A company developing an oral therapy for sickle cell disease has raised more than $60 million in its second major round of financing from several US-based and European investors.
Cambridge, Massachusetts-based Imara said Monday that it had raised the $63 million Series B funding round, led by new investors OrbiMed and Arix Bioscience. Other participants in the round included RA Capital, Rock Springs Capital and existing investors New Enterprise Associates, Pfizer Venture Investments, Lundbeckfonden Ventures, Bay City Capital and Alexandria Venture Investments.
Imara said it planned to use the money to fund development of its lead product candidate, IMR-687, which is currently in a Phase IIa trial in sickle cell disease, and move it into later-stage clinical trials. It also plans to fund the drug’s development as a potential treatment for beta-thalassemia, another blood disorder, and expand its pipeline.
“This is a transformative financing for Imara – one that will allow us to greatly expand our sickle cell disease clinical development, widen our reach into thalassemia and build an exciting pipeline,” CEO Rahul Ballal said in a statement. “We are pleased to be working with leading investors who share our vision for creating a company that helps change the lives of patients living with SCD and other hemoglobinopathies.”
IMR-687 is an inhibitor of phosphodiesterase-9, working on the same pathway as hydroxyurea, a generic chemotherapy drug commonly used to treat blood disorders and blood cancers. The difference, according to the company, is that it doesn’t have the safety issues that come with hydroxyurea. Hydroxyurea’s label carries a boxed warning for severely reduced blood counts and carcinogenicity.
The company announced preclinical and Phase I data in September 2017 showing that IMR-687’s mechanism of action could be effective against the red and white blood cell pathologies associated with SCD and that it could be safely dosed in humans at levels shown as effective in preclinical models.
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.
Several other companies are also developing therapies for SCD and beta-thalassemia. In particular, bluebird bio is developing LentiGlobin, a lentiviral vector-based gene therapy, for both diseases. LentiGlobin is much further along in development for beta-thalassemia, in a Phase III study, while it is in Phase I/II development for SCD. Meanwhile, Acceleron is developing luspatercept, an erythroid maturation agent, for beta-thalassemia and myelodysplastic syndrome, a type of blood cancer, under a partnership with Celgene.
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