The medium-sized biopharma is showing off new results from dordaviprone and Zepzelca, both of which were acquired through Jazz Pharmaceuticals’ dealmaking over the last five years.
Rob Iannone showed me the ASCO 2025 name tag he’s wearing. Below the line where it says “Jazz Pharmaceuticals” is a big yellow stripe announcing that Iannone has been a member of the American Society of Clinical Oncology for a quarter of a century.
“I’ve been coming to ASCO for 25 years; that gives you a hint about my age,” he says with a sly smile. When he began attending the annual meeting in 2000, Iannone was a pediatric oncologist. “In that time, I’ve treated patients with high-grade gliomas. Debulking surgeries and radiation therapy was standard of care back then,” he said. “Here we are 25 years later, and the standard of care is debulking surgery and radiation.”
Iannone, chief medical officer at California-based Jazz, is hoping that a new molecule might change that. Acquired in Jazz’s $935 million buy-out of Chimerix in March, dordaviprone is a multi-target small molecule for pediatric gliomas, a particularly aggressive and deadly brain cancer. Jazz was intrigued enough to pay a 72% premium on Chimerix’s stock to get its hands on the molecule, which has an FDA PDUFA date of August 18.
Jazz is at ASCO to present, among other things, new efficacy and safety data from a Phase II trial of dordaviprone. While the drug’s specific indication (H3K27 mutated gliomas) is rare—affecting around 5,000 patients worldwide, according to Truist analysts writing at the time of the Chimerix deal— the potential applicability of the dordaviprone is much broader. Truist modeled a potential $800 million in yearly sales by 2037 for the drug, and noted that its mechanism of action might make it valuable for other cancers beyond gliomas.
The Chimerix deal also brought with it a next-generation version of dordaviprone, termed ONC206, with a similar mechanism and higher potency in vitro.
Iannone said he is chuffed with the results of the acquisition, noting that it “fits in well” with Jazz’s overall pipeline strategy of looking for areas of unmet needs. And Jazz is a good fit for effectively bringing dordaviprone and its successor to the market, Iannone said.
Earlier in his career, Iannone bounced from a professorship in pediatrics, oncology and bone marrow transplantation at the University of Pennsylvania to work at Merck, where he contributed to the development of the mega blockbuster Keytruda. “I was at the right place at the right time” he said about his time on that project. He then had stints at AstraZeneca and Immunomedics before coming to Jazz six years ago. Compared to the tens of thousands of employees marshalled by Merck and AstraZeneca, Jazz employs a modest 3,000–3,500 people, about 800 of which are in research and development.
“We are a little bit of a Goldilocks size,” Iannone said. “We have all the resources we need to accomplish our goals. We’re small enough that we don’t get in our own way. That makes us a favorable partnership, people are surprised at we can do as partners.”
He noted ease of access as one example. “When we’re discussing acquisitions or licensing, companies have direct access to people like me, which might be harder at a bigger company.”
That ease of liaising with other companies has brought Jazz some of its lead molecules, Iannone said. Notably, Jazz licensed its small molecule therapeutic Zepzelca from PharmaMar in 2021 for a scant $5 million up front plus up to $3 million in sales milestones, as well as tiered royalties.
In addition to its presentation on dordaviprone, Jazz is in Chicago this week to present new data from the Phase III IMforte trial of Zepzelca in combination with Roche’s Tecentriq in small cell lung cancer. Adding Zepzelca to Roche’s PD-1 inhbitor improved patients’ median survival by four months compared with Tecentriq alone (17 months versus 13 months, according to Iannone), while reducing patients’ risk of death by 27%.
Most encouraging was a tolerable safety profile. The combo regimen mostly resulted in neutropenia, which Iannone said can be managed and is a solid trade-off for patients who would otherwise face a rough chemotherapy regimen.
The usual treatment regimen involves four to six cycles of chemo, Iannone explained. “Most patients don’t make it to six, maybe they get to four. Toxicity is such that patients stop, and potentially continue on with anti-PD-1 therapy alone. Survival after patients stop chemo drops rapidly, Iannone added. Results from IMforte indicate that Zepzelca could represent a more palatable option.
The companies have already filed a BLA for the combo but the FDA has yet to set a PDUFA date. With the new survival data in hand, “I’d expect to get priority review, which would allow us to get an approval this year,” Iannone said.
