1stOncology™: Cancer Intelligence Service – Page 13490 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Y-mAbs Announces Positive Pre-BLA Meeting with FDA for Omburtamab

On February 26, 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 it has completed a positive Type B 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 26, 2020, View Source [SID1234554800]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

At the pre-BLA meeting, the Company reached alignment with the FDA on an Accelerated Approval Pathway for omburtamab along with a rolling BLA submission. The Company expects to complete the rolling BLA within approximately 10 weeks.

Under omburtamab’s breakthrough therapy designation ("BTD"), omburtamab qualifies for a rolling BLA submission, which allows for individual modules of the application to be submitted by the Company and reviewed by the FDA on a rolling basis, rather than waiting for all sections of the BLA application to be completed before submission. We believe the rolling application process will provide the Company with the opportunity for ongoing communications with the FDA, and, during this rolling process, the Company anticipates that it will be able to address any substantial matters raised by the FDA.

A previously announced data readout from a single-center study (Study 03-133), at Memorial Sloan Kettering Cancer Center ("MSK") where the 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 50.8 months, with the final median survival not yet being reached. The Company intends to announce the complete clinical data package later this year. In addition, the Company is planning for submission of a Marketing Authorization Application in Europe in the fourth quarter this year.

"We are very pleased with the positive outcome of the Pre-BLA meeting for omburtamab providing a clear regulatory path forward for a rolling BLA submission. We believe omburtamab to be essential in addressing substantial unmet medical needs for children suffering from high-risk neuroblastoma brain tumors," said Thomas Gad, Founder, Chairman, President and Head of Business Development and Strategy.

Dr. Claus Moller, Chief Executive Officer further notes, "We are pleased to firm up the timelines for the omburtamab BLA. We expect to complete submission of the BLA for naxitamab in late March and omburtamab shortly thereafter, and this execution reflects an outstanding performance of our team. We hope to see both compounds approved by the FDA later this year."

Researchers at MSK developed the omburtamab antibody, 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.

OPKO Health Reports 2019 Fourth Quarter Business Highlights and Financial Results

On February 26, 2020 OPKO Health, Inc. (NASDAQ: OPK) reported business highlights and financial results for the three months ended December 31, 2019 (Press release, Opko Health, FEB 26, 2020, View Source [SID1234554799]).

Business Highlights

Successful Phase 3 results for Somatrogon, an investigational long-acting human growth hormone to treat children with growth hormone deficiency: On October 21, 2019, OPKO and Pfizer announced the global Phase 3 trial evaluating somatrogon dosed once-weekly in pre-pubertal children with growth hormone deficiency (GHD) met its primary endpoint of non-inferiority to daily GENOTROPIN (somatropin) for injection, as measured by annual height velocity at 12 months.

Somatrogon Phase 3 clinical trial data to be presented at ENDO on March 28, 2020: Two oral presentations will be delivered at the Endocrine Society’s ENDO 2020 meeting, to be held March 28-31, 2020:

"Somatrogon Growth Hormone in the Treatment of Pediatric Growth Hormone Deficiency: Results of the Pivotal Phase 3" and

"Interpretation of Insulin-like Growth Factor (IGF-1) Levels Following Administration of Somatrogon (a long-acting Growth Hormone-hGH-CTP)"

Somatrogon regulatory submissions: Regulatory submission in the U.S. is anticipated in 2H 2020: Europe is expected to follow upon completion of the open-label study demonstrating benefit and compliance with reduced treatment burden, which is expected to be completed in the third quarter of 2020. The registration study in pediatric GHD patients in Japan is on track for completion in the first quarter of 2020.

RAYALDEE total prescriptions reported by IQVIA increased 89% in 4Q 2019 compared with 4Q 2018: Total prescriptions for the three months ended December 31, 2019 increased to more than 17,700, compared with approximately 9,400 during the comparable period of 2018.

RAYALDEE Phase 2 trial: Interim readout is expected in 1Q 2020 of the open-label Phase 2 trial with RAYALDEE in adults with vitamin D insufficiency and stage 5 chronic kidney disease (CKD) requiring dialysis.

4Kscore test receives final Local Coverage Determination for Medicare payments: On November 15, 2019, OPKO announced that Novitas Solutions issued its final Local Coverage Determination for Medicare payments for the 4Kscore test with defined coverage criteria, effective

December 30, 2019. 4Kscore utilization remained strong with nearly 18,000 tests performed during the fourth quarter of 2019. A premarket approval submission for the 4Kscore test was accepted for review by the U.S. Food and Drug Administration (FDA) in December 2019.

Financial Highlights

Consolidated revenues for the fourth quarter of 2019 increased to $224.3 million compared with $221.9 million for the comparable period of 2018. Revenue from services in the fourth quarter of 2019 was $177.9 million, revenue from products was $32.0 million including RAYALDEE net revenue of $12.6 million, and revenue from licensing and intellectual property was $14.4 million.

Operating expenses for the fourth quarter of 2019 were $336.8 million compared with $311.9 million during the fourth quarter of 2018. Operating expenses during the three months ended December 31, 2019 included a non-cash impairment charge of $91.8 million for goodwill and intangible assets related to the acquisitions of Claros, CURNA and Transition Therapeutics, while the 2018 period included a $21.8 million non-cash impairment charge. Selling, general and administrative expenses decreased during the 2019 period by $16 million to $79.1 million, while research and development expenses decreased to $23.0 million from $33.3 million for the 2018 period, primarily due to the completion of the pediatric Phase 3 study for OPKO’s long-acting human growth hormone product and other ongoing clinical trials.

The net loss for the fourth quarter of 2019, which includes a non-cash impairment charge of $91.8 million, was $112.4 million, or $0.18 per share, compared with a net loss of $76.1 million, or $0.13 per share, for the comparable period of 2018. The 2018 reported net loss benefited from inclusion of an income tax benefit of $28.3 million whereas the 2019 period included an income tax expense of $3.4 million.

Cash, cash equivalents and marketable securities were $85.5 million as of December 31, 2019. In the fourth quarter of 2019, the Company raised gross proceeds of $81.3 million from an underwritten public offering of common stock. On February 25, 2020, the Company entered into an unsecured credit facility providing $100 million of incremental capital on a non-dilutive basis. The credit facility is with Phillip Frost, MD, the Company’s Chairman and CEO.

CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss results in greater detail in a conference call and live audio webcast at 4:30 p.m. Eastern time today, Wednesday, February 26, 2020. The conference call dial-in and webcast information is as follows:
DOMESTIC DIAL-IN:

(877) 783-8475
INTERNATIONAL DIAL-IN:

(614) 999-1827
PASSCODE:

9678678
WEBCAST:

OPKO 4Q19 Results Conference Call

For those unable to participate in the live conference call or webcast, a replay will be available beginning approximately two hours after the close of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 9678678. The replay can be accessed for a period of time on OPKO’s website at OPKO 4Q19 Results Conference Call.

G1 Therapeutics Provides Fourth Quarter and Full-Year 2019 Corporate and Financial Update

On February 26, 2020 G1 Therapeutics, Inc. (Nasdaq: GTHX), a clinical-stage oncology company, reported a corporate and financial update for the fourth quarter and full-year ended December 31, 2019 (Press release, G1 Therapeutics, FEB 26, 2020, View Source [SID1234554797]).

"We achieved significant clinical and regulatory milestones across our pipeline in 2019. In 2020, our primary focus is the execution of our U.S. and European regulatory filings for trilaciclib for patients with small cell lung cancer and preparation for its commercial launch in the U.S.," said Mark Velleca, M.D., Ph.D., Chief Executive Officer. "We believe trilaciclib has the potential to improve outcomes for patients receiving chemotherapy across a broad range of tumor types. In 2020, we will initiate a Phase 3 trial in colorectal cancer and evaluate trilaciclib in the I-SPY 2 breast cancer trial."

Fourth Quarter Regulatory, Clinical and Corporate Highlights

Initiated rolling NDA submission for trilaciclib in small cell lung cancer (SCLC) in 4Q19 and expect to complete the filing in 2Q20. Certain portions of the NDA, including preclinical data, were submitted to the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2019. The company plans to complete the filing in the second quarter of 2020.

Trilaciclib included in I-SPY 2 neoadjuvant breast cancer trial based on compelling overall survival (OS) findings in Phase 2 triple-negative breast cancer (TNBC) trial. At the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Congress, the company presented preliminary data from its randomized, open-label Phase 2 trial of trilaciclib in TNBC showing that the addition of trilaciclib to chemotherapy resulted in a significant increase in OS compared to chemotherapy alone (press release here). These findings contributed to trilaciclib being selected for inclusion in the ongoing Phase 2 I-SPY 2 TRIAL. Two new investigational treatment arms of the trial will evaluate trilaciclib in neoadjuvant treatment of locally advanced breast cancer. The study will generate data that will allow the company to evaluate trilaciclib in combination with several broadly-used chemotherapy classes, an anti-PD-1 immunotherapy, and a range of breast cancer subtypes (press release here).

Findings from Phase 1/2a rintodestrant monotherapy trial in patients with ER+, HER2- breast cancer support initiation of combination trials with CDK4/6 inhibitors. The company announced preliminary safety, tolerability and efficacy data on rintodestrant (formerly G1T48), its oral selective estrogen receptor degrader (SERD), at the 2019 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in September (press release here). Based on these findings, G1 plans to

initiate an additional arm of its ongoing Phase 1/2a trial in the second quarter of 2020 to explore the combination regimen of rintodestrant and the CDK4/6 inhibitor Ibrance (palbociclib) as a treatment for ER+, HER2- breast cancer. Palbociclib will be provided by Pfizer Inc. under a non-exclusive clinical supply agreement.

Reported additional data from Phase 1b/2a clinical trial of lerociclib in combination with fulvestrant for the treatment of ER+, HER2- breast cancer. Updated findings presented at the 2019 San Antonio Breast Cancer Symposium showed lerociclib, dosed without a drug holiday, has a differentiated safety and tolerability profile than observed in clinical trials with currently marketed CDK4/6 inhibitors. Preliminary efficacy findings were consistent with other CDK4/6 inhibitors used in combination with fulvestrant. Additional safety and efficacy data are expected in the third quarter of 2020.

Fourth Quarter/Full-Year 2019 Financial Highlights and 2020 Guidance

Cash Position: Cash and cash equivalents totaled $269.2 million as of December 31, 2019, compared to $369.3 million as of December 31, 2018.

Operating Expenses: Operating expenses were $36.6 million for the fourth quarter of 2019, compared to $26.1 million for the fourth quarter of 2018. GAAP operating expenses include stock-based compensation expense of $4.5 million for the fourth quarter of 2019, compared to $3.3 million for the fourth quarter of 2018. Operating expenses for the full-year 2019 were $129.0 million, compared to $89.3 million for the prior year. Stock-based compensation expense for the full-year 2019 was $16.4 million, compared to $10.2 million for the prior year.

Research and Development Expenses: Research and development (R&D) expenses for the fourth quarter of 2019 were $24.5 million, compared to $19.1 million for the fourth quarter of 2018. R&D expenses for the full-year 2019 were $89.0 million, compared to $70.7 million for the prior year. The increase in R&D expenses was primarily due to an increase in clinical program costs, costs for manufacturing pharmaceutical active ingredients, and personnel costs due to additional headcount.

General and Administrative Expenses: General and administrative (G&A) expenses for the fourth quarter of 2019 were $12.1 million, compared to $7.0 million for the fourth quarter of 2018. G&A expenses for the full-year 2019 were $40.0 million, compared to $18.6 million for the prior year. The increase in G&A expenses was largely due to an increase in compensation due to additional headcount, increase in pre-commercialization activities, increase in medical affairs costs, and an increase in professional fees and other administrative costs necessary to support our operations.

Net Loss: G1 reported a net loss of $35.4 million for the fourth quarter of 2019, compared to $24.1 million for the fourth quarter of 2018. Net loss for the full-year 2019 was $122.4 million, compared to a net loss of $85.3 million for the prior year.

2020 Guidance: The company expects to end 2020 with $110-$130 million in cash and cash equivalents, prior to the consideration of potential proceeds from partnerships, collaboration activities, and/or other sources of capital. The company expects year-end 2019 cash and cash equivalents of $269.2 million to be sufficient to fund its operating expenses and capital expenditure requirements into the second half of 2021.

Key Anticipated 2020 Milestones

Complete NDA submission for trilaciclib in SCLC in 2Q20 and Marketing Authorization Application to the European Medicines Agency in 4Q20.

Begin enrollment in I-SPY 2 clinical trial of trilaciclib in neoadjuvant breast cancer in 2Q20.

Initiate additional arm of rintodestrant Phase 1/2a trial to evaluate combination with Ibrance (palbociclib) in 2Q20; additional Phase 1/2a monotherapy data expected in 4Q20.

Initiate Phase 3 clinical trial of trilaciclib in colorectal cancer in 4Q20.

Webcast and Conference Call

The management team will host a webcast and conference call at 4:30 p.m. ET today to provide a corporate and financial update for the fourth quarter and full-year 2019 ended December 31, 2019. The live call may be accessed by dialing 866-763-6020 (domestic) or 210-874-7713 (international) and entering the conference code: 4188787. A live and archived webcast will be available on the Events & Presentations page of the company’s website: www.g1therapeutics.com. The webcast will be archived on the same page for 90 days following the event.

Halozyme Therapeutics To Present At The Cowen 40th Annual Health Care Conference

On February 26, 2020 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported that it will be presenting at the Cowen and Company 40th Annual Health Care Conference on Wednesday, March 4 at 11:20 a.m. ET / 8:20 a.m. PT in Boston (Press release, Halozyme, FEB 26, 2020, View Source [SID1234554796]). Dr. Helen Torley, president and chief executive officer, will provide a corporate overview.

The presentation will be webcast through the "Investors" section of Halozyme’s corporate website at www.halozyme.com, and a recording will be made available for 90 days following the event. To access a live webcast, please visit Halozyme’s website approximately 15 minutes prior to the presentation to register and download any necessary audio software.

Supernus to Present at the 2020 Cowen Health Care Conference

On February 26, 2020 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported that the Company’s management will present an overview and update, as well as host investor meetings, at the 40th Annual Cowen Health Care Conference (Press release, Supernus, FEB 26, 2020, View Source [SID1234554795]).

Date: Monday, March 2, 2020
Time: 4:10 p.m. ET
Place: Boston Marriott Copley Place, Boston, Mass.

Investors interested in arranging a meeting with the Company’s management during this conference should contact the conference coordinator.

A live webcast of the presentation can be accessed by visiting ‘Events & Presentations’ in the Investor Relations section on the Company’s website at www.supernus.com. An archived replay of this webcast will be available for 60 days on the Company’s website after the conference.