The role that genetic mutations play in causing disease is understood, and there are plenty of drug discovery efforts focused on molecules that address such abnormalities. But the human genome also contains genes that suppress mutations, and Scenic Biotech views them as both valuable and druggable. The startup has discovered molecules that hit these novel gene targets and it has raised $31 million to advance their development in cancer and rare diseases.
Scenic’s research focuses on a type of gene called a genetic modifier. Such genes affect how a disease presents and develops, either exacerbating its severity or suppressing it. According to the company, the presence of genetic modifiers helps explain why some people who have a genetic mutation linked to a severe disease end up with mild symptoms or no symptoms at all. Scenic aims to leverage the ability of genetic modifiers to suppress disease.
Genetic modifiers are scattered among the estimated 20,000 genes that make up the human genome. Scenic’s first task is finding them. The company’s Cell-Seq platform identifies genetic modifiers for multiple diseases. Using computational techniques, the technology analyzes genes and produces “disease maps” that can be used to pinpoint potential targets for new drugs.
Amsterdam-based Scenic was founded in 2017, spun out of the Netherlands Cancer Institute and Oxford University. The company’s lead drug candidate is based on research at the cancer institute and Leiden University Medical Center. This immuno-oncology drug targets QPCTL, an enzyme in cancer cells that regulates the function of a protein called CD47. That protein sends out a signal that macrophages, immune cells that chomp on pathogens, read as “don’t eat me.”
The Scenic cancer drug blocks QPCTL to prevent cancer cells from expressing CD47. Consequently, macrophages should be able to recognize and go after the cancer cells. The promise of CD47 as a drug target has led to several large deals. Gilead Sciences paid $4.9 billion to acquire Forty Seven in 2020; Pfizer acquired Trillium Therapeutics for $2.3 billion last year. Other companies developing CD47-targeting drugs include I-Mab and Arch Oncology. All of these efforts focus on antibodies to block the protein. Scenic stands out with a small molecule approach that could have advantages over CD47-targeting antibodies. Small molecules can be formulated as oral drugs. The company also says its small molecule cancer drug may be able to treat solid tumors, which have been a harder target for biologic therapies.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Scenic’s genetic modifier research has also yielded three programs in rare, metabolic disorders. The first is Niemann Pick disease type C, which is disease that impedes the ability of cells to move cholesterol and other fatty substances. The buildup of these substances in various tissues can become fatal. The second rare disease target is Barth syndrome, a mitochondrial disorder that leads to heart problems. These diseases continue to lack any FDA-approved therapies. In the past year, the FDA has rejected Orphayzme’s Neimann-Pick disease type C drug and issued a refuse to file letter to Stealth BioTherapeutics for its Barth syndrome therapeutic candidate.
Scenic’s third rare disease target is an unspecified severe heritable metabolic syndrome. Scenic said the new funding will help advance all three rare disease programs toward the preclinical research that will support investigational new drug applications.
In addition to internal drug pipeline, Scenic has also been collaborating with Roche’s Genentech subsidiary. The partnership began in 2020, with the goal of identifying drug targets in multiple therapeutic areas. Last May, the partners expanded their genetic modifier alliance but disclosed no financial or drug target details. Scenic said in its funding announcement that the collaboration spans six scientific areas.
The Series A round of funding announced Thursday was co-led by Eir Ventures, BioMedPartners, and Vesalius Biocapital. Earlier investors Inkef Capital, BioGeneration Ventures, and Oxford Science Enterprises also participated, alongside Scenic’s founders and management. In the funding announcement, Stephan Christgau, general partner of Eir Ventures, said that Scenic has the potential to become one of Europe’s most exciting biotech companies.
“It is a pioneer in the ground-breaking and promising new field of genetic modifiers,” he said. “We are impressed by the power of its Cell-Seq platform and how it’s fueling a portfolio of in-house and partnered programs across multiple therapeutic areas. This financing will enable Scenic to continue to develop its platform and move its lead program into human clinical trials while bringing its rare disease programs to key value infection points.”
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