Protalix Biotherapeutics is attempting to use its US FDA-approved Gaucher's disease treatment as a proof of concept and springboard for its plant-based cell manufacturing technology.
The plant cell-based manufacturing technology – which Protalix claims can produce protein drugs more cheaply and safely than mammalian cell culture systems - is being used to develop a number of other enzyme replacement therapies and drugs.
Current projects include a treatment for Fabry disease; an oral Gaucher's disease drug; a biodefense program; and a plant cell version of Amgen’s Enbrel for the treatment of some immune diseases. None of the treatments have entered Phase III trials.
CEO David Aviezer told attendees at the BIO CEO and Investor Conference in New York on Tuesday that - in addition to enabling safer and more cost effective manufacturing – plant cells provide an effective way of delivering protein–based therapies.
Protalix’s candidate oral Gaucher’s drug is contained within lyophilized carrot cells that the patient takes as a drink. The cellulose in the cell’s walls prevents degradation as they pass through the digestive tract enabling the protein to reach the bloodstream in an active form.
Protalix has priced its approved Gaucher’s treatment - Elelyso, the first approved biologic to be created through plant cells -- at 25% less than the price for Genzyme’s Cerezyme, Aviezer said.
He also said the company expects approval in Brazil soon. Brazilian regulators recently completed a good manufacturing practice audit of its manufacturing facility. The enzyme replacement therapy was also approved for use in Israel in September and is expected to be covered by insurers there beginning this year.
Aviezer said there are other marketing applications pending but he did not specify in which countries.
Protalix will need to find additional markets for the drug after the European Commission ruled in November 2012 that it could not market the drug in the EU because Shire’s Gaucher’s treatment velaglucerase alfa received orphan marketing status and has exclusivity until 2020.
The company is also looking to leverage its plant technology with other companies looking for an innovative manufacturing approach. Earlier this month, Protalix confirmed that it engaged Citigroup to assist in reviewing an array of product partnering, technology sharing and other strategic alternatives.
The prospect of a new partnership comes on the heels of one with Pfizer for Elelyso, which the companies expect to compete in a $1.2b (€896m) market. That partnership gives Protalix a 40% share of the drug and allows it to maintain the manufacturing rights.
As far as near-term financials, the public company had $50m in cash as of Sept. 30, 2012, Aviezer added.