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BPS804 · 2 trials · 2 indications
The assessment of safety will be based on primarily on the frequency of adverse events, laboratory abnormalities, and serious adverse events suspected by the investigators to be related to study treatments. The AE collection period extends from the time of first study drug administration drug until study completion. The intensity of each AE will be characterized and classified into 1 of the 3 generic categories (mild, moderate, or severe). The number and percentage of patients with adverse events will be tabulated by treatment group, body system and preferred term. The periods for adverse event tabulation will be from dose administration up to next dose administration if a further dose is given, and from dose administration to EOS for the last dose administration of a patient.
Biomarker data from serum bone formation biomarkers: procollagen type I N-terminal propeptide, procollagen type I C-terminal propeptide, osteocalcin, and bone-specific alkaline phosphatase, and from serum bone resporption mbiomarkers: C-telopeptides of type I collagen cross-links, and N-telopeptides of type I collagen cross-links will be reported as concentration results, measured using a specific assay with a working range defined by the Lower limit of quantification and Upper limit of quantification. It will be considered a sign for efficacy, if any of the above show significant (2-sided, alpha=0.05) increase versus baseline in the BPS804 group on day 43.
Bone mineral density will be assessed by dual-energy X-ray absorptiometry of the lumbar spine. Analysis will include four vertebral levels from L1 to L4. Individual vertebral levels may be excluded due to artifact. Bone mineral density Z-scores will be used as these are a comparison of a patient's BMD to that of a patient of the same age, sex, and ethnicity. The comparison of change from baseline with the matching change in the reference group will be done by 2-sample t-tests (1-sided). It will be considered a sign for efficacy, if any of the above show significant (2-sided, alpha=0.05) increase versus baseline in the BPS804 group on day 141.
| Arm | Type | Description |
|---|---|---|
| BPS804 dose escalation | EXPERIMENTAL | BPS804 IV Setrusumab given in escalating doses from 5mg/Kg to 20mg/Kg |
| Treatment group | EXPERIMENTAL | - |
| Untreated reference group | NO_INTERVENTION | - |
| Name | Type | Description |
|---|---|---|
| BPS804 | DRUG | - |
Inclusion Criteria: * Male and female patients 18 to 60 years of age in good health (other than pre-established clinical diagnosis of HPP) as determined by past medical history, physical examination, vital signs, electrocardiogram, and laboratory tests at screening. * Previously established clinica...
BPS804 is an investigational small molecule being developed for rare bone diseases, including hypophosphatasia, osteogenesis imperfecta, and osteopenia. It has been studied in postmenopausal women with low bone mineral density and in adults with these conditions.
BPS804 is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company traded on NASDAQ under the ticker RARE. The company is focused on therapies for rare and ultrarare diseases.
BPS804 has completed Phase 2 clinical trials. It is an investigational drug and has not been approved by the FDA. All three of its clinical trials are completed, and no active trials are currently listed.
BPS804 has been studied in three completed Phase 2 trials: NCT01406548 in postmenopausal women with osteopenia or osteoporosis, NCT01406977 in adults with hypophosphatasia, and NCT01417091 in adults with osteogenesis imperfecta.
BPS804 is a small molecule, but its specific molecular target has not been disclosed in the available information. It is being investigated for its effects on bone mineral density and related bone disorders.
BPS804 is the primary name used in clinical trials. No alternative names have been reported in the available data, so it is not known to be identical to any other marketed drug.