VYNE Therapeutics Provides Program Update on Oral BET inhibitor VYN202
VYNE Therapeutics (Nasdaq: VYNE) provided an update on its VYN202 program following an FDA clinical hold in April. The FDA has partially lifted the hold, allowing female patients to receive 0.25 mg and 0.5 mg doses in the Phase 1b psoriasis trial. The hold was initially placed due to testicular toxicity observed in dogs during non-clinical studies.
Preliminary data from the small trial (n=7) showed promising results: PASI score improvements ranged from 27% reduction after 1 week to 90% reduction at week 8. Significant reductions in key inflammatory biomarkers were observed, and a patient with psoriatic arthritis showed meaningful improvement in joint pain. The company will discontinue enrollment in the Phase 1b psoriasis study, extending its cash runway into Q4 2026.
VYNE Therapeutics (Nasdaq: VYNE) ha fornito un aggiornamento sul programma VYN202 dopo una sospensione clinica da parte della FDA avvenuta in aprile. La FDA ha parzialmente revocato la sospensione, permettendo alle pazienti di ricevere dosi di 0,25 mg e 0,5 mg nella sperimentazione di fase 1b sulla psoriasi. La sospensione era stata inizialmente applicata a causa di tossicità testicolare riscontrata nei cani durante studi non clinici.
I dati preliminari del piccolo studio (n=7) hanno mostrato risultati promettenti: i miglioramenti del punteggio PASI sono andati da una riduzione del 27% dopo 1 settimana fino al 90% alla settimana 8. Sono state osservate riduzioni significative nei principali biomarcatori infiammatori, e un paziente con artrite psoriasica ha riportato un miglioramento significativo del dolore articolare. L'azienda interromperà l'arruolamento nello studio di fase 1b sulla psoriasi, estendendo la disponibilità finanziaria fino al quarto trimestre 2026.
VYNE Therapeutics (Nasdaq: VYNE) proporcionó una actualización sobre su programa VYN202 tras una suspensión clínica por parte de la FDA en abril. La FDA ha levantado parcialmente la suspensión, permitiendo que pacientes femeninas reciban dosis de 0,25 mg y 0,5 mg en el ensayo de fase 1b para psoriasis. La suspensión se impuso inicialmente debido a toxicidad testicular observada en perros durante estudios no clínicos.
Los datos preliminares del pequeño ensayo (n=7) mostraron resultados prometedores: las mejoras en la puntuación PASI oscilaron entre una reducción del 27% tras 1 semana y una reducción del 90% en la semana 8. Se observaron reducciones significativas en biomarcadores inflamatorios clave, y una paciente con artritis psoriásica mostró una mejora significativa en el dolor articular. La compañía detendrá la inscripción en el estudio de fase 1b para psoriasis, extendiendo su liquidez hasta el cuarto trimestre de 2026.
VYNE Therapeutics (나스닥: VYNE)는 4월 FDA 임상 중단 이후 VYN202 프로그램에 대한 업데이트를 제공했습니다. FDA는 임상 중단을 부분적으로 해제하여 여성 환자들이 1b상 건선 임상시험에서 0.25 mg 및 0.5 mg 용량을 투여받을 수 있도록 허용했습니다. 중단은 비임상 연구 중 개에서 관찰된 고환 독성 때문에 처음에 시행되었습니다.
소규모 시험(n=7)의 예비 데이터는 유망한 결과를 보여주었습니다: PASI 점수 개선은 1주 후 27% 감소에서 8주차 90% 감소까지 나타났습니다. 주요 염증 바이오마커에서 유의미한 감소가 관찰되었고, 건선성 관절염 환자는 관절 통증에서 의미 있는 개선을 보였습니다. 회사는 1b상 건선 연구의 등록을 중단하고 현금 유동성을 2026년 4분기까지 연장할 예정입니다.
VYNE Therapeutics (Nasdaq : VYNE) a fourni une mise à jour sur son programme VYN202 suite à une suspension clinique par la FDA en avril. La FDA a partiellement levé cette suspension, permettant aux patientes de recevoir des doses de 0,25 mg et 0,5 mg dans l'essai de phase 1b sur le psoriasis. La suspension avait été initialement imposée en raison d'une toxicité testiculaire observée chez des chiens lors d'études non cliniques.
Les données préliminaires du petit essai (n=7) ont montré des résultats prometteurs : les améliorations du score PASI allaient d'une réduction de 27 % après 1 semaine à une réduction de 90 % à la semaine 8. Des réductions significatives des biomarqueurs inflammatoires clés ont été observées, et une patiente atteinte d'arthrite psoriasique a montré une amélioration notable des douleurs articulaires. La société cessera l'inclusion dans l'étude de phase 1b sur le psoriasis, prolongeant ainsi sa trésorerie jusqu'au quatrième trimestre 2026.
VYNE Therapeutics (Nasdaq: VYNE) gab ein Update zu seinem VYN202-Programm nach einem klinischen Halt durch die FDA im April bekannt. Die FDA hat den Halt teilweise aufgehoben, sodass weibliche Patientinnen im Phase-1b-Psoriasis-Studie 0,25 mg und 0,5 mg Dosierungen erhalten dürfen. Der Halt wurde ursprünglich aufgrund von testikulärer Toxizität bei Hunden in nicht-klinischen Studien verhängt.
Vorläufige Daten aus der kleinen Studie (n=7) zeigten vielversprechende Ergebnisse: Die PASI-Score-Verbesserungen reichten von einer 27%igen Reduktion nach 1 Woche bis zu einer 90%igen Reduktion in Woche 8. Es wurden signifikante Reduktionen wichtiger entzündlicher Biomarker beobachtet, und ein Patient mit psoriatischer Arthritis zeigte eine deutliche Verbesserung der Gelenkschmerzen. Das Unternehmen wird die Einschreibung in die Phase-1b-Psoriasis-Studie einstellen und seine finanzielle Reichweite bis ins 4. Quartal 2026 verlängern.
- Partial lifting of FDA clinical hold for female patients at 0.25 mg and 0.5 mg doses
- Strong efficacy signals with PASI score improvements up to 90% at week 8
- No serious adverse events or treatment discontinuations reported
- Significant reductions in inflammatory biomarkers (IL17A, IL17F, IL19, IL22)
- Extended cash runway into Q4 2026
- Clinical hold remains in effect for male patients due to testicular toxicity concerns
- 1 mg dose not included in revised protocol due to safety margin concerns
- Company discontinuing enrollment in Phase 1b psoriasis study
- Very small sample size (n=7) limits reliability of efficacy data
Insights
VYNE's VYN202 shows promising early data despite FDA restrictions, with strategic pivot extending cash runway while pursuing less competitive indications.
VYNE Therapeutics has provided a mixed update on its oral BET inhibitor program VYN202. The FDA has partially lifted the clinical hold for female patients at the 0.25mg and 0.5mg doses in their Phase 1b psoriasis trial, following testicular toxicity observed in dogs. The 1mg dose remains on hold due to lower safety margins, and male patients cannot be enrolled without additional toxicology data.
The company made two strategic decisions: unblinding preliminary data from the small cohort of enrolled patients (n=7) and discontinuing enrollment in the psoriasis study entirely. This decision extends their cash runway into Q4 2026, suggesting a strategic pivot rather than continuing to pursue psoriasis as the lead indication.
The preliminary efficacy data, while from an extremely limited sample size, shows encouraging signals with PASI score improvements ranging from ~27% reduction after one week to ~90% reduction at week 8. Reductions in key inflammatory cytokines (IL17A, IL17F, IL19, IL22) were observed in treated patients. Notably, one patient with comorbid psoriatic arthritis showed improvement in joint pain correlating with reduced C-reactive protein levels.
The safety profile appears manageable with no treatment-emergent serious adverse events (TESAEs), no discontinuations due to adverse events, and no observed cytopenias – which is significant as hematological toxicities have been a concern with other BET inhibitors.
This strategic redirection suggests VYNE may be positioning VYN202 for other immune-mediated conditions with higher unmet needs and potentially less competitive landscapes than psoriasis, where multiple effective biologics already exist. The company appears to be waiting for results from their lead candidate repibresib gel (for vitiligo) before announcing their revised development strategy for VYN202.
BRIDGEWATER, N.J., July 02, 2025 (GLOBE NEWSWIRE) -- VYNE Therapeutics Inc. (Nasdaq: VYNE) (“VYNE” or the “Company”), a clinical-stage biopharmaceutical company focused on developing differentiated therapies to treat chronic inflammatory and immune-mediated conditions with high unmet need, today provided a program update for VYN202 following the clinical hold issued by the U.S. Food and Drug Administration (FDA) in April for the Company’s Phase 1b clinical trial in the treatment of moderate-to-severe plaque psoriasis. VYN202 is an oral small molecule BD2-selective bromodomain and extra-terminal domain (BET) inhibitor that is being developed for the treatment of immune-mediated diseases.
VYN202 Program Update
In April, the FDA placed a clinical hold on the Company’s Phase 1b trial following an observation of testicular toxicity in dogs from a non-clinical toxicology study of VYN202. A No-Observed-Adverse-Effect Level (NOAEL) was established covering all clinical doses in females. The FDA has lifted the clinical hold for female patients on the 0.25 mg and 0.5 mg doses in this Phase 1b psoriasis trial. The 1 mg dose was not included at this time in the revised protocol submitted to the FDA due to its lower toxicological safety margin as compared to the 0.25 mg and 0.5 mg doses. Sufficient data from a 12-week non-clinical toxicology study of VYN202 in dogs would be required in order to resume the trial in male clinical subjects, and the design of this toxicology study has been agreed upon with the FDA.
Following the clinical hold, VYNE made the decision to unblind the clinical data from the subjects who were enrolled in the study (VYN202 treated: n=6 across 0.25 mg, 0.5 mg and 1 mg doses; Placebo treated: n=1), the results of which are described below.
The Company believes that the totality of the data from this study, together with promising results from multiple preclinical models, support the continued advancement of VYN202 into serious, immune-mediated diseases with limited effective treatment options. Based on this assessment, the Company will no longer enroll patients in the Phase 1b psoriasis study, extending the Company’s expected cash runway into the fourth quarter of 2026. VYNE expects to provide further updates on its plans for the VYN202 program following the release of top-line results from the ongoing Phase 2b study of its lead candidate repibresib gel (formerly VYN201), a pan-BD BET inhibitor, for the treatment of non-segmental vitiligo.
Preliminary Data from Phase 1b Trial
(n=7 enrolled: VYN202: n=6 across 0.25 mg, 0.5 mg and 1 mg doses; Placebo: n=1)
Please visit the Events and Presentations site on the VYNE Therapeutics corporate website for data presentation.
Safety and tolerability results
- No TESAEs or discontinuations due to a clinical TEAE
- No treatment interruptions due to a clinical TEAE
- No grades of thrombocytopenia, neutropenia or lymphocytopenia
Exploratory efficacy and serum biomarker results
- All subjects treated with VYN202 had an improvement in signs and symptoms of disease, including scalp psoriasis
- Improvement in PASI scores ranged from ~
27% reduction after 1 week of treatment to ~90% reduction at week 8.
- Improvement in PASI scores ranged from ~
- Improvements (reduction) in serum cytokine levels involved in the pathogenesis of plaque psoriasis were observed in subjects treated with VYN202 for greater than 1 week, including IL17A, IL17F, IL19 and IL22 ranging from -
17% to -83% . There was no change in these serum cytokines for the subject receiving placebo.
- Two subjects enrolled co-presented with psoriatic arthritis (n=1 treated with VYN202 0.5mg; n=1 treated with placebo)
- Subject treated with VYN202 0.5 mg reported a four-point improvement in joint pain NRS scale by week 2 which corresponded with a -
48% reduction in serum c-reactive protein level, a biomarker associated with psoriatic arthritis and other rheumatic diseases. - Subject treated with placebo had no improvement in joint pain NRS and no change in serum c-reactive protein levels.
- Subject treated with VYN202 0.5 mg reported a four-point improvement in joint pain NRS scale by week 2 which corresponded with a -
About Repibresib
Repibresib is a pan-bromodomain BET inhibitor designed to be locally administered as a “soft” drug to address diseases involving multiple, diverse inflammatory cell signaling pathways, while providing low systemic exposure. In addition to demonstrating clinical proof-of-concept in vitiligo, repibresib has produced consistent reductions in pro-inflammatory and disease-related biomarkers and improvements in disease severity in several preclinical models (using several different routes of administration).
About VYN202
VYN202 is an innovative, oral small molecule BET inhibitor that has potential class-leading selectivity and potency for BD2 vs. BD1. By maximizing BD2 selectivity, VYNE believes VYN202 has the potential to be a differentiated, more conveniently administered, non-biologic treatment option for both acute control and chronic management of immuno-inflammatory indications, in which the damaging effects of unrestricted inflammatory signaling activity are common.
About VYNE Therapeutics Inc.
VYNE is a clinical-stage biopharmaceutical company focused on developing differentiated therapies to treat chronic inflammatory and immune-mediated conditions with high unmet need. VYNE’s unique and proprietary BET inhibitors, which comprise its InhiBET™ platform, are designed to overcome limitations of early generation BET inhibitors by leveraging alternative routes of administration and enhanced selectivity.
For more information about VYNE Therapeutics Inc. or its product candidates, visit www.vynetherapeutics.com. VYNE may use its website to comply with its disclosure obligations under Regulation FD. Therefore, investors should monitor VYNE’s website in addition to following its press releases, filings with the U.S. Securities and Exchange Commission, public conference calls, and webcasts.
Investor Relations:
John Fraunces
LifeSci Advisors, LLC
917-355-2395
jfraunces@lifesciadvisors.com
Tyler Zeronda
VYNE Therapeutics Inc.
908-458-9106
Tyler.Zeronda@VYNEtx.com
Cautionary Statement Regarding Forward-Looking Statements
This release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the clinical development of VYN202, the potential benefits of VYN202, the timing of reporting results for the Phase 2b study of repibresib, VYNE’s expected cash runway, and other statements regarding the future expectations, plans and prospects of VYNE. All statements in this press release which are not historical facts are forward-looking statements. Any forward-looking statements are based on VYNE’s current knowledge and its present beliefs and expectations regarding possible future events and are subject to risks, uncertainties and assumptions that could cause actual results to differ materially and adversely from those set forth or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: VYNE’s ability to successfully develop its product candidates; the timing of commencement of future preclinical studies and clinical trials; VYNE’s ability to complete and receive favorable results from clinical trials of its product candidates; VYNE’s ability to obtain additional funding, either through equity or debt financing transactions or collaboration arrangements; and VYNE’s ability to comply with various regulations applicable to its business. For a discussion of other risks and uncertainties, and other important factors, any of which could cause VYNE’s actual results to differ from those contained in the forward-looking statements, see the section titled “Risk Factors” in VYNE’s Annual Report on Form 10-K for the year ended December 31, 2024 and Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, and VYNE’s other filings from time to time with the U.S. Securities and Exchange Commission. Although VYNE believes these forward-looking statements are reasonable, they speak only as of the date of this announcement and VYNE undertakes no obligation to update publicly such forward-looking statements to reflect subsequent events or circumstances, except as otherwise required by law. Given these risks and uncertainties, you should not rely upon forward-looking statements as predictions of future events.
Third-party products and company names mentioned herein may be the trademarks of their respective owners.
FAQ
What caused the FDA clinical hold on VYNE Therapeutics' VYN202 trial?
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