A Cambridge, Massachusetts-based sickle cell disease startup that launched about three years ago has sparked the interest of UK life sciences company Arix, which has agreed to co-lead a $63 million round of financing for the drug developer Imara a few months ahead of the completion of a key Phase II study.
Imara burst into the biotech zeitgeist in June 2016 — as a collaboration between orphan drug accelerator Cydan Development and Lundbeck — with $31 million in Series A funding from a slew of investors, including New Enterprise Associates and Pfizer Venture Investments. Since then it has enlisted the services of Rahul Ballal — who is tapping his business development experience at Northern Biologics and Flexion Therapeutics and his VC chops from Versant Ventures and Novartis Venture Fund — in his role as CEO.
Imara is focused on its sole experimental drug, IMR-687, for sickle cell disease (SCD) — a group of inherited red blood cell disorders that largely afflict those of African ancestry. SCD patients have atypical hemoglobin molecules, which can distort red blood cells into a sickle, or crescent, shape. Symptoms such as anemia, repeated infections and periodic episodes of searing pain begin to appear in early childhood. These episodes deprive the body of oxygen-rich blood, which can culminate in widespread tissue and organ damage, particularly in the lungs, kidneys, spleen, heart and brain, and drastically diminish life expectancy.
IMR-687 — an inhibitor of phosphodiesterase-9 (PDE9i) in blood cells — is designed to to treat the underlying causes of SCD by targeting the same biochemical pathway as standard sickle cell treatment (and chemotherapeutic agent) hydroxyurea, but sans its safety issues. Hydroxyurea makes the red blood cells bigger, helping them stay rounder and more flexible — and makes them less likely to turn into a sickle shape. In animal models, IMR-687 has been shown to increase fetal globin, subverting the polymerization of the sickled hemoglobin — thereby reducing red blood cell sickling, red blood cell death and the occlusion of blood vessels. PDE9 inhibition also diminishes white blood cell “stickiness” which further lessens the blockage of blood vessels, Imara said.
The drug is currently being tested in a mid-stage study, which is expected to be concluded by June. Initial data is expected by the second half of this year, and the full readout in the first quarter of 2020, an Arix spokesperson told Endpoints News.
Other than hydroxyurea — which has been used for decades for sickle cell — Emmaus Medical’s Endari was approved in 2017 for adult and pediatric patients to reduce the acute complications of sickle cell disease. Other developers, including Novartis $NVS, Global Blood Therapeutics $GBT, CRISPR Therapeutics $CRSP/Vertex $VRTX and bluebird bio $BLUE (whose gene therapy-in-development garnered a flurry of attention in a recent 60 minutes episode) are also developing drugs for the orphan disease that affects an estimated 100,000 Americans.
The Series B round for Imara was co-led by new investors Arix and Orbimed Advisors, and included the participation of RA Capital and Rock Springs Capital, as well as existing investors NEA, Pfizer Ventures, Bay City Capital, Lundbeckfonden Ventures and Alexandria Venture Investments. The injection will be used for clinical development for the sickle cell program, as well as broader applications in beta thalassemia and other haematological conditions.
As part of the financing, Arix has committed to invest $15 million for a 10% stake in Imara and Arix’s investment director Mark Chin will join Imara’s board, it said on Monday.
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