Rafael Holdings, Inc. · NYSE:RFL
Rafael — Investor Presentation (July 2021)
29 slides from Rafael's investor presentation, Jul 14, 2021
Slide 1: Harnessing the Power of Cancer Metabolism to Develop Innovative Therapeutics
1 Harnessing the Power of Cancer Metabolism to Develop Innovative Therapeutics that Improve and Extend the Lives of Patients “To Save A Life Is To Save A Universe” July 2021 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc .
Slide 2: Safe Harbor Statement
2 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. Safe Harbor Statement This presentation of Rafael Holdings, Inc (Rafael) contains forward - looking statements. Statements that are not historical facts are forward - looking statements and such forward - looking statements are s
Slide 3: Company Overview
3 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, In c . Company Overview
Slide 4: Recently Announced Merger Will Create "new" Rafael Pharmaceuticals
4 Recently Announced Merger Will Create “new” Rafael Pharmaceuticals RAFAEL HOLDINGS,INC. • Rafael Pharmaceuticals will be merged with Rafael Holdings to create a focused late - stage cancer metabolism therapeutics company • Lead investor, Howard Jonas, will be Chair of merged company • Majority int
Slide 5: Rafael Pharmaceuticals is Focused on Cancer Metabolism
5 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. Rafael Pharmaceuticals is Focused on Cancer Metabolism Rafael Pharmaceuticals is a clinical - stage oncology company focused on selectively targeting cancer metabolic pathways while simultaneously harnessing the immune system
Slide 6: Rafael Pharmaceuticals Highlights
6 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. Rafael Pharmaceuticals Highlights Focused Clinical - Stage Cancer Metabolism Therapeutics Company • Portfolio of novel cancer metabolism therapeutics in multiple indications • Advancing compounds from discovery through clinical d
Slide 7: Highly Experienced Leaders Recently Joined Rafael
7 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. Highly Experienced Leaders Recently Joined Rafael Ameet Mallik Chief Executive Officer • 20+ years of biopharma experience • Most recently EVP and Head of the ~$6B U.S. Business at Novartis Oncology responsible for all commercial
Slide 8: Our Scientific Strategy in Cancer Metabolism
8 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. Our Scientific Strategy in Cancer Metabolism: 3 - Pronged Approach to Tip the Balance Specifically target CANCER CELL METABOLIC PATHWAYS Stimulate or preserve CD8, CTL, NK and M1 macrophages to harness the ANTI - CANCER immune sy
Slide 9: Cancer Metabolism Pipeline Targeting Tumors with Novel Approaches
9 C opyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. CPI - 613 (devimistat) • Phase 1 - 3 in multiple indications Dual - SHMT inhibitor • Targeting T - ALL, and other tumor types • Orally available lead, Phase 1 planned for late 2022/early 2023 Metabolic Immune Enhancer • Animal t
Slide 10: World-Leading Scientific and Medical Advisors Shaping the Strategy and Pipeline
10 Copyright @ 2021 Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. World - Leading Scientific and Medical Advisors Shaping the Strategy and Pipeline • Richard Axel, MD - Nobel Laureate, Professor at Columbia University, Genentech and Rafael Board member • Navdeep Chandel, PhD – Professor of Medi
Slide 11: CPI-613 (devimistat)
11 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. CPI - 613® (devimistat)
Slide 12: Mechanism of Action
12 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. CPI - 613® (devimistat) Mechanism of Action • CPI - 613 ® (devimistat) is an analog of normally transient, acylated catalytic intermediates of the enzyme cofactor lipoate • CPI - 613 ® (devimistat) tumor selectivity is enhanced
Slide 13: CPI-613 (devimistat): Key Attributes
13 CPI - 613 ® (devimistat): Key Attributes • Minimally toxic to healthy cells (well tolerated) • Allows for extended treatment • Can be used in combination • Potential to treat elderly patients • Opportunity across lines of therapy • Potential to increase responses in traditionally resistant tumor
Slide 14: (devimistat): A Pipeline in a Molecule
14 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. CPI - 613® (devimistat): A Pipeline in a Molecule ADDRESSABLE PATIENT POPULATION OF >100K Patients in the U.S. T O D A Y T O M OR R O W PIVOTAL PHASE 3 TRIALS UNDERWAY ADDITIONAL INDICATIONS UNDER DEVELOPMENT > 40K patients 20K
Slide 15: Devimistat Clinical Development Programs by Indication and Phase
15 Product Indication Phase I Phase II Phase III SOLID TUMORS devimistat + mFOLFIRINOX First - Line Metastatic Pancreatic Cancer* devimistat + mFOLFIRINOX First - Line Locally Advanced Pancreatic Cancer* devimistat + hydroxychloroquine Relapsed or Refractory Clear Cell Sarcoma # devimistat + gemcita
Slide 16: Pancreatic Cancer: Potential to Improve Outcomes in the Majority of Cases
16 Locally Advanced Pancreatic Cancer CPI - 613® (devimistat) has the Potential to Improve Outcomes in the Majority of Pancreatic Cancers M et a s ta t i c P anc r e a t i c Canc e r FIRST - LINE: Phase 3 Trial: A V E N G E R 500 ® ( N =528 ) devimistat + mFOLFIRINOX vs.FOLFIRINOX Phase 1 Trial: dev
Slide 17: Devimistat with Modified FOLFIRINOX in Metastatic Pancreatic Cancer
Source: *N Engl J Med 2011;364:1817 - 25.; ** N Engl J Med 2013;369:1691 - 703, † Not Evaluated; # Lancet Oncol 2017; 18: 770 - 78 17 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. CPI - 613 ® (devimistat) in Combination with Modified FOLFIRINOX in Metastatic Pancreatic Cancer
Slide 18: Devimistat with Gemcitabine and Nab-paclitaxel in Pancreatic Cancer
18 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. CPI - 613® (devimistat) in Combination with gemcitabine and nab - paclitaxel in Locally Advanced or Metastatic Pancreatic Cancer R e s pon s e S u mma r y Comparison with gemcitabine + nab - paclitaxel (Historical Data)* devimis
Slide 19: Devimistat with Modified FOLFIRINOX in Locally Advanced Pancreatic Cancer
19 50 . 00% 40 . 00% 30 . 00% 20 . 00% 10 . 00% 0 . 00% - 10.00% - 20.00% - 30.00% - 40.00% - 50.00% - 60.00% 11 17 10 7 25 13 40 26 20 3 9 2 22 35 21 24 38 37 27 34 12 28 39 14 16 15 29 4 36 33 18 6 19 5 Patient Number (N = 36) Change of Tumor Size from Baseline (%) Maximum Percent Change of Tumor
Slide 20: Phase 3 Study in First-Line Metastatic Pancreatic Cancer
20 CPI - 613® (devimistat) Phase 3 Study in Combination with Modified FOLFIRINOX in First - Line Metastatic Pancreatic Cancer O N GO IN G S T U D Y NCT03504423 First - Line Metastatic Pancreatic Adenocarcinoma Randomization (1:1) N= 528 Arm 1 ( T e s t Ar m ) N = 266 Arm 2 (Co n t r o l Ar m ) N = 2
Slide 21: May Significantly Improve Outcomes in Relapsed or Refractory AML
21 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. CPI - 613® May Significantly Improve Outcomes in Relapsed or Refractory AML AML TREATMENT PARADIGM RISK S TR A T I FY Co m p l e t e R e s pon s e I ndu c ti o n Therapy Primary R e f r a c t o r y Salvage T h e r a p y Relapse
Slide 22: Devimistat with HiDAC and Mitoxantrone in Relapsed or Refractory AML
*Leuk Res. 2015 September ; 39(9): 945 – 949; **Phase I + Phase II data 22 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. CPI - 613® (devimistat) in Combination with High Dose Cytarabine (HiDAC) and Mitoxantrone (Salvage Trial) in Relapsed or Refractory AML Exhibits Promising A
Slide 23: Devimistat with CHAM in r/r AML: Phase 3 Study Details
23 CPI - 613® (devimistat) in Combination with CHAM in r/r AML: Phase 3 Study Details R el aps e d/ R e f r a c t o r y AML Randomization (1:1) N= 500 Arm 1 ( T e s t Ar m ) N = 25 0 Arm 2 (Co n t r o l Ar m ) N = 25 0 CPI - 613® (devimistat) + high dose Cytarabine + Mitoxantrone (CHAM) Control grou
Slide 24: Clinical Experience in Relapsed or Refractory Burkitt Lymphoma
24 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. Clinical Experience of CPI - 613 ® (devimistat) in Relapsed or Refractory Burkitt Lymphoma B e f o r e d e v imi s t a t : • The patient was suffering from second relapse after first - line therapy in CALGB 10002 study and secon
Slide 25: Clinical Experience in T-Cell Lymphoma and MDS
25 Clinical Experience of CPI - 613 ® (devimistat) in T - Cell Lymphoma and Myelodysplastic Syndrome (MDS) CRi M a r r o w CR SD PD N* 1 1 8 2 % 8% 8% 67% 17% bendamu s t i ne ( B E N T L Y T r i a l * ) devimistat + b e nd a m u s t i ne ORR 50% 75% M ed i a n OS 6. 2 M o n t h s 9. 2 M o n t h s M
Slide 26: Devimistat is a Pipeline in a Molecule With Blockbuster Potential
26 CPI - 613® (devimistat) is a Pipeline in a Molecule With Blockbuster Potential • Clinical development programs underway across multiple indications have potential to treat patient populations exceeding 100,000 in the U.S. and significantly more ex - U.S. • Data readout from registrational Phase 3
Slide 27: Key Priorities
27 Key Priorities
Slide 28: Key Priorities for the Next 18 Months
28 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc . Key Priorities for the Next 18 Months Maximize Potential of CPI - 613® (devimistat) • Readout Phase 3 clinical trial in first - line Metastatic Pancreatic Cancer in 4Q’21, submit NDA in 1H’22, prepare for U.S. commercial launch
Slide 29: Thank You
2 9 Thank You CONTACT: Barbara Ryan, Investor Relations barbara.ryan@rafaelholdings.com (203) 274 - 2825