On November 26, 2020 Golden Biotechnology Corp.(GoldenBiotech, 4132.TWO), a leading Taiwanese biopharmaceutical company, reported that its new drug Antroquinonol (HOCENA) outperforms the other listing drugs for the treatment of relapsed AML (acute myeloid leukemia) patients in its Phase 2 clinical study conducted in Russia (Press release, Golden Biotechnology, NOV 26, 2020, View Source;outperforms-listing-drugs-in-relapsed-acute-myeloid-leukemia-301181008.html [SID1234571803]). The outcome measures demonstrated higher remission rates (50%, CR/Cri) and survival rates guaranteeing fewer patients will require blood transfusions. This has made high safety possible during breakthroughs for AML treatment with remarkable monotherapy without combined chemotherapy. The new drug Antroquinonol has been granted orphan drug designation (ODD) by the U.S. FDA for the treatment of AML in 2015. GoldenBiotech has achieved excellent breakthrough in the unmet medical needs in AML therapy which many global pharmaceutical companies are targeting.

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Breakthrough in AML Treatment! GoldenBiotech’s new drug clinical trial of Antroquinonol.
Clinical Results Summary:

*Primary Outcome Measure- overall remission rate (CR/CRi): 50%

*Secondary Outcome Measures

80% of patients showed that their blasts count were less than 5%
90% of patients did not need blood transfusion during trial period
100% of patients were all alive during trial period (6 months)
No serious side effect
The primary efficacy indicator results exhibit that the overall remission rate (CR/CRi) of patients with relapsed acute myeloid leukemia reaches 50%. This is higher than those of other listing drugs in their Phase 2 trial ranging approximately from 21%~33% in (CR/CRi). 80% of patients’ abnormal blasts counts in bone marrow or blood were decreased to be less than 5% after treatment. For the secondary outcome measures results the overall survival rate of patients with relapsed AML reached 100% by the 6th month after treatment. This amplifies the possibility of patients receiving bone marrow transplantation. Furthermore, 90% of patients do not need to undergo blood transfusion during treatment with Antroquinonol (Hocena). Oral treatments and the decrease in blood transfusions have greatly improved the quality of life of AML patients. There is no significant side effect (SAE) that has occurred during the 6-month treatment period. The dose trial is administered orally at home by taking 200 mg twice a day.

The trial of Antroquinonol (HOCENA), Protocol No. GHAML-2-001, was an open-label, non-randomized, Phase 2a study to evaluate efficacy and safety/tolerability profiles in adult patients with relapsed acute myeloid leukemia (AML) or at initial diagnosis when no intensive treatment is possible.

There is no standard treatment for relapsed/refractory adult acute myeloid leukemia (AML) patients. Based on the confidence achieved in the trial results for the treatment of second-line and recurrent AML patients, GoldenBiotech will apply the clinical trial (IND) to the U.S. FDA for the Phase 2 clinical trials as the first-line treatment of AML and continue to expand the spectrum of indications of Antroquinonol in other leukemia treatment fields including multiple myeloma (MM), chronic myelogenous leukemia (CML), and pediatric leukemia.

The majority of AML patients need to undergo blood transfusions and chemotherapy to kill cancer cells due to abnormal differentiation of blood cells and proliferation of myeloblasts. Current therapies are combined with chemotherapy often causing serious side effects resulting in poor prognosis of treatment for relapsed AML patients. AML ranks the second highest incidence in all leukemia but it has the highest mortality rate and the lowest five-year survival rate (about only 25%).

It is estimated that there will be about 62,000 patients with acute myeloid leukemia (AML) in 2020. According to the reports from GlobalData and other institutions, the global AML therapeutic drug market is expected to increase from USD 1.4 billion in 2019 to 5.1 billion in 2029 with a compound annual growth rate (CAGR) of 13.6%.

About Antroquinonol

Antroquinonol was discovered in 2006 by Golden Biotechnology Corp..Its compound structure, preparation methods and applicable indications have been protected with patents worldwide. The recent approved by FDA and undergoing clinical trial of new drug Antroquinonol is a Phase 2 clinical trial for mild to moderate COVID-19 hospitalized patients in the USA. This is the first Taiwanese new drug approved by FDA for the Phase 2 clinical trial in Covid-19. Antroquinonol has shown anti-viral, anti-inflammatory and anti-fibrotic characteristics in past animal studies, suggesting high likelihood in becoming successful treatment option for coronavirus-induced pneumonia.

Learn more about the trial and contact information: Antroquinonol – Covid-19 Clinical Trial.

Other pipelines include non-small cell lung cancer (NSCLC), pancreatic cancer, acute myeloid leukemia, hypercholesterolemia and hyperlipidemia, atopic dermatitis and hepatitis. Research on neurogenic disorders like Alzheimer’s disease (AD) treatment and prevention are also in good process. Antroquinonol has received ODD (Orphan drug designation) approval by US FDA for treatment of Acute Myeloid Leukemia(AML), Hepatocelluar carcinoma (Liver cancer, HCC) and Pancreatic Cancer in 2015. For ODD in Pancreatic cancer has also received approval by European EMA in 2017.

On November 26, 2020 Sanofi reported that Patients with a difficult to treat form of multiple myeloma will now gain access to a new treatment on the NHS in England and Wales, after cost regulators issued final guidance on the use of Sarclisa (isatuximab) (Press release, Sanofi, NOV 26, 2020, View Source [SID1234571827]).

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Sarclisa, administered as an intravenous infusion, plus pomalidomide and dexamethasone, is recommended for use within the Cancer Drugs Fund (CDF) as an option for treating relapsed and refractory multiple myeloma in adults.

Routine NHS funding was not approved at this stage "because the cost-effectiveness estimates are uncertain as there are limitations in the clinical data" the Institute said.

However, its green light via the CDF will allow doctors to offer the treatment as an option for people who have had lenalidomide and a proteasome inhibitor, and whose disease has progressed from their last treatment if they have had three previous forms of treatment, while more data is collated.

"Our independent appraisal committee has recognised more treatment options are needed for those with difficult-to-treat multiple myeloma," said Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE.

"Some of the data our committee has already seen shows promise that isatuximab plus pomalidomide and dexamethasone delays the disease from progressing and increases how long people live compared with current treatment options.

"Its use via the Cancer Drugs Fund will add a fourth line treatment option while data from an on-going clinical trial and from NHS use is collected to establish whether it is cost effective."

The guidance is dependent on conditions laid out in a confidential managed access agreement for the treatment combination.

Around 500 people a year could benefit from treatment with the combination.

On November 26, 2020 Sentinel Oncology Limited reported that it has entered into a collaboration and license agreement with Taiwan based pharmaceutical company PharmaEngine, Inc. (TWO: 4162) to advance the development of SOL-578 , their checkpoint kinase 1 (Chk1) inhibitor (Press release, Sentinel Oncology, NOV 26, 2020, View Source [SID1234571836]). Under the terms of the agreement, PharmaEngine will fund IND enabling studies for SOL-578.

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Sentinel Oncology Limited is a drug discovery company passionate about the development of novel therapeutics to treat cancer patients for whom there is currently an unmet medical need. The company’s mission is to increase survival and improve outcomes for cancer patients with CNS tumors. SOL-578 is a best-in-class checkpoint kinase 1 (Chk1) inhibitor featuring high kinase selectivity and oral bioavailability which targets the DNA Damage Response (DDR) network.

Under the terms of the Agreement, Sentinel Oncology will receive an exclusivity payment and PharmaEngine will obtain an option to receive the exclusive rights to develop and commercialize SOL-578 worldwide. In the event that PharmaEngine completes the IND enabling studies and exercise its option, Sentinel Oncology will be eligible to receive an upfront and development milestone payments in addition to tiered royalties based on future worldwide net sales of SOL-578.

Robert Boyle, CEO of Sentinel Oncology commented: "We are very excited about the prospects for this programme and the collaboration with PharmaEngine will enable SOL-578 to reach clinical trials and provide a real benefit for patients for whom there is currently a poor prognosis".

On November 26, 2020 Merck, a leading science and technology company, reported that the European Medicines Agency (EMA) has validated for review, the application for tepotinib for the treatment of adult patients with advanced non-small cell lung cancer (NSCLC) harboring mesenchymal-epithelial transition factor gene (MET) exon 14 (METex14) skipping alterations (Press release, Merck & Co, NOV 26, 2020, View Source [SID1234571845]). With this validation, the application is complete, and the EMA will now begin the review procedure.

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Tepotinib is a highly selective oral MET inhibitor that is administered once daily.1 The application to the EMA is based on results from the pivotal Phase II VISION study (NCT02864992) evaluating tepotinib as monotherapy in patients with advanced NSCLC with METex14 skipping alterations, prospectively assessed by liquid biopsy or tissue biopsy. In the ongoing study, the patient population is generally characterized as elderly, with a median age of 74.0 years, and as having poor clinical prognosis typical of NSCLC with METex14 skipping alterations. Data from the primary analysis of the VISION study were published in The New England Journal of Medicine (NEJM) on May 29, 2020.2

Lung cancer is estimated to be the second most common cancer in Europe, and the leading cause of cancer-related mortality, responsible for 388,000 deaths in 2018.3 METex14 skipping occurs in approximately 3–4% of NSCLC cases and correlates with aggressive tumor behavior and poor clinical prognosis.4 Currently, there are no treatments available in Europe for patients with advanced NSCLC harboring METex14 skipping alterations.

Tepotinib became the first oral MET inhibitor indicated for the treatment of advanced NSCLC harboring MET gene alterations to receive a regulatory approval globally, with its approval in Japan in March 2020 through the SAKIGAKE program. Recently, the FDA granted Orphan Drug Designation (ODD) to tepotinib and the FDA is reviewing the application under Priority Review and through the Real-Time Oncology Review pilot program.

About Tepotinib

Tepotinib is an oral MET inhibitor that inhibits the oncogenic MET receptor signaling caused by MET (gene) alterations. Discovered and developed in-house at Merck, it has a highly selective mechanism of action, with the potential to improve outcomes in aggressive tumors that have a poor prognosis and harbor these specific alterations.1

Additional Clinical Investigations: Tepotinib is also being investigated in the Phase II INSIGHT 2 study in combination with osimertinib in MET amplified, advanced or metastatic NSCLC harboring activating EGFR mutations that has progressed following first-line treatment with osimertinib, and in the Phase II PERSPECTIVE study in combination with cetuximab in RAS/BRAF wild-type left-sided metastatic colorectal cancer patients having acquired resistance to anti-EGFR antibody targeting therapy due to MET amplification.

References

Bladt F, et al. Clin Cancer Res. 2013;19:2941-2951.
Paik PK et al. Tepotinib in non–small-cell lung cancer with MET exon 14 skipping mutations. N Engl J Med 2020 May 29; [e-pub]. (View Source)
Ferlay J, et al. Eur J Cancer. 2018;103:356–387.
Reungwetwattana T, et al. Lung Cancer. 2017;103:27–37.
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On November 26, 2020 Vivoryon Therapeutics AG (Euronext Amsterdam: VVY; ISIN DE0007921835) reported its third quarter business update for the period ending September 30, 2020 (Press release, Vivoryon Therapeutics, NOV 26, 2020, View Source [SID1234571781]). The third quarter 2020 report is available for download on the Company website (View Source).

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KEY HIGHLIGHTS

Enrollment of first patient in VIVIAD, the Company’s European Phase 2b Alzheimer’s disease study evaluating varoglutamstat (PQ912)
Announcement of IND approval for varoglutamstat (PQ912)
Successful completion of the Ordinary General Meeting of Shareholders

CORPORATE REVIEW

Financial Review (According to IFRS)

In the third quarter of 2020, research and development expenses amounted to EUR 3,653k and increased compared to the third quarter of 2019 (EUR 1,196k). General and administrative expenses increased to EUR 1,051k (Q3 2019: EUR 768k). The Company did not generate any revenue in the reporting period, in line with corporate planning. Therefore, the net loss of the period was EUR 4,598k compared to EUR 1,935k in the third quarter of 2019.

Vivoryon Therapeutics held EUR 10.0 million in cash and cash equivalents as of September 30, 2020. In addition, the Company holds other securities in amount of EUR 19,967k which can be liquidated at any time.

All results are in line with management expectations.

OPERATIONAL REVIEW

Announcement of Enrollment of First Patient in VIVIAD, European Phase 2b Alzheimer’s Disease Study with Varoglutamstat (PQ912)

The Company announced that the first patient was enrolled in VIVIAD, a Phase 2b, randomized and multi-center clinical study in Europe. The study will evaluate the safety and efficacy of Vivoryon’s lead candidate, varoglutamstat (PQ912), in patients with Alzheimer’s disease .

IND Approval for Phase 2 Study of Varoglutamstat (PQ912) in Patients with Alzheimer’s Disease

The Company announced that the U.S. Food and Drug Administration (FDA) cleared the Company’s Investigational New Drug (IND) application for varoglutamstat (PQ912). FDA clearance of the IND will enable Vivoryon to initiate its U.S. Phase 2 clinical trial program for varoglutamstat (PQ912) in Alzheimer’s disease as planned.

Ordinary General Meeting of Shareholders of Vivoryon Therapeutics AG

The Company announced that its shareholders approved all resolutions proposed by the Company’s management and Supervisory Board at the Company’s Annual General Meeting which took place on Wednesday, September 30, 2020. This included the transfer of the statutory seat to Amsterdam, the Netherlands, leading to a conversion into a public company under the laws of the Netherlands.