On February 28, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX), a biopharmaceutical company developing medicines for patients with B-cell mediated diseases, reported that the Marginal Zone Lymphoma (MZL) cohort of the UNITY-NHL Phase 2b pivotal trial evaluating umbralisib (TGR-1202), our novel, once daily, PI3K delta inhibitor, met the primary endpoint of Overall Response Rate (ORR) as determined by Independent Review Committee (IRC) for all treated patients (n=69) (Press release, Manhattan Pharmaceuticals, FEB 28, 2019, View Source [SID1234533795]). The results met the Company’s target guidance of 40-50% ORR.

Interim safety and efficacy data from this study will be presented in an oral presentation at the upcoming American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting on April 1, 2019 and full data from this study are expected to be presented at a medical meeting later this year. The company plans to discuss the results with the U.S. Food and Drug Administration (FDA) regarding a potential new drug application (NDA) filing for accelerated approval. Umbralisib was recently granted Breakthrough Therapy Designation (BTD) by the FDA for the treatment of adult patients with MZL who have received at least one prior anti-CD20 regimen.

Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics stated, "We are extremely pleased to announce that the UNITY-NHL marginal zone lymphoma cohort evaluating umbralisib monotherapy met the primary endpoint of ORR. While this was an early look at the response data, we were excited to have already met the target ORR, which we previously stated was approximately 40%-50%. Importantly, with many patients still on study, we anticipate the ORR will continue to improve with additional follow-up, which will also provide us with critical information on duration of response, progression free survival and long-term safety and tolerability necessary to support an NDA filing."

Mr. Weiss continued, "There are no fully approved drugs for MZL, and thus remains an unmet medical need and we are excited by the potential to offer a novel treatment for this underserved population. We look forward to discussing the results with the FDA as soon as possible and if all goes well, we believe we could be in a position to file for accelerated approval for umbralisib by year-end."

About the UNITY-NHL Phase 2b Study—Marginal Zone Lymphoma Cohort

The multicenter, open-label, UNITY-NHL Phase 2b study – Marginal Zone Lymphoma cohort was designed to evaluate the safety and efficacy of single agent umbralisib, the Company’s novel, once daily, PI3K delta inhibitor, in patients with MZL who have received at least one prior anti-CD20 regimen. The primary endpoint is overall response rate (ORR) as determined by Independent Review Committee (IRC) assessment. The primary analysis of ORR will be conducted once all treated patients have had at least 9 cycles (Cycle = 28 days) of follow-up. Secondary endpoints include safety, duration of response, and progression-free survival (PFS).

The MZL cohort completed enrollment in August 2018 with a total of 69 patients enrolled and receiving at least one dose of umbralisib. The positive ORR outcome announced today was based on all 69 enrolled and treated patients, however at this time all patients have not yet been followed for a minimum of 9 cycles as required for the primary analysis of ORR. Accordingly, the study is on-going and patients with benefit on therapy (stable disease or in response) remain on study. Safety data are currently being analyzed.

2019 AACR (Free AACR Whitepaper) Oral Presentation Details

Abstract titles are now available online and can be accessed on the AACR (Free AACR Whitepaper) meeting website at www.aacr.org. The complete text of the abstract will be available on the meeting website on Friday, March 29, 2019. The presentation is expected to include a subset of patients from the UNITY-NHL-Marginal Zone Cohort with long-term follow-up as of the data cut-off. Additional details regarding the presentation are included below.

● Title: Umbralisib monotherapy demonstrates efficacy and safety in patients with relapsed/refractory marginal zone lymphoma: A multicenter, open-label, registration directed Phase II study
o Session Date and Time: Monday April 1, 2019 3:00 PM – 5:00 PM ET
o Session Title: The Next Generation of Clinical Trials in Molecularly-driven Therapy
o Session Location: Marcus Auditorium- Bldg A-GWCC
o Presenter: Nathan Fowler, MD, Associate Professor, Department of Lymphoma/Myeloma, The University of Texas MD Anderson Cancer Center, Houston, TX
o Abstract Number: 7821

Following the presentation, the data presented will be available on the Publications page, located within the Pipeline section, of the Company’s website at www.tgtherapeutics.com/publications.cfm.

CONFERENCE CALL INFORMATION

The Company will host a conference call today, Thursday, February 28, 2019 at 8:30 AM ET to discuss the UNITY-NHL MZL results.

In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Update Call.

A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com. An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

ABOUT MARGINAL ZONE LYMPHOMA

Marginal zone lymphoma (MZL) comprises a group of indolent (slow growing) B-cell non-Hodgkin lymphomas (NHLs) that begin forming in the marginal zone of lymphoid tissue. With an annual incidence of approximately 7,500 newly diagnosed patients, MZL is the third most common B-cell NHL accounting for approximately eight percent of all NHL cases.i MZL consists of three different subtypes: extranodal MZL of the mucosal-associated lymphoid tissue (MALT), nodal marginal zone lymphoma (NMZL), and splenic marginal zone lymphoma (SMZL).

ABOUT BREAKTHROUGH THERAPY DESIGNATION

The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of a drug candidate that is planned to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies.

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On February 28, 2019 UroGen Pharma Ltd. (Nasdaq:URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in uro-oncology, reported financial results for the fourth quarter and full year ended December 31, 2018 and provided an overview of the Company’s recent developments (Press release, UroGen Pharma, FEB 28, 2019, View Source;p=RssLanding&cat=news&id=2389450 [SID1234533766]).

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"Our clinical and corporate execution in 2018 places UroGen in a position of strength as we prepare to potentially deliver UGN-101 as the first approved therapy for patients with low-grade upper tract urothelial cancer (LG UTUC)," said Liz Barrett, President and Chief Executive Officer of UroGen. "With this solid foundation, we remain intensely focused on finalizing our rolling New Drug Application (NDA) and accelerating commercial preparation to ensure a successful launch upon approval with seamless adoption of UGN-101. We believe UGN-101 is just the beginning of what may be possible with our proprietary RTGel technology platform."

"LG UTUC represents a clinical challenge as the current treatment of surgical intervention can put patients at risk for the well-known complications associated with repetitive surgical procedures and potential kidney removal," said Mark P. Schoenberg, M.D., Chief Medical Officer of UroGen. "If approved, UGN-101 could be a true breakthrough for patients by providing an option to avoid the risks and downstream consequences associated with surgery while arming urologists in general practice with a simple-to-use, kidney sparing approach to the management of their patient’s disease."

Recent Highlights

UGN-101 Clinical Development:

In the fourth quarter, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for UGN-101 (mitomycin gel) for instillation, an investigational mitomycin formulation for the non-surgical treatment of patients with LG UTUC.

In January 2019, the Company announced positive topline results from the pivotal Phase 3 OLYMPUS clinical trial of UGN-101, in which 57 percent of patients achieved a complete response (CR) rate at their primary disease evaluation (PDE, or the primary endpoint) which was conducted four to six weeks after completion of UGN-101 treatment.

Pipeline Advancement:

The Company continues to enroll patients as part of its Phase 2b OPTIMA II clinical trial of UGN-102 (mitomycin gel) for intravesical instillation as a potential first-line chemoablation agent in the treatment of intermediate risk patients with low-grade non-muscle invasive bladder cancer (LG NMIBC) at risk for recurrence.

Corporate Developments:

UroGen strengthened its leadership team with the appointment of Liz Barrett as President and Chief Executive Officer. Ms. Barrett has over 30 years of industry experience spanning multiple areas including oncology, specialty care, surgical franchises, and consumer marketing in multiple geographic regions, most recently serving as CEO of Novartis Oncology and as a member of the Executive Committee of Novartis.

The Company enhanced its financial position with the completion of a successful public offering of ordinary shares in January 2019, resulting in net proceeds of approximately $162 million. These proceeds leave the company well-capitalized to execute on upcoming milestones, including preparation for potential commercialization of UGN-101, continued clinical development of our second product candidate (UGN-102), and advancement of our pipeline.

Upcoming Milestones:

UGN-101: UroGen remains on track to complete its rolling NDA for UGN-101 in 2H 2019 and is planning for potential approval in 1H 2020. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC.

The company continues to build out its commercial infrastructure and focus on scientific awareness to support adoption and seamless integration of UGN-101 into the urologist practice following potential regulatory approval.

UGN-102: The Company intends to report initial data from the OPTIMA II trial of UGN-102 in 2019.

Similar to UGN-101, UGN-102 has the potential to transform the treatment paradigm for patients with LG NMIBC, as there are currently no drugs approved by the FDA as first-line treatment for LG NMIBC.

UGN-102 represents a substantial opportunity in UroGen’s pipeline with the potential to initially address up to approximately 80,000 patients for whom repetitive surgical resection via Transurethral Resection of Bladder Tumor (TURBT) remains the standard of care.

BotuGel: Allergan continues to enroll patients in its Phase 2 trial of BotuGel, UroGen’s RTGel in combination with BOTOX1, for the treatment of overactive bladder. Phase 2 data is expected in 2019.

Fourth Quarter and Full Year 2018 Financial Results; 2019 Guidance

As of December 31, 2018, cash, cash equivalents, and short-term investments totaled $101.3 million. In addition, the Company raised net proceeds of approximately $162 million from an underwritten public offering in January 2019. Based on anticipated activities, the current cash balance is projected to carry the company for the next 24-36 months.

Research and development expenses for the year ended December 31, 2018 were $36.9 million, including non-cash share-based compensation expense of $12.0 million. Research and development expenses for the three months ended December 31, 2018 were $11.5 million, including non-cash share-based compensation expense of $3.0 million.

General and administrative expenses for the year ended December 31, 2018 were $39.6 million, including non-cash share-based compensation expense of $18.6 million. General and administrative expenses for the three months ended December 31, 2018 were $12.6 million, including non-cash share-based compensation expense of $5.9 million.

The Company reported a net loss of $75.7 million, or basic and diluted net loss per ordinary share of $4.80, for the year ended December 31, 2018. The Company reported a net loss of $23.7

BOTOX is a proprietary trademark of Allergan Pharmaceuticals

million, or basic and diluted net loss per ordinary share of $1.46, for the three months ended December 31, 2018.

The Company anticipates a net loss in the range of $100 to $115 million for 2019, which is expected to include non-cash stock-based compensation expense in the range of $24 to $27 million subject to market conditions. The projected change in net loss for 2019 versus 2018 is expected to be largely attributable to the buildout of commercial infrastructure in anticipation of the potential launch of UGN-101. The Company has 20.4 million ordinary shares outstanding post the closing of our most recent financing in January.

Conference Call & Webcast Information

Members of UroGen’s management team will host a live conference call and webcast today at 8:30 am Eastern Time to review the Company’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call (888) 771-4371 (U.S.) or (847) 585-4405 (International) to listen to the live conference call. The conference ID number for the live call will be 48205742. An archive of the webcast will be available for two weeks on the Company’s website.

On February 28, 2019 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported that the company will present at the Barclays Global Healthcare Conference in Miami Beach, Florida on Tuesday, March 12, 2019 at 8:00 a.m. ET (Press release, BioCryst Pharmaceuticals, FEB 28, 2019, View Source [SID1234533790]).

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Links to a live audio webcast and replay of the presentation may be accessed in the Investors section of BioCryst’s website at http://www.biocryst.com.

On February 28, 2019 Genocea Biosciences, Inc. (NASDAQ: GNCA), a biopharmaceutical company developing neoantigen cancer immunotherapies, reported its operating and financial results for the fourth quarter and fiscal year ended December 31, 2018 (Press release, Genocea Biosciences, FEB 28, 2019, View Source [SID1234533787]). The company also announced the appointment of a new Chief Financial Officer, Diantha Duvall, a seasoned life science industry professional. Ms. Duvall will join the company on March 5th.

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"We are very pleased to welcome Diantha to Genocea," said Chip Clark, president & CEO of Genocea. "She joins us at a truly exciting time. With our recently completed equity financing and advancing pipeline, we look forward to an eventful 2019. We foresee important near-term milestones in two programs, each driven by our unique ATLAS neoantigen identification platform: first, our Phase 1/2a clinical program for our neoantigen cancer vaccine candidate, GEN-009, from which we expect to see the first immunogenicity data in mid-2019, and second, our GEN-011 program, a neoantigen adoptive T cell therapy, for which we plan to file an IND in the first half of 2020."

Ms. Duvall joins Genocea from Bioverativ, Inc., where she served as Vice President, Controller and Chief Accounting Officer since the company’s spin out from Biogen, where she had served as U.S. and then Global Commercial Controller. Prior to Biogen, Ms. Duvall held a variety of financial and accounting positions at Merck and Co. and PricewaterhouseCoopers.

Recent Operational Highlights

Strengthened senior management team, adding Thomas Davis, M.D., as Chief Medical Officer and Derek Meisner, J.D., as General Counsel in the Fall of 2018, Girish Aakalu, Ph.D. as Chief Business Officer in December 2018, and, as announced today, Diantha Duvall as CFO.
Completed a private placement financing in mid-February, the first closing of which resulted in aggregate gross proceeds of $15.0 million (before deducting fees to the placement agents and other expenses).
Announced a major clinical milestone in early January, dosing the first patients and completing enrollment in Part A of its Phase 1/2a clinical trial for GEN-009. The first immunogenicity data from this study are expected in mid-2019.
Presented additional ATLAS data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting in November 2018. The posters further highlighted the advantages of Genocea’s ATLAS platform over in silico methods in identifying both neoantigens for vaccine inclusion and "inhibitory" neoantigens for exclusion, and presented, for the first time, data in a mouse model providing evidence that these neoantigen-specific inhibitory responses could be tumor-promoting.
Fourth-Quarter 2018 Financial Results

Cash position: As of December 31, 2018, cash and cash equivalents were $26.4 million compared to $12.3 million, as of December 31, 2017; this does not include approximately $15.0 million in aggregate gross proceeds (before deducting fees to the placement agents and other expenses) from the company’s recently closed private placement.
Research and Development (R&D) expenses: R&D expenses were $6.3 million for the quarter ended December 31, 2018, compared to $7.9 million for the same period in 2017.
General and Administrative (G&A) expenses: G&A expenses were $2.6 million for the quarter ended December 31, 2018, compared to $2.5 million for the same period in 2017.
Net income/loss: Net income was $0.4 million for the quarter ended December 31, 2018, compared to a net loss of $1.7 million for quarter ended December 31, 2017.
Full-Year 2018 Financial Results

Research and Development (R&D) expenses: R&D expenses were $25.2 million for the year ended December 31, 2018, compared to $39.2 million for the year ended December 31, 2017.
General and Administrative (G&A) expenses: G&A expenses were $14.3 million for the year ended December 31, 2018, compared to $13.4 million for the year ended December 31, 2017.
Net loss: Net loss was $27.8 million for the year ended December 31, 2018, compared to a net loss of $56.7 million for year ended December 31, 2017.
Guidance
Genocea expects that its existing cash and cash equivalents are sufficient to support its operations into the first quarter of 2020.

Genocea continues to explore strategic alternatives for GEN-003, its Phase 3-ready investigational immunotherapy for the treatment of genital herpes.

Genocea’s Board of Directors has adopted a resolution approving and recommending that stockholders support a proposal in the company’s upcoming proxy statement to effect a reverse stock split of the company’s issued and outstanding common stock, if necessary, to maintain the company’s NASDAQ Capital Market listing.

Conference Call
Genocea will host a conference call and webcast today at 9:00 a.m. ET. The conference call may be accessed by dialing (844) 826-0619 (domestic) or (315) 625-6883 (international) and referring to conference ID number 6953999. A live webcast of the conference call will be available online from the investor relations section of the Company’s website at View Source A webcast replay of the conference call will be available on the Genocea website beginning approximately two hours after the event and will be archived for 90 days.

On February 28, 2019 Incyte Corporation (Nasdaq:INCY) reported that seven abstracts showcasing data from its cancer research portfolio will be presented at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 (Press release, Incyte, FEB 28, 2019, View Source [SID1234533786]). The meeting will be held March 29 – April 3, 2019, at the Georgia World Congress Center in Atlanta, Georgia.

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Accepted abstracts feature data from clinical studies involving Incyte’s anti-PD-1 monoclonal antibody, INCMGA00121, in patients with advanced solid tumors, as well as pre-clinical characterizations of the Company’s oral, small molecule PD-L1 inhibitor program and its PD-L1xCD137 bispecific antibody.

"Data accepted for presentation during the upcoming AACR (Free AACR Whitepaper) Annual Meeting demonstrate our ongoing commitment to advancing innovative research as we seek to bring forward novel therapies for patients with cancer," stated Dashyant Dhanak, Ph.D., Chief Scientific Officer, Incyte. "In particular, we are pleased to present, for the first time at a major medical meeting, early data on our oral PD-L1 inhibitor program—whose lead candidate, INCB86550, recently entered clinical trials."

Key abstracts include:

Clinical

Pharmacodynamic correlates in a phase 1 study of INCMGA00012, a PD-1 antagonistic monoclonal antibody (Abstract #CT085/9, Phase 1 Clinical Trials: Part 3)

Monday, April 1, 2019, 1:00 p.m. – 5:00 p.m. ET, Poster Section 16
Assessment of flat dosing strategy for INCMGA00012 in patients with advanced tumors(Abstract #LB-268/14, Late-Breaking Research: Experimental and Molecular Therapeutics)

Wednesday, April 3, 2019, 8:00 a.m. – 12:00 p.m. ET, Poster Section 40
Pre-Clinical

Oral Presentations

Novel small-molecule antagonists of the PD-1/PD-L1 axis that mediate cell surface PD-L1 dimerization and internalization (Abstract #4483, Novel Therapeutics)

Tuesday, April 2, 2019, 3:00 p.m. – 5:00 p.m. ET, Georgia Ballroom 3 – Georgia World Congress Center (Building C)
Preclinical characterization of potent and selective oral PD-L1 small-molecule antagonists (Abstract #4480, Novel Therapeutics)

Tuesday, April 2, 2019, 3:00 p.m. – 5:00 p.m. ET, Georgia Ballroom 3 – Georgia World Congress Center (Building C)
Poster Presentations

A bispecific Fc-silenced IgG1 antibody (MCLA-1452) requires PD-L1 binding to activate CD137 (Abstract #539/3, Therapeutic Antibodies I)

Sunday, March 31, 2019, 1:00 p.m. – 5:00 p.m. ET, Poster Section 23
An unbiased screen identifies a CD137xPD-L1 bispecific IgG1 antibody with unique T cell activation and binding properties (Abstract #541/5, Therapeutic Antibodies I)

Sunday, March 31, 2019, 1:00 p.m. – 5:00 p.m. ET, Poster Section 23
Characterization of human cancer cell line xenografts in humanized mice (Abstract #1050/1, Human in Mouse)

Monday, April 1, 2019, 8:00 a.m. – 12:00 p.m. ET, Poster Section 4
Full session details and data presentation listings for AACR (Free AACR Whitepaper) 2019 can be found at: View Source!/6812.