Biopharmaceutical company Ablynx is developing Nanobodies® to treat a range of serious human diseases. Nanobodies are a novel class of antibody-derived therapeutic proteins. Because of their small size, unique structure and extreme stability, Nanobodies combine the advantages of conventional antibody therapeutics with the key features of small-molecule drugs. Zillion spoke with CEO and Chairman Dr Edwin Moses. Ablynx was established as a spin-off from the Free University of Brussels in 2001. “Biotechnology companies always have to spend more money than they make for many years, so our number one priority was to raise money,” says Dr. Moses. “After two initial financing rounds, we raised another EUR 40 million in 2006, led by new investor KBC, Belgium’s leading bank for private and public equities.” The round was one of the largest finance rounds in Europe during 2006. A year later the company went public on Euronext Brussels, raising an additional EUR 85,2 million. Ablynx is developing Nanobodies as drug candidates. “Our bodies naturally generate antibodies to protect us from viruses and other invaders. Over the last 20 years, the pharmaceutical industry has begun to use these antibodies as drug products. 20 products have already been approved. These include multi million dollar drugs,” Dr. Moses explains. In the early 1990s the existence of Nanobodies was discovered. “Nanobodies have all the interesting properties of antibodies: they are target specific and they tend to have few side effects. Besides these qualities, Nanobodies can penetrate tissue very effectively because they are smaller than antibodies – only one tenth the size of a classic antibody. They are also very stable, which means you can boil them, cool them down and they will reform. That stability means that there is the potential to deliver them through alternative routes rather than just injection, such as orally or by inhalation. That can’t generally be done with classic antibodies.” Nanobodies can also be produced very quickly, because Ablynx’s method avoids the need for construction and screening of large libraries, and for lengthy and unpredictable affinity maturation. “We inject a protein that we are interested in generating a nanobody against into a llama. Six to eight weeks later the immune system of the llama will have produced Nanobodies to that protein. We take a small blood sample, and isolate and sequence those Nanobodies, which we can then engineer ourselves.” Another advantage is that Nanobodies can be easily manipulated. “You can for instance develop a nanobody for one target and one for another target and link them together, so you have a combination drug in one molecule.” While many biotech companies concentrate on one or two disease areas, Ablynx is developing a portfolio of Nanobody-based therapeutic programmes in a number of major disease areas, including inflammation, thrombosis, oncology and Alzheimer’s disease. Mr. Moses: “We look where Nanobodies can be most effective and we’ll develop a product for that, irrespective of the disease.” Ablynx’s lead development programme ALX-0081 is a therapeutic nanobody targeting von Willebrand Factor (vWF), which will reduce the risk of thrombosis in patients with acute coronary syndrome (ACS) and thrombotic thrombocytopenic purpura (TTP). Through its novel, highly selective mode of action, ALX-0081 effectively prevents arterial thrombosis, without interfering with the desired haemostasis (wound heeling) in the patient which results in less bleeding complications. Pre-clinical in vivo and clinical studies confirm that ALX-0081 has the unique potential to set a new standard in anti-thrombotic therapy In addition to developing a proprietary pipeline, Ablynx has entered into a number of partnerships. Partners include Boehringer Ingelheim, Novartis, P&G Pharmaceuticals and Wyeth. The partnership with Wyeth involves a collaboration to discover and develop novel Nanobody-based therapeutics against the tumour necrosis factor alpha (TNF-alpha) protein and its receptors that target diseases in multiple therapeutic areas. Ablynx has granted Wyeth exclusive worldwide rights to Nanobodies targeting the clinically validated target TNF-alpha. Ablynx and Wyeth will collaborate to advance these novel biologics through preclinical development. Ablynx will receive an initial payment, research support and milestone payments. Potential payments to Ablynx could total up to USD 212.5 million for the successful development and commercialisation of multiple products. In addition Ablynx will receive royalties on product sales. based on its immediate onset of action, its ultra-high potency and significantly improved safety compared to the currently marketed therapies in the form of dramatically reduced bleeding complications. Following the positive Phase I results announced in December 2007, Ablynx has initiated a double-blind, randomised, placebo-controlled multiple dose Phase Ib study in May 2008. The study is being conducted in patients with stable angina undergoing percutaneous coronary intervention (PCI). The objectives of the Phase Ib study are to determine safety and tolerance when adding ALX-0081 to a standard anti-thrombotic regimen in patients undergoing PCI and to document biological and clinical response to the therapy. Up to 64 patients with stable angina will be recruited for this study. Ablynx aims to start the process of Phase II development of ALX-0081 by the end of 2008. In addition to developing a proprietary pipeline, Ablynx has entered into a number of partnerships. Partners include Boehringer Ingelheim, Novartis, P&G Pharmaceuticals and Wyeth. The partnership with Wyeth involves a collaboration to discover and develop novel Nanobody-based therapeutics against the tumour necrosis factor alpha (TNF-alpha) protein and its receptors that target diseases in multiple therapeutic areas. Ablynx has granted Wyeth exclusive worldwide rights to Nanobodies targeting the clinically validated target TNF-alpha. Ablynx and Wyeth will collaborate to advance these novel biologics through preclinical development. Ablynx will receive an initial payment, research support and milestone payments. Potential payments to Ablynx could total up to USD 212.5 million for the successful development and commercialisation of multiple products. In addition Ablynx will receive royalties on product sales. top - home