Six weeks after the U.S. FDA issued an alert, the EMA is following suit and starting a review of the safety of six approved CAR T-cell cancer therapies, following 23 reports of patients developing secondary cancers. The EMA said such malignancies were considered “an important potential risk” at the time of approval and are included in the risk management plan. Close monitoring is in place, with companies required to conduct long-term safety and efficacy follow-up studies and to file safety update reports. The EMA announcement came after Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said that sequencing of a few of the cases of secondary cancers it is investigating have shown the chimeric antigen receptor transgene is in the malignant clone.

JPM recap: Biopharma rising to postmarketing challenges in 2024

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.

Without enough data, machine learning can lead analyses astray

Researchers have reported that the predictive abilities of a machine learning algorithm trained using best practices on a large clinical dataset did not generalize beyond the data that was used to train it. The algorithm was able to predict, to a degree, which individual patients would benefit from the medication when the patients were from the dataset the algorithm was trained on. But when it was supposed to predict who would benefit in clinical cohorts that were not part of the training, it performed no better than chance. The findings, which appeared in the Jan. 12, 2024, issue of Science, are a reminder that much like in drug development, good work is no guarantee of success.

Merck, Vaxcyte, GSK among players working on better PCVs

Pfizer Inc. has maintained a veritable monopoly on the pneumococcal vaccine (PCV) space for about two decades, but other developers with what they hope are improved products are moving up the clinical ladder. Among them are GSK plc and Vaxcyte Inc., along with Merck & Co. Inc., which boasted during the J.P. Morgan Healthcare Conference about its V-116 PCV, which bears a PDUFA date of June 17.

China’s Ji Xing signs flurry of Asian deals to advance cardiovascular pipeline and global ambitions

Shanghai-based Ji Xing Pharmaceuticals has signed a number of licensing deals over the last week for China rights to cardiovascular drugs to advance its pipeline and its global ambitions, partnering most recently with Tokyo-based TMS Co. Ltd. after the Chinese company acquired global rights for TMS-007 (also known as BIIB-131) from Biogen Inc. TMS-007 is a plasminogen activator with a mechanism of action associated with breaking down blood clots, and it is believed to work by inhibiting local inflammation at the site of thrombosis.

Sermonix out-licenses China rights of breast cancer drug to Henlius 

U.S. biopharma Sermonix Pharmaceuticals Inc. handed off China rights of lasofoxifene, an oral endocrine therapy in development for breast cancer, to Shanghai’s Henlius Biotech Inc., for an undisclosed up-front payment and up to $58 million in milestone fees. The licensing deal announced on Jan. 11 grants Henlius exclusive rights and sublicenses to lasofoxifene in China for at least two estrogen receptor-positive/human epidermal growth factor receptor-2 negative breast cancer indications. 

Biopharma shatters records as 2023 records the highest-ever deal value 

In December biopharma deals reached $23.56 billion, the second-highest monthly total for the year. This reflects an uptick of 25.04% compared to November’s $18.84 billion in deal value. The full year 2023’s deal value of $217.69 billion marked the highest level ever recorded by BioWorld. This was an increase of 5.58% from $206.18 billion recorded in 2022 and surpassed the previous peak, 2021’s $213.57 billion.

Whole genome sequencing improves outcomes in multiple tumor types

A landmark, real-world study in the U.K. has demonstrated that combining whole genome sequencing with clinical data enabled tailored cancer treatment and improved outcomes. At one health care center, having DNA sequence data led to changes from usual standard of care in 25% of cases. “Mostly, [patients] got into clinical trials; some got medicines they wouldn’t have got. Others avoided medicines because their genetic make-up suggested that if they were exposed to the medicines, they would be at risk of harm,” said Mark Caulfield, professor of clinical pharmacology at Queen Mary University of London, who is co-author of a paper outlining the findings in Nature Medicine, Jan 11, 2024.

Holiday notice

BioWorld's offices will be closed in observance of Martin Luther King Jr. Day in the U.S. No issue will be published Monday, Jan. 15.

Also in the news

Ampio, Arena, Bioarctic, Bond, Bridgebio, Chiesi, Debiopharm, Dermavant, Dyne, Elicio, Emergent, Enamin, Genfleet, Henlius, Indapta, Ladrx, Pharmather, Sermonix, Syntheticgestalt, Wuxi, Xcell, Xoma, Zai Lab