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New Seven-Year Follow-Up Phase 2 Study Results Showed Durable Hemostatic Benefit for Adults with Severe Hemophilia A SAN RAFAEL, Calif., Feb. 6, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that new data on ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) will be presented at the 2024 European Association for Haemophilia and Allied Disorders (EAHAD) Congress, Feb. 6-9, 2024. Results from a Phase 2, open-label study of ROCTAVIAN showed that median factor VIII (FVIII) activity at year seven remained in the mild hemophilia range (10.3 IU/dL per chromogenic assay), and mean annualized bleeding rate (ABR) for treated bleeds over the full follow-up period decreased by 96% from baseline for adults with severe hemophilia A in the 6x1013 vg/kg dose cohort. The majority of participants maintained hemostasis, with only two of the seven participants in the study returning to regular prophylaxis, and no new safety signals emerged. These data will be presented as a poster presentation from 6:30-7:30 p.m. Central European Time (CET) on Wednesday, Feb. 7. "People living with severe hemophilia A face a lifelong treatment burden, including frequent injections or infusions and a high risk of health complications like uncontrolled bleeding and irreversible joint damage, which may persist despite good adherence to prophylactic therapy," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We are pleased to present data showing the impact of one-time treatment with ROCTAVIAN over seven years following the infusion, underscoring the potential of gene therapy to make a meaningful and long-lasting impact for people living with severe hemophilia A." Additional studies will be shared during oral presentations on Friday, Feb. 9 from 8:30-10:00 a.m. CET, including preliminary results from GENEr8-INH, a Phase 1/2 study evaluating the safety and efficacy of ROCTAVIAN in participants with active and prior FVIII inhibitors, as well as one-year results from GENEr8-3, a Phase 3b study evaluating the use of prophylactic corticosteroids with ROCTAVIAN treatment. BioMarin's key presentations at EAHAD include: Poster Presentations: Seven-year follow-up of valoctocogene roxaparvovec gene therapy for haemophilia A #PO124 Wednesday, Feb. 7, 2024, 6:30-7:30 p.m. CET Treatment preferences in people with severe haemophilia A: a discrete choice experiment in the United States #PO314 Wednesday, Feb. 7, 2024, 6:30-7:30 p.m. CET Health-related quality of life and long-term joint damage in people with severe haemophilia A in Brazil #PO144 Wednesday, Feb. 7, 2024, 6:30-7:30 p.m. CET Oral Presentations: Safety and efficacy of valoctocogene roxaparvovec in participants with active and prior FVIII inhibitors: preliminary results from GENEr8-INH, a phase 1/2 study #OR10 Friday, Feb. 9, 2024, 8:30-10:00 a.m. CET Safety and efficacy of valoctocogene roxaparvovec with prophylactic corticosteroids: 1-year GENEr8-3 results #OR02 Friday, Feb. 9, 2024, 8:30-10:00 a.m. CET Human liver biopsy analysis reveals lower RNA transcription may contribute to a decline in FVIII levels following AAV5-hFVIII-SQ gene therapy #OR06 Friday, Feb. 9, 2024, 8:30-10:00 a.m. CET About Hemophilia A Hemophilia A, also called factor VIII (FVIII) deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective FVIII, a clotting protein. Although it is passed down from parents to children, about one-third of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A. About ROCTAVIAN ROCTAVIAN is an adeno-associated virus vector-based gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to adeno-associated virus serotype 5 (AAV5), which is determined by a blood test. The one-time infusion works by delivering a functional gene that is designed to enable the body to produce FVIII on its own, reducing the need for ongoing prophylaxis. The European Commission (EC) granted conditional marketing authorization to ROCTAVIAN on August 24, 2022. The U.S. Food and Drug Administration (FDA) approved ROCTAVIAN on June 29, 2023. More information on testing to determine eligibility to receive ROCTAVIAN can be found at in the U.S. and in Germany. U.S. Important Safety Information ROCTAVIAN U.S. Important Safety Information Contraindications: Patients with active infections, either acute (such as acute respiratory infections or acute hepatitis) or uncontrolled chronic (such as chronic active hepatitis B). Patients with known significant hepatic fibrosis (stage 3 or 4 on the Batts-Ludwig scale or equivalent), or cirrhosis, and patients with known hypersensitivity to mannitol. Infusion-related reactions including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during and for at least 3 hours after ROCTAVIAN administration. Administer ROCTAVIAN in a setting where personnel and equipment are immediately available to treat infusion-related reactions. Discontinue infusion for anaphylaxis. Hepatotoxicity: The safety and effectiveness of ROCTAVIAN in patients with hepatic impairment has not been established. Perform liver health assessments prior to administration. The majority of patients treated with ROCTAVIAN experienced ALT elevations and required corticosteroids for ALT elevation. Assess patient's ability to receive corticosteroids and/or other immunosuppressive therapy that may be required for an extended period. Live vaccines should not be administered to patients while on immunosuppressive therapy. Monitor ALT weekly for at least 26 weeks and as clinically indicated, during corticosteroid therapy and institute corticosteroid treatment in response to ALT elevations as required. Continue to monitor ALT until it returns to baseline. Monitor factor VIII activity levels since ALT elevation may be accompanied by a decrease in factor VIII activity. One case of autoimmune hepatitis was reported during third year follow-up in a patient with history of hepatitis C and steatohepatitis. It is recommended that patients abstain from consuming alcohol for at least 1 year after administration and thereafter limit alcohol use. Concomitant medications may cause hepatotoxicity, decrease factor VIII activity, or change plasma corticosteroid levels which may impact liver enzyme elevation and/or factor VIII activity or decrease the efficacy of the corticosteroid regimen or increase their side effects. Closely monitor concomitant medication use including herbal products and nutritional supplements and consider alternative medications in case of potential drug interactions. Thromboembolic events: Factor VIII activity above ULN has been reported following ROCTAVIAN infusion. Thromboembolic events may occur in the setting of elevated factor VIII activity above ULN. Evaluate patients for risk of thrombosis including general cardiovascular risk factors before and after administration of ROCTAVIAN. Advise patients on their individual risk of thrombosis in relation to their factor VIII activity levels above ULN and consider prophylactic anticoagulation. Advise patients to seek immediate medical attention for signs or symptoms indicative of a thrombotic event. Factor VIII inhibitors and Monitoring for inhibitors: The safety and effectiveness of ROCTAVIAN in patients with prior or active factor VIII inhibitors have not been established. Patients with active factor VIII inhibitors should not take ROCTAVIAN. Following administration, monitor patients for factor VIII inhibitors (neutralizing antibodies to factor VIII). Test for factor VIII inhibitors especially if bleeding is not controlled, or plasma factor VIII activity levels decrease. Monitor Factor VIII using the same schedule for ALT monitoring. It may take several weeks after ROCTAVIAN infusion before ROCTAVIAN-derived factor VIII activity rises to a level sufficient for prevention of spontaneous bleeding episodes. Exogenous factor VIII or other hemostatic products may also be required in case of surgery, invasive procedures, trauma, or bleeds. Consider more frequent monitoring in patients with factor VIII activity levels ≤ 5 IU/dL and evidence of bleeding, taking into account the stability of factor VIII levels since the previous measurement. Factor VIII activity produced by ROCTAVIAN in human plasma is higher if measured with one-stage clotting assays compared to chromogenic substrate assays. When switching from hemostatic products prior to ROCTAVIAN treatment, physicians should refer to the relevant prescribing information to avoid the potential for factor VIII activity assay interference during the transition period. Malignancy: The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of hepatocellular carcinoma development. ROCTAVIAN can also insert into the DNA of other human body cells. Monitor patients with risk factors for hepatocellular carcinoma (e.g., hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, non-alcoholic steatohepatitis, advanced age) with regular liver ultrasound (e.g., annually) and alpha-fetoprotein testing for 5 years following ROCTAVIAN administration. In the event that any malignancy occurs after treatment with ROCTAVIAN, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100. Most Common Adverse Reactions: Most common adverse reactions (incidence ≥ 5%) were nausea, fatigue, headache, infusion-related reactions, vomiting, and abdominal pain. Most common laboratory abnormalities (incidence ≥ 10%) were ALT, AST, LDH, CPK, factor VIII activity levels, GGT and bilirubin > ULN. Patients also experienced adverse reactions from corticosteroid use. Isotretinoin, Efavirenz, and HIV Positive Patients: Isotretinoin is not recommended in patients who are benefiting from ROCTAVIAN. Efavirenz is not recommended in patients treated with ROCTAVIAN. Clinical studies of ROCTAVIAN did not include sufficient numbers of patients with HIV to determine whether the efficacy and safety differs compared to patients without HIV infection. Females and Males of Reproductive Potential: ROCTAVIAN is not intended for administration in women. There are no data on the use of ROCTAVIAN in pregnant women or regarding lactation. For 6 months after administration of ROCTAVIAN, men of reproductive potential and their female partners must prevent or postpone pregnancy using an effective form of contraception, and men must not donate semen. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to BioMarin at 1-866-906-6100. Please see the ROCTAVIAN full Prescribing Information for additional Important Safety Information. About BioMarin Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit . Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the data BioMarin plans to present at the 2024 European Association for Haemophilia and Allied Disorders (EAHAD) Congress, including the three oral and three poster presentations; the development of BioMarin's ROCTAVIAN program; the potential impact and benefits of ROCTAVIAN for people with severe hemophilia A; and the continued clinical development of ROCTAVIAN. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of ROCTAVIAN, including achieving adequate market share and reimbursement levels; whether ROCTAVIAN will have the impacts and benefits as anticipated; the results and timing of current and planned preclinical studies and clinical trials of ROCTAVIAN and the release of data from those trials, including continued monitoring of the participants in the clinical trials and post-approval studies; BioMarin's ability to successfully manufacture ROCTAVIAN for the clinical trials and commercially; the content and timing of decisions by the FDA, EU health authorities and other regulatory authorities regarding ROCTAVIAN; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc. ROCTAVIAN™ is a trademark of BioMarin Pharmaceutical Inc. in the United States. Company Codes: NASDAQ-NMS:BMRN

[28-November-2023] ROCTAVIAN is First Gene Therapy for Hemophilia to Receive Approved Federal Price in Germany SAN RAFAEL, Calif., Nov. 28, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced an agreement with the German National Association of Statutory Health Insurance Funds (GKV-SV) on the reimbursement amount for ROCTAVIAN® (valoctocogene roxaparvovec-rvox) for people with severe hemophilia A. This is the first gene therapy for hemophilia to receive an agreed federal price in Germany. ROCTAVIAN brings value to individuals and the healthcare system in Germany, as a one-time, single-dose therapy delivered as an intravenous infusion over 3-4 hours at a gross price of €28,933.53 per vial. This vial price equates to approximately $900,000 per patient in net revenue to BioMarin at the current exchange rate, accounting for average patient weight and number of vials required for the one-time treatment, estimated reductions for customary rebates and discounts. "We are pleased to have reached an agreement on the reimbursement amount for ROCTAVIAN with the GKV-SV, which is responsible for health insurance that covers approximately 90% of the German population," said Jeff Ajer, executive vice president and chief commercial officer at BioMarin. "This important progress will facilitate access to ROCTAVIAN for eligible individuals among the roughly 2,000 adults with severe hemophilia A in Germany and marks a key step forward for the hemophilia community." BioMarin and GKV-SV have agreed on an outcome-based prospective cohort model for ROCTAVIAN. This will allow future reimbursement to be increased or decreased, based on real-world data collected from the German Haemophilia Registry of patients treated with ROCTAVIAN. This agreement, with a minimum term of 3 years, ensures the supply and reimbursement for eligible patients in Germany with ROCTAVIAN by the GKV-SV. Two patients in Germany have now received ROCTAVIAN commercially, and 60 people are eligible for next steps based on companion diagnostic testing results for adeno-associated virus serotype 5 (AAV5) antibodies. Additionally, final price negotiations with the Italian Medicines Agency are underway and expected to be formalized by year-end 2023. Significant Progress with U.S. Payers BioMarin continues to work with private and public payers in the U.S. to enable access, with the goal of ensuring that every eligible adult interested in ROCTAVIAN is able to receive treatment. Numerous coverage policies that now include ROCTAVIAN have been published by leading U.S. payers. While medical exception processes provide a pathway to coverage, these policies representing millions of lives will help increase access to treatment. The company continues to partner with hemophilia treatment centers (HTCs) across the country to educate them on how to administer ROCTAVIAN, and many HTCs have readiness plans before the end of this year. It is estimated that there are approximately 6,500 adults living with severe hemophilia A in the U.S. BioMarin expects approximately 2,500 of those adults to be eligible to receive ROCTAVIAN at this time. About Hemophilia A Hemophilia A, also called factor VIII (FVIII) deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective FVIII, a clotting protein. Although it is passed down from parents to children, about one-third of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A. About ROCTAVIAN ROCTAVIAN is an adeno-associated virus vector-based gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to adeno-associated virus serotype 5 (AAV5), which is determined by a blood test. The one-time infusion works by delivering a functional gene that is designed to enable the body to produce FVIII on its own, reducing the need for ongoing prophylaxis. The European Commission (EC) granted conditional marketing authorization to ROCTAVIAN on August 24, 2022. The U.S. Food and Drug Administration (FDA) approved ROCTAVIAN on June 29, 2023. More information on testing to determine eligibility to receive ROCTAVIAN can be found at in the U.S. and in Germany. U.S. Important Safety Information Contraindications: Patients with active infections, either acute (such as acute respiratory infections or acute hepatitis) or uncontrolled chronic (such as chronic active hepatitis B). Patients with known significant hepatic fibrosis (stage 3 or 4 on the Batts-Ludwig scale or equivalent), or cirrhosis, and patients with known hypersensitivity to mannitol. Infusion-related reactions including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during and for at least 3 hours after ROCTAVIAN administration. Administer ROCTAVIAN in a setting where personnel and equipment are immediately available to treat infusion-related reactions. Discontinue infusion for anaphylaxis. Hepatotoxicity: The safety and effectiveness of ROCTAVIAN in patients with hepatic impairment has not been established. Perform liver health assessments prior to administration. The majority of patients treated with ROCTAVIAN experienced ALT elevations and required corticosteroids for ALT elevation. Assess patient's ability to receive corticosteroids and/or other immunosuppressive therapy that may be required for an extended period. Live vaccines should not be administered to patients while on immunosuppressive therapy. Monitor ALT weekly for at least 26 weeks and as clinically indicated, during corticosteroid therapy and institute corticosteroid treatment in response to ALT elevations as required. Continue to monitor ALT until it returns to baseline. Monitor factor VIII activity levels since ALT elevation may be accompanied by a decrease in factor VIII activity. One case of autoimmune hepatitis was reported during third year follow-up in a patient with history of hepatitis C and steatohepatitis. It is recommended that patients abstain from consuming alcohol for at least 1 year after administration and thereafter limit alcohol use. Concomitant medications may cause hepatotoxicity, decrease factor VIII activity, or change plasma corticosteroid levels which may impact liver enzyme elevation and/or factor VIII activity or decrease the efficacy of the corticosteroid regimen or increase their side effects. Closely monitor concomitant medication use including herbal products and nutritional supplements and consider alternative medications in case of potential drug interactions. Thromboembolic events: Factor VIII activity above ULN has been reported following ROCTAVIAN infusion. Thromboembolic events may occur in the setting of elevated factor VIII activity above ULN. Evaluate patients for risk of thrombosis including general cardiovascular risk factors before and after administration of ROCTAVIAN. Advise patients on their individual risk of thrombosis in relation to their factor VIII activity levels above ULN and consider prophylactic anticoagulation. Advise patients to seek immediate medical attention for signs or symptoms indicative of a thrombotic event. Factor VIII inhibitors and Monitoring for inhibitors: The safety and effectiveness of ROCTAVIAN in patients with prior or active factor VIII inhibitors have not been established. Patients with active factor VIII inhibitors should not take ROCTAVIAN. Following administration, monitor patients for factor VIII inhibitors (neutralizing antibodies to factor VIII). Test for factor VIII inhibitors especially if bleeding is not controlled, or plasma factor VIII activity levels decrease. Monitor Factor VIII using the same schedule for ALT monitoring. It may take several weeks after ROCTAVIAN infusion before ROCTAVIAN-derived factor VIII activity rises to a level sufficient for prevention of spontaneous bleeding episodes. Exogenous factor VIII or other hemostatic products may also be required in case of surgery, invasive procedures, trauma, or bleeds. Consider more frequent monitoring in patients with factor VIII activity levels ≤ 5 IU/dL and evidence of bleeding, taking into account the stability of factor VIII levels since the previous measurement. Factor VIII activity produced by ROCTAVIAN in human plasma is higher if measured with one-stage clotting assays compared to chromogenic substrate assays. When switching from hemostatic products prior to ROCTAVIAN treatment, physicians should refer to the relevant prescribing information to avoid the potential for factor VIII activity assay interference during the transition period. Malignancy: The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of hepatocellular carcinoma development. ROCTAVIAN can also insert into the DNA of other human body cells. Monitor patients with risk factors for hepatocellular carcinoma (e.g., hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, non-alcoholic steatohepatitis, advanced age) with regular liver ultrasound (e.g., annually) and alpha-fetoprotein testing for 5 years following ROCTAVIAN administration. In the event that any malignancy occurs after treatment with ROCTAVIAN, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100. Most Common Adverse Reactions: Most common adverse reactions (incidence ≥ 5%) were nausea, fatigue, headache, infusion-related reactions, vomiting, and abdominal pain. Most common laboratory abnormalities (incidence ≥ 10%) were ALT, AST, LDH, CPK, factor VIII activity levels, GGT and bilirubin > ULN. Patients also experienced adverse reactions from corticosteroid use. Isotretinoin, Efavirenz, and HIV Positive Patients: Isotretinoin is not recommended in patients who are benefiting from ROCTAVIAN. Efavirenz is not recommended in patients treated with ROCTAVIAN. Clinical studies of ROCTAVIAN did not include sufficient numbers of patients with HIV to determine whether the efficacy and safety differs compared to patients without HIV infection. Females and Males of Reproductive Potential: ROCTAVIAN is not intended for administration in women. There are no data on the use of ROCTAVIAN in pregnant women or regarding lactation. For 6 months after administration of ROCTAVIAN, men of reproductive potential and their female partners must prevent or postpone pregnancy using an effective form of contraception, and men must not donate semen. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to BioMarin at 1-866-906-6100. Please see the ROCTAVIAN full Prescribing Information for additional Important Safety Information. About BioMarin Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit . Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the potential impact of the agreement with the German National Association of Statutory Health Insurance Funds (GKV-SV) on the reimbursement amount for ROCTAVIAN, including ROCTAVIAN's ability to bring value to individuals and the healthcare system in Germany, the estimated per patient net revenue of ROCTAVIAN to BioMarin in Germany, and BioMarin's ability to facilitate access to ROCTAVIAN for eligible individuals in Germany; the impact of the outcome-based prospective cohort model for ROCTAVIAN in Germany, including the ability to increase or decrease future reimbursements and to ensure the supply and reimbursement for eligible patients in Germany; BioMarin's expectations regarding the number of patients who will be eligible to receive ROCTAVIAN in Germany and the United States; BioMarin's expectations regarding its negotiations with the Italian Medicines Agency for the final price of ROCTAVIAN, including the expected timing of formalization; BioMarin's ability to ensure that every eligible adult interested in ROCTAVIAN is able to receive treatment; the ability of the published U.S. coverage policies including ROCTAVIAN to increase access to treatment; and the ongoing clinical development of ROCTAVIAN. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of ROCTAVIAN, including achieving adequate market share and reimbursement levels; whether ROCTAVIAN will have the impacts and benefits as anticipated; the results and timing of current and planned preclinical studies and clinical trials of ROCTAVIAN and the release of data from those trials, including continued monitoring of the participants in the clinical trials and post-approval studies; BioMarin's ability to successfully manufacture ROCTAVIAN for the clinical trials and commercially; the content and timing of decisions by the FDA, EU health authorities and other regulatory authorities regarding ROCTAVIAN; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc. ROCTAVIAN® is a trademark of BioMarin Pharmaceutical Inc., with registration in Europe and pending in the U.S. Company Codes: NASDAQ-NMS:BMRN

This is the longest Niagen NR clinical trial to date spanning two years and showcases that long-term Niagen NR is safe and well tolerated LOS ANGELES--(BUSINESS WIRE)-- ChromaDex, Inc. (NASDAQ:CDXC), one of the global authorities on Nicotinamide Adenine Dinucleotide (NAD+) and healthy aging research, shares results from a phase II clinical study published in the peer-reviewed journal, Movement Disorders, by a team of researchers led by Dr. Hilde Nilsen, University of Oslo, Arkershus University Hospital, Norway. The clinical trial was part of the ChromaDex External Research Program (CERP™), which donated ChromaDex’s patented nicotinamide riboside (NR) ingredient, Niagen®, one of the most efficient and superior NAD+ precursors available, for the advancement of this research. The promising results demonstrate that long-term supplementation with Niagen NR effectively increased whole blood NAD+ levels up to fourfold, improved coordination, and enhanced eye movement while maintaining biomarkers of stable liver and kidney function in Ataxia Telangiectasia (AT) patients. AT is a rare, inherited neurodegenerative disorder characterized by premature aging, cerebellar degeneration, immunodeficiency, and cancer predisposition. This press release features multimedia. View the full release here: Newly published phase II clinical study demonstrates that supplementation with Niagen, patented nicotinamide riboside (NR), elevates NAD+ up to fourfold, improving motor coordination and eye movement in Ataxia Telangiectasia (AT) patients (Graphic: Business Wire) Building on a growing body of research, this is the second published study (Veenhuis et al. in 2021) analyzing the therapeutic effects of Niagen NR supplementation in AT patients and marks a milestone as it is the longest Niagen NR clinical trial to date, spanning two years, with results showcasing that long-term supplementation is safe and well tolerated. "NR supplementation has shown promising potential in supporting brain health and cognition,” said Dr. Andrew Shao the Vice President of Scientific & Regulatory Affairs at ChromaDex. “Independent research through CERP suggests that NR may play a role in maintaining neuronal function and promoting cognitive well-being by supporting NAD+ levels in brain cells." Individuals diagnosed with AT frequently experience profound challenges in motor function, characterized by neurodegenerative conditions like cerebellar ataxia, resulting in impaired movement and coordination and they may exhibit oculomotor apraxia, difficulties controlling eye movements. These factors often result in a loss of independent walking ability, typically necessitating the use of a wheelchair by the age of ten. Dr. Nilsen remarks, “While the exact mechanisms underlying cerebellar degeneration and neurodegeneration in AT are not fully understood, research indicates inability to repair and respond appropriately to DNA damage. AT research has shown that DNA damage depletes NAD+, the crucial cellular coenzyme that plays an essential role in mitochondrial function, DNA repair and cellular energy production. This growing body of research is promising as it demonstrates that NR supplementation may be a potential therapeutic strategy for AT patients.” About the study This two year phase II clinical study was a single-arm open-label intervention trial investigating the safety and efficacy of long-term NR supplementation in 10 patients with AT (6 were female, 4 were male, of which 7 were children, 3 were adults), between the ages of 3 to 27 years. The patients were given oral NR as an intervention starting with an initial dose of 150 mg/day, gradually increasing to 300 mg/day, and reaching a final dose of 500 mg/day. Study highlights NR supplementation was well tolerated, with no serious adverse events. NR supplementation increased whole blood NAD+ levels up to fourfold. Serum levels of AFP, serum alpha-fetoprotein and Nfl, neurofilament light chain, biomarkers of AT progression, remained stable for most participants over two years of NR supplementation, suggesting that NR may slow or delay progression of AT. Long-term NR supplementation reduced ataxia symptoms and improved motor coordination, eye movements, and overall neurological functioning: Positive correlations were observed between NR supplementation and improved AT Neurological Examination Toolkit (NEST) total score, eye movements, SARA coordination scores, ICARS coordination scores, and oculomotor scores. Biomarkers related to liver and kidney function did not show clinically significant changes during the two-year treatment period, indicating the NR supplementation was safe. Long-term blood glucose regulation, measured as HbA1c level, remained stable in all participants. NR supplementation did not cause changes in hematological or immunological parameters. Relevance Reaching a milestone in NR clinical research as the longest published clinical study to date, results of this study demonstrate that NR effectively increased whole blood NAD+ levels up to fourfold, improved coordination, and enhanced eye movement in both adults and children with AT, while maintaining biomarkers of stable liver and kidney function. The outcomes of this study strongly support the inclusion of NR supplementation as a standard clinical approach for AT, however more research needs to be conducted to confirm the impact of NR on motor skills such as standing and walking. For additional information on the science supporting Niagen® visit . About ChromaDex: ChromaDex Corp. is a global bioscience company dedicated to healthy aging. The ChromaDex team, which includes world-renowned scientists, is pioneering research on nicotinamide adenine dinucleotide (NAD+), levels of which decline with age. ChromaDex is the innovator behind NAD+ precursor nicotinamide riboside (NR), commercialized as the flagship ingredient Niagen®. Nicotinamide riboside and other NAD+ precursors are protected by ChromaDex’s patent portfolio. ChromaDex maintains a website at to which ChromaDex regularly posts copies of its press releases as well as additional and financial information about the Company. Forward-Looking Statements: This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended, including statements related to whether the results demonstrate that long-term supplementation with Niagen NR effectively increased whole blood NAD+ levels up to fourfold, improved coordination, and enhanced eye movement while maintaining biomarkers of stable liver and kidney function in Ataxia Telangiectasia (AT) patients, whether the results showcase that long-term supplementation is safe and well tolerated, whether NR supplementation has shown promising potential in supporting brain health and cognition, and whether this growing body of research is promising or if it demonstrates that NR supplementation may be a potential therapeutic strategy for AT patients. Statements that are not a description of historical facts constitute forward-looking statements and may often, but not always, be identified by the use of such words as "expects," "anticipates," "intends," "estimates," "plans," "potential," "possible," "probable," "believes," "seeks," "may," "will," "should," "could" or the negative of such terms or other similar expressions. Risks that contribute to the uncertain nature of these forward-looking statements include the impact of the COVID-19 pandemic on our business and the global economy; our history of operating losses and need to obtain additional financing; the growth and profitability of our product sales; our ability to maintain sales, marketing and distribution capabilities; changing consumer perceptions of our products; our reliance on a single or limited number of third-party suppliers; and the risks and uncertainties associated with our business and financial condition. More detailed information about ChromaDex and the risk factors that may affect the realization of forward-looking statements is set forth in ChromaDex's Annual Report on Form 10-K for the fiscal year ended December 31, 2021, ChromaDex's Quarterly Reports on Form 10-Q and other filings submitted by ChromaDex to the SEC, copies of which may be obtained from the SEC's website at . Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and actual results may differ materially from those suggested by these forward-looking statements. All forward-looking statements are qualified in their entirety by this cautionary statement and ChromaDex undertakes no obligation to revise or update this release to reflect events or circumstances after the date hereof.