– Independent data monitoring committee
recommends pausing study enrollment due to lack of efficacy in
pre-specified interim analysis –
– Company will conduct further analysis of
trial data to determine next steps –
– No new safety findings were observed for
ULTOMIRIS use in COVID-19 –
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the
decision to pause further enrollment in the global Phase 3 study of
ULTOMIRIS® (ravulizumab-cwvz) in adults with severe COVID-19
requiring mechanical ventilation. This decision is based on the
recommendation of an independent data monitoring committee (IDMC),
following their review of data from a pre-specified interim
analysis. The IDMC recommended that additional enrollment be
paused, pending further analysis of the data, due to lack of
efficacy when ULTOMIRIS was added to best supportive care, compared
to best supportive care alone. There were no new safety findings
observed. The study will continue for patients already enrolled,
including completion of all study visits and planned ULTOMIRIS
dosing according to the study protocol.
“We would like to thank the patients and their families, as well
as investigators and healthcare professionals, who were essential
to this study. We greatly value their contributions to help
investigate potential ways to address this devastating pandemic,”
said John Orloff, M.D., Executive Vice President and Head of
Research & Development at Alexion. “While initial anecdotal
reports from compassionate use cases were promising, these results
demonstrate the importance of conducting controlled clinical trials
to fully evaluate the potential of new treatment approaches and
generate the necessary evidence to make informed decisions. We are
disappointed in this initial outcome, but plan to further analyze
the data to identify potential subgroups who may benefit and to
determine next steps. In addition, we remain fully committed to our
efforts to serve the rare disease community and to continuing to
provide ULTOMIRIS to the patients who currently rely on it.”
The IDMC’s recommendation was based on a pre-planned interim
analysis of the primary endpoint – survival at Day 29 – once 122
patients completed the 29-day primary evaluation period. No
secondary endpoints were analyzed as part of the interim
analysis.
In the UK, the TACTIC-R platform study led by Cambridge
University Hospitals NHS Foundation Trust, which includes an
ULTOMIRIS cohort, is evaluating the potential of earlier immune
modulatory treatment (hospitalized patients not requiring
mechanical ventilation) in preventing progression of the virus,
including reducing the need for ICU admission and ventilation. This
independent study remains ongoing.
About ULTOMIRIS® (ravulizumab-cwvz)
ULTOMIRIS® (ravulizumab--cwvz) is the first and only long-acting
C5 complement inhibitor. The medication works by inhibiting the C5
protein in the terminal complement cascade, a part of the body’s
immune system. When activated in an uncontrolled manner, the
complement cascade over-responds, leading the body to attack its
own healthy cells. ULTOMIRIS is administered intravenously every
eight weeks or, for pediatric patients less than 20 kg, every four
weeks, following a loading dose. ULTOMIRIS is approved in the
United States (U.S.), European Union (EU) and Japan as a treatment
for adults with paroxysmal nocturnal hemoglobinuria (PNH). It is
also approved in the U.S. and Japan for atypical hemolytic uremic
syndrome (aHUS) to inhibit complement-mediated thrombotic
microangiopathy (TMA) in adult and pediatric (one month of age and
older) patients, as well as in the EU for the treatment of adults
and children with a body weight of at least 10 kg with aHUS.
INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS®
(ravulizumab-cwvz)
INDICATIONS
What is ULTOMIRIS?
ULTOMIRIS is a prescription medicine used to treat:
- adults with a disease called Paroxysmal Nocturnal
Hemoglobinuria (PNH).
- 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.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about
ULTOMIRIS?
ULTOMIRIS is a medicine that affects your immune system and
can lower the ability of your immune system to fight
infections.
- ULTOMIRIS increases your chance of getting serious and
life-threatening meningococcal infections that may quickly become
life-threatening and cause death if not recognized and treated
early.
- You must receive meningococcal vaccines at least 2 weeks before
your first dose of ULTOMIRIS if you are not vaccinated.
- If your doctor decided that urgent treatment with ULTOMIRIS is
needed, you should receive meningococcal vaccination as soon as
possible.
- If you have not been vaccinated and ULTOMIRIS therapy must be
initiated immediately, you should also receive 2 weeks of
antibiotics with your vaccinations.
- If you had a meningococcal vaccine in the past, you might need
additional vaccination. Your doctor will decide if you need
additional vaccination.
- Meningococcal vaccines reduce but do not prevent all
meningococcal infections. Call your doctor or get emergency medical
care right away if you get any of these signs and symptoms of a
meningococcal infection: 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.
Your doctor will give you a Patient Safety Card about the
risk of meningococcal infection. Carry it with you at all times
during treatment and for 8 months after your last ULTOMIRIS dose.
It is important to show this card to any doctor or nurse to help
them diagnose and treat you quickly.
ULTOMIRIS is only available through a program called the
ULTOMIRIS REMS. Before you can receive ULTOMIRIS, your doctor
must: enroll in the ULTOMIRIS REMS program; counsel you about the
risk of meningococcal infection; give you information and a
Patient Safety Card about the symptoms and your risk of
meningococcal infection (as discussed above); and make sure that
you are vaccinated with a meningococcal vaccine, and if needed, get
revaccinated with the meningococcal vaccine. Ask your doctor if you
are not sure if you need to be revaccinated.
ULTOMIRIS may also increase the risk of other types of
serious infections. Make sure your child receives vaccinations
against Streptococcus pneumoniae and Haemophilis influenzae type b
(Hib) if treated with ULTOMIRIS. Call your doctor right away if you
have any new signs or symptoms of infection.
Who should not receive ULTOMIRIS?
Do not receive ULTOMIRIS if you have a meningococcal
infection or have not been vaccinated against meningococcal
infection unless your doctor decides that urgent treatment with
ULTOMIRIS is needed.
Before you receive ULTOMIRIS, tell your doctor about all of
your medical conditions, including if you: have 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 your unborn baby or if it passes into your breast milk.
You should not breastfeed during treatment and for 8 months after
your final dose of ULTOMIRIS.
Tell your doctor about all the vaccines you receive and
medicines you take, including prescription and over-the-counter
medicines, vitamins, and herbal supplements which could affect your
treatment.
If you have PNH and you stop receiving ULTOMIRIS, your doctor
will need to monitor you closely for at least 16 weeks after you
stop ULTOMIRIS. Stopping ULTOMIRIS may cause breakdown of your red
blood cells due to PNH. Symptoms or problems that can happen due to
red blood cell breakdown include: drop in your red blood cell
count, tiredness, blood in your urine,
stomach-area (abdomen) pain, shortness of breath,
blood clots, trouble swallowing, and erectile dysfunction
(ED) in males.
If you have aHUS, your doctor will need to monitor you
closely for at least 12 months after stopping treatment for signs
of worsening aHUS or problems related to a type of abnormal
clotting and breakdown of your 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.
What are the possible side effects of ULTOMIRIS?
ULTOMIRIS can cause serious side effects including
infusion-related reactions. Symptoms of an infusion-related
reaction with ULTOMIRIS may include lower back pain, pain with the
infusion, feeling faint or discomfort in your arms or legs. Tell
your doctor or nurse right away if you develop these symptoms, or
any other symptoms during your ULTOMIRIS infusion that may mean you
are having a serious infusion reaction, including: chest pain,
trouble breathing or shortness of breath, swelling of your face,
tongue, or throat, and feel faint or pass out.
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 infection, diarrhea, nausea, vomiting,
headache, high blood pressure and fever.
Tell your doctor about any side effect that bothers you or that
does not go away. These are not all the possible side effects of
ULTOMIRIS. For more information, ask your doctor or pharmacist.
Call your doctor right away if you miss an ULTOMIRIS infusion or
for medical advice about side effects. You may report side effects
to FDA at 1-800-FDA-1088.
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 and devastating
conditions through the discovery, development and commercialization
of life-changing medicines. As a leader in rare diseases for more
than 25 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)
as well as the first and only approved Factor Xa inhibitor reversal
agent. In addition, the company is developing several
mid-to-late-stage therapies, including a copper-binding agent for
Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for
rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D
inhibitor as well as several early-stage therapies, including one
for light chain (AL) amyloidosis, a second oral Factor D inhibitor
and a third complement inhibitor. 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, cardiology
and ophthalmology. 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]
Forward-Looking Statement
This press release may include statements that are or may be
deemed to be forward-looking statements. These forward-looking
statements may be identified by the use of forward-looking
terminology, including the terms "believes", "estimates",
"envisages", "plans", "projects", "anticipates", "targets", "aims",
"expects", "intends", "may", "will" or "should" or, in each case,
their negative or other variations or comparable terminology, or by
discussions of strategy, plans, objectives, goals, future events or
intentions and include, but are not limited to the plans of Alexion
with respect to ULTOMIRIS as a potential treatment for severe
COVID-19 patients. Economic, competitive, governmental,
technological and other factors that may affect Alexion's
operations are discussed in the section entitled "Risk Factors,"
Alexion's Quarterly Report on Form 10-Q for the Period ended 30
September 2020, as amended by any subsequent filings made with the
SEC. These forward-looking statements include all matters that are
not historical facts and involve predictions. Forward-looking
statements may and often do differ materially from actual results.
Any forward-looking statements reflect Alexion's current views with
respect to future events and are subject to risks relating to
future events and other risks, uncertainties and assumptions
relating to Alexion's results of operations, financial position,
liquidity, prospects, growth or strategies and the industries in
which they operate. Forward-looking statements speak only as of the
date they are made and cannot be relied upon as a guide to future
performance. Save as required by law or regulation, Alexion
disclaims any obligation or undertaking to release publicly any
updates or revisions to any forward-looking statements in this
press release that may occur due to any change in its expectations
or to reflect events or circumstances after the date of this press
release.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210113005889/en/
Media Megan Goulart, 857-338-8634 Executive Director,
Corporate Communications
Investors Chris Stevo, 857-338-9309 Head of Investor
Relations
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