Ultragenyx Pharma (RARE), Kyowa Kirin Report Submission of Supplemental Biologics License Application to FDA for Crysvita (burosumab)

Ultragenyx Pharma (RARE), GeneTx Report Investigational New Drug Application Active for GTX-102 in Patients with Angelman Syndrome

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and GeneTx Biotherapeutics LLC, today announced that GeneTxs Investigational New Drug (IND) Application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS), filed with the U.S. Food and Drug Administration (FDA) is now active. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2020.

"The FDAs clearance of the IND to evaluate GTX-102 in patients with Angelman syndrome represents a significant milestone for the Angelman community, said Paula Evans, Chief Executive Officer at GeneTx. Assessing an... More

Ultragenyx Pharma (RARE) PT Raised to $53 at Citi

Citi analyst Yigal Nochomovitz raised the price target on Ultragenyx Pharma (NASDAQ: RARE) to $53.00 (from $45.00) while maintaining a Neutral rating.... More

Pre-Open 01/10: (SRNE) (SNX) (DERM) Higher (PTLA) (SIX) (SPWH) Lower (more...)

Today's Pre-Open Stock Movers

Sorrento Therapeutics, Inc. (NASDAQ: SRNE) 49.9% HIGHER; announced today that on January 9, 2020, it received a non-binding proposal from a private equity fund to acquire a majority or all of the issued and outstanding shares of the Company for up to $7.00 per share.

Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) 40.9% LOWER; For the fourth quarter, the Company expects Andexxa global net revenues to be approximately $28 million. For the full year 2019, the Company expects Andexxa global net revenues to be approximately $111 million. (***... More

Ultragenyx Pharma (RARE) PT Raised to $80 at SunTrust

SunTrust analyst Edward Nash raised the price target on Ultragenyx Pharma (NASDAQ: RARE) to $80.00 (from $75.00) while maintaining a Buy rating.... More

After-Hours Movers 01/09: (ACRS) (MRNA) (SNX) Higher; (PTLA) (SPWH) (URBN) Lower (more...)

Today's After-Hours Movers

Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) 38.8% LOWER; For the fourth quarter, the Company expects Andexxa global net revenues to be approximately $28 million. For the full year 2019, the Company expects Andexxa global net revenues to be approximately $111 million. (*** consensus is $134 million)

Sportsmans Warehouse (Nasdaq: SPWH) 16.4% LOWER; For the fourth quarter of fiscal year 2019, net sales are now expected to be in the range of $250 million to $254 million based on same stores sales in... More

Ultragenyx Pharma (RARE) Reports Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene Therapy

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced topline positive 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. In Cohort 3 (n=3), there were two confirmed female responders as well a third potential male responder who requires longer-term follow-up to confirm response status. In Cohort 2, one female patient has newly demonstrated a response starting at... More