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ThromboGenics Signs Potential $494M Ex-U.S. Ocriplasmin Deal

By Nuala Moran
Staff Writer

LONDON – ThromboGenics NV is getting €75 million (US$98.4 million) up front as part of a €375 million-plus-royalties deal signed last week with leading eye care specialist Alcon Inc. for the ex-U.S. rights to ocriplasmin, providing the cash ThromboGenics needs to go it alone in commercializing the vitreous macular adhesion (VMA) treatment in the U.S.

Ocriplasmin, a recombinant form of human plasmin, has been filed with the European Medicines Agency, and ThromboGenics is due to receive a further €90 million milestone from Alcon on European approval and launch, which is expected within the next year.

Alongside plans to launch ocriplasmin in 40 countries worldwide, Alcon, a division of Novartis AG, also intends to widen the indications to include diabetic macular edema and age-related macular degeneration (AMD).

All future clinical development costs will be shared 50:50 between the two partners, and a joint committee will be set up between Alcon and Leuven, Belgium-based ThromboGenics to oversee the program. A second committee will make joint decisions on marketing and sales.

"This is definitely going to be transformational for ThromboGenics," said CEO Patrik de Haes. "It provides significant additional financial resources, with an up-front payment and highly attractive royalty payments," he told investors on a conference call held to discuss the deal.

The royalty rate is "commensurate" with a product that has been filed for regulatory approval after successfully completing Phase III, de Haes said.

Jan de Kerpel, analyst at KBC Securities, said he believes that should be translated as levels anywhere between 25 percent and 30 percent of net sales.

ThromboGenics' current estimates are that 60 percent of the market for ocriplasmin is in the U.S., with 270,000 eligible patients.

The company has a senior management team in place and with the signing of the deal can move on with building the U.S. sales force.

The biotech is in the unusual position in the U.S. of having been granted priority review for ocriplasmin even though it did not request it.

After it submitted a biologics license application (BLA) in December 2011, the FDA told the company it wanted to grant priority review.

That means ThromboGenics must withdraw the BLA and submit a new application. The company expects to do so in April, leading to approval and launch in the first quarter of 2013. (See BioWorld International, Feb. 8, 2012.)

If all goes according to plans, that will coincide with the first European launch by Alcon. Although Alcon has marketing exclusivity in Europe, ThromboGenics has retained some rights to be involved in negotiating market access and preparing sales materials in the top five national markets of Germany, France, the UK, Spain and Italy, and in its home market in Belgium. "This will allow us to create a small operational team, which will be the core of a future ophthalmology franchise," de Haes said.

KBC's de Kerpel said having a partner with the marketing machine and expertise in ophthalmology of Alcon means ocriplasmin "is further de-risked for the difficult European market."

VMA is caused when the vitreous gel in the cavity of the eye contracts, pulling away from the retina. That causes visual distortion that can lead to central field blindness if the gel does not detach completely and continues to pull on the retina. Currently, the only treatment is vitrectomy surgery to remove the gel, but de Haes noted that is only performed after watching and waiting to see if the gel becomes completely detached of its own accord, resolving the problem.

Its ability to dissolve the fibrils that bind the gel to the retina means that ocriplasmin could be used to treat patients at an earlier stage, de Haes said.

"The product could be transformational because it could be used as a pharmacological treatment early on. If a patient has symptomatic VMA, don't wait; treat with an injection of ocriplasmin," he said.

If that does not cure the problem, the patient then can be referred for surgery at an earlier stage of the disease process than is currently the case, leading to a better outcome.

Although Alcon sells surgical vitrectomy kits, that would not be incompatible with selling ocriplasmin, since it will push the product toward earlier-stage patients who are not treated currently.

Alcon, and its parent Basel, Switzerland-based Novartis, also would appear to have competing interests in AMD, where its anti-VEGF drug Lucentis (ranibizumab) is a big seller.

de Haes noted that 30 percent of cases of AMD are complicated by an adhesion, and ocriplasmin will be positioned as a companion treatment for Lucentis in that subset of patients.

In the Phase III trials of ocriplasmin, 652 VMA patients were treated in 90 centers in the U.S. and Europe.

In total, 26.4 percent of the 465 patients treated with ocriplasmin achieved resolution of their VMA at 28 days, compared to 10.2 percent of the 182 patients who received a placebo injection, a highly statistically significant result (p = 0.000002).