Observational data have suggested no increased risk of adverse pregnancy outcomes associated with exposure to interferon-beta (IFNB) before or during pregnancy. After the emergence of these data, the European Medicines Agency approved a label change for IFNB in September 2019, stating that use of IFNB during pregnancy may be considered, if clinically needed. However, limited data on pregnancies exposed in the 2nd and 3rd trimesters were observed.
INFORM is a secondary use of data drug utilisation study (DUS) to determine late pregnancy exposure (i.e. during the 2nd and 3rd trimester) to IFNB in Finland and Sweden, which will inform whether the number of exposed pregnancies is adequate to conduct a cohort study on adverse pregnancy outcomes, with a focus on late pregnancy exposure.
The number of pregnancies will be initially reported three years after the revised label implementation (September 2019) and will include data on pregnancies from 1996 in Finland and from 2005 in Sweden up through 31 December 2022. If the number of pregnancies is deemed adequate for conducting the cohort study on adverse pregnancy outcomes, this DUS will be finalised with the drug utilisation data accrued up through 31 December 2022. If the number of pregnancies until 31 December 2022 is deemed inadequate, this study may be continued and the primary and secondary objectives may be examined five years after the revised label implementation, including pregnancies until 31 December 2024.

开始日期2024-05-01

申办/合作机构

Bayer AG

[+3]

NCT05688436 / RecruitingN/A

Pregnancy Outcomes in Women Exposed to Diroximel Fumarate

The primary objective of the study is to estimate the prevalence of major congenital malformations (MCMs) and compare the prevalence between the diroximel fumarate (DRF) and comparator groups. The secondary objectives of the study are to estimate the incidence of spontaneous abortion (SA) and compare the incidence between the DRF and comparator groups; to estimate the incidence of preterm birth and compare the incidence between the DRF and comparator groups; to estimate the incidence of stillbirth and compare the incidence between the DRF and comparator groups and to estimate the prevalence of small for gestational age (SGA) and compare the prevalence between the DRF and comparator groups.

开始日期2021-09-24

申办/合作机构

Biogen, Inc.

NCT04079088 / Withdrawn临床2期

A Multicenter, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Ranging Phase 2 Study to Evaluate the Efficacy and Safety of Oral BIIB061 as Add-On Therapy to Interferon-Beta 1 or Glatiramer Acetate Therapies in Relapsing Multiple Sclerosis

The primary objectives of the study are to evaluate the safety of BIIB061 versus placebo in participants with Relapsing Multiple Sclerosis (RMS), and to evaluate the efficacy of BIIB061 to improve disability outcome versus placebo in participants with RMS.
The secondary objectives of the study are to evaluate the effects of BIIB061 versus placebo on brain magnetic resonance imaging (MRI) markers of remyelination and axon preservation in chronic Multiple Sclerosis lesions and to evaluate the effects of BIIB061 versus placebo on additional measures of improved disability outcome.

开始日期2021-06-30

申办/合作机构

Biogen, Inc.

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项与聚乙二醇干扰素β-1a 相关的文献（医药）

2024-01-01·European review for medical and pharmacological sciences

Utilization of peginterferon-β-1a in the real-world practice for relapsing-remitting multiple sclerosis.

Article

作者: M Moccia ; L Santoni ; I Vaccari ; G Affinito ; D Caliendo ; F Rubba ; R Lanzillo ; M Triassi ; V Brescia Morra ; R Palladino

OBJECTIVE:

Peginterferon β-1a (PEG-IFN-β-1a) is the most recent interferon beta formulation approved for treating relapsing-remitting multiple sclerosis (RRMS). We aim to describe the real-world utilization of PEG-IFN-β-1a in RRMS and compare it with other injectable disease-modifying therapies (DMTs).

PATIENTS AND METHODS:

In this population-based study, we used 2015-2019 routinely collected healthcare data of the Campania region of Italy from National Healthcare System DMT prescriptions, inpatient and outpatient clinical records of hospitals in Campania, and the Federico II University MS clinical registry for a subset of patients. We included individuals with RRMS receiving new prescriptions of PEG-IFN-β-1a [n=281; age = 38.8±12.3 years; females=70.5%; disease duration = 8.4±8.3 years; Expanded Disability Status Scale (EDSS) at baseline=2.0 (1.0-6.5)], glatiramer acetate [n=751; age = 46.0±11.4 years; females=67.1%; disease duration = 9.8±8.2 years; EDSS=4.0 (1.5-8.5)], and subcutaneous (SC) IFN-β-1a [n=1,226; age = 39.7±11.7 years; females=66.5%; disease duration = 8.2±6.5 years; EDSS 2.5 (1.5-6.5)]. Adherence [medication possession ratio (MPR)], escalation to more effective DMTs, hospitalization rates and costs were measured. We used mixed-effect linear regression models (for adherence, hospitalization rates and costs) and Cox regression models (for escalation) to assess differences between PEG-IFN-β-1a (statistical reference), glatiramer acetate, and SC IFN-β-1a. All models included age, sex, previous treatment/untreated, year of treatment initiation, treatment duration, and adherence as covariates.

RESULTS:

Adherence was lower in glatiramer acetate (MPR = 0.91±0.1; Coeff=-0.11; p<0.01), and IFN-β-1a (MPR = 0.92±0.1; Coeff=-0.08; p<0.01), compared with PEG-IFN-β-1a (MPR = 1.01±0.1). The probability of escalating to more effective DMTs was higher for glatiramer acetate (14.9%; HR=4.09; p<0.01) and IFN-β-1a (9.1%; HR=3.35; p=0.01), compared with PEG-IFN-β-1a (4.9%). No differences in annualized hospitalization rates were identified between glatiramer acetate [annualized hospitalization rates (AHR) = 0.05±0.30; Coeff=0.02; p=0.31), IFN-β-1a (AHR = 0.02±0.21; Coeff=0.01; p=0.97], and PEG-IFN-β-1a (AHR = 0.02±0.24); however, monthly costs for MS admissions were higher for glatiramer acetate (€49.45±€195.27; Coeff=-29.89; p=0.03), compared with IFN-β-1a (€29.42±€47.83; Coeff=6.79; p=0.61), and PEG-IFN-β-1a (€23.91±€43.90).

CONCLUSIONS:

SC PEG-IFN-β-1a and IFN-β-1a were used in relatively similar populations, while glatiramer acetate was preferred in older and more disabled patients. PEG-IFN-β-1a was associated with higher adherence and lower escalation rates toward more effective (and costly) DMTs.

2023-11-30·The Cochrane database of systematic reviews

Adverse effects of immunotherapies for multiple sclerosis: a network meta-analysis.

Review

作者: Irene Tramacere ; Gianni Virgili ; Vittorio Perduca ; Ersilia Lucenteforte ; Maria Donata Benedetti ; Matteo Capobussi ; Greta Castellini ; Serena Frau ; Marien Gonzalez-Lorenzo ; Robin Featherstone ; Graziella Filippini

BACKGROUND:

Multiple sclerosis (MS) is a chronic disease of the central nervous system that affects mainly young adults (two to three times more frequently in women than in men) and causes significant disability after onset. Although it is accepted that immunotherapies for people with MS decrease disease activity, uncertainty regarding their relative safety remains.

OBJECTIVES:

To compare adverse effects of immunotherapies for people with MS or clinically isolated syndrome (CIS), and to rank these treatments according to their relative risks of adverse effects through network meta-analyses (NMAs).

SEARCH METHODS:

We searched CENTRAL, PubMed, Embase, two other databases and trials registers up to March 2022, together with reference checking and citation searching to identify additional studies.

SELECTION CRITERIA:

We included participants 18 years of age or older with a diagnosis of MS or CIS, according to any accepted diagnostic criteria, who were included in randomized controlled trials (RCTs) that examined one or more of the agents used in MS or CIS, and compared them versus placebo or another active agent. We excluded RCTs in which a drug regimen was compared with a different regimen of the same drug without another active agent or placebo as a control arm.

DATA COLLECTION AND ANALYSIS:

We used standard Cochrane methods for data extraction and pairwise meta-analyses. For NMAs, we used the netmeta suite of commands in R to fit random-effects NMAs assuming a common between-study variance. We used the CINeMA platform to GRADE the certainty of the body of evidence in NMAs. We considered a relative risk (RR) of 1.5 as a non-inferiority safety threshold compared to placebo. We assessed the certainty of evidence for primary outcomes within the NMA according to GRADE, as very low, low, moderate or high.

MAIN RESULTS:

This NMA included 123 trials with 57,682 participants_. Serious adverse events (SAEs) Reporting of SAEs was available from 84 studies including 5696 (11%) events in 51,833 (89.9%) participants out of 57,682 participants in all studies. Based on the absolute frequency of SAEs, our non-inferiority threshold (up to a 50% increased risk) meant that no more than 1 in 18 additional people would have a SAE compared to placebo. Low-certainty evidence suggested that three drugs may decrease SAEs compared to placebo (relative risk [RR], 95% confidence interval [CI]): interferon beta-1a (Avonex) (0.78, 0.66 to 0.94); dimethyl fumarate (0.79, 0.67 to 0.93), and glatiramer acetate (0.84, 0.72 to 0.98). Several drugs met our non-inferiority criterion versus placebo: moderate-certainty evidence for teriflunomide (1.08, 0.88 to 1.31); low-certainty evidence for ocrelizumab (0.85, 0.67 to 1.07), ozanimod (0.88, 0.59 to 1.33), interferon beta-1b (0.94, 0.78 to 1.12), interferon beta-1a (Rebif) (0.96, 0.80 to 1.15), natalizumab (0.97, 0.79 to 1.19), fingolimod (1.05, 0.92 to 1.20) and laquinimod (1.06, 0.83 to 1.34); very low-certainty evidence for daclizumab (0.83, 0.68 to 1.02). Non-inferiority with placebo was not met due to imprecision for the other drugs: low-certainty evidence for cladribine (1.10, 0.79 to 1.52), siponimod (1.20, 0.95 to 1.51), ofatumumab (1.26, 0.88 to 1.79) and rituximab (1.01, 0.67 to 1.52); very low-certainty evidence for immunoglobulins (1.05, 0.33 to 3.32), diroximel fumarate (1.05, 0.23 to 4.69), peg-interferon beta-1a (1.07, 0.66 to 1.74), alemtuzumab (1.16, 0.85 to 1.60), interferons (1.62, 0.21 to 12.72) and azathioprine (3.62, 0.76 to 17.19). Withdrawals due to adverse events Reporting of withdrawals due to AEs was available from 105 studies (85.4%) including 3537 (6.39%) events in 55,320 (95.9%) patients out of 57,682 patients in all studies. Based on the absolute frequency of withdrawals, our non-inferiority threshold (up to a 50% increased risk) meant that no more than 1 in 31 additional people would withdraw compared to placebo. No drug reduced withdrawals due to adverse events when compared with placebo. There was very low-certainty evidence (meaning that estimates are not reliable) that two drugs met our non-inferiority criterion versus placebo, assuming an upper 95% CI RR limit of 1.5: diroximel fumarate (0.38, 0.11 to 1.27) and alemtuzumab (0.63, 0.33 to 1.19). Non-inferiority with placebo was not met due to imprecision for the following drugs: low-certainty evidence for ofatumumab (1.50, 0.87 to 2.59); very low-certainty evidence for methotrexate (0.94, 0.02 to 46.70), corticosteroids (1.05, 0.16 to 7.14), ozanimod (1.06, 0.58 to 1.93), natalizumab (1.20, 0.77 to 1.85), ocrelizumab (1.32, 0.81 to 2.14), dimethyl fumarate (1.34, 0.96 to 1.86), siponimod (1.63, 0.96 to 2.79), rituximab (1.63, 0.53 to 5.00), cladribine (1.80, 0.89 to 3.62), mitoxantrone (2.11, 0.50 to 8.87), interferons (3.47, 0.95 to 12.72), and cyclophosphamide (3.86, 0.45 to 33.50). Eleven drugs may have increased withdrawals due to adverse events compared with placebo: low-certainty evidence for teriflunomide (1.37, 1.01 to 1.85), glatiramer acetate (1.76, 1.36 to 2.26), fingolimod (1.79, 1.40 to 2.28), interferon beta-1a (Rebif) (2.15, 1.58 to 2.93), daclizumab (2.19, 1.31 to 3.65) and interferon beta-1b (2.59, 1.87 to 3.77); very low-certainty evidence for laquinimod (1.42, 1.01 to 2.00), interferon beta-1a (Avonex) (1.54, 1.13 to 2.10), immunoglobulins (1.87, 1.01 to 3.45), peg-interferon beta-1a (3.46, 1.44 to 8.33) and azathioprine (6.95, 2.57 to 18.78); however, very low-certainty evidence is unreliable. Sensitivity analyses including only studies with low attrition bias, drug dose above the group median, or only patients with relapsing remitting MS or CIS, and subgroup analyses by prior disease-modifying treatments did not change these figures. Rankings No drug yielded consistent P scores in the upper quartile of the probability of being better than others for primary and secondary outcomes.

AUTHORS' CONCLUSIONS:

We found mostly low and very low-certainty evidence that drugs used to treat MS may not increase SAEs, but may increase withdrawals compared with placebo. The results suggest that there is no important difference in the occurrence of SAEs between first- and second-line drugs and between oral, injectable, or infused drugs, compared with placebo. Our review, along with other work in the literature, confirms poor-quality reporting of adverse events from RCTs of interventions. At the least, future studies should follow the CONSORT recommendations about reporting harm-related issues. To address adverse effects, future systematic reviews should also include non-randomized studies.

2023-08-01·EClinicalMedicine

Interferon β-1a ring prophylaxis to reduce household transmission of SARS-CoV-2: a cluster randomised clinical trial.

Article

作者: José A Castro-Rodriguez ; Eleanor N Fish ; Samuel T Montgomery ; Tobias R Kollmann ; Carolina Iturriaga ; Casey Shannon ; Yuliya Karpievitch ; Joseph Ho ; Virginia Chen ; Robert Balshaw ; Rym Ben-Othman ; Radhouane Aniba ; Francisca Gidi-Yunge ; Lucy Hartnell ; David G Hancock ; Guillermo Pérez-Mateluna ; Marcela Urzúa ; Scott J Tebbutt ; Diego García-Huidobro ; Cecilia Perret ; Arturo Borzutzky ; Stephen M Stick

Background:

Accumulating evidence indicates that an early, robust type 1 interferon (IFN) response to SARS-CoV-2 is important in determining COVID-19 outcomes, with an inadequate IFN response associated with disease severity. Our objective was to examine the prophylactic potential of IFN administration to limit viral transmission.

Methods:

A cluster randomised open label clinical trial was undertaken to determine the effects of pegylated IFNβ-1a administration on SARS-CoV-2 household transmission between December 3rd, 2020 and June 29th, 2021. Index cases were identified from databases of confirmed SARS-CoV-2 individuals in Santiago, Chile. Households were cluster randomised (stratified by household size and age of index cases) to receive 3 doses of 125 μg subcutaneous pegylated IFNβ-1a (172 households, 607 participants), or standard care (169 households, 565 participants). The statistical team was blinded to treatment assignment until the analysis plan was finalised. Analyses were undertaken to determine effects of treatment on viral shedding and viral transmission. Safety analyses included incidence and severity of adverse events in all treatment eligible participants in the standard care arm, or in the treatment arm with at least one dose administered. Clinicaltrials.gov identifier: NCT04552379.

Findings:

5154 index cases were assessed for eligibility, 1372 index cases invited to participate, and 341 index cases and their household contacts (n = 831) enrolled. 1172 participants in 341 households underwent randomisation, with 607 assigned to receive IFNβ-1a and 565 to standard care. Based on intention to treat (ITT) and per protocol (PP) analyses for the primary endpoints, IFNβ-1a treatment did not affect duration of viral shedding in index cases (absolute risk reduction = -0.2%, 95% CI = -8.46% to 8.06%) and transmission of SARS-CoV-2 to household contacts (absolute risk reduction = 3.87%, 95% CI = -3.6% to 11.3%). Treatment with IFNβ-1a resulted in significantly more treatment-related adverse events, but no increase in overall adverse events or serious adverse events.

Interpretation:

Based upon the primary analyses, IFNβ-1a treatment did not affect duration of viral shedding or the probability of SARS-CoV-2 transmission to uninfected contacts within a household.

Funding:

Biogen PTY Ltd. Supply of interferon as 'Plegridy (peginterferon beta-1a).' The study was substantially funded by BHP Holdings Pty Ltd.

项与聚乙二醇干扰素β-1a 相关的新闻（医药）

2024-04-04

·PRNewswire

Cadenza Bio, Inc. Appoints Michael A. Panzara, MD, MPH, to its Board of Directors

OKLAHOMA CITY, April 4, 2024 /PRNewswire/ -- Cadenza Bio, Inc., a pioneering preclinical biotechnology company dedicated to developing innovative small-molecule drugs for the treatment of multiple sclerosis (MS) and other demyelinating and inflammatory diseases, proudly announces the appointment of Michael A. Panzara, MD, MPH, to its esteemed board of directors. Dr. Panzara brings over 22 years of invaluable biopharmaceutical experience to Cadenza Bio. He currently serves as chief medical officer at Neurvati Neurosciences, a neuroscience-focused biotechnology company working to advance the development of clinical-stage product candidates for neurological and neuropsychiatric disorders. Prior to his role at Neurvati, Dr. Panzara held key leadership positions at Wave Life Sciences, Sanofi, Genzyme, and Biogen, showcasing his deep understanding of neurological disorders and drug development. He also serves on the board of directors of Athira Pharma, Inc., a neurology-focused clinical-stage biopharmaceutical company. Over his career, Dr. Panzara oversaw development activities and global regulatory approvals for several MS drugs. He served as the head of the Multiple Sclerosis, Neurology, and Ophthalmology Therapeutic Area for Global Development at Sanofi Genzyme, where he oversaw global regulatory approvals of LEMTRADA® (alemtuzumab) and AUBAGIO® (teriflunomide) and was responsible for development strategy and oversight within these therapeutic areas. During his time at Biogen, he served as global clinical lead for the development of TYSABRI® (natalizumab) for MS and managed clinical development activities for all late-stage MS products including AVONEX® (interferon beta-1a), PLEGRIDY® (PEG-interferon beta-1a), and TECFIDERA® (dimethylfumarate). "We are delighted to welcome Dr. Panzara to our board of directors," said Dr. Carol Curtis, chief executive officer of Cadenza Bio. "His extensive experience in developing therapies for MS and other neurological disorders aligns perfectly with our mission at Cadenza Bio. Dr. Panzara's strategic insights and deep understanding of the industry will be invaluable as we continue to advance our innovative drug pipeline and work towards bringing transformative treatments to patients in need." Dr. Panzara received his undergraduate degree in biology from the University of Pennsylvania and his medical degree from Stanford University School of Medicine. He completed his neurology training at Massachusetts General Hospital and received postdoctoral training in immunology and rheumatology at Brigham and Women's Hospital. Additionally, Dr. Panzara earned his master of public health degree from the Harvard School of Public Health. With Dr. Panzara joining the board of directors, Cadenza Bio is in a stronger position to achieve its goal of developing groundbreaking therapies for MS and other demyelinating and inflammatory diseases. "I look forward to joining the board of directors at Cadenza Bio at such an exciting time in the growth of the company," said Dr. Panzara. "Their approach in developing innovative dual-action therapeutic agents with neuroprotective and immunomodulatory effects has great potential to significantly expand our ability to address the unmet needs that remain for many MS patients." For more information about Cadenza Bio and its groundbreaking research, please visit . About Cadenza Bio, Inc. Cadenza Bio, Inc. is a preclinical biotechnology company focused on developing innovative small-molecule drugs for the treatment of demyelinating and inflammatory diseases. The company's mission is to break the cycle of disease and transform lives by developing therapies that not only alleviate symptoms but also halt progression, promote repair, and restore quality of life for patients. Cadenza Bio's MS drug pipeline, emerging from a collaboration between leading researchers, demonstrates promising preclinical results and offers hope for patients suffering from these debilitating conditions. SOURCE Cadenza Bio, Inc.

高管变更

2024-02-20

·药融圈

渤健财报解读

▲3月7-8日成都生物医药创新者峰会 · 点击立即报名注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。2月13日，美国biotech渤健公司发布了2023年财报，该公司全年营收98.36亿美元，同比下降2%，其中四季度营收23.86亿美元，同比下降6%。其业绩下滑的最主要原因是多发性硬化管线销售额下降，多发性药物共卖出54.30亿美元，同比下降14%。该公司在2023年的销售成本为25.33亿美元，占销售额的26%，毛利水平为74%，相比去年同期下降4%。研发投入24.62亿美元，约占销售额的25%，市场和一般管理费22.77亿美元，约占销售额的23%。因为毛利下降和研发投入增加，该公司的利润水平出现了明显下降，2023年净利润为11.62亿美元，仅为销售额的11.80%。渤健的业务营收分为三个部分，包括产品销售收入、产品授权费、CMO费，分别收入72.47亿美元、16.90亿美元和8.99亿美元。而产品销售收入又主要来自多发性硬化管线、罕见病管线和生物类似药管线，2023年净销售额分别为46.62亿美元、18.03亿美元和7.70亿美元。多发性硬化方面，富马酸二甲酯（TECFIDERA）因为专利已经失效，仅卖出10.13亿美元，相比去年同期销售额下降4亿多美元；VUMERITY是为了应对TECFIDERA专利悬崖而开发的505b2，全年卖出5.76亿美元，几乎相比去年同期持平。β干扰素（AVONEX）是渤健卖了20年的老产品，2023年营收8.11亿美元，相比去年同期也出现了下降。PLEGRIDY是AVONEX的升级品，2023年销售额为2.94亿美元，相比去年同期也存在下降。那他珠单抗(TYSABRI) 营收18.77亿美元，相比去年同期下降约2亿美元，氨吡啶缓释片(Fampridine）虽未出现销售额下滑，但营收不足1亿美元。罕见病管线方面，渤健拥有诺西那生钠(Spinraza）、Qalsody(tofersen)和Skyclarys(omaveloxolone)三款药物，全年营收分别为17.41亿美元、0.06亿美元和0.6亿美元，整个管线销售额几乎与去年持平。生物类似物方面，渤健主要有BENEPALI（依那西普）、IMRALDI（阿达木单抗）、英夫利昔单抗（FLIXABI）和BYOOVIZ（雷珠单抗）四款产品，2023年销售额分别为4.39亿美元、2.22亿美元、0.77亿美元和0.32亿美元，biosimilar总销售额几乎与去年同期持平。相关阅读诺和诺德2023年财报：司美格鲁肽大卖212亿美元诺华2023年业绩：营收454亿美元，同比增长10%GSK 2023年业绩：营收303亿英镑，盈利能力大幅提升辉瑞公布2023年业绩：营收同比下降42%，净利润同比下降93%强生2023年业绩公开，营收852亿美元，同比增长6.5%（附产品销售额）默沙东营收601亿美元，其中药王K药大卖250亿美元赛诺菲2023年营收431亿欧元，Dupixent突破110亿美元，氯吡格雷和甘精胰岛素在中国逆势增长再生元财报：度普利尤单抗卖出116亿美元，阿柏西普94亿美元艾伯维财报：修美乐销售额大幅下跌，正式让出药王宝座罗氏年报：营收下降7%，净利润下降9%礼来财报：GLP-1大卖125亿美元Vertex：囊性纤维化药物比增长11%，破百亿在即吉利德公开业绩：营收271亿美元，其中CAR-T 19亿美元安进2023年业绩：营收282亿美元，同比增长11%阿斯利康2023：中国区收入423亿元，增长7%BMS业绩：总营收450亿美元，同比下降2%版权声明：本文转自药事纵横，如不希望被转载的媒体或个人可与我们联系，我们将立即删除活动推荐 3月 • NDC x 2024新药创新者峰会关键词： ADC，改良型新药，小分子新药，GLP-1药物（点击下方图片查看详情）▼【关于药融圈】药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。

财报生物类似药

2023-09-12

·药渡Daily

渤健如何度过“中年危机”？

渤健（Biogen），成立于1978年，是一家以科学驱动研发的生物技术公司。1991年，渤健在纳斯达克上市。1996年，治疗复发性多发性硬化症（MS）的干扰素β-1a（商品名：Avonex）获FDA批准上市，这是首款被证明能够减缓残疾进展、降低病情恶化频率的药物，日后为渤健赚取丰厚利润的同时也奠定了其在神经科学领域领军地位的关键一步。然而今时不同往日，如今的渤健压力山大。2022年财报显示，渤健全年总营收101.73亿美元，同比下降7.36%，其中，产品净销售额79.88亿美元，同比下降9.7%。今年7月25日，渤健发布的2023H1财报显示：上半年营收49.19亿美元，同比下滑4%，净利润为9.81亿美元，同比下滑23.2%。不仅如此，近4年来，渤健的营收都在持续下降。在其优势领域，多发性硬化症和髓性肌萎缩症产品的增长也不如人意。由于重磅产品遭遇专利悬崖，以及新药不断涌现等因素影响，2022年渤健多发性硬化产品总营收54.30亿美元，同比下降11%；2023H1，该领域产品销售额更是呈现持续下滑态势，上半年总收入仅为23.35亿美元。成立45年的渤健正在遭遇“中年危机”。那么，渤健会如何突围呢？成也MS，“败”也MS多发性硬化症（multiple sclerosis，MS）药物一直是渤健的主要收入来源。MS是一种发生在中枢神经系统的自身免疫性脱髓鞘和神经退行性疾病。在发病初期，MS的症状类似于普通感冒，通常是头痛、四肢无力，渐渐地，身体不受控制，行走困难，甚至大小便失禁。反复发作后，患者可能会致残甚至死亡。全球约有300万人受MS的折磨。由于其发病机制尚不清晰，因此目前尚无一款药物能治愈MS。大多数药物只能延缓发作时间，患者需要终身服药。作为最早探索单抗治疗MS可行性的企业之一，渤健在20世纪初期，推出了治疗复发缓解型MS（RRMS）的单抗--Tysabri（Natalizumab，那他珠单抗），这是一款重组人源化IgG4κ单克隆抗体，能够结合除中性粒细胞以外的所有白细胞表面上表达的α4β1和α4β7整联蛋白的α4亚基，白细胞依靠α4-整联蛋白与内皮细胞上的受体粘附从而进入组织，因此Tysabri可以防止白细胞穿过内皮迁移到发炎的组织中，从而用于治疗炎性疾病。2004年，FDA通过加速审批途径批准Tysabri上市，用于治疗RRMS。不过在获批上市后仅4个月，有两例接受Tysabri的患者发生了进行性多灶性白质脑病（PML），渤健主动将Tysabri从市场上撤回。两年后，通过对临床试验期间的患者重新进行数据收集，证实该副反应在可接受的范围内，FDA再次批准了Tysabri的上市销售。2008年，FDA又批准Tysabri用于治疗克罗恩病。除了Tysabri之外，渤健在MS治疗领域还有几款明星产品，如Avonex（Interferon beta-1a，销售峰值一度超过30亿美金）、Tecfidera（富马酸二甲酯）、Plegridy（干扰素）、Fampyra（4-氨毗R定）、Vumerity（diroximel fumarate）。其中Tecfidera是继芬戈莫德（S1P调节剂）、特立氟胺（DHODH酶抑制剂）之后全球上市的第4款MS口服药物。Tecfidera是一种烟酸受体，通过激活Nrf2相关的抗氧化信号通路，启动中枢神经系统自我保护系统，以减少神经髓鞘质受损。临床研究显示，Tecfidera可将MS复发频率降低53%，残疾进程缩短38%，且副作用小。Tecfidera于2013年获FDA批准上市，当年销售额便达到8.76亿美金，2019年达到销售峰值44.33亿美元，迅速在MS治疗市场站稳了阵脚。长久以来，MS治疗产品都是渤健的中流砥柱。直到2017年，罗氏（基因泰克）的Ocrevus（Ocrelizumab，奥瑞珠单抗）横空出世，力压市面上所有MS治疗药物，迅速卷走MS市场份额。到了2022年，Ocrevus销售额高达65.39亿美元，比渤健所有MS药物销售额加起来都多。同样是在2017年，渤健的核心产品Tecfidera遭遇了仿制药巨头迈兰（Mylan，现属于Viatris）的专利挑战，原本Tecfidera核心专利2028年才会到期。但是在经过了4年官司拉锯战之后，渤健败诉，迈兰的Tecfidera仿制药在2021年8月成功上市销售，随后其它仿制药企业也蜂拥而上。自此，渤健的Tecfidera销售就面临巨大危机，其他多发性硬化症药物的销售情况也不甚乐观。如今，MS治疗药物在渤健总营收中的占比已由最高峰时的82.51%降至50%以下，渤健总营收也在急剧萎缩。根据渤健2023H1财报，上半年MS产品营收23.35亿美元，而2022年同期卖了28.21亿美元。渤健的“中年危机”已上市药物后续乏力，替补产品缺位由于仿制药和生物类似药的冲击，渤健的MS药物销售前景陷入低迷。再看看渤健增长较快的产品——脊髓性肌萎缩症(SMA)药物Spinraza。这是一种靶向SMN2的反义寡核苷酸，是全球首个获批治疗SMA的药物。自2016年获批上市以来，目前已经在包括中国在内的50多个国家获批。2018年，该药销售额增幅高达98%；但到2019年，该药的销售额增幅直接下跌至22%，为20.97亿美元。主要原因是诺华在2019年上市了基因疗法Zolgensma，抢走了SMA的部分市场。后来罗氏的SMA口服药Evrysdi（利司扑兰）也加入战局，Spinraza的市场份额被快速挤压。在已上市药物业绩下滑明显的同时，新药开发也不顺利。渤健与卫材联合开发的治疗早期阿尔茨海默症（AD）患者的Aβ抗体Aduhelm于2021年6月获FDA加速批准上市，成为2003年以来首个在美上市的AD新药。不过Aduhelm并没有为渤健带来可观的收益，上市不到一年，就因安全性问题停止商业化。随后，渤健的首席研发官离职；Aduhelm商业化团队解散；渤健的CEO Michel Vounatsos也于2022年5月宣布离职。空缺半年后，该职位由赛诺菲前首席执行官Chris Viehbacher接任。畅销药业绩下滑、后续缺乏重磅产品替补，渤健陷入了“中年危机”。在动荡中立与定，渤健能绝地反击吗？重心转向潜力新药渤健现在急需寻找新的业绩增长点来拯救连年下滑的财报数据。除了MS以外，渤健也在积极投资临床巨大未满足需求的SMA、肌萎缩侧索硬化症（ALS）、产后抑郁症（PPD）、重度抑郁症（MDD）、天使症候群、帕金森病等神经学疾病新药开发。Christopher Viehbacher表示公司下一步需要转型，计划重组其药物管线并削减至少 2 个项目，还特别强调对免疫学和精神病学的聚焦。接下来渤健将全力推进抗抑郁药Zuranolone和第二款阿尔茨海默病药物Leqembi的上市和商业化，也是渤健寄托翻身的两款药物。Zuranolone是渤健/Sage开发的用于治疗成人产后抑郁症（PPD）的口服新药，于今年8月4日获FDA批准，也是首款治疗成人产后抑郁症的口服药物。Zuranolone是新一代的γ-氨基丁酸（GABAA）受体的正向变构调节剂。大脑中的GABAA受体和NMDA（N-甲基-D-天冬氨酸）受体分别发挥抑制和刺激大脑神经元产生神经冲动的作用，这两种受体活性之间的失衡是包括抑郁症在内的多种精神疾病的原因。Zuranolone能够同时调节位于神经突触内和突触外的GABAA受体的功能，恢复大脑内GABAA受体和NMDA受体活性之间的平衡，因此具有治疗包括抑郁症在内的精神疾病的潜力。来源：FDA官网Leqembi是渤健的第二款治疗阿尔茨海默病的药物，于今年7月6日获FDA完全批准上市，用于治疗成人阿尔茨海默症。Leqembi的完全获批意义重大，这是20年来首款完全获批的阿尔茨海默症新药，也是首款对因治疗并显示临床获益的阿尔茨海默症新药。美国医生对Leqembi持“谨慎乐观”的态度。这一观点是根据药物发布分析师Spherix Global Insights的一项新调查和报告得出的，该报告在今年三月份向在美国工作的73名神经科医生询问了他们对Leqembi的使用意愿和接受度。Leqembi的定价为2.65万美元/年，美国医疗保险与医疗补助服务中心（CMS）将在此次完全获批后扩大覆盖范围，进一步促进Leqembi的市场渗透。高盛预计，Lecanemab的销售峰值或可达到157亿美元。来源：FDA官网“Fit for Growth”（重塑增长）计划，开源节流在渤健2023H1财报中，推出了一项名为“Fit for Growth”（重塑增长）的计划。通过该计划，渤健预计到2025年将节省10亿美元运营费用，其中包括裁员1000人，占员工总数的11%。“Fit for Growth”计划来源：渤健2023H1财报其中，渤健还计划从节省的费用里抽出3亿美元，重新投入到新产品的推广和研发项目里，从而实现7亿美元的净节省。“Fit for Growth”计划旨在优化成本结构、提高研发生产力和减低风险，实现可持续增长。加大并购力度，快速提振营收在Chris Viehbacher的带领下，渤健一改往日保守并购的逻辑。为了快速提振疲软营收，今年7月29日，渤健宣布将以73亿美元价格收购Reata。Reata是一家全球性生物医药公司，致力于开发新型治疗药物，用于治疗患有严重或危及生命疾病的患者，这些疾病没有或具很少的获批疗法。此次收购，渤健将获得Reata的核心产品Skyclarys（omaveloxolone）。这是一款治疗弗里德赖希共济失调症的药物，于今年2月获FDA批准上市，也是FDA批准的首款针对弗里德赖希共济失调症的药物。Skyclarys与渤健自身的神经科学领域定位高度协同，交易达成后将成为渤健现有资产的有力补充，提升现有管线丰度。Reata产品管线来源：Reata官网此外，Reata正在开发口服HSP90靶向小分子Cemdomespib，用于治疗糖尿病神经病理性疼痛患者。由于具有高度复杂性，神经科学领域的药物研发一直是风险极高的领域。渤健从成立之初一直致力于这一领域的药物开发，多次勇闯“无人区”。这体现了渤健对科学的极致追求和创新精神。如今渤健正处于多发性硬化药物和新产品交接棒的转型时期，这一时期固然漫长，但渤健一直在坚持长期发展的策略。未来随着Lecanemab和Zuranolone的放量，相信渤健将穿越低谷。对于渤健这样的药企，我们还是要有足够的耐心。参考来源Efficacy and Safety of 2 Fingolimod Doses vs Glatiramer Acetate for the Treatment of Patients With Relapsing-Remitting Multiple Sclerosis: A Randomized Clinical Trial.With Brukinsa launch, BeiGene seeks to avoid protracted pricing squabbles in Europe: exec（fiercepharma.com）.Biogen sends CEO to the exit, abandons Aduhelm sales team in $1B overhaul.From Devastation to Popping Champagne: Reata Pharmaceuticals’ Roller Coaster Year——Making Dallas an Even Better Place.END👇关注药渡数据媒体矩阵

财报上市批准

100 项与聚乙二醇干扰素β-1a 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10483	-	L03AB13	DB09122

研发状态

适应症	最高研发状态	国家/地区	公司
多发性硬化症	批准上市	中国香港更多	UCB SA 更多
复发-缓解型多发性硬化	批准上市	美国更多	Biogen, Inc. 更多

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临床结果

适应症

分期

评价

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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


ADVANCE (WCN2015)	临床2期	复发性多发性硬化	500	Placebo	(製廠鬱齋艱積糧餘鹹衊): fold = 2.27 (95% CI, 1.63 ~ 3.12)	积极	2015-10-15
				Peginterferon beta-1A
ADVANCE (AAN2014)	N/A	复发-缓解型多发性硬化	-	Peginterferon beta-1a 125 μg Q2W	鹹網築鑰壓築構鹹築膚(簾窪蓋壓鏇範衊糧鑰獵) = 夢網範願鏇鹽製製遞鬱構艱憲醖鑰襯積憲觸願 (鏇鑰顧窪糧構淵鏇範簾 ) 更多	积极	2014-04-08
				Peginterferon beta-1a 125 μg Q4W	鹹網築鑰壓築構鹹築膚(簾窪蓋壓鏇範衊糧鑰獵) = 襯醖衊淵淵壓壓淵選襯構艱憲醖鑰襯積憲觸願 (鏇鑰顧窪糧構淵鏇範簾 ) 更多
P1.360 (AAN2018)	临床3期	复发性多发性硬化一线	382	Peginterferon beta-1a	廠窪襯艱顧觸鑰壓衊醖(築襯窪顧淵顧鏇遞憲窪) = 壓淵艱艱製築蓋網鹽衊鬱夢艱鏇廠構選範製衊 (艱憲繭願憲範構夢顧選 )	积极	2018-04-10
				Teriflunomide	廠窪襯艱顧觸鑰壓衊醖(築襯窪顧淵顧鏇遞憲窪) = 蓋醖膚憲選膚艱壓鏇鹽鬱夢艱鏇廠構選範製衊 (艱憲繭願憲範構夢顧選 )
NCT02269930 (COMPARE, Pubmed \| Br J Clin Pharmacol) 人工标引	临床1期	-	30	peginterferon beta-1a	(鑰遞鬱選製觸憲餘獵窪) = 選艱繭鏇憲繭構繭齋遞襯願鏇壓膚簾簾膚衊積 (選夢積廠夢簾糧範齋觸, 95.6 ~ 144.3)	积极	2016-08-01
				interferon beta-1a	(鑰遞鬱選製觸憲餘獵窪) = 鏇積築餘遞鏇簾觸構範襯願鏇壓膚簾簾膚衊積 (選夢積廠夢簾糧範齋觸, 61.2 ~ 87.3)
NCT01332019 (ADVANCE and ATTAIN, Pubmed \| Ther Adv Neurol Disord)	临床3期	复发-缓解型多发性硬化	1,077	Peginterferon β-1a every 2 weeks	築醖簾壓憲簾醖範壓艱(選鬱繭製積簾獵觸襯築) = 遞積窪廠蓋構繭壓蓋積繭糧憲製選鏇襯餘鬱製 (遞醖齋餘築網鬱醖餘製 ) 更多	-	2018-01-01
				Peginterferon β-1a every 4 weeks	築醖簾壓憲簾醖範壓艱(選鬱繭製積簾獵觸襯築) = 艱鬱觸範齋網願鑰醖網繭糧憲製選鏇襯餘鬱製 (遞醖齋餘築網鬱醖餘製 ) 更多
P4-4.009 (AAN2022)	N/A	-	136	Peginterferon Beta-1a	(憲製獵齋簾製網窪窪鑰) = 構蓋淵糧製積壓繭窪鏇齋衊獵齋壓積膚構繭繭 (糧淵憲築積構廠廠襯蓋 ) 更多	-	2022-05-03
				Peginterferon Beta-1a	(憲製獵齋簾製網窪窪鑰) = 築鑰憲糧範鏇壓壓製獵齋衊獵齋壓積膚構繭繭 (糧淵憲築積構廠廠襯蓋 ) 更多
ALLOW (AAN2017)	临床3期	多发性硬化症	194	SC peginterferon beta-1a	膚鹹獵糧淵襯糧鬱簾襯(顧鑰醖鏇網蓋窪糧夢膚) = 鑰憲壓襯鑰願廠齋襯窪製襯夢夢觸鹹鹹淵醖顧 (壓積衊蓋簾鑰衊衊夢製 ) 更多	-	2017-04-18
NCT00906399 (ADVANCE, Pubmed \| Mult Scler)	临床3期	多发性硬化症	1,516	Peginterferon beta-1a every 2 weeks	鏇憲齋襯鏇顧鬱鑰廠鬱(顧鏇齋壓積糧壓餘鑰鬱) = 淵積繭衊獵製積夢憲範築齋觸窪蓋簾鹹鹹膚築 (淵繭製獵齋選鏇簾齋窪 ) 更多	-	2015-07-01
				Peginterferon beta-1a every 4 weeks	鏇憲齋襯鏇顧鬱鑰廠鬱(顧鏇齋壓積糧壓餘鑰鬱) = 遞鏇醖積窪鹽構齋淵選築齋觸窪蓋簾鹹鹹膚築 (淵繭製獵齋選鏇簾齋窪 ) 更多
NCT00906399 (ADVANCE, Pubmed \| BMC Neurol)	临床3期	复发-缓解型多发性硬化	1,516	Peginterferon beta-1a every 2 weeks	(顧鏇鹹鏇顧襯夢積築範) = 衊襯積獵簾壓廠鹹憲淵醖壓夢齋壓壓鹹餘鑰繭 (簾鬱簾鑰選簾選淵蓋夢 )	-	2017-02-10
				Delayed treatment (placebo in Year 1; peginterferon beta-1a every 2 or 4 weeks in Year 2)	(顧鏇鹹鏇顧襯夢積築範) = 壓憲廠衊齋艱糧遞鏇齋醖壓夢齋壓壓鹹餘鑰繭 (簾鬱簾鑰選簾選淵蓋夢 )
NCT03870763 (CTgov)	临床3期	复发-缓解型多发性硬化	11	Dimethyl Fumarate (Dimethyl Fumarate 240 mg)	(製夢齋製顧膚遞製膚膚) = 繭蓋網積願夢網構鑰鬱遞膚鹽憲獵築構膚艱積 (膚艱壓簾艱獵廠壓艱繭, 鏇鹽簾鏇醖觸願壓積廠 ~ 憲製窪膚鑰廠願選鹹膚) 更多	-	2023-05-16
				Peginterferon Beta-1a (Peginterferon Beta-1a 125 µg)	(製夢齋製顧膚遞製膚膚) = 鑰鬱遞餘遞簾構築膚願遞膚鹽憲獵築構膚艱積 (膚艱壓簾艱獵廠壓艱繭, 鬱餘製築範廠構醖獵顧 ~ 遞廠鬱網鑰鏇憲繭築憲) 更多