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Atsena Therapeutics to Present at Chardan’s 8th Annual Genetic Medicines Conference

 

DURHAM, NC, September 24, 2024 – Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that it will present at Chardan’s 8^th Annual Genetic Medicines Conference taking place September 30 – October 1, 2024, in New York City. Chief Executive Officer Patrick Ritschel will deliver a company presentation on Tuesday, October 1, 2024, at 11:30 a.m. ET. Atsena Chief Medical Officer, Kenji Fujita, MD, and Mr. Ritschel will also participate in one-on-one meetings with potential investors during the conference.

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a progressive genetic condition affecting boys and men that is typically diagnosed in childhood. Another ongoing Phase I/II clinical trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit  https://atsenatx.com/.

 

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Tony Plohoros
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Atsena Therapeutics Receives Orphan Drug Designation from the U.S. FDA for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis

Marks second FDA designation for ATSN-201, which previously received Rare Pediatric Disease designation

 

DURHAM, NC, September 17, 2024 – Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.

“We are honored to receive the FDA’s Orphan Drug Designation for ATSN-201, which was also recently granted Rare Pediatric Disease designation. These designations mark a significant inflection point for the potential of this ocular gene therapy in an inherited retinal disease that currently has no available treatments,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “The Atsena team is passionate and committed to our ongoing work on the XLRS program. We look forward to bringing hope to patients affected by this rare disease and are confident these designations will expedite our path forward.”

The FDA grants Orphan Drug Designation to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain incentives, such as tax credits toward the cost of clinical trials upon approval and prescription drug user fee waivers. If a product receives Orphan Drug Status from the FDA, that product is entitled to seven years of market exclusivity for the disease in which it has Orphan Drug designation, which is independent from intellectual property protection.

Currently, there are no approved treatments for XLRS, which is typically diagnosed in early childhood and affects approximately 30,000 males in the U.S. and EU. The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial in male patients ages six and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860).

 

About X-linked Retinoschisis (XLRS)

XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments.

 

About AAV.SPR

AAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in stark contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and does not cause inflammation. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/.

 

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a progressive genetic condition affecting boys and men that is typically diagnosed in childhood. Another ongoing Phase I/II clinical trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit  https://atsenatx.com/.

 

Media Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
[email protected]

Business Contact:
[email protected]

Atsena Therapeutics Announces 12-Month Safety and Efficacy Data from Phase I/II Clinical Trial of ATSN-101 in LCA1 Published in The Lancet

First time patients with LCA1 have been treated with gene therapy

 ATSN-101 demonstrated durable, clinically significant improvements in vision at the high dose and was well-tolerated 12 months post-treatment in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D

 

DURHAM, NC, September 5, 2024 – Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that 12-month safety and efficacy data from Atsena’s Phase I/II clinical trial evaluating the investigational gene therapy ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1) were published in The Lancet. ATSN-101 was originally developed by Atsena’s founders, Shannon and Sanford Boye at the University of Florida, and is the first gene therapy being studied to treat patients with LCA1.

“There are no approved treatments for LCA1, an inherited retinal disease that affects the retina and causes severe vision impairment or blindness in infancy,” said Artur Cideciyan, PhD, Research Professor of Ophthalmology at the Scheie Eye Institute of the University of Pennsylvania and senior author of the paper. “The improvements in visual function and tolerability we saw at the high dose of ATSN-101 at 12 months post-treatment in the ongoing Phase I/II trial support a future randomized, controlled Phase III trial to further evaluate this subretinal gene therapy in patients with LCA1. ATSN-101 represents a significant advancement in the reversal of blindness that begins in childhood.”

All 15 enrolled patients with genetically confirmed LCA1 received unilateral subretinal injections to determine the safety and preliminary efficacy of ascending doses of ATSN-101. Thirteen of the patients were treated at the Scheie Eye Institute. Two were treated at the Oregon Health & Science University (OHSU) Casey Eye Institute, under the guidance of Paul Yang, MD, PhD, and Andreas Lauer, MD. In the dose escalation phase, three adult cohorts (n=3 each) were treated with three ascending doses: 1.0E10 vg/eye (low dose), 3.0E10 vg/eye (mid dose), and 1.0E11 vg/eye (high dose). In the dose expansion phase, one adult and one pediatric cohort (n=3 each) were treated at the high dose. The primary endpoint of the Phase I/II clinical trial is the incidence of treatment-emergent adverse events (TEAEs), and secondary endpoints include full-field stimulus test (FST) and best-corrected visual acuity (BCVA). A multi-luminance mobility test (MLMT) was also performed.

“This is the first time patients with LCA1 have been treated with gene therapy, making this promising treatment potentially the first-ever gene therapy for this inherited retinal disease,” said Paul Yang, MD, PhD, Associate Professor of Ophthalmology in the OHSU School of Medicine and lead author of the paper. “We are excited to have these results published in The Lancet, a top-tier medical journal, which helps broaden the reach of the important work underway at Atsena to the wider medical community.”

For high-dose subjects, mean change in dark-adapted FST was 20.3 decibels, representing a 100-fold improvement for treated eyes. Improvements were first observed at day 28 and persisted over 12 months (p = 0.012). Modest improvements in BCVA were also observed with an average improvement of -0.16 logMAR or 8 letters 12 months post-treatment for high-dose subjects (p = 0.10). Three of six high-dose subjects that performed MLMT achieved the maximum score in the treated eye at Month 12.

All adverse events were either mild (91%) or moderate (9%) in severity, and TEAEs (including serious adverse events) were similar to those reported in previous subretinal gene therapy studies. No serious TEAEs were related to ATSN-101, and the majority of TEAEs were related to the surgical procedure. Ocular inflammation was mild and reversible with steroid treatment.

“The preliminary efficacy and robust safety data from our ongoing Phase I/II trial underscore the potential of ATSN-101 to be a first-in-class gene therapy for LCA1. It is encouraging that improvements in retinal sensitivity were observed as early as 28 days post-treatment and persisted over at least 12 months, highlighting the durability of our investigational gene therapy,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics and co-author of the paper. “We are thrilled that the 12-month data have been published for the first time in this prestigious medical journal.”

The published manuscript, titled “Safety and Efficacy of ATSN-101 in Patients with Leber Congenital Amaurosis caused by Biallelic Mutations in GUCY2D: A Phase 1/2, Multi-Center, Open-Label, Unilateral Dose Escalation Study,” is available online and will appear in the print issue of The Lancet at a later date.

 

About GUCY2D-associated Leber congenital amaurosis (LCA1)
LCA1 is a monogenic eye disease that disrupts the function of the retina. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1.

About ATSN-101

ATSN-101 is an investigational, subretinal AAV5 gene therapy being evaluated in an ongoing Phase I/II clinical trial for Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). ATSN-101 was originally developed at the University of Florida and introduces functional human GUCY2D to photoreceptors. At 12 months post-treatment, ATSN-101 has demonstrated durable, clinically meaningful improvements in vision at the high dose and is well-tolerated. Atsena has received Rare Pediatric Disease designation, Regenerative Medicine Advanced Therapy designation, and Orphan Drug designation from the U.S. Food and Drug Administration for ATSN-101 for the treatment of GUCY2D-associated LCA1.

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a progressive genetic condition affecting boys and men that is typically diagnosed in childhood. Another ongoing Phase I/II clinical trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit  https://atsenatx.com/.

 

Media Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
[email protected]

Business Contact:
[email protected]