Shire plc Positive lanadelumab topline Phase 3 results

 
TIDMSHP 
 
Shire's Investigational Treatment Lanadelumab Reduces Hereditary Angioedema 
  Monthly Attack Rate by 87% Versus Placebo in Phase 3 26-week Pivotal Trial 
 
  * Significant reduction in monthly Hereditary Angioedema (HAE) attack rate 
    with infrequent subcutaneous administration 
 
  * Data to serve as basis for U.S. BLA filing expected in Q4 2017 to Q1 2018 
 
  * Shire to host Investor Call today at 10 a.m. ET; details below 
 
Lexington, MA - May 18, 2017 - Shire plc (LSE: SHP, NASDAQ: SHPG), the global 
leader in rare diseases, announces positive topline Phase 3 results for the 
HELPT study, a global, multi-center, randomized, double-blind 
placebo-controlled parallel group trial that evaluated the efficacy and safety 
of subcutaneously administered lanadelumab versus placebo over 26 weeks in 
patients 12 years of age or older with Hereditary Angioedema (HAE). Lanadelumab 
is an investigational treatment being evaluated for the prevention of 
angioedema attacks in patients with HAE, a rare genetic disease characterized 
by recurrent swelling of extremities, gastrointestinal tract, and upper 
airways. 
 
This study met its primary endpoint and all secondary endpoints with highly 
statistically significant and clinically meaningful results for all three 
lanadelumab treatment arms compared to placebo. The 300 mg dose administered 
once every two weeks resulted in a statistically significant reduction in mean 
HAE attack frequency of 87% compared to placebo (p <0.001). Results were 
consistent regardless of baseline attack rate. Notably for each of the three 
lanadelumab regimens studied, whether administered biweekly or monthly, a 
significantly higher proportion of patients-compared to placebo-were attack 
free throughout the entire 26 week study period. 
 
This study was representative of the full HAE disease spectrum. Overall, 52% of 
patients experienced three or more attacks per month at baseline, 65% of 
patients reported a history of laryngeal attacks and 56% were on long-term 
prophylaxis (LTP).  Ninety percent of patients completed the study. Ninety-six 
percent of those who completed the study chose to roll-over into the ongoing 
long-term safety study (HELPT Study Extension). 
 
"In the U.S., available treatment options include either injections for acute 
attacks or short-acting intravenous infusions administered twice a week," said 
Aleena Banerji, M.D., Massachusetts General Hospital, Boston, MA and clinical 
trial investigator. "If approved, lanadelumab may offer patients a long-acting 
treatment option that significantly reduces HAE attacks when administered 
subcutaneously as infrequently as every four weeks." 
 
HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 
50,000 people worldwide. The condition results in recurrent, localized edema 
(swelling). The areas of the body most commonly affected are the extremities, 
gastrointestinal tract, and upper airways. The swelling can be debilitating and 
painful, potentially impacting both work and education for people living with 
HAE. Swelling of the throat can be life-threatening due to asphyxiation. 
 
"The possibility of a new way to address the underlying cause of HAE to prevent 
attacks could transform how we treat the disease in the future," said Professor 
Marcus Maurer, M.D., Charité -Universitätsmedizin Berlin, Germany and clinical 
trial investigator. "Patients with HAE want to live independently and without 
fear of an angioedema attack." 
 
Lanadelumab was generally well tolerated over the 26-week treatment period. No 
treatment-related serious adverse events or deaths were reported. The most 
common adverse event was injection site pain (29.3% placebo vs. 42.9 % combined 
lanadelumab arms). 
 
"We are extremely encouraged by these topline Phase 3 results," said Flemming 
Ornskov, M.D., M.P.H., Shire Chief Executive Officer, "We have nearly a decade 
of experience and a strong portfolio and pipeline in HAE and believe these data 
demonstrate high potential for transforming the way patients living with this 
condition are treated." 
 
Shire plans to submit a biologics license application (BLA) for evaluation by 
the U.S. Food and Drug Administration (FDA) by late 2017 or early 2018. 
Lanadelumab has received both Orphan Drug Designation and Breakthrough Therapy 
Designation from the FDA and Orphan Drug Designation from the European 
Medicines Agency (EMA). 
 
About the HELPT Study 
 
The global, multicenter, randomized, parallel group, double-blind, 
placebo-controlled, Phase 3 trial is the largest prevention study conducted to 
date treating 125 patients 12 years of age or older with type I/II HAE. 
Patients were randomized into four arms to receive repeated subcutaneous 
administrations of lanadelumab 300 mg every two weeks, 300 mg every four weeks, 
150 mg every four weeks or placebo in a 2:1 ratio. The volume of drug 
administered at each injection in the clinical trial was 2 mL, administered 
subcutaneously as two 1 mL separate injections in the upper arm to maintain the 
study blind. 
 
The primary efficacy endpoint of the study was the number of 
investigator-confirmed angioedema attacks observed in each lanadelumab 
treatment arm versus placebo arm during the 26 week treatment period. 
 
Shire's Commitment to Hereditary Angioedema (HAE) 
Shire is a dedicated, long-term partner to the HAE community with nearly a 
decade of clinical and real-world experience supporting patients. We believe 
each patient deserves a right-fit approach to treatment, and our existing 
portfolio of products currently includes three distinct therapy options. We are 
committed to serial innovation and rely on our expertise to help fulfill unmet 
treatment needs for patients with HAE. Beyond our focus on developing novel 
treatments, we provide specialized services and support offerings that help 
meet the needs of the HAE community. Learn more at shire.com. 
 
About Lanadelumab 
 
Lanadelumab is an investigational fully human monoclonal antibody that 
specifically binds and inhibits plasma kallikrein and is being developed as a 
treatment for the prevention of angioedema attacks in patients with HAE. 
Lanadelumab is formulated for subcutaneous administration with a half-life of 
approximately 14 days in patients with HAE. 
 
For further information please contact: 
 
 
Media Relations 
 
Elizabeth Kalina                      [e-mail]    [phone] 
 
Katie Joyce                           [e-mail]     [phone] 
 
 
 
Investor Relations 
 
Ian Karp                              [e-mail]      [phone] 
 
Robert Coates                         [e-mail]    [phone] 
 
Live conference call for investors: 
 
Shire's Management Team will host a conference call for investors and analysts 
today, May 18, 2017 at 10 a.m., EDT. 
 
The details of the conference call are as follows: 
 
UK dial in:                  0808 237 0030 or 020 3139 4830 
 
US dial in:                  1 866 928 7517 or 1 718 873 9077 
 
International Access         events.arkadin.com/ev/docs/ 
Numbers:                     NE_FEL_Events_International_Access_List.pdf 
 
Password/Conf ID:            93795050# 
 
Live Webcast:                investors.shire.com/presentations-and-webcasts 
 
Replay: 
 
A replay of the presentation will be available for two weeks by phone and by 
webcast for three months.  Replay information can be found on the Investor 
Relations section of Shire's website at http://investors.shire.com/. 
 
NOTES TO EDITORS 
 
Stephen Williams, Deputy Company Secretary is responsible for arranging the 
release of this announcement. 
 
Inside Information 
 
This announcement contains inside information. 
 
About Shire 
 
Shire is the leading global biotechnology company focused on serving people 
with rare diseases and other highly specialized conditions. We strive to 
develop best-in-class products, many of which are available in more than 100 
countries, across core therapeutic areas including Hematology, Immunology, 
Neuroscience, Ophthalmics, Lysosomal Storage Disorders, Gastrointestinal / 
Internal Medicine / Endocrine and Hereditary Angioedema; and a growing 
franchise in Oncology. 
 
Our employees come to work every day with a shared mission: to develop and 
deliver breakthrough therapies for the hundreds of millions of people in the 
world affected by rare diseases and other high-need conditions, and who lack 
effective therapies to live their lives to the fullest. 
 
www.shire.com 
 
Forward-Looking Statements 
 
Statements included herein that are not historical facts, including without 
limitation statements concerning 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: 
 
  * Shire's products may not be a commercial success; 
 
  * increased pricing pressures and limits on patient access as a result of 
    governmental regulations and market developments may affect Shire's future 
    revenues, financial condition and results of operations; 
 
  * Shire conducts its own manufacturing operations for certain of its products 
    and is reliant on third party contract manufacturers to manufacture other 
    products and to provide goods and services. Some of Shire's products or 
    ingredients are only available from a single approved source for 
    manufacture. Any disruption to the supply chain for any of Shire's products 
    may result in Shire being unable to continue marketing or developing a 
    product or may result in Shire being unable to do so on a commercially 
    viable basis for some period of time; 
 

  * the manufacture of Shire's products is subject to extensive oversight by 
    various regulatory agencies. Regulatory approvals or interventions 
    associated with changes to manufacturing sites, ingredients or 
    manufacturing processes could lead to, among other things, significant 
    delays, an increase in operating costs, lost product sales, an interruption 
    of research activities or the delay of new product launches; 
 
  * certain of Shire's therapies involve lengthy and complex processes, which 
    may prevent Shire from timely responding to market forces and effectively 
    managing its production capacity; 
 
  * Shire has a portfolio of products in various stages of research and 
    development. The successful development of these products is highly 
    uncertain and requires significant expenditures and time, and there is no 
    guarantee that these products will receive regulatory approval; 
 
  * the actions of certain customers could affect Shire's ability to sell or 
    market products profitably. Fluctuations in buying or distribution patterns 
    by such customers can adversely affect Shire's revenues, financial 
    conditions or results of operations; 
 
  * Shire's products and product candidates face substantial competition in the 
    product markets in which it operates, including competition from generics; 
 
  * adverse outcomes in legal matters, tax audits and other disputes, including 
    Shire's ability to enforce and defend patents and other intellectual 
    property rights required for its business, could have a material adverse 
    effect on the Company's revenues, financial condition or results of 
    operations; 
 
  * inability to successfully compete for highly qualified personnel from other 
    companies and organizations; 
 
  * failure to achieve the strategic objectives, including expected operating 
    efficiencies, cost savings, revenue enhancements, synergies or other 
    benefits at the time anticipated or at all with respect to Shire's 
    acquisitions, including NPS Pharmaceuticals Inc., Dyax Corp. or Baxalta 
    Incorporated may adversely affect Shire's financial condition and results 
    of operations; 
 
  * Shire's growth strategy depends in part upon its ability to expand its 
    product portfolio through external collaborations, which, if unsuccessful, 
    may adversely affect the development and sale of its products; 
 
  * a slowdown of global economic growth, or economic instability of countries 
    in which Shire does business, as well as changes in foreign currency 
    exchange rates and interest rates, that adversely impact the availability 
    and cost of credit and customer purchasing and payment patterns, including 
    the collectability of customer accounts receivable; 
 
  * failure of a marketed product to work effectively or if such a product is 
    the cause of adverse side effects could result in damage to Shire's 
    reputation, the withdrawal of the product and legal action against Shire; 
 
  * investigations or enforcement action by regulatory authorities or law 
    enforcement agencies relating to Shire's activities in the highly regulated 
    markets in which it operates may result in significant legal costs and the 
    payment of substantial compensation or fines; 
 
  * Shire is dependent on information technology and its systems and 
    infrastructure face certain risks, including from service disruptions, the 
    loss of sensitive or confidential information, cyber-attacks and other 
    security breaches or data leakages that could have a material adverse 
    effect on Shire's revenues, financial condition or results of operations; 
 
  * Shire incurred substantial additional indebtedness to finance the Baxalta 
    acquisition, which may decrease its business flexibility and increase 
    borrowing costs; and 
 
    a further list and description of risks, uncertainties and other matters 
    can be found in Shire's most recent Annual Report on Form 10-K and in 
    Shire's subsequent Quarterly Reports on Form 10-Q, in each case including 
    those risks outlined in "ITEM 1A: Risk Factors", and in Shire's subsequent 
    reports on Form 8-K and other Securities and Exchange Commission filings, 
    all of which are available on Shire's website. 
 
    All forward-looking statements attributable to us or any person acting on 
    our behalf are expressly qualified in their entirety by this cautionary 
    statement. Readers are cautioned not to place undue reliance on these 
    forward-looking statements that speak only as of the date hereof. Except to 
    the extent otherwise required by applicable law, we do not undertake any 
    obligation to update or revise forward-looking statements, whether as a 
    result of new information, future events or otherwise. 
 
 
 
END 
 

(END) Dow Jones Newswires

May 18, 2017 07:00 ET (11:00 GMT)