AbbVie Announces Positive Topline Results from Phase 3 Trial Evaluating VENCLEXTA™/VENCLYXTO™ (Venetoclax) Tablets in Combination with Rituxan® (rituximab) for the Treatment of Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia

On September 18, 2017 AbbVie (NYSE: ABBV), a global research and development-based biopharmaceutical company, reported that the Phase 3 MURANO study of VENCLEXTA/VENCLYXTO (Venetoclax) Tablets in combination with Rituxan (rituximab) met its primary endpoint (Press release, AbbVie, SEP 18, 2017, View Source [SID1234520550]). Results showed that VENCLEXTA/VENCLYXTO in combination with Rituxan prolonged progression-free survival (PFS) in patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) compared with bendamustine combined with Rituxan. An independent data monitoring committee reviewed this study and made the recommendation to unblind the trial based on the positive results. Doctors will continue to monitor patients who remain active in the MURANO trial in efforts to obtain additional, longer-term safety and efficacy information. VENCLEXTA/VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S.

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"AbbVie is committed to researching the full potential of VENCLEXTA/VENCLYXTO both as monotherapy and combination therapy in patients with CLL and other hematologic malignancies. The analysis of the MURANO trial showed that VENCLEXTA/VENCLYXTO in combination with Rituxan may offer another option for patients with R/R CLL, potentially providing them with a chemotherapy-free therapy," said Michael Severino, M.D., executive vice president, research and development, and chief scientific officer, AbbVie. "We are looking forward to working with regulatory authorities around the world to bring this additional treatment regimen to R/R CLL patients."

The most common type of leukemia in the Western world is CLL, which accounts for approximately one-quarter of new cases of leukemia in the U.S.1,2 CLL is a slow-growing form of leukemia, or blood cancer, in which too many immature lymphocytes (type of white blood cells) are found predominantly in the blood and bone marrow.3 CLL usually affects older patients, with more men than women affected. The median age at diagnosis is approximately 70 years.1

Full data from this study will support regulatory submissions for VENCLEXTA/VENCLYXTO in combination with Rituxan therapy in R/R CLL, and will be presented at an upcoming medical conference. Safety data, including serious and most common adverse events and discontinuation rates, are currently being analyzed.

About the Phase 3 Study
The multicenter, open-label, randomized Phase 3 MURANO study was designed to evaluate the efficacy and safety of VENCLEXTA/VENCLYXTO in combination with Rituxan compared with bendamustine in combination with Rituxan in patients with R/R CLL.4 The primary endpoint was investigator-assessed progression-free survival (PFS), which was determined using standard International Workshop on Chronic Lymphocytic Leukemia (iwCLL) guidelines. Secondary endpoints included Independent Review Committee (IRC)-assessed PFS, as well as PFS in patients with 17p deletion, best overall response (defined as complete response [CR], complete response with incomplete marrow recovery [CRi], nodular partial remission [nPR], or PR), overall survival, event-free survival, duration of response, time to next anti-CLL treatment, and percentage of patients achieving minimal residual disease negativity.

About VENCLEXTA/VENCLYXTO
VENCLEXTA/VENCLYXTO is an oral B-cell lymphoma-2 (BCL-2) inhibitor that targets a specific protein in the body called BCL-2. When you have CLL, BCL-2 may build up and prevent cancer cells from self-destructing naturally. VENCLEXTA/VENCLYXTO targets BCL-2 in order to help restore the process of apoptosis. Through apoptosis, your body allows cancer cells and normal cells to self-destruct.

VENCLEXTA/VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research with venetoclax, which is currently being evaluated in clinical trials in several hematologic cancers.

VENCLEXTA/VENCLYXTO is under evaluation by health authorities in multiple countries, and is currently approved in 16 nations, including the U.S., and in the EU. AbbVie, in collaboration with Roche and Genentech, is currently working with regulatory agencies around the world to bring this medicine to eligible patients in need.

About VENCLYXTO (venetoclax) Tablets (EU)
VENCLYXTO (venetoclax) is indicated in the European Union (EU) for the treatment of chronic lymphocytic leukemia (CLL) in the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor; and for the treatment of CLL in the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B-cell receptor pathway inhibitor.5 It is also being evaluated for the treatment of patients with various blood cancer types.5,6,7,8,9 The BCL-2 protein prevents apoptosis (programmed cell death) of some cells, including lymphocytes, and can be overexpressed in CLL cells.5 VENCLYXTO, which is given once-daily, is designed to selectively inhibit the function of the BCL-2 protein.5

Important VENCLYXTO (venetoclax) EU Safety Information
Contraindications
Hypersensitivity to the active substance or to any of the excipients. Concomitant use of strong CYP3A inhibitors at initiation and during the dose-titration phase. Concomitant use of preparations containing St. John’s wort.

Special Warnings & Precautions for Use
Tumor lysis syndrome (TLS), including fatal events, has occurred in patients with previously treated CLL with high tumor burden when treated with VENCLYXTO. VENCLYXTO poses a risk for TLS in the initial 5-week dose-titration phase. Changes in electrolytes consistent with TLS that require prompt management can occur as early as 6 to 8 hours following the first dose of VENCLYXTO and at each dose increase. Patients should be assessed for risk and should receive appropriate prophylaxis for TLS. Blood chemistries should be monitored and abnormalities managed promptly. More intensive measures (including IV hydration, frequent monitoring and hospitalization) should be employed as overall risk increases.

Neutropenia (grade 3 or 4) has been reported and complete blood counts should be monitored throughout the treatment period.

Live vaccines should not be administered during treatment or thereafter until B-cell recovery.

Drug Interactions
CYP3A inhibitors may increase VENCLYXTO plasma concentrations. At initiation and dose-titration phase: Strong CYP3A inhibitors are contraindicated due to increased risk for TLS and moderate CYP3A inhibitors should be avoided. If moderate CYP3A inhibitors must be used, physicians should refer to the SmPC for dose adjustment recommendations. At steady daily dose: If moderate or strong CYP3A inhibitors must be used, physicians should refer to the SmPC for dose adjustment recommendations.

Avoid concomitant use of P-gp and BCRP inhibitors at initiation and during the dose titration phase.

CYP3A4 inducers may decrease VENCLYXTO plasma concentrations.

Avoid co-administration with strong or moderate CYP3A inducers. These agents may decrease venetoclax plasma concentrations.

Co-administration of bile acid sequestrants with VENCLYXTO is not recommended as this may reduce the absorption of VENCLYXTO.

Adverse Reactions
The most commonly occurring adverse reactions (>=20%) of any grade were neutropenia/neutrophil count decreased, diarrhea, nausea, anemia, upper respiratory tract infection, fatigue, hyperphosphatemia, vomiting and constipation.

The most frequently occurring adverse reactions (>=2%) were pneumonia, febrile neutropenia and TLS.

Discontinuations due to adverse reactions occurred in 9.1% of patients and dosage adjustments due to adverse reactions occurred in 11.8% of patients.

Specific Populations
VENCLYXTO may cause embryo-fetal harm when administered to a pregnant woman. Advise females of reproductive potential to avoid pregnancy during treatment. Advise nursing women to discontinue breastfeeding during treatment.

Safety in patients with severe renal impairment or on dialysis has not been established, and a recommended dose has not been determined. VENCLYXTO should be administered to patients with severe renal impairment only if the benefit outweighs the risk. Monitor closely for signs of toxicity due to increased risk of TLS.

This is not a complete summary of all safety information. See VENCLYXTO full summary of product characteristics (SmPC) at www.ema.europa.eu. Globally, prescribing information varies; refer to the individual country product label for complete information.

About VENCLEXTA (venetoclax) tablets (US)
In April 2016, the U.S. Food and Drug Administration (FDA) granted accelerated approval of VENCLEXTA (venetoclax) tablets for the treatment of patients with CLL with 17p deletion, as detected by an FDA-approved test, who have received at least one prior therapy.10 The FDA approved this indication under accelerated approval based on overall response rate, and continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial. In January 2016, AbbVie announced that the FDA granted Breakthrough Therapy Designation (BTD) for venetoclax in combination with rituximab for the treatment of patients with R/R CLL.

What is VENCLEXTA (venetoclax)?
VENCLEXTA (venetoclax) is a prescription medicine used to treat people with chronic lymphocytic leukemia (CLL) with 17p deletion who have received at least one prior treatment.

VENCLEXTA was approved based on response rate. There is an ongoing study to find out how VENCLEXTA works over a longer period of time.

It is not known if VENCLEXTA is safe and effective in children.

Important VENCLEXTA (venetoclax) US Safety Information

What is the most important information I should know about VENCLEXTA?
VENCLEXTA can cause serious side effects, including:
Tumor lysis syndrome (TLS). TLS is caused by the fast breakdown of cancer cells. TLS can cause kidney failure, the need for dialysis treatment, and may lead to death. Your doctor will do tests for TLS. It is important to keep your appointments for blood tests. You will receive other medicines before starting and during treatment with VENCLEXTA to help reduce your risk of TLS. You may also need to receive intravenous (IV) fluids into your vein. Tell your doctor right away if you have any symptoms of TLS during treatment with VENCLEXTA, including fever, chills, nausea, vomiting, confusion, shortness of breath, seizures, irregular heartbeat, dark or cloudy urine, unusual tiredness, or muscle or joint pain.

Drink plenty of water when taking VENCLEXTA to help reduce your risk of getting TLS. Drink 6 to 8 glasses (about 56 ounces total) of water each day, starting 2 days before your first dose, on the day of your first dose of VENCLEXTA, and each time your dose is increased.

Who should not take VENCLEXTA?
Certain medicines must not be taken when you first start taking VENCLEXTA and while your dose is being slowly increased.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. VENCLEXTA and other medicines may affect each other, causing serious side effects.
Do not start new medicines during treatment with VENCLEXTA without first talking with your doctor.
What should I tell my doctor before taking VENCLEXTA?
Before taking VENCLEXTA, tell your doctor about all of your medical conditions, including if you:

Have kidney or liver problems.
Have problems with your body salts or electrolytes, such as potassium, phosphorus, or calcium
Have a history of high uric acid levels in your blood or gout
Are scheduled to receive a vaccine. You should not receive a "live vaccine" before, during or after treatment with VENCLEXTA until your doctor tells you it is okay. If you are not sure about the type of immunization or vaccine, ask your doctor. These vaccines may not be safe or may not work as well during treatment with VENCLEXTA.
Are pregnant or plan to become pregnant. VENCLEXTA may harm your unborn baby. If you are able to become pregnant, your doctor should do a pregnancy test before you start treatment with VENCLEXTA, and you should use effective birth control during treatment and for 30 days after the last dose of VENCLEXTA.
Are breastfeeding or plan to breastfeed. It is not known if VENCLEXTA passes into your breast milk. Do not breastfeed during treatment with VENCLEXTA.
What should I avoid while taking VENCLEXTA?
You should not drink grapefruit juice or eat grapefruit, Seville oranges (often used in marmalades), or starfruit while you are taking VENCLEXTA. These products may increase the amount of VENCLEXTA in your blood.

What are the possible side effects of VENCLEXTA?
VENCLEXTA can cause serious side effects, including:

Low white blood cell count (neutropenia). Low white blood cell counts are common with VENCLEXTA, but can also be severe. Your doctor will do blood tests to check your blood counts during treatment with VENCLEXTA. Tell your doctor right away if you have a fever or any signs of an infection.
The most common side effects of VENCLEXTA include low white blood cell count, diarrhea, nausea, low red blood cell count, upper respiratory tract infection, low platelet count, and feeling tired.

VENCLEXTA may cause fertility problems in males. This may affect your ability to father a child. Talk to your doctor if you have concerns about fertility.

These are not all the possible side effects of VENCLEXTA. Tell your doctor if you have any side effect that bothers you or that does not go away.

The full U.S. prescribing information for VENCLEXTA can be found here. Globally, prescribing information varies; refer to the individual country product label for complete information.

Patient Assistance
For those who qualify, patient assistance options are available for people taking VENCLEXTA in the U.S.