In one fell swoop yesterday, a federal judge stayed all the votes taken by Health and Human Services Secretary Robert Kennedy’s reconstituted Advisory Committee on Immunization Practices (ACIP), along with 13 appointments to the committee and the CDC’s January memo that revised the U.S. childhood immunization schedule. The injunction will be in place until Judge Brian Murphy, of the U.S. District Court for the District of Massachusetts, rules on the merits of a lawsuit brought by a coalition of medical organizations – or unless it’s overturned on appeal. In any case, the ACIP meeting that had been set for tomorrow isn’t happening.
Phase III substudy shortfalls with Rhythm’s Imcivree for obesity leave shares unfazed
Rhythm Pharmaceuticals Inc. disclosed that all four substudies of the phase III Emanate study with Imcivree (setmelanotide) missed their primary endpoints. The firm was testing Imcivree in rare genetic forms of obesity caused by heterozygous variants in the melanocortin-4 receptor pathway. Imcivree, an agonist in the pathway, was first cleared by the U.S. FDA in November 2020. Shares of Rhythm (NASDAQ:RYTM) were trading at $90.33, down 18 cents.
Pharma USA: The complicated journey of launching rare disease drugs
Rare disease drug development companies battle with economic challenges and small patient populations, but new technology alongside a human connection are helping researchers and marketers to identify patients, educate physicians and build networks. At Pharma USA, a Reuters Events meeting in Philadelphia March 16-17, executives from Astrazeneca plc and UCB SA discussed their successes with rare disease launches, presenting their playbooks for building trust and driving adoption among patients. Astrazeneca gained U.S. FDA approval in 2023 for Wainua (eplontersen) with partner Ionis Pharmaceuticals Inc. for adults with the rare nerve disease polyneuropathy of hereditary transthyretin-mediated amyloidosis, while UCB won its nod for Kygevvi (doxecitine and doxribtimine) last November as the first treatment for the ultrarare mitochondrial disease thymidine kinase 2 deficiency. “For this disease, the primary endpoint is survival,” Kim Moran, senior vice president and head of UCB’s U.S. rare disease unit, told attendees. “That kind of ups the ante of what you’re doing every day.”
Roche secures CE mark for blood test to identify ApoE4 carriers
Roche Holding AG received CE Mark approval for its Elecsys Apolipoprotein E4 (ApoE4) biomarker test, an in vitro diagnostic immunoassay to identify the ApoE4 gene variant from a blood sample. With APOE4 considered as the strongest genetic risk factor for developing Alzheimer’s disease, the test provides a fast, reliable way to determine whether an individual carries the genetic variant without the need for DNA‑based genotyping.
Structure’s oral GLP-1 data mark bullish year for obesity pills
The GLP-1 fight has moved from injectables to pills, and Structure Therapeutics Inc. is in the ring with phase III-ready aleniglipron. According to the biotech, its oral once-daily glucagon-like peptide-1 (GLP-1) drug candidate, aleniglipron, demonstrated an absolute weight loss of “up to 39 pounds” in the 44-week Access II trial. “Clearly, [aleniglipron’s] efficacy stands out,” Structure CEO and Director Raymond Stevens told investors. “This is the most efficacious oral GLP-1 out there from the data that we’ve shown now.”
R1’s $77.5M series A to fund CKD trial of oral phosphate inhibitor
Newco R1 Therapeutics Inc. has launched with an oversubscribed $77.5 million series A, providing the means to take AP-306, a potentially first-in-class hyperphosphatemia therapy through phase IIb development in patients with chronic kidney disease. The company has acquired ex-China rights to AP-306 from Alebund Pharmaceutical Ltd., which earlier completed a phase IIa study demonstrating a significant reduction in serum phosphate levels in patients treated with the oral phosphate transporter inhibitor. Rather than passively binding phosphate, as is the case for currently approved therapies that have been the standard of care for decades, AP-306 blocks the movement of phosphate out of the gut through three different transporters in the gastrointestinal tract.
Mestag raises $40M to take fibroblast program into phase I cancer trial
Mestag Therapeutics Ltd. has raised a $40 million round from existing investors as it completes final preparations for its lead program MST-0312 to enter clinical development in mid-2026. MST-0312 is a bispecific antibody aimed at the lymphotoxin-β receptor, which is essential for the development of lymphoid tissues. Engagement with the target induces the formation of tertiary lymphoid structures (TLS) in tumors that contain B cells, T cells and dendritic cells, and of high endothelial venules, that traffic lymphocytes from the bloodstream into the TLS.
Australia publishes first clinical practice guideline for psychedelics
Australia has released the first clinical practice guideline for the appropriate use of methylenedioxymethamphetamine-assisted psychotherapy for post-traumatic stress disorder, but clinicians caution that evidence, infrastructure and access remain works in progress.
ESMO TAT: Early-phase trials leading the way
In the opening sessions of this year’s ESMO Targeted Anticancer Therapies Congress, Elena Garralda, director of the Molecular Therapeutics Research Unit at Vall d’Hebron Institute of Oncology in Barcelona, described ESMO TAT as “the house of phase I,” a fitting label for a meeting centered on translational research and early drug development, where first-in-human data and new trial designs help shape the next generation of cancer therapies.
Blockbuster BD-Waters deal lifts med-tech M&A in early 2026
Med-tech M&A activity totaled $18.2 billion in January and February 2026, a 66% increase from the $10.98 billion recorded in the first two months of 2025. Becton, Dickinson and Co.‘s previously announced spin-off and merger of its Biosciences & Diagnostic Solutions business with Waters Corp. was the largest med-tech M&A deal to close in February, valued at $17.5 billion.
Aldeyra collects third CRL for reproxalap in dry eye disease
It’s déjà vu all over again for Aldeyra Therapeutics Inc., which disclosed its third complete response letter (CRL) for dry eye disease candidate reproxalap, with the U.S. FDA citing a lack of substantial and consistent evidence of efficacy. The difference this time is that the agency has recommended that reasons for the inconsistent findings be explored rather than asking for additional trials. Aldeyra intends to request a type A meeting to understand further what is needed for potential approval. Shares (NASDAQ:ALDX) were down 68% at midday.