MYO7A

DURHAM, N.C., Jan. 16, 2024 (GLOBE NEWSWIRE) -- 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 U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to ATSN-101, the company’s investigational gene therapy being evaluated in an ongoing Phase I/II clinical trial in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1. RPD designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If a Biologics License Application for ATSN-101 for the treatment of LCA1 is approved by the FDA, Atsena may be eligible to receive a Priority Review Voucher that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred. “Rare Pediatric Disease designation is a significant milestone for our LCA1 program as we explore options to advance ATSN-101 into a pivotal clinical trial,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics. “The FDA designations that have been granted to ATSN-101 not only emphasize the tremendous need for a treatment for patients with LCA1, but also the potential for our subretinal gene therapy to be a major advance in reversing pediatric blindness.” Positive 12-month safety and efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101 will be presented at the 47th Annual Macula Society Meeting, which is being held February 7-10, 2024, in Palm Springs, CA. ATSN-101 has demonstrated clinically meaningful improvements in vision at the highest dose and is well-tolerated 12 months post-treatment. Details of the presentation are as follows: Title: Twelve-month safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1) Session: Inherited Retinal Dystrophy I (Treatment Trials) Date and Time: Wednesday, February 7, 2024, 6:04 p.m. PST Presenter: Christine Nichols Kay, MD 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 Atsena Therapeutics Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 12 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com. Media Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com Business Contact: info@atsenatx.com

ATSN-101 continues to demonstrate clinically meaningful improvements in vision at the highest dose and is well-tolerated 12 months post-treatment Data accepted for presentation at 47th Annual Macula Society Meeting DURHAM, N.C., Dec. 04, 2023 (GLOBE NEWSWIRE) -- 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 12-month safety and efficacy data from the ongoing Phase I/II trial of ATSN-101, the company’s investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1). At 12 months post-treatment, ATSN-101 has conferred clinically meaningful improvements in vision at the highest dose with no serious treatment-emergent adverse events. "We continue to be encouraged by the data emerging from our Phase I/II trial of ATSN-101 in patients with GUCY2D-associated LCA1,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “The durability of clinically meaningful visual improvements in the absence of serious treatment-related adverse events at the 12-month mark underscore the safety, tolerability, and efficacy of our subretinal gene therapy. We believe the 12-month findings provide solid proof of concept that ATSN-101 will exceed the requirements set by the U.S. Food and Drug Administration for ultimate approval. We are exploring partnering and out-licensing options to advance ATSN-101 into a pivotal trial.” In the Phase I/II trial, 15 patients ages 12 to 76 received unilateral subretinal injections of ATSN-101. Three adult cohorts (N=3 each) were treated with three ascending doses. Subsequently, one adult and one pediatric cohort (N=3 each) were treated at the high dose. In total, 9 patients received the high dose. At 12 months, there were no serious treatment-emergent adverse events. Ocular inflammation was mild and reversible with steroid treatment. For high-dose patients, the mean (SE) change from baseline in dark-adapted full-field stimulus testing (FST) (white stimulus) was greater in treated eyes compared with untreated eyes at all six follow-up visits, and some patients exhibited over 10,000-fold improvements in retinal sensitivity. Of the six high-dose patients who were tested with multi-luminance mobility testing (MLMT), three improved by ≥2 levels compared to baseline (when available) or to the untreated eye, and all three demonstrated a maximum score of 6 in the treated eye. At 12 months, patients receiving the high-dose demonstrated a statistically significant improvement in best-corrected visual acuity (BCVA). “The noteworthy improvements in key visual parameters demonstrate the potential of ATSN-101 to make a meaningful impact on the lives of patients affected by GUCY2D-associated LCA1,” said Paul Yang, MD, PhD, Associate Professor of Ophthalmology at OHSU School of Medicine. “As LCA1 causes early and severe vision impairment or blindness and there are no approved treatments, ATSN-101 has the potential to fill a significant unmet need within the LCA community.” The 12-month data have been accepted for presentation at the 47th Annual Macula Society Meeting, which will be held February 7-10, 2024, in Palm Springs, CA. The U.S. Food and Drug Administration (FDA) recently granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101. Atsena has also received orphan drug designation from the FDA for ATSN-101 for the treatment of LCA1. 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 Atsena Therapeutics Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 12 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com. Media Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com Business Contact: info@atsenatx.com

RMAT designation recognizes the potential of ATSN-101 to address unmet medical needs for patients with LCA1 ATSN-101 has demonstrated clinically meaningful improvements in vision at the highest dose with no drug-related serious adverse events 6 months post-treatment in ongoing Phase I/II clinical trial DURHAM, N.C., Nov. 14, 2023 (GLOBE NEWSWIRE) -- 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 U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101, the company’s lead investigational gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). RMAT designation was granted based on positive 6-month efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101. “Receiving RMAT designation from the FDA for ATSN-101 marks a significant regulatory milestone for Atsena, validating the potential of our subretinal gene therapy to improve vision and make a meaningful difference in the lives of patients with LCA1,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics. “As we continue to explore options to advance ATSN-101 into a pivotal clinical trial, we look forward to reporting 12-month data from our ongoing Phase I/II trial by the end of this year.” "There are no approved treatments for LCA1, an inherited retinal disease that results in early and profound vision impairment or blindness," said Jason Menzo, Chief Executive Officer of Foundation Fighting Blindness. "RMAT designation is encouraging recognition of the potential of ATSN-101 to be an important treatment and provides hope to children and adults affected by LCA1." Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for that disease or condition. RMAT designation provides sponsors with intensive FDA guidance on efficient drug development, including the ability to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA), and other opportunities to expedite development and review. Atsena has also received orphan drug designation from the FDA for ATSN-101 for the treatment of LCA1. 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-LCA 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 Atsena TherapeuticsAtsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 6 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com. Media Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com Business Contact:info@atsenatx.com