On June 23, 2022 Minomic International Ltd reported that it has taken a step forward to bring the MiCheck Prostate test to Australians (Press release, Minomic, JUN 23, 2022, View Source [SID1234616197]).

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MiCheck Prostate detects aggressive prostate cancer overcoming the low accuracy of conventional screening tests, reducing unnecessary intervention such as painful biopsies and improving overall management of the patient.

The performance of MiCheck Prostate is supported by numerous clinical studies, including a recent one at Macquarie University Hospital, that found that MiCheck Prostate has a sensitivity of 93% and a specificity of 45%.

With testing available now at selected Sonic Healthcare Australia Pathology laboratories, Minomic can offer MiCheck Prostate to many more Australian men.

Minomic CEO Dr Brad Walsh says the availability of the test will help Australia’s urologists to provide better patient care; "The use of MRI for the detection of prostate cancer, in Australia, has been a revolutionary step forward in the early and accurate detection of this cancer. MRI, when combined with results from MiCheck Prostate provide urologists with an additional aid in clinical decision-making before proceeding to more invasive investigations such as biopsy."

"The test is now market ready in both Australia and the US, while China and other markets such as Europe are also in our sights."

An estimated 25,000 Australian men and two million American men undergo biopsies each year to diagnose prostate cancer after an elevated Prostate-Specific Antigen (PSA) test result. Approximately 50% of these biopsies are unnecessary as the patient does not have cancer or has a low-grade cancer that requires monitoring rather than intervention. In addition, 1-3% of these biopsies can lead to sepsis and death. These unnecessary biopsies lead to patient anxiety and pain that should be avoided.

Prostate cancer remains an ongoing global challenge. Each year around 250,000 men in the US and 20,000 men in Australia are diagnosed with prostate cancer. It is estimated that 1 in 8 males will be diagnosed with a prostate cancer in their lifetime.

Minomic opened a capital raise earlier this year to take the test to market in both Australia and the US ahead of a planned stock market listing in 2023. Last year it received a $400,000 co-investment from the Advanced Manufacturing Growth Centre’s (AMGC) Commercialisation Fund and the Federal Government’s Modern Manufacturing Initiative.

Minomic has established a US based marketing team and obtained a CLIA license enabling availability of MiCheck Prostate in the world’s largest healthcare market.

On June 22, 2022 Acrivon Therapeutics, Inc., a clinical-stage oncology therapeutics company with proprietary proteomics-based technologies driving a new era of precision medicine, reported that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for its lead asset ACR-368 in a Phase 2 master protocol trial to treat patients with ovarian, endometrial and urothelial cancer based on predicted ACR-368 sensitivity (Press release, Acrivon Therapeutics, JUN 22, 2022, View Source [SID1234621404]). ACR-368 is a potent, selective inhibitor of checkpoint kinase 1 and 2 (CHK1/2) which has previously shown durable monotherapy efficacy in a proportion of patients across several high unmet need solid tumor types. Acrivon will stratify patients for its trial into two treatment groups using its pioneering OncoSignature proteomic companion diagnostic. OncoSignature-positive patients will receive ACR-368 monotherapy in a Phase 2 Simon two-stage study design at the recommended Phase 2 dose, while OncoSignature-negative patients will receive ACR-368 in combination with low-dose gemcitabine in a Phase 1b/2 study design.

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Acrivon’s Predictive Precision Proteomics (AP3) technology platform enables the development of drug-tailored OncoSignature companion diagnostics, which apply quantitative protein multiplex imaging tests to pretreatment tumor biopsies and are designed to enable the identification of the patients whose tumors are regulated by and sensitive to the drug. Acrivon’s AP3 platform was also used to identify key mechanisms associated with ACR-368 resistance, and that the addition of low-dose gemcitabine substantially re-sensitizes resistant tumors to ACR-368, which was subsequently confirmed in multiple preclinical studies.

"Our clinical development approach is quite different than the industry norm, and this clinical trial design is equally unprecedented," said Peter Blume-Jensen, M.D., Ph.D., president and chief executive officer of Acrivon. "Acrivon’s next generation proteomics-based precision medicine platform is engineered to uncover the disease-driving mechanisms that are uniquely sensitive to our drugs or rational drug combinations in individual patient tumors, independent of genetic alterations. Our strategy is to apply the ACR-368-tailored OncoSignature test to a tumor biopsy from the patient before the start of treatment and use that result to direct ACR-368 to only those patients predicted to be most likely to benefit from either monotherapy treatment or the combination therapy."

"Acrivon’s OncoSignature tests are designed to predict drug sensitivity regardless of tumor type," said Erick Gamelin, M.D., Ph.D., chief medical officer of Acrivon. "By using OncoSignature to screen across human cancer tissue samples, we found that a proportion of endometrial and urothelial cancers is also sensitive to the drug, and following further confirmation in preclinical patient-derived xenograft models, we have included these together with platinum-resistant ovarian cancer in our upcoming trial. With Acrivon’s rigorous science-based and selective methodologies, we aim to forge a paradigm change in personalized medicine for optimal patient care."

The Phase 2, multicenter, open-label study will enroll patients with histologically confirmed, locally advanced or metastatic, recurrent platinum-resistant high-grade ovarian cancer, or endometrial adenocarcinoma, or platinum-resistant urothelial carcinoma. Based on the results of the company’s proprietary OncoSignature predictive test, patients will be allocated to one of two treatment arms. Patients who test positive for predicted sensitivity to ACR-368 monotherapy will be enrolled into a single-arm Phase 2 study to assess primarily the anti-tumor activity (confirmed overall response rate) of the recommended Phase 2 dose of ACR-368 (105 mg/m2) for each of the three cancers. Patients who test negative on the OncoSignature test will be enrolled in a single-arm Phase 1b/2 study. The Phase 1b portion of the study will evaluate the safety and tolerability of the combination of the recommended Phase 2 dose of ACR-368 with escalating doses of low-dose gemcitabine for each of the three cancers. Once the recommended Phase 2 dose of low-dose gemcitabine has been determined, the study will expand into a Phase 2 study to assess the anti-tumor activity (confirmed overall response rate) of ACR-368 and low-dose gemcitabine for each of the three cancers.

About ACR-368
ACR-368 is a potent, selective inhibitor of CHK1 and CHK2 which has shown deep durable single agent activity, including complete responses, in a proportion of patients across several Phase 2 studies of platinum-resistant ovarian cancer and in squamous cell cancers, including anal cancer for which FDA has granted orphan drug designation. ACR-368 has been tested in >1,000 patients as monotherapy and in combination, showing excellent pharmacokinetic and pharmacological properties and a favorable safety profile at the recommended Phase 2 dose across monotherapy studies. Acrivon has obtained exclusive, world-wide rights to develop and commercialize ACR-368 (also known as prexasertib) under a license agreement with Eli Lilly and Company.

On June 22, 2022 OCTALFA reported that its subsidiary ORPHELIA Pharma, the French biopharmaceutical company dedicated to the development and commercialization of orphan medicines for the treatment of rare and pediatric diseases, has included the first patient in the KIMOZO French Early Access Program for the treatment of relapsed or refractory neuroblastoma (Press release, ORPHELIA Pharma, JUN 22, 2022, View Source;utm_medium=rss&utm_campaign=octalfa-announces-the-inclusion-of-the-first-patient-in-the-french-early-access-program-granted-to-its-subsidiary-orphelia-pharma-for-the-use-of-kimozo-40-mg-ml-temozolomide-oral-suspension-in [SID1234619551]).

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KIMOZO 40 mg/ml (oral suspension of temozolomide) was granted an early access pre-marketing authorization (Autorisation d’Accès Précoce, AAP) in France on March 31st, 2022 by the Haute Autorité de Santé (HAS) for the treatment of relapsed or refractory neuroblastoma in patients aged 1 to 6 years as well as patients unable to swallow temozolomide capsules.

The goal of Early Access Programs in France is to accelerate access to innovative drugs before or after Market Authorization is granted once all conditions specified in Article L.5121-12 of the French Public Health Code (Code de la Santé Publique) are met:

No appropriate treatment exists;
The treatment’s implementation cannot be postponed;
The efficacy and safety of these drugs are strongly presumed based on therapeutic trials;
These drugs are considered to be innovative, particularly when compared to a possible clinically-relevant comparator.
Full details of the early access program can be found at the Haute Autorité de Santé and the ANSM websites.

About KIMOZO 40 mg/ml

KIMOZO 40 mg/ml is a liquid, taste-masked and ready-to-use oral formulation of temozolomide developed to treat rare pediatric cancers.

In France, KIMOZO has been granted an Early Access Program, to be used as monotherapy or in combination with irinotecan or topotecan in the treatment of pediatric patients aged 1 to 6 years and in patients with incapacity to swallow capsules of temozolomide and who suffer from:

refractory high-risk neuroblastoma or presenting an insufficient response to induction chemotherapy;
relapsed high-risk neuroblastoma after at least a partial response to induction chemotherapy followed by myeloablative therapy and stem cell transplantation.
KIMOZO was developed by ORPHELIA Pharma in collaboration with Gustave Roussy.

On June 22, 2022 OCTALFA reported that its subsidiary ORPHELIA Pharma, the French biopharmaceutical company dedicated to the development and commercialization of orphan medicines for the treatment of rare and pediatric diseases, has included the first patient in the KIMOZO French Early Access Program for the treatment of relapsed or refractory neuroblastoma (Press release, ORPHELIA Pharma, JUN 22, 2022, View Source;utm_medium=rss&utm_campaign=octalfa-announces-the-inclusion-of-the-first-patient-in-the-french-early-access-program-granted-to-its-subsidiary-orphelia-pharma-for-the-use-of-kimozo-40-mg-ml-temozolomide-oral-suspension-in [SID1234616508]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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KIMOZO 40 mg/ml (oral suspension of temozolomide) was granted an early access pre-marketing authorization (Autorisation d’Accès Précoce, AAP) in France on March 31st, 2022 by the Haute Autorité de Santé (HAS) for the treatment of relapsed or refractory neuroblastoma in patients aged 1 to 6 years as well as patients unable to swallow temozolomide capsules.

The goal of Early Access Programs in France is to accelerate access to innovative drugs before or after Market Authorization is granted once all conditions specified in Article L.5121-12 of the French Public Health Code (Code de la Santé Publique) are met:

No appropriate treatment exists;
The treatment’s implementation cannot be postponed;
The efficacy and safety of these drugs are strongly presumed based on therapeutic trials;
These drugs are considered to be innovative, particularly when compared to a possible clinically-relevant comparator.
Full details of the early access program can be found at the Haute Autorité de Santé and the ANSM websites.

About KIMOZO 40 mg/ml

KIMOZO 40 mg/ml is a liquid, taste-masked and ready-to-use oral formulation of temozolomide developed to treat rare pediatric cancers.

In France, KIMOZO has been granted an Early Access Program, to be used as monotherapy or in combination with irinotecan or topotecan in the treatment of pediatric patients aged 1 to 6 years and in patients with incapacity to swallow capsules of temozolomide and who suffer from:

refractory high-risk neuroblastoma or presenting an insufficient response to induction chemotherapy;
relapsed high-risk neuroblastoma after at least a partial response to induction chemotherapy followed by myeloablative therapy and stem cell transplantation.
KIMOZO was developed by ORPHELIA Pharma in collaboration with Gustave Roussy.

On June 22, 2022 National Brain Tumor Society reported that the U.S. Food and Drug Administration (FDA) granted accelerated approval to a new treatment combination that will now be available as an option for certain brain tumor patients (Press release, National Brain Tumor Society, JUN 22, 2022, View Source [SID1234616328]).

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The FDA approved the combination of two oral drugs called dabrafenib (brand name: Tafinlar) and trametinib (brand name: Mekinist) for the treatment of advanced tumors that have a mutation called "BRAF V600E." The approval includes use in both adult and pediatric (older than 6 years of age) high- and low-grade glioma patients with this mutation whose tumors progressed after prior treatment.

The approval is based on the results from a number of clinical trials, which included adult and pediatric high-grade and low-grade glioma patients. Data pooled from across these trials showed that the Tafinlar-Mekinist combination successfully shrank a significant percentage of these patients’ tumors. Using a measure called "Overall Response Rate (ORR)," trials found that 33% of high-grade glioma patients responded to the combination, while 50% of low-grade glioma patients saw tumor shrinkage benefits. For the pediatric glioma patients, 25% responded to the treatment.

The BRAF V600E mutation is estimated to be prevalent in 5-15% of all low-grade gliomas (15-20% of pediatric low-grade gliomas), including 60-80% of pleomorphic xanthoastrocytomas (PXA), 20-70% of gangliogliomas, and 10% of pilocytic astrocytomas. The mutation has been identified less frequently in high-grade gliomas, including approximately 3% of glioblastomas.

"This approval will offer a much-needed new treatment option for hundreds of glioma patients with a BRAF V600E mutation," said David Arons, chief executive officer, National Brain Tumor Society. "This is very positive news for the brain tumor community and offers hope that current research and drug development efforts are beginning to yield meaningful breakthroughs for patients that currently have too few options. We thank Novartis for including both adult and pediatric glioma patients in these trials, as well as the researchers and clinicians involved, and most importantly the patients and their families that participated in the studies."

The most common side effects seen in adult patients were fever, fatigue, nausea, rash, chills, headache, hemorrhage, cough, vomiting, constipation, diarrhea, muscle and joint pain, and edema.

The most common side effects seen in pediatric patients included fever, rash, vomiting, fatigue, dry skin, cough, diarrhea, dermatitis acneiform, headache, abdominal pain, nausea, hemorrhage, and constipation.

Because the approval for this treatment was granted through the FDA’s accelerated approval pathway, the sponsor of the clinical trial, Novartis, will be required to complete confirmatory trials to verify the effectiveness of these drugs to date. According to reports, the company may already have additional evidence of effectiveness in pediatric low-grade glioma patients, including in children as young as one. Based on data presented recently at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, the Tafinlar-Mekinist combination resulted in 47% ORR versus chemotherapy (11%) and reduced the risk of progression or death by 69%. Additionally, in a separate, small subset of this trial, pediatric high-grade glioma patients treated with Tafinlar-Mekinist showed an ORR of 56.1% and generally consistent safety results.

Further buoying these results were data also presented at ASCO (Free ASCO Whitepaper) 2022 from Day One Bio of their own BRAF-targeting drug, tovorafenib (DAY101), which also showed effectiveness in relapsed pediatric low-grade glioma. We await additional future results from trials of tovorafenib (DAY101).

"Given this latest approval, and the recent data from ASCO (Free ASCO Whitepaper), NBTS strongly urges that neuro-surgical and oncology providers treating glioma patients secure for patients a test that can identify their BRAF V600E status as part of robust molecular biomarker testing informed by the 2021 World Health Organization’s reclassification of CNS tumors as a routine step that could determine which patients could benefit from these drugs," added Arons.