The Pharma Data

JUNE 12, 2025

The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.

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Drug Target Review

JULY 1, 2025

Clinical trials are expensive, slow and often limited by outdated design constraints. Placebo arms, in particular, create ethical and logistical hurdles, especially in areas like rare disease and oncology. They offer patient-specific outcome predictions, generated using machine learning models trained on real historical clinical data.

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Covalent Modifiers

MAY 9, 2024

4c00220 Multiple sclerosis (MS) is a chronic disease with an underlying pathology characterized by inflammation-driven neuronal loss, axonal injury, and demyelination. George Vandeveer, Ti Wang, Zain Yousaf, Christopher J. Helal, and Brian T. Hopkins Journal of Medicinal Chemistry 2024 DOI: 10.1021/acs.jmedchem.4c00220

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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.

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The Pharma Data

JUNE 6, 2025

These new findings reinforce the company’s growing leadership in rare hematologic diseases, with a focus on transformative treatments for immune thrombocytopenia (ITP) and hemophilia. These designations underscore rilzabrutinib’s potential to offer a meaningful therapeutic advance for patients with limited treatment options.

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Cytel

JANUARY 10, 2024

A clinical development strategy is a comprehensive plan designed to establish the safety and efficacy of new therapeutics. Developing an effective plan requires multidisciplinary expertise and adapting to accumulating learning and changes in clinical practice and the market environment.

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The Pharma Data

JUNE 11, 2025

Dapirolizumab Pegol Demonstrates Promise in Phase 3 SLE Trial, Showing Improvements in Fatigue and Disease Activity at EULAR 2025 UCB (Euronext Brussels: UCB) and Biogen Inc. The latest data from the PHOENYCS GO trial reinforce the potential of dapirolizumab pegol to improve disease outcomes in people with moderate-to-severe SLE.

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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 12, 2025

The data, presented at the annual European Alliance of Associations for Rheumatology (EULAR 2025) congress in Barcelona, Spain, show that dapirolizumab pegol resulted in significant and clinically meaningful improvements in disease activity while addressing a major symptom that profoundly impacts patients’ daily lives — fatigue.

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PPD

JANUARY 15, 2025

As clinical trials become increasingly complex, particularly in decentralized trials and rare disease studies, sponsors experience increased challenges in site selection, forecasting and resourcing, and patient recruitment and enrollment.

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Drug Hunter

JULY 21, 2023

semaglutide/Ozempic), this month features a pair of ultra-hot non-peptide oral GLP-1R modulators in clinical development. With the popular success of diabetes and weight-loss peptide drugs from Novo (e.g. Sign up for our free weekly newsletter here to get more content like this straight to your inbox.

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Conversations in Drug Development Trends

NOVEMBER 21, 2023

Since the advent of modern medicine, the scientific community has developed more than 3,000 drugs for thousands of diseases. Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. There are so many patients suffering from diseases that have no treatments.

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Cytel

MARCH 11, 2024

In 2023, rare diseases accounted for 30% of product pipeline under development, about half of which comprising non-oncology rare diseases. Clinical development in rare diseases has specific challenges.

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SugarCone Biotech

JUNE 19, 2025

Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). For these patients, clinical onset means severe symptoms and a rapid decline in overall health. The tarlatamab results have of course triggered a stampede of development of DLL3-targeted therapies.

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Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.

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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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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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ASPET

FEBRUARY 26, 2024

Significance Statement FXR (farnesoid X receptor) agonists have shown promise in treating non-alcoholic steatohepatitis and other liver diseases in the clinic, but balancing efficacy with undesired side effects has been difficult. Cilofexor is one of few remaining FXR agonists in clinical development.

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PPD

NOVEMBER 21, 2023

To keep a pulse on this rapidly changing industry, the PPD clinical research business of Thermo Fisher Scientific surveyed 150 leaders in the biopharma and biotech industries for the second consecutive year. Differences in size, resources and agility have led the two groups to take different approaches to clinical development.

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PPD

MARCH 11, 2025

While individual rare diseases affect populations that are small in numbers, collectively they impact millions globally, posing significant health and research challenges. Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development.

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The Pharma Data

JUNE 18, 2025

AMVUTTRA’s approval has not only underscored the therapeutic potential of RNAi-based medicines but also signaled Alnylam’s readiness to scale its capabilities and deepen its impact across a wider range of disease areas. As we begin this exciting new chapter, I am energized about what lies ahead for Alnylam R&D and for patients,” he said.

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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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Cytel

MAY 28, 2024

Written by Susannah Sadler and Stacy Grieve Launching a treatment for inflammatory diseases can be a make-or-break endeavor. Here we describe three case study examples of common issues faced in the inflammatory disease space and how these challenges were overcome using novel solutions, ultimately leading sponsors to submission success.

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The Pharma Data

MAY 8, 2021

Sandoz, a Novartis division, today announced progress in the late-stage clinical development program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. About aflibercept.

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Drug Target Review

DECEMBER 13, 2023

Combinatorial analytics approaches identify combinations of features that together are associated with the disease phenotype in patient sub-groups, capturing the non-linear effects of interactions between multiple genes. The combinatorial approach is considerably more sensitive than GWAS and requires much smaller patient populations.

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Cytel

FEBRUARY 13, 2024

Adler, MPA, Director, Global Product Engagement, and Valeria Mazzanti, MPH, Associate Director, Customer Success Rare disease studies come with their own unique challenges, particularly limited patient populations. Written by Boaz N.

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Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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KIF1A

MARCH 16, 2024

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Like hereditary spastic paraplegia, CMT is an umbrella disorder, associated with mutations in over 140 genes, but many diagnosed patients do not know their disease-causing mutation.

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Cytel

JANUARY 17, 2024

Despite accumulating learnings from early phases, several uncertainties remain to be addressed when designing pivotal trials. Adaptive trials can help mitigate uncertainties; however, the trade-offs and their impact differ in the confirmatory setting.

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Cytel

APRIL 10, 2024

Orphan drug designation is a regulatory status granted to pharmaceuticals developed for the treatment of rare diseases. It provides incentives to encourage the research, development, and approval of therapies targeting small patient populations.

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KIF1A

SEPTEMBER 30, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A-Related Research In the rare disease space, a common and straightforward question is: How rare is the disease?

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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

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

JULY 2, 2025

The decision follows an analysis of topline data from a Phase 1b clinical study, which demonstrated encouraging safety and efficacy results in patients with DOK7-CMS, one of the more severe and prevalent subtypes of this ultra-rare neuromuscular disorder. Peter Ulrichts, Ph.D., Peter Ulrichts, Ph.D., per 1 million.

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The Pharma Data

JUNE 18, 2025

While localized prostate cancer boasts a five-year survival rate above 90% , that figure drops precipitously to approximately 31% for metastatic disease. Building on Early Clinical Success The Phase 3 IDeate-Prostate01 trial is based on encouraging results from the earlier IDeate-PanTumor01 Phase 1/2 study.

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Conversations in Drug Development Trends

APRIL 5, 2024

For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.

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Drug Target Review

SEPTEMBER 19, 2024

Based on Nobel Prize-winning research, IRLAB has grown rapidly to become recognised and respected as a world-leader in understanding the complex neuropharmacology of CNS disorders, especially Parkinson’s disease (PD). The third project in clinical phase, IRL757, is in Phase I and is being developed for the treatment of apathy.

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