Accelerating Medicines to Transform Patients' Lives
Corporate Overview
March 20, 2024
Forward Looking Statements & Disclaimer
This presentation contains statements that are not of historical facts, considered 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. Forward-looking statements include, but are not limited to, statements about our expectations regarding the potential benefits, effectiveness, and safety of our product candidates; our expectations with regard to our research, development and regulatory plans, including the design, timing and results of preclinical studies and clinical trials, the timing and availability of data from such studies and trials, and the timing or likelihood of regulatory filings and approvals for our product candidates; our expectations with regard to our ability to license, acquire, discover, and develop additional product candidates and advance such product candidates into, and successfully complete, preclinical studies and clinical trials; the potential market size and size of the potential patient populations for our product candidates and any future product candidates and those indications we target; our expectations about our ability to maintain existing, and establish new, strategic collaborations, licensing, or other arrangements; the scope of protection we are able to establish and maintain for intellectual property ("IP") rights covering our product candidates and any future product candidates; our business strategy; and our future results of operations and financial position.
Such statements reflect the current views of ACELYRIN with respect to future events, and are subject to known and unknown risks (including, without limitation, business, regulatory, economic and competitive risks), uncertainties, assumptions and contingencies about ACELYRIN (including, without limitation, those associated with our successful completion of development and regulatory activities for our product candidates, maintaining and defending IP protection, ability to timely secure adequate supply of our product candidates, legal proceedings, government investigations, macroeconomic conditions, market volatility) and other risks and uncertainties described under the heading "Risk Factors" in the Company's most recent Quarterly Report on Form 10-Q for the three months ended September 30, 2023 filed with the U.S. Securities and Exchange Commission ("SEC") and in subsequent filings made by us with the SEC, which are available on the SEC's website at www.sec.gov.
In light of these risks and uncertainties, many of which are beyond our control, the plans, intentions, expectations or other events described in or implied by our forward-looking statements may not occur. Actual results or events could differ materially and adversely from those expressed in any forward-looking statements. New risks may occur at any time, and we anticipate that subsequent events and developments could cause our views to change. Any reader of this presentation is cautioned not to place undue reliance on these forward-looking statements, which speak to our current beliefs and expectations only as of the date of this presentation. Except as required by law, we disclaim any intention or responsibility for the accuracy and completeness of the forward-looking statements in this presentation, or to update
or revise any forward-looking statements in the event of new information, future developments or otherwise.
No representation is made as to the safety or effectiveness of our product candidates. Additionally, this presentation also contains certain information related to or based on studies, publications, surveys and other data obtained from third-party sources, and our own internal estimates and research, including without limitation relating to market size and growth. While we believe these third-party sources to be reliable as of the date of this presentation, we have not independently verified, and make no representation as to the adequacy, fairness, accuracy or completeness of any third-party information. In addition, market data involves a number
of projections, assumptions, and estimates of our future performance and the future performance of the markets in which we operate, and there can be no guarantee as to the accuracy or reliability thereof as they are necessarily subject to a high degree of uncertainty and risk.
The information in this presentation is as of the date of this presentation, and is subject to change without notice.
TRADEMARKS
This presentation contains trademarks, service marks, trade names and copyrights of ACELYRIN and other companies which are the property of their respective owners.
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"ACELYRIN Is A Leading Clinical-Stage Biopharma Company
Focused On Identifying, Acquiring, And Accelerating
The Development And Commercialization Of
Transformative Medicines In Immunology"
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Creating An Industry Leading Immunology Company
Team of veteran biopharma executives who together bring exceptional track records of developing some of the most successful medicines within immunology and beyond
Building a portfolio of potential new medicines that we think have the opportunity to provide clinically meaningfully differentiated benefit to patients
- We seek "diamonds in the rough" where, based on molecule characteristics, our collective experience and expertise, and the evolving scientific and medical understanding, we can test hypotheses around clinical differentiation for patients
Robust pipeline of clinical programs across several indications representing multi-billion-dollar opportunities in the aggregate
- Izokibep is a "pipeline-in-a-program" in late-stage development for multiple immunological indications including psoriatic arthritis (PsA), hidradenitis suppurativa (HS), axial spondyloarthritis (AxSpA) and uveitis
- Lonigutamab has demonstrated proof-of-concept as a subcutaneously delivered therapy for thyroid eye disease (TED) with the goal to improve upon efficacy, safety as well as convenience for patients
- SLRN-517is an early program targeting mast cell-driven diseases
Well-capitalized having secured more than $1 billion in private and public capital since founding in 2020
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Experienced Leadership Team
Successful Track Record of Delivering Some Of The Most Transformative Medicines For Patients
Shao-Lee Lin | MD, PhD | Melanie Gloria | Mina Kim |
Founder and CEO | COO | CL&AO |
Leaders In Immunology
Gil Labrucherie | Agnes Lee | Ken Lock |
CFO | SVP, IR & Communications | CCO |
Shep Mpofu| MD, MRCP, FRCP | Ron Oyston | Patricia Turney |
SVP, Development | CPO | CTO |
Board of Directors
Shao-Lee Lin | Bruce C. Cozadd | Dan Becker | Alan Colowick | Henry Gosebruch | Patrick Machado | Beth Seidenberg | Dawn Svoronos |
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Executing With A Sense Of Urgency For Patients
Q1 2024
October 2020
Series A Completed
July 2020
ACELYRIN Founded
December 2021
Positive Phase 2 Top-line Data For izokibep in PsA
October 2021
$250 Million
Series B
August 2021
First Program, izokibep, Licensed
November 2022
Add'l Phase 2 RCT PsA
Endpoint & QoL Data
Presented at ACR
September 2022
$300 Million Committed In
Series C; Accelerated PsA
Phase 2b/3 Into 2022
June 2022
Phase 2 RCT PsA Data Presented At EULAR
September 2023
Announced HS Part B Data
May 2023
$621 Million IPO,
April 2023
Announced 46-Week
Phase 2 PsA Data
March 2023
Positive Phase 2b/3 Part A Data For izokibep In Hidradenitis Suppurativa
January 2023
Expanded Portfolio With An
All-stock Company
Acquisition, Adding
Lonigutamab And SLRN-517
2023
Izokibep PsA Phase 2b/3 Data
Izokibep HS 32-week Data
Lonigutamab Phase 1/2 Data
2024
2020
20212022
RCT: Randomized Controlled Trial; QoL: Quality of Life | 6 |
Total Addressable Markets Are Significant And Growing
Pursuing multiple indications with significant unmet need for izokibep and announced positive Phase 2b/3 topline data in PsA and long-term data in HS in 1Q 2024
Proof-of-concept for lonigutamab in Thyroid Eye Disease achieved in 1Q 2024
Strong financial position of $788 million in cash on September 30, 2023 expected to fund operations through key value- driving milestones across our portfolio.
Mast Cell Disease | |||||
e.g., Urticaria | |||||
TED | $5.8B+ | ||||
Uveitis | $4.8B | Market by | |||
2030 | |||||
$790M | Market | ~$39B | |||
AxSpA | by 2030 | ||||
Market | |||||
HS | $6.8B | by 2030 | |||
PsA | $2.9B | Market | |||
by 2030 | |||||
$17.8B | Market | ||||
by 2030 | |||||
Market | |||||
by 2030 |
~$22B
2023 | 2030 |
1 GlobalData Epidemiology Estimates; Helmick C. 2014; Perros P, 2017; Chin Y, 2020; Bartley GB, 1994 See Note 2 on Slide 15 regarding the Phase 2b/3 trial in PsA.
2 Active PsA Market - Emergen Research, 2022; Global AxSpA Market - iHealthcare Analyst, Nov 2022; HS Market Outlook - DelveInsight, Jan 2023; HS | 7 |
Opportunity Analysis - GlobalData 2019; CSU Market - DelveInsight, Dec 2022, Uveitis Global Drug Forecast - GlobalData, June 2021; Tepezza |
Consensus Sales Forecast compiled by GlobalData
Robust Portfolio Of Clinical Programs
With Multiple Indications In Late Stage
Preclinical | Phase 1 | Phase 2 | Phase 3 |
Hidradenitis Suppurativa | ||
Izokibep | Psoriatic Arthritis2 | |
(IL-17Ai)3 | ||
Axial Spondyloarthritis4 | ||
Uveitis1 | ||
Lonigutamab | Thyroid Eye Disease | |
(anti-IGF-1R)5 | ||
SLRN-517 | Mast Cell Diseases | Mast Cell |
(anti-c-KIT)6 | 7 | Diseases |
e.g., Urticaria |
- Phase 2b/3 trial in uveitis. Planned inclusion into registrational package for non-infectious uveitis (as applicable) if granted orphan drug designation and following consultation with relevant health authorities. We have not previously completed any clinical trials for uveitis and are currently conducting our first Phase 2b/3 trial.
- Phase 2b/3 trial in PsA.
- IL-17AInhibitor; Excludes (i) development, commercialization and manufacturing rights in mainland China, Hong Kong, Macau, South Korea and Taiwan, and (ii) development rights in certain other Asia Pacific countries including, without limitation, Australia, India, New Zealand and Singapore. We retain decision making authority for izokibep global development. Potential opportunity to extend certain IP protection into early 2040's.
- Based on data from our Phase 2 and ongoing Phase 2b/3 trials in PsA, we intend to discuss with the FDA initiation of the Phase 3 program in AxSpA without completing earlier clinical trials in AxSpA. The FDA may require us to complete a Phase 2 trial in AxSpA prior to initiating our planned Phase 3 program.
- Worldwide rights to non-oncology indications. Potential opportunity to extend certain IP protection into 2043.
- Potential opportunity to extend certain IP protection to 2039.
- Based on preclinical studies demonstrating highly potent inhibition of the c-KIT pathway targeting mast cell proliferation and degranulation across mast-cell driven diseases such as Chronic Urticaria, an inflammatory disease that is driven by the release of histamine and other vasoactive molecules produced by mast cells
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Izokibep's High Potency & Small Size Enables Potential To Improve Clinical Response With SC Exposures Others Require IV To Achieve
Validated Target
IL-17A is associated with autoimmune inflammation. Marketed monoclonal antibodies have demonstrated targeting IL-17A results in dose-responsive increases in efficacy without dose-limiting toxicity.
Targeting more broadly than IL-17A as a means to more effectively inhibit the IL-17 axis has demonstrated risk for increased fungal infection, suicidal ideation & behavior, and liver toxicity with a requirement for routine monitoring - all raising the potential of association specifically with inhibition of IL-17F.
Hitting IL-17A the hardest may be the sweet spot of achieving increased exposure/efficacy without introducing additional or new safety liability. The high potency and small size of izokibep has the potential to impact clinical response.
High Potency
Blocks the homodimeric IL-17A target protein by binding to both sub-units simultaneously with the high affinity (KD: 0.3 pM) versus other IL-17A inhibitors.
Small Size
~1/10th the size of a mAb (~18.6 kD) enabling potential to reach difficult to treat tissues.
IL-17A homodimer
IL-17A binding domain
IL-17A binding domain
Albumin binding domain
Extends half-life to 12 days
IZOKIBEP (~18.6 kDa)
pM, picomolar; kD, kilodalton | 9 |
PsA
PsA Is A Disease With Multiple Manifestations
Addressing Totality Of Manifestations Is Necessary To Achieve Disease Control & Restore Quality of Life
Arthritis | Psoriasis |
Enthesitis | Dactylitis | Spondylitis |
Psoriatic arthritis (PsA) is a chronic, inflammatory disease with multiple clinical manifestations including
arthritis, psoriasis, enthesitis (inflammation of dense, non- vascular tissues that connect ligaments and tendons to bone), spondylitis, and dactylitis
~1.6M PsA patients in the U.S.
Among moderate-to-severe PsA patients, over a third fail non-biologictherapy
More complete and faster resolution of disease symptoms manifesting in dense tissues (e.g., enthesitis) remain an unmet need
Addressing totality of manifestations is the goal
for patients
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Acelyrin Inc. published this content on 20 March 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 20 March 2024 11:27:05 UTC.