VYNE Therapeutics Announces Positive Preclinical Data for Inhaled Formulation of VYN201 in an In Vivo Model of Idiopathic Pulmonary Fibrosis

• VYN201 at the 0.5 mg/ml and 1 mg/ml doses demonstrated statistically significant reductions in lung fibrosis and hydroxyproline levels compared to placebo
• Reduction in lung fibrosis from VYN201 treatment resulted in significant improvements in blood oxygen saturation and functional lung volume
• Data further supports potential broad utility of VYN201 as a potent locally-administered anti-inflammatory and anti-fibrotic molecule

BRIDGEWATER, N.J., April 19, 2023 (GLOBE NEWSWIRE) — VYNE Therapeutics Inc. (Nasdaq: VYNE) (“VYNE” or the “Company”), a clinical-stage biopharmaceutical company developing proprietary, innovative and differentiated therapies for the treatment of immuno-inflammatory conditions, today announced new preclinical data showing the positive effect of its novel pan-BET inhibitor, VYN201, in a preclinical model of idiopathic pulmonary fibrosis (“IPF”).

“The data from this well-validated preclinical model of IPF clearly demonstrates VYN201’s potential to deliver a potent anti-inflammatory and anti-fibrotic response,” said David Domzalski, President and Chief Executive Officer of VYNE. “These data in IPF support our thesis that VYN201 has potential utility as a locally-administered therapy across a variety of immuno-inflammatory indications and further underscores the potential value of our InhiBET^TM BET inhibitor platform.”

Bleomycin-Induced Mouse Model

IPF is a chronic, life-threatening, fibrosing lung disease with few treatment options. Patients experience debilitating symptoms, including shortness of breath and difficulty performing daily activities. The current standard of care treatment options for IPF have been shown to have only a modest impact on slowing the progression of the disease and have been associated with significant side effects.

In this well-validated preclinical model for IPF, lung fibrosis was induced in mice using a single intratracheal dose of bleomycin. Fibrosis was left to develop for seven days, and thoracic tomography images were obtained to stage fibrotic development. Animals were assigned to six treatment groups: untreated and unstimulated control, placebo, and one of four doses of VYN201 (0.1, 0.2, 0.5, and 1.0 mg/ml) (N=6/group). Each treatment group was dosed intratracheally every other day for 14 days. Changes in blood oxygen saturation, Ashcroft scoring (a standardized numerical scale used to quantify the extent of lung fibrosis in histological samples), lung hydroxyproline (a tissue biomarker for fibrosis), and volumetric lung function were assessed.

Key findings:

• Ashcroft Scoring and Hydroxyproline Levels: VYN201 at 0.5 mg/ml and 1 mg/ml demonstrated statistically significant reductions in Ashcroft scores (a measurement of lung fibrosis) and levels of the tissue fibrosis biomarker, hydroxyproline, compared to the placebo control group at day 21.
  • Mean control-adjusted lung fibrosis scores for VYN201 1 mg/ml were 65.8% lower compared to the placebo control group at day 21.
• Blood Oxygen Saturation: VYN201 demonstrated a dose-dependent improvement in blood oxygen saturation.
  • Mean blood oxygen saturation for the VYN201 1 mg/ml group was 92.4% at day 21, an 8.8% improvement compared to the placebo group (83.6%). Mean blood oxygen saturation for the untreated and unstimulated control group was 95.2%.
• Volumetric Lung Function: Thoracic tomography revealed that VYN201 treatment groups demonstrated a dose-dependent improvement in functional lung volume compared to the placebo control group. These data correlate with an increase in blood oxygen saturation and reduction in Ashcroft fibrosis scores.
  • Treatment with VYN201 1 mg/ml resulted in a 51.8% mean improvement in functional lung volume compared to animals receiving placebo treatment.

About VYN201

VYN201 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. To date, VYN201 has produced consistent reductions in pro-inflammatory and disease-related biomarkers and improvements in disease severity, and demonstrated local activity in several preclinical models (using several different routes of administration). The Company believes that these data suggest the potential broad utility for VYN201 across multiple routes of administration. 

VYNE has completed a Phase 1a study of a topical formulation for VYN201 in healthy volunteers and is now evaluating the topical formulation in a Phase 1b study in patients with non-segmental vitiligo. The Phase 1a study showed that VYN201, administered topically, performed as expected. In particular, VYN201 had minimal systemic exposure and none of the common adverse events commonly associated with systemically administered pan-BD BET inhibitors, such as thrombocytopenia. VYNE expects to report topline results from the Phase 1b study in mid-2023.

About BET Inhibitors

BET proteins play a key role in regulating gene transcription via epigenetic interactions (“reading”), and recent research has determined a key role for these proteins in regulating B cell and T cell activation and subsequent inflammatory processes. As epigenetic readers, BET proteins regulate the recruitment of transcriptional factors that are key to the production of several pro-inflammatory cytokines. BET inhibitors have the potential to treat a range of immuno-inflammatory and fibrotic diseases by blocking pro-inflammatory cytokine transcription with additional potential in myeloproliferative neoplastic disorders.

About VYNE Therapeutics Inc.

VYNE’s mission is to improve the lives of patients by developing proprietary, innovative, and differentiated therapies for the treatment of immuno-inflammatory conditions. The Company’s unique and proprietary bromodomain & extra-terminal (BET) domain platform includes lead programs, VYN201 (locally administered pan-BD BETi) and VYN202 (orally available BD2 selective-BETi), and access to a library of small molecule BET inhibitors for the potential treatment of immuno-inflammatory conditions licensed from Tay Therapeutics Limited.

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.

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 VYNE’s BET inhibitor platform, 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 non-clinical studies and clinical trials; VYNE’s ability to enroll patients and successfully progress, complete, and receive favorable results in, clinical trials for its product candidates; VYNE’s ability to exercise its exclusive option with respect to an oral BET inhibitor candidate pursuant to the terms of the option agreement with Tay Therapeutics Limited; VYNE’s intentions and its ability to obtain additional funding, either through equity or debt financing transactions or collaboration arrangements; disruptions related to COVID-19 or another pandemic, epidemic or outbreak of a contagious disease, on the ability of VYNE’s suppliers to manufacture and provide materials for VYNE’s product candidates, initiating and retaining patients in clinical trials, operating results, liquidity and financial condition; the regulatory approval process for VYNE’s product candidates, including any delay or failure in obtaining requisite approvals; the potential market size of treatments for any diseases and market adoption of products, if approved or cleared for commercial use, by physicians and patients; developments and projections relating to competitors and the pharmaceuticals industry, including competing drugs and therapies; the timing or likelihood of regulatory filings and approvals or clearances for product candidates; VYNE’s ability to comply with various regulations applicable to its business, including Nasdaq continued listing rules; VYNE’s ability to create intellectual property and the scope of protection it is able to establish and maintain for intellectual property rights covering its product candidates, including the projected terms of patent protection; risks that any of VYNE’s patents may be held to be narrowed, invalid or unenforceable or one or more of VYNE’s patent applications may not be granted and potential competitors may also seek to design around VYNE’s granted patents or patent applications; the timing, costs or results of litigation, including litigation to protect its intellectual property; VYNE’s ability to successfully challenge intellectual property claimed by others; estimates of VYNE’s expenses, capital requirements, its needs for additional financing and its ability to obtain additional capital on acceptable terms or at all; VYNE’s ability to attract and retain key scientific or management personnel; VYNE’s defense of any litigation that may be initiated against it; VYNE’s expectations regarding licensing, business transactions and strategic operations; VYNE’s future financial performance and liquidity; and volatility in VYNE’s stock price may result in rapid and substantial increases or decreases in the stock price that may or may not be related to the company’s operating performance or prospects. 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, 2022, as well as discussions of potential risks, uncertainties, and other important factors in VYNE’s subsequent filings 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.

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