Bypassing the blood-brain barrier (BBB) has been one of the greatest hurdles in the development of treatments for patients living with brain cancer with glioblastomas for a long time now.
When a patient is living with a growth inside the brain there are several ways that this can be addressed, but beyond surgery, any method will need to find a way of getting a treatment directly past the BBB.
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A membrane-like surface that prevents viruses and pathogens from entering the brain, the BBB is a hindrance to the direct entry of drugs into the brain similarly by filtering out active ingredients that may be useful in treatment.
Research published in the journal, Nature, found that typically the BBB excludes more than 98% of small-molecule drugs and all macromolecular therapeutics from access to the brain.
It’s for this reason that the field of drug delivery into the brain has become more at home with medical device solutions than pharmacological ones. This issue is not just limited to the treatment of glioblastomas, breaking through the BBB has been a recurring issue in brain-centric indications such as Alzheimer’s disease. Across these spaces, there have been a number of novel approaches, ranging from the use of low-intensity pulsed ultrasound (LIPUS) to the use of drug-delivering nanoparticles.
However, devices need to be manufactured and validated before they can be used and end up incumbent with costs and maintenance during their use. It’s no wonder then that there would be interest in the idea of a pharmacological method of penetrating the BBB.
One Californian biotech, NeOnc Technologies, believes it has developed a new method of delivering cancer treatment drugs directly to the brain. Dr. Thomas Chen, the company’s founder and CEO, claims it can extend life by more than three years past the current standard of care (SOC).
Exploring new delivery pathways
At the start of 2025, the Californian company switched from an initial public offering (IPO), instead seeking a direct listing before the US Securities and Exchange Commission (SEC), indicating a degree of faith in its innovative drug delivery platform.
As opposed to an IPO, a direct offering means the company is offering existing shares in the company, not underwriting new ones. This could be a make-or-break move for NeOnc but it demonstrates a significant amount of confidence in its therapies. The company says it is the first to develop a nasal brain delivery platform for cancer.
The company has two drugs in its portfolio, the first being NEO100, a conjugated formulation of the drug perillyl alcohol (POH) which is being developed as an intranasal therapy. NeOnc’s other drug NEO212 is also the conjugated formulation of POH but is combined with SOC chemotherapy drug, temozolomide. NEO212 is being developed to be delivered either orally or intranasally.
The aim of using nasally delivered POH is so the drug can be absorbed into the cranial nerves and delivered to the brain effectively. Intravenous (IV) delivery is the main method being investigated by other companies to attempt to push a drug past the BBB.
“What NeOnc has done is taken this concept of nasal brain delivery and asked the question ‘can we take some of our drugs that we have been administering systemically and deliver them to the brain itself?” explains NeOnc co-founder and CEO Dr. Chen.
“There are several advantages to intranasal delivery. Number one is that we don’t have to give the drug systemically, which means that while chemotherapy and other types of treatment are taken orally or through IV, instead we can bypass systemic side effects and target it towards the brain.
“This has fewer side effects on the rest of the body and means that more drug, or less toxic drug, can be delivered to the brain itself. The other advantage is that it is non-invasive, it doesn’t require placement of an IV into the arm, and the treatment is administered by inhalation to the brain.”
Citing a previous study published by a Brazilian colleague in which Chen was a corresponding author, Professor Clovis O da Fonseca of Antonio Pedro University Hospital was able to demonstrate POH’s “remarkable” ability to impact glioblastomas, but not when it was administered orally. POH has become the only intranasally delivered compound in the field of cancer therapy that has advanced past pre-clinical trials.
Currently, NeOnc has two ongoing trials, an ongoing Phase I/IIa NEO100-01 GMB trial (NCT02704858) of NEO100 and a Phase I/II (NCT06047379) study of NEO212.
In the Phase I portion of the NEO100 study, results saw four of the 12 patients recruited onto the trial surviving for more than three years. Something Chen claims is an “immense success”.
“For these patients to actually live for more than three years with our treatment and that’s a huge jump over what we expect normally expect, how is that possible?” says Chen.
Is it viable?
The success of NeOnc’s bid to seek a direct listing hinge on the outcome of NEO100 and NEO212 in clinical trials.
NeOnc plans to make its treatment more affordable than temozolomide alone, the current SOC. Temozolomide was developed by MSD, but the company lost its patent over the drug in 2010. According to Chen, the treatment will be priced lower than temozolomide which can cost between $4,000 – $5,000 per cycle on an average US plan. The company also plans to develop a range of nasal devices needed to distribute its therapy.
The unique delivery system makes NeOnc’s product standout in a field that typically sees the use of more complex devices. Senior pharmaceutical analyst for GlobalData, Kevin Marcaida, detailed how the company’s ability to fill unmet needs in the world of brain cancer could make a real difference.
Marcaida said: “The majority of the competition is in discovery or pre-clinical, so I see NEO212 as one of the leading drugs in this space.
“I think NeOnc’s candidates have some real differentiators. That intranasal delivery to bypass the BBB is huge, and the early clinical results for NEO212 are promising, especially the data on increased BBB penetration and cell-killing activity. The FDA Fast-Track designation also gives them a leg up in terms of development.
“With the IPO market showing signs of life, a well-positioned biotech like NeOnc could certainly attract investor attention.”
GlobalData is the parent company of Clinical Trials Arena.
Both trials of NEO100 and NEO212 are set to be completed in 2026. Until then the company will seek investment off the back of the leap that is the extended life span in Phase I trials. But as time goes on, results from either trial could determine whether NeOnc will be successful on the open market.
