Slide 1: RAPIDe-1 Phase 2 Top-line Data
RAPIDe-1 Phase 2 Top-line Data December 8, 2022
Slide 2: Disclaimer
Disclaimer This Presentation may contain 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 forwar
Slide 3: Agenda
Agenda 3 Introduction Berndt Modig, CEO Pharvaris Review of RAPIDe-1 top-line Phase 2 data Peng Lu, MD PhD, CMO Pharvaris KOL perspective Marcus Maurer, MD, Professor of Dermatology and Allergy at the Charité – Universitätsmedizin Berlin; principal investigator on the RAPIDe-1 study Closing Remarks,
Slide 4: Agenda
Agenda 4 Introduction Berndt Modig, CEO Pharvaris Review of RAPIDe-1 top-line Phase 2 data Peng Lu, MD PhD, CMO Pharvaris KOL perspective Marcus Maurer, MD, Professor of Dermatology and Allergy at the Charité – Universitätsmedizin Berlin; principal investigator on the RAPIDe-1 study Closing Remarks,
Slide 5: Significant unmet need in on-demand treatment of HAE attacks
Despite substantial progress there still is a significant unmet need in the on-demand treatment of HAE attacks 5 Treatment today means painful injections … … and often one dose does not suffice … … while finding a place to administer the drug causes an extra burden As a result, people living with HA
Slide 6: Patients hope for rapid, single-dose oral on-demand therapies
People living with HAE are hoping for better on-demand therapies that offer rapid symptom relief with one single, oral dose 6 Patients want rapid onset of symptom relief … … with single dose durability … … in an oral pill Company research, fall 2022, (patients n = 103, HCPs n = 100) Effectively targ
Slide 7: Pharvaris discovered first orally bioavailable bradykinin B2 receptor antagonist
Pharvaris has discovered the first orally bioavailable bradykinin B2 receptor antagonist New molecular entity Potent inhibition of the bradykinin B2 receptor Rapid absorption, exposure, and tolerability in Phase 1 Dose and exposure threshold predicted from human surrogate endpoint Bradykinin challen
Slide 8: Agenda
8 Introduction Berndt Modig, CEO Pharvaris Review of RAPIDe-1 top-line Phase 2 data Peng Lu, MD PhD, CMO Pharvaris KOL perspective Marcus Maurer, MD, Professor of Dermatology and Allergy at the Charité – Universitätsmedizin Berlin; principal investigator on the RAPIDe-1 study Closing Remarks, Q&A Ag
Slide 9: RAPIDe-1 Phase 2 study design
HAE RAPIDe-1 study: Phase 2 study of on-demand treatment of angioedema attacks in patients with Type I or II HAE www.hae-rapide.com; https://clinicaltrials.gov/ct2/show/NCT04618211; https://hae-rapide.us/; https://www.clinicaltrialsregister.eu/ctr-search/search?query=2020-003445-11 9 Screening Perio
Slide 10: RAPIDe-1: Primary, key secondary and other endpoints
RAPIDe-1: Primary, key secondary and other endpoints Primary Endpoint Change in VAS-3 score from pre-treatment to 4h post-treatment Key Secondary Endpoints Time to onset of symptom relief (VAS-3; ≥30% reduction from the pre-treatment score) Time to a ≥50% reduction in VAS-3 score from the pre-treatm
Slide 11: Demographics and baseline characteristics
Demographics and baseline characteristics are generally balanced (mITT Analysis Set) 11 mITT = modified intent-to-treat. The mITT Analysis Set includes all randomized patients who had at least one treated HAE attack and who had non-missing VAS results at both pre-treatment and at least 1 post-treatm
Slide 12: PK profile in HAE patients: rapid absorption confirmed
PK profile in HAE patients: Rapid absorption confirmed, consistent with Phase 1 healthy volunteer studies 12 Rapid absorption with mean plasma levels >EC85 (13.8 ng/mL) reached within 30 min Mean plasma levels maintained >EC85 for approximately 8 h at 10 mg or 20 mg >10 h at 30 mg dose EC85 levels e
Slide 13: Primary endpoint: PHVS416 significantly reduces attack symptoms by VAS-3 at 4h
†Nominal p-value; VAS assessed every 30 minutes up to 4 hours post-treatment, then at 5, 6, 8, 24, 48 hours; N = The number of attacks in the mITT Analysis Set. Attacks in mITT Analysis Set refer to attacks treated with blinded study drug that had non-missing VAS result at pre-treatment and at least
Slide 14: PHVS416 significantly shortened time to onset of symptom relief
†Nominal p-value; VAS assessed every 30 minutes up to 4 hours post-treatment, then at 5, 6, 8, 24, 48 hours; N = The number of attacks in the mITT Analysis Set. Median time based on Kaplan-Meier estimates. p-values based on a marginal Cox proportional hazards model. The combined PHVS416 results are
Slide 15: PHVS416 significantly reduces time to 50% reduction in VAS-3
PHVS416 significantly reduces time to 50% reduction in VAS-3 15 †Nominal p-value; VAS assessed every 30 minutes up to 4 hours post-treatment, then at 5, 6, 8, 24, 48 hours; N = The number of attacks in the mITT Analysis Set. Median time based on Kaplan-Meier estimates. p-values based on a marginal C
Slide 16: Time to almost complete or complete symptom relief
PHVS416 significantly reduces time to almost complete or complete symptom relief (all individual VAS ≤ 10) 16 †Nominal p-value; VAS assessed every 30 minutes up to 4 hours post-treatment, then at 5, 6, 8, 24, 48 hours; N = The number of attacks in the mITT Analysis Set. Median time based on Kaplan-M
Slide 17: Greater improvement in MSCS and TOS with PHVS416 than placebo
Greater improvement in MSCS and TOS with PHVS416 than placebo 17 †Nominal p-value; MSCS = Mean Symptom Complex Severity, TOS = Treatment Outcome Score, CI = confidence interval, LSMD = least-squares mean difference; least-squares mean, LSMD, CIs, and p-values for MSCS change from pre-treatment/TOS c
Slide 18: PHVS416 significantly improves TOS score at 4h
PHVS416 significantly improves TOS score at 4h 18 Source: Vernon M, Rentz AM, Wyrwich KW, et al. Qual Life Res.2009; †Nominal p-value; N = The number of attacks in the mITT Analysis Set. TOS = Treatment Outcome Score. Figure is based on descriptive summary of mean and SEM. The least-squares mean dif
Slide 19: Patients treating with PHVS416 used substantially less rescue medication
Patients treating with PHVS416 used substantially less rescue medication 19 N = The number of attacks in the mITT Analysis Set This presentation includes data for an investigational product not yet approved by regulatory authorities
Slide 20: TOS Patient Reported Outcome (PRO)
TOS Patient Reported Outcome (PRO) 20 TOS PRO captures change in five symptom complexes of HAE attacks At each timepoint, the change in attack symptom from pre-treatment is reported by patient PRO – how do you feel now compared to before receiving study drug?
Slide 21: Time to symptom relief by TOS PRO consistent at all doses
Time to symptom relief by TOS PRO demonstrated consistent efficacy at all doses 21 TOS = Treatment Outcome Score. PRO = Patient Reported Outcome. SC = Symptom Complex. KM = Kaplan-Meier. NE = Not Estimable * Within 48 hours assessments
Slide 22: PHVS416 was well tolerated at all doses
PHVS416 was well tolerated at all doses 22 No treatment-related SAEs or AEs of severe severity No AEs leading to treatment discontinuation No treatment-related AEs of laboratory parameters, vital signs, or ECG parameters Few treatment-related AEs reported within 48 h after administration of study dr
Slide 23: Recap of RAPIDe-1 top-line results
Recap of RAPIDe-1 top-line results A total of 74 patients from 13 countries were enrolled to the study, 62 of them had 147 attacks that were treated with blinded study drug and included in efficacy evaluation The primary endpoint and all key secondary endpoints were met PHVS416 demonstrated rapid on
Slide 24: Agenda
24 Introduction Berndt Modig, CEO Pharvaris Review of RAPIDe-1 top-line Phase 2 data Peng Lu, MD PhD, CMO Pharvaris KOL perspective Marcus Maurer, MD, Professor of Dermatology and Allergy at the Charité – Universitätsmedizin Berlin; principal investigator on the RAPIDe-1 study Closing Remarks, Q&A A
Slide 25: Agenda
25 Introduction Berndt Modig, CEO Pharvaris Review of RAPIDe-1 top-line Phase 2 data Peng Lu, MD PhD, CMO Pharvaris KOL perspective Marcus Maurer, MD, Professor of Dermatology and Allergy at the Charité – Universitätsmedizin Berlin; principal investigator on the RAPIDe-1 study Closing Remarks, Q&A A
Slide 26: On-demand and prophylaxis: two oral products, same active ingredient
On-demand and prophylaxis: Developing two oral products utilizing the same active ingredient 26 PHVS719 Extended-release tablet formulation Aim to maintain compound exposure to prevent attacks, for convenient and effective control* PHVS416 Softgel capsule formulation Potential to provide rapid, easy
Slide 27: Q&A
Q&A
Slide 28: Appendix
Appendix 28
Slide 29: Results summary of primary efficacy endpoint
Results summary of primary efficacy endpoint 29
Slide 30: Results summary of key secondary efficacy endpoints
Results summary of key secondary efficacy endpoints 30
Slide 31: VAS-3 is a measure of HAE attack severity
VAS-3 is a measure of HAE attack severity, based electronically captured numerically assisted visual scale 31 Used in approval of two most recently approved on-demand therapies FIRAZYR® icatibant and RUCONEST® C1 esterase inhibitor [recombinant] VAS, MSCS, TOS are only endpoints listed for attacks i
Slide 32: MSCS and TOS: definitions
MSCS and TOS: definitions 32 Validated patient-reported outcome measures to comprehensively capture symptom severity and change of HAE attacks MSCS (Mean Symptom Complex Severity) score is a point-in-time measure of symptom severity: Patients rated the severity of each affected symptom on a categori
Slide 33: Pharvaris
PHARVARiS Nasdaq: PHVS