Latest news with #MPSIIIB
Business Wire
4 days ago
- Health
- Business Wire
Spruce Biosciences Announces Integrated Long-Term Clinical Data of Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) Demonstrating Profound and Durable Efficacy and Safety in Patients with Sanfilippo Syndrome Type B (MPS IIIB)
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Spruce Biosciences, Inc. (OTCQB: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced results from a long-term data integration of tralesinidase alfa enzyme replacement therapy (TA-ERT) clinical program in patients with Sanfilippo Syndrome Type B (MPS IIIB). Spruce integrated and evaluated group-level efficacy data for cerebral spinal fluid heparan-sulfate non-reducing end (CSF HS-NRE), cortical grey matter volume (CGMV), and Bayley-III Cognitive Raw Score (BSID-C), the cognitive subscale of the Bayley Scales of Infant and Toddler Development - Third Edition (Bayley-III), as well as safety data over a five-year period from clinical studies 201, 202, and 401. Data from treated patients (n=22) in the studies 201, 202, and 401 was compared with data from untreated patients with MPS IIIB in natural history studies 901 and 902. 'These data demonstrate a rapid, profound, and durable effect of ICV-administered TA-ERT on normalizing CSF HS-NRE, the pathogenic factor leading to neurodegeneration, and in stabilizing CGMV and cognitive function, relative to the declines observed in untreated children with Sanfilippo Syndrome Type B (MPS IIIB) in the natural history studies,' said Paul Harmatz, M.D., Principal Investigator in studies 901, 201, and 202 and Professor in Residence, Department of Pediatrics, University of California, San Francisco (UCSF) and UCSF Benioff Children's Hospital Oakland. 'This is an important development toward relief for children and families whose lives are impacted by this devastating condition.' 'Convincing positive outcomes have been experienced by families who resoundingly state that the benefits of TA-ERT outweigh risks in their real-world experiences,' said Cara O'Neill, M.D., FAAP, Co-Founder and Chief Science Officer of Cure Sanfilippo Foundation. 'Biomarker data, along with promising clinical outcomes, are significant and meaningful from the perspective of the patient community. Currently, there is no U.S. FDA-approved therapy for the treatment of MPS IIIB, and disease management consists of limited palliative care. We are eager to see TA-ERT advance under the accelerated approval pathway.' Cerebral Spinal Fluid Heparan-Sulfate Non-Reducing End (CSF HS-NRE) Integrated group-level data from clinical studies 201, 202, and 401 demonstrate that TA-ERT therapy significantly reduced to normal or near normal CSF HS-NRE levels over a five-year period (Figure 1). At 240 weeks, CSF HS-NRE decreased 91.5 ng/mL from baseline (95% CI: -102.10, -80.90; p<0.0001). Most participants experienced normalization of CSF HS-NRE levels eight weeks after initiating therapy. In a 2024 meeting with the U.S. Food and Drug Administration (FDA), the FDA confirmed that CSF HS-NRE is a surrogate biomarker reasonably likely to predict clinical benefit and could serve as a basis for accelerated approval. Cognitive Function In untreated patients with MPS IIIB, cognitive function peaks at around four years of age and then declines over time. In contrast, children with established disease treated with TA-ERT experienced stable cognitive function over time (Figure 2). Untreated children in the natural history studies showed a decline in cognition beginning at approximately five years of age that progressively worsened over time, while cognition in the TA-ERT treated group remained stable. Using a model-based approach, the mean (95% CI) BSID-C over six to 10 years of age was significantly higher in patients treated with TA-ERT, relative to untreated, age-matched children, with differences evident at six years of age (group difference: 10.67, 95% CI: 3.23, 18.11; p=0.005). At 10 years of age, the difference in BSID-C scores between groups increased to 34.66 (95% CI: 24.38, 44.93; p<0.0001). Although TA-ERT treatment stabilized BSID-C scores on average, increases in BSID-C scores were more commonly observed in subjects who initiated therapy at younger ages with higher baseline cognitive function and prior to the establishment of meaningful neurodegeneration. The BSID-C is anticipated to be the primary endpoint for the post-marketing clinical trial. Cortical Grey Matter Volume (CGMV) TA-ERT therapy was also associated with stabilization of CGMV, relative to the decline in CGMV observed in untreated children due to the progressive neurodegenerative nature of MPS IIIB (Figure 3). While CGMV should increase with age in children up to five years of age, there was an average loss of ~32 mL over 48 weeks in untreated children with MPS IIIB observed in study 901. Consistent with TA-ERT's mechanism of action, decreases in CGMV were observed during the initial 24 weeks of TA-ERT treatment, likely reflecting intracellular clearance of cerebral spinal fluid heparan sulfate (CSF HS) and CSF HS-NRE. CGMV stabilized from weeks 48 to 240 with TA-ERT treatment. Safety TA-ERT therapy exposure for up to 7.3 years has demonstrated an adequate safety profile in a serious and fatal disease for which no treatment is currently available. The mean (SD) exposure to TA-ERT was 4.2 (2.0) years. No deaths occurred throughout study 201 and its long-term extension studies 202 and 401. The most frequent treatment-emergent adverse event (TEAE) by preferred term (reported in ≥40% of participants) was vomiting (22 [100%]), followed by pyrexia (20 [90.9%]), upper respiratory tract infection (17 [77.3%]), pleocytosis (11 [50.0%]), COVID-19 infection (10 [45.5%]), and diarrhea (9 [40.9%]). Four (18%) patients discontinued treatment, although three (14%) discontinuations were due to hydrocephalus, a known complication of MPS IIIB. Adverse events related to the ICV device were consistent with other therapies administered by the ICV route. About Sanfilippo Syndrome Type B (MPS IIIB) MPS IIIB is an ultra-rare, serious, and fatal genetic disease characterized by deficiency in N-Acetyl-Alpha-Glycosaminidase (NAGLU), an enzyme required for the catabolism of heparan sulfate (HS) in lysosomes. It is estimated that MPS IIIB affects fewer than 1:200,000 people in the United States (U.S.), but the true incidence and prevalence are difficult to ascertain because MPS IIIB is a disease currently not included in newborn screening. The accumulation of toxic levels of cerebral spinal fluid heparan sulfate in the brain is the underlying pathophysiology of MPS IIIB. Although signs and symptoms of MPS IIIB can vary amongst affected individuals, progressive neurodegeneration typically follows a predictable path to brain atrophy, cognitive and developmental impairment, hyperactivity with aggressive and destructive behavior, delayed speech, hearing loss, and motor skill deficits. Somatic manifestations include coarse facial features, hepatosplenomegaly, and gastrointestinal symptoms. The final stage of MPS IIIB is typically marked by severe dementia, loss of motor function, and seizure activity, with patients largely bed-ridden and requiring constant care, requiring feeding tubes for hydration and nutrition, and ultimately leading to death. The estimated life expectancy of individuals with MPS IIIB ranges from 15 to 19 years of age. Currently, there are no FDA-approved therapies for MPS IIIB, and management of the disease consists of limited palliative care to improve quality of life. About Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) is a fusion protein comprised of recombinant human alpha-N-acetylglucosaminidase (rhNAGLU). TA-ERT is intended as an enzyme replacement therapy for the treatment of patients with MPS IIIB (Sanfilippo Syndrome Type B) who lack rhNAGLU enzyme activity. TA-ERT is expected to restore rhNAGLU enzyme activity in the central nervous system following intracerebroventricular injection. rhNAGLU typically lacks the mannose-6-phosphate residues that are essential for efficient cellular uptake via the M6P receptor pathway. As a result, the naked enzyme is poorly absorbed by cells, including neurons. To address this challenge, TA-ERT is fused to an insulin-like growth factor 2 peptide, which binds to the cation-independent mannose-6-phosphate on cell surfaces. This fusion enables the enzyme to be internalized and delivered to the lysosome, thereby enhancing its therapeutic potential for treating MPS IIIB. By restoring NAGLU enzymatic activity and promoting clearance of lysosomal heparan sulfate and heparan sulfate non-reducing end in the brain, TA-ERT therapy is expected to preserve neuronal cell health and potentially halt or slow the neurological decline and improve clinical outcomes in affected patients. TA-ERT has been evaluated in three clinical studies in participants with MPS IIIB: the interventional study 201 and extension studies 202 and 401. Twenty-two individuals with MPS IIIB have been administered TA-ERT therapy. TA-ERT has demonstrated an adequate safety profile based on integrated five years of safety data. About Spruce Biosciences Spruce Biosciences is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need. To learn more, visit and follow us on X, LinkedIn, Facebook and YouTube. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the ability to seek accelerated approval of TA-ERT for MPS IIIB based on existing clinical data. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as 'anticipate', 'will', 'potential', 'intend', 'expect' and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Spruce's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Spruce's business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Spruce's filings with the U.S. Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Spruce undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
Yahoo
24-06-2025
- Business
- Yahoo
Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by European Commission (EC)
TOKYO & HYOGO, Japan, June 24, 2025--(BUSINESS WIRE)--MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the European Commission (EC) has granted orphan drug designation (ODD) to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B), following the recent the U.S. Food and Drug Administration (FDA) designation.1 MPS IIIB affects an estimated 500 to 1,000 individuals worldwide,2 causing severe central nervous system (CNS) symptoms. Despite the dire need, there are currently no approved treatments available for this condition. JR-446, developed using JCR's proprietary J-Brain Cargo® technology, has shown promising non-clinical results in addressing the CNS symptoms of this challenging disorder, and it is currently being studied in a Phase I/II trial that is being conducted in Japan (JR-446-101) under a collaboration agreement between the two companies. In September 2023, MEDIPAL and JCR entered into a licensing agreement in which MEDIPAL will commercialize JR-446 for MPS IIIB outside of Japan. In addition, MEDIPAL will support JCR in the clinical development of JR-446 in Japan, including the distribution of investigational drugs, disease awareness, and clinical trial advancement.3 With the ODD, JR-446 will be eligible for various incentives to encourage the development in the European Union (EU). About Orphan Drug Designation in the European UnionThe European Commission implements orphan designation drug for promoting new drug development for rare diseases in which the prevalence of the condition affects no more than five in 10,000 people in the European Union (EU). Designated drugs are granted market exclusivity for 10 years in the EU, as well as scientific guidance. Fee reductions are also available depending on the status of the sponsor and the type of service required. About Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)Mucopolysaccharidosis type IIIB, or Sanfilippo syndrome type B, is an autosomal recessive disease caused by pathogenic mutations in the NAGLU gene, encoding a lysosomal enzyme involved in the degradation of heparan sulfate. With the accumulation of heparan sulfate in the central nervous system in the brain, individuals with this condition present rapid neurological decline, including sleep disorders, loss of speech, and behavioral changes, which may significantly affect the quality of life of patients and their families. About the J-Brain Cargo® Platform TechnologyJCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology J- Brain Cargo®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology is IZCARGO® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder. About MEDIPAL HOLDINGS CORPORATIONMEDIPAL is a holding company which controls, administers and supports the operating activities of companies in which it holds shares in the Prescription Pharmaceutical Wholesale Business; the Cosmetics, Daily Necessities and OTC Pharmaceutical Wholesale Business; and the Animal Health Products and Food Processing Raw Materials Wholesale and Related Business, and conducts business development for the MEDIPAL Group. For more information, visit About JCR Pharmaceuticals Co., is a global specialty pharmaceuticals company dedicated to advancing treatments for rare and genetic diseases. With nearly 50 years of expertise in Japan, JCR is expanding to the US, Europe, and Latin America. JCR's innovative therapies address conditions like growth disorder, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. JCR is also developing treatments for rare diseases like MPS I, MPS II, MPS IIIA and B, and more. For more information, visit Cautionary Statement Regarding Forward-Looking StatementsThis document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as "believe," "estimate," "anticipate," "intend," "plan," "will," "would," "target" and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors' pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future. References Reference: Press release on the orphan drug designation for JR-446 for Mucopolysaccharidosis Type IIIB by the U.S. FDA (May 7, 2025). Based on data from JCR's own investigations, referring to the Ministry of Health, Labour and Welfare's public research. Reference: Press release on the licensing agreement for JR-446 between MEDIPAL and JCR (September 28, 2023). View source version on Contacts MEDIPAL HOLDINGS CORPORATIONPublic Relations DepartmentTEL: (+81)-3-3517-5171JCR Pharmaceuticals Co., CommunicationsTEL: (+81)-797-32-1995 Error 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
Business Wire
24-06-2025
- Business
- Business Wire
Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by European Commission (EC)
TOKYO & HYOGO, Japan--(BUSINESS WIRE)-- MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the European Commission (EC) has granted orphan drug designation (ODD) to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B), following the recent the U.S. Food and Drug Administration (FDA) designation. 1 MPS IIIB affects an estimated 500 to 1,000 individuals worldwide, 2 causing severe central nervous system (CNS) symptoms. Despite the dire need, there are currently no approved treatments available for this condition. JR-446, developed using JCR's proprietary J-Brain Cargo ® technology, has shown promising non-clinical results in addressing the CNS symptoms of this challenging disorder, and it is currently being studied in a Phase I/II trial that is being conducted in Japan (JR-446-101) under a collaboration agreement between the two companies. In September 2023, MEDIPAL and JCR entered into a licensing agreement in which MEDIPAL will commercialize JR-446 for MPS IIIB outside of Japan. In addition, MEDIPAL will support JCR in the clinical development of JR-446 in Japan, including the distribution of investigational drugs, disease awareness, and clinical trial advancement. 3 With the ODD, JR-446 will be eligible for various incentives to encourage the development in the European Union (EU). About Orphan Drug Designation in the European Union The European Commission implements orphan designation drug for promoting new drug development for rare diseases in which the prevalence of the condition affects no more than five in 10,000 people in the European Union (EU). Designated drugs are granted market exclusivity for 10 years in the EU, as well as scientific guidance. Fee reductions are also available depending on the status of the sponsor and the type of service required. About Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B) Mucopolysaccharidosis type IIIB, or Sanfilippo syndrome type B, is an autosomal recessive disease caused by pathogenic mutations in the NAGLU gene, encoding a lysosomal enzyme involved in the degradation of heparan sulfate. With the accumulation of heparan sulfate in the central nervous system in the brain, individuals with this condition present rapid neurological decline, including sleep disorders, loss of speech, and behavioral changes, which may significantly affect the quality of life of patients and their families. About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology J- Brain Cargo ®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology is IZCARGO ® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder. About MEDIPAL HOLDINGS CORPORATION MEDIPAL is a holding company which controls, administers and supports the operating activities of companies in which it holds shares in the Prescription Pharmaceutical Wholesale Business; the Cosmetics, Daily Necessities and OTC Pharmaceutical Wholesale Business; and the Animal Health Products and Food Processing Raw Materials Wholesale and Related Business, and conducts business development for the MEDIPAL Group. For more information, visit About JCR Pharmaceuticals Co., Ltd. JCR is a global specialty pharmaceuticals company dedicated to advancing treatments for rare and genetic diseases. With nearly 50 years of expertise in Japan, JCR is expanding to the US, Europe, and Latin America. JCR's innovative therapies address conditions like growth disorder, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. JCR is also developing treatments for rare diseases like MPS I, MPS II, MPS IIIA and B, and more. For more information, visit Cautionary Statement Regarding Forward-Looking Statements This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as 'believe,' 'estimate,' 'anticipate,' 'intend,' 'plan,' 'will,' 'would,' 'target' and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors' pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future. References Reference: Press release on the orphan drug designation for JR-446 for Mucopolysaccharidosis Type IIIB by the U.S. FDA (May 7, 2025). Based on data from JCR's own investigations, referring to the Ministry of Health, Labour and Welfare's public research. Reference: Press release on the licensing agreement for JR-446 between MEDIPAL and JCR (September 28, 2023).
Business Wire
07-05-2025
- Business
- Business Wire
Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by the U.S. FDA
TOKYO & HYOGO, Japan--(BUSINESS WIRE)-- MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B). MPS IIIB affects an estimated 500 to 1,000 individuals worldwide, 1 causing severe central nervous system (CNS) symptoms. Despite the dire need, there are currently no approved treatments available for this condition. JR-446, developed using JCR's proprietary J-Brain Cargo ® technology, has shown promising non-clinical results in addressing the CNS symptoms of this challenging disorder, and is currently being studied in a Phase I/II trial that is being conducted in Japan (JR- 446-101) under a collaboration agreement between the two companies. 'We are extremely pleased that JR-446 has received orphan drug designation from the U.S. FDA,' said Shuichi Watanabe, Representative Director, President and CEO of MEDIPAL. 'We will continue the development of JR-446 in collaboration with JCR so that we can soon deliver this drug to the patients and their families who are desperately waiting, as there is currently no treatment available.' 'This orphan drug designation is an important development step for the JR-446 clinical program,' said Shin Ashida, President, Chairman and CEO of JCR Pharmaceuticals. 'This orphan drug designation supports the potential of this therapy to address the significant unmet medical need of patients with MPS IIIB.' In September 2023, MEDIPAL and JCR entered into a licensing agreement in which MEDIPAL will commercialize JR-446 outside of Japan. In addition, MEDIPAL will support JCR in the clinical development of JR-446 in Japan, including the distribution of investigational drugs, disease awareness, and clinical trial advancement. 2 With the ODD, JR-446 will be eligible for various incentives to encourage the development in the U.S. About Orphan Drug Designation in the U.S. The U.S. FDA's Office of Orphan Products Development grants orphan status to drugs being developed to treat, prevent, or diagnose a rare disease or condition affecting fewer than 200,000 people in the U.S. The designation provides significant incentives to promote the development of the drug including the potential for market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, and waiver of Prescription Drug User Fee Act Application fee. About Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B) Mucopolysaccharidosis type IIIB, or Sanfilippo syndrome type B, is an autosomal recessive disease caused by pathogenic mutations in the NAGLU gene, encoding a lysosomal enzyme involved in the degradation of heparan sulfate. With the accumulation of heparan sulfate in the central nervous system in the brain, individuals with this condition present rapid neurological decline, including sleep disorders, loss of speech, and behavioral changes, which may significantly affect the quality of life of patients and their families. About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology, J- Brain Cargo ®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology is IZCARGO ® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder. About MEDIPAL HOLDINGS CORPORATION MEDIPAL is a holding company which controls, administers and supports the operating activities of companies in which it holds shares in the Prescription Pharmaceutical Wholesale Business; the Cosmetics, Daily Necessities and OTC Pharmaceutical Wholesale Business; and the Animal Health Products and Food Processing Raw Materials Wholesale and Related Business, and conducts business development for the MEDIPAL Group. For more information, visit About JCR Pharmaceuticals Co., Ltd. JCR is a global specialty pharmaceuticals company dedicated to advancing treatments for rare and genetic diseases. With nearly 50 years of expertise in Japan, JCR is expanding to the US, Europe, and Latin America. JCR's innovative therapies address conditions like growth disorder, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. JCR is also developing treatments for rare diseases like MPS I, MPS II, MPS IIIA and B, and more. The core values of reliability, confidence, and persistence drive JCR's mission to enhance global medical progress. For more information, visit Cautionary Statement Regarding Forward-Looking Statements This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as 'believe,' 'estimate,' 'anticipate,' 'intend,' 'plan,' 'will,' 'would,' 'target' and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors' pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future. References Based on data from JCR's own investigations, referring to the Ministry of Health, Labour and Welfare's public research. Reference: Press release on the licensing agreement for JR-446 between MEDIPAL and JCR (September 28, 2023).
Business Wire
06-05-2025
- Business
- Business Wire
Spruce Biosciences Reports First Quarter 2025 Financial Results and Provides Corporate Updates
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Spruce Biosciences, Inc. (OTC: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today reported financial results for the first quarter ended March 31, 2025 and provided corporate updates. 'With no FDA-approved treatments currently available to treat MPS IIIB, TA-ERT has the potential to be a groundbreaking advancement for patients and families impacted by this devastating disease,' said Javier Szwarcberg, M.D., M.P.H., Chief Executive Officer of Spruce. 'Across the landscape, this is an incredibly important and exciting time for patients and families affected by neuropathic MPS diseases. Looking ahead, we remain focused on pursuing accelerated approval of TA-ERT and filing the BLA in the first half of 2026. We also plan to initiate a confirmatory study prior to potential accelerated approval of TA-ERT and enable expanded access programs to ensure that patients have access to therapy.' Corporate Updates TA-ERT for the Treatment of MPS IIIB. Spruce entered into an Asset Purchase Agreement under which the company acquired an exclusive worldwide license agreement for TA-ERT and other enzyme replacement therapy products. TA-ERT is a fusion protein comprised of recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) with modified human insulin-like growth factor 2 via an amino acid linker. TA-ERT is intended as an enzyme replacement therapy for the treatment of patients with MPS IIIB who lack rhNAGLU enzyme activity. In March 2024, in a Type C meeting with the U.S. Food and Drug Administration (FDA), the FDA confirmed that HS-NRE is deemed to be a surrogate biomarker reasonably likely to predict clinical benefit and could serve as a basis for accelerated approval. The FDA also confirmed that the completed clinical and non-clinical studies of TA-ERT were sufficient for a BLA submission and provided guidance around key design elements of a confirmatory trial, which must be initiated prior to potential accelerated approval of TA-ERT. TA-ERT has received Fast Track Designation, Rare Pediatric Disease Designation, and Orphan Drug Designation in the U.S. and EU. Spruce intends to submit the BLA of TA-ERT for the treatment of MPS IIIB in the first half of 2026. Tildacerfont and Cortibon for the Treatment of Major Depressive Disorder (MDD). Spruce entered into a license, development and option agreement (the 'HMNC Agreement') with HMNC Holding GmbH ('HMNC'). Under the terms of the HMNC Agreement, HMNC will fund and conduct a Phase 2 proof-of-concept study of tildacerfont, a potent and highly selective, oral, small-molecule antagonist of the CRF 1 receptor, in patients with MDD who will be screened using Cortibon, HMNC's proprietary genetic test. HMNC has initiated the Phase 2 TAMARIND study, which will explore the efficacy of 400mg twice-daily tildacerfont versus placebo in improving depressive symptoms in MDD patients. Topline results from TAMARIND are anticipated in the first half of 2026. First Quarter 2025 Financial Results Cash and Cash Equivalents: Cash and cash equivalents as of March 31, 2025 were $25.6 million. Cash and cash equivalents are expected to allow the company to fund its current operating plan through the end of 2025. Research and Development (R&D) Expenses: R&D expenses for the three months ended March 31, 2025 were $10.8 million compared to $10.3 million for the same period in 2024. R&D expenses for the three months ended March 31, 2025 include $5.7 million in costs related to the acquisition of SPR202, an anti-corticotrophin releasing hormone monoclonal antibody for the treatment of congenital adrenal hyperplasia. General and Administrative (G&A) Expenses: G&A expenses for the three months ended March 31, 2025 were $3.7 million compared to $4.3 million for the same period in 2024, primarily driven by a decrease in stock-based compensation expense. Total Operating Expenses: Total operating expenses for the three months ended March 31, 2025 were $14.5 million compared to $14.6 million for the same period in 2024. Operating expenses include non-cash stock-based compensation expenses of $0.5 million for the three months ended March 31, 2025 compared to $1.6 million for the same period in 2024. Net Loss: Net loss for the three months ended March 31, 2025 was $14.0 million compared to $11.6 million for the same period in 2024. About Spruce Biosciences Spruce Biosciences is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need. To learn more, visit and follow us on X, LinkedIn, Facebook and YouTube. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the ability to seek accelerated approval of TA-ERT for MPS IIIB based on existing clinical data; the anticipated timing and conduct of Spruce's confirmatory trial for TA-ERT; the timing and likelihood of regulatory filings and approvals for TA-ERT, including the anticipated BLA Submission of TA-ERT for MPS IIIB in the first half of 2026; Spruce's expectation that topline results from the TAMARIND study will be available in the first half of 2026; and Spruce's intended focus on serious diseases with significant unmet medical need and clear biology, are forward-looking statements. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as 'anticipate', 'will', 'potential', 'intend', 'expect' and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Spruce's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Spruce's business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Spruce's filings with the U.S. Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Spruce undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. March 31, 2025 2024 ASSETS Current assets: Cash and cash equivalents $ 25,615 $ 38,753 Prepaid expenses 2,899 3,177 Other current assets 2,062 2,276 Total current assets 30,576 44,206 Right-of-use assets 869 934 Other assets 204 69 Total assets $ 31,649 $ 45,209 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities: Accounts payable $ 1,879 $ 1,295 Accrued expenses and other current liabilities 12,442 12,329 Term loan, current portion 1,345 1,622 Total current liabilities 15,666 15,246 Lease liabilities, net of current portion 659 736 Term loan, net of current portion — 124 Other liabilities — 282 Total liabilities 16,325 16,388 Commitments and contingencies Stockholders' equity: Preferred stock, $0.0001 par value; 10,000,000 shares authorized and no shares issued or outstanding as of March 31, 2025 and December 31, 2024 — — Common stock, $0.0001 par value; 200,000,000 shares authorized as of March 31, 2025 and December 31, 2024; 42,231,285 shares issued and outstanding as of March 31, 2025 and December 31, 2024 4 4 Additional paid-in capital 279,629 279,085 Accumulated deficit (264,309 ) (250,268 ) Total stockholders' equity 15,324 28,821 Total liabilities and stockholders' equity $ 31,649 $ 45,209 Expand SPRUCE BIOSCIENCES, INC. CONDENSED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited) (in thousands, except share and per share amounts) Three Months Ended March 31, 2025 2024 Collaboration revenue $ — $ 2,002 Operating expenses: Research and development 10,837 10,317 General and administrative 3,655 4,318 Total operating expenses 14,492 14,635 Loss from operations (14,492 ) (12,633 ) Interest expense (36 ) (97 ) Interest and other income, net 487 1,105 Net loss and comprehensive loss (14,041 ) (11,625 ) Net loss per share, basic and diluted $ (0.32 ) $ (0.28 ) Weighted-average shares of common stock outstanding, basic and diluted 43,944,946 41,096,231 Expand
