Insmed Provides Corporate Update
- Company Sets Current ARIKACE® Development Priorities -
- Insmed Projects Cash Sufficient to Generate Top-Line Data from Three Priority Studies -
MONMOUTH JUNCTION, N.J., May 30, 2012 /PRNewswire/ -- Insmed Incorporated (Nasdaq CM: INSM), a biopharmaceutical company, today provided a corporate update, including the Company's current strategic development priorities for ARIKACE® (liposomal amikacin for inhalation).
"Insmed has determined that its current resources will be applied towards the CLEAR-108 phase 3 European and Canadian registrational study of ARIKACE in patients with Cystic Fibrosis (CF)-related Pseudomonas aeruginosa lung infections, the TARGET-NTM phase 2 trial of ARIKACE in patients with non-tuberculous mycobacteria (NTM) lung disease and the 9-month dog toxicity study," said Timothy Whitten, President and CEO of Insmed.
"We believe this ARIKACE development program prioritizes the most compelling near-term opportunities for Insmed and its shareholders," continued Mr. Whitten. "The CLEAR-108 trial provides Insmed with the earliest opportunity to garner critical phase 3 ARIKACE data in a broad cystic fibrosis patient population while generating important information on the efficacy and safety of ARIKACE as compared to the current standard of care. In NTM, ARIKACE has the opportunity to become a part of standard therapy for a rapidly growing chronic disease that has a high unmet medical need with limited treatment options. We project our current cash position to be sufficient to generate top-line data from the three prioritized ARIKACE studies."
"Insmed believes that ARIKACE has the potential to address important unmet medical needs in CF and NTM, two key orphan indications with combined global market opportunity of up to $1 billion," concluded Mr. Whitten.
CLinical Evaluation of ARIKACE (CLEAR-108) Phase 3 CF Study
Randomized, phase 3 trial comparing ARIKACE 560 mg, delivered once daily via an optimized, investigational eFlow® Nebulizer System (PARI Pharma GmbH), to twice daily TOBI®(1) 300 mg (inhaled tobramycin solution), a commercially available inhaled antibiotic delivered via the PARI LC® Plus nebulizer
Trial is underway in Europe and Canada and will include approximately 300 patients
Primary endpoint is change in pulmonary function (FEV-1) measured after three 28 day on-treatment and three 28 day off-treatment cycles (approximately six months)
Key secondary endpoint is time to pulmonary exacerbation
Top-line data expected in mid-2013
The study design has been agreed upon by Insmed and the European Medicines Agency. The study's Principal Investigator is Diana Bilton, M.D., Director of Adult CF Centre at the Royal Brompton Hospital in London, England. Eligible patients will have the option to participate in a longer-term open-label multi-cycle safety study, called CLEAR-110.
CF is a rare, life-threatening genetic disease affecting approximately 70,000 children and adults worldwide. Today, the median predicted age of survival for a person with CF is approximately 38 years, but the median age of death remains in the mid-20's. More than half of all CF patients have acquired Pseudomonas lung infections by age 18 and receive extensive and often chronic antibiotic treatments. Antibiotics delivered via inhalation have become part of standard treatment for CF patients with Pseudomonas lung infections. However, due to the thick sticky mucous these patients produce in their lungs, CF patients seldom clear the Pseudomonas, and they become chronically infected. This results in a continuous decline in lung function, despite all currently available antibiotic treatments.
Treatment with ARIKACE to Realize Greater Efficacy Trial (TARGET-NTM) Phase 2 Clinical Trial
Randomized, placebo-controlled study of ARIKACE in approximately 100 adult patients that will include two of the most common species of NTM that cause lung disease - Mycobacterium avium complex (MAC) and Mycobacterium abscessus
NTM culture positive patients will continue with their antibiotic regimen, and receive additionally either ARIKACE 560 mg, delivered once daily via an optimized, investigational eFlow Nebulizer System, or placebo, once daily
Primary efficacy endpoint will be change in mycobacterial density from baseline to the end of 84 days of treatment, which is the end of the randomized portion of the trial
At the conclusion of the randomized portion of the study, eligible patients may receive ARIKACE 560 mg once daily for an additional 84 days in an open-label design. Open-label means patients will know they are receiving ARIKACE.
Patient enrollment currently expected to begin in mid-2012, with top-line results projected in the fourth quarter of 2013
The clinical trial design has been agreed upon by Insmed and the U.S. Food and Drug Administration, and several sites have recently begun screening patients. The Principal Investigator of the study is Kenneth N. Olivier, M.D., M.P.H., staff pulmonologist in the Laboratory of Clinical Infectious Diseases at the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health.
According to a recent company sponsored patient chart study conducted by Clarity Pharma Research, approximately 50,000 patients suffering from NTM lung disease visited physician offices in the U.S. during 2011. More than half of these patients were treated with antibiotics for NTM.
MAC and Mycobacterium abscessus account for the vast majority of NTM lung disease, with combined prevalence rates reported from 75 percent to over 85 percent in the U.S.
U.S. CF Status
Insmed will defer plans to initiate a phase 3 study of ARIKACE in the U.S. for CF patients until the Company reviews top-line results from CLEAR-108, as the data generated from CLEAR-108 could provide additional clarity on the scope, design and conduct of a U.S. CF phase 3 clinical trial. In addition, the deferral of a U.S. CF phase 3 clinical trial allows Insmed to focus its current capital resources on the prioritized ARIKACE clinical studies, CLEAR-108 (together with the follow-on CLEAR-110) and TARGET-NTM, as well as the dog toxicity study, which recently started and is expected to generate top-line results in the second quarter of 2013.