Ultragenyx Pharm Stock Price, News & Analysis

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on developing therapies for rare genetic diseases, has announced its participation in three upcoming investor conferences in September 2024:

• Morgan Stanley 22nd Annual Global Healthcare Conference on September 4
• Cantor Global Healthcare Conference on September 17
• Bank of America Global Healthcare Conference on September 18

Key executives, including CEO Emil Kakkis, CFO Howard Horn, and CMO Eric Crombez, will participate in fireside chats and host one-on-one meetings. Live and archived webcasts of the fireside chats will be available on the company's investor relations website. Ultragenyx is committed to developing novel products for serious rare and ultrarare genetic diseases, with a diverse portfolio of approved therapies and product candidates.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultrarare disease therapies, has granted 37,975 restricted stock units to 12 newly hired non-executive officers. The awards, approved by the company's compensation committee, were issued under the Ultragenyx Employment Inducement Plan on August 16, 2024. This grant aligns with Nasdaq Listing Rule 5635(c)(4) and serves as an employment inducement. The restricted stock units have a four-year vesting schedule, with 25% of the underlying shares vesting annually, contingent on continuous employment with Ultragenyx.

Amlogenyx Inc., a subsidiary of Ultragenyx Pharmaceutical (NASDAQ: RARE), has successfully closed a $14 million seed funding round led by GordonMD® Global Investments LP. The company is developing novel gene therapies for Alzheimer's disease and other amyloid diseases. Amlogenyx's first product candidate is a protease developed in collaboration with St. Jude Children's Research Hospital, which has shown promising results in breaking down Aβ42, a protein believed to be responsible for Alzheimer's effects.

Dr. Craig Gordon, Founder and CIO of GordonMD®, expressed excitement about the collaboration and the potential of Amlogenyx's Alzheimer's disease therapeutic. The funding round also included participation from Ultragenyx and associated investors, demonstrating strong support for Amlogenyx's innovative approach to treating amyloid diseases.

Ultragenyx reported second quarter 2024 financial results with total revenue of $147 million, up 36% YoY. Key revenue drivers were Crysvita ($114 million, +37%) and Dojolvi ($19 million, +17%). The company raised its 2024 revenue guidance to $530-$550 million from $500-$530 million.

Ultragenyx also disclosed significant clinical advancements including positive data from Phase 1/2 study in Angelman syndrome, Phase 2/3 study in osteogenesis imperfecta, and Phase 3 study in GSDIa. Phase 3 studies for Angelman syndrome and UX111 for Sanfilippo syndrome type A are on track to start by end of 2024.

The company reported a net loss of $132 million, down from $160 million YoY. Operating expenses were $263 million, including $39 million in non-cash stock-based compensation. Cash reserves stood at $874 million as of June 30, 2024.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company specializing in novel products for rare and ultrarare genetic diseases, has announced a conference call scheduled for August 1, 2024, at 5:00 p.m. ET. The call will cover the company's financial results and corporate update for the second quarter of 2024, ending June 30. Investors and interested parties can access the live webcast and replay through the company's investor relations website. The replay will remain available for three months following the call, providing extended access to the information presented.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) will present updates on its GTX-102 Angelman syndrome program at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium on July 24, 2024. The presentations will include:

1. A review of positive interim Phase 1/2 results previously disclosed at the American Academy of Neurology Meeting in April.
2. An update on plans to initiate a Phase 3 pivotal trial by the end of 2024.
3. Insights on patient progress, with continued development of new skills across multiple domains and no new serious adverse events reported.

Dr. Eric Crombez, Chief Medical Officer at Ultragenyx, expressed enthusiasm about working with the ASF and the broader community as they prepare for a global randomized study. The presentations will be livestreamed on the ASF website.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultrarare diseases, has announced the grant of 19,875 restricted stock units to 14 newly hired non-executive officers. The awards, approved by the company's compensation committee, were granted under the Ultragenyx Employment Inducement Plan on July 16, 2024. This move aligns with Nasdaq Listing Rule 5635(c)(4), serving as an inducement for new employees joining Ultragenyx.

The restricted stock units have a four-year vesting period, with 25% of the underlying shares vesting annually on the grant date anniversary. Continuous employment with Ultragenyx is required for vesting. This grant demonstrates the company's commitment to attracting and retaining talent in the competitive biopharmaceutical industry.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced a successful end-of-Phase 2 meeting with the FDA for its GTX-102 Angelman syndrome program. The company achieved alignment on the Phase 3 study design, including the primary endpoint of Bayley-4 cognition and key secondary endpoint of Multi-Domain Responder Index (MDRI). The Phase 3 study is set to begin by the end of 2024, involving a global, randomized, double-blind, sham-controlled trial with approximately 120 patients with Angelman syndrome caused by full maternal UBE3A gene deletion. The study will have a 48-week primary efficacy analysis period. Ultragenyx also plans to initiate an open-label study for patients with other Angelman syndrome genotypes and age groups.

Ultragenyx Pharmaceutical announced the grant of 45,525 restricted stock units (RSUs) to 26 newly hired non-executive employees.

The awards, approved by the compensation committee, were granted under the company's Employment Inducement Plan on June 16, 2024, as per Nasdaq Listing Rule 5635(c)(4).

The RSUs will vest over four years, with 25% vesting each year on the anniversary of the grant date, contingent on the employees' continued employment.