Pharming bolsters transgenic platform

- Last updated on GMT

Pharming of the Netherlands has modified two of its commercial
relationships in a move that should accelerate its activities in
developing therapeutic proteins that are made in transgenic
animals, writes Phil Taylor.

The move will enable it to bring more projects into full development and should speed up the commercialisation of lactoferrin, a transgenically-produced protein that has applications across the pharmaceutical and nutraceutical sectors.

After a difficult period over the last few years, culminating with a slide into receivership in 2001, Pharming had narrowed down its business to focus on one product, a complement C1 inhibitor made in the milk of transgenic rabbits that is currently in Phase III testing for patients with a rare genetic disorder called hereditary angioedema.

But in January, the company completed a refinancing package that has brought in the funds needed to allow it to broaden its focus.

"Before the refinancing, Pharming was 90 per cent focused on the C1 inhibitor project,"​ said Frank Pieper, the company's chief scientific and technical officer. "But now we feel it is important to look at a much wider pipeline,"​ he told In-Pharmatechnologist.com​.

To facilitate this, Pharming​ has renegotiated the terms of a licensing agreement with Infigen, a company which has developed a nuclear transfer technology that the Dutch company been using to generate transgenic cattle. The original terms of the agreement limited Pharming's use of the technology to its existing pipeline projects, but it has now modified this to allow additional projects to be undertaken, both in cattle and other species.

The revamped agreement means that Infigen​ has foregone rights to various milestone payments in return for upfront licensing fees, which suits the company as it is in the throes of a downsizing programme that will see 'substantially all' of its staff laid off.

Pharming and Infigen are now in the midst of a due diligence period but expect the new agreement to be in place in July.

Gains control of ProBio

Meanwhile, Pharming has increased its stake in Australian company ProBio, which also has a technology platform in transgenic protein production, from 20 to 45 per cent. This gives it a controlling interest in the company which should help it accelerate collaborative projects, most notably the commercialisation of recombinant human lactoferrin in Asia.

Transgenically-produced lactoferrin is being positioned in the first instance as an ingredient for health foods or nutraceuticals. It is a protein found naturally in milk and other endocrine secretions and is believed to play a role in stimulating the body's immune system to fight cancer and infections, and protect against asthma and other allergic diseases.

In addition to its ambitions for the product in Asia, the company is hoping to get GRAS (Generally Recognised As Safe) approval in the US in 2005, clearing the way for it to be used there in food applications.

On a more general note, Pieper told In-PharmaTechnologist.com​ that he believes transgenic production of proteins in animals is on the cusp of proving itself as a commercially-viable technology.

Despite the problems encountered by Pharming and Scottish company PPL​, products based on transgenic animals look set to reach the market within the next year. Pharming expects to launch its own product in 2005, while another player, GTC Biotherapeutics​, has already filed for approval of goat-produced antithrombin in Europe as a treatment for hereditary antithrombin deficiency.

"The market will respond strongly to getting an approved product based on this technology to market,"​ he predicted. And despite the work being done on alternative transgenic sources, notably plants, he feels animals are the only solution for some proteins.

Transgenic production in animals lends itself to a specific niche, namely the production of bulk complex proteins for human uses that require post-translational modifications that are not achievable with plant systems or mammalian cell culture, he noted.

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