Thread by @biotechinvstr

Sizing up some 2Q biotech events and consistent theme emerging among many of them is the likelihood of spin/deliberate obfuscation/sleight of hand/bait and switch/general smoke and mirror confusion. So to avoid a $FGEN esque repeat, laying out some of the more obvious candidates-

$IFRX - the most likely candidate to have a "announces positive data" headline?
Will it really be positive? Slim chance

Details, assuming they provide any, will be very very important. This is a team that has the gall to blatantly claim that THIS figure is highly stat sig (1/4)

if they can claim that, what's to prevent them from claiming that the obscure (for a reason) stats plan they're using for the upcoming Ph2b, the MCP-Mod led to "stat sig/positive" results? Recall the whole basis of $IFRX 1.2B market cap is a n=12 single center Greek study (2/4)

To be truly "positive", need to see clear dose response, and given C5a MoA, the highest dose NEEDS to be the best dose. Otherwise $IFRX just retrospectively cherrypicked a dose that *happened* to work--> in that scenario trial officially is a failure. Cash ~$4/sh or 90% downside

And then to close the loop, here's a scenario analysis from Leerink, which suggests that even if the trial is "positive" with a 60% response rate on the drug (ie for the highest dose as described above), given the weekly/biweekly administration will not be competitive with Humira

$UBX- another "announces super duper positive results" headline in the making? Not so fast.
Company is running a randomized 6-12 week long pbo controlled study in Osteoarthritis and puts a "How to define study success" slide in deck and that any effect on pain is "upside"!! (1/3)

Instead, $UBX contends we should consider this fantastic use of shareholder capital a "SUCCESS" if any 3 out of 24(!!), yes 24, inflammatory markers they're measuring show a trend towards reduction. (note courtesy Citi). After multiple delays/amendments to the trial, mind. (2/3)

This is the first clear test of $UBX platform, valued at $450m with ~$130m cash end of Q2.
For the trial to be truly "positive", there NEEDS to be some dose response and clear effect on WOMAC and pain endpoints. Anything else is contrived smoke and mirrors, and an absolute fail.

$TOCA- if the trial continues, basically means anyone holding on to the stock is hoping that the effect size which isn't apparent in 200 events shows up in another 57. In other words, hoping can squeeze in a 0.02<p<0.05 window. Leerink analysis on stock reaction in this scenario-

$BCRX- yet another BCRX readout. The setup for the very overpowered HAE ph3 this time is that the trial can be stat sig and therefore tee up a "announces positive results" headline even on 40% pbo adjusted reduction.
Does that matter commercially? No. Takzhyro is 85-90%. (1/2)

Investor threshold for commercial viability is ~low 60s placebo adjusted (very very important to note that nuance). The farther away $BCRX is the worse for the stock. Below 50%... well, net cash is ~$1/sh, no matter how "positive" the topline PR makes the data sound

And of course for all of these, we are looking for the usual (where they apply) ITT population, 95% confidence intervals, 2-sided t... just general courteous practices of being transparent enough to stakeholders, not just propping the stock on crutches to do a raise and get paid

$BCRX where to from here-

Cash as of 3/31 at $128m, burning $30m/q, and $80m debt, so < $1/sh

The issue with HAE is that attacks can be fatal, especially if laryngeal. Need really good coverage to feel “safe”. Tough to prescribe when comp has 85%+ efficacy

$BCRX best shot would’ve been a few hyper-responders. Their PR (with the 70% arbitrary cutoff) actually indicates the other 50% patients have <30% attack reduction, so how does a doc select patients without putting them at risk?

Not to mention that the drug is known to have GI side effects (diarrhea shows up again in this Ph3) and liver tox so the “convenience” aspect is misguiding at best.
Monumental education effort required,which translates to a lot of capital needed (assuming this even gets approved)

This is yet another $BCRX failure at attempting to develop an oral HAE alternative. Good luck trusting this management team to turn the story around (those recent insider sales are definitely not a good look either)

$BCRX one discontinuation due to abnormal liver test, confirming the previous concerns from the Phase 2

$BCRX doesnt state this on the slides but mentioned on the call that they did not hit the secondary endpoints. Another knock against the "convenience" of an oral therapy if they can't improve patient Quality of Life

$BCRX does this get approved?

Placebo underperformed relative to other large HAE trials (See lanadelumab below). ~40% in other trials versus ~20% reduction in BCRX trial today. Was there any unblinding due to tx being oral? Where are time curves?

Should be a fun Adcomm.

For those holding out hope for $BCRX acute program, I would encourage you to ask the mgmt these questions-

- Was the ph2 truly unblinded?
- Doesn't it have a distinct strong unpleasant taste? Is it true patients can't mix it or drink anything 30 min before or after?