Galien week of innovation
OCT. 23-27
RARE/ORPHAN DISEASES - Tuesday Oct. 24
Speakers
4 PM - 5 PM ET I Rare/Orphan Disease Drug
Will progress in medicines that promise a cure for devastating rare diseases lead to similar innovations on patient access to these increasingly high-cost, high-touch therapies?
Kevin Brubacher
Managing Director Bionest, Accenture
Kevin is a Managing Director based in the US, bringing 30 years of innovative commercial and consulting experience to his clients. Combining nearly 20 years of commercial leadership roles in biopharma with over 12 years of hands-on senior leadership in consulting, Kevin supports innovative biotech, pharma, and rare disease companies with commercialization strategy and go to market planning.
Kevin has supported numerous companies in developing and refining their corporate strategy, working side-by-side with senior leadership, promoting alignment, exposing gaps, and pressure-testing ideas. In addition, Kevin has led cross-functional marketing and commercial teams during the development and launch of pharmaceutical products. His expertise includes global commercial planning and market strategy, analytics, market simulation, lifecycle management, launch planning, and competitive gaming. His time in the pharmaceutical industry spans multiple senior commercial roles in the US, Canada, and Europe.
Arup Pramanik
Corporate Vice President, Boehringer Ingelheim
Product: SPEVIGO®
Company: Boehringer Ingelheim
Therapeutic Area: Dermatology
About the Product:
Spevigo® is the first and only clinically validated, FDA-approved treatment for generalized pustular psoriasis (GPP). It directly inhibits IL-36 signaling – the key driver of GPP. Clinical development focused on patient-centric treatment goals. Pivotal clinical endpoints were rapidly achieved with 54% of patients showing pustular clearance within one week of treatment with a favorable safety profile. Recognizing the innovative value, FDA’s Breakthrough Therapy Designation and expedited approval resulted in accelerated availability of a novel intervention for potentially life-threatening acute GPP flares. The breakthrough potential of Spevigo® extends beyond GPP, paving the way for potential advancements in treating other IL-36-mediated conditions.
Bill Mezzanotte
Executive Vice President, Head of Research and Development, CSL / uniQure
Bill Mezzanotte
Executive Vice President, Head of Research and Development, CSL / uniQure
Product: HEMGENIX®
Company: CSL / uniQure
Therapeutic Area: Oncology - Hematology
About the Product:
HEMGENIX® (etranacogene dezaparvovec-drlb) is the first and only FDA-approved gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening bleeding, or have repeated, serious spontaneous bleeding episodes. HEMGENIX has been shown to reduce the rate of annual bleeds versus standard of care, and reduce or eliminate the need for prophylactic treatment in 94% of people who received the therapy. This may provide eligible patients with protection from bleeds and freedom from the rigorous schedules required for prophylactic infusions.
Research team:
- Ying Poi Liu, uniQure
- Jaap Twisk, uniQure
- Erich Ehlert, uniQure
- Sander Gielen, uniQure
- Paula Miranda, uniQure
- Lisa Spronk, uniQure
- Paul Monahan, CSL
- Scott Hambaugh, CSL
- Brahm Goldstein, CSL
- Jason Newman, CSL
- Desiree Devonish, CSL
- Jacqueline Tarrant, CSL
Bahija Jallal
CEO, Immunocore
Product: KIMMTRAK®
Company: Immunocore
Therapeutic Area: Ophthalmology
About the Product:
KIMMTRAK® (tebentafusp-tebn) became the world’s first T cell receptor (TCR) therapy, and the first and only FDA-approved treatment for metastatic uveal melanoma – a disease which, until 2022, had an anticipated median overall survival of approximately 12 months. KIMMTRAK is a soluble bispecific protein comprised of an engineered TCR targeting gp100, an antigen expressed in melanocytes and melanoma, to an anti-CD3 immune-effector function, which attracts and activates T cells to kill tumor cells. KIMMTRAK is the first therapy developed using Immunocore’s pioneering ImmTAX® (Immune Mobilizing Monoclonal TCRs Against X disease) platform, a first-in-class immunotherapy platform with a novel mechanism of action.
Research team:
- Annelise Vuidepot
- Chris Holland
- Cindy Clarke
- Damian Murphy
- David Berman
- Emma Baston
- Giovanna Bossi
- Jane Harper
- Katie Blatt
- Koustubh Ranade
- Mark Moyer
- Mohammed Dar
- Nancy Holdbrook
- Nat Liddy
- Patricia Dickenson
- Peter Molloy
- Shaad Abedin
- Tara Mahon
Monica Kumar
Global Project Head, Sanofi-Aventis U.S. LLC
Product: Xenpozyme®
Company: Sanofi-Aventis U.S. LLC
Therapeutic Area: Internal Medicine
About the Product:
Acid Sphingomyelinase Deficiency (ASMD), historically known as Niemann-Pick disease types A, A/B and B, is rare, progressive genetic disease that can have significant morbidity and can lead to early mortality. Xenpozyme® (olipudase alfa-rpcp) is an enzyme replacement therapy for treatment of non-Central Nervous System (non-CNS) manifestations of ASMD in adult and pediatric patients. Xenpozyme®, the first and only FDA-approved therapy for ASMD, had been under development for over 20 years at Sanofi, a testament to our unwavering commitment to deliver solutions for those who have been waiting a long time for a treatment option. Prior to Xenpozyme® approval, ASMD management was restricted to addressing disease complications and managing symptoms.
Research team:
- Federica Albissola
- Julie Barrier
- Bruce Beyer
- Claudia Buser
- Bee Lin Cheang
- Vanessa Davidson
- Kelly George
- Andreas Jessel
- Jennifer Kelly
- Yong Kim
- Barbara Kittner
- Monica Kumar
- Jing Li
- Blandine Nembo
- Cindy McLean
- Sophie Potdevin
- Sriram Ramakrishnan
- Reinhold Roessler
- Kamalika Saha
- Vanessa Davidson
- Joyce Tay
- Sandrine Turpault
- Abhimanyu Yarrameneni
- Mark Vogt
- Atef Zaher
- Qi Zhang