Emerging therapeutic areas of clinical attention & investment opportunities

Investment opportunities have changed substantially across the life sciences and pharmaceutical industry in the last ten years. It has become obvious that areas like neuroscience and respiratory medicine require additional support due to unmet clinical needs. Investment is key to facilitate drug development programmes and fund scientific research to better understand diseases with limited therapeutic options.

Investment in key therapeutic areas

Neuroscience 

Neuroscience represents one of the most challenging therapeutic areas across the life sciences. It is a multidisciplinary study of the nervous system, encompassing everything from multiple sclerosis to schizophrenia. Despite the prevalence of neurological diagnoses, this area of research has received less clinical and investment attention in comparison to areas like oncology. Today, 1 in 37 people will be diagnosed with Parkinson’s. Unfortunately, there remains no cure, despite being the fastest growing neurological condition in the world.  

Over the last five years a clear, unmet clinical need has been identified within clinical neuroscience. Conditions like Alzheimer’s represent significant societal and financial burdens and have attracted a substantial investment across drug development, clinical trials and academia. Thanks to pharmaceutical and scientific investment, neuroscience is becoming the main focus for the life science industry.

One of the most significant changes involves the multi-million-dollar investments made in neuroscience startups. In October 2018, in collaboration with Bain Capital Private Equity and Bain Capital Life Sciences, Pfizer launched Cerevel Therapeutics. This new biopharmaceutical company was created with a focus on drug development for disorders of the central nervous system (CNS). Funding for the start-up was confirmed by Bain Capital, who have committed $350 million for future developments. The company aims to target a broad range of CNS disorders including Parkinson’s, Alzheimers and Epilepsy. Cereval will continue to develop Pfizer compounds, aiding drug discovery and development programmes for neurological and neuropsychiatric conditions.

The nature of the nervous system in controlling the body results in a vast number of disorders which cross over multiple systems. Complex disorders like multiple sclerosis are an example of a neurological disorder that arises with immunological problems. MS is an inflammatory disorder in which the immune system attacks the insulation around the nerves in the CNS. Unfortunately, there is no cure for MS. 

However, MS clinical research has seen a significant boost in funding thanks to recent investment. In January 2020, Switzerland-based GeNeuro invested €17.5 million into MS clinical trials. The clinical pharmaceutical company, with a focus on autoimmune diseases, sold stock to fund trials testing temelimab in MS patients whose condition progresses without relapse. Unfortunately, early clinical data failed to reach the primary endpoint, which lost significant investment. Thankfully thanks to financial support from Institut Mérieux, (a holding company for Mereieux life science companies) GenNeuro can continue the clinical trials. According to a recent press release by GeNeuro “GeNeuro’s temelimab Phase 2 multiple sclerosis trial cleared to continue following planned data safety monitoring board meeting”.

Respiratory medicine  

Despite the prevalence of serious respiratory conditions, there have been very few novel, approved therapies for respiratory medicine in the last 40 years. Lung health has been a major global challenge in the industry for many years. Respiratory-related deaths are likely to increase with climate change, increased air pollution and allergens. Chronic obstructive pulmonary disorder is an example and is amongst the leading causes of death globally. Asthma, one of the most prevalent diseases in Europe, has shown little progression in treatment development for patients with severe conditions. 

The COVID-19 pandemic has also emphasised the impact of respiratory damage on the human body. However, it appears that respiratory medicine was an already growing area of clinical focus before the pandemic. As of April 2019, Pharmaceutical leader Boehringer Ingelheim completed a major investment for respiratory diseases. A total of €105 million was invested into expanding operations at sites in Dortmund and Ingelheim to increase production of respiratory medicine. The main areas of focus for the company include cystic fibrosis, COPD, asthma and other serious lung diseases.

According to a news release, the global revenue for the respiratory market has also seen a substantial increase from $30.9bn in 2016 to a forecast of $41.3bn in 2023. The approval of novel drugs like mepolizumab is also predicted to have a strong commercial impact on the market. In the same article, it stated “in 2016, GSK was the leading company in terms of revenue generated from respiratory disorder products, accounting for 30.3% of the market, while Vertex had a small market share of 5.3%”. This increase in global revenue for the respiratory market appears to be associated with the launch of novel respiratory drugs. This supports the positive impact of investment in drug development.

Platforms and technologies

Cell and gene therapy  

Cell and gene therapy (CGT) has seen significant investment from the biopharma industry over the last couple of years. This technique offers a more direct approach of targeting a disease at the cellular or genetic level, rather than treating the symptoms. According to a 2021 article “nearly $20 billion in funding flowed into biotech companies developing cell-, gene- and tissue-based therapies last year, widely eclipsing the total invested in 2019 and ending up 50% higher than the previous record of $13.5 billion set in 2018.” Gene therapy involves the modification of genetic expression by repairing or altering abnormal genes.

This innovative approach to treatment can be applied to a multitude of diseases, including those resistant to conventional treatment – hence substantial investment across the industry. In a review of CGT, a comparison was made between the efficacy of standard treatment vs CGT in a study of patients with an aggressive form of non-Hodgkin’s lymphoma (who had relapsed or resisted treatment). In this particular study, it highlighted that “the median overall survival time for the previous standard of care treatment is just 6 month”. However, a clinical trial of Yescarta, a form of gene therapy, showed that “72% of treated patients had an overall response (tumour shrinkage or elimination) and 51% had no detectable cancer (“complete remission”) six months following treatment.” Such evidence of its efficacy is one of the reasons why CGT is becoming a key part of oncology research in particular.

Charlotte Di Salvo, Former Editor & Chief Medical Writer
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