The majority of patients with acromegaly who switched from therapy with injected somatostatin receptor ligands to Crinetics Pharmaceuticals Inc.’s once-daily, oral drug paltusotine maintained stable levels of insulin-like growth factor 1 in a phase III trial, moving the company’s first-in-class lead closer to becoming the potential new standard of care for the rare hormonal disorder.
As part of its quest to advance its three first-in-class heart failure drug candidates toward the clinic, Corteria Pharmaceuticals SAS has closed an oversubscribed €65 million (US$70.7 million) series A, co-led by investors Orbimed and Jeito Capital, companies based in the U.S. and Europe, respectively.
Messenger RNA, or mRNA, represents a relatively new class of therapeutics with the potential to prevent and treat a wide range of diseases. A well-known success story is of the mRNA vaccines that controlled the COVID-19 pandemic, which has fueled enthusiasm for the field. But biotechs are also developing mRNA candidates for several other infectious diseases, including malaria, tuberculosis and HIV, as well for cancer, autoimmune and allergic diseases. However, delivering nucleic acid therapeutics can be challenging, since mRNA cannot get into cells on its own.
Messenger RNA, or mRNA, represents a relatively new class of therapeutics with the potential to prevent and treat a wide range of diseases. A well-known success story is of the mRNA vaccines that controlled the COVID-19 pandemic, which has fueled enthusiasm for the field. But biotechs are also developing mRNA candidates for several other infectious diseases, including malaria, tuberculosis and HIV, as well for cancer, autoimmune and allergic diseases. However, delivering nucleic acid therapeutics can be challenging, since mRNA cannot get into cells on its own. “Nucleic acid therapeutics can be incredibly fragile,” Thomas Madden, CEO of Acuitas Therapeutics, told BioWorld. “When injected into the body without a delivery system, messenger RNA, for example, is rapidly destroyed.”
Ferring Pharmaceuticals A/S has struck a deal with Royalty Pharma for $500 million, capital it plans to plough into achieving a successful launch of its approved bladder cancer gene therapy, Adstiladrin (nadofaragene firadenovec), in the U.S., as well as enabling it to upgrade and expand its manufacturing sites.
The COVID-19 pandemic might be officially over, but future variants could still pose a threat, and serious health consequences of the causative virus continue to arise, a fact that has prompted the U.S. government to offer Regeneron Pharmaceuticals Inc. about $326 million to develop and manufacture a next-generation COVID-19 monoclonal antibody therapy.
The U.S. FDA approved Pfizer Inc.’s respiratory syncytial virus (RSV) prophylactic, Abrysvo (RSVpreF) for maternal use, providing pregnant women with the option of protecting their newborns up to the age of 6 months against RSV for the first time. The regulator’s Vaccines and Related Biological Products Advisory Committee voted 14-0 in favor of approving the BLA for maternal use of the vaccine in May, a few weeks before the agency gave it the go-ahead for use in older adults (those ages 60 and older).
As a cytokine used in the field of immuno-oncology, interleukin-2 (IL-2) can produce durable and even complete responses in some patients, as well as induce immune memory against tumors. However, its rapid metabolism within the body means it has a short serum half-life, so it needs to be given in high doses, which can trigger severe side effects.
Less than a week after approving Johnson & Johnson’s bispecific antibody, Talvey (talquetamab-tgvs), for relapsed or refractory multiple myeloma (r/r MM) under accelerated review, the U.S. FDA has followed suit with Pfizer Inc.’s equivalent, Elrexfio (elranatamab-bcmm). The accelerated clearance of Elrexfio – a B-cell maturation antigen (BCMA) bispecific antibody that targets BCMA on myeloma cells and the CD3 receptor on T cells – covers it for adult patients who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody.
Critically shortened telomeres are known to be a key limiting factor in successful CAR T-cell immunotherapy, with cells from older patients tending to have shorter telomeres with reduced dividing capacity. As such, these cells are unable to fully eliminate malignancies and provide durable and persistent protection against cancer. So, what if you could lengthen these DNA-protein structures found at the end of chromosomes during the ex-vivo manufacturing of CAR T cells, i.e., before they are put back into the body, so increasing their potency against disease? Newly-launched firm Telos Biotech – a subsidiary company of Cambrian Bio – believes you can, with its patent-protected recombinant protein, Telovance, promising to shake up the cell therapy field.
