Alimera Sciences Acquires US Rights to Yutiq

■ Alimera Sciences announced its acquisition of additional commercialization rights for Yutiq (fluocinolone acetonide intravitreal insert) 0.18 mg for the treatment of chronic noninfectious uveitis affecting the posterior segment of the eye from EyePoint Pharmaceuticals, Inc.

“This is a landmark transaction for Alimera, bringing critical mass to our revenue base and leveraging the commercial infrastructure we have built in the US. We know this product very well, having marketed Iluvien for the uveitis indication in Europe and the Middle East for several years now,” said Rick Eiswirth, Alimera’s president and chief executive officer, in a news release. “EyePoint has done a wonderful job growing Yutiq to almost $30 million in revenue in 2022. We believe that deploying a larger commercial team focused on both Iluvien and Yutiq will increase utilization of both products moving forward.”

Alimera now has exclusive global rights to Yutiq excluding China, Hong Kong, Taiwan, Macau, South Korea, and Southeast Asia, where EyePoint has a pre-existing license with Ocumension Therapeutics.

Ophthalmic Companies Announce Merger

■ Medical Consulting Group (MCG), a provider of consulting services to ophthalmic practices and companies, and Corcoran Consulting Group (CCG), a company specializing in billing, coding, and reimbursement issues for ophthalmology and optometry practices, announced a definitive merger agreement. The companies say this merger will enhance the ophthalmic community by providing the broadest range of ophthalmic consulting and educational services in the eye-care marketplace.

Since 1986, CCG has served thousands of physicians in ophthalmic and optometric practices in all 50 states, providing reimbursement, chart audits, and coding education. MCG has been providing services to ophthalmic and optometric practices since 1989, having consulted with clinical practices in over 40 states and developed over 105 ambulatory surgery centers. MCG also provides practice management services, including revenue cycle management, accounting, benchmarking, human resources, and information technology. Both companies will continue to operate under their current names: Medical Consulting Group and Corcoran Consulting Group.

Iridex Launches New Pascal Laser With Micropulse

■ Iridex Corporation announced the full market launch of the next-generation Iridex Pascal (pattern scanning laser). The new platform provides an advanced technology package combining fast and precise pattern scanning capabilities, Endpoint Management Technology, Pattern Scanning Laser Trabeculoplasty, and micropulse technology in a smaller, ergonomically optimized integrated laser platform.

The new laser offers a smaller physical footprint, intuitive touchscreen controls, and wheelchair accessibility compliant with the Americans With Disabilities Act. The Pascal product line continues to efficiently deliver large laser patterns, reducing treatment time and patient discomfort during treatment. The innovative 4-fiber beam design improves depth of focus during treatment, ensuring consistent energy delivery across multiple tissue elevations and in the far periphery of the retina. Intuitive user interface provides easy access to a wide variety of pattern variations, and the auto advance feature delivers sequential patterns without taking physician’s eyes away from oculars. The Iridex Pascal is currently available in 577 nm (yellow) or 532 nm (green) models.

B+L and Heidelberg Introduce SeeLuma Surgical Visualization Platform

■ Bausch + Lomb and Heidelberg Engineering announced the introduction of the SeeLuma Fully Digital Surgical Visualization Platform. SeeLuma features include a heads-up 3D monitor, multiple digital display options, and an intuitive interface designed to offer excellent visualization, ergonomics, smooth workflow, and an immersive educational experience. SeeLuma was introduced in the United States and Western Europe in April, with additional countries to follow.

The platform facilitates 3D heads-up surgery using next-generation 3D 55” and 31” 4K monitors, as well as unique digital binoculars. In addition, embedded functions allow the user to highlight and enhance anatomic landmarks to better distinguish anatomical features.

“Having access to a platform that offers cutting-edge visualization, efficient workflow and ergonomics, and educational features has the potential to really change the landscape and ultimately deliver tremendous benefits for current and future generations of surgeons, as well as their practices and patients,” Siegfried Priglinger, professor, director, and chairman of University Eye Hospital of the Ludwig-Maximilians-Universität in Munich, Germany, said in a news release.

FDA Clears Aviceda’s IND Application for Novel Geographic Atrophy Treatment

■ Aviceda announced that the FDA has cleared the Investigational New Drug (IND) application for AVD-104, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). This enables the company to proceed with the phase 2 SIGLEC clinical trial, which is expected to begin in Q2 2023. AVD-104 is an intravitreal nanoparticle molecule with a unique dual mechanism of action for the treatment of GA.

“The FDA’s acceptance of our IND application to initiate the clinical evaluation of AVD-104 brings us one step closer to introducing a potentially paradigm-shifting treatment for people living with GA and macular degeneration,” Mohamed Genead, MD, Aviceda’s cofounder, president, and CEO, said in a news release.

Atsena Receives FDA Clearance of IND Application for ATSN-201

■ Atsena Therapeutics announced that the FDA has cleared the company’s IND application for the Lighthouse study, a phase 1/2 clinical trial of ATSN-201 in patients with X-linked retinoschisis (XLRS). The Lighthouse study will evaluate subretinal injection of ATSN-201 in male patients ages 6 to 65, with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. ATSN-201 leverages one of the company’s novel spreading capsids, AAV.SPR, in its treatment of XLRS.

“AAV.SPR is well suited for use in XLRS, as it can drive therapeutic levels of gene expression in photoreceptors while avoiding the surgical risks of foveal detachment, which is important because XLRS patients have fragile retinas due to the presence of schisis lesions. We’re excited to progress our novel gene therapy for patients with XLRS who currently lack an approved treatment option,” Shannon Boye, PhD, founder and director of Atsena Therapeutics, said in a news release.

Adverum Biotechnologies Presents Nonclinical Data on Ixoberogene Soroparvovec Gene Therapy in Support of Bilateral Dosing

■ Ixoberogene soroparvovec (Ixo-vec; formerly ADVM-022) is a gene therapy designed to produce robust, durable levels of aflibercept for the treatment of neovascular AMD. A phase 1 clinical trial showed a single intravitreal injection of Ixo-vec at 6E11 and 2E11 vg/eye resulted in therapeutic levels of aflibercept in patients through 3 years. Data were presented at the 2023 Association for Research in Vision and Ophthalmology meeting from a nonclinical study that evaluated whether a lower dose of Ixo-vec can provide robust ocular aflibercept levels, and whether AAV-induced adaptive immunity after the first dose could have an impact on treatment of the fellow eye. This presentation demonstrated Ixo-vec does provide robust, durable aflibercept levels with minimal inflammation and is well tolerated with second eye administration. A Good Laboratory Practices study with lower doses of 1E11 and 3E10 vg/eye (HED 2E11 and 6E10 vg/eye) supports dose choices for an ongoing phase 2 trial, and nonhuman primate data suggest feasibility of bilateral staggered administration of Ixo-vec.

Apellis Presents Post Hoc 24-Month Phase 3 Syfovre Data at ARVO

■ Apellis Pharmaceuticals presented post hoc analyses from the 24-month, phase 3 OAKS and DERBY studies evaluating Syfovre (pegcetacoplan injection) for the treatment of GA secondary to age-related macular degeneration (AMD), at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting in New Orleans.

In the 24-month analysis, Syfovre-treated patients compared to sham demonstrated preservation of 5.6 letters, as measured by best corrected visual acuity (BCVA), and a 4.1-point benefit in vision-related quality-of-life outcomes, as measured by the National Eye Institute Visual Function Questionnaire (NEI-VFQ-25).

“Vision loss caused by GA can profoundly impact a person’s independence and well-being, so it is vital that Syfovre has shown slower vision loss and better quality of life compared to sham in this post hoc analysis. These data also support earlier treatment with Syfovre,” Allen Chiang, MD, who presented the data at ARVO, said in a press release. Dr. Chiang is associate professor of ophthalmology at Wills Eye Hospital, Mid Atlantic Retina, and Thomas Jefferson University.

HuidaGene Receives Orphan Drug Designation for Gene Therapy

■ HuidaGene Therapeutics announced that the FDA has granted orphan drug designation (ODD) for HG004, the company’s gene replacement therapy for the treatment of inherited retinal disease caused by RPE65 mutations. The ODD designation will accelerate drug development and registration action in the United States. The designation affords HuidaGene the potential for benefits, such as tax credits for clinical development, exemptions for certain FDA application fees, 7 years of postapproval market exclusivity, and assistance in the drug development process. In January 2023, the FDA cleared an IND application for a multinational clinical trial of HG004.

“Receiving ODD is an important milestone as we are advancing our HG004 gene replacement therapy program to clinical trials designed to provide safe, durable, and high-quality treatment to children and adults suffering from RPE65 mutation–associated inherited retinal diseases,” Xuan Yao, PhD, cofounder and chief executive officer of HuidaGene, said in a news release.

Nanoscope Reports Positive Results From Phase 2b RESTORE RP Trial

■ Nanoscope Therapeutics announced positive topline results from the phase 2b multicenter, randomized, double-masked, sham-controlled RESTORE clinical trial of MCO-010, an ambient-light activatable multicharacteristic 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 injection. Vision function improvements after treatment with MCO-010 were consistent with previous studies. The primary outcome measure was mean change in a multiluminance y-mobility test (MLYMT) score vs placebo. At 12 months, the change in MLYMT score difference vs placebo was +1.0 (95% CI 0.0, 3.0).

“These participants who were living with severe vision impairment due to RP now have an improved quality of life very different from before receiving MCO-010,” Victor H. Gonzalez, MD, RESTORE investigator and founder of Valley Retina Institute in McAllen, Texas, said in a news release.

FDA Agrees to Allegro Ophthalmics Risuteganib Dry AMD Trial Design

■ Allegro Ophthalmics announced that it has received agreement from the FDA under a special protocol assessment (SPA) for the design of its phase 2b/3 clinical trial of risuteganib (Luminate) for the treatment of intermediate dry AMD. Finalization of this assessment indicates that the FDA agrees that critical elements of the overall protocol design, such as entry criteria, dose selection, endpoints, and planned analyses, are adequate and acceptable for a study intended to support a future marketing application. The phase 2b/3 study design is based on Allegro’s successfully completed phase 2a study, in which the primary endpoint was the proportion of subjects with a gain of ≥8 letters of vision with 2 risuteganib injections vs 1 sham treatment.

“The agreement by the FDA of the SPA provides a clear regulatory path for our first-in-class drug, risuteganib, for the treatment of intermediate dry AMD for an unmet medical need,” said Vicken Karageozian, MD, president and CEO of Allegro Ophthalmics, in a news release.

First Participant Dosed in Phase 2 HELIOS Wolfram Syndrome Trial

■ Amylyx Pharmaceuticals announced that the first participant has been dosed in the HELIOS study, a phase 2 clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of Wolfram Syndrome (WS). Researchers from the Washington University School of Medicine in St. Louis, in collaboration with Amylyx, recently published preclinical data exploring the potential of AMX0035 as a novel therapeutic approach for WS.

“Preclinical data showed that a combination treatment of 2 chemicals, namely AMX0035, may restore cellular functioning in a cellular model of WS. We look forward to building upon our preclinical findings as part of HELIOS to explore the safety, tolerability, and preliminary clinical activity in people living with WS,” Fumihiko Urano, MD, PhD, principal investigator of the HELIOS clinical trial, and professor of medicine and of pathology and immunology in the division of endocrinology, metabolism and lipid research at Washington University School of Medicine, said in a news release.

Samsung Bioepis Proposed Biosimilar Data Presented at ARVO

■ Samsung Bioepis announced that 1-year outcomes of the phase 3 study for SB15, a proposed biosimilar to aflibercept (Eylea; Regeneron), were presented at the 2023 ARVO annual meeting in New Orleans. Researchers reported SB15 demonstrated comparable efficacy, safety, immunogenicity, and pharmacokinetics profiles to reference aflibercept up to week 56. New analytical data on similarity between SB15 and reference aflibercept (AFL), in terms of structural and physicochemical properties and biological functions, were also presented.

“The presentations showcase our ongoing commitment in ophthalmology to bring more access to treatments for patients suffering from retinal vascular diseases. Biosimilars are relatively new in ophthalmology compared to other therapeutic areas, and we will continue to advance with our scientific research, publication, and educational activities to bring more awareness of biosimilars among ophthalmologists,” said Jin-Ah Jung, medical affairs group leader at Samsung Bioepis, in a news release.

FDA Grants Breakthrough Device Designation for Prima System in Dry AMD

■ Pixium Vision announced that the FDA has granted breakthrough device designation (BDD) to the company’s Prima System. The Prima System is a photovoltaic implant that provides simultaneous use of the central prosthetic and peripheral natural vision in patients with atrophic dry age-related macular degeneration (AMD) to partially restore their vision. To receive BDD, a device must have either breakthrough technology, or no approved or cleared alternatives, or significant advantages over existing approved or cleared alternatives, or its availability must be in the best interest of patients.

“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 BDD,” Lloyd Diamond, chief executive officer of Pixium Vision, said in a news release.

Study Shows “Dormant” Cone Photoreceptors May Retain Visual Function

■ New UCLA research in mice suggests that photoreceptors in the degenerating retina that are considered dormant are not dormant at all, but instead continue to function, producing responses to light and driving retinal activity for vision. The study indicates for the first time that the cells are still viable, and it includes recordings of downstream signals from the retina showing that visual processing was not as compromised as expected. The authors say their findings demonstrate that therapeutic interventions to protect these cells, or enhance their sensitivity, have the capability to preserve nearly normal daytime vision.

“These important results may suggest a future path forward for patients with conditions thought to be causing irreversible retinal blindness, as photoreceptor or cone viability in tissue was previously thought to be irreparably damaged,” Steven Schwartz, MD, said in a news release. Dr. Schwartz is the Ahmanso Chair in Ophthalmology at the David Geffen School of Medicine at UCLA, and professor and retina division chief at the UCLA Jules Stein Eye Institute.

Endogena Completes Dose Escalation Stage of Phase 1/2a RP Trial

■ Endogena Therapeutics announced that the dose-escalation stage of its phase 1/2a study of EA-2353 in retinitis pigmentosa (RP) has been successfully completed. No clinically relevant or dose-limiting adverse events were identified after repeated intravitreal injections. Given the positive safety and tolerability profile, the study will now enroll patients into the expansion cohort, using the highest dose evaluated in the study to explore the potential efficacy of the compound. A total of 14 patients with RP have been recruited across up to 6 US sites. The first patient was dosed in July 2022 and 9 patients have been treated to date during the dose-escalation phase.

“I’m very encouraged by the safety and tolerability profile in this dose-escalation stage. We have the best people working tirelessly to bring this novel treatment to the patients who need it as fast as possible,” Matthias Steger, PhD, MBA, chief executive officer of Endogena, said in a news release.

Promise for Leber Congenital Amaurosis

■ A National Eye Institute (NEI)–led team has identified a compound already approved by the FDA that keeps light-sensitive photoreceptors alive in 3 models of the inherited retinal ciliopathy disease, Leber congenital amaurosis type 10 (LCA10). Using a mouse model of LCA10 and 2 types of lab-created tissues from organoid stem cells, the team screened more than 6,000 FDA-approved compounds to identify ones that promoted survival of photoreceptors. The high-throughput screening identified 5 potential drug candidates, including reserpine, which was approved in 1955 for the treatment of high blood pressure. The researchers found that reserpine targets dysregulated intracellular signaling pathways downstream of the primary cilium.

“Patients suffering from vision loss due to photoreceptor cell death might eventually benefit from our findings, as small molecule drugs represent a relatively affordable and scalable option,” Anand Swaroop, PhD, chief of the NEI Neurobiology Neurodegeneration and Repair Laboratory, said in a news release.

Extremely Rare Gene Variants Point to a Potential Cause of AMD

■ Scientists from the National Eye Institute (NEI) identified rare genetic variants that could point to one of the mechanisms driving AMD. The findings, published in the journal iScience, point to membrane attack complex (MAC) as a potential therapeutic target to slow or prevent the development of AMD.

The research revealed that in 4 families, individuals with AMD have mutations in 1 of 2 proteins that form 1 end of MAC: C8-alpha and C8-beta. The team found that the variants from the 4 AMD families all affected the ability of the C8 proteins to stick to each other, which may alter how MAC behaves in the retina. In a news release, the researchers noted, “By looking at large families with ultra-rare variants that track closely with disease across generations, we found 2 proteins that may directly be the driving force behind AMD pathology in affected patients. These proteins could be targets for future drugs.”

Blood Pressure Drug Shows Eye Color Genes Are Critical for Retinal Health

■ Four genes that code for eye color also play a separate role in maintaining retinal tissue health, according to researchers from the Max Planck Institute of Molecular Cell Biology and Genetics (MPI-CBG) in Germany. Their study, which examined the role of 4 Drosophila genes, revealed that the genes regulate the kynurenine metabolic pathway independent of their role in eye color pigmentation. The findings indicate that, in certain circumstances, retinal health can be improved by altering the ratio of metabolites of the kynurenine pathway.

In a news release, Elisabeth Knust, director emerita of MPI-CBG, who managed the study, said, “In the future, the ratio of the various metabolites and the specific sites of their accumulation and activity should be taken into account in therapeutic strategies for diseases with impaired Kynurenine pathway function, observed in various neurodegenerative conditions.”

Phase 2 Results of UBX1325 in Wet AMD and DME Released

■ Unity Biotechnology announced results from part A of the phase 2 ENVISION study of UBX1325, in patients with wet AMD who were not achieving optimum benefit with their ongoing anti-VEGF therapy. UBX1325 treatment generally maintained visual acuity for 6 months (change of -0.8 ETDRS letters from baseline), with most patients not requiring any anti-VEGF rescue. Patients in the every-8-week aflibercept arm had an early and unexpected gain of 3.5 letters at week 2, which was mostly maintained for the duration of the study. However, as a result of the strength on the control arm, the study did not meet the noninferiority threshold compared to aflibercept through 24 weeks.

Unity Biotechnology also announced positive results from the long-term follow-up of the phase 2 BEHOLD study of UBX1325, in patients with DME. A single injection of UBX1325 treatment led to a statistically significant improvement in vision lasting for the duration of the study (48 weeks), marked by a gain of +6.2 ETDRS letters from baseline, representing a difference of +5.6 ETDRS letters compared to sham-treated patients. In addition, patients treated with UBX1325 maintained stable CST compared to worsening in sham-treated patients.

“Based on the strong emerging clinical profile of UBX1325, we are planning to move forward with our phase 2b DME head-to-head study against aflibercept in the second half of 2023,” Anirvan Ghosh, PhD, chief executive officer of Unity Biotechnology, said in a news release.

Recently Acquired Iveric Bio Reports Newest GATHER Data at ARVO

■ Iveric Bio, which was recently acquired by Japan-based Astellas Pharma, announced new findings from exploratory analyses of data for avacincaptad pegol (ACP), at the annual ARVO meeting in New Orleans. A post hoc analysis from the GATHER1 and GATHER2 pivotal phase 3 clinical trials, presented by Carl Danzig, MD, director of vitreoretinal services at Rand Eye Institute in Deerfield Beach, Florida, showed a relationship between GA growth and worsening vision loss. In this combined analysis, greater vision loss was correlated with increased GA growth.

“This is the first time a relationship between disease progression and worsening visual acuity has been observed in GA, connecting anatomy and function. These data suggest that in the ACP-treated group, the reduction in growth of GA resulted in an overall lower rate of vision loss,” Dr. Danzig said in a news release. As previously reported, the post hoc analysis of GATHER1 and GATHER2 combined data signaled a 56% risk reduction in the rate of persistent vision loss in GA patients receiving ACP 2 mg compared to sham over the first 12 months of treatment.

In the acquisition of Iveric Bio, Astellas Pharma has agreed to acquire 100% of the outstanding shares of Iveric. “We are pleased to reach an agreement with Iveric Bio, a company with exceptional expertise in the R&D of innovative therapeutics in the ophthalmology field. We believe that this acquisition will enable us to deliver greater value to patients with ocular diseases at high risk of blindness,” said Naoki Okamura, president and CEO of Astellas.

Regeneron Presented New Aflibercept 8 mg and Eylea Data at ARVO

■ Regeneron Pharmaceuticals presented new data for aflibercept 8 mg and Eylea (aflibercept 2 mg) at the annual ARVO meeting in New Orleans. Notable presentations highlighted the pivotal aflibercept 8 mg PULSAR and PHOTON trials, respectively, in wet AMD and DME with 48-week efficacy and safety results, in addition to an evaluation of baseline characteristics of patients randomized to aflibercept 8 mg who maintained their dosing intervals and those whose dosing intervals were shortened.

One of the 18 ARVO Regeneron presentations was made by David Brown, MD, Retina Consultants of Texas, on behalf of the PHOTON investigators. Dr. Brown reported that most patients with DME who received aflibercept 8 mg maintained 12-week or 16-week dosing, and that patients who did not maintain their randomized dosing intervals appeared to have more severe disease at baseline than patients who maintained the randomized dosing intervals, and that this trend was more pronounced in the 8q16 group. A presentation offered by Diana Do, MD, of Byers Eye Institute at Stanford University School of Medicine, revealed that aflibercept 8 mg met the primary efficacy endpoint in DME, demonstrating noninferiority in BCVA vs aflibercept 2 mg, with no new safety signals through 48 weeks. A presentation by Ghassan Ghorayeb, MD, Eye Institute, West Virginia University, revealed that aflibercept 8 mg achieved meaningful BCVA gains from baseline at week 48 in patients with DME across evaluable subgroups of sex, age, race, and ethnicity.

In a news release, Boaz Hirshberg, MD, senior vice president of clinical sciences general medicine at Regeneron, said, “Our data presentations at ARVO build on the more than 20 years of industry-leading knowledge.” RP