Placebo-controlled study

Timber Pharmaceuticals Provides Business Update and Announces Third Quarter 2021 Financial Results

Retrieved on: 
Monday, November 15, 2021
Securities Exchange Act of 1934, Genta (company), Safety, Private Securities Litigation Reform Act, Industry, Placebo-controlled study, TSC, AFT, Security (finance), Standard of care, Securities Act of 1933, CI, Annual report, Fas, Â, U.S. Securities and Exchange Commission, Company, GLOBE, Ichthyosis, Isotretinoin, Chemistry, NYSE, Nature, Program, Patient, FDA, CMC, Intellectual property, Pharmaceutical industry

BASKING RIDGE, NJ, Nov. 15, 2021 (GLOBE NEWSWIRE) -- via NewMediaWire -- Timber Pharmaceuticals, Inc. ("Timber" or the “Company”) (NYSE American: TMBR), a biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases, today provided a business update and announced financial results for the third quarter ended September 30, 2021.

Key Points: 
  • BASKING RIDGE, NJ, Nov. 15, 2021 (GLOBE NEWSWIRE) -- via NewMediaWire -- Timber Pharmaceuticals, Inc. ("Timber" or the Company) (NYSE American: TMBR), a biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases, today provided a business update and announced financial results for the third quarter ended September 30, 2021.
  • Timber completedthe Phase 2b CONTROL Study of congenital ichthyosis (CI) for its lead asset, TMB-001, during the third quarter and announced top line results subsequent to quarter end.
  • For Timbers complete financial results for the period ended September 30, 2021, see the Companys quarterly Form 10-Q filed with the Securities and Exchange Commission on November15, 2021.
  • Timber Pharmaceuticals, Inc. is a biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases.

Denali Therapeutics Announces Upcoming Presentations on EIF2B Activator DNL343 and RIPK1 Inhibitor SAR443820/DNL788 at the 2021 Annual Northeast Amyotrophic Lateral Sclerosis (NEALS) Meeting

Retrieved on: 
Wednesday, September 29, 2021
CNS, Safety, Neurodegeneration, Pharmacokinetics, PTV, EIF2B, Integrated stress response, PHASE 2, Security (finance), Private Securities Litigation Reform Act, NASDAQ, U.S. Securities and Exchange Commission, Amyotrophic lateral sclerosis, Pathology, Traffic barrier, Biomarker, AD, Prisoner transport vehicle, GLOBE, MS, Efficacy, Risk, COVID-19, Placebo-controlled study, ClinicalTrials.gov, ALS, RIPK1, Forward-looking statement, Randomness, Sanofi, FTD, Frontotemporal dementia, BBB, Multiple sclerosis, SEC, Neuron, Pharmaceutical industry

EIF2B is an intracellular protein complex that regulates protein synthesis and is required for neuronal health and function.

Key Points: 
  • EIF2B is an intracellular protein complex that regulates protein synthesis and is required for neuronal health and function.
  • DNL343 is designed to activate EIF2B and thereby restore protein synthesis, disperse TDP-43 aggregates, and improve neuronal survival.
  • Denali and Sanofi entered into a broad collaboration in October 2018 for the global development and commercialization of RIPK1 inhibitors.
  • During the webinar, Denali will review the NEALS presentations related to its investigational small molecule therapeutics DNL343 and SAR443820.

Longeveron Announces Multiple Presentations at the 11th Annual International Conference on Frailty & Sarcopenia Research

Retrieved on: 
Tuesday, September 28, 2021
Microsoft, Patent, Goal, Safety, Bone marrow, Investor, Ageing, NASDAQ, Intention, Conference, HLHS, Patient, Disability, Adult, Intussusception (medical disorder), Hypoplastic left heart syndrome, Company, Terminology, Efficacy, HERA, Placebo-controlled study, Metabolic syndrome, Cell, Randomness, Forward-looking statement, SEC, GLOBE, Disorder, Pharmaceutical industry, Vaccine

A live panel with Dr. Ruiz and Longeveron management will follow the presentation.

Key Points: 
  • A live panel with Dr. Ruiz and Longeveron management will follow the presentation.
  • Those interested in registering for the conference can find more information at the conference website: https://frailty-sarcopenia.com/ .
  • All three presentations will be posted to Longeverons website after the conference under Events & Presentations in the Investors section of the Companys website at investors.longeveron.com .
  • Longeveron is a clinical stage biotechnology company developing cellular therapies for specific aging-related and life-threatening conditions.

Aimmune’s PALISADE-ARC004 Longitudinal Study Showed PALFORZIA® Safety and Efficacy Increased Over Time in Patients with Peanut Allergy

Retrieved on: 
Wednesday, August 4, 2021
Biotechnology, Pharmaceutical, Health, Clinical Trials, Food allergies, Medical specialties, Arachis, Medicine, Food and drink, Peanut allergy, Peanut allergen powder, Peanut, Placebo-controlled study, Kari Nadeau, Palifermin, PALISADE-ARC004, Aimmune, Nestlé Health Science, PALISADE-ARC004, AIMMUNE, NESTLé HEALTH SCIENCE

The manuscript, titled Open-label follow-on study evaluating the efficacy, safety, and quality of life with extended daily oral immunotherapy in children with peanut allergy, was published online.

Key Points: 
  • The manuscript, titled Open-label follow-on study evaluating the efficacy, safety, and quality of life with extended daily oral immunotherapy in children with peanut allergy, was published online.
  • Patients who completed the PALISADE trial were eligible to enter the ARC004 open-label follow-on study, which evaluated the long-term efficacy and safety of daily PALFORZIA dosing beyond one year (52 weeks).
  • After two years of daily PALFORZIA treatment, 80.8% of study participants tolerated 2000 mg of peanut protein in the double-blind, placebo-controlled food challenge.
  • PALISADE (Peanut Allergy oral Immunotherapy Study of AR101 for Desensitization) was an international, randomized (3:1), double-blind, placebo-controlled, phase 3 trial to evaluate the efficacy and safety of AR101 in patients with peanut allergy.

SciSparc Announces Completion of Corporate Rebranding and Launch of New Website

Retrieved on: 
Wednesday, August 4, 2021
Clinical research, Design of experiments, Health sciences, Health, Medical research, Clinical trial, Drug discovery, Pharmaceutical industry, Placebo-controlled study, Draft:PPP001, Tcelna

The Company recently provided an update on its Phase IIb clinical study in Tourette Syndrome using the proprietary cannabinoid-based treatment, SCI-110.

Key Points: 
  • The Company recently provided an update on its Phase IIb clinical study in Tourette Syndrome using the proprietary cannabinoid-based treatment, SCI-110.
  • SciSparc intends to evaluate the efficacy, safety and tolerability of its SCI-110 in a randomized, double-blind, placebo controlled, cross-over study.
  • "We believe this rebranding and new website reflect SciSparc's vision and dedication to the development of cannabinoid-based treatments."
  • SciSparc Ltd. is a specialty clinical-stage pharmaceutical company led by an experienced team of senior executives and scientists.

Cytokinetics Announces Start of COURAGE-ALS, a Phase 3 Clinical Trial of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis

Retrieved on: 
Monday, August 2, 2021
Health sciences, Clinical research, Design of experiments, Health, Medical research, Cytokinetics, Clinical trial, Placebo-controlled study

SOUTH SAN FRANCISCO, Calif., Aug. 02, 2021 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that COURAGE-ALS (Clinical Outcomes Using Reldesemtiv on ALSFRS-R in a Global Evaluation in ALS), a Phase 3 clinical trial of reldesemtiv in patients with amyotrophic lateral sclerosis (ALS), is open to enrollment. Reldesemtiv, a next-generation fast skeletal muscle troponin activator (FSTA) arising from Cytokinetics’ skeletal muscle contractility program, slows the rate of calcium release from the regulatory troponin complex of fast skeletal muscle fibers, which sensitizes the sarcomere to calcium, leading to an increase in skeletal muscle contractility. COURAGE-ALS follows FORTITUDE-ALS, a Phase 2 clinical trial of reldesemtiv that demonstrated encouraging results supportive of progression to a pivotal Phase 3 clinical trial.

Key Points: 
  • COURAGE-ALS follows FORTITUDE-ALS, a Phase 2 clinical trial of reldesemtiv that demonstrated encouraging results supportive of progression to a pivotal Phase 3 clinical trial.
  • COURAGE-ALS, a Phase 3, multi-center, double-blind, randomized, placebo-controlled trial of reldesemtiv is expected to enroll approximately 555 patients with ALS.
  • Cytokinetics expects to start a Phase 3 clinical trial of CK-274 in patients with obstructive HCM by year end.
  • The company is conducting COURAGE-ALS, a Phase 3 clinical trial of reldesemtiv in patients with ALS.

MediWound Announces Positive Outcome of Interim Assessment for its EscharEx U.S. Phase 2 Adaptive Design Study

Retrieved on: 
Wednesday, July 28, 2021
Design of experiments, Clinical trials, Health sciences, Science, Health, Clinical research, Medical statistics, Drugs, Clinical study design, Adaptive design, Placebo-controlled study, COVID-19 vaccine

YAVNE, Israel, July 28, 2021 (GLOBE NEWSWIRE) -- MediWound Ltd. (Nasdaq: MDWD), a fully-integrated biopharmaceutical company focused on next-generation biotherapeutic solutions for tissue repair and regeneration, today announced a positive outcome from a planned interim sample size re-estimation of its ongoing EscharEx® U.S. phase 2 adaptive design study for the treatment of venous leg ulcers (VLUs), designed to assess the safety and efficacy of EscharEx compared to gel vehicle (placebo control) and non-surgical standard-of-care (either enzymatic or autolytic debridement).

Key Points: 
  • "We are very pleased with the IDMC's recommendation to continue the EscharEx study as originally planned without modifying the study sample size, said Sharon Malka, Chief Executive Officer of MediWound.
  • The multicenter, prospective, randomized, placebo-controlled, adaptive design study, evaluating the safety and efficacy of EscharEx in debridement of VLUs.
  • The study includes a pre-defined interim assessment for futility and potential sample size adjustment.
  • EscharEx is an investigational product, currently under a U.S. phase 2 adaptive design study.

SciSparc Announces Updates Regarding its Phase IIb Study in Tourette Syndrome

Retrieved on: 
Monday, July 26, 2021
Health sciences, Health, Clinical research, Design of experiments, Medical research, Psychiatric diagnosis, Tourette syndrome, Clinical trial, Placebo-controlled study

TEL AVIV, Israel, July 26, 2021 /PRNewswire/ -- SciSparc Ltd. (OTCQB: SPRCY), a specialty, clinical-stage pharmaceutical company focusing on the development of cannabinoid-based treatments, today announced a number of updates regarding its Phase IIb clinical study in Tourette Syndrome using the proprietary cannabinoid-based treatment- SCI-110.

Key Points: 
  • TEL AVIV, Israel, July 26, 2021 /PRNewswire/ -- SciSparc Ltd. (OTCQB: SPRCY), a specialty, clinical-stage pharmaceutical company focusing on the development of cannabinoid-based treatments, today announced a number of updates regarding its Phase IIb clinical study in Tourette Syndrome using the proprietary cannabinoid-based treatment- SCI-110.
  • The company is currently in contact with two clinical sites that will potentially host and conduct the clinical study.
  • Tourette Syndrome (TS) is a movement and neurobehavioral disorder characterized by motor and vocal chronic tics with onset before age 18.
  • SciSparc intends to evaluate the efficacy, safety and tolerability of its SCI-110 in a randomized, double-blind, placebo controlled, cross-over study.

Tonix Pharmaceuticals Announces Outcome of Interim Analysis of Phase 3 RALLY Study of TNX-102 SL for the Management of Fibromyalgia

Retrieved on: 
Friday, July 23, 2021
Clinical research, Design of experiments, Knowledge, Health sciences, Research, Clinical trials, Tonix Pharmaceuticals, Randomized controlled trial, Placebo-controlled study, Placebo

CHATHAM, N.J., July 23, 2021 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, announced today that the Company has decided to stop enrollment in the Phase 3 RALLY study of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) 5.6 mg for the management of fibromyalgia following an unblinded, pre-planned interim analysis by the Independent Data Monitoring Committee (IDMC) of the RALLY study. Based on interim analysis results of the first 50% (n=337) enrolled participants, the IDMC recommended stopping the trial for futility as TNX-102 SL is unlikely to demonstrate a statistically significant improvement in the primary endpoint of overall change from baseline in daily diary pain severity scores between those treated with TNX-102 SL 5.6 mg (2x 2.8 mg tablets) and those receiving placebo. Tonix remains blinded to the detailed interim analysis results and only received the recommendation made by the IDMC. Preliminary blinded safety data from these participants did not reveal any new safety signals, and the decision to discontinue enrolling new participants is not related to safety. The Company intends to continue studying those participants currently enrolled until completion and then proceed with a full analysis of the unblinded data, with the topline results expected to be reported in the fourth quarter of 2021, to determine the next steps in this program.

Key Points: 
  • Tonix remains blinded to the detailed interim analysis results and only received the recommendation made by the IDMC.
  • We are surprised and disappointed that the interim analysis did not support continued enrollment in this Phase 3 RALLY study, especially considering the previous Phase 3 RELIEF study, which had a similar design and achieved statistical significance on the primary endpoint.
  • We thank the patients, caregivers and investigators who participated in the RALLY study.
  • The RALLY study is a double-blind, randomized, placebo-controlled trial designed to evaluate the efficacy and safety of TNX-102 SL (cyclobenzaprine HCl sublingual tablets).

Organicell Announces FDA Approval Of IND Application For The Use Of Zofin™ In COVID-19 Long Haulers

Retrieved on: 
Wednesday, July 21, 2021
COVID-19, Clinical research, Clinical trials, Long Covid, Health, Placebo-controlled study, Cough, Articles

This approved trial design consists of a double blinded, placebo-controlled, randomized phase I/II trial designed to investigate the safety and potential efficacy of Zofin in treating COVID-19 long haulers.

Key Points: 
  • This approved trial design consists of a double blinded, placebo-controlled, randomized phase I/II trial designed to investigate the safety and potential efficacy of Zofin in treating COVID-19 long haulers.
  • These patients, called COVID-19 "long haulers," report lingering shortness of breath, cough, fatigue, and mental fog for months after recovery, impairing their return to work and everyday life.
  • Even by the lowest health care industry estimates, the magnitude of this secondary health crisis of COVID-19 long haulers will result in millions of affected individuals.
  • Organicell has no intention and specifically disclaims any duty to update the information in this press release.