LEXINGTON, Massachusetts,
May 18, 2017 /PRNewswire/ --
- 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
Shire plc (LSE: SHP, NASDAQ: SHPG), the global leader in rare
diseases, announces positive topline Phase 3 results for the HELP™
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 (HELP™ 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 HELP™ 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.
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
Numbers: Click here
Password/Conf ID: 93795050#
Live Webcast: Click here
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.
http://www.shire.com
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