Existing efficacy and safety data support progress of alicaforsen targeted-release tablets into Phase 1 development

Cambridge, UK; May 30, 2017. Atlantic Healthcare plc (“Atlantic Healthcare”), an emerging trans-Atlantic pharmaceutical company with a core focus on gastrointestinal (GI) disorders, announces the development of two separate tablet formulations of alicaforsen, for targeted delivery in the GI tract, to treat Crohn’s disease and Ulcerative Colitis (UC).

Atlantic Healthcare is preparing Phase 1 trials that are designed to confirm safety and the release profile of the drug at the appropriate site in the GI tract. The UC formulation is designed to release at the terminal ileum for delivery to the colon, and the formulation for Crohn’s disease is designed to release in the small intestine.
Alicaforsen, as an enema formulation, is currently being evaluated in a pivotal Phase 3 trial, and has initiated its rolling submission to the U.S. Food and Drug Administration (FDA), to treat pouchitis, a rare and serious form of inflammatory bowel disease (IBD) that has limited treatment options. The company plans to commercialise alicaforsen in the U.S. and Europe and licence commercial rights in the rest of the world.

Toby Wilson Waterworth, CEO at Atlantic Healthcare, said: “The development and approval of alicaforsen tablet formulations would complement the existing enema, and substantially expand the commercial potential of the product. The two new tablet formulations are designed to deliver alicaforsen to specific points in the GI tract, inaccessible to the enema, to treat Crohn’s disease and UC. Alicaforsen has the potential to treat multiple GI inflammatory diseases. We are very excited by the prospect of providing clinicians and patients with additional options to treat these serious diseases, which have substantial unmet clinical need.”

Dr. Janette Thomas, Director of International Operations at Atlantic Healthcare said: “The new tablet formulations harness recent advances in encapsulation and targeted-release technologies for oligonucleotides such as alicaforsen. The aim is to deliver the product to sites of disease, with minimal systemic absorption.
Based on the existing clinical and safety data that includes treatment in over 1,000 patients, a positive read-out from Phase 1, confirming safety and drug release at the appropriate site of disease in the GI tract, would enable progression to Phase 2b, dose escalation studies.”


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


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

+1 212-867-1762


European Investor Relations and Media
Consilium Strategic Communications

Mary-Jane Elliott / Matthew Neal

+44 20 3709 5700



Notes to Editors:

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). The Group is also developing two tablet formulations of alicaforsen, to treat Crohn’s disease and 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 IBD. The enema formulation is currently in a pivotal Phase 3 trial agreed with U.S., Canadian and European regulatory agencies in patients with active 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.

Under a rolling submission agreement with the FDA, Atlantic Healthcare has filed the nonclinical data package of its New Drug Application (NDA) for alicaforsen to treat pouchitis with the regulator. 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 with UC and pouchitis, 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 and down regulate 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 involved in inflammatory responses[2].

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

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)