Adrenal Insufficiency

Evidence demonstrates that SUBLOCADE supports long-term recovery from opioid use disorder (OUD) and is effective and safe. Publications highlight the importance of overcoming access barriers to long-acting injectable treatments for OUD RICHMOND, Va., Oct. 30, 2024 /PRNewswire/ -- Indivior PLC (Nasdaq/LSE: INDV), in recognizing Substance Abuse Prevention Month, provides an update on key findings from the last year that demonstrate the importance of access to medications for the treatment of opioid use disorder (MOUD) and the effectiveness and safety of SUBLOCADE® in the treatment of OUD. These data add to the growing body of evidence that SUBLOCADE can improve outcomes, such as abstinence, retention, and recovery in persons with OUD. Publications In a post hoc analysis of the SUBLOCADE randomized, double-blind, placebo-controlled phase 3 clinical trial, published in Harm Reduction Journal, SUBLOCADE 300 mg maintenance dose resulted in a statistically significant increase in abstinence from opioids among study participants with OUD who injected opioids compared to 100 mg maintenance dose. However, both were equally effective in non-injecting participants. Results from the Community Long-Acting Buprenorphine (CoLAB) Study, a prospective single-arm, multicenter, open label trial in Australia, were published in the International Journal of Drug Policy, and demonstrated that participants could be maintained on SUBLOCADE treatment for 96 weeks with continuous improvements in abstinence, depression, quality of life and medication satisfaction. In a qualitative research survey of 20 participants representing various practice settings, published in American Health & Drug Benefits, it was determined that telemedicine was the most common solution adopted during the COVID-19 pandemic to overcome access barriers. However, today there are still significant challenges in ensuring appropriate access to medications for those living with OUD and continued support, effective solutions and policies in the United States, need to be continually addressed. In a narrowly focused literature, policy, and legal proceedings review, published in Corrections Today, a clear disparity in access to MOUD was found between the general public and incarcerated individuals. Stigma and informed consent were the most discussed barriers used to justify policies limiting MOUD for incarcerated individuals. Presentations Multiple real-world evidence poster presentations highlight challenges and opportunities in treating persons with OUD. 2024 American Association of Psychiatric Pharmacists, April 7-10, Orlando FL Title: Emergency Room (ER) Visits Among Opioid Use Disorder (OUD) Patients Title: Opioid Treatment Programs, Healthcare Resource Utilization, and Healthcare Costs among Patients Initiating Treatment with Buprenorphine Extended-Release 2024 American Telemedicine Association Nexus, May 5-7, Phoenix AZ Title: Impact of Telemedicine on Medication for Opioid Use Disorder Retention during the SARS-CoV-2 Pandemic Period Among Patients with OUD 2024 College on Problems of Drug Dependence, June 15-19, Montreal Quebec Canada Title: Buprenorphine Treatment After an Emergency Department Visit for Non-Fatal Opioid Overdose Title: Pain Predicts Abstinence During Treatment of Opioid Use Disorder for Individuals Reporting Moderate to Severe Pain Title: A Randomized Open-Label Study Comparing Rapid and Standard Inductions to Injectable Buprenorphine Extended-Release (BUP-XR) Treatment Title: A Mechanistic Pharmacological Model to Predict and Inform Effective Buprenorphine Treatment Induction Strategies in the Era of Synthetic Opioids 2024 U.S. Public Health Service Scientific & Training Symposium, June 24-27, Jacksonville FL Title: Clinical and Treatment Characteristics of American Indian/Alaska Native Patients Managing Opioid Use Disorder Compared to the General US Population BUPE2024 – Buprenorphine in Medicine: Clinical and Public Policy Implications, August 5, Virtual conference Title: Healthcare Utilization and Costs Associated with Management of Opioid Use Disorder (OUD) within Residential Treatment Programs (RTP) and Office-Based Opioid Treatment Programs (OBOT) 2024 National Commission on Correctional Health Care (NCCHC) Annual, October 19-23, Las Vegas, NV Title: Outcomes Associated with Medications for Opioid Use Disorder in the Carceral System: A Systematic Literature Review Title: Diversion of Medications for OUD in the Criminal Justice System Dr. Christian Heidbreder, Ph.D., Chief Scientific Officer at Indivior, emphasized, "The collective evidence highlights the importance of access to Medications for OUD, such as SUBLOCADE, in reducing barriers to adherence and improving outcomes for individuals in recovery. Indivior is committed to continuing to advance the understanding of patients with OUD, educate on evidence-based practices, overcome stigma, and focus on recovery outcomes." Among the almost 6 million people aged 12 or older with a past year opioid use disorder in 2023, only 18% (or one million people) received treatment through MOUD.1 About SUBLOCADE® SUBLOCADE ® (buprenorphine extended-release) injection, for subcutaneous use, CIII INDICATION AND HIGHLIGHTED SAFETY INFORMATION INDICATION SUBLOCADE is indicated for the treatment of moderate to severe opioid use disorder in patients who have initiated treatment with a buprenorphine-containing product, followed by dose adjustment for a minimum of 7 days. SUBLOCADE should be used as part of a complete treatment plan that includes counseling and psychosocial support. HIGHLIGHTED SAFETY INFORMATION WARNING: RISK OF SERIOUS HARM OR DEATH WITH INTRAVENOUS ADMINISTRATION; SUBLOCADE RISK EVALUATION AND MITIGATION STRATEGY Serious harm or death could result if administered intravenously. SUBLOCADE forms a solid mass upon contact with body fluids and may cause occlusion, local tissue damage, and thrombo-embolic events, including life threatening pulmonary emboli, if administered intravenously. Because of the risk of serious harm or death that could result from intravenous self-administration, SUBLOCADE is only available through a restricted program called the SUBLOCADE REMS Program. Healthcare settings and pharmacies that order and dispense SUBLOCADE must be certified in this program and comply with the REMS requirements. CONTRAINDICATIONS SUBLOCADE should not be administered to patients who have been shown to be hypersensitive to buprenorphine or any component of Indivior's proprietary buprenorphine gel depot delivery system. WARNINGS AND PRECAUTIONS Addiction, Abuse, and Misuse: SUBLOCADE contains buprenorphine, a Schedule III controlled substance that can be abused in a manner similar to other opioids. Monitor patients for conditions indicative of diversion or progression of opioid dependence and addictive behaviors. Respiratory Depression: Life threatening respiratory depression and death have occurred in association with buprenorphine. Warn patients of the potential danger of self-administration of benzodiazepines or other CNS depressants while under treatment with SUBLOCADE. Opioids can cause sleep-related breathing disorders e.g., central sleep apnea (CSA), sleep-related hypoxemia. Opioid use increases the risk of CSA in a dose-dependent fashion. Consider decreasing the opioid using best practices for opioid taper if CSA occurs. Strongly consider prescribing naloxone at SUBLOCADE initiation or renewal because patients being treated for opioid use disorder have the potential for relapse, putting them at risk for opioid overdose. Educate patients and caregivers on how to recognize respiratory depression and how to treat with naloxone if prescribed. Risk of Serious Injection Site Reactions: The most common injection site reactions are pain, erythema and pruritus with some involving abscess, ulceration, and necrosis. The likelihood of serious injection site reactions may increase with inadvertent intramuscular or intradermal administration. Neonatal Opioid Withdrawal Syndrome: Neonatal opioid withdrawal syndrome is an expected and treatable outcome of prolonged use of opioids during pregnancy. Adrenal Insufficiency: If diagnosed, treat with physiologic replacement of corticosteroids, and wean patient off the opioid. Risk of Opioid Withdrawal With Abrupt Discontinuation: If treatment with SUBLOCADE is discontinued, monitor patients for several months for withdrawal and treat appropriately. Risk of Hepatitis, Hepatic Events: Monitor liver function tests prior to and during treatment. Risk of Withdrawal in Patients Dependent on Full Agonist Opioids: Verify that patient is clinically stable on transmucosal buprenorphine before injecting SUBLOCADE. Treatment of Emergent Acute Pain: Treat pain with a non-opioid analgesic whenever possible. If opioid therapy is required, monitor patients closely because higher doses may be required for analgesic effect. ADVERSE REACTIONS Adverse reactions commonly associated with SUBLOCADE (in ≥5% of subjects) were constipation, headache, nausea, injection site pruritus, vomiting, increased hepatic enzymes, fatigue, and injection site pain. For more information about SUBLOCADE, the full Prescribing information including BOXED WARNING, and Medication Guide, visit . About Opioid Use Disorder (OUD) Opioid Use Disorder (OUD) is a chronic disease in which people develop a pattern of using opioids that can lead to negative consequences.2 OUD may affect the parts of the brain that are necessary for life-sustaining functions.2,3 About Indivior Indivior is a global pharmaceutical company working to help change patients' lives by developing medicines to treat substance use disorders (SUD), overdose and serious mental illnesses. Our vision is that all patients around the world will have access to evidence-based treatment for the chronic conditions and co-occurring disorders of SUD. Indivior is dedicated to transforming SUD from a global human crisis to a recognized and treated chronic disease. Building on its global portfolio of OUD treatments, Indivior has a pipeline of product candidates designed to both expand on its heritage in this category and potentially address other chronic conditions and co-occurring disorders of SUD. Headquartered in the United States in Richmond, VA, Indivior employs over 1,000 individuals globally and its portfolio of products is available in over 30 countries worldwide. Visit to learn more. Connect with Indivior on LinkedIn by visiting . References Substance Abuse and Mental Health Services Administration. (2024). Key substance use and mental health indicators in the United States: Results from the 2023 National Survey on Drug Use and Health (HHS Publication No. PEP24-07-021, NSDUH Series H-59). Center for Behavioral Health Statistics and Quality, Substance Abuse and Mental Health Services Administration. Key Substance Use and Mental Health Indicators in the United States: Results from the 2023 National Survey on Drug Use and Health (samhsa.gov) National Academies of Sciences, Engineering, and Medicine; Health and Medicine Division; Board on Health Sciences Policy; Committee on Medication-Assisted Treatment for Opioid Use Disorder, Mancher, M., & Leshner, A. I. (Eds.). (2019). Medications for Opioid Use Disorder Save Lives. National Academies Press (US). Accessed October 30, 2023, from NIDA. 2022, March 22. Drugs and the Brain. Accessed October 30,2023, from SOURCE Indivior PLC WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Results from VENTURE Phase 2 Study of Subcutaneous VK2735 in Obese Patients and Phase 1 Trial of Oral VK2735 in Healthy Subjects to be Presented SAN DIEGO, Oct. 28, 2024 /PRNewswire/ -- Viking Therapeutics, Inc. ("Viking") (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced that clinical data from the company's VK2735 obesity program will be highlighted in two poster presentations at ObesityWeek® 2024, the annual meeting of the Obesity Society. VK2735 is a dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors being developed for the potential treatment of various metabolic disorders. Viking is evaluating both subcutaneous and oral formulations of VK2735 in clinical trials. ObesityWeek 2024 is being held November 3-6, 2024, in San Antonio, Texas. One presentation will summarize results from the company's Phase 2 VENTURE clinical trial, which evaluated 13 weeks of weekly treatment with a subcutaneous formulation of VK2735 in obese subjects. A second presentation will highlight results from the company's Phase 1 multiple ascending dose (MAD) clinical trial of an oral tablet formulation of VK2735 dosed daily for 28 days. Details of the presentations are as follows: Poster Presentation #018: Title: Results from the 13-Week VENTURE Phase 2 Study of the GLP-1/GIP Co-Agonist VK2735 in Obese Subjects Presenting Author: Joel Neutel, M.D., Orange County Research Center Date/Time: Sunday, November 3, 2024, 7:30 – 8:30 p.m. Central Time Location: Exhibit Hall 4B of the Henry B. Gonzalez Convention Center Poster Presentation #017: Title: First-in-Human Study of an Oral Formulation of the GLP-1/GIP Co-Agonist VK2735 in Healthy Adults Presenting Author: Joel Neutel, M.D., Orange County Research Center Date/Time: Sunday, November 3, 2024, 7:30 – 8:30 p.m. Central Time Location: Exhibit Hall 4B of the Henry B. Gonzalez Convention Center About GLP-1 and Dual GLP-1/GIP Agonists Activation of the glucagon-like peptide 1 (GLP-1) receptor has been shown to decrease glucose, reduce appetite, lower body weight, and improve insulin sensitivity in patients with type 2 diabetes, obesity, or both. Semaglutide is a GLP-1 receptor agonist that has been approved by the U.S. Food and Drug Administration and is currently marketed in various dosage strengths and forms as Ozempic®, Rybelsus®, and Wegovy®. More recently, research efforts have explored the potential co-activation of the glucose-dependent insulinotropic peptide (GIP) receptor as a means of enhancing the therapeutic benefits of GLP-1 receptor activation. Tirzepatide is a dual GLP-1/GIP receptor agonist that has been approved by the U.S. Food and Drug Administration and is currently marketed in various dosage strengths and forms as Mounjaro® and Zepbound®. About Viking Therapeutics, Inc. Viking Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel first-in-class or best-in-class therapies for the treatment of metabolic and endocrine disorders, with three compounds currently in clinical trials. Viking's research and development activities leverage its expertise in metabolism to develop innovative therapeutics designed to improve patients' lives. Viking's clinical programs include VK2735, a novel dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors for the potential treatment of various metabolic disorders. Data from a Phase 1 and a Phase 2 trial evaluating VK2735 (dosed subcutaneously) for metabolic disorders demonstrated an encouraging safety and tolerability profile as well as positive signs of clinical benefit. Concurrently, the company is evaluating an oral formulation of VK2735 in a Phase 1 trial. Viking is also developing VK2809, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist for the treatment of lipid and metabolic disorders. The compound successfully achieved both the primary and secondary endpoints in a recently completed Phase 2b study for the treatment of biopsy-confirmed non-alcoholic steatohepatitis (NASH) and fibrosis. In a Phase 2a trial for the treatment of non-alcoholic fatty liver disease (NAFLD) and elevated LDL-C, patients who received VK2809 demonstrated statistically significant reductions in LDL-C and liver fat content compared with patients who received placebo. The company's newest program is evaluating a series of internally developed dual amylin and calcitonin receptor agonists (or DACRAs) for the treatment of obesity and other metabolic disorders. In the rare disease space, Viking is developing VK0214, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist for the potential treatment of X-linked adrenoleukodystrophy (X-ALD). In a Phase 1b clinical trial in patients with the adrenomyeloneuropathy (AMN) form of X-ALD, VK0214 was shown to be safe and well-tolerated, while driving significant reductions in plasma levels of very long-chain fatty acids (VLCFAs) and other lipids, as compared to placebo. For more information about Viking Therapeutics, please visit . SOURCE Viking Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

iStock/ Lynne Mitchell As therapies for rare and neurological diseases earn accelerated approval, experts laud the program’s intent while remaining concerned about confirmatory trials and clinical efficacy, especially as products greenlit under this pathway are pulled from the market. Last month, Pfizer’s sickle cell disease therapy Oxbryta , which won accelerated approval in 2019, was pulled from market due to risk of death and other complications, illustrating the risk of greenlighting therapeutics through this pathway. The FDA’s accelerated approval program has helped shepherd nearly 300 new drugs to the market, providing access to these medicines an average of 3.2 years earlier than with traditional approvals, according to the industry trade group PhRMA. Focusing on five drugs approved through this program, PhRMA’s report found that “millions of patients have gained faster access to clinical benefits, gaining additional life years, years of symptom control, or disease complication-free years over standard of care.” Having originated during the AIDS pandemic, many accelerated approvals have come in infectious disease, as well as in oncology, but the pathway has recently been leveraged by several high-pro and rare disease therapeutics. Over the past five years, the FDA has granted accelerated approval to novel therapies for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy and more. While about half of drugs approved through this pathway, including Eisai and Biogen’s Alzheimer’s drug Leqembi and AbbVie’s ovarian cancer drug Elahere , have succeeded in securing traditional approval, Pfizer’s Oxbryta is but one example where the therapy was ultimately unsuccessful . Others include Biogen and Eisai’s first Alzheimer’s drug, Aduhelm, which Biogen discontinued in January 2024, and Takeda’s Exkivity, which was withdrawn from the lung cancer market in October 2023 after failing to meet the primary endpoint in a confirmatory study. “The advantage of an accelerated pathway is that the FDA can potentially move drugs to help people with terrible conditions forward quickly,” Joel Perlmutter, a professor of neurology at Washington University School of Medicine in St. Louis, told BioSpace . “The downside is how they do that.” Accelerated approval is granted on the basis of a surrogate endpoint—such as a biomarker of the disease—that is reasonably likely to predict clinical benefit. Companies are then required to conduct a confirmatory trial while the drug hits the market. Gene therapies for rare diseases would seem to be ideal candidates for accelerated approval, given that biomarkers feature prominently in these trials, Emil Kakkis, CEO of rare disease drug developer Ultragenyx, told BioSpace . Yet the accelerated approval pathway has so far been utilized for very few rare disease therapies. Kakkis noted just two: Sarepta Therapeutics’ Elevidys , approved in June 2023 for Duchenne muscular dystrophy (DMD), and bluebird bio’s Skysona , greenlit in September 2022 for cerebral adrenoleukodystrophy. Kakkis indicated he would like to see the pace pick up. In a 2011 study , he and a colleague estimated that better access to the accelerated approval pathway could greatly reduce development costs in the rare disease space, enabling development of three times as many rare disease drugs even if investment stayed flat. Moreover, Kakkis said, prioritizing primary disease activity biomarkers that measure the cause of the disease rather than clinical endpoints for approval is “a step forward in the evolution of drug development and our understanding of disease.” “We should start defining disease as the cause of disease, not the outcomes of disease,” he said. “And if we do that, we’ll actually treat disease better. We’ll treat it before it hurts you.” Accelerated Approval Evolves Beyond Infectious Disease and Oncology The accelerated approval program was implemented in 1992 in response to the HIV/AIDS epidemic. During the first 10 years of the program, 65% of drugs to receive accelerated approval were for infectious diseases. The next two decades saw a multitude of cancer drugs hit the market through this pathway—between 2012 and 2021, 83% of accelerated approvals were in oncology. Now, a third era, accelerated approvals for rare and neurological diseases, appears to be dawning. In June 2021, Biogen and Eisai’s Aduhelm won accelerated approval as the first disease-modifying treatment for Alzheimer’s disease. This was followed by BioMarin’s Voxzogo for achondroplasia in November 2021, Biogen’s Qalsody for a rare form of ALS in April 2023 and Elevidys in June 2023. The approvals of Aduhelm and Elevidys, in particular, set off a firestorm of controversy as experts questioned the clinical benefit of these products. The subsequent market withdrawal of Aduhelm and the confirmatory trial failure of Elevidys added fuel to the fire and sparked questions about the value of the accelerated approval program. Then came Oxbryta. Joseph Ross, a professor of medicine and public health at Yale School of Medicine, expressed concern that other FDA divisions do not appear to be heeding the hard-won learnings of the Oncology Center of Excellence. “We are essentially being forced to relearn the lessons that the Oncology Group learned over the past 25 years,” he told BioSpace . These lessons include the need to avoid confirmatory trial delays and focus on clinical outcomes, he said. The FDA did not respond to BioSpace ’s requests for comment. Better Understanding Biomarkers The accelerated approval pathway primarily relies on biomarkers that may predict subsequent clinical benefit, Perlmutter said. “Therein lies the whole issue.” Investigational medicines are traditionally considered based on their clinical benefit and risk profile, Perlmutter explained. “Using a biomarker to predict clinical benefit for both the benefit and the risk is nearly impossible.” Current biomarkers “will give an idea of target engagement,” Perlmutter said. “It rarely tells you whether it’s safe to do so, and that almost certainly requires clinical observation.” This is where the confirmatory trials come in. But these studies are often delayed. In 2022, NPR found that 42% of currently outstanding confirmatory studies either took more than a year to begin following an accelerated approval, or had not yet started. “Those follow-up studies are rarely done in a timely fashion,” Perlmutter noted. In the end, he added, drugs with “potentially harmful effects” and “without adequate benefits” can remain on the market. Oxbrtya was on the market for nearly five years, while Gilead Sciences’ Zydelig was available to patients with lymphoma for more than seven years before Gilead withdrew the drug in 2022 after failing to complete follow-up studies. Ross noted that the FDA does not require surrogate markers to be validated by clinical evidence of an association between the marker and clinical outcomes. “It seems like a fool’s errand to continue to allow products to be approved on the basis of the same fallible surrogate markers over and over,” he said. “I think we need a much better sense of the surrogate markers that are being used as efficacy endpoints.” Perlmutter offered Aduhelm, which won accelerated approval based on its ability to remove amyloid beta from the brains of patients with Alzheimer’s, as one example of a questionable biomarker. “The problem is, at that time—and actually currently—nobody [has] proved that reduction of amyloid in the brain predicts clinical benefit,” said Perlmutter, who served on the advisory committee that rejected Aduhelm and resigned as a result of its approval. But Kakkis contends that biomarkers are more of an issue when it comes to complex diseases like Alzheimer’s and ALS. “Those situations are trickier because you can’t be sure that the biomarker is reflecting the source of disease.” By contrast, biomarkers are a reliable surrogate endpoint in the rare, genetic disease space where they represent the underlying cause of the disease, he said. “In all cases, that’s transformed into clinical benefit down the road. It has not failed.” Perlmutter agreed that genetic diseases where the correlation between the biomarker and the cause of disease can be proven are an appropriate use of the accelerated approval pathway. “I just think you have to prove it before you approve it,” he said. A Worthy Regulatory Avenue There is a perception that accelerated approval is a regulatory shortcut , but Kakkis takes issue with the idea that it is “a less important or less rigorous approval.” “The biomarker-based approval of HIV drugs was not a compromise,” he said. “It’s far superior to doing it any other way.” He explained that it would have taken too long to run trials with clinical endpoints like opportunistic infections, hospitalizations or death with three or four combination drugs, “and too many people would have to get sick and die.” The FDA has taken steps to dispel this shortcut notion and tighten up the program. Responding to delays in the completion of confirmatory trials, legislation passed in 2022 empowers the agency to require these studies be underway before granting accelerated approval. But the experts who spoke with BioSpace say more changes are needed. Concerning confirmatory trials, Ross said, “I think we need much firmer deadlines that are much shorter in nature to ensure that we have timely evidence that helps the patient and clinician community quickly understand whether the drug is truly safe and effective.” Meanwhile, Perlmutter expressed concern about the potential for bias given that 65% of the FDA’s budget for human drugs comes from companies applying for approval. This is particularly an issue for accelerated approval “where judgment is critical and any potential bias can influence judgment,” he said. “Time is money for Big Pharma. Sooner approval means quicker onset of sales.”