VYNE Therapeutics Announces Topline Results from Phase 2b Trial with Repibresib Gel in Nonsegmental Vitiligo
VYNE Therapeutics (Nasdaq: VYNE) reported disappointing topline results from its Phase 2b trial of Repibresib gel for nonsegmental vitiligo. The trial, involving 177 subjects, failed to meet its primary endpoint of F-VASI50 and key secondary endpoint of F-VASI75 at week 24 compared to vehicle.
Despite missing main endpoints, the 3% concentration showed nominally significant effects in secondary endpoints: -43.6% change from baseline in F-VASI score (vs. -25.6% vehicle) and -28.3% in T-VASI score (vs. -16.2% vehicle). Results were impacted by an unusually high vehicle effect and higher dropout rates in active arms (36.6% for 3% concentration vs. 10.6% for vehicle).
The company will terminate the trial's extension phase and seek an external partner for Repibresib's continued development. VYNE reported approximately $39.6 million in cash and equivalents as of June 30, 2025.
VYNE Therapeutics (Nasdaq: VYNE) ha riportato risultati deludenti dalla sua sperimentazione di fase 2b con il gel Repibresib per la vitiligine non segmentale. Lo studio, che ha coinvolto 177 soggetti, non ha raggiunto l'endpoint primario F-VASI50 n茅 il principale endpoint secondario F-VASI75 alla settimana 24 rispetto al veicolo.
Nonostante il mancato raggiungimento degli endpoint principali, la concentrazione al 3% ha mostrato effetti nominalmente significativi negli endpoint secondari: una riduzione del 43,6% rispetto al basale nel punteggio F-VASI (contro il 25,6% del veicolo) e del 28,3% nel punteggio T-VASI (contro il 16,2% del veicolo). I risultati sono stati influenzati da un effetto veicolo insolitamente elevato e da tassi di abbandono pi霉 alti nei gruppi attivi (36,6% per la concentrazione al 3% contro il 10,6% per il veicolo).
L'azienda interromper脿 la fase di estensione dello studio e cercher脿 un partner esterno per lo sviluppo futuro di Repibresib. Al 30 giugno 2025, VYNE ha riportato circa 39,6 milioni di dollari in liquidit脿 e equivalenti.
VYNE Therapeutics (Nasdaq: VYNE) inform贸 resultados decepcionantes en la l铆nea principal de su ensayo de fase 2b con gel de Repibresib para vitiligo no segmentario. El ensayo, que incluy贸 a 177 sujetos, no logr贸 alcanzar el endpoint primario F-VASI50 ni el endpoint secundario clave F-VASI75 en la semana 24 en comparaci贸n con el veh铆culo.
A pesar de no alcanzar los endpoints principales, la concentraci贸n al 3% mostr贸 efectos nominalmente significativos en los endpoints secundarios: un cambio del -43.6% desde la l铆nea base en la puntuaci贸n F-VASI (vs. -25.6% con veh铆culo) y -28.3% en la puntuaci贸n T-VASI (vs. -16.2% con veh铆culo). Los resultados se vieron afectados por un efecto veh铆culo inusualmente alto y mayores tasas de abandono en los grupos activos (36.6% para la concentraci贸n al 3% vs. 10.6% para veh铆culo).
La compa帽铆a terminar谩 la fase de extensi贸n del ensayo y buscar谩 un socio externo para el desarrollo continuado de Repibresib. VYNE report贸 aproximadamente 39.6 millones de d贸lares en efectivo y equivalentes al 30 de junio de 2025.
VYNE Therapeutics (雮橃姢雼�: VYNE)電� 牍勲秳鞝堨劚 氚彪皹歃� 旃橂鞖� Repibresib 鞝れ潣 2b靸� 鞛勳儊鞁滍棙鞐愳劀 鞁る鞀る煬鞖� 欤检殧 瓴瓣臣毳� 氤搓碃頄堨姷雼堧嫟. 177氇呾潣 頂柬棙鞛愱皜 彀胳棳頃� 鞚措矆 鞁滍棙鞚� 24欤检皑鞐� 彀焿 雽�牍� 1彀� 韽夑皜氤�靾橃澑 F-VASI50瓿� 欤检殧 2彀� 韽夑皜氤�靾橃澑 F-VASI75毳� 於╈”頃橃 氇豁枅鞀惦媹雼�.
欤检殧 韽夑皜氤�靾橂ゼ 雼劚頃橃 氇豁枅鞚岇棎霃� 攵堦惮頃橁碃, 3% 雴嶋弰電� 2彀� 韽夑皜氤�靾橃棎靹� 氇呺靸� 鞙犾潣氙疙暅 須臣毳� 氤挫榾鞀惦媹雼�: F-VASI 鞝愳垬電� 旮办靹� 雽�牍� -43.6% 氤�頇�(彀焿 雽�牍� -25.6%), T-VASI 鞝愳垬電� -28.3%(彀焿 雽�牍� -16.2%) 臧愳唽頄堨姷雼堧嫟. 瓴瓣臣電� 牍勳爼靸侅爜鞙茧 雴掛潃 彀焿 須臣鞕� 頇滌劚甑办潣 雴掛潃 欷戨弰 韮堧澖毳�(3% 雴嶋弰 36.6% vs. 彀焿 10.6%)鞐� 鞚橅暣 鞓來枼鞚� 氚涭晿鞀惦媹雼�.
須岇偓電� 鞁滍棙 鞐办灔 雼硠毳� 膦呺頃橁碃 Repibresib鞚� 歆�靻嶌爜鞚� 臧滊皽鞚� 鞙勴暣 鞕鸽秬 韺岉姼雱堧ゼ 彀眷潉 鞓堨爼鞛呺媹雼�. VYNE電� 2025雲� 6鞗� 30鞚� 旮办 鞎� 3,960毵� 雼煬鞚� 順勱笀 氚� 順勱笀靹� 鞛愳偘鞚� 氤搓碃頄堨姷雼堧嫟.
VYNE Therapeutics (Nasdaq : VYNE) a annonc茅 des r茅sultats d茅cevants pour son essai de phase 2b du gel Repibresib dans le vitiligo non segmentaire. L'茅tude, impliquant 177 sujets, n'a pas atteint son crit猫re d'茅valuation principal F-VASI50 ni le crit猫re secondaire cl茅 F-VASI75 脿 la semaine 24 par rapport au v茅hicule.
Malgr茅 l'茅chec des crit猫res principaux, la concentration 脿 3 % a montr茅 des effets nominalement significatifs sur les crit猫res secondaires : une r茅duction de 43,6 % par rapport au d茅part du score F-VASI (contre 25,6 % pour le v茅hicule) et de 28,3 % pour le score T-VASI (contre 16,2 % pour le v茅hicule). Les r茅sultats ont 茅t茅 affect茅s par un effet v茅hicule anormalement 茅lev茅 et des taux d'abandon plus 茅lev茅s dans les groupes actifs (36,6 % pour la concentration 脿 3 % contre 10,6 % pour le v茅hicule).
L'entreprise mettra fin 脿 la phase d'extension de l'essai et cherchera un partenaire externe pour poursuivre le d茅veloppement de Repibresib. VYNE a d茅clar茅 disposer d'environ 39,6 millions de dollars en liquidit茅s et 茅quivalents au 30 juin 2025.
VYNE Therapeutics (Nasdaq: VYNE) meldete entt盲uschende Ergebnisse aus der Phase-2b-Studie mit Repibresib-Gel bei nicht-segmentalem Vitiligo. Die Studie mit 177 Teilnehmern verfehlte den prim盲ren Endpunkt F-VASI50 sowie den wichtigsten sekund盲ren Endpunkt F-VASI75 nach 24 Wochen im Vergleich zum Vehikel.
Trotz des Verfehlens der Hauptendpunkte zeigte die 3%-Konzentration nominal signifikante Effekte bei sekund盲ren Endpunkten: eine Ver盲nderung des F-VASI-Scores von -43,6% gegen眉ber dem Ausgangswert (vs. -25,6% Vehikel) und -28,3% beim T-VASI-Score (vs. -16,2% Vehikel). Die Ergebnisse wurden durch einen ungew枚hnlich hohen Vehikel-Effekt und h枚here Abbruchraten in den aktiven Gruppen beeinflusst (36,6% bei 3% Konzentration vs. 10,6% beim Vehikel).
Das Unternehmen wird die Verl盲ngerungsphase der Studie abbrechen und einen externen Partner f眉r die weitere Entwicklung von Repibresib suchen. VYNE berichtete 眉ber ca. 39,6 Millionen US-Dollar an liquiden Mitteln und 脛quivalenten zum 30. Juni 2025.
- None.
- Failed to meet primary endpoint (F-VASI50) and key secondary endpoint (F-VASI75)
- High discontinuation rate in active arms (36.6% for 3% concentration vs 10.6% for vehicle)
- Higher rate of treatment emergent adverse events compared to vehicle
- Company forced to terminate trial extension phase
- Need to seek external partner for continued development
Insights
VYNE's vitiligo treatment failed primary endpoints with unexpectedly high vehicle response; company terminating extension trial despite some secondary endpoint benefits.
VYNE Therapeutics' Phase 2b trial evaluating Repibresib gel for nonsegmental vitiligo has failed to meet its primary endpoint of F-VASI50 (facial repigmentation of at least 50%) and the key secondary endpoint of F-VASI75. The highest concentration (3%) achieved 19.5% F-VASI50 response versus 23.4% for vehicle (placebo), falling short of statistical significance.
Despite the primary endpoint miss, the 3% concentration did show nominally statistically significant improvements in two other measures: percent change from baseline in F-VASI (-43.6% vs -25.6% for vehicle, p=0.0020) and T-VASI (-28.3% vs -16.2%, p=0.0436). These results suggest some biological activity, but the overall efficacy profile is compromised.
Two critical factors undermined the trial results: an unusually high vehicle (placebo) effect and disproportionate dropout rates between active treatment (26.1-36.6%) and vehicle (10.6%). This imbalance likely skewed the intent-to-treat analysis, as patients who discontinued were counted as non-responders.
The safety profile revealed higher rates of application site adverse events with Repibresib compared to vehicle, with application site pain being most common (3% concentration: 14.0% vs vehicle: 3.8%). Eight subjects on active treatment discontinued due to adverse events versus none on vehicle, further contributing to the high dropout rate.
Based on these disappointing results, VYNE is terminating the extension phase of the trial and shifting strategy to seek an external development partner for Repibresib while refocusing on their broader InhiBET鈩� platform. With
Trial Did Not Meet Primary Endpoint or Key Secondary Endpoint of F-VASI50 and F-VASI75
Nominally Statistically Significant Effects Observed in Key Secondary and Exploratory Endpoints of Change from Baseline in F-VASI and T-VASI at
Company Will Terminate Extension Phase of Trial and Seek External Partner for Continued Development of Repibresib
BRIDGEWATER, N.J., July 30, 2025 (GLOBE NEWSWIRE) -- VYNE Therapeutics Inc. (Nasdaq: VYNE) (鈥淰YNE鈥� or the 鈥淐ompany鈥�), a clinical-stage biopharmaceutical company focused on developing differentiated therapies to treat chronic inflammatory and immune-mediated conditions with high unmet need, today announced topline results from its Phase 2b trial evaluating Repibresib gel in nonsegmental vitiligo.
The trial, which evaluated 177 subjects, did not meet its primary endpoint of the proportion of subjects achieving an improvement in Facial Vitiligo Area Scoring Index of at least
The Phase 2b trial was a randomized double-blind, vehicle-controlled, multi-center trial that evaluated the safety and efficacy of Repibresib dosed once daily (QD) at
鈥淲e are disappointed with the results of our Phase 2b trial, which were impacted by an unexpectedly high treatment effect in the vehicle arm and a high discontinuation rate in the active arms,鈥� said David Domzalski, President and Chief Executive Officer of VYNE. 鈥淎lthough we missed our F-VASI50 and F-VASI75 endpoints, we did see a meaningful reduction in the percent change from baseline in both F-VASI and T-VASI for our highest dose and are conducting a thorough evaluation of the full dataset to analyze any other data that may inform the results and our strategic next steps. Vitiligo remains an area of significant unmet need and we are grateful to the patients, investigators, and clinical staff who participated in the trial. Despite this outcome, we remain confident in the potential of our InhiBET鈩⑻鼴ET inhibitor platform as a promising and innovative mechanistic approach for the treatment of a broad range of serious immune-mediated diseases. We intend to seek a development and commercialization partner for Repibresib and we will provide an update on our plans, including those for our oral BET inhibitor VYN202, in the coming weeks.鈥�
While the Company has not finalized its full financial results for the quarter ended June 30, 2025, the Company expects to report that it had cash, cash equivalents and investments of approximately
Primary endpoint: proportion of subjects achieving F-VASI50 at Week 24 versus vehicle in modified intent to treat population (mITT)
| Endpoint at Week 24 | Repibresib | Repibresib | Repibresib | Vehicle |
| (N=41) | (N=43) | (N=46) | (N=47) | |
| Proportion of Subjects Achieving F-VASI50 | ||||
| P-Value | 0.1245 | 0.6497 | 0.9718 | 听 |
Key Secondary and Exploratory Endpoints
Proportion of subjects achieving F-VASI75 at Week 24 (mITT)
| Endpoint at Week 24 | Repibresib | Repibresib | Repibresib | Vehicle |
| (N=41) | (N=43) | (N=46) | (N=47) | |
| Proportion of Subjects Achieving F-VASI75 | ||||
| P-Value | 0.1468 | 0.4096 | 0.2946 | 听 |
Percent Change from Baseline (% CFB) in F-VASI at Week 24 (mITT)
| Endpoint at Week 24 | Repibresib | Repibresib | Repibresib | Vehicle |
| (N=41) | (N=43) | (N=46) | (N=47) | |
| Mean Percent Change from Baseline in F-VASI, % (SD) | -43.6 (5.7) | -25.7 (5.7) | -30.2 (5.2) | -25.6 (4.9) |
| P-Value | 0.0020 | 0.9892 | 0.4002 | 听 |
Percent Change from Baseline (% CFB) in T-VASI at Week 24 (mITT)
| Endpoint at Week 24 | Repibresib | Repibresib | Repibresib | Vehicle |
| (N=41) | (N=43) | (N=46) | (N=47) | |
| Mean Percent Change from Baseline in T-VASI, % (SD) | -28.3 (6.1) | -15.2 (6.1) | -16.4 (5.2) | -16.2 (4.9) |
| P-Value | 0.0436 | 0.8757 | 0.9670 | 听 |
Safety and Tolerability
There was a higher rate of treatment emergent adverse events (TEAE) for subjects receiving Repibresib gel compared to vehicle. The most common TEAEs (>
About Repibresib
Repibresib is a pan-bromodomain BET inhibitor designed to be locally administered as a 鈥渟oft鈥� drug to address diseases involving multiple, diverse inflammatory cell signaling pathways, while providing low systemic exposure. 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鈥檚 unique and proprietary BET inhibitors, which comprise its InhiBET鈩� 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鈥檚 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
[email protected]
Tyler Zeronda
VYNE Therapeutics Inc.
908-458-9106
[email protected]
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 VYNE鈥檚 product candidates 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, including the Company鈥檚 intention to seek a development and commercialization partner for repibresib, timing of an update on the Company鈥檚 plans for repibresib and the expectations around its cash position as of June 30, 2025. The Company鈥檚 cash estimate is preliminary and is subject to change pending the actual results of, and completion of, the Company鈥檚 condensed consolidated financial statements for the quarter ended June 30, 2025. Additional information and disclosures would be required for a more complete understanding of the Company鈥檚 financial position and results of operations as of June 30, 2025. The Company鈥檚 independent registered public accounting firm has not reviewed or performed any procedures with respect to this preliminary information and, accordingly, does not express an opinion or any other form of assurance about them. Any forward-looking statements in this release are based on VYNE鈥檚 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鈥檚 ability to successfully develop its product candidates; the timing of commencement of future preclinical studies and clinical trials; VYNE鈥檚 ability to complete and receive favorable results from clinical trials of its product candidates; VYNE鈥檚 ability to find a partner for repibresib; VYNE鈥檚 ability to obtain additional funding, either through equity or debt financing transactions or collaboration arrangements; and VYNE鈥檚 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鈥檚 actual results to differ from those contained in the forward-looking statements, see the section titled 鈥淩isk Factors鈥� in VYNE鈥檚 Annual Report on Form 10-K for the year ended December听31, 2024 and Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, and VYNE鈥檚 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.
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FAQ
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