Ultragenyx Shares Increase 30% on Results from Phase 1/2 OTC Deficiency Study
Source: Streetwise Reports
January 13, 2020 (Investorideas.com Newswire) Rare disease biopharmaceutical company Ultragenyx Pharmaceutical's shares traded 30% higher after the firm reported positive topline results from Cohorts 2 and 3 in its Phase 1/2 study of DTX301 gene therapy in Ornithine Transcarbamylase Deficiency.
After U.S. markets closed yesterday afternoon, Ultragenyx Pharmaceutical Inc. (RARE:NASDAQ), a biopharmaceutical company engaged in the development of novel products for rare and ultra-rare diseases, announced "positive topline safety and efficacy data from Cohort 3 and longer-term data from Cohort 2 of the ongoing Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency."
The company advised that the objective of the Phase 1/2 study of DTX301 is to "evaluate the change in the rate of ureagenesis, ammonia levels, neurocognitive assessment, biomarkers, and safety of DTX301 in patients with OTC deficiency." The firm explained that OTC deficiency is the most common urea cycle disorder that affects more than 10,000 individuals worldwide caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. The company indicated that to date there have been no infusion-related adverse events and no treatment-related serious adverse events reported in the study.
Ultragenyx indicated that DTX301 has been granted Orphan Drug Designation in the U.S. and Europe and is "an investigational AAV type 8 gene therapy designed to deliver stable expression and activity of OTC following a single intravenous infusion, which has been shown in preclinical studies to normalize levels of urinary orotic acid, a marker of ammonia metabolism."
Eric Crombez, M.D., chief medical officer of the Ultragenyx Gene Therapy development unit, commented, "We are encouraged to see a more uniform response at the higher doses including three female responders. To date, three patients in the study have discontinued alternate pathway medication and liberalized their diets while remaining clinically and metabolically stable...We are moving to prophylactic steroid use in the next cohort as we believe this could further enhance the level and consistency of expression that we have demonstrated so far.".
Ultragenyx is headquartered in Novato, Calif., and is a biopharmaceutical company specializing in developing novel products for the treatment of serious rare and ultra-rare genetic diseases. The company aims at addressing diseases with high unmet medical need with clear biology for treatment for which there are no presently approved therapies for treating the underlying disease.
Ultragenyx Pharmaceutical started the day with a market capitalization of approximately $2.5 billion with about 57.77 million outstanding shares and a short interest of around 10.6%. RARE shares opened nearly 12% higher today at $48.91 (+$5.18, +11.85%) over yesterday's $43.73 closing price. The stock has traded today between $44.84 and $58.54 per share and is currently trading at $58.44 (+$14.71, +33.64%).
1) Stephen Hytha compiled this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. He or members of his household own securities of the following companies mentioned in the article: None. He or members of his household are paid by the following companies mentioned in this article: None.
2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees.
3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.
5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the interview or the decision to write an article until three business days after the publication of the interview or article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.
6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.
This news is published on the Investorideas.com Newswire - a global digital news source for investors and business leaders
Disclaimer/Disclosure: Investorideas.com is a digital publisher of third party sourced news, articles and equity research as well as creates original content, including video, interviews and articles. Original content created by investorideas is protected by copyright laws other than syndication rights. Our site does not make recommendations for purchases or sale of stocks, services or products. Nothing on our sites should be construed as an offer or solicitation to buy or sell products or securities. All investing involves risk and possible losses. This site is currently compensated for news publication and distribution, social media and marketing, content creation and more. Disclosure is posted for each compensated news release, content published /created if required but otherwise the news was not compensated for and was published for the sole interest of our readers and followers. Contact management and IR of each company directly regarding specific questions.
More disclaimer info: https://www.investorideas.com/About/Disclaimer.asp Learn more about publishing your news release and our other news services on the Investorideas.com newswire https://www.investorideas.com/News-Upload/ and tickertagstocknews.com