ABL Bio Inc. sealed a potential £2.075 billion (US$2.65 billion) license deal with GSK plc, granting GSK global rights to use ABL’s blood-brain barrier penetrating bispecific antibody platform, Grabody-B, to develop multiple programs in the neurodegenerative disease arena. Under the terms signed April 5, ABL agreed to transfer Grabody-B-related technology and know-how to GSK, upon which GSK will assume responsibility for preclinical and clinical development, manufacturing and commercialization. London-headquartered GSK will use the technology to develop multiple programs for new targets and various modalities including antibodies, polynucleotides or oligonucleotides (such as small-interfering RNA or antisense oligonucleotides). Seongnam-si, South Korea-based ABL, in turn, will receive up to £77.1 million in up-front and near-term payments, including an immediate up-front payment of £38.5 million, research milestones and potential program expansion.
Phase III in hypothalamic obesity wins for Rhythm
Rhythm Pharmaceuticals Inc. rolled out positive top-line data from the pivotal phase III study called Transcend with setmelanotide, a melanocortin-4 receptor agonist, for the treatment of acquired hypothalamic obesity (HO). The global trial met its primary endpoint with a statistically significant and highly clinically meaningful reduction in body mass index in adult and pediatric HO patients vs. placebo. Setmelanotide was first approved as Imcivree by the U.S. FDA for chronic weight management in adult and pediatric patients with obesity due to proopiomelanocortin, PCSK1, or leptin receptor deficiency. Shares of Boston-based Rhythm (NASDAQ:RYTM) were trading at $47.47, up 52 cents.
US court says no to NIH indirect rate cut
The nationwide preliminary injunction keeping the U.S. NIH from slashing its indirect cost rate to a flat 15% has now been turned into a permanent injunction. In issuing final judgment Friday in two challenges to the rate change, Judge Angel Kelley, of the U.S. District Court for the District of Massachusetts, said the NIH’s Feb. 7 notice that it was immediately cutting the rate violated the Administrative Procedure Act, as the action was arbitrary and capricious, was impermissibly retroactive and failed to follow notice-and-comment procedures. The same day Kelley issued the order, Massachusetts Attorney General (AG) Andrea Joy Campbell, who had led one of the challenges to the indirect rate cut, led a coalition of 16 state attorneys general in filing a new suit in the Massachusetts court. This time, the AGs are challenging the NIH’s termination of already-issued research grants and delays in reviewing grant applications.
More Lexeo data in Friedreich ataxia sees stock slump
Lexeo Therapeutics Inc. produced more positive interim data from early stage studies of its gene therapy to treat Friedreich ataxia cardiomyopathy. The results were taken from a Lexeo-sponsored Sunrise-FA phase I/II study and a Weill Cornell Medicine investigator-initiated phase Ia study of LX-2006. They showed clinically significant improvements in cardiac biomarkers and functional measures., along with increased frataxin protein expression in all patients with cardiac biopsies. In July 2024, interim data from the same two studies showed LX-2006 reduced heart muscle thickness. However, the company’s stock (NASDAQ:LXEO) struggled at midday as shares had dropped 30% to $1.64 each.
China clears more first-in-class innovative drugs than ever before
China approved 48 first-in-class innovative drugs, as well as a significant number of medications for pediatric and rare diseases, thanks to measures aimed at enhancing review efficiency and accelerating patient access to novel therapies, according to a report released by China’s National Medical Products Administration (NMPA). The 48 innovative drugs cover nearly 20 therapeutic areas, including oncology, neurological disorders and anti-infectives. China’s NMPA cleared 40 first-in-class drugs in 2023 and 21 in 2022. Among the first-in-class approvals, 17 received market approval through a priority review pathway, 11 gained conditional market approval and 13 were included in breakthrough therapy programs. In addition, 55 drugs to treat rare diseases were approved in 2024.
2seventy, TG, Springworks lead cancer index among Q1 market falls
After gaining 5.78% in January, the BioWorld Cancer Index reversed course, falling 4.74% year-to-date by the end of March. The index’s movement has closely tracked broader market trends. The Nasdaq Biotechnology Index climbed to 5.15% at the end of January but ended the first quarter down 1.54%. Similarly, the Dow Jones Industrial Average rose 4.7% in the first month of the year before slipping to a 1.28% loss through March. In 2024, the BCI ended the year down 17.32%, after briefly rising 11.16% in late February.
CMS rule ditches plan for Medicare coverage of obesity drugs
A late 2024 CMS proposal to include obesity drugs like Novo Nordisk A/S’ Wegovy (semaglutide) and Eli Lilly and Co.’s Zepbound (tirzepatide) under Medicaid and Medicare didn’t make it far into the new administration. A final rule, set to be published in the Federal Register April 15, will not include the provision to add obesity drugs to Part D coverage, restricting coverage of the drugs to indications other than obesity, such as type 2 diabetes or cardiovascular disease.