- 50-week global study to enroll ~350 patients
across broad ALS population -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the
planned initiation of a pivotal Phase 3 study of ULTOMIRIS®
(ravulizumab) in amyotrophic lateral sclerosis (ALS). The 50-week
global study, called CHAMPION-ALS, will evaluate approximately 350
adults across a broad patient population, and the primary endpoint
will be change in ALS functional rating scale-revised (ALSFRS-R)
score. Alexion submitted an investigational new drug application
(IND) for ULTOMIRIS in ALS to the FDA in the fourth quarter of 2019
and plans to initiate the Phase 3 study this quarter.
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“Given the significant need for new and improved treatments for
ALS, we are committed to advancing this clinical program with
urgency,” said John Orloff, M.D., Executive Vice President and Head
of Research & Development at Alexion. “Based on preclinical
data and the significant role complement activation is known to
play in other neuromuscular diseases, we believe ULTOMIRIS has the
potential to inhibit complement-mediated damage in people with ALS,
which may slow disease progression. We thank the ALS community for
their involvement in designing this Phase 3 program and look
forward to continued close collaboration as we move it
forward.”
“We’ve made great progress advancing ALS research in recent
years, but there is still more work to be done to ensure patients
benefit from these advances in the form of new treatments,” said
Calaneet Balas, CEO and President of The ALS Association. “It’s
great that Alexion, which has an established record of bringing new
treatments to patients with devastating rare diseases, is joining
the fight against ALS.”
About the Phase 3 CHAMPION-ALS Study The Phase 3
CHAMPION-ALS trial is a randomized, double-blind,
placebo-controlled multicenter global study designed to evaluate
the efficacy and safety of ULTOMIRIS across a broad ALS population.
The study will enroll approximately 350 adults with sporadic or
familial ALS who have had disease onset (in the form of first motor
symptoms) within the prior 36 months, demonstrate a slow vital
capacity (SVC) of at least 65 percent predicted, and are not
dependent on respiratory support.
Study participants will be randomized on a 2:1 basis to receive
ULTOMIRIS or placebo every 8 weeks following an initial loading
dose and may continue to receive their existing standard of care
treatment for ALS. After 50 weeks, all patients will receive
ULTOMIRIS in a 2-year open-label extension phase of the study. The
study will be conducted at approximately 90 clinical trial sites
across North America, Europe and Asia-Pacific.
The primary study endpoint will be change from baseline in ALS
functional rating scale-revised (ALSFRS-R) score. Secondary
endpoints will include ventilation assistance-free survival (VAFS),
respiratory capacity, muscle strength, neurofilament light chain
(NfL) serum concentrations and safety.
About ALS Amyotrophic lateral sclerosis (ALS) is a
neurological disorder characterized by progressive degeneration of
nerve cells (motor neurons) in the brain and the spinal cord that
control muscles throughout the body. When the nerve cells die, the
brain can no longer initiate and control muscle movement, which
results in severe disability, paralysis and eventually death.
ALS is a relentlessly progressive disorder. People with ALS may
lose the ability to speak, eat, move and breathe. The rate of
progression between individuals is variable and the natural history
generally reflects progressive worsening over time until death
occurs. The average life expectancy from symptom onset is between
two and five years. Currently approved medications may slow disease
progression, but ALS management is mostly supportive, palliative
and symptom based.
An estimated 15,000 to 20,000 people are living with ALS across
the United States, France, Germany, Italy, Spain, the United
Kingdom and Japan. There are two different types of ALS, sporadic
and familial. Sporadic, which is the most common form of the
disease, accounts for 85 to 90 percent of cases and may affect
anyone, anywhere. Familial ALS, the inherited form of the disease,
accounts for 10 to 15 percent of cases.
About ULTOMIRIS® (ravulizumab-cwvz) ULTOMIRIS®
(ravulizumab-cwvz) is the first and only approved long-acting C5
complement inhibitor. It is administered intravenously every eight
weeks or every four weeks for pediatric patients less than 20 kg,
following a loading dose. ULTOMIRIS works by inhibiting the C5
protein in the terminal complement cascade, a part of the body’s
immune system. The terminal complement cascade, when activated in
an uncontrolled manner, plays a role in severe ultra-rare
disorders. ULTOMIRIS is approved in the U.S., Japan, and the EU as
a treatment for adults with PNH and in the U.S. for aHUS to inhibit
complement-mediated thrombotic microangiopathy (TMA) in adult and
pediatric (one month of age and older) patients.
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR
ULTOMIRIS (ravulizumab-cwvz) 300 mg / 30 mL injection for
intravenous use INDICATIONS ULTOMIRIS is a prescription
medicine called a monoclonal antibody. ULTOMIRIS is used to treat
adults with a disease called Paroxysmal Nocturnal Hemoglobinuria
(PNH). ULTOMIRIS is used to treat adults and children 1 month of
age and older with a disease called atypical Hemolytic Uremic
Syndrome (aHUS). ULTOMIRIS is not used in treating people with
Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
It is not known if ULTOMIRIS is safe and effective in children with
PNH. It is not known if ULTOMIRIS is safe and effective in children
younger than 1 month of age in aHUS.
IMPORTANT SAFETY INFORMATION ULTOMIRIS is a medicine that
affects the immune system. ULTOMIRIS can lower the ability of the
immune system to fight infections. ULTOMIRIS increases the chance
of getting serious and life-threatening meningococcal infections.
Meningococcal infections may quickly become life-threatening and
cause death if not recognized and treated early.
Meningococcal vaccines must be received at least 2 weeks before
the first dose of ULTOMIRIS if one has not already had this
vaccine. If one’s doctor decides that urgent treatment with
ULTOMIRIS is needed, meningococcal vaccination should be
administered as soon as possible. If one has not been vaccinated
and ULTOMIRIS therapy must be initiated immediately, 2 weeks of
antibiotics should also be administered with the vaccinations. If
one had a meningococcal vaccine in the past, additional vaccination
might be needed before starting ULTOMIRIS. One’s doctor will decide
if additional meningococcal vaccination is needed. Meningococcal
vaccines reduce the risk of meningococcal infection but do not
prevent all meningococcal infections. Call one’s doctor or get
emergency medical care right away if any of these signs and
symptoms of a meningococcal infection occur: headache with nausea
or vomiting, headache and fever, headache with a stiff neck or
stiff back, fever, fever and a rash, confusion, muscle aches with
flu-like symptoms, and eyes sensitive to light. One’s doctor will
give a Patient Safety Card about the risk of meningococcal
infection. Carry the card at all times during treatment and for 8
months after your the ULTOMIRIS dose.
ULTOMIRIS is only available through a program called the
ULTOMIRIS REMS.
ULTOMIRIS may also increase the risk of other types of serious
infections. People who take ULTOMIRIS may have an increased risk of
getting infections caused by Streptococcus pneumoniae and
Haemophilus influenzae. Certain people may also have an increased
risk of gonorrhea infection. To find out if one is at risk for
gonorrhea infection, about gonorrhea prevention, and regular
testing, talk to the doctor. Call the doctor right away if one has
any new signs or symptoms of infection.
Do not receive ULTOMIRIS if one has a meningococcal infection,
or has not been vaccinated against meningococcal infection unless
the doctor decides that urgent treatment with ULTOMIRIS is
needed.
Before one receives ULTOMIRIS, tell the doctor about all of the
medical conditions, including if one: has an infection or fever,
are pregnant or plan to become pregnant, and are breastfeeding or
plan to breastfeed. It is not known if ULTOMIRIS will harm an
unborn baby. It is not known if ULTOMIRIS passes into the breast
milk. One should not breastfeed during treatment and for 8 months
after one’s final dose of ULTOMIRIS.
Tell the doctor about all the medicines one takes, including
prescription and over-the-counter medicines, vitamins, and herbal
supplements. ULTOMIRIS and other medicines can affect each other
causing side effects. Know the medicines one takes and the vaccines
one receives. Keep a list of them to show the doctor and pharmacist
when one gets a new medicine.
If one has PNH and stops receiving ULTOMIRIS, the doctor will
need to monitor closely for at least 16 weeks after one stops
ULTOMIRIS. Stopping ULTOMIRIS may cause breakdown of the red blood
cells due to PNH. Symptoms or problems that can happen due to red
blood cell breakdown include: drop in the red blood cell count,
tiredness, blood in the urine, stomach-area (abdomen) pain,
shortness of breath, blood clots, trouble swallowing, and erectile
dysfunction (ED) in males. If one has aHUS, the doctor will need to
monitor closely for at least 12 months after stopping treatment for
signs of worsening aHUS symptoms or problems related to a type of
abnormal clotting and breakdown of the red blood cells called
thrombotic microangiopathy (TMA). Symptoms or problems that can
happen with TMA may include: confusion or loss of consciousness,
seizures, chest pain (angina), difficulty breathing, and blood
clots or stroke. If one misses an ULTOMIRIS infusion, call the
doctor right away.
ULTOMIRIS can cause serious side effects including infusion
reactions. Infusion reactions may happen during one’s ULTOMIRIS
infusion. Symptoms of an infusion reaction with ULTOMIRIS may
include lower back pain, pain with the infusion, feeling faint or
discomfort in the arms or legs. Tell the doctor or nurse right away
if these symptoms develop, or any other symptoms during the
ULTOMIRIS infusion that may mean one is having a serious infusion
reaction, including: chest pain, trouble breathing or shortness of
breath, swelling of the face, tongue, or throat, and feel faint or
pass out. One’s doctor will treat the symptoms as needed.
The most common side effects of ULTOMIRIS in people treated for
PNH are upper respiratory infection and headache. The most common
side effects of ULTOMIRIS in people with aHUS are upper respiratory
infections, diarrhea, nausea, vomiting, headache, high blood
pressure, and fever.
Please see the accompanying full Prescribing Information and
Medication Guide for ULTOMIRIS, including Boxed WARNING regarding
serious and life-threatening meningococcal
infections/sepsis.
About Alexion Alexion is a global biopharmaceutical
company focused on serving patients and families affected by rare
diseases through the discovery, development and commercialization
of life-changing therapies. As the global leader in complement
biology and inhibition for more than 20 years, Alexion has
developed and commercializes two approved complement inhibitors to
treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and
atypical hemolytic uremic syndrome (aHUS), as well as the first and
only approved complement inhibitor to treat anti-acetylcholine
receptor (AChR) antibody-positive generalized myasthenia gravis
(gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion
also has two highly innovative enzyme replacement therapies for
patients with life-threatening and ultra-rare metabolic disorders,
hypophosphatasia (HPP) and lysosomal acid lipase deficiency
(LAL-D). In addition, the company is developing several
mid-to-late-stage therapies, including a second complement
inhibitor, a copper-binding agent for Wilson disease and an
anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G
(IgG)-mediated diseases as well as several early-stage therapies,
including one for light chain (AL) amyloidosis and a second
anti-FcRn therapy. Alexion focuses its research efforts on novel
molecules and targets in the complement cascade and its development
efforts on the core therapeutic areas of hematology, nephrology,
neurology, metabolic disorders and cardiology. Headquartered in
Boston, Massachusetts, Alexion has offices around the globe and
serves patients in more than 50 countries. This press release and
further information about Alexion can be found at:
www.alexion.com.
[ALXN-G]
For patient or advocacy inquiries please contact
patientadvocacy@alexion.com.
Forward-Looking Statement This press release contains
forward-looking statements, including statements related to:
Alexion’s future plans to initiate a pivotal Phase 3 study of
ULTOMIRIS® (ravulizumab) in ALS; ULTOMIRIS has the potential to
inhibit complement-mediated damage in people with ALS, which may
slow disease progression, based on preclinical data and the
significant role complement activation is known to play in other
neuromuscular diseases; the potential benefits of ULTOMIRIS to
people with ALS; anticipated future continued close collaboration
with the ALS community as Alexion moves forward with the clinical
trial; the protocol and method of the ULTOMIRIS clinical trial; and
the timing of anticipated completion of clinical trials.
Forward-looking statements involve risks and uncertainties relating
to future events and the future performance of Alexion and are
subject to factors that may cause Alexion's results and plans to
differ materially from those expected by these forward looking
statements, including for example: drug development and
commercialization involve a high degree of risk, and only a small
number of research and development programs result in
commercialization of a product; the initiation of a pivotal Phase 3
study of ULTOMIRIS in ALS may be delayed or may never be initiated;
the results of the pivotal Phase 3 study of ULTOMIRIS in ALS may
not result in regulatory approval necessary to commercialize
ULTOMIRIS for this indication (or regulatory authorities may
require additional trials to confirm safety and efficacy of
ULTOMIRIS in ALS prior to approval which would be expensive and
time-consuming); ULTOMIRIS may not gain market acceptance and/or
may not be recognized by patients and physicians as a treatment for
ALS; the benefits (including safety and efficacy), if any, of
ULTOMIRIS evidenced in clinical trials may not be witnessed in a
broader patient population; any potential post-approval
restrictions that regulatory authorities may impose on ULTOMIRIS as
a treatment for ALS; future competition from biosimilars and other
products; decisions of regulatory authorities regarding the
adequacy of our research, marketing approval or material
limitations on the marketing of our products; delays or the
inability to launch product candidates due to regulatory
restrictions, anticipated expense or other matters; interruptions
or failures in the manufacture and supply of our products and our
product candidates; failure to satisfactorily address matters
raised by regulatory agencies with respect to product candidates;
results in early stage clinical trials may not be indicative of
full results or results from later stage or larger clinical trials
(or broader patient populations) and do not ensure regulatory
approval; the possibility that results of clinical trials are not
predictive of safety and efficacy and potency of our products (or
we may fail to adequately operate or manage our clinical trials)
which could cause us to halt trials, delay or prevent us from
making regulatory approval filings or result in denial of approval
of our product candidates; unexpected delays in clinical trials;
the adequacy of our pharmacovigilance and drug safety reporting
processes; failure to protect and enforce our data, intellectual
property and proprietary rights and the risks and uncertainties
relating to intellectual property claims and challenges against us
(including intellectual property lawsuits relating to ULTOMIRIS
brought by third parties against Alexion); the risk that third
party payers (including governmental agencies) will not reimburse
or continue to reimburse for the use of our products at acceptable
rates or at all; uncertainties surrounding legal proceedings
(including intellectual property suits initiated against Alexion
and our products), company investigations and government
investigations, including investigations of Alexion by the U.S.
Securities and Exchange Commission (SEC) and U.S. Department of
Justice; the risks of changing foreign exchange rates; and a
variety of other risks set forth from time to time in Alexion's
filings with the SEC, including but not limited to the risks
discussed in Alexion's Quarterly Report on Form 10-Q for the period
ended September 30, 2019 and in our other filings with the SEC.
Alexion disclaims any obligation to update any of these
forward-looking statements to reflect events or circumstances after
the date hereof, except when a duty arises under law.
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version on businesswire.com: https://www.businesswire.com/news/home/20200114005254/en/
Media Megan Goulart, 857-338-8634 Senior Director,
Corporate Communications
Investors Susan Altschuller, Ph.D., 857-338-8788 Vice
President, Investor Relations
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