Full Press Release Details
ARS Corporate Presentation Q2 2024
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: the anticipated timing for regulatory review decisions on the neffy NDA and MAA; ARS Pharma's belief that neffy will be approved for the treatment of Type I allergic reactions; the timing for the potential
U.S. launch of neffy, if approved; 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 March 31, 2024, filed with the Securities and Exchange Commission ("SEC") on
May 9, 2024. 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.
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 Response to CRL including positive data submitted April 2, 2024 - up to 6 months FDA review; CHMP opinion in Europe by June 2024 Potential multi-billion-dollar market driven by HCP and consumer preference and adoption
NCE-like IP exclusivity potential until at least 2038 $223.6 million in cash and short-term investments as of 3/31/2023 with an anticipated >$200 million at anticipated FDA approval in H2 2024
Anaphylaxis is Accompanied by Many
Frequent Symptoms 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. 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. Symptoms (>2%) reported during US anaphylaxis events 2-14 Mucosal Respiratory Cardiac GI
urticaria (hives, erythema) or angioedema (swelling of the face, lips, tongue or larynx) >85% >55% gastrointestinal (eg, vomiting, nausea) Common Anaphylaxis Symptoms Include: difficult breathing >40%
Epinephrine: The First Line of Defense
Against Anaphylaxis Patients with Type 1 Severe Allergic Reactions are prescribed epinephrine to use at symptom onset Used for over 100 years Well-known mechanism of action, and only drug known to reverse a systemic allergic reaction
Well-established efficacy and safety profile Products approved based on pharmacologic properties, not clinical efficacy studies All approved products demonstrate efficacy (90% response on a single dose) despite different pharmacokinetic (PK)
properties Clinical studies are considered unethical/unfeasible All approved products are needle-based High unmet need for needle-free, easy-to-carry epinephrine remains Rapid, reliable delivery Small and easy to carry Place and Press administration
Well-tolerated in extensive trials neffy is the first potential "no needle, no injection" solution for Type I allergic reactions to address an unmet market need
Rapid administration without a needle
No risk of needle-related injuries; lacerations2 or cardiotoxic blood vessel injections Less hesitation to dose NO NEEDLE NO INJECTION Fits in your pocket; easy to carry the recommended 2 devices ~10% of cases require repeat doses of epinephrine1
EASIER AND MORE CONSISTENT DOSING 100% of untrained adults and children can dose neffy successfully7 High bioavailability, low 2 mg dose of neffy achieves comparable PK without overexposure risk including any side effects that mimic anaphylaxis
RELIABLE 99.999% delivery of effective dose in reliability testing; not obstructed by any anaphylaxis symptoms; no inhalation required 24-month shelf-life at room temperature, with up to 3 months at high temperatures (122oF) ~50% of patients carry1
(<20% carry two) ~25% - 60% do not administer, 1,3 5, 6 NO TREATMENT AVAILABLE REFUSAL OF TREATMENT ~40% - 60% of patients delay2 DELAY IN TREATMENT 23% - 35% fail to dose correctly4 USER ERROR IN TREATMENT SOLUTION: neffy Unmet Need / Current
Challenges Vast Majority of Type I Allergy Patients Face Significant Limitations with Current Treatment Options 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. 4. Data on file from neffy human factors studies. PROBLEM: ONLY 10% - 20% of patients with active Rx use as indicated7 SMALL
neffy Designed for Ease of Use and
Easy Carry For epinephrine to be effective, patients must: neffy has a simple place and press administration (no hold time) 100% of adults and children able to use neffy successfully without any training Relative Size of neffy two pack Compared to
iPhone 15 and EpiPen Regularly have their device on hand Not hesitate to dose immediately after symptom onset Have a second device on hand if needed (~10% of cases) Administer the device as intended Case holds two neffy 2mg devices 6"
Development Program Centers on
Pharmacology Studies Efficacy studies not ethical / feasible in severe Type I allergy patients neffy clinical studies developed in agreement with FDA and EMA to allow reference to historic efficacy and safety data of epinephrine injection Bracketed
pharmacokinetic (PK) exposures Comparable pharmacodynamic (PD) responses > 1,200 administrations of neffy in > 700 subjects Primary Studies (2 mg dose) Patient Population EPI 15 Adult: Healthy volunteer (HCP administration) - single and
repeat dosing EPI 16 Adult: Type 1 allergy patients (NAC induced rhinitis) - single dosing EPI 17 Adult: Type 1 allergy patients (self-administration) - single dosing EPI 18 Adult: Type 1 allergy patients (NAC induced rhinitis) -
repeat dosing EPI 10 Pediatric: Type 1 allergy patients: 30kg (NDA), 15 30kg (sNDA) - single dosing
Registrational studies demonstrate
comparability on both PD surrogates for efficacy and PK with neffy Safety Data 2 mg neffy met all clinical endpoints PD surrogates for efficacy comparable to approved products (SBP/HR approved injection products) Rapid and significant response on PD
surrogates for efficacy observed even 1 minute after dosing PK bracketed by approved products (exposures IM/SC for efficacy, < EpiPen for safety) Repeat doses (including during rhinitis) within range of approved injection products Adverse
events generally mild in nature with no meaningful nasal irritation or pain up to 4 mg dose Most common adverse events (>5%) were mild nasal discomfort (9.7%) and mild headache (6%), with no correlation of nasal discomfort to pain or irritation
Mean VAS pain scores between 5 to 8 out of 100 No irritation based on formal assessment No serious adverse events in any clinical study No risk of needle-related injuries or blood vessel injections with neffy PD and PK Data Response to FDA submitted
on April 2, 2024 followed by up to 6-month FDA review
Pharmacokinetic Results from neffy
2 mg Studies Satisfies Bracketing Approach agreed with by FDA Mean Epinephrine Concentration (pg/mL) Time (Minutes) neffy 2 mg (HCP) (n=78)* EpiPen 0.3 mg (n=77) SC 0.3 mg (n=35) neffy 2 mg (self-administration) (n=42) IM 0.3 mg (n=178) Upper
bracket for safety Lower bracket for efficacy *Includes Studies EPI-15 and EPI-16 FDA focused on PK properties to ensure efficacious and safe epinephrine exposures within range of approved products ("Bracketing") Minimum exposure must be
IM/SC (efficacy) Maximum exposures must be < EpiPen (safety) No difference in efficacy between all injection products 90% response to single dose irrespective of device
Second Dose Frequency Demonstrates
Similar Efficacy Between IM and Autoinjectors (the only FDA approved products today) Autoinjector reactions n = 79911 IM Needle-in-Syringe reactions n = 570 Treated Reactions Requiring Second Epinephrine Dose (%) ~90% resolution on first dose
Analysis of 12 studies with 100% autoinjector ( 80% EpiPen) or 100% IM-needle-and-syringe use in community or ED setting1-11 Differences in PK profile across products do not impact efficacy based on need for repeat dosing to resolve symptoms
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 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
Robust response on PD surrogate
markers for efficacy shows engagement of receptors that reverse anaphylaxis symptoms Mean Change HR (BPM) Time (Minutes) Heart Rate Response Mean Change SBP (mmHg) Time (Minutes) Systolic Blood Pressure Response First PD 1 minute First PD 1 minute
neffy 2 mg IM 0.3 mg neffy 2 mg IM 0.3 mg ** Significance level: ** p <0.01, *** p <0.001 **** p <0.0001 **** **** *** ** **** **** **** Epi 53 pg/mL (2 min PK) Epi 53 pg/mL (2 min PK) Integrated analysis of ARS clinical studies (EPI-15 and
Exposures of repeat doses of neffy
in healthy subjects are also in the range of FDA approved epinephrine injection products Geometric Mean Plasma Concentration (pg/mL) Time (mins) 1.65x Integrated data from ARS clinical studies, FDA Briefing Document May 2023 PADAC for NDA/BLA#
214697 (neffy) neffy exposure similar to EpiPen to ensure safety neffy exposure greater than IM to ensure efficacy Repeat-dosing (10 min apart) results in healthy subjects
PK/PD profile and ability to dose
may be influenced by anaphylaxis itself, so FDA asked ARS to evaluate rhinitis in clinical studies Anaphylaxis Symptom US % Intranasal Sublingual Oral* Inhalation* Nasal symptoms or rhinitis 4% X X Oropharyngeal edema 10% X X X Vomiting / Emesis 20%
X X X Dysphagia 23% X X Laryngeal Edema 24% X X Bronchospasm 24% X Intraoral Edema or Tongue Swelling 24% X X X Angioedema (e.g. face, lips, tongue or larynx) 45% X X X Difficulty Breathing / Dyspnea 55% X *insufficient oral and inhalation systemic
absorption due to rapid conjugation and oxidation in GI tract or difficulty taking in enough puffs14 References: 1. Pistiner M, et al. J Allergy Clin Immunol Pract. 2021. 2. Jalil M, et al. Abstract at AAAAI 2020 Virtual Meeting. 3. Gonzelez-Estrada
A, et al. Ann Allergy Asthma Immunol. 2018. 4. Lee S, et al. J Allergy Clin Immunol. 2017. 5. Lee S, et al. J Allergy Clin Immunol Pract. 2014. 6. Manivannan V, et al. Am J Emerg Med. 2014. 7. Wood RA, et al. J Allergy Clin Immunol 2014. 8. Walsh
KE, et al. Pharmacoepidemiol Drug Saf 2013. 9. Decker WW, et al. J Allergy Clin Immunol. 2008. 10. Ross MP, et al. J Allergy Clin Immunol. 2008. 11. Webb LM & Lieberman P. Ann Allergy Asthma Immunol. 2006. 12. Ditto AM, et al. Ann Allergy Asthma
Immunol. 1996. 13. Rudders SA, et al. Pediatrics. 2010.14. Simons KJ, et al. J Allergy Clin Immunol. 2004. Note that some publications do not specify angioedema symptom subtype. Angioedema subtype frequency aggregated when reported. Potential effect
on ability to dose or absorption profile by theoretical route of administration for epinephrine Intranasal formulation least impacted by anaphylaxis symptoms compared to alternate non-injectable routes Nasal symptoms or rhinitis only impact only 4%
of cases (analysis of 4,805 US anaphylaxis events)1-12 ARS successfully evaluated patients with rhinitis in response to FDA CRL, which responded positively to single and repeat doses of neffy
Experimental Nasal Allergen
Challenge (NAC)-Induced Rhinitis Does Not Negatively Impact neffy's PK Profile (allergic rhinitis subjects) NAC-induced rhinitis accelerates absorption of single dose neffy, but within the range of injection Repeat doses of neffy under
NAC-induced rhinitis supports similarity to injection for more severe cases of anaphylaxis Time to observe clinical response, and re-dose per guidelines * * * * * * neffy 2.0 mg (single) neffy 2.0 mg rhinitis (single) IM 0.3 mg (single) Mean
Epinephrine Concentration (pg/mL) * Statistically significant differences between neffy with rhinitis compared to IM injection (p <0.05) FDA Advisory Committee viewed single dose neffy NAC data as "encouraging" and
"favorable", but FDA's CRL requested, and ARS successfully completed repeat dose neffy NAC study Dosing 10 min apart Time to observe clinical response, and re-dose per guidelines
Experimental NAC-Induced Rhinitis
Does Not Negatively Impact neffy's PD Profile (Repeat Doses 10 min Apart) Mean Change in Systolic Blood Pressure (mmHg) Mean Change in Heart Rate (bpm) Response to FDA's CRL submitted April 2, 2024 followed by up to 6-month FDA
neffy on track for potential US
launch in H2 2024 with market exclusivity potential until at least 2038 Issued composition of matter patent (US10,576,156) on Intravail + epinephrine provides foundational exclusivity blocking any generic products. Method of treatment patents
(US11,173,209; US11,191,838) block other alkyl glycosides. Issued method of treatment patent (US10,682,414, US11,744,895, US11,717,571, US11,191,655) also blocks intranasal epinephrine product using a different technology using a low dose (<4 mg)
PCT patent granted in Europe (EP19751807), UK (GB2583051), Japan (JP6941224), Canada (3088909), Australia (AUS2019217643), Korea (10-2375232), China (2019800010042), with same claims as the US Extensive studies in the lab and clinic completed to
develop a proprietary product with expected NCE-like exclusivity EXPECTED LAUNCH FIRST PATENT EXPIRATION ADDITIONAL PROTECTION 2024 2038
Significant Opportunity to Address
Unmet Needs in Current US Severe Allergic Reaction Market Promotional Responsiveness ~20M diagnosed and under physician care over the last 3 years11 Epidemiology prevalence data estimates ~40M patients with type 1 allergic reactions2-10 ~50%
increase over market growth trend with consumer promotion (2010 to 20151) Consistent Market Growth (Units) +6.5% CAGR since 2010, +12.7% YoY in 20231 ~3.2M patients filled Rx in 2023, but ~80-90% do not use as indicated11 (1) do not carry (~50%),
(2) do not inject (25-60%), (3) wait to inject (40-60%) or (4) dose incorrectly (23-35%) References: 1. Based on IQVIA prescription data (~5.2 million two-packs sold in 2023) and weighted average generic/branded epinephrine auto-injector net