Sanofi and Regeneron Announce That Cemiplimab (REGN2810) Has Received FDA Breakthrough Therapy Designation for Advanced Cutaneous Squamous Cell Carcinoma

On September 8, 2017 Sanofi and Regeneron Pharmaceuticals, Inc. reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation status to cemiplimab (REGN2810) for the treatment of adults with metastatic cutaneous squamous cell carcinoma (CSCC) and adults with locally advanced and unresectable CSCC, the second deadliest skin cancer after melanoma[i], Anchor[ii] (Press release, Sanofi, SEP 8, 2017, View Source [SID1234520415]). Cemiplimab is an investigational human, monoclonal antibody targeting PD-1.
Sanofi and Regeneron previously reported positive, preliminary results for cemiplimab from two expansion cohorts involving 26 advanced CSCC patients in a Phase 1 study of nearly 400 patients, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2017. EMPOWER-CSCC 1, a Phase 2, potentially pivotal, single-arm, open label clinical trial of cemiplimab is currently enrolling patients for metastatic CSCC and locally advanced and unresectable CSCC. Cemiplimab is being jointly developed by Sanofi and Regeneron under a global collaboration agreement. Pending data results, the companies anticipate submitting a biologics license application for cemiplimab with the FDA in the first quarter of 2018.
CSCC is the second most common type of skin cancer in the United States. Although CSCC has a good prognosis when caught early, it can prove especially difficult to treat when it progresses to advanced stages. Patients at this stage can be disfigured due to multiple surgeries to remove CSCC tumors on the head, neck and other parts of the body.[iii] CSCC is responsible for the most deaths among non-melanoma skin cancer patients.ii
Breakthrough Therapy designation serves to expedite the development and review of drugs that target serious or life-threatening conditions. Drugs qualifying for this designation must show credible evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy. The designation includes all of the Fast Track program features, as well as more intensive FDA guidance and discussion. The Breakthrough Therapy designation is distinct from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.[iv]
Cemiplimab is currently under clinical development, and its safety and efficacy has not been fully evaluated by any regulatory authority.

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ERYTECH ANNOUNCES POSITIVE FULL RESULTS FROM PHASE 2B STUDY OF ERYASPASE IN COMBINATION WITH CHEMOTHERAPY FOR TREATMENT OF METASTATIC PANCREATIC CANCER IN SECOND-LINE

On September 8, 2017 ERYTECH Pharma (Paris:ERYP) (ADR:EYRYY) (Euronext Paris – ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported the presentation of the full data from its Phase 2b study evaluating eryaspase (GRASPA) in combination with chemotherapy for the treatment of metastatic pancreatic cancer (Press release, ERYtech Pharma, SEP 8, 2017, View Source [SID1234520455]). The open-label, multi-center, randomized Phase 2b clinical study met its co-primary endpoints and demonstrated significant improvement in both overall survival (OS) and progression-free survival (PFS). The results will be presented during the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Meeting in Madrid.

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The Phase 2b study evaluated eryaspase, L-asparaginase encapsulated in red blood cells, as a second-line treatment in combination with chemotherapy in 141 patients suffering from metastatic pancreatic cancer. In this study, conducted in France, eryaspase was added to the current standard of care (gemcitabine or FOLFOX) and compared to the standard of care alone in a 2-to-1 randomization. Approximately 90% of patients received gemcitabine. Baseline characteristics and patient demographics were similar between the two treatment groups.

As reported in topline results earlier this year, the study met its co-primary endpoints of OS and PFS with Hazard Ratios (HR) below 0.85 in patients with no or low asparagine synthetase expression (ASNS 0/1), approximately 70% of the study population, and demonstrated statistically significant improvements of OS and PFS in the entire patient population. The associated sensitivity analyses and subgroup evaluations indicate the consistent treatment benefit with eryaspase across the treated populations.

Principal Investigator Professor Pascal Hammel, gastroenterologist-oncologist and head of the Oncology Unit at Beaujon Hospital in Paris, commented, "The full results of this study are highly encouraging and support eryaspase as a potential treatment option for patients with metastatic pancreatic cancer in the second-line setting."

Highlights of the study results include:

Co-primary endpoints met:
HR of 0.65 for OS and 0.72 for PFS in the ASNS 0/1 patient population
Statistically significant improvement of OS and PFS in the entire patient population:
HR of 0.60 for OS (95% CI; 0.40, 0.88) (p=0.009)
median OS of 26.1 weeks (95% CI; 21.0, 28.4) in the eryaspase arm vs. 19.0 weeks (95% CI; 12.3, 26.3) in the standard of care arm
one-year survival of 14.8% vs. 3.0%, respectively
HR of 0.59 for PFS (95% CI; 0.40, 0.89) (p=0.011)
median PFS of 8.6 weeks (95% CI; 7.6, 14.6) in the eryaspase arm vs. 7.0 weeks (95% CI; 6.1, 7.6) in the standard of care arm
16.9% of patients without disease progression at 24 weeks vs. 5.8%, respectively
Improved objective response rate (ORR) and disease control rate (DCR) in the entire patient population:
ORR of 11.6% in the eryaspase arm vs. 6.5% in the standard of care arm
DCR of 47.4% in the eryaspase arm vs. 23.9% in the standard of care arm
Patients with high ASNS-expressing tumors (ASNS2/3) had a worse prognosis, but also a better relative treatment benefit:
HR of 0.45 for OS and 0.38 for PFS
DCR of 51.7% in the eryaspase arm vs. 7.1% in the standard of care arm
The toxicity profile was similar in the two treatment arms:
The percentage of patients with at least one Grade 3 or 4 adverse event (AE) was 77% in the eryaspase-treated arm compared to 86% in the control arm. The most common Grade 3 or 4 AEs were: increased gamma-glutamyl transferase (17% vs. 25%), neutropenia (13% vs. 11%), general health deterioration (13% vs. 2%), and thrombocytopenia (10% vs. 7%), respectively.
The percentage of patients with at least one serious adverse events (SAE) was 45% in the eryaspase-treated arm compared to 50% in the control arm. The most common SAEs were: general health deterioration (9% each), gastro-intestinal hemorrhage (2% vs. 7%, respectively).
Dr. Iman El-Hariry, Chief Medical Officer of ERYTECH, stated, "Despite intense research efforts, limited progress has been made toward increased overall survival and metastatic pancreatic cancer remains a high unmet medical need. We are quite impressed with this study outcome, particularly with the overall survival advantage demonstrated in the eryaspase arm. These results underscore the importance of targeting the metabolic pathways in pancreatic cancer and potentially other solid tumors."

"We are very pleased by the results from this landmark study. The full picture emerging from these data shows a robust clinical benefit in this particularly difficult-to-treat and highly morbid form of cancer," said Gil Beyen, Chairman and CEO of ERYTECH. "Eryaspase adds an entirely new mode of action to the fight against this terrible disease and opens avenues to other solid tumor indications. We are working with the regulatory agencies to develop a Phase 3 plan in pancreatic cancer, and we are exploring other solid tumor indications for our product candidate."

The full poster presentation will be accessible on September 8, 2017 within the "Investors" section of ERYTECH’s website at www.investors.erytech.com.

ERYTECH will also host an investor and analyst event on Monday, September 11 at 6:15 p.m. CEST at ESMO (Free ESMO Whitepaper) 2017, in Madrid. Pre-registration for the live event at ESMO (Free ESMO Whitepaper) 2017 is required. To RSVP, please contact Janhavi Mohite at [email protected].

For those unable to attend, a live webcast will be accessible at the start of the event starting at 06:30pm CEST, and is available for replay on the "Investors" of the ERYTECH’s website at View Source

About pancreatic cancer:

Pancreatic cancer is a disease in which malignant (cancer) cells are found in the tissues of the pancreas. Every year, there are approximately 150,000 new cases of pancreatic cancer diagnosed in Europe and the United States. Pancreatic cancer is a particularly aggressive cancer, with a five-year survival rate of approximately 9%. It is currently the fourth leading cause of cancer death in the United States and is projected to rise to the second leading cause by 2030. Limited therapeutic options are currently available for this indication, thereby reinforcing the need to develop new therapeutic strategies and rational drug combinations with the aim of improving overall patient outcomes and quality of life.

Updated Data for Merck’s KEYTRUDA® (pembrolizumab) in Patients with Advanced Gastric Cancer Across Treatment Settings to Be Presented at ESMO 2017 Congress

On September 8, 2017 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported the presentation of data from all three cohorts of the registrational, phase 2 KEYNOTE-059 trial investigating the use of KEYTRUDA (pembrolizumab), the company’s anti-PD-1 therapy, in patients with advanced gastric or gastroesophageal junction (GEJ) adenocarcinoma, including new data in treatment-naïve patients (Press release, Merck & Co, SEP 8, 2017, View Source [SID1234520453]). Overall, results showed antitumor activity and durability of response with KEYTRUDA across multiple lines of therapy, with higher response rates observed in PD-L1-positive (CPS ≥1) patients:

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In heavily pre-treated patients, KEYTRUDA monotherapy (Cohort 1) showed an overall response rate (ORR) of 12 percent (95% CI, 8-17) in all patients and 16 percent (95% CI, 11-23) in patients with PD-L1 positive tumors.
In treatment-naïve patients, KEYTRUDA in combination with chemotherapy (Cohort 2) showed an ORR of 60 percent (95% CI, 39-79) in all patients and 69 percent (95% CI, 41-89) in patients with PD-L1 positive tumors.
In treatment-naïve patients with PD-L1 positive tumors, KEYTRUDA monotherapy (Cohort 3) showed an ORR of 26 percent (95% CI, 12-45).
Results are being presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress in Madrid, Spain, in an oral presentation on Friday, Sept. 8 from 3:03-3:15 p.m. CEST (Location: Barcelona Auditorium) (Abstract #LBA28_PR).

"Outcomes can vary significantly among patients with gastric or GEJ cancer – across stages of disease and based on a variety of biological and molecular makeups of tumors," said Charles S. Fuchs, M.D., MPH, lead investigator and director of Yale Cancer Center. "KEYNOTE-059 was designed to provide important insight across various treatment settings, simultaneously, and these results show encouraging activity with pembrolizumab in both previously treated and treatment-naïve patients, particularly in those with high levels of PD-L1 expression."

"These findings across lines of therapy in gastric and GEJ cancers continue to support the potential for KEYTRUDA, and PD-L1 as a biomarker, in patients with this advanced stage disease," said Dr. Roger Dansey, senior vice president and therapeutic area head, oncology late-stage development, Merck Research Laboratories. "We are encouraged by the responses observed in heavily pre-treated patients and look forward to continued analysis of data in treatment-naïve patients."

Merck’s broad clinical program encompasses multiple gastrointestinal disorders – such as gastroesophageal cancer and microsatellite instability-high (MSI-H) colorectal cancer – and includes four gastric/GEJ cancer registration-enabling studies and numerous other gastrointestinal cancer studies currently underway.

Data from KEYNOTE-059 Study Cohorts (Abstract #LBA28_PR)

Results presented from KEYNOTE-059 were based on an analysis of efficacy, safety and PD-L1 expression from 315 patients across the study’s three cohorts. Patients were considered PD-L1 positive if they had a PD-L1 combined positive score of one or more (CPS ≥1). Findings showed:

Cohort 1: KEYTRUDA (pembrolizumab) as monotherapy in patients whose disease progressed on or after two or more prior lines of therapy (n=259). Forty-eight percent of these patients had received three or more lines of prior therapy. The efficacy analysis in all patients, with or without PD-L1 expression, showed an ORR of 12 percent (95% CI, 8-17), with complete responses (CR) in three percent (95% CI, 1-6) and partial responses (PR) in nine percent (95% CI, 6-13) of patients. In patients whose tumors expressed PD-L1 (n=148), the ORR was 16 percent (95% CI, 11-23), with CR in three percent (95% CI, 1-8) and PR in 13 percent (95% CI, 8-19) of patients. In PD-L1 negative patients (n=109), the ORR was six percent (95% CI, 3-13), with CR in three percent (95% CI, 1-8) and PR in four percent (95% CI, 1-9) of patients. Median duration of follow-up was 5.6 months (range: 0.5-24.7). In all patients, 42 percent (n=95) experienced a reduction in target lesion size. Median duration of response was 14.2 months (range: 2.4-19.4+).

In all patients, median progression-free survival (PFS) was two months (95% CI, 2.0-2.1), with a six-month PFS rate of 14.6 percent; the median overall survival (OS) was 5.5 months (95% CI, 4.2-6.5), with a six-month OS rate of 45.7 percent. In patients whose tumors expressed PD-L1, the median PFS was 2.1 months (95% CI, 2.0-2.1), with a six-month PFS rate of 18.2 percent; the median OS in these patients was 5.8 months (95% CI, 4.4-7.8), with a six-month OS rate of 48.4 percent. In PD-L1 negative patients, the median PFS was two months (95% CI, 1.9-2.0), with a six-month PFS rate of 9.9 percent; the median OS was 4.6 months (95% CI, 3.2-6.5), with a six-month OS rate of 42.9 percent.
Cohort 2: KEYTRUDA (pembrolizumab) in combination with chemotherapy in treatment-naïve patients (n=25). The efficacy analysis in all patients showed an ORR of 60 percent (95% CI, 39-79), with CR in four percent (95% CI, 0-20) and PR in 56 percent (95% CI, 35-76) of patients. In patients whose tumors expressed PD-L1 (n=16), the ORR was 69 percent (95% CI, 41-89), with no complete responses (95% CI, 0-22) and PR in 69 percent (95% CI, 41-89) of patients. In PD-L1 negative patients (n=8), the ORR was 38 percent (95% CI, 9-76), with CR in 13 percent (95% CI, 0-53) and PR in 25 percent (95% CI, 3-65) of patients. Median duration of follow-up was 13.8 months (range: 1.8-24.1). Across all patients, 96 percent (n=24) experienced a reduction in target lesion size. Median duration of response was 4.6 months (range: 2.6-20.3+). In all patients, median PFS was 6.6 months (95% CI, 5.9-10.6), with a six-month PFS rate of 68.0 percent; the median OS was 13.8 months (95% CI, 8.6-not reached), with a six-month OS rate of 76.0 percent.
Cohort 3: KEYTRUDA as monotherapy in treatment-naïve patients whose tumors expressed PD-L1 (n=31). The efficacy analysis showed an ORR of 26 percent (95% CI, 12-45), with CR in seven percent (95% CI, 1-21) and PR in 19 percent (95% CI, 8-38) of patients. Median duration of follow-up was 17.5 months (range: 1.7-20.7). Seventy-seven percent of patients (n=24) experienced a reduction in target lesion size. The median duration of response was 9.6 months (range: 2.1-17.8+). The median PFS was 3.3 months (95% CI, 2.0-6.0), with a six-month PFS rate of 34.9 percent; the median OS was 20.7 months (95% CI, 9.2-20.7), with a six-month OS rate of 72.9 percent.
The safety profile of KEYTRUDA was consistent with that observed in previously reported studies. In Cohorts 1, 2 and 3, Grade 3-5 treatment-related adverse events (TRAEs) occurred in 46 (18%), 19 (76%), and 7 (23%) patients, respectively. In Cohort 1, Grade 3-5 TRAEs were anemia (3%), fatigue (2%), and dehydration (1%); TRAEs led to discontinuation in seven patients (3%) and death in two patients (1%). In Cohort 2, Grade 3/4 TRAEs were neutropenia (24%), stomatitis (20%), anemia (8%), decreased platelet count (8%), decreased appetite (8%), and fatigue (8%). TRAEs in Cohort 2 led to discontinuation in three patients (12%); there were no treatment-related deaths. In Cohort 3, Grade 3-5 TRAEs occurred in seven patients (23%); there was one treatment-related death (3%) and no discontinuations due to TRAEs.

In Cohorts 1, 2 and 3, Grade 3 or higher immune-mediated adverse events occurred in 13, four, and three patients, respectively. In Cohort 1, Grade 3 immune-mediated adverse events were colitis (n=3), pneumonitis (n=2), thyroiditis (n=1), and hypothyroidism (n=1); there were no Grade 4-5 immune-mediated adverse events. In Cohort 2, Grade 3 immune-mediated adverse events were palmar-plantar erythrodysesthesia (n=2), nephrotic syndrome (n=1), rash (n=1), and maculopapular rash (n=1); there were no Grade 4-5 immune-mediated adverse events. In Cohort 3, Grade 3 immune-mediated adverse events were colitis (n=1) and rash (n=1); there was one Grade 5 adverse event (pneumonitis).

About KEYNOTE-059

KEYNOTE-059 is a registrational, phase 2 non-randomized, multi-cohort study (Cohorts 1, 2 and 3) investigating KEYTRUDA (pembrolizumab) in patients with advanced gastric or GEJ adenocarcinoma. Patients in Cohort 1 received KEYTRUDA monotherapy after two or more prior lines of therapy. Patients in Cohort 2 had received no prior therapy and received KEYTRUDA in combination with cisplatin (80 mg/m2 Q3W) and 5-fluorouracil (800 mg/m2 Q3W) or capecitabine (in Japan only, 1000 mg/m2 BID Q3W) as a first-line therapy. Patients in Cohort 3, which only enrolled patients whose tumors were positive for PD-L1, received KEYTRUDA monotherapy as a first-line therapy. In all cohorts, KEYTRUDA was given at 200 mg every three weeks for up to 24 months. The primary endpoints are safety (all cohorts) and ORR using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Key secondary endpoints are ORR and duration of response by RECIST v1.1, PFS, and OS. Data from Cohorts 1 and 2 were further assessed based on PD-L1 expression. Tumors were considered to have positive PD-L1 expression if the combined positive score (CPS) – as examined by tumor and immune cells – was equal to or greater than one.

About Gastric Cancer

Gastric cancer, also called stomach cancer, is a type of cancer that begins in the stomach and tends to develop slowly over many years. Most gastric cancers are adenocarcinomas, which develop from the cells of the innermost lining (mucosa) of the stomach. Risk factors for gastric cancer include gender, age, ethnicity, geography and infection with Helicobacter pylori. Worldwide, gastric cancer is the fifth most common type of cancer and the third leading cause of cancer death. Each year there are approximately 952,000 newly diagnosed cases of gastric cancer resulting in approximately 723,000 deaths worldwide.

About KEYTRUDA (pembrolizumab) Injection 100mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that 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.

Studies of KEYTRUDA – from the largest immuno-oncology program in the industry with more than 550 trials – include a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand factors that predict a patient’s likelihood of benefitting from treatment with KEYTRUDA, including the exploration of several different biomarkers across a broad range of tumors.

KEYTRUDA (pembrolizumab) Indications and Dosing

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma at a fixed dose of 200 mg every three weeks until disease progression or unacceptable toxicity.

Lung Cancer

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have high PD-L1 expression [tumor proportion score (TPS) ≥50%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, as a single agent, is also indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

KEYTRUDA, in combination with pemetrexed and carboplatin, is indicated for the first-line treatment of patients with metastatic nonsquamous NSCLC. This indication is approved under accelerated approval based on tumor response rate and progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

In metastatic NSCLC, KEYTRUDA (pembrolizumab) is administered at a fixed dose of 200 mg every three weeks until disease progression, unacceptable toxicity, or up to 24 months in patients without disease progression.

When administering KEYTRUDA in combination with chemotherapy, KEYTRUDA should be administered prior to chemotherapy when given on the same day. See also the Prescribing Information for pemetrexed and carboplatin.

Head and Neck Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. In HNSCC, KEYTRUDA is administered at a fixed dose of 200 mg every three weeks until disease progression, unacceptable toxicity, or up to 24 months in patients without disease progression.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after three or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. In adults with cHL, KEYTRUDA is administered at a fixed dose of 200 mg every three weeks until disease progression or unacceptable toxicity, or up to 24 months in patients without disease progression. In pediatric patients with cHL, KEYTRUDA is administered at a dose of 2 mg/kg (up to a maximum of 200 mg) every three weeks until disease progression or unacceptable toxicity, or up to 24 months in patients without disease progression.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who are not eligible for cisplatin-containing chemotherapy. 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 the confirmatory trials.

KEYTRUDA is also indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

In locally advanced or metastatic urothelial carcinoma, KEYTRUDA (pembrolizumab) is administered at a fixed dose of 200 mg every three weeks until disease progression or unacceptable toxicity, or up to 24 months in patients without disease progression.

Microsatellite Instability-High (MSI-H) Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options, or
colorectal cancer that has progressed following treatment with fluoropyrimidine, oxaliplatin, and irinotecan.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

In adult patients with MSI-H cancer, KEYTRUDA is administered at a fixed dose of 200 mg every three weeks until disease progression, unacceptable toxicity, or up to 24 months in patients without disease progression. In pediatric patients with MSI-H cancer, KEYTRUDA is administered at a dose of 2 mg/kg (up to a maximum of 200 mg) every three weeks until disease progression or unacceptable toxicity, or up to 24 months in patients without disease progression.

Selected Important Safety Information for KEYTRUDA (pembrolizumab)

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 94 (3.4%) of 2799 patients receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%) pneumonitis, and occurred more frequently in patients with a history of prior thoracic radiation (6.9%) compared to those without (2.9%). Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 48 (1.7%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%) colitis. Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA (pembrolizumab) for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 19 (0.7%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%) hepatitis. Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

KEYTRUDA can cause hypophysitis. Hypophysitis occurred in 17 (0.6%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%) hypophysitis. Monitor patients for signs and symptoms of hypophysitis (including hypopituitarism and adrenal insufficiency). Administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2; withhold or discontinue for Grade 3 or 4 hypophysitis.

KEYTRUDA can cause thyroid disorders, including hyperthyroidism, hypothyroidism, and thyroiditis. Hyperthyroidism occurred in 96 (3.4%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.8%) and 3 (0.1%) hyperthyroidism. Hypothyroidism occurred in 237 (8.5%) of 2799 patients receiving KEYTRUDA, including Grade 2 (6.2%) and 3 (0.1%) hypothyroidism. The incidence of new or worsening hypothyroidism was higher in patients with HNSCC, occurring in 28 (15%) of 192 patients with HNSCC, including Grade 3 (0.5%) hypothyroidism. Thyroiditis occurred in 16 (0.6%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.3%) thyroiditis. Monitor patients for changes in thyroid function (at the start of treatment, periodically during treatment, and as indicated based on clinical evaluation) and for clinical signs and symptoms of thyroid disorders. Administer replacement hormones for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism.

KEYTRUDA can cause type 1 diabetes mellitus, including diabetic ketoacidosis, which have been reported in 6 (0.2%) of 2799 patients. Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 9 (0.3%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 nephritis.

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA (pembrolizumab) and administer corticosteroids. For signs and symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

KEYTRUDA can cause other clinically important immune-mediated adverse reactions. These immune-mediated reactions may occur in any organ system. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barré syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, and partial seizures arising in a patient with inflammatory foci in brain parenchyma. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and postmarketing use.

Solid organ transplant rejection has been reported in postmarketing use of KEYTRUDA. Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment with KEYTRUDA vs the risk of possible organ rejection in these patients.

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 6 (0.2%) of 2799 patients. Monitor patients for signs and symptoms of infusion-related reactions, including rigors, chills, wheezing, pruritus, flushing, rash, hypotension, hypoxemia, and fever. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic hematopoietic stem cell transplantation (HSCT) after being treated with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after treatment with KEYTRUDA on any trial, 6 patients (26%) developed graft-versus-host-disease (GVHD), one of which was fatal, and 2 patients (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning, one of which was fatal. Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptor–blocking antibody before transplantation. These complications may occur despite intervening therapy between PD-1 blockade and allogeneic HSCT. Follow patients closely for early evidence of transplant-related complications such as hyperacute GVHD, severe (Grade 3 to 4) acute GVHD, steroid-requiring febrile syndrome, hepatic VOD, and other immune-mediated adverse reactions, and intervene promptly.

Based on its mechanism of action, KEYTRUDA (pembrolizumab) can cause fetal harm when administered to a pregnant woman. If used during pregnancy, or if the patient becomes pregnant during treatment, apprise the patient of the potential hazard to a fetus. Advise females of reproductive potential to use highly effective contraception during treatment and for 4 months after the last dose of KEYTRUDA.

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). Adverse reactions leading to interruption of KEYTRUDA occurred in 21% of patients; the most common (≥1%) was diarrhea (2.5%). The most common adverse reactions with KEYTRUDA vs ipilimumab were fatigue (28% vs 28%), diarrhea (26% with KEYTRUDA), rash (24% vs 23%), and nausea (21% with KEYTRUDA). Corresponding incidence rates are listed for ipilimumab only for those adverse reactions that occurred at the same or lower rate than with KEYTRUDA.

KEYTRUDA monotherapy was discontinued due to adverse reactions in 8% of 682

patients with metastatic NSCLC. The most common adverse event resulting in permanent discontinuation of KEYTRUDA was pneumonitis (1.8%). Adverse reactions leading to interruption of KEYTRUDA occurred in 23% of patients; the most common (≥1%) were diarrhea (1%), fatigue (1.3%), pneumonia (1%), liver enzyme elevation (1.2%), decreased appetite (1.3%), and pneumonitis (1%). The most common adverse reactions (occurring in at least 20% of patients and at a higher incidence than with docetaxel) were decreased appetite (25% vs 23%), dyspnea (23% vs 20%), and nausea (20% vs 18%).

When KEYTRUDA was administered in combination with carboplatin and pemetrexed (carbo/pem), KEYTRUDA was discontinued in 10% of 59 patients. The most common adverse reaction resulting in discontinuation of KEYTRUDA (≥2%) was acute kidney injury (3.4%). Adverse reactions leading to interruption of KEYTRUDA occurred in 39% of patients; the most common (≥2%) were fatigue (8%), neutrophil count decreased (8%), anemia (5%), dyspnea (3.4%), and pneumonitis (3.4%).The most common adverse reactions (≥20%) with KEYTRUDA compared to carbo/pem alone were fatigue (71% vs 50%), nausea (68% vs 56%), constipation (51% vs 37%), rash (42% vs 21%), vomiting (39% vs 27%), dyspnea (39% vs 21%), diarrhea (37% vs 23%), decreased appetite (31% vs 23%), headache (31% vs 16%), cough (24% vs 18%), dizziness (24% vs 16%), insomnia (24% vs 15%), pruritus (24% vs 4.8%), peripheral edema (22% vs 18%), dysgeusia (20% vs 11%), alopecia (20% vs 3.2%), upper respiratory tract infection (20% vs 3.2%), and arthralgia (15% vs 24%). This study was not designed to demonstrate a statistically significant difference in adverse reaction rates for KEYTRUDA (pembrolizumab) as compared to carbo/pem alone for any specified adverse reaction.

KEYTRUDA was discontinued due to adverse reactions in 17% of 192 patients with HNSCC. Serious adverse reactions occurred in 45% of patients. The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia, dyspnea, confusional state, vomiting, pleural effusion, and respiratory failure. The most common adverse reactions (reported in at least 20% of patients) were fatigue, decreased appetite, and dyspnea. Adverse reactions occurring in patients with HNSCC were generally similar to those occurring in patients with melanoma or NSCLC, with the exception of increased incidences of facial edema (10% all Grades; 2.1% Grades 3 or 4) and new or worsening hypothyroidism.

KEYTRUDA was discontinued due to adverse reactions in 5% of 210 patients with cHL, and treatment was interrupted due to adverse reactions in 26% of patients. Fifteen percent (15%) of patients had an adverse reaction requiring systemic corticosteroid therapy. Serious adverse reactions occurred in 16% of patients. The most frequent serious adverse reactions (≥1%) included pneumonia, pneumonitis, pyrexia, dyspnea, GVHD, and herpes zoster. Two patients died from causes other than disease progression; one from GVHD after subsequent allogeneic HSCT and one from septic shock. The most common adverse reactions (occurring in ≥20% of patients) were fatigue (26%), pyrexia (24%), cough (24%), musculoskeletal pain (21%), diarrhea (20%), and rash (20%).

In KEYNOTE-052, KEYTRUDA was discontinued due to adverse reactions in 11% of 370 patients with locally advanced or metastatic urothelial carcinoma. The most common adverse reactions (in≥20% of patients) were fatigue (38%), musculoskeletal pain (24%), decreased appetite (22%), constipation (21%), rash (21%), and diarrhea (20%). Eighteen patients (5%) died from causes other than disease progression. Five patients (1.4%) who were treated with KEYTRUDA experienced sepsis which led to death, and 3 patients (0.8%) experienced pneumonia which led to death. Adverse reactions leading to interruption of KEYTRUDA occurred in 22% of patients; the most common (≥1%) were liver enzyme increase, diarrhea, urinary tract infection, acute kidney injury, fatigue, joint pain, and pneumonia. Serious adverse reactions occurred in 42% of patients, the most frequent (≥2%) of which were urinary tract infection, hematuria, acute kidney injury, pneumonia, and urosepsis.

In KEYNOTE-045, KEYTRUDA was discontinued due to adverse reactions in 8% of 266 patients with locally advanced or metastatic urothelial carcinoma. The most common adverse reaction resulting in permanent discontinuation of KEYTRUDA (pembrolizumab) was pneumonitis (1.9%). Adverse reactions leading to interruption of KEYTRUDA occurred in 20% of patients; the most common (≥1%) were urinary tract infection (1.5%), diarrhea (1.5%), and colitis (1.1%). The most common adverse reactions (20%) in patients who received KEYTRUDA vs those who received chemotherapy were fatigue (38% vs 56%), musculoskeletal pain (32% vs 27%), pruritus (23% vs 6%), decreased appetite (21% vs 21%), nausea (21% vs 29%), and rash (20% vs 13%). Serious adverse reactions occurred in 39% of KEYTRUDA-treated patients, the most frequent (≥2%) of which were urinary tract infection, pneumonia, anemia, and pneumonitis.

There is limited experience in pediatric patients. Efficacy for pediatric patients was extrapolated from the results in the adult cHL population. In a study of 40 pediatric patients with advanced melanoma, PD-L1–positive advanced, relapsed, or refractory solid tumors or lymphoma, patients were treated with KEYTRUDA for a median of 43 days (range 1-414 days), with 24 patients (60%) receiving treatment for 42 days or more. The safety profile in pediatric patients was similar to that seen in adults treated with KEYTRUDA. Toxicities that occurred at a higher rate (≥15% difference) in these patients when compared to adults under 65 years of age were fatigue (45%), vomiting (38%), abdominal pain (28%), hypertransaminasemia (28%), and hyponatremia (18%).

It is not known whether KEYTRUDA is excreted in human milk. Because many drugs are excreted in human milk, instruct women to discontinue nursing during treatment with KEYTRUDA and for 4 months after the final dose.

AstraZeneca’s Tagrisso shows potential as a new standard of care in 1st-line EGFR-mutated lung cancer at ESMO 2017 Congress

On September 8, 2017 AstraZeneca reported it has presented the full results of the Phase III FLAURA trial, which support Tagrisso’s (osimertinib) clear potential as a new standard of care (SoC) in the 1st-line treatment of adult patients with locally-advanced or metastatic epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC) (Press release, AstraZeneca, SEP 8, 2017, View Source [SID1234520438]).

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Results of the Phase III FLAURA trial were included at the Presidential Symposium I of the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress in Madrid, Spain, and demonstrate a superior, clinically-meaningful PFS advantage with Tagrisso compared with current SoC EGFR-TKIs (erlotinib or gefitinib).

Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca, said: "The FLAURA data are truly exciting. Until now, even with the therapeutic advances offered by the first- and second-generation EGFR inhibitors, less than 20% of EGFR mutation-positive NSCLC patients survive for five years. The FLAURA data suggest early and sustained benefit with Tagrisso that has the potential to significantly impact long-term patient outcomes and help address the considerable unmet need that remains."

Dr. Suresh S. Ramalingam, Principal Investigator of the FLAURA trial, from the Winship Cancer Institute of Emory University, Atlanta, USA, said: "The FLAURA data for osimertinib are likely to result in a major paradigm shift in the treatment of patients with EGFR mutation-positive advanced lung cancer. Not only did the trial demonstrate a robust improvement in efficacy with osimertinib when compared to other commonly-used EGFR inhibitors, the side effects profile was also more favourable with osimertinib".

Summary of key efficacy results:

Endpoint
Tagrisso
SoC
Hazard ratio (HR)/
Odds ratio (OR)
PFS
(primary endpoint)
18.9 months (median)
10.2 months (median)
HR 0.46
95% CI, 0.37-0.57, p<0.0001
OS at 25% maturity
N/A
N/A
HR 0.63
95% CI, 0.45-0.88, p=0.0068*
Duration of Response (DoR)
17.2 months (median)
8.5 months
(median)
N/A
Objective Response Rate (ORR)
80%
76%
OR 1.28
0.85-1.93, p=0.2335
*0.0015 was the threshold required for statistical significance at the current level of maturity. A final OS analysis is planned at a later stage.

Additional highlights from the FLAURA data include:

Superior progression-free survival (PFS): Patients on Tagrisso had less than half the risk of progression or death compared with patients on erlotinib or gefitinib (hazard ratio [HR] 0.46; 95% confidence interval [CI] 0.37-0.57; p<0.0001). The median PFS was 18.9 months for patients on Tagrisso vs.10.2 months for patients in the comparator arm.
Clinically-meaningful preliminary overall survival (OS) data at 25% maturity: The hazard ratio for OS was 0.63 (95% CI: 0.45-0.88; p=0.0068) favouring Tagrisso. Overall survival data were 25% mature at the time of the interim analysis (21% of the patients on Tagrisso had died and 30% of the patients on the comparator arm had died). The p-value of 0.0068 was not below the threshold of 0.0015 required for statistical significance at the current level of maturity. A final OS analysis is planned at a later stage.
PFS improvements consistent across subgroups: Improvements in PFS with Tagrisso were consistent across all pre-specified patient subgroups, with at least a 40% reduction in the risk of progression or death, including in patients with/without central nervous system (CNS) metastases at study entry, Asian/non-Asian patients, patients with/without prior smoking history, and patients with exon 19 deletion/L858R.
Impressive duration of response (DoR) and objective response rate (ORR): Patients treated with Tagrisso had more than double the median DoR than those on the comparator arm (17.2 months vs. 8.5 months), and an ORR (a measurement of tumour shrinkage) of 80% vs. 76% with the comparator arm (odds ratio 1.28 [0.85-1.93], p=0.2335).
The FLAURA safety data for Tagrisso were in line with those observed in prior clinical trials, with a low rate of Grade ≥3 adverse events (AEs). In patients treated with Tagrisso, the most common AEs were diarrhoea (58% [2% Grade ≥3]) and dry skin (32% [<1% Grade ≥3]), and in the comparator arm group, the most common AEs were diarrhoea (57% [3% Grade ≥3]) and dermatitis acneiform (48% [5% Grade ≥3]). Of the patients on Tagrisso, 33.7% had a Grade ≥3 AE, compared with 44.8% in the comparator arm, and 13.3% of patients on Tagrisso had an AE leading to treatment discontinuation compared with 18.1% in the comparator arm.

AstraZeneca is in discussions with global health authorities regarding regulatory submissions for Tagrisso based on the FLAURA data. A status of regulatory submissions is usually provided with the Company’s quarterly results announcement.

Tagrisso is currently approved in more than 50 countries, including the US, EU, Japan and China, as 2nd-line treatment for patients with advanced NSCLC who progress following treatment with an EGFR-TKI due to the EGFR T790M resistance mutation.

NOTES TO EDITORS

About EGFR-mutated NSCLC

Lung cancer is the leading cause of cancer death among both men and women, accounting for about one-quarter of all cancer deaths, more than breast, prostate and colorectal cancers combined. Approximately 10-15% of patients in the US and Europe, and 30-40% of patients in Asia have EGFRm NSCLC. These patients are particularly sensitive to treatment with currently-available EGFR-TKIs, which block the cell signalling pathways that drive the growth of tumour cells. However, tumours almost always develop resistance to EGFR-TKI treatment, leading to disease progression. Approximately half of patients develop resistance to approved EGFR-TKIs such as gefitinib and erlotinib due to the resistance mutation, EGFR T790M. Tagrisso also targets this secondary mutation that leads to disease progression. There is also a need for agents with improved CNS efficacy, since approximately 25% of patients with EGFRm NSCLC have brain metastases at diagnosis, increasing to approximately 40% within two years of diagnosis.

About FLAURA

FLAURA assessed the efficacy and safety of Tagrisso 80mg orally once daily vs. standard-of-care EGFR-TKIs (either erlotinib [150mg orally, once daily] or gefitinib [250mg orally, once daily]) in previously-untreated patients with locally-advanced or metastatic EGFRm NSCLC. The trial was a double-blinded, randomised study, with 556 patients across 30 countries.

The primary endpoint of the trial was PFS, and secondary endpoints included OS, ORR, DOR, disease control rate (DCR), safety, and measures of health-related quality of life (HRQoL).

About Tagrisso

Tagrisso is a third-generation, irreversible EGFR tyrosine kinase inhibitor (TKI) designed to inhibit both EGFR sensitising and EGFR T790M resistance mutations, with clinical activity against central nervous system (CNS) metastases. Tagrisso (osimertinib) 40mg and 80mg once-daily oral tablets have been approved in more than 50 countries, including the US, EU, Japan and China, for patients with EGFR T790M mutation-positive advanced NSCLC. Tagrisso is also being investigated in the adjuvant and metastatic 1st-line settings, including in patients with and without CNS metastases, in leptomeningeal metastases, and in combination with other treatments.

About AstraZeneca in Lung Cancer

AstraZeneca is committed to developing therapies to help every patient with lung cancer. We have two approved therapies and a growing pipeline that targets genetic changes in tumour cells and boosts the power of the immune response against cancer. Our unrelenting pursuit of science aims to deliver more breakthrough therapies with the goal of extending and improving the lives of patients across all stages of disease and lines of therapy.

AstraZeneca presents superior progression-free survival for Imfinzi in the PACIFIC trial of patients with locally-advanced unresectable lung cancer at ESMO 2017 Congress

On September 8, 2017 AstraZeneca and MedImmune, its global biologics research and development arm, reported that they have presented the full PFS data from a planned interim analysis of the Phase III PACIFIC trial (Press release, AstraZeneca, SEP 8, 2017, View Source [SID1234520437]). Results show that Imfinzi (durvalumab) demonstrated a statistically-significant and clinically-meaningful improvement in PFS compared to current standard of care with active surveillance in patients with locally-advanced (Stage III), unresectable non-small cell lung cancer (NSCLC) who had not progressed following standard platinum-based chemotherapy concurrent with radiation therapy (CRT).

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Results of the Phase III PACIFIC trial, included at the Presidential Symposium I of the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress in Madrid, Spain, show an improvement in PFS of more than 11 months in patients treated with Imfinzi compared to placebo (full details in table below). The PFS improvement with Imfinzi was observed across all pre-specified subgroups, including PD-L1 expression status. Patients receiving Imfinzi also had a lower incidence of metastases than those receiving placebo. The PACIFIC trial continues to evaluate overall survival (OS), the other primary endpoint. Detailed results of the PACIFIC trial are published online in the New England Journal of Medicine.

Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca, said: "The Phase III PACIFIC results are incredibly encouraging for a patient population that until now has been without treatment options. As the first Immuno-Oncology medicine to achieve improvement in progression-free survival in this setting, Imfinzi is showing clear potential to become a new standard of care for patients with locally-advanced, unresectable NSCLC who have not progressed following chemoradiation."

Dr. Luis Paz-Ares, Principal Investigator of the PACIFIC trial, from the Hospital Universitario Doce de Octubre, Madrid, Spain, said: "For patients with locally-advanced unresectable NSCLC who have completed chemoradiation therapy, Imfinzi represents a potential new treatment option in the context of clear unmet clinical need. Durvalumab overtly prolongs the period in which the disease is controlled with reasonable side effects. In addition, it offers hope to increase the cure rate in this setting, but more mature follow-up is needed to assess its impact on survival."

Summary of key efficacy results:

Endpoint
Medicine
Value
Hazard ratio (HR)/
Confidence interval (CI)
PFS*
(first primary endpoint)

Imfinzi
16.8 months (median)
HR 0.52
95% CI, 0.42-0.65, p<0.0001
Placebo
5.6 months
(median)
Duration of response (DoR)
Imfinzi
Not reached
N/A
Placebo
13.8 months
Objective Response Rate (ORR) as measured from baseline scan post-CRT completion
Imfinzi
28.4%
95% CI, 24.28-32.89, p<0.001
Placebo
16.0%
95% CI, 11.31-21.59, p<0.001
* Time from randomisation to the first documented tumour progression, or death in the absence of progression. Randomisation in the PACIFIC trial occurred up to 6 weeks after completion of concurrent chemoradiation therapy (cCRT) and cCRT typically lasted at least 6 weeks. If the PFS had been measured prior to cCRT, it would add approximately 3 months or longer to the PFS value for each arm.

Among patients receiving Imfinzi, the most frequent treatment-related adverse events (AEs) vs. placebo were cough (35.4% vs 25.2%), pneumonitis/radiation pneumonitis (33.9% vs 24.8%), fatigue (23.8% vs 20.5%), dyspnoea (22.3% vs 23.9%) and diarrhoea (18.3% vs 18.8%). 29.9% of patients experienced a grade 3 or 4 AE vs. 26.1% for placebo, and 15.4% of patients discontinued treatment due to AEs compared to 9.8% of patients on placebo.

On 31 July 2017, Imfinzi received Breakthrough Therapy Designation from the US Food and Drug Administration (FDA) as a potential treatment for patients with locally advanced, unresectable NSCLC whose disease has not progressed following platinum-based chemoradiation therapy.

AstraZeneca is in discussions with global health authorities regarding regulatory submissions for Imfinzi based on the PACIFIC data. A status of regulatory submissions is usually provided with the Company’s quarterly results announcement.

Imfinzi received accelerated approval from the US Food and Drug Administration for previously treated patients with advanced bladder cancer and is under review in Canada and Australia for similar use.

NOTES TO EDITORS

About Locally Advanced (Stage III) NSCLC

Stage III lung cancer is divided into two stages (IIIA and IIIB), which are defined by how much the cancer has spread locally and the possibility of surgery. This differentiates it from Stage IV disease, when the cancer has spread (metastasised) to other organs.

Stage III lung cancer represents approximately one-third of NSCLC incidence and was estimated to affect around 105,000 patients in the G7 countries in 2016. More than half of these patients have tumours that are unresectable. The current standard of care is chemotherapy and radiation followed by active surveillance to monitor for progression. The prognosis remains poor and long-term survival rates are low.

About PACIFIC

The PACIFIC trial is a randomised, double-blinded, placebo-controlled, multi-centre trial of Imfinzi as sequential treatment in unselected patients with locally-advanced, unresectable (Stage III) NSCLC who have not progressed following platinum-based chemotherapy concurrent with radiation therapy.

The trial is being conducted in 235 centres across 26 countries involving approximately 700 patients. The primary endpoints of the trial are progression-free survival (PFS) and overall survival (OS), and secondary endpoints include landmark PFS and OS, objective response rate (ORR) and duration of response.

About Imfinzi

Imfinzi (durvalumab), a human monoclonal antibody directed against PD-L1, blocks PD-L1 interaction with PD-1 and CD80 on T cells, countering the tumour’s immune-evading tactics and inducing an immune response.

Imfinzi continues to be studied in multiple monotherapy trials and combination trials with tremelimumab and other potential new medicines in Immuno-Oncology. Imfinzi is being assessed in Phase III trials as a monotherapy in various stages of NSCLC, in small-cell lung cancer (SCLC), in metastatic urothelial cancer (mUC) and in head and neck squamous cell carcinoma (HNSCC). The combination of Imfinzi and tremelimumab is being assessed in Phase III trials in NSCLC, SCLC, mUC and HNSCC and in Phase I/II trials in hepatocellular carcinoma and haematological malignancies.