Vor Biopharma Inc.NASDAQ: VOR

44th Annual J.P. Morgan Healthcare Conference Presentation (a19b30)

· Issued by Vor Biopharma Inc.
Global Science. One Purpose.




44th Annual J.P. Morgan Healthcare Conference

January 2026

Immune Remodulation Through BAFF/APRIL Inhibition

OUR AMBITION

To Transform the Approach to B cell-Driven Autoimmune Disease

TELITACICEPT

Selective BAFF/APRIL inhibitor designed to reduce pathogenic B cells and antibodies while preserving immune protection

LEAD AUTOIMMUNE

PROGRAMS

Myasthenia Gravis: Global Phase 3 topline data in 1H27

Sjögren's Disease: Global Phase 3 to initiate in 1H26

NEAR-TERM EXPANSION OPPORTUNITIES

Broad applicability across B cell-mediated autoimmune diseases

Clinically validated in 8+ autoimmune indications in China Manageable safety and tolerability in tens of thousands of patients

$450M WITH RUNWAY INTO MID-2028*, FUNDED THROUGH ALL KEY CATALYSTS

3 *Pro forma cash, cash equivalents, and short-term investments as of December 2025

Dual BAFF/APRIL Inhibition Targets Autoreactive B Cells and Plasma Cells

Disease modification through upstream control of B cell survival and downstream antibody production



Telitacicept

TACI-Fc fusion protein:

dual inhibition of BAFF/APRIL

Blocking BAFF inhibits abnormal development and maturation of B cells

Telitacicept preferentially disrupts BAFF-dependent autoreactive B-cell maturation and survival thereby reducing disease-driving immune activity

BAFF APRIL

Blocking APRIL inhibits abnormal production of antibodies by plasma cells

Telitacicept inhibits APRIL-dependent differentiation of mature B cells into plasma cells thereby minimizing autoantibody production and contributing to control of disease

Antibodies



Regulated by BAFF / BLyS

Regulated by APRIL

4 doi.org/10.3390/cancers12041045

BAFF, B-cell activating factor; BLyS, B Lymphocyte Stimulator; APRIL, a proliferation inducing ligand

Established Efficacy in China Across Autoimmune Diseases

3 2 3

Commercial Approvals BLA Submissions Best-In-Disease

Validated Commercial Therapy in China Across Diverse Autoimmune Diseases

2021 - Systemic Lupus Erythematosus (SLE)†

2024 - Rheumatoid Arthritis (RA)

2025 - Myasthenia Gravis (MG)

Poised to Further Expand Telitacicept Footprint in Large, Underserved Diseases in China

Filed 2025 - Sjögren's Disease (SD)

Filed 2025 - IgA Nephropathy (IgAN)*

Unique Dual BAFF/APRIL Inhibition Drives Superior Clinical Benefit

Systemic Lupus Erythematosus Myasthenia Gravis

Sjögren's Disease

5 † Conditional Approval, Full Approval in 2023; *Accelerated Approval in China

10s of Thousands

Patients Treated

Commercially in China

Favorable Safety At Scale

Favorable and Predictable Safety Profile Observed Among ~1,800* Patients Studied in Clinical Trials

No Burdensome Vaccination Requirements

No Signature B Cell Depletion Associated SAEs

Mild to Moderate AEs

Upper respiratory tract infection

Injection site reaction

Urinary tract infection

Cough

Diarrhea

Frequency (%) of safety events reported in clinical trials

Telitacicept (n=1211)

35

17

10

5

5

Placebo (n=527)

30

2

9

3

5

*From pooled safety analysis across all telitacicept trials.

6 AEs, adverse events; SAEs, serious adverse events.

Advancing the Leading BAFF/APRIL Inhibitor

Strong cash position of $450M* with runway into mid-2028 expected to cover key milestones

Vor Indications

Marketing Approval Milestones

Phase 3

Phase 2

Phase 1

Preclinical

Phase 3

Myasthenia Gravis (MG) Global Phase 3 Topline Data (1H27)

Phase 3 Ready

Sjögren's Disease (SD) Global Phase 3 Trial Initiation (1H26)

RemeGen Indications

Preclinical

Phase 1

Phase 2

Phase 3

Marketing Approval Milestones

China Marketed

Myasthenia Gravis (MG) OLE 48-wk Data - AANEM (10.29.25)

BLA Accepted



Sjögren's Disease (SD) LBA Poster Presentation - ACR (10.28.25)

BLA Submitted



IgA Nephropathy (IgAN) LBA Oral Presentation - ASN (11.8.25)

China Marketed

Systemic Lupus Erythematosus (SLE) NEJM Publication (10.16.25)

Phase 3

China Marketed

Rheumatoid Arthritis (RA) Neuromyelitis Optica Spectrum

Disorder (NMSOD)

Phase 2

Lupus Nephritis (LN)

Phase 2



Membranous Nephritis (MN) and Other Indications

7 *cash and cash equivalents as of December 2025

Vor - Global Trial RemeGen - China Trial

Primary Endpoint Achieved



Myasthenia Gravis

Moving Beyond IgG Therapies

8



Myasthenia Gravis: The Beachhead Indication

Telitacicept demonstrated depth, durability, and a differentiated upstream mechanism

Across leading mechanisms, telitacicept demonstrates largest placebo-adjusted MG-ADL improvement

24- and 48-week data show continued improvement, suggesting patient benefit deepens over time

Upstream disease control with consistent safety and tolerability over one year of therapy

PLACEBO- ADJUSTED MG- ADL CHANGE FROM BASELINE AT WEEK 24

0

PBO-adjusted LSM MG-ADL Change from Baseline

-1

-2

-3

-4

-5 -4.8

-6

-7

-8

Telitacicept

Efgartigimod

Nipocalimab

Rozanolixizumab

Ravulizumab

Eculizumab

Zilucoplan

Inebilizumab

Based on historical clinical data; not a head-to-head trial

9 gMG, generalized myasthenia gravis; Efgartigimod - ADAPT; Nipocalimab - Vivacity-MG3; Rozanolixizumab - MycarinG; Ravulizumab - CHAMPION-MG; Eculizumab - REGAIN; Zilucoplan - RAISE; Inebilizumab - MINT RemeGen-sponsored trial

Telitacicept Demonstrated Durable MG-ADL Over Time

Sustained and deepening functional improvement through 48 weeks

MEAN CHANGE IN MG- ADL SCORE

1

OLE Period

0 Patients in placebo group

PERCENTAGE OF PATIENTS ACHIEVING IMPROVEMENT IN MG- ADL BY SCORE THRESHOLD AT WEEK 48

Mean change from baseline (95% CI)

-1

-2

-3

-4

-5

-6

-

-7

-8 -7.5

-9

-1.6

-6.4

switched to telitacicept*

-6.3

-7.5

8

-52.3%

-64.2%

-86.8%

-94.3%

-96.2%

-96.2%

-100.0%

7

MG-ADL Score Improvement

6

5

4

3

2

-100% -80% -60% -40% -20% 0%

0 4 8 12 16 20 24 28 32 36 40 44 48

Weeks

Percentage (%)

Telitacicept 240 mg, 48 Weeks

Telitacicept 240 mg, 24 Weeks → Telitacicept 240 mg, 24 Weeks

Placebo 24, Weeks → Telitacicept 240 mg, 24 Weeks

* Telitacicept arm continue with the same treatment, Placebo arm switched to Telitacicept during OLE period. The efficacy analysis was based on descriptive statistical analysis of the actual data in the full analysis set (FAS), and missing data were not filled.

10

RemeGen-sponsored trial

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