Rafael Holdings, Inc. · NYSE:RFL
Rafael — Investor Presentation (June 2021)
30 slides from Rafael's investor presentation, Jun 21, 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”
Slide 2: Safe Harbor Statement
Safe Harbor Statement 2 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 statements made pursuant to the Safe Harbor Provisions of the Private
Slide 3: Company Overview
Company Overview 3
Slide 4: Rafael Holdings Overview
Rafael Holdings Overview Rafael Holdings is comprised of interests in promising privately - held biotechnology companies targeting unmet medical needs in oncology, a cancer research institute, and commercial real estate properties PHARMACEUTICALS Investment in companies pursuing breakthrough therapi
Slide 5: Recently Announced Merger Will Create "new" Rafael Pharmaceuticals
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 inter
Slide 6: Rafael Pharmaceuticals is Focused on Cancer Metabolism
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 6
Slide 7: Rafael Pharmaceuticals Highlights
Rafael Pharmaceuticals Highlights 7 Focused Clinical - Stage Cancer Metabolism Therapeutics Company • Portfolio of novel cancer metabolism therapeutics in multiple indications • Advancing compounds from discovery through clinical development • Unique opportunity ahead of multiple value inflection po
Slide 8: Highly Experienced Leaders Recently Joined Rafael
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 and medical operations • Prior to this was SVP, Global Head of Marketi
Slide 9: Our Scientific Strategy in Cancer Metabolism
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 system to kill the tumor Inhibit PRO - CANCER immune guardians such as MD
Slide 10: Cancer Metabolism Pipeline Targeting Tumors with Novel Approaches
CPI - 613 (devimistat) • Phase 1 - 3 in multiple indications • Lead optimization for next generation compound Dual - SHMT inhibitor • Targeting T - ALL, and other tumor types • Orally available lead, Phase 1 planned for late 2022 Metabolic Immune Enhancer • Animal testing Autophagy inhibitor • Anima
Slide 11: World-Leading Scientific and Medical Advisors Shaping the Strategy and Pipeline
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 Medicine and Cell Biology, Northwestern University • Jorges Cortes, MD - Di
Slide 12: CPI-613 (devimistat)
CPI - 613® (devimistat) 12
Slide 13: CPI-613 (devimistat) Mechanism of Action
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 by tumor - drug retention • CPI - 613 ® (devimistat) turns off the mitoc
Slide 14: Devimistat: Key Attributes
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 typ
Slide 15: CPI-613 (devimistat) A Pipeline in a Molecule
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 – 40K patients 5K – 20K patients < 5K patients BI L IA R Y C O L O R E C
Slide 16: Devimistat Clinical Development Pipeline by Indication
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 + gemcitabin
Slide 17: Locally Advanced Pancreatic Cancer Potential to Improve Outcomes
17 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 18: Devimistat with Modified FOLFIRINOX in Metastatic Pancreatic Cancer
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 b
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 4
Slide 20: Devimistat with Modified FOLFIRINOX 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
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 = 262
Slide 22: CPI-613 May Significantly Improve Outcomes in Relapsed or Refractory AML
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 RISK S TR A T I FY A ll o g e n e i c T r a nsp l a n t • AML affects ~
Slide 23: Devimistat with HiDAC and Mitoxantrone in Relapsed or Refractory AML
CPI - 613® (devimistat) in Combination with High Dose Cytarabine (HiDAC) and Mitoxantrone (Salvage Trial) in Relapsed or Refractory AML Exhibits Promising Activity *Leuk Res. 2015 September ; 39(9): 945 – 949; **Phase I + Phase II data ELDERLY PATIENT POPULATION ( ≥ 50 YEARS) HISTORICAL COHORT* COMB
Slide 24: Devimistat with CHAM in r/r AML: Phase 3 Study Details
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 group 1
Slide 25: Clinical Experience in Relapsed or Refractory Burkitt Lymphoma
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 second - line therapy of hyper - CVAD and myeloablative allogeneic stem cell
Slide 26: Clinical Experience in T-Cell Lymphoma and Myelodysplastic Syndrome
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% 26 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 27: Devimistat is a Pipeline in a Molecule With Blockbuster Potential
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 cl
Slide 28: Key Priorities
Key Priorities 28
Slide 29: Key Priorities for the Next Months
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, and define ex - US strategy • Continue enrollment in r/r AML Phase 3 cl
Slide 30: Thank You
3 0 Thank You CONTACT: Barbara Ryan, Investor Relations barbara.ryan@rafaelholdings.com (203) 274 - 2825