2018, Disease and Trials - Drug Discovery Digest

Otsuka’s startup bet pays off with kidney disease drug results

BioPharma Drive: Drug Pricing

OCTOBER 22, 2024

A medicine the pharma acquired in a $430 million buyout of Visterra in 2018 succeeded in a Phase 3 trial in IgA nephropathy, a crowded corner of drug research.

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At Long Last, FDA Unveils Plan for Rare Disease Innovation Hub

FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. Early Direction for the Rare Disease Innovation Hub The Hub, which will be co-chaired by Drs. By Sarah Wicks & James E.

FDA

FDA Disease Clinical Pharmacology Science

Building better brain models for Parkinson’s disease and beyond

Drug Target Review

MARCH 3, 2025

The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. “For diseases like Parkinson’s, it’s more than sufficient,” he explains.

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Bridging Innovation & Patient Care: The Growing Role of AI

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New avenues for rare disease treatment

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.

Disease

Disease Treatment Small Molecule Therapies

The Long Road to End Tuberculosis

Codon

NOVEMBER 3, 2024

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 million people every year. Subscribe to Asimov Press.

Vaccine

Vaccine Trials Therapies Treatment

The genetic modifier approach: identifying the right target for rare diseases

Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

Disease

Disease Clinical Trials Therapies Science

New molecular insights on medical cannabis

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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Before Clinical Trials: The Long Game of Rare Disease Research

Conversations in Drug Development Trends

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. Over the next few decades, that drug made its way through clinical trials, securing approval in 2007—just 36 months before Rob was diagnosed in 2010. Uplifting Athletes (UA) is on a mission to change that. But what was truly remarkable?

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FDA Creates a New Advisory Committee for Genetic Metabolic Diseases – Could This Be an Opportunity to Support Rare Disease Product Development More Broadly?

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.

Disease

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Jardiance® phase III EMPA-KIDNEY trial will stop early due to clear positive efficacy in people with chronic kidney disease

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.

Trials

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Deliberate Dysentery

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?

Vaccine

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Alzheimer’s disease: driving advancements with precision medicine

Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing. We believe we are at a breakthrough moment in Alzheimer’s disease research.

Disease

Disease Clinical Trials Therapies Science

Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine

The Pharma Data

JUNE 26, 2021

Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine. The trial will evaluate the safety and immunogenicity of a monovalent flu vaccine candidate coding for the hemagglutinin protein of the A/H3N2 strain of the influenza virus. JUNE 22 , 2021.

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Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

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.

Therapies

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Sanofi and Translate Bio initiate Phase 1/2 clinical trial of mRNA COVID-19 vaccine candidate

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.

Clinical Trials

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AstraZeneca reaches deal with RenalytixAI to develop medicine strategies for a variety of diseases

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.

Disease

Disease Clinical Trials Trials

Decoding Clinical Trial Costs and Efficiency Roadblocks

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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Prilenia Enrolls First Subject in Europe in its PROOF-HD Phase 3 Clinical Trial for Huntington’s Disease

The Pharma Data

JANUARY 27, 2021

a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, today announces the enrollment of the first participant in Europe in its global Phase 3 clinical trial for HD. The first US subjects in the PROOF-HD trial were enrolled in October 2020.

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Pfizer Announces Start of Phase 3 Clinical Trial in Adults.

The Pharma Data

SEPTEMBER 2, 2021

RSV is a significant cause of severe respiratory disease in older adults, and it can cause disability and death. There is an important unmet medical need for an effective vaccine that can help protect older adults against this highly-contagious disease,” said Kathrin U. Jansen, Ph.D., 2 In the U.S., Source link: [link].

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Meet the Patient Advocate Panelists in Our Upcoming Webinar: Rare Disease Day 2023 Wrapped, What You Need to Take Away

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.

Disease

Disease Clinical Trials Trials Treatment

SHINE trial on shorter treatment for children with minimal TB

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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VALILTRAMIPROSATE

New Drug Approvals

OCTOBER 12, 2024

Years later, a subgroup analysis of the trial data indicated a potential positive effect in participants who carried two copies of ApoE4 ( Abushakra et al., Years later, a subgroup analysis of the trial data indicated a potential positive effect in participants who carried two copies of ApoE4 ( Abushakra et al., 2018; 32(9): 849–861.

Pharmacokinetics

Pharmacokinetics Small Molecule Disease Licensing

Kala Gets the Greenlight from FDA for Dry Eye Disease Treatment

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. .

Treatment

Treatment Disease FDA FDA Approval

AZD7442 PROVENT Phase III prophylaxis trial met primary endpoint in.

The Pharma Data

AUGUST 22, 2021

The trial accrued 25 cases of symptomatic COVID-19 at the primary analysis.There were no cases of severe COVID-19 or COVID-19-related deaths in those treated with AZD7442. The trial included 5,197 participants in a 2:1 randomisation AZD7442 to placebo. The trial was conducted in 87 sites in the US, UK, Spain, France and Belgium.

Trials

Trials Virus Vaccine Pharmacokinetics

ATUZAGINSTAT

New Drug Approvals

SEPTEMBER 29, 2024

gingivalis has been called a risk factor for Alzheimer’s disease ( Kanagasingam et al., 2018 ; Ding et al., gingivalis is associated with cardiovascular disease. In aged dogs with periodontal disease, ninety days of COR388 reduced oral bacterial load and gum pathology ( Arastu-Kapur et al., 2017 ; Ilievski et al.,

Small Molecule

Small Molecule DNA Disease Trials

MPAACT Consortium Unites Industry and Academia to Establish Measurable Residual Disease as a Surrogate Endpoint in Acute Myeloid Leukemia Drug Development

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.

Disease

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The power of combinations in blood cancers

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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Knopp Biosciences Announces Positive Top-Line Results from the Phase 2 EXHALE Trial of Oral Dexpramipexole in Moderate-to-Severe Eosinophilic Asthma

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.

Trials

Trials Small Molecule Clinical Trials Treatment

Where's the Synthetic Blood?

Codon

JULY 14, 2024

However, the subsequent death of another patient thrust Denys into a contentious trial. In contrast, people with sickle cell disease commonly receive red blood cell transfusions to alleviate vaso-occlusive pain crises. The results of the trial are expected at the end of 2024. In the U.S., million units transfused.

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Phase 3 trial of Libtayo® (cemiplimab) monotherapy in advanced cervical cancer stopped early for positive result on overall survival

The Pharma Data

MARCH 15, 2021

Trial enrolled patients with advanced cervical cancer regardless of PD-L1 status. The trial will be stopped early based on a unanimous recommendation by the Independent Data Monitoring Committee (IDMC), and the data will form the basis of regulatory submissions in 2021. This is reflected in the trial where the average age was 51.”.

Trials

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Human neuronal cells: possibilities in drug safety testing

Drug Target Review

SEPTEMBER 26, 2023

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. Dr Rockley previously completed an MSc in Cancer Pharmacology and have experience in clinical research from an oncology clinical trial coordinator role. 2018 Mar;17(3):167–81.

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Clover Biopharmaceuticals Announces Publication of Phase 1 Clinical Trial Data for its Adjuvanted COVID-19 Vaccine Candidates in The Lancet

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.

Clinical Trials

Clinical Trials Vaccine Trials Virus

Topical Gene Therapy FDA-Approved for Severe Skin Disease, Dystrophic Epidermolysis Bullosa

PLOS: DNA Science

JUNE 1, 2023

This DNA Science post from 2018 traces the history of the efforts. Two clinical trials were conducted and results published in December. Peter Marinkovich, MD, director of the Blistering Disease Clinic at Stanford Health Care, calls DEB a devastating disease. DEB has been a candidate for a gene therapy since 2002.

FDA Approval

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Tezepelumab NAVIGATOR Phase 3 Trial Met Primary Endpoint Of A Statistically Significant And Clinically Meaningful Reduction In Exacerbations In A Broad Population Of Patients With Severe Asthma

The Pharma Data

NOVEMBER 9, 2020

Trial Also Met the Primary Endpoint in Patients With Low Levels of Eosinophils. In the subgroup of patients with baseline eosinophil counts less than 300 cells per microliter, the trial met the primary endpoint with tezepelumab demonstrating a statistically significant and clinically meaningful reduction in AAER.

Trials

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Tiziana Life Sciences announces collaboration with Parexel Biotech to conduct phase 1b/2 clinical trial in patients with Crohn’s Disease

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.

Clinical Trials

Clinical Trials Science Disease Trials

InnoCare Announces Clearance by the US FDA of Phase II Clinical Trial Using orelabrutinib for the Treatment of Multiple Sclerosis

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.

Clinical Trials

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WORLD’S LARGEST BROWSABLE RESOURCE LINKING RARE PROTEIN-CODING GENETIC VARIANTS TO HUMAN HEALTH AND DISEASE

The Pharma Data

JULY 8, 2021

Today, access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE). The browser can be accessed via [link]. About AbbVie.

Disease

Disease Clinical Trials Government Research

How to Get Drugs Into the Brain

Drug Hunter

AUGUST 21, 2023

Obtaining adequate drug exposure in the brain is key to treating CNS diseases effectively. Prodrugs Remain in “Proof-of-Concept” Stages Prodrug strategies for CNS deliver have also been applied with varying success , with the most famous example being L-Dopa for Parkinson’s disease. What is K p,uu ? Figure 18.

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Drugs Disease Targeted Protein Degradation Screening Hits

Gamma delta T cells: a rising star in cancer therapy

Drug Target Review

FEBRUARY 28, 2024

Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers. Human γδ TCR repertoires in health and disease. Trends Immunol 39(6):446-459 (2018).

Therapies

Therapies Engineering Molecular Biology Science

#WhyIScience Q&A: How a computational biologist balances work with life as an elite rower

Broad Institute

MAY 2, 2024

In 2018, she graduated with a bachelor’s degree in computer science and biology and joined the lab of Gad Getz at the Broad Institute of MIT and Harvard to complete her master’s. In 2021, Martin competed in the US Olympic trials in a lightweight double, and won the world-renowned Head of the Charles regatta in lightweight single sculls.

Science

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Tezepelumab regulatory submission accepted and granted FDA Priority Review in the US for the treatment of patients with asthma

The Pharma Data

JULY 8, 2021

Tezepelumab is the first and only biologic to consistently and significantly reduce asthma exacerbations in a broad population across Phase II and III clinical trials. The BLA was based on results from the PATHFINDER clinical trials programme, including results from the pivotal NAVIGATOR Phase III trial.

Treatment

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Article FDA Thank You FDA advisors endorse minimal residual disease (MRD) as accelerated approval endpoint for multiple myeloma

Agency IQ

APRIL 19, 2024

FDA advisors endorse minimal residual disease (MRD) as accelerated approval endpoint for multiple myeloma Last week, FDA’s Oncologic Drugs Advisory Committee (ODAC) voted unanimously in favor of using minimal residual disease (MRD) as an accelerated approval endpoint for multiple myeloma.

FDA

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Q&A: How a stem cell bank is helping scientists understand psychiatric disorders

Broad Institute

JULY 25, 2024

By Allessandra DiCorato July 25, 2024 Credit: Maria Nemchuk, Broad Communications Ralda Nehme When Ralda Nehme, a cell biologist and neuroscientist, first started her lab at the Stanley Center for Psychiatric Research at the Broad Institute of MIT and Harvard in 2018, she realized a gap in the field.

Clinical Trials

Clinical Trials Disease DNA Trials

Update On SOURCE Phase 3 Trial For Tezepelumab In Patients With Severe, Oral Corticosteroid-Dependent Asthma

The Pharma Data

DECEMBER 21, 2020

21, 2020 /PRNewswire/ — Amgen (NASDAQ:AMGN) and AstraZeneca today announced the SOURCE trial did not meet the primary endpoint of a statistically significant reduction in the daily oral corticosteroid (OCS) dose, without loss of asthma control, with tezepelumab compared to placebo. THOUSAND OAKS, Calif. ,

Trials

Trials Production Clinical Trials Therapies

Otsuka’s startup bet pays off with kidney disease drug results

At Long Last, FDA Unveils Plan for Rare Disease Innovation Hub

Webinars

Trending Sources

Building better brain models for Parkinson’s disease and beyond

Webinars

New avenues for rare disease treatment

The Long Road to End Tuberculosis

The genetic modifier approach: identifying the right target for rare diseases

New molecular insights on medical cannabis

Before Clinical Trials: The Long Game of Rare Disease Research

FDA Creates a New Advisory Committee for Genetic Metabolic Diseases – Could This Be an Opportunity to Support Rare Disease Product Development More Broadly?

Jardiance® phase III EMPA-KIDNEY trial will stop early due to clear positive efficacy in people with chronic kidney disease

Deliberate Dysentery

Alzheimer’s disease: driving advancements with precision medicine

Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine

Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

Sanofi and Translate Bio initiate Phase 1/2 clinical trial of mRNA COVID-19 vaccine candidate

AstraZeneca reaches deal with RenalytixAI to develop medicine strategies for a variety of diseases

Decoding Clinical Trial Costs and Efficiency Roadblocks

Prilenia Enrolls First Subject in Europe in its PROOF-HD Phase 3 Clinical Trial for Huntington’s Disease

Pfizer Announces Start of Phase 3 Clinical Trial in Adults.

Meet the Patient Advocate Panelists in Our Upcoming Webinar: Rare Disease Day 2023 Wrapped, What You Need to Take Away

SHINE trial on shorter treatment for children with minimal TB

VALILTRAMIPROSATE

Kala Gets the Greenlight from FDA for Dry Eye Disease Treatment

AZD7442 PROVENT Phase III prophylaxis trial met primary endpoint in.

ATUZAGINSTAT

MPAACT Consortium Unites Industry and Academia to Establish Measurable Residual Disease as a Surrogate Endpoint in Acute Myeloid Leukemia Drug Development

The power of combinations in blood cancers

Knopp Biosciences Announces Positive Top-Line Results from the Phase 2 EXHALE Trial of Oral Dexpramipexole in Moderate-to-Severe Eosinophilic Asthma

Where's the Synthetic Blood?

Phase 3 trial of Libtayo® (cemiplimab) monotherapy in advanced cervical cancer stopped early for positive result on overall survival

Human neuronal cells: possibilities in drug safety testing

Clover Biopharmaceuticals Announces Publication of Phase 1 Clinical Trial Data for its Adjuvanted COVID-19 Vaccine Candidates in The Lancet

Topical Gene Therapy FDA-Approved for Severe Skin Disease, Dystrophic Epidermolysis Bullosa

Tezepelumab NAVIGATOR Phase 3 Trial Met Primary Endpoint Of A Statistically Significant And Clinically Meaningful Reduction In Exacerbations In A Broad Population Of Patients With Severe Asthma

Tiziana Life Sciences announces collaboration with Parexel Biotech to conduct phase 1b/2 clinical trial in patients with Crohn’s Disease

InnoCare Announces Clearance by the US FDA of Phase II Clinical Trial Using orelabrutinib for the Treatment of Multiple Sclerosis

WORLD’S LARGEST BROWSABLE RESOURCE LINKING RARE PROTEIN-CODING GENETIC VARIANTS TO HUMAN HEALTH AND DISEASE

How to Get Drugs Into the Brain

Gamma delta T cells: a rising star in cancer therapy

#WhyIScience Q&A: How a computational biologist balances work with life as an elite rower

Tezepelumab regulatory submission accepted and granted FDA Priority Review in the US for the treatment of patients with asthma

Article FDA Thank You FDA advisors endorse minimal residual disease (MRD) as accelerated approval endpoint for multiple myeloma

Q&A: How a stem cell bank is helping scientists understand psychiatric disorders

Update On SOURCE Phase 3 Trial For Tezepelumab In Patients With Severe, Oral Corticosteroid-Dependent Asthma

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