Participants 2a & 2b
Partner 2a (SME): Zymenex A/S, Roskildevej 12C, DK-3400 Hilleroed, Denmark is a Danish/Swedish biotech company that was established on June 3, 1998. The company is registered in Denmark and has it’s Headquarters in Hilleroed, Denmark and R&D laboratories in Stockholm, Sweden. (Team leader/Responsible Scientist: Dr. Jens Fogh). Key skills: Enzyme replacement
Zymenex is a Danish-Swedish based biopharmaceutical Company focused on the development and commercialization of novel enzyme therapies for the treatment of life-threatening, rare genetic diseases. The Company is leveraging its expertise in the area of enzyme replacement therapies to develop therapies for the heme synthesis disorder Acute Intermittent Porphyria (AIP) and the lysosomal storage disorders Metachromatic Leukodystrophy (MLD) and Alpha-Mannosidosis (MAN). These products address well defined patient populations for whom there are no sufficient therapies available and are therefore eligible for the benefits of Orphan Drug Designation and approval.
Zymenex’s lead product, Porphozym™, is an enzyme replacement therapy currently in international Phase IIb clinical trials for the treatment of AIP. Porphozym™ has been granted Orphan Drug designation in the US and in the EU. The Company’s preclinical pipeline includes enzyme replacement therapies Arylsulfatase A (rhASA) and a-Mannosidase (rhLAMAN) for the lysosomal storage disorders MLD and MAN. The human enzymes have been cloned and expressed and the first compound for MLD has been granted Orphan Drug designation in the EU, is in toxicology testing and is expected to enter clinical trials in 2006. Zymenex has support from the Danish investor groups BankInvest A/S, Vækstfonden and Pen-Sam.
Members involved: To be supported by the project: One senior technician who will be responsible for the development of the cell fermentation process and production of fermented enzyme and responsible for the process development and analysis (36 pm). Jens Fogh will be involved in coordination and supervidion of the enzyme production (11 pm)
TOTAL EXPECTED EFFORT: 47 pm
Partner 2b : Zymenex, A/S, Roskildevej 12C, DK-3400 Hillerod, DENMARK (Team leader/Responsible Scientist: Dr. Christian Meyer). Key skills: Recombinant enzyme development – pre-clinical clinical and regulatory
Company background and technical information: Zymenex (formetly: HemeBiotech) is a Danish-Swedish based biopharmaceutical Company focused on the development and commercialization of novel enzyme therapies for the treatment of life-threatening, rare genetic diseases (see also the information about the company for partner 2a).
Capacity to contribute to the project: Partner 2b has the expertise in pre-clinical and clinical development, project management and regulatory filing and experience in pre-clinical (GLP)- and clinical (GCP) development and regulatory documentation necessary to bring a pharmaceutical product from an idea to regulatory approval in both EU and US. All the necessary equipment and facilities to perform these tasks related to the development of an enzyme replacement therapy of a-Mannosidosis are available.
Members involved: To be supported by the project: One scientist to develop the protocol and regulatory/Ethics Committee activities.One scientist will set up the clinical sites, corresponding hospital- and biochemical laboratories in collaboration with Partners 1,6,7,8,9,11 and 12 and manage the logistics between these sites (36 pm).
Other contributing members of the laboratory: Dr. Christian Meyer ( 8 pm) from Zymenex will be overall responsible for project management.
Collaborators: Central contract laboratories for analyses of samples in the natural history study will be defined later when the protocol for the study is finalized. Contract Laboratories manufacturing organization: It is standard procedure to collaborate with a contract laboratorie in performing sample testing, since the CRO has the approvals necessary to perform the analytical work in human samples, equipment, the personnel and procedures necessary to perform the analytical work according to authority requirements. The CRO contract costs for the analytical tests will be 50.000 EURO.
TOTAL EXPECTED EFFORT: 44 pm