After Danny and I did deeper digging, while we think secondary AML will hit, but the overall NCI denovo AML topline data will not be stat-sig.
However, we also think the company will bury this as they don't have to say anything until way later this year. Remember, officially it's not their trial
The #1 rule in biotech data when especially unblinded, is that the longer it rakes to reveal the said data, the more likely it is that those running the trial are data mining looking for positives because the primary endpoint has missed on HR > P.
This also applies to blinded studies BUT ONLY AFTER the data has been unblinded (too long to receive data after un-blinding also indicates investigators are data mining for a cherry-pick). Remember, The NCI trial here is not blinded. So, we do think sometime on or right before Oct. 2nd, the company received the topline data, but because they have guided "we will tell you the topline when we get all the data set," so we believe they've been sitting on it LEGALLY and have instructed The NCI to data mine/post hoc the entirety of the data set (we do think secondary will show a win but not stat sig due to low power on that because trial lacked stratification for that) = why it's taking so long (In Company's P3 trial still being blinded and has not reached 100% events and follow-up at 26 months and counting, the above absolutely DOES NOT APPLY to this trial).
The real STORY with GLYC in our opinion is transplantation > Mucositis benefit > huge safety benefit, surrogate markers (Company can prove reason for mucositis benefit, show said benefit, and show non-inferiority concerning efficacy at minimum) but more importantly, big mucositis benefit IN MM EQUATES TO HIGH UNMET NEED safety benefit in frontline MM transplantation. FDA has not updated guidance in the Hematology area in ages - we think they hired Dr. Rock because he is an old school FDA guy who can get this approved on these.
Our Take Away;
- Refracted trial will hit big, expect read-out sometime near end of Q1, beginning of Q2 2024, expect HR < 0.60, P value <0.001 for the primary endpoint (OS) = AA certain, possibly full approval depending on how strict or not FDA feels (lately, they're all over the place on their guidance). Peak sales> $800M based on 8000 patients, but pricing uncertain now - could peak out over $1B.
- We believe MM trial has hit already (topline safety, possible some exploratory endpoints, but not needed for AA here, wide open unmet need). this will be the big surprise for the market in our opinion, and why we think CMO and others rushed to buy. We believe this trial design is well-controlled and powered correctly for an AA (NCI trial isn't), so we also expect to hear that company has received break-thru designation for this.
- NCI Topline denovo (newly diagnosed AML patients) data is not/will not be stat sig but eventual secondary AML (Progression to AML from MDS) will, but likely will use a 1687 future trial for that because the powering would not be correct for AA in secondary here based on lacking stratification) which is why it lists 1687 for AML on their corporate deck. 1687 cuts down on hospital costs because it can be used as a subcutaneous solution at-home by patients. We think company will basically "bury" this so-called failure by delaying to receive ALL THE DATA from The NCI. Anyone who has worked with The NCI knows it's easy to get them to comply as they LOVE GRANTING 🙂
- Partner for Sicklecell in which we strongly believe NOVARTIS is here and company is finishing up the deal now (reason for 10K yearly and Q4 earnings delay).
- Acquisition after refracted read out next year, but possibly sooner if conditions are right
Scott
