Joined December 2020
$qure, $xbi, I believe @WSJ will continue to publish several articles/week jabbing MM and VP with insights from more pro pharma/biotech Susie Wiles and Jim O’ Neill until VP is out and maybe MM as well.
WSJ really does not like VP. we now know why Dr. Pazdur is in favor of AA. Do MM and VP need to develop neurodegenerative diseases before they understand patients’ needs to access innovative medicines like AMT-130 from $qure? wsj.com/opinion/technology-i… via @WSJopinion
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Who has I&I bispecifics in their pipelines? These should be better than combining two.
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$abvx plane stalking. As I said before it is not how deals get done. Reading 2nd, 3rd hand info from BB terminal is not how big pharmas and biotech do deals and M&A. Also tracking hires of $qure lower level without being sensitive to lack of leadership change will not help $qure
One month (or so) on @X , a platform unlike any other. Energy, speed, smarts and stupidity. -The best thing in BioX - Rapid and divergent takes on new clinical data, on balance usually right..wisdom of crowds -The worst thing on BioX - Shameless replies and reposts pumping your own book, praying for M&A about your favorite name, plane stalking…do people actually think this is how a real drug company or serious investor makes decisions? Maybe it just makes you feel like you have some agency, then ok.
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$sln so much celebrations of returning M&A only to have $sln buyout price at its IPO price!
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By the time retail gets ~60% of shares outstanding, $abvx will be in $30-40 range. Keeps buying lower dips!
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Hopefully Dr. Weis gets a meaningful job suits his Ph.D. and ambitions after this WSJ piece!
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$abvx bulls and bears, do you feel the tremor?
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canoebrookbl retweeted
From the @FT: "Cracks are appearing in America’s debt-laden $10tn AI infrastructure boom as project delays, public backlash and shaky investor confidence bring a reckoning for the data centre build-out underpinning the US economy." IMO, what makes all these issues even more interesting is that no matter how they evolve, there are significant economic, financial, social, and geopolitical implications #economy #markets #tech #ai
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Déjà vu but happened 10 years ago, hopefully this time $qure ends up with a real DEAL. Hopefully Roche. The HD community deserves someone with deep pockets and long history of commitment to HD.
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It is extremely hard for the HD community. Now MM, VP, TBH are all gone (with periodic resurfacing via social media) but you have to fight their shadows for a potential gene therapy which is not a $qure yet. Shoutout to Christina
Replying to @mahesh_shenai
@mahesh_shenai, Although we have never met, I addressed Dr. Mallika Mundkur at the @FDA's CNPV public meeting, whom I understand is your wife. After reading nearly 20 of your posts concerning Huntington’s disease (HD) and AMT-130, I have to ask: Are you evaluating the totality of the evidence, or predominantly searching for reasons to discount it? You repeatedly express hope that you will someday treat Huntington’s disease patients. But how much direct experience have you had with our community? Have you spent meaningful time with symptomatic patients across disease stages, witnessed their progressive neurological decline, or spoken with families about their risk tolerance? I ask because I have. HD has devastated three generations of my family. My grandmother died after years of suffering. Her chorea was so severe that she wore padded mittens for nearly four years to prevent her involuntary movements from dislodging her feeding tube. I was nearly my daughter’s age when my grandmother developed symptoms. Now both my daughter and son are watching their grandfather and aunt experience the same disease. My father is in the middle stages of Huntington’s disease, while my aunt is further along in its devastating progression. As a mother, I live with the painful reality that my children may one day witness this same deterioration in me—or, even more heartbreaking, experience it themselves. What I witnessed as a little girl, my children are witnessing today. Different generations, the same devastating cycle we are desperately trying to break. The psychological consequences are equally profound. A fellow Michigander and HD advocate recently lost her father to suicide. Another Michigan chapter board member nearly lost her niece, and several HD friends have lost loved ones to suicide. Suicide risk among people with Huntington’s disease is substantially higher than in the general population. Why? Imagine caring for someone you love while witnessing years of relentless neurological deterioration, all while knowing you could face the same fate. And even if you escape inheriting the mutation, you may carry the lifelong burden of survivor’s guilt as those you love continue to suffer. This is the profound, multigenerational trauma of Huntington’s disease. More than 40,000 Americans live with HD, and over 200,000 are at risk. Those figures do not include the countless caregivers and relatives whose lives are also affected. Your anecdote about a surgeon with another movement disorder reinventing his professional life was particularly revealing. When I read it, I immediately questioned your firsthand understanding of HD. HD does not afford patients the luxury of simply reinventing their lives. It progressively damages the brain circuits responsible for cognition, judgment, behavior, movement, and independence. Symptoms can worsen over 10–25 years, gradually taking away the ability to work, communicate, maintain relationships, swallow, walk, talk, and care for oneself. The ability to reinvent one's life requires precisely the neurological capacities HD progressively destroys. HD takes away the ability to meaningfully participate in life altogether. The 2024 HD Symptoms and Treatment Impact Survey found that nearly three-quarters of respondents would accept treatment risks for five years without progression, while approximately 35% would accept risks for even three years. Three more years raising children. Five more years working, communicating, remembering, and living independently. More birthdays, graduations, and time with family. Then consider the economic consequences of inaction. Over 10–25 years, HD families will face neurologists, psychiatrists, hospitalizations, expensive chorea and psychiatric medications, physical and occupational therapy, speech and swallowing therapy, mobility equipment, home modifications, specialized residential care, and eventually around-the-clock assistance. HD removes two people from the workforce: the patient and the family member who becomes their caregiver. Families lose wages, insurance benefits, retirement savings, and years of financial security. Many conventional care facilities lack the specialized neurological and behavioral resources required for HD, leaving families with limited options. These costs accumulate across households, employers, Medicare, Medicaid, disability programs, and society. When evaluating the risks and costs of treatment, why is the cost of inaction so often treated as though it were zero? If AMT-130 meaningfully preserves independence, the potential economic and societal benefits deserve consideration. You have described yourself as one of approximately 80 U.S. neurosurgeons with expertise relevant to this procedure. If AMT-130 does not meet your personal threshold for clinical adoption, that is your judgment. But your threshold is not the FDA's regulatory standard, nor should it determine the choices available to every informed patient and qualified physician. Other neurosurgeons may recognize the broader significance of this investigational gene therapy. If its clinical benefits are confirmed, AMT-130 could help transform how we approach inherited neurodegenerative disorders beyond HD. Regarding surgical risk, stereotactic neurosurgery has been performed in humans since 1947. Trial experience suggests the procedure has generally been tolerated. But the question is not whether neurosurgery carries risk. It does. The question is whether those risks are acceptable relative to the potential benefit for patients facing a progressive, fatal disease without an approved disease-modifying treatment. Your professional risks and our personal risks may look entirely different. For you, this is a discussion of procedural complications, evidence, and professional responsibility. For us, it is progressive neurological deterioration, dependency, and premature death. The reported AMT-130 findings, including favorable Total Functional Capacity trajectories compared with external controls, deserve serious consideration. TFC measures employment, financial management, domestic responsibilities, daily living, and care requirements. It measures the independence HD progressively takes away. Scientific skepticism is essential, but it must be applied symmetrically. Our community has also been wounded by regulatory rhetoric. @VPrasadMDMPH, your wife’s former boss, recently compared patients pursuing unproven treatments to a drowning person reaching for a blade. Regardless of the intended scientific warning, such language risks reducing informed, deeply personal benefit-risk decisions to acts of desperation, overlooking the devastating consequences patients and families face without treatment. Our willingness to accept uncertainty is not desperation. It reflects a rational understanding of what HD will otherwise take from us. Imagine watching someone you love progressively lose the ability to think, communicate, walk, swallow, or even recognize the people they love most. Now imagine living with the knowledge that the same devastating disease could one day affect you, your children, your siblings, or generations of your extended family. This is the reality of Huntington’s disease. What would a few additional years together mean? What would you do to break the cycle? I am not asking you to abandon scientific standards. I am asking you to recognize that the consequences of waiting are as real as the risks of treatment. The absence of treatment is not the absence of risk. It is continued disease progression, escalating dependency, enormous psychological and economic suffering, and irreversible neurological loss. You may see an investigational neurosurgical procedure that has not met your preferred evidentiary threshold. We see a potential opportunity to preserve the ability to think, communicate, work, love, and remain present in our families' lives. We are asking for scientific judgment that accounts for the full reality of Huntington’s disease. P.S. Go Blue! 〽️💙💛 #HuntingtonsDisease #RareDisease
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Maybe if the shadows are two many, the US government will have to train more neurosurgeons who specialize in clearpoint as some of the shadows may decline to perform HD gene therapy as it is not making them money!
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$qqq us capital back to the US (from Korea and Japan) to support the market a bit so they can get out yesterday, now they really start selling.
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To be fair to the bearish view of $qure, if Matt is still the CEO, I can see the bear case definitely. But 400 pts in 3 Y or less than 150/y is still too low. If that is the case, how could the old BB scaling up inpatient gene therapy that quickly and be profitable under Meek?
Replying to @canoebrookbl
I think they are there throughout, Dr Tabrizi mentioned they find it quite boring. In any case gene therapies are hard to scale, very hard. Total across all about 700 P/Y in the USA. My guess is this would do less than 400 procedures in the first 3 years.
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$rvmd could be another $insm, listen to $insm q1 call and you will understand!
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$rvmd like I posted previously downside at least to low $30B when $mrk walked away, perhaps as long as $100. Again do not underestimate big pharma’s BD, M&A teams!
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I think Meek himself can scale up the entire 700/year for the now rebranded BB which requires extensive hospitalization and has multiple competing drugs/modalities. $qure’s 12 hr outpatient “surgery” small potatoes comparing to the extensive hospitalization needed!
Replying to @canoebrookbl
I think they are there throughout, Dr Tabrizi mentioned they find it quite boring. In any case gene therapies are hard to scale, very hard. Total across all about 700 P/Y in the USA. My guess is this would do less than 400 procedures in the first 3 years.
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$omer BTD. Europe is not relevant as current valuation did not price it in anyway. However it is the part of the “does the drug work” manipulation. It works and already penetrated 42% of the US transplantation centers in its 1st full Q. Probably faster than $rvmd’s PC wonder
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$abvx bulls if you think all big pharma/biotech BD and clinical teams can not read data and only you can, you will be proven wrong.
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It does not require 12 h surgery but to scale up this quickly and be profitable in the 1st/2nd full year launch is impressive. If run rate is >150-200 pt/year early launch in a competitive market w multiple competitors, what would be the peak? Genetix is also very relevant here
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What I did not say is the 12 h surgery is outpatient small potatoes comparing to the extensive hospitalization needed for Genetix’s autologous ex vivo hematopoietic stem cell (HSC) gene therapies. If Meek can make this profitable, 12 hr outpatient is much less challenging
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