Confirm shows BG-12 potential

Biogen Idec has not put a foot wrong in its development of oral MS candidate BG-12, and topline data from its second pivotal trial was no different. The Confirm study released today verified its promising efficacy even when compared to Teva’s established injectable treatment Copaxone, clearing the way for regulatory submission in the first half of 2012.

Whilst the data came a week too late to upstage competitors at a major medical meeting, as happened with the Define trial earlier this year, the effect was similarly strong – shares rose 10% to $117 in early trading today, hitting a record high (Biogen steals Teva's thunder with BG-12 data, April 26, 2011). With fewer safety worries than the first MS pill, Novartis’ Gilenya, Biogen executives now are positioning BG-12 as a first-line single-agent treatment.

Looks first line

The Confirm trial in 1,200 patients pitted twice and three-times daily dosages of BG-12 against placebo, with a Copaxone reference arm, in a two-year study of MS relapses (Event – BG-12 needs to confirm blockbuster potential, September 21, 2011). It found the Biogen compound reduced the annualised relapse rate 44% in the twice-daily arm and 51% in the three-times daily arm when compared to placebo, whilst Copaxone reduced the relapse rate by 29%.

Although the trial was not powered to detect superiority for BG-12, observers noted that the findings support the growing belief that it is a more effective treatment than Copaxone or interferons like the Massachusetts group’s own Avonex.

With serious adverse events and discontinuations similar to placebo and Copaxone, and the results mirroring the first pivotal Define trial, observers believe the overall profile of BG-12 is of one that will take significant market share – compared to Gilenya, which has a side effect of slowing the heart rate and requires six hours of observation following the first dose.

“It looks like a first line therapy to me,” Al Sandrock, Biogen’s senior vice president of neurology research and development, said in a call with investors.

Disability progression

If there was a dark spot in the Confirm findings, it was a failure to show a statistically significant reduction in 12-week disability progression, as measured by the expanded disability status scale. Biogen executives noted that there was a trend toward significant improvement and predicted that the miss would not be a barrier to approval, pointing to Gilenya’s approval after it failed to arrest disability progression in its trial against Avonex.

Pointing to the disability findings, UBS analyst Gbola Amusa was one of the few critical voices on BG-12, arguing in a research note that Gilenya, by comparison, showed efficacy on that measure, and in addition was able to demonstrate superiority on relapse rate when compared to Avonex. Thus, Dr Amusa said, Gilenya is a more effective oral therapy.

With BG-12 already Biogen’s most valuable product, with a net present value of $7.91bn, and biggest growth driver through 2016, the strong uplift in the company's stock today is not surprising. What will be interesting to watch in coming weeks is if equity analysts, who have so far viewed it as a strong second-line contender, will now adjust their views in light of Confirm and see it as a first line treatment, as Biogen leaders and even some in the neurology community are doing (ECTRIMS - Expectations build for BG-12 as safety impresses, October 21, 2011).

If so, an increase in BG-12’s already healthy sales forecasts can be expected. Today, Copaxone is forecast to hold on as the biggest selling agent in the space in 2016 at $2.59bn, with BG-12 fifth at $1.83bn (Copaxone just holds MS top spot in 2016 as oral therapies grow, October 19, 2011).

Given regulatory timetables, a BG-12 launch almost certainly will not happen until early 2013, and much can yet go wrong – safety data have not been detailed and the disability findings could throw up unexpected barriers. But the Confirm data provide another sign that this is a drug with the potential to transform the MS space.

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