Galien week of innovation

Paul Adam B.Sc. (Hons) Ph.D. was born in Dumfriesshire, Scotland and was educated at Dundee University (B.Sc with Honors in Biochemistry) and Cambridge University (Ph.D. in Biochemistry). His subsequent academic career included post-doctoral research positions in at the University of Virginia and Cambridge University where he became a Research Fellow of Wolfson College.

In 2000 Dr. Adam began his career in the biopharmaceutical industry at Oxford Glycosciences and then Celltech where he was responsible for oncology biologics research and therapeutic antibody programs.  

Since joining Boehringer Ingelheim in 2005 as Head of Antibody Drug Discovery Dr. Adam has held several roles of increasing responsibility and has led the discovery and development of numerous biological drugs that have advanced into clinical trials for the treatment of cancer. Dr. Adam is currently Head of Cancer Immunology & Immune Modulation at Boehringer Ingelheim’s oncology research campus in Vienna, Austria.

Product: ASPARLAS®

Company: Servier Pharmaceuticals

Therapeutic Area & Indication: Treatment of acute lymphoblastic leukemia.

About the Product:
ASPARLAS is indicated as a component of a multiagent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) in pediatric and young adult patients aged 1 month to 21 years. This next generation ALL treatment is engineered for sustained asparaginase activity and longer asparagine depletion. A more stable bond, ASPARLAS offers the option of 3-week asparaginase dosing when used as part of a multicomponent chemotherapy regimen. ASPARLAS has a longer half-life, resulting in prolonged suppression of plasma asparagine, and 36-month shelf life, which facilitates long-term planning to ensure product availability.

Product: XPOVIO®

Company: Karyopharm Therapeutics, Inc.

Therapeutic Area & Indication: Treatment of multiple myeloma.

About the Product:
XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm's Selective Inhibitor of Nuclear Export (SINE) compounds to be approved for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications. XPOVIO (also known as NEXPOVIO® in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, China, South Korea, Canada and Israel, and is marketed in those areas by Karyopharm's global partners.

Sanjay has a broad and deep expertise in the underpinning science and business challenges in developing and launch-ing both in-vivo and ex-vivo cell & gene therapies.

He directs his C&GT team at Accenture, who has global expertise in R&D, supply chain, manufacturing, and commercial operations.

Product: LUMAKRAS®

Company: Amgen, Inc.

Therapeutic Area & Indication: Oral gene-based treatment for patients with KRAS G12C-positive cases of advanced or metastatic non-small cell lung cancer.

About the Product:
LUMAKRAS is indicated for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-ap¬proved test, who have received at least one prior systemic therapy. The launch of LUMAKRAS marks the culmi¬nation of a 40-year quest to “drug the undrug-gable.” Amgen took on one of cancer’s grea¬test challenges with unprecedented speed and delivered an innovative, first-in-class medicine that changed the NSCLC treatment paradigm. Amgen’s pioneering work with LUMAKRAS continues, with significant potential beyond NSCLC. The comprehensive CodeBreaK clinical development program is evaluating LUMAKRAS in patients with the KRAS G12C mutation to address the longstanding unmet medical need for some of the most difficult-to-treat cancers, including colorectal and pancreatic cancer.

Product: Pemazyre® (pemigatinib)

Company: Incyte Corp.

Therapeutic Area & Indication: Treatment of bile duct cholangiocarcinoma for patients with FGFR2 gene whose cancer has spread.

About the Product:
Pemazyre, a fibroblast growth factor receptor (FGFR) inhibitor, is indicated for the treatment of adults with relapsed or refractory previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or other rearrangement as detected by an FDA-approved test. This indication is approved under accelerated approval based on overall response rate and duration of response (DOR). Pemazyre is also the first targeted treatment approved for use in the United States for treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement.

Product: Braftovi® Mektovi®

Company: Pfizer, Inc.

Therapeutic Area & Indication: Treatment for metastatic melanoma.

About the Product:
Braftovi® (encorafenib) is a selective ATP-competitive small-molecule BRAF(V600E) kinase inhibitor. Mektovi® (binimetinib) is a selective allosteric, ATP-uncompetitive inhibitor of mitogen-activated protein kinase kinase (MEK) 1 and MEK 2. Braftovi® and Mektovi® were developed as a doublet based on scientific rationale that MAPK pathway reactivation is implicated in BRAF-inhibitor monotherapy resistance. These drugs target two different kinases in the RAS/RAF/MEK/ERK pathway. Compared with either drug alone, co-administration of results in greater anti-proliferative activity in BRAF mutation-positive cancer cells in vitro and in vivo. Additionally, this doublet delayed the emergence of drug resistance in BRAF V600E-mutant human melanoma xenografts in mice.

Product: RYBREVANT®

Company: Janssen

Therapeutic Area & Indication: Antibody-based therapy for adults with metastatic and/or EGRF gene mutation-driven non-small cell lung cancer (NSCLC).

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: TIBSOVO®

Company: Servier Pharmaceuticals

Therapeutic Area & Indication: Elderly adults with newly diagnosed acute myeloid leukemia (AML) unable to tolerate chemotherapy.

About the Product:
Cholangiocarcinoma (CCA), a rare and aggressive cancer, forms in the bile ducts inside and around the liver. IDH1 mutations are common genetic alterations in CCA and can play a critical role in disease development. Prior to the approval of TIBSOVO, there were no approved targeted therapies available for IDH1-mutated CCA, and limited chemotherapy options available in the advanced setting. The first treatment of its kind, TIBSOVO works differently from chemotherapy and targets the IDH1 protein, which can slow down progression of advanced CCA. Convenient, once-daily oral dosing allows for TIBSOVO to be taken at home without chemotherapy infusion time requirements.

Sanskriti (Sans) Thakur is a life sciences leader focused on scientific development and corporate/commercial strategy, with early stage digital and medical technology management.

Sans is currently the Chief Growth Officer for Medable, the leading digital platform for clinical development. She is advancing the company’s vision of human-centered research, enabling access to clinical trials regardless of geography, income, and race. In her journey with Medable, Sans has helped the company grow over 300%.

Sans serves on the Global Future Council of the World Economic Forum focusing on data and outcomes in healthcare. She is a thought leader in issues ranging from human longevity, modernizing healthcare and patient advocacy. She is the author of over 18 publications in the areas of science, business, and technology intersections.

Before Medable, Sans served as Global Head Life Sciences Research for Accenture, responsible for market-shaping strategy and research in therapeutics, digital health, and business model for Accenture’s multibillion-dollar life science enterprise.

During her 20+ years in the life sciences industry, Sans has advised more than 30 companies, launched 10 products, advised digital health venture funds, and managed a leading pharmaceutical portfolio. Execution in both industry and strategy consulting has led to her success in advising and leading multinational start-up organizations, from build to scale.

Sans is a strong advocate for women’s health and changing the future for women and girls through technology. She has an MBA from NYU Stern School of Business, degrees in Chemistry and Biology from Union College and certificates in cancer management, and innovation. Sans lives in New York with her husband and two children.

Product: LYBALVI®

Company: Alkermes, Inc.

Therapeutic Area & Indication: Adult monotherapy for schizophrenia or bipolar disorder for patients with acute manic or mixed episodes.

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: LUPKYNIS®

Company: Aurinia Pharmaceuticals Inc.

Therapeutic Area & Indication: Oral immunosuppressant for acute lupus nephritis of the kidneys.

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: JORNAY PM®

Company: Ironshore Pharmaceuticals, Inc.

Therapeutic Area & Indication: Extended release night time stimulant for Attention Deficit Hyperactivity Disorder (ADHD) in patients six years and older.

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.

Research Team:
Bert DeSousa 
Bev Incledon
Chantal Incledon
Jill Kapuscinski
David Lickrish
Mary Mazur-Melnyk
Angus McLean
Randy Sallee
Feng Zhang

Product: VERQUVO®

Company: Merck & Co., Inc.

Therapeutic Area & Indication: Treatment to reduce hospitalization and mortality risk for patients with CVD and heart failure.

About the Product:
VERQUVO’s unique mode of action—stimulation of soluble guanylate cyclase (sGC) independently of and synergistically with nitric oxide (NO)—enables it to restore the NO/sGC/cyclic guanosine 5’-monophosphate (cGMP) signaling pathway whose relative deficiency contributes to the pathology of heart failure. The efficacy and safety of vericiguat for treating heart failure patients with reduced ejection fraction (HFrEF) was explored in VICTORIA, a 5,050-patient randomized, placebo-controlled Phase 3 trial. The positive results of VICTORIA, a statistically significant 10% relative reduction in HF hospitalization or CV death, led to VERQUVO’s approval in over 60 countries for treating patients with worsening HFrEF.

Product: Cibinqo®

Company: Pfizer

Therapeutic Area & Indication: Adults with moderate to severe atopic dermatitis who have not responded to other treatments.

About the Product:
CIBINQO (abrocitinib) is a JAK1 inhibitor that is FDA approved for the treatment of adults with moderate-to-severe eczema (atopic dermatitis) that did not respond to other treatment and is not well-controlled with prescription medicines, including biologics. In clinical trials of over 1,600 people, CIBINQO was proven to help provide clearer skin and itch relief. Furthermore, in studies in 778 people, more people saw 75% skin improvement with CIBINQO alone vs placebo after just 3 months, and more people felt itch reduction taking CIBINQO alone versus placebo after just two weeks: clearer skin and fast itch reduction.

Jessica Federer is an investor and board member adept at deploying technology to make meaningful advancements for both business and society.

She was one of the first Chief Digital Officers in the pharma industry, leading the digital transformation for Bayer A.G. across the Pharmaceutical, Consumer Care, Crop Science and Animal Health businesses. She is recognized as one of the top 100 CDOs and a leading health tech influencer, and served on the United Nations ITU 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: ZOKINVY®

Company: Eiger BioPharmaceuticals, Inc.

Therapeutic Area & Indication: Treatment of Progeria genetic disease of rapid premature aging in children and young adults.

About the Product:
Zokinvy® is a first-in-class, disease-modifying therapy for children and young adults with progeria, the collective term for Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies, ultra-rare, fatal pediatric diseases that cause dramatically accelerated aging and premature death. In patients with HGPS, Zokinvy reduced the incidence of mortality by 72% and increased average survival time by an average 4.3 years. Some progeria patients have received Zokinvy therapy for >10 years. The approval of Zokinvy in the U.S and Europe is the culmination of an innovative, pioneering partnership between Eiger and The Progeria Research Foundation. Eiger offers a global expanded access program.

Research Team:
Rich Franco
Colin Hislop, MBBS
Amy Ding, PhD
Blair Narog
Leslie Gordon, MD, PhD
Monica Kleinman, MD
Marie Gerhard-Herman, MD
Mark Kieran, MD
Leslie Smoot, MD
Francis Collins, PhD
Joe Massaro, PhD
Ralph D’Agostino, PhD
Jennifer DiGiacinto, PharmD
Larry Shen, PhD
Nicole Wallstedt 
Mike Perrot
Ana Iglesias

Product: WELIREG

Company: Merck & Co., Inc.

Therapeutic Area & Indication: Adult patients with genetic mutation that causes rare Von Hippel-Lindau syndrome (VHL) and require therapy for associated cancers including renal cell carcinoma, CNS hemangioblastomas and pancreatic and endocrine tumors.

About the Product:
WELIREG is an inhibitor of hypoxia-inducible factor 2α (HIF-2α). It act by binding to the PAS-B domain of HIF-2α disrupting its dimerization with HIF-1β/ARNT and inhibiting downstream expression of HIF-2α target genes. WELIREG is the first HIF-2α inhibitor approved by the US Food and Drug Administration and the first systemic treatment for certain patients with von Hippel Lindau (VHL)-disease associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors. Furthermore, it is the first approved medicine to be developed against a transcription factor.

Product: LIVMARLI® (maralixibat) oral solution

Company: Mirum Pharmaceuticals, Inc.

Therapeutic Area & Indication: Targets cholestatic pruritis in patients with Alagille Syndrome a genetic condition that causes progressive liver and heart disease in young children.

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. For more information, please visit LIVMARLI.com. 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.

Alison (Ali) Holland is Head of Decentralized Clinical Trials at Medable and is chartered to design and expand the end-to-end capabilities needed to partially or fully virtualize clinical studies, building on the commitment to reduce clinical trial times by 50%.

Ali has over 20 years of experience in a variety of leadership roles, including Feasibility Head and Six Sigma Black Belt at Covance, and Business Head and global General Manager at Roche/Genentech in one of their therapeutic area business groups.

Having been engaged in over 300 studies across Biotechs, Pharma, and all geographies, Ali brings an incomparable level of insight and technical excellence on the matter of operational needs for sites and patients.

Product: Nextstellis® (drospirenone and estetrol tablets) 3 mg/14.2 mg

Company: Mayne Pharma

Therapeutic Area & Indication: Oral birth control medication that prevents pregnancy with a novel form of estrogen that occurs naturally in the body.

About the Product:

Product: Myfembree®

Company: Myovant Sciences, Inc.

Therapeutic Area & Indication: Control of heavy menstrual bleeding due to uterine fibroids in premenopausal women.

About the Product:
Women’s Health MYFEMBREE: At Myovant, we’re invested in advancing women’s health and providing medical treatment options for women with uterine fibroids (UF) and endometriosis. MYFEMBREE® (relugolix 40mg, estradiol 1mg, and norethindrone acetate 0.5mg) is the first and only once-daily oral GnRH receptor antagonist combination product approved for the management of heavy menstrual bleeding associated with UF and moderate to severe pain associated with endometriosis, with a treatment duration of up to 24 months. MYFEMBREE has a black box warning and is contraindicated in women with current or a history of thrombotic or thromboembolic disorders and in women at increased risk for these events.

Product: ORGOVYX™ (relugolix)

Company: Myovant Sciences, Inc.

Therapeutic Area & Indication: Treatment for advanced prostate cancer.

About the Product:
Prostate Cancer ORGOVYX: At Myovant, we’re invested in redefining care for patients with prostate cancer. ORGOVYX® (relugolix) is the only oral gonadotropin-releasing hormone (GnRH) antagonist approved for the treatment of advanced prostate cancer. As a GnRH antagonist, ORGOVYX blocks the GnRH receptor and reduces production of testosterone, a hormone known to stimulate the growth of prostate cancer. The Phase 3 HERO study demonstrated the efficacy and safety of ORGOVYX, including a 96.7% response rate in testosterone suppression to castrate levels (<50 ng/dL) through 48 weeks. Safety warnings and precautions include QT /QTc interval prolongation, embryo-fetal toxicity, and laboratory testing.

Product: Apretude (cabotegravir extended-release injectable suspension)

Company: ViiV Healthcare

Therapeutic Area & Indication: Long acting injectable to reduce risk of contracting HIV-1 virus in adults and adolescents.

About the Product:
With 1.2 million people living with HIV in the U.S. alone, HIV remains a public health crisis. Recognizing the critical role of PrEP in reducing new HIV cases, ViiV Healthcare focused on innovation in delivering a new, effective prevention option. APRETUDE (cabotegravir extended-release injectable suspension) is the first and only long-acting injectable for HIV prevention. It significantly reduces dosing frequency—from 365 days to as few as six injections per year, after initiation. For the first time ever, people vulnerable to acquiring HIV have an option beyond a daily pill, bringing us one step closer to ending the HIV epidemic.