Non-Confidential Overview

November 2017

CAELUMBIOSCIENCES

Forward Looking Statements

2

Statements in this presentation that are not descriptions of historical facts are forward‐looking statements

within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995.

We have attempted to identify forward‐looking statements by terminology including “anticipates,”

“believes,” “can,” “continue,” “could,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,”

“should,” or “will” or the negative of these terms or other comparable terminology. Forward‐looking

statements are based on management’s current expectations and are subject to risks and uncertainties that

could negatively affect our business, operating results, financial condition and stock price. Factors that could

cause actual results to differ materially from those currently anticipated are risks relating to: results of

research and development activities; uncertainties relating to preclinical and clinical testing; our growth

strategy; our ability to obtain, perform under and maintain financing and strategic agreements and

relationships; our dependence on third party suppliers; our ability to obtain, perform under and maintain

financing and strategic agreements and relationships; our ability to attract, integrate, and retain key

personnel; the early stage of products under development; our need for substantial funds; government

regulation; patent and intellectual property matters; and competition. We expressly disclaim any obligation

or undertaking to update or revise any statements contained herein to reflect any change in our

expectations or any changes in events, conditions or circumstances after the date of this presentation.

About Us

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Caelum Biosciences is a clinical stage biotechnology companydeveloping treatments for rare and life-threateningconditions. Caelum’s lead asset, CAEL-101, is a novelpioneering antibody in Phase 1b clinical trials that is beingdeveloped for patients with Amyloid Light chain (AL)Amyloidosis.

Michael Spector, CEO

Potential Best-in-Class Antibody developed to clear amyloid deposits

Sustained clinically significant organ activity even after a single dose

Well-tolerated with no dose limiting toxicity

Rationally designed antibody clears both human AL λ and κ in preclinical studies

Ph1b ongoing - last patient dosed; expect results 2017

Preparing for Phase 3

Broad protection through IP and Orphan Drug and Biologics exclusivity

CAEL-101 Opportunity Overview in AL Amyloidosis

4

Amyloid Fibrils Formation in Tissue

5

Misfolded protein

AL Amyloidosis

patients

Misfolded proteins

collect together

Collection of

misfolded protein

creates Amyloid fibrils

Plasma Cells

Plasma

Cells

Produce

Proteins

Normal Protein

Bone

marrow

produce

plasma

cell

Bone

Amyloid Fibrils

deposit

in tissues

Normal Plasma Protein Cell Production

Mechanism of Amyloid Formation in Tissues

AL Amyloidosis Impact on Organs & Tissue

60%-80% of patients - leads to end-stage

kidney disease

65%-75% of patients - leads to heart failure

and high mortality

20%-45% of patients - peripheral neuropathy

leading to pain, numbness, and weakness

6

5%-35% - Other organs

AL Amyloidosis Opportunity: High Unmet Need and Large

Market Opportunity

7 Source: Kumar et al, Mayo Clinic Proc, 2011 86(1):12-8.

• 1 Year Mortality: 47%

• 5 Year Mortality: 72%

• Median Mortality: 1.5 Years

• Wall Street Analysts estimate a prevalent patient population of 30,000 – 40,000 patients split evenly between the U.S. and EU.

• It is estimated that there are approximately 3,000 – 4,000 new cases of AL amyloidosis annually in the United States, though actual incidence is likely higher as a result of under-diagnosis; a recent epidemiology study is illustrative (see next slide)

• An approved, successful therapy could represent a >$1.5B opportunity

Case Study: Underdiagnosis of AL Amyloidosis in England

8 1Pinney et. al.; Systemic amyloidosis in England: an epidemiological study; BJH 2013

National

Amyloidosis

Centre

Incidence of AL Amyloidosis by region

0 1.000.50

Correct diagnosis following referral appears to be a function of proximity to specialist center: Suggests that AL

Amyloidosis may be widely underdiagnosed

RegionPopulation (in

mm)

Incidence based on

newly diagnosed

caes at NAC1

Incidence

Reference to

London

England 51.4 0.52 71%

1 – East of England 5.7 0.56 77%

2 – East Midlands 4.4 0.47 64%

3 – London 7.7 0.73 100%

4 – Northeast 2.6 0.07 10%

5 – Northwest 6.8 0.35 48%

6 – South Central 4 0.64 88%

7 – Southeast Coast 4.3 0.55 75%

8 – Southwest 5.2 0.47 64%

9 – West Midlands 5.4 0.64 88%

10 – Yorkshire and the

Humber5.2 0.42 58%

Day-1 Day-14

Amyloid fibril suspensions sourced from human postmortem

samples were injected into mice to form amyloidomas. Mice

received either no treatment or 11-1F4 (CAEL-101).

Untreated Mice

CAEL-101

Treated Mice

Day-14Day-1

CAEL-101 expedites the clearance of AL amyloidoma in a

human PDx mouse model

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CAEL-101: Rationally designed antibody

that clears both ALλ and κ deposits

Source: Hrncic et al, Am J Path 2000

CAEL-101 removes amyloidomas in mice bearing human

ALΚ & λ fibrils

10

1415

14

21

2425

28

4 4

89

17

24

28

0

5

10

15

20

25

30

κ-1 κ-2 λ-1 λ-2 λ-3 λ-4 λ-5

Days

Un

til C

leara

nce

Single Dose1 3

Untreated 11-1F4

Notes: Days until clearance of untreated vs 11-1F4 treated human amyloidoma deposits injected subcutaneously into mice

1. 100μg at day 0 2. 100 μg at days 0,2,4,6 . 11-1F4 are mAb designation

Source: Hrncic et al, Am J Path 2000

NT NT NT

24

2625

28

NT NT NT

9 9

67

0

5

10

15

20

25

30

κ-1 κ-2 λ-1 λ-2 λ-3 λ-4 λ-5

Days

Un

til C

leara

nce

Multiple Doses2 3

Untreated 11-1F4

CAEL-101-Mediated Clearance of Human ALκ and ALλ Amyloidomas in Mice

• GMP-grade amyloid fibril-reactive human IgG1 Fc chimeric mAb CAEL-101 was produced by NCI’s Biological Resource Branch for a Phase 1a/b trial

• Open-label, dose-escalation Phase 1a/b study of CAEL-101 (NCT02245867)

• Patients with relapsed or refractory AL Amyloidosis

• Primary Objective:

• Establish the maximum tolerated dose (up to 500 mg/m2) of CAEL-101

• Secondary Objectives:

• Demonstrate reduction in amyloid burden, as evidenced by decrease in affected organomegaly, and/or improved organ function

• Determine the pharmacokinetics of CAEL-101 when given as a single IV infusion (phase 1a) or as a series of weekly IV infusions (phase 1b)

• To determine the difference between 250mg/m2 and 500mg/m2

• https://clinicaltrials.gov/ct2/show/NCT02245867

Phase 1a/b Study of CAEL-101 (11-1F14) in Patients with AL

Amyloidosis presented at ASH 2016

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Palladini et al reported a strong

correlation between reduction in amyloid

free light chains and survival in patients

treated with front line chemotherapy.

Cardiac morbidity is the major

determinant of survival in these patients

generally, and changes in cardiac function

after therapy can be reliably assessed

using the cardiac biomarker N-terminal

natriuretic peptide type B (NT-proBNP).

Changes in amyloid free light chain and

NT-proBNP predicted survival as early as 3

months after treatment initiation.

The international AL amyloidosis expert community is in agreement that the N-terminal fragment of the probrain natriuretic

peptide (NT-proBNP) is analytically validated and clinically qualified as a biomarker for use as a surrogate end point for survival

in patients with AL amyloidosis. (G Merlini et al, Leukemia, 2016)

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NT-proBNP Associated with Survival in Amyloidosis Patients:

Potential for Surrogate Endpoint

CAEL-101 binds to

Liver and Bone

Amyloid Fibrils

Wall, JS et al. Blood. 2010 Sep 30;116(13):2241-4.

Distribution of CAEL-101 Antibody Binding

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Radiolabeled

CAEL-101

Wall’s work shows that CAEL-101 successfully binds AL amyloid.

CAEL-101 Phase 1a/1b Organ Response Rates to Date

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Renal Response Phase 1a

& 1b (n=8)

Cardiac Response Phase

1a & 1b (n=8)

Overall Responders

Best Organ Response

Phase 1a Phase 1b

12 Weeks

4 Weekly Doses

8 Weeks

Single Dose

63%Responders

70%

Responders

5/8 Patients

7/10 Patients

Responder Stable Progressor

>30% and >300

pg/ml decrease

in NT-proBNP

>30% and >300

pg/ml decrease in

NT-proBNP

63% 25% 12.5%

5/8

2/4 from Phase 1a

3/4 from Phase 1b

Patients

2/8Patients

1/8

Responder Stable Progressor

75% 25% 0%

6/8 Patients

2/4 from Phase 1a

4/4 from Phase 1b

2/8 Patients

>30% decrease

in proteinuria or a

decrease to <0.5

g/24 hours

>25% worsening

in eGFR

PATIENT 3 PROFILE

❖ Refractory λ AL Amyloidosis

❖ Baseline NT-proBNP approx. 13,000 ng/L

❖ Previous treatments: 1

❖ Best Hematologic response to chemotherapy: VGPR

❖ No Organ response to chemotherapy

❖ Persistently elevated NT-proBNP

❖ NYHA Class III

Organ response to CAEL-101

❖ NYHA Class I

❖ NT-proBNP Reduction to below 4,000 ng/L

Cardiac response (NT-proBNP) in a patient during Phase 1a/b

clinical trial of CAEL-101 antibody

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Marked and Sustained Cardiac Response After Initial Dose of

CAEL-101

0

2,000

4,000

6,000

8,000

10,000

12,000

14,000

Mar-2015 Jun-2015 Sep-2015 Dec-2015 Mar-2016

NT

pro

-BN

P (

ng

/L)

Started

Phase 1a

CAEL-101 Started

Phase 1b

CAEL-101Wk

4Wk

8

Wk

5

Wk

8

Wk

12

PATIENT 7 PROFILE

❖ Refractory λ AL Amyloidosis

❖ Baseline 24-hr urine protein in mg/24hr: approx. 10,000

❖ Previous treatments: 6

❖ No Organ response to chemotherapy

❖ Persistence of significant proteinuria

Organ response to CAEL-101

❖ 24 hour urine protein in mg/24 hr: approx. 3,000

24 hour urine protein in a patient before and during

Phase 1a/b clinical trial of CAEL-101 antibody

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Marked and Progressive Renal Response After Initial Dose of

CAEL-101

0

2,000

4,000

6,000

8,000

10,000

12,000

Apr-2015 Jul-2015 Oct-2015 Jan-2016 Apr-2016

24

hr

uri

ne p

rote

in (

mg

/24

hr)

Started

Phase 1a

CAEL-101Wk

5

Wk

8

Wk

12

Wk

8

Wk

4

Started

Phase 1b

CAEL-101

• Treatment with CAEL-101 is well tolerated and safe

• No drug-related grade 4 or 5 AEs or dose-limiting toxicity up to an MTD of 500mg/m2

• CAEL-101 is clinically efficacious and rapid-acting

• Early and sustained organ response even as a single infusion, or as a weekly infusion for 4 weeks: 70% response after 4 weekly doses

• Cardiac, Renal, GI, Skin and Soft tissue responses observed

• CAEL-101 represents a novel and promising adjunct to the treatment of AL Amyloidosis

• Safely promotes amyloid resolution in 67% of the patients to date

• Leads to improvement in organ function

Summary Clinical Data CAEL-101

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• Phase1b study in 2017: Awaiting final data report and PK analysis

• Finalize Phase 3 plan and prepare for trial launch

• Strategic Manufacturing Agreement with Patheon Biologics for clinical trial and commercial manufacturing

Next Steps

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19

Broad exclusivity through IP and regulatory protection

Phase 3 to commence in 2018

Organ activity independent of light chain sub-type

Marked and sustained responses even after a single dose

No dose-limiting toxicity

Promising Phase 1a/1b data; results anticipated in 2017

Pioneering, rationally designed antibody

Strategic cGMP manufacturing agreement in place

Experienced Leadership Team

CAEL-101 Summary

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Key Leadership

Lindsay Rosenwald, M.D. - Executive Chairman

Dr. Rosenwald has almost 25 years of experience as a biotechnology entrepreneur, including the

founding and recapitalization of numerous public and private biotechnology and life sciences

companies. In addition to serving as Caelum’s Executive Chairman, Dr. Rosenwald is Chairman,

President and CEO of Fortress Biotech, Caelum’s parent company. Dr. Rosenwald is also a Co-Portfolio

Manager and Partner at Opus Point Partners Management, LLC, a life-science focused asset

management firm. Before moving into his current positions, Dr. Rosenwald served as Chairman of

Paramount BioCapital, Inc. Dr. Rosenwald received an M.D. from Temple University School of Medicine

and a B.S. in finance from Pennsylvania State University.

Michael Spector - President and Chief Executive Officer

Mr. Spector was named President and Chief Executive Officer of Caelum in January 2017. Previously, Mr.

Spector served as Senior Vice President, Global Commercial Operations at Iroko Pharmaceuticals. Earlier in

his career, he spent 15 years at GlaxoSmithKline in multiple senior management positions, including Vice

President and General Manager of GlaxoSmithKline South Africa, where he led the overall business

strategy and was elected to run the South African Pharmaceutical Manufacturers Association. Mr. Spector

holds an M.B.A. from Rider University in Lawrenceville, N.J. and a B.S. in biology from the University of

Pittsburgh. He also serves on the Board of Directors of Jacaranda Health, a nonprofit organization that

seeks to transform maternal and neonatal healthcare in East Africa.

Elisabeth Leiderman, M.D., M.B.A. – Vice President, Corporate Development

Dr. Leiderman has 20 years of experience working in the healthcare industry. Since January 2017, Dr.

Leiderman has served as Vice President and Head of Corporate Development at Caelum which is a

subsidiary of Fortress Biotech. Prior to joining industry, Dr. Leiderman worked as a Healthcare Investment

Banker, where she most recently was an Executive Director at Nomura Securities. She spent close to 10

years on Wall Street and held prior roles at UBS and Credit Suisse. She has covered life science companies

and advised clients on M&A, Equity and Debt transactions. Dr. Leiderman earned her B.A. at The

University of Pennsylvania, her M.D. from Sackler Medical School at Tel-Aviv University and her MBA from

The Wharton School. Dr. Leiderman is an employee of Fortress Biotech and provides services to Caelum

Biosciences pursuant to a Management Services Agreement between Fortress Biotech and Caelum

Biosciences.

Suzanne Lentzsch, M.D., Ph.D. – Scientific Advisory Board Chair

Professor of Medicine at College of Physicians and Surgeons, Columbia University

Director, Multiple Myeloma and Amyloidosis Service Columbia Univ Medical Center

Dr. Lentzsch is Professor of Medicine and the Director of the Multiple Myeloma and Amyloidosis Service at

New York Presbyterian Hospital/ Columbia University Medical Center, New York. After receiving her

degrees from the Humboldt University/ Charité Berlin, Germany, she completed her residency and

fellowship at Humboldt University and a research fellowship, studying the mechanism of action of

thalidomide and its derivatives in multiple myeloma, in the Jerome Lipper Multiple Myeloma Center under

the mentorship of Dr Kenneth Anderson at the Dana-Farber Cancer Institute, Boston. Dr. Lentzsch was

recruited in August 2004 to the University of Pittsburgh and the University of Pittsburgh Cancer Institute.

Her translational research focuses on the identification of novel targets for the treatment of Multiple

Myeloma, Multiple Myeloma bone disease and amyloidosis. Her innovative research in amyloidosis

resulted in a series of translational clinical trials, including the establishment of new treatments for

relapsed AL amyloidosis in a multicenter trial investigating bendamustine.