Neurology is a fascinating space that supports millions of people suffering from debilitating neurological disorders.
This includes diseases of the central and peripheral nervous system. In other words, the brain, spinal cord, cranial nerves, peripheral nerves, nerve roots, autonomic nervous system, neuromuscular junction and muscles.
Finding treatments and therapies for this is challenging because of the complexity and sophistication of both the central and peripheral nervous system. That’s why I get so excited when new treatments are rolled out and am proud to be part a space that bravely takes on this task.
In my role as a specialist recruiter of professionals in neurology, I come across different exciting companies every day. Each has their own unique story and innovative treatments to match. Today, I want to share with you five companies who’s work you need to know about.
Let’s start with the newest company…
Avrion Therapeutics was founded in 2020 and is based on over 10 years of research on the Brain Mind Institute and Bertarelli Foundation’s gene therapy platform at the Swiss Federal Institute of Technology Lausanne (EPFL).
The Swiss start-up has developed a first-in-class precision gene therapy platform to selectively and safely target discrete cells in the brain including neurons and astrocytes. This therapeutic platform is supported by a preclinical discovery engine to identify novel targets to treat neurodegenerative diseases.
Avrion is on a mission to lead the way in precision gene therapies for neurodegenerative diseases. This starts with providing treatment for a form of motor neurone disease (MND) called amyotrophic lateral sclerosis (ALS).
ALS is also known as Lou Gehrig's disease, after the baseball player who was diagnosed with it. It’s a progressive nervous system disease that affects nerve cells in the brain and spinal cord, causing a loss of muscle control.
Avrions’ first clinical candidate to treat this, AVR-001, is preparing to enter IND-enabling. If successful, Avrion will be able to move onto its first-in-human clinical trials.
This treatment has the potential the support an unmet need, change many patients lives and make advances that the healthcare has been trying to achieve for decades. So, it’ll be exciting to see how the new start-up get on.
While we’ll have to wait and see with Avrion, Cyclerion Therapeutics is further down the development pipeline.
This clinical-stage biopharmaceutical is committed to discovering, developing and commercialising innovative medicines for people with serious diseases of the central nervous system (CNS). Its CY6463/MELAS treatment is in phase 2 of clinical trial.
CY6463/MELAS treats mitochondrial encephalopathy, lactic acidosis and stroke-like episodes (MELAS) which is one of the most common and severe mitochondrial diseases. This rare genetic disorder affects the function of mitochondria, the powerhouses of the cell.
Mitochondrial disease can affect almost any part of the body. The tissues and organs that are most often affected are those with the highest energy demands, such as the brain, heart, eyes and skeletal muscles.
With the CY6463/MELAS treatment, Cyclerion aim to unlock the full therapeutic potential of the nitric oxide-cyclic guanosine monophosphate (cGMP) signalling pathway. The company argue that while nitric oxide (NO) is one of several fundamental neurotransmitters, it has yet to be leveraged for its full therapeutic potential in the CNS.
To take this treatment closer to the market, Cyclerion plan to initiate a phase 2a multi-centre, open-label study in MELAS at centres of excellence for mitochondrial diseases. In addition to safety, the study will assess the impact of short-term CY6463 treatment on biomarkers of mitochondrial dysfunction, brain perfusion, neurodegeneration and cognition.
Moving onto one of the larger companies in this piece, Cerevel Therapeutics has also focused much of its efforts on developing new therapies to treat disorders of CNS.
The company has a portfolio of experimental neuroscience therapies, which include three clinical-stage compounds and several pre-clinical compounds designed to target a broad range of CNS disorders including Parkinson’s, Alzheimer’s, epilepsy, schizophrenia and addiction.
Headquartered in the Greater Boston area, Cerevel was formed in 2018 through a partnership between Bain Capital and Pfizer. The company is a clinical-stage biopharmaceutical company that combines a deep understanding of the biology and neurocircuitry of the brain with advanced chemistry and central nervous system (CNS) receptor pharmacology to discover and develop new therapies.
The leadership team has a great track record of drug approvals and commercial success. On April 21st, they added to this by appointing Abraham N. Ceesay as President. He joins a team including CEO, Tony Coles, who served as president of Onyx Pharmaceuticals until its acquisition by pharma powerhouse, Amgen.
As well as Ceesey and Coles, the company are also backed by the experience of Raymond Sanchez, CMO, who has worked for a number of successful companies such as Otsuka Pharma and Bristol-Myers Squibb.
Like Cerevel and Cyclerion, Arvelle Therapeutics is also committed to helping patients who are affected by CNS disorders – more specifically, epilepsy.
The biopharmaceutical’s Cenobamate, formerly known as YKP3089, was originally discovered by SK Biopharmaceuticals and SK life science. In the US, it’s been FDA approved since 2019 as antiseizure medication (ASM) for the treatment of partial-onset seizures in adults (also known as focal-onset seizures).
However, it’s not until this March that Arvelle was able to take the treatment to the European market. Now working as a part of Angelini Pharma, ONTOZRY (also known as Cenobamate) was granted marketing authorization by the European Commission.
In Europe, there’s an estimated 6 billion people with epilepsy and approximately 40% of adult patients with focal epilepsy have inadequate control of seizures after treatment with two anti-seizure medications (ASMs).
This new treatment option could change these people’s lives and marks a breakthrough in the treatment of focal-onset seizures in adults with epilepsy.
Vanda Pharmaceuticals is also traying to make a difference in the world of neurology, releasing the first ever treatment for people with a rare neurobehavioral disorder called Smith-Magenis Syndrome (SMS).
SMS is characterised by a recognizable pattern of physical, behavioural and developmental features. It is caused by genetic changes on the chromosomal region that contains the gene RAI1.
In 2020, Vanda received FDA approval for its HETLIOZ (tasimelteon) capsule and liquid formulations for the treatment of night-time sleep disturbances associated with SMS. This advancement addresses a significant problem for SMS patients and is yet another example of the crucial work that neurology companies are responsible for.
While I’ve only mentioned five companies in this article, it’s important to stress that I could have mentioned so many more. Neurology is a fantastic, innovative space in which so many inspirational people work hard to advance healthcare and change patients’ lives.
I’d love this article to spark a conversation within the space. So, what companies do you think should have been included in this article? And why? You can email me at Harry.Aaronson@lifesci-cm.com or give me a quick message on LinkedIn.
Anyway, thanks for reading this. You can find more of my content and learn more about my recruitment services on my profile page.
These revolutionary companies are changing the game when it comes to neurological drug development, and thanks to recent funding they could be on the market sooner than you think. Click to find out more.
In this live recording, we were joined by a panel of leaders from the digital lab solutions space to discuss how data translates in the lab world, the challenges and strategies surrounding data maturity and the future of lab data. Click to listen.
In CM Life Science's latest webinar, we discussed how data translates into the lab world, the challenges surrounding data maturity and what lab data will look like in the future. Click to watch.
Cell and gene therapy continues to provide a better standard of living for millions of people with serious health conditions. Click to find out about some of the most exciting and innovative start-ups in this space.