LONDON – Autoimmune diseases specialist Apitope Ltd. has raised €12 million (US$13.4 million) in a series B funding, allowing it to take its two most advanced programs into clinical development.

"The first thing on the to-do list now that we have got the money is to file a clinical trial application for our lead program," Apitope CEO Keith Martin told BioWorld Today.

That program, ATX-F8-117, is intended as a treatment for hemophilia A patients who develop antibodies against replacement Factor VIII. The product contains two peptides derived from Factor VIII which are designed to recalibrate the immune system and restore tolerance to the blood-clotting agent.

About 30 percent of hemophilia A patients develop Factor VIII inhibitor antibodies and ATX-F8-117 has FDA and EMA orphan drug status.

ATX-F8-117 will be administered as an intradermal injection every two to four weeks, with patients being treated to bring back tolerance and then requiring ongoing treatment to maintain the effect.

It is planned to recruit 12 patients in the European phase I/II safety study. "We are really excited to have gotten the funding because we've got the orphan drug designation and are working with a product that we potentially could take to market ourselves," Martin said.

Following on the heels of ATX-F8-117 will be a second product for treating Graves' disease, an autoimmune disorder prompting excess activity of the thyroid gland.

Here Apitope has used its discovery platform to select three peptides that prevent the overproduction of antibodies against the thyroid stimulating hormone receptor (TSHR) that leads to Graves' disease. "All the other treatments around suppress activity of the thyroid, they don't treat the underlying causes in the way [ATX-GD-59] will," said Martin.

This product has been developed with €6 million of funding from the European Union's Framework 7 research program and is now ready for clinical development. "We've done all the preclinical work and the clinical trial application will be filed in three to four months," Martin said. The study will recruit around 40 patients.

The ability to attract investors to the series B rests on the phase I data from a third program, ATX-MS-1467, in which Apitope has demonstrated proof of concept for its platform technology for selecting peptides that can redirect the immune response.

Such antigen-specific immunotherapy is designed to correct the immunological imbalance without inducing nonspecific immune suppression that frequently causes unacceptable side effects. While this approach has been shown to be highly effective in animal models there has been slow to progress in the clinic.

In September 2013 Apitope announced the successful conclusion of its second phase I trial of ATX-MS-1467, reporting a significant decrease in the number of brain lesions in patients with relapsing multiple sclerosis treated by intradermal injection. Before that the product delivered positive data on brain lesions in a phase I trial in six patients with secondary progressive multiple sclerosis. There were no safety concerns in either study.

ATX-MS-1467 was licensed to Merck Serono in a $203 million deal in 2009. Following the completion of phase I, the Darmstadt, Germany-based company has taken over all responsibility for the funding and oversight of clinical development, completing recruitment of a phase IIa open-label efficacy trial in August. (See BioWorld Today, Jan. 14, 2009.)

The 18-patient trial, being staged in Latvia and Russia, will monitor lesions and look at other indicators of efficacy including biomarkers.

However, Martin said it will not be long enough to assess if there is any impact on disability or other symptoms. Assuming positive results, he then expects ATX-MS-1467 to progress directly to phase IIb.

Martin is pleased to have backing from all existing investors for the series B, which was led by a new investor, the Wales Life Sciences Fund. As a condition of getting money from the fund, which is backed by the Welsh government as part of its strategy for developing the country's life sciences sector, Apitope is required to relocate its headquarters to Wales.

However, Martin said the majority of the research will be carried out at Apitope's facility in Hasselt, Belgium, and that the company will maintain a laboratory in Bristol, UK, where its roots lie.