Cytokine release syndrome preventative therapy could be a >$10bn market opportunity

Published: 12-Feb-2024

Shifting focus to rare and orphan diseases could be beneficial to patient outcomes and company incomes, respectively

Poolbeg Pharma, a biopharmaceutical company specialising in the development and commercialisation of medicines targeting rare and orphan diseases, today announces that independent research conducted on its behalf confirms a >$10 billion market opportunity for POLB 001 in Cancer Immunotherapy-Induced CRS as an orally delivered preventative therapy. 

Cancer immunotherapies have been approved in rare and orphan blood cancers, so the Company can see potential for POLB 001 in one or more of these cancer types. 

Acquiring and commercialising rare and orphan drugs could potentially lead to attractive revenue generation opportunities

POLB 001 will be developed for use in cancer immunotherapy-induced CRS.

Rare and orphan diseases represent a significant area of unmet need for patients, as they are often understudied.

There remains a substantial opportunity for the formulation of innovative medicines to treat these severe and often chronic conditions. 

It is anticipated that acquiring and commercialising rare and orphan drugs could potentially lead to attractive revenue generation opportunities. 

Poolbeg will draw on the expertise of a wider team, including the individuals of the former Amryt team, who have a proven track record of successfully building and commercialising orphan drugs. 

Regulatory authorities offer incentives to companies developing orphan drugs, such as reduced filing fees, tax breaks and market exclusivity post approval.

The field of cancer immunotherapies, including CAR-T and BsAb, is burgeoning and is expected to grow to US$100bn – US$140bn by 2030. 

The company directors at Poolbeg believe that the company is well positioned to benefit from these opportunities. 

The company also announced further detail on the significant market opportunity for POLB 001 CRS induced by CAR-T and bispecific antibody treatments (BsAb), where CRS can affect the majority of patients and can lead to extended hospital stays, discontinuation of treatment and mortality risk.

There are currently few approved therapies for the management of acute CRS and no preventative therapies indicated for CRS.

The field of cancer immunotherapies, including CAR-T and BsAb, is burgeoning and is expected to grow to US$100bn – US$140bn by 2030. 

CRS occurs in the majority of CAR-T and BsAb treatments frequently impacting >70% of patients and cannot be predicted.

All grades of CRS can lead to extended hospital stays, causing an estimated US$5.5bn of hospitalisation costs to healthcare systems, and further indirect costs through patient treatment and follow up.

An effective oral preventative therapy for cancer immunotherapy-induced CRS has the potential to significantly reduce direct costs in the healthcare system and improve patient access to these therapeutics, potentially including access to these therapies outside of specialist centres, while further augmenting wider market uptake.

Poolbeg recently announced promising in vivo results for POLB 001 in addressing cancer immunotherapy-induced CRS, where CRS clinical symptoms were significantly improved by its administration. Dose dependent reductions were seen in all serum proinflammatory cytokines measured.

Jeremy Skillington, Chief Executive Officer, Poolbeg Pharma plc said: “Rare and Orphan disease medicines fit Poolbeg’s efficient capital model particularly well. Clinical development is typically more cost efficient and the market opportunity remains high because of the high unmet medical need.’’

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