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Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Symptoms and Etiology: Caused by an overproduction of growth hormone, acromegaly primarily affects adults. Acromegaly. Prevalence: 50 to 70 people per 1 million.

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What Innovative Treatments For Metastatic Breast Cancer are being developed?

Find Me Cure

There currently are drugs that do just that but now they’re in clinical trials for metastatic breast cancer as well. CDK4/6 inhibitors are used alongside hormone therapy. There are antibody drugs targetting those cells that are already FDA approved and there are antibody drugs still in trials. Rosenberg, M.D.

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Where are the advances in treatments for triple-negative breast cancer?

Drug Discovery World

However, because these cancers are not driven by any of the three molecules that can be blocked by targeted hormone receptor drugs, current treatment options are limited. The addition of pembrolizumab was supported by further trial results in 2022. TVEC is approved to treat advanced, late-stage melanoma.

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Monthly News Roundup – December 2020

The Pharma Data

The vaccine teaches the cells to make a piece of the spike protein, which triggers an immune response to help prevent illness if later exposed to the virus. In mid-December, the FDA approved Orgovyx (relugolix), the first oral gonadotropin-releasing hormone (GnRH) receptor antagonist for men with advanced prostate cancer.

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FDA Approves Merck’s KEYTRUDA® (pembrolizumab) in Combination With Padcev® (enfortumab vedotin-ejfv) for First-Line Treatment of Certain Patients With Locally Advanced or Metastatic Urothelial Cancer

The Pharma Data

Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. This marks the first time an anti-PD-1 therapy has been approved in combination with anantibody-drug conjugate in the U.S. confirmatory trial for the accelerated approval.

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CheckMate -577, a Phase 3 Trial Evaluating Opdivo (nivolumab) as Adjuvant Therapy for Patients with Resected Esophageal or Gastroesophageal Junction Cancer, Meets Primary Endpoint of Disease-Free Survival

The Pharma Data

.–( 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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CheckMate -649, a Phase 3 Trial Evaluating Opdivo (nivolumab) Plus Chemotherapy vs. Chemotherapy, Meets Primary Endpoints Demonstrating Superior Overall Survival and Progression-Free Survival in First-Line Treatment of Gastric and Esophageal Cancers

The Pharma Data

The safety profiles of Opdivo and chemotherapy in this trial are reflective of the known safety profiles of Opdivo and chemotherapy in first-line gastric and esophageal cancers. The company remains blinded to data from this arm and the trial continues in follow-up to allow the data to mature. PRINCETON, N.J.–(

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