Rafael Holdings, Inc. · NYSE:RFL
Rafael — Investor Presentation (August 2021)
31 slides from Rafael's investor presentation, Aug 10, 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” August 2021
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 • Developing pipeline of novel cancer metabolism therapeutics in multiple indications • Advancing compounds from discovery through
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 Copyright @ 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 te
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: (devimistat)
11 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. CPI - 613® (devimistat)
Slide 12: (devimistat) 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 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. CPI - 613 ® (devimistat): Key Attributes • Appears to be minimally toxic to healthy cells • Potential for extended treatment • Can be used in combination • Potential to treat elderly patients • Opportunity across lines of therap
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 + gemcitab
Slide 16: Locally Advanced Pancreatic Cancer: Potential to Improve Outcomes
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
17 CPI - 613 ® (devimistat) in Combination with Modified FOLFIRINOX in Metastatic Pancreatic Cancer 19 . 9 gemcitabine g e m c i t a b i n e + n a b - paclitaxel FOLFIRINOX m F O LF I R I N O X + devimistat Median Overall Survival (months) 6 . 8* 11.1 * 8.5 ** gemcitabine g e m c i t a b i n e + n a
Slide 18: Most Common Adverse Events in Metastatic Pancreatic Cancer
18 Most common grade 3 or higher adverse events occurring in >10% of patients treated with CPI - 613 ® (devimistat) in metastatic pancreatic cancer ** Lan c e t O n c o l 2017 ; 18 : 77 0 - 78 ; pa ti en t t r ea t e d a t M T D ( 500 m g / m 2 ) Phase 1 study of devimistat monotherapy (N=9) * 2 dea
Slide 19: Devimistat with Gemcitabine and Nab-paclitaxel in Pancreatic Cancer
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)* devimistat + g e m c i t a bin e + N % nab - paclitaxel CR 1 5% PR 9 45% SD 8
Slide 20: Maximum Percent Change of Tumor Size in Locally Advanced Pancreatic Cancer
20 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 21: Phase 3 Study in First-Line Metastatic Pancreatic Cancer
21 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 22: Being Studied as a Potential Treatment for Relapsed or Refractory AML
22 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. CPI - 613® is Being Studied as a Potential Treatment for 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
Slide 23: Devimistat with HiDAC and Mitoxantrone in Relapsed or Refractory AML
*Leuk Res. 2015 September ; 39(9): 945 – 949; represents analysis done on patients ≥ 50 years of age **Phase I + Phase II data Note: There have been no completed comparative randomized clinical trials between devimistat and other agents, data is intended only to provide context on historical cohorts
Slide 24: Most Common Adverse Events in r/r AML
24 Most common grade 3 or higher adverse events occurring in >10% of patients treated with CPI - 613 ® (devimistat) in combination with r/r acute myeloid leukemia (AML) ** Lan c e t O n c o l 2017 ; 18 : 77 0 - 78 ; pa ti en t t r ea t e d a t M T D ( 500 m g / m 2 ) * 2 dea t h s o cc u rr e d dee
Slide 25: Devimistat with CHAM in r/r AML: Phase 3 Study Details
25 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 26: Clinical Experience in Relapsed or Refractory Burkitt Lymphoma
* Case Report #1 from patient in prior Phase 1 clinical trial, Case Report #2 from patient ongoing Phase 2 clinical trial. These represent best results to date and are not typical. 26 Copyright @ 2021. Rafael Holdings, Inc. Rafael Pharmaceuticals, Inc. Case Report #2*: Patient with Relapsed/Refracto
Slide 27: Clinical Experience in T-Cell Lymphoma and Myelodysplastic Syndrome
27 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 28: Devimistat is a Pipeline in a Molecule With Blockbuster Potential
28 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 29: Key Priorities
29 Key Priorities
Slide 30: Key Priorities for the Next 18 Months
30 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 31: Thank You
Thank You RAFAE CONTACT: Pei Barbara Ryan, Investor Relations barbara.ryan@rafaelholdings.com (203) 274-2825
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