STOCKHOLM, SWEDEN, September 17 2024. Karolinska Development AB (Nasdaq Stockholm: KDEV) today announces that its portfolio company BOOST Pharma has presented positive top-line results from a clinical Phase 1/2 study with a potential first-in-class treatment of the rare bone disease osteogenesis imperfecta (OI). The results show that the treatment was safe and well tolerated and that fracture rates were reduced by over 75%.

BOOST Pharma is developing a first-in-class and potentially groundbreaking cell-based treatment for the congenital disease osteogenesis imperfecta (OI), a condition characterized by fragile bones, constant fractures and deformities of bones. The treatment is based on a novel cell therapy, using human stem cells, with especially high bone-forming capabilities. The treatment is developed to be administered directly upon diagnosis, either before or right after birth, providing a possible treatment advantage in the early years of life, when children become more physically active. Preventing or reducing the number of fractures is considered the most important treatment goal for children with OI, but there is still no approved treatment that can achieve this.

BOOSTB4 is a clinical Phase 1/2 study involving 17 patients from seven European countries. The patients, diagnosed with severe osteogenesis imperfecta type III and IV, were treated at Karolinska University Hospital for 12 months. The results show that the treatment was safe and well tolerated both when administered before and after birth. The results also showed that the bone fracture rates were reduced by over 75% up to 12 months after the last dose.

“We are pleased to learn about the fantastic results from the BOOSTB4 trial, showing a significant reduction in the occurrence of fractures over time – a critical metric to demonstrate clinical relevance of the treatment. The results strengthen our belief in the project and bring hope to a patient group that today lacks adequate treatment,” says Viktor Drvota, CEO of Karolinska Development.

BOOST Pharma plans to announce the full study results in future scientific contexts and the continued development program will be discussed with the U.S. Food and Drug Administration (FDA). BOOST Pharma’s cell therapy has received Rare Pediatric Disease designation in the U.S. and Orphan Drug Designation in the U.S. and EU.

Karolinska Development invested in BOOST Pharma in May this year. Following the full investment, which includes a second tranche later in 2024, Karolinska Development's ownership in BOOST Pharma will amount to 10%.