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The EFF program – Q1 2020

Close-up of an eye

Following the success of the first Eye on the Future Forum (EFF) in February 2019, we very much looking forward to seeing all the invitees for the 2020 edition, which will take place on Friday 28th February in Paris.

Published today, the final agenda for the EFF 2020, showcases the running order of presentations and lively discussion that will be had. This year in addition to international clinical experts, we have expanded the invitees to include patients, caregivers and patient organization representatives.

We hope to learn from the multi-stakeholder representatives on what can be improved in the clinical development process for inherited retinal diseases. With a focus on how to increase the patient perspective in all walks of the development lifecycle.

Looking forward to meeting you in Paris!

Jessica Ibbitson

This was the program for the EFF in the first quarter of 2020:

Timetable – Friday 28 Feb 2020

Welcome lunch

Time: 12:00-13:00

Opening Remarks

Presented by: Daniel De Boer, CEO ProQR Therapeutics
Time: 13:00-13:15
Room: Paris (9th Floor)

Keynote – Living with Usher Syndrome

Keynote Speaker: Rebecca Alexander
Time: 13:15-14:00
Room: Paris (9th Floor)
More details

Applying N-of-1 trial methods to precision therapies for IRD

Speaker: David Rodman
Time: 14:00-14:30
Room: Paris (9th Floor)
More details

Overview of LCA10 data

Speaker: Aniz Girach – Chief Medical Officer
Time: 14:30-15:00
Room: Paris (9th Floor)
More details

Interactive Panel: Demystifying Clinical Trials

Facilitator: Jessica Ibbitson
People in the panel: Mike Schwartz, Fritz Asmus, Rebecca Alexander, Isabelle Audo
Time: 15:00-15:30
Room: Paris (9th Floor)
More details

Coffee Break 15:30-16:00

Breakout session 1:
Journey from patient identification to enrollment

Moderator: Jessica Ibbitson
Time: 16:00-17:30
Room: Sydney 1&2 (8th Floor)
More details

Breakout session 2:
Site’s perspective and feedback regarding operational aspects in clinical trials

Moderator: Nigel Tomkinson
Time: 16:00-17:30
Room: Sydney 3 (8th Floor)
More details

Breakout session 3:
Patient’s perspective in clinical research and patient-physician interactions

Moderators: Molly Watt, Russel Wheeler, Andy Bolan
Time: 16:00-17:30
Room: Paris (9th Floor)
More details

Off-site dinner 18:30

Meet us in the lobby at 18:30 for the off-site dinner.

Timetable – Saturday 29 Feb 2020

Global patients and caregivers steering committee

Time: 08:00-13:00
Room: Paris 2&3 (9th floor)

Illuminate (sepofarsen) investigators meeting

Time: 09:00-12:00
Room: Sydney 1&2 (8th floor)

Aurora (QR-1123) investigators meeting

Time: 09:00-12:00
Room: Paris 3 (9th floor)

Study coordinator steering committee

Time: 13:00-17:00
Room: Sydney 3 (8th floor)

Presentation information

Living with Usher Syndrome

Keynote Speaker: Rebecca Alexander
No one would blame Rebecca Alexander if she gave up on life. Born with a rare genetic disorder, Usher syndrome (type III), Rebecca has been simultaneously losing both her vision and hearing since she was a teenager. She was told that by age 30, she’d be completely blind. Then, at 19 she discovered she would lose her hearing as well. Despite these obstacles, Rebecca refused to lose her drive and zest for life and rose above and beyond every challenge she faced.

Applying N-of-1 trial methods to precision therapies for IRD

Speaker: David Rodman
Developing precision medicines targeting specific genetic mutations requires a new way of thinking about clinical trial design. In theory, using a flexible and predictable medicine discovery platform like RNA-targeting antisense oligonucleotides, in combination with human cell-based models of disease allows for rapid development of novel therapeutics. Use of well designed, single subject clinical trials has the potential to dramatically reduce the time required to establish clinical benefit and even assess individual benefit/risk ratio. This presentation will provide an overview of the “n-of-1” approach ProQR is implementing to radically accelerate the pace of new medicine development for people with inherited retinal dystrophies.

Overview of LCA10 data

Speaker: Aniz Girach – chief medical officer
This presentation will showcase the positive top-line results from the phase 1/2 study of Sepofarsen in LCA patients (PQ-110-001). This study is a first-in-human open-label trial that was conducted at specialized centers with significant expertise in inherited retinal disease: The University of Iowa, the Scheie Eye Institute at the University of Pennsylvania and the Ghent University Hospital. PQ-110-001 enrolled five children (age 6 – 17 years) and six adults (≥ 18 years) who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene.

Interactive Panel

Demystifying Clinical Trials

Facilitator: Jessica Ibbitson
People in the panel: Mike Schwartz, Friedrich Asmus, Rebecca Alexander, Isabelle Audo

In the final EFF main hall session, this interactive session will allow the audience the opportunity to ask any question regarding the need to: demystifying clinical trials. The diverse panel brings a range of perspectives and experience in the area of clinical development in the area of inherited retinal diseases.

Breakout Sessions

Breakout session 1:
Journey from patient identification to enrollment

Moderator: Jessica Ibbitson
As recruitment for clinical trials becomes increasingly challenging, ProQR is looking at innovative ways to support sites, help generate quality referrals and engage networks of patient advocacy groups and other health care providers such as low vision specialists and genetic counsellors. This breakout session will share best practices and explore new ideas related to patient identification and recruitment for clinical trials for people with inherited retinal disorders

Breakout session 2:
Site’s perspective and feedback regarding operational aspects in clinical trials

Moderator: Nigel Tomkinson

During this interactive breakout session, we aim to obtain open feedback from site personnel how ProQR can improve the operational conduct of our clinical trials.

Clinical trials are key in the medicine development process. Within this highly regulated environment, operational aspects in conducting trials are an immense undertaking at sites. ProQR acknowledges this and strives towards a model of operational excellence in all aspects. With feedback obtained during this session from site personnel, we aim to improve the processes inherent to clinical trials and tailor them to clinical site’s needs & requirements, increasing efficiency and effectiveness while simultaneously decreasing unnecessary burden. Further during this session a discussion will be held on results of a site survey we conducted prior to EFF, an update ‘one-year on’ from a similar session held at EFF 2019 as well as future operational plans.

Breakout session 3:
Patient’s perspective in clinical research and patient-physician interactions

Moderators: Molly Watt, Russel Wheeler, Andy Bolan

This interactive breakout session will aim at bringing together patients, caregivers, professional advocates, researchers, investigators and ProQRians to discuss how we can further increase and integrate the patient perspective further into clinical trial research and development.

By combining lived experience with clinical expertise, we hope to explore and find solutions to the current challenges and barriers faced in making clinical research person-focused. Throughout health and research development there continues to be a growing desire for the patient perspectives due to the benefits associated with evidence-based programs and policies developed hand in hand with those living with the conditions that companies are striving for a solution. We at ProQR hope as a company we can harness the learnings from this session to help develop and implement the best in-class person focused clinical studies.

Speakers & Moderators

Daniel de Boer

A profile image of Daniel de Boer, CEO of ProQR.

Daniel de Boer is the founding Chief Executive Officer of ProQR. When confronted with the diagnoses of his newborn son with cystic fibrosis, he decided to start ProQR to help patients like his son. He collected a group of experienced biotech entrepreneurs around him and in 2012 founded ProQR with co-founders Dinko ValerioGerard Platenburg and Henri A. Termeer. Daniel is an entrepreneur and passionate advocate for rare disease patients. Since founding ProQR he has assembled a group of successful biotech executives and built a team of a 150 experienced scientists and medicine developers, devoted to creating RNA therapies for patients in need. Under Daniel’s leadership ProQR has initiated clinical trials in multiple development programs for rare diseases. Before founding ProQR, Daniel was founder and Chief Executive Officer of RNA Systems, PC Basic and Running IT, companies he led through phases of growth, developing and launching several products in multiple European countries. In 2018 Daniel was named “Emerging Entrepreneur of the Year” by EY in the Netherlands.

Rebecca Alexander

A profile image of Rebecca Alexander

Rebecca Alexander is an author, psychotherapist, group fitness instructor, advocate, and extreme athlete who was born with Usher syndrome type III, and has been simultaneously losing both her sight and hearing since she was a teenager.

Day after day, challenge after challenge, Rebecca stresses how important it is to be grateful for every sound, every sight and every sense. Now, with only a sliver of sight and significantly deteriorated hearing, Rebecca is a psychotherapist and a Lululemon Athletica ambassador who teaches cycling/spin and HIIT classes and regularly competes in extreme endurance races. She  currently serves on the boards of Cleveland Sight Center, Disability Rights Advocates, No Limits for Deaf Children and the Usher Syndrome Society.

Her critically acclaimed memoir, Not Fade Away: A Memoir of Senses Lost and Found received an Indie Book Award and was honored as one of the MS Society’s Books For A Better Life.

Rebecca is known for her sense of humor, and she is also an attentive listener. Consistently upbeat, Rebecca gives encouragement and inspiration to others who are facing their own challenges, whether physical or emotional, and whether in interpersonal relationships or in the workplace.

Connect with Rebecca:

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David Rodman

David Rodman

David Rodman, MD leads the ProQR Research and Development Team.  David completed his medical education at the University of Pennsylvania after which he relocated to the Rocky Mountain Region, completing post-graduate training in diseases of the lungs.  During a seventeen year academic career he grew interested in understanding the molecular mechanisms underlying rare inherited diseases, including cystic fibrosis.  Interested in taking that passion into the translational realm, he joined the biopharma industry in 2005 and during the past fifteen years has participated in the discovery and development of many successful treatments for cystic fibrosis and other rare diseases. In 2015, while living in Boulder, Colorado, David was living next to a family whose oldest son had been born with X-linked Retinoschisis, a rare inherited form of retinal detachment.  There was not treatment for this disease, and watching his neighbor struggle with the challenges of growing up with progressive vision loss moved Dave to look for ways to help.  This lead to David’s leaving Colorado and joining ProQR in 2017. David’s patient-centric focus, innovative approach and unflagging confidence in the power of science has energized the Research and Development Team and helped in our quest to be a leading biotechnology company in the rare eye disease space.

Connect with David:

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Aniz Girach

A profile image of Aniz Girach

Aniz Girach, MD is the Chief Medical Officer of ProQR. After having spent 11 years in academia, he joined the pharmaceutical industry with Eli Lilly, focusing on retinal diseases. He has in total 22 years industry experience in roles with Merck, as their Global Head of Ophthalmology, and Alcon, where he was Vice President of Clinical Development, and ThromboGenics (now Oxurion), where he was the Global Head of Ophthalmology/Chief Medical Officer.In addition to an Honorary Professorship at Wills Eye Hospital, Philadelphia, USA, he was most recently the Chief Medical Officer at Nightstar Therapeutics, overseeing the development of gene therapies for inherited retinal diseases. He is a member of three Scientific Advisory Boards for international ophthalmic organizations currently, and reviewer for five peer-reviewed journals, including Eye and IOVS. He has edited four books and published over 100 abstracts/manuscripts in peer-reviewed journals in Ophthalmology.

Connect with Aniz:

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Molly Watt

A profile image of Molly Watt

My name is Molly Watt, 25 years old. I am a tech/web, accessibility and usability consultant, motivational speaker, children’s author, illustrator, blogger, International ambassador and advocate for those living with challenges.

My passion lies in assistive technology, tech for good, website and environmental accessibility, inclusion and usability. I currently run my own company and am a co founder of the UK charity Molly Watt Trust where we raise awareness of Usher Syndrome, deafblindness, deafness, blindness, accessibility and inclusion. I also offer support to other UK deafblind charities and across the world.

I also work part-time at Sigma, “a leading specialist in user experience, from research and design to testing, development and optimisation.” I have and continue to work alongside some of the greats in digital, Apple, LinkedIn, ASOS, ATOS, Spotify.  I work with Sigma’s Experience Design Team assisting with the running of website audits and workshops on digital usability and accessibility, opening the eyes of those in web development and design.

I have spoken at Palace of Westminster, Harvard Medical Centre, Government Digital Services, NHS Digital, TEDXNHS, Apple Cupertino, Apple Corporate Sidney, Australia, GN Resound around the world and many many more.

Currently on the Diversity and Inclusion Board at the Institute of Coding where I am able to set out my visions for the future. My aim is to make the world a more open and inclusive place because ‘Nobody knows inclusion like those who have been excluded’.

Connect with Molly:

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Russel Wheeler

A profile image of Russel Wheeler

Russell is a UK citizen now resident in France.  He became involved in patient advocacy in 2012 following his 23 year old son’s sudden unexplained loss of sight, the diagnosis of which showed that his whole family was affected by a rare genetic disorder with no approved treatment.  In addition to his work for the UK based LHON Society, which he cofounded, Russell is an advocate for all rare eye conditions as a patient board member of ERNEYE and for all rare diseases as a volunteer with EURORDIS, where he serves on the ERN (ePAG) Steering Committee and Research and Registries subcommittee, as well as the DITA and HTA Task forces. He also represents EURORDIS at EMA on the Patient and Consumer Working Party. He is active in general patient advocacy via his 5 years work on the “Patient Engagement in Research” special interest group at ISPOR and participation in the PFMD project, which recently resulted in a quality guidance tool to facilitate more effective and measurable patient engagement in healthcare.

Connect with Russel:

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Isabelle Audo

A profile image of Isabelle Audo

Isabelle Audo, M.D., Ph.D., is a clinician scientist working both as a professor at the Quinze-Vingts Hospital and as a group leader in the Department of Genetics at the Vision Institute (Institut de la Vision) in Paris, France.

Her areas of expertise are medical retina, inherited retinal diseases both for clinical and molecular diagnosis, research in retinal physiopathology, visual function explorations with clinical electrophysiology, imaging techniques and clinical trials. Pr. Audo went to medical school at Paris XI University where she received her M.S. degree in Immunology.

She completed a residency program in Lille (North of France) from 1994 to 1997, where she became particularly interested in surgical and medical retina, especially in retinal dystrophies and clinical visual electrophysiology. Following her residency, she earned a Masters degree at Louis Pasteur University in Strasbourg, France with Pr. Sahel, and then in 1998, earned her M.D. Pr. Audo completed a research fellowship from 1999 to 2003 at the Department of Ophthalmology and Visual Sciences, University of Madison, Wisconsin, with Pr. Albert, and earned her Ph.D.

She then completed a medical retina fellowship at the Moorfields Eye Hospital with Prs Alan Bird and Graham Holder followed by a Master degree in genetics. Pr. Audo has co-authored more than 100 scientific papers through her research in inherited retinal diseases.

Andy Bolan

A profile image of Andy Bolan

Andy is the Associate Director for Patient & Medical Community Engagement at ProQR. He is a passionate advocacy and corporate affairs professional with over eight years’ experience of working in the domestic, regional and global biopharmaceutical industry. Beginning his career in London with the Association of the British Pharmaceutical Industry (ABPI) he has worked across a variety of corporate affairs roles, representing the ABPI in industry wide national policy discussions on antimicrobial resistance and vaccination programs.

After four and a half years in London, Andy moved to Amsterdam to join the dynamic global patient organizations & advocacy team at Teva Pharmaceuticals, where he led on the publication of Teva’s disclosure of payments to patient organizations and advocacy groups; covering over 67 countries and over 350 patient groups. He has represented Teva with a wide range of cross-therapeutic stakeholder and policy groups at a European Level, including: the European Brain Council (EBC), European Federation for Allergies and Asthma Patients’ Associations (EFA), & the European Federation for Neurological Associations (EFNA). Leaving Teva in April 2018, Andy joined the Biogen global patient advocacy team with the key responsibility of developing EU wide advocacy relationships in the area of Alzheimer’s Disease. Following the pausing of the Alzheimer’s program at Biogen in early 2019, Andy took wider responsibility for the pipeline advocacy responsibility for the disease areas: Amyotrophic lateral sclerosis, Ophthalmology, Movement Disorders (Parkinson’s & Ataxia), & early stage Alzheimer’s Disease.

He is an ardent believer in harnessing the growing importance of the lived experience of patients to improve and quicken the pace of medical research.

Connect with Andy:

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Jessica Ibbitson

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Jessica Ibbitson leads the Clinical & Development Operations department at ProQR Therapeutics which includes Clinical Operations, Project Management and Digital Health Technologies. Jessica has held various operational roles in biotech, an academic site and contract research organization.  Prior to her role at ProQR, Jessica was the head of Vertex Pharmaceutical’s Clinical Site Services department responsible for start-up of cystic fibrosis clinical development programs which led to the expedited approvals of Kalydeco®, Orkambi® and Symdeko®.  Jessica has spent her career focusing on rare diseases and developing new methods for accelerating clinical trials in order to get potential treatments to patients as quickly as possible.

Connect with Jessica:

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Nigel Tomkinson

A profile of Nigel Tomkinson

Nigel Tomkinson, Director Clinical Operations, joined ProQR in 2015 and leads the dynamic Clinical Operations team at ProQR who, together with partners, are responsible for the day to day management and oversight of the clinical trials. His professional career started in Antwerp, Belgium as an Emergency Care Registered Nurse as well as a Phase I Clinical Trial Nurse. He has over 16 years of experience in positions of increasing responsibility within clinical operations across a wide range of therapeutic indications and in all phases of clinical development (Phase I > IV). With his site level background and experiences with patients, he has a customer-focused mindset and is passionate to implement innovative approaches in clinical trial execution.

Connect with Nigel:

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Mike Schwartz

A profile image of Mike Schwartz

Michael Schwartz, VP, Ophthalmology, Global Project Leader for Sepofarsen, is a biopharmaceutical executive with extensive multi-disciplinary experience with a science-centric approach to R&D program strategy and execution.   He has a strong track record in leading large teams through product development strategy, therapeutic area strategic planning, organizational development, clinical development and project management.  He is highly successful in diverse leadership roles from preclinical research through clinical development.

Connect with Mike:

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Friedrich Asmus

A profile image of Friedrich Asmus

Friedrich Asmus, MD is the Vice President of Clinical Development at ProQR. He is a board-certified neurologist and pharmacist. Friedrich spent 12 years in academia, lead research groups for rare genetic  movement disorders and botulinum toxin therapy at the Hertie Institute for Clinical Brain Research in Tuebingen, Germany, where he was also Deputy Chair of the Department of General Neurology. In 2011, he joined the pharmaceutical industry with his first position in global clinical development of botulinum toxin A at Merz Pharma, Frankfurt, Germany. In 2013, he started working in ophthalmology global clinical development at Bayer Pharma, Berlin, Germany and was a key clinical contributor to the development program of the intravitreal anti-VEGF medicine aflibercept in the indication myopic CNV, retinal vein occlusions, and diabetic eye disease. He has published over 50 peer-reviewed full articles in ophthalmology and neurology.

Connect with Friedrich:

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