Chiesi Farmaceutici SpA scored U.S. FDA clearance of the enzyme replacement therapy (ERT) Lamzede (velmanase alfa-tycv) for non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. An ultra-rare, progressive lysosomal storage disorder, AM is caused by deficiency in the enzyme alpha-mannosidase. Lamzede is the first ERT to win approval in the indication.

Mixed results for Moderna’s flu vaccine

The A’s have it but the B’s don’t in Moderna Inc.’s pivotal phase III study of mRNA-1010, a seasonal flu vaccine for adults living in the southern hemisphere. Interim results showed the vaccine achieved superiority on seroconversion rates for influenzas A/H3N2 and A/H1N1, superiority on geometric mean titer ratios for influenza A/H3N2 and noninferiority on geometric mean titer ratios for influenza A/H1N1. However, noninferiority was not met for the endpoints against the influenza B/Victoria- and B/Yamagata-lineage strains. Participants were randomly assigned to a single dose of mRNA-1010 or a single dose of a licensed seasonal influenza vaccine. The company stock (NASDAQ:MRNA) staggered at the opening but shares had rallied to trading about 5% downward at midday.

Denali, Regenxbio, Takeda among strivers in busy MPS II space

Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, continues to bustle, with the likes of Denali Therapeutics Inc., Regenxbio Inc. and Takeda Pharmaceutical Co. Ltd. in play. Denali provided an extensive update on clinical programs in central nervous system diseases at the start of this year, including lead asset DNL-310 in MPS II. The compound has yielded convincing biomarker and proof-of-concept data, thanks to the South San Francisco-based firm’s transport vehicle technology.

Ascletis’ PD-L1 antibody achieves functional cure of chronic hepatitis B in clinical trial

Ascletis Pharma Inc. presented data from a phase II trial of showing its subcutaneous PD-L1 antibody envafolimab (ASC-22) was able to functionally cure chronic hepatitis B, according to hepatitis B surface antigen (HBsAg) decline following 24-week treatment. The study enrolled 48 patients in the treatment arm, who completed 24 weeks of treatment with 1-mg/kg ASC-22 and had 24 weeks or more of follow-up. In the patient subgroup with baseline HBsAg ≤100 IU/mL, 42.9% of patients (3/7) achieved HBsAg loss during the treatment, which referred to HBsAg below the lower limit of quantification (LLOQ < 0.05 IU/mL). Functional cure is defined as achieving HBsAg loss with a period of treatment, typically 24 to 48 weeks, and maintaining HBsAg loss after at least a 24-week follow-up.

Newco news: Cargene’s siRNA therapy has potential to regenerate the liver and reverse fibrosis

One of the most important transformations in the pharmaceutical industry over the next decade is the ability to modify genes or expression and go after any target, Cargene Biopharma Inc. CEO Kathy He told BioWorld, explaining that small molecules and large molecules can only go after 15% of the known targets. But the technology platform of short-interfering RNA (siRNA) opens a huge opportunity to make those inaccessible targets available, she said.

Holiday notice

BioWorld's offices will be closed in observance of Presidents Day in the U.S. No issue will be published Monday, Feb. 20.

Also in the news

Abivax, Aldeyra, Alpha Cognition, Alveolus, ALX Oncology, Ambrx, Applied, Ascletis, Aura, Axcella, Bellicum, Bergenbio, Boan, Celcuity, Celldex, Disc, Entera, Helocyte, Immuno Cure, Immvira, Innovent, Inovio, Inozyme, Janssen, Jasper, Kancera, Kuros, Lava, Mitsubishi Tanabe, Newron, Nymox, Ocean, OSE, Pfizer, Pharming, Promontory, Quantum Leap, Redhill, Sermonix