Webinar Program on Nominees for best biotechnology Product

TUESDAY, October 27, 2020

1.WEBINAR

to mark the Prix galien usa’s upcoming october 26 “Week of discovery and celebration on the Frontiers of medical innovation,” distinguished members of the usa galien awards committee will chair three webinars highlighting each of the 23 nominee products they have selected for the best biotechnology category. these virtual expert panelist exchanges will offer the opportunity for webinar participants to assess the current state of innovation in biotech, covering key aspects of clinical progress and differentiation; the ways that patient care is now being conducted outside traditional delivery settings; and the next wave of promising breakthrough technologies, including their transformative – and potentially disruptive – effect on health care productivity and outcomes worldwide.

calendarOCT 26-29 2020

The biotech sector continues to pull more than its weight in developing new medicines, a trend that, even in the midst of a global pandemic, has drawn vast amounts of new capital to fund pipeline growth in areas ranging from cell and gene therapy to the microbiome. This year has already broken the all-time record for IPO placements and valuations remain high even for those companies still seeking to launch their first marketed product. Still, small biotech must compete for visibility and resources against the global established players in big pharma, particularly for those with a therapeutic focus on medicines outside the current frenzied search for solutions to the coronavirus pandemic.

category:> chronic conditions

• Where is the growing science and its link to disease leading us in terms of new treatments for patients with unresolved chronic conditions?

Chair: Dr Michael ROSENBLATT, M.D., Chief Medical Officer of Flagship Pioneering, Cambridge, MA

PRODUCT COMPAGNY ThERAPEUTIC AREA & INDICATION

Liletta (levonorgestrel-releasing intrauterine system) Medicines360 IUD to prevent pregnancy through timed release of hormone levonorgestrel.

Aimovig (cerenubab-aooe) Amgen CGRP receptor blocker to treat chronic migraine.

Evenity (romosozumab-aqqg) Amgen Osteoporosis in post-menopausal women at high risk of bone fracture.

Emgality (galcanezumab-ghlm) Eli Lilly Once a month CGRP antibody preventive treatment for adult migraine.

Tremfya (guselkumab) Janssen Research & Development, LLC Medium to severe plaque psoriasis in adults.

Rybelsus (semaglutide) Novo-Nordisk AS GLP-1 agonist treatment to control blood sugar in adults with Type-II diabetes.

Dupixent (dupilumab) Regeneron Pharmaceuticals Single dose pre-filled pen technology to treat adults and adolescents with atopic dermatitis (eczema), asthma and rhinosinusitis with nasal polyposis.

tuesday, october 27, 2020 - 11:00 am - 12:00 pm et

the sessions will be chaired by a member of the Prix galien usa committee and will include a representative of the research team for each nominee product

calendarOCT 26-29 2020

category:> cancer, other rare conditions and vaccines

2.WEBINAR

• Can the foundational biologic understandings now being brought to cancer care be leveraged to secure a targeted, more personalized approach to other conditions?

PRODUCT COMPAGNY ThERAPEUTIC AREA & INDICATION

Enhertu (fam-trastuzumab deruxtecan) Daiichi-Sankyo Pharmaceuticals Treatment of unresectable or metastatic HER2 positive breast cancer.

Polivy (polatuzumab-vedotin-piiq) Roche-Genentech Antibody drug conjugate for treatment of diffuse large B-cell lymphoma.

Ervebo (VSV-EBOV) Merck & Co. First preventive vaccine against the disease caused by the Zaire ebolavirus in adults.

Oxervate (cenegermin-bkbj) Dompe Pharmaceuticals Recombinant human growth factor to treat neurotrophic keratitis, a rare condition affecting the eye cornea.

Takhzyro (lanadelumab-flyo) Takeda Pharmaceuticals Treatment for hereditary angioedema (HAE) in adults.

Cablivi (caplacizumab-yhdp) Sanofi Nanodrug to treat adult acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood disorder.

Hemlibra (emicizumab-kxwh) Roche-Genentech Reduce or prevent bleeding episodes in adults and children with hemophilia A, with or without factor-VIII inhibitors.

Andexxa (coagulation factor XA) Portola Pharmaceuticals Reversal of medicine-specific, anticoagulant-based, and life-threatening bleeding events.

3.WEBINAR

category:> rare genetic disorders

•Will progress in therapies that promise a genetic cure for devastating rare diseases lead to similar innovations in patient access to these high-cost, high-touch treatments?

Chair: Pr Mary-Claire KING, American Cancer Society Professor of Genome Sciences and Medical Genetics at the University of Washington

PRODUCT COMPAGNY ThERAPEUTIC AREA & INDICATION

Tegsedi (inotersen) Aksea Therapeutics and Polyneuropathy caused by hereditary transthyretin-amyloidosis Ionis Pharmaceticals (hATTR) in adults.

Ultomiris (cravulizumab-cwvz) Alexion Pharmaceuticals Paroxysmal nocturnal hemoglobinuria (PNH) that destroys red blood cells in adults.

Givlaari (givosiran) Alnylam Pharmaceuticals Acute hepatic porphyria (AHP) linked to production of toxic porphyrin molecules in the bloodstream in adults.

Onpattro (patisiran) Alnylam Pharmaceuticals Polyneuropathy caused by hATTR amyloidosis leading to loss of nerve and organ function.

Galafold (migalastat) Amicus Therapeutics Oral drug for treatment of Fabry Disease through repair of gene-induced enzyme deficiencies that lead to organ failures, stroke and early death in adults.

Palynziq (pegvaliase-pqpz) BioMarin Pharmaceutical Lowers blood levels of phenylalanine, an amino acid that causes PKU (phenylketoneuria) leading to brain damage and seizures beginning in infancy.

Oxbryta (voxelotor) Blood Therapeutics Prevents inherited propensity for the hemoglobin polymerization that causes sickle cell disease.

Zolgensma (onasemnogene Novartis AG One-time gene replacement therapy for children under age 2 with spinalabeparvovec-xioi) muscular atrophy.

tuesday, october 27, 2020 - 01:15 - 02:30 pm et

tuesday, october 27, 2020 - 12:00 - 01:15 pm et