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Orphan Drug Companies At The 15th Annual BIO CEO And Investor Conference: Protalix BioTherapeutics

February 19, 2013
Protalix BioTherapeutics' Elelyso is produced using inexpensive and easily grown carrot cells. Kander via Wikimedia.

This is the second of a series of Orphan Druganaut Blog Posts that will recap the orphan drug companies at the 15th Annual BIO CEO & Investor Conference, held February 11 – 12, 2013, in New York City. This Blog Post reviews 1 of the 3 companies competing in the Enzyme Replacement Therapy (ERT) Gaucher Disease market, Protalix BioTherapeutics.

Photograph from Kander | Wikimedia Commons.

On the second day of the meeting, Tuesday, February 12, 2013, Protalix BioTherapeutics’ President & CEO, Dr. David Aviezer, gives a presentation (Webcast available).

Protalix BioTherapeutics partners with Pfizer for orphan drug Elelyso (taliglucerase alfa) for the treatment of Gaucher Disease. It is Elelyso’s “unique plant cell-based manufacturing system that is making the small Israeli biotech potentially worth $1 billion”. Per a recent Bloomberg article, Pfizer has expressed interest in buying Protalix BioTherapeutics. This was reported by the Israeli financial newspaper Calcalist, without identifying the source of the information. Calalist reported that Novartis and Merck also may want to buy the company.

Elelyso is the first drug to win FDA approval for a drug made from a genetically engineered plant – it is made from carrot cells. The drug is produced using inexpensive and easily grown carrot cells without use of expensive fermentation vats and without the possibility of viral contamination. Genzyme’s production of Cerezyme was halted in June 2009 after a virus contaminated its Massachusetts facility, resulting in a yearlong, worldwide shortage of the drug.

Dr. Aviezer emphasizes the company’s unique plant cell-based technology, ProCellEx Technology.  ProCellEx is the 1st technology for producing proteins in plant cells. It is this unique technology that produces the company’s first marketed drug, Elelyso. The advantages of the ProCellEx Technology are identified :

  1. Safety – because it uses carrot cells and not mammalian cells & thus  can avoid contamination from viruses
  2. Cost-effective
  3. Potentially permits an entry into certain patent protected markets.

Protalix Therapeutics is using the ProCellEx Technology to make additional proteins for their pipeline. One new canidate is PRX-102, which is in Phase I/II clinical trials for the treatment of Fabry Disease. Dr. Aviezer reviews the regulatory timeline for Elelyso :

  1. FDA approval in May 2012
  2. EMA CHMP Opinion in June 2012 recommends against approval due to Shire plc’s orphan drug exclusivity for Vpriv
  3. In September 2012, approval by the Israeli Ministry of Health
  4. In November 2012, the EC adopts CHMP’s recommendation
  5. Expects approval in Brazil soon.

Dr. Aviezer reviews other positive features about Elelyso : the price is 25% below Cerezyme cost and plans are in place to minimize supply disruptions.

The Pfizer/Protalix BioTherapeutics Commercialization Agreement is outlined :

  1. Protalix BioTherapeutics to manufacture Elelyso
  2. Pfizer has exclusive worldwide rights outside of Israel
  3. Protalix BioTherapeutics has commercialization rights in Israel
  4. Pfizer/Protalix BioTherapeutics to share net profits and losses 60%/40%.

Three posters on clinical trial results for Elelyso are presented at the Poster Session, at the 9th Annual Meeting of the Lysosomal Disease Network (LDN): WORLD Symposium, February 13-15, 2013 :

  1. Long-Term Safety and Efficacy Data of Taliglucerase Alfa, a Plant Cell–Expressed Recombinant Glucocerebrosidase, in the Treatment of Naïve Gaucher Disease Patients: 36-Month Results
  2. A Multicenter, Double-Blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Patients with Gaucher Disease*
  3. Plant Cell–Expressed Recombinant Glucocerebrosidase: Taliglucerase Alfa as Therapy for Gaucher Disease in Adult Patients Previously Treated With Imiglucerase: 24-Month Results.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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