On April 19, 2021 Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM), a clinical stage biopharmaceutical company dedicated to meeting unmet medical needs in oncology and infectious diseases, reported financial results for the fiscal year ended December 31, 2020, and provided an update on clinical and corporate developments (Press release, Aptorum, APR 19, 2021, View Source [SID1234578199]).

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Mr. Darren Lui, President and Executive Director of Aptorum Group Limited, commented "Aptorum’s operational plans remain on track. In particular, one of our lead programs, ALS-4 (targeting infections caused by Staphylococcus aureus including Methicillin-resistant Staphylococcus aureus (MRSA)) has already commenced dosing of the first cohort of human subjects in its Phase I clinical trial as announced in March 2021. With the additional capital raised in October 2020, the Company is also on track to commence validation of our novel liquid biopsy based rapid pathogen identification and detection diagnostics ("RPIDD"). Our other lead program SACT-1, which is targeting an orphan indication for the treatment of neuroblastoma, is also on track in its preparation to open an IND with US FDA for the commencement of clinical trials in 2021. The Company is also focused on the distribution of a non-hormonal based Dioscorea opposita bioactive nutraceutical supplement targeting woman’s health during menopausal or post-menopausal cycles in 2021 in major markets including UK, Europe and Asia initially. In light of the global coronavirus situation, the Company would like to report that its day-to-day operations continue as normal.

Clinical Pipeline Update and Upcoming Milestones

In March 2021, Aptorum Group announced dosing of our first human subject in its Phase I clinical trial for one of our lead programs, ALS-4, an orally administered small molecule drug for the treatment of infections caused by Staphylococcus aureus including MRSA. The Phase I clinical trial is currently conducted in Canada and will target to recruit up to 48 and 24 healthy volunteers for the single-ascending dose (SAD) and multiple-ascending dose (MAD) cohorts, respectively.

In February 2021, Aptorum Group completed a Pre-IND meeting with US FDA with encouraging outcomes regarding SACT-1, another lead program employing an orally administered small molecule repurposed drug for the treatment of neuroblastoma. The Company is now on track in its preparation to open an IND with US FDA for the commencement of clinical trials in 2021.

In September 2020, Aptorum Group initiated an additional R&D program. Our RPIDD technology enables rapid and accurate identification and detection of existing or emerging unknown pathogens (including DNA/RNA-based viruses such as coronavirus, antibiotic-resistant bacteria, fungi, etc.), in a cost-effective, unbiased and broad-spectrum manner, by employing liquid biopsy (patients’ blood samples and is potentially adaptable for other sample types) in conjunction with New Generation Sequencing (NGS) and Artificial Intelligence (AI) driven software analytics. The Company is now on track to commence validation of the RPIDD program in further human blood samples to support its eventual commercialization.

Aptorum Group is commencing the commercialization and distribution of its non-hormonal based Dioscorea oppposita bioactive nutraceutical supplement ("NativusWell") in 2021, targeting woman’s health during menopausal or post-menopausal cycles. The product is currently being manufactured in Canada.

Corporate Highlights

On July 24, 2020, our Class A Ordinary Shares began to trade on the Professional Compartment of the regulated market of Euronext Paris under the symbol "APM" and are denominated in Euros on Euronext Paris.

On September 25, 2020, Aptorum Innovations Holding Pte. Limited ("AIHPL"), one of the Company’s wholly-owned subsidiaries, entered into an Exclusive License Agreement with Accelerate Technologies Pte Ltd. ("Accelerate Technologies"), commercialization arm of the Singapore’s Agency for Science, Technology and Research, to co-develop novel molecular-based RPIDD technology.

In October 2020, Aptorum Group completed a capital raise of approximately $9 million by a public offering of 2,769,231 Class A ordinary shares and warrants to purchase 2,769,231 of its Class A ordinary shares.

On December 30, 2020, AIHPL entered into an Evaluation Agreement with Illumina Inc ("Illumina") to evaluate the data and performance of Illumina’s sequencing technology based on the workflow of AIHPL’s RPIDD technology, at AIHPL’s Singapore based evaluation site.

Full year 2020 research and development expenses of $11.6 million, an increase of $4.6 million over the full year 2019.

Fiscal Year End Financial Results

Aptorum Group reported a net income of $4.9 million in 2020, as compared to net loss of $20.1 million in 2019. The net income in 2020 was mainly driven by the gain on investments in marketable securities, net of $25.2 million.

Research and development expenses were $11.6 million in 2020 as compared to $6.9 million in 2019. The increase in research and development expenses in 2020 was primarily due to the increase in services provided by our consultants, advisors and contracted research organizations as a result of the progress of our projects’ development.

General and administrative fees were $4.9 million in 2020 as compared to $7.4 million in 2019. The decrease in general and administration fees was primarily due to a decrease in compensation expenses and business expenses as well as general travel restrictions due to the COVID 19 outbreak.

Legal and professional fees were $2.9 million in 2020 as compared to $3.4 million in 2019. The decrease in legal and professional fees was primarily due to the reduction of professional services required during 2020.

Aptorum Group reported $3.6 million of cash and restricted cash as of December 31, 2020 compared to $5.3 million as of December 31, 2019. The decrease in cash and restricted cash was mainly the result of the cash used in operating activities of $15.8 million during the year and net repayment of loan from related parties of $4.4 million, partly offset by the net proceeds from issuance of Class A Ordinary Shares and warrants of $16.8 million in 2020 and proceeds from disposal of fixed assets and investment in marketable securities of $1.0 million and $0.9 million, respectively, in 2020.

In addition, Aptorum Group had $28.4 million marketable securities and $13.0 million undrawn line of credit as of December 31, 2020, compared to $1.1 million marketable securities and $8.7 million undrawn line of credit as of December 31, 2019. The increase in marketable securities was due to the public listing and subsequent stock price appreciation of one of our pre-IPO investments.

On April 19, 2021 NeoTX Therapeutics (NeoTX), a clinical-stage immuno-oncology company, reported that it received clearance from the U.S. Food and Drug Administration (FDA) for the Company’s Investigational New Drug (IND) application for naptumomab estafenatox (NAP) (Press release, NeoTX, APR 19, 2021, View Source [SID1234640358]). NeoTX is developing targeted anticancer immunotherapies utilizing its proprietary Tumor Targeted Superantigen (TTS) platform. NAP, the company’s lead TTS molecule, binds a genetically engineered bacterial determinant to the tumor surface while simultaneously activating and expanding tumor specific immune cells. NAP has demonstrated preliminary safety and anti-tumor activity in early-stage clinical trials in solid tumors.

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"This FDA clearance is an exciting milestone for NeoTX," said Asher Nathan, Ph.D., chief executive officer of NeoTX. "Preclinical and preliminary clinical studies have demonstrated that NAP has potential in combination with other treatment modalities. Non-small cell lung cancer is one of the deadliest cancers, and we are looking forward to assessing NAP in the clinic in combination with chemotherapy as a potential new treatment option after failure of current standards of care."

The Phase 2a open label trial will evaluate NAP in combination with docetaxel in 35 patients with checkpoint inhibitor pretreated, advanced or metastatic non-small cell lung cancer. The primary endpoint is objective response rate as measured by RECIST 1.1 criteria. The trial will also evaluate safety, duration of response, progression free survival, overall survival, pharmacokinetics and pharmacodynamics.

On April 19, 2021 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported the completion of enrollment of the first treatment cohort and the escalation to the next and potentially final dose level in a Phase 1 investigator sponsored clinical trial (IST), named rESPECT, of its lead product candidate eryaspase for the first-line treatment of pancreatic cancer (Press release, ERYtech Pharma, APR 19, 2021, https://erytech.com/erytech-announces-completion-of-first-cohort-in-a-phase-1-investigator-sponsored-trial-of-eryaspase-in-first-line-pancreatic-cancer/ [SID1234578158]).

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On April 19, 2021 Amgen (NASDAQ:AMGN) reported that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for investigational bemarituzumab as first-line treatment for patients with fibroblast growth factor receptor 2b (FGFR2b) overexpressing and human epidermal growth factor receptor 2 (HER2)-negative metastatic and locally advanced gastric and gastroesophageal (GEJ) adenocarcinoma in combination with modified FOLFOX6 (fluoropyrimidine, leucovorin, and oxaliplatin), based on an FDA-approved companion diagnostic assay showing at least 10% of tumor cells overexpressing FGFR2b (Press release, Amgen, APR 19, 2021, View Source [SID1234578184]).

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"The FIGHT trial is the first study to evaluate targeting the overexpression of FGFR2b in cancer. Bemarituzumab demonstrated clinically meaningful outcomes in key endpoints for patients with advanced gastric or gastroesophageal cancer as a frontline therapy," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "Amgen looks forward to further investigating the role of FGFR2b and will continue to work with regulatory agencies on next steps to bring this potential first-in-class, frontline therapy to patients."

More than one million new gastric cancer cases are diagnosed annually, and gastric cancer is particularly prevalent in Asia.1,2 Approximately 80 to 85% of patients with advanced gastric and GEJ cancers are HER2-negative, and approximately 30% of these patients present with FGFR2b overexpression.3,4

Following sotorasib, bemarituzumab is the second asset in Amgen’s oncology portfolio to receive Breakthrough Therapy Designation in the past six months. A Breakthrough Therapy Designation is designed to expedite the development and regulatory review of medicines that may demonstrate substantial improvement on a clinically significant endpoint over available medicines.5

The FIGHT trial evaluated bemarituzumab plus chemotherapy (mFOLFOX6) versus chemotherapy alone in patients with FGFR2b+, non-HER2 positive frontline advanced gastric or GEJ cancer. In the study, treatment with bemarituzumab plus chemotherapy demonstrated clinically significant and substantial improvements in the primary endpoint of progression-free survival (PFS) and secondary endpoint of overall survival (OS) in the patient population in which at least 10% of tumor cells overexpressed FGFR2b. Additional analysis showed a positive correlation between benefit and the prevalence of FGFR2b+ tumor cells, affirming both the importance of the FGFR2b target and the activity of bemarituzumab against this target. The Breakthrough Therapy Designation was granted based upon this subset of patients who showed at least 10% of tumor cells overexpressing FGFR2b.

Amgen acquired Five Prime Therapeutics on April 16, 2021. In addition to bemarituzumab, Five Prime’s pipeline complements Amgen’s efforts to bring innovative therapies to oncology patients.

About Bemarituzumab
Bemarituzumab (anti-FGFR2b) is a potential first-in-class investigational targeted antibody that is designed to block fibroblast growth factors (FGFs) from binding and activating FGFR2b, inhibiting several downstream pro-tumor signaling pathways and potentially slowing cancer progression. Bemarituzumab is being developed in gastric and GEJ cancer as a targeted therapy for tumors that overexpress FGFR2b. The company is also evaluating the potential for bemarituzumab in other cancers that overexpress FGFR2b.

Zai Lab (Shanghai) Co. Ltd. was granted an exclusive license to develop and commercialize bemarituzumab in Greater China, and Zai Lab collaborated with Five Prime on the Phase 2 FIGHT trial in Greater China.

About Amgen Oncology
Amgen Oncology is searching for and finding answers to incredibly complex questions that will advance care and improve lives for cancer patients and their families. Our research drives us to understand the disease in the context of the patient’s life – not just their cancer journey – so they can take control of their lives.

For the last four decades, we have been dedicated to discovering the firsts that matter in oncology and to finding ways to reduce the burden of cancer. Building on our heritage, Amgen continues to advance the largest pipeline in the Company’s history, moving with great speed to advance those innovations for the patients who need them.

At Amgen, we are driven by our commitment to transform the lives of cancer patients and keep them at the center of everything we do.

To learn more about Amgen’s innovative pipeline with diverse modalities and genetically validated targets, please visit AmgenOncology.com. For more information, follow us on www.twitter.com/amgenoncology.

On April 19, 2021 Humanigen, Inc. (Nasdaq: HGEN) ("Humanigen"), a clinical-stage biopharmaceutical company focused on preventing and treating an immune hyper-response called ‘cytokine storm’ with its lead drug candidate, lenzilumab, reported positive data from the Phase 1b portion of ZUMA-19 evaluating the efficacy and safety of lenzilumab in patients treated with CAR-T in diffuse large B-cell lymphoma (DLBCL) (Press release, Humanigen, APR 19, 2021, View Source [SID1234578200]). At the recommended Phase 2 dose of lenzilumab, the ORR was 100% and no patient experienced severe cytokine release syndrome (CRS) or severe neurotoxicity (NT).

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ZUMA-19 was a clinical study designed to evaluate the efficacy and safety of lenzilumab and CAR-T (axicabtagene ciloleucel, Axi-Cel) in patients with relapsed or refractory DLBCL.

This study was a standard 3+3 design with three patients administered 600 mg lenzilumab (cohort 1) and three patients administered 1,800 mg lenzilumab (cohort 2) just prior to CAR-T. The recommended Phase 2 dose was determined to be 1,800 mg.

In the six study patients, the ORR was 83% (n=5) which included four complete responses (CR). In cohort 1, there was no severe CRS (≥ grade 3). One patient experienced grade 3 NT with a two-day duration. At the recommended Phase 2 dose (cohort 2), ORR was 100% (n=3) and the toxicity-free CR (CRS and NT < grade 2) was 66% (n = 2). There was no severe CRS or severe NT at the recommended Phase 2 dose. There were no adverse events attributed to lenzilumab across the study.

Inflammatory markers were correlated with reduced rates of CRS and NT. Lenzilumab dose-dependently reduced myeloid cytokines IL-6, IL-8, MCP-1, and IP-10 (CXCL-10) and systemic inflammatory markers CRP, ferritin, and SAA.

"These encouraging results from ZUMA-19 provide further proof of concept that lenzilumab may break the linkage between efficacy and toxicity (CRS and NT) widely-associated with CAR-T, and may improve durability of response," said Dale Chappell, MD, MBA, Chief Scientific Officer, Humanigen. "We believe these data warrant a larger study involving multiple CAR-T therapies."

Humanigen will initiate a randomized, multicenter, potentially registrational, Phase 2 study to evaluate the efficacy and safety of lenzilumab combined with all commercially available CD19 CAR-T therapies in DLBCL. The study plans to enroll approximately 150 patients and the protocol is being submitted to FDA.

Humanigen has terminated the clinical collaboration agreement with Kite related to ZUMA-19 and both parties will collaborate to wind down current study activities.

"Humanigen is pleased to be in a position to proactively develop lenzilumab across the CAR-T landscape and further expand its pipeline," said Cameron Durrant, MD, MBA, Chief Executive Officer, Humanigen. "We thank Kite for their sponsorship and contribution that has allowed Humanigen to progress to this exciting point."