- Results from an early study of a personalized cancer vaccine developed by Neon Therapeutics have given the biotech enough confidence to push forward with plans for a Phase 2 trial next year, marking some progress for a field that's drawn increased interest.
- After at least one year of follow-up study, treatment with Neon's vaccine combined with Bristol-Myers Squibb's immunotherapy Opdivo helped shrink tumors in a minority of patients with metastatic melanoma, lung and bladder cancers, results announced Monday showed.
- The response rates, as well as initial measures of progression-free survival, appear on par or slightly better than what past studies have shown for Opdivo monotherapy — a positive sign for Neon's approach. Yet the data are from an open-label, single-arm study, making comparisons difficult, and questions of survival and duration of response remain unanswered.
In an period of rapid advances in immuno-oncology research, cancer vaccines are getting fresh attention as a potential avenue to improving on checkpoint inhibitors like Opdivo (nivolumab).
While Opdivo and other drugs like it work particularly well for a slice of cancer patients, most don't see a greater benefit than what would be expected with existing treatments, depending on tumor type.
Checkpoint inhibitors help to block suppression of immune response to tumors. Adding a cancer vaccine, it's thought, could complement that approach by giving newly active immune cells a map to better target cancers.
Neon Therapeutics is one company testing this hypothesis, as are better-funded rivals like Moderna and the German BioNTech, which this month raised $325 million in Series B funding.
Neon's most advanced candidate, dubbed NEO-PV-01, is custom made for each individual patient, designed to target tumor-specific proteins known as neoantigens.
In the Phase 1 study that read out results Monday, NEO-PV-01 was combined with Opdivo as a treatment for 82 patients with metastatic melanoma, smoking-associated non-small cell lung cancer or bladder cancer.
Data from the small trial showed responses to the combo varied based on tumor type but, in Neon's view, extended the benefit offered by Opdivo alone. On safety, no serious adverse events were observed among the 60 study participants given at least one vaccine dose, Neon said.
Another 22 patients included in the efficacy results presented Monday had received one dose of Opdivo by an April 2019 data cut.
Neon study results for NEO-PV-O1
|Median follow-up||Median PFS*||Response rate||% receiving prior therapy|
|Melanoma (n=34)||13.4 months||Not yet reached||47%||41%|
|Non-small cell lung cancer (n=27)||12.0 months||5.6 months||22%||67%|
|Bladder cancer (n=21)||14.7 months||5.6 months||24%||71%|
*PFS = progression-free survival SOURCE: Company release
"Monotherapy checkpoint inhibitors have historically shown a range in median progression-free survival in metastatic melanoma, non-small cell lung and bladder cancers of approximately 3-7 months, 2-4 months and 2-3 months, respectively," said Patrick Ott, a clinical director at the Dana-Farber Cancer Institute and lead investigator of the study, in a statement prepared by Neon.
While those numbers do reflect studies to date of checkpoint inhibitors, teasing out the effectiveness of NEO-PV-01 versus Opdivo is made difficult by the study's single-arm design.
Caution is needed, too, as signs of added benefit from combination treatments studied elsewhere in immuno-oncology have spurred excitement, only to disappoint when fuller, randomized data are available. (Incyte's epacadostat being the most well-known example.)
Still, Monday's results are the first serious look at Neon's vaccine and were convincing enough for the biotech to set out plans for further study.
Neon said it will start a randomized Phase 2 study next year of NEO-PV-01 plus checkpoint inhibitor therapy in first-line metastatic melanoma patients. Data will also be available next year from another Phase 1 study of the vaccine together with Merck & Co.'s Keytruda (pembrolizumab) in lung cancer, giving Neon another opportunity to showcase its approach's merits.
Shares in Neon were up 5% in mid-morning trading Monday.