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Atsena Therapeutics Announces Dosing Completed in Part A of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis

Atsena Therapeutics Announces Dosing Completed in Part A of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis

 

DURHAM, NC, December 16, 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 dosing has been completed in Part A of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of 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.

“Nine adults have been treated in Part A of the study in which three dose levels of ATSN-201 are being evaluated for safety and efficacy. We have seen both structural and functional benefits in patients treated at all dose levels. Additionally, no serious adverse events related to treatment have been reported,” said Kenji Fujita, MD, Atsena’s Chief Medical Officer. “Collecting data from Part A of the trial is an important breakthrough for Atsena because it validates the use of our novel capsid to effectively treat inherited retinal disease and it informs the safest and most effective path forward for Part B. We look forward to providing further updates from those patients early next year.”

Currently, there are no approved treatments for XLRS which is typically diagnosed in early childhood and primarily affects males. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and ultimately leads to blindness. Approximately 30,000 males in the U.S. and EU have this inherited retinal disease.

“We are thrilled that dosing is complete in Part A of the LIGHTHOUSE study for ATSN-201 and that we’ve begun hearing positive anecdotes from those involved. This marks a significant milestone for patients with XLRS as we push to bring a therapeutic option to individuals that otherwise have no approved treatment,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “It also represents potential for individuals living with other inherited retinal diseases that could benefit from our novel capsid. We look forward to the insights gained from this trial as we pioneer new approaches to ocular gene therapy.”

The LIGHTHOUSE study is a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial evaluating the safety and tolerability of ATSN-201 in male patients ages six and older with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860). ATSN-201 has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration.

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, 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 genetic condition that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena’s program for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children, has completed a Phase 1 / 2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8) and is moving towards initiation of a pivotal trial. 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]

Nippon Shinyaku and Atsena Therapeutics Enter into an Exclusive Strategic Collaboration for ATSN-101 in the U.S. and Japan

Nippon Shinyaku and Atsena Therapeutics Enter into an Exclusive Strategic Collaboration for ATSN-101 in the U.S. and Japan

 

KYOTO, Japan and Durham, North Carolina, USA, November 13, 2024 – Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto; President, Toru Nakai) and Atsena Therapeutics, Inc. (Atsena; Headquarters: Durham, North Carolina, USA, Chief Executive Officer (CEO): Patrick Ritschel) have entered into an exclusive license agreement for the commercialization of ATSN-101 in the territory of the U.S. and for the development and commercialization of ATSN-101 in the territory of Japan for advancing Atsena’s first- in-class, investigational gene therapy ATSN-101 for Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1).

Under the terms of the licensing agreement, Nippon Shinyaku will receive exclusive commercial rights in the U.S. and Japan, and Atsena will retain commercial rights in the rest of the world. ATSN-101 will be marketed by NS Pharma, Inc. (New Jersey, USA, President: Yukiteru Sugiyama), a wholly owned subsidiary of Nippon Shinyaku in the U.S.

Atsena will receive an upfront payment, additional milestone payments, downstream royalties based on sales and will be reimbursed as it continues development work on ATSN-101, including an anticipated global pivotal trial.

ATSN-101 is a first-in-class, investigational gene therapy for the treatment of LCA1. Atsena has received Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for ATSN-101. In the event Atsena receives a Rare Pediatric Disease Priority Review Voucher (PRV) in connection with the approval of the Biologic License Application for ATSN-101, Atsena shall own and retain all rights, title and interest in such PRV.

“ATSN-101 provides a potential, innovative treatment in an area where no approved solutions currently exist,” said Nippon Shinyaku President Toru Nakai. “We are excited by the opportunity of this novel ocular gene therapy and our collaboration with Atsena and its groundbreaking science.”

“This collaboration creates a path to accelerate the development of ATSN-101 and validates Atsena’s pioneering technology and development capabilities. We anticipate this will be the first of many ocular gene therapy treatments from our clinical portfolio to come,” said Patrick Ritschel, CEO of Atsena Therapeutics. “We look forward to working with Nippon Shinyaku as we advance ATSN-101 into a pivotal trial and potential approval to provide an innovative solution to patients and families affected by LCA1 around the world.”

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. LCA1 is one of the most common forms of LCA and there are no approved treatments for it.

About ATSN-101
ATSN-101 is an investigational, subretinal AAV5 gene therapy being evaluated in an ongoing Phase I/II clinical trial for LCA1. At 12 months post-treatment, ATSN-101 has demonstrated durable, clinically meaningful improvements in vision at the high dose and is well-tolerated. Results from this trial were recently published in The Lancet.

About Rare Pediatric Disease Designation
The Rare Pediatric Disease Designation is granted by the FDA for promoting the development of new drugs for rare diseases that occur in the U.S. before the age of 18 and affect fewer than 200,000 people. With this designation, the product will be eligible to receive a Priority Review Voucher upon approval that could be used to advance another program.

About Regenerative Medicine Advanced Therapy Designation
The Regenerative Medicine Advanced Therapy (RMAT) Designation is an FDA-designated system for advanced regenerative medicine therapies targeted at developing serious diseases that have shown certain effect in clinical trials based on the 21st Century Cures Act in the U.S. Companies with RMAT designations are given the opportunity for priority review and accelerated approval for the product.

About Orphan Drug Designation
The Orphan Drug Designation is provided for orphan drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. With orphan designation, the FDA grants a seven-year market exclusivity for the product and provides certain incentives such as tax credits towards the cost of development, upon approval.

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. In an ongoing Phase I/II clinical trial, the company is evaluating ATSN-101 for LCA1, one of the most common causes of blindness in children. In another ongoing clinical trial, the company is evaluating ATSN-201 for X-linked retinoschisis (XLRS), a genetic condition affecting boys and men that is typically diagnosed in childhood. 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/.

About Nippon Shinyaku
Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness.

Please visit our website (https://www.nippon-shinyaku.co.jp/english/) for products or detailed information.

Atsena contacts:
Media: [email protected]
Business: [email protected]

Nippon Shinyaku contact:
[email protected]

Atsena Therapeutics to Present ATSN-201 Safety and Efficacy Data at the American Academy of Ophthalmology 2024 Annual

Atsena Therapeutics to Present ATSN-201 Safety and Efficacy Data at the American Academy of Ophthalmology 2024 Annual Meeting

 

DURHAM, NC, October 10, 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 safety and efficacy data on ATSN-201 for the treatment of X-linked retinoschisis (XLRS) will be presented during a panel discussion at the American Academy of Ophthalmology (AAO) 2024 Annual Meeting taking place October 18-21, 2024, in Chicago. 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.

Presentation details are as follows:

 

Session: First-time Results of Clinical Trials

Date and Time: Friday, October 18, 2024, 4:54 – 5:33 p.m. CDT

Presentation Title: Interim Safety and Efficacy of ATSN-201 Dose Escalation Study in Patients With X-linked Retinoschisis (XLRS)

Presentation Time: 5:08 p.m. CDT

Presenter: Christine Nichols Kay, MD, Clinical Ophthalmology Advisor, Atsena Therapeutics

 

The safety and tolerability of ATSN-201 is 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). ATSN-201 has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration.

 

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 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 bleb margins 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 bleb margins. This is in stark contrast to benchmark AAV vectors, which remain confined to those original 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 genetic condition affecting boys and men that is typically diagnosed in childhood and leads to blindness later in life. 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 to Present at Chardan’s 8th Annual Genetic Medicines Conference

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 8th 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/.

 

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

Business Contact:
[email protected]

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

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

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]

Atsena Therapeutics Receives Rare Pediatric Disease Designation from the U.S. FDA for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis

Atsena Therapeutics Receives Rare Pediatric Disease Designation from the U.S. FDA for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis

 Priority Review Voucher would be issued upon approval of ATSN-201

Second RPD and potential PRV for Atsena’s ocular gene therapy portfolio

 

DURHAM, NC, August 14, 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 Rare Pediatric Disease designation (RPD) 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 pleased to receive the FDA’s Rare Pediatric Disease designation for ATSN-201, which also marks the second RPD designation granted to Atsena this year. Having both of our clinical-stage, ocular gene therapies receive this designation underscores the potential of our technology to address significant unmet needs for patients with inherited retinal diseases,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “We are committed to advancing ATSN-201 in clinical trials and offering hope to patients and families affected by XLRS.”

Currently, there are no approved treatments for XLRS, which is typically diagnosed in early childhood and primarily affects males. Approximately 30,000 males in the U.S. and EU are affected by this condition. 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).

The FDA grants Rare Pediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases that primarily affect individuals under the age of 18. With this designation, ATSN-201 will be eligible to receive a priority review voucher (PRV) upon approval that could be used to advance another internal program or be sold to an outside company. The FDA’s PRV program greatly incentivizes companies like Atsena to invest in rare pediatric diseases.

 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 Appoints Joseph S. Zakrzewski as Board Chair

Atsena Therapeutics Appoints Joseph S. Zakrzewski as Board Chair

Biotech and pharma veteran brings expertise in therapeutic development and commercialization

 

DURHAM, NC, July 15, 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 the appointment of Joseph (“Joe”) S. Zakrzewski as Chair of its Board of Directors, effective immediately.

“We are thrilled Joe has joined our Board of Directors. As the Chair of our Board, his vast leadership experience and business acumen in the biotechnology and pharmaceutical industries will be an asset to Atsena,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “We look forward to his insights as we continue to advance our potential best-in-class treatments for inherited retinal diseases in the clinic.”

Throughout his distinguished 30-year career, Mr. Zakrzewski has founded several biotechnology companies and has held leadership positions including Chairman and CEO of Amarin Pharmaceuticals, Chairman, President & Chief Executive Officer of Xcellerex and Chief Operating Officer of Reliant Pharmaceuticals. Prior, Mr. Zakrzewski served in a variety of executive-level positions at Eli Lilly & Company for 17 years including R&D, manufacturing, finance and business development. He has served as a member of the board of directors of over 25 publicly and privately held companies, with many successful exits for shareholders. Mr. Zakrzewski holds a B.S. in Chemical Engineering and a M.S. in Biochemical Engineering from Drexel University, and an M.B.A. in Finance from Indiana University.

“I am honored to join Atsena’s Board and work with its experienced leadership team. The company’s commitment to developing innovative gene therapies aligns with my passion for advancing treatments that can significantly improve patients’ lives. With ongoing Phase I/II clinical trials and pioneering adeno-associated virus (AAV) technology, Atsena is driving remarkable progress in the field of ocular gene therapy,” said Mr. Zakrzewski. “I look forward to helping Atsena advance its mission to bring the transformative possibilities of genetic medicine to patients in need.”

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 Positive Clinical Data from the First Cohort of Phase I/II Trial Evaluating ATSN-201 Gene Therapy for the Treatment of X-linked Retinoschisis (XLRS)

Atsena Therapeutics Announces Positive Clinical Data from the First Cohort of Phase I/II Trial Evaluating ATSN-201 Gene Therapy for the Treatment of X-linked Retinoschisis (XLRS)

  • Subretinal injection of ATSN-201 was well tolerated in all patients in the first cohort with extensive resolution of schisis observed in two patients
  • Provides evidence that AAV.SPR spreads laterally outside of subretinal injection blebs in patients
  • Dosing in the mid-dose cohort is underway
  • Safety data from the first cohort will be presented at the Retinal Cell and Gene Therapy Innovation Summit 2024

DURHAM, NC, May 1, 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 positive preliminary data from the first cohort of the ongoing LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201 utilizes 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.

The LIGHTHOUSE study is evaluating ATSN-201 in male patients ages 6 and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. 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 impacts approximately 30,000 males in the U.S. and EU and there are no approved treatments.

In the first (low-dose) cohort of the LIGHTHOUSE study, two of the three patients showed extensive resolution of schisis beginning at 8 weeks after dosing. Additional and continued resolution of schisis was observed through week 24, the latest time point available. Notably, areas of schisis cavity resolution were found both inside and well outside of the subretinal injection blebs, indicating that AAV.SPR is spreading laterally from the injection blebs, consistent with expectations of this novel, laterally spreading capsid.

Early efficacy was also observed using microperimetry, with functional improvements seen in the same locations as structural improvements. Improvements of up to 14 dB were seen in one patient, with 38 loci showing an improvement of greater than 7 dB. The U.S. Food and Drug Administration considers an improvement of at least 7 dB at 5 or more prespecified loci to be clinically meaningful.

ATSN-201 was well tolerated in all three XLRS patients in the first cohort and no serious adverse events were reported. These results demonstrate, for the first time, the ability to safely administer subretinal injections in patients with extensive retinal schisis.

“The favorable safety profile and early efficacy observed in patients treated with ATSN-201 in the low- dose cohort of the LIGHTHOUSE study are very encouraging,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “We’re particularly pleased to have clinical validation of AAV.SPR’s ability to spread laterally well beyond the subretinal injection blebs. With dosing of patients in the mid-dose cohort underway, we look forward to the swift progression of this first clinical trial utilizing our novel spreading capsid and to the continued development of our best-in-class gene therapy candidate for XLRS patients who currently lack an approved treatment option.”

Safety data from the first cohort of the LIGHTHOUSE study will be presented at the Retinal Cell and Gene Therapy Innovation Summit 2024 being held Friday, May 3, 2024, in Seattle. Details are as follows:

Title: Preliminary safety of ATSN-201 subretinal gene therapy in patients with X-linked retinoschisis

Presenter: Christine Kay, MD, Clinical Ophthalmology Advisor, Atsena Therapeutics

Time: 11:05-11:20 a.m. PT

For more information about the Phase I/II open-label, dose-escalation, and dose-expansion clinical trial with four active study sites, 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 Initiation of Dosing in Second Cohort of Phase I/II Clinical Trial Evaluating ATSN-201 Gene Therapy for the Treatment of X-linked Retinoschisis (XLRS)

Atsena Therapeutics Announces Initiation of Dosing in Second Cohort of Phase I/II Clinical Trial Evaluating ATSN-201 Gene Therapy for the Treatment of X-linked Retinoschisis (XLRS)

  •  Subretinal injection of ATSN-201 was well tolerated in all patients in first cohort with no serious adverse events reported
  • Enrollment is ongoing in first clinical trial utilizing AAV.SPR, the company’s novel spreading capsid

 DURHAM, NC, March 13, 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 dosing has been initiated in the second cohort of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of 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.

ATSN-201 was well tolerated in all three XLRS patients in the first cohort and no serious adverse events were reported. The Data and Safety Monitoring Board recommended proceeding with the second cohort, in which patients will receive the next dose level of ATSN-201.

“We are encouraged by the excellent safety observed with the low dose of ATSN-201 in the LIGHTHOUSE study, which represents the first clinical utilization of AAV.SPR. Additionally, initial indications of structural and functional improvement observed within and beyond the injection areas suggest successful lateral spread of the vector,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “Advancing into the second cohort brings us one step closer to potentially bringing a much-needed one-time treatment to patients with XLRS, an inherited retinal disease that causes progressive vision loss and can lead to blindness.”

The LIGHTHOUSE study is a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial evaluating the safety and tolerability of ATSN-201 in male patients ages 6 and older with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. Enrollment in the LIGHTHOUSE 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://atsena.wpenginepowered.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://atsena.wpenginepowered.com/.

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

 Business Contact:
[email protected]

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