Join today and have your say! It’s FREE!

Become a member today, It's free!

We will not release or resell your information to third parties without your permission.
Please Try Again
{{ error }}
By providing my email, I consent to receiving investment related electronic messages from Stockhouse.


Sign In

Please Try Again
{{ error }}
Password Hint : {{passwordHint}}
Forgot Password?


Please Try Again {{ error }}

Send my password

An email was sent with password retrieval instructions. Please go to the link in the email message to retrieve your password.

Become a member today, It's free!

We will not release or resell your information to third parties without your permission.

Ultragenyx Shares Increase 30% on Results from Phase 1/2 OTC Deficiency Study

Streetwise Reports, Streetwise Reports
0 Comments| January 13, 2020

{{labelSign}}  Favorites

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.
4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.
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.

{{labelSign}}  Favorites


No comments yet. Be first to comment!

Leave a Comment

You must be logged in to be able to post a comment.

Get the latest news and updates from Stockhouse on social media


Featured Company