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]).

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"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.

On February 26, 2020 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, announced today that the Company has scheduled the release of its fourth quarter and full year 2019 financial results before market opens on Friday, February 28, 2020 (Press release, Sangamo Therapeutics, FEB 26, 2020, View Source [SID1234554814]). The press release will be followed by a conference call at 8:00 a.m. ET, which will be open to the public via telephone and webcast. During the conference call, the Company will review its financial results and provide a business update.

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The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 4609858. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 4609858.

On February 26, 2020 Oramed Pharmaceuticals Inc. (NASDAQ: ORMP), (TASE: ORMP), a clinical-stage pharmaceutical company focused on the development of oral drug delivery systems, reported that it intends to offer and sell shares of its common stock in an underwritten public offering (Press release, Oramed Pharmaceuticals, FEB 26, 2020, View Source [SID1234554830]). All of the shares in the underwritten public offering are to be sold by Oramed. Oramed intends to grant the underwriters a 45-day option to purchase up to an additional 15% of the shares of common stock offered in the public offering at the public offering price, less the underwriting discount.

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The Company intends to use the net proceeds of the offering for its anticipated Phase 3 clinical trial in ORMD-0801 (Oral Insulin) and for other clinical trials and research and development activities as well as for general corporate purposes.

National Securities Corporation, a wholly owned subsidiary of National Holdings Corporation, is acting as the sole book running manager for the offering.

A.G.P./Alliance Global Partners is acting as financial advisor for the proposed offering.

The shares of common stock are being offered by Oramed pursuant to its shelf registration statement on Form S-3 (File No. 333-236194) previously filed and declared effective by the Securities and Exchange Commission (the "SEC") on February 10, 2020. The offering may be made only by means of a prospectus supplement and accompanying prospectus. Copies of the preliminary prospectus supplement and accompanying prospectus, when available, may be obtained from National Securities Corporation, Attn: Charles Wanyama, 200 Vesey Street, 25th Floor, New York, New York 10281, telephone: (212) 417-3634, or by email at [email protected]; or on the SEC’s website at View Source

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On February 26, 2020 Genprex, a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and other serious diseases, reported that is harnessing the power of a combinational treatment approach that utilizes its leading gene therapy drug candidate, Oncoprex, in combination with approved targeted and immunotherapies in order to overcome treatment limitations and offer patients new treatment options (Press release, Genprex, FEB 26, 2020, View Source [SID1234554850]). In January 2020, the U.S Food and Drug Administration granted Fast Track Designation for Genprex’s lead drug candidate, Oncoprex immunogene therapy for non-small cell lung cancer (NSCLC) in combination therapy with osimertinib (AstraZeneca’s Tagrisso).

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Targeted Therapies
Targeted therapies work by targeting the cancer or disease’s specific gene, protein or tissue that contributes to the disease1 while sparing normal tissue. This therapy is different from chemotherapies, which usually destroy all dividing cells, irrespectively.

Unfortunately, targeted therapies require patients to have an activating genetic mutation specific to the drug. In NSCLC, the vast majority of lung cancer patients do not have the genetic mutations found in lung cancer that qualify them for targeted therapies, such as the EGFR or ALK gene. One study found that only about 10 percent of NSCLC patients had the EGFR mutation2. In addition, the few patients who do have the right genetic mutation to receive some of the available targeted therapies are likely to develop resistance to these drugs over time3.

To overcome the limitations of targeted therapies, many companies and researchers are studying the combination of approved targeted therapies with potential new drug candidates. The hope of these combinations is to improve outcomes for patients. In fact, Genprex has studied its lead drug candidate, Oncoprex, in combination with erlotinib, an approved targeted therapy for NSCLC.

Preclinical and clinical data show that Oncoprex works synergistically with erlotinib to help destroy the cancer cells and may help patients who have become resistant to erlotinib alone.

The company’s recent Fast Track Designation by the FDA is for the combination of Oncoprex and osimertinib (marketed as Tagrisso by Astra Zeneca), a third generation EGFR inhibitor. The company received this designation based on its preclinical data of Oncoprex combined with osimertinib and its clinical trial data for Oncoprex combined with erlotinib. Tagrisso is AstraZeneca’s highest grossing product, which had worldwide sales in 2018 of $1.86 billion and $3.18 billion in 2019.

Immunotherapies
Immunotherapy is a type of treatment that helps the immune system fight disease4. Biomarker testing can help determine if high levels of PD-1/PD-L1 proteins are detected in the patient, signifying that the body’s immune system is not working to fight cancer or disease as it should. These patients could qualify for approved immunotherapies, however, only about 12 percent of patients benefit from immunotherapies5.

Similar to targeted therapies, immunotherapy combinations are being studied to see how these limitations can be overcome. Genprex is conducting preclinical studies to evaluate Oncoprex in combination with checkpoint inhibitors.

Preclinical data shows that Oncoprex is synergistic with anti-PD1 therapy and could result in a stronger antitumor response compared to either agent alone.

Additionally, the combination of Oncoprex increased the effectiveness of anti-PD1 checkpoint blockade combined with chemotherapy in a humanized mouse model with lung metastases.

Genprex believes its lead drug candidate in combination with checkpoint inhibitor pembrolizumab may have similar synergistic results for patients.

The Power of Immunogene Therapy Combinations
Genprex is working to provide new treatment options to a larger group of patients who would not otherwise be candidates for targeted therapies or immunotherapies or who have built up resistance to currently approved drugs.

The company’s lead drug candidate, Oncoprex immunogene therapy, is unlike the approved targeted therapies and immunotherapies available today. Because Oncoprex is a gene therapy that also modulates immune activity around the cancer cell, it is poised and positioned to help NSCLC patients who cannot benefit from today’s treatment options. It may also be able to be combined with a variety of approved drugs to improve outcomes for patients in a variety of cancers.

To learn more about the company’s lead drug candidate, please visit the Technology page on Genprex’s website.

*Oncoprex is currently in development and is not FDA approved.

References:

1. Understanding Targeted Therapy. Cancer.Net. View Source Published December 5, 2019. Accessed February 20, 2020.

2. El-Telbany A, Ma PC. Cancer genes in lung cancer: racial disparities: are there any?. Genes Cancer. 2012;3(7-8):467–480. doi:10.1177/1947601912465177.

3. Meyers DE, Bryan PM, Banerji S, Morris DG. Targeting the PD-1/PD-L1 axis for the treatment of non-small-cell lung cancer. Curr Oncol. 2018;25(4):e324–e334. doi:10.3747/co.25.3976

4. Immunotherapy for Cancer. National Cancer Institute. View Source Accessed February 20, 2020.

5. Haslam A, Prasad V. Estimation of the Percentage of US Patients With Cancer Who Are Eligible for and Respond to Checkpoint Inhibitor Immunotherapy Drugs. JAMA Netw Open. 2019;2(5):e192535. doi:10.1001/jamanetworkopen.2019.2535.

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]).

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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.