ABCD2

Reductions in Very Long-Chain Fatty Acids and Plasma Lipid Levels Observed After 28 Days of Once Daily Dosing VK0214 Shown to be Safe and Well-Tolerated in 28-Day Study SAN DIEGO, Oct. 9, 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 positive data from the company's Phase 1b clinical trial of VK0214, a novel small molecule agonist of the thyroid hormone receptor beta (TRβ), in patients with X-linked adrenoleukodystrophy (X-ALD). Results from this study showed VK0214 to be safe and well-tolerated following once-daily dosing over the 28-day study period. In addition, significant reductions were observed in plasma levels of very long-chain fatty acids (VLCFAs) and other lipids, as compared to placebo. Highlights from the study results include: Reductions in VLCFAs In addition to safety and tolerability, the study included an exploratory assessment of changes in plasma levels of VLCFAs after 28 days of dosing. VLCFAs are considered biomarkers of disease in patients with X-ALD. Treatment with VK0214 resulted in significant reductions in mean VLCFA levels at both doses evaluated, 20 mg/day and 40 mg/day, compared to placebo. Importantly, cohorts receiving VK0214 demonstrated reductions in mean plasma levels of the 26 carbon lysophosphatidyl choline (C26:0-LPC) derivative, a key diagnostic marker. Percent Change from Baseline in VLCFAs Following 28 Days of Treatment of VK0214 Reductions in Plasma Lipids In addition to VLCFA changes, subjects who received VK0214 demonstrated reductions in other plasma lipids. Mean reductions relative to baseline and placebo were observed for low-density lipoprotein cholesterol (LDL-C), apolipoprotein B (ApoB), and lipoprotein (a) [Lp(a)] following 28 days of treatment. Percent Change in Lipid Markers Following 28 Days of Treatment of VK0214 Safety and Tolerability VK0214 demonstrated encouraging safety and tolerability following 28 days of once-daily dosing. Treatment emergent adverse events were reported as mild to moderate; one subject in the placebo cohort experienced a wrist fracture that was characterized as a severe adverse event. Gastrointestinal adverse events were slightly higher among placebo subjects (33%) compared with VK0214-treated subjects (11%). "We are excited to report these initial results evaluating VK0214 in patients with adrenomyeloneuropathy," said Brian Lian, Ph.D., chief executive officer of Viking. "Patients receiving VK0214 demonstrated progressive improvement in plasma levels of very long chain fatty acids in the relatively brief treatment period evaluated in this study. In addition, VK0214 continued to show benefits on broader plasma lipids, such as LDL-C, important for overall cardiometabolic health. Consistent with prior clinical results in healthy volunteers, VK0214 was shown to be safe and well-tolerated in this 28-day study. We look forward to exploring next steps with this important compound." The Phase 1b trial was a multi-center, randomized, double-blind, placebo-controlled study in adult male patients with the adrenomyeloneuropathy (AMN) form of X-ALD. The study enrolled patients across three cohorts: placebo, VK0214 20 mg daily, and VK0214 40 mg daily. The primary objectives were to evaluate the safety and tolerability of VK0214 in subjects with AMN, when administered once-daily over a 28-day dosing period. Secondary objectives included an evaluation of the pharmacokinetics of VK0214 following 28 days of dosing in this population. An exploratory objective was to evaluate the effects of VK0214 on plasma levels of VLCFAs in subjects with AMN. About X-ALD X-ALD is a rare and often fatal metabolic disorder characterized by a breakdown in the protective barriers surrounding brain and nerve cells; a process known as demyelination. In X-ALD, mutations in a gene known as ABCD1 lead to dysfunction of the adrenoleukodystrophy protein (ALDP), an important peroxisomal transporter. In patients, this dysfunction leads to an accumulation of VLCFAs, which can trigger a rapid, inflammatory demyelination, resulting in cognitive impairment, motor skill deterioration, and even death. Research in disease models has shown that increasing the expression of a related gene known as ABCD2, which encodes a compensatory transporter called the adrenoleukodystrophy related protein (ADLRP), can result in normalization of VLCFA levels. Activation of the thyroid hormone beta receptor, as observed in patients treated with VK0214, has been shown to increase the expression of ABCD2 resulting in reductions in VLCFA levels. X-ALD is estimated to occur in approximately 1 in 17,000 births. Currently there are no pharmacologic treatments approved for the disease. 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 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. Viking is also developing 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. The company is also evaluating an oral formulation of VK2735 in a Phase 1 trial. 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). VK0214 is currently being evaluated in a Phase 1b clinical trial in patients with the adrenomyeloneuropathy (AMN) form of X-ALD. For more information about Viking Therapeutics, please visit . Forward-Looking Statements This press release contains forward-looking statements regarding Viking Therapeutics, Inc., under the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995, including statements about Viking's expectations regarding its clinical and preclinical development programs, anticipated timing for reporting clinical data and cash resources. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially and adversely and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: risks associated with the success, cost and timing of Viking's product candidate development activities and clinical trials, including those for VK2735, VK0214, VK2809, the company's incretin receptor agonists, and its amylin and calcitonin receptor agonists; risks that prior clinical and preclinical results may not be replicated; risks regarding regulatory requirements; and other risks that are described in Viking's most recent periodic reports filed with the Securities and Exchange Commission, including Viking's Annual Report on Form 10-K for the year ended December 31, 2023, and subsequent Quarterly Reports on Form 10-Q, including the risk factors set forth in those filings. These forward-looking statements speak only as of the date hereof. Viking disclaims any obligation to update these forward-looking statements except as required by law. SOURCE Viking Therapeutics, Inc. 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A new scientific statement discusses rapid evaluation for transient ischemic attack (TIA) due to high risk of future stroke. Stroke symptoms that disappear in under an hour, known as a transient ischemic attack (TIA), need emergency assessment to help prevent a full-blown stroke, according to a new American Heart Association scientific statement published today in the Association's journal Stroke. The statement offers a standardized approach to evaluating people with suspected TIA, with guidance specifically for hospitals in rural areas that may not have access to advanced imaging or an on-site neurologist. TIA is a temporary blockage of blood flow to the brain. Each year, about 240,000 people in the U.S. experience a TIA, although this estimate may represent underreporting of TIA because symptoms tend to go away within an hour. While the TIA itself doesn't cause permanent damage, nearly 1 in 5 of those who have a TIA will have a full-blown stroke within three months after the TIA, almost half of which will happen within two days. For this reason, a TIA is more accurately described as a warning stroke rather than a "mini-stroke," as it's often called. TIA symptoms are the same as stroke symptoms, only temporary. They begin suddenly and may have any or all of these characteristics: The F.A.S.T. acronym for stroke symptoms can be used to identify a TIA: F Face drooping or numbness; A Arm weakness; S Speech difficulty; T Time to call 9-1-1, even if the symptoms go away. "Confidently diagnosing a TIA is difficult since most patients are back to normal function by the time they arrive at the emergency room," said Hardik P. Amin, M.D., chair of the scientific statement writing committee and associate professor of neurology and medical stroke director at Yale New Haven Hospital, St. Raphael Campus in New Haven, Connecticut. "There also is variability across the country in the workup that TIA patients may receive. This may be due to geographic factors, limited resources at health care centers or varying levels of comfort and experience among medical professionals." For example, Amin said, "Someone with a TIA who goes to an emergency room with limited resources may not get the same evaluation that they would at a certified stroke center. This statement was written with those emergency room physicians or internists in mind -- professionals in resource-limited areas who may not have immediate access to a vascular neurologist and must make challenging evaluation and treatment decisions." The statement also includes guidance to help health care professionals tell the difference between a TIA and a "TIA mimic" -- a condition that shares some signs with TIA but is due to other medical conditions such as low blood sugar, a seizure or a migraine. Symptoms of a TIA mimic tend to spread to other parts of the body and build in intensity over time. Who is at risk for a TIA? People with cardiovascular risk factors, such as high blood pressure, diabetes, obesity, high cholesterol and smoking, are at high risk for stroke and TIA. Other conditions that increase risk of a TIA include peripheral artery disease, atrial fibrillation, obstructive sleep apnea and coronary artery disease. In addition, a person who has had a prior stroke is at high risk for TIA. Which tests come first once in the emergency room? After assessing for symptoms and medical history, imaging of the blood vessels in the head and neck is an important first assessment. A non-contrast head CT should be done initially in the emergency department to rule out intracerebral hemorrhage and TIA mimics. CT angiography may be done as well to look for signs of narrowing in the arteries leading to the brain. Nearly half of people with TIA symptoms have narrowing of the large arteries that lead to the brain. A magnetic resonance imaging (MRI) scan is the preferred way to rule out brain injury (i.e., a stroke), ideally done within 24 hours of when symptoms began. About 40% of patients presenting in the ER with TIA symptoms will actually be diagnosed with a stroke based on MRI results. Some emergency rooms may not have access to an MRI scanner, and they may admit the patient to the hospital for MRI or transfer them to a center with rapid access to one. Blood work should be completed in the emergency department to rule out other conditions that may cause TIA-like symptoms, such as low blood sugar or infection, and to check for cardiovascular risk factors like diabetes and high cholesterol. Once TIA is diagnosed, a cardiac work-up is advised due to the potential for heart-related factors to cause a TIA. Ideally, this assessment is done in the emergency department, however, it could be coordinated as a follow-up visit with the appropriate specialist, preferably within a week of having a TIA. An electrocardiogram to assess heart rhythm is suggested to screen for atrial fibrillation, which is detected in up to 7% of people with a stroke or TIA. The American Heart Association recommends that long-term heart monitoring within six months of a TIA is reasonable if the initial evaluation suggests a heart rhythm-related issue as the cause of a TIA or stroke. Early neurology consultation, either in-person or via telemedicine, is associated with lower death rates after a TIA. If consultation isn't possible during the emergency visit, the statement suggests following up with a neurologist ideally within 48 hours but not longer than one week after a TIA, given the high risk of stroke in the days after a TIA. The statement cites research that about 43% of people who had an ischemic stroke (caused by a blood clot) had a TIA within the week before their stroke. Assessing stroke risk after TIA A rapid way to assess a patient's risk of future stroke after TIA is the 7-point ABCD2 score, which stratifies patients into low, medium and high risk based on Age, Blood pressure, Clinical features (symptoms), Duration of symptoms (less than or greater than 60 minutes) and Diabetes. A score of 0-3 indicates low risk, 4-5 is moderate risk and 6-7 is high risk. Patients with moderate to high ABCD2 scores may be considered for hospitalization. Collaboration among emergency room professionals, neurologists and primary care professionals is critical to ensure the patient receives a comprehensive evaluation and a well-communicated outpatient plan for future stroke prevention at discharge. "Incorporating these steps for people with suspected TIA may help identify which patients would benefit from hospital admission, versus those who might be safely discharged from the emergency room with close follow-up," Amin said. "This guidance empowers physicians at both rural and urban academic settings with information to help reduce the risk of future stroke." This scientific statement was prepared by the volunteer writing group on behalf of the American Heart Association's Emergency Neurovascular Care Committee of the Stroke Council and the Council on Peripheral Vascular Disease. The American Academy of Neurology affirms the value of this statement as an educational tool for neurologists, and it is endorsed by the American Association of Neurological Surgeons/Congress of Neurological Surgeons (AANS/CNS). American Heart Association scientific statements promote greater awareness about cardiovascular diseases and stroke issues and help facilitate informed health care decisions. Scientific statements outline what is currently known about a topic and what areas need additional research. While scientific statements inform the development of guidelines, they do not make treatment recommendations. American Heart Association guidelines provide the Association's official clinical practice recommendations. Co-authors are Vice Chair Tracy E. Madsen, M.D., Ph.D.; Dawn M. Bravata, M.D.; Charles R. Wira, M.D.; S. Claiborne Johnston, M.D., Ph.D.; Susan Ashcraft, D.N.P.; Tamika Marquitta Burrus, M.D.; Peter David Panagos, M.D.; Max Wintermark, M.D., M.A.S.; and

Study Enrollment and Dosing to Resume SAN DIEGO, July 19, 2022 /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 the FDA has lifted the clinical hold placed on the clinical trial of VK0214 in patients with X-linked adrenoleukodystrophy (X-ALD). As a result, the company expects to resume study enrollment in the coming weeks. The FDA lifted the clinical hold following its review of results from an in vivo genotoxicity study that it had requested due to the study being conducted with multiple doses in patients. Viking had planned to conduct this study prior to Phase 2 but accelerated its execution based on FDA's request. The study was completed and the results submitted to the Agency in the second quarter. The results showed no evidence of genotoxicity following exposure to VK0214. The company does not believe that the long-term development timeline for VK0214 has been significantly impacted by the temporary clinical hold. "We look forward to resuming study activities and working to complete patient enrollment as quickly as possible," said Brian Lian, Ph.D., chief executive officer of Viking Therapeutics. "We are confident in the overall safety and potential efficacy profile of VK0214 to date, and are eager to continue its advancement as a potential disease modifying treatment for patients with X-ALD." The Phase 1b study of VK0214 is designed to enroll patients with the adrenomyeloneuropathy (AMN) form of X-ALD. AMN is the most common form of X-ALD, affecting approximately 50% of patients. The trial is a multi-center, randomized, double-blind, placebo-controlled study in adult male patients with AMN. The primary objectives of the study are to evaluate the safety and tolerability of VK0214 administered once-daily over a 28-day dosing period. The study also includes an exploratory assessment of the impact of VK0214 on plasma levels of very long chain fatty acids (VLCFAs), as well as an evaluation of the pharmacokinetics of VK0214 in these patients. VK0214 is a novel, orally available small molecule thyroid hormone beta receptor (TRβ) agonist that has been granted orphan drug designation by the FDA for the treatment of X-ALD. Results from a successful Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) study in healthy subjects showed that VK0214 demonstrated encouraging safety and tolerability, as well as predictable pharmacokinetics. In addition, subjects who received VK0214 experienced reductions in low-density lipoprotein cholesterol (LDL-C), triglycerides, and apolipoprotein B following 14 days of treatment. About X-ALD X-ALD is a rare and often fatal metabolic disorder characterized by a breakdown in the protective barriers surrounding brain and nerve cells; a process known as demyelination. The disease, for which there is no approved treatment, is caused by mutations in the gene for a peroxisomal transporter of VLCFAs, known as ABCD1. These mutations lead to dysfunction of the adrenoleukodystrophy protein (ALDP), an important peroxisomal transporter, which prevents patients from efficiently metabolizing VLCFAs. The resulting accumulation of VLCFAs, which is believed to contribute to the onset and progression of the disease, can trigger demyelination of nerve cells, resulting in cognitive impairment, motor skill deterioration, and even death. X-ALD is estimated to occur in approximately 1 in 17,000 births. The thyroid beta receptor is known to regulate expression of a related gene called ABCD2, which encodes a compensatory transporter called the adrenoleukodystrophy related protein (ADLRP). Research in various preclinical disease models has shown that increasing ABCD2 expression can result in normalization of VLCFA levels. About Viking Therapeutics, Inc. Viking Therapeutics 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. Viking's research and development activities leverage its expertise in metabolism to develop innovative therapeutics designed to improve patients' lives. The company's clinical programs include VK2809, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist for the treatment of lipid and metabolic disorders, which is currently being evaluated in a 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 is also developing 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. VK2735 is currently being evaluated in a Phase 1 clinical trial. The company's third clinical compound is VK0214, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist for the potential treatment of X-linked adrenoleukodystrophy (X-ALD). VK0214 is currently being evaluated in a Phase 1b clinical trial in patients with the adrenomyeloneuropathy (AMN) form of X-ALD. The company holds exclusive worldwide rights to a portfolio of five therapeutic programs, including VK2809 and VK0214, which are based on small molecules licensed from Ligand Pharmaceuticals Incorporated. For more information about Viking Therapeutics, please visit . Follow Viking on Twitter @Viking_VKTX. Forward-Looking Statements This press release contains forward-looking statements regarding Viking Therapeutics, Inc., under the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995, including statements about Viking's expectations regarding its clinical and preclinical development programs. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially and adversely and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: risks associated with the success, cost and timing of Viking's product candidate development activities and clinical trials, including those for VK2809, VK2735, VK0214, and the company's other incretin receptor agonists; risks that prior clinical and preclinical results may not be replicated; risks regarding regulatory requirements; and other risks that are described in Viking's most recent periodic reports filed with the Securities and Exchange Commission, including Viking's Annual Report on Form 10-K for the year ended December 31, 2021, and subsequent Quarterly Reports on Form 10-Q, including the risk factors set forth in those filings. These forward-looking statements speak only as of the date hereof. Viking disclaims any obligation to update these forward-looking statements except as required by law. SOURCE Viking Therapeutics, Inc.