Open-label trial

Ayala Pharmaceuticals Presents Updated AL102 Results from Phase 2 Clinical Trial in Desmoid Tumors at ESMO Congress 2023

Retrieved on: 
Monday, October 23, 2023
Patient, FDA, Research, Open-label trial, MRI, ESMO, Phase 2 (graffiti artist), OTCQX, DT, Congress, Plantar fibromatosis, Phase 3, Poster, OLE, Clinical trial, The Institute, Part, Safety, Chief Medical Officer (Ireland), European Society for Medical Oncology, Royal Marsden Hospital, GSI, Consultant, BIW, Pharmaceutical industry, Sarcoma

REHOVOT, Israel and MONMOUTH JUNCTION, N.J., Oct. 23, 2023 (GLOBE NEWSWIRE) -- Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, today announced that new data from the RINGSIDE study evaluating its lead investigational candidate AL102 for the treatment of desmoid tumors (DT) are being presented today at the European Society for Molecular Oncology (ESMO) Congress 2023, taking place October 20th to 24th in Madrid, Spain. The data are from Phase 2 (Part A) of the study and from the Open Label Extension (OLE). The results are featured in a poster being presented by Professor Robin Jones, Team Leader in Sarcoma Clinical Trials at The Institute of Cancer Research and Consultant Medical Oncologist at The Royal Marsden, UK.

Key Points: 
  • The data are from Phase 2 (Part A) of the study and from the Open Label Extension (OLE).
  • The results are featured in a poster being presented by Professor Robin Jones, Team Leader in Sarcoma Clinical Trials at The Institute of Cancer Research and Consultant Medical Oncologist at The Royal Marsden, UK.
  • “The RINGSIDE results continue to improve over time, with more patients in this latest Phase 2 and OLE data cut demonstrating responses to treatment with AL102,” said Kenneth Berlin, President and CEO of Ayala.
  • The safety results appear consistent with the GSI class.”
    The ongoing Phase 2/3 RINGSIDE clinical trial is a randomized, global multi-center study evaluating AL102 in patients with progressing desmoid tumors.

Clene Reports Significant Long-Term Survival Improvement From CNM-Au8 Treatment in HEALEY ALS Platform Trial Compared to PRO-ACT Historical Controls

Retrieved on: 
Monday, September 25, 2023
ACT, Database, Medicine, CNM, Survival, Clinical trial, Meta-analysis, Multiple sclerosis, SALT, Ventilation, MS, Open-label trial, Microsoft Access, Patient, Data, Risk, Common-pool resource, Principal, Massachusetts General Hospital, Death, ALS, Safety, OLE, Pharmaceutical industry

The HEALEY ALS Platform Trial is a perpetual multi-center, randomized, double-blind, placebo-controlled clinical trial program designed to evaluate the efficacy and safety of multiple investigational products in people living with ALS.

Key Points: 
  • The HEALEY ALS Platform Trial is a perpetual multi-center, randomized, double-blind, placebo-controlled clinical trial program designed to evaluate the efficacy and safety of multiple investigational products in people living with ALS.
  • Participants received CNM-Au8 in addition to ALS standard-of-care and were randomized to the drug or placebo during the 24-week double-blind period.
  • The PRO-ACT dataset is derived from pooled ALS clinical trial data from 29 completed Phase 2 and Phase 3 ALS clinical trials.
  • This database provides a useful and validated surrogate for survival status of past participants in ALS clinical trials with long-term follow-up.

STRATA Skin Sciences Announces First Patients Treated in TheraClear®X Clinical Study for Acne

Retrieved on: 
Tuesday, May 30, 2023
Male, Skin, Marketing, Safety, Efficacy, Acne, Female, FAAD, Open-label trial, Trial of the century, Dermatology, SSKN, Patient, Gland, Research, Pharmaceutical industry, MD

The study is being conducted at the Ablon Skin Institute and Research Center in Manhattan Beach, California.

Key Points: 
  • The study is being conducted at the Ablon Skin Institute and Research Center in Manhattan Beach, California.
  • Ablon’s clinical study will help further validate the efficacy and safety of TheraClearX as a stand-alone acne treatment therapy,” stated Bob Moccia, Chief Executive Officer of STRATA Skin Sciences.
  • We expect patients that have been seen in the clinic to experience visible reductions in acne lesions.”
    “We are excited to have treated the first six patients in our TheraClearX clinical study,” stated Dr. Glynis Ablon, MD, FAAD.
  • “TheraClearX offers patients a non-pharmaceutical option to help treat patients with mild to moderate acne with no numbing cream necessary, and no pain.

NImmune Biopharma Provides Update on Omilancor Clinical Development Programs in Ulcerative Colitis and Crohn’s Disease

Retrieved on: 
Wednesday, May 17, 2023
Biotechnology, FDA, Other Health, Health, General Health, Pharmaceutical, Biometrics, Other Science, Research, Science, Clinical Trials, Mayo, Myelocyte, Maintenance, FCP, Drug development, TNF, AE, IBD, NDA, A.I, Lupus, Ulcerative colitis, Psoriasis, Open-label trial, LANCL2, US, Acquisition, UC, Patient, Kelly Gebo, New Drug Application, Pharmaceutical industry, Phosphorus

Omilancor is a wholly-owned, once-daily gut-restricted oral first-in-class LANCL2 therapeutic entering Phase 3 clinical testing in UC patients.

Key Points: 
  • Omilancor is a wholly-owned, once-daily gut-restricted oral first-in-class LANCL2 therapeutic entering Phase 3 clinical testing in UC patients.
  • Dr. Josep Bassaganya-Riera, Founder & CEO of NImmune, said, "Following our successful acquisition of the LANCL therapeutic portfolio, we have made meaningful progress in advancing our clinical development programs for omilancor in IBD and NIM-1324 in lupus.
  • Importantly, the omilancor program has taken just over four years since inception to reach Phase 3 initiation, representing significant cost savings compared to traditional drug development timelines.
  • PACIFY II aims to randomize 652 active disease UC patients, with 326 patients receiving 440mg of omilancor and 326 patients receiving placebo.

Athira Pharma Reports Full Year 2022 Financial Results and Recent Pipeline and Business Updates

Retrieved on: 
Thursday, March 23, 2023
ACT, Insurance, HGF, Enzyme inhibitor, Society, Doctor of Philosophy, Athira Pharma, G&A, Open-label trial, St. Kevin's Church, Camden Row, Dublin, Neurotherapeutics, Dementia, Journal, District court, Investment, Administration, Neuropathic pain, Annual general meeting, MET, Lewy body, Growth, Research and development, R, ALS, IND, Douglas v. U.S. District Court ex rel Talk America, Conference, Publishing, Hepatocyte growth factor, Fine chemical, Pharmaceutical industry, Management, Cryptocurrency

BOTHELL, Wash., March 23, 2023 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today announced the company’s financial results for the year ended December 31, 2022, and reviewed recent pipeline and business updates.

Key Points: 
  • BOTHELL, Wash., March 23, 2023 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today announced the company’s financial results for the year ended December 31, 2022, and reviewed recent pipeline and business updates.
  • We are targeting to complete enrollment of the LIFT-AD trial in mid-2023 and to have topline data in early 2024.
  • Fosgonimeton (ATH-1017) - Small molecule designed to enhance the activity of the HGF/MET system with the potential to protect and repair neuronal networks.
  • As of year-end 2022, greater than 85% of participants who have completed either study have elected to enroll in the OLEX trial.

First Wave BioPharma Announces Phase 2 Adrulipase Combination Trial Data to be Presented at the 2023 Digestive Disease Week (DDW) Conference

Retrieved on: 
Tuesday, February 21, 2023
CF, Patient, DDW, Cystic fibrosis, Safety, Steatorrhea, Human, CP, Incidence, Body mass index, Clinical trial, EPI, Translation, Exocrine pancreatic insufficiency, Absorption, Open-label trial, Defecation, PERT, Survival, Combination, PPE, Candace Pert, BOCA, First wave, Conference, SPAN, Case Western Reserve University School of Medicine, Pharmaceutical industry, CFA

The conference is taking place May 6-9, 2023, in Chicago.

Key Points: 
  • The conference is taking place May 6-9, 2023, in Chicago.
  • “The research presented at DDW 2023 is intended to highlight the potential benefits that adrulipase may offer in the treatment of EPI above and beyond PERT, the current standard of care,” stated James Sapirstein, President and CEO of First Wave BioPharma.
  • Adding a synthetic lipase like adrulipase to PERT has demonstrated the potential to improve fat absorption and overall nutrition of CF patients.
  • The digestive standard of care for both CF and chronic pancreatitis (CP) patients with EPI are commercially available PERTs.

Athira Pharma Provides 2023 Pipeline Outlook

Retrieved on: 
Thursday, January 5, 2023
ACT, Dementia, MET, Food, Doctor of Philosophy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Neurodegeneration, Neuropathic pain, Lewy body, Hepatocyte growth factor, ALS, Patient, Therapy, IND, HGF, Athira Pharma, Open-label trial, Pharmaceutical industry, Fine chemical, Health insurance, Risk management, Neurotherapeutics

BOTHELL, Wash., Jan. 05, 2023 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today provided an update on its pipeline development programs and outlook for 2023.

Key Points: 
  • Advancing small molecule therapeutic candidates with clinical and preclinical data suggesting potential neuroprotective, neurotrophic, procognitive and disease-modifying effects
    BOTHELL, Wash., Jan. 05, 2023 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today provided an update on its pipeline development programs and outlook for 2023.
  • This includes our recently published preclinical results in the peer-reviewed journal, Neurotherapeutics, supporting the potential of fosgonimeton,” stated Mark Litton, Ph.D., President and Chief Executive Officer of Athira Pharma.
  • Athira enrolled 28 patients in the exploratory Phase 2 SHAPE study of fosgonimeton in participants with Parkinson’s disease dementia or Dementia with Lewy bodies.
  • ATH-1105 demonstrated consistent improvements across measures of motor function, nerve function, and neurodegeneration in a TDP-43 mouse model of ALS.

Fulcrum Therapeutics to Present New Data from the Open Label Extension of Phase 2 ReDUX4 Study at the World Muscle Society (WMS) Congress in Halifax, Canada

Retrieved on: 
Wednesday, October 12, 2022
Gene expression, LinkedIn, U.S. Securities and Exchange Commission, GSK, Orphan, Fulcrum, Therapy, Ascending limb of loop of Henle, Sickle cell disease, Fetal hemoglobin, Review, Quality, Open-label trial, Thalassemia, WMS, Neuro, Muscle weakness, Facial expression, Patient, Facioscapulohumeral muscular dystrophy, Halifax Convention Centre, DUX4, OLE, Clinical trial, MFI, Twitter, HIV disease progression rates, Security (finance), Collection, Nasdaq, Private Securities Litigation Reform Act, Hemoglobinopathy, Șaeș, PGIC, Quality of life, Fast track (FDA), Muscular dystrophy, REACH, Safety, EDT, GLOBE, RWS, MAPK, FDA, COVID-19, Pharmaceutical industry, FSHD, Industry, Losmapimod

CAMBRIDGE, Mass., Oct. 12, 2022 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced today that new data from the open label extension (OLE) portion of the Phase 2 ReDUX4 study of losmapimod for the treatment of FSHD will be featured in a poster presentation at the World Muscle Society (WMS) Hybrid Congress taking place October 11-15 in Halifax, Canada. Fulcrum will also host a symposium entitled “Measuring Progression in FSHD: Implications for Clinical Trials,” featuring an overview of the OLE data on October 12, 2022, at the Halifax Convention Center.

Key Points: 
  • ReDUX4 was a 48-week, placebo-controlled study that enrolled 80 participants between the ages of 18-65 with genetically confirmed FSHD1 (Ricci score 2-4).
  • Of the 77 participants who completed the initial 48-week study, 99% (n=76) enrolled in the OLE.
  • Data from the OLE support findings that losmapimod modifies FSHD disease progression and preserves or improves muscle function.
  • Following the completion of the trial, 99% of eligible participants elected to continue in the Open Label Extension trial.

Coya Therapeutics, Inc. Announces Peer-Reviewed Publication of Phase 2a Clinical Trial Data for COYA 101 in Amyotrophic Lateral Sclerosis (ALS)

Retrieved on: 
Thursday, September 1, 2022
Amyotrophic lateral sclerosis, Inflammation, Neuroinflammation, Patient, COVID-19, HIV disease progression rates, Houston Methodist Hospital, Safety, Therapy, Protein, DB, Disease, ITT, Open-label trial, Phenotype, COYA, Particle-size distribution, OLR1, Disability, GLOBE, FTD, Exosome, Neuro, Coronavirus Scientific Advisory Board (Turkey), Biomarker, Oxidative stress, ALS, OLE, Medical device, Pharmaceutical industry, Person, Regulatory T cell, Regulatory T cell, Neuroimmunology, Scleroderma, Fibrosis

Coya has an exclusive worldwide license from Houston Methodist Hospital to develop, manufacture, and commercialize COYA 101.

Key Points: 
  • Coya has an exclusive worldwide license from Houston Methodist Hospital to develop, manufacture, and commercialize COYA 101.
  • Subjects enrolled in the Phase 2a trial were initially randomized into a double-blind portion to receive active Treg cells or matching placebo every four weeks over 24 weeks.
  • The DB portion of the trial was curtailed due to COVID-19-related restrictions, and the limited number of patients enrolled made statistical analysis not feasible.
  • The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) is a validated rating instrument that gauges the progression of disability in patients with amyotrophic lateral sclerosis (ALS) on a 48-point scale.

Cytokinetics Reports First Quarter 2022 Financial Results

Retrieved on: 
Wednesday, May 4, 2022
Buildout, Investment, American College, Efficacy, European Heart Journal, Disopyramide, GLOBE, NDA, Heart failure, Safety, Food, Open-label trial, Blood, Clinical study design, Patient, Royalty Pharma, Heart, FDA, Cohort, Rationale, New Drug Application, CPET, Manuscript, Cardiology, Lists of scientists, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Atrial fibrillation, Cytokinetics, PDUFA, Risk, Exercise, Medical device, Pharmaceutical industry, HCM

SOUTH SAN FRANCISCO, Calif., May 04, 2022 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the first quarter of 2022. Net loss for the first quarter was $89.4 million, or $1.05 per share, compared to net loss for the first quarter of 2021 of $47.1 million, or $0.66 per share. Cash, cash equivalents and investments totaled $686.1 million at March 31, 2022.

Key Points: 
  • Initial Data from REDWOOD-HCM OLE, the Open Label Extension Study of Aficamten, to be Presented at Heart Failure 2022 on May 23, 2022
    Continued Launch Readiness Activities and Buildout of Commercial Infrastructure in Advance of PDUFA Date of November 30, 2022 for Omecamtiv Mecarbil
    SOUTH SAN FRANCISCO, Calif., May 04, 2022 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the first quarter of 2022.
  • Revenues for the first quarter 2022 were $1.1 million compared to $6.5 million for the corresponding period in 2021.
  • Members of Cytokinetics senior management team will review the companys first quarter results on a conference call today at 4:30 PM Eastern Time.
  • CYTOKINETICS and the CYTOKINETICS and C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries.