Drug Target Review

OCTOBER 11, 2023

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.

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FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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Vial

MAY 13, 2024

However, progress from molecule to approved drug is hampered by extremely high costs and lengthy clinical trials , and approximately 90% of drugs that reach clinical trials fail. only 5% of molecules in oncology Phase I trials reach the market taking, on average, 7.5 An estimated 80% of clinical trials do not finish on time.

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Drug Discovery World

APRIL 11, 2023

12 The trial Tezepelumab was well tolerated in patients with severe asthma and resulted in clinically meaningful reductions in asthma exacerbations in NAVIAGTOR & SOURCE clinical trials and no clinically meaningful differences in safety results between the tezepelumab and placebo groups. 2018; 10 (1): 34‐45 Duchesne M, et al.

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Drug Discovery World

DECEMBER 5, 2022

A new partnership between industry and three rare disease foundations has formed with the goal of accelerating the development of precision drugs to treat rare neurodevelopmental disorders. . Understanding rare diseases remains a significant challenge. Understanding rare diseases remains a significant challenge.

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The Pharma Data

MARCH 16, 2022

The Independent Data Monitoring Committee recommended that the EMPA-KIDNEY trial be stopped early, following a formal interim assessment EMPA-KIDNEY is the largest and broadest dedicated SGLT2 inhibitor trial in chronic kidney disease to date Detailed results are expected to be presented later this year.

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Drug Discovery World

OCTOBER 9, 2023

The reported MOD framework advances the mechanistic rationale of the clinical benefits on fatigue observed in our clinical trial with AXA1125 in the context of the combination’s mechanism of action. This is a new dynamic and efforts on research, clinical trials regulatory, and financial support are required.

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Drug Discovery World

MARCH 17, 2023

Since 2018, GARDP and the University of Liverpool have been working together to advance new treatment options for new-borns with sepsis. Their research has laid the groundwork for GARDP’s upcoming global clinical trial on neonatal sepsis.

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Drug Discovery World

SEPTEMBER 26, 2022

The approval by the European Commission was based on results from the Pathfinder clinical trial programme, which included the pivotal Navigator Phase III trial in which Tezspire demonstrated superiority across every primary and key secondary endpoint in patients with severe asthma, compared to placebo, when added to standard therapy.

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Drug Target Review

NOVEMBER 1, 2023

2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 2018 Jun 13;5. Frontiers in Medicine. J Med Chem.

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Conversations in Drug Development Trends

FEBRUARY 22, 2023

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

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Drug Discovery World

DECEMBER 18, 2023

Digital biomarkers promise to transform preventive medicine and clinical trials, but also raise issues of security, regulation and potential disparities in access. Deepika Khedekar, Clinical Trial Lead at IQVIA, provides an overview of the pros and cons, and suggests a possible solution.

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Drug Discovery World

JANUARY 31, 2023

NICE’s independent appraisal committee considered new evidence, including data from a clinical trial and from people who received axicabtagene ciloleucel through the CDF, before making the recommendation. It is an aggressive disease which progresses quickly and is the most common type of high-grade non-Hodgkin lymphoma (NHL).

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The Pharma Data

MARCH 11, 2021

Clinical trial to assess safety, immune response and reactogenicity, after preclinical data showed high neutralizing antibody levels. The Companies expect interim results from this trial in the third quarter of 2021. About the Phase 1/2 clinical trial. Expected to enroll 415 participants; interim results expected in Q3 2021.

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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 market research report published in late June 2018 by HTF market Intelligence Consulting suggested that the ADC market in China would grow exponentially by 2023 4.

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The Pharma Data

AUGUST 24, 2020

AstraZeneca and RenalytixAI have agreed a deal to develop joint precision medicine strategies for cardiovascular, renal and metabolic diseases. The companies will first target chronic kidney disease and use RenaltytixAI’s diagnostic platform, called KidneyIntelX, to examine how to improve patient outcomes.

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The Pharma Data

MAY 5, 2022

Validated Measurable Residual Disease Assays are Critical for the Rapid Development of Future Novel Therapeutics for Patients with Acute Myeloid Leukemia. 1 Clinical trials for new therapies in AML are becoming increasingly lengthy to complete based on the traditional clinical endpoint of survival. About Acute Myeloid Leukemia.

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PLOS: DNA Science

MARCH 21, 2024

Lenmeldy adds functional genes that encode the enzyme arylsulfatase A (ARSA) to bone marrow stem cells taken from children who have inherited the disease, but haven’t yet developed symptoms, which typically begin around age 2 or 3. Two of their children, Liviana and Giovanni, participated in the clinical trial.

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Codon

JANUARY 21, 2024

Today, refined versions of these human challenge studies have become standard practice in testing vaccines for vector-borne diseases (e.g., yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops. Why are challenge trials becoming more popular?

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The Pharma Data

NOVEMBER 2, 2020

The SHINE trial is a phase 3 randomised open-label trial comparing four versus six months of treatment with rifampicin, isoniazid, pyrazinamide plus or minus ethambutol in children with smear-negative, non-severe TB. 1204 children aged under 16 years participated in the trial, including 127 children living with HIV infection.

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The Pharma Data

OCTOBER 26, 2020

Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . adults have been diagnosed with dry eye disease, a chronic, episodic, multifactorial disease. It also increases your risk for inflamed cornea, cornea eye disease, corneal ulcers and injuries, and eye infections. .

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The Pharma Data

JANUARY 13, 2021

PITTSBURGH–( BUSINESS WIRE )– Knopp Biosciences LLC today announced positive top-line results in a Phase 2 dose-ranging trial of the novel oral drug dexpramipexole in patients with moderate-to-severe eosinophilic asthma. The trial was conducted at 28 U.S. 14, 2021 11:00 UTC. study centers.

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The Pharma Data

NOVEMBER 10, 2020

P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease. Crohn’s Disease Therapeutics Market Size $4.7 The trial is a dose-ranging, open-label study that will enroll 60 patients in the U.S. Billion by 2025. NEW YORK and LONDON, Nov.

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Drug Discovery World

MARCH 29, 2023

UMNs have been defined in the context of the draft as diseases where there’s a lack of good treatments, and which have a high burden 3. For rare disease drugs, a variable RDP period of five, nine, or ten years, depending on the novelty of the drug.

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The Pharma Data

JANUARY 31, 2021

Adjuvanted S-Trimer COVID-19 vaccine candidates demonstrated favorable safety and tolerability profiles and strong neutralizing immune responses in a phase 1 trial. Clover plans to initiate a global phase 2/3 trial in the first half of 2021 with an interim analysis for vaccine efficacy potentially in the middle of 2021.

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The Pharma Data

NOVEMBER 1, 2020

The trial is expected to enroll 160 patients. MS is an autoimmune, inflammatory disease of the central nervous system. billion in 2018, and it is expected to be up to US$48.9 BTK inhibitors have the potential to transform the treatment paradigm of autoimmune diseases including MS. billion by 2030 2.

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Drug Target Review

NOVEMBER 6, 2023

Advances in disease understanding and combination strategies in haematology We now know that blood cancers are characterised by molecular and phenotypic heterogeneity. Published 2018 Nov 13. 2 It is this complexity that necessitates powerful, targeted combination therapies. Available at: [link] Accessed October 2023. Front Pharmacol.

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Agency IQ

MARCH 29, 2024

FDA’s oncology advisory committee to consider MRD as a multiple myeloma endpoint Yesterday, the FDA announced that its Oncologic Drugs Advisory Committee would weigh in on the potential use of minimal residual disease (MRD) as an endpoint in multiple myeloma clinical trials. immune cells and other blood cell types) lack.

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The Pharma Data

MARCH 15, 2022

The sBLA submitted to the FDA by AstraZeneca included data from the OSTRO Phase III trial, which met both co-primary endpoints with a safety profile consistent with the known profile of the medicine. The Company remains committed to bringing Fasenra to patients with CRSwNP and a second Phase III trial, ORCHID , in this indication is ongoing.

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Drug Discovery World

OCTOBER 20, 2022

In 2018, for instance, Tohoku University signed a partnership agreement with Phillips Japan, a subsidiary of Dutch multinational Phillips, to conduct joint research in the field of healthcare. Indeed, Phillips has a co-creation space at Tohoku University’s CRIETO hub. . Eisai and Biogen’s anti-amyloid beta (A?)

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Drug Target Review

NOVEMBER 7, 2023

To target proteins involved in causing or spreading disease, typically a drug will obstruct a protein’s active site so it cannot function. Proteins can become part of a cancerous tumour and allow for its unrestricted growth, cause diseases like Alzheimer’s , and build up in the heart to impact how it pumps blood around the body.

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The Pharma Data

DECEMBER 31, 2020

Tabibiazar co-founded private Aravive Biologics and served as the Chairman of its board of directors and as President and Chief Executive Officer from its inception to April 2017 and as Executive Chairman from May 2017 until October 2018. since October 2018. to form the combined company, Aravive, Inc.

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LifeSciVC

JANUARY 25, 2024

Testing drugs that make it into clinical trials has its own specific challenges. Designing trials to show safety and efficacy in people and doing it in a way that complies with all the regulatory and ethical guidelines is where many drugs fail. I am proud that these drugs are still helping patients today.

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The Pharma Data

JANUARY 14, 2021

(NASDAQ: FPRX) today announced clinical results from the global, randomized, double-blind placebo-controlled Phase 2 FIGHT trial evaluating first-in-class targeted therapy bemarituzumab in advanced gastric or gastroesophageal junction (GEJ) cancer. Phase 2 FIGHT Trial: Summary of Efficacy*. Wainberg, M.D., .

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The Pharma Data

MARCH 29, 2022

The approval by the Japanese Ministry of Health, Labour and Welfare was based on positive results from the ANNEXA-4 Phase III clinical trial showing Ondexxya rapidly and markedly reversed anti-FXa activity in patients with acute major bleeding. During the trial, 10.4% of patients died during the trial.

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The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. When the company launched an IPO in late 2018, its valuation was $7 billion, which at the time was a record biotech IPO.

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The Pharma Data

JUNE 18, 2021

1 Clinical remission was defined by Crohn’s Disease Activity Index (CDAI) in the U.S. “These results represent another step towards the development of risankizumab for these patients, many of whom do not find sufficient disease control with current treatments.” NORTH CHICAGO, Ill. vice chairman and president, AbbVie.

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