For biotech firms with platform applied sciences, a typical purpose is to seek out a big pharma companion taken with leaping on that platform ultimately. For miRecule, the collaboration got here earlier. Inside 4 years of its founding and nonetheless within the preclinical part, the startup collaborated with Sanofi to develop an RNA remedy for a uncommon muscle illness.

The illness, facioscapulohumeral muscular dystrophy (FSHD), is the second most typical kind of muscular dystrophy after Duchenne muscular dystrophy. However in contrast to Duchenne, FSHD has no FDA-approved therapies. MiRecule co-founder and CEO Anthony Saleh mentioned he linked with Sanofi at a BIO convention, the place the 2 firms realized that they had complementary approaches to R&D for a brand new FSHD remedy.

“They have been already engaged on antibodies that they might use to ship to the muscle,” Saleh mentioned. “They have been additionally taken with FSHD. That's how we bought collectively. We have been going to do it on our personal. However we all know that by working collectively, we might have a greater probability of success.”

The FSHD companion program is now in preclinical lead growth, and miRecule can be making progress on its different RNA remedy applications. That progress was acknowledged at MedCity Information’ INVEST convention in Might, the place miRecule was judged a winner within the biopharmaceutical element of the occasion’s Pitch Excellent startup competitors.

Saleh started his analysis in RNA chemistry at Johns Hopkins College, the place he earned his Ph.D. He continued working with RNA on the Nationwide Institutes of Well being, first as a postdoctoral fellow after which as a scientist within the NIH’s Division of Tumor Biology. Saleh helped create a platform that screened genomic information to seek out targets that could possibly be drugged with RNA therapies. He left the NIH to work at Mimetis, which gives drug discovery and screening companies to pharmaceutical and biotech firms. After a couple of years at Mimetis, Saleh returned to RNA therapies, licensing genomic algorithms and head and neck most cancers analysis he had labored on on the NIH and co-founding miRecule in Gaithersburg, Maryland, in 2018.

miRecule’s expertise platform, DREAmiR, integrates genomic and scientific end result information from private and non-private datasets to establish genetic drivers of illness. With that perception, miRecule designs RNA therapies that correlate with these disease-causing abnormalities. The therapeutic candidate for head and neck most cancers, MC-30, is miRecule’s most superior program. In some sufferers with head and neck most cancers, the tumor suppressor exercise of a sort of RNA referred to as microRNA 30 is misplaced. MC-30 is designed to revive this operate. In preclinical research, outcomes present that this RNA remedy resulted in higher tumor shrinkage and longer responses in comparison with normal therapy, Saleh mentioned. The corporate goals to file an Investigational New Drug Software for this program in 2025.

Whereas miRecule bought its begin in oncology, DREAmiR has potential purposes in a variety of ailments, Saleh mentioned. He credit the corporate’s foray into neuromuscular therapies to early traders who noticed ailments like FSHD as notably amenable to RNA therapies. However FSHD can be private for Saleh, because the illness runs in his household’s genes. After his father was recognized with FSHD, Saleh and a number of other members of the family additionally realized they’ve the dysfunction.

FSHD is attributable to irregular expression of the DUX4 gene. Expression of the DUX4 protein in muscle results in progressively worsening muscle weak point. Nonetheless, in comparison with Duchenne, FSHD progresses rather more slowly and sometimes doesn’t have an effect on the center and lungs. The miRecule FSHD program, referred to as MC-DX4, makes use of microRNA to suppress the expression of the DUX4 gene. This remedy is delivered to muscle cells by a Sanofi nanobody, an engineered antibody that’s a lot smaller than a standard antibody.

MiRecule and Sanofi are getting some competitors in FSHD. Final month, Avidity Biosciences reported early scientific trial outcomes exhibiting that its antibody-guided RNA remedy decreased DUX4 expression and improved muscle operate. Dyne Therapeutics has a preclinical RNA remedy for FSHD, although the corporate has delayed plans for scientific testing whereas it focuses on its extra superior myotonic dystrophy kind 1 program. Avidity and Dyne’s FSHD applications each goal the transferrin receptor. Whereas that concentrate on is plentiful on muscle cells, additionally it is discovered on different kinds of cells. Saleh mentioned miRecule might differentiate itself from different RNA firms with its remedy, which targets a goal discovered solely on muscle cells. That focus on stays unknown.

The Sanofi partnership earned miRecule $30 million in upfront and near-term funds, and will pay the startup as much as $400 million extra if the analysis hits extra milestones. In the meantime, miRecule has one other preclinical partnership with an undisclosed firm. That alliance is creating an immuno-oncology drug for an undisclosed goal. Nancy Sullivan, CEO and managing director of Illinois Ventures and one of many Pitch Excellent judges, mentioned miRecule stood out for its pioneering work in RNA therapeutics and its success in securing companions.

“Their skilled management group and strategic partnerships with main analysis establishments and pharmaceutical firms improve their means to effectively carry novel therapies to market,” Sullivan mentioned.

MiRecule first raised cash in 2021, a $5.7 million seed spherical led by Alexandria Enterprise Investments. With the progress made by its lead applications and extra applications in growth, the corporate is now looking for a Sequence A spherical of funding. Saleh mentioned miRecule is looking for $25 million to $30 million, which might assist present applications in addition to novel applications for myotonic dystrophy kind 1 and glycogen storage ailments.

“We’re positively in search of partnerships,” Saleh mentioned. “However our major focus proper now could be on personal fundraising so we are able to develop our expertise platform and develop extra applications.”

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