Promising Cystic Fibrosis Therapy ARIKACE From Insmed To Treat Pseudomonas Aeruginosa Reported as Well-Tolerated In Two-Year, Open-Label Extension Study

While Cystic Fibrosis news is never in short supply, often times, many clinical trials currently underway to test experimental CF therapies are underreported by the media, who instead seek to cover fundraising and human interest stories about the disease. While these types of news articles are indeed important to the cystic fibrosis community, compelling, new data from investigational CF drugs that could make a world of difference for the cystic fibrosis patient population are often missed.

A recent example of this is a clinical trial from Insmed Incorporated, which posted data in mid-February that went largely unreported in the news. Insmed is a drug development company working to develop an inhaled anti-infective therapy in order to treat patients with deadly lung infections in life-threatening orphan diseases such as cystic fibrosis.

In their press release, Insmed reported an update on the company’s two-year, open-label extension study of ARIKACE (antibiotic amikacin), as a treatment for Pseudomonas aeruginosa in CF patients.  The report includes information about longer-term safety and durability of effect for the therapy. The data indicates that ninety-eight patients had completed the first 12 month (six cycles) of the CLEAR-110 extension study at the time of the report.  The data was collected as part of the study’s planned data safety monitoring board (DSMB) review of the study, dubbed “CLEAR-110,” and concluded that ARIKACE was well tolerated among study participants, and that investigators observed a sustained improvement from baseline level in Forced Expiratory Volume in One Second (FEV1). The participants also experienced a sustained reduction in density of Pa sputum while taking the therapy.

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An extension in the ARIKACE study for eligible patients from the open-label, multi center, randomized Phase 3 CLEAR-108 clinical trial gives them the option to participate in a two-year, open-label, multi-cycle extension research to evaluate longer-term safety and tolerability of ARIKACE in cystic fibrosis patients.  Seventy-seven percent of the patients completed the randomized portion of Phase 3 trial and received at least one dose of the drug.

Thus far, data shows that patients receiving ARIKACE for six cycles (12 months) in the extension saw an increase in relative change in FEV1, which is sustained during both on-treatment and off-treatment months.  The researchers report that ARIKACE was well tolerated over the 12 month period with side effects being consistent with those expected in a population of cystic fibrosis patients receiving inhaled medications.

According to Renu Gupta, MD, FAAP, Executive Vice President Development and Chief Medical Officer of Insmed, “As we announced on July 1, 2013, the Phase 3 study of ARIKACE achieved its primary endpoint and demonstrated that ARIKACE administered once a day is non-inferior to the standard of care which is administered twice a day. This longer-term study demonstrates our continued commitment to the CF patients and to gathering longer term data to clarify the safety and efficacy profile of our drug candidate. The data we are reporting today are consistent with the findings from our earlier longer term study of ARIKACE and we believe further strengthen the clinical data packages we plan to submit to European and Canadian regulatory authorities later this year.”

Will Lewis, President and Chief Executive Officer of Insmed, notes, “We are especially pleased that these data demonstrated that patients taking ARIKACE actually saw their FEV1 levels remain above baseline on average. In addition we continue to see reduction of bacterial density and consistency in our overall safety profile. Further, we believe that once-daily administration of ARIKACE will support patient convenience and compliance.  With the results achieved in our Phase 3 trial, along with these positive interim data, we continue our preparation for regulatory filings with the European Medicines Agency and Health Canada, which remain on target for mid-year.”

Lewis adds, “I would like to recognize Dr. Gupta and our clinical team for designing and conducting such an important trial. This is the first trial to investigate the safety and efficacy of inhaled antibiotics in CF patients for a period of up to two years and it speaks to Insmed’s ongoing commitment to understanding the safe and effective use of this drug in this target population.”

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