Respiratory Today—October 31, 2025

LucidQuest

1 week ago

This week’s respiratory news delivers the latest updates, developments, and regulatory milestones from biopharma companies active in the space.

In Today’s Newsletter

💉 Sanofi’s efdoralprin alfa hits Phase 2 endpoints in AATD emphysema [1] [23 Oct 2025]

https://www.newsmp.com/news/articleView.html?idxno=249528
Context: ElevAATe global Phase 2 compared Q3W/Q4W dosing vs weekly plasma-derived augmentation.
Key point: Met primary and key secondary endpoints with higher functional AAT in normal range vs augmentation; safety comparable (endpoint details not specified).
Implication: May influence prescriber choice and payer reviews pending full data.

🧬 Sionna NBD1 stabilizers show clean Phase 1 profile; CFTR half-life restored in preclinical data [2] [US • 24 Oct 2025]

https://investors.sionnatx.com/news-releases/news-release-details/sionna-therapeutics-announces-presentation-sion-719-and-sion-451
Context: Two randomized, placebo-controlled Phase 1 trials in >200 healthy volunteers; NACFC presentations.
Key point: SION-719 and SION-451 were generally well tolerated and exceeded exposure targets; preclinical data show F508del-CFTR half-life restored up to wild-type levels.
Implication: May influence prescriber choice and payer reviews pending full data.

🧪 Vertex ties lower sweat chloride to better outcomes, highlights ALYFTREK data at NACFC [3] [US • 23 Oct 2025]

https://news.vrtx.com/news-releases/news-release-details/vertex-presents-new-data-across-portfolio-cystic-fibrosis
Context: Multiple abstracts and posters across ages and genotypes, including post-hoc SKYLINE analyses.
Key point: Achieving sweat chloride <60 and <30 mmol/L associated with lower antibiotic use; ALYFTREK showed greater sweat chloride reductions vs TRIKAFTA in Phase 3 (numerics not restated here).
Implication: Observational/RWE. Could inform practice and payer discussions, interpretation depends on design and confounding control.

🏥 FDA expands WINREVAIR label to include clinical worsening reduction in PAH [4] [US • 27 Oct 2025]

https://www.merck.com/news/u-s-fda-approves-updated-indication-for-winrevair-sotatercept-csrk-in-adults-with-pulmonary-arterial-hypertension-pah-who-group-1-pulmonary-hypertension-based-on-phase-3-zenith-study/
Context: ZENITH Phase 3 in high-risk PAH on background therapy.
Key point: Label now includes improved exercise capacity and WHO FC, plus reduced risk of hospitalization for PAH, lung transplant and death.
Implication: May influence prescriber choice and payer reviews pending full data.

🧷 GSK licenses EMP-012 siRNA for COPD from Empirico [5] [UK • 28 Oct 2025]

https://www.gsk.com/en-gb/media/press-releases/gsk-and-empirico-enter-license-agreement-for-clinical-stage-first-in-class-oligonucleotide-candidate/
Context: EMP-012 targets a distinct inflammatory pathway; Phase 1 ongoing; worldwide rights to GSK.
Key point: $85m upfront, up to $660m milestones; potential for longer dosing intervals and non-T2 COPD populations.
Implication: Partnerships/BD. Signals pipeline investment and modality expansion.

🦠 MicrobiotiX gets Korea’s first bacteriophage IND for MP101 in pneumonia [6] [KR • 27 Oct 2025]

https://www.globenewswire.com/news-release/2025/10/27/3174461/0/en/MicrobiotiX-Receives-IND-Approval-for-Novel-Bacteriophage-Drug-MP101.html
Context: MFDS approved Phase 1 in adult acute Pseudomonas aeruginosa pneumonia.
Key point: Two-phage cocktail showed synergistic antibacterial effects preclinically; FIH, randomized, double-blind, placebo-controlled study planned.
Implication: May influence prescriber choice and payer reviews pending full data.

🌬️ Amneal secures tentative FDA nod for generic QVAR MDI [7] [US • 29 Oct 2025]

https://investors.amneal.com/news/press-releases/press-release-details/2025/Amneal-Receives-U-S–FDA-Tentative-Approval-for-Beclomethasone-Dipropionate-HFA-Inhalation-Aerosol/default.aspx
Context: Beclomethasone dipropionate HFA 40/80 mcg, maintenance therapy for asthma, first MDI for Amneal.
Key point: Generic equivalent to QVAR; establishes company presence in complex respiratory inhalation.
Implication: Regulatory/generics. Introduces competition that may affect pricing and formulary access.

💸 Savara lines up $75m royalty funding with RTW for potential MOLBREEVI launch [8] [US • 29 Oct 2025]

https://investors.savarapharma.com/news/news-details/2025/Savara-announces-75M-Royalty-Funding-Agreement-with-RTW-to-Support-the-Potential-Launch-of-MOLBREEVI-in-Autoimmune-Pulmonary-Alveolar-Proteinosis-Autoimmune-PAP/default.aspx
Context: Funding available upon FDA approval; BLA resubmission planned for December.
Key point: Tiered single-digit US royalties to RTW, capped; no approved therapies for autoimmune PAP in US/EU.
Implication: Partnerships/BD. Signals pipeline investment and modality expansion.

🫀 Tectonic’s TX45 improves hemodynamics in PH-HFrEF Phase 1b Part B [9] [US • 29 Oct 2025]

https://investors.tectonictx.com/news-releases/news-release-details/tectonic-therapeutic-announces-positive-topline-data-phase-1b
Context: Single IV dose, open-label, 14 patients; follow-up to prior PH-HFpEF Part A; APEX Phase 2 ongoing in PH-HFpEF.
Key point: Meaningful directional improvements in PCWP, PVR, CO, TPR and mPAP; well tolerated with no serious or severe AEs.
Implication: May influence prescriber choice and payer reviews pending full data.

Why it matters

Regulatory momentum in PAH and generics shows near-term impact on access and standard of care [4][7].
CF field is converging on biomarkers like sweat chloride to guide treatment goals and earlier intervention [2][3].
Modality diversification in respiratory disease continues, from siRNA to bacteriophages and Fc-relaxin biologics [5][6][9].
Financing and BD moves are positioning assets for commercialization in rare respiratory diseases with high unmet need [5][8].

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FAQ

What did ElevAATe show for Sanofi’s efdoralprin alfa?

Sanofi reported ElevAATe met primary and key secondary endpoints, with higher functional AAT levels in the normal range and less frequent dosing vs augmentation therapy; safety was similar to plasma-derived products. Detailed endpoints were not specified in the source [1].

Are Sionna’s NBD1 stabilizers ready for Phase 2 in CF?

Phase 1 data showed both SION-719 and SION-451 were generally well tolerated and exceeded exposure targets. Sionna has initiated a Phase 2a proof-of-concept study, with further readouts planned later, per the company [2].

How does WINREVAIR’s updated label change PAH treatment?

The FDA added reduced risk of clinical worsening events, including hospitalization for PAH, lung transplantation and death, based on ZENITH. This broadens the labeled benefits alongside exercise capacity and WHO FC improvements [4].

What is EMP-012 and why did GSK license it?

EMP-012 is a first-in-class siRNA in Phase 1 for COPD that targets a distinct inflammatory pathway, potentially enabling longer dosing intervals and benefit in non-type 2 inflammation. GSK paid $85m upfront with up to $660m in milestones [5].

What makes MicrobiotiX’s MP101 notable?

It is Korea’s first bacteriophage IND for a therapeutic, a two-phage cocktail targeting Pseudomonas aeruginosa in acute pneumonia, moving phage therapy into first-in-human testing in the country [6].

What did Tectonic’s TX45 show in PH-HFrEF?

A single IV dose produced meaningful improvements across key hemodynamic measures and was well tolerated; echocardiography suggested effects persisted to day 29. The ongoing APEX Phase 2 is in PH-HFpEF [9].

Entities / Keywords

Sanofi; efdoralprin alfa; SAR447537; INBRX-101; ElevAATe; AATD • Sionna Therapeutics; SION-719; SION-451; NBD1 stabilizers; CFTR • Vertex; ALYFTREK; vanzacaftor/tezacaftor/deutivacaftor; TRIKAFTA; sweat chloride • Merck; WINREVAIR; sotatercept-csrk; ZENITH; PAH • GSK; Empirico; EMP-012; siRNA; COPD • MicrobiotiX; MP101; bacteriophage; Pseudomonas aeruginosa; MFDS • Amneal; beclomethasone dipropionate HFA; QVAR; MDI • Savara; MOLBREEVI; molgramostim; aPAP; RTW • Tectonic Therapeutic; TX45; Fc-relaxin; PH-HFpEF; PH-HFrEF.