Filing of the nonclinical package marks a key milestone in the process towards obtaining marketing approval of alicaforsen for the treatment of pouchitis
Cambridge, UK; May 1, 2017. Atlantic Healthcare plc (“Atlantic Healthcare”), an emerging trans-Atlantic pharmaceutical company with a core focus on gastrointestinal (GI) disorders, announces that it has initiated the rolling submission of its New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA), and filed its nonclinical package for alicaforsen to treat pouchitis.

Pouchitis is a rare and serious form of inflammatory bowel disease (IBD) for which there are limited treatment options. Alicaforsen enema has been granted Orphan Drug designation by the FDA and European Medicines Agency (EMA), and FDA Fast-Track designation in recognition of the unmet medical need for this disease.

Alicaforsen enema is currently being evaluated in a pivotal Phase 3 trial in pouchitis.
Toby Wilson Waterworth, CEO at Atlantic Healthcare, said: “The filing of the nonclinical data package initiates the regulatory process for obtaining marketing approval for alicaforsen in the treatment of pouchitis.This is a significant milestone in our corporate development.

“As an antisense therapy, alicaforsen enema has the potential to be first to market in a new class of drugs for the treatment of IBD. Alicaforsen also has the potential to treat multiple inflammatory diseases of the GI tract and elsewhere. Atlantic Healthcare is currently exploring additional formulations to extend the use of alicaforsen into other IBD indications.”

Janette Thomas, Director of International Operations at Atlantic Healthcare, said: “Earlier this year we reached agreement with the FDA on our request for a rolling submission for approval of alicaforsen in the treatment of pouchitis. We are delighted to announce today that the nonclinical data package has now been filed with the agency. This takes Atlantic Healthcare another step closer to bringing alicaforsen to market for patients with pouchitis, a debilitating disease where there remain unmet medical needs and no approved treatments.”

The rolling submission follows previously granted FDA Fast-Track designation for alicaforsen in the treatment of pouchitis. Fast-Track designation is designed to facilitate the development, and when appropriate expedite the review, of medicines that are intended to treat serious conditions and address unmet medical needs. It allows more frequent communication with the FDA, the option for rolling submission, and potential eligibility for FDA and EMA accelerated approvals.

For more information please contact:

Atlantic Healthcare
Toby Wilson Waterworth (CEO)
+44 1799 513 391

Adam Michael (Head of Communications)

+44 1799 512 055
+44 777 588 1813

adam.michael@atlantichc.com

U.S. Investor Relations and Media
Lazar Partners
Fern Lazar / David Carey

+1 212-867-1762

atlantic.healthcare@lazarpartners.com

European Investor Relations and Media
Consilium Strategic Communications

Mary-Jane Elliott / Matthew Neal

+44 20 3709 5700

atlantichealthcare@consilium-comms.com

About Atlantic Healthcare plc (www.atlantichc.com)

“Atlantic Healthcare” or “the Group” is an emerging trans-Atlantic pharmaceutical company with a core focus on gastrointestinal disorders including Inflammatory Bowel Disease (IBD). The Group’s lead product is alicaforsen enema, in a pivotal Phase 3 trial for pouchitis, and in preparation for Phase 3 clinical development in ulcerative colitis (UC). Alicaforsen is generating early repeat revenues in Europe through unsolicited requests via the Named Patient Supply protocols. The Group has FDA and EMA orphan drug designations, and FDA Fast-Track designation, in pouchitis. Atlantic Healthcare has a highly committed investor base, and an experienced international leadership team. Atlantic Healthcare’s fundraising to date includes £1.9m through SBRI funding with Innovate UK, the UK Government’s innovation agency (www.innovateuk.gov.uk). In 1Q 2016 the Group closed a $24m round led by LDC (the private equity division of Lloyd’s Banking Group) with new and existing investors.

About Alicaforsen

Alicaforsen is being developed as a locally active, topical treatment with the potential to establish a new class of therapy, with clear differentiating features, for the treatment of multiple inflammatory GI disorders.

Alicaforsen is currently in development for the treatment of inflammatory bowel disease (IBD). The enema formulation is currently in a Phase 3 trial agreed with U.S., Canadian and European regulatory agencies in patients with active, chronic pouchitis. The trial is recruiting 138 patients to approximately 40 trial centres across the U.S., Canada, Europe, and Israel. Alicaforsen enema is in preparation to commence a pivotal Phase 3 for active distal ulcerative colitis (UC). There is currently no approved treatment for pouchitis in the U.S. or Europe and there are limited treatment options for UC.

Atlantic Healthcare has received agreement from FDA to initiate a rolling submission of its New Drug Application (NDA) for alicaforsen to treat pouchitis. Alicaforsen has also been granted FDA Fast-Track designation, plus U.S. and European Orphan Drug designations for this indication.

Alicaforsen enema has shown, in five Phase 2 clinical studies involving 377 patients, to reduce inflammation and promote mucosal healing with good tolerability and a durable effect. Alicaforsen also has a good safety profile based on data from more than 1,000 patients (enema, parenteral, and IV delivery).

Alicaforsen is an antisense oligonucleotide which has been designed to interact with mRNA for ICAM-1, a well-recognised cell-surface protein that is involved in the inflammatory response of both the adaptive and innate components of the immune system, and is over-expressed in patients with IBD[1]. Research at Atlantic Healthcare has also provided evidence that alicaforsen can interact with TLR-9, an intracellular receptor involved in TLR-9 signalling pathways that activate cells of the innate immune system, and are involved in inflammatory responses[2].

Alicaforsen is generating early repeat revenues in Europe through unsolicited requests via Named Patient Supply protocols. This has provided further evidence of efficacy and safety in the pouchitis[3] and UC indications[4,5].

References:
Inflammatory Bowel Disease. Imre Szabo.ISBN 978-953-51-0879-5
Toll-like receptors: the swiss army knife of immunity and vaccine development Jennifer K Dowling and Ashley Mansell. Clinical & Translational Immunology (2016) 5, e85; doi:10.1038/cti.2016.22
Alicaforsen, an antisense inhibitor of ICAM-1, as treatment for chronic refractory pouchitis after proctocolectomy: A case series. Thomas Greuter, Luc Biedermann, Gerhard Rogler, Bernhard Sauter and Frank Seibold. UEG Journal (2015) DOI: 10.1177/2050640615593681

Alicaforsen Retention Enema Induces Long-Term Remission in Patients with Ulcerative Colitis. Zaid Heetun, David Gibson, Denise Keegan, Kathryn Byrne, Hugh E Mulcahy, Garret Cullen, Glen A Doherty. Irish Soc Gastroenterol Nov 2014

Alicaforsen, ICAM-1 enema as a treatment option when treating distal ulcerative colitis . LA. Bark 1 , I. Löfberg 1 , B. Håkansson 1 , U. Sjöqvist 1 (www.atlantichc.com/la-bark-abstract-pdf)