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Milan, February 22, 2023.

European submission in the second half of 2023. US regulatory pathway defined: Special Protocol Assessment Agreement (SPA) with the FDA

EryDel SpA, a late-stage global biotechnology company whose goal is to develop and commercialize therapeutics for the treatment of rare diseases using its proprietary red blood cell technology, today announced that the company is preparing a European Marketing Authorization Application (MAA) for its presentation in H2 of 2023 for EryDex for the treatment of Ataxia Telangiectasia (AT). AT is a rare pediatric neurodegenerative disorder for which there is no approved treatment. Following positive consultation with the European Medicines Agency (EMA), subsequent correspondence concluded that the data package available for submission is complete and meets the requirements for the regulatory authority to complete its review of EryDex. In addition, EryDel has reached an agreement with the US Food and Drug Administration (FDA) under the Special Protocol Review (SPA) process on the design, endpoints, and statistical analysis of a new Phase 3 clinical trial. (the NEAT trial). The SPA states that the intended trial design and analysis, as set forth in the protocol submitted to the FDA, adequately addresses the objectives necessary to support the New Drug Application submission for EryDex. "We are pleased to have completed significant steps, which clarify the regulatory pathway for EryDex for the treatment of AT," said Luca Benatti, EryDel's CEO. "In Europe we are on track to introduce the European MAA and in the US we have made progress with the FDA on the SPA. These are important milestones as we move forward in our work to bring this valuable therapy to the AT community." EryDel will continue to work with both the FDA and the EMA to advance the submission of EryDex. EU marketing authorization submission is scheduled for the second half of 2023. About Ataxia Telangiectasia Ataxia telangiectasia (AT) is a devastating, autosomal recessive, inherited, multisystem disorder. TA has an estimated worldwide prevalence of between 1 in 40,000 and 1 in 100,000 live births (Rothblum-Oviatt et al., 2016). ED is a disease with high morbidity and mortality. The disease is characterized by progressive cerebellar degeneration, leading to increasing impairment of motor function during the early school years and wheelchair dependence in the second decade of life. Other features are telangiectasia, immunodeficiency, recurrent pulmonary infections, sensitivity to radiation, and a predisposition to the development of malignant tumors. There is currently no approved treatment for this devastating disease. About EryDel SpA EryDel SpA is a late-stage global biotechnology company whose goal is to develop and commercialize therapies for the treatment of rare diseases using its proprietary red blood cell technology. Its most advanced product, EryDex, is in late-stage development for the treatment of ataxia telangiectasia (AT), a rare autosomal recessive neurological disorder for which there is currently no established therapy. EryDex is an automated ambulatory technology to encapsulate dexamethasone sodium phosphate (DSP; an inactive prodrug) ex vivo in the patient's red blood cells, which are then reinfused, allowing slow release into circulation of low doses of dexamethasone (active drug). throughout a month. EryDex has received orphan drug designation for the treatment of AT from both the FDA and the EMA. A pivotal phase 3 international multicenter study, ATTeST, and its long-term extension have been successfully completed and regulatory submission in the EU is being prepared. In addition to EryDex, EryDel's technology platform in enzyme replacement therapy supports a wide range of therapeutic opportunities led by Ery-PAL for the treatment of PKU.

ContactContact name: Kate BarretteContact description: RooneyPartners LLCContact phone: 1 212-223-0561