Advicenne Announces Positive Pivotal Phase III Clinical Data for ADV7103
11 Sep 2017» News
ADV7103 could potentially be the first approved treatment for dRTA
Nîmes, France, 11th September, 2017 – Advicenne, a late-stage biopharmaceutical company focused on the development of paediatric-friendly therapeutics for the treatment of orphan renal and neurological diseases, announces positive top-line data from a pivotal phase III study assessing ADV7103 in adults and children suffering from distal Renal Tubular Acidosis (dRTA). dTRA is a disease characterised by an unbalanced pH in the body associated with additional disorders such as biochemical impairments that may result in failure to thrive, rickets/osteomalacia, lithiasis and nephrocalcinosis that can lead to renal failure.
The objective of the study was to evaluate the efficacy, safety and acceptability of ADV7103, an innovative oral product, formulated in paediatric-friendly coated granules, that combines two active pharmaceutical ingredients. ADV7103 is given twice a day in contrast to the current standard of care (SoC), which are usually various unapproved products administered every four to six hours to attempt to re-balance the body’s pH and to normalise blood potassium level (kalaemia).
The Phase III study of ADV7103 was shown to restore the main biological defects observed with the disease meeting positively primary and secondary endpoints.
Normal blood bicarbonate levels were attained in most patients treated with doses of ADV7103 ranging from 0.75 to 8.45 mEq/kg/day. Mean doses of 1.7, 2.3, 3.8 and 6.1 mEq/kg/day ADV7103 were given, respectively, in adults, adolescents, children, and infants. Non-inferiority of ADV7103 vs. SoC or baseline literature data was consistently demonstrated (per protocol, intention-to-treat, as well as sensitivity analyses). Kalaemia was normalised with ADV7103 with only two doses per day.
These analyses were able to show that ADV7103 is superior to the SoC (p<0.0047).
Further important advantages of ADV7103 which were demonstrated in the study were its good palatability, ease of administration and gastrointestinal tolerability.
The data from this pivotal phase III study was presented at the 50th European Society of Paediatric Nephrology (ESPN) 2017 Annual Meeting which was held from the 7th to 9th of September in Glasgow, UK, during an oral communication on Saturday, 9th September.
Dr Luc-André Granier, CEO and cofounder of Advicenne, commented, “We are delighted to announce the positive results from this pivotal phase III study which clearly show that ADV7103 has the potential to provide a meaningful and durable benefit to patients with dRTA. The success we have achieved with ADV7103 reflects our ability to leverage our scientific knowledge, network of key opinion leaders, formulation expertise and unwavering commitment to patients to deliver paediatric friendly therapeutics for the treatment of orphan renal diseases for which there are currently no approved treatment options.”
Advicenne also sponsored a scientific Symposium event dedicated to dRTA on 8th September at ESPN.