Sionna Secures $182M for Cystic Fibrosis Treatment Development to Rival Vertex

Sionna Therapeutics, based in Massachusetts, has successfully concluded a Series C funding round, raising $182 million. This funding is aimed at further developing small molecules designed to stabilize the first nucleotide-binding domain (NBD1) of the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) protein, potentially restoring its function.

The Series C round was led by Enavate Sciences and included new investors like Perceptive Advisors and Viking Global Investors. Established investors such as OrbiMed, RA Capital, The Rise Fund of TPG, Atlas Venture, the Cystic Fibrosis Foundation, and Q Healthcare Holdings also contributed. Previously, Sionna raised $111 million in a Series B round in 2022.

CEO Mike Cloonan expressed confidence in their team’s expertise and the potential of their NBD1-targeting small molecules. He also acknowledged the strong support from investors, which is crucial for advancing their clinical development agenda through 2026.

Preclinical data from Sionna indicates that their NBD1 stabilizers can effectively restore ΔF508-CFTR function when used alongside modulators. Their Phase I trial for the candidate SION-638 has shown that it is generally safe and well-tolerated. The company is also planning clinical trials for two additional NBD1 stabilizers, SION-451 and SION-719, set to begin in 2024. Another compound, SION-109, which interacts with the CFTR intracellular loop 4 region, entered Phase I trials in January 2024.

Sionna's primary competitor in the CF market is Vertex Pharmaceuticals, which reported positive outcomes for its investigational CF treatment, Vanza, in a Phase III trial conducted in February 2024. Vanza is expected to undergo regulatory review later in the year. Additionally, companies like Intellia Therapeutics and ReCode Therapeutics are making strides in CF treatment development through their respective technological innovations.

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