Author: Rachael Hansford

PIVOT-HD Phase 2 Clinical Trial in Huntington’s Disease

PTC Therapeutics, Inc. announced on 31st March 2022 the initiation of the PIVOT-HD Phase 2 clinical trial evaluating PTC518 in people with Huntington’s disease (HD). PIVOT-HD is a global trial starting in the United States. PTC518 is an oral, small molecule splicing modifier that was specifically designed to selectively lower huntingtin mRNA and protein. There are no current treatments for the underlying cause of HD.

“We are excited to advance our Huntington’s disease programme,” said Stuart W. Peltz, CEO, PTC Therapeutics.

In the PIVOT-HD trial, we aim to confirm the dose-dependent lowering of huntingtin protein that was demonstrated in our Phase 1 clinical study and gain insight to biomarker data that could provide meaningful evidence of treatment effect.

Stuart W. Peltz, CEO, PTC Therapeutics.

The PIVOT-HD Phase 2 clinical trial is designed in two parts: an initial 12-week placebo-controlled phase focused on PTC518 pharmacology and pharmacodynamic effect, followed by a 9-month placebo-controlled phase focused on PTC518 biomarker effect.

About Huntington’s Disease

Huntington’s disease (HD) is a rare, inherited disease that causes the progressive degeneration of nerve cells in the brain that has broad impact on a person’s motor and functional capabilities, resulting in both movement disorders and cognitive loss. While HD can present at any age, it is most prevalent in people aged 30 to 50, and it affects approximately 45,000 people in the United States. HD is caused by a mutation in the huntingtin gene, which is responsible for creating huntingtin protein (HTT). As time progresses, the mutated huntingtin protein forms clumps in the brain cells, resulting in damaged cells and eventually cell death. HD has broad impact on a person’s motor and functional capabilities that results in both movement disorders cognitive loss. There are no treatments for the underlying cause of HD.

About PTC518

PTC518 is a small molecule splicing modifier that acts via a unique mechanism to promote the inclusion of a novel pseudoexon containing a premature termination codon, thus triggering HTT mRNA degradation and subsequent reduction in HTT protein levels. In a Phase 1 healthy volunteer study of PTC518 for Huntington’s disease, PTC518 demonstrated a dose-dependent lowering of HTT mRNA and protein to the targeted 30-50% reduction. In addition, PTC518 showed that it passes the blood brain barrier and has minimal efflux which is a key factor in the targeting the underlying cause of Huntington’s disease.

Innovative IQoro neuromuscular treatment device achieves NHS prescription status

IQoro, a treatment device for swallowing dysfunction and acid-reflux based diseases, will be added to the Drug Tariff from May 1st this year. It has previously been used by around 75,000 individuals that have self-purchased, and by SLTs and neuro-rehab clinicians in 40 or more NHS trusts.

IQoro is a simple hand-held device that is used for 30 seconds per session, three times per day.

It is inserted pre-dentally and pulled forwards to create a low pressure in the oral cavity and stimulate all the 148 muscles involved in a normal swallow. The sensory cranial nerves in the mouth are stimulated to send intense afferent signals to the brain stem, thus invoking a sensory motor reflex arc that exercises and strengthens muscles beyond the command of voluntary system. The training effect promotes neuroplasticity in those patients with neurological conditions.

CTE Biobank launches in Australia

A national biobank to collect information and test samples relating to repeated traumatic brain injuries often associated with sport was launched on 22nd March 2022 in Australia, with New Zealand announcing its intention to establish a similar clinic and biobank.

Chronic traumatic encephalopathy (CTE) is a form of dementia that results from repeated head injuries, and is found in a cross-section of the community, from those engaged in contact and combat sports, to members of the military and survivors of domestic violence.

The symptoms include mood disturbance with depression and anxiety, behavioural disturbance with tendency of impulsivity and anger, changes in personality, impaired judgement and confusion with cognitive decline, and the effect on the people diagnosed and their families is devastating.

Currently there are no clinical tests to identify CTE; the disease can only be conclusively diagnosed by examining the brain after death.

The two biobanks will work together and seek to gather quality data and stored samples over the long term that can be reanalysed in future.

Macquarie University CTE researcher and Australian CTE Biobank (ACB) Director Dr Rowena Mobbs says patients and their families are desperate for more support from the community, and the clubs for whom they played.

“Make no mistake, CTE shatters lives – not only those of patients, but their families,” she says.

“Our aim is to develop specific biomarkers for CTE, detecting and treating it early – and ultimately preventing it.

“We hope that this could mean blood testing for CTE within five years, just as we see on the horizon for Alzheimer’s and other forms of dementia.

“If we are to prevent it, there needs to be major national reform and greater cohesion across policy, clinical practice, and occupational health and safety for professional athletes at risk of repetitive brain injury and CTE.

“New patients are presenting with CTE every week, highlighting just how important this research is for athletes and the sports they represent.

“We are inviting support for the research programme from the NRL, AFL, and Rugby Australia, all of whose players have been demonstrated to have CTE in Australia. CTE can be compared to other occupational diseases.”

Professor Maurice Curtis is head of the Centre for Brain Research at the University of Auckland, CoDirector of the CBR Neurological Foundation Human Brain Bank, and one of NZ’s leading neuroscientists in the field of neurodegenerative research.

“Momentum has been building towards the formation of the NZ CTE Research Clinic Biobank alongside other researchers on both sides of the Tasman,” he says.

“The work done here in New Zealand will complement that of our Australian colleagues and other leading groups around the world in this critical area of brain research.

The Australian CTE Biobank held its launch at Macquarie University on Tuesday, 22 March, with the assistance of Biobank Ambassadors including former NRL player James McManus and his wife Eshia McManus, former rugby union player Michael and his wife Francis Lipman, and former boxer Jeff Fenech.


Call For Evidence Consultation Launched For ABI Strategy

A Call for Evidence has been launched for the recently agreed cross departmental Government strategy for Acquired Brain Injury (ABI).

The UK Acquired Brain Injury Forum (UKABIF) joined other brain injury charities and organisations and people with acquired brain injury at the House of Commons this week where the Minister of State for Care and Mental Health, Gillian Keegan and Chris Bryant MP announced the launch of the Call for Evidence.

Chris Bryant, Chair of the All-Party Parliamentary Group on Acquired Brain Injury, secured the ABI strategy at the end of 2021 and he and Gillian Keegan co-chair the ABI strategy programme board which first met earlier this year.

Chloe Hayward, Executive Director of UK Acquired Brain Injury Forum (UKABIF), said: “We are very pleased the government is giving acquired brain injury the attention it deserves with the ABI Strategy.

The call for evidence will help the panel to focus and prioritise their efforts, so we need people with lived experience of brain injury – whether, survivors, carers or professionals – to participate. This will ensure the panel has the best available information to develop their strategy.

Chloe Hayward

Minister of State for Care and Mental Health, Gillian Keegan, said: “It is absolutely essential people living with acquired brain injury get the best possible care and treatment and that we take steps to prevent these injuries wherever possible.

“Together the cross-government programme board and the call for evidence will allow us to deliver a strategy to address issues that matter most to those with acquired brain injuries and other neurological conditions.”

Chris Bryant MP said: “I’m delighted that the government is starting to pull together a cross government strategy on acquired brain injury.

“We need people to come forward with ideas and suggestions based on their experience of brain injury as practitioners, patients or family members so we can get this strategy right.

“I urge everyone to take part if they think they have an insight to offer.”

To complete the Call for Evidence survey go to

If you require further information, contact

WFNR Franz Gerstenbrand Award 2022

Brain Awareness Week – WFNR Franz Gerstenbrand Award 2022 now open for entries

As we mark Brain Awareness Week, I’m delighted to announce that the World Federation for Neurorehabilitation (WFNR) Franz Gerstenbrand Award 2022 is now open, and we particularly encourage entries from individuals under the age of 35 years who are clinicians, researchers or allied health professionals currently working in neurorehabilitation.       

Professor David Good, WFNR President

The Award, worth £3,000, is open to WFNR members and non-members and recognises and rewards a neurorehabilitation project that has benefitted patients.  It is named after Professor Franz Gerstenbrand in recognition of his contribution to neurorehabilitation.

Entries for the WFNR Award must demonstrate a difference to patient outcomes and can involve any aspect of neurorehabilitation, such as a patient or clinic management initiative, research project, best practice development or the use of a new technological development.   The work described must be completed and produced results or been published in the last 12 months.

The Award is for a travel bursary to a clinical/scientific conference, professional development course or research project.  The deadline for the Award is the 31st October 2022.

Further information and an application form.

Music Therapy strikes a chord with traumatised emergency workers ​

The Blue Light Symphony Orchestra (BLSO), in collaboration with Chroma, successfully completed a pilot project delivering group Music Therapy to emergency service workers to help them recover from traumatic experiences.

Working with Surrey and Sussex Police, Surrey and East Sussex Fire and Rescue and Southeast Coast Ambulance Service, the project, a UK first, delivered a bespoke music therapy program for emergency workers. It drew inspiration from the USA where music therapy is widely used to treat PTSD and trauma-related issues is in army veterans.

The BLSO was awarded £10,000 funding from the Coronavirus Community Support Fund, distributed by The National Lottery Community Fund, recognising that Emergency Workers have been exposed to increased levels of trauma while responding to the Covid-19 pandemic.

Over 12 weeks, using a combination of psychodynamic music therapy, neurologic music therapy and dialectical behaviour techniques, emergency service personnel were able to experience, reflect, learn, and then transfer coping strategies into everyday life.

The group incorporated free improvisation, the learning of simple drumming techniques, blended with health and wellness education and interactive improvised music-making. Throughout the programme, the sense of camaraderie increased, alongside the enjoyment and playfulness that developed.

Seb Valentine, founder of the BLSO and serving Detective Sergeant with Surrey Police said: “We are extremely happy with the success of this project, bringing music therapy to emergency workers who develop mental health issues relating to the trauma they experience daily.

“Positive feedback from participants has meant we are planning to run another project in the Surrey Sussex area. I hope that when my colleagues see how successful this pilot was, it will reduce scepticism and encourage more to take advantage and benefit from the healing power of music.  

I would love to hear from any other emergency services organisations in England or Wales that would like to host a Music Therapy project. We are actively seeking funding opportunities and know that music therapy can help support the mental wellbeing of emergency service workers, so please do get in touch.

Seb Valentine, founder of the BLSO and serving Detective Sergeant with Surrey Police

Chroma will also be expanding the support, with the West Midlands Ambulance Service starting a similar project in May 2022.

Daniel Thomas, managing director at Chroma, added: “This was a wonderful project to be a part of. By the end of the 12 weeks, 71% of the participants’ recorded reduced levels of distress compared to their CORE-OM scores when they signed up for the program. “As a result of this success, we are pleased to be able to expand the project to offer more emergency service personnel vital support to support their physical, emotional and mental wellbeing.”

Click here for further information and a documentary video:
For more information regarding hosting a music therapy project, contact: Seb Valentine on 0777 337 2575 or via email

Copaxone® for use in breastfeeding mothers with RMS

  • Change to COPAXONE® (Glatiramer Acetate (GA)) Summary of Product Characteristics (SmPC) in breastfeeding is relevant for the Multiple Sclerosis (“MS”) community and provides information for neurologists and patients of the positive benefit/risk balance of use in breastfeeding
  • Relapsing Multiple Sclerosis (RMS) is 2-3 times more likely to affect womeni, with diagnosis most common during childbearing years
  • The SmPC update follows the recent COBRA Real World Evidence study of infants breastfed by mothers with MS undergoing GA treatment

Teva Pharmaceuticals Europe BV confirmed on 10th February 2022 that the SmPC for COPAXONE® (Glatiramer Acetate (GA) injection) 20mg/mL and 40mg/mL, indicated for the treatment of relapsing forms of multiple sclerosis (RMS) in Europe, has been updated. The product is now approved by EU health authorities for use in breastfeeding. The label update follows the review of clinical and non-clinical evidence, including latest data from the COBRA real world evidence study that investigated safety outcomes in infants breastfed by mothers with MS undergoing GA treatment during the first 18 months of life.ii

COBRA, the largest standardised analysis of data from the National German Multiple Sclerosis and Pregnancy Registry, assessed safety outcomes in a total of 120 infants including 60 of them breastfed by mothers under GA. It concluded that no evidence was found to suggest that infants were adversely affected by maternal exposure to GA during breastfeeding. This was measured by number of hospitalisations, antibiotic treatments, developmental delays and growth parameters in the first 18 months of life.iii The label update provides information for neurologists and other healthcare professionals treating MS patients of GA’s positive benefit/risk balance in breastfeeding.

Professor Kerstin Hellwig, Principal Investigator of COBRA RWE Study, Department of Neurology, Katholisches Klinikum Bochum, Germany says: “The benefits of breastfeeding for both mothers and their offspring are clinically meaningful and well-documented, but historically there has been limited clinical safety data for infants breastfed by mothers undergoing MS treatment. It is now believed breastfeeding could be protective for mothers with MS. The COBRA study results support mothers with MS in their choice to breastfeed without having to preclude MS treatment. This is an important contribution to current significant medical need.”

There are almost half a million women in Europe living with MS1; it is most common during childbearing age and about half of mothers with MS start their families after diagnosis. The pregnancy rate in MS is constantly increasingiv and recent research shows pregnancy does not worsen the disease progression (which traditionally has been a concern for patients).v

However, studies have found an increase in relapse incidence after child birth, in the postpartum periodvi, so MS treatment may need to be resumed. The majority of MS therapies’ labels advise against breastfeeding, so mothers are often faced with a choice to breastfeed their babies or restart their treatment. Given that according to a U.S. study approximately half of women with MS want to breastfeed vii, the safety of medications used to treat MS while breastfeeding is of concern to mothers. A further treatment option that can be used during breastfeeding may help address a significant medical need for mothers with MS.

Danilo Lembo M.D. VP Medical Europe, Teva Pharmaceuticals comments: “Our mission at Teva is to improve the lives of patients. This includes addressing gender inequalities in healthcare and understanding the unique challenges that women face during pregnancy and breastfeeding. The COPAXONE® label change provides breastfeeding patients with MS the choice to breastfeed while on MS treatment.”


i Pugliatti M et al. The epidemiology of multiple sclerosis in Europe. Eur J Neurol 2006;13(7):700-22.

ii Ciplea A, Kurzeja A, Thiel S, Haben S, Alexander J, Adamus E, Hellwig K. Safety analysis of offspring breastfed by mothers on glatiramer acetate therapy for relapsing multiple sclerosis. Eur.J.Neurol. 2021; 28(SUPPL 1): 201-202. 10.1111/ene.14973

iii Teva Pharmaceutical Industries Limited, 2021. New Safety Data on treatment with COPAXONE® (glatiramer acetate) of Breastfeeding Mothers who Live with Relapsing Multiple Sclerosis: COBRA study presented at the 7th Congress of the European Academy of Neurology (EAN). [online] Available at: [Accessed 1 February 2022].

iv Maria K. Houtchens, MD, Natalie C. Edwards, MSc, Gary Schneider, ScD, Kevin Stern, BA and Amy L. Phillips, Pregnancy rates and outcomes in women with and without MS in the United States, Neurology® 2018;91:e1559-e1569. doi:10.1212/WNL.0000000000006384

v Langer-Gould AM, Multiple Sclerosis & other CNS Inflammatory Diseases p. 773-792 June 2019, Vol.25,No.3,doi: 10.1212/CON.0000000000000745

vi Manson J. European Women With Multiple Sclerosis Feel Unprepared and Uneducated About Family Planning and Their Ability to Have Children – How Do We Improve Patient Education? European Neurological Review. 2018;13(1):21–4 DOI:

vii Lorifice L, et al. Neurol Ther 2021; doi: 10.1007/s40120-021-00297-6. Online ahead of print.

UCB announces positive data in myasthenia gravis with zilucoplan phase 3 study results

  • Positive topline results show the Phase 3 RAISE (NCT04115293) zilucoplan trial met primary and all key secondary endpoints in adults with generalised myasthenia gravis
  • The results show a favourable safety profile and good tolerability  
  • UCB plans to proceed with zilucoplan regulatory submissions later this year 
  • Results follow recent positive topline data from the Phase 3 MycarinG study investigating rozanolixizumab, a monoclonal antibody also being developed by UCB in the same indication
  • These results are the latest in a series of positive phase 3 data announcements by the company across its product pipeline

February 04, 2022 – 07:00 CET: UCB, a global biopharmaceutical company, announced positive topline results from the RAISE (NCT04115293) trial1 evaluating its investigational treatment zilucoplan, a self-administered, subcutaneous (SC) peptide inhibitor of complement component 5 (C5 inhibitor), versus placebo in adults with generalised myasthenia gravis (gMG).

The primary endpoint of the trial was met; a clinically meaningful and statistically significant improvement from baseline in Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at Week 12 was observed for the zilucoplan treatment group vs placebo.

All key secondary endpoints were also met, including statistically significant improvements from baseline in Quantitative Myasthenia Gravis (QMG) score, Myasthenia Gravis Composite (MGC) score and MG-QoL15r score at Week 12 for the zilucoplan treatment group vs placebo. 

The results show zilucoplan was well-tolerated and no major unexpected safety findings were identified compared to earlier zilucoplan studies. The incidence of serious treatment emergent adverse events (TEAEs) in the zilucoplan and placebo treatment arms was similar.

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. 

Based on these results, UCB plans to progress with regulatory filings for zilucoplan in gMG in the United States (US), European Union (EU) and Japan, beginning later this year.  

gMG patients can experience varying and debilitating symptoms that impact their everyday lives in unique ways,” said James F. Howard, MD, Distinguished Professor of Neuromuscular Disease, Chief, Neuromuscular Disorders Section, University of North Carolina School of Medicine and lead investigator in the RAISE trial. “These exciting results give us additional reason to believe that zilucoplan can offer an important step forward in addressing the unmet needs of people living with gMG. As we strive to improve the management of this complex and unpredictable disease, any new medicines will be welcomed by physicians to help us realize our goal of offering effective and flexible treatment approaches in gMG which are tailored to the needs of individual patients.”

These findings from RAISE build on the positive results from the Phase 3 MycarinG study evaluating UCB’s investigational treatment rozanolixizumab, an SC-infused monoclonal antibody targeting the neonatal Fc receptor (FcRn) which also met its primary and secondary endpoints with statistical significance in gMG.2

UCB is currently the only company investigating two potential treatments with different mechanisms of action in gMG. Detailed results from both Phase 3 trials will be presented at forthcoming medical meetings in 2022.

While there has been recent progress in the treatment of MG, there is still a significant unmet need for new, effective treatment options that address the unpredictable, fluctuating symptoms of MG – some of which require urgent treatment or hospitalisation – to improve patient outcomes and quality of life. New research into additional treatment options will be welcomed by the global MG community.

Raquel Pardo, Spanish Myasthenia Association (AMES), Spain

Iris Loew-Friedrich, Executive Vice-President and Chief Medical Officer added: “This is the latest in a series of positive Phase 3 data announcements across UCB’s product pipeline validating our patient value strategy and laying foundations for future sustainable growth. Today’s results represent another significant milestone in UCB’s efforts to bring transformational outcomes to those living with myasthenia gravis. Positive results for zilucoplan and rozanolixizumab – each with a different mechanism of action – bring us one step closer to achieving our ambition of delivering choice and flexibility for a broad range of patients and physicians at each step of their treatment journey, addressing significant unmet needs and offering unique patient value. We thank the MG community for their continued insights, partnership and participation in this study.”

About Generalized Myasthenia Gravis (gMG)

Myasthenia gravis is a rare disease impacting almost 200,000 patients in the U.S., EU and Japan. ,  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 life- threatening weakness of the muscles of respiration.5-8

gMG is a 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 ability of the nerves to stimulate the muscle and results in a weaker contraction. gMG can occur at any age and in any race, although previous studies have shown that women are more often impacted than men.9 Complement activation, a key mediator of antibody function, is recognized as an important driver of pathology in gMG. 

About the zilucoplan RAISE study10,11 

The RAISE study (Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis (NCT04115293)) is a multi-center, Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of zilucoplan in adult patients with gMG. 

Patients were randomised in a 1:1 ratio to receive daily subcutaneous (SC) doses of zilucoplan or placebo for 12 weeks. The study was designed to determine if complete complement inhibition can bring clinical benefit to people with gMG and if complement inhibition was effective across a broad spectrum of patients with acetylcholine receptor antibody positive (AChR Ab+) MG regardless of disease duration, prior treatment or response to previous therapies. 

The primary endpoint for RAISE study is change from baseline at Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score, an eight-item patient-reported scale developed to assess MG symptoms and their effects on daily activities. 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; time to rescue therapy; the percentage with minimum symptom expression (MSE) (defined as MG-ADL of 0 or 1), the percentage with a ≥3-point reduction in MG-ADL and the percentage with a ≥5-point reduction in QMG, all measured at Week 12.

For more information about the trial visit

About the rozanolixizumab MycarinG study12 

The MycarinG study (NCT03971422) is a completed 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. 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). 

A preliminary analysis of results shows the trial met its primary endpoint, demonstrating a statistically significant and clinically meaningful change from baseline in the 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.13  

For more information about the trial, visit

About Zilucoplan11,14

Zilucoplan targets complement component 5 (C5), a component of the terminal complement activation pathway, and binds to C5 with high affinity and specificity. This prevents its cleavage by C5 convertases into the complement components C5a and C5b. In addition, zilucoplan is understood to bind to the domain of C5 that corresponds to C5b and thereby block binding of C5b to complement component C6. 

Inhibition of C5 cleavage prevents the downstream assembly and activity of membrane attack complex (MAC). This dual mechanism of action of zilucoplan has the potential to prevent activation of the terminal complement pathway and downstream assembly and activity of MAC that can damage and destroy the postsynaptic membrane, disrupt ionic channel conductance and impair neuromuscular transmission. 

Zilucoplan is being investigated in the RAISE study, a multi-center, Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of zilucoplan in subjects with gMG.
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.

About Rozanolixizumab
Rozanolixizumab is a subcutaneously infused 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.15,16 

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 autoimmune 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.


  1. Data on file. UCB. February 2022.
  2. UCB Press Release, December 10. 2021 Accessed February 2022
  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. Myasthenia Gravis Foundation of America. Clinical Overview of MG. Accessed November 2021.
  10. Clinical ‘Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis (RAISE)’: Accessed November 2021.
  11. Howard JF, Jr., et al. JAMA Neurol 2020;77:582–92.
  12. Clinical ‘A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis’: Accessed February 2022.
  13. Data on file. UCB. December 2021
  14. Ricardo A, et al. Blood 2015;126:939.
  15. Kiessling P, et al. The FcRn inhibitor rozanolixizumab reduces human serum IgG concentration: A randomized phase 1 study. Sci Transl Med. 2017;9(414).eaan1208
  16. 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.

Positive phase III data for Neurocrine’s Ingrezza in Huntington’s disease

Neurocrine Biosciences has recently announced that its pipeline asset, Ingrezza (valbenazine), achieved the primary endpoint of reducing the symptoms of chorea (involuntary muscle movements) associated with Huntington’s disease (HD) in the Phase III clinical trial, KINECT-HD (NCT04102579). This will propel Ingrezza into a dominant position in the HD market, due to its convenient once-daily dosing regimen and improved safety profile, according to GlobalData, a data and analytics company.

GlobalData’s report, ‘GlobalData (2021) Huntington’s Disease: Opportunity Assessment and Forecast to 2030’, reveals that given Ingrezza’s competitive advantage and the sparseness of the US HD therapeutics market, it is set to generate strong sales of $326m for HD in the US through 2030*.

Compared to available competitors within the same therapeutic class, Ingrezza requires less-frequent dosing and does not pose the risk of increasing depression and suicidality. This is a key advantage, as the development of psychiatric and cognitive symptoms is a common comorbidity in HD patients.”

Sarah Elsayed, Neurology Analyst at GlobalData

Neurocrine plans to submit a supplemental new drug application in HD chorea, meaning that the drug could be available for HD patients in the US by Q1 2023. Key opinion leaders (KOLs) previously interviewed by GlobalData highlighted that one of the most pressing unmet needs in HD is for improved symptomatic treatments that can demonstrate superior clinical efficacy and/or safety to the current standard of care.

Chorea occurs in over 80% of patients with adult-onset HD and is not effectively managed by current treatment options. Thus, KOLs believed that Ingrezza will be a welcome addition to the HD armamentarium and will fulfill a key unmet need.”

Sarah Elsayed, Neurology Analyst at GlobalData

Currently, there are only two drugs approved in the US for the treatment of chorea associated with HD: Bausch’s Xenazine (tetrabenazine) and Teva’s Austedo. Additionally, several generic versions exist for tetrabenazine in the US, offering comparable efficacy at lower costs.

Elsayed continues: “That said, tetrabenazine generics may not be preferred by many patients as they require dosing three times per day and have a boxed warning on the label regarding the increased risk of suicidality. These issues directly affect patient compliance and subsequently limit the utility of those treatments. Ingrezza is therefore more likely to compete for new patients against Teva’s Austedo, which has been widely adopted by physicians since its launch in 2017, owing to the convenience of its twice-daily dosing. However, like tetrabenazine, Austedo also has a boxed a warning regarding the risks of depression and suicidality.”

GlobalData believes that the clinical advantages Ingrezza has shown so far in trials will allow it to move to the front lines of treatment for HD chorea and gain significant market share upon launch in the US.”

Sarah Elsayed, Neurology Analyst at GlobalData