Scarlet Therapeutics Ltd. has demonstrated its manufactured red blood cells (RBCs) act in the same way as their natural counterparts in vivo, opening the way for the cells to be used as drug delivery vehicles and raising the possibility they could replace conventional blood transfusions. To build on this, Scarlet has closed a $4 million seed round to work on the first clinical application, in which RBCs loaded with therapeutic proteins will be used to treat rare metabolic diseases.
The U.S. FDA approved Corcept Therapeutics Inc.’s oral, selective glucocorticoid receptor antagonist, Lifyorli (relacorilant), nearly four months ahead of schedule for adults with platinum-resistant epithelial ovarian, fallopian tube or primary peritoneal cancer. A short time after the agency approved Lifyorli, it cleared Denali Therapeutics Inc.’s Avlayah (tividenofusp alfa) under the accelerated approval pathway for mucopolysaccharidosis II, also called Hunter syndrome, ahead of the April 5 PDUFA date.
A new study has reinforced that GLP-1 receptor agonists are unlikely to produce durable weight loss, but indicates that rather than returning to the starting weight, individual weight gain will plateau at 75.5% of the weight lost.
Targeting an ultrarare disease affecting about 250 U.S. residents, Immedica Pharma AB’s Loargys (pegzilarginase) won accelerated FDA approval to treat hyperargininemia in those ages 2 and older with arginase 1 deficiency (ARG1-D).
After expanding development of DM-199 (rinvecalinase alfa) into preeclampsia last year, Diamedica Inc. rolled out early phase II results showing the recombinant form of human tissue kallikrein-1 reduced the mother’s blood pressure, did not cross the placental barrier, and potentially improves blood flow to the fetus.
Alteogen Inc. completed a merger between two subsidiaries – Altos Biologics Inc. and Alteogen Healthcare Inc. – branding the new entity as Alteogen Biologics Inc.
It’s been a big week for Inozyme Pharma Inc. On the heels of a promising interim readout for phase III-stage enzyme replacement therapy (ERT) candidate INZ-701 in ENPP1 deficiency, the firm agreed to be acquired by Biomarin Pharmaceuticals Inc. in a deal valued at about $270 million, putting the rare disease ERT in the hands of an experienced commercial team.
Just two months after Inozyme Pharma Inc. cut its workforce by 25% and prioritized activities to focus on a BLA filing for INZ-701 for ENPP1 deficiency, interim phase III data from its Energy 3 trial showed consistent safety and immunogenicity and increased phosphate levels in patients treated with the rare disease enzyme replacement therapy.
After dropping development in December of its lead program, Spruce Biosciences Inc. has found new life by acquiring a BLA-ready enzyme replacement therapy for the rare genetic neurodegenerative disease Sanfilippo syndrome type B. If approved, the therapy, tralesinidase alfa, could bring Spruce a priority review voucher.
Alteogen Inc. signed its first billion-dollar partnership this year for ALT-B4 technology through two separate contracts with Astrazeneca plc’s U.K.- and U.S.-based Medimmune subsidiaries worth up to $1.35 billion combined.
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