CEO John Leonard said Intellia Therapeutics Inc. plans “to share information on a cohort-by-cohort basis, so we get a consistent readout” and, as the year goes on, longer-term follow-up findings will emerge from the phase I trial with the company’s lead in vivo genome editing candidate, NTLA-2001.
Shares in Orphazyme A/S cratered after the FDA rejected its arimoclomol for Niemann-Pick disease type C, a rare and potentially fatal inherited condition where fat builds in tissues and organs.
LONDON – Newco Amyl Therapeutics Ltd. has raised €18.3 million (US$22.3 million) in a series A, to pick up the baton on a technology for neutralizing toxic protein aggregates by binding to their amyloid folds.
Scohia Pharma Inc. has secured an agreement with Huadong Medicine Co. Ltd., enabling the former to tap the greater China market for SCO-094, its GLP-1R and GIPR dual agonist.
Protalix Biotherapeutics Inc. saw shares dip after releasing initial top-line data from an interim analysis of the phase III Balance clinical trial of pegunigalsidase alfa (PRX-102) to treat Fabry disease. The 24-month, randomized, double-blind, active control study is evaluating the safety and efficacy of 1 mg/kg of PRX-102 dosed every two weeks compared to agalsidase beta (Fabrazyme). Two of the study’s 78 patients dropped out because of treatment emergent adverse events and one left because of a related adverse event.
As investors await an MAA submission to the EMA for U.S.-approved Mycapssa (octreotide) in acromegaly, Chiasma Inc. disclosed its plan to merge with Amryt plc in an all-stock deal, thereby gaining a global commercial presence. Terms call for the exchange of each Chiasma share for 0.396 American depositary share of Amryt. The latter closed May 4 at $12.95, so the deal values Chiasma at $5.13 per share, an 81% premium over the previous day’s ending price, noted Piper Sandler analyst Edward Tenthoff. The stock (NASDAQ:CHMA) rose $1.14, or 40%, to finish at $3.98.
Shares of Protalix Biotherapeutics Inc. (NYSE:PLX) fell 33% to $3.93 on April 28 after the company reported receiving an FDA complete response letter (CRL) in answer to its BLA for pegunigalsidase alfa, a galactosidase enzyme replacement therapy it has long advanced for the potential treatment of Fabry disease.
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.
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