Our cells do more than replace damaged tissue. They also release molecular signals that can influence how nearby or distant cells behave.
Those released signals are known as a secretome. A secretome is the collection of molecules that cells secrete into their surroundings, including proteins, cytokines, growth factors, lipids and extracellular vesicles.
Noveome Biotherapeutics is developing ST266, an investigational cell-free secretome derived from amniotic membrane progenitor cells. The therapy is currently being evaluated in a Phase I/II trial for necrotizing enterocolitis (NEC), and Noveome has received orphan disease and rare pediatric disease designations for the indication.
In an interview with Xtalks, Karin Potoka, MD, FAAP, Chief Medical Officer at Noveome Biotherapeutics, discussed how Noveome is studying ST266 for NEC, which is a rare and serious intestinal disease that primarily affects premature infants.
She also described how Pittsburgh’s biotech ecosystem, specialized manufacturing talent and rare disease trial strategy are shaping the company’s development path.
The Science Behind ST266
ST266 is cell-free, meaning it is based on the molecules cells release. ST266 is built around the biological signals released by amniotic membrane progenitor cells. Dr. Potoka explained that the platform traces back to fetal surgery observations dating to the 1970s, when clinicians began noticing that tissue operated on before birth did not appear to form the same type of scarring seen in adult wound healing.
“Once that baby was born, there was no scar tissue where that surgery had taken place in utero,” Dr. Potoka said.
Adult tissues can repair injury, but full regeneration is limited. In many cases, healing leads to scar formation or changes in tissue structure.
The 1970 study’s observation pointed researchers toward the fetal environment, including amniotic fluid and the molecules that may support tissue repair. Noveome’s platform is designed around a collection of those cell-released signals in a cell-free biologic product.
Noveome is thinking about supporting the recovery of damaged tissue. ST266 could become relevant across disease processes involving epithelial injury, inflammation and impaired tissue repair.
“We’re able to not only address inflammation, but we’re also able to see anti-apoptotic effects and we’re able to see maturational effects of epithelial cells,” she explained.
Anti-apoptotic effects refer to helping protect cells from programmed cell death. Epithelial maturation is also important because epithelial cells form protective linings in organs such as the intestine.
Why Noveome Is Focused on NEC
NEC, as aforementioned, is a severe inflammatory disease of the intestine that is most often seen in premature newborns.
In severe cases, the condition can lead to tissue death and surgery. Infants who survive may experience long-term complications, including developmental delays or disabilities.
“As a neonatologist, it is an extremely frustrating disease because, like many of the rare neonatal diseases, there are no therapies that have been developed that are targeted specifically at NEC,” said Dr. Potoka.
The company sees NEC as a fit for ST266 because the disease involves several biological processes that the therapy is designed to address, including inflammation, intestinal injury and epithelial immaturity.
Dr. Potoka also noted that NEC can have effects beyond the intestine. Inflammatory processes linked to the disease may contribute to developmental complications in some infants. Noveome is exploring whether ST266’s activity could help dampen these broader inflammatory effects while supporting intestinal recovery.
Pittsburgh’s Role in Noveome’s Development Strategy
Noveome was founded in Pittsburgh and has remained situated there as its platform has developed over roughly two decades.
According to Dr. Potoka, the city has a strong environment for cell- and gene-based therapy work, supported by institutions such as the University of Pittsburgh, UPMC and Carnegie Mellon University.
“I think we’re really seeing a renaissance here in terms of cell and gene-based therapy,” she noted.
The local ecosystem is crucial to rare disease drug development. Noveome needs access to researchers, clinicians, regulatory specialists and manufacturing talent who understand biologics and cell-based production systems.
Dr. Potoka said Noveome has considered how it can expand its capabilities while remaining in Pittsburgh, particularly as it works toward full manufacturing capacity.
“Biologics are so different from your typical small molecule or pharmaceutical manufacturing,” she said. “It takes a different skillset and a different type of team.”
This skillset includes expertise in good manufacturing practices, biologics production, regulatory communication and process development.
Noveome’s team has developed a process supported by more than 70 patents, including the use of bioreactors over more than 18 months to collect paracrine signaling molecules.
Pittsburgh has helped Noveome recruit and retain people with the capabilities needed to support this type of work. She also pointed to the city’s ability to keep talent over time. In her view, people who move to Pittsburgh often choose to stay, which can be important for a small biotech building a specialized team.
Rethinking Trial Sites in Rare Neonatal Disease
Developing treatments for rare neonatal diseases presents unique operational challenges.
Noveome’s ST266 program is being evaluated in a Phase I/II trial. In rare neonatal diseases, Dr. Potoka said trial planning requires a more nuanced approach than simply choosing the largest academic medical centers.
While important, academic institutions are not always the most efficient or practical sites for enrollment.
“In rare disease, especially in neonatal rare disease, you can’t necessarily hang your hat on a large academic institution being the best place to implement your clinical trial,” Dr. Potoka explained.
Noveome considered several factors when selecting trial sites for NEC. These included whether a site was likely to see eligible infants, whether it had clinical trial infrastructure already in place and whether research coordinators could support enrollment and treatment protocols at the bedside.
Smaller clinical trial sites can sometimes be better positioned for bedside recruitment and protocol implementation.
The company also considered geographic and patient diversity. As NEC is multifactorial, risk can be influenced by gestational age, maternal health factors and social determinants that affect preterm birth risk.
Trial site strategy has to account for where eligible infants are likely to receive care and not just where the most prominent investigators are based.
Regulatory Flexibility and Investor Inflection Points
It is well known that rare disease trials can be expensive and difficult to run, particularly when patient populations are small and families have limited treatment options.
Traditional double-blind, placebo-controlled trial designs can be challenging in this setting. Natural history data, open-label designs and smaller modified Phase I/II studies become vital tools for advancing rare disease therapies.
“Allowing us to get away from placebo control, allowing us to use the natural history in these rare diseases and allowing us to do open-label gives us that data that we can use at inflection points for investors,” she said.
Natural history data can help researchers understand how a disease typically progresses without treatment.
Rare disease programs often depend on generating enough clinical evidence to support continued investment.
Dr. Potoka shared that rare disease developers could benefit from more shared learning across companies, clinical teams and patient communities. Many challenges only become visible once a trial is underway, from site activation timelines to bedside enrollment workflows.
“You don’t know what you don’t know until you actually get in there and you start to run a trial,” she said.
Together, Noveome’s ST266 brings secretome biology, neonatal medicine, biologics manufacturing and rare disease trial design into focus as part of a broader effort to advance treatment for an area of significant unmet need.
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