AnonStatSci

37 posts

AnonStatSci

AnonStatSci

@AnonStatSci

Katılım Mart 2026
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AnonStatSci
AnonStatSci@AnonStatSci·
@avidresearch Every ‘funky’ approval (even if accelerated) is precedent-setting and forever cited by sponsors in the future. Contribution of components is a fundamental tenet of drug development. aPD-1 re-challenge (particularly if different aPD/L-1) has shown activity in this setting.
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avidresearch
avidresearch@avidresearch·
Popular high level narrative was that $REPL drug deserved to be approved and then there were the minor details about leadership and review teams.
Adam Feuerstein ✡️@adamfeuerstein

Whether or not you believe $REPL RP1 deserves to be approved or not, let's please be truthful about what has transpired so far: Richard Pazdur and others in the FDA's cancer office were responsible for RP1's initial rejection. They objected late in the review process. CBER ie Vinay Prasad and his team signed off on the rejection. This doesn't fit the popular narrative, but it's what happened.

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AnonStatSci
AnonStatSci@AnonStatSci·
@kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology @PeterKolchinsky @MPN_RF @AaronGerds @SGO_org @AACR @CIUSSS_COMTL @MDmasarova While MF is DoA, I can’t see how the EC study can fail against placebo control, and given SIENDO readout. This should be a slam-dunk, SENTRY was an overhang that had to clear. NSMP EC in EU will see uptake, the US will be an uphill commercial challenge absent strong OS.
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@KinatSofrim
@KinatSofrim@kinatsofrim·
@harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology @PeterKolchinsky @MPN_RF @AaronGerds @SGO_org @AACR @CIUSSS_COMTL @MDmasarova $KPTI Catalio’s co-founder father is Dr. Bert Vogelstein the world's premier TP53 scientist who discovered TP53’s role as a tumor suppressor, the exact biology underlying why selinexor works in EC. Bert is also a venture partner in Catalio. /400 x.com/kinatsofrim/st…
@KinatSofrim tweet media
@KinatSofrim@kinatsofrim

Jacob's dad the legendary Dr. Bert Vogelstein "has been involved in research that led to the dev. of tests to detect EC" & "discovered that the p53 gene is a tumor suppressor gene that's frequently mutated in human cancers". x.com/kinatsofrim/st…

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@KinatSofrim
@KinatSofrim@kinatsofrim·
$KPTI MF timeline. P1 update at ASH Dec. 2022, then April 2023 at AACR, initiates P3 June 2023, receive FT July 2023 (received OD in Oct. 2022), Nov. 2023 CEO @ Jefferies "Very open to the possibilities to make sure that we maximize the potential of Selinexor + Rux $INCY". /1
Juan P. Serrate, DVM@JPZaragoza1

From Cantor: biotech M&A transactions took an avg of 12.9 mos in 2023. From start of deals talks to deal announcement. Assume deal talks start after data, confirming this and how long after data.

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AnonStatSci
AnonStatSci@AnonStatSci·
@bradloncar @US_FDA @Replimune Re-challenge with alternative aPD-1 to has shown activity in aPD-1 failures. Contribution of components is a central concept of drug development. FDA would be forced to set a new precedent that would then be referenced by countless future submissions - equitable treatment…
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Brad Loncar
Brad Loncar@bradloncar·
Some observers, including @US_FDA, have criticized @Replimune for the design of the trial that led to the decision not to grant accelerated approval of their melanoma treatment. I asked the company's Chairman about that view. Watch the full interview: biotechtv.com/post/replimune…
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AnonStatSci
AnonStatSci@AnonStatSci·
@mountainliren @bradloncar @US_FDA @Replimune Re-challenge with alternative aPD-1 to has shown activity in aPD-1 failures. Contribution of components is a central concept of drug development. FDA would be forced to set a new precedent that would then be referenced by countless future submissions - equitable treatment…
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Mountain Ren
Mountain Ren@mountainliren·
@bradloncar @US_FDA @Replimune Thank you for doing this. His key argument is that for anti-PD1 drug to work, it needs RP1 to sensitize the patient, That’s why they have to combine RP1 with Nivolumab and Nivolumab can not work on those patients alone. This is at most a hypothesis to be confirmed.
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AnonStatSci
AnonStatSci@AnonStatSci·
@doepke_michel Smart money saw MF readout as clearing out a -ve catalyst ahead of the EC read-out which should be nicely +ve; smart money will take profit and leave before control-arm relevance, regulatory uncertainty and commercial uptake questions arise…
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Michel Doepke
Michel Doepke@doepke_michel·
$KPTI higher than ahead of MF phase 3 data. Interesting. Keep my small position.
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AnonStatSci
AnonStatSci@AnonStatSci·
@kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology @PeterKolchinsky @MPN_RF In the Ph1 mono sel study (R/R MF); 3/17 pts got cateracts; 5/17 eye disorders; 12/17 fatigue. Full safety data not yet presented. How can pts feel worse/same on symptoms (excl, fatigue) with the combo when half the TSS items are spleen-related?!
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@KinatSofrim
@KinatSofrim@kinatsofrim·
@harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology @PeterKolchinsky @MPN_RF RBC "Hosted a leading heme onc physician and MPN KOL, as well as an investigator in the SENTRY trial, to further evaluate $KPTI's P3 MF data" /380
@KinatSofrim tweet media
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AnonStatSci
AnonStatSci@AnonStatSci·
@Komatinsky21199 In the Ph1 mono sel study (R/R MF); 3/17 pts got cateracts; 5/17 eye disorders; 12/17 fatigue. Est. off-label use when full safety data not yet known is tricky. How can pts feel worse/same on symptoms with the combo when half the TSS items are spleen-related?! (excl, fatigue!)
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Sean Komatinsky
Sean Komatinsky@Komatinsky21199·
$KPTI RBC consulted KOL and while data didn't hit both coprimary endpoints the KOL is interested in using Selinexor. Estimates 30-40% penetration with NCCN incorporation in guidelines. MF currently a $2B+ market in the U.S.
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AnonStatSci
AnonStatSci@AnonStatSci·
@avidresearch @mino_cappmont @komatinskys @sak77328 @kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology So lower SVR rate and less VAF reduction tha nav/rux but expectation of more durable SVR with sel/rux? - how come? - aren’t the SVR durability data confounded by discons? Nav daily vs sel weekly dosing? Guess we are still waiting for durability from SENTRY with next update.
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avidresearch
avidresearch@avidresearch·
@AnonStatSci @mino_cappmont @komatinskys @sak77328 @kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology OS benefit need to be validated but if it holds up, could be attributed to XPO1 inhibition having better synergistic effect with Jakafi compared to NAVI and Pela
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AnonStatSci
AnonStatSci@AnonStatSci·
@avidresearch @mino_cappmont @komatinskys @sak77328 @kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology Thought we were talking about Navitoclax. SVR35 63% at Wk-24 and 53% at wk-48? Isn’t that on par with SENTRY? Why do you think mechanistically Sel is showing this early OS imbalance and Navitoclax did not?
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avidresearch
avidresearch@avidresearch·
@AnonStatSci @mino_cappmont @komatinskys @sak77328 @kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology Its not SVR35 but “durable SVR35” that’s more relevant for OS benefit. Pelabraisb SVR35 durability was awful.
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AnonStatSci
AnonStatSci@AnonStatSci·
@avidresearch @mino_cappmont @komatinskys @sak77328 @kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology Possible, but that would be contrary to Ph2 REFINE findings. FDA does not acknowledge SVR as a surrogate to survival in MF. You highlighting the potential discrepant TRANSFORM-1 vs SENTRY relationships only serves to prove that it is not. Valid surrogate has to be drug agnostic.
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avidresearch
avidresearch@avidresearch·
@AnonStatSci @mino_cappmont @komatinskys @sak77328 @kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology Maybe because SVR35 benefit they saw didn’t lead to survival benefit ? 10% mortality seen (similar to Sentry control arm) “In each arm, 13 (10%) patients died; 6 with NAV + RUX and 5 with PBO + RUX died ≤30 days post-final dose.”
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AnonStatSci
AnonStatSci@AnonStatSci·
@komatinskys @kinatsofrim @mino_cappmont @sak77328 @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology Company could always repeat the study with a prospective OS endpoint and power for a HR < 0.5 with an interim at 25% events. FDA will definitely support that study. Might take a few years… SIENDO déjà vu…. Would be a game-changing study if it hit.
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komokazi
komokazi@komatinskys·
@AnonStatSci @kinatsofrim @mino_cappmont @sak77328 @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology SVR maintenance correlation with OS has been demonstrated. We’ll obviously see the FDA’s opinion on the Selinexor trial data when $kpti reports the feedback they receive. OS is the prize that FDA really wants not TSS.
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AnonStatSci
AnonStatSci@AnonStatSci·
@avidresearch @mino_cappmont @komatinskys @sak77328 @kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology And $KPTI will succeed where AbbVie failed? - pull AbbVie’s transcript on the earnings call when the dropped Navitoclax - they even poked fun of MorphoSys’ ‘Totality of data’ regulatory strategy at the time. Ie, “we tried, didn’t work”; FDA can’t be inequitable in their reviews.
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avidresearch
avidresearch@avidresearch·
TSS is critical for monotherapy. It’s pretty obvious TSS benefit on top of a highly effective JAK monotherapy is extremely hard to demonstrate in a trial. If Selinexor can double SVR35 compared to JAK alone and also improve survival, it’s not worthy of approval because there was no TSS benefit (something 3 ph3 trials has shown is nearly impossible to achieve) ?
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AnonStatSci
AnonStatSci@AnonStatSci·
@kinatsofrim @komatinskys @mino_cappmont @sak77328 @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology I think we’re straying into realms of opinions and whatifery. I couldn’t condone, in good conscience, all the naive declarations of ‘survival benefit’ on an extremely immature (6% events) OS analysis; HR with nominal p-value on a failed study; no disclosed KM’s. Good luck! Adios.
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AnonStatSci
AnonStatSci@AnonStatSci·
@mino_cappmont @komatinskys @sak77328 @kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology You saw $KPTI are withdrawing accelerated approval in 3L+ DLBCL following request from FDA? It would pay folks to read the Vonjo FDA clinical review doc to provide an understanding of how FDA view SVR as a standalone (tldr: NOT a validate surrogate for OS). TSS is critical >> SVR
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Beniamino Cappelletti Montano
Beniamino Cappelletti Montano@mino_cappmont·
@komatinskys @AnonStatSci @sak77328 @kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology The problem is that we'll have to wait quite a while for the data to mature (from a statistical standpoint, we need at least 100 to 150 events, and we're currently only at 23)
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AnonStatSci
AnonStatSci@AnonStatSci·
@sak77328 @kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology Agree there is unmet need specifically in those that do not respond well - but why expose the pts who respond well to additional toxicity and potential harm or dose-reductions leading to suboptimal dosing of standard-of-care? Unmet need for all-comers is not there to allow AA.
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AnonStatSci
AnonStatSci@AnonStatSci·
@kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology Wait… what…? ChatGPT wrote all of this rubbish, right? So the ‘unmet need’ here is that no combo standard-of-care tx exists in the front-line MF setting?
AnonStatSci tweet media
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AnonStatSci
AnonStatSci@AnonStatSci·
@kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj @Blood_Cancers @RaajitRampal @ByMadeleineA @LongwoodLeaders @mpnadvocacy @HealthTree @mpdrc @IcahnMountSinai @pankitvachhani @OncLive @MPN_BMTDOC @sk_tantravahi @AshKishtagari @Yi_Chai18 @CNBCFastMoney @MelissaLeeCNBC @UofUHematology Is this analyst very junior? This analysis seems to be confabulating SVR, TSS (signs & symptoms), PFS, OS; MF vs solid tumours - MF is reviewed by non-malignant heme division. The FDA summary review for Vonjo would be a good read… the FDA workshop on TSS in MPN too.
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AnonStatSci
AnonStatSci@AnonStatSci·
@Stmkrs EC study should be positive - commercial potential uncertain tho given IO use in 1L maintenance. How do u know RA had access to the data? It’s not fileable as -ve study. All EPs tested after Abs.TSS are ‘exploratory’. Putting on a brave face until EC read-out methinks.
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Biotech Pharma Investor
Biotech Pharma Investor@Stmkrs·
@AnonStatSci RA Capital had access to the data and gave then $30M upfront at $6.8. So it cant be that bad. But I agree with all your points. too early to make any conclusions. For trading purposes RA + runway through endometrial ca readout + hope for AA / off-label treatment are enough for me
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AnonStatSci
AnonStatSci@AnonStatSci·
@Stmkrs Where do you draw the line? What if the deaths occurred within the first 2 weeks? What if the event rate maturity was 2%? What if the numbers were 4 vs 1 in favour? What if week-48 OS rate is half that of historical precedent? You need to see the curves, otherwise just messaging.
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Biotech Pharma Investor
Biotech Pharma Investor@Stmkrs·
@AnonStatSci Sure I can say the effect size is large. HR 0.44 and half the deaths is a large effect size. But as I said I agree there is high uncertainty as to where the true effect is.
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AnonStatSci
AnonStatSci@AnonStatSci·
@komatinskys @Stmkrs Not sure switching your calculation from ITT to evaluable changes any conclusions, though it does introduce survivorship bias. 31% combo vs 20% Rux discon by wk-36. Guess a non-SVR35 on Rux is more likely to stay on tx than same patient on combo? Wonder why…
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komokazi
komokazi@komatinskys·
@AnonStatSci @Stmkrs So 97 of 134 (combo) vs 23 of 76 (Rux) are SVR 35 at week 36. Wow, even more compelling reason to approve the drug as those who respond, really respond.
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Keşfet

@avidresearch @kinatsofrim @harrisoncn1 @peter_mpn @KartosThera @buysidebio @Biopharmaddict @bose_prithviraj