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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The Pharma Data

JUNE 13, 2025

Standard single-target CD19-directed CAR T-cell therapies typically enable long-term remissions in roughly 40% of patients, emphasizing a significant unmet need for new and more effective treatment strategies. Among patients who had previously undergone two or more lines of therapy (n = 12), the ORR was 92% and the CRR was 75%.

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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. This has led drug developers to unintentionally limit their potential within chosen therapeutic spaces.

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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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The Pharma Data

JUNE 9, 2025

These results further validate the potential of Ascendis’ proprietary TransCon technology to deliver long-acting therapies that improve both efficacy and patient experience. TransCon hGH , marketed as SKYTROFA® for pediatric growth hormone deficiency, is a once-weekly prodrug of somatropin (human growth hormone).

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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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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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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. SCLC generally has a “hot” tumor microenvironment (TME) meaning that it is infiltrated by immune cells, including the T cells that are activated by immune checkpoint therapies.

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The Pharma Data

JUNE 26, 2025

Details of the VEGA-3 Phase 3 Trial The VEGA-3 study is part of Viatris’ broader clinical development program to evaluate MR-141 for presbyopia. The trial was conducted as a randomized, placebo-controlled, double-blind study and enrolled a total of 545 adult participants.

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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. This is an AI generated image.

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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 24, 2025

The strategic funding arrangement is designed to support the continued advancement and global commercialization of Revolution Medicines’ RAS(ON) inhibitor portfolio, including its lead candidate daraxonrasib, as the company scales its operations independently across the international oncology market.

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Drug Target Review

JUNE 19, 2025

Still, Bazan says “Vertex’s work has been so important for the pain field – to get eyes on the large market size that matches the significant societal and clinical need for pain innovation.” Bazan sees it as part of a broader shift towards combination therapies – an approach that will likely define the next generation of pain management.

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Clinical Trials Molecular Biology Treatment Trials 52

Conversations in Drug Development Trends

OCTOBER 14, 2024

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. Given the complexity and widespread impact of autoimmune and bone health conditions, developing new therapies is essential.

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The Pharma Data

JUNE 18, 2025

The company’s lead candidate, NCX-L2 , is specifically designed to slow or even halt the progression of Parkinson’s disease—a neurodegenerative condition that currently has no disease-modifying therapies on the market. NCX-L2 takes a fundamentally different approach.

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Drug Patent Watch

DECEMBER 9, 2024

From Lab to Market: The Long Road of Drug Development Once a promising compound is identified, it enters the long and costly process of drug development. While the compound itself couldn’t be patented, various derivatives and combination therapies have been patented, allowing for continued development and distribution.

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The Pharma Data

JUNE 22, 2025

This endorsement reflects growing confidence in Sarclisa’s clinical potential when added to standard-of-care therapies and could contribute to reshaping the induction treatment landscape for patients preparing to undergo stem cell transplantation. These findings further reinforce the transformative potential of the Sarclisa-based regimen.

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The Pharma Data

JUNE 25, 2025

Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to riliprubart , an investigational immunology therapy developed by Sanofi, for the treatment of antibody-mediated rejection (AMR) in solid organ transplantation. FDA Orphan Drug Designation for Antibody-Mediated Rejection in Solid Organ Transplantation The U.S.

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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 18, 2025

The study successfully met its primary endpoint, showing that significantly fewer patients treated with atogepant discontinued therapy due to AEs compared to those on topiramate. This underscores the need for more effective and tolerable options, as current therapies often fall short of achieving satisfactory outcomes or long-term adherence.

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BioPharma Drive: Drug Pricing

JULY 8, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. Drugmakers are working to coopt this process with targeted therapies. You can unsubscribe at anytime.

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New Drug Approvals

JULY 9, 2025

5] In China, it was conditionally approved in 2023 for the treatment of NSCLC and full approval is contingent on results of phase 3 clinical trials. [6] 7] Efficacy was evaluated in WU-KONG1B (NCT03974022), a multinational, open-label, dose randomization trial. [1] 1] History Sunvozertinib is being developed by Dizal Pharmaceutical. [5]

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The Pharma Data

JUNE 27, 2025

This data includes results from two pivotal studies: Part C of the ongoing DEVOTE clinical trial and the final eight-year data from the NURTURE study. Head of the Neuromuscular Development Unit at Biogen. Jude Children’s Research Hospital.

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The Pharma Data

JUNE 6, 2025

The data underscored the significant clinical benefits associated with cystic fibrosis transmembrane conductance regulator (CFTR) modulators—particularly its next-generation triple therapy, ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor).

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The Pharma Data

JUNE 25, 2025

BeOne Medicines Receives Positive CHMP Opinion for New Film-Coated Tablet Formulation of BRUKINSA® (zanubrutinib), Paving the Way for European Market Approval BeOne Medicines Ltd. This dual regulatory progress signals strong global confidence in the new formulation and sets the stage for expanded availability in key markets.

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The Pharma Data

JUNE 20, 2025

FDA Approves Dupixent as the First and Only Targeted Therapy for Adults with Bullous Pemphigoid In a groundbreaking development for patients suffering from a rare and debilitating autoimmune skin disease, the U.S. This has underscored the urgent need for non-immunosuppressive, targeted therapies with more favorable safety profiles.

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The Pharma Data

JUNE 9, 2025

This new monoclonal antibody therapy is designed to prevent lower respiratory tract disease caused by respiratory syncytial virus (RSV) in neonates and infants born during or entering their first RSV season. Key results from the CLEVER trial include: A 60.5% Octavio Ramilo, Chair of the Department of Infectious Diseases at St.

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Conversations in Drug Development Trends

JULY 3, 2025

Approximately two in 10 patients with hematologic malignancies are estimated to be eligible and able to receive cell therapy. The REMS Burden Explained Once deemed necessary for managing the risks associated with CAR T therapies, the REMS requirements imposed significant operational burdens on treatment centers.

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Therapies Regulations Clinical Trials FDA 40

The Pharma Data

JUNE 6, 2025

Commitment to Innovation in Rare Blood Disorders Sanofi’s pipeline, centered on the complex biological mechanisms that drive rare blood diseases, reflects a strategic investment in therapies that aim to address persistent unmet medical needs. In addition, two poster presentations will offer deeper insights into the LUNA 3 trial.

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The Pharma Data

JUNE 24, 2025

This expanded partnership reflects Bayer’s ongoing commitment to strengthening its presence in China—home to the world’s second-largest pharmaceutical market—and underscores its strategic vision to bolster healthcare innovation through cross-sector collaboration.

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The Pharma Data

JUNE 9, 2025

The acceptance, which follows the CDE’s grant of Priority Review status in May, represents an accelerated regulatory pathway for what may become the first approved systemic therapy for TGCT in China. Food and Drug Administration, with additional filings planned in other markets.”

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The Pharma Data

JUNE 24, 2025

Royalty Pharma and Revolution Medicines Forge $2 Billion Strategic Funding Pact to Accelerate Global Development of RAS Cancer Therapies Royalty Pharma plc, a prominent funder of innovation in the biopharmaceutical industry, announced a transformative agreement with Revolution Medicines, Inc., securing a funding package of up to $2 billion.

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Clinical Development Packaging Therapies Treatment 40

Drug Target Review

JULY 7, 2025

Their unique suitability has made them valuable for evaluating pharmacokinetics, toxicology and safety in drug candidates before human clinical trials. While some therapeutic areas still rely heavily on NHPs – like biologics and gene therapies – many others are exploring models that offer better scalability and ethical acceptability.

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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. The biotech industry is a dynamic sector, rapidly evolving and poised for continued growth. FSP outsourcing is growing faster than FSO.

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The Pharma Data

JUNE 20, 2025

Individuals with T1D must rely on lifelong insulin therapy to regulate blood glucose levels. Zimislecel, Vertex’s investigational product, is a stem cell-derived, fully differentiated islet cell therapy designed to replace the function of the damaged pancreatic beta cells in individuals with T1D.

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BioPharma Drive: Drug Pricing

JULY 14, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. The most common adverse events reported were insomnia as well as the sudden urge to urinate.

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DrugBank

JUNE 18, 2025

Before a therapy can be approved for patient use, it must undergo extensive clinical testing and strictly adhere to regulatory guidelines. The failure rate in clinical trials exceeds 90%, often due to insufficient safety data, efficacy concerns, or regulatory non-compliance. Drug development is a complex and highly regulated process.

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Drug Development Clinical Trials Compliance Drugs 52