Renasant Bio secures $55M for ADPKD drug development

In the quest to develop effective treatments for autosomal dominant polycystic kidney disease (ADPKD), the pharmaceutical industry has experienced a mix of progress and setbacks. ADPKD is a rare kidney condition characterized by the growth of cysts within the kidneys, leading to diminished organ function. Currently, patients have limited treatment options, with Jynarque, a drug approved by U.S. regulators in 2018, being one of the few viable options available to slow the disease's progression. Sanofi’s attempt to develop a drug, venglustat, ceased in 2021 due to unsatisfactory clinical trial results.

Despite these challenges, the disease remains a focal point for drug developers. In a notable development, Novartis recently invested $800 million to acquire Regulus Therapeutics and its experimental treatment, farabursen, which has shown potential in clinical efficacy and safety. Furthermore, a fresh endeavor is underway with the launch of a new biotechnology company, Renasant Bio, which is setting its sights on creating another treatment option for ADPKD.

Renasant Bio has secured $54.5 million in seed funding from investors such as 5AM Ventures, Atlas Venture, OrbiMed, and Qiming Venture Partners USA. The company emerged from the innovative efforts of Jeremy Reiter and Markus Delling, University of California, San Francisco professors, who collaborated with 5AM Ventures after presenting their research insights. The company is led by Emily Conley, a former scientist at Stanford University and previous CEO of Federation Bio. She also oversaw business development at 23AndMe, a genetic testing firm.

Renasant Bio draws inspiration from drug development in cystic fibrosis, another genetic disorder. Both cystic fibrosis and ADPKD involve issues with ion channels, albeit in different organs. Drugs like Trikafta from Vertex Pharmaceuticals have been successful in treating cystic fibrosis by correcting and enhancing the activity of these channels. Renasant Bio aims to adopt a similar approach for ADPKD by developing treatments known as correctors and potentiators, which help proteins fold properly and keep channels open, respectively. According to Conley, if the company can fix the misfolded proteins associated with ADPKD, it could lead to significant improvements in patient health outcomes.

Currently, Renasant Bio's lead drug candidate is in the preclinical testing phase. This candidate is a small molecule corrector that the company hopes will be effective across the spectrum of mutations that cause ADPKD. In addition, the company is developing a potentiator treatment that could be used in combination with the corrector, aiming to prevent the formation and growth of kidney cysts. Clinical testing is anticipated to commence within the next few years.

Guiding Renasant's scientific endeavors is Gus Gustafson, who brings experience from major pharmaceutical companies such as Johnson & Johnson and Merck & Co. The board of directors is chaired by Natalie Holles, the former CEO of Third Harmonic Bio, and includes other notable figures like Charlotte McKee from Sionna Therapeutics. Investors like 5AM Ventures, which played a crucial role in incubating Renasant, express confidence in the company's scientific strategy and experienced team.

Estimates suggest that ADPKD affects around 12 million individuals worldwide. Alongside Renasant, companies like Vertex and Novartis are also pursuing drug developments for this condition, indicating a growing interest in finding effective therapies for ADPKD.