Paxmedica, Inc. · OTC:PXMD
Paxmedica — Investor Presentation (October 2022)
24 slides from Paxmedica's investor presentation, Oct 12, 2022
Slide 1: Addressing Urgent Needs In Serious Neurologic Disorders
© 2022. PaxMedica , Inc. Proprietary Information. All rights reserved Addressing Urgent Needs In Serious Neurologic Disorders Nasdaq: PXMD October 2022
Slide 2: Cautionary Note on Forward-Looking Statements and Disclaimers
2 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved Cautionary Note on Forward - Looking Statements and Disclaimers This Presentation contains "forward - looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, opera
Slide 3: Overview
© 2022. PaxMedica , Inc. Proprietary Information. All rights reserved 3 Overview PaxMedica is a clinical stage biopharmaceutical company focusing on the development of anti - purinergic drug therapies (“APT”) for the treatment of disorders with intractable neurologic symptoms including Autism Spectr
Slide 4: Investment Highlights
4 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved Investment Highlights • Multiple potential catalysts expected through 2024 • Pursuing clinical indications with significant unmet needs and few options for drug therapy o Autism Spectrum Disorder (ASD) o Orphan Designated Rare T
Slide 5: Management Team Led By Experienced Pharma Entrepreneurs
5 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved 5 Management Team Led By Experienced Pharma Entrepreneurs Michael Derby Executive Chairman Zach Rome Chief Operating Officer Howard J. Weisman Chief Executive Officer Stephen Sheldon Chief Financial Officer NASDAQ: PXMD Stefan S
Slide 6: Board of Directors
6 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved 6 Board of Directors NASDAQ: PXMD Michael Derby Howard J. Weisman Zach Rome Karen LaRochelle Dr. John F. Coelho Charles J. Casamento
Slide 7: NDA Submission Plans
© 2022. PaxMedica , Inc. Proprietary Information. All rights reserved 7 PAX - 101 NDA Submission Plans 7 NASDAQ: PXMD © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved
Slide 8: Potential Value and Liquidity Via Early NDA Submission
8 8 Potential Value and Liquidity Via Early NDA Submission Priority Review Voucher Program for Neglected Tropical Diseases - HAT • IV suramin is the standard of care in the treatment of potentially lethal infections caused by Stage 1 T. Brucei Rhodesiense Human African Trypanosomiasis (HAT), aka Afr
Slide 9: Anticipated Near-term Milestone: Complete NDA for HAT
9 9 Anticipated Near - term Milestone: Complete NDA for HAT Plan for Potential Approval and Receipt of Transactable Voucher: • Primary efficacy data expected from a retrospective data analysis of patients treated with suramin for HAT compared to a natural history cohort from a prior HAT epidemic bef
Slide 10: Program for NDA and PRV Underway
10 10 Program for NDA and PRV Underway Regulatory – FDA Guidance Received • HAT is listed as a neglected tropical disease in the FDA Tropical Disease PRV Program • HAT submission will be a 505(b)(2) - NDA o FDA concurs that a prospective efficacy trial would not be feasible due to the rare incidence
Slide 11: Development Goals
11 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved 11 PAX - 101 Development Goals PAX - 101 NDA, if approved, will be the first approval of Suramin in the US market Potential Milestones Through 2024 Priority Review Voucher Sale Up to 7 Years of US Commercial Exclusivity PAX - 1
Slide 12: PAX-101 and PAX-102: Potential Game Changing Treatments for Autism
© 2022. PaxMedica , Inc. Proprietary Information. All rights reserved 12 PAX - 101 and Pax - 102 Potential Game Changing Treatments for Autism © 2022. PaxMedica, Inc. Proprietary Information. All rights reserved 12 NASDAQ: PXMD
Slide 13: Autism Spectrum – A Significant Clinical Opportunity
13 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved • Global Autism treatment market reached $3.3B in 2018 and is expected to exceed $4.6 Billion in 2026 (CAGR 4.3%) 1 o Irritability, a non - core symptom associated with ASD, is treated with available anti - psychotic drugs but
Slide 14: A New Treatment Pathway Proposed for Autism
14 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved A New Treatment Pathway Proposed for Autism PAX - 101 may improve symptoms of ASD by blocking the action of ATP on purine receptors that are over - expressed during immune system responses PAX - 101 Purine Receptor ATP Cell Res
Slide 15: PAX-102 – Proprietary Intranasal Delivery
15 15 PAX - 102 – Proprietary Intranasal Delivery to Expand Clinical Applications of Antipurinergic Drugs • Promising pre - clinical data • Patents pending which would potentially extend exclusivity through at least 2039 • More convenient and cost - effective as “use at home” versus IV infusion © 20
Slide 16: Forging a New Path in ASD Treatment: Phase 2 Results in ASD
© 2022. PaxMedica , Inc. Proprietary Information. All rights reserved 16 Forging a New Path in ASD Treatment: PAX - 101 Phase 2 Results in ASD 16 NASDAQ: PXMD © 2022. PaxMedica, Inc. Proprietary Information. All rights reserved
Slide 17: Clinical Trial Results - 2021
17 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved PAX - 101 Clinical Trial Results - 2021 Randomized, double - blind, placebo controlled, dose - ranging proof of concept study • Primary: ABC Core (subscales 2, 3, & 5) • Secondaries: ABC Total Score, CGI - I, Autism Treatment E
Slide 18: Patient Population - Autism Clinical Trial
18 18 Patient Population - Autism Clinical Trial • 6 sites in South Africa • Diverse population of 52 boys, mean age 8.4 years • 44 completers o 5 withdrawals due to COVID - 19 lockdowns and site closures o 1 for an adverse event o 2 for other reasons 18 © 2022. PaxMedica , Inc. Proprietary Informat
Slide 19: Primary Endpoint - Aberrant Behavior Checklist (ABC) Change from Baseline
19 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved 19 Primary Endpoint - Aberrant Behavior Checklist (ABC) Change from Baseline through Week 14 Individual ABC Subscale Changes -15 -13 -11 -9 -7 -5 -3 -1 1 ABC-I (Irritability) ABC-II (Social Withdrawal) ABC-III (Stereotypy) ABC-
Slide 20: Primary Endpoint - ABC Low Dose Outperforms Placebo
20 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved 20 Primary Endpoint - Aberrant Behavior Checklist (ABC) PAX - 101 Low Dose (10mg/kg) Outperforms Placebo in Core and Total Symptoms Measures * Drug administration visit -16.00 -14.00 -12.00 -10.00 -8.00 -6.00 -4.00 -2.00 0.00 W
Slide 21: Secondary Endpoint - CGI-I Overall Severity Change from Baseline
21 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved 21 No Change CGI - I Overall Severity Score, Scaled Change from Baseline to Week 14 (ITT population, mean ± SE) 0.0 1.0 2.0 3.0 Placebo (N=18) PAX-101, 10 mg/kg (N=15) p=0.016 PAX - 101 is IV suramin Minimally Improved Much Imp
Slide 22: Phase 2 Trial Summary Results
22 22 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved PAX - 101 – Phase 2 Trial Summary Results • Both dose regimens showed improvement trends from baseline on ABC Core, but study was not statistically powered • 10 mg/kg dose group showed a statistically significant and clinica
Slide 23: PaxMedica Business Goals 2022
23 © 2022. PaxMedica , Inc. Proprietary Information. All rights reserved 23 PaxMedica Business Goals 2022 - 24 • Raise awareness of company mission to develop therapeutics for urgent and unmet needs in serious neurologic disorders • Complete all necessary pre - clinical, non - clinical and clinical
Slide 24: Thank You! For More Information Contact
© 2022. PaxMedica , Inc. Proprietary Information. All rights reserved THANK YOU! For More Information Contact: Stephanie Prince PCG Advisory sprince@pcgadvisory.com (646) 863 - 6341 www.paxmedica.com NASDAQ: PXMD 25