On September 1, 2020 Aptorum Group Limited (NASDAQ: APM, Euronext Paris: APM) ("Aptorum Group" or the "Company"), a biopharmaceutical company focuses on the development of novel therapeutics to address global unmet medical needs, reported a business update and announced financial results for the six months ended June 30, 2020 (Press release, Aptorum, SEP 1, 2020, View Source [SID1234564195]).

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"Throughout the COVID-19 crisis, we remained focused on advancing the development of our therapeutic programs. As announced today, further positive data showing significant in vivo activities of ALS-4 (for MRSA wound healing and MRSA bacteraemia) and also in vitro and in vivo studies of SACT-1 (for neuroblastoma and other potential tumor types). Also, as an emerging company, we have been expanding our global strategic presence. In July, Aptorum Group became the first Nasdaq listed biopharmaceutical company admitted to trading on Euronext Paris. We are also delighted about 3 new appointments we made to support the development of our various programs. Looking forward, we remain committed to accelerating the Company’s commercial growth and transformation into a biopharmaceutical company with exciting clinical stage assets being developed."

Clinical Pipeline Update and Upcoming Milestones

SACT-1–lead program of the Smart-ACTTM platform, a repurposed drug for neuroblastoma and others: Undergoing preparation and on track for IND submission to commence Phase 1b/2a human clinical trials targeting the US FDA’s 505(b)(2) pathway. Further in vitro screening to assess SACT-1’s potential effect on over 300 cancer cell lines has been completed and showed promising effect on including, but not limited to, colorectal cancer, leukemia and lymphoma.

ALS-4–lead program of the Acticule platform, a small drug molecule candidate for methicillin resistant Staphylococcus aureus ("MRSA" superbug): ALS-4 is undergoing final stages of IND enabling studies and is targeted for regulatory submission in Q4 2020 to commence a Phase 1 human clinical trial thereafter.

CLS-1–lead program of the Claves platform, a macromolecule approach for obesity: Currently in lead optimization stage, aimed for IND enabling studies to commence in 2021.

NLS-2 NativusWell–a dietary supplement for woman’s health, including menopause and osteoporosis: Undergoing registration in the United Kingdom, Europe and Asia, aimed for distribution to market in 2020.

Corporate Highlights

Commenced trading on Euronext Paris stock exchange:

Aptorum Group became the first Nasdaq listed biopharmaceutical company admitted to trading on Euronext Paris. The Class A Ordinary Shares of Aptorum Group have commenced trading on the Professional Compartment of Euronext in Paris under the Euronext ticker symbol "APM" and ISIN Code: KYG6096M1069 on 24 July 2020.

Three new personnel appointed to Aptorum Group’s team:

Dr. Herman Weiss, M.D., Chief Executive Officer and Executive Director of Claves Life Sciences Limited and Senior Medical Advisor of Aptorum Group
Dr. Kira Sheinerman, Senior Strategic Consultant of Aptorum Group
Dr. Robbie Majzner, Scientific Advisor of Aptorum Group
Financial Results for the Six Months Ended June 30, 2020

Aptorum Group reported a net loss of $7.0 million for the six months ended June 30, 2020 compared to $9.6 million for the same period in 2019. The decrease in net loss in current period was driven by decrease in interest expenses, net of $3.6 million, partly offset by the increase in research and development expenses by $1.6 million.

Research and development expenses were $4.3 million for the six months ended June 30, 2020 compared to $2.7 million for the same period in 2019. The increase was primarily due to the increase in consultation service provided by our consultants, advisory and contracted research organization as a result of the progress of our projects’ development.

General and administrative fees were $2.1 million for the six months ended June 30, 2020 compared to $3.2 million for the same period in 2019. The decrease was mainly driven by the decrease in bonus related expenses to our directors, employees, external consultants and advisors. Also, there was a significant decrease in business trips and sponsoring conference in 2020 due to the outbreak of COVID-19.

Legal and professional fees were $1.5 million for the six months ended June 30, 2020 compared to $2.0 million for the same period in 2019. The decrease in legal and professional fees was mainly due to the decrease of consultancy service fees during the period.

Interest expenses, net were $0.1 million for the six months ended June 30, 2020 compared to $3.7 million for the same period in 2019. The decrease in interest expenses, net was mainly due to the convertible debts were fully repaid in 2019. The interest expenses, net for the six month ended June 30, 2019 contained $3.1 million amortization of beneficial conversion feature of our convertible debts.

As of June 30, 2020, cash, restricted cash and marketable securities totaled approximately $4.4 million and total equity was approximately $17.5 million.

Aptorum Group expects that its existing cash, restricted cash and marketable securities, together with undrawn line of credit facility from related parties, will enable it to fund its operating and capital expenditure requirements to the end of 2021.

On September 1, 2020 Brisbane Cancer patients reported that it will soon have access to the latest in personalised cancer diagnoses with the launch of a validation study of genomiQa’s whole genome analysis platform, CapeDX, in partnership with Icon Group (Press release, QIMR Berghofer Medical Research Institute, SEP 1, 2020, View Source [SID1234564186]).

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genomiQa is a spin-off company from QIMR Berghofer Medical Research Institute and is focused on bringing precision whole genome analytics into routine clinical practice.

genomiQa’s CapeDX platform supports the personalisation of a patient’s cancer treatment by analysing the whole genome of the patient (hereditary) and their cancer tumour.

The pilot study will be conducted in collaboration with Icon Cancer Centre Wesley – part of the global Icon Group – and will involve genomic testing of up to 15 patients.

Based on genomiQa’s analysis of the patients’ genomes, each patient may be matched to the most suitable treatment option for their cancer, including approved drugs and those available through clinical trials.

genomiQa’s CEO, Colin Albert, said the validation study was a major step towards rolling out the company’s precision analytics platform more widely.

"Through this pilot study, we will validate genomiQa’s offerings and service delivery," Mr Albert said.

"Our partnership with Icon Group is essential to the success of the project, and the fact that Icon has a rich history of supporting world leading research is a key benefit."

Icon Group’s Director of Research, Dr John Bashford, said the company’s principle was that every patient deserved the opportunity to have the best and latest treatment that was appropriate for them.

"We are delighted to offer patients the opportunity to access this Australian-first study and ensure treatment is personalised to their unique genomic information," Dr Bashford said.

"No other technique currently simultaneously examines the hereditary drivers of cancers as well as more than 1500 known cancer genes in a tumour."

The CEO of Rare Cancer Australia, Richard Vines, has welcomed the pilot study.

"The challenge we face is that more than 145 000 people are diagnosed with cancer in Australia every year, and we have been treating the symptoms not the root cause," Mr Vines said.

"But with the advent now of technology like genomiQa’s, it may give patients more reason to hope."

This project is supported by the Commonwealth Department of Industry, Science, Energy and Resources through the Entrepreneurs’ Programme, which has contributed nearly $500 000 of matched funding.

The rest of the funding was provided by QIMR Berghofer Medical Research Institute, which supports the commercialisation of discoveries by the Institute’s scientists.

This Commonwealth Government grant will also support the accreditation of genomiQa’s whole genome analysis product for rare diseases, Gulf DX.

On September 1, 2020 Carina Biotech and biopharmaceutical company Biosceptre reported that they have signed an agreement that will see the advancement of a promising cancer immunotherapy (Press release, Carina Biotech, SEP 1, 2020, http://carinabiotech.com/carina-biotech-completes-its-first-commercial-agreement/ [SID1234564185]).

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Biosceptre, developing targeted therapeutics and immune-oncology products, have acquired the exclusive rights to Carina Biotech’s nfP2X7-targeted CAR-T cells and associated intellectual property, which have shown encouraging cancer-killing capacity against a number of cancers in pre-clinical testing. Biosceptre and Carina will collaborate to further develop the nfP2X7 CAR-T program, with the aim of advancing towards clinical trials in the near future. Under the terms of the deal, Carina received an upfront cash payment and equity in Biosceptre.

Deborah Rathjen, PhD, CEO of Carina Biotech, said: "This agreement with Biosceptre represents a significant milestone for Carina as the first commercial agreement involving our proprietary CAR-T technology. We believe that nfP2X7-targeting CAR-T cells hold significant promise as a potential treatment for a range of solid cancers, and we are pleased to be working with Biosceptre to further bolster their pipeline of therapies exploiting the novel nfP2X7 target."

"I would like to pay particular tribute to the Carina Board of Directors, led by our Chair Dr Leanna Read, for their role in bringing this deal to fruition," Dr Rathjen added.

Gavin Currie, CEO of Biosceptre said: "With this deal we are building on our commitment to therapies focussed on non-functional P2X7, a cancer target that has been shown to be present on the majority of cancers. Our goal is to bring novel therapeutics to patients suffering from a wide range range of malignancies, and CAR-T therapy is an important part of our immune-oncology approach."

ABOUT nfP2X7
P2X7 is an ATP-gated cation channel on the surface of some normal cells. In response to certain extracellular conditions, P2X7 forms a pore in the cell that is associated with the induction of cell death. It was demonstrated by Biosceptre that a dysfunctional variant of P2X7 (nfP2X7) is expressed by cancer cells and is unable to open the large pore that drives normal cell death. Biosceptre has shown that nfP2X7 is widely distributed on over 20 types of cancer yet substantially absent in healthy cell types – making it an ideal target to treat cancer.

In 2015, Carina founding CEO Dr Justin Coombs, together with Professor Simon Barry and his laboratory at the Women’s & Children’s Hospital, created the first CAR-T cells targeted against nfP2X7. Carina Biotech has since demonstrated pre-clinical anti-cancer activity against a broad range of cancer types including prostate, breast and ovarian cancers.