New OrbiMed-backed biotech aims to replicate Vertex's wildly successful cystic fibrosis pills in other diseases
A new biotech aiming to tackle protein misfolding has landed a substantial Series A from some blue-chip investors.
Congruence Therapeutics burst onto the scene Tuesday with a $50 million round, including funds from OrbiMed. The biotech will use the money to advance its platform and move some of its programs forward, CEO Clarissa Desjardins told Endpoints News.
Tuesday’s round was led by Amplitude Ventures and Fonds de solidarité FTQ, a public development fund in Quebec, Canada.
There are few other details about the company so far. Though Congruence says its platform utilizes machine learning and other mathematical and physics-based modeling systems to characterize protein misfolding disorders, the biotech is not yet naming the diseases it intends to research.
But the approach appears to resemble Vertex’s cystic fibrosis treatments, in which the company utilized small molecule drugs to target certain sites on the affected protein to restore sodium transport. Vertex’s drug was hugely successful, making the deadly genetic disease treatable for 90% of patients, and proved it was possible to treat genetic diseases with different mutations with a pill.
No one has been able to replicate the results in other diseases, however, though several groups are now trying. Notably, AbbVie is attempting this approach in cystic fibrosis as well, and the new Atlas-backed startup Rectify Pharmaceuticals launched last October with Vertex vets.
Desjardins did say that Congruence is also building out a library of the biophysical properties of various mutant proteins in hopes that a deeper analysis will point to ways of correcting them. The biotech is also keeping its disease research areas close to the vest for now.
After researchers identify what they believe could be the misfolded proteins, they can screen potential drug candidates to see which “can revert the mutant and make it congruent with the wild type, so that we can functionally rescue that protein,” she said.
Desjardins founded Congruence after serving as CEO of Clementia Pharmaceuticals, acquired in a $1.3 billion deal with Ipsen in 2019. She told Endpoints that after Clementia, she wanted to continue researching rare diseases but was looking for the right opportunity to make an impact.
“In rare diseases, many of these single amino acid partial loss of function mutants were what you call temperature sensitive, that means that they could be corrected in vitro simply by lowering the temperature,” Desjardins said. “If you lower the temperature, you actually correct the misfolding and restore cellular function. So that was fascinating to me.”
Though this has been a well-known phenomenon, she added, no one really knows why it occurs. The foundational goal of Congruence is to treat such proteins as polymers, making it possible to map everything out and then use the machine learning platform to address questions that normally wouldn’t be possible in a wet lab.
Going forward, Desjardins said what sets Congruence apart from the pack is it’s going after targets that are already validated, rather than trying to find completely new ones. The company’s platform also allows it to reverse engineer diseases in the hopes of advancing programs more efficiently, she said.
In addition to the lead investors and OrbiMed, Congruence also received funding from Lumira Ventures, Investissement Quebec, Driehaus Capital Management and other unnamed backers.