Geron (NASDAQ:GERN) executives said the company is focused on expanding use of its recently launched blood cancer drug RYTELO while preparing for a key myelofibrosis readout later this year, speaking at the annual Bank of America Vegas Healthcare Conference.
Chief Executive Officer Harout Semerjian described Geron as a commercial-stage company focused on hematologic diseases. Its first marketed product, imetelstat, sold under the brand name RYTELO, is a telomerase inhibitor being used in lower-risk myelodysplastic syndromes, or LR-MDS.
RYTELO Launch Metrics Remain Central Focus
Asked what investors should watch as evidence that RYTELO is gaining durable traction, Semerjian said the company’s story is “one of focused execution,” with net revenue as the most important metric.
Geron reported $51.8 million in net revenue in the first quarter, which Semerjian called the company’s “biggest quarter ever.” He also highlighted several commercial metrics the company has been sharing with investors:
- 1,450 U.S. accounts have ordered RYTELO for the first time since launch.
- 33% of sales are now coming from first-line and second-line patients, rather than later-line patients.
- Demand increased 6% quarter over quarter.
- Revenue increased 8% quarter over quarter.
Semerjian said those indicators show Geron’s ability to reach more patients with lower-risk MDS. He added that the company has focused its messaging on the second-line patient population, which it views as the core commercial opportunity today.
In the U.S., Semerjian said there are more than 8,000 second-line lower-risk MDS patients who could benefit from RYTELO. He said the drug’s indication and NCCN Guidelines support use in that setting, while acknowledging that additional patients in later lines of therapy may also benefit.
Geron Emphasizes Cytopenia Management
Chief Medical Officer Joseph Eid said cytopenias associated with RYTELO are “on target” and follow a predictable pattern. He said patients typically experience drops in platelets and neutrophils after about two cycles on average, and that in more than 80% of cases, patients recover within two weeks after the drop.
Eid said data from the IMerge trial and information presented at the American Society of Hematology meeting showed that patients experiencing this cytopenia pattern tended to have more robust responses overall.
“The drug does not work on the symptoms of the MDS, meaning just improvement of anemia. It works on the disease itself,” Eid said.
He said Geron is continuing to educate physicians on managing patients through cytopenias by following the label, including dose adjustments, treatment breaks and resuming therapy when appropriate. Semerjian added that the company is reinforcing the message that cytopenias can be predictable and should be managed so patients can remain on therapy.
Real-World Evidence Data Expected at ASCO and EHA
Geron executives also discussed real-world evidence for RYTELO that is expected to be presented at ASCO and EHA. Eid said the IMerge trial was conducted mainly in European markets, with relatively limited U.S. site participation, making U.S.-based real-world experience important.
The real-world evidence comes from Moffitt Cancer Center and satellite settings, according to Eid. He said the study includes a retrospective component that will be presented and a prospective component that is continuing to enroll patients.
Eid said the data are replicating the results of IMerge in a real-world clinical setting, and in some cases appear better when patients are well managed. He said the findings provide confidence that appropriate management can support outcomes across first-line, second-line, third-line and later-line patient populations.
European Strategy Under Review
Semerjian said RYTELO is fully owned by Geron, which holds global rights. He noted that the drug has received EMA approval, reducing regulatory risk outside the U.S.
However, Semerjian said about 90% of the company’s focus remains on the U.S. launch. Geron is evaluating multiple options for Europe and plans to update the market on its European commercial strategy before the end of the year.
Semerjian said partnerships remain an option. He also said Geron is not looking to follow the traditional European commercial model of building large country-by-country salesforces, citing cash and resource allocation considerations. Instead, the company is considering more restricted partnerships, including potential use of contract sales organizations, while remaining closely involved in areas such as pricing.
He also referenced broader industry volatility and said “MFN is a real topic” that companies are considering.
Myelofibrosis Program Targets Survival Endpoint
Geron’s pipeline discussion focused on IMpactMF, a trial evaluating imetelstat in relapsed or refractory myelofibrosis after JAK inhibitor treatment.
Eid said earlier clinical work in essential thrombocytosis and the IMbark study provided the basis for confidence in myelofibrosis. He said IMbark enrolled 109 patients randomized to high-dose versus low-dose imetelstat and showed symptom improvement, along with what he described as an “almost tripling” of survival.
He also cited indicators of disease modification, including reduction in mutation load, reversal of marrow fibrosis, symptom relief and cytokine reduction.
IMpactMF is designed with two-to-one randomization and an overall survival endpoint, which Eid called unique in the space. The study compares high-dose imetelstat, at 8.9 mg given every three weeks, against best available therapy.
Semerjian said Geron expects an interim myelofibrosis readout in the second half of the year.
About Geron (NASDAQ:GERN)
Geron Corporation (NASDAQ: GERN) is a clinical-stage biotechnology company dedicated to developing and commercializing novel treatments that target telomerase, an enzyme critical to cancer cell immortality. The company’s research is focused on hematologic malignancies and solid tumors, with a pipeline designed to address diseases that have historically had limited therapeutic options.
The lead product candidate, imetelstat, is a first-in-class telomerase inhibitor currently in Phase II and Phase III clinical trials for myelofibrosis and myelodysplastic syndromes.
