Anixa Biosciences Announces Collaboration with Urology Centers of Alabama on Cchek™ Prostate Cancer Study

On November 22, 2019 Anixa Biosciences, Inc. (NASDAQ: ANIX), a biotechnology company focused on harnessing the body’s immune system in the fight against cancer, reported a collaboration with Urology Centers of Alabama (UCA) in the Cchek early prostate cancer detection study (Press release, Anixa Biosciences, NOV 22, 2019, View Source [SID1234551607]). UCA will provide patient samples in Anixa’s ongoing study, including support of clinical validation of Anixa’s Cchek Prostate Cancer Confirmation test.

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UCA is one of several sites across the U.S. participating in this study. Dr. Charles Edward Bugg, Jr. will serve as the Principal Investigator at UCA.

Amit Kumar, Ph.D., Chief Executive Officer of Anixa, stated, "We are pleased to have another highly regarded urology practice join our team of collaboration partners. We look forward to UCA’s participation as we continue the clinical validation phase of the Cchek Prostate Cancer Confirmation test in preparation for commercial launch later this year."

About Cchek
Cchek is an early cancer detection technology, that measures a patient’s immunological response to a malignancy by analyzing immune system cells in peripheral blood. The goal is to utilize the technology to determine a patient’s cancer status from a simple blood draw, eliminating the need for a biopsy, which can be an expensive, painful and invasive procedure. Further, conventional methods using current cancer screening tests often lack accuracy and reliability. Anixa’s orthogonal approach using flow cytometry coupled with artificial intelligence provides an alternative method that offers improved affordability, efficacy and efficiency. To date, Anixa has successfully used Cchek to detect the presence of 20 different cancers including lung, colon, breast and prostate. The robust cancer detection performance of Cchek makes it a platform from which multiple cancer diagnostic tests may be developed. The first such test, a prostate cancer confirmation test, is slated for commercial launch by the end of 2019.

Urology Centers of Alabama
For over 60 years, Urology Centers of Alabama has been committed to providing the highest quality medical services in a compassionate and caring environment. With 24 skilled urologists, two urogynecologists, two radiation oncologist, one medical oncologist, one pathologist, a team of physicians’ assistants and nurse practitioners and over 160 employees, the practice has earned a well deserved reputation for excellence.

City of Hope to lead national research aimed at improving testing/treatment for devastating cancer-causing syndrome

On November 22, 2019 City of Hope reported that persons who inherit Li-Fraumeni syndrome (LFS) have an extremely increased risk of developing cancer (Press release, City of Hope, NOV 22, 2019, View Source [SID1234551623]). For instance, females with the syndrome have a 50% chance of developing cancer by the age of 30 and a 90% chance over their lifetime; males with the syndrome have a 70% chance of cancer over their lifetime.

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LFS was first described 50 years ago, and 30 years ago the syndrome was linked to inheriting a mutation in the TP53 gene. City of Hope scientists and others worldwide have made advances in identifying and treating people with the syndrome, but more work remains to be done.

A new $8.5 million, five-year grant from the National Institutes of Health seeks to advance research and treatment for this serious syndrome, which can cause multiple cancers, including sarcomas, brain and breast tumors, and adrenocortical cancers.

"This is a devastating syndrome to have – we’ve cared for families with babies who had brain tumors at 18 months," said Jeffrey Weitzel, M.D., director of City of Hope’s Division of Clinical Cancer Genomics, the Dr. Norman & Melinda Payson Professor in Medical Oncology and the grant’s co-principal investigator. "City of Hope and our partner institutions are determined to develop a better understanding of the cancer risks associated with different TP53 mutations so we can better tailor screening, prevention and treatment for these patients. We are seeing the light at the end of the tunnel but more research is needed."

The project, termed LiFTUP (Li Fraumeni and TP53 Understanding and Progress), which is co-led by Judy E. Garber, M.D., M.P.H., of Dana-Farber Cancer Institute and Christopher I. Amos, Ph.D., of Baylor College of Medicine, will provide the largest and most comprehensive examination of TP53-associated cancer risk.

Traditionally, clinical TP53 testing was limited to individuals and families who met specific criteria (typically multiple cancers at very young ages). With the introduction of next-generation-based multigene panel testing, which sequences a person’s genome faster and is less costly than previous genetic tests, TP53 testing is now performed on large numbers of people who do not meet the syndrome’s criteria. However, the testing has also raised concerns about false positives, or a test result that incorrectly indicates the syndrome is present.

That’s because blood samples are often used for genetic testing and people may develop TP53 gene mutations in rapidly growing blood cells as they age. Researchers will also examine this group of people to find out why some people in this group develop blood cancers while others do not.

The inherited syndrome is rare. Only about 1,000 children and adults nationwide are included in the Li-Fraumeni Exploration Consortium, and researchers working on this project will also recruit TP53 carriers identified through broader, more agnostic approaches to testing, including commercial genetic testing laboratories, the Geisinger MyCode project, the PROMPT study of individuals with germline mutations and the ORIEN tumor/germline sequencing project.

"Carriers of true germline TP53 mutations may bear the psychological, medical and financial costs of striking personal and family cancer risks, the burden of intensive surveillance, the high risks of cancer deaths at disproportionately young ages and the weight of possibly passing TP53 variants to offspring," according to the team.

Consequently, a crucial question researchers want to answer is why some people with a TP53 mutation develop cancer while others do not.

"We’re looking at thousands of markers across the whole genome to find out if there are patterns that influence why two TP53 carriers with the same mutation have different outcomes," Weitzel added. "We will do a clinical and molecular interrogation of the factors that influence the development of cancer."

"Our hope is that by focusing more research on children and adults with the TP53 mutation, we will be able to take better care of these individuals," Weitzel said.

Agilent Companion Diagnostic Expands CE-IVD mark in Europe to include Head and Neck Squamous Cell Carcinoma (HNSCC)

On November 22, 2019 Agilent Technologies, Inc. (NYSE: A) reported that its PD-L1 IHC 22C3 pharmDx assay is now labeled for use in HNSCC in the European Union (Press release, Agilent, NOV 22, 2019, https://www.agilent.com/about/newsroom/presrel/2019/22nov-ca19033.html [SID1234551608]).

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The assay is CE-IVD–marked as an aid in identifying HNSCC patients for treatment with KEYTRUDA, an anti-PD-1 therapy manufactured by Merck (known as MSD outside the United States and Canada). KEYTRUDA, as monotherapy or in combination with platinum and 5-fluorouracil (5-FU) chemotherapy, is indicated for the first-line treatment of metastatic or unresectable recurrent HNSCC in adults whose tumors express PD-L1 with a CPS ≥ 1.

PD-L1 IHC 22C3 pharmDx is the only clinical trial-proven PD-L1 assay CE-IVD–marked as an aid in identifying HNSCC patients for treatment with KEYTRUDA. PD-L1 IHC 22C3 pharmDx is now CE-IVD–marked as a companion diagnostic to KEYTRUDA in three tumor indications – NSCLC, urothelial carcinoma, and HNSCC. In the United States, the FDA approved PD-L1 IHC 22C3 pharmDx in a HNSCC indication in June.

"PD-L1 is an essential biomarker for anti-PD-1 therapies like KEYTRUDA," said Sam Raha, president of Agilent’s Diagnostics and Genomics Group. "The expanded indication of PD-L1 IHC 22C3 pharmDx will give physicians in Europe critical information to inform first-line treatment decisions for patients with metastatic or unresectable recurrent HNSCC and provide the diagnostic confidence of a clinically relevant test."

HNSCC is the seventh most common cancer worldwide3 and accounts for more than 90% of head and neck cancer cases.4 In Europe, approximately 170,000 new head and neck cancer cases are diagnosed annually.5 Worldwide, the five-year overall survival rate for HNSCC is approximately 40–50%.6

KEYTRUDA is a humanized monoclonal antibody that increases the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes, which may affect both tumor cells and healthy cells. KEYTRUDA and other targeted immunotherapies are revolutionizing cancer treatment, and their therapeutic value is being demonstrated across a growing list of cancer types.

Agilent is a worldwide leader in partnering with pharmaceutical companies to develop immunohistochemical-based diagnostics for cancer therapy. Agilent developed PD-L1 IHC 22C3 pharmDx in collaboration with Merck. PD-L1 IHC 22C3 pharmDx also helps physicians identify non-small cell lung cancer (NSCLC) and urothelial carcinoma patients for treatment with KEYTRUDA. PD-L1 expression in NSCLC tissues is interpreted using Tumor Proportion Score (TPS). PD-L1 expression in urothelial carcinoma tissues is interpreted using Combined Positive Score (CPS). PD-L1 expression in HNSCC tissues can be interpreted using CPS and/or TPS.

References:
1. PD-L1 IHC 22C3 pharmDx [package insert]. Carpinteria, CA: Dako, Agilent Pathology Solutions; 2019.
2. Keytruda [Summary of Product Characteristics]. European Medicines Agency; 2019.
3. Seiwert, T.Y.; Burtness, B.; Mehra, R.; Weiss, J.; Berger, R.; Eder, J.P.; Heath, K.; McClanahan, T.; Lunceford, J.; Gause, C.; Cheng, J.D.; Chow, L.Q. Safety and Clinical Activity of Pembrolizumab for Treatment of Recurrent or Metastatic Squamous Cell Carcinoma of The Head and Neck (KEYNOTE-012): An Open-label, Multicenter, Phase 1b Trial. Lancet Oncol. 2016, 17 (7), 956–965.
4. Gupta, B.; Johnson, N.W.; Kumar, N. Global Epidemiology of Head and Neck Cancers: A Continuing Challenge. Oncology. 2016, 91, 13–23.
5. World Health Organization. Global Cancer Observatory. View Source (accessed October 9, 2019).
6. World Health Organization. 2014 Review of Cancer Medicines on the WHO List of Essential Medicines: Locally Advanced Squamous Carcinoma of the Head and Neck. View Source
expert/20/applications/HeadNeck.pdf. Published 2014 (accessed October 9, 2019).

Takeda Presents Long-Term Data in ALK+ NSCLC Showing ALUNBRIG® (brigatinib) Continues to Demonstrate Superiority in the First-Line After Two Years of Follow-Up

On November 22, 2019 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) reported updated data from the Phase 3 ALTA-1L trial, which evaluated ALUNBRIG versus crizotinib in adults with advanced anaplastic lymphoma kinase-positive (ALK+) non-small cell lung cancer (NSCLC) who had not received a prior ALK inhibitor (Press release, Takeda, NOV 22, 2019, View Source [SID1234551624]). Results show after more than two years of follow-up, ALUNBRIG reduced the risk of disease progression or death by 76% (hazard ratio [HR] = 0.24, 95% CI: 0.12–0.45) as assessed by investigators in newly diagnosed patients whose disease had spread to the brain at time of enrollment. ALUNBRIG also demonstrated a 57% (HR = 0.43, 95% CI: 0.31–0.61) reduction in risk of disease progression or death in all patients. These data will be presented during the Presidential Session at the 2019 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Asia Congress on Saturday, November 23 in Singapore.

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Results from the ALTA-1L trial were evaluated by two separate review bodies – study investigators and a blinded independent review committee (BIRC) – and results from both assessments were reported. At the data cutoff for the second interim analysis (June 28, 2019), the BIRC-assessed HR of progression-free survival (PFS), which is the primary endpoint, was 0.49 (95% CI: 0.35–0.68, log-rank P<0.0001), demonstrating a reduced risk of disease progression or death by 51%.

"Given the complexity of this disease and the expected longevity of the population, it is important for physicians to have multiple well-tolerated and durable treatment options to address the needs of their patients," said D. Ross Camidge, M.D., Ph.D., Joyce Zeff Chair in Lung Cancer Research at the University of Colorado Cancer Center and the lead investigator of ALTA-1L. "With 25 months of follow up from the ALTA-1L trial, brigatinib continues to demonstrate overall and intracranial effectiveness, while also significantly improving quality of life compared to crizotinib, reinforcing its potential as a first-line therapy for ALK+ NSCLC."

Additional data from the long-term analysis showed that newly diagnosed patients treated with ALUNBRIG benefited regardless of the presence or absence of brain metastases at baseline, which is one of the most common sites of first progression and associated with poor quality of life.

ALUNBRIG demonstrated high and durable responses in the brain, with patients with baseline brain metastases having superior efficacy compared to crizotinib, as assessed by a BIRC, and an early separation of the PFS curves in these patients was observed.
ALUNBRIG reduced the risk of intracranial disease progression or death by 69% in patients with brain metastases at baseline (HR = 0.31, 95% CI: 0.17–0.56), with a median intracranial PFS of 24 months compared to 5.6 months with crizotinib. Median PFS for patients with brain metastases at baseline was not reached with ALUNBRIG and was 5.9 months with crizotinib, as assessed by investigators.
Confirmed intracranial objective response rate (ORR) for patients with measurable brain metastases at baseline was 78% (95% CI: 52.4–93.6) for patients treated with ALUNBRIG and 26% (95% CI: 10.2–48.4) for patients treated with crizotinib.
Median intracranial duration of response (DOR) in confirmed responders with measurable brain metastases at baseline was not reached (95% CI: 5.7–NE) with ALUNBRIG and was 9.2 months (95% CI: 3.9–9.2) with crizotinib.
ALUNBRIG demonstrated consistent overall efficacy (intent to treat population) with a longer follow-up of 25 months.
Median PFS with ALUNBRIG was 29.4 months (95% CI: 21.2–NE) versus 9.2 months (95% CI: 7.4–12.9) with crizotinib, as assessed by investigators. The BIRC-assessed median PFS was 24.0 months (95% CI: 18.5–NE) for ALUNBRIG and 11.0 months (95% CI: 9.2–12.9) for crizotinib.
Confirmed ORR was 74% (95% CI: 65.5–80.9) for ALUNBRIG and 62% (95% CI: 52.9–69.7) for crizotinib as assessed by a BIRC.
Median DOR was not reached (95% CI: 19.4–NE) with ALUNBRIG and was 13.8 months (95% CI: 9.3–20.8) with crizotinib as assessed by a BIRC.
Quality of life (QoL) for newly diagnosed ALK+ NSCLC patients was also evaluated, with results showing patients treated with ALUNBRIG experienced significant improvements in health-related QoL (HRQoL).
ALUNBRIG delayed median time to worsening in Global Health Score (GHS)/QoL score (≥10 point worsening in score) by 27 months versus 8 months with crizotinib.
Patients treated with ALUNBRIG had longer duration of improvement in GHS/ QoL, with duration of improvement not yet reached versus 12 months with crizotinib.
ALUNBRIG also delayed time to worsening and prolonged duration of improvement in multiple subscales such as fatigue, nausea and vomiting, appetite loss, and emotional and social functioning.
"At Takeda, we are committed to developing products that seek to advance the lung cancer treatment landscape and address the unmet needs of patients," said Phil Rowlands, Head, Oncology Therapeutic Area Unit. "We are proud of the progress made thus far, including these updated results from the ALTA-1L trial, which show that ALUNBRIG delayed disease progression by more than two years and significantly reduced the risk of disease progression in patients with baseline brain metastasis. We look forward to submitting these data to regulatory authorities around the globe with the goal of making ALUNBRIG available to ALK+ NSCLC patients worldwide."

"Individual treatment needs for patients with ALK+ NSCLC are diverse because cancer is not a one-size-fits-all disease," said Bonnie Addario, Co-Founder, Board Chair, GO2 Foundation for Lung Cancer. "Ongoing research and clinical trials such as ALTA-1L are critical to achieving our goal of improving outcomes and quality of life for patients early on in their treatment journey. We’re grateful for the patients, families and investigators who participated in this clinical trial, which shows meaningful results for those with newly diagnosed ALK+ NSCLC."

The safety profile of ALUNBRIG in the ALTA-1L trial was generally consistent with the existing U.S. prescribing information.

Most common treatment-emergent adverse events (TEAEs) Grade ≥3 in the ALUNBRIG arm were increased CPK (24.3%), increased lipase (14.0%) and hypertension (11.8%); and for crizotinib were increased ALT (10.2%), AST (6.6%), and lipase (6.6%).
The frequency of early pulmonary events (interstitial lung disease/pneumonitis) in the ALTA-1L trial was slightly lower compared with the ALTA study in a post-crizotinib population.
Pulmonary events at any time occurred in 5.1% of patients in the ALUNBRIG arm and 2.2% in the crizotinib arm.
Discontinuations due to AEs occurred in 12.5% of patients in the ALUNBRIG arm and 8.8% in the crizotinib arm.
ALUNBRIG is not currently approved for use in the first-line.
About the ALTA-1L Trial
The Phase 3 ALTA-1L (ALK in Lung Cancer Trial of BrigAtinib in 1st Line) trial of ALUNBRIG in adults is a global, ongoing, randomized, open-label, comparative, multicenter trial, which enrolled 275 patients (ALUNBRIG, n=137, crizotinib, n=138) with anaplastic lymphoma kinase-positive (ALK+) locally advanced or metastatic non-small cell lung cancer (NSCLC) who have not received prior treatment with an ALK inhibitor. Patients received either ALUNBRIG, 180 mg once daily with seven-day lead-in at 90 mg once daily, or crizotinib, 250 mg twice daily.

The median age was 58 years in the ALUNBRIG arm and 60 years in the crizotinib arm. Twenty-nine percent of patients had brain metastases at baseline in the ALUNBRIG arm versus 30% in the crizotinib arm. Twenty-six percent of patients received prior chemotherapy for advanced or metastatic disease in the ALUNBRIG arm versus 27% in the crizotinib arm.

Blinded independent review committee (BIRC)-assessed progression-free survival (PFS) was the primary endpoint. Secondary endpoints included objective response rate (ORR) per RECIST v1.1, intracranial ORR, intracranial PFS, overall survival (OS), safety and tolerability.

About ALUNBRIG (brigatinib)
ALUNBRIG is a potent and selective next-generation tyrosine kinase inhibitor (TKI) that was designed to target and inhibit the anaplastic lymphoma kinase (ALK) fusion protein in non-small cell lung cancer (NSCLC). In April 2017, ALUNBRIG received Accelerated Approval from the U.S. Food and Drug Administration (FDA) for anaplastic lymphoma kinase-positive (ALK+) metastatic NSCLC patients who have progressed on or are intolerant to crizotinib. This indication is approved under Accelerated Approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

ALUNBRIG is currently approved in more than 40 countries, including the U.S., Canada and the European Union, for the treatment of people with ALK+ metastatic NSCLC whose disease has worsened during crizotinib treatment or they could not tolerate taking crizotinib.

ALUNBRIG received Breakthrough Therapy Designation from the FDA for the treatment of patients with ALK+ NSCLC whose tumors are resistant to crizotinib and was granted Orphan Drug Designation by the FDA for the treatment of ALK+ NSCLC, ROS1+ and EGFR+ NSCLC.

Takeda in Lung Cancer
Takeda is dedicated to expanding experience in the ALK+ NSCLC and EGFR exon 20 treatment landscapes. Our comprehensive programs include the following clinical trials to continue to address unmet needs for people living with lung cancer:

ALUNBRIG

Phase 1/2 trial, which was designed to evaluate the safety, tolerability, pharmacokinetics and preliminary anti-tumor activity of ALUNBRIG.
Pivotal Phase 2 ALTA trial investigating the efficacy and safety of ALUNBRIG at two dosing regimens in patients with ALK+ locally advanced or metastatic NSCLC who had progressed on crizotinib.
Phase 3 ALTA-1L, global, randomized trial assessing the efficacy and safety of ALUNBRIG in comparison to crizotinib in patients with ALK+ locally advanced or metastatic NSCLC who have not received prior treatment with an ALK inhibitor.
Phase 2 J-ALTA, single-arm, multicenter trial in Japanese patients with ALK+ NSCLC, focusing on patients who have progressed on alectinib. This trial is now enrolling.
Phase 2 ALTA 2, global, single-arm trial evaluating ALUNBRIG in patients with advanced ALK+ NSCLC who have progressed on alectinib or ceritinib. This trial is now enrolling.
Phase 3 ALTA 3, global randomized trial comparing the efficacy and safety of ALUNBRIG versus alectinib in participants with ALK+ NSCLC who have progressed on crizotinib. This trial is now enrolling.
TAK-788

Phase 1/2 study evaluating the safety, pharmacokinetics and antitumor activity of oral EGFR/HER2 inhibitor TAK-788 in patients with NSCLC.
Phase 2 EXCLAIM, pivotal extension cohort of the Phase 1/2 trial, which was designed to evaluate the efficacy and safety of TAK-788 at 160 mg once daily in previously treated patients with EGFR exon 20 insertion mutations. This trial is closed to enrollment.
Phase 3 EXCLAIM 2, global, randomized study evaluating the efficacy of TAK-788 as a first-line treatment compared to platinum-based chemotherapy in treatment-naïve patients with locally advanced or metastatic NSCLC whose tumors harbor EGFR exon 20 insertion mutations.
Phase 1, open-label, multicenter, dose-escalation study evaluating the safety, tolerability and pharmacokinetics of TAK-788 in Japanese patients with locally advanced or metastatic NSCLC. This trial has been fully enrolled.
Phase 2, open label, single-arm study evaluating the efficacy of TAK-788 in treatment-naïve patients with locally advanced or metastatic NSCLC whose tumors harbor EGFR exon 20 insertion mutations.
Phase 1, open-label, two-period, fixed-sequence study designed to characterize drug-drug interaction between TAK-788 and either a strong cytochrome P-450 (CYP)3A inhibitor, itraconazole (Part 1) or a strong CYP3A inducer, rifampin (Part 2) in healthy adult subjects.
For additional information on the ALUNBRIG and TAK-788 clinical trials, please visit www.clinicaltrials.gov.

About ALK+ NSCLC
Non-small cell lung cancer (NSCLC) is the most common form of lung cancer, accounting for approximately 85% of the estimated 1.8 million new cases of lung cancer diagnosed each year worldwide, according to the World Health Organization.1,2 Genetic studies indicate that chromosomal rearrangements in anaplastic lymphoma kinase (ALK) are key drivers in a subset of NSCLC patients.3 Approximately three to five percent of patients with metastatic NSCLC have a rearrangement in the ALK gene.4,5,6

Takeda is committed to continuing research and development in NSCLC to improve the lives of the approximately 40,000 patients diagnosed with this serious and rare form of lung cancer worldwide each year.7

ALUNBRIG IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS
Interstitial Lung Disease (ILD)/Pneumonitis: Severe, life-threatening, and fatal pulmonary adverse reactions consistent with interstitial lung disease (ILD)/pneumonitis have occurred with ALUNBRIG. In Trial ALTA (ALTA), ILD/pneumonitis occurred in 3.7% of patients in the 90 mg group (90 mg once daily) and 9.1% of patients in the 90→180 mg group (180 mg once daily with 7-day lead-in at 90 mg once daily). Adverse reactions consistent with possible ILD/pneumonitis occurred early (within 9 days of initiation of ALUNBRIG; median onset was 2 days) in 6.4% of patients, with Grade 3 to 4 reactions occurring in 2.7%. Monitor for new or worsening respiratory symptoms (e.g., dyspnea, cough, etc.), particularly during the first week of initiating ALUNBRIG. Withhold ALUNBRIG in any patient with new or worsening respiratory symptoms, and promptly evaluate for ILD/pneumonitis or other causes of respiratory symptoms (e.g., pulmonary embolism, tumor progression, and infectious pneumonia). For Grade 1 or 2 ILD/pneumonitis, either resume ALUNBRIG with dose reduction after recovery to baseline or permanently discontinue ALUNBRIG. Permanently discontinue ALUNBRIG for Grade 3 or 4 ILD/pneumonitis or recurrence of Grade 1 or 2 ILD/pneumonitis.

Hypertension: In ALTA, hypertension was reported in 11% of patients in the 90 mg group who received ALUNBRIG and 21% of patients in the 90→180 mg group. Grade 3 hypertension occurred in 5.9% of patients overall. Control blood pressure prior to treatment with ALUNBRIG. Monitor blood pressure after 2 weeks and at least monthly thereafter during treatment with ALUNBRIG. Withhold ALUNBRIG for Grade 3 hypertension despite optimal antihypertensive therapy. Upon resolution or improvement to Grade 1 severity, resume ALUNBRIG at a reduced dose. Consider permanent discontinuation of treatment with ALUNBRIG for Grade 4 hypertension or recurrence of Grade 3 hypertension. Use caution when administering ALUNBRIG in combination with antihypertensive agents that cause bradycardia.

Bradycardia: Bradycardia can occur with ALUNBRIG. In ALTA, heart rates less than 50 beats per minute (bpm) occurred in 5.7% of patients in the 90 mg group and 7.6% of patients in the 90→180 mg group. Grade 2 bradycardia occurred in 1 (0.9%) patient in the 90 mg group. Monitor heart rate and blood pressure during treatment with ALUNBRIG. Monitor patients more frequently if concomitant use of drug known to cause bradycardia cannot be avoided. For symptomatic bradycardia, withhold ALUNBRIG and review concomitant medications for those known to cause bradycardia. If a concomitant medication known to cause bradycardia is identified and discontinued or dose adjusted, resume ALUNBRIG at the same dose following resolution of symptomatic bradycardia; otherwise, reduce the dose of ALUNBRIG following resolution of symptomatic bradycardia. Discontinue ALUNBRIG for life-threatening bradycardia if no contributing concomitant medication is identified.

Visual Disturbance: In ALTA, adverse reactions leading to visual disturbance including blurred vision, diplopia, and reduced visual acuity, were reported in 7.3% of patients treated with ALUNBRIG in the 90 mg group and 10% of patients in the 90→180 mg group. Grade 3 macular edema and cataract occurred in one patient each in the 90→180 mg group. Advise patients to report any visual symptoms. Withhold ALUNBRIG and obtain an ophthalmologic evaluation in patients with new or worsening visual symptoms of Grade 2 or greater severity. Upon recovery of Grade 2 or Grade 3 visual disturbances to Grade 1 severity or baseline, resume ALUNBRIG at a reduced dose. Permanently discontinue treatment with ALUNBRIG for Grade 4 visual disturbances.

Creatine Phosphokinase (CPK) Elevation: In ALTA, creatine phosphokinase (CPK) elevation occurred in 27% of patients receiving ALUNBRIG in the 90 mg group and 48% of patients in the 90 mg→180 mg group. The incidence of Grade 3‑4 CPK elevation was 2.8% in the 90 mg group and 12% in the 90→180 mg group. Dose reduction for CPK elevation occurred in 1.8% of patients in the 90 mg group and 4.5% in the 90→180 mg group. Advise patients to report any unexplained muscle pain, tenderness, or weakness. Monitor CPK levels during ALUNBRIG treatment. Withhold ALUNBRIG for Grade 3 or 4 CPK elevation. Upon resolution or recovery to Grade 1 or baseline, resume ALUNBRIG at the same dose or at a reduced dose.

Pancreatic Enzyme Elevation: In ALTA, amylase elevation occurred in 27% of patients in the 90 mg group and 39% of patients in the 90→180 mg group. Lipase elevations occurred in 21% of patients in the 90 mg group and 45% of patients in the 90→180 mg group. Grade 3 or 4 amylase elevation occurred in 3.7% of patients in the 90 mg group and 2.7% of patients in the 90→180 mg group. Grade 3 or 4 lipase elevation occurred in 4.6% of patients in the 90 mg group and 5.5% of patients in the 90→180 mg group. Monitor lipase and amylase during treatment with ALUNBRIG. Withhold ALUNBRIG for Grade 3 or 4 pancreatic enzyme elevation. Upon resolution or recovery to Grade 1 or baseline, resume ALUNBRIG at the same dose or at a reduced dose.

Hyperglycemia: In ALTA, 43% of patients who received ALUNBRIG experienced new or worsening hyperglycemia. Grade 3 hyperglycemia, based on laboratory assessment of serum fasting glucose levels, occurred in 3.7% of patients. Two of 20 (10%) patients with diabetes or glucose intolerance at baseline required initiation of insulin while receiving ALUNBRIG. Assess fasting serum glucose prior to initiation of ALUNBRIG and monitor periodically thereafter. Initiate or optimize anti-hyperglycemic medications as needed. If adequate hyperglycemic control cannot be achieved with optimal medical management, withhold ALUNBRIG until adequate hyperglycemic control is achieved and consider reducing the dose of ALUNBRIG or permanently discontinuing ALUNBRIG.

Embryo-Fetal Toxicity: Based on its mechanism of action and findings in animals, ALUNBRIG can cause fetal harm when administered to pregnant women. There are no clinical data on the use of ALUNBRIG in pregnant women. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective non-hormonal contraception during treatment with ALUNBRIG and for at least 4 months following the final dose. Advise males with female partners of reproductive potential to use effective contraception during treatment and for at least 3 months after the last dose of ALUNBRIG.

ADVERSE REACTIONS
Serious adverse reactions occurred in 38% of patients in the 90 mg group and 40% of patients in the 90→180 mg group. The most common serious adverse reactions were pneumonia (5.5% overall, 3.7% in the 90 mg group, and 7.3% in the 90→180 mg group) and ILD/pneumonitis (4.6% overall, 1.8% in the 90 mg group and 7.3% in the 90→180 mg group). Fatal adverse reactions occurred in 3.7% of patients and consisted of pneumonia (2 patients), sudden death, dyspnea, respiratory failure, pulmonary embolism, bacterial meningitis and urosepsis (1 patient each).

The most common adverse reactions (≥25%) in the 90 mg group were nausea (33%), fatigue (29%), headache (28%), and dyspnea (27%) and in the 90→180 mg group were nausea (40%), diarrhea (38%), fatigue (36%), cough (34%), and headache (27%).

DRUG INTERACTIONS
CYP3A Inhibitors: Avoid coadministration of ALUNBRIG with strong or moderate CYP3A inhibitors. Avoid grapefruit or grapefruit juice as it may also increase plasma concentrations of brigatinib. If coadministration of a strong or moderate CYP3A inhibitor cannot be avoided, reduce the dose of ALUNBRIG.
CYP3A Inducers: Avoid coadministration of ALUNBRIG with strong or moderate CYP3A inducers. If coadministration of moderate CYP3A inducers cannot be avoided, increase the dose of ALUNBRIG.
CYP3A Substrates: Coadministration of ALUNBRIG with sensitive CYP3A substrates, including hormonal contraceptives, can result in decreased concentrations and loss of efficacy of sensitive CYP3A substrates.

USE IN SPECIFIC POPULATIONS
Pregnancy: ALUNBRIG can cause fetal harm. Advise females of reproductive potential of the potential risk to a fetus.

Lactation: There are no data regarding the secretion of brigatinib in human milk or its effects on the breastfed infant or milk production. Because of the potential adverse reactions in breastfed infants, advise lactating women not to breastfeed during treatment with ALUNBRIG.

Females and Males of Reproductive Potential:
Pregnancy Testing: Verify pregnancy status in females of reproductive potential prior to initiating ALUNBRIG.
Contraception: Advise females of reproductive potential to use effective non-hormonal contraception during treatment with ALUNBRIG and for at least 4 months after the final dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with ALUNBRIG and for at least 3 months after the final dose.
Infertility: ALUNBRIG may cause reduced fertility in males.

Pediatric Use: The safety and effectiveness of ALUNBRIG in pediatric patients have not been established.

Geriatric Use: Clinical studies of ALUNBRIG did not include sufficient numbers of patients aged 65 years and older to determine whether they respond differently from younger patients.

Hepatic or Renal Impairment: No dose adjustment is recommended for patients with mild or moderate hepatic impairment or mild or moderate renal impairment. Reduce the dose of ALUNBRIG for patients with severe hepatic impairment or severe renal impairment.

Agios Presents New Pharmacodynamic and Response Data from Both Cohorts of the Perioperative Study of Vorasidenib and TIBSOVO® (ivosidenib) in Patients with IDH1 Mutant Positive Low-Grade Glioma

On November 22, 2019 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported updated data from the ongoing perioperative study, confirming brain penetrance and robust biomarker suppression with treatment of single agent vorasidenib or TIBSOVO (ivosidenib) in low-grade glioma with an IDH1 mutation (Press release, Agios Pharmaceuticals, NOV 22, 2019, View Source [SID1234551609]). The data were featured in an oral presentation at the Society for Neuro-Oncology (SNO) Annual Meeting in Phoenix. Vorasidenib, an investigational, oral, selective, inhibitor of the mutant isocitrate dehydrogenase-1 (IDH1) and IDH2 enzymes, was designed for enhanced brain penetrance and selected for pivotal development in IDH mutant low-grade glioma.

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"Now with data from both cohorts of the perioperative study, we have further evidence demonstrating that vorasidenib has excellent brain penetrance and suppresses 2-HG in IDH1 mutant gliomas," said Ingo Mellinghoff, M.D., Memorial Sloan Kettering Cancer Center, an investigator for the study. "In addition, it is encouraging that the safety profile continues to be consistent, and preliminary efficacy data show objective tumor responses and durable disease control with postoperative treatment. These data support the selection of vorasidenib for pivotal development and help establish the potential role for IDH inhibitors in the treatment of low-grade glioma."

"These data build on our initial findings that led us to the selection of vorasidenib for a pivotal study in low-grade glioma," said Chris Bowden, M.D., chief medical officer at Agios. "Having now demonstrated brain penetrance, robust 2-HG suppression and an encouraging disease-control rate with vorasidenib, we’re confident in our ability to make a difference for patients with IDH mutant low-grade glioma and are on track to initiate the Phase 3 INDIGO study next month."

Perioperative Study of Vorasidenib and TIBSOVO
Vorasidenib and TIBSOVO are being evaluated as a single agent in an ongoing perioperative study in IDH1 mutant Grade 2/3 glioma. The primary endpoint is 2-hydroxyglutarate (2-HG) concentration in tumors resected following presurgical treatment with vorasidenib and TIBSOVO compared with untreated control tumors. Patients were randomized to 500 mg TIBSOVO once daily, 50 mg vorasidenib once daily or the control arm in cohort 1; and 250 mg TIBSOVO twice daily or 10 mg vorasidenib once daily in cohort 2. Patients were treated for four weeks prior to surgery and had the option to continue postoperative treatment until disease progression.

As of the July 26, 2019 data cutoff, 49 patients were randomized before surgery, and 39 remain on treatment. The median (range) postoperative treatment duration for all doses was 5.42 (0.9–13.5) months for vorasidenib and 6.93 (1.0–13.2) months for TIBSOVO. Baseline characteristics were similar across the control and treated groups. Overall, 88% of patients had World Health Organization (WHO) classified Grade 2 tumors. Less than a third of patients had prior radiation therapy and approximately half had prior systemic therapy.

Safety Data
The safety analysis conducted for all 49 patients as of the data cut-off demonstrated that vorasidenib and TIBSOVO continue to have favorable safety profiles consistent with the Phase 1 data.

The most common adverse events (AE) occurring in >25% of patients in the vorasidenib arm were diarrhea (29.2%), fatigue (29.2%) and nausea (29.2%). In the TIBSOVO arm, the most common AEs were headache (32%), diarrhea (28%) and anemia (28%).
8.3% of patients in the vorasidenib group developed transaminase elevations, including one Grade 3 transaminase elevation at 50 mg daily which resolved with dose interruption.
Grade 3 or higher events occurred in six (25.0%) vorasidenib patients and four (16.0%) TIBSOVO patients, with the majority related to postoperative complications.
No patient discontinued treatment due to an AE.
Pharmacokinetics and Pharmacodynamics

Vorasidenib and TIBSOVO demonstrated brain penetrance, with mean brain:plasma ratios of 3.16 (vorasidenib 10 mg), 1.74 (vorasidenib 50 mg), 0.13 (TIBSOVO 250 mg) and 0.10 (TIBSOVO 500 mg).
Mean percent reduction in 2-HG (95% CI) were 92.6% (76.1, 97.6) and 91.1% (72.0, 97.0), for vorasidenib 50 mg and TIBSOVO 500 mg, respectively, relative to untreated samples.
Efficacy Data
Data from the 42 efficacy evaluable patients (21 in the vorasidenib arm; 21 in the TIBSOVO arm) as of the data cut-off showed:

Among patients treated post-operatively with 50 mg vorasidenib, four patients (31%) achieved objective tumor responses (two achieved a partial response, defined as tumor shrinkage based on T2/FLAIR signal of at least 50%, and two achieved a minor response, defined as tumor shrinkage based on T2/FLAIR signal of at least 25% but less than 50%) according to the investigator by Response Assessment in Neuro-Oncology for low-grade glioma (RANO-LGG).
Among patients treated post-operatively with 500 mg TIBSOVO, four patients (31%) achieved confirmed responses (two achieved a partial response and two achieved a minor response) according to the investigator by RANO-LGG.
15 patients treated with vorasidenib and 16 patients treated with TIBSOVO experienced stable disease, resulting in disease control rates of 90% and 95% respectively.
About the Phase 3 INDIGO Study
The Phase 3 INDIGO study will evaluate 366 patients with IDH mutant Grade 2 non-enhancing glioma in a 1:1 double-blind randomization to either 50 mg of vorasidenib once daily or placebo.

The primary endpoint is progression free survival, as assessed by a blinded independent review committee.
Secondary endpoints include safety and tolerability, tumor growth rate as assessed by volume, overall response rate, time to next intervention and quality of life.
Crossover from placebo to vorasidenib upon centrally confirmed radiographic progression will be permitted.
The study will initiate by the end of 2019.

TIBSOVO and vorasidenib are not approved in any country for the treatment of patients with low-grade glioma.

About Glioma
Glioma presents in varying degrees of tumor aggressiveness, ranging from slower growing (low-grade glioma) to rapidly progressing (high-grade glioma-Glioblastoma Multiforme). Common symptoms include seizures, memory disturbance, sensory impairment and neurologic deficits. The long-term prognosis is poor, and regardless of treatment, the majority of patients with low-grade gliomas will have recurrent disease that will progress over time. Approximately 11,000 low-grade glioma patients are diagnosed annually in the U.S. and EU and approximately 80 percent have an IDH mutation.

About TIBSOVO (ivosidenib)

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.