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.

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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.

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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.

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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.

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The Pharma Data

JUNE 2, 2023

Nasdaq: GILD) and Kite, a Gilead Company, will present 17 abstracts from its industry-leading cell therapy portfolio and growing blood cancer pipeline at the upcoming 2023 European Hematology Association (EHA) Annual Congress being held in Frankfurt, Germany, June 8-11, and virtually June 14-15. & SANTA MONICA, Calif.–(BUSINESS

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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.”.

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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.

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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.

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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.

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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.

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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.

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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.,

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The Pharma Data

OCTOBER 21, 2020

(Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced that it will present a poster on SYNB8802, an investigational Synthetic Biotic medicine for the treatment of Enteric Hyperoxaluria (HOX), during the American Society of Nephrology (ASN) Kidney Week, Oct.

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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.

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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

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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.

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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

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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.

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The Pharma Data

OCTOBER 14, 2020

a clinical-stage biopharmaceutical company focused on discovering and developing a new generation of antibody therapeutics for oncology, announced today that the U.S. Binding of APX005M to CD40 on antigen presenting cells (i.e., SAN CARLOS, Calif. , 15, 2020 /PRNewswire/ — Apexigen, Inc. ,

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Drug Target Review

MAY 7, 2024

However, some biomarkers may only be present in a minority of patients, requiring a large patient population to identify such markers. Moreover, the application of synthetic control arms presents the opportunity to transform the trial process itself. Not all biomarkers have a limited prevalence.

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Conversations in Drug Development Trends

MAY 30, 2024

Since marketing authorization for the first breakthrough treatment in 1994, the steady increase in clinical trials reflects the community’s commitment to finding effective ALS treatments despite the numerous hurdles associated with clinical trial design, from proof-of-concept to pivotal trials.

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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.

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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.

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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.

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The Pharma Data

NOVEMBER 3, 2020

Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Commenting on the announcement, Daniel Gruskin, M.D.,

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The Pharma Data

OCTOBER 26, 2020

Food and Drug Administration (FDA) has granted Fast Track designation for the Company’s experimental gene therapy program, PR001, for the treatment of neuronopathic Gaucher disease (nGD). In addition, the FDA has granted Fast Track designation for PR001 for the treatment of Parkinson’s disease with GBA1 mutations.

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The Pharma Data

OCTOBER 18, 2020

Food and Drug Administration (FDA) has granted a rare pediatric disease designation (RPDD) to its lead product candidate VS-01, an ammonia clearance enhancer, for the treatment of Urea Cycle Disorders (UCD). Current treatment options are associated with poor outcomes. This voucher may be sold or transferred to another sponsor.

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The Pharma Data

JUNE 29, 2021

ENSPRYNG is the first and only NMOSD treatment that is administered subcutaneously every four weeks, allowing home-dosing after appropriate training. Importantly, people with NMOSD now have the flexibility to administer treatment at home, which may alleviate the need to travel for hospital appointments.”.

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The Pharma Data

SEPTEMBER 19, 2020

Results from the Phase III IMpassion131 study, evaluating Tecentriq in combination with paclitaxel for the treatment of people with metastatic TNBC and whose tumours expressed PD-L1, did not meet its primary endpoint of progression-free survival . Roche’s Chief Medical Officer and Head of Global Product Development. “We

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The Pharma Data

JULY 12, 2021

The clinical data from FIGARO-DKD are scheduled for presentation at the European Society of Cardiology (ESC) Congress 2021 on August 28th. The cardiology franchise at Bayer already includes a number of products and several other compounds in various stages of preclinical and clinical development. Source link: [link].

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LifeSciVC

JULY 11, 2023

Redig, SVP and Head of Clinical Development at HotSpot Therapeutics, as part of the From The Trenches feature of LifeSciVC The American Cancer Society for Oncology (ASCO) annual meeting is among the highlights of the clinical oncology calendar. By Amanda J. His remarks are worth reading in their entirety.

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The Premier Consulting Blog

OCTOBER 11, 2022

In this article, we review two case studies involving the successful use of RWD or RWE in advancing the clinical development of treatments for rare diseases. During its first meeting with the FDA, the sponsor: Presented data by subject to the FDA in order to underscore changes over time within an individual patient.

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Drug Target Review

AUGUST 28, 2024

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.

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SugarCone Biotech

JUNE 10, 2024

[link] Immunity is complex and can be dangerous when exploited clinically, as demonstrated by the lethal administration of TNF, or anti-CD40L antibody (Biogen) or CAR-T cells expressing the CD16 Fc-receptor (Unum), among many other examples. This observation may be important given the critical role of DC in tumor antigen presentation.

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The Premier Consulting Blog

JULY 9, 2023

Formally, both of these documents apply solely to marketing approval; informally, however, they are often used as general guidelines during clinical development to avoid obstacles that could result in extensive process changes, product reformulation, or safety qualification efforts. evaluating the safety of) impurities and degradants.

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The Pharma Data

JUNE 25, 2021

protofibril antibody for the treatment of Alzheimer’s disease (AD). Breakthrough Therapy designation is an FDA program intended to expedite the development and review of medicines for serious or life-threatening conditions. and clinical decline.

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The Pharma Data

DECEMBER 13, 2020

Nasdaq: SYBX ), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced SYNB1891 has advanced into the combination therapy stage of the ongoing Phase 1 trial. Results of the SYNB1891 Phase 1 study will be presented at a future medical meeting. CAMBRIDGE, Mass. ,

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