DesertDweIIer_RAOR_4_A_CERU_4_DH

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DesertDweIIer_RAOR_4_A_CERU_4_DH

DesertDweIIer_RAOR_4_A_CERU_4_DH

@datIkhan

Katılım Nisan 2011
23 Takip Edilen26 Takipçiler
DesertDweller_ROAR_4_A_CURE_4_HD
DesertDweller_ROAR_4_A_CURE_4_HD@DesertDweller93·
For those who haven't seen it, here is last night's episode of @BeckyQuick CNBC Cures. A story of hope, determination and anguish for those suffering rare diseases. Hopefully a future episode will feature the travesty surrounding @uniQure_NV $QURE AMT130 and the needless delays that are 100% the fault of @DrMakaryFDA @VPrasadMDMPH who are killing people by their actions. They must go and be prosecuted. cnbc.com/video/2026/03/… @SenRonJohnson @SenRickScott @RepAuchincloss @adamfeuerstein @l_e_whyte @laurencurehd
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DesertDweller_ROAR_4_A_CURE_4_HD
DesertDweller_ROAR_4_A_CURE_4_HD@DesertDweller93·
Last time I updated my pinned tweet was after $QURE released the incredible data on 09/24/25. Considering the huge disappointment that we all suffered, most of all the HD patients on 11/04/25, I felt I should update my feelings towards QURE as an investment and resulting treatment for those in the HD Community. Remember, I am an investor and retired CPA by profession, not a scientist. Take my opinions with that in mind. There are far more knowledgeable people on X and elsewhere concerning the science behind AMT130. Personally, I trust what QURE management and KOL's stated about the science behind the numbers after following them for 10+ years. These views are my own and make sure you perform your own due diligence and don’t blindly follow anyone. Don’t risk what you can’t afford to lose. Everyone is wondering where QURE goes from here. I can’t say for sure and my predictions could be totally wrong. I have been invested in this company for 10+ years and thankfully, it began paying off as I had hoped on 09/24/2025. Sadly, we know what happened on 11/04/25 with the resulting reduction in stock price from high $60’s to now under $24. My individual buys and sales over the past 10 years, at current prices, still leave me with QURE being my #1 investment return of all time. No doubt, I am lucky to be sitting in this position. I am a complete fool risking as much as I do in a single stock but luckily for me, it is still working out, even though it went horribly wrong and unexpected in November. I firmly believe the future gains will dwarf my past returns in the coming months and year or more. QURE is my largest holding and I currently have more shares than I ever had. I had sold some in September but bought those back on the resulting drop after November. I plan on reducing my exposure by 10-20% in the near future if things work out like I believe, then hold on to the rest until even higher prices as the story plays out. I will then sell more shares along the way as it rises to new all-time highs that I expect within the next few months then hold the majority of my shares until the ultimate payoff which I believe will be a sale of the company in 1-2 years, slightly longer than my previous expectations of a sale in mid-2026. Why do I feel this way? Nothing has changed with QURE data and prospects as a result of the data they released in September, other than an unexpected change in how the FDA currently views the data which caused a delay in my timeline expectations. The data are still incredible and the only treatment to date that has shown a 75% slowing of disease progression 3 years after treatment. All other treatment data from the many other clinical trials show changes in surrogate endpoints, not clinical endpoints as is the case with uniQure. These other trials measure mHTT in the spinal fluid and blood and assume if it goes down, it will result in efficacy. Roche has shown that may not be the case. I believe most if not all the trials will not show efficacy on clinical endpoints. I hope for the HD community that I am wrong on this. AMT130 is delivered directly where it needs to go, in the deep portion of the brain to halt/slow progression. No way to see reductions in mHTT in the brain without brain biopsy and that isn’t going to happen. Since this portion of the brain only makes up ~1% of total brain volume, the reductions in mHTT in the striatum are not indicative of what is happening in the blood and spinal fluid. AMT130 has 3 years of clinical data showing patients in hi dose cohort fare better than historical comparison. This is a huge distinction between QURE and its competitors. We have the best management in a small biotech that I have ever invested in. They are cautious when it comes to cash position and has been so forever. They have raised money when they could, not when they need to like so many failed biotechs. Scrapping programs early when data became available to do so instead of continuing to fund programs that were not going to succeed. Implementing several restructurings over the past 2 years including the sale of their manufacturing facilities to streamline operations and reduce cash burn even before they had to. I firmly believe this was in order to set the company up for a sale, only time will tell if I am correct on this prediction. These are just a few of the things I’ve witnessed and why I believe they are the best management of any company I have invested in. uniQure’s approach to HD treatment is different than others which sets them apart from their competitors. As stated by Ignacio Muñoz Sanjuán who has been involved in the AMT130 program from the start in a recent publication, uniQure’s approach is different and historic. valenciaplaza.com/revistaplaza/i… Upcoming catalysts: POSSIBLE reversal of the reversal from FDA for AMT130 program. The November 3rd announcement caught me by complete surprise. I did not have that on my bingo card for 2025 after all of the positive commentary over the years about alignment with the FDA and granting of FTD, BTD, RMAT designations for the program. We have no clue as to why the FDA changed its opinion on alignment, though many people write opinions as to the WHY, these opinions are just that, an opinion. The company has never publicly stated the WHY. Just as surprising as the November PR was, we could get another PR any day with a reversal from the FDA not only allowing a BLA filing but encouraging one. The Huntington community has mobilized by contacting Senators, Congressional representatives, the FDA, the media and anyone else who could exert pressure on the FDA to reverse their decision. We know the company has requested a follow-up meeting with the FDA to discuss a possible path forward. I expect a positive outcome from this meeting. Completion of cohort 4. The company announced in July they added a 4th cohort to treat 6 additional patients in order to expand potential patient population. When trial was originally designed, in an abundance of caution, they screened out patients who didn’t have enough striatal volume to receive the therapy without increased risk of side effects. With the knowledge gained, along with cohort 3 where they added steroids and immunosuppressants to reduce brain swelling, it makes perfect sense ahead of BLA filing to demonstrate that you can give it to more of those suffering. Another example of this management team being so forward-thinking. On November 10, 2025 they announced that the cohort was fully enrolled during October. Since this cohort was added for safety reasons and any adverse events occur early after treatment, I expect an update in early 2026. My guess is that most, if not all, patients have been treated by now. Since we haven’t heard anything new about any limiting AE’s on patients treated so far, I expect it to show no new AE’s and this opens up potential population available to be treated. While I don’t believe this will have a huge market impact initially, it is a vital step forward for commercialization. 3 other clinical trial programs. QURE has guided the first half of 2026 for updates on TLE, Fabry and SOD1 ALS programs. We already have early data on the TLE and Fabry programs. The TLE update is the one I am looking forward to most due to its potential market opportunity, even larger than Huntington’s. Administration could basically be done on an outpatient basis where patient goes into hospital in the morning, gets the treatment and goes home same day, or at worst the next day. Current plan is immunosuppressant will not be required unlike a cell therapy currently further along than QURE in clinical trials but with potential lifelong immunosuppressants needed. The first patient treated and reported on in uniQure’s program had a 92% reduction in seizures in the months following treatment and more importantly, zero seizures the last 2 months after treatment. This could be huge. FDA approval. I still expect FDA approval by the 4thquarter of 2026. Approval on its own will be huge but if somehow there is a broad label, the impact will be even bigger. The roadblock will be caused by the payers but that will be overcome. With zero other treatment options available, the payers will need to pay for treatment. EU update. The company recognizes the importance of moving this along ASAP and will act accordingly. With the FDA surprise, they stated they are looking at other market opportunities to advance AMT130 in UK and EU. I still view the US FDA approval as being the quickest path to commercialization but we shouldn’t overlook these other markets. Hopefully we get an update during the 1st quarter of 2026 but more likely during the 2nd quarter. Potential revenue updates: During the September update, the company stated that right now there are 68 sites that could administer AMT130, that is in only the US; what about rest of world? As I have complained about, I believe the analysts are woefully underestimating peak sales in their reports. AMT130 will be the most successful commercial gene therapy within a year or two of approval is my expectation. The demand will be tremendous after approval. The HD community will be very vocal and demand coverage from the payers and will mobilize for the government to figure out a way to get this critical treatment out to patients. This will be a big deal and its impact is underestimated. While the FDA definitely slowed down the approval pathway, I believe the approval will happen and then the analysts will be revising their targets accordingly. Education is needed. One of the keys to success is educating the FDA, the public, healthcare community and payers about the treatment itself. So many talk about how it is “invasive brain surgery” but don’t really understand it. Yes, it is administered deep in the brain but describing it as invasive is not entirely accurate as it is administered by use of a tiny catheter using robotic type surgery. While the patient is under anesthesia for 12 hours or so, the procedure itself is not all that complicated. This will be overcome in time. Price targets. I honestly don’t know how high QURE can get. The more I think about potential positive scenarios, the more insane my targets become. With only 70 million fully diluted shares outstanding, I can justify eventual target prices in the many hundreds of dollars with the right sequence of events. That may sound crazy to those who don’t understand QURE and instead rely on the fact that it had a low of $5/share during the past year or so, but what is a reasonable market cap for a company with QURE’s potential revenue? Looking at just AMT130, on a worldwide basis, I think a reasonably good number that will be treated annually is about 5,000/year for as long as the eye can see. Should this be priced at 1X revenue? 2X revenue? 5X revenue? Remember the treatment will be priced similar to other gene therapies which currently are priced $2 million - $4.5 million per treatment. Using just $2.5 million, annual revenue based on 5,000 patients annually gets you to $12.5 Billion in annual revenue just from AMT130. What happens if AMT260 becomes the standard of care for TLE? You can double/triple that revenue projection. Are there risks involved with these projections? Sure but as I’ve been saying, I believe big pharma will be buying QURE within the next year or two so those revenue risks will be on the acquirer, not current QURE investors. If an acquirer paid only 1X my revenue projection, that translates into $175/share. 1X revenue is ridiculously low and is why I said earlier, I can come up with price targets into the many hundreds of dollars/share. Matt and the board are not stupid and are not going to give away the company. When the time and offer are right, I believe we will see the announcement of a sale. Best part is, if there isn’t a high enough offer, they are positioning themselves to go it alone if they have to. Good luck to all investors and the HD community. I hope my current thesis proves accurate but do your own due diligence and risk what you can afford. Before investing, make sure you understand the risk factors involved in UniQure which could pop up unexpectedly as evidenced by the unforeseen FDA reversal in November. Every investment carries risk, don’t ignore them unless you are nuts like me. Here is a link to my prior analysis drafted after the initial data release in September: x.com/DesertDweller9…
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DesertDweIIer_RAOR_4_A_CERU_4_DH
Authentic Mexican Street Tacos Alert! Crispy corn tortillas, tender al pastor pork, fresh cilantro + a squeeze of lime = flavor explosion in every bite. Who’s grabbing a plate
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Arlei Soder
Arlei Soder@rhbaker1·
"When you thought you nailed the presentation… until your slideshow accidentally switched to your weekend dog-walking album. Colleagues now request ‘puppy interludes’ in all meetings. #OfficeChaos #WinningByAccident"
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DesertDweIIer_RAOR_4_A_CERU_4_DH
Tired of scrolling Pick 1 tiny hobby today—draw a doodle, grow a succulent, or learn 2 guitar chords. Small steps = big joy.
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Siadn
Siadn@faye51626038·
Catch tonight’s Twilight Cabaret at 8PM! Soulful vocals + jazz twists—don’t miss the magic. Grab last-minute tix via link in bio! #LiveTheater #CabaretNight
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DesertDweIIer_RAOR_4_A_CERU_4_DH
" 3 Quick Green Tips! 1. Water only when soil is dry (stick finger 2cm deep!). 2. Rotate plants weekly for even light. 3. Wipe leaves to boost photosynthesis. Keep your greens happy! "
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本杰眀乌萨奇.
本杰眀乌萨奇.@MurielleLambert·
"Morning sync → deep work → team check-in. Grateful for small wins (and good coffee ) today! #WorkLife #DailyVibe"
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Make GcId Garet
Make GcId Garet@ManziLoL·
[Dined at Sunny Side Bistro Tried their signature honey-glazed salmon + truffle mashed potatoes—flaky fish, buttery mash, chef’s kiss! Cozy vibe, quick service. 10/10, will revisit! #FoodieFind #LocalEats],
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DesertDweIIer_RAOR_4_A_CERU_4_DH
Catch the live show tonight—lights, energy, and unforgettable moments! Grab your tickets (link in bio) and join the fun!
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Petei DjCerIo
Petei DjCerIo@jentorres7·
KTV Night Alert! Grab your crew, belt out your faves, and let the good vibes roll—no stage fright, just pure fun! #KTVVibes #SingYourHeartOut
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