Spinal Cord Disorders

January 21, 2013

Mesoblast released results from a phase II trial of NeoFuse, comprised of allogeneic Mesenchymal Precursor Cells (MPCs), for the treatment of lumbar spinal fusion. This randomized, multi-center study enrolled 24 patients. Subjects were divided into three arms and received bone autograft standard of care (control), or 25M MPCs or 75M MPCs. Patients underwent the surgical procedure, one or two level fusions using a posterior approach to the spine, and were evaluated for safety and efficacy. The median follow-up times for the three treatment groups were 23.9, 20.7 and 22.9 months, respectfully. Data demonstrated that at 12 months, fusion was achieved in 85.7% of patients in the 25M treatment group compared to 62.5% in the 75M and 75% in the control patient groups. Overall, patients from all three treatment groups had a clinically significant and comparable decrease in low back and leg pain, assessed on the Visual Analogue Scale, and functional improvement, assessed by the Oswestry Disability Index questionnaire. Notably, MPC treated groups had 30%-43% lower mean estimated blood loss during surgery compared to the autograft treatment group (p<0.05 for the 25M group). NeoFuse was well tolerated, with no cell-related serious adverse events and no ectopic bone formation at all. Based on these results, Mesoblast plans to initiate a phase III trial for interbody lumbar fusion in 2013, with patients to be enrolled across multiple sites in the U.S., Europe and Australia.

November 12, 2012

Novartis reported results from a phase IV trial of once-yearly Aclasta for the prevention of spine fractures in men with osteoporosis. This randomized, placebo-controlled, parallel-group study enrolled 1,199 males ages 50 to 85 with primary osteoporosis or osteoporosis associated with low serum testosterone levels. Subjects received Aclasta or placebo as an annual 15-to-30-minute intravenous infusion at baseline and at 12 months. Patients also received daily calcium 1,000-1,500mg and vitamin D 800-1200IU. Data showed a significant reduction in the risk of spine fractures by 67% versus placebo over two years (p=0.002). The results also showed Aclasta reduced the risk of one or more new moderate-to-severe spine fractures by 81% at month 12 (p=0.01) and 63% at month 24 (p=0.03) compared with the placebo group. In addition, Aclasta produced significant and sustained improvements in bone mineral density at the spine and hip bones (lumbar spine, total hip and femoral neck bone [p≥0.05 for all comparisons]) and reduced the risk of height loss (-2.2mm and -4.5mm at month 24 for Aclasta and placebo [p=0.002], respectively). The drug was well tolerated. The most frequent adverse events were similar between treatment groups.

October 15, 2012

Alexion Pharmaceuticals released results from a phase II trial of eculizumab for the treatment of neuromyelitis optica (NMO). This 12-month, single-arm, open-label, investigator-initiated study enrolled 14 patients with severe, relapsing NMO who had experienced two or more disease relapses in the preceding six months or three such attacks in the preceding year. Subjects received eculizumab 600mg weekly for the first four weeks, followed by 900mg at week five and 900mg biweekly thereafter. The study achieved its primary endpoint with statistical significance: a decline in the median annualized attack rate from three attacks per patient pre-eculizumab treatment to zero attacks per patient during 12 months of chronic eculizumab treatment (p<0.0001). After 12 months, 86% (12 of 14) of these severely affected patients were completely attack-free. The median expanded disability status scale (EDSS) score improved from 4.3 pre-treatment to 3.5 after 12 months of eculizumab (p<0.01). All patients experienced either improvement or stability in all key outcome measures, including EDSS, ambulatory function and visual function. Eculizumab was well tolerated. The most frequent adverse events were headache, nausea and dizziness. Alexion expects to participate in a series of discussions with researchers and regulators to design a company-sponsored clinical study to further evaluate potential.

May 19, 2008

Alseres released positive interim results from a phase I/IIa trial of Cethrin for the treatment of neurological recovery following acute spinal cord injury. This open label study enrolled 48 subjects in the US and Canada with thoracic or cervical spinal cord injury. The subjects received escalating doses of Cethrin (0.3, 1, 3, 6 or 9 mg) administered to the injured spinal cord during spinal decompression surgery. Neurological outcomes were measured using the American Spinal Injury Association (ASIA) Impairment Scale at 0, 1.5, 3, 6 and 12 months after treatment. Data is from 37 subjects, all with cervical injuries, who received doses up to 6 mg and who had reached the final 12 month follow up evaluation. Data from six and twelve months showed 38% of the subjects demonstrated a 2-grade or better ASIA grade improvement. Subgroup analysis indicated a dose related response with 43% of the subjects from the 1 and 3 mg dose groups demonstrating improvement of at least 2 ASIA grades. Two of these three subjects improved 3 levels from ASIA grade A to ASIA grade D. Subgroup analysis of twelve month mean motor score changes showed improvement in both the 1 and 3 mg dosage groups with mean motor score changes of 16.3 points in the 1 mg and 27.3 points in the 3 mg doses. Hence, the most effective Cethrin doses for this population were determined to be 1 and 3 milligrams. Dosing in the 9 mg cervical and thoracic cohorts is currently underway. Based on positive data Alseres plans to commence a phase IIb trial in the second half of 2008.

September 3, 2007

Orthogen released positive results from a clinical trial of Orthokine for the treatment of nerve root disorders of the lumbar spine. This randomized, prospective, double-blinded study enrolled 84 subjects at the University of Bochum in Germany. Subjects were placed into three treatment groups to receive Orthokine, 10 mg of cortisone or 5 mg of cortisone. All three drugs were injected into the affected nerve root three times once a week. The subjects were subsequently observed for six months, with interim checks at 6, 10 and 22 weeks after the first injection. The primary endpoint was reduction of symptoms as measured via the visual analogue scale (VAS), which measures pain intensity on a scale of 0 (no pain) to 100 (intense pain). At baseline all subjects rated their pain as moderate to intense (VAS = 80) and had been in pain for at least six weeks. At four weeks post-treatment a statistically significant reduction in pain symptoms was observed for all treatment groups (VAS = 3034). As the trial progressed the Orthokine arm reported a further reduction in pain level (VAS = 15) while the pain intensity in the cortisone arm fluctuated between VAS = 24 and VAS = 34. Based on positive clinical results, Orthogen has filed for US approval of Orthokine.

December 4, 2006

Bioaxone Therapeutics issued positive interim results from a phase I/IIa trial of Cethrin for the treatment of acute spinal cord injury. This trial enrolled 37 subjects across the US and Canada who suffered a complete thoracic or cervical injury. Subjects received Cethrin at four dose levels (0.3, 1, 3 and 6 mg) and were to be followed for a year to assess safety, tolerability and neurological outcome profiles. Six month data has revealed treatment to be safe and well tolerated. In addition, 31% of the subjects had recovered some sensory and/or motor function below the level of their injury and converted from a complete injury to an incomplete injury. Bioaxone plans to move development of Cethrin into further development.

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