Aquilon Pharmaceuticals has passed an important milestone in the development of its first treatment AQ001S.

Aquilon Pharmaceuticals has passed an important milestone in the development of its first treatment AQ001S, based on a technological complex combining Budesonide and a Cyclodextrin (HP-Betadex).

Following the successful completion of its toxicity trials, Aquilon Pharmaceuticals initiated two clinical trials in humans.

The first pharmacokinetic trial, BORA (NCT05148312) has been conducted in 24 healthy volunteers to compare the AQ001S solution with a Budesonide reference product (Pulmicort suspension). Results show that the pharmacokinetics of our AQ001S product are linear. More, no side effects have been reported, demonstrating the safety of AQ001S.

AQ has also successfully finalized its Phase IIa pivotal BOREAS trial (NCT04933383). The BOREAS trial, conducted to establish the proof-of-concept (POC) of our AQ001S solution, assessed and compared AQ001S efficacy, safety, and pharmacokinetics to those of a comparator.

Conducted as an active-controlled randomized clinical trial encompassing mild asthmatic patients from Belgium, the BOREAS trial yielded the following notable outcomes:

  1. Patients administered with our AQ001S solution were provided a better bronchoprotection than the patients administered with the comparator showing that even at very low dose, our product helps patients to relieve their asthma.
  2. No side effects have been reported, demonstrating that AQ001S is safe and well tolerated.

Those excellent results strengthen the overall belief in this very promising new formulation technology.

We were able to demonstrate that the technology we have developed meets its expectations for the treatment of patients with mild asthma. The study report highlights that both primary endpoints on safety and efficacy are met, which is not common at this stage”, says Paul Maes, CEO of the company.

Prof Dr Didier Cataldo explains: “This all means that the product AQ is developing proved its efficiency and value in preventing asthma events in patients. Indeed, despite the availability of effective inhaled therapies, many patients with asthma have poor asthma control. Uncontrolled asthma presents a significant burden on the patient and society, and, for many, remains largely preventable”.

With these results, AQ is continuing its work to establish a solid basis for the use of cyclodextrin in the field of inhaled medication.

As an expert in this field, Dr Hassan Benameur is delighted with these findings and sees a great  future for the company: “These data, together with the excellent results from the phase I BORA trial obtained in healthy volunteers, open a large spectrum of opportunities for this formulation technology, based on HP-Betadex, which could very specifically bring solutions for products where inhaled administration proved to be more difficult while answering patients unmet needs”.

Aquilon Pharmaceuticals tend to believe that these positive results will pave the way for a simplified registration procedure with the FDA (USA) and allow a shortened time to marketing authorization. The US market is one of the focus markets for Aquilon Pharmaceuticals, who recently brought in some industry experts who will bring all the required expertise and experience necessary to successfully complete the registration file with the FDA in the USA.