HONG KONG – Canbridge Pharmaceuticals Inc. signed a collaboration and licensing agreement that could be worth $581 million, gaining global rights to develop, manufacture and commercialize gene therapy candidates from Logicbio Therapeutics Inc. for the treatment of Fabry and Pompe diseases. The candidates are based on Logicbio’s adeno-associated virus (AAV) sL65, the first produced from its Saavy capsid development platform.
About a year and a half after Enzyvant Inc.'s tissue-based therapy for children born without a thymus met with a complete response letter over chemistry, manufacturing and controls concerns, its BLA is once again on track for FDA review, the company told BioWorld. Following a resubmission intended to fully address the agency's concerns, the application has a new PDUFA date of Oct. 8.
Advances lately in the genome-editing space include Beam Therapeutics Inc. publication in The CRISPR Journal details of its work with inlaid base editors, which the firm is applying in the BEAM-102 program for sickle cell disease. IBEs’ predictable, shifted editing window lets researchers go after disease-causing mutations that canonical base editors cannot reach, Beam said, and do the job with high efficiency and few off-target effects on the genome. The hottest news due in the near-term future from the sector will spill from Intellia Therapeutics Inc., of Cambridge, Mass., which is due to roll out first-in-human data with a systemic CRISPR-based genome editing therapy, NTLA-2001, in hereditary transthyretin amyloidosis.
New and updated preclinical and clinical data presented by biopharma firms at the ENDO 2021, the Endocrine Society's annual meeting, including: 89bio, Amolyt, Biomarin, Bridgebio, Chiasma, Clarus, Crinetics, Lumos, Neurocrine, Strongbridge, Rezolute, Rhythm, Theratechnologies.
Molybdenum cofactor deficiency (MoCD) type A, an ultra-rare metabolic disorder causing intractable seizures, brain injury and death, now has a world-first treatment in Nulibry (fosdenopterin), a new I.V. therapy developed by Bridgebio Pharma Inc. subsidiary Origin Biosciences Inc. The agency's priority review, supported by its orphan, breakthrough and rare pediatric disease programs, also yielded a priority review voucher (PRV) for Origin.
A failure of Tricida Inc.'s most recent efforts to overcome FDA objections to an NDA for the company's sole candidate, veverimer, for treating metabolic acidosis, sent company shares (NASDAQ:TCDA) 30.6% lower to $5.11 on Feb. 26. The complex story appears focused now on the agency's desire for additional and more reliable data to support a potential approval. Tricida President and CEO Gerrit Klaerner on Thursday suggested the ongoing renal outcomes study, Valor-CKD, might provide it.
Amicus Therapeutics Inc.’s results from the phase III trial called Propel with AT-GAA (cipaglucosidase alfa and miglustat) for late-onset Pompe disease (LOPD) met with split opinions, though Wall Street took a decidedly dim view and left shares (NASDAQ:FOLD) to close at $12.57, down $6.16, or 33%.
Almost three years after announcing it would quit development of its humanized immunoglobulin G1 candidate, birtamimab, in light chain (AL) amyloidosis, Prothena Corp. plc is revving the program back up with a new focus, sending shares (NASADQ:PRTA) 30.5% higher to close at $14.35 on Feb. 2.
Alnylam Pharmaceuticals Inc. said the 164-patient Helios-A phase III study with next-generation RNAi drug vutrisiran hit its primary endpoint as well as both secondary goals in the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy. The primary endpoint was change from baseline in the modified Neuropathy Impairment Score at nine months as compared to historical placebo data from the Apollo phase III study of Alnylam’s Onpattro, cleared by the FDA for ATTR polyneuropathy in August 2018.
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