Latest news with #SanfilippoSyndrome
Yahoo
11-08-2025
- Business
- Yahoo
Denali Therapeutics Reports Second Quarter 2025 Financial Results and Business Highlights
Tividenofusp alfa BLA for Hunter syndrome accepted for priority review and assigned PDUFA target action date of January 5, 2026; company preparing for commercial launch DNL126 accelerated approval path for Sanfilippo syndrome Type A aligned with FDA; Phase 1/2 study nearing completion of enrollment; planning underway for a global Phase 3 confirmatory study On track to submit regulatory applications in 2025 to begin clinical testing of one to two additional TransportVehicleTM (TV)-enabled programs Preclinical research on ATV:Abeta program for Alzheimer's disease published in the journal Science SOUTH SAN FRANCISCO, Calif., Aug. 11, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today reported financial results for the second quarter ended June 30, 2025, and provided business highlights. 'The FDA's priority review of our BLA for tividenofusp alfa and alignment on an accelerated approval path for DNL126 are key milestones highlighting the potential of our Transport Vehicle (TV) platform to catalyze a new class of blood-brain barrier-crossing therapeutics,' said Ryan Watts, Ph.D., CEO of Denali Therapeutics. 'With launch readiness in motion and a growing portfolio of TV-enabled enzyme, antibody, and oligonucleotide programs, Denali is poised to deliver meaningful treatments for people living with lysosomal, neurodegenerative, and other serious diseases.' Second Quarter 2025 and Recent Program Updates CLINICAL PROGRAMS Tividenofusp alfa (DNL310, ETV:IDS) for Hunter syndrome (MPS II) In July 2025, Denali announced that the U.S. Food and Drug Administration (FDA) accepted its Biologics License Application (BLA) for tividenofusp alfa for priority review, assigning a Prescription Drug User Fee Act (PDUFA) target action date of January 5, 2026. The BLA seeks accelerated approval based on a data package including results from the Phase 1/2 study in individuals with Hunter syndrome. Tividenofusp alfa is an investigational, next-generation enzyme replacement therapy designed to cross the blood-brain barrier (BBB) and deliver the iduronate-2-sulfatase (IDS) enzyme throughout the body and brain. The FDA previously granted tividenofusp alfa Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations. Denali continues to prepare for commercial launch and is conducting the Phase 2/3 COMPASS study to support global regulatory submissions. DNL126 (ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA) Today, Denali announced that it has reached alignment with the FDA's Center for Drug Evaluation and Research (CDER) that cerebrospinal fluid heparan sulfate (CSF HS) may be considered a reasonably likely surrogate endpoint to predict clinical benefit and may therefore be used to support accelerated approval of DNL126 for MPS IIIA. Additional 49-week data from the ongoing open-label Phase 1/2 study are consistent with previously announced 25-week data, demonstrating a significant reduction in CSF HS from baseline, including normalization, and a safety profile that supports continued development. Enrollment in the Phase 1/2 study is nearly complete, and planning is underway for a confirmatory global Phase 3 study. TAK-594/DNL593 (PTV:PGRN) for GRN-related frontotemporal dementia Denali and Takeda continue their collaboration to develop DNL593, an investigational therapeutic designed to deliver progranulin across the BBB for the treatment of granulin (GRN) mutation-associated frontotemporal dementia (FTD-GRN). A Phase 1/2 study is ongoing. BIIB122/DNL151 (small molecule LRRK2 inhibitor) for the treatment of Parkinson's disease (PD) Denali and Biogen are co-developing LRRK2 inhibitors for Parkinson's disease. In May 2025, Biogen announced that the Phase 2b LUMA study of BIIB122 completed enrollment, with a readout expected in 2026. Denali is also conducting the Phase 2a BEACON study focused on LRRK2-associated PD. IND-ENABLING STAGE PROGRAMS Denali expects to submit regulatory applications to begin clinical testing of one to two TV-enabled programs each year over the next three years across its Enzyme TV (ETV), Antibody TV (ATV), and Oligonucleotide TV (OTV) franchises. The most advanced programs include: DNL952 (ETV:GAA) for Pompe disease; DNL111 (ETV:GCase) for Parkinson's/Gaucher disease; DNL622 (ETV:IDUA) for MPS I; DNL921 (ATV:Abeta) for Alzheimer's disease; DNL628 (OTV:MAPT) for Alzheimer's disease; and DNL422 (OTV:SNCA) for Parkinson's disease. Denali announced publication of preclinical data on ATV:Abeta in the August 7, 2025, issue of the journal Science. The research demonstrated that delivering an anti-amyloid beta antibody across the BBB using Denali's TV platform improved brain distribution and reduced the risk of amyloid-related imaging abnormality (ARIA) in a mouse model of Alzheimer's disease, compared to conventional antibody treatment. The findings suggest that TV platform-enabled brain delivery of immunotherapy bypasses amyloid-laden large vessels by traveling through smaller capillaries, offering a potential strategy to mitigate ARIA risk seen with first-generation anti-amyloid therapies. The Science article can be accessed here. Participation in Upcoming Investor Conferences Cantor Global Healthcare Conference 2025, September 3 - 5 (New York City) Morgan Stanley 23rd Annual Global Healthcare Conference, September 8 - 10 (New York City) Baird 2025 Global Healthcare Conference, September 9 - 10 (New York City) H.C. Wainwright 27th Annual Global Investment Conference, September 8 - 10 (New York City) Deutsche Bank BioPharm Corporate Day, September 18 - 19 (Austria) Stifel 2025 Healthcare Conference, November 11 - 13 (New York City) Jefferies Global Healthcare Conference, November 17 - 20 (London) Second Quarter 2025 Financial Results Net loss was $124.1 million for the quarter ended June 30, 2025, compared to net loss of $99.0 million for the quarter ended June 30, 2024. Total research and development expenses were $102.7 million for the quarter ended June 30, 2025, compared to $91.4 million for the quarter ended June 30, 2024. The increase of approximately $11.3 million was attributable to an increase of $7.3 million in TV program external research and development expenses, primarily driven by increased spend on multiple preclinical programs, and increases of $7.6 million and $6.2 million in other research and development expenses and personnel-related expenses, respectively, both driven by the commencement of operations at Denali's large molecule manufacturing facility in Salt Lake City, Utah. These increases were partially offset by a $9.8 million decrease in small molecule programs, primarily due to the winding down of activities related to the Phase 2/3 HEALEY ALS Platform Trial. General and administrative expenses were $32.3 million for the quarter ended June 30, 2025, compared to $25.2 million for the quarter ended June 30, 2024. The increase of $7.1 million was primarily driven by activities related to preparations for a potential commercial launch for tividenofusp alfa. Cash, cash equivalents, and marketable securities were approximately $977.4 million as of June 30, 2025. About Denali Therapeutics Denali Therapeutics is a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases. Denali pursues new treatments by rigorously assessing genetically validated targets, engineering delivery across the BBB, and guiding development through biomarkers that demonstrate target and pathway engagement. Denali is based in South San Francisco. For additional information, please visit Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements expressed or implied in this press release include, but are not limited to, statements regarding expectations for Denali's TV platform and its therapeutics and commercial potential; statements made by Denali's Chief Executive Officer; plans, timelines, and expectations relating to DNL310, including the PDUFA target action date and the timing, likelihood of, and scope of regulatory approval, the ongoing global Phase 2/3 COMPASS study and the likelihood of global approvals, and planned commercial launch; plans, timelines, and expectations related to DNL126, including enrollment in the ongoing Phase 1/2 study, plans regarding the confirmatory global Phase 3 study, planned engagement with the FDA, and the likelihood and scope of regulatory approvals; plans regarding DNL593 and the ongoing Phase 1/2 study; plans, timelines, and expectations regarding DNL151, including with respect to the ongoing Phase 2b LUMA study and the timing and likelihood of readout, and the ongoing Phase 2a BEACON study; plans and expectations for Denali's preclinical programs, including the timing of advancement to clinical studies; the findings from Denali's recent Science publication and their therapeutic potential regarding ARIA risk; Denali's participation in upcoming investor conferences; and Denali's future operating expenses and anticipated cash runway. All drugs currently being developed by Denali are investigational and have not received regulatory approval for any indication. Actual results are subject to risks and uncertainties and may differ materially from those indicated by these forward-looking statements as a result of these risks and uncertainties, including but not limited to, risks related to: the impact of adverse economic conditions, tariffs, and inflation on Denali's business and operations; the occurrence of any event, change, or other circumstance that could give rise to the termination of Denali's agreements with Sanofi, Takeda, Biogen, or other collaborators; Denali's transition to a late-stage clinical drug development company; Denali's and its collaborators' ability to complete the development and, if approved, commercialization of its product candidates; Denali's and its collaborators' ability to enroll patients in its ongoing and future clinical trials; Denali's reliance on third parties for the manufacture and supply of its product candidates for clinical trials; Denali's dependence on successful development of its blood-brain barrier platform technology and its programs and product candidates; Denali's and its collaborators' ability to conduct or complete clinical trials on expected timelines; the risk that preclinical profiles of Denali's product candidates may not translate in clinical trials; the potential for clinical trials to differ from preclinical, early clinical, preliminary or expected results; the risk of significant adverse events, toxicities, or other undesirable side effects; the uncertainty that product candidates will receive regulatory approval necessary to be commercialized; Denali's ability to continue to create a pipeline of product candidates or commercialize products; developments relating to Denali's competitors and its industry, including competing product candidates and therapies; Denali's ability to obtain, maintain, or protect intellectual property rights related to its product candidates; implementation of Denali's strategic plans for its business, product candidates, and blood-brain barrier platform technology; Denali's ability to obtain additional capital to finance its operations, as needed; Denali's ability to accurately forecast future financial results and hedge against financial risk in the current environment; and other risks and uncertainties, including those described in Denali's most recent Annual Report and Quarterly Reports on Forms 10-K and 10-Q filed with the Securities and Exchange Commission (SEC) on February 27, 2025 and May 6, 2025, and Denali's future reports to be filed with the SEC. Denali does not undertake any obligation to update or revise any forward-looking statements, to conform these statements to actual results, or to make changes in Denali's expectations, except as required by law. Denali Therapeutics Consolidated Statements of Operations(Unaudited)(In thousands, except share and per share amounts) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024 Operating expenses: Research and development $ 102,696 $ 91,399 218,923 198,415 General and administrative 32,267 25,194 61,620 50,430 Total operating expenses 134,963 116,593 280,543 248,845 Gain from divestiture of small molecule programs — — — 14,537 Loss from operations (134,963 ) (116,593 ) (280,543 ) (234,308 ) Interest and other income, net 10,844 17,567 23,454 33,480 Net loss $ (124,119 ) $ (99,026 ) $ (257,089 ) $ (200,828 ) Net loss per share, basic and diluted $ (0.72 ) $ (0.59 ) $ (1.50 ) $ (1.26 ) Weighted average number of shares outstanding, basic and diluted 171,449,847 168,831,329 171,336,568 159,117,759 Denali Therapeutics Consolidated Balance Sheets(Unaudited)(In thousands) June 30, 2025 December 31, 2024 Assets Current assets: Cash and cash equivalents $ 141,207 $ 174,960 Short-term marketable securities 757,745 657,371 Prepaid expenses and other current assets 35,754 32,105 Total current assets 934,706 864,436 Long-term marketable securities 78,463 359,373 Property and equipment, net 58,717 55,236 Finance lease right-of-use asset 50,363 47,533 Operating lease right-of-use asset 21,022 22,861 Other non-current assets 22,970 24,741 Total assets $ 1,166,241 $ 1,374,180 Liabilities and stockholders' equity Current liabilities: Accounts payable $ 10,844 $ 11,137 Accrued compensation 12,068 24,728 Accrued clinical and other research & development costs 23,379 22,822 Accrued manufacturing costs 9,028 12,779 Operating lease liability, current 8,871 8,308 Deferred research and development funding liability, current 19,861 14,129 Other accrued costs and current liabilities 7,006 8,305 Total current liabilities 91,057 102,208 Operating lease liability, less current portion 32,110 36,673 Finance lease liability, less current portion 5,577 5,615 Deferred research funding and development liability, less current portion 10,444 — Total liabilities 139,188 144,496 Total stockholders' equity 1,027,053 1,229,684 Total liabilities and stockholders' equity $ 1,166,241 $ 1,374,180 Investor Contact:Laura Hansen, Media Contact:Erin Pattonepatton@ 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
Yahoo
13-07-2025
- Business
- Yahoo
Ultragenyx (RARE) Falls 26% as New Treatment Fails to Get FDA Green Light
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the Ultragenyx fell by 26.05 percent week-on-week as investor sentiment was largely dragged down by the Food and Drug Administration's (FDA) rejection of its biologics license application (BLA) for its UX111 (ABO-102) treatment. In a regulatory filing, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said that the FDA requested additional information and improvements in relation to certain chemistry, manufacturing, and controls (CMC) aspects. It noted that the observations were not related to product quality concerns product, adding that many of the issues have already been addressed. UX111 was developed to treat a common type of Sanfilippo syndrome, a group of genetic conditions that begin in early childhood and causes severe brain damage and early death. A research team in a laboratory peering through microscopes at a biologic product. The treatment involves managing symptoms, as there are currently no approved disease-modifying medicines. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said it plans to resubmit updated clinical data from current patients after resolving the FDA's concerns. A new review could take another six months upon resubmission of a revised BLA application. While we acknowledge the potential of RARE as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an extremely cheap AI stock that is also a major beneficiary of Trump tariffs and onshoring, see our free report on the . READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now. Disclosure: None. This article is originally published at Insider Monkey.
Yahoo
30-06-2025
- Health
- Yahoo
'She Stopped Talking At 2': 5-Year-Old's Dementia Diagnosis Began With These Early Warning Signs
A grandparent has opened up about the early warning signs that led to her grandchild's devastating dementia diagnosis. Sabrina Peake said her granddaughter Esmay Ford, who is five, stopped talking at the age of two. She was also constantly 'picking up colds', remaining ill 'for weeks at a time', and her hair was falling out in 'big clumps', according to the Mail Online. Esmay's mum Alisha Morris was concerned for her daughter, so she took her to the doctor's and was told she had an autoimmune disease and overactive thyroid. But after genetic testing more recently, the family were given the heartbreaking news that their daughter has Sanfilippo syndrome, a rare form of childhood dementia. Children with this disease lack an enzyme which breaks down large sugar molecules. As the molecules accumulate in the cells of the body, they cause irreparable damage to the organs, including the brain. Childhood dementia occurs because of progressive brain damage. It is caused by over 100 rare genetic disorders, which children are born with, according to the Childhood Dementia Initiative. It's estimated one in every 2,900 babies globally is born with a condition that can cause childhood dementia. Unfortunately, because of the nature of the disease, many children diagnosed with the cruel condition don't live past 18 years old. The symptoms are similar to those in adults, and can include: Memory loss Confusion Trouble concentrating, understanding, learning and communicating Personality changes Disturbed sleep Behavioural issues such as hyperactivity Emotional issues like anxiety and fear. There are also specific clinical features of Sanfilippo syndrome, such as: Coarse facial features / coarse and thick hair Frontal bossing (prominent forehead) Macrocephaly (a larger sized head) Chronic nasal congestion Toe-walking Seizures Hearing loss Lack of fear (of danger) Gastrointestinal discomfort and colic Increased food intake or nursing. Esmay's family, who are based in Devon, said her condition will progressively worsen. They are currently raising funds to take her to Disneyland and also buy specialist equipment to care for her, like a modified bed. The Sanfilippo Children's Foundation notes: 'As the brain gets progressively damaged, children experience severe hyperactivity, disordered sleep, loss of speech, cognitive decline, cardiac issues, seizures, loss of mobility, and finally death, usually before adulthood.' Esmay's grandmother Sabrina Peake told the Mail Online: 'We've had to come to terms with the fact that we will lose her, and that things will be extremely tough going forward as she gets worse. 'Our biggest fear aside from losing her at such a young age, is watching her suffer.' It's estimated that 700,000 children globally live with childhood dementia. In the UK, it's estimated 204 die from the disease each year. The Sanfilippo Children's Foundation said there is no treatment or cure for those with a diagnosis, however several clinical trials have already been completed or are underway. So far, the foundation said gene therapy looks to be the most promising. 'My Child Is Worried He's Not Real. He Ended Up Sobbing Because Of It' It's Not Social Media – What Teens Say Is Damaging Their Mental Health Most Dementia Risk Starts In Childhood And Even Infancy, Scientists Warn
Daily Mail
29-06-2025
- Health
- Daily Mail
My five-year-old daughter has childhood Alzheimer's and isn't expected to live past her teen years - these are the two early symptoms I spotted
A heartbroken mother spotted her daughter's 'childhood Alzheimer's ' after she stopped talking aged two - and is now desperate to make memories before it's 'too late'. Esmay Ford, five, was diagnosed with Sanfilippo Syndrome (MPS IIIA) on May 15 - a rare, life-limiting genetic form of childhood dementia that causes mental deterioration, memory loss and personality changes. Esmay's mother, Alisha Morris, 24, and grandmother, Sabrina Peake, 45, grew concerned about the 'healthy' tot in 2021, after she stopped talking and was constantly 'picking up colds'. The family was initially told by doctors that Esmay had an autoimmune disease and overactive thyroid, which had caused her brain to stop developing. They 'hoped and prayed' that medication would help with her brain development, but after a series of genetic testing, Esmay was diagnosed with MPS IIIA. Also known as 'childhood Alzheimer's' due to the similarity in symptoms, the disorder affects the brain and nervous system causing cognitive decline. The degenerative disorder means that Esmay's life expectancy is between 10 to 18 years old, and her condition will get progressively worse until 'her body totally gives up on her'. Five-year-old Esmay is now unable to recognise her family and needs 'constant supervision' as she is unable to walk and has 'no awareness of danger'. Her family are desperate to 'give her the best life' before her condition becomes too severe, and are raising money for a trip to Disney Land, as well as vital equipment such as a modified bed and shower. Sabrina, a flight attendant, from Crediton, Devon, said: 'We knew something was wrong when she stopped talking, and then her hair started to fall out in big clumps. 'Esmay doesn't know much about it as she's in her own little world most the time, but it's been incredibly tough for us. 'She can barely walk and she can't talk, and she has no awareness and doesn't interact with anyone. 'I don't think she always knows who we are. She is now is the healthiest she'll be, she'll get worse every day until her body totally gives up on her.' Sabrina was 'over the moon' when her daughter Alisha gave birth to a 'happy and healthy' baby girl on 9th August 2019. As a toddler, Esmay started to talk and loved playing in the park, dancing, and interacting with others. In 2021, at two and a half years old, Sabrina and Alisha noticed that Esmay had become withdrawn and had stopped talking. Sabrina explained: 'We noticed she didn't like doing the things she used to like doing like going down the slide at the park and dancing to music. 'She would pick up bugs and colds and would be ill for weeks at a time - we knew something was wrong. 'When her hair started to fall out, we took her to Exeter Hospital A&E and had to really push for answers.' Blood tests revealed that little Esmay had an auto immune disease and overactive thyroid, and after further genetic testing she was diagnosed with MPS IIIA. Doctors have warned her family that her mobility and cognitive ability will continue to deteriorate, and that she will have a significantly shorter life expectancy. Sabrina said: 'We've had to come to terms with the fact that we will lose her, and that things will be extremely tough going forward as she gets worse. Our biggest fear aside from losing her at such a young age, is watching her suffer.' Esmay's family are now raising money to cover the costs of the equipment she will need as her condition deteriorates, as well as 'making memories' with her while they can. Sabrina said: 'We don't know what the future holds, but we're raising money to get Esmay all the things she will need to support her, and make the remainder of her life as comfortable as possible. 'She will need special car seats, a bath and shower chair, a padded, zip up cot and bed, a reclining disabled special buggy, plus many more things. 'We also want to give her the best life now, before she gets worse. 'We'd love to take her to Disneyland, but this is the healthiest she'll be so we'd need to go soon. 'I have watched my beautiful granddaughter rapidly decline over the last few years which is heartbreaking. 'I would give anything to hear her talk, laugh, or even smile again. 'To know that I will never hear her speak again is so hard to accept.' You can donate to the GoFundMe here - WHAT IS SANFILIPPO SYNDROME? Sanfilippo syndrome is a genetic and terminal disorder that affects around one in 70,000 live births. Victims lack an enzyme that is essential for normal cellular function. The condition eventually causes a buildup of a toxic material - heparin sulfate - resulting in dementia, loss of speech, blindness and eventual death. The disease is referred to as childhood Alzheimer's due to the effects. Sufferers typically die before they are out of their teens. There is no known cure, however clinical trials are being conducted in order to find one.
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).
