Author: Donna Earl

UKABIF Award Winners Announced at Time for Change Summit

Posted on January 18, 2022 by - Acquired Brain Injury

The winners of the annual UK Acquired Brain Injury Forum (UKABIF) awards have been honoured at this year’s Time for Change summit.

The Awards include the UKABIF Film Award, the Mike Barnes Award for Innovation, the Stephen McAleese Award for Inspiration and the UKABIF Poster Award. The Awards were kindly sponsored by Elysium Healthcare.

Nicola Leyland won the UKABIF Film Award for her moving film titled ‘A Freak Accident’. The animation tells the story of her son who sustained a brain injury aged eight years and documents the trauma they both went through and his journey to significant recovery. You can watch the film here

The Mike Barnes Award for Innovation, went to MyAbility created by Dr Andrea Pickering, Consultant Clinical Psychologist of Recolo; Fran Sephton, Emma Bowers and Niki Dutton, Highly Specialist Speech and Language Therapists of ATtherapy. They will receive support from the National Institute for Health Research to advance their project and mentoring from Dr Penny Trayner of Goal Manager.

Anita Pascoe received the Stephen McAleese Award for Inspiration. In July 2000, Anita Pascoe’s 54-year-old Mum, Ann, collapsed following a brain hemorrhage and she remained severely brain injured until her death earlier this year. For 20 years Anita fought to get her Mum properly supported. Accepting the Award, Anita said: “I have been inspired by so many people who have survived acquired brain injury and overcome huge challenges. We had to fight so hard over the years and that is just not right. You should not need to have someone fighting your corner in order for your needs to be met.”

The UKABIF Poster Award was won by Lead Neuropsychologist Dr Henk Swanepoel of Cygnet Health Care for his project ‘Implementing a Study to Compare Diagnostic Accuracy of Cognitive Screening Instruments: A Weighted Comparison Approach in Acquired Brain Injury.”

Chloe Hayward, Chief Executive of UKABIF, said: “Our Awards are our small way of recognising the amazing people and organisations who are doing their bit to improve the lives of those affected by acquired brain injury.

“These awards are held every year so we would encourage people to nominate others so we can celebrate the unsung heroes and recognise the great work being done across the country.”  

www.ukabif.org.uk

ACNR Podcast – celebrating 20 years of ACNR

Posted on January 10, 2022 by - ACNR News

It is a great pleasure to mark ACNR’s 20th anniversary with a new departure for the reviews section. This is our first podcast review. And what better inaugural podcast to review than the ACNR’s very own, in the form of an interview by Sri Kodali. Sri questions no less a pair than Rachael Hansford, ACNR publisher for the entirety of its first 20 years, and ACNR’s founding editor, Professor Roger Barker. I hope I may be forgiven for throwing all objectivity and caution to the wind, and saying that I thought it was absolutely great. Seriously though, it really was. This was a delightful half-hour of two friends enjoying a bright and breezy yet erudite conversation, with a few judicious prompts from Sri, and an occasional ‘mensh’ for such luminaries as Alasdair Coles and Mike Zandi. Regaining my composure, I can say objectively that it’s all very well produced and well presented. And what a great story! These are people who have found a way of working on stuff they enjoy. Better still, it’s a story of being proactive with pragmatism to create something worthwhile…ACNR. I thought it was a mini masterclass in managing, and avoiding, both undue hesitancy and excess risk.
This podcast encompasses life, brain, anxiety, motivation, and joy, and of course it’s all Neuroscience.

Reviewed by: Rhys Davies, Consultant Neurologist at the Walton Centre for Neurology and Neurosurgery in Liverpool and at Yssbyty Gwynedd in Bangor, North Wales, UK.

UCB announces positive Phase 3 results for rozanolixizumab in generalised myasthenia gravis

Posted on December 10, 2021 by - Myasthenia Gravis

  1. Positive topline results from UCB MycarinG study investigating the efficacy and safety of rozanolixizumab in patients with generalised myasthenia gravis (gMG)
  2. Study met primary and all secondary endpoints with statistical significance
  3. Rozanolixizumab was well tolerated with no new observed safety signals 
  4. UCB plans regulatory submissions for rozanolixizumab in gMG from Q3 2022 

December 10th, 2021 – UCB, a global biopharmaceutical company, announced positive topline results from the Phase 3 MycarinG study [1] evaluating rozanolixizumab, a subcutaneously (SC) infused monoclonal antibody targeting the neonatal Fc receptor (FcRn), versus placebo in adults with generalised myasthenia gravis (gMG).

The trial met its primary endpoint, demonstrating a statistically significant and clinically meaningful change from baseline in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at Day 43. All secondary endpoints were also met with statistical significance. 

Overall rozanolixizumab was well tolerated and no new safety signals were identified. 

The safety and efficacy of rozanolixizumab have not been established, and it is not approved for use in any indication by any regulatory authority worldwide. The final Phase 3 data from the study and additional details will be presented at a forthcoming medical meeting in 2022. 

Based on these results, UCB anticipates regulatory filings in the US, European Union and Japan from Q3 2022.  

“Today’s encouraging findings from the MycarinG study show the potential of rozanolixizumab in the treatment of myasthenia gravis, and further reinforce the suggestion that FcRn inhibition may be a promising approach for this disease,” explained Professor Vera Bril, MycarinG study Lead Investigator, Professor of Medicine (Neurology), University of Toronto, and Director of the Neuromuscular Section, Division of Neurology, University of Toronto and University Health Network, Toronto, Canada.

Alongside rozanolixizumab, UCB is also investigating whether its developmental medicine zilucoplan, a peptide inhibitor of complement component 5 (C5 inhibitor), could deliver patient value to people living with gMG. Preliminary results from the company’s RAISE study are expected in the coming weeks. The safety and efficacy of zilucoplan have not been established, and it is not approved for use in any indication by any regulatory authority worldwide.

“For the many thousands of people living with myasthenia gravis around the world, current treatment options can be very limited,” said Samantha Masterson, Chief Executive Officer of the Myasthenia Gravis Foundation of America (MGFA). “Given that this disease causes a wide range of symptoms, some of which can require urgent intervention or hospitalization, there is a critical need for new treatment options that could address the unmet needs of patients living with myasthenia gravis.”  

UCB’s portfolio approach of two medicines with different but potentially complimentary mechanisms of action creates a unique opportunity for UCB to deliver choice, flexibility and impact to patients and healthcare professionals, giving them options best suited to their individual needs.

“We are enthusiastic about these positive and clinically meaningful results, which mark a critical step forward for rozanolixizumab and UCB’s commitment to delivering differentiated solutions for people living with rare diseases, such as myasthenia gravis,” said Iris Loew-Friedrich, Executive Vice-President and Chief Medical Officer at UCB. “In line with our ambition to deliver a portfolio of treatment options which could improve and simplify the treatment experience for patients and physicians, we are committed to bringing transformational outcomes and experiences to those in need. We wholeheartedly thank the MG community for their ongoing partnership and participation in this study.”

References

  1. Data on file. UCB. December 2021.
  2. Myasthenia Gravis Foundation of America. Clinical Overview of MG. https://myasthenia.org/Professionals/Clinical-Overview-of-MG. Accessed November 2021.
  3. Chen J, et al. Incidence, mortality, and economic burden of myasthenia gravis in China: A nationwide population-based study. Lancet Reg Health West Pac.2020;5:10063.
  4. Gilhus N. Myasthenia Gravis. N Engl J Med. 2016;375:2570-2581.
  5. Lisak, RP. Best Practice Myasthenia gravis. BMJ Best Practice. 2021. Last accessed December 2021
  6. Robertson NP, et al. Myasthenia gravis: a population based epidemiological study in Cambridgeshire, England. J Neurol Neurosurg Psychiatry. 1998;65:492-496.
  7. Kupersmith MJ et al. Development of generalized disease at 2 years in patients with ocular myasthenia gravis. Arch Neurol. 2003;60(2):243-248. 
  8. Hansen JS, et al. Mortality in myasthenia gravis: A nationwide population-based follow-up study in Denmark. Muscle Nerve. 2016;53:73-77.
  9. Clinical Trials.gov ‘A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis’:  https://clinicaltrials.gov/ct2/show/NCT03971422. Accessed November 2021.
  10. Clinical Trials.gov ‘Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis (RAISE)’: https://clinicaltrials.gov/ct2/show/NCT04115293. Accessed November 2021.
  11. Kiessling P, et al. The FcRn inhibitor rozanolixizumab reduces human serum IgG concentration: A randomized phase 1 study. Sci Transl Med. 2017;9(414).
  12. Smith B, et al. Generation and characterization of a high affinity anti-human FcRn antibody, rozanolixizumab, and the effects of different molecular formats on the reduction of plasma IgG concentration. MAbs.2018;10:1111-30.

About Generalized Myasthenia Gravis (gMG)

gMG is a rare, chronic and unpredictable auto-immune disease in which pathogenic autoantibodies can inhibit synaptic transmission at the neuro-muscular junction by targeting specific proteins on the post-synaptic membrane. This disrupts the way that the nerves can communicate with muscles. gMG can occur at any age and in any race, although previous studies have shown that women are more often impacted than men [2].

Myasthenia gravis is a rare disease impacting almost 200,000 patients in the US, EU and Japan [3,4]. People living with gMG can experience a variety of symptoms, including drooping eyelids, double vision and difficulty swallowing, chewing and talking, as well as severe muscular weakness that can result in life threatening weakness of the muscles of respiration [5-8]. 

About the rozanolixizumab MycarinG study [9]

The MycarinG study (NCT03971422) is a multi-center, Phase 3, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of rozanolixizumab in adult patients with gMG, with an open-label extension. 

The primary endpoint for the MycarinG study is change in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) score, an eight-item patient-reported scale developed to assess MG symptoms and their effects on daily activities. Secondary endpoints include response rates, changes in the Myasthenia Gravis composite (MGC) score, the Quantitative MG (QMG) score, patient-reported outcomes and adverse events (AEs). 

For more information about the trial, visit https://clinicaltrials.gov/ct2/show/NCT03971422. 

About the zilucoplan RAISE study [10]

The RAISE study (NCT04115293) is a multi-center, Phase 3, randomized, double-blind, placebo-controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in patients with gMG. Patients will be randomized in a 1:1 ratio to receive daily subcutaneous (SC) doses of zilucoplan or placebo for 12 weeks.

The primary endpoint for RAISE study is change from baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score. Secondary endpoints include change in the Quantitative Myasthenia Gravis (QMG) score, the Myasthenia Gravis Composite (MGC) and the Myasthenia Gravis Quality of Life 15 revised (MG-QoL15r) from baseline to Week 12; the proportion requiring rescue therapy; the proportion with minimum symptom expression (MSE) (defined as MG-ADL of 0 or 1), the proportion with a ≥3-point reduction in MG-ADL and the proportion with a ≥5-point reduction in QMG, all measured at Week 12.

For more information about the trial visit https://clinicaltrials.gov/ct2/show/NCT04115293. 

About Rozanolixizumab

Rozanolixizumab is a SC administered, humanized monoclonal antibody that specifically binds, with high affinity, to human neonatal Fc receptor (FcRn). It has been designed to block the interaction of FcRn and Immunoglobulin G (IgG), accelerating the catabolism of antibodies and reducing the concentration of pathogenic IgG autoantibodies [11,12].

Rozanolixizumab is under clinical development with the aim of improving the lives of people with pathogenic IgG-autoantibody-driven autoimmune diseases, including gMG, primary immune thrombocytopenia (ITP), myelin oligodendrocyte glycoprotein antibody-associated disease (MOG-AD) and auto immune encephalitis (AIE) by driving removal of pathogenic IgG autoantibodies. 

The safety and efficacy of rozanolixizumab have not been established and it is not approved for use in any indication by any regulatory authority worldwide.

About Zilucoplan 

Zilucoplan is a once-daily self-administered SC peptide inhibitor of complement component 5 (C5 inhibitor) under clinical development by UCB in gMG. Topline results from the RAISE study, a multi-center, Phase 3, randomized, double-blind, placebo-controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with gMG, are expected in H1 2022.

Further indications that are potentially addressable by zilucoplan include amyotrophic lateral sclerosis (ALS) and other tissue-based complement-mediated disorders with high unmet medical need. 

Zilucoplan was selected as one of the first drugs to be tested in a multi-center ALS platform study sponsored by the Sean M Healey & AMG Center for ALS at Massachusetts General Hospital, Boston, MA. 

The safety and efficacy of zilucoplan have not been established and it is not currently approved for use in any indication by any regulatory authority worldwide.