Human normal immunoglobulin(Takeda)

Vertex bins Moderna-partnered cystic fibrosis candidateTakeda touts pivotal win in immune disorder trialAmgen commits $300M more to Puerto Rico site Latus rounds off series A at $97MVertex bins Moderna-partnered cystic fibrosis candidate Vertex is discontinuing the Phase I/II study of VX-522, an experimental cystic fibrosis treatment designed to deliver mRNA directly to the lungs and restore CFTR protein function. The biopharma had been working on the inhaled asset in partnership with Moderna. According to Moderna, the programme was ended after the two teams "were not able to achieve a profile that supported continued development given tolerability issues." The partners will halt all development of VX-522."This programme represented our only inhaled therapeutic approach and utilised a unique formulation and delivery system, with no broader implications for our portfolio," Moderna said in a statement. -Elizabeth EatonTakeda touts pivotal win in immune disorder trialTakeda reported Phase II/III data showing its experimental treatment TAK-881 can deliver the required immunoglobulin dose for patients with primary immunodeficiency disease (PID) at half the infusion volume of its established product HyQvia, while maintaining up to once-monthly dosing.In the study, TAK-881 demonstrated pharmacokinetic comparability with HyQvia, an immune globulin with a recombinant human hyaluronidase that US regulators approved for PID in 2014. Takeda said the experimental treatment delivered equivalent IgG exposure, comparable infection rates and immune protection to HyQvia, and a similar safety profile.TAK-881 could offer "the potential advantages of fewer injection sites, a flexible treatment schedule and shorter infusion times," said Kristina Allikmets, head of plasma-derived therapies R&D at Takeda. The company plans to share full data at upcoming medical meetings and submit regulatory applications in 2026 across the US, Europe and Japan.-Anna BratulicAmgen commits $300M more to Puerto Rico site Amgen is pouring an additional $300 million into its biologics manufacturing facility in Juncos, Puerto Rico to boost its US-based production capacity. The investment comes less than a year after the pharma initially committed $650 million towards a major build-out of the Juncos site. According to Amgen, the funding increase will help strengthen existing advanced manufacturing roles at the facility, support workforce development across the region and generate hundreds of construction jobs. The latest manufacturing pledge adds to other recent investments from Amgen, including $900 million in Ohio and $600 million in a science and innovation centre in California, as well as a total $1.5-billion commitment in North Carolina.-Elizabeth EatonLatus rounds off series A at $97MLatus Bio on Monday closed a series A extension at $43 million, bringing the round's total to $97 million. The financing, led by 8VC, will help the biotech reach key clinical readouts for two of its gene therapy programmes: LTS-201 for Huntington’s disease and LTS-101 for late-infantile neuronal ceroid lipofuscinosis type 2 disease.The former, an AAV gene therapy designed to knock-down MSH3, is on track for an IND submission in the third quarter. The latter gene therapy is due to begin a first-in-human investigator-initiated trial in the third quarter, with initial safety, biomarker and clinical results expected by year-end. Funds from the series A will also go towards additional preclinical programmes that leverage Latus' AAV discovery platform.Existing investors DCVC Bio, BioAdvance, Benjamin Franklin Technology Partners, Modi Ventures, Gaingels and Hatch BioFund participated in the round, alongside new investors Korea Development Bank and Helen’s Pink Sky Foundation.-Elizabeth Eaton

The increased concentration in TAK-881 is designed to deliver the immunoglobulin (IG) dose while reducing infusion duration and maintaining once-monthly dosing for patients with primary immunodeficiency disease. \n A pivotal study of a Takeda primary immunodeficiency disease (PID) asset has hit its primary endpoint, demonstrating pharmacokinetic (PK) comparability between the asset and the company’s HyQvia, which was approved to treat PID in 2014.Takeda’s TAK-881 is a subcutaneous therapy comprising an immunoglobulin 20% solution and Halozyme’s recombinant human hyaluronidase. The candidate holds the promise of treating PID with half the infusion volume of HyQvia, which contains immunoglobulin 10%. The increased concentration is designed to deliver the immunoglobulin (IG) dose while reducing infusion duration and maintaining once-monthly dosing for patients with PID, according to a May 4 release.Secondary endpoints of the phase 2/3 TAK-881-3001 trial also demonstrated safety, efficacy and tolerability profiles for TAK-881 that are comparable to HyQvia, according to Takeda. The trial evaluated the candidate in adults and pediatric patients aged 2 and older with PID who had been previously treated with IG therapy, comparing the data with HyQvia\'s profile in patients aged 16 and older.Topline results from the trial showed that TAK-881 demonstrated equivalent immunoglobulin G (IgG) exposure to HyQvia, as shown by a geometric mean ratio of 99.67% for the areas under the concentration-time curves over one dosing interval. The 90% confidence interval was 95.1% to 104.46%.The asset also demonstrated comparable infection rates and immune protection to HyQvia, along with a comparable safety profile, Takeda said.PID is a collection of over 500 rare, chronic immune disorders, making patients susceptible to severe and unusual infections. IG replacement therapy is the only treatment option to maintain immune protection, but often requires frequent and high-volume infusions.Both TAK-881 and HyQvia are part of Takeda’s plasma-derived therapies, one of the company’s key business areas. “These phase 2/3 results showed the pharmacokinetic profile of TAK-881 was comparable to HyQvia, an established IG standard of care in patients with PID, while offering the potential advantages of fewer injection sites, a flexible treatment schedule and shorter infusion times,” Kristina Allikmets, M.D., Ph.D., Takeda\'s SVP and head of plasma-derived therapies R&D, said in a May 4 statement.  Full analysis of the phase 2/3 is ongoing, but Takeda said it expects to submit applications for the drug to the FDA, European Union and Japan in the 2026 fiscal year, which ends in March 2027.HyQvia came to Takeda by way of Shire, which it acquired in 2018 for $62 billion. Shire bought Baxter’s Baxalta spinoff for $32 billion and became the owner of HyQvia in 2016. In recent years, HyQvia has seen its indications expand. The FDA approved it as a treatment for children with PID in 2023 and as a maintenance therapy for the progressive neurological disorder chronic inflammatory demyelinating polyneuropathy in 2024.