Novocure Announces 10 Oral Presentations and a Special Session on Tumor Treating Fields at the 41st International Engineering in Medicine and Biology Conference

On July 22, 2019 Novocure (NASDAQ: NVCR) reported 10 oral presentations and a special session on Tumor Treating Fields at the 41st International Engineering in Medicine and Biology (EMB) Conference, July 23 through July 27, in Berlin (Press release, NovoCure, JUL 22, 2019, View Source [SID1234537651]). The volume of Tumor Treating Fields presentations marks a record number of abstracts for Novocure at this conference.

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The conference, hosted by the IEEE Engineering in Medicine and Biology Society (EMBS) will focus on the theme, "Biomedical engineering ranging from wellness to intensive care." During an innovative and interactive special session on Tumor Treating Fields, a neurosurgeon, a radiologist, two biomedical engineers who have worked on novel solutions to treat brain cancer, and a glioblastoma patient will discuss the treatment process, evaluating candidate therapeutic technologies. The oral presentations will describe studies utilizing numerical simulations to better understand Tumor Treating Fields distribution within the body and how Tumor Treating Fields interact with cells. These studies are setting the foundation for optimizing Tumor Treating Fields dosimetry in treatment planning.

"The EMB Conference showcases the latest technologies, innovations and applications to human health and well-being and provides a roadmap for the future," said Dr. Uri Weinberg, Novocure’s Vice President of Clinical Development. "We are pleased that the focus on Tumor Treating Fields continues to expand at this conference. We look forward to participating in this important scientific exchange and exposing the biomedical engineering community to the science of Tumor Treating Fields therapy."

Oral Presentations

(SaA10.1) Advanced Imaging for monitoring response to TTFields in Glioblastoma patients. S. Mohan. 8:30 to 8:45 a.m. CEST on Saturday, July 27.

(SaA10.2) The Dielectric Properties of Brain Tumor Tissue. M. Proescholdt. 8:45 to 9 a.m. CEST on Saturday, July 27.

(SaA10.6) A computational study of Joule heating during TTFields therapy. P. Cavaleiro. 9:45 to 10 a.m. CEST on Saturday, July 27.

(SaA10.3) Determination of parameter values for conductivity, capacitance and inductance of microtubules and a refined model of their bioelectric circuitry elucidate the mode of action of TTFields. J. Tuszynsk. 9 to 9:15 a.m. CEST on Saturday, July 27.

(SaA10.5) A Theory Connecting Mechanisms Underlying 200 kHz AC Electric Fields Effects on Tumor Cell Structures. K. Carlson. 9:30 to 9:45 a.m. CEST on Saturday, July 27.

(SaA10.2) The Dielectric Properties of Brain Tumor Tissue. M. Proescholdt. 8:45 to 9 a.m. CEST on Saturday, July 27.

(FrA09.1) Skull-Remodeling with Tumor Treating Fields. The Role of Finite Element Methods in Surgery Planning and Treatment Evaluation. A. Korshoej. 8:30 to 8:45 a.m. on Friday, July 26.

(WeC10.3) Adapting water-content based electrical properties tomography for the creation of computational head models of brain tumor patients. C. Wenger. 2:30 to 2:45 p.m. on Wednesday, July 24.

(FrA09.3) Development of a Framework for Tumor Treating Fields Dosimetry and Treatment Planning using Computational Phantoms. Z. Bomzon. 9 to 9:15 a.m. on Friday, July 26.

(WeC10.4) A Method for High-Throughput Creation of Patient Specific Head Models. Z. Bomzon. 2:45 to 3 p.m. on Wednesday, July 24.

Special Session

A physician, an engineer and a patient walk into a room: A team-based approach to developing brain cancer treatments

Details: 2 to 3:30 p.m. CEST on Thursday, July 25

Organizers: Punit Prakash, Govindarajan Srimathveeravalli, Ze’ev Bomzon

About IEEE Engineering in Medicine and Biology Society

IEEE Engineering in Medicine and Biology Society (EMBS) is the world’s largest international society of biomedical engineers. The organization’s 11,000 members reside in some 97 countries around the world. EMBS provides its members with access to the people, practices, information, ideas and opinions that are shaping one of the fastest growing fields in science.

IMFINZI® (durvalumab) US Label Updated With Overall Survival Data in Unresectable, Stage III Non-small Cell Lung Cancer

On July 22, 2019 AstraZeneca reported that the US Food and Drug Administration (FDA) has approved the inclusion of overall survival (OS) data from the Phase III PACIFIC trial in an update to the IMFINZI (durvalumab) US Prescribing Information for patients with unresectable, Stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy (CRT) (Press release, AstraZeneca, JUL 22, 2019, View Source [SID1234537652]).

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The label update was based on results from the primary OS analysis published in The New England Journal of Medicine in September 2018 which demonstrated a significant and clinically-proven OS benefit for treatment with IMFINZI vs placebo in this setting, reducing the risk of death by 32% (HR 0.68, 95% CI, 0.53-0.87; P=0.0025).

In a follow-up three-year post-hoc OS analysis presented at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting, results for IMFINZI remained consistent with the two-year data showing a 31% reduction in the risk of death vs placebo (HR 0.69, 95% CI 0.55-0.86) and showing that 57% (95% CI 52.3-61.4) of patients on IMFINZI were still alive vs 44% (95% CI 37.0-49.9) of patients on placebo.

Olivier Nataf, Head of US Oncology, said, "This label update coupled with the recent three-year overall survival data continue to reinforce the PACIFIC regimen as the standard of care in this curative-intent setting. For patients and providers, this announcement provides further evidence and confidence in the survival benefit of IMFINZI."

IMFINZI is approved for the treatment of unresectable, Stage III non-small cell lung cancer in more than 45 countries, including the US, EU and Japan, based on the Phase III PACIFIC trial. Since the first approval in February 2018, over 20,000 patients have been treated with IMFINZI in this setting. The National Comprehensive Cancer Network clinical practice guidelines in oncology (NCCN Guidelines) recommend durvalumab (IMFINZI) as the only Category 1 post-CRT consolidation immunotherapy for unresectable, Stage III NSCLC patients.1,2

IMFINZI can cause serious, potentially fatal adverse events (AEs). In the previous two-year OS analysis, the most common adverse reactions (greater than or equal to 20% of patients) for patients receiving IMFINZI versus placebo were cough (35.2% vs 25.2%), fatigue (24.0% vs 20.5%), dyspnea (22.3% vs 23.9%) and radiation pneumonitis (20.2% vs 15.8%). 30.5% of patients experienced a grade 3 or 4 AE with IMFINZI vs 26.1% with placebo, and 15.4% of patients discontinued treatment due to AEs with IMFINZI vs 9.8% of patients on placebo.

IMPORTANT SAFETY INFORMATION

There are no contraindications for IMFINZI (durvalumab).

IMFINZI can cause serious, potentially fatal adverse reactions including immune-mediated pneumonitis, hepatitis, colitis or diarrhea, endocrinopathies, nephritis, rash or dermatitis, other immune-mediated adverse reactions, infection, and infusion-related reactions. Please refer to the full Prescribing Information for important dosage modification and management information specific to adverse reactions.

Immune-Mediated Pneumonitis

IMFINZI can cause immune-mediated pneumonitis, defined as requiring use of corticosteroids. Fatal cases have been reported. Monitor patients for signs and symptoms of pneumonitis and evaluate with radiographic imaging when suspected. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold IMFINZI for Grade 2 pneumonitis; permanently discontinue for Grade 3 or 4 pneumonitis.

In clinical studies enrolling 1889 patients with various cancers who received IMFINZI, pneumonitis occurred in 5% of patients, including Grade 3 (0.8%), Grade 4 (<0.1%), and Grade 5 (0.3%) pneumonitis. Pneumonitis led to discontinuation of IMFINZI in 1.5% of the 1889 patients. In the PACIFIC study, the incidence of pneumonitis (including radiation pneumonitis) was 34%, including Grade 3 (3.4%) and Grade 5 (1.1%) pneumonitis in the IMFINZI arm. In the PACIFIC study, pneumonitis led to discontinuation of IMFINZI in 6% of patients.

Immune-Mediated Hepatitis

IMFINZI can cause immune-mediated hepatitis, defined as requiring use of corticosteroids. Fatal cases have been reported. Monitor patients for signs and symptoms of hepatitis during and after discontinuation of IMFINZI, including clinical chemistry monitoring. Administer corticosteroids for Grade 2 or higher elevations of ALT, AST, and/or total bilirubin. Withhold IMFINZI for ALT or AST greater than 3 but less than or equal to 8 times the ULN or total bilirubin greater than 1.5 but less than or equal to 5 times the ULN; permanently discontinue IMFINZI for ALT or AST greater than 8 times the ULN or total bilirubin greater than 5 times the ULN or concurrent ALT or AST greater than 3 times the ULN and total bilirubin greater than 2 times the ULN with no other cause.

In clinical studies enrolling 1889 patients with various cancers who received IMFINZI, hepatitis occurred in 12% of patients, including Grade 3 (4.4%), Grade 4 (0.4%), and Grade 5 (0.2%) hepatitis. Hepatitis led to discontinuation of IMFINZI in 0.7% of the 1889 patients.

Immune-Mediated Colitis

IMFINZI can cause immune-mediated colitis, defined as requiring use of corticosteroids. Administer corticosteroids for Grade 2 or greater colitis or diarrhea. Withhold IMFINZI for Grade 2 colitis or diarrhea; permanently discontinue for Grade 3 or 4 colitis or diarrhea.

In clinical studies enrolling 1889 patients with various cancers who received IMFINZI, colitis or diarrhea occurred in 18% of patients, including Grade 3 (1.0%) and Grade 4 (0.1%) colitis. Diarrhea or colitis led to discontinuation of IMFINZI in 0.4% of the 1889 patients.

Immune-Mediated Endocrinopathies

IMFINZI can cause immune-mediated endocrinopathies, including thyroid disorders, adrenal insufficiency, type 1 diabetes mellitus, and hypophysitis/hypopituitarism. Monitor patients for clinical signs and symptoms of endocrinopathies.

Thyroid disorders—Monitor thyroid function prior to and periodically during treatment. Initiate hormone replacement therapy or medical management of hyperthyroidism as clinically indicated. Withhold IMFINZI for Grades 2–4 hyperthyroidism, until clinically stable. Continue IMFINZI for hypothyroidism. In clinical studies enrolling 1889 patients with various cancers who received IMFINZI, hypothyroidism occurred in 11% of patients, while hyperthyroidism occurred in 7% of patients. Thyroiditis occurred in 0.9% of patients, including Grade 3 (<0.1%). Hypothyroidism was preceded by thyroiditis or hyperthyroidism in 25% of patients.
Adrenal insufficiency—Administer corticosteroids as clinically indicated and withhold IMFINZI until clinically stable for Grade 2 or higher adrenal insufficiency. In clinical studies enrolling 1889 patients with various cancers who received IMFINZI, adrenal insufficiency occurred in 0.7% of patients, including Grade 3 (<0.1%) adrenal insufficiency.
Type 1 diabetes mellitus—Initiate treatment with insulin as clinically indicated. Withhold IMFINZI for Grades 2–4 type 1 diabetes mellitus, until clinically stable. In clinical studies enrolling 1889 patients with various cancers who received IMFINZI, type 1 diabetes mellitus occurred in <0.1% of patients.
Hypophysitis—Administer corticosteroids and hormone replacement as clinically indicated and withhold IMFINZI until clinically stable for Grade 2 or higher hypophysitis. Hypopituitarism leading to adrenal insufficiency and diabetes insipidus occurred in <0.1% of 1889 patients with various cancers who received IMFINZI.
Immune-Mediated Nephritis

IMFINZI can cause immune-mediated nephritis, defined as evidence of renal dysfunction requiring use of corticosteroids. Fatal cases have occurred. Monitor patients for abnormal renal function tests prior to and periodically during treatment with IMFINZI. Administer corticosteroids as clinically indicated. Withhold IMFINZI for creatinine greater than 1.5 to 3 times the ULN; permanently discontinue IMFINZI and administer corticosteroids in patients with creatinine greater than 3 times the ULN.

In clinical studies enrolling 1889 patients with various cancers who received IMFINZI, nephritis (reported as any of the following: increased creatinine or urea, acute kidney injury, renal failure, decreased glomerular filtration rate, tubulointerstitial nephritis, decreased creatinine clearance, glomerulonephritis, and nephritis) occurred in 6.3% of the patients including Grade 3 (1.1%), Grade 4 (0.2%), and Grade 5 (0.1%) nephritis. IMFINZI was discontinued in 0.3% of the 1889 patients.

Immune-Mediated Dermatologic Reactions

IMFINZI can cause immune-mediated rash. Bullous dermatitis and Stevens Johnson Syndrome (SJS)/toxic epidermal necrolysis (TEN) have occurred with other products in this class. Administer corticosteroids for Grade 2 rash or dermatitis lasting for more than 1 week or for Grade 3 or 4 rash or dermatitis. Withhold IMFINZI for Grade 2 rash or dermatitis lasting longer than 1 week or Grade 3 rash or dermatitis; permanently discontinue IMFINZI in patients with Grade 4 rash or dermatitis.

In clinical studies enrolling 1889 patients with various cancers who received IMFINZI, 26% of patients developed rash or dermatitis and 0.4% of the patients developed vitiligo. Rash or dermatitis led to discontinuation of IMFINZI in 0.1% of the 1889 patients.

Other Immune-Mediated Adverse Reactions

IMFINZI can cause severe and fatal immune-mediated adverse reactions. These immune-mediated reactions may involve any organ system. While immune-mediated reactions usually manifest during treatment with IMFINZI, immune-mediated adverse reactions can also manifest after discontinuation of IMFINZI. For suspected immune-mediated adverse reactions, exclude other causes and initiate corticosteroids as clinically indicated. Withhold IMFINZI for Grade 3 immune-mediated adverse reactions, unless clinical judgment indicates discontinuation; permanently discontinue IMFINZI for Grade 4 adverse reactions.

The following clinically significant, immune-mediated adverse reactions occurred at an incidence of less than 1% each in 1889 patients who received IMFINZI: aseptic meningitis, hemolytic anemia, immune thrombocytopenic purpura, myocarditis, myositis, and ocular inflammatory toxicity, including uveitis and keratitis. Additional clinically significant immune-mediated adverse reactions have been seen with other products in this class (see Warnings and Precautions Section 5.7 of IMFINZI full Prescribing Information).

Infection

IMFINZI can cause serious infections, including fatal cases. Monitor patients for signs and symptoms of infection and treat as clinically indicated. Withhold IMFINZI for Grade 3 or 4 infection, until clinically stable.

In clinical studies enrolling 1889 patients with various cancers who received IMFINZI, infections occurred in 43% of patients, including Grade 3 (8%), Grade 4 (1.9%), and Grade 5 (1.0%). In patients with Stage III NSCLC in the PACIFIC study, the most common Grade 3 or higher infection was pneumonia, which occurred in 5% of patients.

Infusion-Related Reactions

IMFINZI can cause severe or life-threatening infusion-related reactions. Monitor patients for signs and symptoms of an infusion-related reaction. Interrupt or slow the rate of infusion for Grades 1–2 infusion-related reactions; permanently discontinue for Grades 3–4 infusion-related reactions.

In clinical studies enrolling 1889 patients with various cancers who received IMFINZI, infusion-related reactions occurred in 2.2% of patients, including Grade 3 (0.3%).

Embryo-Fetal Toxicity

Based on its mechanism of action and data from animal studies, IMFINZI can cause fetal harm when administered to a pregnant woman. There are no data on the use of IMFINZI in pregnant women. Advise pregnant women of the potential risk to a fetus and advise women of reproductive potential to use effective contraception during treatment and for at least 3 months after the last dose of IMFINZI.

Lactation

There is no information regarding the presence of IMFINZI in human milk; however, because of the potential for adverse reactions in breastfed infants from IMFINZI, advise women not to breastfeed during treatment and for at least 3 months after the last dose.

Most Common Adverse Reactions

In patients with Stage III NSCLC in the PACIFIC study (IMFINZI n=475), the most common adverse reactions (≥20% of patients) were cough (40%), fatigue (34%), pneumonitis or radiation pneumonitis (34%), upper respiratory tract infections (26%), dyspnea (25%), and rash (23%). The most common Grade 3 or 4 adverse reaction (≥3%) was pneumonia (7%).
In patients with Stage III NSCLC in the PACIFIC study (IMFINZI n=475), discontinuation due to adverse reactions occurred in 15% of patients in the IMFINZI arm. Serious adverse reactions occurred in 29% of patients receiving IMFINZI. The most frequent serious adverse reactions (≥2% of patients) were pneumonitis or radiation pneumonitis (7%) and pneumonia (6%). Fatal pneumonitis or radiation pneumonitis and fatal pneumonia occurred in <2% of patients and were similar across arms.
The safety and effectiveness of IMFINZI have not been established in pediatric patients.

Indication

IMFINZI is indicated for the treatment of patients with unresectable Stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy.

Please see complete Prescribing Information, including Medication Guide.

NOTES TO EDITORS

About PACIFIC

The PACIFIC trial is a Phase III, randomized, double-blinded, placebo-controlled, multi-center trial of IMFINZI (durvalumab) as treatment in "all-comer" patients (ie, regardless of PD-L1 status) with unresectable Stage III NSCLC whose disease has not progressed following platinum-based chemotherapy concurrent with radiation therapy (CRT).

The trial is being conducted in 235 centers across 26 countries involving 713 patients. The primary endpoints of the trial are progression-free survival (PFS) and overall survival (OS), and secondary endpoints include landmark PFS and OS, overall response rate (ORR) and duration of response (DoR).

About Stage III NSCLC

Stage III (locally advanced) non-small cell lung cancer (NSCLC) is commonly divided into three sub-categories (IIIA, IIIB and IIIC), defined by how much the cancer has spread locally and the possibility of surgery. Stage III disease is different from Stage IV disease, when the cancer has spread (metastasized) to distant organs, as Stage III is currently treated with curative intent.

Approximately one in four patients with NSCLC in the United States present with Stage III disease, which is estimated to affect over 43,000 patients. The majority of Stage III NSCLC patients are diagnosed with unresectable tumors. Until recently, the standard of care beyond CRT was active surveillance to monitor for progression as there had been no FDA approved treatments following CRT.

About IMFINZI (durvalumab)

IMFINZI (durvalumab) is a human monoclonal antibody that binds to PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80, countering the tumor’s immune-evading tactics and releasing the inhibition of immune responses.

IMFINZI is approved for unresectable, Stage III NSCLC in more than 45 countries including the US, EU and Japan based on the Phase III PACIFIC trial.

As part of a broad development program, IMFINZI is also being tested as a monotherapy and in combination with tremelimumab, an investigational anti-CTLA4 monoclonal antibody and potential new medicine, as a treatment for patients with NSCLC, small cell lung cancer (SCLC), bladder cancer, head and neck cancer, liver cancer, cervical cancer, biliary tract cancer and other solid tumors.

About AstraZeneca Support Programs

AstraZeneca strives to ensure that appropriate patients and their oncologists have access to IMFINZI and relevant support resources. These include educational resources, an Oncology Nurse Educator program and affordability and reimbursement programs, such as Access 360.

Additionally, AstraZeneca has launched Lighthouse, a program that provides support to patients during any immune-mediated adverse events they may encounter during treatment, through medically trained Lighthouse Advocates. The program aims to make patients’ treatment experience as comfortable as possible. Find out more about Lighthouse at LighthouseProgram.com or call 1-855-LHOUSE1 (1-855-546-8731).

About AstraZeneca in Lung Cancer

AstraZeneca has a comprehensive portfolio of approved and investigational medicines in late-stage clinical development for the treatment of different forms of lung cancer spanning several stages of disease and lines of therapy. We aim to address the unmet needs of patients with EGFR-mutated tumors as a genetic driver of disease, which occur in approximately 7-23% of patients in Western populations, and 30-40% of Asian patients, with our approved medicines osimertinib and ongoing Phase III trials FLAURA, ADAURA and LAURA, as well as the Phase II exploratory combination trials SAVANNAH and ORCHARD.

Our extensive late-stage Immuno-Oncology program focuses on 75-80% of patients with lung cancer without a known genetic mutation. IMFINZI, an anti-PD-L1 antibody, is in development as monotherapy (Phase III trials ADJUVANT BR.31, PACIFIC-4, PACIFIC-5 and PEARL) and in combination with tremelimumab and/or chemotherapy (AEGEAN, PACIFIC-2, NEPTUNE, POSEIDON, ADRIATIC and CASPIAN Phase III trials).

About AstraZeneca’s Approach to Immuno-Oncology (IO)

Immuno-Oncology (IO) is a therapeutic approach designed to stimulate the body’s immune system to attack tumors. At AstraZeneca, our IO portfolio is anchored by immunotherapies that have been designed to overcome anti-tumor immune suppression. We believe that IO-based therapies will offer the potential for life-changing cancer treatments for the clear majority of patients.

We are pursuing a comprehensive clinical trial program that includes IMFINZI (anti-PD-L1) as monotherapy and in combination with tremelimumab (anti-CTLA-4) in multiple tumor types, stages of disease, and lines of therapy. In addition, the ability to combine our IO portfolio with small, targeted molecules from across our Oncology pipeline, and from our research partners, may provide new treatment options across a broad range of tumors.

About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance Oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy as illustrated by our investment in Acerta Pharma in hematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumor Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalized combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

CStone receives approval in China to initiate ivosidenib Phase I bridging registrational study for the treatment of IDH1 mutant relapsed or refractory AML

On July 22, 2019 CStone Pharmaceuticals ("CStone"; HKEX: 2616) reported that it has received approval from China National Medical Products Administration (NMPA) to initiate a Phase I bridging registrational study of ivosidenib (TIBSOVO) for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML) with an IDH1 mutation (Press release, CStone Pharmaceauticals, JUL 22, 2019, View Source [SID1234537637]). This stand-alone trial is designed to evaluate the efficacy, safety and pharmacokinetics of ivosidenib in Chinese patients with IDH1 mutant R/R AML.

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AML is the most common type of acute leukemia in adults and is characterized by rapid disease progression. There are approximately 20,000 new cases of AML in the U.S. each year, with a five-year survival rate of approximately 27%, as compared to 30,000 new cases in China annually and a five-year survival rate of below 20%. The majority of AML patients develop tolerance to treatments or eventually relapse, leading to R/R AML which has a poor prognosis. With an increasing life expectancy and aging population in China, the incidence of AML may rise significantly in the country. Interruption to the differentiation of hematopoietic stem cells due to IDH1 mutation is associated with around 6%-10% of all AML cases. Among the currently approved treatments for AML in China, there is no effective therapy for this patient population.

Discovered and developed by CStone’s partner Agios Pharmaceuticals, ivosidenib was approved by the U.S. FDA in July 2018 for the treatment of adult R/R AML patients with an IDH1 mutation as detected by an FDA-approved test.

"Ivosidenib is the first and only approved precision therapy in the U.S. that targets IDH1 mutation in R/R AML and the only asset in CStone’s pipeline that has obtained a marketing authorization. This approval for the registrational study in China marks a big milestone for us," commented Dr. Frank Jiang, Chairman and CEO of CStone. "Through precision therapies, patients can potentially benefit from treatments targeting the specific genetic mutations that drive the cancer. CStone remains committed to advancing precision therapies and bringing more targeted treatment options to patients."

"Ivosidenib was first approved as a treatment for IDH1 mutant R/R AML. The U.S. FDA recently approved a supplemental NDA for ivosidenib to include newly diagnosed AML patients with an IDH1 mutation who are ≥ 75 years old or have comorbidities that preclude the use of intensive induction chemotherapy. In March 2019, the U.S. FDA granted Breakthrough Therapy designation for ivosidenib in combination with azacitidine for the treatment of the same newly diagnosed AML patients. These milestones demonstrate ivosidenib’s promising clinical utility. We are poised to rapidly roll out this trial for ivosidenib as a monotherapy for R/R AML in China. We hope the study will generate favorable clinical data, paving the way for the drug to quickly enter the China market," noted Dr. Jason Yang, CStone’s Chief Medical Officer.

About TIBSOVO (ivosidenib)

In the United States, TIBSOVO is indicated for the treatment of acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in:

Adult patients with newly-diagnosed AML who are ≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
Adult patients with relapsed or refractory AML.
IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Patients treated with TIBSOVO have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome: See Boxed WARNING. In the clinical trial, 25% (7/28) of patients with newly diagnosed AML and 19% (34/179) of patients with relapsed or refractory AML treated with TIBSOVO experienced differentiation syndrome. Differentiation syndrome is associated with rapid proliferation and differentiation of myeloid cells and may be life-threatening or fatal if not treated. Symptoms of differentiation syndrome in patients treated with TIBSOVO included noninfectious leukocytosis, peripheral edema, pyrexia, dyspnea, pleural effusion, hypotension, hypoxia, pulmonary edema, pneumonitis, pericardial effusion, rash, fluid overload, tumor lysis syndrome, and creatinine increased. Of the 7 patients with newly diagnosed AML who experienced differentiation syndrome, 6 (86%) patients recovered. Of the 34 patients with relapsed or refractory AML who experienced differentiation syndrome, 27 (79%) patients recovered after treatment or after dose interruption of TIBSOVO. Differentiation syndrome occurred as early as 1 day and up to 3 months after TIBSOVO initiation and has been observed with or without concomitant leukocytosis.

If differentiation syndrome is suspected, initiate dexamethasone 10 mg IV every 12 hours (or an equivalent dose of an alternative oral or IV corticosteroid) and hemodynamic monitoring until improvement. If concomitant noninfectious leukocytosis is observed, initiate treatment with hydroxyurea or leukapheresis, as clinically indicated. Taper corticosteroids and hydroxyurea after resolution of symptoms and administer corticosteroids for a minimum of 3 days. Symptoms of differentiation syndrome may recur with premature discontinuation of corticosteroid and/or hydroxyurea treatment. If severe signs and/or symptoms persist for more than 48 hours after initiation of corticosteroids, interrupt TIBSOVO until signs and symptoms are no longer severe.

QTc Interval Prolongation: Patients treated with TIBSOVO can develop QT (QTc) prolongation and ventricular arrhythmias. One patient developed ventricular fibrillation attributed to TIBSOVO. Concomitant use of TIBSOVO with drugs known to prolong the QTc interval (e.g., anti-arrhythmic medicines, fluoroquinolones, triazole anti-fungals, 5-HT3 receptor antagonists) and CYP3A4 inhibitors may increase the risk of QTc interval prolongation. Conduct monitoring of electrocardiograms (ECGs) and electrolytes. In patients with congenital long QTc syndrome, congestive heart failure, or electrolyte abnormalities, or in those who are taking medications known to prolong the QTc interval, more frequent monitoring may be necessary.

Interrupt TIBSOVO if QTc increases to greater than 480 msec and less than 500 msec. Interrupt and reduce TIBSOVO if QTc increases to greater than 500 msec. Permanently discontinue TIBSOVO in patients who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia.

Guillain-Barré Syndrome: Guillain-Barré syndrome occurred in <1% (2/258) of patients treated with TIBSOVO in the clinical study. Monitor patients taking TIBSOVO for onset of new signs or symptoms of motor and/or sensory neuropathy such as unilateral or bilateral weakness, sensory alterations, paresthesias, or difficulty breathing. Permanently discontinue TIBSOVO in patients who are diagnosed with Guillain-Barré syndrome.

ADVERSE REACTIONS

The most common adverse reactions including laboratory abnormalities (≥20%) were hemoglobin decreased (60%), fatigue (43%), arthralgia (39%), calcium decreased (39%), sodium decreased (39%), leukocytosis (38%), diarrhea (37%), magnesium decreased (36%), edema (34%), nausea (33%), dyspnea (32%), uric acid increased (32%), potassium decreased (32%), alkaline phosphatase increased (30%), mucositis (28%), aspartate aminotransferase increased (27%), phosphatase decreased (25%), electrocardiogram QT prolonged (24%), rash (24%), creatinine increased (24%), cough (23%), decreased appetite (22%), myalgia (21%), constipation (20%), and pyrexia (20%).
In patients with newly diagnosed AML, the most frequently reported Grade ≥3 adverse reactions (≥5%) were fatigue (14%), differentiation syndrome (11%), electrocardiogram QT prolonged (11%), diarrhea (7%), nausea (7%), and leukocytosis (7%). Serious adverse reactions (≥5%) were differentiation syndrome (18%), electrocardiogram QT prolonged (7%), and fatigue (7%). There was one case of posterior reversible encephalopathy syndrome (PRES).
In patients with relapsed or refractory AML, the most frequently reported Grade ≥3 adverse reactions (≥5%) were differentiation syndrome (13%), electrocardiogram QT prolonged (10%), dyspnea (9%), leukocytosis (8%), and tumor lysis syndrome (6%). Serious adverse reactions (≥5%) were differentiation syndrome (10%), leukocytosis (10%), and electrocardiogram QT prolonged (7%). There was one case of progressive multifocal leukoencephalopathy (PML).
DRUG INTERACTIONS

Strong or Moderate CYP3A4 Inhibitors: Reduce TIBSOVO dose with strong CYP3A4 inhibitors. Monitor patients for increased risk of QTc interval prolongation.

Strong CYP3A4 Inducers: Avoid concomitant use with TIBSOVO.

Sensitive CYP3A4 Substrates: Avoid concomitant use with TIBSOVO.

QTc Prolonging Drugs: Avoid concomitant use with TIBSOVO. If co-administration is unavoidable, monitor patients for increased risk of QTc interval prolongation.

LACTATION

Because many drugs are excreted in human milk and because of the potential for adverse reactions in breastfed children, advise women not to breastfeed during treatment with TIBSOVO and for at least 1 month after the last dose.

Vanda Pharmaceuticals to Announce Second Quarter 2019 Financial Results on July 31, 2019

On July 22, 2019 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported it will release results for the second quarter 2019 on Wednesday, July 31, 2019, after the market closes (Press release, Vanda Pharmaceuticals, JUL 22, 2019, View Source [SID1234537654]).

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Vanda will host a conference call at 4:30 PM ET on Wednesday, July 31, 2019, during which management will discuss the second quarter 2019 financial results and other corporate activities. To participate in the conference call, please dial 1-866-688-9426 (domestic) or 1-409-216-0816 (international) and use passcode 2589983.

The conference call will be broadcast simultaneously and archived on Vanda’s website, www.vandapharma.com. Investors should go to the website at least 15 minutes early to register, download, and install any necessary audio software.

A replay of the call will be available on Wednesday, July 31, 2019, beginning at 7:30 PM ET and will be accessible until Wednesday, August 7, 2019, at 7:30 PM ET. The replay call-in number is 1-855-859-2056 for domestic callers and 1-404-537-3406 for international callers. The passcode number is 2589983.

Kazia to test GDC-0084 with Radiotherapy in Phase I Clinical Trial at Leading US Cancer Center

On July 22, 2019 Kazia Therapeutics Limited (ASX: KZA;NASDAQ: KZIA), an Australian oncology-focused biotechnology company, is reported that Memorial Sloan Kettering Cancer Center (MSK) in New York, NY will investigate the potential use of Kazia’s investigational new drug, GDC-0084, in combination with radiotherapy in a phase I clinical trial for cancer that has spread to the brain (brain metastases and leptomeningeal metastases) (Press release, Kazia Therapeutics, JUL 22, 2019, View Source [SID1234537655]). This research will explore a new use of GDC-0084 and will run concurrently with other ongoing studies in different forms of brain cancer.

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Dr James Garner, Chief Executive Officer of Kazia Therapeutics commented, "MSK is one of the world’s leading cancer treatment centers, and we are privileged to be supporting them in this state-of-the-art project. Many cancers have the potential to spread to the brain, and they become very difficult to treat when they do. The work being done at MSK will investigate whether GDC-0084 has the potential to enhance the effects of radiotherapy, which remains the current standard of care in most cases."

Key Points

MSK will initiate a phase I clinical trial of GDC-0084 in combination with radiotherapy for patients with solid tumor brain metastases (cancer that has spread to the brain) and leptomeningeal metastases that harbors a genetic alteration in the PI3K pathway.
The trial is expected to recruit 18-30 patients and will take about two years to complete.
The trial will be led by MSK, with Kazia providing support including study drug and a financial grant.
Initiation of this study brings to five the total number of ongoing clinical trials with GDC-0084, each in different forms of brain cancer.
Up to 30% of patients with metastatic cancer will develop secondary tumors (metastases) in the brain. Radiotherapy remains the standard of care, but 30-50% of patients will progress within one year, despite best available treatment. In animal models of certain cancers, activation of the PI3K pathway has been shown to contribute to radiotherapy resistance. GDC-0084 is a PI3K inhibitor that can cross the blood-brain barrier, and as such it may be able to reduce the problem of resistance to radiotherapy. This clinical trial has been developed to test that hypothesis.

The trial is expected to recruit 18-30 patients, all of whom will have cancer that has spread to the brain. Patients will be genetically tested for a specific alteration in the PI3K pathway, and only those with a relevant mutation will be enrolled. This is an example of an approach to clinical research that is sometimes referred to as ‘precision medicine’ or ‘personalized medicine’, in which treatments are carefully targeted to those patients most likely to benefit. It is expected that the trial will begin recruitment in the second half of calendar 2019.

The study in two parts. The first part will aim to determine the maximum tolerated dose (MTD) of GDC-0084 when given together with radiotherapy. Once that dose has been determined, the second part of the study will enroll an additional twelve patients at that dose to explore preliminary signals of efficacy.

The Principal Investigator for the study is Dr T Jonathan Yang, Director of Metastatic Disease Program in MSK’s Department of Radiation Oncology. Dr Yang is a graduate of Yale University School of Medicine and a Board-certified radiation oncologist, with a specialist interest in treating tumors of the central nervous system. He is an extensively-published clinical researcher who has participated in a substantial number of clinical trials in brain cancer.

The initiation of the study brings to five the number of ongoing clinical trials with GDC-0084:

Sponsor

Phase

Indication

Registration

Kazia Therapeutics

II

Glioblastoma

NCT03522298

Alliance for Clinical Trials in Oncology

II

Brain metastases

NCT03994796

Dana-Farber Cancer Institute

II

Breast cancer brain metastases

(with Herceptin)

NCT03765983

St Jude Children’s Research Hospital

I

DIPG (childhood brain cancer)

NCT03696355

Memorial Sloan Kettering Cancer Center

I

Brain metastases

(with radiotherapy)

(TBA)

It is expected that the study will take approximately two years to complete. Kazia will provide support, including a financial grant for a portion of the costs. The study will be conducted under an ‘investigator IND’ with the US FDA, in which the primary regulatory responsibilities for the study will be assumed by MSK. Implementation of the study is conditional upon approval from the Institutional Review Board at MSK, and this approval has yet to be obtained.