Unknown Kentucky biotech Bexion tests new glioma med on first patients. Yes, it’s the same cancer Sen. McCain is fighting, and Round B funding is still open.

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The News:

Bexion Pharmaceuticals burst out of the shadows of near total obscurity last week as its investigational cancer treatment, BXQ-350, grabbed plenty of mainstream media coverage as a potential game-changer.

BXQ-350 is a formulation of a synthetically produced, human lysosomal protein, Saposin C (sphingolipid activator protein, or SapC), and the phospholipid dioleoylphosphatidylserine (DOPS). It is being developed as a first-in-class treatment for solid tumors and gliomas, according to Biospace.

In animal models, the effect of BXQ-350 has been significant. The Cincinnati Enquirer / cincinnati.com chimed in, saying that the medication fused to the walls of cancer cells in mice and killed them–without harming healthy cells. As animal research continued on various cancer types, the results seemed to be the same–death for the cancer cells.

As development progressed on the drug, the newly-formed Bexion began to receive financial support from a number of agencies, including the National Institutes of Health, and the National Cancer Institute. The drug was finally cleared for human testing and Bexion got straight to work.

Earlier this year at the American Society for Clinical Oncology (ASCO), Kentucky-based Bexion unveiled preliminary data of a Phase 1a trial in a poster presentation that revealed some promising results.

Of the 17 patients, nine with glioblastoma multiforme and eight with other solid tumors, the company saw a positive response in seven patients after 113 days. One patient with appendiceal carcinoma saw a partial response and six patients showed signs of disease stability. Of those six, one was a high-grade glioma patient who showed stable disease signs for greater than 19 months, according to company data.

The glioma patient is Bob Rulli, a retired airplane engine designer. He is the focus of the USA Today article that is racing across social media platforms. Rulli has already lived with his glioblastoma multiforme for nearly five years. Average life expectancy is less than two years. He’s been on the Bexion treatment for 19 months.

Although his cancer hasn’t been eradicated, the tumor has proven to be stable during that time–meaning it’s not become worse. That wasn’t the remarkable news, however. In May, Rulli underwent an MRI and the scan showed what appeared to be dead tumor cells. Bexion and other medical officials were quick to note that Rulli is something of an outlier when it comes to drug response, particularly with glioblastoma multiforme.

At ASCO’s June meeting in Chicago, Bexion also shared additional good news about BXQ-350. Not only did those seven patients have positive responses to the drug, there were also no dose-limiting toxicities observed and no treatment-related serious adverse events.

Bexion now has its sights set on further clinical testing. Ray Takigiku, founder and CEO of Bexion, said the company is enrolling patients with solid tumors and gliomas in Phase 1b testing and is eying a Phase 2 trial in adults. Bexion is also initiating efforts for a Phase 1 trial in pediatric patients, Takigiku said in a statement following ASCO.

Meanwhile, Rulli has carried on with his treatments and continues to defy the odds.

Steve’s Take:

Talk about hiding in plain sight. Well, in Covington, KY–just across the Ohio River from Cincinnati and Sausage Row. It’s the fifth-most-populous city in Kentucky. But I digress.

In early November, I posted a piece on the announcement by the University of New Mexico Comprehensive Cancer Center (UNMCC) that the first clinical trials of a new drug that targets solid cancer tumors had been conducted. The Phase 1A clinical trials marked the first time ever the drug, called BXQ-350, was being used in people, according to Science Daily.

The trials had opened at several sites across the country and patients like Sen. John McCain (R-AZ), with a type of brain cancer called glioblastoma, had new treatment options. Olivier Rixe, MD, PhD, at UNMCC, oversaw the clinical trials’ national protocol development and directed the Phase 1A trial in New Mexico.

The drug, called BXQ-350, had been shown in pre-clinical studies to induce cancer cells to die but have little effect on normal cells.

Steve's Take: @BexionPharma potential #cancer cure is definitely on my watch list! Click To Tweet

Now we have more news that tiny Bexion does apparently have a truly exciting product under development. But as with so many other promising cancer treatments, moving forward, the company will need to raise additional funding. Bexion executives told USA Today that the company has about $40 million cash on hand, but will need to raise significantly more to advance testing of BXQ-350. A lot more.

Bringing BXQ-350 into the realm of clinical trials in humans under the eyes of the US Food and Drug Administration is a punishing ultra-marathon. About 90% of experimental drugs fail FDA testing. Along the way to this point, several federal agencies gave Bexion’s work votes of confidence.

In 2010, the National Cancer Institute granted $1.5 million for research. In 2013, the NCI bestowed its Bridge Award of $2.9 million. In 2015, the Small Business Administration took note of Bexion’s “innovative technologies that address national priorities.”

But in 2014, the government waved the stop sign on BXQ-350. The FDA demanded more animal studies before considering human trials. The setback meant even more fundraising just to get to human trials. Though frustrated, CEO Takigiku said he understood the hesitation.

“Everything was new, a new mechanism, a new compound, and that just causes pause for a regulatory body that is concerned with patient safety,” Tajugiku said. “They’ve never had a drug with ambitions of attacking any solid tumor with no damage to surrounding tissue.”

Bexion now is planning for a Phase 2 trial to measure the efficacy of BXQ-350 on any solid tumors in adults, and a Phase 1 study in children. If the FDA approves, one of the pediatric sites will be Cincinnati Children’s Hospital, where 16 years ago, researcher Xiaoyang Qi looked in his microscope and got a surprise. (More on Qi below.)

“Cancer is very smart”

The performance of BXQ-350 in humans entices researchers. But experience dampens optimism. It’s far too early to predict whether it will be effective or who will ultimately benefit. The sheer cost of more testing is daunting. Bexion has raised about $40 million in private financing and grants. Cracking the secrets of BXQ-350 will simply take far more dough.

“God’s honest truth is: We just don’t know how it works,” CEO Takigiku said. “We’re going to find out, I hope.”

Bob Rulli’s doctors say that years from now, if the FDA eventually adds it to the arsenal against cancer, BXQ-350 could be used in novel combinations with the traditional weapons. They hope the drug might give patients a real edge against such a pervasive killer.

“We have to be smarter than cancer,” said genetics researcher Xiaoyang Qi, credited with first discovering the action of a similar human protein on mice. “We can’t let cancer be smarter than us. Right now, cancer is very smart,” Qi informs.

For brave, high-stakes investors only:

 According to ClinicalTrials.gov, as of August 14, 2018, there are 130,271 registered drug or biologic trials ongoing in all 50 States and 204 other countries. That affords some context to the handful of trials Bexion is seeking to undertake. And all of these trials are expensive, whether or not the meds ever get to market. It’s very, very early, and the competition for funding in this space is brutal. Plus Bob Rulli may be a true outlier and not at all representative of the majority of trial patients.

Bexion, however, seems to have a rational shot at some success with BXQ-350. It is ground-breaking science, and that’s rarely the case. The company recently increased its Series B open-round raise from $10 million to $15 million. Information for ultra high-risk/reward investors can be found at https://www.bexionpharma.com/investors/. Those investors interested in participating in this round should contact Margaret van Gilse; [email protected].

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