2026, Disease and Trials - Drug Discovery Digest

Drug candidate for ALS patients authorised for Phase II clinical trial

Drug Discovery World

APRIL 16, 2024

Axoltis Pharma has received authorisation from ANSM, the French agency for the safety of health products, to launch the SEALS study for Amyotrophic Lateral Sclerosis (ALS). This Phase II clinical trial of drug candidate NX210c in patients with ALS is the first to target the integrity of the blood brain barrier (BBB).

Clinical Trials

Clinical Trials Trials Drugs Pharmacokinetics

Next steps for the CALYX trial for cerebral Adrenoleukodystrophy

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

Trials

Trials Clinical Trials FDA Approval FDA

Study finds dual receptor beta-agonist promotes healthy weight loss

Drug Discovery World

MAY 30, 2024

We are now advancing our next generation small molecules for the treatment of obesity to IND preparatory studies in H1 next year and expect to enter the clinic in 2026.” Atrogi is also conducting trials into ATR-258, a novel b2adrenoceptor agonist for the treatment of type 2 diabetes and comorbidities.

Small Molecule

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Manchester to get £60m development to enhance lab space

Drug Discovery World

MAY 16, 2024

Greenheys at Manchester Science Park is scheduled for completion in Summer 2026 and will span 131,000 sq ft across six floors. This includes direct access to R&D and funding support, highly skilled talent, and clinical trial opportunities. Work has started on a £60 ($75.1)

Laboratories

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Could GLP-1 receptor agonists treat addiction and dementia?

Drug Discovery World

AUGUST 21, 2023

Semaglutide manufacturer Novo Nordisk reported earlier this month that the drug reduces the risk of major adverse cardiovascular events by 20% in adults with overweight or obesity, as shown in the SELECT trial. However, these trials won’t be complete until 2026 at the earliest. It’s an exciting area,” he told The Guardian.

Clinical Trials

Clinical Trials Trials Treatment Licensing

Antibody Drug Conjugates: windows of opportunity

Drug Target Review

JULY 4, 2023

The global ADC market is expected to continue this steep upward trajectory and is anticipated to exceed $16 Billion by 2026. In practice, some patients may be left managing a syndrome associated with the therapy along with the disease. 3D rendering of Antibody Drug Conjugate Molecules. 3D rendering of Antibody Drug Conjugate Molecules.

Small Molecule

Small Molecule Drugs Clinical Pharmacology Trials

Novartis Entresto receives positive CHMP opinion for pediatric heart failure

The Pharma Data

MARCH 31, 2023

However, the disease is largely managed with treatments approved for adults, based on data from adult studies,” said David Soergel, M.D., This would support extension of the regulatory data protection to November 2026. Global Head, Cardiovascular, Renal and Metabolism Development Unit, Novartis.

Hospitals

Hospitals Trials Treatment Clinical Trials

The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. In 2021, levels of investment hit an all-time high at $22.7 billion, compared to $19.9

Drugs

Drugs Therapies Clinical Trials Cell Based Assays

From cradle to grave: Novo Nordisk Foundation and cardiometabolic diseases

Drug Discovery World

JULY 1, 2024

Following a trip to Copenhagen, Denmark, DDW’s Megan Thomas reflects on the Novo Nordisk Foundation’s efforts to tackle cardiometabolic diseases. The World Health Organization (WHO) reports that cardiovascular diseases (CVDs) are the leading cause of death globally, stating that an estimated 17.9 million ($3.4 million ($3.4

Disease

Disease International Research Science

Understanding the Impact of the New EU Artificial Intelligence Act on Clinical Research

Advarra

AUGUST 8, 2024

While some of the AI Act’s compliance dates are set for August 2024, the full Act will be enforced in March 2026. High Risk AI-powered Systems: Key Requirements The AI Act will likely consider many AI-based systems used in clinical trials today as “high risk.” This is not an exhaustive list; reference the AI Act for complete details.):

Clinical Research

Clinical Research Research Compliance Clinical Trials

The challenges and trends of cell & gene therapies

Drug Discovery World

SEPTEMBER 6, 2022

This area of medicine, which turns our own bodies into agents of combat to fight disease, has quickly become one of the most promising fields in treating deadly diseases such as cancer, central nervous system disorders and even musculoskeletal conditions. . billion, compared to $19.9 billion, compared to $19.9

Therapies

Therapies Clinical Trials Vaccine Disease

Ionis highlights achievements, commercial strategy and technology advancements at Investor Day

The Pharma Data

DECEMBER 8, 2020

The company’s commercial priorities are three-fold: (1) Initially focusing its commercial efforts on rare diseases within its prolific neurology and cardiology franchises (2) pioneer new markets where there are no available treatments (3) create new standards of care where there has been a lack of innovation to optimize patient care.

RNA

RNA Pharmaceuticals Production Disease

GSK reaches agreement to acquire late-stage biopharmaceutical company Sierra Oncology for $1.9bn

The Pharma Data

APRIL 15, 2022

In January 2022, Sierra Oncology announced positive topline results from the MOMENTUM phase III trial. New GSK reaffirms its full-year 2022 guidance, the medium-term outlook for 2021-2026 of more than 5% sales and 10% adjusted operating profit CAGR* at CER**, and long-term sales ambition. GSK is a science-led global healthcare company.

FDA Approval

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Article FDA Thank You What’s on FDA’s new Diagnostics Chief’s mind? Transitions, the LDT rule and cybersecurity

Agency IQ

AUGUST 23, 2024

At a high level, the pilot program was intended to offer sponsors of certain drug products that would need a CDx with the opportunity to submit information about their Clinical Trial Assay (CTA) – the assay they’re using in a pivotal trial – to the FDA [see here for background on that topic].

FDA

FDA Laboratories Regulations Clinical Trials

Analysis Life Sciences Thank You What We Expect the FDA to do in August and September 2024

Agency IQ

AUGUST 2, 2024

Rare Pediatric Disease Priority Review Voucher Program Expiration Threat: The FDA’s popular voucher program for rare pediatric diseases is currently scheduled to expire as of September 30, 2024 unless it is renewed by Congress.

FDA

FDA Science Regulations Biosimilars

Sandoz strengthens pipeline expansion through partnership to develop and manufacture multiple biosimilars

The Pharma Data

MAY 15, 2023

Work is set to begin this year, with full operations provisionally planned for late 2026. In addition, Sandoz recently signed a Memorandum of Understanding to build a new biologics production plant in Slovenia – an expected investment of at least USD 400 million to support increasing global demand for biosimilar medicines.

Five cell and gene therapy advances in neurodegeneration

Drug Discovery World

JULY 29, 2024

Neurodegenerative diseases have recently increased at an alarming rate due to the ageing global population. A neurorestorative treatment Asklepios BioPharmaceutical’s (AskBio’s) AB-1005 is an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of moderate-stage Parkinson’s disease.

Therapies

Therapies Clinical Trials RNA Trials

Fibrosis research: Advances and challenges

Drug Discovery World

JANUARY 24, 2024

Fibrocor is on track to complete all IND-enabling work to support IND filing and initiation of clinical trials in 2024. PW: The big challenge with fibrotic diseases is that they evolve slowly and are diagnosed late in the disease process. Can you elaborate on the clinical timeline for these small molecules?

Small Molecule

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Preparing for new-era drug modalities with technology

Drug Discovery World

OCTOBER 13, 2022

Prior to the pandemic, messenger RNA (mRNA) drug development was niche and predominantly pointed at different disease targets, including infectious diseases and cancers. mRNA presents versatility plus flexibility to target a broader range of diseases. . billion by 2026 9. CDMOs adapting fast .

Vaccine

Vaccine Therapies Drugs RNA

Scaling up: The need for human cells to give better access to cell therapies

Drug Discovery World

FEBRUARY 26, 2024

Cell therapies have emerged as transformative and potentially curative treatments across a range of disease areas. She and bit.bio are going beyond this cell type and looking at producing other cell types which may provide answers to a range of diseases beyond blood cancers. 2020 May;72(5):877-884.

Therapies

Therapies Treatment Engineering Disease

Models of Life

Codon

SEPTEMBER 29, 2024

The earliest ones relied on simple linear regression and attempted to correlate genetic variations with observable traits or disease risks — such as drug metabolization rates or cancer susceptibility. The average pass rate of phase I trials went up by 5 percent. 2024 Statistical models of organisms have existed for decades.

Engineering

Engineering Virus DNA Therapies

GSK to acquire clinical-stage biopharmaceutical company Affinivax, Inc.

The Pharma Data

JUNE 2, 2022

Pneumococcal disease includes pneumonia, meningitis, bloodstream infections, and milder diseases such as sinusitis and otitis media and remains a significant unmet medical need despite the availability of current pneumococcal vaccines. .” pneumoniae invasive disease and pneumonia in adults 50 years and above.

Vaccine

Vaccine Immune Response Clinical Trials Disease

GSK to acquire clinical-stage biopharmaceutical company Affinivax, Inc.

The Pharma Data

MAY 31, 2022

Pneumococcal disease includes pneumonia, meningitis, bloodstream infections, and milder diseases such as sinusitis and otitis media and remains a significant unmet medical need despite the availability of current pneumococcal vaccines. pneumoniae invasive disease and pneumonia in adults 50 years and above.

Vaccine

Vaccine Immune Response Clinical Trials Disease

Analysis Life Sciences Thank You 166 guidance documents the FDA is actively working on in 2024 (and beyond)

Agency IQ

FEBRUARY 15, 2024

If you’re looking for something specific, we recommend using the search function in your browser (Ctrl-F) to look for keywords of interest.

FDA

FDA Science Biosimilars Production

BridgeBio Pharma, Inc. Prices Upsized Offering of $650 Million Convertible Senior Notes

The Pharma Data

JANUARY 25, 2021

The Company may not redeem the notes prior to February 6, 2026. BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. The initial conversion price of the notes represents a premium of approximately 47.5%

Marketing

Marketing Disease Clinical Trials Production

Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas

The Pharma Data

SEPTEMBER 15, 2020

27 key programs highlighted, including assets that could potentially contribute revenue by 2025 and others in the 2026-2028 time frame. Major revenue contributions through 2025 anticipated from Oncology, Vaccines, Rare Disease and Inflammation and Immunology. BNT162 mRNA-based Vaccine Program. Protease Inhibitor Program.

Vaccine

Vaccine Therapies Trials Clinical Trials

Article EMA Thank You What will the orphan drug market exclusivity haircut mean for industry?

Agency IQ

JUNE 9, 2023

The problems: Although the current regulation stimulated orphan drug research, it didn’t do enough The regulation fostered development of medicines for rare diseases since implementation, but not enough. However, 95% of the over 6,000 recognized orphan diseases still have no treatment options. countries examined between 2010 and 2017.

Marketing

Marketing Biosimilars Regulations Drugs

Nordic Nanovector ASA: Results for the Third Quarter 2020

The Pharma Data

NOVEMBER 18, 2020

. Approval of amendments to PARADIGME protocol is proceeding as planned and completed in the best-recruiting countries Designed to enlarge the eligible patient population and increase the rate of enrolment into the trial. 59 patients enrolled as of 18 November 2020. Financial Highlights.

Trials

Trials Clinical Supply Production Therapies

Advances in neuroscience drug discovery

Drug Discovery World

FEBRUARY 22, 2024

Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In With the breadth and range of central nervous system disorders – for example Parkinson’s disease and multiple sclerosis – facing the global population there is an unmet need within this area of drug discovery.

Drugs

Drugs Clinical Trials Disease Therapies

Sequana Medical Appoints Two Additional Experts as Heart Failure Scientific Advisors

The Pharma Data

OCTOBER 14, 2020

15, 2020 (GLOBE NEWSWIRE) — Sequana Medical NV ( Euronext Brussels: SEQUA ) , an innovator in the management of fluid overload in liver disease, malignant ascites and heart failure, today announces the appointments of Dr. Michael Felker and Dr. James Udelson as new Heart Failure Scientific Advisors. and EU5 by 2026.

Clinical Trials

Clinical Trials Clinical Research Therapies Marketing

Analysis Life Sciences Thank You A deep dive into MDR and IVDR implementation: Where we are and where we’re going

Agency IQ

AUGUST 18, 2023

Otherwise, EUDAMED has been proceeding with finishing testing on the remaining modules in order to complete the minimum viable product (MVP) for the complete database by the end of the year, for a database audit in Q1/Q2 2024 and all modules mandatory to use by Q2 2026. We haven’t seen these revisions come through yet.

Science

Science Regulations Laboratories Production

Article FDA Thank You FDA updates set the stage for broader use of harmonized standards for safety reporting

Agency IQ

APRIL 8, 2024

In 2012, the passage of the FDA Safety and Innovation Act (FDASIA) created a new requirement for sponsors to submit all drug submissions, including clinical trial applications, in an electronic format as specified by FDA guidance. database-to-database submissions).

FDA

FDA Production Regulations Vaccine

Article FDA Thank You Diagnostics landscape analysis: Key issues and a look ahead at 2024

Agency IQ

FEBRUARY 2, 2024

BY LAURA DIANGELO, MPH | JAN 30, 2024 10:25 AM CST Diagnostic regulation: A quick overview Diagnostics products are those intended to identify, signal or detect a specific health condition, infection or disease, or monitor a person’s health or health status. In-house tests (similar to LDTs in the U.S.,

FDA

FDA Regulations Laboratories Production

How Recent Drug Pricing Laws Impact Drug Discovery (H.R. 5376)

Drug Hunter

APRIL 24, 2023

More older adults will also benefit from drug hunters’ previous pursuits to cure cancer, multiple sclerosis, and other serious diseases due to lower out of pocket maximums for Medicare beneficiaries. III registrational trial, significant new investment is triggered to expand the medicine into new indications. For perspective, 4.1

Small Molecule

Small Molecule Biosimilars Drugs Vaccine

Drug Discovery Digest

Drug candidate for ALS patients authorised for Phase II clinical trial

Next steps for the CALYX trial for cerebral Adrenoleukodystrophy

Trending Sources

Study finds dual receptor beta-agonist promotes healthy weight loss

Manchester to get £60m development to enhance lab space

Could GLP-1 receptor agonists treat addiction and dementia?

Antibody Drug Conjugates: windows of opportunity

Novartis Entresto receives positive CHMP opinion for pediatric heart failure

The trends driving ELRIG Drug Discovery 2022

From cradle to grave: Novo Nordisk Foundation and cardiometabolic diseases

Understanding the Impact of the New EU Artificial Intelligence Act on Clinical Research

The challenges and trends of cell & gene therapies

Ionis highlights achievements, commercial strategy and technology advancements at Investor Day

GSK reaches agreement to acquire late-stage biopharmaceutical company Sierra Oncology for $1.9bn

Article FDA Thank You What’s on FDA’s new Diagnostics Chief’s mind? Transitions, the LDT rule and cybersecurity

Analysis Life Sciences Thank You What We Expect the FDA to do in August and September 2024

Sandoz strengthens pipeline expansion through partnership to develop and manufacture multiple biosimilars

Five cell and gene therapy advances in neurodegeneration

Fibrosis research: Advances and challenges

Preparing for new-era drug modalities with technology

Scaling up: The need for human cells to give better access to cell therapies

Models of Life

GSK to acquire clinical-stage biopharmaceutical company Affinivax, Inc.

GSK to acquire clinical-stage biopharmaceutical company Affinivax, Inc.

Analysis Life Sciences Thank You 166 guidance documents the FDA is actively working on in 2024 (and beyond)

BridgeBio Pharma, Inc. Prices Upsized Offering of $650 Million Convertible Senior Notes

Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas

Article EMA Thank You What will the orphan drug market exclusivity haircut mean for industry?

Nordic Nanovector ASA: Results for the Third Quarter 2020

Advances in neuroscience drug discovery

Sequana Medical Appoints Two Additional Experts as Heart Failure Scientific Advisors

Analysis Life Sciences Thank You A deep dive into MDR and IVDR implementation: Where we are and where we’re going

Article FDA Thank You FDA updates set the stage for broader use of harmonized standards for safety reporting

Article FDA Thank You Diagnostics landscape analysis: Key issues and a look ahead at 2024

How Recent Drug Pricing Laws Impact Drug Discovery (H.R. 5376)

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