GMTX - Gemini stock rises 15% as Disc's bitopertin gets FDA orphan drug tag

• The U.S. Food and Drug Administration (FDA) granted orphan drug designation to Disc Medicine's therapy bitopertin to treat erythropoietic protoporphyria (EPP).
• EPP is a rare inherited disorder caused by a deficiency of an enzyme called ferrochelatase (FECH), which leads to increased levels of the protoporphyrin in the red blood cells. Symptom of the disorder include pain and burning sensation  on exposure to sunlight and certain types of artificial light.
• Disc said bitopertin is an investigational oral, selective inhibitor of glycine transporter 1 (GlyT1) designed to modulate heme biosynthesis, and has been shown in preclinical studies to reduce accumulation of protoporphyrin IX, the toxic metabolite that causes the disease in the patients with EPP.
• The drug is being evaluated in two ongoing phase 2 studies called AURORA and BEACON in EPP.
• Privately held Disc and Gemini Therapeutics ( NASDAQ: GMTX ) are merging in an all-stock transaction, which was announced in August.
• The FDA grants orphan drug status to therapies which treat or prevent rare diseases that affect fewer than 200K people in the U.S. The designation provides certain incentives, including seven years of market exclusivity, if approved.
• GMTX +15.48% to $1.79 premarket Dec. 27

For further details see: