JPM: Imara CEO Ballal on fighting sickle cell disease during COVID-19

Rahul Ballal, Ph.D.
(Business Wire)

COVID-19 threw a wrench in Imara’s plans. The company was testing a treatment for sickle cell disease—which occurs more often in people of African descent—during a pandemic that disproportionately affected communities of color. But it learned some lessons along the way that aren’t just useful for its own work, but for anyone working on drugs for diverse populations (so really, anyone working on drugs, period).

“We had the phase 2a data last week and one of the things we saw in that dataset was a large number of missing visits, mostly due to COVID-19,” said Imara CEO Rahul Ballal, Ph.D. “This is an immunocompromised patient population with a chronic disease … So for a clinical trial as opposed to a regular visit, there was a very high barrier for them to come in.”

The company ran its phase 2a study out of a few sites mostly in the U.K. at a time where regulators advised patients with sickle cell disease not to come into clinics. Many patients may not be able to take days off work to get to a clinical trial site, or they may not have access to private transportation and so, must risk exposure on a bus or train to participate in a study.

“Because we have an oral therapeutic, we attempted to ship the drug to patients and do telephone visits, but we couldn’t get the patient samples needed to analyze if the drug was working well,” Ballal said. “It affected our ability to look at the data in a robust way … There were just too many holes to make any conclusions about it.”

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That said, Imara looks forward to testing the drug, IMR-687, in a larger phase 2b program that’s—critically—not taking place during the first wave of COVID-19. The study will take place in 50 sites across the world, including Lebanon, Tunisia, Senegal and Ghana, Ballal said. It will test higher doses of the drug—at least four times higher than the dose tested in the phase 2a study—for longer periods of time and expects the study to read out in the second half of 2021.

IMR-687 is an oral, small-molecule drug that is temperature- and humidity-stable, meaning it doesn’t need refrigeration or cold-chain transport to get where it needs to go, Ballal said.

Accessibility advantages aside, Imara believes its drug can go where approved medicines cannot. The generic drug hydroxyurea works, but not particularly well for various reasons.

“One of which is because it’s cytotoxic. It’s a mutagenic compound and it’s used in cancer treatment,” Ballal said.

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Patients with sickle cell disease have seen two new approvals in recent years: Global Blood Therapeutics’ daily pill Oxbryta—a landmark approval in Ballal’s eyes—and Novartis’ intravenous drug Adakveo. Each addresses a specific facet of sickle cell disease, while Imara’s IMR-687 tackles multiple aspects.

“We sometimes say to other people, we do a little bit of crizanlizumab (Adakveo), a little bit of Oxbryta and a little bit of fetal hemoglobin induction,” Ballal said. “Put together, the thesis is our drug may work in a lot of different ways and may be slightly different in different people. It takes into consideration many mechanisms of action.”

Beyond pushing IMR-687 forward in sickle cell disease and beta-thalassemia, Imara is working to support patients with sickle cell disease and their communities. Under its Real Impacts Grants program, the company awarded $125,000 to 25 community-based organizations serving patients and families affected by sickle cell.

“Imara can’t buy blankets for people. We can’t get them gas cards or fund transportation. There are limits to what biotech companies can do,” Ballal said. But companies can put funds in the hands of organizations that work in those communities, understand people’s needs and support them.

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“We will run the same program in 2021. We’re aiming to do something similar in terms of 25 to 30 organizations, with approximately $5,000 or so each,” Ballal said. And this year, the company hopes to fund organizations outside the U.S.