With the BLA for the same compound in atopic dermatitis (AD) accepted for review by the U.S. FDA, Galderma Holding SA scored clearance from the agency of Nemluvio (nemolizumab) as a prefilled pen for subcutaneous injection to treat adults with prurigo nodularis (PN). A monoclonal antibody directed against the IL-31 receptor alpha, Nemluvio gained breakthrough therapy designation for Zug, Switzerland-based Galderma in December 2019 and priority review in February 2024. PN is an under-diagnosed neuroimmune skin disease that affects up to 181,000 people in the U.S. A decision about the drug in AD is expected later in 2024.

Syros halts enrollment in phase II AML study

Data from an interim analysis has convinced Spyros Therapeutics Inc. to stop enrollment in its phase II study of treating newly diagnosed acute myeloid leukemia and RARA gene overexpression. The company was using tamibarotene, an oral, selective, retinoic acid receptor alpha agonist, combined with Venclexta (venetoclax, Abbvie Inc.) and azacitidine as the treatment. The data revealed a low probability of success for the triple regimen to demonstrate superiority to the combination of only venetoclax and azacitidine. The triple regimen has the U.S. FDA’s fast track designation. Syros’ stock (NASDAQ:SYRS) was shuddering at midday, with shares trading 60% lower at $1.99 each.

Visen’s palopegteriparatide hits endpoints in hypoparathyroidism 

Visen Pharmaceuticals Co. Ltd.’s palopegteriparatide met both primary and secondary endpoints in the phase III Pathway trial conducted in China in adults with chronic hypoparathyroidism, according to top-line data. In the 26-week randomized, double-blind, placebo-controlled Pathway trial, 77.6% (45 of 58) of patients treated with palopegteriparatide achieved the primary multicomponent endpoint compared to 0% (0 of 22) for placebo. The primary multicomponent endpoint is defined as serum calcium levels in the normal range (8.3–10.6 mg/dL) and independence from conventional therapy (requiring no active vitamin D and ≤600 mg/day of oral calcium), with no increase in prescribed study drug within the four weeks prior to the week 26 visit.

IPRs not the US patent killer once feared

With inter partes reviews (IPR) once feared as patent killers, the mere fact that an IPR petition challenging a drug or device patent had been filed with the U.S. Patent and Trademark Office (USPTO) was enough to send a company’s stock tumbling. But in the dozen years since the America Invents Act established IPRs and post-grant reviews, investors and companies have learned to take them in stride. Afterall, of the 10,363 patents challenged from 2012 to Sept. 30, 2023, only 10% – or 1,037 – have been fully invalidated, according to the USPTO.

Tevard’s tRNA therapies gain ground for genetic epilepsies

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 cardiovascular conditions and muscular dystrophies. In fact, Tevard (pronounced Tay-vard) is Dravet spelled backwards. The disease itself is at the heart of the company’s efforts as two of its founders have children with Dravet, a rare and severe epilepsy affecting one in 16,000 individuals, with 85% of cases caused by a mutation in one copy of the SCN1A gene. The gene’s large size means it cannot be delivered by common viral vectors, and its overexpression can lead to harmful effects; therefore, the gene is not amenable to traditional gene therapies. With its tRNA approach, Tevard’s “moving as fast as we can to the clinic,” Daniel Fischer, the company’s cofounder, president and CEO, told BioWorld.

Treating HIV with defective, thieving HIV

A strategy inspired by deficient HIV replication could be used as a treatment to reduce viral load in patients living with HIV and help control the pandemic of the retrovirus. Scientists from the University of California San Francisco want to use HIV against itself by using a parasitic version of the pathogen. They have engineered the virus genome and designed a model of the so-called therapeutic interfering particles of HIV, which was successfully evaluated in human cells, mice and nonhuman primates.

Also in the news

Actinogen, Amylyx, Ascidian, Atara, Avidity, Conduit, Finch, Fore, Halda, Holoclara, Hyloris, Kano, Lobe, Locus, Meiragtx, Pfizer, Plus, Scpharmaceuticals, Seaport, Syndax, Tonix, Xbrane