What the company does
Last year, Amryt acquired US group Aegerion Pharmaceuticals out of Chapter 11 bankruptcy protection in a transaction backed by convertible bondholders, creditors and Nasdaq-listed Novelion Therapeutics, Aegerion’s parent company.
The complex transaction gave Amryt full control of the Lojuxta cholesterol treatment.
One of its lead candidates is the AP101 cream, branded FILSUVEZ, developed for epidermolysis bullosa (EB), a chronic and distressing genetic skin disorder that causes the skin layers and internal body linings to separate.
The company is also focusing on a pre-clinical gene-therapy platform technology, AP103, which offers a potential treatment for patients with Recessive Dystrophic Epidermolysis Bullosa, a subset of EB. This gene-therapy platform also has potential applicability across a range of other genetic disorders.
How it is doing
In September, a European Medicines Agency (EMA) committee has delivered what’s called a “positive opinion” on AP103, which is normally followed 30 days later by full ratification. It means it may soon receive orphan designation, designed to aid the development and sign-off of life-threatening or chronically debilitating conditions affecting no more than five in 10,000 people. Incentives include ten years’ EU exclusivity following approval.
Clinical development of AP103 is expected to start in the first half of 2022.
Meanwhile, the phase III study on FILSUVEZ achieved its main goal, known in the scientific parlance as the primary endpoint, of accelerating the wound healing process. The next step is a rolling submission programme to the regulatory authorities, with the company applying for speedy approval in both the US and EU.
The company previously received fast track designation and rare paediatric disease designation from the US Food & Drug Administration (FDA), and it expects to be eligible to apply for a rare paediatric disease priority review voucher.
Submissions to the US and EU regulators are expected to be made late in the first quarter of next year.
Amryt estimates there are as many as 30,000 youngsters and adults affected by it in the US and there are 500,000 sufferers worldwide. The potential market for FILSUVEZ, therefore, is in excess of US$1bn.
Full-year revenues are expected to come in at between US$170-175mln after good demand for key drugs metreleptin and lomitapid.
What the boss says: Joe Wiley, chief executive
“Receiving this positive opinion from the EMA for our gene-therapy candidate, AP103, is a significant development for patients suffering from epidermolysis bullosa and provides additional momentum to our development pipeline.”
“These milestones relating to our efforts in epidermolysis bullosa are significant for all Amryt stakeholders as we build a global epidermolysis bullosa franchise and become a leader in rare and orphan diseases.”
What the broker says
House broker Shore Capital said if regulatory submissions are approved for FILSUVEZ, Amryt is also eligible for a priority review voucher (PRV), which could be worth up to US$100mn and is not yet reflected in its forecasts and hence represents potential upside.
“We see this as a transformational milestone for Amryt and its management team, with the positive headline data a hugely de-risking event.”
The shares trade at a significant discount to fair value, the ShoreCap analysts said, representing less than 2x sales versus global peers on at least five times sales, and with “significant upside from EB and the dual-listing on Nasdaq as key drivers for a re-rating”.
Published at Fri, 16 Oct 2020 13:17:00 +0000-Amryt Pharma cheers orphan designation for pre-clinical skin treatment