A Prospective, Open-Label, Multicenter Study of the Efficacy, Safety, Tolerability, and Pharmacokinetics of Therapure PlasmaCap IG in Adults and Children With Primary Immune Deficiency Diseases

The purpose of this study is to investigate the efficacy, safety, tolerability, and pharmacokinetic profile of the investigational medicinal product (IMP) and to determine, on the basis of historical control data, how it compares with other 10% intravenous immunoglobulin (IGIV) products currently licensed in North America for the treatment of subjects with primary immune deficiency diseases (PIDD).

开始日期2017-09-05

申办/合作机构

Therapure Biopharma, Inc.

100 项与 Immune globulin (Evolve Biologics) 相关的临床结果

登录后查看更多信息

100 项与 Immune globulin (Evolve Biologics) 相关的转化医学

登录后查看更多信息

100 项与 Immune globulin (Evolve Biologics) 相关的专利（医药）

登录后查看更多信息

4,135

项与 Immune globulin (Evolve Biologics) 相关的文献（医药）

2025-01-01·JOURNAL OF AFFECTIVE DISORDERS

Parent perceptions of various treatment approaches for PANS and PANDAS

Article

作者: Muscal, Eyal ; Storch, Eric A. ; Dailey, Megan M ; Sadek, Sarah J ; Pinciotti, Caitlin M. ; Dailey, Megan M. ; Saxena, Kirti ; Goodman, Wayne K ; Goodman, Wayne K. ; Storch, Eric A ; Colombo, Gianna M ; Pinciotti, Caitlin M ; Colombo, Gianna M. ; Sadek, Sarah J.

Pediatric autoimmune neuropsychiatric disorder (PANDAS) is characterized by sudden, dramatic onset obsessive-compulsive disorder (OCD) following a Group A Streptococcus infection. Pediatric acute neuropsychiatric syndrome (PANS) refers to sudden, dramatic onset OCD and/or restricted eating triggered by infections and other inflammatory reactions. A variety of treatments have been utilized for PANS/PANDAS; however, there is no "gold standard" intervention protocol. Parental expectations of a given treatment have been found to improve a child's overall treatment experience; however, parent attitudes towards PANS/PANDAS treatments are unknown, which was the purpose of this study. An online survey was distributed to 208 parents of children with self-reported PANS/PANDAS. Treatments were grouped together within overarching categories (i.e., psychotherapy, psychiatric/psychotropic, inflammation/infection mitigation, supplements, lifestyle changes, and surgery). Categorically, parents rated inflammation/infection mitigation interventions and lifestyle changes as most appropriate, and psychiatric/psychotropic interventions as least appropriate. At the individual level, treatments including antibiotics, non-steroidal anti-inflammatory drugs, intravenous immunoglobulin, and family counseling received ratings between "appropriate" and "extremely appropriate" Alternatively, treatments including deep brain stimulation, transcranial magnetic stimulation, antidepressant medications, and exposure and response prevention received ratings between "inappropriate" and "extremely inappropriate." Study limitations include a lack of gender and race representation in our sample. Findings indicate a need for dissemination of current, relevant research to the parent population as well as further examination of the parent experience throughout onset, diagnosis, and treatment.

2024-12-31·NMC case report journal

Deep Brain Stimulation Leads to Long-term Improvement of Neuropathic Tremor due to Chronic Inflammatory Demyelinating Polyneuropathy: A Case Report

作者: Takabatake, Kazuhiko ; Hirata, Sachiko ; Fujimaki, Takamitsu ; Wakiya, Kenji ; Ujihara, Masaki ; Kobayashi, Masahito

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a peripheral neuropathy caused by immune-mediated demyelination, causing tremors in 3.9%-58% of affected patients. This neuropathic tremor may persist after treatment and is known to be refractory to conventional medication. We present two cases of neuropathic tremor due to CIDP in which deep brain stimulation (DBS) over a long-term period led to marked improvement. Case 1: A 66-year-old woman presented with severe 2-3-Hz resting, postural, and kinetic tremors of both hands. The tremor was refractory to medication but improved well after bilateral VIM-DBS. However, 2 months after the procedure, the tremor worsened and was accompanied by sensory disturbance in the extremities. A diagnosis of CIDP was made, and treatment with corticosteroids and intravenous immunoglobulin achieved remission 6 months later. Although there was residual tremor after CIDP remission, it has been well controlled by DBS for the last 10 years. Case 2: A 56-year-old man presented with a 6-year history of CIDP after developing sensory dullness and tremors in the extremities. The CIDP had gone into remission 1 year previously and the sensory deficits had improved, but the tremors had gradually worsened: severe 8-12-Hz postural, kinetic, and resting tremors were present in both upper extremities. Right VIM-DBS was performed and the tremors on the left side showed marked improvement. Over the next 8 years, the tremors were well controlled and there were no relapses of CIDP. DBS may achieve long-term improvement of neuropathic tremor caused by CIDP if the CIDP is in remission.

2024-12-31·JOURNAL OF DERMATOLOGICAL TREATMENT

Successful serial plasmapheresis for solar urticaria, a case report and literature review

Review

作者: Fassihi, Hiva ; Boontaveeyuwat, Einapak ; Willis, Fenella ; Sarkany, Robert P E

BACKGROUND/PURPOSE:

Amidst the emergence of new therapeutic options, traditional therapeutic plasmapheresis (TPE) used in diseases involving a toxic substance in the plasma, remains a viable alternative for cases of recalcitrant solar urticaria (SU). We emphasize the importance of documenting successful experience with repeated plasmapheresis to increase awareness amongst physicians and dermatologists regarding this effective treatment option.

MATERIAL AND METHOD:

We reported a case of recalcitrant SU that had not responded to a combination of H1-antihistamines, immunosuppressants, omalizumab and intravenous immunoglobulin. We introduced serial TPE, which involved two consecutive days of procedures for each course was introduced. We detailed the regimen and highlighted the clinical and objective benefits observed with multiple treatments. Additionally, we compared this to other plasmapheresis regimens and their treatment responses previously reported for solar urticaria.

RESULTS:

Our patient underwent serial TPE, totaling 42 procedures over five years. Following the last TPE session, phototesting showed a sustained prolongation of minimal urticating doses (MUDS), which exceeded the maximum tested doses across nearly all ultraviolet (UV) and visible light ranges, with the exception of the two short ultraviolet B (UVB) wavelengths. MUDs increased to 25 from 6 mj/cm2 at 307.5± 5nm, and to 500 from 15 mj/cm2 at 320 ± 10nm, before the initial TPE. In our review, we included five articles covering eight SU patients who received TPE. Of these, the five patients with positive intradermal tests responded particularly well immediately after treatment. However, the condition relapsed within two weeks in one patient and within two months in another. In contrast, the other three patients with negative intradermal tests, showed no significant benefits from the treatment. No serious side effects from TPE were reported amongst the patients.

CONCLUSIONS:

This review underscores the efficacy of serial plasmapheresis procedures in treating refractory cases of SU, high3lighting the robust results observed.

项与 Immune globulin (Evolve Biologics) 相关的新闻（医药）

2024-06-03

·BioSpace

Opinion: Annexon Tackles Guillain-Barre, Other Inflammatory Diseases with Novel Antibody

Pictured: A volcano erupting of damaged and inflamed nerves/ Nicole Bean for BioSpace This quarter, Annexon Biosciences expects to release Phase III trial results from its flagship program for Guillain-Barre syndrome. At the same time, the company is looking beyond GBS and investigating the possibility of fighting other inflammation-driven diseases with the antibody, ANX005. Historically, GBS has been difficult to treat and understand due to a relative lack of understanding around the cause of the disease. With causative associations ranging from viral infections to surgeries, GBS is a rare autoimmune disease affecting approximately 12,000 people in the U.S. and Europe each year. It can debilitate patients by paralyzing peripheral nerves in the limbs. There is currently no curative or long-term treatment for GBS, although intravenous immunoglobulin (IVIg, available as a generic from multiple companies) and plasmapheresis are used for acute bouts of the disease. As a pooled antibody from human blood sources, IVIg does not lead to better outcomes in subsequent administrations for GBS after the first dose due to poor prognosis, a 2021 randomized controlled study found. The study found that compared with placebo, patients who received a second IVIg dose had more serious adverse events such as thromboembolic events. By contrast, plasmapheresis is a procedure that uses a machine to separate plasma from blood cells, then replaces the patient’s plasma before returning the blood to the body. In doing so, plasmapheresis removes antibodies in the plasma, easing the inflammation characteristic of GBS. But the procedure requires access to a central line via catheters and carries a risk of bleeding and infections. ANX005’s mechanism is different: it inhibits a protein complex called C1q that is part of the classical complement pathway, which is in turn a component of the innate immune system. ANX005 thus targets the classical complement pathway upstream while—unlike IVIg—leaving the lectin pathway and the alternative complement pathway intact. In other words, patients treated with ANX005 will still be able to activate C3 via the lectin pathway and the alternative complement pathway, but not the classical complement pathway. This mechanism likely leads to less immunosuppression than does IVIg, enabling the body to continue defending itself against pathogens. In Phase Ib trials, ANX005 was administered along with IVIg in an open-label treatment arm to GBS patients. But in Phase III trials, Annexon is betting on comparing two different ANX005 doses compared to placebo. As this trial’s primary endpoints measure the GBS Disability Score at eight weeks and look at the number of patients with adverse effects for six months, there is much at stake in terms of safety and efficacy. By targeting GBS from a new angle, ANX005 is offering hope in a therapeutic area where corticosteroids and interferon-beta 1a have failed in the past. Beyond GBS While Annexon continues to assemble data on its Phase III GBS trial for upcoming release, it is also moving toward testing C1q inhibition in other inflammatory diseases, and these trials provide additional information about its safety profile. Huntington’s disease, a progressive movement disorder, is also driven by the activation of the classical complement pathway. Based on Phase IIa safety data from its Huntington’s disease trial with ANX005, ANX005’s adverse effect pro limited to mainly infusion-site reactions that appear only upon the first dose, making it safer than IVIg for these patients. By contrast, with various side effects that appear even with the first dose such as chills, fever, flushing, flu-like muscle pains or joint pains, fatigue, nausea, vomiting, and rash, IVIg is only used when necessary to boost immune responses due to systemic adverse effects. With ANX005 also being investigated for use in amyotrophic lateral sclerosis (ALS), where the drugmaker aims to reduce disease progression, there are certainly exciting developments ahead for Annexon in the coming months. Jia Jie Chen writes analyses focusing on drug development in the biotech and pharma industries for BioSpace. He has a doctorate degree in pharmacy and experiences ranging from biotech equity research to business intelligence analysis. Follow him on LinkedIn.

临床结果临床2期临床3期

2024-01-29

·PRNewswire

Halozyme Announces Takeda Received European Commission Approval for HYQVIA® Co-formulated with ENHANZE® as Maintenance Therapy for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

SAN DIEGO, Jan. 29, 2024 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme") today announced that Takeda received European Commission (EC) approval for HYQVIA® [Immune Globulin Infusion 10% (Human)] co-formulated with Halozyme's ENHANZE® drug delivery technology as maintenance therapy in patients of all ages with chronic inflammatory demyelinating polyneuropathy (CIDP) after stabilization with intravenous immunoglobulin therapy (IVIG). HYQVIA® also recently received U.S. Food and Drug Administration (FDA) approval as maintenance therapy for adults with CIDP. HYQVIA® is the only FDA and now EC-approved combination of immunoglobulin (IG) and hyaluronidase for CIDP and is administered by facilitated subcutaneous infusion. For patients with CIDP, HYQVIA® can be infused up to once monthly (every two, three or four weeks). HYQVIA® can be administered by a healthcare professional or self-administered in the patient's home after appropriate training. "The demand for new personalized maintenance treatment options for patients with CIDP continues to grow, and we are pleased to partner with Takeda to expand the HYQVIA offering to Europe," said Dr. Helen Torley, president and chief executive officer of Halozyme. The centralized marketing authorization for HYQVIA® in CIDP is valid in all EU member states as well as in Iceland, Liechtenstein, Norway and Northern Ireland. About Halozyme Halozyme is a biopharmaceutical company bringing disruptive solutions to significantly improve patient experiences and outcomes for emerging and established therapies. As the innovators of the ENHANZE® technology with the proprietary enzyme rHuPH20, Halozyme's commercially-validated solution is used to facilitate the delivery of injected drugs and fluids in order to reduce the treatment burden to patients. Having touched more than 800,000 patient lives in post-marketing use in seven commercialized products across more than 100 global markets, Halozyme has licensed its ENHANZE® technology to leading pharmaceutical and biotechnology companies including Roche, Takeda, Pfizer, AbbVie, Eli Lilly, Bristol Myers Squibb, Alexion, argenx, Horizon Therapeutics, ViiV Healthcare, Chugai Pharmaceutical and Acumen Pharmaceuticals. Halozyme also develops, manufactures and commercializes, for itself or with partners, drug-device combination products using its advanced auto-injector technology that are designed to provide commercial or functional advantages such as improved convenience and tolerability, and enhanced patient comfort and adherence. The Company has a commercial portfolio of proprietary products including Hylenex® and XYOSTED® and partnered commercial products and ongoing product development programs with several pharmaceutical companies including Teva Pharmaceuticals and Idorsia Pharmaceuticals. Halozyme is headquartered in San Diego, CA and has offices in Ewing, NJ and Minnetonka, MN. Minnetonka is also the site of its operations facility. For more information visit and connect with us on LinkedIn and Twitter. Safe Harbor Statement In addition to historical information, the statements set forth in this press release include forward-looking statements including, without limitation, statements concerning the Company's ENHANZE® drug delivery technology including the possible benefits and attributes of ENHANZE®, the possible method of action of ENHANZE®, its potential application to aid in the dispersion and absorption of other injected therapeutic drugs, facilitating more rapid delivery and administration of higher volumes of injectable medications through subcutaneous delivery and certain other benefits of ENHANZE® including lowering the treatment burden for patients and alleviating pressure on health care system resources, including potential out-of-hospital administration of HYQVIA® . Forward-looking statements regarding the Company's ENHANZE® business may include potential growth driven by our partners' development and commercialization efforts (including anticipated regulatory submissions, PDUFA dates, potential regulatory approvals, indications and product launches). These forward-looking statements are typically, but not always, identified through use of the words "believe," "enable," "may," "will," "could," "intends," "estimate," "anticipate," "plan," "predict," "probable," "potential," "possible," "should," "continue," and other words of similar meaning and involve risk and uncertainties that could cause actual results to differ materially from those in the forward-looking statements. Actual results could differ materially from the expectations contained in these forward-looking statements as a result of several factors, including unexpected results or delays in the growth of the Company's business, or in the development, regulatory review or commercialization of the Company's partnered products, regulatory approval requirements, unexpected adverse events or patient outcomes and competitive conditions. These and other factors that may result in differences are discussed in greater detail in the Company's most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commission. Contacts: Tram Bui VP, Investor Relations and Corporate Communications 609-359-3016 [email protected] Samantha Gaspar Teneo 617-877-9710 [email protected] References: European Medicines Agency. HyQvia product information. HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] U.S. Prescribing Information. SOURCE Halozyme Therapeutics, Inc.

上市批准免疫疗法

2024-01-19

·FiercePharma

Fierce Pharma Asia—Roche's TIGIT readout; Daiichi's ADC patent win; LegoChem's $411M raise

Tiragolumab's esophageal cancer readout, the latest episode in Daiichi Sankyo and Seagen's patent fight, and an antibody-drug conjugate specialist's fundraising effort made our news this week. Roche's TIGIT antibody has posted a positive phase 3 result that probably won't lead anywhere. In a win for Daiichi Sankyo, a U.S. agency has invalidated a Seagen patent. LegoChem has sold a large stake, worth $411 million, to fuel antibody-drug conjugate R&D. And more. 1. Roche racks up phase 3 TIGIT win, but trial design leaves doubts Roche’s TIGIT drug tiragolumab delivered positive data in a phase 3 esophageal cancer trial in Asia. But the trial design means the results probably won’t lead to any approvals. The SKYSCAPER-08 trial showed that combining tiragolumab and Roche's Tecentriq with chemotherapy helped patients live longer than chemo alone did. But there’s no data supporting the contribution of the TIGIT component. 2. US agency nixes Seagen patent, handing Daiichi Sankyo a win in long-running Enhertu entanglement The U.S. Patent and Trademark Office has invalidated Seagen’s so-called '039 patent at the center of a yearslong legal battle with Daiichi Sankyo. The patent covers certain peptides conjugated to an antibody through various linkers; Seagen had argued that Daiichi’s AstraZeneca-partnered Enhertu is associated with the technology. The two companies are simultaneously fighting in court. 3. LegoChem, riding ADC sugar rush, raises $411M from confectionery company The booming antibody-drug conjugate field has attracted Korea’s confectionery giant Orion to take stake in local firm LegoChem Bioscience. Orion has become LegoChem’s largest shareholder after spending 548.5 billion South Korean won ($411 million) for a 25% stake. The investment supports LegoChem’s plan to invest 1 trillion won in R&D over the next five years. 4. J&J, AZ/Daiichi Sankyo cancer therapies lead Clarivate's annual rundown of notable new meds In its annual drugs to watch list, Clarivate expects AstraZeneca and Daiichi Sankyo’s TROP2 antibody-drug conjugate datopotamab deruxtecan will reach $2.7 billion in sales in 2029. Astellas’ CLDN18.2 agent zolbetuximab, which was recently rejected by the FDA because of third-party manufacturing issues, is projected to reach $1.1 billion by 2029. 5. After 17-year courtship, Sun and Taro finally agree on $348M buyout Sun Pharma is finally able to bring Taro into its fold in its entirety. The Indian drugmaker agreed to pay $43 per share, or $348 million in total, for the remaining stake it didn’t already own in the Israeli manufacturer. Sun’s first attempt to acquire Taro came back in 2007, and the company had to sweeten its latest offer to win over Taro. 6. AstraZeneca to drop $26.5M in China for new production line for diabetes drugs: report AstraZeneca will invest $26.5 million to build a new production line at its manufacturing facility in China’s Taizhou. The line will be used to make the company’s Xigduo, which is a combination of dapagliflozin and metformin hydrochloride. The British pharma’s Taizhou base was launched in 2014 and is expected to have an annual output value of $1.4 billion, according to local reports. Other News of Note: 7. Takeda picks up 2nd FDA-approved indication for immune globulin treatment HyQvia 8. Bora plants its flag in the US with $210M acquisition of generics manufacturer Upsher-Smith 9. Windtree blows towards Lee's Pharma once again, signing new deal 10. Asia-based financial group launches $50M US biotech fund

上市批准临床结果并购临床3期抗体药物偶联物

100 项与 Immune globulin (Evolve Biologics) 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
原发性免疫缺陷疾病	临床3期	美国	Therapure Biopharma, Inc.	2017-09-05
原发性免疫缺陷疾病	临床3期	加拿大	Therapure Biopharma, Inc.	2017-09-05
原发性T细胞免疫缺陷	临床3期	美国	Evolve Biologics, Inc.初创企业	2017-08-30

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


AAAAI2019	临床3期	原发性免疫缺陷疾病	48	PlasmaCap IG	齋廠遞餘願選鏇願憲願(壓廠襯夢範淵膚餘艱網) = 31% (15/48) of subjects experienced a headache at some point, the rate of headaches per infusion rate was 1.8% (15/816 infusions; max. infusion rate 0.08 mL/min) 醖齋衊醖鹹淵窪膚鹹積 (獵衊夢鹹壓淵廠憲鹹齋 )	积极	2019-02-01