TG Therapeutics Announces Publication of Final Results from the Phase 3 GENUINE Trial Evaluating Ublituximab Plus Ibrutinib in Patients with Relapsed/Refractory High-Risk Chronic Lymphocytic Leukemia in The Lancet Haematology

On February 23, 2021 TG Therapeutics, Inc. (NASDAQ: TGTX) reported the publication of final results from the Phase 3 GENUINE trial evaluating ublituximab, the Company’s investigational glycoengineered anti-CD20 monoclonal antibody, in combination with ibrutinib, in patients with relapsed or refractory high-risk chronic lymphocytic leukemia (CLL), in The Lancet Haematology (Press release, TG Therapeutics, FEB 23, 2021, View Source [SID1234575477]).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer stated, "The Phase 3 data published yesterday, and previously presented, demonstrated that the addition of ublituximab to ibrutinib significantly improved overall response rate, complete response rate as well as prolonged progression-free survival. Significant unmet need still exists within the CLL landscape, and patients with high-risk relapsed or refractory CLL progress more rapidly than those without high-risk cytogenetics. The outcome of the GENUINE study is therefore very encouraging, and we believe these data are supportive of pursuing combination strategies with ublituximab for high-risk CLL patients." Mr. Weiss continued, "We look forward to bringing ublituximab to market as soon as possible as we pursue completion of a BLA submission with the FDA in the first half of 2021 for the combination of ublituximab plus umbralisib for patients with CLL."

Jeff P. Sharman, MD, Director of Research at Willamette Valley Cancer Institute and Medical Director of Hematology Research for The US Oncology Network and Study Chair for the GENUINE trial stated, "The utility of adding anti-CD20 therapy in combination with BTK inhibitors, such as ibrutinib, has long been unclear with prior studies using rituximab having failed to demonstrate an improvement in long-term outcomes. These results published from the GENUINE study are encouraging and may suggest that next generation anti-CD20 antibodies could have value in combination approaches to treating CLL."

The manuscript includes data from 126 patients with relapsed or refractory high-risk CLL who were randomized on study, of which 117 received at least one dose of treatment and were included in safety analyses, with 59 receiving ublituximab plus ibrutinib and 58 receiving ibrutinib monotherapy. Ibrutinib was given orally daily at 420 mg for all cycles. Ublituximab was given intravenously in 28-day cycles with up to 150 mg on day 1, 750 mg on day 2, and 900 mg on days 8 and 15 of cycle 1, and continuing at 900 mg on day 1 of cycles 2 through 6. Beyond cycle 6, ublituximab was given at 900 mg every 3 months. Ublituximab and ibrutinib were continued until unacceptable toxicity, disease progression, or withdrawn consent. The primary endpoint was independent review committee (IRC) assessed overall response rate (ORR) per iwCLL 2008 criteria. Key highlights from this manuscript include:

The IRC-assessed ORR among treated patients was 90% (53 of 59) in the ublituximab-ibrutinib arm and 69% (40 of 58) in the ibrutinib arm (p=0.0060), with a CR/CRi rate of 20% (12 of 59) and 5% (3 of 58), respectively (p=0.024).
After a median follow-up of 41.6 months, median IRC-assessed progression-free survival (PFS) in all treated patients was not reached in the ublituximab-ibrutinib group (95% CI, not estimable [NE]) after 15 PFS events and 35.9 months (95% CI, 17·0-NE) in the ibrutinib group after 25 PFS events (hazard ratio [HR], 0.46; 95% CI, 0.24-0.87).
The most common grade 3/4 adverse events in the ublituximab-ibrutinib group and the ibrutinib group were neutropenia (19%; 12%), anaemia (8%; 9%), and diarrhea (10%; 5%).
These data are described further in the manuscript entitled, "A Phase 3, Randomized Trial of Ublituximab Plus Ibrutinib for Patients With Relapsed/Refractory High-Risk Chronic Lymphocytic Leukaemia," which was published [online yesterday] in The Lancet Haematology. The online version of the article can be accessed at https://www.thelancet.com/journals/lanhae/home.

Phio Pharmaceuticals to Participate in the H.C. Wainwright Global Life Sciences Conference on March 9th-10th

On February 23, 2021 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported that its Chief Executive Officer, Dr. Gerrit Dispersyn, will present at the upcoming H.C. Wainwright Global Life Sciences Conference, which is being held virtually from March 9-10, 2021 (Press release, Phio Pharmaceuticals, FEB 23, 2021, View Source [SID1234575476]).

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During the conference, a corporate presentation by Dr. Dispersyn will be made available on-demand via the H.C. Wainwright conference portal starting at 7 a.m. Eastern Time on March 9, 2021. Interested parties may also access the webcast through the "Investors – Events and Presentations" section of the Company’s website. The webcast will be archived and available on the Company’s website for 90 days.

BioLineRx Reports Year-End 2020 Financial Results and Provides Corporate Update

On February 23, 2021 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, reported its financial results for the year ended December 31, 2020 and provides a corporate update (Press release, BioLineRx, FEB 23, 2021, View Source [SID1234575475]).

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Significant events and achievements during the fourth quarter 2020 and subsequent period:

Announced positive results from an interim analysis of its GENESIS Phase 3 trial of motixafortide in stem-cell mobilization (SCM). The interim analysis found statistically significant evidence for the primary endpoint favoring treatment with motixafortide. Based on a recommendation from the independent Data Monitoring Committee (DMC), enrollment was ceased early at 122 patients (instead of 177 originally planned), and top-line data, including full primary and secondary efficacy endpoints, is anticipated in early second quarter of 2021. In parallel, the Company is proceeding with all activities in support of an NDA submission in this indication anticipated in the first half of 2022, including a pre-NDA meeting with the FDA planned for the second half of 2021.
Reported positive final results from the triple combination arm of the Company’s COMBAT/KEYNOTE-202 study evaluating motixafortide in combination with KEYTRUDA (pembrolizumab) and chemotherapy in patients with second-line stage IV pancreatic ductal adenocarcinoma (PDAC). The results of the study showed substantial improvement as compared to historical results across all study endpoints. The Company is currently planning next development steps for this program, including discussions with potential collaboration partners and development of a protocol for a randomized controlled study.
Announced initiation of a Phase 2 investigator-initiated clinical trial evaluating motixafortide in combination with LIBTAYO and chemotherapy in first-line metastatic PDAC. The study is led by Columbia University.
Announced initiation of a Phase 1b investigator-initiated clinical trial evaluating motixafortide in patients suffering from acute respiratory distress syndrome (ARDS) secondary to COVID-19 and other respiratory viral infections.
Completed underwritten public offering with gross proceeds of $34.5 million.
"The fourth quarter 2020 was perhaps our most significant so far, having achieved positive data milestones in two programs with significant unmet medical needs – stem-cell mobilization and PDAC," stated Philip Serlin, Chief Executive Officer of BioLineRx. "The Phase 3 SCM interim data that we reported in October were overwhelmingly positive, and based on the DMC’s recommendation, we ceased enrollment at 122 out of the originally planned 177 patients. We now look forward to presenting full top-line results from the study, including data related to 100 days of post-transplantation follow-up, by early second quarter of this year. SCM remains our most expeditious path to registration, and we therefore view these data as potentially transformational for our company. In parallel, we are moving forward very aggressively with all activities in support of an NDA submission, which we expect in the first half of next year.

"We are equally excited about the final results from our Phase 2a COMBAT/KEYNOTE-202 PDAC study that we announced in December last year. The data demonstrated that the triple combination of motixafortide, KEYTRUDA and chemotherapy outperformed historical data across all endpoints, including median overall survival, median progression free survival, confirmed and overall response rates and disease control rate. In a cancer population as difficult to treat as second-line metastatic PDAC, and even more specifically those patients initially diagnosed with unresectable stage IV disease, we view these results as highly encouraging and are planning our next development steps forward in this program, likely in collaboration with a biopharmaceutical partner.

"Finally, subsequent to the end of the year, we strengthened our balance sheet through a financing that resulted in gross proceeds of $34.5 million. These funds will allow us to continue to execute on our strategy for motixafortide in both SCM and PDAC, while in parallel advancing our second clinical candidate, the anti-cancer immunotherapy AGI-134, through clinical development. In summary, we exited 2020 on a very positive note, with two data sets that demonstrate both the effectiveness and versatility of motixafortide across multiple indications, and we plan to build upon these successes this year," concluded Mr. Serlin.

Upcoming Significant Expected Milestones

Top-line results from the Phase 3 GENESIS trial in SCM in early Q2 2021.
Initial results from Part 2 of the Phase 1/2a trial of AGI-134 in solid tumors in the second half of 2021.
Pre-NDA meeting with the FDA for SCM in the second half of 2021
NDA submission for SCM in the first half of 2022
Financial Results for the Year Ended December 31, 2020

Research and development expenses for the year ended December 31, 2020 were $18.2 million, a decrease of $5.2 million, or 22.5%, compared to $23.4 million for the year ended December 31, 2019. The decrease resulted primarily from termination of the BATTLE clinical study for motixafortide in 2019, from lower expenses associated with the motixafortide COMBAT clinical trial and from lower expenses associated with the AGI-134 study, as well as a decrease in share-based compensation and payroll due to a company-wide salary reduction related to the COVID-19 pandemic.

Sales and marketing expenses for the year ended December 31, 2020 were $0.8 million, similar to sales and marketing expenses for the year ended December 31, 2019.

General and administrative expenses for the year ended December 31, 2020 were $3.9 million, an increase of $0.1 million, or 2.6% compared to $3.8 million for the year ended December 31, 2019. The increase resulted primarily from an increase in D&O insurance expenses and share-based compensation, offset by small decreases in a number of G&A expenses.

The Company’s operating loss for the year ended December 31, 2020 amounted to $22.9 million, compared to an operating loss of $28.1 million for the year ended December 31, 2019.

Non-operating expenses amounted to $5.7 million for the year ended December 31, 2020, compared to non-operating income of $4.2 million for the year ended December 31, 2019. Non-operating expenses for the year ended December 31, 2020 primarily relate to fair-value adjustments of warrant liabilities on the Company’s balance sheet, warrant offering expenses and ATM issuance expenses. Non-operating income for the year ended December 31, 2019 primarily relates to fair-value adjustments of warrant liabilities on the Company’s balance sheet, offset by warrant offering expenses.

Net financial expenses amounted to $1.4 million for the year ended December 31, 2020, compared to net financial expenses of $1.5 million for the year ended December 31, 2019. Net financial expenses for both periods primarily relate to interest paid on loans, offset by investment income earned on bank deposits.

The Company’s net loss for the year ended December 31, 2020 amounted to $30.0 million, compared with a net loss of $25.5 million for the year ended December 31, 2019.

The Company held $22.6 million in cash, cash equivalents and short-term bank deposits as of December 31, 2020. Subsequent to year end, the Company raised gross proceeds of $34.5 million in an underwritten public offering, and received another $9.8 million in gross proceeds from the exercise of outstanding warrants.

Net cash used in operating activities for the year ended December 31, 2020 was $23.2 million, compared to $22.7 million for the year ended December 31, 2019. The $0.5 million increase in 2020 was primarily the result of a decrease in accounts payable and accruals.

Net cash provided by investing activities for the year ended December 31, 2020 was $16.7 million, compared to $5.3 million for the year ended December 31, 2019. The changes in cash flows from investing activities relate primarily to investments in, and maturities of, short-term bank deposits.

Net cash provided by financing activities for the year ended December 31, 2020 was $17.9 million, compared to $19.2 million for the year ended December 31, 2019. The cash flows in 2020 primarily reflect the registered direct offerings of the Company’s ADSs in May and June 2020, as well as net proceeds from the ATM program, offset by repayments of the loan from Kreos Capital. The cash flows in 2019 primarily reflect the underwritten public offering of the Company’s ADSs in February 2019, as well as net proceeds from the ATM program.

Conference Call and Webcast Information

BioLineRx will hold a conference call today, Thursday, February 23, 2021 at 10:00 a.m. EST. To access the conference call, please dial +1-866-744-5399 from the US or +972-3-918-0610 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until February 25, 2021; please dial +1-888-782-4291 from the US or +972-3-925-5904 internationally.

Agios to Present at March Investor Conferences

On February 23, 2021 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, reported that the company is scheduled to present at the following March investor conferences (Press release, Agios Pharmaceuticals, FEB 23, 2021, View Source [SID1234575474]):

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Cowen 41st Annual Virtual Healthcare Conference on Monday, March 1, 2021 at 11:40 a.m. ET; and
Oppenheimer 31st Annual Virtual Healthcare Conference on Wednesday, March 17, 2021 at 9:20 a.m. ET.
Live webcasts of the presentations can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. Replays of the webcasts will be archived on the Agios website for at least two weeks following each presentation.

Spectrum Pharmaceuticals Announces Two Presentations at Upcoming ESMO TAT Virtual Congress 2021

On February 23, 2021 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported two presentations, one oral and one eposter, at the upcoming European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Virtual Congress 2021 (Press release, Spectrum Pharmaceuticals, FEB 23, 2021, View Source [SID1234575473]). The oral presentation will provide some initial safety and efficacy data for poziotinib from the twice daily dosing cohort. The meeting takes place on March 1 – 2, 2021. Details of the presentations are as follows:

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Title: IGN002 (antiCD20-IFNα2b) intravenously administered tumor targeted delivery of IFNα2b and its effects in non-Hodgkin lymphoma
Speaker: Sribalaji Lakshmikanthan, Ph.D.
Session: Immunotherapy (ID 361)
Date and Time: March 1, 2021, 9:41 a.m. CET / 3:41 a.m. ET
Presentation Number: #20P (poster)

Title: Safety, tolerability and preliminary efficacy of poziotinib with twice daily strategy in EGFR/HER2 Exon 20 mutant non-small cell lung cancer
Speaker: Adrian Sacher, M.D.
Session: Mini Oral Session (ID 17) Channel 1
Date and Time: March 2, 2021, 17:25 CET / 11:25 a.m. ET
Presentation Number: 36MO (mini-oral)

Access to the presentations is available to members of ESMO (Free ESMO Whitepaper) and can be found here: View Source;module=attendee#signin