Irvine, California, 4th October 2021—Targeted Therapy Technologies, LLC (3T Ophthalmics) has been awarded Breakthrough Therapy Designation by the U.S. Food and Drug Administration for its lead anti- cancer drug product candidate, Episcleral Topotecan, for the treatment of pediatric Retinoblastoma. Breakthrough Therapy Designation requires that the drug candidate treat a serious or life-threatening disease or condition. It also requires preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The designation has the potential to expedite the development and regulatory review process.
The Breakthrough Therapy Designation was largely based on remarkable interim results of an ongoing phase I clinical trial conducted by Brenda Gallie, M.D. at the Hospital for Sick Children (SickKids) in Toronto, Canada (NCT04156347) and Aparna Ramasubramanian, M.D. at Phoenix Children’s Hospital in Phoenix, Arizona (NCT04428879). Most patients in the phase I trial had refractory or recurrent Retinoblastoma following numerous interventions including triple-agent systemic chemotherapy, melphalan-based intra-arterial chemotherapy, intravitreal melphalan, radiation therapy, enucleation of one eye, and/or combinations thereof. Nonetheless, the safety and preliminary efficacy results were impressive warranting Breakthrough Therapy Designation.
Episcleral Topotecan was designed for sustained delivery of topotecan hydrochloride targeting the retina and tissues in the back of the eye therefore substantially limiting the systemic exposure of the drug and its side effects. By enabling a minimally invasive procedure that does not penetrate the eye its ease of use may significantly change the paradigm for Retinoblastoma treatment.
Episcleral Topotecan is one of six investigational drug products for which 3T Ophthalmics has obtained investigational new drug (IND) application approval by the FDA and other regulatory agencies for clinical testing.
“We are delighted with the Breakthrough Therapy designation. After more than a decade of research we were able to fine-tune Episcleral Topotecan to precisely deliver this potent anti-cancer drug in the right amount over the right period of time targeted to the right tissues in the back of a growing-infant eye with Retinoblastoma,” said Ricardo Carvalho, M.D. Ph.D., inventor and lead investigator of Episcleral Topotecan and 3T research programs. “Faced with the unique challenges posed by this disease and the uniqueness of our drug delivery platform over a decade, 3T laboratories progressed designing the platform, formulating and testing it across numerous drugs and disease targets directed toward optimizing and validating controlled delivery to desired tissues and avoidance of systemic exposure. All 3T drug products have benefited from this comprehensive and iterative drug development approach spanning many years,” stated Dr. Carvalho. 3T has been and is committed to offering a reliable, simplified and ease of use treatment for Retinoblastoma. “We will continue working quietly and hard on such disruptive therapies. Saving eyes and restoring vision is our life’s work and goal. We are thrilled that Retinoblastoma patients may benefit from our drug delivery platforms.”
Targeted Therapy Technologies LLC (3T Ophthalmics) has treated Expanded Access patients in the US and Canada and will consider such requests on a case-by-case basis subject to clinical supply availability and options for clinical trial participation. Expanded Access request should be submitted to info@3tophthalmics.com. Responses will be handled within two weeks of request dependent upon volume of requests and clinical trial alternatives. The Episcleral Topotecan Retinoblastoma clinical trial is listed on clinicaltrials.gov (NCT04156347 for US and NCT04428879 for Canada).
Retinoblastoma is the most common pediatric malignant intraocular tumor and originates from the retina. Treatment of eyes with advanced intraocular Retinoblastoma remains a challenge. The historic standard of care for patients with unilateral disease is enucleation; and for those with bilateral disease, a variety of modalities have been tried. These include radiation therapy, systemic chemotherapy, periocular administration of chemotherapy, selective intra-arterial chemotherapy and intravitreal chemotherapy. Currently, there are no agents or interventions specifically approved to treat Retinoblastoma.
The above-mentioned Episcleral Topotecan Retinoblastoma clinical trial is partially supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award R01FD005388 totaling $660,000. Other support is from foundations and non-governmental sources. The contents of this press release are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.
About FDA Breakthrough Therapy Designation
Breakthrough Therapy Designation is an FDA program intended to expedite the development and regulatory review of investigational therapies that are designed to address serious or life-threatening conditions. The criteria for Breakthrough Therapy Designation requires preliminary clinical evidence that indicates that the candidate may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. This designation provides the Company with more intensive FDA guidance on an efficient drug development program, and eligibility for other actions to expedite the FDA review, such as a rolling review of a New Drug Application (NDA), where the FDA may review sections of the NDA before the complete application is submitted. An NDA for a product candidate receiving Breakthrough Therapy Designation may also be eligible for priority review if the relevant criteria are met. Breakthrough Therapy Designation does not change the standards for approval. For more information, please visit the FDA website at www.fda.gov.