Dynacure news 2020

Clinical trial recruitment

Unite-CNM, the Dynacure sponsored clinical trial of their antisense product, DYN101, began recruiting in early 2020 at sites in the UK, Belgium and Netherlands, with an additional five European sites to follow.

Pre-clinical studies have shown that ‘turning down’ Dynamin 2 can reduce the symptoms of centronuclear myopathy, so the clinical trial will evaluate DYN101 for safety, tolerability, pharmacokinetics (how the drug works around the body) and preliminary efficacy (the effect on symptoms). It will include approximately 18 patients, who are over 16 years of age and have XLMTM or DNM2 mutations.

Further information about the trial, including inclusion and exclusion criteria, site location, recruitment status and contact details can be found below.

March 2020

Dynacure announces first patient dosed in phase 1 / 2 ‘UNITE-CNM’ Study of DYN101 for the treatment of centronuclear myopathies 

In March, Dynacure announced that a patient had been dosed with DYN101. This milestone marks the first time any company has dosed a CNM patient with an antisense medicine.  For further information view the press release below.

April 2020

Dynacure Announces €50M ($55M) Series C Financing

In March, Dynacure announced €50M ($55M) Series C Financing. The funding will support ongoing Phase 1 /2 ‘Unite-CNM’ study of DYN101 in patients with centronuclear and myotubular myopathy.

May 2020

Dynacure announce that circulating myostatin levels are altered in centronuclear myopathy mice and patients and myostatin levels respond directly to DNM2-therapy in mice. Myostatin is now currently being explored as a biomarker in our Unite-CNM clinical trial.

July 2020

In July the first two patients were dosed with investigational drug DYN101 in UNITE-CNM trial and Dynacure CSO Belinda Cowling was also named a finalist for the EU Prize for Women Innovators 2020.

Dynacure also received rare pediatric disease designation from the US Food and Drug Administation for DYN101 for myotubular and centronuclear myopathies.

Dynacure logo.

The Big Sunflower Project (mid year report)

2020 is the tenth year of The Big Sunflower Project and a milestone event for the little project, which originally was only meant to last for one year.

Dwarf sunflowers growing in wellington boots.

Obviously, no one expected the coronavirus situation and in March seed distribution was suspended.  We tentatively re-started in April and during late April and early May, were able to get some final batches of seeds safely in the post. Recipients included children currently home from school, schools that remained open to the children of key workers, a charity that supports people recovering from homelessness and addiction, a specialist worker for the Early Help and Prevention Service, an allotment project for children and adults with additional support needs, Enable Scotland (a charity which works with people who have learning disabilities), a residential home for older people with dementia and a hospital caring for a child with myotubular myopathy.

Sunflowers growing at Westminster Primary School.

Our intention at the beginning of the year had been to distribute 300 packets of seeds and we now know of over 290 people participating in the UK, on the Isle of Man, France, Greece, Germany, Sweden,  Australia and the Philippines, so we are feeling a tiny bit proud of ourselves for getting so close to our target at this difficult time. 238 places are currently plotted on the project map which can be viewed below. Click anywhere on the map to open it up and click a sunflower to learn about the people growing sunflowers in a particular location.  If you are growing sunflowers but cannot see yourself on the map, please ask to be added. As always, we wholeheartedly welcome anyone who grows sunflowers to take part in the project, even if they did not obtain their sunflower seeds from us.

In addition to posting seeds out, seeing as we had been thrown a curve ball, we decided to do a few things differently this year too.

Earlier in the year the project received a large donation of vegetable seeds. During the first three months of the year, these were sent together with sunflower seeds, to anyone who applied to the project and advised they had an allotment or stated they wanted seeds for a gardening club but from late April onwards, we began to give away our sunflower and vegetable seeds locally and we planted dwarf sunflowers, peas, cucumbers and runner beans and gave small plants away too.

Free seeds and plants.

Secondly, we decided to send seeds to schools we knew were still open for the children of key workers.  We thought being a small person with all this chaos going on right now, together with not being able to be with your friends and watching your parents go off to work each day must be quite a scary time, so decided to send a few surprise packages to schools, in the hope it would bring some cheer, make the children feel a bit special and give them something to look forward to – hopefully staff and parents would get some enjoyment from seeing the sunflowers too. It has been very lovely to hear from some of the schools and other recipients that the seeds and plants have been well received.

Jen and Holger planting sunflower seeds.

ZNM-Zusammenstark! e.v. 

This year the project is once again being joined by ZNM-Zusammenstark! e.v. growing sunflowers in memory of Emil, who was diagnosed with myotubular myopathy and sadly passed away in 2016.  Founded in 2015, ZNM-Zusammenstark! e.v. is a German association for those affected by centronuclear and  myotubular myopathy. Visit their website to read what they have to say about being part of The Big Sunflower Project.

Bear watering sunflowers.

In the news

The project has received some lovely publicity this year which can be read below and we are incredibly grateful to those who have taken the time to write about what we do.

Sunflowers growing at Spitalfields Crypt.

Resources

If you are  using your sunflower seeds for educational purposes, fundraising events or would like to raise awareness of centronuclear and myotubular myopathy at the place where you are growing your sunflowers, you can download flyers from the resources area of the website. You will also find resources for teaching children and to start conversations about equality and diversity.

Make a donation

The Big Sunflower Project is an initiative of The Information Point for Centronuclear and Myotubular Myopathy. The aim of the project is to raise awareness of the rare neuromuscular conditions known as centronuclear and myotubular myopathy, by sending seeds to people who have never heard of the conditions and requesting photos in return, which are shared in the Information Point newsletter and on the project social media pages, again raising awareness of centronuclear and myotubular myopathy. There is no charge for project seeds or the cost of postage, the project does however, welcome donations to ensure the future of the project and to enable seeds to be sent to as many people as possible each year. If you have donated for your seeds, thank you. If you would like to donate, you can learn more about how to do this below.

Our friends

This year project seeds have been donated by Thompson and Morgan and Tamar Organics. Seeds were also donated by Mike Rogers, Linda Fowler and Flower Power Lymo who grew sunflowers during the 2019 project and saved their seeds.

We are also grateful to everyone who has donated to The Big Sunflower Project since 2011, enabling us to celebrate our 10th anniversary.  You can read about these people below.

Looking forward to seeing everyone’s sunflower photos over the summer. Until then  stay safe everyone.

Planting sunflower seeds in Birkenhead.

Further information

Further information about the project can be found on The Big Sunflower Project website and on social media, where photos can be shared using #TheBigSunflowerProject. Use #centronuclear, #centronuclearmyopathy, #myotubular and #myotubularmyopathy to help raise awareness of centronuclear and myotubular myopathy too.

 

Powerwheelchair for Milosz

Milosz lives with his family in Market Harborough, Leicestershire and is diagnosed with X-linked myotubular myopathy. Over the last six months Milosz’s needs have changed and he now requires a specialised powered wheelchair.

Milosz

Milosz’s manual wheelchair has become too small to him and the back support is no longer efficient. In addition, Milosz had developed mild scoliosis which requires specialised back support. And while Milosz can self propel his manual wheelchair indoors, he relies on people to push him on longer journeys outdoors. As a pre teenage boy, Milosz craves some freedom and a powered wheelchair would enable him to be more independent.

Milosz’s family began the process of arranging for a power wheelchair in September 2019, applying for one through a charity but due to coronavirus, the charity were unable to fund all of it and organising fundraising events at this time is difficult, so the family are now aiming to raise the £6,700 needed themselves, before Milosz starts secondary school in September.

Stanley

Milosz’s friend Stanley is helping to fundraise also, by running a marathon. Starting on 26 May Stanley began running a mile every day for 26 days and is aiming to raise £2,000.

Could help fund this essential piece of equipment for Milosz? Please visit the websites below to learn more and donate.

Gordon W. Evans Art Leadership Award

Connie Bonfy lives in Kansas, USA and is diagnosed as an x-linked manifesting carrier of myotubular myopathy. She was recently awarded the Gordon W. Evans Art Leadership Award at Wichita’s Arts Council 50th Annual Art Awards.

Connie Bonfy

The annual Arts Council Awards were established in 1969 by the Wichita/Sedgwick County Arts and Humanities Council to recognise and honour businesses, foundations and individuals who have consistently supported the arts and humanities in the Wichita community.  You can learn more about Connie and the award below.

A graduate of Emporia State University (Drawing and Painting, Psychology) and Wichita State University (MA) in arts education/community development, Connie’s career has taken her from leading a small rural Kansas arts council, to Santa Fe, New Mexico and the Desert Chorale – a professional chorus, to a metropolitan ballet company – Ballet Wichita and more recently to Salina Arts and Humanities, a department of the City of Salina. Her diverse career also includes serving as the head grant writer for two community colleges in Kansas as well as the performing arts presenter at one. 

Connie is also a talented artist in her own right, including printmaking and painting in her studio and exhibiting regionally. Solo exhibitions include Cafe Life (2012) and Waiting for the Bus (2016) as well as several group shows.

In 2000 Connie was honoured by Kansas Governor Bill Graves with the Governor’s Arts Award for her lifelong committed work as an advocate for the arts. In 2013, she was selected to be part of the Climate Reality Project, lead by former Vice President Al Gore.

Officially retired, Connie is currently the CEO of Prairie Muses, a non profit organisation dedicated to celebrating the arts and engaging community through music, developing a short course for adults who wish to learn more about how to understand and view art and retraining to teach Art Appreciation online at Butler Community College.

Easyfundraising

The Myotubular Trust Easyfundraising eStore raises donations for the charity everytime a purchase is made there. The store has now raised a grand total of £1,429.21 for research into centronuclear and myotubular myopathy. If you haven’t heard of Easyfundraising before, you can see how it works in the short film below.

With Christmas fast approaching, The Information Point asked The Myotubular Trust to tell us why the donations are so important. Mel Spring, Communications Officer at the Trust said:

‘We are incredibly grateful for the regular donations that we receive via Easyfundraising and to those who support us this way.  We love that donations come to us at absolutely no extra cost to our supporters, and by doing something that many of us do on a regular basis – online shopping.  We were impressed at just how quick and easy it is to register and that you can even set up a reminder that pops up whenever you shop with a store that is registered with the scheme. The money that is raised on Easyfundraising is used to fund research and whilst each individual purchase may raise anything from a few pence to a few pounds, when several people join forces and register to support us, it really can make a difference. Please do consider supporting us this way – there’s no better time to register than in the lead in to the festive season when many of us become more frequent online shoppers.’

If you would like to learn more about how to fundraise for Myotubular Trust or about how they spend the money that is raised in their name, visit the Myotubular Trust website. 

 

Easyfundraising logo.

A decade of connections: US MTM-CNM Family Conference 2019

This year the MTM-CNM Family Connection hosted their sixth biennial US MTM-CNM Family Conference. Below they write about the event. Photos courtesy of Levi Gershkowitz of Living in the Light.

Erin, Mark and Marie from the MTM CNM Family Connection at the MTM - CNM Family Conference 2019.

Erin, Mark and Marie from the MTM CNM Family Connection at the MTM – CNM Family Conference 2019.

We are so grateful that we had the opportunity to celebrate ‘A Decade of Connections’ while hosting the sixth biennial US MTM-CNM Family Conference in St Louis, MO from  19 – 21 July 2019. Approximately 175 people attended the conference gathering 48 families and over 30 professionals from our rare disease community, as well as so many families represented in spirit who were not able to travel.

Our conference theme ‘Through the Gateway: Expanding Possibilities’” echoed St Louis’ iconic arch and gave our community an opportunity to reflect on ‘gateway moments’ that attendees have experienced on this rare disease journey – whether living with MTM/CNM, caring for someone, or working tirelessly for our community and look with hope towards the expanding possibilities before us.

The Kalejaiye family.

The Kalejaiye family.

We were grateful to have several of our lead researchers and pharma industry professionals working on behalf of our community join us and share the very latest on MTM and CNM research and advancements in treatments and also engage in an interactive clinical care discussion along with patients and families.   A strength of our conference is also providing medical professionals an opportunity to hear our patient and family stories, experiences, and insights as well, and collaboratively learn together.

Clinical Care Panel Discussion at the MTM-CNM Family Conference 2019.

Clinical Care Panel Discussion at the MTM-CNM Family Conference 2019.

Presentations covered such topics as genetics, diagnosis, clinical care, breathing, carrier issues, and the development of three potential treatments.  The three potential treatments that were presented at the conference this year included: Gene Therapy sponsored by Audentes, ASO Knockdown of DMN2 sponsored by Dynacure and re-purposing an existing drug (Tamoxifen) lead by Dr Jim Dowling’s Lab at The Hospital for Sick Children, Toronto.

We are also appreciative of the collaborative patient/family and professional discussion forum that followed the formal presentations on Saturday afternoon.  This year’s focus was on exploring clinical care guidelines and the potential need to expand resources and research in this area to ensure that optimal care is given to our loved ones.  It was an exceptional opportunity for both families and professionals to exchange information and experiences with each other in thoughtful and open dialog.

Attendees at the MTM_CNM Family Conference 2019.

Attendees at the MTM_CNM Family Conference 2019.

Our conference certainly highlighted the ‘Expanding Possibilities’ for our MTM-CNM community both in terms of promising treatments in development, as well as activating our community to become engaged partners and participants in the drug development process.  Our MTM-CNM Family Connection team held a panel on the importance of legislative advocacy and announced the development of COLA (Committee on Legislative Advocacy).  We will continue to educate, advocate, and activate our community to be the best advocates for our loved ones as we continue to pursue therapeutic treatments for all.

Families and individuals also shared valuable information about living with MTM and CNM and helpful resources with each other.  Strong connections were made over the weekend and the strength and support of our MTM-CNM Family Conference community was palpable. It gave us energy to continue our journeys and we made lasting memories to carry home with us.

US MTM-CNM family conference group photo.

US MTM-CNM family conference group photo.

 

Dynacure news 2019

April 2019

Clinical trial application (CTA) for DYN101 approved

In April the UK regulatory authority – the Medicines and Healthcare products Regulatory Agency (MHRA) – approved the Clinical Trial Application (CTA) for DYN101 – Dynacure’s antisense product in development for centronuclear myopathy. This antisense drug has been developed in collaboration with Ionis Pharmaceuticals who have an excellent track record in creating these drugs and is built on the DNM2 work of Jocelyn Laporte and his team at IGBMC in Strasbourg.

Dynacure receives research grant

Also in April, Dynacure announced they had been awarded €450,000 research grant funding from a French organisation, Bpifrance, to help them speed up current development plans for their lead project (an antisense compound/drug named DYN101). The funds will be used to see if it will help improve muscle strength in patients with centronuclear myopathy; support them to find out if they can potentially expand the application of DYN101 antisense therapy to help treat other human diseases and help them discover new measurements (known as biomarkers) which can prove the effectiveness of using this drug to treat patient symptoms.

July 2019

Dynacure receives orphan drug designation in the EU for DYN101

In July, Dynacure announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) had granted Orphan Drug Designation to DYN101 for the treatment of centronuclear myopathies (CNM). DYN101, an antisense medicine.

The European Commission grants Orphan Drug Designation in the EU to products that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 people in the European Union (EU) and where no satisfactory treatment is available.

“With Orphan Drug Designation in the EU, we have achieved a significant regulatory milestone in our development plans for DYN101 and we look forward to working with the EMA as we progress,” said Stephane van Rooijen (M.D. MBA), Chief Executive Officer of Dynacure.

Leen Thielemans, Chief Development Officer of Dynacure, added, “Orphan drug status is granted to investigational therapies that are expected to make a significant impact on patient care and we believe DYN101 holds great promise to treat a broad range of centronuclear and myotubular myopathies. The formal designation has been granted based on compelling preclinical data in several forms of CNM. As we prepare to initiate the UNITE-CNM clinical study, we also intend to expand the use of DYN101 to explore additional indications where the overexpression of DNM2 is a disease-driving factor.”

September 2019

Phase 1/2 clinical trial pipeline for DNM2 and MTM1, plus patient questions answered

In September 2019 at the request of CNM patient organisations, Dynacure provided  information on their ongoing work to bring their investigational product DYN101 through clinical development and approval for use in the treatment of CNM patients with MTM-1 or DNM2 mutations.

Please be aware that as the company are still in the phase of developing DYN101, they are limited as to what they can present according to regulations around the world. The scientific background is complex – it has been simplified as much as possible to be understandable and there is a glossary at the end that tries to explain some of the terms used.

Belinda Cowling awarded Prix Scientifiques 2019 (Scientific Prize) from the University of Strasbourg. 

Also in September Dynacure CSO Belinda Cowling was recently received the Prix scientifiques 2019 (Scientific Prize) from the University of Strasbourg. Belinda was recognized for her research publications and success in academic/industrial partnerships.

Belinda Cowling

Belinda Cowling receiving Prix Scientifiques 2019 (Scientific Prize).

October 2019

Dynacure presents new preclinical results on muscle targeting with antisense oligonucleotides

In October Dynacure, presented New Preclinical Results on Muscle Targeting with Antisense Oligonucleotides at the 24th Annual International Congress of the World Muscle Society (WMS).

Preclinical mouse work has shown ASO-mediated DNM2 knockdown can efficiently correct skeletal muscle defects due to loss of MTM1, providing an attractive therapeutic strategy for this disease. Dynacure scientists presented new preclinical data investigating skeletal muscle targeting of DNM2 reduction in mice with new palmitate-conjugated antisense. Effective muscle targeting will be an important step in translation of this approach to the clinic for patients.

“We are pleased to present new preclinical results at World Muscle Society that support our clinical approach of modulating DNM2 to treat CNM as well as data from a natural history study,” said Belinda Cowling, Chief Scientific Officer of Dynacure.  “In preclinical models, we’ve demonstrated that antisense oligonucleotide knockdown can efficiently correct muscle defects in certain genetic forms, XLCNM and ADCNM, which represent a large portion of the treatable patient population.  This provides an effective therapeutic strategy to treat several forms of centronuclear myopathies due to the mutations in different genes.  In addition, the data measuring clinical changes over time collected from patients enrolled in the ongoing European natural history study will continue to inform our clinical plans.  We look forward to evaluating our lead therapeutic candidate DYN101 in patients with the initiation of our Phase 1 / 2 UNITE-CNM study.”

Dynacure logo.

Audentes news 2019

May 2019

In May Audentes released new data at the American Society for Gene and Cell Therapy (ASGCT) to include more positive feedback about their AT132 gene therapy trial programme (ASPIRO), which is aimed at patients with x-linked myotubular myopathy (MTM1) aged up to five years.

Outcomes from nine patients in total were shared at the meeting. Chairman and Chief Executive Officer of Audentes, Matthew R Patterson said “We are excited to share today’s results and are working hard toward our goal of making AT132 available to patients living with XLMTM globally as rapidly as possible. We look forward to important next steps for the program, including selection of the optimal dose and further discussions with regulators in the U.S. and Europe regarding possible pathways to license applications.”

October 2019

In October Audentes presented new positive data from ASPIRO, the clinical trial evaluating AT132 in patients with X-Linked myotubular myopathy, at the 24th International Annual Congress of the World Muscle Society. The data was presented  by Dr James J Dowling from the Toronto Hospital for Sick Children. Dr Dowling said “The new ASPIRO data shared today builds upon the encouraging efficacy and safety profile seen to date with AT132. Treated patients across both dose cohorts show significant reductions in ventilator dependence and the progressive attainment of developmental motor milestones, suggesting that AT132 has the potential to deliver transformative benefit to patients and families living with XLMTM.”

Natalie Holles, President and Chief Operating Officer of Audentes said “We remain focused on our goal of rapidly progressing AT132 toward global regulatory approvals. Importantly, we have fully enrolled 14 patients into the ASPIRO dose escalation cohorts, and plan to complete enrolment of the ASPIRO pivotal expansion cohort imminently. ”

November 2019

In November the organisation reported its third quarter 2019 financial results and provided a corporate update. Matthew R Patterson, Chairman and Chief Executive Officer stated “AT132 continues to show a promising safety and efficacy profile in patients with XLMTM, with the first seven treated patients now ventilator independent.  Beyond AT132, we are excited about the significant momentum building across our entire pipeline of development candidates.”

Share your experience with x linked myotubular myopathy (xlmtm): a survey for the US X linked myotubular myopathy community

Also in November, Audentes Therapeutics  launched the xlmtm parent and caregiver community survey in the US.  Initially the survey is live for US based families, with UK and German versions coming in early 2020.

Families and caregivers economic and quality of life impact of xlmtm study.

The survey aims to gather important data related to the quality of life and economic impacts of xlmtm and to create a more thorough understanding of the quality of life and economic impact of xlmtm on families and caregivers. The results of this survey will be published and shared with the xlmtm community and may be shared with health policy makers and insurance companies to support patient access to future treatments. 

More detailed information about the study is available below.

Share your experience

Audentes logo.

 

2020 European conference

The next European centronuclear and myotubular myopathy family conference will take place in Bad Nauheim, near Frankfurt from 21 – 24 May 2020.

The conference will be hosted by:

A booking form for the event will be available shortly.

European family conference.

Attendees at European family conference.