Sionna revives AbbVie's cystic fibrosis drugs, targeting Vertex

Biotechnology startup Sionna Therapeutics has secured the licensing rights from AbbVie for a group of experimental cystic fibrosis drugs. This strategic acquisition, announced on Tuesday, gives Sionna global rights to two drugs currently in Phase 2 trials and a third in Phase 1 testing. The financial specifics of the deal were not disclosed, though AbbVie will receive an upfront payment, a stake in Sionna, future development and sales milestones, and potential royalties from any successful market entries.

The acquisition enhances Sionna's cystic fibrosis drug portfolio, which already includes several innovative treatments aiming to overcome the limitations of existing therapies. Sionna's approach is distinctive; it focuses on developing drugs that work differently from those of Vertex Pharmaceuticals, the current market leader in cystic fibrosis treatment.

Numerous companies have attempted to challenge Vertex's dominance in cystic fibrosis treatments, but none have succeeded so far. For instance, Proteostasis Therapeutics failed to develop a combination treatment superior to Vertex's. Similarly, Translate Bio's messenger RNA-based approach and PTC Therapeutics' prospective medicine did not meet expectations. During this period, Vertex continued to enhance its position by developing two- and three-drug regimens, thereby expanding the number of treatable cystic fibrosis patients. A new triplet regimen is currently under regulatory review, which could further cement Vertex's market position.

Sionna faces significant challenges in its quest to compete with Vertex. However, the startup's approach sets it apart from previous competitors. Sionna focuses on therapies designed to stabilize a challenging area of the CFTR protein known as NBD1, which is defective in cystic fibrosis patients. The company believes that this strategy could potentially restore normal CFTR function more effectively than Vertex's drugs. To date, Sionna has publicly disclosed four drug candidates.

The collaboration with AbbVie provides Sionna with critical drug components for its research. AbbVie was once considered a major contender against Vertex, acquiring a collection of cystic fibrosis drugs from Belgian company Galapagos, designed to work similarly to Vertex's treatments. However, a three-drug combination from AbbVie did not perform well in Phase 2 testing in 2022, leading the company to abandon the research in the following year.

Sionna believes that the therapies acquired from AbbVie—galicaftor, navocaftor, and ABBV-2851—may complement its own drugs more effectively. The company intends to select one of these drugs for a two-drug combination, citing preclinical tests that indicate potential for superior efficacy compared to the current standard of care.

This year, Sionna plans to initiate Phase 1 studies of two NBD1 stabilizers. Additionally, by 2024, the company aims to complete an early-stage study of SION-109, a drug that operates through a different mechanism. However, the timeline for trials involving AbbVie's drugs has not been disclosed.

In summary, Sionna Therapeutics' recent deal with AbbVie represents a significant step in its mission to develop more effective cystic fibrosis treatments. While the path ahead is challenging, Sionna's unique approach and the strategic acquisition of AbbVie's drug candidates provide a foundation for potential breakthroughs in the field.