Insmed (INSM) is surging today (+123%) on positive clinical news for its lead product candidate. In case you're not familiar, Insmed is a biopharma focused on rare diseases. Its lead product candidate is ALIS for adult patients with treatment refractory NTM lung disease caused by MAC, which is a rare and often chronic infection that is capable of causing irreversible lung damage and can be fatal. Insmed is not aware of any approved inhaled therapies specifically indicated for refractory NTM lung disease caused by MAC in North America, Japan or Europe.
What makes ALIS is that it's an inhaled, once daily formulation of amikacin, which is an established drug that has activity against a variety of NTM. The problem is that its use is limited by the need to administer it intravenously and by toxicity to hearing, balance, and kidney function. Insmed's technology uses charge neutral liposomes to deliver amikacin directly to the lung where it is taken up by the lung macrophages where the NTM infection resides. This prolongs the release of amikacin in the lungs while minimizing systemic exposure thereby offering the potential for decreased systemic toxicities.
ALIS's ability to deliver high levels of amikacin directly to the lung distinguishes it from intravenous amikacin. ALIS is administered once daily using an optimized, investigational eFlow Nebulizer System manufactured by PARI Pharma, a portable aerosol delivery system.
Insmed's earlier-stage clinical pipeline includes INS1007, a novel oral reversible inhibitor of dipeptidyl peptidase 1 with therapeutic potential in non-cystic fibrosis bronchiectasis, and INS1009, an inhaled nanoparticle formulation of a treprostinil prodrug that may offer a differentiated product profile for rare pulmonary disorders, including pulmonary arterial hypertension.
Turning to today's news, Insmed announced top-line data from its Phase 3 CONVERT study. The study met its primary endpoint of culture conversion by Month 6 with statistical and clinical significance. The study demonstrated that the addition of ALIS to guideline-based therapy (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 alone.
Insmed considers these compelling top-line data to be a remarkable accomplishment in a rare disease state with no currently approved therapies. The company says it's particularly encouraged by the consistency of these data when compared with its Phase 2 study results, and looks forward to additional data as the CONVERT study continues over the next two years.
The company believes this marks an important advance in its quest to bring a safe and effective treatment to patients who suffer from NTM lung disease caused by MAC. This represents the first ever global Phase 3 study in patients with NTM, a rare, progressive and destructive infection that is associated with irreversible lung damage and increased rates of mortality.
The current guideline-based therapy to which the study was compared is not approved for the treatment of this disease, but is generally regarded as the best available option for these patients. On the other hand, ALIS delivers high levels of an aminoglycoside directly to the lung macrophages and pulmonary tissue where the infection resides.
In sum, the stock is surging today on the news so investors are clearly happy with the results. Based on the size of the move, it appears this result caught investors off guard. Looking ahead, Insmed plans to pursue accelerated approval of ALIS under subpart H, which will be reviewed by the Division of Anti-Infective Products. The FDA previously granted this product breakthrough therapy designation and fast track status and designated ALIS as a qualified infectious disease product (QIDP).