Author: Rachael Hansford

Company’s multiple sclerosis Imotope™ shows promising preclinical efficacy and histology results while eliciting immune responses supporting the proposed mode of action and providing proof of concept

Liège, Belgium, September 12, 2019 – Imcyse, a clinical-stage company developing active and specific immunotherapeutics for the treatment and prevention of severe chronic diseases, today announces the presentation of a poster describing results obtained in the experimental autoimmune encephalomyelitis model (EAE), the widely used model for multiple sclerosis (MS).

Results of the experiments have shown an excellent inhibition of disease development, a statistically significant histological improvement and immunological markers supporting the mode of action.

The poster (P982) will be presented at ECTRIMS, Stockholm, Sweden by Dr David Walgraffe on September 12, during poster session 2, from 5:15pm to 7:15pm CEST.

Imcyse’s unique technology platform is based on specifically modified peptides (Imotopes™) which drive the generation of cytolytic CD4 T cells. These cytolytic CD4 T cells are able to actively and specifically target the immune cells involved in the pathogenesis of the respective autoimmune disease. There is potential with this therapy to disrupt undesirable autoimmune responses that drive the myelin sheath destruction and to stop disease progression in MS.

Imcyse’s MS Imotope™ is the company’s second most advanced product candidate after the type 1 diabetes (T1D) Imotope™ IMCY-0098. Imcyse is currently preparing for a phase 1-2 clinical study with an MS Imotope™.

The positive results of the Imcyse MS Imotope™ in the EAE model are very encouraging,” said Thomas Taapken, executive chairman of Imcyse. “After the positive clinical results in T1D, they further validate Imcyse’s unique technology platform.”

The MS program has received financial support from the Walloon Region (DGO6).

About Imcyse
Imcyse develops active targeted immunotherapies to treat and prevent severe chronic diseases caused by disruptions of the immune system. The company’s unique active immunotherapy technology platform allows it to locally target immune cells involved in the destruction of the diseased organ. This platform is based on the administration of Imotopes™, which are specifically modified peptides, allowing for the generation of cytolytic CD4 T cells. Imcyse’s approach, sustained over time, helps to prevent and treat diseases with no current therapeutic alternative and to potentially cure patients without impairing immune defense. The company has established proof of concept in several indications and has completed its first clinical trial in type 1 diabetes in seven European countries. Beyond type 1 diabetes, Imcyse is developing a pipeline of Imotopes™ for the treatment of several autoimmune diseases. Based in Liège, Belgium, Imcyse was originally founded in 2010 as a spin-off from the KU Leuven, Belgium.
www.imcyse.com

People living with conditions like Parkinson’s could benefit from quicker diagnosis and better coordinated care as part of a new NHS initiative, which is also set to free up millions of pounds to reinvest in patient care.

NHS experts have joined forces with seven leading charities to produce a toolkit that will help local health groups improve services for people with conditions including Multiple Sclerosis (MS), Motor Neurone Disease (MND) and Parkinson’s, including rolling out fast-tracked blood tests and consultant appointments over Skype for those who want them.

Up to 2,500 emergency admissions to hospital a year could be avoided for patients with these conditions as a result, with up to £10 million freed up to fund improved services.

The guidance aims to help Clinical Commissioning Groups (CCGs) deliver faster diagnosis and treatment for progressive neurological conditions, better join-up the different health professionals and organisations supporting each individual, and improve access to specialist physical and mental health care.

People will also be supported to better understand and manage their symptoms at home, and to take a more informed and active role in making decisions about the treatment they receive.

Created with Parkinson’s UK, MS Society, MS Trust, MND Association, Sue Ryder, MSA Trust and PSP Association, the guidance comes as local health groups are working with staff and communities to develop their plans to join up and improve care over the next five years, to deliver the benefits for patients set out in the NHS Long Term Plan.

Dawn Chamberlain, Programme Director – Clinical Improvement at NHS England and NHS Improvement, said:

This is an opportunity for the NHS to work directly with patients and their families to deliver better care closer to home for the thousands of people with progressive neurological conditions. As they work with staff, patients and stakeholders to implement the NHS Long Term Plan, this toolkit provides the information local health leaders need to understand how to deliver high quality neurology services. Many areas are already providing high quality care in line with best practice, and by supporting others to come up to the same standard, we can deliver faster, more joined-up and better care for thousands more people – supporting them to stay well in their own homes.

The Progressive Neurological Conditions Toolkit is the latest publication from the NHS RightCare programme, which was set up to provide support to local CCGs to improve care in key areas, based on the best-available data, evidence and intelligence.

Alongside the best practice guidance, CCGs will receive extensive data on local performance, and tailored support to improve their offer to patients.

The charities who have worked closely with the NHS RightCare team to develop the toolkit will continue to support efforts to see it implemented effectively, with the shared aim of improving care for everyone living with a progressive neurological condition in England.

www.england.nhs.uk/rightcare/products/pathways/progressive-neurological-conditions-toolkit/

PathMaker Neurosystems Inc. (“PathMaker”), a clinical-stage bioelectronic medicine company developing breakthrough non-invasive systems for the treatment of patients with spasticity and paralysis has announced the first publication of clinical trial results for its MyoRegulator® device for the non-invasive treatment of spasticity. Published in Bioelectronic Medicine, the results provide the first clinical evidence using MyoRegulator to treat upper extremity spasticity in subjects with chronic stroke. MyoRegulator is an investigational medical device and is limited by US Federal law to investigational use only.

Spasticity is a chronic condition characterised by painful muscle contractions and is common in patients suffering from stroke, cerebral palsy, multiple sclerosis, spinal cord injury, traumatic brain injury and other neurological disorders. Management of spasticity is a difficult challenge and is currently managed primarily by pharmacological agents and injected botulinum neurotoxins, and there is tremendous unmet medical need.  MyoRegulator is a first-in-class non-invasive device based on PathMaker’s proprietary DoubleStim™ technology (combining anodal trans-spinal direct current stimulation (tsDCS) and peripheral nerve direct current stimulation (pDCS)), which provides simultaneous non-invasive stimulation intended to suppress hyperexcitable spinal neurons involved with spasticity.

“Current pharmacological approaches to managing spasticity have, at best, short-term efficacy, are confounded by adverse effects, and are often unpleasant for the patient,” said co-author Zaghloul Ahmed, Ph.D., Professor and Chairman, Department of Physical Therapy and Professor, Center for Developmental Neuroscience, CUNY and Scientific Founder of PathMaker Neurosystems. “The initial study results demonstrate the potential of a novel, non-invasive treatment to reduce spasticity and improve functional recovery in patients with upper motor neuron syndrome after stroke.”

The publication, Non-Invasive Treatment of Patients with Upper Extremity Spasticity Following Stroke Using Paired Trans-spinal and Peripheral Direct Current Stimulation, was authored by researchers at Feinstein Institute for Medical Research at Northwell Health (Manhasset, NY) led by Bruce Volpe, M.D. The study included patients with upper limb hemiparesis and wrist spasticity at least 6 months after their initial stroke in a single-blind, sham-controlled, crossover design study to test whether MyoRegulator treatment reduces chronic upper-extremity spasticity.

Twenty subjects received five consecutive 20-minute daily treatments with sham stimulation followed by a 1-week washout period, then five consecutive 20-minute daily treatments with active stimulation. Subjects were told that the order of active or sham stimulation would be randomized. Clinical and objective measures of spasticity and motor function were collected before the first session of each condition (baseline), immediately following the last session of each condition, and weekly for 5 weeks after the completion of active treatments. The results demonstrated significant group mean reductions from baseline in both Modified Tardieu Scale scores (summed across the upper limb, P<0.05), and in objectively measured muscle resistance at the wrist flexor (P<0.05) following active treatment as compared to following sham treatment. Motor function also improved significantly (measured by the Fugl-Meyer and Wolf Motor Function Test; P<0.05 for both tests) after active treatment, even without additional prescribed activity or training. The effect of the active MyoRegulator treatment was durable for the 5-week follow-up period.

We are highly encouraged by these clinical results which demonstrate the potential of MyoRegulator to improve outcomes for patients suffering from spasticity, without the need for surgery or drugs. Building on these results and our ongoing clinical trial in Europe, we expect to initiate a US multi-center, pivotal, double-blind clinical trial supported by the National Institute of Neurological Disorders and Stroke (NINDS) in early 2020.

Nader Yaghoubi, M.D., Ph.D., President and Chief Executive Officer of PathMaker

About PathMaker Neurosystems Inc.

PathMaker Neurosystems is a clinical stage bioelectronic medicine company developing breakthrough non-invasive systems for the treatment of patients with chronic neuromotor conditions. With offices in Boston (US) and Paris (France), we are collaborating with world-class institutions to rapidly bring to market disruptive products for treating spasticity, paralysis and muscle weakness. In January 2019, we announced a collaboration and distribution agreement with WeHealth Digital Medicine to commercialise the MyoRegulator® device worldwide, excluding US and Japan territories retained by PathMaker.  More than 48 million patients in the US, Europe and China suffer disabilities due to stroke, cerebral palsy, multiple sclerosis, spinal cord injury, traumatic brain injury, Parkinson’s disease and other neurological disorders. At PathMaker, we are opening up a new era of non-invasive neurotherapy for patients suffering from chronic neuromotor conditions. For more information, please visit the company website at www.pmneuro.com.

Source: PathMaker Neurosystems Inc.

Biogen has announced new results from the NURTURE study, adding data to the longest study of spinal muscular atrophy (SMA) in pre-symptomatic infants (n=25).

These data reported, after up to 45.1 months of analysis, continue to demonstrate efficacy and safety in patients treated pre-symptomatically with nusinersen in comparison to the natural history of this disease. These new data also showed that patients treated with nusinersen had continuous improvement in sitting and walking independently, with the overwhelming majority of patients achieving motor milestones in a normal timeframe.¹

These data were presented at the Cure SMA Annual SMA Conference in Anaheim, CA (June 28-July 1, 2019) and the 5th Congress of the European Academy of Neurology (EAN) in Oslo, Norway (June 29-July 2, 2019). “These study results demonstrate the durable impact of pre-symptomatic, proactive treatment on transforming the natural course of this disease. We are seeing an extensive number of patients continually meeting child motor development milestones,” said Darryl De Vivo, M.D., Sidney Carter Professor of Neurology and Paediatrics, Columbia University Irving Medical Center in New York, New York. “Nusinersen is setting patients on a path toward survival, greater mobility and independence from the start of treatment, which is helping improve outcomes for patients of all ages.”

Results from NURTURE, an ongoing, Phase 2, open-label study of 25 pre-symptomatic patients with SMA (most likely to develop SMA Type 1 or 2) who received their first dose of nusinersen before six weeks old, demonstrated results not before seen in comparison to the natural history of SMA. As of March 20191:

• 100 percent were alive without a need for permanent ventilation.¹
• The median age of the study participants was nearly three years old. The majority of untreated patients with SMA Type 1 never reach their second birthday without permanent ventilation.¹
• 100 percent of the infants were sitting independently, in comparison to the natural history of this disease where no patients with SMA Type 1 would be able to do so and patients with SMA Type 2 would need assistance.¹
• 88 percent of the infants were walking independently with many of them doing so in the normal timeframe for a toddler. In the natural history of SMA, patients with SMA Type 1 or Type 2 are never able to walk independently.¹
• Patients were approaching the maximum mean score of 64 on the CHOP INTEND measure of motor function– 63.4 for patients with 3 SMN 2 copies (n=10) and 62.1 for those with 2 SMN 2 copies (n=15), demonstrating the impact of early treatment.¹
• Nusinersen demonstrated efficacy up to nearly 4 years, with participants continuing to make progress and showing no signs of loss of motor function.¹
• Nusinersen was well-tolerated with no new safety concerns identified after up to nearly 4 years of treatment.¹

“A few years ago, SMA patients had no treatment options and faced significant care challenges,” said Kenneth Hobby, President of Cure SMA, a patient advocacy organisation dedicated to the treatment and cure of SMA. “However, the future of SMA has changed and especially with early treatment patients now have a chance to reach age appropriate developmental milestones. These new data demonstrate the impact where children are now walking independently at four years of age, when the usual lifespan, for those who are most severe, would be under two if untreated¹. This study provides additional evidence on the maintenance of these improvements. It’s critical that research in SMA continues to support the generation of real-world evidence in patients of all ages so that we better understand the long-term implications of SMA and treatment across all types.”

Additional presentations at the two meetings highlighted results from the ongoing open-label SHINE extension study of children with infantile and later-onset SMA as well the CS2/CS12 analysis of older patients. Biogen also continues to explore the scientific value of phosphorylated neurofilament heavy chain (pNF-H) and  presented new data on the ongoing evaluation of its potential as a biomarker in SMA.