JHL Biotech Submits Application for Phase 1 Trial of Proposed Dornase Alfa Biosimilar

JHL Biotech, a biopharmaceutical startup based in Taiwan, announced that it has submitted a phase 1 clinical trial application to the Dutch Healthcare Authority (DHA) for its proposed dornase alfa (Pulmozyme) biosimilar, JHL1922, to improve pulmonary function in cystic fibrosis patients. The clinical trial under the DHA is scheduled to be conducted in the Netherlands beginning March 2018.

Cystic fibrosis affects over 100,000 people worldwide, and dornase alfa is an important part of the treatment regimen, showing significant results in improving pulmonary function. Unlike many biologic therapies, which are infused, dornase alfa is administered using a rapid nebulizer system or a jet nebulizer connected to an air compressor.

According to Diana Bilton, MBChB, consultant chest physician at the Royal Brompton Hospital, London, United Kingdom, quoted in The Pharmaceutical Journal, “[Dornase alfa] is now so much a part of the furniture of basic cystic fibrosis treatment that it’s easy to forget how hard people used to have to work to clear their pulmonary secretions...[the drug] is now a fundamental part of aiding airway clearance and pediatricians are likely to think about it in children with cystic fibrosis as young as 5 or 6 years old who have nasty coughs and mucus plugging.” According to the article, the outlook for children and young adults with cystic fibrosis has improved dramatically in recent decades, partially due to the introduction of dornase alfa.

The proposed dornase alfa biosimilar is, like the reference product, a recombinant human deoxyribonuclease I (rhDNase I) enzyme that selectively treats cystic fibrosis based on genetic markers located in DNA. The reference drug, originally approved by the FDA in 1993, is sold by Genentech. Estimates show that the cost of dornase alfa treatment is $12,000 to $40,00 per year, with only about 30,000 patients a year receiving this rare treatment.

“JHL1922 would increase affordable access to an important therapeutic for cystic fibrosis patients, and we look forward to conducting the phase 1 trial in Europe,” said Racho Jordanov, CEO, JHL Biotech, in a statement.