Iveric Bio Goes Ahead with Second Pivotal Phase 3 Study of Zimura
Iveric Bio (ISEE) announced that it has dosed the first patients for its second pivotal Phase 3 clinical trial GATHER2. The study seeks to evaluate Zimura’s potential for treating patients with geographic atrophy secondary to age-related macular degeneration. The estimated primary completion date for the study is June, 2022.
GATHER2 trial is expected to recruit nearly 400 patients. These participants will be randomized to receive either monthly administration of Zimura 2 mg or sham during the first 12 months of the trial. At that point of time, a primary efficacy analysis of the mean rate of change of GA growth at 12 months is scheduled to be performed. Kourous A. Rezaei, M.D., Chief Medical Officer of IVERIC bio said, “We have experienced impressive enthusiasm by our investigators to initiate the second Phase 3 trial, GATHER2, based on the robustness of efficacy, the strength of the statistical evidence, and the favorable safety profile of Zimura in the GATHER1 Phase 3 trial.” The company also stated that potentially GATHER1 is the only Phase 3 clinical trial demonstrating early inhibition of GA growth which sustained for 18 months with continuous treatment.
Following the primary efficacy analysis, the company will be re-randomizing patients to Zimura 2mg to be administered monthly or every other month. At month 24, Iveric will carry out the final evaluation. Iveric plans to submit an application with the FDA contingent upon getting the positive results from its primary efficacy analysis. The company will be also be seeking marketing approval for the drug candidate for treating GA from the European Medicines Agency.
For GATHER1, the company reported that the drug candidate met its pre-specified primary efficacy endpoint. It demonstrated statistically significant results. Currently, there is no FDA approved or EMA approved treatment available for treating patients with GA secondary to AMD.
Earlier this year, the company reported positive 18-month results from GATHER1. The data supported the previously reported 12 month data from the trial. For 18 months, the drug candidate showed 28.11 percent decline in the mean rate of GA growth for the Zimura 2mg group. The Zimura 4mg group showed 29.97 percent decline of the same metric. The trial also showed that the impact of the treatment was observable as early as 6 months. The drug candidate also maintained its safety profile over the period of 18 months.
Zimura aims to control complement factor C5 cleavage into C5a and C5b. By regulating the development of complement C5 terminal fragments, the drug candidate may help in reducing the activation of inflammasomes. It may also help in controlling the development of membrane attack complex. Complement factor C5 is a key element of the complement cascade. It is believed to be associated with the development and progress of AMD.
Iveric Bio is a pharmaceutical company and focuses on discovering novel treatment options for retinal ailments. The company is working on developing therapeutic product candidates for age-related retinal diseases. It is also carrying out studies for gene therapy product candidates for orphan inherited retinal diseases.
Rubius Therapeutics Offers Data for RTX-240 Clinical Trial
Rubius Therapeutics Inc. (RUBY) provided update for its clinical trial of RTX-240. The drug candidate is being evaluated for treating patients with relapsed/refractory or locally advanced solid tumors. The company reported that it has completed dosing of the first dose-escalation cohort.
The Phase 1/2 trial seeks to test RTX-240 for its safety, tolerability, pharmacokinetics, maximum tolerated dose. The data collected from this trial will also be used for recommending Phase 2 dose and dosing regimen of RTX-240. Pablo J. Cagnoni, M.D., president and chief executive officer of Rubius Therapeutics. “Based on our preclinical data, we believe these effects will translate into anti-tumor killing and clinical responses in patients. Our fully owned manufacturing facility in Smithfield, RI, continues to successfully manufacture clinical supply of RTX-240.” The trial will also assess the pharmacodynamic impacts of RTX-240.
The study further seeks to assess production of granzyme B, which may indicate activated NK and T cells capable of tumor killing. The trial will also evaluate anti-tumor activity. This metric will be gauged by overall response rate, progression-free survival and overall survival. The company will conduct expansion cohorts in specified tumor types during Phase 2 of the trial, following the current dose escalation phase.
RTX-240 is an allogeneic cellular therapy product candidate. It works by presenting multiple copies of the costimulatory 4-1BB ligand and the trans-presented cytokine interleukin-15 in their native forms. This process helps in activating and expanding NK and T cells. 4-1BBL is also believed to aid production of interferon gamma. IL-15, on the other hand, is a cytokine and promotes proliferation of NK and T cell. It also helps in supporting NK cell cytotoxicity.
Zynerba Pharmaceuticals Fails to Impress in Fragile X Syndrome Trial
Zynerba Pharmaceuticals Inc. (ZYNE) reported topline results from its 14 week pivotal CONNECT-FX trial. The study failed to meet statistical significance versus placebo for its primary endpoint. It also failed to show statistical significance versus placebo in the three key secondary endpoints. The company plans to meet with the FDA for deciding the regulatory path forward for the drug candidate.
CONNECT-FX is a multi-national, randomized, double-blind, placebo-controlled trial assessed the efficacy and safety of Zygel™ CBD gel as a treatment in for behavioral symptoms of Fragile X syndrome. Armando Anido, Zynerba’s Chairman and Chief Executive Officer said, “The results from CONNECT-FX identified a significant patient population who responded well to Zygel and may provide us with a pathway towards licensure.” The trial involved 212 patients.
A pre-planned ad hoc analysis of the most severely impacted patients in the trial showed that patients administered the drug candidate achieved statistical significance in the primary endpoint of improvement at 12 weeks of treatment in the Social Avoidance subscale of the ABC-CFXS compared to placebo. The company plans to use this data for conducting a meeting with the FDA.
The primary endpoint for the trial was improvement in the Social Avoidance subscale of the Aberrant Behavior Checklist – Community FXS. The three key secondary endpoints for the trial were the change from baseline to the end of the treatment period in the Irritability subscale score of the ABC-CFXS, the Socially Unresponsive/Lethargic subscale score of the ABC-CFXS and Improvement in Clinical Global Impression (CGI-I).
However, the drug candidate was found to be well tolerated. Its safety profile was in line with the data received from other Zygel clinical trials. 54 percent of the patients reported experiencing a treatment emergent adverse event. Seven patients reported psychiatric disorder TEAEs. Five of these patients were from placebo group.
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