DEVON, PA — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE)this week reported financial results for the fourth quarter and full year ended December 31, 2022, and provided an overview of recent operational highlights and a pipeline update.
“We remain focused on enrolling patients in our confirmatory pivotal Phase 3 RECONNECT trial, and expect topline results in the first half of 2024,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We are committed to bringing the first pharmaceutical product indicated for the treatment of behavioral symptoms of Fragile X syndrome to market, and with a cash runway to mid-year 2024, we remain well-positioned on achieving that goal.”
Operational Highlights and Pipeline Update
Zygel in Fragile X Syndrome (FXS)
- The Company expects topline results from RECONNECT, a confirmatory pivotal Phase 3 trial of Zygel in patients with FXS, in the first half of 2024. The Company believes that the results from RECONNECT, if positive, will be sufficient to support the submission of a New Drug Application (NDA) in the U.S. and a Marketing Authorization Application (MAA) in Europe for Zygel in patients with FXS.
- Published data in the Journal of Neurodevelopmental Disorders describing the role of the endocannabinoid system and cannabidiol therapy in FXS. The review of evidence suggests a central role for the endocannabinoid system in neuronal development and cognitive function and in the pathogenesis of FXS, and the potential role of cannabidiol as a treatment for FXS. (Press Release).
- Published data from the Phase 3 CONNECT-FX study of Zygel in the Journal of Neurodevelopmental Disorders. The Phase 3 study demonstrated that patients with FXS who have a highly methylated FMR1 gene that were treated with Zygel showed a significant reduction in behavioral symptoms compared to those treated with placebo, and that Zygel was well tolerated. (Press Release).
- The Company announced that the U.S. Patent and Trademark Office (USPTO) issued a patent titled “Treatment of Fragile X Syndrome With Cannabidiol,” which includes claims directed to methods of treating FXS with cannabidiol. This new patent, which expires in 2038, is part of an expanding international intellectual property portfolio covering Zygel. (Press Release)
Zygel in 22q11.2 Deletion Syndrome (22q)
- Based on the positive Phase 2 INSPIRE trial data announced in June 2022 (Press Release), the Company held an initial meeting with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2022 to obtain feedback on the Phase 2 data and regulatory pathway for Zygel in patients with 22q. The Company expects to continue its productive dialogue with the FDA on this topic and arrive at an acceptable trial design by the end of 2023. The Company currently plans to initiate a Phase 3 program in children and adolescents with 22q following topline results from RECONNECT.
- In November 2022, the Company announced that the European Commission granted orphan drug designation to cannabidiol, the active ingredient in Zygel, for the treatment of 22q.
- The Company presented positive long-term 38-week data from the Phase 2 INSPIRE trial with Zygel in children and adolescents with 22q at the 61st Annual Meeting of the American College of Neuropsychopharmacology. The data showed that through 38-weeks of treatment, sustained, statistically significant improvements were seen in children and adolescents treated with Zygel in the Pediatric Anxiety Rating Scale (PARS-R), all five scales of the Anxiety, Depression and Mood Scale (ADAMS), and all five subscales of the Aberrant Behavior Checklist – Community (ABC-C). These results are consistent with the previously reported 14-week treatment data suggesting a positive risk-benefit profile for Zygel in improving anxiety-related and behavioral symptoms in children and adolescents with 22q when added to a stable standard of care. (Press Release).
- During the fourth quarter of 2022, the Company announced that the USPTO issued a patent titled “Treatment of 22q11.2 Deletion Syndrome With Cannabidiol,” which includes claims directed to methods of treating one or more behavioral symptoms of 22q with cannabidiol, and expires in 2040. (Press Release).
Fourth Quarter and Full Year 2022 Financial Results
Research and development expenses were $5.5 million for the fourth quarter of 2022, including stock-based compensation of $0.5 million. General and administrative expenses were $3.2 million in the fourth quarter of 2022, including stock-based compensation expense of $0.5 million. Net loss for the fourth quarter of 2022 was $8.0 million, with basic and diluted loss per share of $(0.18).
Research and development expenses were $21.1 million for full year 2022, including stock-based compensation of $2.0 million. General and administrative expenses were $14.2 million for full year 2022, including stock-based compensation expense of $2.4 million. Net loss for full year 2022 was $35.0 million, with basic and diluted loss per share of $(0.82).
Financial Outlook
As of December 31, 2022, cash and cash equivalents were $50.6 million, compared to $67.8 million as of December 31, 2021. On March 13, 2023, the Company issued a statement confirming that it does not hold cash deposits or securities at Silicon Valley Bank.
On May 11, 2021, the Company entered into a Controlled Equity OfferingSM Sales Agreement (2021 Sales Agreement), with Cantor Fitzgerald & Co., Canaccord Genuity, LLC, H.C. Wainwright & Co. LLC and Ladenburg Thalmann & Co. Inc., as sales agents, pursuant to which the Company may sell, from time to time, up to $75.0 million of its common stock. During the year ended December 31, 2022, the Company sold and issued 4,608,274 shares of its common stock under the 2021 Sales Agreement in the open market resulting in gross proceeds of $6.2 million and net proceeds of $5.8 million, after deducting commissions and offering expenses. From January 1, 2023 through March 22, 2023, the Company sold and issued 1,179,077 shares of its common stock under the 2021 Sales Agreement in the open market resulting in gross proceeds of $0.7 million and net proceeds of $0.6 million, after deducting commissions and offering expenses.
On July 21, 2022, the Company entered into a Purchase Agreement and registration rights agreement for up to $20 million with Lincoln Park Capital Fund, LLC (LPC), a Chicago-based institutional investor. During the year ended December 31, 2022, the Company sold and issued 350,000 shares of its common stock under the 2022 Purchase Agreement with LPC in the open market resulting in gross proceeds of $0.3 million and net proceeds of $0.2 million, after deducting offering expenses. From January 1, 2023 through March 22, 2023, the Company sold and issued 1,950,000 shares of its common stock under the Purchase Agreement, resulting in gross and net proceeds of $1.0 million.
Management believes that the Company’s cash and cash equivalents are sufficient to fund operations and capital requirements into mid-year 2024. Top-line results from the Company’s confirmatory pivotal Phase 3 RECONNECT trial of Zygel in patients with FXS are expected in the first half of 2024.
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