OncoCyte Reports Positive Analytical Validation Study Results of DetermaVu™ Lung Cancer Diagnostic Test

On September 18, 2017 OncoCyte Corporation (NYSE American:OCX), a developer of novel, non-invasive blood-based liquid biopsy tests to assist in the early detection of cancer, reported positive final results from the Analytical Validation Study of its liquid biopsy lung cancer diagnostic test, DetermaVU (Press release, BioTime, SEP 18, 2017, View Source [SID1234520552]). The data were presented by Philip McQuary Ph.D., Director of Product Development, OncoCyte Corporation, at the International Association for the Study of Lung Cancer (IASLC), in Chicago.

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The accuracy results of the Analytical Validation Study reported today demonstrate sensitivity of 94.4%, specificity of 67.5% and Area Under the Curve (AUC) of 0.93, which means that 93 percent of the samples tested during the Analytical Validation were correctly diagnosed. These data are consistent with the data reported in May at the American Thoracic Society 2017 International Conference.

Sensitivity and specificity are statistical measures of test performance, with sensitivity measuring the percentage of malignant nodules that are identified correctly by the test and specificity measuring the percentage of benign nodules correctly identified. The AUC of a test is a measure of overall global accuracy that combines sensitivity and specificity, with 1.0 being perfect accuracy and 0.50 being a random result. The score of 0.93 reported at the recent IASLC meeting means that 93 percent of the samples were correctly identified.

The next step in the process leading to commercial launch of DetermaVu is a CLIA Validation study, which is now underway and expected to be completed in the third quarter of this year. If the CLIA Validation study is successful, the final step will be a Clinical Validation study, which is expected to be completed in the fourth quarter of this year. If the Clinical Validation study is successful, OncoCyte plans to launch DetermaVu. OncoCyte believes that at launch DetermaVu will be the only commercially available liquid biopsy lung cancer product in what the Company estimates is an up to $4.7 billion annual market opportunity in the U.S.

"The new data seen in the Analytical Validation Study provide further evidence of the reliability of the DetermaVu assay system in identifying cancerous nodules," stated Lyndal Hesterberg, Ph.D., Senior Vice President, Research and Development. "These data give support our belief that physicians will be able to use DetermaVu with confidence in their clinical practice to help patients make more informed treatment decisions."

William Annett, President and Chief Executive Officer, commented, "We are excited that the results reported at the IASLC conference confirm the positive data reported in May at the American Thoracic Society meeting. If our upcoming Clinical Validation study is successful, we intend to commercialize DetermaVu in the fourth quarter of 2017."

Analytical Validation

The studies required for Analytical Validation have been established in the CLSI (Clinical Lab Standards Institute) Guidelines. These guidelines cover the testing for such matters as limits of quantitation, precision, reproducibility, and interfering substances. OncoCyte has completed all of these studies successfully.

The new Analytical Validation data support expectations that the test’s performance will continue to be robust. The completion of the study establishes the performance characteristics of OncoCyte’s lung cancer diagnostic test and, if the Clinical Validation studies are successful, will allow for industrial-scale operations under real world conditions.

OncoCyte believes that DetermaVu could result in a substantial reduction in the number of unnecessary, expensive lung biopsies performed annually in the U.S., thereby representing a fundamental advancement in the more accurate diagnosis of suspicious lung nodules by allowing physicians to determine which patients need biopsies versus those who may only need follow-up imaging. The Company estimates that approximately 1.4 million patients annually in the U.S. could benefit from the test. Depending on market penetration and reimbursable pricing, this could translate into a market opportunity of up to $4.7 billion annually.

Clinical Validation Stage Underway

The final stage of development following the now completed Analytical Validation Study is Clinical Validation. This stage consists of two distinct sets of studies that will be carried out in OncoCyte’s new CLIA approved clinical laboratory. The first step is CLIA Lab Validation. In this study, OncoCyte will assay approximately 120 samples previously tested in the 299-patient study presented at the ATS meeting, with the goal of demonstrating that OncoCyte’s new clinical laboratory provides the same results on clinical samples as those obtained in OncoCyte’s R&D lab. This study is underway.

Upon successful completion of the CLIA Lab Validation study, the second step will be two CLIA Lab Clinical Validation studies. In these studies, OncoCyte will perform assays on blinded prospectively collected samples to assess the performance of the full diagnostic system against clinically confirmed diagnoses. OncoCyte will perform Clinical Validation on two sets of samples. The first study will consist of approximately 300 samples. If the results of the study are consistent with results to date, OncoCyte will launch DetermaVu.

The second study will be conducted post-launch on approximately 200 additional samples to provide additional data to increase the likelihood that physicians will adopt the test and that insurance companies and Medicare will provide reimbursement coverage for the test.

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."