Amryt, a revenue-generating orphan drug company focused on acquiring, developing and commercialising products that help improve the lives of patients where there is a high unmet medical need, today announces its preliminary unaudited results for the year ended 31 December 2018. It also provides a trading update for Q1 2019. The 2018 Annual Report will be published in May 2019.
Revenues for 2018 amounted to €14.5m which represented a 13.3% increase on 2017 and this positive revenue momentum has continued into Q1 2019 with Lojuxta revenues growing by 28.1% compared to Q1 2018. This growth has been driven by the recent reimbursement decisions in the UK and France, with initial orders received from UK patients in Q4 2018 and from French patients in Q1 2019. Revenue growth has been further strengthened by a 118% increase in quarterly revenues from Saudi Arabia from Q4 2018 to Q1 2019.
The Company’s lead development asset, AP101, continues to progress, having recently successfully completed an unblinded interim efficacy analysis by an Independent Data Monitoring Committee (“IDMC”) of the Phase 3 EASE study in Epidermolysis Bullosa (“EB”). The Committee recommended that the trial continue with a modest increase in the number of patients in the study, to 230 evaluable patients, to maintain 80% statistical power. The IDMC has also recently expanded the eligible patient population to include infants and children with EB between the ages of 21 days to 4 years of age. The Company is also pleased to announce the commencement of its AP101 early access program for patients with EB in Colombia who are not eligible for the EASE study with the first shipments of product in late Q4 2018.
2018 Financial Highlights:
- Revenue growth of 13.3% to €14.5m (2017: €12.8m)
- Revenues from Lojuxta (lomitapide), which treats HoFH, a rare, genetic, life-threatening disorder that causes abnormally high levels of “bad” cholesterol, increased to €13.6m, which represents a growth rate of 14.2% year-on-year
- Gross profit margin increased to 63% (2017: 58%)
- Cash balance at 31 December 2018 of €9.8m (2017: €20.5m)
- Post period-end : additional €5m drawn down from the EIB debt facility in Q1 2019
2018 Operational Highlights:
Lead Commercial Asset – Lojuxta
- Eight new distribution agreements signed in 2018, now covering 23 countries in total
- Reimbursement approval received in the UK and France resulting in first orders for the UK in late 2018
- Initial orders received for patients in Saudi Arabia in Q4
- Continued expansion in the licenced territories for Lojuxta, including Russia, the Commonwealth of Independent States (“CIS”), and the non-EU Balkan states
Lead Development Asset – AP101
- Significant continued progress made in the development of AP101, a potential treatment for EB, a rare life limiting genetic skin condition
- An Investigational New Drug (“IND”) approval, recently obtained from the FDA, permits the Group to open clinical trial sites in the US, which is expected to help enrolment into the EASE Phase3 study in EB
- A Paediatric Rare Disease designation was granted by the U.S. Food and Drug Administration (“FDA”) which means if a New Drug Application (“NDA”) for AP101 is approved, Amryt will be eligible to receive a priority review voucher that can be used, sold or transferred. Publicly disclosed sale prices for such vouchers have ranged from US$67.5m to US$350m
- Early access programme in EB initiated in Colombia with first AP101 shipments in response to unsolicited requests for named patient access in Q4 2018
Gene Therapy Platform -AP103
- Exclusive in-licence agreement signed in March 2018 with University College Dublin (‘UCD’) for a novel non-viral gene therapy platform technology, which offers a potential treatment for patients with both EB and beyond
- Preliminary data suggests that the treatment could be disease-modifying for patients with Recessive Dystrophic Epidermolysis Bullosa (“RDEB”), a major subset of EB
- Significant grant funding totalling €8.4m awarded by the Irish Government to develop the AP103 gene therapy platform over the next three years
Post Period-End – Q1 2019 Highlights
Lead Commercial Asset – Lojuxta ▼
- Lojuxta unaudited revenues for Q1 2019 of €3.9m, which represents an increase of 28.1% on the same period in 2018, continuing the momentum generated in 2018
- Significant expansion in patient numbers in the UK in Q1 2019 following the launch in this new market in Q4 2018
- First patient order received from France in Q1 2019
- Sales to Saudi Arabia in Q1 2019 increased by 118% compared to Q4 2018
Lead Development Asset – AP101
- Following an assessment of the results of an unblinded interim efficacy analysis of its pivotal Phase 3 EASE trial for AP101 as a potential treatment for EB, the IDMC recommended that the trial should continue with an increase of 48 patients in the study to a total of 230 evaluable patients, in order to maintain 80% statistical power
- Following an assessment in February by the EASE trial’s IDMC of pharmacokinetic (“PK”) data received from patients already enrolled in the trial (aged four years and older), Amryt can now enrol infants and children with EB between the ages of 21 days to 4 years of age into the trial
- The EASE study is progressing well and it is expected that the final patient will be enrolled in H2 2019
Gene Therapy Platform – AP103
- Two pre-clinical studies showed that topical application of AP103 restored production of collagen VII in pre-clinical models of EB to levels exceeding those produced by healthy human keratinocytes and to levels similar to those observed following delivery with a viral vector
- In addition, AP103 exhibited no evidence of cellular toxicity after repeated administration
Dr Joe Wiley, CEO of Amryt Pharma, commented: “2018 was another strong year for Amryt as we continued to expand our business and make progress towards our goal of becoming a global leader in rare and orphan diseases. We continued to grow Lojuxta sales and we believe that we now have in place the commercial platform and critical infrastructure to make this a significant business and cash generator for Amryt in the future. During the year, we expanded our Lojuxta licenced territories to 23 countries, and importantly we were granted approval for funding both as an NHS treatment in England, and for patients in France. We also received our first prescriptions for patients in Saudi Arabia in Q4. These very significant achievements are already having a significant positive impact on our financial & operational performance in 2019. Lojuxta revenues for Q1 2019 are already 28.1% higher than the same period in 2018, and we are now able to reach more people living with the ultra-rare and life-threatening condition HoFH than ever before. Amryt will continue to actively review further growth opportunities that could expand the Group’s commercial product portfolio.
“Our lead development asset, AP101, achieved a significant milestone with the completion of an unblinded interim efficacy analysis of our EASE Phase 3 trial. This resulted in the IDMC recommendation that we continue with the trial with only a modest increase in patient numbers. We are now expanding EASE recruitment with the opening of a number of new trial sites in the US. The IDMC has also allowed us to open our study to children with EB between the ages of 21 days to four years of age.
“In addition, we continued our strategy to acquire new products with the in-licencing of AP103 in 2018. We are excited by the potential for this novel gene therapy in EB and beyond. We continue to evaluate new opportunities and expect to further deliver on our strategy to acquire, develop and commercialise orphan drugs as we move through 2019. We expect that this continued progress will bring us closer to realising our vision of becoming a global leader in rare and orphan diseases.”