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Zokinvy

(lonafarnib)

ApprovedSmall molecule

SSentynl Therapeutics, Inc.

Lonafarnib is used to treat progeria, a rare genetic disease that causes rapid aging in children. It works by slowing down the production of an abnormal protein that damages cells.[2] Zokinvy is currently approved and in use across 16 indications[4].[1]

Zokinvy is one of 398 small molecule programs targeting Glioma. It is currently in Approved.

16

Indications

27

Total Trials

2

Active Trials

397

Competitors

1

FDA Application

NDA/BLA/ANDA regulatory filings

1

Patents

Pipeline

Status by Indication[4][1]

Indication

GliomaActive, not recruiting

MetastasisApproved

HepatitisActive, not recruiting

Central Nervous System NeoplasmApproved

Progeroid SyndromeActive, not recruiting

Hutchinson-Gilford Progeria SyndromeActive, not recruiting

Chronic HepatitisActive, not recruiting

Chronic Myelogenous LeukemiaActive, not recruiting

Liver CancerActive, not recruiting

Chronic Myelomonocytic LeukemiaActive, not recruiting

GliomaPhase 1

Active, not recruiting

MetastasisApproved

Approved

HepatitisPhase 2

Active, not recruiting

Central Nervous System NeoplasmApproved

Approved

Progeroid SyndromeApproved

Active, not recruiting

Hutchinson-Gilford Progeria SyndromePhase 2

Active, not recruiting

Chronic HepatitisPhase 2

Active, not recruiting

Chronic Myelogenous LeukemiaPhase 1

Active, not recruiting

Liver CancerPhase 2

Active, not recruiting

Chronic Myelomonocytic LeukemiaPhase 3

Active, not recruiting

Showing 10 of 16 indications

Competition

Competitive Landscape

2828 competitors in Breast Cancer

#DrugCompanyPhaseTrials

Zokinvy

SSentynl Therapeutics, Inc.

Trodelvy

Halaven

Adriamycin

GGynecologic Oncology Group

Gemzar

GGynecologic Oncology Group

Feraheme

AAzurity Pharmaceuticals

Chirocaine

PPurdue Pharma LP

Systemic Therapy

Bristol-Myers Squibb

Vfend

PPrism Pharmaceuticals

Aquasol E

NNational Eye Institute (NEI)

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Clinical

Trials by Indication[1]

Breast Cancer3 trials

Lonafarnib, Trastuzumab, and Paclitaxel in Treating Patients With HER2/Neu-Overexpressing Stage IIIB, Stage IIIC, or Stage IV Breast Cancer

Completed·EEuropean Organisation for Research and Treatment of Cancer - EORTC·Phase 1·NCT00068757·23 enrolled

Anastrozole Plus Lonafarnib (SCH 66336) or Plus Placebo for the Treatment of Advanced Breast Cancer (P03480)

Completed· Merck & Co. logo

Merck & Co.·Phase 2·NCT00081510·110 enrolled

Lonafarnib in Metastatic Breast Cancer

Terminated·GGeorge Sledge·Phase 2·NCT00773474·29 enrolled

Advanced Cancer3 trials

Interaction of Docetaxel and Lonafarnib in Patients With Advanced Cancer

Terminated·EEmory University·Phase 1·NCT00288444·38 enrolled

SCH 66336 Plus Gemcitabine in Treating Patients With Advanced Cancer of the Urinary Tract

Completed·EEuropean Organisation for Research and Treatment of Cancer - EORTC·Phase 2·NCT00006351·34 enrolled

Combination Chemotherapy in Treating Patients With Advanced Cancer

Completed·MMemorial Sloan Kettering Cancer Center·Phase 1·NCT00003956·25 enrolled

Hepatitis2 trials

Treatment of Chronic Delta Hepatitis With Lonafarnib, Ritonavir and Lambda Interferon

Completed·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT03600714·26 enrolled

Treatment of Chronic Delta Hepatitis With Lonafarnib and Ritonavir

Completed·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT02511431·22 enrolled

Hutchinson-Gilford progeria syndrome2 trials

Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy

Approved for marketing·EEiger BioPharmaceuticals··NCT03895528

Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)

Active, not recruiting·PPRG Science & Technology Co., Ltd.·Phase 2·NCT06775041·10 enrolled

Chronic Myelomonocytic Leukemia1 trial

Study of Lonafarnib Versus Placebo in Subjects With Either Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) (Study P02978AM3)(TERMINATED)

Terminated· Merck & Co. logo

Merck & Co.·Phase 3·NCT00109538·47 enrolled

14 more indications

Progeria1 trial

A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria

Completed·—·Phase 2·NCT00879034·5 enrolled

Phase II1 trial

Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria

Completed·—·Phase 2·NCT00425607·29 enrolled

Chronic Myelogenous Leukemia1 trial

Study of Lonafarnib and Gleevec in Chronic Myelogenous Leukemia

Completed·MM.D. Anderson Cancer Center·Phase 1·NCT00047502·23 enrolled

Non-Small Cell Lung Cancer1 trial

Study of Lonafarnib in Combination With Paclitaxel and Carboplatin in Patients With Non-Small Cell Lung Cancer (Study P01901)(TERMINATED)

Terminated· Merck & Co. logo

Merck & Co.·Phase 3·NCT00050336·702 enrolled

Chronic Hepatitis1 trial

Lonafarnib for Chronic Hepatitis D

Completed·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT01495585·14 enrolled

Brain Tumors1 trial

SCH 66336 in Treating Children With Recurrent or Progressive Brain Tumors

Completed·PPediatric Brain Tumor Consortium·Phase 1·NCT00015899·53 enrolled

TED1 trial

Lonafarnib Boosted With Ritonavir With and Without Peginterferon Alfa-2a (PEG IFN-a) in HDV (LOWR-2)

Completed·EEiger BioPharmaceuticals·Phase 2·NCT02430194·55 enrolled

Efficacy and Safety1 trial

Study of the Efficacy and Safety of Lonafarnib / Ritonavir With and Without Pegylated Interferon -Alfa-2a

Completed·EEiger BioPharmaceuticals·Phase 3·NCT03719313·407 enrolled

Liver Cancer1 trial

SCH 66336 With or Without Gemcitabine Followed by Surgery Compared With Surgery Alone in Treating Patients With Primary Liver Cancer

Withdrawn·JJonsson Comprehensive Cancer Center·Phase 2·NCT00020774

Hepatitis D Virus Infection1 trial

Once Daily Dosing of Lonafarnib Co-administered With Ritonavir for Treatment of Chronic Hepatitis D Virus Infection

Unknown status·SSoroka University Medical Center·Phase 3·NCT05229991·30 enrolled

Glioma1 trial

Lonafarnib and Temozolomide in Treating Patients With Recurrent Primary Supratentorial Gliomas

Unknown status·EEuropean Organisation for Research and Treatment of Cancer - EORTC·Phase 1·NCT00083096·30 enrolled

Ovarian Cancer1 trial

Comparison of Paclitaxel/Carboplatin and Lonafarnib to Paclitaxel/Carboplatin for First-line Treatment of Ovarian Cancer

Completed·AAGO Study Group·Phase 2·NCT00281515·105 enrolled

Glioblastoma Multiforme1 trial

Lonafarnib and Temozolomide in Treating Patients with Glioblastoma Multiforme That is Recurrent or Did Not Respond to Previous Treatment with Temozolomide

Active, not recruiting·MM.D. Anderson Cancer Center·Phase 1·NCT00102648·34 enrolled

Unspecified3 trials

Lonafarnib With and Without Ritonavir in HDV (LOWR-1)

Completed·EEiger BioPharmaceuticals·Phase 2·NCT02430181·21 enrolled

Titrating-Dose of Lonafarnib in Combination With Ritonavir

Completed·EEiger BioPharmaceuticals·Phase 2·NCT02527707·15 enrolled

A Study of Lonafarnib With or Without Ritonavir in Patients With HDV

Withdrawn·EEiger BioPharmaceuticals·Phase 2·NCT02968641

Regulatory

FDA Approval Timeline[5]

Nov 20, 2020

ZOKINVY

NDA NDA213969

Sources

References

[1]
ClinicalTrials.gov — 27 trial records for lonafarnib. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Zokinvy drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — lonafarnib compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for lonafarnib. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA213969). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Zokinvy. U.S. Food & Drug Administration.
[7]
SEC EDGAR — Sentynl Therapeutics, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Protein Farnesyltransferase Inhibitor[3]
Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: SSentynl Therapeutics, Inc.

Patent Landscape[6]

Patent Cliff

Jul 26, 2029

Earliest patent expiration — generic entry possible after this date

Patent #Expires

7838531Jul 26, 2029

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