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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FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. The draft guidance describes various methods to assess either cell distribution for cell therapy products or vector biodistribution for gene therapy products.

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Drug Target Review

AUGUST 24, 2023

Neutralising GDF-15 with CatalYm’s anti-GDF-15 antibody visugromab was shown to reverse its inhibitory effects and to re-sensitise tumours to anti-PD-1 treatment, achieving commensurate survival benefit of anti-GDF-15-anti-PD-1 combination therapy in vivo.

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Treatment Therapies Clinical Development Cell Biology 98

The Pharma Data

DECEMBER 2, 2020

The incidence and severity of thrombotic events will also be assessed. treatments that “bypass” the need for clotting factor treatment to help the body form a normal clot). treatments that “bypass” the need for clotting factor treatment to help the body form a normal clot). About the BASIS study.

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Therapies Treatment Disease Clinical Trials 52

The Pharma Data

SEPTEMBER 11, 2021

– Designation is based on results from the EMPEROR-Preserved phase III trial, which established Jardiance as the first therapy to show statistically significant improvement in heart failure outcomes in adults with heart failure with preserved ejection fraction. vice president, Product Development, Lilly.

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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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Clinical Development Therapies Disease Research 52

Advarra

FEBRUARY 24, 2023

How do you know your experimental therapy is working? But what does “working” really mean when efficacy means something subjective, like quality of life, reduced psychiatric events, or less symptoms? All key questions to consider and address before the therapy even leaves the lab.

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Clinical Trials Trials FDA Therapies 52

Conversations in Drug Development Trends

DECEMBER 20, 2023

Single-Arm Trial Design Single-arm trials have also become an increasingly common development strategy to support regulatory approval and allow patients expedited access to novel therapies, particularly in the accelerated access setting.

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Clinical Trials Trials Pharmacokinetics FDA 83

LifeSciVC

APRIL 24, 2024

There is a continuum of evidence for a given target – at one end are novel targets with some evidence of importance in disease, and at the other end are “de-risked” targets where the biology is precedented with an approved product or late-stage clinical asset(s). with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g.,

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Production Small Molecule Regulations Trials 111

The Pharma Data

JANUARY 27, 2021

The two-hour event will cover various topics, including an overview of the Company’s board and executive leadership, its commitment to environment, social and governance (ESG) matters, and progress updates on the Company’s critical care programs, manufacturing and distribution approach, and commercial readiness plans. About MultiStem ®.

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Clinical Trials Trials Therapies Production 52

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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Treatment Bioinformatics Clinical Development Therapies 52

The Pharma Data

JANUARY 20, 2021

The CEC comprises clinical experts in cardiology and Intensive Care and has been established to ensure accurate and consistent assessment of the trial endpoints and/or serious adverse events. In order to ensure an unbiased endpoint assessment, members of the CEC are blinded to treatment assignment. About Cardiol Therapeutics.

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Hospitals Trials International Clinical Trials 52

The Pharma Data

APRIL 1, 2021

In 2017, Tecartus was granted Breakthrough Therapy Designation by the FDA for relapsed or refractory adult B-cell precursor ALL. If approved, Tecartus would become the first and only chimeric antigen receptor (CAR) T-cell therapy approved for adults (?18 Tecartus is an autologous, anti-CD19 CAR T cell therapy. About Tecartus.

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Treatment Virus Therapies FDA 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. Corneal events were reported more frequently in the bemarituzumab arm (67.1%

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Trials Treatment Therapies Clinical Trials 52

The Pharma Data

DECEMBER 29, 2020

HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced that the company was officially included into the Shenzhen-Hong Kong Stock Connect program (the “Hong Kong Stock Connect”).

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Clinical Development FDA Regulations Clinical Trials 52

The Pharma Data

SEPTEMBER 6, 2021

In the study, eliapixant demonstrated a positive benefit-risk profile, with the vast majority of adverse events considered mild or moderate. Discontinuation of the study due to adverse events occurred in 8% of patients treated with eliapixant. These results will guide us to advance our clinical development strategy of eliapixant.”.

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Trials Clinical Trials International Treatment 52

The Pharma Data

MAY 27, 2022

of patients refractory to their initial therapy and about one-third (33.1%) having received prior CAR T-cell therapy.¹ Cytokine release syndrome (CRS) was the most common adverse event occurring in 63.0% Incidence of Grade 3+ CRS was low (3.9%), with no Grade 5 events.¹. After a median follow-up of 12.6 months, 39.4%

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Treatment Therapies Clinical Trials Disease 52

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. These adverse events suggest widespread, uncoordinated and nonspecific immune activation.

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Immune Response Production Regulations Clinical Trials 88

Advarra

JUNE 20, 2023

Additionally, in 2021, a literature review estimated the average capitalized research and development (R&D) costs per new cancer medicine at between $944 million and $4.54 Typical clinical development timelines for anticancer drugs average an estimated 6.7 This means study startup times can vary greatly.

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Trials Clinical Trials FDA Approval Therapies 52

The Pharma Data

AUGUST 30, 2021

years versus placebo when added to maximum tolerated dose of guideline-directed therapy. A kidney outcome event occurred in 360 (5.5%) patients receiving finerenone and 465 (7.1%) receiving placebo. 0.98]; p=0.0264) over a median duration of follow-up of 3.4 0.88]; p=0.0002). 0.98]; p=0.0264) over a median duration of follow-up of 3.4

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Disease Clinical Trials Hospitals Therapies 52

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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Disease Trials Treatment Clinical Trials 52

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? Diseased cells are likely to be more fragile or less abundant, making it difficult to obtain enough for therapy.

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Therapies Immune Response Clinical Trials Disease 52

The Pharma Data

JUNE 25, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for lecanemab (BAN2401), an investigational anti-amyloid beta (A?) Breakthrough Therapy designation is an FDA program intended to expedite the development and review of medicines for serious or life-threatening conditions. About Eisai Co.,

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Disease Treatment Therapies FDA 40

The Pharma Data

JUNE 28, 2021

Senior Vice President and Head of Oncology Development at Bayer. “We We are excited about the potential of this combination therapy based on the findings from CHRONOS-3 and we look forward to working with global regulatory authorities.”. Fields, M.D., In China, the filing for this indication was granted priority review.

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Treatment Therapies Trials Clinical Development 52

The Pharma Data

AUGUST 11, 2020

.–( BUSINESS WIRE )– Bristol Myers Squibb (NYSE: BMY) today announced the Phase 3 CheckMate -577 trial evaluating Opdivo (nivolumab) as an adjuvant therapy for patients with resected esophageal or gastroesophageal junction (GEJ) cancer met its primary endpoint of disease-free survival (DFS) at a pre-specified interim analysis.

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Therapies Trials Disease Treatment 52

The Pharma Data

MARCH 4, 2021

Baricitinib has received Breakthrough Therapy designation from the U.S. No deaths, major adverse cardiovascular events (MACE) or venous thromboembolic events (VTEs) were reported in the study. vice president of immunology development at Lilly. Food and Drug Administration (FDA) for the treatment of AA.

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Trials Therapies Treatment Clinical Trials 52

The Pharma Data

JUNE 7, 2023

Rahway, NJ, USA), an anti-PD-1 therapy. Grade 3 or greater treatment-related adverse events (TRAEs) occurred in 31% of patients receiving doublet therapy and 58% of patients receiving triplet therapy. The percentage of ILD or pneumonitis events was similar across cohorts.

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Trials Protein Expression Therapies Treatment 40

DrugBank

SEPTEMBER 26, 2024

While some clinical trials are necessary to confirm safety and efficacy in humans, these are typically smaller and shorter in duration compared to the extensive trials required for novel biopharmaceuticals. These molecules pose major scientific and regulatory hurdles due to their intricate structures and potential for immunogenicity.

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Biosimilars Clinical Trials Production FDA 74

The Pharma Data

NOVEMBER 8, 2021

The cardiology ballot at Bayer formerly includes a number of products and several other composites in colorful stages of preclinical and clinical development.

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Clinical Trials Trials Therapies Treatment 52

The Pharma Data

DECEMBER 17, 2020

Senior Vice President and Head of Global Clinical Development at Regeneron and lead author of the publication. patients under an Emergency Use Authorization, and we also continue a robust clinical development program.” In the initial 275 patients, rates of adverse events (AEs) were similar among groups.

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Hospitals Production Clinical Trials Therapies 52

The Pharma Data

OCTOBER 29, 2020

In the Phase 2 portion, the most common adverse event was upper respiratory tract infection which occurred in 17.9%, 11.1% No serious adverse events, deaths, thrombotic events or new safety concerns were reported. vice president of immunology development at Lilly. vice president of immunology development at Lilly.

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Therapies Treatment Disease Clinical Trials 52

The Pharma Data

JANUARY 10, 2021

About MorphoSys MorphoSys is a commercial-stage biopharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for patients suffering from cancer and autoimmune diseases.

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Clinical Trials Clinical Development Clinical Supply Trials 52

Conversations in Drug Development Trends

APRIL 5, 2024

Each day throughout the month of February, we heard what was on the minds and hearts of patients and patient advocacy organizations, clinical developers driving treatments, and family members supporting people with rare diseases. We were overwhelmed by how excited the community was to participate!

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Disease Clinical Trials Trials Treatment 52

The Pharma Data

JULY 22, 2021

This should significantly enhance and accelerate the development and potential commercialization of ARV-471 while also advancing Arvinas’ strategy of building a global, integrated biopharmaceutical company,” said John Houston, Ph.D., Chief Scientific Officer for Oncology Research and Development at Pfizer.

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Targeted Protein Degradation Therapies Clinical Trials Treatment 52

The Pharma Data

JANUARY 19, 2021

The Company’s business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.

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Clinical Trials Trials Clinical Development Pharmaceutical Companies 52

Drug Target Review

JULY 1, 2024

Overall, SRP-001’s modulation of pain signalling genes and pathways through endocannabinoid enhancement and FAAH inhibition supports its potential as an effective non-opioid pain therapeutic, validating the planned clinical trials. was founded in 2016 to develop safer, non-opioid therapies for acute and chronic pain.

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Treatment Clinical Trials Therapies Molecular Biology 59

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. Key Takeaway RWD and RWE play an important role in rare diseases, where capturing relevant data to support drug development is challenging.

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