BEVERLY, MA–(Marketwired – August 23, 2015)- Cellceutix Corporation (OTC: CTIX) (the “Company”), a clinical stage biopharmaceutical company developing innovative therapies with oncology, dermatology, anti-inflammatory and antibiotic applications, is requesting a meeting with the U.S. Food and Drug Administration (FDA) to discuss the advancement of the Company’s anti-cancer drug candidate Kevetrin for the treatment of pediatric retinoblastoma.
Retinoblastoma is the most common eye cancer affecting children usually before age 3. It is sight threatening and potentially fatal if not caught early. Occurrence is 250-300 cases per year in the U.S. The tumors arise from the retina, the light sensitive nerve tissue which lines the back of the eye. If caught early and treated it may spare the child from having the eye removed (enucleated) which occurs in late stages to prevent metastasis.
The FDA recently released draft guidance on development of drugs intended to treat rare diseases (http://www.fdanews.com/ext/resources/files/08-15/08-17-15-rarediseases.pdf?1439565970). This guidance demonstrates the FDA’s flexibility in drug discovery targeting rare disease and encourages sponsors to meet with the agency to design and implement clinical studies. The FDA also encourages development of drugs and biologics to prevent and treat rare pediatric diseases through its Pediatric Disease Priority Review Voucher (PPRV) incentive program. The program is set to expire in March 2016, but could be extended under a provision in the 21st Century Cures Act. Last week United Therapeutics Corporation agreed to sell its Priority Review Voucher to AbbVie for $350 Million.
In 2013, Cellceutix conducted pre-clinical studies using human retinoblastoma cells (WERI-Rb-1) in nude mice that were implanted either subcutaneously or directly into the eye, intravitreally. Treatment with Kevetrin significantly reduced the tumor volume by more than half in the subcutaneous tumor model and showed a significant improvement in the clarity of the eye in mice treated with Kevetrin.
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.
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.