FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field. In October, FDA announced seven new clinical trial grants awarded in fiscal year (FY) 2024 – including one for a Phase 3 trial – totaling $17.2

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Chemical Biology and Drug Design

JUNE 19, 2025

The presence of thienopyrimidine derivatives in several FDA-approved drugs and clinical trial candidates underscores their therapeutic potential and safety profile. As our understanding of their structure–function relationships deepens, we can expect further advancements in the development of thienopyrimidine-based therapies.

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Drug Target Review

APRIL 30, 2025

In the rapidly evolving field of cell and gene therapy (CGT), the ability to manage complex biological data, optimise manufacturing processes, and accelerate drug discovery is crucial. How AI is solving CGTs biggest challenges Cell and gene therapy is inherently complex. The result? Faster time-to-market and reduced costs.

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Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome.

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PPD

NOVEMBER 11, 2024

As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients. Accelerate your drug development and clinical trial goals and benefit from our 360° CDMO and CRO solutions and expertise.

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Drug Target Review

JUNE 9, 2025

“This experience underscored the gaps in rare disease innovation, motivating me to lead Alltrna, a company pioneering engineered tRNA therapies to address a broad range of genetic diseases in a profound way unlike any other genetic modality.” While commonly used in oncology, the basket trial approach is novel for rare genetic diseases.

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PPD

OCTOBER 31, 2024

From adapting to complex new trial designs to embracing cutting-edge technologies, staying ahead requires a deep understanding of the current landscape. Drug development The rising cost of clinical trials is the top challenge this year, due to increasingly complex protocol designs and difficult patient recruitment.

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New Drug Approvals

JULY 5, 2025

Clinical Trials: Palazestrant is currently in clinical trials, including Phase 1/2 and Phase 3 studies, for the treatment of ER+, HER2- metastatic breast cancer. Combination Therapy: Palazestrant is being evaluated in combination with other drugs like CDK4/6 inhibitors (e.g., ribociclib).

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Quanticate

JUNE 29, 2025

The development of oncology drugs is a complex, multi-phase process, where safety, efficacy, and optimal dosing are determined progressively through clinical trials. To ascertain the RP2D effectively, Phase I trials are often split into two critical sub-phases, dose escalation and dose expansion.

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The Pharma Data

JUNE 18, 2025

The investigational ADC is built using Daiichi Sankyo’s proprietary DXd technology and represents one of the newest generation of targeted cancer therapies. Addressing a Pressing Clinical Need in Prostate Cancer Prostate cancer remains one of the most prevalent malignancies among men worldwide.

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The Pharma Data

JUNE 23, 2025

Study Overview and Population The HYPERION study was designed to assess the efficacy and safety of WINREVAIR when used in combination with standard background therapy, compared to placebo, in adult patients recently diagnosed with pulmonary arterial hypertension. It follows two earlier landmark studies: STELLAR and ZENITH.

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Drug Target Review

JUNE 6, 2025

At the forefront of this transformation is Dr Ebrahim Delpassand, a nuclear medicine physician and the driving force behind RadioMedix (RMX), a radiopharmaceutical company focused on developing targeted diagnostics and therapies. Why targeted radiopharmaceuticals? The SPICA Center was built to address this gap.”

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Drug Target Review

JULY 3, 2025

It is becoming increasingly evident that generative artificial intelligence (GenAI) is a resourceful tool for helping pharmaceutical companies reduce manual tasks required by clinical trials. Because LLMs are trained on extensive, internet-scale datasets, they can learn to identify contexts linking words and language.

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The Pharma Data

JUNE 26, 2025

FDA Approves Streamlined Monitoring Requirements and REMS Program Removal for Bristol Myers Squibb’s CAR T Cell Therapies Breyanzi and Abecma, Marking Milestone Toward Expanding Access to Cancer Treatment In a significant regulatory development, Bristol Myers Squibb announced that the U.S.

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Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. Highlighting data integration.

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Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Operationalizing these trials requires proactive and flawless management at every stage.

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New Drug Approvals

JUNE 24, 2025

Clinical Trials: Phase 3 clinical trials have shown that nerandomilast can slow lung function decline in patients with IPF and PPF. Efficacy: The trials demonstrated that nerandomilast led to a smaller decline in forced vital capacity (FVC), a measure of lung function, compared to placebo.

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The Pharma Data

JUNE 27, 2025

The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options. The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options.

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PPD

MARCH 11, 2025

Breaking through research barriers Challenge #1: Small groups of patients Rare diseases impact a small number of individuals, making it difficult to recruit enough participants for clinical trials. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.

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SugarCone Biotech

JUNE 19, 2025

They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. Each target and each therapeutic modality induce varying degrees of clinical efficacy, as well as causing toxicities. This leads us to Amgen’s T cell-engager therapeutic, tarlatamab.

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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. To manage these costs, sponsors need to address the root contributors to trial complexity.

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Drug Target Review

MARCH 4, 2025

Knowing a patients subtype or phenotype can be used to guide obesity treatments of all kinds, including drug therapy, devices, bariatric surgery and even diet and lifestyle interventions, Bagnall explains. Without understanding these differences, treatment becomes costly and a game of trial and error, Bagnall points out.

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PPD

FEBRUARY 10, 2025

By harnessing the full range of innovative technologies and taking advantage of an FSP partners extensive skills and experience, sponsors are able to bring their therapies to market more quickly and within budget even in the face of complicated global regulations and widely fluctuating workloads.

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The Pharma Data

JUNE 22, 2025

The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C., The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C.,

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Conversations in Drug Development Trends

OCTOBER 14, 2024

Fortunately, advances in clinical research are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide Clinical Trials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.

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Drug Target Review

JULY 8, 2025

Her team sits right at the edge of innovation, where discovery science meets clinical execution. Not only are they chasing new targets, they are also working to understand the biology behind resistance, discover biomarkers that matter and build smarter therapies from the ground up.

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The Pharma Data

JUNE 25, 2025

Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.

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The Pharma Data

JUNE 23, 2025

These pivotal findings were unveiled at the 85th Scientific Sessions of the American Diabetes Association (ADA) and were concurrently published in The New England Journal of Medicine , underscoring the significant promise of MariTide in the growing field of anti-obesity therapies.

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The Pharma Data

JUNE 26, 2025

The encouraging data builds on the foundation established by earlier studies and further strengthens the clinical case for MR-141 as a potential non-invasive, once-daily ophthalmic solution for millions affected by the age-related decline in near vision.

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Drug Target Review

OCTOBER 30, 2024

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. 13-18 Confidence in organoid-generated data that translates in vitro efficacy to efficacy in the clinic is growing.

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The Pharma Data

JUNE 11, 2025

Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. This trial will build on previous positive data from the earlier Phase 1/2 HALOS study and is designed to rigorously assess the potential of ION582 as a disease-modifying therapy. “We

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Broad Institute

NOVEMBER 25, 2024

As a proof of concept, a drug designed to target a newly discovered biological node is showing efficacy in treating rare genetic diseases in the kidney, the eye, and the brain and is now making its way to clinical trials in collaboration with a pharmaceutical partner. You can learn more about how clinical trials work here.

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The Pharma Data

JUNE 6, 2025

Otsuka Unveils Promising Phase 3 Results for Sibeprenlimab in IgA Nephropathy, Marking Significant Proteinuria Reduction and Advancing a Novel APRIL-Targeted Therapy Otsuka Pharmaceutical Development & Commercialization, Inc., in those receiving placebo—further underscoring the tolerability of this investigational therapy.

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Drug Target Review

JUNE 19, 2025

Bazan sees it as part of a broader shift towards combination therapies – an approach that will likely define the next generation of pain management. South Rampart Pharma has completed its Phase 1 clinical trial, demonstrating strong safety, tolerability and pharmacokinetics. From bench to bedside: what is next?

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PPD

JANUARY 2, 2025

Its ability to maneuver persistent drug development challenges, like patient recruitment, trial complexity and rising costs, will ultimately determine its success. This blog explores the value of functional service provider (FSP) models and how they help biotech companies augment their clinical development needs.

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Drug Target Review

MAY 23, 2025

In the ever-evolving landscape of immuno-oncology, managing the side effects of advanced therapies has become just as important as enhancing their efficacy. Our goals are to progress our pipeline and position each asset for clinical and commercial success.

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The Pharma Data

JUNE 22, 2025

In the trial, participants were directly switched to Mim8 without a washout period. Strong Safety Profile Reinforces Mim8’s Potential Mim8’s safety profile was a central focus of the FRONTIER5 trial. Throughout the 26-week study period, the investigational therapy was well-tolerated.

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