Podcasts about Actelion

Swiss biopharmaceutical company

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Best podcasts about Actelion

Latest podcast episodes about Actelion

Women to Watch™
Eylem Demir Sentürk, Women on Stage: "I'm a survivor."

Women to Watch™

Play Episode Listen Later Sep 25, 2024 65:34


Eylem Demir Sentürk, Founder of Women on Stage, shared the story behind her title with us on September 25, 2024.⭐⭐⭐⭐⭐Rating: 5 out of 5.Of the interview, our founder and host, Sue Rocco, says: "Listen in as I sit down with Eylem this week to talk about Eylem's family having to flee Turkey as Kurdish members, the challenge of integrating into a new culture in Switzerland, her experience with workplace bullying by her manager in a large Pharma company that led to a mental and physical breakdown, and the founding of her new venture to help working women thrive."ABOUT EYLEM: Beginning her career in the banking world, Eylem swiftly transitioned into the Pharma Industry, leaving an indelible mark at renowned companies such as Actelion, Celgene, Vifor Pharma, Novartis, and Bayer. Over a span of 22 years, she not only led international teams but also collaborated with professionals from 40 different nationalities. Eylem's commitment to excellence extended beyond corporate boundaries as she coached, and trained over 1000 individuals.Support this podcast at — https://redcircle.com/women-to-watch-r/donationsAdvertising Inquiries: https://redcircle.com/brandsPrivacy & Opt-Out: https://redcircle.com/privacy

Rare Disease Discussions
Kidney Involvement in Lysosomal Disorders

Rare Disease Discussions

Play Episode Listen Later Mar 5, 2024 60:00


Ozlem Goker-Alpan, MD, Founder and President, LDRTC and David G. Warnock, MD. Professor of Medicine (Emeritus) at University of Alabama at Birmingham discuss best practices to identify and treat kidney problems associated with lysosomal disorders.This CME/CE activity describes the pathophysiologies and management options for lysosomal disease patients with kidney problems. This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees. To receive credit for this program, go to https://checkrare.com/learning/ Speakers Ozlem Goker-Alpan, MD, Founder and President, LDRTC David G. Warnock, MD. Professor of Medicine (Emeritus)University of Alabama at BirminghamDisclosuresAffinityCE staff, LDRTC staff, CheckRare staff, planners, and reviewers, have no relevant financial interests to disclose. All faculty disclosures are listed below and are included in the beginning of each presentation.Dr. Goker-Alpan is a consultant, a principal investigator and /or on the speaker bureau, or has received grant support, from the following pharmaceutical companies: Actelion, Amicus Therapeutics, Sanofi, Takeda, Pfizer/Protalix.Dr. Warnock has had research support and/or consulting arrangements with Genzyme Corporation (Sanofi), Shire LLC (Takeda), Amicus, Protalix and Chiesi, Zebra Bio, Walking Fish, Hanmi, and Vera Therapeutics.Mitigation of Relevant Financial RelationshipsAffinityCE adheres to the ACCME's Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity. Conflicts of interest for presenting faculty with relevant financial interests were resolved through peer review of content by a non-conflicted reviewer.Learning ObjectivesAt the end of this activity, participants should be able to:Describe the role of the nephrologist in the team approach to careDescribe best practices to monitor kidney function in lysosomal disordersDescribe best practices to treat kidney disorders lysosomal disordersSupport for this educational activity was provided by Takeda, Sanofi, Amicus Therapeutics and Chiesi USA.

Rare Disease Discussions
Assessing, Monitoring, and Managing Respiratory Involvement in Lysosomal Disorders

Rare Disease Discussions

Play Episode Listen Later Mar 3, 2024 51:44


Ozlem Goker-Alpan, MD of LDRTC and John Bach, MD, Professor of Neurology at Rutgers School of Medicine discuss best practices to manage respiratory complications in persons with lysosomal disorders.This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees. To receive credit for this program, visit https://checkrare.com/learning/p-ldrtc2023-webinar3-assessing-monitoring-managing-respiratory-involvement-in-lysosomal-disorders/DisclosuresAffinityCE staff, LDRTC staff, CheckRare staff, planners, and reviewers, have no relevant financial interests to disclose. All faculty disclosures are listed below and are included in the beginning of each presentation.Ozlem Goker-Alpan, MDFounder and President, Lysosomal & Rare Disorders Research & Treatment Center (LDRTC).Dr. Goker-Alpan is a consultant, a principal investigator and /or on the speaker bureau, or has received grant support, from the following pharmaceutical companies: Actelion, Amicus Therapeutics, Sanofi, Takeda, Pfizer/Protalix.John Bach, MDProfessor of Physical Medicine and Rehabilitation, Professor of Neurology, Rutgers New Jersey Medical Center.Dr. Bach has no relevant financial interest to disclose.Mitigation of Relevant Financial Relationships AffinityCE adheres to the ACCME's Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity. Conflicts of interest for presenting faculty with relevant financial interests were resolved through peer review of content by a non-conflicted reviewer.Learning ObjectivesAt the end of this activity, participants should be able to:• Describe the most common LSDs that have pulmonary complications. • Describe best practices to manage pulmonary symptoms in Pompe disease. • Describe best practices to manage pulmonary symptoms in MPSs.• Describe best practices to manage sleep apnea in lysosomal diseases.PhysiciansThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.AffinityCE designates this enduring activity for a maximum of 1.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.Physician AssistantsAffinityCE designates this enduring activity for a maximum of 1.25 AMA PRA Category 1 Credits™. Physician Assistants should claim only the credit commensurate with the extent of their participation in the activity.NursesContinuing Nursing Education is provided for this program through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center's Commission on Accreditation (ANCC). This activity provides a maximum of 1.25 hours of continuing nursing education credit.Nurse PractitionersAffinityCE designates this enduring activity for a maximum of 1.25 AMA PRA Category 1 Credits™. Nurse practitioners should claim only the credit commensurate with the extent of their participation in the activity.Genetic Counselors Category 2 CEUAffinityCE designates this enduring activity for a maximum of 1.25 AMA PRA Category 1 Credit™. Genetic counselors should claim only the credit commensurate with the extent of their participation in the activity.Other ProfessionalsAll other health care professionals completing this continuing education activity will be issued a statement of participation indicating the number of hours of continuing education credit. This may be used for professional education CE credit. Please consult your accrediting organization or licensing board for their acceptance of this CE activity.Commercial Support Support for this educational activity was provided by Takeda, Sanofi, Amicus Therapeutics and Chiesi USA. Participation CostsThere is no cost to participate in this activity.CME InquiriesFor all CME policy-related inquiries, please contact us at ce@affinityced.com.Send customer support requests to cds_support+ldrtc@affinityced.com.

The NACE Clinical Highlights Show
The APAPP Podcast Ep. 7: What's new in Pulmonary Hypertension in 2023

The NACE Clinical Highlights Show

Play Episode Listen Later Sep 12, 2023 34:36


Show NotesNACE is excited to provide you with this podcast episode from our educational collaborator, The Association of Pulmonary Advanced Practice Providers (APAPP).  APAPP is the first  association of Advanced Practice Providers (APPs), both Nurse Practitioners and Physician Assistants, who work in the field of pulmonary medicine. APAPP's mission is to work together for the advancement of the profession and for the well-being of  patients with pulmonary diseases. To learn more about APAPP and to get involved, please visit https://www.pulmapp.com.GuestMartha Kingman, FNP-C, DNP Family Nurse Practitioner in the pulmonary hypertension clinic at the University of Texas Southwestern Medical Center at Dallas. She is presently serving as sub-investigator in numerous ongoing pulmonary hypertension clinical trials. She is a member of the speakers' bureaus for Actelion, United Therapeutics, and Gilead, and she gives her time and expertise to several nursing advisory boards. Martha is an active member of the PH Professional Network, having previously served on the national conference planning committee, and she has chaired the Research and Publications Committee of the Pulmonary Hypertension Association. She currently serves on the Editorial Board of Advances in Pulmonary Hypertension, the PHA's journal.Dr. Kingman earned a bachelor's degree from Tulsa University School of Nursing in Tulsa, following which she worked in emergency department settings as a staff nurse and then as director of the ED after which she obtained her Master of Science in Nursing degree from Texas Woman's University in Dallas. When she became board certified as a Family Nurse Practitioner, she accepted the job of nurse practitioner in the pulmonary department at UTSW Medical Center and remains in that role today.  The majority of her clinical work involves the follow-up outpatient care of a large group of pulmonary hypertension patients at UTSW Medical Center. In May 2011, Dr. Kingman obtained her Doctorate in Nursing Practice from Texas Christian University.Martha has published in the area of pulmonary hypertension and has spoken at local and national conferences.  Her main area of research interest involves the safety of intravenous prostacyclins in the hospital setting.HostCorinne R. Young, MSN, FNP-C, FCCPDirector of APP and Clinical Services, Colorado Springs Pulmonary ConsultantsPresident, APAPP, Colorado Springs, COCorinne Young is a Nurse Practitioner began working in the area of pulmonary disease in California in 2005. Since 2011, she has worked in a private pulmonary practice in Colorado Springs, Colorado. Involvement and the representation of advanced practice providers in the pulmonary world has been an important priority for Corinne.  To that end,  Corinne is the Founder and President of the Association of Pulmonary Advanced Practice Providers.In addition to her work with APAPP, Corinne is currently one of ten NPs nationwide to become a Fellow of the American College of Chest Physicians. She works closely with the American College of Chest Physicians CHEST programs, and serves on the Interprofessional Team Network, Clinical Research Network, and on the Executive Programing Committee. Additionally, Corinne serves on the American Board of Internal Medicine Pulmonary Disease Board.This Podcast episode does not offer CME/CE Credit.Please visit http://naceonline.com to engage in more live and on demand CME/CE content.

Nurse Wellness Podcast
What Is the True Art and Science of Yoga and Nursing? Wendy with Grant Scott, PhD, RN, CHFN-K

Nurse Wellness Podcast

Play Episode Listen Later Jun 22, 2023 46:02


Dr. Latora is the owner of ECLIPSE Health Consultants, LLC and it's affiliate The Nurse Doc Is Zen™ merging the art and science of Nursing and Yoga for holistic lifestyle coaching. Our mission is to normalize Self-care as an activity of daily living.Dr.Latora is passionate about empowering others to reach their optimal level of physical health and emotional wellbeing. With 29 years of RN clinical experience, she is a Certified Heart Failure Nurse (CHFN) with additional training in Pastoral Health Ministry; YogaNurse(R) Yoga Teacher and Psychotherapeutic Yoga. As a Holistic Wellness Practitioner, Dr. Latora offers virtual and in-person holistic wellness experiences. Consultation services include wellness meditations; therapeutic journaling; private and group Yoga classes. Dr. Latora is an international contractspeaker, facilitator and presenter with experience working with Actelion, Amtrak, American Cancer Society, Genentech, Gilead and the National Institute of Health. Join the conversation by sending comments or questions to hello@stressblueprint.com. Put NWP in the subject line.1. Subscribe to the Nurse Wellness Podcast 2. Download your FREE resource 3 Question to Ask When You're Stressed 3. Join the Wellness Hub for Stress Solutions community4. Email Nurse Wellness Podcast at hello@stressblueprint.comIntro and outro music produced by DNMbeats

Oral Arguments for the Court of Appeals for the Federal Circuit
Actelion Pharmaceuticals Ltd v. Mylan Pharmaceuticals Inc.

Oral Arguments for the Court of Appeals for the Federal Circuit

Play Episode Listen Later Jun 7, 2023 32:54


Actelion Pharmaceuticals Ltd v. Mylan Pharmaceuticals Inc.

ESC TV Today – Your Cardiovascular News
Episode 3: Factor XIa Inhibitors: A Breakthrough in Anticoagulation? - Heart Failure Treatment: New Concepts for 2023 and Beyond

ESC TV Today – Your Cardiovascular News

Play Episode Listen Later Oct 27, 2022 22:07


ESC TV Today brings you concise analysis from the world's leading experts, so you can stay on top of what's happening in your field quickly. This episode tackles: Cardiology this Week Factor XIa Inhibitors: A Breakthrough in Anticoagulation? Heart Failure Treatment: New Concepts for 2023 and Beyond Snapshots Host: Susanna Price Guests: Carlos Aguiar, Robert Storey, Carolyn Lam and Stephan Achenbach Want to watch that episode? Go to: https://esc365.escardio.org/event/602 Disclaimer: This programme is intended for health care professionals only and is to be used for educational purposes. The European Society of Cardiology (ESC) does not aim to promote medicinal products nor devices. Any views or opinions expressed are the presenters' own and do not reflect the views of the ESC. Declarations of interests Stephan Achenbach, Nicolle Kraenkel, Susanna Price and Robert Storey declared to have no potential conflict of interest to report. Carlos Aguiar declared to have potential conflict of interest to report: personal fees for consultancy and/or speaker fees from Abbott, Alnylam, Amgen, AstraZeneca, Bayer, Boehringer-Ingelheim, Daiichi-Sankyo, Ferrer, Gilead, Lilly, Novartis, Pfizer, Sanofi, Servier, Tecnimede. Davide Capodanno declared to have potential conflict of interest to report: Sanofi, Daiichi Sankyo, Terumo, Medtronic, Chiesi. Carolyn Lam declared to have potential conflict of interest to report: Carolyn S.P. Lam is supported by a Clinician Scientist Award from the National Medical Research Council of Singapore; has received research support from Bayer and Roche Diagnostics; has served as consultant or on the Advisory Board/ Steering Committee/ Executive Committee for Actelion, Alleviant Medical, Allysta Pharma, Amgen, AnaCardio AB, Applied Therapeutics, AstraZeneca, Bayer, Boehringer Ingelheim, Boston Scientific, Cytokinetics, Darma Inc., EchoNous Inc, Eli Lilly, Impulse Dynamics, Intellia Therapeutics, Ionis Pharmaceutical, Janssen Research & Development LLC, Medscape/WebMD Global LLC, Merck, Novartis, Novo Nordisk, Prosciento Inc, Radcliffe Group Ltd., ReCor Medical, Roche Diagnostics, Sanofi, Siemens Healthcare Diagnostics and Us2.ai; and serves as co-founder & non-executive director of Us2.ai. Emma Svennberg declared to have potential conflict of interest to report: Institutional research grants from Bayer, Bristol-Myers, Squibb-Pfizer, Boehringer- Ingelheim, Johnson & Johnson, Merck Sharp & Dohme

Medication Talk
New Heart Failure Guidelines

Medication Talk

Play Episode Listen Later Jun 1, 2022 30:09


TRC Editor, Dr. Lori Dickerson, PharmD, FCCP talks with two distinguished experts about new heart failure guidelines. Carolyn Lam, MBBS, PhD, FRCP, Professor at Duke-NUS Medical School and a Senior Consultant at the National Heart Centre SingaporeSteven E. Nissen, MD, MACC, the Chief Academic Officer at the Heart and Vascular Institute and the Lewis and Patricia Dickey Chair in Cardiovascular Medicine Professor of Medicine at the Cleveland Clinic Lerner School of Medicine at Case Western Reserve UniversityListen in as they discuss new guidelines for managing heart failure with reduced ejection fraction (HFrEF)...and put this new guidance in perspective.You'll also hear practical advice from panelists on TRC's Editorial Advisory Board:Andrea Darby Stewart, MD, Associate Director, Family Medicine Residency at Honor HealthAnthony A. Donato, Jr., MD, MHPE, Associate Program Director, Internal Medicine from the Reading Health System, and Professor of Medicine at the Sidney Kimmel Medical College at Thomas Jefferson UniversityCraig D. Williams, PharmD, FNLA, BCPS, Clinical Professor, Department of Pharmacy Practice at the Oregon Health and Science UniversityFor the purposes of disclosure, Dr. Carolyn Lam reports relevant financial relationships with Bayer, Roche Diagnostics (grants/research support); Abbott, Actelion, Alleviant Medical, Allysta Pharmaceuticals, Amgen, AnaCardio AB, Applied Therapeutics, AstraZeneca, Bayer, Boehringer Ingelheim, Boston Scientific, Cytokinetics, Darma Inc., EchoNous Inc, Impulse Dynamics, Ionis Pharmaceuticals, Janssen, Merck, Novartis, Novo Nordisk, Roche Diagnostics, Sanofi, Siemens Healthcare Diagnostics, Us2.ai (advisory board/steering committee/executive committee); Us2.ai (stock shareholder); Us2.ai (non-executive director).Dr. Steven Nissen reports a relevant financial relationship with AbbVie, Amgen, AstraZeneca, Bristol-Myers Squibb, Eli Lilly, Esperion, Medtronic, Novartis, Pfizer, Silence Therapeutics (grants/research support).The other speakers have nothing to disclose. All relevant financial relationships have been mitigated.Pharmacist's Letter offers CE credit for this podcast. Log in to your Pharmacist's Letter account and look for the title of this podcast in the list of available CE courses.If you're not yet a Pharmacist's Letter subscriber, find out more about our product offerings at trchealthcare.com. Follow or subscribe, rate, and review this show in your favorite podcast app. You can also reach out to provide feedback or make suggestions by emailing us at ContactUs@trchealthcare.com. 

Remo Uherek
#067: Idorsia: A High-Potential Biopharmaceutical Company (Investment Thesis)

Remo Uherek

Play Episode Listen Later Jan 20, 2022 15:56


Idorsia is a high-potential Swiss biotech company, founded by the same people that built Actelion, a biotech company that was sold to Johnson & Johnson for $30B in cash. Jean-Paul and Martine Clozel now intend to build a second Actelion, but instead of owning just 5%, they now own 29% of the company. Here's my investment thesis. Check out my course on personal finance and investing https://courses.remo.org/p/building-wealth (use code "TESLA50" for 50% off) For detailed show notes, check out the YouTube description to this video: https://youtu.be/X1GGygW_9Nk Social: Blog: https://remo.org Twitter: https://twitter.com/remouherek I'm Remo Uherek, investor, writer, and former startup founder, based in Basel, Switzerland. Retired from startups after 15 years. Today, I'm a private investor, help to run a real estate portfolio and write about health, wealth, love, and happiness. I teach online courses and am obsessed with innovation, Tesla and Elon Musk. Please subscribe if you want to hear more episodes like this. (This episode was originally published on YouTube: https://youtu.be/X1GGygW_9Nk) --- Send in a voice message: https://anchor.fm/remouherek/message

NGMC Continuing Medical Education
PERT CME:Operationalizing PERT

NGMC Continuing Medical Education

Play Episode Listen Later Nov 19, 2020 62:10


Enduring CME will expire on 11/19/2022. Presenter: Sara Hegab, MD, Henry Ford Health System, Detroit, MI Objectives - Introduction and Case Presentation - Philosophy and Rationale - Set Up and Initiation - Operations - Performance - Challenges Disclosures - Research support: Henry Ford Hospital receives financial support in the form of research grants from Actelion, Bayer, United Therapeutics and Gilead in the area of pulmonary hypertension - Bayer-Speaker, Advisory Board Accreditation and Designation: The Northeast Georgia Medical Center & Health System, Inc. is accredited by the Medical Association of Georgia to provide continuing medical education for physicians. The Northeast Georgia Medical Center & Health System, Inc. designates this live activity for a maximum of 1 AMA PRA Category 1 Credit(s) TM. Physicians should only claim credit commensurate with the extent of their participation in the activity.

NGMC Continuing Medical Education
PERT CME: Pulmonary Embolism Response Teams: Sometimes It Takes a Village

NGMC Continuing Medical Education

Play Episode Listen Later Nov 16, 2020 66:09


Enduring CME will expire on 11/16/2022. Presenter: Sara Hegab, MD, Henry Ford Health System, Detroit, MI Objectives •Challenges in management of patients with massive and submassivePE •Team based approach to PE management •Post-PE Syndrome Disclosures - Research support: Henry Ford Hospital receives financial support in the form of research grants from Actelion, Bayer, United Therapeutics and Gilead in the area of pulmonary hypertension - Bayer-Speaker, Advisory Board Accreditation and Designation: The Northeast Georgia Medical Center & Health System, Inc. is accredited by the Medical Association of Georgia to provide continuing medical education for physicians. The Northeast Georgia Medical Center & Health System, Inc. designates this live activity for a maximum of 1 AMA PRA Category 1 Credit(s) TM. Physicians should only claim credit commensurate with the extent of their participation in the activity.

Divine Downloads
Ep. 77 - Meditations From The Mat with Dr. Latora Scott, aka The Nurse Doctor is Zen™

Divine Downloads

Play Episode Listen Later Jul 17, 2020 52:27


Today’s episode of the Divine Downloads podcast (soon to permanently become The Spiritually Savvy Nurse Podcast) is like a yoga session filled with many jewels of wisdom and lessons that you can apply to your own life today. Dr. Latora Scott is a Registered Nurse with a doctorate degree in public health. She is also a yoga teacher and being in her energy is like receiving a yoga session just with her words and intention. She masterfully blends the art and science of Nursing with her holistic approach in life and her care of her clients. Grab your fav beverage and take a seat, you are going to want to pay FULL attention to the wisdom she shares. 3 jewels from this episode are: how nursing found her how she parents her black son as a woman of color how being a “Freedom Baby” influenced her feelings about the right to vote in this country If you could use a hand crafted meditation for your specific needs or need a Holistic SWAT analysis of your health, Dr. Latora is your girl. To get your Zen on, connect with her on the socials and her website: www.instagram.com/thenursedociszen https://thenursedociszen.com/ Dr. Latora is the owner of ECLIPSE Health Consultants, LLC and its affiliate The Nurse Doc Is Zen™ merging the art and science of Nursing and Yoga for holistic lifestyle coaching. She is passionate about empowering others to reach their optimal level of physical health and emotional wellbeing. As a Holistic Wellness Practitioner, Dr. Latora offers clients virtual and in person holistic health session options. Consultation services include wellness meditations; therapeutic journaling; private and group Yoga classes. Dr. Latora is an experienced Nurse Speaker, having worked as a contract speaker for Actelion and Gilead Pharmaceuticals as an PAH content expert. With 25 years of RN clinical experience, she is a Certified Heart Failure Nurse (CHFN) and holds certificates in RN Palliative Care Nursing; Holistic Health Coaching . Additional Dr. Latora has training in YogaFit ® Seniors; YogaFit® Level 1 and Yoga Nurse® Essentials. #publichealth #nurse #nursing #RYT #yoga #therapeuticyoga #meditation #mindfulness #holistichealth #zen #thenursedociszen #communityhealth #wellness #nurseentrepreneur #nurseconsultant #speaker #nursespeaker #nurseeducator #nursesofinstagram #nursesinbusiness #holistichealthcoach #yoganurse #palliativecarenurse #yogafit

Medical Industry Feature
Results of an Expert Delphi Consensus Survey on Clinical Scenarios Considered When Initiating Oral Prostacyclin Pathway Agents for Pulmonary Arterial Hypertension

Medical Industry Feature

Play Episode Listen Later Dec 16, 2019


Results of an Expert Consensus Survey on the Treatment of Pulmonary Arterial Hypertension with Oral Prostacyclin Pathway Agents Vallerie V McLaughlin, MD, FACC, FCCP; Richard Channick, MD; Teresa De Marco, MD, FACC, FHFSA; Harrison W Farber, MD, FCCP; Sean Gaine, MD, PhD; Nazzareno Galié, MD; Richard A Krasuski, MD; Ioana Preston, MD; Rogerio Souza, MD, PhD; J Gerry Coghlan, MD; Robert P. Frantz, MD; Anna Hemnes, MD; Nick H Kim, MD; Irene M Lang, MD; David Langleben, MD; Mengtao Li, MD; Olivier Sitbon MD, PhD; Victor Tapson, MD; Adaani Frost, MD Funding was provided by Actelion to support the use of independent providers of Delphi methodology expertise and nominal group technique, survey creation, data analysis, medical communication, and meeting management. The authors were not paid an honorarium for their participation. Actelion played no role in the literature search and analysis, development of surveys used to gather consensus, or data analysis; and no Actelion employee was present at the meeting during which consensus statements were finalized. The manuscript was drafted, critically reviewed, and edited solely by the authors with support from an independent professional medical communications agency. Actelion Pharmaceuticals reviewed the final manuscript only to ensure accuracy of UPTRAVI® (selexipag) background ...

AudioAbstracts
Results of an Expert Delphi Consensus Survey on Clinical Scenarios Considered When Initiating Oral Prostacyclin Pathway Agents for Pulmonary Arterial Hypertension

AudioAbstracts

Play Episode Listen Later Dec 16, 2019


Results of an Expert Consensus Survey on the Treatment of Pulmonary Arterial Hypertension with Oral Prostacyclin Pathway Agents Vallerie V McLaughlin, MD, FACC, FCCP; Richard Channick, MD; Teresa De Marco, MD, FACC, FHFSA; Harrison W Farber, MD, FCCP; Sean Gaine, MD, PhD; Nazzareno Galié, MD; Richard A Krasuski, MD; Ioana Preston, MD; Rogerio Souza, MD, PhD; J Gerry Coghlan, MD; Robert P. Frantz, MD; Anna Hemnes, MD; Nick H Kim, MD; Irene M Lang, MD; David Langleben, MD; Mengtao Li, MD; Olivier Sitbon MD, PhD; Victor Tapson, MD; Adaani Frost, MD Funding was provided by Actelion to support the use of independent providers of Delphi methodology expertise and nominal group technique, survey creation, data analysis, medical communication, and meeting management. The authors were not paid an honorarium for their participation. Actelion played no role in the literature search and analysis, development of surveys used to gather consensus, or data analysis; and no Actelion employee was present at the meeting during which consensus statements were finalized. The manuscript was drafted, critically reviewed, and edited solely by the authors with support from an independent professional medical communications agency. Actelion Pharmaceuticals reviewed the final manuscript only to ensure accuracy of UPTRAVI® (selexipag) background ...

AudioAbstracts
Results of an Expert Delphi Consensus Survey on Clinical Scenarios Considered When Initiating Oral Prostacyclin Pathway Agents for Pulmonary Arterial Hypertension

AudioAbstracts

Play Episode Listen Later Dec 15, 2019


Results of an Expert Consensus Survey on the Treatment of Pulmonary Arterial Hypertension with Oral Prostacyclin Pathway Agents Vallerie V McLaughlin, MD, FACC, FCCP; Richard Channick, MD; Teresa De Marco, MD, FACC, FHFSA; Harrison W Farber, MD, FCCP; Sean Gaine, MD, PhD; Nazzareno Galié, MD; Richard A Krasuski, MD; Ioana Preston, MD; Rogerio Souza, MD, PhD; J Gerry Coghlan, MD; Robert P. Frantz, MD; Anna Hemnes, MD; Nick H Kim, MD; Irene M Lang, MD; David Langleben, MD; Mengtao Li, MD; Olivier Sitbon MD, PhD; Victor Tapson, MD; Adaani Frost, MD Funding was provided by Actelion to support the use of independent providers of Delphi methodology expertise and nominal group technique, survey creation, data analysis, medical communication, and meeting management. The authors were not paid an honorarium for their participation. Actelion played no role in the literature search and analysis, development of surveys used to gather consensus, or data analysis; and no Actelion employee was present at the meeting during which consensus statements were finalized. The manuscript was drafted, critically reviewed, and edited solely by the authors with support from an independent professional medical communications agency. Actelion Pharmaceuticals reviewed the final manuscript only to ensure accuracy of UPTRAVI® (selexipag) background ...

Medical Industry Feature
Results of an Expert Delphi Consensus Survey on Clinical Scenarios Considered When Initiating Oral Prostacyclin Pathway Agents for Pulmonary Arterial Hypertension

Medical Industry Feature

Play Episode Listen Later Dec 15, 2019


Results of an Expert Consensus Survey on the Treatment of Pulmonary Arterial Hypertension with Oral Prostacyclin Pathway Agents Vallerie V McLaughlin, MD, FACC, FCCP; Richard Channick, MD; Teresa De Marco, MD, FACC, FHFSA; Harrison W Farber, MD, FCCP; Sean Gaine, MD, PhD; Nazzareno Galié, MD; Richard A Krasuski, MD; Ioana Preston, MD; Rogerio Souza, MD, PhD; J Gerry Coghlan, MD; Robert P. Frantz, MD; Anna Hemnes, MD; Nick H Kim, MD; Irene M Lang, MD; David Langleben, MD; Mengtao Li, MD; Olivier Sitbon MD, PhD; Victor Tapson, MD; Adaani Frost, MD Funding was provided by Actelion to support the use of independent providers of Delphi methodology expertise and nominal group technique, survey creation, data analysis, medical communication, and meeting management. The authors were not paid an honorarium for their participation. Actelion played no role in the literature search and analysis, development of surveys used to gather consensus, or data analysis; and no Actelion employee was present at the meeting during which consensus statements were finalized. The manuscript was drafted, critically reviewed, and edited solely by the authors with support from an independent professional medical communications agency. Actelion Pharmaceuticals reviewed the final manuscript only to ensure accuracy of UPTRAVI® (selexipag) background ...

I'm Aware That I'm Rare: the phaware® podcast

Hall Skåra is a pulmonary hypertension patient from Norway. He became part of PHA Europe’s staff after his diagnosis. He has helped develop their “White Spots” program, which establishes new PH patient associations in countries where they don’t exist. In this episode, Hall discusses the first ever PAH Patient Charter that was launched on November 12, 2019 to coincide with PH Awareness Month. The Charter was facilitated and sponsored by Actelion, a Janssen Pharmaceutical Company of Johnson & Johnson and a PAH-expert Steering Committee made up of patients, patient advocates and healthcare professionals. #PAHPatientCharter #phawarenessmonth  Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate #phaware #ClinicalTrials  @actelion_com @EuropePHA @PHAssociation @wginsing @PhDeutschland @LatinosConHP @Hellenic_PH @phsingapore

I'm Aware That I'm Rare: the phaware® podcast
Wendy Gin-Sing, RN - phaware® interview 299

I'm Aware That I'm Rare: the phaware® podcast

Play Episode Listen Later Nov 15, 2019


Wendy Gin-Sing RN, MSc, MRes, is an advanced nurse practitioner with the Pulmonary Hypertension Service at the Hammersmith Hospital in London. In this episode, she discusses the first ever PAH Patient Charter that was launched on November 12, 2019 to coincide with PH Awareness Month. The Charter was facilitated and sponsored by Actelion, a Janssen Pharmaceutical Company of Johnson & Johnson and a PAH-expert Steering Committee made up of patients, patient advocates and healthcare professionals. #PAHPatientCharter #phawarenessmonth  Read the PAH Patient Charter here: bit.ly/36K0Hoz My name's Wendy Gin-Sing. I'm a Pulmonary Hypertension Nurse Specialist. I work at the Hammersmith Hospital in London. I've been looking after pulmonary hypertension patients for about 24 years, and the last 20 years as a Nurse Specialist. I've seen quite a few changes over the time. Today, I want to talk to you about the new PAH Patient Charter that's just been released. But first, let me give you a brief explanation about what pulmonary hypertension is. To understand pulmonary hypertension, you need to know what the normal circulation in the body is. You have two circulations, one which pumps blood all the way around the body, and that's the blood pressure that can be measured on your arm. And then there's a second circulation where the heart pumps the blood through the lungs so that it picks up oxygen ready to start off again. In pulmonary hypertension, the pressure in the blood vessels in the lungs goes up. Normally, this is just very low pressure, because you don't need the blood to go very far. It just goes from one side of the heart to the other by the lungs, and the oxygen moves over from the lungs into the blood vessels at low pressure. In pulmonary hypertension, blood vessels in the lungs change. The walls become thicker. They become less elastic, and the pressure goes up in the blood vessels. This then places more demand on the right side of the heart, which, over time, becomes tired and can sometimes fail. The PAH Patient Charter came about through Actelion, a Janssen Pharmaceutical Company of Johnson & Johnson. They got a group of us together: patients, patient associations, doctors, nurses. We sat down together and discussed about what we felt was really important when looking after patients with pulmonary hypertension. Both from the medical side and the patient side. We came together and we agreed on many points, and have produced the Charter. This Charter is going to be important for patients, caregivers, and clinicians looking after pulmonary hypertension. It sets out what patients with pulmonary hypertension should expect. Some of the very simple things like an earlier and accurate diagnosis, being involved in decision making, access to the therapies that are available in their country, and also, and probably the most important, the holistic care for patients so that they can actually live life well and have a better quality of life. When this is released, I think patients can use it in a variety of ways to speak to their health care professionals, and to try and ensure that they are getting the standard of care that's set out in the Charter, but also to be able to use it for lobbying politically and through the health care organizations in the countries they're in to improve the care of all patients with pulmonary hypertension. In the UK, we're very lucky that we have designated centers and access to all the pulmonary hypertension medications. But I am aware that there's many places in the world that don't have specialist centers. They have health care that they can't access all the drugs. For some patients, it takes a very long time to get a diagnosis. The optimal patient journey really starts in the very beginning, getting an early and accurate diagnosis. It still takes around two years in countries with developed health care to achieve a diagnosis of pulmonary hypertension. Whilst the first year, maybe, patients perhaps [are] not seeking medical attention, they have often seen four or five different physicians over the next year before they get an accurate diagnosis. Once patients have their diagnosis, they should have access to all available pulmonary hypertension therapies within their country. They should also have their care from a most disciplinary team so that they can look after their holistic needs. Not only their physical needs, such as rehabilitation and doing more exercise and managing their symptoms, but also the psychological care that patients, and not only patients, but their carers as well really need as they're going through the journey with pulmonary hypertension. For some patients who pulmonary hypertension therapy doesn't control all their symptoms, they need supportive care to help manage those symptoms. As the disease progresses, they should then have input from the palliative care teams, so that they have a really good quality of life throughout their pulmonary hypertension journey. I was honored to be asked to take part in the Patient Charter group. It was really good to meet up with patients, physicians, patients, advocacy groups from around the world: PHA Europe, PHA (US), phaware global association®, the PH Association for Latin America, Singapore, Greece, and Germany. It was really important that advocacy groups were involved in the PAH Patient Charter. As health care professionals, we see pulmonary hypertension very much from our point of view, and the associations have many members, all of which who live with the disease or care for somebody who does. It's so important to get their views so that this PAH Patient Charter is really a whole global initiative. The PAH Patient Charter is really important, because it will help patients be more empowered so that they can be part of making the decisions about their care. Not only because they're better educated, but also they have more confidence in being able to talk to you, [the health care professionals,] but making decisions about the care that really affects them. Pulmonary hypertension can affect anybody, from little children up to people who are very old. It affects people in every country, and that's why it's important to have a global PAH Patient Charter that we use to improve quality and quantity of life with patients with pulmonary hypertension. We're excited that the PAH Patient Charter's been launched during Pulmonary Hypertension Awareness Month. To learn more, search the hashtag #PAHPatientCharter. My name's Wendy Gin-Sing, and I'm aware that I'm rare. Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate #phaware #ClinicalTrials #phawareMD  @actelion_com @EuropePHA @PHAssociation @wginsing @PhDeutschland @LatinosConHP @Hellenic_PH @phsingapore

I'm Aware That I'm Rare: the phaware® podcast
Episode 299 - Wendy Gin-Sing, RN

I'm Aware That I'm Rare: the phaware® podcast

Play Episode Listen Later Nov 14, 2019 6:09


Wendy Gin-Sing RN, MSc, MRes, is an advanced nurse practitioner with the Pulmonary Hypertension Service at the Hammersmith Hospital in London. In this episode, she discusses the first ever PAH Patient Charter that was launched on November 12, 2019 to coincide with PH Awareness Month. The Charter was facilitated and sponsored by Actelion, a Janssen Pharmaceutical Company of Johnson & Johnson and a PAH-expert Steering Committee made up of patients, patient advocates and healthcare professionals. #PAHPatientCharter #phawarenessmonth  Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate #phaware #ClinicalTrials #phawareMD  @actelion_com @EuropePHA @PHAssociation @wginsing @PhDeutschland @LatinosConHP @Hellenic_PH @phsingapore

FirstWord Pharmaceutical News
FirstWord Pharmaceutical News for Tuesday, Apr 18 2017

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Play Episode Listen Later Apr 18, 2017 1:50


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Play Episode Listen Later Apr 7, 2017 1:51


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Play Episode Listen Later Feb 16, 2017 1:38


S&P Pharma Dose
Episode 12: Pharma Chat - It's All in the Transparency

S&P Pharma Dose

Play Episode Listen Later Feb 2, 2017 9:12


Analysts Arthur Wong and Adam Dibe discuss pharma trying to improve their public image, pharma pricing and transparency, Merck's disclosing their gross and net pricing, and Johnson & Johnson's acquisition of Actelion.

TipTV Business
M&A Update - J&J/Actelion deal, CH2M/WS Atkins deal

TipTV Business

Play Episode Listen Later Jan 30, 2017 9:04


Ben Harrington, Business Journalist and Betaville blogger, updates us about the major news in the Merger & Acquisition (M&A) world. The segment includes discussion on the $30 billion Johnson & Johnson/ Actelion deal, Allianz's takeover talks with QBE and the $4 billion merger of CH2M and Britain's WS Atkins. #Johnson&Johnson, #Actelion, #Allianz, #QBE, #CHRM, #WS #Atkins, #M&A, #business, #macro, #UK, #US

What The Pharma
Episode 7 - Science, PhRMA campaign and new discoveries and J&J's R&D productivity stop gap M&A

What The Pharma

Play Episode Listen Later Jan 30, 2017 17:51


In this episode, I discuss the new Pfizer campaign around scientists as well as Martin Shkreli's response to the Davos comments.  Then we discuss J&J's move to acquire Actelion and more importantly, their decision to spin off the R&D unit to be independent.  Near the end, discussion around scientific discoveries in the area of CAR-T treatment of two babies and new findings in breast cancer including drug resistance and passive/backseat genes that manipulate the cancer progression.

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FirstWord Pharmaceutical News for Thursday, Jan 26, 2017

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Play Episode Listen Later Jan 26, 2017 1:56


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FirstWord Pharmaceutical News for Monday, Jan 23, 2017

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Play Episode Listen Later Jan 23, 2017 1:35


Deal of the Week
60: J&J Nears Actelion Takeover Despite Twists and Turns

Deal of the Week

Play Episode Listen Later Jan 18, 2017 13:25


Neither Bloomberg's M&A executive editor Jeff McCracken nor host Alex Sherman can remember a deal quite like Johnson & Johnson's pursuit of Swiss drugmaker Actelion. First J&J publicly stated it was in talks with Actelion. Then, J&J said talks were over, and another party (Sanofi) had entered talks to buy Actelion. But just a week later, J&J and Actelion announced they'd re-entered talks. Now Bloomberg reports the sides have reached a tentative agreement on price, and a deal may be announced before the end of the month. McCracken explains why Actelion is such a coveted asset and what other large health care deals may emerge in 2017.

FirstWord Pharmaceutical News
FirstWord Pharmaceutical News for Friday, Jan 6, 2017

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Les experts Europe 1
Sanofi convoite le petit laboratoire suisse Actelion et la guerre des jupes à la Knesset : les experts d'Europe 1 vous informent

Les experts Europe 1

Play Episode Listen Later Dec 15, 2016 6:15


Axel de Tarlé, Géraldine Woessner et Sophie Larmoyer font le point sur l'actualité du jour.

FirstWord Pharmaceutical News
FirstWord Pharmaceutical News for Wednesday, Dec 14, 2016

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Play Episode Listen Later Dec 14, 2016 1:15


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Today in FirstWord:

Multiple Sclerosis Discovery: The Podcast of the MS Discovery Forum
Multiple Sclerosis Discovery -- Episode 49 with Dr. Hugh Rosen

Multiple Sclerosis Discovery: The Podcast of the MS Discovery Forum

Play Episode Listen Later Jul 21, 2015 22:38


[intro music]   Host – Dan Keller Hello, and welcome to Episode Forty-nine of Multiple Sclerosis Discovery, the podcast of the MS Discovery Forum. I’m your host, Dan Keller.   This week’s podcast features Dr. Hugh Rosen of the Scripps Research Institute. But first here are some new items in the MS Discovery Forum.   If you’re an MS researcher, you may want to keep an eye on our Bulletin Board section, where we post a variety of news items that may be of interest. One of the items we posted this week is directly related to Dr. Rosen’s work. It’s a notice that a phase 3 trial of a sphingosine 1-phosphate receptor modulator called RPC1063 has started recruiting twelve hundred patients with relapsing remitting MS in the US. RPC1063 had its origins in Dr. Rosen’s lab.   We also recently added a notice of another clinical trial to the Bulletin Board. That one’s a phase 2 trial of oral laquinimod in primary progressive MS. And a third new Bulletin Board announcement is a request for information from the Patient Centered Outcomes Research Institute to identify patient registries and research groups with established cohorts of patients for potential collaborative research opportunities on comparative effectiveness research in MS treatment.   To read any of these announcements, go to msdiscovery.org and click first on Professional Resources and then on Bulletin Board. And if you have an announcement you think may be of interest to MS researchers, please send it to editor@msdiscovery.org. We won’t post purely promotional press releases, but if we judge the notice to be of general interest, we’ll be happy to post it at no charge.   In other news, it was a relatively slow week in published MS research. According to our curated list of the latest scientific articles related to MS, only 22 such articles were published last week. Typically at least 40 MS-related peer-reviewed articles are published weekly, and we’ve seen some weeks with more than a hundred. To see the weekly lists going back to March 2012, go to msdiscovery.org and click on Papers.   Our Drug-Development Pipeline includes continually updated information on 44 investigational agents for MS. This past week we added 2 new trials and 7 other pieces of information. The drugs with important additions are dalfampridine, fingolimod, masitinib, and natalizumab. To find information on all 44 compounds, visit msdiscovery.org and click first on Research Resources and then on Drug-Development Pipeline   [transition music]   Now to the interview. Dr. Hugh Rosen studies chemical and biological approaches to the molecular mechanisms regulating lymphocyte trafficking. I met with him in his office at the Scripps Research Institute in La Jolla, California.   Interviewer – Dan Keller We're talking about mostly new compounds, S1P1 receptor compounds; the prototype now I suppose is fingolimod. What's in development and do they appear to offer advantages?   Interviewee – Hugh Rosen So, firstly, let me disclose that I am a cofounder of a biotechnology company called Receptos that has licensed an S1PR1 agonist from the Scripps Research Institute, so I have and my institution have a significant interest in this particular field.   Sphingosine 1-phosphate receptors act in a number of ways to modulate immune tissue damages in both autoimmune diseases and in viral infections. They've proven to be particularly efficacious in multiple sclerosis. Gilenya, of course discovered by Yoshitomi in Japan and developed by Novartis, has proven to be a clinically useful compound in the treatment of relapsing-remitting multiple sclerosis. And it appears to do so, at least in part, by altering the ability of lymphocytes to recirculate, and thus lymphocytes to reach the target tissues where they, in fact, produce demyelinating damage to the white matter of the central nervous system, and then the signs and symptoms of multiple sclerosis. So clearly these are useful compounds.   Gilenya, of course, is not a selective small molecule, it is an agonist of four of the five high affinity receptors for sphingosine 1-phosphate – S1P1, 3, 4, and 5 – and some of the associated side effects may be attributable in part to activity of Gilenya on other receptors like the S1P3 receptor that are not required for modulation in the treatment of multiple sclerosis.   MSDF I see that it's referred to as an immunomodulator, not necessarily referred to as a receptor agonist. Does it not have pure agonist effect? Does it have any effects either because of the other receptors or at that same S1P1 receptor?   Dr. Rosen No. In fact, Gilenya when phosphorylated is a full agonist of the sphingosine 1-phosphate receptors, and the newer compounds that are much more selective are also agonists of the sphingosine 1-phosphate 1 receptor. And some of the effects on them for cyto-mediated by downmodulation of the receptor, but I don't use the term modulators or immunomodulators because of the activity on the sphingolipid receptors per se, I use the term immunomodulator because of some of the unique advantages that we've demonstrated in model systems and in man about altering the activity of the sphingosine 1 receptor, because one of the beauties of immunomodulation is to blunt the immune response that causes collateral damage to the tissues whilst leaving sufficient of the immune response intact to allow protection from opportunistic pathogens – bacteria, viruses, and yeasts.   So one of the most striking features that we found – and these have been in some experiments done as a collaboration between my laboratory and the laboratory of Professor Michael Oldstone here at Scripps – has been in the area of influenza; pandemic influenza causes significant collateral tissue damage by having an overactive immune response. What we show is that the sphingosine 1-phosphate 1 receptor blunts that immune response and blunts the amplification of cytokines and chemokines so that you protect from the collateral tissue damage, but you leave intact the ability to mount protective, sterilizing T cell and B cell immunity to the virus. So you can eradicate the virus, sterilize it, you can provide a long-term memory both on the T-lymphocyte side as well as on the antibody side; there's class switching, there's affinity maturation, there are good protective immunity that is produced, and all this while blunting the immune response.   This is the Holy Grail as we think about treating patients, because the window for patients with autoimmune diseases like multiple sclerosis is that window between effective blunting of the immune response and the prevention of deleterious opportunistic infections that can have life-threatening consequences. So one of the advantages that I suspect we will see over time is that the sphingosine 1-phosphate agonists will prove to be particularly well-tolerated and have a wide window between the ability to limit tissue damage and progression of RRMS, and the need to protect patients from intercurrent infections or subclinical infections that become expressed later.   MSDF Do the other sphingosine 1-phosphate receptors interfere with lymphocyte trafficking also, or do they have other effects which nonselective ligands would then induce these adverse effects through them, or do they also have some effect in terms of trafficking?   Dr. Rosen They don't have significant effects on lymphocyte trafficking the way that S1PR1 does, both from the chemical approaches and the genetic evidence. S1P1 is clearly a toggle switch for lymphocyte trafficking. S1P2 is involved in the maintenance of hearing and in the function of vascular smooth muscle, so it regulates blood pressure. S1P3 is involved in cardiac contractility and also in the control of coronary artery caliber and the control of the airways, so S1P3 agonism is not a useful thing, it's actually quite deleterious. S1P4 and 5 have really no rate-limiting functions, at least of which I am aware, so there may be some redundancy and may not play a critical role in the modulation of health and disease.   MSDF Do you see compounds coming along which will be more selective and therefore not lead to the adverse effects so much? And if so, are these compounds chemically similar or do they have different structures to attach to the receptor, the S1P1?   Dr. Rosen These are clearly different structures, they're structurally very distinct from Gilenya and from each other. Novartis have a backup called siponimod. Actelion had a compound but it's only being used in psoriasis called ponesimod. Receptos has a compound now known as ozanimod – formerly known as RPC1063 – that is in two phase 3 studies for relapsing-remitting multiple sclerosis, a two-year study called RADIANCE and a one-year study called SUNBEAM, both of which are enrolling twelve hundred patients each.   MSDF And the RADIANCE trial results looked pretty good; I mean, you had 85, 90% effects at 12 to 24 weeks or even at a year in terms of relapse rate. Does this look like the next compound to emerge?   Dr. Rosen I think it's likely that ozanimod will be the next compound to be submitted for the regulatory process here in the United States and probably in Europe as well. The pleasing thing about the phase 2 data for ozanimod was, in fact, both the strong efficacy signal and a very well-tolerated safety profile; in fact the adverse effect profile of ozanimod and placebo were, in fact, indistinguishable and overlapping in the phase 2 studies. In addition, this very well-tolerated, favorable safety profile has been replicated in a highly successful phase 2 study in ulcerative colitis called TOUCHSTONE that was released recently. So clearly this is a mechanism of immunomodulation that could well prove to be useful for relapsing-remitting multiple sclerosis, but also in a range of other autoimmune diseases where treatments are hard to come by.   MSDF Even with Gilenya, I think there have been reports of a couple of cases of progressive multifocal leukoencephalopathy, so it gives a nice balance between immune surveillance and inhibiting T cell trafficking, but it seems like not a perfect balance. Does it look like that margin will be narrowed in the future with other compounds?   Dr. Rosen It's possible that it will be. I think the critical point to bear in mind is that real-world experience in tens of thousands of patients with hundreds of thousands of patient-years is really ultimately what is required to define these very rare events that on occasions do occur, and preexisting treatments with other immune-modifying agents such as Tysabri, for instance, may predispose to issues being seen later with PML. And I think that we always have to say that long-term patient experience and physician comfort are ultimately the best guides to the risk-benefit ratio.   MSDF I think you've identified something like four compounds in development, those are some that I had seen. Are there others, or these are really the ones to focus on at this point for people to keep an eye on?   Dr. Rosen There may well be others that are further behind. There have been a number of others that have had safety signals, particularly liver enzyme elevations, and significant first-dose cardiac effects. Arena have a compound that has recently completed a phase 1 multiple-dosing study and will go on to phase 2. So, you know, there are additional compounds and there will be additional compounds. Ultimately, patients do best when the best compounds appear, and the only way one knows that is to test them in man over the long-haul and define that risk-benefits for patients. And, you know, these multiple efforts really reflect the fact that a field has advanced, and that advancing field really does improve through intelligent intervention our ability to offer patients a better set of choices and a better set of long-term outcomes, which is what we're all about.   MSDF We're still focusing here on RRMS, none of this applies to the progressive phase. Is there anything coming along there?   Dr. Rosen You know, there's been one trial in primary-progressive; this was the Gilenya trial which didn't meet its endpoints. It may be that the mechanisms in rapidly progressive MS are a little different and that we don't yet, I think, understand the pathogenesis of that rather different presentation. So I'm not aware of a good alternate approaches to that, but that doesn't mean that the understanding isn't there for that to happen over time, it simply means that I'm not yet aware of it.   MSDF Finally, in secondary-progressive MS, we can understand what's going on, what led to it; if you limit relapses, that's good. But does it look like primary and secondary really may be overlapping but not the same disease?   Dr. Rosen I think there may be balances of pathogenesis where you can intervene more easily in some than in others. Clearly the sphingosine 1-phosphate agonists work particularly well by inhibiting the movement of lymphocytes into the brain. The movement of lymphocytes from the perivascular cuff into the parenchyma, into the white matter, where the demyelination proceeds. However, in parallel in multiple sclerosis, there are also events where there is collateral damage to neurons; we see axonal severing, we see elements of neuronal loss. Certainly with the sphingosine 1-phosphate agonists, there is some evidence that there is a diminution of cortical thinning over time with treatment, and that may be a really good thing.   I think that the neurodegenerative components is one that is hard to get a handle on right now, and that I think that these differences will become more obvious with early treatments of the immunopathology of multiple sclerosis. And that may well separate the autoimmune inflammatory damage and its sequelae from neurodegenerative mechanisms that may be entrained, and I think we will learn a lot from looking at those subsets of patients over time, particularly as more, better, and earlier treatment modalities allow the avoidance of significant damage in most patients.   MSDF Is there anything important we've missed or you'd like to add?   Dr. Rosen You know, I think for all of us who try to work at this interface of therapeutics, we do so because disease is, in fact, personal. We all know patients, we've all seen the multigenerational impact and depredations of multiple sclerosis on friends and family. And I think this is the very strong underlying motivator that drives us as scientists and as physician scientists to really try and bear in mind that the basic mechanisms and the basic therapeutic approaches that we pursue ultimately need a safe and effective human face to change the lives of patients in a positive way.   MSDF Very good. Thank you.   [transition music]   Thank you for listening to Episode Forty-nine of Multiple Sclerosis Discovery. This podcast was produced by the MS Discovery Forum, MSDF, the premier source of independent news and information on MS research. MSDF’s executive editor is Robert Finn. Msdiscovery.org is part of the non-profit Accelerated Cure Project for Multiple Sclerosis. Robert McBurney is our President and CEO, and Hollie Schmidt is vice president of scientific operations.   Msdiscovery.org aims to focus attention on what is known and not yet known about the causes of MS and related conditions, their pathological mechanisms, and potential ways to intervene. By communicating this information in a way that builds bridges among different disciplines, we hope to open new routes toward significant clinical advances.   We’re interested in your opinions. Please join the discussion on one of our online forums or send comments, criticisms, and suggestions to editor@msdiscovery.org.    [outro music]

FirstWord Pharmaceutical News
FirstWord Pharmaceutical News for Monday, June 8, 2015

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Play Episode Listen Later Jun 8, 2015 1:32


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FirstWord Pharmaceutical News for Tuesday, April 21, 2015

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