Conversations in Drug Development Trends

NOVEMBER 5, 2024

In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.

Disease 52

Disease Clinical Trials Therapies Clinical Research 52

New Drug Approvals

APRIL 2, 2025

1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] 2] Medical uses Suzetrigine is indicated for the treatment of moderate to severe acute pain in adults. [1] The FDA has long supported development of non-opioid pain treatment.

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FDA Therapies Trials Treatment 62

SugarCone Biotech

MARCH 2, 2025

This is a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy of ficerafusp alfa plus pembrolizumab versus pembrolizumab alone as a first-line treatment for patients with HPV-negative population recurrent/metastatic HNSCC. No chemotherapy regimen is used in the trial. months and the median OS = 12.5

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Therapies Clinical Trials Trials Clinical Development 70

Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial. As a result, waiting lists continue to grow.

Therapies 102

Therapies Disease RNA Engineering 102

Fierce BioTech

MARCH 6, 2025

AI-powered digital phenotyping and real-world data (RWD) offer new ways to locate overlooked patient populations, identify gaps between clinical guidelines and real-world treatment patterns, and optimize market positioning and messaging. On Demand Start Date Tue, 04/08/2025 - 14:00 End Date/Time Tue, 04/08/2025 - 14:00

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Treatment Therapies Marketing 75

DS in Pharmatics

APRIL 4, 2025

The Center for Biologics Evaluation and Research (CBER) plays a critical role in overseeing the approval of innovative biologic products, including gene therapies, cell-based treatments, and vaccines.

Vaccine 52

Vaccine Therapies Treatment Production 52

H1 Blog

DECEMBER 11, 2024

In this edition, our CEO and co-founder, Ariel Katz, along with Regional VP of Trial Landscape, Ryan Brown, share their top predictions for 2025. While this approach aims to improve ROI before potential price controls kick in, it complicates trial design and recruitment, increasing the time it takes to get treatments to market.

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Clinical Trials Trials Pharma Companies Treatment 52

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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Treatment Trials Clinical Trials Drugs 69

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.

Clinical Trials 52

Clinical Trials Disease Trials Therapies 52

Fierce BioTech

JANUARY 16, 2025

The complex clinical planning, potential for nuanced regulatory hurdles, payer scrutiny, and supply chain complexity all necessitate early and strategic planning to optimize patient access to these novel treatment options at launch. On Demand Start Date Thu, 03/06/2025 - 13:00 End Date/Time Thu, 03/06/2025 - 12:00

Therapies 52

Therapies Treatment Disease Marketing 52

Fierce BioTech

AUGUST 7, 2024

A Focus on ADC and Radioisotope Therapy pesurya Wed, 08/07/2024 - 11:14 Wed, 10/16/2024 - 14:00 Resource Type Webinar Promotion Start Fri, 08/16/2024 - 11:17 Promotion End Sun, 02/16/2025 - 11:00 Duration 60 Minutes Medical imaging is critical in clinical trials, serving as a key component in assessing treatment efficacy and safety.

Therapies 52

Therapies Clinical Trials Trials Treatment 52

Vial

DECEMBER 8, 2023

With technological innovation and scientific research propelling the development of new therapies, anticipation for the future is high. As we look forward to 2024, several groundbreaking therapies are poised to radically change the landscape of ophthalmology, providing hope and improved treatment options for patients with ocular conditions.

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Therapies Clinical Trials Trials Treatment 52

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. Our lead program utilises OSK for the treatment of age-related optic neuropathies.

Science 145

Science Disease FDA Approval Clinical Trials 145

Drug Target Review

JULY 1, 2024

As a non-opioid, SRP-001 also eliminates abuse potential, positioning it as a safer and effective drug candidate for the treatment of acute and neuropathic pain and migraine headache. Could you elaborate on the potential market size for SRP-001 and how it fits into the broader landscape of pain therapies? South Rampart Pharma, Inc.

Treatment 59

Treatment Clinical Trials Therapies Molecular Biology 59

Fierce BioTech

JANUARY 3, 2025

This joint webinar presentation by West and Groninger, explores the unique challenges of Cell and Gene Therapies, mRNA LNP therapeutics particularly regarding cold storage requirements and their impact on packaging solutions. On Demand Start Date Wed, 02/12/2025 - 11:00 Listing Image WestPharma_ListingLogo_250x190.png

Packaging 52

Packaging Therapies Pharmaceuticals Treatment 52

Fierce BioTech

JANUARY 3, 2025

Innovations in aseptic processing for container closure systems dwunderlin Fri, 01/03/2025 - 14:26 Wed, 02/12/2025 - 10:00 Resource Type Webinar Jim Thompson Julian Petersen Duration 60 Minutes The pharmaceutical landscape is shifting from blockbuster drugs to personalized treatments, driving the need for innovative container closure systems.

Packaging 52

Packaging Therapies Pharmaceuticals Treatment 52

Fierce BioTech

AUGUST 1, 2024

Jones Promotion Start Wed, 08/14/2024 - 13:35 Promotion End Fri, 02/14/2025 - 13:35 Duration 60 Minutes In the fast-evolving field of cell and gene therapy, overcoming bottlenecks at every stage of the process – both upstream and downstream of manufacturing – is crucial to ensure timely and effective treatment delivery.

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Therapies Treatment 52

The Pharma Data

JANUARY 15, 2021

for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen. .–(BUSINESS WIRE) January 15, 2021 — Daiichi Sankyo Company, Ltd. In the U.S., Sammons Cancer Center and the W.W. Caruth, Jr.

Treatment 52

Treatment Trials Disease Therapies 52

Codon

SEPTEMBER 29, 2024

2025 While the broader life sciences community had pivoted towards working on traditional mechanistic interpretations of biology, one graduate student still believed there was something to be learned using the models of life so celebrated previously. The 2025 genetic network paper fit that bill exactly.

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Engineering Virus DNA Therapies 142

KIF1A

AUGUST 28, 2024

2025 Conference Interest Survey : Help shape the future of our community by sharing your interest in the upcoming 2025 conference. Dr Verden’s Blog Post n-Lorem’s Press Release KIF1A-ASO-Article Download 2nd KAND patient receiving ASO treatment Meet Sloane. We kindly ask that everyone completes this 5 minute survey.

Therapies 52

Therapies Compound Screening Research Treatment 52

Drug Target Review

JULY 17, 2024

How does GigaGen’s single-cell discovery and development platform differ from traditional methods of producing polyclonal antibody therapies? Current methods for producing pAb therapies rely on regular plasma donations for their development. However, recombinant pAbs do not rely on continuous plasma sources for production.

Immune Response 59

Immune Response Therapies Laboratories Vaccine 59

The Pharma Data

SEPTEMBER 16, 2020

SLB linked to 2025 Patient Access Targets to increase patients reached in low- and middle-income countries (LMICs) with strategic innovative therapies by 200% and the Novartis Flagship Programs by 50%. Weak healthcare systems in LMICs also hinder timely diagnosis and treatment.

Therapies 52

Therapies Regulations Government Disease 52

The Pharma Data

JANUARY 6, 2021

CAMBRIDGE, England & WETHERBY, England–( BUSINESS WIRE )– Avacta Group plc (AIM: AVCT), the developer of innovative cancer therapies and diagnostics based on its proprietary Affimer ® and pre|CISION platforms, is pleased to announce that it has entered into a license agreement with POINT Biopharma Inc. 7, 2021 08:49 UTC.

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Licensing Licensing Therapies Clinical Trials 52

Conversations in Drug Development Trends

MAY 23, 2024

The Critical EU-CTR Transition Deadline The three-year transition period for clinical trials authorized under the CTD that are expected to continue beyond the critical deadline of 30 January 2025 are required to transition to the new EU-CTR via a transition application through the CTIS. However, they also bring unique regulatory challenges.

Trials 52

Trials Clinical Trials Therapies Treatment 52

The Pharma Data

FEBRUARY 22, 2022

Pfizer’s 2025 Opportunity Parity goals represent the company’s commitment to increasing representation of women and minorities at senior -level positions of the organization. Progress made from June 2019 to December 2021 towards different aspects of its 2025 Opportunity Parity Goals are highlighted below.

Government 52

Government Vaccine Science Therapies 52

PPD

OCTOBER 12, 2023

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. These treatments use a vector to introduce the desired nucleic acid code to replace or modify protein expression or use cells to alter/restore a specific cell type.

Laboratories 52

Laboratories Clinical Trials Protein Expression Pharmacokinetics 52

The Pharma Data

SEPTEMBER 15, 2020

27 key programs highlighted, including assets that could potentially contribute revenue by 2025 and others in the 2026-2028 time frame. Major revenue contributions through 2025 anticipated from Oncology, Vaccines, Rare Disease and Inflammation and Immunology.

Vaccine 52

Vaccine Therapies Trials Clinical Trials 52

The Pharma Data

JULY 15, 2021

TRAILBLAZER-ALZ 3 will evaluate whether treatment with donanemab can slow the clinical progression of Alzheimer’s disease in trial participants. The collaboration will introduce a more virtual approach to the evaluation of Alzheimer’s prevention therapies. vice president of pain and neurodegeneration, Lilly.

Trials 52

Trials Therapies Clinical Trials Disease 52

The Premier Consulting Blog

DECEMBER 14, 2022

Digital health products are widely used for the prevention, diagnosis, treatment, and management of health and disease. The introduction of digital medicines—and, more recently, digital therapeutics (DTx)—has begun to influence the treatment and management of certain diseases, both by consumers and by clinicians.

Production 52

Production Regulations Therapies Pharmaceuticals 52

The Pharma Data

AUGUST 3, 2021

Breakthrough results in the treatment of a type of heart failure, which is extremely difficult to manage Strong progress in mental health pipeline Good business performance in all areas, despite ongoing impact of COVID-19. This pipeline has the potential to deliver up to 15 medicines for approval by 2025.

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Research Treatment Clinical Trials Production 52

The Pharma Data

NOVEMBER 2, 2020

The notes will mature on November 1, 2025, unless earlier repurchased, redeemed or converted. Prior to the close of business on the business day immediately preceding August 1, 2025, the notes are convertible at the option of the holders only upon the satisfaction of certain conditions and during certain periods.

Clinical Trials 52

Clinical Trials Production Trials Treatment 52

LifeSciVC

AUGUST 21, 2024

At this point of the year, the JPMorgan conference seems like ancient history and you are looking into flights for JPM 2025. And importantly, breaks from treatment or treatment protocols can literally make or break trials. For some, a Midsummer’s Madness can be a Midsummer’s Nightmare. These goals were believable and achievable.

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Clinical Trials Trials Disease Treatment 72

Vial

APRIL 18, 2024

Market research indicates significant growth in the ophthalmic clinical trials market, driven by increased disease prevalence, demand for ocular treatments, and rising research funding. Food and Drug Administration (FDA), aiming to provide patients with an effective and convenient treatment option for post-operative inflammation and pain.

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Clinical Trials Trials Treatment Clinical Research 52

The Pharma Data

NOVEMBER 10, 2020

Billion by 2025. We believe the scientific rationale for oral treatment with Foralumab is logical to facilitate topical action at the inflamed sites in the gastrointestinal tract. billion by 2025, according to Grand View Research 1. Crohn’s Disease Therapeutics Market Size $4.7 NEW YORK and LONDON, Nov.

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Clinical Trials Science Disease Trials 40

New Drug Approvals

SEPTEMBER 13, 2024

2] [3] [4] A regulatory application for approval of the medication is expected to be submitted by 2025. [2] 7] Aticaprant was also under development for the treatment of alcoholism , cocaine use disorder , and smoking withdrawal , but development for these indications was discontinued. [2] 2] Aticaprant is taken by mouth. [1]

Treatment 62

Treatment Clinical Trials Pharmacokinetics Pharmaceuticals 62

The Pharma Data

NOVEMBER 16, 2020

In 2020, Regeneron has reinforced our commitment to ‘doing well by doing good,’ as colleagues across the company have worked tirelessly to discover and develop a potential antibody therapy for COVID-19, while at the same time responding to the needs of our colleagues, patients and communities during this public health crisis.”

FDA Approval 52

FDA Approval Science Disease Laboratories 52

The Pharma Data

JANUARY 4, 2021

SENS-401 has demonstrated highly encouraging efficacy in preclinical models and we are excited to progress this small molecule towards clinical trials to address this significant unmet medical need in cancer patients undergoing cisplatin treatment. About Sensorion. www.sensorion-pharma.com.

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Clinical Trials Small Molecule Therapies Treatment 40