Pharvaris N.v. · NASDAQ:PHVS
Pharvaris — Investor Presentation (September 2024)
54 slides from Pharvaris's investor presentation, Sep 5, 2024
Slide 1: Pioneering science for patient choice
Pioneering science for patient choice Corporate Presentation September 2024 Exhibit 99.4
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 Source: 1Betschel SD, et al. J Allergy Clin Immunol Pract. 2023. 2Christiansen SC, et al. Ann Allergy Asthma Immunol. 2023. 3Bork K et al. Al
Slide 5: U.S. LTP and on-demand market expected to grow significantly 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 6: U.S. HAE market is dynamic, with people seeking a better product
453 pts 384 pts 339 pts 329 pts Despite treatment satisfaction, the U.S. HAE market is dynamic, with people actively seeking a better1 product 1Treatment selection is driven by physicians and patient preference. Source: 2Evaluate Pharma uptake curves 2008-2023 3SEC filings (BioCryst, CSL Behring, Ph
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 This presentation includes data for an investigational product not yet approved by regu
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 Directly blocks the main mediator of swelling and inflammation1,3 Has the potential to prevent or treat bradykinin-mediated angioedema irrespective of the source of bradykinin
Slide 9: Deucrictibant has the potential to become a preferred therapy for HAE
Deucrictibant has the potential to become a preferred therapy for people living with HAE *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 2024. 3Crabbe et al,
Slide 10: Deucrictibant differentiated profile for LTP and ODT
Deucrictibant differentiated profile for LTP and ODT Sources: 1Company research. 2Leasge et al, IDDST 2024. 3Groen K et al, ACAAI 2022. 4Li H et al, EAC 2024. 5Maurer M et al. HAEi Workshop, 2022. 6Maurer M et al, AAAAI 2023. 7Riedl et al, WSAAI 2024. 8Maurer M et al. BKS 2024. This presentation inc
Slide 11: Wholly-owned pipeline focused on bradykinin B2 receptor mechanism
Wholly-owned pipeline focused on bradykinin B2 receptor mechanism LTE: long-term extension, OLE: open-label extension This presentation includes data for an investigational product not yet approved by regulatory authorities. MOLECULE FORMULATION INDICATION deucrictibant Undisclosed PHAXXX HAE On-Dem
Slide 12: 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. 2NCT04618211. 3NCT05396105. 4NCT06343779. Phase 22 Complete Phase 2/3 LTE3 Ongoing Phase 3 pivotal4 Ongoing Phase 21 Par
Slide 13: Injectable-like efficacy and oral convenience potential in HAE
Efficacy With injectable-like efficacy and oral convenience, deucrictibant shows potential to become a preferred therapy to manage HAE 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 a
Slide 14: Long-Term Prophylaxis
Long-Term Prophylaxis Deucrictibant extended-release tablets
Slide 15: Deucrictibant clinical development for LTP
On-Demand Treatment (ODT) Long-Term Prophylaxis (LTP) Deucrictibant clinical development for LTP OLE: open-label extension. LTE: long-term extension. Source: 1NCT05047185. 2NCT04618211. 3NCT05396105. 4NCT06343779. Phase 22 Complete Phase 2/3 LTE3 Ongoing Phase 3 pivotal4 Ongoing Phase 21 Part 1: ran
Slide 16: Positive top-line data from CHAPTER-1 Phase 2 study
Positive top-line data from CHAPTER-1, a Phase 2 prophylactic study of deucrictibant in HAE, announced in December 20231 92.3% reduction in occurrence of moderate and severe attacks* 92.6% reduction in occurrence of attacks treated with on-demand medication* Clinically meaningful results across prim
Slide 17: 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 18: 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 19: Summary of safety data in LTP ongoing open-label extension
Summary of safety data in LTP ongoing open-label extension IR, immediate-release; TEAE, treatment emergent adverse event. N = number of participants who received at least 1 dose of blinded study treatment. aDeucrictibant IR capsule, 20 mg twice daily. bDeucrictibant IR capsule, 10 mg twice daily. Th
Slide 20: Early-onset attack reduction sustained 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 21: Occurrence of 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 22: 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 23: 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 24: 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 25: Phase 2 data could position deucrictibant as a preferred LTP
Positive Phase 3 data could position deucrictibant to become a preferred LTP with injectable-like efficacy and the oral 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.
Slide 26: CHAPTER-3 and CHAPTER-4 Clinical Studies
CHAPTER-3 and CHAPTER-4 Clinical Studies
Slide 27: 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 maintain exposure more consistently versus twice-daily IR (used in pr
Slide 28: CHAPTER-3: Global Phase 3 study of prophylactic treatment
CHAPTER-3 study: A global Phase 3 study of prophylactic treatment of angioedema attacks in people with HAE Target enrollment of approximately 81 adolescents and adults living with HAE Initiating by year-end 2024 Study objectives Evaluation and characterization of investigator-confirmed HAE attacks d
Slide 29: On-Demand
On-Demand Deucrictibant immediate-release capsules
Slide 30: Deucrictibant clinical development for ODT
On-Demand Treatment (ODT) Long-Term Prophylaxis (LTP) Deucrictibant clinical development for ODT OLE: open-label extension. LTE: long-term extension. Source: 1NCT05047185. 2NCT04618211. 3NCT05396105. 4NCT06343779. Phase 22 Complete Phase 2/3 LTE3 Ongoing Phase 3 pivotal4 Ongoing Phase 21 Part 1: ran
Slide 31: 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 32: 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. Patient Global Impression of Change PGI-C Much worse Worse A little worse Same A little better Better Much better Pa
Slide 33: RAPIDe-1 Phase 2 on-demand study
RAPIDe-1, a Phase 2 on-demand study of deucrictibant in HAE End of symptom progression in 25-26 minutes* (based on AMRA-3)†2 Onset of symptom relief occurs in 2.4 hours* (≥30% reduction in AMRA-3)1 5-fold reduction in rescue medication use*1 81.7% of attacks achieved symptom resolution in 24 hours*
Slide 34: RAPIDe-2 study design and objective
RAPIDe-2 study design and objective1 IR, immediate-release. aIncluding laryngeal attacks (without breathing difficulties). Source: 1Maurer M et al. BKS 2024. This presentation includes data for an investigational product not yet approved by regulatory authorities. Objective: To evaluate the long-ter
Slide 35: Baseline characteristics
Baseline characteristics1 265 attacks from 17 patients included in the mITT efficacy analysis set (data cutoff: 01 March 2024) 337 attacks from 19 patients included in the safety analysis set (data cutoff: 10 June 2024) 7 of 337 attacks were laryngeal BMI, body mass index; HAE, hereditary angioedema
Slide 36: Summary of safety profile
Summary of safety profile1 No treatment-related serious or severe TEAEs No treatment-related TEAEs in laboratory parameters, vital signs, or ECG findings were reported No TEAEs leading to treatment discontinuation, study withdrawal, or death IR, immediate-release; TEAE, treatment-emergent adverse ev
Slide 37: Rapid median onset of symptom relief at ~1.1 hours
Rapid median onset of symptom relief at ~1.1 hours: 98.5% of attacks achieved onset of symptom relief by 12 hours1 Time to onset of symptom relief is defined as PGI-C rating of at least 'a little better' for two consecutive timepoints post-treatmenta PGI-C, Patient Global Impression of Change. aSymp
Slide 38: 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 hours1 Reduction in attack severity is defined as achieving ≥1 point reduction in PGI-S from pre-treatment for two consecutive time pointsa PGI-S, Patient Global Impression of Severity. aR
Slide 39: 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 Time to complete attack resolution is defined as the time to post-treatment PGI-S rating achieving 'none' PGI-S, Patient Global Impression of Severity. a261 attacks hav
Slide 40: Mixed-methods study of standard of care
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-Inhibiitor This presentation includes data for an investigational product not yet approved by regulato
Slide 41: Propensity-score-matching analysis versus standard of care
In a propensity-score-matching analysis, deucrictibant showed favorable symptom relief outcomes versus standard of care1 This presentation includes data for an investigational product not yet approved by regulatory authorities. AMRA, Angioedema syMptom Rating scAle; CI, confidence interval; IR, imme
Slide 42: RAPIDe-3 Clinical Study
RAPIDe-3 Clinical Study PHARVARiS ©2024
Slide 43: RAPIDe-3 Phase 3 on-demand study enrolling
HAE RAPIDe-31 study enrolling: A global Phase 3 study of on-demand treatment of angioedema attacks in people with HAE-1/2 Target enrollment of approximately 120 adolescents and adults (between 12 and 75 years old) Endpoints Onset of symptom relief Patient Global Impression of Change (PGI-C) rating o
Slide 44: Phase 3 data could position deucrictibant as a preferred ODT
Plasma-derived C1NH (23%) - Icatibant (60%) – Recombinant hC1INH (9%) - Other (9%) Positive Phase 3 data could position deucrictibant to become a preferred ODT with rapid-onset and complete symptom resolution with a single oral capsule References on following slide This presentation includes data fo
Slide 45: 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 46: Our strategy is to become a market leader in HAE
Our strategy 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 B2R
Slide 47: Going Beyond HAE
Going Beyond HAE
Slide 48: 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 49: Bradykinin B2 receptor inhibition is broadly applicable across angioedema
Bradykinin B2 receptor inhibition is broadly applicable across angioedema Source: Busse 2020 J Allergy Clin Immunol Pract; Bork et al 2021 J Allergy Clin Immunol; Zanichelli et al 2012 Allergy; Longhurst et al 2016 Clin. Exp. Immunol.; Otani, Banerji 2017 Immunol. Allergy Clin. N. Am.; Bova et al 20
Slide 50: Deucrictibant proof-of-concept in acquired C1-INH deficiency angioedema
Deucrictibant proof-of-concept in angioedema due to acquired C1-INH deficiency (AAE)1 Estimated prevalence of 1:100,000 to 1:500,000 or ~ 10% of HAE type 1/2 Currently, no therapies approved for AAE Investigator-initiated trial (IIT) by the Amsterdam UMC Source: 1Petersen RS et al. J Allergy Clin Im
Slide 51: POP-AID investigator-initiated study
POP-AID investigator-initiated study: Deucrictibant prevention and treatment of acquired angioedema Source: 1Petersen RS et al. J Allergy Clin Immunol. 2024. This presentation includes data for an investigational product not yet approved by regulatory authorities. Attacks per month Patient 1 Patient
Slide 52: 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
Slide 53: www.pharvaris.com
www.pharvaris.com NASDAQ: PHVS This presentation includes data for an investigational product not yet approved by regulatory authorities.
Slide 54: Ambition to realize deucrictibant potential for bradykinin-mediated conditions
PHVS 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. Immunopharmacology, 2