GSK plc has agreed to pay $1 billion cash up front for Aiolos Bio Inc., plus as much as $400 million in regulatory milestone rewards and tiered royalties. The deal gives London-based GSK asthma candidate AIO-001, a phase II-ready, long-acting antibody that binds to the human thymic stromal lymphopoietin (TSLP) ligand to inhibit TSLP signaling and relieve inflammation. AIO-001 could redefine the standard of care in asthma via the prospect of dosing every six months, the companies said.

Blueprint discontinues early clinical lung cancer candidates, Gavreto marketing outside US and China

Blueprint Medicines Corp. shifted its development priorities away from two of its early clinical therapies for EGFR-mutant non-small-cell lung cancer and made the strategic decision to drop development and commercialization of lung and thyroid cancer therapy Gavreto (pralsetinib) for areas outside of the U.S. and greater China, as it continues to reduce its operating expenses and prepare for increasing sales of Ayvakit and the development of some of its other assets. The San Francisco-based company moved several programs through development in 2023, and “we were able to get to those early phase I datasets or beyond … to allow us to make very strategic investment decisions on the opportunities we think are most compelling in terms of their ability to help patients and drive growth of the company,” said Blueprint CEO, Kate Haviland, during a Jan. 8 presentation at the J.P. Morgan Healthcare Conference.

The year of gene therapy? FDA’s Marks calls for solutions to regulatory, manufacturing challenges

The U.S. FDA is promising to make 2024 a “breakout” 12 months for gene therapies, with a number of initiatives to promote clinical development, approvals and uptake. “This is a great year to focus on gene therapy,” said Peter Marks, director of the Center for Biologics Evaluation and Research at the FDA. With 17 products having passed FDA scrutiny and made it to market, the list is “building nicely,” but now gene therapy as a whole is at a “critical juncture.” Manufacturing challenges and issues with clinical development guidelines, plus differing global regulatory requirements “create a real problem,” Marks said.

Cure thing: Cell and gene therapies innovate but cures aren’t always the goal

Cell and gene therapy companies continue moving away from traditional treatment modalities into a future that’s often unclear. A panel of CEOs said at the Biotech Showcase in San Francisco that instead of aiming for developing silver bullet therapies that knock out indications in a single blow, they tend to only be able to take incremental steps. Some CEOs even said they believe that these new treatments don’t need to be curative as the health care system is not set up to reward that kind of innovation. The CEOs also agreed that partnering is essential to bringing a cell or gene therapy to market.

Innovent’s mazdutide meets endpoints in phase III obesity trial

Innovent Biologics, Inc. said its glucagon-like peptide-1 receptor and glucagon receptor dual agonist, mazdutide, met the primary endpoints and all key secondary endpoints in its first phase III trial in Chinese adults who were overweight or obese. The company plans to submit the first mazdutide NDA for weight management to China's NMPA shortly.

US FDA issues complete response for Astellas’ zolbetuximab BLA

The U.S. FDA issued Astellas Pharma Inc. a complete response letter for its BLA for zolbetuximab, citing unresolved deficiencies following its pre-license inspection of a third-party manufacturing facility for claudin 18.2-targeting drug. “The FDA has not raised any concerns related to the clinical data, including efficacy or safety, of zolbetuximab, and is not requesting additional clinical studies,” Astellas said, adding that it is working closely with the FDA and the third-party manufacturer to establish a timeline to quickly resolve the issue.

Praxis rallies on $279M China deal for tremor drug, pipeline updates

Boston-based Praxis Precision Medicines Inc.’s shares rose nearly 25% on Jan. 8 after it announced a pipeline update and licensing deal for its tremor drug, ulixacaltamide (PRAX-944), with Shanghai’s Tenacia Biotechnology Co. Ltd. Following a foreshadowing of its advancing pipeline, announced on Jan. 8, Praxis stock (NASDAQ:PRAX) soared by 24.85%, or up $6.26, from the day prior, with shares closing at $31.45. “We are thrilled to continue building on the momentum of an exciting 2023 and start the year with four assets in the clinic, including our lead program, ulixacaltamide, and PRAX-628, which continues to show its potential as a best-in-class drug in epilepsy,” Praxis President and CEO Marcio Souza said.

US prices of asthma, COPD inhalers under HELP microscope

When life-saving inhalers sell in Europe at 1.5% to about 8% of their list price in the U.S., they’re bound to attract scrutiny, especially in this era of anger over U.S. prescription drug prices. Senate Health, Education, Labor and Pensions (HELP) Committee Chair Bernie Sanders (I-Vt.) once again expressed that anger yesterday when he announced that the committee is “launching a major investigation into the extremely high prices” Astrazeneca plc, Boehringer Ingelheim GmbH, GSK plc and Teva Pharmaceutical Industries Ltd. charge for inhalers used by millions of Americans with asthma or chronic obstructive pulmonary disease (COPD). Sanders accused the companies of “pump[ing] up their profits by artificially inflating and manipulating the price of asthma inhalers that have been on the market for decades.”

Boehringer backs £30M study to find new treatments for liver disease

Boehringer Ingelheim GmbH has partnered with Newcastle University and the University of Edinburgh to fund a £30 million (US$38 million) study into liver cirrhosis in a bid to provide scientists with new insights into liver health. The partners hope that the study will not only enhance the understanding of nonalcoholic or metabolic dysfunction-associated steatohepatitis cirrhosis, but also help identify translational biomarkers using a cutting-edge approach called single-cell RNA sequencing to accelerate the development of future therapies.

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