Galien week of innovation

Product: GAVRETO^®

Company: Blueprint Medicines

Therapeutic Area: Pneumology

About the Product:

Discovered by Blueprint Medicines and under a collaboration with Roche, GAVRETO® (pralsetinib) is a kinase inhibitor approved by the FDA for the treatment of adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA approved test, adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant MTC who require systemic therapy, and adults and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).

Product: TAZVERIK^®

Company: Epizyme Inc.

Therapeutic Area: Oncology - Hematology

About the Product:

Product: Janssen Pharmaceutical Companies of Johnson & Johnson

Company: RYBREVANT® (amivantamab-vmjw)

Therapeutic Area: Pneumology

About the Product:

RYBREVANT received accelerated approval in the United States in May 2021 and was the first fully-human, bispecific antibody approved in lung cancer. By binding to two different receptors and providing immune cell-directing activity, RYBREVANT has broadened the scope of patients for whom targeted therapy is available and effective. Beyond EGFR ex20ins mutations, RYBREVANT is being evaluated as monotherapy or in combination with other therapies in patients with NSCLC harboring other mutations.

Research team:

• Sylvie Laquerre
• Sheri Moores
• Amy Roshak
• Kiran Patel
• Roland Knoblauch
• Meena Thayu
• Martin Shreeve
• Patricia Lorenzini
• Dawn Millington
• Nahor Haddish-Berhane
• Joshua Curtin
• Francis Meacle
• Leonid Freytor
• Julie Man
• Maria Kan
• Jason Lanier

Product: Braftovi® Mektovi®

Company: Pfizer, Inc.

Therapeutic Area: Oncology - Hematology

About the Product:

Description Metastatic melanoma is one of the most serious and life-threatening types of skin cancer. There are a variety of gene mutations that can lead to metastatic melanoma, the most common being BRAF, which occurs in approximately half of patients with unresectable or metastatic disease. BRAFTOVI® (encorafenib) and MEKTOVI® (binimetinib) are kinase inhibitors indicated for use in combination for the treatment of patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation as detected by an FDA-approved test. BRAFTOVI is not indicated for treatment of patients with wild type BRAF melanoma.

Product: TIBSOVO®

Company: Servier Pharmaceuticals

Therapeutic: Oncology - Hematology

About the Product:

TIBSOVO is a prescription medicine used to treat adult patients with previously treated locally advanced or metastatic cholangiocarcinoma (CCA) with an isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. TIBSOVO is also approved in the U.S. as monotherapy for the treatment of adults with IDH1-mutated relapsed or refractory acute myeloid leukemia (AML), and for adults with newly diagnosed IDH1-mutated AML who are ≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy. 

Product: LYBALVI®

Company: Alkermes, Inc.

Therapeutic Area: Neurology

About the Product:

LYBALVI® is a combination of olanzapine, an atypical antipsychotic, and samidorphan, an opioid antagonist, indicated for the treatment of schizophrenia in adults and bipolar 1 disorder in adults as an acute treatment of manic or mixed episodes as monotherapy and as an adjunct to lithium or valproate and as maintenance monotherapy. Weight gain has limited the utility of olanzapine, an established and highly effective antipsychotic. Focusing research efforts on a new mechanism while leveraging a body of literature, our development program for LYBALVI sought to reduce olanzapine-associated weight gain with the addition of samidorphan.

Product: RELYVRIO® AMX0035

Company: Amylyx Pharmaceuticals, Inc.

Therapeutic Area: Internal Medecine

About the Product:

RELYVRIO® (also known as AMX0035), an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol (known as ursodoxicoltaurine outside of the U.S.), is approved to treat amyotrophic lateral sclerosis (ALS) in adults in the U.S. and approved with conditions as ALBRIOZA™ for the treatment of ALS in Canada. AMX0035 is being explored for the potential treatment of other neurodegenerative diseases. The formulation of RELYVRIO, ALBRIOZA, and AMX0035 are identical.

Research team:

• Joshua Cohen
• Justin Klee

Product: VABYSMO®

Company: Genentech, a Member of the Roche Group

Therapeutic Area: Endocrinology

About the Product:

Age‑related macular degeneration and diabetic macular edema affect millions of adults worldwide. While anti-VEGF therapies, which we pioneered, have been helpful at staving off blindness in retinal vascular diseases, the underlying multifactorial processes of these diseases make their management challenging. Vabysmo offers a new class of molecular and technological innovation that targets two pathological pathways (VEGF and Ang2) within a single molecule. Vabysmo provides robust vision improvement and maintenance, while requiring dosing as infrequently as once every four months, compared to every four or eight weeks of the previously available therapies, thus dramatically improving our patients’ quality of life.

Product: JORNAY PM®

Company: Ironshore Therapeutics

Therapeutic Area: Neurology

About the Product:

JORNAY PM is a first-in-class evening-dosed methylphenidate indicated for the treatment of Attention Deficit Hyperactivity Disorder (ADHD) in patients 6 and older. Unique gradual absorption in the colon underlies key characteristics of JORNAY PM: (1) it is the only oral long-acting methylphenidate that achieves therapeutic levels upon waking; and (2) exposure can be personalized in a smooth, dose-dependent manner. Therefore, the duration of ADHD symptom control can be tailored by simply titrating to a single dose of JORNAY PM that works when the patient wakes and continues to last until evening.

Jessica is a Chief Digital Officer turned managing director, board member and investor adept at deploying technology to make meaningful advancements for both business and society. She was one of the first chief digital officers for the life sciences industry, leading the global digital transformation for Bayer A.G. across the Pharmaceutical, Consumer Care, Crop Science and Animal Health businesses.

Her data-focused career includes regulatory, market access, record-breaking product launches and enterprise readiness. After almost a decade at Bayer, Jessica moved back to the US and advised both Fortune Global 100 companies and industry disrupting start-ups on digital innovation across the life sciences continuum, including drug discovery, clinical development, decentralized research and care, reg-tech, real world data, digital biomarkers, digital therapeutics, data integration and analytics, marketing, market access, and business strategy. During the pandemic, she joined Europe’s fastest growing digital health company, Huma, as Managing Director, establishing a U.S. team and expanding into decentralized clinical trials for international Covid-19 vaccine studies. Jessica was a Partner at a Boston based venture capital firm and also raised a women’s health fund. She serves on the board of Sage Therapeutics, Pluto Health, and Angelini Ventures.

Jessica is recognized as a 2023 Digital Health “Icon” by Rock Health, one of the top 100 CDOs, and a leading health tech influencer. She serves on the Yale Ventures Blavatnik board and the Yale IRB, and previously was on the United Nations ITU advisory board, and the BHP Technology Advisory Board. Federer began her public health career as an analyst at the Agency for Healthcare Research and Quality in the US Department of Health and Human Services. She earned a Bachelor of Science from the George Washington University, and a Master of Public Health from Yale.

Product: Sunlenca®

Company: Gilead Sciences, Inc.

Therapeutic Area: Infectiology

About the Product:

Sunlenca® (lenacapavir) is a first-of-its-kind capsid inhibitor that offers a new option to help adults with multi-drug-resistant HIV achieve and maintain viral suppression. Its unique mode of action, picomolar potency, and long half-life, allowing dosing once every six months, represent a significant breakthrough for heavily treatment-experienced individuals with multi-drug-resistant HIV. Beyond the immediate impact for this population, the distinctive properties of lenacapavir lay the groundwork for a new era of long-acting, person-centric HIV treatment and prevention options that will meet the diverse needs of people with HIV and those who may benefit from pre-exposure prophylaxis (PrEP).

Research team:

• John Link
• Winston Tse
• Scott Schroeder
• Jim Zheng
• Tomas Cihlar
• John Somoza
• Eric Hu
• Roman Sakowicz
• Anita Niedziela-Majka
• Latesh Lad
• Anne Chester
• William Rowe
• Anna Chiu
• Jenny Sager
• Martin Rhee
• Scott Schroeder
• Roshy Pakdaman
• Jared Baeten
• Stephen Yant

Dr. Michael Ringel has been at BCG 25 years and is currently Managing Director and Senior Partner.

During his time at BCG, he has worked on a range of strategic, operational and organizational topics in biopharma, with a TED talk that can be viewed at TED.com.

He is also an active advocate for longevity science and innovation, sitting on the Boards of Hevolution US and AFAR.

Dr. Ringel 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: LUPKYNIS®

Company: Aurinia Pharmaceuticals Inc.

Therapeutic Area: Gastroenterology - Hepatology

About the Product:

LUPKYNIS® is the first U.S. FDA-approved oral medicine for the treatment of adult patients with active lupus nephritis, a serious and life-threatening manifestation of systemic lupus erythematosus, a chronic and complex autoimmune disease. LUPKYNIS is a novel, structurally modified calcineurin inhibitor with a dual mechanism of action, acting as an immunosuppressant through inhibition of T-cell activation and cytokine production and promoting podocyte stability in the kidney. Limitations of Use: Safety and efficacy of LUPKYNIS have not been established in combination with cyclophosphamide. Use of LUPKYNIS is not recommended in this situation.

Research Team:
Robert Huizinga, Research
Neil Solomons, CMO
Mark Abel, CMC
Ron Demers, CMC
Laura Lisk, Clinical Development
Nicole England, Clinical Development
Sue Evans, Regulatory
Larry Mandt, Regulatory
Jeremy Hanman, Regulatory
Laura Veasey, Pharmacovigilance
Jennifer Cross, Experimental Medicine
Kory Engelke, Pharmacology
Matt Truman, Statistics
Toni Coeshall, Clinical Operations

Product: LIVMARLI® (maralixibat) oral solution

Company: Mirum Pharmaceuticals, Inc.

Therapeutic Area: Gastroenterology - Hepatology

About the Product:

LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the FDA for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older and is the only FDA-approved medication to treat this indication. LIVMARLI is currently being evaluated in late-stage clinical studies in other rare liver diseases including PFIC and biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia.

Product: CIBINQO^TM

Company: Pfizer Inc.

Therapeutic Area: Dermatology

About the Product:

Atopic dermatitis (AD) is one of the most common inflammatory skin diseases, affecting people of all ages and genders. CIBINQO™ offers hope to millions of patients 12 years of age and older with moderate to severe eczema who are suffering daily with this immuno-inflammatory condition that can cause intense and persistent itching and skin rash. CIBINQO™ is an oral Janus Kinase inhibitor that targets JAK1. Inhibition of JAK1 is thought to modulate multiple cytokines involved in AD pathophysiology, including interleukin (IL)-4, IL-13, IL-31, IL-22, and thymic stromal lymphopoietin (TSLP). The clinical relevance specific JAK inhibition is not known.

Product: Ocrevus® (ocrelizumab)

Company: Genentech, a Member of the Roche Group

Therapeutic Area: Neurology

About the Product:

Ocrevus® (ocrelizumab) has transformed the MS treatment landscape as the first and only disease-modifying medicine approved by the FDA for both relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS).1 Five years later, it remains the only disease-modifying medicine for PPMS in the U.S.2 Roche’s novel approach to selectively target B cells began a new era for the MS community and redefined our understanding of MS biology.3 We remain dedicated to learning more about underlying MS disease progression and continuing Ocrevus innovation in our ongoing clinical trial program, including an investigational formulation for subcutaneous injection.4,5 References 1. Roche Press Release. FDA approves Roche’s OCREVUS™ (ocrelizumab) for relapsing and primary progressive forms of multiple sclerosis. Available at: https://www.roche.com/media/releases/med-cor-2017-03-29. Published March 2017. 2. Multiple Sclerosis Association of America. Long-Term Treatments for Multiple Sclerosis. Available at: https://mymsaa.org/ms-information/treatments/long-term/. Accessed August 2023. 3. Hauser SL. The Charcot Lecture | beating MS: a story of B cells, with twists and turns. Mult Scler. 2015;21(1):8-21. 4. Roche Press Release. Positive Phase III results for Roche’s OCREVUS (ocrelizumab) twice a year, 10-minute subcutaneous injection in patients with multiple sclerosis. Available at: https://www.roche.com/media/releases/med-cor-2023-07-13. Published July 2023. 5. Roche. Product Development Portfolio. Available at: https://www.roche.com/solutions/pipeline. Accessed August 2023.

Product: TZIELD® (teplizumab-mzwv)

Company: Sanofi

Therapeutic Area: Endocrinology

About the Product:

TZIELD® (teplizumab-mzwv) is an anti-CD3 monoclonal antibody approved by US FDA in 2022 to delay onset of Stage 3 Type 1 Diabetes in patients eight years and older with Stage 2 T1D. TZIELD® ushers in a new era as the first drug approved to delay the clinical onset of an autoimmune disease. In the TN-10 study, a single 14-day course of teplizumab delayed median time to onset of Stage 3 T1D by 25 months longer than placebo in Stage 2 patients. Median follow-up time was 51 months. The approval of TZIELD® represents a tremendous step forward for patients and families.

Research team:

Industry: • Sanofi: Leni Ramos, Francisco Leon, Ann Andre, Gail Comer, Sharon Rowland, Shobha Gopalakrishnan, Katya Gibiansky, Ralph Raymond, Paul Dunford, Donald Jung, Jun Yan, Laura Knecht, Wei Tian, Linda Arterburn, Rob Adamoski, Jackie Brown, Helen Pentikis, Rachel Garner, Mike Hussey, John Dingerdissen and Christina Yi • Ortho/JNJ: Robert Zivin • MacroGenics: Anastasia Daifotis, Ezio Bonifacio and David Carlin Academia: • U. Chicago/UCSF: Jeffrey Bluestone • U. Chicago/Yale: Kevan Herold • Necker/INSERM: Lucienne Chatenoud and Jean-Francois Bach • TrialNet and collaborators: Carla Greenbaum, Jeffrey Krischer, Jay Skyler and Alice Long