Polaris Pharmaceuticals, Inc.

An innovative treatment significantly increases the survival of people with malignant mesothelioma, a rare but rapidly fatal type of cancer with few effective treatment options. An innovative treatment significantly increases the survival of people with malignant mesothelioma, a rare but rapidly fatal type of cancer with few effective treatment options, according to results from a clinical trial led by Queen Mary University of London. The phase 3 clinical trial, led by Professor Peter Szlosarek at Queen Mary and sponsored by Polaris Pharmaceuticals, has unveiled a breakthrough in the treatment of malignant pleural mesothelioma (MPM), a rare and often rapidly fatal form of cancer with limited therapeutic options. Mick's journey with mesothelioma: "I have five grandchildren and two great-grandchildren now -- I wouldn't want to miss all that." The ATOMIC-meso trial, a randomised placebo-controlled study of 249 patients with MPM, found that a treatment -- which combines a new drug, ADI-PEG20, with traditional chemotherapy -- increased the median survival of participants by 1.6 months, and quadrupled the survival at 36 months, compared to placebo-chemotherapy. The findings are significant, as MPM has one of the lowest 5-year survival rates of any solid cancer of around 5-10%. This innovative approach marks the first successful combination of chemotherapy with a drug that targets cancer's metabolism developed for this disease in 20 years. MPM is a rare, aggressive cancer that affects the lining of the lungs and is associated with exposure to asbestos. It's usually treated with potent chemotherapy drugs, but these are seldom able to halt the progression of the disease. The premise behind this new drug treatment is elegant in its simplicity -- starving the tumour by cutting off its food supply. All cells need nutrients to grow and multiply, including amino acids like arginine. ADI-PEG20 works by depleting arginine levels in the bloodstream. For tumour cells that can't manufacture their arginine due to a missing enzyme, this means their growth is thwarted. The ATOMIC-meso trial is the culmination of 20 years of research at Queen Mary's Barts Cancer Institute that began with Professor Szlosarek's discovery that malignant mesothelioma cells lack a protein called ASS1, which enables cells to manufacture their own arginine. He and his team have since dedicated their efforts to using this knowledge to create an effective treatment for patients with MPM. Professor Szlosarek said: "It's truly wonderful to see the research into the arginine starvation of cancer cells come to fruition. This discovery is something I have been driving from its earliest stages in the lab, with a new treatment, ADI-PEG20, now improving patient lives affected by mesothelioma. I thank all the patients and families, investigators and their teams, and Polaris Pharmaceuticals for their commitment to defining a new cancer therapy." Dr Tayyaba Jiwani, Science Engagement Manager at Cancer Research UK, said: "This study shows the power of discovery research which allows us to dig deep into the biology of mesothelioma to uncover vulnerabilities that we can now target with ADI-PEG20. "Cancer Research UK is delighted to have funded the early stages of this research, including a preliminary clinical trial which established the safety and effectiveness of this drug." There are ongoing studies assessing ADI-PEG20 in patients who have sarcoma or glioblastoma multiforme (a type of brain tumour) and other cancers dependent on arginine. The success of this novel chemotherapy in MPM also suggests that the drug may be of benefit in the treatment of multiple other types of cancer.  Mick's journey with mesothelioma Mick worked in a factory boiler room in the 1970s, where he was exposed to asbestos. In 2018, he visited his doctor after he began to feel unwell and had lost three stone in weight. He became anaemic and was eventually diagnosed with mesothelioma. "It was a bit of a shock: I was given four months to live," Mick explains. His doctor referred him to Professor Szlosarek, who enrolled him in the ATOMIC-meso trial. "I always believed in Peter. I said: 'I'm in it to win it -- you're not getting rid of me.' And here I am five years later." For two years, Mick visited St Bartholomew's Hospital every week, accompanied by his wife, Jackie, or one of his children or grandchildren. "I'd have two injections of the new treatment -- one in each arm. I didn't have any serious side effects," Mick explains. "I met many of the other people on the trial. Over time, some of them disappeared. But I kept going." Mick was awarded compensation from his former employer responsible for the asbestos exposure that ultimately led to his mesothelioma. Around 80% of mesothelioma cases are caused by workplace exposure. Two and a half years after Mick enrolled on the ATOMIC-meso trial, his mesothelioma returned and he received a second course of treatment, this time immunotherapy. He experienced more side effects with this therapy, including encephalitis. But his cancer remains under control, and recently he was able to celebrate his 80th birthday. Professor Szlosarek and his team plan to study why certain patients, such as Mick, benefit so greatly from ADI-PEG20, in the hope of discovering how to extend this benefit to more people. Mick says: "This trial has changed the lives of people with mesothelioma, allowing us to live longer. I have five grandchildren and two great-grandchildren now -- I wouldn't want to miss all that."

Polaris Now Enrolling in GBM AGILE, Registrational Phase 2-3 Adaptive Platform Trial for Patients with Glioblastoma Polaris lead biologic ADI-PEG 20 to be Evaluated Across Multiple International Trial Sites LARKSPUR, Calif.--(BUSINESS WIRE)-- Global Coalition for Adaptive Research (LARKSPUR, CA) and Polaris Pharmaceuticals, Inc. (SAN DIEGO, CA), a subsidiary of Polaris Group (TWSE:6550)— The Global Coalition for Adaptive Research (GCAR) in collaboration with Polaris, today announced the activation of ADI-PEG 20 in GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment – NCT03970447). GBM AGILE is a revolutionary patient-centered, adaptive platform trial for registration that evaluates multiple therapies for patients with newly-diagnosed and recurrent glioblastoma (GBM) – the deadliest form of brain cancer. This press release features multimedia. View the full release here: Polaris’ ADI-PEG 20 is entering the GBM AGILE trial, which initially opened in July 2019 and has screened over 1,500 patients to date. The investigational therapy will be evaluated at clinical trial sites in the U.S., Canada, Europe, and Australia. The ADI-PEG 20 arm of GBM AGILE will be enrolling patients with newly-diagnosed unmethylated MGMT, newly-diagnosed methylated MGMT, and recurrent GBM. Dr. Macarena de la Fuente, Associate Professor of Neurology and Chief, Neuro-Oncology at University of Miami Sylvester Comprehensive Cancer Center and Dr. Nicholas Blondin, Assistant Professor of Clinical Neurology at Yale School of Medicine are serving as arm Principal Investigators for the ADI-PEG 20 arm. “GBM AGILE is designed to maximize learning and rapidly identify effective therapies for patients with glioblastoma by simultaneously and dynamically evaluating the effects of multiple new drug candidates,” said Dr. Macarena de la Fuente. “We look forward to evaluating ADI‑PEG 20 in GBM AGILE. This investigational drug has the potential to support improved outcomes for GBM patients, who deserve better options for their care.” “Glioblastoma is a deadly brain cancer with few effective treatment options. Patients are in desperate need of better treatment options,” said Dr. Nicholas Blondin. “With the addition of ADI-PEG 20 to GBM AGILE, a global clinical trial for newly diagnosed and recurrent GBM patients, we are able to offer GBM patients access to the latest and most promising investigational therapies.” GBM AGILE is an international, innovative platform trial designed to more rapidly identify and confirm effective therapies for patients with glioblastoma through response adaptive randomization and a seamless phase 2/3 design. The trial, conceived by over 130 key opinion leaders, is conducted under a master protocol, allowing multiple therapies or combinations of therapies from different pharmaceutical partners to be evaluated simultaneously. With its innovative design and efficient operational infrastructure, data from GBM AGILE can be used as the foundation for a new drug application (NDA) and biologics license application (BLA) submissions and registrations to the FDA and other health authorities. ADI-PEG 20 is a biologic being developed by Polaris Group to treat various cancers. Arginine is an amino acid that is essential for protein synthesis and survival of cells. Some cancer cells become dependent upon the external supply of arginine to survive and grow. ADI-PEG 20 is designed to deplete the external supply of arginine, which causes arginine-dependent cancer cells to die while leaving the patient’s normal cells unharmed. Multiple cancers have been reported to have a high degree of arginine dependency. ADI-PEG 20 has been tested in more than twenty clinical trials globally for a wide variety of cancers, including hepatocellular carcinoma, mesothelioma, pancreatic cancer, non-small cell lung cancer, melanoma, acute myeloid leukemia, and others. “We are excited to initiate the evaluation of ADI-PEG 20 in GBM AGILE, a cutting-edge adaptive trial designed to rapidly identify whether drugs such as ADI-PEG 20 can bring benefit to patients with glioblastoma,” said Howard Chen, Chairman and CEO, Polaris Group. “We have seen encouraging data with ADI-PEG 20 in previous clinical studies, and look forward to rapidly advancing the development of ADI-PEG 20 for the treatment of glioblastoma, a devastating and deadly disease with a critical and unmet need for more effective treatments.” About Global Coalition for Adaptive Research (GCAR) Global Coalition for Adaptive Research (GCAR) is a 501(c)(3) non-profit corporation uniting biotech/pharma, physicians, clinical researchers, advocacy and philanthropic organizations, health authorities, and other key stakeholders in healthcare. Together, GCAR and its partners expedite the discovery and development of treatments for patients with rare and deadly diseases. GCAR leads and implements innovative and complex trials including master protocols and platform trials and is the sponsor of GBM AGILE, a patient-centric adaptive platform trial for patients with GBM – the deadliest of malignant primary brain tumors. Key advocacy partners for the GBM AGILE trial effort include the National Brain Tumor Society, National Foundation for Cancer Research, Asian Fund for Cancer Research, and Cure Brain Cancer Foundation, non-profit organizations working together to provide philanthropic support and assist in communicating with patients and families. We invite others to support this innovative approach to brain tumor treatment development. To learn more about GCAR and its initiatives, visit gcaresearch.org. About Polaris Group Polaris Group is a multinational biotechnology company specializing in the research and development of novel pharmaceuticals to treat cancer. The company’s lead therapeutic, ADI‑PEG 20, is a novel biologic in late-stage clinical development. It has been tested in more than twenty clinical trials globally for a wide variety of cancers, including hepatocellular carcinoma, mesothelioma, pancreatic cancer, non-small cell lung cancer, melanoma, acute myeloid leukemia and others. As a fully integrated company, Polaris is engaged in applying protein structure-based drug design technology to develop novel drug candidates for cancers, conducting clinical studies at top-tier cancer centers worldwide, and manufacturing all clinical materials at its cGMP facility in northern California. For additional information please visit polarispharma.com

LARKSPUR, Calif.--( BUSINESS WIRE )--Global Coalition for Adaptive Research (GCAR), a 501(c)(3) non-profit research organization, announced that together with their patient/advocacy and pharma/biotech partners, they will ring the Nasdaq Stock Market Closing Bell today to spotlight National Brain Tumor Awareness Month and their innovative, global brain tumor trial called GBM AGILE. The GBM AGILE trial, lead by GCAR, is a multi-stakeholder adaptive platform trial for patients with glioblastoma (GBM) – the deadliest of malignant primary brain tumors. GBM accounts for 48% of all primary brain cancers and has a median survival rate of less than 15 months, with only 3 treatments approved in the last 40 years. GCAR is joined by their GBM AGILE partners: Bayer, Kazia Therapeutics, Kintara Therapeutics, Vigeo Therapeutics, Biohaven, Polaris Pharmaceuticals, National Brain Tumor Society, National Foundation for Cancer Research, Asian Fund for Cancer Research, and Cure Brain Cancer Foundation. “ We are honored to ring the Nasdaq closing bell alongside our GBM AGILE partners, patients, caregivers, and physicians united to raise awareness of the critical, unmet need for new treatment options for patients with brain tumors,” said Dr. Meredith Buxton, CEO and President of GCAR. “ Innovative trials like GBM AGILE accelerate drug development through a more cost-effective and rapid path to market approval; however, more investment is needed to advance new treatment options and to drive breakthroughs in patient care.” Each year, approximately 300,000 people worldwide are diagnosed with primary brain tumors. May is National Brain Tumor Awareness Month, a time to support and magnify the voice of the brain tumor community and draw attention to the critical, unmet need for faster and better treatment options. #GoGrayinMay About Global Coalition for Adaptive Research (GCAR) Global Coalition for Adaptive Research (GCAR) is a 501(c)(3) non-profit corporation uniting biotech/pharma, physicians, clinical researchers, advocacy and philanthropic organizations, health authorities, and other key stakeholders in healthcare. Together, GCAR and its partners expedite the discovery and development of treatments for patients with rare and deadly diseases. GCAR leads and implements innovative and complex trials including master protocols and platform trials and is the sponsor of GBM AGILE, a patient-centric adaptive platform trial for patients with GBM – the deadliest of malignant primary brain tumors. Key advocacy partners for the GBM AGILE trial effort include the National Brain Tumor Society , National Foundation for Cancer Research , Asian Fund for Cancer Research , and Cure Brain Cancer Foundation , non-profit organizations working together to provide philanthropic support and assist in communicating with patients and families. We invite others to support this innovative approach to brain tumor treatment development. To learn more about GCAR and its initiatives, visit gcaresearch.org .