Agios Pharmaceuticals Inc. reported top-line data from its 52-week Rise Up trial testing oral pyruvate kinase activator mitapivat in sickle cell disease (SCD), which hit statistical significance on one of two primary endpoints and two of three key secondary endpoints. While the Street focused on the missed goals, sending company shares (NASDAQ:AGIO) down nearly 50% at midday, company officials touted the positive findings, particularly those in the subgroup of hemoglobin responders, while noting the dearth of effective and accessible treatments in SCD, and disclosed plans to meet with the U.S. FDA early in 2026 to discuss a potential supplemental NDA filing. In the meantime, Agios is looking ahead to a Dec. 7 PDUFA date by which the FDA is expected to decide whether to expand use of mitapivat, already approved as Pyrukynd to treat hemolytic anemia, in patients with thalassemia.

Rhetoric heats up in partisan Rx price-lowering war

In a verbal war over who can deliver the lowest drug prices in the U.S., several Senate Democrats urged President Donald Trump this week to immediately release the list of second-round Medicare-negotiated drug prices, instead of doing “ambiguous” and “opaque” pricing deals with individual biopharma companies. (The deadline for the release of the 2027 maximum fair prices isn’t until Nov. 30 under the timetable established last year by the Biden administration.) Bristling to Health and Human Services Secretary Robert Kennedy’s suggestion last month that the discounts offered under Trump’s voluntary agreements with industry outperform those seen with Medicare drug price negotiations, the lawmakers said, “We are skeptical.” In a letter to Trump, led by Sens. Ron Wyden, D-Ore., and Chuck Schumer, D-N.Y., the senators challenged the president to release the latest negotiated prices to “lay bare the facts: Medicare drug price negotiations meaningfully lowers [sic] the price of prescription drugs, unlike your opaque Oval Office announcements.”

Protara phase II positive in lymphatic malformations

Protara Therapeutics Inc. disclosed interim results from its ongoing phase II open-label, 29-subject Starborn-1 trial testing intracystic injection of cell-based immunopotentiator TARA-002 in pediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs). Eighty percent of patients who completed treatment and 100% of patients who completed the eight-week response assessment achieved clinical success, Protara said. Formed during pregnancy, LMs are rare abnormalities of the lymphatic system that cause cyst-like masses. Shares of New York-based Protara (NASDAQ:TARA) were trading at $6.25, down 50 cents.

UK study reveals inconsistencies in global microbiome research

The U.K. Medicines and Healthcare products Regulatory Agency is calling for unified standards to harmonize microbiome research, after revealing major inconsistencies in the results when labs around the world analyzed identical reference samples of gut bacteria. Across 23 labs in 11 countries and four continents, species identification varied from 63% to 100% accuracy, meaning some labs failed to name a third of bacteria in a sample. In some cases, labs incorrectly identified bacteria that were not in a sample, with false positives across all the labs ranging from 0% to 41%.

SfN 2025: How ‘latent’ sex differences can trip up drug discovery

At the 2025 annual meeting of the Society for Neuroscience in San Diego this week, Catherine Woolley’s plenary lecture was an unusual combination of debunking and affirming the importance of sex differences in the brain. She recounted that in her conversations with both research scientists and the general public, there are recurrent themes of structural and hormonal differences between the brains of men and women, along with speculations about how such differences might be related to differences between men and women in society. But Woolley, who is the William Deering Chair in Biological Sciences and a professor of neurobiology and neurology at Northwestern University, suggested that such structural and hormonal differences are less dichotomous, and less important, than molecular-level differences. “I have come to think that sex differences in the brain could be very important, less for figuring out how our minds work, and more for ensuring that we maximize the benefits of science for medicine,” she told the audience at her plenary talk.

Tempest gains CAR Ts for 65% of the company; stock drops

Tempest Therapeutics Inc. entered into definitive agreements approved by its board to acquire certain dual-targeting CAR T programs from Factor Bioscience Inc. and its affiliates in an all-stock transaction expected to close in early 2026. The deal, under which Brisbane, Calif.-based Tempest will issue 8.27 million shares to an affiliate of Cambridge, Mass.-based Factor, and immediately exercisable warrants (one warrant per share) with an initial exercise price of $18.48 each to Tempest stockholders, is subject to stockholder approval. Upon closing, Tempest shareholders will own about 35%, while Factor and its affiliates will own 65%. Tempest’s stock (NASDAQ:TPST) fell 46.6%, or $4.30, to $4.93 in early trading Nov. 19.

GSK, LTZ partner to develop myeloid cell engagers

Immunotherapy-focused biotech company LTZ Therapetics Inc. and GSK plc are partnering to develop up to four potential first-in-class myeloid cell engagers using LTZ’s immune-engager platform to target blood cancers and solid tumors. Under the deal terms, LTZ will receive an up-front payment of $50 million and is eligible to receive undisclosed success-based preclinical, clinical, regulatory and commercial milestone payments, plus sales-based royalties. In exchange, GSK gains an exclusive option to license worldwide development and commercial rights for these preclinical therapies.

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