Galien week of innovation

Alexandra Moens is a PharmD who has been part of the clinical industry for over 6 years. Her aim is to make a positive impact on patient lives by working with innovative solutions, implementing enhanced clinical strategies and increasing diversity and equity in the drug development lifecycle.

Her scientific background and experience working with pharmaceutical partners hand-in-hand have positively impacted clinical teams to get new insights to successfully manage their portfolio strategy in the fast evolving clinical industry.

Product: VYVGART®

Company: argenx

Therapeutic Area & Indication: Immunologic drug for patients with generalized myasthenia gravis causing muscle weakness and loss of motor control.

About the Product:
VYVGART (efgartigimod alfa-fcab) is a human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating immunoglobulin G (IgG) autoantibodies. It is the first and only approved FcRn blocker. VYVGART is approved in the United States and Europe for the treatment of adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive, and in Japan for the treatment of adults with gMG who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs).

Product: Bylvay®

Company: Albireo Pharma, Inc.

Therapeutic Area & Indication: A ileal bile acid transport inhibitor for pediatric and adult patients with progressive familial intrahepatic cholestasis (PFIC) which leads to liver disease and failure.

About the Product:
Bylvay® (odevixibat) is approved in the U.S. for the treatment of pruritus in patients 3 months of age and older with all types of progressive familial intrahepatic cholestasis (PFIC), and in Europe and the UK for the treatment of PFIC in patients aged 6 months or older. This once-daily, non-systemic ileal bile acid transport inhibitor has minimal systemic exposure and acts locally in the small intestine. It can be taken as a capsule or opened and sprinkled onto food. Most common adverse reactions: diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency. For full PI visit www.bylvay.com and www.albireopharma.com.

Product: TAVNEOS® (avacopan)

Company: ChemoCentryx, Inc.

Therapeutic Area & Indication: Adults with severe active and anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis, an auto-immune disorder affecting small blood vessels in the body.  

About the Product:
TAVNEOSÒ (avacopan) is a first-in-class, orally administered small molecule approved by the FDA as an adjunctive treatment for ANCA-associated vasculitis. In the pivotal ADVOCATE study, patients adding TAVNEOS achieved superior remission rates at 52 weeks, experienced lower relapse rates, and improved renal function (eGFR). TAVNEOS is the first therapy approved for patients with ANCA-associated vasculitis in more than 10 years, and the first and only FDA-approved therapy that selectively inhibits the C5a receptor (C5aR).

Product: Zolgensma® (onasemnogene abeparvovec-xioi)

Company: Novartis Gene Therapies

Therapeutic Area & Indication: Treatment of Spinal Muscular Atrophy (SMA) in infants and
adults.

About the Product:
Zolgensma® (onasemnogene abeparvovec-xioi) is the only gene therapy for spinal muscular atrophy (SMA) and the only SMA treatment designed to address the genetic root cause of the disease by replacing the function of the missing or non-working SMN1 gene with a single, one-time dose. As a transformative gene therapy designed to provide long-term benefit, Zolgensma represents a completely new class of SMA treatment. Zolgensma is approved in 40+ countries and regions and as of June 2022 more than 2,300 patients worldwide have been treated with the therapy across clinical trials, managed access programs, and in the commercial setting.

Research team:
Colleagues who developed Zolgensma are no long with Novartis GTx, instead please recognize Novartis Gene Therapies as a whole instead of individual colleagues.

Product: Cablivi®

Company: Sanofi

Therapeutic Area & Indication: Treatment of thrombotic thrombocytopenic purpura (aTTTP).

About the Product:
Cablivi is a first-in-class humanized nanobody therapeutic approved for acquired Thrombotic Thrombocytopenic Purpura (aTTP), a life-threatening autoimmune blood disorder. Cablivi prevents pathological platelet aggregation by blocking glycoprotein Ib receptor on von Willebrand’s Factor (vWF). In clinical trials, treatment with caplacizumab resulted in significantly shorter time to platelet count normalization, lower incidence of a composite endpoint of TTP-related death, recurrence, or a major thromboembolic event, and lower incidence of recurrence. As the world’s first approved nanobody drug, Cablivi addresses significant unmet medical need in aTTP and represents a milestone in pharmaceutical sciences, paving the way for a new class of therapeutics.

Michael Ringel is a Managing Director and Senior Partner at BCG, and BCG’s Global Topic Leader for Growth and Innovation.

He is a frequent author and speaker on R&D and innovation topics. His TED talk on innovation can be viewed at TED.com. He is also Strategic Advisor to Life Biosciences, a company targeting aging biology to address multiple diseases.

Prior to BCG, Michael worked in academia, pursuing research in theoretical population dynamics and conducting field experiments in the Amazon basin near Manaus, Brazil. Michael holds a B.A. summa cum laude in biology from Princeton, a Ph.D. in biology from Imperial College, and a J.D. cum laude from Harvard Law School.

Product: Brukinsa®

Company: BeiGene

Therapeutic Area & Indication: Kinase inhibitor for adult patients with mantle cell and marginal zone lymphoma as well as the rare blood cancer, Waldenstrom macroglobulinemia.

About the Product:
BRUKINSA® (zanubrutinib) is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver targeted and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.

Researcher:
Lusong Luo

Product: Abecma® (idecabtagene vicleucel)

Company: Bristol Myers Squibb and 2seventy bio

Therapeutic Area & Indication: CAR-T personalized cell therapy for multiple myeloma.

About the Product:
Developed in partnership with bluebird bio (now 2seventy bio), Abecma is the first-in-class BCMA-directed CAR T cell immunotherapy for the treatment of adult patients with relapsed or refractory MM after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 monoclonal antibody. As the first CAR T cell therapy approved for MM and the first anti-BCMA CAR T, Abecma is a transformative, single-infusion, individualized treatment that has changed the treatment paradigm and set a new standard of care by offering patients who have limited effective treatment options the potential for long-term disease control.

Product: ENHERTU®

Company: Daiichi Sankyo and AstraZeneca

Therapeutic Area & Indication: Treatment of breast cancer.

About the Product:
ENHERTU® is a HER2 directed antibody drug conjugate (ADC) designed using Daiichi Sankyo’s proprietary DXd ADC technology and is jointly developed and commercialized by Daiichi Sankyo and AstraZeneca. ENHERTU is FDA approved for the treatment of certain types of breast, lung and gastric cancers. 

Daiichi Sankyo and AstraZeneca have a comprehensive development program underway globally, focused on evaluating the efficacy and safety of ENHERTU across HER2 targetable cancers, including breast, gastric, lung and colorectal cancers. Trials in combination with other anticancer treatments, such as immunotherapy, are also underway.

Product: Rylaze®

Company: Jazz Pharmaceuticals

Therapeutic Area & Indication: Injectable multi-agent chemotherapeutic agent for treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adults and pediatric patients.

About the Product:
Rylaze® is indicated as a component of a multiagent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients who have developed hypersensitivity to E. coli-derived asparaginase. With a worldwide shortage of an alternative non-E. coli-derived treatment causing disruptions in product availability, Jazz partnered with the Children’s Oncology Group to accelerate the development of Rylaze to ensure patients could complete their full treatment course. Rylaze represents a significant advancement through recombinant, modern manufacturing, which results in a ready-to-use, high concentration formulation with reliable supply, ensuring uninterrupted therapy for patients.

Alexandra Moens is a PharmD who has been part of the clinical industry for over 6 years. Her aim is to make a positive impact on patient lives by working with innovative solutions, implementing enhanced clinical strategies and increasing diversity and equity in the drug development lifecycle.

Her scientific background and experience working with pharmaceutical partners hand-in-hand have positively impacted clinical teams to get new insights to successfully manage their portfolio strategy in the fast evolving clinical industry.

Product: VAXNEUVANCE

Company: Merck & Co., Inc.

Therapeutic Area & Indication: Pneumococcal conjugate vaccine to immunize against 15 types of invasive pneumococcal bacteria, including blood infections and in meningitis affecting brain and spinal cord.

About the Product:
Streptococcus pneumoniae remains associated with significant residual disease burden. VAXNEUVANCE is a novel pneumococcal conjugate vaccine (PCV) approved for individuals 6 weeks of age and older for the prevention of invasive disease (e.g. meningitis, bloodstream infection) due to 15 pneumococcal serotypes. Administered as a single dose in adults and a four-dose series in young children, it maintains robust immunogenicity for all serotypes shared with the 13-valent PCV (PCV13) while inducing significantly greater immune responses for shared serotype 3 and unique serotypes 22F and 33F, key contributors to remaining disease. VAXNEUVANCE represents a bold innovation in the manufacturing of glycoconjugates.

Product: TEPEZZA® (teprotumumab-trbw)

Company: Horizon Therapeutics

Therapeutic Area & Indication: Treatment of Thyroid Eye Disease (TED).

About the Product:
TEPEZZA® is a fully human monoclonal antibody (mAb) and a targeted inhibitor of the insulin-like growth factor-1 receptor (IGF-1R) indicated for the treatment of Thyroid Eye Disease (TED). TEPEZZA® is the first and only FDA-approved medicine to treat TED, a serious, progressive and vision-threatening rare autoimmune disease. People living with TED often experience long-term functional, psychosocial and economic burdens, including inability to work and perform activities of daily living. TEPEZZA® treats TED at the source – a first for patients and doctors. As an infused biologic, TEPEZZA® completely changed how physicians treat patients living with TED.

Product: PreHevbrio™

Company: VBI Vaccines, Inc.

Therapeutic Area & Indication: Prevention of infection caused by all known subtypes of hepatitis B virus. PreHevbrio is approved for use in adults 18 years of age and older.

About the Product:
PreHevbrio™ [Hepatitis B Vaccine (Recombinant)] is the only 3-antigen adult prophylactic hepatitis B vaccine that contains the full antigenic composition of the hepatitis B surface antigen, including the S, Pre-S2, and Pre-S1 hepatitis B surface antigens. PreHevbrio was approved in the U.S. in late 2021, in the EU/EEA/UK in 2022 under the brand name PreHevbri®, and in Israel in 2000 under the brand name Sci-B-Vac®.

Product: XVIVO Perfusion System (XPS™) with STEEN Solution™

Company: XVIVO Perfusion, Inc.

Therapeutic Area & Indication: For the flushing and temporary continuous warm machine perfusion of donor lung transplant organ to allow clinicians to evaluate and/or reassess its acceptability for transplant in patients with end-stage lung disease.

About the Product:
The XPS™ with STEEN Solution™ is a fully integrated cardiac bypass system that uses innovative technology to perform normothermic ex-vivo lung perfusion (EVLP). EVLP is an advanced preservation technique where donor lungs are kept alive outside the body via perfusion and ventilation in an optimized environment so that a surgeon can properly assess, and potentially restore, lungs that may have been damaged at the time of the donor’s death. STEEN Solution™ is a buffered extracellular solution optimally designed to facilitate prolonged evaluation and promote stability of isolated lungs ex vivo without edema formation.