Recognition of alicaforsen in Rare Disease Drug Development category highlights commitment to addressing unmet need in inflammatory bowel disease

Cambridge, UK, February 28, 2018. Atlantic Healthcare plc (“Atlantic Healthcare”), an emerging trans-Atlantic pharmaceutical company with a core focus on gastrointestinal (GI) disorders, announces that it has been selected as a finalist for the Clinical & Research Excellence (CARE) Awards 2018 in the Excellence in Rare Disease Drug Development category. The award in this category is given to the drug development program or trial with the largest potential impact in the rare disease space. The 2018 CARE Award winners will be announced at a ceremony on April 25th.

Toby Wilson Waterworth, CEO at Atlantic Healthcare said: “Being chosen as a finalist for this prestigious award is especially gratifying today, which is Rare Disease Day 2018. Pouchitis is one of a number of rare and serious gastrointestinal diseases with significant unmet need that alicaforsen could potentially address. We are committed to improving the care and outcomes for patients with these rare diseases, and we are honoured that a prestigious group of healthcare industry leaders recognizes the value and important potential of our efforts to achieve this important objective.”

The nomination in the Excellence in Rare Disease Drug Development reflects Atlantic Healthcare’s leadership in advancing alicaforsen for the treatment of pouchitis, a rare and serious form of inflammatory bowel disease (IBD) for which there is no approved therapy and limited treatment options. Alicaforsen is an antisense oligonucleotide, which has been designed to interact and down regulate mRNA for ICAM-1, a well-recognized 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. Atlantic Healthcare is currently conducting a Phase 3 randomized, double-blind, placebo-controlled trial designed to assess the safety and efficacy of alicaforsen, as an enema formulation, in patients with active, chronic pouchitis who have failed to adequately respond to a course of antibiotics, or who are contraindicated for antibiotics.

Atlantic Healthcare expects to complete the ongoing Phase 3 trial in pouchitis and report preliminary results by the end of 2018. A rolling submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for alicaforsen in the treatment of pouchitis was initiated in May 2017. Alicaforsen has also been granted Orphan Drug status by the FDA and the European Medicines Agency (EMA), and received FDA Fast-Track designation in this indication.

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. Atlantic Healthcare makes alicaforsen available to patients 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 Pouchitis

Many people with ulcerative colitis need to have their diseased colon removed and the bowel reconnected with a procedure called pouch surgery (ileoanal anastomosis — IPAA). Surgeons use the end of the small intestine (ileum) to create a pouch, typically shaped like the letter J. The pouch is attached internally to the area just above the anus to hold waste before being eliminated.

Pouchitis is a complication of J pouch surgery. It occurs in approximately half of the people who have the procedure. Signs and symptoms of pouchitis can include inflammation and ulceration, diarrhea, abdominal pain, cramps, fever, an increased number of bowel movements, night-time fecal seepage, fecal incontinence, and a strong urge to have a bowel movement.

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. Alongside the pouchitis study, the alicaforsen enema formulation 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 signaling pathways that activate cells of the innate immune system involved in inflammatory responses.

[2] Atlantic Healthcare makes alicaforsen available to patients 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. [4-6]

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)
Alicaforsen, an antisense inhibitor of Intercellular Adhesion Molecule-1, in the treatment for left-sided ulcerative colitis and ulcerative proctitis. Thomas Greuter, Stephan Vavricka, Luc Biedermann, Julia Pilz, Jan Borovicka, Frank Seibold, Bernard Sauter, Gerhard Rogler. Dig Dis (2017) DOI: 10.1159/000484979

 

Forward Looking Statements

While Atlantic Healthcare believes the forward-looking statements in this press release are accurate, the statements represent Atlantic Healthcare’s beliefs only as of the date of this press release. A number of factors could cause actual events or results to differ materially from those indicated by such statements. These forward-looking statements involve risks and uncertainties, including, among others, that the development of alicaforsen for the stated uses may not proceed due to safety, efficacy or other reasons. In addition, risks and results in clinical trials may not be indicative of risks or results from later stage or larger scale trials, and there is no assurance of regulatory approval. Existing and prospective investors should not place undue reliance on the forward-looking statements contained in this press release and instead should make their own determinations as to the reliability of such statements. Atlantic Healthcare undertakes no intent or obligation to update the information contained in this press release as new information becomes available.

 

For more information please contact:

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

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atlantic.healthcare@lazarpartners.com

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Consilium Strategic Communications

Mary-Jane Elliott / Matthew Neal

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