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.
Media Contact
Lee WeingartPresidentmedia@discothera.com
Investor Contact
Jason M. SmithSenior Vice President, Program Management &
Business Developmentinvest@discothera.com
For more information, visit https://www.discothera.com/