Diagnostics & Imaging Week Contributing Writer
And D&IWs

Providing further evidence of the ongoing revival of investor interest in gene therapy, Amsterdam Molecular Therapeutics (AMT; Amsterdam, the Netherlands) raised EUR 22 million ($27.6 million) in a Series A funding round.

The funds will support further development of its lead program, AMT-011, which is in a Phase II trial for genetic lipoprotein lipase (LPL) deficiency. The condition, a rare disorder also known as Type I hyperlipoproteinemia, arises from the absence of lipoprotein lipase, an enzyme responsible for breakdown of fat molecules. It is characterized by high levels of blood triglycerides, accelerated onset of atherosclerosis and recurrent bouts of pancreatitis, a life-threatening and highly damaging inflammation of the pancreas.

AMT, which was founded in 1998 by a group of scientists based at the Academic Medical Center (AMC) of the University of Amsterdam, aims to use the new injection of cash to expand clinical development of AMT-011 and to take several additional preclinical programs into the clinic.

The company, which is based at the AMC, is developing gene therapies for tackling the metabolic conditions acute intermittent porphyria and hyperoxaluria and for combating a rare form of progressive retinal blindness.

"All are not that far from the clinic," AMT co-founder and chief scientific officer Sander Van Deventer told Diagnostics & Imaging Week's sister publication BioWorld International.

AMT-011 has received orphan drug designation from the European Medicines Agency (London). The current trial, which involves just eight patients, is due to report in the first quarter of 2007. If successful, the company would rapidly expand clinical development with the aim of securing an early product launch.

"We believe we will be on the market with this product in 2008," Van Deventer said. The company aims to commercialize the product itself. The addressable market comprises around 4,000 patients worldwide. "We know who they are and where they are," he said. The therapy is delivered via several intramuscular injections that are administered in a single visit. "The process is too simple to be true," he said.

AMT also plans to develop a gene therapy for a less severe form of the condition, Type V hyperlipoproteinemia, which has an addressable market of around 100,000.

AMT met several key milestones, Van Deventer said, that enabled the company to raise cash at this point.

In preclinical studies, it obtained high levels of expression of the transferred gene over sustained periods, leading to an almost total correction of the defect. The company is using an adeno-associated vector, which, he said, the FDA has endorsed because it does not integrate into the host chromosomes. And the company has developed a method that ensures it can obtain sufficient quantities of vector for commercial use. "That has been a major, major hurdle for a long time," Van Deventer said.

ABN Amro Capital, Life Sciences, the venture capital arm of Amsterdam-based ABN Amro Bank NV, led the financing round. Other participants included London-based Advent Venture Partners; Gilde Investment Management BV, of Utrecht, the Netherlands; and Paris-based Crédit Agricole Private Equity. Sander Slootweg of ABN Amro Capital joined AMT's board.

MTM's C round raises EUR 22 million

MTM Laboratories (Heidelberg, Germany) also raised EUR 22 million ($27.6 million) in a Series C financing to fund further clinical development of its products for detecting cervical cancer. The company, which has raised a total of EUR 41 million since its formation in 1999, has developed diagnostic methods for detecting cancerous and precancerous cells based on a biomarker called p16INK4a, a cell-cycle protein that is significantly overexpressed in cancers induced by human papillomavirus (HPV).

In 2001 it assigned rights to two products — CINtec Histology and CINtec Cytology — to Dako (Glostrup, Denmark), but, having restructured that agreement earlier this year, it will take on full responsibility for the production and the sales and marketing of both starting in 2007.

"Now we can really leverage its value ourselves," MTM CEO Peter Pack said.

The company had never out-licensed a third product, Cervatec, a biochemical screening tool available in an ELISA assay format. MTM's strategy is based on reducing the strong element of doubt associated with routine smear tests.

"A lot of these Pap tests are ambiguous. You don't get a clear result," Pack said. In the U.S., he said, $6 billion is spent every year on administering smear tests and on follow-up. And $3.6 billion of that total, he added, is spent on follow-up, even though fewer than 10 percent of tests require further analysis.

CINtec Cytology is designed for the cytological analysis of cervical smears and liquid-based cytological samples. CINtec Histology is designed to aid in diagnosis of cervical cancer in tissue biopsies. In each case, immunohistochemical staining of p16INK4a provides additional information to aid diagnosis, which otherwise is dependent on expert visual analysis of cell and nuclear morphology.

The company aims to remove the necessity for expert analysis — a current bottleneck, Pack said - by establishing its Cervatec product as a clinically validated test. "We have to change clinical practice. We are fully aware there is a long way to go," Pack said. At present, it is positioned, along with CINtec Cytology, as an adjunct to current screening methods.

The company's products have a CE mark and a Class I research reagent designation in the U.S. It will use its latest cash injection to fund large-scale clinical trials in the U.S. and in Europe in order to obtain more clinical validation and to establish them as standalone in vitro diagnostics.

"We are talking about substantial clinical trials," Pack said. The studies will involve several thousand subjects.

Vital Diagnostics in Inter Medico pact

Clinical Data (Newton, Massachusetts) reported that its Vital Diagnostics division has signed a distribution agreement with Inter Medico (Markham, Ontario) to sell the Excyte line of Automated Erythrocyte Sedimentation Rate (ESR) Analyzers.

Gus Gardner, president, Clinical Data Sales and Services, said, "Inter Medico is the first Canadian distributor to sell the Excyte ESR product line. Our opportunity to sell automated ESR analyzers in Canada is considerable as the majority of ESR tests performed in Canada today are done individually by manual methods."

He said that the agreement allows it to place the Excyte Automated ESR Analyzers in small-to-large hospital laboratories and in all private laboratories.