Largest U.S. electric grid expects surge in generation outages as cold snap continues

 The largest U.S. power grid operator, PJM Interconnection, said Tuesday it issued extra alerts to transmission and generation firms, and warned demand will exceed 147 GW, an all-time high for winter.

PJM, which serves more than 67M people in 13 states and the District of Columbia, issued Maximum Generation and Load Management alerts for companies to see if any maintenance or testing could be deferred or canceled to keep units online during the critical period.

Depending on temperatures, PJM said it could set a new all-time winter peak load on Tuesday, partly due to data center electricity needs, with predicted overall demand at 147.2 GW, above the record winter demand of 143.7 GW set in January a year ago.

The operator said peak demand could exceed 130 GW for seven straight days, a winter streak never before experienced.

Energy Secretary signed an order Monday authorizing PJM to direct data centers, factories and other large facilities to switch on back-up generators to reduce their burden on the grid.

Power grids so far have avoided rolling blackouts in the current cold snap, with most of the weekend's outages caused by storm damage and ice, but icy temperatures and dangerous wind chills are expected to continue into next weekend across much of the U.S., with forecasters at the U.S. National Weather Service calling it "the longest duration of cold in several decades."

https://www.msn.com/en-us/weather/topstories/largest-us-power-grid-issues-alerts-as-winter-peak-demand-could-hit-new-highs/ar-AA1V5kQt

Caribou Biosciences initiated with a Buy at Clear Street

 Clear Street initiated coverage of Caribou Biosciences (CRBU) with a Buy rating and $13 price target Caribou is developing a pipeline of off-the-shelf allogeneic CAR-T cell therapies, including lead asset vispa-cel, the analyst tells investors. Vispa-cel is preparing to enter a registrational Phase 3 study in second line or later large B-cell lymphoma, notes the analyst, whose model includes vispa-cel peak sales of $992M in 2040 and 30% odds of success as well as CB-011 peak sales of $734M in 2040 and 20% odds of success.

https://www.tipranks.com/news/the-fly/caribou-biosciences-initiated-with-a-buy-at-clear-street-thefly

Ultragenyx: Gene Therapy Shows Lasting Benefits, Slows Brain Decline In Fatal Childhood Disorder

 Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) on Tuesday announced new long‑term data from clinical studies evaluating UX111 (rebisufligene etisparvovec).

It is an investigational AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA), a fatal neurodegenerative lysosomal storage disorder.

What is MPS IIIA?

MPS IIIA is caused by mutations in the SGSH gene, leading to a deficiency of the enzyme heparan N-sulfatase, causing toxic accumulation of heparan sulfate in the brain and body.

It causes rapid cognitive decline, severe behavioral issues, and premature death.

Trial Data

The results demonstrate substantial and durable biomarker improvements and meaningful functional benefits compared with natural history.

Cognitive function, expressive and receptive communication, and fine and gross motor skills were measured using Bayley-III and compared to natural history data from untreated patients with reported rapid progressor phenotypes.

Children under two years of age or with earlier stage disease at the time of treatment (n=17) demonstrated a +23.2 point (p<0.0001) treatment effect in the mean Bayley-III cognitive raw score compared to natural history data during 24-60 months of age.

In addition to cognitive function, clinical improvements were also observed.

On a separate caregiver-reported outcome utilizing Vineland 3, there were comparable improvements in the communication, motor, and personal subdomains.

Eight children reached a 36‑month cognitive developmental age, enabling higher‑level testing—none of the natural‑history patients reached this milestone, the company said in a press release on Tuesday.

Patients with older age or having more advanced disease at the time of treatment (n=10) showed retention of functional abilities in at least one of three areas at the time of last assessment that exceeded typical decline patterns in untreated children with Sanfilippo syndrome Type A.

As of the September 2025 cutoff date, the median reduction in cerebrospinal fluid heparan sulfate (CSF-HS) exposure was 63.98% (p<0.001).

CSF-HS is a biomarker for monitoring neurodegenerative lysosomal storage disorders.

The majority of children treated (88.2% of younger patients and 81.5% of the overall efficacy set) achieved a 50% or greater reduction.

UX111 was well-tolerated, and the safety profile remains favorable.

What Next?

These longer-term data were included in the resubmitted Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for UX111.

The company anticipates up to a 6-month review period from the date of resubmission, with a PDUFA date expected in the third quarter of 2026.

https://www.benzinga.com/news/health-care/26/02/50345196/ultragenyx-says-gene-therapy-shows-lasting-benefits-slows-brain-decline-in-fatal-childhood-disorder

aTyr Sets FDA Type C Meeting to Discuss Efzofitimod Program in Pulmonary Sarcoidosis

aTyr Pharma (Nasdaq: ATYR) said the FDA accepted a Type C meeting request to discuss efzofitimod for pulmonary sarcoidosis, scheduled for mid-April 2026. The company will review Phase 3 EFZO-FIT results and report meeting minutes afterwards.

The Phase 3 study enrolled 268 patients; it did not meet the primary endpoint of corticosteroid dose reduction at week 48, but 5.0 mg/kg efzofitimod showed statistically significant benefits on multiple patient-reported outcomes and maintained lung function, with a safety profile consistent with prior trials.

https://www.stocktitan.net/news/ATYR/a-tyr-pharma-announces-scheduling-of-fda-type-c-meeting-to-discuss-yx144up3xwvc.html

Pricey healthcare stocks with halting momentum - low momentum and high valuation

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• AIM ImmunoTech Inc. (AIM), Quant Rating: 2.50.
• AirSculpt Technologies, Inc. ...
• Applied Therapeutics, Inc. ...
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• Lyra Therapeutics, Inc. ...
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https://seekingalpha.com/news/4546480-pricey-healthcare-stocks-with-halting-momentum---low-momentum-and-high-valuation

Altman Says OpenAI May Back Firms Using AI for Drug Discovery

 

OpenAI Chief Executive Officer Sam Altman said his company may consider investing in or subsidizing firms that make significant use of its AI technologies to discover new drugs or therapies, and possibly take a royalty in exchange

The AI developer could choose to cover the cost of using its artificial intelligence models in partnership with a drug company and then “get some royalty” from discoveries made by that firm, Altman said at Cisco Systems Inc.’s AI conference in San Francisco on Tuesday. The ChatGPT maker has no such partnerships at present, he said.

https://www.bloomberg.com/news/articles/2026-02-03/altman-says-openai-may-back-firms-using-ai-for-drug-discovery

Novo’s Next-Gen Obesity Drug Beats Wegovy on Blood Sugar Control in Phase III

 

While CagriSema bested Novo Nordisk’s Wegovy on blood sugar control in a late-stage trial, the next-gen weight loss drug still has not met the pharma’s 25% weight loss goal.

Novo Nordisk’s next-generation weight-loss drug CagriSema beat current blockbuster GLP-1 Wegovy at controlling blood sugar in a Phase III trial, while falling below the Danish pharma’s lofty 25% weight loss goal.

In the Phase III REIMAGINE 2 study, which enrolled more than 2,700 adults with type 2 diabetes, Novo tested a 2.4-mg dose of CagriSema delivered via a weekly subcutaneous injection. The company compared that against the same sized doses of semaglutide, the amylin analog cagrilintide alone and placebo. The trial also included a 1-mg dose arm for CagriSema compared with same doses of semaglutide, cagrilintide and placebo.

At 68 weeks, patients treated with 2.4-mg CagriSema saw a 1.91%-point reduction in HbA1c, a measure of average blood sugar over the past two to three months, according to a news release on Monday. HbA1c in patients on semaglutide dropped by 1.76%-points, whereas placebo comparators saw a 0.09%-point increase.

CagriSema’s treatment benefit was significantly stronger than semaglutide’s, Novo said.

Similarly, CagriSema elicited significantly greater weight-loss versus semaglutide. Patients on the investigational treatment lost 14.2% of their body weight at 68 weeks, as opposed to 10.2% in the semaglutide group. CagriSema had not yet shown a weight-loss plateau at the time of readout, Novo said.

Despite stronger weight reduction numbers of CagriSema, the drug still came far below Novo’s own target. In a June 23, 2025 note to investors, analysts at William Blair noted that the pharma had set a “lofty benchmark” of 25% placebo-adjusted weight-loss—a bar that the drug has yet to meet.

The closest Novo has so far come to hitting this target was in December 2024, when CagriSema in the Phase III REDEFINE 1 study elicited a 22.7% drop in body weight at 68 weeks, versus 2.3% in placebo. Topline data from a second study, dubbed REDEFINE 2 and released in March 2025, showed 15.7% weight-loss with CagriSema versus 3.1% in placebo.

Novo in December 2025 nevertheless pushed through with an FDA filing for CagriSema as a weight-loss medicine, proposing weekly use in patients with obesity or who are overweight with at least one related comorbidity. A similar application for diabetes is also planned, according to Monday’s news release, pending data from the REIMAGINE 1 and REDEFINE 3 trials. REIMAGINE 1 tests CagriSema in treating type 2 diabetes while REDEFINE 3 is for controlling cardiovascular disease.

CagriSema is a fixed-dose combination of the GLP-1 receptor agonist semaglutide and the long-acting amylin analog cagrilintide. If approved, the drug “could be the first amylin-based combination therapy” for weight-loss and type 2 diabetes on the market, Martin Holst Lange, Novo’s chief scientific officer, said in a statement on Monday.

Novo is also running a head to head study of CargiSema against Eli Lilly’s tirzepatide, with a readout expected this quarter.

https://www.biospace.com/drug-development/novos-next-gen-obesity-drug-beats-wegovy-on-blood-sugar-control-in-phase-iii