Only Project is a common umbrella for all projects supported by the Association Monégasque contre les Myopathies (AMM) via the funds raised from Only Watch and donations. AMM was launched in 2001 by a group of parents following the diagnosis of their children with Duchenne muscular dystrophy (DMD) with the aim of raising funds to accelerate research towards a cure for this rare genetic disease.
DMD causes the progressive weakening of all muscles (including the heart), the loss of ambulation and global autonomy, and ending in premature death. It affects 1 in 3,500 boys, amongst them two Monégasque boys: Paul Pettavino, and his friend and neighbor Philippe Ferreyrolles. Their diagnoses in 2000 led Paul’s parents Luc and Monique Pettavino to found AMM.
Shortly after creating AMM, the founders met with other parents and researchers from around the world and their goal became clear: establish a new stream of substantial funding for researchers to be able to explore therapeutic avenues. Only Watch – the biennial charity auction of unique watches – first took place in 2005 and has allowed AMM to fund very significant scientific and medical progress to the extent that a phase 1 clinical trial was launched in June 2023 to test a proprietary molecule, SQY51, thanks to the support of the charity.
➜ Get familiar with the research journey of the last 20 years and the subsequent funding strategy that was adopted in this letter to the Only Watch community by researcher Luis Garcia (Director of Research at CNRS, Director of the ENDICAP laboratory – UMR 1179 Inserm-University of Versailles-St-Quentin, UFR santé Simone Veil).
➜ 🎥 Look back on 20 years of history of our commitment with the film “The Story of Us”.
Funds allocation strategy and decisions: an agile and potent partner for researchers.
After years of organizing international roundtables and a large research program that financed projects led by scientists in universities around the world to refine the therapeutic avenues on DMD, the general strategy adopted by AMM has been to give priority support to the “Tricyclo-DNA” project: a technological breakthrough for which the AMM has provided itself, through Only Watch, the financial resources to support “from the laboratory bench to the patients“. As a result, in the past years, the Association’s funding has been distributed in majority to biotech organizations directly associated with the charity and representatives of the families of patients. These biotech organizations have worked on the development of a drug that is currently being tested in a phase 1 clinical trial in France (more information on the trial here: clinical trials.gov) on 12 boys and young men. Although we remain cautious while waiting for outcomes, it is an achievement that we can collectively be proud of.
This choice has never ruled out support for other projects, be they therapeutic alternatives, more fundamental research, or even research into diseases other than Duchenne muscular dystrophy but which could have an impact on Duchenne research. As a result, around 10% of the charity’s funds are allocated to grants as well as doctoral and post-doctoral fellowships for researchers in universities and hospitals internationally. This allows the scientific community to explore new therapeutic avenues without detracting from the overall strategy.
On an operational level, teams requesting support may submit their projects to the AMM, which are then evaluated by its network of experts. There is no fixed call for tenders; the door is always open and processed on an ad hoc basis, with the aim of allocating funds as quickly as possible once a project has been selected. This has made us a particularly agile partner for researchers – in a field where response time is usually very long and hinders progress.
The attribution criteria that are evaluated with our community of researchers are: quality of the teams, interest of the projects in the strategic context as defined by the college of parents surrounded by the experts of their choice.
To be noted, for the past three years, a specific fund has been allocated via the FSRMM (Swiss Muscular Dystrophy Research Association) for doctoral and post-doctoral grants, jointly evaluated by the FSRMM scientific council and validated by the AMM.
Management of funds for future developments.
The financial resources required for current and future clinical developments are considerable, running into millions of euros and increasing at an exponential rate with each new phase. The association has adopted a conservative approach to spending the funds to make sure that it is in capacity to honor its commitment to the clinical developments that it is supporting and that necessitate very substantial means to go through, namely: following phases of the SQY51 clinical trial, development of the molecule for other exons of Duchenne muscular dystrophy (exons 16 and 44 are in preparation) as well as for other genetic diseases (research partnerships have been sealed on spinal muscular atrophy and epidermolysis bullosa), and the launch of new clinical trials testing the safety and efficacy of the molecules.
Financial statements 2018-2022.
Want to know more about how the charity funds are distributed and spent? Please find below the detailed financial statements of the AMM for years 2018 through 2022 as well as an activity highlight year by year for this period. We invite you to consult the more detailed versions of the accounts following the summarized versions, which are more informative and provide a fuller context.
Please note that:
☝️Only Watch being the charity’s biennial fundraising event, yearly “functioning expenses” vary significantly according to whether Only Watch takes place that year or not. Overall, they represent between 1 and 2% of funds raised.
☝️Only Watch taking place once every two years, usually in November, part of the auction proceeds are transferred by the auction house at the beginning of the following year and are therefore accounted for in the following financial statement.
☝️Funds distributed to biotechs that are working on a potential therapy (SQY Therapeutics, Synthena) appear as debts in the “Participating Interest” section for sums not yet abandoned. All sums advanced by the Association are intended to be waived. They are then transferred to the expenses account.