Cellceutix Requesting Meeting With FDA on Kevetrin for Pediatric Retinoblastoma - Seite 2

The ongoing Phase 1 trial of Kevetrin for solid tumors has given Cellceutix a better understanding of Kevetrin's safety and mechanistic profiles making this the right time to request a meeting with the FDA. Although no retinoblastoma patient was enrolled in the Phase 1 study, the development plan for Kevetrin has always included investigating the novel compound as an important alternative to treat a rare disease to avoid such a drastic measure as enucleation (removal) of the eye of a young child. Cellceutix feels that there are many development options available in this indication, including accelerated programs such as a Pediatric Disease Priority Review Voucher, which we intend to discuss with the FDA based on the unique properties of Kevetrin.

"This is consistent with Cellceutix objectives," commented Leo Ehrlich, Chief Executive Officer at Cellceutix. "Cellceutix is now in a Phase 2 trial for Brilacidin for the prevention of oral mucositis in patients with head and neck cancer. Should the study show immunomodulatory and anti-inflammatory activity, Brilacidin will be advanced for the indications of ulcerative colitis and hidradenitis suppurativa. Likewise, a successful Phase 3 trial of Brilacidin to Treat Acute Bacterial Skin and Skin Structure Infections (ABSSSI), would lead us on our gateway path to address diabetic foot infections (DFI) with a concentrated formulation of Brilacidin that may be effective against both gram-positive and gram-negative infections required by the FDA to treat DFI. Certainly now that we have a growing understanding of Kevetrin's profile in cancer, it is appropriate for us to begin our long-standing plans to address rare pediatric diseases."

Kevetrin's mechanism and Retinoblastoma

Retinoblastoma is a malignant tumor of the developing eye that occurs in children. Retinoblastoma tumors rarely contain a mutation in the p53 gene. Tumors with wild-type p53 rely on various other mechanisms to inactivate p53. One of the most common mechanisms is increased activity of MDMX or MDM2 gene which occurs frequently in human retinoblastoma. (http://www.nature.com/nature/journal/v444/n7115/full/nature05194.html).

The antitumor activity in this type of tumor is to reactivate wild-type p53. Since many pediatric tumors maintain wild type p53 and their loss of function mutation of p53 is due to a secondary mutation affecting a growth-regulatory pathway (RB1), the reactivation of wild type p53 is therefore of particular value for the treatment of childhood tumors. The non-genotoxic activation of p53 after Kevetrin treatment is expected to result in significantly less toxicity than classical chemotherapy.