Shares of Travere Therapeutics Inc. (NASDAQ:TVTX) were trading $11.22, up $1.29, or 13%, on word of full approval of Filspari (sparsentan) to slow kidney function decline in adults with primary IgA nephropathy (IgAN) who are at risk of disease progression. The endothelin and angiotensin II receptor antagonist won accelerated approval from the U.S. FDA in February 2023. Full approval is based on positive long-term confirmatory results from the Protect study in IgAN showing that Filspari significantly slowed kidney function decline over two years compared to the angiotensin II receptor antagonist irbesartan, which first became available in 1997.
Vor’s stem cell transplant delays cancer relapse
Vor Biopharma Inc.’s trem-cel, a stem cell transplant designed to block the toxicity from some cancer treatments, has produced some positive early stage results, including delayed relapse in patients. Phase I/II study data showed participants with relapsed/refractory acute myeloid leukemia (AML) benefited from trem-cel followed by treatment with Pfizer Inc.’s antibody-drug conjugate cancer drug Mylotarg (gemtuzumab ozogamicin). There was reliable engraftment and toxicity from Mylotarg was reduced. The results also suggested improved relapse-free survival compared to some other high-risk AML comparators. After an opening-bell spike, the company’s stock (NASDAQ:VOR) had settled at midday to a 6% increase with shares going for 88 cents each.
Biopharma financings near $78B, nearly doubling last year’s value
Biopharma financing activity surged in the first eight months of 2024, reaching $77.49 billion, a 91.25% increase from the $40.52 billion raised during the same period in 2023. August saw a slight dip in funding, with $3.48 billion raised compared to $3.71 billion in July.
Epimab licenses BCMA bispecific to startup Vignette in $635M deal
In a deal that brings $60 million in cash and equity up front, plus up to $575 million in milestone payments, Shanghai-based Epimab Biotherapeutics Inc. and San Diego-based Vignette Bio Inc. entered a licensing agreement for Epimab’s BCMA-targeting T-cell engager (TCE), EMB-06, for autoimmune disease. Vignette, a recent startup incubated by Foresite Labs, will pick up exclusive rights for EMB-06 worldwide, with the exception of greater China, encompassing mainland China, Hong Kong, Macau and Taiwan, where Epimab will retain rights to the TCE.
Updating Oscar Wilde’s visions of rejuvenation at ARDD 2024
Immortality and eternal youth have been the stuff of myths and legends from ancient times on. Now, in the 21st century, real studies of current medicine could be applied to repair tissues and organs damaged by age. During the 11th Aging Research & Drug Discovery (ARDD) Meeting held at the University of Copenhagen at the end of August, scientists explained the molecular keys of rejuvenation, as many artists imagined in the past. The Greek goddess Hebe revitalized the gods of Olympus by serving them nectar and ambrosia. The German Lucas Cranach the Elder painted the renewing power of water in The Fountain of Youth. The Elixir of Life, by the French writer Honoré de Balzac, and the famous The Picture of Dorian Gray, by the Irish playwright Oscar Wilde, captured the Victorian concern about aging. No one knew about DNA, mutations, telomeres, epigenetics, or how proteins and transcription factors could regulate aging.
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Abdera, Advanz, Crestone, Cyclo, Eli Lilly, Ena Respiratory, Evotec, Genetic Leap, Hdax, Hoth, Iambic, In8bio, Indaptus, Lisata, Lykos, Medigene, Medincell, Moderna, NRG, Orbital, Perspective, Pleopharma, Poseida, Qualigen, Simcere, Transcode, Vivet, Voyager, X-Chem