Here is where I stand on $PEPG now
Obv very happy with the result. Considered selling in the AM but didn't because I think it's worth more. I may regret that as it continues to fade - think a lot of folks were in this for the readout.
40k pts in US so even if you only assume 10% penetration that’s $1b peak rev at $250k net price. That tracks with Novartis having bought avidity for 12 billion (a good chunk of that being the DM1 program).
Thats also conservative penetration given 1) BIC splicing correction and vHOT and 2) Novartis having just failed their pivotal and losing first mover adv.
There is probably some concern in the market about vHOT being noisy and hard to get a repeat hit on in a pivotal, after what happened to Novartis.
PEPG will read out 12.5 mpk MAD dose in 1h27 and I think they might see renal tox there. I don’t think that matters given 10mpk (what they just read out) looks BIC but there could be a negative reaction to it.
Also PEPG tends to go up by more than 100% on these readouts and then fade back down. So that could happen.
Even if you only assume 10% penetration and 33% PoS I think it’s worth $1b right now ($1B peak w/ 3x multiple x 33% pos... maybe a bit less if you discount it back to present from time of launch, but still conservative assumptions). So my default is holding but I may sell some if I’m feeling skiddish in the coming days.
I definitely won't tell you you SHOULDN'T sell if you own this, but I have not sold anything yet
I am back in $PEPG for the next readout, the odds are too rich.
I think a reasonably likely outcome is that 10mg MAD looks more or less the same safety wise as 10mg SAD. Renal biomarker (15mg SAD) appears to have more to do with acute exposure post-dose than accumulation (muscle accumulation increased ~12x from 5mg SAD to MAD but safety looked the same).
Also think it's likely 10mg MAD shows BIC splicing correction. PEPG's differentiated MOA (bind/block the repeats) appears more potent than Novartis/Dyne's (knockdown the transcript). We've yet to see saturation of the dose effect on splice index improvement, and the amount of drug in muscle is about to go way up.
I do not care about the outlier in the 5mg MAD cohort - it's unlikely (not impossible) to cause an issue in the 10mg MAD. Mgmt says it was a 97th percentile event. Literature suggests 86th percentile. Either way the odds are on your side.
I think the degree 5mg SAD splicing correction was actually encouraging.
US clinical hold is related to blood pressure drops in animals. Mgmt says they worked out the problem years ago (FDA saw this data in 2024) and this has never been seen in humans.
40K US x 10% penetration x $250K gets you to $1B in peak rev. I think DYNE and Avidity's DM1 programs account(ed) for at least a billion or two of NPV.
Could certainly crash and burn but at a little over $100M FD market cap, seems like the house is offering good odds. If it does crash let's hope RA can engineer another CLYM like transaction
I am back in $PEPG for the next readout, the odds are too rich.
I think a reasonably likely outcome is that 10mg MAD looks more or less the same safety wise as 10mg SAD. Renal biomarker (15mg SAD) appears to have more to do with acute exposure post-dose than accumulation (muscle accumulation increased ~12x from 5mg SAD to MAD but safety looked the same).
Also think it's likely 10mg MAD shows BIC splicing correction. PEPG's differentiated MOA (bind/block the repeats) appears more potent than Novartis/Dyne's (knockdown the transcript). We've yet to see saturation of the dose effect on splice index improvement, and the amount of drug in muscle is about to go way up.
I do not care about the outlier in the 5mg MAD cohort - it's unlikely (not impossible) to cause an issue in the 10mg MAD. Mgmt says it was a 97th percentile event. Literature suggests 86th percentile. Either way the odds are on your side.
I think the degree 5mg SAD splicing correction was actually encouraging.
US clinical hold is related to blood pressure drops in animals. Mgmt says they worked out the problem years ago (FDA saw this data in 2024) and this has never been seen in humans.
40K US x 10% penetration x $250K gets you to $1B in peak rev. I think DYNE and Avidity's DM1 programs account(ed) for at least a billion or two of NPV.
Could certainly crash and burn but at a little over $100M FD market cap, seems like the house is offering good odds. If it does crash let's hope RA can engineer another CLYM like transaction
$PEPG
Serious TRAE in 10mg/kg MAD readout seems incrementally less likely now that DSMB has recommended proceeding to 12.5mg/kg after reviewing the safety data to date
$GUTS Filing a PR to announce the results of some surveys you asked consultants to run for you after your stock fell 90% is one of the most depressing and pitiful moves I’ve ever seen in biotech @Harithr
@puppyeh1 Feels like $STOK at $6 when they got crushed after the biogen ex-US deal and data were years away (but funded). No way this doesn’t run up into Ph3 readout from the current price IMO
@Biohazard3737 What do mean exactly? Like validated MOA but questionable market opp? Awesome market opp but questionable MOA? Asking because you juxtaposed these against "quality" names