The phase IIb study results of Supernus Pharmaceuticals Inc.’s SPN-820 in treatment-resistant depression had pulled the company’s stock (NASDAQ:SUPN) down 15% at midday. The randomized, double-blind, placebo-controlled study of adults failed to show a statistically significant improvement on the primary endpoint of change from baseline on the Montgomery-Åsberg Depression rating scale. Also, there was no treatment difference between SPN-820 and placebo in the change on the scale from baseline to week 4 for the secondary endpoints. Shares at midday were going for $33.65 each. Supernus had a win on Feb. 5 when the U.S. FDA approved Onapgo (apomorphine hydrochloride; SPN-830), a subcutaneous apomorphine infusion device, for motor fluctuations in adults with advanced Parkinson’s disease.

Developers busy with candidates that could address Wilson disease

Research seems to be gaining ground in Wilson disease (WD), a rare inherited condition that causes copper levels to accumulate in the liver, brain and eyes. Most people are diagnosed as children or around middle age, but the age spectrum can be wide. Monopar Therapeutics Inc. has drawn Wall Street’s attention in the space. Others at work in WD at earlier stages include Arbormed Co. Ltd., Depymed Inc., Mabwell Bioscience Co., Skyline Therapeutics Inc. and Telomir Pharmaceuticals Inc.

Drug developers struggle in 2024; Fibrogen, Acadia, Atai post gains

The BioWorld Drug Developers Index (BDDI) extended its decline through year-end, closing 2024 down 15.25%. After dropping 11.3% by October, the index briefly rebounded, narrowing losses to 5.36% in November, before slipping again. By year-end, 20 of the 30 BDDI companies posted annual losses, with just 10 recording gains. Over November and December, Fibrogen Inc. jumped 77%, with Acadia Pharmaceuticals Inc. (up 26%), Atai Life Sciences NV (up 25%) and TG Therapeutics Inc. (up 20%) also gaining. 

China prioritizes mental health, pediatric care in 2025

China’s National Health Commission (NHC) is designating a three-year period from 2025 to 2027 as the “years of pediatric and mental health services,” in line with a new pledge to achieve eight initiatives in the health care sector aimed at servicing underserved populations. The NHC of the People’s Republic of China (PRC) announced Jan. 2 its intent to bolster both psychiatric and pediatric care for the masses, according to PRC’s official news agency Xinhua, outlining concrete directives to implement the changes. Included in the report was the Beijing-headquartered agency’s mandate for every city in mainland China with secondary (county-level) or tertiary hospitals (city- or provincial-level) to start offering psychological and sleep disorder treatment at outpatient departments by the end of 2025.

GUK1 is metabolic gate in ALK-driven lung cancer

Using ALK+ lung cancer patient-derived cell lines, researchers have performed phosphoproteomic screening and identified guanylate kinase 1 as a TKI sensitive metabolic molecule in ALK-driven lung cancer. They reported their results online in Cell on Feb. 6, 2025. The gene encoding anaplastic lymphoma kinase (ALK) is rearranged (ALK+) in some types of lung cancer. ALK+ lung cancers are sensitive to ALK inhibition using tyrosine kinase inhibitors (TKIs). On the other hand, dysregulated metabolism is a hallmark of cancer cells that leads to the generation of biomolecules crucial for energy production and cell proliferation and survival, but how metabolic dependencies contribute to TKI sensitivity in oncogenic fusion-positive non-small cell lung cancer is not well understood.

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