Corporate Presentation

July 2025

Some statements in this presentation that are not descriptions of fact may be forward-looking statements, for which we 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 relating to our growth strategy; government

regulation; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating

to research, development and manufacturing activities; uncertainties relating to preclinical and clinical testing; risks relating to

regulatory approvals and eligibility of our product candidates under certain government regulations; risks pertaining to drug

safety; our dependence on third-party suppliers; our ability to attract, integrate, and retain key personnel; our need for additional

funds; 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.

Cover Page Image Sources: Kaler SG, NIH

Forward Looking Statements

Cyprium Overview

Cyprium Therapeutics is

focused on the development of novel

therapies for the treatment of Menkes

disease, a rare and fatal pediatric

condition, and related copper

metabolism disorders

CUTX-101*

(Copper Histidinate Injection)

▪ PDUFA target action date of Sept. 30, 2025

▪ Eligible for Priority Review Voucher worth $100M+

AAV-ATP7A

Gene Therapy

▪ Preclinical; expect to nominate candidate for clinical

development in next 12 months

▪ Granted Orphan Drug Designation from FDA

*Cyprium completed Asset Transfer to Sentynl Therapeutics, Inc. in December 2023; Sentynl to complete development of

CUTX-101 and responsible for commercialization

AAV-ATP7A Gene Therapy for Menkes Disease

▪ Mottled–brindled mouse model recapitulates the disease phenotype

▪ Atp7a

mo-br

phenotype

▪ A 6 bp in-frame deletion in exon 11 of Atp7a

▪ Depigmented coat color and curly whiskers

▪ Premature death (~13 days of age)

▪ Poor growth; Neurological symptoms

▪ Low brain copper; abnormal catecholamine levels

▪ NICHD developed several constructs for reduced size, codon-optimized AAV-

ATP7A gene therapy

▪ AAV-ATP7A + SC copper histidinate administration led to:

▪ Improvements in muscle strength, balance and coordination in preclinical

model

▪ Improved biochemical phenotype (Cu and catecholamine)

▪ Improved survival

Source: Stephen G. Kaler, MD; Haddad et al, 2018

CUTX-101 for Menkes Disease

▪ CUTX-101 (Copper Histidinate Injection)

▪ NDA accepted and granted priority review by FDA; target PDUFA action date of Sept. 30, 2025

▪ Sentynl Therapeutics assumed development from Cyprium in December 2023

▪ Sentynl to complete development of CUTX-101 and responsible for commercialization

▪ Sentynl also continuing CYP-001 (Intermediate-Size Expanded Access Protocol (NCT04074512)) to provide

CUTX-101 for newly diagnosed Menkes disease patients

▪ Received $4.5M milestone; Cyprium remains eligible to receive royalties and up to $129M in aggregate

development and sales milestones

▪ Cyprium retains 100% ownership over any FDA Priority Review Voucher (PRV) that may be issued

▪ Previously reported positive topline clinical efficacy data showed a nearly 80% reduction in the risk of death

(Hazard Ratio = 0.21, p<0.0001)

▪ FDA granted Breakthrough Therapy, Orphan Drug, Fast Track, and Rare Pediatric Disease Designations

ODD FT RPD OMPBTD

Copper is Required in Human Development and Health

Source: de Bie, et al, 2007

Connective tissue formation

Lysyl

oxidase

Pigment formation

Tyrosinase

Catecholamine production

Dopamine



-hydroxylase

Mitochondrial respiration

Cytochrome C oxidase

Iron and copper transport

Ceruloplasmin

Peptide

amidation

Peptidylglycine



-amidating monooxygenase

Antioxidant defense

Superoxide dismutase

Brain Development

Biological Functions Copper Containing Proteins

Menkes Disease is a Rare Pediatric Disease Causing a Disorder of Copper

Metabolism

Menkes Disease

▪ First described by Dr. John Menkes in 1962

▪ X-linked recessive disease: affecting mostly boys

▪ Minimum birth prevalence for Menkes disease believed to be 1 in 34,810 live male

births, but could potentially be as high as 1 in 8,664 live male births, higher than

previously recognized

▪ Disorder of copper metabolism caused by mutations in the Copper transporter ATP7A

▪ If untreated, premature death in under 2 years

Distinctive clinical phenotypes

▪ Sparse, depigmented hair (“kinky hair”)

▪ Onset of neurologic symptoms: seizures, hypotonia, and developmental delays

▪ Failure to thrive

▪ Connective tissue problems

Diagnosis

▪ Low serum copper and ceruloplasmin levels

▪ Abnormal catecholamine levels

▪ ATP7A gene sequencing confirmation

Image Source: Stephen G. Kaler, MD; NCBI

Menkes Disease is Under-estimated and Under-diagnosed

New study estimated birth prevalence of Menkes disease based on the Genome Aggregation Database

▪ Accessed Genome Aggregation Database (gnomAD) at MIT/Broad Institute → over 200,000 ATP7A alleles

▪ Identified 1,106 ATP7A variants

▪ 4 Loss-of-Function (LOF) variants → 4 alleles → 1:34,810 live male births → 56 patients per year

▪ 28 potentially pathogenic missense variants (PolyPhen-2) → 12 alleles with high confidence (REVEL >0.85)

▪ Including both LOF and pathogenic missense variants → 1:8,664 live male births → 225 patients per year

▪ Newborn screening (NBS) will likely increase the number of Menkes disease patients identified for early diagnosis

and treatment with CUTX-101

References: [1] Kaler, et al, 2020; [2] Kaler, SG, 1998; [3] Danks DM, 1971; [4] Tonnesen et al 1991

100

110

120

130

140

150

160

170

180

190

200

210

220

230

240

250

953813

225

~1:300,000

Live births [4]

~1:100,000

Live births [2]

1:34,810

Live Male births [1]

~1:40,000

Live births [3]

1:8,664

Live Male births [1]

ATP7A LOF

Variants only

ATP7A LOF + Pathogenic

Missense Variants

Genome Aggregation

Database (gnomAD)

(Kaler et al, 2020)

Old

Literature

Number of newborns

with Menkes

disease per year

Adapted from: Bandmann et al, Lancet Neuro 2015

Copper Transport is Impaired in Menkes Disease

Menkes

Disease

Impaired GI

absorption

via ATP7A

Impaired Cu

transport to the

brain via ATP7A

Adapted from: Bandmann et al, Lancet Neuro 2015

Therapeutic Strategy for Menkes Disease:

CUTX-101 (Copper Histidinate)

Menkes

Disease

CUTX-101 Copper Histidinate (CuHis)

SC injection to replenish CuHis

▪ Bypass GI absorption of Cu

(impaired in

Menkes patients)

▪ Better tolerability (pH 7.4)

▪ May not be sufficient alone in some Menkes

patients

NDA accepted and granted priority review

by the FDA; target PDUFA action date of

September 30, 2025

 Positive topline clinical efficacy data

Expanded Access protocol ongoing

Impaired GI

absorption

via ATP7A

Impaired Cu

transport to the

brain via ATP7A

Therapeutic Strategy for Menkes Disease:

CUTX-101 (Copper Histidinate) + AAV-ATP7A Gene Therapy

Menkes

Disease

CUTX-101 Copper Histidinate

SC injection to replenish CuHis

▪ Bypass GI absorption of Cu

(impaired in

Menkes patients)

▪ Better tolerability (pH 7.4)

▪ May not be sufficient alone in some Menkes

patients

AAV-ATP7A Gene Therapy

▪ Codon-optimized reduced-sized ATP7A to be

delivered via AAV vector

▪ May restore Cu

transport

▪ Co-administration with CUTX-101 injections

Impaired GI

absorption

via ATP7A

Restored Cu

transport to the

brain via ATP7A

gene therapy

Preclinical

Adapted from: Bandmann et al, Lancet Neuro 2015

NDA accepted and granted priority review

by the FDA; target PDUFA action date of

September 30, 2025

 Positive topline clinical efficacy data

Expanded Access protocol ongoing

Compelling Top-Line Clinical Efficacy Data for CUTX-101

Early-treatment cohort: initiated treatment with CUTX-101 within 4 weeks of age

Data presented as a virtual poster at the 2021 American Academy of Pediatrics (AAP) National Conference & Exhibition:

Kaler SG, et al, Copper Histidinate Treatment for Menkes Disease (Kinky Hair Syndrome)

▪ CUTX-101 showed significant clinical benefit in both CuHis-ET and CuHis-LT cohorts, with 75-79% reduction in

risk of death compared to untreated Historical Control (HC-ET and HC-LT) arms, and increase in Median OS from

1.3 years to 14.8 years in the ET cohort

Early-Treatment (ET) Cohort Late-Treatment (LT) Cohort

CuHis-ET

(n=31)

Historical Control

(HC-ET) (n=18)

CuHis-LT

(n=35)

Historical Control

(HC-LT) (n=17)

Median Overall Survival

14.8 years

(177.1 months)

1.3 years

(15.9 months)

5.2 years

(62.4 months)

1.5 years

(17.6 months)

Hazard Ratio (95% CI)

0.208

(0.094, 0.463)

0.253

(0.119, 0.537)

-value <0.0001 <0.0001

Reduction in Risk of Death

79% 75%

Kaplan-Meier Overall Survival Curves for Early Treatment Cohorts

▪ Data presented as a virtual poster at the 2021 American Academy of Pediatrics (AAP) National Conference & Exhibition:

Kaler SG, et al, Copper Histidinate Treatment for Menkes Disease (Kinky Hair Syndrome)

Thank you!

Investor Contacts:

Cyprium Therapeutics, Inc.

Jaclyn Jaffe, Investor Relations

ir@cypriumtx.com