Ultragenyx Pharmaceutical RARE announced that the FDA has accepted its investigational new drug (IND) application for UX016, an investigational small-molecule prodrug of sialic acid, which is being developed as a substrate replacement therapy for GNE myopathy (GNEM).
GNEM, also known as hereditary inclusion body myopathy or Nonaka myopathy, is a rare inherited neuromuscular disorder caused by reduced production of sialic acid. This deficiency leads to progressive muscle wasting, resulting in severe disability and loss of mobility. Currently, there are no approved treatments for this disease in the United States.
Year to date, RARE’s shares have lost 16.1% against the industry’s 0.3% growth.
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Clinical Development Plan
The program is being externally funded by a patient organization through early clinical development, with the first-in-human phase I/II study expected to be initiated in the second half of 2026 in the United States.
Study Design & Endpoints
The study will enroll approximately 24 adults aged 18 to 55 years with GNEM. It will assess the safety, effectiveness and pharmacokinetics of UX016, including its delivery to the muscle. Participants will be given two dose levels compared with placebo over the initial 12 weeks, followed by continued evaluation of muscle strength, patient-reported outcomes and overall functional improvements through 48 weeks of treatment.
UX016 is a prodrug that combines sialic acid with a hydrophobic fatty acid tail to improve its delivery to muscle compared with natural sialic acid. It is designed to overcome limitations that have reduced the effectiveness of earlier substrate replacement therapies.
RARE’s Commercial Portfolio
Ultragenyx Pharmaceutical has built a portfolio of four therapies, namely Crysvita, Mepsevii, Dojolvi and Evkeeza. Crysvita is approved for treating X-linked hypophosphatemia, an inherited disorder and tumor-induced osteomalacia, an ultra-rare disease. Mepsevii is approved to treat mucopolysaccharidosis VII, also known as Sly syndrome. Dojolvi is approved for the treatment of all forms of long-chain fatty acid oxidation disorders. Evkeeza is indicated for homozygous familial hypercholesterolemia.
RARE's Zacks Rank & Stocks to Consider
Ultragenyx Pharmaceutical currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Catalyst Pharmaceuticals CPRX and Indivior Pharmaceuticals INDV, each currently sporting a Zacks Rank #1 (Strong Buy), and ANI Pharmaceuticals ANIP, which presently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 earnings per share have risen from $2.55 to $2.87. CPRX shares have lost 0.6% over the past year.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have risen from $2.89 to $3.08. INDV shares have surged 206.8% over the past year.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 74.53%.
Over the past 60 days, estimates for ANI Pharmaceuticals’ earnings per share have increased from $8.28 to $8.99 for 2026. Over the past year, shares of ANIP have rallied 8.4%.
ANI Pharmaceuticals' earnings beat estimates in each of the trailing four quarters, with the average surprise being 22.21%.
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