Advanz Pharma Ltd. has won a stay on the withdrawal of its primary biliary cholangitis (PBC) drug, Ocaliva (obeticholic acid), after the European Commission (EC) said on Sept. 3 that the conditional marketing approval should be revoked. Following that, London-based Advanz launched a legal challenge, announcing on Sept 5 that the General Court of the EU has temporarily suspended the EC’s decision. As a result, the conditional marketing authorization for Ocaliva remains in place until further notice from the court, and the 7,000 existing patients – and new ones – will still have access to the farnesoid X receptor agonist.

Novo idles as Lilly produces positive phase III once-weekly insulin results

With Novo Nordisk A/S hobbled by a complete response letter (CRL), Eli Lilly and Co. has forged ahead in developing a once-weekly type 2 diabetes treatment. Top-line data from Lilly’s phase III Qwint-1 and Qwint-3 studies of efsitora alfa showed noninferior average levels of blood glucose compared to those using another daily basal insulin treatment. The study participants were adults using basal insulin for the first time and or were those who have switched from daily basal insulin injections. Novo received a CRL from the U.S. FDA for its BLA of its once-weekly insulin Awiqli in July, with the agency asking for more information on the manufacturing process and the type 1 diabetes indication before the review of the application can be completed.

ESC data fuel Cytokinetics, BMS face-off in cardiomyopathy

Cytokinetics Inc. continues to gain ground against competitor Bristol Myers Squibb Co. (BMS) in cardiomyopathy. South San Francisco-based Cytokinetics on Sept. 1 offered further mid- and late-stage data with myosin inhibitor aficamten at the European Society of Cardiology (ESC) congress in London. BMS, of New York, disclosed results at ESC, too, with same-class mavacamten, approved as Camzyos by the U.S. FDA in late April 2022 for New York Heart Association class II-III obstructive hypertrophic cardiomyopathy to improve functional capacity and symptoms.

Iterum looks to adcom on path to US FDA approval

After receiving a complete response letter from the U.S. FDA more than three years ago and conducting another phase III trial, Iterum Therapeutics International Ltd. is preparing to make its case before an advisory committee Monday for sulopenem etzadroxil/probenecid as an oral treatment option for women with uncomplicated urinary tract infections caused by specific microorganisms. According to the draft questions for the meeting, the FDA’s Antimicrobial Drugs Advisory Committee will not be asked to vote on anything. Instead, the group has two questions to discuss: the overall benefit-risk assessment of the fixed-dose combo and labeling information to ensure appropriate use of the product. That second question suggests the FDA may be leaning toward approval this time around.

Vaxcyte prices $1.3B follow-on on the back of strong VAX-31 data

On news that drove shares up by 36% on Sept. 3, San Carlos, Calif.-based Vaxcyte Inc. priced a $1.3 billion follow-on offering a day later. It is the fourth highest amount raised through a follow-on offering of shares in BioWorld’s records, as well as the second largest financing of all types for 2024, behind Pfizer Inc.’s $3.1 billion global sale of shares in March, not including debt financings. Shares (NASDAQ:PCVX) ended the day Sept. 3 at $110.15, up $29.39, following positive top-line phase I/II data of Vaxcyte’s 31-valent pneumococcal conjugate vaccine candidate, VAX-31, which is now primed for a phase III trial. Analysts have suggested the vaccine could achieve majority market share in what is expected to be a $10-billion-plus market by 2030.

Pharos iBio wins MFDS orphan drug designation for AML drug 

South Korean artificial intelligence (AI)-based drug developer Pharos iBio Co. Ltd. said that the Ministry of Food and Drug Safety granted an orphan drug designation for PHI-101, a second-line therapy for acute myeloid leukemia. Shares of Anyang-si, Korea-based Pharos (KOSDAQ:388870) rose 6.73% from the day prior on Sept. 4 to ₩11,260 (US$8.44) but dropped back down 6.39% Sept. 5, closing at ₩10,540. As an FMS-like tyrosine kinase 3 (FLT3)-targeting cancer therapy, PH-101 has been in a global phase I study since July 2023, having shown “significant efficacy” in patients with disease relapse after treatment with existing FLT3 inhibitors, Pharos said.  

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