In a blast from the past, U.S. President Donald Trump signed an executive order late yesterday to deliver on his 2016 campaign promises and the efforts of his first administration to drive down prescription drug prices. The order tasks the FDA and the Health and Human Services (HHS) secretary with a laundry list of reviews, reports and recommendations that need to be forthcoming over the next several months to pave the way for new pricing models, increased competition and greater access to affordable life-saving medicines. The order also instructs the HHS secretary to work with Congress to improve the Inflation Reduction Act drug price negotiations by doing away with the so-called “pill penalty” that’s hurting investment in small-molecule drugs and improving the transparency of the program.

Duality Biotherapeutics raises $211M with Hong Kong IPO

Antibody-drug conjugate (ADC) developer Duality Biotherapeutics Inc. debuted on the Hong Kong Stock Exchange (HKEX) with a HK$1.64 billion (US$211.4 million) IPO April 15, making it one of the biggest offerings made under the HKEX’s Chapter 18A listing regime since 2020. Shares of Shanghai-based Duality (HKEX:9606) rose to HK$222.00 in intraday trading before closing at HK$205 April 15 – 116.7% above its listing price of HK$94.60. Duality shares dropped 8.68% on April 16, ending at HK$187.20.The IPO marks Duality’s second attempt at a public listing since 2024, after the company let its first securities filing made on Aug. 26, 2024, lapse amid rising tension between the U.S. and China.

Stem cell therapies show safety in clinical trials in Parkinson's

Stem cell implantation is a step closer to becoming the next strategy against Parkinson's disease (PD). Current therapies try to alleviate physical or mental symptoms. PD is commonly treated with dopaminergic agents, such as levodopa combined with carbidopa. Others, including the experimental compound IkT-148009 by Inhibikase Therapeutics Inc. (in phase II) prevents the accumulation of misfolded α-synuclein. However, laboratories are developing other approaches to restore dopamine signaling, such as the implantation of stem cell derivatives that will produce new dopaminergic cells, as seen in two trials presented on April 16, 2025, in Nature.

Glycomine snags $115M to advance its PMM2-CDG treatment

Glycomine secured $115 million in a series C financing round to support the advancement of GLM-101 into a phase IIb study in patients with phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG), an autosomal recessive disorder where the patients have a mutation in the enzyme that converts mannose-6-phosphate to mannose-1-phosphate. Rather than replace the enzyme, GLM-101 replaces the missing mannose-1-phosphate, which is required for the glycosylation of proteins.

M42 enters R&D with investment in longevity firm Juvenescence

The Abu Dhabi health care company M42 is to make an investment in biotech longevity specialist Juvenescence as a route to moving into drug discovery and development, with the two forming a partnership to work together on products that extend the healthy lifespan and improve the treatment of chronic diseases. The investment is part of a $100 million series B1 that Juvenescence is in the process of raising, with the round expected to close in the next two to three months. Although the size of its contribution was not disclosed, Richard Marshall, CEO of Juvenescence, said M42 “will become a very significant investor.” Alongside the discovery and development partnership, M42’s investment gives it an interest in Juvenescence’s inhouse pipeline, with two products in clinical development and three that are due to enter the clinic in 2025, and in the biotech’s portfolio companies, of which there currently are seven.

WHO member states outline pandemic plan

After more than three years of discussion, the World Health Organization’s Intergovernmental Negotiating Body (INB) has agreed on a proposal to prevent, prepare and respond to a pandemic. Particulars include creating a pathogen access and benefit sharing system, building geographically diverse R&D capacities, setting up a coordinating financial mechanism, and establishing a global supply chain and logistics network. The group’s draft agreement now goes to the 78^th World Health Assembly to be held next month. The INB began meeting in 2021, during the COVID-19 pandemic, and has since held 13 formal rounds of meetings.

China’s NMPA accepts Innocare’s zurletrectinib NDA for solid tumors

China’s National Medical Products Administration has accepted Innocare Pharma Ltd.’s NDA for its second-generation pan-tropomyosin receptor kinase (TRK) inhibitor, zurletrectinib (ICP-723), for treating adults and adolescents with advanced solid tumors harboring neurotrophic tyrosine receptor kinase (NTRK) gene fusions. In the phase II registrational trial for adult and adolescent patients (aged 12 to 18) with NTRK fusion-positive solid tumors, zurletrectinib demonstrated strong efficacy with a good safety profile. An overall response rate (ORR) of 80% to 90% was observed in adult patients with various cancers carrying NTRK gene fusion who received dosages of 8 mg or more, Innocare said. Zurletrectinib was also shown to overcome acquired resistance to the first-generation TRK inhibitors.

Commander proteins linked to lysosomal dysfunction in Parkinson’s

Genes associated with lysosomal dysfunction increase the risk of Parkinson’s disease (PD), according to a study led by scientists at Northwestern University. The discovery also explains why some people who carry a pathogenic variant of the GBA1 gene develop PD or dementia with Lewy bodies and others do not. The key lies in the Commander complex, involved in the transport of proteins to this organelle. This discovery raises the need for combinatorial therapies that act on more than one pathway for this type of neurodegenerative disorder. “Carriers of pathogenic genetic variants in Commander genes will have some lysosomal dysfunction, which will contribute to Parkinson’s and potentially other diseases linked to lysosomal function. We focused on Parkinson’s, but we have to see if it is relevant for others,” senior author Dimitri Krainc told BioWorld. Krainc is a professor and the chair of the Department of Neurology at Feinberg School of Medicine, Northwestern University in Chicago, and the director of the Feinberg Neuroscience Institute.

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