Dive Brief:
- Biotechnology startup Sionna Therapeutics has licensed a group of experimental cystic fibrosis drugs from AbbVie, planning to test them in combination regimens that could challenge Vertex Pharmaceuticals’ market-leading medicines.
- In a deal announced Tuesday, Sionna has acquired global rights to two prospects in Phase 2 trials and a third in Phase 1 testing. Financial terms weren’t disclosed, but AbbVie will get an upfront payment and a stake in Sionna. It’s also eligible to receive future development and sales milestones as well as royalties, if any of the drugs ever reach market.
- The transaction adds to the cystic fibrosis drug portfolio of Sionna, a well-funded startup built to address what it views as the limitations of current treatments. Prior to the deal, the company already had several drugs in development, all of which work differently than Vertex’s therapies.
Dive Insight:
Several would-be competitors have taken a shot at Vertex’s dominant cystic fibrosis business over the years. So far, they’ve all fallen short.
Biotech Proteostasis Therapeutics couldn’t produce a drug combination outperformed Vertex’s therapies, for instance. A messenger RNA-based approach from Translate Bio also disappointed, as did a prospective medicine from PTC Therapeutics.
In the meantime, Vertex raised the bar for potential challengers, adding two- and three-drug regimens that allowed it to reach more people with the disease. Another triplet is under regulatory review and could further solidify Vertex’s position.
Sionna therefore faces long odds in its bid to compete. But the startup is unique among those who’ve come after Vertex. It has multiple therapies designed to stabilize what’s known as NBD1, a tough-to-drug region of CFTR — the protein that’s defective in cystic fibrosis. Sionna has argued this approach could restore normal CFTR function better than Vertex’s medicines. To date, it has publicly disclosed four drug candidates.
The deal with AbbVie gives Sionna drug components with which to work. AbbVie was once seen as Vertex’s largest threat, having acquired a group of cystic fibrosis medicines from Belgian drugmaker Galapagos designed to work similarly to Vertex’s. But a three-drug combination underperformed in Phase 2 testing in 2022, and AbbVie abandoned research the following year.
Sionna says those therapies — galicaftor, navocaftor and ABBV-2851 — may be a better fit alongside its own drugs. The company plans to pick one to use in a two-drug combo, claiming in a statement that preclinical tests it’s done showed potential for “superior efficacy” compared to current standard of care.
The company will start Phase 1 studies of two NBD1 stabilizers this year and also in 2024 complete an early-stage study of a drug called SION-109 that works via a different mechanism. It didn’t say when to expect trials involving AbbVie’s drugs.