Nearly five years after submitting its first NDA seeking U.S. approval for Vanflyta (quizartinib) for treating a subset of patients with acute myeloid leukemia (AML), Daiichi Sankyo Co. Ltd. finally cleared the last hurdle. The FDA on July 20 approved the FLT3 inhibitor for use in combination with cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed disease that is FLT3 ITD-positive.
Nearly five years after submitting its first NDA seeking U.S. approval for Vanflyta (quizartinib) for treating a subset of patients with acute myeloid leukemia (AML), Daiichi Sankyo Co. Ltd. finally cleared the last hurdle. The FDA on July 20 approved the FLT3 inhibitor for use in combination with cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed disease that is FLT3 ITD-positive.
While the 12-month data from Bridgebio Pharma Inc.’s phase III ATTRibute-CM study testing acoramidis in transthyretin amyloid cardiomyopathy were deemed “puzzling” in late 2021, the 30-month results reported July 17 proved far more pleasing to investors. The trial hit all its endpoints and showed promising results on exploratory markers, sending company shares (NASDAQ:BBIO) up 76%, to close at $32.04, up $13.82.
On the back of what Arthrosi Therapeutics LLC has called “remarkable efficacy and safety” data from a phase IIb trial of AR-882, a next-generation URAT1 inhibitor for treating gout, the company has padded its coffers with a $75 million series D round led by Guangrun Health Industry Co. Ltd. and backed by investors that include Reichstein Biotech Co. Ltd., a subsidiary of Arthrosi’s China joint venture partner Apichope Pharmaceuticals.
Extending its efforts in the booming antibody-drug conjugate (ADC) space, Beigene Co. Ltd. tapped Duality Biologics Co. Ltd. in a deal that could be worth a potential $1.3 billion, picking up an option for rights to a preclinical-stage ADC candidate targeting select solid tumors. It marks the seventh $1 billion-plus ADC-focused deal so far in 2023 and the second for Dualitybio, which inked a potential $1.7 billion deal in April.
Extending its efforts in the booming antibody-drug conjugate (ADC) space, Beigene Co. Ltd. tapped Duality Biologics Co. Ltd. in a deal that could be worth a potential $1.3 billion, picking up an option for rights to a preclinical-stage ADC candidate targeting select solid tumors. It marks the seventh $1 billion-plus ADC-focused deal so far in 2023 and the second for Dualitybio, which inked a potential $1.7 billion deal in April.
Opko Health Inc. stands to receive a $90 million milestone payment from partner Pfizer Inc. on the U.S. FDA approval of once-weekly human growth hormone analogue Ngenla (somatrogon) for pediatric patients with growth hormone deficiency. Pfizer expects to make the drug available to patients starting in August 2023.
While investors focused on the top-line miss from Avalo Therapeutics Inc.’s phase II study of AVTX-002 (quisovalimab) in non-eosinophilic asthma, sending company shares (NASDAQ:AVTX) falling 89% to close at 50 cents June 26, the company pointed to promising trends seen in an exploratory analysis affirming the potential of targeting LIGHT.
Intercept Pharmaceuticals Inc.’s second attempt to score an expanded U.S. FDA approval of its farnesoid X receptor agonist, obeticholic acid, in patients with non-alcoholic steatohepatitis (NASH) went the way of the first, with the agency issuing another complete response letter (CRL), prompting the company to drop all NASH-related investment and cut a third of its workforce.
Sarepta Therapeutics Inc. is set to introduce the first gene transfer therapy for ambulatory patients diagnosed with Duchenne muscular dystrophy (DMD), after the U.S. FDA granted accelerated approval to SRP-9001 (delandistrogene moxeparvovec). Branded Elevidys, the therapy marks Sarepta’s fourth approved treatment for DMD and the first to offer patients a one-time treatment option.
