Unprecedented Clinical data from the Landmark BOOSTB4 Trial to be presented at the 15th International Conference on Osteogenesis Imperfecta in Hong Kong

COPENHAGEN, Denmark, Oct. 31, 2025 (GLOBE NEWSWIRE) -- BOOST Pharma, a clinical-stage cell biotech company focused on rare skeletal pediatric diseases, is proud to announce that new long-term data from the first-in-class BOOSTB4 stem cell therapy trial has been selected for presentation by BOOST Pharma co-founder Dr. Cecilia Götherström at the prestigious 15th International Conference on Osteogenesis Imperfecta (OI), taking place from October 29-31, 2025, in Hong Kong.

The presentation will provide two-year follow-up data from the landmark BOOSTB4 Phase I/II clinical study. Notably, the results demonstrate that more than 50% of the treated patients experienced zero bone fractures in the second year after last dose (Range 0-5). The previously reported ~70% reduction in fractures during the first year of follow-up was not only sustained but further improved, with an overall reduction of nearly 78% compared to the pre-treatment period. This indicates a durable therapeutic effect of the BT-101 stem cell therapy. These data suggest that BT-101 has the potential to be a disease-modifying cellular therapy, which may effectively prevent new bone fractures in patients with severe OI. Such a treatment could represent a significant advancement for the OI patient community, which currently lacks any approved therapy that modifies the fundamental course of the disease.

“We are excited and honored by the presentation of these groundbreaking results with the global OI and rare disease community in Hong Kong,” said Ingelise Saunders, Chairman of BOOST Pharma. “Demonstrating such a dramatic reduction in fracture rates over a two-year period marks an extraordinary milestone and supports our mission to significantly improve the lives of children born with this severe genetic disorder.”

BOOSTB4 is the world’s first clinical trial to evaluate postnatal and prenatal allogeneic stem cell therapy for OI—also known as brittle bone disease. The therapy, leveraging mesenchymal stem cells with high bone-forming capability, aims to address the genetic cause of OI, moving beyond supportive care and toward lasting, disease-modifying improvement.

About BOOST Pharma ApS

BOOST Pharma is a clinical-stage biopharmaceutical company focused on the development of novel cell therapy treatments. The company is currently developing a first-in-class therapy to treat Osteogenesis Imperfecta, a severe, inherited rare genetic disease leading to significant physical disability. BOOST Pharma is supported by Industrifonden and Karolinska Development, Sweden.

About Osteogenesis Imperfecta

Osteogenesis Imperfecta (OI), also know as Brittle Bone Disease, is a rare and devastating genetic disease, with currently no approved therapies. OI is characterized by fragile bones and reduced bone mass resulting in bones that break easily, loose joints, and weakened teeth. In severe cases, those with OI may experience hundreds of fractures in a lifetime. In addition, people with OI often suffer muscle weakness, early hearing loss, fatigue, curved bones, scoliosis, respiratory problems, and short stature, leading to significant effects on overall health and quality of life. Current treatment of OI is only supportive, focusing on minimizing fractures and maximizing mobility, but to date, there are no FDA or EU approved treatments. OI is estimated to affect 1 in 15,000 people globally.

About the BOOSTB4 Clinical Study

“Boost Brittle Bones Before Birth” (BOOSTB4) is an exploratory, open label, multiple dose, multicenter Phase I/II trial evaluating safety and efficacy of postnatal, or prenatal and postnatal administration, of allogeneic expanded human stem cells for the treatment of severe Osteogenesis Imperfecta (OI) compared with a combination of historical and untreated prospective controls. The aim is to develop a first-in-class cell therapy to reduce the severity of inherited OI in unborn and young children. The study received funding from the European Union’s Horizon 2020 Research and Innovation Program (681045) and from the Swedish Research Council (921-2014-7209) with Karolinska Institutet as study sponsor.

For further information, please contact:

Jonathan Ilicki, Board member
Email: jonathan.ilicki@industrifonden.com

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