Shire plc Positive lanadelumab topline Phase 3 results
May 18 2017 - 6:00AM
UK Regulatory
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 ekalina@shire.com +1 781 482 2713
Katie Joyce kjoyce@shire.com +1 781 482 2779
Investor Relations
Ian Karp ikarp@shire.com +1 781 482 9018
Robert Coates rcoates@shire.com +44 1256 894874
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
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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
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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
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