New Drug Approvals

APRIL 11, 2025

1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1]

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Drug Target Review

OCTOBER 30, 2024

Imagine being able to create an in vitro replica of a diseased organ to study the molecular mechanism underlying the illness. Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first.

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Conversations in Drug Development Trends

MAY 14, 2024

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.

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Drug Patent Watch

DECEMBER 9, 2024

In the vast realm of pharmaceutical research and development, there’s a fascinating intersection between ancient wisdom and modern science. “Pharmacognosy is the bridge between traditional medicine and modern pharmaceutical science, offering a treasure trove of potential new drugs waiting to be discovered.”

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DrugBank

OCTOBER 4, 2024

Deal Structuring Deal structuring in pharmaceutical M&A requires a balance of financial, legal, and strategic considerations. Intellectual Property Valuation Valuing intellectual property (IP) assets is a critical component of pharmaceutical M&A, as these assets often represent a significant portion of a company's value.

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Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.

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PPD

OCTOBER 31, 2024

As the pharmaceutical industry continues to evolve, drug developers encounter new challenges and opportunities in their pursuit of innovation. From adapting to complex new trial designs to embracing cutting-edge technologies, staying ahead requires a deep understanding of the current landscape.

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Broad Institute

DECEMBER 18, 2023

Brain disorders are difficult to study and many drug candidates have failed in clinical trials, causing pharmaceutical companies to reduce their investments or even exit the field entirely. But a new path for bringing treatments to patients is starting to emerge. The following conversation was edited for length and clarity.

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LifeSciVC

JULY 17, 2024

By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets.

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Drug Target Review

MARCH 4, 2025

A personalised approach to obesity treatment Traditional obesity treatments have often followed a one-size-fits-all model, despite mounting evidence that obesity is not a singular disease but rather a collection of subtypes. This growing health challenge increases the risk of diabetes, heart disease, and other conditions.

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The Pharma Data

AUGUST 30, 2021

Positive high-level results from the FoCus Phase III trial in Wilson disease showed ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilisation from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments. Wilson disease. 1, 2, 3.

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Disease Trials Therapies Treatment 52

Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

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Drug Target Review

SEPTEMBER 9, 2024

functions to reset the immune system and potentially provide remission for allergic diseases? In allergic diseases, APCs identify and present unknown antigens to the immune system. This can lead to inadequate long-term effectiveness with low rates of disease remission. They also require frequent administration.

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Disease Immune Response Clinical Trials Treatment 64

Drug Target Review

JUNE 5, 2024

How do you prioritise which therapeutic areas or diseases to focus on in your drug discovery efforts? We have several international trials ongoing or being initiated in a range of indications. About the author James Graham, CEO of Recce Pharmaceuticals Recce Pharmaceuticals.

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Drug Target Review

JULY 4, 2023

In practice, some patients may be left managing a syndrome associated with the therapy along with the disease. 2 However, when dosed at the MTD, ADCs display improved efficacy over small molecules in oncology trials. 2 However, when dosed at the MTD, ADCs display improved efficacy over small molecules in oncology trials.

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The Pharma Data

JANUARY 10, 2021

Dr. Ken Kobayashi, CMO, ASLAN Pharmaceuticals, commented: “The emerging safety profile of ASLAN004 has allowed us to move directly into the expansion cohort at the highest dose, 600mg weekly, based on blinded safety data, accelerating the completion of the study. SINGAPORE, Jan. Media and IR contacts. Forward looking statements.

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National Institute on Drug Abuse: Nora's Blog

MARCH 6, 2025

Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&

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Conversations in Drug Development Trends

MAY 14, 2024

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.

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Disease Clinical Trials Trials Pharmaceutical Companies 52

PPD

SEPTEMBER 16, 2024

The pharmaceutical industry is currently experiencing a significant transformation. Real-world data paired with machine learning is a game changer in drug development RWD is becoming increasingly important in influencing the drug development landscape, particularly when used to develop the natural history and patient journey through disease.

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H1 Blog

DECEMBER 4, 2023

As the pharmaceutical and biotech industries continue to push for faster drug development, the importance of equity and diversity in clinical trial recruitment cannot be overlooked. Data is a powerful tool in identifying and reaching vulnerable populations for clinical trials.

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DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

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Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

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The Pharma Data

DECEMBER 29, 2020

Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy. galactosidase-A product candidate under development for the treatment of Fabry disease. CARMIEL, Israel and BOSTON , Dec. 30, 2020 /PRNewswire/ — Protalix BioTherapeutics, Inc. Male patients improved from -6.36

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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The Pharma Data

JANUARY 12, 2021

13, 2021 /PRNewswire/ — Adastra Pharmaceuticals, Inc., 13, 2021 /PRNewswire/ — Adastra Pharmaceuticals, Inc., The trial investigated the safety and efficacy of ZTR, in combination with temozolomide (TMZ), in patients with recurrent high-grade gliomas. NCI’s Principal Investigator of the 17-C-0009 trial.

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Clinical Trials Trials Treatment Pharmaceuticals 52

Drug Target Review

AUGUST 6, 2024

A number of areas represent significant opportunities for Antibody Drug Conjugates (ADCs) beyond oncology, leveraging their ability to deliver therapeutic agents specifically to diseased cells or tissues while minimising off-target effects. Oncology also offers high returns and the opportunity to address severe, life-threatening conditions.

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PPD

DECEMBER 16, 2024

Cost and complexity go hand-in-hand The rising costs and growing complexity in clinical trials are deeply linked, with patient recruitment, extended timelines and meeting regulatory demands emerging as some of the key drivers. Nearly half (49%) of clinical trial sponsors surveyed identified rising costs as their foremost concern in 2024.

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Drug Target Review

NOVEMBER 25, 2024

At this point, I knew that I wanted to join the biotech and pharmaceutical industry, so I moved to a small biotech company in London, where I spent three to four years learning the fundamentals of the biotech industry. Additionally, we are preparing to launch several other trials over the coming months.

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Treatment Small Molecule Clinical Trials Trials 52

Vial

JUNE 24, 2024

Contract research organizations (CROs) and pharmaceutical companies can leverage these cutting-edge technologies to streamline clinical trials and introduce automation in drug discovery. As clinical trials grow in complexity, the volume of data being gathered and utilized for these studies is expanding.

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The Pharma Data

JANUARY 13, 2021

“Receiving Fast Track designation is an important acknowledgment of the results of our COVID-19 laboratory research,” commented Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. Alerts Sign-up for Innovation Pharmaceuticals email alerts is available at: [link].

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Clinical Trials Trials Treatment Pharmaceuticals 52

The Pharma Data

OCTOBER 9, 2021

This designation is predicated on data showing that gantenerumab significantly reduced brain amyloid pillar, a pathological hallmark of Notice, in the ongoing SCarlet RoAD and Marguerite RoAD open- tag extension trials, as well as other studies. Roche’s Chief Medical Officer and Head of Global Product Development.

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Disease FDA Therapies Trials 52

The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

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Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

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Vial

MARCH 25, 2024

Introduction Worldwide Clinical Trials vs. Vial. Worldwide Clinical Trials is a mid-size, full-service global contract research organization (CRO) that works with biotech and pharma to advance new medications. With more than 30 years of experience in cardiovascular research, Worldwide has global trial experience in all phases.

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Drug Target Review

MARCH 17, 2025

Advancing drug candidates across key therapeutic areas Dr John Donello brings over 25 years of experience in pharmaceutical drug discovery, development and collaborations. Hopes for the Phase IIb trial Donello discusses the key outcomes he hopes to see in the Phase IIb VITALIZE study of zelquistinel for major depressive disorder (MDD).

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Treatment Licensing Licensing Science 52

Advarra

MARCH 6, 2025

For more than 35 years, Advarra has been committed to protecting the rights and welfare of clinical trial participants while helping to improve healthcare outcomes, advancing medical knowledge, and bringing innovative, life-extending treatments to market that benefit millions of patients worldwide.

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Clinical Research Research Clinical Trials Bioethics 52

DrugBank

JUNE 27, 2024

Currently, about one-third of recent drug approvals and 25% of the pharmaceutical industry's annual revenue is coming from repurposed drugs.This trend is being championed industry-wide, by public organizations and nonprofits, with numerous small companies diving in and academic publications on the subject showing great popularity.  

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