CEO William Rice, PhD, is making an Aptose presentation at an oncology conference today in Valencia, Spain. We spoke briefly yesterday, and he offered to forward the slide deck for the talk to BioPub for perusal. Of note, it includes important new data, done in collaboration with Michael Andreeff, MD, PhD, of UT MD Anderson in Houston.

Please pull up the new deck from the link below and scroll to slide 15. Here we have comprehensive patient sample data for how CG’806 kills, in a potent manner at low concentrations, leukemic cells harboring a considerable variety of FLT-3 mutations. Mutated FLT-3 is a consequence of the times: the more agents are invoked to inhibit FLT-3, the more often we are seeing escape and resistance mutations, many of which go on to become fierce oncogenic drivers. The evidence here is that CG’806 appears capable of subduing any mutant form of FLT-3 hurled at it. FLT-3 is a tyrosine kinase, and tyrosine kinases are enzymes that phosphorylate the benzyl side chain of selected tyrosine residues on target proteins. (“Kinase” is purely in the metaphorical sense, as in imparting kinetic energy.  kinase makes its target go vrrroom vrrroom.) This phosphorylation induces shape changes in the target protein and has a way of either activating that protein or putting it into idle mode (usually the former). Kinases are switches, on/off thingies, for their targets, and usually the switch gets turned on. Some proteins have low (but not zero) level activity until acted on by a tyrosine kinase, which turbo-charges their biological avidity for getting involved in cell biochemical reactions.

Please all review additional data on slide 16 as well as discursive points on slide 17.

Spain GCC 2018 Presentation CG-806-Aug 02 2018

Why is this new data so important? While it doesn’t quite rise to the level of being clinical data—and clinical data are what potential institutional investors are here hungry for—it is very robust, in-your-face, pre-clinical data and is so fine a simulacrum of a real world road test of CG’806 as to be very persuasive to clinicians. One of the most compelling strengths of MD Anderson as an institution is its enormous experience with a vast range of problematic cancer cases, this in addition to its enormous clinical volume and throughput. Anderson has a way of regarding every patient’s case as potential academic material, as harboring potential information to move the investigative clinical ball forward. Anderson also has led the pack in databasing its patients. So fertile is the trove of clinical data stored at MD Anderson that fellows and junior faculty can easily make first forays into making a name for themselves by mining institutional data, data current faculty are often hard-pressed to find time to do much with. Anderson faculty are arguably the hardest working in academia, so much so that when you sign an employment contract there, as I did, you are advised that during your time of working at the institution you WILL be stressed and find yourself in “intensive interpersonal situations” working very hard with your colleagues and facing large patient loads. And that you need to embrace that or not sign on. When I first began working at MD Anderson, my life seemed to have returned to something as demanding of me as a medical internship, though I was happy with that.

The new data allows CG’806 to strut its stuff against difficult to treat AML patients in test tube proving ground. Andreeff and colleagues have stockpiled for years bone marrow specimens from AML patients; the institution has one of the largest AML practices in the world, perhaps the largest. Andreeff meticulously analyzed those genomically, tracking and cataloguing sometimes arcane and seemingly insurmountable mutations, including FLT-3 variants with rare mutations. Andreeff has here thrown the most brutal of FLT-3 mutations at CG’806, and proven that the agent makes short work of them. CG’806 continues to pile up evidence that it is an anti-cancer Leviathan. And it’s an elegant clinically relevant kind of experiment. Even if expression clones of all those variant enzymes existed, doing tyrosine kinase activity assays is tedious. Instead, take cells known to be driven by mutant tyrosine kinases, show that a versatile tyrosine kinase inhibitor kills the cells, and you have nice proof in an indirect but integrative way of the drug’s powers.

I’ve always said that good biotech has a foot in academia and that good clinical academics has a foot in biotech. Having conversed with Rice as heavily as I have, I find it easy to regard him as drug-development royalty, but then so is Andreeff, whom I also know. Here we get an external validation of the great merits Aptose’s work has, in the form of active collaborations between Rice, Andreeff, and also Brian Druker, MD, inventor of imatinib (Gleevec, an inceptional malignancy-associated tyrosine kinase inhibitor). These men are the holy trinity of oncogenic tyrosine kinases. For Druker to be a future Nobel laureate in medicine would be utterly unsurprising. Druker, if you didn’t know, is head of Aptose’s strategic advisory board.

Dr Rice and Aptose SVP and CFO Greg Chow will discuss the company and take your questions in a live call with BioPub in early September.

Disclosure: This is not a solicitation or recommendation that anyone trade in $APTO shares. Dr. KSS has a long position in $APTO and will not trade in those shares for 7 days, reckoned in business days, after this column appears. BioPub has no pecuniary relationship with Aptose or its management. Personal diligence is due before purchasing any biotech equity, and readers are expected to be accountable for their own investing decisions.

Aptose CEO Rice is a director at $ONCY. Dr. KSS has a long position in $ONCY.

[NB: If any reader or editor knows how to place the above slide deck in the BioPub media center along with a label, I’d be grateful. I personally don’t know the trick. Thank you.]