Company description

We are a biopharmaceutical company focused on the development of innovative therapeutics for rare diseases. We have developed a robust portfolio of clinical stage product candidates. Our two rare disease product candidates are setrusumab for the treatment of osteogenesis imperfecta (OI) and alvelestat for the treatment of severe alpha-1-anti-trypsin deficiency-associated lung disease (AATD-LD) and Bronchiolitis Obliterans Syndrome (BOS) following allogenic stem cell transplant. Following the announcement of the results for setrusumab in a Phase 2b study in adults with OI which demonstrated a dose dependent statistically significant increase in bone mineral density and bone strength, we announced a strategic partnership with Ultragenyx in December 2020 for the development of setrusumab in children and adults with OI. Ultragenyx initiated a pivotal, Phase 2/3 pediatric and young adult study (5-25 years old) in the first half of 2022, with the Phase 3 transition expected in mid-2023. Ultragenyx also intends to initiate a study of pediatric patients under age five with OI in the first half of 2023. We announced successful completion of a Phase 2, dose-finding study for alvelestat in AATD-LD in May 2022 which demonstrated statistically significant changes in Neutrophil Elastase (NE) activity and biomarkers of disease severity at different time points up to 12 weeks. Further, in October 2022 we announced that Fast Track designation was granted by the U.S. Food and Drug Administration (FDA) for alvelestat in AATD-LD, along with additional program updates.

Read the full filing description · filed 28 Mar 2023

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