Ascendis Pharma A/S Reports Full-Year 2019 Financial Results

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.

Y-mAbs Announces Submission of Naxitamab Biologics License Application to U.S. FDA

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.

NEC charts global growth by appointing new CEO of Indian business

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.