DURHAM – Tellus Therapeutics is getting closer to approval for its neonatal brain injury treatment.
This week, the Durham-based biotech company focused on neonatal health announced that the U.S. Food and Drug Administration has granted Orphan Drug and Rare Pediatric Drug designations for its TT-20 program.
The program targets diffuse white matter injury (DWMI) in preterm infants less than 32 weeks gestational age.
“[These] designations from the FDA is an important milestone for our TT-20 program and underscores the need to find treatments for DWMI, the predominate form of brain injury among neonates who survive preterm birth,” said Jason Kralic, Ph.D., chief executive officer and co-founder of Tellus.
There are currently no FDA-approved treatments for DWMI.
With this designation, Tellus is now eligible for a priority review voucher, which can be used to obtain FDA review of a New Drug Application (NDA) for another product in an expedited period of six months.
In December 2019, Tellus received a $250,000 loan from the North Carolina Biotechnology Center to develop its treatment.
“Both the financial and intellectual capital provided by NCBiotech has been tremendously helpful to Tellus,” Kallic said, adding that the FDA designation applications was covered by the loan.
“[It’s] a prime example of how [this] funding has facilitated our ability to achieve research and development, and company milestones.”
Breaking new ground
Founded in 2018, Tellus is focused on developing safe and effective treatments for unmet needs in newborns.
Each year, about one in 10 babies in the United States is born prematurely.
The result: a significantly increased risk for white matter (myelin) injury, leading to life-long neurological impairments, including deficits in cognitive function and autism-spectrum disorders.
DWMI is the most prevalent form of preterm neonatal cerebral injury and is a strong predictor of poor neurologic outcomes.
To combat this, Tellus is developing novel small molecules derived from human maternal breast milk as a form of treatment in newborns.
The company’s lead molecule has been shown to promote myelination and subsequent reversal of motor deficits in animal models of white matter injury.
Tellus is now pursuing a “First-in-Neonate” regulatory path to evaluate safety and efficacy in newborns with brain injury.
“With no FDA-approved drugs for DWMI, we look forward to continued collaboration with the FDA as we further define a regulatory path for TT-20 in DWMI and other neonatal conditions,” Kralic said.
In addition to the NCBiotech loan, Tellus has raised $75,000 in the form of a convertible note from MedBlue, won $100,000 from a MassChallenge pitch competition and is supported by a $500,000 Duke Clinical & Translational Science Institute Award.
Tellus aims to raise a Seed/Series A investment before January 2021, Kralic told NCBiotech.
He didn’t identify the exact sum but said the funds would cover a pre-investigational New Drug Application meeting in late 2021, along with its filing for a “First-in-Neonate” clinical trial in 2022.
Additionally, Tellus will be funding “a pipeline development targeting other neonatal unmet needs.”
The company has also beefed up its scientific advisory board to help guide its evolution, appointing Terrie Inder, M.D., and Chi Hornik, PharmD, BCPS, to its board.
Inder is the chair of the Department of Pediatric Newborn Medicine at Brigham and Women’s Hospital in Boston. She is also the Mary Ellen Avery Professor of Pediatrics in the Field of Newborn Medicine at Harvard Medical School.
Hornik is the director of clinical research in the Division of Pediatric Critical Care Medicine at Duke University.
They are expected to guide the company on clinical development programs, particularly its lead DWMI program, otherwise known as TT-20. Its aim is motor complications and other disabilities associated with cerebral palsy in premature babies.
“We are thrilled to be tapping into their diverse perspectives, profound expertise and tremendous insights in neonatal research and critical care,” said Eric Benner, M.D., Ph.D., Tellus’ chief scientific officer and co-founder.
They join Simon Gregory, Ph.D., scientific co-founder, who is professor in the Department of Neurology and research director of the Duke Center of Autoimmunity and Multiple Sclerosis at Duke University.