Avenue Therapeutics, Inc. · OTC:ATXI
Avenue Therapeutics — IPO Roadshow (October 2023)
27 slides from Avenue Therapeutics's IPO roadshow presentation, Oct 4, 2023
Slide 1: Avenue Therapeutics Corporate Presentation
AVENUE THERAPEUTICS, INC. ⎸ NASDAQ: ATXI ⎸ OCTOBER 2023 Avenue Therapeutics Corporate Presentation
Slide 2: Disclaimers: Cautionary Statement Regarding Forward-Looking Statements
2 Disclaimers Cautionary Statement Regarding Forward - Looking Statements This presentation of Avenue Therapeutics, Inc . (“we,” “us,” “our,” “Avenue,” or the “Company”) contains predictive or “forward - looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 .
Slide 3: Disclaimers: Forward-Looking Statements (cont'd) and Important Information
3 Disclaimers Cautionary Statement Regarding Forward - Looking Statements (cont’d) ability to maintain compliance with the obligations under our intellectual property licenses and funding arrangements with third parties, without which licenses and arrangements we could lose rights that are important
Slide 4: Offering Summary
4 Offering Summary ISSUER: Avenue Therapeutics, Inc. (“Avenue” or the “Company”) EXCHANGE/SYMBOL: NasdaqCM : ATXI OFFERING SIZE: Up to $12.0 Million OFFERING TYPE : Best Efforts S - 1 Follow - On Offering SECURITIES OFFERED: Units consisting of one share of Common Stock (or Pre - Funded Warrants in
Slide 5: Executive Summary
5 Executive Summary Two drugs are expected to deliver data in 2024 including the IV tramadol Phase 3 development program • Avenue Therapeutics is a company focused on developing therapies to treat neurologic diseases including acute hospital pain and a rare neuromuscular condition • IV tramadol has
Slide 6: Diverse portfolio including a first-in-class program in high-value neurologic landscape
6 Diverse portfolio including a first - in - class program in high - value neurologic landscape with significant unmet patient need Source: 1. Acute Pain Market to Observe Growth at a CAGR of 8.3% During the Study Period (2019 - 2032), Assesses DelveInsight , 2023. 2. M. Zanovello et al., Oxford Uni
Slide 7: Multiple potential near-term catalysts in CNS-focused pipeline
7 Multiple potential near - term catalysts in CNS - focused pipeline Indication Phase 1 Phase 2 Phase 3 Next Milestone Rights IV Tramadol Post - operative Pain Start and Complete Phase 3 Safety Study U.S. AJ201 Spinal and Bulbar Muscular Atrophy (SBMA) / Kennedy’s Disease Phase 1b/2a Topline Results
Slide 8: Executing to plan with multiple value-driving milestones ahead
8 Executing to plan with multiple value - driving milestones ahead BAER - 101 in Epilepsy & Acute Anxiety AJ201 in SBMA Compelling Phase 1 safety data in healthy volunteers Dosed eight patients in lead Phase 1b/2a study of AJ201 in SBMA since first patient in 3Q23 Final results for Phase 1b/2a study
Slide 9: Tramadol has unique dual mechanism of action among IV analgesics
9 Tramadol has unique dual mechanism of action among IV analgesics designed to block patient’s pain signal with reduced abuse potential Note: Schedule IV substances are defined as drugs with a low potential for abuse and low risk of dependence. Schedule II subs tan ces are defined as drugs with a hi
Slide 10: IV tramadol expected to be used before moving to stronger opioids
10 IV tramadol expected to be used before moving to stronger opioids STEP 1 Non - opioid analgesics IV acetaminophen (+ IV NSAIDs) STEP 2 Atypical opioid IV tramadol + Non - opioid analgesics IV acetaminophen (+ IV NSAIDs) + Strong conventional opioids IV morphine/ hydromorphone STEP 3 Non - opioid
Slide 11: Phase 3 efficacy studies conducted in more than 750 patients
11 Phase 3 efficacy studies conducted in more than 700 patients Orthopedic Model (Bunionectomy) 405 Patients Placebo (n=135) IV tramadol 25 mg (n=135) IV tramadol 50 mg (n=135) Soft Tissue Model (Abdominoplasty) 360 Patients Placebo (n=135) IV tramadol 50 mg (n=135) Morphine (n=90) PRIMARY ENDPOINT
Slide 12: IV tramadol achieved primary endpoint and all key secondary endpoints
12 IV tramadol 50 mg achieved primary endpoint and all key secondary endpoints Study AVE - 901 - 103 (Abdominoplasty) SPID24 Study AVE - 901 - 102 (Bunionectomy) SPID24 Both pain relief study models show benefit of tramadol over placebo
Slide 13: Regulatory history and now a path forward for IV tramadol
13 Regulatory history and now a path forward for IV tramadol NDA 2024+ 2022 2023 Meeting held in August with FDA Division of Anesthesia, Analgesia, and Addiction Products (DAAAP) on potential path forward for a safety study design that could address FDA’s concerns identified in previous Complete Res
Slide 14: Phase 3 safety study to show non-inferiority of IV tramadol compared to IV morphine
14 Phase 3 safety study to show non - inferiority of IV tramadol compared to IV morphine in opioid induced respiratory depression endpoint • When patients require rescue medication for breakthrough pain for either arm (IV tramadol or IV morphine), they will receive IV hydromorphone, thereby creating
Slide 15: AJ201 in development as novel, first-in-class treatment for SBMA
15 AJ201 in development as novel, first - in - class treatment for SBMA * Orphan Drug Designation SBMA is Spinal and Bulbar Muscular Atrophy, also known as Kennedy’s Disease Potential “Pipeline in a Product” for PolyQ diseases Promising preclinical efficacy and clinical safety data Novel protein deg
Slide 16: SBMA: Devastating, rare neurodegenerative disease with no FDA approved treatments
16 SBMA: Devastating, rare neurodegenerative disease with no FDA approved treatments for patients Source: 1. M. Zanovello et al., Oxford University Press on behalf of the Guarantors of Brain. 2023. • Rare, X - linked PolyQ disease primarily affecting men • Weakening of bulbar muscles affects chewing
Slide 17: AJ201 awarded ODD from U.S. FDA in SBMA, HD and select SCA indications
17 AJ201 awarded ODD* from U.S. FDA in SBMA, HD and select SCA indications Polyglutamine (PolyQ) diseases are characterized by mutant protein aggregation and progressive neurodegeneration * Orphan Drug Designation • 9+ neurodegenerative diseases (NDD) caused by expansion of CAG repeats encoding poly
Slide 18: Potential therapeutic activity of AJ201 includes enhancing mutant AR protein degradation
18 Potential therapeutic activity of AJ201 includes enhancing mutant AR protein degradation and decreasing neuroinflammation Source: Bott et al., Hum Mol Genet. 2016 SBMA disease pathway Nrf1 Pathway Activation Nrf2 Pathway Activation Mutant Androgen Receptor (AR) Degradation AJ201 potential activit
Slide 19: Phase 1b/2a study of AJ201 in SBMA patients expected to report data in 1H 2024
19 Phase 1b/2a study of AJ201 in SBMA patients expected to report data in 1H 2024 Phase 1b/2a multicenter double blind randomized clinical trial overview 4 weeks Screening AJ201, 600mg, QD, 12 weeks Treatment (n=15) 4 weeks Follow - up Placebo, QD, 12 weeks Treatment (n=5) Primary Objective Assessin
Slide 20: AJ201 is currently the lead clinical program targeting SBMA
20 AJ201 is currently the lead clinical program targeting SBMA Product MoA Preclinical Phase I Phase II Phase III Approved Leuprorelin Gonadotropin releasing hormone stimulant Japan only & limited efficacy AJ201 Activation of Nrf1 / Nrf2 and promotion of AR degradation NIDO361 AR BF3 modulator AAV -
Slide 21: AJ201 has multiple orphan disease designations that provide regulatory exclusivity
21 AJ201 has multiple orphan disease designations that provide regulatory exclusivity upon approval Orphan drug status granted Intellectual property overview • Orphan drug designation (ODD) provides 7 years of market exclusivity in the U.S. and 10 years in the EU • Patents to provide market protecti
Slide 22: BAER-101 in development as a differentiated targeted therapy for epilepsy
22 BAER - 101 in development as a differentiated targeted therapy for treatment of epilepsy Phase 2a ready Preclinical disease model efficacy with reduced abuse liability Selective in vitro profile designed to improve safety Existing treatments have numerous AEs that affect treatment Safety in human
Slide 23: BAER-101 targets GABA α2 and α3 subtypes
23 BAER - 101 targets GABA α2 and α3 subtypes more than α1 and α5, potentially improving side effect profile compared to nonselective benzodiazepines Source: Jacob et al., Nature Reviews Neuroscience, 2008; Luo, Y., & Balle, T. Basic and Clinical Pharmacology & Toxicology, 2 022 ; McKernan, et al.,
Slide 24: BAER-101 is differentiated from others in the class
24 BAER - 101 is differentiated from others in the class Novel development candidate with alpha 2/3 subtype - preferring selectivity, an important differentiating factor in improving tolerance and safety Company Asset Selectivity Phase Indications Avenue Therapeutics BAER - 101 α2/3 - preferring Pha
Slide 25: Executing to plan with multiple value-driving milestones ahead
25 Executing to plan with multiple value - driving milestones ahead BAER - 101 in Epilepsy & Acute Anxiety AJ201 in SBMA Compelling Phase 1 safety data in healthy volunteers Dosed eight patients in lead Phase 1b/2a study of AJ201 in SBMA since first patient in 3Q23 Final results for Phase 1b/2a stud
Slide 26: Led by experienced management team and board of directors
26 Led by experienced management team and board of directors Jay Kranzler MD PhD CEO, Urica Therapeutics Lindsay Rosenwald MD CEO, Fortress Biotech Neil Herskowitz Founder, ReGen Capital Curtis Oltmans Chief Legal Officer, Fulcrum Therapeutics Faith Charles Partner, Thompson Hine LLP Alexandra MacLe
Slide 27: Avenue Therapeutics, Inc. October 2023
AVENUE THERAPEUTICS, INC. ⎸ NASDAQ: ATXI ⎸ OCTOBER 2023