BioInItaly Newsletter

La vetrina del biotech italiano all’estero

BioInItaly nr. 4 - 2013

05 apr, 2013

Presented the "Italian Biotechnology Report 2013" by Assobiotec and Ernst & Young


On May 7th, in Milan, it was presented the "Italian Biotechnology Report 2013" by Assobiotec and Ernst & Young in collaboration with Farmindustria and the Italian Trade Promotion Agency, which analyzes industry data in its various areas of application and its performance.
The biotech sector in Italy is a growing and promising reality: the biotech industry is composed of 407 companies, mostly small, employing less than 50 employees. With 235 companies operating in the health-care field, the “Red Biotech” is the prevalent sector.
Total turnover equals € 7,152 million, with a 6% increase compared to the previous year, whilst R&D investments have grown to € 1,832 million, with a further 2.9% increase. It demonstrates vigorous growth and an excellent innovation capacity. In view of the tender age of this industry such characteristics are, in some respects, very surprising. Thanks to them, Italy has managed to meet a challenge that would have been unthinkable a few years ago: to finally appear on the radar screens of the major biopharmaceutical and biotechnological companies and venture capital operators. There are tangible facts behind this phenomenon: new enterprises emerging at a progressively faster rate, companies reaching maturity and consolidating themselves, but first and foremost, major R&D investments being made, which a very significant growth rate in the last few years.
"Biotechnology is certainly an area of fundamental importance to ensure economic growth, high skilled employment, quality of life and well-being for Italy. Being an anti-cyclical sector that involves many areas of application, it also provides a distinctive business model, competitive and sustainable development "said Alessandro Sidoli, President of Assobiotec, the Italian Association for the development of biotechnology.
"In Italy, however, after several years of growth at a fast pace, the biotech sector shows the first signs of plateau, due to the lack of timely measures to support research and development, and protect innovative products. We refer for example to the absence of the tax credit on expenditure on R&D, or the Governamental growth decree 2.0 which effectively excludes almost all the biotech SMEs focused on drug discovery and development. We need now strong central policies to support the Italian growth, which, combined with the regional policies, will be able to strengthen a sector that, globally, as an industrial meta-sector, worths 0.4% - 1.1% of GDP”.

EryDel announces US IND approval for EryDex


EryDel SpA (www.erydel.com), a Drug Delivery Company specialized in the development of drugs and diagnostics delivered through red blood cells (RBCs), announced that it has received approval to start its first study in the US. The Investigational New Drug (IND) Application for EryDex (dexamethasone delivered through autologous erythrocytes) was filed on March 24, 2013.
EryDex is an innovative treatment for Ataxia Telangiectasia (AT), a rare autosomal recessive neurological disorder with high mortality. No treatment has been approved for the treatment of the neurological symptoms of AT.
The IND was filed after a pre-IND meeting with FDA representatives from the Division of Neurological Products and the Center for Biologics, who provided valuable input concerning
requirements for the program. FDA previously granted EryDex Orphan Status for the treatment of AT.
Erydel reported earlier (2012) that treatment with Erydex met the primary endpoint of improvement in neurological symptoms in a 6-month prospective study in patients with AT.

Pre-clinical results on naproxcinod in models of muscular dystrophy presented at MDA Scientific Conference


Nicox S.A. (NYSE Euronext Paris: COX) recently announced promising pre-clinical results on naproxcinod, a CINOD (Cyclooxygenase-Inhibiting Nitric Oxide-Donating) anti-inflammatory candidate, in models of muscular dystrophies. The data were presented by Nicox and the Center for Genetic Medicine Research, a center within the Children's Research Institute, Children's National Medical Center, in a poster session on April 22nd at the Muscular Dystrophy Association (MDA) Scientific Conference in Washington, DC.
The research study data were presented in a poster entitled "Long-term treatment with naproxcinod significantly improves skeletal and cardiac disease phenotype in mdx mouse model of dystrophy" (poster #175). In this study, the effects of naproxcinod were investigated on skeletal and cardiac muscle function in mdx mice. Three doses of naproxcinod (10, 21 and 41 mg/kg) were given orally to 4-week-old mdx mice (a reference model for Duchenne Muscular Dystrophy, DMD) for 9 months and compared to 0.9 mg/kg of prednisolone. The results of the study suggest that naproxcinod may have potential as a safe therapeutic option for the treatment of muscular dystrophies. Naproxcinod treatment at 10 and 21mg/kg resulted in significant improvements in hind limb grip strength as well as an approximately 25-30% decrease in inflammation in fore and hind limbs measured by in vivo optical imaging. Furthermore, there were significant improvements in heart function as evidenced by improved fraction shortening and ejection fraction, measured using echocardiography, along with improvements in systolic blood pressure. In addition, the long term detrimental effects of prednisolone typically seen in skeletal and heart function were not observed at the effective doses of naproxcinod.
Naproxcinod is a CINOD (Cyclooxygenase-Inhibiting Nitric Oxide-Donating) anti-inflammatory candidate originally developed by Nicox for the relief of the signs and symptoms of osteoarthritis. The results presented on April 22nd 2013 were from an exploratory research study sponsored by Nicox and conducted at the Center for Genetic Medicine Research. The objective was to investigate the potential for the use of naproxcinod in Duchenne Muscular Dystrophy (DMD) as there is evidence that nitric oxide can play a critical role in the functioning of skeletal muscle.
The Nicox's current strategy is to evaluate the opportunity to out-licence naproxcinod for the treatment of the signs and symptoms of osteoarthritis of the knee. Separately, the Company is also seeking specialized partners in the field of rare diseases who would be able to undertake and fund the potential development of naproxcinod in muscular dystrophy. This approach is aimed at maximising the opportunities to progress the development of naproxcinod in one of these indications.