BioWorld International Correspondent

Start-up firm Vivendy Therapeutics AG raised CHF17 million (US$15.5 million) in a Series A round to finance development of an enzyme replacement therapy for Morbus Morquio Type A, a rare lysosomal storage disorder characterized by severely restricted growth, skeletal malformation, corneal opacity and hearing problems.

"This funding is entirely and utterly focused on the development of enzyme replacement therapy for Morbus Morquio," Roland Toder, CEO of Basel, Switzerland-based Vivendy, told BioWorld International.

The company estimates that between 1,200 and 1,500 people in the U.S., Europe and Japan have the condition, of which about 800 would be eligible for treatment. The others may not benefit because their condition is too advanced. "It's truly an orphan drug," Toder said.

An autosomal recessive condition, also known as mucopolysaccharidosis (MPS) Type IV A, Morbus Morquio is caused by the absence of the lysosomal enzyme (N-acetyl)-6-sulphate sulphatase (GALNS), which catalyzes the degradation of keratan sulphate, an important component of cartilage and the cornea.

It is one of several mucopolysaccharidoses, which are caused by the absence of one or more of the 11 lysosomal enzymes involved in degrading glycosaminoglycans (also called mucopolysaccharides).

"The really good news is we are a boring 'me-too' [company]," Toder said. Enzyme replacement therapies for several MPS indications already have gained approval. Aldurazyme (laronidase), marketed by Genzyme Corp., of Cambridge, Mass, is approved for treating patients with Hurler and Hurler-Scheie forms of MPS I. Elaprase (idursulfase), marketed by Basingstoke, UK-based Shire Pharmaceuticals Group plc, is approved for treatment of MPS II or Hunter syndrome. And Naglazyme (galsulfase), marketed by Novato, Calif.-based BioMarin Pharmaceutical Inc., is approved for treating MPS VI.

Those with MPS Type IV A live on average to 25 years, although those with less severe forms of the condition can live for several decades more. In addition to providing a survival benefit, enzyme replacement therapy also could lead to quality-of-life improvements and reductions in pain associated with the condition.

Vivendy's program is based on intellectual property created by Shinju Tomatsu at the St. Louis University School of Medicine. Tomatsu has developed a modified recombinant version of GALNS, which is more stable than the native form and which therefore should provide greater activity when it penetrates target tissues, such as bone and the cornea. It will be administered intravenously on a weekly basis.

"We are planning to go into the clinic in two years - in 2010," Toder said. Vivendy is upscaling production of the enzyme - in Chinese hamster ovary cells - at present. It also is consulting the entire Morbus Morquio clinical community, he said, to develop endpoints that can be deployed in clinical trials.

Measures, such as walking and stair climbing tests used in trials of previously approved MPS therapies, are likely to be included, along with other parameters.

The company will conduct a Phase I/II trial initially, following which it aims to move directly to a Phase III registration trial, Toder said.

In all, it will need to recruit between 70 and 80 people with the condition, and the clinical program will cost around CHF28 million to run. Vivendy aims to gain product approval by 2013.

Basel-based BioMedInvest AG led the investment. LSP Life Sciences Partners, of Amsterdam, The Netherlands, and Munich, Germany-based TVM Capital were co-lead investors.

Vivendy was spun out from Inotech Biotechnologies AG, a Basel-based contract manufacturer of biopharmaceuticals. Inotech retains a minor equity stake in the company.

In other financing news:

• Action Pharma A/S, of Aarhus, Denmark, raised €15 million (US$21.8 million) in a Series D round to fund development of its pipeline of drugs for treating Type II diabetes, organ failure and other conditions. Global Life Science Ventures, of Zug, Switzerland, led the round. Scandinavian Life Science Venture, of Stockholm, Sweden, also joined as a new investor. Existing backers InnovationsKapital, of Gothenburg, Sweden; Sunstone Capital A/S, of Copenhagen, Denmark; and Inventure Capital A/S also participated.