The Pharma Data

JULY 28, 2021

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for avalglucosidase alfa, a long-term enzyme replacement therapy for the treatment of people with Pompe disease. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

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Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

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The Pharma Data

AUGUST 3, 2021

In the first half of 2021, Boehringer Ingelheim has supplied more humans and animals worldwide with innovative medicines than ever before. The company’s success is based on its rich R&D portfolio with a focus on diseases with high unmet medical need. More than 60 million patients worldwide suffer from this disease, approx.

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The Pharma Data

JANUARY 26, 2021

Novel chronic kidney disease (CKD) target from the partnership enters AstraZeneca’s portfolio. CKD is a complex, and debilitating disease which affects around 10% of the world’s population. LONDON , Jan. LONDON , Jan.

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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. Accessed April 2021].

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The Pharma Data

AUGUST 15, 2021

The US Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the New Drug Application (NDA) for roxadustat for the treatment of anaemia of chronic kidney disease (CKD), in both non-dialysis dependent (NDD) and dialysis-dependent (DD) adult patients. Anaemia is particularly prevalent in patients with CKD.

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The Pharma Data

DECEMBER 18, 2020

18 December 2020 — The US Food and Drug Administration (FDA) has requested further clarifying analyses of clinical data, to complete its review of the New Drug Application (NDA) for roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor for patients with anemia of chronic kidney disease (CKD).

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The Pharma Data

JANUARY 19, 2021

With this kind of a start, 2021 may be scorching. The company hopes to raise $140 million of common stock, which will be used to advance its clinical and earlier stage programs and for R&D, working capital and general corporate purposes. Last year was a record number of IPOs in the biotech industry, with 81 raising $13.5

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The Pharma Data

MAY 31, 2022

the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of of BRT-DA01 in patients with Parkinson’s disease.?. The purpose of the?Ph1

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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

JANUARY 19, 2021

19, 2021 (GLOBE NEWSWIRE) — Fulcrum Therapeutics, Inc. The offering is expected to close on January 22, 2021, subject to customary closing conditions. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19.

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The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

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The Pharma Data

JANUARY 14, 2021

15, 2021 /PRNewswire/ — Calliditas Therapeutics AB (publ) (“Calliditas”) (Nasdaq OMX – CALTX) (NASDAQ – CALT) today announced the program for the upcoming virtual R&D Day for investors, analysts and journalists on January 20, 2021 , which will take place between 1pm and 5pm CET. STOCKHOLM , Jan.

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The Pharma Data

SEPTEMBER 8, 2021

today announced that data reinforcing the potential of immunotherapies to impact clinical outcomes for patients will be featured at the European Society for Medical Oncology (ESMO) 2021 Virtual Congress from September 16-21. To date, the Opdivo clinical development program has treated more than 35,000 patients.

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Drug Target Review

AUGUST 24, 2023

These findings were mirrored in the clinical results from an analysis of two metastatic melanoma patient cohorts who received anti-PD-1 treatment: patients with responses and disease control had significantly lower GDF-15 serum levels (measured before treatment start), while none of the patients with elevated GDF-15 levels showed a lasting response.

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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

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 20, 2021

20, 2021 /PRNewswire/ — INOVIO Pharmaceuticals, Inc. The offering is expected to close on or about January 25, 2021 , subject to customary closing conditions. PLYMOUTH MEETING, Pa. , Gross proceeds to INOVIO from the offering are expected to be approximately $150.5 All of the shares are being sold by INOVIO. About INOVIO.

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The Pharma Data

JANUARY 25, 2021

– January 26, 2021) – NervGen Pharma Corp. The AD-SAB will work closely with NervGen as the Company plans its upcoming preclinical studies and clinical trials and in the analyses of the results from these studies. Vancouver, British Columbia–(Newsfile Corp. Dr.

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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

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). Passage Bio expects to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021. About Krabbe Disease.

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The Pharma Data

NOVEMBER 5, 2021

The data were presented at the American Society of Nephrology (ASN) 2021 Annual Meeting. Novartis moment blazoned that a Phase II study of investigational iptacopan (LNP023) – a first-in- class, oral, picky factor B asset – in cases with C3 glomerulopathy (C3G) met primary endpoints in both patient cohorts1.

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The Pharma Data

JANUARY 18, 2021

Potential first-in-class disease-modifying biologic in late-stage clinical studies. Experienced executive development team from Gilead Sciences. 19, 2021 /PRNewswire/ — Vera Therapeutics, Inc. , SOUTH SAN FRANCISCO, Calif. ,

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The Pharma Data

MARCH 17, 2021

percent compared to ET alone – a statistically significant improvement in invasive disease-free survival for HR+, HER2- high risk early breast cancer (HR: 0.713; 95% CI: 0.583, 0.871; p = 0.0009). Gallen will be published in a supplement issue of The Breast, available on March 17, 2021. Available on-demand on March 10, 2021.

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Drug Target Review

NOVEMBER 30, 2023

From an early age, I’ve been passionate about finding cures for difficult-to-treat diseases. I accepted and I’ve been CEO ever since; our goal, from the beginning, was to pioneer new ways to diagnose, treat and prevent neurodegenerative diseases. That’s missing the point – we have learned how to slow the disease.

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Drug Target Review

JANUARY 15, 2024

As T cells develop, chromosomal DNA encoding the TCR of the CD8 T cells of each individual is rearranged and ‘educated’ to respond only to peptides presented in the context of a specific HLA Class I molecule present in that individual. The FEBS Journal 288:6159–6173 (2021). References Robinson R, McMurran C, McCully M, Cole D.

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The Pharma Data

APRIL 11, 2021

The data were presented at the 2021 American Association for Cancer Research (AACR) Annual Meeting, held virtually April 10-15, 2021. was approved by the European Commission in February 2021. The Phase 1/2 LIBRETTO-001 trial is the largest clinical trial of patients with RET -driven cancers treated with a RET inhibitor.

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The Pharma Data

OCTOBER 1, 2021

Deucravacitinib demonstrated efficacy no matter baseline characteristics, including weight , disease severity and former treatment with biologic or non-biologic therapies. Deucravacitinib is being studied in multiple immune-mediated diseases, including psoriasis, rheumatoid arthritis , lupus and inflammatory bowel disease.

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Disease Laboratories Clinical Pharmacology Treatment 52

The Pharma Data

JANUARY 17, 2021

The companies announced in January 2019 a collaboration agreement to develop, manufacture and commercialize SPR206 in the Territory. SPR206 is in clinical development as an innovative option for the treatment of multi-drug resistant (MDR) Gram-negative bacterial infections. SHANGHAI , Jan. SHANGHAI , Jan. ” About SPR206.

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The Pharma Data

JANUARY 10, 2021

11, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. Previously, the Company received Rare Pediatric Disease designation from the FDA for EDIT-301.

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Drug Target Review

SEPTEMBER 26, 2023

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. 1-4 This approach saves time, money, and resources, and ultimately leads to safer medicines going forward to the clinic. 2021 Jan 1;179(1):3–13. 2021 Jun;17(6):381–92.

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The Pharma Data

JANUARY 12, 2021

13, 2021 /PRNewswire/ — EVOQ Therapeutics today announced a license and collaboration agreement with Amgen for the discovery and development of novel drugs for autoimmune disorders. ANN ARBOR, Mich. , www.evoqtherapeutics.com.

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The Pharma Data

JUNE 2, 2022

Affinivax is pioneering the development of a novel class of vaccines, the most advanced of which are next-generation pneumococcal vaccines. A 30-plus valent pneumococcal candidate vaccine is also in pre-clinical development. pneumoniae invasive disease and pneumonia in adults 50 years and above.

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The Pharma Data

MARCH 27, 2021

Dr. Martin will assume responsibility for Gilead’s research organization effective April 12, 2021. He completed his postdoctoral training at the University of Alabama at Birmingham in the Division of Developmental and Clinical Immunology. Bill Lee, PhD, is retiring from Gilead after three decades. Source link:[link].

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The Pharma Data

MAY 31, 2022

Affinivax is pioneering the development of a novel class of vaccines, the most advanced of which are next-generation pneumococcal vaccines. A 30-plus valent pneumococcal candidate vaccine is also in pre-clinical development. pneumoniae invasive disease and pneumonia in adults 50 years and above. Pneumococcal disease.

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The Pharma Data

JANUARY 27, 2021

– Second Product Candidate Expected to Enter Clinic in First Half of 2021. Preclinical Data Underscore Treatment Potential for PBFT02 in Frontotemporal Dementia with Granulin (GRN) Mutations, a Devastating, Progressive Disorder Impacting Adults with No Approved Disease-Modifying Therapy Options. PHILADELPHIA, Jan.

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