Pancreatic Insufficiency - Altus Pharmaceuticals Plans To Submit New Drug Application For Trizytek Approval

Main Category: Endocrinology
Also Included In: GastroIntestinal / Gastroenterology;  Regulatory Affairs / Drug Approvals
Article Date: 18 Nov 2008 - 9:00 PDT

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Altus Pharmaceuticals Inc. (NASDAQ: ALTU) announced today that it is reaffirming its plan to submit a new drug application (NDA) for Trizytek TM (liprotamase) in the first-half of 2009. Trizytek is a non-porcine derived enzyme replacement therapy for patients with pancreatic insufficiency.

Based on a positive FDA response to Altus' pre-NDA meeting submission material, Altus and the FDA have agreed that the Trizytek clinical development program supports submission of a license application for Trizytek. In the pre-NDA meeting package, Altus provided the FDA comprehensive data from its completed Phase 3 efficacy and Phase 2 studies as well as interim safety and health-outcomes data from the ongoing Phase 3 studies to evaluate the long-term safety of Trizytek over one year of open-label treatment in cystic fibrosis and chronic pancreatitis patients with exocrine pancreatic insufficiency. As a result, the November pre-NDA meeting for Trizytek is no longer necessary and Altus' previously stated timelines for the Trizytek NDA submission remain unchanged.

"With this communication from the FDA, we are reaffirming our goal of filing a new drug application for Trizytek in the first half of 2009. We are focusing our resources on moving all aspects of the Trizytek program forward, including the ongoing long-term safety study, which is on track to be completed in the first half of 2009," stated Dr. Georges Gemayel, President and Chief Executive Officer of Altus Pharmaceuticals. "The outcome from our discussions with the FDA only strengthens our belief in the clinical benefit that Trizytek will bring to patients who require pancreatic enzyme replacement therapy. In addition, completing a corporate collaboration around Trizytek is key to our plan going forward and we believe this information will be an important part of the evaluation of the program by any potential partner."

Altus' Phase 3 Trizytek efficacy trial in patients with cystic fibrosis (CF) successfully met its primary endpoint of improvement in fat absorption with statistical significance. In August 2008, the Company released top-line results from its 163 patient, double-blind, placebo-controlled trial of Trizytek, which is a stable and pure combination of three active enzymes in a fixed-ratio that is designed to improve fat, protein and carbohydrate absorption in pancreatic insufficient individuals. Trizytek was well-tolerated and had a favorable safety profile in the trial. There were no serious adverse events attributed to the Trizytek treatment. Altus' Trizytek Phase 3 clinical program is the largest ever conducted to evaluate the efficacy and safety of pancreatic enzyme replacement therapy in cystic fibrosis patients.

About TrizytekTM (liprotamase)

Trizytek has the potential to be the first porcine-free enzyme replacement therapy for patients with pancreatic insufficiency. Pancreatic insufficiency is a condition that affects most cystic fibrosis patients, as well as many patients with chronic pancreatitis. In these diseases, a deficiency of pancreatic enzymes causes poor absorption of essential nutrients, which often leads to malnutrition, impaired growth and reduced survival. Trizytek is intended to replace missing digestive enzymes with one capsule per-meal to promote and maintain proper digestion and growth in affected patients. Altus is developing Trizytek to enhance health outcomes by offering significant patient advantages such as improved and more consistent dosing that we expect will drive better long-term compliance. Utilizing recombinant technology, Trizytek is manufactured by blending three drug substance enzymes: lipase, protease and amylase. This consistent and pure enzyme combination is designed to improve fat, protein and carbohydrate absorption in pancreatic insufficient individuals.

About Altus Pharmaceuticals Inc.

Altus Pharmaceuticals, headquartered in Waltham, MA, is a biopharmaceutical company focused on the development and commercialization of oral and injectable protein therapeutics for patients with gastrointestinal and metabolic disorders. The Company is listed on the Nasdaq Global Market under the symbol ALTU. http://www.altus.com

Safe Harbor Statement
The statements in this press release regarding the filing of an NDA with the FDA for Trizytek and its implications for the future development of Trizytek, our ability to file an NDA and the timing of any filing of the NDA for Trizytek, and our ability to enter into a corporate collaboration for Trizytek are forward-looking statements that are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. In particular, these statements are subject to the risk that we may be unable to file the NDA in the first half of 2009 and that the FDA may conclude that our NDA filing, when submitted, is not complete or adequate. In addition, our ability to file an NDA with the FDA for Trizytek will also depend on our ability to either enter into a corporate collaboration for Trizytek, which may not occur within the timeframe that we require or at all, or commit sufficient financial resources to the project, which resources may be unavailable to us or may be deployed for other purposes. There can be no assurance that Trizytek will ever receive regulatory approval or be successfully commercialized. Other risks and uncertainties that could cause actual results to differ materially from those expressed or implied by these forward-looking statements include those detailed from time to time in the Company's periodic reports filed with the Securities and Exchange Commission, including the Company's current reports on Form 8-K, quarterly reports on Form 10-Q and annual report on Form 10-K, particularly the discussion under the caption "Item 1A, Risk Factors" in the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2008, which was filed with the Securities and Exchange Commission on August 5, 2008. The forward-looking statements in this press release are qualified by these risk factors. We assume no obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.

Altus and the Altus logo are the registered trademarks of Altus Pharmaceuticals. Trizytek and DIGEST Trials are trademarks of Altus Pharmaceuticals.