On March 22, 2019 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq:YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for 2018 (Press release, Y-mAbs Therapeutics, MAR 22, 2019, View Source [SID1234534567]).

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"We are very pleased to report our first full year financials after Y-mAbs’ successful IPO in September, which we believe has put us in a strong financial position to continue the important work on our two lead pediatric compounds, naxitamab and omburtamab. We plan to file BLAs for both of these compounds by the end of 2019." stated Thomas Gad, Founder, President and Head of Business Development and Strategy.

Dr. Moller, Chief Executive Officer continued, "We believe we have made solid clinical progress with naxitamab and omburtamab during 2018. In 2019, we have much to do and also many catalysts that have the potential to solidify us as a leader in pediatric oncology and as a company focused on rapidly developing therapies to extend and enhance the lives of those living with rare pediatrics cancers."

Financial Year 2018 and Corporate Development Highlights

·On December 13, 2018, Y-mAbs announced the appointment of Dr. Gérard Ber, PhD to its board of directors, and the planned departure of Dr. Michael Buschle, PhD. Dr. Ber most recently served as Chief Operating Officer of Advanced Accelerator Applications SA, which he co-founded in 2002. Dr. Ber brings over 30 years of experience in molecular nuclear medicines, including development, production and commercialization of diagnostics and therapeutic products for several indications in oncology, cardiology, neurology and infectious/inflammatory diseases.

·On December 10, 2018, Y-mAbs announced FDA clearance of the IND for the Company’s humanized bispecific GD2 antibody. The IND was submitted by Memorial Sloan Kettering Cancer Center, who has licensed the antibody to Y-mAbs. A Phase 1/2 clinical trial has been initiated for patients with relapsed/refractory neuroblastoma, high grade osteosarcoma and other GD2(+) solid tumors, where patients have relapsed or refractory disease that is resistant to standard therapy. Y-mAbs expects that this bispecific GD2 antibody may have potential advantages over other bispecific antibodies, such as improved potency due to bivalency, binding to neonatal Fc receptor and longer serum half-life, which obviates continuous infusion and enables more convenient administration to the patient.

·On November 1, 2018, Y-mAbs announced that that Dr. Jeong A Park from the Department of Pediatrics of Memorial Sloan-Kettering Cancer Center would present preclinical data from the Company’s bispecific GD2 antibody at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Bispecific GD2 antibodies were tested in solid tumors in preclinical models with T-cells and were shown to exert anti-tumor effect against GD2(+) tumor xenografts or PDX tumors. Further, the bispecific GD2 antibodies induced rapid and quantitative T-cell homing to tumors, mediating antibody dependent T-cell mediated cytotoxicity (ADTC) against GD2, and were shown to infiltrate tumors with little to no immune response, also known as cold tumors.

·On October 23, 2018, Y-mAbs announced that the Committee for Orphan Medicinal Products of the European Medicines Agency recommended the granting of orphan medicinal product designation in the European Union for naxitamab for the treatment of relapsed or refractory high-risk neuroblastoma. Further, the positive opinion for orphan medicinal product designation had been sent to the European Commission, which subsequently granted the orphan drug designation.

·On September 25, 2018, Y-mAbs announced the closing of its initial public offering of 6,900,000 shares of its common stock, including the exercise in full of the underwriters’ option to purchase 900,000 additional shares of common stock, at a public offering price of $16.00 per share. The gross proceeds to Y-mAbs, before deducting underwriting discounts and commissions and estimated offering expenses payable by the Company, were approximately $110.4 million. All of the shares of common stock were offered by the Company, and Y-mAbs’ common stock is listed on The Nasdaq Global Select Market.

·On August 21, 2018, Y-mAbs announced that it had received Breakthrough Therapy designation for naxitamab, in combination with GM-CSF, for the treatment of high risk neuroblastoma refractory to initial therapy or with incomplete response to salvage therapy in patients older than 12 months of age with persistent, refractory disease limited to bone marrow with or without evidence of concurrent bone involvement.

· On July 10, 2018, Y-mAbs announced that it had entered into an exclusive sublicense with MabVax Therapeutics for its patented neuroblastoma vaccine, which is a bi-valent ganglioside based vaccine intended to treat neuroblastoma. The neuroblastoma vaccine was originally developed by Memorial Sloan Kettering Cancer Center and licensed to MabVax as part of a portfolio of anti-cancer vaccines. MabVax was granted Orphan Drug Designation for the vaccine. Under the terms of the sublicense, the Company has agreed to pay MabVax up to $1.3 million, consisting of an upfront payment of $700,000, and an additional payment of $600,000 on the first anniversary of the sublicense.

·On April 16, 2018, Y-mAbs announced positive Phase I data from treatment with omburtamab in Desmoplastic Small Round Cell Tumor (DSRCT), which is a rare sarcoma of adolescents and young adults. The phase I study of radioiodinated omburtamab was conducted at Memorial Sloan Kettering Cancer Center evaluate toxicity, pharmacokinetics, biodistribution and efficacy. A total of 41 DSRCT patients were treated with escalated doses of intraperitoneal omburtamab as radioimmunotherapy, and the compound had a satisfactory safety profile and appeared to have activity against micro-metastatic DSRCT.

Financial Results

Y-mAbs reported a net loss of $43.3 million, or $1.50 per basic and diluted share, for the financial year 2018 compared to a net loss of $19.2 million, or $0.99 per basic and diluted share for 2017.

Cost and Operating Expenses

Research and development

Research and development expenses were $34.3 million for the year ended December 31, 2018, compared to $14.3 million for 2017, an increase of $20.0 million. The increase in research and development expenses primarily reflects the following:

·$7.5 million increase in outsourced manufacturing for our lead product candidates, naxitamab and omburtamab

·$4.6 million increase in outsourced research and supplies to support expanding development activities

·$2.8 million increase in clinical trial costs due to an increasing number of ongoing clinical trials

·$3.2 million increase in personnel costs

·$0.6 million increase in milestone payments

General and administration

General and administrative expenses were $9.0 million for the year ended December 31, 2018, compared to $4.9 million for the same period of 2017, an increase of $4.1 million. The increase in general and administrative expenses primarily reflects the following:

· $1.8 million increase in personnel costs

·$0.7 million increase in fees for auditors, legal advice and other consultancy services

·$0.4 million increase in leasehold expenses

Cash and Cash Equivalents

The Company had approximately $147.8 million in cash and cash equivalents as of December 31, 2018 compared to $90.5 million as of December 31, 2017. The increase is primarily driven by the $99.8 million net proceeds from the Company’s initial public offering, which is partly offset by the use of cash to fund the Company’s ongoing operations during 2018.

On March 22, 2019 Gossamer Bio, Inc. (Nasdaq:GOSS), a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics in the disease areas of immunology, inflammation and oncology, reported its financial results for the year ended December 31, 2018 and provided an update on recent corporate developments (Press release, Gossamer Bio, MAR 22, 2019, View Source [SID1234534560]).

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"Since the launch of Gossamer just over a year ago, we have built a robust and balanced pipeline of product candidates, including three clinical-stage assets and multiple preclinical programs, all with potential to meet high unmet medical needs across multiple disease areas," said Sheila Gujrathi, M.D., Gossamer’s Co-Founder and Chief Executive Officer. "With the recent successful completion of our initial public offering, we added to our already strong cash position, extending our runway and providing additional resources to rapidly advance our product candidates. I am extremely proud of all that the Gossamer team has accomplished to date. Our objective is to leverage our deep experience in immunology to enhance and extend the lives of patients with inflammatory and immune-related diseases, including cancer, and I look forward to sharing our progress as we work to bring forth important new medicines to patients."

Recent Highlights

Commenced Phase 2b trial for GB001. In October 2018, Gossamer commenced a Phase 2b trial of the company’s lead candidate, GB001, an oral antagonist of prostaglandin D2 receptor 2 (DP2), in patients with moderate-to-severe eosinophilic asthma. Gossamer expects to conduct an interim analysis for this trial in the first half of 2020 and to announce topline data from the trial in the second half of 2020.

Completed initial public offering (IPO). In February 2019, Gossamer closed its IPO of 19,837,500 shares of its common stock at a price to the public of $16.00 per share. Gossamer raised aggregate gross proceeds of $317.4 million from the offering, prior to deducting underwriting discounts and other offering expenses.

Full-Year 2018 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of December 31, 2018 were $228.7 million. Subsequent to year end, Gossamer raised approximately $295.2 million in its IPO, net of underwriting discounts.
Research and Development (R&D) Expenses: For the year ended December 31, 2018, R&D expenses were $55.3 million, which included $6.0 million of costs related to personnel and external consultants.
In-Process Research and Development (IPR&D) Expenses: For the year ended December 31, 2018, IPR&D expenses were $49.7 million, which was primarily attributable to approximately $27.5 million of costs related to up-front in-licensing and product acquisition payments related to GB004 and GB1275, and $19.1 million of costs associated with the issuance of stock in connection with the acquisition of GB001.
General and Administrative (G&A) Expenses: For the year ended December 31, 2018, G&A expenses were $44.1 million, which was primarily attributable to $30.3 million in stock-based compensation costs, $6.0 million in personnel-related costs, $3.7 million in professional fees, $1.3 million in legal fees and $0.8 million in facility-related costs.
Net Loss: For the year ended December 31, 2018, net loss was $147.0 million, or a loss of $22.59 per share.

On March 22, 2019 Novocure (NASDAQ: NVCR) reported that it has initiated INNOVATE-3, a phase 3 pivotal trial testing the efficacy of Tumor Treating Fields combined with paclitaxel in patients with recurrent, platinum-resistant ovarian cancer (Press release, NovoCure, MAR 22, 2019, View Source [SID1234534559]). INNOVATE-3 is Novocure’s fourth phase 3 pivotal trial initiated to study solid tumors beyond glioblastoma.

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"Ovarian cancer is one of the most aggressive forms of cancer," said Professor Ignace Vergote, Chairman of the Department of Obstetrics and Gynaecology and Gynaecologic Oncology at the Catholic University of Leuven, European Union. "Most ovarian cancer patients are diagnosed at an advanced stage, which makes the disease difficult to treat. Current treatment options are not enough for these patients. We are pleased to begin this trial that has the potential to improve survival in recurrent ovarian cancer."

INNOVATE-3, a prospective, open-label study, will include 540 patients with recurrent, platinum-resistant ovarian cancer. Patients will be randomized to receive either weekly paclitaxel alone or weekly paclitaxel in combination with Tumor Treating Fields tuned to 200 kHz until progression. The primary endpoint is overall survival. Secondary endpoints include progression free survival, objective response rate, severity and frequency of adverse events, time to undisputable deterioration in health-related quality of life or death, and quality of life. Patients may have had a maximum of two prior lines of systemic therapy following diagnosis of platinum-resistance.

Novocure developed the trial design for INNOVATE-3 after learning the results of its phase 2 pilot trial of Tumor Treating Fields in combination with weekly paclitaxel, the INNOVATE trial. In 31 evaluable patients, the INNOVATE trial suggested a more than doubling of progression free survival and an improvement in overall survival among patients who received Optune with paclitaxel compared to paclitaxel alone.

"INNOVATE-3 is Novocure’s fourth phase 3 pivotal trial beyond glioblastoma, demonstrating our commitment to developing Tumor Treating Fields for a variety of solid tumors," said Asaf Danziger, Novocure’s Chief Executive Officer. "At Novocure, we strive to extend survival in some of the most aggressive forms of cancer. Ovarian cancer has been an important area of focus for our research because of the great unmet need faced by these patients. We are now working closely with trial sites and institutional review boards to open sites and enroll patients as quickly as possible."

About Ovarian Cancer `

In the United States, ovarian cancer ranks fifth in cancer deaths among women, accounting for more deaths than any other cancer of the female reproductive system. Ovarian cancer incidence increases with age, and the median age at time of diagnosis is 63 years old. The incidence of ovarian cancer is approximately 22,500 new cases annually in the United States, approximately 68,000 new cases annually in Europe, and approximately 10,000 new cases annually in Japan. Tumor Treating Fields is not approved for the treatment of ovarian cancer by the U.S. Food and Drug Administration. The safety and effectiveness of Tumor Treating Fields for ovarian cancer has not been established.

On March 22, 2019 Genmab A/S (Nasdaq Copenhagen: GEN) reported that Janssen Pharmaceutica NV (Janssen) has submitted a Type II variation application to the European Medicines Agency (EMA) (Press release, Genmab, MAR 22, 2019, View Source [SID1234534557]). The application seeks to broaden the existing marketing authorization for daratumumab (DARZALEX) to include use in combination with lenalidomide and dexamethasone (Rd) as treatment for newly diagnosed multiple myeloma patients who are not candidates for high dose chemotherapy and autologous stem cell transplant (ASCT) . The submission is based on data from the Phase III MAIA study of daratumumab in combination with Rd as treatment for patients with newly diagnosed multiple myeloma, who are not candidates for high dose chemotherapy and ASCT. Data from the study was presented at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which took place in San Diego, California in December 2018. In August 2012, Genmab granted Janssen an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

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"At Genmab we’re committed to improving the lives of patients by creating and developing innovative antibody products. With this potential label expansion, we are hopeful that DARZALEX will become available to an even wider group of multiple myeloma patients in the first line setting," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

About the MAIA (MMY3008) study
The Phase III study (NCT02252172) is a randomized, open-label, multicenter study that includes 737 newly diagnosed patients with multiple myeloma who are not candidates for high dose chemotherapy and ASCT. Patients were randomized to receive either daratumumab in combination with lenalidomide (an immunomodulatory drug) and dexamethasone (a corticosteroid) or lenalidomide and dexamethasone alone. In the daratumumab treatment arm, patients received 16 milligrams per kilogram (mg/kg) weekly for first 8 weeks (Cycles 1 and 2), every other week for 16 weeks (Cycles 3 to 6) and then every 4 weeks (Cycle 7 and beyond) until progression of disease or unacceptable toxicity. Lenalidomide is administered at 25 mg orally on days 1 through 21 of each 28-day cycle, and dexamethasone was administered at 40 mg once a week for both treatment arms. Participants in both treatment arms will continue Rd until disease progression or unacceptable toxicity. The primary endpoint of the study is PFS.

About multiple myeloma
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Multiple myeloma is the third most common blood cancer in the U.S., after leukemia and lymphoma.2 Approximately 26,000 new patients were expected to be diagnosed with multiple myeloma and approximately 13,650 people were expected to die from the disease in the U.S. in 2018.3 Globally, it was estimated that 160,000 people were diagnosed and 106,000 died from the disease in 2018.4 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.5

About DARZALEX(daratumumab)
DARZALEX (daratumumab) injection for intravenous infusion is indicated in the United States in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.6 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S. In Japan, DARZALEX is approved in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for treatment of adults with relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market. For more information, visit www.DARZALEX.com.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).6,7,8,9,10

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and frontline multiple myeloma settings and in amyloidosis. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases, such as NKT-cell lymphoma, B and T-ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA, for multiple myeloma, as both a monotherapy and in combination with other therapies.

On March 22, 2019 Celyad (Euronext Brussels and Paris, and Nasdaq: CYAD), a clinical-stage biopharmaceutical company focused on the development of CAR-T cell-based therapies, reported that the Company will report full year 2018 financial and operating results on the evening of Thursday, March 28, 2019 (Press release, Celyad, MAR 22, 2019, https://www.celyad.com/en/news/celyad-to-announce-full-year-2018-financial-results-and-host-conference-call [SID1234534556]).

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Following the press release, Celyad management will host a conference call on Friday, March 29 at 1p.m. CET / 8a.m. EDT to discuss full year 2018 results and provide an update on the Company’s recent progress and upcoming milestones.

Participants may access the conference call by dialing +44 (0) 2071 928000. The conference ID for the call is 8745558. Alternatively, participants may access the conference call by dialing the following local numbers: Belgium 024009874, France 0176700794, Netherlands 0207143545 and U.S. 16315107495.

To access the subsequent archived recording, visit the "Events & Webcasts" section of the Celyad website.