AP101 trial discussions completed with FDA & EMA

Released : 06 Mar 2017 07:00

RNS Number : 5424Y
Amryt Pharma PLC
06 March 2017
 

 

AIM: AMYT, ESM: AYP

 

Amryt Pharma plc

("Amryt" or the "Company")

 

Completion of Pivotal Clinical Trial Discussions with FDA and EMA

 

Phase 3 trial now ready to commence for

AP101 as a treatment in Epidermolysis Bullosa

 

Amryt, the pharmaceutical company focused on best-in-class treatments for rare and orphan diseases, is pleased to announce the completion of discussions with the Food and Drug Administration ("FDA") and European Medicines Agency ("EMA") regarding the design of its pivotal phase 3 clinical trial for AP101 as a potential treatment for Epidermolysis Bullosa ("EB"). EB is a rare genetic skin disorder that leads to exceptionally fragile skin and children with the disorder are often referred to as "Butterfly Children". The global market for a treatment in EB is estimated to be in excess of EUR 1.3 billion.

 

With regulatory authority discussions now completed with FDA and EMA and the design of the clinical trial established, Amryt is on track to commence the phase 3 trial at the end of March. Amryt has also agreed to conduct some further non-clinical studies in parallel with this phase 3 study. INC Research has been appointed as the contract research organisation for the phase 3 study, and approximately 30 clinical trial sites in 15 countries have already been pre-qualified.

 

Adult and paediatric patients with EB will be enrolled into a randomised double blind placebo controlled trial. A total of 164 evaluable patients will be treated for a 90 day blinded period. The proportion of patients with completely healed target wounds within 45 days will be evaluated as the primary endpoint. Secondary endpoints include the time to achieve wound healing and changes in pain and pruritus (itch).

 

An important component of the phase 3 study is an independent data monitoring committee that will conduct an un-blinded interim efficacy analysis after 50% enrolment. The potential outcomes of this interim analysis include continuation of the study unchanged, discontinuation of the study for futility, or an increase in the number of patients in the study to preserve adequate statistical power.  

 

Joe Wiley, Chief Executive Officer of Amryt, commented:

 

"We are delighted to have reached this critical milestone in the development of Amryt. Reaching agreement with the regulatory authorities for our phase 3 study in EB was a precursor to commencing our pivotal study, which we now expect will enrol its first patients in the coming weeks. Significantly, we now have agreement from the regulatory agencies in both the US and in Europe for a single phase 3 study, which we hope in due course will lead to approval in EB. There remains substantial unmet need for drugs which can address the symptoms and we are delighted to be progressing our potential treatment to this pivotal phase."

 

Mark Sumeray, Chief Medical Officer of Amryt, commented:

 

 "We are very pleased to have reached an understanding with the US and EU regulatory authorities on the design of our pivotal phase 3 trial for AP101 as a potential treatment for EB. We believe that the adaptive approach to study sample size will help to ensure that we achieve a reliable assessment of the potential benefit of our new skin healing treatment in this orphan disease."

 

Enquiries:

Amryt Pharma plc

c/o KTZ Communications

+44 (0)20 3178 6378

Joe Wiley, CEO

Rory Nealon, CFO/COO

 

 

 

Shore Capital

+44 (0) 20 7408 4090

Nomad and Joint Broker

 

Bidhi Bhoma, Edward Mansfield

 

 

 

Davy

+353 (1) 679 6363

ESM Adviser and Joint Broker

 

John Frain, Anthony Farrell

 

 

 

Stifel

+44 (0) 20 7710 7600

Joint Broker

 

Jonathan Senior, Ben Maddison

 

KTZ Communications

+44 (0) 20 3178 6378

Katie Tzouliadis, Emma Pearson

 

 

About Amryt Pharma plc - see www.amrytpharma.com

Amryt Pharma is a specialty pharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare or orphan diseases. The Company is building a diversified portfolio of commercially attractive, best-in-class, proprietary new drugs to help address some of these rare and debilitating illnesses for which there are currently no available treatments.

It recently acquired an exclusive licence to sell LOJUXTA (lomitapide), across the EU and other territories including the Middle East, North Africa, Turkey and Israel. LOJUXTA is used to treat a rare life-threatening disease called Homozygous Familial Hypercholesterolemia. 

Amryt's product, AP101, received marketing approval for the treatment of partial-thickness wounds from the European Commission in January 2016.  Amryt intends to develop AP101 as a new treatment for Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin disorder affecting young children for which there is currently no treatment.   AP101 has been granted US and EU orphan drug designation.  It has patent grants in the US and in Europe, as well as other territories including Japan, Canada and Australia. The global market opportunity for EB is estimated to be in excess of EUR 1.3 billion.

Amryt's earlier stage product AP102 is focused on developing novel, next generation somatostatin analogue ("SSA") peptide medicines for patients with rare neuroendocrine diseases, where there is a high unmet medical need, including acromegaly and Cushing's disease. AP102 was recently granted orphan designation in the US in acromegaly by the FDA.

The Company joined AIM and Dublin's ESM in April 2016 following the reverse takeover of Fastnet Equity PLC.

 

 

 


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