Full Press Release Details
Forward Looking Statements Statements in this presentation that are not
purely historical in nature are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this presentation include, without limitation, statements regarding:
ARS Pharma's plan to file its NDA early in the second quarter of 2024, with an anticipated PDUFA action date and launch of neffy, if approved, in the second half of 2024; the timing of the EMA's decision and submissions to other foreign
regulatory authorities; the potential market, demand and expansion opportunities for neffy; ARS Pharma's expected competitive position; whether the results will be sufficient to demonstrate that neffy is at least as effective as injectable
epinephrine; the timelines for potential regulatory filings, approvals and commercialization of neffy in ex-US regions; ARS Pharma's marketing and commercialization strategies, including potential partnerships in foreign jurisdictions;
potential benefits of neffy, if approved, including the likelihood that doctors will prescribe neffy and that allergy patients and caregivers will choose to carry and dose neffy compared to needle-bearing options; the expectation of neffy attaining
coverage, including without restriction for 80% of commercial lives within a year of launch; ARS Pharma's anticipated cash, cash equivalents and short-term investments on hand upon any future approval and launch of neffy; the expected size,
composition and reach of ARS Pharma's sales force; the availability and functionality of neffyExperience and neffyConnect; the anticipated pricing and co-pay buydown; the anticipated timing and costs of future studies and commercialization
efforts, and their impact on operating runway; ARS Pharma's projected operating runway; expected intellectual property protection; and any statements of assumptions underlying any of the foregoing. These forward-looking statements are subject
to the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking
statements. Words such as "anticipate," "could," "demonstrate," "expect," "indicate," "may," "plan," "potential," "will" and similar
expressions are intended to identify forward-looking statements. These forward-looking statements are based upon ARS Pharma's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results
and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation: the PDUFA target action date may be further delayed due to
various factors outside ARS Pharma's control; the ability to obtain and maintain regulatory approval for neffy; the results of the new clinical trial may not support the approval of neffy; results from clinical trials may not be indicative of
results that may be observed in the future; potential safety and other complications from neffy; the labelling for neffy, if approved; the scope, progress and expansion of developing and commercializing neffy; potential for payers to delay, limit,
or deny coverage for neffy; the size and growth of the market therefor and the rate and degree of market acceptance thereof vis- -vis intramuscular injectable products; ARS Pharma's ability to protect its intellectual property position;
uncertainties related to capital requirements; and the impact of government laws and regulations. Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking
statements are included under the caption "Risk Factors" in ARS Pharma's Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, filed with the Securities and Exchange Commission ("SEC") on November 9,
2023. This and other documents ARS Pharma files with the SEC can also be accessed on ARS Pharma's website at ir.ars-pharma.com by clicking on the link "Financials & Filings" under the "Investors & Media" tab.
The forward-looking statements included in this presentation are made only as of the date hereof. ARS Pharma assumes no obligation and does not intend to update these forward-looking statements, except as required by law.
Today's Speakers ARS Management Key Opinion Leaders Richard
Lowenthal, M.S., MSEL Jonathan Spergel, M.D., Ph.D. Chief Executive Officer, Co-Founder Chief of Allergy Program Led FDA approvals for multiple Children's Hospital of Philadelphia nasal spray products 25+ years of experience Eric Karas Thomas
B. Casale, M.D. Chief Commercial Officer Professor of Medicine & Pediatrics Led Narcan commercial ops at Chief, Allergy & Immunology Emergent/Adapt, and Auxilium specialty University of South Florida 25+ years of experience
Potential to Transform the Treatment of Type I Allergic Reactions
neffy : first potential "no needle, no injection" solution for Type I allergic reactions to address an unmet market need Registration program demonstrates comparable PK and PD, without risk of needle-related safety
concerns, fear and hesitation Rapid and statistically significant response on PD surrogates for efficacy (SBP, HR) observed even 1 minute after dosing with neffy vs. injection Significant opportunity to disrupt current epinephrine
injectables market Completed repeat dose NAC study requested by FDA in Sept 2023 CRL NDA on track to respond to FDA CRL by early Q2 2024, with FDA action date and potential US launch in H2 2024 Potential multi-billion-dollar
market driven by HCP and consumer preference and adoption NCE-like IP exclusivity potential until at least 2038 $228 million in cash and short-term investments as of 12/31/2023 with an anticipated $195 million at anticipated FDA
What We Will Cover Today Unmet need in type I allergic reactions
including anaphylaxis Dr. Jonathan Spergel neffy clinical profile, registrational studies Richard Lowenthal neffy benefit-risk for type I allergic reactions including anaphylaxis Dr. Thomas Casale US market opportunity and commercialization strategy
Unmet Need / Current Challenges Vast Majority of Type I Allergy Patients
Face Significant Limitations with Current Treatment Options NO TREATMENT REFUSAL OF DELAY IN USER ERROR PROBLEM AVAILABLE TREATMENT TREATMENT IN TREATMENT ONLY 10% - 20% of 1 ~40% - 60% of ~50% of patients carry ~25% - 60% do not 23% - 35% fail to
patients with active 2 7 (<20% carry two) , 1,3 5, 6 4 Rx use as indicated patients delay administer dose correctly EASIER AND MORE RELIABLE neffy NO NEEDLE SMALL CONSISTENT DOSING SOLUTIONS NO INJECTION 99.999% delivery of Fits
in your pocket; effective dose in can carry more than 1 Rapid administration 0% critical dosing errors reliability testing; no without a needle in registration ~10% of cases require inhalation required self-administration
study repeat doses of No risk of needle-related 2 1 24 month shelf-life at injuries; lacerations or epinephrine High bioavailability, low 2 room temperature, with cardiotoxic blood vessel mg dose of neffy achieves up to 3
months at high injections comparable PK without o temperatures (122 F) overexposure risk Less hesitation to dose 6 References: 1. Warren CM, et al. Ann Allergy Asthma Immunol. 2018. 2. Rooney E, et al. Poster Presentation at ACAAI 2022
(Louisville, KY). 3. Brooks C, et al. Ann Allergy Asthma Immunol. 2017. 4. El Turki A, et al. Emerg Med J. 2017. 5. Asthma and Allergy Foundation of American Patient Survey Report 2019. 6. Mehta GD, et al. Expert Rev Clin Immunol. 2023. 7. ARS
company estimates based on IQVIA data and references 1 through 6.
Significant Catalysts for neffy in 2024 o US NDA response to CRL by
early Q2 2024 FDA o PDUFA action date anticipated in H2 2024 o EMA decision (CHMP Opinion) expected by mid-2024 EMA o China NDA filing expected by YE 2024 o Japan NDA filing expected by YE 2024 o Planning in progress for filing in other major ex-US
regions including Canada o Expansion opportunities Positive data from Phase 2 chronic urticaria study reported in Feb 2024 Initiation of Phase 2b outpatient urticaria study 7
Type I Allergies & Unmet Needs Jonathan M. Spergel, M.D., Ph.D.
Professor of Pediatrics Chief of the Allergy Program Children's Hospital of Philadelphia
Type I Allergic Reactions: Systemic Hypersensitivity Reaction More than
500,000 ER visits ~40 Million people in each year due to systemic Type I allergic US with systemic Type I allergic 1 2 reactions , costing an average of $1600+ per visit reaction to allergens Other Type I allergy indications Significant
co-morbidities and symptomatic Caused by exposure to a specific allergen, (e.g. urticaria flares) impact on quality of life. most commonly food, venom, drugs References: 1. Carillo-Martin I, et al. J Allergy Clin Immunol Pract 2020. 2. BlueCross
BlueShield of America. Childhood Allergies in America 2018 9 Images reproduced with permission
Mucosal Respiratory Cardiac GI Anaphylaxis Diagnosis Criteria and
Symptoms 2-14 Symptoms (>2%) reported during US anaphylaxis events 0% 20% 40% 60% 80% 100% Urticaria or Angioedema Urticaria Flushing Pruritus Angioedema (e.g. face, lips, tongue or larynx) Intraoral Edema or Tongue Swelling Laryngeal Edema
Throat pruritus Orophyarngeal Edema Difficulty Breathing / Dyspnea Throat Tightness Wheezing Bronchospasm Hoarseness Cough Chest tightness / Chest pain Respiratory failure Stridor Nasal symptoms / rhinitis Tachycardia Presyncope / Dizziness /Light
headedness Hypotension Confusion/somnolence Loss of consciousness Dysphagia Vomiting / Emesis Diarrhea + Abdominal Pain Nausea Abdominal pain or cramping Diarrhea References: 1. Shaker MS, et al. J Allergy Clin Immunol. 2020. 2. Pistiner M, et al. J
Allergy Clin Immunol Pract. 2021. 3. Jalil M, et al. Abstract at AAAAI 2020 Virtual Meeting. 4. Gonzelez-Estrada A, et al. Ann Allergy Asthma Immunol. 10 2018. 5. Lee S, et al. J Allergy Clin Immunol. 2017. 6. Lee S, et al. J Allergy Clin Immunol
Pract. 2014. 7. Manivannan V, et al. Am J Emerg Med. 2014. 8. Wood RA, et al. J Allergy Clin Immunol 2014. 9. Walsh KE, et al. Pharmacoepidemiol Drug Saf 2013. 10. Decker WW, et al. J Allergy Clin Immunol. 2008. 11. Ross MP, et al. J Allergy Clin
Immunol. 2008. 12. Webb LM & Lieberman P. Ann Allergy Asthma Immunol. 2006. 13. Ditto AM, et al. Ann Allergy Asthma Immunol. 1996. 14. Rudders SA, et al. Pediatrics. 2010. Note that some publications do not specify angioedema symptom subtype.
Angioedema subtype frequency aggregated when reported.
Most frequently reported symptoms are difficulty breathing, angioedema
(face, lips, tongue, larynx) and urticaria (hives) References: 1. LoVerde D, et al. Chest. 2018. 2. Images reproduced with permission 11
Epinephrine: Well Known Mechanism of Action Adrenergic Pharmacological
Effect Clinical Effect Receptor of Epinephrine of Epinephrine Stabilizes mast cells and basophils - Reverses pathological histamine cascade Inhibits inflammatory mediators Increase in bronchial airflow 2
Relaxation of bronchial smooth muscles Increases blood to skeletal muscle Vasodilation in skeletal vasculature Increases blood pressure and heart rate Relieves hypotension and shock 1 Increases systolic
blood pressure Relieves hypotension and shock Causes blood vessel constriction 1 Relieves upper airway obstruction Decreases mucosal edema Receptor Sensitivity 12
Second Dose Demonstrates Similar Efficacy Between IM and Autoinjectors
(the only FDA approved products today) ~90% resolution on first dose 12% Analysis of 12 studies with 100% autoinjector ( 80% EpiPen) or 10% 9.3% 100% IM-needle-and-syringe use in 1-11 community or ED setting 8% Differences in
PK profile across 6% products do not impact efficacy based on need for repeat dosing to resolve symptoms 4% 2% 0% IM Needle-in-Syringe Autoinjector % r equiring second dose reactions reactions 11 n = 570 n = 799 References: 1. Patel N, et al. J
Allergy Clin Immunol. 2021. 2. Kahveci M, et al. Pediatr Allergy Immunol. 2020. 3. Oya S, et al. J Emerg Med. 2020. 4. Kondo A, et al. Air Med J. 2018. 5. Cardona V, et al. Int Arch Allergy 13 Immunol. 2017. 6. Arkwright PD. J Allergy Clin Immunol.
2008. 7. Gold MS & Sainsbury R. J Allergy Clin Immunol. 2020. 8. Noimark L, et al. Clin Exp Allergy. 2011. 9. Soller L, et al. J Allergy Clin Immunol Pract. 2019. 10. Webb LM & Lieberman P. Ann Allergy Asthma Immunol. 2006. 11. White MV, et
al. Allergy Ashm Proc. 2015; note that this publication reports data on 636 reactions treated exclusively with EpiPen Treated Reactions Requiring Second Epinephrine Dose (%)
Prompt Treatment with Epinephrine is Critical ANTIGEN EXPOSURE
Consequence of Delayed Treatment Risk Factor 1 Abnormal vitals (HR, SBP, Respiration) p<0.001 Patients / Caregivers wait 2 Repeat Epinephrine Doses OR = 5.0 up to 18 minutes 3 Hospitalization (500,000 ER visits / yr) HR = 4.0 to dose epinephrine
4 Biphasic anaphylaxis OR = 3.4 5 Fatality References: 1. Andrew E, et al. Prehosp Emerg Care. 2018. 2. Hochstadter E, et al. J Allergy Clin Immunol. 2016. 3. Fleming JT, et al. J Allergy Clin Immunol Pract. 2015. 4. Liu X, et al. J Allergy Clin
Immunol Pract. 2020. 5. Turner PJ, 14 et al. J Allergy Clin Immunol. 2017.
% dosed with epinephrine in b community setting (pre-ED) Only ~40% of
ER Anaphylaxis Patients are Dosed with Epinephrine Pre-Arrival in the Community Setting Median Year of Study Period 15 References: 1. Mehta GD, et al. Expert Rev Clin Immunol. 2023. Percentage Treated with Epinephrine
Delays in Treatment with Epinephrine are Principally Due to
Autoinjector Limitations and Accompanying Patient Reluctance ~40% of patients do not >40% of patients do not fill or refill 40% 2 1 40% administer epinephrine at all their epinephrine prescription >50% of parents are afraid or 55%-60%
don't consistently somewhat afraid to administer 50% 2-4 60% carry epinephrine 5 their child's epinephrine ~20% of patients Patients that Do Not ~70% of patients 20% 70% with Training Without Training 7 Administer Correctly References:
1. Data on File. ARS Pharmaceuticals. 2023. 2. Warren CM, et al. Ann Allergy Asthma Immunol. 2018. 3. Brooks C, et al. Ann Allergy Asthma Immunol. 2017. 4. Curtis C, et al. Ann Allergy Asthma Immunol. 2014. 5. Chad L, 16 et al. Eur J Allergy Clin
Immunol. 2013. 6. Sicherer SH, et al. Pediatrics. 2000. 7. El Turki A, et al. Emerg Med J. 2017.
Needle-Related Safety Risks & Use Errors Needle-related risks
defined in labeling for all autoinjectors Lacerations and bone injections IV bolus injection (blood vessel injections) - likely result in most serious AEs Accidental self-injection into extremity by patient or caregiver 1
~ 3,500 events per year reported Requires immediate medical attention (treatment in ER typical) 2 Injection site pain, infection and other reactions Wet injections (withdraw needle too quickly) and other dosing errors 3,4,5,6,7,8,9 User
errors and device malfunctions References: 1. Anshien M, et al. Am J Ther. 2019. 2. Guerlain S, et al. Ann Allergy Asthma Immunol. 2011. 3. El Turki A, et al. Emerg Med J. 2017. 4. Moss RB, et al. Ann Allergy Asthma Immunol. 2018. 5. 17 Sanofi.
Auvi-Q Recall. October 2015. 6. FDA. EpiPen and EpiPen Jr Recall. March 2017. 7. FDA. EpiPen auto-injector errors related to device malfunctions and user administration. March 2020. 8. FDA. Amneal and Impax Laboratories epinephrine auto-injector
device malfunctions. June 2020. 9. FDA. Recall of Symjepi (epinephrine) injection for potential manufacturing defect. March 2022.
2023 AAAAI guidelines updated so that EMS activation not required for
90% of events that resolve with single dose Historic guidelines recommended ED visit following use of epinephrine for anaphylaxis, which may result in families not giving epinephrine to avoid ED visits Based on outcomes of anaphylaxis in EDs and the
COVID-19 pandemic, data indicates treatment and monitoring of anaphylaxis can occur at home If signs and symptoms resolve within minutes of dosing, monitor at home after first dose If signs and symptoms improve within minutes of
dosing, monitor at home if comfortable, while considering EMS activation and possible second dose of epinephrine If signs and symptoms are not resolving, activate EMS immediately, and consider second dose of epinephrine Prompt use of
epinephrine and monitoring at home will decrease healthcare utilization 18
neffy (epinephrine nasal spray) Can Fill Great Unmet Medical Need for
Patients and Caregivers Epinephrine has a well-established efficacy and safety profile Efficacy same across epinephrine injection products despite PK differences Immediate administration of epinephrine is critical Patients and caregivers
reluctant to use or carry current devices Needle-phobia Concerns with safety Cumbersome to carry Unmet need for needle-free, easy to use, easy to carry, safe and effective epinephrine option neffy can fit that need for our
neffy Profile Richard Lowenthal CEO, ARS Pharma
neffy is a High Bioavailability, Low 2 mg Dose Saline-Based Nasal
Spray: Proven Triad of FDA-Approved Components High bioavailability with low dose minimizes side effects that mimic anaphylaxis (GI symptoms) > 9 FDA Approvals in Allergy and risk of cardiotoxicity from overdosing (> 100 years of clinical
experience) Epinephrine 7 FDA Approvals of Sprayer 1 (> 55 million Rx, 99.999% reliable) 3 FDA Approvals incl. Intravail 1 (> 1 million Rx) NARCAN VALTOCO NAYZILAM TOSYMRA VALTOCO Intravail Sprayer
ZAVZPRET dodecyl-maltoside: IMITREX TOSYMRA GRAS absorption enhancer OPVEE OPVEE 21 References: 1. IQVIA Prescription data, and ARS company data on file
Intravail Allows Injection-Like PK at a Low Intranasal Dose
Without Irritation or Pain, and Robust IP Protection to 2038+ NCE-like exclusivity enabled by Intravail No systemic absorption via nose without Intravail when epinephrine is put in water-based solution Intravail allows systemic
intranasal absorption of epinephrine within the known therapeutic dose window for n-Dodecyl beta-D-maltoside (Intravail ) injection products Generally recognized as safe (GRAS) absorption No inhalation required -