A Phase 2a Study to Evaluate Safety and Tolerability After Single Administration of PER-001 Intravitreal Implant in Participants With Diabetic Retinopathy

This is a Phase 2a, randomized, single-masked (participant), sham controlled clinical study.

开始日期2023-08-05

申办/合作机构

Perfuse Therapeutics, Inc.

NCT05822245

/ Active, not recruiting临床1/2期

A Phase 1/2a Study to Evaluate Safety, Tolerability, and Pharmacodynamic Effect After Single Administration of PER-001 Intravitreal Implant in Participants With Open-Angle Glaucoma

This clinical study has two phases: Phase 1 and Phase 2a. Phase 1 is an open-label, single ascending dose (SAD), while Phase 2a is a randomized, single-masked (participant) with a sham control.

开始日期2023-05-25

申办/合作机构

Perfuse Therapeutics, Inc.

100 项与 PER-001 相关的临床结果

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100 项与 PER-001 相关的转化医学

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100 项与 PER-001 相关的专利（医药）

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项与 PER-001 相关的文献（医药）

2019-06-12·Endocrine, metabolic & immune disorders drug targets

Prevalence of Extended-Spectrum β-Lactamases in Multi-drug ResistantPseudomonas aeruginosafrom Diabetic Foot Patients

Article

作者: Arshad, Muhammad ; Sehgal, Sheikh Arslan ; Qudus, Muhammad Suhaib ; Ul Hassan, Faaiz ; Siraj, Sami ; Mumtaz, Shahina ; Ul Haq, Khayam ; Khan, Momin ; Ahmed, Jawad

Introduction::

Pseudomonas aeruginosa is one of the major pathogens associated with the acute tissue damage in patients having Diabetic Foot Ulcer (DFU). The treatment of such infections can be an uphill battle due to the serious resistance to all the mainstay antibiotics, owing to overzealous production of Extended-Spectrum Beta-Lactamases (ESBLs). Pakistan also has a high prevalence of diabetes and complications related to it, however genetic disposition of the pathogens remains underinvestigated.

Aim::

The main objective of the study was to determine the frequency of ESBLs in Multi-drug resistant P. aeruginosa from diabetic foot patients.

Methods::

The duration of the present study was one year and 100 patients having DFU were enrolled. All the pus samples were subjected to the bacterial culture, gram staining, catalase test, oxidase test and antimicrobial susceptibility pattern to various antibiotics for the confirmation of P. aeruginosa. Of 23 positive isolates of P. aeruginosa, 10 were ESBLs positive as detected by double disk diffusion test. The positive ESBL strain shows an increase of ≥5mm in the zone of inhibition of the combination discs in comparison to the alone ceftazidime disc.

Results::

The ESBLs positive strains were also tested for TEM-1, SHV-1, PER-1, and VEB-1, where: (07/10) strains carried SHV-1, (05/10) strains were positive for TEM-1, while none of the isolates were PCR-positive for PER-1 and VEB-1.

Conclusion::

The findings of the current study show a difference in the pattern of ESBL genes compared to that of other such endeavors. The present study also warrants the PCR-based detection of the type of ESBL as a potential factor to consider in deciding the therapeutic strategy at any point during the treatment.

1998-12-01·The Journal of hospital infection3区 · 医学

An outbreak of extended-spectrum, β-lactamase-producing Salmonella senftenberg in a burns ward

3区 · 医学

Review

作者: G.L French ; B.K. Jain ; P.D. Stapleton ; K.P. Shannon ; G. Revathi

A strain of Salmonella senftenberg resistant to ceftazidime, gentamicin, chloramphenicol and ciprofloxacin was isolated from burn wounds of eight patients on a burns ward of a hospital in Delhi, India. The organism, which had probably been spread from patient to patient on staff hands, produced the extended-spectrum beta-lactamase SHV-5 and the aminoglycoside-modifying enzymes AAC(3)II + AAC(6'). The strain was not isolated from stool cultures of any of the patients or staff, apart from the index patient who had a history of diarrhoea and fever before admission. The outbreak ended in three weeks, after the implementation of strict handwashing. This is the first report of SHV-5 beta-lactamase in Salmonella spp. and also the first report of SHV-5 in India. The extended-spectrum beta-lactamases that have been reported in Salmonella spp. now include the Group 2 be enzymes SHV-2, SHV-5, TEM-3, TEM-25, TEM-27, CTX-M2, PER-1 and PER-2, and the Group 1 enzymes DHA-1 and CMY-2. The types of extended-spectrum beta-lactamases produced by salmonellas, their association with aminoglycoside resistance and their geographical distribution are now similar to those seen in klebsiella. Increasing antibiotic resistance in these organisms is reducing therapeutic options for the treatment of invasive disease.

1974-03-01·Molecular & general genetics : MGG

A perithecial color mutant of Neurospora crassa

Article

作者: Ernest W. Benson ; H. Branch Howe

A mutation, per-1, was found in N. crassa which caused orange instead of black perithecia when the protoperithecial parent contained the per-1 allele. No effect of per-1 was found on vegetative morphology or nutritional requirements. The per-1 locus was mapped proximal to iv (?6201) in the right arm of linkage group V.

项与 PER-001 相关的新闻（医药）

2025-05-06

·PRNewswire

Perfuse Therapeutics Announces Positive Results from the Completed Phase 1/2a Clinical Trial of PER-001 Intravitreal Implant in Patients with Glaucoma

– A single intravitreal administration of PER-001 improved both visual function and anatomic structure compared to control at 24 weeks – – These data support the potential of PER-001 as the first disease-modifying treatment for glaucoma – –PER-001 improved ocular blood flow, validating target engagement and the sustained release profile of PER-001, enabling a convenient six-month dosing frequency – – PER-001 was safe and well-tolerated over 24 weeks – – Company plans to initiate a pivotal-enabling Phase 2b trial in the second half of 2025 – SAN FRANCISCO, May 6, 2025 /PRNewswire/ -- Perfuse Therapeutics, Inc. ("Perfuse Therapeutics"), a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases, today announced positive 24-week results from its completed Phase 1/2a trial of PER-001, a first in class endothelin antagonist intravitreal implant for glaucoma. Steven Mansberger, MD, MPH, Chief of Ophthalmology and Director of Glaucoma Services at Legacy Devers Eye Institute, and an Investigator in the study, shared the results today in an oral presentation at the Association for Research in Vision and Ophthalmology (ARVO) Meeting. Data from the trial showed a single intravitreal administration of PER-001, added to existing standard-of-care intraocular pressure (IOP) reducing therapies, increased ocular blood flow and improved both visual function and anatomic structure, each of which are registrable outcomes, as measured by visual field (VF) sensitivity and optical coherence tomography (OCT) retinal nerve fiber layer (RNFL) thickness. PER-001 was safe and well-tolerated over the six-month study period. "Our mission is to develop the first disease-modifying treatment for glaucoma, the leading cause of irreversible global blindness with no approved therapies," said Sevgi Gurkan, MD, CEO and Founder of Perfuse Therapeutics. "These data are very promising, with PER-001 improving vision in glaucoma patients. We plan to advance the clinical development of PER-001, with initiation of an adaptive Phase 2b trial in glaucoma in the second half of 2025 that we believe could serve as one of two pivotal studies required for registration." "For decades, glaucoma treatments have focused on lowering intraocular pressure (IOP). Yet, many patients still experience vision loss and progress to blindness," said Dr. Mansberger. "What is particularly remarkable is that treatment with PER-001 led to improvements in ocular blood flow, optic nerve structure, and visual function. These findings suggest a potentially transformative approach for the treatment of glaucoma by targeting the endothelin pathway, a well-established mediator of vascular dysfunction in the disease. Notably, this represents the first time endothelin antagonism has been studied in the human eye, marking the potential start of an exciting new era of treatment for patients with glaucoma." PER-001 Phase 1/2a Study Design The Phase 1/2a clinical trial was a multi-center two-part trial designed to assess the safety and tolerability of the PER-001 intravitreal implant in patients with glaucoma. Exploratory objectives included pharmacodynamic (optic nerve head blood flow), functional (VF), and structural (OCT RNFL) effects of PER-001. The Phase 1 part of the trial, a first-in-human, open-label, single ascending dose (SAD) study, assessed the safety and tolerability of two doses of a single PER-001 administration in six patients with advanced glaucoma (n=3 per dose). Phase 2a, a randomized, patient and reading center masked study, evaluated the PER-001 low and high dose against sham control in patients with progressive mild-to-moderate glaucoma (n=approximately 8 for each of the low dose, high dose, and sham control groups). A total of 33 participants were enrolled and received a single intravitreal PER-001 administration or sham injection while continuing their existing pre-study IOP-lowering treatments. Participants were followed for 24 weeks after dosing. PER-001 Safety Results For the Phase 1/2a study, the 24-week data demonstrated that PER-001 was safe and well-tolerated. There were two drug-related adverse events of vitreous floaters, both of which were mild, intermittent, and self-resolved. No other drug-related adverse events or serious adverse events were reported. Additionally, there were no reports of endophthalmitis, intraocular inflammation, uveitis, or retinal vasculitis. There were no changes in IOP therapy, and best-corrected visual acuity was stable during the trial. PER-001 Exploratory Efficacy Results Increased ocular blood flow (Mean Blur Rate). For patients treated with PER-001, a clinically meaningful increase in optic nerve head blood flow from baseline was observed starting at Week 1 and was sustained out to Week 24 for both doses combined across Phase 1/2a. Improved function (VF). Patients treated with PER-001 showed improved vision; the improvement in VF mean deviation from baseline was +1.5 and +1.1 decibel (dB) per year at 24 weeks for high dose and low dose, respectively, in Phase 2a. In contrast, the sham control group worsened in VF mean deviation of –1.2 dB per year over the same period. Improved structure (OCT RNFL). Patients treated with PER-001 showed improved anatomic structure; the increase in RNFL thickness from baseline was +7.7 and +2.4 microns per year for low dose and high dose, respectively, as measured by OCT at 24 weeks in Phase 2a. In contrast, the sham control group had a decrease in RNFL thickness of –6.8 microns per year over the same period. There was a strong, linear relationship between the functional (VF) and structural (RNFL) improvements (Pearson Correlation Coefficient = 0.721; p = 0.0003; R² = 0.52). The presentation, "PER-001, an endothelin antagonist, increased optic nerve head blood flow with structural and functional improvements in patients with glaucoma", is available under the News section of the Company's website. The abstract can be found on the ARVO website. About PER-001 Intravitreal Implant, a Long-Acting Endothelin Receptor Antagonist PER-001 is a novel (new chemical entity), first-in-class small molecule endothelin receptor antagonist. Endothelin is the most potent vasoconstrictor in the human body and is upregulated in glaucoma. Endothelin is involved in inflammation and cell death through its receptors expressed in retinal vascular and neuroretinal cells. The PER-001 intravitreal implant is a bio-erodible implant administered into the vitreous cavity of the eye using a single-use, 25-gauge applicator and designed to provide a sustained release of PER-001, allowing for a convenient every 6 months dosing frequency. About Open-Angle Glaucoma In the United States, approximately four million people are diagnosed with glaucoma, and its prevalence is projected to double within the next 15 years. The current standard of care is solely directed towards lowering intra-ocular pressure (IOP), the only known modifiable risk factor. Despite the availability of pharmacological, laser, and surgical treatments to lower IOP, there are no FDA-approved therapies for glaucoma. Unfortunately, even with effective IOP-lowering treatments, many patients continue to experience vision loss. Globally, glaucoma remains the leading cause of irreversible blindness. About Perfuse Therapeutics Perfuse Therapeutics is a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases. Enabled by its proprietary sustained-release drug delivery platform, the Company is developing a robust pipeline of novel assets against validated targets. Headquartered in South San Francisco, California, and with R&D facilities in Durham, North Carolina, Perfuse Therapeutics has established a strong team committed to advancing disease-modifying therapies to patients with ocular disease around the world. For more information, please see and on LinkedIn. Contact: Lori Rosen Red House Communications [email protected] SOURCE Perfuse Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床结果临床2期

2025-04-21

·PRNewswire

Perfuse Therapeutics Announces Oral Presentation on PER-001 Intravitreal Implant for Glaucoma at the Association for Research in Vision and Ophthalmology (ARVO) Meeting

– Oral presentation to highlight 6-month data from all cohorts of the completed Phase 1/2a clinical trial – SAN FRANCISCO, April 21, 2025 /PRNewswire/ -- Perfuse Therapeutics, Inc. ("Perfuse Therapeutics"), a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases, today announced that an oral presentation on PER-001 intravitreal implant for glaucoma has been accepted for the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2025 being held May 4 to May 8, 2025, in Salt Lake City, Utah. PER-001 is a first-in-class endothelin antagonist for ocular diseases being developed for the treatment of glaucoma and other ocular indications driven by ischemia. The oral presentation will share data from all three cohorts of patients with glaucoma in the completed Phase 1/2a clinical trial. The data expand beyond earlier findings from the Phase 1 and Phase 2a (low dose) cohorts presented at the American Glaucoma Society Meeting in March 2025, demonstrating that PER-001 is well tolerated and shows promise as the first disease-modifying therapy for glaucoma. Oral Presentation Details Title: PER-001, an endothelin antagonist, increased optic nerve head blood flow with structural and functional improvements in patients with glaucoma Date & Time: Tuesday, May 6, 2025 at 2:45–3:00 p.m. MDT Abstract Number: 3322 Session Title: Glaucoma: Ocular blood flow and vascular pathophysiology Location: Ballroom G Presenter: Steven Mansberger, MD, MPH, Chief of Ophthalmology and Director of Glaucoma Services, Legacy Devers Eye Institute About PER-001 Intravitreal Implant, a Long-Acting Endothelin Receptor Antagonist PER-001 is a novel (new chemical entity), first-in-class small molecule endothelin receptor antagonist. Endothelin is the most potent vasoconstrictor in the human body and is upregulated in glaucoma. Endothelin is involved in inflammation and cell death through its receptors expressed in retinal vascular and neuroretinal cells. The PER-001 intravitreal implant is a bio-erodible implant, administered into the vitreous cavity of the eye using a single use, 25-gauge applicator and designed to provide a sustained release of PER-001, allowing for a convenient every 6 months dosing frequency. About the Phase 1/2a Clinical Trial The Phase 1/2a is a multi-center, two-part clinical trial designed to assess the safety, tolerability and pharmacodynamics of PER-001 in patients with glaucoma. The Phase 1 part of the trial was a first-in-human, open-label, single ascending dose (SAD) study that evaluated the safety and tolerability of two doses (low dose, high dose) of PER-001 intravitreal implant in patients with advanced glaucoma. The Phase 2a part of the trial was a patient- and reading-center masked, randomized, sham-controlled study that evaluated the safety, tolerability, and pharmacodynamics of the same two doses of PER-001 in patients with progressing, mild to moderate glaucoma. All patients received a single intravitreal administration of PER-001 while maintaining their pre-study intraocular pressure (IOP)-lowering therapies. In the Phase 1 SAD part of the trial, two doses of PER-001 intravitreal implant were tested in patients (n=6). In the Phase 2a part of the trial, the same two doses, along with a sham, were subsequently tested in patients (n=27). All patients were followed for 24 weeks after receiving a single dose. About Perfuse Therapeutics, Inc. Perfuse Therapeutics is a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases. Enabled by its proprietary sustained release drug delivery platform, the Company is developing a robust pipeline of novel assets against validated targets. Headquartered in South San Francisco, California and with R&D facilities in Durham, North Carolina, Perfuse Therapeutics has established a strong team committed to advancing disease modifying therapies to patients with ocular disease around the world. For more information, please see and on LinkedIn. Contact Information: Lori Rosen Red House Communications [email protected] SOURCE Perfuse Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床1期临床结果临床2期

2025-03-09

·药明康德

快速持久降解目标蛋白超80%的小分子疗法；有望使患者停止激素替代治疗的创新生物器官疗法…… | 一周盘点

▎药明康德内容团队编辑本期看点 1. 小分子蛋白降解剂BHV-1300的1期临床试验结果积极，目标IgG快速、深度和持续地降低，降幅最高达84%，中位降幅为80%。 2. 创新生物混合器官Cell Pouch获批一项新的IND申请，它与自体甲状腺细胞疗法相结合，旨在恢复自然的甲状腺功能，使患者无需使用甲状腺激素替代药物或免疫抑制疗法。 3. 巨噬细胞疗法Allocetra治疗中度至重度膝骨关节炎，在一项1/2期临床试验中使患者报告的疼痛平均减少47.0%，关节功能改善46%。药明康德内容团队整理 BHV-1300：公布1期临床试验的新数据 Biohaven公司公布了其皮下给药的在研小分子蛋白降解剂BHV-1300的1期临床试验的新结果。BHV-1300在选择性清除目标IgG（IgG1、IgG2和IgG4）方面具有独特优势，同时保留IgG3水平。IgG3在对抗细菌、寄生虫和病毒中发挥关键作用。BHV-1300的设计目标是避免与其他IgG降低方法相关的广泛免疫抑制，同时在治疗自身免疫性疾病的过程中允许免疫系统对潜在感染做出反应。在为期四周的1期研究中，每周皮下注射1000 mg剂量的BHV-1300可使总IgG快速、深度和持续地降低，降幅最高达84%，中位降幅为80%。在每次给药后的数小时内，患者的IgG就会逐渐降低，并且与基线值相比，在四周内持续降低。BHV-1300在高达2000 mg的皮下给药剂量下安全性和耐受性良好。大多数不良事件轻微，可自行消退，没有因与研究药物相关的不良事件而停药，也没有严重的不良事件。 ▲BHV-1300的作用方式和1期临床试验结果（图片来源：参考资料[7]） Cell Pouch：IND申请获得FDA许可 Sernova Biotherapeutics公司宣布，美国FDA已批准其IND申请，允许在因结节性甲状腺疾病接受甲状腺手术的患者中评估Cell Pouch与自体移植甲状腺细胞的联合疗法。Sernova公司的Cell Pouch是一种创新的生物混合器官，由非生物材料与活体组织结合而成，旨在恢复或增强受损器官的功能。Cell Pouch与自体甲状腺细胞疗法相结合，旨在通过提供一种稳定、长期的生理解决方案，恢复自然甲状腺功能，而无需使用甲状腺激素替代药物或免疫抑制疗法。临床前研究表明，在没有免疫抑制的情况下，将甲状腺组织重新植入预先血管化的Cell Pouch中，成功地将两种主要的甲状腺激素——甲状腺素（T4）和三碘甲状腺原氨酸（T3）恢复到甲状腺切除术前水平，而无需激素补充。放射性同位素成像和组织学分析证实，植入后5个月内Cell Pouch内存在健康、功能性的甲状腺组织。 Allocetra：公布1/2期临床试验的中期数据 Enlivex Therapeutics公司公布了其巨噬细胞疗法Allocetra治疗中度至重度膝骨关节炎的1/2期临床试验的中期数据。败血症、骨关节炎、银屑病关节炎等疾病会使巨噬细胞失去平衡状态。这些非稳态巨噬细胞在很大程度上导致了相应疾病的严重性。Allocetra是一种通用、现货型细胞疗法，通过对巨噬细胞进行重编程，旨在恢复其稳态（homeostasis），进而治疗疾病。Allocetra提供创新的免疫调节机制，具潜力作为单药或组合疗法治疗这些具未竟医疗需求的疾病。此次公布的结果显示，治疗后第6个月，患者报告的疼痛平均显著减少了47.0%（P=0.0001），关节功能改善了46%。此外，83%接受治疗的患者在6个月时仍被认为对治疗有应答。这表明Allocetra作为骨关节炎的潜在突破性治疗方法，具有良好的持久性和持续疗效。安全性方面，没有报告严重的不良事件。 PER-001：公布1/2a期临床试验数据 Perfuse Therapeutics公司公布了其玻璃体内植入疗法PER-001在青光眼患者中的首次人体1/2a期试验的24周数据。PER-001是一种新型的小分子长效内皮素受体拮抗剂。内皮素是人体内最强的血管收缩剂，在青光眼中上调，并通过其在视网膜血管和神经视网膜细胞中的受体参与炎症和细胞死亡。PER-001是一种生物可降解的植入疗法，通过一次性25号注射器注入眼球玻璃体腔中。该疗法被设计为每6个月给药一次，能够持续释放药物。 24周的数据表明，PER-001具有良好的耐受性，仅有一例药物相关不良事件（轻微且短暂的玻璃体飞蚊症），没有严重药物相关不良事件或眼内炎症报告。接受PER-001治疗的患者从第1周开始，视神经头血流量较基线平均增加了至少10%，并持续至第24周。第24周时，光学相干断层扫描（OCT）显示，PER-001治疗组患者的视野平均偏差改善了1 dB/年，视网膜神经纤维层厚度平均增加了3微米。相比之下，假手术对照组患者的视野平均偏差下降了0.63 dB/年，OCT视网膜神经纤维层厚度减少了1.3微米。这些结果表明，PER-001在青光眼患者中不仅安全性良好，还显示出显著的功能和结构改善，为其作为青光眼治疗的潜在疗法提供了概念验证。 RPESC-RPE-4W：公布1/2a期临床试验数据 Luxa Biotechnology公司公布了一项1/2a期临床试验的数据，该试验评估了其专有的视网膜色素上皮（RPE）细胞疗法RPESC-RPE-4W在干性年龄相关性黄斑变性（AMD）患者中的应用。RPESC-RPE-4W是一种基于成人RPE干细胞的疗法，旨在通过替换受损的RPE细胞来恢复视力功能。干性AMD是一种导致中心视力模糊或丧失的退行性眼病，目前尚无有效治疗方法。在1/2a期试验的最低剂量组中，6名干性AMD患者被分为视力较差组和视力较好组。视力较差组的3名患者在12个月的随访中，平均在ETDRS视力表上获得了21.67个字母的改善，视力较好组的3名患者在3个月时，平均改善了3.3个字母。 VXA-G1.1-NN：公布1b期临床试验数据 Vaxart公司宣布，其第一代口服诺如病毒候选疫苗VXA-G1.1-NN在老年人群（55-80岁）中的1b期临床试验完整数据已发表在学术期刊Science Translational Medicine上。VXA-G1.1-NN是一种口服疫苗片剂，将表达诺如病毒GI.1主要衣壳蛋白VP1的非复制性腺病毒载体递送到小肠，旨在预防由诺如病毒引起的急性胃肠炎。该试验评估了VXA-G1.1-NN在55-65岁和66-80岁两个健康老年人群中的安全性和免疫原性。受试者以2：1的比例随机接受疫苗或安慰剂，并在第28天进行加强接种。63名志愿者完成了研究，安全性及免疫原性评估持续至第390天。免疫原性结果显示，与安慰剂相比，疫苗在所有三个剂量组中均诱导了强效且持久的血清抗体反应，第29天和第57天的血清抗VP1 IgA水平显著增加，且抗VP1 IgG水平也显著高于接种前水平，呈剂量依赖性增加。口服疫苗还刺激了唾液和鼻腔中强烈且持久的IgA反应。安全性方面，疫苗在老年人群中表现出良好的安全性和耐受性，未发生与疫苗相关的3级不良事件。最常见的不良反应为头痛（14%）和不适/疲劳（16%），与安慰剂组的发生率相似。 APG990：公布1期临床试验的中期数据 Apogee Therapeutics公司公布了其新型半衰期延长的OX40L单克隆抗体APG990的首个人体试验的积极中期结果。APG990是一种皮下注射疗法，靶向炎症通路中位于IL-13上游的OX40L，从而对1型、2型和3型炎症通路产生广泛影响。此次公布的结果显示，APG990的半衰期约为60天，可支持每3个月或6个月一次的维持给药方案。在所有5个剂量组（最高剂量为1200 mg）中，APG990均表现出良好的耐受性。Apogee公司还开发了APG279，即APG777（一种深度抑制2型炎症的药物）与APG990的联合制剂，旨在实现单次注射2 mL联合制剂，每年仅需给药2-4次的治疗方案，为患者提供更有效的治疗选择，同时最大限度地减少其他可用疗法的副作用。 BL-001：公布1期临床试验数据 Bloom Science公司公布了其在研口服疗法BL-001在一项涉及32名健康成年人（体重从正常到超重）的1期试验中的疗效和安全性结果。BL-001是一种潜在“first-in-class”、口服给药的活生物治疗产品（LBP），旨在模拟生酮饮食的治疗效果。临床前数据表明，BL-001在癫痫模型中复制了生酮饮食的治疗效果，并进一步展示了其对γ-氨基丁酸（GABA）和其他关键生物能途径的调节作用，以抑制癫痫发作的关键驱动因素——过度兴奋性。 BL-001在28天的治疗期内在体重减轻方面的整体治疗效果具有统计学显著性（p=0.0424）。超重组（BMI≥25）中接受BL-001治疗的受试者与所有接受安慰剂的受试者相比，体重的百分比变化具有统计学意义（p=0.0007）。此外，BL-001治疗的超重和正常体重个体之间的体重百分比变化也具有统计学意义（p=0.0003），体重减轻最高为4.9%。80%在BL-001治疗期间体重减轻的受试者在最后一次给药两周后仍保持体重减轻，其中接受最高剂量的所有超重受试者的体重继续下降，在两周随访期结束时体重减少了3.4%。此外，BL-001治疗组观察到了剂量依赖性的变化，包括尿酮、食欲降低和体重减轻，而安慰剂组未观察到这些变化。靶向生物标志物分析显示，与食欲控制、代谢调节和消化相关的关键代谢激素发生了具有统计学意义的变化。更大规模的代谢组学分析还揭示了循环酮体的统计学显著变化，这些发现进一步支持了BL-001的潜在生酮和减肥效果。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看） [1] Perfuse Therapeutics Announces Positive Visual, Structural and Safety Data from a Ph1/2a Trial of PER-001 in Glaucoma Patients at the American Glaucoma Society Meeting. Retrieved March 7, 2025, from https://www.prnewswire.com/news-releases/perfuse-therapeutics-announces-positive-visual-structural-and-safety-data-from-a-ph12a-trial-of-per-001-in-glaucoma-patients-at-the-american-glaucoma-society-meeting-302387598.html [2] Sernova Biotherapeutics Receives FDA Clearance for IND Application for its Cell Pouch Bio-hybrid Organ with Autograft Thyroid Cells in Patients with Hypothyroidism. Retrieved March 7, 2025, from https://www.globenewswire.com/news-release/2025/03/03/3035709/0/en/Sernova-Biotherapeutics-Receives-FDA-Clearance-for-IND-Application-for-its-Cell-Pouch-Bio-hybrid-Organ-with-Autograft-Thyroid-Cells-in-Patients-with-Hypothyroidism.html [3] Enlivex Therapeutics Announces Investor Webinar to Discuss Positive Interim Data from Phase I/II Allocetra™ Trial in Knee Osteoarthritis. Retrieved March 7, 2025, from https://www.globenewswire.com/news-release/2025/03/04/3036995/0/en/Enlivex-Therapeutics-Announces-Investor-Webinar-to-Discuss-Positive-Interim-Data-from-Phase-I-II-Allocetra-Trial-in-Knee-Osteoarthritis.html [4] Bloom Science Reports Positive Data from Phase 1 Trial of BL-001 Demonstrating Statistically Significant, Durable Weight Loss in Overweight Individuals. Retrieved March 7, 2025, from https://www.prnewswire.com/news-releases/bloom-science-reports-positive-data-from-phase-1-trial-of-bl-001-demonstrating-statistically-significant-durable-weight-loss-in-overweight-individuals-302391446.html [5] Bioheng Therapeutics Announces FDA Clearance of IND Application for CD7 UCAR T Cell Therapy in T-ALL/LBL. Retrieved March 7, 2025, from https://www.prnewswire.com/news-releases/bioheng-therapeutics-announces-fda-clearance-of-ind-application-for-cd7-ucar-t-cell-therapy-in-t-alllbl-302392846.html [6] Neurocrine Biosciences Announces Initiation of Phase 1 Clinical Study Evaluating NBI-1140675, a Second-Generation VMAT2 Inhibitor, in Healthy Adults. Retrieved March 7, 2025, from https://www.prnewswire.com/news-releases/neurocrine-biosciences-announces-initiation-of-phase-1-clinical-study-evaluating-nbi-1140675-a-second-generation-vmat2-inhibitor-in-healthy-adults-302392294.html [7] Biohaven Reports Positive Degrader Data Achieving > 80% Sustained Reductions in Total IgG with Potential First-in-Class BHV-1300. Retrieved March 7, 2025, from https://www.prnewswire.com/news-releases/biohaven-reports-positive-degrader-data-achieving--80-sustained-reductions-in-total-igg-with-potential-first-in-class-bhv-1300-302389515.html [8] Adiso Therapeutics Announces Data Publication of Two Phase 1 Studies of First-in-Class Neutrophil Modulator ADS051 in the American Journal of Gastroenterology. Retrieved March 7, 2025, from https://www.prnewswire.com/news-releases/adiso-therapeutics-announces-data-publication-of-two-phase-1-studies-of-first-in-class-neutrophil-modulator-ads051-in-the-american-journal-of-gastroenterology-302393958.html [9] Apogee Therapeutics Announces Positive Interim Phase 1 Results from the APG990 Healthy Volunteer Trial, Unlocking Potential Maintenance Dosing Every Three and Six Months for APG279 (APG777 + APG990). Retrieved March 7, 2025, from https://www.globenewswire.com/news-release/2025/03/03/3035377/0/en/Apogee-Therapeutics-Announces-Positive-Interim-Phase-1-Results-from-the-APG990-Healthy-Volunteer-Trial-Unlocking-Potential-Maintenance-Dosing-Every-Three-and-Six-Months-for-APG279-.html [10] Acadia Pharmaceuticals and Saniona Announce Initial Positive Results from ACP-711 (formerly SAN711) Phase 1 Study. Retrieved March 7, 2025, from https://acadia.com/en-us/media/news-releases/acadia-pharmaceuticals-and-saniona-announce-initial-positive [11] Actio Biosciences Announces First Participant Dosed in Phase 1 Clinical Trial of ABS-0871, a Novel TRPV4 Inhibitor, for the Treatment of Charcot Marie Tooth Disease 2C. Retrieved March 7, 2025, from https://actiobiosciences.com/actio-biosciences-announces-first-participant-dosed-in-phase-1-clinical-trial-of-abs-0871-a-novel-trpv4-inhibitor-for-the-treatment-of-charcot-marie-tooth-disease-2c/ [12] Contineum Therapeutics Initiates Patient Dosing in Phase 1b Positron Emission Tomography (PET) Trial of PIPE-791. Retrieved March 7, 2025, from https://www.businesswire.com/news/home/20241216396391/en/Contineum-Therapeutics-Initiates-Patient-Dosing-in-Phase-1b-Positron-Emission-Tomography-PET-Trial-of-PIPE-791 [13] Phio Pharmaceuticals Announces Completion of Enrollment in Third Safety Cohort in PH-762 Phase 1b Dose-Escalation Study. Retrieved March 7, 2025, from https://phiopharma.com/phio-pharmaceuticals-announces-completion-of-enrollment-in-third-safety-cohort-in-ph-762-phase-1b-dose-escalation-study/ [14] Bambusa Therapeutics Announces First Subject Dosed in Phase 1 Clinical Trial of BBT001, a Novel Multi-Targeting, Half-Life Extended Bispecific Antibody for the Treatment of Atopic Dermatitis and Other Inflammatory Skin Diseases. Retrieved March 7, 2025, from https://www.prnewswire.com/news-releases/bambusa-therapeutics-announces-first-subject-dosed-in-phase-1-clinical-trial-of-bbt001-a-novel-multi-targeting-half-life-extended-bispecific-antibody-for-the-treatment-of-atopic-dermatitis-and-other-inflammatory-skin-diseases-302388661.html [15] Luxa Biotechnology Presents Clinical Data from First-in-Human Retinal Pigment Epithelium Cell Therapy Trial for Dry Age-Related Macular Degeneration at the 77th Annual Wills Eye Conference. Retrieved March 7, 2025, from https://www.businesswire.com/news/home/20250306998366/en/Luxa-Biotechnology-Presents-Clinical-Data-from-First-in-Human-Retinal-Pigment-Epithelium-Cell-Therapy-Trial-for-Dry-Age-Related-Macular-Degeneration-at-the-77th-Annual-Wills-Eye-Conference [16] ImmunoGenesis Doses First Patient in Phase 1/2 Clinical Trial of IMGS-101 in Combination With Balstilimab (anti-PD-1) and Zalifrelimab (anti-CTLA-4) in Relapsed or Refractory Advanced Prostate, Pancreatic, and HPV(-) Head & Neck Tumors. Retrieved March 7, 2025, from https://www.prnewswire.com/news-releases/immunogenesis-doses-first-patient-in-phase-12-clinical-trial-of-imgs-101-in-combination-with-balstilimab-anti-pd-1-and-zalifrelimab-anti-ctla-4-in-relapsed-or-refractory-advanced-prostate-pancreatic-and-hpv--head--neck-t-302393719.html [17] Vaxart Announces Publication Demonstrating the Immunogenicity and Safety of its First-Generation Oral Pill Norovirus Vaccine Candidate in Elderly Adults. Retrieved March 7, 2025, from https://investors.vaxart.com/news-releases/news-release-details/vaxart-announces-publication-demonstrating-immunogenicity-and 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

临床申请临床1期细胞疗法免疫疗法临床结果

100 项与 PER-001 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
非增殖性糖尿病视网膜病变	临床2期	美国	Perfuse Therapeutics, Inc.	2023-08-05
青光眼相关色素分散综合征	临床2期	美国	Perfuse Therapeutics, Inc.	2023-05-25
干性年龄相关性黄斑病变	药物发现	美国	Perfuse Therapeutics, Inc.	2024-07-18
地图样萎缩	药物发现	美国	Perfuse Therapeutics, Inc.	2024-07-18
视网膜静脉阻塞	药物发现	美国	Perfuse Therapeutics, Inc.	2024-07-18

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05822245 (PRNewswire) 人工标引	临床1/2期	青光眼	33	PER-001 low dose	願壓壓範繭夢膚齋壓顧(膚鏇簾蓋築蓋製遞蓋鹽) = 獵願夢齋壓夢積鏇鬱壓觸鬱淵願製鬱製網艱範 (鏇壓憲壓衊醖淵繭鑰鹽 ) 更多	积极	2025-05-06
				PER-001 high dose	願壓壓範繭夢膚齋壓顧(膚鏇簾蓋築蓋製遞蓋鹽) = 齋遞築構窪餘膚廠網鏇觸鬱淵願製鬱製網艱範 (鏇壓憲壓衊醖淵繭鑰鹽 ) 更多