For biotech companies with platform technologies, one common goal is finding a big pharmaceutical partner interested in jumping onto that platform sooner or later. For miRecule, the collaboration came sooner. Within four years of its formation and still preclinical, the startup found itself working with Sanofi to develop an RNA therapy for a rare muscle disorder.
The disease, facioscapulohumeral muscular dystrophy (FSHD), is the second most common type of muscular dystrophy behind Duchenne muscular dystrophy. But unlike Duchenne, FSHD has no FDA-approved treatments. MiRecule co-founder and CEO Anthony Saleh said he connected with Sanofi at a BIO conference, where the two companies realized they have complementary approaches to the R&D of a novel FSHD therapy.
“They were already working on antibodies that they can use for delivery to the muscle,” Saleh said. “They were also interested in FSHD. That’s how we came together. We were going to do it on our own. But we know that working together, we would be more likely to be successful.”
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The partnered FSHD program is now in preclinical lead development, and miRecule is also making progress with its other RNA therapy programs. That progress was recognized at MedCity News’s INVEST conference in May, where miRecule was judged the winner in the biopharma track of the event’s Pitch Perfect startup contest.
Saleh started researching RNA chemistry at Johns Hopkins University, where he earned his Ph.D. He continued working with RNA at the National Institutes of Health, first as a postdoctoral researcher and then as a scientist in the NIH’s tumor biology section. Saleh helped to create a platform that screened genomic data to look for targets that can be drugged by RNA therapies. He left the NIH to work at Mimetis, which provides drug discovery and screening services to pharma and biotech companies. After a few years at Mimetis, Saleh returned to RNA therapies, licensing genomic algorithms and the head and neck cancer research he had worked on at the NIH and then co-founding Gaithersburg, Maryland-based miRecule in 2018.
The miRecule technology platform, DREAmiR, integrates genomic and clinical outcomes data from public and private datasets to identify genetic drivers of disease. With that insight, miRecule designs RNA therapies that correlate with those disease-driving abnormalities. The head and neck cancer therapeutic candidate, MC-30, is miRecule’s most advanced program. In some head and neck cancer patients, tumor suppressing activity of a type of RNA called microRNA 30 is lost. MC-30 is intended to restore this function. In preclinical research, results show this RNA therapy resulted in better tumor shrinkage and longer responses compared to standard of care, Saleh said. The company aims to file an investigational new drug application for this program in 2025.
Though miRecule started in oncology, DREAmiR has potential applications in a wide range of diseases, Saleh said. He attributes the company’s pursuit of neuromuscular therapies to early investors who saw diseases such as FSHD as particularly amenable to RNA therapies. But FSHD is also personal to Saleh, as the disease is in his family’s genes. After his father was diagnosed with FSHD, Saleh himself and several family members also learned they have the disorder.
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FSHD is caused by abnormal expression of the DUX4 gene. Expression of DUX4 protein in muscles leads to progressively worsening muscle weakness. However, compared to Duchenne, FSHD progresses much more slowly and usually does not affect the heart and lungs. The miRecule FSHD program, called MC-DX4, uses microRNA to knock down expression of the DUX4 gene. This therapy is delivered to muscle cells by a Sanofi nanobody, an engineered antibody that is much smaller than a traditional antibody.
MiRecule and Sanofi face competition in FSHD. Last month, Avidity Biosciences reported early clinical trial results showing its antibody-guided RNA therapy reduced DUX4 expression and improved muscle function. Dyne Therapeutics has a preclinical RNA therapy for FSHD, though the company has deferred plans for clinical testing while it focuses on its more advanced myotonic dystrophy type 1 program. The Avidity and Dyne FSHD programs both target the transferrin receptor. While this target is abundant on muscle cells, it’s also found on other types of cells. Saleh said miRecule can stand apart from other RNA companies with its therapy, which goes after a target found only in muscle cells. That target remains undisclosed.
The Sanofi partnership brought miRecule $30 million in upfront and near-term milestone payments, and could pay the startup up to $400 million more if the research achieves additional milestones. Meanwhile, miRecule has another preclinical partnership with an undisclosed company. This alliance is developing an immuno-oncology drug for an undisclosed target. Nancy Sullivan, CEO and managing director of Illinois Ventures and one of the Pitch Perfect judges, said miRecule stood out due to its pioneering work in RNA therapeutics and its success landing partners.
“Their experienced leadership team and strategic partnerships with top research institutions and pharmaceutical companies enhance their ability to bring new therapies to market efficiently,” Sullivan said.
MiRecule first raised money in 2021, a $5.7 million seed financing led by Alexandria Venture Investments. With the progress made by its lead programs and more programs in development, the company is now looking to raise a Series A round of funding. Saleh said miRecule is seeking $25 million to $30 million, which would support current programs as well as new ones for myotonic dystrophy type 1 and glycogen storage diseases.
“We definitely are looking for partnerships,” Saleh said. “But our primary focus right now is private fundraising so we can expand our technology platform, develop more programs.”
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