Seelos Therapeutics, Inc. · OTC:SEELQ
Seelos Therapeutics — Investor Presentation (March 2023)
41 slides from Seelos Therapeutics's investor presentation, Mar 10, 2023
Slide 1: Seelos Therapeutics March 2023
SEELOS THERAPEUTICS March 2023 “We are a company focused on achieving the most efficient development of products that address significant unmet needs in CNS disorders and in rare diseases” 1 Exhibit 99.1
Slide 2: Forward-Looking Statements
FORWARD - LOOKING STATEMENTS This corporate presentation includes certain forward - looking statements within the meaning of Section 21E of the Securities Exch ange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These forward - looking statements include, but are
Slide 3: Experienced Management Team
EXPERIENCED MANAGEMENT TEAM Raj Mehra, PhD Chairman, Founder, and Chief Executive Officer 23 years experience as VC/investor Michael Golembiewski Chief Financial Officer 28 years experience in public company accounting and finance Tim Whitaker, MD Chief Medical Officer 26 years experience with 19 ND
Slide 4: Seelos is a CNS and rare disease focused biotech company
Advancing multiple late - stage therapeutic candidates for CNS disorders Developing therapeutics with demonstrated mechanisms of action Identifying opportunities in large markets SEELOS IS A CNS AND RARE DISEASE FOCUSED BIOTECH COMPANY SLS - 004 Parkinson’s Disease (PD) Gene therapy program targetin
Slide 5: SLS-002 (Intranasal Racemic Ketamine)
SLS - 002 (INTRANASAL RACEMIC KETAMINE) 5
Slide 6: History of ketamine development and use
Initially developed as an animal sedative, ketamine injections were first tested in humans in 1964 as an anesthetic and the first paper on human use was published in 1966 1 Use and abuse of ketamine began to increase, especially among certain sub - cultures (e.g., mind explorers and New Age spiritua
Slide 7: SLS-002 for Acute Suicidal Ideation and Behavior (ASIB)
SLS - 002 (INTRANASAL RACEMIC KETAMINE) FOR ACUTE SUICIDAL IDEATION AND BEHAVIOR (ASIB) Seelos Therapeutics owns the rights to Javelin’s post - operative pain program 7
Slide 8: Suicidal Ideation and Behaviors Among U.S. Adults (2020)
SUICIDAL IDEATION AND BEHAVIORS AMONG U.S. ADULTS (2020) 12.2 million adults had serious thoughts of committing suicide 2 1.2 million adults attempted suicide 2 3.2 million adults made suicide plans 2 920,000 adults made plans and attempted suicide 2 283,000 adults made no plans and attempted suicid
Slide 9: Overview of PK/PD and Safety in Phase I for SLS-002
• All doses administered to date have demonstrated to be well - tolerated with no new or unique safety signals • No serious adverse events (SAEs) and all AEs were transient and clinically manageable • Expected transient increase in Clinician Administered Dissociative State Scale (CADSS) scores at th
Slide 10: Registration Directed Trial Design for SLS-002
REGISTRATION DIRECTED TRIAL DESIGN FOR SLS - 002 Design • Two - part – Open label (n=17) followed by double blind (n~175 (1) ) • Initiated in hospital, followed by outpatient phase once stabilized • Two - week (16 days) treatment period (~7 days inpatient / 9 days outpatient) • Dosing 2x/week for to
Slide 11: Part 1 (Open Label) Results: SLS-002 Clinical Improvements Across All Scales
PART 1 (OPEN LABEL) RESULTS: SLS - 002 SHOWED CLINICAL IMPROVEMENTS ACROSS ALL SCALES BASELINE (n=17) DAY 1 (n=17) DAY 16 (n=14) MADRS (0 - 60 scale, ≤12 is remission) 39.4 14.5 7.4 CGIS - SI/B (1 - 5 scale, 1=not at all, 5=extremely suicidal) 3.8 1.6 1.1 S - STS (0 - 52 scale, higher score equated
Slide 12: Part 1 (Open Label) Results: Detailed Summary of Safety Results
Clinician Administered Dissociative States Scale (CADSS) • Change from baseline (at 40 minutes) = 3.8 after first dose and 1.0 at 1 hour post first dose, 2.6 on Day 4 (after second dose) and 0.3 on Day 8 (after third dose) Hemodynamic Effects Systolic BP • Baseline = 119.8; 1 hour (post first dose)
Slide 13: Results from Esketamine Phase 3 Trials in ASIB/MDD
Change from Baseline Day 1 Day 4 Day 15 Placebo (1) 11(n=111) 13(n=110) 17(N=99) Esketamine (1) 16(n=111) 18(n=109) 20(N=104) (p=0.006) RESULTS FROM ESKETAMINE PHASE 3 TRIALS IN ASIB/MDD (CHANGE IN MADRS FROM BASELINE TO DAY 15)** Reference: (1) J Clin Psychiatry 81:3, May/June 2020 **These data are
Slide 14: Part 2 Interim Blinded/Blended Data: SLS-002 Change in MADRS
Change from Baseline Day 1 Day 5 Day 16 SLS - 002 14.1 20.2 23.9 ( n=117) (n=115) (N=102) PART 2 INTERIM BLINDED / BLENDED DATA: SLS - 002 CHANGE IN MADRS FROM BASELINE TO DAY 16 * Includes 50% placebo subjects 14 Data cut - off date: Feb 16, 2023. The data presented on this slide remain blinded, an
Slide 15: SLS-002 Intranasal Administration Device
SLS - 002 INTRANASAL ADMINISTRATION DEVICE Manufactured by Aptar Pharma (NYSE:ATR) Seelos has Co - Exclusive Use of Bidose (BDS) Liquid System Bi - dose device administers 0.1 mL sprays in each nostril Disposable device for single use Convenient, ready to use administration 15
Slide 16: SLS-002 Has the Potential to Transform ASIB in MDD
~700,000 people die globally due to suicide annually 1 In 2019, Suicide was the 10th leading cause of deaths in the U.S. (45,000 lives per year) 2 There is one death by suicide in the U.S. every 11 minutes 5 Depression currently affects 264 million people globally 3 According to WHO: MDD will become
Slide 17: SLS-005 (Trehalose)
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Slide 18: SLS-005 (Trehalose) for ALS and SCA
Current studies ongoing/planned in ALS and Spinocerebellar Ataxia SLS - 005 (TREHALOSE) FOR ALS AND SCA Dose IN ketamine hydrochloride (50 mg) or saline solution Duration Each treatment period (ketamine or placebo) consisted of 7 days. Assessments occurred at 40 min, 120 min, 240 min, 24 hrs, 48 hrs
Slide 19: Trehalose Mechanism of Action
TREHALOSE MECHANISM OF ACTION Immediate enhancement of autophagy 1 Prolonged enhancement of lysosomal pathway 2 Protein stabilization 1. Mayer AL, Higgins CB, Heitmeier MR, et al. SLC2A8 (GLUT8) is a mammalian trehalose transporter required for trehalose - induced autophagy. Scientific Reports. 2016
Slide 20: Pathogenesis and Proposed Approaches in ALS
• ALS is a fatal neurodegenerative disease • C haracterized by progressive muscle paralysis determined by the degeneration of motoneurons in the motor cortex brainstem and spinal cord • P athogenetic mechanisms are still unclear • P roposed therapeutic strategies target only one or a few of alterati
Slide 21: Pathogenetic Mechanisms Involved in ALS
PATHOGENETIC MECHANISMS INVOLVED IN ALS Bonafede R, Mariotti R. ALS Pathogenesis and Therapeutic Approaches: The Role of Mesenchymal Stem Cells and Extracellular Ves icl es. Front Cell Neurosci. 2017;11:80. Published 2017 Mar 21. doi:10.3389/fncel.2017.00080 21
Slide 22: SLS-005 (Trehalose) in ALS
Induces autophagy via lysosomal - mediated TFEB activation in models of motoneuron degeneration SLS - 005 (TREHALOSE) IN ALS • Sanfilippo syndrome (or MPS III) is an inherited monogenic lysosomal storage disorder divided into four subtypes • Occurrence of 1 in every 70,000 • Progressive disorder, wi
Slide 23: SLS-005 (Trehalose) in ALS: Mouse Model
MTOR - independent, autophagic enhancer trehalose prolongs motor neuron survival and ameliorates the autophagic flux defect in a mouse model of ALS SLS - 005 (TREHALOSE) IN ALS • Sanfilippo syndrome (or MPS III) is an inherited monogenic lysosomal storage disorder divided into four subtypes • Occurr
Slide 24: SLS-005 (Trehalose) in ALS: SOD1 Mouse Model
Trehalose decreases mutant SOD1 expression and alleviates motor deficiency in early but not end - stage ALS in a SOD1 - G93A mouse model SLS - 005 (TREHALOSE) IN ALS • Sanfilippo syndrome (or MPS III) is an inherited monogenic lysosomal storage disorder divided into four subtypes • Occurrence of 1 i
Slide 25: SLS-005 Phase II/III in ALS
• Randomized double blind placebo - controlled trial • 160 Patients with sporadic or familial ALS with onset of ALS < 36 months • Randomized 3:1 trehalose to placebo and 24 - week treatment period • ~90% power to detect a slowing in the rate of ALSFRS - R progression of 25% Primary Efficacy Endpoint
Slide 26: SLS-005 in Spinocerebellar Ataxia (SCA)
SCA Is a serious neurodegenerative disease caused by degeneration of the cerebellum characterized by progressive unsteadiness of gait and stance, impaired coordination of limb movements, slurred speech and abnormal eye movements SCA type 3 , also known as Machado - Joseph Disease (MJD), is character
Slide 27: SLS-005 in Humans with SCA Type 3
Natural History Control – (SARA) 1 • Natural history data suggests that individuals with SCA3 would be expected to show a measurable increase on SARA which is indicative of disease progression and worsening of symptoms BB - MJD - 201: 15 patients, open - label 24 - wk study 2 • Weekly infusions of S
Slide 28: SLS-005 Phase II/III in SCA
Primary Endpoint • Change from baseline in the modified Scale for Assessment and Rating Ataxia (m - SARA) total score at Week 52 in participants with SCA3 Key Secondary Endpoints • Change from baseline in Clinical Global Impression of Severity (CGI - S) at Week 52 • Change from baseline in Patient G
Slide 29: SLS-004 (Gene Therapy)
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Slide 30: SLS-004: Alpha-Synuclein as a Therapeutic Target
SLS - 004: ALPHA - SYNUCLEIN AS A THERAPEUTIC TARGET Brundin P, Dave KD, Kordower. Therapeutic approaches to target alpha - synuclein pathology. Experimental Neurology. 2017;298:225 - 2 35. α - SYN LEWY BODY TOXIC PROTEIN DNA mRNA CELL NUCLEUS INSIDE CELL OUTSIDE CELL 1 2 3 4 5 PRODUCTION Lower α -
Slide 31: SLS-004: Targeted Epigenome-Editing
All in one vector Lentiviral particles SNCA - intron 1 methylation SNCA - mRNA expression α - synuclein protein levels Cellular phenotypes SLS - 004: TARGETED EPIGENOME - EDITING Kantor B, et al. (2018) Downregulation of SNCA Expression by Targeted Editing of DNA Methylation: A Potential Strategy fo
Slide 32: SLS-004 Evaluated in Established PD Model
SLS - 004 EVALUATED IN ESTABLISHED PD MODEL Right Hemisphere SNpc* injection Left Hemisphere SNpc* injection AAV - A53T w/ intron1 + Control Vector AAV - A53T w/ intron1 + SLS - 004 • Human A53T α - Synuclein viral vector 1 with human intron 1 • SLS - 004 directed towards human intron 2 • Well chara
Slide 33: SLS-004: CRISPR Epigenetic Modulation of SNCA Is Successful
RIGHT LEFT Loss of TH+ Dopamine neurons in SNpc with Control Recovery of TH+ Dopamine neurons in SNpc with SLS - 004 SLS - 004: CRISPR EPIGENETIC MODULATION OF SNCA IS SUCCESSFUL AAV - A53T + Control vector AAV - A53T + SLS - 004 vector
Slide 34: Robust Pipeline Promises Continued Growth in CNS and Beyond
ROBUST PIPELINE PROMISES CONTINUED GROWTH IN CNS AND BEYOND Indication(s) Pre - IND Phase I Phase II Phase III Status SLS - 002 (intranasal ketamine ) Acute Suicidal Ideation and Behavior (ASIB) in Major Depressive Disorder (MDD) Registration Directed study Part 2 expected readout Q3 2023 SLS - 005
Slide 35: Seelos Is Advancing Innovative Therapeutics to Address Unmet Medical Needs
SEELOS IS ADVANCING INNOVATIVE THERAPEUTICS TO ADDRESS UNMET MEDICAL NEEDS FOR PATIENTS WITH PSYCHIATRIC AND MOVEMENT DISORDERS Developing a broad portfolio of late - stage CNS product candidates with demonstrated mechanism of action Assembling a team of established industry leaders Advancing clinic
Slide 36: Appendix
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Slide 37: SLS-002 Study Enrollment Criteria
This study enrolled 17 patients diagnosed with MDD requiring psychiatric hospitalization due to significant risk of suicide and: • Baseline total score of ≥ 28 points on the Montgomery - Åsberg Depression Rating Scale (MADRS) • Score of 5 or 6 on MADRS Item - 10 • Total score of ≥ 15 points on the S
Slide 38: Detailed Summary of MADRS and MADRS Item-10 Data
MADRS • 76.5% response rate on Day 1; 92.9% response rate on Day 16; and 100% response rate on Day 29 (14 days after the last dose on Day 15) • Remission rate on Day 1 = 35.3%; Day 16 = 78.6%; and Day 29 = 76.9% • Total Score (baseline) = 39.4; Day 1, mean = 14.5; Day 16, mean = 7.4; and Day 29, mea
Slide 39: Detailed Summary of Suicide Scales Data
CGIS - SI/B • 94% patients had ≥ 1 point within - patient change from their baseline on Day 1 and 100% of patients on Day 16 had ≥ 1 point change and 86% had 2 or 3 point within - patient change from their baseline • Baseline = 3.8; Day 1, mean = 1.6; and Day 16, mean = 1.1 S - STS • 94% of patients
Slide 40: SLS-002-201 Study Design
SLS - 002 - 201 STUDY DESIGN Open - Label Trial Design SLS - 002 - 201 is a 2 - Part Study Part 1 – Pilot, Open - Label – Completed (17 subjects) Part 2 – Double - Blind, Placebo - Controlled – Ongoing 40
Slide 41: SLS-002-201 Part 1 Efficacy Results
SLS - 002 - 201 STUDY – PART 1 EFFICACY RESULTS Clinical Improvements in Four Scales over Study Duration 41
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