Ensayo clínico piloto, unicéntrico, controlado con placebo, a doble-ciego, aleatorio y cruzado con alfa-1-antitripsina humana en sujetos con fibromialgia.A single-centre, randomised, double-blind, controlled with placebo, cross-over, pilot clinical trial with human alpha-1-antitrypsin in subjects with fibromyalgia.

Start Date28 Aug 2006

Sponsor / Collaborator

Instituto Grifols SA

100 Clinical Results associated with Alpha-antitrypsin(PPL Therapeutics Plc)

100 Translational Medicine associated with Alpha-antitrypsin(PPL Therapeutics Plc)

100 Patents (Medical) associated with Alpha-antitrypsin(PPL Therapeutics Plc)

676

Literatures (Medical) associated with Alpha-antitrypsin(PPL Therapeutics Plc)

03 Oct 2024·Europace

Sex- and age-specific differences in the use of antiarrhythmic therapies among atrial fibrillation patients: a nationwide cohort study

Article

Author: Aro, Aapo L ; Inkovaara, Jaakko ; Teppo, Konsta ; Kalatsova, Ksenia ; Halminen, Olli ; Linna, Miika ; Mustonen, Pirjo ; Putaala, Jukka ; Salmela, Birgitta ; Penttilä, Tero ; Haukka, Jari ; Jaakkola, Jussi ; Hartikainen, Juha ; Lehto, Mika ; Airaksinen, K E Juhani

AbstractAimsAtrial fibrillation (AF) patients frequently require active rhythm control therapy to maintain sinus rhythm and reduce symptom burden. Our study assessed whether antiarrhythmic therapies (AATs) are used disproportionately between men and women after new-onset AF.Methods and resultsThe nationwide Finnish anticoagulation in AF registry-based linkage study covers all patients with new-onset AF in Finland during 2007–2018. Study outcomes included initiation of AATs in the form of antiarrhythmic drugs (AADs), cardioversion, or catheter ablation. The study population constituted of 229 565 patients (50% females). Women were older than men (76.6 ± 11.8 vs. 68.9 ± 13.4 years) and had higher prevalence of hypertension or hyperthyroidism, but lower prevalence of vascular disease, diabetes, renal disease, and cardiomyopathies than men. Overall, 17.6% of women and 25.1% of men were treated with any AAT. Women were treated with AADs more often than men in all age groups [adjusted subdistribution hazard ratio (aSHR) 1.223, 95% confidence interval (CI) 1.187–1.261]. Cardioversions were also performed less often on women than on men aged <65 years (aSHR 0.722, 95% CI 0.695–0.749), more often in patients ≥ 75 years (aSHR 1.166, 95% CI 1.108–1.227), while no difference between the sexes existed in patients aged 65–74 years. Ablations were performed less often in women aged <65 years (aSHR 0.908, 95% CI 0.826–0.998) and ≥75 years (aSHR 0.521, 95% CI 0.354–0.766), whereas there was no difference in patients aged 65–74 years.ConclusionWomen used more AAD than men in all age groups but underwent fewer cardioversion and ablation procedures when aged <65 years.

01 Oct 2024·Heart Rhythm

Catheter ablation vs antiarrhythmic therapy for atrial fibrillation in heart failure with preserved ejection fraction

Article

Author: DeLuca, Marisa R. ; Tarabichi, Yasir ; Ali, Bilal ; Verma, Beni R. ; Verma, Beni R ; Karim, Saima ; DeLuca, Marisa R

BACKGROUNDClinical outcomes of patients with atrial fibrillation (AF) and heart failure with preserved ejection fraction (HFpEF) treated with catheter ablation (CA) vs antiarrhythmic therapy (AAT) are not well known.OBJECTIVEThis study compared morbidity and mortality of patients with AF and HFpEF treated with CA vs AAT.METHODSAF and HFpEF patients from January 2017 to June 2023 were identified in TriNetX, a large global population-based database. Patients with prior diagnosis of heart failure with reduced ejection fraction (HFrEF) or crossover between AAT and CA were excluded. Baseline characteristics including age, sex, body mass index, type of AF, comorbidities, and cardiovascular medications were compared. The 2 groups were 1:1 propensity score matched for outcomes analysis. All-cause mortality, cerebrovascular accident/transient ischemic attack, and acute heart failure were compared with Kaplan-Meier curves.RESULTSPatients treated with CA (n = 1959) and AAT (n = 7689) were 1:1 propensity score matched, yielding 3632 patients with no significant differences in baseline characteristics. Compared with AAT, CA was associated with decreased mortality (9.2% vs 20.5%; hazard ratio [HR], 0.431; 95% confidence interval [CI], 0.359-0.518; P < .001). In addition, CA was associated with reduced HFpEF (HR, 0.638; 95% CI, 0.550-0.741; P < .001) and acute HFrEF (HR, 0.645; 95% CI, 0.452-0.920; P = .015). There was no difference in composite of cerebrovascular accident/transient ischemic attack (HR, 0.935; 95% CI, 0.725-1.207; P = .607).CONCLUSIONIn this retrospective study of patients with AF and HFpEF, CA was associated with lower mortality and risk of acute heart failure compared with AAT.

01 Sep 2024·Vascular Pharmacology

Ex vivo study on the human blood neutrophil circadian features and effects of alpha1-antitrypsin and lipopolysaccharide

Article

Author: Si, Wenzhang ; Welte, Tobias ; Sivaraman, Kokilavani ; Janciauskiene, Sabina ; Held, Julia ; Wrenger, Sabine ; Immenschuh, Stephan

AIMSNeutrophils perform various functions in a circadian-dependent manner; therefore, we investigated here whether the effect of alpha1-antitrypsin (AAT), used as augmentation therapy, is dependent on the neutrophil circadian clock. AAT is a vital regulator of neutrophil functions, and its qualitative and/or quantitative defects have significant implications for the development of respiratory diseases.METHODSWhole blood from 12 healthy women age years, mean (SD) 29.92 (5.48) was collected twice daily, 8 h apart, and incubated for 30 min at 37 °C alone or with additions of 2 mg/ml AAT (Respreeza) and/or 5 μg/ml lipopolysaccharide (LPS) from Escherichia coli. Neutrophils were then isolated to examine gene expression, migration and phagocytosis.RESULTSThe expression of CD14, CD16, CXCR2 and SELL (encoding CD62L) genes was significantly higher while CDKN1A lower in the afternoon than in the morning neutrophils from untreated blood. Neutrophils isolated in the afternoon had higher migratory and phagocytic activity. Morning neutrophils isolated from AAT-pretreated blood showed higher expression of CXCR2 and SELL than those from untreated morning blood. Pretreatment of blood with AAT enhanced migratory properties of morning but not afternoon neutrophils. Of all genes analysed, only CXCL8 expression was strongly upregulated in morning and afternoon neutrophils isolated from LPS-pretreated blood, whereas CXCR2 expression was downregulated in afternoon neutrophils. The addition of AAT did not reverse the effects of LPS.SIGNIFICANCEThe circadian clock of myeloid cells may affect the effectiveness of various therapies, including AAT therapy used to treat patients with AAT deficiency, and needs further investigation.

News (Medical) associated with Alpha-antitrypsin(PPL Therapeutics Plc)

19 Sep 2023

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Inhibrx Retains Rights to INBRX-101 for the Treatment of Alpha-1 Antitrypsin Deficiency Outside of the United States and Canada

SAN DIEGO, Sept. 19, 2023 /PRNewswire/ -- Inhibrx, Inc. (Nasdaq: INBX), a clinical-stage biopharmaceutical company dedicated to the development of therapeutics for oncology and rare diseases, announced today that Chiesi Farmaceutici S.p.A ("Chiesi") declined to exercise its option for the ex-North American rights to develop and commercialize INBRX-101 for the treatment of patients with emphysema due to Alpha-1 Antitrypsin Deficiency ("AATD"). Inhibrx's delivery of the European Medicines Agency ("EMA") scientific advice to Chiesi triggered a 60-day option period to obtain an exclusive license to develop and commercialize INBRX-101 for the treatment of patients with AATD outside of the United States and Canada. On September 18, 2023, Chiesi notified Inhibrx it was declining this option. "We believe Inhibrx is now optimally positioned with full global rights to INBRX-101. This enables broader strategic optionality, including potentially launching Graft versus Host Disease ("GvHD") as a first indication in Europe and Japan. The number of allogeneic transplants in Europe and Japan are double that of the United States and since GvHD therapies have a clear reimbursement path in these markets, we see this as a significant opportunity to help more patients desperately in need," said Mark Lappe, CEO of Inhibrx. About INBRX-101 and AATD INBRX-101 is a precisely engineered recombinant human AAT-Fc fusion protein designed to safely achieve and maintain levels of alpha-1 antitrypsin ("AAT"), found in healthy individuals with the potential for a less frequent dosing interval compared to the weekly infusion interval of the currently available plasma-derived AAT therapies. AATD is an inherited orphan disease affecting an estimated 100,000 patients in the United States and is characterized by deficient levels of the AAT protein, which causes loss of lung tissue and function and decreased life expectancy. Augmentation therapy with plasma-derived AAT is the current standard of care but does not maintain patients in the normal AAT range, requires frequent and inconvenient once-weekly IV dosing, and relies on plasma collection practices that might not be sustainable. Inhibrx is currently conducting the ElevAATe trial, which is designed as a randomized, controlled, double-blind, head-to-head superiority study examining INBRX-101 against plasma-derived AAT. The primary endpoint of the trial is the mean change in the average functional AAT ("fAAT") concentration as measured by anti-neutrophil elastase capacity from baseline to average serum trough fAAT concentration at steady state (Ctrough,ss). Secondary endpoints are a comparison of the mean change in fAAT concentration from baseline to fAAT average concentration at steady state (Cavg,ss), and the percentage of days with fAAT above the lower limit of the normal range during steady-state dosing. The initial read-out from the ElevAATe trial is expected to occur in late 2024 and the Company plans to meet with the FDA following completion of that study. About INBRX-101 and GvHD Hematopoietic stem cell transplants ("HSCTs") provide an often-curative option for many patients with hematological and oncological conditions, with multiple myeloma, Non-Hodgkin lymphoma, and acute myelogenous leukemia being the most common diseases requiring an HSCT treatment. GvHD is a significant problem that can occur post-surgery, usually after an allogeneic transplant, where the cells donated during the transplant see the recipient's body as foreign and attack the body. Strong clinical data and established guidelines for the use of AAT therapy already exist for acute cases of GvHD. Current clinical data for plasma-derived AAT therapies for the treatment of GvHD show promising response rates and favorable safety profiles over approved therapies, but half-life of the therapies is short and therefore requires dosing every two to four days. Based on the observed favorable safety profile of INBRX-101 to date coupled with its extended half-life, we believe INBRX-101 has the potential for greater efficacy due to the ability to dose at higher levels less frequently, while also eliminating the pathogenic risk from plasma-derived products, which can be life threatening in immunocompromised patients susceptible to infection. Inhibrx plans to initiate studies for both acute and chronic GvHD during the first half of 2024. About Inhibrx, Inc. Inhibrx is a clinical-stage biopharmaceutical company focused on developing a broad pipeline of novel biologic therapeutic candidates in oncology and orphan diseases. Inhibrx utilizes diverse methods of protein engineering to address the specific requirements of complex target and disease biology, including its proprietary protein engineering platforms. For more information, please visit . Forward-Looking Statements Inhibrx cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on Inhibrx's current beliefs and expectations. Forward-looking statements include, but are not limited to, statements regarding: Inhibrx's and its investigators' judgments and beliefs regarding the observed safety and efficacy to date of its therapeutic candidate, INBRX-101, discussions with and beliefs regarding future action by the FDA, including any potential accelerated regulatory pathway, whether a trial is registration-enabling, evaluations and observations of FDA discussions, statements and beliefs regarding the current standard of care for AAT and GvHD and the sustainability of current plasma collection practices, potential payor reimbursement, future clinical development, application and dosage of INBRX-101 and any presumption of or implied presumption of positive results from pre-clinical studies, Phase 1 clinical trials, or later clinical trials. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Inhibrx's business, including, without limitation, risks and uncertainties regarding: the initiation, timing, progress and results of its preclinical studies and clinical trials, and its research and development programs; its ability to advance therapeutic candidates into, and successfully complete, clinical trials; its interpretation of initial, interim or preliminary data from its clinical trials, including interpretations regarding disease control and disease response; the timing or likelihood of regulatory filings and approvals; the successful commercialization of its therapeutic candidates, if approved; the pricing, coverage and reimbursement of its therapeutic candidates, if approved; its ability to utilize its technology platform to generate and advance additional therapeutic candidates; the implementation of its business model and strategic plans for its business and therapeutic candidates; its ability to successfully manufacture therapeutic candidates for clinical trials and commercial use, if approved; its ability to contract with third-party suppliers and manufacturers and their ability to perform adequately; the scope of protection it is able to establish and maintain for intellectual property rights covering its therapeutic candidates; its ability to enter into strategic partnerships and the potential benefits of these partnerships; its estimates regarding expenses, capital requirements and needs for additional financing and financial performance; and other risks described from time to time in Inhibrx's filings with the U.S. Securities and Exchange Commission (the SEC), including under the heading "Risk Factors" in Inhibrx's Annual Report on Form 10-K filed with the SEC on March 6, 2023 and subsequently filed reports. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Inhibrx undertakes no obligation to update these statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. This press release contains estimates and other statistical data made by independent parties and by Inhibrx. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. Investor and Media Contact: Kelly Deck, CFO [email protected] 858-795-4260 SOURCE Inhibrx Inc.

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01 May 2023

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Significant Positive Shift in the Glioma Market with 13% CAGR During the Study Period (2019-2032) Expected by Analysts at DelveInsight

The dynamics of the glioma market are anticipated to change in the coming years owing to the positive outcomes of the emerging pipeline candidates during the developmental stage by key players, such as Bayer, VBL Therapeutics, Candel Therapeutics, AstraZeneca, DNAtrix, DelMar Pharmaceuticals, Chimerix, KaryoPharma, VBI Vaccines, Kazia Therapeutics, and others LAS VEGAS, May 1, 2023 /PRNewswire/ -- DelveInsight's Glioma Market Insights report includes a comprehensive understanding of current treatment practices, glioma emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan]. Key Takeaways from the Glioma Market Report As per DelveInsight analysis, the glioma market size in the 7MM was approximately USD 1 billion in 2022. According to the assessment done by DelveInsight, the estimated total incident glioma cases in the 7MM were approximately 47K in 2022. Leading glioma companies such as Bayer, Chimerix, Aivita Biomedical, Denovo Biopharma, Northwest Therapeutics, VBL Therapeutics, Laminar Pharmaceuticals, MedImmune, DNAtrix, Immunomic Therapeutics, Imvax, MimiVax, CNS Pharmaceuticals, Epitopoietic Research Corporation (ERC), Istari Oncology, SonALAsense, Kintara Therapeutics, Bristol Myers Squibb, Medicenna Therapeutics, BioMimetix, Eisai and Merck Sharp & Dohme, Kazia Therapeutics, Oblato, Genenta Science, Enterome, Inovio Pharmaceuticals, Karyopharm Therapeutics, VBI Vaccines, TME Pharma, Day One Biopharmaceuticals, Servier, Orbus Therapeutics, TVAX Biomedical, AnHeart Therapeutics, Beigene, SpringWorks Therapeutics, Forma Therapeutics, Hoffmann-La Roche, Incyte Corporation, Eli Lilly, and others are developing novel glioma drugs that can be available in the glioma market in the coming years. The promising glioma therapies in the pipeline include Regorafenib, ONC201, AV-GBM-1, Enzastaurin (DB-102), DCVax-L, Ofranergene Obadenovec (VB-111), LAM561 (2-OHOA), MEDI4736 (durvalumab), Tasadenoturev (DNX-2401), ITI-1000 (pp65 DC Vaccine), IGV-001, SurVaxM, Berubicin, GLIOVAC (Sitoiganap), Lerapolturev + Pembrolizumab, SONALA-001 + Exablate 2.0 Device, VAL-083 (dianhydrogalactitol), Pomalidomide, MDNA55, BMX-001, Lenvatinib ± Pembrolizumab, Paxalisib (GDC-0084), OKN-007, Temferon, EO2401, INO-5401+ INO-9012+ LIBTAYO (cemiplimab), Selinexor (KPT-330), Olutasidenib (FT-2102), VBI-1901, NOX-A12 (Olaptesed Pegol), Tovorafenib (DAY101), Vorasidenib (AG-881), Eflornithine, TVI-Brain-1, AB-218, BGB-290, Mirdametinib, FT-2102 (Olutasidenib), Vinblastine + Bevacizumab, Cobimetinib, PEMAZYRE (Pemigatinib), VERZENIO (abemaciclib) , and others. In December 2022, Chimerix announced the successful launch of the ONC201 Phase III ACTION study in patients with H3 K27M-mutant glioma. In June 2022, Day One Biopharmaceuticals announced positive initial data from its pivotal Phase II (FIREFLY-1) study with tovorafenib (DAY101) in relapsed pediatric low-grade glioma (pLGG). Discover which therapies are expected to grab the major glioma market share @ Glioma Market Report Glioma Overview Glioma is the most common kind of glial cell tumor in the central nervous system (CNS). Gliomas are infiltrative tumors that affect the brain tissue surrounding them. Glioblastoma is the most hazardous type of brain tumor, whereas pilocytic astrocytomas are the least dangerous. Previously, diffuse gliomas were classified into subgroups and grades depending on histopathologies, such as diffuse astrocytomas, oligodendrogliomas, or mixed gliomas/oligoastrocytomas. The specific etiology of glioma, like other primary brain tumors, remains unknown. However, certain variables may increase the risk of getting a brain tumor. Glioma symptoms vary depending on tumor type, size, location, and growth rate. Some common glioma symptoms include headache, nausea or vomiting, memory loss, urine incontinence, speech problems, and others. A medical history and a physical examination are used for glioma diagnosis. Glioma Epidemiology Segmentation DelveInsight estimates that there were approximately 47K incident cases of glioma in the 7MM in 2022. As per our analysis, the United States contributed to the largest incident population of glioma, acquiring ~40% of the 7MM in 2022. The glioma market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into: Total glioma incident cases Glioma grade-specific cases Glioma age-specific cases Glioma type-specific cases Download the report to understand which factors are driving glioma epidemiology trends @ Glioma Epidemiological Insights Glioma Treatment Market Glioma treatment is based on the type of glioma, its size and location, and the patient's unique characteristics. Chemotherapy and radiation therapy will be administered after surgery, particularly in individuals when the tumor cannot be removed entirely because it has invaded critical parts of the brain or is inaccessible. The typical glioma treatment protocol comprises surgery, chemotherapy, and radiation. Chemotherapy may include carmustine (BCNU), lomustine (CCNU), or gleostine (generic), cisplatin, carboplatin, etoposide, and irinotecan in case of low-grade glioma whereas temozolomide (TEMODAR) is the standard chemotherapy used in case of high-grade glioma majorly, Gliadel wafer implants (biodegradable discs injected with BCNU) are usually given as adjunctive treatment after surgery, although usage of Gliadel wafers remains a point of controversy due to its questionable survival benefits. Avastin (Genentech) and Temodar/Temodal (Merck), among others, are approved medications for HGG. However, due to patent expiration, the generic version of Temodar and biosimilars of Avastin are available on the glioma market. As generics and biosimilars are more cost-effective for patients than new medicines, intense competition exists among them. Furthermore, due to a lack of knowledge concerning bevacizumab's overall survival benefit, it is not recommended in unselected patients with newly diagnosed GBM. Currently, only one medication for LGG is approved, Tafinlar (dabrafenib) plus Mekinist (trametinib). Moreover, the current therapeutic market lacks a viable method for curing glioblastoma; therefore, the survival rate of people diagnosed with glioma remains poor. Glioma is incurable, and treatment choices are limited. Furthermore, the tumor has a high recurrence rate and a poor patient prognosis. In addition, there is currently no approved therapy for the unmethylated MGMT patient pool. To know more about glioma treatment guidelines, visit @ Glioma Management Glioma Therapies and Key Companies Regorafenib: Bayer ONC201: Chimerix AV-GBM-1: Aivita Biomedical Enzastaurin (DB-102): Denovo Biopharma DCVax-L: Northwest Therapeutics Ofranergene Obadenovec (VB-111): VBL Therapeutics LAM561 (2-OHOA): Laminar Pharmaceuticals MEDI4736 (durvalumab): MedImmune Tasadenoturev (DNX-2401): DNAtrix ITI-1000 (pp65 DC Vaccine): Immunomic Therapeutics IGV-001: Imvax SurVaxM: MimiVax Berubicin: CNS Pharmaceuticals GLIOVAC (Sitoiganap): Epitopoietic Research Corporation (ERC) Lerapolturev + Pembrolizumab: Istari Oncology SONALA-001 + Exablate 2.0 Device: SonALAsense VAL-083 (dianhydrogalactitol): Kintara Therapeutics Pomalidomide: Bristol Myers Squibb MDNA55: Medicenna Therapeutics BMX-001: BioMimetix Lenvatinib ± Pembrolizumab: Eisai and Merck Sharp & Dohme Paxalisib (GDC-0084): Kazia Therapeutics OKN-007: Oblato Temferon: Genenta Science EO2401: Enterome INO-5401+ INO-9012+ LIBTAYO (cemiplimab): Inovio Pharmaceuticals Selinexor (KPT-330): Karyopharm Therapeutics VBI-1901: VBI Vaccines NOX-A12 (Olaptesed Pegol): TME Pharma Tovorafenib (DAY101): Day One Biopharmaceuticals Vorasidenib (AG-881): Servier Eflornithine: Orbus Therapeutics TVI-Brain-1: TVAX Biomedical AB-218: AnHeart Therapeutics BGB-290: Beigene Mirdametinib: SpringWorks Therapeutics FT-2102 (Olutasidenib): Forma Therapeutics Vinblastine + Bevacizumab: Hoffmann-La Roche Cobimetinib: Hoffmann-La Roche PEMAZYRE (Pemigatinib): Incyte Corporation VERZENIO (abemaciclib): Eli Lilly Learn more about the FDA-approved drugs for glioma @ Drugs for Glioma Treatment Glioma Market Dynamics Glioma market growth can be ascribed to various factors, including an aging population, evolving research, better diagnostic tests, developing surgical procedures, improved radiotherapy techniques, and novel systemic medicines. Commercially, there is a greater emphasis on high-grade glioma than low-grade glioma because patients with low-grade glioma often live longer than those with high-grade glioma. As a result, just a few therapies are in late-stage studies, while the majority are in early-stage trials. In the case of GBM, on the other hand, the pipeline is robust, with numerous potential therapies in late and mid-stage research that have yet to be marketed. The pipeline includes drugs with varied modes of action and administration routes. It is interesting to note that the emerging market of glioma includes budding gene therapy, i.e., ofranergene obadenovec (VB-111) by VBL Therapeutics, followed by four vaccine/immunotherapy candidates such as VBI-1901, AV-GBM-1 and ITI-1000 (pp65 DC Vaccine), tasadenoturev (DNX-2401) by VBI Vaccines, Aivita Biomedical, Immunomic Therapeutics, and DNAtrix, respectively, and these therapies are going to alter the glioma market dynamics in the upcoming years. The current glioma market has been categorized based on the dominant treatment pattern across the 7MM, which shows slight variances in the overall prescription pattern. The forecast model considers surgery, chemotherapy/radiation therapy, bevacizumab + radiotherapy/chemotherapy, and OPTUNE with temozolomide. The anticipated launch of new therapies and improved integration of early patient screening, medication in secondary care and other clinical settings, research on optimum implementation methods, and increased awareness may eventually facilitate the development of successful treatment alternatives. However, there are a few challenges to the timely identification and treatment of these patients, such as a lack of a reliable biomarker that can aid in diagnosis and patient classification and identifying recurrence and signaling medication response. Furthermore, the failure rate of high-phased drug trials is significant, making it impossible to predict trial success, exacerbating the need for medicines that could assist ease the patient segment with this severe kind of cancer. Scope of the Glioma Market Report Therapeutic Assessment: Glioma current marketed and emerging therapies Glioma Market Dynamics: Conjoint Analysis of Emerging Glioma Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's Views, Analyst's Views, Glioma Market Access and Reimbursement Discover more about glioma drugs in development @ Glioma Clinical Trials Table to Contents Related Reports Glioma Epidemiology Forecast Glioma Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted glioma epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Glioma Pipeline Glioma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key glioma companies, including PTC Therapeutics, Ono Pharmaceuticals, Philogen, SpringWorks Therapeutics, Xennials Therapeutics, AnHeart Therapeutics, Istari Oncology, among others. High-Grade Glioma Pipeline High-Grade Glioma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key high-grade glioma companies, including PTC Therapeutics, Aadi, Lee's Pharmaceutical, among others. Malignant Glioma Pipeline Malignant Glioma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key malignant glioma companies, including Bexion Pharmaceuticals, Inc., Lee's Pharmaceutical Limited, Merck & Co, Oblato, Inc., OncoSynergy, Inc, among others. Recurrent Malignant Glioma Pipeline Recurrent Malignant Glioma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key recurrent malignant glioma companies, including Bexion Pharmaceuticals, Inc., Merck, Oblato, OncoSynergy, Inc, among others. Glioblastoma Pipeline Glioblastoma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key glioblastoma companies, including AstraZeneca, BioNTech, Merck, Pfizer, Roche, among others. 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It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn |Facebook|Twitter Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

Phase 3Phase 2VaccineImmunotherapy

20 Apr 2023

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Parkinson's Disease Clinical Trial Pipeline Space Brims With Novel Emerging Therapies with Over 120 Key Companies Working in the Domain | DelveInsight

Parkinson's disease is a progressive disorder that is caused by the degeneration of nerve cells in the part of the brain called the substantia nigra, which controls movement. These nerve cells die or become impaired, losing the ability to produce an important dopamine chemical. Studies have shown that symptoms of Parkinson's develop in patients with an 80% or greater loss of dopamine-producing cells in the substantia nigra. Most Parkinson's patients are treated with medications to relieve the symptoms of the disease. These medications work by stimulating the remaining cells in the substantia nigra to produce more dopamine (levodopa medications) or by inhibiting some of the acetylcholine that is produced (anticholinergic medications), therefore restoring the balance between the chemicals in the brain. LAS VEGAS, April 19, 2023 /PRNewswire/ -- DelveInsight's ' Parkinson's Disease Pipeline Insight – 2023 ' report provides comprehensive global coverage of available, marketed, and pipeline Parkinson's disease therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Parkinson's disease pipeline domain. Key Takeaways from the Parkinson's Disease Pipeline Report DelveInsight's Parkinson's disease pipeline report depicts a robust space with 120+ active players working to develop 140+ pipeline therapies for Parkinson's disease treatment. Key Parkinson's disease companies such as Cerevel Therapeutics, Inhibikase Therapeutics, Neuraly, Peptron, Biogen, Roche, Brain Neurotherapy Bio, Inc., Modag, Annovis Bio Inc., BioVie Inc., United Neuroscience Ltd., Luye Pharma Group, AbbVie, UCB Biopharma SRL, InnoMedica Schweiz AG, Integrative Research Laboratories AB, H. Lundbeck A/S, Shanghai WD Pharmaceutical Co., Ltd., Cerevance Beta, Inc., Nobilis Therapeutics Inc., BlueRock Therapeutics, Taiwan Mitochondrion Applied Technology Co., Ltd., NeuroDerm Ltd., Pharma Two B, Serina Therapeutics, Sun Pharma Advanced Research Company Limited, CuraSen Therapeutics, Inc., Takeda, Xoc Pharmaceuticals, Sumitomo Pharma Co., Ltd., XWPharma, Athira Pharma, Aptinyx, VistaGen Therapeutics, Inc., 1ST Biotherapeutics, Inc., Novo Nordisk A/S, AstraZeneca, MedImmune, Anavex Life Sciences Corp., Living Cell Technologies, Intra-Cellular Therapies, Inc., Alkahest, Inc., Kissei Pharmaceutical Co., Ltd., and others are evaluating new Parkinson's disease drugs to improve the treatment landscape. Promising Parkinson's disease pipeline therapies in various stages of development include Tavapadon, IkT-148009, NLY01, PT320, BIIB122, Prasinezumab, KDT-3594, AAV2-GDN, AKST4290, anle138b, ITI-214, NTCELL, Buntanetap, ANAVEX2-73, ATH-1017, NE3107, MEDI1341, AZD0328, Liraglutide, UB-312, LY03003, FB-101, AV-101, ABBV-951, NYX-458, DSP-9632P, Valiloxybate, SER-214, UCB7853, TAK-071, XC130-A10H, K0706, Talineuren, Pirepemat, Lu AF28996, WD-1603, CVN424, CST-103, NBTX-001, MSK-DA01, Aadipose-Derived Mesenchymal Stem Cells, ND0612, P2B001, 1ST-103, 1ST-105, and others. In January 2023, Neurocrine Biosciences and Voyager Therapeutics announced the formation of a new strategic collaboration to advance multiple gene therapies for the treatment of neurological diseases. The collaboration includes Voyager's preclinical, intravenously administered GBA1 gene therapy program for Parkinson's and other GBA1-mediated diseases, combining a GBA1 gene replacement payload with novel capsids from Voyager's TRACERTM (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) platform. In addition, Neurocrine Biosciences and Voyager have agreed to collaborate on three new gene therapy programs directed to rare CNS targets, each also leveraging Voyager's novel TRACER capsids. In October 2022, Biogen Inc. and Denali Therapeutics Inc. announced that dosing has commenced in the global Phase 3 LIGHTHOUSE study to evaluate the efficacy and safety profile of BIIB122 (DNL151), as compared to placebo in approximately 400 participants with Parkinson's disease and a confirmed pathogenic mutation in the leucine-rich repeat kinase 2 (LRRK2) gene. In September 2022, Neuron23, Inc. an early-stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases, and QIAGEN announced a collaboration to develop a companion diagnostic for Neuron23's LRRK2 inhibitor for Parkinson's disease. Request a sample and discover the recent advances in Parkinson's disease drug treatment @ Parkinson's Disease Pipeline Report The Parkinson's disease pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage Parkinson's disease drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the Parkinson's disease clinical trial landscape. Parkinson's Disease Overview Parkinson's disease is a progressive disorder caused by nerve cell degeneration in the substantia nigra, a part of the brain that controls movement. These nerve cells die or become impaired, losing the ability to produce dopamine, an important chemical. According to research, symptoms of Parkinson's disease develop in patients who have an 80% or greater loss of dopamine-producing cells in the substantia nigra. Dopamine works typically in tandem with other neurotransmitters to help coordinate the millions of nerve and muscle cells involved in the movement. This balance is disrupted when there is insufficient dopamine, resulting in tremors (trembling in the hands, arms, legs, and jaw), rigidity (stiffness of the limbs), slowness of movement, and impaired balance and coordination - the hallmark Parkinson's disease symptoms. The cause of Parkinson's disease is largely unknown. However, as potential causes of the disease, theories involving oxidative damage, environmental toxins, genetic factors, and accelerated aging have been discussed. The majority of Parkinson's patients are given medications to alleviate their symptoms. These Parkinson's disease medications work by either stimulating the remaining cells in the substantia nigra to produce more dopamine (levodopa medications) or inhibiting some of the acetylcholine produced (anticholinergic medications), restoring the balance of chemicals in the brain. Find out more about drugs for Parkinson's disease @ New Parkinson's Disease Drugs A snapshot of the Parkinson's Disease Pipeline Drugs mentioned in the report: Learn more about the emerging Parkinson's disease pipeline therapies @ Parkinson's Disease Clinical Trials Parkinson's Disease Therapeutics Assessment The Parkinson's disease pipeline report proffers an integral view of Parkinson's disease emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Parkinson's Disease Pipeline Report Coverage: Global Parkinson's Disease Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Parkinson's Disease Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Parkinson's Disease Therapeutics Assessment By Route of Administration: Inhalation, Intranasal, Intravenous, Oral, Parenteral, Subcutaneous Parkinson's Disease Therapeutics Assessment By Molecule Type: Antibody, Antisense oligonucleotides, Immunotherapy, Monoclonal antibody, Peptides, Protein, Recombinant protein, Small molecule, Stem Cell, Vaccine Parkinson's Disease Therapeutics Assessment By Mechanism of Action: Dopamine D1 receptor agonists, Dopamine D5 receptor agonists, Bcr-abl tyrosine kinase inhibitors, Glucagon like peptide 1 receptor agonists, LRRK2 protein inhibitors, Alpha-synuclein inhibitors, Dopamine receptor agonists Key Parkinson's Disease Companies: Cerevel Therapeutics, Inhibikase Therapeutics, Neuraly, Peptron, Biogen, Roche, Brain Neurotherapy Bio, Inc., Modag, Annovis Bio Inc., BioVie Inc., United Neuroscience Ltd., Luye Pharma Group, AbbVie, UCB Biopharma SRL, InnoMedica Schweiz AG, Integrative Research Laboratories AB, H. Lundbeck A/S, Shanghai WD Pharmaceutical Co., Ltd., Cerevance Beta, Inc., Nobilis Therapeutics Inc., BlueRock Therapeutics, Taiwan Mitochondrion Applied Technology Co., Ltd., NeuroDerm Ltd., Pharma Two B, Serina Therapeutics, Sun Pharma Advanced Research Company Limited, CuraSen Therapeutics, Inc., Takeda, Xoc Pharmaceuticals, Sumitomo Pharma Co., Ltd., XWPharma, Athira Pharma, Aptinyx, VistaGen Therapeutics, Inc., 1ST Biotherapeutics, Inc., Novo Nordisk A/S, AstraZeneca, MedImmune, Anavex Life Sciences Corp., Living Cell Technologies, Intra-Cellular Therapies, Inc., Alkahest, Inc., Kissei Pharmaceutical Co., Ltd., and others. Key Parkinson's Disease Pipeline Therapies: Tavapadon, IkT-148009, NLY01, PT320, BIIB122, Prasinezumab, KDT-3594, AAV2-GDN, AKST4290, anle138b, ITI-214, NTCELL, Buntanetap, ANAVEX2-73, ATH-1017, NE3107, MEDI1341, AZD0328, Liraglutide, UB-312, LY03003, FB-101, AV-101, ABBV-951, NYX-458, DSP-9632P, Valiloxybate, SER-214, UCB7853, TAK-071, XC130-A10H, K0706, Talineuren, Pirepemat, Lu AF28996, WD-1603, CVN424, CST-103, NBTX-001, MSK-DA01, Aadipose-Derived Mesenchymal Stem Cells, ND0612, P2B001, and others. Dive deep into rich insights for new drugs for Parkinson's disease treatment; visit @ Parkinson's Disease Medications Table of Contents For further information on the Parkinson's disease pipeline therapeutics, reach out @ Parkinson's Disease Drug Treatment Related Reports Parkinson's Disease Market Parkinson's Disease Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Parkinson's disease companies, including Cerevel Therapeutics, Inhibikase Therapeutics, Neuraly, Peptron, Biogen, Roche, among others. Parkinson's Disease Epidemiology Forecast Parkinson's Disease Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and Parkinson's disease epidemiology trends. Dementia with Lewy Bodies Pipeline Dementia with Lewy Bodies Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key dementia with Lewy bodies companies, including Eli Lilly and Company, CuraSen Therapeutics, Inc., Athira Pharma, Aptinyx Inc., among others. Dementia with Lewy Bodies Epidemiology Forecast Dementia with Lewy Bodies Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted dementia with Lewy bodies epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Dementia Market Dementia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key dementia companies, including Hoffmann-La Roche, Forest Laboratories, Inc., Janssen Pharmaceuticals, Inc., Merck and Co., Inc., Novartis, among others. Parkinson's Disease-Related Dementia Market Parkinson's Disease-Related Dementia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Parkinson's disease-related dementia companies, including AbbVie, UCB Biopharma SRL, InnoMedica Schweiz AG, Integrative Research Laboratories AB, H. Lundbeck A/S, Shanghai WD Pharmaceutical Co., Ltd., Cerevance Beta, Inc., among others. Other Trending Reports Tay-sachs Disease Or Gm2 Gangliosidosis Market | Onycholysis Market | Diagnostic Imaging Equipment Market | Chemotherapy-induced Peripheral Neuropathy Market | Global Electrophysiology Devices Market | Anaphylaxis Market | Atherectomy Devices Market | Helicobacter Pylori Infections Market | Ophthalmic Imaging Equipment Market | Androgenetic Alopecia Market | Allergic Rhinitis Market | Chronic Inflammatory Demyelinating Polyneuropathy Market | Chronic Inflammtory Demyelinating Polyneuropathy Market | Colorectal Cancer Crc Market | Opioid Induced Constipation Market | Vertigo Market | Bone Anchored Hearing Systems Market | Wound Closure Devices Market | Hip Replacement Devices Market | Hemodynamic Monitoring Systems Market | Egfr Non-small Cell Lung Cancer Market | Helicobacter Pylori Infection Market | Hyperkalemia Market | Polycythemia Market Neurostimulation Devices Market | Carpal Tunnel Syndrome Market | Ventilator Market | Cerebral Aneurysm Market | Alpha Antitrypsin Market | Binge Eating Disorder Market | Bunion Market | Concussions Market Size | Exocrine Pancreatic Insufficiency Market | Healthcare Due Diligence Services | Minimal Residual Disease Market | Hypertrophic Scar Market | Lung Fibrosis Market | Anterior Uveitis Market | 22q11.2 deletion syndrome Market | X-Linked Retinitis Pigmentosa (XLRP) Market | Acute Radiation Syndrome Market | Alpha-1 Protease Inhibitor Deficiency Market | Androgenetic Alopecia Market | Hyperlipidemia Market | Cardiotoxicity Market | Hypertrophic Cardiomyopathy Market | Fatty Acid Oxidation Disorders (FAODs) Market | Androgen Insensitivity Syndrome Market | Emphysema Market | Canaloplasty Market | Dravet Syndrome Market | Celiac Disease Market | Chlamydia Infections Market | Syphilis Market | Renal Tubular Acidosis Market | Palmoplantar Pustulosis (PPP) Market | Aplastic Anemia Market | Bacterial Pneumonia Market | B cell Chronic Lymphocytic Leukemia Market | B cell Lymphomas Market | Behcets Disease Market Neoantigen-based Personalized Cancer therapeutic Vaccines Competitive Landscape and Market Forecast—by 2035 | Glioblastoma Market Related Healthcare Services Healthcare Consulting Healthcare Competitive Intelligence Services Healthcare Asset Prioritization Services About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn |Facebook|Twitter Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

Phase 3

100 Deals associated with Alpha-antitrypsin(PPL Therapeutics Plc)

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Indication	Highest Phase	Country/Location	Organization	Date
Alpha 1-Antitrypsin Deficiency	Phase 2	-	PPL Therapeutics Plc	-
Alpha 1-Antitrypsin Deficiency	Phase 2	-	Bayer AG	-
Cystic Fibrosis	Phase 2	-	Bayer AG	-
Cystic Fibrosis	Phase 2	-	PPL Therapeutics Plc	-
Respiratory Distress Syndrome, Adult	Phase 2	-	Bayer AG	-
Respiratory Distress Syndrome, Adult	Phase 2	-	PPL Therapeutics Plc	-

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


P219 (EBMT2023)	Not Applicable	Steroid Refractory Graft Versus Host Disease	-	Alpha-1-antitrypsin	(ygdggtwwsq) = The most frequent infection was cytomegalovirus reactivation (viremia) and occurred in all patients jyphzjbwvv (ctpxplvlts )	Positive	23 Apr 2023
ERS2021	Not Applicable	Pulmonary Disease, Chronic Obstructive PiZZ	-	alpha-1-antitrypsin	uyxtwtdpor(iaztugjfmj) = eiyzyxmrgt cklcfiktpl (cqqwumshxc )	-	05 Sep 2021
				emphysema PiZZ patients without AAT augmentation therapy	uyxtwtdpor(iaztugjfmj) = uttrlbemff cklcfiktpl (cqqwumshxc )
EULAR2017	Not Applicable	Granulomatosis With Polyangiitis α-1-antitrypsin (AAT) \| proteinase 3 (PR3)	72	Alpha-1-Antitrypsin	xeizikrojd(ccfitldjsx) = dggupetpka qbimtqtubl (rzzslzlewp ) View more	-	14 Jun 2017
				Alpha-1-Antitrypsin	xeizikrojd(ccfitldjsx) = zaqmgcbpey qbimtqtubl (rzzslzlewp ) View more
ERS2011	Not Applicable	Emphysema PiZ \| PiSZ	-	alpha-1-antitrypsin	eczwkewauy(joxbduveki) = yusojdczff ugyamchqei (dxtfwgnsxz )	-	01 Sep 2011
				alpha-1-antitrypsin	eczwkewauy(joxbduveki) = dqifeghlls ugyamchqei (dxtfwgnsxz )
RTOG 9601 (ASCO_GU2011)	Phase 3	Salivary Gland Adenoma, Pleomorphic \| Prostatic Cancer	771	RT + AAT	cdtwnuuxsj(clwvzfntik) = nnytbkgtcl akadmnsufj (ljscrpqiwd ) View more	-	01 Mar 2011
				RT alone	cdtwnuuxsj(clwvzfntik) = uhamvqljgb akadmnsufj (ljscrpqiwd ) View more

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