VYNE Therapeutics Announces FDA Clearance of IND Application for VYN202, a Novel BD2-Selective BET Inhibitor

Rhea-AI Summary

VYNE Therapeutics announced the FDA clearance of its Investigational New Drug application for VYN202, a novel BD2-selective BET inhibitor. In preclinical studies, VYN202 showed consistent reductions in biomarkers and disease severity across various inflammatory and fibrotic models. The Phase 1a trial in healthy volunteers is set to begin this quarter, with top line results expected in the second half of the year.

Positive

• VYN202 achieved consistent reductions in pro-inflammatory and disease-related biomarkers in preclinical studies.

• The FDA clearance of the IND application is a major step forward for VYNE Therapeutics.

• The Phase 1a trial for VYN202 in healthy volunteers is anticipated to start this quarter.

Negative

• Results from the Phase 1a trial are not expected until the second half of the year.

• The Phase 1b trials in subjects with psoriasis and arthritis are not set to begin until 2025.

Insights

Medical Research Analyst

VYNE Therapeutics' initiative to progress VYN202, an oral BD2-selective BET inhibitor, into human trials is a pivotal move in the drug development pipeline. BET inhibitors are a class of compounds targeting bromodomain and extraterminal domain proteins, which play a key role in regulating gene expression, especially in inflammatory processes. The transition from preclinical to Phase 1 clinical trials is significant, as it marks the first test of the drug's safety and pharmacokinetics in humans. Safety, tolerability and optimal dosing regimens will be scrutinized. For investors, the successful completion of the Phase 1a trial could enhance confidence in the compound's potential, thus impacting VYNE's stock positively. However, the associated risks of clinical trials, coupled with the fact that the vast majority of drugs fail to make it past clinical trials, should be taken into account. The therapeutic focus on autoimmune diseases such as psoriasis and rheumatoid arthritis taps into a multi-billion dollar market, but competition is stiff and VYNE's commercial success will hinge not only on safety and efficacy but also on differentiating VYN202 from existing treatments.

Financial Analyst

The IND clearance for VYN202 and the intended commencement of Phase 1a trials by VYNE Therapeutics holds potential implications on the company's financial health. The move is indicative of VYNE's progression along the drug development pathway, with associated costs likely to escalate as clinical trials advance. Investors should note that R&D expenses will be a focal point in future earnings reports and short-term financial strain is typical for clinical-stage biopharmaceutical companies. The promise of VYN202 as a treatment for autoimmune diseases like psoriasis and rheumatoid arthritis, conditions with substantial patient populations, could signal a substantial future revenue stream, but this potential is balanced by the high risk inherent to pharmaceutical R&D. The stock's performance in the short term could fluctuate with trial outcomes and investor sentiment, while long-term performance is contingent on regulatory approvals and market penetration capabilities. It's imperative for stakeholders to monitor the company's burn rate and fundraising activities, as these will be critical for sustaining operations through the lengthy and costly trial process.

Market Research Analyst

The market for autoimmune disease treatments is crowded but continues to grow, driven by an increasing prevalence of these diseases and a deeper understanding of their underlying mechanisms. VYN202's specificity as a BD2-selective BET inhibitor positions it within a niche, target-specific approach that may offer advantages over less selective therapies. The successful clearance of the IND application by the FDA allows VYNE Therapeutics to gain a foothold in the market, but the true test will come with the demonstration of clinical efficacy and safety in upcoming trials. For investors, the focus should be on the potential market size for VYN202 and the competitive landscape. If successful, VYN202 could capture a segment of the market currently underserved by existing treatments. The attention garnered by advancing to human trials could also attract partnership opportunities or acquisition interest from larger pharmaceutical companies looking to expand their pipelines in the immuno-inflammatory space. Nevertheless, market entry is still several years away and investor optimism should be tempered by the recognition that many investigational drugs do not ultimately achieve commercialization.

In preclinical studies, VYN202 achieved consistent reductions in pro-inflammatory and disease-related biomarkers and improvements in disease severity across a variety of inflammatory and fibrotic models

Phase 1a SAD/MAD trial expected to start this quarter

BRIDGEWATER, N.J., May 06, 2024 (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 the clearance of its Investigational New Drug application (“IND”) by the U.S. Food and Drug Administration for VYN202, an oral BD2-selective BET inhibitor. VYNE plans to initiate a first-in-human Phase 1a single ascending dose/multiple ascending dose (“SAD/MAD”) trial in healthy volunteers this quarter and expects to report top line results from the SAD/MAD trial in the second half of this year.

“VYN202 is a highly selective and potent orally administered BET inhibitor that we believe has significant potential as a treatment option for autoimmune diseases. Clearance of the IND for VYN202 marks a major step forward in this effort,” said David Domzalski, President and CEO of VYNE. “We look forward to initiating the Phase 1a trial in the coming weeks to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of VYN202.”

The VYN202 Phase 1a trial is a double-blind, placebo-controlled study in healthy volunteers and consists of SAD and MAD components. The study is expected to enroll approximately 64 healthy adult subjects into five SAD and three MAD cohorts to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of VYN202. If the Phase 1a portion of the trial is successfully completed, VYNE plans to initiate Phase 1b trials in subjects with moderate-to-severe plaque psoriasis and moderate-to-severe adult-onset rheumatoid arthritis, with top line results anticipated in the second half of 2025.

About VYN202
VYN202 is an innovative, oral small molecule BD2-selective BET inhibitor that has been designed to achieve class-leading selectivity (BD2 vs. BD1), maximum potency versus BD2 and optimal oral bioavailability. By maximizing BD2 selectivity, VYNE believes VYN202 has the potential to be a 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 is common. VYN202 is structurally distinct from VYNE’s pan-BET inhibitor (VYN201) and covered by distinct PCT and provisional composition of matter patent applications directed to new chemical entities and their uses.

About BET Inhibitors
BET proteins play a key role in regulating gene transcription via epigenetic interactions (“reading”), and recent research has identified 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 inhibitors, which comprise its InhiBET™ platform, include a locally administered pan-BD BET inhibitor (VYN201) and an orally available BD2-selective BET inhibitor (VYN202) that were licensed from Tay Therapeutics Limited. 

For more information about VYNE Therapeutics Inc. and 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 related to future clinical trials for VYN202 and timing for reporting top line results from those trials. 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, 2023, 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.

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

FAQ

<p>What is VYN202?</p>

VYN202 is an oral BD2-selective BET inhibitor developed by VYNE Therapeutics.

<p>When will the Phase 1a trial for VYN202 start?</p>

The Phase 1a trial in healthy volunteers is expected to commence this quarter.

<p>What are the top line results anticipated for the Phase 1a trial?</p>

The top line results for the Phase 1a trial are projected to be reported in the second half of this year.

<p>What are the Phase 1b trials for VYN202?</p>

The Phase 1b trials will focus on subjects with moderate-to-severe plaque psoriasis and moderate-to-severe adult-onset rheumatoid arthritis, with top line results expected in the second half of 2025.