Sionna Therapeutics, a US-based life sciences company, announced on Wednesday. that it has received approval from the US Food and Drug Administration (FDA) for its Investigational New Drug application (IND) for SION-638, a novel small molecule that targets the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
The company has dosed its first subject in a phase one study to assess the safety and pharmacokinetics of SION-638 in healthy volunteers.
Mike Cloonan, Sionna Therapeutics president and chief executive officer, said, 'Despite advances in CF treatment, the unmet need is high as current therapies can only partially correct the function of the CFTR protein, which means many patients are still at risk of experiencing exacerbations, complications, and a burden on daily life. We have achieved an important milestone of advancing SION-638, a first-in-class NBD1 modulator, into a Phase 1 study. We are encouraged by data from clinically predictive CF models demonstrating the potential to normalise folding, maturation, and function of the CFTR protein, and we look forward to evaluating our ability to drive improved outcomes for people with CF in clinical trials.'
Circio unveils circVec proof-of-concept
GSK releases decade-long data on Shingrix efficacy
BiVictriX receives FDA Orphan Drug Designation for AML treatment candidate
GSK announces positive EAGLE-1 results for gepotidacin in gonorrhoea treatment
Boehringer Ingelheim reports strong growth in 2023 and accelerates late-stage pipeline
Amneal announces launch of PEMRYDI RTU, a ready-to-use oncology injectable
PureTech completes enrollment in Phase 2b ELEVATE IPF trial for LYT-100
GSK's meningitis vaccine candidate accepted for FDA review
Medivir and Vetbiolix report positive results from VBX-1000 Proof-of-Concept clinical study
Norgine submits first marketing authorisation application submissions for approval of eflornithine
Innovent Biologics names new Oncology CMO
UroGen's UGN-103 IND accepted by FDA for bladder cancer treatment
ProAxsis secures GBP1.8m investment
Innate Pharma advances Sanofi-developed NK cell engager to Phase 2 for blood cancer patients