Exhibit 99.1
Ziopharm Oncology Presents Encouraging Clinical Data for
ControlledIL-12 for the Treatment of Recurrent Glioblastoma
at the 2020 American Society of Clinical Oncology
– Longer termfollow-up from ControlledIL-12 monotherapy studies reinforces encouraging
median overall survival and favorable safety profile –
– ControlledIL-12 in combination withPD-1 inhibitor has favorable safety profile and initial
survival data are encouraging –
– Data again consistent with immune-mediated anti-tumor effects –
Boston, May 29, 2020 —Ziopharm Oncology, Inc. (Nasdaq: ZIOP), today announced the presentation of final clinical data from its phase 1 monotherapy (“Main”) study of ControlledIL-12,Ad-RTS-hIL-12 plus veledimex (Ad+V), as well as updated clinical data from two phase 1 substudies of Ad+V, a monotherapy expansion study (“Expansion”) and a combination study with aPD-1 inhibitor, for the treatment of adult recurrent or progressive glioblastoma multiforme (rGBM) at the 2020 American Society of Clinical Oncology (ASCO) Annual (Virtual) Meeting.
“The results we have seen from the two ControlledIL-12 monotherapy studies are particularly promising, with median overall survival in unifocal patients after monotherapy Ad+V treatment remaining at 16.2 months after longer termfollow-up, as well as encouraging preliminary data from thePD-1 combination study where median overall survival has not yet been reached,” said Dr. Antonio Chiocca, M.D., Ph.D., Trial Investigator and Professor of Neurosurgery at Harvard Medical School, Surgical Director of the Center for Neuro-Oncology at Dana-Farber Cancer Institute, and Chairman of Neurosurgery andCo-Director of the Institute for the Neurosciences at Brigham and Women’s Hospital. “We also reported three additional partial responses, one in the monotherapy Main study, one in the Expansion study and one in the combination study, bringing the total number of partial responses (PRs) to five. Observing responses in brain tumors in the setting of recurrence is unusual and highly encouraging, and, along with the survival data, highlight the potential of Ad+V for the treatment of rGBM.”
Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm, added, “According to most recent data, even with the best available therapies, median overall survival for unifocal rGBM patients appears to be6-12 months. We are therefore heartened by the collection of data presented at ASCO across our three studies, which demonstrate survival benefits beyond a year supported by imaging studies showing tumor regression and biopsies revealing that Ad+V administration turns ‘cold’ tumors ‘hot’ by recruiting T cells into the tumor. We look forward to continuing to reportfollow-up monotherapy and combination phase 1 data, as well as initial data from the ongoing phase 2 study of Ad+V in combination with Libtayo®, which is nearing completion of enrollment.”
Ad-RTS-hIL-12 plus 20 mg/day veledimex is currently being examined in a phase 1 monotherapy “Expansion” substudy for the treatment of rGBM (NCT03679754) that enlarged the phase 1 “Main” veledimex dose escalation trial (NCT02026271) by an additional 36 patients. New clinical results in monotherapy were shared in poster presentations.
Final data highlights from the “Main” dose escalation monotherapy study, titled“Final results of ControlledIL-12 Monotherapy in Adults with Grade III or IV Gliomas,”(Abstract #3040)include:
| • | Subjects (n=6, unifocal, craniotomy) who receivedlow-dose (£ 20 mg cumulative) corticosteroids during veledimex dosing (Days 0 to 14, coinciding with administration of veledimex) had a median overall survival (mOS) of 17.8 months (meanfollow-up of 18.4 months) |
| • | 15 subjects (n=14, unifocal, craniotomy) treated with Ad (Day 0) and 20 mg veledimex with any dosing of corticosteroids had a mOS of 12.7 months (meanfollow-up of 13.1 months) |
| • | Serial MRIs show patient with confirmed PR at 72 weeks, with durability at 96 weeks and monitoring ongoing |
| • | Veledimex-dependent and proportional increases inIL-12 andIFN- |
, resulting in immune activation
| • | Favorable safety profile: |
| • | Ad+V was safely administered and tolerable in both craniotomy (Group 1, n=31) and stereotactic subjects (Group 2, n=7) |
| • | 52 serious adverse event (SAEs) were reported in 21 subjects (55%) and 14 related SAEs were reported in 12 subjects (32%). There have been no study treatment related deaths |
| • | The 20 mg veledimex dose is the recommended phase 2 dose as confirmed in the “Expansion” substudy focusing on veledimex 20 mg (n=36; ASCO 2020 #2564) |
| • | The 10 mg veledimex dose level was studied to move forward as the starting dose in the monotherapy study for pediatric subjects (NCT03330197) and in combination therapy withPD-1 inhibitor in adults with rGBM (ASCO 2020 #2510) |
Data highlights from the “Expansion” study, titled“Survival of Subjects with Recurrent Glioblastoma Receiving Intratumoral Administration of ControlledIL-12 with Limited Exposure to Dexamethasone,”(Abstract #2564)include:
| • | Subjects receiving Ad (Day 0, craniotomy) and 20 mg (Days 0 to 14) veledimex with unifocal disease (“Main” and “Expansion” n=20) administeredlow-dose corticosteroids showed mOS of 16.2 months (meanfollow-up of 14.1 months) |
| • | Serial MRIs show patient with previously reported pseudoprogression now has confirmed PR at 30 weeks and response durability out to 48 weeks(follow-up ongoing), in addition to the PR previously reported1 |
| 1 | Lukaset al. (2019).Survival of Subjects with Recurrent Glioblastoma Receiving Intra-tumoral Administration ofIL-12 Managed withLow-dose Dexamethasone; Society of Neuro-Oncology Annual Meeting |
| • | Adverse reactions remained consistent with previously reported results, being predictable and promptly reversible upon discontinuation of veledimex, and there were no drug-related deaths |
| • | Veledimex dosing compliance was comparable to and slightly higher than the “Main” study |
Combination of Ad+V with thePD-1 inhibitor nivolumab (nivo) is being examined in a phase 1 substudy for the treatment of rGBM (NCT03636477). Data highlights shared in a poster discussion titled“ControlledIL-12 in Combination with aPD-1 Inhibitor: Subjects with Recurrent Glioblastoma”(Abstract #2510)include:
| • | mOS has not been reached, with meanfollow-up at 8.3 months |
| • | Drug-related toxicities were comparable to monotherapy, being predictable, dose-related, and promptly reversible upon discontinuation of veledimex |
| • | As previously reported from Ad+V monotherapy, plasma pharmacokinetics (PK) demonstrates an exposure-response relationship for veledimex |
| • | SerumIL-12 was detected in all subjects following initiation of Ad+V, typically followed by a transient increase in downstream serumIFN- |
| • | There is evidence of immune-mediated anti-tumor effects, with serial MRIs showing pseudoprogression and one new PR, in addition to the PR previously reported2 |
To further investigate Ad+V in combination with an immune checkpoint inhibitor in rGBM subjects, a phase 2 trial of Ad+V in combination with cemiplimab-rwlc (Libtayo®) is currently ongoing (NCT04006119).
More information about ControlledIL-12 is available on the Company’s website athttps://ziopharm.com/controlled-il-12/. Additionally, the posters presented at the ASCO 2020 Virtual Meeting will be available on the Company’s website in the “Scientific and Medical Publications” section.
About Ziopharm Oncology, Inc.
Ziopharm is developingnon-viral and cytokine-driven cell and gene therapies that weaponize the body’s immune system to treat the millions of people globally diagnosed with a solid tumor each year. With its multiplatform approach, Ziopharm is at the forefront of immuno-oncology with a goal to treat any type of solid tumor. Ziopharm’s pipeline is built for commercially scalable, cost effectiveT-cell receptorT-cell therapies based on itsnon-viral Sleeping Beauty gene transfer platform, a precisely controlledIL-12 gene therapy, and rapidly manufactured Sleeping Beauty-enabled CD19-specificCAR-T program. The Company has clinical and strategic partnerships with the National Cancer Institute, The University of Texas MD Anderson Cancer Center and others. For more information, please visit www.ziopharm.com.
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| 2 | Chioccaet al. (2019).PD-1 Inhibition can be Combined withIL-12 in Subjects with Recurrent Glioblastoma; Society of Neuro-Oncology Annual Meeting |
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Investor Relations Contacts:
Ziopharm Oncology:
Chris Taylor
VP, Investor Relations and Corporate Communications
T: 617.502.1881
E:ctaylor@ziopharm.com
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Managing Director
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E:mmoyer@lifesciadvisors.com
Media Relations Contact:
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