San Marcos

Researchers are evaluating intravitreal EYE103 in participants with neovascular age-related macular degeneration (NVAMD) or macular edema following branch retinal vein occlusion (BRVO). This Phase 2, randomized, dose-masked study includes four patient cohorts: treatment-naive NVAMD participants, incomplete responder (IR) NVAMD participants as monotherapy, IR NVAMD participants receiving EYE103 combined with aflibercept 2.0 mg, and treatment-naive BRVO participants. The study aims to assess safety and efficacy of different doses of EYE103 in these conditions. Participants in each cohort will be randomly assigned to receive either a low or high dose of EYE103 via intravitreal injection. All participants will receive three injections spaced four weeks apart. IR NVAMD participants in the combination therapy cohort will also receive an injection of aflibercept 2.0 mg on Day 1. The timing of enrollment into each cohort is determined by the Sponsor. Participants will undergo safety and efficacy assessments at each injection visit, with some cohorts returning two weeks after injections for further evaluations. Assessments include measuring best-corrected visual acuity using the ETDRS chart, slit-lamp biomicroscopy, fundoscopy, and spectral domain optical coherence tomography (SD-OCT) to measure central subfield thickness. The study concludes at Week 12, which is the end-of-study visit for all participants.

Researchers are evaluating the safety and effectiveness of three different doses of MORF-057 in adults with moderately to severely active Crohn's disease (CD). This Phase 2 study is randomized, double-blind, placebo-controlled, and conducted at multiple centers. It aims to compare MORF-057 to placebo to see how well it works in reducing disease activity and symptoms in this patient population. Participants will first go through a 14-week induction period where they receive one of three doses of MORF-057 or a matching placebo, all given orally. After this, all participants will enter a 38-week maintenance phase where they receive open-label MORF-057. Those who complete these 52 weeks of treatment may continue in a 52-week long-term extension to further monitor treatment effects and safety. Throughout the study, participants will have evaluations to assess their response to treatment using endoscopic scoring at Week 14. Researchers will monitor safety, symptom changes, and disease activity over the full treatment and extension periods. Study visits will include assessments, questionnaires, and clinical monitoring to track participants' health and treatment adherence over time.

Researchers are evaluating the effectiveness, safety, tolerability, and pharmacodynamics of multiple doses of APL-3007 combined with Syfovre/Pegcetacoplan (APL-2) in patients aged 60 years and older diagnosed with geographic atrophy secondary to age-related macular degeneration. This Phase 2, randomized, placebo-controlled, multicenter, masked study focuses on measuring changes in retinal pigment epithelium lesions using advanced artificial intelligence-based SD-OCT imaging. Participants will receive either the combination of APL-3007 with pegcetacoplan (APL-2) or a placebo. The study includes a treatment period with multiple doses administered, aiming to assess the impact on geographic atrophy lesions over a 12-month period. Syfovre injections at 6-8 week intervals prior to enrollment are part of the inclusion criteria. During the study, participants will undergo various eye imaging assessments such as OCT and FAF to monitor lesion size and progression. Researchers will evaluate changes in lesions at month 12 compared to baseline. Safety and tolerability will be closely monitored through laboratory tests, clinical evaluations, and vaccination status requirements. The study duration includes regular visits for treatment administration and monitoring over at least one year.

Researchers are evaluating a new approach to prevent cardiovascular events in patients at increased risk due to age and conditions like type 2 diabetes, prediabetes, or metabolic syndrome but without known symptomatic cardiovascular disease. The study compares a Cleerly Coronary Artery Disease (CAD) Staging System-based care strategy with standard risk factor-based care to see if the former can better reduce cardiovascular events. The Cleerly system uses imaging to visualize and quantify coronary artery disease and guides personalized treatment and education based on this assessment. The trial uses the Cleerly CAD Staging System device, which employs a proprietary algorithm to detect and stage coronary artery disease and generate a risk score to guide treatment decisions. Participants receive either this stage-based care or the usual care based on traditional risk factors. The study is prospective, randomized, and pragmatic, designed to follow patients over an average of 3.5 years to compare cardiovascular event outcomes between these two care approaches. Participants will be monitored through cardiovascular event tracking throughout the study period. Data collected includes imaging results, risk scores, and treatment adherence to evaluate the impact of the care strategies. The primary outcome is the comparison of cardiovascular event risk between the Cleerly stage-based care and risk factor-based care groups. The study also includes ongoing safety monitoring and personalized management by a cardiologist-led team via digital communication devices.

Researchers are investigating the safety and effectiveness of efruxifermin in people with non-cirrhotic nonalcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH) who have moderate to advanced liver fibrosis (stage 2 or 3). This Phase 3 study is randomized, double-blind, and placebo-controlled, enrolling a total of 1650 participants in two groups to evaluate treatment outcomes. Participants will receive either efruxifermin or a placebo by subcutaneous injection. The study involves two cohorts, with Cohort 1 including patients who have biopsy-confirmed NASH or MASH and specific liver fibrosis and activity scores. The treatment period and detailed dosing schedules are not provided but the study compares the effects of the active drug against placebo. During the study, participants will be monitored for improvement in liver disease status, including resolution of NASH/MASH and at least a one-stage improvement in liver fibrosis after 52 weeks for Cohort 1. Long-term outcomes such as event-free survival will be observed over 240 weeks. Safety and efficacy assessments will be conducted throughout the study period, including evaluations of liver histology and metabolic health.

Researchers are evaluating the effectiveness of dotinurad compared to allopurinol in lowering serum uric acid (sUA) levels at 24 weeks in adults with tophaceous gout. This condition involves the presence of measurable tophi, or deposits of uric acid crystals, in joints such as hands, wrists, feet, or ankles. The study is a Phase 3, randomized, double-blind, multicenter trial focused on adults aged 18 to 75 years who have had gout for at least one year. Participants receive either dotinurad or allopurinol in over-encapsulated tablet form, taken orally. The treatments are compared to see which better lowers sUA levels below 5.0 mg/dL after 24 weeks. The study includes a screening period before treatment begins, during which eligibility is confirmed, including measurements of tophi size and uric acid levels. During the study, participants will have regular assessments to monitor serum uric acid levels and the size of tophi. Safety and side effects will also be monitored throughout the 24-week treatment period. The main outcome is the percentage of participants who achieve sUA levels less than 5.0 mg/dL at week 24, helping to understand the comparative efficacy and safety of the two medications.

Researchers are evaluating the effectiveness and safety of pegozafermin in adults aged 18 to 75 years who have compensated cirrhosis caused by metabolic dysfunction-associated steatohepatitis (MASH), previously known as nonalcoholic steatohepatitis (NASH). Participants in this phase 3 study must have biopsy-confirmed advanced liver fibrosis (stage F4) due to MASH and meet specific metabolic health criteria. The study aims to understand how well pegozafermin can help improve liver fibrosis and delay disease progression over time. Participants will receive either pegozafermin or a placebo through subcutaneous injections. The study will monitor participants over a long period, up to five years, to observe changes in liver fibrosis and any clinical events related to disease progression. The treatment is given to those with compensated cirrhosis, meaning their liver is damaged but still functioning, and the study carefully evaluates the safety and potential benefits of pegozafermin in this group. Throughout the study, participants will undergo regular assessments to track liver health, including fibrosis regression and timing of disease progression. Researchers will use clinical events and laboratory tests to measure outcomes from the start of the study through 24 months and up to five years. Safety and health will be monitored closely, ensuring any side effects or complications are identified promptly. This comprehensive follow-up helps provide detailed information on the long-term effects of the treatment and participants' liver condition.

Researchers are investigating whether the medicine vicadrostat, when taken together with empagliflozin, can lower the risk of heart-related problems in adults who have type 2 diabetes, high blood pressure, and cardiovascular disease but no history of heart failure. This study is a Phase III trial that compares the effects of vicadrostat plus empagliflozin to a placebo plus empagliflozin in people with these conditions. Participants are randomly assigned to one of two groups: one group takes vicadrostat and empagliflozin tablets, and the other group takes placebo tablets that look like vicadrostat along with empagliflozin. All participants take one tablet daily for a period ranging from two and a half years up to four years and three months. Throughout the study, participants continue their usual medications for diabetes, high blood pressure, and cardiovascular disease. During up to 51 months of participation, participants visit the study site regularly where doctors collect health information and blood samples. Researchers track when participants experience cardiovascular events such as heart-related deaths or heart failure events. The study also monitors participants’ overall health and any side effects they may experience to assess the safety and effects of the treatments.

Researchers are evaluating the safety and effectiveness of Ixoberogene Soroparvovec (Ixo-vec) for treating neovascular (wet) age-related macular degeneration (nAMD) in adults aged 50 years and older. This Phase 3, multi-center, randomized, double-masked study compares a single intravitreal injection of Ixo-vec with an active comparator, Aflibercept. The study focuses on a broad population including both treatment-naïve and treatment-experienced participants, aiming to assess if Ixo-vec is not less effective than the comparator in improving vision. Participants will receive one intravitreal injection of either Ixo-vec or Aflibercept. Ixo-vec is a gene therapy designed to reduce the treatment burden by potentially decreasing the need for frequent injections that are common in current anti-VEGF treatments for nAMD. The study includes a screening period followed by treatment administration and ongoing safety and efficacy assessments. During the study, participants will undergo regular evaluations including measurements of best corrected visual acuity (BCVA) at baseline and at Weeks 52 and 56 to monitor changes in vision. Safety, tolerability, and treatment response will be closely observed throughout the study duration. Researchers will also monitor anatomical responses to therapy and overall eye health through clinical assessments and adherence to eye drop use as required by the protocol.

Researchers are investigating the safety and effectiveness of a gene therapy called Ixo-vec in people aged 50 and older with neovascular (wet) age-related macular degeneration (nAMD), a condition that causes abnormal blood vessel growth in the retina and can lead to vision loss. This Phase 3, multi-center, randomized, double-masked study compares a single injection of Ixo-vec with the standard treatment of intravitreal aflibercept. The main goal is to see if Ixo-vec is not worse than aflibercept in improving best corrected visual acuity (BCVA) at one year after treatment. Participants will receive one intravitreal injection of either Ixo-vec or aflibercept in the affected eye. Ixo-vec is delivered as a gene therapy designed to reduce the need for frequent anti-VEGF injections, while aflibercept is an active drug comparator given intravitreally. The study includes a screening period and treatment followed by assessment visits, with the primary efficacy measurement taken as an average of BCVA changes at Weeks 52 and 56. Throughout the study, participants will undergo regular eye exams, visual acuity tests, and safety monitoring to evaluate how well the treatments work and their tolerability. Researchers will track changes in vision and any side effects from the treatments. The study is designed to assess both short-term and longer-term outcomes related to vision and safety, with total participation lasting over one year including screening and follow-up visits.