KZIA Kazia Therapeutics Limited

9.99
-0.19  -2%
Previous Close 10.18
Open 10.17
52 Week Low 2.47
52 Week High 15.85
Market Cap $94,528,737
Shares 9,462,336
Float 9,442,536
Enterprise Value $88,875,730
Volume 215,025
Av. Daily Volume 550,102
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Upcoming Catalysts

Drug Stage Catalyst Date
GDC-0084
Breast cancer brain metastases
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
GDC-0084
Glioblastoma multiforme
Phase 2
Phase 2
Phase 2 data to be presented at SNO meeting November 2020. Median PFS 8.4 months vs 5.3 months for temozolomide; OS 17.5 months vs 12.7 months for temozolomide.
Paxalisib (GDC-0084)
Diffuse Intrinsic Pontine Glioma
Phase 1
Phase 1
Phase 1 initial data presented at SNO Meeting November 2020. Yet to show survival benefit in comparison to historical controls.
GDC-0084
Glioblastoma
Phase 2/3
Phase 2/3
Pivotal trial enrollment to commence 1Q 2021.

Latest News

  1. SYDNEY, Nov. 17, 2020 /PRNewswire/ -- Kazia Therapeutics Limited ((ASX: KZA, NASDAQ:KZIA), an Australian oncology-focused biotechnology company, is pleased to share a summary of new paxalisib data presented at the Society for Neuro-Oncology (SNO) Annual Meeting, which is being held virtually from 19-21 November 2020.

    Key Points

    • New interim analysis of paxalisib phase II study in glioblastoma (NCT03522298) is highly consistent with prior data
    • Median progression-free survival (PFS) of 8.4 months reported on this analysis (versus 5.3 months for temozolomide, the existing standard of care)
    • Median overall survival (OS) of 17.5 months reported (versus 12.7 months for temozolomide)
    • First substantial presentation of safety data at a 60mg dose shows…

    SYDNEY, Nov. 17, 2020 /PRNewswire/ -- Kazia Therapeutics Limited ((ASX: KZA, NASDAQ:KZIA), an Australian oncology-focused biotechnology company, is pleased to share a summary of new paxalisib data presented at the Society for Neuro-Oncology (SNO) Annual Meeting, which is being held virtually from 19-21 November 2020.

    Key Points

    • New interim analysis of paxalisib phase II study in glioblastoma (NCT03522298) is highly consistent with prior data
    • Median progression-free survival (PFS) of 8.4 months reported on this analysis (versus 5.3 months for temozolomide, the existing standard of care)
    • Median overall survival (OS) of 17.5 months reported (versus 12.7 months for temozolomide)
    • First substantial presentation of safety data at a 60mg dose shows profile very similar to prior experience, with the most common toxicities including rash, stomatitis (mouth ulcers), and hyperglycemia (high blood sugar), consistent with other PI3K and mTOR inhibitors
    • Phase I study in DIPG (NCT03696355) shows paediatric maximum tolerated dose (MTD) of 27 mg/m2, with safety profile and pharmacokinetics similar to adult data

    Kazia CEO, Dr James Garner, commented, "this is very reassuring data from the glioblastoma study, confirming our earlier results with the data now much more mature. In studies such as this, volatility is the enemy of dependability. From the very first efficacy data we reported from this study, in November 2019, through the ASCO and AACR presentations in June 2020, to today's latest analysis, the PFS and OS figures have remained extremely stable as the study has progressed. This gives us a great deal of confidence that what we are seeing is representative and reliable."

    He added, "we expect this study to conclude in the first half of calendar 2021, but it has already provided useful information to guide the development of paxalisib. We have moved into the operational phase of the GBM AGILE pivotal study, and we expect that study to now be the primary focus of our work in glioblastoma from this point forward."

    The poster presentation is available for download via the Kazia website at:-

    https://www.kaziatherapeutics.com/researchpipeline/paxalisib

    Summary of Paxalisib Data in Comparison to Temozolomide (existing standard of care)



    Temozolomide[1]

    (FDA-approved treatment)

    Paxalisib

    (interim phase II data)

    Progression-Free Survival (PFS)

    5.3 months

    8.4 months

    Overall Survival (OS)

    12.7 months

    17.5 months

    Professor Patrick Wen, the first author on the poster, commented "as this study has matured, we have seen encouraging results that are very stable over successive analyses, and very consistent with prior clinical experience in this drug. Paxalisib is now moving into the GBM AGILE study in glioblastoma, and we expect this to provide definitive data regarding the drug's potential use in this disease and, if successful, a basis for regulatory approval. There remains a profound need for new treatments in glioblastoma, and paxalisib has proven to be an exciting potential candidate."

    Initial Data from St Jude Study of Paxalisib in DIPG and Diffuse Midline Gliomas

    Dr Christopher Tinkle, lead investigator for the SJPI3K study of paxalisib in DIPG and diffuse midline glioma (NCT03696355), gave an invited oral presentation on interim results from that study.

    The SJPI3K study is a first-in-paediatric study, designed to establish the safety and pharmacokinetics of paxalisib in children, and to explore potential early signals of efficacy in this patient population.

    The study recruited 27 patients, ranging from 3 to 16 years of age. Four patients discontinued participation prior to receiving a first dose of paxalisib, generally due to disease progression. At the time of analysis, five patients remain on paxalisib treatment, and several patients remain in post-treatment follow-up.

    The paediatric maximum tolerated dose (MTD) was determined to be 27 mg/m2. The dose-limiting toxicities (DLTs) included hyperglycaemia, oral mucositis, and rash, which are entirely consistent with the adult experience.

    The pharmacokinetics of the drug, a term which describes the concentration of the drug in plasma over time, was very consistent with the adult experience. The study found no meaningful difference between administration of intact capsules and administration via opening of capsules and sprinkling of contents onto a food carrier.

    The study has not at this stage shown a clear survival benefit for paxalisib in comparison to historical controls. In terms of PFS, the proportion of patients alive and progression-free at six months (PFS6) was 96%, which compares favourably to an historical control of 58%[2]. However, the authors note that PFS can be a complex endpoint to interpret in DIPG trials due to the confounding effect of incidental radiological changes associated with radiation therapy.

    Dr Tinkle commented, "my colleagues and I are very pleased with the outcome of this study. We have determined an appropriate dose for future paediatric work, established an acceptable tolerability profile in children, and demonstrated pharmacokinetic equivalence between intact capsule and open and sprinkled administration, which are critical steps in the development of any new drug for paediatric cancer."

    He added, "DIPG is an extremely treatment-resistant disease, and no drug has ever shown convincing efficacy as a monotherapy. Our view has always been that the treatment of this disease will consist in combination therapy, and we have shown that paxalisib is eminently suitable to now be evaluated alongside other agents. We look forward to discussing follow-on work that will explore these opportunities and further investigate paxalisib's potential."

    Dr Garner commented, "we are grateful to have had the opportunity to collaborate with one of the world's leading paediatric oncology hospitals in this study. The results provide an excellent foundation for the further development of paxalisib in DIPG, and we will be excited to discuss the next phase of work with our collaborators in coming months."

    Next Steps

    The paxalisib phase II study remains ongoing, with final data expected in 1H CY2021. The paxalisib arm of the GBM AGILE study has moved into an operational phase, and first patient in is expected early in 1Q CY2021.

    The St Jude study in DIPG remains ongoing, with final data expected during 1H CY2021.

    Investor Conference Call

    Kazia is pleased to invite investors to attend a conference call to discuss the results further.

    The call will be held on Thursday 19 November 2020 at 12:00pm, Sydney time (AEDT), which is 5pm on Wednesday 18 November 2020 in San Francisco (PST) and 8pm on Wednesday 18 November 2020 in New York (EST).

    Participants will need to pre-register for the call via the following link:

    https://s1.c-conf.com/diamondpass/10011029-8iqiBr.html

    Click the 'Register Now' button and follow the prompts to complete pre-registration. You will then receive a calendar invite with dial in numbers, a passcode and a PIN to dial into the conference call.


    [1] ME Hegi, A-C Desirens, T Gorlia, et al. N Engl J Med (2005); 352:997-1003

    [2] T Cooney, A Lane, U Bartels, et al. Neuro-Oncology (2017); 19(9):1279-1280

     

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  2. SYDNEY, Oct. 16, 2020 /PRNewswire/ -- Kazia Therapeutics Limited ((ASX: KZA, NASDAQ:KZIA), an Australian oncology-focused biotechnology company, is pleased to announce that it has executed a definitive agreement with the Global Coalition for Adaptive Research (GCAR) to commence Kazia's participation in the GBM AGILE pivotal study in glioblastoma. The study will open a new arm with Kazia's investigational new drug, paxalisib (formerly GDC-0084), and will now move into an operational phase with recruitment of patients to the paxalisib arm expected to begin in Q1 CY2021.

    Kazia Therapeutics Limited Logo

    Key Points

    • GBM AGILE (NCT03970447) is intended to serve as the pivotal study for registration of paxalisib in key markets
    • Dr Ingo Mellinghoff (Memorial Sloan Kettering Cancer…

    SYDNEY, Oct. 16, 2020 /PRNewswire/ -- Kazia Therapeutics Limited ((ASX: KZA, NASDAQ:KZIA), an Australian oncology-focused biotechnology company, is pleased to announce that it has executed a definitive agreement with the Global Coalition for Adaptive Research (GCAR) to commence Kazia's participation in the GBM AGILE pivotal study in glioblastoma. The study will open a new arm with Kazia's investigational new drug, paxalisib (formerly GDC-0084), and will now move into an operational phase with recruitment of patients to the paxalisib arm expected to begin in Q1 CY2021.

    Kazia Therapeutics Limited Logo

    Key Points

    • GBM AGILE (NCT03970447) is intended to serve as the pivotal study for registration of paxalisib in key markets
    • Dr Ingo Mellinghoff (Memorial Sloan Kettering Cancer Center) and Dr Eudocia Q Lee (Dana-Farber Cancer Institute) have been named as Principal Investigators for the paxalisib arm; Dr Timothy Cloughesy (UCLA) is the Principal Investigator for the overall study
    • Kazia will pay an initial fee of US$ 5 million to GCAR, with further milestone payments payable throughout the course of the study
    • The duration of paxalisib's enrollment period in GBM AGILE is expected to total approximately 30 – 36 months, plus follow-up, but will depend on emerging study data, recruitment rates, and other variables

    Kazia CEO, Dr James Garner, commented, "we have spent the last nine months or so working closely with the GCAR team to plan paxalisib's entry into GBM AGILE, and we are very gratified to now be moving into the operational phase of the study. GBM AGILE is truly a ground-breaking clinical trial, driven by some of the world's leading experts in the field, and we are proud to be a part of it. We expect GBM AGILE to provide definitive clinical evidence for the approval of paxalisib by regulatory agencies in key markets. This is a faster, more cost effective, and higher quality study than any company of our size could mount independently, and we are confident that it will provide the best possible opportunity for paxalisib to demonstrate its potential in this very challenging disease."

    Dr Meredith Buxton, Chief Executive Officer at GCAR added, "We are pleased to welcome paxalisib into GBM AGILE. Our mission is to help drive the development of new therapies for glioblastoma, by creating an efficient model for testing and confirming new potentially beneficial treatments for patients with GBM. We look forward to continuing to work closely with the Kazia team to bring paxalisib into the study and support its evaluation."

    Principal Investigators

    Dr Ingo Mellinghoff and Dr Eudocia Q Lee will serve as Principal Investigators for the paxalisib arm. Dr Timothy Cloughesy is the Principal Investigator for the overall study.

    Dr Mellinghoff is the Chair of the Department of Neurology at Memorial Sloan Kettering Cancer Center in New York, NY. He is a highly experienced neuro-oncologist with an extensive track record of published research in brain tumours, and is a Professor at the Gerstner Sloan Kettering Graduate School of Biomedical Sciences and the Graduate School of Medical Sciences at Weill Cornell University. His laboratory focuses on the study of biochemical pathways that regulate the growth of brain cancer, and he has participated in numerous clinical trials for glioblastoma and other forms of brain cancer.

    Dr Mellinghoff commented, "we have seen little progress in the treatment of glioblastoma for over two decades, and the need for new therapies is urgent. We have seen encouraging signals from the paxalisib program thus far, and my colleagues and I look forward to exploring its potential in the GBM AGILE pivotal study."

    Dr Lee is a neuro-oncologist at Dana-Farber Cancer Institute in Boston, MA, Director of Clinical Research at the Center for Neuro-Oncology at Dana-Farber, and an Assistant Professor of neurology at Harvard Medical School. She is a widely published clinical researcher, with a primary research interest in tumours of the brain and spinal cord, and their neurologic complications. Dr Lee has been an investigator in previous clinical trials of paxalisib in glioblastoma and has first-hand clinical experience with the drug.

    Dr Lee added, "GBM AGILE has been designed to provide a definitive assessment of the efficacy of new drugs for glioblastoma. Paxalisib has already been evaluated in two clinical trials in this disease, and GBM AGILE will now greatly enrich our understanding of how best to use it for the benefit of patients."

    GBM AGILE

    GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment) is an international platform study that has been established specifically to facilitate the approval of new medicines for glioblastoma.

    The scientific leadership of GBM AGILE comprises many of the leading experts in glioblastoma, and they have worked in collaboration with the US FDA on its development. It is sponsored by the Global Coalition for Adaptive Research (GCAR), a US-based 501(c)(3) non-profit organisation. At present, the study is underway in 30 sites in the United States and Canada, with plans to launch in Europe and China during CY2021. One drug candidate is currently participating, and paxalisib will be the second candidate to join the study.

    GBM AGILE is an adaptive study, so the number of patients recruited, and their allocation within the study, will be continuously adjusted in the light of emerging results. It is expected that between 50 and 200 patients will receive paxalisib, depending on the safety and efficacy of the drug. The data from these patients will be compared against data from an estimated several hundred patients in a shared control arm, allowing for considerable operational efficiency.

    The paxalisib arm of GBM AGILE will recruit newly diagnosed patients with unmethylated MGMT promotor status, which is the same population that has been investigated in Kazia's ongoing phase II study. In addition, GBM AGILE will recruit recurrent patients to the paxalisib arm. The drug may ultimately be considered efficacious in either or both of these patient groups, and Kazia will frame any future application for regulatory approval on the basis of this data.

    Dr Mellinghoff added, "we see interesting signals of activity in the phase I study of paxalisib in recurrent glioma patients, and so my colleagues and I consider it important to evaluate the drug also in this later-stage group, where the unmet medical need is very substantial. Including both newly diagnosed and recurrent patients in GBM AGILE enables us to observe how paxalisib performs across the spectrum of the disease, and provides us with a significant amount of additional data as we move towards registration."

    The primary endpoint of GBM AGILE is overall survival (OS), which is considered the gold standard endpoint for the assessment of new cancer therapies.

    Indicative Costs and Timelines

    Kazia will initially pay a fee of US$ 5 million to GCAR in consideration for paxalisib joining GBM AGILE. Additional payments will be due throughout the duration of the study, dependent on the attainment of key milestones. The full financial terms of the agreement between Kazia and GCAR are considered commercially confidential. In addition, the total cost of the study will depend on the number of patients ultimately recruited and other operational variables.

    Kazia and GCAR expect that necessary regulatory filings and submissions to institutional review boards will be actioned during 4Q CY2020. First patient in to the paxalisib arm is currently anticipated to occur early in CY2021.

    The duration of paxalisib's participation in GBM AGILE is unpredictable due to the adaptive nature of the study. As an indicative base case estimate, Kazia expects at this stage that paxalisib will enrol patients for between 30 – 36 months, plus follow-up. However, this figure could change, either in an upward or downward direction, depending on emerging data from the study as well as operational matters such as recruitment rates.

    About Kazia Therapeutics Limited

    Kazia Therapeutics Limited ((ASX: KZA, NASDAQ:KZIA) is an innovative oncology-focused biotechnology company, based in Sydney, Australia. Our pipeline includes two clinical-stage drug development candidates, and we are working to develop therapies across a range of oncology indications.

    Our lead program is paxalisib (formerly GDC-0084), a small molecule inhibitor of the PI3K / AKT / mTOR pathway, which is being developed to treat glioblastoma, the most common and most aggressive form of primary brain cancer in adults. Licensed from Genentech in late 2016, paxalisib entered a phase II clinical trial in 2018. Interim data was reported most recently at AACR in June 2020, and further data is expected in 2H 2020. Five additional studies are in start-up or ongoing in other forms of brain cancer. Paxalisib was granted Orphan Drug Designation for glioblastoma by the US FDA in February 2018, and Fast Track Designation for glioblastoma by the US FDA in August 2020. In addition, paxalisib was granted Rare Pediatric Disease Designation and Orphan Designation by the US FDA for DIPG in August 2020.

    TRX-E-002-1 (Cantrixil), is a third-generation benzopyran molecule with activity against cancer stem cells and is being developed to treat ovarian cancer. TRX-E-002-1 has completed a phase I clinical trial in Australia and the United States with the final data expected in the second half of calendar 2020. Interim data was presented most recently at the AACR conference in June 2020. Cantrixil was granted orphan designation for ovarian cancer by the US FDA in April 2015.

    For more information, please visit www.kaziatherapeutics.com.

    This document was authorized for release to the ASX by James Garner, Chief Executive Officer, Managing Director.

     

     

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  3. SYDNEY, Sept. 22, 2020 /PRNewswire/ -- Kazia Therapeutics Limited ((ASX: KZA, NASDAQ:KZIA), an Australian oncology-focused biotechnology company, is pleased to announce that it has entered into a collaboration with Dana-Farber Cancer Institute (DFCI) in the United States, to investigate the use of Kazia's investigational new drug, paxalisib (formerly GDC-0084), in primary central nervous system (CNS) lymphoma, a potential new indication for the drug.

    Key Points

    • Lymphoma is a cancer of white blood cells. It occurs in the lymphatic system and can spread almost anywhere in the body; primary CNS lymphoma (PCNSL) occurs exclusively in the brain and central nervous system
    • The PI3K inhibitor class is well validated in lymphoma outside the brain; three of the four FDA-approved PI3K inhibitors are treatments for forms of lymphoma, but they are assumed ineffective for PCNSL since they cannot cross the blood-brain barrier
    • DFCI will initiate an open-label phase II clinical trial of paxalisib in PCNSL
    • The study is expected to recruit up to 25 patients, taking up to 2 years to complete
    • Kazia will provide support including study drug and a financial grant
    • This study will be the sixth ongoing clinical trial of paxalisib in brain cancer

    Dana-Farber Cancer Institute (DFCI) is a world-leading cancer treatment and research centre, based in Boston, Massachusetts. It is a principal teaching affiliate of Harvard Medical School and has been designated a Comprehensive Cancer Center by the US National Cancer Institute. DFCI participates in as many as 600 clinical trials at any given time and has been an important contributor to the development of many important new cancer therapies.

    Kazia CEO, Dr James Garner, commented, "this is an exciting new opportunity for the paxalisib program. We are delighted to support the team at Dana-Farber to explore the potential for paxalisib to benefit patients with PCNSL. Dana-Farber is one of the world's leading centres of excellence in this disease, so we are immensely fortunate to be working with them. We are pleased also to see a new and important target added to the broader paxalisib clinical program, and we look forward to seeing the project commence."

    Kazia's financial support to the study will use a portion of the funds contributed by shareholders in the Share Purchase Plan (SPP) conducted in May 2020.

    Primary CNS Lymphoma

    Lymphoma is a haematological malignancy (blood cancer) that originates from lymphocytes, a type of white blood cell involved in the immune system. PCNSL is a specific form of the disease that originates in the brain and central nervous system.

    Three of the four PI3K inhibitors approved by the US Food and Drug Administration (FDA) are treatments for various forms of lymphoma, provide a strong validation for PI3K as a target in this disease. Paxalisib is the only PI3K inhibitor in mainstream development with the ability to penetrate the blood-brain barrier, and as such has a unique rationale for development in PCNSL.

    PCNSL accounts for approximately 4% of brain tumours, and the incidence is increasing with time. Patients are typically in their 60s or older, and the disease is slightly more common in men.[1] The mainstays of treatment comprise chemotherapy and radiotherapy, but recurrence is common and only approximately 30% of patients remain alive five years after diagnosis.[2] Many of the drugs used to treat lymphoma elsewhere in the body are ineffective in PCNSL due to their inability to cross the blood-brain barrier.

    Clinical Trial Design

    Dana Farber Cancer Institute will launch a single-arm phase II clinical trial in patients with relapsed or refractory PCNSL, who are resistant to existing treatments. The primary endpoint will be to assess efficacy via overall response rate (ORR), which measures the ability of paxalisib to shrink tumours. Safety and other efficacy endpoints will also be captured. The study will also examine tissue and cerebrospinal fluid samples to identify potential predictors of response.

    The principal investigator for the study is Dr Lakshmi Nayak, Director of the CNS Lymphoma Center at Dana-Farber Cancer Institute. Dr Nayak is an Assistant Professor of Neurology at Harvard Medical School and a board-certified neuro-oncologist. Her research interests focus on metastatic brain cancer, glioblastoma, and PCNSL, and she is extensively published in the field of brain cancer. She has been an investigator for multiple clinical trials of experimental drugs in this disease area.

    Commencement of recruitment to the study is expected in early CY2021, but is subject to receiving necessary approvals from FDA and from institutional review boards. The study will be listed on clinicaltrials.gov closer to the commencement of recruitment.

    Dana-Farber Cancer Institute

    The CNS Lymphoma Center (CNSLC) at Dana-Farber / Brigham and Women's Cancer Center is the first centre of its kind in the world dedicated to providing comprehensive care and research for patients with primary or secondary CNS lymphoma. With the most advanced treatment options available — including surgery, chemotherapy, stem cell transplant, radiation therapy, immunotherapy, and targeted therapies, and with extensive clinical trial options - CNSLC is uniquely qualified to treat patients with CNS lymphoma and advance the outcome of patients with these tumors.

    Other clinicians at Dana-Farber Cancer Institute are currently involved in clinical trials of paxalisib in other forms of brain cancer. Dr Jose Pablo Leone is the principal investigator on a phase II clinical trial of paxalisib in combination with Herceptin (trastuzumab) for breast cancer brain metastases (breast cancer that has spread to the brain). Professor Patrick Wen has been centrally involved in the ongoing phase II study of paxalisib in glioblastoma, and plays a leadership role in the international GBM AGILE study.

    Paxalisib Clinical Program

    The initiation of this trial in PCNSL brings the number of ongoing clinical studies of paxalisib in brain cancer to six.

    Indication

    Phase

    Sponsor

    Registration

    Glioblastoma

     

     

    II

    Kazia Therapeutics

    NCT03522298

    DIPG

     

     

    I

    St Jude Children's Research Hospital

    NCT03696355

    Breast Cancer Brain Metastases

    (with trastuzumab)

    II

    Dana-Farber Cancer Institute

    NCT03765983

    Brain Metastases

     

     

    II

    Alliance for Clinical Trials in Oncology

    NCT03994796

    Brain Metastases

    (with radiotherapy)

     

    I

    Memorial Sloan-Kettering Cancer Center

    NCT04192981

    Primary CNS Lymphoma

     

    II

    Dana-Farber Cancer Institute

    (TBD)

    Next Steps

    Recruitment to this study is expected to commence in 1H CY2021.

    Kazia completed recruitment to a phase II clinical trial of paxalisib in newly-diagnosed glioblastoma in February 2020, and interim clinical data was presented at the AACR Virtual Annual Meeting II in June 2020. Overall survival was calculated at 17.7 months, which compares favourably to an historical figure of 12.7 for temozolomide, the existing FDA-approved standard of care. Kazia expects to present further data from this study in 2H CY2020, and to conclude the study in early CY2021.



    [1] J Rubenstein et al. (2008) Leuk Lymphoma 49(0 1):43-51

    [2] MS Shiels et al. (2016) Br J Haematol. 174(3):417-424

     

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  4. SYDNEY, Aug. 23, 2020 /PRNewswire/ -- Kazia Therapeutics Limited ((ASX: KZA, NASDAQ:KZIA), an Australian oncology-focused biotechnology company, is pleased to announce that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Kazia's paxalisib (formerly GDC-0084) for the treatment of malignant glioma, which includes Diffuse Intrinsic Pontine Glioma (DIPG), a rare and highly aggressive childhood brain cancer.

    Key Points

    • Orphan Drug Designation (ODD) is a special status accorded to drugs which are considered promising potential treatments for rare ('orphan') diseases, generally defined as those which affect less than 200,000 cases per annum in the United States
    • ODD can provide drug developers with up…

    SYDNEY, Aug. 23, 2020 /PRNewswire/ -- Kazia Therapeutics Limited ((ASX: KZA, NASDAQ:KZIA), an Australian oncology-focused biotechnology company, is pleased to announce that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Kazia's paxalisib (formerly GDC-0084) for the treatment of malignant glioma, which includes Diffuse Intrinsic Pontine Glioma (DIPG), a rare and highly aggressive childhood brain cancer.

    Key Points

    • Orphan Drug Designation (ODD) is a special status accorded to drugs which are considered promising potential treatments for rare ('orphan') diseases, generally defined as those which affect less than 200,000 cases per annum in the United States
    • ODD can provide drug developers with up to seven years of Orphan Drug Exclusivity (ODE), extending the effective life of a commercial product. It also provides opportunities for grant funding, protocol assistance, and financial benefits, such as a waiver of New Drug Application fees, and tax credits
    • Receipt of ODD follows award of Rare Pediatric Disease Designation (RPDD) for DIPG on 7 August 2020

    Kazia CEO, Dr James Garner, commented, "Taken together, RPDD and ODD provide a powerful suite of incentives, opportunities, and protections for the development of paxalisib in DIPG. We look forward to seeing initial data from the ongoing phase I study in DIPG at St Jude Children's Research Hospital during the second half of calendar 2020. In parallel, we are working closely with collaborators, advisors, and researchers to determine the best path forward for paxalisib in this devastating disease."

    He added, "This award of ODD concludes a program of regulatory optimisation that Kazia has initiated for paxalisib over the past six months. As we orient paxalisib towards commercialization, these special designations from FDA will allow us to move forward in the swiftest and most effective way possible."

    Orphan Drug Designation

    ODD exists to recognise the development of a drug for a rare disease, which may affect adults or children. ODD provides an additional period of 7.5 years data exclusivity (for a paediatric disease), which allows companies to better defend their products against competition. It also results in a waiver by FDA of fees for a marketing application, under the Prescription Drug User Fees Act (PDUFA fees), which are just under US$ 3 million in FY2020. In addition, drugs with ODD may be eligible for orphan grants by FDA.

    Kazia previously received ODD for paxalisib in glioblastoma in February 2018.

    Summary of Paxalisib Regulatory Status



    Glioblastoma

    Most common and most aggressive adult brain cancer

    DIPG

    Highly aggressive childhood brain cancer

    Orphan Designation

    February 2018

    August 2020

    Fast Track Designation

    August 2020



    Rare Pediatric Disease Designation

    n/a

    August 2020

    Next Steps

    Kazia expects to present further data from its ongoing phase II study of paxalisib in glioblastoma at the Society for Neuro-Oncology (SNO) Annual Meeting in November 2020.

    Initial efficacy data from the ongoing phase I study of paxalisib in DIPG at St Jude Children's Research Hospital is expected during 2H CY2020. Precise timing remains uncertain due to pandemic-related disruption in conference schedules, but Kazia expects to provide an update to investors at the earliest opportunity.

    Paxalisib has been selected to join the international GBM AGILE pivotal study in glioblastoma, and recruitment is expected to begin in 2H CY2020.

     

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  5. SYDNEY, Aug. 20, 2020 /PRNewswire/ -- Kazia Therapeutics Limited ((ASX: KZA, NASDAQ:KZIA), an Australian oncology-focused biotechnology company, is pleased to announce that the United States Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to Kazia's paxalisib (formerly GDC-0084) for the treatment of glioblastoma, the most common and most aggressive form of primary brain cancer.

    Key Points

    • Fast Track Designation (FTD) is designed to expedite development of pharmaceutical products which demonstrate the potential to address unmet medical needs in serious or life-threatening conditions
    • FTD provides Kazia with substantially enhanced access to FDA, including opportunities for face-to-face meetings and written consultation throughout the remaining development of paxalisib
    • Drugs with FTD are eligible to apply for Accelerated Approval and Priority Review at the time of a New Drug Application (NDA) submission, which may result in faster product approval
    • FTD also allows for 'rolling review', whereby Kazia may submit completed sections of the paxalisib NDA as they become available, rather than at the end of development
    • Kazia consequently plans to begin initial preparatory activities for NDA filing for paxalisib in CY2021

    Kazia CEO, Dr James Garner, commented, "in awarding Fast Track Designation to paxalisib, FDA has recognised the drug's potential to meaningfully improve outcomes for patients with glioblastoma. This is a very powerful acknowledgement. The opportunities that Fast Track Designation creates, as we move towards an NDA filing, are of great value and have the potential to substantially accelerate the commercialisation of paxalisib. In particular, the 'rolling review' process enables Kazia to complete and submit substantial sections of our NDA filing in advance, saving time and reducing risk for the product. We look forward to working closely with FDA as we move into the final stage of development for paxalisib."

    The specific indication for which FTD has been approved is "for the treatment of patients with newly diagnosed glioblastoma with unmethylated O6-Methylguaninemethyltransferase (MGMT) promotor status who have completed initial radiation with concomitant temozolomide." This language precisely reflects the patient population studied in the ongoing phase II study, and is the primary proposed population for the GBM AGILE pivotal study, and is the intended indication at commercial launch.

    Kazia announced on 7 August 2020 that FDA had granted paxalisib Rare Pediatric Disease Designation (RPDD) for DIPG, an aggressive childhood brain cancer. For clarity, this granting of FTD for glioblastoma is not specifically connected to the prior granting of RPDD in DIPG.

    Fast Track Designation

    Introduced under the FDA Modernization Act (1997), Fast Track Designation (FTD) may be awarded by FDA to investigational drugs which treat a serious or life-threatening condition, and which fill an unmet medical need. FDA notes that 'the purpose [of the Fast Track program] is to get important new drugs to the patient earlier.'[1] FTD must be requested by the sponsor company and must be accompanied by a detailed review of both preclinical and clinical data. To be awarded FTD, drugs must generally be able to show some potential advantage over existing therapies, either in terms of safety or efficacy.

    The key benefits of FTD comprise enhanced access to FDA, with regular and more frequent opportunities for consultation and discussion. In addition, drugs with FTD may be eligible for Accelerated Approval, in which a new medicine is approved prior to the availability of definitive data, and Priority Review, in which the standard 12-month review process is reduced to six months. Drugs with FTD may also enter a 'rolling review' of their NDA submission, in which sections are submitted and reviewed as they become available, substantially expediting the approval process.

    Next Steps

    Kazia completed recruitment to its phase II clinical trial of paxalisib in newly diagnosed glioblastoma in February 2020, and interim clinical data was presented at the American Association of Cancer Research (AACR) Virtual Annual Meeting II in June 2020. Overall survival was calculated at 17.7 months, which compares favourably to a historical figure of 12.7 months for temozolomide, the existing FDA-approved standard of care.

    Kazia expects to present further data from this study in 2H CY2020, and to conclude the study in early CY2021.

    Paxalisib has been selected to join the international GBM AGILE pivotal study in glioblastoma, and recruitment is expected to begin in 2H CY2020.


    [1] https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track

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    SOURCE Kazia Therapeutics Ltd

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