- Target enrolment of 35 infants, aged 3-24 months, completed
- On track to release top line results in the first half of 2016
- Study extended for infants aged 1-5 months
GHENT, Belgium, Dec. 17, 2015 (GLOBE NEWSWIRE) -- Ablynx [Euronext Brussels: ABLX; OTC: ABYLY] today announced that it has completed target enrolment of the first-in-infant Phase I/IIa safety study with its anti-RSV Nanobody, ALX-0171, and is on track to publish top line results in the first half of 2016. ALX-0171 is a wholly-owned Nanobody drug candidate, administered through inhalation, for the treatment of respiratory syncytial virus (RSV) infection in infants.
The Company also announced that following a positive recommendation from an independent Data Monitoring Committee (DMC) based on data available for the first 15 infants from the placebo-controlled part of this Phase I/IIa safety study, the age requirement for infants in the trial has been reduced from 3 months to 1 month. The Phase I/IIa study will now continue with an expansion cohort enrolling younger infants aged 1-5 months during the remaining RSV season in Northern Europe. This will enable the collection of additional data on ALX-0171 in a younger patient population. Results from this extension will be available in the second half of 2016.
Edwin Moses, Chief Executive Officer of Ablynx, said: "Ablynx is pioneering the development of a potentially first-in-class treatment for RSV infection, an area of major unmet need, especially in infants. We are utilising a key characteristic of our Nanobodies to deliver them via nebulisation directly to the site of infection, a capability which may be beneficial in other pulmonary diseases. ALX-0171 is one of our wholly-owned, proprietary programmes in clinical development and we believe that completing target enrolment of this first-in-infant study is a major accomplishment for our Company. We are looking forward to releasing top line results from this safety study in the first half of 2016 with further data from the expansion cohort anticipated in the second half of 2016. In parallel, we will continue with the preparations to initiate the next clinical study in infants, which is expected to start recruiting by the end of 2016."
About the Phase I/IIb safety inhalation study
In December 2014, Ablynx opened recruitment for the first-in-infant Phase I/IIa safety study with inhaled ALX-0171 in otherwise healthy infants (aged 3-24 months) diagnosed with RSV and hospitalised for lower respiratory tract infection. The study has recruited 35 hospitalised infants in multiple clinical centres in Europe and the Asia-Pacific region and consisted of two parts: 1) a lead-in phase with five infants who received inhaled ALX-0171, once daily, for three consecutive days; and 2) a placebo-controlled phase in 30 infants who received either placebo or inhaled ALX-0171, once daily, for three consecutive days. The primary endpoint of the study is safety and tolerability of ALX-0171. Secondary endpoints include clinical effect (including effect on feeding, respiratory rate, wheezing, coughing, general appearance and clinical scores), immunogenicity, pharmacokinetics (PK) and pharmacodynamics (PD) of inhaled ALX-0171.
About RSV and ALX-0171
Respiratory syncytial virus (RSV) is the most common cause of lower respiratory tract infections and the leading viral cause of severe lower respiratory tract disease in infants and young children worldwide. It is the primary cause of infant hospitalisation and virus associated deaths in infants, with estimated global annual infection and hospitalisation rates of 34 million and 3-4 million respectively. It is associated with an estimated 3,000-8,500 deaths in infants <2 years globally per year. In addition, RSV infections have been linked to an increased risk of asthma development later in life. Current treatment of RSV infection is primarily focussed on symptomatic relief, hence the need for an effective and specific anti-RSV therapeutic.
ALX-0171 has been developed to address this unmet medical need and has first-in-class potential for the treatment of RSV infection in infants. It is a wholly-owned trivalent Nanobody that inhibits RSV replication and which neutralises RSV activity by blocking virus uptake into cells. The physical robustness of the Nanobody allows administration via inhalation directly to the site of infection, i.e. the respiratory tract including the lungs. ALX-0171 has shown a potent anti-viral effect against a broad range of RSV strains and it demonstrated to have a strong therapeutic effect following administration via nebulisation in a neonatal animal model for infant RSV infection. Repeated daily inhalation of ALX-0171 was proven to be well-tolerated in multiple Phase I clinical studies, including a study in subjects with hyper-reactive airways. At the end of September 2014, the European Medicines Agency (EMA) approved the Paediatric Investigation Plan (PIP) for ALX-0171.
Ablynx is a biopharmaceutical company engaged in the development of Nanobodies®, proprietary therapeutic proteins based on single-domain antibody fragments, which combine the advantages of conventional antibody drugs with some of the features of small-molecule drugs. Ablynx is dedicated to creating new medicines which will make a real difference to society. Today, the Company has approximately 40 proprietary and partnered programmes in development in various therapeutic areas including inflammation, haematology, immuno-oncology, oncology and respiratory disease. The Company has collaborations with multiple pharmaceutical companies including AbbVie, Boehringer Ingelheim, Eddingpharm, Genzyme, Merck & Co., Inc., Merck KGaA, Novartis, Novo Nordisk and Taisho Pharmaceuticals. The Company is headquartered in Ghent, Belgium. More information can be found on www.ablynx.com.
For more information, please contact
Dr Edwin Moses
t: +32 (0)9 262 00 07
m: +32 (0)473 39 50 68
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t: +32 (0)9 262 00 82
m: +32 (0)479 49 06 03
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Ablynx media relations Instinctif Partners:
Sue Charles, Daniel Gooch
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Belgium/Dutch and French language
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 Byington et al, Pediatric 2014
 Sigurs et al, Thorax 2010; Backman et al, Acta Pediatr 2014
Pdf version of the press release http://hugin.info/137912/R/1974218/722293.pdf