ElephantsRKewl

@adamfeuerstein Other than repeat PP injections, Anktiva is a great drug for this indication with durable responses and less likelihood of mutating tumor into more aggressive state as cytotoxic chemo often does.

$IBRX -- The FDA accepted the sBLA to expand Anktiva to include the treatment of MNBIC papillary. The PDUFA date is Jan. 6, 2027. Standard review. A positive development for ImmunityBio, but like all things, details matter. Approval is not a given. In fact, the FDA reiterated its "concerns relating to single-arm trials in papillary disease alone..." ImmunityBio acknowledged. I'd love to read the entire FDA letter, and not just the company's curated excerpt. If ImmunityBio wins approval to expand Anktiva's use based on single-arm study data, the agency will establish a new approval standard that can, and likely will, be used by other companies to seek the same. The same competitive risks that are impacting ImmunityBio and Anktiva in bladder cancer today will remain because $JNJ, for instance, can accelerate expanded approval of Inlexzo.

$QURE’s AMT-130 efficacy claims hinge on a historical control comparator based on “virtual patients” synthesized from the Enroll-HD database. The issue is that this is a horribly flawed comparator as it massively deviates from the placebo CTRL based on the company’s own data!!!

@Christina4HD @TinMarkProd @RetailInve87233 @rachelreising96 Absolutely it all goes back to data and sufficiently powered trials. After the company saw that the therapy wasn’t killing patients, and the low dose group had bad outcomes they should have expanded the hd group treatment count to much more than 17.

“Compensated’ cuts both ways and pretending otherwise is selective storytelling. Arnold Ventures has been pushing stricter approval standards in rare disease and gene therapy. Some FDA leadership also came from Arnold. So let’s spare the ‘only patients & advocates are biased’ framing.

theblaze.com/columns/opinio… Read my second article here💙

@Christina4HD @TinMarkProd @RetailInve87233 @rachelreising96 There are also leading neurologists who are not being compensated who find the data suspect. I am glad there is a review team of PhD and MD scientists at the FDA who can fully evaluate every datapoint. You can try to advocate to get an AdCom.

The study has shown sustained multi-year clinical and biomarker signals that many leading HD neurologists believe are meaningful enough to warrant serious attention. You can debate study design, sample size, and external controls without pretending the data are irrelevant or calling people liars. I’d be happy to connect you with HD neurologists and researchers directly involved in this field.

@libsoftiktok Muslims are transphobic. No wonder trans are Islamophobic.

This is one of the alleged Islamic Center shooters His name is Cain Clark…

@Christina4HD @TinMarkProd @RetailInve87233 @rachelreising96 It hasn’t repeatedly shown anything. That’s just a lie. A small 17 patient study with no blinded placebo control showed a signal at 36 months. relative to a historical database which has not been externally validated. It underperformed true placebo at 12 months. Facts matter.

Medicine rarely advances by jumping straight to perfection. When we spoke to the FDA at the HDSA EL-PFDD meeting in 2024, no one was expecting perfection. In fact, the FDA specifically asked families that if a treatment cannot address every part of the disease, which symptoms and areas of progression matter most to us to improve or preserve. That conversation alone reflects the reality of HD and the urgency families live with every day. Dismissing AMT-130 entirely is unreasonable. If a therapy repeatedly shows slowing across multiple clinical measures over several years in a fatal disease with no approved disease-modifying treatment, that deserves serious attention.

@TinMarkProd @RetailInve87233 @Christina4HD @rachelreising96 No position. But their lobbying effort has been success. If you just wanna make money buy the stock. If you want help for HD patients there needs to be a drug that effectively silences mHTT in all neural cells. And doesn’t silence the normal copy.

@RetailInve87233 @HASurfer297 @Christina4HD @rachelreising96 He’s clearly shorting Uniqure’s stock - probably freaking out right now that Makary and Prasad cocksuckers are gone

@Christina4HD @HuffmanPat56675 @KitCarsonNV The treatment group was only 17 patients. And the main issue is that Enroll HD has been proven to flawed in a rigorous peer reviewed study.

Enroll-HD is the world’s largest HD observational study, with 30,000+ participants and 112,000+ visits collected by HD doctors & researchers across the globe. Randomized placebo-controlled data remain the gold standard. But we cannot ignore the rigor of Enroll-HD and the reality that HD families still have no approved disease-modifying therapy. Also, AMT-130 is the first HD brain-delivered gene therapy trial, not a simple pill study. Early-stage gene therapy trials were intentionally small for safety. The high-dose group was compared against a matched Enroll-HD external control cohort of ~940 patients, not “just 17 people.”

“We don’t approve drugs that require invasive brain surgery” — @HASurfer297 This raises an important question: who is “we” in this context?

@Christina4HD @rachelreising96 Should therapies be approved based on hope and hopeful signals or do you think the onus is on $qure to prove their drug actually works and isn’t harming patients? The issue isn’t that the drug is being investigated, it’s the company making claims based on no placebo data on 17 pt

You are selectively focusing on the low-dose total motor score result & acting like that alone proves AMT-130 is harmful or doesn’t work. Why are you ignoring the high-dose group and the rest of the data? HD families can be hopeful & realistic at the same time. Our families are living with a fatal, inherited brain disease that slowly steals a person’s movement, thinking, independence, & eventually their life. Children & families live for decades under the fear of inheriting it themselves. There is still no approved disease-modifying therapy for Huntington’s disease. Any treatment showing signs of slowing progression deserves serious consideration, because time matters to families living this nightmare every single day. It does a disservice to the HD community to present incomplete data as settled fact while minimizing the urgency & reality families are forced to live with every day.

@HuffmanPat56675 @Christina4HD @KitCarsonNV Has there been any external audit of Enroll HD data? Regardless you need more than 17 patients to study a drugs effect. There are literally tens of thousands of HD patients. Why didn’t the company enroll more?

@Christina4HD interesting question. @KitCarsonNV your assumption is intriguing. However @HASurfer297 is missing a basic understanding of pharmacy knowledge. I’m a retired RPh who tries really hard to keep up. I would be really concerned to know if @HASurfer297 is a medical professional with a scalpel. He won’t respond because he didn’t the last time I asked. My questions/comments. Is AMT130 a drug? It does not have the characteristics of a drug with regard to pharmacology. The pharmacodynamic, pharmacokinetics, half life, area under the curve. Gene therapy is advanced science and cannot be compared to “what we’ve always done” drug evaluation. Low dose vs high dose data is important. Different again from a drug (small molecule) . Introducing genetic material is not the same as 325 mg vs 500 mg. I believe the high dose refers to the amount of microRNA packed into the viral vector. Viral vector is stripped of its disease causing characteristics. It can cause immune reaction but not illness from the virus itself. Again advanced science I am not an expert just someone wanting to learn. If all this is true wouldn’t logic suggest that low dose may not integrate to the same degrees as high dose. Additionally, if immune response occurs and is stabilized by way of corticosteroids then we have wait to see the results. How long does it take for transcription to occur? This is very advanced and cannot be simplified by saying the therapy doesn’t work because of the data at 12 months. This requires seeing the big picture. Enroll HD is true data. A person signs all of the same consent forms as any interventional clinical trial. Our information is de- identified. This data is a valid comparison. My thoughts on objection to this therapy. If someone is looking at risk vs reward in a dollar and cents evaluation the therapy will be rejected. But humans can’t be evaluated as one is more valuable than another. That is a future too dismal to imagine.

@RoxannPoIk @nickshirleyy Your fat ass would def leave some elephant size footprints lol

@nickshirleyy No footprints in the snow? Lol you are an idiot! Why don’t you go and harass Trump about all the money HE and his family has stole from the American people? You look stupid!

Here’s your daily reminder that your tax dollars are going to men like this to run “daycares” who can’t even answer a basic question about the $2,250,000 they receive from our tax dollars “F*cking million dollars, don’t worry about it!” ARREST ALL THE FRAUDSTERS

@Christina4HD @rachelreising96 Clearly you haven’t seen the entire dataset and are relying on PRs from a for profit company. None of which is peer reviewed. It’s okay to be hopeful, but also be reasonable. And again why was there accelerated decline in LD group?

The 4-year data is expected this year, and based on commentary from neurologists and researchers who actually specialize in Huntington’s disease, the signal appears to be strengthening over time, not weakening. You framed this as “we don’t approve invasive surgeries,” but who exactly is “we”? Are you an FDA reviewer? If you’re going to speak as an expert while discouraging experimental therapies for a fatal disease, it’s reasonable to ask that you be transparent about your qualifications, affiliations, and any potential conflicts of interest. And regarding invasiveness, if a procedure had the potential to slow or alter the course of Huntington’s disease, why would “invasive” automatically disqualify it? HD is one of the cruelest neurodegenerative diseases imaginable. People progressively lose the ability to think, work, speak, eat, walk, and care for themselves, often over many years before death. Families facing that reality are willing to consider serious interventions because the alternative is relentless decline with no approved disease modifying therapies currently available. No one is saying experimental therapies should bypass scientific scrutiny. They absolutely need rigorous evaluation. But presenting incomplete data while dismissing hopeful signals outright does a disservice to the HD community and to honest discussion.

@Christina4HD @rachelreising96 You are correct in that it’s an experimental therapy. And it absolutely needs more investigation. We don’t approve drugs that require invasive brain surgery and show inferiority to placebo at 12 months and then claim they work based on a ghost virtual control group.

You're only posting the low-dose cohort while ignoring the high-dose data entirely. That's not an objective analysis. High-dose group has shown stronger signals across multiple endpoints over time, including functional measures & biomarkers. If you're going to state a therapy "does not work," at least present the complete dataset. Huntingtons disease -> a fatal neurodegenerative disorder with no approved disease modifying therapies. Families living this reality deserve better than cherry-picked commentary from someone who appears more interested in dismissing progress than discussing the science honestly. Also, what are your credentials, affiliations, and conflicts of interest? Why are you focused on discouraging experimental HD therapies patients are fighting for??

@JackDangerLIVE They let so many in that the constituency radically changed in 30 years. Saying whatever it takes to win elections is nothing new. They let the problem get OOC. Cat is totally out of the bag and demanding welfare and/or defrauding social safety nets.

Dad, what were Democrats like in the 1990s? Volume up 🎧

@FundamentEdge This is excellent news. Now tell those finance PhDs to pickup a hammer and do the Collar-free work that needs to be done like building houses and cleaning up parks and streets.

A big pivot from Ken Griffin on AI: “Number one is, in the last few months, there has been a step change in the productivity of the AI toolkit. It is profoundly more powerful than it was just nine months ago. And for us at Citadel, that has allowed us to unleash a much broader array of use cases for AI. And it has been really interesting to watch, to be blunt, work that we would usually do with people with masters and PhDs in finance over the course of weeks or months being done by AI agents over the course of hours or days. These are not these are not mid-tier white collar jobs. These are like extraordinarily high skilled jobs being, I'm going to pick a word, automated by agentic AI. And I gotta tell you, I went home one Friday actually fairly depressed by this because you could just see how this was going to have such a dramatic impact on society. When you witness it in your own four walls, when you see work that used to be man years of work being done in days or weeks, it's like, wow, like that's the first time I've seen real impact in our four walls.” This echoes my own experience with agents and the conversations I am having with students, friends & clients. The toolkit has dramatically transformed and it feels like in finance, for the first time, AI is real.

@docrodwong Yes! Let's approve drugs based on iffy placebo-free n=17 datasets and make investors rich, leave patients with unproven drugs and say fuck it to Medicare solvency!!!

in addition to makary and prasad, without waiting for new leadership to decide, the administration went ahead and showed the rest of the leadership team associated with arnold ventures the door. this sends a strong message this administration is serious about innovation, cares about rare disease patients, and won't let american biotech lose its leadership position in the world.

@Christina4HD @rachelreising96 AMT-130 accelerated decline of Total Motor Score in the low-dose group at 36 months. This is one of the more objective measures. AMT-130 likely does not work and causes harm from invasive surgery/injecting the brain with a virus. It's noisy low n data and needs more study.

How many times do HD families have to explain this to you? You’re cherry-picking an early 12-month snapshot while ignoring the longer-term follow-up data showing slowing of progression in a fatal neurodegenerative disease. Yes, some patients initially worsened after invasive brain surgery, and that is not unexpected in CNS gene therapy. What matters is where they trended afterward. Even some low-dose participants improved beyond 12 months. Also, pretending the FDA has never exercised flexibility on smaller or imperfect datasets before is simply false. The difference here is that Huntington’s families are the ones begging for urgency while facing guaranteed decline and death. So what exactly do you have to gain by constantly trying to roadblock a rare disease community seeking access to potential treatment options? Patients and families deserve the right to evaluate risk, consider the data in full context, and advocate for their own future. We will not have those decisions dismissed by outside voices with no stake in the outcome.

@RighttoTryGuy @US_FDA I imagine that Texas is probably the most self-sufficient freedom-loving state for those kinds of opportunities. I think the law needs to be improved.

@HASurfer297 @US_FDA Just setting up a facility like that would likely take years and tens of millions of dollars. And even if we manufactured it in Montana, if even one molecule comes from outside Montana then interstate commerce is triggered and FDA reserves the right to shut them down.

@Christina4HD @rachelreising96 I understand you are hopeful and HD sucks. I wish you the best of luck, but there is evidence of harm and lack of activity. The appropriate mechanism is an EAP for desperate patients while trials continue. Need more patients to better understand if the drug works or causes harm.