RARE News & Events

Catch up on the weekend's top five stories with this list compiled by The Fly: 1) KKR (KKR) is working on a sale of CoolIT Systems, hoping to fetch a price tag in excess of $3B, Oliver Barnes and Antoine Gara of The Financial Times reports, citing people familiar with the matter. This is a massive increase from the unit's $270M valuation when KKR bought a majority stake in 2023. 2) Dr. Vinay Prasad, who issued several controversial decisions, including having refused to accept Moderna's (MRNA) application for a new mRNA flu vaccine and refused an application for a Huntington's drug's review from UniQure (QURE), is leaving the agency at the end of April, reported The New York Times' Christina Jewett on Friday evening, citing a Health and Human Services spokesman. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks that could be impacted by the news include Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Avidity Biosciences (RNA), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Moderna (MRNA), Novavax (NVAX) and Lexeo Therapeutics (LXEO). 3) Plug Power (PLUG) plans to offer hydrogen electricity, as much as 250 megawatts, in an auction President Donald Trump is eager to make happen later this year, Will Wade and Naureen Malik of Bloomberg reports, citing chairman Andy Marsh. PJM Interconnection, which is facing power shortages due to surging consumption by data centers to power AI, has been urged by the Trump administration to hold an emergency auction to bolster supplies. 4) Prudential (PRU) disclosed yesterday that on March 6, the Prudential Gibraltar Financial Life Insurance Co., a subsidiary of Prudential Financial, issued a press release in Japan reporting instances of the unauthorized removal of information by certain employees of PGFL seconded to financial institutions in Japan. The investigation confirmed that 11 employees in total removed 379 instances of information at seven contracted agencies. The removed information pertained to operational matters such as sales performance at the assigned agencies. "After confirming the content of all cases with the agencies, no issues were identified that would raise concerns under the Unfair Competition Prevention Act, nor was there any inappropriate removal of contract information," Prudential Gibraltar Financial said. 5) Activist investor Starboard Value has built a stake in Lamb Weston (LW) and has been pushing to company to speed up improvements and cost cutting to boost stock performance, Lauren Thomas of The Wall Street Journal reports, citing people familiar with the matter. Starboard is now one of the largest shareholders in the company, the sources added.

Dr. Vinay Prasad, who issued several controversial decisions, including having refused to accept Moderna's (MRNA) application for a new mRNA flu vaccine and refused an application for a Huntington's drug's review from UniQure (QURE), is leaving the agency at the end of April, reported The New York Times' Christina Jewett on Friday evening, citing a Health and Human Services spokesman. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks that could be impacted by the news include Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Avidity Biosciences (RNA), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Moderna (MRNA), Novavax (NVAX) and Lexeo Therapeutics (LXEO).

Ultragenyx Pharmaceutical announced the U.S. Food and Drug Administration, FDA, has accepted for review the Biologics License Application, BLA, seeking approval of DTX401 AAV gene therapy for the treatment of Glycogen Storage Disease Type Ia. The FDA granted the BLA Priority Review and assigned a Prescription Drug User Fee Act, PDUFA, action date of August 23, 2026. "Current dietary approaches to managing GSDIa place an extraordinary burden on individuals and families while still leaving patients with significant medical needs, including the risk of potentially life-threatening episodes of acute hypoglycemia and accumulation of long-term complications over their lifetime," said Eric Crombez, M.D., chief medical officer at Ultragenyx. "If approved, DTX401 would be the first treatment to address the disease at its root cause. We appreciate the FDA's timely acceptance of the BLA and will continue to work with the Agency throughout its review process."