Mathias Bigger

I agree that this is the smartest approach for evaluating the impact of a drug on the American people. It’s important that along with stakeholders, the patients, their families and the advocacy groups that support them can make informed commentary in the open. Their perspective acts as data points to determine the state of a treatment deprived community and the risk and urgency required in a situation. $QURE and the Huntington’s disease community stood strong, signed a petition 40k times and used X to openly challenge the decision the fda made. This dialogue is extremely valuable.

Feels like the FDA is slowly healing itself.

The FDA commissioner needs to be someone who understands that clinical biology is an approximate science, and when it comes to rare diseases any sign of efficacy needs to be nurtured and explored not thwarted and roadblocked. Based off of Houman’s tweets I think he’s an excellent candidate!

Totally agree Desert. Makary goes on media discussing his incredible progress at the FDA using accelerated pathways, data, and AI, then turns around and recommends a sham control when the comparative data already exists. For heavens sake the fda aligned on it in April 2025, Makary reneged. He does not have the interest of the patients in mind.

You got it wrong again. I was not comparing liver transplant to @uniQure_NV $QURE AMT130 only how tough a RCT would be in this population is and if you would risk your daughter's life in a similar situation. I'll make it simple for you; would you have taken a risk of a 12-hour anesthesia and ventilator for a procedure for your daughter if you had a 50/50 chance of her getting nothing other than a death sentence and potential risk from the anesthesia itself? Not to mention that during the 3 years of the sham portion of the trial where you received nothing other than 12 hours of anesthesia during the procedure, medical follow ups including invasive spinal taps, continuous loss of brain cells that will never come back, and the inability to take part in any other trial for a 100% fatal, hideous disease. @DrMakaryFDA, who is a media whore, whenever he got the chance to talk, talked about real world evidence and how the FDA would use other measurements to determine efficacy for rare disease indications. There is no other database as comprehensive for a rare disease as the cUHDRS database and yet he wants patients to undergo a 12-hour sham procedure that carries immense risks. I am not wrong on what the FDA is asking for. The original sham controlled portion had patients undergo 12+ hours of anesthesia, ventilator and drilling into the scalp. The drill holes did not go all the way in but they had drills in their head so they would think they got the treatment. This very likely contributed to a strong placebo effect for those patients when compared to the treated patients at ONLY 12 months when they were allowed to cross over. 4 of the 10 patients crossed over but the other 6 were no longer eligible for the treatment due to the progression of their disease and they no longer met the original screening criteria. DON'T tell me I was wrong on the trial design. You are playing it down as if it is a "nick in the scalp" while downplaying the 12-HOURS OF ANESTHESIA and VENTILATOR. You are being so disingenuous and you are ill informed. I post my profile so people understand who I am and I am fully transparent. I have no issue with people questioning me or AMT130 results from an informed perspective, not one based on supporting some very unqualified professionals screwing the HD community at the FDA. You say "What if 30% had blood clots..." Why ask a question for something that DID NOT HAPPEN during the trial as support for why the FDA should continue to require more patients to die needlessly in a future sham portion all in the name of science when they have data that allows a direct comparison for efficacy. I have followed uniQure for over a decade and read and listened to every trial result over that timeframe. I've spoken to HD advocates who tell me of the patients that they know personally who have gotten the treatment. I've listened to experts IN THE FIELD, not some medical expert with almost zero knowledge of this particular debilitating disease and the trial. Sarah Tabrizi knows of at least 2 HD patients who received the high dose who were medically disabled who returned to work after treatment. She said she has NEVER SEEN this before in the HD community. The prior FDA had not 1, not 2 but 3 Type B meetings and after ALL 3, agreed upon accelerated filing of a BLA in the first quarter of 2026. @VPrasadMDMPH decided he knew better and pulled that alignment only after officials from @johnarnold Arnold Ventures met with FDA officials to discuss "Items of Mutual Interest". What does a hedge fund manager have in common with FDA interests? Also, Makary lied when questioned about this on CNBC and said uniQure never had alignment which is a blatant lied disproven by the minutes of those meetings. Based on Prasad's public comments, even though he idiotically tried to hide behind "senior FDA official" only to give himself up, he blatantly lied about the sham procedure itself like you are doing by misrepresenting what it takes. You continue to support these bought and paid for monsters for what purpose? I make it clear, don't trust me, trust the experts in the field and you are quite clearly NOT ONE OF THEM. Sarah Tabrizi is and below are comments made by her describing the procedure on 3/30/26. I realize after posting my transcript of Sarah that I didn't provide a link to her discussion so here it is if you want a chance of listening to an expert, not a podcaster and media whore. player.rcp.ac.uk/ee/live/samuel…

Crazy bio investor is comparing liver transplant to AMT-130 for HD. Thats a terrible comparison. A better one in the transplant story is the FDA requirement for an RCT for a novel immunosuppressant called tacrolimus at a time Starzl had single arm data showing tacrolimus rescuing organs rejecting with older immunosuppressant regimens. The FDA got that wrong, I know because I wrote on it (the essay was titled randomized trialomania) But that wasn’t some benefit measured against some manufactured historical control ! And desert dweller is also wrong on what sham the fda is asking for. Again, I detail this in my daily caller piece.. the first sham was a Nick in the scalp , with partial burrholes. There are risks to being under anesthesia - but if 30% of patients had a blood clot , how would we know if this was the therapy or related to being immobile for that length of time? A word of advice ; Don’t get medical advice from ppl with $anything in their post or profile.

1. Put time pressure on $BEAT regulatory agent who then rejected their 12 lead ecg algorithm, they appealed and two weeks later it got approved 2. Claims that $QURE AMT 130 data is not enough to support approval, proceeds to go on cnbc and announce npmi and bash AMT130 saying it does not work. If the data wasn’t powered to meet his standards how can he then say the drug is ineffective? This slowed down AMT130 path to patients - costing them valuable time. 3. Other cases of pressuring his scientists to issue CRL and is forced to then review those very CRL due to outcry. 4. Introduced so much risk to biotech investing because the word of the FDA lost integrity. The companies who need cash to fund a trial based on the confidence of the fda alignment are struggling to raise money to fund trials.

@BiggerMathias What did he do that did not have the lives of patients in mind?

Did not have the lives of patients in mind, show him the door. Prasad and Makary will be giving tutoring lessons next fall. $QURE $CLPT wsj.com/health/healthc…

@idalopirdine @houmanhemmati

Who replaces Makary?

President Trump just demonstrated something important. When he wants the FDA to act, the FDA acts. Per WSJ this week, he pressured Makary directly on flavored vapes and the agency reversed. Huntington's families have been waiting six months for that same intervention. AMT-130 works. The data is overwhelming. One conversation could change everything. $QURE #AMT130 wsj.com/politics/polic…

I supported his nomination because he spoke truth during COVID. He immediately fired the best leaders at the FDA, replaced them with anti-Trump leftists who hollowed out FDA, harmed patients, stifled innovation & drove bio-tech to China then lied about it. wsj.com/opinion/presid…

This is the smoking gun. A career FDA reviewer resigned over political pressure to block a rare disease drug, named Prasad directly, and said the review document supported approval. The HD community has been screaming this for 6 months. Now it’s on the Congressional record. #AMT130 $QURE

#ClearPoint Neuro Announces FDA Clearance of the Velocity Alpha(R) MR High Speed Surgical Drill System and First Clinical Use, Further Expanding Our Drug Delivery Ecosystem and Global Footprint Read More Here: ir.clearpointneuro.com/news-events/pr… #CLPT

Holy bullish for $CLPT - partners are undervalued, they are undervalued^2

Massive win for $CLPT! UCB just acquired Neurona Therapeutics for up to $1.15B to accelerate regenerative cell therapy for drug-resistant epilepsy. Every dose of NRTX-1001 is delivered via $CLPT. This is the kind of validation that moves the needle hard. Full speed ahead 🚀

If you hate billionaires, go all the way, pussy. Ditch the iPhone, the laptop, the tablet. Get off the internet. No Amazon, no big box stores, no Starbucks. Grow your own food. Thrift your clothes. Walk everywhere. And obviously no working for, buying from, or interacting with any company that scales beyond a farmers market. Enjoy your abundance.

@SnackBioTech @biggercapital Someone needs to change his diaper

Jealous of what? I suppose you could show great returns for @biggercapital but then you’d have to explain why he benefitted when the companies and retail investors haven’t. Jealous of a kid with 1 co-authorship getting a job at a fund? But oops then you admit you’re nothing on your own. This isn’t hard. You’re a nepo hire into a vulture fund who can only say they’re too dumb to realize why their original bull thesis was wrong or admit they don’t care and they just wanted to take advantage of retail liquidity. And they’re hoping that retail is too dumb to see why the $BIRD brag is ridiculous. Just like $CLPT $PLUG and $NWBO

For those too slow to follow along @BiggerMathias, the bull thesis on $BIRD died today. They are no longer a shoe company. Michael didn’t call anything. And it’s not pocket watching to point out that taking a victory lap on a losing trade is silly. Unless you only have losing trades I guess.

@SnackBioTech @biggercapital Here’s the little guy again. Pocket watching others with no accomplishments to show for himself.

@biggercapital Brother, you’re up $4 wtf are you talking about. That loses to any and all indexes. $BIRD

$BIRD

If you are an FDA employee under pressure from the commissioner’s incompetent leadership, I want to hear from you. To submit a disclosure to my office, please email Auchincloss.Oversight@mail.house.gov

all because this douchebag @johnarnold doesn't think rare disease patients' lives are worth anything easy to say until it is your family member who has DMD, SMA, CF, HD, etc.