After a long and bumpy road to approval, Reata Pharmaceuticals Inc. is to roll out the first treatment for Friedreich’s ataxia in the U.S. for patients ages 16 and older after the FDA gave its anti-inflammatory drug, Skyclarys (omaveloxolone), the green light. Read More
The U.S. FDA issued Cytokinetics Inc. a complete response letter (CRL) for omecamtiv mecarbil, branding the clinical evidence behind it as not “persuasive” enough to establish its effectiveness for reducing the risk of heart failure events and cardiovascular death. The company is developing the drug as an add-on therapy for patients with worsening heart failure who remain at high risk for heart failure events and hospitalization. Read More
With a $200 million oversubscribed and upsized series A round completed, Cargo Therapeutics Inc. will advance its autologous CD22 chimeric antigen receptor (CAR) T-cell therapy, CRG-022, which has breakthrough therapy designation in the U.S. “The proceeds are critical for us,” said Gina Chapman, CEO of San Mateo, Calif.-based Cargo. “We will demonstrate manufacturability this year and kick off the pivotal phase II.” Read More
Chroma Medicine Inc. closed a $135 million series B round as momentum builds at one of the early movers in the emerging field of epigenetic editing. It’s little more a year since Cambridge, Mass.-based Chroma emerged from stealth by disclosing a $125 million series A round and a stellar line-up of company founders. “It’s not all that long ago, but we have made a huge amount of progress,” CEO Catherine Stehman-Breen told BioWorld. Read More
Junshi Biosciences Co. Ltd.’s ongericimab, a recombinant humanized anti-PCSK9 monoclonal antibody, met the primary endpoints of reducing the levels of low-density lipoprotein cholesterol in two phase III trials in primary hypercholesterolemia and mixed hyperlipidemia. Read More
Although the U.S. FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously, 12-0, March 1 that the data GSK plc presented was adequate to support the safety of its respiratory syncytial virus vaccine, several panelists cautioned the FDA against viewing the vote as a recommendation to license the vaccine before more data are available. Read More
First the stick and now the carrots. The pharma industry in the U.K. on March 1 published its proposal for a new pricing scheme, under which it is offering to pay a fixed rebate of 6.88% across all eligible drug sales, an offer it said will deliver more than £1 billion (US$1.2 billion) per annum back to the National Health Service. Read More
Oricell Therapeutics Co. Ltd. raised $45 million in a series B1 round to expand in the U.S. market. RTW Investments LP and the Qatar Investment Authority led the financing, which followed the completion of a $125 million series B round in July 2022. Shanghai-based Oricell plans to use the new funds to support the clinical development of its lead candidates, including Oricar-017, in the U.S. Read More
Performing experiments and potentially manufacturing products in space offers some unique advantages in a near-zero gravity environment. Space changes buoyancy, hydrostatic pressure and convective heat flow. Researchers are studying how those changes affect cells, but also looking to take advantage of the changes to create products in manufacturing processes that wouldn’t be possible on earth. Read More
The U.S. House Committee on Oversight and Accountability launched an investigation March 1 into the tactics of pharmacy benefit managers (PBMs). Read More
New hires and promotions in the biopharma industry, including: Bioarctic, Centessa, Kineta, Lipella, Marengo, Poseida, Revance, Strand, Tango. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Aim, Aldeyra, Aptinyx, Arrowhead, Aslan, Cybin, Eloxx, Innovent, Junshi, JW, Merck & Co., Nascent, Qsam. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Alessa, Allogene, Altea, Biostax, Biontech, Celanese, Eisai, ICER, Immune, Immunogen, Vertex. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: BMS, Cstone, Dynavax, Eisai, Incyclix, Jaguar, Laevoroc, Ocugen, Transcode, Verrica, Ymmunobio. Read More
