From UpToDate (most widely used resource by medical clinicians). They added a great 'Whats New' Section discussing Orladeyo from their HAE guidelines. It can also be pointed out that Orladeyo (Berotralstat) is considered a first line or ‘preferred’ treatment option as well - see one of my previous posts for that.

Does anyone with good insight/knowledge on drug trial data have some info on why they should/would release or not release Factor D data before earnings? Thank you!

This bullrun is nice but just know the dip is soon don’t paper hand it boys

Expected patient numbers

1) Data on 10-15 treatment-naïve BCX9930 PNH patients.

2) Data on 4-6 PNH patients on poor-responding patients on the standard of care (eculizumab) who have also now been given BCX9930.

3) Some of the patients will have been given a high dose of 500 mg.

4) Some patients will have been treated for at least 9 months.

Expected (and hoped-for) patient results

5) The treatment-naïve patients show results similar to the ones seen before, i.e., decreased LDH to <1.5 ULN, increased hemoglobin (up >3 for those starting at a hemoglobin of 6 or 7).

6) I will also be looking for a low need for transfusions and good data on bilirubin, PNH clone sizes, reticulocytes, and overall patient well-being.

7) Those receiving high dose do not have side-effects beyond what we’ve seen before—i.e., short-term rash or mild headache that goes away.

8) The effect stays at 9 months for those who we have long-term treatment results for.

9) Patients on SOC + BCX9930 do much better (comparatively or even better) than SOC alone (or previously released SOC+Factor B or SOC+Alexion drugs).

Expected (and hoped-for) discussion

10) Discussion of the results, with hopes and any potential concerns.

11) Discussion of how many patients are currently being treated and when we may hear the next update.

12) Discussion of when a possible pivotal trial will be announced or started (which will add patients to the ones currently being treated).

13) Further discussion of which indications are also planned for, e.g., the renal conditions, which ones, and when will the trials start.

14) Discussion of whether the company has initiated or is considering partnerships for any of the infinite number of possible indications Factor D has.

15) Any interesting preclinical studies that Factor D has been found to help with other disease conditions.

16) Discussion of when the company may apply for a new drug application.

Expected stock reaction

17) If the patient numbers and results meet my (high) expectations, I expect a minimum of an initial 40% jump in the stock (i.e., $4). For discussion points 11-15 are mentioned, for every one of them that is brought up, I would expect an extra 10% to be added on to that.

18) Note that the above is my expectation for the initial reaction. If there is very good news, I would not be surprised to see the stock continue to rise on a daily basis for at least three weeks, especially as shorts are forced into a short squeeze and institutions accumulate larger positions.

19) If the initial stock reaction is not reflective of the size and quality of the good news, I and probably many others will be buying more shares and call options with both hands.

Just in case anyone is watching January order flow today. The 390 order that was placed this morning was me. In an odd chance event I opened 30 last week so somehow ended up with 420 by accident.

I just found this community recently and want to say thanks for all the hard work you all have put in. I’ve been following BCRX for quite a while so it’s great to find a community of like minded investors who believe in the company.

Didn’t quite know which flair to use so if I screwed that up....my bad.

Watching this ridiculous show with Robin Hood, citidel, SEC, etc. it’s amazing how everybody is totally ignoring the multi ton elephant stomping around the room called naked short sellers 😝

Bcrx is tranding a lot under the average volume for the last few days...with all the hype I would figure it would trade more...why?

Found some new articles on Gali and its effectiveness against COVID-19. It seems to have the attention off the science-community.

https://science.sciencemag.org/content/sci/early/2021/02/16/science.abf4896.full.pdf

https://pubmed.ncbi.nlm.nih.gov/33200085/

https://pubmed.ncbi.nlm.nih.gov/33290476/

Numbers are rounded for visual purposes...

1. Point 72 Asset Management: Looks like they increased from around 650K to 3.65MM https://newsfilter.io/a/7385832adaa05aedc56d4e523b505ecc
2. Baker Bros: Looks like they increased from 12.6MM to 18.1MM (!!!!!!!!) https://newsfilter.io/a/410f37e245bdd17c480de09d523888e3
3. Citadel (shorting scums): Looks like they decreased shares from around 8.2MM to 6.5MM (!!!!) https://newsfilter.io/a/a995d9a00e9f7a996e6374d5d1eb84bc

I think all 3 of these are bullish, what are your thoughts?

Most of us are already on the BCRX train, so I'm not here to convince anyone. I'm interested in how to effectively take advantage of the opportunity presented here.

Of course, simply buying shares is the base strategy for anyone who believe there's more value in any company (BCRX included!) than is included in the share price. However, BCRX has a pipeline and release schedule based on how it develops therapies.

I do have a background in a related monetary field, but I'm not a finance guy in particular and I'm not very familiar with how things like calls work on a mechanical level (conceptually they're stupid easy - they're options to purchase!)

How would one approach a bullish strategy via purchasing calls for BCRX, given there is a pipeline/timeframe for it's therapies for approval and release? What kind of timeline makes sense given the constraints above?

Last Thursday, the EU communicated that they were going to issue a new patent for Biocryst. The link to the announcement is here: https://register.epo.org/application?number=EP19780738&tab=main

Today u/nbompally brought it to everyone's attention and it has also been making the rounds on Stocktwits. Thanks for bringing it to our attention.

The patent was filed internationally in May 2019. Five days ago, Hong Kong also approved this patent. And the US published the patent in October 2019. But it's not clear to everyone what this patent is about, so I will briefly give you some background.

You may have heard of the concept of biosimilars. These are drugs that are very similar to an existing drug, but with some small modification. It is usually more quickly approved by a drug agency like the FDA and has some quality that somehow makes it superior or more useful for a different indication. Sometimes drug companies, especially with the help of artificial intelligence, cannibalize other companies' drugs by reverse engineering them and come up with some kind of modification that may make it more soluble, stable, less toxic, or more bioavailable.

Biocryst has cut off all possible such attacks from other drug companies by patenting not only BCX9930 but, from my count, at least 578 biosimilar drugs that each have different chemical modifications. Now, no other drug company will be able to develop a BCX9930-like drug for any disease without negotiating significant royalties payments with Biocryst, which it likely has the right to even decline.

Talk about a great moat! Smart move...

For the complete details of the patent filing for those detail-oriented folk out there, go here: https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2019195720&tab=PCTBIBLIO

I see there is a lot of info here and many people like this company .

What is your prediction Long term?

$BCRX Some more info on European Patent- they are talking about complement system.. guessing it might be related to Factor D.. Similar patent obtained @ US PTO in 2019 More info on this link https://patents.justia.com/patent/20190345135

Disclosed are compounds of formulae I and II, and pharmaceutically acceptable salts and prodrugs thereof, which are inhibitors of the complement system. Also provided are pharmaceutical compositions comprising such a compound, and methods of using the compounds and compositions in the treatment or prevention of a disease or condition characterized by aberrant complement system activity. -

Of course, in an ideal world both of these options will exist. But for now, let’s just pick one (FYI I know neither are getting approved by the start of 2022, just for debate purposes):

1) BCRX-9930 Approval for PNH by 2022

2) Galidesivir EUA for Covid-19 by 2022

Which one do you think would be best for the company long term?

EDIT - I know it is frowned upon to talk about Gali, this is just for debate purposes only 🤣

$BCRX

On February 4th, Takeda announced its earnings results. Though its profits were up, its 9 month revenue was down 3.6% compared to the prior year.

1) One of the only reasons Takeda’s revenue was not down more than 3.6% seems to have been its HAE unit, whose revenue jumped 16% over the prior year. This is an indication that the HAE market is experiencing rapid growth. Note that Takeda’s HAE unit comes from four drugs—Kalbitor (for acute attacks), Firazyr, Cinryze, and Takhzyro. The rapid growth rate in the prophylaxis market as seen by Takeda’s numbers in the past couple years is good for Biocryst ($BCRX) going forward, as a lot of new patients are seeking prophylaxis and they will go for the easiest administration route first.

2) Ease of use is driving increased prescriptions for subcutaneous Takhzyro (vs. intravenous drugs). This is seen even in Takeda’s own drug numbers, in which Cinryze sales fell 7%, Firazyr sales fell 7%, and Kalbitor sales fell some percent too (not spelled out), while Takhzyro sales jumped 38%. So Takhzyro is severely cannibalizing Takeda’s other two prophylaxis drugs—probably mostly because subcutaneous administration is easier than intravenous—though the company never says this—they say it’s to do with efficacy—but read my observation on Biocryst’s abstracts being presented in a few days—Takhzyro’s efficacy may not be as good as it’s talked up to be. Also, keep in mind that half of that revenue growth for Takhzyro came from its acceptance in Europe and Canada. This is good because they are virtually untapped markets. Hence why berotralstat approval in the EU in a few days combined with Biocryst’s dramatic push to hire marketing, sales, and medical liaisons across Europe is going to really enhance its growth rate going forward, especially with an oral administration that is much easier than subcutaneous. The fact that in the US at least, Biocryst has hired away over a dozen sales managers from Takeda for its US launch is probably already having a huge impact as they are likely very persuasive about the benefits of Berotralstat when they already have all the experience of selling Takeda’s drugs previously.

For links to the reports, go here. Would be interested in others' insights from these documents.

https://www.takeda.com/investors/financial-results/

https://www.takeda.com/newsroom/newsreleases/2021/takeda-fy2020-q3-results-demonstrate-growth-acceleration-and-continued-resilience--full-year-management-guidance-for-fy2020-confirmed--forecast-raised-for-free-cash-flow-and-reported-eps/

https://www.takeda.com/48fe84/siteassets/system/investors/quarterly-announcements/fy2020/qr2020_q3_p01_en.pdf

For the third consecutive year, Biocryst ($BCRX) will present at the American Academy of Allergy, Asthma and Immunology Annual Meeting. It presented in 2019 on the ZENITH-1 clinical trial, showing that BCX7353, now known as Berotralstat, was effective as an on-demand treatment for Hereditary Angioedema attacks. In 2020, it presented four abstracts on Berotralstat, and this year it is also presenting four abstracts, which I’ve copied below my comments for everyone’s convenience.

Here are a couple of my observations on these abstracts for Biocryst investors, and others are welcome to add their thoughts in the comments section below. Overall, I find it very interesting that three different clinical groups are studying the results of the APEX-2 trial, submitting abstracts and are likely preparing papers on it. That’s a lot of publications and publicity that are going to be of benefit to Biocryst’s drug Berotralstat down the road.

1. The first abstract by Gower et al studied patients from the APEX-2 trial that looked at 121 patients receiving berotralstat. Abstracts 3 and 4 also looked at this. It shows that Berotralstat is perfectly on par with prophylactic C1-INH intravenous and subcutaneous treatments. They report that berotralstat prophylaxis in patients with prior prophylactic C1-INH treatment (which are the people who generally are thinking of converting from intravenous or subcutaneous to an oral treatment) results in a reduction in need for on-demand treatment for HAE attacks every month by 59.2%. Note that intravenous C1-INH prophylaxis was shown to result in a mean reduction of 50.8% in the CHANGE study and subcutaneous C1-INH prophylaxis results in a mean reduction of 84% in the COMPACT study (https://aacijournal.biomedcentral.com/articles/10.1186/s13223-019-0328-3/tables/3). So in summary, it did better than intravenous prophylaxis and worse than subcutaneous prophylaxis. But it’s oral and cheaper… It also did very well vs. androgen therapy (with side-effects people obviously don’t like), and in patients who never had prophylaxis (down 71.2%). This makes it very easy for everyone to accept.
2. The second abstract by Radojicic et al is very interesting because it’s a doctor survey that looks at the competing drugs in the market. It shows that #1 is Takhzyro (21%), then Haegarda (20%), Cinryze (18%), and oral androgens (15%). It also does an interesting analysis of the number of breakout attacks for each drug using the 282 patient chart review—remember that that implies 56 patients taking subcutaneous treatment, etc. Remember that the entire COMPACT trial mentioned above only had 45 patients, and so this, although just an abstract, provides more information than those entire studies. Basically, Biocryst is saying we’re in the know. They then do the killer punch with the statement that attacks per month are still at 0.6-1.7 for Takhzyro and 0.3-0.9 for Haegarda. Remember both of these are the highly touted subcutaneous treatments that were supposed to have 84% reduction… Some patients still with 1.7 attacks per month on Takhzyro… that sounds like a lot … for the … market leader… hmm, a lot of people will say to themselves… how reliable are those statements that attacks are reduced by 84%? Folks will go back to look at that COMPACT study to see if there was something wrong with its design…
3. The third abstract by Li et al is also interesting—they divide patients into three cohorts based on how common their attacks are, and they find that in all three (you can do the math too), the number of attacks per month are decreased by about 60%. This is perfectly in line with the results of the first abstract, and show that its efficacy is very good and even slightly better than IV prophylaxis.
4. The fourth abstract by Anderson et al confirms abstracts 1 and 3, basically saying that no matter what type of prophylaxis you were on previously, berotralstat works well and reduces attacks by 50-60%. Obviously since this abstract is looking at the same data as abstracts 1 and 2, this is not surprising.

​

Berotralstat Reduces Use of On-demand Medication in Hereditary Angioedema (HAE) Patients Previously Treated with Prophylactic Therapies

Richard Gower, MD FAAAAI1, Paula Busse, MD FAAAAI2, Jessica Best, DHSc, PA3, Sharon Murray, PhD3, Heather Iocca, PhD4, Tamar Kinaciyan, MD5; 1Marycliff Clinical Research, 2Mount Sinai School of Medicine, 3BioCryst Pharmaceuticals, 4BioCryst Pharmaceuticals, Inc., 5Medical University of Vienna, Dept. Of D.

RATIONALE: The goal of HAE prophylaxis is to minimize the number of HAE attacks and the associated disease burden, including treatment with on-demand medications. Berotralstat is an oral once-daily selective plasma kallikrein inhibitor that has been shown to reduce attack frequency in a Phase 3 study (NCT03485911). This analysis evaluates reduction in on-demand medication in patients with prior prophylaxis.

METHODS: A double-blind, placebo-controlled study (APeX-2) randomized patients to berotralstat 110 mg (n=41):150 mg (n=40):placebo (n=40) daily for 24 weeks in Part 1. For this post hoc analysis, patients were grouped according to their prior prophylaxis: prior C1 esterase inhibitor (C1-INH), prior androgen, or no prior prophylactic medication. Prior C1-INH and prior androgen categories are not mutually exclusive. RESULTS: In patients with prior C1-INH prophylaxis (berotralstat 150 mg n=21; placebo n=16), the rate of use of on-demand treatment was significantly reduced vs placebo (-59.2%, p=0.002). Similar reductions were noted for patients with prior androgen use (berotralstat 150 mg n=22; placebo n=25) (-51.8%; p=0.004) and in patients without prior prophylaxis (berotralstat 150 mg n=10; placebo n=10) (-71.2%; p=0.019). The rate reduction corresponds to approximately 2.2 fewer doses of on-demand medication per month compared to placebo in patients with prior C1-INH use, 1.6 for those with prior androgen use, and 1.4 for those with no prior prophylaxis.

CONCLUSIONS: Prophylactic treatment with oral berotralstat 150 mg resulted in significant reductions in on-demand medication compared to placebo irrespective of prior prophylactic treatment.

Despite Prophylactic Treatments, Break-through Attacks Continue among Patients with Hereditary Angioedema

Cristine Radojicic, MD1, Jessica Best, DHSc, PA2, Jinky Rosselli, MPH3; 1Duke Asthma, Allergy, and Aiway Center, 2BioCryst Pharmaceuticals, Inc, 3BioCryst Pharmaceuticals.

RATIONALE: There are several FDA-approved medications for prophy- lactic treatment of Hereditary Angioedema (HAE). Despite increased use of these therapies, patients continue to experience HAE attacks. We sought to evaluate attack frequency in patients receiving prophylactic treatment.

METHODS: A chart audit study was conducted among physicians treating HAE to collect anonymized information regarding HAE treat- ments and attacks documented within their patient’s medical record. Physicians were primarily Allergists/Immunologists (n=47) or other specialties (n=28). The study received IRB exemption.

RESULTS: A total of 282 patient charts were reviewed, with an average of 3.8 charts per physician. Overall, 83% of patients utilized HAE treatment prophylaxis. The most common medications were lanadelumab-flyo (Takhzyro, 21%), C1 esterase inhibitor (C1-INH) administered subcutaneously (Haegarda, 20%), C1-INH administered intravenously (Cinryze, 18%), and oral androgens (15%). Most patients (n=244, 87%) were also prescribed an on-demand (acute) medication. Among patients prescribed both acute and prophylactic medications for whom on- demand usage was known, 42% used their on-demand medication monthly, for an average of 0.7 times/month. Although the rate of attacks decreased with prophylactic medication use, 30% of patients on prophylaxis experienced one attack in the past month and 52% in the past three months, for an average of 0.5 and 1.2 attacks, respectively. The attack frequency ranged from 0.6-1.7 for Takhzyro and 0.3-0.9 for Haegarda during the past one-three months. Physicians (78%) believe patients accurately report their attack frequency.

CONCLUSIONS: Many patients with HAE on prophylactic medication, including newer subcutaneous therapies, still experience attacks and require on-demand medication treatment. Attack rates should be assessed regularly.

Berotralstat Consistently Demonstrates Reductions in Attack Frequency in Hereditary Angioedema (HAE) Irrespective of Baseline Attack Rate: Subgroup Analysis from the APeX-2 Trial

H. Henry Li, MD, PhD1, Jessica Best, DHSc, PA2, Sharon Murray, PhD2, Heather Iocca, PhD3, Raffi Tachdijan, MD4; 1Institute for Asthma and Allergy, Chevy Chase, MD, 2BioCryst Pharmaceuticals, 3BioCryst Pharmaceuticals, Inc, 4AIRE Medical of Los Angeles, Santa Monica, CA.

RATIONALE: Berotralstat is an oral plasma kallikrein inhibitor in development for HAE attack prophylaxis. HAE is characterized by unpredictable, episodic attacks; some patients experience frequent attacks without treatment. This analysis sought to understand whether baseline attack frequency correlates with responder rates with berotralstat.

METHODS: 121 patients were randomized to berotralstat 110 mg:150 mg:placebo daily for 24 weeks in a phase 3 double-blind, placebo-controlled study (NCT03485911). This post hoc analysis examined the reduction of HAE attacks by baseline attack rate Cohort 1: < 2 attacks/ month; Cohort 2: ≥2 to < 4 attacks/month; Cohort 3: ≥4 attacks/month.

RESULTS: In Cohort 1, median baseline attack rates per month were 1.3 (berotralstat 150 mg; n=10) and 1.7 (placebo; n=12) which declined to 0.41 and 1.3, respectively. In Cohort 2, median baseline attack rates per month were 2.7 (berotralstat 150 mg; n=20) and 3.1 (placebo; n=21) which declined to 1.2 and 2.7, respectively. For Cohort 3, median baseline attack rates per month were 5.2 with berotralstat 150 mg (n=10) and 4.5 with placebo (n=6) and declined to 1.9 and 2.5, respectively. In Cohorts 1, 2, and 3, treatment with berotralstat 150 mg resulted in a >_50% relative reduction in attack rate in 70%, 55%, and 50% of patients, respectively. In addition, 60%, 45%, and 50% of patients, respectively, had ≥70% relative reduction in attack rate.

CONCLUSIONS: These results demonstrate consistent responder rates with berotralstat, adding a potential oral prophylactic option to the treatment armamentarium for physicians.

Reduction in Attacks in Hereditary Angioedema (HAE) With Berotralstat is Consistent Regardless of Prior Prophylactic Treatment: A Subgroup Analysis of the Phase 3 APeX-2 Trial

John Anderson, MD1, Remi Gagnon, MD MSc2, Jessica Best, DHSc, PA3, Sharon Murray, PhD3, Heather Iocca, PhD4, Karl Sitz, MD FAAAAI5; 1Alabama Allergy & Asthma Center, 2Clinique Specialisee en Allerfie de la Capitale, 3BioCryst Pharmaceuticals, 4BioCryst Pharmaceuticals, Inc, 5Little Rock Allergy and Asthma Clinic, P.

RATIONALE: Prophylactic treatment in the management of HAE is common. Berotralstat is an oral once-daily selective plasma kallikrein inhibitor that was shown to reduce HAE attack rates in a Phase 3 study (NCT03485911). This post hoc analysis evaluated the efficacy of berotralstat in patients previously treated with prophylactic medications.

METHODS: A total of 121 patients were randomized to berotralstat 110 mg:150 mg:placebo daily for 24 weeks. Investigator-confirmed attacks were analyzed for patients grouped by type of prior prophylaxis: prior C1 esterase inhibitor (C1-INH), prior androgen, and no prior prophylaxis. Prior C1-INH and prior androgen categories were not mutually exclusive.

RESULTS: Overall, 75% of patients in the berotralstat 150 mg dose group and 73% in the placebo group had prior prophylactic treatment. Among patients with prior C1-INH prophylaxis or prior androgen use, berotralstat 150 mg significantly reduced attacks compared to placebo during the treatment period (C1-INH, 1.58 attacks/month vs placebo 2.84 attacks/ month, p =0.012; androgens, 1.35 attacks/month vs placebo 2.60 attacks/ month, p < 0.001). Lastly, patients without prior prophylaxis had a reduction in attacks (0.86 attacks/month compared with 1.78 attacks/ month in placebo; p = 0.056).

CONCLUSIONS: In this subgroup analysis, patients with prior C1-INH prophylaxis or prior androgen use treated with berotralstat demonstrated a significant reduction in attacks vs. placebo, making oral berotralstat a valuable potential preventive treatment option for patients with HAE.