Clinical
Development progress and planned direction
for BT-101
Clinical progress
BT-101 is in clinical development for severe pediatric Osteogenesis Imperfecta (OI).
The Phase I/II BOOSTB4 study primarily evaluated safety and tolerability of repeated administration, while also providing early data on fracture-related outcomes.
Clinical findings from BOOSTB4 supported progression to a planned pivotal Phase III study.
BT-101 development status
Indication
Severe pediatric
Osteogenesis Imperfecta (OI)
Clinical foundation
Phase I/II study (BOOSTB4)
Current status
Phase III-ready
Regulatory recognition
ODD in EU & US • FDA RPDD
Scientific foundation
Early clinical
Phase I/II
Phase III
Development milestones
Next
Initiation of the planned pivotal Phase III study in Europe and US
Regulatory submissions to enable initiation of the pivotal Phase III study
Publication of data from the Phase I/II BOOSTB4 study
Current
Current activities are focused on the clinical, regulatory and operational steps required to enable initiation of the planned pivotal Phase III study
Achieved
Positive FDA pre-IND feedback supported the planned path toward a pivotal Phase III study
BT-101 has received Orphan Drug Designation (ODD) in the EU and US, as well as FDA Rare Pediatric Disease Designation (RPDD)
BT-101 advanced into multicenter clinical development through the Phase I/II BOOSTB4 study
Initial clinical experience helped shape the later development of BT-101 and supported the rationale for early intervention
BT-101 builds on decades of research in OI and pioneering translational work originating at Karolinska Institutet
Phase I/II experience
The Phase I/II BOOSTB4 study was an international, multicenter clinical trial in severe pediatric OI. It was designed primarily to evaluate safety and tolerability of repeated administration, while also providing early data on fracture-related outcomes.
Study scope
BOOSTB4 investigated prenatal (in utero) and postnatal administration in children with severe OI (Types III and IV), exploring intervention during early skeletal development.
Key takeaways from BOOSTB4
Safe and well tolerated
BT-101 was reported to be safe and well tolerated
No treatment-related serious adverse events
No serious adverse events related to BT-101 were observed
Encouraging fracture-related findings
Follow-up data showed encouraging findings related to fracture burden
The results from BOOSTB4 supported progression toward a planned pivotal Phase III study.
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Science
Biological and scientific rationale
BT-101
Program concept and treatment approach
Company
Team, board, and scientific advisors