FDA Law Blog: Drug Discovery

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” A considerable roadblock in the development of RMT products is a lack of regulatory predictability.

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The Pharma Data

FEBRUARY 24, 2022

Bayer announced today the initiation of the CONFIDENCE study, a Phase II, three-arm study that will investigate simultaneous initial combination therapy with finerenone and the SGLT2 inhibitor empagliflozin, compared with finerenone alone and empagliflozin alone respectively in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D).

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The Pharma Data

OCTOBER 21, 2020

and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinical development programme. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.

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FDA Law Blog: Drug Discovery

JANUARY 4, 2023

Brevig, Senior Regulatory Device and Biologics Expert — On December 7, 2022, FDA’s Center for Biologics Evaluation and Research (CBER) and the Office of Tissues and Advanced Therapies (OTAT) held a town hall to answer questions related to cell therapy and tissue-engineered products chemistry, manufacturing, and contr­­ols (CMC).

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Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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The Pharma Data

MARCH 23, 2022

Baynes, head of Global Clinical Development (GCD) and Chief Medical Officer, Merck Research Laboratories (MRL), will be retiring from Merck in July. He also led the expansion of Merck’s research and development in China and Japan. “It Eliav Barr, senior vice president, Global Clinical Development.

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Agency IQ

APRIL 12, 2024

Draft guidance on potency assays for CGT products garners extensive stakeholder input Late last year, the FDA published a draft update to its 2011 guidance on potency assays for cell and gene therapy products, unveiling a major shift in approach to the issue. How does “potency” apply to cell and gene therapy (CGT) products?

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The Pharma Data

NOVEMBER 29, 2020

Prior to joining Gannex, Dr. Palmer was Head of Liver Disease Clinical Development at Takeda Pharmaceuticals. She has also held senior leadership positions at Shire Pharmaceuticals and Kadmon Corporation, where she was in charge of the global development of NASH and other liver disease programs. . ” About Ascletis.

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Drug Target Review

OCTOBER 16, 2024

It’s an exciting process—anticipating development risks, predicting outcomes, and aligning these with the greater vision of medical advancement. The true joy comes from navigating these complexities and asking the right questions early on, because these foundational steps are critical to the success of any clinical development process.

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The Pharma Data

SEPTEMBER 29, 2021

Patients are going to be randomized to receive either finerenone 10mg or 20mg or placebo on top of individually tolerated maximum labeled doses of a renin-angiotensin system (RAS)-blocking therapy like an angiotensin-converting enzyme (ACE) inhibitor or an angiotensin II receptor blocker (ARB). In the U.S.,

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Agency IQ

FEBRUARY 12, 2024

BY RACHEL COE, MSC | FEB 7, 2024 10:21 PM CST The Bespoke Gene Therapy Consortium A collaborative effort, the Bespoke Gene Therapy Consortium (BGTC) was launched in October 2021 to “accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease.”

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Drug Target Review

OCTOBER 10, 2024

What are the risks of over-reliance on healthy donor material in early-stage research, and how can decision-makers ensure a balanced integration of diseased material to avoid potential gaps when transitioning to clinical trials? This can lead to low cell yields, poor product quality, and process variability.

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The Pharma Data

DECEMBER 22, 2020

Merck will assist with preclinical development and has the option to exclusively develop, manufacture and commercialize the candidate following Phase 1. Under the agreement A2 will receive an upfront payment, and will be eligible for opt-in and milestone payments, plus royalties on sales of any approved product.

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PPD

MARCH 26, 2024

A large majority of drug developers utilize FSP or hybrid FSP/FSO models, half are “heavy users” Heavy users: Those that use FSP or hybrid FSP/FSO models for most of their clinical development outsourcing. Heavy users leverage these models for nearly three-quarters of their outsourcing.

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DrugBank

SEPTEMBER 26, 2024

This reduction in cost does not equate to a compromise in quality or efficacy, as biosimilars undergo rigorous analytical and functional characterization to demonstrate biosimilarity to the reference product. biosimilars are often priced slightly lower than their reference products, limiting their potential for cost savings.

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The Pharma Data

JANUARY 12, 2021

Working alongside KSQ will facilitate smart drug discovery and development of what we hope will be transformative new therapies for patients with intractable forms of cancer.”. KSQ is also eligible to receive tiered royalties on net sales of each approved product. sales for that product. sales for that product.

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The Pharma Data

JANUARY 17, 2021

LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. The new business will continue to develop Ixaka’s proprietary technologies – concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics.

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The Pharma Data

JUNE 2, 2022

Boehringer Ingelheim and the Agency for Science, Technology and Research (A*STAR) today announced a global licensing agreement under which Boehringer Ingelheim will obtain exclusive worldwide rights to research, develop and commercialize products based on a panel of innovative, tumor-specific antibodies from A*STAR.

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Drug Target Review

JANUARY 8, 2024

Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.

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The Pharma Data

DECEMBER 13, 2020

NRG1 fusions are a rare genetic mutation that are increasingly recognized as a driver of multiple tumor malignancies, and an actionable target for HER3 targeted therapy. Up to 1% of all solid tumors harbor NRG1 fusions, therefore, it is important to identify this patient population and develop therapies that can treat them. [1].

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Vial

APRIL 24, 2024

In addition to the areas above (aside from DTx), Syneos Health offers biosimilars , cell and gene therapy , endocrine and metabolic, immunology and inflammation, infectious diseases, pediatrics, respiratory, and women’s health services. Digital therapeutics is not explicitly listed as a therapeutic area of expertise for Syneos Health.

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PPD

OCTOBER 2, 2024

To support these functions, PPD™ Functional Service Partnership (FSP) Clinical Operations solutions harness the full range of innovative technologies, applying extensive skill and expertise to empower our customers to bring therapies to market quickly and within budget.

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The Pharma Data

SEPTEMBER 11, 2021

– Designation is based on results from the EMPEROR-Preserved phase III trial, which established Jardiance as the first therapy to show statistically significant improvement in heart failure outcomes in adults with heart failure with preserved ejection fraction. vice president, Product Development, Lilly.

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The Pharma Data

JANUARY 13, 2021

This is our sixth global approval for an internally-developed product, and our first approval for tislelizumab in a lung cancer indication, an area where we believe tislelizumab can have a large impact for patients.”. We also market or plan to market in China additional oncology products licensed from Amgen Inc.,

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Alta Sciences

OCTOBER 30, 2023

TOP NONCLINICAL SCIENTIFIC RESOURCES eBook : Safety Assessment for Ophthalmic Products Designing preclinical studies for ocular therapies take a lot of deliberation. The Altascientist : Issue No. Read or listen now. Watch the video.

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The Pharma Data

DECEMBER 2, 2020

Based on the Phase 2 study findings to date, marstacimab may have the potential to offer improved bleed control via subcutaneous injection and potentially eliminate the need for prophylactic factor replacement, providing an enhanced treatment option compared to factor replacement therapy.”. About Pfizer Rare Disease. Pfizer Inc.:

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Cytel

FEBRUARY 26, 2024

Adler, MPA, Director, Global Product Engagement, and Valeria Mazzanti, MPH, Associate Director, Customer Success In our previous post , we provided an overview of some of the challenges in studying rare disease therapies, as well as some of the statistical methods and design types that can be deployed to mitigate these challenges.

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Drug Target Review

OCTOBER 11, 2023

1,2 Many, however, do have dysfunctional proteins at their core, and for these diseases, a new option is being explored: autophagy-boosting therapies. Gene therapy, of course, also holds great promise. Most of these conditions are genetic in origin and the majority have no effective treatment.

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The Pharma Data

JUNE 25, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for lecanemab (BAN2401), an investigational anti-amyloid beta (A?) Breakthrough Therapy designation is an FDA program intended to expedite the development and review of medicines for serious or life-threatening conditions. About Eisai Co.,

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The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

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Drug Target Review

JUNE 3, 2024

This process results in anergy, deletion, production of regulatory T cells and downregulation of the antigen-specific T cell response. Could you explain the development of CNP-108 for T cell immunogenicity in gene therapy, and how it induces the immune system to build a tolerance to gene therapy treatments?

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The Pharma Data

APRIL 27, 2023

It is also an off-the-shelf therapy, meaning that people do not have to wait for cell collection and genetic engineering before starting treatment, which could be particularly important for patients who are at a high-risk of their disease progressing. “New Roche’s Chief Medical Officer and Head of Global Product Development.

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SugarCone Biotech

JUNE 10, 2024

[link] Immunity is complex and can be dangerous when exploited clinically, as demonstrated by the lethal administration of TNF, or anti-CD40L antibody (Biogen) or CAR-T cells expressing the CD16 Fc-receptor (Unum), among many other examples. This is the T cell type most closely associated with anti-tumor immune responses. 2023.09.011 ).

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Precision for Medicine

APRIL 7, 2023

Our new state-of-the-art laboratory, combined with the deep knowledge of our scientists, gives an advantage to clients developing novel therapeutics and diagnostic products in oncology, liver disease, and skin diseases. This service accelerates the validation of these products and helps shorten the time for regulatory approvals.

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The Pharma Data

MAY 26, 2023

Pfizer Presents Scientific Advancements from its Leading Oncology Portfolio at ASCO 2023 Annual Meeting will present data across its Oncology portfolio and growing pipeline, covering multiple tumor types and novel mechanisms of action at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago from June 2 through June 6.

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The Pharma Data

APRIL 27, 2022

We plan to discuss these data with health authorities, and we will continue to expand our tislelizumab clinical development program in pursuit of novel, synergistic combinations with the ultimate goal of extending survival for more patients.”. About 108,000 people of more than 140 nationalities work at Novartis around the world.

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The Pharma Data

MARCH 23, 2023

The new Phase III clinical study, ARASTEP, will investigate the efficacy of darolutamide plus androgen deprivation therapy (ADT) versus ADT alone in hormone-sensitive prostate cancer, in patients with high-risk biochemical recurrence (BCR) who have no evidence of metastatic disease by conventional imaging and a positive PSMA PET/CT at baseline.

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