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Phase III PEMPHIX Study Showed That Genentech’s Rituxan (Rituximab) is Superior to Standard of Care in Achieving Sustained Remission in Patients with Pemphigus Vulgaris

June 13, 2019 Off By BusinessWire

Rituxan met the primary and secondary endpoints in the Phase III
PEMPHIX study
Rituxan was the first and only biologic therapy approved by the FDA
in June 2018 for the treatment of pemphigus vulgaris based on results
from the Ritux 3 clinical trial

Results will be submitted to health authorities around the world,
including the U.S. Food and Drug Administration (FDA)

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY),
today announced positive top line results from the Roche-sponsored Phase
III PEMPHIX study evaluating the efficacy and safety of Rituxan^®
(rituximab) compared to mycophenolate mofetil (MMF) in adults with
moderate to severe pemphigus vulgaris (PV). The study met the primary
endpoint, and demonstrated that Rituxan is superior to MMF in achieving
sustained complete remission.

“The PEMPHIX study provides additional clinical evidence for the use of
Rituxan for the treatment of pemphigus vulgaris,” said Sandra Horning,
M.D., chief medical officer and head of Global Product Development.
“These data also demonstrated that Rituxan may provide complete
remission rates and successful tapering of corticosteroid therapy that
is superior to MMF in adults with pemphigus vulgaris.”

The primary endpoint was the percentage of participants who achieved
sustained complete remission off corticosteroid therapy (no disease
activity, as evaluated by Pemphigus Disease Area Index, without the use
of steroids for 16 consecutive weeks or more) at Week 52. Rituxan also
met the secondary endpoints, including cumulative corticosteroid dose,
number of flares, time to sustained remission and time to disease flare.
Adverse events were generally consistent with those seen in previous
Rituxan clinical studies for other autoimmune indications.

PV is a rare, serious and potentially life-threatening condition
characterized by progressive painful blistering of the skin and mucous
membranes.¹ MMF is an unapproved treatment for PV that is
accepted as standard of care. In June 2018, Rituxan became the first
biologic therapy approved by the FDA for PV and the first major
advancement in the treatment of the disease in more than 60 years. This
approval was based on the Ritux 3 clinical trial.² This
PEMPHIX trial provided additional clinical evidence on the effectiveness
of Rituxan for PV. Complete data from the PEMPHIX study will be
presented at an upcoming medical congress.

About the PEMPHIX study

PEMPHIX is a Phase III, randomized, double-blind, double-dummy,
active-comparator, parallel-arm multicenter study (NCT02383589) designed
to evaluate the efficacy and safety of Rituxan compared with
mycophenolate mofetil (MMF) in patients with moderate to severe active
pemphigus vulgaris requiring 60-120 mg/day oral prednisone (or
equivalent). Participants were randomly assigned to receive Rituxan plus
MMF placebo or Rituxan placebo plus MMF. Rituxan was administered at a
dose of 1000 mg via IV infusion on day 1 and 15, with a repeat
administration on days 168 and 182. MMF was administered at a dose of 2
grams orally daily from day 1 to week 52. The primary endpoint is the
percentage of participants who achieved sustained complete remission,
evaluated by the Pemphigus Disease Area Index (PDAI) Activity Score, for
at least 16 consecutive weeks at Week 52. Secondary endpoints include
cumulative oral corticosteroid dose, number of disease flares, time to
sustained complete remission, time to disease flare and health-related
quality of life, as measured by the Dermatology Life Quality Index.

About Pemphigus Vulgaris

Pemphigus vulgaris is an autoimmune blistering disease affecting the
skin and mucous membranes.¹ This rare, potentially
life-threatening condition accounts for up to 80 percent of cases of
pemphigus, a group of autoimmune disorders which affect 30,000 to 40,000
people in the United States.³

What autoimmune diseases does Rituxan treat?

Rheumatoid arthritis (RA): with another prescription medicine called
methotrexate, to reduce the signs and symptoms of moderate to severe
active RA in adults, after treatment with at least one other medicine
called a tumor necrosis factor (TNF) antagonist has been used and did
not work well enough.

Granulomatosis with Polyangiitis (GPA) (Wegener’s Granulomatosis) and
Microscopic Polyangiitis (MPA): with glucocorticoids, to treat GPA and
MPA.

Pemphigus vulgaris (PV): to treat adults with moderate to severe PV.

It is not known if Rituxan is safe or effective in children.

Important Side Effect Information

What is the most important information patients should know about
Rituxan?

Rituxan can cause serious side effects that can lead to death,
including:

Infusion-Related Reactions: Infusion-related
reactions are very common side effects of Rituxan treatment. Serious
infusion-related reactions can happen during your infusion or within
24 hours after your infusion of Rituxan. Your healthcare provider
should give you medicines before your infusion of Rituxan to decrease
your chance of having a severe infusion-related reaction
Severe Skin and Mouth Reactions: Tell
your healthcare provider or get medical help right away if you get any
of these symptoms at any time during your treatment with Rituxan:
painful sores or ulcers on your skin, lips, or in your mouth;
blisters; peeling skin; rash; or pustules
Hepatitis B Virus (HBV) Reactivation: If
you have had hepatitis B or are a carrier of hepatitis B virus,
receiving Rituxan could cause the virus to become an active infection
again. Hepatitis B reactivation may cause serious liver problems,
including liver failure and death. You should not receive Rituxan if
you have active hepatitis B liver disease. Your healthcare provider
will monitor you for hepatitis B infection during and for several
months after you stop receiving Rituxan
Progressive Multifocal Leukoencephalopathy
(PML): PML is a rare, serious brain infection
caused by a virus that can happen in people who receive Rituxan.
People with weakened immune systems can get PML. PML can result in
death or severe disability. There is no known treatment, prevention,
or cure for PML

Before receiving Rituxan, patients should tell their healthcare
provider if they:

have had a severe reaction to Rituxan or a rituximab product
currently have or have a history of other medical conditions,
especially heart disease
have had a severe infection, currently have an infection, or have a
weakened immune system
have had a recent vaccination or are scheduled to receive vaccinations
are pregnant or planning to become pregnant. Females who are able to
become pregnant should use effective birth control (contraception)
during treatment with Rituxan and for 12 months after the last dose of
Rituxan
are breastfeeding or plan to breastfeed. Patients should not
breastfeed during treatment and for at least 6 months after the last
dose of Rituxan
are taking any medications, including prescription and
over-the-counter medicines, vitamins, and herbal supplements

What are the possible side effects of Rituxan?

Rituxan can cause serious and life‐threatening side effects, including:

Tumor Lysis Syndrome (TLS): TLS is caused by the fast
breakdown of cancer cells. TLS can cause you to have kidney failure
and the need for dialysis treatment or may cause an abnormal heart
rhythm. Tell your healthcare provider right away if you have any of
the following signs or symptoms of TLS: nausea, vomiting, diarrhea, or
lack of energy
Serious Infections: Serious infections can happen during
and after treatment with Rituxan and can lead to death. Rituxan can
increase your risk of getting infections and can lower the ability of
your immune system to fight infections. People with serious infections
should not receive Rituxan
Heart Problems: Rituxan may cause chest pain, irregular
heartbeats, and heart attack. Your healthcare provider may monitor
your heart during and after treatment with Rituxan if you have
symptoms of heart problems or have a history of heart problems
Kidney Problems: especially if you are receiving Rituxan
for non–Hodgkin’s lymphoma (NHL). Your healthcare provider should do
blood tests to check how well your kidneys are working
Stomach and Serious Bowel Problems That Can Sometimes Lead to
Death: Tell your healthcare provider right away if you have
any stomach-area pain during treatment with Rituxan

What are the most common side effects during treatment with Rituxan?

The most common side effects of Rituxan include:

infusion-related reactions
infections (may include fever, chills)
body aches
tiredness
nausea

In patients with GPA or MPA, the most common side effects of Rituxan
also include:

low white and red blood cells
swelling
diarrhea
muscle spasms

Other side effects include:

aching joints during or within hours of receiving an infusion
more frequent upper respiratory tract infections

These are not all of the possible side effects with Rituxan. For more
information, ask a doctor or pharmacist.

Contact a doctor for medical advice about side effects. Report side
effects to the FDA at (800) FDA‐1088 or http://www.fda.gov/medwatch.
Patients may also report side effects to Genentech at (888) 835‐2555.

Please see the Rituxan Prescribing Information and Medication Guide
including Most Serious Side Effects for additional Important Side Effect
Information at http://www.rituxan.com.

Genentech and Biogen collaborate on Rituxan in the United States, and
Roche markets MabThera in the rest of the world, except Japan, where
Rituxan is co-marketed by Chugai and Zenyaku Kogyo Co. Ltd.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology
company that discovers, develops, manufactures and commercializes
medicines to treat patients with serious and life-threatening medical
conditions. The company, a member of the Roche Group, has headquarters
in South San Francisco, California. For additional information about the
company, please visit http://www.gene.com.

References

1. Medscape. Pemphigus Vulgaris. Available at: http://emedicine.medscape.com/article/1064187-overview.
[Last accessed: February 14, 2019]

2. Joly P, et al. First-Line Rituximab Combined with Short-Term
Prednisone Versus Prednisone Alone for the Treatment of Pemphigus (Ritux
3): A Prospective, Multicentre, Parallel-Group, Open-Label Randomised
Trial. The Lancet. March 22, 2017

3. International Pemphigus & Pemphigoid Foundation. Pemphigus. Available
at: http://www.pemphigus.org/research/clinically-speaking/pemphigus/.
[Last accessed: February 14, 2019]