Last update 01 Jul 2024

Sapropterin Dihydrochloride

Last update 01 Jul 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

+ [30]

Target

PAH

Mechanism

PAH agonists(Phenylalanine hydroxylase agonists)

Therapeutic Areas

Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]

Active Indication

Hyperphenylalaninemia, BH4-Deficient, BHyperphenylalaninemiaPhenylketonurias+ [1]

Inactive Indication

AlbuminuriaAnemia, Sickle CellAutistic Disorder+ [9]

Originator Organization

Daiichi Sankyo Co., Ltd.

Active Organization

BioMarin Pharmaceutical, Inc.BioMarin International Ltd.

Daiichi Sankyo Co., Ltd.

Inactive Organization

Merck KGaA

Drug Highest PhaseApproved

First Approval Date

JP (27 Mar 1992),

Phenylketonurias

RegulationOrphan Drug (JP), 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

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

NCT05138887 / Unknown statusPhase 2IIT

Clinical Professor

This present study envisaged a single-center, prospective, open-label, placebo and randomized controlled phase II clinical study to assess the efficacy and Safety of Tetrahydrobiopterin in Treating Radiation Enteritis in Gynecological Cancer Patients.

Start Date01 Mar 2022

Sponsor / Collaborator

West China Second University Hospital

100 Clinical Results associated with Sapropterin Dihydrochloride

100 Translational Medicine associated with Sapropterin Dihydrochloride

100 Patents (Medical) associated with Sapropterin Dihydrochloride

1,377

Literatures (Medical) associated with Sapropterin Dihydrochloride

01 Mar 2024·Journal of colloid and interface science

Tetrahydrobiopterin inhibitor-based antioxidant metabolic strategy for enhanced cancer ferroptosis-immunotherapy

Article

Author: Yang, Jie ; Yu, Hongjie ; Pan, Xiuhua ; Shen, Qi ; Deng, Wanli ; Jia, Zengguang ; Yang, Ning ; Kang, Ruixin ; Liu, Zengyi ; Zhang, Jun

The induction of immunogenic ferroptosis in cancer cell is limited by the complex and delicate antioxidant system in the organism. Synergistic induction of oxidative damage and inhibition of the defensive redox system in tumor cells is critical to promote lethal accumulation of lipid peroxides and activate immunogenic death (ICD). To address this challenge, we present a multifunctional and dual-responsive layered double hydroxide (LDH) nanosheet to enhance immunogenic ferroptosis. The MTX-LDH@MnO₂ nanoplatform is constructed by intercalating methotrexate (MTX) into LDH interlayers and electrostatically absorbing biomineralized ovalbumin (OVA)-MnO₂ onto the LDH surface. Specifically, the released Mn²⁺ from the incorporated MnO₂ triggers a Fenton-like reaction, leading to reactive oxygen species (ROS) accumulation, while the depletion of reduced glutathione (GSH) further intensifies oxidative stress, resulting in the induction of ferroptosis. MTX is released in response to the acidic environment of tumor cells and inhibits the regeneration of tetrahydrobiopterin (BH4), modulating the GTP cyclic hydrolase 1 (GCH1)/BH4 axis. MTX disrupts the antioxidant metabolic activity regulated by GCH1/BH4 axis and inhibits ROS consumption, further boosting the ferroptosis effect, which promoted the release of damage-associated molecular patterns (DAMPs) and triggered ICD in the tumor. This activation subsequently leads to significant antitumor immune reactions, including DCs maturation, infiltration of CD4⁺/CD8⁺ T cells and cytokines release. The redox-controllable nanoplatform demonstrates promising anticancer efficacy in a mouse breast model providing a novel strategy for cancer immunotherapy.

01 Mar 2024·Water research

Carbon metabolism characteristics of quorum quenching bacteria Rhodococcus sp. BH4 determine the bioaugmentation efficiency under different carbon source conditions

Article

Author: Li, Jun ; Li, Dongyue ; Guo, Wei ; Wang, Xiujie ; Liang, Dongbo ; Wu, Yaodong ; Liu, Xiaohui

Carbon sources are critical factors influencing bacterial bioaugmentation, however, the underlying mechanisms, particularly the metabolic characteristics of bioaugmented bacteria remain poorly understood. The bioaugmented bacterium Rhodococcus sp. BH4 secretes the quorum quenching (QQ) enzyme QsdA to disrupt the quorum sensing (QS) in the activated sludge (AS) process, reducing AS yield in-situ. This study investigated the carbon metabolic characteristics of BH4 and explored the effects on bioaugmentation with different influent carbon sources. Because of the absence of glucose-specific phosphoenol phosphotransferase system (PTS), BH4 prefers sodium acetate to glucose. However, the lactones produced during extracellular glucose metabolism enhance BH4 qsdA expression. Moreover, BH4 possess carbon catabolite repression (CCR), acetate inhibits glucose utilization. BH4 microbeads were added to reactors with different carbon sources (R1: sodium acetate; R2: glucose; R3: a mixture of sodium acetate and glucose) for in-situ AS yield reduction. During operation, AS reduction efficiency decreased in the following order: R1 > R3 > R2. R2 and R3 microbeads exhibited similar QQ activity to R1, with less BH4 biomass at 5 d. ¹³C labeling and Michaelis-Menten equation showed that, due to differences in the competitiveness of carbon sources, R1 BH4 obtained the most carbon, whereas R2 BH4 obtained the least carbon. Moreover, acetate inhibited glucose utilization of R3 BH4. Transcriptome analysis showed that R1 BH4 qsdA expression was the lowest, R2 BH4 was the most serious form of programmed cell death, and the R3 BH4 PTS pathway was inhibited. At 10 d, R1 BH4 biomass and microbead QQ activity were higher than that in R3, and the R2 BH4 lost viability and QQ activity. This study provides new insights into bioaugmentation from the perspectives of carbon source competitiveness, carbon metabolism pathways, and CCR.

01 Mar 2024·Molecular genetics and metabolism

Evaluating change in diet with pegvaliase treatment in adults with phenylketonuria: Analysis of phase 3 clinical trial data

Article

Author: Whitehall, Kaleigh ; Lilienstein, Joshua ; Rohr, Fran ; van Calcar, Sandra ; Singh, Rani ; Rose, Sarah ; Lindstrom, Kristin ; MacLeod, Erin ; Dee, Anne ; Burton, Barbara ; Harding, Cary O

Phenylketonuria (PKU), a genetic disorder characterized by phenylalanine hydroxylase (PAH) deficiency and phenylalanine (Phe) accumulation, is primarily managed with a protein-restricted diet and PKU-specific medical foods. Pegvaliase is an enzyme substitution therapy approved for individuals with PKU and uncontrolled blood Phe concentrations (>600 μmol/L) despite prior management. This analysis assessed the effect of pegvaliase on dietary intake using data from the Phase 3 PRISM-1 (NCT01819727), PRISM-2 (NCT01889862), and 165-304 (NCT03694353) clinical trials. Participants (N = 250) had a baseline diet assessment, blood Phe ≥600 μmol/L, and had discontinued sapropterin; they were not required to follow a Phe-restricted diet. Outcomes were analyzed by baseline dietary group, categorized as >75%, some (>0% but ≤75%), or no protein intake from medical food. At baseline, mean age was 29.1 years, 49.2% were female, mean body mass index was 28.4 kg/m², and mean blood Phe was 1237.0 μmol/L. Total protein intake was stable up to 48 months for all 3 baseline dietary groups. Over this time, intact protein intake increased in all groups, and medical protein intake decreased in those who consumed any medical protein at baseline. Of participants consuming some or >75% medical protein at baseline, 49.1% and 34.1% were consuming no medical protein at last assessment, respectively. Following a first hypophenylalaninemia (HypoPhe; 2 consecutive blood Phe measurements <30 μmol/L) event, consumption of medical protein decreased and consumption of intact protein increased. Substantial and sustained Phe reductions were achieved in all 3 baseline dietary groups. The probability of achieving sustained Phe response (SPR) at ≤600 μmol/L was significantly greater for participants consuming medical protein versus no medical protein in an unadjusted analysis, but no statistically significant difference between groups was observed for probability of achieving SPR ≤360 or SPR ≤120 μmol/L. Participants with alopecia (n = 49) had longer pegvaliase treatment durations, reached HypoPhe sooner, and spent longer in HypoPhe than those who did not have alopecia. Most (87.8%) had an identifiable blood Phe drop before their first alopecia episode, and 51.0% (n = 21/41) of first alopecia episodes with known duration resolved before the end of the HypoPhe episode. In conclusion, pegvaliase treatment allowed adults with PKU to lower their blood Phe, reduce their reliance on medical protein, and increase their intact and total protein intake. Results also suggest that HypoPhe does not increase the risk of protein malnutrition in adults with PKU receiving pegvaliase.

News (Medical) associated with Sapropterin Dihydrochloride

06 Jun 2024

·www.biospace.com

Cycle Pharmaceuticals Announces All-Cash Proposal to Acquire Vanda Pharmaceuticals for $8.00 Per Share

Represents an immediate, compelling cash premium of 98% over Vanda’s closing share price prior to the initial Future Pak proposal announced on April 17, 2024 Vanda shares have not traded at $8.00 per share since January 2023 and have traded as low as $3.30 per share in the past twelve months Cycle has an extensive U.S. operational footprint and distribution footprint, a strong strategic fit for Vanda’s commercialized therapies and clinical development pipeline Cycle and its advisors are standing by to work immediately with Vanda’s Board and management team to quickly negotiate and finalize a definitive agreement BOSTON & CAMBRIDGE, England--(BUSINESS WIRE)-- Cycle Pharmaceuticals Ltd (“Cycle” or the “Company”), a rapidly growing pharmaceutical company focused on treatments for the underserved rare disease patient community, today confirmed that it made a proposal to the Board of Directors of Vanda Pharmaceuticals, Inc. (“Vanda”) (NASDAQ: VNDA) on May 24, 2024 to acquire 100% of Vanda’s issued and outstanding shares for a cash consideration of $8.00 per Vanda share, representing a total cash consideration of $466 million. Cycle’s proposal represents: - Immediate and certain cash value for all Vanda shareholders; - A 98% premium above Vanda’s closing share price on April 16, 2024, the day prior to the public disclosure of the initial offer by Future Pak LLC of $4.05 per share; - A 63% premium above Vanda’s closing share price on May 23, 2024, the day before Cycle submitted its proposal to the Vanda Board; - An all-cash upfront offer exceeding the cash portion of Future Pak’s revised proposal for Vanda on May 7, 2024; and - A 58% premium above Vanda’s closing share price on June 5, 2024. Cycle has substantial cash on hand, and both of Cycle’s financial advisors are highly confident that, following limited due diligence, committed financing will be put in place for the transaction. Cycle will obtain fully underwritten, binding commitment letters for any debt financing prior to signing a definitive merger agreement, which will not contain any financing contingency. Cycle issued the following statement regarding its proposal: “Our proposal for Vanda delivers immediate, compelling and certain cash value for Vanda shareholders with a highly attractive premium. Cycle’s proposal represents a better outcome for shareholders, who would receive all-cash upfront value exceeding that of Future Pak’s cash portion of its latest offer announced May 7, 2024. It would also benefit patients, as Cycle has a proven commercial strategy in the U.S., a strong distribution footprint and an established track record of delivering medicines and individualized support to patients suffering from conditions with high unmet medical need. While we would have preferred to reach an agreement privately, Cycle is publicly disclosing our proposal for the benefit of Vanda shareholders and to encourage Vanda shareholders to express their views on this proposal to the independent directors of the Vanda Board of Directors. Given our familiarity with Vanda, its brands and our extensive knowledge of the industry, we believe we can efficiently and quickly complete our diligence. Once we receive access to the required information, we believe that we can complete our due diligence within 2-3 weeks and reach a definitive agreement shortly thereafter. We stand ready to work immediately with Vanda’s Board and management team to reach an agreement that would provide a compelling premium and certain cash value today for all Vanda shareholders.” About Cycle Pharmaceuticals Cycle Pharmaceuticals was founded in 2012 with the sole aim of delivering drug treatments and product support to the underserved rare disease patient community, and the healthcare professionals and communities that support them. Cycle focuses on rare metabolic, immunological, and neurological genetic conditions. Within neurological conditions, Cycle focuses on multiple sclerosis. During May 2024, Cycle launched its 6th drug product in the U.S. market. Cycle’s U.S. commercial drug products are: NITYR® (nitisinone) Tablets (since 2017) SAJAZIR™ (icatibant) Injection (since 2021) JAVYGTOR™ (sapropterin dihydrochloride) Tablets and Powder (since 2022) TASCENSO ODT® (fingolimod) (since 2023) TIOPRONIN delayed-release tablets (since 2024) ORMALVI™ (dichlorphenamide) Tablets (since 2024) Cycle achieved $109m net sales and $40m operating profit in 2023 (according to FRS102 accounting principles). Extracts of the Company’s unaudited financial results for the year ending December 31, 2023 are available here. Cycle is headquartered in Cambridge, U.K. and has offices in Boston, Massachusetts. For more information, please visit and follow us on X, LinkedIn and Facebook. Additional Information The terms of this announcement are non-binding and intended solely to provide the basis on which Cycle is presently willing to negotiate definitive transaction documentation in respect of the proposal, and are not intended to, and should not be deemed or construed to, create or constitute any sort of binding offer, commitment, right or obligation on the part of Cycle, Vanda, Vanda’s shareholders, or their respective affiliates. Any such offer, commitment, right or obligation will come into existence only by the execution and delivery of binding definitive transaction documentation by the parties setting forth the terms and conditions of such offer, right, commitment or obligation, and will be subject in each case to the satisfaction or waiver of all applicable conditions. The failure for any reason to execute and deliver the definitive transaction documentation or consummate the proposal will impose no liability on any party hereto or their respective affiliates.

Financial StatementAcquisition

13 May 2024

·www.biospace.com

New tiopronin delayed-release tablets launch with patient support for the treatment of cystinuria in the US

BOSTON--(BUSINESS WIRE)-- Cycle Pharmaceuticals Limited (Cycle) is pleased to announce the launch of tiopronin delayed-release tablets as a treatment option for patients with cystinuria.1 Tiopronin delayed-release tablets are available in 100 mg and 300 mg doses, and are approved by the US Food and Drug Administration (FDA).1 Generic tiopronin delayed-release tablets must be taken without food.1 Cystinuria is the most common inheritable cause of kidney stone disease. Incidence of the condition is estimated to be 1 in 7,000 worldwide.2 The launch of tiopronin delayed-release tablets is a partnership between Cycle and Torrent Pharma Inc. (Torrent) utilizing Anovo Specialty Pharmacy (Anovo) in which all three companies are committed to providing patients with rare diseases the medication and patient support they need. Tiopronin delayed-release tablets are a treatment option for patients with cystinuria who want to benefit from the support services provided by Cycle Vita™, a dedicated support platform which provides individualized product support* for patients, and ongoing dispensing and support services provided by Anovo, the exclusive specialty pharmacy for this product. “Cystinuria is a chronic lifelong condition in which stones commonly reoccur.3 In addition, close control of a patient’s diet, specifically a reduction in salt and methionine intake, can have an impact on the likelihood of stones forming.3 We are fully aware of the unique personal challenges these patients face in effectively managing their cystinuria. That’s why we’re providing individualized support including a Bridge Program* and Co-pay Assistance Program*. In addition, a Patient Nutrition Program* will be available to reinforce the clinic’s nutrition plan,” says Chikai Lai, SVP & Chief Commercial Officer, Cycle. Tiopronin delayed-release tablets are Cycle’s fifth commercial product to be launched in the US, building on the launch of: NITYR® (nitisinone) Tablets in 2017 SAJAZIR™ (icatibant) Injection in 2021 JAVYGTOR™ (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution in 2022; and TASCENSO ODT® (fingolimod) in 2023 Tiopronin delayed-release tablets are indicated in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in adults and pediatric patients 9 years of age and older with severe homozygous cystinuria, who are not responsive to these measures alone. Tiopronin delayed-release tablets must be taken without food.1 To find out more about Cycle Vita, please visit or call +1 (888) 360-8482 and find the Full Prescribing Information available at . Important Safety Information Important Safety Information is below. Full Prescribing Information is available at . INDICATIONS Tiopronin delayed-release tablets are a reducing and complexing thiol indicated, in combination with high fluid intake, alkali, and diet modification, for prevention of cystine stone formation in adult and pediatric patients 9 years of age and older with severe homozygous cystinuria who are not responsive to these measures alone. WARNINGS AND PRECAUTIONS Proteinuria, including nephrotic syndrome, and membranous nephropathy, have been reported with tiopronin use. Pediatric patients receiving greater than 50 mg/kg of tiopronin per day may be at increased risk for proteinuria. Hypersensitivity reactions (drug fever, rash, fever, arthralgia and lymphadenopathy) have been reported. MOST COMMON ADVERSE REACTIONS Most common adverse reactions (≥10%) are nausea, diarrhea or soft stools, oral ulcers, rash, fatigue, fever, arthralgia, proteinuria, and emesis. DRUG INTERACTIONS Alcohol: Tiopronin is released faster from tiopronin delayed-release tablets in the presence of alcohol and the risk for adverse events associated with tiopronin delayed-release tablets when taken with alcohol is unknown. Avoid alcohol consumption 2 hours before and 3 hours after taking tiopronin delayed-release tablets. USE IN SPECIFIC POPULATIONS: Pregnancy: Available published case report data with tiopronin have not identified a drug-associated risk for major birth defects, miscarriage, or adverse maternal or fetal outcomes. Renal stones in pregnancy may result in adverse pregnancy outcomes. Tell your healthcare provider immediately if you become pregnant or planning to become pregnant. Lactation: There are no data on the presence of tiopronin in either human or animal milk or on the effects of the breastfed child. A published study suggests that tiopronin may suppress milk production. Because of the potential for serious adverse reactions, including nephrotic syndrome, advise patients that breastfeeding is not recommended during treatment with tiopronin delayed-release tablets. Tell your healthcare provider immediately if you plan on breast feeding. Pediatric Use: Tiopronin delayed-release tablets are indicated in pediatric patients 9 years of age and older with severe homozygous cystinuria, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation who are not responsive to these measures alone. Tiopronin delayed-release tablets are not approved for use in pediatric patients weighing less than 20 kg or in pediatric patients unable to swallow tablets. Geriatric Use: This drug is known to be substantially excreted by the kidney, and the risk of adverse reactions to this drug may be greater in patients with impaired renal function. Because elderly patients are more likely to have decreased renal function, care should be taken in dose selection, and it may be useful to monitor renal function. For more detailed information, please refer to the full Prescribing Information at . If you get any side effects, talk to your healthcare provider. This includes possible side effects not listed in this material. You may also report side effects directly by calling Torrent Pharma Inc. at: 1-800-912-9561 or FDA at: 1-800-FDA-1088 or . This material is for education purposes only. It is not intended to be, and should not be used as, a substitute for medical advice. US-TIO-2400004 (February 2024) References Tiopronin Delayed-Release Tablets. Prescribing Information. Torrent Pharma Inc. Leslie, S.W., Sajjad, H. and Nazzal, L. (2023). Cystinuria. [online] Nih.gov. Available at: [Accessed Feb 9 2024]. Cystinuria UK - Diet - what should I eat (2023). Cystinuria UK - Diet - what should I eat? [online] Cystinuriauk.co.uk. Available at: [Accessed Feb 9 2024]. * Some areas of support may not be accessible to all patients. Eligibility criteria may apply to ensure compliance with all applicable federal and state requirements, and benefits may be limited to commercially insured patients only. For more detailed information about eligibility, terms and conditions, please contact the Cycle Vita team at +1 (888) 360-8482. The US Prescribing Information provides the indications for which the product is approved in the US. Please consult the US Prescribing Information and product labeling for information about the specific uses for which the product is indicated in the US. –ENDS– Cycle Vita™, SAJAZIR™ and JAVYGTOR™ are trademarks of Cycle Pharmaceuticals Limited in the United States. NITYR® is a registered trademark of Cycle Pharmaceuticals Limited in the United States. TASCENSO ODT® is a registered trademark of Handa Neuroscience, LLC. ©2024 Cycle Pharmaceuticals Limited. All rights reserved. About Cycle Pharmaceuticals Cycle Pharmaceuticals was founded in 2012 with the sole aim of delivering drug treatments and product support to the underserved rare disease patient community. Cycle focuses on rare metabolic, immunological, and neurological genetic conditions. Within neurological conditions, we focus on multiple sclerosis. Cycle is headquartered in Cambridge, UK and has offices in Boston, Massachusetts. For more information, please visit and follow us on X, LinkedIn and Facebook. About Torrent Pharma Inc. Torrent Pharma is one of the leading Pharma companies in India. Torrent is the pioneer in initiating the concept of niche marketing in India and today is ranked amongst the leaders in therapeutic segment of cardiovascular (CV), central nervous system (CNS), gastro-intestinal (GI) and women healthcare (WHC). Torrent also has significant presence in diabetology, pain management, gynecology, oncology and anti-infective segments. Torrent Pharma is present in more than 40 countries. It is ranked first amongst Indian Companies for having largest market share in Brazil and Germany. About Anovo Anovo was founded in 2012 as an exclusive provider of comprehensive end-to-end services for manufacturers of rare disease drugs and patients who need the drugs, including specialty wholesale/3PL distribution services, specialty pharmacy services, and patient support hub programs. Anovo benefits all parties involved by navigating all the complexities of managing rare disease medications and providing the related support needed by patients, caregivers and prescribers. View source version on businesswire.com: Contacts Marketing@cyclepharma.com Cycle Pharmaceuticals Tel: +44 1223 354 118 Source: Cycle Pharmaceuticals Limited View this news release online at:

Drug Approval

30 Apr 2024

·www.biospace.com

Relief Therapeutics has Published its Annual Report

GENEVA, SWITZARLAND / ACCESSWIRE / April 30, 2024 / RELIEF THERAPEUTICS Holding SA (SIX:RLF)(OTCQB:RLFTF)(OTCQB:RLFTY) (Relief), a biopharmaceutical company committed to delivering innovative treatment options for select specialty, unmet and rare diseases, today published its Annual Report 2023 and provided a corporate update. "Our commitment to improving patient outcomes in rare diseases has never been stronger," said Michelle Lock, interim chief executive officer of Relief. "Realigning our business model and prioritizing our strategic approach has allowed us to optimize our operations and accelerate our priority programs. We are excited about the potential of RLF-TD011 to bring relief to patients suffering from epidermolysis bullosa, as well as about the development of RLF-OD032, for the treatment of phenylketonuria (PKU)." The Annual Report 2023 is available on the company's website. ABOUT RELIEF Relief is a commercial-stage biopharmaceutical company committed to advancing treatment paradigms and delivering improvements in efficacy, safety, and convenience to benefit the lives of patients living with select specialty and rare diseases. Relief's portfolio offers a balanced mix of marketed, revenue-generating products, our proprietary, globally patented TEHCLO™ and Physiomimic™ platform technologies and a targeted clinical development pipeline consisting of risk-mitigated assets focused in three core therapeutic areas: rare skin diseases, rare metabolic disorders, and rare respiratory diseases. In addition, Relief is commercializing several legacy products via licensing and distribution partners. Relief's mission is to provide therapeutic relief to those suffering from rare diseases and is being advanced by an international team of well-established, experienced biopharma industry leaders with extensive research, development and rare disease expertise. Relief is headquartered in Geneva and listed on the SIX Swiss Exchange under the symbol RLF. For more information, please visit our website or follow Relief on LinkedIn. CONTACT : RELIEF THERAPEUTICS Holding SA Avenue de Sécheron 15 1202 Geneva Switzerland contact@relieftherapeutics.com DISCLAIMER This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks, uncertainties, including its ability to achieve its corporate, development and commercial goals, and other factors which could cause the actual results, financial condition, performance or achievements of Relief to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. A number of factors, including those described in Relief's filings with the SIX Swiss Exchange and the U.S. Securities and Exchange Commission (SEC), could adversely affect Relief. Copies of Relief's filings with the SEC are available on the SEC EDGAR database at . Relief does not undertake any obligation to update the information contained herein, which speaks only as of this date. SOURCE: Relief Therapeutics Holdings AG View the original press release on accesswire.com

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.

to view more data

Indication	Country/Location	Organization	Date
Hyperphenylalaninemia, BH4-Deficient, B	EU	-	16 Feb 2022
Hyperphenylalaninemia, BH4-Deficient, B	IS	-	16 Feb 2022
Hyperphenylalaninemia, BH4-Deficient, B	LI	-	16 Feb 2022
Hyperphenylalaninemia, BH4-Deficient, B	NO	-	16 Feb 2022
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.

to view more data

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

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


SSIEM2022	Not Applicable	Phenylketonurias	80	Sapropterin dihydrochloride	whfwmvvxii(hupsbaabit) = cutvmmsmsr zlbbcdbszv (uknjskqjdg ) View more	-	30 Aug 2022
SSIEM2022	Not Applicable	Phenylketonurias	79	Sapropterin dihydrochloride	pukgfeawby(pumzunsnbh) = dewmoqahoh ukgumudsdu (odefzgfjrm, 52.1)	Positive	30 Aug 2022
NCT02677870 (CTgov)	Phase 4	Phenylketonurias	7	Diet treatment (Diet Treatment)	uvkjhugxub(pwbhbjnjmg) = luxrfrcvsk twigarazgb (eofqbpqbur, nbphutwyrw - wpzywpgkal) View more	-	27 Apr 2022
				Diet treatment+saproterin dihydrochloride (Standard Dose Saproterin Dihydrochloride)	uvkjhugxub(pwbhbjnjmg) = xwrcjkjgcz twigarazgb (eofqbpqbur, vepftjhtad - pazfadrbcs) View more
NCT01376908 (SPARK, Pubmed) Manual	Phase 3	Phenylketonurias	51	sapropterin (extension' group)	gynluuroqi(mqjgwqszlw) = timnjogrjd lziyhmupju (hfsgjzakmo, 2.8 ~ 13.8)	Positive	03 Aug 2021
				sapropterin (continuous' group)	gynluuroqi(mqjgwqszlw) = kwrjspzgsd lziyhmupju (hfsgjzakmo, 28.9 ~ 48.6)
NCT00532844 (CTgov)	Phase 2	Endothelial dysfunction	52	Sapropterin Dihydrochloride (Sapropterin Dihydrochloride)	rvmoxgioop(kvvqfpwwjf) = skbquwcpdp yccdnuphtg (xhodoxidcm, jckkucmmgz - tnuwbsauzo) View more	-	17 May 2021
				vitamin C+Sapropterin Dihydrochloride (Sapropterin Dihydrochloride+Vitamin C)	rvmoxgioop(kvvqfpwwjf) = kozwgdqjqk yccdnuphtg (xhodoxidcm, mvkuzmmulo - nzsnrdyide) View more
NCT02774226 (CTgov)	Phase 2	Pulmonary Disease, Chronic Obstructive	15	Tetrahydrobiopterin (BH4) (Tetrahydrobiopterin (BH4))	erwnxphoij(wgefoxidgn) = uvjwfrujuc ctruwbbbnq (opdirqyjoi, utnkffhwfb - ynyhpewidf) View more	-	23 Feb 2021
				Antioxidant Cocktail (Antioxidant Cocktail)	erwnxphoij(wgefoxidgn) = kfabkztfth ctruwbbbnq (opdirqyjoi, vejatnstsn - fmeaslaiaj) View more
NCT00445978 (CTgov)	Phase 2	Anemia, Sickle Cell	32	Sapropterin Dihydrochloride	argkuobvqv(zqqrhsiuwp) = rrpfrzunhv mluphyexdq (wcauyaetem, ivcoqwreec - zpcseitqpb) View more	-	28 Jan 2021
NCT00403494 (CTgov)	Phase 2	Intermittent Claudication \| Peripheral Arterial Disease	190	Sapropterin Dihydrochloride (Sapropterin Dihydrochloride)	roiyshhxxc(qwzllbptek) = flatlbqvmy lujzlprkjk (zpzwmkvoxz, dypzpjropn - lvxbzqsgmc) View more	-	07 Jan 2021
				Placebo (Placebo)	roiyshhxxc(qwzllbptek) = rgjffmaquk lujzlprkjk (zpzwmkvoxz, amwljeywps - gfgflhjukw) View more
NCT01439555 (CTgov)	Phase 2	Alzheimer Disease	11	Simvastatin+Tetrahydrobiopterin+L-Arginine	bwvesvcivd(iwpbedoanc) = vjxcassmqn mkfbfpbhrh (jcxcmjvelj, epvedsfvqb - kacssljtpo) View more	-	30 Jul 2019
NCT01706965 (CTgov)	Phase 1/2	Schizoaffective disorder \| Schizophrenia	27	Kuvan (Kuvan)	dcvtzdcjxz(fupyifxtej) = qvpqxbwtsz jkmdgurdxs (jfoynfhimy, fthligzphb - tpfnmurzos) View more	-	13 Sep 2017
				Multivitamin (Multivitamin)	dcvtzdcjxz(fupyifxtej) = kwvccbnsrq jkmdgurdxs (jfoynfhimy, vthamjmyjv - rfbyblnyrp) View more

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