RegulationFast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Rare Pediatric Disease (European Union)

Structure/Sequence

Molecular FormulaC8H12NO7P

InChIKeyCEEQUQSGVRRXQI-UHFFFAOYSA-N

CAS Registry41468-25-1

Clinical Trials associated with Pyridoxal Phosphate Hydrate

NCT04706013

/ RecruitingPhase 3

Study of Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency

The proposed clinical study is intended to evaluate oral P5P for the treatment of patients confirmed to have Pyridox(am)ine 5'-Phosphate Oxidase (PNPO) deficiency via genetic analysis. There is an unmet clinical need for pharmaceutical grade P5P, as to date none has been made commercially available. Patients will receive pharmaceutical grade P5P according to their normal oral P5P dosing regimen, as previously established by their physicians.

Start Date16 Feb 2024

Sponsor / Collaborator

Medicure, Inc.

NCT05755152

/ CompletedNot ApplicableIIT

Fruit-based Chewing Gums for Improving Oral Health

This study aims to conduct a randomized cross-over study investigating the release of cranberry polyphenols from chewing gum formulated with polyphenol-lecithin precipitate (PLP). 10 participants will be on study for up to 2 weeks and will each complete 2-30 minute study intervention visits.

Start Date10 Oct 2023

Sponsor / Collaborator

University of Wisconsin Madison

[+1]

NCT04645966

/ TerminatedPhase 2

A PHASE 2b TRIAL TO ASSESS THE SAFETY, TOLERABILITY, AND IMMUNOGENICITY OF MenABCWY IN HEALTHY INFANTS 2 AND 6 MONTHS OF AGE

The aim of the study is to describe the safety, tolerability, and immunogenicity of MenABCWY in healthy infants 2 and 6 months of age.

Start Date26 Nov 2020

Sponsor / Collaborator

Pfizer Inc.

100 Clinical Results associated with Pyridoxal Phosphate Hydrate

100 Translational Medicine associated with Pyridoxal Phosphate Hydrate

100 Patents (Medical) associated with Pyridoxal Phosphate Hydrate

9,341

Literatures (Medical) associated with Pyridoxal Phosphate Hydrate

01 Aug 2025·JOURNAL OF BIOTECHNOLOGY

Genome mining of an endophytic natural yeast isolate Rhodotorula sp. Y090 and production of the potent antioxidant ergothioneine

Article

Author: Chen, Chao ; Zhao, Xin-Qing ; Li, Yu-Zhen ; Wang, Qi ; Zhang, Feng-Li ; Li, Jun ; Cheng, Cheng ; Zou, Yue

Endophytic yeast strains are promising sources of various valuable bioactive compounds. However, studies on endophytic strains derived from lichen samples remain limited. In this study, we isolated and characterized Rhodotorula sp. Y090 from a Usnea longissima lichen sample, and investigated its biosynthetic potential. Genome mining revealed distinct genetic features that differed from its closest relative strain R. graminis WP1. Ergothioneine (EGT) is a potent antioxidant and rare sulfur-containing histidine derivative. However, so far, the EGT biosynthetic enzymes by natural yeast strains have been limitedly studied. In this study, combining genome mining and transcriptomic analysis, genes encoding the potential enzymes for the production of EGT and xylose utilization were identified in Rhodotorula sp. Y090. Further studies demonstrated that Rhodotorula sp. Y090 was capable of producing EGT using xylose, glucose, glycerol, or sucrose as the sole carbon source, and the highest titer reached 363.6 mg/L in shake flask culture, which is significantly higher than that of the most reported levels in the other natural yeasts. Rhodotorula sp. Y090 also exhibited a good ability of EGT export, which could facilitate cost-effective production. These findings suggest that the lichen-derived endophytic yeast Rhodotorula sp. Y090 represents a promising natural candidate for bio-production of the potent antioxidant.

01 Jul 2025·BIOCHEMICAL ENGINEERING JOURNAL

One-pot synthesis of arginine decarboxylase@ZIF-8 using intrinsic pyridoxal 5′-phosphate cofactor for agmatine production

Author: Zhao, Yunying ; Li, Guohui ; Ding, Bo ; Wang, Yan ; Deng, Yu ; Bao, Qingqing

Agmatine is an important basic drug and nutritional supplements, which exhibits significant physiol. activities.Traditional methods, such as whole-cell catalysis, require substantial pyridoxal 5′-phosphate (PLP) addition, which accounts for 15% of production costs.In this study, we utilized purified arginine decarboxylase (AdiA-M2) that leverages the intrinsic PLP cofactor, without the need for addnl. PLP supplementation, and ZIF-8 was employed to enhance the pH tolerance.The successful incorporation of AdiA-M2 into the ZIF-8 structure was confirmed through SEM (SEM) and confocal laser scanning microscopy (CLSM).The immobilized arginine decarboxylase demonstrated markedly enhanced performance under alk. conditions, along with improved thermal stability and storage activity compared to its free enzyme counterpart.Specifically, the immobilized AdiA@ZIF-8 system achieved an initial conversion rate of 95.16%, after five recycles the titer attained 266.69 g/L without the extra PLP addition, which is 1.67 times higher than the best current production approach.The adopted enzyme-catalysis system eliminated the need for addnl. cofactors, thereby lowering costs and providing valuable insights for the industrial production of agmatine.

01 Jun 2025·BIORESOURCE TECHNOLOGY

Systems metabolic engineering and process optimization for efficient l-tyrosine production from high-purity glucose syrup in Escherichia coli

Article

Author: Ma, Ling ; Chen, Zhichao ; Yu, Zichen ; Xu, Qingyang ; Fan, Xiaoguang ; Yuan, Meng ; Liu, Weiwei ; Li, Lanxiao ; Li, Changgeng

l-tyrosine (l-tyr) is a valuable aromatic amino acid that can be produced via microbial fermentation, providing a sustainable alternative to costly and polluting chemical synthesis. However, industrial production is limited by poor strain performance and inefficient resource utilization. In this study, a high-performance Escherichia coli strain was engineered to address key fermentation bottlenecks for efficient l-tyr synthesis from high-purity glucose syrup. Initially, a "rapid channel" for l-tyr biosynthesis was established by overexpressing aroG^fbr and tyrA^fbr genes, introducing the pyridoxal 5'-phosphate synthesis pathway, and strengthening tyrosine efflux. Precursor pools of phosphoenolpyruvate and erythrose-4-phosphate were enriched using modular metabolic engineering and dynamic regulation strategies. Co-metabolism of glucose, maltose, and isomaltose was achieved by integrating Bacillus subtilis-derived membrane permease and maltose-6'-phosphate glucosidase, alongside Bifidobacterium adolescentis-derived oligo-1,6-glucosidase, and by employing a suboptimal glucose supplementation feeding strategy. To overcome oxygen limitation, Vitreoscilla hemoglobin was localized to the periplasm via the twin-arginine translocation pathway. Systematic fermentation optimization further improved strain performance, achieving an l-tyr titer, yield, and productivity of 109.2 g/L, 0.292 g/g, and 2.18 g/L/h, respectively-the highest reported to date. This research demonstrates a promising strategy for enhancing l-tyrosine biosynthesis, providing a scalable approach for industrial production and broader applications in microbial metabolic engineering.

News (Medical) associated with Pyridoxal Phosphate Hydrate

31 Oct 2024

·synapse.patsnap.com

miRNA Therapeutics: Cutting-Edge Advances from Mechanism Exploration to Clinical Applications and Market Outlook

MicroRNA (miRNA) is a crucial class of non-coding RNA molecules that play a key role in the regulation of gene expression. Abnormal expression of miRNAs is closely associated with the occurrence and progression of various diseases. Consequently, miRNA-based therapies are viewed as a promising treatment strategy, especially for targeting "undruggable" targets that are challenging for traditional drugs. According to statistical data, the miRNA drug market was estimated at approximately $160.5 million in 2017, with an expected compound annual growth rate (CAGR) of 18.6%. As research advances and technology improves, the global MicroRNA market size is projected to expand further by 2030. Additionally, with increasing global interest in precision medicine and personalized therapies, miRNAs are gaining recognition as both biomarkers and therapeutic targets. This is particularly true in the field of cancer treatment, where miRNA regulation can influence tumor initiation, progression, and metastasis, making the development of drugs targeting specific miRNAs a research focus. The Role of miRNA in Diseases [1]Mechanism of Action of miRNA DrugsThe mechanisms of action of miRNA drugs are diverse, enabling them to simultaneously target multiple genes. This is especially important for complex diseases such as cancer, which often involve the aberrant expression of numerous genes. By employing miRNA mimics to supplement tumor-suppressive miRNAs or using miRNA inhibitors to suppress oncogenic miRNAs, these drugs can offer a novel therapeutic strategy. miRNAs have become attractive therapeutic targets due to their unique role in regulating gene expression. They achieve precise modulation of gene expression by binding to specific mRNAs, providing new possibilities for treating various diseases. The primary mechanism of action involves binding to the 3' untranslated region (3' UTR) of target mRNAs, inhibiting translation or promoting mRNA degradation. miRNA-mediated gene silencing [2]Broad Market Prospects for miRNA DrugsAccording to searches conducted via Patsnap Synapse, miRNA drugs demonstrate significant market potential due to their applications in disease prevention, diagnosis, and treatment, particularly in oncology, immunological disorders, and metabolic diseases. Although no miRNA drugs have yet entered Phase III clinical trials, the successful commercialization of siRNA drugs highlights the commercial value of RNA interference technology in drug development. Notable miRNAs, such as miR-21, which is overexpressed in various cancers, miR-122, associated with liver disease, and the frequently deleted miR-15 and miR-16 in chronic lymphocytic leukemia, have become hot topics of research. Moreover, global pharmaceutical giants like Novartis have begun aggressively acquiring siRNA assets, indicating a promising market outlook for this field. Indication Distribution of miRNA Drug Translational Medical ResearchRecent queries in the Patsnap Synapse translational medicine research section have revealed a significant focus on numerous miRNA targets, with several drugs currently in the clinical translation stage. For instance, miR-486-5p is a miRNA that plays a role in non-malignant diseases, particularly active in liver cells and the immune system, and is abundant in muscle and human plasma. miR-155 is crucial in the immune system, particularly expressed in lymphocytes, where it plays a vital regulatory role in immune development and function. miR-128 is considered a potential biomarker and therapeutic target due to its possible involvement in various clinical processes. miR-203a-3p has shown promise in chronic pain treatment by specifically regulating pain sensitivity responses in the hypothalamic arcuate nucleus. miR-223-3p is associated with the side effects of pyrimidine-based drugs in hepatocellular carcinoma, indicating its potential role in early diagnosis and treatment of side effects in liver cancer. miR-186-5p plays a role in tumor angiogenesis by regulating pro-angiogenic pathways in endothelial cells, making it a potential target for anti-angiogenic cancer therapies. Additionally, miR-621 is viewed as a potential radiosensitizer due to its ability to enhance radiotherapy effects. Finally, the miR-17-92 cluster plays significant roles in various biological processes, including regulation of B-cell lymphoma and other immune cells, in contexts like the immune system and cardiovascular diseases. The expression and function of these miRNAs in different disease states reveal their tremendous potential as diagnostic markers and therapeutic targets. Hot targets in miRNA research and developmentGlobal Competitive Landscape of miRNA Drug DevelopmentmiRNA drug development activities are widely distributed globally, particularly in North America, Europe, and Asia. Alnylam Pharmaceuticals, based in the United States, leads the RNAi therapy field, with several RNAi drugs in late-stage clinical development. Alnylam is not only technologically advanced but has also established collaborations with other major pharmaceutical companies, further solidifying its leadership in the RNAi sector. The top 5 countries/regions in miRNA drug research and developmentIn China, companies such as Ribo Life Science, Sirnaomics Biopharmaceuticals, and GenePharma are playing increasingly important roles in miRNA drug development. Notably, Ribo Life Science has collaborated with Boehringer Ingelheim to jointly develop innovative small nucleic acid therapies for non-alcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH). Sirnaomics Biopharmaceuticals has its own development pipeline in the small nucleic acid drug field, demonstrating its vitality and innovation in this domain. GenePharma is renowned for its expertise in siRNA chemical synthesis, providing a solid foundation for miRNA drug development. UniQure, a company with deep expertise in gene therapy, particularly in utilizing AAV delivery systems, also has multiple therapeutic pipelines in the miRNA space, with AMT-260 and AMT-130 having entered clinical phases I/II, showcasing its development strength in miRNA therapies. Looking ahead, the miRNA drug market is expected to continue growing with ongoing technological advancements and industry development. While no miRNA drug products are currently on the market, we can infer the future potential of the miRNA drug market by examining the development of mRNA technology. mRNA technology has demonstrated significant market potential in preventive vaccines, tumor immunotherapy, and other therapeutic areas, with projections estimating that the mRNA pharmaceuticals market could reach approximately $23 billion by 2035. Given the similarities between miRNA drugs and mRNA technology, both being RNA-based therapeutic approaches involved in regulating gene expression, it is reasonable to speculate that the miRNA drug market may also achieve rapid development in the coming years. Challenges in the Application of miRNA DrugsThe technological challenges facing miRNA drugs primarily include target validation, optimization of delivery systems, and enhancement of stability. Target validation is a crucial step in determining whether a miRNA is suitable as a drug target, requiring the identification of specific miRNAs associated with particular diseases and clarifying their central role in disease progression. Optimizing delivery systems is essential to ensure that miRNAs effectively reach target tissues and release within cells, while improving stability is necessary to resist degradation by intracellular nucleases. Additionally, advancements in delivery technologies, particularly non-viral carriers like GalNac (N-acetylgalactosamine) and LNPs (lipid nanoparticles), are critical to the success of miRNA drugs. GalNac, as a novel delivery system, can enhance the delivery efficiency and targeting of miRNA drugs in vivo, while LNPs are favored for their excellent performance in protecting miRNAs from nuclease degradation. These technological advancements help overcome barriers related to the stability and targeted delivery of miRNA drugs, paving new pathways for their development. Moreover, chemical modifications of miRNA molecules can improve their stability and half-life in vivo, thereby enhancing their therapeutic efficacy. With ongoing advancements in delivery and chemical modification technologies, the development of miRNA drugs is moving toward safer and more effective solutions, bringing hope for their clinical application in the future. Administration routes of miRNA therapy [3]Hiro LS: Supporting miRNA Drug Development and Market AnalysisThrough the biopharmaceutical AI model " Hiro LS", you can gain in-depth access to the latest insights in miRNA research and industry trends. Hiro LS not only efficiently integrates and analyzes the latest global scientific achievements but also utilizes advanced algorithmic models to predict potential drug targets and therapeutic mechanisms, summarizing the historical development and milestones of miRNA, helping researchers identify the most promising research directions at early stages and avoid inefficient resource allocation. In terms of market competition analysis, Hiro LS can monitor global market dynamics for miRNA drugs in real-time, predict market capacity, and provide in-depth analyses of competitors' strategic layouts and technological advancements, offering customized competitive intelligence for enterprises. Through Hiro LS's intelligent analysis, companies can promptly adjust their R&D strategies and seize market opportunities. Importantly, Hiro LS provides an exceptionally smooth interactive Q&A experience, akin to having face-to-face discussions with seasoned industry experts, capable of addressing professional challenges while offering insightful advice. This intelligent support significantly enhances work efficiency and injects innovative thinking and strategic perspectives into research and business decisions. With comprehensive support from Hiro LS, enterprises and research institutions can not only keep pace with industry advancements but also lead future innovation directions, securing a competitive edge in a fierce market landscape. For an experience with the large-scale biopharmaceutical model Hiro-LS, please click here for a quick and free trial of its features！ ConclusionAs a significant class of non-coding RNA molecules, miRNAs play a critical role in gene expression regulation, with their abnormal expression closely linked to the onset and progression of various diseases. Consequently, miRNA drugs are viewed as a promising therapeutic avenue, particularly suitable for targeting “undruggable” targets that traditional drugs struggle to reach. The mechanism of miRNA action primarily involves binding to the 3' untranslated region (3' UTR) of target mRNAs, inhibiting gene translation or promoting mRNA degradation; this multi-target regulatory approach is particularly important for treating complex diseases like cancer. By utilizing miRNA mimics or inhibitors, specific miRNAs can be supplemented or suppressed, offering new strategies for treating diseases such as cancer. Although no miRNA drugs have yet entered phase III clinical trials, the successful commercialization of siRNA drugs indicates the commercial value of RNA interference technology in drug development, suggesting a promising market outlook for miRNA drugs. From a market perspective, miRNA drugs exhibit tremendous potential in disease prevention, diagnosis, and treatment, especially in areas like oncology, immune diseases, and metabolic disorders. Companies like Ribo Life Science, Sirnaomics Biopharmaceuticals, and GenePharma are actively participating in the development of miRNA drugs in China, showcasing the country’s rise in this field. However, the development of miRNA drugs still faces numerous challenges, including target validation, optimization of delivery systems, and drug stability. To overcome these challenges, researchers are exploring novel delivery technologies such as LNPs and GalNac, along with employing chemical modifications to enhance the stability of miRNAs. With continuous technological advancements and deeper clinical research, miRNA drugs are expected to achieve breakthrough progress in the coming years and ultimately be applied in clinical practice, providing new treatment options for patients. Reference: 1.Rupaimoole, R. and F.J. Slack, MicroRNA therapeutics: towards a new era for the management of cancer and other diseases. Nat Rev Drug Discov, 2017. 16(3): p. 203-222.2.Fabian, M.R. and N. Sonenberg, The mechanics of miRNA-mediated gene silencing: a look under the hood of miRISC. Nat Struct Mol Biol, 2012. 19(6): p. 586-93.3.Diener, C., A. Keller, and E. Meese, Emerging concepts of miRNA therapeutics: from cells to clinic. Trends Genet, 2022. 38(6): p. 613-626.

24 Jun 2024

·www.biospace.com

Medicure Announces Signing of Asset Purchase Agreement with CanAm Bioresearch Inc. for Acquisition of Intellectual Property

WINNIPEG, MB / ACCESSWIRE / June 24, 2024 / Medicure Inc. ("Medicure" or the "Company") (TSXV:MPH)(OTC PINK:MCUJF), a company focused on the development and commercialization of pharmaceuticals and healthcare products for patients and prescribers in the United States market, today announces it has signed an asset purchase agreement ("Agreement") with CanAm Bioresearch Inc. ("CanAm") for the acquisition of the patent and intellectual property related to the discovery of new chemical entities that can be developed for therapeutic use (the "Proposed Transaction"). Medicure believes that the new chemical entities hold promise to provide improvements over existing lead compounds in alignment with the treatment of diseases being targeted by Medicure, and could provide significant value upon completion of all required non-clinical and clinical studies and regulatory approval. These new chemical entities are not being applied in Medicure's current Phase 3 study evaluating the use of MC-1 for the prevention or treatment of seizures associated with pyridox(am)ine 5'-phosphate oxidase (PNPO) deficiency. Key Terms of the Agreement Pursuant to the Agreement, Medicure International Inc., a wholly owned subsidiary of Medicure, has agreed to acquire, subject to the conditions contained in the Agreement, all of the assets of CanAm as they relate to the business of developing pyridoxal 5'-phosphate analogues ("P5P Analogues"), the processes for their preparation, compositions containing P5P Analogues, and methods of medical treatment containing P5P Analogues (the "Assets"). In consideration for the Assets, Medicure has agreed to pay to CanAm a $100,000 cash payment on the closing date, in addition to the following milestone payments: (i) $500,000, earned upon Medicure filing its first investigational new drug application ("IND Application") related to P5P Analogues; (ii) $250,000, earned upon the Medicure filing its first New Drug Application ("NDA") related to P5P Analogues; and (iii) $500,000, earned upon Medicure obtaining an NDA approval for P5P Analogues. In addition, Medicure shall pay to CanAm 10% of net proceeds received with respect to transactions relating to the Assets, including: (i) the sale or transfer of all or substantially all of the Assets to a third party purchaser who is not an affiliate of Medicure; (ii) any license to develop, commercialize, use, offer for sale, sell, import, export or exploit P5P Analogues up to a maximum value payable to CanAm of $20,000,000; and (iii) the sale of an United State Food and Drug Administration priority review voucher obtained in connection with the development of P5P Analogues. In the event Medicure retains a contract research organization ("CRO") to provide services related to development of P5P Analogues, CanAm, a CRO, would be entitled to tender a bid for such services, in addition to having an opportunity to match quotes for CRO services provided by third-party organizations. In the event that Medicure does not IND Application within 7 years from the closing date of the Proposed Transaction, CanAm shall have the option at any time after such date to repurchase the Assets from Medicure, at a price equal to the aggregate of all expenses incurred by Medicure in connection with the development of P5P Analogues, but, in any event, not exceeding the sum of $6,500,000 USD. Closing of the Proposed Transaction is subject to a number of terms and conditions, including without limitation, CanAm completing certain intellectual property filings with respect to the patent application it holds for P5P Analogues, the parties obtaining all necessary consents, orders and regulatory approvals, including approval of the TSX Venture Exchange ("TSXV"), and certain other customary closing conditions. All dollars are Canadian dollars unless otherwise noted. About CanAm Founded in 2000, CanAm is a privately held medicinal chemistry contract research organization providing drug discovery services, and is located in Winnipeg, Canada. As Medicure's CEO and Chairman of the board of directors, Dr. Albert D. Friesen, is the President and shareholder of the parent company of CanAm, the Proposed Transaction will constitute a "related party transaction" in accordance with Multilateral Instrument 61-101 - Protection of Minority Security Holders in Special Transactions ("MI 61-101"). The Proposed Transaction will be exempt from the formal valuation and the minority shareholder approval requirements of MI 61-101, as no securities of Medicure are listed or quoted on any of the specified markets listed in section 5.5(b) of MI 61-101 and, at the time the Proposed Transaction was agreed to, neither the fair market value of the subject matter of, nor the fair market value of the consideration for, the Proposed Transaction, insofar as it involves interested parties, exceeded 25% of Medicure's market capitalization. No finder's fee is expected to be payable in connection with the Proposed Transaction. Due to Dr. Friesen's involvement with CanAm, a special committee (the "Special Committee") of independent directors was established to consider the Proposed Transaction without conflict of interest and to negotiate the terms of the Agreement. The Special Committee unanimously determined that the Proposed Transaction is in the best interests of Medicure and recommended that the board of directors of Medicure approve the Agreement. About Medicure Inc. Medicure is a company focused on the development and commercialization of pharmaceuticals and healthcare products for patients and prescribers in the United States market. The present focus of the Company is the marketing and distribution of AGGRASTAT® (tirofiban hydrochloride) injection and ZYPITAMAG® (pitavastatin) tablets in the United States, where they are sold through the Company's U.S. subsidiary, Medicure Pharma Inc. Medicure also operates Marley Drug Inc. ("Marley Drug®"), a pharmacy subsidiary servicing all 50 states, Washington D.C. and Puerto Rico. Marley Drug® is committed to improving access to medications for all Americans together with exceptional customer service and free home delivery. For more information visit . For more information about Medicure please visit . For additional information about AGGRASTAT®, please visit or refer to the full Prescribing Information. For additional information about ZYPITAMAG®, please visit or refer to the full Prescribing Information. To be added to Medicure's e-mail list, please visit: Neither the TSXV nor its Regulation Services Provider (as that term is defined in policies of the TSXV) accepts responsibility for the adequacy or accuracy of this release. Forward Looking Information: Statements contained in this press release that are not statements of historical fact, including, without limitation, statements containing the words "believes", "may", "plans", "will", "estimates", "continues", "anticipates", "intends", "expects" and similar expressions, may constitute "forward-looking information" within the meaning of applicable Canadian and U.S. federal securities laws (such forward-looking information and forward-looking statements are hereinafter collectively referred to as "forward-looking statements"). Forward-looking statements, include estimates, analysis and opinions of management of the Company made in light of its experience and its perception of trends, current conditions and expected developments, as well as other factors which the Company believes to be relevant and reasonable in the circumstances. More particularly and without limitation, this press release contains forward-looking statements and information regarding the completion of the Proposed Transaction, the acquisition of the Assets by Medicure and the ability of the new chemical compounds acquired to provide improvements over existing lead compounds. Except as may be required by Canadian securities laws, the Company does not undertake any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Inherent in forward-looking statements are known and unknown risks, uncertainties and other factors beyond the Company's ability to predict or control that may cause the actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements, and as such, readers are cautioned not to place undue reliance on forward-looking statements. Such risk factors include, among others, failure of the parties to satisfy the conditions for the completion of the Proposed Transaction, termination of the Agreement, the Company's future product revenues, expected results, including future revenue from P5P, the likelihood of receiving a priority review voucher from the United State Food and Drug Administration, expected future growth in revenues, stage of development, additional capital requirements, risks associated with the completion and timing of clinical trials and obtaining regulatory approval to market the Company's products, the ability to protect its intellectual property, dependence upon collaborative partners, changes in government regulation or regulatory approval processes, and rapid technological change in the industry. Such statements are based on a number of assumptions which may prove to be incorrect, including, but not limited to, assumptions about: general business and economic conditions; the impact of changes in Canadian-US dollar and other foreign exchange rates on the Company's revenues, costs and results; the timing of the receipt of regulatory and governmental approvals for the Company's research and development projects; the availability of financing for the Company's commercial operations and/or research and development projects, or the availability of financing on reasonable terms; results of current and future clinical trials; the uncertainties associated with the acceptance and demand for new products and market competition. The foregoing list of important factors and assumptions is not exhaustive. The Company undertakes no obligation to update publicly or otherwise revise any forward-looking statements or the foregoing list of factors, other than as may be required by applicable legislation. Additional discussion regarding the risks and uncertainties relating to the Company and its business can be found in the Company's other filings with the applicable Canadian securities regulatory authorities or the US Securities and Exchange Commission, and in the "Risk Factors" section of its current Form 20F. AGGRASTAT® (tirofiban hydrochloride) injection, ZYPITAMAG® (pitavastatin) tablets, and Marley Drug® are registered trademarks. For more information, please contact: Dr. Albert D. Friesen Chief Executive Officer Tel. 888-435-2220 Fax 204-488-9823 E-mail: info@medicure.com SOURCE: Medicure Inc. View the original press release on accesswire.com

Phase 3NDA

23 Apr 2024

·www.biospace.com

Medicure Receives US FDA Fast Track Designation for MC-1 for PNPO Deficiency

WINNIPEG, MB / ACCESSWIRE / April 23, 2024 / Medicure Inc. ("Medicure" or the "Company") (TSXV:MPH)(OTC PINK:MCUJF), a company focused on the development and commercialization of pharmaceuticals and healthcare products for patients and prescribers in the United States market, announced today that through its subsidiary, Medicure International Inc., the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for MC-1, an investigational product for the prevention or treatment of seizures associated with pyridox(am)ine 5'-phosphate oxidase (PNPO) deficiency. Fast Track designation is intended to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need. The designation was requested based on the potential for MC-1 to address an unmet medical need for PNPO deficiency, a serious and life-threatening condition. Medicure is in the launch phase of its Phase 3 clinical trial to treat approximately 10 PNPO deficient patients at sites in the U.S. and Australia with daily doses of MC-1. PNPO deficiency is a rare neurometabolic disorder, often leading to neonatal onset seizures that are resistant to other antiseizure medications, resulting in severe neurological dysfunction and ultimately death if untreated. The FDA has granted both a Rare Pediatric Disease Designation and an Orphan Drug Designation to MC-1 for the treatment of seizures associated with PNPO deficiency. Additionally, the European Medicines Agency ("EMA") has granted an Orphan Drug Designation to MC-1 for the treatment of PNPO deficiency. If a new drug application ("NDA") for MC-1 for patients with PNPO deficiency is approved, Medicure is eligible to receive a priority review voucher from the FDA, which can be redeemed or sold, and provides significant value. About Medicure Inc. Medicure is a pharmaceutical company focused on the development and commercialization of pharmaceuticals and healthcare products for patients and prescribers in the United States market. The present focus of the Company is the marketing and distribution of AGGRASTAT® (tirofiban hydrochloride) injection and ZYPITAMAG® (pitavastatin) tablets in the United States, where they are sold through the Company's U.S. subsidiary, Medicure Pharma Inc. Medicure also operates Marley Drug, Inc. ("Marley Drug"), a pharmacy located in North Carolina that offers an Extended Supply drug program serving all 50 states, Washington D.C. and Puerto Rico. Marley Drug® is committed to improving the health status of its patients and the communities they serve while reducing overall health care costs for employers and other health care consumers. For more information visit . To learn more about The Extended Supply Generic Drug Program call 800.286.6781 or email info@marleydrug.com. For more information on Medicure please visit . For additional information about AGGRASTAT®, please visit or refer to the full Prescribing Information. For additional information about ZYPITAMAG®, please visit or refer to the full Prescribing Information. To be added to Medicure's e-mail list, please visit: Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release. Forward Looking Information: Statements contained in this press release that are not statements of historical fact, including, without limitation, statements containing the words "believes", "may", "plans", "will", "estimates", "continues", "anticipates", "intends", "expects" and similar expressions, may constitute "forward-looking information" within the meaning of applicable Canadian and U.S. federal securities laws (such forward-looking information and forward-looking statements are hereinafter collectively referred to as "forward-looking statements"). Forward-looking statements, include estimates, analysis and opinions of management of the Company made in light of its experience and its perception of trends, current conditions and expected developments, as well as other factors which the Company believes to be relevant and reasonable in the circumstances. Inherent in forward-looking statements are known and unknown risks, uncertainties and other factors beyond the Company's ability to predict or control that may cause the actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements, and as such, readers are cautioned not to place undue reliance on forward-looking statements. Such risk factors include, among others, the Company's future product revenues, expected results, including future revenue from P5P, the likelihood of receiving a priority review voucher from the United State Food and Drug Administration, expected future growth in revenues, stage of development, additional capital requirements, risks associated with the completion and timing of clinical trials and obtaining regulatory approval to market the Company's products, the ability to protect its intellectual property, dependence upon collaborative partners, changes in government regulation or regulatory approval processes, and rapid technological change in the industry. Such statements are based on a number of assumptions which may prove to be incorrect, including, but not limited to, assumptions about: general business and economic conditions; the impact of changes in Canadian-US dollar and other foreign exchange rates on the Company's revenues, costs and results; the timing of the receipt of regulatory and governmental approvals for the Company's research and development projects; the availability of financing for the Company's commercial operations and/or research and development projects, or the availability of financing on reasonable terms; results of current and future clinical trials; the uncertainties associated with the acceptance and demand for new products and market competition. The foregoing list of important factors and assumptions is not exhaustive. The Company undertakes no obligation to update publicly or otherwise revise any forward-looking statements or the foregoing list of factors, other than as may be required by applicable legislation. Additional discussion regarding the risks and uncertainties relating to the Company and its business can be found in the Company's other filings with the applicable Canadian securities regulatory authorities or the US Securities and Exchange Commission, and in the "Risk Factors" section of its current Form 20F. AGGRASTAT® (tirofiban hydrochloride) injection, ZYPITAMAG® (pitavastatin) tablets, and Marley Drug® are registered trademarks. For more information, please contact: Dr. Albert D. Friesen Chief Executive Officer Tel. 888-435-2220 Fax 204-488-9823 E-mail: info@medicure.com SOURCE: Medicure, Inc. View the original press release on accesswire.com

Phase 3Fast TrackOrphan DrugNDAPriority Review

100 Deals associated with Pyridoxal Phosphate Hydrate

External Link

KEGG	Wiki	ATC	Drug Bank
D00006	Pyridoxal Phosphate Hydrate	A11HA06	DB00114

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Vitamin B 6 Deficiency	Japan	Taiyo Pharma Co., Ltd.	17 Mar 1965

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Pyridoxamine 5-Prime-Phosphate Oxidase Deficiency	Phase 3	United States	Medicure, Inc.	23 Nov 2023
Coronary Artery Disease	Phase 3	United States	Medicure, Inc.	01 Nov 2006
Coronary Artery Disease	Phase 3	Canada	Medicure, Inc.	01 Nov 2006
Reperfusion Injury	Phase 3	United States	Medicure, Inc.	01 Nov 2006
Reperfusion Injury	Phase 3	Canada	Medicure, Inc.	01 Nov 2006
Schizoaffective disorder	Phase 2	Canada	Medicure, Inc.	01 May 2009
Schizoaffective disorder	Phase 2	India	Medicure, Inc.	01 May 2009
Schizophrenia	Phase 2	Canada	Medicure, Inc.	01 May 2009
Schizophrenia	Phase 2	India	Medicure, Inc.	01 May 2009
Tardive Dyskinesia	Phase 2	Canada	Medicure, Inc.	01 May 2009

Clinical Result

Indication

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


SAT-382 (ENDO2020)	Not Applicable	TNSALP \| PLP \| ALP ... View more	2	Pyridoxal 5’-Phosphate (Pre-treatment CSF PLP levels)	ftyhzgbcht(guidvnzuph) = jnuqbbaezf pnpvwdhgnn (cjdnkjsdkp )	-	08 May 2020
				Pyridoxal 5’-Phosphate (Pre-treatment CSF PLP and neurotransmitter levels)	mgxvgqspcx(iwqnzyqldk) = xujxzdkydp ofwvjglrsk (mxtruvahvk )
NCT00402506 (MEND-CARBG II, Pubmed)	Phase 3	High-Risk Pregnancy	3,023	MC-1, 250 mg/d	aryrzmvftn(uurgimwesj) = cbrqkektzu kymahuojmu (lttdptdtfa )	Negative	16 Apr 2008
				Placebo	aryrzmvftn(uurgimwesj) = tsejokhrtz kymahuojmu (lttdptdtfa )

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