Prokidney Corp 8-K, filed Jun 10, 2024
Prokidney Corp. (PROK)
About this 8-K
Prokidney Corp filed this 8-K on June 10, 2024.
Items reported
- Item 7.01: Regulation FD disclosure
- Item 8.01: Other events
- Item 9.01: Financial statements and exhibits
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| Item 7.01 | Regulation FD Disclosure. |
On June 10, 2024, ProKidney Corp. (the “Company”) issued a press release titled “ProKidney Announces Positive Interim REGEN-007 Phase 2 Trial Data and Provides Clinical and Operational Updates”. A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The information in Item 7.01 of this Current Report on Form 8-K (including Exhibit 99.1) is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the Exchange Act), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference into any filing by the Company under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
| Item 8.01. | Other Events. |
On June 10, 2024, the Company announced REGEN-007 Phase 2 Trial Interim Efficacy & Safety Data as well as certain clinical and operational updates. On June 10, 2024, the Company also updated its corporate presentation for use in meetings with investors, analysts and others. A copy of this presentation is filed as Exhibit 99.2 to this Current Report on Form 8-K. The Company undertakes no obligation to update, supplement or amend the presentation.
REGEN-007 Phase 2 Trial Interim Efficacy & Safety Data
REGEN-007 is an ongoing multi-center Phase 2 open-label 1:1 randomized two-armed trial in patients with diabetes and chronic kidney disease (CKD) who have an estimated glomerular filtration rate (eGFR) of 20 - 50 mL/min/1.73m². At randomization, patients are allocated to two treatment groups using different dosing regimens. Group 1 replicates the dosing schedule for our Phase 3 clinical study program in which patients receive two rilparencel injections – one in each kidney, three months apart. Group 2 tests an exploratory dosing regimen to investigate whether physiological triggers, rather than a time-based trigger, could optimize multiple administrations of rilparencel. In Group 2, patients receive a single rilparencel dose in one kidney and a second dose in the contralateral kidney only if triggered by a sustained eGFR decline of ≥ 20%, and/or an increase in the urine albumin to creatinine ratio (UACR) from baseline of ≥ 30% and ≥ 30 mg/g.
In Group 1, as of May 7, 2024, patients with at least 12 months follow-up after the second injection of rilparencel (n=13) show stabilized kidney function for 18 months (average eGFR change from baseline to 18 months was -1.3 ml/min/1.73m2). Importantly, similar results were observed in a subset of these patients (n=10) who met key inclusion criteria currently used in our Phase 3 clinical study program (average eGFR change from baseline to 18 months was -0.6 ml/min/1.73m2). Additional analyses will be performed as Group 1 data matures.
Twenty-five patients received at least one rilparencel injection in Group 2; 12 patients received a second rilparencel injection based on eGFR criteria (n=3) or UACR criteria (n=9). Patients in Group 2 who received two injections are scheduled to have up to 18 months of follow-up after their second injection. No rilparencel-related serious adverse events were observed across all patients in the study who received at least one rilparencel injection (n=49).
Clinical and Operational Update
| • | Effective June 1, 2024, ProKidney resumed manufacturing for U.S. and non-European clinical study sites |
| • | Anticipate a potential QP Declaration of Equivalence to EU GMPs to be received by the end of June 2024; this will allow ProKidney to ship rilparencel to clinical study sites in Europe |
| • | In its PROACT 1 study, ProKidney has resumed screening patients under an amended protocol that has been enriched with higher risk patients |
| • | In its PROACT 2 study, ProKidney recently activated sites in Spain in anticipation of receipt of the QP Declaration of Equivalence to EU GMPs |
Forward Looking Statements
This disclosure in this report contains includes “forward-looking statements” within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. ProKidney’s actual results may differ from its expectations, estimates and projections and consequently, you should not rely on these forward-looking statements as predictions of future events. Words such as “expect,” “estimate,” “project,” “budget,” “forecast,” “anticipate,” “intend,” “plan,” “may,” “will,” “could,” “should,” “believes,” “predicts,” “potential,” “continue,” and similar expressions (or the negative versions of such words or expressions) are intended to identify such forward-looking statements. These forward-looking statements include, without limitation, the potential of rilparencel to preserve kidney function in patients with moderate to severe CKD, the potential QP Declaration of Equivalence to EU GMPs, the potential benefits and impact of the Company’s products, if approved, and potential regulatory approvals. Most of these factors are outside of the Company’s control and are difficult to predict. Factors that may cause such differences include, but are not limited to: the inability to maintain the listing of the Company’s Class A ordinary shares on the Nasdaq; the inability to implement business plans, forecasts, and other expectations or identify and realize additional opportunities, which may be affected by, among other things, competition and the ability of the Company to grow and manage growth profitably and retain its key employees; the risk of downturns and a changing regulatory landscape in the highly competitive biotechnology industry; the inability of the Company to raise financing in the future; the inability of the Company to obtain and maintain regulatory clearance or approval for its products, and any related restrictions and limitations of any cleared or approved product; the inability of the Company to identify, in-license or acquire additional technology; the inability of the Company to compete with other companies currently marketing or engaged in the biologics market and in the area of treatment of kidney diseases; the size and growth potential of the markets for the Company’s products, if approved, and its ability to serve those markets, either alone or in partnership with others; the Company’s estimates regarding expenses, future revenue, capital requirements and needs for additional financing; the Company’s financial performance; the Company’s intellectual property rights; uncertainties inherent in cell therapy research and development, including the actual time it takes to initiate and complete clinical studies and the timing and content of decisions made by regulatory authorities; the fact that interim results from our clinical programs may not be indicative of future results; the impact of geo-political conflict on the Company’s business; and other risks and uncertainties included under the heading “Risk Factors” in the Company’s most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. The Company cautions readers that the foregoing list of factors is not exclusive and cautions readers not to place undue reliance upon any forward-looking statements, which speak only as of the date made. The Company does not undertake or accept any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements to reflect any change in its expectations or any change in events, conditions or circumstances on which any such statement is based.
| Item 9.01. | Financial Statements and Exhibits. |
(d) Exhibits
| 99.1 | Press Release dated June 10, 2024 | |
| 99.2 | Investor Presentation dated June 10, 2024 | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document). | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| PROKIDNEY CORP. | ||||||
| Date: June 10, 2024 | By: | /s/ James Coulston | ||||
| James Coulston | ||||||
| Chief Financial Officer | ||||||
Exhibit 99.1
ProKidney Announces Positive Interim REGEN-007 Phase 2 Trial Data and Provides Clinical and Operational Updates
| • | Interim results of REGEN-007 Phase 2 trial show stabilization of kidney function for 18 months |
| • | Safety profile consistent with prior studies and comparable to kidney biopsy |
| • | Resumed manufacturing and both PROACT 1 and PROACT 2 Phase 3 trials |
| • | Management to host live webcast today at 8:00 a.m. ET |
WINSTON-SALEM, N.C., June 10, 2024 — ProKidney Corp. (Nasdaq: PROK) (“ProKidney”), a leading late clinical-stage cellular therapeutics company focused on chronic kidney disease (CKD), today announced positive interim results from the Phase 2 REGEN-007 trial evaluating the Company’s renal autologous cell therapy, rilparencel, in patients with CKD caused by diabetes and provided clinical and operational updates. Management will host a live webcast today at 8:00 a.m. ET to discuss the data.
REGEN-007 Phase 2 Trial Interim Efficacy & Safety Data
REGEN-007 is an ongoing multi-center Phase 2 open-label 1:1 randomized two-armed trial in patients with diabetes and CKD who have an estimated glomerular filtration rate (eGFR) of 20 - 50 mL/min/1.73m². At randomization, patients are allocated to two treatment groups using different dosing regimens. Group 1 replicates the dosing schedule for our Phase 3 clinical study program in which patients receive two rilparencel injections – one in each kidney, three months apart. Group 2 tests an exploratory dosing regimen to investigate whether physiological triggers, rather than a time-based trigger, could optimize multiple administrations of rilparencel. In Group 2, patients receive a single rilparencel dose in one kidney and a second dose in the contralateral kidney only if triggered by a sustained eGFR decline of ≥ 20%, and/or an increase in the urine albumin to creatinine ratio (UACR) from baseline of ≥ 30% and ≥ 30 mg/g.
In Group 1, as of May 7, 2024, patients with at least 12 months follow-up after the second injection of rilparencel (n=13) show stabilized kidney function for 18 months (average eGFR change from baseline to 18 months was -1.3 ml/min/1.73m2). Importantly, similar results were observed in a subset of these patients (n=10) who met key inclusion criteria currently used in our Phase 3 clinical study program (average eGFR change from baseline to 18 months was -0.6 ml/min/1.73m2). Additional analyses will be performed as Group 1 data matures.
Twenty-five patients received at least one rilparencel injection in Group 2; 12 patients received a second rilparencel injection based on eGFR criteria (n=3) or UACR criteria (n=9). Patients in Group 2 who received two injections are scheduled to have up to 18 months of follow-up after their second injection. No rilparencel-related serious adverse events were observed across all patients in the study who received at least one rilparencel injection (n=49).
Clinical and Operational Update
| • | Effective June 1, 2024, ProKidney resumed manufacturing for U.S. and non-European clinical study sites |
| • | Anticipate a potential QP Declaration of Equivalence to EU GMPs to be received by the end of June 2024; this will allow ProKidney to ship rilparencel to clinical study sites in Europe |
| • | In its PROACT 1 study, ProKidney has resumed screening patients under an amended protocol that has been enriched with higher risk patients |
| • | In its PROACT 2 study, ProKidney recently activated sites in Spain in anticipation of receipt of the QP Declaration of Equivalence to EU GMPs |
“These are critically important operational milestones for ProKidney. Manufacturing has restarted, and our Phase 3 clinical study program has resumed. Furthermore, the interim results from REGEN-007 are promising and reveal the potential of rilparencel to preserve kidney function in patients with moderate to severe CKD,” said Bruce Culleton, Chief Executive Officer. “This is our first clinical study using bilateral kidney dosing and cryopreserved rilparencel replicating our approach in both PROACT 1 and PROACT 2 Phase 3 studies. I am very excited for the next phase of ProKidney’s evolution as we endeavor to demonstrate preservation of kidney function using rilparencel in a patient population with limited therapeutic options.”
Webcast Information
Management will host a live conference call and webcast at 8:00 a.m. ET today, June 10, 2024, to discuss the REGEN-007 data, the restart of manufacturing, and the resumption of the Phase 3 trials. The conference call can be accessed by dialing 1-877-407-0784 from the United States or 1-201-689-8560 internationally, followed by conference ID: 13747006. The live webcast will be available here and in the Events & Presentation section of ProKidney’s website at www.prokidney.com, with an archived replay available for approximately 90 days following the event.
About ProKidney
ProKidney, a pioneer in the treatment of CKD through innovations in cellular therapy, was founded in 2015 after a decade of research. ProKidney’s lead product candidate, rilparencel (also known as REACT®), is a first-of-its-kind, patented, proprietary autologous cellular therapy being evaluated to potentially preserve kidney function in diabetic patients at high risk of kidney failure. Rilparencel has received Regenerative Medicine Advanced Therapy (RMAT) designation, as well as FDA and EMA guidance, supporting its ongoing Phase 3 clinical program. For more information, please visit www.prokidney.com.
Forward-Looking Statements
This press release includes “forward-looking statements” within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. ProKidney’s actual results may differ from its expectations, estimates and projections and consequently, you should not rely on these forward-looking statements as predictions of future events. Words such as “expect,” “estimate,” “project,” “budget,” “forecast,” “anticipate,” “intend,” “plan,” “may,” “will,” “could,” “should,” “believes,” “predicts,” “potential,” “continue,” and similar expressions (or the negative versions of such words or expressions) are intended to identify such forward-looking statements. These forward-looking statements include, without limitation, the potential of rilparencel to preserve kidney function in patients with moderate to severe CKD, the potential QP Declaration of Equivalence to EU GMPs, the potential benefits and impact of the Company’s products, if approved, and potential regulatory approvals. Most of these factors are outside of the Company’s control and are difficult to predict. Factors that may cause such differences include, but are not limited to: the inability to maintain the listing of the Company’s Class A ordinary shares on the Nasdaq; the inability to implement business
plans, forecasts, and other expectations or identify and realize additional opportunities, which may be affected by, among other things, competition and the ability of the Company to grow and manage growth profitably and retain its key employees; the risk of downturns and a changing regulatory landscape in the highly competitive biotechnology industry; the inability of the Company to raise financing in the future; the inability of the Company to obtain and maintain regulatory clearance or approval for its products, and any related restrictions and limitations of any cleared or approved product; the inability of the Company to identify, in-license or acquire additional technology; the inability of the Company to compete with other companies currently marketing or engaged in the biologics market and in the area of treatment of kidney diseases; the size and growth potential of the markets for the Company’s products, if approved, and its ability to serve those markets, either alone or in partnership with others; the Company’s estimates regarding expenses, future revenue, capital requirements and needs for additional financing; the Company’s financial performance; the Company’s intellectual property rights; uncertainties inherent in cell therapy research and development, including the actual time it takes to initiate and complete clinical studies and the timing and content of decisions made by regulatory authorities; the fact that interim results from our clinical programs may not be indicative of future results; the impact of geo-political conflict on the Company’s business; and other risks and uncertainties included under the heading “Risk Factors” in the Company’s most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. The Company cautions readers that the foregoing list of factors is not exclusive and cautions readers not to place undue reliance upon any forward-looking statements, which speak only as of the date made. The Company does not undertake or accept any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements to reflect any change in its expectations or any change in events, conditions or circumstances on which any such statement is based.
Investor Contacts:
ProKidney
Ethan Holdaway
Ethan.Holdaway@prokidney.com
LifeSci Advisors, LLC
Daniel Ferry
Daniel@lifesciadvisors.com
Exhibit 99.2
Developing Solutions for Dialysis Prevention REGEN-007 Interim Results March 2024 & Updates June 2024
Forward-looking Statements This presentation includes “forward-looking statements” within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. ProKidney’s actual results may differ from its expectations, estimates and projections and consequently, you should not rely on these forward-looking statements as predictions of future events. Words such as “expect,” “estimate,” “project,” “budget,” “forecast,” “anticipate,” “intend,” “plan,” “may,” “will,” “could,” “should,” “believes,” “predicts,” “potential,” “continue,” and similar expressions (or the negative versions of such words or expressions) are intended to identify such forward-looking statements. These forward-looking statements include, without limitation, the Company’s expectations with respect to financial results, future performance, development and commercialization of products, if approved, the potential benefits and impact of the Company’s products, if approved, potential regulatory approvals, and the size and potential growth of current or future markets for the Company’s products, if approved. Most of these factors are outside of the Company’s control and are difficult to predict. Factors that may cause such differences include, but are not limited to: the inability to maintain the listing of the Company’s Class A ordinary shares on the Nasdaq; the inability to implement business plans, forecasts, and other expectations or identify and realize additional opportunities, which may be affected by, among other things, competition and the ability of the Company to grow and manage growth profitably and retain its key employees; the risk of downturns and a changing regulatory landscape in the highly competitive biotechnology industry; the inability of the Company to raise financing in the future; the inability of the Company to obtain and maintain regulatory clearance or approval for its products, and any related restrictions and limitations of any cleared or approved product; the inability of the Company to identify, in-license or acquire additional technology; the inability of Company to compete with other companies currently marketing or engaged in the biologics market and in the area of treatment of kidney diseases; the size and growth potential of the markets for the Company’s products, if approved, and its ability to serve those markets, either alone or in partnership with others; the Company’s estimates regarding expenses, future revenue, capital requirements and needs for additional financing; the Company’s financial performance; the Company’s intellectual property rights; uncertainties inherent in cell therapy research and development, including the actual time it takes to initiate and complete clinical studies and the timing and content of decisions made by regulatory authorities; the impact of COVID-19 or geo-political conflict such as the war in Ukraine on the Company’s business; and other risks and uncertainties indicated from time to time in the Company’s filings with the Securities and Exchange Commission. The Company cautions readers that the foregoing list of factors is not exclusive and cautions readers not to place undue reliance upon any forward-looking statements, which speak only as of the date made. The Company does not undertake or accept any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements to reflect any change in its expectations or any change in events, conditions or circumstances on which any such statement is based. This presentation shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. 2
An Introduction to ProKidney Preserve kidney function in advanced CKD patients Goal Preserve kidney function in patients with moderate to severe chronic kidney disease caused by diabetes who are faced with limited options for care beyond transplantation or dialysis A proprietary autologous cellular therapy with RMAT designation Rilparencel Currently in pivotal Phase 3 clinical development with REGEN-006 (PROACT 1) and REGEN-016 (PROACT 2) Supported by three Phase 2 clinical trials in advanced CKD patient populations Leadership Team with Clinical Development & Regulatory Experience Leadership Together the team brings over 150 years cumulative experience in the discovery, development, manufacturing and commercialization of biotechnology, pharmaceutical, and device products Recent Meaningful Recent Developments Developments Phase 2 REGEN-007 interim results published in June 2024 Resumed PROACT 1 and PROACT 2 Phase 3 trials; resumed manufacturing for our clinical studies CKD = chronic kidney disease RMAT = regenerative medicine advanced therapy 3
What is Rilparencel and Why is it Relevant? Unmet Needs Our Goals Our Product Our Plan Over 35 million U.S. adults Preserve kidney function Rilparencel is a proprietary Phase 3 clinical program have chronic kidney disease cell therapy using the PROACT 1 and PROACT 2 (CKD)1 Reduce or potentially patient’s own kidney cells are focused on patients eliminate time spent on with Stage 3b/4 CKD More than 135,000 of these dialysis Early clinical data caused by type 2 diabetes CKD patients progress to demonstrate a potential to dialysis every year1 Return autonomy to preserve kidney function Potential label expansion to patients and their families re-dose rilparencel for long-Total annual costs to May provide greater benefit term dialysis prevention Medicare for patients with to higher-risk CKD patients CKD (including ESRD) exceed $138B2 4 1. CDC Fact Sheet. https://www.cdc.gov/kidneydisease/publications-resources/ckd-national-facts.html 2. USRDS 2023 Annual Report
Overview of the Rilparencel Clinical Program PRECLINICAL IND PHASE 1 PHASE 2 PHASE 3 STATUS Pivotal Trial Program Diabetes Type II – Prevent/Delay ESRD in Stage 3b/4 CKD 006/PROACT 1 Ongoing (20-35 ml/min/1.73m2, n=685) Diabetes Type II – Prevent/Delay ESRD in Stage 3b/4 CKD 2 016/PROACT 2 Ongoing (20-44 ml/min/1.73m , n=600) Long term follow-up study for patients previously treated with rilparencel 008 Ongoing Supportive Trials Diabetes Type II – Prevent/Delay ESRD in Stage 3/4 CKD Final Data (20-50 ml/min/1.73m2, n=83) 002 Presented Diabetes Type I & II – Prevent/Delay ESRD in Stage 3/4 CKD Fully (20-50 ml/min/1.73m2, n=53) 007 Enrolled Completed Trials Diabetes Type II – Delay ESRD in Stage 4/5 CKD Trial (14-20 ml/min/1.73m2, n=10) 003 Completed Congenital Anomalies – Prevent/Delay ESRD 004 Trial (14-50 ml/min/1.73m2, n=5) Completed Frozen product Unilateral injections Bilateral injections ESRD = End-Stage Renal Disease 5 As of June 2024
With Relaunch of Manufacturing and Phase 3 Studies, We Look Forward to Advancing our Clinical Program Manufacturing Relaunch Resumption of Phase 3 Program • In our PROACT 1 study, we filed a protocol • Effective June 1st, we restarted manufacturing amendment with the FDA in March, 2024; Central for our clinical studies IRB approval has been received; sites are now open for enrollment under the amended protocol • We anticipate QP Declaration of Equivalence to EU GMPs to be received by the end of June • In our PROACT 2 study, we initiated sites in Spain in 2024 anticipation of receipt of our QP Declaration of Equivalence to EU GMPs 6
Advancing a Comprehensive Clinical Plan REGEN-003 Phase 2 Trial; Results published 1Q 2023 2023 • Safety & efficacy of rilparencel in Stage 4/5 CKD caused by type 2 diabetes (eGFR 14-20) • Assessed impact on progression and time to dialysis in patients with imminent risk of dialysis 2024 RMCL-002 Phase 2 Trial; Results published 2Q 2024 • Open-label safety & efficacy of rilparencel in Stage 3/4 CKD caused by type 2 diabetes (eGFR 20-50) • Potential to preserve kidney function for up to 30 months in several patient groups REGEN-007 Phase 2 Trial; Enrollment complete; Interim results published 2Q 2024 • Open-label safety & efficacy of rilparencel in Stage 3/4 CKD caused by diabetes (eGFR 20-50) • Bilateral kidney injections & cryopreserved commercial formulation Phase 3 Randomized Controlled Clinical Trials – Stage 3b/4 CKD caused by type 2 diabetes • PROACT 1 resumed enrollment in 2Q 2024 • PROACT 2 commenced site activations in 2Q 2024 REGEN-007 Phase 2 Trial; Full 12 month data from Group 1 expected in 1H 2025 2025 and beyond Update on Mechanism of Action in 2H 2025 Phase 3 Randomized Controlled Clinical Trials – Stage 3b/4 CKD caused by type 2 diabetes • Completion of both studies anticipated in 2027 7
Unmet Clinical and Payer Need in High-Risk CKD Patients • CKD is defined as abnormalities of kidney structure or function, present for > 3 months • CKD is classified based on Cause, GFR category (G1-G5), and Albuminuria (A1-A3), abbreviated as CGA Standard of Care Antihypertensives o ACEi Risk for ESRD o ARB Glucose & Inflammation Reduction Low o SGLT2i Moderately o DPP-4 d o GLP-1 Increased Rilparencel’s High Target Population Very High Today, clinical priorities for patients with Stage 4 CKD (G4) are largely focused on treating co-morbidities and preparing patients for transplantation or dialysis 8 Kidney Disease: Improving Global Outcomes (KDIGO) Diabetes Work Group. KDIGO 2020 clinical practice guideline for diabetes management in chronic kidney disease. Kidney Int 2020;98:S1–115.
Therapeutic Options that Delay the Need for Dialysis in Patients with Stage 4 Chronic Kidney Disease are Limited Study Active Product Subjects with Stage 4 CKD Canagliflozin and Renal Outcomes in Type 2 Diabetes and Nephropathy1 Canagliflozin (SGLT2 inhibitor) 0% Dapagliflozin in Patients with CKD2 Dapagliflozin (SGLT2 inhibitor) 14% Empagliflozin in Patients with CKD3 Empagliflozin (SGLT2 inhibitor) 34% Effect of Finerenone on Cardiovascular and Kidney Outcomes in Patients with Type 2 Finerenone (Selective MRA) 7% Diabetes and CKD4 Effects of Semaglutide on Chronic Kidney Disease in Patients with Type 2 Diabetes5 Semaglutide (GLP-1RA) 11% All recent landmark clinical trials in CKD primarily focus on Stage 2 and 3 CKD 9 1. Perkovic V et al. N Eng J Med 2019 3. Herrington et al. N Engl J Med 2023 5. Perkovic V et al. N Engl J Medicine 2024 2. Heerspink H et al. N Engl J Med 2020 4. Agarwal. R et al. Eur Heart J. 2022; Sarafidis. P et al. Clin J Am Soc Nephrol 2023
While New Therapies Are a Step Forward, Patients Still Lose Kidney Function and Experience Clinically Significant Events SGLT2 inhibitors Do Not Prevent Progression of Advanced CKD Standard of Care has Limitations Patients continue to lose kidney function on SGLT2 inhibitors and Current standard of care1 does not prevent events in ~50-75% progress to Stage 4/5 CKD of people with diabetic kidney disease2 Approx. Change in 2 Dapagliflozin Placebo eGFR (ml/min/1.73m ) Month 12 -4.5 -4 Month 18 -5.5 -6 of GFR Month 24 -6.5 -7.5 kidney (%) ) 2 death . 73m eGFR, in Estimated and Incidence in (ml/min/1 failure DAPA: -2.86 ml/min/1.73m2 per year versus decrease Change SoC: -3.79 ml/min/1.73m2 per year Cumulative 50% While dapagliflozin demonstrated <1.0 ml/min/yr difference in eGFR, Dapagliflozin: 19 patients required treatment to prevent one primary it was able to achieve a reduction in clinically important events outcome event 10 1. Standard of care includes ACE inhibitors, angiotensin receptor blockers and SGLT2 inhibitors 2. Heerspink HJL et al. N Eng J Med 2020
REGEN-007 Interim Analysis May 7, 2024
REGEN-007 Trial Design Group 1 Dosing Regimen and Use of Cryopreserved Product Mirrors Phase 3 Program Key Entry Criteria Group 1 1st & 2nd EOS CKD with type 1 or type 2 diabetes (Ph3 Dosing Regimen) Rilparencel Month 18 Following Injections 2nd Injection Subjects 30-80 years of age n = 25 2 eGFR ≥20 and ≤50 mL/min/1.73m UACR 30-5000 mg/g ïµïµïµïµïµïµ HbA1c <10% Screening Visit R st 1 EOS and Biopsy 1:1 Rilparencel Month 18 Following Injection 1st Injection Day –60 to Day 30 Group 2 Re-Dosing Trigger Group 2 Sustained 30-Day: (Exploratory Dosing ïµïµïµïµïµïµ Assess 2nd Injection • Decline in eGFR of ≥20% from Regimen) Trigger (Months 3-15) baseline, and/or n = 25 • Increase of ≥30% and ≥30 mg/g in UACR from baseline 2nd EOS = 3 Months Rilparencel Month 18 Following Injection 2nd Injectionïµ = Follow-up Visit After Last Injection ïµïµïµïµïµïµ 12 eGFR = estimated glomerular filtration rate; UACR = urine albumin-to-creatinine ratio (a measure of albuminuria)
REGEN-007 Interim Analysis Objectives and Endpoints in Group 1 • In subjects with at least 12 months follow-up after 2 injections, assess the Objectives safety and efficacy of cryopreserved rilparencel delivered into the biopsied and non-biopsied contralateral kidney using a percutaneous approach • Procedural and investigational product-related adverse events Endpoints • Change in kidney function as measured by eGFR 13
Current Enrollment Status & Completion Expectations 53 Subjects were Randomized in REGEN-007 with 27 Subjects Randomized to Group 1 (1 Subject Withdrew Consent Pre-Biopsy) 26 Subjects in Group 1 Of the 26 Subjects who were Biopsied, 24 Subjects Received at-least 1 Injection (2 Subject’s Biopsies had Insufficient Cells for Injection) 24 Subjects Of the 24 Subjects, 1 Subject had a Contra-indication (Bleeding Risk) for a 2nd Injection & 1 Subject Died before 12 Months Follow-up 22 Subjects Expected to Receive 2 Injections with 12 Months Follow-up As of May 7, 2024: 13 Subjects Have Received 2 Injections with a Minimum of 12 Months Follow-up post 2nd Injection 14
Baseline Characteristics in Group 1 Subjects with a Minimum of 12 Months Follow-up after Two Rilparencel Injections SUBJECTS WITH MINIMUM 12 MONTHS SUBJECTS WITH MINIMUM 12 MONTHS FOLLOW-FOLLOW-UP AFTER 2ND INJECTION UP AFTER 2ND INJECTION AND COMPARABLE TO (n=13) PHASE 3 INCLUSION CRITERIA (n=10) Age, years (mean +/- SD) 62.8 +/- 8.2 63.9 +/- 8.7 Female : Male, % 54% : 46% 60% : 40% Hispanic or Latino, % 0% 0% Race, % Black or African American 0% 0% White 100% 100% Other 0% 0% Blood pressure, mm HG 135 / 72 138 / 74 eGFR, ml/min/1.73m2 (mean +/- SD) 29.7 +/- 9.5 25.6 +/- 4.9 UACR mg/g (median, min max) 239 (4, 2392) 390 (35, 2392) HbA1c, % (mean +/- SD) 7.3 % +/- 1.6 7.3 % +/- 1.6 ACE/ARB Use, % 69% 60% SGLT2 Use, % 31% 20% GLP-1 Use, % 46% 60% 15 HbA1c = hemoglobin A1c; SGLT2 = sodium glucose cotransporter 2 protein inhibitor; ACE = angiotensin converting enzyme; ARB = angiotensin II receptor blockers
Externally Developed Control Arm to Contextualize REGEN-007 Interim Data • Explore how 18 month change in kidney function in subjects enrolled in Objective REGEN-007 might compare against matched contemporaneous controls • In partnership with Dr. Navdeep Tangri (University of Manitoba), 13 subjects Methods from REGEN-007 were matched 10:1 with diabetic subjects from recent CKD clinical trials • Matching was independently performed based upon 2-year risk of kidney failure using Klinrisk1 software and comparable usage of SGLT2 inhibitors 16 1. https://www.klinrisk.com/
Klinrisk Founding Team Navdeep Tangri J.D. McCullough Co-Founder and CEO Chief Operating Officer Founder and Scientific Director, Chronic Health tech executive specializing in regulated Disease Innovation Centre AI commercialization Professor of Medicine, University of Manitoba • Global leader in risk prediction who developed the most widely used • First autonomous AI FDA clearance and SaMD reimbursement including algorithms in nephrology worldwide CMS coverage at Digital Diagnostics • Published more than 380 manuscripts • Closed seven figure deals with health systems, payors, labs, and biopharma companies • Risk equations have been integrated in electronic health records (Epic), laboratory information systems, and national & international clinical • Led FDA strategy and engagement for 10+ SaMD products, including practice guidelines Breakthrough, PMA, De Novo, and 510(k) • Strong track record of leading international clinical trials, developing trial • Licensed over 50M patient records globally to drive AI & drug development endpoints with FDA and participating FDA discussions on drug approval and • Strategic advisor to Top 20 Biopharma, regulatory & reimbursement firms, labeling and venture-backed startups • Relationships with large pharmaceutical companies – considered a key • Previous Commercial & Product Executive positions at Aegis Ventures, opinion leader internationally in the CKD space Arcturis Data, Digital Diagnostics 17
Matched Controls Showed a Continuous Decline in Kidney Function over 18 Months Klinrisk Matched Subjects (n=125) Average Change in eGFR from Baseline at 18 Months -1.2 -2.2 -6.2 ml/min/1.73m2 (95% CI -7.8, -4.6) -4.0 -6.2 18
Kidney Function Stabilizes for 18 Months After 1st Injection Group 1 Subjects (n=13) with Minimum 12 Months Follow-up Data Post 2nd Injection Average Change from Baseline eGFR (ml/min/1.73m2) 10 1st Inj 2nd Inj 2nd Inj + 3m 2nd Inj + 6m 2nd Inj + 9m 2nd Inj + 12m 2nd Inj + 15m 5 1.7 1.0 0.9 -0.5 -1.3 -2.7* 0 -5 N= 13 12 13 13 13 13 12 -10 0 3 6 9 12 15 18 Months Average eGFR (ml/min/1.73m2) 40 1st Inj 2nd Inj 2nd Inj + 3m 2nd Inj + 6m 2nd Inj + 9m 2nd Inj + 12m 2nd Inj + 15m 30.7 30.6 29.7 29.8 29.2 28.8 30 27.0* 20 N= 13 12 13 13 13 13 12 10 0 3 6 9 12 15 18 Months 19 Interim analysis; Data points are mean +/- SEM ; Data as of May 7, 2024 *eGFR highly influenced by 1 patient that recovered on measurements at month 15 and month 18 Average Change from Baseline with 18 Months Follow-up Post 1st Injection -1.3 ml/min/1.73m2 (95% CI -5.1, 2.5) Average eGFR in Group 1 was 29.7 at Baseline vs 28.8 at 18 Months Post 1st Injection [absolute difference -0.9 ml/min/1.73m2 at 18-months]
Kidney Function Stabilizes for 18 Months After 1st Injection in Subjects Meeting Phase 3 Criteria Group 1 Subjects Ph 3 Eligible Subgroup (n=10) with Minimum 12 Months Follow-up Data Post 2nd Injection Average Change from Baseline eGFR (ml/min/1.73m2) 10 1st Inj 2nd Inj 2nd Inj + 3m 2nd Inj + 6m 2nd Inj + 9m 2nd Inj + 12m 2nd Inj + 15m 3.2 2.9 5 1.5 0.7 -0.6 -1.3* 0 -5 N= 10 10 10 10 10 10 9 -10 0 3 6 9 12 15 18 Months Average eGFR (ml/min/1.73m2) 40 1st Inj 2nd Inj 2nd Inj + 3m 2nd Inj + 6m 2nd Inj + 9m 2nd Inj + 12m 2nd Inj + 15m 28.8 28.5 27.1 25.6 26.3 30 24.3* 25.1 20 N= 10 10 10 10 10 10 9 10 0 3 6 9 12 15 18 Months 20 Interim analysis; Data points are mean +/- SEM ; Data as of May 7, 2024 *eGFR highly influenced by 1 patient that recovered on measurements at month 15 and month 18 Average Change from Baseline with 18 Months Follow-up Post 1st Injection -0.6 ml/min/1.73m2 (95% CI -4.7, 3.6) Phase 3 Criteria: CKD caused by type 2 diabetes Subjects 30-80 years of age eGFR ≥20 and ≤44 mL/min/1.73m2 Not on kidney dialysis, HbA1c <10% UACR ≤5000 mg/g Average eGFR in Group 1 was 25.6 at Baseline vs 25.1 at 18 Months Post 1st Injection [absolute difference -0.5 ml/min/1.73m2 at 18-months]
Kidney Function After 1st Injection Across All Subjects All Subjects (n=24) and All Phase 3 Eligible Subjects (n=20) Treated in Group 1 Kidney Function After 1st Injection Across All Subjects (n=24): Avg Change from Baseline eGFR (ml/min/1.73m2) 10 1st Inj 2nd Inj 2nd Inj + 3m 2nd Inj + 6m 2nd Inj + 9m 2nd Inj + 12m 2nd Inj + 15m 5 0.9 -1.0 -1.3 -1.3 -1.3 0 -3.1* -5 N= 24 21 19 17 14 13 12 -10 0 3 6 9 12 15 18 Months Kidney Function After 1st Injection Across All Ph3 Eligible Subjects (n=20): Avg Change from Baseline eGFR (ml/min/1.73m2) 10 1st Inj 2nd Inj 2nd Inj + 3m 2nd Inj + 6m 2nd Inj + 9m 2nd Inj + 12m 2nd Inj + 15m 1.5 5 -0.5 -0.5 -0.6 -2.0* -0.6 0 -5 N= 20 19 16 14 11 10 9 -10 0 3 6 9 12 15 18 Months 21 Interim analysis; Data points are mean +/- SEM ; Data as of May 7, 2024 *eGFR highly influenced by 1 patient that recovered on measurements at month 15 and month 18 Additional analyses will be performed as Group 1 data matures
No Rilparencel-related Serious Adverse Events have been Observed BIOPSY RILPARENCEL INJECTION ADVERSE EVENT # of SAEs # of SAEs (n=51) (n=49) Hematoma 2 1 Thrombosis 1 0 Hematuria 1 0 Hydronephrosis 1 0 Death 0 0 Acute Kidney Injury 2 0 22 Data as of May 7, 2024
REGEN-007 Interim Analysis Summary Next Steps • We look forward to providing full results in 1H • In Group 1 participants who had at least 12 months 2025 follow up after a second rilparencel injection, kidney function was preserved for 18 months. Similar results were observed in participants who • We have enriched our Phase 3 PROACT 1 Study to were evaluated under Phase 3 inclusion criteria include more subjects with the highest risk of kidney failure • Bilateral dosing of cryopreserved product showed safety profile consistent with prior studies and • We have resumed PROACT 1 and PROACT 2 and comparable to kidney biopsy look forward to enrolling new subjects in the near future 23
Headquarters: Boston, MA Winston-Salem, NC Financial Highlights Justin Zelin BTIG Yigal Nochomovitz Citigroup Inc. Jonathan Miller Evercore ISI Judah Frommer Morgan Stanley Eliana Merle UBS Kelly Shi Jefferies *As of May 10, 2024 **Cash, cash equivalents and marketable securities as of March 31, 2024
Appendix 25 PROKIDNEY:~:~· .....
Group 2 Patients After Receiving At Least One Injection (n=25) 12 patients received a 2nd rilparencel injection based on eGFR criteria (n=3) or UACR criteria (n=9) Group 2 Patients After Receiving At Least One Injection (n=25): Average Change from Baseline eGFR (ml/min/1.73m2) Group 2 Re-Dosing Trigger After 1 Injection After 2 Injections, UACR Trigger After 2 Injections, eGFR Trigger Sustained 30-Day: • Decline in eGFR of ≥20% from baseline, and/or • Increase of ≥30% and ≥30 mg/g in 0.8 2.8 -0.5 -0.9 -1.7 UACR from baseline -2.6 0.0 -3.0 -2.7 -2.5 -2.0 -4.5 13 of 25 patients have received 1 injection -20.7 -16.0 9 of 25 patients received a 2nd injection -26.5 based on the UACR trigger -17.0 -22.7 3 of 25 patients received a 2nd injection N= 13 13 11 9 6 5 3 N= 9 7 5 2 2 based on the eGFR trigger N= 3 3 3 2 2 Months 26 Interim analysis; Data points are mean +/- SEM ; Data as of May 7, 2024
Approximate Change in eGFR in Canagliflozin and Empagliflozin Clinical Trials SGLT2 Inhibitors Do Not Prevent Progression of Advanced CKD and Patients Lose ~4 to 7 eGFR in the First 18 Months Canagliflozin (CREDENCE) Empagliflozin (EMPA-KIDNEY) Approx. Change in Approx. Change in 2 Canagliflozin Placebo 2 Empagliflozin Placebo eGFR (ml/min/1.73m ) eGFR (ml/min/1.73m ) Month 12 -5.5 to -6 -5.5 to -6 Month 12 -3 -2.5 ) 2 Month 18 -7 -8 Month 18 -4 -4 ) 2 73m Month 24 -8 -10 Month 24 -4.5 -4.5 to -5 . 73m (ml/min/1 . (ml/min/1 GFR GFR Estimated in Change Estimated 27 1. Standard of care includes ACE inhibitors, angiotensin receptor blockers and SGLT2 inhibitors 2. Perkovic V et al. N Eng J Med 2019 3. Herrington et al. N Engl J Med 2023