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Top Pharmaceutical Company Evaluates Optimer for Precision Liver Medicine

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Aptamer Group plc, the developer of novel Optimer® binders to enable innovation in the life sciences industry, today announces that its Optimer binder against cells associated with fibrotic liver disease, developed for targeted delivery of therapeutic payloads to fibrotic liver disease, has been shipped to a top 15 pharmaceutical company for evaluation in their own therapeutic applications.

The Optimer delivery vehicle was developed to selectively target cells linked to fibrotic liver disease, as part of a fee-for-service partnership with the pharmaceutical company that began in 2020. Subsequent work at Aptamer Group combining the Optimer with a model gene therapy payload has shown that the Optimer enables selective delivery of the gene therapy specifically to fibrotic human liver cells in laboratory tests, with no interaction with other liver cell types. This allows the gene therapy to be effective in target fibrotic liver disease cells, with no effect in the non-target liver cells. A significant effect was seen from the Optimer-delivered gene therapy, compared to the gene therapy alone, with a p-value of less than 0.01, indicating the probability of these results is more than 99% due to the Optimer delivery of the gene therapy. As these data demonstrate the successful therapeutic delivery using the Optimer binder, the pharmaceutical partner requested samples of the material for their own evaluation, with a view to licensing this binder for further development of new precision liver medicines. The required material has been manufactured by Aptamer Group and, following internal testing by the Company, has been released to the pharmaceutical partner.

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Targeted delivery to specific cell populations is showing increasing use across the pharmaceutical sector, as it holds the potential for increased efficacy and reduced side effects from medications. Exemplar publicised partnerships for specifically targeted precision medicine developments include Ionis Pharmaceuticals Inc. licensing a single delivery vehicle from Bicycle Therapeutics Inc. for $45 million upfront, with additional milestone payments and royalties for each program developed under the collaboration; Partnering of Takeda Pharmaceutical with Peptidream Inc, with Peptidream Inc receiving up to $3.5 billion excluding royalty payments for delivery vehicles targeting the central nervous system. Both these deals demonstrate the urgent requirement and high values associated with developments and assets of this nature in the sector for successful targeting delivery vehicles.

Estimates suggest that globally, two million lives are lost annually to liver disease, and 1.5 billion people suffer from chronic liver disease. The most common liver disease is steatotic liver disease, accounting for ~60% of cases, which in its worst stages is known as metabolic dysfunction-associated steatohepatitis (MASH) and can progress to liver cancer. Despite this prevalence, treatment options are limited, with only one medication currently approved for MASH and liver fibrosis. Aptamer’s delivery vehicle can be simply combined with gene therapies or other therapeutic payloads to enable the development of new precision medicine approaches for the treatment of fibrotic liver disease and liver cancers in this area of high unmet need.

“We are delighted to be able to supply our Optimer binders to our pharma partner for evaluation. Their positive feedback on our in-house data suggests that the Optimer binders could outperform the alternative molecules they are currently testing.

“For gene therapy, enabling targeted delivery of functional medicines to new tissues remains a major translational challenge preventing the full recognition of gene therapies potential. Our in-house studies suggest we have overcome these issues in developing these Optimer binders and could offer new hope for precision medicines for liver disease, a patient group that is hugely underserved with limited treatment options.

“I look forward to supporting our partners in their use of Optimer delivery vehicles and updating the market as this partnership progresses.” Dr Arron Tolley, Chief Technical Officer of Aptamer Group