250113 - roche 罗氏
250113 - roche 罗氏
This presentation contains certain forward-looking statements. These forward-looking statements may be identified by words such
as ‘believes’, ‘expects’, ‘anticipates’, ‘projects’, ‘intends’, ‘should’, ‘seeks’, ‘estimates’, ‘future’ or similar expressions or by discussion
of, among other things, strategy, goals, plans or intentions. Various factors may cause actual results to differ materially in the
future from those reflected in forward-looking statements contained in this presentation, among others:
Any statements regarding earnings per share growth is not a profit forecast and should not be interpreted to mean that Roche’s earnings or earnings
per share for this year or any subsequent period will necessarily match or exceed the historical published earnings or earnings per share of Roche.
For marketed products discussed in this presentation, please see full prescribing information on our website www.roche.com
All mentioned trademarks are legally protected.
Unlocking the next era of innovation
Teresa Graham
CEO Roche Pharmaceuticals
Delivering on the Pharma Ambition
Looking ahead
4
Sales momentum expected to continue into 2025
Strong growth by both divisions: Pharma and Diagnostics
%CER %CER
+15% +15%
Total COVID-19 sales of CHF ~19bn2 No significant COVID-19 impact going forward
All growth rates at Constant Exchange Rates (avg full year of the respective years); 1Base business=Pharma excluding Ronapreve and Diagnostics excluding COVID-19-related products; 2COVID-19 sales
referring to COVID-19 diagnostic tests, Ronapreve and Actemra sales 5
Young portfolio to drive growth in the near- to mid-term
2024 NME update: Achieved approval for Itovebi (inavolisib) in HR+ breast cancer and PiaSky in PNH
CHFm % of Pharma Sales2
20.0 56%
18.0 50%
16.0
42%
14.0
12.0 34%
10.0
8.0
1 6.0
4.0
2.0
0.0
YTD Sep 2021 YTD Sep 2022 YTD Sep 2023 YTD Sep 2024
2015 2016 2017 2018 2019 2020 2021 2022 2023 2024
Cotellic Alecensa Tecentriq Ocrevus Hemlibra
Luxturna Xofluza Polivy Rozlytrek Enspryng
Phesgo Evrysdi Ronapreve Susvimo Vabysmo
Lunsumio Columvi Elevidys Piasky
Young portfolio defined as all launches since end of 2015; 1Elevidys: Accelerated US approval by partner company Sarepta; 2Venclexta sales booked by AbbVie and therefore not included;
NME=new molecular entity; PNH=paroxysmal nocturnal hemoglobinuria; HR=hormone receptor 6
Our new Pharma Strategy focusing on five TAs
Ambition to deliver 20 transformative medicines in areas of highest societal burden
Where we play: Priority Therapeutic Areas Global Burden of Disease (‘23 & ’35, mn DALYs)
+9%
Neurology Oncology/ 2,408 Growth (p.p %)
Hematology 2,208 33
25 225 33%
184 286
231 22%
268 328
24%
531 617
22%
Ophthalmology Immunology
16%
970 920
-5%
Source: IHME, Clarivate (DRG); 1 Immune-mediated diseases incl. selection of diseases across TAs, i.e., COPD, IBD, CSU, CF, RA, urticaria, idiopathic interstitial pneumonia (incl. ILD), chronic cough, glomerulonephritis, LN/SLE, asthma,
IgAN; 2 Others incl. Infectious Diseases, Reproductive Health incl. neonatal and maternal health, non-immunological gastrointestinal diseases, vitamin deficiencies, among others; CVRM=cardiovascular, renal and metabolism 7
One asset team approach allows us to maximize MoAs across TAs
For example, incretins have the potential to become a foundational target within several TAs
Drug target
VEGF
IL6
CD19
C5
GLP-1/GIP
NLRP3
Latent myostatin
VEGF=vascular endothelial growth factor; IL6=interleukin 6; GLP-1=glucagon-like peptide-1; GIP=gastric inhibitory polypeptide; NLRP3=NOD-, LRR- and pyrin domain-containing protein 3 8
Oncology/Hematology
Neoadj
Ph I Ph II Ph III
INAVO120 (1L PIK3CA-mut HR+HER2-
Itovebi SURGERY
mBC (endocrine resistant))
INAVO121 (post-CDK4/6 PIK3CA-mut 3
Itovebi 3
HR+/HER2- BC) gire
gire gire
Adj
ET + CDKi
Itovebi
INAVO122 (1L PIK3CA-mut HER2+ BC)
+ Phesgo
Non-visceral disease Visceral crisis
INAVO123 (1L PIK3CA-mut HR+/HER2-
Itovebi De novo/AI-sensitive (60%) AI-resistant (40%)
mBC (endocrine sensitive))
giredestrant persevERA (1L ER+/HER2- mBC 1 2
1 2025 gire + gire+
1L
+ palbociclib (endocrine sensitive)) CDKi CDKi
giredestrant Chemo ADC
+ everolimus
evERA (post CDKi ER+/HER2- mBC) 4 2025 +/- bev
giredestrant
+ Phesgo
heredERA (1L maintenance ER+/HER2+
mBC)
• Leading SERD with head-to-head adjuvant trial vs. endocrine therapy
HER2 TKI HER2+ BC • Evaluating combination with abemaciclib in eBC with single arm sub study as part of Ph III lidERA
RGT-419B
HR+ BC
• Plan to initiate combination with RGT-419B (CDK4/2i)
(CDK4/2i)
runimotamab
HER2+ BC
• Readouts for Ph III (persevERA) in 1L ER+/HER2- mBC and Ph III (evERA) in postCDKi ER+/HER2-
(HER2xCD3) Approved
mBC expected in 2025
1Risk definitions vary
according to guidelines and tools used: stage at diagnosis based on internal estimates using SEER data; ET=endocrine therapy; PIK3CA-mut=phosphatidylinositol 3-kinase, catalytic, alpha polypeptide
mutated; HR+=hormone-receptor positive; ER+=estrogen receptor positive; HER2=human epidermal growth factor receptor 2; e/mBC = early/metastatic breast cancer; CDKi=cyclin dependent kinase inhibitor; TKI=tyrosine kinase 9
inhibitor; neoadj=neoadjuvant; adj=adjuvant; ADC=antibody drug conjugate; bev=bevacizumab; BIC=best-in-class; SoC=standard of care; AKT=serine/threonine kinase 1; mTOR=mammalian target of rapamycin
Oncology/Hematology
Non-viral gene insertion and gene “On-demand” delivery from P-BCMA-ALLO1 Oncology MM Phase I
• Technology features set Poseida ALLO-CAR-Ts apart as potentially best-in-class in • Early clinical data for P-BCMA-ALLO1 in MM indicates
Oncology, Immunology and Neurology strong activity, comparable to auto. BCMA CAR-Ts
• FDA awarded P-BCMA-ALLO1 orphan drug designation (ODD) for treatment of MM, • Most recent data for P-BCMA-ALLO1 and P-
and Regenerative Medicine Advanced Therapy Designation (RMAT) for R/R MM CD19CD20-ALLO1 presented at ASH
• FDA INDs filed for P-CD19CD20-ALLO1 in MS and SLE
CAR-T=chimeric antigen receptor T-cell; MM=multiple myeloma; ODD=orphan drug designation; RMAT=regenerative medicine advanced therapy designation, RR=relapsed refractory; BCMA=B-cell 10
maturation antigen; MS=multiple sclerosis; SLE=systemic lupus erythematosus; IND=investigational new drug
Oncology/Hematology
Ph I Ph II Ph III Ph IV
• BEYOND ABR evaluating overall health, • Potential to achieve zero treated bleeds for Hem A pts, without need for additional FVIII
physical activity, and joint outcomes treatment
• NXT007 Ph II data expected in 2025 • Engineered based on Hemlibra, to enhance binding affinities, extend half-life, and allow for low
volume, infrequent subcutaneous injections
• Ph IIb of Next Gen SPK gene therapy with
enhanced function FVIII variant in HemA to • NXT007 is ~30-fold more potent than Hemlibra and in vitro assay indicates that thrombin
initiate in H2 2025 generation is within the range of people without Hem A2
*Based RWD from McCary I, et al. Haemophilia 2020, Wall C, et al. ISTH 2020, Poon M-C, et al. ASH 2022 and Khairnar R, et al. ASH 2021; 1Koga et al. MABS 2023, 15 (1), 2222441; 2Teranishi-Ikawa et 11
al. Journal of Thrombosis and Haemostasis 2024.22 (2):430-440; SOC=standard of care; Q2W/Q4W=once every 2/4 weeks; SC=subcutaneous; MMS=mild-moderate-severe; IA=interim analysis
Neurology
• Trontinemab crosses the BBB using Roche’s proprietary BrainshuttleTM technology, and removes Aβ rapidly and robustly
• Amyloid clearance is accompanied by decreases in key biomarkers
• Sustained low incidence of ARIA vs other mAbs and favourable overall safety and tolerability
• Further data from Ph II (available H1 2025) will ungate Ph III clinical program
1Whole-brain imaging highlights the distribution of classical IgG vs. BrainshuttleTM IgG targeting a neuronal target in mouse brains; Lecanemab CLARITY AD: van Dyck et al. N Engl J Med 2023; 388:9-21; Donanemab TRAILBLAZER-
ALZ-2: Sims et al .JAMA. 2023;330(6):512-527; Trontinemab: Kulic et al, CTAD 2024; AD=Alzheimer’s disease; PET=Positron Emission Tomography; mAb=Monoclonal antibody; Aβ=Amyloid Beta; IgG1=immunoglobulin G1; BBB=blood
brain barrier; *For illustrative purposes - not based on head-to-head data, values from different trials 12
Neurology
Selectivity
PPMS Ocrevus FENtrepid Low Low High
High
Ocrevus RMS/ RMS, RMS, SPMS,
Ocrevus MUSETTE/GAVOTTE RMS RMS
HD PPMS PPMS (vs Ocrevus) PPMS (vs placebo)
• All Ph III fenebrutinib studies in RMS and • Fenebrutinib binds reversibly to BTK with kinetics that may positively influence efficacy and safety
PPMS to read out in 2025 • Fenebrutinib is the most potent BTKi in the inhibition of FcR and BCR signalling2
• Ph III (FENtrepid) in PPMS is the only • In an in vitro kinase activity assay, fenebrutinib only inhibited three off-target kinases3
H2H study vs Ocrevus
• Ocrevus HD Ph III (MUSETTE/GAVOTTE)
in RMS/PPMS results expected in 2025
Fenebrutinib could disrupt the oral market segment, currently comprising >40% of the global MS market
1Kramer, et al. (2023) Nat Rev Neurol. 2023;19(5):289-304 ;Crawford et al.(2018) J Med Chem 61, 2227-2245; Francesco et al., ACTRIMS-ECTRIMS (2017) 200644. Haselmayer et al. (2019) J Immunol 202, 2888-2906; Angst et al.
(2020) J Med Chem 63, 5102-5118; 2Weber MS, et al. AAN 2021 (Oral presentation P15.091); 3Johnson et al. Presented at MSVirtual 2020 (Presentation number P0338) H2H=head-to-head; MS=Multiple sclerosis; RMS=relapsing
multiple sclerosis; PPMS=primary progressive multiple sclerosis; SPMS=Secondary progressive multiple sclerosis; BTK=Bruton’s tyrosine kinase; nM=nanomolar; WB=whole blood; *As of Sept 2024: including non-MS Ph I/II studies
Immunology
Fibrosis Ph I Ph II Ph III
TGF-β, IL-6 Fibrosis
• TL1A binds and activates the DR3 receptor, stimulating downstream inflammation • Ph III in UC ongoing; Ph III in CD with FPI expected Q1
and fibrosis processes • Initiating Ph IIb trial in atopic dermatitis and Ph Ib trial
• TL1A is dysregulated in patients with immune-mediated diseases, with clinical and in MASH in Q1’25
translational links to IBD, rheumatoid arthritis amongst others • Continuing to explore additional indications
• Non-clinical and translational studies demonstrated its involvement in • Added anti-p40/TL1A bi-specific antibody to
pathogenesis of fibrotic conditions pipeline*; Ph II in IBD to initiate in 2025
*Global collaboration with Pfizer; 1 Xu WD, Li R, Huang AF. Role of TL1A in Inflammatory Autoimmune Diseases: A Comprehensive Review. Front Immunol. 2022 Jul 14;13:891328. doi: 10.3389/fimmu.2022.891328. PMID: 35911746; 2 Herro R, et al.
TL1A Promotes Lung Tissue Fibrosis and Airway Remodeling. J Immunol. 2020 Nov 1;205(9):2414-2422. doi: 10.4049/jimmunol.2000665. Epub 2020 Sep 21. PMID: 32958689; PMCID: PMC7577982.; PMCID: PMC9329929; TL1A=tumor necrosis
factor-like cytokine 1A; DR3=dopamine 3; IBD=inflammatory bowel disease; UC=ulcerative colitis; CD=Crohn’s disease; MASH=metabolic dysfunction-associated steatohepatitis 14
Immunology
Follow-up
(n=1290)
20% 30% Patients with R Astegolimab SC Q4W
High Current moderate to
70% severe COPD
1:1:1
Placebo
80% Low Former
• Near-term biologics in COPD are focused on EOS high or former smokers only
• Astegolimab pivotal trials enrolled broad patient population including former and current smokers, and EOS low to high
• Astegolimab has the potential to address neutrophilic inflammation as well as eosinophilic inflammation that could be key to reducing
exacerbations across a broader population of patients
• Early results show benefit in key endpoints throughout broad patient populations2
• Ph IIb (ALIENTO) and Ph III (ARNASA) pivotal results expected 2025
1 Global strategy for prevention, diagnosis and management of COPD: 2023 report; 2 Yousuf et al. Lancet Respir. Med. 2022;10 (5):469-77; *EOS high defined as ≥300 eosinophil cells per microliter of blood and EOS low defined as
<300 eosinophil cells per microliter of blood; FIC=first in class; COPD=chronic obstructive pulmonary disease; SoC=standard of care; SC=subcutaneous; Q2/4W=every 2/4 weeks; EOS=eosinophils 15
Ophthalmology
BCVA (letters)
Vabysmo POYANG (CNV)
BARDENAS/ALLUVIUM
vamikibart Weeks
(DME)
satralizumab SatraGO-1/2 (TED) • Vamikibart inhibits all known forms of IL-6 signaling; specifically designed for intraocular use
and optimized for a rapid systemic clearance
OpRegen GA
BURGUNDY
• 25-36% of patients gained 15 letters or more at week 12
zifibancimig
VEGF-Ang2 DutaFab (nAMD)
• All doses of vamikibart were well tolerated across all patients, with no treatment-related
Filed
serious AEs, sustained IOP increase, or new cataracts
• Vabysmo PFS: EU approval achieved • Ph III (SANDCAT/MEERKAT) trials in UME ongoing, data expected 2025
• Susvimo in DME/DR: US filing accepted • Ph II (BARDENAS/ALLUVIUM) trials in DME ongoing
1. Sharma et al. ARVO 2023; UME/DME=uveitic /diabetic macular edema; PFS=pre-filled syringe; TED=thyroid eye disease; GA=geographic atrophy; CNV=choroidal neovascularization; IOP=intraocular
pressure; IL-6=interleukin-6; AE=adverse event; BCVA=best-corrected visual acuity; SE=standard error; IRF=intraretinal fluid; SFR=subretinal fluid 16
Cardiovascular, Renal & Metabolism
Ph I Ph II
Efficacy / safety by NME
CT-388 103/104 (Obesity +/- T2D)
Ph I cohort 13 Impact on Improved
data Q4 ‘24
comorbidities convenience
004 (T1D w. OW/OB as adjunct Ph II data
CT-868
treatment) H2 2025
Combinations & comorbidities
201 (Obesity Ph I T2D
CT-996 (FPI 2025)
+/- T2D) data 2025 Improved tolerability Improved durability
CT-173 Obesity
(PYY analogue) (FPI 2025) Holistic patient solutions
Combo study
GYM 329 Obesity
(FPI 2025)
Muscle preservation Advanced diagnostics
Market segmentation
• Pipeline of potentially differentiated assets • CT-388 and CT-996 with BIC potential, as well as CT-868 with BIC/FIC potential in OW/OB
• Positive Ph I data for CT-388 & CT-996 patients with T1D, based on Ph I results
shared at EASD • Obesity market expected to segment by e.g. comorbidities, weight loss goal, oral vs SC
• CT-868 Ph II data expected in H2 2025 • Combination potential with Roche assets in several TAs; expansion into adjacent indications
• CT-173 Ph I in obesity to initiate in 2025 • SC devices in development; synergies with Roche Diagnostics Diabetes & Digital Health solutions
T2D=type-2 diabetes; T1D=type-1 diabetes; OW=overweight; OB=obese; FPI=first patient in; BIC/FIC=best-/first-in-class; SC=subcutaneous 17
Cardiovascular, Renal & Metabolism
background
anti- • Change at Month 3 and Month 6 in 24-hour
hypertensive Cohort B Zilebesiran 300 mg SC Q6M mean SBP and DBP, assessed by ABPM
regimen (N~60-120,
KARDIA-2
• Change at Month 3 in seated office DBP
n=15-30/arm) Zilebesiran 150 mg SC Q6M
• Change at Month 6 in seated office SBP and
Placebo KARDIA-3 FPI Q2 2024
DBP
6-month double-blind treatment period: • Change in serum AGT
Continue background antihypertensive regimen CVOT based on KARDIA-3 Exp. FPI H2 2025
aHTN intensification allowed only after M3
• Ph II (KARDIA-1): Monotherapy in mild/mod hypertension; primary endpoint of reduction of 24-h mean systolic blood pressure at 3 months met
• Ph II (KARDIA-2): Add-on to 1 SoC in uncontrolled hypertension; primary endpoint of systolic blood pressure reductions in all arms at month 3 met
• Ph II (KARDIA-3): FPI 2024; Cohort A fully enrolled (results will inform pivotal Ph III trial design); Cohort B currently enrolling
• Ph III (CVOT): Composite MACE endpoint in uncontrolled hypertension at high CV risk, to deliver robust label and access with CV outcomes
benefits at launch; Potential for expansion to other CV indications
1 NCT06272487; aHTN=antihypertensive; ABPM=ambulatory blood pressure monitoring; D/SBP=diastolic/systolic blood pressure; SoC=standard of care; CV=cardiovascular; CVOT=CV outcomes
trial; MACE=major adverse cardiovascular events; R=randomization; M=month; SC=subcutaneous; Q6M=every 6 months; QD=daily; zilebesiran in partnership with Alnylam Pharmaceuticals 18
Delivering on the Pharma Ambition
Looking ahead
19
Accelerating R&D Excellence
We are implementing six solutions to achieve top quartile performance
Adopt a unified portfolio Transform our portfolio Access the best external
framework management & governance innovation
Introduce the ‘Bar’ to recognize assets Reconfigure end-to-end governance to Identify and bring in exciting external
with transformative potential meet the ‘Bar’ assets which clear the ‘Bar’
Embrace ambitious R&D Evolve our R&D engine and Align our incentives with the
objectives invest in its excellence new R&D strategy
Set bold R&D objectives linked to Invest in technologies and platforms Link our R&D productivity objectives to
Pharma ambition that enable top-tier R&D productivity individual and team performance
Focus for today 20
The Bar is applied to each asset entering and progressing in the portfolio
Applying rigor to our R&D resource allocation
The Bar
Answers a clear & Engages a Possesses worthy Achieves meaningful Unlocks a path to
addressable unmet ‘foundational pharmacologic therapeutic value
need target’ characteristics differentiation
21
‘Fast-track’ of selected programs with exceptional potential
Material progress achieved across all three programs since Pharma Day
1 Includes both Ulcerative Colitis and Crohn’s Disease; TL1-A=tumor necrosis factor-like cytokine 1A 22
We have materially reshaped our pipeline through Partnering and M&A
Key deals completed to complement our pipeline across the Pharma focus therapeutic areas
Dual GLP-
Molecular Allogeneic
1/GIP RA & siRNA Anti-TL1A in
Wnt agonist glue ADCs in CDKi CAR-Ts in
PYY targeting inflammatory
in degraders in solid tumors, portfolio in oncology,
analogue in AGT in bowel
retinopathies oncology and incl. SCLC breast cancer immunology &
obesity hypertension disease
neurology neurology
±diabetes
CVRM=cardiovascular, renal & metabolism; siRNA=small interfering RNA; AGT=angiotensinogen; TL1A=Tumor necrosis factor-like cytokine 1A; GLP-1=glucagon-like peptide-1; GIP=glucose-dependent
insulinotropic polypeptide; RA=receptor agonist; CDKi=cyclin dependent kinase inhibitor; ADC=antibody-drug conjugate; WNT=wingless-related integration site; CAR-T=chimeric antigen receptor T- cell 23
Delivering on the Pharma Ambition
Looking ahead
24
2024: Key newsflow*
Ph II prasinezumab missed primary endpoint, but suggests possible benefit in early-stage Parkinson’s disease
Compound Indication Milestone
Alecensa Adjuvant ALK+ NSCLC US/EU approval
inavolisib + palbociclib + fulvestrant 1L PIK3CA-mut HR+ BC US/EU filing
Tecentriq Subcutaneous administration US/EU approval
PiaSky (crovalimab) PNH US/EU approval
Elevidys DMD EU filing
Ocrevus 6m SC RMS/PPMS US/EU approval
Regulatory
Susvimo DME/DR US filing
Vabysmo RVO EU approval
Xolair Food allergy US approval
tiragolumab + Tecentriq 1L PDL1+ NSCLC Ph III SKYSCRAPER-01
Venclexta + azacitidine 1L high risk MDS Ph III VERONA 2025
Columvi + GemOx 2L+ DLBCL Ph III STARGLO
Lunsumio + Polivy 2L+ DLBCL Ph III SUNMO 2025
Gazyva Lupus nephritis Ph III REGENCY
Enspryng generalized Myasthenia gravis Ph III LUMINESCE ( ) Not to be filed
Evrysdi + GYM 329 SMA Ph II MANATEE 2025
prasinezumab Parkinson’s disease Ph IIb PADOVA Next steps TBD
(CTAD)
Clinical results trontinemab Alzheimer’s disease Ph I/II BrainshuttleTM AD
vamikibart DME Ph II BARDENAS/ALLUVIUM 2025
ASO factor B Geographic atrophy Ph II GOLDEN STUDY
zilebesiran Hypertension Ph II KARDIA-2
CT-388 Obesity w/wo T2D (QW SC) Ph I
CT-868 T1D w. Obesity (QD SC) Ph II 2025
CT-996 Obesity w/wo T2D (QD oral) Ph I
● Itovebi (Inavolisib) US approval in1L PIK3CA-mut HR+ BC ● Columvi in 2L+ DLBCL filed with global regulators
Additional 2024 newsflow: ● Tecentriq EU approval in 1L NSCLC ineligible for platinum-based Tx ● Xofluza positive Ph III (Centerstone) in influenza transmission
*Outcome studies are event-driven: timelines may change ● Tecentriq + lurbinectedin positive Ph III (IMforte) in ES-SCLC maintenance 25
2025: Significant key newsflow ahead*
Compound Indication Milestone
inavolisib + palbociclib + fulvestrant 1L PIK3CA-mut HR+ BC EU approval
Columvi + GemOx 2L+ DLBCL US/EU approval
Lunsumio SC 3L+ FL US/EU approval
Elevidys DMD EU approval
illustrative
Business development
• BD to accelerate pipeline portfolio
• In-house pipeline with upside
Pharma pipeline potential
Note: Graph is purely conceptual to outline portfolio trends; 1 Pharma: On-market portfolio including young portfolio (products launched since end of 2015); COP=core operating profit 27
Unlocking the next era of innovation
3 Looking ahead
●
and beyond
12 pivotal Ph III readouts
● Multiple Ph III starts for novel NMEs
28
Doing now what patients need next