Valiltramiprosate

Today, a brief rundown of news involving Amgen and the Parker Institute, as well as updates from J&J, Alzheon and Amylyx Pharmaceuticals that you may have missed.Amgens bispecific cancer antibody Imdelltra helped people with previously treated small cell lung cancer live longer than those who received chemotherapy, the company said Friday. The conclusion was made at an interim analysis of the Phase 3 DeLLphi-304 trial, which enrolled 500 people around the world and randomized half to receive Imdelltra and half to receive standard-of-care chemotherapy. Amgen didnt disclose specific data, but said treatment with Imdelltra led to a statistically significant and clinically meaningful improvement in survival. The data, if confirmed, could help it convert Imdelltras conditional U.S. approval into a full clearance. Jonathan GardnerThe Parker Institute for Cancer Immunotherapy has appointed two well-known researchers to its leadership in recent weeks. In March, the institute announced its new CEO would be Karen Knudsen, who recently served as the CEO of the American Cancer Society and its Cancer Action Network. And on Thursday the Parker Institute appointed Ira Mellman as its president of research. A Genentech veteran, Mellman played a major role in the development of new cancer medicines such as anti-TIGIT antibodies, patient-specific neo-antigen mRNA and DNA vaccines and the immunotherapy Tecentriq, which has been on the market for years. Delilah AlvaradoJohnson & Johnsons experimental autoimmune drug icotrokinra helped 84% of adolescents with psoriasis achieve clear or nearly clear skin after 16 weeks, compared with 27% of those receiving a placebo, the company said Thursday. The data came from an analysis presented at the World Congress of Pediatric Dermatology of the ICONIC-LEAD trial, which enrolled adolescents aged 12 and older along with adults to test the oral peptide drug. Icotrokinra targets IL-23, and if approved, would offer an oral alternative to injectable drugs like AbbVies Skyrizi and J&Js own Tremfya in the skin condition. J&J is developing the drug as part of a collaboration with Protagonist Therapeutics. Jonathan GardnerAlzehons experimental Alzheimers disease drug valiltramiprosate failed to delay cognitive decline in a trial of people with genetic mutations that lead to early onset of the disease, the company said Thursday. The Phase 3 trial enrolled 325 early Alzheimers patients with two copies of the 4 allele of the apolipoprotein E gene and randomized them to take valiltramiprosate or placebo. Researchers found no difference between people given the drug versus those on placebo when examining a measure called ADAS-Cog13, nor on any of the studys secondary endpoints. Trial investigators identified a nominal benefit on ADAS-Cog13 and two other measures among participants who were categorized as having mild cognitive impairment. That difference didnt meet pre-specified statistical analysis goals, however, and would likely need to be confirmed by additional trials before regulators would review valiltramiprosate. Jonathan GardnerAmylyx Pharmaceuticals on Wednesday said it dosed the first participant in a Phase 1 trial of a new treatment for ALS. The company, which previously pulled the ALS drug Relyvrio from market and switched its R&D focus, is looking again to target the neurodegenerative condition. Known as Lumina, the trial will evaluate the tolerability, safety, pharmacokinetics, and pharmacodynamics of Amylyxs new experimental drug, an antisense oligonucleotide targeting the protease calpain-2. The trial will enroll 48 individuals with ALS in the U.S. who will be randomized 3-to-1 to receive either placebo or Amylyxs drug. Early data from the trial is expected this year. Delilah Alvarado '

Alzheon saw signs of efficacy in patients with the earliest symptomatic stage of the disease. \n Sixteen years after tramiprosate flunked a phase 3 trial, Alzheon’s prodrug form of the molecule has joined the lengthy list of pivotal Alzheimer’s disease failures.The biotech picked tramiprosate off the scrap heap after identifying shortcomings with the failed phase 3 study and seeing signs of efficacy in patients with copies of the APOE4 gene. After public investors rebuffed its IPO plans twice in nine months, Alzheon rounded up $150 million across two private rounds and started a phase 3 trial of its valiltramiprosate prodrug formulation.Valiltramiprosate was no better than placebo at slowing cognitive decline, as assessed by ADAS-Cog13, in the latest phase 3 trial, causing the study of 325 people with early Alzheimer’s to miss its primary endpoint. The result favored valiltramiprosate numerically, but the difference fell short of statistical significance. That story repeated across the secondary endpoints. The study drug was numerically but not statistically better than placebo on cognition and function scale CDR-SB, cognitive impairment test MMSE and disability scale DAD. Scores on the daily living scale A-IADL favored placebo.  Following an age-old playbook for failed Alzheimer’s trials, Alzheon looked to a subgroup of patients with the earliest symptomatic stage of the disease for signs of efficacy. The prespecified analysis revealed a nominally statistically significant improvement in cognition versus placebo, as measured by ADAS-Cog13. Valiltramiprosate’s numeric advantage over placebo on other measures widened in the subgroup. Alzheon saw the signs of efficacy in a subpopulation of patients who are poorly served by existing drugs. The study enrolled people with two copies of the APOE4 gene. People with the genes are more likely to develop amyloid-related imaging abnormalities than other patients when taking drugs such as Eisai and Biogen’s Leqembi and Eli Lilly’s Kisunla, a fact the FDA communicated in boxed warnings.Susan Abushakra, M.D., chief medical officer of Alzheon, said in a statement that “a precision medicine approach is key to addressing the needs of Alzheimer’s patients who have the APOE4/4 genotype, and we are committed to this patient population.”