Novel AAV-based Gene Therapies For

Complement Mediated Diseases

Non-Confidential Corporate Presentation

November 2021

Forward Looking Statements

Aevitas Therapeutics, Inc. is a subsidiary of Fortress Biotech, Inc.

Some statements in this presentation that are not descriptions of fact may be forward-looking statements, for which we and our majority-owned parent

company Fortress Biotech claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of

1995. Such statements include, but are not limited to, any statements relating to our growth strategy, products and product development programs.

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, and financial condition. 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 the Securities and Exchange

Commission filings of our parent, Fortress Biotech, Inc. 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.

Aevitas – Our Mission

Aevitas

advances AAV gene therapies of short-form human factor H (sFH)

to restore homeostasis in disorders of complement dysregulation:

dry age-related macular degeneration (Dry AMD),

atypical hemolytic uremic syndrome (aHUS)

and other disorders

SCIENTIFIC ADVISORY BOARD

Management and SAB with Deep Experience in

Complement Space, Ophthalmology and AAV Gene Therapies

Lindsay Rosenwald, MD

Executive Chairman

• Currently also Chairman, President, and CEO at Fortress Biotech (NASDAQ: FBIO)

• Previously Founder/Board Member of multiple biotech companies including: Caelum Biosciences (acquired by AstraZeneca),

Cougar Biotechnology (acquired by JNJ), Chelsea Therapeutics (acquired by Lundbeck), Indevus Pharma (acquired by Endo),

Biocryst Pharmaceuticals, Keryx Pharmaceuticals, CorMedix, Ziopharm Oncology

Markus Peters, PhD, MSc

Chief Executive Officer

• Previously COO at Gemini Therapeutics (NASDAQ: GMTX)

• Previously CCO at Agilis Biotherapeutics (acquired by PTC Therapeutics)

• Previously VP of Global Marketing/Commercial at Synageva (acquired by Alexion) and Head of Global Market Nephrology and

Transplant at Alexion (launched Soliris for aHUS)

AJ Ross

Chief Business Officer

• Currently also VP of Business Development at Fortress Biotech and Chief Business Officer at Oncogenuity (a Fortress subsidiary)

• Previously healthcare investment banker at Cowen & Co and Roth Capital

Dr. Wenchao Song

University of Pennsylvania

• Inventor of Aevitas’ engineered, fully functional, shortened Factor H

• Professor of Pharmacology at UPenn with extensive experience and research on complement-mediated inflammatory,

autoimmune, and thrombotic vasculopathy disorders

Dr. Guangping Gao

University of Massachusetts

Medical School

• Professor of Microbiology and Physiological Systems at UMass Medical School

• Internationally recognized gene therapy researcher who has played a key role in the discovery and characterization of new families

of AAV serotypes

• Former ASGCT President

Dr. Cathy Bowes Rickman

Duke University

• Professor of Ophthalmology and Cell Biology at Duke

• Leading researcher in age-related macular degeneration and in the development and use of murine models to elucidate the

mechanisms of pathology and progression for AMD

• Research over the last decade has focused on studying the impact of the complement system on the pathobiology of AMD

EXECUTIVE MANAGEMENT

Executive Summary

Aevitas advances AAV gene therapies of short-form human Factor H (sFH) to restore homeostasis in disorders of

complement dysregulation.

Engineered sFH is fully functional, and its transgene can be cloned into AAV; full length FH is too large to fit into

AAV.

Approach

Good vector genome transfer to ocular target tissues with intravitreal administration was demonstrated in

Yorkshire pigs. Transgene product sFH was present in all eye compartments with potentially clinically relevant

FH levels in the porcine eye.

Supraphysiological expression of sFH with subretinal administration was shown in non-human primate eye.

sFH restored complement regulation in eyes of FH knockout mice.

Key anticipated milestones: Pre-IND meeting mid 2022; IND filing mid 2023.

Dry AMD

Long-term systemic expression of sFH in aHUS mice. sFH reversed mortality, completely prevented phenotype,

and restored key hematological parameters.

Key anticipated milestones: Pre-IND meeting mid 2022; IND filing mid 2023.

aHUS

CDMOs are under contract for GMP manufacturing.

Manufacturing

Achievement of above milestones is conditioned on multiple factors, including ability to raise sufficient funds or partner the applicable program, and CRO capacity

Lead Programs in Dry AMD/Geographic Atrophy and aHUS

aHUS: atypical hemolytic uremic syndrome

AMD: age-related macular degeneration

sFH: shortened human Factor H

DISCOVERY

PRECLINICAL

PHASE 1 ANTICIPATED NEAR-TERM MILESTONES

AAV.sFH for Dry AMD/

Geographic Atrophy

• Confirmatory NHP study for development

candidate & route of administration 1H ‘22

• Initiation of GLP tox study in 1H ‘23

• Begin GMP manufacturing process 2H ‘22

• Pre-IND meeting mid ‘22, IND filing mid ‘23

AAV.sFH for aHUS

• Long-term biodistribution mouse study in

1H ‘22

• Initiation of GLP tox study in 2H ‘22/1H ‘23

• Start of GMP manufacturing process 2H ‘22

• Pre-IND meeting mid ‘22, IND filing mid ‘23

AAV.sFH for Undisclosed

Targets

• Complement dysregulation plays a role in

multiple diseases, Aevitas intends to

explore the potential for treatment with

AAV-sFH

SYSTEMIC

OCULAR

Achievement of above milestones is conditioned on multiple factors, including ability to raise sufficient funds or partner the applicable program, and CRO capacity

Classical Pathway Lectin Pathway Alternative Pathway

C1q MBL, ficolins C3

C4b2b

(C3 convertase)

C3a

C3b

C3bBb

(C3 convertase)

Factor H

C4b2bC3b

(C5 convertase)

C3bBbC3b

(C5 convertase)

C5a

C5b

C5b-9

(Membrane Attack Complex)

Amplification

Loop

Factor H is a Key Regulator of the Complement System

• The complement system is an essential component of the innate

immune response. It constitutes a cascade of plasma proteins and cell

surface receptor interactions which can be triggered via 3 pathways

(alternative, classical and lectin).

• Factor H (FH) is a master regulator of the complement system:

 Inhibits assembly of the C3 and C5 convertase enzymes via

competition with factor B for C3b binding

 Facilitates disassembly of the convertases by displacing bound

factor Bb (‘decay accelerating activity’)

 Inactivates C3b by acting as a cofactor for the serine protease

factor I (‘cofactor activity’).

• Dysregulation of the complement system through genetic

predisposition and/or environment can lead to autoimmune and

inflammatory diseases.

• FH variants are among the leading genetic causes of dysregulation.

• Aevitas’ short-form FH (sFH) is expected to restore complement

regulation and therefore

 Inhibit the disadvantageous effects of complement activation,

including overshooting immune responses and inappropriate cell

lysis, while

 Retaining the advantageous effects, including repair of oxidative

damage and clearance of extracellular debris.

 Complement pathway inhibitors may block both the detrimental

and beneficial effects of complement activation.

Inflammation chemotaxis,

cell activation

Inflammation chemotaxis,

cell activation

Opsonization

Cell lysis

Significant Industry Investment into the Complement Space

Source: GlobalData, Public Filings

MBL, MASP-2

Complement Regulation

Complement B, P, D

Complement C5a/C5aR

Complement C3

Complement C5

Complement C1

Classical Pathway Lectin Pathway Alternative Pathway

C1q MBL, ficolins C3

C4b2b

(C3 convertase)

C3a

C3b

C3bBb

(C3 convertase)

Factor H

C4b2bC3b

(C5 convertase)

C3bBbC3b

(C5 convertase)

C5a

C5b

C5b-9

(Membrane Attack Complex)

Amplification

Loop

Variants in Complement Regulatory Proteins are Linked to

Various Diseases

Aevitas’ AAV.sFH is initially targeting dry age-

related macular degeneration (Dry AMD) and

atypical hemolytic uremic syndrome (aHUS)

Infectious diseases, sepsis, anaphylaxis

I/R injury

Paroxysmal nocturnal hemoglobinuria

Dense deposit disease

Psoriasis

Myasthenia gravis

Systemic lupus nephritis

Multiple sclerosis

Transplant rejection

Cancer

Age-related macular degeneration

Asthma

Myocardial infarction

Atypical hemolytic uremic syndrome

Crohn’s disease

Alzheimer’s disease

Stroke

Engineering Factor H to Make it Suitable for Delivery by AAV

• Factor H: 155 kd protein with 20 short consensus repeat (SCR) units, making it too

large to fit in AAV

• N-terminus is the critical site for co-factor activity/decay accelerating activity, the

C-terminus for cell surface regulation

• Collaboration with the Song (UPenn) and Gao (UMass Medical) labs led to an optimized

sFH which can fit into AAV with robust expression and full functionality

Source: Ferreira et al. J Immunol 2006 Nov 1; 177(9): 6308-15; Rodriguez de Cordoba et al, Clin and Experimental Immunology 2008, 151: 1-13

951 192 3 4 6 7 8 10 11 12 13 14 15 16 17 18 20

4012bp

N glycosylation site

Full length FH

HC3B BINDING

0.5

1.5

2.5

OD450

Protein Conc (nM)

hfH

hsfH

0.5

1.5

2.5

OD450

Protein Conc (nM)

HEPARIN BINDING

Confirmed Pharmacological Functionality and Dose Response

in Mice

hC3b/Heparin binding is a key indicator of sFH functionality

Source: Aevitas Internal

DOSE RANGING IN WT B6 MICE

sFH (uM)

Serum sFH near normal

human level at 3e10 vg

(1.5e12 vg/kg) dose

hfH

hsfH

AAV.sFH for Dry AMD

Dry AMD Overview

• Serious disease with high and growing prevalence

 ~15M Dry AMD patients in the US (90% of all AMD)

 1.3M patients with advanced AMD or Geographic Atrophy, GA (10% of all AMD)

 Expected to grow 30% by 2040 due to aging of the population

 42% of GA patients are legally blind

• ~40% of Dry AMD patients have a pathogenic variant of FH

 FH is the most significant genetic driver of DryAMD, with estimated > 6M patients in the US

 Y402H is the most common variant and leads to an ~8x higher likelihood of developing the disease

 R1210C, a rare variant, leads to ~20x higher likelihood of disease and is associated with other CFH diseases

like aHUS

• There are currently no approved treatments for Dry AMD or Geographic Atrophy

• Wet AMD is 10% of all AMD and a $13B market

Source: JAMA Ophthalmol. 2015 Jul; 133(7): 785–791, PNAS February 26, 2019 116 (9) 3703-3711. National Eye Institute. Facts About Age-Related Macular Degeneration. National Institutes of Health.

ttps://nei.nih.gov/health/maculardegen/armd_facts. Accessed September 9, 2015

Dry AMD and Wet AMD Natural History

Geographic Atrophy

(GA)

Source: Wong et al Lancet Glob Health 2014;2: e106-16; Klein et al., Arch Ophthalmol 129(1), 2011, 75-80, http://www.afb.org/info/blindness-statistics/key-definitions-of-statistical-terms/25. Accessed September 9, 2015; National Eye Institute.

Facts About Age-Related Macular Degeneration. National Institutes of Health.

https://nei.nih.gov/health/maculardegen/armd_facts. Accessed September 9, 2015

10%

80%

Wet AMD

(Neo-vascularization)

Summary of Selected Dry AMD Data for AAV.sFH

• Good AAV vector genome transfer to ocular target tissues (retina, RPE and choroid) was demonstrated in

Yorkshire pigs with intravitreal administration of several AAV serotypes. Vector genome levels observed in

pigs were comparable to published data for AAV vectors administered intravitreally in NHPs

• Transgene product sFH was present in all eye compartments with potentially clinically relevant FH levels in

the porcine eye.

Good Vector Genome Transfer in Pig Eyes with Intravitreal Administration

• Dose-dependent sFH expression was achieved in RPE/choroid/sclera.

• FB depletion, an important biomarker for complement overactivation, was reversed only in AAV.sFH

injected eyes.

sFH Restored Complement Regulation in Eyes of FH Knockout Mice

Invest Ophthalmol Vis Sci 2017; 58:5792-5801

FH knockout (k/o) mouse model: Variable presentation of drusen and photoreceptor damage; FH k/o mice expressing the Y402H

mutation develop an AMD phenotype in age and diet dependent manner

Supraphysiological sFH Levels in NHP Eye with Subretinal Administration

• Supraphysiological sFH levels were achieved in all ocular target tissues.

AAV.sFH is Well Differentiated in Dry AMD

Aevitas Gyroscope Gemini AGTC Apellis Iveric Hemera

(Janssen)

Description

AAV-short-frm FH

IVT or SC

IND Enabling

AAV-Factor I

Subretinal

Ph2

Recombinant FH

IVT

Ph2

AAV-trunc’d-FH

Subretinal

IND Enabling

C3 inhibitor

IVT

Ph3

C5 inhibitor

IVT

Ph3

AAV-soluble CD59

IVT

Ph 1, (Ph2: WetAMD)

Complement

Regulator

Y Y Y Y N N Y

Non-surgical

Outpatient Treatment

Y N Y N Y Y Y

Potential

One-time Therapy

Y Y N Y N N Y

Expected Use in GA

with FH Variant

(40% of GA)

Y TBD Y Y Y Y TBD

Expected Use

in All Other GA

TBD Y TBD TBD Y Y TBD

• Subretinal injection is deemed unsuitable for the AMD population because the age of most AMD patients and the integrity of their retina may make

subretinal injections a significant risk to cause damage. Subretinal injections are a surgical procedure and require hospitalization, thus creating significant

demand on resources and associated commercial challenges, given the size of the addressable patient population.

• One-time treatment of intermediate Dry AMD patients who are on average younger and less physically impacted by their condition than GA patients is

believed to be preferred over and easier to implement than chronic monthly or bi-monthly treatment (Gemini, Apellis, Iveric)

List of Dry AMD treatments in development (selection)

IVT: intravitreal; SR: subretinal; SC: suprachoroidal administration; FI: Complement Factor I

Recent Developments in Ophthalmologic Gene Therapy

Lending additional support to the Aevitas approach to develop AAV.sFH:

• Further clinical proof of concept for the therapeutic role of complement inhibition and

regulation in Dry AMD (Apellis and Gyroscope)

• Safety and efficacy of intravitreal AAV gene therapy even at higher doses (>10e

) in Wet

AMD (Adverum), and in Retinitis Pigmentosa and Leber Hereditary Optic Neuropathy

(GenSight)

• Safety and efficacy of suprachoroidal AAV gene therapy in Diabetic Retinopathy and Wet

AMD (RegenXBio)

• Confirmation of the Aevitas strategy to develop an AAV gene therapy with the short form of

FH (Gemini discontinuing their AAV gene therapy program in Dry AMD with full-length FH)

• Continued attractiveness of the field to pharma (e.g. AbbVie collaboration with RegenXBio

and Novartis acquisition of Arctos, both announced in 2H 2021)

Source: Corporate Websites, Publications at XIX

International Symposium on Retinal Degeneration 2021, Publications at the 39

Annual Meeting of American Retina Specialists 2021

Anticipated Upcoming Milestones

• Studies testing routes of administration and capsids

• Analysis of harvested human eyes to determine target FH levels for dose selection in

clinical trials

• Confirmatory NHP study to finalize development candidate & route of administration in

1H ‘22

• Initiation of GLP tox study in 1H ‘23

• Start of GMP manufacturing process in 2H ‘22

• Pre-IND meeting mid ‘22

• IND filing mid ‘23

Near Term Studies & Activities

Achievement of above milestones is conditioned on multiple factors, including ability to raise sufficient funds or partner the applicable program, and CRO capacity

AAV.sFH for aHUS

aHUS Overview

• Serious chronic disorder with high morbidity and increased mortality

 Complement overactivation can lead to thrombotic microangiopathy with associated anemia,

thrombocytopenia, kidney failure, other organ damage and death

• Ultra-rare disease

 3,500 patients in the US (prevalence ca. 10 in 1,000,000)

• FH mutations are largest genetic driver representing 30% of cases

 Overall, more than 50% of aHUS has genetic causes

• Factor H mutation is associated with higher risk of treatment relapse, end-stage renal

disease and relapse following transplant

• Significant unmet need for one-time (“one-and-done”) treatment

 Currently only chronic IV anti-C5 antibody therapy (Soliris, Ultomiris) available

 Annual cost of therapy between $500k – $700K ($1.8Bn projected 2022 sales)

Med Genet. 2018; 30(4): 400–409, Hematol Rep. 2017 Jun 1; 9(2): 7053, J Immunol Methods. 2018 Oct;461:15-22, Nefrologia. 2015;35(5):421-47

Source: Rarediseases.org, Evaluate Pharma, National Organization of Rare Disorders; Afshar-Khargan, Hematology Am Soc Hematol Educ Program. 2016 Dec 2; 2016(1): 217–225

aHUS mice pre-treated with murine anti-C5 for 4 weeks prior to AAV.sFH at week 8 and removal at week 9

Long Term POC with aHUS Mouse Model

Experimental Procedure

sFH (Human)-AAV

(1 X 10

/Mouse)

CBC Analysis

Serum/Plasma Collection

Pre

Tissue Collection

R/R

(Male)

Weeks of Age

Prior anti-C5 mAb Treatment

BB5.1 (1mg, Twice A Week)

BW & CBC Check

4 8 20 32

Summary of Selected aHUS Data

• 60% of AAV Control mice die within 7 weeks of removing murine anti-C5

• 100% survival for AAV.sFH treated mice out 32 weeks

Long Term Systemic Expression and Survival in aHUS

Mice

Complete Prevention of Measured aHUS Phenotype

• Platelets, hemoglobin and reticulocytes

Restoration of Normal Complete Blood Count Levels

Anticipated Upcoming Milestones

• Long-term biodistribution mouse study in 1H ‘22

• Initiation of GLP tox study in 2H ‘22/1H ‘23

• Start of GMP manufacturing process in 2H ‘22

• Pre-IND meeting mid ‘22

• IND filing mid ‘23

Near Term Studies & Activities

Achievement of above milestones is conditioned on multiple factors, including ability to raise sufficient funds or partner the applicable program, and CRO capacity

Aevitas Engineered Factor H Patent Portfolio

The applications listed in the table below have been licensed by Aevitas from the University of Pennsylvania.

These applications all claim priority to U.S. Provisional Application 62/232,008, which was filed 9/24/2015,

and international application PCT/US2016/053347, which was filed 9/23/2016 and published as WO 2017/053732.

Any patent issuing from the applications listed below should expire no earlier than September 23, 2036.

COUNTRY APPLICATION NO. STATUS

U.S. 15/762,721 Allowed – Issue Fee Due May 11, 2021

U.S. 17/156,023 Continuation application filed January 22, 2021 – Awaiting examination

Europe 16849709.7 Office Action Response filed October 25, 2020 – Awaiting further examination

Australia 2016326627 Substantive examination has not yet begun

Brazil BR112018005684-7 Substantive examination has not yet begun

Canada 2,999,299 Substantive examination has not yet begun

China 201680064219 Substantive examination has not yet begun

Israel 258024 Substantive examination has not yet begun

Japan 2018-515539 Office Action Response filed November 19, 2020 – Awaiting further examination

Russia 2018114907 Issued under Patent # 2727411 on July 21, 2020

South Korea 10-2018-7011034 Substantive examination has not yet begun

Hong Kong 18111116.6 Published February 1, 2019 under Pub. # HK1251484

Aevitas Engineered Factor H Patent Portfolio

The applications listed in the table below have been licensed by Aevitas from the University of Massachusetts.

U.S. Provisional Application 62/831,870, which was filed 4/10/2019, and international application

PCT/US2020/027452, which was filed 4/9/2020 and published as WO 2020/210480.

Any patent issuing from the applications listed below should expire no earlier than April 9, 2040.

COUNTRY APPLICATION NO. STATUS

U.S. 62/831,870 Expired April 10, 2020, converted into PCT on April 9, 2020

PCT PCT/US2020/027452 30 Month National Phase Deadline is October 10, 2021

Recent M&A and Capital Raising Activity for Complement-

Mediated Diseases and Ophthalmological Gene Therapy

ACQUIRER ACQUIREE PRIMARY ASSET(S)

Acquisition of intravitreal gene therapy HMR59 for Geographic Atrophy

HMR59 designed to increase the ability of retina cells to make a soluble form of CD59

Undisclosed

Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease

Undisclosed (Development

Collaboration)

AAV delivery of optogenetic therapies for the treatment of blindness Undisclosed

Co-develop and commercialize RGX-314, an investigational gene therapy for wet age-

related macular degeneration, diabetic retinopathy and other chronic retinal diseases

$370MM Upfront

$1.38B in Milestones

Source: Company Press Releases, FierceBiotech

ACQUISITION PRICE

COMPANY RAISE

Gemini raised $216 million through a SPAC merger in Feb 2021 $216MM

Gyroscope raised $148 million in March 2021 in a Series C $148MM

AMOUNT

Executive Summary

Aevitas advances AAV gene therapies of short-form human Factor H (sFH) to restore homeostasis in disorders of

complement dysregulation.

Engineered sFH is fully functional, and its transgene can be cloned into AAV; full length FH is too large to fit into

AAV.

Approach

Good vector genome transfer to ocular target tissues with intravitreal administration was demonstrated in

Yorkshire pigs. Transgene product sFH was present in all eye compartments with potentially clinically relevant

FH levels in the porcine eye.

Supraphysiological expression of sFH with subretinal administration was shown in non-human primate eye.

sFH restored complement regulation in eyes of FH knockout mice.

Key anticipated milestones: Pre-IND meeting mid 2022; IND filing mid 2023.

Dry AMD

Long-term systemic expression of sFH in aHUS mice. sFH reversed mortality, completely prevented phenotype,

and restored key hematological parameters.

Key anticipated milestones: Pre-IND meeting mid 2022; IND filing mid 2023.

aHUS

CDMOs are under contract for GMP manufacturing.

Manufacturing

Achievement of above milestones is conditioned on multiple factors, including ability to raise sufficient funds or partner the applicable program, and CRO capacity

Aevitas Contact Details:

Markus Peters (CEO) – mpeters@aevitastx.com

AJ Ross (CBO) – aross@aevitastx.com

David Jin (FBIO Corp Dev) – djin@fortressbiotech.com

Aevitas Therapeutics, Inc.

Subsidiary of Fortress Biotech

(NASDAQ: FBIO)

2 Gansevoort Street, 9th Floor

New York, NY 10014