FDA Grants Aplidin Orphan Drug Status for Acute Lymphoblastic Leukaemia.

MADRID, Spain -- PharmaMar announced today that Aplidin(R)(a), its second marine-derived compound in clinical development, has been granted orphan drug status by the US Food and Drug Administration (FDA) for the treatment of acute lymphoblastic leukaemia (ALL).

Orphan drug designation is awarded to companies developing therapies for the treatment of diseases that affect fewer than 200,000 individuals in the US, at the time of application. Further criteria include the ability of the product to address an unmet medical need where no approved treatment option exists or provide significant benefit over available treatments. This designation would grant PharmaMar a special seven-year period of market exclusivity in the US for Aplidin in this indication, subject to successful marketing authorization. Aplidin was awarded orphan drug status for ALL by the European Commission in July 2003.

Acute lymphoblastic leukaemia (ALL) is a malignant disease of the bone marrow. This type of leukaemia is the leading cause of death by cancer under the age of 35 years. It is estimated that more than 40,000 people are living with ALL in US(1), approximately 3,830 new cases will be diagnosed in 2004(2) and that about 1,500 deaths will occur due to the disease in the same year(3).

Commenting on the orphan drug designation, Dr Miguel Angel Izquierdo, Director of Clinical Development at PharmaMar, said:

"The FDA and EC have recognised the urgent need for new therapies to treat this life threatening disease. Patients with ALL have a poor prognosis, as current treatment options are often ineffective in the long term. We are very encouraged with the activity showed in the clinic to date in other tumour types, together with the compound's favourable side-effect profile. We look forward to continuing with the development of Aplidin as a potential treatment for ALL patients, based on the strong preclinical evidence of activity seen in ALL and other haematological malignancies."

About Aplidin(R)

Aplidin(a) is a novel antitumour agent derived from the marine tunicate Aplidium albicans. It induces rapid and persistent activation of apoptosis combined with blocking of cell division in the G1/G2 phase of the cell cycle in tumour cells. It also inhibits the secretion of vascular endothelial growth factor (VEGF), a crucial protein involved in the vascularization and growth of a number of tumours, and the expression of the VEGF receptor 1 (VEGFR1).

Aplidin is currently in clinical development in Europe and Canada for the treatment of solid tumours, haematological malignancies and paediatric tumours. Phase II trials are ongoing for melanoma, colorectal, renal, lung (NSCLC and SCLC), medullary thyroid, head and neck and pancreatic carcinomas. Aplidin is also in Phase I in paediatric trials for solid and haematological tumours. PharmaMar started Phase I paediatric trials in solid tumours and leukaemias (including ALL) in Europe in March 2004. European Phase II trials in adults with haematological malignancies are planned to commence this year.

(a) Aplidin(R) is a PharmaMar registered trademark.

PharmaMar

PharmaMar is a biopharmaceutical company, advancing cancer care through the discovery and development of innovative marine-derived medicines. PharmaMar's clinical portfolio currently includes Yondelis(TM) in phase II clinical trials (co-developed with Johnson & Johnson Pharmaceutical Research & Development), designated Orphan Drug for STS by the EMEA in 2001 and Orphan Drug for ovarian cancer in 2003; Aplidin(R), in phase II, designated Orphan Drug for acute lymphoblastic leukaemia in 2003; Kahalalide F in phase II and ES-285 in phase I clinical trials.

PharmaMar, based in Madrid, Spain, is a subsidiary of the Zeltia Group (Spanish stock exchange: ZEL.MC; Bloomberg: ZEL SM; Reuters: ZEL.MC). PharmaMar can be found on the Web at http://www.pharmamar.com.

(1) Source: SEER Cancer Statistics Review 2004

(2) Source: Cancer Facts and Figures 2004, ACS 

(3) Source: Cancer Facts and Figures 2004, ACS