LogMAR chart

Taysha Gene Therapies Provides Clinical Updates for Investigational Programs TSHA-120 in Giant Axonal Neuropathy (GAN) and TSHA-102 in Rett Syndrome at R&D Day

Retrieved on: 
Wednesday, June 28, 2023
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DALLAS, June 28, 2023 (GLOBE NEWSWIRE) --  Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), announced new data analyses for TSHA-120 in GAN and initial clinical observations for TSHA-102 in Rett syndrome. Taysha will host a virtual R&D Day today at 10:00 AM ET to discuss these updates. The webcast link can be accessed on the Events and Presentations section of Taysha’s website.

Key Points: 
  • Taysha will host a virtual R&D Day today at 10:00 AM ET to discuss these updates.
  • The webcast link can be accessed on the Events and Presentations section of Taysha’s website.
  • We’ve requested a formal FDA meeting to discuss these new developments to support a potential regulatory path forward for TSHA-120.
  • TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform designed to regulate cellular MECP2 expression.

Annexon Topline Data from ARCHER Phase 2 Trial of ANX007 in Geographic Atrophy Demonstrated Statistically Significant, Dose-Dependent Preservation of Visual Function

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Wednesday, May 24, 2023
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Patients in the pooled treatment group showed a 59% reduction in risk of >15-letter loss (n=181, p=0.008).

Key Points: 
  • Patients in the pooled treatment group showed a 59% reduction in risk of >15-letter loss (n=181, p=0.008).
  • The primary endpoint of mean rate of change (slope) in GA lesion area compared to sham at 12 months did not reach statistical significance.
  • The totality of the data on ANX007 from the ARCHER trial are promising, with the demonstrated preservation of functional vision in GA patients, regardless of their lesion location or size.
  • Preclinical models have demonstrated that inhibition of C1q protects photoreceptor cell synapses, and importantly, photoreceptor cell function.

Nanoscope Therapeutics Announces Presentation of Key Results from Phase 2b RESTORE Trial of MCO-010 for the Treatment of Retinitis Pigmentosa at the ARVO Annual Meeting

Retrieved on: 
Thursday, April 27, 2023
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MCO-010 is an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy for vision restoration in advanced retinitis pigmentosa (RP), irrespective of gene mutation.

Key Points: 
  • MCO-010 is an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy for vision restoration in advanced retinitis pigmentosa (RP), irrespective of gene mutation.
  • In the RESTORE trial, 18 patients with severe vision impairment due to RP received a single intravitreal injection of MCO-010, while 9 received a sham intravitreal injection.
  • Results showed vision function improvements after treatment with MCO-010 consistent with previous studies as well as a favorable safety profile.
  • Key efficacy measures included Multi-Luminance Y-Mobility Test (MLYMT, vision-guided mobility), Multi-Luminance Shape Discrimination Test (MLSDT, near-field object recognition), and Best-Corrected Visual Acuity (BCVA) scores.

Atsena Therapeutics Announces Positive 6-month Data from Ongoing Phase I/II Clinical Trial of ATSN-101 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1)

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Tuesday, April 25, 2023
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The data were presented by Christine Nichols Kay, MD, Clinical Ophthalmology Advisor for Atsena, at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting.

Key Points: 
  • The data were presented by Christine Nichols Kay, MD, Clinical Ophthalmology Advisor for Atsena, at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting.
  • We look forward to reporting 12-month data later this year and are exploring options to advance ATSN-101 into a pivotal trial.”
    In the Phase I/II trial, 15 patients, including three pediatric patients, received ATSN-101 via subretinal delivery.
  • Two high-dose patients demonstrated best corrected visual acuity (BCVA) improvement greater than 0.3 logMAR, and no treated eyes had a decrease in BCVA.
  • To date, no drug-related serious adverse events have been reported and ocular inflammation has been infrequent, minimal, and reversible with steroid treatment.

Pixium Vision announces FDA Breakthrough Device Designation for the Prima System in Dry AMD

Retrieved on: 
Friday, March 31, 2023
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"To receive this Breakthrough Device Designation and have the FDA recognize the therapeutic potential of our Prima System is a significant achievement for Pixium Vision, especially as only a small proportion of devices awarded the designation are intended to treat ophthalmologic conditions1," said Lloyd Diamond, Chief Executive Officer of Pixium Vision.

Key Points: 
  • "To receive this Breakthrough Device Designation and have the FDA recognize the therapeutic potential of our Prima System is a significant achievement for Pixium Vision, especially as only a small proportion of devices awarded the designation are intended to treat ophthalmologic conditions1," said Lloyd Diamond, Chief Executive Officer of Pixium Vision.
  • This designation not only helps us to expedite the development of the Prima System but also affords us the opportunity of working closely with the FDA in refining the Prima System for its US regulatory submission.
  • In addition, after receiving market authorization, there are outpatient and inpatient reimbursement pathways that are more readily accessible as a result of receiving Breakthrough Device Designation."
  • The PRIMAvera study design is based on the positive data generated in a French feasibility study, showing the ability of patients with dry AMD to improve visual acuity with the Prima System.

Aldeyra Therapeutics Announces Positive Top-Line Results from 12-Month Safety Clinical Trial of Reproxalap in Patients with Dry Eye Disease

Retrieved on: 
Tuesday, February 28, 2023
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Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced top-line results from a 12-month, vehicle-controlled, multicenter, parallel-group safety clinical trial of reproxalap, an investigational new drug, in dry eye disease patients.

Key Points: 
  • Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced top-line results from a 12-month, vehicle-controlled, multicenter, parallel-group safety clinical trial of reproxalap, an investigational new drug, in dry eye disease patients.
  • The primary endpoints of treatment-related serious adverse events in ocular safety were not observed in any patient.
  • The 12-month safety clinical trial population was comprised of 447 dry eye disease patients; 299 patients were treated with reproxalap and 148 patients were treated with vehicle.
  • The detailed results of the clinical trial are expected to be presented at a major medical meeting.

Pixium Vision announces completion of implantations in the European pivotal trial PRIMAvera and confirms target read-out around the end of 2023 and regulatory submission in Europe in H1 2024

Retrieved on: 
Thursday, December 15, 2022
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A total of 38 patients have been implanted with the Prima System in the PRIMAvera study (NCT04676854), an open-label, baseline-controlled, non-randomized, multi-center, prospective, single-arm pivotal trial.

Key Points: 
  • A total of 38 patients have been implanted with the Prima System in the PRIMAvera study (NCT04676854), an open-label, baseline-controlled, non-randomized, multi-center, prospective, single-arm pivotal trial.
  • The study is being conducted in leading clinical centers in France, Germany, the UK, the Netherlands, and Italy.
  • A read-out of the PRIMAvera study's primary endpoints is expected around the end of 2023.
  • Pixium Vision is creating a world of bionic vision for those who have lost their sight, enabling them to regain visual perception and greater autonomy.

Editas Medicine Announces Clinical Data Demonstrating Proof of Concept of EDIT-101 from Phase 1/2 BRILLIANCE Trial

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Thursday, November 17, 2022
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ET

Key Points: 
  • ET
    CAMBRIDGE, Mass., Nov. 17, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical stage genome editing company, today announced clinical data from the Phase 1/2 BRILLIANCE trial of EDIT-101, an in vivo CRISPR/Cas9 genome editing medicine in a Company-sponsored webinar.
  • No other baseline characteristics that could pre-select a responder patient population were identified in the BRILLIANCE dataset.
  • As a result, Editas Medicine is pausing further enrollment in the BRILLIANCE trial and will continue long term follow-up of all patients who have been treated to date.
  • The results from the BRILLIANCE trial provide a proof of concept and important learnings for our inherited retinal disease programs.

KYS Vision Reports Clinical Trial Results of Its Remote Physiologic Monitoring System for Patients With Chronic Retinal Conditions

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Monday, October 31, 2022
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NEW YORK, Oct. 31, 2022 (GLOBE NEWSWIRE) -- KYS Vision, Inc., an eye care digital health company, today announced clinical trial results of its Macustat macular function scan for the remote monitoring of central retinal function.

Key Points: 
  • NEW YORK, Oct. 31, 2022 (GLOBE NEWSWIRE) -- KYS Vision, Inc., an eye care digital health company, today announced clinical trial results of its Macustat macular function scan for the remote monitoring of central retinal function.
  • The study is one of the first clinical trials to validate the clinical utility of real-world remote physiologic monitoring using an on-demand vision-as-a-service (VaaS) extension to the specialty retina clinic to enable remote monitoring of patients with chronic diseases such as AMD, diabetic retinopathy, and other retinal conditions.
  • Macustat is a part of a suite of cloud-based VaaS digital health applications which includes remote monitoring tests for glaucoma, AMD, DME, and other chronic conditions.
  • KYS Vision is a digital eye health company for remote patient monitoring and care management using a next-generation vision-as-a-service technology service model.

Pixium Vision announces reaching its enrollment target in the European pivotal trial PRIMAvera and implantations planned to be completed by end 2022

Retrieved on: 
Monday, September 19, 2022
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A total of 38 patients have been enrolled in the PRIMAvera study (NCT04676854), an open-label, baseline-controlled, non-randomized, multi-center, prospective single-arm pivotal trial.

Key Points: 
  • A total of 38 patients have been enrolled in the PRIMAvera study (NCT04676854), an open-label, baseline-controlled, non-randomized, multi-center, prospective single-arm pivotal trial.
  • The study was initiated in Q4 2020 in France, where the first patient was enrolled in December 2020.
  • Pixium Vision has since established additional clinical sites and implanted patients in France, Germany, the UK, the Netherlands, and Italy.
  • Implantations are due to be completed by the end of 2022, and a read-out of the PRIMAvera study's primary endpoints is expected around the end of 2023.