In one fell swoop March 16, a U.S. federal judge stayed the CDC’s January memo revising the childhood vaccine schedule and the Advisory Committee on Immunization Practices (ACIP) as reconstituted by Health and Human Services Secretary Robert Kennedy, along with everything that committee has done since early June. Read More
Low expectations for Rhythm Pharmaceuticals Inc.’s phase III study dubbed Emanate with Imcivree (setmelanotide) in a handful of rare genetic obesities meant hardly any share price consequence, and the stock (NASDAQ:RYTM) closed March 17 at $87.68, down $2.83. “We’re not going to file [for approval] on any of these,” CEO David Meeker said, but the latest findings together represent “a solid building block going forward,” he added. Read More
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 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. Read More
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. Read More
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 Bay Area 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. Read More
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. Read More
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 (LTBR), which is essential for the development of lymphoid tissues. Read More
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. Read More
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. Read More
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. Read More
Biopharma and med-tech companies raising money in public or private financings, including: Acumen, Alto, Biovie, JATT II, Option, Outlook, Sharp, Unnatural Products, Zenas. Read More
Clinical updates for biopharma and med tech, including data readouts and publications: Alessa, Almirall, Bayer, Biomarin, Biomea, Cytomx, Eledon, Eli Lilly, J&J, Nasus, Relay, Sarepta, Structure. Read More
Biopharma and med-tech happenings, including deals and partnerships, and other news in brief: Arrotex, Caris, Cipla, Commave, Fennec, George, PRG S&T, Senseonics, Sentynl, Zevra, Zydus. Read More
Regulatory snapshots for biopharma and med tech, including global submissions and approvals, and other regulatory decisions and designations: Argo, Astrazeneca, Cogent, Glafabra, Koru Medical, Novartis, Roche, Sun, Telix. Read More