Syntara Limited (SNT) Earnings Call Transcript & Summary

With me this morning from Pharmaxis is Gary Phillips. Gary, good morning.

Good morning, Andrew.

Good to see you, Gary. Important update this morning on your lead clinical program in bone marrow cancer, where you've completed the dose escalation phase here. Tell us a bit more about the results.

Yes, it's an important step in the study. As a reminder, this is an FDA approved under an IND study. It's in myelofibrosis, which is a sort of rare kind of bone marrow cancer. And the study is split into 2 halves, and we've reported on the first half today. The patients being recruited into the study all have myelofibrosis, and they're not allowed to be on the current standard of care, which is a JAK inhibitor. So these patients are ineligible for the current treatment. So they tend to be quite sicker end of the patients as well. And the objective of this first half was to find the right dose for the study that comes after it. So we trialed 3 different doses, each of them higher than the last. And each of the patients went on each of the doses for a 1-month period. And the design of that was so that we could look at safety. So are there any adverse events coming out, and then what were the inhibition levels we achieved of the enzymes that we're targeting. And on both counts, the study, I think, we can say is an unqualified success. So the -- from a safety perspective, we didn't see any adverse events at any of the 3 increasing dose levels, which is really great. And secondly, we saw good inhibition of all of this family of lysyl oxidase enzymes, which again, we were very pleased to see.

As you say, it sounds as though the drug is inhibiting these lysyl oxidase enzymes. What does it mean for myelofibrosis patients here, Gary?

So we're extrapolating from some preclinical work that was done in the U.S. by some independent investigators. And the lysyl oxidase family of enzymes are involved in fibrosis. So they get involved in cross-linking of collagen and elastin fibers, and that causes scar tissue. And in myelofibrosis patients, it's scarring of the bone marrow. And once the bone marrow gets scarred, then it stops producing red cells, white cells and platelets, which causes a host of other problems. And these patients have about a 5-year life expectancy from diagnosis. So in the preclinical models, we found that by inhibiting these lysyl oxidase enzymes with our drug, then in the animal models we use, those animals saw improved cell counts, reduced fibrosis of the bone marrow and reduced spleen size, all hallmarks of the disease. So it looks as though if you can inhibit these enzymes, then you actually cause a disease-modifying effect, which is what we and the patient community and the clinicians are all looking for. So I think at this stage, after that first stage of the study, where we're showing we give full inhibition of all those enzymes, that's as good as it gets. And now we go to the second part to Trial C, well, what does that -- what happens clinically.

Well, tell us more. Where to from here then from the study?

So the second part of the study will start very quickly now. We've recruited sites in Australia, South Korea, Taiwan and the U.S.A. We've got to recruit 24 patients in total, and we're going to treat them for 6 months at the highest dose that we tried in the dose escalation of the study, which was just completed. So because we saw no side effects at all, we've got the luxury of picking the highest dose. That highest dose gives the highest inhibition of all those enzymes. Those patients will be treated for 6 months. And at the end of that, we'll be looking to see, a, again, whether we have -- we've repeated the safety profile, which we've seen in the first part of the study. But then we'll be looking to see what happens to their bone marrow. So we'll be taking bone biopsies to look at the fibrosis of the bone marrow to see whether we can change that. We'll also be looking at their blood cell counts to see whether we can improve their platelet red cells and white cell counts, which is -- would be something which existing treatments don't do. And then we'll also be looking at the spleen size and their symptom scores as well. So by the end of next year, this study should be reporting, and we should see whether we're getting a disease-modifying effect with our drug. So a big tick for the first start of the study and really looking forward now to getting these patients on for a longer period of time.

Good to see you, Gary. Thanks very much.

Thanks very much, Andrew. Good to talk. Thank you.