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GBT - Bull Thesis


  • GBT overall corporate and executive summary

  • Cash of $735M in Q4 ‘21, almost doubled in last quarter

  • A little over 2 years of cash on hand

  • Fairly cheap commercial company with EV ~10X trailing 12 month revenue

  • ~$2.2B Market cap and EV

  • $190M 2021 revenue

  • Possible acquisition target with $200M revenue with valuable pipeline assets

  • Oxbryta

  • Approved 1st in class sickle cell disease therapeutic

  • $190M ‘21 revenue

  • Impressive hemoglobin level increases shown in clinical trials

  • Additional benefits shown in post-hoc and real-world studies

  • RW studies published Feb ‘22

  • Leg ulcer resolution

  • Less transfusions, VOCs, hospital admissions, and iron chelation

  • Pipeline

  • Phase 3 SCD therapeutic anti-P selectin Ab candidate for VOCs

  • Same SCD market as Oxbryta

  • Oxbryta (Voxelotor) (sickle hemoglobin polymerization inhibitor)

  • Approved to treat SCD in Nov 2019

  • Expanded approval for patients 4 and up in December 2021

  • Approved in November 2019 and first full quarter of launch (Q1 2020) sales of $14.1M

  • Fourth quarter 2021 sales of $56.1M and full year 2021 of $194.6M

  • 57% increase year over year for full year sales

  • 36% increase year over year for quarterly sales

  • Oxbryta continues to have broad payer coverage for ages 12 years and older

  • More than 90% of covered lives having access

  • European Commission approval in February 2022

  • Phase 3 HOPE study announced positive results throughout 2018

  • Primary endpoint achieved in June 2018

  • Statistically significant increase in hemoglobin was seen

  • Post-hoc analysis of HOPE study showed improvement in leg ulcers

  • Recent real world experience trials show additional benefits

  • Less transfusions, VOCs, hospital admissions, and iron chelation

  • Inclacumab (p-selectin inhibitor)

  • Reduce freq of vaso-occlusive crises (VOCs) & hospital readmissions

  • Addresses major immediate health issue in SCD

  • Established mechanism of action for treating VOCs

  • Phase 3 trials in Sickle Cell Disease (THRIVE)

  • Two ongoing phase 3 trials provide upcoming catalysts

  • Phase 1 data presented at ASH 2021

  • Binds more natural binding site than competitive Novartis drug (crizanlizumab)

  • Potential for longer duration of exposure and near complete inhibition of platelet-leukocyte aggregation over 12 week period

  • Best-in-class potential as a quarterly-dosed P-selectin inhibitor

  • Are side effects better than crizanilizumab

  • Criz has >=10% nausea, back pain, and pyrexia

  • GBT21601 (sickle hemoglobin polymerization inhibitor)

  • GBT021601 Phase 2 to initiate by mid 2022 in SCD

  • Next gen hemoglobin polymerization inhibitor

  • Phase 1 results proved 601 has a favorable safety and tolerability profile while lowering the pill burden of patients

FS and MV 4/5/22

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