Discovery Therapeutics Caribe Receives Study May Proceed Letter from FDA to initiate a Phase 3 Clinical Trial of intralesional rhEGF for the treatment of diabetic foot ulcers

Cleveland, OH

CLEVELAND, April 30, 2024 (GLOBE NEWSWIRE) -- Discovery Therapeutics Caribe (DTC), a clinical-stage biotechnology company dedicated to improving the health outcomes of Americans through the development of regenerative therapies for serious and life-threatening diseases, today announced it has achieved a significant milestone reflecting progress in its clinical program.

In the first quarter of 2024, DTC submitted an Investigational New Drug (IND) application to the United States Food and Drug Administration (FDA). The IND submission included a phase 3 protocol for a randomized, double-blind, placebo-controlled trial of the company’s lead product, an intralesional recombinant human Epidermal Growth Factor (rhEGF) for the treatment of complex and complicated refractory neuropathic, ischemic, and neuroischemic diabetic foot ulcers (DFU).

The clinical grades of complex and complicated refractory DFUs are comparable to Wagner’s classification system grades 3 and 4; University of Texas classification system grades 2 and 3; and Wound/Ischemia/Foot Infection (WIfI) classification system stages 3 and 4. These severe ulcers are difficult to heal and carry a greater risk of amputation and death.

The FDA completed its review of the IND and concluded that DTC may proceed with its proposed Phase 3 clinical investigation. DTC received a Study May Proceed (SMP) letter from the FDA on April 10, 2024. Building upon the clinical efficacy and safety data gathered from international studies, DTC management believes they are on track to initiate this pivotal U.S.-based trial of intralesional rhEGF by year-end 2024. While intralesional rhEGF, globally marketed as Heberprot-P®, is available to patients in twenty-six countries, this is the first time it will be studied in U.S. patients suffering from DFU.

Dr. David Armstrong, DPM, MD, PhD, a distinguished podiatric surgeon at the University of Southern California and a globally recognized researcher in diabetic foot ulcers, emphasized the critical need for innovative therapies. “There is a pressing need for treatments that can halt the progression of diabetic foot ulcers before amputation becomes the inevitable solution. Historically, treatment options have been limited, but with the introduction of advanced therapies like intralesional rhEGF, which I have seen used effectively abroad, we are hopeful. The upcoming Phase 3 trial in the U.S. will rigorously assess its efficacy for DFU patients. This trial represents an exciting potential to shift the current paradigm and provide new hope for those who desperately need it.”

Lee Weingart, Co-Founder and President of DTC commented, “We are pleased to commence the next stage of intralesional rhEGF’s global journey here in the United States. The upcoming Phase 3 clinical trial will help us to understand this biologic’s clinical profile within the FDA’s regulatory framework and build upon rhEGF’s extensive international clinical experience.”

Dr. Charles Zelen, DPM FACFAS, President of the Professional Education & Research Institute (PERI) said, “Preventing the severe outcomes associated with diabetic foot ulcers is critical in saving American limbs and lives. Alarmingly, nearly half of patients who undergo DFU-related lower extremity amputation do not survive beyond five years. Among U.S. veterans, the prognosis is even more grim, where survival past two years is uncommon for patients presenting with gangrene. Particularly concerning is the disproportionate impact on our African American communities, where Medicare beneficiaries are nearly twice as likely to undergo lower limb amputation within a year of DFU diagnosis compared to their non-Hispanic White counterparts. Innovative therapeutic options for severe diabetic foot ulcers are not just necessary—they are long overdue for patients in need. PERI is committed to making these important clinical trials a reality to evaluate the efficacy and safety of new biologics like intralesional rhEGF.”

About intralesional rhEGF

Naturally occurring human Epidermal Growth Factor (hEGF) plays a crucial role in the body’s healing process. This well-understood protein works by activating the EGF receptor on the surface of cells, a process that can stimulate cell growth, migration, and differentiation. With respect to wound healing, the primary function of EGF is to encourage the growth of essential cells, such as those that form the skin (keratinocytes), connective tissue (fibroblasts), and blood vessels (vascular endothelial cells). Additionally, EGF aids in guiding these cells to the wound site, helps them develop into mature cells, and promotes the formation of new blood vessels—a process known as angiogenesis. Together, these actions accelerate the formation of new tissue and help wounds heal effectively.

In a process developed by the Center for Genetic Engineering and Biotechnology (Spanish: Centro de Ingeniería Genética y Biotecnología [CIGB]) in Havana, Cuba, hEGF is expressed using genetically engineered yeast (Saccharomyces cerevisiae), utilizing recombinant DNA technology. Unlike currently available therapies that use growth factors applied directly to the surface of wounds, intralesional injection of rhEGF delivers the active ingredient directly in the target area, a process intended to penetrate past the chronic wound environment that can otherwise degrade and reduce the effectiveness of EGF treatments.

Heberprot-P® currently has marketing authorization in 26 countries for the treatment of DFUs. During 16 years of foreign post-marketing surveillance, an estimated 426,232 DFU patients (as of December 30, 2022) have been administered Heberprot-P® worldwide since its initial marketing authorization in the Republic of Cuba in June 2006. In the international markets where Heberprot-P® is commercially available, the drug is an important option in the therapeutic armamentarium for patients with this serious condition.

About diabetic foot ulcers

DFUs are a severe and common complication of diabetes, marked by wounds that do not heal properly or quickly. These ulcers significantly contribute to morbidity and are a major reason for hospitalizations, lower limb amputations, and even death among the more than 37 million Americans living with diabetes, both diagnosed and undiagnosed. It is estimated that up to 34% of individuals with diabetes will develop a DFU in their lifetime. Annually, the United States sees approximately 1.6 million new cases of DFUs. Of these, around 154,000 patients with non-healing ulcers undergo amputation each year, a number that has been on the rise.

About Discovery Therapeutics Caribe

Discovery Therapeutics Caribe (DTC) is a clinical stage biotechnology company developing rhEGF for marketing in the United States under an exclusive collaboration agreement. DTC is an Ohio-based biotechnology company following the BLA licensure pathway under the provisions of 351(a) of the Public Health Service (PHS) Act. DTC has also executed an exclusive license agreement to market Heberprot-P® in the United States and other territories.

Forward-Looking Statements

This press release contains forward-looking statements. Any statements contained herein that do not describe historical facts are considered forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “estimate,” “potential,” and similar expressions are designed to identify such forward-looking statements. These statements include, without limitation, discussions regarding our future operations, business strategies, research and development efforts; the ongoing approval of intralesional rhEGF, which may hinge on the verification and delineation of clinical benefits in confirmatory trials; the anticipated advantages and potential risks associated with rhEGF; and our strategic plans and milestones, notably our aim to expediently proceed with a Biologics License Application (BLA) submission following the anticipated completion of the Phase 3 trial.

Actual outcomes may significantly differ from those projected in these forward-looking statements due to a variety of crucial factors and uncertainties, including, but not limited to, the timing and results of our Phase 3 clinical trial; the process and outcomes of a BLA submission to the FDA; challenges in patient enrollment for clinical trials; the results of future discussions and inspections by the FDA or other regulatory bodies, pertaining to both clinical and preclinical programs; the capacity to secure necessary regulatory endorsements; the effects of shifts in financial markets, global economic conditions, or U.S. foreign policy; and the complexities tied to data analysis and reporting.

Discovery Therapeutics Caribe expressly disclaims any obligation to update or revise these forward-looking statements to reflect any change in expectations or events, conditions, or circumstances on which any such statements may be based, except as required by federal securities laws.

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Lee Weingart

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Jason M. Smith
Senior Vice President, Program Management & Business Development

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