BIOTECH man LONGTERM FOCUS many positions-also trade around my CORE holdings my opinion only do your own research with me

Joined September 2015
Wonderful Day for me. - celebrating 25 GREAT years with my WIFE & best friend today. Best decision I ever made - happy #anniversary to my WIFE ❤️❤️
Tomorrow 23 year anniversary +8 years of dating -like @SheffStation I started young 😃 MY WIFE best #LONGTERM Decisions I ever made ❤️❤️❤️ xitter.cf/tgtxdough/status/78728…
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$TGTX older article by cowen on Azer Cel TG Therapeutics Entered Cell Therapy Race With Azer-Cel, An "Off-The-Shelf" CD- 19-Directed CAR-T Candidate In January 2024, TG Therapeutics entered into a partnership with Precision Biosciences to license azercabtagene zapreleucel (azer-cel), a CD19-directed allogeneic CAR-T cell therapy program, for autoimmune diseases and all other non- oncology indications. Under the agreement, TG will receive exclusive worldwide rights to develop and commercialize azer-cel in non-oncology indications in exchange for upfront and potential near-term economics to Precision valued at $17.5MM. TG will also provide up to $288MM in additional payments based on the achievement of certain clinical, regulatory, and commercial milestones, in addition to high-single-digit to low double-digit royalties on net sales. Azer-cel is engineered to overcome manufacturing challenges associated with autologous CAR-T by leveraging an allogenic ("off the shelf") approach. Unlike auto CAR-Ts, which involve modifying the patient's own cells, azer-cel is produced from donor-derived T cells that have been modified via gene editing technology, which would allow for broader scalability, simplified logistics, and reduced costs. The company believes azer-cel has potential to be a one-time treatment for MS, largely due to its CD19-directed approach, which achieves broader coverage of the B-cell compartment compared to CD20 and thus confers deeper and more durable B-cell depletion. Conversely, the company notes CD20-directed approaches are better suited for repeat treatment in MS. TG Therapeutics received IND clearance in August 2024 for a Phase I trial focused on progressive MS patients. Trial results are anticipated by YE 2026. Experts Need To See Three To Five Years Of Remission With CAR-T's To Justify Use In MS Patients surveyed physicians and payors on the minimum duration of treatment-free remission from treatment with CAR-T therapies that would justify their use in the MS setting. Physicians and payors agreed on a weighted average of approximately 3 years. Our academic KOL agreed with this threshold to justify the high cost of CAR-T treatment.TD Cowen Global Research September 28, 2026 Our academic KOL believes that CAR-T will be safer in the MS setting relative to hematological malignancies as a consequence of the smaller magnitude of cell destruction. While the concept of resetting a patient's B-cells through CAR-T therapy could certainly be effective, the KOL note that the same strategy could theoretically be achieved with anti-CD20's. Along these lines, he cited an ongoing study out of the University of Pennsylvania that is randomizing patients going on Ocrevus to either stay on through the whole four years of the study or to stop therapy after two years. This study will read out well before well before CAR-T therapies could reasonably reach the MS market, which he estimates is at least eight years away.
$TGTX older UBS report on $KYTX & market opportunity for CAR T therapies in autoimmune diseases? addressable market for CAR T in autoimmune as fairly broad with the potential for multiple efforts on goal given its wide applicability across the B cell- mediated disease spectrum, however we highlight in particular: ˜ Summary of market opportunities as of US prevalence by indication (UBSe): SLE (300-400k)/LN (~150k), SSc (~85k), ~myositis (~90k)/DM+IMNM+JM +anti-synthetase syndrome (~64k), gMG (~93k), MS, PPMS+SPMS (~170k), SPS (less than 1k). ˜ Unmet need in lupus nephritis (LN) driven by significant rates of morbidity and mortality; broader systemic lupus erythematosus (SLE) market represents potential upside, but risk/benefit for CAR Ts less clear in patients without renal involvement.
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$TGTX older UBS report on $KYTX & market opportunity for CAR T therapies in autoimmune diseases? addressable market for CAR T in autoimmune as fairly broad with the potential for multiple efforts on goal given its wide applicability across the B cell- mediated disease spectrum, however we highlight in particular: ˜ Summary of market opportunities as of US prevalence by indication (UBSe): SLE (300-400k)/LN (~150k), SSc (~85k), ~myositis (~90k)/DM+IMNM+JM +anti-synthetase syndrome (~64k), gMG (~93k), MS, PPMS+SPMS (~170k), SPS (less than 1k). ˜ Unmet need in lupus nephritis (LN) driven by significant rates of morbidity and mortality; broader systemic lupus erythematosus (SLE) market represents potential upside, but risk/benefit for CAR Ts less clear in patients without renal involvement.
$TGTX - Peter remember they all ready expanded to other indications AZER -cel (CD-19 CAR-T) – more than 20 PPMS (primary progressive MS) patients have been enrolled & trial expanded to include other indications (MG, CIDP, NMO or neuromyelitis optica) also PPMS yes smaller then MS but still lots of patients & complete unmet need PPMS accounts for roughly 10–15% of MS diagnoses (and a similar share of current cases). That corresponds to an estimated 310,000–465,000 people globally
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In 2024 UBS modeled in model global peak sales of ~$0.91bn for KYV-101 in MS, assigning a 10% POS with an anticipated launch in 2028. assume list pricing at a discount to currently approved CAR Ts ($300k in the US),
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$PTGX ICOTYDE $JNJ now been approved in the United Kingdom for adults and adolescents with moderate-to-severe plaque psoriasis. latest approval builds on approvals in the U.S., China, and EU
Protagonist is very pleased to see another global approval for ICOTYDE™ (icotrokinra)! ICOTYDE, co-discovered by Protagonist and Johnson & Johnson scientists, has now been approved in the United Kingdom for adults and adolescents with moderate-to-severe plaque psoriasis. This latest approval builds on approvals in the U.S., China, and EU, bringing ICOTYDE to more patients around the world. gov.uk/government/news/mhra-… #ICOTYDE #PeptideTherapeutics #PTGX
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$TGTX been around long enough to go thru few bear thesis with $TGTX - imho this was one of last ones that few was still shorts were hanging on to in the BTK class - imho - that concern was put to rest big time today
$TGTX the Roche BTK looks far better but it is not a competitor to Briumvi for RRMS: $NVS vs. $RHHBY: The MS BTK Inhibitor Showdown at #MSToronto2026 🧠💊 Phase III data are out for both Novartis’s remibrutinib and Roche’s fenebrutinib (tested against Aubagio/teriflunomide in RMS). The head-to-head profile comes down to a clear trade-off: Relapse Efficacy vs. Liver Safety. ⚙️ MECHANISM & TRIALS • Novartis (remibrutinib): Covalent, irreversible oral BTKi | Phase III REMODEL-1 & REMODEL-2 • Roche (fenebrutinib): Non-covalent, reversible oral BTKi | Phase III FENhance 1 & 2 (RMS) + FENtrepid (PPMS) 📉 ANNUALIZED RELAPSE RATE (ARR) REDUCTION (Street expectations: 45%–60% needed to rival anti-CD20s like Ocrevus, Kesimpta & Briumvi) • Roche: 55% reduction (pooled) | ~1 relapse every 17 yrs ✅ (Beat expectations) • Novartis: 35.2% (REMODEL-1) & 31.2% (REMODEL-2) | ~1 relapse every 13 yrs ⚠️ (Missed Street expectations) 🎯 NEDA-3 (NO EVIDENCE OF DISEASE ACTIVITY) • Roche: 75.0% (70% reduction in risk of not achieving NEDA-3 vs. comparator) • Novartis: 55.0% (vs. 32.1% for comparator) 🧠 DISABILITY PROGRESSION & BRAIN VOLUME • Novartis: ~30% delay in 6-month confirmed disability progression (6mCDP, nominal significance); over 92% of patients progression-free at 24 months • Roche: 16% lower risk of 12-week composite confirmed disability progression (cCDP12) in RMS; 23% reduction in annual total brain volume loss; proven non-inferiority to Ocrevus in PPMS 🔬 MRI & LESION ACTIVITY • Novartis: T2 lesions reduced by 65%–70%; Gd+ T1 lesions reduced by 66% • Roche: Slowly expanding lesions (SELs) reduced by up to 30%; paramagnetic rim lesions (PRLs) reduced by 62% 🛡️ LIVER SAFETY PROFILE (The BTKi Class Hurdle) • Novartis: CLEAN. Zero liver safety signal observed, no Hy's Law cases, and numerically fewer overall AEs (79.5% vs. 81.5%) and SAEs (6.0% vs. 7.0%) than teriflunomide • Roche: ELEVATED RISK. Significantly higher liver enzyme elevations vs. comparator (likely requiring hepatic monitoring on label) 📌 REGULATORY & COMMERCIAL OUTLOOK • Novartis: Already FDA-approved as Rhapsido in CSU & symptomatic dermographism; Phase III ongoing in SPMS (REMASTER). Est. $4B+ peak sales potential. Positioned as a best-in-class safe, convenient first-line oral without chronic B-cell depletion. • Roche: Granted FDA Priority Review across both RMS & PPMS. Positioned as a high-efficacy oral powerhouse targeting both relapses and chronic "smoldering" neuroinflammation, balanced against liver monitoring requirements. ⚖️ THE VERDICT Roche wins on headline relapse efficacy and broad RMS/PPMS reach, while Novartis wins decisively on hepatic safety—giving neurologists a choice between maximum oral potency and a clean, low-monitoring safety profile.
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$TGTX Excellent News! The safety is solid. They have achieved these results with relatively low levels of chemo conditioning and an off-the-shelf product - That is really important! Efficacy will only get clearer and better as there is more data from the high dose cohorts. Efficacy appears good enough to proceed given low levels of chemo conditioning and an off-the-shelf product. They need to run a few more subjects at the higher doses. Then, setup a meeting with the FDA to plan a registrational study. Given other recent CAR-T autoimmune registrational studies, FDA will probably require under 50 participants. This is much closer to FDA approval than many are aware!
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$TGTX as much as I like the AZER data & also how nothing was priced in - the MOVE in $TGTX today I am confident in has all to do with $NVS underwhelming data - as that was one of last bear thesis even BofA had kelo.com/2026/10/23/novartis…
$TGTX AZER CEL data even more impressive when you look at baseline of these hard to treat patients - All 26 participants had PIRA in the prior 12 months; all had prior anti-CD20 therapy
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$XBI #Biotechs good to hear more comments from @daphnezohar that investment banking & M&A alive in well
On today's #BiotechHangout – it’s our 200th episode! @daphnezohar, @EricSchmidt151, @gline aka “woodchipper”, @cngarabedian, @bradloncar, @biotech1 (Josh Schimmer), @SamFazeli8 will kick off with a look back to our 1st episode in December 2021.  $MRNA was influencing industry sentiment & continues today melanoma data w/$MRK & #ESMO data leak & recent impact on market sentiment (degrossing), @endpts Q3 sentiment index, does specialist sentiment lag generalist?, M&A w/  $SLN Merck KGaA deal & special guest @AAMortazavi, China outlook: Roche/Hansoh ($400M upfront) & hawkish treasury proposal, @DaveARicks social media presence, data: $ABVX, $ROIV, #UEGW2026, #ESMO2026 incl. $GSK SCLC, $TNGX pancreatic cancer, @DMSabatini judgment/settlement, & much more: xitter.cf/i/spaces/1qxoNYv…
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$TGTX AZER CEL data even more impressive when you look at baseline of these hard to treat patients - All 26 participants had PIRA in the prior 12 months; all had prior anti-CD20 therapy
$TGTX AZER CEL does not disappoint- 89% of evaluable participants (16/19) had stable or improved disability scores at Day 90, despite documented progression prior to enrollment on high-efficacy therapies
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$TGTX unreal to think we’re talking -REGISTRATION trials for something not ONE analyst had anything in their models for - but that’s what GREAT data in UNMET needs does - Q3 call in weeks. Azer & SUBq should dominate the call - I fully believe Weiss & $DTIL moves quickly as they can- Enthusiasm by DOCS & patients quickly growing
$TGTX Thank you R. In PPMS stopping progression would be a huge win, but reduced EDSS scores is a home run. Looks like TGTX has another winner. But unless they can shorten the time line to approval, there is still little or no PV. A big and gutsy move would be to design and roll out a pivotal trial sufficient for approval. One trial of stat-sig size could get approval in this underserved disease.
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$TGTX - Peter remember they all ready expanded to other indications AZER -cel (CD-19 CAR-T) – more than 20 PPMS (primary progressive MS) patients have been enrolled & trial expanded to include other indications (MG, CIDP, NMO or neuromyelitis optica) also PPMS yes smaller then MS but still lots of patients & complete unmet need PPMS accounts for roughly 10–15% of MS diagnoses (and a similar share of current cases). That corresponds to an estimated 310,000–465,000 people globally
Progressive MS is a terrible disease with no approved treatments. Much smaller market than Relapsing Remitting MS (85% of patients), so from a purely stock price perspective this isn't going to be that big a driver for $TGTX, but very important for the patients.
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$TNGX next data catalyst may forget ?? Jan 2027 initial vopimetostat monotherapy data in NSCLC and TNG456 monotherapy data in NSCLC, glioblastoma, and other cancers expected.
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dough retweeted
$TGTX 1 of 2 Azer-cel trial site updated w/ all 8 sites recruiting & the new indications that the trial includes w/ their inclusion criteria. Obviously, I'm interested w/ all results but in particular how it'll do w/ people that have RRMS now that is included w/ SPMS & PPMS.
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$TGTX #PPMS 10-15% of #MS patients with some hope today - other indications being tested - NOTHING at all in any model for AZER. - none that should change soon - next up SUBq data as that is & will be clear driver for $TGTX
$TGTX PPMS accounts for roughly 10–15% of MS diagnoses (and a similar share of current cases). That corresponds to an estimated 310,000–465,000 people globally. PPMS is progressive from the start, with gradual worsening and few or no relapses. It is diagnosed later on average (typically 40s–50s) and affects men and women at more similar rates than relapsing MS. multiplesclerosisnewstoday.c…
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$TGTX AZER CEL - #MS excitement was palpable on social media boards - will only increase when patients see #EDSS scores like this—Despite documented progression prior to enrollment, EDSS scores were stable or improved in 89% of evaluable participants at Day 90 (16/18) and 88% at Day 180 (7/8). Three participants achieved EDSS improvements of at least 2.0 points at Day 90, including one with a 4.0-point improvement. •At Day 90, most evaluable participants demonstrated stable or improved walking performance (94%; 16/17), upper extremity function (94%; 17/18) and cognitive processing speed (78%; 14/18), with no gadolinium-enhancing T1 lesions or new or enlarging T2 lesions observed in any of the 22 participants evaluable by MRI.
$TGTX Weiss has now mentioned AZER 2 more times on how it can work — imho under appreciated asset & catalyst 2h26
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$TGTX definitely some hope for these #MS patients with not many options Despite documented progression prior to enrollment, EDSS scores were stable or improved in 89% of evaluable participants at Day 90 (16/18) and 88% at Day 180 (7/8). Three participants achieved EDSS improvements of at least 2.0 points at Day 90, including one with a 4.0-point improvement.
$TGTX AZER CEL does not disappoint- 89% of evaluable participants (16/19) had stable or improved disability scores at Day 90, despite documented progression prior to enrollment on high-efficacy therapies
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$TGTX AZER CEL •Azer-cel demonstrated an encouraging initial safety profile in 26 participants, with no dose-limiting toxicities or high-grade CAR T associated toxicities observed across dose levels including no Grade ≥3 cytokine release syndrome (CRS), Grade ≥2 immune effector cell-associated neurotoxicity syndrome (ICANS), graft-versus-host disease, immune effector cell-associated hemophagocytic syndrome (IEC-HS) and no Grade ≥3 infections. •Despite documented progression prior to enrollment, EDSS scores were stable or improved in 89% of evaluable participants at Day 90 (16/18) and 88% at Day 180 (7/8). Three participants achieved EDSS improvements of at least 2.0 points at Day 90, including one with a 4.0-point improvement. •At Day 90, most evaluable participants demonstrated stable or improved walking performance (94%; 16/17), upper extremity function (94%; 17/18) and cognitive processing speed (78%; 14/18), with no gadolinium-enhancing T1 lesions or new or enlarging T2 lesions observed in any of the 22 participants evaluable by MRI. •Peripheral blood B-cell counts were depleted to below 10 cells/µL in 96% of participants (25/26), and cerebrospinal fluid (CSF) samples from 21 participants showed a median reduction in CD19-positive B-cell events of 52.3% at Day 10. B-cell reconstitution was observed as early as Day 42 and favored transitional and naïve populations.
$TGTX AZER CEL does not disappoint- 89% of evaluable participants (16/19) had stable or improved disability scores at Day 90, despite documented progression prior to enrollment on high-efficacy therapies
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