On February 11, 2022 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that it recently completed a Pre-Biologics License Application ("pre-BLA") meeting with the U.S. Food and Drug Administration ("FDA") regarding a potential pathway for FDA approval of omburtamab for the treatment of patients with CNS/leptomeningeal metastases from neuroblastoma (Press release, Y-mAbs Therapeutics, FEB 11, 2022, View Source [SID1234608021]). The Company expects to resubmit the BLA for omburtamab by the end of the first quarter 2022.

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A data readout from a single-center clinical study (Study 03-133) of omburtamab conducted at Memorial Sloan Kettering ("MSK"), where 107 evaluable patients with CNS/leptomeningeal metastases from neuroblastoma received up to two doses of radiolabeled omburtamab, showed that patients had a median survival of 50.0 months, with the final median not yet being reached. The Company intends to submit the complete clinical data package in the BLA and announce the data later this year.

"We are pleased with the outcome of the pre-BLA meeting for omburtamab providing a clear regulatory path forward for the resubmission of the BLA. We believe omburtamab has the potential to make a meaningful impact in addressing a substantial unmet medical need for children suffering from high-risk neuroblastoma brain tumors and may potentially add an important treatment option to doctors and families facing this diagnosis," said Thomas Gad, founder, Chairman and President.

Dr. Claus Moller, Chief Executive Officer further notes, "We believe that we can resubmit the omburtamab BLA by the end of the first quarter 2022. We have been working closely with the agency to get to this point, and we will be applying for full approval. I am very grateful to the FDA and my team for the high-level constructive collaboration that has been exercised to get to this pivotal point."

Researchers at MSK developed omburtamab, which is exclusively licensed by MSK to Y-mAbs. As a result of the licensing arrangement, MSK has institutional financial interest related to the compound.

On February 11, 2022 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global, clinical-stage biotechnology company developing and manufacturing novel therapies, reported that it has achieved two milestones under its collaboration agreement with Janssen Biotech, Inc. for ciltacabtagene autoleucel (cilta-cel), resulting in aggregate payments to Legend Biotech of $50 million (Press release, Legend Biotech, FEB 11, 2022, View Source [SID1234608039]). Cilta-cel is a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor T-cell (CAR-T) therapy.

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Legend Biotech entered into the agreement with Janssen to develop, manufacture and commercialize cilta-cel for the treatment of multiple myeloma. Under the agreement, Legend Biotech received an upfront payment of $350 million and is entitled to receive additional payments upon achievement of landmarks for development, production performance, regulatory and sales.

The global agreement specifies a 50-50 cost and profit-sharing agreement in all markets, excluding Greater China, where the split is 70 percent for Legend and 30 percent for Janssen. Including the payments announced above, Legend has achieved $250 million in milestone payments during the collaboration.

About Cilta-cel
Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy, formerly identified as JNJ-4528 in the United States and Europe and LCAR-B38M CAR-T cells in China, that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and in earlier lines of treatment. The design consists of a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies. In addition to a Breakthrough Therapy Designation (BTD) granted in the United States in December 2019, cilta-cel received a Priority Medicines (PRiME) designation from the European Commission in April 2019, and a BTD in China in August 2020. In addition, Orphan Drug Designation was granted for cilta-cel by the U.S. Food and Drug Administration (FDA) in February 2019, and by the European Commission in February 2020. A Biologics License Application seeking approval of cilta-cel was submitted to the U.S. FDA and a Marketing Authorization Application was submitted to the European Medicines Agency.

On February 11, 2022 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, reported that it will report its fourth quarter and full year financial results on Thursday, February 24, 2022 (Press release, Iovance Biotherapeutics, FEB 11, 2022, View Source [SID1234608022]). Management will host a conference call and live audio webcast to discuss these results and provide a corporate update at 4:30 p.m. ET.

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To participate in the conference call, please dial 1-844-646-4465 (domestic) or 1-615-247-0257 (international) and reference the access code 2877242. The live webcast can be accessed in the Investors section of the Company’s website at www.iovance.com. The archived webcast will also be available for one year in the Investors section at www.iovance.com.

On February 11, 2022 Syncromune, Inc., a clinical stage biopharmaceutical company focused on the development of intratumoral immunotherapy reported that Charles J. Link, M.D., Executive Chairman & Chief Medical Officer of Syncromune will deliver a presentation at the 2022 BIO CEO & Investor Conference (Press release, Syncromune, FEB 11, 2022, View Source [SID1234608040]). Taking place February 14 – 17, 2022 at the New York Marriot Marquis, the conference will feature a hybrid of virtual and in-person participation.

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Dr. Link will be highlighting Syncromune’s recent corporate achievements, including the recent completion of the in-licensing of four target assets. He will also review the company’s proprietary Syncrovax platform, which is a next-generation personalized cancer therapy being developed to optimize intratumoral immunotherapy for the treatment of metastatic solid tumors.

Members of the company’s executive team will be on site for one-on-one meetings with investors and companies. A copy of the presentation will be available on Syncromune’s website following the conclusion of the BIO CEO & Investor Conference.

On February 11, 2022 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology platform company Drugging the Genome to address disease at the base level using a new class of precision genetic medicines, reported its financial results for the three-month period ended December 31, 2021, and other recent developments (Press release, NeuBase Therapeutics, FEB 11, 2022, View Source [SID1234607987]).

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"We are making significant progress in advancing the IND-enabling studies for the development candidate for our myotonic dystrophy type 1 (DM1) program, and we expect to file an IND with the FDA in Q4 CY2022," said Dietrich A. Stephan, Ph.D., Founder, Chief Executive Officer, and Chairman of NeuBase. "These studies are on track for data readouts to occur throughout CY2022, with the first presentation of rodent pharmacokinetic and bioavailability data to occur at an upcoming scientific meeting. We expect these data to illustrate the differentiated potential for our candidate to be a whole-body solution to treat DM1 and the unique ability of our delivery shuttle for distribution into the brain. The ability to cross the blood brain barrier and reach the deep brain is also especially relevant for our Huntington’s disease program, where we are planning to initiate scale-up and toxicology activities this year."

Dr. Stephan added, "In addition, I’m especially excited to have welcomed Todd to the executive team as Chief Financial Officer. The team and science are strong, and I believe we are at a pivotal moment for NeuBase as we are building a robust data package that is expected to support bringing our first candidate into the clinic for DM1, validate our genetic medicine technology platform to efficiently deliver genetic medicines with broad tissue distribution, including into the deep brain, and to precisely engage genetic mutations in a manner that is well-tolerated with the potential for sustained efficacy."

First Quarter of Fiscal Year 2022 and Recent Operating Highlights

Myotonic Dystrophy Type 1 (DM1) Program: NeuBase is making steady progress advancing IND-enabling studies for its development candidate in the DM1 program, which includes PK, absorption, distribution, metabolism, and excretion (ADME), and bioavailability via intravenous (IV) and subcutaneous (SQ) routes of administration, exploratory and IND-enabling Good Laboratory Practice (GLP) toxicology, and mechanism of action studies. In addition, GMP manufacturing of NeuBase’s development candidate to support Phase 1/2 clinical trials has been successfully implemented via contract manufacturing organizations. The Company plans to announce a robust data package through posters and presentations at scientific meetings and peer-reviewed publications throughout CY2022. NeuBase expects these data to support the submission of an IND filing to the FDA in the Q4 CY2022.
Huntington’s Disease (HD) Program: The HD program is currently in preclinical development. In CY2022, NeuBase expects to nominate a development candidate and initiate scale-up and toxicology activities to support an IND filing to the FDA in CY2023.
KRAS Oncology Program: The Company is conducting in vitro mechanistic studies and in vivo pharmacology studies for the KRAS program (KRAS G12V and G12D mutations).
Appointed New Chief Financial Officer: The Company appointed Todd P. Branning as CFO. Mr. Branning brings more than 25 years of experience leading corporate finance and accounting, tax, financial planning and analysis, and investor relations for several publicly traded pharmaceutical companies.
Financial Results for the Fiscal Quarter Ended December 31, 2021

As of December 31, 2021, the Company had cash and cash equivalents of approximately $47.3 million, compared with approximately $52.9 million as of September 30, 2021.
NeuBase estimates its current cash and cash equivalents are sufficient to fund currently planned operating and capital expenditures into the first quarter of CY2023.
For the fiscal quarter ended December 31, 2021, the Company reported a net loss of approximately $7.7 million, or a net loss of $0.24 per share, compared with a net loss of approximately $4.1 million, or a net loss of $0.18 per share, for the same period last year.
For the fiscal quarter ended December 31, 2021, total operating expenses were approximately $7.3 million, consisting of approximately $2.9 million in general and administrative expenses and $4.4 million of research and development expenses. This compares with total operating expenses of approximately $4.7 million for the same period last year, consisting of approximately $2.7 million in general and administrative expenses and $2.0 million in research and development expenses.