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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."

PRINCETON, N.J., Aug. 09, 2023 (GLOBE NEWSWIRE) -- Medunik USA (member of Duchesnay Pharmaceutical Group) is pleased to announce that an increasing number of Americans with certain urea cycle disorders (UCDs) now have access to Pheburane® (sodium phenylbutyrate) through a rapidly expanding network of key pharmacy benefit managers (PBMs), state Medicaid programs or Managed Medicaid plans. Six state Medicaid programs, namely Texas, Oklahoma, North Dakota, Utah*, New Hampshire and New Mexico*, have now included Pheburane® on their Preferred Drug Lists as well as being included on California’s Contract Drug List with no prior authorization required. Pheburane® is also covered with preferred status at PBMs serving ~40% of covered individuals. Pheburane® offers an affordable taste-masked treatment option for patients affected by certain UCDs, used along with a specific diet, at one-third of the cost of RAVICTI® (glycerol phenylbutyrate) and half the price of OLPRUVA™, the most recently launched UCD treatment (based on published pricing information). Pheburane® is not indicated for the treatment of acute hyperammonemia. Pheburane® is an innovative, palatable formulation designed with a special coating to mask the awful taste of sodium phenylbutyrate (NaPB) which leads some patients to not being able to consistently or correctly take their non-taste-masked medication, resulting in life-threatening complications. The most common side effects associated with sodium phenylbutyrate are menstrual dysfunction, decreased appetite, body odor and bad taste or taste aversion.1 “We are glad our efforts to bring Pheburane® to Americans with certain UCDs are coming to fruition.” said Tanya Carro, Executive Vice-President of Medunik USA. “The competitive price of Pheburane® combined with increasing coverage from public and private payers is a testimony of Medunik USA’s commitment to the UCD community and its stakeholders.” UCDs are rare, chronic, genetic conditions that can be fatal if left untreated, and can impact children from the time of birth. UCDs disrupt the body’s urea cycle and, therefore, the body is unable to remove the dangerous buildup of toxic chemicals, particularly ammonia, that are created from the digestion of protein. One in 35,000 people in the United States or about 28 per one million residents suffer from UCDs of different levels of severity.2 Pheburane® consists of small oral pellets (about the size of sugar crystals) which are easily measured with its reusable, calibrated dosing spoon. It does not require any mixing or oral syringes and there is no weight restriction for patients. Pheburane® has a proven history in the global treatment of UCDs, as it has been available in Europe since 2013, and in Canada since 2015. Medunik USA has provided access to Pheburane® for Americans affected by certain UCDs since September 2022 and has since been working diligently at securing nation-wide coverage at a competitive price. Medunik USA offers Pheburane® through its UNIK Support Program – designed to support each unique patient. UNIK offers specialized services including a copay savings program, patient care liaison services, mail order pharmacy and other support services. Visit Pheburane.com for more information. * Certain Managed Medicaid plans About urea cycle disorders (UCDs) The urea cycle is a process which allows the body to keep and use positive contents of food and filter and remove any toxic substances that can be ingested or created. UCDs are rare genetic disorders that cause errors in this process, allowing toxic substances to build up in the bloodstream, potentially to dangerous and fatal levels. The key marker for UCDs is high levels of ammonia. Removal of ammonia from the bloodstream normally occurs via its conversion to urea, which is then excreted by the kidneys. Ammonia is extremely toxic, particularly to the central nervous system. UCDs can cause catastrophic illness in newborns within 36 to 48 hours of birth despite the infants appearing normal, so they can be discharged from hospital before signs of UCDs develop. UCDs require lifelong monitoring and treatment.2 About Pheburane® Pheburane® is a taste-masked oral formulation of sodium phenylbutyrate, approved by the Food and Drug Administration (FDA) as adjunctive therapy to standard of care, which includes dietary management, for the chronic management of adult and pediatric patients with UCDs, involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinic acid synthetase (AS). Pheburane® is not indicated for the treatment of acute hyperammonemia.1 INDICATION AND IMPORTANT SAFETY INFORMATION What is Pheburane®? Pheburane® is a prescription medicine, used along with a specific diet, for the long-term management of adults and children with urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinate synthetase (AS). Episodes of sudden, rapid increase of ammonia in the blood (acute hyperammonemia) may happen in people during treatment with Pheburane®. Pheburane® is not used for the treatment of acute hyperammonemia, which can be life-threatening and requires emergency medical treatment. BeforetakingPheburane®, tell your healthcare provider about all of your medical conditions, including if you: have heart problems. have kidney or liver problems. have diabetes (Pheburane® contains sucrose), or have a history of fructose intolerance, glucose-galactose malabsorption, or sucrose-isomaltase insufficiency. are pregnant or plan to become pregnant. It is not known if Pheburane® will harm your unborn baby. are breastfeeding or plan to breastfeed. It is not known if Pheburane® passes into your breastmilk. Talk to your healthcare provider about the best way to feed your baby during treatment with Pheburane®. Tell your healthcare provider about all the medicines you or your child take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Certain medicines may increase the level of ammonia in your blood or cause serious side effects when taken during treatment with Pheburane®. Especially tell your healthcare provider if you or your child take: corticosteroids valproic acid haloperidol probenecid Know the medicines you take. Keep a list of them to show your or your child’s healthcare provider and pharmacist when you get a new medicine. What are the possible side effects of Pheburane®? Pheburane® can cause serious side effects, including: Nervous system problems (neurotoxicity). Call your healthcare provider right away if you get any of the following symptoms during treatment with Pheburane®: sleepiness tiredness lightheadedness vomiting nausea headache confusion Low potassium levels in your blood (hypokalemia). Your healthcare provider will monitor your blood potassium levels during treatment with PHEBURANE and treat if needed. Conditions related to swelling (edema).Pheburane® contains salt (sodium), which can cause swelling from salt and water retention. Your healthcare provider will decide if PHEBURANE is right for you if you have certain medical conditions that can cause swelling, such as heart failure, liver problems or kidney problems. The most common side effects of Pheburane® include: absent or irregular menstrual periods decreased appetite body odor bad taste or avoiding foods that you ate prior to getting sick (taste aversion) Your healthcare provider may do certain blood tests to check you or your child for side effects during treatment with Pheburane®. These are not all the possible side effects of Pheburane®. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088. Please read the Full Prescribing Information and Patient Information at Pheburane.com. Pheburane®, developed by Lucane Pharma, is distributed exclusively in the U.S. by Medunik USA. For further information, visit Pheburane.com. About Medunik USA Based in Princeton, New Jersey, Medunik USA is part of Duchesnay Pharmaceutical Group and works to improve the health and quality of life of Americans living with rare diseases by making orphan drug therapies available in the United States. With strategic partnerships at the global level, the company has critical experience in approval and market access processes as well as the marketing of orphan drug therapies. Medunik USA makes critical medications to treat rare diseases available to American patients who might not otherwise have access to these medications. For more information, visit . About Duchesnay Pharmaceutical Group Duchesnay Pharmaceutical Group (DPG), with its affiliated companies, is headquartered in Blainville, Quebec, Canada. The Group consists of five pharmaceutical companies to meet the needs of patients in Canada, the U.S. and abroad. The companies are Duchesnay (Canada) and Duchesnay USA, both dedicated to women's health; Médunik Canada and Medunik USA, which provide treatments for rare diseases; and Analog Pharma, an American generic drugs company, specializing in authorized generics and orphan drugs. From its state-of-the-art manufacturing plant, DPG exports its innovative treatments to more than 50 countries. The Group, through its proprietary research and development, and through exclusive partnerships, offers innovative treatments for a variety of medical conditions in women's health, urology, oncology as well as for rare diseases. DPG recognizes the dedication and professionalism of its employees and promotes an inclusive culture and flexible work environment. It is deeply committed to environmental responsibility and to giving back to the community through the support of various charitable organizations. For more information, visit duchesnaypharmaceuticalgroup.com. References 1. Pheburane® (sodium phenylbutyrate) oral pellets [Prescribing Information]. Medunik USA, Inc. 2. Cleveland Clinic, Urea Cycle Disorder, Media contact: Email: msavchuk@duchesnay.com Telephone: 1-877-833-7734 ext. 149 A photo accompanying this announcement is available at Pheburane® Now on the Preferred Drug List in 6 states and included on the Contract Drug List in California.