Sionna Therapeutics, Inc. ( SION ) released data from its Phase 1 trials of SION-719 and SION-451, the company’s first-in-class nucleotide-binding domain 1 (NBD1) stabilizers, in healthy volunteers.

One hundred subjects were dosed in the SION-719 trial, and 110 subjects were dosed in the SION-451 trial.

Data Highlights

Both NBD1 stabilizers were generally well tolerated and achieved pharmacokinetic (PK) concentration targets established using Sionna’s preclinical cystic fibrosis (CF) human bronchial epithelial (CFHBE) model.

These data reinforce the potential of both NBD1 stabilizers to provide clinically meaningful benefit for CF patients, including the potential for wild-type CFTR function, either as an add-on to standard of care (SOC), or in a proprietary dual combination with complementary modulators.

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Both compounds were generally well tolerated in the Phase 1 trials. There were no serious adverse events, treatment-emergent adverse events (TEAEs) that led to discontinuation of the drug, or dose-limiting TEAEs observed. Most TEAEs were mild to moderate.

One Grade 1 TEAE related to liver function tests (LFTs) was observed in a SION-451-treated subject who tested positive for influenza; no TEAEs related to LFTs were observed in the other cohorts of SION-451.

There were no TEAEs related to LFTs in SION-719-treated subjects.

The data from Part C of each trial support the use of a tablet formulation in future studies and indicate that both compounds can be dosed in a fed or fasted state.

SION-719 and SION-451 achieved the desired target PK concentrations at multiple doses twice daily.

Both stabilizers met exposure thresholds that can provide clinically meaningful benefit if administered as an add-on to SOC or in a proprietary dual combination with complementary modulators.

Next Steps For SION-719:

Sionna is progressing SION-719 into a Phase 2a POC trial evaluating the compound as an add-on to SOC in CF patients.

Sionna is on track to initiate this trial in the second half of 2025, with topline data anticipated in mid-2026.

The U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for SION-719. The first subject has been dosed in a midazolam drug-drug interaction (DDI) study to confirm that SION-719 can be dosed in combination with the SOC according to its label.

The study will be completed before the Phase 2a trial is initiated.

Next Steps For SION-451:

Sionna plans to initiate a Phase 1 healthy volunteer dual combination trial evaluating SION-451 combined with SION-2222 (galicaftor) and SION-109.

The trial will assess the safety, tolerability, and PK of varying doses of the dual combinations. It will inform the selection of a dual combination for a Phase 2b trial in CF patients.

Sionna is on track to initiate the healthy volunteer trial in the second half of 2025, with topline data expected in mid-2026.

In February, Sionna Therapeutics ( SION ) closed its upsized initial public offering of 12.18 million shares at $18.00 per share, with approximately $219.2 million in gross proceeds.

Price Action: SION stock is trading higher by 6.80% to $15.47 at last check Wednesday.

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