On March 11, 2014, Ultragenyx Pharmaceutical announced the first patient enrolled in the Phase 2 study of triheptanoin for the treatment of glucose transporter type-1 deficiency syndrome, or Glut1 DS, at Columbia University. Glut1 DS, also known as De Vivo disease, is a rare, severely debilitating disease characterized by seizures, developmental delay, and movement disorder. The global, randomized, double-blind, placebo-controlled, parallel-group Phase 2 clinical trial will evaluate safety and efficacy in up to 50 Glut1 DS patients between 3 and 17 years of age inclusive, who are currently not on or not fully compliant with ketogenic diet and continue to have seizures. The primary efficacy objective is the reduction in frequency of seizures compared to placebo following a 6-week baseline period and subsequent 8-week placebo-controlled treatment period. The blinded treatment period will be followed by an open-label extension period in which all patients will be treated with triheptanoin through week 52. Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) stock opened at $67.18 in last trading session, and closed at $60.48, trading in the range of $56.89 – $69.77. The stock showed a positive weekly performance of 11.22%.
On March 10, 2014, Telik, Inc. (NASDAQ:TELK) reported a net loss of $0.8 million, or $0.17 per share, for the three months ended December 31, 2013, compared with a net loss of $1.8 million, or $0.66 per share, for the comparable period in 2012. For the year ended December 31, 2013, net loss was $5.2 million, or $1.17 per share, compared with a net loss of $8.0 million, or $3.64 per share, for the year ended December 31, 2012. For the quarter ended December 31, 2013, total operating costs and expenses were $0.8 million compared with $1.8 million in the fourth quarter of 2012, representing a reduction of approximately 56%, primarily due to lower headcount, reduced clinical trial expenses, and lower corporate and stock-based compensation expenses. Telik, Inc. (NASDAQ:TELK) shares closed at $1.44 on last trade day, by losing -9.43%. Stock 52 week range is $1.05 – $2.15. Company’s market capitalization is $6.60 billion.
On March 13, 2014, Aratana Therapeutics Inc (NASDAQ:PETX), a pet therapeutics company focused on licensing, developing and commercializing innovative biopharmaceutical products for cats, dogs and other companion animals, today announced its fourth quarter and full year 2013 financial results. Aratana Therapeutics Inc (NASDAQ:PETX) stock opened at $19.41 in last trading session and closed at $17.60. The 52 week range of the stock is $6.56 – $29.32 and the day range was $17.34 – $20.74.
On Feb. 28, 2014 — Chelsea Therapeutics International Ltd. (NASDAQ:CHTP)), which recently received Food and Drug Administration (FDA) accelerated approval of NORTHERATM (droxidopa) for the treatment of symptomatic neurogenic orthostatic hypotension (NOH), supports raising awareness for Rare Disease Day as a member of National Organization for Rare Disorders (NORD). Chelsea Therapeutics International Ltd. (NASDAQ:CHTP) stock advanced 13.98% and finished the last session at $5.95. The EPS of the stock remained -0.24. Company’s market capitalization is $466.68 million.