November 2019 proved to be a fruitful month for patients with blood disorders known as hemoglobinopathies. Within days, the FDA ushered two drugs for sickle cell disease and another for beta thalassemia to the market — livening up a barren field.
Imara, a relatively young plower, is riding on that enthusiasm as it shoots for an $86.25 million IPO.
Imara emerged from New Enterprise Associates’ orphan drug accelerator Cydan in 2016 as a single-product company. $77.3 million in private financing later IMR-687 remains the sole asset in its pipeline; the difference is the drug is now in Phase II for sickle cell disease, with topline data slated for later this year and two other mid-stage beta thalassemia studies lined up.
IMR-687 was one of a group of small molecule PDE9 inhibitors that Imara had licensed from Lundbeck for a song — $1.8 million to-date — after evidence emerged that the class of therapies, originally designed for neurological diseases, could help address abnormalities in red blood cells. Pfizer is also developing a PDE9 inhibitor for SCD after discontinuing a program in Alzheimer’s.
The theory is that blocking PDE9 increases cyclic GMP levels, which is associated with reactivation of fetal hemoglobin and thus restore some functionality impaired in blood disorders.
In contrast, Novartis’ Adakveo is an injection that blocks P-selectin in hopes of stopping vaso-occlusion, while Global Blood Therapeutics’ Oxbryta inhibits hemoglobin S polymerization to prevent red blood cells from forming the sickle shape. Reblozyl, marketed by Celgene (now Bristol-Myers Squibb) and Acceleron, is a fusion protein that boosts red blood cell growth in anemic patients by targeting TGF-beta proteins.
While Reblozyl is only approved for patients who require regular transfusions, Imara hopes to also address those who are not dependent on the procedure.
While allogeneic hematopoietic stem cell transplants can be curative for both sickle cell disease and beta thalassemia, Imara noted that it’s rarely used due to a cumbersome process. Similarly, while gene therapies by bluebird and gene editing approaches being advanced by CRISPR Therapeutics are promising, they are “complex, costly, difficult to administer and potentially only suitable for a limited subset of patients,” execs wrote in the S-1.
CEO Rahul Ballal leads the team of 16. His compensation package last year totaled $2 million, thanks in large part to option awards. CFO Michael Gray received $1.2 million-plus while CEO Willem Scheele got a little over $1 million.
NEA, represented on the board by chairman David Mott as well as Sara Nayeem, remains the largest shareholder at 31.8%. Lundbeckfond Invest holds 16.3%; Pfizer is in for 11.2%, OrbiMed and Arix 10.8%, Bay City Capital 6.4% and RA Capital 5.6%.