SION-638 / Sionna Therap - LARVOL DELTA

Sionna Therapeutics Announces $182 Million Series C Financing to Advance Clinical Development of Novel Small Molecules in Cystic Fibrosis (PRNewswire) - "Sionna Therapeutics...today announced the closing of a $182 million Series C financing to support the clinical development of first-in-class small molecules designed to fully restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein by stabilizing the first nucleotide-binding domain (NBD1)....This capital raise provides financial flexibility positioning us to execute our clinical development plan with funding through 2026 and multiple value-creating clinical readouts."

Financing • Cystic Fibrosis

Sionna Therapeutics to Present Updates on Clinical Progress in Cystic Fibrosis at 42nd Annual J.P. Morgan Healthcare Conference (PRNewswire) - "Today the company announced it has completed Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) cohorts with SION-638....In the first half of 2024, the Company expects to complete part C of the Phase 1 study, which includes a bioequivalence tablet study and a food effect study....The company also announced plans to advance three additional clinical programs to Phase 1 in 2024, including two compounds from its second series of highly potent NBD1 stabilizers, SION-451 and SION-719, pending results from ongoing Good Laboratory Practice (GLP) toxicology studies. SION-109, which targets NBD1's interface with the ICL4 region, will start Phase 1 in the first quarter of 2024 and will be combined with an NBD1 stabilizer later in development to form a dual modulator combination."

New P1 trial • Trial completion date • Cystic Fibrosis

Beyond SION-638: a novel series of highly potent stabilizers of NBD1 enable full correction of ΔF508-CFTR in a dual combination with Sionna CFTR modulators, providing a path to wild-type function. (NACFC 2023) - "Sionna is advancing a second series of highly potent first-in-class NBD1 stabilizers and complementary CFTR modulators to use in combination with NBD1. Development candidates from this series enable dual-modulator combinations that provide multiple potential paths to full restoration of CFTR function for most people with CF and are progressing toward clinical studies."

CFTR

Sionna Therapeutics to Present at 2023 North American Cystic Fibrosis Conference (PRNewswire) - "Sionna Therapeutics...announced that the company's data will be presented at the 2023 North American Cystic Fibrosis Conference (NACFC) to be held November 2-4 in Phoenix, Arizona. Greg Hurlbut, Ph.D., Co-Founder and Senior Vice President, Discovery Research of Sionna, will present poster number 289...during a poster session scheduled on Friday, November 3 from 11:40am-12:40pm MST."

Clinical data • Cystic Fibrosis • Genetic Disorders

Sionna Therapeutics Announces Presentation of Preclinical Data on NBD1 Stabilizers at 46th European Cystic Fibrosis Conference (PRNewswire) - "Sionna Therapeutics...today announced the presentation of preclinical data demonstrating the activity of the company's portfolio of compounds targeting the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The results are being presented at the European Cystic Fibrosis Society (ECFS) 46th European Cystic Fibrosis Conference held June 7-10 in Vienna, Austria....Sionna is currently conducting a Phase 1 clinical trial evaluating its lead NBD1 modulator, SION-638, and has nominated two additional NBD1 modulators, SION-719 and SION-451, entering Investigational New Drug application (IND) enabling studies in the second half of 2023....The company is also advancing the development of compounds targeting complementary mechanisms including SION-109, which targets NBD1's interface with the ICL4 region, and SION-676, which targets the TMD1 of CFTR."

Pipeline update • Preclinical • Cystic Fibrosis • Genetic Disorders

Sionna Therapeutics Announces Advancement of Cystic Fibrosis Pipeline Programs and Presentation at J.P. Morgan 41st Annual Healthcare Conference (PRNewswire) - "Sionna Therapeutics...today announced advancement of the company's preclinical and clinical development programs for novel small molecules designed to target the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and complementary mechanisms....The first subjects have been dosed in a Phase 1 study to evaluate the safety and pharmacokinetics of SION-638. Sionna expects data from this study in the second half of 2023. The company is also advancing the development of compounds targeting complementary mechanisms and plans to file an IND for SION-109, targeting NBD1's interface with the intracellular loop 4 (ICL4) region, in the first half of 2023."

IND • P1 data • Trial status • Cystic Fibrosis

Sionna Therapeutics Announces IND Clearance and Phase 1 Initiation for SION-638 in Cystic Fibrosis (PRNewswire) - "Sionna Therapeutics...announced the clearance of its Investigational New Drug application (IND) from the U.S. Food and Drug Administration (FDA) for SION-638, a novel small molecule designed to target the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The first subject has been dosed in a Phase 1 study to evaluate the safety and pharmacokinetics of SION-638 in healthy volunteers....'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 normalize 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.'"

IND • New P1 trial • Cystic Fibrosis

Sionna Therapeutics to Present at the North American Cystic Fibrosis Conference (PRNewswire) - "Sionna Therapeutics...announced that it will present preclinical data on novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator combinations at the Cystic Fibrosis Foundation's North American Cystic Fibrosis Conference (NACFC) 2022 to be held November 3-5 at the Pennsylvania Convention Center in Philadelphia. These combinations include Sionna's first-in-class molecules that target the first nucleotide-binding domain (NBD1) of CFTR, with the potential to enable full restoration of CFTR function in CF patients with the ΔF508 mutation."

Preclinical • Cystic Fibrosis • Genetic Disorders

Cystic Fibrosis Foundation Invests $5 Million in Sionna Therapeutics to Develop New CFTR Modulator Therapy (Businesswire) - "New modulators in development by the CF-specific company could provide unique treatment options for the underlying cause of cystic fibrosis for many people with the disease....The Cystic Fibrosis Foundation today announced its investment of $5 million in Sionna Therapeutics....The company is focused exclusively on developing cystic fibrosis transmembrane conductance regulator (CFTR) modulators, a type of therapy that treats the underlying cause of CF."

Financing • Cystic Fibrosis • Genetic Disorders

Sionna Therapeutics Launches with $111 Million Series B Financing to Advance Pipeline of Novel Small Molecules with the Potential to Fully Restore CFTR Function in Cystic Fibrosis (PRNewswire) - "Sionna Therapeutics...today announced the official launch of the company and the closing of a $111 million Series B financing. The round was led by OrbiMed with participation from funds advised by T. Rowe Price Associates, Inc., Q Healthcare Holdings, LLC., a wholly owned subsidiary of QIA, the sovereign wealth fund of Qatar, and previous investors including RA Capital, TPG's The Rise Fund, Atlas Venture, and the Cystic Fibrosis Foundation. Sionna has raised approximately $150 million to date....The company plans to advance development of a franchise of small molecules targeting NBD1 and complementary modulators, including NBD1's interface with the intracellular loop 4 (ICL4) region and the transmembrane domain 1 (TMD1) of CFTR. Sionna plans to submit Investigational New Drug applications (IND) for its first NBD1 targeted program, SION-638, and for its lead ICL4 program, SION-109, within the next 12 months."

Financing • IND • Cystic Fibrosis