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.

Formula Pharmaceuticals Announces Initiation of the First Clinical Trial for Allogeneic CIK-CAR Cancer Immunotherapy

On September 18, 2017 Formula Pharmaceuticals, Inc. ("Formula") reported the opening of the first clinical trial for allogeneic Cytokine Induced Killer (C.I.K.) cell-based Chimeric Antigen Receptor (CAR) cancer immunotherapy (Press release, Formula Pharmaceuticals, SEP 18, 2017, View Source [SID1234520545]).

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This open-label, dose-escalating clinical Phase 1/2a trial will evaluate the safety, pharmacokinetics (PK), and antitumor activity of modified allogeneic C.I.K. cells administered to pediatric and adult patients with relapsed or refractory acute lymphoblastic leukemia (ALL). This clinical trial, sponsored by Fondazione Matilde Tettamanti and supported in part by Formula in accordance with the Italian/EU regulations on investigator-initiated trials, will be conducted at the MBBM Foundation/San Gerardo Hospital in Monza, Italy, and the Azienda Ospedaliera Papa Giovanni XXIII in Bergamo, Italy, both of which are affiliated with the University of Milan-Bicocca.

"We are very proud to have led the C.I.K. CAR program from the bench to the clinic and look forward to demonstrating the therapeutic potential of this platform for patients with persistent unmet need," commented Prof. Andrea Biondi, Director of the Pediatric and Pediatric Hematologic Oncology Clinic at the University of Milan-Bicocca, MBBM Foundation/San Gerardo Hospital, and Scientific Director at the Fondazione M. Tettamanti in Monza, Italy. "We look forward to expanding our close collaboration with Formula into additional hematologic cancer applications."

"This clinical trial is the first to evaluate non-virally transfected, allogeneic C.I.K. CAR in patients with cancer and is designed to demonstrate important clinical proof of concept for our entire platform," said Maurits Geerlings, MD, president and CEO of Formula. "We are honored to support Fondazione M. Tettamanti in reaching this important milestone."

Formula’s product pipeline is based on a proprietary non-viral, allogeneic C.I.K. CAR technology platform, which has the potential to overcome practical, therapeutic and commercial limitations related to existing CAR-T approaches that involve autologous blood, viral transfection and restricted immune effector functionality. Formula’s C.I.K. CAR technology activates T cell and Natural Killer cell functionality within one effector cell population, sourced from healthy donor or cord blood. With this approach, the Company is developing off-the-shelf CAR immunotherapies for various hematologic and solid tumor indications.

Autolus Announces First-Dose Cohort Completed in APRIL Study of AUTO2: A Phase I/II Study in Patients with Multiple Myeloma

On September 18, 2017 UCL Business spinout company, Autolus Limited, a clinical-stage biopharmaceutical company focused on the development and commercialisation of next-generation engineered T-cell therapies, reported completion of the first-dose cohort of its phase I/II study of its novel, dual-targeted Chimeric Antigen Receptor (CAR) in patients with relapsed/refractory multiple myeloma (Press release, UCLB, SEP 18, 2017, http://www.uclb.com/news-and-events/news-post/autolus-announces-first-dose-cohort-completed-in-april-study-of-auto2-a-phase-iii-study-in-patients-with-multiple-myeloma [SID1234520543]).

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AUTO2 is a chimeric antigen receptor T-cell (CAR-T cell) therapy that targets both B-cell maturation antigen (BCMA) and transmembrane activator and calcium modulator and cyclophilin ligand interactor (TACI). The APRIL Study is a dose-escalation phase I/II study in which cohorts of patients receive ascending doses of AUTO2 to determine the maximum tolerated dose and establish a recommended dose. The second part of the study is an expansion phase where patients receive AUTO2 to further evaluate the safety, tolerability and clinical activity at this recommended dose.

Dr Jesus G. Berdeja, Director of Myeloma Research & Senior Investigator, Hematologic Malignancies, Sarah Cannon Research Institute said:
"BCMA CAR-T cell therapies have shown considerable promise in early clinical studies. A dual-targeted approach may minimise the risk for antigen negative escape and extend CAR-T treatment to patients with low density of BCMA antigen on the surface their cancer cells."

Dr Christian Itin, Autolus’ CEO added:
"Breaking the defence mechanisms of cancers against T-cells is key to unlocking the curative potential of CAR-T cell therapies. AUTO2 is a first example of Autolus’ approach to specifically re-programme the patient’s own T-cells to minimise the risk of the cancer cells escaping treatment. With the start of the APRIL study we have transitioned to a clinical stage company; an important step on our path to build a fully integrated autologous CAR-T cell company with a portfolio of differentiated therapies for the treatment of patients with cancer."

RedHill Biopharma Receives Notice of Allowance for a New U.S. Patent Covering its Combination Therapy for Hard-to-Treat Cancers

On September 18, 2017 RedHill Biopharma Ltd. (NASDAQ:RDHL) (Tel-Aviv Stock Exchange:RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company primarily focused on late clinical-stage development and commercialization of proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, reported that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a new patent covering the use of two of RedHill’s Phase II-stage proprietary investigational compounds, YELIVA and MESUPRON in combination with a known antibiotic (Press release, RedHill Biopharma, SEP 18, 2017, View Source [SID1234520542]).

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Upon issuance, on top of existing intellectual property protection covering the individual compounds, the new patent will provide RedHill with intellectual property protection covering its combination for the potential treatment of cancer, prevention of cancer recurrence or progression and inhibition of growth and proliferation of cancer cells.

Danielle Abramson, Ph.D., RedHill’s VP of Intellectual Property and Research, stated: "We are very pleased with the allowance of this key patent, covering the combination of our proprietary, first-in-class, orally-administered Phase II-stage drug candidates, YELIVA and MESUPRON, both of which are new chemical entities, with a known antibiotic. As part of our oncology program, RedHill is currently evaluating the new combination therapy across several oncology indications with limited treatment options where strong unmet medical need exists."

DelMar Pharmaceuticals Receives IND Allowance from FDA to Initiate Clinical Trials of VAL-083 for the Treatment of Ovarian Cancer

On September 18, 2017 DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) ("DelMar" and "the Company"), a biopharmaceutical company focused on the development of new cancer therapies, reported that the U.S. Food and Drug Administration ("FDA") has allowed an additional Investigational New Drug Application ("IND") to study its lead drug candidate VAL-083 as a potential treatment for ovarian cancer (Press release, DelMar Pharmaceuticals, SEP 18, 2017, View Source [SID1234520541]).

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"The opening of this new IND to study VAL-083 in ovarian cancer marks a major milestone for our Company as we continue to investigate this agent as an important potential therapy for the treatment of multiple cancers," said Jeffrey Bacha, DelMar’s president and chief executive officer.

VAL-083 is a first-in-class, DNA targeting agent that demonstrated clinical activity against a range of tumor-types in prior clinical trials sponsored by the U. S. National Cancer Institute ("NCI"). Published results from NCI studies include recommendations for further study of VAL-083 in advanced clinical trials for ovarian cancer and other gynecologic malignancies.

DelMar’s clinical trial will be a multi-center, Phase 1/2 Study of VAL-083 in patients with Recurrent Platinum Resistant Ovarian Cancer ("VAL-083 REPROVe Trial"). DelMar’s research demonstrates that VAL-083’s unique mechanism of action has the potential to overcome chemo-resistance to platinum-based chemotherapy in ovarian, lung and other solid tumors.

Ovarian cancer remains the leading cause of death among women with gynecological cancers and the fifth most frequent cause of cancer deaths in women overall. The American Cancer Society estimates that in 2017, approximately 22,440 women in the US will be diagnosed with ovarian cancer and approximately 14,080 will die from their disease. The majority of these deaths were patients whose tumors had become resistant to platinum-based chemotherapy regimens. Currently, there are no high-efficacy therapeutic options for platinum-resistant ovarian cancer, leaving these cancer patients with very poor prognosis. According to published literature, the overall response rate ("ORR") to second line therapy is in the 10-15% range and overall survival is approximately 12-months.

"The development of new treatments to overcome platinum resistance represents the largest unmet medical need in the treatment of ovarian cancer," stated Dr. Bradley J. Monk, MD, principal investigator of the VAL-083 REPROVe Trial and director of the Division of Gynecologic Oncology Research at Arizona Oncology. "Based on DelMar’s recent presentation of pre-clinical data demonstrating activity of VAL-083 against platinum-resistant ovarian cancer, we are enthusiastic about exploring the drug’s potential in this important clinical setting."

About the VAL-083 REPROVe Trial

The VAL-083 REPROVe Trial is an open label, multi-center, Phase 1/2 clinical trial to evaluate the safety and efficacy of VAL-083 in patients with recurrent adenocarcinoma of the ovary, who have been previously treated with a minimum of two courses of platinum-based chemotherapy, and whose cancer has recurred within six months of the most recent platinum-based chemotherapy. Patients enrolled in the trial will receive VAL-083 intravenously once per week for 16 weeks or until disease progression.

VAL-083 activity against platinum-resistant ovarian cancer will be measured based on ORR using the Response Evaluation Criteria in Solid Tumors ("RECIST") version 1.1 criteria as well as response duration, progression free survival, and a measurement of CA-125 biomarker levels in the blood. The study’s primary endpoint is to demonstrate an ORR benefit compared to historical control of 12-15%.

Twenty-four patients will be enrolled under the first phase of the VAL-083 REPROVe Trial with top line results expected within 18-24 months from trial initiation of patient treatment. DelMar plans to request a meeting with the FDA following the completion of Phase 1. If successful, DelMar expects that data from Phase 1 will lead to a Phase 2 expansion study. If Phase 2 is successful, and subject to FDA feedback, DelMar may be positioned to file an application for accelerated approval or, alternatively, to advance VAL-083 to a pivotal Phase 3 trial.

"Our planned work in ovarian cancer will be complementary to our ongoing Phase 2 and 3 clinical trials with VAL-083 as a potential treatment for refractory and MGMT-unmethylated glioblastoma multiforme ("GBM"). Based on our research, we believe that the REPROVe Trial has the potential to provide significantly improved outcomes for patients suffering from platinum-resistant ovarian cancer," added Mr. Bacha. Subject to availability of funding, DelMar anticipates opening the VAL-083 REPROVe Trial for patient enrollment in early 2018.

Further details on the VAL-083 REPROVe Trial and other VAL-083 clinical trials can be found at View Source

About VAL-083

VAL-083 (dianhydrogalactitol) is a "first-in-class", DNA-targeting agent that introduces interstrand DNA cross-links at the N7-position of guanine leading to DNA double-strand breaks and cancer cell death. VAL-083 has demonstrated clinical activity against a range of cancers including GBM in historical clinical trials sponsored by the U.S. National Cancer Institutes.

VAL-083 has been granted an orphan drug designation by the U.S. FDA Office of Orphan Products for the treatment of glioma, medulloblastoma and ovarian cancer, and in Europe for the treatment of malignant gliomas. VAL-083 is currently being studied in multiple clinical trials as a potential new treatment for GBM, the most common and aggressive form of brain cancer.

DelMar has demonstrated that VAL-083’s mechanism of action is distinct from multiple chemotherapies widely used in the treatment of cancer and that this unique mechanism may offer opportunities to overcome treatment resistance thereby offering new treatment options to cancer patients. Further details regarding these studies can be found at View Source