Praxis Precision Medicines, Inc.

Praxis Precision Medicines, Inc. is a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company that translates insights from genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. The company operates through two proprietary platforms: Cerebrum, its small molecule platform that utilizes deep understanding of neuronal excitability and neuronal networks to develop orally available precision therapies, and Solidus, its antisense oligonucleotide (ASO) platform that is an efficient, targeted precision medicine discovery and development engine anchored on a proprietary, computational methodology. The company applies genetic insights to the discovery and development of therapies for neurological disorders through these platforms, each of which currently has multiple programs with significant potential for additional program and indication expansion.

The company faces substantial competition from many different sources, including large and small pharmaceutical and biotechnology companies, academic research institutions, governmental agencies, and public and private institutions. Key competitors named in the filing include SK-Pharma, Xenon Pharmaceuticals, Biohaven Pharmaceuticals, Lundbeck, and Stoke Therapeutics for sodium channel blocker or similar ion channel-targeting programs in development for common epilepsies, as well as approved therapies including other existing ion channel blockers. For essential tremor, competitors include approved therapies such as propranolol and off-label therapies such as primidone. The company believes continuing to develop medicines that significantly advance the standard of care for patients is the optimal strategy to differentiate itself from competitors.

The company generates revenue through the development and potential commercialization of product candidates for CNS disorders, though it has no products approved for commercial sale and has not generated any revenue to date. The company's business model is centered on its two proprietary platforms, Cerebrum and Solidus, which are used to discover and develop first- and best-in-class CNS therapies. The company's strategy involves leveraging the genetics of epilepsy as a gateway for CNS drug discovery and development, utilizing translational tools to validate targets and product candidates, pursuing efficient clinical development paths to proof-of-concept in humans, and applying patient-guided development strategies. The company's primary customer segments are patients suffering from CNS disorders, including movement disorders and epilepsies.

The company has advanced four product candidates to clinical stage. Ulixacaltamide is a differentiated and highly selective small molecule inhibitor of T-type calcium channels in development for the treatment of essential tremor (ET). The FDA granted Breakthrough Therapy Designation for ulixacaltamide for the treatment of patients with ET based on positive topline data from the Essential3 program. The company submitted a New Drug Application (NDA) for ulixacaltamide for the treatment of ET to the FDA. Relutrigine is a first-in-class small molecule in development for the treatment of developmental and epileptic encephalopathies (DEEs) as a preferential inhibitor of persistent sodium current. Relutrigine has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for the treatment of SCN2A-DEE, SCN8A-DEE and Dravet Syndrome, as well as Breakthrough Therapy Designation from the FDA and Orphan Drug Designation from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE. The company has submitted an NDA for relutrigine for the treatment of SCN2A-DEE and SCN8A-DEE to the FDA. Vormatrigine is a next-generation, functionally selective small molecule targeting the hyperexcitable state of sodium-channels in the brain and is currently being developed as a once daily, oral treatment for adult focal onset seizures (FOS) and generalized epilepsy. Elsunersen is a clinical-stage ASO designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early seizure-onset SCN2A-DEE. Elsunersen has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, and Orphan Drug Designation and PRIME designations from the European Medicines Agency for the treatment of SCN2A-DEE.

The company has an exclusive collaboration and license agreement with Tenacia Biotechnology (Shanghai) Company, Ltd. to develop and commercialize ulixacaltamide for the treatment of ET in China, Hong Kong, Macau and Taiwan. The company has a Cooperation and License Agreement with RogCon, Inc. to collaborate to develop ASOs for the treatment of epilepsy caused by mutations of the SCN2A gene, under which RogCon is eligible to receive a one-time milestone payment of $3.0 million ¹ as well as profit share payments as a percentage of net profits in the mid-teens. The company also has a Research Collaboration, Option and License Agreement with Ionis Pharmaceuticals, Inc. to discover and develop ASOs to treat forms of epilepsy caused by mutations of the SCN2A gene, under which Ionis is eligible to receive certain contingent payments including development milestones of $5.0 million ² for each product developed under the agreement, an additional one-time milestone payment of $5.0 million ³ upon certain triggers, and interest payments equal to 10% ⁴ simple interest per annum on the Additional Milestone. In the second quarter of 2023, Ionis earned the Additional Milestone fee of $5.0 million ⁵, as well as the Initial Interest Amount of $1.9 million ⁶, and the $6.9 million ⁷ milestone fee was paid in July 2023. The company has conducted multiple public offerings, including a June 2023 public offering, a January 2024 follow-on offering, an April 2024 public offering, and an October 2025 public offering, each involving common stock and pre-funded warrants. As of February 16, 2026, the company had 168 ⁸ employees.

The company is a clinical-stage biopharmaceutical company that has incurred significant losses since its inception and has no products approved for commercial sale. Net losses were $303.3 million ⁹ and $182.8 million ¹⁰ for the years ended December 31, 2025 and 2024, respectively. As of December 31, 2025, the company had an accumulated deficit of $1.1 billion ¹¹. The company expects to continue to incur significant losses for the foreseeable future as it continues research and development of and seeks regulatory approvals for its product candidates and prepares for potential commercialization. The aggregate market value of common stock held by non-affiliates of the registrant as of June 30, 2025 was approximately $883.2 million ¹². As of February 16, 2026, the company had 27,850,833 ¹³ shares of common stock outstanding.

The company's primary growth vector is the advancement of its four clinical-stage product candidates through clinical development and regulatory approval. For ulixacaltamide, the company has submitted an NDA for the treatment of ET to the FDA and has commenced commercial preparations and pre-launch activities. For relutrigine, the company has submitted an NDA for the treatment of SCN2A-DEE and SCN8A-DEE to the FDA based on data from the EMBOLD study, and commercial preparations and pre-launch activities are underway. The company believes the EMERALD study evaluating relutrigine in patients with broad DEEs, if positive, could serve as the basis for a supplemental NDA submission in 2027 ¹⁴. For vormatrigine, the ENERGY program consists of five studies, with POWER1 expected to announce topline results in the second quarter of 2026 ¹⁵, POWER2 completion expected in the second half of 2026 ¹⁶ with topline results expected in 2027 ¹⁷, and if positive, the company plans to submit an NDA for vormatrigine in 2027 ¹⁸. The company intends to initiate POWER3, a clinical trial evaluating vormatrigine as a single-agent treatment for FOS, in the first half of 2026 ¹⁹. For elsunersen, the company expects to disclose topline results from the EMBRAVE Study Part A in the first half of 2026 ²⁰, and expects to announce topline results from the EMBRAVE3 Phase 3 study in 2027 ²¹. The company also has three novel ASOs with preclinical proof of mechanism that it expects to nominate development candidates for in the first half of 2026 ²² — PRAX-080 targeting PCDH19 mosaic expression disorders, PRAX-090 targeting SYNGAP1 loss-of-function mutations, and PRAX-100 targeting SCN2A loss-of-function mutations.

The company's second major growth vector is the expansion of its pipeline through its two proprietary platforms. Cerebrum has the potential to continue delivering first- and best-in-class orally available therapies for genetic CNS targets, and Solidus is uniquely positioned to continue discovering and advancing other new ASOs for novel genetic CNS targets. The company is applying genetic insights to the discovery and development of therapies for neurological disorders through these platforms, with significant potential for additional program and indication expansion. The company estimates there are approximately 200,000 ²³ patients in the United States living with a DEE, and up to 40% ²⁴ of DEEs are caused by single gene mutations, enabling precision medicine approaches. For essential tremor, the company notes it affects roughly seven million ²⁵ people in the United States alone, representing a multi-billion dollar commercial opportunity. For common epilepsies, an estimated 3.5 million ²⁶ people in the United States suffer from common epilepsies, with FOS being the most common type of epilepsy, accounting for approximately 60% ²⁷ of all cases.The company does not own or operate any manufacturing facilities and currently sources all non-clinical and clinical compound supply through third-party contract development and manufacturing organizations (CDMOs). The company expects to rely on third parties for manufacturing processes and the production of all clinical supply drug substance and drug product and currently expects to continue to do so for commercial supplies of its product candidates, if approved. The company uses additional contract manufacturers to fill, label, package, store and distribute its investigational drug products and currently expects to continue to do so for commercial supplies of its product candidates, if approved. It is the company's intent to identify and qualify additional manufacturers to provide active pharmaceutical ingredient and fill-and-finish services prior to submission of an NDA to the FDA for any product candidates that complete clinical development. As of February 16, 2026, the company has 168 ²⁸ employees and plans to continue to grow, with the majority of anticipated hiring focused on building commercial capabilities.

The company believes that its current cash, cash equivalents and marketable securities will be sufficient to fund its operating expenditures and capital expenditure requirements into 2028 ²⁹. The company expects its expenses to increase substantially as it continues to develop and conduct clinical trials, seeks regulatory approvals, prepares for potential commercialization, and expands its workforce. The company has no committed source of additional capital and if unable to raise additional capital when needed, could be forced to delay, reduce or eliminate its product discovery and development programs or commercialization efforts.

The company faces significant structural headwinds including the lengthy, complex and expensive nature of preclinical and clinical drug development with uncertain outcomes. The regulatory approval processes of the FDA and comparable foreign regulatory authorities are lengthy, time-consuming, and inherently unpredictable. The company may need additional funding and if unable to raise capital when needed, could be forced to delay, reduce or eliminate product discovery and development programs or commercialization efforts. The company also faces intense competition in an environment of rapid technological and scientific change, with the possibility that competitors may achieve regulatory approval before the company or develop therapies that are safer, more advanced or more effective. Additionally, the company's product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval, limit their commercial potential or result in significant negative consequences following regulatory approval, if obtained.

The company faces execution risks including difficulties enrolling patients in clinical trials, which could delay or otherwise adversely affect clinical development activities. The markets for the company's product candidates may be smaller than expected. The company depends on collaborations with third parties for the research, development and commercialization of certain product candidates, and if any such collaborations are not successful, the company may not be able to realize the market potential of those product candidates. The company also depends heavily on its executive officers, principal consultants and others, and the loss of their services could materially harm the business.

The company is a clinical-stage biopharmaceutical company with no products approved for commercial sale and has incurred significant losses since inception, with net losses of $303.3 million ³⁰ and $182.8 million ³¹ for the years ended December 31, 2025 and 2024, respectively, and an accumulated deficit of $1.1 billion ³² as of December 31, 2025. The company may need additional funding and if unable to raise capital when needed, could be forced to delay, reduce or eliminate product discovery and development programs or commercialization efforts. The development and commercialization of drug products is subject to extensive regulation, and the regulatory approval processes of the FDA and comparable foreign regulatory authorities are lengthy, time-consuming, and inherently unpredictable. The outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials, and the results of clinical trials may not satisfy the requirements of the FDA or comparable foreign regulatory authorities. The company faces significant competition in an environment of rapid technological and scientific change, and there is a possibility that competitors may achieve regulatory approval before the company or develop therapies that are safer, more advanced or more effective. The company's success depends in part on its ability to protect its intellectual property, and it is difficult and costly to protect proprietary rights and technology.

Management's message emphasizes that the company is a fully integrated, leading CNS precision neuroscience biopharmaceutical company translating insights from genetic epilepsies into the development of therapies for CNS disorders. The company has established a diversified, multimodal CNS portfolio with four clinical-stage product candidates across movement disorders and epilepsy. Management highlights positive topline results from the Essential3 program for ulixacaltamide, with Study 1 meeting its primary and all key secondary endpoints, and the registrational cohort of the EMBOLD study for relutrigine, which was stopped early for efficacy. Management also notes positive topline results from the RADIANT Phase 2 study for vormatrigine. The company has submitted NDAs for ulixacaltamide for the treatment of ET and for relutrigine for the treatment of SCN2A-DEE and SCN8A-DEE to the FDA, and commercial preparations and pre-launch activities are underway. Management's strategic priorities include advancing the clinical development programs for all four product candidates, preparing for potential commercialization of ulixacaltamide and relutrigine if approved, and continuing to discover and develop new product candidates through the Cerebrum and Solidus platforms.