Date: 2016-05-02
Type of information: DSMB assessment
phase: 1-2
Announcement: DSMB assessment
Company: Novartis (Switzerland) Genec (USA - MD)
Product: CGF166
Action
mechanism: gene therapy. CGF166 is a gene therapy product for the treatment of severe hearing loss. CGF166 is engineered to express Atoh1, an atonal gene that induces the differentiation of sensory cells in the inner ear during embryonic development. CGF166 utilizes Genvec's proprietary GV11 gene delivery system, featuring an E1-, E3-, E4-deleted human adenovector serotype 5 (Ad5) backbone. CGF166 also features a cell-specific promoter that limits the expression of the atonal gene to the supporting cells of the inner ear.Experiments in animal models testing CGF166 conducted by Novartis have confirmed that this experimental biological triggers the regeneration of sensory cells in both animal and human tissue models and the restoration of hearing in animal models. CGF166 is being developed by the Novartis Institute for Biomedical Research, a subsidiary of Novartis AG, under a research collaboration and license agreement) with GenVec.
Disease: severe-to-profound bilateral hearing loss with intact vestibular function in the non-operative ear
Therapeutic area: Otorhinolaryngology
Country: USA
Trial
details: This phase I-II study will evaluate the safety, tolerability, and potential efficacy of CGF166 and the associated delivery procedures in patients with severe-to-profound bilateral hearing loss. Patient dosing will be staggered; dosing the next patient in a cohort will be based on a safety review of all available data through 4 weeks post-dose of the previously dosed patient(s). Part B includes a volumetric escalation design to evaluate infusion volumes of the same CGF166 concentration (5.0 x 10E11vp/mL) in 4 cohorts of patients (n=3/cohort; total of 12 patients). Part C is an expansion cohort of the highest safe and tolerable dose identified in Part B, for further assessment of efficacy. (NCT02132130)
Latest
news: * On May 2, 2016, GenVec announced that it was notified by its collaborator Novartis that the Data Safety Monitoring Board (DSMB) for the Phase 1/2 clinical trial of CGF166 in patients with severe to profound hearing loss has recommended that the trial continue, subject to approval by the FDA. This recommendation was based on a review of safety and efficacy data from the nine patients currently enrolled in the study. Novartis is developing CGF166, GenVec's lead product candidate for hearing loss, under a Research Collaboration and License Agreement with GenVec. As previously disclosed, on January 8, 2016, GenVec was notified by Novartis that enrollment was paused in the clinical study for CGF166 in accordance with criteria in the trial protocol. * On January 8, 2016, GenVec was notified by its partner Novartis that enrollment was paused in the clinical study for CGF166. This pause was based on a review of data by the trial's Data Safety Monitoring Board in accordance with criteria in the trial protocol. Novartis will continue to collect safety and efficacy data on the nine patients currently enrolled in the study before determining whether to proceed with incremental patients. GenVec is awaiting further guidance from its partner on the CGF166 study and is committed to updating shareholders when it gains greater clarity. The first patient was treated in October 2014, representing the first direct drug delivery to the inner ear. * On June 18, 2014, Novartis announced at its first "Meet Novartis Management" investor day, that patients are currently being recruited for Proof of Concept clinical trials for CGF166, a novel gene therapy for sensorineural hearing loss,
