efficacy of marketed products or product candidates in development; and (8) integration activities with acquired companies.

The forward-looking statements contained in this press release speak only as of the date of this press release, and Takeda and NPS undertake no obligation to revise or update any forward-looking statements to reflect new information, future events or circumstances after the date of the forward-looking statement. If Takeda and NPS do update or correct one or more of these statements, investors and others should not conclude that Takeda will make additional updates or corrections.

Contact:

Media Contacts, Takeda Pharmaceutical Company Limited,

Public Relations and Investor Relations, +81-3-3278-2037;

European media, Tobias Cottmann, +41445551510,
tobias.cottmann@takeda.com;
NPS Pharmaceuticals, Inc.,

Susan Mesco, +1 908-450-5516, smesco@npsp.com



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