Pharvaris N.v. · NASDAQ:PHVS
Pharvaris — Investor Presentation (January 2025)
113 slides from Pharvaris's investor presentation, Jan 13, 2025
Slide 1: Pioneering science for patient choice – Corporate Presentation January 2025
Pioneering science for patient choice Corporate Presentation January 2025 Exhibit 99.2
Slide 2: Disclaimer
Disclaimer This Presentation contains certain “forward‐looking statements” within the meaning of the federal securities laws that involve substantial risks and uncertainties. All statements contained in this Presentation that do not relate to matters of historical fact should be considered forward-l
Slide 3: Deucrictibant: pioneering science for patient choice for HAE
FDA orphan drug designation1 Robust IP on CoM (granted in multiple territories, initial term to 2038) and formulations2,3 DEUCRICTIBANT Pioneering science for patient choice for hereditary angioedema (HAE) Source: 1U.S. FDA OOPD listing. 2World Intellectual Property Organization. 3European Patent Of
Slide 4: HAE: A rare, life-long genetic condition with significant burden
Median: 14 attacks/year Females: 19 (range: 2-165 attacks/y) Males: 9 (range: 1-42 attacks/y) HAE: A rare, life-long genetic condition with significant burden HAE: hereditary angioedema Source: 1Betschel SD, et al. J Allergy Clin Immunol Pract. 2023. 2Christiansen SC, et al. Ann Allergy Asthma Immun
Slide 5: The HAE market is dynamic, with people actively seeking a better product
The HAE market is dynamic, with people actively seeking a better* product HAE: hereditary angioedema. *Treatment selection is driven by physicians and patient preference. Source: 1Evaluate Pharma uptake curves 2008-2023 2SEC filings (BioCryst, CSL Behring, Pharming, Takeda). 3U.S. Chart Audit 2023-2
Slide 6: Significant growth expected in U.S. LTP and ODT market over next decade
In the U.S., significant growth in the long-term prophylaxis (LTP) and on-demand therapy (ODT) market is expected over the next decade1 Source: 1IQVIA market evolution and company data. 2Evaluate Pharma uptake curves 2008-2023. 3SEC filings (BioCryst, CSL Behring, Pharming, Takeda). This presentatio
Slide 7: People living with HAE are seeking a life not defined by their condition
People living with HAE are seeking a life not defined by their condition nor burdened by its management1 Source: 1Lumry WR et al. Allergy Asthma Proc. 2020. 2Geba et al, J Drug Access.2021. 3U.S. Chart Audit 2023-2024, ADIVO. This presentation includes data for an investigational product not yet app
Slide 8: Bradykinin B2 receptor antagonism is a foundational mechanism
Bradykinin B2 receptor antagonism is a foundational mechanism to treat and prevent bradykinin-mediated angioedema attacks1,2 Source: 1Maurer M, et al. Allergy. 2022. 2Zuraw BL World Allergy Orphan J. 2010. 3Lumry WR et al. Allergy Asthma Proc. 2020. 4Riedl MA et al. AAAAI 2024. 5Maurer M et al. AAAA
Slide 9: Deucrictibant has the potential to address unmet needs of people living with HAE
Deucrictibant has the potential to address unmet needs of people living with HAE HAE: hereditary angioedema. *To be confirmed with clinical data from Phase 3 studies Source: 1Company data: single-dose cross-over PK study in healthy volunteers (n=14) under fasting conditions. 2Lesage A et al. IDDST 2
Slide 10: Deucrictibant differentiated profile for LTP and ODT
Deucrictibant differentiated profile for LTP and ODT LTP: long-term prophylaxis. ODT: on-demand therapy. XR: extended-release tablet formulation of deucrictibant. IR: immediate-release capsule formulation of deucrictibant. Sources: 1Company research. 2Leasge et al. IDDST 2024. 3Groen K et al. ACAAI
Slide 11: Wholly-owned pipeline focused on bradykinin B2 receptor mechanism
Wholly-owned pipeline focused on bradykinin B2 receptor mechanism HAE: hereditary angioedema. AAE: acquired angioedema. OLE: open-label extension This presentation includes data for an investigational product not yet approved by regulatory authorities. MOLECULE INDICATION deucrictibant HAE On-Demand
Slide 12: Deucrictibant clinical development program
On-Demand Treatment (ODT) Long-Term Prophylaxis (LTP) Deucrictibant clinical development program OLE: open-label extension. LTE: long-term extension. Source: 1NCT05047185. 2NCT06669754. 3NCT06679881. 4NCT04618211. 5NCT05396105. 6NCT06343779. Phase 24 Complete Phase 2/3 LTE5 Ongoing Phase 3 pivotal6
Slide 13: Deucrictibant shows the potential to address unmet needs of people living with HAE
Deucrictibant shows the potential to address unmet needs of people living with HAE HAE: hereditary angioedema. OLE: open-label extension. LTE: long-term extension. PGI-C: patient global impression of change. PGI-S: patient global impression of severity. Source: 1Riedl MA et al. BKS 2024. 2Valerieva
Slide 14: Long-Term Prophylaxis
Long-Term Prophylaxis Deucrictibant extended-release tablets
Slide 15: Deucrictibant clinical development for LTP and ODT
On-Demand Treatment (ODT) Long-Term Prophylaxis (LTP) Deucrictibant clinical development for LTP and ODT OLE: open-label extension. LTE: long-term extension. Source: 1NCT05047185. 2NCT06669754. 3NCT06679881. 4NCT04618211. 5NCT05396105. 6NCT06343779. Phase 24 Complete Phase 2/3 LTE5 Ongoing Phase 3 p
Slide 16: Positive topline data from CHAPTER-1 Phase 2 study
Positive topline data from CHAPTER-1, a Phase 2 study of deucrictibant the for prophylaxis of HAE attacks *40 mg/day deucrictibant treatment group; %reduction in monthly attack rate is based on a Poisson regression model. All attacks reported herein are investigator-confirmed; attack rate is number
Slide 17: Continuing treatment sustained attack reduction for over one year
Continuing deucrictibant treatment sustained the early-onset attack reduction for over one year IR, immediate release; OLE, open label extension; RCT, randomized controlled trial. N = number of participants randomized in each treatment group in Part 1 of the study. N’ = number of participants in the
Slide 18: Efficacy and tolerability profile addressing unmet needs in prophylaxis
Deucrictibant efficacy and tolerability profile could address unmet needs in the prophylactic setting, with the convenience of a daily tablet § Crossover, 12 weeks/treatment. §§ Crossover, 16 weeks/treatment (results reported for weeks 3-16 for each treatment arm). † Parallel-arms, 26 weeks. †† Para
Slide 19: Commercial XR formulation maintains exposure above therapeutic level for 24 hours
Commercial XR formulation maintains exposure above therapeutic level for at least 24 hours Extended-release matrix controls release and absorption of compound in small intestine as well as in colon Supports once-daily dosing while maintaining exposure more consistently versus twice-daily IR (used in
Slide 20: CHAPTER-3 RCT and CHAPTER-4 OLE
CHAPTER-3 RCT and CHAPTER-4 OLE RCT: randomized clinical trial. OLE: open-label extension. HAE: hereditary angioedema. XR: extended-release tablet. This presentation includes data for an investigational product not yet approved by regulatory authorities. Two-part, global Phase 3 study of deucrictiba
Slide 21: On-Demand
On-Demand Deucrictibant immediate-release capsules
Slide 22: Deucrictibant clinical development for LTP and ODT
On-Demand Treatment (ODT) Long-Term Prophylaxis (LTP) Deucrictibant clinical development for LTP and ODT OLE: open-label extension. LTE: long-term extension. Source: 1NCT05047185. 2NCT06669754. 3NCT06679881. 4NCT04618211. 5NCT05396105. 6NCT06343779. Phase 24 Complete Phase 2/3 LTE5 Ongoing Phase 3 p
Slide 23: RAPIDe-1, a Phase 2 on-demand study of deucrictibant in HAE
RAPIDe-1, a Phase 2 on-demand study of deucrictibant in HAE HAE: hereditary angioedema. IR: immediate-release. AMRA, Angioedema Symptom Rating Scale. *pooled 10, 20, 30 mg deucrictibant treatment group †based on post-hoc analysis. Source: 1Maurer M et al. AAAAI 2023. 2 Riedl MA et al. ACAAI 2023. Th
Slide 24: RAPIDe-2, a long-term extension of RAPIDe-1
RAPIDe-2*, a long-term extension of RAPIDe-1 *A total of 265 attacks from 17 participants were included in the modified intention-to-treat efficacy analysis set (data cutoff: 1 March 2024); a total of 337 attacks from 19 participants were included in the safety analysis set (data cutoff: 10 June 202
Slide 25: Deucrictibant's rapid onset and complete symptom resolution vs standard of care
Plasma-derived C1NH (23%) - Icatibant (60%) – Recombinant hC1INH (9%) - Other (9%) Deucrictibant’s rapid-onset and complete symptom resolution may address unmet medical need in HAE with a single oral capsule References on following slide This presentation includes data for an investigational product
Slide 26: ODT comparison data references
ODT comparison data references * Non-laryngeal and laryngeal attacks included for treatment with study drug. # Symptom resolution assessed by PGI-S. † Symptom resolution assessed by VAS/AMRA. ‖ Symptom resolution assessed by TOS. a. Time to onset of symptom relief by VAS/AMRA defined as ‘VAS-3 ≥30%
Slide 27: RAPIDe-3 Phase 3 study for on-demand treatment
RAPIDe-31 RCT RCT: randomized clinical trial. *Adolescent patients receive a non-attack dose for PK sampling prior to randomization. Source: 1Maurer M et al. EAACI 2024. 20 mg capsule This presentation includes data for an investigational product not yet approved by regulatory authorities. Global Ph
Slide 28: Clinical trial endpoints span the entire attack timecourse
Symptom evolution towards relief and resolution Symptom progression Clinical trial endpoints span the entire attack timecourse AMRA, Angioedema Symptom Rating Scale; EoP, end of progression; HAE, hereditary angioedema; IR, immediate release; PGI-C, Patient Global Impression of Change; PGI-S, Patient
Slide 29: Patient-reported outcomes (PRO) assessments
Patient-reported outcomes (PRO) assessments Source: Riedl MA et al. BKS 2024. This presentation includes data for an investigational product not yet approved by regulatory authorities. PGI-C PGI-S AMRA Much worse Worse A little worse Same A little better Better Much better Very severe Severe Moderat
Slide 30: Acquired Angioedema
Acquired Angioedema PHARVARiS ©2025 30
Slide 31: Bradykinin B2 receptor inhibition broadly applicable across angioedema
Bradykinin B2 receptor inhibition broadly applicable across angioedema Notes: bold = known or potential role for bradykinin involvement in disease. †also designated as Normal C1INH Angioedema (HAE-nC1INH) HMWK: high-molecular-weight kininogen; cHMWK: cleaved high-molecular-weight kininogen; FXII(a):
Slide 32: Deucrictibant proof-of-concept in AAE-C1INH
Deucrictibant proof-of-concept in acquired angioedema due to C1-INH deficiency (AAE-C1INH)1,2 Source: 1Petersen RS et al. J Allergy Clin Immunol. 2024. 2Petersen RS et al. BKS 2024. This presentation includes data for an investigational product not yet approved by regulatory authorities. POP-AID ONC
Slide 33: Deucrictibant XR tablet for prevention of AAE-C1INH attacks
Deucrictibant XR tablet for the prevention of acquired angioedema (AAE-C1INH) attacks1,2 Attacks before and during deucrictibant XR treatment Notes: the baseline attack rate covers 90 days prior to randomization for prophylactic treatment in the randomized controlled trial for Patients 1,2, and 3, a
Slide 34: Our aspiration is to become a market leader in HAE
Our aspiration is to become a market leader in HAE Notes: Aspirational, to be confirmed with Phase 3 clinical data This presentation includes data for an investigational product not yet approved by regulatory authorities. 1 2 4 3 Win the oral LTP market Become preferred LTP Leverage portfolio with B
Slide 35: www.pharvaris.com
www.pharvaris.com NASDAQ: PHVS
Slide 36: Appendix
Appendix
Slide 37: Only compound with potential for injection-like efficacy orally across LTP and ODT
Deucrictibant: Only compound today1 with the potential to deliver injection-like efficacy orally across both LTP and ODT *To be confirmed with clinical data from Phase 3 studies. **Patient preference varies. Source: 1Company research. 2Company data: target threshold exceeded on first day in single-d
Slide 38: People living with HAE are seeking better therapies
People living with HAE are seeking: Source: Proprietary Pharvaris research, 2022 (representative sample of patients, n = 103, and doctors, n = 100) This presentation includes data for an investigational product not yet approved by regulatory authorities. Highly effective, well-tolerated and less bur
Slide 39: Ambition to realize deucrictibant potential across bradykinin-mediated conditions
Pharvaris has the ambition to realize the potential of deucrictibant to become a preferred option for bradykinin-mediated conditions Source: 1Riedl MA et al. BKS 2024. 2Maurer M et al. BKS 2024. 3Company research. 4Petersen RS et al. J Allergy Clin Immunol. 2024. 5Lesage et al. Int. Immunopharmacolo
Slide 40: Leveraging B2R expertise beyond HAE
Rare allergy & immunology conditions (commercial) BK-mediated diseases (R&D) BK-mediated angioedema: nC1, AAE Pharvaris aspires to leverage its foundational B2R expertise to develop therapies for conditions beyond HAE HAE Type 1/2 This presentation includes data for an investigational product not ye
Slide 41: Clinical dosing guided by bradykinin challenge model
Clinical dosing is guided by prediction from a validated in vivo surrogate-marker model, the bradykinin challenge Notes: BK: bradykinin; NHP: non-human primates; SQ: sub-cutaneous; EC85: effective concentration achieving 85% inhibition of bradykinin effect Source: 1FDA Clinical Pharmacology and Biol
Slide 42: CHAPTER-1 Randomized Clinical Trial Topline Data
CHAPTER-1 Randomized Clinical Trial (RCT) Topline Data Long-term Prophylaxis Data
Slide 43: CHAPTER-1 study design
CHAPTER-1 HAE, hereditary angioedema; IR, immediate release; R, randomization. *Deucrictibant IR capsule, 10 mg twice daily; †Deucrictibant IR capsule, 20 mg twice daily; c1 month = 4 weeks. CHAPTER-1 is a Pharvaris-sponsored clinical trial. Source: NCT05047185 This presentation includes data for an
Slide 44: CHAPTER-1 (RCT): Participant disposition
CHAPTER-1 (RCT): Participant disposition aDeucrictibant IR capsule, 10 mg twice daily. bDeucrictibant IR capsule, 20 mg twice daily. Source: Aygoren-Pursun E et al. EAACI 2024. This presentation includes data for an investigational product not yet approved by regulatory authorities. All screened (n=
Slide 45: Balanced demographics and baseline characteristics
Balanced demographics and baseline characteristics BMI, body mass index; HAE, hereditary angioedema; IR, immediate-release; N = number of randomized participants. aDeucrictibant IR capsule, 10 mg twice daily. bDeucrictibant IR capsule, 20 mg twice daily. c1 month = 4 weeks. Source: Aygoren-Pursun E
Slide 46: Primary endpoint met: significantly reduced attack rate
Primary endpoint met: deucrictibant significantly reduced attack rate BL, baseline; IR, immediate-release; LS, least squares. N = number of randomized participants. Model-based inferences are based on a Poisson regression model adjusted for baseline attack rate and time on treatment. No multiplicity
Slide 47: Significant attack reduction and no severe attacks
Significant attack reduction and no severe attacks with deucrictibant *20 mg/day = deucrictibant immediate release (IR) capsules 10 mg twice daily; †40 mg/day = deucrictibant IR capsules 20 mg twice daily. Source: CHAPTER-1 Topline Data Presentation. December 2023. This presentation includes data fo
Slide 48: Moderate or severe attack rate reduction
Primary endpoint met: deucrictibant significantly reduced attack rate BL, baseline; IR, immediate-release; LS, least squares. N = number of randomized participants. Model-based inferences are based on a Poisson regression model adjusted for baseline attack rate and time on treatment. No multiplicity
Slide 49: Reduction in attacks treated with ODT
Primary endpoint met: deucrictibant significantly reduced attack rate BL, baseline; IR, immediate-release; LS, least squares. N = number of randomized participants. Model-based inferences are based on a Poisson regression model adjusted for baseline attack rate and time on treatment. No multiplicity
Slide 50: Substantial attack rate reduction from baseline
Deucrictibant substantially reduced attack rate from baseline IR, immediate release. N = Participants with ≥4 weeks of treatment. aDeucrictibant IR capsule, 10 mg twice daily. bDeucrictibant IR capsule, 20 mg twice daily. Source: Wedner HJ et al. ACAAI 2024. This presentation includes data for an in
Slide 51: Reduced days with symptoms regardless of baseline attack rate
Deucrictibant reduced the proportion of days with symptoms, and the monthly attack rate, regardless of baseline attack rate IR, immediate-release. N = number of randomized participants with ≥4 weeks of treatment. aBased on time normalized number of attacks per 4 weeks. bDeucrictibant IR capsule, 10
Slide 52: Unmet need for additional HAE therapies improving disease control and HRQoL
Unmet need for additional HAE therapies that improve disease control and HRQoL Source: 1Maurer M et al. Allergy. 2022. 2Bork K, et al. Allergy Asthma Clin Immunol. 2021. 3Bygum A, et al. Front Med. 2017. 4Mendivil J, et al. Orphanet J Rare Dis. 2021. 5Chong-Neto HJ. World Allergy Organ J. 2023. 6Lu
Slide 53: Measuring disease control, HRQoL, and treatment satisfaction
Measuring disease control, HRQoL, and treatment satisfaction HAE, hereditary angioedema; HRQoL, health-related quality of life. Source: 1Weller K, et al. Allergy. 2020. 2Weller K, et al. J Allergy Clin Immunol Pract. 2020. 3Weller K, et al. Allergy. 2012. 4Weller K, et al. Allergy. 2016. 5Vanya M, e
Slide 54: Improvements in disease control and quality of life paralleled attack reduction
Improvements in disease control and health-related quality of life paralleled attack reduction during deucrictibant treatment1,2 AE-QoL, Angioedema Quality of Life Questionnaire; 4-week AECT, Angioedema Control Test (4-week recall period); IR, immediate-release; RCT, randomized controlled trial. N =
Slide 55: Greater patient satisfaction versus placebo (TSQM)
Deucrictibant shows greater patient satisfaction versus placebo across effectiveness and global satisfaction (TSQM instrument) IR, immediate release; TSQM, Treatment Satisfaction Questionnaire for Medication. N′ = number of participants with TSQM results at week 12. aDeucrictibant IR capsule, 10 mg
Slide 56: AE-QoL: HRQoL improved across all domains
AE-QoL: HRQoL improved across all domains AE-QoL, Angioedema Quality of Life Questionnaire; IR, immediate-release; HRQoL, health-related quality of life. N = number of randomized participants with AE-QoL data at baseline. N′ = number of participants with AE-QoL data at week 12. aDeucrictibant IR cap
Slide 57: AE-QoL: Total score improved from baseline
AE-QoL: Total score improved from baseline by week 4 and throughout treatment AE-QoL, Angioedema Quality of Life Questionnaire; IR, immediate-release. N = number of participants with AE-QoL data at week 12. aDeucrictibant IR capsule, 10 mg twice daily. bDeucrictibant IR capsule, 20 mg twice daily. S
Slide 58: TSQM: Greater satisfaction with effectiveness
TSQM: Greater patient satisfaction with effectiveness vs placebo IR, immediate-release; TSQM, Treatment Satisfaction Questionnaire for Medication; XR, extended release. N = number of participants with TSQM results at week 12. aDeucrictibant IR capsule, 10 mg twice daily. bDeucrictibant IR capsule, 2
Slide 59: TSQM: Greater overall satisfaction
TSQM: Greater overall patient satisfaction vs placebo IR, immediate-release; TSQM, Treatment Satisfaction Questionnaire for Medication. N = number of participants with TSQM results at week 12. aDeucrictibant IR capsule, 10 mg twice daily. bDeucrictibant IR capsule, 20 mg twice daily. Source: Valerie
Slide 60: Deucrictibant was well tolerated at both doses
Deucrictibant was well tolerated at both doses GGT, gamma-glutamyltransferase; IR, immediate-release; TEAE, treatment-emergent adverse event. N = number of participants who received at least one dose of blinded study treatment. aDeucrictibant IR capsule, 10 mg twice daily. bDeucrictibant IR capsule,
Slide 61: CHAPTER-1 Part 2: open-label extension
CHAPTER-1 Part 2: open-label extension Long-term Prophylaxis Data
Slide 62: CHAPTER-1: Two-part Phase 2 study design
CHAPTER-1: Two-part, Phase 2 study of deucrictibant for long-term prophylaxis of HAE attacks HAE, hereditary angioedema; OLE, open-label extension; IR, immediate-release; R, randomization; RCT, randomized controlled trial. aDeucrictibant IR capsule, 10 mg twice daily. bDeucrictibant IR capsule, 20 m
Slide 63: All participants who completed the RCT entered the OLE
CHAPTER-1: All participants who completed the RCT entered the OLE FDA, Food and Drug Administration; IR, immediate-release; OLE, open label extension; RCT, randomized controlled trial. n = number of participants. aDeucrictibant IR capsule, 10 mg twice daily. bDeucrictibant IR capsule, 20 mg twice da
Slide 64: Balanced demographics and baseline characteristics
Balanced demographics and baseline characteristics BMI, body mass index; HAE, hereditary angioedema; IR, immediate-release. N = number of randomized participants; RCT, randomized controlled trial. aDeucrictibant IR capsule, 10 mg twice daily. bDeucrictibant IR capsule, 20 mg twice daily. c1 month =
Slide 65: Well-tolerated with no new safety signals
Deucrictibant was well-tolerated with no new safety signals Includes participants who received at least one dose of blinded study treatment in the OLE by the cutoff date of 10 June 2024. aDeucrictibant IR capsule, 20 mg twice daily. bDeucrictibant IR capsule, 10 mg twice daily. ECG: electrocardiogra
Slide 66: Reduced attack rate in the RCT remained low in the OLE
Reduced attack rate in the RCT remained low in the OLE IR, immediate release; OLE, open-label extension; RCT, randomized controlled trial; SEM, standard error of the mean. (n) = number of patients analyzed at each timepoint. LS mean estimates of attack rate are based on Poisson regression models adj
Slide 67: Sustained early-onset attack reduction for over one year
Continuing deucrictibant treatment sustained the early-onset attack reduction for over one year IR, immediate release; OLE, open-label extension; RCT, randomized controlled trial; SEM, standard error of the mean. (n) = number of patients analyzed at each timepoint. LS mean estimates of attack rate a
Slide 68: Moderate and severe attacks remained low in OLE
Occurrence of moderate and severe attacks remained low in the OLE treatment period IR, immediate release; LS, least squares; OLE, open-label extension; RCT, randomized controlled trial. N = number of participants randomized in each treatment group in the RCT. N’ = number of participants in the OLE.
Slide 69: Less than one attack per year treated with rescue medication
On average less than one attack per year per participant was treated with rescue medication IR, immediate release; LS, least squares; OLE, open-label extension; RCT, randomized controlled trial. N = number of participants randomized in each treatment group in the RCT. N’ = number of participants in
Slide 70: On-Demand Program
On-Demand Program PHARVARiS ©2025 70
Slide 71: VAS renamed AMRA, reflecting evolution to electronic assessment
We have renamed VAS to AMRA, reflecting its evolution from a paper-based to electronic attack assessment1 What is a Visual Analogue Scale (VAS)? Simple, reproducible, paper-based tool to allow patient self-assessment of symptom severity Analog scale with an ‘X’ hand-marked to reflect severity of att
Slide 72: Real-world time to symptom relief: PGI-C vs AMRA-3
In a real-world study using standard-of-care therapy, median time to symptom relief for PGI-C “a little better” is 0.8 h faster than AMRA-3 ≥30% reduction Source: Mendivil et al., UCARE 2023. PRO instrument Events (n) Median time to, h (95% CI) PGI-C “a little better” 90 2.147 (1.518, 3.017) AMRA-3
Slide 73: RAPIDe-1 Randomized Clinical Trial Topline Data
RAPIDe-1 Randomized Clinical Trial (RCT) Topline Data On-demand Data
Slide 74: RAPIDe-1 study design
RAPIDe-1 HAE: hereditary angioedema; IR, immediate-release capsule formulation of deucrictibant. VAS: visual analog scale. Source: NCT04618211. This presentation includes data for an investigational product not yet approved by regulatory authorities. Phase 2 study of on-demand treatment of attacks i
Slide 75: Baseline characteristics
Baseline characteristics BMI, body mass index; HAE, hereditary angioedema; IR, immediate release; mITT, modified intent-to-treat; VAS, visual analog scale. Source: Misra L, et al. Indian J Anaesth. 2016. This presentation includes data for an investigational product not yet approved by regulatory au
Slide 76: PK analysis confirmed rapid absorption on oral dosing
PK analysis in HAE patients confirmed rapid absorption on oral dosing, consistent with Phase 1 healthy volunteer studies HAE, hereditary angioedema; PK, pharmacokinetic. Source: Jacobs JS, et al. WSAAI 2024. This presentation includes data for an investigational product not yet approved by regulator
Slide 77: Primary endpoint: significantly reduced attack symptoms by VAS-3 at 4h
Primary endpoint: deucrictibant IR significantly reduced attack symptoms by VAS-3 at 4h *VAS assessed every 30 minutes up to 4 hours post-treatment, then at 5, 6, 8, 24, 48 hours; †Nominal p-value. Note: Attacks in mITT Analysis Set refer to attacks treated with blinded study drug that had non-missi
Slide 78: Significantly shortened time to onset of symptom relief
Deucrictibant IR significantly shortened time to onset of symptom relief (30% reduction in VAS-3) *VAS assessed every 30 minutes up to 4 hours post-treatment, then at 5, 6, 8, 24, 48 hours; †Nominal p-value. Note: N = The number of attacks in the mITT Analysis Set. Median time based on Kaplan-Meier
Slide 79: Significantly reduced time to 50% reduction in VAS-3
Deucrictibant IR significantly reduced time to 50% reduction in VAS-3 *VAS assessed every 30 minutes up to 4 hours post-treatment, then at 5, 6, 8, 24, 48 hours; †Nominal p-value. Note: N = The number of attacks in the mITT Analysis Set. Median time based on Kaplan-Meier estimates. p-values based on
Slide 80: End of progression within 25 to 26 minutes
In a post-hoc analysis, patients on deucrictibant achieved end of progression by VAS-3 within 25 to 26 minutes EoP: end of progression. HAE: hereditary angioedema. VAS: visual analog scale. Source: Riedl MA, et al. ACAAI 2023. This presentation includes data for an investigational product not yet ap
Slide 81: Reduced time to almost complete or complete symptom relief
Deucrictibant IR significantly reduced time to almost complete or complete symptom relief (all individual VAS ≤10) *VAS assessed every 30 minutes up to 4 hours post-treatment, then at 5, 6, 8, 24, 48 hours; †Nominal p-value. Note: N = The number of attacks in the mITT Analysis Set. Median time based
Slide 82: Substantially less rescue medication used
Patients treating with deucrictibant IR used substantially less rescue medication Note: n = The number of attacks in the mITT Analysis Set. CI, confidence interval; IR; immediate release; VAS, visual analog scale. Source: Jacobs JS, et al. WSAAI 2024. This presentation includes data for an investiga
Slide 83: Deucrictibant IR was well tolerated at all doses
Deucrictibant IR was well tolerated at all doses Note: n = The number of subjects (Part I) and number of attacks (Part II) in the Safety Analysis Set; The Safety Analysis Set includes all randomized patients who received any dose of study drug; Treatment-related AEs within 48 h post-treatment are in
Slide 84: Greater improvement in MSCS and TOS than placebo
Greater improvement in MSCS and TOS with deucrictibant than placebo *Nominal p-value; Note: LS mean, LSMD, CIs, and p-values for MSCS change from pre-treatment/TOS come from mixed-effect models with repeated measures (MMRM). Data after rescue medication use is not included. The combined deucrictiban
Slide 85: Time to symptom relief by TOS PRO
Time to symptom relief by TOS PRO demonstrated consistent efficacy at all doses *Within 48-hour assessments. KM, Kaplan-Meier; NE, not estimable; PRO, patient reported outcome; SC, symptom complex; TOS, treatment outcome score. Source: Manning ME, et al. CIIC 2023. This presentation includes data fo
Slide 86: RAPIDe-2 long-term extension data
RAPIDe-2 long-term extension data On-demand Data
Slide 87: RAPIDe-2 study design
RAPIDe-2 IR, immediate-release. aIncluding laryngeal attacks (without breathing difficulties). *RAPIDEe-2 is an extension of RAPIDe1, a phase 2 study of on-demand treatment of attacks in patients with HAE-C1INH-Type 1/2; †≥1 symptom with VAS score ≥30. HAE, hereditary angioedema; IR, immediate-relea
Slide 88: Study endpoints
Study endpoints ECG, electrocardiogram; PGI-C, Patient Global Impression of Change; PGI-S, Patient Global Impression of Severity; PRO, patient-reported outcome; TEAE, treatment-emergent adverse event. Source: 1Guy W (ed). ECDEU Assessment Manual for Psychopharmacology.1976. 2Cohn DM, et al. Clin Tra
Slide 89: Baseline characteristics
Baseline characteristics *All participants who had ≥1 attack treated with deucrictibant and non-missing PGI-C results from ≥1 post-treatment timepoint. †All participants who received any dose of deucrictibant in the study. ‡Number by the cutoff date of 10 June 2024. BMI, body mass index; HAE, heredi
Slide 90: Well-tolerated across all doses
Deucrictibant was well-tolerated across all doses *Number in the safety analysis set (data cutoff: 10 June 2024). †Tooth caries unrelated to treatment.ECG, electrocardiogram; IR, immediate-release; TEAE, treatment-emergent adverse event (defined as adverse event occurring during time window from fir
Slide 91: PGI-C 'a little better' is the RAPIDe-3 primary endpoint
PGI-C “a little better” is the RAPIDe-3 primary endpoint CI, confidence interval; IR, immediate-release; PGI-C, Patient Global Impression of Change. *Includes 10 mg, 20 mg, and 30 mg dose groups. †PGI-C rating of at least “a little better” for 2 consecutive timepoints by 12 hours post-treatment. Sou
Slide 92: Rapid median reduction in attack severity at 2.6 hours
Rapid median reduction in attack severity at 2.6 hours: 97.7% of attacks achieved a reduction in severity by 12 hours *≥1 point reduction in PGI-S from pre-treatment for 2 consecutive timepoints by 12 hours post-treatment; †261 attacks have non-missing pre-treatment PGI-S. PGI-S, Patient Global Impr
Slide 93: Median attack resolution time 11.5 hours
Median attack resolution time 11.5 hours: 85.8% of attacks completely resolved within 24 hours (90.2% of which with one only dose)1 *PGI-S rating of “none” at 24 hours post-treatment. †261 attacks have non-missing pre-treatment PGI-S. ‡Percentage of 224 attacks achieving complete attack resolution u
Slide 94: Mixed-methods study of standard-of-care attack assessments
Mixed-methods study: Non-interventional collection of HAE attack symptoms assessments following treatment with standard of care Note: Standard of care was icatibant or plasma derived/recombinant C1-Inhibitor. AMRA, angioedema Symptom Rating Scale; HAEA, US Hereditary Angioedema Association; PGI-C, P
Slide 95: Propensity-score-matching analysis versus standard of care
In a propensity-score-matching analysis, deucrictibant showed favorable symptom relief outcomes versus standard of care AMRA, Angioedema syMptom Rating scAle; CI, confidence interval; IR, immediate-release; PGI-C, Patient Global Impression of Change; PGI-S, Patient Global Impression of Severity. N=7
Slide 96: Introduction to HAE
Introduction to HAE
Slide 97: Locations of HAE attacks
Locations of HAE attacks HAE: hereditary angioedema. Source: 1Zuraw BL. N Engl J Med. 2008. 2Nzeako UC, et al. Arch Intern Med. 2001. 3Lumry WR. Am J Manag Care. 2013. 4Farkas H. Allergy Asthma Clin Immunol. 2010. 5Nygren A, et al. Acta Paediatr. 2016. 6Lumry WR. Am J Manag Care. 2013. 7Agostoni A,
Slide 98: Characteristics of HAE attacks
Characteristics of HAE attacks *In Chinese patients, the percentage of abdominal attacks is lower (approximately 34%).6 CT: computed tomography. GI: gastrointestinal. HAE: hereditary angioedema. Source: 1Agostoni A, et al. J Allergy Clin Immunol. 2004. 2Frank MM. eMedicine. September 2010. 3Frank MM
Slide 99: Triggers of HAE attacks
Triggers of HAE attacks *Medications include estrogen-containing oral contraceptives, hormone replacement therapies, ACE inhibitors.1,2 ACE, angiotensin-converting enzyme; HAE, hereditary angioedema. Source: 1Lumry WR. Am J Manag Care. 2013. 2Farkas H, et al. Allergy. 2017. 3Steiner UC, et al. Orpha
Slide 100: A timely and accurate diagnosis is key
A timely and accurate diagnosis is key HAE, hereditary angioedema. Source: 1Zanichelli A, et al. Ann Allergy Asthma Immunol. 2016. 2Greve J, et al. Allergo J Int. 2022. 3Farkas H, et al. Allergy. 2017. 4Christiansen SC, et al. Clin Pediatr. 2016. 5Banerji A, et al. Allergy Asthma Proc. 2018. 6Schöff
Slide 101: HAE treatment goals
HAE treatment goals EAACI, European Academy of Allergy and Clinical Immunology; HAE, hereditary angioedema; LTP, long-term prophylaxis; ODT, on-demand treatment; QoL, quality of life; WAO, World Allergy Organization. Source: 1Maurer M, et al. Allergy. 2022. 2Betschel S, et al. Allergy Asthma Clin Im
Slide 102: Reasons physicians and patients do not treat attacks
Not all HAE attacks are treated: physicians and patients report reasons for not treating most recent attack HAE, hereditary angioedema; ODT, on-demand therapy. Source: Mendivil J et al. ACAAI 2023. This presentation includes data for an investigational product not yet approved by regulatory authorit
Slide 103: Current treatments vary in efficacy, requiring additional healthcare resources
Current treatments vary in efficacy, leading to the need for additional healthcare resources to fully treat attacks HAE, hereditary angioedema; INH, inhibitor; pd, plasma-derived; rh, recombinant humanized. Source: 1Banerji A, et al. Allergy Asthma Proc. 2018. 2Bernstein JA, et al. J Manag Care Spec
Slide 104: Current on-demand treatments for HAE approved in the U.S.
Current on-demand treatments for HAE approved in the U.S. ADR, adverse drug reaction; HAE, hereditary angioedema; INH, inhibitor; ODT, on-demand treatment; pd, plasma-derived; rh, recombinant humanized. Source: 1Maurer M, et al. World Allergy Organ J. 2022. 2Riedl MA, et al. Ann Allergy Asthma Immun
Slide 105: Patients are not fully satisfied with current treatment options
Patients are not fully satisfied with current treatment options Source: 1Mendivil J, et al. ACAAI 2023. 2Tachdjian R, et al. Allergy Asthma Proc. 2024. 3Kelbel T. Ann Allergy Asthma Immunol. 2022. This presentation includes data for an investigational product not yet approved by regulatory authoriti
Slide 106: Unmet needs: self-administration and carrying on-demand medication
Self-administration and people’s ability to always carry their on-demand medication outside their home remain important unmet needs HAE, hereditary angioedema; ODT, on-demand treatment. Source: 1Radojicic C, et al. AAAAI 2023. 2Geng B, et al. ACAAI 2023. This presentation includes data for an invest
Slide 107: Current ODTs present burden due to difficult routes of administration
Current ODTs for HAE present a substantial burden for patients due to their difficult routes of administration *Patients used this treatment as prophylaxis and ODT; †Six percent of respondents (n=2) reported that they did not know or that the question did not apply to them. HAE, hereditary angioedem
Slide 108: Patients desire alternative routes of administration
Patients express a desire for alternative routes of administration, reflecting a general desire to move away from injectables Source: Radojicic C, et al. Allergy Asthma Proc. 2021. This presentation includes data for an investigational product not yet approved by regulatory authorities. Survey of pa
Slide 109: Oral acute therapy would increase likelihood of treating more attacks, earlier
Both doctors and patients consider an oral acute therapy would increase likelihood that patients would treat more attacks, earlier Note: Percentages may not sum to 100 due to rounding. Source: Proprietary Pharvaris research, 2022 (representative sample of patients, n=103, and doctors, n=100) This pr
Slide 110: Switching treatment is moderately easy for HCPs
For both patients on prophylaxis or on-demand therapy, switching treatment is moderately easy for HCPs1 *Based on HCPs experience, considering all the barriers there may be from an access/coverage and clinical perspective. Source: 1Company Research (October 2024). Switching barriers Base: Total U.S.
Slide 111: Efficacy is prime for HCPs; patients prefer oral administration
Efficacy is prime for HCPs, but patient preference drives choice for oral administration1 Source: 1Company Research (October 2024). This presentation includes data for an investigational product not yet approved by regulatory authorities. HCPs top reasons for selecting a therapy (current users, n =
Slide 112: Efficacy vs. convenience trade-off
I prefer the CONVENIENCE of an ORAL ON-DEMAND therapy, even if EFFICACY IS LOWER than injectable routes of administration (IV or subcutaneous) I prefer the MOST EFFECTIVE ON-DEMAND therapy available, even if the route of administration is an INJECTABLE I prefer the CONVENIENCE of an ORAL PROPHYLACTI
Slide 113: Breakthrough attacks still common despite high compliance on novel therapies
Despite high compliance on novel therapies, including Orladeyo®, breakthrough attacks are still common with nearly 3 attacks per year1 Total TAKHZYRO® CINRYZE® ORLADEYO® HAEGARDA® DANOCRINE® Base: current users 216 83 22 55 46 *10 Compliance High 65% 64% 50% 71% 70% 60% Medium 33% 35% 50% 27% 28% 30