Sequela

FDA grants Medivir´s MIV-711 Rare Pediatric Disease Designation and Orphan Drug Designation for the treatment of Legg-Calvé-Perthes Disease

Retrieved on: 
Thursday, April 25, 2024
Leg, FDA, Cancer, Hip, Medivir, Sequela, Priority review, Risk, Osteoclast, Osteoarthritis, Partnership, Depression, Disease, Obesity, Patient, Cathepsin K, ODD, Ageing, Pharmaceutical industry

The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years of age.

Key Points: 
  • The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years of age.
  • Pediatric diseases recognized as "rare", affect fewer than 200,000 people in the United States.
  • We are delighted that MIV-711 has been granted RPDD by the FDA with the potential to become the first approved treatment option.
  • To gain RPDD, there must be supportive data suggesting that the drug may be effective in the disease.

Inari Medical Announces Interim Two-Year Results from the ClotTriever CLOUT Registry

Retrieved on: 
Tuesday, March 12, 2024
Deep vein thrombosis, Pulmonary embolism, VTE, Incidence, Sequela, CAVA, Patient, Post-thrombotic syndrome, Research, PTS, DVT, Randomized controlled trial, Safety, FACS, American Venous Forum, Data, Pharmaceutical industry

IRVINE, Calif., March 12, 2024 (GLOBE NEWSWIRE) -- Inari Medical, Inc. (NASDAQ: NARI) (“Inari”) a medical device company focused on developing products to treat and transform the lives of patients suffering from venous and other diseases, announced positive two-year interim results from the CLOUT registry.

Key Points: 
  • IRVINE, Calif., March 12, 2024 (GLOBE NEWSWIRE) -- Inari Medical, Inc. (NASDAQ: NARI) (“Inari”) a medical device company focused on developing products to treat and transform the lives of patients suffering from venous and other diseases, announced positive two-year interim results from the CLOUT registry.
  • The interim results are the largest prospective, multi-center two-year dataset in deep vein thrombosis (DVT) since the ATTRACT trial and confirm the excellent safety, effectiveness, and long-term outcomes of the ClotTriever system in real-world DVT patients.
  • Patients had low incidence of independently adjudicated safety events related to rethrombosis, with only 5.0% and 8.4% at 30-days and 6 months, respectively.
  • We remain committed to establishing the ClotTriever and FlowTriever systems as standard of care for deep vein thrombosis and pulmonary embolism (PE) patients.”

Cellectar Biosciences Announces Positive Topline Data Achieving Primary Endpoint in Pivotal Clinical Study of Iopofosine I 131 in Waldenstrom’s Macroglobulinemia

Retrieved on: 
Monday, January 8, 2024
Sequela, Quality of life, Febrile neutropenia, WM, Death, Anemia, International, IWMF, Disease, Neutropenia, NDA, WAM, News, Cancer, Thrombocytopenia, SCR, Macroglobulinemia, MCI, MRR, Mayo Clinic, Mitt, Patient, Safety, Medicine, Cytopenia, Immunoglobulin M, Pharmaceutical industry

The CLOVER WaM study met its primary endpoint with a major response rate (MRR) of 61% (95% confidence interval [44.50%, 75.80%, two-sided p value

Key Points: 
  • The CLOVER WaM study met its primary endpoint with a major response rate (MRR) of 61% (95% confidence interval [44.50%, 75.80%, two-sided p value
  • Notably, iopofosine monotherapy achieved an 8% stringent complete remission (sCR) in this highly refractory WM population.
  • “The results from this pivotal study utilizing just four doses of iopofosine monotherapy in heavily pretreated patients are very compelling, demonstrating deep and durable remissions.
  • “Iopofosine’s high major response rate and achievement of the study’s primary endpoint in highly refractory, Waldenstrom’s macroglobulinemia patients exhibits its potentially practice-changing clinical profile.

atai Life Sciences Reports Second Quarter 2023 Financial Results and Operational Highlights, and Announces Clinical Data from the Phase 1 Study of DMX-1002 (Ibogaine)

Retrieved on: 
Thursday, August 10, 2023
Consensus, Psilocybin, Sequela, PK, Partnership, MCCB, PD, CPT, CSO, OTF, Perception, Continuity, Anorexia nervosa, QT interval, Aes, Pharmacokinetics, Neuroscience, OUD, Cmax, MOAA, Language, QT, American Medical Association, Plasma, Cardiac electrophysiology, Patient, Investment, Current Procedural Terminology, Risk, Administration, AUC, Pivotal, Imperial College London, Entrepreneurship, Research and development, Cardiology, Research, SQ, R, TRD, Arrhythmia, III, Schizophrenia, Cognitive deficit, PTSD, Safety, Disease, Nursing, Pharmaceutical industry, Vaccine, Palladium, Medical device, Surveying, Cryptocurrency, Ibogaine

“Looking ahead to the second half of 2023 and beyond, we believe we are well positioned to continue advancing our key clinical programs.

Key Points: 
  • “Looking ahead to the second half of 2023 and beyond, we believe we are well positioned to continue advancing our key clinical programs.
  • Drug-induced prolongation of the QT interval is a phenomenon that has been, with many but not all drugs, associated with cardiac arrhythmias.
  • The single-blinded Phase 1 study assessed the safety, tolerability and pharmacokinetics of single-ascending doses of DMX-1002 in healthy volunteers.
  • Enrollment has been recently completed and the company expects to report initial clinical data in Q4 2023.

Novartis presents new five-year data on disability outcomes and safety of Kesimpta® (ofatumumab) in people living with relapsing multiple sclerosis

Retrieved on: 
Thursday, April 20, 2023
Sequela, Sclerosis, Cleveland Clinic, Disability, AAN, Infection, Overalls, COVID-19, Aes, Immunoglobulin M, Urinary tract infection, American Academy, CDW, Multiple sclerosis, Barrow Neurological Institute, MS, RMS, Novartis, Patient, Risk, Common, Immunoglobulin G, Septic, American Academy of Neurology, Pneumonia, LLN, Safety, LGG, Disease, Pharmaceutical industry, MD, Ofatumumab

A separate analysis showed that treatment with Kesimpta for up to five years was well-tolerated, with no new or increased safety risks identified2.

Key Points: 
  • A separate analysis showed that treatment with Kesimpta for up to five years was well-tolerated, with no new or increased safety risks identified2.
  • These data will be presented at the American Academy of Neurology (AAN) Annual Meeting held in Boston and virtually on April 22-27, 2023.
  • Along with the five-year safety analysis, these data support this treatment as a well-tolerated, efficacious treatment option for people living with relapsing multiple sclerosis."
  • "Novartis remains committed to the multiple sclerosis community in our continued study of Kesimpta and to supporting those living with MS and their families throughout their journey."

HR Positive/ Her2 Negative Breast Cancer - Pipeline Insight, 2023 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, March 22, 2023
Health, Oncology, CISH, Sequela, Triple-negative breast cancer, FISH, Therapy, Cleavage, HER2, CDK, Bone metastasis, Survival, Clinical trial, Route of administration, Quality of life, Lead, Woman, ERBB2, AstraZeneca, Rated R, IHC, II, Sapporo Medical University, Combination therapy, Route, Daiichi Sankyo, Administration, US, Discovery, ET, Patient, Breast cancer, Risk, DXD, Immunoglobulin G, HR, In situ hybridization, Immunohistochemistry, ADC, Growth, Mortality, Spacecraft, Lymph node, Recurrence, News, III, SERD, DFS, Birth control, Medical imaging, Pharmaceutical industry, Dietary supplement, Fine chemical

This "HR Positive/ HER2 Negative Breast Cancer - Pipeline Insight, 2023" report provides comprehensive insights about 50+ companies and 53+ pipeline drugs in HR Positive/ HER2 Negative Breast Cancer pipeline landscape.

Key Points: 
  • This "HR Positive/ HER2 Negative Breast Cancer - Pipeline Insight, 2023" report provides comprehensive insights about 50+ companies and 53+ pipeline drugs in HR Positive/ HER2 Negative Breast Cancer pipeline landscape.
  • Depending on the levels of HER2 or ERBB2 in the laboratory test breast cancer is classified as HER2 or ERBB2 positive or HER2 negative breast cancer.
  • A detailed picture of the HR Positive/ HER2 Negative Breast Cancer pipeline landscape is provided which includes the disease overview and HR Positive/ HER2 Negative Breast Cancer treatment guidelines.
  • HR Positive/ HER2 Negative Breast Cancer pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration.

GenSight Biologics Announces 1 Year Safety Data and Efficacy Signals from PIONEER Phase I/II Clinical Trial of GS030, an Optogenetic Treatment Candidate for Retinitis Pigmentosa

Retrieved on: 
Monday, February 13, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, DSMB, Retinitis pigmentosa, Aes, Safety, Patient, Entrepreneurship, Eye, Inflammation, ISIN, RP, Sequela, VG, Data monitoring committee, Genetic disorder, Pharmaceutical industry, EU, Euronext, MD

PIONEER is a first-in-human, multi-center, open-label dose escalation clinical trial evaluating the safety and tolerability of GS030, an optogenetic treatment candidate combining an AAV2-based gene therapy (GS030-DP) with the use of light-stimulating goggles (GS030-MD) in patients with end-stage RP.

Key Points: 
  • PIONEER is a first-in-human, multi-center, open-label dose escalation clinical trial evaluating the safety and tolerability of GS030, an optogenetic treatment candidate combining an AAV2-based gene therapy (GS030-DP) with the use of light-stimulating goggles (GS030-MD) in patients with end-stage RP.
  • A Data Safety Monitoring Board (DSMB) reviewed the safety data of all treated subjects in each cohort and made recommendations before the extension cohort was enrolled.
  • The safety data and early efficacy signals from our PIONEER trial are highly encouraging and suggest that our optogenetic treatment candidate could offer hope to the many patients affected," said Bernard Gilly, Co-Founder and Chief Executive Officer of GenSight Biologics. "
  • We are looking forward to further data from the extension cohort that is under recruitment and expect then to move GS030 to efficacy trials."

Positive Data Presentations at AAO Annual Meeting Demonstrate Utility and Versatility of Clearside Biomedical’s Proprietary Suprachoroidal Space Platform

Retrieved on: 
Tuesday, October 4, 2022
Association, Injection, Neutralizing antibody, CLS, AES, SCS, Eye, Sequela, Therapy, Axitinib, CRT, Visual acuity, NASDAQ, Uveitis, Neoplasm, Intraocular pressure, American Academy, Triamcinolone, Severe cognitive impairment, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Clearside, Macular edema, Surgeon, Intravitreal injection, AAO, Inflammation, Ranibizumab, Adult, Food, MBA, Highway Rider, Treatment, GLOBE, Incidence, Șaeș, Episcleritis, Retina, Conjunctivitis, Choroid, Safety, NYSE, Pharmaceutical industry, Medical device, Medical imaging, Vaccine, Cohort, Patient

ALPHARETTA, Ga., Oct. 04, 2022 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (NASDAQ:CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), announced today that clinical data from multiple internal and partnered programs were presented at the recent 2022 Annual Meeting of the American Academy of Ophthalmology (AAO), the world's largest association of eye physicians and surgeons.

Key Points: 
  • The interest of the medical community in delivering drugs to the suprachoroidal space is extraordinary.
  • We look forward to additional near-term data being generated and reported across all of these clinical-stage programs, Dr. Ciulla concluded.
  • Cohorts 5 and 6 received up to three cycles of therapy, which was considered the therapeutic regimen for evaluation.
  • Clearside Biomedical, Inc. is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS).

Arix Portfolio Company Aura Biosciences Presents Interim Phase 2 Data Highlighting Belzupacap Sarotalocan (AU-011) Suprachoroidal Efficacy and Safety at AAO 2022

Retrieved on: 
Monday, October 3, 2022
Annual general meeting, VDC, Clinical trial, SC, Sequela, Visual acuity, Cancer, NASDAQ, Neoplasm, American Academy of Ophthalmology, Immune system, American Academy, Severe cognitive impairment, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Patient, AAO, Adult, Radiation, Associate, Food, Twitter, MBA, Acral lentiginous melanoma, Highway Rider, ID, Interim, Company, Ear, LinkedIn, Șaeș, List of life sciences, Industry, MD, Cohort, Harvard Medical School, Aura, Safety, NMIBC, Pharmaceutical industry, Ophthalmology

As a listed company, we are able to bring this exciting growth phase of our industry to a broader range of investors.

Key Points: 
  • As a listed company, we are able to bring this exciting growth phase of our industry to a broader range of investors.
  • Preliminary analysis of the data from the Phase 2 trial using suprachoroidal administration supports tolerability up to three cycles of therapy and shows a dose-dependent anti-tumor response.
  • The overall safety profile of belzupacap sarotalocan was generally favorable, with no dose-limiting toxicities or treatment-related SAEs reported as of August 19, 2022.
  • In addition, leveraging Auras technology platform, Aura is developing belzupacap sarotalocan more broadly across multiple cancers, including in patients with non-muscle invasive bladder cancer (NMIBC).

Aura Biosciences Announces Interim Phase 2 Data Evaluating Suprachoroidal Administration of Belzupacap Sarotalocan (AU-011) for the First-Line Treatment of Patients with Early-Stage Choroidal Melanoma Presented at AAO 2022

Retrieved on: 
Monday, October 3, 2022
Research, FDA, Clinical Trials, Biotechnology, Pharmaceutical, Health, Optical, Science, Oncology, Other Science, U.S. Securities and Exchange Commission, Annual general meeting, Review, VDC, Clinical trial, Private Securities Litigation Reform Act, SC, COVID-19, Annual report, Goal, Sequela, Visual acuity, Cancer, NASDAQ, Neoplasm, American Academy of Ophthalmology, Immune system, American Academy, Severe cognitive impairment, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Quality of life, Patient, Intention, AAO, Adult, SEC, Radiation, Associate, Food, Twitter, MBA, Acral lentiginous melanoma, Highway Rider, ID, Interim, Risk, Company, Ear, LinkedIn, Șaeș, MD, Cohort, Harvard Medical School, Aura, Safety, Compte rendu, NMIBC, Pharmaceutical industry, Ophthalmology

The Phase 2 interim safety and efficacy data that was presented at AAO is very encouraging for patients with primary choroidal melanoma, as the majority of patients are diagnosed with early-stage disease and have no vision-preserving treatment options.

Key Points: 
  • The Phase 2 interim safety and efficacy data that was presented at AAO is very encouraging for patients with primary choroidal melanoma, as the majority of patients are diagnosed with early-stage disease and have no vision-preserving treatment options.
  • Preliminary analysis of the data from the Phase 2 trial using suprachoroidal administration supports tolerability up to three cycles of therapy and shows a dose-dependent anti-tumor response.
  • We believe that the data to date provides proof of concept for an additional intraocular route of administration and further supports belzupacap sarotalocans target product profile.
  • Aura plans to pursue development of belzupacap sarotalocan across its ocular oncology franchise including for the treatment of patients with choroidal metastasis.