TAGRISSO® (osimertinib) granted Breakthrough Therapy Designation by US FDA for the 1st-line treatment of patients with EGFR-mutation positive non-small cell lung cancer

On October 9, 2017 AstraZeneca reported that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for TAGRISSO (osimertinib) for the 1st-line treatment of patients with metastatic epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) (Press release, AstraZeneca
, OCT 9, 2017, View Source [SID1234520816]).

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Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca, said: "The Breakthrough Designation acknowledges not only TAGRISSO’s potential as a 1st-line standard of care in advanced EGFR mutation-positive NSCLC, but also the significant need for improved clinical outcomes in this disease. The results of the FLAURA trial have the potential to redefine clinical expectations and offer new hope for patients who currently have a poor prognosis."

The FDA granted the BTD based on data from the Phase III FLAURA trial of osimertinib versus standard-of-care EGFR tyrosine kinase inhibitor (TKI) therapy in previously-untreated patients with locally-advanced or metastatic EGFR mutation-positive NSCLC. In the trial, median progression-free survival was 18.9 months for osimertinib compared with 10.2 months for EGFR-TKIs (erlotinib or gefitinib). Improvements were seen in all pre-specified subgroups, including patients with and without brain metastases.

In the FLAURA trial, the safety profile of osimertinib was consistent with previous experience. In patients treated with osimertinib, the most common AEs were diarrhea (58%, any grade [2% Grade ≥3]) and dry skin (32%, any grade [<1% Grade ≥3]), and in the comparator arm group the most common AEs were diarrhea (57%, any grade [2% Grade ≥3]) and dermatitis acneiform (48%, any grade [5% Grade ≥3]). Of the patients on osimertinib, 34% had a Grade ≥3 AE, compared to 45% in the comparator arm, and 13% of patients on osimertinib had an AE leading to treatment discontinuation compared to 18% in the comparator arm.

On September 28, 2017, the US NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) were updated to include the use of osimertinib in the 1st-line treatment of patients with locally-advanced or metastatic EGFR mutation-positive NSCLC. The use of osimertinib for the first-line treatment of patients with locally-advanced or metastatic EGFR mutation-positive NSCLC is not yet FDA approved.

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. TAGRISSO once-daily tablets are approved by the FDA for the treatment of patients with metastatic EGFR T790M mutation-positive NSCLC, as detected by an FDA-approved test, whose disease has progressed on or after an EGFR TKI therapy. TAGRISSO is the first and only approved medicine in the US indicated for NSCLC patients who have tested positive for the EGFR T790M mutation.

This is the sixth BTD that AstraZeneca has received from the FDA for an oncology medicine since 2014. BTD is designed to expedite the development and regulatory review of new medicines that are intended to treat a serious condition and that have shown encouraging early clinical results, which demonstrate substantial improvement on a clinically- significant endpoint over available medicines and when there is significant unmet medical need.

TAGRISSO (osimertinib) Important Safety Information

There are no contraindications for TAGRISSO
Interstitial Lung Disease (ILD)/Pneumonitis occurred in 3.5% and was fatal in 0.6% of 833 TAGRISSO-treated patients. Withhold TAGRISSO and promptly investigate for ILD in patients who present with worsening of respiratory symptoms indicative of ILD (e.g., dyspnea, cough, and fever). Permanently discontinue TAGRISSO if ILD is confirmed
Heart rate-corrected QT (QTc) interval prolongation occurred in TAGRISSO-treated patients. Of the 833 TAGRISSO-treated patients, 0.7% of patients were found to have a QTc > 500 msec, and 2.9% of patients had an increase from baseline QTc > 60 msec. No QTc-related arrhythmias were reported. Conduct periodic monitoring with ECGs and electrolytes in patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval. Permanently discontinue TAGRISSO in patients who develop QTc interval prolongation with signs/symptoms of life-threatening arrhythmia
Cardiomyopathy occurred in 1.9% and was fatal in 0.1% of 833 TAGRISSO-treated patients. Left Ventricular Ejection Fraction (LVEF) decline ≥ 10% and a drop to < 50% occurred in 4% of 655 TAGRISSO-treated patients. Conduct cardiac monitoring, including an assessment of LVEF at baseline and during treatment in patients with cardiac risk factors. Assess LVEF in patients who develop relevant cardiac signs or symptoms during treatment. For symptomatic congestive heart failure or persistent, asymptomatic LV dysfunction that does not resolve within 4 weeks, permanently discontinue TAGRISSO
Keratitis was reported in 0.7% of 833 TAGRISSO-treated patients in clinical trials. Promptly refer patients with signs and symptoms suggestive of keratitis (such as eye inflammation, lacrimation, light sensitivity, blurred vision, eye pain, and/or red eye) to an ophthalmologist
Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during TAGRISSO treatment and for 6 weeks after the final dose. Advise males with female partners of reproductive potential to use effective contraception for 4 months after the final dose
The most common adverse reactions (≥20%) in patients treated with TAGRISSO were diarrhea (41%), rash (34%), dry skin (23%), nail toxicity (22%), and fatigue (22%)
Please see complete Prescribing Information including Patient Information.

NOTES TO EDITORS

About Non-Small Cell Lung Cancer (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% to 15% of patients in the US and Europe, and 30% to 40% of patients in Asia have epidermal growth factor receptor mutation-positive (EGFRm) NSCLC. These patients are particularly sensitive to treatment with currently-available EGFR tyrosine kinase inhibitors (TKIs), which block the cell signaling pathways that drive the growth of tumor cells. However, tumors 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 targets this secondary mutation that leads to disease progression. There is also a need for agents with improved central nervous system efficacy since approximately 25% of patients with EGFRm NSCLC have brain metastases at first diagnosis, increasing to approximately 40% within two years of diagnosis.

About TAGRISSO (osimertinib)
TAGRISSO (osimertinib) is a third-generation, irreversible epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) designed to inhibit both EGFR sensitizing and EGFR T790M resistance mutations, with clinical activity against central nervous system (CNS) metastases. TAGRISSO 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 non-small cell lung cancer. Eligibility for treatment with TAGRISSO is dependent on confirmation that the EGFR T790M mutation is present in the tumor.

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 FLAURA
FLAURA assessed the efficacy and safety of osimertinib 80mg orally once daily vs. standard-of-care epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (TKIs) (either erlotinib [150mg orally, once daily] or gefitinib [250mg orally, once daily]) in previously untreated patients with locally advanced or metastatic EGFR mutation-positive non-small cell lung cancer. The trial was a double-blinded, randomized study, with 556 patients across 30 countries.

The primary endpoint of the trial was progression-free survival, and secondary endpoints included overall survival, objective response rate, duration of response, disease control rate, safety and measures of health-related quality of life.

PPD Wins Best CRO at World ADC Awards for Advancing Cancer Research

On October 9, 2017 Pharmaceutical Product Development, LLC (PPD), a leading global contract research organization (CRO), reported it was named Best CRO Providerat the World ADC Awards (Press release, PPD, OCT 8, 2017, View Source [SID1234520813]). This recognition acknowledges the excellence and dedication of PPD Laboratories professionals in supporting clients’ antibody-drug conjugate (ADC) research to develop new anticancer therapies.

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ADCs are complex biotherapeutic molecules, combining monoclonal antibodies with an attached payload of highly potent cytotoxic drugs. They are designed to target and destroy cancer tumor cells with high specificity and efficiency, while minimally impacting healthy cells. To date, PPD Laboratories’ experience includes more than 100 different ADC molecules.

PPD Laboratories has supported numerous ADC development programs since 2009, ranging from early development projects to preclinical good laboratory practice (GLP) and human clinical trials.

"This award is evidence that PPD Laboratories’ employees are at the leading edge of ADC research and are driven to help clients succeed in combating cancer," said Chris Fikry, M.D., executive vice president of PPD Laboratories for PPD. "In the past five years, the bioanalytical lab has completed more than 5,000 studies, providing vital pharmacokinetic, pharmacodynamic and immunogenicity data to support our clients’ efforts to develop new medicines to improve health."

PPD Laboratories’ scientists have extensive experience in bioanalytical testing utilizing diverse measurement technologies, including immunochemistry, chromatography-mass spectrometry, cell-based assays, flow cytometry and molecular genomics. PPD’s expertise includes small molecules, biologics, vaccines and biomarkers across a wide variety of therapeutic areas. In oncology, our largest therapeutic area, PPD Laboratories helped develop all of 2016’s top 20 cancer therapies.

The fourth annual World ADC Awards, which recognize distinction within ADC research, were held Sept. 21 in conjunction with the eighth annual World ADC San Diego Conference.

Incyte and Cancer Support Community Delaware Announce the Establishment of the Incyte Cancer Care Assistance Fund for Delaware

On October 6, 2017– Incyte Corporation (Nasdaq:INCY) and Cancer Support Community Delaware (CSCDE) reported the establishment of the Incyte Cancer Care Assistance Fund for Delaware which will provide emergency financial assistance for cancer patients, their caregivers and family members living in Delaware (Press release, Incyte, OCT 6, 2017, View Source;p=RssLanding&cat=news&id=2305148 [SID1234520802]).

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Through the Incyte Charitable Giving Foundation, Incyte has committed to providing a maximum of $100,000 each year to CSCDE over the next 5 years, beginning in 2018.

"We are very pleased to announce the creation of the Incyte Cancer Care Assistance Fund for Delaware with Cancer Support Community Delaware, an organization which, like Incyte, is dedicated to supporting patients with cancer and the local community," said Paula Swain, Executive Vice President of Human Resources and Chair of The Incyte Charitable Giving Foundation. "Today, as we celebrate the opening of our newly expanded headquarters in Wilmington, we reaffirm our commitment to Delaware and to the well-being of the communities we serve."
The fund will be administered by Cancer Support Community Delaware. Additional details about the Incyte Cancer Care Assistance Fund for Delaware will be made available in January 2018.

"Through the establishment of this fund, we aim to address the emergent needs that patients with cancer in Delaware face as they navigate their disease and its impact across all aspects of their lives," said Nicole Topkis Pickles, Executive Director of Cancer Support Community Delaware.

ERYTECH Files Registration Statement for Proposed Initial Public Offering in the United States

On October 6, 2017 ERYTECH Pharma (Paris:ERYP) (ADR:EYRYY) (Euronext Paris: ERYP) ("ERYTECH"), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported that it has filed a registration statement on Form F-1 with the U.S. Securities and Exchange Commission ("SEC") relating to a proposed initial public offering of its American Depositary Shares ("ADSs"), each representing one ordinary share, in the United States and a concurrent private placement of its ordinary shares in Europe and other countries outside of the United States and Canada (together, the "Global Offering") (Press release, ERYtech Pharma, OCT 6, 2017, View Source;p=RssLanding&cat=news&id=2305243 [SID1234520826]). All securities to be sold in the Global Offering will be offered by ERYTECH. The number of securities to be sold and the price range for the proposed Global Offering have not yet been determined. ERYTECH has applied to list its ADSs on the NASDAQ Global Market under the ticker symbol "ERYP." The ordinary shares are listed on Euronext Paris under the symbol "ERYP."

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Jefferies LLC is acting as global coordinator and joint book-runner for the Global Offering. Cowen and Company, LLC is acting as joint book-runner and JMP Securities LLC is acting as lead manager for the offering of ADSs in the United States. Oddo BHF SCA is acting as joint book-runner for the offering of the Company’s ordinary shares in Europe.

The securities referred to in this press release will be offered only by means of a prospectus. When available, copies of the preliminary prospectus relating to and describing the terms of the Global Offering may be obtained from Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at (877) 821-7388, or by email at [email protected]; or from Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, or by telephone at (631) 274-2806.

A registration statement relating to the securities referred to herein has been filed with the SEC but has not yet become effective. These securities may not be sold, nor may offers to buy be accepted, prior to the time the registration statement becomes effective. This press release does not constitute an offer to sell or the solicitation of an offer to buy securities in any jurisdiction, and shall not constitute an offer, solicitation or sale in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that jurisdiction.

Apogenix Granted Orphan Designation by the European Commission for
Asunercept to Treat Myelodysplastic Syndromes

On October 5, 2017 Apogenix AG, a biopharmaceutical company developing next-generation immuno-oncology therapeutics, reported that its lead product candidate, asunercept (APG101), has been granted orphan designation from the European Commission (EC) for the treatment of myelodysplastic syndromes (MDS) (Press release, Apogenix, OCT 5, 2017, View Source [SID1234524529]). MDS is a bone marrow disorder characterized by ineffective hematopoiesis (blood cell formation) and can lead to severe anemia. Patients often suffer from life-threatening infections and are at risk of developing acute myeloid leukemia.

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Orphan designation includes access to a centralized marketing authorization procedure for the European Union, ten years of protection from market competition with similar medicines in similar indications and fee reductions for consultations with the EMA. Earlier, asunercept received Orphan Drug Designation for MDS from the US Food and Drug Administration (FDA).

Dr. Harald Fricke, Chief Medical Officer of Apogenix, commented: "The vast majority of patients suffering from MDS are anemic and dependent on frequent regular blood transfusions. Asunercept prevents premature death of red blood cells in the bone marrow and thus reduces the need of blood transfusions, even making them superfluous in many patients. We are highly encouraged by the data from our clinical phase I trial with asunercept in these patients and are currently preparing to initiate a clinical phase II proof-of-concept trial to further evaluate the efficacy of asunercept in MDS."

Asunercept has been evaluated in an open label, single-arm phase I clinical trial in 20 patients with low to intermediate risk MDS, in which treatment with asunercept was well tolerated and led to a significant decrease in transfusion frequency. In addition, investigation of parameters involved in erythropoiesis delineated how asunercept stimulates the production of red blood cells in these patients.

Asunercept binds to the CD95 ligand (CD95L) and blocks the activation of the CD95 receptor. Excessive stimulation of the CD95 receptor on hematopoietic precursor cells in the bone marrow of MDS patients inhibits erythropoiesis. As a result, transfusion-dependent anemia develops, which is refractory to erythropoiesis-stimulating agents. Treatment with asunercept, which inhibits the CD95 system, addresses this major cause of the disorder.

About Myelodysplastic Syndromes (MDS)
MDS is a bone marrow disorder that is characterized by ineffective hematopoiesis and can lead to severe anemia. In most cases, the anemia is treated with blood transfusions that eventually result in an iron overload, which can damage the liver and other organs. At the same time, the number of thrombocytes that are responsible for coagulation and the number of leucocytes that are responsible for immune defense significantly decreases in patients with this disorder. As a result, MDS patients frequently suffer from sudden bleeding and life-threatening infections. In addition, they are at risk of developing acute myeloid leukemia, a type of blood cancer.

About asunercept (APG101)
Apogenix’ lead immuno-oncology candidate asunercept is a fully human fusion protein that consists of the extracellular domain of the CD95-receptor and the Fc domain of an IgG1 antibody. Asunercept is being developed for the treatment of solid tumors and malignant hematological diseases. The World Health Organization (WHO) has assigned the international nonproprietary name (INN) "asunercept" for APG101.