VYNE Therapeutics Inc

Previous wording

Our lead program is VYN201, a locally administered pan-BD BET inhibitor designed as a “soft” drug to address diseases involving multiple, diverse inflammatory cell signaling pathways while providing low systemic exposure. In preclinical testing, VYN201 produced consistent reductions in pro-inflammatory and disease-related biomarkers and improvements in disease severity across a variety of inflammatory and fibrotic models. In November 2022, we initiated a Phase 1 clinical trial evaluating a topical formulation of VYN201 for the treatment of nonsegmental vitiligo. In the first quarter of 2023, we announced positive preliminary safety and tolerability, pharmacokinetic and hematology data from the Phase 1a portion of the trial. The first nonsegmental vitiligo patient was dosed in the Phase 1b portion of the trial in January 2023, and on October 30, 2023, we announced positive data from the Phase 1b trial, in which significant clinical improvement in F-VASI was observed in the 1% and 2% dose cohorts after 16 weeks of treatment. We initiated a Phase 2b trial of VYN201 in June 2024. The Phase 2b trial in subjects with nonsegmental vitiligo is a randomized, double-blind, vehicle-controlled trial evaluating the efficacy, safety and pharmacokinetics of once-daily VYN201 gel in three dose cohorts (1%, 2% and 3% concentrations) compared to vehicle for 24 weeks, followed by a 28-week active treatment extension. We expect to enroll approximately 40 patients in each arm and to report top-line results from the 24-week double-blind portion of the trial in mid-2025.

Current wording

Our lead program is VYN201, a locally administered pan-BD BET inhibitor designed as a “soft” drug to address diseases involving multiple, diverse inflammatory cell signaling pathways while providing low systemic exposure. In preclinical testing, VYN201 produced consistent reductions in pro-inflammatory and disease-related biomarkers and improvements in disease severity across a variety of inflammatory and fibrotic models. In November 2022, we initiated a Phase 1 clinical trial evaluating a topical formulation of VYN201 for the treatment of nonsegmental vitiligo. In the first quarter of 2023, we announced positive preliminary safety and tolerability, pharmacokinetic and hematology data from the Phase 1a portion of the trial. The first nonsegmental vitiligo patient was dosed in the Phase 1b portion of the trial in January 2023, and on October 30, 2023, we announced positive data from the Phase 1b trial, in which significant clinical improvement in F-VASI was observed in the 1% and 2% dose cohorts after 16 weeks of treatment. We initiated a Phase 2b trial of VYN201 in June 2024. The Phase 2b trial in subjects with nonsegmental vitiligo is a randomized, double-blind, vehicle-controlled trial evaluating the efficacy, safety and pharmacokinetics of once-daily VYN201 gel in three dose cohorts (1%, 2% and 3% concentrations) compared to vehicle for 24 weeks, followed by a 28-week active treatment extension. We expect to enroll approximately 40 to 50 patients in each arm and to report top-line results from the 24-week double-blind portion of the trial in mid-2025.

Previous wording

Our second program is VYN202, an oral small molecule BD2-selective BET inhibitor. VYN202 has been designed to achieve potential class-leading selectivity and potency (BD2 vs. BD1). By maximizing BD2 selectivity, we believe 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 are common. We initiated a Phase 1a single ascending dose/multiple ascending dose ("SAD/MAD") trial of VYN202 in June 2024 and expect top-line results from the SAD and MAD portions in the third quarter and fourth quarter of 2024, respectively. If the Phase 1a trial is successfully completed, we plan to initiate Phase 1b trials in adult 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.

Current wording

Our second program is VYN202, an oral small molecule BD2-selective BET inhibitor. VYN202 has been designed to achieve potential class-leading selectivity and potency (BD2 vs. BD1). By maximizing BD2 selectivity, we believe 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 are common. We initiated a Phase 1a single ascending dose/multiple ascending dose ("SAD/MAD") trial of VYN202 in June 2024 and announced positive data from the SAD portion of the trial in September 2024. We expect to report top-line results from the MAD portion of the trial in the fourth quarter of 2024. If the Phase 1a trial is successfully completed, we plan to initiate Phase 1b trials in adult 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.

Previous wording

We have incurred net losses since our inception. Except from the period from 2020 to 2022, when we conducted commercial operations, our business activities have been primarily limited to developing product candidates, raising capital and performing research and development activities. As of June 30, 2024, we had an accumulated deficit of $707.0 million. We recorded net losses of $15.7 million for each of the six months ended June 30, 2024 and 2023.

Current wording

We have incurred net losses since our inception. Except from the period from 2020 to 2022, when we conducted commercial operations, our business activities have been primarily limited to developing product candidates, raising capital and performing research and development activities. As of September 30, 2024, we had an accumulated deficit of $719.1 million. We recorded net losses of $27.8 million and $22.3 million for the nine months ended September 30, 2024 and 2023, respectively.

Previous wording

On April 28, 2023, we exercised the Oral Option and entered into a license agreement (the "VYN202 License Agreement" and together with the VYN201 License Agreement, the "Tay License Agreements") with Tay granting us a worldwide, exclusive license that is sublicensable through multiple tiers to exploit certain of Tay’s Oral BETi Compounds in all fields. We have the sole responsibility for development, regulatory, marketing and commercialization activities to be conducted for the licensed products at our sole cost and discretion, and shall use commercially reasonable efforts to develop and, if approved, commercialize such products. We may sublicense our rights to a third party without Tay’s consent. Pursuant to the VYN202 License Agreement, a joint development committee consisting of one representative from each party reviews the progress of the development plan for the licensed products.

Current wording

On April 28, 2023, we exercised the Oral Option and entered into a license agreement (the "VYN202 License Agreement" and together with the VYN201 License Agreement, the "Tay License Agreements") with Tay granting us a worldwide, exclusive license that is sublicensable through multiple tiers to exploit certain of Tay’s Oral BETi Compounds in all fields. We have the sole responsibility for development, regulatory, marketing and commercialization activities to be conducted for the licensed products at our sole cost and discretion, and shall use commercially reasonable efforts to develop and, if approved, commercialize such products. We may sublicense our rights to a third party without Tay’s consent.

Previous wording

Historically, we have generated revenues under development and license agreements, including royalty payments from sales of Finacea foam. We previously licensed the rights to Finacea to LEO Pharma A/S ("LEO Pharma"). This license was not part of the sale of our commercial business to Journey. For the three months ended June 30, 2024 and 2023, royalty revenues from LEO Pharma in connection with sales of Finacea were $0.2 million and $0.1 million, respectively. For the six months ended June 30, 2024 and 2023, royalty revenues from LEO Pharma in connection with sales of Finacea were $0.3 million and $0.2 million, respectively.

Current wording

Historically, we have generated revenues under development and license agreements, including royalty payments from sales of Finacea foam. We previously licensed the rights to Finacea to LEO Pharma A/S ("LEO Pharma"). This license was not part of the sale of our commercial business to Journey. For both the three months ended September 30, 2024 and 2023, royalty revenues from LEO Pharma in connection with sales of Finacea were $0.1 million. For the nine months ended September 30, 2024 and 2023, royalty revenues from LEO Pharma in connection with sales of Finacea were $0.4 million and $0.3 million, respectively.

Previous wording

Our total research and development expenses for the three months ended June 30, 2024 and 2023 were $7.3 million and $7.2 million, respectively. Total research and development expenses for the six months ended June 30, 2024 and 2023 were $11.0 million and $10.0 million, respectively.

Current wording

Our total research and development expenses for the three months ended September 30, 2024 and 2023 were $10.2 million and $3.3 million, respectively. Total research and development expenses for the nine months ended September 30, 2024 and 2023 were $21.3 million and $13.3 million, respectively.

Previous wording

Revenues totaled $0.2 million and $0.1 million for the three months ended June 30, 2024 and 2023, respectively, consisting of royalty revenue from our royalty agreement with LEO Pharma.

Current wording

Revenues totaled $0.1 million for both the three months ended September 30, 2024 and 2023, consisting of royalty revenue from our royalty agreement with LEO Pharma.

Previous wording

Other income, net for the three months ended June 30, 2024 and 2023 was $1.0 million and $0.3 million, respectively, and was related to interest income earned on cash, cash equivalents and marketable securities.

Current wording

Other income, net for the three months ended September 30, 2024 and 2023 was $0.9 million and $0.2 million, respectively, and was related to interest income earned on cash, cash equivalents and marketable securities.

Previous wording

Revenues totaled $0.3 million and $0.2 million for the six months ended June 30, 2024 and 2023, respectively, consisting of royalty revenue from our royalty agreement with LEO Pharma.

Current wording

Revenues totaled $0.4 million and $0.3 million for the nine months ended September 30, 2024 and 2023, respectively, consisting of royalty revenue from our royalty agreement with LEO Pharma.

Previous wording

Our research and development expenses for the six months ended June 30, 2024 were $11.0 million, representing an increase of $1.0 million, or 10.5%, compared to $10.0 million for the six months ended June 30, 2023. The increase was primarily driven by expenses associated with preparatory and clinical trial activities for a Phase 2b trial of VYN201 in subjects with nonsegmental vitiligo, which was initiated in June 2024, of $3.5 million, which included a $1.0 million milestone payment due to Tay. Additionally, employee-related expenses increased by $0.4 million following the hiring of additional research and development personnel. These increases were partially offset by a decrease in expenses associated with VYN202 of $2.8 million, primarily driven by lower amounts owed to Tay under the VYN202 License Agreement. During the six months ended June 30, 2023, we made cash payments to Tay totaling $4.0 million in connection with entering into the VYN202 License Agreement. We incurred a$1.0 million development milestone payment due to Tay following the initiation of a Phase 1a single ascending dose/multiple ascending dose trial of VYN202 in June 2024.

Current wording

Our research and development expenses for the nine months ended September 30, 2024 were $21.3 million, representing an increase of $8.0 million, or 60.1%, compared to $13.3 million for the nine months ended September 30, 2023. The increase was primarily driven by expenses associated with preparatory and clinical trial activities for a Phase 2b trial of VYN201 in subjects with nonsegmental vitiligo, which was initiated in June 2024, of $8.5 million, which included a $1.0 million milestone payment due to Tay. Additionally, employee-related expenses increased by $0.7 million following the hiring of additional research and development personnel. These increases were partially offset by a decrease in expenses associated with VYN202 of $1.2 million, primarily driven by lower amounts owed to Tay under the VYN202 License Agreement, partially offset by an increase in clinical trial expenses. During the nine months ended September 30, 2023, we made cash payments to Tay totaling $4.0 million in connection with entering into the VYN202 License Agreement. We incurred a $1.0 million development milestone payment due to Tay following the initiation of a Phase 1a SAD/MAD trial of VYN202 in June 2024.

Previous wording

Our general and administrative expenses for the six months ended June 30, 2024 were $7.1 million, representing an increase of $0.6 million, or 9.3%, compared to $6.5 million for the six months ended June 30, 2023. The increase was primarily related to $0.4 million of consulting and professional fees and $0.2 million of employee-related expenses.

Current wording

Our general and administrative expenses for the nine months ended September 30, 2024 were $10.0 million, representing an increase of $0.5 million, or 5.6%, compared to $9.5 million for the nine months ended September 30, 2023. The increase was primarily related to $0.4 million of consulting and professional fees and $0.1 million of employee-related expenses.

Previous wording

Other income, net for the six months ended June 30, 2024 and 2023 was $2.1 million and $0.5 million, respectively, and was related to interest income earned on cash, cash equivalents and marketable securities.

Current wording

Other income, net for the nine months ended September 30, 2024 and 2023 was $3.1 million and $0.7 million, respectively, and was related to interest income earned on cash, cash equivalents and marketable securities.

Previous wording

As of June 30, 2024, we had cash, cash equivalents, restricted cash and marketable securities of $78.1 million and an accumulated deficit of $707.0 million. We had no outstanding debt as of June 30, 2024. For the six months ended June 30, 2024, we incurred a net loss of $15.7 million and used $16.5 million of cash in operations. Based on our current operating plan, we believe our existing cash, cash equivalents, restricted cash and marketable securities are sufficient to fund our operating and capital expenditure requirements for a period of at least 12 months from the date of issuance of the unaudited interim condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q.

Current wording

As of September 30, 2024, we had cash, cash equivalents, restricted cash and marketable securities of $70.2 million and an accumulated deficit of $719.1 million. We had no outstanding debt as of September 30, 2024. For the nine months ended September 30, 2024, we incurred a net loss of $27.8 million and used $25.0 million of cash in operations. Based on our current operating plan, we believe our existing cash, cash equivalents, restricted cash and marketable securities are sufficient to fund our operating and capital expenditure requirements for a period of at least 12 months from the date of issuance of the unaudited interim condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q.

Previous wording

Our sources of funding for both the six months ended June 30, 2024 and 2023 are further evaluated in the cash flow section below. Other than our obligations pursuant to the Tay License Agreements, we have no ongoing material financial commitments that may affect our liquidity over the next five years. See the section titled “—Development and License Agreements—Agreements with Tay” for additional discussion of our financial obligations under the Tay License Agreements.

Current wording

Our sources of funding for both the nine months ended September 30, 2024 and 2023 are further evaluated in the cash flow section below. Other than our obligations pursuant to the Tay License Agreements, we have no ongoing material financial commitments that may affect our liquidity over the next five years. See the section titled “Development and License Agreements—Agreements with Tay” for additional discussion of our financial obligations under the Tay License Agreements.

Previous wording

The following table summarizes our cash flows for the six months ended June 30, 2024 and 2023:

Current wording

The following table summarizes our cash flows for the nine months ended September 30, 2024 and 2023:

Previous wording

During the six months ended June 30, 2024, net cash used in operating activities was $16.5 million and primarily reflected our net loss of$15.7 million adjusted for non-cash stock-based compensation expense of $1.9 million, partially offset by the amortization of premium on marketable securities of $1.4 million. The remainder of the cash used in operations was driven by changes in operating assets and liabilities.

Current wording

During the nine months ended September 30, 2024, net cash used in operating activities was $25.0 million and primarily reflected our net loss of $27.8 million adjusted for non-cash stock-based compensation expense of $2.6 million, partially offset by the amortization of premium on marketable securities of $1.9 million. The remainder of the cash used in operations was driven by changes in operating assets and liabilities.

Previous wording

During the six months ended June 30, 2023, net cash used in operating activities was$15.1 million and primarily reflected our net loss of $15.7 million adjusted for the $1.7 million of non-cash stock-based compensation expense. The remainder of the cash used in operations was driven by changes in operating assets and liabilities.

Current wording

During the nine months ended September 30, 2023, net cash used in operating activities was $20.2 million and primarily reflected our net loss of $22.3 million adjusted for the $2.6 million of non-cash stock-based compensation expense. The remainder of the cash used in operations was driven by changes in operating assets and liabilities.

Previous wording

During the six months ended June 30, 2024, net cash provided by investing activities was $15.5 million and consisted of $37.5 million of proceeds received from the sale and maturity of marketable securities, partially offset by $22.0 million paid for the purchase of marketable securities.

Current wording

During the nine months ended September 30, 2024, net cash provided by investing activities was $10.6 million and consisted of $61.1 million of proceeds received from the sale and maturity of marketable securities, partially offset by $50.5 million paid for the purchase of marketable securities.

Previous wording

During the six months ended June 30, 2023, net cash provided by investing activities of $5.0 million consisted of deferred payment received from Journey in January 2023.

Current wording

During the nine months ended September 30, 2023, net cash provided by investing activities of $5.0 million consisted of the deferred payment received from Journey in January 2023.

Previous wording

During the six months ended June 30, 2024, net cash used in financing activities was immaterial.

Current wording

During the nine months ended September 30, 2024, net cash used in financing activities was immaterial.

Previous wording

During the six months ended June 30, 2023, net cash used in financing activities was $0.2 million and consisted of $0.4 million paid for the redemption of convertible preferred stock, partially offset by $0.2 million of proceeds received from the issuance of common stock under our at-the-market equity facility.

Current wording

During the nine months ended September 30, 2023, net cash used in financing activities was $0.3 million and consisted of $0.4 million paid for the redemption of convertible preferred stock, partially offset by $0.2 million of proceeds received from the issuance of common stock under our at-the-market equity facility.

Previous wording

Our critical accounting policies are described in our Annual Report on Form 10-K for the year ended December 31, 2023 filed with the SEC on March 1, 2024. There have been no material changes to these policies for the six months ended June 30, 2024, except as set forth below.

Current wording

Our critical accounting policies are described in our Annual Report on Form 10-K for the year ended December 31, 2023 filed with the SEC on March 1, 2024. There have been no material changes to these policies for the nine months ended September 30, 2024, except as set forth below.

Previous wording

As of June 30, 2024, product revenue, net, product returns, and discontinued operations were no longer deemed critical accounting policies and research and development expenses was newly identified as a critical accounting policy.

Current wording

Effective June 30, 2024, product revenue, net, product returns, and discontinued operations were no longer deemed critical accounting policies and research and development expenses was newly identified as a critical accounting policy.