An Affiliate of Rafael Holdings (NYSE:RFL) Strictly …...Harnessing the Power of Cancer Metabolism...

Harnessing the Power of Cancer Metabolism to Develop Innovative Oncology Therapeutics

June 2019

An Affiliate of Rafael Holdings (NYSE:RFL)

“To Save A Life Is To Save A Universe” Strictly Confidential

2019 Healthcare Conference

Safe Harbor Statement

This presentation contains forward-looking statements. These statements relate to future events or the company’s future financial performance. Insome cases, you can identify forward-looking statements by terminology such as "may", "will", "should", "expect", "plan", "anticipate", "believe","estimate", "predict", "potential" or "continue", the negative of such terms, or other comparable terminology. These statements are only predictions.Actual events or results may differ materially from those in the forward-looking statements as a result of various important factors. Although webelieve that the expectations reflected in the forward-looking statements are reasonable, such statements should not be regarded as arepresentation by the company, or any other person, that such forward looking statements will be achieved. The business and operations of thecompany are subject to substantial risks which increase the uncertainty inherent in forward-looking statements. We undertake no duty to updateany of the forward-looking statements, whether as a result of new information, future events or otherwise. In light of the foregoing, readers arecautioned not to place undue reliance on such forward-looking statements.

2Copyright © 2019. Rafael Pharmaceuticals, Inc. Confidential and Proprietary.

Table of Contents

Introduction: Company Overview

CPI-613® (devimistat): Mechanism of Action

CPI-613® (devimistat) in Pancreatic Cancer

CPI-613® (devimistat) in Acute Myeloid Leukemia

CPI-613® (devimistat) in Other Hematological Malignancies

Commercial Plan


Introduction: Company Overview

Company Overview

Company Clinical Stage, Metabolic Oncology Therapeutics Company

Mission To develop innovative, highly selective, well tolerated and highly effective anti-cancer agents by selectively targeting altered metabolism in cancer cells

Our Platform Altered Metabolism Directed (AMD) drugs disrupt widely occurring, tumor-specific, regulation of glucose and glutamine conversion to building block molecules and energy

Our Lead Molecule CPI-613® (devimistat) in multiple clinical trials for different solid tumors and hematological malignancies

Important Milestones2 Ongoing Phase III pivotal trials of CPI-613® (devimistat): AVENGER 500: Pancreatic Cancer ARMADA 2000: Acute Myeloid Leukemia

Our Management Extensive experience in discovery, development and commercialization of oncology products

Copyright © 2019. Rafael Pharmaceuticals, Inc. Confidential and Proprietary. 5

Clinical Trial Status

Ongoing Phase III trials: Pancreatic Cancer, Acute Myeloid Leukemia (AML) Ongoing Phase I & II trials: Peripheral T-Cell Lymphoma (PTCL), Burkitt / High-Grade B-cell

Lymphoma, Myelodysplastic Syndromes (MDS) and several other trials in hematological malignancies and solid tumors

Estimated NDA Submission: 2020

Orphan Drug Designation (ODD)FDA: Pancreatic Cancer, AML, PTCL, Burkitt’s Lymphoma and MDSEMA: Pancreatic Cancer and AMLOnly Oncology Company with 5 ODDs in U.S. and 2 in EU

Intellectual Property Protected until 2029 and beyond across U.S., Canada, EU, Israel, Australia and major markets of Asia (China, Hong Kong, Japan, South Korea, Taiwan)

Other Products in Pipeline RFL-618 (oral CPI-613®) Internal Discovery Compounds: pre-clinical studies are ongoing for several NCEs

More than 350 patients dosed with CPI-613® (devimistat) to date in 16 ongoing or completed clinical trials; another 5 new trials started enrolling participants (including 2 Phase III trials)

CPI-613® (devimistat): A Snapshot


CPI-613® (devimistat): Ongoing / Upcoming Clinical Trials

Preclinical PHASE I PHASE I/II PHASE II PHASE III Milestones*

Solid

Tum

ors

Interim Analysis for Safety & Efficacy:

Q2 2020

FDA: 2020

Phase II/III is expected to start in

2019

Hem

atol

ogic

al M

alig

nanc

ies Interim Analysis for

Safety & Efficacy: Q2 2020

Expected NDA Submission:

Q2 2020

Will be completed by 2019 Q2, pivotal

study will start in Q3

6 Ongoing Trials and 2 Upcoming Trial in Solid Tumors & Hematological Malignancies

First-Line Metastatic Pancreatic Cancerdevimistat + mFOLFIRINOX Randomized (~107 Sites)

First-Line Locally Advanced Pancreatic Cancerdevimistat + mFOLFIRINOX

Metastatic Colorectal Cancer devimistat + FOLFOX + bevacizumab

First-Line Locally Advanced or Metastatic Pancreatic Cancer

devimistat + gemcitabine + nab-paclitaxel

Relapsed or Refractory Acute Myeloid Leukemia (AML)

devimistat + cytarabine + mitoxantrone

Randomized(~87 Sites)

Relapsed or Refractory Burkitt’s Lymphoma / Leukemia or High-grade B-cell Lymphomadevimistat

Relapsed or Refractory Myelodysplastic Syndrome (MDS)

devimistat + hydroxychloroquine

Relapsed or Refractory T-Cell Lymphomadevimistat + bendamustine

Ongoing

Ongoing

Ongoing

Ongoing

Ongoing

Ongoing

*As per internal estimationCopyright © 2019. Rafael Pharmaceuticals, Inc. Confidential and Proprietary. 7

2020* 2025*

AML

Pancreatic Cancer

Burkitt / High-Grade B-cell Lymphoma

T-Cell Lymphoma

Myelodysplastic Syndrome

Future Indications:Other

Gastrointestinal Cancers (e.g. Colon,

Gastric)

Step 1

Burkitt’s/High-Grade B-cell Lymphoma/ T-Cell Lymphoma

(Accelerated Path to Approval)

Step 2

Acute Myeloid Leukemia & Pancreatic Cancer

U.S.:Estimated

3,122Patients/Year1-3

U.S.:Estimated

10,836Patients/Year4-6

U.S.:Estimated

21,380 Patients/Year7

U.S.:Estimated

53,670Patients/Year8

U.S.:Estimated

13,000 Patients/Year9

Step 3

Myelodysplastic Syndrome

Ongoing Phase III registrational trials

Step 4

Other Formulations & IndicationsCPI-613® (devimistat)

Sustainable Horizons for Growth:Targeting Large Unmet Need & Orphan Indications in Terms of Patient Pool

CHINA:Estimated 27,710 Patients/Year10

JAPAN:Estimated ~15,552

Patients/Year14

CHINA:Estimated 80,344Patients/Year11

JAPAN:Estimated 39,800 Patients/Year15

CHINA:Estimated

310,244 (Colon)12; 679,100 (Gastric)13

Patients/Year

JAPAN:Estimated 135,800

(Colon/Rectal)16; 133,000 (Gastric)17

Patients/Year

EUROPE:Estimated 132,559

Patients/Year19EUROPE:Estimated 30,000 Patients/Year18


*As per internal estimation

Experienced Leadership Team:Oncology Development & Commercialization

Sanjeev Luther President & CEO

25+ years experience in healthcare including spec pharma, bio-pharma etc. in strategy, BD, alliance, commercialization and operations

Under his leadership Rafael has made significant progress towards strategy, R&D, clinical development and business portfolio optimization

Robert G. L. Shorr, PhD, DIC Chief Scientist/Co-founder

40-year track record in drug discovery from concept through approval, market launch Enzon Pharma, responsible for co-development of PEG INTRON A with Schering Plough

Timothy S. Pardee, MD, PhD Chief Medical Officer

Physician-Scientist with an NCI funded research program focused on metabolic contributions to resistance in AML

Played a lead role in the development of the novel metabolic-targeting agent devimistat

Mike Hu, Ph.D.Chief Development Officer

18 years of experience in clinical and pre-clinical research, pharmaceutical drug development, and academia

Contributed to the development, submission, approval and life cycle management of 18 drugs in various indications

Mona M. Wahba, MD, MSM Senior VP Clinical Operations

31 years of experience in pharmaceutical industry Associated with global submissions, approvals, launch and growth of Lonsurf, Xofigo, Stivarga,

Ilaris, Reclast, Orencia, Bextra, Celebrex, Viagra and Tykson

Paul Bingham PhD VP, Research

Co-discoverer of first AMD compound Faculty member, Stony Brook University; PhD, Harvard University

Rumin Zhang PhD VP, Discovery

28 years of experience in drug discovery Head of research for the discovery of many drug candidates in diverse therapeutic area including

targeted cancer therapy and immuno-oncology

Jehan Rowlands, Pharm.D. VP, Regulatory Affairs

19 years of experience in regulatory affairs Worked closely with the FDA to develop and execute regulatory strategies for drug candidates,

associated with the development of Namenda and Natpara

Mike StelmahVP, Manufacturing and CMC

Regulatory Affairs

Extensive experience of CMC and regulatory strategies for pharmaceutical industry Associated with the submission of IMPD, IND, DHF, MAA, NDA and other technical files to multiple

health authorities including FDA, EMA, HPRA and PMDA


CPI-613® (devimistat): Mechanism of Action (MoA)

pyruvate

citrate

isocitrate

α-ketoglutaratesuccinyl-CoA

oxaloacetate

acetyl-CoA

succinate

fumarate

malate

glucose

glutamate

glutamine

PDH

KGDH

LipidsProteinsNucleic Acids

Cancer cells extensively reconfigure normal metabolism:

re-regulated carbon flux for controlled provision of metabolic building blocks needed for growth

Increased requirement for carbon backbones

Two major re-regulated control points input virtually all carbon into cancer cell Tricarboxylic Acid (TCA) cycle:

Pyruvate Dehydrogenase (PDH); inputs pyruvate carbon Alpha-Ketoglutarate Dehydrogenase (KGDH); inputs glutamine-derived

carbon

Multi Targeted Approach – selectively blocks PDH and KGDH, through their tumor cell re-regulation, each by a distinct mechanism, ultimately

triggering cell death, while sensitizing to most other drugs.

Copyright © 2018. Rafael Pharmaceuticals, Inc. Confidential and Proprietary.

devimistat

MoA: CPI-613® (devimistat) Targeting is Highly Selective for Tumor Cells, Producing High Therapeutic Index & Very Low Clinical Toxicity

11

This powerful approach to target cancer metabolism is currently in development

CPI-613® (devimistat): Key Differentiators

devimistat

Highly Selective:selectively targets altered metabolism in cancer cells

Less Toxic: minimally toxic to healthy cells (highly safe & tolerable)

Allows for extended treatment courses (reduces likelihood of relapse)

Can be used in combination with other drugs Potential to treat elderly patients Potential to treat surgically unresectable cancers

Attacks Multiple Targets

(PDH, KGDH)

Potential to treat different cancers including: Difficult-to-treat cancers High risk cancers Treatment-resistant

cancers Advanced stage cancers

etc.

Less vulnerable to emergence of drug

resistance

Broad Spectrum Activities:

altered metabolism observed in different types of cancers

Targets Metabolic Pathway

Unique target that increases responses in traditionally resistant tumor types

Expected to suppress metabolism-based resistance to companion Standard of Care(s)


CPI-613® (devimistat) in Pancreatic Cancer: Preclinical, Clinical Experience and Ongoing Trials

Pancreatic Cancer Strategy:Immediate Goal to Become a Pancreatic Cancer Company

Metastatic PANC, 45%

Locally advanced

PANC, 35%

Borderline resectable PANC, 10%

Resectable PANC, 10%

2018 Q4Pancreatic Cancer (devimistat + mFOLFIRINOX)Global Phase III Randomized Registrational Trial in First-line Metastatic Pancreatic Cancer

2018 Q4:Pancreatic Cancer (devimistat + gemcitabine + nab-paclitaxel)Phase I/II IST for Metastatic or Locally Advanced Pancreatic Adenocarcinoma

2018 Q4Pancreatic Cancer (devimistat + mFOLFIRINOX): Neoadjuvant TherapyPhase II IST for Locally Advanced Pancreatic Adenocarcinoma

Near Future:Second-line Cross Over with FOLFIRINOX & gemcitabine

Future:devimistat in Combination with Immuno-oncology Therapies

Rafael plans to investigate devimistat in all segments of pancreatic adenocarcinoma to become a ‘Pancreatic Cancer Company’

Ongoing


Ongoing

Ongoing

Significant Synergy is Observed withFOLFIRINOX, nab-paclitaxel (Abraxane) or gemcitabine

FOLFIRINOX = 25μM oxaliplatin, 30μM irinotecan,100μM 5-FU in RPMI/2mM glucose, 48 hrs.

We expect CPI-613® (devimistat) suppression of oncogene signaling to engender synergy with most Standard of Care (SoC), including radiation, RTK inhibitors, and immunotherapy

This in vitro synergy is consistent with the strong in vivo synergy observed in PDAC clinical trials


Unpublished data

devimistat enhancement of Gemcitabine

Unpublished data

Phase I Study - CPI-613® (devimistat) in Combination with Modified FOLFIRINOX in Metastatic Pancreatic Cancer: Enrolled More Older & Sicker Patients

Characteristic FOLFIRINOX (ACCORD11/PRODIGE4; N Engl J Med 2011;364:1817-25.) devimistat + mFOLFIRINOX

N 171 20Age- Yrs

Median 61 65.5Range 25-76 48-73

Sex % NMale 62% 55%

Female 38% 45%ECOG Score % N

0 37% 15%1 62% 85%2 0.6% 0.0%

Tumor location % NHead 39% 60%Body 31% 10%Tail 26% 30%

Multicentric 4% 0%Biliary Stent % N

Yes 16% 10%No 84% 90%

No. of metastatic site involvedMedian 2 1Range 1-6 1-4

CA-19-9 level % NNormal 15% 10%

Elevated <59xULN 44% 30%Elevated >59xULN 42% 60%

Unknown 0% 0%No: of metastatic sites % N

Liver 88% 100%lung 19% 30%

peritoneal 19% 30%

In devimistat phase I study, patients were older, had

more patients who were sicker (higher

ECOG score and elevated CA19-9

>59xULN) compared with

ACCORD11/PRODIGE4 study


Phase I Study - CPI-613® (devimistat) in Combination with Modified FOLFIRINOX in Metastatic Pancreatic Cancer: Percent Growth or Reduction of Tumor Size & Response*

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

0%20%40%60%80%

100%120%140%

Cha

nge

of T

umor

Siz

e fr

om B

asel

ine

(%)

Patient Number (N = 18)

Maximum Percent Change of Tumor Size from Baseline

*RECIST guideline version 1.1†As per RECIST guideline version 1.1, PR was defined as at least a 30% decrease in the sum of diameters of target lesions, taking the baseline sum diameters as reference

- 30%†

CR: 17%PR: 44%SD: 17%PD: 22%


Phase I Study - CPI-613® (devimistat) in Combination with Modified FOLFIRINOX in Metastatic Pancreatic Cancer: Survival Statistics


Overall Survival (OS) Progression-Free Survival (PFS)

Median OS: 19.9 months Median PFS: 9.9 months

Phase I Study - CPI-613® (devimistat) in Combination with Modified FOLFIRINOX in Metastatic Pancreatic Cancer: Comparison with FOLFIRINOX (Historical Data)*

11.119.9

FOLFIRINOX mFOLFIRINOX + devimistat

Median Overall Survival (months)

6.49.9


Median Progression-Free Survival (months)

5.99.2


Median Duration of Response (months)

31.6%61.0%


Objective Response Rate (ORR)

*Historical Data: N Engl J Med 2011;364:1817-25; #Relative Increase


93%#

79%#55%#

56%#

CPI-613® (devimistat) in Combination with Modified FOLFIRINOX in MetastaticPancreatic Cancer: Phase 3 Study Details


NCT03504423

First-Line Metastatic Pancreatic

Adenocarcinoma

Randomization (1:1) N= 500

Arm 1 (Test Arm) N= 250

Arm 2 (Control Arm) N= 250

devimistat + Modified FOLFIRINOX

FOLFIRINOX

Primary Endpoints: PFS ORR

Secondary Endpoints: OS DOR Safety PK PROs

Potential Timelines:

Q2 2020 (Futility)

Q4 2020 (Interim)

H2 2021 (Final)

Patient Population: Female and male patients with Metastatic (Stage IV) adenocarcinoma of pancreas; 18 – 75 years of age; ECOG Stage 0-1

Open Label, Multicenter,

Randomized Trial

https://clinicaltrials.gov/ct2/show/NCT03504423

Phase I Study - CPI-613® (devimistat) in Combination with gemcitabine and nab-paclitaxel in Locally Advanced or Metastatic Pancreatic Cancer: Enrolled More Older Patients


N (%) gemcitabine + nab-paclitaxel*(Historical)


(N = 431) (N = 13)Age (years):

Median 62 66Range 27 - 86 56 - 80

Age Distribution:<65 years 254 (59%) 6 (46%)≥65 years 177 (41%) 7 (54%)

Sex:Male 245 (57%) 3 (23%)Female 186 (43%) 10 (77%)

ECOG Performance Status Score**:0 69 (16%) 5 (38%)1 328 (76%) 6 (46%)2 32 (7%) 0 (0%)Not Available 0 (0%) 2 (15%)

Disease StatusLocally Advanced 0 (0%)*** 1 (8%)Metastatic 431 (100%) 12 (92%)

**Converted Karnofsky Grade to ECOG grade as per: https://oncologypro.esmo.org/Oncology-in-Practice/Practice-Tools/Performance-Scales, measured in 429 patients; ***study included only patients with metastatic disease

*N Engl J Med 2013;369:1691-703; gemcitabine + nab-paclitaxel is recommended in patients with locally advanced pancreatic cancer based on extrapolations from trials in patients with metastatic disease (NCCN guidelines version 2.2019)

In devimistat + gemcitabine + nab-paclitaxel phase I study (locally advanced + metastatic), patients were older compared with historical cohort of gemcitabine + nab-paclitaxel* (only metastatic)

https://oncologypro.esmo.org/Oncology-in-Practice/Practice-Tools/Performance-Scales

CPI-613® (devimistat) in Combination with gemcitabine and nab-paclitaxel in Locally Advanced or Metastatic Pancreatic Cancer: Result to Date & Comparison with gemcitabine + nab-paclitaxel (Historical Data)*


Result to Date Comparison with gemcitabine + nab-paclitaxel (Historical Data)*

devimistat + gemcitabine + nab-paclitaxel N %

CR 2 25%

PR 3 38%

SD 3 38%

Not Evaluable 2

Not Evaluated 3

Total Evaluated for Efficacy 8

ORR (CR + PR) 5 63%Note: Numbers may not add up due to rounding

23%

63%

gemcitabine + nab-paclitaxel

devimistat + gemcitabine +nab-paclitaxel

Objective Response Rate (ORR)

*Historical Data: N Engl J Med 2013;369:1691-703; #Relative Increase

174%#

CPI-613® (devimistat) in Acute Myeloid Leukemia: Preclinical, Clinical Experience and Ongoing Trials

Significant Synergy is Observed with cytarabine or doxorubicin

OCI-AML3 (monocytic AML) and MFL2 (murine AML) cell lines were exposed to cytarabine (Ara) or doxorubicin (Dox) with and without devimistat at 25 or 50 mmol/L (clinically achievable doses) and assessed viability

Cells exposed to chemotherapy and devimistat were significantly more sensitive compared with cells treated with chemotherapy alone

Pharmacologic inhibition of the TCA with CPI-613® (devimistat) increases sensitivity to cytarabine (Ara) or doxorubicin (Dox) in AML cell lines

Pardee et. al. Clin Cancer Res. 2018 May 1;24(9):2060-2073.


CPI-613® (devimistat) in Combination with High Dose Cytarabine (HiDAC) and Mitoxantrone (Salvage Trial) in Relapsed or Refractory AML: Enrolled More Poor Risk Patients


Elderly Patient Population (≥ 60 Years)Historical Cohort* Combined Data**

High dose cytarabine +

mitoxantrone

devimistat+


mitoxantroneN 66# 21

Age:Median 68 68Range 60 - 84 60 - 76

Gender:Female 30 (45%) 7 (33%)Male 36 (55%) 14 (67%)

Risk CategoryGood 2 (5%) 2 (10%)Intermediate 29 (65%) 10 (48%)Poor 13 (30%) 9 (43%)

Median Duration of First CR (months): 7.2 10

Refractory disease (%) 23 (35%) 6 (29%)*Leuk Res. 2015 September ; 39(9): 945–949; **Phase I + Phase II data; #Risk category available for 44 patients

devimistat in combination with high

dose cytarabine and mitoxantrone

had higher percentage of patients

under poor risk category in

comparison with historical cohort of

high dose cytarabine and

mitoxantrone

Elderly Patient Population (≥ 60 Years)

Historical Cohort* Combined Data**


mitoxantrone

devimistat+


mitoxantrone(1,500 mg/m2 devimistat)

devimistat+


mitoxantrone(2,000 mg/m2 devimistat)

N 66 19 21

CR 27% 29% 48%

CR + CRi 33% 35% 52%Median OS (Months) 5.2 5.4 12.4

Pooled dataset** of both Phase I and Phase II trials demonstrate a dose response relationship in patients 60 years of age or older and strongly support the dose selection of 2,000 mg/m2 for the proposed phase III trial in this target population

CPI-613® (devimistat) in Combination with High Dose Cytarabine (HiDAC) and Mitoxantrone (Salvage Trial) in Relapsed or Refractory AML: Exhibited Dose Response Relationship & Higher Activity


*Leuk Res. 2015 September ; 39(9): 945–949; **Phase I + Phase II data

CPI-613® (devimistat) in Combination with High Dose Cytarabine (HiDAC) and Mitoxantrone (Salvage Trial) in Relapsed or Refractory AML: Survival by Dose & Age

EHA Learning Center. Pardee T. Jun 16, 2018; 215322 Abstract: PS998

Overall Survival was Superior in the 2,000 mg/m2 Dose Cohort Compared to 1,500 mg/m2, especially in older patients


CPI-613® (devimistat) in Combination with High Dose Cytarabine (HiDAC) and Mitoxantrone (Salvage Trial) in Relapsed or Refractory AML: Phase 3 Study Details


NCT03504410

Elderly Patients (≥ 60 Years) with

Relapsed / Refractory AML

Randomization(1:1) N= 500

Arm 1 (Test Arm) N= 250

Arm 2 (Control Arm) N= 250

Devimistat + High Dose Cytarabine + Mitoxantrone (CHAM)

High Dose Cytarabine + Mitoxantrone (HAM)

Patient Population: Female and male patients with AML that is relapsed from, or refractory to,prior standard therapies; ECOG Stage 0 - 2

Primary Endpoint: CR

Key Secondary Endpoints: OS CRh Safety PK PRO’s

Potential Timelines:

Q2 2020 (1st Interim )

Q4 2020 (2nd Interim)

H2 2021 (Final)

Open Label, Multicenter,

Randomized Trial

https://clinicaltrials.gov/ct2/show/NCT03504410

CPI-613® (devimistat) in Other Hematological Malignancies: Preclinical & Clinical Experience

Synergy is Observed with bendamustine


*Standard tissue culture conditions, 72-hour exposure

Acute Lymphoblastic Leukemia Cell Line (CCRF-CEM) Acute Lymphoblastic Leukemia Cell Line (MOLT-4)

devimistat in combination

with bendamustine

exhibited synergistic

effect across different cell-

lines*CI < 1 meaning synergy

Combinatorial Index (CI)* for devimistat + bendamustineAcute T-cell Leukemia Cell Line (Jurkat)

Clinical Experience of CPI-613® (devimistat) in T-cell Lymphoma, Myelodysplastic Syndrome (MDS) & Burkitt’s Lymphoma: A Summary

Case Report: Relapsed Burkitt’s Lymphoma

Before devimistat After 3 Cycles of devimistat

Relapsed or Refractory T-Cell Lymphoma

Phase I study of devimistat in combination with bendamustine exhibited significant signal for efficacy

Relapsed or Refractory Myelodysplastic Syndrome (MDS)

Phase II Study of devimistat (Single Agent)

19-year-old female with relapsed Burkitt’s Lymphoma

Before devimistat:

The patient was suffering from second relapse after first-line therapy of CALGB 10002 and second-line therapy of hyper-CVAD and myeloablative allogeneic stem cell transplant

After devimistatMonotherapy: Achieved and maintained

radiographic partial response status after the third cycle and maintainedfor 17 cycles of therapy over 51 weeks

Then the patient pursued surgical resection of residual tumor

Clinical follow-up on the patient indicated no evidence of disease more than 36 months

CRi Marrow CR SD PD

N* 1 1 8 2

% 8% 8% 67% 17%*3 patients are still alive with one patient SD after 6 years


bendamustine (BENTLY Trial*)

devimistat + bendamustine

ORR 50% 75%Median OS 6.2 Months 9.2 MonthsMedian PFS 3.6 Months 6.4 Months

*Note: This is an illustrative comparison of clinical experience of devimistat + bendamustine with data from one historical trials of bendamustine alone (BENTLY Trial)

CPI-613® (devimistat): Clinical Summary


Trial ID Phase Indication Treatment Detail # Evaluated / Planned

Efficacy / Potential Timelines

Panc

reat

ic C

ance

r

Completed CCCWFU 57112 IFirst-line Metastatic Pancreatic Cancer

devimistat + modified FOLFIRINOX

18

Median OS: 19.9 months Median PFS: 9.9 months ORR: 61.0% Median DoR*: 9.2 months

Ongoing AVENGER 500®

(Randomized) III 500§ Q2 2020 (Futility) Q4 2020 (Interim) H2 2021 (Final)

Ongoing GA CPI-613 I Locally Advanced or Metastatic Pancreatic Cancer


8 ORR: 63.0%

Ongoing CASE2218 II Locally Advanced Pancreatic Cancer

devimistat + modified FOLFIRINOX 33§ -

Acut

e M

yelo

id

Leuk

emia

(A

ML)

Completed CCCWFU 22112; CCCWFU 22215 I & II Relapsed or Refractory

Acute Myeloid Leukemia (AML) in Elderly Patients (≥ 60 years)

devimistat + cytarabine + mitoxantrone

21** CR: 48% CR + CRi: 52% Median OS: 12.4 months

Ongoing ARMADA 2000 (Randomized) III 500§

Q2 2020 (1st Interim ) Q4 2020 (2nd Interim) H2 2021 (Final)

Oth

er H

emat

olog

ical

M

alig

nanc

ies

Ongoing CCCWFU 28314 I Relapsed or Refractory T-Cell Lymphoma

devimistat + bendamustine

8 Median OS: 9.2 months Median PFS: 6.4 months ORR: 75%

Upcoming(Q3 2019) - II (Pivotal) ~100§ -

Ongoing 18-443 IIRelapsed or Refractory

Burkitt Lymphoma/Leukemia or High-Grade B-Cell Lymphoma

devimistat 34§ -

Not yet recruiting WFBCCC 99119 I/II High Risk Myelodysplastic Syndrome devimistat +

Hydroxychloroquine 18 – 35 -

*Duration of Response; **Pooled dataset; §Planned

Commercial Plan of CPI-613® (devimistat)

Commercial Plan

Out-Licensing

Out-Licensing

LATAM

Co-Development/Marketing or Out-Licensing

Out-Licensing

Rafael is looking for collaboration (joint venture / out-licensing / co-development) to realize full potential of devimistat

Objective of the Collaboration: Reach more patients across

the world in different commercial regions

The collaborator may gain an exclusive option to develop and commercialize devimistator gain royalties on product sales for specific region

Explore opportunity for scientifically meaningful, rational and innovative combination with other investigational small molecule, biologics or companion diagnostics

Fully Owned or Co-Development/Marketing


Key Takeaway:Rafael is a Lucrative De-risked Investment Opportunity

Novel and safe approach to

cancer treatment

Multiple indications

targeted with high unmet need

Large amount of safety data on

devimistat

Two phase III pivotal trials

Management with extensive experience in discovery, development and commercialization of oncology products

Strong IP: Protected until 2029 and beyond across U.S., Canada,

EU, Australia and major markets of Asia

Robust pipeline


“Extending and enhancing the lives of patients with cancer”

“To Save A Life Is To Save A Universe”

Thank You


An Affiliate of Rafael Holdings (NYSE:RFL) Strictly …...Harnessing the Power of Cancer Metabolism...

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Transcript of An Affiliate of Rafael Holdings (NYSE:RFL) Strictly …...Harnessing the Power of Cancer Metabolism...