Launch of Aptadir Therapeutics With a Novel Class of RNA Inhibitors

• A novel approach for intractable cancers and genetic conditions
• Science originates from 3 top international institutions – the Beth Israel Deaconess Medical Center of the Harvard Medical School, the Italian Research National Council (CNR) and the Cancer Science Institute of Singapore
• First investment for EXTEND technology transfer biotech hub joint venture, comprising CDP Venture Capital SGR, Angelini Ventures and Evotec SE
• Appointment of Giovanni Amabile as Executive Chairman and acting CEO

Aptadir Therapeutics (Aptadir), a biotech company developing RNA inhibitor-based therapeutics for treating intractable cancers and genetic conditions, announces its launch and the appointment of Giovanni Amabile as Executive Chairman and acting CEO.

Aptadir was founded by Dr Annalisa Di Ruscio and Dr Vittorio De Franciscis, both leaders in the field of RNA biology and DNA methylation, alongside Prof. Daniel Tenen, an expert in MDS and leukaemia, and Prof. Marcin Kortylewski, an expert in oligonucleotide immunotherapeutics. They are based at world class institutions: the Beth Israel Deaconess Medical Center of the Harvard Medical School, the Italian Research National Council, the Cancer Science Institute of Singapore and City of Hope National Medical Center.

The basis for the Company was the landmark discovery of a new class of RNA inhibitors called DNMTs Interacting RNAs (DiRs)^1,2. DiRs are capable of blocking aberrant DNA methylation at a single gene level reactivating previously hypermethylated genes – a key feature in cancer and genetic disorders. By reactivating specific genes, there is the opportunity to target the underlying cause of and potentially reverse specific intractable cancers and genetic conditions in adults, such as Myelodysplastic Syndrome (MDS) and, in children, such as Fragile X Syndrome.

Aptadir’s proprietary platform can generate multiple DiRs for specific genes suppressed in cancer and genetic conditions, re-establishing the pathway functionality. The first programme, Aptadir Ce-49 is being developed in MDS and the aim is to enter the clinic by the end of 2025.

Leading the management team is Giovanni Amabile who has been appointed Executive Chairman and acting CEO; he is also Entrepreneur in Residence of EXTEND. Previously, he was CEO at Enthera and before then, CSO at ADIENNE Pharma. He has a successful track record in raising seed and Series A funds, and for leading IND approvals, complex clinical trials and regulatory approvals in the US.

Giovanni Amabile, Executive Chairman and CEO, said: “Today represents the launch of Aptadir Therapeutics and is an exciting day for the founders and team. Aptadir’s business is based on real innovation - a landmark discovery of a new class of molecules which have the potential to be best-in-class therapeutics for intractable conditions. From data already generated, DiRs are highly selective, stable and non-toxic, and have the potential to be used across multiple indications. This is a really exciting new field and we are looking forward to pushing our first candidate forward into the clinic.”

Aptadir has received $1.6m in pre-seed funding from the EXTEND initiative to optimize its lead asset, Aptadir Ce-49. The Company is the first to receive funding from EXTEND, which is the Italian National Technology Transfer Hub wholly dedicated to the biopharmaceutical sector for the development of new therapeutic approaches. The Hub was initiated by CDP Venture Capital SGR, and joint funded with co-investors, Angelini Ventures and Evotec SE. Its purpose is to develop translational drug discovery partnerships with highly renowned universities and research centers in Italy to accelerate their promising therapeutics for commercialization.

Claudia Pingue, senior partner and head of the Technology Transfer Fund of CDP Venture Capital SGR, added: “Aptadir is the first biotech company born from EXTEND, National Technology Transfer hub established on the initiative of CDP Venture Capital and funded with co-investment partners, Angelini Ventures and Evotec. EXTEND aims to develop high quality science coming from top Italian universities and to help build new start-ups that can develop that science for the benefit of future patients. Aptadir represents one of the best examples of where science innovation can create a new potential therapeutic for underserved conditions in adults and children, such as MDS and Fragile X Syndrome. We’ll be watching its progress with great interest.”

1. Di Ruscio A et al 2013, Nature
2. Esposito CL et al 2023, Nature Communication

Notes to Editors

About Aptadir Therapeutics

Aptadir Therapeutics is a preclinical biotech company developing a new class of RNA therapeutics. The Company’s therapeutic focus is underserved intractable cancers and genetic conditions, including underserved intractable conditions in the oncology and rare diseases fields such as Myelodysplastic Syndrome and Fragile X Syndrome.

The technology was developed and validated by a group of key opinion leaders in the field of RNA therapeutics based at three world class institutions: the Harvard Medical School, Cancer Science institute of Singapore, Italian CNR Institute and City of Hope National Medical Center. The platform is based on a landmark discovery of a new class of RNA inhibitors called DNMTs Interacting RNAs (DiRs)^1,2. DiRs are capable of blocking aberrant DNA methylation at a single gene level reactivating previously hypermethylated genes – a key feature in cancer and genetic disorders.

The first programme is developing a treatment for Myelodysplastic Neoplasms (MDS), a group of blood cancers that occur when immature blood cells in the bone marrow don't mature into healthy blood cells. Sufferers of MDS go on to develop acute myeloid leukaemia (AML).

To find out more, please see: www.aptadir.com

About EXTEND

EXTEND is the Italian National Technology Transfer Hub (the Hub) wholly dedicated to the biopharmaceutical sector for the development of new therapeutic approaches and it was formed as an initiative of CDP Venture Capital to transform the results of this scientific research into new companies.

Alongside CDP Venture Capital, the Hub is funded by co-investors, Evotec, which also acts as an entrepreneurial partner by supporting research groups along the path of scientific study up to the establishment of startups ready to raise capital on the market to start the clinical phase, and Angelini Ventures, the new Angelini Industries Group company operating in the VC sector with a focus on digital health and biotech.

The scientific promoters of the initiative are IRSCC San Raffaele Hospital in Milan, the Universities of Milan, Florence, Modena and Reggio Emilia, Padua, Trento, La Sapienza in Rome, the regional research district of Puglia through H-Bio, and Human Technopole.

To learn more, please visit: www.extend-tt.vc

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