UCL Institute of Ophthalmology

Vigil Neuroscience Reports Positive Interim Data from Phase 2 IGNITE Proof-of-Concept Clinical Trial Evaluating Iluzanebart (VGL101) as a Treatment for ALSP and from Ongoing Natural History Study ILLUMINATE

Retrieved on: 
Thursday, November 16, 2023

"The positive interim results from our Phase 2 IGNITE trial represent the first clinical data reported from an interventional study in patients with ALSP and reaffirm our belief in the potential of iluzanebart as a novel treatment option.

Key Points: 
  • "The positive interim results from our Phase 2 IGNITE trial represent the first clinical data reported from an interventional study in patients with ALSP and reaffirm our belief in the potential of iluzanebart as a novel treatment option.
  • Key Highlights from Phase 2 IGNITE Interim Data:
    Favorable safety and tolerability profile, including no hematologic adverse events.
  • We believe the quality and consistency of the interim data further support the continuation of IGNITE without modification.
  • Interim Montreal Cognitive Assessment (MoCA) and Cortical Basal Ganglia Functional Scale data support use as clinical endpoints in ALSP at 12 months.

Nanoscope Therapeutics Strengthens Research and Development Leadership with Appointment of Najam Sharif, PhD, DSc.

Retrieved on: 
Thursday, October 26, 2023

DALLAS, Oct. 26, 2023 /PRNewswire/ -- Nanoscope Therapeutics Inc. , a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, today announced the appointment of Najam Sharif, PhD, DSc, a veteran in drug/device discovery and development, as Vice President of Global Research and Development.

Key Points: 
  • DALLAS, Oct. 26, 2023 /PRNewswire/ -- Nanoscope Therapeutics Inc. , a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, today announced the appointment of Najam Sharif, PhD, DSc, a veteran in drug/device discovery and development, as Vice President of Global Research and Development.
  • "We are delighted to welcome Naj to the Nanoscope team," said Sulagna Bhattacharya, Co-Founder and Chief Executive Officer of Nanoscope.
  • "I am honored to join Nanoscope, a company that I see as transformative in the field of ophthalmology.
  • Dr. Naj Sharif brings over three decades of experience in pharmaceutical drug/device discovery and development to Nanoscope, including specialized expertise in ophthalmology and neuroscience.

Findings from Published Abstracts Showcase the Importance of Nicotinamide Adenine Dinucleotide (NAD+) in Glaucoma Patients and that Supplementation with Nicotinamide Riboside (NR) Demonstrate Promising Effects

Retrieved on: 
Tuesday, July 18, 2023

Together, the promising results from these abstracts suggest that glaucoma patients have lower cellular NAD+ levels and thus replenishing NAD+ levels with a precursor, such as nicotinamide riboside (NR), may be a potential therapeutic strategy.

Key Points: 
  • Together, the promising results from these abstracts suggest that glaucoma patients have lower cellular NAD+ levels and thus replenishing NAD+ levels with a precursor, such as nicotinamide riboside (NR), may be a potential therapeutic strategy.
  • Dr. Garway-Heath’s abstract titled “ Primary open angle glaucoma patients have lower systemic mitochondrial function, associated with lower systemic nicotinamide adenine dinucleotide (NAD) levels, compared to Controls ” observed significantly lower cellular NAD+ levels and impaired mitochondrial function in patients with primary open-angle glaucoma (POAG).
  • Further, higher NAD+ levels were strongly associated with higher mitochondrial function parameters, suggesting increased NAD+ levels are associated with improved energy production and cellular activity.
  • POAG is a condition where pressure builds inside the eye, causing damage to the optic nerve and gradual vision loss.

Mallinckrodt to Present Data on TERLIVAZ® (terlipressin) for Injection in Adult Patients with Hepatorenal Syndrome (HRS) at the European Association for the Study of the Liver (EASL) Congress 2023

Retrieved on: 
Thursday, June 15, 2023

DUBLIN, June 15, 2023 /PRNewswire/ -- Mallinckrodt plc (NYSE American: MNK), a global specialty pharmaceutical company, today announced that two scientific posters detailing Mallinckrodt's latest research findings on the clinical outcomes of treatment with TERLIVAZ® (terlipressin) for injection and clinical management criteria for adult patients with hepatorenal syndrome (HRS) will be presented at the European Association for the Study of the Liver (EASL) Congress 2023 in Vienna, Austria, taking place June 21-24, 2023. Both posters will be presented on June 24, 2023, 9:00 a.m. – 5:00 p.m. CEST / 3:00 a.m. – 11:00 a.m. EDT.

Key Points: 
  • Both posters will be presented on June 24, 2023, 9:00 a.m. – 5:00 p.m. CEST / 3:00 a.m. – 11:00 a.m. EDT.
  • The CONFIRM trial is the largest-ever prospective, randomized clinical trial of terlipressin compared to placebo in patients with HRS type 1 (HRS-1).
  • Ineligibility for Liver Transplant: TERLIVAZ-related adverse reactions (respiratory failure, ischemia) may make a patient ineligible for liver transplantation, if listed.
  • For patients with high prioritization for liver transplantation (e.g., MELD ≥35), the benefits of TERLIVAZ may not outweigh its risks.

Belite Bio Reports First-Quarter 2023 Operational Highlights and Financial Results

Retrieved on: 
Thursday, May 11, 2023

ET

Key Points: 
  • ET
    SAN DIEGO, May 10, 2023 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite” or the “Company”), a clinical stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting retinal degenerative eye diseases which have significant unmet medical needs, today announced its financial results for the first quarter ended March 31, 2023 and provided a general business update.
  • “We remain focused on advancing late-stage development of Tinlarebant and are pleased with the 60% enrollment seen to date in our pivotal Phase 3 DRAGON trial.
  • Among the minority subjects who did transition to DDAF lesions, Tinlarebant slowed the rate of progression compared to those reported in a natural history study.
  • Belite Bio will host a webcast to discuss the Company’s financial results and provide a business update.

Belite Bio to Host Key Opinion Leader Webinar Discussing Progression of Childhood-onset STGD1 and Relevance of the Tinlarebant 18-month Phase 2 Data

Retrieved on: 
Thursday, May 4, 2023

SAN DIEGO, May 04, 2023 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE), a clinical stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting retinal degenerative eye diseases which have significant unmet medical needs, today announced it will host a virtual Key Opinion Leader event on focusing on adolescent patients with Stargardt disease (STGD1) on Wednesday, May 10, 2023 at 1:00 PM Eastern Time.

Key Points: 
  • The event will feature Michel Michaelides, BSc, MB BS, MD(Res), FRCOphth, FACS (Moorfields Eye Hospital and UCL Institute of Ophthalmology) who will discuss progression of childhood-onset STGD1 and Belite Bio’s Phase 2 study evaluating the safety and efficacy of Tinlarebant (LBS-008) in adolescent STGD1 patients.
  • Tinlarebant (LBS-008) is an oral, once daily RBP4 antagonist designed to reduce the accumulation of ocular vitamin-A based toxins which have been implicated in the onset and progression of STGD1.
  • Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., and Orphan Drug Designation in the U.S. and Europe for the treatment of STGD1.
  • A live question and answer session will follow the formal presentations.

Vigil Neuroscience to Host ALSP KOL Event on December 6, 2022

Retrieved on: 
Monday, November 28, 2022

The event will provide further details on the ALSP disease background, epidemiology and patient journey.

Key Points: 
  • The event will provide further details on the ALSP disease background, epidemiology and patient journey.
  • To access a live webcast of this event, please visit Events & Presentations in the Investors section of the Vigil website at www.vigilneuro.com .
  • Vigil Neuroscience routinely posts information that may be important to investors in the Investors section of its website at https://www.vigilneuro.com .
  • The company encourages investors and potential investors to consult our website regularly for important information about Vigil Neuroscience.

SIFI ANNOUNCES EMA VALIDATION OF ITS MARKETING AUTHORISATION APPLICATION FOR AKANTIOR® FOR THE TREATMENT OF ACANTHAMOEBA KERATITIS AND THE OPENING OF AN EARLY ACCESS PROGRAM

Retrieved on: 
Tuesday, June 7, 2022

"We are incredibly pleased to be working in partnership with SIFI on this important access program," said Dan Piggott, Managing Director of Uniphar Group's Product Access Division.

Key Points: 
  • "We are incredibly pleased to be working in partnership with SIFI on this important access program," said Dan Piggott, Managing Director of Uniphar Group's Product Access Division.
  • "We look forward to helping facilitate broader access to polihexanide for patients with critical unmet medical needs suffering from acanthamoeba keratitis."
  • AKANTIOR (polihexanide) stands to become the first approved drug for the treatment of acanthamoeba keratitis in the world.
  • Polihexanide is also being developed by SIFI for the treatment of fungal keratitis for which indication it also has FDA Orphan Drug Designation.

SIFI ANNOUNCES EMA VALIDATION OF ITS MARKETING AUTHORISATION APPLICATION FOR AKANTIOR® FOR THE TREATMENT OF ACANTHAMOEBA KERATITIS AND THE OPENING OF AN EARLY ACCESS PROGRAM

Retrieved on: 
Tuesday, June 7, 2022

"We are incredibly pleased to be working in partnership with SIFI on this important access program," said Dan Piggott, Managing Director of Uniphar Group's Product Access Division.

Key Points: 
  • "We are incredibly pleased to be working in partnership with SIFI on this important access program," said Dan Piggott, Managing Director of Uniphar Group's Product Access Division.
  • "We look forward to helping facilitate broader access to polihexanide for patients with critical unmet medical needs suffering from acanthamoeba keratitis."
  • AKANTIOR (polihexanide) stands to become the first approved drug for the treatment of acanthamoeba keratitis in the world.
  • Polihexanide is also being developed by SIFI for the treatment of fungal keratitis for which indication it also has FDA Orphan Drug Designation.

MeiraGTx Presents Clinical Data on Botaretigene Sparoparvovec for the Treatment of X-Linked Retinitis Pigmentosa at the Association for Research in Vision and Ophthalmology 2022 Annual Meeting

Retrieved on: 
Wednesday, May 4, 2022

of Genetics

Key Points: 
  • of Genetics
    Patients in the multicenter, open-label Phase 1/2 trial were given botaretigene sparoparvovec subretinally at 1 of 3 doses to the worse-seeing eye.
  • Exploration of the association between the location of botaretigene sparoparvovec delivery and changes in retinal sensitivity was undertaken by overlaying bleb topography onto sensitivity heat maps.
  • Data from the study indicates improvements in photoreceptor function assessed through 12 months post-treatment suggesting local efficacy of botaretigene sparoparvovec gene therapy in XLRP patients.
  • These data are consistent with the reported Phase 1/2 trial positive results for botaretigene sparoparvovec in patients with RPGR-associated XLRP.