On May 13, 2019 Forty Seven Inc. (NASDAQ:FTSV), a clinical-stage, immuno-oncology company focused on developing therapies to activate macrophages in the fight against cancer, reported financial results for the first quarter ended March 31, 2019 and provided a business Update (Press release, Forty Seven, MAY 13, 2019, View Source [SID1234536217]).

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"Our achievements year-to-date reflect our commitment to maximizing the potential of 5F9 as a novel, first-in-class therapeutic for the treatment of cancer," said Mark McCamish, M.D., Ph.D., President and Chief Executive Officer of Forty Seven, Inc. "Building on the promising results observed in our Phase 1b NHL trial, we recently entered into additional clinical collaborations to evaluate 5F9 as part of two triplet regimens for patients with the most aggressive forms of the disease. With these partnerships, we continue to cost-efficiently explore the full therapeutic potential of 5F9 across multiple treatment modalities. Looking ahead, we are eager to report updated data from our Phase 1b/2 trial of 5F9 plus rituximab in r/r NHL at the EHA (Free EHA Whitepaper) and ICML meetings, as well as new clinical data from our Phase 1b trial of 5F9 in combination with azacitidine in AML and MDS at the ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) medical meetings. These data will provide key insights as we chart our long-term development plan for 5F9.

First Quarter and Recent Business Highlights:

Pipeline:

In May 2019, Forty Seven entered into a collaboration with Acerta Pharma, AstraZeneca’s hematology research and development center of excellence, to evaluate 5F9 in combination with rituximab plus AstraZeneca/Acerta Pharma’s Bruton’s tyrosine kinase (BTK) inhibitor, acalabrutinib (CALQUENCE), in patients with diffuse large B-cell lymphoma (DLBCL). This approach aims to optimize the treatment of DLBCL patients by inhibiting BTK. In B-cells, BTK signaling results in the activation of pathways associated with B-cell proliferation, trafficking, chemotaxis and adhesion, and BTK inhibition is a proven strategy for inducing the regression of B-cell lymphomas.

In April 2019, Forty Seven extended its existing collaboration with Genentech, a member of the Roche Group, to include a third clinical trial evaluating 5F9 in combination with rituximab plus Genentech’s anti-PD-L1 antibody, atezolizumab (TECENTRIQ), in patients with DLBCL. This approach aims to optimize the treatment of DLBCL patients whose tumors are associated with high levels of macrophages expressing PD-L1.

Upcoming Presentations:

Forty Seven plans to present updated data from the Phase 1b/2 trial of 5F9 in combination with rituximab in patients with relapsed or refractory non-Hodgkin’s lymphoma (r/r NHL), including safety, efficacy and duration of response across various dosing cohorts, at the 24th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) and the International Conference on Malignant Lymphoma (ICML) meetings in June 2019.

Forty Seven plans to present safety and initial efficacy data from the Phase 1b trial evaluating 5F9 as a monotherapy and in combination with azacitidine in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the 24th Congress of the EHA (Free EHA Whitepaper) in June 2019.

Key Milestones:

Additionally, the company expects to achieve the following milestones by the end of 2019:

Report data from the Phase 1b trial of 5F9 in combination with avelumab in patients with ovarian cancer in the fourth quarter.

Report data from the Phase 1b trial of 5F9 in combination with cetuximab in patients with colorectal cancer in the fourth quarter.

Report preclinical data and complete investigational new drug (IND)-enabling studies for FSI-174, an anti-cKIT antibody with potential use as a novel conditioning regimen for bone marrow transplantation, in the second half.

Complete IND-enabling studies for FSI-189, an anti-SIRPα antibody with therapeutic potential for cancer and other indications, in the second half.

First Quarter 2019 Financial Results:

Cash Position: As of March 31, 2019, cash, cash equivalents and short-term investments were $113.6 million, as compared to $139.0 million as of December 31, 2018. This decrease reflects cash used to fund operating activities, including approximately $7.7 million in an advance payment for contract manufacturing, in the first quarter of 2019. The company expects that its cash, cash equivalents and short-term investments will fund operating expenses and capital expenditure requirements through the first half of 2020.

R&D Expenses: R&D expenses were $19.1 million for the first quarter of 2019, as compared to $11.2 million for the first quarter of 2018. The increase was primarily due to a $3.7 million increase in third-party costs related to advancing the company’s clinical development programs for 5F9 and associated contract manufacturing costs Additionally, there was an increase of $2.8 million in costs associated with the advancement of the company’s preclinical and discovery programs, and an increase of $1.4 million in personnel-related costs, including stock-based compensation.

G&A Expenses: G&A expenses were $4.6 million for the first quarter of 2019, as compared to $3.8 million for the first quarter of 2018. The increase was primarily due to a $0.9 million increase in personnel-related costs driven by an increase in headcount and a $0.2 million increase in directors and officers insurance expense, partially offset by a $0.4 million decrease in accounting and consulting expenses incurred in connection with operating as a public company.

Net Loss: Net loss was $23.0 million for the first quarter of 2019, or a net loss per share of $0.74, as compared to $14.8 million for the first quarter of 2018, or a net loss per share of $2.24.

Conference Call Information:

Forty Seven will host a live conference call and webcast at 4:30 p.m. ET today to discuss first quarter 2019 financial results and recent business activities. The conference call may be accessed by (866) 953-0780 (domestic) or (630) 652-5854 (international), and by referring to conference ID 4283836. A webcast of the conference call will be available in the Investors section of the Forty Seven website at View Source The archived webcast will be available on Forty Seven’s website approximately two hours after the conference call and will be available for 30 days following the call.

On May 13, 2019 Alexion Pharmaceuticals (Nasdaq: ALXN) reported that management will present at the RBC Capital Markets Annual Healthcare Conference in New York, NY on Tuesday, May 21, 2019 at 9:30 a.m. ET (Press release, Alexion, MAY 13, 2019, View Source [SID1234536218]).

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An audio webcast of the presentation will be available live. You can access the webcast at: View Source An archived version of the remarks will also be available through the Company’s website for a limited time following the conference.

On May 13, 2019 F-star, a clinical-stage biopharmaceutical company delivering tetravalent bispecific antibodies for a paradigm-shift in cancer therapy, reported the reconfiguration of its immuno-oncology collaboration, established in 2017, with Merck KGaA, Darmstadt, Germany as it executes on its transition to a wholly-owned portfolio and builds scale and value as a world-class biopharmaceutical company (Press release, f-star, MAY 13, 2019, View Source [SID1234536235]).

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Under the terms of the new agreement, F-star retains exclusive rights to develop and commercialise FS118, a clinical-stage tetravalent bispecific antibody. At the same time, Merck has exercised its option for one discovery stage programme and retains the right to option a second discovery programme from the 2017 original agreement. No financial terms of the agreement are being disclosed.

Eliot Forster, CEO of F-star, said: "This new agreement reflects our pivot to building a wholly-owned pipeline, that allows for rapid progress into the clinic and secures greater long-term value from our products. With full rights to FS118, we have an opportunity to accelerate the development of this first-in-class medicine for a group of targeted cancer patients. We are also pleased to continue our long-term collaboration with Merck KGaA, Darmstadt, Germany by advancing assets from F-star’s Modular Antibody TechnologyTM into their pipeline."

FS118 is a potential first-in-class tetravalent bispecific antibody for the treatment of cancer, developed to overcome tumour evasion mechanisms promoted by two molecules (LAG-3: Lymphocyte-Activation Gene 3 and PD-L1: Programmed Death-Ligand 1) with the potential to restore the natural ability of the immune system to fight cancer(1). Initiated in April 2018 under F-star’s sponsorship, the Phase I trial (NCT03440437) continues as originally planned and is expected to read out during 2020.

(1) LAG-3/PD-L1 mAb² can overcome PD-L1-mediated compensatory upregulation of LAG-3 induced by single-agent checkpoint blockade. Faroudi et al. (March 2019) – Poster at the annual AACR (Free AACR Whitepaper) meeting

On May 10, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the first quarter ended March 31, 2019 and recent company developments (Press release, TG Therapeutics, MAY 10, 2019, View Source [SID1234536163]).

Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "2019 has been an extremely productive year thus far with the achievement of many key milestones, the most important of which has been the significant progress made with our Marginal Zone Lymphoma program, including the release of positive top-line results and the receipt of breakthrough therapy designation and orphan drug designation. Moving forward, we believe further clarity on our potential MZL NDA filing by year-end along with the presentation of final MZL data later this year has the potential to unlock significant value for our shareholders." Mr. Weiss continued, "Between now and the middle of next year, we expect to have pivotal data readouts across three trials, including umbralisib in non-Hodgkin’s lymphoma, umbralisib plus ublituximab (U2) in chronic lymphocytic leukemia and ublituximab in multiple sclerosis. This is an extremely exciting time for us and we look forward to an impactful remainder of 2019 and 2020."

Recent Developments and Highlights

●Marginal Zone Lymphoma – Breakthrough Therapy and Orphan Drug Designations: Received breakthrough therapy designation (BTD) and orphan drug designation for umbralisib (TGR-1202), for the treatment of patients with all three types of marginal zone lymphoma (MZL): nodal, extranodal and splenic MZL.
●Positive Results from MZL Cohort of UNITY-NHL Trial: Announced positive outcome from the MZL cohort of the UNITY-NHL trial, which met the primary endpoint of Overall Response Rate (ORR). Interim safety and efficacy data from this study were presented in an oral presentation at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in Atlanta.
●DSMB Updates: Meetings held by the independent Data Safety Monitoring Boards (DSMBs) for both the UNITY-CLL trial and the UNITY-NHL trial did not raise any safety concerns and recommended that both trials continue unmodified.
●TG-1801 (Anti-CD47/CD19): Commenced a Phase 1 first-in-human, dose-escalation study of TG-1801 in patients with relapsed or refractory B-cell lymphoma.
●Multiple Sclerosis Data: Presented long-term safety data from the open label extension (OLE) of the Phase 2 trial of ublituximab in multiple sclerosis, which demonstrated that ublituximab continues to be well tolerated.

Remaining 2019 Milestones

●Potential top-line progression free survival (PFS) results from the Phase 3 UNITY-CLL trial evaluating U2 in patients with CLL
●Present final data from the MZL cohort of the UNITY-NHL registration directed trial evaluating umbralisib in MZL
●Potential UNITY-NHL NDA filing in MZL
●Present updated data from our pipeline products and combination studies at upcoming major medical conferences

Financial Results for the First Quarter 2019

●Cash Position: Cash, cash equivalents and investment securities were $92.5 million as of March 31, 2019. Pro-forma cash, cash equivalents and investment securities as of March 31, 2019 (excluding our second quarter 2019 operations) are approximately $116.7 million, after giving effect to $24.2 million of net proceeds from the utilization of the Company’s at-the-market ("ATM") sales facility during the second quarter of 2019.

●R&D Expenses: Other research and development (R&D) expense (not including non-cash compensation) was $30.9 million for the three months ended March 31, 2019 compared to $32.2 million for the three months ended March 31, 2018. Included in other research and development expense for the three months ended March 31, 2019 was $13.8 million of clinical trial expense and $6.4 million of manufacturing and CMC expenses for Phase 3 clinical trials and potential commercialization. The current period decrease in Other R&D expenses is primarily due to full enrollment in our pivotal Phase III clinical development programs completed in the prior period. We expect the decrease in other R&D expenses to continue over the next several quarters.

●G&A Expenses: Other general and administrative (G&A) expense (not including non-cash compensation) was $1.9 million for the three months ended March 31, 2019 as compared to $2.1 million for the three months ended March 31, 2018. Other G&A expenses for the three months ended March 31, 2019 remained relatively flat compared to the first quarter of 2018, and we expect Other G&A expenses to increase modestly through the remainder of 2019.

●Net Loss: Net loss was $35.2 million for the three months ended March 31, 2019, compared to a net loss of $41.5 million for the three months ended March 31, 2018. Excluding non-cash items, the net loss for the three months ended March 31, 2019 was approximately $33.3 million.

●Financial Guidance: Net cash utilized for operating activities during the three months ended March 31, 2019 was approximately $33.5 million. The Company believes its cash, cash equivalents and investment securities on hand as of March 31, 2019, inclusive of the proceeds raised subsequent to the first quarter, will be sufficient to fund the Company’s planned operations through mid-2020.

Conference Call Information

The Company will host a conference call today, May 10, 2019, at 8:00 am ET, to discuss the Company’s first quarter 2019 financial results and provide a business outlook for the remainder of 2019.

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 First Quarter 2019 Business Update Call. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source 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.

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On May 10, 2019 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported first quarter 2019 financial results and business highlights, which will be discussed on a conference call scheduled for Tuesday, May 14 at 10:00 a.m. ET (Press release, Abeona Therapeutics, MAY 10, 2019, View Source [SID1234536157]). Interested parties are invited to participate in the call by dialing 844-369-8770 (toll-free domestic) or 862-298-0840 (International) or via webcast at View Source

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"We’ve had a great start to 2019, with continued progress across our pipeline, including the completion of CMC work in advance of initiating our Phase 3 VITAL clinical trial evaluating EB-101, our gene-corrected cell therapy for patients with recessive dystrophic epidermolysis bullosa," said João Siffert, M.D., Chief Executive Officer. "The study, which will utilize clinical material produced at our Cleveland manufacturing facility, is on track to start mid-2019 following completion of ongoing FDA review."

"We have continued to advance our lysosomal storage disease programs. Our increased efforts around patient recruitment are starting to bear fruit as we observe an uptick in patient screening for both MPS programs. In addition, we have submitted the IND for our CLN1 program." added Dr. Siffert.

First Quarter Financial Results:

Cash, cash equivalents and marketable securities as of March 31, 2019 were $68.3 million, compared to $85.0 million as of December 31, 2018. The decrease in cash of $16.7 million was driven primarily by the net cash used for operating activities of $15.1 million.

Research and development expenses for the first quarter ended March 31, 2019 were $11.7 million, compared to $8.2 million for first quarter 2018. The increase in research and development expense was primarily attributable to increased R&D headcount, related facility costs and internal manufacturing costs.

General and administrative expenses for the first quarter ended March 31, 2019 were $5.7 million, compared to $2.9 million for the first quarter 2018. The increase in general and administrative expenses was primarily attributable to increased headcount and related facility costs.

Net loss was $0.39 per share for the first quarter of 2019, compared to $0.22 per share in the same period of 2018.

First Quarter and Recent Highlights:

·May 8, 2019: Abeona Therapeutics Announces Presentation of EB-101 Data at Society for Investigative Dermatology Annual Meeting
oFollow up data showed that three years after treatment with investigational product, EB-101, a majority of RDEB patients had durable wound healing and improvements in pain and itching. There were no serious treatment-related adverse events three years post-treatment and no replication competent virus present at any time point.
·May 1, 2019: Reported Preclinical Data Demonstrating Broad Therapeutic Potential of AIM Gene Therapy in Retinal Diseases at Association for Research in Vision and Ophthalmology Annual Meeting
oIntravitreal administration of the Company’s novel AIM AAV204 capsid in non-human primates led to robust transgene expression in the inner and outer retina. These preclinical data support the potential use of intravitreal administration to deliver gene therapy in an out-patient setting for a wide range of inherited and acquired retinal diseases.
·April 30, 2019: Reported New Preclinical Data Demonstrating Therapeutic Potential of ABO-401 for Treatment of Cystic Fibrosis at American Society of Gene and Cell Therapy Annual Meeting
oABO-401, the Company’s novel gene therapy for cystic fibrosis (CF) efficiently delivered a highly-expressed, functional copy of human mini-CFTR (hCFTR) to the lung of CF mice and restored CFTR function in human CF patient nasal and bronchial epithelial cells.
·April 4, 2019: Received FDA Fast Track Designation for ABO-101 for Treatment of Sanfilippo Syndrome Type B (MPS IIIB)
·February 11, 2019: Appointed João Siffert, M.D. Chief Executive Officer
·January 31, 2019: Presented New Supportive Data for Novel Gene Therapies at WORLDSymposium
oPlatform and poster presentations highlighted study results on biodistribution and tissue tropism of the next-generation AIM AAV vector platform in Pompe and Fabry diseases, as well as data from programs in MPS IIIA and CLN3 disease.
·January 8, 2019: Strengthened Financial Leadership with New Executive Appointments: Christine Silverstein as Chief Financial Officer and Edward Carr as Chief Accounting Officer

"We expect 2019 to be a transformative year for Abeona, as we prepare to execute on a number of milestones in the clinic, in our mission to develop gene and cell therapies that could transform the treatment of serious diseases," said Steven H. Rouhandeh, Chairman of the Board and Executive Chairman. "We are encouraged to continue advancing the proprietary technology of our AIM vector platform, especially following recent promising data presented across multiple indications, for which we continue to evaluate avenues of development internally, and with potential partners."