The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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The Pharma Data

MAY 2, 2021

Holzkirchen, May 3, 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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The Pharma Data

JANUARY 25, 2021

“The establishment of an Advisory Board focusing on Alzheimer’s disease, and the recruitment of such an impressive group of scientific and clinical leaders in this field is an extremely important step forward for the Company,” stated Paul Brennan, NervGen’s President & CEO. Dr.

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Conversations in Drug Development Trends

FEBRUARY 22, 2023

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

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The Pharma Data

APRIL 26, 2022

NYSE: PFE) today reported positive Phase 2 pediatric data for their Lyme disease vaccine candidate, VLA15. No vaccine-related serious adverse events (SAEs) were observed. About VLA15 VLA15 is the only Lyme disease vaccine candidate currently in clinical development. The study is being conducted at U.S.

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The Open Targets Blog

JUNE 26, 2023

This new target-disease association evidence is presented in a “CRISPR Screens” widget, contributing to our Pathways and Systems Biology data type. Thanks to the expertise of our colleagues at the Wellcome Sanger Institute, 23 screens included in CRISPRbrain were mapped to disease or diseases in the Open Targets Platform.

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The Pharma Data

JULY 12, 2021

Finerenone 10 mg or 20 mg is indicated to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, cardiovascular death, non-fatal myocardial infarction, and hospitalization for heart failure in adult patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D).

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The Pharma Data

MARCH 28, 2022

Dupixent peak sales ambition raised to more than €13 billion Chronic obstructive pulmonary disease 2023 pivotal readouts provide potential for additional Dupixent sales ambition upgrade 13 potential new medicines currently in the clinic to treat chronic inflammatory diseases, with 17 readouts expected by the end of 2024.

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The Pharma Data

JUNE 30, 2021

With a median follow-up of two years, the study met the primary endpoint of event-free survival (EFS; hazard ratio 0.398, p <0.0001). Event-free survival is defined as time from randomization to disease progression, commencement of new lymphoma therapy, or death from any cause. This press release features multimedia.

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The Pharma Data

JULY 27, 2021

AbbVie (NYSE: ABBV) and Calico Life Sciences today announced an extension of their leading-edge collaboration to discover, develop and bring to market new therapies for patients with age-related diseases, including neurodegeneration and cancer. This is the second extension of the collaboration originally established in 2014.

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The Pharma Data

OCTOBER 18, 2020

ZURICH, Switzerland–( BUSINESS WIRE )– Versantis AG , a clinical-stage company developing novel therapies for orphan liver diseases, today announced that the U.S. Up to 90% of newborns do not survive the initial event of acute hyperammonemia or suffer from severe neurological sequelae.

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Drug Target Review

OCTOBER 10, 2024

What are the risks of over-reliance on healthy donor material in early-stage research, and how can decision-makers ensure a balanced integration of diseased material to avoid potential gaps when transitioning to clinical trials? This can lead to low cell yields, poor product quality, and process variability.

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The Pharma Data

OCTOBER 27, 2020

Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease (RPD) designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). About Krabbe Disease. Passage Bio expects to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021.

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The Pharma Data

DECEMBER 29, 2020

(NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A.,

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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The Pharma Data

JANUARY 10, 2021

The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial. EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Enrolled patients will receive a single administration of EDIT-301.

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The Pharma Data

OCTOBER 26, 2020

(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. 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

JUNE 7, 2023

1,2 While first-line treatment with immune checkpoint inhibitors with or without chemotherapy has improved outcomes for patients with NSCLC without AGAs, like EGFR or ALK, most patients eventually experience disease progression. Disease control rates (DCR) of 84% and 87% were observed in the doublet and triplet cohorts, respectively.

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The Pharma Data

NOVEMBER 10, 2020

P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease. Crohn’s Disease Therapeutics Market Size $4.7 Phase1b/2 clinical study to be conducted in the United States and several European countries. Billion by 2025. and Europe.

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The Pharma Data

APRIL 28, 2022

In the trial, there was no statistical difference in estimated hypoglycaemia rates, and no severe hypoglycaemia events were observed for people treated with insulin icodec. The rates of severe or clinically significant hypoglycaemia (blood glucose below 3 mmol/L) were 0.73 events per patient-year exposed to insulin degludec.

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The Pharma Data

SEPTEMBER 16, 2020

executive vice president of Research and Development at Amgen, along with members of Amgen’s clinical development team and clinical investigators, will discuss Phase 1 data being presented on the Company’s investigational KRAS G12C inhibitor sotorasib (AMG 510). Reese , M.D., On Monday, Sept. PDT , David M.

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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. Lecanemab selectively binds to neutralize and eliminate soluble, toxic amyloid-beta (A?)

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The Pharma Data

OCTOBER 26, 2020

Participants may access the live webcast via a link on the Synlogic website in the Events Calendar of the Investors and Media section. The forward-looking statements contained in this press release reflect Synlogic’s current views with respect to future events. The conference ID number for the call is 8557525.

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LifeSciVC

APRIL 24, 2024

Target validation : Do we believe the target(s) plays a central role in disease biology and that modulation will modify disease? I will note that “de-risked” is never fully risk-free in therapeutics investing, as even precedented mechanisms hit stumbling blocks in preclinical or clinical development.

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Drug Target Review

JANUARY 15, 2024

Gaining access inside cells Fortunately, evolution has provided a mechanism, found in all higher vertebrates, for the immune system to monitor intracellular events. Previously, as a member of the faculty at Harvard Medical School , Dr Kavanagh was engaged in preclinical and clinical development of cellular immunotherapy and products.

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The Pharma Data

AUGUST 30, 2021

The FIGARO-DKD study demonstrated cardiovascular benefits of finerenone across a wide range of patients, including those with earlier stages of chronic kidney disease.”. A kidney outcome event occurred in 360 (5.5%) patients receiving finerenone and 465 (7.1%) receiving placebo. 0.88]; p=0.0002).

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LifeSciVC

JANUARY 16, 2024

recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Rise of the best-in-class biologics plays: Are you looking to in-license or invest in “de-risked” biology that is already in the clinic? Join the club. Figure 2 shows pipelines across modalities for some of the competitive targets today.

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The Pharma Data

OCTOBER 15, 2020

ASLAN believes ASLAN003 has the potential to bethe most potent oral inhibitor of DHODH currently in development for autoimmune disease, more than 30 times more potent at inhibiting the DHODH enzyme than teriflunomide. SINGAPORE, Oct. The company expects to share further details in early 2021. M edia and IR contacts.

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The Pharma Data

MARCH 18, 2021

The most common adverse events were gastrointestinal. Across all groups, 5–14% of people discontinued any trial treatment due to adverse events. NASH is a disease with a high unmet medical need, as no drugs are currently approved to treat this potentially life-threatening condition. About NASH.

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Advarra

FEBRUARY 24, 2023

But what does “working” really mean when efficacy means something subjective, like quality of life, reduced psychiatric events, or less symptoms? Also known as a clinical event committee (CEC), EAC members evaluate a clinical event in the context of the protocol and the individual participant’s medical information.

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The Pharma Data

NOVEMBER 5, 2021

EMPEROR Program clinical investigator and Emeritus Professor of Rectifiers and Cardiology at the University of Lorraine, France.”The vice chairman, Clinical Development & Medical Affairs, Cardio-Metabolism & Respiratory Medicine, Boehringer Ingelheim Pharmaceuticals,Inc.”The

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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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The Pharma Data

OCTOBER 14, 2020

Live Webcast : accessible from the Company’s website at www.eloxxpharma.com under Events and Presentations or with this link: [link]. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis.

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The Pharma Data

DECEMBER 10, 2020

executive vice president of research and development. “We We look forward to working closely with the FDA throughout the clinical development process to bring this potential new innovative treatment to patients as quickly as possible.”. Safe Harbor Statement. Source link.

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The Pharma Data

OCTOBER 25, 2020

We are advancing the clinical development of mRNA-1273 with our Phase 3 COVE study, which is now fully enrolled with a representative demography of participants across ages, ethnicities and high-risk populations. Moderna currently has strategic alliances for development programs with AstraZeneca PLC and Merck & Co.,

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The Pharma Data

MARCH 2, 2021

This new collaboration builds on the positive experience from previous partnerships with Gubra and combines Gubra’s proprietary streaMLine platform and expertise in obesity and peptide chemistry with Boehringer Ingelheim’s expertise in lead optimization and clinical development. population will have obesity. About Boehringer Ingelheim.

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The Pharma Data

NOVEMBER 11, 2021

27 kg/m 2 ) with at least one weight-related comorbidity such as high blood pressure, heart disease or type 2 diabetes. The positive CHMP opinion is based on results from the STEP phase 3a clinical trial programme. About obesity Obesity is a chronic disease that requires long-term management. Once-weekly semaglutide 2.4

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