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Lupus Clinical Trials

New Medical Therapies™

Multiple Sclerosis

Patient Medical Areas

October 28, 2013

Genmab reported results of a phase II study of the subcutaneous formulation of ofatumumab in relapsing-remitting multiple sclerosis (RRMS). The multi-center, randomized, double-blind, placebo-controlled phase II study included 232 subjects who had RRMS. Subjects were randomized to three treatment arms: 3mg, 30mg or 60mg of subcutaneous ofatumumab every 12 weeks, 60mg of subcutaneous ofatumumab every 4 weeks or placebo followed by 3mg of subcutaneous ofatumumab at week 12. The treatment period for all subjects was 24 weeks; subjects were then followed until B-cell repletion for at least an additional 24 weeks. There was a clear separation from placebo on the cumulative number of new gadolinium enhancing lesions (active brain lesions) over a period of 12 weeks in subjects treated with all doses of ofatumumab compared to subjects treated with placebo [p = 90% reduction in the cumulative number of new T1 gadolinium enhancing lesions for all cumulative doses of ofatumumab ≥ 30mg].

April 15, 2013

Biogen Idec released results from a phase IIb trial of DAC HYP (daclizumab high-yield process) for multiple sclerosis (MS). This randomized, double-blind, placebo-controlled, one-year, dose-ranging study, SELECT, enrolled 621 patients with relapsing-remitting MS (RRMS) per McDonald criteria 1-4 and a baseline EDSS score between 0.0 and 5.5. Subjects received DAC HYP 150mg or 300mg once every four weeks, or placebo. Results demonstrate that both 150mg and 300mg doses of DAC HYP significantly reduced the annualized relapse rate (ARR) by 54% (p<0.0001) and 50% (p=0.0002), respectively, compared to placebo. In addition, results demonstrated DAC HYP reduced MS brain lesions compared to placebo. Both doses of DAC HYP also demonstrated a trend in improvements in quality of life (QoL) compared to placebo, as measured by the Multiple Sclerosis Impact Scale (MSIS-29) physical impact score. The drug was well tolerated. The most frequent adverse events were similar in all three study groups. Biogen Idec did not note its plans for DAC HYP.

April 8, 2013

Novartis Pharmaceuticals released results from three phase III trials of Gilenya (fingolimod) for multiple sclerosis. These studies (TRANSFORMS, FREEDOMS and FREEDOMS II) enrolled over 3,600 patients with relapsing forms of multiple sclerosis. Data from the three studies showed a significant reduction in the rate of brain volume loss versus a comparator consistent with previously reported results. In the TRANSFORMS study over one year, Gilenya reduced the rate of brain volume loss by 32% (p<0.001) compared to Avonex (interferon beta-1a IM), a commonly prescribed injectable treatment. Over two years, Gilenya reduced the rate of brain volume loss compared to placebo by 35% (p<0.001) in the FREEDOMS study and by 33% (p<0.001) in the FREEDOMS II study.

March 25, 2013

Biogen Idec reported results from a phase III trial of Plegridy (peginterferon beta-1a) for the treatment of relapsing-remitting multiple sclerosis (RRMS). This multi-center, randomized, double-blind, parallel-group, placebo-controlled study, ADVANCE, enrolled 1,516 patients with RRMS. Subjects received 125mcg Plegridy subcutaneously every two weeks or every four weeks, or placebo. Results showed both Plegridy arms met the primary endpoint of reducing annualized relapse rate (ARR), with the two-week arm achieving 36% at one year compared to placebo (p=0.0007). The two-week cycle of Plegridy also reduced the proportion of patients who relapsed by 39% compared to placebo (p=0.0003). Futhermore, the two-week arm of Plegridy demonstrated significant positive effects on disability progression by reducing the risk of 12-week confirmed disability progression, as measured by the Expanded Disability Status Scale (EDSS), by 38% compared to placebo (p=0.0383). The drug was well tolerated. The most frequent adverse events were infections, redness at the injection site and influenza-like illness. Based on these data, Biogen Idec has initiated an open-label extension study called ATTAIN.

February 4, 2013

Biogen Idec reported results from a phase III trial of peginterferon beta-1a for the treatment of relapsing-remitting multiple sclerosis (RRMS). This global, multi-center, randomized, double-blind, parallel-group, placebo-controlled, two-year study enrolled 1,516 patients with RRMS. Subjects received 125mcg peginterferon beta-1a via subcutaneous injection every two weeks or every four weeks compared to placebo. After the first year, patients on placebo were re-randomized to one of the peginterferon beta-1a arms for the duration of the second year of the study. Data demonstrated 125mcg peginterferon beta-1a significantly reduced the annualized relapse rate (ARR) at one year. Compared to placebo, ARR reduction with two-week dosing was 35.6% (p<0.001) and with four-week dosing was 27.5% (p<0.02). In addition, peginterferon beta-1a reduced the risk of 12-week confirmed disability progression as measured by the Expanded Disability Status Scale (EDSS) by 38% in both dosing arms (p<0.04), as well as reduced the proportion of patients who relapsed by 39% in the once-every-two-week dosing arm (p<0.001) and by 26% in the once-every-four-week dosing arm (p<0.03). The drug was well tolerated. The most frequent adverse events were redness at the injection site and influenza-like illness. Based on these data, regulatory submissions in the U.S. and E.U. are planned for peginterferon beta-1a in 2013.

October 15, 2012

Teva Pharmaceutical reported results from a phase III trial of Copaxone (glatiramer acetate) for multiple sclerosis. This multinational, randomized, double-blind, placebo-controlled study, GALA, enrolled 1,400 patients with relapsing-remitting MS (RRMS). Subjects received Copaxone 40mg/1ml (double the drug’s current marketed dose) or placebo via subcutaneous injection three times a week for 12 months. Data demonstrated Copaxone 40mg/1ml significantly reduced annualized relapse rates (ARR) by 34.4% (p<0.0001) versus placebo. A significant 34.4% reduction in the cumulative number of new and enlarging T2 lesions (p<0.0001) and a significant 44.8% reduction in the cumulative number of gadolinium-enhancing (GdE) legions (p<0.0001) was observed in patients treated with Copaxone 40mg/1ml versus placebo. At 12 months, there was no significant difference in percent change of brain volume between Copaxone and placebo. The drug was well tolerated. The most frequent adverse events were injection site reactions, headaches and nasopharyngitis.

June 25, 2012

Teva Pharmaceutical Industries issued results from a phase III trial of glatiramer acetate for the treatment of relapsing-remitting multiple sclerosis. This multinational, randomized, double-blind, placebo-controlled study, GALA, enrolled more than 1,400 patients. Subjects received 40mg/1ml glatiramer acetate injection (20mg/1ml higher than approved Copaxone) administered subcutaneously three times a week or placebo. The trial met its primary endpoint by significantly reducing the annualized relapse rate (ARR) by 34.4% compared to placebo (p<0.0001). The drug was well tolerated. The most frequent adverse events were injection site reactions, headaches and nasopharyngitis. Teva is extending the study into an open-label trial.

April 23, 2012

Ono Pharmaceuticals issued results from a phase II trial of ONO-4641 for the treatment of multiple sclerosis. This double-blind placebo-controlled study, DreaMS: Drug Research EvaluAtion for Multiple Sclerosis, enrolled 407 subjects between the ages of 18 and 55 with relapsing-remitting multiple sclerosis. The subjects received placebo or one of three active doses of ONO-4641 (0.05, 0.10, or 0.15 mg) once daily for 26 weeks. The primary endpoint was based on the total number of new MS-related brain lesions obtained with MRI at four week intervals for 26 weeks. At the end of the study, subjects in the 0.05, 0.10, or 0.15 mg ONO-4641 dose arms had 82%, 92% and 77% fewer Gd-enhancing brain lesions (all p<0.0001), respectively, compared to placebo. Adverse events appeared to be dose related and included cardiovascular events and liver enzyme elevations.

October 31, 2011

Biogen reported results from a phase III trial of BG-12 for multiple sclerosis. This global, randomized, double-blind, placebo-controlled, dose-comparison study enrolled 1,430 subjects with relapsing remitting multiple sclerosis. The subjects received two dose regimens of BG-12, 240 mg twice a day (BID) and 240 mg three times a day (TID), as well as a reference comparator of glatiramer acetate (GA). Both BG-12 and GA groups were evaluated versus placebo. BG-12 met the primary endpoint by significantly reducing annualized relapse rate (ARR) by 44% for BID (p<0.0001) and by 51% for TID (p<0.0001) versus placebo at two years. The reference comparator, GA; (20 mg subcutaneous daily injection), reduced the ARR by 29% (p<0.02) compared with placebo at two years. BG-12 also significantly reduced the number of new or newly enlarging T2-hyperintense lesions, reduced new T1-hypointense lesions and the proportion of subjects who relapsed.

August 15, 2011

Biogen and Abbott reported results from a phase IIb trial of daclizumab high-yield process (DAC HYP) for the treatment of multiple sclerosis (MS). This randomized, placebo-controlled, dose-ranging study, SELECT, enrolled 600 subjects with relapsing-remitting MS. The subjects received DAC HYP 150 mg or 300 mg or placebo as a subcutaneous injection every four weeks for 48 weeks. The primary endpoint was reduction in the annualized relapse rate after one year. Daclizumab significantly reduced annualized relapse rate by 54% in the 150 mg dose arm (p<0.0001) and 50% in the 300 mg dose arm (p≡0.0002) compared to the placebo arm at one year. DAC HYP also met key secondary endpoints, providing a highly statistically significant reduction in the cumulative number of new gadolinium-enhancing lesions between weeks eight and 24; in the number of new or newly enlarging T2 hyperintense lesions at one year and in the reduction in the proportion of subjects who relapsed. DAC HYP also reduced the risk of sustained disability progression at one year by 57% in the 150 mg dose arm and by 43% in the 300 mg dose arm compared to placebo.

August 8, 2011

Actelion reported results from a phase IIb trial of ponesimod for relapsing-remitting multiple sclerosis. This multicenter, randomized, double-blind, placebo controlled, parallel-group trial enrolled 464 subjects who received one of three doses of ponesimod (10, 20 or 40 mg) or matching placebo administered orally once daily for 24 weeks. Ponesimod significantly reduced the cumulative number of new active lesions on monthly magnetic resonance imaging (MRI) brain scans performed from weeks 12 to 24, with the most effective dose at p<0.0001. Data also showed a clinically meaningful effect observed on annualized relapse rate.

July 18, 2011

Genzyme and Bayer reported results from a phase III trial of alemtuzumab for multiple sclerosis. This randomized, rater-blinded study, CARE-MS I, enrolled 581 subjects with relapsing-remitting MS, who had not received treatment. The trial compared alemtuzumab (12 mg/day intravenous for five days) to Rebif, the current standard of care, according to two co-primary endpoints: the annualized relapse rate and the time to sustained accumulation of disability. All the subjects were followed for two years. Two annual cycles of alemtuzumab treatment resulted in a 55% reduction in relapse rate compared to Rebif over the two years of the study (p<0.0001), meeting one primary endpoint. Statistical significance was not achieved for the second primary endpoint, time to six month sustained accumulation of disability, as compared to Rebif. At the two year time point, 8% of the alemtuzumab arm had a sustained increase in their Expanded Disability Status Scale score as compared to 11% of the Rebif arm.

April 18, 2011

Biogen Idec reported results from a phase II trial of BG-12 for the treatment of multiple sclerosis. This global, randomized, double-blind, placebo-controlled, dose-comparison study, DEFINE (Determination of the Efficacy and safety of oral Fumarate IN rElapsing-remitting MS), enrolled over 1,200 subjects who received BG-12 at 240 mg twice a day or 240 mg three times a day. Results showed that both BG-12 doses met the primary study endpoint, demonstrating a highly statistically significant reduction (p<0.0001) in the proportion of subjects who relapsed at two years compared with placebo. Both doses of BG-12 also met all of the secondary study endpoints, providing a statistically significant reduction in annualized relapse rate, in the number of new or newly enlarging T2 hyperintense lesions, in new gadolinium-enhancing lesions, and in the rate of disability progression, as measured by the Expanded Disability Severity Scale at two years. BG-12 also demonstrated a favorable safety and tolerability profile.

Teva and Active Biotech released results from a phase III trial of laquinimod for the treatment of relapsing multiple sclerosis. This global double-blind study, ALLEGRO (assessment of oral laquinimod in preventing progression of multiple sclerosis), enrolled 1,106 subjects who received oral laquinimod 0.6 mg once daily or placebo. The primary endpoint was reached, with laquinimod showing a statistically significant 23% reduction in annualized relapse rate (p≡0.0024) over two years. Data also showed a significant 36% reduction in the risk of confirmed disability progression, as measured by Expanded Disability Status Scale (p≡0.0122). Treatment with laquinimod was also associated with a significant reduction in brain tissue loss, as measured by a 33% reduction in progression of brain atrophy (p<0.0001). Laquinimod was safe and well-tolerated without immunosuppressive effects.

October 25, 2010

Genentech and Biogen released positive results from a phase II trial of ocrelizumab for the treatment of multiple sclerosis. This multicenter, randomized, parallel-group, partially blinded, placebo and active controlled study enrolled 220 subjects with relapsed, remitting multiple sclerosis. The subjects received two ocrelizumab intravenous infusions of 300mg or two intravenous infusions of 1000mg given at day 1 and day 15 or placebo for 24 weeks. The primary endpoint was the total number of gadolinium-enhancing T1 lesions observed on MRI scans of the brain at weeks 12, 16, 20 and 24 compared with placebo. The reductions were highly significant: 96% for 2000mg ocrelizumab and 89% for 600mg ocrelizumab compared to placebo. Disease activity was also measured by reduction in annualized relapse rate (ARR). At week 24, ARR was significantly lowered versus placebo with a reduction of 73% for ocrelizumab 2000mg and 80% for ocrelizumab 600mg. Both ocrelizumab doses were generally well tolerated and no opportunistic infections were reported.

Sanofi Aventis reported positive results from a phase III trial of teriflunomide for the treatment of relapsing multiple sclerosis. TEMSO was an international, randomized, double-blind, placebo controlled study and enrolled 1,088 subjects who received teriflunomide (7mg or 14mg) or placebo once daily for two years. The primary endpoint was annualized relapse rate, defined as the number of confirmed relapses per patients-year. Both doses of teriflunomide significantly reduced annualized relapse rate by 31% versus. placebo (p<0.0005). The risk of disability progression (sustained for 12 weeks) was also significantly reduced by 30% for the 14mg dose (p≡0.02) and numerically reduced by 24% for the 7mg dose (p≡0.08). Both doses of teriflunomide were well tolerated with a similar number of patients reporting adverse events.

September 27, 2010

GW Pharmaceuticals reported positive results from a phase II trial of Sativex for the treatment of bladder dysfunction related to multiple sclerosis. This double-blind, randomized, placebo-controlled, parallel-group trial enrolled 135 subjects with multiple sclerosis and overactive bladder that was not responding well to current standard of care. The subjects received Sativex or placebo in addition to standard of care for eight weeks. Sativex resulted in statistically significant improvement in a range of bladder symptoms, including nocturia (p≡0.01), daytime frequency, frequency for 24 hours (p≡0.044) and bladder symptom severity (p≡0.001). A significant effect was also seen in the patients global impression of change (p≡0.005). There was no significant effect on incontinence.

July 12, 2010

Genmab reported positive interim results from a phase II trial of ofatumumab for multiple sclerosis. This double blind, randomized, dose-escalation trial enrolled 38 subjects with relapsing-remitting multiple sclerosis. The subjects received two infusions of 100 mg, 300 mg or 700 mg of ofatumumab or placebo. After 24 weeks, they switched: those randomized to placebo received ofatumumab and those who received ofatumumab received placebo. The treatment was well tolerated, with no dose limiting toxicities or unexpected adverse events. Statistically significant reductions in the number of brain lesions (gadolinium-enhancing T1 lesions and new/enlarging T2 lesions), as measured on serial MRI scans from week 8 to week 24, were seen with ofatumumab as compared to placebo. The reductions were seen in all dose groups.

October 5, 2009

Novartis reported positive results from a phase III trial of oral FTY720 (fingolimod) for the treatment of multiple sclerosis. This double-blind, placebo-controlled study, dubbed FREEDOMS (FTY720 Research Evaluating Effects of Daily Oral therapy in Multiple Sclerosis), enrolled 1,272 subjects with relapsing-remitting multiple sclerosis. The subjects received placebo or 1.25 or 0.5 mg doses of FTY720 once daily over two years. The primary endpoint was reduction in annual relapse rate. FTY720 reduced the relapse rate by 54% for the 0.5 mg dose and 60% for the 1.25 mg dose compared to placebo (both p<0.001). In addition, FTY720 reduced the progression of disability by 30% for the 0.5 mg arm (p≡0.024) and 32% for the 1.25 mg arm (p≡0.017) compared to placebo over two years. FTY720 was generally well tolerated with a lower incidence of adverse events at the 0.5 mg dose than 1.25 mg dose.

August 31, 2009

Peptimmune reported positive results from a phase Ib trial of PI-2301 for the treatment of Secondary Progressive Multiple Sclerosis. This 12-week, multiple-ascending dose, double-blind, placebo-controlled, randomized study enrolled 50 subjects, 36 of whom completed the study. The subjects received PI-2301 once weekly for 8 weeks followed by an open label extension of an additional 4 weeks. The doses ranged from 1 to 60 mg. Safety was established at all doses, including potentially therapeutic doses. The most frequent adverse events were dose-dependent site reactions which were mild to moderate, transient, and resolved without specific therapy. Dose-dependent increases in serum levels of anti-inflammatory markers were consistent with PI-2301 exposure.

May 18, 2009

Merck Serono reported positive long-term results from a phase III trial of Mylinax (oral cladribine) for the treatment of multiple sclerosis. This two-year (96-week), randomized, double-blind, placebo-controlled, international trial, dubbed CLARITY (CLAdRIbine Tablets in treating MS orallY), enrolled 1,326 subjects with relapsing-remitting MS. During the first year, oral cladribine would be given in two or four treatment courses, with each course consisting of once daily administration for four to five consecutive days. In the second year, two treatment cycles would be given to all groups. The primary endpoint was the qualifying relapse rate at 96 weeks. Secondary endpoints included MRI endpoints, proportion of subjects qualifying relapse-free and disability progression at 96 weeks. Two year data demonstrated a statistically significant reduction in the annualized rate of relapses compared to placebo. Subjects in the low-dose cladribine arm experienced a 58% relative reduction in annualized relapse rates with respect to placebo (0.14 versus 0.33 for the placebo group; p<0.001) while those in the high-dose cladribine arm experienced a 55% relative reduction in annualized relapse rates with respect to placebo (0.15 versus 0.33; p<0.001). Over the two-year period of the study, 80% of the low dose regimen Cladribine arm and 79% of the high-dose regimen arm experienced no clinical relapse, compared with 61% of the placebo group (p<0.001 for both dose regimens). Treatment with Cladribine also led to a more than 30% reduction in the risk of disability progression relative to placebo over two-years (low-dose regimen: p≡0.018; high-dose regimen: p≡0.026). In addition, both dose regimens of cladribine demonstrated statistically significant reductions in different types of brain lesions as measured by magnetic resonance imaging (MRI) compared to placebo (reductions ranged from 73% to 88% depending on MRI measure and dose group; p<0.001 for each of these MRI measures and for both dose regimens). Adverse events were similar between the cladribine treatment arms and placebo.

March 16, 2009

GW Pharmaceuticals reported positive preliminary results from a phase III trial of Sativex for the treatment of spasticity due to Multiple Sclerosis (MS). This double-blind, randomized, placebo- controlled study initially enrolled 573 subjects in Europe. All subjects had inadequate spasticity relief with existing therapies. All subjects initially received Sativex for four weeks in a single blind manner (Phase A), following which Sativex responders (n≡241) were randomized to continue on Sativex or switch to placebo for a further 12 weeks in a double-blinded manner (Phase B). The primary endpoint, the difference between the mean change in spasticity severity of Sativex versus placebo in Phase B, was reached with statistical significance in favor of Sativex (p≡0.0002). The numeric difference between the two groups as measured on a Numeric Rating Scale was 0.84 units from a baseline of 3.89. Sativex also reached significance over placebo on the following secondary endpoints. An improvement of greater than 30% in spasticity score over the entire study was reached by 74% of the Sativex arm versus 51% on placebo (p≡0.0003). In addition, statistically significant improvements were also seen in spasm frequency (p≡0.005), sleep disturbance (p<0.0001), patient global impression of change (p≡0.023), and physician global impression of change (p≡0.005). Sativex was well tolerated through the study.

February 2, 2009

Merck issued positive results from a phase III trial of cladribine tablets for the treatment of multiple sclerosis (MS). This two-year (96 weeks), randomized, double-blind, placebo-controlled, international trial, dubbed CLARITY (CLAdRIbine Tablets Treating MS OrallY), enrolled 1,326 subjects with relapsing-remitting MS. The subjects were randomized to one of three different treatment groups: low dose cladribine, high dose cladribine or matching placebo tablets. Cladribine tablets were given in two or four treatment courses in the first year, with each course consisting of once daily administration for four to five consecutive days hence, cladribine tablets were taken for 8 to 20 days during the year. In the second year, two treatment courses were administered to all groups. The primary endpoint, the qualifying relapse rate at 96 weeks, was reached. Both dose regimens demonstrated a statistically significant reduction in the annualized rate of relapses compared to placebo. In the lower total dose group a 58% relative reduction in annualized relapse rates was observed with respect to placebo (0.14 versus 0.33 for the placebo group; p<0.001). In the higher total dose group a 55% relative reduction in annualized relapse rates was observed with respect to placebo (0.15 versus 0.33; p<0.001). Secondary endpoints were also met, including reduction of lesion activity as measured by magnetic resonance imaging (MRI), proportion of subjects relapse-free and disability progression. Adverse events were low in the cladribine tablet treatment groups and were comparable to those observed in the placebo group. Based on positive phase III data, Merck plans to file for EU and FDA approval in mid-2009.

October 27, 2008

Avigen issued negative results from a phase IIb trial of AV650 for the treatment of multiple sclerosis spasticity. This double blind, randomized study enrolled 150 subjects in Europe. Following a four-week double-blind assessment, where the subjects received doses of AV650 at up to 900 mg, a six month open-label safety study was offered. The primary endpoint was increased movement as measured using the Ashworth scale. Treatment with AV650 failed to achieve the primary endpoint; the reduction from baseline of Ashworth scores as compared to placebo did not reach statistical significance. Secondary endpoints, including reduction of muscle spasm, also failed to achieve statistical significance. Based on the results, Avigen plans to shift their development focus to other products in their pipeline.

September 29, 2008

Opexa released top-line results from a phase IIb trial of Tovaxin for the treatment of multiple sclerosis. This multicenter, randomized, double blind, placebo-controlled study, dubbed TERMS, enrolled 150 subjects with clinically isolated syndrome (CIS) and early relapsing-remitting MS. Patients received five subcutaneous injections of Tovaxin or placebo at 0, 4, 8, 12 and 24 weeks. The primary efficacy endpoint was the cumulative number of gadolinium-enhanced brain lesions (CELs) using MRI scans summed over weeks 28, 36, 44 and 52. Secondary efficacy endpoints included annualized relapse rate (ARR). Although a positive trend was seen, statistical significance was not reached for either endpoint. The ARR for the Tovaxin-treated group was 0.214 as compared to 0.339 for the placebo-treated group. For subjects who had an ARR greater than 1 at baseline, Tovaxin demonstrated a 55% reduction in ARR as compared to placebo. Treatment was safe and well tolerated. Based on the results, Opexa plans to meet with the FDA to determine a phase III trial design

June 9, 2008

Acorda reported positive results from a phase III trial of Fampridine-SR for the treatment of multiple sclerosis. This double-blind, placebo-controlled trial enrolled 240 subjects in the US and Canada. The subjects were randomized to treatment with Fampridine-SR, at a dose of 10 mg twice a day, or placebo. The primary endpoint was response on the Timed 25-Foot Walk, defined as a participant whose walking speed was faster at a majority of the four on-drug visits than any speed measured during the five off-drug visits. A significantly greater proportion of the subjects receiving Fampridine-SR reached this endpoint compared to the placebo group (42.9% versus 9.3%, respectively; p < 0.001). The secondary endpoint, leg strength, was reached as well, with a statistically significant increase in the Fampridine-SR Timed Walk responders compared to placebo (p = 0.028). In addition, the average increase in walking speed over eight weeks of treatment compared to baseline was 24.7% for the Fampridine-SR Timed Walk responders compared to 7.7% for the placebo group. Treatment was well tolerated. Based on the results Acorda plans to file an NDA with the FDA in Q1 of 2009.

April 21, 2008

BTG issued positive results from a phase II trial of BGC20-0166 for the treatment of obstructive sleep apnea (OSA). This study enrolled thirty nine subjects with mild to severe OSA who received placebo, a single agent or one of two doses of BGC20-0166 daily for a period of twenty eight days. Each subject's apnea-hypopnea index (AHI) was measured in overnight sleep laboratory polysomnograph studies on days fourteen and twenty eight. The primary endpoint was a reduction in the AHI at day twenty eight. The treatment group receiving the high-dose demonstrated a statistically significant reduction in AHI compared to subjects receiving placebo at both day fourteen and twenty eight. AHI was reduced by a mean of 40% in this treatment group, with individual responses ranging between 10% and 85%. Three of ten subjects in the high-dose group were considered complete responders, with a reduction in AHI of 50% or more and an AHI below 10 at day twenty eight. This group also showed reduced AHI in both REM and non-REM sleep stages and independent of sleep position and a trend towards improved oxygen saturation levels relative to placebo. Treatment was well tolerated. Based on the results, BTG plans to continue with the development of BGC20-0166.

Genentech and Biogen released negative results from a phase II/III trial of Rituxan for the treatment of primary-progressive multiple sclerosis. This randomized, double-blind, placebo-controlled study enrolled four hundred and thirty nine subjects in the US and Canada. The subjects received either four treatment courses of Rituxan six months apart or placebo. MRI evaluations were conducted at baseline, weeks six, forty-eight, ninety-six and one hundred and twenty-two. The study did not meet its primary endpoint as measured by the time to confirmed disease progression during the ninety six-week treatment period. The incidence of overall adverse events was comparable between Rituxan and placebo treatment groups. The companies plan to fully analyze the data in order to determine the best path forward.

Santhera reported positive results from a phase II trial of SNT-MC17 for the treatment of Duchenne Muscular Dystrophy (DMD). This double-blind, randomized, controlled study was dubbed DELPHI (Duchenne Efficacy Study In Long-Term Protocol Of High Dose Idebenone). A total of twenty-one pediatric subjects, aged eight to sixteen years, with DMD and cardiac dysfunction were enrolled in Belgium. The subjects received SNT-MC17 450 mg/day or placebo for twelve months. The primary endpoint was the percent change from baseline on the peak systolic radial strain of the left ventricular (LV) inferolateral cardiac wall. In the group treated with SNT-MC17, this was improved by 104%, a significant difference over the placebo group who improved by 29% (p=0.03). In addition, peak systolic longitudinal strain of the LV lateral-mid cardiac region also improved significantly, indicating a beneficial effect of SNT-MC17 on early and systolic myocardial dysfunction in DMD. Secondary endpoints included respiratory function tests. Direct measures of respiratory weakness, including peak expiratory flow, improved in subjects on SNT-MC17. Peak flow expressed as percentage of the predicted value for subjects on SNT-MC17 improved by 2.8% while those on placebo deteriorated by 8.5% (p=0.042). Treatment was determined to be safe and well tolerated. Additional phase II studies are currently underway.

April 14, 2008

GW Pharmaceuticals reported mixed preliminary results from a phase III trial of Sativex for the treatment of central neuropathic pain due to Multiple Sclerosis. This double-blind, randomized, placebo-controlled study enrolled three hundred and thirty three subjects who had achieved inadequate pain relief with existing therapies. The subjects self-administered Sativex at will in order to find their optimum dose level and were to maintain this level for the fourteen week trial duration. The primary endpoint was the proportion of subjects whose pain was reduced by at least 30% as measured on a 0-10 numerical rating scale. Of the subjects who received Sativex, 50% reached this endpoint. While this response was in favor of Sativex over placebo, statistical significance was not reached due to an unexpectedly large placebo response. Key secondary endpoints followed the same trend. The results were considered to be due to the self-administration design and the fact that the subjects on placebo took significantly more doses than subjects on Sativex. Treatment was determined to be safe and well tolerated. Based on the results, GW plans to move forward with the development if Sativex utilizing a fixed dose approach.

MediciNova issued positive results from a phase II trial of MN-166 for the treatment of multiple sclerosis (MS). This two-year randomized, double-blind, placebo-controlled study enrolled two hundred and ninety seven subjects with relapsing MS. The subjects who received 30 or 60 mg of MN-166 or placebo per day during the first twelve months of the study remained on the assigned dose for the second twelve months of the study. The subjects who received placebo during the first twelve months of the study were randomized to receive either 30 or 60 mg of MN-166 per day (double-blind maintained) during the second twelve months of the study. Of the two hundred and ninety seven subjects enrolled, two hundred and forty five completed the full two year study duration. Disease progression, measured as a greater than or equal to 1.0 point increase from baseline in the Expanded Disability Status Scale (EDSS) score for four consecutive months, was one of the primary objectives. Disease progression was significantly less likely (by approximately 50 percent) in those subjects receiving MN-166 at either 30 or 60 mg per day for twenty four months than in those receivingMN-661 for twelve months (p=0.026). In addition, brain volume loss was significantly less in subjects receiving 60 mg per day of MN-166 for twenty four months compared to the other treatment groups (p=0.030). MN-166 at 60 mg per day significantly reduced the relative risk for conversion of new inflammatory lesions, identified at month two, to Persistent Black Holes (PBH) at month ten by 37% (p=0.011) and MN-166 at 30 mg per day resulted in a trend toward reducing evolution to PBH (p=0.074). Treatment was well tolerated at both doses. Based on the results, MediciNova plans to move forward with the development of MN-661.

December 10, 2007

Teva released positive interim results from a phase III trial of Copaxone for the treatment of multiple sclerosis (MS). This multi-national, multi-center, prospective, double-blind, randomized study, dubbed PreCISe, enrolled 481 subjects presenting with a single clinical episode and MRI suggestive of MS. The subjects received either Copaxone 20mg/day or placebo as a subcutaneous injection. Treatment was continued for up to 36 months, unless a second attack was experienced and they were diagnosed with clinically definite MS (CDMS).The primary outcome was time to CDMS, based on a second clinical attack. Copaxone reduced the risk of developing CDMS by 44% versus placebo, and prolonged the quartile time to disease conversion to 722 days versus 336 days in those subjects receiving placebo (hazard ratio 0.56, p=0.0005). At this interim analysis, the proportion of subjects who had developed CDMS was reduced from 43% in the placebo group to only 25% in the Copaxone group (p less than 0.0001). Based on the results, Teva plans to file for regulatory approval in Europe, the U.S. and Canada for the treatment of subjects with a first clinical event suggestive of MS.

October 22, 2007

Genzyme issued positive three-year results from a phase II trial of alemtuzumab for the treatment of multiple sclerosis (MS). This study enrolled 334 subjects with active relapsing-remitting MS in the US and Europe. The subjects were randomized to receive alemtuzumab at one of two doses (12 or 24/mg per day intravenously for five days at initial treatment, and three days of re-treatment after 12 months with an option to treat again at 24 months), or Rebif (44 mcg administered by subcutaneous injection three times per week). The co-primary endpoints, the rate of relapse of MS symptoms and the time to Sustained Accumulation of Disability over six months as measured by Expanded Disability Status Scale [EDSS], were both reached with statistical significance. The subjects treated with alemtuzumab experienced at least a 73% reduction in the risk for relapse after three years of follow up when compared to those treated with Rebif (p=0.00396). In addition, the subjects in the alemtuzumab arm experienced at least a 70% reduction in the risk for progression of clinically significant disability when compared to those treated with Rebif (p=0.01646). Full results from this trial are expected to be released in the spring of 2008.

PDL Biopharma and Biogen released positive results from an ongoing phase II trial of daclizumab for the treatment of multiple sclerosis. This randomized, double-blind, placebo-controlled trial enrolled 230 subjects in the US and Europe. Subjects received daclizumab at 2 mg/kg every two weeks, daclizumab at 1 mg/kg every four weeks or placebo, all in combination with ongoing interferon beta treatment, for a treatment duration of 24 weeks. The primary endpoint, a significant reduction of the number of new or enlarged gadolinium-contrast-enhancing lesions (Gd-CELs) at week 24 compared to placebo, was achieved. The subjects in the 2 mg/kg group experienced 72% fewer new or enlarged Gd+ on average compared to the placebo group (p=0.004). While not statistically significant, the subjects in the 1 mg/kg group experienced a 25% reduction in new lesions compared to placebo. The secondary endpoint, relapse rate, indicated that daclizumab reduced the annual relapse rate over placebo. However, the results did not reach statistical significance. Based on the results PDL and Biogen plan to move ahead with the development of daclizumab.

October 15, 2007

Bayhill Therapeutics released positive results from a phase IIb trial of BHT-3009 for the treatment of multiple sclerosis. This multi-center, randomized, double-blind placebo-controlled study enrolled 289 subjects with relapsing, remitting multiple sclerosis. The subjects received monthly intramuscular injections of BHT-3009 for one year. The primary endpoints were brain magnetic resonance imaging (MRI) measures of disease activity including gadolinium-enhancing lesions, T2 lesions and T1 black holes. Subjects in a prospectively defined group with high anti-myelin basic protein (MBP) antibodies in their cerebral spinal fluid (CSF) showed statistically significantly fewer gadolinium-enhancing lesions in their brain after treatment with 0.5 mg BHT-3009 compared to placebo. Reductions in T2 lesions and T1 black holed were also observed in this population. In addition, significant reductions in several CSF myelin-specific autoantibodies were achieved in all the subjects treated with 0.5 mg BHT-3009 compared to placebo. Based on the results Bayhill plans to meet with the FDA to discuss a phase III trial design.

August 27, 2007

Biogen and Elan announced positive results from two phase III trials of Tysabri for the treatment of multiple sclerosis. These two-year, randomized, double-blind, placebo-controlled, multi-center trials, dubbed AFFIRM and SENTINEL, enrolled 2,113 subjects with relapsing multiple sclerosis. The primary endpoint was to assess the relationship between disease activity and health-related quality-of-life (HRQoL) measures in subjects treated with Tysabri compared to placebo. HRQoL was assessed using a standardized patient evaluated survey, the SF-36, which measures physical and mental components (SF-36 PCS and SF-36 MCS, respectively) and the Visual Analogue Scale (VAS). Both measurements were reviewed at weeks 24, 52 and 104. Tysabri led to statistically significant improvements in SF-36 PCS beginning at week 24 through week 104 and in SF-36 MCS at week 104, compared with a decline in the placebo group. Statistically significant improvements on the VAS were also observed over placebo at week 52 and week 104. Tysabri was recently approved by the FDA.

Eli Lilly reported positive results from a phase III trial of Cymbalta for the treatment of fibromyalgia. This randomized, placebo-controlled trial enrolled subjects with fibromyalgia, with or without depression, who received Cymbalta (60 mg or 120 mg) or placebo. They were subsequently measured at three and six months for improvements on the Brief Pain Inventory Average Pain Score (BPI) and the Patient's Global Impression of Improvement questionnaire (PGI- I). At three months, subjects in both Cymbalta arms showed significantly greater reduction in pain and improvement in PGI-I scores compared with the placebo group. In addition, more subjects treated with Cymbalta (both 60mg and 120 mg) showed significantly greater reduction in pain as measured by a 30% improvement in baseline BPI scores (50.7% and 52.1%, respectively), compared with those taking placebo (36%). At the end of the six-month trial, more subjects treated with both doses of Cymbalta showed a response to treatment, defined as a 50% reduction of baseline BPI scores, (32.6% and 35.9%, respectively), compared with subjects taking placebo (21.6%). A sNDA is currently under review by the FDA.

Merck Serono released mixed results from a phase III trial of safinamide for the treatment of Parkinsons disease. This randomized, double blind, placebo-controlled study enrolled 227 subjects with early stage Parkinsons. Of the original 227 subjects, 187 completed this 12-month extension study. The subjects were placed into one of three arms to receive safinamide 50 to 100 mg once daily, 150 to 200 mg once daily or placebo all in addition to dopamine agonist therapy. The primary objectives of the trial were long-term safety and efficacy profiles. Treatment was well tolerated, with reported adverse events similar between the safinamide and placebo groups. The primary efficacy endpoint, time to intervention, did not reach statistical significance in a pooled analysis of the safinamide arms when compared to placebo. Safinamide treatment delayed the onset of time to intervention by 93 days, as measured by the median time to the event bringing on the intervention (559 days versus 466 days; p=0.334). In a post-hoc analysis permitting an evaluation of events beyond the initial phase, subjects receiving safinamide (50 to 100mg) experienced a significantly lower rate of events compared to the subjects receiving placebo (25% versus 51%; p=0.049). A secondary efficacy endpoint was change in motor symptoms, as measured by the Unified Parkinsons Disease Rating Scale Part III Motor Score (UPDRS III). Post-hoc analysis revealed that safinamide (50 to 100 mg) added to dopamine resulted in a statistically significant improvement in motor symptoms over placebo (minus 4.79.34 versus minus 1.957.41; p=0.019). This was associated with a statistically significant improvement in quality of life, as measured by the Euro quality of life (EuroQoL), both in the pooled dose group (safinamide: 01.85, placebo: 0.421.69; p=0.0046) and in the individual dose groups (safinamide 50-100 mg: 0.031.95; p==0.017; safinamide 150-200 mg: minus 0.031.73; placebo: 0.421.69; p=0.011). Additional phase III trials are currently underway.

April 9, 2007

GlaxoSmithKline announced positive results from a clinicaltrial of ReQuip XL 24-Hourfor the treatment of Parkinson's disease (PD). Thisdouble-blind, placebo- controlled adjunct study, dubbed EASE-PD, enrolled 393subjects with idiopathic Parkinson's disease not adequately controlled withL-dopa. Subjects were randomized 1:1 to receive ReQuip XL 24 or placebo, inaddition to L-dopa, once daily for 24 weeks. The primary endpoint was the meanchange from baseline in awake time spent 'off', defined as the return ofParkinson's symptoms as medication wears off. Secondary endpoints includedvarious motor and non-motor functions, including 'on' time. REQUIP XL 24-Hourdecreased 'off' time by an average of 2. 1 hours per day, while placebodecreased time spent 'off' by 0.3 hours per day. Treatment also significantlyincreased both 'on' time and 'on' time without troublesome dyskinesia by 1. 6hours per and reduced the percentage of 'off' time by more than 12% compared tobaseline. Based on the results, GlaxoSmithKline plans to move forward with thedevelopment of ReQuip XL 24-hour.

Opexa reported positive top-line data from a phase I/IItrial of Tovaxin for the treatment of multiple sclerosis. This one-year trialenrolled 10 subjects who received escalating subcutaneous doses of Tovaxin overa period of 20 weeks. The doses were administered at 6 - 9 x 10(6), 30 - 45 x10(6) or 60 - 90 x 10(6) attenuated T-cells. Treatment was shown to be safe andwell tolerated, with no adverse events related to T-cell vaccination. Tovaxintherapy achieved a 90% reduction in annualized relapse rate (p = 0.0039), withthe 30 - 45 x 10(6) T-cell dose group reaching a 100% reduction in annualizedrelapse rate. A phase IIb trial of Tovaxin is currently enrolling subjects.

March 19, 2007

PDL BioPharma and Biogen Idec announced positive interim results from anongoing phase II trial of daclizumab for the treatment of multiple sclerosis. This randomized, double-blind, placebo-controlled trial enrolled 230 subjectsin the US and Europe. Subjects received daclizumab at 2 mg/kg every two weeks,1 mg/kg every four weeks or placebo, all in combination with ongoing interferonbeta treatment, for 24 weeks. Treatment was well tolerated. The trial met theprimary endpoint of a significant reduction of the number of new or enlargedgadolinium-contrast-enhancing lesions (Gd-CELs) at week 24. Based on theresults PDL and Biogen plan to initiate a phase II monotherapy trial in 2007.

October 2, 2006

Acorda released positive results from a phase III trial of Fampridine-SR for the treatment of walking in subjects with multiple sclerosis. This double-blind, placebo-controlled trial enrolled 301 subjects in the US and Canada who had a definite diagnosis of MS and some degree of walking disability. Subjects were randomized receive Fampridine-SR (n=229) or placebo (n=72), for 14 weeks. Treatment was well tolerated with falls and urinary tract infections the most commonly reported adverse events. Efficacy data revealed that the trial met the primary endpoint of improvement in walking speed, as measured by the Timed 25-Foot Walk, with 34.8% of the Fampridine-SR group showing improvement versus 8.3% of the placebo group (p less than 0.001). This improvement was maintained throughout the length of the treatment. In addition, after 14 weeks the average increase in walking speed compared to baseline was 25.2% for the treated group versus 4.7% for the placebo group. Based on this data Acorda plans to move Fampridine-SR into further development.

CytRx issued positive results from a phase IIa trial of arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS). This multi-center, double-blind, placebo-controlled trial enrolled 84 subjects with ALS, who received arimoclomol, or placebo, in one of three dose levels (25 mg, 50 mg or 100 mg) three times daily for 12 weeks. They were then studied for an additional four weeks without treatment. Treatment was shown to be safe and well tolerated across all dose ranges, with no statistically significant adverse events reported. Pharmacokinetic data indicated that arimoclomol effectively entered the cerebral spinal fluid. The amount of the drug in the cerebral spinal fluid was similar to the amount in the blood and increased as the dose increased. Based on these results a phase IIb efficacy trial, using arimoclomol at the highest dose level, is planned for H1 2007.

Medivation issued positive results from a phase II trial of Dimebon for the treatment of Alzheimer's disease. This six-month randomized, double-blinded, placebo-controlled trial enrolled 183 subjects at 11 sites in Russia, who received treatment for 26 weeks. Treatment was well tolerated with the most commonly reported adverse event dry mouth. Efficacy data revealed that all endpoints were met. Subjects receiving Dimebon demonstrated a statistically significant improvement on the study's primary efficacy endpoint, the Alzheimer's Disease Assessment Scale-cognition, with a 4.0 point improvement in the mean change from baseline to week 26 as compared to placebo (p less than 0.0001). The secondary key endpoint, mean change in Clinical Global Impression of Change, was met as well, with a 0.6 point improvement from baseline to week 26 as compared to placebo (p less than 0.0001). Additional phase II trials are ongoing, with results expected in Q2 2007.

September 11, 2006

Teva and Active Biotech issued positive reports from a phase IIb trial of laquinimod for the treatment of relapsed remitting multiple sclerosis. This multi-center, randomized, double-blind, placebo-controlled trial enrolled 300 subjects in 8 European countries and Isreal. Subjects received laquinimod or placebo, orally, once-daily for 36 weeks. Efficacy data revealed that treatment with the drug significantly reduced the rate of inflammatory disease activity, as measured by the cumulative number of Gadolinium enhancing lesions on brain MRI scans. Treatment also reduced the number of clinical relapses when compared to placebo. Teva and Active Biotech plan to move laquinimod forward into phase III trials.

September 5, 2006

Genentech and Biogen announced positive preliminary results from a phase II trial of Rituxan for the treatment of relapsing-remitting multiple sclerosis (RRMS). This randomized, double-blind, parallel-group, placebo-controlled, multi-center trial enrolled 104 subjects who were randomized to receive either a single treatment course of Rituxan or placebo. Gadolinium enhancing T1 lesions were then observed by MRI brain scans at 12, 16, 20 and 24 weeks. Treatment was well tolerated with adverse events comparable between the two groups, although there was an increase in nasopharyngitis, upper respiratory tract infections, urinary tract infections and sinusitis in the Rituxan treated group. Efficacy data revealed a statistically significant reduction in the total number of gadolinium enhancing T1 lesions. Data from this study was undergoing further analysis and was to be submitted for presentation at an upcoming medical conference.

August 21, 2006

Schering AG/Berlex Laboratories issued positive results from a phase III study of Betaseron for the treatment of multiple sclerosis. This international, multi-center, double-blind, randomized trial, dubbed BENEFIT, enrolled 487 subjects who presented with a single clinical episode suggestive of MS. At the onset of this episode subjects received subcutaneous injections of Betaseron (250 mcg), or placebo, every other day for up to 24 months. Data revealed that subjects in the Betaseron treatment group had a 50% reduced risk of developing clinically definite MS compared with the placebo group. Additionally, subjects in the treated group were two times better protected against developing MS, as defined by the McDonald diagnostic criteria, than those in the placebo group. An sBLA, requesting an expanded label for Betaseron, is currently under review by the FDA.

June 5, 2006

Biogen Idec and Fumapharm issued positive results of a phase II trial of BG-12, for the treatment of relapsed/remitting multiple sclerosis (MS), at the European Neurological Society annual meeting in Lausanne, Switzerland. This double-blind, placebo-controlled, dose-ranging study enrolled 257 patients across sites in 10 European countries. Subjects received one of three doses of the drug (120 mg, 360 mg, or 720 mg) or placebo daily for 6 months. Trial data indicated that the highest investigational dose produced a 69% reduction in the mean number of gadolinium-enhancing lesions on monthly observations for weeks 12-24, compared to placebo. The dose also produced a 48% reduction in newly enlarging T2-hyperintense lesions, and a non-significant, positive-trending 32% reduction in relapse rate. The two lower trial doses did not produce significantly superior efficacy to placebo. No incidence of opportunistic infections was noted.

April 17, 2006

Sangamo announced positive results of a phase I trial of SB-509, for the treatment of diabetic neuropathy, at the 58th Annual Meeting of the American Academy of Neurology (AAN) in San Diego. Primary safety data were positive, with no serious adverse events reported and mild injection site reactions the only noted all-severity adverse events. Dose limiting toxicities were not observed. Single administrations of the drug produced improvements in severity of pain, numbness and neurological symptoms in roughly 50% of subjects. Additional preliminary efficacy was noted in neurologic exam scores and electrophysiological testing. This single blind, single-dose dose-escalation study enrolled 12 subjects with mild to moderate disease, who received intramuscular injections of the drug. Based on these results, the company announced plans to initiate a phase II trial of SB-509 in the second half of 2006.

Somaxon reported positive results of their first phase III trial of Silenor (doxepin HCl), for the treatment of chronic insomnia. The drug was shown to significantly reduce 8-hour Wake After Sleep Onset (WASO), with a mean improvement of 26 minutes for the low dose and 31 minutes for the high dose groups, vs. placebo (p<0.0001). Improvement in secondary measures was also noted: Total Sleep Time was 415 min. and 421 min. for the two Silenor doses (respectively), vs. 374 min. for placebo (p<0.0001); Latency to Persistent Sleep during the initial treatment period was 27 minutes for both doses, vs. 45 minutes for placebo (p=0.011, p=0.0018); these improvements were not maintained at subsequent timepoints (due in part to improvements in the placebo group). This randomized, double-blind, placebo-controlled, parallel-group, multi-center study enrolled 229 patients, who received one of two doses of the drug (3 mg or 6 mg) or placebo nightly for 35 days.

April 10, 2006

Bayhill Therapeutics announced positive results of a phase I/II trial of BHT-3009, for the treatment of multiple sclerosis, at the 58th Annual Meeting of The American Academy Of Neurology. This randomized, double-blind, placebo-controlled study enrolled 30 MS patients across 3 dose-escalating cohorts, each including 3 treatment arms: subjects in each cohort received placebo injections and placebo oral capsules (n=4 per cohort); injections of BHT-3009 (0.5 mg, 1.5 mg and 3.0 mg) in weeks 1, 3, 5, and 9 plus placebo capsules (n=3 per cohort); or one of the three BHT-3009 regimens plus 80 mg of the approved drug atorvastatin via oral capsule daily for 13 weeks (n=3 per cohort). Preliminary data from the first 2 cohorts yielded a positive safety and tolerability profile, with all observed adverse events mild-to-moderate in nature. One laboratory abnormality was observed (low LDL cholesterol in a patient on atorvastatin). Immune response was noted following BHT-3009 administration, with reductions in MBP-reactive Th1 cells observed after administration.

March 27, 2006

GW Pharmaceuticals has issued positive results of a phase III trial of Sativex, for the treatment spasticity related to multiple sclerosis (MS). Results from the per-protocol patient cohort (subjects completing the full study protocol) yielded significant improvement in the trial's primary endpoint, a reduction in spasticity symptom severity score on a 0-10 point assessment scale (p<0.05). Secondary endpoints were also met for this population, including the Responder Analysis and the Carer Global Impression of Change. Trial data failed to meet primary or secondary efficacy endpoints in the intent-to-treat patient population, which included patients who did not complete the study protocol (p> 0.05). This randomized placebo-controlled parallel group study enrolled 335 patients, who received Sativex or placebo for 14 weeks, in addition to continued therapy with best-standard-of-care medication.

March 13, 2006

BioMS Medical has issued positive results of a phase II extension trial of MBP8298, for the treatment of multiple sclerosis (MS). Results from the extension study, designed to investigate the drug's ability to slow disease progression, indicated that in a subset of patients with progressive disease who expressed the HLA-DR2 or HLA-DR4 immune response genes, the drug significantly increased median progression-free survival to 78 months, compare to 18 months for placebo (p=0.004). Patients expressing these genes comprise roughly 75% of MS patients. This placebo-controlled follow-up treated 20 HLA-DR2 or HLA-DR4 patients, who received 500 mg MBP8298 or placebo every 6 months. These data were seen to support an ongoing phase II/III trial of the drug, currently enrolling up to 553 patients.

Corgentech issued positive results of a phase II trial of ALGRX 4975, for the treatment of elbow tendonitis pain. Trial data met their primary efficacy endpoint, with 64% of subjects receiving the drug (n=14/22) achieving clinical response (no or slight pain upon wrist dorsiflexion), compared to 30% (n=7/23) for placebo at week 4 (p=0.0256). Efficacy was maintained through week 8, and a trend for reduced pain score for weeks 2 through 12 was noted. Treatment was generally well tolerated at all time points through 24 weeks, and secondary endpoints were also positive. This randomized, double-blind, placebo-controlled study enrolled 45 patients at 2 sites, who received an injection of the drug or placebo, prior to treatment with a local anesthetic.

January 16, 2006

Biogen Idec and Fumapharm AG reported positive results of a phase II trial of their oral fumarate compound BG-12, for the treatment of multiple sclerosis (MS). Results of the trial met their primary efficacy endpoint, producing a statistically significant reduction in the number of gadolinium-enhancing brain lesions at 6 months, compared to placebo. This multi-center, double-blind, placebo-controlled study enrolled 250 patients across 10 sites in Europe.

October 10, 2005

Human Genome Sciences announced mixed results of a phase II trial of LymphoStat-B (belimumab), for the treatment of systemic lupus erythematosus (SLE). Trial data failed to meet either of the co-primary endpoints, producing neither statistically significant reductions in SLE symptom severity at 24 weeks on the SELENA SELDAI scale, nor increasing time to first disease flare through 52 weeks. However, the drug was found to produce a significant decline in SLE symptom severity at 52 weeks in the seropositive patient subgroup (~75% of subjects) compared to baseline on both the SELENA SLEDAI (p=0.021) and the Physician's Global Disease Assessment (p=0.016) scales; efficacy in this measure in the broader study population was non-significant, but trended positively. Also, the drug produced significant reductions vs. placebo in circulating B cell counts and anti-dsDNA autoantibodies in all treatment arms. This double-blind, placebo-controlled, dose-ranging study enrolled 449 SLE patients, who were to receive one of 3 doses of the drug (1 mg/kg, 4 mg/kg or 10 mg/kg) or placebo via intravenous infusion on days 0, 14 and 28, then at 28 day intervals through 52 weeks.

PharmaFrontiers issued positive interim results of a phase I/II trial of Tovaxin, for the treatment of multiple sclerosis (MS), at the ECTRIMS/ACTRIMS congress in Thessalonica, Greece. Trial data indicated that the drug reduced myelin-peptide reactive T cell counts dose dependently, with patients receiving the higher dose of the drug experiencing a 100% reduction at 5 weeks. These reductions strongly correlated with improvements on the Multiple Sclerosis Impact Scale (p<0.0086). Annual relapse rate was reduced by 92%, compared to baseline. No dose-limiting toxicities were observed. This dose-escalation study enrolled patients with relapsing-remitting or secondary-progressive MS, who received subcutaneous injections of either 6-9 million cells (low dose) or 30-45 million cells (high dose) at weeks 0, 4, 12 and 20.

September 26, 2005

Genzyme and Schering AG have reported interim one-year results of a phase II trials of Campath for the treatment of multiple sclerosis (MS). Campath is currently approved for the treatment of B-cell chronic lymphocytic leukemia. Results indicated superior efficacy in disease treatment compared to Rebif (interferon beta-1a), with a significantly greater portion of subjects achieving a 75% or greater reduction in risk of relapse at 1 year with both low and high doses of Campath (p=0.00267). With respect to the endpoint of reducing risk of clinically significant disability, Campath did yield a reduction in risk of at least 60% (p<0.05), but this value did not reach the pre-designated endpoint (p=0.00015). Serious adverse events were noted among patients receiving Campath, including 3 cases of severe idiopathic thrombocytopenic purpura (1 fatal). This randomized, open-label study enrolled 334 patients across 49 sites in Europe and the US, who received one of two doses of Campath once yearly via intravenous infusion, or Rebif thrice weekly per its dosing label.

June 27, 2005

Novartis announced positive results of a phase II trial of FTY720, for the treatment of multiple sclerosis, at the 15th European Neurological Society (ENS) meeting in Vienna. The drug produced significant reductions in relapse rate at both trial doses, vs. placebo (55% low dose, p=0.009; 53% high dose, p=0.014). Efficacy was also noted in extending time to first relapse (p=0.007, p=0.012 respectively) and in increasing the portion of patients remaining relapse free over 6 months (86% vs. 70% for placebo; p=0.007, p=0.008 for each dose, respectively). MRI-calculated inflammatory disease activity was reduced by up to 80% (p<0.001, p<0.006). This double-blind, placebo-controlled study enrolled 281 patients with relapsing multiple sclerosis across 32 sites in Europe and Canada. Patients were randomized 1:1:1 to receive one of two doses of FTY720 (1.25 mg or 5 mg) or placebo for 6 months. Based on these results Novartis announced plans to initiate a phase III trial of the drug in Europe and North America in Q4 2005.

June 13, 2005

CeNeS Pharmaceuticals has announced positive results of a phase IIa trial of CNS 5161, their NMDA receptor antagonist under investigation for the treatment of neuropathic pain. Trial data met primary safety and tolerability endpoints, establishing a maximum tolerated dose (MTD) of 500 mcg. Incidence of hypertension occurred in the highest cohort (750 mcg), and enrollment in this cohort was not completed. No psychotomimetic effects were observed. Treatment at the MTD yielded trends towards improvement in pain levels a 2, 6 and 12 hours after the start of infusion, especially in patients with diabetic neuropathy. This multi-centre, double blind, cross-over, dose escalating proof- of-concept study enrolled 48 patients with etiologically varied neuropathic pain across sites in Europe. Subjects received one of four escalating regimens of the drug (125 mcg, 250 mcg, 500 mcg and 750 mcg) via intravenous infusion over 6 hours.

PharmaFrontiers has reported interim results of a pair of phase I/II trials of Tovaxin, their trivalent formulation of attenuated myelin-peptide reactive T cells (MRTCs) for the treatment of multiple sclerosis (MS). Preliminary results of the companys dose-escalation study indicated that administration of the drug produced reductions in MRTC levels, along with improvements in disability scores on the Kurtzke Expanded Disability Status Scale (EDSS) and disability neurological assessments for psychological scores Multiple Sclerosis 29 point Impact Scale (MSIS-29). Disease exacerbation was reported by 1 subject during the 6 month evaluable period of the study. Results of the company's extension study indicated a maintained a mean reduction in all categories of MRTCs at 3 and 6 months. This included a greater than 60% reduction in proteolipid protein T-cells. The dose- escalation study had treated 6 MS patients to date, who received a low- (6-9 million MTRCs) or mid-dose (30-45 million MRTCs) during a 6 month evaluation period. The extension study had enrolled 9 patients, who received one of two mid-dose-level doses of the drug during a 6 month evaluation. Based on these results, the company announced plans to initiate phase IIb/III trials by the end of 2005 or early 2006.

February 22, 2005

Elan and Biogen Idec have issued results of a phase III monotherapy trial of their recently approved monoclonal antibody Tysabri (natalizumab), for the treatment of multiple sclerosis. Study data met their primary efficacy endpoint, producing a significant 42% reduction in the risk of disability progression vs. to placebo (p<0.05). Efficacy was also noted in the prevention of clinical relapse, with a 67% reduction in risk, vs. placebo. Incidence of adverse events was consistent with expectations; overall rates of serious adverse events, including bacterial infections and systemic hypersensitivity, was 3.2% for Tysabri and 2.6% for placebo. This multi-center, placebo-controlled, double-blind study randomized 942 patients across 99 sites worldwide to receive either a 300 mg IV infusion dose of Tysabri (n=627) or placebo (n=315) once every 4 weeks for 2 years.

November 15, 2004

Biogen Idec and Elan have announced positive one-year data from a phase III trial of Antegren (natalizumab), which is currently under regulatory review in the US and Europe for the treatment of multiple sclerosis. Data from the ongoing study met their primary endpoint, significantly reducing clinical relapse rate in MS patients by 66% compared with placebo (p<0.05). The one-year data also met their secondary endpoints, including significant reductions in the number of new or enlarging T2-hyperintense lesions or gadolinium-enhancing lesions, and a significantly higher portion of patients who were relapse free, compared with placebo. This ongoing, two-year, double-blind, placebo-controlled study has enrolled 942 MS patients with a history of relapse across 99 clinical sites worldwide, who were randomized to receive either Antegren monotherapy or placebo. The companies announced that they expected full two-year data by the end of Q2, 2005.

May 3, 2004

The Immune Response Corporation announced positive results from a phase I/II trial investigating NeuroVax, a T-cell receptor for the treatment of multiple sclerosis. Results demonstrated the drug produced a peptide-specific immune response in 94% of the subjects treated. Data showed that 94% of subjects given NeuroVax had a significantly greater TCR vaccine response compared with 7% of subjects in the TCR/saline group and 0% in the adjuvant group. NeuroVax was well tolerated with no serious side effects reported. The three-armed, randomized study enrolled 37 subjects who received monthly injections for 24 weeks. Subjects were administered three TCR peptides with saline, NeuroVax or adjuvant alone. The study was discontinued early due to the high rate of response. Results were reported at the 54th annual meeting of the American Academy of Neurology in San Francisco.

April 19, 2004

Acorda Therapeutics reported mixed results from a phase II trial and two phase III trials investigating Fampridine-SR, a selective neuronal potassium channel blocker for the treatments of multiple sclerosis (MS) and chronic spinal cord injuries (SCI). Results from the placebo-controlled MS trial showed significant improvements in leg muscle strength as well as positive trends in walking speed improvement. Data showed a statistically significant improvement in its secondary endpoint, the Lower Extremity Manual Muscle Test, a standardized, 5-point manual assessment of strength. Results from the two SCI studies showed the data did not reach statistical significance in both primary endpoints, reduction of spasticity as measured by the Ashworth score, a validated, 5-point clinician assessment of spasticity and improvement in patients' Subject Global Impression rating. The most common adverse events with Fampridine were insomnia, paresthesias, dizziness and nausea.

September 29, 2003

Active Biotech reported positive results from a phase II trial investigating laquinimod (SAIK-MS), an immunomodulator for the treatment of multiple sclerosis (MS). Results showed a statistically significant reduction in disease activity in subjects treated with laquinimod, the studies primary endpoint. Data demonstrated that subjects treated with 0.3 mg/day of laquinimod showed a reduction in MRI activity of 30% compared with placebo. The 0.1 mg/day dose of laquinimod showed a decline in disease activity but was not statistically significant from the placebo group. The six-month, randomized, placebo-controlled, three-arm study enrolled 200 subjects at 20 sites in Sweden, the UK, the Netherlands and Russia. Subjects were treated orally with, 0.1 or 0.3 mg of laquinimod or placebo, daily for six months.

June 9, 2003

BioMS Medical reported positive results from a phase II trial investigating MBP8298, a synthetic peptide for the treatment of multiple sclerosis. The study had two parts, a two-year randomized double-blinded, placebo-controlled phase, followed by a two-year open label phase. Clinical progression was measured by changes in score on the Expanded Disability Status Scale (EDSS), as the primary clinical indicator. Results from part one showed none of the subjects taking MBP8298 progressed on EDSS as compared with 60% of the subjects on placebo. Subjects injected with MBP8298 showed a significant and sustained reduction in anti-MBP antibodies. During part one, subjects were given MBP8298 (500 mg) intravenously every 6 months. Results from part two showed only three of the subjects receiving MBP8298 (30%) had EDSS progression at 42 months. In addition, only five out of 16 subjects receiving MBP8298 showed EDSS progression as compared with nine out of 16 patients taking placebo, a 44% reduction. The complete four-year study enrolled 32 subjects with either primary or secondary progressive MS.

January 6, 2003

Elan and Biogen reported positive results from a phase II trial investigating Antegren (natalizumab) for the treatment of multiple sclerosis (MS). Data showed the drug reduced new inflammatory brain lesions and relapses. Results showed that subjects treated with natalizumab for six months had up to a 93% reduction in new gadolinium-enhancing lesions compared to subjects treated with placebo. A mean of 9.6 new enhancing lesions developed during the treatment period in the placebo group versus 0.7 and 1.1 in the low dose (3 mg/kg) and high dose (6 mg/kg) groups respectively. There was a 50% reduction in the number of subjects experiencing a relapse in the treatment group compared to the placebo group. The double blind, placebo-controlled study enrolled 213 subjects with MS at 26 sites in the U.S., Canada and the U.K.

July 8, 2002

A new data analysis suggests that early treatment with Avonex (interferon beta-1a) significantly reduces the rate of progression to clinically definite multiple sclerosis (CDMS) in high-risk subjects. Furthermore, Avonex provided a greater benefit to subjects with more abnormal disease activity as measured by MRI. The data analyzed was from a CHAMPS trial subgroup that included 91 subjects with high MRI lesion burden and evidence of inflammation. Results showed that the risk of progression to CDMS was decreased by 66% in the Avonex-treated group compared to those receiving placebo.

March 5, 2002

Results demonstrated that a phase II trial of Paxceed for multiple sclerosis did not meet its primary objective. The double-blind, randomized, placebo-controlled trial was conducted at nine centers in Canada. Subjects received either placebo or Paxceed (50 mg/m2 or 75 mg/m2) every four weeks for a total of six doses, with a follow-up period of 12 weeks. As the primary objective, the trial utilized magnetic resonance imaging to compare new lesion activity in Paxceed-treated subjects to activity in the control group. Angiotech Pharmaceuticals does not plan to pursue phase III development in this patient population.

This information does not represent a Lupus Research Institute endorsement of any listed study. It is merely a notice that the study is available. If you are presently under the care of a physician for lupus or other conditions, you should not disrupt your current program without discussing it with your doctor(s). Do not contact the Lupus Research Institute for information on these studies. Only contact the listed numbers. The Lupus Research Institute does not have any jurisdiction over or further involvement with these studies, other than to make people aware that they are being conducted.