Corporate Presentation
March 2023
Fortress Biotech
Forward Looking Statements
This presentation may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section
21E of the Securities Exchange Act of 1934, as amended. For such forward-looking statements, we claim the protection of the safe harbor for forward-
looking statements contained in the Private Securities Litigation Reform Act of 1995. As used below and throughout this presentation, the words “we”,
“us” and “our” may refer to Fortress individually or together with one or more partner companies, as dictated by context. Such statements include, but
are not limited to, any statements relating to our growth strategy, products and product development programs and any other statements that are not
descriptions of fact. 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 include: risks related to our growth strategy; risks relating to the results of research and development activities; our
ability to obtain, perform under and maintain financing and strategic agreements and relationships; uncertainties relating to preclinical and clinical
testing; our dependence on third party suppliers; our ability to attract, integrate, and retain key personnel; the early stage of products under
development; our need for and continued access to additional funds; government regulation; patent and intellectual property matters; competition; as
well as other risks described in our Securities and Exchange Commission filings. We expressly disclaim any obligation or undertaking to release
publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in
events, conditions or circumstances on which any such statement is based, except as may be required by law. The information contained herein is
intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this
presentation should be read as applying mutatis mutandis to every other instance of such information appearing herein. This presentation may
contain depictions of Fortress’ percentage ownership positions in several of its affiliated companies; while we endeavor to update such figures
regularly, these percentages are subject to periodic change for a variety of reasons, and updates may not occur more frequently than every calendar
month or quarter. Accordingly, you should understand that the percentage figures presented herein may only portray Fortress’ ownership positions as
of the most recent update, and not necessarily as of the date on which you are reviewing these materials.
2
Commercial Late Clinical Early Clinical Preclinical
Qbrexza
®
CUTX-101 MB-106 MB-109
Targadox
®
Cosibelimab Dotinurad In vivo CAR T Technology
Ximino
®
Olafertinib AJ201 AAV.sFH Gene Therapy
Exelderm
®
CAEL-101
**
BAER-101 AAV-ATP7A Gene Therapy
AMZEEQ
®
MB-107 MB-101 ConVax
ZILXI
®
MB-207 MB-108 CEVA-102
8 Total Marketed
Dermatology Products
*
DFD-29 MB-102 CEVA-D
Triplex MB-103 CK-103
CEVA-101 MB-104 CK-302
IV Tramadol MB-105 CK-303
MB-110
Fortress product portfolio by stage
Portfolio includes product candidates in development at Fortress, at its majority-owned and majority-controlled partners, and at partner companies that Fortress may otherwise have an economic interest in
*
8 total products includes Qbrexza, Targadox, Ximino, Exelderm, Amzeeq, and Zilxi
**AstraZeneca’s Alexion acquired Caelum Biosciences on 10/5/2021 for up to $500 million, including $150 million upfront and up to $350 million in future contingent milestone payments. FBIO received ~$56.9 million of such upfront
amount and is eligible to receive ~42% of the proceeds from all future milestone payments.
Therapeutic Areas/Modalities
Dermatology Rare Disease Rheumatology
Oncology / Hematology Other
3
Building shareholder value through long-term growth and scale
4
Fortress’ business model has the potential for
“snowballing” growth as we acquire new assets and as
our subsidiary/partner companies grow in value
Monetizations
Milestones
Equity
Holdings
Product
Revenue
Royalties
Equity
Dividends
2021 was a record year for Fortress with our extensive portfolio of assets
progressing/scaling and business development engine generating new opportunities
5
Portfolio includes product candidates in development at Fortress, at its majority-owned and majority-controlled partners, and at partner companies that Fortress may otherwise have an economic interest in
* Includes two trials at partner company Caelum Biosciences, which was sold to AstraZeneca in Oct 2021 and with respect to which Fortress remains eligible to receive up to an additional ~$148 million
Pipeline Development
21 clinical-stage programs
31
6
2
$150M
ongoing clinical trials
ongoing pivotal /
registrational trials*
NDA submissions
anticipated
over next twelve months
spent on R&D across
pipeline candidates by
Fortress and our partner
companies in 2021
Commercialization & Monetization
8 marketed programs
2 recent program monetizations
$63.1M
$56.9M
$8.0M
Journey net sales (2021)
42% growth over 2020
in monetizations to
Fortress from sale of
Caelum to AstraZeneca
in upfront milestones from
Sentynl at deal execution
(up to $12M remaining through
approval, plus sales milestones and
royalties)
Business Development Engine
9 recent acquisitions
(2021-2023 YTD)
Dotinurad
ZILXI
®
, AMZEEQ
®
,
QBREXZA
®
, DFD-29
MB-110, in vivo CAR-T
4
marketed products
4
clinical-stage assets
1
pre-clinical technology
platform
1
BLA submission accepted
by FDA on March 2, 2023
AJ201
Fortress has an extensive portfolio of commercial and development assets
and a business development engine for generating new opportunities
6
Portfolio and pipeline includes product candidates in development at Fortress, at its majority-owned and majority-controlled partners, and at partner companies that Fortress may otherwise have an economic interest in
* Includes two trials at partner company Caelum Biosciences; AstraZeneca’s Alexion acquired Caelum Biosciences on 10/5/2021 for up to $500 million, including $150 million upfront and up to $350 million in future contingent milestone
payments. FBIO received ~$56.9 million of such upfront amount and is eligible to receive ~42% of the proceeds from all future milestone payments.
21 clinical-stage programs
31
ongoing clinical trials
6*
ongoing pivotal /
registrational trials
8 marketed programs
2
NDA
submissions
anticipated over
next twelve
months
Preclinical and Early-to-Mid-Stage Clinical
Progression
Late-Stage and Regulatory Execution Commercial Scaling
$57.7M
Journey net revenue
(2022 Q1-Q3)
77%
CAGR on net revenue
(2016-2021)
Over $150M spent on R&D activities across pipeline candidates by Fortress and our
partner companies in 2021
1
BLA submission
accepted by FDA
on March 2, 2023
7
Potential revenue and cashflow events
Approval, data, and trial timings are internal estimates based on current knowledge and potential timelines are subject to change. Potential peak sales are based on internal forecasts and/or market comps and assume approval in all
denoted indications and are subject to change; * Asset is currently fully controlled by AstraZeneca through the acquisition of Caelum, all estimates and dates related to CAEL-101 are based on Fortress estimates and not guidance
provided by Caelum or AstraZeneca. Fortress remains eligible to receive up to an additional ~$155 million in escrow release and milestone payments from the transaction;
^ Cyprium is currently in a dispute with its contract manufacturing organization (the “CMO”), regarding the CMO’s attempt to terminate a Master Services Agreement (together with related work orders, the “MSA”) between Cyprium and
the CMO. Cyprium believes the CMO’s grounds for purporting to terminate the MSA are without merit and is currently availing itself of all appropriate legal remedies in efforts to ensure that the CMO abides by its obligations under the
MSA and/or to pursue monetary damages claims against the CMO. To that end, Cyprium obtained a temporary restraining order in August 2022 and a preliminary injunction in September 2022 from a court in New York State; the
injunction enjoined the CMO from terminating the MSA and prohibited the CMO from further attempts to terminate the MSA during the pendency of dispute resolution procedures.
= Anticipated product revenue/royalties
= Potential PRV/milestone/monetization proceeds
= Potential regulatory approval
< $500M $500M - $1B
> $1B
Partner
Company
Asset(s) 2020 2021 2022 2023 2024 2025 2026+
Potential Peak Sales
(Global)
Journey
(DERM)
Commercial Portfolio
Qbrexza, Ximino, Targadox, etc.
$44.5M
Net revenue
$63.1M
Net revenue
$57.7M
Q1
-
Q3 Net revenue
DFD
-29 (oral small molecule)
Two Phase 3s for rosacea
Caelum*
CAEL
-101 (fibril-reactive mAb)
Pivotal trial for AL amyloidosis
$56.9M
Monetization
Checkpoint
(CKPT)
Cosibelimab (anti
-PD-L1 mAb)
BLA accepted for metastatic and locally
advanced
cSCC
Olafertinib
(3
rd
gen EGFRi)
Phase 3 trial in Asia in NSCLC (partner)
(Asia)
Cyprium^
CUTX
-101 (copper histidinate)
NDA submission for Menkes Disease
$8M
Milestone
Mustang
(MBIO)
MB
-107 (gene therapy)
Pivotal Phase 2 trial for newly diagnosed
XSCID planned 2023
MB
-207 (gene therapy)
Pivotal Phase 2 trial for previously
transplanted XSCID planned 2023
MB
-109 (CAR-T + oncolytic virus)
IND filing for GBM planned 2023
MB
-106 (CD20 CAR-T)
Phase 1/2 trial for NHL & CLL
Urica
Dotinurad (URAT1 inhibitor)
Phase 1 trial ongoing
Potential near-term value-creating events for Fortress shareholders
8
CUTX-101, DFD-29, MB-106, MB-107, MB-109, MB-207, Cosibelimab, Olafertinib, BAER-101, IV Tramadol and Dotinurad are product candidates in development at Fortress subsidiary/partner companies
* CAEL-101 is currently fully controlled by AstraZeneca through the acquisition of Caelum, all estimates and dates related to CAEL-101 are based on Fortress estimates and not guidance provided by Caelum or AstraZeneca
Approval, data, and trial timings are internal estimates based on current knowledge and potential timelines are subject to change
^ Refer to Footnote regarding CUTX-101 on Slide 14
+
Urica Therapeutics, Inc. renamed from UR-1 Therapeutics, Inc.
Category Company Asset
Anticipated Milestone Anticipated Timing
Monetization
Events
Cyprium^
CUTX-101
o Potential milestone payments and PRV sale 2023 / 2024
o Eligible to receive ~70% of up to $267M in remaining regulatory and sales milestones 2023+
Caelum*
CAEL-101
o Eligible to receive ~42% of additional potential payments to Caelum shareholders totaling up to
$350M from regulatory and commercial milestones
2023+
Regulatory
Decisions
Cyprium^
CUTX-101
o FDA Decision for Menkes Disease 2023 / 2024
Checkpoint
Cosibelimab
o FDA Decision for metastatic and locally advanced cSCC January 2024
Caelum*
CAEL-101
o FDA Decision for AL Amyloidosis 2023+
Clinical
Data
Urica
+
Dotinurad
o Phase 1 data readout 2023
Mustang
MB-106
o Potential Phase 1 data readout from Mustang-IND clinical trial 2023
Journey
DFD-29
o Anticipate readouts of two Phase 3 clinicals trials for rosacea 1H 2023
Caelum*
CAEL-101
o Potential readout of the CAELUM CARES Phase 3 2023+
Checkpoint
Olafertinib
o Potential Phase 3 data readout in NSCLC in Asia 2024+
Avenue
AJ201
o Potential readout of the Phase 1b/2a trial in SBMA patients 2024+
Trial Initiation
Mustang
MB-107
o Initiation of pivotal trial for XSCID in newly diagnosed patients 2023
MB-207
o Initiation of pivotal trial for XSCID in previously transplanted patients 2023
MB-109
o Initiation of novel combination (CAR-T + oncolytic virus) trial for GBM 2023
MB-106
o Initiation of Phase 2 pivotal study in NHL/CLL 2023
Avenue
BAER-101
o Initiation of Phase 1b photosensitivity (epilepsy) and/or CO2 inhalation studies (panic disorder) 2023
IV Tramadol
o Initiation of pivotal safety study (two pivotal efficacy trials already positive) 2023
Urica
+
Dotinurad
o Initiation of pivotal trial 2024
Pipeline Detail
9
10
Late-Stage Portfolio – Multiple near-term value inflection points
Candidate Indication(s) Phase 1 Phase 2
Pivotal /
Phase 3
Status / Upcoming Anticipated Milestones
FBIO Ownership % /
Royalty
†
Potential Peak
Sales (Global)
^
CUTX-101^^
Copper Histidinate
Menkes Disease
Rolling NDA submission in process and is expected
to be completed 2023^^
71% of Cyprium
4.5% Royalty
2.5% Annual Equity Dividend
Cosibelimab
Anti-PD-L1 mAb
Recurrent
or metastatic cancers
BLA accepted by the FDA for metastatic and locally
advanced cSCC, PDUFA in January 2024
19% of CKPT
4.5% Royalty
2.5% Annual Equity Dividend
Olafertinib
Mut.-EGFR Inh.
EGFR
+
NSCLC
Phase 3 study ongoing by partner
CAEL-101
mAb 11-1F4
Light chain (AL)
amyloidosis
Acquired by AstraZeneca in Oct 2021
Two ongoing global Phase 3 studies for AL
amyloidosis
42% of future proceeds to
Caelum from AstraZeneca**
$56.9M received**
~$150M in potential
future proceeds
MB-107
Gene Therapy
XSCID
(newly diagnosed)
Anticipate dosing first patient in MB-107
registrational trial in 2023
19% of MBIO
4.5% Royalty
2.5% Annual Equity Dividend
MB-207
Gene Therapy
XSCID
(previously transplanted)
Anticipate dosing first patient in MB-207
registrational trial in 2023
DFD-29
Oral Small Molecule
Rosacea
Topline data from Phase 3 program in rosacea
expected 1H 2023
58% of DERM
Triplex
Vaccine
Cytomegalovirus (CMV)
Initiated HIV/CMV co-infection Phase 2 trial;
received NIAID/NIH grant of potentially more than
$20M to fund Phase 2 study in liver transplant
82% of Helocyte
4.5% Royalty
2.5% Annual Equity Dividend
CEVA-101
Cell Therapy
Traumatic brain injury
(pediatric and adult)
Phase 2 study in Peds completed 1H 2021
Phase 2 data in Adults expected 2023
78% of Cellvation
4.5% Royalty
2.5% Annual Equity Dividend
IV Tramadol
Post-
operative acute pain
management
Continue dialogue with FDA and submit study
protocol to gain alignment on single safety study
11% of Avenue^*
4.5% Royalty
2.5% Annual Equity Dividend
Portfolio includes product candidates in development at Fortress, at its majority-owned and majority-controlled partners, and partner companies that Fortress may otherwise have an economic interest in.
**AstraZeneca’s Alexion acquired Caelum Biosciences on 10/5/2021 for up to $500 million, including $150 million upfront and up to $350 million in future contingent milestone payments. FBIO received ~$56.9 million of such
upfront amount (net of transaction expenses and escrow) and is eligible to receive ~42% of the proceeds from all future milestone payments.
^
Potential peak sales are based on internal forecasts and/or market comps and assume approval in all denoted indications and are subject to change
†
Ownership estimated as of Sep 30, 2022 ; FBIO ownership percentage estimate of Avenue as of January 2023’
^^ Refer to Footnote regarding CUTX-101 on Slide 14
^* FBIO ownership percentage estimate of Avenue as of Sep 30, 2022, does not account for dilution from Avenue’s public offering on Oct 11, 2022
< $500M $500M - $1B
> $1B
11
Early-to-Mid Stage Portfolio – Expansive pipeline for high unmet need areas
Candidate Indication(s) Preclinical Phase 1 Phase 2 Phase 3
Status / Upcoming Anticipated Milestones
FBIO Ownership % /
Royalty
†
Potential Peak
Sales (Global)
^
MB-106
CD20 CAR-T
B-Cell Non-Hodgkin
Lymphoma and CLL
Initial safety and efficacy data from Mustang-IND
trial anticipated in 2023
19% of MBIO
4.5% Royalty
2.5% Annual Equity Dividend
Dotinurad
URAT1 inhibitor
Gout and Chronic Kidney
Disease
Phase 1 data expected 2023
65% of Urica
4.5% Royalty
2.5% Annual Equity Dividend
AJ201
Nrf2 activator
Spinal and Bulbar
Muscular Atrophy (SBMA)
Phase 1b/2a in SBMA patients ongoing with
potential top-line data in 2024
11% of Avenue^*
4.5% Royalty
2.5% Annual Equity Dividend
MB-109
IL13Rα2 CAR-T + OV
Recurrent GBM and
anaplastic astrocytoma
File IND for Phase 1 combination trial for MB-109
(MB-101 + MB-108) in 2023
19% of MBIO
4.5% Royalty
2.5% Annual Equity Dividend
MB-101
IL13Rα2 CAR-T
Recurrent glioblastoma
(GBM)
MB-108
Oncolytic Virus (OV)
Recurrent GBM
MB-102
CD123 CAR-T
Blastic plasmacytoid
dendritic cell neoplasm
Mustang IND trial enrolling
MB-103
HER2 CAR-T
GBM and Metastatic
Breast Cancer to Brain
Data disclosure from COH Phase 1 trials expected
MB-104
CS1 CAR-T
Multiple Myeloma (MM)
Data disclosure from COH Phase 1 trial expected
MB-105
PSCA CAR-T
Prostate & Pancreatic
Cancers
Next data disclosure from COH Phase 1 prostate
cancer trial expected 1H 2023
MB-110
Gene Therapy
RAG1-SCID
Ongoing Phase 1/2 multi-center trial in Europe
BAER-101
α2/3–subtype-GABA A
PAM
CNS Disorders
Phase 1b photosensitivity and/or CO2 inhalation
studies planned for 2023
11% of Avenue^*
4.5% Royalty
2.5% Annual Equity Dividend
Portfolio includes product candidates in development at Fortress, at its majority-owned and majority-controlled partners, and partner companies that Fortress may otherwise have an economic interest in.
^ Potential peak sales are based on internal forecasts and/or market comps and assume approval in all denoted indications and are subject to change
†
Ownership estimated as of Sep 30, 2022 ; FBIO ownership percentage estimate of Avenue as of January 2023’
^* FBIO ownership percentage estimate of Avenue as of Sep 30, 2022, does not account for dilution from Avenue’s public offering on Oct 11, 2022
< $500M $500M - $1B
> $1B
12
Preclinical Portfolio – Long-term value potential for key therapeutic areas
Candidate Indication(s) Preclinical Phase 1 Phase 2 Phase 3
Status / Upcoming Anticipated Milestones
FBIO Ownership % /
Royalty
†
Potential Peak
Sales (Global)
^
In vivo CAR-T
Off-the-shelf CAR-T
Platform
Data publication in 2023
19% of MBIO
4.5% Royalty
2.5% Annual Equity Dividend
AAV.sFH
AAV Gene Therapy
Dry AMD and aHUS
Non-human primate (NHP) long-term toxicology
data and additional POC in Dry AMD
55% of Aevitas
4.5% Royalty
2.5% Annual Equity Dividend
AAV-ATP7A
AAV Gene Therapy
Menkes Disease
Nominate candidate for clinical development
71% of Cyprium
4.5% Royalty
2.5% Annual Equity Dividend
CK-103
BET Inhibitor
Solid Tumors
Potential Phase 1 initiation
19% of CKPT
4.5% Royalty
2.5% Annual Equity Dividend
ConVax
Vaccine
CMV Prevention & Control
IND filing
82% of Helocyte
4.5% Royalty
2.5% Annual Equity Dividend
CEVA-102
Cell Therapy
TBI, GvHD, ARDS, CHF,
Crohn’s (Off-the-Shelf)
IND filing
78% of Cellvation
4.5% Royalty
2.5% Annual Equity Dividend
CEVA-D
Bioreactor Device
Mechano-transduction
Device for Cell Therapies
Data published in Frontiers in Immunology (July
2022)
Portfolio includes product candidates in development at Fortress, at its majority-owned and majority-controlled partners, and partner companies that Fortress may otherwise have an economic interest in.
^ Potential peak sales are based on internal forecasts and/or market comps and assume approval in all denoted indications and are subject to change
†
Ownership estimated as of Sep 30, 2022
< $500M $500M - $1B
> $1B
13
Cosibelimab
Anti-PD-L1 mAb for treatment of metastatic and
locally advanced cSCC
Asset Overview
• Fully human IgG1 monoclonal antibody that is potentially
differentiated versus marketed PD-(L)1s
• ASCO 2022 presentation of top-line data in metastatic cSCC
(n=78) showed a confirmed objective response rate (ORR) by
independent central review in the modified intent to treat
population of 48.7% and 13.2% of patients achieving a
complete response in target lesions.
• In locally advanced cSCC, as of March 2022 data cutoff,
confirmed ORR by independent central review in 31 patients
was 54.8%.
• Composition of Matter patent issued in US, expiring no earlier
than 2038
Est. Market
PD
-L1 mAbs: $40B+/year
Status
BLA accepted by FDA for metastatic and locally
advanced cSCC in March 2023
Next Steps
Prescription Drug User Fee Act (PDUFA) goal date
of January 3, 2024
Near
-Term
Value Creation
Potential near
-term value creation from clinical
progression, regulatory approvals, royalties, and
CKPT equity appreciation
Product candidate in development at Checkpoint Therapeutics, Inc., an entity which was founded by Fortress and in which Fortress still
maintains a large ownership position.
14
CUTX-101
Subcutaneous injectable formulation of Copper
Histidinate for patients with Menkes Disease
Asset Overview
• Reported positive top-line clinical efficacy data, showing a
nearly 80% reduction in the risk of death (Hazard Ratio = 0.21,
p<0.0001)
Monetization Overview
• Upon FDA approval, Sentynl to acquire CUTX-101 for up to
$20M in upfront and regulatory milestone payments through
NDA approval – $8M was paid upon execution of the
agreement in February 2021
• Cyprium eligible to receive sales milestones totaling up to
$255M and tiered royalties (6% of net sales up to $75M, 17.5%
between $75M and $100M, 25% over $100M)
• Cyprium will retain 100% ownership over any FDA PRV that
may be issued at NDA approval for CUTX-101**
• FBIO owns ~71% of Cyprium
Est. Market
Estimated 50
-225 patients per year in the US
1
alone
with Menkes with potential for PRV worth
approximately $100M
-110M**
Status
Ongoing rolling submission of NDA to FDA
Next Steps
Expected to complete rolling submission of NDA in
2023^
Near
-Term
Value Creation
Potential near
-term value creation from milestones,
royalties, and PRV monetization
Product candidate in development at Cyprium Therapeutics, Inc., an entity which was founded by Fortress and in which Fortress still maintains a
majority ownership position.
1
Kaler SG, Ferreira CR, Yam LS. Estimated birth prevalence of Menkes disease and ATP7A-related disorders based on the Genome
Aggregation Database (gnomAD). Mol Genet Metab Rep. 2020;24:100602.
**In the event of a sale of a PRV by Cyprium, Cyprium would be obligated to make payments to the NIH and to holders of Cyprium’s 9.375%
Series A Cumulative Redeemable Perpetual Preferred (all as disclosed in Fortress’ public filings).
^ Cyprium is currently in a dispute with its contract manufacturing organization (the “CMO”), regarding the CMO’s attempt to terminate a Master
Services Agreement (together with related work orders, the “MSA”) between Cyprium and the CMO. Cyprium believes the CMO’s grounds for
purporting to terminate the MSA are without merit and is currently availing itself of all appropriate legal remedies in efforts to ensure that the
CMO abides by its obligations under the MSA and/or to pursue monetary damages claims against the CMO. To that end, Cyprium obtained a
temporary restraining order in August 2022 and a preliminary injunction in September 2022 from a court in New York State; the injunction
enjoined the CMO from terminating the MSA and prohibited the CMO from further attempts to terminate the MSA during the pendency of dispute
resolution procedures.
15
Dotinurad
URAT1 inhibitor for gout, chronic kidney disease,
and heart failure
Asset Overview
• Potential to be the most potent oral therapy for lowering serum
uric acid levels (sUA) with excellent safety profile
• Over 1000 patients treated in clinical trials
• Improved selectivity profile versus other uricosurics with
extensive data in humans showing excellent efficacy and safety
profile (>500 patients in Japan Phase 3 trials treated for up to
58 weeks)
• Dotinurad (URECE
®
tablet) was approved in Japan in 2020 as a
once-daily 1
st
line oral therapy for gout and hyperuricemia
• Unique product design and positioning that address key unmet
needs in US gout treatment paradigm
Est. Market
US: 2
-3 million refractory gout patients and >10
million diabetic CKD2/3 patients
EU and UK combined with potentially larger
addressable population than US
Status
Phase 1 ongoing in the United States
Next Steps
Phase 1 data expected in 2023
Near
-Term
Value Creation
Clinical development including first pivotal trial
starting in 2024
Product candidate in development at Urica Therapeutics, Inc., an entity which was founded by Fortress and in which Fortress still maintains a
majority ownership position.
FUJI YAKUHIN
16
MB-106
CD20 Autologous CAR-T Cell Therapy for NHL and
CLL
Asset Overview
• Third generation fully-human CD20 targeted autologous CAR-T
• FDA granted Orphan Drug Designation for Waldenstrom
macroglobulinemia (WM) in June 2022
• Latest data from FHCC Phase 1/2 trial
– Overall Response Rate (ORR) of 96% and Complete Response (CR)
rate of 75% was observed (n=28) across all dose levels across a
range of hematologic malignancies
– Three patients that had prior CD19-directed CAR T therapy have
responded to treatment
– Durable responses observed in wide range of hematologic
malignancies including FL, CLL, DLBCL, and WM (12 patients
remain in CR for greater than 1 year)
– Favorable safety profile was observed in all patients, with no grade 3
or 4 cytokine release syndrome or immune effector cell‐associated
neurotoxicity syndrome (ICANS)
Est. Market
Peak sales potential in U.S. of >$1 billion
Status
Continue to enroll patients in both the FHCC
investigator
-IND Phase 1 trial and multicenter
Mustang
-IND Phase 1 trial
Next Steps
Anticipate initial safety and efficacy data from the
Mustang
-IND trial in 2023
Near
-Term
Value Creation
Potential near
-term value creation from clinical
development progression and MBIO equity
appreciation
Product candidate in development at Mustang Bio, Inc., an entity which was founded by Fortress and in which Fortress still maintains a large
ownership position.
17
MB-109
IL13Rα2 CAR-T + HSV-1 Oncolytic Virus for
Glioblastoma Multiforme
Asset Overview
• MB-101 + MB-108 combination is designed to turn the tumor
microenvironment “hot”, which may improve the efficacy of CAR-
T cell therapy
– MB-108 HSV-1 (herpes simplex virus 1) oncolytic virus infects tumor
cells, which reshapes the tumor microenvironment (TME) through
cytokine release and recruitment of endogenous CD8-positive
effector T cells
– “Hot” TME may enable MB-101 CAR T cells to better infiltrate the
tumor mass, undergo activation and effect tumor cell killing
Est. Market
U.S. incidence of 12K
Status
MB
-101 (IL13Rα2 CAR-T) Phase 1 complete and
MB
-108 (HSV-
1 Oncolytic Virus) Phase 1 ongoing at
University of Alabama at Birmingham
Next Steps
File IND for Phase 1 combination trial for MB
-109
(MB
-101 + MB-108) in 2023
Near
-Term
Value Creation
Potential near
-term value creation from clinical
development progression
Product candidate in development at Mustang Bio, Inc., an entity which was founded by Fortress and in which Fortress still maintains a large
ownership position.
18
MB-107 & MB-207
Ex vivo lentiviral vector gene therapy for XSCID
“Bubble Boy” Disease
Asset Overview
• MB-107 for treatment of newborn XSCID patients and MB-207
for treatment of previously transplanted XSCID patients
• MB-107 published clinical results demonstrated**:
– Broad immune reconstitution with excellent safety profile
– 100% T cell reconstitution with no loss of immunity
– Up to 5 years of follow up for patients
• MB-207 published clinical results demonstrated***:
– T cell reconstitution achieved in all patients; no loss of immunity
• No insertional mutagenesis observed in either program
FDA and EMA Designations
Est. Market
107: XSCID incidence of ~1 in 225K newborns per
year (worldwide)
207: ~400 patients living with XSCID post
-
transplant
in the US and ~650
patients in ex-US high/mid-
income markets
Status
Entering pivotal multi
-center Phase 2 trials
Next Steps
MB
-107: First patient dosing in Phase 2 reg. trial in
newly diagnosed patients expected 2023
MB
-207: First patient dosing in Phase 2 reg. trial in
previously transplanted patients expected 2023
Near
-Term
Value Creation
Potential near
-term value creation from clinical
development progression, potential approvals/PRV
monetizations
^
and MBIO equity appreciation
Product candidates in development at Mustang Bio, Inc., an entity which was founded by Fortress and in which Fortress still maintains a large
ownership position.
** ASGCT 2022 update by Mamcarz E, et al
***De Ravin SS et al. Blood (2019) 134 (Suppl 1): 608.
^ Recent data suggests PRVs may be worth ~$100M to ~$110M, both MB-107 and MB-207 are each eligible for PRVs
MB-107 MB-207
FDA Designations
RMAT
✓
(requesting in 2023)
Rare Pediatric Disease
✓ ✓
Orphan Drug
✓ ✓
EMA Designations
Orphan Drug
✓ ✓
ATMP
✓ ✓
PRIME
✓
19
CAEL-101*
Monoclonal antibody (mAb) for the treatment of
patients with amyloid light chain (“AL”) amyloidosis
Asset Overview
• Granted Orphan Drug designations in the U.S. and EU
• No FDA, EMEA, or PMDA approved therapies for indication
• Potentially understated market size given AL Amyloidosis often
misdiagnosed
Monetization Overview
• The agreement triggered upfront payment of approximately
$150M to Caelum shareholders (of which approximately
$56.9M** was payable to Fortress Biotech) and provides for
additional potential payments to Caelum shareholders totaling
up to $350M upon the achievement of regulatory and
commercial milestones
• Fortress is eligible to receive approximately 42% of all proceeds
from the transaction
*As Caelum was acquired by AstraZeneca in 2021, Fortress may not be apprised of ongoing developments pertaining to CAEL-101 to the same
degree that Fortress had been prior to such acquisition; accordingly, the information presented on this slide may not reflect the latest disposition
of the product candidate
**In each case, figures are net of transaction expenses and escrow. Fortress remains eligible to receive up to $6 million of the $15 million in
escrow.
Est. Market
30K to 45K patients in U.S. and EU with ~4.5K
newly
-diagnosed patients (U.S.) per year
Status
Two ongoing global Phase 3 Trials
Next Steps
Ongoing enrollment in the CAELUM CARES Phase
3 program
Near
-Term
Value Creation
AstraZeneca acquired Caelum Biosciences on
October 5, 2021; potential additional near
-term
value from milestone payments
Strategy & Examples
20
Our strategy is focused on finding and developing lower risk opportunities
efficiently, which creates inherent advantages
21
Key Success Drivers
Deep pharmaceutical
development experience
across our team and network
of leading advisors
Focused BD search
and evaluation criteria
that consistently finds assets
with high unmet need
and balanced clinical /
development risk
Lower risk investment
and development strategy
due to diverse pipeline
across therapeutic area,
modality, stage and number
of shots on goal
Proven track record
in licensing attractive
therapies fast and first,
which avoids excessively
competitive processes
Differentiated business
model provides synergistic
benefits of scale and
increased opportunity
Fortress pipeline and structure presents a de-risking strategy with
diversified exposure and many shots on goal
22
Preclinical
Late-Stage and Regulatory
Execution
Commercial Scaling
Fortress Asset Pipeline
Therapeutic Areas/Modalities
Dermatology Rare Disease Rheumatology
Oncology / Hematology Other
Early-to-Mid-Stage Clinical
Progression
Diversified portfolio with programs in
multiple therapeutic areas across all
development and commercial stages
‒ Approach reduces risk to portfolio building versus
single asset/platform competitors
‒ Exposure to various technologies and therapeutic
areas, all with significant upside potential
14K + Clinical-stage
Asset Universe
Core expertise in quickly identifying and in-licensing/acquiring de-risked
therapeutics, creating a continuous BD engine
23
Fortress NewCo
Fortress Existing
Partner Company
High unmet need for
patients
Proof-of-concept data or
validated mechanism/pathway
Capital efficient
development path
Market opportunity
Fortress Portfolio
24
Fortress develops and markets therapeutic products through a portfolio of
majority-owned and controlled companies
I/O & Targeted Oncology Menkes Disease
CAR-T Cell Therapy
& Gene Therapy
Dermatology
• Each subsidiary/partner company is focused on clinical and commercial execution of their products with support from Fortress
• Partner company format allows for flexibility to pursue deals, partnerships and fundraising
• Each partner company provides multiple ways to create meaningful streams of revenue and equity
Rheumatology:
Gout
Gene Therapy:
Dry AMD, aHUS
Immunotherapy:
Cytomegalovirus
Cell Therapy:
Traumatic Brain Injury
Pain + Neuro Disorders
Novel Oligonucleotide
Delivery Platform
Fortress and our partner companies share mutually beneficial relationships
25
*Applicable to most Fortress subsidiaries, but not all
Benefits of Fortress and Subsidiary/Partner Company
Relationship
• Fortress provides all business development efforts including
active identification of synergistic portfolio assets
• Fortress supports with ongoing operational, strategic,
administrative and finance support
• Most subsidiary/partner companies provide Fortress an annual
2.5% equity dividend and a 4.5% royalty on net sales*, which
incentivizes Fortress to continue to build value over time
• Subsidiary/partner companies and Fortress can share resources,
personnel, and expertise
Asset
Monetizations
Fortress share of
distributions from PRV
sales* and asset
monetizations
Upon FDA approval,
Sentynl to acquire CUTX-101
and pay to Cyprium up to $20M,
sales milestones up to $255M,
and royalties^ + PRV monetization
of ~$100M-$110M
(Fortress owns ~70% of Cyprium)
26
Shareholder value is generated through asset monetizations, equity
holdings/appreciation, and equity dividend and royalty revenues
+
* Priority Review Vouchers (PRV) are granted by the FDA for certain rare pediatric disease approvals and can be transferred between companies (recent PRVs have sold for $100M-$110M)
** 10% of upfront payment held in escrow to satisfy potential indemnification obligations, if any
*** Applicable to most Fortress subsidiaries, but not all
^ All Payments subject to potential reductions as per Sentynl Agreement, refer to Footnote regarding CUTX-101 on Slide 14
Fortress Biotech Value Drivers
+ =
Equity
Holdings
Fortress holds a large
stake of equity in each
subsidiary company and
shareholders gain value
during appreciation of
equity or through exits
AstraZeneca acquired Caelum
for an upfront payment of ~$150M**
and additional potential payments
totaling up to $350M payable
to Caelum Shareholders
(Fortress eligible to receive ~42%)
Dividend &
Royalty Revenue
Subsidiary companies***
pay to Fortress an
annual equity dividend
worth 2.5% of subsidiary
market cap and a 4.5%
royalty on net sales
Increasing
Shareholder
Value
Recent/near-term monetization opportunities
27
* Remains eligible to receive up to $6 million of the $15 million in escrow
Blue text represents proceeds received already by respective company and Fortress
^ Refer to Footnote regarding CUTX-101 on Slide 14
Cyprium^ Development & Asset
Purchase Agreement with Sentynl
February 2021
Caelum Acquired by AstraZeneca
October 2021
• Upon FDA approval, Sentynl to acquire CUTX-101 for up to
$20M in upfront and regulatory milestone payments through
NDA approval – $8M was paid upon execution of the
agreement in February 2021
• Cyprium eligible to receive sales milestones up to $255M
and royalties on CUTX-101 net sales are also payable:
– 6% due on portion of annual net sales up to $75M
– 17.5% due on portion of annual net sales between $75M and
$100M
– 25% due on portion of annual net sales over $100M
• Cyprium will retain 100% ownership over any FDA PRV that
may be issued at NDA approval for CUTX-101. Recent data
suggests PRVs may be worth $100M to $110M
• Option exercise triggered upfront payment of
approximately $150M to Caelum shareholders, of which
~$56.9M* was paid to Fortress
• Additional potential payments to Caelum shareholders
totaling up to $350M, payable upon the achievement of
regulatory and commercial milestones
• AstraZeneca intends to advance and accelerate the Phase 3
development of CAEL-101 for light chain (AL) amyloidosis
• Fortress is eligible for up to a total of $212M in proceeds
(~42%) from this transaction
$75 million debt
financing closed with
Runway Growth Capital
Fortress found the
CD20 CAR-T at FHCC*
Fortress enabled Mustang’s $94.5 million
private placement financing and buildout
of cell processing facility
Fortress found two CAR-T
assets from City of Hope and
founded Mustang Bio
Fortress identified an ex-vivo
lentiviral gene therapy asset
from St. Jude and NIH
Fortress identified an
oncolytic virus (C134) at
Nationwide Children’s
Fortress found a preclinical in vivo CAR-T
asset (Mayo Clinic) and Phase 1/2 RAG1-SCID
asset (Leiden University Medical Center)
28
Assets In-Licensed
To Mustang Portfolio
Fortress Value Add +
Key Events
Mustang Bio
Founded
Two CAR-T assets
(MB-101 & MB-102)
CD20 CAR-T
(MB-106)
LVV gene therapy
(MB-107/MB-207)
C134 Oncolytic Virus
(MB-108)
*Fred Hutchinson Cancer Center (FHCC)
in vivo CAR-T technology and
RAG1 program (MB-110)
Fortress has continuing equity and royalty interests in Mustang, incentivizing us to keep building value in Mustang
2015 2016 2017 2018 2019 2020 2021
Adding more horsepower to Mustang over time
2022
29
Caelum Biosciences – acquired by AstraZeneca in Oct 2021
Fortress and Caelum complete a third-party
financing of Convertible Notes raising $9.9M
3Q 2017
Caelum announces positive Phase 1a/1b data, which
demonstrated that CAEL-101 improved organ function
in AL amyloidosis
December 2017
Caelum initiates Phase 2 study
of CAEL-101 in AL Amyloidosis
March 2020
Caelum initiates Phase 3 studies
of CAEL-101 in AL Amyloidosis
September 2020
Agreement includes additional potential payments
totaling up to $350M payable to Caelum Shareholders
upon the achievement of regulatory and commercial
milestones (Fortress eligible to receive ~42%)
2022 & Beyond
January 2017
October 2021
January 2019
Caelum
Founded
Caelum
Acquired
R&D Funding
Agreement
Fortress found a novel antibody at Columbia University
being evaluated in a Phase 1a/1b study with
promising interim data and licensed the asset
AstraZeneca acquired Caelum for an upfront payment
of ~$150M, of which ~$56.9M was paid to Fortress*
Caelum entered into a strategic agreement with Alexion
Pharmaceuticals to advance CAEL-101 where Alexion
would pay up to $60M for continued development/equity
and have an exclusive option to acquire Caelum
Note: *AstraZeneca’s Alexion acquired Caelum Biosciences on 10/5/2021 for up to $500 million, including $150 million upfront and up to $350 million in future contingent milestone payments. FBIO received ~$56.9 million of such upfront
amount (and remains eligible to receive up to $6 million of the $15 million in escrow); FBIO also is eligible to receive ~42% of the proceeds from all future milestone payments.
30
2016
2017
2018
2019
2020
2021
Journey Net Revenue (2016 – 2021)
$ in millions
*CAGR for net sales growth from 2016-2021
Journey of constant growth and portfolio expansion
Launched / acquired 3 new products
in 2021, including Phase 3 asset DFD-29
for rosacea
Record $63.1M in 2021 net revenue
and $57.7M in 2022 Q1-Q3 net revenue
Acquired 2 new commercial products
from Vyne Therapeutics in January 2022
(AMZEEQ and ZILXI)
Top-tier academic & commercial partners
FUJI YAKUHIN
31
THANK YOU!