We are at an inflection point with
#AI
development and need to take action as a society to mitigate our risks. Here I propose a step forward that could move us toward a safety engineering mindset. Why not this, and why not now?
#AGI
#AIsafety
Biotech thread of the day; $INFI. This company has a first-in-class, oral, once-daily, immuno-oncology development candidate that selectively inhibits PI3K-gamma. I think the valuation of this company is not reflective of the massive market opportunity for this drug. 1/10
Here are some notes on a few stocks I like that I haven’t seen getting much attention on
#biotwitter
. Not investment advice.
$LRMR – I think 50 mg has a good shot at showing FXN increase because of dosing schedule and prior data. In general I think therapeutic window and margin
Biotech Idea of the Day: aTyr Pharma ( $LIFE ). For those that follow me, yes, I have posted on aTyr before, but I wanted to do a more comprehensive deep dive on the story, and now could be a good time to take another look.
These wall-cross pipes are getting absurd. Funds must think the $KURA data must be quite good to give them a 30% premium after the recent run-up. Lots of funds must have seen the data, so leaks are very possible. Meanwhile holders won't even see the data until next week? At what
Cryptocurrencies are worth $2.5 Trillion.
$TSLA has gained >$200B after a $4B deal with Hertz.
$PFE gains $30B for world-changing Covid-eliminating pill.
Imagine what good we could do if society was better at rewarding what's important. $XBI $IBB
$PDSB cervical data responses were good (100% ORR / 89% CR vs 74% CR comp) but to me the more exciting part is the elimination of circulating tumor DNA, t-cell infiltration (vs none seen SOC), and other biomarkers. This more than the small CR improvement shows it’s working. 1/5
Biotech Idea of the Day: $LIFE (Atyr Pharma) recently demonstrated excellent proof of concept for their drug Atyr1923 in pulmonary sarcoidosis in a P1b/2a study. The market has not caught up with how transformational this data is, leaving the stock significantly undervalued. 1/34
1. I’m embarking on a tweet storm explaining why I think $GBT is significantly undervalued relative to analyst estimates (>10 with PTs of $60-$80 / share). I will cover 1) Efficacy, 2) Safety and 3) Market Potential. First efficacy.
I rarely buy on M&A speculation but bought some $AUPH today. So many signs suitors are circling, and these deals take a while to execute, especially with multiple parties involved. I think the speculators gave up too early here. We'll see.
I have accumulated a fairly sizable $AVRO position. They have a $40M MC, ~130 M cash (my Q2 end estimate), ~$15 M debt. Some possible upside scenarios:
My new book has been released! DETONATION has been awarded the Kirkus Star (awarded to 10% of the books they assign for review).
#scifi
#ai
#greatreads
#fiction
“…highly entertaining and absorbing…” —Kirkus Reviews (starred review)
Discover it here:
Re $OCUL, to add my 2c. I don't know a lot about ophtho, but I know something about dealmaking, and I think the high expectations of the market for a BP deal in June were misplaced. When you have multiple parties in a licensing scenario with different geographic footprints, large
Biotech Idea of the Day: $OTIC. This co. has a number of injectable treatments for the ear, including ones for hearing loss and tinnitus that both read out mid-2022. Market cap is ~$105 M (including pre-funded warrants) and they have $98 M cash on hand as of the end of Q2. 1/18
Think this is likely just the beginning of the dance for $MYOV. Sumitomo had nothing to lose by floating a lowball offer. When Roche bought Genentech it was a similar situation (Roche was majority 55% shareholder and started with lowball).
These $URGN results are unequivocally positive. . I am quite bullish on the stock at these levels. A few notes from the call today:
LG - NMIBC market does look > 10x the size of Jelmyto. With this scale a higher penetration is possible because even
In a year of depressing biotech days today is one of the worst IMO. Transformational data barely bought ($AFMD). AACR otherwise all losers. Good data / rebound companies getting sold off ($FULC, $CLDX). Market seems totally broken. $XBI $NBI
No position for me in $TPST unfortunately, but encouraging for the sector. Lots of other companies could be multibaggers on data. I hope this makes companies think twice about the recent toxic and unethical tendency to finance before the market can react to material news. $xbi
This is going to cast a dark cloud over CAR-T for immunology unfortunately. Has to be very safe for indications outside of oncology. $CABA already down >30%.
FDA is investigating reports of T-cell malignancies, including chimeric antigen receptor (CAR) positive lymphoma, in patients who received treatment with BCMA- or CD19-directed autologous CAR T-cell immunotherapies.
Sad to me that as a society we value a company like $INZY at < cash. High infant mortality with ENPP1. Treatments desperately needed. With ABCC6 could be billion dollar drug. The bias toward hypercompetitive megablockbuster indications is getting a bit ridiculous.
So far today we have good data from $IMVT, $SLNO, $PLRX, $INZY and a surprising buyout of $ICPT at a 90% premium. If $XBI doesn't go up today I'm not sure what can move it.
Today $INFI showed positive results showing 85% increase in response rates and 57% vs 14% DCR in PD-L1 low patients with urothelial cancer. As this is a controlled study, I believe this demonstrates that the drug is active in this PD-L1 negative population. 2/10
These wall-cross pipes are getting absurd. Funds must think the $KURA data must be quite good to give them a 30% premium after the recent run-up. Lots of funds must have seen the data, so leaks are very possible. Meanwhile holders won't even see the data until next week? At what
Biotech idea of the day; $HELP! How do we come out of this madness! Here is a thread on what I think could happen to the biotech sector. It’s more of a wish list, because if these things don’t happen the sector will continue to be toxic. $XBI $IBB 1/14
@bradloncar
In 2021 many disappointments but the following were wow moments for me. New modalities in this list that I'm surprised aren't getting more attention.
Paxlovid
$NTLA
$CLDX CDX-0159
$TVTX IGAN
$PDSB triple combo
$LIFE Atyr1923
$PRQR Ushers syndrome
$MRUS MCLA-158
$XENE Xen1101
$ocul a good case study in long term biotech investing. Weekend update was good but I think the last readout was more derisking and fundamentally value generating than this one, even though the stock dropped. Sometimes the market is just plain wrong for no good reason.
Congrats to $CCXI holders. Ceo Tom Schall and mgmt team deserve a lot of credit here. Navigated the company through a very tough FDA and negotiated a strong premium in one of the worst biotech markets.
Biotech Idea of the day: $ICVX. Icosavax is a low-profile vaccine player that has the potential for attractive long term appreciation due to its strong strategic positioning and the scarcity value of its hMPV vaccine.
Biotech idea of the day: $LOOKOUT! No, that’s not a ticker. Today I’m not going to talk about a specific company, but rather an often underappreciated threat to biotech businesses. $XBI, $IBB 1/9
Congrats to $gbt holders. The results look quite good. PRO outcome is a bit strange but probably because of high HU use and low symptom scores. The fact that they are talking about accelerated approval is the strongest testament to the excellent results IMO.
$LPTX, $BLU and $LIFE all with data that should increase POS in very large $ indications, and collectively net valuation increase today across all three is about $20M. Meanwhile, billion dollar IPOs with only preclinical data lumber on. That's biotech for you.
$PDSB being red today is pretty ridiculous. For anyone following the company closely, today's news has positive read-through implications for the Keytruda combo study, the platform overall, and potential partnering opportunities. 🤷♂️
Congratulations to my brother Karl and his co-founders Josh, Jon and Carter for selling Limbus AI today! Limbus is a company that develops AI-based automatic contouring solutions for radiation therapy.
For those that follow $PDSB some nice nuggets in HCW presentation: 1) The 3/8 ORR efficacy threshold for the NCI trial expansion was met BEFORE they enrolled all 8 patients, so early ORR is at least 42%. 1/2
Some thoughts on $INZY's ABCC6 data. It's fairly noisy. For example, cIMT reduction mostly driven by one patient. Fairly nice broad consistency across cIMT, Choroidal measurements and BCVA though. The PRO results don't mean much to me. Was initially concerned about ADAs but
Have been buying more $AVRO since the valuation is bizarrely low to me and strat alternatives could be wrapping up soon. MC < $60 M and MRQ cash $105 M. Last quarter was "kitchen sink" to pay separation charges and terminate licenses so burn will be very low in Q4. Also let go
Blows my mind that anyone still listens to Favus. From what I have seen, his arguments are always facile. Remember this is the guy who bet against $SRPT, $RLYP and also $gbt below $20. Just picked up a few more $GBT here.
This is ludicrous. Can you imagine having only 5 years on the market to generate ROI of a drug that costs >$1B to develop (and >90% of drugs fail)? Virtually all drug development would essentially stop.
Total drugs negotiated would reach 300 by 2033 instead of current 140, according to CMS.
The timeline is a big change: small molecules now get 7 years on the market before they're eligible to be picked for negotiation, biologics 11. Both would be shortened to 5.
Congrats to $STML holders. Despite today's 150% premium I think Menarini got a good deal considering expansion opportunities in CMML, XPO inhibitor etc... Bodes well for
#biotech
. Lots of other diamonds in the rough out there $xbi $ibb
What is the significant of this EAP? Well, since you asked (no one asked), at the very least it speaks to the unmet need in Sarcoidosis which I think is being mostly overlooked by the market, because these EAPs are intended to be for a “serious or life-threatening condition”.
Congrats to $GBT holders. People still don't understand Vox potential IMO. AA is being granted because based on the totality of the data and MOA they believe Vox is halting polymerization and thus arresting disease progression. Should eventually improve all downstream comps.
My early read on $LRMR data is that dose response looks highly likely, despite skin and buccal cell measurement variability. Non-quantifiable results muddy the waters a bit but even if you assign the most bearish outcome to missing data hard to see how that changes dose-response
@alexriesart
Adrian Tchaikovsky’s final architecture series seems to meet that criteria. Some similarities to Hamilton’s style. Also really like Tchaikovsky’s expert systems novellas.
FWIW a number of $PTGX insider buys today:
CEO 30,000 shares at $5.55 / $5.65
EVP Clinical 30,000 shares at $5.13
CFO 1,175 shares at $5.09 / $5.1
Director 8,000 at $5.7
Everyone get your picks in for
#biopick2024
! With the sector resurgence 2024 should be a lively year. Test your mettle at finding those multibaggers!
I also enjoy participating in this long-running biotech contest, which is more about portfolio
#Biopick2024
is now open.
Rules:
1. Only 1 pick (+1 alternate if you feel your 1st pick might not meet criteria)
2. Must be >$1.0 by eoy 2023
3. Biotechs only (*medical devices allowed)
4. Deadline 31-Dec
5. Must be listed on US exchange
Click to enter:
30% average return is exceptional, and I would argue more important than win rate. And not a small n there. In my experience Jonathan is also completely transparent, open to dissent, and a class act.
Everyone on
#biotwitter
should be following him
#ff
Not sure how accurate these ratings are, but still far short of my goal of 70% win rate
No idea what a reasonable objective for avg return is either, other than trying to keep losses minimal, cutting them short and let winners run on multi-year timeframe
Onward and upward👍
The year is 2040.
99% of the population is enslaved to machine mind overlords.
Covid 19 Variant Delta Plus Epsilon Gamma Double Zeta Plus continues to ravage the unvaccinated population.
And Novavax still hasn’t filed for FDA approval.
As far as insider buys go, this $LIFE one is pretty significant, not just is size, but because it's Paul Schimmel, founder or co-founding director of $ALNY, $ALKS, Cubist, $RGEN.
I have accumulated a fairly sizable $AVRO position. They have a $40M MC, ~130 M cash (my Q2 end estimate), ~$15 M debt. Some possible upside scenarios:
Biotech idea of the day: Cabaletta Bio. I haven’t seen the story comprehensively articulated on twitter so I thought I’d offer an overview. In short, $CABA is on the vanguard of new cell therapy modalities that could offer a curative benefit for some immunological disorders. $XBI
There is a fair amount of preclinical and clinical data showing $PDSB is a strong driver of the triplet's efficacy in HPV16+, but the fact that 0/7 non-HPV16+ patients had no tumor reduction is even more telling (PDS0101 is the most targeted element).
One of the more remarkable things during the $XBI bear market has been the lack of BP licensing deals. This is when BP should be taking advantage of company financing needs to position themselves to gain access to promising therapies. Yes, there's a lot of garbage out there, but
A couple things to be mindful of when looking at today's $GBT data. 1) Most pts are less SCD symptomatic than Part A pts will be to begin with. 2) 88% of these patients are on HU; It's hard to improve symptom scores of pain / fatigue when on chronic therapy with a chemo drug.
Biotech idea thread of the day; $MIRM. This stock is a recent IPO that has been underfollowed. Today’s surprising news is a dramatic transformation that the market may be catching up to for some time to come. $XBI
Just finished The Queen’s Gambit on Netflix. Wow. You don’t have to like chess to appreciate this show. The characters are unique and the performances exemplary. The game scenes are very cleverly done. There is a newness and originality to it as well. Superb.
Imagine if $PDSB announced that their Covid vaccine is back on track to begin trials next week in Brazil? Given T-cell focus might be a better approach than other existing vaccines for nu. I know, not likely, but one can dream...
Today's $GBT food for thought, from NEJM HU study: "A difference in the frequency of painful crises between the hydroxyurea and placebo groups began to emerge within about two months of the initiation of treatment and was clearly evident at four months." Part A is 3 mths long.
Buying a lot of $TARS this morning. I often worry about the 2nd quarter in new launches because first quarter can be a false signal caused by a bolus of approval demand or stocking issues. This is why for me TARS beat on 2Q is a very strong sign. Selloff unwarranted IMO.
Rather than write dozens of tweets, in $CABA's case I decided to write up the details on Medium (link below). I post these to test my thesis and look for feedback, so I welcome comments or critiques posted here on twitter or via DM.
Congrats to any $FPRX holders. Interesting that the $FPRX p2 data was imperfect in a number of ways. I'm biased of course, but I see it as the same caliber of dataset as $INFI P2, (when considering strong TNBC data), and yet $INFI is a puny fraction of the valuation of $FRPX.
With the clinical hold lift part of my thesis has played out on $BLCM but at $8-9 it is still quite cheap and partnering could be quite lucrative given recent solid tumor CAR-T glimpse and cell therapy switch capabilities. Holding most of my position.
Amazing that $INSM hit right on their clinically meaningfulness target of 20%. I'm sure some will question that now. Looks like lack of dose response in P2 is because doses approximately equivalent, which was one of the most likely explanations for Willow. But TEAESI does not
I have built a mid-sized position in $MEIP. 1) PI3Kδ inhibitor possibly best in class by large margin - 15k pt opportunity. 2) Early clinical data excellent 3) NEA / Perceptive just invested. 4) Nice pipeline of other drugs including 1 with BTD. 5) ~$100M in cash / ~$150 M EV
Biotech thread of the day; $ZYNE. This one is a short term trade going into a high risk Fragile X Pivotal readout (late June). I might not hold the stock into the binary, but I think it’s currently undervalued. 1/10
Small U.S. study suggests T cells, a key player in the immune response to coronavirus, are still able to recognize the Brazilian, South African, and UK variants
Biotech Idea of the day: $ICVX. Icosavax is a low-profile vaccine player that has the potential for attractive long term appreciation due to its strong strategic positioning and the scarcity value of its hMPV vaccine.
I just tried reading the $AVRO / Tectonic S-4 background of the merger. If you ever wonder how these deals happen, they ran quite a process. They had so many targets they had to use three letter designations. Here is just a 3 day snippet.
Thought I would highlight $PTGX for one of my episodic
#biotech
threads. Bought a fairly sizable position last week. They have three early stage (P1/P2) products, one partnered to J&J. Seems like it is not well followed and way oversold. 1/9
Not sure what $FIXX was thinking, to merge with a company that has a "returned rights" Phase 2 alopecia and AD program? Look around! That's hot garbage in this market. Nobody will give this a positive NPV. Still some value in CVRs here but wow read the room guys.
There is no way in which this will help anyone; disruptive to western mfgs, less developed countries won't be able to develop the capabilities anytime soon, will massively curtail future innovation and will disincentivize pharma from saving the world.
Great that $XBI has been up the last couple of days, but I have to say I am discouraged by:
$CRSP pipeline
$GLPG deal
$NVS $DTIL deal.
Themes: hyper competitive, undifferentiated, risky, high cost, targeting the same patients.
Can't we do something different?
$GBT all evidence points to sign. red. in polymerization, and potentially safer / more potent that HU. Should be the SOC to prevent progression of disease IMO. Valuation is silly here. If $TSRO can go for $5.1B, what can a comp with >>$2B peak rev pot. with ltd competition fetch?
@buysidebio
Numbers are good but for context they have a rebate program that drives sales in last month of every quarter. So March comparators should be Dec and Sep.
Bought quite a bit of $CABA today. I see this as a huge overreaction, and a possible fund liquidation event. From a translational standpoint today's clinical efficacy noise was what the market should have expected at these dose levels.
This is a special situation, and we don’t know the details of the basis for the EAP, but it’s strange that the stock gained < $5M EV when this development should be incrementally bullish on the safety profile, incrementally bullish on unmet need / demand, and incrementally
I strongly disagree w latest
@AppleHelix
bear thesis on $GBT AA. The fact that 64% were on HU shows that the benefit is additive. Vox has to be "reasonably likely" to predict clinical benefit. I think it is highly likely, and unlike other Hb modifiers, without safety risk.
For anyone wanting a distraction I'm offering my short story TRANSITION for free for a limited time. It’s a 60-90 min kindle read, dealing with themes of family, personal isolation and what it means to be human.
#COVID19
#scifi
#sciencefiction
@bradloncar
This doesn't list all the programs but reviews a lot of the different modalities and has citations of other reviews. Probably not the nice summary you're looking for but possibly a place to start if nothing better shows up.
I know $CRBP is behind $BCYC and could have a long road to market but is there a bear case on today's data cut? I am particularly intrigued by the lack of any cases of peripheral neuropathy or skin reactions. Is it possible they could be differentiated enough to beat $BCYC and
@bradloncar
I just don't see how waiving IP rights helps anyone. Tech transfer takes many many months. All this does is throw everything into disarray and disincentives pharma
The fact that $LIFE receives a $10M milestone (~1/6 of MC) from Kyorin for P3 initiation in a small geography like Japan, from a pact that was forged well before excellent POC data, is telling on how ridiculously undervalued the company is.
Biotech idea of the day; $CALT. This is a Swedish company that has recently IPO’d on Nasdaq. MC of ~$700M. Their lead drug is called Nefecon, and it is addressing IgA Nephropathy (IgAN), a large orphan indication with a poor prognosis (~130K pts US, 200k EU) 1/12