With all of the buzz around big data and artificial intelligence (AI) in drug discovery, the research team at the biopharma company BPGbio focuses on the source of that data — patients.

“We talk about patient centricity literally,” said Niven Narain, the Chief Executive Officer of BPGbio. “We start our discovery processes and our development by taking, for example, tissue from disease patients, from patients without the disease, tumor tissues, organ tissues, blood, urine, etc. And then, we have all the patient records.”

At the recent American Association for Cancer Research meeting (AACR 2025), Narain explained how his company’s focus on patients has powered their advances in drug discovery and development. This is especially true for their mitochondrial-targeted drug BPM31510, which is showing promise in hard-to-treat cancers including glioblastoma (GBM) and pancreatic cancer.

The core of BPGbio’s research starts with their biobank of patient samples. They perform all sorts of multiomics analyses on these samples: genomics, proteomics, spatial profiling, lipidomics, metabolomics, and more. From there, they feed that information into a Bayesian AI program, which helps them identify patterns in different tumor types or identify potential biomarkers or drug targets.

“The last step is the most important step, which is to go back in the wet lab and do CRISPR, RNAi knockouts, etc., to see, do we actually see a change in phenotypic functions that are disease relevant?” Narain said.

This patient-AI-validation workflow pointed the team toward the mechanism of their drug BPM31510. This therapy is a formulation of coenzyme Q10 (CoQ10), which is a vital enzyme in the energy production process that occurs in mitochondria. This energy production pathway often becomes altered in cancer in a phenomenon called the Warburg effect. Rather than using oxidative phosphorylation in mitochondria to create energy, cancer cells generate most of their energy through glycolysis and lactic acid fermentation (1).

In new data presented at AACR 2025, Narain and his team showed that in a rat GBM model BPM31510 reverts the cell’s metabolism from the Warburg effect back to normal (2).

“What we're finding is that this drug is actually intercalating and reconstituting mechanisms where we're making the tumor metabolically cold but immunologically hot, so [these are] tumors like pancreatic and GBM and triple-negative and certain high-grade prostates,” said Narain.

The standard of care for GBM is chemotherapy with temozolomide and radiotherapy, but Narain said that tumors can become resistant to radiotherapy and that temozolomide can cause mitochondrial instability. But with BPM31510 treatment, the team found that “we're abrogating the resistance that you would usually see in radiotherapy,” said Narain. “We're also making mitochondrial membranes more rigid.”

A lot of comments were like, ‘Are you guys crazy? Are you going to do the two worst cancers? You're going to set yourself up for failure,’ essentially, because they called [GBM and pancreatic cancer] the graveyard of drug development.
- Niven Narain, BPGbio

The team is currently also in the middle of a Phase 2 trial testing BPM31510 in newly diagnosed GBM patients (3). That trial is continuing to recruit patients, but Narain is excited by the results he’s seeing so far.

“We're confirming that the drug is not only well tolerated, but we are starting to see some really, really promising signs of efficacy, of activity, in this Phase 2b trial,” he said.

When they first started out targeting both GBM and prostate cancer with BPM31510, that hopeful outcome seemed almost foolhardy to hope for, according to some of Narain’s colleagues.

“A lot of comments were like, ‘Are you guys crazy? Are you going to do the two worst cancers? You're going to set yourself up for failure,’ essentially, because they called [GBM and pancreatic cancer] the graveyard of drug development,” said Narain. “But the platform helped us not fall prey to the graveyard. It helped us to do construction around it.”

In addition to their Phase 2 trial in GBM, the BPGbio team is testing BPM31510 in clinical trials in pancreatic cancer and the ultra-rare disease primary CoQ10 deficiency. They also have drugs in clinical trials for sarcopenia and epidermolysis bullosa. They continue to take advantage of their biobank and AI platform to pursue new molecules and targets. But, most of all, Narain and his team always bring their focus back to the patients.

“When you're at the hospitals and meeting the patients and talking to physicians, and when you're literally listening to their journeys, that gets me,” said Narain. “That’s what matters.”

References

1. Liberti, M.V. et al. The Warburg Effect: How Does it Benefit Cancer Cells? Trends Biochem Sci 41, 211 - 218 (2016).
2. Kiebish, M.A. et al. Abstract 1527: Reversal of the Warburg Effect with BPM31510 treatment in a quinone deficient C6 Glioma resulting in efficacy. Cancer Res 85, 1527 (2025).
3. Stocksdale, B. et al. Abstract CT242: A phase 2 study of BPM31510 (a lipid nanodispersion of oxidized CoQ10) with vitamin K in combination with standard of care (SOC) RT and TMZ in glioblastoma multiforme (GBM) patients without prior therapy. Cancer Res 85, CT242 (2025).