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Lumos Pharma’s Patient-Centric Strategy for Rare Disease Drug Development

XTalks

Among these, rare pediatric diseases present unique challenges and opportunities for rare disease drug development. Pediatric growth hormone deficiency (PGHD) is one such condition, affecting a child’s growth and development.

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Sym-021 by Les Laboratoires Servier for Solid Tumor: Likelihood of Approval

Pharmaceutical Technology

Sym-021 is under clinical development by Les Laboratoires Servier and currently in Phase I for Solid Tumor. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data.

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ARVINAS AND PFIZER ANNOUNCE GLOBAL COLLABORATION TO DEVELOP AND COMMERCIALIZE PROTAC® PROTEIN DEGRADER ARV-471

The Pharma Data

(NYSE: PFE) today announced a global collaboration to develop and commercialize ARV-471, an investigational oral PROTAC® (PROteolysis TArgeting Chimera) estrogen receptor protein degrader. The companies will equally share worldwide development costs, commercialization expenses, and profits. Chief Executive Officer at Arvinas.

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Merck Receives Positive EU CHMP Opinion for KEYTRUDA® (pembrolizumab) together With Chemotherapy surely Patients With Locally Recurrent Unresectable or Metastatic Triple-Negative carcinoma Whose Tumors Express PD-L1 (CPS ?10)

The Pharma Data

Recommendation supported Data From Phase 3 KEYNOTE-355 Trial. Merck is rapidly advancing a broad portfolio in gynecologic and breast cancers through an in depth clinical development program for KEYTRUDA and a number of other other investigational and approved medicines across these areas. About Triple-Negative carcinoma.

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A Complex Chromosomal Abberation Called Adrenoleukodystrophy

Delveinsight

Adrenoleukodystrophy (ALD/ X-ALD) is a rare, X-linked disorder secondary to a mutation in the ABCD1 gene, which encodes for a peroxisomal membrane protein, resulting in an excessive accumulation of very-long-chain fatty acids (VLCFAs) in the brain and adrenal glands. The disorder can occur at any stage of life and vary with severity.

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Three trends in the antibody-drug conjugate (ADC) market 

Drug Discovery World

Roberto Iacone, CEO at Alentis Therapeutics, said: “There are huge unmet needs in organ fibrosis and cancer, and this funding enables us to continue with the important work we’re doing in the CLDN1 space and generate clinical data from both our programs.

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Roche to present a broad range of data across multiple cancer types at the ESMO Virtual Congress 2020

The Pharma Data

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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