The map of cystic fibrosis (CF) research is being redrawn in the U.K. as improvements in treatment, and in particular the introduction of CF modulator drugs, mean people with the rare inherited disease are living much longer. Amongst the many implications, people on modulator drugs do not cough up sputum, making it difficult to diagnose the cause of recurring lung infections; the long-term, chronic inflammation caused by CF is likely to increase the risk of cardiovascular and other diseases; and processes associated with CF, such as oxidative stress and immunosenescence, also are processes associated with accelerated aging.

Uncertainties for Kallyope’s nutrient receptor agonists for obesity

The prospects of an oral obesity candidate used as a monotherapy or combination therapy is in question after Kallyope Inc. posted disappointing phase II data on a government website, indicating that its lead candidate, K-757, failed to meet all endpoints. When used with Kallyope’s K-833, however, the results showed promise, suggesting a combination effort may be the way forward, although data from the combination still fell short of that seen with marketed injectable drugs assessed at a similar time point. Both K-757 and K-833 are first-in-class oral nutrient receptor agonists that were tested in the K-757 P006 trial with the goal of demonstrating weight-loss efficacy, safety and tolerability for patients with obesity.

Mesoblast prices pediatric stem cell treatment at $1.55 million

Regenerative medicine company Mesoblast Ltd. is preparing to launch its allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy, Ryoncil, (remestemcel-L), in March in the U.S. and has priced the treatment at roughly $1.55 million for a full course. The U.S. FDA approved Ryoncil in December 2024 for steroid-refractory acute graft-vs.-host disease (SR-aGvHD) in children 2 months and older, including adolescents. Ryoncil is the only MSC therapy approved in the U.S. for any indication, and the only approved therapy for SR-aGvHD in children 2 months and older. Ryoncil is derived from allogeneic culture-expanded MSCs that have been isolated from bone marrow aspirate collected from healthy human donors.

Biopharmaceutical index drops in 2024, but starts new year on a rebound

The BioWorld Biopharmaceutical Index ended 2024 down 2.24%, despite hitting a peak of 25.19% in late August. Eli Lilly and Co. led the index with a 32% rise over the year, followed by Sarepta Therapeutics Inc. (up 26%), Alnylam Pharmaceuticals Inc. (up 23%), Abbvie Inc. (up 15%) and Gilead Sciences Inc. (up 14%).

Astrazeneca, Olema advance in SERD combo bids

Astrazeneca plc’s positive high-level results made known Feb. 26 from a planned interim analysis of the Serena-6 phase III trial with oral selective estrogen receptor degrader (SERD) camizestrant provided hope Olema Pharmaceuticals Inc., which is pursuing a similar approach. Astrazeneca is testing its SERD in HR-positive breast cancer (BC) with an emergent ESR1 tumor mutation, when combined with one of three CDK4/6 inhibitors. Olema is testing oral palazestrant, which acts as a SERD as well as a complete estrogen receptor antagonist, alongside CKD4/6-blocking ribociclib. In December, Olema updated results from the ongoing phase Ib/II study with the combo in ER-positive, EGFR2-negative advanced or metastatic BC.

FDA accepts Telix BLA for kidney cancer imaging agent

After a second round, the U.S. FDA has accepted for review radiopharmaceutical company Telix Pharmaceuticals Ltd.’s BLA for its kidney cancer PET imaging agent, TLX250-CDx (Zircaix, 89Zr-DFO-girentuximab), granting it a priority review with a PDUFA date of Aug. 27, 2025.

BioWorld Insider Podcast: Mega money well spent? Mammoth mergers fall short of the dream

Billion-dollar M&As are commonplace now, but not too long ago they were a rarity. So many have occurred in the past few years, they’ve become the norm. But were all these multi-billion-dollar mega mergers worth the money? Karen Carey, BioWorld’s senior managing editor and chief analyst, crunched the numbers on 21 of the biggest M&As in a three-part BioWorld series and found very few have been, so far, good deals for the buyer. In this podcast, Carey sifts through the winners and losers and explains the analysis.

Also in the news

Alligator, Allogene, Arcutis, Ardelyx, Arialys, Astrazeneca, Auron, Biocity, Biomx, BMS, Cancervax, Candel, Celltrion, Cognition, Cstone, Eli Lilly, Emalex, Enodia, Enveda, Foresight, Gilead, Glaukos, Idorsia, Ignota Labs, Imunon, Indivior, Innocare, Innovent, Insmed, Invivyd, Madrigal, Mediwound, Moleculin, Novita, Nxera, Opus, PDS, Pfizer, Porosome, Pyxis, Regeneron, Renovaro, Sanofi, Takeda, Telix, Teva, Viatris, Wugen