Ultragenyx Pharmaceutical Inc (NASDAQ:RARE), a development-stage biopharmaceutical company, Monday released encouraging results from a Phase 2 extension study of sialic acid extended-release (SA-ER, UX001) tablets in patients with hereditary inclusion body myopathy that it said support the company's plans to move forward with this program. Also known as GNE myopathy, hereditary inclusion body myopathy is a rare, progressive muscle-wasting disease. SA-ER is designed to replace the deficient sialic acid substrate in patients with HIBM.The results include the 49 out of 59 patients who had 24 weeks at the higher dose. While the 12 grams/day data don't suggest any clinically meaningful advantage over 6 grams/day, the 12 gram data do provide additional data that support clinical activity with SA-ER treatment, Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) said. The 12 gram daily dose of SA-ER appeared to be generally safe and well tolerated with no drug-related serious adverse events, although the rate of mild to moderate gastrointestinal adverse events did appear to be greater with this dose, it noted. Over the entire approximate two-year study, treatment with SA-ER appeared to slow the progression of upper extremity disease when compared to the 24-week placebo group extrapolated out to two years.The initial Phase 2 data, which were presented in April, showed a statistically significant difference in the upper extremity composite of muscle strength at 48 weeks with the higher dose group compared with the lower dose group. SA-ER appeared to be safe and well-tolerated with no serious adverse events observed to date. Most adverse events were mild to moderate and most commonly gastrointestinal in nature. Based on the 48-week and extension study data, RARE intends to discuss with regulatory authorities a potential pivotal study of SA-ER in HIBM patients. The company will also continue to treat patients in the ongoing extension study.CME Group Inc (NASDAQ:CME) shares declined Monday, after the provider of exchange-traded and over-the-counter derivatives received an investment-rating downgrade to market perform from outperform from analysts at Wells Fargo. The brokerage also gave the company a price target range of $75 to $85, down from a previous range of $85 to $95. Shares in CME Group Inc (NASDAQ:CME) were trading 1% lower at $80.77 recently, shortly after Monday's open. Over the past 52 weeks, the stock has traded between $66.44 and $84.71.Intermolecular Inc (NASDAQ:IMI) shares slumped Monday, after the research-and-development company for semiconductor and clean energy industries said it appointed Chairman Bruce McWilliams as its new president and CEO, succeeding David Lazovsky, who resigned "to pursue other interests." Prior to joining Intermolecular, McWilliams served as CEO of SuVolta from June 2009 and as CEO of Tessera from 1999 to 2008, the company said. McWilliams served on Intermolecular's board since 2005 and as chairman of the board since June 2014. Intermolecular Inc (NASDAQ:IMI) shares were off 2.2% at $2.20 shortly after the opening below, moving below the low end of the stock's 52-week range of $2.10 to $6.41.
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