Slide 1: Supplemental Slides ASH 2021: Phase 1b data in Waldenström's Macroglobulinemia
Supplemental Slides ASH 2021: Data from ongoing Phase 1b clinical trial in double- mutation patients with Waldenström’s Macroglobulinemia December 16, 2021
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,
Slide 3: Waldenström's Phase 1b Trial: Generating PoC in Patients with CXCR4MUT
Waldenström’s Phase 1b Trial: Generating PoC in Patients with CXCR4MUT Inclusion: Patients with MYD88 + CXCR4 mutations who are naïve to ibrutinib Design: Multi-national Phase 1b trial of mavorixafor in combination with ibrutinib (n=12 to 18) • Intrapatient dose-escalation: cycles of 200 mg, 400 mg,
Slide 4: ASH 2021: Data Support Mavorixafor Proof-of-Concept in Waldenström's
ASH 2021: Data Support Mavorixafor Proof-of-Concept in Waldenström’s Particularly in Refractory Patient Population 4
Slide 5: Response Rate: X4 ASH Data vs. Benchmarks of Ibrutinib Monotherapy
Response Rate: X4 ASH Data vs. Benchmarks of Ibrutunib Monotherapy After 6-Months of Treatment in R/R Patients with CXCR4 Mutations X4 ASH 2021 Poster X4 ASH 2021 Vs. Benchmarks Overall Response 100% 80% (n=21) Ibrutinib Monotherapy1 60% 40% (n=7) 20% Mav+Ibrutunib 0% ORR (minor+major) 1. Treon S et
Slide 6: Additional Patient-Level Data from X4's Trial
Additional Patient-Level Data from X4’s Trial Clinical Responses in Individual R/R Patients (N=7) (based on Best IgM Response) Minor Response Major response (PR/VGPR*) • Major Responses achieved after median 52 weeks on treatment * VGPR • Additional patients treated for median 30 weeks; all remain o
Slide 7: Patient Specific Information Related to Reported Safety in WM Phase 1b Trial
Patient Specific Information Related to Reported Safety in WM Phase 1b Trial Patient with Grade 5 Fatality: 81 yo with relapsing disease Patient with Cryptococcal Infection: 71 yo • Crypto-positive lung lesion identified before • Prior to study, patient required plasmapheresis and red treatment bloo
Slide 8: www.x4pharma.com
61 North Beacon Street 4th Floor www.x4pharma.com Boston, MA 02134 8