Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) (FRA:6ZY) believes its operations and capital requirements to be fully funded into 2024, the company announced as part of its fourth-quarter and full-year results published Wednesday.
As of December 31, the company had $59.2 million in cash and equivalents on hand, compared to $70.9 million on the same day a year earlier. Since then, Zynerba has sold and issued more than 10 million shares of common stock, good for net proceeds of $42.2 million. All told, the company said that is enough capital to last “well into the first half of 2024.”
For the quarter, research and development expenses were $5.6 million, while general and administrative expenses were $4.6 million. The company’s net loss for the quarter was $9.6 million, with a basic and diluted loss per share of $0.33. For the full year, R&D costs were $35.7 million, G&A came in at $16.4 million and the net loss was $51.3 million, or $1.90 per share.
Zynerba is currently developing Zygel, a CBD-based gel, for use in four therapeutic indications: Fragile X Syndrome (FXS), Deletion Syndrome, Autism Spectrum Disorder (ASD) and Developmental and Epileptic Encephalopathies (DEE).
“We expect to make significant progress in 2021 on all four indications for which we are developing Zygel, including initiating a pivotal trial in patients with Fragile X syndrome, who have a highly methylated FMR1 gene to confirm the positive results in this population of responders in the CONNECT-FX trial,” CEO Armando Anido said in a statement. “Screening in the INSPIRE trial of patients with 22q11.2 deletion syndrome has resumed, now that COVID-19 restrictions in Australia have begun to ease. Once enrollment is complete, we will update our expectation on when we will see topline results for this trial.”
The company expects to initiate its single double-blind, placebo-controlled pivotal FXS trial before the end of 2021 to confirm the positive results observed in this population of responders in the previous CONNECT-FX trial. Zynerba said it believes that positive results from this confirmatory pivotal trial will be sufficient to support the submission of a New Drug Application (NDA) for Zygel in FXS.
The company plans to review the trial design and protocol for the new confirmatory pivotal trial through a Type C meeting with the US Food and Drug Administration (FDA) in the first half of 2021.
Zynerba also intends to discuss with the FDA data supporting the potential efficacy of Zygel in ASD, including the results of the Phase 2 BRIGHT trial in children and adolescents with moderate to severe ASD, to determine the regulatory path forward.
With regard to Australia, the company recently initiated a second clinical site for the 14-week open-label Phase 2 INSPIRE trial in children and adolescents with genetically confirmed 22q. Once enrollment is complete, a timeframe for disclosing topline results of the trial will be provided, the company said.
Additionally, Zynerba expects to conduct an observational trial that will help finalize target syndrome selection in one or more DEE syndromes in 2021. Due to the heterogeneity of patients who fall under the DEE umbrella, Zynerba will pursue individual syndromes rather than considering DEE as a single disorder or condition, the company said.
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