This biweekly Respiratory Video Recap highlights key developments across clinical progress, regulatory actions, and the ongoing push for innovative treatments in respiratory diseases. These updates reflect significant advancements in therapies and efforts to improve patient access.

🎯 Watch Our Video Summary Capturing Respiratory News from the Last Two Weeks

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Top Stories Covered in This Video

Chapters

0:00 Introduction
0:08 Krystal Biotech Announces Positive Interim Data from Phase 1 CORAL-1 Study for KB407 in Cystic Fibrosis
0:43 Clarametyx Biosciences Reports Positive Phase 2a Data for CMTX-101 in Cystic Fibrosis
1:19 Kinaset Therapeutics Raises $103M in Series B to Advance Frevecitinib for Severe Asthma
1:45 AN2 Therapeutics Receives FDA Clearance for Epetraborole in M. Abscessus Lung Disease
2:11 Tessera Therapeutics Receives FDA Clearance for TSRA-196 Gene-Editing Therapy in AATD
2:39 Boehringer Ingelheim Initiates Phase II Trial for IL-11 Inhibitor in Idiopathic Pulmonary Fibrosis (IPF)
3:10 Rein Therapeutics Awarded Orphan Drug Designation for LTI-03 in IPF
3:37 Surge in Respiratory Drug Sales in Ahmedabad Driven by Pollution-Induced Respiratory Ailments
4:00 How to reach us

Transcript

Welcome to the latest edition of Respiratory Research Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest.

Krystal Biotech has announced positive interim data from the highest-dose cohort of KB407 in their Phase 1 CORAL-1 study for cystic fibrosis. The study confirmed successful delivery of wild-type CFTR protein to lung cells, showing promising results in patients with cystic fibrosis. This breakthrough paves the way for advancing KB407 into Phase 3 trials, offering potential as a mutation-agnostic treatment for cystic fibrosis.

Next, Clarametyx Biosciences reported positive topline data from their Phase 2a study of CMTX-101 in cystic fibrosis patients. The study demonstrated a significant 77% reduction in neutrophil elastase levels and improvements in lung function, providing encouraging results in targeting inflammation in cystic fibrosis. Moving forward, Clarametyx will begin a Phase 2 study in bronchiectasis in 2026, furthering the potential of this treatment.

Kinaset Therapeutics has successfully raised $103 million in an oversubscribed Series B financing round to advance frevecitinib, an inhaled JAK inhibitor for severe asthma. The funding will support Phase 2 trials for both eosinophilic and non-eosinophilic asthma, addressing a critical unmet need in asthma management with a novel therapeutic approach.

AN2 Therapeutics has received FDA clearance for an Investigational New Drug (IND) application for epetraborole in M. abscessus lung disease. This approval will allow the company to proceed with a Phase 2 study of epetraborole, an oral treatment option for a lung infection resistant to traditional therapies, offering new hope for patients who have been reliant on IV treatments.

Tessera Therapeutics has also received FDA clearance for TSRA-196, a gene-editing therapy for Alpha-1 Antitrypsin Deficiency (AATD). This groundbreaking approach aims to permanently correct the genetic cause of AATD, which affects the lungs and liver, representing a significant advancement in gene medicine for genetic diseases with high unmet needs.

Boehringer Ingelheim has initiated a Phase II trial for BI 765423, an IL-11 inhibitor in idiopathic pulmonary fibrosis (IPF). The trial aims to evaluate the efficacy of BI 765423 in restoring lung function and targeting fibrosis in IPF patients. This could represent a major step forward in the treatment of this progressive, life-shortening disease.

Rein Therapeutics has been awarded orphan drug designation for LTI-03 in IPF by the European Medicines Agency (EMA). LTI-03 aims to target fibrosis and improve lung function in IPF patients, with orphan drug status accelerating its clinical development and regulatory interaction, providing hope for patients in need of new treatment options.

In Ahmedabad, India, respiratory drug sales have surged by 11% due to pollution-induced respiratory ailments. Prolonged exposure to air pollution has led to increased demand for asthma and chronic obstructive pulmonary disease (COPD) therapies, highlighting the growing public health concern and the need for effective respiratory treatments.

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Why It Matters

  • Gene and biologic therapies: Companies like Krystal Biotech and Tessera Therapeutics are leading advancements in gene-editing and biologic therapies for cystic fibrosis and AATD, which may redefine treatment paradigms.
  • Inhaled therapies: Kinaset’s pan-JAK inhibitor and Apogee’s asthma therapy show the growing trend in targeted inhaled treatments for chronic respiratory diseases.
  • Regulatory advancements: FDA clearances for epetraborole in M. abscessus and LTI-03 in IPF highlight critical milestones in addressing unmet needs in rare lung diseases.
  • Pollution’s impact: Rising pollution levels in regions like Ahmedabad are causing an uptick in respiratory diseases, emphasizing the need for effective treatment options and access to medication.

🗓️ Explore weekly details and sources

📚 See the full Respiratory archive on our research hub page.

FAQ

What did the interim results of Krystal Biotech’s CORAL-1 study for KB407 show?

The study confirmed successful wild-type CFTR delivery in the highest dose cohort, suggesting the potential of KB407 as a mutation-agnostic treatment for cystic fibrosis.

How does Clarametyx’s CMTX-101 impact cystic fibrosis treatment?

CMTX-101 demonstrated significant reductions in inflammatory biomarkers and microbial burden, providing a new therapeutic avenue for cystic fibrosis patients, especially those with chronic infections.

What is the significance of the FDA clearance for AN2 Therapeutics’ epetraborole?

The FDA’s clearance opens the door to an oral treatment option for M. abscessus lung disease, a hard-to-treat chronic lung infection that currently requires IV therapies.

How does Boehringer Ingelheim’s IL-11 inhibitor work in IPF?

BI 765423 targets IL-11, a key driver of fibrosis, with the potential to not only slow disease progression in IPF but also restore lung function.

Entities / Keywords

Krystal Biotech: KB407, CFTR, cystic fibrosis

Clarametyx Biosciences: CMTX-101, cystic fibrosis, immune-enabling therapies

Kinaset Therapeutics: Frevecitinib, severe asthma, JAK inhibitors

AN2 Therapeutics: Epetraborole, M. abscessus, lung disease

Tessera Therapeutics: TSRA-196, AATD, gene-editing

Boehringer Ingelheim: IL-11, IPF, fibrosis

Rein Therapeutics: LTI-03, orphan drug, IPF

Ahmedabad: Respiratory drug sales, pollution

References

  1. https://www.globenewswire.com/news-release/2026/01/08/3215774/0/en/Krystal-Biotech-Announces-Positive-Interim-Clinical-Update-from-KB407-Phase-1-CORAL-1-Study-with-Confirmation-of-Wild-Type-CFTR-Delivery-to-the-Lungs-of-Patients-with-Cystic-Fibros.html
  2. https://clarametyx.com/clarametyx-biosciences-announces-positive-topline-data/
  3. https://www.kinasettherapeutics.com/kinaset-therapeutics-announces-103-million-oversubscribed-series-b-financing
  4. https://investor.an2therapeutics.com/news-releases/news-release-details/an2-therapeutics-announces-fda-clearance-proceed-90-patient
  5. https://www.tesseratherapeutics.com/news/tessera-therapeutics-announces-fda-clearance-of-ind-application-for-its-lead-in-vivo-gene-editing-program-tsra-196-for-aatd
  6. https://www.boehringer-ingelheim.com/science-innovation/human-health-innovation/new-trial-explores-advancements-ipf-treatment
  7. https://ir.reintx.com/news-releases/news-release-details/rein-therapeutics-receives-orphan-drug-designation-european
  8. https://timesofindia.indiatimes.com/city/ahmedabad/pollution-delivers-double-digit-boost-to-respiratory-drug-sales/articleshow/126850971.cms

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