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GEMoaB Announces Presentation of Translational Data From Ongoing Phase I Clinical Study of Their Lead Asset UniCAR-T-CD123 in Relapsed/Refractory AML at the Upcoming 2021 AACR Annual Meeting

On February 10, 2021 GEMoaB, a biopharmaceutical company focused on the development of next-generation immunotherapies for hard-to-treat cancers, reported the acceptance of a presentation on translational data obtained from their ongoing Phase I study of their lead asset UniCAR-T-CD123 in relapsed/refractory acute myeloid leukemia (rrAML) at the 2021 AACR (Free AACR Whitepaper) Annual Meeting, being held from April 9-14 (Press release, GEMoaB, FEB 10, 2021, View Source [SID1234574880]).

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The data of UniCAR-T-CD123 in rrAML provided as poster presentation at the AACR (Free AACR Whitepaper) congress highlight key features of GEMoaB’s rapidly switchable universal CAR-T platform UniCAR and focus on expansion kinetics, cytokine profiles and UniCAR-T persistence in heavily pre-treated rrAML patients.

"We have recently presented our initial clinical data on UniCAR-T-CD123 in rrAML, highlighting the advantages of our rapidly switchable UniCAR platform in terms of an extremely encouraging benefit/risk profile, the ability to abrogate and avoid acute and long-term side effects and the ability to re-activate UniCAR-T cells during a 2nd cycle with our soluble adapter molecule termed Targeting Module," said Dr. Armin Ehninger, Chief Scientific Officer of GEMoaB. "We are running an extensive translational program with our clinical studies to fully understand the unique mode-of-action of UniCAR and are very pleased that the data obtained are underpinning our vision of a highly active, controllable and persistent CAR-T product for multiple hematology and solid tumor indications as we enter into the next phase of our clinical development program."

Phase I studies of UniCAR-T-CD123 for the treatment of rrAML and UniCAR-T-PSMA directed against CRPC and other PSMA-expressing late-stage solid tumors are ongoing.

About the UniCAR-T-CD123 Phase IA Study

This first-in-human phase I study is an open-label, non-randomized, dose-finding study designed to evaluate the safety and activity of UniCAR-T-CD123 in up to 16 CD123 positive patients with relapsed/refractory AML. Its purpose is to determine the maximum tolerated dose (MTD) as well as Dose limiting toxicities (DLT) of the combined application of a single dose of UniCAR-T and the continuous infusion of TM123 over 25 days. Application follows post bridging therapy and lymphodepletion. The study also investigates response rates, response duration, persistence of UniCAR-T cells over time as well as the ability to rapidly switch UniCAR-T cells on and off in case of side effects through stopping TM infusion. The study takes place at selected Phase I, Acute Leukemia and CAR-T experienced University centers in Germany. The study is supported by a grant from the German Federal Ministry for Education and Research (project "TurbiCAR"). To learn more about the trial, please visit clinicaltrials.gov.

About UniCAR

GEMoaB is developing a rapidly switchable universal CAR-T platform, UniCAR, to improve the therapeutic window and increase efficacy and safety of CAR-T cell therapies in challenging cancers, including acute leukemias and solid tumors. Conventional CAR-T cells depend on the presence and direct binding of cancer antigens for activation and proliferation. An inherent key feature of the UniCAR platform is a rapidly switchable on/off mechanism (less than 4 hours after interruption of TM supply) enabled by the short pharmacokinetic half-life and fast internalization of soluble adaptors termed TMs. These TMs provide the antigen-specificity to activate UniCAR gene-modified T-cells (UniCAR-T) and consist of a highly flexible antigen-binding moiety, linked to a small peptide motif recognized by UniCAR-T.

I-Mab Announces China NMPA Clearance for Phase 1 Study of TJ210/MOR210 in Patients with Advanced Solid Tumors

On February 10, 2021 I-Mab (the "Company") (Nasdaq: IMAB), a clinical stage biopharmaceutical company committed to the discovery, development and commercialization of novel biologics, reported that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has cleared the Investigational New Drug (IND) application for TJ210/MOR210 to initiate a phase 1 clinical trial to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of TJ210/MOR210 monotherapy in patients with advanced solid tumors (Press release, I-Mab Biopharma, FEB 10, 2021, View Source [SID1234574879]).

TJ210/MOR210 is a monoclonal antibody developed by MorphoSys that is directed against complement factor C5a receptor 1 (C5aR1). Produced in the tumoral microenvironment, its ligand C5a acts as a chemoattractant to recruit tumor-promoting cells such as myeloid-derived suppressor cells, M2 macrophages and neutrophils. TJ210/MOR210 is designed to induce anti-tumor properties by blocking the activation and migration of C5aR1-expressing myeloid cells.

Preclinical studies have shown that targeting the C5aR-C5a axis exerts anti-tumor activity with immune checkpoint inhibitors. Furthermore, in vitro activity was observed for blocking the C5a/C5aR pathway also at very high C5a concentrations, leading to a long duration of action. TJ210/MOR210 demonstrated a good safety profile with no observed adverse effects up to the highest dose tested in non-clinical safety studies.

The phase 1 clinical trial is an open-label dose escalation study with multiple doses to evaluate the safety, tolerability, and PK/PD and preliminary efficacy of TJ210/MOR210 in subjects with relapsed or refractory advanced solid tumors. I-Mab is also conducting a phase 1 dose escalation clinical trial in patients with r/r advanced solid tumors in the U.S. The first patient in the U.S. study was dosed in January 2021.

"We are pleased to obtain the IND clearance for TJ210/MOR210 into clinical trials in China. Now with clinical trials both in the U.S. and China, we expect to accelerate this investigational drug development. The clinical data generated will enable us to further explore TJ210/MOR210’s potentials in treating patients with cancers, especially those who failed with or relapsed from the existing therapies," said Dr. Joan Shen, CEO of I-Mab.

About TJ210/MOR210

TJ210/MOR210 is a novel human antibody directed against C5aR1 derived from MorphoSys’s HuCAL Platinum technology. C5aR1, the receptor of the complement factor C5a, is investigated as a potential new drug target in the field of immuno-oncology and autoimmune diseases. Tumors have been shown to produce high amounts of C5a, which, by recruiting and activating myeloid-derived suppressor cells (MDSCs), M2 macrophages and neutrophils, is assumed to contribute to an immune-suppressive pro-tumorigenic microenvironment. TJ210/MOR210 is intended to block the interaction between C5a and its receptor, thereby potentially neutralizing the immune suppressive function of C5a and enabling immune cells to attack the tumor.

HuCAL Platinum is a registered trademark of MorphoSys AG.

VolitionRx Limited Announces Pricing of $20 Million Underwritten Public Offering of Common Stock

On February 10, 2021 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition"), a multi-national epigenetics company that applies its Nucleosomics platform through its subsidiaries to develop simple, easy to use, cost-effective blood tests to help diagnose a range of cancers and other diseases, reported the pricing of its underwritten public offering of 3,809,524 shares of its common stock (the "Offering") for gross proceeds of approximately $20 million, before deducting the underwriting commissions and other estimated offering expenses payable by Volition (Press release, VolitionRX, FEB 10, 2021, View Source [SID1234574878]). All of the shares to be sold in the Offering will be sold by Volition, subject to customary closing conditions. The Offering is expected to close on or about February 12, 2021, subject to customary closing conditions. In addition, Volition has granted the underwriter for the Offering a 30-day option to purchase up to an additional 571,428 shares of its common stock.

Cantor Fitzgerald & Co. is acting as the sole book running manager of the Offering.

The underwriter may offer the shares from time to time for sale in one or more transactions on the NYSE American market, in the over-the-counter market, through negotiated transactions or otherwise at market prices prevailing at the time of sale, at prices related to prevailing market prices or at negotiated prices. On February 9, 2021, the last sale price of the shares as reported on the NYSE American was $6.27 per share.

Volition intends to use the net proceeds of the Offering for general corporate purposes, which may include continued product development, clinical studies, product commercialization, working capital and other general corporate purposes, including potential strategic acquisitions.

The Offering is being made pursuant to a "shelf" registration statement on Form S-3 (File No. 333-227248) previously filed by Volition with the Securities and Exchange Commission (the "SEC") on September 26, 2018 and declared effective by the SEC on September 28, 2018. The Offering is being made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A preliminary prospectus supplement relating to, and describing the terms of, the Offering has been filed with the SEC. The final prospectus supplement relating to and describing the final terms of the Offering will be filed with the SEC and also will be available on the SEC’s website at www.sec.gov.

Before you invest, you should read the registration statement, the prospectus supplement and accompanying prospectus, the documents that Volition has filed with the SEC that are incorporated by reference into the registration statement, and the other documents Volition has filed with the SEC for more complete information about Volition and the Offering. You may get these documents for free by visiting EDGAR on the SEC website at www.sec.gov. Alternatively, copies of the final prospectus supplement and the accompanying prospectus relating to the Offering can be obtained, when available, from Cantor Fitzgerald & Co., Attn: Capital Markets, 499 Park Avenue, 6th floor, New York, NY 10022; Email: [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

Vanda Pharmaceuticals Reports Fourth Quarter and Full Year 2020 Financial Results

On February 10, 2021 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported financial and operational results for the fourth quarter and full year ended December 31, 2020 (Press release, Vanda Pharmaceuticals, FEB 10, 2021, View Source [SID1234574877]).

"I am very proud of the significant accomplishments we realized during this challenging year," said Mihael H. Polymeropoulos, M.D., President and CEO of Vanda. "As we look forward to the new year, it is worth noting some of our significant accomplishments from 2020: Vanda achieved record commercial revenue despite extraordinary difficulty in the field, which we believe is a testament to the value our products bring to patients; HETLIOZ received FDA approval for nighttime sleep disturbances in patients with Smith-Magenis Syndrome; our tradipitant gastroparesis Phase III study continued recruitment; the FDA approved individual Expanded Access to tradipitant for multiple gastroparesis patients; we launched the tradipitant study for COVID-19 pneumonia; the FDA approved the Investigational New Drug application for VSJ-110 in allergic conjunctivitis; the United States Supreme Court affirmed the patent ruling on Fanapt; and our research and development efforts advanced the clinical programs for our commercial assets as well as those in our pipeline. We look forward to another great year of accomplishments, including further revenue growth, the commercial launch of HETLIOZ in patients with Smith-Magenis Syndrome, and the results of the tradipitant Phase III study in gastroparesis, to highlight a few."

Key Financial and Corporate Highlights

Fourth Quarter of 2020

Total net product sales from HETLIOZ and Fanapt were $67.7 million in the fourth quarter of 2020, an 11% increase compared to $60.9 million in the fourth quarter of 2019.
HETLIOZ net product sales were $44.2 million in the fourth quarter of 2020, a 14% increase compared to $38.6 million in the fourth quarter of 2019.
Fanapt net product sales were $23.5 million in the fourth quarter of 2020, a 5% increase compared to $22.3 million in the fourth quarter of 2019.
Income before taxes was $10.9 million in the fourth quarter of 2020 compared to $5.8 million in the fourth quarter of 2019.
Full Year 2020

Total net product sales from HETLIOZ and Fanapt were $248.2 million for the full year 2020, a 9% increase compared to $227.2 million for the full year 2019.
HETLIOZ net product sales were $160.7 million for the full year 2020, a 12% increase compared to $143.0 million for the full year 2019.
Fanapt net product sales were $87.5 million for the full year 2020, a 4% increase compared to $84.2 million for the full year 2019.
Income before taxes was $31.7 million for the full year 2020 compared to $29.0 million for the full year 2019.
Cash, cash equivalents and marketable securities (Cash) was $367.7 million as of December 31, 2020, representing an increase to Cash of $55.6 million compared to December 31, 2019.
Key Product and Pipeline Highlights

Products

Vanda is encouraged by the strength of its commercial performance during the fourth quarter of 2020. Vanda continues to implement marketing and sales strategies aimed at supporting growth and minimizing the impact of disruptions caused by the COVID-19 pandemic, including the Fanapt for schizophrenia direct-to-consumer campaign, which was launched in 2020. Vanda is continuing its activities to support and facilitate the treatment of individuals in the U.S. living with Smith-Magenis Syndrome (SMS), and is committed to its awareness campaign and the support of patients suffering with Non-24-Hour Sleep-Wake Disorder.

Pipeline

Tradipitant

The gastroparesis Phase III clinical study (VP-VLY-686-3301) is ongoing. The study has a target enrollment of 200 randomized patients and is expected to complete enrollment in the first half of 2021, with a New Drug Application (NDA) filing projected in the second half of 2021.
The COVID-19 pneumonia Phase III clinical study (ODYSSEY VLY-686-3501) is ongoing.
HETLIOZ (tasimelteon)

In December 2020, the U.S. Food and Drug Administration (FDA) approved HETLIOZ capsule and liquid formulations for the treatment of adults and children, respectively, with nighttime sleep disturbances in SMS.1 HETLIOZ capsules, for adults with SMS, were immediately available after approval and the HETLIOZ LQ liquid formulation, for children with SMS, is expected to be available in the first quarter of 2021. SMS is estimated to affect 1/15,000-25,000 births in the U.S.2 HETLIOZ is the first and only FDA approved medication for patients with SMS.
A Phase III clinical study for HETLIOZ in delayed sleep phase disorder (DSPD) is expected to be initiated in the first quarter of 2021.
A clinical development program for HETLIOZ in autism spectrum disorder (ASD) is expected to be initiated in the first quarter of 2021.
Fanapt (iloperidone)

Development of the long acting injectable (LAI) formulation of Fanapt is ongoing.
A clinical program for Fanapt in Parkinson’s disease psychosis (PDP) is expected to begin in the first quarter of 2021.
GAAP Financial Results

Income before taxes was $10.9 million in the fourth quarter of 2020 compared to $5.8 million in the fourth quarter of 2019. Net income was $8.2 million in the fourth quarter of 2020 compared to net income of $4.2 million in the fourth quarter of 2019. Diluted net income per share was $0.15 in the fourth quarter of 2020 compared to diluted net income per share of $0.08 in the fourth quarter of 2019.

Income before taxes was $31.7 million for the full year 2020 compared to $29.0 million for the full year 2019. Net income was $23.3 million for the full year 2020 compared to net income of $115.6 million for the full year 2019. The full year 2019 net income of $115.6 million and the 2019 income tax benefit of $86.5 million include the favorable impact of the release of Vanda’s deferred tax asset valuation allowance.

Diluted net income per share was $0.42 for the full year 2020 compared to diluted net income per share of $2.11 for the full year 2019.

2021 Financial Guidance

Vanda expects to achieve the following financial objectives in 2021:

Conference Call

Vanda has scheduled a conference call for today, Wednesday, February 10, 2021, at 4:30 PM ET. During the call, Vanda’s management will discuss the fourth quarter and full year 2020 financial results and other corporate activities. Investors can call 1-866-688-9426 (domestic) or 1-409-216-0816 (international) and use passcode number 3557867. A replay of the call will be available on Wednesday, February 10, 2021, beginning at 7:30 PM ET and will be accessible until Wednesday, February 17, 2021 at 7:30 PM ET. The replay call-in number is 1-855-859-2056 for domestic callers and 1-404-537-3406 for international callers. The passcode number is 3557867.

The conference call will be broadcast simultaneously on Vanda’s website, www.vandapharma.com. Investors should click on the Investors tab and are advised to go to the website at least 15 minutes early to register, download, and install any necessary software or presentations. The call will also be archived on Vanda’s website for a period of 30 days.

Transcenta Holding Announces Appointment of Dr. Michael Shi as Executive Vice President, Head of Global R&D and Chief Medical Officer

On February 10, 2021 Transcenta Holding Limited (Transcenta), a global biotherapeutics company with fully-integrated capabilities in discovery, development and manufacturing of antibody-based therapeutics, reported the appointment of Dr. Michael Shi as Executive Vice President, Head of Global R&D and Chief Medical Officer (Press release, Transcenta, FEB 10, 2021, View Source [SID1234574876]). Dr. Shi will lead the global research and development of all our pipeline molecules and registration approvals. Dr. Li Xu, current acting Chief Medical Officer, will continue to help company and will serve on its scientific advisory board and as a strategic advisor to CEO.

Before joining Transcenta, Dr. Michael Shi was Global Program Clinical Head at Novartis Pharmaceuticals Corp. in East Hanover, New Jersey, USA. He played key leadership role in the clinical development of multiple novel oncology/hematology products, including Tabrecta (capmatinib), Zykadia (ceritinib), Adakveo (crizanlizumab), Exjade/Jadenu, BGJ398, TKI258 (dovitinib) and PDR001. Most recently, he led the development teams to achieve accelerated approval of Tabrecta and Zykadia in lung cancer indication and Adakveo in sickle cell disease, all under the US FDA breakthrough therapy designation.

"We are honored to have Dr. Shi joining the Transcenta team," said Dr. Xueming Qian, Co-Founder and CEO of Transcenta. "As a seasoned biopharmaceutical industrial expert, Dr. Shi worked extensively in the areas of clinical development, translational medicine, biomarker development, and discovery research in several well-known companies. He will lead our global clinical development and registration of all therapeutic area products."

"The biopharmaceutical industry has made great progress in China in recent years," said Dr. Shi. "As an integrated biopharmaceutical company focusing on innovative biological products, Transcenta has great potential. I’m very excited to join the company and look forward to accelerating the research and development of innovative biologic pipeline molecules with the excellent team, and benefiting patients around the world."

Dr. Shi received his medical education from Peking Union Medical College, a Ph.D. in Molecular Pharmacology and Toxicology from the University of Southern California and conducted a postdoctoral fellowship at Harvard Medical School. He also worked as the Program Director of Genetics Variation at NIH and an assistant professor at the University of Michigan Medical School.