What had been viewed as a major catalyst for Gilead Sciences Inc. going into 2024 turned into a disappointment on the stock market, as antibody-drug conjugate Trodelvy (sacituzumab govitecan) failed to meet the overall survival primary endpoint in the phase III Evoke-01 study in previously treated metastatic non-small-cell lung cancer (NSCLC).
Kyverna Therapeutics Inc. disclosed a filing to raise up to $100 million in an IPO, becoming the sixth firm to announce plans for a U.S. listing in the new year, offering tentative hope that the public markets might prove more welcoming to biopharma firms after a lackluster 2023.
“This is a tough business. It’s never a straight line from start to success.” Those words, from Exelixis Inc. CEO Michael Morrissey, during a presentation at the J.P. Morgan Healthcare Conference (JPM), could easily sum up any aspect of the biopharma industry. But with more biopharma firms than ever having reached commercial status, along with the introduction of new therapeutic modalities into the health care market, many are finding the toughest part comes after regulatory approval, whether it’s navigating a competitive landscape, getting payers and physicians on board, or satisfying regulators’ stringent postmarketing requirements. As industry players and observers head home after a busy week in San Francisco, BioWorld offers a brief glimpse at a few firms taking on those post-approval challenges in 2024.
The zeal for antibody-drug conjugates (ADCs), a prominent focus for dealmaking in 2023, shows no signs of waning, as Johnson & Johnson greeted attendees of the J.P. Morgan Healthcare Conference (JPM) with news of its $2 billion buyout of Ambrx Biopharma Inc., picking up rights to an ADC platform along with a promising candidate targeting advanced prostate cancer. Under the terms, J&J agreed to acquire all outstanding shares of San Diego-based Ambrx for $28 apiece, marketing a 105% premium to the firm’s Jan. 5 closing price. Unsurprisingly, Ambrx’s stock (NASDAQ:AMAM) gained 101.5% to close Jan. 8 at $27.47.
With its launch barely three years in the rearview mirror, Remix Therapeutics Inc. has inked its second potential $1 billion pharma collaboration, drawing Roche Holding AG to the table with the promise of its RNA processing platform and the potential to develop small-molecule therapeutics targeting the underlying drivers of disease. At the same time, the Watertown, Mass.-based firm added $60 million in a venture round, bringing its total financing to date to $201 million.
Shares of Corcept Therapeutics Inc. (NASDAQ:CORT) fell 26% Jan. 2 on news that a U.S. court determined that Teva Pharmaceuticals Ltd. does not infringe on patents related to use of cortisol receptor blocker Korlym (mifepristone) in Cushing’s syndrome, opening the possibility of Teva’s generic version to enter the market.
A safe and effective vaccine for preventing respiratory syncytial virus (RSV), a common and sometimes serious respiratory infection, had eluded biopharma for decades. But in 2023, the world saw the first – and second – vaccine hit the market.
The data package for Filsuvez (birch triterpenes), a topical gel aimed at treating partial thickness wounds, satisfied U.S. reviewers the second time around. Chiesi Group said the FDA approved the treatment Dec. 19 for use in patients 6 months and older with rare blistering skin diseases junctional epidermolysis bullosa and dystrophic epidermolysis bullosa.
Following the recommendation of its Oncology Drugs Advisory Committee, the U.S. FDA approved U.S. Worldmeds LLC’s eflornithine 192-mg tablets for use as a maintenance therapy in patients with high-risk neuroblastoma. Branded Iwilfin, the specific, irreversible inhibitor of ornithine decarboxylase previously known as DFMO, is expected to be available in the coming weeks.
While early stage and involving a relatively small patient population, the interim phase Ib readout from the combination cohort testing estrogen receptor (ER)-targeting candidate vepdegestrant in combination with CDK4/6 inhibitor Ibrance (palbociclib) in heavily pretreated patients with ER-positive/HER2-negative breast cancer was impressive enough to prompt partners Arvinas Inc. and Pfizer Inc. to expand development work on the program. The results also struck a chord on the Street, with shares of Arvinas (NASDAQ:ARVN) gaining 31% on the day.
