Delving into the Latest Updates on RPH-104 with Synapse

Overview

Basic Info

Drug Type

Fusion protein

Synonyms

Goflikicept

Target

IL-1β

Mechanism

IL-1β inhibitors(Interleukin-1 beta inhibitors)

Therapeutic Areas

Infectious DiseasesCardiovascular DiseasesRespiratory Diseases+ [3]

Active Indication

Idiopathic Recurrent PericarditisCOVID-19Pericarditis+ [5]

Inactive Indication

PainPrimary goutSARS-CoV-2 acute respiratory disease+ [2]

Originator Organization

TRPHARM Ilaç Sanayi Tic. AS

Active Organization

R-Pharm International, LLCR-Pharm Overseas, Inc.

TRPHARM Ilaç Sanayi Tic. AS

Inactive Organization

R-Pharm GroupR-Pharm JSC

Drug Highest PhasePhase 3

First Approval Date-

Regulation-

Gene Sequence

Sequence Code 9964977

Source: *****

Sequence Code 9964965

Source: *****

The primary objective of the study is to evaluate the efficacy of RPH-104 when administered at a dose of 160 mg on Day 0, Day 7, Day 21 and then once every 2 weeks (Q2W) subcutaneous (SC) in patients with Adult Onset Still's Disease (AOSD). Furthermore, the study is scheduled to investigate pharmacokinetic (PK) and pharmacodynamic (PD) parameters of RPH-104.

Start Date01 Dec 2023

Sponsor / Collaborator

R-Pharm International, LLC

[+1]

NCT05107934 / Not yet recruitingPhase 2/3

International, Double-blind, Randomized, Placebo-controlled Study to Evaluate the Efficacy and Safety of RPH-104 Treatment in Patients With Recurrent Pericarditis

The primary purpose of this study is the evaluation of the efficacy and safety of RPH-104 treatment in patients with recurrent pericarditis.
Pharmacokinetic and pharmacodynamic parameters of RPH-104 multiple doses in this patient population will be assessed as well.

Start Date01 Dec 2023

Sponsor / Collaborator

R-Pharm Overseas, Inc.

[+2]

NCT04213274 / Not yet recruitingPhase 2

A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Phase 2 Study of the Efficacy and Safety of RPH-104 in Adult Subjects With Schnitzler Syndrome

The primary goal of the study is to assess the efficacy and safety of RPH-104 in subjects with Schnitzler Syndrome using Schnitzler Disease Activity Score (SDAS), which includes the Physician's Global Assessment (PGA) and the local laboratory C-reactive protein (CRP) result

Start Date01 May 2023

Sponsor / Collaborator

R-Pharm Overseas, Inc.

[+1]

100 Clinical Results associated with RPH-104

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100 Translational Medicine associated with RPH-104

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100 Patents (Medical) associated with RPH-104

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5

Literatures (Medical) associated with RPH-104

01 Sep 2023·Current cardiology reports

Paradigm Shift in Diagnosis and Targeted Therapy in Recurrent Pericarditis.

Review

Author: Wang, Tom Kai Ming ; Dong, Tiffany ; Klein, Allan L

PURPOSE OF THE REVIEWWe review the pathophysiology, diagnosis, and contemporary treatment for recurrent pericarditis, with focus on interleukin-1 (IL-1) inhibitors.RECENT FINDINGSRecurrent pericarditis occurs in about 15-30% of patients who have acute pericarditis. With increased understanding of the autoinflammatory pathophysiology of recurrent pericarditis, IL-1 inhibitors including anakinra, canakinumab, and rilonacept have been applied to this condition with great promise. In particular, the RHAPSODY trial found rilonacept significantly improves pain and inflammation, while also reducing recurrence with few adverse events. The next IL-1 inhibitor on the block for pericarditis, goflikicept, is also discussed. Understanding the role of the inflammasome via the autoinflammatory pathway in pericarditis has led to incorporation of IL-1 inhibitors in the treatment of recurrent pericarditis, with proven efficacy and safety and randomized trials. This will lead to increase uptake of this agent which demonstrated lower rates of recurrence and faster time to resolution.

04 Jul 2023·Journal of the American College of Cardiology

Treatment of Idiopathic Recurrent Pericarditis With Goflikicept: Phase II/III Study Results.

Article

Author: Lavrovsky, Yan ; Abbate, Antonio ; Gleykina, Ekaterina V ; Egorova, Alina N ; Myachikova, Valentina Yu ; Schedrova, Margarita L ; Samsonov, Mikhail Yu ; Grishin, Sergey A ; Maslyanskiy, Alexey L ; Moiseeva, Olga M ; Vinogradova, Oksana V

BACKGROUNDIdiopathic recurrent pericarditis (IRP) is a rare autoinflammatory disease. Interleukin (IL)-1α and IL-1β are the pivotal cytokines in the pathophysiology of acute pericarditis and its recurrence. We created a phase II/III study with a new IL-1 inhibitor-goflikicept in IRP.OBJECTIVESThis study sought to evaluate the efficacy and safety of goflikicept treatment in patients with IRP.METHODSWe conducted a 2-center open-label study of goflikicept in patients with IRP with and without recurrence at time of enrollment. The study consisted of 4 periods: screening, run-in (open-label treatment period), randomized withdrawal, and follow-up. Patients with clinical response to goflikicept in the run-in period were randomized (1:1) to a placebo-controlled withdrawal period, where the time to first pericarditis recurrence (primary endpoint) was evaluated.RESULTSWe enrolled 22 patients, and 20 of these patients were randomized. Reduction of C-reactive protein level accompanied by reduction of chest pain and pericardial effusion compared to baseline was demonstrated during the run-in period. Recurrence of pericarditis occurred in 9 of 10 patients in the placebo group, and there were no recurrence events in goflikicept group within 24 weeks after randomization (P < 0.001). A total of 122 adverse events were reported in 21 patients (95.5%), with no deaths and no new safety signals identified for goflikicept.CONCLUSIONSTreatment with goflikicept prevented recurrences and maintained IRP remission with a favorable risk-benefit ratio. Goflikicept reduced the risk of recurrence compared with placebo. (Study to Evaluate the Efficacy and Safety of RPH-104 Treatment in Patients With Idiopathic Recurrent Pericarditis; NCT04692766).

01 Dec 2021·Journal of Translational MedicineQ2 · MEDICINE

Interleukin-1 blockade with RPH-104 in patients with acute ST-elevation myocardial infarction: study design and rationale

Q2 · MEDICINE

ArticleOA

Author: Bogin, V ; Abbate, A ; Van Tassell, B W ; Samsonov, M

AbstractBackgroundMyocardial injury of ST-segment elevation myocardial infarction (STEMI) initiates an intense inflammatory response that contributes to further damage and is a predictor of increased risk of death or heart failure (HF). Interleukin-1 (IL-1) is a key mediator of local and systemic inflammatory response to myocardial damage. We postulate that the use of the drug RPH-104, which selectively binds and inactivates both α and β isoforms of IL-1 will lead to a decrease in the severity of the inflammatory response which will be reflected by decrease in the concentration of hsCRP, as well as the rate of fatal outcomes, frequency of new cases of HF, changes in levels of brain natriuretic peptide (BNP) and changes in structural and functional echocardiographic parameters.MethodsThis is a double blind, randomized, placebo-controlled study in which 102 patients with STEMI will receive a single administration of RPH-104 80 mg, RPH-104 160 mg or placebo (1:1:1). The primary endpoint will be hsCRP area under curve (AUC) from day 1 until day 14. Secondary endpoints will include hsCRP AUC from day 1 until day 28, rate of fatal outcomes, hospitalizations due to HF and other cardiac and non-cardiac reasons during 12-month follow-up period, frequency of new cases of HF, changes in levels of brain natriuretic peptide (BNP, NT-pro-BNP), changes in structural and functional echocardiographic parameters during 12-month follow-up period compared to baseline. The study started in October 2020 and is anticipated to end in 2Q 2022.Trial registration: ClinicalTrials.gov, NCT04463251. Registered on July 9, 2020

1

News (Medical) associated with RPH-104

21 Jan 2021

·www.biospace.com

R-Pharm to study RPH-104 in acute ST-Elevation Myocardial Infarction

Moscow, January 21st, 2021. R-Pharm announces first patients enrolled in international double blind placebo-controlled clinical trial of RPH-104 in acute ST-Elevation Myocardial Infarction (STEMI). The study will enroll 102 patients in Russia and the USA receiving single subcutaneous injection. Thereafter patients will stay under cardiologist supervision for one year in order to evaluate the effect on parameters of systemic inflammation and disease outcomes. Mikhail Samsonov, Chief Medical Officer, R-Pharm: “Acute myocardial infarction is the most prevalent form of coronary heart disease characterized by high mortality. Interleukin-1 (IL-1) plays important role in inflammatory response after myocardial infarction and is involved in cardiac remodeling. Inflammatory response contributes to further cardiac damage and is a predictor of increased risk of death or heart failure. We believe that inhibition of IL-1 by RPH-104 could lead to better outcomes in patients with STEMI.” RPH-104 is an original biological drug developed at the R-Pharm laboratory in the USA, which is a new heterodimeric protein inhibiting IL-1. The drug is developed for the treatment of a number of socially significant and orphan diseases characterized by systemic inflammation, including acute myocardial infarction, Schnitzler syndrome, COVID-19, recurrent pericarditis, familial Mediterranean fever, and gout. The United States Food and Drug Administration (US FDA) has designated RPH-104 as orphan for the treatment of Schnitzler syndrome. There are currently no drugs approved by the FDA for the treatment of Schnitzler syndrome. ST-segment elevation myocardial infarction (STEMI) is a clinical syndrome of acute myocardial ischemia and necrosis associated with high risk of in-hospital and long-term morbidity and mortality. According to the European Register, mortality among patients with STEMI can exceed 12% over 6 months, during the first year is about 10% and over 5 years can reach 20%. About R-Pharm Group Headquartered in Moscow, Russia, R-Pharm Group is an integrated specialty pharmaceutical company, focused on research and development, manufacturing and commercialization of pharmaceuticals, laboratory equipment and medical devices. R-Pharm was founded in 2001 and occupies leading positions in the Russian healthcare market ever since. Operating in more than 30 countries and having a big number of production facilities in Russia and CIS, a plant in Germany and a research lab in the USA, R-Pharm succeeds in manufacturing and distributing of medicines to treat cancer, viral infections, autoimmune and other severe diseases to meet patients` needs. Media Contact: Valeria Andronenkova – Global Communications Specialist, R-Pharm Group andronenkova@rpharm.ru Mobile: +7 9166249614

100 Deals associated with RPH-104

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External Link

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB16396

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Idiopathic Recurrent Pericarditis	Phase 3	RU	R-Pharm International, LLC	11 Dec 2020
COVID-19	Phase 3	-	TRPHARM Ilaç Sanayi Tic. AS	-
ST Elevation Myocardial Infarction	Phase 2	-	R-Pharm Overseas, Inc.	07 Dec 2020
Myocardial Infarction	Phase 2	RU	R-Pharm Group	06 Dec 2020
Severe Acute Respiratory Syndrome	Phase 2	RU	R-Pharm International, LLC	23 Apr 2020
Pain	Phase 2	RU	TRPHARM Ilaç Sanayi Tic. AS	23 Mar 2018
Pericarditis	Preclinical	RU	R-Pharm Group	05 Mar 2020
SARS-CoV-2 acute respiratory disease	Discovery	RU	R-Pharm Group	09 Apr 2020

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04067492 (CTgov)	Phase 2	Primary gout	47	RPH - 104 (RPH - 4 mg)	(vlklgjoblp) = iasurhialp niwhyixgfw (zrxpmobido, rqzwhvevzx - kfdmcswzqz) View more	-	25 Jul 2022
				RPH - 104 (RPH - 20 mg)	(vlklgjoblp) = lulrsaekap niwhyixgfw (zrxpmobido, btfndzvsla - octqedrbmv) View more
NCT04463251 (CTgov)	Phase 2	Inflammation \| ST Elevation Myocardial Infarction	102	Placebo+RPH-104 80 mg (RPH-104 80 mg)	vjeedlypcy(dcvzgrzesf) = bokoijymkn bslvgqhsrs (verntbubtj, lxngiskgav - hbntnwhnaz) View more	-	24 Jan 2024
				RPH-104 80 mg (RPH-104 160 mg)	vjeedlypcy(dcvzgrzesf) = zjkrwbbeao bslvgqhsrs (verntbubtj, akclulkizl - tauukulffy) View more
NCT02667639 (CTgov)	Phase 1	-	35	RPH-104 (Treatment)	(cwydnprnnl) = gazkoyzfba todmdufzgn (thvieoweio, xjnlwheidu - qlliqoanoi) View more	-	04 Oct 2019
				Sodium chloride Sterile Injection 0.9% w/v (Placebo)	(cwydnprnnl) = nsqmtypxtv todmdufzgn (thvieoweio, jhzntfxstw - hggzvcrghy) View more
NCT04380519 (CTgov)	Phase 2/3	COVID-19 \| Severe Acute Respiratory Syndrome	372	RPH-104 80 mg (RPH -104 80 mg)	(sytsawiojx) = itsbkubxjf owirrafzxj (mqzsiqqdph, cuvtwmxjsr - glbdsqxrof) View more	-	24 Jan 2022
				Olokizumab 64 mg (Olokizumab 64 mg)	(sytsawiojx) = rbkgbduhzv owirrafzxj (mqzsiqqdph, kgrlkvtkzu - kztkalnijd) View more
NCT02667639 (ACR2017) Manual	Phase 1	-	35	Rph-104	(rceuacfmzt) = Totally 70 adverse events (AE) were reported, none of them were serious. bckvkfgwtz (xcozdowjvd )	Positive	18 Sep 2017