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Novo Annual Review 2016
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innovative
treatment
for rare muscular diseases nmd
pharma
is a new drug development company that develops new treatment for chronic muscle wasting diseases such as myasthenia gravis and amyotrophic lateral sclerosis, better known as als. a pre-seed grant and, this year, a seed investment lead by novo seeds helped to build this pioneering company. b reathing, walking, running. these are physical activities that most people perform without even thinking. although they might seem basic, such actions are the result of complex signal transmissions between our nerves and muscles, all of which are activated and controlled by our nervous system. in a range of neuromuscular diseases and in some clinical conditions, however, this critical transmission from the nervous system to the muscles fails. such signal failure reduces mobility, compromises quality of life, and can lead to life-threatening muscle weakness. nmd pharma is developing a novel treatment that strengthen this critical communication between nerve and muscle. the company?s main focus is rare neuromuscular disorders with clear unmet medical needs, such as myasthenia gravis and amyotrophic lateral sclerosis, also known as als. but the treatments may work across a wide range of other neuromuscular diseases too. a new treatment approach the research that led to the discovery of the new treatment was conducted in the muscle physiology laboratory of nmd pharma?s founders, associate professor thomas holm pedersen and professor ole bękgaard nielsen ? in close collaboration with senior researcher and co-founder claus elsborg olesen at aarhus university, denmark. the decisive soft funding for nmd pharma?s ground- breaking discoveries originally came from a pre-seed grant from novo a/s in 2014. this enabled the three scientists to develop a proof-of-concept data package. they demonstrated that nmd pharma?s treatment approach could produce a five- fold increase in physical performance (running) in an animal model of myasthenia gravis (mg). a result that far exceeded current treatment options. the nmd pharma treatment is highly original in that it engages a target not previously used in neuromuscular disorders. in addition, the target is muscle specific, allowing it to be pharmacologically modulated with minimal side effects. from pre-seed grant to seed investment having seen the impressive results that the initial pre-seed grant had helped to achieve, novo?s seeds engaged with the founders to prepare a seed financing round. in may 2016, we led a dkk 20 million financing round in nmd pharma, together with lundbeckfond emerge and capnova. this round will fund further medicinal chemistry development as well as the testing of the company?s compounds in animal models of muscoskeletal disease such as mg, amylotrophic lateral sclerosis (als) and spinal muscle atrophy (sma). if successful, this will lead to a large financing that could take the treatment to test in human subjects by 2019. ?novo seeds is excited to be working with the founder team behind nmd pharma?, says stephan christgau, partner at novo seeds. ?this company is an excellent example of a prominent scientific group with an idea to address a major unmet medical need ? where a pre-seed grant has catalysed initial commercial development, and where we have been able to support the company in orchestrating the first financing round.? novo seeds looks forward to continue working with the nmd pharma team, providing them with the support they need to provide better treatment for people with neuromuscular diseases. ?nmd pharma is an excellent example of how access to crucial early-stage soft funding can transform academic discoveries into vibrant and specialised biotech startups.? thomas holm pedersen, ceo at nmd pharma. 44 novo a/s 2016
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