In this section
"The interim results announced today from our ongoing INS-212 and INS-312 studies provide confirmation of the initial positive clinical data released in
The company previously announced that the global Phase 3 INS-212 study met its primary endpoint of culture conversion by Month 6 with statistical significance. Those results demonstrated that the addition of ALIS to GBT eliminated evidence of NTM lung disease caused by MAC in sputum by Month 6 in 29% of patients, compared to 9% of patients on GBT only (p < 0.0001). Serious treatment emergent adverse events (TEAEs) were similar between treatment arms.
Patients that achieved culture conversion by Month 6 in either arm of INS-212 continue in the INS-212 study for an additional 12 months of treatment plus a follow-up period off all therapy to assess durability. Patients in either arm of the INS-212 study who did not achieve culture conversion by Month 6 had the option to enroll in INS-312 at month 8, an extension study that is evaluating treatment with ALIS plus GBT for 12 months.
Interim Data from INS-312 Extension Study for Non-Converters from INS-212 Study
INS-312 is a 12-month extension study for patients who completed six months of treatment in the INS-212 study, but did not demonstrate culture conversion by Month 6. The trial is designed to evaluate: 1) the impact of longer term treatment with ALIS plus GBT and 2) the impact of the addition of ALIS to background GBT therapy. Under the study protocol, patients in the ALIS plus GBT arm of the INS-212 study receive an additional 12 months of ALIS plus GBT. Patients who crossed over from the GBT only arm of the INS-212 study receive 12 months of treatment of ALIS plus GBT.
In the INS-312 study, serious treatment emergent adverse events were similar to the previous findings announced in September of 2017. At this point in time, the dropout rate in INS-312 is 24% (n=39/163).
The Company plans to continue to monitor and evaluate patients throughout the duration of the INS-312 study.
Long-Term Durability Data from INS-212 Study
At this point in time, the overall dropout rate in INS-212 is 18% (n=60/336).
The Company plans to continue to monitor and evaluate patients throughout the duration of the INS-212 study and expects to report additional data in late 2018 or early 2019 when the INS-312 study has completed its full 12 months and the data has been analyzed pursuant to its statistical analysis plan as agreed with
"The new data announced today amplify the results previously released from the CONVERT study demonstrating the potential of ALIS to produce sputum conversion in difficult-to-treat MAC lung disease patients which is the first and most important goal for the successful treatment of MAC lung disease. Further, these new data provide evidence of durable MAC eradication in these patients," stated
Regulatory and Commercial Progress
- The Company remains on track to file its New Drug Application (NDA) for accelerated approval of ALIS with the
U.S. Food and Drug Administration(FDA) before the end of March. Insmedexpects to receive a six-month priority review from the FDA.
- The Company is accelerating its production efforts, including the construction of a larger capacity manufacturing facility at a contract manufacturing organization as well as initiation of manufacturing of commercial product supply from existing facilities.
- The Company announced the hiring of
John D. Sorianoas Chief Compliance Officer. John most recently served as Senior Vice President, Chief Compliance Officer for Celgene.
- The Company is hiring the field teams that will help to support the potential
U.S.product launch in late 2018, as well as senior leadership to direct and advance our Japanese opportunity.
- The Company opened a Japanese subsidiary, a requirement for regulatory approval in
Japan, and plans to reconfirm with the Pharmaceuticals and Medical Device Agency(PMDA) the approval path for ALIS in Japanbased on the clinical data. Insmedplans to file an application with the PMDA in late 2018 or early 2019.
"We continue to make solid progress in our regulatory and pre-commercial efforts focused on our first two target markets,
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About NTM Lung Disease
NTM lung disease is a rare and serious disorder associated with increased rates of morbidity and mortality. There is an increasing prevalence of lung disease caused by NTM, and we believe it is an emerging public health concern worldwide. Patients with NTM lung disease may experience a multitude of symptoms such as fever, weight loss, cough, lack of appetite, night sweats, blood in the sputum, and fatigue. Patients with NTM lung disease frequently require lengthy hospital stays to manage their condition. We are not aware of any approved inhaled therapies specifically indicated for refractory NTM lung disease caused by MAC in
The prevalence of human disease attributable to NTM has increased over the past two decades. In a decade long study (1997 to 2007), researchers found that the prevalence of NTM lung disease in the
ALIS is a novel, inhaled, once-daily formulation of amikacin that is in late-stage clinical development for adult patients with treatment-refractory NTM lung disease caused by MAC. Amikacin solution for parenteral administration is an established drug that has activity against a variety of NTM; however, its use is limited by the need to administer it intravenously and by toxicity to hearing, balance, and kidney function.
About CONVERT (INS-212) and INS-312
CONVERT is a randomized, open-label, global Phase 3 trial designed to confirm the culture conversion results seen in Insmed's Phase 2 clinical trial of ALIS in patients with refractory NTM lung disease caused by MAC. CONVERT is being conducted in 18 countries at more than 125 sites. The primary efficacy endpoint is the proportion of patients who achieve culture conversion at Month 6 in the ALIS plus GBT arm compared to the GBT-only arm. Patients who achieve culture conversion by Month 6 will continue in the CONVERT study for an additional 12 months of treatment following the first monthly negative sputum culture. Patients who do not culture convert have the option of enrolling in our INS-312 study. INS-312 is a single-arm open-label study where patients will receive ALIS plus GBT for 12 months.
This press release contains forward looking statements. "Forward-looking statements," as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a number of risks and uncertainties. Words herein such as "may," "will," "should," "could," "would," "expects," "plans," "anticipates," "believes," "estimates," "projects," "predicts," "intends," "potential," "continues," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) may identify forward-looking statements.
The forward-looking statements in this press release are based upon the Company's current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company's actual results, performance and achievements and the timing of certain events to differ materially from the results, performance, achievements or timing discussed, projected, anticipated or indicated in any forward-looking statements. Such risks, uncertainties and other factors include, among others: risks that the full six-month data from the CONVERT study or subsequent data from the remainder of the study's treatment and off-treatment phases will not be consistent with the top-line six-month results of the study; uncertainties in the research and development of our existing product candidates, including due to delays in data readouts, such as the full data from the CONVERT study, patient enrollment and retention or failure of our preclinical studies or clinical trials to satisfy pre-established endpoints, including secondary endpoints in the CONVERT study and endpoints in the CONVERT extension study (the 312 study); risks that subsequent data from the 312 study will not be consistent with the interim results of the study; failure to obtain, or delays in obtaining, regulatory approval from the
For additional information about the risks and uncertainties that may affect our business, please see the factors discussed in Item 1A, "Risk Factors," in the Company's Report on Form 10-Q for the quarter ending
The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date of this press release. The Company disclaims any obligation, except as specifically required by law and the rules of the