DURHAM – Tellus Therapeutic is approaching approval for treatment for neonatal brain injury.
This week, the Durham-based biotech company specializing in neonatal health announced that the United States Food and Drug Administration has granted orphan drug and rare pediatric drug designations to its TT-20 program.
The program targets diffuse white matter injury (DWMI) in premature infants under 32 weeks gestational age.
“[These] The FDA designations are a milestone for our TT-20 program and underscore the need to find treatments for DWMI, the predominant form of brain injury in newborns who survive premature birth, ”said Jason Kralic , Ph.D., Managing Director and Co-Founder of Tellus.
There is currently no FDA approved treatment 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 within an accelerated six-month period.
In December 2019, Tellus received a loan of $ 250,000 from the North Carolina Biotechnology Center to develop its treatment.
“The financial and intellectual capital provided by NCBiotech has been of tremendous benefit to Tellus,” Kallic said, adding that the applications for FDA designation were covered by the loan.
“[It’s] a great example of how [this] the funding facilitated our ability to achieve research and development and business milestones.
Founded in 2018, Tellus is focused on developing safe and effective treatments for the unmet needs of newborns.
Each year, about one in ten babies in the United States is born prematurely.
The result: a significantly increased risk of white matter (myelin) damage, leading to lifelong neurological disorders, including cognitive function deficits and autism spectrum disorders.
DWMI is the most common form of premature neonatal brain injury and is a strong predictor of poor neurological outcomes.
To combat this, Tellus is developing new small molecules derived from human breast milk as a form of treatment in newborns.
The company’s flagship molecule has been shown to promote myelination and subsequent reversal of motor deficits in animal models of white matter injury.
Tellus is currently pursuing a “first newborn” regulatory pathway to assess safety and efficacy in neonates with brain damage.
“In the absence of FDA-approved drugs for DWMI, we look forward to continuing our collaboration with the FDA as we further define a regulatory pathway for TT-20 in DWMI and other neonatal conditions,” said said Kralic.
In addition to the NCBiotech loan, Tellus has raised $ 75,000 in the form of a convertible note from MedBlue, won $ 100,000 in a MassChallenge pitch competition and is backed by a Duke Clinical & Translational Science Institute Award of 500 $ 000.
Tellus aims to raise a Seed / Series A investment before January 2021, Kralic told NCBiotech.
He did not identify the exact sum, but said the funds would cover a pre-investigation meeting on a new drug application at the end of 2021, as well as its deposit for a “first newborn” clinical trial in 2022.
Additionally, Tellus will fund “pipeline development targeting other unmet neonatal needs.”
The company has also strengthened its scientific advisory board to help guide its evolution, appointing Terrie Inder, MD, and Chi Hornik, PharmD, BCPS, to its board of directors.
Inder is the chair of the pediatric neonatal medicine department at Brigham and Women’s Hospital in Boston. She is also the Mary Ellen Avery Professor of Pediatrics in the area of Neonatal Medicine at Harvard Medical School.
Hornik is Director of Clinical Research in the Division of Pediatric Critical Care Medicine at Duke University.
They should guide the company on clinical development programs, especially its main DWMI program, also known as TT-20. Its focus is motor complications and other disabilities associated with cerebral palsy in premature babies.
“We are excited to tap into their diverse perspectives, deep expertise and extraordinary knowledge in neonatal research and critical care,” said Eric Benner, MD, Ph.D., Scientific Director and Co-Founder of Tellus.
They join Simon Gregory, Ph.D., scientific co-founder, professor in the department of neurology and research director of the Duke Center of Autoimmunity and Multiple Sclerosis at Duke University.