Author: Rachael Hansford

Trial shows benefits in patients with Duchenne Muscular Dystrophy

Topline Data from Phase 3 Trial shows beneficial effect of Givinostat 

22nd June, 2022: Italfarmaco Group announced positive topline data from its completed Phase 3 EPIDYS trial with Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor, in boys with Duchenne Muscular Dystrophy (DMD). The primary objective of the study was to evaluate the effects of Givinostat on slowing disease progression in ambulant DMD boys aged 6 years and above on chronic steroids. The study compared Givinostat to placebo and met the primary endpoint (change from baseline in the time to climb 4 stairs) following 18 months of treatment in the target population1 with key secondary endpoints consistent with the functional primary endpoint. Givinostat continued to demonstrate a tolerability profile in line with previous studies. The topline data were presented by Italfarmaco Group’s Chief Medical Officer, Dr. Paolo Bettica on June 25, 2022, at the hybrid Parent Project Muscular Dystrophy (PPMD) Annual Conference.

In October 2020, the US Food and Drug Administration (FDA) granted a Rare Pediatric Disease designation to Givinostat for the treatment of DMD. Earlier, the company also received an Orphan Drug Designation from the FDA and EMA, and a Fast Track designation from the FDA. Based on the study results, which show that the addition of Givinostat to steroid treatment leads to clinical benefits for the individuals in the study, the company plans to meet with US and EU regulators to discuss the potential for marketing application submission and seek input on a future submission of the application for regulatory approval. The company intends to submit the complete results of the EPIDYS study for publication in a peer-reviewed journal in due course.

The EPIDYS Phase 3 clinical trial is a randomised, double-blind, placebo-controlled, multicentre study evaluating the efficacy and safety of Givinostat in individuals with DMD (Cinicaltrials.gov: NCT02851797). The trial enrolled 179 male ambulant individuals with a mean age of 9 years and on stable steroids for at least 6 months. Boys were randomised 2:1 and treated chronically with an oral suspension of Givinostat or placebo for a period of 18 months. Of these, 120 boys formed the target population1.

Overview of Clinical Results

Primary Endpoint: 

The mean change from baseline to climb 4 stairs was selected as the primary endpoint to assess the efficacy of Givinostat compared to placebo. The results demonstrated a slower decline to perform this functional task in the Givinostat-treated group (difference vs Placebo of 1.78 seconds, p=0.0345).

Key Secondary Endpoints: 

A variety of secondary endpoints were analysed that showed results consistent with the functional primary endpoint. These included functional tests such as the North Star Ambulatory Assessment (NSAA) and the time to rise (TTR) test along with muscle strength analyses. Fat infiltration in the vastus lateralis (VL) muscle of the thigh is a characteristic of disease progression in DMD patients and was measured using a non-invasive objective imaging method called Magnetic Resonance Spectroscopy (MRS) to assess the efficacy of Givinostat. The data indicated that treatment with Givinostat delayed fat infiltration by approximately 30% (difference vs Placebo, -2.9%, nominal p=0.035). Additional exploratory endpoints were also analysed in the clinical study. These results further support the potential of Givinostat in providing a clinical benefit for boys with DMD.

Safety and Tolerability: 

The majority of Adverse Events (AE) were mild to moderate in severity (95%). Three (2.5%) boys treated with Givinostat withdrew from the trial because of an AE. Similar to what had been previously observed, the AE occurring in at least 1/10 subjects were diarrhea, abdominal pain, thrombocytopenia, hypertriglyceridemia, platelet decrease and triglyceride increase. Givinostat tolerability was managed with appropriate monitoring and dose adjustments. No other safety concerns were observed.

These results show Givinostat’s beneficial effect in DMD boys providing evidence of its ability to slow down disease progression.

Prof. Eugenio Mercuri, Professor of Paediatric Neurology at the Catholic University, Rome, Italy

Prolonged release capsules for pain

Neuraxpharm launches Tapimio® (tapentadol) prolonged release capsules in the UK

Neuraxpharm announced on June 30th 2022 the launch of Tapimio® (tapentadol) for the management of severe chronic pain in adults, which can be adequately managed only with opioid analgesics. Tapimio® is the first and only prolonged release capsule formulation of tapentadol to be made available in the UK.1,4,5

Tapentadol is a centrally acting analgesic which has a dual mode of action to produce acute and chronic pain relief.6 As Tapimio® is a capsule it can be taken whole or opened and the contents sprinkled on cold, soft food.1 This formulation gives adult patients with chronic severe pain which can be adequately managed only with opioid analgesics and who cannot swallow tablets or prefer capsules an alternative dosage form.

Tapimio® has demonstrated equivalent efficacy and safety to tapentadol medicines which are available in tablet form.2 Tapentadol showed superior quality of life results compared to oxycodone controlled release – a commonly used opioid.7 Tapimio® also confers a minimum cost saving of 15% to the NHS compared to existing Palexia (tapentadol) products available in the UK.3

One in four (26%) UK adults are living with chronic pain, and 24% of those are currently taking opioid painkillers.8

This is the first time tapentadol has been made available in capsule form and we are delighted to finally be bringing it to the UK market. It’s a critical time for the NHS when it comes to saving money, so we’re glad to also be able to offer this cost benefit to the NHS.

Craig Bowen, General Manager, Neuraxpharm UK

References

  1. Tapimio 150mg prolonged-release capsules. Summary of Product Characteristics. Available from: https://www.medicines.org.uk/emc/product/13719/smpc#gref. Accessed: June 2022  
  2. Data on File. Tapentadol Bioavailability REF CSR 021B19-0522 (M). 
  3. BNF. Available from: https://bnf.nice.org.uk/medicinal-forms/tapentadol.html. Accessed June 2022.
  4. Ationdo SR 100mg prolonged release tablets. Summary of Product Characteristics. Available from: https://www.medicines.org.uk/emc/product/12658/smpc#gref. Accessed: June 2022
  5. Palexia 50mg film-coated tablets. Summary of Product Characteristics. Available from: https://www.medicines.org.uk/emc/product/5159/smpc#gref. Accessed: June 2022
  6. Zajączkowska, R., et al. Pharmacol Rep 2018; 70(4), pp.812-820. Available from: https://link.springer.com/article/10.1016/j.pharep.2018.01.005
  7. Lange, B., et al. Efficacy and safety of tapentadol prolonged release for chronic osteoarthritis pain and low back pain. Adv Ther 2010; 27(6), pp.381–399. Available from: https://link.springer.com/article/10.1007/s12325-010-0036-3.
  8. IPSOS Chronic Pain Survey. Carried out on behalf of the BBC. 9-14 March 2022. Available from: https://www.ipsos.com/sites/default/files/ct/news/documents/2022-05/chronic-pain-survey-tables-ipsos-research-for-bbc-may-2022.pdf Accessed: June 2022

Preserving cognitive function in RRMS

Bristol Myers Squibb presents new data showing effect of early Zeposia (ozanimod) treatment in improving and preserving cognitive function in people with Relapsing Multiple Sclerosis.

On June 24th, Bristol Myers Squibb presented results of the DAYBREAK open-label extension trial at the 8th European Academy of Neurology Congress in Vienna, Austria.

New post-hoc analyses from the Zeposia (ozanimod) Phase 3 DAYBREAK open-label extension (OLE) and Phase 3 SUNBEAM trials, showed early Zeposia use demonstrated cognitive benefits in people with relapsing multiple sclerosis (MS), with the greatest effect seen in people with high thalamic volume (TV), supporting an association between preserved brain volume (BV) and improved long-term cognitive outcomes.

Multiple sclerosis can lead to significant, irreversible brain volume loss and decreased cognition if not treated quickly upon diagnosis. These new analyses show the potential of early treatment with Zeposia to help stabilize and even improve cognition in people with multiple sclerosis with high brain volume, which is important for doctors and people with multiple sclerosi

John DeLuca, PhD, senior vice president for research and training, Kessler Foundation, and professor, Department of Physical Medicine & Rehabilitation and of Neurology, Rutgers New Jersey Medical School

In these new exploratory analyses, Zeposia treatment showed improved or preserved cognitive function in a majority of patients, with the greatest improvement seen when used early in the disease when TV remains high, supporting a positive association between preserved BV and long-term cognitive performance. Zeposia was well tolerated with more than 80% of people who started the Phase 3 SUNBEAM trial (N=399 at baseline) remaining on continuous therapy through 48 months of the Phase 3 DAYBREAK OLE study (N=326).

Findings from the new research showed that people with high versus low BV, particularly TV, had higher cognitive performance, as assessed by the symbol digit modalities test (SDMT) score, at baseline. This trend remained stable or improved over 4-5 years of Zeposia treatment, leading to improved or preserved cognitive function in almost 80% of people with high TV (SDMT improved: 45.1%; SDMT preserved: 34.4%) and approximately 66% of people with low BV (SDMT improved: 35.6%; SDMT preserved: 30.7%) at Month 48 of the Phase 3 DAYBREAK OLE study.

Aubagio study: no disease activity after 2 years

More than half (58% )of the people with multiple sclerosis (MS) treated with Aubagio (teriflunomide) showed no evidence of disease activity after two years on treatment, according to an Italian study.

The study, “Evolution of teriflunomide use in multiple sclerosis: A real-world experience,” was published in the Journal of the Neurological Sciences.

Aubagio is an oral disease-modifying therapy that has been approved since 2013 in the EU to treat adults with relapsing-remitting MS. It is marketed by Sanofi, which was not involved in the study.

Scientists at the University of Cagliari analysed patients who started treatment with Aubagio at their clinic between 2016 and 2020. The analysis included data for 319 patients — about two-thirds were women, and the average age was late 40s.

Most of the patients (79%) had switched to Aubagio from a different MS treatment, while for the remaining 21% Aubagio was their first MS treatment.

By the end of 2020, 240 (75.2%) of the patients were still being treated with Aubagio after a median follow-up time of nearly four years (45.7 months). Among those who stopped taking Aubagio, the median treatment duration was just under two years, and just over a third (35.4%) cited a lack of effectiveness as the reason for stopping.

Efficacy data was available for 204 patients who had taken Aubagio for at least two years. Of these, more than half (58.8%) showed no evidence of disease activity (NEDA) over two years, meaning they had no relapses, no worsening disability, and no signs of new inflammatory damage on MRI scans. NEDA was also achieved by more than half of the patients (56.8%) who remained on the treatment for three years.

The likelihood of NEDA at two years was highest for those who were younger and those who had milder disability scores and fewer relapses before starting Aubagio.

Early is better than late treatment, but late is better than never

Study authors

The researchers noted that the proportion of treatment-naïve patients increased from about 10% in 2016 to roughly 30% in 2020.

The proportion of patients who were women under age 45 — so-called “childbearing age” — also increased, from about 20% in 2016 to about 30% in 2020. Aubagio is not recommended for use in pregnancy. The researchers noted that 82.3% of the women under 45 in this study already had children before starting Aubagio.

News source: Multiplesclerosistoday.com

Novel Early Intervention Study for Stroke Patients

MindMaze and The University of Auckland partner on novel early intervention study for stroke patients, expanding global breadth of research supporting the company’s brain tech solutions

MindMaze, (digital therapeutics (DTx) for neurological recovery and care), in partnership with The University of Auckland, has announced a new interventional study that will aim to show the merits of early, immersive, and intensive intervention to recover movement for patients who have experienced a stroke. The Phase 2 trial, Enhancing Spontaneous Recovery After Stroke Study (ESPRESSo, ACTRN12620000871943), will evaluate the effect of the use of MindPod in a three-week programme of high-intensity, high-dose exploratory arm and hand movements initiated within two weeks of stroke on upper limb motor capacity.

ESPRESSo is actively recruiting, orienting the medical community towards earlier intervention by demonstrating the marked benefits it can have following a stroke, both immediate and long-term.

The MindPod, a novel, immersive neuro-animation experience designed to promote the recovery of motor skill and cognitive function following a stroke or other neurological injury or disease, was chosen for ESPRESSo as it takes a unique approach to brain repair: it is a holistic multiscale intervention that simultaneously delivers a cognitive challenge, motor skill training and a cardiovascular workout. This training can be expected to lead to more generalised effects that are both cognitive and physical.

“Research shows that an injured brain can get better, especially if the recovery work starts early.

John Krakauer, M.A., M.D., Professor of Neurology, Johns Hopkins University School of Medicine

“MindPod was designed with this knowledge in mind and the result is a high-dose, high-intensity solution that patients actually enjoy using,” said John Krakauer, M.A., M.D., Professor of Neurology, Johns Hopkins University School of Medicine and Chief Medical Advisor to MindMaze. “This immersive, reinforcing environment is key for motivation and therapy adherence, each of the utmost importance in particular in early recovery settings when morale is most challenged.”

In addition, the ESPRESSo Trial is utilising a pioneering predictive tool which allows accurate predictions about recovery about three-month outcomes of upper limb function by combining clinical and neurophysiological assessments. Used in New Zealand hospitals, the tool is being validated in the United States, and is a useful and unprecedented reference for physicians when guiding their patients through what can be expected during the recovery process and eventual outcomes. In the difficult and uncertain time following an event like a stroke, the tool can provide more certainty and realistic possibilities for patients and their physicians to work towards.

“What we are evaluating in the ESPRESSo Trial is ambitious, multifaceted, and novel,” said Winston Byblow, PhD, Director of the Movement Neuroscience Laboratory at The University of Auckland and Principal Investigator on the ESPRESSo Trial. “Our aim is to show that there is a real, sound biological reason for urgency of intervention and that it directly correlates with improved outcomes for patients who have experienced a stroke. With the predictive tool, we’re meeting each patient where they are, getting the goals right early and working with state of the art, immersive technology to meet or exceed their predicted outcome.”

DTx protocols enabled by the MindPod are the subject of many ongoing trials worldwide for indications such as stroke, Parkinson’s disease, dementia and healthy ageing, underscoring its potential for more general use in neurological disease, injury and ageing.

Trial failure for Jazz Pharmaceuticals’ cannabis-derived drug in MS

Jazz Pharmaceuticals nabiximols oromucosal spray did not meet the primary endpoint in a phase 3 trial, failing in its aim of improving Lower Limb Muscle Tone-6 (LLMT-6) between baseline and Day 21, as measured by the Modified Ashworth Scale (MAS).

The spray is formulated from extracts of the cannabis sativa plant and contains tetrahydrocannabinol (THC) and cannabidiol (CBD). It has been approved in 29 countries for adults with MS spasticity who have not responded adequately to other anti-spasticity treatments.

Jazz said it will continue to evaluate the spray for use in MS patients.

With 68 participants, the Release MSS1 trial was the first and smallest of three studies Jazz is conducting with the spray in MS. Release MSS3 is examining the improvement of muscle spasms in 446 MS patients over a 12-week period. Release MSS5 is investigating velocity-dependent lower-limb muscle tone over a 21-day period in 190 MS patients.

We look forward to additional data from two other ongoing trials that have the potential to support a US FDA new drug application submission

Rob Iannone, M.D., EVP, global head of research and development at Jazz Pharmaceuticals

Promising interim results for Ajovy

PEARL STUDY Interim data presented at the European Academy of Neurology 2022 shows that 54.7% of patients in the study had their monthly-migraine-days reduced by 50% or more over the six-month period from the start of treatment.

25th June 2022: Teva Pharmaceuticals Europe B.V. announced promising interim results from its Pan-European Real World study (PEARL), presented for the first time at the European Academy of Neurology (EAN) Congress in Vienna, Austria.

The two-year Pan-European Real World (PEARL) prospective, observational study of AJOVY® (fremanezumab), looks at its effectiveness in patients with chronic migraine or episodic migraine, and is an ongoing study sponsored by Teva Pharmaceuticals Europe BV.[i]  These findings further offer insight into the treatment of migraine in real-world clinical practice.

The interim findings were presented by Faisal Mohammad Amin, Associate Professor of Neurology at the University of Copenhagen, Denmark.  Out of the total planned 1100 patients in PEARL, 389 patients are included in the interim analysis presented.  These findings show that 54.7% of patients in the study had their monthly-migraine-days reduced by 50% or more, over the six-month period from the start of treatment.  Additionally improvements could be seen in migraine-related disability in the six-month period after the first dose.    

The study is particularly relevant to clinicians due its patient cohort, who come from 11 countries and approximately 100 study centres.1 This patient group illustrates both diverse populations and also a range of reimbursement settings which are important for treatment access in Europe.  

The study will continue to capture data on effectiveness, and safety of fremanezumab, as well as the reason for and the outcomes of stopping and re-starting treatments. 

Commenting on the findings, Professor Messoud Ashina, PEARL Coordinating Investigator from the Danish Headache Center and Department of Neurology in Rigshospitalet Glostrup, Denmark, said:

Patients with severe migraine could benefit from preventive therapy but usage of those treatments is far from optimal. These interim findings provide real-world evidence of how the burden of migraine can be reduced when the patient has access to monoclonal antibodies like fremanezumab – something neurologists around the world are already seeing in patients who did not respond to previous preventive treatments.

Dr. Danilo Lembo, Vice President and Head of EU Medical Affairs at Teva, said: “The PEARL study is encouraging as these findings confirm that preventive treatment of chronic and episodic migraine is appropriate with fremanezumab in a real world setting. Our commitment to real-world evidence studies helps clinicians to better understand the lived experience of people with migraine, support the evolution of best clinical practice and demonstrate the impact of migraine and what preventive treatments can achieve. ”

The real life experience of European patients being able to access preventive treatment is bleak. Beyond our own data and research, a recent study from the European Migraine and Headache Alliance showed that 40% of patients needed more than five years to access migraine treatments.[ii] We need real structural change to come from healthcare systems if we are to ultimately reduce the burden of migraine.

Dr. Danilo Lembo, Vice President and Head of EU Medical Affairs at Teva

Chronic migraine is defined as a headache occurring on 15 or more days per month for more than three months, which, on at least 8 days per month, has the features of migraine headache;[iii] episodic migraine is defined as having headaches on fewer than 15 days a month.[iv] The significant personal impact and burden of this severity of migraine has been shown in many studies including Teva’s own survey of 12,545 adults with migraine – ‘Beyond Migraine’, in which 45% said migraine impacts their ability to be a good partner and 42% to be a good parent, while 49% said migraine diminished their ability in the workplace. In terms of broader social impact, 46% reported hiding migraine from others.[v]

41 million people in Europe live with migraine[vi] and the disease is three times more common in women.[vii]  Migraine is the second leading cause of disability in the world and the first among young women.[viii] Migraine often begins at puberty and mostly affects people aged between 35 and 45 years.[ix] It strikes during people’s most productive years (late teens to 50s).[x]

Further reading

A publication in the journal Headache presented data that shows, for AJOVY®, there is no reduction in efficacy over a dosing period. This means that the efficacy of AJOVY® was similar at the end of the dosing period compared to the start of the same dosing period, immediately following treatment admininistration. Patients using AJOVY® therefore experience the benefits of treatment throughout the entire dosing period. See Blumenfeld AM, Stevanovic DM, Ortega M et al. No “wearing-off effect” seen in quarterly or monthly dosing of fremanezumab: Subanalysis of a randomized longterm study. Headache 2020; 60: 2431–2443.


References:

[i]Ashina, M. et al, PEARL study protocol. Pain management, 11(6), 647–654. (v0.1) – The two year Pan-European Real

World (PEARL) prospective, observational study of AJOVY® (fremanezumab)

2. KPMG, prepared for the European Migraine and Headache Alliance (EMHA). “Access to Care” project: final assessment. July 2021. [online] Available at: https://www.emhalliance.org/wp-content/uploads/ATC-EMHA-Dossier.pdf

[iii] Headache Classification Committee of the International Headache Society (IHS) The International Classification of Headache Disorders, 3rd edition. (2018). Cephalalgia, 38(1), 1–211. https://doi.org/10.1177/0333102417738202

[iv] Lipton, R. B., & Silberstein, S. D. (2015). Episodic and chronic migraine headache: breaking down barriers to optimal treatment and prevention. Headache, 55 Suppl 2, 103–126. https://doi.org/10.1111/head.12505_2

[v] Beyond Migraine – The Real You. Survey conducted 2020. Teva Pharmaceuticals. Data on file.

[vi] Stovner, L. J., Andrée, C., & Eurolight Steering Committee (2008). Impact of headache in Europe: a review for the Eurolight project. The journal of headache and pain, 9(3), 139–146. https://doi.org/10.1007/s10194-008-0038-6

[vii] Al-Hassany L, Haas J, Piccininni M, Kurth T, Maassen Van Den Brink A and Rohmann J.L. (2020) Giving Researchers a Headache – Sex and Gender Differences in Migraine. Front. Neurol. 11:549038. https://doi:10.3389/fneur.2020.549038

[viii] Steiner, T.J., Stovner, L.J., Jensen, R. et al. Migraine remains second among the world’s causes of disability, and first among young women: findings from GBD2019. J Headache Pain 21, 137 (2020). https://doi.org/10.1186/s10194-020-01208-0

[ix] Who.int. 2016. Headache disorders. [online] Available at: https://www.who.int/news-room/fact-sheets/detail/headache-disorders

[x] EMHA. 2021. Migraine in the EU – Bringing women out of the shadows. [online] Available at: https://www.emhalliance.org/wp-content/uploads/Women-M-Policy-Paper-FINAL23MARCH.pdf

About PEARL
The Pan-European Real World (PEARL) study will generate important information about real-world effectiveness of fremanezumab in adult patients with chronic migraine or episodic migraine (EM). PEARL is an ongoing, 24-month, multicentre, prospective, observational, Phase IV study.

PEARL will evaluate:

  • Effectiveness
  • Treatment adherence and persistence
  • Effectiveness in patients switching from another mAb targeting the CGRP pathway
  • Concomitant preventive and acute migraine medication use

About AJOVY (fremanezumab-vfrm) injection
AJOVY is indicated for prophylaxis of migraine in adults who have at least 4 migraine days per month. AJOVY is available as a 225 mg/1.5 mL single dose injection in a pre-filled syringe or, in some countries, in a pre-filled pen. Two dosing options are available: 225 mg once monthly administered as one subcutaneous injection (monthly dosing), or 675 mg every three months (quarterly dosing), which is administered as three subcutaneous injections.

AJOVY can be administered either by a healthcare professional or at home by a patient or caregiver. No starting dose is required to begin treatment.

Information for Europe about AJOVY can be found here.

This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse events.  Information can be found at https://www.hpra.ie.

About Teva

Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) has been developing and producing medicines to improve people’s lives for more than a century. We are a global leader in generic, biosimilar and specialty medicines with a portfolio consisting of over 3,500 products in nearly every therapeutic area. Around 200 million people around the world take a Teva medicine every day and are served by one of the largest and most complex supply chains in the pharmaceutical industry. Along with our established presence in generics, we have significant innovative research and operations supporting our growing portfolio of specialty and biopharmaceutical products. Learn more at http://www.tevapharm.com

Breakthrough device for glioblastoma

Carthera receives FDA breakthrough device designation for SonoCloud-9 system, allowing for enhanced interaction with FDA and recognising SonoCloud system as innovative and promising approach for treatment of recurrent glioblastoma

Carthera, a French company that designs and develops SonoCloud, an ultrasound-based medical device to treat a wide range of brain diseases, announced on June 22 2022 that its SonoCloud-9® system has been listed as a Breakthrough Device by the Center for Devices and Radiological Health (CDRH) at the US Food and Drug Administration (FDA).


 The SonoCloud device uses low intensity pulsed ultrasound to temporarily disrupt the Blood-Brain Barrier (BBB), to allow higher brain exposure to therapeutic compounds. By using SonoCloud, the therapeutic efficacy of new and existing therapies can be unlocked and harnessed to improve the treatment of a wide range of brain diseases, such as glioblastoma.
 
“There is a significant unmet need for new treatments for glioblastoma patients, who have very few available therapeutic options,” said Michael Canney, chief scientific officer at Carthera. “We’re excited that the FDA has acknowledged the innovative potential of the SonoCloud approach through the granting of this Breakthrough Designation.”
 
The Breakthrough Devices Designation, intended to expedite the development and review of the medical device, was based on preliminary phase 1/2a clinical data that indicates substantial improvement over available second-line therapy (publication under preparation).
 
“The FDA Breakthrough Device Designation will help Carthera to efficiently advance to a pivotal trial and Premarket Approval (PMA),” said Sandra Thiollière, head of quality and regulatory affairs at Carthera. “We look forward to working closely with the FDA through this accelerated process to bring SonoCloud to patients with recurrent glioblastoma.”
 
This designation will give Carthera access to priority review, more intensive FDA interaction to build an efficient device development programme and commitment from experts and senior managers from the FDA, who will assist the company in addressing any potential challenges during the premarket review phase.

“Following the recent successful completion of our phase 1/2 trial in recurrent glioblastoma, this Breakthrough Device Designation is another important milestone supporting the potential of SonoCloud.”

Frédéric Sottilini, CEO of Carthera.

About SonoCloud-9
The SonoCloud-9 device is implanted in a skull window, below the skin; once in place it is invisible. When activated for few minutes using a transdermal needle connection to an external control unit, the BBB is disrupted for several hours; a window during which drug therapies can be administered. By administering therapies when the BBB is disrupted, drugs can reach the brain in higher and more effective concentrations. This treatment can be repeated at each cycle of drug therapy.
 
About the FDA Breakthrough Device Programme
The goal of the Breakthrough Devices Program is to provide patients and health care providers with timely access to certain medical devices by speeding up their development, assessment and review, while preserving the statutory standards for Premarket Approval, 510(k) clearance and De Novo marketing authorization, consistent with the Agency’s mission to protect and promote public health.

Study: Improvements in TBI Patients with Persistent Brain Fog

A new study has shown that plasticity-based brain training can drive improvements in patients with persistent symptoms of Traumatic Brain Injury (TBI). Gains were observed in standard measures of cognition, standard measures of self-reported symptoms, and the connectivity between brain regions. The intervention used in the study was brain exercises from BrainHQ, made by Posit Science.

Traumatic Brain Injury affects millions of Americans every year, with an estimated 2.5 million treated in emergency rooms. Most recover fully, but for some, the symptoms persist for years and disrupt earning a livelihood and personal relations. An estimated two percent of Americans live with these long-term effects.

Researchers at New York University enrolled 21 patients with chronic TBI that had persisted, on average, for more than seven years. Patients were diagnosed with mild, moderate, or severe TBI, and assigned to either an intervention group or a control group. The intervention group was asked to complete a total of 40 hours of training over 13 weeks (or about 3 hours per week). Before and after the 13-week period, cognitive function was measured with standardised neurocognitive tests, and brain connectivity was measured with fMRI brain imaging.

The researchers found that the brain training group showed significant improvements in standard cognitive tests of attention, memory, and executive function, as well as in standard measures of self-reported symptoms, as compared to the control group. Imaging revealed significant improvement in the functional connectivity across a key network of brain regions – known as the Default Mode Network (DMN). Lower connectivity is associated with cognitive dysfunction and deficits.

“This new study confirms and extends what has been seen in seven prior studies of chronic TBI and BrainHQ, which have shown study participants improved measures of cognitive abilities and symptoms, with imaging showing functional re-organization of the brain” observed Dr. Henry Mahncke, CEO of Posit Science.

These type of persistent symptoms — following not just Traumatic Brain Injuries, but other types of injuries such as “chemobrain,” “cardiobrain,” and “long COVID” — are increasingly known as “brain fog.” Researchers increasingly believe that common brain mechanisms underly the brain fog seen across these disparate conditions – which may explain the similar pattern of results seen in three studies of BrainHQ in cancer patients and seven studies in heart failure patients showing similar types of improvement.

More than 100 published studies of the exercises in BrainHQ have shown benefits, including gains in standard measures of cognition (attention, speed, memory, executive function, social cognition), in standard measures of quality of life (mood, confidence and control, managing stress, health-related quality of life) and in real world activities (gait, balance, driving, everyday cognition, maintaining independence). BrainHQ is now offered, without charge, as a benefit by leading national and 5-star Medicare Advantage plans and by hundreds of clinics, libraries, and communities. Consumers can also try BrainHQ for free at http://www.brainhq.com.