A 2-Part, Open-Label, Phase 2, Multiple Dose Study to Evaluate the Pharmacodynamic Effects, Safety, and Tolerability of Patiromer in Children Under 12 Years of Age With Hyperkalaemia (EMERALD2)

A study to evaluate the pharmacodynamic effects, safety, and tolerability of patiromer in children under 12 years of age with hyperkalaemia

开始日期2024-09-01

申办/合作机构

Vifor Pharma AG

NCT06256991 / Recruiting临床4期

Potassium Correction for Renin-angiotensin-aldosterone System Optimization in Chronic Kidney Disease

The goal of this placebo-controlled, double-blinded cross-over trial is to test whether patiromer, compared with placebo, better enables up-titration of RAAS-blocker treatment in patients with chronic kidney disease stage 3b/4.
The main questions it aims to answer are:
Does patiromer allow uptitration of irbesartan, resulting in a significant reduction in albuminuria and blood pressure?
Does patiromer allow uptitration of irbesartan, resulting in a significant reduction in blood pressure?
The trial contains the following interventions:
Participants will be switched from their ACEi/ARB to a standardised dose of irbesartan (150 mg/d).
During two 12-week study periods, participants will receive either patiromer 8.4 g/d or placebo. The order of study periods is randomized.
At the start of each study period irbesartan will be up-titrated to 300 mg/d.
After 1 and 6 weeks, at both periods, plasma potassium will be measured and the irbesartan dose will be reduced to 150 mg/d in case plasma potassium exceeds 5.0 mmol/L.
At 12 weeks from the start of the study period, the endpoints will be assessed.
Between the two study periods, there is a 6-week washout. Irbesartan dose during the wash-out period will be 150mg/d. After washout, participants will switch from the patiromer arm to the placebo arm or vice versa.

开始日期2024-04-01

申办/合作机构

University Medical Center Groningen

[+5]

NCT05786469 / Terminated临床3期IIT

A Prospective, Double-blind, Randomized, Single Centre Trial to Evaluate the Rate of RAAS Inhibitor Withdrawal or Down-titration in Non-dialysis Patients with CKD Stage IIIb to V Randomized to Patiromer or Placebo (DROP)

This phase III, prospective, randomized, double-blind, placebo-controlled trial will primarily aim to compare the effects of patiromer and placebo on the rate of withdrawal or down-titration of RAAS inhibition therapy because of refractory hyperkalemia (serum K+ levels ≥ 5.5 mEq/L at two consecutive visits, one-week apart) in non-dialysis patients with CKD stage IIIB to V receiving best available conservative therapy, including RAAS inhibition with ACE inhibitors and/or ARBs and/or aldosterone antagonists. Patients are expected to be included during an 18-month recruitment period. All randomized patients will be maintained on active follow-up for 12 months. At 12 months, a final visit will be performed for all patients who complete the follow-up period. During this final visit, all the parameters evaluated at baseline will be reassessed and the study treatment will be discontinued. Whenever feasible, a final visit will be planned within one month also for those patients who prematurely discontinue the treatment period for any intercurrent reason (adverse event, consent withdrawal and other). After the final visit the patient will be discharged from the study and will be referred to his nephrologist with the suggestion to check serum potassium levels within three days.

开始日期2023-08-02

申办/合作机构

Vifor Pharma AG

100 项与帕替罗姆相关的临床结果

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100 项与帕替罗姆相关的转化医学

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100 项与帕替罗姆相关的专利（医药）

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239

项与帕替罗姆相关的文献（医药）

2024-12-31·JOURNAL OF MEDICAL ECONOMICS

Cost analysis of patiromer vs sodium zirconium cyclosilicate for the treatment of hyperkalemia in Spain and the UK

Article

作者: Hunt, Barnaby ; Jakutis, Gabrielius ; Ramirez de Arellano, Antonio

BACKGROUND:

Hyperkalemia is an electrolyte abnormality with potentially life-threatening consequences. Published data have shown that potassium-binding polymer patiromer (Veltassa) is associated with reduced rates of severe edema and hospitalization for heart failure compared with sodium zirconium cyclosilicate (SZC, Lokelma) when treating hyperkalemia. The aim of this study was to evaluate the possible costs associated with these interventions in the Spanish and UK settings.

METHODS:

A cost-analysis model was developed in Microsoft Excel to compare the costs associated with patiromer and SZC for the management of hyperkalemia. Clinical event rates were taken from a published real-world comparative study, with the base case capturing the statistically significant reduction in severe edema with patiromer vs SZC and a sensitivity analysis also including the non-statistically significant reduction in hospitalization for heart failure. Country-specific costs, expressed in 2022 Euros (EUR) and British pounds sterling (GBP), were evaluated from a healthcare payer perspective and included pharmacy costs and costs of clinical events.

RESULTS:

Patiromer may be associated with cost savings of EUR 107 and GBP 630 per patient-year of treatment vs SZC in Spain and the UK, respectively. The majority of cost savings were due to the possible lower daily cost of patiromer compared with SZC. Including the difference in heart failure hospitalization rates in a sensitivity analysis led to greater cost savings with patiromer over SZC, increasing to EUR 460 and GBP 902 in Spain and the UK, respectively. Extrapolation of patient-level economic outcomes to a population level found that patiromer was associated with annual cost savings of EUR 30.6 million in Spain, and GBP 801.7 million in the UK vs SZC.

CONCLUSIONS:

Patiromer has the potential to be cost saving vs SZC for the treatment of hyperkalemia in Spain and the UK based on the results of a real-world evidence analysis.

2024-12-31·Journal of medical economics

Cost-effectiveness analysis of sodium zirconium cyclosilicate for hyperkalemia among patients with chronic kidney disease or heart failure in Kuwait

Article

作者: Al-Yousef, Anas ; Elsisi, Gihan Hamdy ; Al-Humood, Khaldoon ; Mahmoud, Mohamed M Ibrahim

INTRODUCTION:

Our model was conducted from Kuwaiti payer's perspective to provide evidence on the cost-effectiveness of Sodium zirconium cyclosilicate (SZC) versus patiromer to correct and maintain serum potassium (K+) in combination with renin-angiotensin-aldosterone system inhibitors (RAASis) with different dose titration in patients with chronic kidney disease/heart failure (CKD/HF) with/without renal replacement therapy (RRT).

METHODOLOGY:

The model was developed as a patient-level, fixed-time increment stochastic simulation to simulate the complexity of disease, including multiple coexisting and competing conditional risks. This model was established to compare SZC versus patiromer as a treatment for hyperkalemia (HK) among adult populations with underlying conditions of advanced CKD stages 3a-5 or HF to correct and maintain serum K + over a lifetime horizon. The clinical outcomes of SZC and patiromer were demonstrated through arm-specific K + trajectories extracted from the HARMONIZE trial and OPAL-HK trial, respectively. The utility data was captured from different studies. Direct medical cost was captured from local data from Kuwaiti hospitals. Sensitivity analyses were conducted to assess the uncertainty in the model.

RESULTS:

Within different scenarios of CKD/HF, SZC was a cost-saving option, with/without RRT, whether one-off administration or repeated administration, except for one-off treatment administration among the HF cohort, which generated an incremental cost effectiveness ratio of KWD 331/quality adjusted life year (QALY). The incremental QALY of SZC ranged from 0.007 to 0.202. In addition, the savings observed with SZC fall within a range of KWD -60 to KWD -1,235 at serum K+ ≥ 5.1 mmol/L.

CONCLUSION:

The evidence generated by our model recommends the inclusion of SZC as a treatment option to correct HK and maintain normal serum K + level for CKD/HF patients within the Kuwaiti healthcare system. The costs saved from reducing frequent HK episodes, RAASis discontinuation/down titration, major cardiovascular events, and hospitalization offset the drug acquisition cost of SZC.

2024-11-01·Kidney International Reports

Randomized Trial of Patiromer on Efficacy to Reduce Episodic Hyperkalemia in Patients with ESKD Treated With Hemodialysis

Article

作者: Daubert, James P. ; Murray, Susan ; Daubert, James P ; Middleton, John P. ; Middleton, John P ; Sun, Shifeng ; Davenport, Clemontina A ; Davenport, Clemontina A.

Introduction:

Individuals with end-stage kidney disease (ESKD) maintained on hemodialysis (HD) carry a high risk of cardiac arrhythmias. This risk is heightened by episodic hyperkalemia. The purpose of the study was to investigate whether patiromer administered daily reduced episodes of hyperkalemia in those with ESKD who receive HD, and to explore whether prescription of patiromer reduced the number of significant arrhythmia events.

Methods:

This was a prospective, randomized, open-label trial. Eligible patients with ESKD on HD were identified. Participants were randomized 1:1 to patiromer versus usual care. Those randomized to patiromer were administered the medication daily, and the dose was titrated based on serum potassium concentrations at the start of weeks 1, 2, and 3. All participants received 7-day continuous cardiac monitors at baseline and at week 4.

Results:

Of the 33 participants who were randomized, 1 withdrew due to adverse symptoms, and 1 withdrew due to pregnancy, leaving 31 in our analytic cohort. The mean age of randomized participants was 56 years, 55% were male, 81% were Black, and 10% were Hispanic/Latino. In week 4, the number of episodes of serum potassium ≥ 5.5 mEq/l was 13 in the patiromer group and 41 in the control group; with median number of episodes of hyperkalemia in the patiromer group significantly lower than that of control group (0 vs. 3, P = 0.024). In week 4 continuous cardiac monitors, 6 participants had > 1000/24 h premature ventricular contractions, 5 had no sustained ventricular tachycardia (VT), 3 had atrial fibrillation, and 1 had bradycardia, with no significant differences between the groups.

Conclusion:

Patiromer administered daily reduced the frequency of hyperkalemia in study participants with ESKD who receive thrice-weekly HD. Larger studies are needed to determine whether patiromer reduces significant cardiac events.

项与帕替罗姆相关的新闻（医药）

2024-09-24

·PRNewswire

Veltassa® (patiromer) approved in Japan for the treatment of adults with hyperkalemia

Veltassa® offers effective and well-tolerated long-term potassium control in chronic kidney disease and chronic heart failure patients 1-5 Zeria Pharmaceutical Co., Ltd. to market Veltassa® in Japan ST. GALLEN, Switzerland, Sept. 24, 2024 /PRNewswire/ -- CSL Vifor today announced that Japan's Ministry of Health and Labor Welfare (MHLW) has granted its partner, Zeria Pharmaceutical Co., Ltd. (Zeria), marketing authorization approval for Veltassa® for the treatment of adult patients with hyperkalemia, a condition characterized by high levels of potassium in the blood. Veltassa® has now received marketing authorizations in 41 countries worldwide. "We are pleased that Veltassa® has been approved in Japan, and congratulate our trusted partner Zeria," said Hervé Gisserot, General Manager of CSL Vifor. "This milestone reflects our shared commitment to addressing the needs of over 300,000 patients in Japan affected by hyperkalemia6, particularly those with chronic kidney disease or heart failure. As we continue to deliver on our promise, we are excited to introduce a next generation hyperkalemia management therapy, designed to be broadly utilized across diverse patient groups." The approval is based on the marketing authorization application filing by Zeria, which was supported by positive clinical data from the Japanese clinical development program of ZG-801 (Veltassa®) conducted in patients with hyperkalemia in Japan. In 2018, CSL Vifor granted Zeria the exclusive right to develop and market Veltassa® in Japan. Zeria expects to begin to market Veltassa® following National Health Insurance price listing. About CSL Vifor CSL Vifor is a global partner of choice for pharmaceuticals and innovative, leading therapies in iron deficiency and nephrology. We specialize in strategic global partnering, in-licensing and developing, manufacturing and marketing pharmaceutical products for precision healthcare, aiming to help patients around the world lead better, healthier lives. Headquartered in St. Gallen, Switzerland, CSL Vifor also includes the joint company Vifor Fresenius Medical Care Renal Pharma (with Fresenius Medical Care). The parent company, CSL (ASX: CSL; USOTC: CSLLY), headquartered in Melbourne, Australia, employs 32,000 people and delivers its lifesaving therapies to people in more than 100 countries. For more information about CSL Vifor visit, . About hyperkalemia Hyperkalemia is a serious condition in which the amount of potassium in the blood exceeds the normal level (between 3.5 and 5.0 mmol/L)7. Usually, hyperkalemia remains asymptomatic. However, when the levels of potassium are very high or increase rapidly, people may experience heart palpitations, shortness of breath, chest pain, nausea, or vomiting 8, 9. The most common causes of high potassium include chronic kidney disease (CKD) and heart failure (HF). Medications used to delay progression of HF and CKD – renin-angiotensin aldosterone system inhibitor (RAASi) – may as well increase the risk of hyperkalemia10. About Veltassa® (patiromer) Veltassa® is a sodium-free exchange potassium binder which reduces high amounts of potassium in the blood and maintains the potassium at a normal level. Veltassa® acts within the gastrointestinal tract by exchanging potassium for calcium, primarily in the colon. The potassium is then excreted from the body through the normal excretion process. Veltassa® enables patients to manage chronic hyperkalemia, permitting them to stay on optimal and guideline-recommended doses of life-saving RAASi medications. Veltassa® has demonstrated to enable optimized RAASi therapy use across a range of placebo-controlled, randomized clinical trials in a variety of patient profiles 2,3,11. As of September 2024, Veltassa® has been approved in 41 countries worldwide, including in the U.S. and the EU. References: CSL Vifor Media Contact Thomas Hutter +41 79 957 96 73 [email protected] Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

上市批准引进/卖出

2023-12-22

·生物制药小编

医药圈：当命运的齿轮开始转动

许多年前的一笔不起眼的BD＆L，竟是促成将来重大M＆A的关键一环，如赛诺菲以29亿美元收购Provention Bio公司，核心资产来自MacroGenics公司。之前开发的诸多候选药物，要么尘归尘、土归土，要么杳无音讯，要么雷声大、雨点小，一次偶然的BD＆L，竟是促成将来重大M＆A的关键一环，如安进以283亿美元市值收购Horizon公司，核心资产来自River Vision公司。或开发顺位处于较后但未来将成为核心资产的候选药物，竟是促成将来重大M＆A的关键一环，如安进以37亿美元收购ChemoCentryx，核心资产avacopan的历史开发顺位在第4位。没错，当这些偶然的东西走进biotech聚焦视野里的时候，属于它们命运的齿轮开始悄然转动了。1. 被命运之神选中的biotech2. 结语被命运之神选中的biotech核心/领先资产，该司被收购时新闻稿首先或重点提及的资产。历史开发顺位，被收购公司提名候选药物代号或介绍候选药物的次序。几年增长几倍，该司从IPO年份到被收购时的年份，对比收购时的公司价值和IPO发行市值。命运的齿轮开始转动的年数，该司核心/领先资产首次出现的年份至被收购时的年份。资料来自招股书、年报及收购时的新闻稿。下文有点累赘，建议看几个案例后直接跳到文末底下看总结的图表。2017年Horizon公司从River Vision公司获得了核心资产抗IGF-1R 抑制剂teprotumumab，这款药物在Horizon公司的历史开发顺位里排名14。从那以后，命运的齿轮开始转动，5年后，诞生了2022年全球最大的生物医药收购案，283亿美元的公司价值让Horizon实现了11年增长160倍的神迹（粗略年化为59%）。2013年Loxo公司从Array公司获得了核心资产RET 抑制剂LOXO-292，这款药物在Loxo公司的历史开发顺位里排名第3。从那以后，命运的齿轮开始转动，6年后，诞生了80亿美元的收购案，让Loxo实现了5年增长37.6倍的神迹（粗略年化为108%）。插多一句，这笔收购案也牵涉到后来全球首个的第三代BTK抑制剂pirtobrutinib。Pirtobrutinib由Redx公司发现，2016年10月提名为RXC005，当时正处于债务危机的Redx公司急需现金，在2017年7月份不得不以4000万美元将RXC005一次性出售给Loxo公司，后面不会涉及里程碑付款和未来的销售分成。事后回想起来，不知道Redx公司有没有后悔的，把大腿拍断了。2018年Provention Bio公司从MacroGenics公司获得了核心资产抗CD3抗体teplizumab，这款药物在Provention Bio公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，5年后，诞生了29亿美元的收购案，让Provention Bio实现了5年增长20.4倍的神迹（粗略年化为85%）。2016年Biohaven公司从BMS公司获得了核心资产CGRP受体拮抗剂rimegepant，这款药物在Biohaven公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，6年后，诞生了2022年全球生物医药最大的小分子药收购案，116亿美元的公司价值让Biohaven实现了5年增长18.9倍的神迹（粗略年化为82%）。2012年TESARO公司从默沙东获得了核心资产PARP抑制剂niraparib，这款药物在TESARO公司的历史开发顺位里排名第2。从那以后，命运的齿轮开始转动，6年后，诞生了51亿美元的收购案，让TESARO实现了6年增长13.2倍的神迹（粗略年化为56%）。 2014年Zogenix公司从Brabant公司获得了核心资产低剂量芬氟拉明制剂，这款药物在Zogenix公司的历史开发顺位里排名第4。从那以后，命运的齿轮开始转动，8年后，诞生了19亿美元的收购案，让Zogenix实现了12年增长13.2倍的佳绩（粗略年化为25%）。2017年Endocyte公司从ABX公司获得了核心资产PSMA RDC 177Lu-PSMA-617，这款药物在Endocyte公司的历史开发顺位里排名20。从那以后，命运的齿轮开始转动，1年后，诞生了21亿美元的收购案，让曾经濒临溃败的Endocyte实现了7年增长11.6倍的佳绩（粗略年化为44%）。2017年ChemoCentryx公司自主研发的核心资产C5aR拮抗剂avacopan，这款药物在ChemoCentryx公司的历史开发顺位里排名第4。遭受前3款候选药物的开发挫折后，排名老4的avacopan终在2021年获批上市，并在1年后，诞生了37亿美元的收购案，让ChemoCentryx实现了10年增长9.5倍的佳绩（粗略年化为27%）。2013年Ignyta公司从Nerviano Medical Sciences公司获得了核心资产TRK/ROS1抑制剂entrectinib，这款药物在Ignyta公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，4年后，诞生了17亿美元的收购案，让Ignyta实现了3年增长8.7倍的神迹（粗略年化为113%）。2011年FIVE PRIME公司从Galaxy公司获得了核心资产抗FGFR2b抗体bemarituzumab，这款药物在Ignyta公司的历史开发顺位里排名第3。从那以后，命运的齿轮开始转动，10年后，诞生了19亿美元的收购案，让FIVE PRIME实现了8年增长8.1倍的佳绩（粗略年化为32%）。 2011年ZS Pharma公司从UOP公司获得了核心资产钾结合剂硅酸锆复合物ZS-9，这款药物在ZS Pharma公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，4年后，诞生了27亿美元的收购案，让ZS Pharma实现了1年增长6.6倍的神迹（粗略年化不适用）。2007年Hyperion公司从Ucyclyd公司获得了核心资产苯丁酸甘油酯口服液RAVICTI，这款药物在Hyperion公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，8年后，诞生了11亿美元的收购案，让ZS Hyperion实现了3年增长5.9倍的神迹（粗略年化为90%）。2005年CoLucid公司从礼来获得了核心资产5-HT(1F)受体激动剂lasmiditan，这款药物在CoLucid公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，12年后，诞生了9.6亿美元的收购案，让CoLucid实现了2年增长5.3倍的神迹（粗略年化为151%）。2007年Trius公司从Dong-A公司获得了核心资产恶唑烷酮类抗生素tedizolid，这款药物在Trius公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，6年后，诞生了7.07亿美元的收购案，让Trius实现了3年增长5倍的神迹（粗略年化为82%）。2007年Relypsa公司从Ilypsa/安进公司获得了核心资产钾结合剂patiromer，这款药物在Relypsa公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，9年后，诞生了15.3亿美元的收购案，让Relypsa实现了3年增长3.9倍的神迹（粗略年化为70%）。 2009年Durata公司从Vicuron/辉瑞公司获得了核心资产半合成糖肽抗生素dalbavancin，这款药物在Durata公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，5年后，诞生了6.75亿美元的收购案，让Durata实现了2年增长3.4倍的神迹（粗略年化为110%）。2018年Sierra公司从吉利德获得了核心资产JAK1/JAK2抑制剂momelotinib，这款药物在Sierra公司的历史开发顺位里排名第4。从那以后，命运的齿轮开始转动，4年后，诞生了19亿美元的收购案，让曾一度陷于困境的Sierra实现了7年增长2.9倍的佳绩（粗略年化为21%）。2005年Radius公司从Ipsen公司获得了核心资产甲状旁腺激素相关肽abaloparatide，这款药物在Radius公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，17年后，诞生了8.9亿美元的收购案，让Radius实现了8年增长2.8倍的成绩（粗略年化为18%）。2017年Dermira公司从罗氏获得了核心资产IL-13抑制剂lebrikizumab，这款药物在Dermira公司的历史开发顺位里排名第6。从那以后，命运的齿轮开始转动，3年后，诞生了11亿美元的收购案，让Dermira实现了6年增长2.6倍的佳绩（粗略年化为24%）。2011年Kadmon公司从NT Life Sciences公司获得了核心资产ROCK2抑制剂belumosudil，这款药物在Kadmon公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，10年后，诞生了19亿美元的收购案，让Kadmon实现了5年增长2.5倍的佳绩（粗略年化为28%）。 2018年Viela Bio公司从阿斯利康获得了核心资产抗CD19单抗inebilizumab，这款药物在Viela Bio公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，3年后，诞生了30.5亿美元的收购案，让Viela Bio实现了2年增长2.2倍的神迹（粗略年化为79%）。2012年ARMO公司从默沙东获得了核心资产PEG-IL-10 pegilodecakin，这款药物在ARMO公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，6年后，诞生了16亿美元的收购案，让ARMO实现了4个月增长2.2倍的神迹（粗略年化不适用）。2016年Myovant公司从武田获得了核心资产非肽GnRH受体拮抗剂relugolix，这款药物在Myovant公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，6年后，诞生了29亿美元的收购案，让Myovant实现了6年增长2.2倍的佳绩（粗略年化为21%）。2019年CinCor公司从罗氏获得了核心资产醛固酮合成酶抑制剂baxdrostat，这款药物在CinCor公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，4年后，诞生了18亿美元的收购案，让CinCor实现了1年增长2.1倍的佳绩（粗略年化不适用）。2013年Clementia公司从罗氏获得了核心资产RARγ激动剂palovarotene，这款药物在Clementia公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，6年后，诞生了13.1亿美元的收购案，让Clementia实现了5年增长1.9倍的佳绩（粗略年化为24%）。 2016年Dova公司从卫材获得了核心资产TPO受体激动剂avatrombopag，这款药物在Dova公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，3年后，诞生了9.15亿美元的收购案，让Dova实现了2年增长1.2倍的佳绩（粗略年化为48%）。2006年AVEO公司从Kyowa Hakko Kirin公司获得了核心资产泛靶点VEGFR抑制剂tivozanib，这款药物在AVEO公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，16年后，诞生了5.66亿美元的收购案，让AVEO实现了12年增长1.1倍的成绩（粗略年化为6%）。2011年Biotie公司从Synosia/罗氏获得了核心资产A2A受体拮抗剂tozadenant，这款药物在Biotie公司的历史开发顺位里排名第1。从那以后，命运的齿轮开始转动，5年后，诞生了3.63亿美元的收购案，让Biotie实现了7个月增长1倍的成绩（粗略年化不适用）。图1 发生重大M＆A的biotech（丰硕创投整理）结语医药熊市持续了近3年，悲观情绪渗透在每个角落里。有的企业招不到人，因为应聘者怕公司撑不了多久，倒闭了，附近的外卖还没尝尽就得卷铺盖走人了；有的企业裁好多人，因为公司实在没那么多资源去养这么多员工了；有的职员依旧打着这份工，但薪水福利明显不如以前了。医药投资圈也是如此，公募基金经理不得不辞职，才能平息基民的愤懑。私募基金掀起的注销潮，更像是无声的战争，发生的那么剧烈，但在公众里传播的影响力却又如此的低。似乎只有逃离整个医药圈，才有可能与这些悲观情绪绝缘。行业的周期更替、热胀冷缩是一种十分合乎常理的行为，奈何道理大家都懂，仍免不了随波逐流，很难去做些违背人体正常生理的事，比如正遭受着疼痛，却叫喊着舒服。但是往往在最悲观，最不受人关注的时候，蕴藏的机会才有可能是最大的。像本文提到的这批企业，资产出让方若知道自己的资产在多年后能值这么多钱，怎么会如此低价地转让出去。哪怕当时有人穿越回去，千叮咛，万嘱咐地劝诫资产出让方不要卖，或抬高价卖，但当时的环境已经决定了待交易资产就值这个价了，不可能啥亮眼数据都不多，就能卖出高价格的。同样的情况也适用于现在悲观的医药圈，当下的我们好比本文提到的这些在当年不怎么起眼的资产，每当我们多坚持一分，多努力一秒，多往前推进一下，说不定未来我们回忆过往的时候，可以掷地有声地说出，原来那时候命运的齿轮开始转动了。声明：以上内容仅供参考，不构成投资建议。

并购IPO临床3期

2023-05-10

·BioSpace

Pharma’s Biggest Losses: Once-Dropped Drugs Make Lucrative Comebacks

Pictured: Illustration of stacks of coins and a graph charting growth / iStock, Intpro In March 2023, Sanofi acquired the diabetes drug Tzield, along with its owner Provention Bio, for $2.9 billion. While not among the largest deals in the biopharma industry, it’s still a sizeable sum of money—especially for a treatment once discarded by another pharma giant. Initially developed by MacroGenics, the intravenous antibody treatment was scooped up by Eli Lilly in 2007, but Lilly dropped it three years later after a clinical trial in recently diagnosed type 1 diabetes patients failed to show a significant reduction in daily insulin dose or blood sugar levels in the treatment group compared to placebo. But it wasn’t the end of the road for Tzield. In 2018, MacroGenics licensed the drug to Provention for $6.1 million, according to SEC filings. In 2022, Sanofi took an interest, partnering with Provention to commercialize the drug. Late last year, the investment paid off—the FDA approved Tzield, and Sanofi moved forward with a complete acquisition of Provention and its drug. Such a convoluted history for therapeutics is not uncommon. Pharmaceutical companies may decide to sell the rights to their drug development programs when they have no intention of manufacturing the drug, or the research process doesn’t turn out as planned. However, sometimes the offloaded products perform better elsewhere, leading to big buyouts as companies try to expand their portfolios. The decision to license or sell a drug depends on profitability and a company’s strategic mission, said Andrew Lo, director of the Laboratory of Financial Engineering at MIT Sloan School of Management. “Even if a particular drug program is likely to be successful, and even if it generates a couple hundred million dollars a year in revenue, that might not be enough to justify putting the resources of the company to that drug because the same resources can be developing a billion dollar a year drug,” Lo told BioSpace. Similarly, if a company halves its work in a specific area, it may drop all products even if they’re successful. These decisions also depend on whether a company “believes” in the science of a drug, Lo said. Even if a drug doesn’t do well in clinical trials, another company may predict it will do well in the future and decide to buy it. In those cases, the original owner often retains an ownership stake in the product if it does well—to avoid the “egg on your face” of giving up a good product, as Lo described. As for acquisitions, those decisions depend on whether the acquiring company values all of the target company’s assets or just some. Lo said the trend of drugs changing hands would continue as some companies fall into financial need and sell their assets. “Stay tuned because the next six to 12 months are going to be periods of very active licensing deals and acquisitions." Here are a few more examples of drugs that ended up under the purview of large pharmaceutical companies after being dropped. 1. Momelotinib: Gilead’s Loss, GSK’s Gain First sale: YM Biosciences to Gilead Sciences for $510M (2012) Second sale: Gilead Sciences to Sierra Oncology for $3M (2018) Third sale: Sierra to GSK for $1.9B (2022) Momelotinib is a medication for patients with myelofibrosis, a form of bone marrow cancer. Gilead acquired the drug for $510 million in its 2012 acquisition of YM Biosciences, which had merged with Cytopia Limited in 2009. Gilead sold it to Sierra Oncology in 2018 for $3 million after clinical trials showed mixed results. Under the new ownership, the drug succeeded in clinical trials, and in June 2022, it was submitted to the FDA for approval. By then, GSK had already agreed to acquire the company for nearly $2 billion. Sierra awaits the agency’s decision, expected in June 2023. 2. Lebrikizumab: Roche’s Loss, Lilly’s Gain First sale: Roche to Dermira for $80M (2017) Second sale: Dermira to Eli Lilly for $1.1B (2020) Lebrikizumab is a monoclonal antibody used to treat atopic dermatitis. Dermira paid $80 million upfront to acquire the drug from Roche Group in 2017, and two years later, the FDA granted lebrikizumab fast-track designation. In 2020, Eli Lilly acquired Dermira for $1.1 billion. Lilly submitted the drug for approval in November 2022. 3. Zejula: Merck’s Loss, GSK’s Gain First sale: Merck to Tesaro for $7M (2012) Second sale: Tesaro to GSK for $5.1B (2019) Zejula (niraparib) is a poly ADP-ribose polymerase inhibitor that targets a family of proteins involved in cell response. Merck accepted $7 million upfront from Tesaro in 2012 to unload the drug from its portfolio. Five years later, the drug was approved for ovarian, fallopian tube or primary peritoneal cancer patients. Zejula’s success led GSK to acquire Tesaro for $5.1 billion in 2019. However, later concerns about PARP inhibitors led GSK to restrict the drug’s indications, even as the company explores Zejula’s utility for other cancer types. 4. Veltassa: Amgen’s Loss, Galenica’s Gain First sale: Amgen to Relypsa for $8.2M upfront (2009) Second sale: Relypsa to Galenica for $1.5B (2016) Veltassa (patiromer) is a drug developed for treating hyperkalemia, or high potassium in the blood. Relypsa, an offshoot of Amgen, received the rights to Veltassa from the parent company in 2009 for an upfront cost of $8.2 million. The drug was approved in 2015, and around the same time, Relypsa partnered with Vifor Fresenius Medical Care Renal Pharma—owned by Galenica—to commercialize the drug outside of the U.S. In 2016, Galenica fully acquired Relypsa for $1.5 billion and renamed the company CSL Vifor. 5. Cubicin: Lilly’s Loss, Merck’s Gain First sale: Eli Lilly to Cubist Pharmaceuticals for an undisclosed cost (1997) Second sale: Cubist to Merck for $8.4B (2014) In the late 1980s and early 1990s, Eli Lilly developed daptomycin, an intravenous antibiotic. However, the company suspended clinical trials after volunteers experienced adverse effects and discontinued its work on antibiotics altogether. Lilly then granted Cubist Pharmaceuticals the rights to develop and distribute daptomycin, which was approved in 2003 under the brand name Cubicin and 11 years later acquired by Merck for $8.4 billion. 6. Anidulafungin: Lilly’s Loss, Pfizer’s Gain First sale: Eli Lilly to Vicuron for $14M (1999) Second sale: Vicuron to Pfizer for $1.9B (2005) Vicuron obtained the antifungal drug anidulafungin from Eli Lilly in 1999 for $14 million. Initial Phase III clinical trials were successful, but in 2004 the FDA said a New Drug Application for the drug didn’t have enough data to support its efficacy, leading to lawsuits by shareholders per SEC filings. A year later, Pfizer acquired Vicuron for $1.9 billion. After submitting additional clinical data and adding another indication, the drug was approved in 2006 under the brand name Eraxis. Nadia Bey is a freelance reporter in North Carolina. She can be reached at beynadiaa@gmail.com.

上市批准并购临床3期

100 项与帕替罗姆相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
D11037	帕替罗姆	V03AE09	DB09263

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适应症	国家/地区	公司	日期
高钾血症	美国	Vifor Pharma AG	2015-10-21

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适应症	最高研发状态	国家/地区	公司	日期
心脏衰竭	临床3期	美国	Vifor Pharma AG	2019-04-24
心脏衰竭	临床3期	阿根廷	Vifor Pharma AG	2019-04-24
心脏衰竭	临床3期	比利时	Vifor Pharma AG	2019-04-24
心脏衰竭	临床3期	巴西	Vifor Pharma AG	2019-04-24
心脏衰竭	临床3期	保加利亚	Vifor Pharma AG	2019-04-24
心脏衰竭	临床3期	加拿大	Vifor Pharma AG	2019-04-24
心脏衰竭	临床3期	捷克	Vifor Pharma AG	2019-04-24
心脏衰竭	临床3期	法国	Vifor Pharma AG	2019-04-24
心脏衰竭	临床3期	格鲁吉亚	Vifor Pharma AG	2019-04-24
心脏衰竭	临床3期	德国	Vifor Pharma AG	2019-04-24

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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


NCT03888066 (DIAMOND, Pubmed \| JACC Heart Fail)	临床3期	高钾血症 serum potassium	-	Patiromer	鏇襯構鏇齋願簾簾顧築(淵壓齋網艱鏇窪窪衊觸) = 製淵鹽築窪鬱齋觸糧壓壓鹹網齋餘壓顧膚遞夢 (醖壓繭構網壓顧獵積顧 ) 更多	积极	2024-09-01
				Placebo	鏇襯構鏇齋願簾簾顧築(淵壓齋網艱鏇窪窪衊觸) = 憲淵簾範築築願壓餘鬱壓鹹網齋餘壓顧膚遞夢 (醖壓繭構網壓顧獵積顧 ) 更多
ESC2024	N/A	心脏衰竭	-	Patiromer	願艱膚顧齋鑰網夢淵齋(鹽窪獵餘襯鑰艱積築鑰) = mild or moderate gastrointestinal events 網齋糧範構齋膚淵遞廠 (窪衊觸衊構淵糧壓蓋膚 ) 更多	-	2024-05-13
ESC2024	N/A	高钾血症	-	Patiromer therapy	膚鹹網積製淵糧襯淵構(選襯鹹範網艱鏇衊鹽廠) = 齋範襯艱製膚願淵鬱獵鹽憲鑰願繭齋淵獵淵鹽 (鬱憲餘鑰簾繭觸簾壓顧, 49 ~ 86.5) 更多	-	2024-05-12
NCT03740048 (TWOPLUS-HD, CTgov)	临床3期	终末期肾脏病	51	Hemodialysis+Patiromer Oral Product (Hemodialysis and Pharmacologic Therapy)	築廠鏇齋構範獵淵窪憲(鹹顧選壓蓋餘遞範齋構) = 醖選廠壓夢淵繭積製醖鏇構廠醖構鬱獵壓鏇鏇 (範積網網鏇壓蓋壓願範, 鏇構壓窪憲夢鏇憲衊遞 ~ 鑰積齋製積淵簾顧糧齋) 更多	-	2023-06-05
				Hemodialysis (Conventional Hemodialysis Regimen)	築廠鏇齋構範獵淵窪憲(鹹顧選壓蓋餘遞範齋構) = 蓋鬱夢襯鏇獵糧憲觸築鏇構廠醖構鬱獵壓鏇鏇 (範積網網鏇壓蓋壓願範, 餘夢積範糧積衊蓋艱淵 ~ 願齋範願膚鏇構繭艱鹹) 更多
DIAMOND (ESC2023)	N/A	射血分数降低的心力衰竭 \| 高钾血症 N-terminal pro-B-type natriuretic peptide (NT-proBNP)	-	Patiromer	簾艱憲齋鹹夢製廠選鑰(艱鑰繭夢夢鹽憲鬱鹹築) = 製糧鏇餘廠網鑰蓋簾壓選衊憲獵襯壓遞窪鹹範 (膚鹹廠襯鬱鏇鏇襯鹹齋 )	-	2023-05-20
				Placebo	簾艱憲齋鹹夢製廠選鑰(艱鑰繭夢夢鹽憲鬱鹹築) = 願夢窪鏇醖構膚鏇衊鏇選衊憲獵襯壓遞窪鹹範 (膚鹹廠襯鬱鏇鏇襯鹹齋 )
NCT03888066 (DIAMOND, CTgov)	临床3期	心脏衰竭 \| 高钾血症	1,195	patiromer (Patiromer)	窪鏇糧鑰範遞獵簾構鏇(壓鏇製壓網壓製廠網願) = 構壓鹽鏇壓廠獵蓋襯鏇夢獵淵選網構鑰壓壓願 (蓋鏇積襯齋積積齋淵壓, 構艱網艱衊範齋網夢廠 ~ 獵衊遞憲廠鬱範製顧構) 更多	-	2023-02-24
				placebo+potassium (Placebo)	窪鏇糧鑰範遞獵簾構鏇(壓鏇製壓網壓製廠網願) = 獵積鑰願糧鬱繭憲糧繭夢獵淵選網構鑰壓壓願 (蓋鏇積襯齋積積齋淵壓, 夢鏇繭積鹹顧網糧構簾 ~ 觸蓋淵製鑰選膚膚鑰觸) 更多
ASN2022	N/A	终末期肾脏病 \| 高钾血症	458	Patiromer	網鬱膚簾繭簾鬱醖構顧(壓窪憲繭醖觸膚壓窪夢) = 顧襯鏇壓壓醖鑰繭鏇壓齋顧鹽糧艱餘膚遞鏇顧 (壓蓋構醖糧糧鬱壓餘繭 ) 更多	积极	2022-11-05
ASN2022	N/A	肾功能不全 \| 高钾血症	2,048	Patiromer	選鹹醖窪窪簾願蓋夢艱(淵選製選窪積淵遞積製) = 襯鏇鏇觸夢鬱鑰製憲獵構築壓構憲艱齋壓膚積 (齋遞鑰糧鏇鑰範餘淵範 ) 更多	积极	2022-11-04
NCT03087058 (EMERALD, CTgov)	临床2期	慢性肾病 \| 高钾血症	23	Patiromer (Cohort 1)	廠糧顧觸壓齋鹽淵簾蓋(製獵衊餘壓廠膚鏇鹹膚) = 願鹹鹹築鑰構壓簾積簾醖夢齋繭範膚選製鹽餘 (築製遞廠製積衊製築窪, 網壓窪遞繭鹽餘糧蓋遞 ~ 鏇壓鑰襯繭鹽製醖壓蓋) 更多	-	2022-09-26
				Patiromer (Cohort 2)	廠糧顧觸壓齋鹽淵簾蓋(製獵衊餘壓廠膚鏇鹹膚) = 衊顧艱範顧繭夢鹽醖鹹醖夢齋繭範膚選製鹽餘 (築製遞廠製積衊製築窪, 繭鬱窪繭網鹽選選鏇窪 ~ 構築淵製壓簾構憲顧鹽) 更多
NCT03888066 (DIAMOND, Pubmed)	临床3期	射血分数降低的心力衰竭	1,642	Patiromer	衊網製廠廠餘蓋夢糧襯(構鬱糧餘餘艱簾淵齋觸): HR = 0.62 (95% CI, 0.45 ~ 0.87), P-Value = 0.006 更多	积极	2022-07-28
				Placebo