Research & Development

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

Obeticholic acid failed to separate from placebo in a Phase III study of non-alcoholic steatohepatitis patients with cirrhosis, but Intercept said it still plans to refile an NDA for pre-cirrhotic NASH by year’s end.

The US biotech’s LOXL2 inhibitor candidate has reduced collagen fibrosis of the bone marrow in a small number of Phase II patients, helping validate a historically tricky approach attempted by few.

China is knocking on the door of the world’s top 10 most innovative economies, helped by sustained investment.

Japan has granted to the first approval worldwide to Daiichi Sankyo’s dual EZH1/2 inhibitor valemetostat, for the treatment of adult T-cell leukemia/lymphoma. Taisho’s trivalent anti-TNFα nanobody ozoralizumab also received its global first approval, as part a large new batch of regulatory nods in the country.

Axcella’s AXA1125, a therapeutic combination of amino acids, shows liver stiffness, liver enzyme and fat reduction benefits in Phase IIb interim look, but the firm lacks the cash to complete the trial.

Pfizer is buying Biohaven's migraine portfolio, but the rest of the pipeline is poised to be spun out and there have been two development setbacks. Despite verdiperstat's failure in ALS, the field had a same-day win with Amylyx's Relyvrio approval. 

New data from anemic myelofibrosis patients showed the drug reduced transfusion dependence, which that could unlock a more sizeable portion of the market for CTI BioPharma.

As the first CRISPR-based therapies draw closer to approval, the Flagship-backed biotech believes its gene writing tech is the ‘definitive’ genetic medicine platform.

The Dutch firm’s US filing for its first-in-class PI3K inhibitor has been accepted under priority review, bringing the asset closer to becoming the first disease-modifying option for a rare, genetic disorder.

Emerging Company Profile: US firm Peptilogics is advancing through the clinic with its lead orthopedic anti-infective drug candidate based on an AI platform that utilizes algorithms to improve peptide design.

Eisai and Biogen, who have an accelerated approval decision pending in the US, said they will seek traditional approvals for lecanemab by Q1 2023 based on positive results from the Phase III Clarity AD trial.