Alnara Releases Results From Phase 3 Study For Liprotamase

Alnara Pharmaceuticals (Alnara) has reported results from an international, phase 3 open-label, long-term safety study demonstrating the safety and nutritional benefits of liprotamase. Reportedly, liprotamase is a novel pancreatic enzyme replacement therapy (PERT) for patients with cystic fibrosis (CF). The nutritional parameters measured during the study showed nutritional maintenance relative to the US population, which is a major finding for this historically challenged patient group.

Liprotamase is designed to treat maldigestion, malabsorption and malnutrition as a result of exocrine pancreatic insufficiency associated with CF, chronic pancreatitis (CP), pancreatic cancer, pancreatectomy and other pancreatic conditions.

The study has showed that nutritional status is maintained in CF patients with respect to nutritional measures such as height, weight and vitamin levels over 12 months relative to the healthy US population. This trial has completed the liprotamase new drug application (NDA) clinical development program which has included approximately 600 subjects in various efficacy and safety studies.

The open-label phase 3 safety study evaluated 214 patients, of which 145 CF patients, ages 7 and above, completed 12 months of treatment with liprotamase based on key nutritional parameters, including weight, height, Body Mass Index (BMI), and fat soluble vitamin absorption, which are important measures of PERT performance.

Reportedly, in the study the investigators found that 99% of CF patients ages 7 to 20 who received liprotamase for 12 months maintained or gained weight, while 96% of all CF patients who received liprotamase for 12 months maintained or gained weight. Patients who completed 12 months of treatment with liprotamase demonstrated that they maintained their nutritional status as assessed through a variety of nutritional parameters including BMI over the course of treatment relative to the US population.

The results suggested that Liprotamase has successfully completed two well-controlled, efficacy clinical trials and an additional long-term safety and nutritional study, representing the largest CF population studied in prospective clinical trials for a PERT. Liprotamase met the primary endpoint in both the phase 2 and phase 3 efficacy trials and demonstrated clinically significant improvement in fat and protein absorption.

Alnara has claimed that the results from these previously reported studies showed that liprotamase was well tolerated and in addition, improved other important clinical measures such as stool weight and frequency. Liprotamase has also been evaluated in a long-term nutritional tracking and safety study in patients with chronic pancreatitis or pancreatectomy.

Alexey Margolin, president and CEO of Alnara, said: “There is a clear need for new and improved treatments that address the serious issues related to pancreatic insufficiency and these positive and clinically relevant results demonstrate that liprotamase has the potential to make a meaningful difference in the management of patients with cystic fibrosis. We are delighted with the data from this study and appreciate the support we have received from the Cystic Fibrosis Foundation Therapeutics, the CF community and everyone who worked to complete the liprotamase clinical program. This marks another important milestone for Alnara and we are now focused on filing a new drug application with the FDA for liprotamase as soon as possible, as well as preparing to launch this important therapy.”

Robert Beall, president and CEO of the Cystic Fibrosis Foundation, said: “The long-term safety data on liprotamase has been eagerly awaited by the community since it captures a unique date set, and we are very pleased with the progress that has been made in developing this drug by Alnara. Our collaboration with Alnara on the development of liprotamase plays an important role in our goal of supporting the development of a non-porcine pancreatic enzyme replacement therapy for patients with cystic fibrosis.”