AN2 Therapeutics Inc.’s decision to pause enrollment in the phase III portion of its phase II/III testing epetraborole in treatment-refractory Mycobacterium avium complex lung disease, citing potentially lower-than-expected efficacy from a blinded aggregate analysis of the phase II portion, left analysts and investors with little to do but speculate on the program’s viability going forward.
Wall Street’s hoped-for phase III derisking event from Insmed Inc. materialized, and shares of the firm (NASDAQ:INSM) closed Sept. 5 at $26.37, up $3.73, or 16.5%, on positive top-line results from the study called Arise with inhaled Arikayce (amikacin) in patients with newly diagnosed or recurrent nontuberculous mycobacterial lung infection by Mycobacterium avium complex (MAC) who had not started antibiotics. “We crushed it” on culture conversion with Arikayce, CEO William Lewis said. “We could not be happier about the results of this study. It exceeded all of our expectations on every front.”
A paper published May 3 in Nature Communications about a patient infected with Mycobacterium chelonae who was cured by way of bacteriophage treatment sparked more intrigue around how to attack the nontuberculous mycobacteria (NTM) group of bugs that cause lung infections.
Even as antimicrobial resistance is expected to continue to grow, the development of much-needed novel antibiotics and antifungals remains trapped in a catch-22 in which funding is available for early stage research but not necessarily for the translational work necessary to bring the drugs to market.
Shares of Bridgewater, N.J.-based Insmed Inc. (NASDAQ:INSM) closed at $28.88, up $8.34, or 40.6%, on positive top-line results from the global, randomized, double-blind placebo-controlled phase II study called Willow, testing INS-1007 once daily in adults with non-cystic fibrosis bronchiectasis (NCFBE).
Patient engagement has become more than a buzzword for the FDA and drug and device developers. But for payers, not so much. When valuing new drugs and devices, payers often undervalue or ignore what they may consider convenience updates, giving little to no consideration to the difference a seemingly minor improvement could make to patients debilitated by fatigue, pain, the burden of treatment and the burden of a disease itself.