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Aptorum Group Limited Reports 2018 Fiscal Year Financial Results and Business Update

On April 15, 2019 Aptorum Group Ltd. (NASDAQ: APM), a preclinical stage pharmaceutical company developing and commercializing a broad portfolio of projects under development of therapeutic and diagnostic technologies to tackle unmet medical needs, reported its financial results for the fiscal year ended December 31, 2018 (Press release, Aptorum, APR 15, 2019, View Source [SID1234535133]).

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Recent Business Updates

Successfully completed an initial public offering ("IPO") on the Nasdaq Global Market of 761,419 Class A ordinary shares at a public offering price of $15.8 per shares
Private placement of convertible bonds in April 2018 of $15 million of which $1.5 million was converted into Class A ordinary shares upon IPO in December 2018
Full year 2018 research and development expenses of $3.1 million, increased $0.5 million compared to the period March 1, 2017 to December 31, 2017
"Last year was monumental for Aptorum Group. On the business front, we completed our IPO in the fourth quarter of 2018," said Ian Huen, Founder and Chief Executive Officer of Aptorum Group. "In 2019, we look forward to continued advancement of our projects under development, and expect to advance our first lead project into IND-enabling studies."

2018 Fiscal Year Financial Results Highlights

Aptorum Group reported a net loss of $15.1 million in 2018, as compared to $2.6 million for the period March 1, 2017 through December 31, 2017. The increase in net loss in 2018 was driven by the decrease in gain on investment in marketable securities due to the a one-off investment disposed in last period, increased in general and administration fees due to the increased headcount in the Group to support the business development, and increased in net interest expenses due to the convertible debts issued in 2018.

Research and development expenses were $3.1 million in 2018 as compared to $2.6 million for the period March 1, 2017 through December 31, 2017. The increase in research and development expenses in 2018 was primarily due to the expansion of research and development team to support growing business and projects.

General and administrative fees were $4.9 million in 2018 as compared to $1.5 million for the period March 1, 2017 through December 31, 2017. The increase in general and administrative expenses 2018 was mainly driven by increased headcount in the Group to support the business development.

Legal and professional fees were $1.8 million in 2018 as compared to $1.4 million for the period March 1, 2017 through December 31, 2017. The increase in legal and professional fees was mainly due to the preparation of IPO and business expansion.

Aptorum Group ended the 2018 with $26.1 million in cash and restricted cash as compared to $16.7 million as of December 31, 2017. The increase in cash and restricted cash was mainly the result of the $15 million of convertible bonds issued during the year.

Can-Fite Receives $1,000,000 Payment from CKD for Distribution of Namodenoson in South Korea

On April 15, 2019 Can-Fite BioPharma Ltd. (NYSE MKT: CANF) (TASE:CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs that address cancer, liver and inflammatory diseases, reported it has received a $1,000,000 payment from Chong Kun Dang Pharmaceuticals (CKD) (Korean Stock Exchange: 185750.KS) of South Korea (Press release, Can-Fite BioPharma, APR 15, 2019, View Source [SID1234535131]). CKD has licensed the distribution rights to Namodenoson in South Korea for the treatment of liver cancer and non-alcoholic steatohepatitis (NASH) in a deal totaling $6,000,000 in upfront and milestone payments, plus a transfer price for delivering finished products to CKD. The current $1,000,000 payment is an upfront payment for the expansion of CKD’s existing agreement with Can-Fite to include the rights to distribute Namodenoson for the treatment of NASH in South Korea.

"As we advance our pipeline of drugs, we are pleased to work with our expanding network of global distribution partners for Namodenoson and Piclidenoson," stated Can-Fite CEO Pnina Fishman.

Zymeworks Opens Phase 2 Clinical Trial for ZW25 in First-Line HER2-Expressing Metastatic Gastroesophageal Cancers

On April 15, 2019 Zymeworks Inc. (NYSE/TSX: ZYME), a clinical-stage biopharmaceutical company developing multifunctional therapeutics, reported it has initiated its global multicenter Phase 2 clinical trial evaluating ZW25 in combination with standard of care chemotherapy for the first-line treatment of HER2-positive metastatic gastric, gastroesophageal junction, and esophageal adenocarcinomas (Press release, Zymeworks, APR 15, 2019, View Source [SID1234535130]).

"Advancing ZW25 into a Phase 2 clinical trial represents another key milestone for Zymeworks," said Ali Tehrani, Ph.D., Zymeworks’ President & CEO. "This reaffirms our commitment to execute our ambitious drug development strategy to address unmet need in patients with HER2-expressing cancers across multiple indications and lines of therapy. We anticipate that data from this trial will support initiation of a first-line registrational trial, which could position ZW25 as the new standard of care for HER2-positive metastatic gastric cancer."

In addition to the Phase 2 metastatic gastroesophageal cancer trial, ZW25 continues to be evaluated in multiple expansion cohorts in the ongoing Phase 1 trial as a single agent in gastrointestinal, gynecological, and other HER2-expressing cancers, and in combination with chemotherapy in breast and gastroesophageal cancers.

About the Trial

The Phase 2 trial is a two-part open-label study. The primary objectives of this trial are to confirm the safety, tolerability, and anti-tumor activity of ZW25 in combination with global standard of care regimens for gastroesophageal adenocarcinoma, including platinum and fluoropyrimidine-based regimens.

"While major advances have been made in the treatment of HER2-positive breast cancer, patients with HER2-positive gastroesophageal adenocarcinomas still have limited options," said Diana Hausman, M.D., Chief Medical Officer of Zymeworks. "Based on the encouraging single agent anti-tumor activity we observed in patients with gastroesophageal adenocarcinomas in our Phase 1 study, we believe that ZW25 has the potential to address this need, and we are excited to be starting this first-line trial."

About ZW25

ZW25 is being evaluated in Phase 1 and Phase 2 clinical trials across North America and South Korea. It is a bispecific antibody, based on Zymeworks’ Azymetric platform, that can simultaneously bind two non-overlapping epitopes of HER2, known as biparatopic binding. This unique design results in multiple mechanisms of action including dual HER2 signal blockade, increased binding and removal of HER2 protein from the cell surface, and potent effector function leading to encouraging anti-tumor activity in patients. Zymeworks is developing ZW25 as a HER2-targeted treatment option for patients with any solid tumor that expresses HER2. The FDA has granted Orphan Drug Designation to ZW25 for the treatment of both gastric and ovarian cancers.

About the Azymetric Platform

The Azymetric platform enables the transformation of monospecific antibodies into bispecific antibodies, giving the antibodies the ability to simultaneously bind two different targets. Azymetric bispecific technology enables the development of multifunctional biotherapeutics that can block multiple signaling pathways, recruit immune cells to tumors, enhance receptor clustering degradation, and increase tumor-specific targeting. These features are intended to enhance efficacy while reducing toxicities and the potential for drug-resistance. Azymetric bispecifics have been engineered to retain the desirable drug-like qualities of naturally occurring antibodies, including low immunogenicity, long half-life, and high stability. In addition, they are compatible with standard manufacturing processes with high yields and purity, potentially significantly reducing drug development costs and timelines.

INSYS Therapeutics Implements Leadership Changes

On April 15, 2019 INSYS Therapeutics, Inc. (NASDAQ: INSY), a leader in the development, manufacture and commercialization of pharmaceutical cannabinoids and spray technology, reported that the Board of Directors has appointed Andrew G. Long as Chief Executive Officer, effective immediately (Press release, Insys Therapeutics, APR 15, 2019, View Source [SID1234535129]). Mr. Long succeeds Saeed Motahari, who has mutually agreed with the INSYS Board of Directors to resign as President and CEO of the Company.

In connection with INSYS’ leadership changes, Andrece Housley, INSYS’ Corporate Controller, has been appointed Chief Financial Officer, succeeding Mr. Long. Additionally, Dr. Venkat Goskonda has been promoted to Chief Scientific Officer, overseeing the Company’s R&D and manufacturing activities.

Steven Meyer, Chairman of the Board of Directors, remarked, "We believe that now is the right time to transition leadership and that Andy is an excellent choice to serve as the Company’s CEO. Andrece and Dr. Goskonda’s promotions are a reflection of their contributions to INSYS. On behalf of the Board, I thank Saeed for his commitment to INSYS and meaningful contributions to the enterprise. We appreciate Saeed’s dedication to INSYS’ employees and stakeholders and we wish him all the best in the future."

Mr. Long joined INSYS as Chief Financial Officer in August 2017 with more than three decades of experience in the life sciences, bio-pharma and industrial sectors. Prior to joining INSYS, he served as Senior Vice President of Global Finance at Patheon, where he worked on a number of initiatives leading up to Patheon’s IPO. Prior to working at Patheon, Mr. Long spent nine years as Vice President of Finance for multiple divisions at Thermo Fisher Scientific. He previously spent five years leading the global finance and supply chain functions for the BioScience Division of Cambrex Corporation. Previously, Mr. Long also spent almost a decade building his financial expertise in various roles at Abbott Laboratories.

Intellia Therapeutics Announces Three Oral Presentations on In Vivo and Engineered Cell Therapy Data at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy

On April 15, 2019 Intellia Therapeutics, Inc. (NASDAQ:NTLA), reported three oral presentations at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper), taking place April 29-May 2, 2019, in Washington, D.C (Press release, Intellia Therapeutics, APR 15, 2019, View Source [SID1234535128]).

Intellia’s data includes important updates from the company’s programs and platform development activities:

"CRISPR/Cas9-Mediated Targeted Insertion of Human F9 Achieves Therapeutic Circulating Protein Levels in Mice and Non-Human Primates"

Intellia will present data showing that its targeted gene insertion platform achieved therapeutic levels of Factor IX protein in non-human primates (NHP). The company employs a proprietary hybrid delivery system, comprised of both lipid nanoparticles (LNPs) and adeno-associated virus (AAV), to insert the desired gene sequence. Factor 9 (F9) is a gene that encodes Factor IX (FIX), a blood-clotting protein that is missing or defective in hemophilia B patients.

The data showing therapeutic levels of FIX achieved in NHPs is from an ongoing research collaboration between Intellia and Regeneron Pharmaceuticals, Inc.

Presenter: Hon-Ren Huang, Ph.D., associate director, Vector Biology, Intellia
Abstract number: 11
Session: Advances in Genome Editing and Hemophilia Gene Therapies
Presentation date/time: Mon., April 29, 2019, 9-9:15 a.m. ET
Location: Heights Courtyard 2
"Exploiting Clonal Tracking of WT1-Specific T Cells to Generate a Library of Tumor-Specific T Cell Receptors (TCR) for TCR Gene Editing of Acute Leukemia"

This presentation will focus on Intellia’s ongoing research collaboration with IRCCS Ospedale San Raffaele in Italy to develop CRISPR-edited T cell therapies to address intractable cancers, such as acute myeloid leukemia (AML). Researchers generated and tested a library of TCRs with different epitope specificities and human leukocyte antigen (HLA) restrictions.

Presenter: Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy
Abstract number: 123
Session: Cancer Adoptive Immunotherapy
Presentation date/time: Mon., April 29, 2019, 5-5:15 p.m. ET
Location: Georgetown
"CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria"

Intellia will provide information demonstrating successful knockout of two targets of interest, lactate dehydrogenase A (LDHA) and hydroxyacid oxidase 1 (HAO1), to address primary hyperoxaluria type 1 (PH1) in a PH1 mouse model. The data shows the continued progression of the company’s modular platform capability using CRISPR to knock out liver gene targets. The data being presented includes results from an ongoing collaboration with researchers at the University of Alabama at Birmingham.

Presenter: Anette Hübner, Ph.D., associate director, Liver Biology, Intellia
Abstract number: 1000
Session: Use of New Technologies for Hepatic Therapy
Presentation date/time: Thur., May 2, 2019, 12-12:15 p.m. ET
Location: Heights Courtyard 3
"Delivering on the Therapeutic Promise of CRISPR/Cas9"

Intellia also will participate in the ASGCT (Free ASGCT Whitepaper) Gene Editing Workshop, which will provide an overview of current gene editing technologies and approaches, as well as emerging uses and applications.

Presenter: Sean Burns, M.D., senior director, Hematology and New Therapeutic Areas
Session: Corporate Review II
Session date/time: Sun., April 28, 2019, 5-6 p.m. ET
Location: Lincoln