Despite big boasts about the novelty of its 5-hydroxytryptamine6 (5-HT6) pathway approach to treat Alzheimer's disease (AD), Axovant Sciences Ltd. found its phase III effort just as wanting as those of every other biopharma that's sought in recent years to tame the deadly neurodegenerative disease.

Argenx NV, of Breda, the Netherlands, said it started a phase II proof-of-concept trial testing ARGX-113 in pemphigus vulgaris (PV) patients. The open-label, noncontrolled trial will enroll up to 12 patients with mild to moderate PV who are either newly diagnosed or relapsing.

Txcell SA, of Valbonne, France, presented preclinical data at the Congress of the European Society for Organ Transplantation meeting in Barcelona, Spain, describing results with its second-generation CAR-Tregs (HLA-A2 CAR-Tregs) in a graft-vs.-host disease (GvHD) model.

An international research team led by Australia’s Walter and Elizabeth Hall Institute of Medical Research (WEHI) has shown for the first time that carbohydrates on the surface of malaria parasites play a key role in infecting their mosquito and human hosts, which may lead to the development of improved new vaccines.

HONG KONG – CSPC Pharmaceutical Group Ltd. received orphan drug status for its mitoxantrone liposome product from the FDA, though the Chinese company warned that much work remains to get the phase II-stage drug across the finish line in the initial U.S. and China markets.

Regenera Pharma Ltd. CEO Jordan Rubinson told BioWorld that “there’s over $1 billion dollars of market value, of revenues, in the first five years” if the company can succeed in a planned phase III trial and win approval for RPH-201 in nonarteritic anterior ischemic optic neuropathy (NAION), “essentially a stroke of the optic nerve.”

Soligenix Inc., of Princeton, N.J., said it will participate in a research project grant awarded to the University of Hawai’i at Manoa for the development of a thermostabilized Ebola vaccine, with Soligenix awarded funding of about $700,000 over five years.

For the second time this year, the FDA released a draft biosimilar guidance. The latest one describes what type of information a biosimilar sponsor should obtain about the structural/physicochemical and functional attributes of the reference biologic, how that information should be used to develop an analytical similarity assessment plan for the proposed biosimilar and what statistical approaches are recommended for evaluating analytical similarity.

After a multiyear incubation period at New York-based Pfizer Inc., Springworks Therapeutics LLC sprang to life with four Pfizer-derived candidates and an oversubscribed $103 million kitty to see them to the next stage of development. The syndicate for the company’s series A included Bain Capital Life Sciences, Bain Capital Double Impact, Orbimed and Lifearc (formerly MRC Technology), along with Pfizer, which also handed over global rights to the initial compounds.

Fusion Pharmaceuticals Inc., of Hamilton, Ontario, said it completed a second closing of its series A financing, securing an additional $21 million in capital and bringing the total capital raised to $46 million.