创新药周报20250713： 证券研究报告 证券分析师刘浩 执业编号：S0360520120002 邮箱：liuhao@hcyjs.com 联系人段江瑶 邮箱：duanjiangyao@hcyjs.com @2021 华创版权所有 第一部分 本周创新药重点关注 国内创新药回顾 02 全球首个口服HAE急性发作按需治疗药物获批上市 本报告由华创证券有限责任公司编制 卖的出价或询价。本报告所载信息均为个人观点，并不构成对所涉及证券的个人投资建议。 请仔细阅读PPT后部分的分析师声明及免责声明。 2025-07-13 全球新药速递 03 01 遗传性血管性水肿（HAE） 证券研究报告 致。 ┃ HAE诊断流程 ┃ HAE分型及特征 | HAE 分型 | 发病机制 | C1-INH 浓度 | CI-INH 功能 | | --- | --- | --- | --- | | HAE-C1-INH 1 型 | C1-INH 基因突变 | 降低 | 降低 | | HAE-C1-INH2 型 | C1-INH 基因突变 | 正常或增高 | 障低 | | HAE-nC1-INH | FXII、 ANGPTI、PLG 基因突变 | ...

Key Takeaways PROK surged 515% on positive Phase II data for rilparencel in diabetic chronic kidney disease patients. Group 1 showed a 78% improvement in kidney decline rate, meeting statistical and clinical benchmarks. ProKidney will seek FDA confirmation of eGFR slope as a surrogate endpoint in its ongoing Phase III trial.Shares of ProKidney Corp. (PROK) skyrocketed 515% yesterday after the company reported encouraging top-line data from the phase II REGEN-007 study evaluating its lead pipeline drug, ri ...

Core Viewpoint - ProKidney Corp. has experienced significant stock volatility following positive Phase 2 clinical trial results for its REGEN-007 study, which shows promise for treating chronic kidney disease (CKD) and diabetes [2][5]. Group 1: Clinical Trial Outcomes - The Phase 2 trial results indicated a 78% improvement in the annual decline of estimated glomerular filtration rate (eGFR) for Group 1 participants who received two rilparencel injections [6]. - Group 2 participants, who received a single injection with a conditional second injection, showed a 50% improvement in annual eGFR decline, although this result was not statistically significant [6]. - These findings represent a significant clinical milestone, as CKD affects millions globally and treatment options are limited [2]. Group 2: Market Response and Stock Performance - Following the positive trial results, ProKidney's stock surged over 500% on July 8, although it remains over 70% below its 2023 peak of approximately $13 [2]. - Current analyst consensus price targets are around $4.00, suggesting the stock may be fairly valued at its current levels [3]. Group 3: Financial Position - ProKidney holds a robust cash balance of $358 million, representing 81% of its total assets, and has a minimal debt load of $3.2 million, resulting in a debt-to-equity ratio below 1% [3]. - The company incurred operating expenditures totaling $185 million over the past year and has no commercial products generating revenue, necessitating continuous observation of its cash burn rate [4]. Group 4: Risks and Challenges - The uncertainty surrounding the Phase 3 trial poses a significant risk, as success in Phase 2 does not guarantee success in subsequent phases or regulatory approvals [5]. - ProKidney faces competitive risks from other companies developing therapies for CKD and has a limited pipeline reliant on rilparencel, which adds to its risk profile [5]. - The stock has shown substantial price fluctuations, with a notable decline of 91% from a high of $13.78 on March 13, 2023, to $1.21 on November 17, 2023 [5].

Core Viewpoint - ProKidney Corp. is actively engaging with investors through participation in the H.C. Wainwright 4 Annual Kidney Virtual Conference, highlighting its focus on chronic kidney disease and its innovative cellular therapy approach [1]. Company Overview - ProKidney Corp. is a pioneer in chronic kidney disease treatment through cellular therapy innovations, founded in 2015 after a decade of research [2]. - The company's lead product candidate, rilparencel (REACT), is a first-in-class autologous cellular therapy currently in Phase 2 and Phase 3 studies aimed at preserving kidney function in diabetic patients at high risk of kidney failure [2]. - Rilparencel has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, indicating its potential significance in the treatment landscape [2].

ProKidney (PROK 515.00%) shares closed at $3.73, rising 515% today while the S&P 500 fell 0.1% and the Dow Jones Industrial Average dropped 0.4%.The chronic kidney disease-focused biotech experienced significant volatility, trading between $0.46 and $4.86 before settling near its upper range. The gain follows ProKidney's announcement of statistically and clinically meaningful topline results from its Phase 2 REGEN-007 clinical trial evaluating is autologous cell therapy treatment, called rilparencel, in pat ...

Market Overview - U.S. stocks showed mixed performance with the Dow Jones index declining by 0.35% to 44,249.09, while the NASDAQ increased by 0.07% to 20,427.63 and the S&P 500 fell by 0.03% to 6,228.39 [1] - Energy shares experienced a notable increase of 2.1%, while utilities stocks decreased by 1.9% [1] Used Vehicle Market - The US Manheim Used Vehicle Value Index rose by 1.6% month-over-month for June, following a 1.4% decline in May [2][11] Commodity Market - Oil prices increased by 0.3% to $68.14, while gold prices decreased by 1.1% to $3,306.90. Silver fell by 0.8% to $36.625, and copper dropped by 0.5% to $5.0000 [5] European Market - European shares showed positive movement with the eurozone's STOXX 600 rising by 0.40%, Spain's IBEX 35 Index up by 0.10%, London's FTSE 100 gaining 0.46%, Germany's DAX 40 increasing by 0.68%, and France's CAC 40 rising by 0.46% [6] Asian Market - Asian markets closed higher, with Japan's Nikkei gaining 0.26%, Hong Kong's Hang Seng climbing 1.09%, China's Shanghai Composite increasing by 0.70%, and India's BSE Sensex rising by 0.32% [7] Company News - ENDRA Life Sciences Inc. saw its shares surge by 134% to $8.10 after announcing a US patent for a "Radio Frequency Applicator" [9] - ProKidney Corp. shares surged by 218% to $1.95 following positive topline results from its Phase 2 REGEN-007 trial for chronic kidney disease and diabetes [9] - Humacyte, Inc. shares increased by 20% to $2.5750 after receiving Electronic Catalog Listing approval from the US Defense Logistics Agency [9] - Blue Gold Limited shares dropped by 21% to $58.75 after announcing the formation of its inaugural Advisory Board for Blockchain Evolution [9] - Sunrun Inc. shares fell by 11% to $9.84 after President Trump signed an executive order to end clean-energy tax credits [9] - Apogee Therapeutics, Inc. shares decreased by 8% to $36.24 following the release of data from its Phase 2 APEX clinical trial for atopic dermatitis [9]

Core Insights - ProKidney Corp. reported positive topline results from the Phase 2 REGEN-007 trial of rilparencel, showing significant improvement in kidney function for patients with chronic kidney disease (CKD) and diabetes [1][3][4] Group 1: Trial Results - In Group 1 (n=24), the annual decline in estimated glomerular filtration rate (eGFR) improved by 78%, from -5.8 mL/min/1.73m² to -1.3 mL/min/1.73m² after rilparencel injections, with a statistically significant difference of 4.6 mL/min/1.73m² per year (p<0.001) [3] - Of the 24 patients in Group 1, 15 (63%) met the key Phase 3 PROACT 1 inclusion criteria, showing similar efficacy results to the full Group 1 [4] Group 2: Additional Findings - In Group 2 (n=25), the eGFR slope improved by 50%, from -3.4 mL/min/1.73m² to -1.7 mL/min/1.73m², with a 1.7 mL/min/1.73m² per year difference that was not statistically significant (p=0.085) [5] - Of the 25 patients in Group 2, 15 (60%) received a second rilparencel injection after approximately 11 months, with no serious adverse events reported [6] Regulatory and Market Response - An FDA Type B meeting is scheduled for this summer to discuss the use of eGFR slope as a surrogate endpoint in the ongoing Phase 3 PROACT 1 study, aiming for accelerated approval [7] - Following the announcement, PROK stock rose by 56.6% to $0.95 during the premarket session [7]

Full results from REGEN-007 are being held and will be submitted to the American Society of Nephrology 2025 Kidney Week as a late-breaking clinical trialIn Group 1 (n=24), kidney function stabilized in patients randomized to receive two rilparencel injections (one in each kidney). The annual decline in eGFR slope improved by 78% from -5.8 mL/min/1.73m2 in the pre-injection period to -1.3 mL/min/1.73m2 in the period following the last rilparencel injection. This 4.6 mL/min/1.73m2 per year difference was stat ...

Core Points - ProKidney Corp. has completed the domestication process, changing its jurisdiction of incorporation from the Cayman Islands to Delaware, effective July 1, 2025 [1] - Shareholders approved the domestication at the Annual General Meeting on May 29, 2025, with Class A and Class B ordinary shares converting to common stock [1] - ProKidney's common stock will begin trading on Nasdaq under the unchanged symbol "PROK" starting July 2, 2025 [1] - The company does not expect any impact on day-to-day business operations due to the domestication [1] Company Overview - ProKidney is focused on chronic kidney disease (CKD) and is a pioneer in cellular therapy, founded in 2015 after a decade of research [3] - The lead product candidate, rilparencel (REACT), is a first-in-class autologous cellular therapy in Phase 2 and Phase 3 studies aimed at preserving kidney function in diabetic patients at high risk of kidney failure [3] - Rilparencel has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA [3]

Core Insights - ProKidney Corp. reported financial results for Q1 2025, highlighting its focus on chronic kidney disease (CKD) and upcoming milestones in clinical trials [1][2] Financial Highlights - As of March 31, 2025, ProKidney had cash, cash equivalents, and marketable securities totaling $328.5 million, down from $358.3 million at the end of 2024, which is expected to fund operations into mid-2027 [4][8] - Research and development expenses were $27.3 million for Q1 2025, slightly up from $27.2 million in Q1 2024, with increases in cash compensation and facility costs offset by a decrease in clinical trial costs [5] - General and administrative expenses rose to $14.4 million in Q1 2025 from $12.8 million in Q1 2024, primarily due to higher cash compensation and professional fees [6] - The net loss before noncontrolling interest was $38.0 million for Q1 2025, compared to $35.3 million for the same period in 2024 [7] Clinical Development - The company is approaching key milestones, including the release of full Group 1 data from the Phase 2 REGEN-007 study and a regulatory update on the accelerated approval pathway for rilparencel following a Type B meeting with the FDA [2][8] - Full data from Group 1 of the Phase 2 REGEN-007 study is expected in Q2 2025, involving approximately 20 patients with an average follow-up of 18 months [9] - The Phase 3 REGEN-006 (PROACT 1) clinical trial is ongoing, focusing on patients with type 2 diabetes and advanced CKD, with a total planned enrollment of approximately 685 subjects [10] Company Overview - ProKidney Corp. specializes in cellular therapeutics for chronic kidney disease, with its lead product candidate, rilparencel, being evaluated in late-stage clinical trials [12]