NOVATO, Calif., April 28, 2020 -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products.

Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the BofA Securities Global Health Care Conference on Thursday, May 14, 2020 at 10:20 AM ET. The live and archived webcast of the presentation will be accessible from the company’s website at http://ir.ultragenyx.com/events.cfm. Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases.

Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced positive initial data from the confirmatory third cohort and longer-term data from the first two cohorts of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus (AAV) based gene therapy for the treatment of glycogen storage disease type Ia (GSDIa). All three patients in Cohort 3 responded to therapy and demonstrated more rapid reductions in cornstarch requirements compared to the first two cohorts.

GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to GTX-102 for the treatment of Angelman syndrome (AS). GTX-102 is an investigational antisense oligonucleotide currently being evaluated in a Phase 1/2 study for the treatment of AS. “GeneTx is committed to improving the quality of life of individuals living with Angelman syndrome,” said Paula Evans, Chief Executive Officer, GeneTx.

Ultragenyx (RARE) delivered earnings and revenue surprises of -27.50% and -3.70%, respectively, for the quarter ended March 2020. Do the numbers hold clues to what lies ahead for the stock?

Q1 2020 Ultragenyx Pharmaceutical Inc Earnings Call

Ultragenyx (RARE) reports wider-than-expected first-quarter 2020 loss.

Six of nine patients in the study have responded to the gene therapy (three female, three male), including all three patients in Cohort 3 who are now confirmed responders. The three previously disclosed complete responders, who have discontinued all ammonia scavengers and liberalized their diet, remain clinically and metabolically stable after longer-term follow-up.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) last week reported its latest quarterly results, which makes it a good...

Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Wednesday, May 6, 2020 at 5pm ET to discuss first quarter 2020 financial results and provide a corporate update. Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

Ultragenyx Pharmaceutical, Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases and Arcturus Therapeutics Holdings Inc. (ARCT), a leading messenger RNA medicines company, today announced that Ultragenyx has exercised its option to purchase 600,000 shares of Arcturus common stock at $16.00 per share. “We are encouraged by the advancement of Arcturus’s broad nucleic acid platform across multiple therapeutic areas, including their self-replicating mRNA-based COVID-19 vaccine candidate,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx.

Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced a strategic partnership and non-exclusive license and technology access agreement with Daiichi Sankyo Company, Limited for Ultragenyx’s proprietary AAV-based gene therapy manufacturing technologies. Ultragenyx’s HeLa producer cell line (PCL) platform enables large commercial-scale AAV-based gene therapy product manufacturing that is intended to be highly reproducible, more consistent, and less expensive than other gene therapy manufacturing platforms.

As of late, it has definitely been a great time to be an investor Ultragenyx Pharmaceutical

Ultragenyx (RARE) is seeing favorable earnings estimate revision activity and has a positive Zacks Earnings ESP heading into earnings season.

RARE earnings call for the period ending March 31, 2020.

Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the Jefferies Healthcare Conference on Wednesday, June 3, 2020 at 2 PM ET. Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will hold a virtual presentation at the Goldman Sachs 41st Annual Global Healthcare Conference on Tuesday, June 9, 2020 at 3:50 PM ET.

Biopharma Ultragenyx (RARE), which focuses on rare and ultra-rare diseases, has announced a key strategic partnership with Japanese global pharma Daiichi Sankyo.The deal grants Daiichi a non-exclusive license to RARE’s HeLa PCL and HEK293 mammalian cell AAV manufacturing system. Ultragenyx’s HeLa producer cell line (PCL) platform is intended to enable large scale AAV-based gene therapy product manufacturing that is highly reproducible, more consistent, and less expensive than other gene therapy manufacturing platforms.In return, RARE will receive $125M cash and $75M equity (at $60/ share) upfront, $25M upon completion of tech transfer, and single-digit royalties on net sales of future products. The company is currently trading at $44/ share.Crucially, RARE retains an option to co-develop and co-commercialize Daiichi’s rare disease candidates and retains the exclusive right to use its manufacturing system for current and future targets.“We view this last night’s deal with Daiichi Sankyo for non-exclusive rights to RARE’s mammalian AAV manufacturing system as a positive by further validating the company’s gene therapy platform and helping to further bolster the balance sheet” cheered JP Morgan analyst Cory Kasimov on April 1.According to the analyst’s estimates, the $200 million upfront extends the company’s cash runway all the way into 2022, taking any near-term dilutive financing off the table. Plus he is reassured that the non-exclusive nature of the deal does not pose a competitive risk to RARE’s current or future targets.As a result, the analyst reiterated his buy rating on the stock with a $76 price target (71% upside potential). This isn’t the only analyst singing RARE’s praises- overall TipRanks shows that Ultragenyx holds a bullish Strong Buy consensus from the Street with a $72 average analyst price target. (See RARE’s stock analysis on TipRanks)Related News: Battered Cruise Operator Carnival Seeks $6 Billion To Deal With Covid-19 Fallout Verint Systems Misses Profit Estimates, Sees Growing Cloud Demand Momentum Facebook’s User Engagement and Business Interaction Make It Unstoppable, Says Top Analyst More recent articles from Smarter Analyst: * Amazon to Put New Online Grocery Shoppers on Wait List to Meet Surging Demand * Pfizer Joins Virus Vaccine Hunt in Partnership With German Drugmaker * Gilead’s Experimental Coronavirus Drug Shows “Hopeful” Signs in Small Group * KB Home Declares Second-Quarter Cash Dividend

Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended March 31, 2020 and maintained its full year 2020 financial guidance. The FDA reviews for Crysvita for TIO and UX007 for LC-FAOD are on track to be completed in the coming months.

Alkermes (ALKS) beats earnings and sales estimates for the first quarter of 2020. The company withdraws its previously provided outlook for 2020 amid uncertainty related to coronavirus.

Ultragenyx (RARE) possesses the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations.