Monday, 28 September 2026

Ranosidenib

 

Ranosidenib

CAS 2301974-60-5

MF C15H16F9N5O MW 453.31 g/mol

(1S)-3-[4,6-bis[[(2R)-1,1,1-trifluoropropan-2-yl]amino]-1,3,5-triazin-2-yl]-2,6,6-trifluorocyclohex-2-en-1-ol

(1S)-3-(4,6-bis{[(2R)-1,1,1-trifluoropropan-2-yl]amino}-1,3,5-triazin-2-yl)-2,6,6-trifluorocyclohex-2-en-1-ol
isocitrate dehydrogenase (IDH) inhibitor, antineoplastic, [14C] HMPL-306, HMPL 306, PC64OXS2C2

  • OriginatorHutchison MediPharma
  • DeveloperHUTCHMED
  • ClassAntineoplastics; Small molecules
  • Mechanism of ActionIsocitrate dehydrogenase 1 inhibitors; Isocitrate dehydrogenase 2 inhibitors
  • Phase IIIAcute myeloid leukaemia
  • No development reportedHaematological malignancies; Solid tumours
  • 28 Sep 2025No recent reports of development identified for phase-I development in Haematological-malignancies(Late-stage disease, Second-line therapy or greater) in Spain (PO)
  • 28 Sep 2025No recent reports of development identified for phase-I development in Haematological-malignancies(Late-stage disease, Second-line therapy or greater) in USA (PO)
  • 19 Sep 2025No development reported - Phase-I for Solid tumours (Late-stage disease, Metastatic disease, Second-line therapy or greater) in USA (PO)

Ranosidenib is a small molecule drug. Ranosidenib is under investigation in clinical trial NCT06387069 (A Study to Evaluate HMPL-306 in Patients With IDH1- and IDH2-mutated Acute Myeloid Leukemia). Ranosidenib has a monoisotopic molecular weight of 453.12 Da.

Ranosidenib is an orally bioavailable inhibitor of mutated forms of both isocitrate dehydrogenase type 1 (IDH1, IDH1 [NADP+] soluble) in the cytoplasm and type 2 (IDH2, isocitrate dehydrogenase [NADP+], mitochondrial) in the mitochondria, with potential antineoplastic activity. Upon administration, ranosidenib specifically targets and inhibits mutant forms of IDH1 and IDH2, thereby inhibiting the formation of the oncometabolite 2-hydroxyglutarate (2HG) from alpha-ketoglutarate (a-KG). This prevents 2HG-mediated signaling and leads to both an induction of cellular differentiation and an inhibition of cellular proliferation in tumor cells expressing IDH mutations. IDH1 and 2, metabolic enzymes that catalyze the conversion of isocitrate into a-KG, play key roles in energy production and are mutated in a variety of cancer cell types. Mutant forms of IDH1 and 2 catalyze the formation of 2HG and drive cancer growth by blocking cellular differentiation and inducing cellular proliferation.

A Study of HMPL-306 in Advanced Hematological Malignancies With mIDHCTID: NCT04764474Phase: Phase 1Status: TerminatedDate: 2026-01-29

A Study of HMPL-306 in Advanced Solid Tumors With IDH MutationsCTID: NCT04762602Phase: Phase 1Status: TerminatedDate: 2025-09-16

A Study to Evaluate HMPL-306 in Patients With IDH1or IDH2-mutated Acute Myeloid LeukemiaCTID: NCT06387069Phase: Phase 3Status: RecruitingDate: 2025-08-14

Phase I Study of HMPL-306 for the Treatment of Gliomas With IDH1 and/or IDH2 MutationsCTID: NCT07025018Phase: Phase 1Status: RecruitingDate: 2025-08-01

A Study of HMPL-306 in Patients With IDH1 and/or IDH2 Mutation of Relapsed/Refractory Myeloid Leukemia/NeoplasmsCTID: NCT04272957Phase: Phase 1Status: Unknown statusDate: 2020-06-16

SYN

https://pubs.acs.org/doi/10.1021/acsmedchemlett.4c00625

aReagents and conditions: (i) Na2PdCl4, DTBPPS, K2CO3, MeCN, H2O, 60 ℃; (ii) TBSOTf, Et3N,
DCM, 0~5 ℃; Selectfluor®, MeCN, 0~5 ℃; (iii) TBSOTf, Et3N, DCM, 0~5 ℃; Selectfluor®,
MeCN, 0~5 ℃; (iv) NaBH4, CeCl3·7H2O, EtOH, 0 ℃; (v) SFC separation.

Pat

Cycloolefin substituted heteroaromatic compounds and their use

Publication Number: US-2021363115-A2

Priority Date: 2017-09-07

PAT

https://patentscope.wipo.int/search/en/detail.jsf?docId=US325708969&_cid=P22-MMFPFM-26361-1

Intermediate I-3

6-Chloro-N2,N4-bis((R)-1,1,1-trifluoropropan-2-yl)-1,3,5-triazine-2,4-diamine

At 0° C., to a flask were added 1,4-dioxane (50 mL), 2,4,6-trichloro-1,3,5-triazine (1.84 g, 10 mmo), (R)-1,1,1-trifluoropropan-2-amine hydrochloride (2.99 g, 20 mmol) and DIEA (5.17 g, 40 mmol). The reaction was heated to 60° C. and stirred for 4 hours. After the reaction was completed, the mixture was condensed and purified by flash column chromatography (eluting with gradient water/MeOH=100:0-0:100) to give Intermediate I-3 as yellow solid (2.50 g, yield: 74%). MS (m/z): 338.0 [M+H] +

Compounds 197 and 198

3-(4,6-Bis(((R)-1,1,1-trifluoropropan-2-yl)amino)-1,3,5-triazin-2-yl)-2,6,6-trifluorocyclohex-2-en-1-ol, optically pure diastereoisomers

The Compound 196 was resolved by chiral HPLC to provide a pair of optically pure diastereoisomers, Compounds 197 and 198 (Chiral HPLC conditions: Preparation instrument: Shimadzu LC-10AD vp; Column: Daicel AD-H(250 mm*30 mm, 5 um); mobile phase: n-heptane/isopropanol=90/10; flow rate: 40 mL/min; column temperature: 40° C.). The first eluent (RT=4.203 min) was concentrated and purified by flash column chromatography (eluting with gradient PE/EA=100:0-0:100) to give a compound named as Compound 197, de %=99.27%, MS (m/z): 454.1 [M+1] +. The second eluent (RT=5.906 min) was concentrated and purified by flash column chromatography (eluting with gradient PE/EA=100:0-0:100) to give a compound named as Compound 198, de %=97.82%, MS (m/z): 454.2 [M+1] +.
      Compound 197: 1H NMR (400 MHz, CD 3OD): δ 5.00-4.86 (m, 2H), 4.36-4.17 (m, 1H), 2.80-2.65 (m, 1H), 2.58-2.42 (m, 1H), 2.25-2.05 (m, 2H), 1.37-1.31 (m, 6H).
      Compound 198: 1H NMR (400 MHz, CD 3OD): δ 5.00-4.86 (m, 2H), 4.36-4.17 (m, 1H), 2.80-2.65 (m, 1H), 2.58-2.42 (m, 1H), 2.25-2.05 (m, 2H), 1.37-1.31 (m, 6H).
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/////////ranosidenib, isocitrate dehydrogenase (IDH) inhibitor, antineoplastic, [14C] HMPL-306, HMPL 306, PC64OXS2C2

#ranosidenib, #isocitrate dehydrogenase (IDH) inhibitor, #antineoplastic, #[14C] HMPL-306, #HMPL 306, #PC64OXS2C2

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Relicpixant

 

Relicpixant

CAS 2445366-94-7

MF C20H19ClF2N4O5S, Mw 500.9 g/mol

methyl (2S)-2-{[7-chloro-2-(2,6-difluoro-4-sulfamoylphenyl)imidazo[1,2-a]pyridin-3-yl]methyl}morpholine-4-
carboxylate
purinoreceptor (P2X) antagonist, 3NWJ8FHG2R

Relicpixant is a small molecule drug. The usage of the INN stem '-pixant' in the name indicates that Relicpixant is a purinoreceptor (P2X) antagonist. Relicpixant has a monoisotopic molecular weight of 500.07 Da.

SYN

US20240034729,

https://patentscope.wipo.int/search/en/detail.jsf?docId=US393005051&_cid=P10-MMH5CF-50302-1

Example 172

Step (8) Preparation of methyl (S)-2-((7-chloro-2-(2,6-difluoro-4-sulfamoylphenyl)imidazo[1,2-a]pyridin-3-yl)methyl)morpholine-4-carboxylate

Aqueous ammonia (2 mL) was diluted with acetonitrile (1 mL) and added dropwise to the above reaction system at 0° C. The reaction system was reacted at room temperature for 0.5 h. The starting material was consumed completely, and a target product was generated as detected by LCMS. The reaction system was extracted with water and ethyl acetate twice, washed with saturated brine, dried over anhydrous sodium sulfate, and concentrated. The residue was purified by preparative chromatography to give compound 172 (185 mg, 99.74% purity) in the form of a white solid. LC-MS: [M+H] +=501.1.
       1H NMR (400 MHZ, DMSO-d 6) δ=8.11 (d, J=7.4, 1H), 7.29 (d, J=1.6, 1H), 7.22 (s, 2H), 7.14 (d, J=6.6, 2H), 6.60 (dd, J=7.4, 2.1, 1H), 3.33 (d, J=12.8, 1H), 3.13 (d, J=11.3, 2H), 3.07 (s, 3H), 2.97 (d, J=7.8, 1H), 2.77-2.69 (m, 1H), 2.69-2.61 (m, 1H), 2.53 (dd, J=15.5, 8.3, 1H).

PAT

https://patentscope.wipo.int/search/en/detail.jsf;jsessionid=2DC90C13ECD61FAEE133C661D1AB5451.wapp1nA?docId=US438195702&_cid=P10-MMH55V-46834-1

Example 1: Preparation of Compound of Formula A

 To the above reaction mixture was added dropwise ammonia water (2 mL) diluted with acetonitrile (1 mL) at 0° C., and reaction mixture was reacted at room temperature for 0.5 hours. The raw material completely disappeared, and a target product was generated, as shown by LC-MS. The reaction mixture was extracted twice with water and ethyl acetate, washed with brine solution, dried over anhydrous sodium sulfate, concentrated, and purified by C18 chromatography column (water/acetonitrile, RRt=22.5 min). Compound A in an amorphous state (compound A is the compound of formula A) (185 mg, purity: 99.74%) was obtained as a white solid. LC-MS: [M+H]=501.1.
       1H NMR (400 MHz, DMSO-d 6) δ=8.11 (d, J=7.4, 1H), 7.29 (d, J=1.6, 1H), 7.22 (s, 2H), 7.14 (d, J=6.6, 2H), 6.60 (dd, J=7.4, 2.1, 1H), 3.33 (d, J=12.8, 1H), 3.13 (d, J=11.3, 2H), 3.07 (s, 3H), 2.97 (d, J=7.8, 1H), 2.77-2.69 (m, 1H), 2.69-2.61 (m, 1H), 2.53 (dd, J=15.5, 8.3, 1H).

Step 6: Preparation of methyl (S)-2-((7-chloro-2-(2,6-difluoro-4-sulfamoylphenyl)imidazo[1,2-a]pyridin-3-yl)methyl)morpholine-4-carboxylate AS ABOVE

PAT

https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2020135771&_cid=P10-MMH5AS-49292-1

PAT

PAT

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///////relicpixant, purinoreceptor (P2X) antagonist, 3NWJ8FHG2R

#relicpixant, #purinoreceptor (P2X) antagonist, #3NWJ8FHG2R

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Remlifanserin

 

Remlifanserin

CAS 2289704-13-6

MF C24H29F2N3O2 MW 429.5 g/mol

3-[(4-cyclopropyloxyphenyl)methyl]-1-[(2,4-difluorophenyl)methyl]-1-(1-methylpiperidin-4-yl)urea

N'-{[4-(cyclopropyloxy)phenyl]methyl}-N-[(2,4-difluorophenyl)methyl]-N-(1-methylpiperidin-4-yl)urea
serotonin receptor (5-HT2A) inverse agonist, ACP-204, ACP 204, H4L2AF2XB7

Remlifanserin is a small molecule drug. The usage of the INN stem '-anserin' in the name indicates that Remlifanserin is a serotonin receptor antagonist. Remlifanserin has a monoisotopic molecular weight of 429.22 Da.

Remlifanserin (INNTooltip International Nonproprietary Name;[4] developmental code name ACP-204) is a selective serotonin 5-HT2A receptor inverse agonist which is under development for the treatment of Alzheimer's disease psychosis.[1][5][6][7][8][9] It is taken by mouth.[1]

The drug is an improved follow-up compound to its developer's earlier drug pimavanserin (Nuplaizid; ACP-103).[6] It is more potent and selective than pimavanserin as a serotonin 5-HT2A receptor inverse agonist.[10] Remlifanserin shows 32- to 123-fold selectivity for antagonism and inverse agonism of the serotonin 5-HT2A receptor over the serotonin 5-HT2C receptor depending on the bioassay.[10] For comparison, pimavanserin's selectivity was 8- to 37-fold depending on the assay.[10] Remlifanserin shows very low affinity for the serotonin 5-HT2B receptor compared to the serotonin 5-HT2A and 5-HT2C receptors.[10] It is expected to have less QT prolongation than pimavanserin.[10] The drug blocks the head-twitch response induced by the serotonergic psychedelic DOI and the hyperlocomotion induced by the NMDA receptor antagonist dizocilpine (MK-801) in rodents.[10]

Remlifanserin is under development by Acadia Pharmaceuticals.[1][5] As of January 2025, it is in phase 3 clinical trials.[1][5] Its clinicaltrials.gov identifier (nct number) is NCT06159673.[11]

SYN

https://patentscope.wipo.int/search/en/detail.jsf?docId=US304654267&_cid=P10-MMILCB-95793-1

Example 17: 3-[(4-cyclopropoxyphenyl)methyl]-1-[(2,4-difluorophenyl)methyl]-1-(1-methylpiperidin-4-yl)urea; hemitartrate (17)

3-[(4-cyclopropoxyphenyl)methyl]-1-[(2,4-difluorophenyl)methyl]-1-(1-methylpiperidin-4-yl)urea; hemitartrate

      N-[(2,4-difluorophenyl)methyl]-1-methylpiperidin-4-amine (1.89 mmol, 478 mg), phenyl N-[(4-cyclopropoxyphenyl)methyl]carbamate (97%, 585 mg, 2.0 mmol) and potassium carbonate (2.5 mmol, 350 mg) were suspended in toluene (5.0 ml). The mixture was stirred at 70° C. for 16 hours, then partitioned between toluene and sodium hydroxide (aqueous, 0.5 M). The organic phase was separated and concentrated. The material was purified by silica gel chromatography, eluting with 0-50% methanol in ethyl acetate. Fractions containing the desired product were pooled and concentrated. Diethyl ether (10 ml) was added. The mixture was filtered and concentrated to give 3-[(4-cyclopropoxyphenyl)methyl]-1-[(2,4-difluorophenyl)methyl]-1-(1-methylpiperidin-4-yl)urea (725 mg, 1.688 mmol, 89% yield). This material (725 mg, 1.688 mmol) and L-(+)-tartaric acid (0.844 mmol, 127.3 mg) were dissolved in ethanol (5.0 ml) using an ultrasonication bath. The solvents were then evaporated to give the title compound as the hemitartrate salt (glassy foam, 906 mg). 1H NMR (400 MHz, Chloroform-d) δ 7.16 (q, 1H), 7.05 (d, 2H), 6.94 (d, 2H), 6.84-6.73 (m, 2H), 4.70 (bt, 1H), 4.62-4.48 (m, 1H), 4.41 (s, 2H), 4.33 (s, 1H), 4.28 (d, 2H), 3.70 (m, 1H), 3.42 (t, 2H), 2.72-2.56 (m, 2H), 2.63 (s, 3H), 2.18 (m, 2H), 1.81 (d, 2H), 0.76 (m, 4H); LC-MS: 430.3 [M+H] +.

PAT

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Clinical data
Other namesACP-204; ACP204
Routes of
administration		Oral[1]
Drug classSerotonin 5-HT2A receptor inverse agonist
Pharmacokinetic data
Onset of action4–6 hours (6 hours fasted, 9 hours fed) (TmaxTooltip time to peak levels)[2][3]
Elimination half-life17.8–19.8 hours[2]
Identifiers
IUPAC name
CAS Number2289704-13-6
PubChem CID137520242
UNIIH4L2AF2XB7
Chemical and physical data
FormulaC24H29F2N3O2
Molar mass429.512 g·mol−1
3D model (JSmol)Interactive image
SMILES
InChI

References

  1.  "ACP 204". AdisInsight. 23 January 2025. Retrieved 22 February 2025.
  2.  Darwish M, Feng X, Dirks B, Raether B, Pathak SS (2025). "Pharmacokinetics in Healthy Adult and Elderly Patients of ACP-204, a Novel 5-HT 2A Receptor Selective Antagonist/Inverse Agonist". Alzheimer's & Dementia. 21 (S5) e105732. doi:10.1002/alz70859_105732. ISSN 1552-5260. PMC 12741707.
  3.  Darwish M, Dirks B, Feng X, Raether B, Pathak SS (2025). "Effect of Food Consumption on the Pharmacokinetics of ACP-204, a Novel 5-HT 2A Receptor Selective Antagonist/Inverse Agonist". Alzheimer's & Dementia. 21 (S5) e105644. doi:10.1002/alz70859_105644. ISSN 1552-5260. PMC 12741626.
  4.  "Proposed INN: List 131 International Nonproprietary Names for Pharmaceutical Substances (INN)" (PDF). WHO Drug Information. 38 (2): 421. 2024. remlifanserin N'-{[4-(cyclopropyloxy)phenyl]methyl}-N-[(2,4- difluorophenyl)methyl]-N-(1-methylpiperidin-4-yl)urea serotonin receptor (5-HT2A) inverse agonist [...] C24H29F2N3O2 2289704-13-6 [...]
  5.  "Delving into the Latest Updates on ACP-204 with Synapse". Synapse. 4 February 2025. Retrieved 22 February 2025.
  6.  "ACP-204". ALZFORUM. 5 February 2024. Retrieved 22 February 2025.
  7.  Imbimbo C, Cotta Ramusino M, Leone S, Mazzacane F, De Franco V, Gatti A, et al. (February 2025). "Emerging Pharmacological Approaches for Psychosis and Agitation in Alzheimer's Disease". CNS Drugs. 39 (2): 143–160. doi:10.1007/s40263-024-01133-9. PMC 11769872. PMID 39623197.
  8.  IsHak WW, Meyer A, Freire L, Totlani J, Murphy N, Renteria S, et al. (2024). "Overview of Psychiatric Medications in the Pipeline in Phase III Trials as of June 1, 2024: A Systematic Review". Innovations in Clinical Neuroscience. 21 (7–9): 27–47. PMC 11424068. PMID 39329027.
  9.  Kwon KJ, Kim HY, Han SH, Shin CY (October 2024). "Future Therapeutic Strategies for Alzheimer's Disease: Focus on Behavioral and Psychological Symptoms". International Journal of Molecular Sciences. 25 (21) 11338. doi:10.3390/ijms252111338. PMC 11547068. PMID 39518892.
  10.  Burstein E, Markus Dey P, Pathak S (December 2024). "ACNP 63rd Annual Meeting: Poster Abstracts P305-P608: P497. Nonclinical Characterization of ACP-204, a Novel Second Generation 5-HT2A Inverse Agonist" (PDF). Neuropsychopharmacology. 49 (Suppl 1): 236–417 (346–347). doi:10.1038/s41386-024-02012-z. PMID 39643634.
  11.  ACADIA Pharmaceuticals Inc. (2025-02-21). A Master Protocol for Three Independent, Seamlessly Enrolling, Double-blind, Placebo-controlled Efficacy and Safety Studies of ACP-204 in Adults with Alzheimer's Disease Psychosis (Report). clinicaltrials.gov.

////////////remlifanserin, ANAX, serotonin receptor (5-HT2A) inverse agonist, ACP-204, ACP 204, H4L2AF2XB7

#remlifanserin, #ANAX, #serotonin receptor (5-HT2A) inverse agonist, #ACP-204, #ACP 204, #H4L2AF2XB7

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Repinatrabit

 

Repinatrabit

CAS 2837993-05-0

MF C18H22F4N4O3 MW 418.4 g/mol

(3R)-3-[cyclopropyl-[[2-fluoro-4-(trifluoromethoxy)phenyl]methylcarbamoyl]amino]piperidine-1-carboxamide

(3R)-3-[cyclopropyl({[2-fluoro-4-(trifluoromethoxy)phenyl]methyl}carbamoyl)amino]piperidine-1-
carboxamide
solute carrier family 6 member 19 (SLC6A19) inhibitor(phenylketonuria), JNT-517, JNT 517, orphan drug, rare pediatric disease designations, Jnana Therapeutics, 5P44NDU6AC, JN 11804, JN-11804

Repinatrabit (JNT-517) is an investigational, oral, small-molecule drug developed by Jnana Therapeutics (now part of Otsuka Pharmaceutical) to treat Phenylketonuria (PKU). It acts as a selective inhibitor of the SLC6A19 transporter, reducing blood phenylalanine (Phe) levels by increasing its urinary excretion. 

Key Details About Repinatrabit:

  • Mechanism: It targets a novel, cryptic allosteric site to block kidney reabsorption of phenylalanine, aiming to be a first-in-class oral therapy for all PKU patients, regardless of age or genotype.
  • Clinical Trials: Otsuka initiated a global Phase 3 study (NCT06971731) in December 2025 to evaluate its safety and efficacy, following positive results from earlier studies.
  • Status: The FDA has granted it orphan drug and rare pediatric disease designations.
  • A Study to Evaluate the Safety and Efficacy of JNT-517 in Participants With Phenylketonuria (PKU)CTID: NCT06971731Phase: Phase 3Status: RecruitingDate: 2026-02-04
  • A Phase 2 Study of JNT-517 in Adolescent Participants With PhenylketonuriaCTID: NCT06637514Phase: Phase 2Status: RecruitingDate: 2025-08-19
  • First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With PhenylketonuriaCTID: NCT05781399Phase: Phase 1/Phase 2Status: Active, not recruitingDate: 2025-07-31
  • A Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants With PhenylketonuriaCTID: NCT06628128Phase: Phase 3Status: Not yet recruitingDate: 2025-06-03

EMA Drug Information,

Type, Orphan designations

Active Substance,

(R)-3-(1-Cyclopropyl-3-(2-fluoro-4-(trifluoromethoxy)benzyl)ureido)piperidine-1-carboxamide

Intended Use, Treatment of hyperphenylalaninaemia, Status of Orphan Designation, Positive

First Published Date, 2024-08-22

PAT

https://patentscope.wipo.int/search/en/detail.jsf?docId=US463725634&_cid=P12-MMK00V-42348-1

SYN

https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2024118721&_cid=P12-MMJZU4-37251-1

PAT

https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2025217521&_cid=P12-MMJZU4-37251-2

PAT

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///////////repinatrabit, ANAX, JNT-517, JNT 517, orphan drug, rare pediatric disease designations, Jnana Therapeutics, 5P44NDU6AC, JN 11804, JN-11804

#repinatrabit, #ANAX, #JNT-517, #JNT 517, #orphan drug, #rare pediatric disease designations, #Jnana Therapeutics, #5P44NDU6AC, #JN 11804, #JN-11804

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Sunday, 27 September 2026

Rivasterat

 

Rivasterat

CAS 2446590-96-9

MF C37H54O8 MW626.8 g/mol

methyl (E)-6-[(3S,8S,9S,10R,13S,14S,17R)-3-[[(2R,3S,6S)-3-acetyloxy-2-(acetyloxymethyl)-3,6-dihydro-2H-pyran-6-yl]oxy]-10,13-dimethyl-2,3,4,7,8,9,11,12,14,15,16,17-dodecahydro-1H-cyclopenta[a]phenanthren-17-yl]hept-5-enoate

27-NORCHOLESTA-5,20(22)-DIEN-26-OIC ACID, 3-((4,6-DI-O-ACETYL-2,3-DIDEOXY-.ALPHA.-D-ERYTHRO-HEX-2-ENOPYRANOSYL)OXY)-, METHYL ESTER, (3.BETA.,20E)-

methyl (20E)-3β-[(4,6-di-O-acetyl-2,3-dideoxy-α-Derythro-hex-2-enopyranosyl)oxy]-27-norcholesta5,20(22)-dien-26-oate
cholesterol-derived steroid, anti-inflammatory, CURACLE, CU-06, CU-06-RE, CU06-1004, CU06-CERE/CV, CU06-EYE, CU06-HAE, CU06-IBD, CU06-ONCO, Sac-1004, 2X23JA5AKW

  • OriginatorCURACLE
  • ClassAnti-inflammatories; Anti-ischaemics; Antineoplastics; Eye disorder therapies; Ischaemic heart disorder therapies; Small molecules; Vascular disorder therapies
  • Mechanism of ActionActin modulators; Chemokine CCL2 inhibitors; Histamine release inhibitors; Interleukin 1 beta inhibitors; Thrombin inhibitors; Vascular endothelial growth factors inhibitors
  • Phase IIDiabetic macular oedema
  • No development reportedAge-related macular degeneration; Cancer; Crohn's disease; Diabetic retinopathy; Hereditary angioedema; Lung disorders; Macular degeneration; Myocardial infarction; Retinal oedema; Stroke; Ulcerative colitis; Unstable angina pectoris; Wet macular degeneration
  • 28 Aug 2025No recent reports of development identified for research development in Unstable-angina-pectoris in South Korea (PO)
  • 28 Jul 2025No recent reports of development identified for phase-I development in Wet macular degeneration in USA (PO)
  • 28 May 2025No recent reports of development identified for phase-I development in Age-related-macular-degeneration in South Korea (PO)

Developer and Code Name

  • Original code name: CU06-1004
  • Developer: Curacle Co., Ltd. (South Korea)
  • Drug class: Endothelial dysfunction blocker (EDB)

This class of drugs aims to restore endothelial barrier integrity rather than directly blocking VEGF like most retinal drugs.

The molecule contains:

  • Steroid (cyclopenta[a]phenanthrene) core
  • Unsaturated heptenoate side chain
  • Acetylated sugar moiety (pyranose)

This glycosylated steroid structure is unusual for vascular-protective drugs.

Clinical Development

Phase I (Healthy Volunteers)

Key findings:

  • Dose tested: 100–1200 mg
  • Exposure increased more than dose proportional
  • Food greatly increased absorption
  • No significant drug accumulation after repeated dosing
  • Minimal renal excretion detected

Phase II

Early clinical trials investigated oral therapy for diabetic macular edema with improvements in:

  • Best-corrected visual acuity
  • Inflammatory biomarkers

Summary

ItemDetails
DrugRivasterat (CU06-1004)
OriginatorCuracle
Core patentWO2013011939
Chemistrysteroid glycoside
Key stepsteroid glycosylation
Priority~2011
Expiry~2031–2033

SYNTHESIS

WO2013011939

US20140148474

EP2741074

KR20130007373

SYN

https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2021225233&_cid=P11-MMMURO-25899-1

Manufacturing Example  1> Manufacturing of compound 1

[170]Compound 1 represented by the following chemical formula 1 can be manufactured using the manufacturing method described in Korean unpublished patent application number 10-2019-0166864. Specifically, it can be manufactured using the method according to the following reaction scheme 1 or reaction scheme 2.

Step    1:    Preparation of 6-1

A thermometer was installed in a 5 L flask, and 200 g (0.632 mol) of pregnenolone was added to 2000 mL of dichloromethane, and 173 mL (1.896 mol) of 3,4-dihydro-2H-pyran was added. After lowering the temperature to 0-5 ℃, 3.0 g (15.8 mmol) of p-toluenesulfonic acid monohydrate dissolved in 50 mL of tetrahydrofuran (THF) was added dropwise and stirred at 0 ℃ for 1.5 hours. At 0 ℃, 800 mL of saturated sodium bicarbonate aqueous solution and 10 mL of triethylamine (TEA) were added to the reaction mixture and stirred. After separating the layers, the organic layer was washed with 800 mL of brine, and the aqueous layers were extracted again with 200 mL of dichloromethane, combined into the organic layers, dried over 200 g of anhydrous sodium sulfate, filtered, and distilled under reduced pressure. 1000 mL of MeOH and 5 mL of TEA were added to the obtained residue, heated to completely dissolve, and the temperature was lowered and stirred at -5 °C for 1 hour. The resulting solid was filtered and washed with 200 mL of MeOH to obtain 232.0 g (0.579 mol) of 6-1 (THP-Pregnenolone) as a pure white solid in a yield of 91.6%. 

[204]1H-NMR (400 MHz, CDCl 3): δ 5.33-5.36 (m, 1H), 4.71-4.72 (m, 1H), 3.85-3.94 (m, 1H), 3.46-3.56 (m, 2H), 1.00-2.55 (m, 32H), 0.62 (s, 3H).

 Step  2:  Preparation of 4-2

After installing a condenser, heating mantle, and mechanical stirrer in a 5L reactor, the reactor was heated to 119℃ (external temperature), cooled to room temperature while flowing nitrogen for 5 minutes, dried, and 332.5 g (0.75 mol) of 4-(carboxybutyl)triphenylphosphonium bromide and 168.1 g (1.50 mol) of potassium t-butoxide were added. Then, 2000 mL of anhydrous toluene and 750 mL of anhydrous tetrahydrofuran were added, and the reactor was heated to 119℃ (external temperature, internal mild reflux) and stirred for about 2 hours. 

[209]6-1 100.0 g (0.250 mol) was dissolved in 500 mL of anhydrous toluene, added to the reaction solution, and reacted for about 20 hours. After the reaction was completed, the reaction mixture was cooled to room temperature, 320 mL (5.14 mol) of methyl iodide and 1000 mL of acetone were added, and stirred at room temperature for 15 hours. Most of the organic solvent was removed from the reaction mixture by distillation under reduced pressure, 1500 mL of ethyl acetate was added to dissolve, and the mixture was washed with 1000 mL of saturated ammonium chloride aqueous solution. The organic layer was washed twice with 1000 mL of water and 1000 mL of brine, dried with 100 g of sodium sulfate, filtered using 80 g of Celite, and concentrated. 

[210]The obtained residue was dissolved in 2000 mL of methanol, stirred at 10°C for 13 hours and at 4-5°C for 1 hour, and the resulting solid was filtered, washed with 200 mL of methanol, and dried in vacuum to obtain 66.2 g of 4-2 as a white solid with a yield of 53.2%. 

[211]

1H NMR(400MHz, CDCl 3): δ 5.36(t, J=5.80 Hz, 1H), 5.16(t, J=7.00 Hz, 1H), 4.71(m, 1H), 3.93(m, 1H), 3.66(s, 3H), 3.56(m, 2H), 2.37-0.88(m, 38H), 0.54(s, 3H).

Step    3:    Preparation of 2-1

After installing a thermometer and a water bath in a 1 L flask, 42.0 g (0.101 mol) of compound 2-1 and 34.5 g (0.126 mol) of triiO-acetyl D-glucal were dissolved in 126 mL of anhydrous toluene and 252 mL of acetonitrile, and while maintaining the temperature at 30-35 ℃, 3.87 g (0.0130 mol) of lithium nonafluoro-1-butylsulfonate and 0.117 g (0.0005 mol) of (s)-camphor sulfonic acid were added and stirred for 2 hours. After completion of the reaction, the reaction was quenched with 504 mL of saturated sodium bicarbonate aqueous solution and extracted with 630 mL of heptane. The organic layer was washed twice with 504 mL of saturated sodium bicarbonate aqueous solution and then with 504 mL of brine. The organic layer was stirred with 42 g of anhydrous sodium sulfate and 34 g of charcoal, filtered with 34 g of celite, washed with 210 mL of methylene chloride, combined with the filtrate, concentrated, and dried under vacuum. 

[222]

[223]

1H-NMR (400 MHz, CDCl3) : δ 5.79-5.88 (m, 2H), 5.35-5.36 (m, 1H), 5.27-5.29 (m, 1H), 5.12-5.16 (m, 2H), 4.15-4.24 (m, 3H), 3.66 (s, 3H), 3.54-3.57 (m, 1H), 0.91-2.32 (m, 38H), 0.54 (s, 3H).

PAT

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////////rivasterat, cholesterol-derived steroid, anti-inflammatory, CURACLE, CU-06, CU-06-RE, CU06-1004, CU06-CERE/CV, CU06-EYE, CU06-HAE, CU06-IBD, CU06-ONCO, Sac-1004, 2X23JA5AKW

#rivasterat, #cholesterol-derived steroid, #anti-inflammatory, #CURACLE, #CU-06, #CU-06-RE, #CU06-1004, #CU06-CERE/CV, #CU06-EYE, #CU06-HAE, #CU06-IBD, #CU06-ONCO, #Sac-1004, #2X23JA5AKW

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