Niagen Bioscience receives FDA, EMA designations for rare disease drug

Niagen Bioscience receives FDA, EMA designations for rare disease drug

Gold slides as U.S.-Iran conflict lifts oil, hawkish Fed bets grow LOS ANGELES - Niagen Bioscience, Inc. (NASDAQ:NAGE) announced today that the U.S. Food and Drug Administration granted Rare Pediatric Disease Designation for its drug candidate NB4168 for the treatment of Ataxia Telangiectasia. The European Medicines Agency also granted Orphan Medicinal Product Designation to NB4168 for the same condition, according to a company press release. The company, with a market capitalization of $271 million, has seen its stock decline 70% over the past year to $3.39, though InvestingPro analysis suggests the stock is currently undervalued.

Ataxia Telangiectasia is a rare genetic disease caused by mutations in the ATM gene that typically presents in early childhood. The condition is characterized by progressive loss of motor coordination, impaired immune function, increased susceptibility to infections, pulmonary complications, elevated cancer risk, and premature aging. The disease affects approximately 1 in 40,000 people in the U.S. and 1 in 150,000 people in Europe.

There are currently no FDA-approved therapies for the condition. NB4168 is an oral small molecule therapy designed to deliver nicotinamide riboside exposure while maintaining what the company describes as a differentiated pharmacokinetic and safety profile. The compound is designed to increase NAD+, a coenzyme involved in DNA repair, mitochondrial function, cellular energy production, and stress responses.

The FDA granted the designation based on its determination that Ataxia Telangiectasia is a serious and life-threatening disease that primarily affects individuals from birth through adolescence and meets the statutory definition of a rare disease. The EMA’s Committee for Orphan Medicinal Products concluded that NB4168 met the criteria for orphan designation for the treatment of the condition. For deeper insights into Niagen Bioscience’s financial health and growth prospects, investors can access the comprehensive Pro Research Report, available exclusively on InvestingPro for this and 1,400+ other US equities.

NB4168 is the first investigational therapeutic candidate from NAD Pharmaceuticals Corp., a wholly owned subsidiary of Niagen Bioscience focused on developing therapies for rare genetic diseases and age-related disorders. The company stated it is advancing preclinical development activities and plans to submit an Investigational New Drug application to the FDA in anticipation of initiating human clinical studies. In other recent news, Niagen Bioscience has launched its first drug candidate, NB4168, through its subsidiary NAD Pharmaceuticals Corp.

The drug targets Ataxia Telangiectasia, a rare genetic disease with no current FDA-approved therapies. This development has led Canaccord Genuity and H.C. Wainwright to reiterate their Buy ratings for Niagen Bioscience, with price targets of $10.00 and $11.00, respectively.

Freedom Broker also initiated coverage on the company with a Buy rating and a $9.00 price target, based on a five-year discounted cash flow analysis. In another development, H.C. Wainwright reiterated its Buy rating after the U.S. District Court for the District of Delaware ruled in favor of Niagen Bioscience in ongoing patent litigation. The court’s decision maintains the enforceability of a patent related to crystalline nicotinamide riboside.

These recent developments highlight Niagen Bioscience’s ongoing efforts in drug development and intellectual property protection. This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.