Second Quarter and Recent Highlights
- Global phase 3 study expanding. Patient enrollment is advancing in the company's global phase 3 study of ARIKAYCE™ (liposomal amikacin for inhalation or LAI) in nontuberculous mycobacteria (NTM) lung disease (the "212" or CONVERT™ study). The company has secured health authority clearance in 10 countries with CONVERT study sites. In addition to the U.S., patient recruitment is underway in
Australia, Canada, Europe, and New Zealand. With strong interest among clinical investigators, Insmedis expanding the total number of CONVERT clinical sites to more than 100.
- Preliminary long-term data from phase 2 clinical study (112 study) support durability of ARIKAYCE treatment effect. One-year post-ARIKAYCE treatment follow-up data is now available for 14 of 23 patients who achieved culture conversion during the 196-day 112 study. Twelve of the 14 patients remained culture negative for NTM at their one-year follow-up visit. Cultures from the remaining two patients grew in liquid medium only and not on solid medium, which may represent contamination (false-positive) or a new infection rather than a relapse of the original infecting strain.
- EMA regulatory review progressing.
The European Medicine Agency's(EMA) review of the company's marketing authorization application (MAA) for ARIKAYCE is ongoing. Insmedhas received the EMA's 120-day questions and anticipates responding before the end of 2015.
- ATU approval secured in
Franceand first product shipped. The French National Agency for Medicines and Health Products Safety(ANSM) granted LAI a nominative Temporary Authorization for Use (Autorisation Temporaire d'Utilisation or ATU). Pursuant to this program, in July the company shipped product after receiving a request from a physician for a patient in France. Expanded access programs are generally intended to make products available to patients before they are approved or commercially available in accordance with local regulations.
- INS1009 advancing toward clinical development. The company is completing a number of IND-enabling preclinical studies and expects to submit its Investigational New Drug (IND) Application and initiate a phase 1 clinical study of INS1009 in the fourth quarter of 2015. INS1009 is the company's nebulized treprostinil prodrug that is being developed for pulmonary arterial hypertension (PAH).
- Pre-commercial and manufacturing activities advancing. NTM disease awareness campaigns are advancing in
Europeand the U.S. In addition, the build-out of additional third-party commercial-scale manufacturing capacity is proceeding according to plan and the company is on track to have two sources of ARIKAYCE supply established by year-end.
- Significantly strengthened balance sheet.
Insmedcompleted a successful equity offering in April raising net proceeds of $222.9 millionand finished the second quarter with $335 millionin cash. The company's cash operating expenses for the six months ended June 30, 2015were approximately $46 million.
- Management team enhancements. The company continues to make key additions to its leadership team, including the recent additions of
Ela Bochenek, vice president global compliance and George Georges, MD, vice president global medical affairs. Ms. Bochenek's prior experience includes NPS Pharmaceuticals, C.R. Bard, Schering-Plough, and Bristol-Myers Squibb. Dr. Georgespreviously served in numerous U.S. and global medical affairs leadership roles at InterMune, GlaxoSmithKline, and Sanofi.
"Throughout the first half of the year we made considerable progress advancing our stated corporate objectives each of which brings us closer to attaining our vision of building a self-sustaining biopharmaceutical company focused on rare diseases," said
For the second quarter of 2015,
Research and development expenses were
General and administrative expenses for the second quarter of 2015 were
Balance Sheet Highlights and Cash Guidance
As previously reported, the company will continue to invest in the following activities: (i) clinical development of ARIKAYCE, (ii) third-party manufacturing capacity, (iii) regulatory and pre-commercial initiatives for ARIKAYCE, and (iv) filing an Investigational New Drug Application and initiating a phase 1 clinical study of INS1009. As a result,
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ARIKAYCE is a form of the antibiotic amikacin, which is enclosed in nanocapsules of lipid called liposomes. This advanced pulmonary liposome technology prolongs the release of amikacin in the lungs while minimizing systemic exposure. The treatment uses biocompatible lipids endogenous to the lung that are formulated into small (0.3 micron), charge-neutral liposomes. ARIKAYCE is administered once-daily using an optimized, investigational eFlow® Nebulizer System manufactured by
About eFlow® Technology and PARI Pharma
ARIKAYCE is delivered by an investigational eFlow® Nebulizer System developed by PARI Pharma and optimized specifically for ARIKAYCE. The optimized device uses eFlow Technology to enable highly efficient aerosolization of medication including liposomal formulations via a vibrating, perforated membrane that includes thousands of laser-drilled holes. Compared with other nebulization technologies, eFlow Technology produces aerosols with a very high density of active drug, a precisely defined droplet size and a high proportion of respirable droplets delivered in the shortest possible period of time. eFlow Technology is not an ultrasonic nebulizer technology and is not a general purpose electronic aerosol generator nebulizer technology. Combined with its quiet mode of operation, small size, light weight and battery use, eFlow Technology reduces the burden of taking daily, inhaled treatments.
About Nontuberculous Mycobacteria
Nontuberculous mycobacteria are organisms found in the soil and water that can cause serious lung disease in susceptible individuals, for which there are currently limited effective treatments and no approved therapies in
NTM lung disease is often a chronic condition that can lead to progressive inflammation and lung damage, and is characterized by bronchiectasis and cavitary disease. NTM infections often require lengthy hospital stays for medical management. Treatment usually involves multi-drug regimens that can be poorly tolerated and have limited effectiveness, especially in patients with severe disease or in those who have failed prior treatment attempts. According to a company-sponsored patient chart study conducted by
INS1009 is the company's nebulized treprostinil prodrug for the treatment of pulmonary arterial hypertension (PAH), a chronic, life-threatening disorder characterized by abnormally high blood pressure in the arteries between the heart and lungs.
About Pulmonary Arterial Hypertension
Pulmonary arterial hypertension is a chronic, life-threatening form of pulmonary hypertension that is characterized by abnormally high blood pressure in the arteries between the heart and lungs. Pulmonary arteries carry blood from the heart to the lungs, where it picks up oxygen to be delivered throughout the body. In PAH, the pulmonary arteries constrict abnormally. This forces the heart to pump harder to maintain adequate blood flow, which causes blood pressure within the lungs to rise. Common early symptoms include shortness of breath, fatigue, weakness, chest pain and syncope (fainting), particularly during physical activity. PAH worsens over time and is life-threatening because the pressure in a patient's pulmonary arteries rises to dangerously high levels, which strains the heart and may lead to heart failure. The one-year mortality rate among patients with PAH is 15% despite currently available treatments. The cause of some cases of PAH is unknown and there is no cure. PAH is considered an orphan disease, afflicting 30,000 to 40,000 people in the U.S. and 200,000 people globally.
There are several prescription medications approved by the
This release contains forward-looking statements. Words, and variations of words, such as "intend," "expect," "will," "anticipate," "believe," "continue," "propose" and similar expressions are intended to identify forward-looking statements. Investors are cautioned that such statements in this release, including statements relating to the status, results and timing of clinical trials and clinical data, the anticipated benefits of Insmed's products, the anticipated timing of regulatory submissions, and the ability to obtain required regulatory approvals, bring products to market and successfully commercialize products constitute forward-looking statements that involve risks and uncertainties that could cause actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, without limitation, market conditions, failure or delay of European, Canadian,
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