Minoryx science approach

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title="Other CNS diseases">Other CNS diseases</a></div> </div> <div class="sta-hoverMobile"> <a href="javascript:openLink('/our-pipeline-overview/')" title="Our Programs">Our Programs <span class="minusplus"><i class="plus"></i></span><span class="minusplus"><i class="minus"></i></span></a> <div class="m-submenu sta-hoverOFF"> <a href="/our-pipeline-overview/" title="Overview">Overview</a><a href="/leriglitazone/" title="Leriglitazone">Leriglitazone</a></div> </div> <div class="sta-hoverMobile"> <a href="javascript:openLink('/clinical-studies/')" title="Clinical Studies">Clinical Studies <span class="minusplus"><i class="plus"></i></span><span class="minusplus"><i class="minus"></i></span></a> <div class="m-submenu sta-hoverOFF"> <a href="/clinical-studies/" class="tablet-only">Clinical Studies</a> <a href="/clinical-studies/clincal-study-calyx/" title="CALYX">CALYX</a><a href="/clinical-studies/clinical-study-nexus/" title="NEXUS">NEXUS</a><a 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data-tablet="/content/imgsxml/panel_herointerior/tab-brain709.jpg" data-desktop="/content/imgsxml/panel_herointerior/brain709.jpg"> <div class="m-content"> <div class="m-title"><div>Overview</div></div> <div class="mdl-hilo"> <div class="m-content" itemscope="" itemtype="http://schema.org/BreadcrumbList"> <span itemprop="itemListElement" itemscope="" itemtype="http://schema.org/ListItem"><a itemprop="item" class="home" href="/"><span itemprop="name" class="dnone">Home</span></a><meta itemprop="position" content="0"/></span><i class="fa fa-angle-right" aria-hidden="true"></i> <span itemprop="itemListElement" itemscope="" itemtype="http://schema.org/ListItem"><a itemprop="item" title="Our Science" href="/our-science-overview/"><span itemprop="name">Our Science</span></a><meta itemprop="position" content="1"/></span> <i class="fa fa-angle-right" aria-hidden="true"></i></span><span itemprop="itemListElement" itemscope="" itemtype="http://schema.org/ListItem"><a itemprop="item" href="https://www.minoryx.com/our-science-overview/"></a><span><a itemprop="item" href="/our-science-overview/"></a><span class="selected" title="Overview" itemprop="name">Overview</span></span><meta itemprop="position" content="2"/></span> </span> </div> </div> </div> </article> </section></li> </ul> <ul id="panel_texto4_0" class="panel_texto"> <li data-index="0" id="panel_texto_item11_0" class="item col-0 clearfix"><section class="mdl-texto"> <div class="m-content"> <div class="m-title"><div>Orphan CNS diseases</div></div> <div class="m-text sta-see_more"> <div data-mas="Read more" data-menos="Read less" s_see_more-data="3,,,"><p style="text-align: justify;">Minoryx focuses on <strong>orphan diseases of the central nervous system (CNS)</strong>, a large group of rare diseases often characterized by <strong>neurodegeneration</strong> and for which there are currently no treatments available. These diseases are usually chronic, progressively debilitating and often life-threatening, with <strong>high unmet medical need.</strong></p> <p style="text-align: justify;">Some orphan CNS diseases are of genetic origin, but they can also be triggered by external unknown factors and may affect other organs in addition to the brain. Several complex, interplaying pathways are contributing to the neurodegenerative process: mitochondrial dysfunction, oxidative stress, and neuro-inflammation all play an important role in neurodegeneration, independent of the underlying cause of disease. Research into novel effective treatments, like leriglitazone, addresses this complexity, rather than focus on a single pathway approach.</p> <p style="text-align: justify;"><strong>PPAR gamma</strong> controls multiple genes that are central to mitochondrial biogenesis, oxidative stress, inflammation, and myelination. The potential beneficial effects of PPAR gamma agonists in CNS disorders have been demonstrated preclinically in several orphan and non-orphan indications such as Multiple Sclerosis, Parkinson鈥檚 Disease, Alzheimer鈥檚 Diseases, Huntington鈥檚 Disease, Amyotrophic Lateral Sclerosis, Stroke and Traumatic Brain Injury. However, to date clinical studies failed to show beneficial effects of PPAR gamma agonists in these indications, due to insufficient target engagement in the CNS.</p> <p style="text-align: justify;">Minoryx have discovered and developed a novel, selective PPAR gamma agonist,<span style="color: #00ccff;"><a style="color: #00ccff;" href="/leriglitazone/">聽leriglitazone</a></span>, which is currently in clinical development for multiple orphan CNS disorders. The lead indication for leriglitazone is X-linked Adrenoleukodystrophy (X-ALD), a devastating neurodegenerative disease that exists in two forms: cerebral ALD (cALD), characterized by brain lesions that can become rapidly progressive leading to death, and adrenomyeloneuropathy (AMN) characterized by spinal cord degeneration. Leriglitazone demonstrated robust preclinical proof-of-concept in relevant animal models of disease and successfully completed phase 1 clinical trials. Leriglitazone completed a <span style="color: #00ccff;"><a style="color: #00ccff;" href="/clinical-studies/clinical-study-advance/">phase 2/3 clinical study in adult聽</a><a style="color: #00ccff;" href="/clinical-studies/clinical-study-advance/">patients with X-ALD (ADVANCE)</a></span>聽in the EU and US showing a significant reduction of cerebral lesion progression and a reduction of incidence of progressive cALD and myelopathy symptoms.聽Additionally, a separate <a href="/clinical-studies/clinical-study-nexus/"><span style="color: #00ccff;">study in paediatric patients with cALD (NEXUS)</span></a>聽is currently ongoing in EU and after 24 weeks of treatment, all evaluable patients in NEXUS were clinically stable and radiologically demonstrated disease arrest or lesion growth stabilization. Finally, a聽<span><a href="/clinical-studies/clincal-study-calyx/"><span style="color: #00ccff;">phase 3 study in adult male patients with progressive cALD (CALYX)</span></a></span> is currently recruiting in the US. The marketing authorization application (MAA) for adult male X-ALD patients is currently under review by the EMA. Leriglitazone offers a strong potential for indication expansion into other CNS diseases. In this regard, a proof of concept study in Friedreich's Ataxia (FRDA) showed clinical benefit in this population.聽Leriglitazone is also being evaluated in other rare CNS disorders.</p></div> <footer class="see_more" s_see_more></footer> </div> </div> </section></li> </ul> <ul id="panel_destacado4_1" class="panel_destacado"> <li data-index="0" id="panel_destacado_item12_1" class="item col-0 clearfix"><section class="mdl-destacado"> <article> <div class="m-content"> <div class="m-image deferbg" data-mobile="/content/imgsxml/panel_destacado/neurona-blue450.jpg" data-tablet="/content/imgsxml/panel_destacado/neurona-blue450.jpg" data-desktop="/content/imgsxml/panel_destacado/neurona-blue450.jpg"> <img src="/content/imgs/white.png" class="deferimg" data-mobile="/content/imgsxml/panel_destacado/neurona-blue450.jpg" data-tablet="/content/imgsxml/panel_destacado/neurona-blue450.jpg" data-desktop="/content/imgsxml/panel_destacado/neurona-blue450.jpg" alt="" title="" > </div> <div class="m-texto"> <div> <div class="m-pre" data-mod="titulo"><div>Our focus</div></div> <div class="m-title"><div>Adrenoleukodystrophy</div></div> <div class="m-text"><p style="text-align: justify;">X-linked Adrenoleukodystrophy (X-ALD) is an orphan neurodegenerative disease caused by a mutation in the ABCD1 gene. X-ALD presents two main neurologic phenotypes. The most common phenotype is adrenomyeloneuropathy (AMN), characterized by progressive severe motor and sensory dysfunction and which affects young male adults and adult women.聽Cerebral ALD (cALD) is characterized by demyelinating brain lesions that may become rapidly progressive, leading to acute neurological decline and death. These lesions can produce severe symptoms such as loss of voluntary movements, inability to swallow, loss of communication, cortical blindness and total incontinence and when progressive death with a mean survival of 3 to 4 years. Progressive cALD occurs in 31-35% of X-ALD patients in childhood with typical onset between the age of 2-12 and up to 60% of adult patients, with X-ALD will develop progressive cALD over time.</p></div> </div> <div> <div class="m-button"><a class="m-boton sta-hoverOFF" href="/adrenoleukodystrophy-orphan-disease/" target="_self"><i></i><i></i><i></i><i></i>Read more</a></div> </div> </div> </article> <article> <div class="m-content"> <div class="m-image deferbg" data-mobile="/content/imgsxml/panel_destacado/neurone-darkblue569.jpg" data-tablet="/content/imgsxml/panel_destacado/neurone-darkblue569.jpg" data-desktop="/content/imgsxml/panel_destacado/neurone-darkblue569.jpg"> <img src="/content/imgs/white.png" class="deferimg" data-mobile="/content/imgsxml/panel_destacado/neurone-darkblue569.jpg" data-tablet="/content/imgsxml/panel_destacado/neurone-darkblue569.jpg" data-desktop="/content/imgsxml/panel_destacado/neurone-darkblue569.jpg" alt="" title="" > </div> <div class="m-texto"> <div> <div class="m-pre" data-mod="titulo"><div>Our focus</div></div> <div class="m-title"><div>Other CNS diseases</div></div> <div class="m-text"><h2><strong>FRDA</strong></h2> <p style="text-align: justify;">Friedreich's ataxia (FRDA) is an orphan neurodegenerative genetic disease characterized by frataxin deficiency and resulting in mitochondrial dysfunction affecting the central nervous system and the heart. In the majority of cases, FRDA has an onset before the age of 25 and progressively impairs motor function, leading to ataxic gait, cardiac abnormalities and other medical co-morbidities.</p></div> </div> <div> <div class="m-button"><a class="m-boton sta-hoverOFF" href="/friedreich-ataxia-orphan-disease/" target="_self"><i></i><i></i><i></i><i></i>Read more</a></div> </div> </div> </article> </section></li> </ul> <ul id="panel_footer4_2" class="panel_footer"> <li><section class="mdl-footer"> <div class="m-content"> <div class="m-inter"> <div class="m-offices"> <a href="/contact/" class="m-boton2 sta-hoverOFF"><i></i><i></i><i></i><i></i>Contact</a> <article itemscope itemtype="http://schema.org/LocalBusiness"> <header itemprop="name">Headquarters</header> <span itemprop="image" class="hide">https://www.minoryx.com/content/imgs/mail/logo.png</span> <address itemprop="address" itemscope itemtype="http://schema.org/PostalAddress"> <span itemprop="streetAddress">Av. 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Minoryx science approach