Yesterday, Proteostasis Therapeutics, Inc. (PTI) announced it had raised $37 million in new funding that will be used to develop its lead product candidate for the treatment of cystic fibrosis (CF) in human clinical studies as well as expansion of its therapeutic portfolio.
PTI is a US-based pharmaceutical company focusing its development efforts on new therapeutics addressing diseases caused by protein processing defects, such as cystic fibrosis (CF). CF results from physiological mutations in the membrane of protein regulators — the cystic fibrosis transmembrane conductance regulator (CFTR). Some of the symptoms associated with the disease include frequent lung infections, persistent coughing, salty-tasting skin, shortness breath and male infertility.
PTI has worked to develop a new class of CFTR regulators that are able to amplify the effect of the common CFTR modulators, such as potentiators and corrector amplifiers, for the treatment of CF. CFTR amplifiers are a novel drug class that is able to enhance the effect of potentiators and correctors. In preclinical studies, PTI has indicated that the amplifier that the company is currently developing was effective across several CFTR mutation classes. These previous results have led PTI to begin developing a broad-acting combination therapy that the company believes will be able to treat most of the genetic mutations associated with cystic fibrosis.
PTI’s financing was made possible by a mezzanine financing strategy in which the investors have the rights to convert to an ownership or equity interest in the company if the investment is not paid back in time. The lead investors in this project are Cormorant Asset Management and Rock Springs Capital Management but names such as New Enterprise Associates, Elan Science One, HealthCare Ventures, Fidelity Biosciences, Novartis Bioventures, Novartis Venture Fund, and Sanofi-Genzyme BioVentures also participated in the financing. Leerink Partners LLC acted as the exclusive placement agent to the company for the offering.
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“Cormorant Global Healthcare Fund is pleased to participate in the mezzanine equity financing of PTI. We believe PTI’s pipeline of drug candidates has the potential to provide significant benefits to a broad group of cystic fibrosis patients. We are excited to support this experienced management team as they advance a series of compounds through clinical trials. We are happy to join such an innovation-focused investor group and to help PTI achieve its mission to improve the lives of patients with cystic fibrosis,” stated Bihua Chen, Managing Member and Chief Executive Officer of Cormorant Asset Management.
When asked about the successful fundraising effort for the company’s experimental CF therapy, Meenu Chhabra, PTI’s President and Chief Executive Officer added, “As we advance our programs into clinical development, PTI is in a strong position to improve upon present treatments for patients with cystic fibrosis. We are confident that we will continue to build on our promising preclinical results to advance our lead product in to the clinic.”