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Endeavor BioMedicines Receives Priority Medicines (PRIME) Designation from the European Medicines Agency for Taladegib (ENV-101) for the Treatment of Idiopathic Pulmonary Fibrosis

via Business Wire

Taladegib is the first and only investigational IPF therapy to receive PRIME designation

Phase 2b WHISTLE-PF trial is on track to complete enrollment in H1 2026

Endeavor BioMedicines (“Endeavor”), a clinical-stage biotechnology company developing medicines with the potential to deliver transformational benefits to patients with life-threatening diseases, today announced that the European Medicines Agency (EMA) has granted PRIority MEdicines (PRIME) designation for its investigational therapy, taladegib (ENV-101), for the treatment of idiopathic pulmonary fibrosis (IPF).

IPF is a life-threatening lung disease characterized by progressive fibrosis of lung tissue leading to deterioration in lung function, respiratory failure, and death. IPF represents a significant unmet medical need as currently approved therapies only delay progression of the disease but are not effective in preventing deterioration in lung function.

The PRIME designation was granted based on review of positive clinical results from the Phase 2a proof-of-concept trial evaluating the safety and efficacy of taladegib that indicated promising potential of this therapy to address the significant unmet need in IPF.

  • The clinical trial demonstrated that treatment with taladegib rapidly and significantly improved lung function and total lung capacity from baseline, and reversed key measures of lung fibrosis in patients with IPF.
  • Patients who received taladegib experienced a statistically significant improvement in lung function through the 12 weeks of the trial, with a +1.9% mean improvement in percent predicted forced vital capacity (ppFVC) from baseline as compared to a mean decline in ppFVC of -1.3% for patients in the placebo group (p=0.035).
  • By week 6, ppFVC exhibited an increase from baseline in the taladegib treatment group and a decrease in the placebo group that continued until the treatment was discontinued at week 12.
  • Taladegib also demonstrated a favorable safety and tolerability profile.
  • These results were recently published in The Lancet Respiratory Medicine.

PRIME designation is granted by the EMA to drug candidates that may offer a significant therapeutic advantage over existing treatments or that benefit patients without treatment options. These medicines are considered priority medicines by the EMA, whose aim is to optimize development plans and speed up evaluations so these medicines that address significant unmet medical needs can reach patients faster.

Taladegib is the first and only investigational IPF therapy to receive PRIME designation, underscoring the program’s potential to address the significant unmet need in IPF. Notably, only 26% of all applications for PRIME eligibility have been granted to date, highlighting the rigor of the selection process and further emphasizing the significance of taladegib’s inclusion. The PRIME designation will enable Endeavor to conduct proactive regulatory dialogue with the EMA, optimizing development and accelerating the path toward the goal of delivering an effective treatment for patients living with IPF. The PRIME designation follows Orphan Drug designations received from the EMA and U.S. Food and Drug Administration (FDA) earlier this year.

“IPF has devastating effects on lung function, yet patients with this disease lack effective therapeutic options,” said John Hood, Ph.D., Co-founder, CEO and Chairman, Endeavor BioMedicines. “Our Phase 2a trial data suggest that taladegib can significantly improve lung function and lung capacity above baseline, and reverse key measures of fibrosis. Receiving EMA’s PRIME designation recognizes the potential of taladegib to transform the lives of people living with IPF. We are committed to working closely with the EMA and regulatory agencies across the world to bring this promising therapy to patients as quickly as possible.”

About Idiopathic Pulmonary Fibrosis

IPF is a chronic, progressive lung disease that affects more than 150,000 adults in the United States. Although the exact cause of IPF is unknown, various environmental factors can deliver repeated injuries to lung cells that trigger abnormal wound-healing processes and life-threatening lung scarring. IPF is a chronic disease with limited treatment options and a very poor prognosis: the average life expectancy is only three to five years after diagnosis.

About Taladegib

Endeavor BioMedicines’ investigational medicine taladegib (ENV-101) is a Hedgehog signaling pathway inhibitor. By binding to and inhibiting a key receptor in the Hedgehog pathway, taladegib eliminates the myofibroblasts that cause fibrosis. This may resolve the excessive wound-healing process seen in pulmonary fibrosis, improving lung volume and function.

About Endeavor BioMedicines

Endeavor BioMedicines is a clinical-stage biotechnology company developing medicines with the potential to deliver transformational clinical benefits to patients with life-threatening diseases. Endeavor’s lead candidate, taladegib (ENV-101), is an inhibitor of the Hedgehog signaling pathway in development for fibrotic lung diseases, including idiopathic pulmonary fibrosis (IPF). More information is available at www.endeavorbiomedicines.com and on LinkedIn or X.

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