On May 18, 2021 CEL-SCI Corporation (NYSE American: CVM) reported financial results for the quarter ended March 31, 2021, as well as key clinical and corporate developments (Press release, Cel-Sci, MAY 18, 2021, View Source [SID1234580223]).

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Clinical and Corporate Developments include:

In December 2020, CEL-SCI stated that its pivotal Phase 3 head and neck cancer study completed database lock and entered the statistical analysis phase. CEL-SCI remains blinded to the study data and is not involved in the analysis process which is conducted by independent contractors. The statistical analysis plan follows the protocol stated objectives and is designed to meet FDA requirements to define safety and efficacy, those clinical benefits that Multikine might provide for patients newly diagnosed with advanced primary (not yet treated) squamous cell carcinoma of the head and neck.
In preparation for the anticipated commercial launch of Multikine, CEL-SCI has been expanding and upgrading its dedicated cGMP manufacturing facility for Multikine. The construction, which began in 2020, is expected to be completed soon and will double the facility’s capacity to accommodate two shifts for increased production of Multikine.
"We believed in cancer immunotherapy when few did. We were the first to administer an immunotherapy drug right after diagnosis, ahead of surgery, radiation and chemotherapy, because we believe that the activation of an anti-tumor response by the immune system is best achieved before standard therapies damage it. We believe that it is possible to develop a non-toxic cancer drug. Our Phase 3 study took years longer than expected because it took longer than expected to reach the required number of events (patient deaths) in the comparator arms of the study. It is all about the final data," stated CEL-SCI CEO, Geert Kersten.

CEL-SCI reported an operating loss of $17.3 million for the six months ended March 31, 2021 versus an operating loss of $13.6 million for the six months ended March 31, 2020. CEL-SCI reported an operating loss of $8.5 million for the quarter ended March 31, 2021 versus an operating loss of $6.7 million for the quarter ended March 31, 2020.

During the six and three months ended March 31, 2021, CEL-SCI incurred approximately $6.9 million and $3.3 million, respectively, in capitalized costs to upgrade its manufacturing facility to prepare for the potential commercial production of Multikine. Total estimated costs of this upgrade are approximately $10.6 million, of which approximately $9.5 million has been incurred through March 31, 2021. The landlord of the property has agreed to finance the final $2.4 million of costs and allow for the repayment through increased lease payments beginning March 1, 2021.

On May 18, 2021 The Pershing Square Sohn Cancer Research Alliance reported the six winners of the 2021 Pershing Square Sohn Prize for Young Investigators in Cancer Research, awarded annually to cancer research scientists and physician-scientists based in the greater New York City area (Press release, The Pershing Square Sohn Cancer Research Alliance, MAY 18, 2021, View Source [SID1234580222]). The Prize, totaling $3.6 million, empowers investigators early in their independent careers to pursue research projects at a critical stage when traditional funding is lacking. Recipients receive $200,000 per year for three years.

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Over the past eight years, the Alliance has awarded over $30 million to 52 scientists. With this funding, the recipients have contributed greatly to New York’s growing biomedical research hub. In addition to funding, the Alliance provides Prize winners with opportunities to present their work to scientific and business audiences to encourage collaboration and help bridge the gap between academia and industry.

"We are in awe of the incredible research the scientists have demonstrated, particularly this past year amid such challenging circumstances," said Pershing Square Foundation Trustee Neri Oxman. "Our commitment to enabling early-stage research remains core to our mission, and we are proud to support this year’s prize winners and their innovative work."

The winners of the 2021 Pershing Square Sohn Prize are:

Sidi Chen, PhD, Yale School of Medicine: The laboratory of Dr. Chen works to create more effective cell-based immunotherapy against cancer. They have developed novel platforms for highly efficient and parallel engineering of therapeutic cell candidates. The goal of the project is to build transformative platforms for next generation cell therapy, an urgent unmet need since cell therapy has demonstrated little activity in solid tumors and refractory/recurrent hematological cancers.
Christine Iok In Chio, PhD, Columbia University Irving Medical Center: Dr. Chio’s project will employ genetic and chemical proteomic approaches to ascertain how reversible methionine (an amino acid) oxidation regulates the signaling pathways that promote pancreatic ductal adenocarcinoma. These insights will then be exploited to develop novel therapeutic strategies to combat this highly lethal form of pancreatic cancer.
Marcin Imielinski, MD, PhD, Weill Cornell Medicine: Using innovative methods to characterize structural changes in whole genome sequences, Dr. Imielinski hopes to explain why certain acral melanomas (which occur on the hands and feet) and other similar cancers with few small mutations respond to immunotherapy by examining a novel class of genetic alterations called tyfonas. This study can transform the basic understanding of cancer evolution and provide evidence for a whole genome sequencing immunotherapy biomarker to help navigate the best course for patient treatment.
Lilian Kabeche, PhD, Yale University School of Medicine: Dr. Kabeche’s goal is to understand how the diverse roles of a protein called ATR protects cells from chromosomal instability throughout the whole cell cycle. In particular, she focuses on pancreatic cells, which have extremely high rates of instability. By targeting the process leading to chromosomal instability, her work may lead to the creation of a novel therapy that selectively and effectively treats cancers that exhibit continuous chromosome missegregation.
Elena Piskounova, PhD, Weill Cornell Medicine: Using a combination of cutting-edge technologies, the laboratory of Dr. Piskounova will characterize how tRNA modifications enable adaptation and survival of metastasizing cancer cells under a variety of stresses throughout the metastatic cascade. This will ultimately allow the identification of the stress-response proteome as a driver of metastatic disease. By defining these molecular mechanisms in the context of metastasis, Dr. Piskounova will be able to identify novel targets that can be harnessed therapeutically to specifically eradicate deadly metastatic disease.
Xuebing Wu, PhD, Columbia University Irving Medical Center: Dr. Wu is working to develop a platform for precise elimination of tumor cells by directly targeting tumor mutations using a new CRISPR genome engineering system and advanced machine learning. If successful, this platform will accelerate the development of precision therapies for many types of cancer.
"We remain extremely committed to cancer research during this global pandemic. We are continually impressed by the superb quality of the proposals we receive as well as the exceptionally talented researchers that are working in the greater New York area institutions," said Olivia Tournay Flatto, PhD, Co-Founder and Executive Director of the Pershing Square Sohn Cancer Research Alliance and President of The Pershing Square Foundation. "It is our goal to build a community of creative and talented individuals, and to connect them with like-minded peers in pursuit of novel ideas, therapies, technologies, and, ultimately, knowledge that can further our understanding of disease and fundamental human biology."

"The work to find new treatments and cures for cancer continues to be an urgent global health priority so we are heartened by the research approaches of this year’s Prize winners," said Evan Sohn, Vice President of the Sohn Conference Foundation. "We are confident that with the passion, creativity, and insights we’ve seen from this group of scientists, they will make discoveries that will have a lasting impact for the patients for whom our Foundation fights."

As part of the selection process, the Pershing Square Sohn Cancer Research Alliance benefitted from the guidance of a highly accomplished advisory board.

Prize Advisory Board members include: Jeanne B. Ackman, MD, Director, Thoracic MRI, Radiologist, Massachusetts General Hospital, and Assistant Professor, Harvard Medical School; Mikael Dolsten, MD, PhD, President, Worldwide Research and Development, Pfizer, Inc.; Allan Goodman, PhD, President and CEO, The Institute of International Education; Pablo Legorreta, Founder and Chief Executive Officer, Royalty Pharma; Richard P. Lifton, MD, PhD, President, The Rockefeller University; Siddhartha Mukherjee, MD, PhD, Assistant Professor, Department of Medicine, Division of Oncology, Columbia University Medical Center, and Author of The Emperor of All Maladies: A Biography of Cancer and The Gene: An Intimate History; James E. Rothman, PhD, Fergus F. Wallace Professor of Biomedical Sciences, Yale University and 2013 Nobel Prize Winner in Physiology or Medicine; Bruce Stillman, PhD, President and CEO, Cold Spring Harbor Laboratory; Craig Thompson, MD, President and CEO, Memorial Sloan Kettering Cancer Center; and George D. Yancopoulos, MD, PhD, President and Chief Scientific Officer, Regeneron.

Additional details about the Prize winners can be found on the PSSCRA website at View Source

On May 18, 2021 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications reported its unaudited financial results for the first quarter of 2021 (Press release, Legend Biotech, MAY 18, 2021, View Source [SID1234580221]).

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"We built on the momentum of 2020 during the first quarter of this year for our BCMA CAR-T therapy cilta-cel with our collaboration partner, Janssen Biotech, Inc.*(Janssen), completing the Biologics License Application to the U.S. FDA and the Marketing Authorisation Application to the EMA," said Ying Huang, PhD, CEO and CFO of Legend Biotech. "We look forward to an exciting year with new and updated data from the CARTITUDE clinical development program and reaching our goal of bringing a new CAR-T treatment option to patients living with multiple myeloma worldwide pending regulatory approvals."

*In December 2017, Legend Biotech entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. to develop and commercialize cilta-cel.

First Quarter 2021 & Recent Highlights

In the first quarter of 2021, the rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) was completed by Legend Biotech’s collaborator, Janssen, for cilta-cel, for the treatment of adults with RRMM.
On April 30, 2021 the Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) was made by Legend Biotech’s collaborator, Janssen, for cilta-cel for the treatment of adults with RRMM. This follows the granting of an accelerated assessment for this MAA by the EMA’s Committee for Medicinal Products for Human Use (CHMP) in February 2021.
On May 13, 2021, Legend Biotech entered into a subscription agreement with an institutional investor for the offer and sale of 20,809,805 ordinary shares in a private placement at a purchase price of $14.41625 per ordinary share (equivalent to $28.8325 per American Depositary Share, or ADS) and the issuance of a warrant exercisable for up to an aggregate of 10,000,000 ordinary shares, exercisable for a two-year period at an exercise price of $20.00 per ordinary share (equivalent to $40.00 per ADS).
Key Upcoming Milestones

Updated clinical data, including longer term follow up results from the CARTITUDE-1 trial, will be presented at the virtual 2021 ASCO (Free ASCO Whitepaper) Annual Meeting taking place on June 4-8 , 2021 (oral presentation, abstract #8005) along with initial data from the CARTITUDE-2 trial (abstracts #8013, #8028). In addition, there will be three poster presentations featuring real-world data (abstracts #8045, #8030 and #8041).
Nine abstracts will be presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress taking place virtually on June 9-17, 2021. (abstracts #S190, #EP964, #EP1003, #EP987, #EP990, #EP1049, #EP978, #EP977 and #EP972).
Legend Biotech intends to use the data from the CARTIFAN-1 study in support of a regulatory submission to the China Center for Drug Evaluation (CDE) in the second half of 2021 seeking approval of cilta-cel for the treatment of adults with RRMM.
Legend Biotech’s collaboration partner, Janssen, anticipates submitting a New Drug Application (NDA) to the Japan Ministry of Health, Labor and Welfare (JMHLW) in the second half of 2021 seeking approval of cilta-cel for the treatment of adults with RRMM.
Legend Biotech expects to initiate its Phase 1 clinical trial of LB1901 in RR T-cell lymphoma (TCL) in the United States in 2021.
Legend Biotech anticipates supporting investigators with publishing a clinical data update from LEGEND-2 study in 2021.
Financial Results for First Quarter Ended March 31, 2021

Cash and Cash Equivalents and Time Deposits

As of March 31, 2021, Legend Biotech had approximately $412.3 million of cash and cash equivalents and approximately $50.0 million in time deposits.

Revenue

Revenue for the three months ended March 31, 2021 was $13.7 million compared to $11.5 million for the three months ended March 31, 2020. The increase of $2.2 million was primarily due to revenue recognition of additional milestone payment achieved pursuant to Legend Biotech’s agreement with Janssen. Milestone payments are constrained as a result of the uncertainty of whether the milestone will be achieved, but recognized when the associated milestone is achieved and the uncertainty relieved. In the first quarter of 2021, this resulted in a larger amount of revenue recognized from the contract liabilities. Legend Biotech has not generated any revenue from product sales to date.

Research and Development Expenses

Research and development expenses for the three months ended March 31, 2021 were $71.1 million compared to $48.0 million for the three months ended March 31, 2020. This increase of $23.1 million was primarily due to a higher number of clinical trials with more patients enrolled and a higher number of research and development product candidates.

Administrative Expenses

Administrative expenses for the three months ended March 31, 2021 were $8.7 million compared to $3.4 million for the three months ended March 31, 2020. The increase of $5.3 million was primarily due to Legend Biotech’s expansion of supporting administrative functions to aid continued research and development activities.

Selling and Distribution Expenses

Selling and distribution expenses for the three months ended March 31, 2021 were $13.4 million compared to $6.5 million for the three months ended March 31, 2020. This increase of $6.9 million was primarily due to increased costs associated with commercial preparation activities for cilta-cel.

Other Income and Gains

Other income and gains for the three months ended March 31, 2021 was $0.7 million compared to $2.5 million for the three months ended March 31, 2020. The decrease of $1.8 million was primarily due to lower government grant and interest income received in first quarter of 2021.

Other Expenses

Other expenses for the three months ended March 31, 2021 was $2.0 million compared to $0.05 million for the three months ended March 31, 2020. The increase was primarily due to higher foreign currency exchange loss in first quarter of 2021.

Finance Costs

Finance costs for the three months ended March 31, 2021 was $0.04 million compared to $4.0 million for the three months ended March 31, 2020. The decrease was primarily due to finance costs related to the issuance of convertible redeemable preferred shares in 2020, which were fully converted into ordinary shares upon the completion of Legend Biotech’s initial public offering in June 2020.

Loss for the Period

For the three months ended March 31, 2021, net loss was $80.9 million, or $0.30 per share, compared to a net loss of $44.2 million, or $0.22 per share, for the three months ended March 31, 2020.

On May 18, 2021 Novocure (NASDAQ: NVCR) reported the U.S. Food and Drug Administration (FDA) has approved the company’s Investigational Device Exemption (IDE) supplement, reducing the enrollment requirement for its LUNAR trial to 276 patients with 12 months follow-up (Press release, NovoCure, MAY 18, 2021, View Source [SID1234580220]). LUNAR is a phase 3 pivotal trial testing the effectiveness of Tumor Treating Fields in combination with immune checkpoint inhibitors or docetaxel versus immune checkpoint inhibitors or docetaxel alone for patients with stage 4 NSCLC who progressed during or after platinum-based therapy.

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The IDE supplement incorporates recommended changes from the interim analysis of the LUNAR trial conducted by an independent data monitoring committee (DMC). After review of the interim analysis report earlier this year, the DMC concluded that the LUNAR trial should continue with no evidence of increased systemic toxicity. The DMC also stated that it is likely unnecessary and possibly unethical for patients randomized to the control arm of the trial to continue accrual according to the original protocol. The DMC recommended a reduced sample size of approximately 276 patients with 12 months follow-up which it believes will provide sufficient overall power for both primary and secondary endpoints. The DMC recommended no other changes to the trial design. Novocure remains blinded to all data.

"We are very pleased with the FDA approval of the DMC’s recommended protocol adjustments and are grateful for the FDA’s prompt review," said Asaf Danziger, Novocure’s CEO. "We now anticipate last patient enrollment in the LUNAR trial in the fourth quarter of 2021 with final data available in 2022 and look forward to sharing data from the LUNAR trial as quickly as possible."

About LUNAR

LUNAR is a phase 3 pivotal trial testing the effectiveness of TTFields in combination with immune checkpoint inhibitors or docetaxel versus immune checkpoint inhibitors or docetaxel alone for patients with stage 4 NSCLC who progressed during or after platinum-based therapy. It is estimated that approximately 46,000 patients receive second-line treatment for stage 4 NSCLC each year in the U.S. The primary endpoint is superior overall survival of patients treated with TTFields plus immune checkpoint inhibitors or docetaxel versus immune checkpoint inhibitors or docetaxel alone. TTFields is intended principally for use in combination with other standard-of-care treatments, and LUNAR was designed to generate data that contemplates multiple outcomes, all of which Novocure believes will be clinically meaningful.

About Tumor Treating Fields

Tumor Treating Fields, or TTFields, are electric fields that disrupt cancer cell division.

When cancer develops, rapid and uncontrolled division of unhealthy cells occurs. Electrically charged proteins within the cell are critical for cell division, making the rapidly dividing cancer cells vulnerable to electrical interference. All cells are surrounded by a bilipid membrane, which separates the interior of the cell, or cytoplasm, from the space around it. This membrane prevents low frequency electric fields from entering the cell. TTFields, however, have a unique frequency range, between 100 to 500 kHz, enabling the electric fields to penetrate the cancer cell membrane. As healthy cells differ from cancer cells in their division rate, geometry and electric properties, the frequency of TTFields can be tuned to specifically affect the cancer cells while leaving healthy cells mostly unaffected.

Whether cells are healthy or cancerous, cell division, or mitosis, is the same. When mitosis starts, charged proteins within the cell, or microtubules, form the mitotic spindle. The spindle is built on electric interaction between its building blocks. During division, the mitotic spindle segregates the chromosomes, pulling them in opposite directions. As the daughter cells begin to form, electrically polarized molecules migrate towards the midline to make up the mitotic cleavage furrow. The furrow contracts and the two daughter cells separate. TTFields can interfere with these conditions. When TTFields are present in a dividing cancer cell, they cause the electrically charged proteins to align with the directional forces applied by the field, thus preventing the mitotic spindle from forming. Electrical forces also interrupt the migration of key proteins to the cell midline, disrupting the formation of the mitotic cleavage furrow. Interfering with these key processes disrupts mitosis and can lead to cell death.

TTFields is intended principally for use together with other standard-of-care cancer treatments. There is a growing body of evidence that supports TTFields’ broad applicability with certain other cancer therapies, including radiation therapy, certain chemotherapies and certain immunotherapies. In clinical research and commercial experience to date, TTFields has exhibited no systemic toxicity, with mild to moderate skin irritation being the most common side effect.

Fundamental scientific research extends across two decades and, in all preclinical research to date, TTFields has demonstrated a consistent anti-mitotic effect. The TTFields global development program includes a broad range of clinical trials across all phases, included four phase 3 pivotal trials in a variety of tumor types. To date, more than 18,000 patients have been treated with TTFields.

Use of Tumor Treating Fields for the treatment of NSCLC is investigational only.

On May 18, 2021 Brooklyn ImmunoTherapeutics, Inc. (NYSE American: BTX) ("Brooklyn" or the "Company"), a biopharmaceutical company currently focused on exploring the role that cytokine and gene editing/cell therapy can have in treating patients with cancer, blood disorders and monogenic diseases, reported that management will host a conference call on Tuesday, May 25, 2021 at 4:30 p.m. Eastern Time (ET) (Press release, Brooklyn ImmunoTherapeutics, MAY 18, 2021, View Source [SID1234580219]).

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Participants are asked to pre-register for the call through the following link: View Source Please note that registered participants will receive their dial in number upon registration and will dial directly into the call.

The conference call will also be available through a live webcast found here.

The Company invites participants to pre-submit questions to [email protected] until 5:00 p.m. Eastern Time (ET) on May 20, 2021 (live questions will not be accepted during the call). Please use the subject line, "BTX Call Questions" for this correspondence. The Company will do its best to address all pre-submitted questions at the conclusion of prepared remarks.