Sionna raises $111 million to bring molecules against CF target into clinic

Sionna Therapeutics has raised US$111 million to advance small molecules against a once untreatable target with the potential to improve the treatment of cystic fibrosis (CF). Vertex has transformed the lives of many cystic fibrosis patients in recent years with a series of molecules, but some unmet needs remain, leaving opportunities for rivals to gain a foothold in the market. Sionna reportedly identified full restoration of function of the CFTR, the chloride channel at the heart of cystic fibrosis, as a way to enhance the existing arsenal of treatment options.

While people with cystic fibrosis live longer and more fulfilling lives, the vast majority do not achieve full CFTR correction, which often means they continue to need ancillary medications and are at increased risk of infections and diseases. There is a real opportunity to improve the treatment of cystic fibrosis as there remains a risk of exacerbations, infections and burden on daily life including nutritional status and use of inhalerssaid Sionna CEO Mike Cloonan.

Mike Cloonan, CEO, Sionna Therapeutics

The question is how to completely restore CFTR function. Analysis of the question led the researchers behind Sionna to NBD1, a nucleotide-binding domain that is commonly mutated in CF patients. Deletion of F508 in NBD1 reduces CFTR trafficking to the cell surface and thermal stability of the binding domain.

Evidence of the role NBD1 plays in cystic fibrosis has attracted several research groups, but the target has thwarted efforts to drug it. In 2016, Pfizer researchers reported on a collaboration that resulted in “discouraging“results suggesting NBD1 may be missing”the functionalities needed to build high affinity interactions.

Sionna says they are the first group to solve the NBD1 puzzle, leading to the discovery of small molecules which, when used in combination with other complementary modulators, have shown the potential of in vitro models to normalize the folding, maturation and stability of the CFTR protein. Exactly how Sionna solved the mystery remains a secret.

Our ability to target NBD1 correction is really new. Our team has leveraged over a decade of extensive CF research to develop a first-in-class small molecule franchise targeting NBD1 and complementary modulators. It took years of focus and expertisesaid Cloonan.

More information is on the horizon. With OrbiMed leading a $111 million Series B in Sionna, the biotech could file two new investigational drug submissions, including an application for the NBD1 program, within the next 12 months.

Series B has also attracted support from T. Rowe Price Associates, Qatar’s sovereign wealth fund, RA Capital, TPG’s The Rise Fund, Atlas Venture and the Cystic Fibrosis Foundation. Sionna’s total funding now stands at approximately $150 million.

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