- Two Phase 2 compounds, ABBV-2222 and
ABBV-3067, and a Phase 1 compound, ABBV-2851, expand and accelerate
Sionna's pipeline of complementary modulators to combine with its
novel NBD1 stabilizers –
- Previously conducted clinical studies of
ABBV-2222 and ABBV-3067 demonstrate clinical efficacy as measured
by sweat chloride and ppFEV1 –
- Sionna NBD1 stabilizers in dual combination
with SION-109, ABBV-2222, ABBV-3067, or ABBV-2851 demonstrate the
potential for superior efficacy to standard of care in the CFHBE
assay –
BOSTON, July 16,
2024 /PRNewswire/ -- Sionna Therapeutics, a
clinical-stage life sciences company dedicated to developing highly
effective and differentiated treatments for cystic fibrosis (CF),
today announced that it has obtained exclusive worldwide rights to
develop and commercialize multiple clinical-stage compounds through
a license agreement with AbbVie. Under the terms of the agreement,
Sionna will assume all development responsibilities for galicaftor
(ABBV-2222), a transmembrane domain 1 (TMD1)-directed cystic
fibrosis transmembrane conductance regulator (CFTR) corrector, and
navocaftor (ABBV-3067), a CFTR potentiator, both of which have
completed Phase 2 studies, and a Phase 1 TMD1-directed corrector,
ABBV-2851. Sionna will prioritize advancing one of the AbbVie
compounds and SION-109 as potential dual combination options with a
first nucleotide binding domain (NBD1) stabilizer. AbbVie will
receive an upfront payment, an equity investment in Sionna and will
be eligible to receive late-stage development and commercial
milestones and royalties.
"Our strategy is to build a CF franchise anchored on our novel
correctors that stabilize the NBD1 of the CFTR protein," said
Mike Cloonan, President and Chief
Executive Officer of Sionna. "Combining NBD1 stabilizers with just
one complementary CFTR modulator gives us the potential to deliver
superior efficacy over the current standard of care. With this
agreement, we are significantly expanding and accelerating our
pipeline of complementary modulators to combine with NBD1 and
creating multiple options for potentially transformational
combinations to fully normalize CFTR function. We are
well-positioned to execute our differentiated dual combination
development path to provide more efficacious options for people
with CF."
In clinical studies conducted by AbbVie, ABBV-2222 and ABBV-3067
have been generally safe and well-tolerated. In Phase 2 studies,
ABBV-2222 in combination with ABBV-3067 demonstrated clinical
efficacy, including increased percent predicted forced expiratory
volume in 1 second (ppFEV1) and reduced sweat chloride
(SwCl) levels, comparable to approved dual modulator
combinations.[1] In pre-clinical assays, including the Cystic
Fibrosis Human Bronchial Epithelial (CFHBE) assay, performed by
Sionna, dual combinations of a Sionna NBD1 stabilizer and a
licensed complementary AbbVie modulator demonstrated the potential
for superior efficacy over the current standard of care and some
combinations have the potential to fully correct ΔF508 CFTR and
achieve wild type levels of CFTR function.
"With the CF therapies available today approximately two-thirds
of people still do not achieve normal CFTR function," said
Patrick Flume, M.D., Professor of Medicine and Pediatrics at the
Medical University of South Carolina
and clinical advisor to Sionna. "It is exciting to see new
potential modulator combinations being developed based on the novel
ability to stabilize NBD1 and encouraging to see that NBD1
modulators combined with the three licensed programs from AbbVie
have the potential for superior efficacy compared to standard of
care. There is more we can do for people with ΔF508 CF, and I look
forward to seeing these novel double combinations advanced in
clinical development."
As previously announced, Sionna expects to complete a Phase 1
study of its complementary modulator SION-109, targeting the
interface between the intracellular loop 4 (ICL4) region and the
NBD1 domain of the CFTR protein, in the second half of 2024. The
company also plans to initiate Phase 1 studies of two NBD1
stabilizers, SION-451 and SION-719, this year.
CF is caused by mutations in the CFTR gene, which encodes
an epithelial ion channel that is essential for producing healthy,
freely flowing mucus in the airways, digestive system, and other
organs. The most common mutation in CFTR, ΔF508, causes NBD1 to
unfold at body temperature and severely impairs CFTR function.
Sionna is developing the first-ever clinical-stage NBD1 corrector,
SION-638, and a second series of highly potent NBD1 correctors
including SION-451 and SION-719, which are advancing to Phase 1 in
2024. The company is also developing complementary modulators to
enable combination therapies, including SION-109, an ICL4 directed
corrector, and SION-676, a TMD1-directed corrector.
About Sionna Therapeutics
Sionna Therapeutics is a
clinical-stage life sciences company dedicated to developing highly
effective and differentiated treatments for cystic fibrosis (CF) by
normalizing the function of CFTR, the key protein associated with
disease progression in CF. Building on over a decade of extensive
research on the genetic mutations associated with CF and founded in
2019, Sionna is advancing a pipeline of small molecules engineered
to correct the protein defects caused by ΔF508, the most common
mutation that affects the CFTR protein. The company has a
first-in-class portfolio of programs directly targeting correction
of NBD1, the key and unique mechanism to enable full restoration of
ΔF508-CFTR function, and complementary programs targeting ICL4 and
TMD1. Sionna's pipeline has the potential to deliver best-in-class
efficacy and reach previously unachievable levels of long-term
benefit for people with CF. For information about Sionna visit
https://www.sionnatx.com/.
Media Contact
Adam
Daley
Berry & Company Public Relations
212.253.8881
adaley@berrypr.com
Investor Contact
ir@sionnatx.com
1 https://www.clinicaltrials.gov/study/NCT03969888
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SOURCE Sionna Therapeutics