Avrobio Inc. said Feb. 8 that the first person dosed with AVR-RD-01, an investigational ex vivo lentiviral gene therapy from its upgraded manufacturing platform, Plato, experienced a complete clearance of the toxic substrate lyso-Gb3 in a kidney biopsy.
Covicept Therapeutics Inc., a young San Diego-based company focused on developing a small molecule to inhibit the replication and spread of SARS-CoV-2 and other RNA viruses, has launched with $2.3 million in seed funding from European VC firm Forbion. The company, spun out of research at the University of California, San Diego, aims to initiate its first clinical study in the middle of 2021.
Just weeks after seeing one late-stage candidate hit a wall in a subtype of non-small-cell lung cancer (NSCLC), Merck KGaA's EMD Serono has won accelerated approval from the FDA for another important NSCLC therapy, Tepmetko (tepotinib).
Vaxart Inc. investors who stuck by company shares Feb. 3 had to swallow a bitter pill – watching shares of the oral vaccine developer (NASDAQ:VXRT) fall 57.8% to $9.85. Driving the trade in part was news that, during a phase I trial, neutralizing antibodies to SARS-CoV-2 were not detected in most of the five subjects given two doses of VXA-CoV2-1, its oral recombinant protein vaccine for the potential prevention of SARS-CoV-2 infection. Furthermore, the company concluded it was "unable to evaluate" a study of the candidate organized by Operation Warp Speed.
Almost three years after announcing it would quit development of its humanized immunoglobulin G1 candidate, birtamimab, in light chain (AL) amyloidosis, Prothena Corp. plc is revving the program back up with a new focus, sending shares (NASADQ:PRTA) 30.5% higher to close at $14.35 on Feb. 2.
Seeking to accelerate the company's long-term growth through an expansion of its rare disease pipeline, Horizon Therapeutics plc said Feb. 1 it will buy Viela Bio Inc. for $3.05 billion, or $53 per share (NASDAQ:VIE). Once closed, the deal would add a new commercial-stage asset, Uplizna (inebilizumab), to Horizon's portfolio alongside its current lead products, Tepezza (teprotumumab) for the treatment of thyroid eye disease and Krystexxa (pegloticase) for uncontrolled gout. Dublin-based Horizon, which had $2.08 billion in cash at the end of 2020, borrowed $1.3 billion to help finance the transaction. Astrazeneca plc also enabled the deal by agreeing to divest its 26.7% share in Viela Bio for a profit of between $760 million and $780 million.
Triggering a wave of commentary over its import, Biogen Inc. on Jan. 29 said the FDA has extended to June 7 its review of a BLA for the experimental Alzheimer's disease therapy aducanumab after the company submitted additional analyses and clinical data, making for a major amendment to the application. The three-month delay, from an earlier assigned PDUFA date of March 7, followed a thumbs-down vote by agency advisors in November. It sparked both sunny optimism and a bit of pessimism about the program's prospects on Friday.
In a move that will enable both a broadening of its portfolio and a strengthening of its IP position, Neubase Therapeutics Inc. has acquired technology from Vera Therapeutics Inc. for creating peptide-nucleic acid scaffolds. The deal will extend the ability of Neubase's Patrol platform, a tool for addressing the causal mechanism of genetic disease, to directly modulate the human genome to resolve both rare and common diseases, including cancers, the company said.
Little more than half-a-year after closing its $78 million series A financing, Artiva Biotherapeutics Inc. has inked a new global licensing deal with Merck & Co. Inc. to develop up to three natural killer (NK) cell therapies engineered with chimeric antigen receptors (CAR) against tumor-associated antigens of its choosing. Merck will pay Artiva $30 million up front for two initial programs, plus $15 million more should it opt into the third. Each program carries $612 million in potential development and commercial milestone payments. With unfettered success, total deal value could hit $1.88 billion, plus royalties.
Slate Bio Inc., a startup developing an interleukin 2 fusion cytokine for the potential treatment of autoimmune and inflammatory diseases, has closed a $1.75 million seed financing led by Epidarex Capital. Financing for the company, a spinout of the University of Virginia, also came from the UVA Licensing & Ventures Group Seed Fund, Center for Innovative Technology's GAP Biolife Fund, VTC Seed Fund, Pharmadirections Inc., company management and others.
