Yes E*TRADE and Fidelity offer extended trading as well.

Thanks. I should have been prepared with my Fidelity account. By the time I woke up, it was 8 AM. I could have gotten $5.50. There will probably be more opportunities within the next many months.

Thanks.

any thoughts appreciated especially you MM – ive been with you a looooooooong time!

Chaz, what meeting is on Monday?

AKBA chat.

AKBA won’t say that much. The bell rings early August with Q2 ER. You have a year to buy CAPR hopefully at lower prices, maybe $5. Now I say that the money will be made first in AKBA. CAPR if it gets approval will advance much more over several years, because it has a monopoly on THE best DMD treatment, unlike AKBA which is fighting to get ESA patients to switch to V.

JGMD when you say you have a year to buy CAPR, is that because you expect this delay to set the approval back that long? Obviously, until the next meeting with the FDA to give additional guidance regarding their willingness to accept the additional data and how much of it, we can only speculate. Do you think the next meeting could happen within the next 30 days or is that too aggressive?
Given my lack of liquid funds, I’m wondering if shifting my CAPR money to AKBA or NGENF for a period of time, then back to CAPR as the time gets closer has any merit. Of course, as of today that’s just an idea, not the plan which is premature until after the next FDA meeting which will provide guidance and hopefully a better timeline will become clearer.

I don’t understand the arbitrary timelines of FDA meetings with companies seeking approval, CRL’s, resubmissions, new BLA applications, and plenty of other bureaucratic nonsense. If the FDA wanted to work together with CAPR to get this therapy approved ASAP while adequately assessing efficacy and safety, they would be more efficient. Their objections based on small numbers of patients and use of historical controls could have been addressed long ago, so it is obvious political corruption that in the 11th hour they issue a CRL. The FDA misled the company by saying that they accepted the small numbers and historical controls because of the rare DMD and compelling need to approve therapy desperately sought by DMD patients and families. How come the mid cycle review was fine and they now issue the CRL? This fiasco should be a lesson to any stupid voter who wants incessant oppressive regulations that do little to protect the public, but 1000X more harm In screwing them The FDA and regulatory agencies in many other fields all have blood on their hands.

Stocktwits has many posters who understand the intricacies of FDA timing much better than I. It’s hard enough to select small bios that have mega payoffs from the 99% of trash. Then to figure out the optimal timing of how to move your money around is another problem. Too many unknowns as we have just seen. My best, least risky idea would be for new money, buy AKBA rather than CAPR. But AKBA has already more than doubled over my $1.75 average cost. CAPR may retreat to $5 from $7.64 now, but the realistic potential of CAPR is so great that even if your average cost is $20, I think you’ll get there in 2026 when it is approved, and much higher several years later when it is rolled out to the DMD patients who ALL want it. To keep it simple, hold your CAPR, AKBA, my best bets. Scale into NGENF and ACHV over the next year. ACHV is nearest its bottom, so even though the big money won’t be made for another year, it is a safe buy at this price.

I missed ENVX not because I was asleep. I didn’t think it was investable because of the silence in how many big contracts would be signed, and the already high market cap. I can’t dance at every party, but I can spend my time on things I understand better. We can retire on any one of these bios that make it big. Just have a choice of a few of them.

Back to CAPR. Many CAPR investors are blaming Linda when the blame rests with the FDA who misled the company and all of us. I feel the HOPE 3 is nearly guaranteed to be successful as an academically accepted double blind placebo controlled much larger study building on the good data from the small HOPE 2 and its OLE over 4 years. Short term, AKBA is probably the best gainer from here, but long term, CAPR seems to be the best. I remember a recent video of Linda saying that if the Adcom was shaky, she would trot out her big guns, the HOPE 3 existing data to clinch approval, maybe even getting dual approval for skeletal muscle and cardiomyopathy. We might actually be in better shape now than a few days ago for long term gains.

Thanks for all your insight and details on these stocks.

Too bad it is hard to get the full report on any brokerage recommendation. My assessment is that the timeline for CAPR has been delayed for up to 1 year. Suppose that a month ago, HCW projected $77 after 2 years of sales. So now I say, use that target 3 years from now, possibly less depending on whether dilution is needed to raise money for 1 more year of survival without revenue. CAPR said they have cash until 2027, so maybe they can squeak by with launching just before cash runs out, so no dilution. If they prudently decide to dilute and raise more cash, then $77 in 2 years becomes $50 in 3 years. I’ll still be happy.

But maybe HCW figured $24 after 1 year of launch which is 2 years away, instead of $77 after 2 years of launch and the same 2 years away. That’s fair.

This should get you to the webcast replay

. To participate via webcast, click here to access the live stream.

Thanks. I feel even more confident after listening to Linda. I’m not an expert in statistics, but here are my impressions. She said that HOPE 3 is overpowered for LVEF (cardio) and well powered for PUL (skeletal muscle), more powered than in HOPE 2. To understand power, you look at the chance of advantage and ask how many sample sizes it would take to be confident. To prove which of two top race horses is more valuable, you might need 10 races to do this. Horse A wins 7/10, B wins 3/10. 7 vs 3 is good enough to draw a reliable conclusion. 10 races are powered enough to show this. Now suppose that A and B are identical twins with the same height, weight and trainer. It might take 1000 races with 550 A wins and 450 B wins to conclude A is better than B. 10 would be underpowered, 1000 is enough power, and 100,000 is overpowered. P value of 0.05 means that there is 5% chance that the results are wrong. The lower the P value, the better.

Linda said that even HOPE 2 had P values less than 0.05 (great) and good power, although the FDA thought that only 7 patients wasn’t enough power. She now says that 104 patients is overpowered, with estimated P values even lower. Even if the FDA thinks less optimistically than Linda, they probably will agree at the type A meeting coming up that HOPE 3 will be well powered. I’m still wondering how Linda could know that the HOPE 3 P values are very low and it is overpowered WITHOUT KNOWING THE HOPE 3 RESULTS?

Opie, I recall you used to be an experimental psychologist and probably have good knowledge of statistics. What do you think?

I’m not a statistician, so this is just my two cents. (My background is undergrad applied math, philosophy of science/logic, and MA/PhD in psychology. Taught psych stat courses (among many other courses for five years). Few scientific publications. About 20 years as an engineer designing new products and services. Also design, build and renovate homes, sort of a hobby.)

I think you know most or all of this….but to add some explanation: P-value refers to the probability that the results may have occurred by chance. Generally, a P-value less than .05 (less than 5% of the time would occur just by chance) is considered “statistically significant”, and the results are considered to confirm that there’s a reliable difference between the intervention (med) and the control condition or control group (no med). (I’d say p<0.05 is good rather than great, but that’s in psych studies, maybe not in medical research.) Sometimes especially with large sample sizes, investigators like to see P-values less than .01 (1% or less probability these results occurred just due to chance variation). And its always good if someone else replicates these results, with a different sample of people, done by a different investigator, under somewhat different conditions. (We have tried to replicate some studies and failed to confirm results that others found.)

I would also guess that the FDA would like to see excellent safety results, or take this into account when considering approval. My understanding is that Phase 1 studies are supposed to be a safety study with a small sample of “gineau pig” subjects, to verify that the med doesn’t cause harm, before doing larger or repeated trials, although investigators are going to look for evidence of the effectiveness of their med candidate too. For example, with Arena’s weight loss drug, there was a concern about cancer in studies done with rats, although the mechanism was different for humans, so it was not supposed to be relevant, but was used as one excuse to reject Arena’s drug during the first Adcom meeting. Effectiveness of an drug candidate isn’t good enough if there are safety concerns or negative side effects; or else label warnings are required on the med container. Not sure if this may be an issue with CAPR.

There’s also a concept of the power of a study to be able to detect an effect. Did you design a study that would likely find an effect if there is one? I don’t see why this use of the word power should have anything to do with FDA review, since they did find a statistically significant effect (I presume, I haven’t read anything about it.)

Then there is the medical power of the proposed intervention. If a med or procedure doesn’t do much, not a strong effect, even tho it is shown to have a statistically proven “statistically significant” effect, the intervention isn’t very useful. When most people say “significant” in common parlance, it means powerful, whereas statistical significance just means it likely has an effect due to the trial drug, not necessarily an obvious powerful effect. I’m sure the FDA would care about the magnitude of the effect of a proposed new med. Later dosing studies might be required. And larger doses might create worse side effects or begin to create safety issues. (For example, a little salt in the diet doesn’t matter or could be good in hot environments. A LOT of salt in one’s diet creates medical problems.) So the FDA might get biased against approval if the proposed med is shown to work, but not work as substantially as they might desire to see to risk putting that med into use in the general population. I don’t really know, but these judgments might create bias that is technically not warranted in some of the FDA meetings or reviews of trial results.

Not sure if this is answering your question.

Good tutorial, thanks. In the case of “power” Linda and the FDA are primarily concerned with statistical power–how many patients it takes to demonstrate whatever effect or efficacy or goal. For deramiocel, in early research, they gave only a single injection which had negligible effect over a certain time period. They decided to treat every 3 months, and efficacy was shown. (I worry that NGENF will face a similar problem. The early trial was a decent success as a proof of concept in humans, but it is not ready for prime time.)

Cancer chemotherapy drugs get approved if efficacy is only a 4 month prolongation of survival. With months of side effects, it is questionable whether it is worth it, although I am not a dying patient who deserves a chance to accept risks and benefits of a drug like that. CAPR and NGENF patients don’t die as quickly as cancer patients do, so the standards for approval can be more rigorous for CAPR and NGENF.

BTW, higher rates of cancer were eventually seen in ARNA’s drug, Belviq. It nice to know that rats can teach us humans something. For NGENF, it’s nice to know that rats predicted responses in humans. I think NGENF has enormous potential, and I don’t know exactly how to play accumulating more shares.

I agree. She sounded pissed off on the call. Once approved, she will be swinging for the fences.

This issue between CAPR and the FuDA appears similar to ARNA’s first Advisory Committee meeting rejection, which seemed blatantly corrupt. ARNA CEO walked out of the meeting, some in Congress started questioning FDA about this meeting. I doubled down, and almost tripled down until discussing with someone who worked in biotech. Michael Murphy was at his best with guidance approaching the second AdCom meeting, which is why I’m still here after all these years. ARNA eventually became my second largest lifetime investment profit, 13x gain for bulk of shares sold about $10 on day of approval, bought below $1.

CAPR could become a similar investment win. Agree with El Capitan Nemo about adding CAPR shares (now on sale for a discount 😉 If not already heavily invested. Look at it as a potential opportunity, but remember biotech is a risky minefield, and remember to consider appropriate position sizing risks. (A rule I break sometimes….)

MM was in his heyday in 2012 for ARNA. On approval day, the stock hit $13, then 12 the next day, and over a few months was down to 8. I was following MM who said to hold after approval, hoping for EU approval and $20 target. I sold 3/4 of my shares at $4, only $1 gain from my cost. But I held 1/4 of my shares. ARNA re-invented itself with its autoimmune drug, Estrasimod. A 10 reverse split occurred, and on approval ARNA got a buyout at $90 (original price $9), so I did well on those 1/4 shares. Lessons from this are contradictory. Most pro investors take profits on spikes on approvals, unlike MM and myself. But holding ARNA for its different drug was better. I believe that CAPR will get a mildly delayed approval, with a mild risk coming from political sabotage from FDA payoffs from competing BP interests or even SRPT which does gene therapy. But I think the long term potential is so great that I won’t sell upon approval.

SRPT is under a big cloud from the 2 deaths from liver failure.

I have to learn more about Elevidys, SRPT’s one time genetic therapy for DMD. Prevention is always the best strategy, but I don’t know if E can be used to slow disease once symptomatic. There’s where deramiocel has the most promise. Ideally, E will be used for primary prevention, and Deramiocel for secondary prevention of severe disease. I still think that D will be promoted for all patients. If all 23K worldwide MD patients get D, whenever that is, we have $1200 target stock price. For 2K patients, $160 target 2 years after launch. If Chris and Asher77 of ST are right that approval will be only mildly delayed to Oct/Nov 2025, we won’t have dilution so these targets are still in place.

Reading on X “Upcoming CAPR catalysts that will move the share price are Hope-3 results, and a PR with a new approval strategy and timeline following the FDA meeting. I expect good news on both catalysts, but don’t believe we’ll see either one until August/September”. Do all agree that we may see positive stock price movement by September?

Another boost in the stock may come soon if the type A meeting goes well. There are stupid discussions by bears on ST about the FDA agreeing on changing the secondary endpoint of LVEF to primary endpoint for this BLA for cardiomyopathy. Idiot bureaucratic FDA. Of course for a cardio approval, use the cardiac endpoint of LVEF and other MRI data. Whether the wordsmiths consider it primary or secondary misses the point. Bureaucratic stupidity is the only risk for this stock, since I think a good HOPE 3 is highly likely (overpowered with very low p value, according to Linda’s 7/11 CC).

In other words, a bear who thinks CAPR gets a final rejection despite good HOPE 3 is betting that Prasad and company are the devil bogeymen devoid of reason.

Yes I do

Oh yeah, I forgot to include food. Same story. I don’t know where some of you get your facts, but if they are coming from Trump, best check for yourself.
https://fred.stlouisfed.org/series/CPIUFDNS

I subscribe to WSJ, NYT, WAPO and watch FoxNews, CNN, MSNBC and Meet the Press, This Week and Face the Nation. And you?

You can watch all you want, if youre not intelligent enough to understand the complexities or know the difference between fact and biased opinion then it doesnt matter what you watch or read. When you said the government shouldnt be generating a profit (also known as a budget surplus) you revealed your lack of political and business sense

You’re the one who can’t spell or punctuate properly — hardly proof of intelligence.

This is THE most intelligent post of all.The fact that it got 3 thumbs down shows the ignorance of those people. They want more and more govt debt. They are ignorant or stubbornly refuse to understand that out of control spending leading to gargantuan debt is what destroyed civilizations. None of them run out of control debt in their own households. They are fiscally responsible in their own households or even small localities. Why don’t they understand that the same applies nationally?

All leftwing rags except Fox, maybe WSJ. The NYT denied the Nazi Holocaust because it was too embarrassing to admit the atrocities of a socialist dictatorship. NAZI = National Socialist, by definition. Suggest getting a common sense education from the publications of the Ludwig von Mises Institute from Auburn University in Alabama. Don’t disparage Auburn as not having the ranking of the Harvard ilk. These prestigious bastions of liberalism are anti-American and pro-Arab. You could cite statistics from leftwing rags that support their views. Aside from inherent bias inherent in what they report, it is more important to understand the principles of a free lifestyle and respect for others’ rights to the same, without using big govt to impose actions that interfere with same.

In case you didn’t know, many studies show that Fox News viewers are the least informed about current events.
https://www.forbes.com/sites/quora/2016/07/21/a-rigorous-scientific-look-into-the-fox-news-effect/

Commies and socialists don’t spell out what bad things they are planning. Campaigns promise giving as many groups as possible freebies so they can get votes from all these special interest groups. Social justice for all, and numerous other platitudes, all false. Universities consist of academics who are sheltered by these platitudes who have little work experience. Years ago, I asked a Chinese lady who witnessed the rise of Mao, how could people fall for his crap? She basically said they were gullible and were suffering so much that they wanted to believe what Mao promised. Early on, Hitler was also charming, charismatic and wooed the crowd.

You are one of the gullible people who deny that Mamdami will attempt to seize means of production. He is an avowed Commie, advocates violent worldwide intifada and is supported by Bernie Sanders and AOC. Another friend thought Biden was a moderate in the early days of his campaign. But Biden brought criminals across the border which has terrorized and killed many people. Why? Not as a humanitarian mission, but as a means to buy votes for Dems who gave them freebies and a means to survive, that even the poor citizens of the US have to wait behind them. NYC mayor Eric Adams initially embraced making NYC a sanctuary city, but after facing reality that there was no money to give all the illegals benefits without sacrificing the needs of his resident poor, realized that it was a mistake. I have respect for people like Adams who admit their mistakes, but I have disgust for incorrigible academic assholes who never give up their wrongheaded dogma. I wouldn’t mind these academics who have the right of free speech, sticking to themselves, but when their free speech progresses to give mandates and control other people, that is wrong. It is true that Trump is attempting to micromanage world economies and dictate tariffs and other things, but he has a tough job trying to correct damage done by socialist Dems like Biden who efficiently destroyed social conditions in the US like defunding the police. Worse, the rise of terrorism from China, Russia, Iran directly relates to Biden’s weak America Last behavior. Don’t retort with flawed claims from leftwing media which I don’t trust at all.

You think Mamdami is only MY problem? No, it is also your problem because other Mamdami type Dems have destroyed major cities. A typical liberal makes excuses that bad things will be “contained” and as long as it is not in their neighborhood, everything is OK. No, a Commie NY will see mass exodus of billionaires, resulting in declines in quality jobs available for all income groups. In the last several years, productive people who could move did so and moved to Repub states like Florida, Tenn. Real estate is booming there.

Stick to your excellent stock analysis. Your liberalism is nurtured by left wing media and left wing academics. My views have been shaped by my experience in the real world, starting from my mother who was a housewife but had more common sense than the majority of damn professors I knew or read about. There were a few brilliant professors like Milton Friedman who had common sense. I respected economist Walter Williams who was a poor black child from Phila who grew up to understand free market wisdom. He had productive influence on some black people and tried to convince them that Dem black leaders were holding them back.

Regarding more subtle socialist indoctrination beginning in early grade school, a child is taught that his own needs are subservient to that of the group. “Don’t be selfish.” 40 years ago, I visited a Kibbutz in Israel. The young lady was interested in buying a camera for her hobby, but lamented that the group didn’t permit it because the group’s need for essentials was more important. She should have relocated to a private living situation where she could have had incentives and opportunities for her own fulfillment. I admired Ayn Rand’s writings such as “The Virtue of Selfishness” whose title was deliberately provocative. Rand promoted rational self interest which respects nonviolent free competition but not the typical connotation of selfishness achieved by theft, cheating and sabotage. She promoted her concept of rational self interest as the moral basis of free market capitalism. The academics rejected this due to their upbringing indoctrination in socialism. They deliberately transmuted “self interest” to “selfishness.” Then they justified their control with big govt, as long as the intellectual elites had control because they supposedly knew better what all classes of people want and need, better than individuals themselves.

Rand was atheist, and disliked the attempts of religion to justify the moral freedom of capitalism. Most religions support self-sacrifice as a noble ideal. That actually is more consistent with socialism to promote higher taxes to take more money from “selfish” people who should practice self-sacrifice for the benefit of the general public and especially special interest groups. An example was Biden’s forgiveness of student loans to buy student votes. So the support for capitalism by religious Repubs is on shaky ground. Religious groups asked Rand to throw in a little religion in her works to make her teachings more saleable to the general public, but she refused to harbor such inconsistency. There is a nice collection of her essays in “Capitalism, the unknown ideal.”

1. You claim those who disagree with you have bad intentions that they are hiding. That’s true for criminals, but not everyone who has a difference of opinion is hiding their true intentions. This paranoia can blind you from really understanding what they actually intend to do, or from working with them. Just because someone has a difference of opinion doesn’t mean everything they believe is wrong and they should be condemned as communists,
2. Communism and socialism are not the same thing. Reread what I reposted. Then try to describe the differences please. Biden is not a socialist. Trump is not a Nazi. Similarities in some ways (“Early on, Hitler was also charming, charismatic and wooed the crowd.“) don’t make them the same. Stereotyping might make you feel righteous and superior but you are confusing yourself.
3. “Universities consist of academics who are sheltered by these platitudes who have little work experience.” That is so insulting. I worked as a paperboy age 10-12. I worked as a janitor at age 16-17. I worked in mills for years, cleaning looms, eventually working up to running the shipping department, sometimes working there full-time while going to college full-time. I had a full-time job while going to grad school until UCLA figured out they were giving me paychecks in biomed while I was enrolled in the psych grad program and cut off my paychecks. I’ve performed 40 different jobs in my lifetime.
4. In my psych classes I focused on teaching practical skills like how to improve studying, quit smoking, start and maintain exercise routines, listen to others’ emotions in conversations and reflect back what you heard in your responses, shape children’s behaviors, … Is that socialism communism?
5. For someone who is so intelligent and dedicated to medicine and health, you can be incredibly stereotyping and close-minded. Try summarizing what others said and reading it back to them to see if you understood them. Hint: You probably didn’t, you were too busy ranting about communists etc.

You deny basic principles underlying free societies, and why people are fleeing countries that have excessive regulations and limit freedoms. Commies are just worse socialists. You believe what you want to believe. Read Rand, the most eloquent defender of the moral basis for free market capitalism. If you have any intelligent refutations of her, I’m listening.

We can learn basic life skills in schools run by commies, socialists, religious and private groups. Your parents taught you how to go to the bathroom, get dressed, how to read at a beginning level, how to do home chores, etc. But public education institutions have had a decidedly leftwing bias. When it comes to voting, people need an education about why more govt power leads to worse social and economic results in the aggregate and esp also for individuals to be free of coercion. You are willfully blind if you deny this. You are free to live your own life according to your standards, as long as you don’t interfere with others’ right to the same. But govt workers have no moral right to enforce regulations that hurt others’ rights to live according to their standards. That means as little interference with others as possible. It is axiomatic that socialism/communism = interference, and free market capitalism = liberty. Most voters are poorly educated to understand this, and just vote for whichever candidate will give them freebies at the expense of others.

Dont care enough about the phony to know the correct soelling of his name

Unfortunately, he is not a phony for his real agenda, deliberately planned just like Mao, HItler, Xi, Kim of NK, Putin, numerous ayatollahs of Iran, Bernie Sanders, AOC. He’s got the backing of academics, who I consider the real phonies. Academics lend an air of respectability, which is actually the opposite of reality because of their intellectual damage to generations of college kids grooming to be future leaders.

I’ve learned to forgive people who make spelling errors, but I engage them in substantive discussions. Spell checks are flawed–often my spelling is “corrected” incorrectly because the stupid AI thinks I mean something else. With Mamdami, his name hasn’t been written until recently. In school, I was a good speller from my experience in reading, but even I made an error in spelling his name. No reflection on my literacy.