Nose-Only Inhalation of microRNA Shows Promise for Pulmonary Fibrosis

Lovelace Biomedical designed a nose-only inhalation exposure system to evaluate an investigational microRNA-based therapy for pulmonary fibrosis — and through its findings, has helped to further advance a novel approach to treating a long-underserved disease.

Lovelace worked closely with miRagen Therapeutics Inc., a clinical-stage biopharmaceutical company focused on RNA-targeted therapies, to test preclinical safety and feasibility of an inhaled version of miRagen’s drug candidate, MRG-201.

By normalizing the level of microRNA-29, miRagen’s drug seeks to address uncontrolled fibrotic tissue deposition, a hallmark of pulmonary fibrosis. MiRagen’s inhaled-delivery method builds on promising data from the drug’s systemic administration. By using a nose-only inhalation model, miRagen was able more effectively assess accurate dose, compound integrity, particle size and exposure.

MiRagen presented the data at the recent European Respiratory Society (ERS) International Congress in Milan, Italy. Among the highlights:

  • MRG-201 was nebulized with its chemical integrity maintained.
  • Exposure to lung tissue after inhalation was high, while distribution to tissues tested beyond the lung was minimal, suggesting a potentially effective localized effect and limited systemic impact.
  • Following multiple administrations, aerosolized MRG-201 at 1.0mg/kg was observed to result in down-regulation of intended target genes important in fibrotic tissue deposition induced by bleomycin in rats.

In a statement announcing the results, miRagen President and CEO William S. Marshall, Ph.D., said the company is encouraged by the preclinical inhalation feasibility results for MRG-201, which it believes offers an innovative approach for the potential treatment of pulmonary fibrosis.

“Patients suffering from pulmonary fibrosis have limited treatment options, and this data demonstrates our commitment to developing a safe and effective therapy to treat this debilitating disease,” said William S. Marshall, Ph.D., president and CEO of miRagen, in a statement announcing the results. He noted that miRagen will continue to advance MRG-201 for the indication of cutaneous fibrosis, but will further explore its potential utility for pulmonary fibrosis.

Lovelace Biomedical has deep experience is solving the preclinical challenges of pulmonary fibrosis drug development, and in helping to advance inhaled biologics into clinical testing. Contact us to learn more.