Over 64,000 pounds of butter recalled over undeclared milk, FDA says
Food ingredient company Bunge North America recalled 1,800 cases of its NH European Style Butter Blend that were distributed at 12 centers located across the United States and one in Dominican Republic, a July 14 FDA alert reported.
The FDA classified the recall under its second-highest risk warning on July 31 warning that consumption could pose temporary or medically reversible adverse health consequences.
USA TODAY has reached out to Bunge North America for comment.
Based in Chesterfield, Missouri, Bunge North America is an agribusiness that produces and markets multiple food ingredients including corn, wheat, rice, soybeans and feed peas.
Which butter products are recalled?
The FDA has issued a recall for 64,800 pounds of Bunge North America's NH European Style Butter Blend with:
The product was packaged in white paperboard cases each containing 36 blocks.
This story has been updated to resolve an inaccuracy in the headline.
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3 hours ago
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Bristol Myers Squibb's Application for Breyanzi (lisocabtagene maraleucel) Accepted for Priority Review by U.S. Food and Drug Administration (FDA) in Fifth Cancer Type for Relapsed or Refractory Marginal Zone Lymphoma (MZL)
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental biologics license application (sBLA) for Breyanzi ® (lisocabtagene maraleucel; liso-cel) as a potential treatment for adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least two prior lines of systemic therapy. The FDA has granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 5, 2025. BMY announces an FDA acceptance of the regulatory filing with priority review for their personalized treatment option for adult patients with relapsed or refractory marginal zone lymphoma #MZL Share 'While initial therapy for MZL can be effective, multiple relapses over the course of several years are common, leaving patients in need of a new treatment option that can provide high, lasting response rates,' said Rosanna Ricafort, vice president, Senior Global Program Lead for Hematology and Cell Therapy, Bristol Myers Squibb. 'This FDA acceptance brings us one step closer to potentially standardizing CAR T cell therapy as a treatment option for MZL, while building on our commitment to bring this personalized therapy to as many eligible patients as possible.' The application is based on results from the primary analysis of the MZL cohort in TRANSCEND FL, an open-label, multicenter, Phase 2, single-arm study, which was shared in an oral presentation during the 2025 International Conference on Malignant Lymphoma (ICML) in June 2025. The development progress of Breyanzi reflects BMS' continued efforts to collaborate across the healthcare ecosystem, with the ultimate goal of reaching more patients and democratizing access to cell therapy. Recently, the FDA approved streamlined patient monitoring requirements and the removal of the REMS program for Breyanzi, easing known barriers to treatment and administration while maintaining patient safety. About TRANSCEND FL TRANSCEND FL (NCT04245839) is an open-label, global, multicenter, Phase 2, single-arm study to determine the efficacy and safety of Breyanzi in patients with relapsed or refractory indolent B-cell non-Hodgkin lymphoma, including follicular lymphoma and marginal zone lymphoma. The primary outcome measure is overall response rate. Secondary outcome measures include complete response rate, duration of response, and progression-free survival. About MZL Marginal zone lymphoma (MZL) is the third most common lymphoma, accounting for about 7% of all non-Hodgkin lymphoma cases. Most patients with MZL are at a median age of 67 years when they are diagnosed. MZL develops when white blood cells cluster together to form lumps in a person's lymph nodes or organs. Initial therapy often leads to remission, but relapse is common, sometimes occurring several times over many years. A small portion of MZL cases transform into diffuse large-B-cell lymphoma, a more aggressive lymphoma. About Breyanzi Breyanzi is a CD19-directed CAR T cell therapy with a 4-1BB costimulatory domain, which enhances the expansion and persistence of the CAR T cells. Breyanzi is made from a patient's own T cells, which are collected and genetically reengineered to become CAR T cells that are then delivered via infusion as a one-time treatment. The treatment process includes blood collection, CAR T-cell creation, potential bridging therapy, lymphodepletion, administration, and side-effect monitoring. Breyanzi is approved in the U.S. for the treatment of relapsed or refractory large B-cell lymphoma (LBCL) after at least one prior line of therapy, has received accelerated approval for the treatment of relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior lines of therapy and relapsed or refractory follicular lymphoma (FL) in the third-line plus setting, and is approved for the treatment of relapsed or refractory mantle cell lymphoma (MCL) in the third-line plus setting. Breyanzi is also approved in Japan, the European Union (EU), Switzerland, the United Kingdom, and Canada for the treatment of relapsed or refractory LBCL after at least one prior line of therapy; in Japan for the treatment of patients with relapsed or refractory high-risk FL after one prior line of systemic therapy, and in patients with relapsed or refractory FL after two or more lines of systemic therapy; and in the EU for the treatment of relapsed or refractory FL after two or more lines of systemic therapy. Bristol Myers Squibb's clinical development program for Breyanzi includes clinical studies in several types of lymphoma. For more information, visit Breyanzi U.S. FDA-Approved Indications BREYANZI is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of: adult patients with large B-cell lymphoma (LBCL), including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B, who have: refractory disease to first-line chemoimmunotherapy or relapse within 12 months of first-line chemoimmunotherapy; or refractory disease to first-line chemoimmunotherapy or relapse after first-line chemoimmunotherapy and are not eligible for hematopoietic stem cell transplantation (HSCT) due to comorbidities or age; or relapsed or refractory disease after two or more lines of systemic therapy. Limitations of Use: BREYANZI is not indicated for the treatment of patients with primary central nervous system lymphoma. adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least 2 prior lines of therapy, including a Bruton tyrosine kinase (BTK) inhibitor and a B-cell lymphoma 2 (BCL-2) inhibitor. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). adult patients with relapsed or refractory follicular lymphoma (FL) who have received 2 or more prior lines of systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have received at least 2 prior lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor. Breyanzi U.S. Important Safety Information WARNING: CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES, AND SECONDARY HEMATOLOGICAL MALIGNANCIES Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients receiving BREYANZI. Do not administer BREYANZI to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab with or without corticosteroids. Neurologic toxicities, including fatal or life-threatening reactions, occurred in patients receiving BREYANZI, including concurrently with CRS, after CRS resolution, or in the absence of CRS. Monitor for neurologic events after treatment with BREYANZI. Provide supportive care and/or corticosteroids as needed. T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including BREYANZI. Cytokine Release Syndrome Cytokine release syndrome (CRS), including fatal or life-threatening reactions, occurred following treatment with BREYANZI. In clinical trials of BREYANZI, which enrolled a total of 702 patients with non-Hodgkin lymphoma (NHL), CRS occurred in 54% of patients, including ≥ Grade 3 CRS in 3.2% of patients. The median time to onset was 5 days (range: 1 to 63 days). CRS resolved in 98% of patients with a median duration of 5 days (range: 1 to 37 days). One patient had fatal CRS and 5 patients had ongoing CRS at the time of death. The most common manifestations of CRS (≥10%) were fever, hypotension, tachycardia, chills, hypoxia, and headache. Serious events that may be associated with CRS include cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), cardiac arrest, cardiac failure, diffuse alveolar damage, renal insufficiency, capillary leak syndrome, hypotension, hypoxia, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS). Ensure that 2 doses of tocilizumab are available prior to infusion of BREYANZI. Neurologic Toxicities Neurologic toxicities that were fatal or life-threatening, including immune effector cell-associated neurotoxicity syndrome (ICANS), occurred following treatment with BREYANZI. Serious events including cerebral edema and seizures occurred with BREYANZI. Fatal and serious cases of leukoencephalopathy, some attributable to fludarabine, also occurred. In clinical trials of BREYANZI, CAR T cell-associated neurologic toxicities occurred in 31% of patients, including ≥ Grade 3 cases in 10% of patients. The median time to onset of neurotoxicity was 8 days (range: 1 to 63 days). Neurologic toxicities resolved in 88% of patients with a median duration of 7 days (range: 1 to 119 days). Of patients developing neurotoxicity, 82% also developed CRS. The most common neurologic toxicities (≥5%) included encephalopathy, tremor, aphasia, headache, dizziness, and delirium. CRS and Neurologic Toxicities Monitoring Monitor patients daily for at least 7 days following BREYANZI infusion for signs and symptoms of CRS and neurologic toxicities and assess for other causes of neurological symptoms. Continue to monitor patients for signs and symptoms of CRS and neurologic toxicities for at least 2 weeks after infusion and treat promptly. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated. Manage neurologic toxicity with supportive care and/or corticosteroid as needed. Advise patients to avoid driving for at least 2 weeks following infusion. Counsel patients to seek immediate medical attention should signs or symptoms of CRS or neurologic toxicity occur at any time. Hypersensitivity Reactions Allergic reactions may occur with the infusion of BREYANZI. Serious hypersensitivity reactions, including anaphylaxis, may be due to dimethyl sulfoxide (DMSO). Serious Infections Severe infections, including life-threatening or fatal infections, have occurred in patients after BREYANZI infusion. In clinical trials of BREYANZI, infections of any grade occurred in 34% of patients, with Grade 3 or higher infections occurring in 12% of all patients. Grade 3 or higher infections with an unspecified pathogen occurred in 7%, bacterial infections in 3.7%, viral infections in 2%, and fungal infections in 0.7% of patients. One patient who received 4 prior lines of therapy developed a fatal case of John Cunningham (JC) virus progressive multifocal leukoencephalopathy 4 months after treatment with BREYANZI. One patient who received 3 prior lines of therapy developed a fatal case of cryptococcal meningoencephalitis 35 days after treatment with BREYANZI. Febrile neutropenia developed after BREYANZI infusion in 8% of patients. Febrile neutropenia may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad- spectrum antibiotics, fluids, and other supportive care as medically indicated. Monitor patients for signs and symptoms of infection before and after BREYANZI administration and treat appropriately. Administer prophylactic antimicrobials according to standard institutional guidelines. Avoid administration of BREYANZI in patients with clinically significant, active systemic infections. Viral reactivation: Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against B cells. In clinical trials of BREYANZI, 35 of 38 patients with a prior history of HBV were treated with concurrent antiviral suppressive therapy. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing. In patients with prior history of HBV, consider concurrent antiviral suppressive therapy to prevent HBV reactivation per standard guidelines. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing. In patients with prior history of HBV, consider concurrent antiviral suppressive therapy to prevent HBV reactivation per standard guidelines. Prolonged Cytopenias Patients may exhibit cytopenias not resolved for several weeks following lymphodepleting chemotherapy and BREYANZI infusion. In clinical trials of BREYANZI, Grade 3 or higher cytopenias persisted at Day 29 following BREYANZI infusion in 35% of patients, and included thrombocytopenia in 25%, neutropenia in 22%, and anemia in 6% of patients. Monitor complete blood counts prior to and after BREYANZI administration. Hypogammaglobulinemia B-cell aplasia and hypogammaglobulinemia can occur in patients receiving BREYANZI. In clinical trials of BREYANZI, hypogammaglobulinemia was reported as an adverse reaction in 10% of patients. Hypogammaglobulinemia, either as an adverse reaction or laboratory IgG level below 500 mg/dL after infusion, was reported in 30% of patients. Monitor immunoglobulin levels after treatment with BREYANZI and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement as clinically indicated. Live vaccines: The safety of immunization with live viral vaccines during or following BREYANZI treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during BREYANZI treatment, and until immune recovery following treatment with BREYANZI. Secondary Malignancies Patients treated with BREYANZI may develop secondary malignancies. T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including BREYANZI. Mature T cell malignancies, including CAR-positive tumors, may present as soon as weeks following infusion, and may include fatal outcomes. Monitor lifelong for secondary malignancies. In the event that a secondary malignancy occurs, contact Bristol Myers Squibb at 1-888-805-4555 for reporting and to obtain instructions on collection of patient samples for testing. Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome (IEC-HS) Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome (IEC-HS), including fatal or life-threatening reactions, occurred following treatment with BREYANZI. Three of 89 (3%) safety evaluable patients with R/R CLL/SLL developed IEC-HS. Time to onset of IEC-HS ranged from 7 to 18 days. Two of the 3 patients developed IEC-HS in the setting of ongoing CRS and 1 in the setting of ongoing neurotoxicity. IEC-HS was fatal in 2 of 3 patients. One patient had fatal IEC-HS and one had ongoing IEC-HS at time of death. IEC-HS is a life-threatening condition with a high mortality rate if not recognized and treated early. Treatment of IEC-HS should be administered per current practice guidelines. Adverse Reactions The most common adverse reaction(s) (incidence ≥30%) in: LBCL are fever, cytokine release syndrome, fatigue, musculoskeletal pain, and nausea. The most common Grade 3-4 laboratory abnormalities include lymphocyte count decrease, neutrophil count decrease, platelet count decrease, and hemoglobin decrease. CLL/SLL are cytokine release syndrome, encephalopathy, fatigue, musculoskeletal pain, nausea, edema, and diarrhea. The most common Grade 3-4 laboratory abnormalities include neutrophil count decrease, white blood cell decrease, hemoglobin decrease, platelet count decrease, and lymphocyte count decrease. FL is cytokine release syndrome. The most common Grade 3-4 laboratory abnormalities include lymphocyte count decrease, neutrophil count decrease, and white blood cell decrease. MCL are cytokine release syndrome, fatigue, musculoskeletal pain, and encephalopathy. The most common Grade 3-4 laboratory abnormalities include neutrophil count decrease, white blood cell decrease, and platelet count decrease. Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide. Bristol Myers Squibb: Unlocking the Full Potential of Cell Therapy A pioneer in harnessing the immune system to fight cancer and an established leader in cell therapy, Bristol Myers Squibb is uniquely positioned to unlock the full potential of this technology across blood cancers and within new frontiers, including autoimmune disease. Bristol Myers Squibb is currently the only company with two approved CAR T cell therapies with two distinct targets, available in major markets around the world. Our bold vision for the future is one in which hundreds of thousands of patients can be treated with cell therapy's transformational potential. The building blocks to realize this ambition—a promising and differentiated pipeline, extensive translational and clinical data sets, a deep bench of talent, and robust manufacturing capabilities—are in our cells. We are laser-focused on advancing the field of cell therapy toward a true revolution for patients. Learn more about the science behind cell therapy and ongoing progress at Bristol Myers Squibb here. Cautionary Statement Regarding Forward-Looking Statements This press release contains 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, that Breyanzi (lisocabtagene maraleucel) may not receive regulatory approval for the additional indication described in this release in the currently anticipated timeline or at all, any marketing approvals, if granted, may have significant limitations on their use, and, if approved, whether Breyanzi for such indication will be commercially successful. No forward-looking statement can be guaranteed. It should also be noted that acceptance of the sBLA does not change the standards for FDA approval. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb's business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol Myers Squibb's Annual Report on Form 10-K for the year ended December 31, 2024, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, Bristol Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.
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Ultragenyx Pharmaceutical Inc. (RARE): A Bull Case Theory
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Praxis Precision Medicines Provides Corporate Update and Reports Second Quarter 2025 Financial Results
RADIANT study with vormatrigine in focal onset seizure (FOS) patients over eight weeks demonstrated 56.3% median reduction in seizure frequency from baseline, with 22% at 100% seizure reduction in last 28 days Initiated two registrational studies for Developmental and Epileptic Encephalopathies (DEEs) programs: EMERALD for broad DEEs with relutrigine and EMBRAVE3 for SCN2A Gain-of-Function (GoF) with elsunersen Relutrigine granted U.S. FDA Breakthrough Therapy Designation for the treatment of seizures associated with SCN2A and SCN8A DEEs, enabling expedited development Cash and investments of approximately $447 million as of June 30, 2025 maintains runway into 2028 Praxis to host a conference call today, August 4, at 8:30am to discuss Phase 2 RADIANT study results and provide a corporate update BOSTON, Aug. 04, 2025 (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 second quarter 2025. 'In the second quarter, we continued to make remarkable progress across our portfolio and believe we are positioned to revolutionize treatment in both common and rare epilepsy. Earlier today we reported the positive results from the RADIANT study, where vormatrigine has shown an impressive 56.3% reduction in seizures in 8 weeks in a heavily pre-treated population. Enrollment is going well for the POWER1 pivotal study for vormatrigine, and we plan to shortly initiate POWER2 as well as the POWER3 study investigating vormatrigine as a standalone agent. Our relutrigine program continues to progress strongly, first with the breakthrough therapy designation for SCN2A and SCN8A DEEs, which we expect will allow us to expedite the regulatory process, and second with the initiation of the EMERALD registrational study in broad DEEs. Completing our progress across epilepsy, we also initiated the registrational EMBRAVE3 study of elsunersen in SCN2A patients. We remain well-capitalized as we look ahead to an exciting second half of 2025,' said Marcio Souza, president and chief executive officer of Praxis. Recent Highlights and Anticipated MilestonesVormatrigine for FOS and Generalized Epilepsy: Vormatrigine is the most potent sodium-channel modulator ever designed to precisely target the hyperexcitable state of sodium-channels in adult common epilepsies. Earlier today Praxis shared positive results of the RADIANT study evaluating patients with FOS. Dosing with vormatrigine over 8 weeks led to 56.3% median reduction in seizure frequency. Approximately 22% of patients reached 100% reduction in seizure frequency in the last 28 days. Rapid and sustained response, with approximately 60% of patients achieving 50% response in the study. Vormatrigine was generally well tolerated and continues to demonstrate a favorable safety profile. Additional data are expected to be presented at the 36th International Epilepsy Congress on August 31, 2025, in Lisbon, Portugal. Praxis has also submitted a late-breaker abstract to present the full study results at the American Epilepsy Society Annual Meeting in December 2025 in Atlanta, Georgia. The POWER1 Phase 2/3 registrational study for FOS is enrolling well, and is on track to complete in the fourth quarter of 2025. The POWER2 Phase 2/3 registrational study for FOS trial design is completed based on data from RADIANT, and is expected to initiate in the third quarter of 2025 and complete enrollment in the second half of 2026. The POWER3 study to evaluate vormatrigine as a standalone agent is expected to initiate in the first half of 2026. The overall ENERGY program recruitment initiative, including the EMPOWER study, has attracted approximately 20,000 patients with epilepsy and continues to support patient identification for the epilepsy studies. Relutrigine for DEEs: Relutrigine is Praxis' second sodium channel modulator designed to precisely target the hyperexcitable state of sodium-channels, with therapeutic potential across developmental epilepsies. In July 2025, relutrigine received FDA Breakthrough Therapy Designation (BTD) for the treatment of seizures associated with SCN2A and SCN8A DEEs, potentially expediting its development. In May 2025, Praxis provided an update on the EMBOLD cohort 1 study group in SCN2A and SCN8A DEEs, showing patients in the open-label extension up to 11 months had a ~90% reduction in seizures from their baseline, and the mean period between seizures increased from three days at baseline to 67 days at month 11. The EMBOLD registrational cohort 2 in SCN2A and SCN8A DEEs continues to enroll well, with topline results expected no later than the first half of 2026, followed by a potential NDA submission. The EMERALD registrational study in broad DEEs has been initiated and is planned to include up to 160 patients, with enrollment expected to complete in 2026. Ulixacaltamide for Essential Tremor (ET): Essential Tremor is the largest movement disorder affecting approximately seven million people in the U.S. During the recruitment phase of the trial, started in November 2023, over 200,000 patients demonstrated interest in participating in the study. Praxis has completed enrollment of both Phase 3 studies in the Essential 3 program, with topline results expected in early fall of 2025. After reviewing the results of both studies, Praxis will determine if there is sufficient evidence to support an NDA for early-seizure-onset SCN2A DEE: SCN2A GoF-DEE is a rare, genetic epilepsy characterized by early-onset seizures and severe impact on development. Elsunersen is currently being evaluated in two registrational studies: The EMBRAVE Part A Phase 1/2 study is continuing to enroll up to 16 patients. Patients are randomized 3:1 drug to sham for a six-month period on a once-monthly dose, with the potential to escalate from 1 mg to 8 mg. Topline results are expected in the first half of 2026. The EMBRAVE3 registrational study for SCN2A GoF-DEE has been initiated. Cohort 1, aiming to enroll up to 40 patients ages 2 to 18 years, has started enrolling patients. In cohort 1, patients will be randomized 1:1 to receive 1 mg of elsunersen or sham once monthly for a six-month period. The primary endpoint assesses median percent change in seizure frequency from baseline. Cohorts 2 and 3 will evaluate patients from ages 1 to 2 and 0 to 1 years, respectively. Praxis remains on track to nominate a development candidate for each of its early stage ASO therapeutic initiatives in 2025: PRAX-080: Focused on targeting PCDH19 mosaic expression disorder, nominated by end of year. PRAX-090: Designed to address SYNGAP1 loss-of-function (LoF) mutations, a leading cause of severe intellectual disability and epilepsy in DEEs, nominated by end of year. PRAX-100: Targeting SCN2A LoF mutations, the predominant genetic link to de novo autism spectrum disorders, nominated mid-year. Second Quarter 2025 Financial Results: As of June 30, 2025, Praxis had $446.6 million in cash, cash equivalents and marketable securities, compared to $469.5 million in cash, cash equivalents and marketable securities as of December 31, 2024. The decrease of $22.9 million is primarily attributable to cash used in operating activities partially offset by net proceeds from at-the-market offerings of common stock. The Company's cash, cash equivalents and marketable securities as of June 30, 2025 are expected to fund operations into 2028. Praxis did not recognize any collaboration revenue during the three months ended June 30, 2025, compared to $0.4 million during the three months ended June 30, 2024. The decrease of $0.4 million is related to its Option and License Agreement with UCB. In December 2024, UCB exercised its option to in-license global development and commercialization rights for a KCNT1 small molecule development candidate, and as such, Praxis has no further research service obligations under the terms of the Option and License Agreement. Research and development expenses were $63.0 million for the three months ended June 30, 2025, compared to $27.3 million for the three months ended June 30, 2024. The increase in research and development expenses of $35.7 million was primarily attributable to an increase of $32.0 million in Praxis' Cerebrum™ platform, an increase of $3.0 million in personnel-related costs, an increase of $0.4 million in indirect expenses and an increase of $0.3 million in Praxis' Solidus™ platform. General and administrative expenses were $13.1 million for the three months ended June 30, 2025, compared to $10.6 million for the three months ended June 30, 2024. The increase in general and administrative expenses of approximately $2.5 million was primarily attributable to an increase of $1.3 million in indirect expenses, and an increase of $0.8 million in personnel-related costs. Praxis reported a net loss of $71.1 million for the three months ended June 30, 2025, including $7.8 million of stock-based compensation expense, compared to $32.7 million for the three months ended June 30, 2024, including $5.9 million of stock-based compensation. As of June 30, 2025, Praxis had 21.0 million shares of common stock outstanding. Conference CallPraxis will discuss the study results, as well as its second quarter 2025 financial results and business highlights on a conference call taking place today, August 4 at 8:30 am ET. Individuals may register for the conference call by clicking the registration link. Once registered, participants will receive dial-in details and a unique PIN which will allow them to access the call. An audio webcast will be accessible through the Events & Presentation page under the Investor Relations section of the Company's website. About Vormatrigine (PRAX-628)Vormatrigine is a next-generation, functionally selective small molecule targeting the hyperexcitable state of sodium-channels in the brain that is currently being developed as a once daily, oral treatment for adult focal onset seizures and generalized epilepsy. Preclinical data demonstrates vormatrigine is differentiated from standard of care, with the potential to be best-in-class for focal epilepsy. In vitro, vormatrigine has demonstrated superior selectivity for disease-state NaV channel hyperexcitability. In vivo studies of vormatrigine have demonstrated unprecedented potency in the maximal electroshock seizure (MES) model, a highly predictive translational model for efficacy in focal epilepsy. Data from the first cohort of patients in the RADIANT study demonstrated a robust seizure reduction and generally safe and well tolerated profile. To learn more about the POWER1 study, please visit POWER1 study. About Relutrigine (PRAX-562)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, shown to be a key driver of seizure symptoms in severe DEEs. Relutrigine's mechanism of precision sodium channel (NaV) modulation is consistent with superior selectivity for disease-state NaV channel hyperexcitability. In vivo studies of relutrigine have demonstrated dose-dependent inhibition of seizures up to complete control of seizure activity in SCN2A, SCN8A and other DEE mouse models. Relutrigine has been generally well-tolerated in three Phase 1 studies and has demonstrated biomarker changes indicative of NaV channel modulation. Data from cohort 1 of the Phase 2 EMBOLD study demonstrated a well-tolerated, robust, short- and long-term improvement in motor seizures in a heavily pre-treated population, alongside maintained seizure freedom in some patients with SCN2A- and SCN8A-DEE. Relutrigine has received Orphan Drug Designation (ODD) 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 (BTD), and ODD from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE. To learn more about the EMERALD and EMBOLD studies, please visit About UlixacaltamideUlixacaltamide is a differentiated and highly selective small molecule inhibitor of T-type calcium channels designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit correlated with tremor activity. Ulixacaltamide, the most advanced program within Praxis' Cerebrum™ small molecule platform, is currently in late-stage development for the treatment of essential tremor. About Elsunersen (PRAX-222)Elsunersen is an antisense oligonucleotide (ASO) designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early-seizure-onset SCN2A-DEE to treat seizures and other symptoms in patients with gain-of-function SCN2A mutations. In vitro studies of elsunersen have demonstrated reduction in both SCN2A gene expression and protein levels. In vivo, elsunersen has demonstrated significant, dose-dependent reduction in seizures, improvement in behavioral and locomotor activity and increased survival in SCN2A mouse models, with potential to be the first disease-modifying treatment for SCN2A-DEE. Elsunersen has received ODD and RPDD from the FDA, and ODD and PRIME designations from the European Medicines Agency for the treatment of SCN2A-DEE. The elsunersen program is ongoing under a collaboration with Ionis Pharmaceuticals, Inc., and RogCon, Inc. To learn more about the EMBRAVE study, please visit 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 epilepsy and movement disorders, with four clinical-stage product candidates. For more information, please visit and follow us on Facebook, LinkedIn and Twitter/X. Forward-Looking StatementsThis 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 the anticipated timing of our clinical trials, the development of our product candidates and plans to initiate new clinical programs, the anticipated timing of regulatory submissions and interactions and our projected cash runway, 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; preliminary analyses from ongoing studies differing materially from final data from preclinical studies and completed clinical trials; the expected timing of clinical trials, data readouts and the results thereof, and submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials; and other risks concerning Praxis' programs and operations as described in its Annual Report on Form 10-K for the year ended December 31, 2024 and as updated in the Quarterly Report on Form 10-Q for the period ended June 30, 2025, as well as 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, CONSOLIDATED BALANCE SHEETS(Amounts in thousands)(Unaudited) June 30, 2025 December 31,2024 Assets Cash and cash equivalents $ 157,415 $ 215,372 Marketable securities 289,229 254,156 Prepaid expenses and other current assets 5,304 11,805 Property and equipment, net 258 230 Operating lease right-of-use assets 626 1,131 Other non-current assets — 416 Total assets $ 452,832 $ 483,110 Liabilities and stockholders' equity Accounts payable $ 28,832 $ 12,528 Accrued expenses 19,021 23,763 Operating lease liabilities 755 1,369 Common stock 14 14 Additional paid-in capital 1,380,978 1,281,522 Accumulated other comprehensive gain 395 654 Accumulated deficit (977,163 ) (836,740 ) Total liabilities and stockholders' equity $ 452,832 $ 483,110 PRAXIS PRECISION MEDICINES, CONSOLIDATED STATEMENTS OF OPERATIONS(Amounts in thousands, except share and per share amounts)(Unaudited) Three Months Ends June 30, Six Months Ended June 30, 2025 2024 2025 2024 Collaboration revenue $ — $ 357 $ — $ 788 Operating expenses: Research and development 63,006 27,260 123,812 54,244 General and administrative 13,061 10,585 26,983 25,918 Total operating expenses 76,067 37,845 150,795 80,162 Loss from operations (76,067 ) (37,488 ) (150,795 ) (79,374 ) Other income: Other income, net 4,940 4,811 10,372 7,144 Total other income 4,940 4,811 10,372 7,144 Net loss $ (71,127 ) $ (32,677 ) $ (140,423 ) $ (72,230 ) Net loss per share attributable tocommon stockholders, basic and diluted $ (3.31 ) $ (1.74 ) $ (6.60 ) $ (4.41 ) Weighted average common sharesoutstanding, basic and diluted 21,474,827 18,824,479 21,266,490 16,364,421 CONTACT: Investor Contact: Praxis Precision Medicines investors@ 857-702-9452 Media Contact: Dan Ferry Life Science Advisors Daniel@ 617-430-7576Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
