In what were essentially “he-said/she-said” presentations this morning, the U.S. FDA and Stealth Biotherapeutics Inc. gave starkly different perspectives on the effectiveness of elamipretide in treating patients with Barth syndrome, an ultra-rare disease that currently affects 129 males in the U.S. While the FDA chalked up improvements seen in the small trials to placebo or effort bias, Stealth officials and Barth experts spent the greater part of their presentations boring holes in the agency’s claims. For instance, Jim Carr, Stealth’s chief clinical development officer, questioned the notion that improvements in functional tests such as the six-minute walk distance were due to effort bias. Barth patients, who suffer a multitude of symptoms including extreme fatigue and muscle weakness, can’t perform better at will, Carr said. And they can’t train their way to better scores, he added. The FDA’s Cardiovascular and Renal Drugs Advisory Committee will weigh in this afternoon with a vote on whether the data Stealth has submitted meets the regulatory requirement of “substantial evidence” of effectiveness.

Ligachem, Ono Pharma ink two ADC deals worth max $700M

Ligachem Bioscience Inc., of Daejeon, South Korea, and Osaka, Japan-based Ono Pharmaceutical Co. Ltd. agreed to two antibody-drug conjugate (ADC) deals that could reach $700 million (₩943.5 billion) combined. Under terms of the first agreement announced Oct. 10, Ono Pharma will gain exclusive global development and commercialization rights to Ligachem’s ADC therapy for solid cancers called LCB-97, a L1 cell adhesion molecule-targeting candidate. With the second deal, Ono will gain exclusive global rights to use Ligachem’s proprietary ADC platform technology, Conjuall, to discover and develop ADC candidates for multiple targets selected by Ono Pharma.

Booster launches with $15M, new approach in proteasome activation

Booster Therapeutics is ready to open up a new arm of the proteasome after raising $15 million in seed funding to advance small molecules it says can degrade multiple types of harmful proteins. Rather than tagging single disease proteins with a ubiquitin marker for degrading via 26S proteasomes, these compounds directly activate 20S proteasomes that naturally recognize disordered proteins without the need for ubiquitin tagging. The aim is to promote restoration of protein homeostasis in complex diseases that are driven by multiple dysfunctional proteins. Berlin-based Booster cites Alzheimer’s and Parkinson’s diseases as examples.

Innocare’s TYK2 inhibitor meets psoriasis endpoints in phase II

Innocare Pharma Ltd.’s tyrosine kinase 2 (TYK2) inhibitor, ICP-488, met the primary endpoint in a phase II trial in Chinese patients with moderate to severe plaque psoriasis, and the results clear the way to accelerate clinical development in psoriasis and other autoimmune diseases. Conducted in China, the multicenter, randomized, double-blind, placebo-controlled phase II trial enrolled 129 patients with moderate to severe plaque psoriasis. Patients were randomly assigned to one of three treatment groups in a 1-to-1-to-1 ratio: a 6-mg once-daily group, a 9-mg once-daily group and a placebo group, for 12 weeks of treatment.

Oncolytic virus therapy may soon change glioma care: Bio Japan

Virus is associated with sickness, but oncolytic virus therapies, which harness viruses to attack and kill cancer cells, may soon change the standard of treatment for cancer, including those long deemed uncurable like malignant glioma. Tomoki Todo, professor of surgical neuro-oncology at the University of Tokyo, was a driving force behind the development of the world’s first approved oncolytic virus therapy for malignant glioma called G47Δ, jointly developed by Tokyo-headquartered Daiichi Sankyo Co. Ltd. and the University of Tokyo’s Institute of Medical Science. During the Bio Japan 2024 meeting, Todo described some of the work involving the therapy to date.

BioWorld Insider podcast: Gene and cell therapies will propel innovation, says Astellas CCO

Gene and cell therapies will drive innovation for the next 10 years, Claus Zieler, the chief commercial officer at Astellas Pharma Inc., said in this episode of the BioWorld Insider podcast. Developers are on the cusp of breakthroughs because a gene can now be replaced “and that means we can potentially cure a disease rather than intervening in a disease.” Zieler also shared his thoughts on creating sustainable health care in aging societies, the innovation cycle gap between the U.S. and Europe, and how collaboration between business and government is critical in forging ahead in this wide-ranging discussion.

Also in the news

Alkem, Alnylam, Alpha Fusion, Anthos, Arda, Aurigene, Bayer, Biogen Bio-Path, Bio-Thera, Boehringer Ingelheim, Can-Fite, Cansino, Capricor, Cardiol, Circle, Ciconia, Clearside, Clock.Bio, Crinetics, Denovo, Dyne, Eli Lilly, Entrada, Gemmabio, Holobiome, Inhibikase, Innocare, Insitro, Insmed, Leads, Maxcyte, Medigene, Neurosense, Nona, Novavax, Ocugen, Qinotto, Panavance, Rege Nephro, Richter, Scholar Rock, Sonnet, Tevogen, Vanqua, Viatris, Viking, Windtree, Xortx, Zealand, Ziphius