Predictably, weekly vs. daily subcutaneous injections made a big difference in the achondroplasia space, where shares of Copenhagen, Denmark-based Ascendis Pharma A/S (NASDAQ:ASND) closed Sept. 16 at $139.57, up $20.35, or 17%, on favorable, pivotal top-line data with Transcon CNP (navepegritide), as competitor Biomarin Pharmaceutical Inc.’s stock (NASDAQ:BMRN) ended the day at $69.86, down $15.04, or 17.7%.
Genentech’s newly approved multiple sclerosis (MS) injection takes about 10 minutes to administer, dramatically reducing the four to six hours required by its intravenous predecessor. The U.S. FDA approved the humanized monoclonal antibody Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) for relapsing MS and primary progressive MS on Sept. 13.
After missing the primary and secondary endpoints in its phase III study of losmapimod in treating the rare disease facioscapulohumeral muscular dystrophy (FHSD), Fulcrum Therapeutics Inc. is yanking the program’s plug. The selective p38α/β mitogen activated protein kinase inhibitor had a lot of money behind it. It was originally in-licensed from GSK plc and then, in May, Sanofi SA signed on to help Fulcrum develop and commercialize losmapimod for FHSD worldwide, excluding the U.S., in a deal worth $1.06 billion.
The U.S. FDA on Sept. 6 granted fast track designation to MM-II – a novel, non-opioid injectable candidate for knee osteoarthritis (OA) co-developed by Sun Pharmaceutical Industries Ltd. and Moebius Medical Ltd. Mumbai, India-based Sun and Tel Aviv, Israel-based Moebius agreed, under undisclosed terms, to co-develop the novel liposomal non-opioid pain therapy in an exclusive global licensing deal in 2016.
Immortality and eternal youth have been the stuff of myths and legends from ancient times on. Now, in the 21st century, real studies of current medicine could be applied to repair tissues and organs damaged by age. During the 11th Aging Research & Drug Discovery (ARDD) Meeting held at the University of Copenhagen at the end of August, scientists explained the molecular keys of rejuvenation, as many artists imagined in the past.
While Dyne Therapeutics Inc.’s DYNE-251 demonstrated high levels of dystrophin expression and functional improvement in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, investors focused on three serious adverse events related to the drug, driving shares (NASDAQ:DYNE) down by 31% on Sept. 3.
Sanofi SA’s brain-penetrant Bruton's tyrosine kinase (BTK) inhibitor, tolebrutinib, met the primary endpoint in the phase III Hercules trial in non-relapsing secondary progressive multiple sclerosis (nrSPMS). The first compound to show reduction in disability accumulation in MS, tolebrutinib delayed the time to onset of confirmed disability progression in people with nrSPMS, a population for which there are currently no approved therapies.
Aging is part of the life cycle and, although the effects are not manifest until after adulthood, it actually occurs from birth. The concept of senescence has traditionally been associated with aging. However, an embryo has senescent cells. In that case, what is aging, how can it be measured, and from what point in the life cycle?
Since the publication of The Hallmarks of Aging in 2013, aging research has exploded. The field now has more than 300,000 articles on the biological signals of the effect of time on the body. What would Marty McFly, the legendary character from the Back to the Future saga who traveled with his DeLorean time machine from the ‘80s to the ‘50s, think if he visited 2024 and saw laboratories experimenting with techniques to turn back the biological clocks of cells or increase the lifespan of rejuvenated mice?
With a move into Lilly Gateway Labs in Boston’s Seaport District, privately held Tevard Biosciences Inc. is ramping up development of its transfer RNA (tRNA)-based therapies to cure everything from Dravet syndrome and other neurological conditions to cardiology indications and muscular dystrophies.
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