Sramana Mitra: I’d like to explore two directions based on what you said. Have you actually developed a drug using this methodology or have you facilitated drug development for a pharmaceutical? If you’re allowed to talk about that, I want to do a use case of stepping through the whole process.

Joga Gobburu: Absolutely.

Sramana Mitra: The second direction is the economics of it. Yes, it costs billion plus to develop a drug and 12 to 15 years to bring it to market. Applying your solution to this, what is that doing? Does that compress the timeline and optimize the budget?

Joga Gobburu: This is a case study which started in the clinical development phase for this midsized company. The disease is rare and there are not treatments at that point when we got involved.

Sramana Mitra: When was this?

Joga Gobburu: Three to four years ago. The company was far ahead in terms of clinical trials for adults. It was a very long clinical trial. It spanned over five years. That was the basis for the Drug approval by FDA. Where we got involved is what do we do now with pediatrics? Pediatrics poses multiple problems. The body is maturing. There are changes going on rapidly compared to an adult.

Whatever clinical measures that are applicable to adults can be applicable to pediatrics. Because there is growth, these trials last 10 years. It’s infeasible to take a case of a patient who’s one year old. By the time the trial finishes, he will be nine years old. Why would we want to wait for nine years for a rare disease? I wouldn’t want that for anybody I know.

The trial is infeasible in pediatrics, but they do need a drug. The advantage is that the drug is shown to be efficacious in others. Pumas comes in. We took an entire database from the adult trials. Several markers were collected both from efficacy and safety point of view. That was thoroughly analyzed to identify a surrogate marker. That would be a marker that can be used in lieu of the ultimate clinical measure.

We came up with an abbreviated program for identifying preemptive markers where you don’t have to wait seven years. We found something that can be used to extend the drugs to pediatrics.

Sramana Mitra: How?

Joga Gobburu: From the clinical experience for the adult program, you have lab measures. You have what patients feel about the drug. What are the side effects? Because there was no drug approved before this, we didn’t understand the biological interplay. Let us say that you take a disease like diabetes. People are worried about retinopathy or nephropathy. Those are the bad things that can develop. It takes time for those to develop. The way it’s managed is through glucose measurements.

Sramana Mitra: And medication.

Joga Gobburu: That’s because it’s proximal to action. You can look at the glucose measurement and make changes. You don’t have to wait till somebody loses an eye. Same thing with oncology. People make decisions based on tumor sizes and such.

Ultimately, people are worried about survival. Similarly, there wasn’t a similar preemptive measure that, if I showed that, I could effect this in the right direction. You don’t have to wait till the long-term effect in pediatrics to get the drug approved. Thereby, you leverage the knowledge using extensive data analytics.

This is a very rigorous and regulated industry. Pharmaceutical is a very highly-regulated industry. It has be scientifically perfect. The company has to submit it to FDA for approval even to start the trial.