Avenue Therapeutics, Inc. · OTC:ATXI
Avenue Therapeutics — Investor Presentation (March 2023)
35 slides from Avenue Therapeutics's investor presentation, March 2023
Slide 1: Corporate Overview
Corporate Overview AVENUE THERAPEUTICS, INC. ⎸ NASDAQ: ATXI ⎸ March 2023
Slide 2: Forward looking statement
Forward looking statement This presentation contains predictive or “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of current or historical fact contained in this presentation, including statements that exp
Slide 3: Executive Summary
Executive Summary Avenue Therapeutics is a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of rare and neurologic diseases In February 2023, we executed a license with AnnJi Pharmaceutical for AJ201; this business development effor
Slide 4: Our clinical stage assets address large unmet patient and market needs
Our clinical stage assets address large unmet patient and market needs in the neurologic therapeutic space Pipeline Asset AJ201 IV Tramadol BAER-101 Spinal and Bulbar Epilepsy and Indication Muscular Atrophy Post operative pain acute anxiety (Kennedy’s Disease) Activation of Nrf1 / Nrf2 Opioid agoni
Slide 5: Our pipeline has potential near-term value inflection points
Our pipeline has potential near-term value inflection points Indication Phase 1 Phase 2 Phase 3 Rights Phase 3 Pain Model Studies Post-operative IV Tramadol Pain US Confirmatory Safety Study Reach agreement with FDA Spinal and Bulbar on confirmatory safety study US, EU, Muscular Atrophy required f
Slide 6: AJ201 IV Tramadol BAER-101
1 2 3 AJ201 IV Tramadol BAER-101 6
Slide 7: AJ201 is being developed for Spinal and Bulbar Muscular Atrophy and other PolyQ diseases
AJ201 is being developed for Spinal and Bulbar Muscular Atrophy and may address other PolyQ diseases AJ201 Spinal and Bulbar Muscular Other PolyQ diseases Atrophy (SBMA)* (e.g., Huntington’s disease, spinocerebellar ataxia, etc.) Note: Spinal and Bulbar Muscular Atrophy is also known as Kennedy’s di
Slide 8: AJ201 adds a cutting-edge asset to Avenue's neurologic asset portfolio
AJ201 adds a cutting-edge asset to Avenue’s neurologic asset portfolio Ongoing Rare Neuro Phase 1b/2a Disease Clinical Trial Potential to No Effective Treat Other First-in-Class Treatments PolyQ Diseases AJ201 was licensed from AnnJi Pharmaceutical in Q1 2023 with an impressive preclinical and clini
Slide 9: SBMA is a polyglutamine (PolyQ) disease with mutant protein aggregation
SBMA is a polyglutamine (PolyQ) disease, which is characterized by mutant protein aggregation and progressive neurodegeneration Normal Polyglutamine Expansion At least nine neurodegenerative diseases (NDD) are caused by the expansion of CAG repeats encoding polyglutamine (polyQ) tracts in affected
Slide 10: SBMA affects multiple organ systems with symptoms worsening over time
SBMA affects multiple organ systems with symptoms worsening over time SBMA is an X-linked polyQ disease, which results in only men developing the full spectrum of symptoms Although there is a range of cited prevalence rates in the literature, a recent study used genetic analysis to estimate dise
Slide 11: AJ201 is a pleiotropic small molecule that may treat SBMA
AJ201 is a pleiotropic small molecule that may treat SBMA by enhancing mutant protein degradation and decreasing neuroinflammation SBMA disease pathway Inherited X-linked Mutation Androgen Receptor (AR) Gene Cytotoxic CAG CAG CAG CAG CAG CAG CAG CAG Polyglutamine Expansion Misfolded Accumulation of
Slide 12: AJ201 supported by preclinical efficacy data in SBMA models
The development of AJ201 is supported by preclinical efficacy data in SBMA models AJ201 led to improved motor function in AJ201 reduced levels and accumulation of the mutant symptomatic animals compared with vehicle control AR protein in mouse muscle tissues Grip test in SBMA disease mouse model (AR
Slide 13: Phase 1 SAD and MAD showed favorable pharmacokinetic and safety profile
The Phase 1 SAD and MAD in healthy volunteers showed a favorable pharmacokinetic and safety profile SAD and MAD* study overview SAD cohorts AUC0-last (hr*ng/mL) A total of 72 subjects enrolled; no subjects were withdrawn due 3500 to safety concerns 3000 2500 2000 Well-tolerated with no serious a
Slide 14: Phase 1b/2a study of AJ201 in SBMA patients expected to read out in 2024
Phase 1b/2a study of AJ201 in SBMA patients is expected to read out top-line data in 2024 Phase 1b/2a multicenter double blind randomized clinical trial overview Primary Objective Assessing the safety, tolerability of AJ201 in subjects with clinically and genetically defined SBMA Secondary Assessing
Slide 15: AJ201 is the lead clinical program targeting SBMA
AJ201 is the lead clinical program targeting SBMA Product Developer MoA Preclinical Phase I Phase II Phase III Approved Japan only & Gonadotropin releasing Leuprorelin Takeda Limited hormone stimulants efficacy Activation of Nrf1 / Nrf2 AJ201 AnnJi and promotion of AR degradation NIDO361 Nido Biosci
Slide 16: AJ201 has orphan disease designations
AJ201 has orphan disease designations to enable a robust market presence Orphan drug status granted Indication US FDA EMA SBMA Huntington’s disease Spinocerebellar ataxia Intellectual property overview Orphan drug designation (ODD) provides 7 years of market exclusivity in the US and 10 ye
Slide 17: AJ201 IV Tramadol BAER-101
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Slide 18: Tramadol has a unique dual mechanism of action among IV analgesics
Tramadol has a unique dual mechanism of action among IV analgesics Opioid Inhibitor of Norepinephrine Opioid Agonist Efficacy & Serotonin Re-uptake Blocking pain signal transmission with Less at both the spinal and brain levels Abuse Blocking pain signal transmission at the spinal level Potential Sc
Slide 19: Safety and efficacy demonstrated in two Phase 3 trials in over 700 patients
Safety and efficacy profiles have been demonstrated in two Phase 3 trials in over 700 patients Both pain relief study models show benefit of Tramadol over placebo Study AVE-901-102 (Bunionectomy) SPID24 Study AVE-901-103 (Abdominoplasty) SPID24 IV Tramadol 50 mg achieved primary endpoint and all key
Slide 20: Regulatory history and path forward for IV Tramadol NDA
Regulatory history and now a path forward for IV Tramadol NDA 2019 2021 2022 December 2019: We submitted a New February 2021: We addressed the August 9, 2022: We met with the FDA to Drug Application (“NDA”) for IV manufacturing issue identified in the discuss a study design to address the Tramadol a
Slide 21: AJ201 IV Tramadol BAER-101
1 2 3 AJ201 IV Tramadol BAER-101 21
Slide 22: BAER-101 is being developed for epilepsy and acute anxiety
BAER-101 is being developed for epilepsy and acute anxiety BAER-101 Epilepsy Acute Anxiety Conditions Anti-Convulsant Effects (e.g., Panic Disorder) Anxiolytic Effects 22
Slide 23: BAER-101 is a selective GABAα receptor agonist
BAER-101 is a selective GABAα receptor agonist that produces anticonvulsant and anxiolytic activity GABA receptors are the major inhibitory neurotransmitter receptors in the mammalian brain GABA receptors have three major subunits α β γ, which are organized into a pentameric structure Each sub
Slide 24: BAER-101 may have a more tolerable side effect profile than benzodiazepines
BAER-101 may have a more tolerable side effect profile compared to nonselective benzodiazepines due to greater GABA subtype selectively Predicted effect of targeting GABAα subtypes GABAA subtypes Therapeutic Effect α1 α2 α3 α5 Anti-convulsant Positive Anxiolysis Analgesia Musc
Slide 25: Epilepsy and acute anxiety patients have significant unmet needs
Epilepsy and acute anxiety patients are prescribed benzodiazepines, but have significant unmet needs for therapies with improved safety profiles Epilepsy Acute Anxiety Conditions U.S. Prevalence ~6M patients (~360M patients with any anxiety 3 - 4M patients (~65M patients worldwide) condition worldwi
Slide 26: BAER-101 can target the large market of patients prescribed BZDs
BAER-101 can target the large market of patients prescribed BZDs by targeting drug resistant epilepsy, orphan epilepsy, or acute anxiety Addressable market Patients prescribed benzodiazepines Drug Resistant Epilepsy 800K – 1M prevalent Acute Anxiety patients U.S. 6.8M prevalent patients U.S. Orphan
Slide 27: BAER-101 has a compelling profile to address this market
BAER-101 has a compelling profile to address this market Preclinical High receptor disease model occupancy efficacy Safety Preclinical in humans Selective disease model demonstrated in vitro profile safety and in clinical reduced abuse trials in liability >700 patients BAER-101 was licensed from Ast
Slide 28: AstraZeneca completed clinical studies and demonstrated safety
AstraZeneca completed 10 clinical studies and demonstrated safety across trials at selected doses BAER-101/AZD7325 was tested in over 700 subjects (healthy volunteers and patients) Side effects were mild or moderate with the most common side effects being dizziness and somnolence In two Phase
Slide 29: Clinical studies demonstrate positive sedation and cognition effects
Clinical studies demonstrate positive sedation and cognition effects Example: subset analysis from Phase 2 generalized anxiety study Subjects experiencing sleepiness % of subjects (N=36-66) 35 30 Lorazepam 2mg BID 25 BAER-101 15mg BID 20 15 BAER-101 5mg BID 10 Placebo 5 0 3 6 9 12 15 18 21 24 27 30
Slide 30: We plan to initiate two Phase 1b studies
We plan to initiate two Phase 1b studies that have the potential to translate well into later development programs Epilepsy: Photosensitivity Study Acute Anxiety: Hypercapnia CO2 Inhalation Model The epilepsy photosensitivity model is a clinical The CO2 inhalation challenge is a clinical transla
Slide 31: BAER-101 is differentiated from others in the class
BAER-101 is differentiated from others in the class Differentiation vs other selective GABAα therapies Company Asset Selectivity Phase Indications Avenue Therapeutics BAER-101 α2/3-preferring Phase 1 Epilepsy and panic disorder Cerevel (Nasdaq: CERE) darigabat α2/3/5-preferring Phase 2 Epilepsy and
Slide 32: Company Overview
Company Overview A AVENUE
Slide 33: We are searching the world for first-rate neuro / rare disease assets
We are searching the world for first-rate neuro / rare disease assets with these characteristics Imaging and Clinical Stage Biomarker with Human Driven Data Endpoints Feasible Clinical Development Programs with Reasonable Costs 33
Slide 34: Experienced management team and board of directors
We are led by an experienced management team and board of directors Management Board of Directors Lindsay Rosenwald MD CEO, Fortress Biotech Jay Kranzler MD PhD CEO, Urica Therapeutics Alexandra MacLean MD David Jin Michael Ryan Neil Herskowitz CEO Interim CFO VP Clinical Operations & Founder, ReGen
Slide 35: Executive Summary
Executive Summary Avenue Therapeutics is a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of rare and neurologic diseases In February 2023, we executed a license with AnnJi Pharmaceutical for AJ201; this business development effor
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