Prohost Letter #401 SYNOPSIS PART 1 In this Letter CONTENTS Duchenne Muscular Dystrophy (DMD) FDA Compassionate Approval of Eteplirsen! PART 2 In The Letter Issue #402 CONTENTS EYE ON IONIS (IONS) Antisense Proof of Concept and Feasibility EYE ON INVIVA (INVA) AND THERAVANCE BIOPHARMA (TBPH) Sales of The Ellipta Inhalers Started to Pick up Before an expected Boom Not Losing . . . This content …

Prohost Letter #396 CELEBRATING MEMORIAL DAY - Wish you celebrated the Memorial Day. Memorial Day’s purpose was remembering the people who died while serving in the country’s armed forces. The Memorial Day was initiated as Decoration Day after the Civil War in the year 1868 when the Grand Army of the Republic - an organization of Union Veterans in Illinois established it to decorate the graves …

EXELIXIS AGAIN They said: Sell EXEL, as no partner is interested in the firm’s FDA approved cabozantinib to date. What really happened: It did not take long for IPSEN to come forward and take the responsibility of marketing cabozantinib abroad and pay $200 million upfront payment to Exelixis. They said: Although the FDA approved Cabometyx (cabozantinib) for advanced renal cell carcinoma (RCC), the drug will …

Prohost Letter #388 TOPICS - 1. AN INDISPENSABLE SHORT INTRODUCTION 2. GENE EDITING 3. GENE THERAPY 4. CANCER IMMUNOTHERAPY 1. GENE EDITING Exon Skipping: Biomarin (BMRN); Sarepta (SRPT); These firms are waiting FDA approvals for Duchenne Muscular Dystrophy (DMD) Gene Editing Technology Competitors: Cellectis S.A. (CLLS); CRISPR; PTC Therapeutics (PTCT) Firms Using Different Approaches For DMD Treatment (see Below) Zink Finger Tech: Sangamo (SGMO); RNAi …

Prohost Letter #387 Topic of the Week DUCHENNE MUSCULAR DYSTROPHY Which Firms Will Get It Right – Deleting DMD Children’s Despair? The topic is important because, first and foremost, Duchenne muscular dystrophy (DMD) disables children. Also because, from no treatment at all, now we have several biotech firms developing various drugs based on different scientific strategies. The new approaches have increased in number and have …