Recent ATM

Forum rules
- Comments must be civil and on topic
- Back up claims with evidence/reasoning/sources (posting links is allowed)
- No commercials/harassment/spam
Post Reply
biopearl123
Posts: 430
Joined: Fri Jul 20, 2018 5:13 pm

Recent ATM

Post by biopearl123 » Sun Sep 06, 2020 5:58 pm

There has been a lot of discussion about the recent (surprise) ATM. Speculation has included raising funds for the long promised AML study, a de facto "poison pill" to prevent a hostile take over, acquisition of some synergistic company etc. But these are not normal times. My own opinion is pretty straightforward. Here we are through the first quarter of September and we have yet to see the promised PR that the half way point in the enrollment of the MDS PIII study has been reached. We ain't there. I am anticipating a modification in the promised timeline that represents complete enrollment by the end of March. Many large potential enrolling sites in the US, and around the world are still wrestling with COVID. This is just a fact. Enrollment can't be proceeding apace. The ATM ensures viability as the timeline gets pushed out. A potential acquisitor will use this as an argument to devalue the company. The ATM will help Dr. Scarlett shore up the long term viability, independence and value of the company. Over the short term however, maybe we should expect (more) bumps and yet more dilution. bp

CKTC
Posts: 12
Joined: Sun May 31, 2020 4:26 am

Re: Recent ATM

Post by CKTC » Sun Sep 06, 2020 6:31 pm

Another challenge with enrollment is the approval of luspatercept (FDA Nov/2019, EMA Apr/2020). Sales have ramped up very quickly ($55 million 2nd qtr. 2020). Many doctors will want to give their patients a shot at luspatercept first before putting them into a trial where one in three will be getting a placebo.

biopearl123
Posts: 430
Joined: Fri Jul 20, 2018 5:13 pm

Re: Recent ATM

Post by biopearl123 » Sun Sep 06, 2020 6:52 pm

CKTC, Good point but since Lus has shown efficacy mainly in RS positive patients (15% or so of MDS), I am not sure docs will be attracted to use Lus in all comers for that reason and may be selective. Also, and I base this on intuition only (not always an accurate commodity), since Lus works "downstream" and does not seem to impact the malignant clone itself, that actual progression of disease and subsequent risk of transition to high risk and subsequent AML in some proportion of patients might be a consideration. The control group is only 55 or so patients and considering this is a world wide study, (and oncologists recognize the need for better drugs than Lus), so while enrollment might be slowed, some significant proportion of Lus patients especially if RS neg, might still find their way to enrollment. By the time that happens, the study might be closed and the drug no longer available until approval. I hope oncologists will recognize this and facilitate enrollment. (We know we won't see compassionate use-- Compassion is a limited commodity these days.).What the ramp up for Lus shows us is how great the demand for Imetelstat might be when it comes to market. Best Regards, bp

CKTC
Posts: 12
Joined: Sun May 31, 2020 4:26 am

Re: Recent ATM

Post by CKTC » Mon Sep 07, 2020 6:09 pm

You have to be careful when interpreting the market size of patients with MDS-RS (RARS). Technically, to be considered MDS-RS, the bone marrow must have greater than 15% of erythroid precursors with ringed sideroblasts, or 5% in the presence of an SF3B1 mutation. So yes, it has been estimated that up to 15% of all MDS patients “technically” qualify as having MDS-RS. But in reality, up to 70% of MDS patients have some level of ringed sideroblasts in their bone marrow. With this in mind, and considering luspatercept’s benign safety profile, Celgene will push for the drug to be tried in all of these patients. Moreover, the higher the level of ringed sideroblasts, the less responsive the patient is to ESA’s. So percentage-wise, there will be more patients with ringed sideroblasts in need of blood transfusions out there, which is why 71% (27/38) of the patients enrolled in IMerge were classified as RARS or RCMD-RS.

biopearl123
Posts: 430
Joined: Fri Jul 20, 2018 5:13 pm

Re: Recent ATM

Post by biopearl123 » Mon Sep 07, 2020 7:40 pm

Ah, that would explain Dr. Scarlett's careful parse about likely increased acceptance and usage in Europe because of the strictness required in following approval parameter wording there. bp

biopearl123
Posts: 430
Joined: Fri Jul 20, 2018 5:13 pm

Re: Recent ATM

Post by biopearl123 » Thu Sep 10, 2020 8:29 pm

To put a point on CKTCs comments, it seems likely that in light of the ready adoption of Luspatercept, at least some impact will be had on enrollment in the PIII MDS study. bp

Post Reply