Reddit Posts
NTLA Deep Dive: Market Mispricing After Flawless Phase 3 CRISPR Data Published in NEJM Today - 35-40% short interest
NTLA Deep Dive: Why Today’s Flawless Phase 3 CRISPR Data in NEJM Creates a Massive Valuation Disconnect
NTLA Deep Dive: Why Today’s Flawless Phase 3 CRISPR Data in NEJM Changes Everything (And Why the 39% Short Interest is Mispriced
NTLA beginning to gain serious traction
In vivo CRISPR and potentially major catalysts ahead for NTLA
KalVista (KALV): FDA Approval Secured, $22M Non-Dilutive Cash, Commercial Launch Underway
$KALV ($15.06) KalVista FDA breakout. First oral HAE drug + bullish options setup
[KALV] FDA APPROVED 🚀 | First-in-class oral HAE drug is here — buckle up
KALV Announces FDA Approval for EKTERLY (sebetralstat) - First and Only Oral On-Demand Treatment for Hereditary Angioedema
Kalvista Pharmaceuticals. Short squeeze potential but otherwise a good buy. Shorts can’t cover. Not enough volume.
$NTLA - The New FDA Commissioner Wants Cures, Intellia Can Deliver.
BioSig (BSGM) Grows in Arrhythmia Care With Subscription Model
Good company great product strong financials
Shorting Opportunities - Companies that Have Yet to Feel JPOWs Wrath
$BCRX DD, the perfect entry point 🌝 🚀
some times markets just arent intressting long investments
$BCRX Fundamentally sound with the potential for a short squeeze. Join me fellow apes.
Main Reasons why I would INVEST in $BCRX before Q2 ER
What happened to @HAE and do you think they will recover?
Wanna get retarded with me? Grab $HAE while it’s still on sale.
Stocks that saw a major correction and that may be worth picking up in the next few weeks
Revival of a company Wall St. believed to be a zombie - Cathies' Takeda about to get burned? - BCRX investor and activist investor dreams becoming realized!
Mentions
I agree on all your key negative points,but calling this weekend's EAACI and New England Journal of Medicine data stale news from April misses how institutional analysts and healthcare payers evaluate biotech. There is a massive disconnect between the April topline press release and the actual peer-reviewed publication that dropped this weekend. In April, the market only knew the headline eighty-seven percent overall attack reduction. The newly released data revealed a ninety-one percent reduction in moderate to severe attacks, alongside an eighty-nine percent reduction in attacks requiring emergency on-demand treatment. In the insurance landscape, this is the holy grail because it proves to payers that once a patient gets this one-time infusion, insurers almost completely stop paying for ongoing rescue medications, completely changing the cost-benefit math for commercialization. Furthermore, moving from a corporate press release to an independently verified publication in the most prestigious medical journal in the world means everything for institutional research analyst teams who use these papers to adjust their clinical probability of success models. Regarding the nex-z platform risk, it is completely fair to argue that the previous patient death prevents this from being a fully de-risked platform story, and that risk is exactly why the stock trades at a 1.7 billion dollar market cap instead of 5 billion. But it is also crucial to remember why the FDA lifted those clinical holds earlier this year, as the toxicity was deemed manageable via protocol adjustments and heavily tied to that specific patient's comorbidities. With a clear regulatory path to a first half 2027 launch for HAE and a newly fortified safety profile, the current valuation gap remains wild, regardless of how old the initial headlines are.
I agree on all your key negative points,but calling this weekend's EAACI and New England Journal of Medicine data stale news from April misses how institutional analysts and healthcare payers evaluate biotech. There is a massive disconnect between the April topline press release and the actual peer-reviewed publication that dropped this weekend. In April, the market only knew the headline eighty-seven percent overall attack reduction. The newly released data revealed a ninety-one percent reduction in moderate to severe attacks, alongside an eighty-nine percent reduction in attacks requiring emergency on-demand treatment. In the insurance landscape, this is the holy grail because it proves to payers that once a patient gets this one-time infusion, insurers almost completely stop paying for ongoing rescue medications, completely changing the cost-benefit math for commercialization. Furthermore, moving from a corporate press release to an independently verified publication in the most prestigious medical journal in the world means everything for institutional research analyst teams who use these papers to adjust their clinical probability of success models. Regarding the nex-z platform risk, it is completely fair to argue that the previous patient death prevents this from being a fully de-risked platform story, and that risk is exactly why the stock trades at a 1.7 billion dollar market cap instead of 5 billion. But it is also crucial to remember why the FDA lifted those clinical holds earlier this year, as the toxicity was deemed manageable via protocol adjustments and heavily tied to that specific patient's comorbidities. With a clear regulatory path to a first half 2027 launch for HAE and a newly fortified safety profile, the current valuation gap remains wild, regardless of how old the initial headlines are.
I have an agentic debate for vibe investing: NTLA (Intellia): The Bull Case Has a Hole You Should Know About The Setup A bull thesis is circulating: Intellia crushed HAE Phase 3 data (87% attack reduction, super clean safety), stock got beat down on dilution, now shorts are trapped. Squeeze incoming. The thesis is partially real. The clinical data is solid. But it’s missing the single most important fact of the last 8 months, and that changes everything. What the Bull Case Got Right ✅ HAE data is genuinely good: 87% reduction in attack rate, 62% of patients attack-free for 6+ months, zero serious adverse events in this trial. ✅ Regulatory path is real: Rolling BLA submission underway, targeting H1 2027 launch. One-time IV infusion beats lifelong twice-weekly prophylaxis. Commercial moat exists. ✅ Balance sheet is fine: \~$670M cash (Q3’25) + $180M raise in May = runway into mid-2027, maybe later. No dilution death spiral coming. ✅ Shorts do exist: \~34% of float short. A pop could trigger covering. What the Bull Case Left Out 🚨 A patient died from liver toxicity on the same platform in October 2025. The facts: • nex-z (Intellia’s ATTR therapy, same LNP/Cas9 platform as HAE lonvo-z) dosed a patient Sept 30, 2025 • That patient developed Grade 4 liver transaminase elevations and bilirubin spike • Patient was hospitalized → FDA placed clinical holds on both MAGNITUDE trials (Oct 29) → patient died Nov 5 • Holds were lifted in Jan-Mar 2026 after the company promised enhanced liver monitoring This death sits on the exact same delivery platform that the bull thesis claims is “validated” by the HAE data. That’s the wrong read-across. The Real Situation HAE lonvo-z: De-risked. Clean data. Realistic 2027 launch. This is the actual asset. ATTR nex-z: Alive but wounded. Was supposed to be the platform’s exponential call option. Now it’s a “will the liver toxicity recur” binary. The holds being lifted doesn’t erase that you watched an 80-year-old die from liver injury on this exact mechanism. Squeeze narrative: Stale. The 87% number broke in May; June was just conference detail. Clinical catalysts for NTLA average a -0.44% move. Don’t expect shorts to panic into covering on old news. The Grid | |Bull |Bear | |-------------------|---------------|---------------------------------| |\*\*HAE data\*\* |✅ Real & good |✅ Agree | |\*\*Regulatory path\*\*|✅ Rolling BLA |⚠️ Binary on FDA response to nex-z| |\*\*Platform safety\*\*|❌ Ignores death|🔴 Death on same mechanism is huge| |\*\*Squeeze setup\*\* |Shorts exist |Old catalyst, poor base rates | |\*\*nex-z upside\*\* |Free call |Survivability question now | Bottom Line If you love the HAE asset: Fair entry argument exists. One-time therapy, orphan disease, runway secured. If you came for the squeeze: The catalyst is a month old, clinical newsflow for NTLA historically doesn’t move the stock, and shorts aren’t forced into anything yet. What’s actually priced in: A company with one solid near-term asset (HAE) and one scarred pipeline asset (nex-z) that needs to prove liver toxicity was patient-specific, not mechanism-linked. That’s not “de-risked platform validated.” That’s “one leg works, other leg is on crutches.” Trade accordingly. Don’t let a clean HAE readout blind you to the fact that this platform killed someone 7 months ago. TL;DR: HAE data is real. Regulatory path is real. But the bull thesis erased a treatment-related patient death on the same delivery platform, which is… not ideal. This is a one-asset story now, not a platform story.
Your observation about the June 4th price action is right. If new shorts piled in at the $14 level, a major gap-up on Monday is going to put them under immediate pressure, even if the overall squeeze parameters look calm on paper.The competition from existing mAbs and RNA therapies is real, and market penetration will be a steady climb. However, the cost economics are what most shorts completely miss. HAE is one of the most expensive chronic conditions in the world. takzyhro/ Orladeyo cost insurers roughly 500 k per Patient every year. Over 10 years, a single well-controlled patient costs health insurance $5M+. Intellia is likely to cost less than this. But the true macro comed from Platform validation. Proving that the in-vivo delivery mechanism is safe and durable in a Phase 3 trial completely changes the risk profile for NTLA's entire pipeline (like ATTR).
NTLA will announce they have the HAE cure and the first in-vivo treatment which is actually commercially viable unlike the CRSP ex-vivo cure. If data is good, they will go from market cap of 1.6B back to 10B in the next year since the HAE market is substantial.
The $160 peak was fundamentally driven by excitement around NTLA-2001 (ATTR) and NTLA-2002 (HAE), representing the potential of CRISPR to deliver curative therapies. However, dilution from significant capital raises, inherent clinical/execution risks, market volatility, and a reassessment of valuation expectations have prevented the stock from sustaining that level. Future gains depend on successful clinical milestones, regulatory approvals, and commercial execution, but the path back to $160 is steep due to the factors above.
If both Intellia's CRISPR-Cas trials demonstrate durable efficacy and safety, the company could see its valuation increase 3–10x from current levels, driven by access to a combined $20+ billion market and validation of its in vivo gene editing platform. The ATTR-CM program (nex-z) likely carries the greater valuation weight given the larger market size and partnership with Regeneron, while the HAE program (lonvo-z) provides diversification and proof-of-concept for the KLKB1 target. Not point to sell very soon, like coming week.😁
Anyone taking about NTLA here? Great WSB play. Trading at $26 in fall based on expectations of approval of HAE gene therapy treatment, had a patient die in phase 3 and an FDA hold. Stock dropped to $8. Turns out patient died of unrelated surgical complications. Stock now surging based on expectations of lifting FDA hold. Back up to $13.50 now and very likely headed back to $20s. Also, with the death, there's a 35-40% short float getting...dare I say it...squeezed!
Yes, ACOG trading $6-7 vs $18 - 21 target. KALV's first-in-class oral HAE with 10-year exclusivity, trading $12-14 vs $28-36 targets, both prove the trailing revenue point.
Exact thing with KALV. They launched their HAE drug in July, just crushed Q3 earnings with $13.7M revenue solid uptake metrics, Germany launch coming Q4, and analysts have $28-36 targets. Stock's at $14. Market's totally ignoring the forward trajectory and just anchored to that one quarter. It's wild because the commercial ramp is obvious - growing scripts, payer coverage expanding, international rollout starting, and they just proved the revenue model works, seems like a real inefficiency in how early commercial biotechs get priced. Check out r/KALV_Stock for DD and discussion if interested.
Just play Q3 earnings. I played OSS and SD and HAE, although I put way less in HAE because stupid AI pushed me to SD more. Look at industries doing well, energy is a great earnings to bet on. I bet on DK today and they blew earnings out of the water.
That’s two weeks ago, around the time intellia announced HAE results which already made the stocks jump. Today’s jump is so large that it’s clearly not just momentum.
I like NTLA as both a short and long term play. Excited about their HAE results in January.
I know the profit would be smaller. But at least there would be some profit, or at least stability. Here are some of my losses, to show it is not only all green. The main point is stability of the portfolio — almost never going down in a big bang crash like with experimental stocks. A few of my current red positions: AVGO: –1,481 WM: –1,252 AMD: –1,009 PAYX: –921 TTD: –841 UPST: –525 HAE: –465 TGT: –468 SMCI: –285 The last up wave was only in a small tech related corner. Like I said, I did not put everything only in M7. I made diversification, so I feel safe. My real risk is if the whole economy collapses, not if 50 shares will run down the hill from 0.99 to 0.01. My portfolio is on margin. Total value: $698,500.
Convinced HAE is being manipulated
What's HAE? Hereditary angioedema? If so, isn't that a rare disorder (1 in 50,000)? Are they really going to be making a lot of money with that drug that would be used by so few people?
KalVista (KALV) is on the edge of something big with sebetralstat—the first oral, on-demand treatment for HAE. No injections, just a fast-acting pill taken at the start of an attack. In trials, it consistently stopped progression in 20 minutes and delivered full relief in ~1.3 hours—even for severe and mucosal attacks. It works across all patient types, even those on long-term prophylaxis. Real-world data shows over half of HAE patients still struggle with breakthrough attacks, and injectables delay treatment. The FDA missed the June 17 PDUFA due to internal delays—not safety or efficacy concerns. A final decision is expected by mid-July. Analysts are bullish, with targets from $19 to $39 (vs. ~$12 now). KalVista has global filings in progress and a Japan deal in place. With strong data, low risk, and a clear market need, KALV is set up for a major move.
Why are you holding gene editing trash like NTLA? Get out of it. Quick biotech lesson - gene therapy or editing are only commercially viable for diseases that are terrible (like a death sentence, particularly for children) and do not have viable alternative therapies that are pretty efficacious already that are generally safe. The two main indications- HAE (busting out in hives…multiple drugs that generally work or will work) and ATTR (lower mortality but PFE/ BBIO/ALNY all have drugs that you would take instead) are not commercially appealing, hence why the stock keeps bleeding down.
# **TLDR** --- **Ticker:** $KALV **Direction:** Up **Prognosis:** Buy **FDA Approval Date:** June 17, 2025 **Catalyst:** Potential for significant upside if Sebetralstat (oral HAE treatment) gets FDA approval. Analysts have a buy rating with target prices suggesting 2-4x potential increase. **Risk:** Pre-revenue, so heavily reliant on FDA approval. Potential for market to remain bearish even with approval. **Bonus Meme:** This is like buying a lottery ticket, but instead of a number, it's a drug that could cure a rare disease.
Gene therapies are priced in millions of dollars, so even for rare genetic diseases such as HAE, AATD, SCD (diseases currently being targeted by gene editing companies) there is the potential to make billions in revenue. Also, the patient population will still grow as new humans are born with the disease since these therapeutic edits are somatic and not heritable. The first CRISPR based drug (Casgevy) was approved at the end of last year for sickle cell and is priced at $2.2M, and the manufacturer (Vertex) anticipates they can dose 35k patients across US and Europe.
Could be me but HAE is a very rare disease. Medical centers do have meds for HAE but this is not one of them unless for some reason RUCONEST is significantly cheaper and/or more efficacious than its competitors.
Biocryst Pharmaceuticals Inc Current valuation overly discounts Orladeyo; Upgrade to Buy Rating Change: BUY | PO: 10.00 USD | Price: 7.19 USD Recent stock pull-back offers attractive buying oppy Biocryst Pharmaceuticals (BCRX) is commercial stage company focused on developing oral medicines for rare diseases. BCRX’s lead asset Orladeyo is approved as a prophylactic (preventative) treatment for hereditary angioedema (HAE), which is a rare genetic disease characterized by rapid, uncontrolled swelling of body tissues. Current HAE treatment is classified as either acute or prophylactic with several approved treatment options on the market. In 2020, Orladeyo entered this market as the first and only oral prophylactic therapy for HAE. We note recent pullback in shares (based on no fundamental change) now presents meaningful upside potential to our $10 PO. We model $78mn in 2Q sales and model $320mn in FY23 sales, which we think is achievable. BCRX guidance is for sales of at least $320mn in 2023. Given our view that our $629mn peak sales estimate does not require outsized future growth, we raise our rating for BCRX shares to Buy from Neutral. Mgmt anticipates $1bn in peak Orladeyo sales We estimate Orladeyo 2Q sales of $78mn. Mgmt continues to highlight the strong launch, reporting >1k pts on therapy. As such, we continue to view the Orladeyo launch positively though we note mgmt’s comments about peak sales of $1bn still need additional validation from sales trends in the coming years. We also think achievement of such sales still needs additional physician support (KOL feedback noting pt hesitancy on switching from current prophylaxis injectables), 60% first-year retention rate for Orla’ and increased competition as newer therapies could enter the space (e.g. PHVS, NTLA). ‘10013 clinical program delay remains ongoing On pipeline, BCX10013 (an oral complement inhibitor) remains on clinical delay due to dose-related nonclinical observations with preclinical dose-related toxicology studies on- track to complete by YE. On the 2Q earnings call, we look for updates on the extent of the clinical delay as well as company guidance on next steps for the program. Changes to our DCF-based model We roll the qtr and adjust op-ex to be in-line with trend. We note Orladeyo is the sole driver to our valuation contributing $12/sh (offset by negative NPV for pipeline spend). Risks to our thesis are higher than expected dropout rate and stronger competition. 13 July 2023 Equity Key Changes ( US$) Tazeen Ahmad Research Analyst
They also have 200million cash and asset on balance sheet. CEO salary is only paid less than 800k, super legit biotech company. Makes capsule to treat HAE instead of traditional IV treatment.
MODS HAE SAID ALL FLAIRLESS WILL BE BANNED. don't @me
True. Observe Estonian "Harju Elekter" (HAE1T.TL) Dividend payer! They own part of [SKELETON ](https://www.skeletontech.com/) Only way to buy "skeleton " growing and biggest super-conductor builder. Now started the supper-battery project (with "Shell") If someone is in to it for next 10y.
GSAT and HAE, both have been in strong uptrends all year. I got these screening for historically low IV and gains. What do you think about shorting these? I'm still learning so here to make conversation, really.
BCRX!! But lemme explain(just a little), unlike everyone else here. Prior to 2020 basically we’re doing about 15M in sales, had their drug Orladeyo approved for HAE, and expect to do no less than $250M in revenue. Management (and myself) expects it to grow to $1B+ in sales per year. Massive growth. Phase 3 factor D treatment to treat PNH going right now. It was paused by the FDA, but the dose was lowered and thus far is still continuing. The phase one and safety data is exceptional, and the treatment is a pill instead of IV, and many have to get blood transfusions multiple times a week. That’s not necessary with our drug. It may be a ways out from approval (I’m thinking 2024 most likely), but that’s going to be 10 Orladeyos in one drug. This treatment can also be parlayed in to treating about 8 other rare diseases, so you can see the upside of the company which is currently valued at just $2.25B, trading around $13/share currently.
Tech will not give you a 2X. The sector that led to the correction does not lead out of the correction. You need to look elsewhere. We are in/entering a normal, cyclical recession. What does well out of a recession? Industrials, energy, financials. Maybe you can find some tech related to those fields like PYPL (financial). Medical devices have been quite weak because of COVID. Lots of quality names there that have tread water, small and large cap: BAX, HAE, SIBN. Another place to look is broken SPACs. SPACs were a major bubble that has burst spectacularly, leaving micro cap stocks trading at or below cash. Most of them are real businesses: SHCR, SHPW, ETWO. The next bull market will NOT look like the last one. Expand your knowledge beyond tech now.
HAE, poors selling plasma at record numbers 
HAE having a huge quarter Aug 10 earnings
Poors selling Plasma like crazy. HAE earnings will boom.
Finally back in the green on HAE
As with anything on the internet at all, do your own research. Remember, I’m just as stupid as you, not a financial analyst. I highly recommend the stock BCRX.great pipeline, huge potential, and still a great price. It’s up ~275% already I’ve Ethel year after their drug got approval. Mind you they have the only once daily oral medication for a rare disorder known as HAE. Europe, Japan, Britain, and the US as well as a multitude of other countries have already approved their drug Orladeyo. There is currently no meaningful competition in the treatment of moderate HRE, because all other treatments involve needles. If you’re diabetic, you know that sucks balls. Next earnings will crush in my opinion. Do your research, but BCRX has a beautiful future ahead
BCRX is 90% of my portfolio. Great drug pipeline, FDA approved drug to treat HAE that is the only oral treatment. Factor d inhibitor that can treat up to 10 different indications in phase 3 pivotal trial for 1 indication currently (pnh) and starting proof of concept trials for other indications 2H21. Great management team, enough cash to keep the company and trials going until ‘23 without diluting shares. This company is going to be a monster if they grow organically but they are also a good candidate for a buyout.
HAE been doing surprisingly well for me lately. Up 9% in the last 10 days.
HAE is straight up carrying my portfolio today. Everything else is red.
I think without any question that there are going to be a lot of patients with HAE jumping to get Orladeyo and revenue will be higher than the market expects. If positive enough, would not be surprised at all to see BCRX double or more. On the other hand no idea how production is moving along nor how much awareness there is of this yet, so uptake might be a little slower and we may not see the strong positive movement till later this year or next. Kind of the problem with timing this. Shares, I believe, will be solid and have a decent chance within a year or two of making a 5-10X return. Only two concerns are, one, what if something goes wrong with the drug and it gets pulled and two, some concerns about competition (but I think that is a few years off and BCRX should be fine). I have a mix of shares and options as well (about $70K worth).
Too late, they're deleting the fuck outta comments. [Deletions](https://i.imgur.com/hOS8HAE.png)
Looked a little deeper into BCRX's drop today. There's sentiment that the drop is related to NTLA's rise, since NTLA's one of their key competitors to treat HAE. BCRX still has the only oral treatment available, and the thesis that BCRX's oral drug will continue to pump their earnings isn't really changed -- it's not like NTLA is going to develop a gene editing cure to HAE and bring it to market in the next year(s). I added more to my 12/17 14Cs during the dip today, and will probably add a bunch more at higher strike tomorrow as well. A good dip to buy if you agree with the BCRX thesis.
At the moment it’s a bio company burning through cash and not making a profit (never has). However it really feels like they have turned a corner. Orladeyo has the potential to lift the stock price to over $40 and making the company profitable and cash flow positive in 2022. They need to be able to convince patients to move from the daily needle to a daily pill. Seems simple right? But it will take some time. I’m all in on this company, 7000 shares and leaps so believe it will happen. However I think the OP has overstated the potential revenues from Orladeyo. The paying HAE market for licensed drugs in Europe is likely to be 6k and not the 12k quoted. Only the wealthiest countries can pay for it. Also $485k may be the price quoted but after rebates and other incentives the price will be lower. $450k is a more accurate number IMO.
That’s per year - so in Europe they will pay €200k however the OP has the total number wrong. There may be 12k HAE patients in Europe but only the wealthiest countries can afford to pay licensed drug fees. A more accurate number would be 6k (Germany, Italy, France etc.) Countries like Romania and Poland can’t afford €200k per year.
I very much doubt that OP is CEO. This is a $3 billion company that started a long time ago. They recently were approved by FDA, European equivalent, Japan equivalent, and are like to get more approvals soon for a drug that helps patients with HAE. That drug will take them from $3 billion to $7 billion. But it's their other drug you should be interested in as an investor...
My realistic year end price predictions over the next couple years: 2021: $30 - $40 2022: $60 - $80 (or buyout) 2023: $100 - $200 (or buyout) What are these valuations based upon: - Continued increasing quarterly sales and patient enrollment of Orladeyo for HAE, with possible +EPS quarters successively to come later this year. - Starting pivotal trials for BCX9930 (Factor-D) in 2H for PNH patients, resulting in blockbuster results in 2022. - FDA approval for use of Factor-D for PNH patients by early 2023. These estimates do not factor in other drugs in their pipeline. Those will be added to the valuation if/when they mature. These are just my personal price predictions and not investment advise. Please do your own DD to determine what investments meet your financial goals.
Picking up more HAE today. Lots of potential with things opening back up.
i hope you are aware insurance companies are flocking to orledayo since it is the cheapest HAE treatment option out right now. Don't hate the player hate the game
Mark my words. Every quarter Wall Street will try to make a conservative guess at how much revenue BCRX will make and we will crush those expectations every time. We did it in Q1 from US sales alone. Q2 will have USA, Japan, and EU sales. We haven’t even finished becoming easily accessible via insurance in USA. Each month we have new patients with reoccurring revenue from a growing list of once a day pill popping HAE patients. We are changing lives and making bank! Don’t be a retard and slap the ask if you haven’t already!
My realistic year end price predictions over the next couple years: 2021: $30 - $40 2022: $60 - $80 (or buyout) 2023: $100 - $200 (or buyout) What are these valuations based upon: - Continued increasing quarterly sales and patient enrollment of Orladeyo for HAE, with possible +EPS quarters successively to come later this year. - Starting pivotal trials for BCX9930 (Factor-D) in 2H for PNH patients, resulting in blockbuster results in 2022. - FDA approval for use of Factor-D for PNH patients by early 2023. These estimates do not factor in other drugs in their pipeline. Those will be added to the valuation if/when they mature. These are just my personal price predictions and not investment advise. Please do your own DD to determine what investments meet your financial goals.
i have a put on HAE and i want to beat up anyone who bought into it with this fugazzi 10% jump this morning!? wtf!!!!
Crazy the number of stocks in my watchlist at 52 week lows. BFLY, HAE, UBSFY, SEER. Just a bloodbath out there.
Nope! u/mr-nefarious Biocryst Pharma ($BCRX). in december we launched an oral drug for HAE, the only oral option for patients to be more precise. In q1 we recently smashed analyst expectations by tripling expected revenues and this terminal screenshot ([https://charts.stocktwits.com/production/original\_328617028.png\_](https://charts.stocktwits.com/production/original_328617028.png_)) shows no signs of us slowing down any time soon and we're set to beat next quarter (and probably the one after as well since our EU and UK approvals will be contributing to revenues by that point as well) expectations by similar margins. On top of all this, we're set to start pivotal trials for BCX9930 which is an oral factor d inhibitor straight from phase 1 because the p1 data is absolutely phenomenal. In the q1 earnings call and a conference earlier today we learned that the FDA has agreed that hemoglobin levels and transfusion rates will be the primary and secondary endpoints in the pivotal trials. when looking at our competitors, hemoglobin levels and transfusion rates is our strongest data ([https://charts.stocktwits.com/production/original\_329540329.png](https://charts.stocktwits.com/production/original_329540329.png)). They want this drug, and once again, we'll be the only oral option available. I can talk at great length about this company if i've at least gotten your interest but i don't want to bore you with everything up front haha. let me know if you have any questions or just want more detail in general.
I think BCRX is solid (as long as ORLADEYO does not get pulled at some point and a moderate percent of HAE patients pick it up). As to to their other drugs, I have no opinion, but on ORLADEYO alone I think the company could easily go up 3-6X from where they are now.
Should I put money into $HAE? It looks bull to me but I don’t see anyone talking about it
Just curious, i see AYX and HAE are at 52 week lows. What made you chose them?
## Numbers look good. Bought some. ## [Fundamental Analysis](https://snapshot.fidelity.com/fidresearch/snapshot/landing.jhtml#/fundamentalAnalysis?symbol=HAE) [MORE Fundamental Analysis](https://snapshot.fidelity.com/fidresearch/snapshot/landing.jhtml#/fundamentalAnalysis?symbol=HAE)PROVIDED BY [S&P GLOBAL MARKET INTELLIGENCE](https://research2.fidelity.com/fidelity/research/reports/release2/Research/StandardAndPoors.asp) AS OF 05/03/2021Analysis is driven by underlying factors specific to the Health Care sector. * Valuation current =**80** Overvalued= 0 Undervalued= 100 * Quality current =**94** Low= 0 High= 100 * Growth Stability current =**22** Low= 0 High= 100 * Financial Health current =**75** Less Healthy= 0 Healthy= 100
HAE IS Best Stock To Buy And Hold 🚀🚀🚀🚀
Going full retard on HAE calls 
Anyone in HAE? Market overreaction to losing a customer next yr? Low float, earnings and conference in May, saw unusual whale alert for 90 calls for May. Low volume on options but seems interesting
Regarding the leverage, in a nutshell you can [look at the amount of borrowing on margin](https://www.yardeni.com/pub/stmkteqmardebt.pdf), which is like taking out a loan to purchase stocks or derivatives like options. And like loans, sometimes things get hairy on the debtor's end and they get in trouble making good on their word to pay back the debt. In the post, leverage is referring to the liability of taking on margin debt to, for example, buy stocks. If you use margin to increase your exposure to risky assets and those assets become devalued in the market (as they tend to do from time to time), your portfolio's net value can decrease below a threshold that can trigger a **margin call**. In this situation, the lender basically tells the debtor to either add more cash/assets to the account, or the lender assumes authority of your positions and can liquidate them **as they see fit**. This is basically what happened to Archegos: They took on a lot of water in their exposures to certain risky assets/companies, and their authorities like Credit Suisse, Goldman, and Morgan Stanley stomped them out and sold all of their positions. The screenshot seems to be referring to the idea that there are more situations like this, wherein hedge funds are overly exposed to risk on borrowed money from banks. If this is true, and the right (or perhaps "wrong") parts of the market lose enough value such that leveraged funds begin to make lenders worry too much, perhaps that record-high amount of margin debt can become a big problem. It's key to understand that if, for instance, a hedge fund managing a few billion dollars gets margin called, **all of their positions are vulnerable to sale, indiscriminately**. So if that fund lost a bunch of money on, say, HAE and some shitty SPACs, but also held $1 billion in MSFT, there may very well be a huge block of MSFT entering the market all at once for sale. And that can cause more problems for other hedge funds and leveraged accounts that could, for instance, be borrowing on margin to hold $100,000 worth of MSFT.
Thoughts on Haemonetics $HAE making a quick comeback after the CSL Pact fall through?
Thoughts on Haemonetics $HAE making a quick comeback after the CSL Pact fall through?
Anyone loading up on HAE calls?
Well, shit. I stand corrected. $HAE drilled -36% yesterday. Please 🙏, let's see that bottom today so I can grab an 80c weekly.
$HAE got absolutely crushed yesterday when it was announced they lost a contract that makes up about 10% of their sales. Down 30%. I'm thinking calls once the bottom is finally hit, they're a solid company and have consistently beat earnings in the past. Mr market done freaked the fuck out and will be all apologetic.
Curious what thoughts are on $HAE ? Appears they lost their contract with CSL Behring which accounts for ~12% of their revenue. Stock dropped 36% today. Feels like oversold to me. NOTES: - Contract ends in 2022 (so this isn't an immediate impact) - After this announcement the company reaffirmed their guidance - Price cratered essentially at opening bell and did not recover all day. If it was significantly oversold, surprised it did not rebound by EOD/AH, though.
If 15% of HAE patients use Oraladeyo (which is pretty much a no-brainer unless the drug has issues) they hit the $500M mark.
Good afternoon everyone, what's your thoughts on HAE that has dropped like a rock.
Too much speculation on the current revenue of its drugs. Oraladeyo is for a very specific group of people (how many people have HAE?), has competition from Takeda and is very expensive. The rapivab isn't worth billions. Galidisivar and antivirals don't seem to be catching much traction. They can have as many great drugs as possible, until they have consistent sales, it shouldn't have a massive valuation.
HAE looking like a juicy steak right now
No mention of BCRX here today. Share price is up nicely on news that Japan approved their drug pricing. Comes to $681 per 150mg pill, which is taken daily. Basically, means in Japan their drug will sell for about $250K per year. No idea the exact nature of their distribution agreement, but assuming the drug is picked up by HAE patients should help ensure they become profitable. No idea what the stock price should be, but I think tons of upside. I have shares and calls. Mostly LEAPs.
Bro do a little reading before typing, it’s not a clinical stage Bio anymore, it’s commercial stage now. Q1 sales are the start of their HAE ramp up in US and Japan and EU coming Q2. Their clinical for BCX9930 hit it out of the park in Phase 1 and FDA approved to go straight to Phase 3 pivotal trial. No gambling here
IONS just came out with their HAE drug meeting the endpoints for phase 2 . However, I don’t think that we should be overly concerned, since it appears that their drug is designed to alleviate an oncoming attack, rather than regular maintenance therapy. Its also administered with injections and final approval is at least 1 year away. Your thoughts anybody?
There are approximately 12.000 people in the world who suffers from PNH . The expected revenues from the drug worldwide by 2025 is 5.7 billion. Getting into phase 3 study in 2h, positions BCRX perfectly to give Alexion and others a kick stage exit left. But that is just the start. At least 6 other deceases are going to benefit from the HAE study. Smart move of Stonehouse to adapt the poison pill. At least us who are long, have a chance to harvest the fruits of our investments.
They have stated that they have enough money to get them to 2023 which doesn't sound to bad to me by then their other drug Orladeyo should have a decent size of the HAE market and sustain the companies future endeavors. From what I've been able to dig up Sheridan had a scheduled automatic sale of his options that were executed, I assume that's the 80% you're referring to.
I'm a believer in the science and in the leadership of Stonehouse - I've been following this stock for roughly 6 months and feel all sorts of optimism. Like you said, it's currently at a great entry point. De risked with an oral HAE drug and a pipeline in a molecule with massive potential- what's not to like!? Buy outs are impossible to foresee , but this company is also a candidate to get swooped up at a very nice premium
I hear you, I’m just saying your clickbait title about TAK being burned is a bit too much. TAK has sold off entire product lines, trimmed down to a core focus of products, and sold off a lot of garbage after acquiring Shire. I just don’t think you can say TAK will be burned by any of this and I will look into this BCRX company out of curiosity. You should probably stick to promoting the company that your DD is about and not make borderline irrational speculations about HAE somehow impacting the bottom line of an absolute worldwide giant like TAK
Dude I do not give a fuck about Takeda. This company has taken many of it's prior HAE sales force away in favor of Orladeyo and that's a signal to any investor, even Cathie, would raise eyebrows @ that fact.
WELL DONE!!! HOLD THE FINE... WE TAKE THIS HUGE PUNCH.... BUT WE WILL GO UP!! this drop is ARTIFICIAL!!! THEY WANT YOU TO BELIEVE THAT THIS IS DONE.... BUT IS FAR FROM DONE!! "**To the extent aggregate short exposure exceeds the number of shares of our Class A Common Stock available for purchase on the open market"... THIS WAS IN THE REPORT..... IS NOT SO COMMON TO HAE THIS IN A REPORT.... THEY ARE FUCKED!! GOOD EARNINGS GOOD VIEW.... FUCK FUNDAMENTAL.... AFTER THE SQEEZE IN A 1 YEAR OR 2 THIS GONNA BE A 80-100B COMPANY!! FUCK THEM FUCK WS!!! I WANT MY TENDIES...... THE SHOW MUST AND WILL GO ON!!! DON T PANIC SELL!! LESS GO FELLLAS LOVE YOU ALL!!** ROAD TO 100K IS STILL LONG!!!! DON T PANIC SELL
this drop is ARTIFICIAL!!! THEY WANT YOU TO BELIEVE THAT THIS IS DONE.... BUT IS FAR FROM DONE!! "**To the extent aggregate short exposure exceeds the number of shares of our Class A Common Stock available for purchase on the open market"... THIS WAS IN THE REPORT..... IS NOT SO COMMON TO HAE THIS IN A REPORT.... THEY ARE FUCKED!! GOOD EARNINGS GOOD VIEW.... FUCK FUNDAMENTAL.... AFTER THE SQEEZE IN A 1 YEAR OR 2 THIS GONNA BE A 80-100B COMPANY!! FUCK THEM FUCK WS!!! I WANT MY TENDIES...... THE SHOW MUST AND WILL GO ON!!! DON T PANIC SELL!! LESS GO FELLLAS LOVE YOU ALL**
this drop is ARTIFICIAL!!! THEY WANT YOU TO BELIEVE THAT THIS IS DONE.... BUT IS FAR FROM DONE!! "**To the extent aggregate short exposure exceeds the number of shares of our Class A Common Stock available for purchase on the open market"... THIS WAS IN THE REPORT..... IS NOT SO COMMON TO HAE THIS IN A REPORT.... THEY ARE FUCKED!! GOOD EARNINGS GOOD VIEW.... FUCK FUNDAMENTAL.... AFTER THE SQEEZE IN A 1 YEAR OR 2 THIS GONNA BE A 80-100B COMPANY!! FUCK THEM FUCK WS!!! I WANT MY TENDIES**
Yeah, my price target was $40+. Of course if it is taken up by 50% plus HAE patients and they can sell it for $300k+ per year the thing can go way higher, but I think that is far from certain. I did not suggest $180 for the PT. I fully agree there are significant risks with drugs. All it takes is a few bad side effects and your drug goes in the trash. I think this one has a good chance of actually paying off. One way or another, I find out sooner or later.
Gleevec, in 2012 (per Wiki) brought in annual revenues of $4.7B to Novartis. That was for approximately 50K patients per year. Insurance covered the full cost of it for the record with no caps. The cost to produce the drug per patient per year, is likely under $1K, which means that the drug essentially added $4.7B to profit before tax. I am not looking back at their old financials, but I am pretty certain the majority of their income came from this. Oh, also make sure to add Tasigna (a second-generation variant of Gleevec that is generally more effective) to this as they are both from Novartis and treat the same disease and cost similar amounts (currently I think over $100K per year, fully covered by insurance... could be some countries get it for a lower price, but it is still crazy expensive and covered by insurance in just about every place, otherwise people would need bone marrow transplants and would be dying from this still). Basically, CML which is a relatively rare disease, is, I think, the most significant source of profit for Novartis by far. Just to be clear, Gleevec and Tasigna are essentially pure profit (cost to produce annually I think is estimated under $1,000, whereas revenue is about $100,000 per patient and there is little competition for first-line treatment of CML. They may have other drugs whose revenues exceed these, but the profit margins are much less on them. I am not making this stuff up, the massive profits of Gleevec and Tasigna has been brought up here and there, but due to the relatively few people with CML, it has not been a pressing legislative matter to address. If you want to know how insane the story with Gleevec is, read up about the development of it and Dr. Brian Drucker's research. Whatever, I don't invest in Novartis, but just pointing out a situation where a drug for a rare disease where there were not effective alternative treatments can lead to massive profits. With Bertrolast, the competitive treatments, I think, cost between $350k-$400k. Bertrolast, when I looked at it, was not necessarily going to be suitable or effective for everyone, but at the time I ran my estimates, I thought 15% of HAE sufferers choosing them would be a conservative estimate. Pricing on these drugs, even if competition shows up, will remain high as no other way to recoup the development costs. So even if we are looking at a price reduction to $200k per year, it seemed reasonable that net $150k would easily be achievable per patient treated. Based on that, I get to $500m+ in income per year. These numbers seem within reason to me. I know this stuff is a gamble as drugs can get pulled for various reasons, but they seem on track to me to a major payoff. It might take a while for people to start using it and for revenues to reflect it, so even if it is a company that wills see its stock go up to $40+, timing that is not certain. I have a mix of shares, calls for 6/18/21 and calls for 1/21/22. I feel very confident about the Jan 2022 calls as I think revenues will have a chance to start reflecting this and, if it does work out, investors will jump on the stock accordingly before then.
This is BS. If I recall correctly, Novartis's most profitable drug was/is Gleevec which is used to treat CML, which is a rare disease. The annual cost of the drug is about $100k. Guess what, insurance pays for it. With the drug BCRX has, even if the disease is rarer, still insurance pays for this stuff so pretty much everyone can afford it. The question is whether they choose this course of treatment or an alternative. Since all the options are insanely expensive, BCRX does not have pressure to sell it for cheap. Frankly they should make some good money on it as otherwise no one would waste the money going bankrupt trying to find these drugs. About the history, I am not familiar, but there is always that possibility. That said, the drug I made some effort to research is Berotralstat which has passed approval already and even if a fraction of the people with HAE use it (say 15%, which seems very reasonable to me) the company net of expenses will easily make between $500M+ (between, US, Japan, Europe at a net profit of $150K per patient... the retail price for these drugs can be well over $300K a year) a year. With a current market cap of about $2B the company seems insanely undervalued. Only thing I can tell you, is do as much research into what HAE is and what that drug I mentioned is and let me know why I am wrong here.
**Financial highlights** * Revenues for the full year increased by 9.9% to €185.7 million (FY2019: €169.0 million), primarily driven by sales growth of RUCONEST® (recombinant human C1 inhibitor) in the US * Revenues in the US increased €48.1 million in Q4 to €177.4 million (FY2019: €162.7 million) driven by seasonally strong demand and some COVID-19 related additional ordering by patients in Q4 2020, despite significant negative currency effects from the weakened US Dollar versus the Euro * As result of the strong US sales performance in Q4 2020, the net sales level that triggers the payment of the final $25 million milestone to Bausch Health Inc. has been achieved. The payment of this final milestone will take place in Q2 2021 and concludes all obligations under the agreement with Bausch Health Inc. * Revenues in Europe and RoW increased by 69% to €8.3 million (FY2019: €4.9 million), driven by increasing demand in Q3 and Q4 2020 and as the Company continues to build out its EU commercial infrastructure and expands into new territories following the re-acquisition of EU rights for RUCONEST® from Swedish Orphan Biovitrum AB (Sobi). * Gross profit increased 11.8% to €165.1 million (FY2019: €147.7 million), due to increased sales in the US and EU, coupled with economies of scale in manufacturing, leading to lower cost of sales. * Operating profit improved strongly to €67.4 million (FY2019: €60.9 million), an increase of 10.7% despite an increase in clinical and R&D activity * Net profit of €32.7 million represented a decrease of 9.8% (FY2019: €36.2 million). This despite an increase in operating profit and reflects a significant increase in finance cost of €13.9 million. This was mainly caused by negative currency effects (€12.6 million). * Cash and cash equivalents, together with restricted cash increased to €168.3 million at the year end, compared with €68.6 million for the year ended 31 December 2019. This was mainly due to strong cash flow from operating activities and proceeds from the issue of a convertible bond which were partly offset by the repayment of loans. **Operational highlights** * Approval of second production facility for RUCONEST® starting material by the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) * Received European Commission (EC) approval to treat acute HAE attacks in children with RUCONEST®. This followed a positive opinion and recommendation from the EMA's Committee for Medicinal Products for Human Use on the extension of the indication for RUCONEST® * Received EC grant of orphan drug designation for leniolisib for the treatment of APDS, based on a positive opinion from the EMA's Committee for Orphan Medicinal Products * Announced positive results from a compassionate use study in patients with confirmed SARS-CoV-2 infections hospitalized with related severe pneumonia that were treated with RUCONEST®. Results were subsequently published in *Frontiers in Immunology* * Initiated two studies into the use of RUCONEST® in the prevention of severe SARS-CoV-2 infections in patients hospitalized with related severe pneumonia across Switzerland and the US, as well as in Brazil and Mexico * Successfully completed a secondary listing of American Depositary Shares (ADS) on the Nasdaq Global Market * Appointed Jeroen Wakkerman as Chief Financial Officer * In addition to a corporate governance change from a two-tier board to a one-tier board structure, Barbara Yanni and Mark Pykett were appointed as Non-Executive Directors of the Board, and Deborah Jorn succeeded Juergen Ernst as Vice-Chair of the Board * Accepted into the Euronext Amsterdam MidKap index (AMX) ***Chief Executive Officer, Sijmen de Vries, commented:*** *"We are pleased to announce continued growth during 2020, especially, having achieved a net sales level that requires the payment of the final* *$25 million* *milestone to Bausch Health Inc. significantly earlier than originally anticipated and despite the impact of the COVID-19 pandemic on sales and marketing activities. Strong growth in our operating result was also achieved, despite a significant decrease in exchange rate of the US dollar versus the Euro.* *We have also continued to deliver regulatory and clinical progress, despite the pandemic causing an initial halt in development across our existing pipeline, through the approval of RUCONEST® to treat children with HAE attacks and the grant of orphan drug designation for leniolisib for the treatment of APDS, both by the European Commission, as well as the initiation of two studies into the use of RUCONEST® for the prevention of severe complications of SARS-CoV-2 infections.* *In addition, we remain focused on building on our solid foundations to deliver long-term growth. In line with this strategy, we successfully refinanced the Company early in the year, under very favorable terms, received EMA and FDA approval of our second RUCONEST® starting material production facility and implemented plans to expand our in-house processing capability.* *Lastly, at the end of the year, we completed a secondary listing of American Depositary Shares on the Nasdaq Global Market, which we believe will enable us to accelerate our growth strategy to deliver significant value to our patients and other stakeholders."* **About Pharming Group N.V.** Pharming Group N.V. is a global, commercial stage biopharmaceutical company developing innovative protein replacement therapies and precision medicines for the treatment of rare diseases and unmet medical needs. The flagship of our portfolio is our recombinant human C1 esterase inhibitor, or rhC1INH, franchise. C1INH is a naturally occurring protein that down regulates the complement cascade in order to control swelling in affected tissues. Our lead product, RUCONEST® is the first and only plasma-free rhC1INH protein replacement therapy. It is approved for the treatment of acute hereditary angioedema, or HAE, attacks. We are commercializing RUCONEST® in the United States, the European Union and the United Kingdom through our own sales and marketing organization, and the rest of the world through our distribution network. We are also developing rhC1INH for subsequent indications, including pre-eclampsia, acute kidney injury and we also investigating the clinical efficacy of rhC1INH in COVID-19. In addition, we are studying our oral precision medicine, leniolisib (a phosphoinositide 3-kinase delta, or PI3K delta, inhibitor), for the treatment of activated PI3K delta syndrome, or APDS, in a registration enabling Phase 2/3 study in the United States and Europe. Furthermore, we are also leveraging our transgenic manufacturing technology to develop next-generation protein replacement therapies most notably for Pompe disease, which program is currently in the preclinical stage.
I will add more a bit later, but for starters BCRX was just approved by FDA for Orladeyo, which helps prevent HAE. This is a very debilitating health issue for the thousands of patients that experience this rare disease.
I think a large amount of the bullish sentiment is mainly due to Biocryst having other drugs coming down the pipeline in the future. Bcx9930 (on paper, at least) does look very promising to me. However, I'm an investor, not a doctor. And I'm fully aware that the information I am given could very well be skewed. Simply due to the fact that Orladeyo was given orphan status it is hard to believe they aren't onto something with it. But that could simply be because of demand created by a life threatening blood disease and not necessarily because of the FDA's confidence in the drug itself. Thinking objectively a drug that costs just shy of 500k only needs to sell to 120 people in order to generate roughly 60 million in revenue for the year. I don't necessarily need BCRX to own the entire market. 120 people out of the 140,000 affected by HAE attacks seems very much in reach. Pair that with other drugs being added to the pipeline in the next year or two, a sales team that has literally already sold the competition and the very large possibility of a short squeeze in the future I find it hard to believe that I should pull my money out on Monday. But that's why I'm here to brainstorm. To consider a very real possibility that I'm throwing money down the drain. I very much appreciate you sharing your point of view with me.
If there are only 10,000 people in the US suffering from HAE, then how can the drug be in high-demand?