- \n
- \nAnticipate initiating a Phase 3 trial of adjunctive ganaxolone in tuberous sclerosis complex (TSC) in Q3 2021, targeting first patient enrolled during Q4 2021; continue to expect topline Phase 2, open label data in Q3 2021\n<\/li>\n
- \nEuropean Medicines Agency (EMA) is expected to accept accelerated assessment of ganaxolone for the treatment of seizures associated with CDKL5 deficiency disorder (CDD); Marinus plans to submit a marketing authorization application to the EMA by the end of Q3 2021\n<\/li>\n
- \nReceived notice of allowance from the U.S. Patent and Trademark Office for refractory status epilepticus (RSE) dosing and method of treatment patent application\u00a0\n<\/li>\n
- \nU.S. Food and Drug Administration (FDA) indicated alignment on overall trial design for the established status epilepticus (ESE) trial; company expects to begin U.S. enrollment in 1H 2022\n<\/li>\n
- \nSubmitted New Drug Application to FDA for ganaxolone for the treatment of seizures associated with CDD\n<\/li>\n
- \nEntered exclusive collaboration with Orion Corporation for European commercialization of ganaxolone\n<\/li>\n
- \nSite initiations for Phase 3 RSE trial were affected by COVID-19-related delays in Q2 2021; expect vast majority of sites to be open by end of Q3 2021, with topline data anticipated for release 2H 2022\n<\/li>\n<\/ul>\n
RADNOR, Pa.–(BUSINESS WIRE<\/a>)–Marinus Pharmaceuticals, Inc<\/a>. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today provided an update on its clinical and regulatory development activities and reported its financial results for the second quarter ended June 30, 2021.\n<\/p>\n
\n\u201cWe have seen tremendous progress over the past few months, including the recent announcements of our European collaboration with Orion Corporation and the submission of our NDA for ganaxolone in CDKL5 deficiency disorder,\u201d said Scott Braunstein, M.D., Chief Executive Officer of Marinus Pharmaceuticals. \u201cOrion\u2019s leadership in the European rare orphan disease market and its ability to commercialize both oral and IV ganaxolone makes them a strong partner.\u201d\n<\/p>\n
\nDr. Braunstein added, \u201cWe are also pleased to report that the FDA indicated they are in overall alignment with the design of our TSC Phase 3 clinical trial, which we expect to initiate in the fourth quarter of the year. We anticipate the remainder of 2021 to be equally eventful.\u201d\n<\/p>\n
Pipeline Update<\/b><\/p>\n
CDKL5 Deficiency Disorder (CDD)<\/span><\/p>\n
- \n
- \nSubmitted New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ganaxolone for the treatment of seizures associated with CDD in July 2021; expect to receive a filing notification letter from the FDA prior to the end of Q3 2021<\/p>\n
- \n
- \nThe NDA includes a request for Priority Review, which, if granted, accelerates completion of the FDA review goal to six months following the 60-day filing review, compared to a standard review of 10 months\n<\/li>\n
- \nThe NDA also includes a request for a rare pediatric disease (RPD) priority review voucher. The FDA granted RPD designation for ganaxolone for the treatment of CDD in July 2020\n<\/li>\n
- \nNDA acceptance for filing enables the opportunity to draw $30 million under the existing Oaktree Capital Management, L.P. facility, subject to the satisfaction of certain customary conditions described in the credit agreement\n<\/li>\n<\/ul>\n<\/li>\n
- \nEuropean Medicines Agency (EMA) is expected to accept accelerated assessment of ganaxolone for the treatment of seizures associated with CDD; if granted, accelerated assessment would reduce target review time to 150 from 210 days\n<\/li>\n
- \nMarinus expects to file a marketing authorization application with the EMA for ganaxolone for the treatment of seizures associated with CDD by the end of Q3 2021\n<\/li>\n
- \nMarinus, with the support of its strategic collaborator, Orion Corporation, expects to announce a European expanded access program\n<\/li>\n<\/ul>\n
Tuberous Sclerosis Complex (TSC)<\/span><\/p>\n
- \n
- \nTop-line, open label, Phase 2 trial (CALM Study) data expected to be reported in Q3 2021<\/p>\n
- \n
- \nTrial evaluates the effectiveness, safety and tolerability of adjunctive ganaxolone treatment in patients with TSC\n<\/li>\n<\/ul>\n<\/li>\n
- \nShould ganaxolone be approved in CDD, the FDA has indicated a single pivotal TSC Phase 3 trial could be sufficient for the approval\n<\/li>\n
- \nA scientific advice meeting with the EMA is targeted for Q1 of 2022\n<\/li>\n
- \nA global Phase 3 randomized, double blind, placebo-controlled trial in approximately 160 patients is expected to begin enrollment during Q4 2021\n<\/li>\n<\/ul>\n
Status Epilepticus<\/span><\/p>\n
- \n
- \nFDA indicated alignment on overall trial design for the established status epilepticus (ESE) RESET (Researching Established Status Epilepticus Treatment) trial<\/p>\n
- \n
- \nThe Phase 2 RESET trial of adjunctive ganaxolone in ESE is planned to begin U.S. enrollment in 1H 2022\n<\/li>\n
- \nThe RESET trial will examine a shorter dosing paradigm in conjunction with a first line antiepileptic drug (AED) and will target convulsive status epilepticus\n<\/li>\n<\/ul>\n<\/li>\n
- \nAs previously announced, due to COVID-19 priorities in several participating Phase 3 academic centers, including staff turnover and the need for clinical sites to devote significant resources to patients with COVID-19, the Phase 3 RAISE (RAndomized therapy In Status Epilepticus) trial has experienced site initiation and enrollment delays. Given these most recent challenges, Marinus has shifted its top-line data readout to 2H 2022<\/p>\n
- \n
- \nCompany expects the vast majority of sites to be open by the end of Q3 2021\n<\/li>\n<\/ul>\n<\/li>\n
- \nThe RAISE trial is a randomized, double-blind, placebo-controlled trial that will evaluate the efficacy and safety of intravenous (IV) ganaxolone in patients with refractory status epilepticus (RSE) who have failed benzodiazepines and two or more second line IV AEDs\n<\/li>\n
- \nPlanning continues for the RAISE II trial to support European registration of IV ganaxolone in RSE and is on track to initiate enrollment in the first half of 2022<\/p>\n
- \n
- \nOverall alignment on the clinical development plan was achieved at an end-of-Phase 2 scientific advice meeting held in March 2021 with the EMA\n<\/li>\n
- \nThe RAISE II trial differs from the RAISE trial in the U.S., with RAISE II using ganaxolone that can be initiated earlier in the course of RSE. If successful, this trial will provide complementary data to the Phase 3 RAISE trial in the U.S.\n<\/li>\n<\/ul>\n<\/li>\n
- \nCompany continues to field requests from investigators for IV ganaxolone for patients with super refractory status epilepticus under emergency Investigational New Drug Applications\n<\/li>\n<\/ul>\n
Corporate Update<\/b><\/p>\n
- \n
- \nReceived notice of allowance from the U.S. Patent and Trademark Office for refractory status epilepticus (RSE) dosing and method of treatment patent application\n<\/li>\n
- \nSigned exclusive collaboration with Orion Corporation for European commercialization of ganaxolone in CDD, TSC and RSE,generating \u20ac25 million (~$30 million) in cash as an upfront payment and up to \u20ac97 million (~$115 million) in R&D reimbursement, development and commercialization milestone payments, as well as tiered double-digit royalties on net sales in Europe\n<\/li>\n
- \nOrganization further strengthened through key hires in patient advocacy, marketing, R&D, finance and commercial operations\n<\/li>\n
- \nAppointment of Santiago Arroyo, M.D., Ph.D. to the Board of Directors<\/p>\n
- \n
- \nDr. Arroyo brings a distinguished career in academic neurology and clinical research and development, including his leadership in the neurology department at the Johns Hopkins Hospital. In addition, he has extensive expertise in epilepsy drug development contributing to numerous new therapeutic alternatives. Most recently, he served as Chief Medical Officer of Momenta Pharmaceuticals\n<\/li>\n<\/ul>\n<\/li>\n
- \nSecond generation formulation of ganaxolone to be discussed during the Marinus October 5, 2021, R&D Day\n<\/li>\n<\/ul>\n
Financial Update<\/b><\/p>\n
- \n
- \nAt June 30, 2021, the company had cash, cash equivalents, and investments of $112.5 million, compared to $140.0 million at December 31, 2020. This excludes the ~$30 million upfront payment associated with the recently completed European collaboration with Orion\n<\/li>\n
- \nMarinus recognized $1.9 million and $3.7 million in Biomedical Advanced Research and Development Authority (BARDA) federal contract revenue for the three and six months ended June 30, 2021, respectively; no federal contract revenue was recognized in the six months ended June 30, 2020\n<\/li>\n
- \nResearch and development expenses were $18.6 million and $37.2 million for the three and six months ended June 30, 2021, respectively, as compared to $11.8 million and $26.8 million, respectively, for the same periods in the prior year; the increase was due primarily to start-up of the RSE Phase 3 trial, regulatory activities associated with the CDD NDA submission, and increased R&D headcount\n<\/li>\n
- \nGeneral and administrative expenses were $6.8 million and $17.2 million for the three and six months ended June 30, 2021, respectively, as compared to $4.1 million and $8.0 million, respectively, for the same periods in the prior year; the primary drivers of the increase were additional support for scale up of the company\u2019s operations as well as preparation for commercialization\n<\/li>\n
- \nThe company reported net losses of $23.8 million and $51.0 million for the three and six months ended June 30, 2021, respectively; cash used in operating activities increased to $39.1 million for the six months ended June 30, 2021, compared to $30.0 million for the same period a year ago\n<\/li>\n
- \nReaders are referred to, and encouraged to read in its entirety, the company\u2019s Quarterly Report on Form 10-Q for the three months ended June 30, 2021, to be filed with the Securities and Exchange Commission, which includes further detail on the European partnership with Orion and the company\u2019s business plans, operations, financial condition, and results of operations\n<\/li>\n<\/ul>\n
Corporate Guidance <\/b><\/p>\n
- \n
- \nFor the fiscal year 2021, the company expects total GAAP operating expenses (selling, general & administrative expenses and R&D expenses) to be in the range of $113 to $118 million, of which the company expects stock-based compensation to be approximately $16 million. 2021 BARDA contract revenues are projected to be in the range of $7 to $10 million for the full year. Previously expected BARDA contract revenues for 2021 were in the range of $9 to $12 million. Total GAAP operating expenses and stock-based compensation estimates remain unchanged from the prior guidance\n<\/li>\n<\/ul>\n
\n
\n \n Marinus Pharmaceuticals, Inc.<\/b><\/p>\n
Selected Financial Data (in thousands, except share and per share amounts) <\/b><\/p>\n<\/td>\n<\/tr>\n
\n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n \n\u00a0\n<\/p>\n<\/td>\n
\n \n June 30, <\/b><\/p>\n<\/td>\n
\n \n December 31, <\/b><\/p>\n<\/td>\n<\/tr>\n
\n \n \n\u00a0\n<\/p>\n<\/td>\n
\n \n 2021<\/b><\/p>\n<\/td>\n
\n \n 2020<\/b><\/p>\n<\/td>\n<\/tr>\n
\n \n \n\u00a0\n<\/p>\n<\/td>\n
\n \n \n\u00a0\n<\/p>\n<\/td>\n
\n \n \n\u00a0\n<\/p>\n<\/td>\n<\/tr>\n
\n \n ASSETS<\/b><\/p>\n<\/td>\n
\n \n \n\u00a0\n<\/p>\n<\/td>\n
\n \n \n\u00a0\n<\/p>\n<\/td>\n<\/tr>\n
\n \n \nCash and cash equivalents\n<\/p>\n<\/td>\n
\n \n \n$112,493\n<\/p>\n<\/td>\n
\n \n \n$138,509\n<\/p>\n<\/td>\n<\/tr>\n
\n \n \nInvestments\n<\/p>\n<\/td>\n
\n \n \n\u2014\n<\/p>\n<\/td>\n
\n \n \n1,474\n<\/p>\n<\/td>\n<\/tr>\n
\n \n \nOther assets\n<\/p>\n<\/td>\n
\n \n \n12,496\n<\/p>\n<\/td>\n
\n \n \n10,479\n<\/p>\n<\/td>\n<\/tr>\n
\n \n \nTotal assets\n<\/p>\n<\/td>\n
\n \n \n$124,989\n<\/p>\n<\/td>\n
\n \n \n$150,462\n<\/p>\n<\/td>\n<\/tr>\n
\n \n LIABILITIES AND STOCKHOLDERS\u2019 EQUITY<\/b><\/p>\n<\/td>\n
\n \n \n\u00a0\n<\/p>\n<\/td>\n
\n \n \n\u00a0\n<\/p>\n<\/td>\n<\/tr>\n
\n \n \nCurrent liabilities\n<\/p>\n<\/td>\n
\n \n \n$16,694\n<\/p>\n<\/td>\n
\n \n \n$10,729\n<\/p>\n<\/td>\n<\/tr>\n
\n \n \nLong Term Debt, Net\n<\/p>\n<\/td>\n
\n \n \n$10,970\n<\/p>\n<\/td>\n
\n \n \n$0\n<\/p>\n<\/td>\n<\/tr>\n
\n \n \nOther long-term liabilities\n<\/p>\n<\/td>\n
\n \n \n2,266\n<\/p>\n<\/td>\n
\n \n \n2,534\n<\/p>\n<\/td>\n<\/tr>\n
\n \n \nTotal liabilities\n<\/p>\n<\/td>\n
\n \n \n29,930\n<\/p>\n<\/td>\n
\n \n \n13,263\n<\/p>\n<\/td>\n<\/tr>\n
\n \n \nTotal stockholders\u2019 equity\n<\/p>\n<\/td>\n
\n \n \n95,059\n<\/p>\n<\/td>\n
\n \n \n137,199\n<\/p>\n<\/td>\n<\/tr>\n
\n \n \nTotal liabilities and stockholders\u2019 equity\n<\/p>\n<\/td>\n
\n \n \n$124,989\n<\/p>\n<\/td>\n
\n \n \n$150,462\n<\/p>\n<\/td>\n<\/tr>\n<\/table>\n
- \nFor the fiscal year 2021, the company expects total GAAP operating expenses (selling, general & administrative expenses and R&D expenses) to be in the range of $113 to $118 million, of which the company expects stock-based compensation to be approximately $16 million. 2021 BARDA contract revenues are projected to be in the range of $7 to $10 million for the full year. Previously expected BARDA contract revenues for 2021 were in the range of $9 to $12 million. Total GAAP operating expenses and stock-based compensation estimates remain unchanged from the prior guidance\n<\/li>\n<\/ul>\n
- \nFDA indicated alignment on overall trial design for the established status epilepticus (ESE) RESET (Researching Established Status Epilepticus Treatment) trial<\/p>\n
- \nTop-line, open label, Phase 2 trial (CALM Study) data expected to be reported in Q3 2021<\/p>\n
- \nSubmitted New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ganaxolone for the treatment of seizures associated with CDD in July 2021; expect to receive a filing notification letter from the FDA prior to the end of Q3 2021<\/p>\n
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