Conversations in Drug Development Trends

NOVEMBER 21, 2023

Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. As a provider of end-to-end clinical development solutions, we plan to leverage our expertise to refine drug-disease matches identified by Every Cure’s use of AI and data analysis.

Disease 78

Disease Treatment Clinical Trials Clinical Development 78

PPD

NOVEMBER 9, 2023

Instead of the standard approach where studies are designed to address a primary focus, a parallel study method — a format that incorporates multiple endpoints and objectives, modifications, treatment arms and dose selections into a single protocol — is being rapidly adopted.

Clinical Development 52

Clinical Development Drug Development Clinical Research Treatment 52

The Pharma Data

JULY 29, 2021

Eisai and Biogen Present Preliminary Assessment of the Clinical Effects of Lecanemab Following 18 Months of Treatment in the Open-Label Extension of the Phase 2 Proof of Concept Study at 2021 Alzheimer’s Association International Conference (AAIC) . United States and virtually from July 26 to 30, 2021 (Presentation No.:

Clinical Trials 52

Clinical Trials Treatment Clinical Development Disease 52

PPD

MARCH 11, 2025

Developing treatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. These conditions pose substantial challenges for patients, families and health care systems.

Drug Development 52

Drug Development Clinical Trials Disease Trials 52

SugarCone Biotech

MARCH 2, 2025

Where TGF- is present there is no or limited IFN- secretion by T cells and that means no PD-L1 expression within the tumor microenvironment, aka the TME. The recent integrin inhibition program that was being developed by Pliant has not yet demonstrated a positive/risk benefit profile at least in pulmonary fibrosis.

Therapies 67

Therapies Clinical Trials Trials Clinical Development 67

The Pharma Data

JULY 12, 2021

Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the presentation of data from the company’s HIV clinical development program at the 11th International AIDS Society Conference on HIV Science (IAS 2021) from July 18-21. Late Breaking Oral Presentation OALC01LB03. Abstract 2361.

Pharmacokinetics 52

Pharmacokinetics Clinical Trials Trials Treatment 52

The Pharma Data

JANUARY 10, 2021

Chief Executive Officer of MorphoSys, will present at the 39 th Annual J.P. EST. Live audio of the presentation can be accessed from the Media and Investors section under Conferences on MorphoSys’ website, www.morphosys.com. The presentation as well as a replay of the webcast will also be available on MorphoSys’ website.

Clinical Trials 52

Clinical Trials Clinical Development Clinical Supply Trials 52

The Pharma Data

OCTOBER 21, 2020

and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinical development programme. These findings were presented today at the 2020 Infectious Diseases Society of America (IDSA) IDWeek.

Clinical Development 52

Clinical Development Therapies Clinical Trials Treatment 52

The Pharma Data

SEPTEMBER 8, 2021

Amgen (NASDAQ: AMGN) today announced results from two analyses of the Phase 2 CodeBreaK 100 clinical trial evaluating LUMAKRAS™ (sotorasib), the first and only KRAS G12C inhibitor approved in the U.S., in the treatment of previously treated patients with advanced or metastatic KRAS G12C-mutated non-small cell lung cancer (NSCLC).

Trials 52

Trials Clinical Trials Therapies Treatment 52

Drug Target Review

JANUARY 15, 2024

Monoclonal antibodies (mAbs) targeting tumour-specific antigens play an essential role in the treatment of many cancers. An important limitation of antibodies against tumour antigens is that these agents direct responses to molecular targets present on the surface of the cell.

Engineering 110

Engineering DNA Virus Immune Response 110

The Pharma Data

NOVEMBER 9, 2021

(Nasdaq GILD) moment blazoned the donation of new clinical and real- world data at the American Association for the Study of Liver Conditions (AASLD) The Liver Meeting ®, taking place from November 12-15. Case Impact of HDV Treatment. Bulevirtide is an investigational agent in theU.S.

Disease 52

Disease Clinical Trials Treatment Science 52

The Pharma Data

OCTOBER 28, 2021

Bayer will present new renal and cardiovascular (CV) analyses from the comprehensive finerenone (Kerendia®) clinical trial program, including the Phase III FIGARO-DKD and FIDELIO-DKD studies, and the prespecified pooled analysis FIDELITY at the American Society of Nephrology (ASN)’s Kidney Week 2021 from 4-7 November.

Clinical Trials 52

Clinical Trials Trials Disease Hospitals 52

PPD

OCTOBER 17, 2024

In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches. Site selection presents barriers and opportunities As cell therapies expand in Asia-Pacific, areas with large hospitals are often the main hubs for innovation.

Therapies 97

Therapies Clinical Trials Clinical Research Trials 97

KIF1A

SEPTEMBER 30, 2023

Read the Article Both of these studies reflect a global effort to understand the genetic underpinnings of disorders so people can find the right treatments sooner. In addition to benefiting awarded sponsors, this pilot program will help create best practices of communication with the FDA during rare disease therapeutic development.

Disease 111

Disease FDA Clinical Trials Therapies 111

KIF1A

MARCH 16, 2024

Whole genome sequencing can identify additional mutations that aren’t included on CMT panels, providing patients with a more accurate diagnosis to find resources and treatments. The study identified 8 groups based on symptom presentation, and found over 70 disease-associated genes, including 4 patients with KIF1A mutations.

Disease 105

Disease Clinical Development Research Treatment 105

The Pharma Data

OCTOBER 26, 2020

(Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening infections, today announced the Company will present at the ROTH Capital Partners 2020 MedTech Innovation Forum on Wednesday, October 28, 2020. About Aridis Pharmaceuticals, Inc.

Pharmaceuticals 52

Pharmaceuticals Virus Hospitals Clinical Trials 52

Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? But commitment to neuroscience drug development is critically important given the significant unmet medical needs and the ways in which patients, caregivers, and families suffer.

Therapies 104

Therapies Drug Development Disease Clinical Development 104

The Pharma Data

SEPTEMBER 29, 2021

to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. Christian Rommel, Member of the chief Committee of Bayer AG’s Pharmaceutical Division and Head of Research and Development.

Clinical Development 52

Clinical Development Disease Clinical Trials Trials 52

The Pharma Data

SEPTEMBER 16, 2020

New integrated analyses from our tumour agnostic Rozlytrek ® (entrectinib) clinical development programme. Blueprint Medicines will present new data from the registrational phase I/II ARROW trial, investigating Gavreto TM (pralsetinib) for the treatment of people with RET-mutant medullary thyroid cancer.

Trials 52

Trials Treatment Disease Pharmaceuticals 52

The Pharma Data

SEPTEMBER 2, 2020

New data further reinforce OCREVUS (ocrelizumab) as a highly effective treatment option offering a favourable and consistent benefit:risk profile, with high treatment persistence and adherence. Initiation of Phase IIIb OCREVUS higher dose clinical trial programme and Phase IV study evaluating OCREVUS in minority populations.

Clinical Trials 52

Clinical Trials Treatment Disease Therapies 52

The Pharma Data

NOVEMBER 14, 2020

The results were presented at The Liver Meeting Digital Experience (TLMdX), November 13–16, 2020 (Late Breaker #L02). Across all groups, 5–14% of people discontinued any trial treatment due to AEs. The companies are also presenting preclinical data supporting the development of combination approaches in NASH. About NASH.

Trials 52

Trials Science Treatment Disease 52

The Pharma Data

JULY 28, 2021

– If Approved, Investigational TAK-994 May Provide a Future Treatment Option Targeting the Orexin Deficiency Underlying NT1. TAK-994 is currently being studied for the treatment of excessive daytime sleepiness (EDS) in patients with narcolepsy type 1 (NT1), 2 a chronic neurological disorder that alters the sleep-wake cycle.

Clinical Development 52

Clinical Development Therapies Laboratories Treatment 52

The Pharma Data

JANUARY 14, 2021

(NASDAQ: FPRX) today announced clinical results from the global, randomized, double-blind placebo-controlled Phase 2 FIGHT trial evaluating first-in-class targeted therapy bemarituzumab in advanced gastric or gastroesophageal junction (GEJ) cancer. The ASCO GI presentation slides are available on the company’s website. Wainberg, M.D.,

Trials 52

Trials Treatment Therapies Clinical Trials 52

The Pharma Data

JANUARY 13, 2021

Food and Drug Administration (FDA) has granted Orphan Drug Designation for HPN217 for the treatment of multiple myeloma. Harpoon has four drug product candidates in clinical development for the treatment of solid and hematologic malignancies based on its proprietary TriTAC platform. “I

Treatment 52

Treatment FDA Clinical Trials Trials 52

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 52

Drug Target Review

JANUARY 8, 2024

Following that work, it was discovered by Dr David Sinclair’s lab at Harvard that turning on only three of the four factors (OSK; Oct4, Sox2, Klf4) is the sweet spot for developing therapeutics where you can partially reprogram cells and restore cells to a more youthful state, while maintaining cell identity.

Science 145

Science Disease FDA Approval Clinical Trials 145

The Pharma Data

AUGUST 19, 2021

Oral presentation at the International Association for the Study of Lung Cancer’s (IASLC) 2021 World Conference on Lung Cancer (WCLC) shows evidence that the bispecific mechanism of action for RYBREVANT TM can provide anti-tumor activity against either EGFR-mutated or MET-mutated non-small cell lung cancer. 1 Treatment-related Grade ?3

Treatment 52

Treatment FDA Approval Pharmacokinetics FDA 52

The Pharma Data

FEBRUARY 24, 2022

If successful, the results of the CONFIDENCE study could be of great significance to clinicians when considering how to optimize disease management and further delay kidney disease progression, while also providing additional insights into the safety of using these treatments in combination.”.

Clinical Development 52

Clinical Development Disease Therapies Treatment 52

Drug Target Review

APRIL 3, 2024

Our research demonstrated that GDF-15 acts as a versatile tool for tumours to inhibit the mounting of an effective antitumoral immune response at various steps of its process, including antigen presentation, immune cell activation, and their migration and infiltration into the tumour.

Science 105

Science Clinical Development Treatment DNA 105

Conversations in Drug Development Trends

JANUARY 24, 2024

Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare Disease Clinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. One diagnostic example that I discussed in my presentation is autism.

Trials 75

Trials Disease Clinical Trials Clinical Research 75

Broad Institute

OCTOBER 11, 2023

Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. His mother had a presentation of the disease that suggested her immune system was already on the job.

Research 137

Research Immune Response Clinical Trials Therapies 137

The Pharma Data

APRIL 27, 2022

Novartis intends to submit these data to regulatory authorities, and will collaborate with BeiGene to present them at an upcoming medical meeting. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need.

Treatment 52

Treatment Clinical Development Therapies Clinical Trials 52

The Pharma Data

JANUARY 27, 2021

. “The responses observed in six out of nine patients are very encouraging and clearly suggest that BI-1206 may restore the response to rituximab in patients who have few treatment alternatives. ET ) to discuss the results and next steps in clinical development of BI-1206. CEO of BioInvent.

Pharmaceuticals 52

Pharmaceuticals Clinical Trials Licensing Licensing 52

The Pharma Data

APRIL 12, 2022

20% variant allele frequency reduction – At the time of analysis with > 2 year follow up the survival estimate was 100% in patients who had improvements in bone marrow fibrosis or variant allele frequency – Results were presented at the American Association for Cancer Research annual meeting.

Medical Schools 52

Medical Schools Trials Disease Clinical Development 52

The Pharma Data

OCTOBER 28, 2020

SIGNAL Phase 2 trial data continue to support cognitive benefit and reduced brain atrophy with pepinemab treatment in Huntington’s disease. Data provide strong rationale for continued development in Huntington’s and other slowly progressive neurodegenerative diseases, including Alzheimer’s. ROCHESTER, N.Y., About Vaccinex, Inc.

Clinical Trials 40

Clinical Trials Treatment Disease Clinical Development 40

The Pharma Data

MARCH 29, 2022

Opdivo -based treatments have shown clinical benefit in four Phase 3 trials in earlier stages of cancer. Food and Drug Administration approved Opdivo in combination with chemotherapy for the treatment of adult patients with resectable (tumors ?4 CT, in New Orleans, Louisiana. Source link: [link].

Treatment 52

Treatment Clinical Trials Trials Immune Response 52

LifeSciVC

OCTOBER 10, 2024

At the congress, we were thrilled to share some of the clinical data emerging from our Phase 1 program , an ongoing study evaluating our potent and selective investigational CBL-B inhibitor, HST-1011, in patients with advanced solid tumors.

Therapies 66

Therapies Clinical Development Pharmacokinetics Treatment 66