The “remarkably appealing” route of administration and every-three-month dosing put Ferring Pharmaceuticals SA’s nadofaragene firadenovec (rAd-IFN/Syn3, also known as Instiladrin) in strong position for approval in high-grade Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC).
In the summer of 2016, when Victoria, British Columbia-based Aurinia Pharmaceuticals Inc. offered phase IIb results with its calcineurin inhibitor voclosporin in lupus nephritis (LN), Wall Street ignored the otherwise-positive results and zeroed in on the trial’s death rate: 13 casualties across three arms of the 265-subject Aura-LV study.
Bispecific: It might sound to some like a self-contained contradiction, as in “doubly singular” or “twice particular.” But if the word “specific” is taken in its primary meaning as “clearly defined or identified,” then the matter becomes clear – and, in the case of bispecific antibodies, it’s becoming clearer by the year.
Audentes Therapeutics Inc. CEO Matthew Patterson early last month characterized results with lead compound AT-132 in X-linked myotubular myopathy (XLMTM) as “unprecedented in neuromuscular disease,” and the value apparently wasn’t lost on Tokyo-based Astellas Pharma Inc., which signed a deal worth about $3 billion to take over the company. Shares of Audentes (NASDAQ:BOLD) closed at $58.93, up $30.32, or 106%, on word of the buyout – which pairs the two firms’ gene therapy expertise and is slated to close in the first quarter of next year – at a cost of $60 per share in cash.
<p>Shares of Audentes Therapeutics Inc. (NASDAQ:BOLD) were trading pre-market at $58.97, up $30.36, or 106% on word of the takeover by Astellas Pharma Inc., which is paying $60 per share in cash for an equity value of about $3 billion.</p>
Commissioner of the FDA for five years starting in 1984, Frank Young relished his position “at the vortex of controversy” as he sought to deal with the AIDS crisis and public furor over drug tampering, said his son, Jonathan Young, co-founder and chief operating officer of South San Francisco-based Akero Therapeutics Inc.
Commissioner of the FDA for five years starting in 1984, Frank Young relished his position “at the vortex of controversy” as he sought to deal with the AIDS crisis and public furor over drug tampering, said his son, Jonathan Young, co-founder and chief operating officer of South San Francisco-based Akero Therapeutics Inc. Post-FDA, Frank Young would help grapple with the opioid epidemic as well – a scourge that began with the passage of the Compassionate Pain Relief Act (CPRA), passed the year he was appointed. Young, 88, died Nov. 24 of B-cell lymphoma.
Veloxis Pharmaceuticals A/S’ board chairman, Michael Heffernan, said investors will find out in “the next four weeks” more details related to the $1.3 billion takeover by Tokyo-based Asahi Kasei Corp., which gets control of Envarsus XR, an improved formulation of tacrolimus for prophylaxis of organ rejection in kidney transplant patients converted from tacrolimus and for use in de novo kidney transplant patients.
Less than two weeks after giving the go-ahead to Novartis AG for Adakveo (crizanlizumab) to reduce the frequency of vaso-occlusive crises (VOCs) in adult and pediatric patients ages 16 and older with sickle cell disease (SCD), the FDA cleared – well ahead of its Feb. 26, 2020, PDUFA date – Oxbryta (voxelotor), from Global Blood Therapeutics Inc. (GBT), for SCD in adults and pediatric patients ages 12 and up.
On its PDUFA date Thursday, the FDA cleared Xcopri (cenobamate) tablets from South Korea’s SK Biopharmaceuticals Co. Ltd. to treat partial-onset seizures in adults. The drug’s mechanism of action is not fully understood, but it's believed to work through two separate mechanisms: enhancing inhibitory currents through positive modulation of GABA-A receptors and decreasing excitatory currents by inhibiting the persistent sodium current.
