Oncology alone accounts for one-third of the global clinical pipeline across all therapeutic areas. In 2024, the global oncology therapeutics sales are forecasted to hit $250 billion. As the world’s largest therapeutic area, it is a highly competitive market with ten of the largest pharmaceutical companies in the world declaring oncology as a focus area. Immunotherapy and rare cancer research are a few of the areas in oncology receiving significant investment and clinical attention.

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In order to address some of the pressing challenges in cancer research, numerous partnerships have been formed in order to share capital, knowledge and resources. In June 2019, Pfizer announced the acquisition of biopharmaceutical company Array. The merger, valued at $11.4 billion, was formed under an agreement “to focus on the discovery, development and commercialization of targeted small molecule medicines to treat cancer and other diseases of high unmet need.” This is one of many pharmaceutical collaborations within the industry to strategise about the direction for research and development and accelerating the drug development process. 

More recently, Scottish investment firm Eos Advisory entered into a strategic partnership with Kineticos to co-invest in early-stage Scottish life sciences companies. The goal from this partnership appears to be addressing substantial unmet clinical needs across neuroscience, oncology, and rare diseases with new approaches to precision medicine. According to a recent BBC article, it is expected that £10 million will be invested over the next five years in “oncology-focused firms”.

Precision oncology: Immunotherapy

In the last decade, cancer research has seen a significant shift in disease management with approval of immuno-oncology therapies like checkpoint inhibitors. While traditional cancer treatment remains successful for many patients, immunotherapy has shown potential for developments in precision oncology. This offers hope for patients resistant to conventional therapies and those suffering from the rarest forms of the disease.

In a recent article, it was declared that in January 2021, BMS and ArsenalBio announced a multi program ”discovery collaboration to advance next-generation T-cell therapies for the treatment of solid tumors”. ArsenalBio primarily focuses on CRISPR and other gene editing technologies, which BMS showed great interest in. According to the article, the desired outcome of this collaboration is to achieve controlled modification of the T-cell genome. From here, the CRISPR expertise of ArsenalBio could allow for “strategic placement of gene edits” that can induce a more effective immune response at the site of the tumour.

Investment in immuno-oncology has been focused on combination studies. Combination therapy – the use of a combination of products or modalities to treat cancer – is one way to use biomarkers to reveal the tumour microenvironment and identify targets for specific immunotherapy treatment. In January 2020, gene therapy company Genprex “secured FDA Fast Track Designation for its Oncoprex immunogene therapy in combination with the EGFR tyrosine kinase inhibitor (TKI) osimertinib (AstraZeneca’s Tagrisso®)”. Following this significant step forward in cancer drug development, in February 2020, Genprex secured a $7.5 million at-the-market offering to fund Oncoprex drug development. Oncoprex is a nanoparticle delivery system. The investment in this particular combination therapy continues, with $10 million raised by Genprex to be used toward continuing the company’s clinical trial of Oncoprex.

It is worth noting that cell and gene therapies are also a prime area for investment in precision oncology. 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. 

Clinical research: Rare cancers 

Survival rates for rare cancer types like gastric cancer continue to be low in comparison with prevalent types like breast or prostate cancer. Unfortunately, rare cancers also have a worse five-year survival rate in comparison to common forms of the disease. Poor outcomes in clinical research are limiting progression in precision medicine for rare cancer, leading to recent investment. 

In March 2021, RareCan, a UK-based rare cancer start-up company, secured a six-figure investment for research to fast track treatment development and diagnostics. According to a recent article the funds raised will be used to “recruit a cohort of patients, further develop data capabilities and grow the company’s operational and marketing capacity.” Investment in patient recruitment is an important part of rare disease research, and has been notoriously difficult due to the smaller patient populations. 

In 2019, the Australian government pledged to invest $55 million to research rare cancers and diseases. Of this, $15 million will be used for research into reproductive cancers and another, $5 million is for Childhood Brain Cancer clinical trials, $15 million will address other significant gaps in current research and/or knowledge in rare cancers, rare diseases and areas of unmet medical need. While ovarian cancer on average affects one in 70 women in the US, there are rare forms of the cancer with limited therapeutic treatment. In addition, only 35% of ovarian cancer patients survive for ten years or more. 

While an increasing number of organisations are moving towards rare cancer research, achieving investment is the first challenge. Grant funding agencies face a problem in the assessment of rare cancer research proposals. A successful application for cancer research funding requires the following components:

• Adequate sample calculations for biomarker discovery and validation

• Preliminary in vitro evidence of the research question

• Cell lines and animal models to test the research hypothesis

• Adequately designed and powered clinical trials 

A number of these components are problematic in rare cancer research, which results in a vicious cycle. For example, an adequate number of successful clinical trials is often a challenge for rare diseases with small patient populations distributed across the globe. This essentially creates a vicious cycle: without a sufficient number of powered clinical trials, or animal models to support research, funding will not be granted due to the poor quality of the rare cancer proposal. However, clinical and scientific research needed to create a strong funding proposal cannot run without funding.

As a result, the majority of funding for rare cancer comes from investment within big pharmaceutical companies or partnerships between big pharmas and smaller biotech companies.

Charlotte Di Salvo, Junior Medical Writer
Proventa International