On April 1, 2020 Veracyte, Inc. (Nasdaq: VCYT) reported results of a study that identified over 100 novel or rare NTRK, ALK, RET and BRAF fusions in fine needle aspiration (FNA) samples of patients undergoing evaluation for thyroid cancer (Press release, Veracyte, APR 1, 2020, View Source [SID1234556070]). These gene fusions – 70 of which are previously unreported – may potentially be targeted with specific kinase inhibitor drugs that are currently available or in development for use in thyroid cancer patients. Veracyte also announced that it has launched an expanded version of its Afirma Xpression Atlas (XA) test, which uses RNA sequencing to detect gene alterations – including these novel or rare fusions – at the time of diagnosis.

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The new data are featured in an abstract that was accepted for an oral presentation this week at ENDO 2020*, the annual meeting of The Endocrine Society. The study was one of two Afirma XA-related abstracts accepted for the meeting.

For the new study, researchers performed RNA whole-transcriptome sequencing on over 37,000 thyroid nodule FNA samples whose cytopathology results were either indeterminate (Bethesda III/IV) or suspicious for cancer (Bethesda V/VI). They found 104 novel or rare gene fusions – 7 NTRK1/3, 8 ALK, 17 RET and 72 BRAF – none of which were previously reported for thyroid cancer in The Cancer Genome Atlas (TCGA) program, a United States government catalogue of gene alterations associated with cancer. The authors subsequently examined over 50,000 FNA samples that had undergone testing with Veracyte’s Afirma Genomic Sequencing Classifier (GSC) and found that none of the novel or rare gene fusions were detected among those deemed benign. NTRK, ALK, RET and BRAF fusions, including those in the new study, were identified in 3.2 percent of the 16,594 Bethesda III/IV samples that were deemed suspicious for cancer by the Afirma GSC and in 8.0 percent of the 1,692 Bethesda V/VI samples.

"Identification of receptor tyrosine kinase fusions is important because they are potential targets for small molecule inhibitors that are now FDA-approved or in clinical trials to treat advanced thyroid cancers," said Lori J. Wirth, M.D., medical director of the Center for Head and Neck Cancers at Massachusetts General Hospital and lead author of the study. "By examining a large cohort of patients with RNA whole-transcriptome sequencing, we identified potentially actionable RTK fusions in thyroid nodules beyond those described in TCGA, which may have an impact on treatment decisions for many patients."

Veracyte also announced the introduction of its expanded Afirma XA, which provides physicians with additional gene alteration content – including the novel or rare NTRK, ALK, RET and BRAF fusions from the new study – to further inform surgery and treatment decisions for patients with suspected or confirmed thyroid cancer. The Afirma XA utilizes RNA sequencing on the same FNA sample used for Afirma GSC testing. Compared to the original gene alteration panel, the expanded Afirma XA now reports 905 DNA variants (versus 761) and 235 RNA fusion partners (versus 130) in 593 genes (versus 511).

"By including additional gene fusion data that can potentially be targeted with new therapies, the expanded Afirma XA provides physicians with even more powerful information with which to guide surgery and treatment decisions in their patients with thyroid cancer," said Richard T. Kloos, M.D., senior medical director of endocrinology at Veracyte. "Importantly, the Afirma XA provides this information pre-operatively through a minimally invasive FNA sample."

In the second Afirma XA-focused abstract accepted for ENDO 2020 – as a poster presentation – researchers reported on the positive predictive value of TP53 gene variants among thyroid nodules deemed suspicious for cancer by the Afirma GSC following indeterminate cytopathology results.

About Afirma

The Afirma Genomic Sequencing Classifier (GSC) and Xpression Atlas provide physicians with a comprehensive solution for thyroid nodule diagnosis. The Afirma GSC was developed with RNA whole-transcriptome sequencing and machine learning and helps identify patients with benign thyroid nodules among those with indeterminate cytopathology results in order to help patients avoid unnecessary diagnostic thyroid surgery. For those with suspected thyroid cancer, the Afirma Xpression Atlas provides physicians with genomic alteration content from the same fine needle aspiration samples that are used in Afirma GSC testing and may help physicians decide with greater confidence on the surgical or therapeutic pathway for their patients.

On April 1, 2020 Forbius, a clinical-stage protein engineering company that develops biotherapeutics to treat cancer and fibrosis, reported that Ilia Tikhomirov, CEO of Forbius, will present a corporate update at the upcoming Solebury Trout Virtual Investor Conference (Press release, Forbius, APR 1, 2020, View Source [SID1234556068]).

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The 25 minute presentation includes an interactive Q&A with participants prompted to submit questions electronically. Details are as follows:

Date/Time:

Thursday, April 2, 2020, at 2:30p.m. EDT

Access: https://78449.themediaframe.com/dataconf/productusers/solebury/mediaframe/36519/indexl.html

On April 1, 2020 iTeos Therapeutics Inc., a privately-held clinical-stage biotechnology company developing innovative cancer immunotherapies, reported the closing of an oversubscribed Series B2 financing, which raised a total of $125 million (Press release, iTeos Therapeutics, APR 1, 2020, View Source [SID1234556067])

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The Series B2 financing round was co-led by RA Capital Management and Boxer Capital, and included new investors Janus Henderson Investors, RTW Investments and Invus along with existing investors MPM Capital, HBM Partners, 6 Dimensions Capital, Curative Ventures, Fund+, VIVES Louvain Technology Fund, SRIW, and SFPI.

Proceeds from the financing will support the Company’s continued growth and advance the clinical development of its two lead product candidates, EOS-850, a best-in-class adenosine A2A receptor antagonist and EOS-448, an ADCC-enabled anti-TIGIT antibody. EOS-850 is currently being evaluated in a Phase 1/2 clinical trial both as a single agent and in combination in several solid tumor indications. The Company plans to initiate dosing for the combination cohorts for this trial in the second quarter of 2020. The Company also recently initiated the Phase 1 portion of a Phase 1/2 clinical study of EOS-448, currently being evaluated in patients with solid tumors and hematological malignancies. Proceeds from this financing will also support the advancement of additional first-in-class preclinical programs targeting the adenosine pathway and Tregs.

"We are very pleased to have the strong support of this leading class of investors who share our excitement for the highly innovative oncology therapies we have developed at iTeos," said Michel Detheux, PhD, President and Chief Executive Officer at iTeos. "We look forward to accelerating and expanding our clinical development efforts to identify the most promising indications and combinations for people suffering with cancer. This financing maximizes our ability to execute our development plans alone or in partnership."

"iTeos’ pipeline of best-in-class agents has the potential to usher in a new wave of immuno-oncology therapeutics and the Company is well-positioned to make a significant, positive impact in the treatment of patients with a wide variety of cancer types," commented Derek DiRocco, Principal, RA Capital Management. "We are thrilled to collaborate with this experienced group of high-quality investors and partner with the iTeos management team as they continue to innovate and progress these exciting programs through clinical development."

"The iTeos team has done a remarkable job developing a pipeline of differentiated, potentially best-in-class cancer therapies," said Aaron Davis, Co-Founder and Chief Executive Officer, Boxer Capital of the Tavistock Group. "We are pleased to be joining them as they continue to execute and implement their vision for transforming the cancer treatment landscape."

Derek DiRocco and Aaron Davis will join the iTeos Board as Non-Executive Directors.

On April 1, 2020 Blueprint Medicines Presented the Corporate Presentation (Presentation, Blueprint Medicines, APR 1, 2020, View Source [SID1234556065]).

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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.