Modis Announces PRIME Designation by European Medicines Agency

Modis Therapeutics announced the European Medicines Agency (EMA) has granted PRIME (PRIority MEdicines) designation to MT1621, Modis' investigational treatment for patients with thymidine kinase 2 deficiency (TK2d).

The PRIME designation is awarded by the EMA to promising medicines that target an unmet medical need. Since the program was launched in March 2016, the EMA has granted PRIME designation to only 36 medicines (21% of 177 requests). The application was supported by data from initial clinical studies in TK2d patients, some of which was presented by Dr. Michio Hirano at the International World Muscle Society (WMS) Congress last month.

"We are pleased that MT1621 has been granted PRIME designation by the EMA," said Joshua Grass, Chief Executive Officer of Modis Therapeutics. "We believe this designation from a major health authority provides validation of our clinical data and therapeutic approach and recognition that TK2 deficiency represents a significant unmet medical need. We look forward to collaborating with the EMA to accelerate development of this important therapy for patients with TK2 deficiency."

MT1621 has also been granted Orphan Drug designation by both the FDA and EMA.

The PRIME designation program focuses on medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. These medicines are considered priority medicines by the EMA. To be accepted for PRIME, a medicine has to show its potential to benefit patients with unmet medical needs based on early clinical data.

Through PRIME, the EMA offers enhanced support to medicine developers including early interaction and dialogue and a pathway for accelerated evaluation by the agency. The program is intended to optimize development plans and expedite the review and approval process so that these medicines may reach patients as early as possible.

Thymidine kinase 2 deficiency (TK2d) is a genetic disorder that results in mitochondrial dysfunction, leading to inadequate energy production in cells. TK2d may present at all ages and causes progressive and severe muscle weakness, respiratory insufficiency, and is often fatal.

MT1621 is an investigational deoxynucleoside combination therapy that targets the underlying pathophysiology of TK2 deficiency. Deoxynucleoside combination therapy has been shown to improve cell function and prolong life in preclinical models of TK2d. Data from initial clinical studies suggest that this therapy may meaningfully alter the course of disease in patients with TK2d.

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