On October 16, 2019 ArQule, Inc. (Nasdaq: ARQL) reported it will report financial results for the third quarter of 2019 before the market opens on Wednesday, October 30, 2019 (Press release, ArQule, OCT 16, 2019, View Source [SID1234542297]). The Company will hold a conference call and webcast on the same day at 9:00 a.m. ET to discuss these results and provide a general business update.

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The live webcast can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events & Presentations." You may also listen to the call by dialing (877) 868-1831 within the U.S. or (914) 495-8595 outside the U.S. and entering the conference ID: 4289763. A replay will be available two hours after the completion of the call and can be accessed in the "Investors & Media" section of our website, www.arqule.com, under "Events and Presentations."

On October 16, 2019 Novocure (NASDAQ: NVCR) reported that results from the STELLAR trial were published in The Lancet Oncology (Press release, NovoCure, OCT 16, 2019, View Source [SID1234542313]). The STELLAR trial was a prospective, single-arm trial including 80 patients that studied the use of Tumor Treating Fields, delivered via the NovoTTF-100L System, in combination with pemetrexed plus cisplatin/carboplatin as a first-line treatment for patients with unresectable, locally advanced or metastatic malignant pleural mesothelioma (MPM).

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Data showed a median overall survival of 18.2 months (95 percent CI, 12.1 months-25.8 months) for patients treated with NovoTTF-100L and pemetrexed plus cisplatin or carboplatin. One- and two-year survival rates were 62.2 percent (95 percent CI, 50.3 percent-72.0 percent) and 41.9 percent (95 percent CI, 28.0 percent-55.2 percent), respectively. No serious systemic adverse events were considered to be related to the use of NovoTTF-100L. The most common mild to moderate adverse event was skin irritation beneath the transducer arrays.

"The STELLAR trial demonstrated encouraging overall survival results with no increase in systemic toxicity observed in MPM patients treated with Tumor Treating Fields and standard chemotherapy," said Giovanni Luca Ceresoli, MD, Head of Pulmonary Oncology at the Humanitas Gavazzeni Hospital in Bergamo, Italy, and Principal Investigator in the STELLAR trial. "The median overall survival of 18.2 months is impressive given that MPM is a tumor with a dismal prognosis and few effective therapeutic options."

"Mesothelioma is an aggressive cancer that is extremely difficult to treat," said Rupesh Kotecha, MD, Chief of Radiosurgery, Radiation Oncology, Miami Cancer Institute in Florida. "With such limited treatment options, I’m encouraged by the FDA approval of NovoTTF-100L, adding another therapy with the potential to improve outcomes for people with unresectable MPM."

Median progression free survival was 7.6 months (95 percent CI, 6.7 months-8.6 months) for patients treated with NovoTTF-100L and pemetrexed plus cisplatin or carboplatin. There was a 97 percent disease control rate in patients with at least one follow-up CT scan performed (n=72). 40 percent of patients had a partial response, 57 percent had stable disease and 3 percent had progressive disease.

"MPM patients are in need of better treatment options," said Novocure CEO Asaf Danziger. "We are proud that NovoTTF-100L is the first FDA-approved therapy for MPM in more than 15 years. We hope it is just the beginning of continued innovation for patients with this disease."

About Malignant Pleural Mesothelioma (MPM)

Malignant pleural mesothelioma is a rare thoracic solid tumor cancer that has been strongly linked to asbestos exposure. Mesothelioma has a long latency period of 47.9 years on average for men and 53.3 years on average for women following asbestos exposure. There are approximately 3,000 new cases of mesothelioma annually in the United States. Globally, more than 14,000 people are diagnosed with mesothelioma each year. Global mesothelioma incidence is increasing in countries where asbestos is still in use. The prognosis of mesothelioma patients is very poor.

About STELLAR

STELLAR was a phase 2, prospective, single-arm trial designed to study the safety and efficacy of NovoTTF-100L, a Tumor Treating Fields delivery system, and pemetrexed plus cisplatin or carboplatin as a first-line treatment for 80 patients with unresectable, locally advanced or metastatic malignant pleural mesothelioma (MPM). The trial was designed for patients to receive Tumor Treating Fields at 150 kHz at least 18 hours a day until disease progression. The primary endpoint of the trial was overall survival. Secondary endpoints included objective response rate, progression-free survival and safety.

Eligible patients had no prior chemotherapy or radiation treatment for MPM, were at least 18 years old, had an Eastern Cooperative Oncology Group performance status of 0-1, and had at least one measurable or evaluable lesion according to the modified RESIST criteria. Adequate hepatic, renal and hematological functions were required, as well as a life expectancy of at least 3 months. The study was conducted at 12 sites in Europe.

About the NovoTTF-100L System

NovoTTF-100L is a noninvasive, antimitotic cancer treatment for MPM. NovoTTF-110L delivers Tumor Treating Fields to the region of the tumor.

Tumor Treating Fields is a cancer therapy that uses electric fields tuned to specific frequencies to disrupt cell division, inhibiting tumor growth and causing affected cancer cells to die. Tumor Treating Fields does not stimulate or heat tissue and targets dividing cancer cells of a specific size. Tumor Treating Fields causes minimal damage to healthy cells. Mild to moderate skin irritation is the most common side effect reported. Tumor Treating Fields is approved in certain countries for the treatment of adults with glioblastoma and mesothelioma, two of the most difficult cancer types to treat. The therapy shows promise in multiple solid tumor types – including some of the most aggressive forms of cancer.

Caution: Federal law restricts this device to sale by or on the order of a physician. Humanitarian Device. Authorized by Federal Law for use in the treatment of adult patients with unresectable, locally advanced or metastatic, malignant pleural mesothelioma concurrently with pemetrexed and platinum-based chemotherapy. The effectiveness of this device for this use has not been demonstrated.

Approved Indications

The NovoTTF-100L System is indicated for the treatment of adult patients with unresectable, locally advanced or metastatic, malignant pleural mesothelioma (MPM) to be used concurrently with pemetrexed and platinum-based chemotherapy.

Important Safety Information

Contraindications

Do not use the NovoTTF-100L System in patients with implantable electronic medical devices such as pacemakers or implantable automatic defibrillators, etc. Use of the NovoTTF-100L System together with implanted electronic devices has not been tested and may lead to malfunctioning of the implanted device.

Do not use the NovoTTF-100L System in patients known to be sensitive to conductive hydrogels. Skin contact with the gel used with the NovoTTF-100L System may commonly cause increased redness and itching, and may rarely lead to severe allergic reactions such as shock and respiratory failure.

Warnings and Precautions

The NovoTTF-100L System can only be prescribed by a healthcare provider that has completed the required certification training provided by Novocure.

The most common (≥10%) adverse events involving the NovoTTF-100L System in combination with chemotherapy were anemia, constipation, nausea, asthenia, chest pain, fatigue, medical device site reaction, pruritus, and cough.

Other potential adverse effects associated with the use of the NovoTTF-100L System include: treatment related skin toxicity, allergic reaction to the plaster or to the gel, electrode overheating leading to pain and/or local skin burns, infections at sites of electrode contact with the skin, local warmth and tingling sensation beneath the electrodes, muscle twitching, medical device site reaction and skin breakdown/skin ulcer.

If the patient has an underlying serious skin condition on the chest, evaluate whether this may prevent or temporarily interfere with the NovoTTF-100L System treatment.

Do not prescribe the NovoTTF-100L System for patients that are pregnant, you think might be pregnant or are trying to get pregnant, as the safety and effectiveness of the NovoTTF-100L System in these populations have not been established.

Please visit www.novottf100l.com to see the NovoTTF-100L System Instructions For Use (IFU) for complete information regarding the device’s indications, contraindications, warnings, and precautions.

On October 16, 2019 NOXXON Pharma N.V. (Euronext Growth Paris: ALNOX), a biotechnology company focused on improving cancer treatments by targeting the tumor microenvironment (TME), reported the enrollment and first treatment of a patient with newly diagnosed brain cancer in a phase 1/2 clinical trial (Press release, NOXXON, OCT 16, 2019, View Source [SID1234542276]). The study investigates a combined therapy of increasing doses of the CXCL12 inhibitor, NOX-A12, and external-beam radiotherapy. Within this study, NOX-A12 administration in planned for up to six months. The anticipated mode of action of NOX-A12 is the inhibition of the unwanted influx of bone marrow-derived "repair cells" to the tumor following radiotherapy-induced breakdown of the vasculature in the tumor. These "repair cells" act by replacing the tumor’s blood vessels that were destroyed by the irradiation, which ultimately results in disease recurrence.

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The study is designed to deliver safety and first efficacy data to support the definition of a Recommended Phase 2 Dose (RP2D) for this new treatment approach. In addition, the noninvasive assessment of the changes in tumor vascularization is expected to confirm the predicted mechanism of action for the combination of NOX-A12 and radiotherapy.

"The trial represents a new option for very difficult-to-treat patients. The candidate drug should block the influx of two distinct types of cells that the damaged brain tumor tries to recruit to help repair itself and survive. A plethora of pre-clinical studies as well as a recent clinical trial at Stanford yielded promising results with interventions targeting the CXCL12 pathway. We learnt some lessons from these data, and we are very optimistic that blocking both cell types for a longer duration will be even more effective," commented Dr. Frank Giordano, Interim Director of the Department of Radiation Oncology at the University Medical Center Mannheim.

"Based on promising preclinical data in rat and mouse models, we are excited to evaluate NOX-A12 in this indication. It is a unique and promising approach with the potential to effectively treat brain cancer patients for whom there are currently no optimal therapies. The demand from the clinical community to test this combination has been very strong and we are pleased that the dosing of patients has been initiated. We expect data from the first cohort of patients to be available in mid-2020," added Dr. Jarl Ulf Jungnelius, Chief Medical Officer of NOXXON.

The trial is being conducted at three hospitals in Germany. Up to three escalating doses of NOX-A12 will be administered in combination with standard radiotherapy as a first line treatment to newly diagnosed patients with brain tumors who would not benefit from the current standard of care of chemotherapy and whose tumors cannot be fully resected by surgery. The main objective of the study is to assess the safety and tolerability of this combination. Secondary endpoints include activity of the therapy, assessed through the monitoring of tumor vascularization by MRI scans, progression-free survival, overall survival and rates of response.

On October 16, 2019 Eli Lilly and Company (NYSE: LLY) reported top-line results from its Phase 3 SEQUOIA trial evaluating pegilodecakin plus FOLFOX (folinic acid, 5-FU, oxaliplatin) compared to FOLFOX alone in patients with metastatic pancreatic cancer whose disease had progressed during or following a first-line gemcitabine-containing regimen (Press release, Eli Lilly, OCT 16, 2019, View Source [SID1234542298]). The SEQUOIA trial did not meet its primary endpoint of overall survival.

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The most common Grade 3/4 adverse events occurring at a higher rate (>5% difference) on the pegilodecakin-plus-FOLFOX arm compared to the FOLFOX arm were neutropenia, thrombocytopenia, fatigue and anemia. Detailed efficacy and safety results will be submitted for presentation at a future medical meeting.

Metastatic pancreatic cancer is one of the deadliest major cancers, with just three percent of patients in the U.S. living five years after the cancer is diagnosed. In the U.S., pancreatic cancer is the third leading cause of cancer death and is expected to become the second leading cause of cancer-related death in the next decade.1 Globally, pancreatic cancer is the seventh leading cause of cancer-related death.2

"More than 56,700 Americans – mothers, daughters, fathers, sons, colleagues and friends – will be diagnosed with pancreatic cancer this year alone," said Julie Fleshman, JD, MBA, president and CEO of the Pancreatic Cancer Action Network (PanCAN). "Because this is an aggressive disease and the current scope of treatment options is limited, there remains an urgent need for meaningful solutions to improve outcomes for pancreatic cancer patients."

"Pancreatic cancer has proven to be one of the most difficult tumor types to treat and there have been very few recent treatment advancements in the later-line metastatic setting. We are grateful to the patients, investigators and researchers who participated in the study," said Maura Dickler, M.D., vice president, late phase development, Lilly Oncology. "While we are disappointed by the outcome of the SEQUOIA study, we look forward to the upcoming results in lung cancer, learning from those results and increasing our understanding of pegilodecakin’s novel mechanism of action in cancer immunotherapy."

Lilly gained pegilodecakin with the acquisition of ARMO BioSciences in June 2018. SEQUOIA was initiated by ARMO in March 2017 based on results of the Phase 1/1b IVY study, which evaluated pegilodecakin – used as a single agent and in combination with chemotherapy and with checkpoint inhibitor therapy – across multiple tumor types including pancreatic, non-small cell lung and renal cell cancers. Results from the IVY trial were also the basis for the ongoing Phase 2 CYPRESS 1 and CYPRESS 2 studies of pegilodecakin in combination with checkpoint inhibitors in non-small cell lung cancer (NSCLC). The CYPRESS studies were initiated by ARMO in March 2018 and results, expected in early 2020, will inform future studies of pegilodecakin in NSCLC. For the next stage of pegilodecakin’s clinical development, Lilly is focused on assessing biomarkers and conducting studies in NSCLC and other tumor types including renal cell carcinoma, where the molecule has shown promising activity.

Notes to Editors

About SEQUOIA
SEQUOIA is a global, multi-center, randomized Phase 3 study designed to compare the efficacy and safety of pegilodecakin in combination with FOLFOX (folinic acid, 5-FU, oxaliplatin) compared to FOLFOX alone in participants with metastatic adenocarcinoma of the pancreas whose disease had progressed on one prior gemcitabine-containing regimen. The primary endpoint of the study is overall survival and key secondary endpoints are progression-free survival and objective response rate. SEQUOIA was initiated by ARMO BioSciences in March 2017 and enrolled 567 patients.

About Pancreatic Cancer
Metastatic pancreatic cancer is one of the deadliest major cancers, with just three percent of patients in the U.S. living five years after the cancer is diagnosed. In the U.S., pancreatic cancer is the third leading cause of cancer death and is expected to become the second leading cause of cancer-related death in the next decade.1 Globally, pancreatic cancer is the seventh leading cause of cancer-related death.2

About Pegilodecakin
Pegilodecakin is an immunotherapy which stimulates the body’s natural defenses against cancer and expands tumor-attacking T cells. This class of T cells can infiltrate and destroy cancer cells. Pegilodecakin, a pegylated IL-10, has shown clinical activity as a single agent in renal cancer and promising results in combination with both chemotherapy and checkpoint inhibitor therapy across several tumor types including non-small cell lung cancer and renal cell carcinoma.

On October 16, 2019 Amarna Therapeutics, a privately held biotechnology company developing a next-generation SV40-based gene delivery vector platform named SVac that promises to transform gene-replacement and immunotherapy across many disease areas, reported that it has raised €10 million (Press release, amarna therapeutics, OCT 16, 2019, View Source [SID1234542314]). The round was led by Flerie Invest AB, a Swedish investment company with another substantial contribution coming from an innovation credit from the "Netherlands Enterprise Agency" (RVO.nl) and existing shareholder Pim Berger.

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The Company plans to use this funding to progress development of its SVac platform towards a first in man clinical study to commence in two to three years from now.

In addition to raising new funds, Amarna has recruited a new Supervisory Board to help underpin this new ‘clinical’ phase of its growth and development. Thomas Eldered has been appointed as Supervisory Board Chairman. Bernhard Kirschbaum, Maarten de Chateau, Ted Fjällman, Pim Berger and Guillaume Jetten have also joined the Supervisory Board (see biographies at the end of the release).

Ben van Leent, CEO of Amarna said:
"We are very happy to have attracted such strong investors. This significant new funding allows Amarna to accelerate the development of SVac and our lead product AMA001, and to help us achieve our ultimate goal: to become a leading global gene therapy player".
"We would like to extend a warm welcome to all of the members of our new Supervisory Board, led by the outstanding healthcare and biotech pioneer Thomas Eldered. They bring many years of in-depth life science knowledge and entrepreneurship to Amarna".

Thomas Eldered, new Chairman of Amarna’s Supervisory Board, commented:
"I’m very much looking forward to working at Amarna. Its viral gene delivery vector platform has the potential to make major medical breakthroughs possible, so that patients can be actually cured of "significant" diseases for which, to date, effective treatment have not become available. Together with my highly qualified and experienced colleagues in our new Supervisory Board, I’m fully committed to help progress Amarna into the next important clinical stages of development of its groundbreaking technology".

SV40 Vectors are non-immunogenic in humans
Viral gene delivery vectors that are currently used for in vivo gene therapy are ineffective because the particles are instable upon injection (in the case of lentiviral vectors) or because the particles are immunogenic in humans (in the case of AAV vectors). Gene delivery vectors derived from the macaque polyomavirus Simian Virus 40 (SV40) are an attractive alternative to lentiviral and AAV vectors for clinical gene therapy. Humans can be considered naïve to SV40 since the virus only replicates in macaques, where it causes symptomless infections. Replication-defective SV40 vectors are non-immunogenic in humans and moreover, have the capacity to induce immune tolerance to the transgene products. SV40 vectors therefore hold a great potential for clinical applications treating genetic disorders, cancer, allergies and degenerative/inflammatory conditions such as neurodegenerative and psychiatric diseases, atherosclerotic cardiovascular disease, diabetes mellitus, arthritis, chronic obstructive pulmonary disease and many more.

Amarna’s SVac platform: the benefits
Amarna has genetically engineered the SV40 genome used for the production of vector particles and in parallel generated a novel Vero-based packaging cell line named SuperVero that produces similar numbers of vector particles to the currently used packaging cell lines but without contaminating wild type SV40 particles. Since SVac is safe, highly efficient, non-immunogenic in humans and vector particles can be cost effectively produced in SuperVero cells, Amarna’s vector platform paves the way to clinically evaluate a whole new generation of SVac-based therapeutics for today’s major diseases.