Macro First

@msp12389 you clearly dont know the company or follow it closely

$REPL While we are at it, has $QURE ever shown evidence of all the "agreements in writing" they have with the FDA?

@adamfeurnstein @DesertDweller93 @Benchpressers @PDSBiotech Go back to jerking off to UFC

Happy Franky Friday to all that celebrate! $pdsb

@docrodwong It's so ridiculous

this is not helping cber/fda credibility. in the replimune CRL they say 'to account for potential bias' they switched to a new review team. this is a new concept for fda... and obviously will be subject to skepticism since we know the former review team head peter bross posted on linkedin that his team had a positive review (which was overruled by leadership). so now it is still flipflopping but adding a new element, the review team u've been working with for the past couple yrs can be switched to a new one when we don't like their opinion? download.open.fda.gov/crl/CRL_BLA125… ir.replimune.com/news-releases/…

@WhiteHouse Great Can you now deal with fraud @DrMakaryFDA ?

OFFICIAL STATEMENT OF IRAN:

@DrToonces @HarrieScarlet @DrMakaryFDA @SenRonJohnson @RepAuchincloss 🤞 l have heard that makary personally hates qure, but I hope you're right and im keeping expectations low until I see evidence.

I get the frustration but I'm not sure that's right. Makary reversed course on the Moderna flu vaccine after White House pressure. He reinstated Prasad after firing him, then let him go again when the heat got too high. The pattern isn't a vendetta. It's a guy who bends when the political cost gets high enough.

Is this the "common sense approach" you keep talking about Dr Makary @DrMakaryFDA ? How is it that FDA is insisting on a sham surgery for a fatal disease on placebo subjects who are then going to die a certain death? @SenRonJohnson @RepAuchincloss Right this wrong. This is unethical, cruel and not common sense based. @BeckyQuick @adamfeuerstein @l_e_whyte @ZacharyBrennan #AMT-130 $QURE

@DrToonces @HarrieScarlet @DrMakaryFDA @SenRonJohnson @RepAuchincloss Yeah but Makary has a vendetta against $QURE so nothing matters until he's gone or changes which is hughly unlikely

And the FDA's own Plausible Mechanism Framework, launched in February 2026, was specifically designed to avoid exactly this situation. Biomarker data, natural history comparisons, flexible trial designs for rare diseases. AMT-130 checks every box. The FDA built the framework and then ignored it for the one therapy that fits it best.

@viatoCEO @uniQure_NV All they are saying is Type B meeting and pursuing UK/Europe which has a different process. Nothing new.

Can @uniQure_NV please break the silence on AMT-130? It’s been 5 weeks since the last press release. The HD community and investors want to know what regulatory pathway is being pursued in response to the FDA’s hostility toward AMT-130. $QURE

@pawcio2009 +ve This management team have a history of selling / getting acquired. -ve I am not sure that their drug is especially potent and think $NGNE might win in Rett. Like the CEO but CMO doesn't know hoe to answer a question straight.

I again asked grok to name one bioturd with best R/R going forward from here. It gave me $tsha this time. Last year’s pick went >120% in a year. Let’s track this one now 🍿

@DesertDweller93 @uniQure_NV @VPrasadMDMPH @DrMakaryFDA @SenRonJohnson @SenRickScott @RepAuchincloss @adamfeuerstein @l_e_whyte @Temple_West @laurencurehd @JRenz0418 It's definite collusion.

@uniQure_NV $QURE AMT130. @VPrasadMDMPH @DrMakaryFDA and @johnarnold need to be fully investigated for possible collusion in delaying treatment to HD patients. @SenRonJohnson @SenRickScott @RepAuchincloss @adamfeuerstein @l_e_whyte @temple_west @laurencurehd @JRenz0418 @BeckyQuick @MariaBartiromo @docrodwong @LuckyPenguin10 @SECGov @POTUS @LauraLoomer @SenateAging @SenBillCassidy @SenRandPaul @WhiteHouse @SusieWiles @StevenCheung47 @StevenCheung @Scavino47 @PressSec @SecKennedy @MariaBartiromo @FoxNews @bariweiss @60Minutes @CNBC @WSJopinion @WSJ @statnews @BillAckman @Loftus @MartinShkreli

@NiklasAnzinger Makary is trash and a criminal. Needs to be fired.

This is making the rounds, and it's great - but we need to go much further to stay competitive with China: They key is to let states do it, the FDA just needs to create acceptance for state-led programs. - New Hampshire's HB1734 creates the option for private/non-profit/independent scientific review boards (like IRBs) to apply regulatory frameworks for phase-1s or equivalent (like Australia CTN). Federal rules would prohibit actual phase-1/INDs to be conducted this way, but if the FDA just issues guidance to accept such trials this would unleash state innovation. Everyone wins: the FDA already accepts Australia's trials, and this way those can just be brought into the US territory. By doing it through states, no federal law needs change. - Montana's SB 535 and New Hampshire HB 1734 allow for post-phase-1 "right-to-try 2.0" access. The key is that they're a) very broad, any patient qualifies instead of required proof they die in 6 months, and b) the provider has better monetization options. Again, we're already doing that: a) off-label drugs have just passed a phase-1 but not proven efficacy, b) right-to-try is federal policy and the moral case for patients is obvious. This creates a whole alternative pathway post-phase-1 that gives biotechs many more options to innovate. Montana & New Hampshire are creating oversight mechanisms that ensure safety but are administered more efficiently through scientific review boards (which again have oversight by state health departments, i.e. if there are bad actors their licenses can be revoked). Again, everyone wins: through these official state pathways there is less grey market for stem cell clinics and these programs could collect outcome data that improves official INDs for approval by the FDA. These state frameworks, including also e.g. Utah, Florida, Texas are genuinely innovative. @FDA @DrMakaryFDA need to create recognition for state frameworks, so there is clarity about federal-state legal conflicts - otherwise these programs will attract grey markets (larger, credible players need clarity). This is much simpler than federal-level changes, and unleashes decentralized regulatory innovation. @sytses @RuxandraTeslo @cremieuxrecueil @ATabarrok @dr4liberty @zachweinberg @patricksmalone @GraniteBio

@nxtplse @HowieLongggg unfortunately MM has a vendetta against QURE so it's at a stand off until something changes at the FDA

@HowieLongggg It will come back. This is criminal.

I have been building a long position in $QURE. The nimrod at the FDA didn’t even read the data. The technology works, Huntingtons is an ugly disease, one of the worst. This has to succeed.

@LauraLoomer you were right about Vinay Prasad. What about Marty Makary?

I have broken so many stories ahead of the media. I have also been right about every single person who I said should be fired from the admin, and I am right over a year before action is usually taken. Sometimes I feel like my talents are wasted on the outside. There’s so many incompetent people in DC. I wonder if people realize I do the work of 1,000 federal employees. Very frustrating. We have lost so much time.

@BillAckman @X 💯 Fight until the bitter end

I am reaching out to the @X community for advice with the likely risk of sharing TMI. I have been sufficiently upset about the whole matter that I have lost sleep thinking about it and I am hoping that this post will enable me to get this matter off my chest. By way of background, I started a family office called TABLE about 15 years ago and hired a friend who had previously managed a family office, and years earlier, had been my personal accountant. She is someone that I trusted implicitly and consider to be a good person. The office started small, but over the last decade, the number of personnel and the cost of the office grew massively. The growth was entirely on the operational side as the investment team has remained tiny. While my investment portfolio grew substantially, the investments I had made were almost entirely passive and TABLE simply needed to account for them and meet capital calls as they came in. While TABLE purchased additional software and other systems that were supposed to improve productivity, the team kept increasing in size at a rapid rate, and the expenses continued to grow even faster. While I would periodically question the growing expenses and high staff turnover, I stayed uninvolved with the office other than a once-a-year meeting when I briefly reviewed the operations and the financials and determined bonus compensation for the President and the CFO. I spent no time with any of the other employees or the operations. The whole idea behind TABLE was that it would handle everything other than my day job so that I would have more time for my job and my family. Over the last six years, expenses ballooned even further, employee turnover accelerated, and I became concerned that all was not well at TABLE. It was time for me to take a look at what was going on. Nearly four years ago, I recruited my nephew who had recently graduated from Harvard and put him to work at Bremont, a British watchmaker, one of my only active personal investments to figure out the issues at the company and ultimately assist in executing a turnaround. He did a superb job. When he returned from the UK late last year after a few years at Bremont, I asked him to help me figure out what was going on with TABLE. When I explained to TABLE’s president what he would be doing, she became incredibly defensive, which naturally made me more concerned. My nephew went to work by first meeting with each employee to understand their roles at the company and to learn from them what ideas they had on how things could be improved. He got an earful. Our first step in helping to turn around TABLE was a reduction in force including the president and about a third of the team, retaining excellent talent that had been desperate for new leadership. Now here is where I need your advice. All but one of the employees who were terminated acted professionally and were gracious on the way out (excluding the president who had a notice period in her contract, is currently still being paid, and with whom I have not yet had a discussion). The highest compensated terminated employee other than the president, an in-house lawyer (let’s call her Ronda), told us that three months of severance was not enough and demanded two years’ severance despite having worked at the company for only two and one half years. When I learned of Ronda's request for severance, I offered to speak with her to understand what she was thinking, but she refused to do so. A few days ago, we received a threatening letter from a Silicon Valley law firm. In the letter, Ronda’s counsel suggests that her termination is part of longstanding issues of ‘harassment and gender discrimination’ – an interesting claim in light of the fact that Ronda was in charge of workplace compliance – and that her termination was due to: “unlawful, retaliatory, and harmful conduct directed towards her. Both [Ronda] and I [Ronda’s lawyer] have spoken with you about [Ronda’s] view of what a reasonable resolution would include given the circumstances. Thus far, TABLE has refused to provide any substantive response. This letter provides the last opportunity to reach a satisfactory agreement. If we cannot do so, [Ronda] will seek all appropriate relief in a court of competent jurisdiction.” The letter goes on to explain the basis for the “unsafe work environment” claim at TABLE: “In early 2026, Pershing Square’s founder Bill Ackman installed his nephew in an unidentified role at TABLE, Ackman’s family office. [His nephew]—whose only work experience had been for TABLE where he was seconded abroad for the last four years to a UK watch company held by Ackman—began appearing at TABLE’s offices and conducting interviews of employees without a clear explanation of his role or the purposes of these interviews. During this period, he made a series of inappropriate and genderbased [sic] comments to multiple employees that created an unsafe work environment. Among other things, [his nephew] made remarks about female employees’ ages (“Tell me you are nowhere near 40”), physical appearance (“Your body does not look like you have kids”), as well as intrusive questions about family planning and sexual orientation (“Who carried your son? Who will carry your next child?”). These incidents were reported to senior leadership at TABLE and Pershing Square. Rather than being addressed appropriately, the response from senior management reflected, at best, willful blindness to the inappropriateness of [his nephew]’s remarks and, at worst, tacit endorsement.” The above allegations about my nephew had previously been brought to my attention by TABLE’s president when they occurred. When I learned of them, I told the president that I would speak to him directly and encouraged her to arrange for him to get workplace sensitivity training. The president assured me that she would do so. When I spoke to my nephew, he explained what he actually had said and how his actual remarks had been received, not at all as alleged in the legal letter from Ronda’s counsel. I have also spoken to others at the lunch table who confirmed his description of the facts. In any case, he meant no harm, was simply trying to build rapport with other employees, and no one, as far as I understand, was offended. Ironically, Ronda claims in her legal letter that TABLE didn’t take HR compliance seriously, yet Ronda was in charge of HR compliance at TABLE and the person who gave my nephew his workplace sensitivity training after the alleged incidents. In any case, Ronda, as head of compliance, should have kept a record or raised an alarm if indeed there was pervasive harassment or other such problems at the company, and there is no evidence whatsoever that this is true. So why does Ronda believe she can get me to pay her nearly $2 million, i.e., two years of severance, nearly one year of severance for each of her years at the company? Well, here is where some more background would be helpful. Over the last two months, I have been consumed with a major family medical issue – one of my older daughters had a massive brain hemorrhage on February 5th and has since been making progress on her recovery – and I am in the midst of a major transaction for my company which I am executing from a hospital room office next to her . While the latter business matter is publicly known, the details of my daughter’s situation are only known to Ronda because of her role at our family office. Now, let’s get back to the subject at hand. Unfortunately, while New York and many other states have employment-at-will, there has emerged an industry of lawyers who make a living from bringing fake gender, race, LGBTQ and other discrimination employment claims in order to extract larger severance payments for terminated employees, and it needs to stop. The fake claim system succeeds because it costs little to have a lawyer send a threatening letter and nearly all of the lawyers in this field work on contingency so there is no or minimal cash cost to bring a claim. And inevitably, nearly 100% of these claims are settled because the public relations and legal costs of defending them exceed the dollar cost of the settlement. The claims are nearly always settled with a confidentiality agreement where the employee who asserts the fake claims remains anonymous and as a result, there is no reputational cost to bringing false claims. The consequences of this sleazy system (let’s call it ‘the System’) are the increased costs of doing business which is a tax on the economy and society. There are other more serious problems due to the System. Unfortunately, the existence of an industry of plaintiff firms and terminated employees willing to make these claims makes it riskier for companies to hire employees from a protected class, i.e., LGBTQ, seniors, women, people of color etc. because it is that much more reputationally damaging and expensive to be accused of racism, sexism, and/or intolerance for sexual diversity than for firing a white male as juries generally have less sympathy for white males. The System therefore increases the risk of discrimination rather than reducing it, and the people bringing these fake claims are thereby causing enormous harm to the other members of these protected classes. So what happened here? Ronda was vastly overpaid and overqualified for the job that she did at TABLE. She was paid $1.05 million plus benefits last year for her work which was largely comprised of filling out subscription agreements and overseeing an outside law firm on closing passive investments in funds and in private and venture stage companies, some compliance work, and managing the office move from one office to another. She had a very good gig as she was highly paid, only had to go into the office three days a week, and could work from anywhere during the summer. Once my nephew showed up and started to investigate what was going on, she likely concluded that there was a reasonable possibility she would be terminated, as her job was in the too-easy-and-to-good-to-be-true category. The problem was that she was not in a protected class due to her race, age or sexual identity so she had to construct the basis for a claim. While she is female and could in theory bring a gender-based discrimination claim, she reported to the president who is female and to whom she is very close, which makes it difficult for her to bring a harassment claim against her former boss. When my nephew complimented a TABLE employee at lunch about how young she looked – in response to saying she was going to her 40-year-old sister’s birthday party, he said ‘she must be your older sister’ – Ronda immediately reported it to our external HR lawyer. She thereby began building her case. The other problem for Ronda bringing a claim is that she was terminated alongside 30% of other TABLE employees as part of a restructuring so it is very difficult for her to say that she was targeted in her termination or was retaliated against. TABLE is now hiring an external fractional general counsel as that is all the company needs to process the relatively limited amount of legal work we do internally. In short, Ronda was eminently qualified and capable and did her job. She was just too much horsepower for what is largely an administrative legal role so she had to come up with something else to bring a claim. Now Ronda knew I was a good target and it was a good time to bring a claim against me. She also knew that I was under a lot of pressure because on March 4th when Ronda was terminated, my daughter had not yet emerged from consciousness, she was not yet breathing on her own, and my daughter and we were fighting for her life. I was and remain deeply engaged in her recovery while at the same time I was working on finishing the closing for the private placement round for my upcoming IPO. Ronda also knew that publicity about supposed gender discrimination and a “hostile and unsafe work environment” are not things that a CEO of a company about to go public wants to have released into the media. And she may have thought that the nearly $2 million she was asking for would be considered small in the context of the reputational damage a lawsuit could cause, regardless of the fact that two years of severance was an absurd amount for an employee who had only worked at TABLE for 30 months. She also likely considered that I wouldn’t want to embarrass my nephew by dragging him into the klieg lights when her claims emerged publicly. So, in summary, game theory would say that I would certainly settle this case, for why would I risk negative publicity at a time when I was preparing our company to go public and also risk embarrassing my nephew. Notably, she hired a Silicon Valley law firm, rather than a typical NY employment firm. This struck me as interesting as her husband works for one of the most prominent Silicon Valley venture firms whose CEO, I am sure, has no tolerance for these kinds of fake claims that sadly many venture-backed companies also have to deal with. I mention this as I suspect her husband likely has been working with her on the strategy for squeezing me as, in addition to being a computer scientist, he is a game theorist. My only advice for him is to understand more about your opponent before you launch your first move. All of the above said, gender, race, LGBTQ and other such discrimination is a real thing. Many people have been harmed and deserve compensation for this discrimination, and these companies and individuals should be punished for engaging in such behavior. Which brings me to the advice I am seeking from the X community. I am not planning to follow the typical path and settle this ‘claim.’ Rather, I am going to fight this nonsense to the end of the earth in the hope that it inspires other CEOs to do the same so we shut down this despicable behavior that is a large tax on society, employment, and the economy and contributes to workplace discrimination rather than reducing it. Do you agree or disagree that this is the right approach?

@Jebaim3 $QURE nothing compares. PT $250

Best names under 2b market cap? - Insiders with skin in the game - Low debt / Low risk of dilution - Turnarounds at an inflection point Any ideas?

@LogicalValueMx @LuckyPenguin10 The problem is @johnarnold seems to also have influence over UK, and lack of sites in the UK/Europe to administer the therapy. But I agree it will be approved there, but much better outcome if US is first.

@LuckyPenguin10 Prof. Tabrizi presenting data on 2 HD patients who returned to work after medical retirement, to this audience, at this moment? Powerful clinical message. Right audience. Perfect political timing. While the FDA blocks, the UK path is being built in real time. $QURE

The significance of this can't be overstated re: $QURE AMT-130. Punchline → AMT-130 will get approved overseas and the U.S. will continue to fall behind in the biotech race globally. First, some relevant dates: - This presentation: Last week, Mar 30, 2026 - 4y cohort cutoff: Jun 2026 - 4y data readout: ~Sep 2026 Why this presentation is so important: > Dr. Tabrizi obviously, and of course, hasn't seen the 4y dataset yet... > ...BUT, she's a clinician — she sees these patients on an *ongoing* basis. > Dr. Tabrizi would not be speaking w/ this level of conviction if she were seeing deterioration leading into the 4y assessment. > Remember — she's been involved in many HD trials, and she doesn't sugarcoat results. For example, in Mar 2021, she publicly stated that Roche’s P3 tominersen (Gen HD1) did not work. [1] Dr. Tabrizi — one of the world's leading HD researchers — continues to pound the table: "... and two people [from the high-dose AMT-130 cohort] — and this is unheard of — went back to work. Having medically retired, and then gone back to work." [2] This is how the U.S. falls behind. Cc @fjeg1987 @JTLonsdale — @SenGillibrand | @lukaske @SenRickScott | @ClareLattanze @SenRonJohnson | @GraceCarnathan @RepAuchincloss | @GeorginaBurros — #FDA $XBI

@commonnotcommon @BStarcrash34293 @thatdna @peptidemaxxer incorrect. he nailed the quantum short and sold qure before the big drop. now long qure again

@BStarcrash34293 @thatdna @peptidemaxxer He did the same with $QURE

this guy is wrong about peptides/pharma in every way. complete idiot. no wonder he was in prison for fraud

💯 Everyone needs to follow @LuckyPenguin10 $QURE @SenRonJohnson @RonJohnsonWI @RepAuchincloss @adamfeuerstein

@jawnzilla HODL

$QURE I have been out but looking to get back in shortly. Waiting for some other plays with catalysts to run their course. Hopefully I am leveraging gains and not scraps.

@WhiteHouse Fire @DrMakaryFDA he is a disgrace to your party and the American people. Anti innovation. Bribed by @johnarnold who hates Trump anyway

TRADE DEFICIT DOWN 55% 🇺🇸 THANK YOU, MR. TARIFF!

$qure Listen to Sarah Tabrizi explaining AMT-130 and how two patients went back to work. Please share and retweet. This was last week at the Samuel Gee lecture.