After three-decade journey, Geron sees new life in PhIII data on MDS drug, plots path to approval

Geron, founded in 1990 as an anti-aging company, has almost completed its metamorphosis.

Wednesday morning, the Bay Area biotech unveiled that its drug imetelstat succeeded in a Phase III trial of nearly 180 patients with low-risk myelodysplastic syndromes, or MDS, helping more patients go eight weeks without blood transfusions than placebo. But patients who got the drug also experienced significant side effects, including low platelet and white blood cell counts.

MDS is a blood disorder in which the bone marrow doesn’t make red blood cells correctly, and the condition can progress into leukemia.

Geron CEO John Scarlett said the biotech plans to file for approval for imetelstat in low-risk patients with the FDA by the middle of this year and with European authorities later in the year. If approved, Geron hopes to launch its drug on the market in 2024, where it would compete with Bristol Myers Squibb, which already has Reblozyl on the market for a subset of low-risk MDS patients.

In the Phase III trial, 47 patients, or just under 40% of the treatment arm, were transfusion-independent for eight weeks, compared to 15% of the placebo arm. In a key secondary outcome, 28% of patients who got imetelstat were transfusion independent for 24 weeks compared to 3.3%, or just two patients on placebo.

Faye Feller

However, in terms of safety, over 60% of patients who got Geron’s drug experienced grade 3 or 4 low platelet and low white blood cell counts, compared to just a few patients on placebo. Geron CMO Faye Feller did note during an investor call that few of those events resulted in clinical consequences. But in comparison, the most common adverse events with Reblozyl in its Phase III low-risk MDS were fatigue and diarrhea, which impacted approximately one-fourth of patients.

Geron was built around telomeres, these tails that protect the ends of DNA but get shorter the more a cell divides. Imetelstat inhibits telomerase, the enzyme that rebuilds those tails, which scientists say is more active in cancer cells and helps them survive.

J&J was once a partner on imetelstat, penning a deal worth $35 million upfront with $900 million in total milestones in 2014. But in 2018, it dropped Geron, leaving it to finish out its clinical trials on its own.

But now to reach its launch goal, Geron may have to find a partner once again, or find more money. According to its Q3 report, Geron only has enough cash to keep going through the middle of 2024, which is around the time it hopes to launch. When asked in the investor call, Geron’s chief commercial officer Anil Kapur said the biotech was open to talking with potential partners, but didn’t comment further.

Geron’s stock $GERN went up by over 30% this morning, jumping from $2.40 to around $3.40.

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