Ars Pharmaceuticals, Inc. · NASDAQ:SPRY
Ars Pharmaceuticals — Investor Presentation (March 2023)
41 slides from Ars Pharmaceuticals's investor presentation, Mar 13, 2023
Slide 1: The First No-Needle, No-Injection Solution for Type I Allergic Reactions
Exhibit 99.1 THE FIRST NO-NEEDLE, NO-INJECTION SOLUTION for Type I Allergic Reactions Q1 2023 1
Slide 2: Forward looking statements
Forward looking statements This presentation contains forward-looking statements which include, but are not limited to, statements regarding the design and potential benefits of neffy; the anticipated Prescription Drug User Fee Act (PDUFA) date for neffy; the timing of regulatory approval for and th
Slide 3: Potential to Transform the Treatment of Type I Allergic Reactions
Potential to Transform the Treatment of Type I Allergic Reactions • neffy®: first “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 hesita
Slide 4: Proven leadership team with track record developing and commercializing intranasal medicines
Proven leadership team with track record developing and commercializing intranasal and consumer-driven medicines Sarina Tanimoto, M.D. Richard Lowenthal, M.S. Eric Karas Chief Medical Officer, Co-Founder Chief Executive Officer, Co-Founder Chief Commercial Officer ® Led FDA approvals for Led FDA app
Slide 5: Top-tier board of directors
Top-tier board of directors Peter Kolchinsky, Ph.D. Rajeev Dadoo, Ph.D. Managing Partner and Founder Managing Partner at SR One at RA Capital Pratik Shah, Ph.D. Chairman of Board of Directors Executive Chairman at Design, Former Chairman of Synthorx (acq. $2.5B), Former CEO at Brent Saunders Michael
Slide 6: Type I allergic reactions: a life-threatening hypersensitivity reaction
Type I allergic reactions: a life-threatening hypersensitivity reaction Caused by exposure to a ~25 to 40 million people in US specific allergen, with systemic Type I allergic most commonly food, reaction to allergens venom, drugs (e.g., 2+ organ systems involved) 10+ million people with other Type
Slide 7: Epinephrine is effective, but significant device limitations exist
Epinephrine is effective, but significant device limitations exist Epinephrine recognized as the only first-line therapy by 1 allergy society treatment guidelines , but… Safety concerns: lacerations, Apprehension to dose due to caregiver self-injection, blood needle vessel hits Lack of portability L
Slide 8: Early intervention with epinephrine is critical in a Type I allergic reaction
Early intervention with epinephrine is critical in a Type I allergic reaction REACTION 2,3,4 SERIOUS PATIENT DISCOMFORT HIGHER RISK OF HOSPITALIZATION AND DISEASE PROGRESSION PROGRESSION 5 MINUTES 15 MINUTES 30 MINUTES TYPE I SEVERE LIKELIHOOD OF ANAPHYLAXIS ALLERGIC REACTION LIFE-THREATENING REACTI
Slide 9: Limitations of injection lead to hesitation and decreased or ineffective usage
Limitations of injection lead to hesitation and decreased or ineffective usage neffy may address these limitations to transform the treatment paradigm 1 2 3 4 PROBLEM NO TREATMENT REFUSAL OF FAILURE OF DELAY IN Only 10% - AVAILABLE TREATMENT TREATMENT TREATMENT 20% of Rx 1, 3, 5 2 4 ~50% of ~25% - 5
Slide 10: Approved injection products have a range of PK profiles, but are all deemed efficacious
Approved injection products have a range of PK profiles, but are all deemed efficacious (no known difference across products) Median or Study • FDA stated neffy should be Mean Study Mean T max TREATMENT Source N C bracketed by PK of approved products max Study T range (pg/mL) max (min) (min) • 0.3 m
Slide 11: neffy clinical program supports NDA filed and accepted by FDA
neffy clinical program supports NDA filed and accepted by FDA FDA confirmed three primary registration studies required for neffy approval EPI-15: Single dose and twice EPI-17: Self-administration in EPI-16: Nasal challenge in dosing in healthy volunteers (n=42) allergic rhinitis patients (n=36) Typ
Slide 12: neffy meets FDA-confirmed endpoints in primary studies
neffy meets FDA-confirmed endpoints in 3 primary studies* Integrated PK data summary for neffy and comparators Plasma Epinephrine C (pg/mL) Median t (minutes) Mean Early Partial AUCs max max 100 Mean early partial AUCs bracketed by approved products Cmax bracketed Tmax bracketed by approved pAUC pAU
Slide 13: neffy well-tolerated across 600+ individuals dosed in clinical program
neffy well-tolerated across 600+ individuals dosed in clinical program Risk of blood vessel injection during self- administration that could lead to adverse events • Well-tolerated at all single-doses (0.5 mg to 2 mg) and repeat doses up to 4 mg within 10 minutes 13.8% • Mostly grade 1 events and co
Slide 14: neffy market exclusivity potential until at least 2038
neffy market exclusivity potential until at least 2038 ® ✓ Issued composition of matter patent (US10,576,156) on Intravail + epinephrine Extensive studies in provides foundational exclusivity blocking any generic products. Method of treatment patents (US11,173,209; US11,191,838) block other alkyl gl
Slide 15: Commercial Opportunity and Strategy
Commercial Opportunity and Strategy 15
Slide 16: Significant existing US market opportunity for neffy penetration
Significant existing US market opportunity for neffy penetration 1 CURRENT ~$1 BILLION ANNUAL EPINEPHRINE MARKET IS THE IMMEDIATE OPPORTUNITY MULTIPLE LEVERS OF CURRENT MARKET GROWTH ~16M Consistent market growth diagnosed and HCP- +5% y/y in the last ~15 years managed patients with severe type I al
Slide 17: Physicians supportive of adopting neffy into practice
Physicians supportive of adopting neffy into practice 8.5 out of 10 rating viewed as a major advance in therapy 10 = MAJOR ADVANCE / 1 = NOT AN ADVANCE AT ALL n = 75 Physicians Would prescribe neffy 100% if their patients asked for it No difference in uptake of neffy by physician specialty 17
Slide 18: neffy addresses the unmet need and is better aligned with what HCPs, patients and parents want
neffy addresses the unmet need and is better aligned with what healthcare providers, patients and parents want OF PATIENTS EXPECTED TO ~80% SWITCH TO neffy n = 150 Current Users OF NON-FILLING PATIENTS STATED THEY WOULD ASK THEIR 75% PHYSICIAN ABOUT neffy RX n = 100 Non-fillers 65% to 72% 69% OF THE
Slide 19: Caregivers are enthusiastic about neffy and its benefits
Caregivers are enthusiastic about neffy and its benefits Time from Onset of Symptoms to Epinephrine Administration Current neffy I want this. Is it 9.3 This is fantastic. available yet? Let me 8.8 Much easier than know when it is, I will 8.3 jabbing the thigh. literally call the doctor from my car.
Slide 20: By Addressing Needle Injector Deficiencies neffy can Become the Standard in Treatment
By Addressing Needle Injector Deficiencies neffy can Become the Standard in Treatment Propensity of Current SAR Patients neffy Patient Research Shows… Prescribed a Needle Injector 45% 47% 1 Don’t Use Properly 42% 3 3 More Likely to Fill Rx Faster Treatment Time 2 50% Don’t Keep On Hand 100% 86% 4 3
Slide 21: Payer research supports positive reimbursement environment
Payer research supports positive reimbursement environment Key findings from discussions with ~50 decision-makers within the major payers and PBMs: • Category is generally not restricted, unlike biologics and orphan disease drugs with high WACs • Payers view neffy as a valuable and differentiated tr
Slide 22: Commercial strategy and imperatives
Commercial strategy and imperatives From needle to neffy: • Convert the existing market • Bring back patients that are lapsed • Bring in patients who should be carrying epinephrine now, but do not carry Ensure broad and rapid neffy coverage as well as affordable access for patients 1 Change HCP habi
Slide 23: Strategic Imperatives and CSFs: From Needle to neffy
Strategic Imperatives and CSFs: From Needle to neffy 2 3 1 Drive neffy Ensure broad and Change HCP habits awareness and new rapid neffy coverage as and switch prescribing patient growth (into and well as affordable from needle to neffy back into) the market access for patients • HCP awareness at lau
Slide 24: Integrated HCP Promotion to Drive Awareness and Reach
Integrated HCP Promotion to Drive Awareness and Reach with Current Epinephrine Prescribers Representing >40% of Prescriptions* Specialty Virtual Salesforce Non-Personal Promotion Salesforce FTEs ~125 FTEs Top 50,000 Decile 5 to 10 HCP ~ 15,000 HCPs Reach HCP promotion will be supported by DTC promot
Slide 25: neffy is positioned potentially to transform the treatment of serious allergic reactions
neffy is positioned potentially to transform the treatment of serious allergic reactions PK superior to IM No needle, no fear, no wait Less (Reference listed drug & Easier to use and more reliable gold standard) Effective Hesitation delivery than with autoinjectors offers therapeutic exposures to Do
Slide 26: Supplemental Slides
Supplemental Slides 26
Slide 27: Many patients/caregivers do not administer treatment or delay use during reaction
Many patients/caregivers do not administer treatment or delay use during reaction Approx. 40,000,000 people with serious Type I Allergic Reactions ~5,800,000 people received Rx from a Physician in Last 3 Years Did not fill or re-fill their 2.5 Rx in last 3 1 years 25-50% 5.2 5.8 ~50% do Patients ref
Slide 28: IM Needle & Syringe is the Historical Product Clinically Proven to Be Effective
IM Needle & Syringe is the Historical Product Clinically Proven to Be Effective: No Difference in Efficacy Between IM and EAIs • Meta-analysis of 88 studies with 36,557 anaphylaxis Autoinjector IM Needle-in-Syringe events w/ epinephrine use in 50.4% of events (approx. 10.9% 10% repeat dose needed) 9
Slide 29: History of FDA Approved Community Use Products
History of FDA Approved Community Use Products • No observed clinical differences between autoinjectors and IM injection (time to onset or efficacy) Device (Approved) Approval Basis Pharmacokinetics (any data including literature) EpiPen No PK Data Significant differences (EpiPen vs. IM) only known
Slide 30: neffy Development Approach Agreed with FDA
neffy Development Approach Agreed with FDA • neffy should demonstrate primary PK parameters C , t and pAUC max max 0- within the range of injection products 20/0-45 – AUC was secondary for safety only 0-t • IM injection (needle and syringe) is RLD for efficacy; EpiPen is the upper limit for safety (
Slide 31: Integrated Results neffy Studies
Integrated Results neffy 2 mg Studies (EPI 15 & EPI 16) 400 neffy 2.0 mg IN 350 Epinephrine 0.3 mg IM 300 EpiPen 0.3 mg 250 Mean Plasma 200 Concentration (pg/mL) 150 100 50 0 0 30 60 90 120 150 180 210 240 270 300 330 360 Time (Minutes) 31
Slide 32: neffy Single Dose Bracketed by Injection
neffy 2 mg Single Dose Bracketed by Injection Mean C Median T pAUC pAUC AUC max max 0-20 0-45 0-t (pg/mL) (minutes) (min*pg/mL) (min*pg/mL) (min*pg/mL) Product N (CV%) (range) Mean (CV%) Mean (CV%) Mean Epi 0.3 mg IM 178 277 (65) 45 (4-360) 2090 (86) 6290 (61) 27900 (39) Symjepi 0.3 mg 36 438 (65) 3
Slide 33: neffy PK is Also Bracketed by EpiPen Studies
neffy PK is Also Bracketed by EpiPen Studies Median Study T range Mean Study max Treatment Study Reference N Administration (pg/mL) (min) (min) C T max max EpiPen (0.3 mg) AQST-109 EPIPHAST II Results (2022) 22 869 22 5 to 40 HCP EpiPen (0.3 mg) ARS EPI-JP01 Data (2020) 30 676 10 2 to 45 HCP EpiPen
Slide 34: Pediatric neffy Pharmacokinetics (EPI-10 Interim Analysis)
Pediatric neffy Pharmacokinetics (EPI-10 Interim Analysis) Mean C Median T pAUC pAUC AUC max max 0-20 0-45 0-t (pg/mL) (minutes) (min*pg/mL) (min*pg/mL) (min*pg/mL) Product N (CV%) (range) Mean (CV%) Mean (CV%) Mean neffy (1.0 mg) 26 253 (66) 20 (7.5-120) 2,570 (78) 5,960 (52) 14,000 (53) Children 3
Slide 35: neffy Twice Dosing – Two Doses Minutes Apart
neffy 2 mg Twice Dosing – Two Doses 10 Minutes Apart Mean C Median t max max Treatment N (pg/mL) (%CV) (min) (range) 800 neffy 2.0 mg IN twice (L/R) neffy 2.0 mg 39 1000 (93) 30 (6 - 150) neffy 2.0 mg IN twice (R/R) 700 twice (L/R) Epinephrine 0.3 mg IM twice (L/R) neffy 2.0 mg 39 992 (75) 30 (4 - 1
Slide 36: neffy integrated PK is dose-proportional based on mean Cmax
neffy integrated PK is dose-proportional based on mean Cmax neffy 1mg 276 neffy 1mg (L/R) 474 Single x 2 is the theoretical dose neffy 1mg (L/L) proportional outcome for comparison 544 Single x2 552 neffy 2mg 485 neffy 2mg (L/R) 1000 neffy 2mg (R/R) 992 Single x2 970 IM 277 IM (L/R) 436 Single x2 55
Slide 37: Mean SBP Response from a Single neffy Dose Comparable to Injection Products
Mean SBP Response from a Single neffy 2 mg is Comparable to or Better than Injection Products • SBP response from intranasal administration is more consistent with mean effect greater than injection products SBP 20 neffy 2.0 mg IN Epinephrine 0.3 mg IM EpiPen 0.3 mg 15 10 5 0 0 20 40 60 80 100 120 -
Slide 38: Mean DBP Change for a Single neffy Dose Demonstrates Less Early Decrease
Mean DBP Change for a Single 2 mg neffy Demonstrates Less Early Decrease than Injection Products • Initial greater decrease in DBP after IM administration suppresses SBP and is not desirable in patients with hypotension. Direct to systemic routes of administration result in less decrease in DBP. DBP
Slide 39: Mean HR Response from a Single neffy Dose Comparable to Injection Products
Mean HR Response from a Single 2 mg neffy is Comparable to or Better than Injection Products • HR response from intranasal administration is more consistent with mean effect greater than injection products HR neffy 2.0 mg IN 14 Epinephrine 0.3 mg IM EpiPen 0.3 mg 12 10 8 6 4 2 0 0 20 40 60 80 100 12
Slide 40: neffy PK greater than 0.3 mg IM during Nasal Allergen Challenge
neffy PK greater than 0.3 mg IM during Nasal Allergen Challenge (Worse Case Nasal Condition: EPI-16 study) AUC last C (pg/mL) *significant difference (p<0.05) neffy vs. IM t (min) max max (min*pg/mL) Treatment N median 300 (range) mean (%CV) mean (%CV) 250 ARS-1 2.0 mg IN neffy 2.0 mg 36 20 (2-120)
Slide 41: neffy Early Partial Exposures greater than 0.3 mg IM after Nasal Allergen Challenge
neffy Early Partial Exposures greater than 0.3 mg IM injection after Nasal Allergen Challenge Rhinitis Induction (EPI-16 study) pAUC pAUC pAUC pAUC pAUC 0-10 0-15 0-20 0-30 0-45 (min*pg/mL) (min*pg/mL) (min*pg/mL) (min*pg/mL) (min*pg/mL) Product N Mean (CV%) Mean (CV%) Mean (CV%) Mean (CV%) Mean Epi
ARS Pharmaceuticals — Investor Presentation (March 2023) on 6ix