Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)

CAPS Rating: 4 out of 5

Caps

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All Players

30 Outperform
3 Underperform
 

All-Star Players

9 Outperform
0 Underperform
 

Wall Street

1 Outperform
0 Underperform
 

Top RARE Bull/Bear Pitches

The best Bull and Bear pitches based on recency and number of recommendations.

warrenout (98.49)
Submitted July 18, 2015

Um, yeah, I know. Right at the peak of the peak. I mean, it's bound to fall. Yeah but.Yeah?Okay. Go for it. It's only a thumb.This is a ten-bagger for sure?Yup. And we're gonna need bags, right?Yup. As long at their not plastic and you live in an… More

OklaBoston (52.06)
Submitted March 28, 2014

Insiders have never payed more than $21 for shares of this company which appears to have gone private a few years ago and is returning to the public sphere. Paying twice as much as directors for any stock however long it may have been public makes no… More

RARE VS S&P 500 (SPY)

Recent Community Commentary

Read the most recent pitches from players about RARE.

Recs

0
Member Avatar Bigsef77 (71.49) Submitted: 8/29/2018 1:49:49 PM : Outperform Start Price: $54.94 RARE Score: +49.58

Ultragenyx Announces FDA Accepts Proposal to Submit an NDA for UX007 for the Treatment of Long-Chain Fatty Acid Oxidation Disorders
GlobeNewswire•August 29, 2018
NOVATO, Calif., Aug. 29, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted Ultragenyx’s most recent proposal to submit a New Drug Application (NDA) for UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) based on existing data. Further details regarding timing will be forthcoming following a pre-NDA meeting, which is being scheduled for the second half of 2018.

“We appreciate FDA’s review of multiple data submissions and collaboration with us to develop a path for an early filing, and it is our commitment to get this important potential treatment to patients with this serious disease as quickly as possible,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We will meet with the FDA to discuss the details of the NDA submission and, if approved, appropriate post-approval commitments to further evaluate long-term outcomes of UX007 in patients with LC-FAOD.”

The data submitted to the FDA for evaluation included the recently published 78-week Phase 2 study results in 29 patients, a now published retrospective medical record review of 20 patients, and 56 emergency IND cardiomyopathy and other patients. In addition, these data were supported by results of a published randomized controlled investigator study of 32 patients showing an effect of triheptanoin on cardiac function. In the 78-week sponsored Phase 2 study, the data showed a 48.1 percent reduction in the mean annualized rate of major clinical events (MCEs) and a 50.6 percent reduction in the median annualized rate of MCEs after 78 weeks of treatment with UX007 compared to an annualized rate of MCEs in the 18 months prior to treatment with UX007. There was also a 50.3 percent reduction in the mean annualized duration of MCEs and a 76.7 percent reduction in the median annualized duration of MCEs following 78 weeks of UX007 treatment. The safety profile was consistent with what has been previously observed with UX007.

In the EU, Ultragenyx will discuss these data with the European Medicines Agency (EMA) and expects to have an update in the second half of 2018.

Recs

0
Member Avatar PViddy (< 20) Submitted: 9/28/2014 8:01:15 AM : Underperform Start Price: $56.41 RARE Score: -1.63

Red thumbing 2014 IPOs. Most of these stocks are over-hyped and overvalued.

This biotech company has no revenues yet, and sports $1.8 billion market cap.

Leaderboard

Find the members with the highest scoring picks in RARE.

Score Leader

RonaldSpeth1

RonaldSpeth1 (59.50) Score: +119.44

The Score Leader is the player with the highest score across all their picks in RARE.

Top
Pick
Member Name Member
Rating
Start
Date
Call Time
Frame
Start
Price
Stock
Gain
Index
Gain
Score Commentary
stainsolution 36.76 12/18/2017 Outperform 5Y $45.00 +88.60% +8.91% +79.69 0 Comment
StockElections 28.74 5/15/2014 Outperform 5Y $36.00 +135.75% +56.42% +79.33 0 Comment
arnoldrkelly3515 69.64 11/20/2017 Outperform 5Y $48.11 +76.41% +13.11% +63.30 0 Comment
SaintCroix 99.22 4/10/2018 Outperform 5Y $51.40 +65.12% +10.49% +54.63 0 Comment
efarev 98.57 4/23/2014 Outperform 5Y $41.15 +106.25% +55.47% +50.77 0 Comment
Bigsef77 71.49 3/13/2018 7/31/2018 Outperform 1Y $54.94 +54.48% +4.90% +49.58 1 Comment
icebox40 29.25 12/18/2014 Outperform 1Y $44.91 +88.98% +42.62% +46.36 0 Comment
portefeuille2 99.26 6/18/2014 Outperform 5Y $47.98 +76.89% +49.97% +26.92 0 Comment
portefeuille 98.48 6/18/2014 Outperform 5Y $47.98 +76.89% +49.97% +26.92 0 Comment
Traced < 20 5/9/2017 Outperform 5Y $60.62 +40.00% +21.68% +18.32 0 Comment

Wall Street

See what the Wall Street professionals think, according to their public statements and filings.

Player Name Player
Rating
Start
Date
Call Time
Frame
Start
Price
Stock
Gain
S&P
Gain
Score End Date Commentary
TrackCanaccord 68.83 2/25/2014 Outperform NS $60.00 +41.45% +57.81% -16.36 0 Comment

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