Zymenex in Phase 3 clinical trial in patients with the rare disease alpha-Mannosidosis

Phase 2 trials demonstrated that the enzyme is clinically effective, safe and well tolerated and a placebo-controlled Phase 3 clinical trial is ongoing.

Use of the biotechnologically derived human enzyme product rhLAMAN (LamazymTM), produced by the Danish biotech company Zymenex and developed for the treatment of patients suffering from alpha-Mannosidosis, has been successfully evaluated in a 12 month dose/response Phase 2a clinical trial and a 6 month follow-up Phase 2b clinical trial in 9 patients. The aim of the trials, which took place at the Department of Clinical Genetics, Copenhagen University Hospital, Denmark, was to confirm the chosen weekly minimum effective dose of 1 mg/kg and to validate the clinical effect of repeated i.v dosing.

Positive changes from baseline after 18 months of dosing have been demonstrated in the primary measured clinical and biochemical endpoints.

Statistically significant changes were demonstrated in oligosaccharide levels in serum, in the functional tests: 3 Minute Stair Climb (+39 steps, p=0.004) and 6 Minute Walk Test (+71 meters, p=0.009) as well as in the Leiter-R test of intellectual capacity (+0.5, p=0.022). The forced vital capacity (FVC), lung function test showed a marked increase (+9.2%, p=0.157) .

A Multi-Center, Double-Blind, Randomized and Placebo-Controlled trial in 25 patients with alpha-Mannosidosis, receiving a dose of 1 mg/kg weekly, is ongoing in Denmark, Germany, France, Spain, Belgium and Norway. The dosing takes place in the patient's home countries and all evaluations are performed at the Department of Clinical Genetics, Copenhagen University Hospital, in Denmark.

Alpha-Mannosidosis is a rare inherited disease, with a prevalence of 1 in 500,000 people. The disease is due to a rare inborn disorder that causes a lack of the lysosomal enzyme α-Mannosidase, resulting in increased levels of oligosaccharides and in severe skeletal- and muscle changes, hearing loss and recurrent infections and mental retardation. The disease belongs to the group of lysosomal disorders, for which Zymenex has a documented research competence.

Zymenex A/S www.zymenex.com is a Danish biopharmaceutical company, founded in 1998, with headquarters in Hilleroed north of Copenhagen, Denmark and research laboratories in Stockholm, Sweden. The company is focused on research and development of pharmaceutical products for the treatment of rare, genetic diseases, for which there is no treatment today. Lamazym has received Orphan Drug Designation in both Europe and the US. The clinical trials are partly sponsored by an EU Framework 7 grant, www.alpha-man.eu.

Denmark, Hilleroed, January 2015

Further information:

Dr. Jens Fogh, DVM, President, CEO
Zymenex A/S
Roskildevej 12 C
3400 Hilleroed
Denmark
Tel +45 48250054
Fax +45 48251054

Dr. Allan Meldgaard Lund, MD, DMSc, Chief Physician
Department of Clinical Genetics
Center for Inherited Metabolic Diseases
Juliane Marie Center
Copenhagen University Hospital, Rigshospitalet
Blegdamsvej 9
2100 Copenhagen
Denmark
Tel + 45 35452793

Zymenex A/S Roskildevej 12 C
3400 Hillerød, DK
Tel  +45 48250054
Fax  +45 48251054
Affiliate Sweden Agavägen 58
181 55 Lidingö, S
Tel  +46 8 7670203
Fax  +46 8 7670233

© 2004 Zymenex A/S