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Chemistry

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Also known as: Arimoclomol, 289893-25-0, Arimoclomol (usan), Brx-220 free base, Dsstox_cid_31490, Dsstox_rid_97375
Molecular Formula
C14H20ClN3O3
Molecular Weight
313.78  g/mol
InChI Key
SGEIEGAXKLMUIZ-XUVDNFPMSA-N

Arimoclomol
1 2D Structure

Arimoclomol

2 Identification
2.1 Computed Descriptors
2.1.1 IUPAC Name
(3Z)-N-[(2R)-2-hydroxy-3-piperidin-1-ylpropoxy]-1-oxidopyridin-1-ium-3-carboximidoyl chloride
2.1.2 InChI
InChI=1S/C14H20ClN3O3/c15-14(12-5-4-8-18(20)9-12)16-21-11-13(19)10-17-6-2-1-3-7-17/h4-5,8-9,13,19H,1-3,6-7,10-11H2/b16-14-/t13-/m1/s1
2.1.3 InChI Key
SGEIEGAXKLMUIZ-XUVDNFPMSA-N
2.1.4 Canonical SMILES
C1CCN(CC1)CC(CON=C(C2=C[N+](=CC=C2)[O-])Cl)O
2.1.5 Isomeric SMILES
C1CCN(CC1)C[C@H](CO/N=C(/C2=C[N+](=CC=C2)[O-])\Cl)O
2.2 Synonyms
2.2.1 MeSH Synonyms

1. Arimoclomol

2. Brx-220

3. Brx220

2.2.2 Depositor-Supplied Synonyms

1. Arimoclomol

2. 289893-25-0

3. Arimoclomol (usan)

4. Brx-220 Free Base

5. Dsstox_cid_31490

6. Dsstox_rid_97375

7. Dsstox_gsid_57701

8. Chembl2107726

9. Dtxsid5057701

10. Schembl12254225

11. Tox21_113718

12. Ncgc00253575-01

13. Hy-106443

14. Cas-289893-25-0

15. Cs-0025833

16. D11374

17. (3z)-n-[(2r)-2-hydroxy-3-piperidin-1-ylpropoxy]-1-oxidopyridin-1-ium-3-carboximidoyl Chloride

2.3 Create Date
2006-10-23
3 Chemical and Physical Properties
Molecular Weight 313.78 g/mol
Molecular Formula C14H20ClN3O3
XLogP31.2
Hydrogen Bond Donor Count1
Hydrogen Bond Acceptor Count5
Rotatable Bond Count6
Exact Mass313.1193192 g/mol
Monoisotopic Mass313.1193192 g/mol
Topological Polar Surface Area70.5 Ų
Heavy Atom Count21
Formal Charge0
Complexity337
Isotope Atom Count0
Defined Atom Stereocenter Count1
Undefined Atom Stereocenter Count0
Defined Bond Stereocenter Count1
Undefined Bond Stereocenter Count0
Covalently Bonded Unit Count1
4 Pharmacology and Biochemistry
4.1 ATC Code

N - Nervous system

N07 - Other nervous system drugs

N07X - Other nervous system drugs

N07XX - Other nervous system drugs

N07XX17 - Arimoclomol


Drugs in Development

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Details:

Miplyffa (arimoclomol) is a first in class oral HSF1 activator approved for Niemann-Pick disease type C in combination with miglustat.


Lead Product(s): Arimoclomol,Miglustat

Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa

Study Phase: Phase II/ Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 21, 2024

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BIO Partnering at JPM
Not Confirmed
BIO Partnering at JPM
Not Confirmed

Details : Miplyffa (arimoclomol) is a first in class oral HSF1 activator approved for Niemann-Pick disease type C in combination with miglustat.

Brand Name : Miplyffa

Molecule Type : Small molecule

Upfront Cash : Not Applicable

November 21, 2024

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Details:

Miplyffa (Arimoclomol) is indicated for use in combination with miglustat for the treatment of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.


Lead Product(s): Arimoclomol,Miglustat

Therapeutic Area: Rare Diseases and Disorders Brand Name: Miplyffa

Study Phase: ApprovedProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 20, 2024

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02

BIO Partnering at JPM
Not Confirmed
BIO Partnering at JPM
Not Confirmed

Details : Miplyffa (Arimoclomol) is indicated for use in combination with miglustat for the treatment of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.

Brand Name : Miplyffa

Molecule Type : Small molecule

Upfront Cash : Not Applicable

September 20, 2024

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Details:

The net proceeds from this offering will used to support the pre-commercial launch activities for BRX-345 (arimoclomol) for the treatment of Niemann-Pick Disease Type C.


Lead Product(s): Arimoclomol

Therapeutic Area: Rare Diseases and Disorders Brand Name: BRX-345

Study Phase: Phase II/ Phase IIIProduct Type: Small molecule

Sponsor: Cantor

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Public Offering August 08, 2024

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BIO Partnering at JPM
Not Confirmed
BIO Partnering at JPM
Not Confirmed

Details : The net proceeds from this offering will used to support the pre-commercial launch activities for BRX-345 (arimoclomol) for the treatment of Niemann-Pick Disease Type C.

Brand Name : BRX-345

Molecule Type : Small molecule

Upfront Cash : Undisclosed

August 08, 2024

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Details:

BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.


Lead Product(s): Arimoclomol

Therapeutic Area: Rare Diseases and Disorders Brand Name: BRX-345

Study Phase: Phase II/ Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable August 02, 2024

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04

BIO Partnering at JPM
Not Confirmed
BIO Partnering at JPM
Not Confirmed

Details : BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.

Brand Name : BRX-345

Molecule Type : Small molecule

Upfront Cash : Not Applicable

August 02, 2024

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Details:

BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.


Lead Product(s): Arimoclomol

Therapeutic Area: Rare Diseases and Disorders Brand Name: BRX-345

Study Phase: Phase II/ Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 09, 2024

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05

BIO Partnering at JPM
Not Confirmed
BIO Partnering at JPM
Not Confirmed

Details : BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.

Brand Name : BRX-345

Molecule Type : Small molecule

Upfront Cash : Not Applicable

July 09, 2024

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Details:

BRX-345 (arimoclomol) is a first in class investigational HSF1 activator, which is being evaluated in phase 2/3 clinical trials for the treatment of Niemann-Pick disease type C.


Lead Product(s): Arimoclomol

Therapeutic Area: Rare Diseases and Disorders Brand Name: BRX-345

Study Phase: Phase II/ Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 04, 2024

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06

BIO Partnering at JPM
Not Confirmed
BIO Partnering at JPM
Not Confirmed

Details : BRX-345 (arimoclomol) is a first in class investigational HSF1 activator, which is being evaluated in phase 2/3 clinical trials for the treatment of Niemann-Pick disease type C.

Brand Name : BRX-345

Molecule Type : Small molecule

Upfront Cash : Not Applicable

March 04, 2024

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Details:

XOMA acquired royalty and milestone rights for LadRx Corporation’s BRX-345 (arimoclomol) for Niemann-Pick Disease Type C, and aldoxorubicin, an albumin-linked formulation of doxorubicin.


Lead Product(s): Arimoclomol

Therapeutic Area: Rare Diseases and Disorders Brand Name: BRX-345

Study Phase: Phase II/ Phase IIIProduct Type: Small molecule

Sponsor: XOMA

Deal Size: $58.6 million Upfront Cash: $5.0 million

Deal Type: Agreement January 11, 2024

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LadRx

U.S.A
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BIO Partnering at JPM
Not Confirmed

LadRx

U.S.A
arrow
BIO Partnering at JPM
Not Confirmed

Details : XOMA acquired royalty and milestone rights for LadRx Corporation’s BRX-345 (arimoclomol) for Niemann-Pick Disease Type C, and aldoxorubicin, an albumin-linked formulation of doxorubicin.

Brand Name : BRX-345

Molecule Type : Small molecule

Upfront Cash : $5.0 million

January 11, 2024

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Details:

Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.


Lead Product(s): Arimoclomol

Therapeutic Area: Rare Diseases and Disorders Brand Name: Undisclosed

Study Phase: Phase II/ Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 08, 2024

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08

BIO Partnering at JPM
Not Confirmed
BIO Partnering at JPM
Not Confirmed

Details : Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.

Brand Name : Undisclosed

Molecule Type : Small molecule

Upfront Cash : Not Applicable

January 08, 2024

blank

Details:

Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.


Lead Product(s): Arimoclomol

Therapeutic Area: Rare Diseases and Disorders Brand Name: Undisclosed

Study Phase: Phase II/ Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable December 27, 2023

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09

BIO Partnering at JPM
Not Confirmed
BIO Partnering at JPM
Not Confirmed

Details : Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.

Brand Name : Undisclosed

Molecule Type : Small molecule

Upfront Cash : Not Applicable

December 27, 2023

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Details:

Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. It is being developed as a treatment for Niemann-Pick disease type C (NPC).


Lead Product(s): Arimoclomol

Therapeutic Area: Rare Diseases and Disorders Brand Name: Undisclosed

Study Phase: Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 23, 2023

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10

BIO Partnering at JPM
Not Confirmed
BIO Partnering at JPM
Not Confirmed

Details : Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. It is being developed as a treatment for Niemann-Pick disease type C (NPC).

Brand Name : Undisclosed

Molecule Type : Small molecule

Upfront Cash : Not Applicable

February 23, 2023

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STOCK RECAP #PipelineProspector

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Pipeline Prospector Sept 2024: BMS wins landmark FDA approval for schizophrenia med; Sanofi’s Dupixent okayed for COPD
Pharma indices settled slightly lower in September after four months of solid gains. The three major pharma indices dropped about 2 percent each. The Nasdaq Biotechnology Index (NBI) closed at 4,767.84, down from 4,870.17, the SPDR S&P Biotech ETF (XBI) dropped to 98.8 from 101.1, and the S&P Biotechnology Select Industry Index (SPSIBI) fell to 7,707.36 from 7,897.9. Despite these declines, September was a dynamic month for the pharmaceutical industry, marked by significant regulatory approvals.September saw notable developments in the field of chronic obstructive pulmonary disease (COPD). After over a decade, COPD saw two significant approvals. The US Food and Drug Administration (FDA) expanded the label of Regeneron and Sanofi’s mega-blockbuster drug, Dupixent, to include the treatment of COPD. Already a game-changer in treating conditions like asthma and atopic dermatitis, Dupixent became the first-ever biologic medicine for COPD patients in the US.Additionally, GSK’s asthma drug Nucala scored a vital win in a late-stage study for treating COPD. The British drugmaker reported that the monoclonal antibody significantly and meaningfully reduced the annualized rate of moderate to severe exacerbations of what is the world’s third leading cause of death. These developments come on the heels of FDA’s approval of Verona’s Ohtuvayre in June. Ohtuvayre has a novel mechanism of action for the maintenance treatment of COPD. Access the Pipeline Prospector Dashboard for September 2024 Newsmakers (Free Excel)BMS’ Karuna bet pays off with schizo drug approval; FDA okays Astra’s nasal spray flu shotIn what was one of the most closely watched decisions this year, BMS’ KarXT won a landmark FDA approval for treating schizophrenia. Branded Cobenfy, the drug is the first antipsychotic that targets cholinergic receptors. Ever since the 1950s, when antipsychotics began being used to treat schizophrenia, they have worked by blocking a dopamine receptor. But those have been associated with serious side effects like weight gain, high rates of cardiac disease, early death and patients complaining about feeling sluggish and unmotivated. Cobenfy’s new approach has experts excited, and its peak sales are expected to come in at US$ 7.5 billion a year, validating BMS’ US$ 14 billion acquisition of Karuna Therapeutics. BMS’ stock gained 2 percent.That wasn’t the only landmark move. FDA approved two treatments for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disorder. Zevra Therapeutics’ Miplyffa became the first-ever treatment approved for NPC, addressing a critical unmet need for patients who, on average, only live about 13 years. Soon after Miplyffa’s approval, IntraBio’s Aqneursa was also granted approval by the FDA. Designed to alleviate neurological symptoms in both adults and children weighing at least 15 kilograms, Aqneursa stands out as the only FDA-approved stand-alone therapy for NPC.Just in time for winter, AstraZeneca’s first self-administered flu vaccine — FluMist — also bagged FDA approval last month. This nasal spray vaccine can be used at home, potentially increasing vaccination rates among needle-averse individuals. Access the Pipeline Prospector Dashboard for September 2024 Newsmakers (Free Excel) Astra’s Tagrisso okayed for inoperable lung cancer; UCB Bimzelx bags 3 new approvalsMonths after AstraZeneca’s Tagrisso posted impressive phase 3 results, the drug received FDA approval for treating inoperable, stage 3 epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC). This makes Astra’s blockbuster the first targeted therapy for the indication.UCB’s Bimzelx secured three new FDA approvals for treating adults with active psoriatic arthritis, non-radiographic axial spondyloarthritis, and ankylosing spondylitis. With a novel approach to treating these chronic inflammatory diseases, the company expects Bimzelx to significantly bolster sales, with peak sales of the med projected at € 4 billion (US$ 4.4 billion) or more.Eli Lilly’s Ebglyss was approved for moderate-to-severe eczema in patients who are unable to control symptoms with topical or other systemic treatments. Notably, Ebglyss offers a more convenient once-monthly dosing compared to twice-monthly for competitors, a feature that analysts note as attractive to both experts and patients. Access the Pipeline Prospector Dashboard for September 2024 Newsmakers (Free Excel)Astra-Daiichi’s ADC suffers double whammy; Intercept’s Ocaliva faces setbackThe month also brought its share of challenges. AstraZeneca (stock down 10 percent) and Daiichi Sankyo’s (stock down 23 percent) investigational antibody-drug conjugate (ADC) datopotamab deruxtecan suffered a double blow in late-stage trials. It first failed to significantly outperform the standard-of-care chemotherapy — docetaxel — in treating NSCLC. And then it failed to show notable improvement in overall survival compared to chemotherapy in breast cancer.Analysts had expected datopotamab deruxtecan to potentially become one of Astra’s best-selling drugs. The British-Swedish drugmaker had paid Daiichi US$ 1 billion upfront and promised an additional US$ 5 billion in milestone payments.Intercept Pharmaceuticals faced a major setback as FDA’s advisory committee voted against the confirmatory data for Ocaliva, which was granted accelerated approval in 2016 for primary biliary cholangitis.Vanda Pharmaceuticals hit back at FDA after the agency issued it a Complete Response Letter, declining to approve its drug — tradipitant — to treat gastroparesis. The company expressed deep frustration, highlighting the urgent need for new treatments. Access the Pipeline Prospector Dashboard for September 2024 Newsmakers (Free Excel)GSK’s asthma drug halves attacks; Abbvie’s Parkinson’s med meets all endpointsAmong late-stage wins, GSK’s new long-acting asthma drug, depemokimab, reduced asthma exacerbations by 54 percent and achieved a 72 percent reduction in exacerbations that required hospitalization or an ER visit. GSK is counting depemokimab among one of its 12 blockbuster launches and expects it to generate £ 3 billion (US$ 3.9 billion) in annual peak-sales.Similarly, Abbvie’s tavapadon, which was the subject of its US$ 8.7 billion buyout of Cerevel Therapeutics, met its primary and secondary endpoints in patients with early Parkinson's disease. Akeso and Summit Therapeutics’ experimental drug ivonescimab beat the world’s best-selling drug Keytruda hands down in a late-stage NSCLC trial. Ivonescimab reduced the risk of disease progression or death by 49 percent compared to Keytruda. Akeso and Summit’s stock jumped 40 percent and 69 percent, respectively.And, Ascendis Pharma announced topline data from a pivotal trial on TransCon CNP, a therapy to treat achondroplasia, a common form of dwarfism. TransCon CNP demonstrated a statistically significant annualized growth velocity of 5.89 cm/year in children treated, compared to 4.41 cm/year in the placebo group. Access the Pipeline Prospector Dashboard for September 2024 Newsmakers (Free Excel) Our viewWhile September didn’t witness a mega-deal, it did see groundbreaking advancements being signed off by the FDA. We have seen enough ups and downs in the pharma indices this year. Let’s hope the final quarter of the year ends on a strong note, both in terms of drug approvals and indices. Access the Pipeline Prospector Dashboard for September 2024 Newsmakers (Free Excel)

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03 Oct 2024

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ABOUT THIS PAGE

Arimoclomol Manufacturers

A Arimoclomol manufacturer is defined as any person or entity involved in the manufacture, preparation, processing, compounding or propagation of Arimoclomol, including repackagers and relabelers. The FDA regulates Arimoclomol manufacturers to ensure that their products comply with relevant laws and regulations and are safe and effective to use. Arimoclomol API Manufacturers are required to adhere to Good Manufacturing Practices (GMP) to ensure that their products are consistently manufactured to meet established quality criteria.

Arimoclomol Suppliers

A Arimoclomol supplier is an individual or a company that provides Arimoclomol active pharmaceutical ingredient (API) or Arimoclomol finished formulations upon request. The Arimoclomol suppliers may include Arimoclomol API manufacturers, exporters, distributors and traders.

Arimoclomol GMP

Arimoclomol Active pharmaceutical ingredient (API) is produced in GMP-certified manufacturing facility.

GMP stands for Good Manufacturing Practices, which is a system used in the pharmaceutical industry to make sure that goods are regularly produced and monitored in accordance with quality standards. The FDA’s current Good Manufacturing Practices requirements are referred to as cGMP or current GMP which indicates that the company follows the most recent GMP specifications. The World Health Organization (WHO) has its own set of GMP guidelines, called the WHO GMP. Different countries can also set their own guidelines for GMP like China (Chinese GMP) or the EU (EU GMP).

PharmaCompass offers a list of Arimoclomol GMP manufacturers, exporters & distributors, which can be sorted by USDMF, JDMF, KDMF, CEP (COS), WC, API price, and more, enabling you to easily find the right Arimoclomol GMP manufacturer or Arimoclomol GMP API supplier for your needs.

Arimoclomol CoA

A Arimoclomol CoA (Certificate of Analysis) is a formal document that attests to Arimoclomol's compliance with Arimoclomol specifications and serves as a tool for batch-level quality control.

Arimoclomol CoA mostly includes findings from lab analyses of a specific batch. For each Arimoclomol CoA document that a company creates, the USFDA specifies specific requirements, such as supplier information, material identification, transportation data, evidence of conformity and signature data.

Arimoclomol may be tested according to a variety of international standards, such as European Pharmacopoeia (Arimoclomol EP), Arimoclomol JP (Japanese Pharmacopeia) and the US Pharmacopoeia (Arimoclomol USP).

Inform the supplier about your product requirements, specifying if you need a product with particular monograph like EP (Ph. Eur.), USP, JP, BP, or any other quality. In addition, clarify whether you need hydrochloride (HCl), anhydricum, base, micronisatum or a specific level of purity. To find reputable suppliers, utilize the filters and select those certified by GMP, FDA, or any other certification as per your requirement.
For your convenience, we have listed synonyms and CAS numbers to help you find the best supplier. The use of synonyms and CAS numbers can be helpful in identifying potential suppliers, but it is crucial to note that they might not always indicate the exact same product. It is important to confirm the product details with the supplier before making a purchase to ensure that it meets your requirements.
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