1ST Biotherapeutics, Inc., a clinical-stage biotechnology company focused on neurodegenerative diseases, immuno-oncology, and orphan diseases, and twoXAR Pharmaceuticals, a company that discovers drugs with AI, today announced the companies will advance three novel drug leads for the potential treatment of glioblastoma into in vivo efficacy testing less than one year from launching the drug discovery collaboration.
The traditional drug development approach takes four to six years on average to go from target identification to Investigational New Drug (IND)-enabling studies, including years until the first preclinical in vivo study. The twoXAR process, propelled by the company’s proprietary and unbiased AI-driven platform to identify promising first-in-class drug discovery hits, initiates preclinical in vivo screening within months and programs advance to IND-enabling studies in one to three years.
Glioblastoma is the most common and most aggressive primary malignant brain tumor and developing effective treatments for this complex cancer has been historically difficult. It presents unique challenges, including multiple resistance mechanisms and the necessity for therapies to cross the blood brain barrier. Glioblastoma treatment options are limited with only four FDA-approved therapies available. With currently available treatments, glioblastoma is uncurable and has a median survival time of 15 months.i
“There is an urgent need to identify more efficiently novel targets for challenging diseases like glioblastoma and more rapidly assess the treatment efficacy of those candidates in preclinical studies,” said Jamie Jae Eun Kim, CEO of 1ST Biotherapeutics. “The speed at which we were able to work with twoXAR to identify these three new targets is unprecedented. The combination of twoXAR’s process and AI-driven platform designed specifically for target identification and our deep expertise in neurodegenerative disease and oncology enabled us to rapidly discover and advance these exciting new approaches to potentially treat this devastating disease.”
Under a partnership agreement announced in 2019, twoXAR identified a set of drug discovery hits with the potential to slow, stop, or reverse the progression of glioblastoma. twoXAR and 1ST Biotherapeutics then selected leads from this initial set to test in preclinical efficacy models of glioblastoma.
“This is a prime example of how AI, together with drug discovery and therapeutic expertise, can bring tremendous efficiency in identifying new targets for traditionally difficult-to-treat diseases,” said Mark G. Eller, PhD, Senior Vice President, Research and Development at twoXAR. “We will continue to collaborate with 1ST Biotherapeutics and leverage our combined drug discovery expertise to progress these promising candidates through the remaining preclinical research steps.”
Glioblastoma is the most common and lethal primary malignant brain tumor with a median survival of 15 months.i Just six percent of patients diagnosed after age 40 survive five years or more. It is characterized by an aggressive tumor that infiltrates various portions of the brain and is associated with rapid onset of neurological symptoms, including nausea, vomiting, severe headaches, cognitive issues, and seizures.
Current treatment options consist of surgical resection, radiation, chemotherapy, and angiogenesis inhibitors. However, most glioblastoma patients experience disease relapse despite these aggressive therapies. It is estimated that 25,000 people will be diagnosed with glioblastoma in 2018.i
About 1ST Biotherapeutics
1ST Biotherapeutics is a science-driven biopharmaceutical company focused on the development of breakthrough therapies in neurodegenerative diseases, immuno-oncology, and rare diseases. Founded in 2016, 1ST Biotherapeutics has rapidly developed a deep pipeline of first-in-class investigational therapeutics with clear mechanism for proper disease modification and with good pharmacological properties. 1ST Biotherapeutics is based in Seongnam, South Korea. For more information, please visit 1stbio.com.
i Lee, J.H., Lee, J.E., Kahng, J.Y. et al. Human glioblastoma arises from subventricular zone cells with low-level driver mutations. Nature 560, 243–247 (2018). https://doi.org/10.1038/s41586-018-0389-3
This article first appeared on PR Newswire.