Far from the big hubs, Apellis is steering its rival to Alexion’s Soliris into a PhIII program

A Kentucky biotech says they’re laying the foundation for a pivotal program for their C3 inhibition therapy, which execs believe can replace Soliris in treating PNH.

Louisville-based Apellis has been making progress on its lead drug far away from the spotlight that concentrates attention on the big biotech hubs. But it’s been well funded, with a $47 million D round that dropped early last year after they gave up on an IPO in chilly market waters. And the company says they’ve been nailing down hard human evidence that by moving upstream from C5 inhibition, where Soliris hits, they can do a better job in controlling anemia and transfusion dependence among patients with this extremely rare condition.

This week, Apellis is reporting on two tiny studies of 3 and 6 patients. In 3 patients never treated with Soliris, investigators reported that all of them experienced a quick correction on a key biomarker for lactate dehydrogenase, or LDH. In 6 patients not responding well to Soliris, the average hemoglobin level was brought up an average of 36%, LDH was corrected and transfusions dropped from 3.4/month on eculizumab monotherapy to 0.3/month when APL-2 was added to eculizumab. And the biotech raised no unusual red flags on the safety side.

As one of the world’s most expensive therapies, Soliris has inspired a range of rivals all looking to replace it with their own drug. Companies like Ra Pharmaceuticals and Akari have been on the trail, while Soliris’ manufacturer, Alexion, has been making advances with a second-gen product for their key moneymaker. ALXN1210 — an anti-C5 antibody that inhibits terminal complement for patients with paroxysmal nocturnal hemoglobinuria (PNH) — was about the only experimental product that earned much respect from new CEO Ludwig Hantson when he took over earlier this year.

Cedric Francois

Apellis has attracted considerable financial support for its work. At the time it filed its S-1, the biotech reported that Morningside Venture Investments owned 32.6% of the company, making the VC their biggest investor. And they say that they’re just getting started with a lead focus on PNH, with a range of other diseases that they believe can be treated through the same pathway.

CEO Cedric Francois said he found the data encouraging as he steers the company to a Phase III study in a few months.

We believe that C3-inhibitor APL-2 can be the next generation PNH treatment offering patients a powerful solution to meaningfully improve their quality of life.

Image: Downtown Louisville, Kentucky Shutterstock

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