PERTH, Australia – Stem cell therapy company Mesoblast Ltd. provided new subgroup analyses from its phase III advanced chronic heart failure trial showing a greater treatment benefit from rexlemestrocel-L in high-risk patients with chronic heart failure and low ejection fraction (HFrEF) with diabetes or ischemia. The company had reported in December 2020 that the allogeneic mesenchymal cell therapy failed to meet the primary endpoint of a reduction in hospitalizations in its DREAM-HF phase III trial.
Longeveron Inc.’s share price (NASDAQ:LGVN) jumped 11.7% on Dec. 3 after the FDA awarded orphan designation to Lomecel-B, allogeneic bone marrow-derived mesenchymal stem cells, for treating hypoplastic left heart syndrome. The designation came two weeks after the FDA granted rare pediatric disease designation for Lomecel-B to treat the life-threatening congenital heart defect in infants.
In the December 3, 2021, issue of Science, researchers reported that a missense variant that is highly enriched in the Amish population was associated with reduced levels of both LDL cholesterol and fibrinogen. To date, very few variants have been identified that affect more than one risk factor for cardiovascular disease.
NHS England has struck new pricing agreements that expands access in the U.K. to blood thinning direct oral anticoagulants (DOACs) to tackle strokes in patients with atrial fibrillation. Though still available to NHS clinicians, Boehringer Ingelheim GmbH’s blockbuster DOAC Pradaxa (dabigatran) is notably not involved.
LONDON – Four decades of academic research at the University of California, San Francisco, is to be translated through to the clinic with funding from a €1 million (US$1.2 million) European Commission grant. The money, awarded to a Dutch/German consortium will fund the generation and optimization of antibodies designed to neutralize oxidized phospholipids that mediate inflammatory changes in a swathe of diseases. The consortium that won the Eurostars award is led by Oxitope Pharma BV.
When James Peyer, Cambrian Biopharma Inc.’s CEO, watched his grandfather fail every cancer treatment and eventually pass away, he came to a realization that now forms the backbone of his company. “The more I learned about cancer, the more convinced I became that we were approaching cancer as a disease in the wrong way,” Peyer told BioWorld. “We were waiting until people were sick and only then doing something about it.” Cambrian just closed on an oversubscribed series C that brought in $100 million to develop a pipeline of therapies designed to treat and prevent age-related diseases.
Omeros Corp.’s complete response letter (CRL) from the FDA regarding its BLA for narsoplimab did not come as much of a surprise to Wall Street, but still was enough to drive shares (NASDAQ:OMER) down 26.6% to close Oct. 18 at $5.67, a loss of $2.06.
Less than two weeks after Japan’s MHLW became the first regulatory agency to clear avacopan for anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis, the FDA has followed suit. It cleared the oral, small-molecule C5aR antagonist for use as an adjunct therapy for adults with the two main forms of the rare autoimmune renal disease, granulomatosis with polyangiitis and microscopic polyangiitis, in combination with standard therapy.
Voyager Therapeutics Inc. is getting $30 million up front in a potential $630 million gene therapy deal with Pfizer Inc., the company’s first such agreement since a strategic refocusing effort earlier this year and a much-needed endorsement of a next-generation AAV capsid platform that has shown promising though early stage data.
DUBLIN – Treefrog Therapeutics SA closed a $75 million series B round this week, which will help to increase its reach and its profile, as it pursues its highly ambitious objective to drive the adoption of a new way of making induced pluripotent stem cells (iPSCs) at scale. The Bordeaux, France-based firm is not a CDMO in any sense, however. It is a fully fledged biotech, with early stage iPSC-based programs in Parkinson’s disease, cardiovascular disease and bone marrow transplant, among others. It’s just that it is also attempting to revolutionize how those cells are cultivated before it administers them as therapies.
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