At least there is something real and tangible in sight for beleaguered share holders - again the Company today suggesting DIPG data this half.
I would suggest, for sure - the most important data readout, so far from the company - pretty much within weeks. The data will come, with the market cap, at just US$27 m. And seemingly no overhang of an immediate capital raise.
This DIPG data drop, this time is perfectly set up for real share price action. SURELY a positive set of figures - in maybe up to 100 patients in this high profile Phase 2 study - would see the shareprice move about US$100m. ie 80 cents - such a SP though, would be a disgrace, but investors here, used to morsels.
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Its real and its close - but disappointment abounds with this company. What can go wrong ? I don't think it is going to be data.
See some background from the company - in particular the highlighted parts, which you probably are already are aware of. Because of the deadly nature of this disease NOTE in particular though, the short endpoint for overall survival.
Just definitely my views follows .....with this drug combination, it may be that nearly all patients survive longer than 7 months (post that initial radiation treatment). Lets hope so.
See also at very bottom, what the other drug ONC201, is capable of. Very good. Add ONC201 to Paxalisib and the bits of snippets of the success of this combo trial, so far.....will be confirmed
Unusually this PNOC trial extended the number of patients by 100, from an initial recruitment of 217.
Chimerix - the drug company behind ONC201, have in the past....been aggressive in their pursuit of accelerated approval from the FDA, in other drug studies. Robust could be a definition of their dealings in fact, with the FDA, in the past. They can drag Kazia alone - liven our company up, to seek early approval.
In a children cancer study, with likely very positive results from 100 patients - the high profile nature of this disease....a highly credentialed Clinical study FDA Orphan Disease Accreditation- it is likely that the FDA would grant accelerated approval. (well my views at least).
Paxalisib approved - would open the door to immediate and extensive off label use of the drug, given its treatment in studies so far, have always been for late stage patients. And backed up by current positive Phase 1 and 2 studies ..eg paxalisib with radiation - GBM / PLUS vast and compelling research studies.
I read 80% of cancer drug use, is off label. Anyway off label use and its revenue stream - something to talk about later.
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SYDNEY, Oct. 6, 2022 /PRNewswire/ -- Kazia Theriputics
"We are very pleased with progress in the study," commented Dr James Garner, Chief Executive Officer at Kazia. "Brain cancer is the leading cause of childhood cancer death, and DMGs represent one of the most challenging types of tumour. The preclinical data supporting the combination of paxalisib and ONC201 is substantial and persuasive. We look forward to seeing initial data from this ongoing clinical study next year."
Clinical Trial DesignPNOC022 enrols children and young adults with DMGs, a category of brain tumours that includes DIPG. The study will include separate cohorts comprising patients with newly diagnosed disease, patients who have completed initial radiotherapy, and patients who have experienced disease progression after treatment.
All patients will receive some combination of ONC201 with paxalisib. The study employs an adaptive design, in which different arms will be opened and closed based on emerging preclinical and clinical data.
The primary endpoint will be the proportion of patients who are progression-free at six months (PFS6) for newly diagnosed patients, and overall survival at seven months (OS7) for recurrent patients.Professor Dun's work was the subject of an oral presentation at the International Symposium on Pediatric Neuro-Oncology (ISPNO) in Hamburg, Germany, in June 2022. In the ISPNO presentation, Professor Dun outlined an extensive body of preclinical work demonstrating the synergy between ONC201 and paxalisib and described two patients from compassionate use experience who had demonstrated "dramatic reductions in tumor volume and complete resolution of disease symptoms, extending overall survival".
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Preclinical and clinical evaluation of German-sourced ONC201 for the treatment of H3K27M-mutant diffuse intrinsic pontine gliomaKeith Desserich | January 13, 2022
Neuro-Oncology Advances
Dr. Matt Dun, Associate Professor, University of Newcastle
In a study funded by the DIPG/DMG Collaborative, Dr. Dun discusses the initial effects of differing formulations of ONC201.BackgroundDiffuse intrinsic pontine glioma (DIPG) is a fatal childhood brainstem tumor for which radiation is the only treatment. Case studies report a clinical response to ONC201 for patients with H3K27M-mutant gliomas. Oncoceutics (ONC201) is only available in the United States and Japan; however, in Germany, DIPG patients can be prescribed and dispensed a locally produced compound—ONC201 German-sourced ONC201 (GsONC201). Pediatric oncologists face the dilemma of supporting the administration of GsONC201 as conjecture surrounds its authenticity. Therefore, we compared GsONC201 to original ONC201 manufactured by Oncoceutics Inc.
MethodsAuthenticity of GsONC201 was determined by high-resolution mass spectrometry and nuclear magnetic resonance spectroscopy. Biological activity was shown via assessment of on-target effects,
in vitro growth, proliferation, and apoptosis analysis. Patient-derived xenograft mouse models were used to assess plasma and brain tissue pharmacokinetics, pharmacodynamics, and overall survival (OS). The clinical experience of 28 H3K27M+ mutant DIPG patients who received GsONC201 (2017–2020) was analyzed.
ResultsGsONC201 harbored the authentic structure, however, was formulated as a free base rather than the dihydrochloride salt used in clinical trials. GsONC201
in vitro and
in vivo efficacy and drug bioavailability studies showed no difference compared to Oncoceutics ONC201. Patients treated with GsONC201 (n = 28) showed a median OS of 18 months (
P = .0007). GsONC201 patients who underwent reirradiation showed a median OS of 22 months compared to 12 months for GsONC201 patients who did not (
P = .012).
ConclusionsThis study confirms the biological activity of GsONC201 and documents the OS of patients who received the drug; however, GsONC201 was never used as a monotherapy.
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