On April 1, 2020 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, reported financial results for the full year ended December 31, 2019 (Press release, Ascendis Pharma, APR 1, 2020, View Source [SID1234556064]).

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"Following a transformative 2019, Ascendis remains on track with our corporate milestones for an even stronger 2020," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "Our flexible, global workforce, corporate structure and supply chain, supported by our information technology infrastructure, have allowed our teams to continue work unabated, guided by our corporate values and vision, and adapt to the global pandemic. We look forward to reporting our top-line results for the TransCon PTH PaTH Forward Trial mid-April, and submitting our marketing applications for TransCon hGH in the United States (U.S.) and Europe, as planned, in the second and fourth quarters, respectively. I would like to acknowledge the extraordinary commitment of our employees, the patients in our clinical trials, and the teams at our investigator sites to move forward during this time."

Corporate Highlights & Progress

TransCon hGH: TransCon hGH is an investigational long-acting prodrug of somatropin (human growth hormone or hGH) that releases somatropin in phase 3 development as a once-weekly treatment for growth hormone deficiency (GHD):
— Following discussions with the U.S. Food and Drug Administration (FDA), submitted an Investigational New Drug amendment to initiate the global, phase 3 foresiGHt Trial in adult GHD. The foresiGHt Trial is expected to begin enrollment later this year.
— Held two pre-BLA meetings with FDA to review the Chemistry, Manufacturing and Controls (CMC), and clinical/non-clinical packages for TransCon hGH as a potential treatment for pediatric GHD. The company is on track for planned submission of a Biologics License Application (BLA) to the FDA in the second quarter of 2020 and a Marketing Authorisation Application (MAA) to the European Medicines Agency in the fourth quarter of 2020.
— Received Orphan Designation from the European Commission for TransCon hGH in pediatric GHD.
— Advanced TransCon hGH in Greater China following initiation of a phase 3 trial for TransCon hGH in pediatric GHD by VISEN Pharmaceuticals, the company’s strategic investment to establish global reach in Greater China.
TransCon PTH: TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in development as a once-daily replacement therapy for hypoparathyroidism (HP) designed to replace PTH at physiologic levels for 24 hours each day, and address both short-term symptoms and long-term complications of the disease:
— Completed enrollment of 59 subjects in the PaTH Forward Trial, a global phase 2 trial evaluating the safety, tolerability and efficacy of TransCon PTH in adult HP subjects.
— The company expanded the trial in November 2019 to expedite the enrollment of subjects affected by the NATPARA recall. Final enrollment of PaTH Forward included 17 subjects previously treated with NATPARA.
— The goal of PaTH Forward is to identify a starting dose (15, 18, or 21 mg per day) for a pivotal phase 3 trial, establish a titration regimen for complete withdrawal of standard of care (i.e., active vitamin D and calcium supplements), and evaluate TransCon PTH control of serum and urinary calcium.
— Following the one-month blinded portion of PaTH Forward, subjects entered an open-label extension where they will receive a customized maintenance dose of TransCon PTH (6 to 30 mg per day) titrated to optimize their calcium control and evaluated on the primary composite endpoint, both as planned for phase 3. Fifty-nine subjects completed the blinded portion, and 58 subjects continued in the open-label extension, with one subject withdrawing for reasons unrelated to safety or efficacy of the study drug.
— The company expects to report top-line results from the one-month blinded portion of PaTH Forward in mid-April, with six-month data from the open-label extension expected during the third quarter of 2020.
TransCon CNP: TransCon CNP is an investigational long-acting prodrug of C-type natriuretic peptide (CNP) in development as a therapy for children with achondroplasia (ACH), the most common form of dwarfism, for which there is no FDA-approved treatment. TransCon CNP is designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose:
— The ACcomplisH Trial is a global, phase 2, randomized, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of TransCon CNP at escalating doses in children (ages 2 to 10 years) with ACH. The company continues to work towards escalating sequential dose cohorts throughout the year, while ensuring the safety of subjects during the current pandemic and access to investigator site staff for future monitoring visits.
— VISEN Pharmaceuticals remains on track to initiate a phase 2 trial in children with ACH during the fourth quarter of 2020.
Oncology: The company advanced its pipeline of multiple programs in oncology for clinically validated pathways, including TransCon IL-2 b/g, TransCon TLR7/8 Agonist and TransCon VEGF-TKI, with the goal to file an IND or equivalent for the company’s first oncology candidate in the fourth quarter of 2020.
Corporate milestones remain on track for 2020 despite the current global pandemic. The company continues to monitor and adapt to the impact of COVID-19 and expects to provide further updates to the investment community if the update is warranted.
Ended 2019 with cash and cash equivalents of €598.1 million.
Full Year 2019 Financial Results

For the full year 2019, Ascendis Pharma reported a net loss of €218.0 million, or €4.69 per share (basic and diluted) compared to a net loss of €130.1 million, or €3.17 per share (basic and diluted) for the same period in 2018.

Revenue for 2019 was €13.4 million compared to €10.6 million during 2018. The increase reflects recognition of revenue related to our strategic investment in VISEN Pharmaceuticals.

Research and development (R&D) costs for 2019 were €191.6 million compared to €140.3 million during 2018. Higher R&D costs in 2019 reflect an increase in personnel and external costs for development and manufacturing of TransCon hGH, TransCon PTH and TransCon CNP, and other research programs, including oncology.

General and administrative expenses for 2019 were €48.5 million compared to €25.1 million during 2018. The increase is primarily due to higher personnel-related costs and other increasing costs of preparing to become a commercial organization.

As of December 31, 2019, Ascendis had cash and cash equivalents of €598.1 million compared to €277.9 million as of December 31, 2018. As of December 31, 2019, Ascendis Pharma had 47,985,837 ordinary shares outstanding.

Conference Call and Webcast information

Ascendis Pharma will host a conference call and webcast today at 4:30 p.m. Eastern Time (ET) to discuss its full year 2019 financial results. Details include:

Date April 1, 2020
Time 4:30 p.m. ET
Dial In (U.S.) 844-290-3904
Dial In (International) 574-990-1036
Access Code 7387576
A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will also be available on this website shortly after conclusion of the event for 30 days.

On April 1, 2020 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 that the Company has completed the submission of its Biologics License Application ("BLA") under the FDA’s Rolling Review process for naxitamab after market close on March 31, 2020. Naxitamab is an investigational, monoclonal antibody that targets GD2 (Press release, Y-mAbs Therapeutics, APR 1, 2020, View Source [SID1234556063]). The naxitamab BLA is for the treatment of patients with relapsed/refractory high-risk neuroblastoma. The submission is based on the safety and efficacy results of the pivotal Phase 2 studies 201 and 12-230, which the Company expects to present at a venue later this year.

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"As the father of a long-term high-risk neuroblastoma survivor, I am excited to see Y-mAbs first BLA submission in neuroblastoma completed. We believe this is a key milestone for families facing high-risk neuroblastoma and for Y-mAbs. We are very grateful to all our employees, consultants and clinical sites involved in developing naxitamab," stated Thomas Gad, Founder, Chairman, President and Head of Business Development and Strategy.

Dr. Claus Moller, Chief Executive Officer, continued, "With this submission, we look forward to working with the Agency to bring naxitamab to appropriate patients. We are excited to complete this submission and believe naxitamab can address a significant unmet medical need for children with relapsed/refractory high-risk neuroblastoma."

Researchers at Memorial Sloan Kettering Cancer Center ("MSK") developed naxitamab, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests in the product and in Y-mAbs.

On April 1, 2020 NEC Corporation and NEC Technologies India reported the appointment of Mr. Aalok Kumar as the new President and Chief Executive Officer (CEO) of NEC Technologies India (Press release, NEC, APR 1, 2020, View Source [SID1234556061]).

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This change reflects NEC Corporation’s strong commitment to the country and India’s growing importance to the company’s global business.

With this appointment, Mr. Takayuki Inaba, former Chairman & Managing Director, has been appointed as Executive Chairman of NEC Technologies India.

Mr. Akihiko Kumagai, President of the Global Business Unit, NEC Corporation, said, "This management restructuring exercise is aimed at accelerating our evolution in India. Capacity building in India will not only bolster our business in this country but also support our global businesses."

Mr. Kumagai continued, "Aalok is an accomplished leader who brings a good mix of strategic thinking and operational excellence. His rich experience in business transformation and performance improvement, especially in Japan and other Asian markets, will help us accelerate our growth. With him onboard, we are confident that he will take NEC Technologies India to even greater heights and help India achieve its goals."

Mr. Aalok Kumar, President & CEO, NEC Technologies India, said, "India is a key market in NEC’s global growth strategy. With our huge pool of engineering and technology talent across various verticals, including public safety, communications infrastructure, aviation, logistics and transportation solutions, NEC is contributing significantly to the digital transformation of India."

"Our R&D efforts at NEC Laboratories India have enabled our ‘In India- For India’ strategy to develop innovative solutions that create social value and are globally scalable," he added.

Supported by a strong and talented workforce of over 6,000 local employees, NEC has successfully deployed a number of cross industry solutions for the infrastructure, aviation, logistics and transportation sectors.

Some key NEC projects in India include the Chennai-Andaman submarine cable project, which will bridge the digital gap between Andaman and Nicobar Islands by ensuring speedy internet access. In the aviation sector, NEC’s biometric-based paperless boarding solution will enable a seamless airport experience for passengers.

Container tracking and optimization solutions deployed by DMICDC Logistics Data Services – a joint venture between the Government of India and NEC – has allowed efficient management of 95% of the import-export container traffic at seaports throughout India, resulting in improved port operations across the country.

In collaboration with local government authorities, NEC is working on a number of smart city projects as well by setting up digital command and control centers for traffic management and public safety.

By collaborating with NEC Laboratories India in Bangalore, NEC Technologies India develops solutions across verticals such as Big Data, biometrics, mobile communications and retail that can be deployed both locally and internationally.

NEC Technologies India currently operates offices in New Delhi (head office), Ahmedabad, Bengaluru, Chennai, Mumbai, Noida and Surat.

On March 31 2020 A team led by Pawel Kalinski, MD, PhD, of Roswell Park Comprehensive Cancer Center reported that it has earned a five-year, $14.54 million award from the National Cancer Institute (NCI) to expand a promising immunotherapy platform. Funded through the NCI’s Program Project Grant program, this prestigious five-year grant will fund five clinical trials, all focused on a strategy for making some of the most common immunotherapies work for more cancer patients (Press release, OmniSeq, MAR 31, 2020, View Source [SID1234556463]).

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"Our goal with this project is to convert cancers that are traditionally checkpoint-resistant into treatable, ‘hot’ tumors so that more patients will be able to benefit from some of the most commonly prescribed immunotherapies," says Dr. Kalinski, who is Vice Chair for Translational Research, the Rustum Family Professor for Molecular Therapeutics and Translational Research and Director of Cancer Vaccine and Dendritic Cell Therapies at Roswell Park.

"This five-year National Cancer Institute grant continues a long tradition of groundbreaking immunotherapy research at Roswell Park Comprehensive Cancer Center," says Congressman Brian Higgins. "The research the clinicians at Roswell are conducting represents the next generation of transformational care for cancer patients."

"Cancer touches every family in one way or another," says Congressman Tom Reed. "We are pleased to see this critical grant funding delivered to right here in our backyard to promote innovative breakthroughs for cancer treatment. We will continue to be a constant advocate for Roswell Park in the halls of Congress."

Currently, depending on their cancer type and the genetic characteristics of their tumors, only about 20% of cancer patients are good candidates for a newer class of treatments called checkpoint inhibitors — drugs such as pembrolizumab (Keytruda), nivolumab (Opdivo) and ipilimumab (Yervoy). Even among those patients who are likely to respond initially to treatment with a checkpoint inhibitor, 60% to 70% are likely to have their cancers recur or progress.

"Dr. Kalinski and colleagues have built a compelling case in support of their out-of-the-box approach to cancer immunotherapy," says Kunle Odunsi, MD, PhD, FRCOG, FACOG, Deputy Director and Executive Director of the Center for Immunotherapy at Roswell Park Comprehensive Cancer Center. "All of us in the field will be eagerly following this elegant work, which involves combinations that would uniquely harness previously untapped properties of the human immune system, and accompanied by innovative translational science that should yield greater understanding of immune responses to solid tumors."

Dr. Kalinski has spent the last 15 years developing a unique approach to cancer immunotherapy, or cancer treatment strategies that engage the immune system — first as a faculty member at University of Pittsburgh Medical Center (UPMC), and more recently at Roswell Park.

The NCI grant, which involves partners from both UPMC and The Tisch Cancer Institute at Mount Sinai, will enable five new phase II or phase IIA clinical trials to assess the efficacy of this multipronged approach — two studies in patients with metastatic colorectal cancer, one in patients with checkpoint-resistant advanced melanoma and two in advanced ovarian cancer. Three of the five studies will be conducted at Roswell Park, with the two ovarian cancer studies to be led at UPMC’s Hillman Cancer Center, with participation from Roswell Park.

Chemokines are a type of signaling protein with the ability to control the movements and growth of many immune cells, including cancer-fighting cells called cytotoxic T lymphocytes (CTLs). Together, the trials will explore three complementary strategies for targeting chemokines to alter the tumor microenvironment, or cells surrounding cancer cells, to promote the attraction and activation of CTLs, the key antitumor immune cells.

"Based on our earlier studies both in the lab and in early-phase clinical trials, we have evidence that we can selectively promote entry of antitumor killer cells into tumor microenvironments and reduce local accumulation of suppressive cells in order to sensitize ‘cold’ tumors to immune checkpoint-inhibition therapy," notes Dr. Kalinski.

The clinical trials will employ a chemokine-modulating regimen targeting three separate pathways of immune response — the toll-like receptor-3, type-1 interferon and prostaglandin networks — as well as specialized dendritic-cell therapeutic vaccines.

The team will also work to assess the immunologic and clinical efficacy of this new therapeutic strategy; identify the biomarkers of response; and develop optimized treatment combinations for many patients with hard-to-treat ovarian and colorectal tumors. The program will also address the pressing challenge of determining the best treatment for patients with melanoma whose tumors recur or persist despite treatment with immunotherapy.

OmniSeq Inc. will conduct advanced molecular diagnostic testing on participating patients’ tumor cells as part of the studies. AIM ImmunoTech Inc. has agreed to provide rintatolimod (brand name Ampligen), the chemokine-modulating agent to be incorporated into these studies.

The clinical trials are not yet underway. Dr. Kalinski and team expect to open all five studies by year-end 2021. When active and open to accrual, the studies will be listed on both roswellpark.org/clinicaltrials and clinicaltrials.gov.

On March 31, 2020, Bellicum Pharmaceuticals, Inc. (the "Company") reported that it has entered into a Second Amendment to Loan and Security Agreement (the "Amendment") with Oxford Finance LLC (the "Collateral Agent") and the lenders listed on Schedule 1.1 to the Loan Agreement (as defined below) or otherwise party thereto from time to time (the "Lenders"), effective as of March 31, 2020, related to the Company’s Loan and Security Agreement, dated as of December 21, 2017, as amended on December 24, 2019 (the "Loan Agreement"), by and among the Company, the Collateral Agent and the Lenders, in connection with the proposed sale of certain assets of the Company pursuant to the previously announced asset purchase agreement, dated as of January 21, 2020 (the "Asset Purchase Agreement"), by and between the Company and The University of Texas M.D. Anderson Cancer Center (Filing, 8-K, Bellicum Pharmaceuticals, MAR 31, 2020, View Source [SID1234556144]). Pursuant to the Amendment, the Loan Agreement was amended to, among other things: (i) provide for the Collateral Agent’s and the Lenders’ consent to the Company’s consummation of such asset sale pursuant to the Asset Purchase Agreement, provided such sale occurs on or prior to June 30, 2020; (ii) if such asset sale occurs on or prior to June 30, 2020, extend the interest-only period through as late as July 31, 2021; (iii) if the proposed asset sale closes on or prior to June 30, 2020, provide for a partial repayment to the Lenders of a significant percentage of the proceeds of the asset sale that varies in accordance with the timing of closing and the associated amortization schedule, a portion of which will be applied as partial payment of the Final Payment Percentage (as defined in the Loan Agreement); and (v) grants the Lenders and the Collateral Agent a security interest in the Company’s intellectual property as of the date of the Amendment, in each case as set forth in the Amendment. In the event the proposed asset sale does not close on or prior to June 30, 2020, the Amendment provides that the Company, the Collateral Agent and the Lenders shall renegotiate the foregoing terms.

The foregoing description of the Amendment is only a summary and is qualified in its entirety by reference to the Amendment. The Company intends to file a copy of the Amendment as an exhibit to its Quarterly Report on Form 10-Q for its fiscal quarter ending March 31, 2020.

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