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FDA grants Orphan Drug Designation to osteosarcoma treatment

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to a treatment for osteosarcoma.
SOT106, developed by biopharmaceutical company SOTIO Biotech, is a next-generation antibody-drug conjugate (ADC) targeting leucine-rich repeat-containing 15 (LRRC15), a clinically validated target broadly expressed across sarcoma subtypes and in tumour associated stroma.
Preclinical data has shown strong anti-tumour activity in both soft tissue and osteosarcoma models and favourable tolerability supporting a high therapeutic index.
Sarcomas are a diverse group of cancers arising in bones and soft tissues, comprising more than 70 distinct subtypes. Their rarity and biological heterogeneity have made therapeutic innovation challenging, including the development of targeted approaches such as ADCs.
Patients are primarily treated with a combination of surgery, radiation and/or chemotherapy, but outcomes remain poor for patients with aggressive, recurrent or metastatic disease. This is especially clear in osteosarcoma, the most common bone cancer in children and adolescents, where a significant number of patients require amputation of the affected limb.
“Orphan Drug Designation for SOT106 underscores both the urgent need for new treatment options in osteosarcoma and the strength of our ADC platform,” said Radek Spisek, Chief Executive Officer of SOTIO.
“Osteosarcoma is a devastating disease that has seen little therapeutic innovation over the past four decades. Treatment continues to rely on intensive chemotherapy regimens associated with significant toxicities and limited long-term benefit.
“We are encouraged by this recognition from the FDA and look forward to advancing SOT106 into the clinic later this year.”
SOTIO says it expects to initiate a first-in-human clinical trial of SOT106 in the second half of 2026.
The post FDA grants Orphan Drug Designation to osteosarcoma treatment appeared first on Drug Discovery World (DDW).
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New webinar: Tackling drug discovery challenges in cancer research

Hosted by Drug Discovery World and supported by Sartorius and BioIVT, this webinar will explore the opportunities and challenges that exist within cancer research drug discovery and development.
You will hear from Dr Sudha Rao, Chief Scientific Officer of Kazia Therapeutics, Karol Budzik, PhD, Business Development Associate at Vyriad Therapeutics and Lars van der Veen, Chief Scientific Officer at iOnctura.
Presentations will cover how cancer treatments have shifted towards reprogramming the biology driving tumour growth, immune escape and treatment resistance, the trajectory that in vivo CAR-T treatments are taking, and how challenging tumours burdened by stroma and immune-mediated resistance can be tackled.
Q&A with the speakers follows the presentations.
The post New webinar: Tackling drug discovery challenges in cancer research appeared first on Drug Discovery World (DDW).
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Psilocybin proves promising in neuropathic pain mouse study
Amid the rise of psychedelics in the mental health space, researchers have begun to explore psilocybin as a treatment for chemotherapy-induced peripheral neuropathy.
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New Spectrometry Technique Could Aid Formulation Development
New Spectrometry Technique Could Aid Formulation Development
A new technique combining two forms of spectrometry could help biopharmaceutical companies improve their choice of formulation buffer for antibody manufacturing by revealing how molecular forms and three-dimensional shapes of complex biologics respond to their environment. That’s the view of Christian Bleiholder, PhD, a professor at Florida State University who helped develop the technique.
According to Bleiholder, what happens structurally when a complex biological molecule, such as an antibody or viral spike protein, binds to its target is currently poorly understood.
“This is where [this approach] can help with the bioprocessing and formulation,” he says, as structural changes “can affect the lifespan [of the product] and lead to issues, such as aggregation.”
Because antibodies are complex, existing techniques tend to be powerful at different levels of complexity, he explains. Mass spectrometry is particularly powerful for distinguishing molecular composition, while structural approaches such as X-ray crystallography and cryo-electron microscopy can provide high-resolution structural information.
The challenge is understanding the link between these things within a heterogeneous sample, he says.
To overcome this, Bleiholder and his team worked with Bruker Daltonics to develop Tandem-Trapped Ion Mobility Spectrometry (Tandem-TIMS). This combines tandem ion mobility spectrometry with tandem mass spectrometry to disentangle three overlapping layers of molecular complexity: molecular form, three-dimensional shape, and binding or assembly state, he says.
He explains that, if the proteins have different structures, they can be characterized with tandem ion mobility spectrometry, and then mass spectrometry can be used to look at their molecular forms and binding states.
Going forward, Bleiholder hopes the technique can be used for formulation development but also earlier, during drug discovery of new products, such as multi-specific antibodies, to determine which molecular states are important and how those change when a biologic engages its target. He also plans to look at automating the technique.
Bleiholder spoke about using Tandem-TIMS at the Bioprocessing Summit in Boston earlier this year.
The post New Spectrometry Technique Could Aid Formulation Development appeared first on GEN – Genetic Engineering and Biotechnology News.
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