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.
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.
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.
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.
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.
Shares of Cytomx Therapeutics Inc. hit a new 52-week high as the company unveiled preliminary data from an ongoing phase I study of varsetatug masetecan (Varseta-M) in late-line metastatic colorectal cancer.
The disjointed health care system that patients face today blocks a significant proportion of them from accessing life-changing, innovative drugs, prompting many in the biopharma industry to explore more direct-to-consumer (DTC) pathways. During Pharma USA, a Reuters Events meeting held in Philadelphia March 16-17, executives from Eli Lilly and Co., Roche Holding AG’s Genentech unit, Amgen Inc. and consulting firm ZS Associates, among others, offered glimpses into a future where R&D is no longer enough.
Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine.
Stryker Corp. is scrambling to recover from a cyberattack that’s disrupting its global network and being claimed by pro-Iranian hackers. “At this time, there is no indication of malware or ransomware, and we believe the situation is contained to our internal Microsoft environment only,” the Kalamazoo, Mich.-based med-tech company said March 12.
The Advanced Research Projects Agency for Health (ARPA-H) launched its Delphi program March 10 to advance the development of the next generation of wearable and ingestible biosensors that can securely report deep biological data to optimize health care.