Shares of Outlook Therapeutics Inc. (NASDAQ:OTLK), were trading at $1.18, down $3.71, or 75%, on word of preliminary data from Norse Eight, the second phase III trial testing ONS-5010, an ophthalmic formulation of Avastin (bevacizumab, Roche AG) in wet age-related macular degeneration (AMD). ONS-5010 did not meet the prespecified noninferiority endpoint at the eighth week as set forth in the special protocol assessment with the U.S. FDA. But preliminary data showed biologic activity and an improvement in vision, as well as a continued favorable safety profile. Analysis is ongoing as three-month data are being collected, and more should be known in January. Outlook plans to resubmit the BLA for ONS-5010 in the first quarter of 2025. Plans for a potential launch the same year of the drug, approved and branded Lytenava in the U.K. and Germany, are ongoing. The FDA delivered a complete response letter to Outlook in the summer of 2023 due to concerns related to chemistry, manufacturing and controls.

PBC therapy Ocaliva gets conditional nod revoked in Europe

The conditional marketing approval for Ocaliva (obeticholic acid) has been revoked with immediate effect, following a standoff between the EMA and Advanz Pharma Ltd., the company that markets the primary biliary cholangitis (PBC) therapy in Europe. On Sept. 5, Advanz won a short reprieve after challenging the EMA’s June 28 ruling that the marketing authorization for Ocaliva should be revoked, when the General Court of the EU granted a temporary suspension of EMA’s decision. However, on Nov. 27, Advanz announced the court had said it would not be extending the suspension.

Arovella heads toward clinic with CAR-19-iNKT cells

Arovella Therapeutics Ltd. is heading toward the clinic with its lead product, ALA-101, which consists of a chimeric antigen receptor (CAR) targeting CD19 and invariant natural killer T (iNKT) cells. The company is combining its iNKT cell technology with CARs to target blood cancers and solid tumors. Arovella CEO Michael Baker told BioWorld that the combo therapy was more effective than conventional CAR T therapy in preclinical studies. ALA-101 is being developed as an allogeneic cell therapy, and the company has a clear path forward for phase I trials for ALA-101 following a recent pre-IND meeting with the U.S. FDA.

UK bioethics council seeks updated regulations for embryo models

The U.K.’s leading bioethics body is calling for a new law to regulate the generation of stem cell-derived human embryo models and ensure that research does not cross ethical red lines. Currently, there is no statute governing the oversight of these models. In particular, bioethicists say there should be a statutory ban on the transfer of embryo models into the reproductive tract of humans and animals, with legal penalties for contraventions. They also want an explicit ban on the generation of embryo models that have the capacity to feel pain or awareness.

Newly identified signaling pathway affects both ends of energy balance

Researchers at the University of Copenhagen have identified a signaling pathway that simultaneously increased energy expenditure and decreased food intake. In both human and primate studies, agonists of the tachykinin NK2 receptor (NK2R) led to both decreased food intake and increased energy expenditure. And in behavioral tests, they were not aversive, suggesting they do not cause the nausea that is a major side effect of GLP-1 agonists. The findings, which will be published in the Nov. 28, 2024, edition of Nature, surprised even their discoverers in several different ways.

HIF 2024: Immune system could be key to neurodegenerative disease

Our immune cells are not just “defenders” against deadly viruses and pathogens but also a great balancer for tissue homeostasis. For neurological disorders, understanding the neuro-immune axis could be key to treating previously untreatable conditions such as autism spectrum disorder (ASD), according to Jun R. Huh, professor of immunology at Harvard Medical School. Immune cells maintain homeostatic function of many different tissues, including those related to the gut, brain, muscles and even temperature and metabolism. Harnessing the immune system, via activating or suppressing inflammation, could theoretically help correct various historically difficult to treat disorders.

Scholar Rock’s apitegromab set for BLA as 14 phase III wins recorded in October

In October 2024, BioWorld recorded 219 clinical trial updates, including 14 successful phase III trial updates. Shares of Scholar Rock Holding Corp. soared 362% on Oct. 7, after the firm disclosed top-line data from the phase III Sapphire study testing apitegromab in patients with spinal muscular atrophy. Visterra Inc. reported positive top-line data from the ongoing Visionary phase III study of sibeprenlimab, an anti-APRIL monoclonal antibody for immunoglobulin A nephropathy.

Healing the health divide: BioWorld reports on women’s health space

It’s difficult to fathom that the health of half the world’s population is underserved. But it’s a hard truth. Women’s health has traditionally focused on what Seema Kumar, the CEO of Cure, coined as the “bikini zone.” However, reproduction and breast health are a drop in the bucket when it comes to women’s health. In this multipart, in-depth series, BioWorld provides insight into the oft-overlooked space of women’s health, exposing the disparities in research and funding while looking toward future opportunities for bridging the gender gap in health care.

BioWorld Insider podcast: Healing the health divide for women

As a new BioWorld investigative report shows, decades of research excluding women from clinical trials and investment decisions made in male-dominated boardrooms have led to half the world’s health needs being underserved. Of the drug development companies working on women's health solutions, the proportion of funding and partnering for the sector is quite small. But it’s slowly growing, as is the depth of science. Karen Carey, BioWorld’s managing editor and chief analyst, and the managing editor of Bioworld Science, Anette Breindl, join this episode of the BioWorld Insider podcast to talk about the business and the science of women’s health.

Holiday notice

BioWorld's offices will be closed in observance of Thanksgiving in the U.S. No issues will be published Thursday, Nov. 28, or Friday, Nov. 29.

Also in the news

35pharma, Arrowhead, Axsome, Barinthus, Cognition, Genentech, Iovance, Maat, Medigene, Menarini, NS Pharma, Pharmather, Replimune, S.Biomedics, Samsung Bioepis, Soleno, Theratechnologies, Urogen