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Diffuse Intrinsic Pontine Glioma (DIPG) -How Aggressive the Disease Pipeline is?

Diffuse Intrinsic Pontine Glioma

Ziopharm Oncology recently received the rare paediatric disease for their gene therapy for Diffuse Intrinsic Pontine Glioma (DIPG). The company has the ability to deliver DNA and stimulate a controlled therapeutic immune response with their gene therapy “Ad-RTS-hIL-12”.

In addition to Ziopharm Oncology, many companies are working in this space to bring their treatment to market as quickly as possible with their priority review voucher offered by the FDA. 

DIPG -a childhood brain cancer

Diffuse Intrinsic Pontine Glioma (DIPG) is an incurable pediatric brain tumor that occur in the pons and brainstem and have a peak onset of age between 6–9 years of age. DIPG accounts for 10-15% of all new pediatric brain tumor diagnoses and is the leading cause of brain tumor-related death in children. Recently somatic mutations in the H3F3A gene have been detected in 75% of DIPG cases. H3F3A encodes histone H3 variant H3.3 and the predominant mutation at H3F3A leads to amino acid change at lysine (K) 27 residue of H3.3 to methionine (M). In human cells, H3F3A is one of the two genes that encode H3.3

Key Facts and Unmet needs

  • Diffuse Intrinsic Pontine Glioma (DIPG) is the leading cause of brain tumor-related death in children
  • Median survival of less than one year.
  • Radiation is currently used as standard of care.
  • Chemotherapy has shown no improvements in survival.
  • Despite decades of clinical trials, there has been no improvement in prognosis since the introduction of radiotherapy over thirty years ago
  • The market is too small for targeted drug development in the existing commercial business model

About only Gene Therapy in the pipeline for DIPG

Ad-RTS-hIL-12 is an inducible adenoviral vector encoding human pro-inflammatory cytokine interleukin-12 (IL-12; IL12), which is under the transcriptional control of the RheoSwitch Therapeutic System (RTS) (Ad-RTS-hIL-12), with potential immunomodulating and antineoplastic activities. Controlled IL-12 platform is an investigational gene therapy designed to induce and control the production of human interleukin 12 (hIL-12), a master-regulator of the immune system.

Key findings by Mellalta Meets-


Current Development Status of Ad-RTS-hIL-12”

Ad-RTS-hIL-12Dosing Commenced in Pediatric Brain Tumors Trial

  • 12 patients to be enrolled in Phase 1 DIPG cohort
  • Leading pediatric centers including Lurie Children’s in Chicago; Dana-Farber in Boston; University of California in San Francisco

Leading Companies in DIPG and Current Status

NCT NumberStatusTherapySponsor/CollaboratorsPhases
NCT03566199Active, not recruitingPanobinostat Nanoparticle Formulation MTX110Midatech Pharma US IncPhase 1|Phase 2
NCT03605550RecruitingPTC596PTC TherapeuticsPhase 1
NCT03416530RecruitingONC201Oncoceutics, Inc.Phase 1
NCT02960230RecruitingK27M peptide|NivolumabBristol-Myers SquibbPhase 1|Phase 2
NCT02750891CompletedDSP-7888Sumitomo Dainippon Pharma Co., Ltd.Phase 1|Phase 2
NCT03330197RecruitingAd-RTS-hIL-12ZiopharmPhase 1|Phase 2
NCT04341311RecruitingMarizomib|PanobinostatCelgene|Secura BioPhase 1
NCT03893487RecruitingFimepinostatCuris, Inc.Early Phase 1
NCT03690869RecruitingREGN2810 (monotherapy)Regeneron PharmaceuticalsPhase 1|Phase 2
NCT03355794Active, not recruitingribociclib|EverolimusNovartisPhase 1
NCT01837862RecruitingMebendazoleJanssen PharmaceuticalsPhase 1|Phase 2
NCT03389802RecruitingAPX005MApexigen, Inc.Phase 1
NCT02502708CompletedIndoximod|Temozolomide|EtoposideNewLink Genetics Corporation|Lumos PharmaPhase 1
NCT03709680RecruitingPalbociclib|Temozolomide|IrinotecanPfizerPhase 1


From the insights and findings above, we, Mellalta Meets, conclude that there is an urgent need for therapeutic innovations for the treatment of DIPG, as demonstrated by the multiple treatment modalities evaluated in the first clinical neuro-oncology studies.

There are many companies that have received rare pediatric designations for DIPG including the FDA voucher benefit, which can be redeemed for priority review for any subsequent marketing application such as Mateon Therapeutics, Oblate, Kazia Therapeutics, AbbVie, Life Science Pharmaceuticals, Seattle Genetics, etc. Overall, we believe the DIPG market space is very competitive and future results can only tell how effective these therapies are, including the Ziopharm Oncology Gene Therapy.

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