Description of the Business and Liquidity | 1. Description of the Business and Liquidity We are a clinical stage pharmaceutical company focused on developing and commercializing innovative therapeutics to treat patients suffering from seizure disorders. Our clinical stage product candidate, ganaxolone, is a positive allosteric modulator of GABA A A In December 2019, an outbreak of a novel strain of coronavirus (COVID-19) was identified in Wuhan, China. This virus was declared a pandemic by the World Health Organization in March 2020 and has spread to nearly every country in the world, including the United States (U.S.). Efforts to contain the spread of COVID-19 have intensified and many countries, including the U.S., have implemented travel restrictions, business shutdowns and social distancing measures that have impacted clinical development through supply chain shortages and clinical trial enrollment difficulties as hospitals reduce and redeploy staff, divert resources to patients suffering from COVID-19 and limit hospital access for non-patients. The pandemic poses the risk that we, our employees, contractors, suppliers, or other partners may be prevented from conducting normal business activities for an indefinite period of time, including those due to restrictions that may be requested or mandated by governmental authorities. The continued global spread of COVID-19 has not materially adversely impacted our operating results, financial condition or cash flows as of and for the nine months ended September 30, 2021. However, COVID-19 has impacted our clinical operations and timelines. In response to COVID-19, for our ongoing clinical trials, we have implemented multiple measures consistent with the U.S. Food and Drug Administration’s guidance on the conduct of clinical trials of medical products during the COVID-19 pandemic, including implementing remote site monitoring and remote visits using telemedicine where needed. However, COVID-19 may still adversely impact our clinical trials. For example, our Phase 3 clinical trial in refractory status epilepticus (RSE) is conducted in hospitals, including academic medical centers, which have experienced high rates of COVID-19 admissions. Due to COVID-19, priorities in several academic medical centers participating in the RAISE Trial, including staff turnover and the need for clinical sites to devote significant resources to patients with COVID-19, the trial has experienced site initiation and enrollment delays. Given these challenges, we previously updated our expectation of top-line data readout for the RAISE Trial to be available in the second half of 2022. In addition, our ganaxolone clinical trials in the outpatient setting may be negatively impacted if patients and their caregivers do not want to participate while COVID-19 outbreaks continue. We are unable to predict the impact that COVID-19 will have in the future on our business, operating results, financial condition and cash flows. The duration and severity of the pandemic and its long-term impact on our business are uncertain at this time, and our ability to raise sufficient additional financing depends on many factors beyond our control, including the current volatility in the capital markets as a result of the COVID-19 pandemic. Liquidity We have not generated any product revenues and have incurred operating losses since inception, including losses of $70.5 million for the nine months ended September 30, 2021 . There is no assurance that profitable operations will ever be achieved, and if achieved, could be sustained on a continuing basis. In addition, development activities, clinical and preclinical testing, if approved, and commercialization of our product candidates will require significant additional financing. Our accumulated deficit as of million and we expect to incur substantial losses in future periods. We plan to finance our future operations with a combination of proceeds from the issuance of equity securities, the issuance of debt, government funding, collaborations, licensing transactions, other transactions, including the sale of priority review vouchers if awarded, and revenues from future product sales, if any. We have not generated positive cash flows from operations, and there are no assurances that we will be successful in obtaining an adequate level of financing for the development and, if approved, commercialization of our product candidates. On July 30, 2021, we entered into a collaboration agreement (the Collaboration Agreement) with Orion Corporation (Orion), whereby Orion received exclusive rights to commercialize the oral and IV dose formulations of ganaxolone in the European Economic Area, United Kingdom and Switzerland in multiple seizure disorders, including CDD, tuberous sclerosis complex (TSC) and RSE. Under the agreement, we received a 90 On May 11, 2021 (the Closing Date), we entered into a Credit Agreement and Guaranty (as amended by that certain letter agreement on May 17, 2021, the Credit Agreement) with Oaktree Fund Administration, LLC, as administrative agent and the lenders party thereto that provides for a five-year tranches (collectively, the Term Loans). Refer to Note 9. Notes Payable for additional information. In September 2020, we entered into a contract (BARDA Contract) with the Biomedical Advanced Research and Development Authority (BARDA), a division of the U.S. Department of Health and Human Services’ Office of the Assistant Secretary for Preparedness and Response. Under the BARDA Contract, we receive d an award of up to an estimated $51 million for development of IV-administered ganaxolone for the treatment of RSE. The BARDA Contract provides for f unding to support , on a cost-sharing basis , the completion of a Phase 3 clinical trial of IV-administered ganaxolone in patients with RSE , which covers the RAISE Trial , funding of pre-clinical studies to evaluate IV-administered ganaxolone a s an effective treatment for RSE due to chemical nerve gas agent exposure, and funding of certain ganaxolone manufacturing scale-up and regulatory activities. The BARDA Contract consists of an approximately two-year five years In connection with the closing of an equity financing in December 2020, we issued a total of 5,000,000 shares of common stock in an underwritten public offering resulting in aggregate net proceeds, after underwriting discounts and commissions in the public offering and other estimated offering expenses, of $64.9 million. In connection with the closing of an equity financing in June 2020, we issued a total of 4,600,000 shares of common stock in an underwritten public offering resulting in aggregate net proceeds, after underwriting discounts and commissions in the public offering and other estimated offering expenses, of $42.9 million. We believe that our cash and cash equivalents balance as of September 30, 2021 will be sufficient to maintain the minimum cash balance required under our debt facility and to fund our operating expenses and capital expenditure requirements for at least the next 12 months from the date of this filing. Reverse stock split On September 23, 2020, we effected a 1 . All of the share and per share amounts included in the accompanying financial statements and these notes have been adjusted to reflect the Reverse Split . |