Catabasis Pharmaceuticals - Our mission is to bring hope and life-changing therapies to patients and families affected by rare diseases ...
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Our mission is to bring hope and
life-changing therapies to patients and
families affected by rare diseases
Catabasis Pharmaceuticals
October 2020
Forward Looking Statements
Any statements in the slides for our presentation, or accompanying oral presentations, about future expectations, plans and prospects
for the Company, including statements about: ongoing clinical trials and potential regulatory activities and timelines for edasalonexent
including, among other things, statements about the Company’s global Phase 3 PolarisDMD trial, including the anticipated timing for top-
line results, the data that is expected to be included in the topline results, the potential therapeutic benefits of edasalonexent and the
rationale therefore, the potential safety profile of edasalonexent, the clinical significance and acceptability and validity of the primary
endpoint of the trial, and the potential timing for the filing of an NDA, FDA approval and commercial launch; the status and plans of the
GalaxyDMD trial, the planned trial for edasalonexent in non-ambulatory patients; the potential for studying edasalonexent for additional
indications and the timing of planned clinical trials therefore; the potential commercial opportunity for edasalonexent, including its
potential as a foundational, life long therapy for all DMD patients, where it potentially fits within the DMD treatment paradigm, its
potential to be used as a monotherapy or in combination with other treatments, the potential value of edasalonexent to payers, KOLs
and patients, the estimated prevalence of DMD, the estimated total DMD market opportunity and the potential U.S. launch; and
Company’s potential growth, along with other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “may” and
similar expressions, constitute forward-looking statements within the meaning of applicable securities regulations and laws.
Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors,
including risks and uncertainties: inherent in the initiation, completion and results of clinical trials and clinical development; related to
whether the results of earlier stage clinical trials will be predictive of the results of later stage clinical trials; related to the regulatory
review and approval process; related to the impact of the COVID-19 pandemic and the effectiveness of the steps we have implemented
to address the pandemic, including the use of telehealth visits; inherent in the commercialization and market potential of marketed
products; related to successfully managing the Company’s potential transformation into a fully integrated company; related to
competitive products, including those already approved and those in development; inherent in transitioning from a clinical to
commercial supply chain, including the ability to enter into long-term agreements with key contract manufacturers, overseeing such
manufacturers, and managing inventory, particularly where the Company expects to use sole source manufacturers for the foreseeable
future; related to the availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital
expenditure requirements; related to other matters that could affect the clinical development, regulatory status, availability or
commercial potential of edasalonexent; and related to general market and economic conditions, as well as the risks and uncertainties
discussed in the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the period ended June 30, 2020, which is on
file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange
Commission in the future. In addition, the forward-looking statements included in this press release represent the Company’s views as of
the date of this press release. The Company anticipates that subsequent events and developments will cause the Company’s views to
change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company
specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the
Company’s views as of any date subsequent to the date of this release.
2
Catabasis and Edasalonexent: A Compelling Opportunity in DMD
Potential New ‣ Promising disease-modifying oral NF-κB inhibitor
Foundational ‣ Slowed disease progression and preserved muscle function compared to off-treatment
control period in Phase 2 MoveDMD trial and open-label extension
Therapy in
‣ Fast Track, Rare Pediatric, and Orphan Drug designations from FDA
Duchenne ‣ Orphan Medicinal Product designation from European Commission
Muscular ‣ Pivotal Phase 3 PolarisDMD trial fully enrolled, top-line results expected in Q4 2020
Dystrophy (DMD) ‣ NDA filing expected in 2021
‣ Potential differentiated foundational treatment for all DMD patients
Significant ‣ High unmet medical need in clear target market with strong patient advocacy and
concentrated Centers of Excellence
Commercial ‣ Unique mechanism enables development as mono- or combination therapy with other
Opportunity treatments such as exon skipping, gene therapies and other approaches
‣ Market research indicates high likelihood of physician adoption and payer coverage
Expansion in DMD ‣ Additional trial planned in non-ambulatory DMD patients
and Beyond ‣ Potential to leverage benefits of inhibiting NF-κB in other indications
‣ Accomplished industry, financial and clinical leaders
Leadership Depth
‣ Seasoned team with experience in rare diseases and commercialization
and Focus ‣ Strong IP position and wholly-owned assets
3
Edasalonexent Global Market Opportunity in DMD
Rare Orphan Disease with Significant Unmet Medical Need
Patient Impact
Prevalence Diagnosis Unmet Need
1 in ~ 4 years old MA JORITY
3,500-5,000 Progressive loss of of those who
Males1 muscle function1 discontinue steroids
do so because of side
effects2
Market Impact
Prognosis Treatment Options Global Market Value
~ 30 years No Cure >$4.0 Billion
Median survival: Limited options Global market for
Death from heart for all DMD DMD drug
and lung failure1 patients treatments by 20233
1 https://www.parentprojectmd.org/about-duchenne/is-it-duchenne/signs-and-symptoms/
2 Cowen, 2019
3 www.grandviewresearch.com/press-release/global-duchenne-muscular-dystrophy-dmd-drugs-market 4
Edasalonexent: Potential for Broad Therapeutic
Benefit
Activated NF-κB Potential
leads to disease for edasalonexent, an
progression in DMD NF-κB inhibitor
Skeletal Muscle
Loss of ambulation, Goal: Improve
upper limb function, skeletal muscle function
respiratory failure
Heart
Goal: Preserve
Cardiomyopathy cardiac function
Bone
Goal: Reduce
Fractures risk of fractures
In DMD, the loss of dystrophin leads to chronic activation of NF-κB, which
is a key driver of skeletal muscle and cardiac disease progression
5
Edasalonexent: Potential to Slow Disease
Progression for All Those Affected by DMD
‣ Our Vision for Edasalonexent
– Foundational therapy for all DMD patients,
regardless of underlying mutation, from time
of diagnosis onwards
– Address skeletal and cardiac muscle disease
and bone health
– Unique mechanism enables development as
mono- or combination therapy with other
treatments such as exon skipping, gene
therapies and other approaches
– Favorably differentiated safety and tolerability Developing
profile from other treatments a potential
foundational
therapy
‣ Commercial Approach in DMD
– Disease-focused specialty sales force in US
– Establish global “go-to-market” strategies
Edasalonexent is an investigational agent not currently approved in any territory
6
Fully Enrolled Edasalonexent Phase 3 PolarisDMD
Trial Designed for Global Registration
12-month, randomized, double-blind
Open-label extension
placebo-controlled trial (n=131)
Edasalonexent 100 mg/kg
Edasalonexent
Placebo
Primary Endpoint
‣ Eligibility:
– All mutations
– Age 4 to 7 (up to 8th birthday); off steroids for ≥6 months
‣ Endpoints: Consistent with regulatory guidance
– Primary: Change in North Star Ambulatory Assessment
– Key secondary: Age-appropriate timed function tests
– Additional assessments include growth, cardiac and bone measures
‣ Top-line results expected in Q4 2020
7
Phase 3 PolarisDMD Clinical Trial Fully Enrolled
‣ Enrolled 131 patients in 8 countries
8
Phase 3 PolarisDMD Trial Incorporates Critical Aspects
of Daily Function and Differentiating Assessments
Physical Function Outcomes Additional Outcomes
Primary Endpoint: North Star Ambulatory 3 Timed
Assessment Function Tests
Assessment measures — from most to least difficult Growth
Hop right leg Climb box step right
Hop left leg Climb box step left
Time to
Stand on heels Stand on one leg right Stand Cardiac
Rise from floor Stand on one leg left Health
Run Get to sitting
Jump Rise from chair
Lift head Walk
4-Stair
Bone
Descend box step right Stand Climb Health
Descend box step left
How measures are scored:
Patient
2 Can perform 1 Can perform 0 Unable to Reported
with difficulty perform 10-Meter Outcomes
Walk/Run
9
Overview of the North Star Ambulatory Assessment
(NSAA)
NSAA is a validated scale specifically developed to measure physical
performance in ambulatory boys with Duchenne
Holistic Reproducible
• Measures 17 domains of function:
NSAA • Studies show NSAA is reliable for
Hop on leg* Climb up* Get to sitting measuring change over time
Stand on heels Climb down* Rise from chair
Rise from floor Stand on leg* Walk • In edasalonexent trials, NSAA
Run Hop on leg* Stand demonstrated reproducibility
Jump
Patient-centric Widely Accepted
• Selected as a meaningful • Catabasis was an early adopter of
endpoint with patients and NSAA use in clinical trials
caregivers in mind • Many other active clinical trials in
DMD now also use NSAA as
endpoint
Recommended
• Recommended as clinical trial
endpoint by FDA & EMA
• Recommended for clinical use in
DMD practice guidelines
*These are tested individually on the left and right leg
10
Mazzone 2009, Mayhew 2011, FDA 2018, EMA 2016, Birnkrant 2018, MDA 2020 e-posterPhase 3 PolarisDMD Trial Was Designed Based on
Promising MoveDMD Trial Results
In Phase 2 MoveDMD ® Trial and Open-Label Extension:
NF-κB Target
Engagement Biomarkers Muscle MRI Functional
▸ Inhibited ▸ Decreased CK ▸ Improved rate of ▸ Preserved NSAA
NF-κB targeted and other change in MRI T2 and Timed
gene set in muscle enzymes compared to off- Function Tests
peripheral blood ▸ Decreased CRP, treatment vs. off-treatment
biomarker of control control period
inflammation
11Edasalonexent Demonstrated Clinically Meaningful
Slowing of Disease Progression
In Phase 2 MoveDMD Trial and Open-Label Extension:
North Star Ambulatory Assessment 4-Stair Climb
25 Edasalonexent
0.4 Edasalonexent
100 mg/kg 100 mg/kg
20
0.3
Speed (1/Seconds)
Time (Seconds)
NSAA Score
15
0.2 5
10 Better
0.1 10
5 Control Control
15
Period Period
0
-36 -24 -12 0 12 24 36 48 60 72 -36 -24 -12 0 12 24 36 48 60 72
Weeks Weeks
10-Meter Walk/Run Time to Stand
0.20 Edasalonexent 5 0.3
100 mg/kg Edasalonexent
0.18 100 mg/kg
Speed (1/Seconds)
Speed (1/Seconds)
Time (Seconds)
Time (Seconds)
0.2 5
0.16
0.14
0.1 10
0.12 Control Control
15
Period Period
10 0
-36 -24 -12 0 12 24 36 48 60 72 -36 -24 -12 0 12 24 36 48 60 72
Weeks Weeks
Means ± SEM shown. Includes data of all boys initially started on 100 mg/kg dose (n=16) with 11 boys participating through 72 weeks. 12
Results are compared to the off-treatment control period changes measured prior to boys in the MoveDMD trial receiving 100 mg/kg edasalonexent.Muscle Enzymes Significantly Decreased on Edasalonexent,
Supporting a Positive Impact on Muscle Health
In Phase 2 MoveDMD Trial and Open-Label Extension:
Muscle Enzymes: Change from Baseline
Creatine Kinase Aspartate Aminotransferase
0 0
-5000 -50
-10000 -100
IU/mL
IU/mL
-15000 * -150 *
-20000 -200
-25000 -250
12 24 36 48 60 72 12 24 36 48 60 72
Alanine Aminotransferase Lactate Dehydrogenase
0 0
-50 -200
IU/mL
IU/mL
-100 -400
-150 -600
* *
-200 -800
12 24 36 48 60 72 12 24 36 48 60 72
Weeks on 100 mg/kg Edasalonexent
Plasma muscle enzymes are elevated 10 to 100 fold in DMD, indicative of leakage from damaged myocytes
13
Means ± SEM shown; * pEdasalonexent Significantly Improved Rate of Change
of MRI T2 Compared to Off-Treatment Control Period
In Phase 2 MoveDMD Trial and Open-Label Extension:
MRI T2: Composite of 5 Lower Leg Muscles
6
Edasalonexent
100 mg/kg
Annualized Rate of Change (m sec/year)
4
Better
2
* *
* *
*
0
Off-treatment 12 24 36 48 72
control
Weeks on Edasalonexent
-4
‣ MRI T2 increases over time in DMD as inflammation and fat content of muscle increases
‣ A composite MRI T2 measure of five lower leg muscles correlated well with current ability
to perform time function tests in the ImagingDMD natural history database
Means + SEM; mixed model comparison with off-treatment period
* Week 12: p=0.002, n=16; Week 24: p=0.004, n=14; Week 36: p=0.032, n=13; Week 48: p=0.018, n=12; Week 72: p=0.052, n=9
14
Willcocks, et al, 2016, Ann Neurol. Presented at WMS 2018.Edasalonexent Showed Potential for Positive
Cardiac Effects in DMD
In Phase 2 MoveDMD Trial and Open-Label Extension:
Heart Rate: Change from Baseline
Baseline
0 99 beats/min
* pEdasalonexent Has Been Well-Tolerated in Clinical
Trials
In Phase 2 MoveDMD Trial and Open-Label Extension:
‣ Well tolerated, with majority of adverse
events mild in nature
– Most common related adverse event was
diarrhea, generally mild and transient
‣ Boys on edasalonexent in our Phase 2
MoveDMD trial and open-label extension
grew similarly to unaffected boys
– Height increased by an average of 2 inches
per year
– Weight increased by an average of 3 pounds
per year
– Both increases are in line with typical height
and weight increases of unaffected boys
Over 150 patient years of exposure to date across all clinical trials
16Phase 3 PolarisDMD and Phase 2 MoveDMD
Trials Have Similar Baseline Characteristics
‣ Analysis shows that Phase 3 trial enrolled the expected patient population
– Comparison of baseline age and function (NSAA, time to stand, 4-stair climb, and 10-
meter walk/run) were similar in both trials; there were no significant differences in
baseline characteristics between the two trials*
‣ Findings support the assumptions on which the Phase 3 trial was powered
PolarisDMD (n=131) MoveDMD (n=23)
Age (years) 5.7 ± 1.0 6.0 ± 1.1
Percent enrolled patients that had not taken steroids 98% 100%
North Star Ambulatory Assessment (NSAA) score 20.8 ± 4.7 20.1 ± 5.5
10-Meter Walk/Run speed (1/s) 0.181 ± 0.037 0.168 ± 0.045
4-Stair Climb speed (1/s) 0.265 ± 0.097 0.254 ± 0.110
Time to Stand speed (1/s) 0.212 ± 0.070 0.193 ± 0.080
Means ± standard deviation shown
*Kolmogorov-Smirnov test used to assess for population distribution differences 17Edasalonexent Commercialization Approach Will be
Focused and Targeted
High unmet need
Well-defined and characterized patient population
Established and focused treatment centers of excellence
Therapeutic option providing value for patients
18
Edasalonexent US market launch is contingent upon FDA approvalUnmet Need: Clear Market Need in DMD with
Limited Treatment Options
‣ Currently, there is no cure for DMD
‣ Today, the majority of DMD patients are treated with corticosteroids
– Despite broad market utilization, steroids have long-term negative consequences
‣ Only a small portion of the DMD population can be treated with eteplirsen, golodirsen or
viltolarsen (US) or ataluren (EU)
Current Landscape of DMD Medical Management
Steroids Mutation Targeted
Deflazacort and Eteplirsen, Golodirsen, and Viltolarsen (US);
Prednisone1 Ataluren (EU)
Known Benefits: Known Side Effects: ‣ Approvals require additional studies
‣ Delayed loss of ‣ Osteoporosis ‣ Limited suitable patient populations2,3
muscle function with fractures ‣ ~13% for mutation 51
‣ Metabolic effects ‣ ~9% for mutation 53
‣ Weight gain, obesity
‣ Growth retardation
‣ Delayed puberty
‣ Cataracts
‣ Muscle atrophy
‣ Behavioral issues
‣ Cushingoid appearance
1 Deflazacort and prednisone package inserts
2 Aartsma-Rus A, et al. Theoretic applicability of anitsense-mediated exon skipping for Duchenne muscular dystrophy mutations. Human
Mutation. 2009;X:1-7.
3 Fletcher, S., et. al. Dystrophin Isoform Induction In Vivo by Antisense-mediated Alternative Splicing. The American Society of Gene & Cell 19
Therapy. 2010;18(6):1218-1223.Defined Population: DMD Patient Prevalence Is Well
Defined
~15,000 ~19,000 ~35,000
Males* in the US1 Males* in the EU2 Males* in other countries with
access to rare disease therapeutics
(Ex-US & Ex-EU)3
Canada UK Europe Sweden
US
Israel
Globally, Advocacy Organizations Drive Australia
Awareness and Education for DMD Patients
and Families
‣ Collaboration in research and education
‣ Partners for clinical trials and market access
Affects 1 in 3,500-5,000 Males* Worldwide
1 Landfeldt
20
Eur J of Epi,2020, Catabasis internal market research; 2 Mah. Neuropsych Dis Treat. 2016 12:1795-1807 ; 3 Bionest Partners, qualitative market research June 2020
* Some females do present with DMD, exact prevalence unknownCenters of Excellence: Most US and EU Duchenne
Patients Have Access to Expert Care and Treatment
Concentrated treatment centers enable targeted field force efforts
– Enables ability to use small group of field-based employees to educate and
increase awareness of the role of NF-κB and edasalonexentTherapeutic Option/Value: Edasalonexent Potential
Profile for the Duchenne Community
Meaningful Clinical Endpoints Assess Efficacy
PolarisDMD Phase 3 trial has the potential to demonstrate change in NSAA,
a functional outcome measure
Well-tolerated to date
Over 150 cumulative years of patient exposure; most common related adverse
event was diarrhea, generally mild and transient
A potential foundational treatment for all Duchenne boys
Edasalonexent aims to be a foundational therapy for all patients affected by
Duchenne, regardless of dystrophin mutation
Potential use as mono- or potential combo-therapy
The Phase 3 PolarisDMD and GalaxyDMD open-label extension trials were
designed to allow concomitant treatment with exon-skipping therapies
Age appropriate development shown in Phase 2 study, similar to peers
Boys on edasalonexent in the Phase 2 MoveDMD trial and open-label
extension grew similarly to unaffected boys
1. Pitchforth, et al. Neuromusc. Disorders 2018 28 (S17) doi:10.1016/S0960-8966(18)30340-7 22Therapeutic Option/Value: Global Blinded Market
Research Conducted with Key Stakeholders
Assessment of the proposed edasalonexent target product profile including:
‣ Primary and secondary endpoints, safety, mechanism of action, and dosing
‣ Efficacy for the Phase 3 PolarisDMD trial extrapolated from Phase2 MoveDMD trial
‣ Probability for use and reimbursement
DMD Patients and
Physicians Caregivers Payers
(n=23 US/30 EU) (n=10 US) (n=18 US/15 EU)
Overall, KOLs view a “ I think the benefits are Positive payer reactions to
blinded edasalonexent slowing the progression a new DMD product with
profile as a moderate to and the fact that it may efficacy demonstrated by
high therapeutic help the child stand and NSAA outcome but
improvement for patients walk longer and then it without the long-term side
with DMD and believe its is exciting it can be effects associated with
steroid sparing safety safely used in steroids
effect is its primary value combination with other
driver treatments.”
23
Sources: Blueprint Partnership Market Research 2019, Two Labs 2019 Market Research with physicians and payers in the USTherapeutic Option/Value: Potential Edasalonexent Value
Profile Supports Pricing Within the Spectrum of Other
Rare Disease Modifying Therapies
Steroid used in Other approved
DMD treatments in DMD
DMD
$121K1 $958K1 $998K1
CF $291K2
$272K $332K2
$100K $340K3 $383K (Post Year 1: Annual Maintenance dosing) $892K3 (Year 1: 7 doses initial year)
SMA
$426K3 $2.13M3
Annual payment for 5 years One-time cost
0 $100,000 $200,000 $300,000 $400,000 $500,000 $600,000 $700,000 $800,000 $900,000 $1,000,000
1 Dosing calculated based on 40kg patient, Redbook Wholesale Acquisition Cost (WAC) pricing accessed August 18, 2020
2 CF Therapies based on PI dosing regimens Kalydeco ($25,603/Package), Orkambi ($20,919.23/Package ), Symdeco
24
($22,400/package); All prices based on Wholesale Acquisition Cost (WAC) August 18, 2020
3 SMA Therapies based on PI dosing regimens Evrysdi ($11,170.43/80ml package), Spinraza ($127,500/5ml package) and Zolgensma ($2.125M)Edasalonexent Is Poised for Potential Commercial Success
in Duchenne
High unmet need
Well-defined and characterized patient population
Established and focused treatment centers of excellence
Therapeutic option providing value for patients
25
Edasalonexent US market launch is contingent upon FDA approvalEdasalonexent: A Potential
Foundational Treatment in DMD
Potential Upcoming
Foundational Milestones
therapy for all
with DMD 2020
regardless of
mutation
‣ Q4: Top-line Phase 3 results
2021
‣ NDA filing
‣ Initiate non-ambulatory DMD trial
Edasalonexent 2022
‣ Launch edasalonexent in U.S. for DMD
Oral NF-kB Potential for ‣ Initiate trial for edasalonexent in a
inhibitor positive effects second indication
on skeletal
muscle, cardiac
and bone health
26Catabasis Goal: Maximize the Potential Impact of
Edasalonexent with Muscular Dystrophy Patients
For use in other
dystrophies
Opportunity
Duchenne:
Standard
of Care
Duchenne:
Use in combination
with other
Duchenne therapies
Edasalonexent
monotherapy
Time and Evidence Generation
27Catabasis Is Striving to Improve
the Lives of Patients Affected by DMD
NF-κB • Chronic activation of NF-κB is a well-recognized driver of disease progression in DMD
Targeted • Edasalonexent inhibits NF-κB and has a novel mechanism among the therapies available or in
development for DMD with broad potential benefits
MOA
• Edasalonexent slowed disease progression with a favorable safety profile in MoveDMD trial
• Being developed as a potential treatment for all patients affected by DMD regardless of the
underlying mutation, from time of diagnosis onward
Potential
• We are developing edasalonexent as a monotherapy and for use with other therapies such as exon
Foundational skipping
Therapy • We believe that based on its mechanism of action, edasalonexent has the potential for use with
other approaches in development such as gene therapy
Favorable • Strong interest from physicians and KOLs
Market • Market research indicates high likelihood of physician adoption and payer coverage
Profile • Potential to meet the needs and desires of the DMD community
Relationship • Developing best-in-class internal capabilities and forming critical partnerships to execute a clinical
trial and prepare for potential NDA filing and subsequent launch
Focus
Market • Hired Chief Commercial Officer in September 2019
Preparation • Commercialization planning underway
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