MedPath

RAPT Therapeutics Discontinues Zelnecirnon Program Following FDA Safety Concerns

• RAPT Therapeutics halts its zelnecirnon (RPT193) program after the FDA placed clinical holds on Phase 2 trials for asthma and atopic dermatitis due to liver injury. • The clinical hold was initiated after a patient in the atopic dermatitis trial experienced a serious adverse event involving liver injury requiring a transplant. • RAPT plans to advance next-generation CCR4 compounds with improved safety profiles and expects to identify a new candidate in the first half of 2025. • The company will also actively pursue in-licensing opportunities for clinical-stage assets to bolster its pipeline after this setback.

RAPT Therapeutics, Inc. (Nasdaq: RAPT) has announced the termination of its zelnecirnon (RPT193) program after the U.S. Food and Drug Administration (FDA) placed clinical holds on two Phase 2 trials evaluating the drug in asthma and atopic dermatitis. The decision follows safety concerns raised by the FDA after a serious adverse event (SAE) of liver injury requiring a transplant occurred in one patient participating in the atopic dermatitis trial. No other instances of liver toxicity or treatment-related SAEs were reported among other trial participants. The company closed both studies before completing planned enrollment.

Safety Concerns Prompt Program Halt

The clinical holds were implemented in February 2024 after the single case of severe liver injury was reported. This adverse event led the FDA to request additional safety data and prompted a thorough review of the zelnecirnon program. "In light of the agency’s feedback, we do not see a viable path forward for zelnecirnon," stated Brian Wong, M.D., Ph.D., President and CEO of RAPT. Despite this setback, Dr. Wong affirmed the company's belief in CCR4 as a promising target for inflammatory diseases.

Pivot to Next-Generation CCR4 Compounds

RAPT Therapeutics is now shifting its focus to developing next-generation CCR4 compounds with the goal of improved safety margins. The company anticipates identifying a new drug candidate in the first half of 2025. This strategic pivot aims to leverage the potential of CCR4 inhibition while mitigating the safety risks observed with zelnecirnon. The company is also actively seeking in-licensing opportunities for clinical-stage assets to expand its pipeline.

Company Statement and Future Plans

"We plan to continue advancing our next generation CCR4 compounds with improved safety margins for inflammatory disease and expect to identify a new candidate in the first half of 2025," Dr. Wong commented. The company's focus remains on developing oral small molecule therapies for inflammatory diseases and oncology, addressing significant unmet needs in these areas. RAPT Therapeutics will continue to advance its preclinical pipeline and explore external opportunities to strengthen its portfolio.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

RAPT Therapeutics Discontinues Zelnecirnon Program Following FDA Feedback

RAPT Therapeutics halts the development of zelnecirnon (RPT193) after receiving feedback from the FDA regarding safety concerns.
The decision follows a clinical hold placed on Phase 2 trials for asthma and atopic dermatitis due to a severe liver injury in one patient.

RAPT Therapeutics Discontinues Zelnecirnon Program After FDA Feedback, Stock Plummets

RAPT Therapeutics halts the development of zelnecirnon (RPT193) for inflammatory disorders following unfavorable feedback from the FDA after a clinical hold.
The FDA's decision followed a serious adverse event involving liver injury and a subsequent transplant in a patient during zelnecirnon trials.

RAPT Therapeutics Halts Zelnecirnon Development After FDA Feedback and Liver Injury

RAPT Therapeutics discontinues the development of zelnecirnon, a CCR4 antagonist, following regulatory feedback from the FDA and a case of liver failure in trials.
The FDA had previously placed clinical holds on Phase IIb and Phase IIa trials of zelnecirnon for atopic dermatitis and asthma due to safety concerns.

FDA Updates: Clinical Hold on Rapport's RAP-219 for DPNP, Phase 2 Trial for Mivelsiran in CAA, and Review of Ataluren Resubmission

The FDA has placed a clinical hold on Rapport Therapeutics' RAP-219 IND application for diabetic peripheral neuropathic pain (DPNP) due to protocol design concerns.
A phase 2 trial (cAPPricorn-1) will evaluate Alnylam's mivelsiran, an RNAi therapeutic targeting APP, for cerebral amyloid angiopathy (CAA) in a 200-patient global study.

Sarepta Halts Development of Vesleteplirsen for Duchenne Muscular Dystrophy

Sarepta Therapeutics discontinues the development of vesleteplirsen (SRP-5051), an exon-skipping antisense oligonucleotide (ASO) for Duchenne muscular dystrophy (DMD).
The decision was influenced by an updated risk-benefit analysis, FDA feedback, and the evolving therapeutic landscape for DMD.

FDA Halts Rapport Therapeutics' RAP-219 Trial for Diabetic Neuropathic Pain

The FDA has placed a clinical hold on Rapport Therapeutics' IND application for RAP-219, an investigational therapy for diabetic peripheral neuropathic pain (DPNP).
The agency is requesting additional information on the trial's protocol design, which Rapport Therapeutics believes it can address promptly.

Sarepta Therapeutics Halts Development of Vesleteplirsen for Duchenne Muscular Dystrophy

Sarepta Therapeutics discontinues vesleteplirsen (SRP-5051) development for Duchenne muscular dystrophy due to safety concerns and FDA feedback.
The decision follows a Phase 2 trial hold in 2022 due to hypomagnesemia and kidney function decrease observed in some patients.

Sarepta Discontinues Next-Gen DMD Drug Vesleteplirsen Amidst Regulatory Hurdles and Elevidys Success

Sarepta Therapeutics halts the development of vesleteplirsen, a next-generation exon 51-skipping therapy for Duchenne muscular dystrophy, after FDA feedback and internal analysis.
The decision follows concerns about persistent hypomagnesemia and the FDA's indication that the accelerated approval pathway is not viable for vesleteplirsen.

RAPT Therapeutics Halts Zelnecirnon Trials in Atopic Dermatitis and Asthma Following FDA Clinical Hold

RAPT Therapeutics has decided to close and unblind its Phase 2b trial of zelnecirnon in atopic dermatitis and its Phase 2a trial in asthma after an FDA clinical hold.
The clinical hold was initiated due to a serious adverse event of liver failure requiring a transplant in one patient during the atopic dermatitis trial.

FDA Places Clinical Hold on RAPT Therapeutics' Zelnecirnon Trials Following Liver Failure Case

The FDA has placed a clinical hold on RAPT Therapeutics' zelnecirnon (RPT193) trials for atopic dermatitis and asthma due to a serious adverse event.
The adverse event was a case of liver failure in a patient participating in the atopic dermatitis trial, with potential links to zelnecirnon under investigation.

Reference News

[1]
RAPT Therapeutics Halts Key Drug Program After FDA Safety Concerns, Liver Injury
stocktitan.net · Nov 11, 2024

RAPT Therapeutics terminates zelnecirnon program after FDA clinical holds due to severe liver injury in one patient, hal...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy