Much like other biotechs which have thought they had the “Holy Grail” because evidence pointed to it early on, we could also be one of those which fails in the end stages - Ph3.
Those who have been paying close attention will realize that we have already had a chance to fail in Ph1 and we didn’t. We gathered stronger data by “letting the body decide” as Dr. Missling said recently - great quote for our approach. We used the indicators of efficacy which were taking place, on whatever scale, and used data analytics and precision medicine to enrich further phases.
We are now are a critical juncture in the process - ph 2, where many other previous drugs have failed on their quest also - or began to show signs of undesirable, intolerable side effects, or, still, no signs of efficacy above the SOC. That’s the toughest place to be...not bad enough to fail; not strong enough to know you will succeed...I guess you press on and find out in phase 3? If you have the resources on hand (dilute, only if you absolutely know you have a strong case for ph 3 approval but lack cash - and you make that clear to investors) you’ve already invested enough money and time in a drug...why fold until you absolutely have to? That’s the lack of process mindset - Nidan. That’s the gamble. We are there NOW.
Do we press on?
Well...I’m very relieved that researchers and patients and families and caregivers are asking for EXTENSIONS. I’m very relieved that, as more time passes, there appears to be no brain swelling or similar. I’m encouraged that the studies are still gathering more data which involves improvement in areas other than cognitive...the sleep studies, the motor signal detection, eeg’s for brain signals and I think we will have seizures quantified during Rett.
Ph 2 looks as though it’s pointing toward a clearer glimpse into ph 3. We don’t have any clear reason to fold - the extensions appear to indicate the opposite (and we know that a total of 50% of PDD patients are FINISHED with the phase 2 in 10 days or so). Hmmm... And they all go on the active drug from placebo group. How do we like them apples?
So, no thought of folding? No reason? Personally, I believe this IS Plan B. Dr. Missling strikes me as the type who would consider a lack of any demonstrable efficacy at this point as a losing hand, even in the light of tolerable side effects. I think his benchmark is improvement - of something(!), or forget it. He doesn’t have the cash to do a wait and see for clear signal. He would not have invested our capital in drug inventory on a whim. He would not continue to open new sites (cost) and pay for extensions (more cost) and continue partnering with pharma tech gadget companies - Cognision, consortium, if he were turning the place upside down looking for the Holy Grail. He would not continue looking for strategic “fits” at the partnering conferences (cost). He would not continue attending the investor conferences - raise capital for what??? No efficacy...hardly seems like a point and he actually needs a point.
Look at what’s in it for him: Axovant’s CEO is a great example - $600M personal worth, who needs a drug approval with that parachute...platinum? Rhodium?? Missling is making $500k salary. That’s rent control in Manhattan. That’s what some Sr VP’s in BP and other industries make. At Lilly, in Indianapolis, the SVP’s make roughly the same and one can buy a home for $450k in Fishers (a burb of Indy, although, technically they have no burbs), with plenty of creature comforts. Missling is not pulling down the big bucks. Nor is he worth hundreds of millions. Nor is he young enough, at 52ish, to start over again and attain leadership of a BP making beau coup bucks within 10 years of his career - after coming off a failed biotech stint. I’m sorry - the man is going big or going home. If the drug fails, he will lose the Manhattan zip code, the model, the career, the options, ...everything but the memories. That’s just obvious. He’s not a Branson or a Musk who will be a serial entrepreneur with fingers in so many pies and sizzling lifestyles. He will be wrecked like the Hesperus and all of our disposable investments. He could go back to managing hedge funds. Yeah.
I see this as Plan B:
Stay relevant and get approved for something(!). Period.
Based on this:
Tennis players get two serves. If you can demonstrate that you can consistently get your serves in, you will/can stay in the game and get to the professional level. If you can’t, you never make the professional level. You get 2 tries. (Not counting lets) And for those who have put in the practice, 2 is plenty - it HAS to be. Point is: there’s a limit of 2 - more is a waste of time. The analogy - Companies with more cash than skill are like tennis pros with a shopping cart filled with balls. They could theoretically keep trying to get it in all day. What a waste of time! If you can’t do it in 2, you can’t play professional tennis. You can’t compete. And often the single most relevant metric of winning a match is keeping your first service percentage up. Go figure. Basket of balls...that’s for amateurs, beginners, practice, and time wasters.
Go big or go home, Ace.
Ace has got this! He HAS to. And his service percentage is looking good - he’s really seeing the ball and hitting all the targets - short angles, out to the side, deep hard body serves, down the T... translation: extension requests, measuring data for other improvements - quality of sleep, reducing seizures, mood/agitation/BP/cardio vascular improvement, attracting highest level personnel and SAB, the preferred shares in our arsenal, the commercial inventory.
I’m predicting we got this in straight sets, without dropping serve! (French Open! Clay. Or grass, or hard - We Got it! Nice game, Raptors - xodcode! & Toronto!!)
Failure not an option...!
GL and Good Weekend,
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