Biogen (BIIB) Reports Significant Data from BG-12 Phase 3 in Treating MS

Biogen Idec (NASDAQ: BIIB) announced new data from studies evaluating oral BG-12 (dimethyl fumarate), which provide further evidence supporting its strong clinical and radiological effects in people with relapsing-remitting multiple sclerosis (RRMS) and reinforce its favorable safety profile seen to date. These data were presented at the 28th Congress of the European Committee for the Treatment and Research of Multiple Sclerosis (ECTRIMS) in Lyon, France.

In a pre-specified analysis of integrated, or pooled, data from the Phase 3 DEFINE and CONFIRM studies, dimethyl fumarate showed statistically significant and clinically relevant effects in reducing multiple sclerosis (MS) relapses and progression of disability, as well as reductions in magnetic resonance imaging (MRI) measures of disease activity. In addition, interim safety data from a Phase 3 extension study indicate that continued exposure to dimethyl fumarate did not result in any new or worsening safety signals, and that its safety and tolerability profiles were consistent with previous studies.

Analyses of Pooled Phase 3 Efficacy Results

DEFINE and CONFIRM were randomized, double-blind studies that compared

the efficacy and safety of dimethyl fumarate 240 mg, administered twice
daily (BID) or three times daily (TID), to placebo over two years.
CONFIRM also included a reference comparator of glatiramer acetate (GA;
20 mg subcutaneous daily injection). A pooled analysis of the efficacy
data from more than 2,300 patients in these two studies was performed in
order to provide the medical community with a more precise estimate of
dimethyl fumarate’s treatment effects versus placebo on relapse,
progression and MRI outcomes.

Analyses of the pooled clinical efficacy results of DEFINE and CONFIRM
show that treatment with dimethyl fumarate led to significant reductions
in MS relapses and disease progression. At two years compared to
placebo, dimethyl fumarate significantly reduced:

• 
  Annualized relapse rate (ARR) by 49 percent for both BID and TID
  (p<0.0001 for both)
  


• 
  Proportion of patients who relapsed by 43 percent for BID and 47
  percent for TID (p<0.0001 for both)
  


• 
  Risk of 12-week confirmed disability progression, as measured by the
  Expanded Disability Status Scale (EDSS), by 32 percent for BID
  (p=0.0034) and 30 percent for TID (p=0.0059)
  

In MRI cohorts from DEFINE and CONFIRM, treatment with dimethyl fumarate
significantly improved MRI outcomes over two years compared to placebo
by reducing:

• 
  Mean number of new or newly enlarging T2-hyperintense lesions by 78
  percent for BID and 73 percent for TID (p<0.0001 for both)
  


• 
  Mean number of new non-enhancing T1-hypointense lesions by 65 percent
  for BID and 64 percent for TID (p<0.0001 for both)
  


• 
  Odds of having a greater number of gadolinium-enhancing (Gd+) lesions
  by 83 percent for BID and 70 percent for TID (p<0.0001 for both)
  

“These pooled results demonstrate that dimethyl fumarate had a
significant effect on measures that MS patients are acutely aware of –
the frequency of the relapses they experience and the progression of
disability,” said Ralf Gold, Ph.D., professor/chair of the Department of
Neurology at St. Josef-Hospital/Ruhr-University in Bochum, Germany. “As
a physician who treats patients with MS, the strong results observed in
the Phase 3 studies of dimethyl fumarate indicate that it may provide an
attractive combination of efficacy, safety and tolerability.”

Pooled DEFINE and CONFIRM efficacy data are included in one platform and
two poster presentations:

• 
  Clinical Effects of BG-12 in Subgroups of Patients with
  Relapsing-Remitting Multiple Sclerosis: An Integrated Analysis of the
  Phase 3 DEFINE and CONFIRM Studies was available for viewing on
  Thursday, Oct. 11, 2012 from 3:30-5:00 p.m. CEST
  


• 
  Clinical Efficacy of BG-12 in Relapsing-Remitting Multiple
  Sclerosis: An Integrated Analysis of the Phase 3 DEFINE and CONFIRM
  Studies will be presented by Prof. Ralf Gold on Friday, Oct. 12,
  2012�at 2:40 p.m. CEST
  


• 
  Effects of BG-12 on Magnetic Resonance Imaging Outcomes in
  Relapsing-Remitting Multiple Sclerosis: An Integrated Analysis of the
  Phase 3 DEFINE and CONFIRM Studies will be available for viewing
  on Friday, Oct. 12, 2012 from 3:30-5:00 p.m. CEST
  

Pooled Safety Results

Safety and tolerability results were pooled from three
placebo-controlled studies (DEFINE; CONFIRM; and a Phase 2 dose-ranging
study) involving more than 2,400 patients who had received placebo or
240 mg of dimethyl fumarate twice or three times a day. These pooled
data were consistent with results presented at previous medical
conferences. The overall incidence of adverse events (AEs: 92%
placebo, 95% BID, 93% TID) and serious adverse events (SAEs: 21%
placebo, 18% BID, 15% TID) was similar for all treatment groups. The
most common AEs associated with dimethyl fumarate treatment were
flushing and gastrointestinal (GI) events; the incidence of these events
was highest during the first month and decreased thereafter.

Mean lymphocyte counts decreased during the first year of dimethyl
fumarate treatment and then plateaued, staying within normal limits
throughout the entire treatment period. The incidence of hepatic and
renal events was comparable among all study groups. The incidence of
serious infections (≤2%) and malignancies (<1%) was low and balanced
across the study groups. There were no opportunistic infections.

Pooled DEFINE and CONFIRM safety data are included in one poster
presentation:

• 
  Safety and Tolerability of BG-12 in Patients with
  Relapsing-Remitting Multiple Sclerosis: An Integrated Analysis of the
  Placebo-Controlled Studies was available for viewing on Thursday,
  Oct. 11 from 3:30-5:00 p.m. CEST
  

Interim Extension Study Safety Results

Positive interim safety data from ENDORSE, a dose-blind, multi-center,
Phase 3 extension study evaluating the long-term safety and efficacy of
dimethyl fumarate 240 mg BID and TID, will also be presented at the
congress. At the time of analysis, 1,736 patients with RRMS who
completed the DEFINE study or the CONFIRM study had been dosed in
ENDORSE.

Patients who received two years of dimethyl fumarate in DEFINE and
CONFIRM continued on the same dimethyl fumarate dose (BID or TID) in
ENDORSE. Patients who had previously received placebo or GA (CONFIRM
only) were randomized 1:1 to dimethyl fumarate 240 mg BID or TID.

At the time of analysis, more than half of the patients in ENDORSE had
been followed for more than one year. Overall, the safety profile for
those first exposed to dimethyl fumarate in ENDORSE was consistent with
the safety results established in the DEFINE and CONFIRM studies. There
were no new safety signals observed in patients who had previously been
on dimethyl fumarate.

In ENDORSE, the incidence of serious infections was low (≤2%) in all
treatment groups and there were no opportunistic infections. There was
no increased risk of infection in patients treated long-term with
dimethyl fumarate. The overall incidence of malignancies was low (<1%).
The types and frequency of malignancies that were observed in the
treatment groups were expected in the population under study, and no
specific pattern of malignancies was observed.

These data are included in one poster presentation:

• 
  Long-Term Safety and Tolerability of Oral BG-12 (Dimethyl Fumarate)
  in Relapsing-Remitting Multiple Sclerosis: Interim Results from ENDORSE
  will be available for viewing on Friday, Oct. 12 from 3:30-5:00 p.m.
  CEST (late-breaking news)
  

The data presented at ECTRIMS were included in Biogen Idec’s regulatory
submissions for dimethyl fumarate around the world.

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