Event - FDA adcom should provide clear vision of ThromboGenics's future
Following two big pipeline disappointments last month that helped to wipe over $100m off its market capitalisation, ThromboGenics is in definite need of some better news when its lead product, ocriplasmin, comes in front of an FDA advisory committee on July 26 for vitreomacular adhesion (VMA).
A good showing at the panel would go a long way to restoring confidence in the group. Following the failure of TB-402, its anti-clotting drug in phase IIb trials, and Roche's decision to hand back the rights to the solid tumour treatment TB-403, ocriplasmin remains the only drug of value in ThromboGenics’ very thin clinical pipeline.
Ocriplasmin is forecast to generate sales of $459m by 2018, making it worth $1bn to ThromboGenics, according to the EvaluatePharma NPV Analyzer. This equates to pretty much all of the group’s current market capitalisation, so failure is not an option.
|% of market cap||93%|
|Event type||FDA Advisory Committee|
|Date||July 26, 2012|
The need for success has been hammered home by events in June. ThromboGenics suffered the double blow of having to scrap TB-402 when phase IIb data pitting it against Xarelto following hip replacement surgery failed to show superiority. The drug also caused excessive bleeding in a number of patients, leading ThromboGenics and its partner BioInvent International to halt development.
On the same day ThromboGenics reported that Roche had handed back the global rights to its monoclonal antibody TB-403. The Swiss group described the decision as a prioritisation of resources. With the drug back in the hands of its original developers, it is partner BioInvent’s resources, or lack of them, that has analysts worried. The group has about $20m on its balance sheet and is unlikely to want to invest heavily in a drug that has limited proven effectiveness.
With such weight of expectation riding on it, luckily for Thrombogenics so far the signs for ocriplasmin have been good. The drug showed excellent efficacy in previous trials and in March landed Alcon as an ex-US marketing partner.
This handed the Belgian company not only €75m ($99m) up front, in a deal potentially worth €375m, but also valuable third-party validation of its product with a company that can grow sales in a region ThromboGenics would have found difficult (ThromboGenics scores Novartis and sets US in its sights, March 16, 2012). More recently, the drug was given priority review status by the FDA.
Widening the opportunity
There are also few options other than surgery for patients with the disorder, which causes the vitreous gel in the eye to stick to the macula at the centre of the retina, causing vision impairment and blindness. So despite the need for a J-code, lack of which could initially slow sales, the drug is expected to do well in this indication, with some analysts forecasting peak sales of up to $700m.
However, getting past the FDA panel and ultimately approval by the October 17 PDUFA date is just the start for ocriplasmin, which ThromboGenics to sell itself in the US. The plan is to develop the drug so it addresses much wider patient populations.
Currently it is being studied in the much bigger opportunities of diabetic macular oedema, retinal vein occlusion and age-related macular degeneration. It is this last indication that will be watched most eagerly, and ThromboGenics is expecting phase II data later this year. Given its action on preventing the gel in the eye from sticking to the macula, ultimately many believe the drug could be used in combination with Lucentis to improve outcomes in age-related macular degeneration.
So while a positive outcome at this month’s advisory panel and approval would be huge bonuses for ThromboGenics, the real hard work could be yet to come.