September 27, 2024
Company Name
miRecule, Inc.
Location
Gaithersburg, Maryland
Website
I-Corps Cohort
2019 NIH I-Corps short course; Saleh also participated in an I-Corps short course in 2013 with MIMETAS US
Year Founded
2018
I-Corps Participant
Anthony D. Saleh, Ph.D., CEO
Employees
14
What the Company Does/Product(s)
miRecule™ is a biotechnology company developing RNA therapies for cancer and muscular dystrophy.
The company’s proprietary DREAmiR™ platform utilizes genomic and outcome data from thousands of patients to identify underlying genetic changes that cause their disease, and then creates a novel RNA therapeutic that can directly target and fix that genetic abnormality.
miRecule™ is applying the DREAmiR™ platform to develop first-in-class therapies, with lead programs in head and neck cancer and facioscapulohumeral muscular dystrophy (FSHD).
In October 2022, miRecule™ announced a strategic collaboration and exclusive license agreement with Sanofi to develop and commercialize a best-in-class antibody-RNA conjugate (ARC) for the treatment of FSHD. The collaboration marked miRecule’s first licensing transaction leveraging its proprietary DREAmiR platform.
Through the collaboration, miRecule™ received an upfront payment of $30 million, including two milestones, with potential future milestone payments of nearly $400 million in total, in addition to tiered royalties.
In July 2023, Sanofi released a partner spotlight on its partnership with miRecule™ to develop RNA therapeutics for rare diseases.
How I-Corps Helped the Company
“When I participated in I-Corps in 2013 [with MIMETAS US], it was exploratory,” said Saleh. “It was still useful. All those phone calls helped in establishing our business. We found grant partners. It helped us crystallize what we were going to work on first.
“When I did I-Corps with miRecule [in 2019], the program had a great online platform for tracking [customer discovery] interviews. I-Corps helped us connect with people.
“We were developing a head/neck cancer drug. We conducted 40 physician interviews, asking them about the drug and what their unmet needs were. It was super helpful talking to doctors about patients, and speaking with patients as well. We asked which patients might be eligible. What were their outcomes, and what side effects did they experience?
“We also interviewed a number of CMC [chemistry, manufacturing and controls]-related and IND [investigational new drug]-related consultants and organizations to learn, regulatory-wise, what we would need to do.”
Additional Mtech/USMD Programs Utilized
miRecule™ was awarded a Maryland Industrial Partnerships (MIPS) matching grant in February 2022, round 69, to work with Robert Bloch, physiology professor, University of Maryland, Baltimore, on a project titled “MC-DX4 Treatment and Biomarker SLC34A2 for FSHD.”
The Maryland Momentum Fund has invested in miRecule™ twice, each time for $250,000.
Funding Acquired
$8 million: private investors
$6 million: non-dilutive grant funding
$30 million: Sanofi partnership up-front payment plus first two milestones; potential future milestone payments of nearly $400 million total, in addition to tiered royalties
Intellectual Property Owned or Licensed
miRecule™ has six patent families, five of which were developed inside of the company, while one was licensed for its cancer drug.
What’s Next?
miRecule™ is in the pre-clinical phase of its FSHD drug development with Sanofi. The companies are hoping to pick a drug candidate soon. Sanofi will bring the drug through clinical testing and regulatory approval.
