Phase III PEMPHIX Study Shows Genentech’s Rituxan (rituximab) Superior to Mycophenolate Mofetil in Patients With Pemphigus Vulgaris

• 40% of patients with pemphigus vulgaris (PV) achieved sustained complete remission, without the use of steroids for 16 weeks or more, when treated with Rituxan compared to 9.5% of patients on mycophenolate mofetil
• Study reinforces efficacy and safety of Rituxan for treatment of PV, a rare autoimmune condition characterized by blistering of the skin and mucous membranes
• Full data of the 52-week treatment period presented at 28th Congress of the European Academy of Dermatology and Venereology (EADV) in Madrid

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced data from the 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 at week 52 and demonstrated that Rituxan is superior to MMF, with 40.3% of patients treated with Rituxan achieving sustained complete remission (CR) without the use of steroids for 16 consecutive weeks or more, compared to 9.5% in the MMF arm (p<0.0001). All secondary endpoints were statistically significant in favor of Rituxan: lower cumulative oral corticosteroid dose (mean difference: 1595 mg; p=0.0005), fewer flares (6 vs. 44, p<0.0001), a greater likelihood of sustained CR (hazard ratio [HR]=4.83; p=0.0003), a lesser likelihood of flare (HR=0.15; p<0.0001) and a greater improvement in the Dermatology Life Quality Index (DLQI) at week 52 (estimated mean change from baseline -8.87 vs. -6.00, p=0.0012) compared to the MMF arm. Adverse events were generally consistent with those seen in previous Rituxan clinical studies in PV and other approved autoimmune indications. Results were presented as a late-breaking oral presentation at the 28th Congress of the European Academy of Dermatology and Venereology in Madrid, Spain on October 12 at 9:00 a.m. – 9:15 a.m. CEST (Presentation D3T01.1C).

“The approval of Rituxan for the treatment of pemphigus vulgaris was the first major advancement in the treatment of this rare, serious disease in more than 60 years,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “The PEMPHIX study showed that 40% of people in the study could achieve complete remission from painful blistering without the need for corticosteroids for 16 weeks or more and that Rituxan may be a superior treatment option to mycophenolate mofetil.”

The study is ongoing, with patients participating in a 48-week safety follow-up period after treatment completion or discontinuation.

PV is a rare, serious and potentially life-threatening condition characterized by progressive painful blistering of the skin and mucous membranes. MMF is an unapproved, commonly used treatment for PV that is recommended in published treatment guidelines. Rituxan became the first biologic therapy for PV when it was approved by the U.S. Food and Drug Administration (FDA) in June 2018 and the European Commission in March 2019. These approvals were based on data from the Roche-supported Ritux 3 clinical trial. The PEMPHIX study provides additional clinical evidence of the effectiveness of Rituxan for PV.

About the PEMPHIX Study

PEMPHIX is a Phase III, randomized, double-blind, double-dummy, active-comparator, parallel-arm, international, 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 for 52 weeks, in combination with 60 or 80 mg oral prednisone, with the aim of tapering to 0 mg/day by week 24. Rituxan was administered at a dose of 1000 mg via intravenous (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 (starting at 1 g/day on day 1 and titrated to achieve a goal of 2 g/day by week 2). The primary endpoint at week 52 was the percentage of participants who achieved sustained complete remission without experiencing treatment failure. Sustained complete remission was defined as achieving healing of lesions with no new active lesions (i.e., Pemphigus Disease Area Index activity score of 0) while on 0 mg/day prednisone or equivalent, and maintaining this response for at least 16 consecutive weeks, during the 52-week treatment period. Secondary endpoints were 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. This rare, potentially life-threatening condition accounts for up to 80% of cases of pemphigus, a group of autoimmune disorders which affect 30,000 to 40,000 people in the United States.

What autoimmune diseases does Rituxan treat?

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

People with Granulomatosis with Polyangiitis (GPA) (Wegener’s Granulomatosis) and Microscopic Polyangiitis (MPA) ages 2 years and above: with glucocorticoids.

Adults with Pemphigus Vulgaris (PV): to treat moderate to severe PV.

Rituxan is not indicated in children less than 2 years of age with GPA or MPA or in children with conditions other than GPA or MPA.

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
• have a history of other medical conditions including:
  • Heart problems
  • Irregular heartbeat
  • Chest pain
  • Lung or kidney problems
• 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. You should not get certain vaccines before or during treatment with Rituxan
• have any other medical conditions
• are pregnant or planning to become pregnant. Talk to your healthcare provider about the risks to your unborn baby if you receive Rituxan during pregnancy. 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. Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with Rituxan
• are breastfeeding or plan to breastfeed. It is not known if Rituxan passes into your breast milk. Do not breastfeed during treatment and for at least 6 months after your last dose of Rituxan
• are taking any medications, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Especially tell your healthcare provider if you take or have taken:
  • A tumor necrosis factor (TNF) inhibitor medicine
  • A disease modifying anti-rheumatic drug (DMARD)

What are the possible side effects of Rituxan?

Rituxan can cause serious 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 an abnormal heart rhythm. TLS can happen within 12-24 hours after an infusion of Rituxan. 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. Tell your healthcare provider right away if you have chest pain or irregular heartbeats during treatment with Rituxan
• Kidney Problems: especially if you are receiving Rituxan for non–Hodgkin’s lymphoma (NHL). Rituxan can cause severe kidney problems that lead to death. 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: Bowel problems, including blockage or tears in the bowel, can happen if you receive Rituxan with chemotherapy medicines. Tell your healthcare provider right away if you have any stomach-area pain during treatment with Rituxan

Your healthcare provider will stop treatment with Rituxan if you have severe, serious, or life-threatening side effects.

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

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

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.

Contact a healthcare provider 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.

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