KCa4.1

The prevalence of Epilepsy has been rising over the past few years, which prompts the growing demand for treatment options. The increasing prevalence of Epilepsy and the growing research and development activities to develop novel therapies to treat Epilepsy to drive the market. The companies developing the potential therapies in the last stage of development include Xenon Pharmaceuticals, Engage Therapeutics, Janssen Biotech, and several others LAS VEGAS, April 11, 2023 /PRNewswire/ -- DelveInsight's ' Epilepsy Pipeline Insight – 2023 ' report provides comprehensive global coverage of available, marketed, and pipeline epilepsy therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the epilepsy pipeline domain. Key Takeaways from the Epilepsy Pipeline Report DelveInsight's epilepsy pipeline report depicts a robust space with 70+ active players working to develop 75+ pipeline therapies for epilepsy treatment. Key epilepsy companies such as Alexza Pharmaceuticals, Xenon Pharmaceuticals, GW Pharmaceuticals, Equilibre Biopharmaceuticals, Takeda Pharmaceuticals, Abide therapeutics, Otsuka pharmaceutical, H. Lundbeck A/S, Spark Therapeutics, Equilibre Biopharmaceuticals, ES Therapeutics, Supernus Pharmaceuticals, Inc., MGC Pharmaceuticals, Engrail Therapeutics INC, SK biopharmaceuticals, Longboard Pharmaceuticals, Janssen Research & Development, LLC, Equilibre Biopharmaceuticals, Cerevel Therapeutics, LLC, Neurona Therapeutics, Praxis Precision Medicines, UCB Pharma, Receptor Life Sciences, NeuroPro Therapeutics, Inc., Avicanna, Amring Pharmaceuticals Inc., Ovid Therapeutics, Addex Therapeutics, IAMA Therapeutics, CODA Biotherapeutics, Cerebral Therapeutics, Engrail Therapeutics, and others are evaluating new epilepsy drugs to improve the treatment landscape. Promising epilepsy pipeline therapies in various stages of development include Alprazolam, XEN1101, XEN-496, XEN 901, GWP42006, EQU-001, TAK-935, Lu AG06466, OPC-214870, CG 01, EQU-001, ES-481, SPN-817, MGCND00EP1, NBI 827104, ENX-101, Cenobamate, SKL-24741, LP-352, JNJ-40411813, EQU-001, CVL-865, NRTX 1001, PRAX 628, Padsevonil, RLS103, NPT 2042, AVCN-319302, PRAX-020, AMZ002, OV 350, IAMA-6, CT-010, ENX-101, and others. In March 2023, IAMA Therapeutics, a preclinical-stage pharmaceutical company focused on the discovery, development, and commercialization of novel medicines to make a difference in the lives of individuals suffering from brain disorders, announced that preclinical data supporting its IAMA-6 program in focal refractory epilepsy will be presented at the 75th American Academy of Neurology (AAN) Annual Meeting, taking place on April 22-27 in Boston, Massachusetts. In February 2023, Addex Therapeutics announced that enrollment into Part 1 of Phase 2 clinical study of ADX71149 (JNJ-40411813) for the treatment of epilepsy had been completed. ADX71149 is a selective metabotropic glutamate type 2 (mGlu2) receptor-positive allosteric modulator (PAM). Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, is conducting the trial. In January 2023, Equilibre Biopharmaceuticals Corp., reported positive topline results from the Phase 2 clinical trial evaluating the clinical safety, tolerability, and preliminary efficacy of EQU-001 – a novel anti-inflammatory anti-seizure medication - administered as once-daily oral adjunctive therapy for focal seizures in adults with epilepsy. The results of the EQU-001 study are encouraging because they demonstrate impressive tolerability and encouraging efficacy for adult patients with focal epilepsy, especially for those with seizures that are difficult to treat. In January 2023, Signant Health, introduced a novel electronic diary (eDiary) data capture solution designed to simplify the experience for patients and research sites participating in epilepsy trials while ensuring robust outcome assessment data to support trial endpoints. Developed in collaboration with The Epilepsy Study Consortium (TESC), the new patient-reported outcome measure offers clinical research sponsors and organizations a standardized solution to capture common seizure data elements with high-quality data. In June 2022, CODA Biotherapeutics, Inc. announced that preclinical data from the Company's focal epilepsy program demonstrated that treatment with the Company's lead-engineered receptor and small molecule activator drug significantly reduced focal seizure frequency in vivo. Results showed that the rapid reduction in seizures is consistent with the pharmacokinetics of the activator drug in mouse models. Data also demonstrated that the expression of CODA's lead receptor is highly correlated with a reduction in seizure frequency. In September 2022, Avicanna Inc. was pleased to announce that it has expanded its research collaboration in the field of Epilepsy with a new collaboration with the University of Toronto and Dr. Mac Burnham's research team to explore the efficacy of Avicanna's proprietary formulations in pre-clinical models for Epilepsy. The research collaboration led by Dr. Mac Burnham's team will explore the efficacy of Avicanna's drug candidates, including AVCN319302, in animal models for Epilepsy. In December 2022, Praxis Precision Medicines, Inc. and UCB announced a strategic collaboration, based upon Praxis' PRAX-020 program, for the discovery of small molecule therapeutics as potential treatments of KCNT1-related epilepsies. Under the terms of the collaboration, UCB retains an exclusive option to in-license global development and commercialization rights to any resulting KCNT1 small molecule development candidate. In January 2022, Ovid Therapeutics Inc. announced the company has entered into an exclusive license agreement with AstraZeneca for a library of early-stage small molecules targeting the KCC2 transporter, including lead candidate OV350. The company seeks to optimize and accelerate the development of these KCC2 transporter activators in epilepsies and potentially other neuropathic conditions. In August 2021, Marinus Pharmaceuticals, Inc. announced that it has entered into an agreement with Orion Corporation whereby Orion received exclusive rights to commercialize the oral and intravenous (IV) dose formulations of ganaxolone in the European Economic Area, United Kingdom, and Switzerland for CDKL5 deficiency disorder (CDD), tuberous sclerosis complex (TSC) and refractory status epilepticus (RSE). Request a sample and discover the recent advances in epilepsy drug treatment @ Epilepsy Pipeline Report The epilepsy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage epilepsy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the epilepsy clinical trial landscape. Epilepsy Overview Epilepsy is a chronic condition characterized by unprovoked, recurrent seizures. A seizure is an uncontrollable surge of electrical activity in the brain. Seizures are classified into two types. Seizures that affect the entire brain are known as generalized seizures. Focal seizures, also known as partial seizures, affect only one part of the brain. A mild seizure can be difficult to detect. It can last a few seconds, and you are unaware of it. Stronger seizures, lasting from a few seconds to several minutes, can cause spasms and uncontrollable muscle twitches. Epilepsy symptoms vary according to the area of the brain where seizure activity occurs and the type of seizure. Epilepsy symptoms may include: stiff muscles, Become stiff and falling backward, muscles relaxing and going floppy, confusion, breathing becomes difficult, unconscious, visual disturbances/hallucinations, uncontrollable jerking and shaking, called a "fit," losing awareness and staring blankly into space, strange sensations, such as a "rising" feeling in the tummy, unusual smells or tastes, and a tingling feeling in your arms or legs and collapsing. Epilepsy treatment can help most people with epilepsy have fewer seizures or completely stop having seizures. Find out more about drugs for epilepsy @ New Epilepsy Drugs A snapshot of the Epilepsy Pipeline Drugs mentioned in the report: Learn more about the emerging epilepsy pipeline therapies @ Epilepsy Clinical Trials Epilepsy Therapeutics Assessment The epilepsy pipeline report proffers an integral view of epilepsy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Epilepsy Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Ophthalmic, Oral, Parenteral, Subcutaneous, Topical, Transdermal Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule Therapeutics Assessment By Mechanism of Action: KCNQ potassium channel agonists, Monoacylglycerol lipase inhibitors, Cholesterol 24-hydroxylase inhibitors, NMDA receptor modulators, AMPA receptor antagonists, GABA A receptor agonists, Cannabinoid receptor modulators Key Epilepsy Companies: Alexza Pharmaceuticals, Xenon Pharmaceuticals, GW Pharmaceuticals, Equilibre Biopharmaceuticals, Takeda Pharmaceuticals, Abide therapeutics, Otsuka pharmaceutical, H. Lundbeck A/S, Spark Therapeutics, Equilibre Biopharmaceuticals, ES Therapeutics, Supernus Pharmaceuticals, Inc., MGC Pharmaceuticals, Engrail Therapeutics INC, SK biopharmaceuticals, Longboard Pharmaceuticals, Janssen Research & Development, LLC, Equilibre Biopharmaceuticals, Cerevel Therapeutics, LLC, Neurona Therapeutics, Praxis Precision Medicines, UCB Pharma, Receptor Life Sciences, NeuroPro Therapeutics, Inc., Avicanna, Amring Pharmaceuticals Inc., Ovid Therapeutics, Addex Therapeutics, IAMA Therapeutics, CODA Biotherapeutics, Cerebral Therapeutics, Engrail Therapeutics, and others. Key Epilepsy Pipeline Therapies: Alprazolam, XEN1101, XEN-496, XEN 901, GWP42006, EQU-001, TAK-935, Lu AG06466, OPC-214870, CG 01, EQU-001, ES-481, SPN-817, MGCND00EP1, NBI 827104, ENX-101, Cenobamate, SKL-24741, LP-352, JNJ-40411813, EQU-001, CVL-865, NRTX 1001, PRAX 628, Padsevonil, RLS103, NPT 2042, AVCN-319302, PRAX-020, AMZ002, OV 350, IAMA-6,CT-010, ENX-101, and others. Dive deep into rich insights for new drugs for epilepsy treatment; visit @ Epilepsy Medications Table of Contents For further information on the epilepsy pipeline therapeutics, reach out @ Epilepsy Drug Treatment Related Reports Epilepsy Market Epilepsy Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key epilepsy companies including Takeda Pharmaceuticals, Abide therapeutics, Otsuka pharmaceutical, among others. Epilepsy Epidemiology Forecast Epilepsy Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epilepsy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Refractory Epilepsy Pipeline Refractory Epilepsy Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key refractory epilepsy companies, including Eisai Co.LTD., Alexza Pharmaceuticals, Xenon Pharmaceuticals, among others. Partial Epilepsy Pipeline Partial Epilepsy Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key partial epilepsy companies, including GW Pharmaceuticals, Equilibre Biopharmaceuticals, among others. Refractory Epilepsy Market Refractory Epilepsy Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key refractory epilepsy companies including Eisai Co.LTD., Alexza Pharmaceuticals, Xenon Pharmaceuticals, among others. Partial Epilepsy Market Partial Epilepsy Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key partial epilepsy companies, including GW Pharmaceuticals, Equilibre Biopharmaceuticals, among others. Other Trending Reports Tay-sachs Disease Or Gm2 Gangliosidosis Market | Onycholysis Market | Diagnostic Imaging Equipment Market | Chemotherapy-induced Peripheral Neuropathy Market | Global Electrophysiology Devices Market | Anaphylaxis Market | Atherectomy Devices Market | Helicobacter Pylori Infections Market | Ophthalmic Imaging Equipment Market | Androgenetic Alopecia Market | Allergic Rhinitis Market | Chronic Inflammatory Demyelinating Polyneuropathy Market | Chronic Inflammtory Demyelinating Polyneuropathy Market | Colorectal Cancer Crc Market | Opioid Induced Constipation Market | Vertigo Market | Bone Anchored Hearing Systems Market | Wound Closure Devices Market | Hip Replacement Devices Market | Hemodynamic Monitoring Systems Market | Egfr Non-small Cell Lung Cancer Market | Helicobacter Pylori Infection Market | Hyperkalemia Market | Polycythemia Market Neurostimulation Devices Market | Carpal Tunnel Syndrome Market | Ventilator Market | Cerebral Aneurysm Market | Alpha Antitrypsin Market | Binge Eating Disorder Market | Bunion Market | Concussions Market Size | Exocrine Pancreatic Insufficiency Market | Healthcare Due Diligence Services | Minimal Residual Disease Market | Hypertrophic Scar Market | Lung Fibrosis Market | Anterior Uveitis Market | 22q11.2 deletion syndrome Market | X-Linked Retinitis Pigmentosa (XLRP) Market | Acute Radiation Syndrome Market | Alpha-1 Protease Inhibitor Deficiency Market | Androgenetic Alopecia Market | Hyperlipidemia Market | Cardiotoxicity Market | Hypertrophic Cardiomyopathy Market | Fatty Acid Oxidation Disorders (FAODs) Market | Androgen Insensitivity Syndrome Market | Emphysema Market | Canaloplasty Market | Dravet Syndrome Market | Celiac Disease Market | Chlamydia Infections Market | Syphilis Market | Renal Tubular Acidosis Market | Palmoplantar Pustulosis (PPP) Market | Aplastic Anemia Market | Bacterial Pneumonia Market | B cell Chronic Lymphocytic Leukemia Market | B cell Lymphomas Market | Behcets Disease Market Neoantigen-based Personalized Cancer therapeutic Vaccines Competitive Landscape and Market Forecast—by 2035 | Glioblastoma Market Related Healthcare Blogs Telepyschiatry Market Digital Therapeutics for Mental Health Related Healthcare Services Healthcare Consulting Healthcare Competitive Intelligence Services Healthcare Asset Prioritization Services About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn |Facebook|Twitter Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

Ulixacaltamide (PRAX-944) Phase 2b Essential1 study topline results for essential tremor expected in 1Q23; Praxis to enter quiet period following market close on Thursday, February 9 Topline results expected for each of three clinical-stage epilepsy programs in 2023 – PRAX-222 first-in-patient EMBRAVE Study safety data mid-2023, PRAX-628 first-in-human Phase 1 data mid-2023, PRAX-562 Phase 2 EMBOLD Study results in 2H23 Cash and investments of $100.5 million as of December 31, 2022 supports runway into 1Q24 BOSTON, Feb. 07, 2023 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today provided a corporate update and reported financial results for the fourth quarter and full year 2022. “This year is set up to be transformative for Praxis and for the patients that we serve, with topline results for the ulixacaltamide Essential1 study imminent and data expected from each of our four clinical-stage programs in 2023,” said Marcio Souza, president and chief executive officer of Praxis. “Based on our understanding of epilepsy genetics and unique capabilities to translate these insights into therapies for patients suffering from a broad range of CNS disorders, we have built two proprietary platforms, Cerebrum™ for small molecules and Solidus™ for antisense oligonucleotides, that we expect will drive continuous innovation and value creation this year and beyond.” Recent Business Highlights and Upcoming Milestones: Cerebrum™ Small Molecule Platform Praxis expects topline results from the ongoing ulixacaltamide (PRAX-944) Essential1 study for the treatment of moderate to severe essential tremor (ET) in the first quarter of 2023. Essential1 is a randomized, double-blind, placebo-controlled, dose-range-finding Phase 2b trial evaluating the efficacy, safety and tolerability of once-daily daytime treatment of 60 or 100 mg of ulixacaltamide compared to placebo after 56 days. The primary endpoint is change from baseline to day 56 in the modified Activities of Daily Living (mADL1) score. Following topline results, Praxis intends to request an end-of-Phase 2 meeting with the FDA and initiate its ulixacaltamide Phase 3 development program for the treatment of ET in mid-2023.The Company is also conducting a randomized, double-blind, placebo-controlled Phase 2 trial to evaluate the efficacy, safety and tolerability of once-daily treatment of up to 100 mg of ulixacaltamide as a non-dopaminergic treatment for the motor symptoms of Parkinson’s disease. The primary endpoint for the study is change from baseline in the International Parkinson and Movement Disorder Society Unified Parkinson’s Disease Rating Scale (UPDRS), Part III (motor examination) score in the OFF state. Topline results are expected in the fourth quarter of 2023.In November 2022, Praxis announced plans to initiate the PRAX-562 Phase 2 EMBOLD study for the treatment of pediatric patients with developmental and epileptic encephalopathies (DEEs), following FDA authorization to proceed with the study as Praxis proposed, up to the planned maximum dose of 1.0 mg/kg/day. The EMBOLD study is a randomized, double-blind, placebo-controlled Phase 2 clinical trial to evaluate the safety, tolerability, efficacy (motor seizure frequency) and pharmacokinetics (PK) of PRAX-562 in pediatric participants aged 2 to 18 years with DEEs, followed by an open-label extension. Approximately 20 participants will be enrolled initially in two distinct cohorts (n≈10 for SCN2A-DEE and n≈10 for SCN8A-DEE). Topline results for both cohorts are expected in the second half of 2023.The Company is conducting a Phase 1 healthy volunteer study of PRAX-628 to evaluate the tolerability, PK, pharmacodynamics and food effect of PRAX-628 across single and multiple ascending dose cohorts. Topline results from the Phase 1 study are expected in mid-2023, with plans to initiate a Phase 2 study in focal epilepsy in the fourth quarter of 2023.In December 2022, Praxis announced that it entered into a strategic collaboration, based upon its PRAX-020 program, with UCB for the discovery of small molecule therapeutics as potential treatments for KCNT1 epilepsies. Under the terms of the collaboration, UCB retains an exclusive option to in-license global development and commercialization rights to any resulting KCNT1 small molecule development candidate. Praxis received an upfront payment from UCB, and if the option is exercised by UCB, would be eligible to receive an option fee and future success-based development and commercialization milestone payments, for a total of up to approximately $100 million, in addition to tiered royalties on net sales of any resulting products from the collaboration.In December 2022, Praxis presented the following posters at the American Epilepsy Society (AES) 2022 Annual Meeting: PRAX-562 is a Well-tolerated, Novel Persistent Sodium Channel Blocker with Broad Anticonvulsant Activity in Multiple DEE Mouse Models (Abstract Number: 1.281)A Phase 1 Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Food Effect of PRAX-562 in Healthy Volunteers (Abstract Number: 2.24)A Phase 1 Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of PRAX-562 in Healthy Volunteers (Abstract Number: 2.478)Disease Impact and Burden in Patients with SCN2A-Related Developmental and Epileptic Encephalopathy (Abstract Number: 2.092)A Novel Approach to Assess the Impact of Disease in Patients with SCN8A-Related Developmental and Epileptic Encephalopathy (Abstract Number: 2.096)PRAX-628: A Novel Sodium Channel Blocker with Greater Potency and Activity Dependence Compared to Standard of Care (Abstract Number: 3.311)PRAX-628 is a Novel, Well-tolerated, Activity Dependent Sodium Channel Blocker with Potent Anticonvulsant Activity (Abstract Number: 3.28) Solidus™ Antisense Oligonucleotide (ASO) Platform Praxis is conducting the first dose cohort (Part 1) of the PRAX-222 EMBRAVE study for the treatment of pediatric patients with early-onset SCN2A-DEE in the U.S. Following collection of the safety and efficacy data from the initial cohort of patients in the EMBRAVE study, the data will be evaluated and submitted to the FDA to support further dose escalation. Part 1 of the EMBRAVE study is a 21-week open label cohort, in which participants will receive PRAX-222 for up to 13 weeks, designed to determine the safety and tolerability of intrathecal delivery of PRAX-222. Topline results from Part 1 of the PRAX-222 EMBRAVE study are expected in mid-2023.The Company remains on track to nominate a development candidate for its most advanced preclinical ASO program, PRAX-080 for the treatment of PCDH19, in the second half of 2023. Fourth Quarter and Full Year 2022 Financial Results: As of December 31, 2022, Praxis had $100.5 million in cash, cash equivalents and marketable securities, compared to $275.9 million in cash, cash equivalents and marketable securities as of December 31, 2021. This decrease of $175.4 million primarily reflects cash used in operations of $185.0 million during the year ended December 31, 2022, partially offset by $9.6 million in net proceeds from at-the-market offerings of shares of the Company’s common stock. The Company’s cash, cash equivalents and marketable securities as of December 31, 2022 are expected to fund operations into the first quarter of 2024. Research and development expenses were $28.3 million for the fourth quarter of 2022, compared to $43.5 million for the fourth quarter of 2021. Research and development expenses were $155.0 million for the year ended December 31, 2022, compared to $120.3 million for the year ended December 31, 2021. The increase in research and development expenses for full year 2022 of $34.7 million was primarily attributable to $29.9 million in increased expenses related to the Company’s Cerebrum™ and Solidus™ platforms. General and administrative expenses were $13.1 million for the fourth quarter of 2022, compared to $15.1 million for the fourth quarter of 2021. General and administrative expenses were $59.9 million for the year ended December 31, 2022, compared to $47.1 million for the year ended December 31, 2021. The increase in general and administrative expenses for full year 2022 of $12.8 million was primarily attributable to an increase of $11.8 million in personnel-related expenses due to changes in headcount, including an increase of $5.3 million in stock-based compensation expense. Praxis reported a net loss of $41.2 million for the fourth quarter of 2022, including $6.4 million of stock-based compensation expense, compared to $58.6 million for the fourth quarter of 2021, including $6.1 million of stock-based compensation expense. Praxis reported a net loss of $214.0 million for the year ended December 31, 2022, including $28.6 million of stock-based compensation expense, compared to a net loss of $167.1 million for the year ended December 31, 2021, including $22.7 million of stock-based compensation expense. As of December 31, 2022, Praxis had 49.4 million shares of common stock outstanding. About PraxisPraxis Precision Medicines is a clinical-stage biopharmaceutical company translating insights from genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Praxis is applying genetic insights to the discovery and development of therapies for rare and more prevalent neurological disorders through our proprietary small molecule platform, Cerebrum™, and antisense oligonucleotide (ASO) platform, Solidus™, using our understanding of shared biological targets and circuits in the brain. Praxis has established a diversified, multimodal CNS portfolio including multiple programs across movement disorders and epilepsy, with four clinical-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on LinkedIn and Twitter. Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Praxis’ future expectations, plans and prospects, including, without limitation, statements regarding expectations, plans and timing for our clinical data, the anticipated timing of our clinical trials and regulatory interactions, the development of our product candidates, including the design of our clinical trials and the treatment potential of our product candidates, and the sufficiency of our cash, cash equivalents and marketable securities, as well as other statements containing the words “anticipate,” “believe,” “continue,” “could,” “endeavor,” “estimate,” “expect,” “anticipate,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “target,” “will” or “would” and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials; the expected timing of submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials; Praxis’ ability to continue as a going concern; and other risks concerning Praxis’ programs and operations as described in its Annual Report on Form 10-K for the year ended December 31, 2022 to be filed and other filings made with the Securities and Exchange Commission. Although Praxis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on information and factors currently known by Praxis. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. Praxis undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise. PRAXIS PRECISION MEDICINES, INC.CONDENSED CONSOLIDATED BALANCE SHEETS(Amounts in thousands)(Unaudited) December 31, 2022 2021 Assets Cash and cash equivalents$61,615 $138,704 Marketable securities 38,874 137,207 Prepaid expenses and other current assets 10,351 11,498 Property and equipment, net 971 1,213 Operating lease right-of-use assets 2,901 3,653 Other non-current assets 416 472 Total assets$115,128 $292,747 Liabilities and stockholders’ equity Accounts payable$14,672 $10,780 Accrued expenses 15,850 26,844 Operating lease liabilities 3,500 4,311 Deferred revenue 5,000 — Common stock 5 5 Additional paid-in capital 606,918 567,598 Accumulated other comprehensive loss (173) (176)Accumulated deficit (530,644) (316,615)Total liabilities and stockholders’ equity$115,128 $292,747 PRAXIS PRECISION MEDICINES, INC.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Amounts in thousands, except share and per share amounts)(Unaudited) Three Months EndedDecember 31, Year EndedDecember 31, 2022 2021 2022 2021 Operating expenses: Research and development$28,329 $43,511 $155,040 $120,257 General and administrative 13,124 15,146 59,946 47,075 Total operating expenses 41,453 58,657 214,986 167,332 Loss from operations (41,453) (58,657) (214,986) (167,332)Other income: Other income, net 280 70 957 271 Total other income 280 70 957 271 Loss before benefit from income taxes (41,173) (58,587) (214,029) (167,061)Benefit from income taxes — 5 — — Net loss$(41,173) $(58,582) $(214,029) $(167,061)Net loss per share attributable to common stockholders, basic and diluted$(0.87) $(1.30) $(4.64) $(3.94)Weighted average common shares outstanding, basic and diluted 47,594,823 44,964,580 46,096,737 42,454,055 1mADL is a composite sum of items 1 to 11 of the TETRAS-ADL subscale and items 6 (bilateral) and 7 of the TETRAS-PS; mADL score is calculated as the sum of all 13 items (item 6 of TETRAS-PS x2) and ranges from 0 to 42

Praxis said its internal research efforts give it confidence that small molecules can selectively inhibit the KCNT1 potassium channel. It’s been a tough year of layoffs and pipeline culls for Praxis Precision Medicines, but the biotech is ending 2022 on a positive note courtesy of a collaboration with UCB to develop a potential first-ever treatment for a specific type of epilepsy. The collaboration is based upon Praxis’ PRAX-020 program to discover small-molecule therapeutics as potential treatments of KCNT1-related epilepsies, for which there are currently no approved treatments. As well as an upfront payment from the Belgian biopharma, Praxis will be eligible for development and commercial milestone payments to the tune of up to $100 million, on top of royalties. UCB retains an exclusive option to in-license global development and commercialization rights to any KCNT1 small-molecule candidate that results from the agreement. The money will come as welcome news to Praxis, which laid off staff and halted a clutch of clinical trials after its nervous system drug PRAX-114 failed to deliver in a phase 2 study in June. The company moved to prioritize its programs in movement disorders and epilepsy, a decision that appears to have paid off with the UCB deal. Not that Praxis’ bank account is empty—the Boston-based company had $123.7 million in cash and equivalents as of the end September. It may be a drop of over $150 million on the funds the biotech entered the year with, but Praxis reckoned it was still enough to fund it through the start of 2024, even before any payments from UCB are taken into account. “Our internal research efforts give us confidence that small molecules can selectively inhibit the KCNT1 channel and potentially could be an effective treatment for individuals suffering from KCNT1-related epilepsy,” Praxis CEO Marcio Souza said in a postmarket release Tuesday. “The collaboration with UCB validates this approach and will allow us to accelerate efforts toward a potential treatment for KCNT1 patients.” As well as a number of other potential epilepsy programs in preclinical development, Praxis has one already in phase 2, with others due to enter phase 1 and 2 before the end of the year. One of these, PRA-562, was recently released from an FDA clinical hold.