CDKL5 program (Amicus Therapeutics)

ZTALMY® (ganaxolone) first quarter U.S. net product revenue of $3.3 million Now planning to conduct an interim analysis for the Phase 3 refractory status epilepticus trial in the second half of 2023 CHMP opinion for ZTALMY in CDKL5 deficiency disorder expected by end of May; European Medicines Agency communicated that the major objection related to the choice of regulatory starting material has been resolved Phase 3 tuberous sclerosis complex (TSC) trial data now expected mid-2024 Received a notice of allowance from the USPTO for a new method of use patent for ganaxolone in TSC; when granted, the term will run through 2040 Entered exclusive distribution and supply agreement with Biologix for ZTALMY in select markets in the Middle East and North Africa Cash runway into second half of 2024 with cash, cash equivalents and short-term investments of $199.2 million as of March 31, 2023 Marinus to host conference call on May 11, 2023, at 8:30 a.m. ET RADNOR, Pa.--(BUSINESS WIRE)--Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the first quarter ended March 31, 2023. “We are pleased with our 2023 first quarter ZTALMY revenue in the U.S. and are on track to meet our previously stated full year revenue guidance of $15-17 million,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. “As part of our commitment to provide ganaxolone to a growing number of patients in several important global regions, we are collaborating with Orion Corporation to prepare for a potential launch in Europe, have entered into a distribution agreement with Biologix for the Middle East and North Africa, and continue to work closely with Tenacia on development and commercialization in China.” “We are now planning to conduct an interim analysis in RAISE, our Phase 3 refractory status epilepticus trial, and if it meets the predefined stopping criteria, plan to share these results before year end,” continued Dr. Braunstein. ZTALMY® ZTALMY® (ganaxolone) oral suspension CV U.S. net product revenue of $3.3 million for the first quarter of 2023 Continued growth in commercial patients with approximately 100 patients active on therapy at the end of the first quarter of 2023 Expanded commercial coverage for ZTALMY with approximately 225 million covered lives, including both commercial and government programs Clinical Pipeline Status Epilepticus Phase 3 RAISE trial of intravenous (IV) ganaxolone in refractory status epilepticus (RSE) continues to advance with Marinus now planning to conduct an interim analysis, and if it meets the pre-defined stopping criteria, to report topline data before year end 2023 IV formulation of ganaxolone with new citrate buffer planned to be incorporated into the RAISE trial in the second quarter of 2023 Registration batches of this formulation have been placed on stability; shelf life expected to be 24 months 16 patients have now been treated for super refractory status epilepticus under emergency INDs Phase 3 RAISE II trial in RSE (for European registration) enrollment anticipated to begin in the second half of 2023 Phase 2 RESET trial in established status epilepticus (ESE) completion of first cohort anticipated before year end 2023 Ganaxolone development in the RAISE trial is being funded in part by the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response at the U.S. Department of Health and Human Services, under contract number 75A50120C00159. CDKL5 Deficiency Disorder Marketing Authorization Application (MAA) The Committee for Medicinal Products for Human Use (CHMP) opinion on the MAA is expected by the end of May 2023 The European Medicines Agency communicated that the major objection related to the choice of regulatory starting material has been resolved Collaborating with Orion Corporation to prepare for a potential commercial launch in Europe Tuberous Sclerosis Complex (TSC) Continue to enroll patients in the Phase 3 TrustTSC trial of oral ganaxolone at sites in the U.S. and Europe with topline data now anticipated mid-2024, primarily due to administrative and regulatory delays in certain ex-U.S. countries Expect vast majority of clinical sites will be ready to enroll patients by mid-year 2023, including sites in Canada, Israel, China, Italy, Belgium and Australia Received notice of allowance from the United States Patent and Trademark Office for a new method of use patent for ganaxolone in TSC; when granted, the term of this patent will run through 2040 Second Generation Product Development Pending FDA feedback and agreement, multiple ascending dose (MAD) trial now expected to initiate in the third quarter of 2023 with preliminary data expected by year end 2023 Planning to finalize clinical program design for Lennox-Gastaut syndrome in the first quarter of 2024, pending results of the MAD trial Prodrug development continues to advance with lead oral candidate selected; Phase 1 data targeted for 2024 General Business and Financial Update For the fiscal year 2023, the Company maintains its prior guidance with expected ZTALMY U.S. net product revenues of between $15 million and $17 million, BARDA revenues in the range of $8 million to $11 million and total GAAP operating expenses, inclusive of SG&A and R&D, in the range of $165 to $175 million, of which the company expects stock-based compensation of approximately $16 million. Entered exclusive distribution and supply agreement with Biologix for ganaxolone in select markets in the Middle East and North Africa. Initiated U.S. onshoring of the manufacturing capabilities for ganaxolone API which has the potential to drive a greater than 30% reduction in API supply cost. Expect that cash, cash equivalents, and short-term investments of $199.2 million as of March 31, 2023, will be sufficient to fund the Company’s operating expenses, capital expenditure requirements and maintain the minimum cash balance of $15 million required under the Company’s debt facility into the second half of 2024. Financial Results Recognized $3.3 million in net product revenues for the three months ended March 31, 2023. Net product revenue consists of ZTALMY product sales, which was launched in the U.S. in the third quarter of 2022. Recognized $7.0 million in Biomedical Advanced Research and Development Authority (BARDA) federal contract revenue for the three months ended March 31, 2023, as compared to $1.5 million for the same period in the prior year. The increase was primarily driven by activity associated with start-up of the API onshoring initiative. Research and development expenses were $27.9 million for the three months ended March 31, 2023, compared to $18.0 million for the same period in the prior year; the increase was due primarily to increased costs associated with our API onshoring effort, increased TSC and RSE clinical trial activity, and increased headcount. Selling, general and administrative (SG&A) expenses were $15.2 million for the three months ended March 31, 2023, as compared to $11.7 million for the same period in the prior year; the primary drivers of the change were increased headcount and commercial support for the U.S. launch of ZTALMY. The Company reports a net loss of $34.7 million for the three months ended March 31, 2023; cash used in operating activities increased to $41.5 million for the three months ended March 31, 2023, compared to $27.7 million for the same period a year ago. At March 31, 2023, the Company had cash, cash equivalents, and short-term investments of $199.2 million, compared to $240.6 million at December 31, 2022. Readers are referred to, and encouraged to read in its entirety, the Company’s Quarterly Report on Form 10-Q for the three months ended March 31, 2023, to be filed with the Securities and Exchange Commission, which includes further detail on the company’s business plans, operations, financial condition, and results of operations. Selected Financial Data (in thousands, except share and per share amounts) March 31, 2023 (Unaudited) December 31, 2022 ASSETS Cash and cash equivalents $ 146,904 $ 240,551 Short-term investments 52,297 - Other assets 27,616 18,967 Total assets $ 226,817 $ 259,518 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities $ 21,514 $ 25,017 Long term debt, net 71,498 71,018 Revenue interest financing payable, net 30,893 29,857 Other long-term liabilities 17,827 17,626 Total liabilities 141,732 143,518 Total stockholders’ equity 85,085 116,000 Total liabilities and stockholders’ equity $ 226,817 $ 259,518 Three Months Ended March 31, 2023 (unaudited) 2022 (unaudited) Revenue: Product revenue, net $ 3,332 $ - Federal contract revenue 7,048 1,513 Collaboration revenue - 12,673 Total revenue 10,380 14,186 Expenses: Research and development 27,933 17,991 Selling, general and administrative 15,204 11,737 Cost of product revenue 206 - Cost of IP license fee - 1,169 Total expenses: 43,343 30,897 Loss from operations (32,963) (16,711) Interest income 2,343 12 Interest expense (4,147) (1,692) Other income (expense), net 37 (970) Net loss $ (34,730) $ (19,361) Per share information: Net loss per share of common stock- basic and diluted $ (0.67) $ (0.52) Basic and diluted weighted average shares outstanding 51,769,685 36,890,568 Other comprehensive income Unrealized gain on available-for-sale securities 74 - Total comprehensive loss $ (34,656) $ (19,361) Conference Call Information Participants may access the conference call via webcast on the Investor page of Marinus’ website at ir.marinuspharma.com/events-and-presentations. An archived version of the call will be available approximately two hours after the completion of the event on the website. About Marinus Pharmaceuticals Marinus is a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. The Company’s commercial product, ZTALMY® (ganaxolone) oral suspension CV, has been approved by the U.S. FDA for the treatment of seizures associated with CDKL5 deficiency disorder in patients two years of age and older. The potential of ganaxolone is also being studied in other seizure disorders, including in Phase 3 trials in tuberous sclerosis complex and refractory status epilepticus. Ganaxolone is a neuroactive steroid GABAA receptor modulator that acts on a well-characterized target in the brain known to have anti-seizure effects. It is being developed in IV and oral formulations to maximize therapeutic reach for adult and pediatric patients in acute and chronic care settings. For more information visit . Forward-Looking Statements To the extent that statements contained in this press release are not descriptions of historical facts regarding Marinus, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as "may", "will", "expect", "anticipate", "estimate", "intend", "believe", and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Examples of forward-looking statements contained in this press release include, among others, statements regarding our commercialization and marketing plans for ZTALMY; the potential benefits ZTALMY will provide for physicians and patients; the potential benefits from the U.S. onshoring of the manufacturing capabilities for ganaxolone API; our expectations regarding the ZTALMY One program; statements regarding our expected clinical development plans, enrollment in our clinical trials, regulatory communications and submissions for ganaxolone, and the timing thereof; our expected cash runway; our expectations regarding BARDA funding; our expectations and beliefs regarding the FDA and EMA with respect to our product candidates; our expectations regarding the development of new formulations and prodrug candidates; our expectation regarding the impact of the COVID-19 pandemic on our business and clinical development plans; our financial projections; the potential safety and efficacy of ganaxolone, as well as its therapeutic potential in a number of indications; and other statements regarding the company's future operations, financial performance, financial position, prospects, objectives and other future event. Forward-looking statements in this press release involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, unexpected results or delays in the commercialization of ZTALMY; unexpected market acceptance, payor coverage or future prescriptions and revenue generated by ZTALMY; unexpected actions by the FDA or other regulatory agencies with respect to our products; competitive conditions and unexpected adverse events or patient outcomes from being treated with ZTALMY, uncertainties and delays relating to the design, enrollment, completion, and results of clinical trials; unanticipated costs and expenses; the varying interpretation of clinical data; our ability to comply with the FDA’s requirement for additional post-marketing studies in the required time frames; the timing of regulatory filings for our other product candidates; the potential that regulatory authorities, including the FDA and EMA, may not grant or may delay approval for our product candidates including with respect to the CDD MAA; early clinical trials may not be indicative of the results in later clinical trials; clinical trial results may not support regulatory approval or further development in a specified indication or at all; actions or advice of the FDA or EMA may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional clinical trials; our ability to obtain and maintain regulatory approval for our product candidate; our ability to develop new formulations of ganaxolone or prodrugs; our ability to obtain, maintain, protect and defend intellectual property for our product candidates; the potential negative impact of third party patents on our or our collaborators’ ability to commercialize ganaxolone; delays, interruptions or failures in the manufacture and supply of our product candidate; the size and growth potential of the markets for the company’s product candidates, and the company’s ability to service those markets; the company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated; the company’s expectations, projections and estimates regarding expenses, future revenue, capital requirements, and the availability of and the need for additional financing; the company’s ability to obtain additional funding to support its clinical development and commercial programs; the potential for our ex-US partners to breach their obligations under their respective agreements with us or terminate such agreements in accordance with their respective terms; the risk that drug product quality requirements may not support continued clinical investigation of our product candidates and result in delays or termination of such clinical studies and product approvals; the effect of the COVID-19 pandemic on our business, the medical community, regulators and the global economy; and the availability or potential availability of alternative products or treatments for conditions targeted by us that could affect the availability or commercial potential of our product candidate. This list is not exhaustive and these and other risks are described in our periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at . Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

CHERRY HILL, N.J., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Vyant Bio, Inc. (“Vyant Bio” or “Company”) (Nasdaq: VYNT) is an innovative biotechnology company reinventing drug discovery for complex neurodevelopmental and neurodegenerative disorders. The Company’s proprietary central nervous system (“CNS”) drug discovery platform combines human-derived organoid models of brain disease, scaled biology, and machine learning to identify and validate drug targets and therapeutic candidates. Today, Vyant Bio provided key takeaways from its Platform and Poster presentations delivered by Matthew Green, Ph.D., Vyant Bio’s Senior Scientist, at the 2022 CDKL5 Forum Hosted by the Loulou Foundation on November 7th and 8th, 2022, in Boston, MA. CDKL5 (cyclin-dependent kinase-like 5) deficiency disorder (CDD) is a rare neurodevelopmental condition caused by pathogenic mutations in the CDKL5 gene. The most common symptoms include early-onset, difficult to control seizures and neurodevelopmental impairment that affects cognitive, motor, speech and visual function. The platform and poster presentations can be found at Presentations :: Vyant Bio, Inc. (VYNT) In summary, phenotypic and target-based screening of patient-derived CDKL5 organoids identified several promising drug targets and therapeutic candidates that reversed the abnormal neuronal hyperactivity in a CDD disease-specific manner. Key takeaways from the presentation included: Vyant Bio’s proprietary CNS Drug Discovery Platform is a paradigm for high-throughput biomarker-based screening that utilizes patient biology, and data science to drive de-risked target and hit discovery. The screening process also indicates human efficacy at the outset of a drug discovery program. Vyant Bio has developed a CDKL5-patient derived cortical organoid model that provides a robust disease-relevant model of CDKL5 deficiency disorder (CDD). CDD patient-derived organoids exhibit a hyperexcitable functional phenotype suitable for high-throughput screening. The phenotypic and target-based screening process identified several promising therapeutic targets and small molecules that rescued the disease phenotype, including potential new chemical entity (NCE) and repurposing candidates. In collaboration with Cyclica, a novel receptorwas identified that showed a disease-specific deficiency in function in CDD organoids. In silico based screening identified small molecule compounds that were able to restore sensitivity of the receptor in CDD cortical organoids. Robert T. Fremeau, Jr., Ph.D., Chief Scientific Officer of Vyant Bio, commented: “Our presentations at the Loulou Foundation CDKL5 Forum highlighted the promise and potential of our human first CNS drug discovery platform to drive drug discovery for CNS genetic disorders. The key element of our platform is obtaining a deeper understanding of human disease biology at the cellular level through the application of innovative enabling technologies. We are excited by the opportunity to apply our proprietary CNS drug discovery platform to discover breakthrough therapies for patients with complex neurodevelopmental and neurodegenerative disorders.” ABOUT CDKL5 CDKL5 stands for cyclin-dependent kinase-like 5, which is a protein whose gene is on the X chromosome. Mutations in it cause deficiencies in the protein level and severely affect brain development. About 90% of patients with CDKL5 deficiency disorder (CDD) are girls. CDD patients suffer from seizures that usually begin within the first few months of life, as well as from profound neurodevelopmental delay. Estimates suggest that one in 40,000-60,000 live births develop the disorder, making it difficult to study when compared to common diseases like cancer and heart disease. ABOUT THE LOULOU FOUNDATION The Loulou Foundation is a private non-profit organization founded in 2015 to support the development of effective therapeutics and eventual cures for CDD. Through robust grant and directed research programs, the Foundation provides tools and resources to basic and clinical scientists to enable the development of disease-modifying therapeutics for CDD. These programs include support for pre-clinical, translational, and clinical research into basic CDKL5 biology, CDD disease mechanisms, and the proof-of-concept studies for gene therapy and genome modifying therapeutics. Visit for more information. ABOUT VYANT BIO, INC. Vyant Bio, Inc. (“Vyant Bio” or the “Company”) (Nasdaq: VYNT) is an innovative biotechnology company focused on identifying unique biological targets and novel and repurposed therapeutics for treating the debilitating neurodevelopmental and neurodegenerative disorders for which there are no current therapies. Vyant Bio has built a platform of therapeutics seeking to treat neurodevelopmental and neurodegenerative diseases, with current programs targeting Rett Syndrome (“Rett”), CDKL5 Deficiency Disorders (“CDD”), and familial Parkinson’s Disease. The Company’s approach to drug discovery integrates human-derived biology with artificial intelligence and machine learning technologies to de-risk candidate selection, with the goal of improving the potential effectiveness of drugs discovered earlier in the development cycle. Vyant Bio’s management believes that drug discovery needs to progressively shift to more efficient methods as the widely used models for predicting safe and effective drugs have under-performed, as evidenced by the significant time and cost of bringing novel drugs to market. By combining sophisticated data science capabilities with highly functional human cell derived disease models, Vyant Bio seeks to leverage its current ability to screen and test therapeutic candidates, and create a unique approach to assimilating data that supports decision making iteratively throughout the discovery phase of drug development to identify both novel and repurposed CNS therapeutic candidates. For more information, please visit or follow Vyant Bio at: Internet: LinkedIn: Twitter: @VyantBio Forward Looking Statements: This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements pertaining to Vyant Bio, Inc.’s expectations regarding future financial and/or operating results, the efficacy of our drug screening and discovery process, and potential for our services, future revenue or growth in this press release constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to, statements that contain words such as “will,” “believes,” “plans,” “anticipates,” “expects,” and “estimates”) should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in our attempts to discover drug candidates, partner with pharmaceutical and other biotechnology companies, achieve profitability, adapt to the global coronavirus pandemic, raise capital to meet our liquidity needs, and other risks discussed in the Vyant Bio, Inc. Form 10-K for the year ended December 31, 2021, and any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date hereof. Vyant Bio disclaims any obligation to update these forward-looking statements. Investor Contact: Skyline Corporate Communications Group, LLC Scott Powell, President One Rockefeller Plaza, 10th Floor New York, NY 10020 USA Office: (646) 893-5835 x2 Email: info@skylineccg.com ###