At Liminal, we believe that we have a novel, differentiated approach to treating the complex biology of fibrotic disease in multiple organ systems. We have developed a strategy to build a broader portfolio of novel small molecule compounds over time as well as opportunities for collaboration in both our small molecule development programs and for Ryplazim® (Plasminogen) or Ryplazim, a unique near-term commercial asset if FDA approval is granted. The key elements of our strategy include:
Advancing the clinical development of our lead product candidate, Fezagepras
Fezagepras (PBI-4050) is our lead candidate for the treatment of patients with diseases related to fibrosis, including respiratory, liver or kidney disease indications. We have generated preclinical data that we believe demonstrates proof-of-mechanism of fezagepras for the treatment of respiratory, liver and kidney disorders associated with chronic or severe fibrosis. We plan to initiate an additional Phase 1 clinical trial to evaluate multiple ascending doses of fezagepras in healthy volunteers. The data from this trial will help define optimal dose levels and dosing regimen to inform the design of any future clinical trials evaluating fezagepras in fibrosis related diseases. We also plan to initiate a Phase 1b/2a clinical trial of fezagepras in patients with high triglyceride levels. Our lead clinical indication for fezagepras is for the treatment of patients with idiopathic pulmonary fibrosis, for which we expect to initiate a Phase 2b clinical trial in 2021.
Seek marketing approval in the United States for Ryplazim® (plasminogen) for the treatment of congenital plasminogen deficiency
We completed our Phase 2/3 clinical trial evaluating Ryplazim in pediatric and adult patients with congenital plasminogen deficiency in October 2018. In September 2020, we resubmitted our BLA for Ryplazim, and FDA assigned a PDUFA target action date of March 5, 2021. We currently plan to launch Ryplazim in the United States in 2021 ourselves with a small, focused commercial infrastructure, or with a marketing collaboration partner, while exploring potential strategic collaborations to provide access to patients in certain other selected markets outside of the U.S. in 2021 and 2022. If our BLA for Ryplazim is approved by the FDA, we may be eligible to receive one Pediatric Review Voucher, or PRV, from the FDA, which we would anticipate seeking to monetize in 2021. Beyond this initial indication, our intention is to conduct additional clinical trials for other applications of plasma-derived plasminogen where there appears to be a scientific rationale for treatment via increasing plasminogen activity levels.
Leveraging our drug discovery platform and knowledge base to develop an early-stage drug portfolio
Our early-stage drug development efforts are focused on expanding our R&D pipeline both for the treatment of diseases associated with fibrosis and for other inflammatory diseases. Accordingly, we first intend to develop oral, selective GPR84 antagonists to treat fibrosis, primarily for patients with respiratory and renal disease. GPR84 is a pro-inflammatory target primarily expressed on cells associated with the immune system and its expression levels increase significantly during periods of inflammatory stress. In addition to GRP84, we intend to develop a novel, selective OXER1 antagonist candidate. OXER1 is a GPCR that is highly selective for 5-oxo-ETE, believed to be one of the most potent human eosinophil chemo-attractants. Eosinophils play a key role in Type 2 inflammation-driven diseases, including respiratory diseases and gastrointestinal diseases. Our GPR84 antagonist and OXER1 antagonist R&D programs are currently both at the pre-clinical research stage.