Vasculitis

Adverum Biotechnologies Reports First Quarter 2024 Financial Results and Provides Pipeline Highlights

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torsdag, maj 9, 2024
Adverum Biotechnologies, FDA, AMD, Patient, Inflammation, Retinitis, Investment, Safety, CST, Research, Chorioretinitis, Compensated emancipation, Vascular occlusion, Employment, Macula, Episcleritis, Data, OPTIC, Three Months, Vasculitis, Society, LUNA, Intraocular pressure, VEGF, ASRS, Injection, Pharmaceutical industry

REDWOOD CITY, Calif., May 09, 2024 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company pioneering the use of gene therapy as a new standard of care for highly prevalent ocular diseases, today reported financial results for the first quarter of 2024. The company also provided recent pipeline highlights.

Key Points: 
  • “Our presentation will feature a 26-week landmark interim analysis, which is expected to serve as the basis of our FDA end-of-Phase 2 meeting.
  • The award was granted under the Adverum Biotechnologies, Inc. 2017 Inducement Plan, as amended and restated, and will vest over four years, subject to continued service with Adverum.
  • Stock-based compensation expense included in research and development expenses was $1.1 million for the first quarter of 2024.
  • Stock-based compensation expense included in general and administrative expenses was $3.0 million for the first quarter of 2024.

Annexon Presents New Neuroprotection Data Showing ANX007 Protects Vision and Vision-Associated Structures in Geographic Atrophy at ARVO 2024 Annual Meeting

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tisdag, maj 7, 2024
Brain, ARCHER, Complement component 1q, CNV, Research, Microglia, Incidence, Patient, Eye, Pivotal, Vasculitis, Paratriathlon, Retinal pigment epithelium, Geographic atrophy, RPE, Atrophy, Annual general meeting, Synapse, ARVO, European Medicines Agency, Overalls, Visual acuity, Medical imaging

BRISBANE, Calif., May 07, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a biopharmaceutical company advancing a late-stage clinical platform of novel therapies for people living with devastating classical complement-mediated neuroinflammatory diseases of the body, brain, and eye, today presented new analyses of ANX007 from the Phase 2 ARCHER trial in geographic atrophy (GA), and new preclinical data on the role of C1q in the pathogenic elimination of photoreceptor synapses and their protection with C1q blockade in GA. ANX007 is a first-in-class, non-pegylated antigen-binding fragment (Fab) designed to block C1q and activation of the classical complement cascade locally in the eye with an intravitreal formulation, and is the first therapeutic candidate for the treatment of GA to receive Priority Medicine (PRIME) designation by the European Medicines Agency. The data were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting.

Key Points: 
  • The data were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting.
  • “We are pleased to present additional clinical data from the ARCHER trial that are the first to show preservation of both vision and relevant anatomical structures following ANX007 treatment,” said Douglas Love, president and chief executive officer of Annexon.
  • “Importantly, the statistically significant preservation of photoreceptor anatomy measured by ellipsoid zone change highlights protection of key retinal structures associated with vision.
  • These two registrational trials are designed to confirm the Phase 2 ARCHER findings of protection against vision loss and underscore the unique neuroprotective mechanism of action of ANX007 and its competitive differentiation in visual function.

AMGEN HIGHLIGHTS NEW COPD, ASTHMA AND VASCULITIS RESEARCH AT ATS 2024

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onsdag, maj 1, 2024
AMGN, MPA, Effectiveness, Political moderate, Prednisone, ATS, Chronic obstructive pulmonary disease, Emphysema, Polyp (medicine), Inflammation, Hospital, Exercise, ANCA, Post hoc analysis, Abstract, PDT, Research and development, Biomarker, Initiation, Conference, Development, Literature, Vasculitis, History, GPA, Clinical, Amgen, Bronchitis, COPD, Pharmaceutical industry

THOUSAND OAKS, Calif., May 1, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced the presentation of new respiratory data at the American Thoracic Society (ATS) 2024 International Conference taking place May 12-22 in San Diego. Thirteen abstracts supporting two approved medicines and one investigational treatment will be presented.

Key Points: 
  • THOUSAND OAKS, Calif., May 1, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced the presentation of new respiratory data at the American Thoracic Society (ATS) 2024 International Conference taking place May 12-22 in San Diego.
  • Thirteen abstracts supporting two approved medicines and one investigational treatment will be presented.
  • "Our efforts underscore Amgen's commitment to pioneering new treatments for respiratory diseases with currently limited treatment options."
  • Data from the COURSE trial will be presented within the Clinical Trials Symposium.

Recludix Pharma Announces the Appointment of Dr. Ajay Nirula, Accomplished Pharma Executive and Immunology Expert, as Executive Vice President and Head of Research and Development, and Strengthens Scientific Advisory Board with the Addition of Dr. Adrian

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tisdag, april 23, 2024
Nature Immunology, School, University, Cancer, Biogen, COVID, SAB, Remdesivir, Medical school, Merck & Co., Los Angeles School, Eli Lilly, Amgen, Developmental Biology (journal), Inflammation, STAT3, Molecular biology, Yale University, Research, Lupus, STAT6, Growth, SH2, JAMA, Eli Lilly and Company, UCSF, Patient, Doctor of Pharmacy, Rheumatoid arthritis, Psoriasis, Vasculitis, Morphic, Lilly, Multiple sclerosis, Drug discovery, SAN, Division, Investigational New Drug, Science and nature, Pharmaceutical industry

“We are thrilled to welcome Ajay and Adrian to Recludix,” said Nancy Whiting, PharmD, CEO of Recludix. “Ajay joins us from Eli Lilly with over two decades of executive leadership experience spanning all stages of drug discovery and development. He has extensive expertise in immunology and has led the advancement and strategic oversight of deep pipelines with more than ten product candidates reaching the clinic during his Lilly tenure. At Recludix, we have a robust pipeline of validated targets, starting with our two lead programs -- oral and reversible inhibitors of STAT3 and STAT6. Additionally, our proprietary drug discovery platform has enabled us to drug many other exciting previously undruggable SH2 domain targets. Ajay’s deep experience leading a broad discovery and development immunology portfolio – both internally and through strategic partnerships -- is ideally suited to capitalize on the many opportunities at Recludix.”

Key Points: 
  • “We are thrilled to welcome Ajay and Adrian to Recludix,” said Nancy Whiting, PharmD, CEO of Recludix.
  • “Ajay joins us from Eli Lilly with over two decades of executive leadership experience spanning all stages of drug discovery and development.
  • At Recludix, we have a robust pipeline of validated targets, starting with our two lead programs -- oral and reversible inhibitors of STAT3 and STAT6.
  • Additionally, our proprietary drug discovery platform has enabled us to drug many other exciting previously undruggable SH2 domain targets.

Ocular Therapeutix™ Announces Positive Topline Phase 1 Data for AXPAXLI™ in Diabetic Retinopathy

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torsdag, april 18, 2024
Steps, FDA, AMD, Uveitis, Inflammation, Safety, Diabetic retinopathy, MD, Retina, Incidence, Patient, Eye, Vasculitis, CSO, DME, Consultant, Phase 3, Quality of life, DR, Pharmaceutical industry

BEDFORD, Mass., April 18, 2024 (GLOBE NEWSWIRE) -- Ocular Therapeutix, Inc. (NASDAQ:OCUL) (“Ocular”), a biopharmaceutical company committed to enhancing people’s vision and quality of life through the development and commercialization of innovative therapies for wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR), and other diseases and conditions of the eye, today announced positive topline results from the Phase 1 HELIOS study evaluating AXPAXLI versus a sham control in patients with moderately severe to severe non-proliferative diabetic retinopathy (‘NPDR’, NCT05695417) without diabetic macular edema (DME). Ocular plans to present the study results at an upcoming meeting.

Key Points: 
  • Ocular plans to present the study results at an upcoming meeting.
  • Next Steps: Positive HELIOS results support decision to move directly to a Phase 3 study with AXPAXLI in patients with NPDR, pending discussions with the FDA
    “We are pleased to announce positive topline data for AXPAXLI in patients with diabetic retinopathy.
  • “It is promising that the efficacy signals seen to date in this Phase 1 study consistently favor AXPAXLI.
  • These data demonstrated DRSS improvements and durability for up to 40 weeks,” said Dilsher Dhoot, MD.

FASENRA approved for treatment of children aged 6 to 11 with severe asthma

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torsdag, april 11, 2024
Health, FDA, General Health, Research, Science, Pharmaceutical, Biotechnology, Efficacy, Safety, Gastroenterology, HES, Chronic obstructive pulmonary disease, Sinusitis, Social media, Polyp (medicine), Pharmacokinetics, Rheumatology, Conference call, Asthma, TATE, AstraZeneca, FY, Hypereosinophilic syndrome, Death, ClinicalTrials.gov, Dermatology, Conference, COPD, Webcast, Growth, Disease, Vasculitis, Respiratory Medicine, EGPA, Immunology, Metabolism, Diagnosis, Biomarker, PD, Biopharmaceutical, Ageing, Pharmaceutical industry

TATE was an open-label, Phase III trial evaluating the safety of FASENRA in children aged 6 to 11 years with severe eosinophilic asthma.

Key Points: 
  • TATE was an open-label, Phase III trial evaluating the safety of FASENRA in children aged 6 to 11 years with severe eosinophilic asthma.
  • The Company aims to transform the treatment of asthma and COPD by focusing on earlier biology-led treatment, eliminating preventable asthma attacks, and removing COPD as a top-three leading cause of death.
  • Benralizumab in children with severe eosinophilic asthma: pharmacokinetics and long-term safety (TATE study).
  • Severe uncontrolled asthma in children: practical approach on diagnosis and management.

Orphan designation: Methyl-(1-{[6-{[(1S)-1-cyclopropylethyl]amino}-2-(pyrazolo[5,1-b][1,3]thiazol-7-yl)-pyrimidin-4-yl]carbonyl}piperidin-4-yl)carbamate mono(4-methylbenzenesulfonate) Treatment of eosinophilic granulomatosis with polyangiitis, 12/01/[...]

Retrieved on: 
torsdag, april 18, 2024
Patient, Committee, EMA, IRIS, COMP, European Commission, Vasculitis, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of eosinophilic granulomatosis with polyangiitis on 12 January 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Adverum Biotechnologies Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Pipeline Highlights and Corporate Updates

Retrieved on: 
måndag, mars 18, 2024
Research, Adverum Biotechnologies, Vasculitis, Three Months, Macula, Chorioretinitis, Inflammation, Patient, Episcleritis, American Academy, Anti-VEGF, LUNA, Intraocular pressure, Investment, Clinical trial, Injection, Safety, Drug development, Paratriathlon, AMD, AAO, OPTIC, Retinitis, CST, Vascular occlusion, Annual general meeting, VEGF, Pharmaceutical industry

REDWOOD CITY, Calif., March 18, 2024 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company pioneering the use of gene therapy as a new standard of care for highly prevalent ocular diseases, today reported financial results for the fourth quarter and full year 2023. The company also provided recent pipeline highlights and corporate updates.

Key Points: 
  • The company also provided recent pipeline highlights and corporate updates.
  • 85% and 68% of patients at the 2E11 and 6E10 doses, respectively, received no supplemental injections through 26 weeks.
  • Stock-based compensation expense included in research and development expenses was $1.0 million for the fourth quarter of 2023.
  • Stock-based compensation expense included in general and administrative expenses was $3.1 million for the fourth quarter of 2023.

Adverum Biotechnologies Announces Positive Preliminary Efficacy and Safety Data from LUNA Phase 2 Trial of Ixo-vec in Patients with Wet AMD

Retrieved on: 
torsdag, februari 8, 2024
Chorioretinitis, Injection, Principal, Inflammation, Life, Adverum Biotechnologies, VEGF, AMD, Partner (business rank), Vasculitis, Patient, Safety, Paratriathlon, Prednisone, University of Nevada, Reno, Intraocular pressure, Data, Webcast, University, Retinitis, M.A, Clinical research, CST, Vascular occlusion, Pharmaceutical industry

These data are being presented by Dr. Arshad Khanani today at the 47th Annual Meeting of the Macula Society.

Key Points: 
  • These data are being presented by Dr. Arshad Khanani today at the 47th Annual Meeting of the Macula Society.
  • “Wet AMD is a leading cause of blindness in people over the age of 65, requiring life-long anti-VEGF injections.
  • Our goal with Ixo-vec is to provide patients virtually injection-free management of their wet AMD lasting years and potentially for life,” stated Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies.
  • The LUNA trial builds on our experience with the OPTIC study, where landmark 2-year data was recently published in The Lancet’s eclinicalmedicine.

Autoimmune Disease and Pregnancy: ISB Study Challenges Prevailing Wisdom, Unveils Nuances

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onsdag, januari 31, 2024
Other Health, Research, Consumer, Healthcare Reform, Parenting, Science, Baby, Maternity, COVID-19, Other Science, Health, Diabetes, Public Policy, Government, Multimedia, Multiple sclerosis, Sarcoidosis, ISB, Mother, Health information management, Vasculitis, MD, Preterm birth, Inflammatory bowel disease, Antiphospholipid syndrome, Patient, Rheumatoid arthritis, Spondyloarthropathy, Cohort (statistics), Providence, Lupus, Hypertension, Pregnancy, Autoimmunity, Thought, Risk, Psoriatic arthritis, Psoriasis, Autoimmune disease, Gestational age, Birth control

View the full release here: https://www.businesswire.com/news/home/20240131144327/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20240131144327/en/
    In a just-published study in the journal Lancet eClinical Health , researchers at the Institute for Systems Biology (ISB) and Providence showed nuanced pregnancy outcomes for pregnant individuals with autoimmune disease.
  • Each type of autoimmune disease is different, each person has their own medical history, and risk may change over the course of pregnancy.
  • This study highlights the importance of taking comorbidities into consideration,” said ISB Associate Professor Jennifer Hadlock, MD , who led the study.
  • “These results can help reshape discussions surrounding autoimmune disease and pregnancy, and bring fresh perspective to a traditionally understudied area in research,” Hadlock said.