Lightstone Ventures’ Post

We are excited to share data from the first cohort of the LIGHTHOUSE study, our ongoing Phase I/II clinical trial evaluating our investigational #GeneTherapy ATSN-201 for the treatment of X-linked retinoschisis (#XLRS). For the first time in patients, we have validated the ability of AAV.SPR, our novel spreading #capsid, to spread laterally well beyond the subretinal injection blebs. Additionally, we have demonstrated the ability to safely administer subretinal injections in patients with extensive retinal schisis. Subretinal injection of ATSN-201 was well tolerated in all three patients in the first cohort with two patients showing 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. Read more about the data in our press release: https://lnkd.in/eAcpdUHS

Occlusive Retinal Vasculitis Following Pegcetacoplan Interview with Srinivas Sai A. Kondapalli MD "The GALE study was a 36-month open-label extension of the OAKS and DERBY studies. If you remember, OAKS and DERBY are phase III clinical trials evaluating pegcetacoplan, or Syfovre, in the treatment of geographic atrophy, which is currently FDA-approved in the United States. Pegcetacoplan recently came onto market as Syfovre, and the real-world safety data have been presented recently at the ASRS 2023 meeting. There were found to be 7 cases of occlusive retinal vasculitis, which have been confirmed both by the drug company as well as the ReST committee. Of note, and especially troubling, is the fact that occlusive retinal vasculitis results in significant vision loss. So, this safety signal is one that I think is troubling physicians like myself in terms of its adoption. It's yet to be determined why the occlusive retinal vasculitis is happening, and I think that if that can be determined and, if we can avoid that in the future, it would definitely help in terms of patients receiving the medication in the future." More insights available here: https://lnkd.in/dh_yj-25

Phase 2 clinical trial update on NETs! 📊 Lenvatinib and pembrolizumab combo in advanced GI and thoracic NETs showed 10% partial response, with median PFS of 8 months. Also, 60% experienced grade 3 adverse events, leading to dose adjustments or discontinuation. The trial did not show sufficient response to be continued. More details in the article: https://ow.ly/hwrG50R0Vbj #MedEd

#ICYMI, our ADG126 masked anti-CTLA-4 SAFEbody® is in the news with a strong efficacy signal in MSS CRC! Enabled by a safety profile for higher, more frequent and repeat dosing, we are evaluating ADG126 plus pembrolizumab in a homogenous patient group of advanced/metastatic MSS CRC patients. Following Simon’s two-stage statistical design for a single arm phase 2 trial, we’re excited to announce a strong efficacy signal was observed in the first stage at the ADG126 10 mg/kg every three weeks dosing regimen. The study is advancing to now enroll ten more patients in the second stage, at this active dosing regimen. Check out the recent update: https://loom.ly/xpvFdXA https://loom.ly/xpvFdXA

In the latest article of Clinical Trials Arena, Halia’s CEO David Bearss, Ph.D., spoke about the Company starting a Phase II clinical trial with their lead product, HT-6184, in myelodysplasia syndrome (MDS) early next year. The trial is aimed at improving MDS patient hemoglobin and to measure improvement in transfusion dependency. The site selection for the trial will initially be conducted initially outside the US, but the Company plans to expand to the US as well as other countries in Asia as well as Europe. To read about the full Phase II trial details, and the better-than-expected results of their Phase I trial visit: https://lnkd.in/gmmzeTQV #inflammasome #NLRP3 #clinicaltrial #clinicalstudy #myelodysplasiasyndrome #MDS #MDStreatment #transfusion

Surprising decision at FDA to appove a new drug dedicated to #Duchenne Musculary Distrophy #DMD which failed to validate its primary endpoint. In its announcement the FDA states: "In making this decision, the FDA considered the totality of the #evidence, including the potential risks associated with the product, the life-threatening and debilitating nature of the disease and the urgent medical #unmetneeds." This is in line with the recommandation of the guidance dedicated to this disease: " "Sponsors should engage FDA early during the selection and/or development of efficacy endpoints. The sponsor should include an assessment of #multipleefficacyendpoints, when feasible, to characterize the breadth of effects on dystrophin-related pathologies, including skeletal, respiratory, and cardiac muscle function, even if the primary endpoint is only one of these measures. " Innovative drug will face more and more the need to assess #multipleoutcomes and uncover their overall #nettreatmentbenefit for the benefit of the patients https://lnkd.in/emDTbqUd

⚡ Nivolumab plus platinum-doubletchemotherapy in treatment-naive patientswith advanced grade 3 NeuroendocrineNeoplasms of gastroenteropancreatic orunknown origin: The multicenter phase 2NICE-NEC trial (GETNE-T1913) 🦓 #NeuroendocrineTumors https://buff.ly/46MiN8T

We're still reflecting on the incredible scientific advancements shared in 2023. Dr. Erika Hamilton joined VuMedi to recap #SABCS23 Insights: "CAMBRIA 1 Study of Extended Adjuvant Therapy With Camizestrant vs. Standard ET in Patients With ER+/HER2- Early BC and an Intermediate/High Risk of Recurrence." Watch the full video on VuMedi. https://lnkd.in/eD4pdZqd #ClinicalTrials #OncologyResearch #BCSM #OncologyExpert

Opthea Completes Enrollment in First Pivotal Trial with Sozinibercept Today, Opthea announced that it has completed enrollment of all patients in the COAST Phase 3 pivotal clinical trial investigating sozinibercept (OPT-302, a vascular endothelial growth factor (VEGF)-C/D inhibitor) in combination with aflibercept, an anti-VEGF-A therapy, for the treatment of wet Age-related Macular Degeneration (wet AMD). The sozinibercept clinical program includes two Phase 3 pivotal trials, COAST and ShORe. Enrollment in ShORe is expected to be completed in calendar Q2 2024. Opthea intends to report topline results from these two trials by mid-2025. #maculardegeneration #ophthalmology #retinalspecialists #progress #wetAMD #WetAMDClinicalTrials #wetAMDstudies #clinicaltrials Frederic Guerard Ian Leitch Peter Lang Judy J. Robertson Fang Li, PhD RAC Julie Clark Bruno Gagnon Sarah Weeks Ashima Brar, MD, MBA Silvana Guerci-Lena Learn More: https://lnkd.in/e8wcMCdi

Ahead of #JPM24 Week, Celltelligence is publishing a series of 2 blasts with the most important #celltherapy-related news items that occurred during the holidays. In this second blast, the following news are covered: • On Thursday, December 21, 2023, #Legend shared that the FDA (SEC filing: https://bit.ly/3S7PMhl) approved a label update for #Carvykti (#JNJ / Legend’s #BCMA CAR-T) including longer-term safety and efficacy information from the Ph1/2 CARTITUDE-1 trial (https://bit.ly/490CkTh), which incorporated the risk of secondary hematological malignancies to the Boxed Warning • On the same day, #Gilead announced (press release: https://bit.ly/3vp4FUF) that the FDA approved a label update for #Yescarta (#CD19 CAR-T) including the significant OS benefit induced by the CAR-T in ≥2L LBCL in the Ph3 ZUMA-7 trial (https://bit.ly/3H64O1V) • On Tuesday, December 19, #Galapagos announced (press release: https://bit.ly/3H6gula) that the Ph1/2 #PAPILIO trial evaluating #GLPG5301 (BCMA CAR-T) in ≥3L #MM has started recruiting patients in Europe Celltelligence provides insights and context for each news story: https://bit.ly/CT-845902