Drug Discovery World

NOVEMBER 23, 2023

Minoryx has announced enrollment of first patients with cerebral Adrenoleukodystrophy (cALD) in the US Phase III clinical trial, CALYX. This will push the Phase III CALYX trial forward and bring leriglitazone to patients as quickly as possible,” said Sílvia Pascual, VP Clinical Development, Minoryx. “In

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Drug Discovery World

JULY 21, 2023

Madrigal Pharmaceuticals has submitted an application for resmetirom, a drug for nonalcoholic steatohepatitis (NASH) with liver fibrosis, to the US Food and Drug Administration (FDA) and has requested a priority review. The post First drug for NASH with liver fibrosis submitted to FDA appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MAY 15, 2023

The US Food and Drug Administration (FDA) has given the go-ahead to CytoAgents to initiate a US Phase Ib/IIa clinical trial investigating CTO1681 for cytokine release syndrome (CRS) in lymphoma patients receiving CAR-T cell therapy.

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Drug Discovery World

JUNE 16, 2023

The Phase Ia trial will assess the safety and tolerability of escalating doses of LP-184 to determine the maximum tolerated dose (MTD) in patients with advanced pancreatic cancer, recurrent high-grade gliomas/glioblastoma (GBM), metastatic brain and CNS cancers, and other solid tumours with DNA damage response (DDR) deficiencies.

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Drug Discovery World

JULY 12, 2024

The US Food and Drug Administration (FDA) has granted Fast Track Designation for Asklepios BioPharmaceutical’s (AskBio) AB-1005 which is being developed for moderate Parkinson’s disease. AB-1005 is an investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy.

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Drug Discovery World

AUGUST 22, 2023

Bloom Science has announced positive topline data from a Phase I clinical trial of BL-001 , a potential first-in-class therapeutic the company is developing for Dravet syndrome and amyotrophic lateral sclerosis (ALS). BL-001 shows promise as a novel treatment option that can change lives.

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Drug Discovery World

JUNE 21, 2023

The company has stated its intention to schedule an end of Phase II meeting with the US Food and Drug Administration (FDA) to begin planning the next steps in the regulatory and clinical development programme for CITI-002. For the trial, Citius developed a PRO instrument to assess the efficacy of treatments in this disease.

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Drug Discovery World

MARCH 24, 2023

This synergy was demonstrated by the AIPAC Phase IIb trial’s encouraging efficacy and safety, including a over 2.9-month Regulatory approval The company and the US Food and Drug Administration (FDA) agreed to the integrated Phase II/III trial design for AIPAC-003 that will help inform a Biologics License Application (BLA).

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Drug Discovery World

SEPTEMBER 20, 2022

SKYSONA has become the first FDA approved therapy to slow the progression of neurologic dysfunction in boys with cerebral adrenoleukodystrophy (CALD). As a condition of the SKYSONA Accelerated Approval, the manufacturer will provide confirmatory long-term clinical data to the FDA.

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Drug Discovery World

MARCH 30, 2023

Likang Life Sciences has been granted implied approval by China’s National Medical Products Administration (NMPA) for the clinical trial of its innovative personalised neoantigen-targeted vaccine LK101 Injection for advanced solid tumours. Hence, there is a need to increase clinical investigations in this promising area.”

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Drug Discovery World

DECEMBER 22, 2023

Neuroscience drug discovery company Sapient Therapeutics successfully completed Part B of the Phase I trial comparing its CBD therapy SAP021 with Epidiolex. When SAP021 was given with food the data were in alignment with both our expectations from Part A of the study and with previously published trial results.

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Alta Sciences

JULY 22, 2024

Insights in FIH studies provide valuable information on potential dosing, efficacy, and safety, and serve as a good guide for the regulatory requirements needed throughout clinical development. jpg Tags Clinical Trials Weight 1

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Drug Discovery World

AUGUST 12, 2022

Pfizer has launched a Phase III clinical trial of a vaccine to protect people against Lyme disease, in collaboration with pharmaceutical company Valneva. . The trial will enrol up to 6,000 participants aged five years and older and will be conducted across 50 sites where Lyme disease is highly endemic. Official comments .

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Drug Discovery World

JUNE 26, 2024

Biotechnology company EG 427 has revealed plans to start a Phase Ib/IIa clinical study in neurogenic detrusor overactivity (NDO), following Investigational New Drug (IND) clearance from the US Food and Drug Administration (FDA).

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Cytel

OCTOBER 27, 2023

Speakers discussed investigator, regulatory (FDA), industry, and patient perspectives during the special symposium “Challenging the Status Quo of Early Phase Clinical Trial Design: Project Optimus.” To address this challenge, the FDA Oncology Center of Excellence initiated Project Optimus.

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Alta Sciences

FEBRUARY 8, 2024

The FDA guidance, Evaluating Drug Effects on the Ability to Operate a Motor Vehicle , indicates that testing in early-phase clinical development should emphasize sensitivity over specificity in CNS effects. Tags Clinical Trials Weight 12 View the Driving Simulation Fact Sheet for more information.

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Drug Discovery World

FEBRUARY 14, 2024

AlphaMedix is a targeted alpha therapy currently in Phase II clinical development, which consists of an SSTR-targeting peptide complex radiolabeled with lead-212 (212Pb) that serves as an in vivo generator of alpha particles. In the Phase I study, treatment was well-tolerated, with a response rate of 62.5%

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The Pharma Data

DECEMBER 14, 2020

The trial, which will explore the pharmacokinetics and safety of ATX01 in healthy volunteers, is due to start in January 2021. AlgoTherapeutix recently raised a 12M€ Series A that will fund the Phase 1 and 2 clinical development of ATX01. More information at www.algotx.com. View source version on businesswire.com: [link].

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Drug Discovery World

JUNE 6, 2024

Professor Charles Swanton, Cancer Research UK’s chief clinician, said the melanoma trial results were “extremely impressive”, adding: “The new vaccine approach is another piece of the puzzle that will allow more patients to be cured, hopefully, or fewer patients to suffer disease relapse. The follow-up of the Phase IIb study took place 34.9

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Drug Discovery World

APRIL 12, 2024

The Food and Drug Administration (FDA) has cautioned that continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. This latest approval means that Enhertu now has five approved indications, including for breast cancer.

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The Pharma Data

JUNE 2, 2023

Food and Drug Administration (FDA) for the treatment of adult patients with deleterious or suspected deleterious BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC). Patients should be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

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Drug Discovery World

APRIL 13, 2023

In 2018, the US Food and Drug Administration (FDA) introduced a policy for accepting real-world data (RWD) and real-world evidence (RWE) in addition to traditional clinical trial data in new drug applications. Since September 2022, additional FDA submission guidance has further highlighted the importance of RWD and RWE.

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Alta Sciences

MARCH 21, 2024

Top 5 Scientific Resources in CNS Clinical Trials blussier Thu, 03/21/2024 - 18:09 HTML CNS Center of Excellence Resource Library Altasciences has an extensive understanding of psychiatric and neurologic disorders, stemming from our decades of conducting complex studies in various therapeutic indications impacting the central nervous system.

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Drug Discovery World

FEBRUARY 26, 2024

The US Food and Drug Administration (FDA) has granted Fast Track Designation for Certa Therapeutics’ investigational therapy FT011 for the treatment of systemic sclerosis (scleroderma). The post Scleroderma drug gets fast-tracked in the US appeared first on Drug Discovery World (DDW).

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The Pharma Data

NOVEMBER 30, 2020

Two of the three planned late-stage efficacy trials for NVX-CoV2373 sponsored by Novavax are fully enrolled, and more than 20,000 participants have been dosed to-date. In alignment with Novavax’ commitment to transparency, Phase 3 clinical trial protocols are posted to the company’s website at Novavax.com/resources upon finalization.

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Drug Discovery World

MARCH 7, 2024

A combined pipeline of therapeutic candidates Akari’s nomacopan is a bispecific recombinant inhibitor of complement C5 and leukotriene B4 (LTB4) being evaluated in a Phase III clinical trial for paediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA).

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Drug Discovery World

MAY 3, 2023

Vicore Pharma, a Swedish clinical-stage pharmaceutical company unlocking the potential of angiotensin II type 2 receptor agonists (ATRAGs), has dosed its first patient with C21 in a clinical study of endothelial dysfunction. Vicore Pharma states that results from the trial are expected in Q4, 2023.

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Drug Discovery World

NOVEMBER 10, 2023

Topline results from a Phase I/II clinical trial have shown that Kiora’s light-restoring small molecule KIO-301 has the potential to meaningfully improve vision in patients with retinitis pigmentosa (RP). KIO-301 was safe and well tolerated with no ocular and non-ocular serious adverse events, nor any signs of retinal inflammation.

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Advarra

JUNE 20, 2023

of new cancer drugs tested in Phase I were likely to receive Food and Drug Administration (FDA) approval. Additionally, in 2021, a literature review estimated the average capitalized research and development (R&D) costs per new cancer medicine at between $944 million and $4.54

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The Pharma Data

AUGUST 24, 2020

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. In a paper published in Molecular Therapy: Methods & Clinical Development , the team discussed why they choose this type of gene therapy to treat HIV. Air Force photo by Kemberly Groue.

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FDA Law Blog: Drug Discovery

JUNE 12, 2024

Sasinowski — Hyman, Phelps & McNamara (HPM) would like to congratulate the 7 rare disease programs selected for the inaugural class of the FDA’s “Support for clinical Trials Advancing Rare disease Treatment” (START) pilot program. HPM is proud to aid 5 of the 7 selected participants for the START pilot program.

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Drug Discovery World

JUNE 1, 2023

According to NCBI, most of the ADCs developed so far are for use in the treatment of cancer, but there is plenty of potential for using ADCs to treat other diseases 1. A Nature publication confirmed that there are currently 12 FDA-approved ADCs on the market, and nine of these secured FDA approval in the past six years 2.

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Advarra

FEBRUARY 24, 2023

In most cases, the results you are really looking for and trying to prove in a trial can be complex. One area receiving increased focus from the Food and Drug Administration (FDA) are trial designs incorporating multiple endpoints to support efficacy.

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The Pharma Data

JULY 8, 2021

Food and Drug Administration (FDA) has approved an updated label for ADUHELM (aducanumab-avwa) injection 100 mg/mL solution. The update includes an addition to the Indications and Usage section of the label (Section 1) to emphasize the disease stages studied in the clinical trials, as seen below ( italics to note updated language).

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Drug Discovery World

DECEMBER 20, 2022

The therapy was also recently approved by the FDA for patients in the US. . The clinical development of etranacogene dezaparvovec was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment. . World’s most expensive drug.

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Cytel

SEPTEMBER 1, 2023

Data is the cornerstone of any clinical trial, driving the decision-making process of drug development, and is a fundamental requirement for approval of new therapies by regulatory agencies like the FDA or EMA.

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Drug Discovery World

FEBRUARY 28, 2024

Using IgE to treat cancer In the UK, a Cancer Research UK-funded clinical trial has shown, for the first time, that a new class of antibody could benefit cancer patients whose existing treatments have stopped working. We eagerly anticipate ongoing engagement with the FDA as we progress in the development of this promising treatment.”

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