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October 27, 2014
Repros Therapeutics reported results of
a phase III study of Androxal (ZA-303) to
evaluate the effects on bone mineral density
of administration of Androxal for
52 weeks to overweight men with acquired
hypogonadotropic hypogonadism. The
study enrolled 317 men younger than 60
years old with BMI greater than 25 if they
had morning testosterone of <300ng/dL on
two screening visits. The single-blind,
placebo-controlled, multi-center study
initiated dosing at 12.5mg and dose escalated
as necessary to 25mg. Of enrolled
subjects, 213 and 104 subjects were treated
with Androxal and placebo, respectively.
The study results showed no evidence of a
negative effect on bone mineral density in
subjects treated with Androxal in comparison
to the placebo treatment group.
However, placebo-treated subjects experienced
a statistically significant decrease
from baseline in Total Hip bone mineral
density (-0.63%) than subjects treated with
Androxal (0.01%, p = 0.0043). The percentage
of Androxal-treated subjects obtaining
a morning testosterone over 300ng/dL in
a 12-month treatment window was 79.3%,
and 71.4% had a normal testosterone at
their last observation on treatment. Androxal
was well-tolerated. Repros plans
to file an NDA for Androxal at the end
October 29, 2012
Celgene International reported results from a phase III trial of pomalidomide plus low-dose dexamethasone compared to high-dose dexamethasone for multiple myeloma. This multi-center, randomized, open-label study enrolled patients with relapsed and/or refractory multiple myeloma. Subjects in Arm 1 received 4mg pomalidomide on days 1-21 of each 28-day cycle, with patients 75 years or younger also receiving 40mg of low-dose dexamethasone and patients older than 75 receiving 20mg of low-dose dexamethasone on days 1, 8, 15 and 22 of each 28-day cycle, until disease progression. Subjects in Arm 2 received 40mg high-dose dexamethasone on days 1-4, 9-12 and 17-20 of each 28-day cycle if they were 75 years or younger, and patients older than 75 received 20mg high-dose dexamethasone on days 1-4, 9-12 and 17-20 of a 28-day cycle, until disease progression. The study met its primary endpoint of improvement in progression-free survival (PFS) at the PFS final analysis. Additionally, the study crossed the superiority boundary for overall survival (OS), a key secondary endpoint. As a result, patients who had not yet progressed in the high-dose dexamethasone arm should be crossed-over to the pomalidomide plus low-dose dexamethasone arm. Celgene has submitted an NDA application to the FDA, as well as an MAA application to the EMA.
October 22, 2012
Merck issued results from a phase II trial of odanacatib for the treatment of osteoporosis. This randomized, double-blind, placebo-controlled, multi-center study enrolled 243 women with post-menopausal osteoporosis who had been previously treated with alendronate. Patients were at least 60 years of age with low Bone Mineral Density (BMD) T-scores (=-2.5 and >-3.5) at any hip site. Subjects received odanacatib 50mg or placebo once weekly for 24 months. All subjects received vitamin D3 (5600IU/week) and also calcium supplementation, if needed. Data demonstrated that odanacatib (compared to placebo) significantly increased BMD at all three hip sites (+1.73%, +1.83%, +0.83% for the femoral neck, hip trochanter and total hip, respectively, versus -0.94%, -1.35%, -1.87% with placebo), and the lumbar spine (+2.28% versus -0.30% change with placebo). At the distal forearm, BMD changes from baseline at 24 months were -0.92% and -1.14%, which was not statistically significant. Odanacatib was well tolerated. The most frequent adverse events were urinary tract infection, back pain, arthralgia, fractures, bronchitis, nasal pharyngitis and upper respiratory infection. Based on these data, Merck anticipates submitting regulatory applications for odanacatib in the U.S. and E.U. in the first half of 2013, and in Japan in the second half of 2013.
January 17, 2011
Ablynx issued positive interim results from an ongoing phase I trial of ALX-0141, a nanobody under development for the treatment of degenerative bone diseases. The double- blind, placebo-controlled study enrolled 42 healthy, post-menopausal women who received single subcutaneous injections of ALX-0141 at doses ranging from 0.003 mg/kg to 1 mg/kg. At the nine month follow-up time point, statistically significant suppression of the bone biomarker CTX-1 was noted. Following a single dose of 1mg/kg, 4 out of 6 subjects (67%), in this highest dose group, showed statistically significant suppression of CTX-1 at 9 months.
Enobia Pharma released positive results from a phase II trial of ENB-0040 for the treatment of juveniles with hypophosphatasia (HPP). This six-month, open label trial enrolled 13 pediatrics, ages five to 12 years, with rickets and gross motor deficits from HPP. The subjects received subcutaneous injections of either 2 mg/kg or 3 mg/kg ENB-0040 three times weekly for 24 weeks. The primary endpoint was reached, with data demonstrating a statistically significant improvement in rickets when compared with historical matched cohort controls (p≡0.002). Nine of 13 subjects enrolled (69%) and nine of 12 who completed the study (75%) achieved a substantial improvement in rickets as assessed by skeletal radiographs of the wrists and knees, compared with two of 17 (12%) historical controls.
October 4, 2010
Enobia Pharma reported positive results from a phase II trial of ENB-0040 for the treatment of juvenile hypophosphatasia. This six-month, randomized, open label, dose comparison study enrolled 13 subjects, ages five to 12 years with rickets and gross motor deficits from hypophosphatasia. The subjects received subcutaneous injections of either 2 mg/kg or 3 mg/kg ENB-0040 three times weekly for 24 weeks. The efficacy of ENB-0040 was primarily based on improvements in rickets as measured by X-rays. Data showed radiographic improvement of rickets noted as early as six weeks after starting treatment. EMB-0040 also led to improvement in muscle strength and agility over baseline, including an average improvement of 125m on six-minute walk test. In addition, amelioration of pain was reported in six of seven patients experiencing pain at baseline. Subcutaneous injections of ENB-0040 were generally well tolerated and no serious adverse events were reported.
June 28, 2010
Enobia released positive interim results from a phase II trial of ENB-0040 for the treatment of hypophosphatasia (HPP). This six-month, randomized, open label, dose comparison study enrolled 13 pediatrics with HPP between the ages of 5 and 12 years across sites in North America. ENB-0040 (2 mg/kg or 3 mg/kg) was administered as a subcutaneous injection three times weekly for 24 weeks. After 12 weeks of treatment with ENB-0040, there were marked improvements in bone mineralization and function, including increases in strength, endurance and mobility and reduction in pain.
December 16, 2002
Celgene reported positive results from a phase II trial investigating Thalomid (thalidomide) in combination with prednisone for the treatment of myelofibrosis. The data demonstrated that 13 of 21 subjects (62%) achieved an objective clinical response that was defined by an increase in red blood cells or platelets. Seven of ten subjects (70%) who were red cell transfusion dependent responded to the combination therapy and four of the ten subjects (40%) became transfusion independent. Six of eight subjects (75%) with thrombocytopenia experienced a more than 50% increase in their platelet count. The most commonly reported adverse events were constipation, leukocytosis, mild neuropathy and mild sedation. The study enrolled 21 subjects with myelofibrosis who received 50 mg/day of Thalomid and a tapering regimen of prednisone.