Lexington, Mass. – January 25, 2016 – Shire plc (LSE: SHP, NASDAQ: SHPG) today announced it has resubmitted the New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational candidate, lifitegrast, for the treatment of signs and symptoms of dry eye disease in adults. Shire resubmitted the NDA in response to the complete response letter (CRL) the company received from the FDA on October 16, 2015.
Addressing the FDA request for an additional study, Shire included in its NDA resubmission package data from OPUS-3, a Phase 3 efficacy and safety trial with a primary endpoint of patient-reported symptom improvement. The resubmission package also included information requested by the FDA regarding product quality.
“The NDA for lifitegrast now includes data from five randomized controlled clinical trials, with more than 2,500 patients, making it the largest data set for an investigational stage compound in dry eye disease to date,” said Philip J. Vickers, Ph.D., Head of Research & Development, Shire. “Because we believe that, if approved, lifitegrast has the potential to help the millions of U.S. adults living with symptoms of dry eye disease, we worked diligently to submit our response to the CRL as quickly as possible. This is an important milestone for Shire and underscores our commitment to providing innovative new treatment options in areas of unmet medical need.”
The new drug application for lifitegrast now includes data from one Phase 2 study, three Phase 3 efficacy and safety studies (OPUS-1, OPUS-2 and OPUS-3), and one long-term (one-year) Phase 3 safety study (SONATA).
The FDA has 30 days after resubmission of an NDA to acknowledge receipt and determine if the submission is a complete response. Upon acceptance, the FDA will provide Shire with a PDUFA date anticipated to be within six months of the date of submission.
Lifitegrast binds to the integrin lymphocyte function-associated antigen-1 (LFA-1), a cell surface protein found on leukocytes and blocks the interaction of LFA-1 with its cognate ligand intercellular adhesion molecule-1 (ICAM-1). ICAM-1 is over-expressed in corneal and conjunctival tissues in dry eye disease. LFA-1/ICAM-1 interaction contributes to formation of an immunological synapse resulting in T-cell activation and migration to target tissues. In vitro studies have demonstrated that lifitegrast inhibits T-cell adhesion to ICAM-1 expressing cells and inhibits secretion of key inflammatory cytokines (IFNγ, TNFα, IL-2) as well as inhibiting other proinflammatory cytokines: IL-1α, IL-1β, IL-2, IL-4, IL-5, and IL-13, all of which are known to be associated with dry eye disease.
OPUS-3, a Phase 3 study that evaluated the efficacy and safety of lifitegrast versus placebo, met the primary endpoint of significantly improving patient-reported symptoms of dry eye disease from baseline to day 84 (p=0.0007), and met key secondary endpoints of symptom improvement from baseline to days 14 and 42 (p<0.0001 for both endpoints).
About Dry Eye Disease
Dry eye is a multifactorial disease of the tears and ocular surface diagnosed by an eye care professional. It is diagnosed based on patient reported symptoms, such as eye dryness, overall eye discomfort, stinging, burning, a gritty feeling or fluctuating blurry vision, as well as signs, which can be measured by an eye care professional to determine the presence of dry eye disease (e.g., Schirmer test, corneal fluorescein staining, conjunctival lissamine green staining, and tear break-up time). Age and gender are recognized as traditional risk factors of dry eye disease while modern risk factors include prolonged screen time, contact lenses and cataract or refractive surgery. Dry eye is an often chronic ocular disease associated with inflammation that may eventually lead to damage to the surface of the eye. Dry eye may be progressive and is one of the most common complaints to eye care professionals.
Shire’s Commitment to Ophthalmics
In May 2014, Shire established its Ophthalmics Business Unit, solidifying its commitment to growing in this therapeutic area. Shire’s multi-faceted approach to discovery, development, and delivery in both rare diseases and specialty conditions includes our efforts to address unmet needs in eye care.
Shire’s ophthalmics business has been driven by a combination of strategic acquisitions and organic growth. Committed to growing its reputation as a leading biotech company, Shire is focused on continuing to expand its ophthalmics portfolio to include treatment options for rare diseases and those for anterior and posterior segment eye conditions. In just over two years, acquisitions include Foresight Biotherapeutics, SARcode Bioscience, Premacure AB, and BIKAM Pharmaceuticals, which have helped bolster Shire’s early-, mid- and late-stage ophthalmics pipeline. The Company currently has an ophthalmics pipeline of investigational candidates in dry eye disease, infectious conjunctivitis, retinopathy of prematurity, autosomal dominant retinitis pigmentosa, and glaucoma.
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Shire enables people with life-altering conditions to lead better lives.
Our strategy is to focus on developing and marketing innovative specialty medicines to meet significant unmet patient needs.
We focus on providing treatments in Rare Diseases, Neuroscience, Gastrointestinal and Internal Medicine and we are developing treatments for symptomatic conditions treated by specialist physicians in other targeted therapeutic areas, such as Ophthalmics.
Statements included herein that are not historical facts, including without limitation statements concerning our proposed business combination with Baxalta Incorporated (“Baxalta”) and the timing and financial and strategic benefits thereof, our 20x20 ambition that targets $20 billion in combined product sales by 2020, as well as other targets for future financial results, capital structure, performance and sustainability of the combined company, the combined company’s future strategy, plans, objectives, expectations and intentions, the anticipated timing of clinical trials and approvals for, and the commercial potential of, inline or pipeline products are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire’s results could be materially adversely affected. The risks and uncertainties include, but are not limited to, the following:
other risks and uncertainties detailed from time to time in Shire’s, Dyax’s or Baxalta’s filings with the Securities and Exchange Commission (“SEC”), including those risks outlined in Baxalta’s current Registration Statement on Form S-1, as amended, and in “Item 1A: Risk Factors” in Shire’s Annual Report on Form 10-K for the year ended December 31, 2014.
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