Hunter syndrome

Denali Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Business Highlights

Retrieved on: 
Tuesday, February 27, 2024

Enrollment continues in the global Phase 2/3 COMPASS study and is expected to be completed in 2024.

Key Points: 
  • Enrollment continues in the global Phase 2/3 COMPASS study and is expected to be completed in 2024.
  • Sanofi intends to present the detailed efficacy and safety results of the ALS Phase 2 HIMALAYA study at a future scientific forum.
  • There was no collaboration revenue for the quarter ended December 31, 2023, compared to $10.3 million for the quarter ended December 31, 2022.
  • Further, for the quarter ended December 31, 2023, there was also a decrease in other unallocated research and development expenses as a result of reduced facility costs.

JCR Pharmaceuticals’ Research Presentations at WORLDSymposium™ 2024 Showcase JR-141 (Pabinafusp Alfa) and Other Investigational Treatments for Lysosomal Storage Disorders

Retrieved on: 
Wednesday, February 14, 2024

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).
  • JCR is dedicated to address the unmet medical needs for this community,” said Shin Ashida, President and CEO of JCR Pharmaceuticals.
  • Finally, the third JR-141 presentation is pre-clinical and highlights the recovery of retinal function in mice with MPS II.
  • “We are pleased to be able to offer IZCARGO® to patients with MPS II in Japan,” said Yoshikatsu Eto, M.D., Ph.D., Institute of Neurological Disorders, Advanced Clinical Research Center, Kanagawa, Japan.

GC Biopharma Presents Updates on its LSD Treatments at the WORLDSymposium 2024

Retrieved on: 
Wednesday, February 14, 2024

YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.

Key Points: 
  • YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.
  • WorldSymposium 2024 is an international forum for Lysosomal Diseases experts to share and exchange insights for researching better treatment of the disease.
  • In 2012, GC Biopharma succeeded in developing the world's second treatment for Hunter syndrome, "Hunterase" solely using domestic technology.
  • GC Biopharma, together with Hanmi Pharmaceutical, is developing GC1134A/HM15421, a long-acting alpha-galactosidase that can be administered subcutaneously once a month to improve patient convenience.

Denali Therapeutics Announces New Data and Expansion of Its Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapy Programs at WORLDSymposium™

Retrieved on: 
Wednesday, February 7, 2024

Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.

Key Points: 
  • Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.
  • New two-year peripheral biomarker data demonstrate high magnitude and sustained reduction of urine heparan sulfate and dermatan sulfate, including in participants who switched from standard-of-care enzyme replacement therapy to tividenofusp alfa, suggesting enhanced peripheral activity.
  • Denali also announced that dosing has begun in the Phase 1/2 study of DNL126 for the potential treatment of MPS IIIA.
  • “Our goal is to bring effective new medicines to MPS families as soon as possible,” said Carole Ho, M.D., Chief Medical Officer of Denali.

REGENXBIO Announces Presentations at the 20th Annual WORLDSymposium 2024

Retrieved on: 
Wednesday, January 31, 2024

ROCKVILLE, Md., Jan. 31, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations on its program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be shared at the 20th Annual WORLDSymposium™ 2024, taking place in San Diego, CA February 3-9, 2024.

Key Points: 
  • ROCKVILLE, Md., Jan. 31, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations on its program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be shared at the 20th Annual WORLDSymposium™ 2024, taking place in San Diego, CA February 3-9, 2024.
  • The presentations include the topline results from the pivotal phase of the Phase I/II/III CAMPSIITE® trial of RGX-121 for the treatment of MPS II.
  • The poster presentations will be presented as follows:
    REGENXBIO will host a conference call Wednesday, February 7 at 4:30 p.m.
  • A replay of the webcast will also be available via the Company's investor website approximately two hours after the call's conclusion.

JCR Pharmaceuticals to Present at the 20th Annual WORLDSymposium™ 2024

Retrieved on: 
Thursday, January 25, 2024

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present six presentations at the 20th Annual WORLDSymposium™ 2024, to be held February 4-9, 2024 in San Diego, Calif.

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present six presentations at the 20th Annual WORLDSymposium™ 2024, to be held February 4-9, 2024 in San Diego, Calif.
  • In addition to posters being available for each of the presentations, two of them will be shared as oral presentations given by a clinical investigator and a researcher from JCR Pharmaceuticals.
  • A global phase III study of pabinafusp alfa (JR-141) for neuronopathic mucopolysaccharidosis type II: updated study design (Ikeda, et al.)
  • WORLDSymposium™ attendees who would like to receive more information about JCR Pharmaceuticals can visit JCR’s on-site conference booth (#506) or visit its virtual booth on the WORLDSymposium™ conference website.

Denali Therapeutics Announces Key Anticipated 2024 Milestones and Priorities to Further Advance Its Therapeutics Portfolio for Neurodegeneration and Lysosomal Storage Diseases

Retrieved on: 
Monday, January 8, 2024

The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.

Key Points: 
  • The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.
  • Upon completion of the ongoing Phase 1/2 study, and together with data from COMPASS, this combined data package is intended to support registration.
  • Increased RIPK1 activity in the CNS is hypothesized to drive neuroinflammation and cell necroptosis and to contribute to neurodegeneration.
  • Denali will maintain ownership of and continue to advance its current portfolio of clinical stage small molecule programs.

Denali Therapeutics Reports Third Quarter 2023 Financial Results and Business Highlights

Retrieved on: 
Tuesday, November 7, 2023

SOUTH SAN FRANCISCO, Calif., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), 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, today reported financial results for the third quarter ended September 30, 2023, and provided business highlights.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), 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, today reported financial results for the third quarter ended September 30, 2023, and provided business highlights.
  • Denali has selected five ASO targets for further development and is focused on advancing two OTV candidates towards clinical development.
  • 2023 Guidance on Operating Expenses:
    Cash, cash equivalents, and marketable securities were approximately $1.12 billion as of September 30, 2023.
  • General and administrative expenses were $25.3 million and $23.3 million for the three months ended September 30, 2023 and 2022, respectively.

Denali Therapeutics Announces New Interim Data from Phase 1/2 Study of DNL310 (ETV:IDS) in MPS II (Hunter Syndrome) at SSIEM 2023

Retrieved on: 
Wednesday, August 30, 2023

DNL310 is an investigational enzyme replacement therapy designed to cross the BBB and address the behavioral, cognitive, and physical manifestations of MPS II.

Key Points: 
  • DNL310 is an investigational enzyme replacement therapy designed to cross the BBB and address the behavioral, cognitive, and physical manifestations of MPS II.
  • The interim data from the Phase 1/2 study of DNL310 were highlighted in an oral presentation at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023 in Jerusalem, Israel.
  • A PDF of the Phase 1/2 presentation is available on Denali’s website on the Events page of the Investor section.
  • We are encouraged to see positive changes across multiple clinical outcomes measures in the ongoing Phase 1/2 study.

Initial Clinical Data of First Pediatric CLN2 Patient Dosed with RGX-181 Presented at SSIEM Annual Symposium

Retrieved on: 
Wednesday, August 30, 2023

"CLN2 is a debilitating disease caused by mutations in the CLN2 gene resulting in a deficiency of the TPP1 enzyme, which is needed to break down specific peptides associated with cellular waste.

Key Points: 
  • "CLN2 is a debilitating disease caused by mutations in the CLN2 gene resulting in a deficiency of the TPP1 enzyme, which is needed to break down specific peptides associated with cellular waste.
  • "We are encouraged by the initial results demonstrating that RGX-181 is well tolerated and dramatically reduced the number of seizures in the patient enrolled in this trial."
  • "The remarkable decrease in seizures, encouraging safety results and reduction in ERT frequency highlight the potential of this gene therapy to provide a meaningful treatment option to the CLN2 patient community."
  • Today, a physician investigator from the Hospital de Clinicas in Porto Alegre, Brazil reported initial results from a five-year-old child who received a one-time intracisternal dose of RGX-181.