Last update 12 Dec 2024

Sapropterin Dihydrochloride

Last update 12 Dec 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

(6R)-L-erythro-5,6,7,8-tetrahydrobiopterin, (6R)-L-erythro-tetrahydrobiopterin, (−)-(6R)-2-amino-6-((1R,2S)-1,2-dihydroxypropyl)-5,6,7,8-tetrahydro-4(3H)-pteridinone

+ [32]

Target

PAH

Mechanism

PAH agonists(Phenylalanine hydroxylase agonists)

Therapeutic Areas

Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]

Active Indication

Hyperphenylalaninemia, BH4-Deficient, BTetrahydrobiopterin DeficiencyHyperphenylalaninemia+ [2]

Inactive Indication

AlbuminuriaAnemia, Sickle CellAutistic Disorder+ [10]

Originator Organization

Daiichi Sankyo Co., Ltd.

Active Organization

APR Applied Pharma Research SASam OH Pharm Co. Ltd.

BioMarin Pharmaceutical, Inc.

+ [4]

Inactive Organization

Merck KGaA

Drug Highest PhaseApproved

First Approval Date

JP (27 Mar 1992),

Phenylketonurias

RegulationOrphan Drug (JP), Orphan Drug (KR), Orphan Drug (AU), Overseas New Drugs Urgently Needed in Clinical Settings (CN)

Structure

Molecular FormulaC9H17Cl2N5O3

InChIKeyRKSUYBCOVNCALL-NTVURLEBSA-N

CAS Registry69056-38-8

Clinical Trials associated with Sapropterin Dihydrochloride

NCT06481709 / RecruitingPhase 1

A Pilot, Phase 1, Randomized, Open-Label, Single-Dose, Four-Way Crossover Study to Compare the Pharmacokinetics of Sapropterin Dihydrochloride 100 mg/mL Oral Suspension (Product Code: RLF-OD032) (Test) With Kuvan® (Sapropterin Dihydrochloride) 100 mg Powder for Oral Solution (Reference) and to Evaluate the Effect of Food and the Effect of Water on the Bioavailability of Sapropterin Dihydrochloride 100 mg/mL Oral Suspension in Healthy Subjects

This is a single center, Phase 1, randomized, open-label, single-dose, 4 treatment, 4-period, 4-sequence, crossover study designed to compare the pharmacokinetics (PK) of sapropterin from the Test and Reference products, and to evaluate the effect of food and the effect of water administration on the bioavailability of sapropterin from the Test product in healthy subjects.
.

Start Date30 Jul 2024

Sponsor / Collaborator

APR Applied Pharma Research SA

NCT06396637 / RecruitingPhase 2IIT

The Efficacy and Safety of PD-1 Antibody Combined With Sapropterin Dihydrochloride in Patients With Advanced Pancreatic Cancer Who Failed to Standard Treatment.

The prognosis for pancreatic cancer remains dismal, with current guidelines favoring FOLFIRINOX or AG (consisting of Gemcitabine and Abraxane) as the primary chemotherapeutic option. However, research has indicated limited benefits for patients with pancreatic cancer undergoing immunotherapy using Anti-PD-1 antibodies. In this context, researchers aim to investigate the therapeutic potential of Sapropterin Dihydrochloride combined with PD-1 antibody in patients with metastatic pancreatic cancer who failed to standard treatment.

Start Date30 Apr 2024

Sponsor / Collaborator

Sun Yat-Sen University

[+1]

NCT05299203 / Unknown statusPhase 1IIT

Phase Ia/Ib Clinical Trial of Sapropterin Dihydrochloride Tablets in the Prevention and Treatment of Radiation-induced Skin Reactions in Intensity Modulated Radiotherapy for Head and Neck Squamous Cell Carcinoma (Including Nasopharyngeal Carcinoma)

Head and neck squamous cell carcinoma (include nasopharyngeal carcinoma) is one of the most common malignant tumor in China, and radiotherapy is the main treatment method. Radiation-induced dermatitis is one of the most common complications of head and neck squamous cell carcinoma patients during radiotherapy. Severe radiation dermatitis will cause secondary infection, severe pain, and even lead to the interruption of radiotherapy. On the one hand, it will affect the efficacy of tumor treatment, which may affect the long-term survival of patients, and on the other hand, it will also seriously affect the quality of life of patients. Tetrahydrobiopterin（BH4）, also known as sapropterin, has been shown not only to reduce the severity of acute radiation dermatitis but also to enhance the repair of skin injury in animal experiments. It may be a new approach and method for the prevention and treatment of radiation-induced dermatitis

Start Date01 May 2022

Sponsor / Collaborator

West China Hospital

100 Clinical Results associated with Sapropterin Dihydrochloride

100 Translational Medicine associated with Sapropterin Dihydrochloride

100 Patents (Medical) associated with Sapropterin Dihydrochloride

1,388

Literatures (Medical) associated with Sapropterin Dihydrochloride

11 Dec 2024·Journal of biomolecular structure & dynamics

Synthesis, anticancer activity and molecular modeling study of novel substituted triazole linked tetrafluoronaphthalene hybrid derivatives

Article

Author: Comert Onder, Ferah ; Erdoğan, Musa

To create some novel anticancer molecules, a library of novel series of various triazoles linked to the hydroxyl group of 5,6,7,8-tetrafluoronaphthalen-1-ol (3) was designed and synthesized via CuAAC reaction 'Click Chemistry' of tetrafluoronaphthalene based terminal alkyne with substituted organic azides. The structural characterizations of the targeted Click products 9-18 were confirmed by FTIR, ¹H NMR, ¹⁹F NMR, ¹³C NMR and HRMS spectroscopy. Synthesized compounds were tested in two triple negative breast cancer (TNBC) cell lines to understand their anticancer potentials. According to our findings, compounds 14 and 13 showed high cytotoxicity in BT549 cells at 20 μM and 30 μM, respectively. Moreover, these compounds blocked the migration of BT549 cells. In the MDA-MB-231 cell line, compound 18 exhibited high cytotoxicity and can block cell migration for 24 h. Molecular docking study with synthesized novel compounds was performed by Glide/SP method against SphK1 drug target. Furthermore, molecular dynamics (MD) simulation was carried out for the compounds 12-14 and 18. The compounds 13 and 14 may be potential inhibitor candidates in place of a reference inhibitor. A pharmacophore model was generated with the most potent compound 14, and the approved drugs were screened using the modules of Discovery Studio to find similar drugs. Consequently, this comprehensive study encompassing design, synthesis, in vitro and in silico analyses were correlated with the structure-activity relationship between compounds. The findings have the potential to unveil promising drug candidates for future studies.Communicated by Ramaswamy H. Sarma.

01 Dec 2024·Clinical Nutrition ESPEN

Nutrition in phenylketonuria

Review

Author: Eshraghi, Peyman ; Talebi, Saeedeh

Phenylketonuria (PKU) is a genetic metabolic disease resulting from a deficiency in the enzyme phenylalanine hydroxylase. This defect prevents the conversion of phenylalanine to tyrosine, and as a result, the level of phenylalanine in the body increases abnormally. The accumulation of this amino acid may cause brain damage and grows development in these patients. The primary approach to treating the disease involves dietary management that restricts protein and phenylalanine intake, often alongside Tetrahydrobiopterin (BH4) supplementation. Medical foods specifically formulated for these patients are utilized to supply the necessary energy, protein, and tyrosine. Other treatment options, including the use of large neutral amino acids (LNAAs) and the enzyme Phenylalanine ammonia lyase (PAL), can be utilized to manage symptoms in adult patients. Advancements in gene therapy aimed at correcting defective genes in patients may help restore phenylalanine metabolism, though further research in this area is necessary. Effective personal management of phenylketonuria necessitates enhanced knowledge and collaboration among healthcare professionals to achieve optimal treatment outcomes for patients. This article emphasis the nutritional management of individuals with phenylketonuria across various stages of their lives.

01 Oct 2024·EPILEPSIA

Progress report on new medications for seizures and epilepsy: A summary of the 17th Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVII). I. Drugs in preclinical and early clinical development

Article

Author: White, H. Steve ; Johannessen, Svein I. ; Perucca, Emilio ; Tomson, Torbjörn ; Koepp, Matthias J. ; Bialer, Meir ; Perucca, Piero

Abstract:

For >30 years, the Eilat Conference on New Antiepileptic Drugs and Devices has provided a forum for the discussion of advances in the development of new therapies for seizures and epilepsy. The EILAT XVII conference took place in Madrid, Spain, on May 5–8, 2024. Participants included basic scientists and clinical investigators from industry and academia, other health care professionals, and representatives from lay organizations. We summarize in this article information on treatments in preclinical and in early clinical development discussed at the conference. These include AMT‐260, a gene therapy designed to downregulate the expression of Glu2K subunits of kainate receptors, in development for the treatment of drug‐resistant seizures associated with mesial temporal sclerosis; BHV‐7000, a selective activator of heteromeric Kv7.2/7.3 potassium channels, in development for the treatment of focal epilepsy; ETX101, a recombinant adeno‐associated virus serotype 9 designed to increase NaV1.1 channel density in inhibitory γ‐aminobutyric acidergic (GABAergic) neurons, in development for the treatment of SCN1A‐positive Dravet syndrome; GAO‐3‐02, a compound structurally related to synaptamide, which exerts antiseizure activity at least in part through an action on cannabinoid type 2 receptors; LRP‐661, a structural analogue of cannabidiol, in development for the treatment of seizures associated with Lennox–Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex; OV329, a selective inactivator of GABA aminotransferase, in development for the treatment of drug‐resistant seizures; PRAX‐628, a functionally selective potent sodium channel modulator with preference for the hyperexcitable state of sodium channels, in development for the treatment of focal seizures; RAP‐219, a selective negative allosteric modulator of transmembrane α‐amino‐3‐hydroxy‐5‐methyl‐4‐isoxazolepropionic acid receptor regulatory protein γ‐8, in development for the treatment of focal seizures; and rozanolixizumab, a humanized anti‐neonatal Fc receptor monoclonal antibody, in development for the treatment of LGI1 autoimmune encephalitis. Treatments in more advanced development are summarized in Part II of this report.

News (Medical) associated with Sapropterin Dihydrochloride

29 Oct 2024

·www.prnewswire.com

BioMarin Announces 28% Y/Y Total Revenue Growth in the Third Quarter and Increase in Full-year 2024 Guidance; Reaffirms Long-term Guidance and Outlook

Third Quarter 2024 Total Revenues of $746 million (+28% Y/Y and +32% at Constant Currency Y/Y); Year-to-date 2024 Total Revenues of $2.11 billion (+19% Y/Y and +23% at Constant Currency Y/Y) During the Quarter, Strong Demand Drove 54% Y/Y Revenue Growth for VOXZOGO® During the Quarter, Revenues from Enzyme Therapies Portfolio Increased 27% Y/Y Third Quarter 2024 GAAP Diluted Earnings Per Share (EPS) of $0.55 (+162% Y/Y); Year-to-date 2024 GAAP Diluted EPS of $1.56 (+103% Y/Y) Third Quarter 2024 Non-GAAP Diluted EPS of $0.91 (+98% Y/Y); Year-to-date 2024 Non-GAAP Diluted EPS of $2.60 (+63% Y/Y) Conference Call and Webcast Scheduled Today at 4:30 p.m. ET SAN RAFAEL, Calif., Oct. 29, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the quarter and nine months ended September 30, 2024. "The strategic and operational decisions we have made over the last nine months are driving strong performance, reflected in year-over-year revenue growth in the third quarter of 28% and accelerated profitability," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "We are executing on our new corporate strategy, focused on Innovation, Growth, and Value Commitment, as demonstrated by another quarter of strong financial performance and clinical progress as we advance potential new medicines for the patients we serve." Mr. Hardy added, "Strong global demand for VOXZOGO led to over 3,800 infants and children receiving treatment as of the end of the third quarter. In the U.S., the majority of new patient starts in the third quarter were for children under 5 years of age, reflecting strong adoption from families seeking VOXZOGO therapy for their infants and young children. Our Enzyme Therapies portfolio also delivered impressive results, with third quarter revenue growth of 27% year-over-year, evidence of the continued growth potential of this portfolio," added Mr. Hardy. "Additionally, we made good progress advancing all of our innovative therapies under development. Within our CANOPY clinical program, we advanced our studies in five new indications with VOXZOGO. For hypochondroplasia, we are on track to complete enrollment in the Phase 3 registration-enabling study in the first half of 2025." Financial Highlights: Total Revenues for the third quarter of 2024 were $746 million, an increase of 28%, compared to the same period in 2023, driven by strong VOXZOGO contributions from new patient starts in all regions. In the quarter, revenues from BioMarin's Enzyme Therapies (VIMIZIM®, NAGLAZYME®, ALDURAZYME®, BRINEURA® and PALYNZIQ®) increased 27% compared to the third quarter of 2023. The increase was driven by a combination of the timing of order fulfillment to Sanofi as the company recognizes ALDURAZYME revenues when the product is released and control is transferred to Sanofi, increased patient demand, and the timing of large government orders in certain regions outside the U.S. Partially offsetting the increase were lower KUVAN® product revenues attributed to continued generic competition as a result of the loss of market exclusivity in 2022. GAAP Net Income increased by $66 million to $106 million in the third quarter of 2024 compared to the same period in 2023. The increase was primarily due to higher gross profit driven by the factors noted above. The increase was partially offset by higher spend in Selling, General and Administrative (SG&A), primarily due to severance and other restructuring costs associated with organizational redesign efforts executed during the third quarter of 2024, higher income tax expense and the impact of ROCTAVIAN® inventory reserves on Cost of Sales. Non-GAAP Income increased by $89 million to $178 million in the third quarter of 2024 compared to the same period in 2023. The increase in Non-GAAP Income was primarily due to higher gross profit and lower SG&A expenses primarily related to sales and marketing activities for ROCTAVIAN outside of the U.S., Germany and Italy as the company executes on its updated strategy to focus commercial launch efforts on those three countries. The increase was partially offset by higher income tax expense and the impact of ROCTAVIAN inventory reserves on Cost of Sales. 3Q'24 Execution on New Corporate Strategy: Innovation, Growth, and Value Commitment Innovation Skeletal Conditions: During the quarter, BioMarin advanced development across its CANOPY clinical program with VOXZOGO (vosoritide) in idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency, with the pivotal study in hypochondroplasia expected to complete enrollment in the first half of 2025. BioMarin's long-acting C-type natriuretic peptide (CNP), BMN 333, remains on track for initiation of the first-in-human study in early 2025. At the 16th International Skeletal Dysplasia Society meeting (ISDS) in September, BioMarin and its external research partners contributed to eight presentations (including four orals) discussing the value of vosoritide and the burden of illness in achondroplasia and related disorders. These data included showing that children with achondroplasia treated with VOXZOGO experienced meaningful improvements in addition to height, such as gains in health-related quality of life (HRQoL), and increased bone length while maintaining bone strength. Researchers also presented encouraging data from ongoing investigator-led studies of treatment in children with other genetic skeletal conditions, including hypochondroplasia and Noonan syndrome, as well as those with genetic variants often associated with idiopathic short stature such as aggrecan (ACAN) deficiency and heterozygous NPR2 mutations. Other Clinical Pipeline Programs: With BMN 351, BioMarin's next generation oligonucleotide for Duchenne Muscular Dystrophy, the program has completed enrollment into the first dose cohort and initial proof-of-concept data is expected in 2025 (including muscle dystrophin levels after 25 weeks of dosing). With BMN 349, an oral therapeutic for Alpha-1 antitrypsin deficiency (AATD)-associated liver disease, the program completed the single-ascending dose (SAD) phase of the first-in-human study and is expected to start dosing the multiple-ascending dose (MAD) phase of the study by end of the year. Enrollment is complete in the phase 3 study with PALYNZIQ in adolescents ages 12-17, and the study is on track for data readout in 2025 to support a potential U.S. Supplemental Biologics License Application (sBLA) in the second half of the year. Pre-clinical Programs: With BMN 390, a compound for phenylketonuria which may lower hypersensitivity and enhance exposure, an IND is expected to be submitted in the second half of 2025. With BMN 370, a targeted nanobody for the prevention of bleeding in patients with low levels of von Willebrand factor levels, the company is progressing with pre-clinical work and targeting a potential IND submission for the second half of 2025. Growth As of the end of the third quarter, over 3,800 children globally, many from infancy, were receiving VOXZOGO for the treatment of achondroplasia. VOXZOGO's broad label has been especially important to those families pursuing maximum therapeutic benefit by beginning therapy at an early age. In the U.S., the largest single market opportunity, the majority of new patient starts in the quarter were for children under the age of 5 years. VOXZOGO's extensive safety and efficacy profile led more families to begin therapeutic intervention early to potentially impact craniofacial volume, foramen magnum area, body proportionality and quality of life, in addition to durable increases in growth velocity. Achondroplasia represents a global 24,000 total addressable patient population (TAPP). While the U.S. is the largest single market opportunity, markets outside of the U.S. represent approximately 90% of eligible patients. BioMarin is in the process of pursuing VOXZOGO access into more than 20 additional countries by 2027, providing the opportunity for even more children of all ages to benefit from the only approved medicine for the treatment of achondroplasia. Enzyme Therapies continue to be a significant driver of growth, with revenues increasing 13% year-to-date, compared to the same period in 2023. As outlined at Investor Day, BioMarin is implementing new initiatives to drive sustained growth of the Enzyme Therapies across the approximately 80 countries where these medicines are available. Value Commitment During the quarter, the company made significant progress executing its financial strategy to deliver on its value commitment to stakeholders. Year-to-date, BioMarin's GAAP Operating Margin of 15.3% expanded 6.4 percentage points Y/Y and Non-GAAP Operating Margin of 27.7% expanded 7.6 percentage points Y/Y while GAAP Diluted EPS of $1.56 increased 103% Y/Y and Non-GAAP Diluted EPS of $2.60 increased 63% Y/Y. These measures of profitability increased at rates faster than revenue growth, representing the company's focus on operational efficiency. During the quarter, the company continued to benefit from its ongoing $500 million cost transformation program announced at Investor Day through the impact of prioritized program decisions and ongoing execution of the enterprise-wide reorganization. The company generated operating cash flows totaling $221 million in the third quarter, an increase of 63% compared to the same period last year. Total cash and investments at the end of the third quarter were approximately $1.5 billion, and with its increasing profitability, BioMarin is positioned to generate increasing operating cash flow into the future. In addition, BioMarin settled $495 million of convertible debt in cash during the quarter as planned, resulting in the retirement of approximately four million potentially dilutive shares. This was the first time that BioMarin retired a convertible note without issuing a new convertible instrument, thereby returning value to shareholders. Today, BioMarin increased full-year 2024 guidance for Total Revenues, Non-GAAP Operating Margin, and Non-GAAP Diluted EPS. The updated guidance highlights the sustained strong demand for VOXZOGO and growth trajectory of Enzyme Therapies, as well as BioMarin's commitment to expand profitability while investing in innovation. During the quarter, BioMarin reaffirmed long-term guidance and outlook previously provided at Investor Day on September 4, 2024, targeting: Approximately $4 billion in Total Revenues in 2027; 40% Non-GAAP Operating Margin(1) starting in 2026 and growing to the low- to mid-40% range over time; More than $1.25 billion operating cash flow per year starting in 2027; Mid-teen compound annual growth rate (CAGR) for total revenues through 2034; and Treatments for Skeletal Conditions to represent a greater than $5 billion revenue opportunity over time. Forward-Looking Non-GAAP Financial Information BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from its strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors. 2024 Full-Year Financial Guidance (in millions, except % and EPS amounts) (Updated) BioMarin will host a conference call and webcast to discuss third quarter 2024 financial results today, Tuesday, October 29, 2024, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at . About BioMarin BioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin pursues treatments that offer new possibilities for patients and families around the world navigating rare or difficult to treat genetic conditions. To learn more, please visit .   Forward-Looking Statements This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Total Revenues, Non-GAAP Operating Margin percentage, and Non-GAAP Diluted EPS for the full-year 2024 and the underlying drivers of those results, such as the revenue opportunity represented by treatments for skeletal conditions, as well as the expectations relating to operating cash flow per year starting in 2027 and BioMarin's compound annual growth rate (CAGR) for total revenues through 2034; BioMarin's new corporate strategy and belief that such strategy will build on BioMarin's legacy of innovation to deliver even greater value to the patients it serves around the world and position BioMarin for significant growth; BioMarin's commitment to expand profitability while investing in innovation; BioMarin's ability to accelerate the VOXZOGO opportunity; the continued growth potential of BioMarin's Enzyme Therapies portfolio; the anticipated benefits from its organizational redesign efforts; BioMarin's updated strategy for ROCTAVIAN and its anticipated benefits; the timing of orders for commercial products; BioMarin's ability to meet product demand; the timing of BioMarin's clinical development and commercial prospects, including announcements of data from clinical studies and trials; the clinical development and commercialization of BioMarin's product candidates and commercial products, including (i) the potential to leverage VOXZOGO in conditions beyond achondroplasia, such as hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, SHOX deficiency and other genetic short stature pathway conditions, (ii) the expected completion of enrollment for the pivotal study of VOXZOGO in hypochondroplasia in the first half of 2025, (iii) the expected expansion of VOXZOGO in the U.S. and BioMarin's ability to enable VOXZOGO access into more than 20 additional countries by 2027, (iv) BioMarin's expectation to initiate the first-in-human study for BMN 333 in early 2025, (v) BioMarin's expectation to receive initial proof-of-concept data regarding BMN 351 in 2025, (vi) BioMarin's expectation to start dosing the multiple-ascending dose phase of the study for BMN 349 by the end of the year, (vii) BioMarin's expectation to receive data from the phase 3 study with PALYNZIQ in adolescents ages 12-17 in 2025 to support a potential U.S. Supplemental Biologics License Application in the second half of the year, (viii) BioMarin's expectations to submit an IND for BMN 390 in the second half of 2025, and (ix) BioMarin's expectation to potentially submit an IND for BMN 370 during the second half of 2025; the expected benefits and availability of BioMarin's commercial products and product candidates; and potential growth opportunities and trends, including BioMarin's plans to drive growth of its Enzyme Therapies across the 80 countries in which they are available. 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 its commercial products; impacts of macroeconomic and other external factors on BioMarin's operations; results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the Food and Drug Administration, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products; actual sales of BioMarin's commercial products; 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 June 30, 2024 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®, BRINEURA®, KUVAN®, NAGLAZYME®, PALYNZIQ®, ROCTAVIAN®, VIMIZIM® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners. Non-GAAP Information The results presented in this press release include both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP Weighted-Average Diluted Shares Outstanding. Non-GAAP Weighted-Average Diluted Shares Outstanding is defined by the company as GAAP Weighted-Average Diluted Shares Outstanding, adjusted to include any common shares issuable under the company's equity plans and convertible debt in periods when they are dilutive under Non-GAAP. The company's presentation of percentage changes in total revenues at Constant Currency rates, which is computed using current period local currency sales at the prior period's foreign exchange rates, is also a Non-GAAP financial measure. This measure provides information about growth (or declines) in the company's total revenue as if foreign currency exchange rates had not changed between the prior period and the current period. BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. Because Non-GAAP Income, Non-GAAP Operating Margin percentage, Non-GAAP Diluted EPS, Non-GAAP Weighted-Average Diluted Shares Outstanding and Constant Currency are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the company's results from period to period and to its forward-looking guidance, and to identify operating trends in the company's principal business. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Non-GAAP Income and its components are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information: SOURCE BioMarin Pharmaceutical Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Financial StatementPhase 3Drug ApprovalOligonucleotide

06 Sep 2024

·www.globenewswire.com

Jnana Therapeutics Presents New Positive Data from Phase 1/2 Study of JNT-517 in Individuals with Phenylketonuria – Results Support Planned Pivotal Study in Early 2025

Data presented at SSIEM 2024 Annual Symposium Twice-daily oral treatment with JNT-517 at 150mg dose delivered highly statistically significant mean blood phenylalanine reduction of 60% versus baseline in individuals with PKU JNT-517 was safe and well tolerated with no serious adverse events observed JNT-517 has been granted Orphan Drug Designation by the U.S. FDA for the treatment of PKU, as well as Orphan Designation by the European Commission and eligibility Sept. 04, 2024 -- Jnana Therapeutics, a clinical-stage biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, today released results from the second-dose cohort of 150mg BID (twice daily) from its Phase 1/2 clinical trial of JNT-517 in adults with phenylketonuria (PKU). Both the 75mg and 150mg cohorts delivered clinically meaningful, statistically significant effects, and these results were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium in Porto, Portugal. JNT-517, a small molecule inhibitor of the phenylalanine (Phe) transporter SLC6A19, is being evaluated as a potential first-in-class oral treatment for PKU across all ages and genotypes. “Treatment with JNT-517 in individuals with PKU, regardless of disease severity, demonstrated rapid, clinically meaningful reductions in plasma Phe, the registrational endpoint for PKU, and was well tolerated with a favorable safety profile,” said George Vratsanos, M.D., Chief Medical Officer and Head of R&D at Jnana Therapeutics. “We are grateful to the study participants and highly encouraged by these results, which are prompting us to move quickly toward a registrational study.” JNT-517 is being studied in a randomized, double-blind, placebo-controlled trial in individuals with mild to severe, or classical, PKU. Following a 28-day screening period, study participants were randomized to 150mg BID of JNT-517 or placebo in the recently completed second-dose cohort. Importantly, all qualifying study participants were randomized without a run-in period to test for response to JNT-517. The 150mg BID cohort data presented at SSIEM were based on 18 participants, 11 dosed with JNT-517 and 7 dosed with placebo over 28 days, and demonstrated the following results: The 150mg BID dose of JNT-517 led to a statistically significant (p=<0.0001 vs. placebo) mean blood Phe reduction from baseline of 60% on average across days 14, 21, and 28 and a 58% reduction from baseline to day 28. This compares favorably to the previously studied 75mg BID dose, which led to a mean blood Phe reduction of 44% on average across days 14, 21, and 28 and a 51% reduction from baseline to day 28. Going forward, Jnana intends to use a measure of Phe reduction across multiple timepoints as it believes this is a more representative measure of drug effect. A rapid onset of effect was observed with statistically significant blood Phe reduction achieved within seven days after commencing dosing, which was sustained through the full 28 days of dosing. Mean (across days 14, 21, and 28) plasma Phe levels A robust response was observed across all participants irrespective of baseline blood Phe levels and prior PKU treatment. A mean baseline blood Phe of 920 μM and PAH genotypes indicated a majority of subjects had classical (severe) disease. Eight of 11 participants had previously tried sapropterin, and 1 out of 8 was deemed a responder to sapropterin. Seven of 11 participants had previously tried pegvaliase, and 0 out of 7 were deemed a responder to pegvaliase. JNT-517 was well tolerated with no serious adverse events, no clinically significant changes in laboratory parameters, and no clinically significant changes in plasma amino acids other than Phe observed, all consistent with the safety profile demonstrated in the 75mg BID cohort and Phase 1a healthy volunteer study. “These clinical results indicate that JNT-517 can drive a robust reduction in plasma Phe in individuals with high baseline Phe levels who have failed or had an inadequate response to the currently approved therapies,” said Nicola Longo, M.D., study investigator and Professor and Vice-Chair of Human Genetics at the University of California Los Angeles (UCLA) David Geffen School of Medicine. “JNT-517 has the potential to be a major new treatment option for PKU, particularly for those individuals with the most severe disease who currently lack effective treatment options.” On the basis of these positive results and recently granted regulatory designations from the FDA, the European Commission and EMA, Jnana plans to engage in discussions with U.S. and European regulators in the second half of 2024 with the goal of advancing JNT-517 directly into a pivotal Phase 3 study in early 2025. Jnana’s JNT-517 recently received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of PKU, Orphan Designation from the European Commission, and eligibility to the European Medicines Agency (EMA) PRIME scheme for the treatment of hyperphenylalaninemia, which collectively reinforce JNT-517 as a potential new medicine for PKU, a disease that lacks adequate therapies and where 60% of individuals with PKU are not currently on any therapy. JNT-517 was granted Rare Pediatric Disease Designation by the FDA in September 2022 for the treatment of PKU. The Phase 1/2 clinical program includes a randomized, double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics, and effect on blood and urinary Phe of JNT-517 dosed over a four-week period in individuals diagnosed with PKU. The study dosed its first participant with PKU in August 2023 and is enrolling individuals aged 18 to 65 at clinical sites in the United States and Australia. For more information about the study, please see clinicaltrials.gov (NCT05781399). JNT-517 is a selective small molecule inhibitor of the Phe transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe levels. JNT-517 has received Rare Pediatric Disease Designation and Orphan Drug Designation from the FDA for the treatment of PKU, as well as Orphan Designation from the European Commission and has been granted eligibility to the EMA PRIME scheme for the treatment of hyperphenylalaninemia. PKU is a rare inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). This enzyme is required for the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood (hyperphenylalaninemia). If left untreated, toxic levels of Phe in the blood can result in progressive and severe neurological impairment and neuropsychological complications. The SLC transporter SLC6A19 is responsible for kidney reabsorption of Phe back into the bloodstream, and the inhibition of SLC6A19 offers a novel, oral approach for the treatment of PKU by facilitating urinary excretion of excess Phe. Jnana Therapeutics is a clinical-stage biotechnology company leveraging its next-generation RAPID chemoproteomics platform to discover medicines for highly validated, challenging-to-drug targets to treat diseases with high unmet needs. Jnana is focused on developing first- and best-in-class therapies to treat a wide range of diseases, including rare diseases and immune-mediated diseases. Jnana’s wholly owned lead program, JNT-517, which targets an allosteric site on the phenylalanine transporter SLC6A19, is a potential first-in-class oral approach for the treatment of PKU, a rare genetic metabolic disease. Located in Boston, Jnana brings together scientific leaders in small molecule drug discovery and development, a highly experienced management team, and the backing of leading life science investors Bain Capital Life Sciences, RA Capital Management, Polaris Partners, Versant Ventures, Avalon Ventures, Pfizer Ventures, and AbbVie Ventures. Recently, Jnana announced that it has entered into an agreement to be acquired by Otsuka Pharmaceutical Co., Ltd., which is expected to close in the third quarter of 2024. For more information, please visit www.jnanatx.com and follow us on Twitter/X and LinkedIn. The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultOrphan Drug

04 Sep 2024

·www.prnewswire.com

BioMarin Holds Investor Day; Provides New Corporate Strategy and Introduces 2027 Financial Guidance

NEW YORK, Sept. 4, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) hosted an Investor Day earlier today, where President and Chief Executive Officer Alexander Hardy and other members of BioMarin's leadership team provided an overview of the company's new corporate strategy to deliver sustained value creation and introduced longer-term Total Revenue and Non-GAAP Operating Margin guidance. A copy of the presentation and a replay of the webcast are available at investors.biomarin.com. "Over the last 9 months, we have undertaken the transformation of BioMarin's operations and ways of working with the goal of accelerating and delivering substantial value to all our stakeholders – patients, employees and shareholders," said Mr. Hardy. "Our new corporate strategy harnesses BioMarin's unique capabilities creating and leading new therapeutic markets around the world to deliver innovative medicines to the patients we serve. We are confident in our ability to realize the ambitious plans outlined today and excited to deliver on our new vision for BioMarin's future." Corporate Strategy The company reviewed BioMarin's differentiated strategy including its new company structure and updated organizational model, now built around three business units, Enzyme Therapies, Skeletal Conditions and ROCTAVIAN®, designed to support the focus on sustainable growth. The updated corporate strategy also includes the implementation of a $500 million cost transformation program that will contribute to Non-GAAP Operating margin targets set for 2026 and beyond. Value Commitment BioMarin provided an overview of its long-term financial outlook, based on its strategic plan to drive revenue growth and expand Non-GAAP Operating Margin. The summary table below includes the company's full year 2024 guidance, reaffirmed today, and the newly introduced outlook for 2026 with respect to Non-GAAP Operating Margin and other metrics for 2027, shared at BioMarin's Investor Day. Innovation The company highlighted its innovation strategy that supports BioMarin's pipeline of high impact medicines, including 11 anticipated product launches by 2034 and two by 2027. Building on its 25-year history developing medicines that target serious genetic conditions, several programs that are currently advancing through the pipeline, include: Phase 3 enrollment of VOXZOGO® for the treatment of hypochondroplasia, with expected data readout targeted in 2026, and potential approval in 2027 Clinical updates on four additional skeletal conditions, including idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX Deficiency, all progressing through or beginning Phase 2 studies PALYNZIQ® age label expansion to include 12-17-year-olds in the United States and 12-15-year-olds outside of the United States, with targeted filings in late 2025 and early 2026, respectively BMN 390 for the treatment of phenylketonuria (PKU) with a targeted profile that reduces immunogenicity from novel pegylation, and targeted Investigational New Drug (IND) application planned for late 2025 BMN 351 for the treatment of Duchenne Muscular Dystrophy that targets a unique novel site for exon skipping that may enable dystrophin expression up to 40%, based on observations preclinically. Target Proof of Concept (POC) is expected in 2025 BMN 349 for the treatment of Alpha-1 Antitrypsin Deficiency with a unique mechanism of action that has the potential to transform liver health and with target POC in 2026 BMN 370 for the treatment of von Willebrand Disease (vWD) has the potential to normalize bleeding events, based on results from a vWD preclinical model, and is designed to be delivered with a single subcutaneous injection. The targeted IND is planned for late 2025. Growth: The company highlighted its strategy for optimizing its growing and durable Enzyme Therapies business unit, as well as its plan for sustainable leadership across multiple Skeletal Conditions, building on the strength of VOXZOGO for the treatment of achondroplasia. The combined business units are expected to drive a targeted mid-teen Compounded Annual Growth Rate through 2034. A replay of today's event and accompanying presentation slides can be found at investors.biomarin.com. 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 and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Total Revenues, Non-GAAP Operating Margin percentage, Non-GAAP Diluted EPS, Operating Cash Flow and Revenue Compound Annual Growth Rate (CAGR) for, in certain instances, the full-year 2024 and future periods and the underlying assumptions; BioMarin's new corporate strategy, including plans and expectations regarding innovation and growth, the $500 million cost optimization program, capital allocation, and organizational redesign efforts, as well as the anticipated benefits of such strategy, including BioMarin's ability to achieve top quartile biopharma revenue growth, double its Non-GAAP Operating Margin, achieve innovation and efficiencies, optimize cash flow and capital allocation, and deliver significant and sustained value creation to stakeholders; BioMarin's future strategy for ROCTAVIAN and its anticipated benefits, including BioMarin's expectations regarding reduction of annual direct ROCTAVIAN expenses beginning in 2025 and ROCTAVIAN being profitable by the end of 2025; ability of BioMarin's approved products, including VOXZOGO and BioMarin's enzyme therapies, to drive long-term revenue growth; the clinical development and commercialization of BioMarin's product candidates and commercial products, including plans and expectations regarding (i) the ability to expand BioMarin's leadership in achondroplasia with VOXZOGO and leverage VOXZOGO in other skeletal conditions, including hypochondroplasia, idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency; (ii) development of BMN 333 for the treatment of achondroplasia and hypochondroplasia, (iii) expansion of PALYNZIQ for the treatment of adolescents with phenylketonuria (PKU), (iv) development of BMN 390 for the treatment of PKU, (v) development of BMN 351 for the treatment of Duchenne Muscular Dystrophy, (vi) development of BMN 349 for the treatment of alpha-1 antitrypsin deficiency, and (vii) development of BMN 370 for the treatment of von Willebrand disease; the expected benefits and availability of BioMarin's product candidates and commercial products; the timing of BioMarin's clinical development and commercial prospects, including announcements of data from clinical studies and trials; and potential growth opportunities and trends, including the assumptions and expectations regarding Total Addressable Patient Population with respect to the conditions targeted by BioMarin's product candidates and commercial products. 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, 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 June 30, 2024, as such factors may be updated by any subsequent reports. You should carefully consider that information before you make an investment decision. You should not place undue reliance on forward-looking statements, which speak only as of the date hereof. These forward-looking statements are based on the beliefs and assumptions of the Company's management based on information currently available to management and should be considered in connection with any written or oral forward-looking statements that the Company may issue in the future as well as other cautionary statements the Company has made and may make. Except as required by law, BioMarin does not undertake any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin®, BRINEURA®, KUVAN®, NAGLAZYME®, PALYNZIQ®, ROCTAVIAN®, VIMIZIM® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners. Non-GAAP Information This press release includes both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations, excluding amortization of intangible assets, stock-based compensation expense, and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP weighted-average diluted shares outstanding. Non-GAAP weighted-average diluted shares outstanding is defined by the company as GAAP weighted-average diluted shares outstanding, adjusted to include any common shares issuable under the company's equity plans and convertible debt in periods when they are dilutive under Non-GAAP. BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. Because Non-GAAP Income, Non-GAAP Operating Margin percentage, Non-GAAP Diluted EPS and Non-GAAP weighted-average diluted shares outstanding are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin's GAAP information for historical results enhances investors' and analysts' ability to meaningfully compare the company's results from period to period and to its forward-looking guidance, and to identify operating trends in the company's principal business. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Non-GAAP measures are not meant to be considered in isolation or as a substitute for, or superior to, comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. Forward-Looking Non-GAAP Financial Measures BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from its strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors. SOURCE BioMarin Pharmaceutical Inc.

Phase 3

100 Deals associated with Sapropterin Dihydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D01798	Sapropterin Dihydrochloride	A16AX07	DB00360

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hyperphenylalaninemia, BH4-Deficient, B	EU	Dipharma Francis Srl	16 Feb 2022
Hyperphenylalaninemia, BH4-Deficient, B	IS	Dipharma Francis Srl	16 Feb 2022
Hyperphenylalaninemia, BH4-Deficient, B	LI	Dipharma Francis Srl	16 Feb 2022
Hyperphenylalaninemia, BH4-Deficient, B	NO	Dipharma Francis Srl	16 Feb 2022
Tetrahydrobiopterin Deficiency	AU	BioMarin Pharmaceutical, Inc.	28 Oct 2010
Hyperphenylalaninemia	EU	BioMarin International Ltd.	02 Dec 2008
Hyperphenylalaninemia	IS	BioMarin International Ltd.	02 Dec 2008
Hyperphenylalaninemia	LI	BioMarin International Ltd.	02 Dec 2008
Hyperphenylalaninemia	NO	BioMarin International Ltd.	02 Dec 2008
Phenylketonurias	JP	Daiichi Sankyo Co., Ltd.	27 Mar 1992

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Neuropsychiatric syndrome	Phase 3	US	BioMarin Pharmaceutical, Inc.	01 Aug 2010
Neuropsychiatric syndrome	Phase 3	CA	BioMarin Pharmaceutical, Inc.	01 Aug 2010
Autistic Disorder	Phase 3	US	BioMarin Pharmaceutical, Inc.	01 Aug 2009
Lesch-Nyhan Syndrome	Phase 3	US	BioMarin Pharmaceutical, Inc.	01 Apr 2009
Phenylketonuria, Maternal	Phase 3	US	BioMarin Pharmaceutical, Inc.	01 Feb 2006
Albuminuria	Phase 2	US	BioMarin Pharmaceutical, Inc.	01 May 2008
Chronic Kidney Diseases	Phase 2	US	BioMarin Pharmaceutical, Inc.	01 May 2008
Endothelial dysfunction	Phase 2	US	BioMarin Pharmaceutical, Inc.	01 Sep 2007
Anemia, Sickle Cell	Phase 2	US	BioMarin Pharmaceutical, Inc.	01 May 2007
Intermittent Claudication	Phase 2	US	BioMarin Pharmaceutical, Inc.	01 Dec 2006

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


SSIEM2022	Not Applicable	Phenylketonurias	80	Sapropterin dihydrochloride	ffkuyjbkmx(jofmvseykm) = klrwyidylv zzjzlmdisy (tiukjcdnlz ) View more	-	30 Aug 2022
SSIEM2022	Not Applicable	Phenylketonurias	79	Sapropterin dihydrochloride	esqixncodj(pwprixfxex) = ptwuaamgix wfhwsuxqyq (zyjwricuuv, 52.1)	Positive	30 Aug 2022
NCT02677870 (CTgov)	Phase 4	Phenylketonurias	7	Diet treatment (Diet Treatment)	mqfilnfopc(iavzhxlgtp) = imzhxgarsj euloaeextu (udzotgndxv, dqruxipswd - bgeygzleit) View more	-	27 Apr 2022
				Diet treatment+saproterin dihydrochloride (Standard Dose Saproterin Dihydrochloride)	mqfilnfopc(iavzhxlgtp) = yvwdkaqyct euloaeextu (udzotgndxv, dvxpznpykm - myitahuxwc) View more
NCT01376908 (SPARK, Pubmed \| Orphanet J Rare Dis) Manual	Phase 3	Phenylketonurias	51	sapropterin (extension' group)	vxancszrmz(oxwspgzwcc) = fwotgymxcf sfjenkghqr (tkpkrydpcl, 2.8 - 13.8)	Positive	03 Aug 2021
				sapropterin (continuous' group)	vxancszrmz(oxwspgzwcc) = vmdkrxrcor sfjenkghqr (tkpkrydpcl, 28.9 - 48.6)
NCT00532844 (CTgov)	Phase 2	Endothelial dysfunction	52	Sapropterin Dihydrochloride (Sapropterin Dihydrochloride)	jarscqylal(jjnoravdtd) = ijiqrxtqtu ufxblzpqzl (fjyskquwjh, hwmnwgqsca - dwkqhuarag) View more	-	17 May 2021
				Vitamin C+Sapropterin Dihydrochloride (Sapropterin Dihydrochloride+Vitamin C)	jarscqylal(jjnoravdtd) = lsrliwbhld ufxblzpqzl (fjyskquwjh, oqzyzrlnly - vtfdlzzadb) View more
NCT02774226 (COPD-LT, CTgov)	Phase 2	Pulmonary Disease, Chronic Obstructive	15	Tetrahydrobiopterin (BH4) (Tetrahydrobiopterin (BH4))	mxqrtthyin(mcqbzchque) = ycfzkwekxy ebefoobnsf (wlyuvtycbw, ueazzrqgon - gsdyonrcgw) View more	-	23 Feb 2021
				Antioxidant Cocktail (Antioxidant Cocktail)	mxqrtthyin(mcqbzchque) = rkuaapuiap ebefoobnsf (wlyuvtycbw, adwjhuyeeo - zclfzhujui) View more
NCT00445978 (CTgov)	Phase 2	Anemia, Sickle Cell	32	Sapropterin Dihydrochloride	teeqhgpkfq(yrwlgggqjz) = jjdbnlvyta mdlajmcfqi (yapbkonvvk, rvadkbkvgd - fjghdiaagv) View more	-	28 Jan 2021
NCT00403494 (CTgov)	Phase 2	Intermittent Claudication \| Peripheral Arterial Disease	190	Sapropterin Dihydrochloride (Sapropterin Dihydrochloride)	kuazyafckh(oshlkrupdv) = kmrpxbqgnx zbbogmuonq (ninlapkaog, iarktldsme - hpaldjnray) View more	-	07 Jan 2021
				Placebo (Placebo)	kuazyafckh(oshlkrupdv) = zvsspykqtu zbbogmuonq (ninlapkaog, qxlchyufwo - wuxkoywwvm) View more
NCT01439555 (CTgov)	Phase 2	Alzheimer Disease	11	Simvastatin+Tetrahydrobiopterin+L-Arginine	yvdlqvapxs(zbvbwbtxux) = mhliqzooka btpqlqguos (oegccsdoza, homasifzre - mqdpttdbzj) View more	-	30 Jul 2019
NCT01706965 (CTgov)	Phase 1/2	Schizoaffective disorder \| Schizophrenia	27	Kuvan (Kuvan)	japnadvwir(fwzvjiwamo) = rfbpwcfkso mspsflrbrw (nftznubjkh, vbiyymdlim - siqnyrxpev) View more	-	13 Sep 2017
				Multivitamin (Multivitamin)	japnadvwir(fwzvjiwamo) = hdjxnluglb mspsflrbrw (nftznubjkh, tegcgsxzli - hlksqzirtz) View more

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