Turbulent years of development that included a clinical hold, a COVID-19 stumble, a withdrawn approval application and an extended PDUFA date has yielded to FDA approval of CTI Biopharma Corp.’s Vonjo (pacritinib) to treat bone marrow cancer myelofibrosis. The oral JAK2, IRAK1 and CSF1R inhibitor’s NDA received priority approval to treat adults with intermediate or high-risk primary or secondary myelofibrosis, which affects about 21,000 patients in the U.S. The approval unlocked a $60 million payment to CTI from Toronto-based DRI Healthcare Trust to purchase a tiered royalty on sales of potential future sales. At midday, CTI stock (NASDAQ:CTIC) had leapt about 60%.

Janssen-Legend BCMA-targeting CAR T therapy crosses the line with US FDA approval

Janssen Pharmaceutical Cos. Inc. has become the second company to get a BCMA-targeting CAR T therapy to market with the approval of its ciltacabtagene autoleucel for adults with relapsed or refractory multiple myeloma. U.S. FDA approval of the medicine, licensed from Legend Biotech USA Inc. and to be marketed as Carvykti, comes after an extended review during which Janssen submitted further analytical information to the regulator, although no additional clinical data were requested.

Abbvie expanding neurosci portfolio with $1B Syndesi buyout

Abbvie Inc. has paid $130 million up front to acquire Syndesi Therapeutics SA, a company working on a new mechanism to mitigate synaptic dysfunction associated with cognitive deficits. The deal includes up to $870 million in milestone payments for Syndesi's first-in-class modulators of the synaptic vesicle protein 2A, which Abbvie said could have applications across a range of neuropsychiatric and neurodegenerative disorders. The lead molecule, the phase Ib asset SDI-118, is under evaluation for targeting nerve terminals to enhance synaptic efficiency.

US PTAB may have spoken, but CRISPR patent battle wages on

The U.S. Patent Trial and Appeal Board (PTAB) decision yesterday that Broad Institute scientists were the first to invent the use of CRISPR/Cas9 genome editing in eukaryotic cells is likely not the final answer to the question of CRISPR inventorship. The Regents of the University of California, the University of Vienna and Emmanuelle Charpentier (CVC), which had filed competing patent applications, are expected to appeal to the U.S. Circuit Court of Appeals for the Federal Circuit. That could leave biopharma companies with CVC licenses in limbo as they wait for the court to weigh in.

As questions mount, battle rages on for COVID-19 solutions

Two years ago, BioWorld reported on 30 therapeutics and vaccines in development for COVID-19, about 3,000 people had died from the disease, and society locked down to stem the flow of hospital cases. Today, therapeutics and vaccines have ballooned to 1,048, deaths are at nearly 6 million worldwide, and highly transmissible variants and breakthrough infections have dictated every step taken. One thing remains the same. Scientists still don’t know conclusively where the SARS-CoV-2 virus originated. Meanwhile, new options for treatment and prevention are consistently emerging.

Newco news: Teclison raises $5.9M to advance solid tumor program

Teclison Ltd. has raised $5.9 million to support further development of its lead candidate, TEC-001, an agent designed to induce tumor necrosis and enhance immune checkpoint inhibitors in solid cancers with liver metastasis. Wtt Investment Ltd., the Taiwan-based family office of the late Taiwanese banker Tsai Wan-tsai, led the financing. Tsai was one of Taiwan’s richest men and the founder of the financial services conglomerate Fubon Financial Holding Co. Ltd.

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