Lack of NAD does not alter muscle function or accelerate aging
Adult skeletal muscle tolerates a lack of the coenzyme nicotinamide adenine dinucleotide (NAD), according to a study led by scientists at the University of Copenhagen. Their results suggest that adverse effects previously associated with congenital NAD depletion in this tissue may be due to impaired muscle development rather than to a deficiency of this molecule.

Lack of NAD does not alter muscle function or accelerate aging
Adult skeletal muscle tolerates a lack of the coenzyme nicotinamide adenine dinucleotide (NAD), according to a study led by scientists at the University of Copenhagen. Their results suggest that adverse effects previously associated with congenital NAD depletion in this tissue may be due to impaired muscle development rather than to a deficiency of this molecule.
Astrazeneca’s AZD-0022 solid preclinical results support further development
Astrazeneca plc recently reported new preclinical data regarding their KRAS G12D inhibitor AZD-0022, currently in phase I/II clinical studies. The company presented the pharmacokinetic and pharmacodynamic profiling of the compound with the aim to push forward with its clinical development.
Mammoth nominates development candidate for FCS and severe hypertriglyceridemia
Mammoth Biosciences Inc. has nominated its first clinical development candidate – MB-111 – a potential one-time treatment for patients with very high triglycerides, including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia. IND-enabling studies are on track to begin this year.
Nutshell gains IND clearance for p53 Y220C reactivator NTS-071
Nutshell Therapeutics (Shanghai) Co. Ltd. has received IND clearance from the FDA to initiate a phase I trial in the U.S. with NTS-071 for anticipated use in solid tumors with a p53 Y220C mutation. The trial is expected to begin in the second half of this year.
Other news to note for May 5, 2025
Additional early-stage research and drug discovery news in brief.
Tyligand develops new dual-action tumor immune agonists for cancer treatment
Tyligand Bioscience Ltd. has reported data on TSN-222, their new cyclic dinucleotide designed as a dual-action tumor immune agonist (DATIA) compound.
Seasonal flu vaccine found to trigger a broadly protective antibody targeting neuraminidase
While current influenza vaccines primarily target the variable hemagglutinin protein, the slower-evolving neuraminidase presents a promising target for the development of new vaccines providing broader protection.
Revolution Medicines patents new GTPase KRAS mutant inhibitors
Revolution Medicines Inc. has disclosed macrocyclic compounds acting as GTPase KRAS (G12V mutant) inhibitors reported to be useful for the treatment of cancer.
KGX-105 shows promising preclinical profile as cancer immunotherapeutic
Kangabio Co. Ltd. has developed and presented data for their EGFRxCD3 TCE prodrug KGX-105 for the treatment of EGFR-expressing solid tumors.

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Conferences

SOT-106: a next-gen LRRC15-directed ADC for sarcoma and aggressive solid tumors
American Association for Cancer Research
The leucine-rich repeat-containing protein 15 (LRRC15) is a cell surface protein involved in cell-cell and cell-matrix interactions, with limited expression in normal tissues.
Bispecific antibody GEN-1057 induces apoptosis through DR4 and FAPα dual engagement
American Association for Cancer Research
The use of mimicking antibodies that activate death receptors (DRs) to selectively induce cell death in cancer cells has shown limited clinical success so far, primarily due to suboptimal efficacy and safety concerns, particularly hepatotoxicity. Emerging strategies to enhance efficacy include the...
Selective BCL6 degrader for B-cell malignancies exhibits robust preclinical activity
New compound
The transcriptional repressor B-cell lymphoma 6 (BCL6) plays a central role in the development and progression of various B-cell malignancies, particularly diffuse large B-cell lymphoma (DLBCL), where it is associated with poor prognosis and resistance to standard immunochemotherapy.
AACR 2025: Induced proximity strategy, beyond degraders
Science
Targeted protein degradation has yet to notch its first approval. But with more than two dozen agents now in clinical trials, the strategy’s ultimate clinical validation appears to be a matter of time.

Cancer

A high-affinity small-molecule inhibitor of STAT3 against triple-negative breast cancer
Degradation inducer
Triple-negative breast cancer (TNBC), which accounts for up to 20% of cases of breast cancer, is one of the most aggressive and difficult to treat subtypes of the disease.

Infection

Key genes and educated natural killer cells keep HIV in remission
Immune
Scientists at Institut Pasteur have gained new insights into how some people control HIV-1 replication after interruption of antiretroviral treatment (ART). The investigators found a fingerprint involved in long-term viral remission.

Nuvobio divulges new peptides to treat C. neoformans infection
Patents
Nuvobio Corp. has synthesized peptides reported to be useful for the treatment of Cryptococcus neoformans infection.
Ciprofloxacin prodrug effectively disrupts biofilm formation
Researchers from Foshan University described a ciprofloxacin prodrug designed to treat biofilm-associated drug-resistant P. aeruginosa infections.

Neurology/psychiatric

Unravel seeks Colombian approval to study RVL-001 for Rett and Pitt-Hopkins syndromes
Regulatory
Unravel Biosciences Inc. has submitted clinical study applications in Colombia seeking to begin proof-of-concept clinical trials for RVL-001 for Rett syndrome and Pitt-Hopkins syndrome.
GW Research discovers new cannabinoid compounds to treat epilepsy and seizures
Patents
GW Research Ltd. has described cannabinoid compounds reported to be useful for the treatment of epilepsy and seizures.
New NMDAR modulator shows promise for pain relief and stress reduction in early animal studies
N-Methyl-D-aspartate (NMDA)-subtype glutamate receptors are an attractive target for chronic pain and posttraumatic stress disorder treatments.
AFC-5128 mitigates acute and chronic EAE via P2X7 receptor antagonism
Multiple sclerosis (MS) is characterized by persistent inflammation, primarily driven by CNS-resident immune cells like microglia, particularly in the progressive stages. The purinergic P2X7 receptor (P2X7R) is widely expressed on immune cells and is elevated on reactive astrocytes and microglia of...
T-cell ANXA2 is potential target in ischemic stroke
Following a stroke, multiple immune cell types contribute to the neuroinflammatory response, with T cells being among the most consistently associated with both the initial and secondary phases of brain injury.

Endocrine/metabolic

Innorna’s mRNA therapy granted US orphan drug designation for Wilson disease
Regulatory
Innorna Co. Ltd.’s investigational mRNA therapy, IN-013, has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of Wilson disease.

Biomarkers

Variants in MED16 gene cause intellectual disability MEDopathy
The Mediator complex is a system that regulates protein-coding gene transcription, where mediator of RNA polymerase II transcription subunit 16 (MED16) is a subunit belonging to this system. Pathogenic genetic variants in Mediator complex subunits usually lead to neurodevelopmental and...

Plasma ANG2 predicts poor outcomes after traumatic brain injury
Conferences
Investigators from the University of Pennsylvania have presented data regarding the relationship of angiopoietin-2 (ANG2) and its prognostic impact on traumatic brain injury (TBI). The ANG2 plasmatic levels were measured in patients with TBI (n=362), orthopedic injury controls (n=89) and healthy...
Ignite Biomedical, Liquid Biosciences discover new biomarkers for substance use disorder
Substance use and poisoning
Ignite Biomedical Inc. and Liquid Biosciences have announced the discovery of biomarkers to diagnose substance use disorder (SUD). The set of biomarkers includes some that have not previously been associated with addiction and spans all 6 SUDs (opioid, cocaine, methamphetamine, alcohol, nicotine...

Gastrointestinal

Equillium advancing new AhR modulator
Inflammatory
Equillium Inc. is advancing EQ-504, a potent aryl hydrocarbon receptor (AhR) modulator acquired through the acquisition of Ariagen Inc., a company that was majority owned by Equillium’s largest investor, Decheng Capital.
Bristol Myers Squibb divulges new RIPK1 inhibitors
Patents
Bristol Myers Squibb Co. has synthesized receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of inflammatory bowel disease, psoriasis, rheumatoid arthritis, nonalcoholic or...
CREG1 protects against acetaminophen-induced liver damage by regulating autophagy
Substance use and poisoning
Acetaminophen (APAP), although recognized as a safe and effective antipyretic and analgesic drug, can lead to acute liver failure if taken in excess.
PVT-401 achieves milestone under Parvus and Abbvie inflammatory bowel disease collaboration
Nanotech
Parvus Therapeutics Inc. has announced the achievement of a milestone under its collaboration with Abbvie Inc. to develop treatments for inflammatory bowel disease (IBD). PVT-401, a novel peptide-major histocompatibility complex (pMHC)...
Germline variants’ impact on pan-cancer proteome
Omics
A large-scale study has revealed the impact of germline variants on proteins in 10 cancer types. Scientists from the National Cancer Institute’s Clinical Proteomic Tumor Analysis Consortium (CPTAC) conducted a precision proteogenomic analysis in a...
Huons Global discovers new reversible H+/K+ ATPase inhibitors
Patents
Huons Global Co. Ltd. has described reversible H+/K+ ATPase inhibitors, particularly acid pump antagonists (APA), reported to be useful for the treatment of gastric and duodenal ulcers, gastritis, gastroesophageal reflux disease, Helicobacter pylori...

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