In this study, we are trying to see if vitamin B6 can minimize the amplified blood pressure response to exercise following ischemia-reperfusion injury. We are interested in a protein called P2X3, of which function can be blocked by vitamin B6, in the neurons of our nervous system. It is very important for blood pressure regulation. We would like to see if the P2X3 plays a role in patients' rising blood pressure during exercise. The results of the proposed studies will provide a base for those two potential economic and non-invasive inventions to improve the overall health and well-being of PAD patients.

Start Date01 Dec 2025

Sponsor / Collaborator

Hershey Medical Center

[+1]

CTRI/2024/07/071163 / Not yet recruitingPhase 2IIT

Efficacy and safety of Pyridoxine as add-on therapy in prevention of chemotherapy induced nausea and vomiting in patients of Cancer Breast receiving Epirubicin and Cyclophosphamide (PiN study):A Phase II, single arm study - PiN

Start Date31 Jul 2024

Sponsor / Collaborator

Jawaharlal Institute of Postgraduate Medical Education and Research

NCT05994742 / RecruitingPhase 3

Co-SAM: An Adaptive Multi-arm Trial to Improve Clinical Outcomes Among Children Recovering From Complicated Severe Acute Malnutrition

Malnutrition underlies 45% of child deaths, and has far-reaching educational, economic and health consequences. Severe acute malnutrition (SAM) affects 17 million children globally and is the most life-threatening form of malnutrition. Community-based management of acute malnutrition using ready-to-use therapeutic food (RUTF) has transformed outcomes for children with uncomplicated SAM, but those presenting with poor appetite or medical complications (categorised as having 'complicated' SAM) require hospitalisation. Data show that pneumonia, diarrhoea and malaria are leading causes of death in children with complicated SAM after discharge from hospital. High risk of infectious deaths suggests that sustained antimicrobial interventions may reduce mortality following discharge from hospital. Furthermore, children with complicated SAM respond less well to nutritional rehabilitation, and oftentimes are discharged to a home environment characterised by poverty and multiple caregiver vulnerabilities including depression, low decision making autonomy, lack of social support, gender-restricted family relations, and competing demands on scarce resources. Caregivers have to navigate diverse challenges that impede engagement with clinical care after discharge from hospital. The objective is to address the biological and social determinants of multimorbidity in children with complicated SAM by developing multimodal packages of interventions and testing them in a 5-arm adaptive randomized controlled clinical trial, with death/hospitalization or failed nutritional recovery as the primary outcome.

Start Date15 Jul 2024

Sponsor / Collaborator

Queen Mary University of London

[+6]

100 Clinical Results associated with Pyridoxine Hydrochloride

100 Translational Medicine associated with Pyridoxine Hydrochloride

100 Patents (Medical) associated with Pyridoxine Hydrochloride

10,775

Literatures (Medical) associated with Pyridoxine Hydrochloride

01 Feb 2025·Infectious Disorders - Drug Targets

Vigilance Needed in Treating a Child with Disseminated TB: A Case Report

Author: Neelambaram, Krishnapriya ; Velmurugan, Hemasri ; Thangaraju, Pugazhenthan ; Chouhan, Dushyant ; Gaikwad, Nitin Rewaram ; Gurunthalingam, Meenalotchini Prakash ; Keshari Kar, Bikram

Background:Tuberculosis is still one of the biggest causes of infection-related death around the world. Disseminated tuberculosis is a potentially fatal disease caused by the haematogenous spread of Mycobacterium tuberculosis. First-line anti-tuberculosis drugs in-clude isoniazid, rifampicin, pyrazinamide, and ethambutol. The first three drugs are known to cause hepatotoxicity.Case Presentation:We have, herein, reported a case of Drug-induced Liver Injury (DILI) due to anti-tuberculosis therapy in a one-year-old male child with disseminated tuberculosis. He was started on a fixed-dose combination of Anti-tuberculosis Therapy (ATT; isoniazid 50 mg, rifampicin 75 mg, and pyrazinamide 150 mg) and pyridoxine 10 mg orally. Initially, liver pa-rameters were normal, but later on with the course of the treatment, there was a rapid rise in liver enzymes, suggesting liver injury.Discussion:The association between liver injury and anti-tuberculosis therapy has been con-firmed by applying various causality association scales. It is obvious that proper treatment of disseminated tuberculosis can avoid the development of drug-resistant strains that can be harm-ful, worsening the prognosis as there are fewer therapeutic alternatives available. At the same time, there is a need to monitor the patient with ATT-induced DILI.Conclusion:The diagnosis of tuberculosis in children is difficult because of the mild, nonspe-cific clinical presentation, which usually reflects the implicated underlying organ. In addition to prompt diagnosis and treatment of disseminated TB, careful monitoring is equally important.

01 Jan 2025·Journal of Ethnopharmacology

Protective effects and mechanisms of HuDiChangRong capsule on TNBS-induced ulcerative colitis in mice

Article

Author: Chen, Shengnan ; Wei, Feng ; Zhang, Dongqi ; Xu, Beilei ; Liu, Jingjing ; Sun, Wenbin ; Ma, Shuangcheng ; Liu, Jing ; Liu, Shusen

ETHNOPHARMACOLOGICAL RELEVANCEUC, characterized by chronic inflammation primarily affecting the colon and rectum, follows a protracted relapsing course marked by inflammation and an abundance of free radicals at the onset. Hudichangrong Capsule (HDCRC), a traditional Chinese medicinal formula, has long been employed in the treatment of UC and chronic bacillary dysentery, exhibiting positive therapeutic outcomes and a high rate of cure in clinical practice.AIM OF THE STUDYThe precise mechanism underlying its efficacy for UC remains elusive. Our objective was to investigate the anti-inflammatory effect and underlying mechanisms of HDCRC on TNBS-induced UC.MATERIALS AND METHODSHere, we introduced HDCRC and induced UC using TNBS. SPF BALB/c mice were divided into 6 groups as follows: control group, colitis model group, colitis treated with sulfasalazine (400 mg/kg) group, and colitis treated with HDCRC (156, 312, and 624 mg/kg) groups. To assess the effects of HDCRC on colitis, we measured body weight loss, disease activity index (DAI), colon length, tissue damage, degree of inflammation, immune capacity, and oxidative stress. Additionally, we evaluated the TLR-4/MyD88 pathway and its downstream signaling using immunohistochemistry, real-time qPCR, and Western blot. Network pharmacology was used for main target prediction. 16s rRNA was employed for gut microbiota detechtion and UPLC-QTOF-MS was used for its and its metabonomics.RESULTSHDCRC significantly slowed weight loss, ameliorated DAI, restored colon length, alleviated TNBS-induced tissue damage. It exerted the therapeutic effects via reducing oxidative stress, restoring immune balance, normalizing the inflammatory mediator levels and restoring intestinal barrier integrity. Furthermore, HDCRC mainly alleviate UC via suppressing the TLR-4/MyD88 pathway and its downstream signaling. The key components of the downstream pathway, including TLR-4, MyD88, NF-κB p65, ERK, p-JNK, p38, p-JAK1, JAK1, p-STAT3, and STAT3, were improved, thereby ameliorating the TNBS-induced injury. In addition, HDCRC could regulate gut microbiota (eg. Erysipelaloclostridium,etc.) and its metabonomics (eg. Vitamin B6 metabolism) in UC mice.CONCLUSIONSIn conclusion, HDCRC exerts a protective effect against TNBS-induced UC in mice by inhibiting the TLR-4/MyD88 pathway and its downstream signaling, and partially JAK1/STAT3, suppressing oxidative stress, regulating immunity, restoring intestinal barrier integrity, and regulating gut microbiota and its metabonomics.

01 Jan 2025·Food Chemistry

Omics analysis of key pathway in flavour formation and B vitamins synthesis during chickpea milk fermentation by Lactiplantibacillus plantarum

Article

Author: Huang, Jian ; Li, Xu ; Fan, Jinxin ; Ni, Yongqing ; Dong, Li ; Luo, Jingying ; Tian, Fengwei ; Lu, Yanzhuan

Chickpea milk is a nutrient-rich plant-based milk, but its pronounced beany flavour limits consumer acceptance. To address this issue, chickpea milk was fermented using two strains of Lactiplantibacillus plantarum, FMBL L23251 and L23252, which efficiently utilize chickpea milk. L. plantarum FMBL L23251 demonstrated superior fermentation characteristics. Fermentation with L. plantarum FMBL L23251 resulted in a 1.90-fold increase in vitamin B3 (271.66 ng/ml to 516.15 ng/ml) and a 1.58-fold increase in vitamin B6 (91.24 ng/ml to 144.16 ng/ml) through the L-aspartic acid pathway and the 1-deoxy-D-xylulose-5-phosphate (DXP)-independent pathway, respectively. Furthermore, L. plantarum FMBL L23251 effectively removed beany flavours due to its enhanced pathway for pyruvate metabolism. The main aldehydes are converted into corresponding alcohols or acids, resulting in 87.74 % and 96.99 % reductions in hexanal and 2-pentyl-furan, respectively. In summary, the fermentation of L. plantarum FMBL L23251 generated fermented chickpea milk that is rich in B vitamins and provides a better flavour.

News (Medical) associated with Pyridoxine Hydrochloride

10 Oct 2024

·www.prnewswire.com

Duchesnay Pharmaceutical Group celebrates expansion of its treatment portfolio for nausea and vomiting of pregnancy to 40 countries around the world

Achievement highlights continued growth of Quebec-based business BLAINVILLE, QC, Oct. 10, 2024 /PRNewswire/ - Duchesnay Pharmaceutical Group (DPG), one of the eight companies across the country chosen to participate in the Government of Canada's Global Hypergrowth Project, is very proud to announce that its treatment portfolio for nausea and vomiting of pregnancy (NVP) global footprint has now expanded to include additional regulatory approvals in over 40 countries around the world. Continue Reading DPG celebrates expansion of its NVP treatment portfolio to 40 countries around the world (CNW Group/Duchesnay Pharmaceutical Group (DPG)) Developed, patented and manufactured locally at DPG's state-of-the-art facility in Blainville, Quebec, this unique product portfolio consists of the combination of doxylamine succinate and pyridoxine hydrochloride, available in different strengths and formulations under different trade names (such as Xonvea® and Bonjesta®) around the world. "This portfolio has a 40-year history of safe use by pregnant women to manage and treat NVP, often referred to as 'morning sickness,'" said Michael Gallo, Vice-President, Regulatory & Medical Affairs, Duchesnay Pharmaceutical Group. "In fact, the recognition of nausea and vomiting of pregnancy as a medical condition is a result of the efforts of the Duchesnay team, who have pioneered the scientific landscape around this serious condition since the early 2000s." This combination of doxylamine succinate and pyridoxine hydrochloride has already received market authorization in an additional 13 countries in 2024 with new approvals expected in the short term and for several years to come. "We are very proud of this accomplishment, and it would not be possible without the commitment of our teams and the collaboration and dedication of our international partners,'' said Suzanne Paszkiewicz, Vice-President, Global Partnerships & Business Development, Duchesnay Pharmaceutical Group. "The issue of NVP and the lack of approved treatment options is a universal one, therefore we are committed to continuing to address this medical need around the world." ABOUT DUCHESNAY PHARMACEUTICAL GROUP Duchesnay Pharmaceutical Group (DPG), with its affiliated companies, is headquartered in Blainville, Quebec. The Group consists of six 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; Medunik Canada and Medunik USA, which provide treatments for rare and debilitating diseases; and Analog Pharma Canada and Analog Pharma, specializing in generic medications. From its state-of-the-art manufacturing plant, DPG exports its innovative treatments to more than 50 countries. DPG is one of the eight companies across the country chosen to participate in the Government of Canada's Global Hypergrowth Project. This appointment offers exclusive and personalized support for at least two years, in order to accelerate its growth to become an anchor firm in the Canadian economy. DPG, 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 and for rare and debilitating diseases, plus lower-cost generic medications. DPG recognizes the dedication and professionalism of its employees and promotes a positive 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, please visit . SOURCE Duchesnay Pharmaceutical Group (DPG) WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug Approval

26 Sep 2024

·www.globenewswire.com

Travere Therapeutics Announces Voluntary Pause of Enrollment in the Phase 3 HARMONY Study of Pegtibatinase Related to Commercial Manufacturing Scale-Up

Company to host conference call today at 4:30 p.m. ETSAN DIEGO, Sept. 26, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced a voluntary pause of enrollment in the Phase 3 HARMONY Study evaluating pegtibatinase for the treatment of classical homocystinuria (HCU). The voluntary enrollment pause enables the Company to work to address necessary process improvements in manufacturing scale-up to support commercial scale manufacturing as well as full enrollment in the HARMONY Study. Patients currently enrolled in pegtibatinase studies continue to receive study medication from small scale batches which are unaffected by the scale-up process. Currently enrolled patients will be able to continue on study medication as scheduled for the duration of the trials they are participating in. The voluntary enrollment pause was enacted following the Company’s determination that the desired drug substance profile was not achieved in the recent scale-up process. The Company is in the process of notifying all study investigators of the decision to pause enrollment of new patients into the study until additional material is available. “We believe the potential for pegtibatinase to become the first disease-modifying therapy for classical HCU remains unchanged. Our internal team and external manufacturing partners have extensive biologics expertise, and we are confident that we will be able to make the necessary manufacturing process improvements to continue with this pivotal program. We remain committed to supporting the patients currently enrolled in our clinical trials while working with our manufacturing partners to optimize our commercial-scale manufacturing process,” said Bill Rote, Ph.D., senior vice president of research and development at Travere Therapeutics. The Company expects to further evaluate the necessary commercial process improvements to enable the continuation of the Phase 3 program and anticipates the earliest date to restart enrollment in the Phase 3 HARMONY Study will be in 2026. Previously planned investments related to clinical enrollment in HARMONY and large-scale production are expected to be delayed beyond 2025. Together with the expected decline in costs associated with the development of sparsentan as the Phase 3 programs advance towards completion, the Company now estimates that research and development expenses may be reduced by more than $30 million in 2025 compared to 2024. The Company continues to anticipate that its cash, cash equivalents and marketable securities of $325.4 million as of June 30, 2024 can support its operations into 2028. Conference call information Travere Therapeutics will host a conference call and webcast today, Thursday, September 26, 2024 at 4:30 p.m. ET to discuss the voluntary pause in enrollment. To participate in the conference call, dial +1 (323) 994-2093 (U.S.) or +1 (888) 394-8218 (International), confirmation code 5880214. The webcast can be accessed on the Investor page of Travere’s website at ir.travere.com/events-presentations. Following the live webcast, an archived version of the call will be available for 30 days on the Company’s website. About Classical Homocystinuria Classical homocystinuria (HCU) is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). CBS is a pivotal enzyme that is essential for the management of methionine and cysteine in the body. Classical HCU leads to toxic levels of homocysteine that can result in life-threatening thrombotic events such as stroke, pulmonary embolism and deep vein thrombosis, ophthalmologic and skeletal complications, as well as developmental delay. Current treatment options are limited to protein-restricted diet and use of vitamin B6 and betaine. About Pegtibatinase Pegtibatinase is an investigational PEGylated, recombinant enzyme replacement therapy designed to address the underlying cause of classical HCU. In preclinical studies, pegtibatinase has demonstrated an ability to reduce total homocysteine levels and improve clinical parameters. In December 2023 the Company initiated the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase for the treatment of classical HCU. The HARMONY Study is a global, randomized, multi-center, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate the efficacy and safety of pegtibatinase as a novel treatment to reduce total homocysteine (tHcy) levels. In May 2023 the Company announced that data from four patients treated with the highest dose of pegtibatinase in the Phase 1/2 COMPOSE Study showed a clinically meaningful 67.1% mean relative reduction in total homocysteine from baseline and was generally well-tolerated after 12 weeks of treatment. To date, the pegtibatinase program has been granted Breakthrough Therapy designation, Rare Pediatric Disease and Fast Track designations by the FDA, as well as Orphan Drug designation in the U.S. and Europe. About Travere Therapeutics At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com. Forward-Looking Statements This press release contains “forward-looking statements” as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words “on-track,” “positioned,” “look forward to,” “will,” “would,” “may,” “might,” “believes,” “anticipates,” “plans,” “expects,” “intends,” “potential,” or similar expressions. In addition, expressions of strategies, intentions or plans are also forward-looking statements. Such forward-looking statements include, but are not limited to, references to: the potential for pegtibatinase to become the first disease-modifying therapy for classical HCU; statements and expectations regarding future process improvements in manufacturing scale-up to support commercial scale manufacturing as well as full enrollment in the HARMONY Study, and related timing expectations; statements regarding ongoing clinical trials, including statements regarding medication for patients currently enrolled in such trials; and statements and expectations related to future research and development expenses and the expected timing thereof, including with respect to investments related to clinical enrollment in HARMONY and large scale production, and the expected decline in costs associated with the development of sparsentan as the Phase 3 programs advance towards completion; and expectations regarding cash runway. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with manufacturing processes and improvements, and risks related to the regulatory review and approval process, as well as risks and uncertainties associated with the Company’s business and finances in general, the success of its commercial products and risks and uncertainties associated with its preclinical and clinical stage pipeline. Specifically, the Company faces risks associated with the challenges of manufacturing scale-up, the ongoing commercial launch of FILSPARI, market acceptance of its commercial products including efficacy, safety, price, reimbursement, and benefit over competing therapies, as well as risks associated with the successful development and execution of commercial strategies for such products. The risks and uncertainties the Company faces with respect to its preclinical and clinical stage pipeline include risk that the Company’s clinical candidates will not be found to be safe or effective and that current or anticipated future clinical trials will not proceed as planned. The Company also faces the risk that its cash runway might not last as long as currently anticipated and the risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates, including as a result of macroeconomic conditions; risks relating to the Company’s dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; and risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of the Company’s products, and technological changes that may limit demand for the Company’s products. The Company also faces additional risks associated with global and macroeconomic conditions, including health epidemics and pandemics, including risks related to potential disruptions to clinical trials, commercialization activity, supply chain, and manufacturing operations. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties, including under the heading “Risk Factors”, as included in the Company’s most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission. Contact Info Media:888-969-7879 mediarelations@travere.comInvestors:888-969-7879 IR@travere.com

Phase 3Fast TrackBreakthrough TherapyOrphan DrugClinical Result

22 Aug 2024

·www.globenewswire.com

Travere Therapeutics to Present Abstracts at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2024

Posters to be presented highlight clinical study designs investigating pegtibatinase as the first potential disease-modifying treatment for classical homocystinuriaSAN DIEGO, Aug. 22, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present two posters in classical homocystinuria (HCU) at the Society for the Study of Inborn Errors of Metabolism (SSIEM) annual symposium in Porto, Portugal, September 3-6, 2024. At SSIEM, the Company will present the trial designs of the pivotal Phase 3 HARMONY Study and ENSEMBLE long-term extension study of pegtibatinase, a novel investigational enzyme replacement therapy for the treatment of classical HCU. Additionally, the Company will share the trial design of cohort 7 in the Phase 1/2 COMPOSE Study of pegtibatinase in pediatric participants with classical HCU. “We believe that pegtibatinase has the potential to effectively replace the deficient CBS enzyme activity, and that it could ultimately become the first disease-modifying therapy for people living with classical HCU,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics. “We look forward to sharing the study designs of our innovative clinical programs in this rare metabolic disorder at the SSIEM symposium this year.” SSIEM Poster PresentationsRosa Mota Pavilion, Porto, Portugal Pegtibatinase, an Investigational Enzyme Replacement Therapy for ClassicalHomocystinuria (HCU): design of the HARMONY and ENSEMBLE Phase 3 studies Poster: EP-013Abstract Category: Clinical studies, patient-reported outcome measures Wednesday, September 4, 2024, 6:15 – 8:15 p.m. GMT+1 COMPOSE Phase 1/2, Dose-Escalation Cohort 7 Design: safety of pegtibatinase inchildren aged ≥5–<12 years with classical homocystinuria (HCU)e-Poster: PO-072Abstract Category: Clinical studies, patient reported outcome measures Wednesday, September 4, 2024, 6:15 – 8:15 p.m. GMT+1 About Classical Homocystinuria Classical homocystinuria (HCU) is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). CBS is a pivotal enzyme that is essential for the management of methionine and cysteine in the body. Classical HCU leads to toxic levels of homocysteine that can result in life-threatening thrombotic events such as stroke, pulmonary embolism and deep vein thrombosis, ophthalmologic and skeletal complications, as well as developmental delay. Current treatment options are limited to protein-restricted diet and use of vitamin B6 and betaine. About Pegtibatinase Pegtibatinase is an investigational PEGylated, recombinant enzyme replacement therapy designed to address the underlying cause of classical HCU. In preclinical studies, pegtibatinase has demonstrated an ability to reduce total homocysteine levels and improve clinical parameters. In December 2023 the Company initiated the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase for the treatment of classical HCU. The HARMONY Study is a global, randomized, multi-center, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate the efficacy and safety of pegtibatinase as a novel treatment to reduce total homocysteine (tHcy) levels. In May 2023 the Company announced that data from four patients treated with the highest dose of pegtibatinase in the Phase 1/2 COMPOSE Study showed a clinically meaningful 67.1% mean relative reduction in total homocysteine from baseline and was generally well-tolerated after 12 weeks of treatment. To date, the pegtibatinase program has been granted Breakthrough Therapy designation, Rare Pediatric Disease and Fast Track designations by the FDA, as well as Orphan Drug designation in the U.S. and Europe. About Travere Therapeutics At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com Forward Looking Statements This press release contains “forward-looking statements” as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words “on-track,” “positioned,” “look forward to,” “will,” “would,” “may,” “might,” “believes,” “anticipates,” “plans,” “expects,” “intends,” “potential,” or similar expressions. In addition, expressions of strategies, intentions or plans are also forward-looking statements. Such forward-looking statements include, but are not limited to, references to: the potential for pegtibatinase to effectively replace the deficient CBS enzyme activity, the potential for pegtibatinase to ultimately become the first disease-modifying therapy for people living with classical HCU, and statements relating to clinical studies, including but not limited to trial design, anticipated results and timing related thereto. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks related to the timing and outcome of the studies described herein and uncertainties associated with the regulatory review and approval process, as well as risks and uncertainties associated with enrollment of clinical trials for rare diseases, and risks that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. The Company also faces risks related to its business and finances in general, the success of its commercial products and risks and uncertainties associated with its preclinical and clinical stage pipeline. Specifically, the Company faces risks associated with the ongoing commercial launch of FILSPARI, market acceptance of its commercial products including efficacy, safety, price, reimbursement, and benefit over competing therapies, as well as risks associated with the successful development and execution of commercial strategies for such products, including FILSPARI. The risks and uncertainties the Company faces with respect to its preclinical and clinical stage pipeline include risk that the Company’s clinical candidates will not be found to be safe or effective and that current or anticipated future clinical trials will not proceed as planned. Specifically, the Company faces risks related to the timing and potential outcome of the studies described herein, and the timing and potential outcome of the FDA’s review of the Company’s sNDA submission for full approval of FILSPARI in IgAN. There is no guarantee that regulators will grant full approval of sparsentan for IgAN or FSGS. The Company also faces the risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates, including as a result of macroeconomic conditions; risks relating to the Company’s dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; and risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of the Company’s products, and technological changes that may limit demand for the Company’s products. The Company also faces additional risks associated with global and macroeconomic conditions, including health epidemics and pandemics, including risks related to potential disruptions to clinical trials, commercialization activity, supply chain, and manufacturing operations. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties, including under the heading “Risk Factors”, as included in the Company’s most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission. Contact Info Media:888-969-7879mediarelations@travere.comInvestors:888-969-7879IR@travere.com

Phase 3Breakthrough TherapyOrphan DrugFast Track

100 Deals associated with Pyridoxine Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
-	Pyridoxine Hydrochloride	A11HA02	DB00165

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Vitamin B 6 Deficiency	US	-	-

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Progressive pulmonary fibrosis	Phase 2	CN	Shanxi Bethune Hospital	01 Mar 2024

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05024422 (Pubmed \| Am J Obstet Gynecol)	Phase 3	Breast Feeding	-	Cabergoline	ksrarsoyvb(ximaoiqvzh) = okfoqxucbj kokiklkinr (jkvpeijsdw ) View more	Positive	01 May 2024
				Pyridoxine	ksrarsoyvb(ximaoiqvzh) = zcxhgvaeau kokiklkinr (jkvpeijsdw ) View more
EADV2023	Not Applicable	-	61	Oral biotin	vjrqjhtlld(lykhijyljq) = nwbgodcsij kmxoqtxvtf (mtkommnmno ) View more	Positive	11 Oct 2023
				Pyridoxine	vjrqjhtlld(lykhijyljq) = dkpalikckt kmxoqtxvtf (mtkommnmno ) View more
Pubmed	Not Applicable	-	5,612	Vitamin B6 (Older adults (≥65y))	clubukfkko(lsfgdkwfcl) = zkozoudzbz smvzswsntw (jkjfawfdwu )	-	20 Oct 2022
				Vitamin B6 (Younger adults (<65y))	clubukfkko(lsfgdkwfcl) = bjntcguhcu smvzswsntw (jkjfawfdwu )
Alienor Study (ARVO2022)	Not Applicable	Age Related Macular Degeneration	963	Vitamin B5	aotufwdkfk(apgmngmgbx) = eojwrvludy spqwsjipdu (jlymxjzahv, 1.84mg)	-	01 May 2022
				Vitamin B6	aotufwdkfk(apgmngmgbx) = vsgyolhsrs spqwsjipdu (jlymxjzahv, 0.60mg)
compassionate use (ASN2021)	Not Applicable	Primary Hyperoxaluria Type 1 Maintenance	2	Pyridoxine	(adgqdpicev) = tdxeuuveit jwllatsqnu (vujjolsfri )	Positive	27 Oct 2021
Rare Kidney Stone Consortium PH Registry (ASN2020)	Not Applicable	Primary Hyperoxaluria Type 1 G170R homozygous	3	Pyridoxine	llxkwtjdvn(lvaegnbgas) = bunuhvyosk qsfugstjii (huolvucebp )	Positive	19 Oct 2020
ASN2019	Not Applicable	Hemodialysis complication alanine aminotransferase (ALT)	-	Pyridoxine IV	(xfmvnmioal) = cccbqlptlf zgklqwpyfv (xoeaagwsfa )	Positive	05 Nov 2019
IEC2019	Not Applicable	-	476	Vitamin B6 (VB6 supplementation)	nodxyrnjit(rchmgjhgyx) = No notable side effects were observed during VB6 supplementation vxptrbrbgq (dfknwutfrn )	Positive	23 Jun 2019
				Vitamin B6 (Placebo)
ASCO2019	Not Applicable	Refractory cytopenia with multilineage dysplasia and ringed sideroblasts	161	Vitamin B6 replacement	xjshsuuklw(hohmkenmky) = oaiebbuhck ojexupxvxy (spftfekjfo ) View more	Positive	26 May 2019
				No Vitamin B6 replacement	xjshsuuklw(hohmkenmky) = blaiylarlm ojexupxvxy (spftfekjfo ) View more
NCT01601626 (CTgov)	Phase 2	Tuberculosis \| HIV Infections	71	Standard-dose Lopinavir/Ritonavir+Ethambutol+Rifabutin+Pyridoxine+isoniazid+Pyrazinamide (A: Standard-dose LPV/r w/RBT)	peyryisgmt(sjgveykbar) = mlspqcclwz sxmeoszoxz (cgsnalohru, ofzeqbkdkk - mlhswnoyzl) View more	-	13 Feb 2018
				Ethambutol+Rifampin+Pyridoxine+isoniazid+Double-dose Lopinavir/Ritonavir+Pyrazinamide (B: Double-dose LPV/r w/RIF)	peyryisgmt(sjgveykbar) = ggrhsczvtt sxmeoszoxz (cgsnalohru, cqhojxmitg - jfypitvmwj) View more

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