On February 25, 2019 Exicure, Inc. (OTCQB: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) constructs, reported that it has dosed the first patient in its multicenter, open-label, Phase 1b/2 study of AST-008 combined with pembrolizumab (Press release, Exicure, FEB 25, 2019, View Source;p=RssLanding&cat=news&id=2388720 [SID1234533672]). Enrollment in the trial is open to patients with superficial injectable tumors in advanced or metastatic solid tumor conditions including Merkel cell carcinoma, head and neck squamous cell carcinoma, cutaneous squamous cell carcinoma and melanoma. clinicaltrials.gov NCT03684785

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"We believe that combining our immune system agonist drug with checkpoint inhibitors is an important strategy for leveraging the patient’s own immune system to fight cancer. We are excited to bring this approach into cancers like Merkel cell carcinoma, where patients have limited success using currently available treatments," said Exicure CEO Dr. David Giljohann. "It is also an important milestone for Exicure in the development of our platform technology, which allows us to digitally design drug candidates and potentially bring them into clinic faster."

The primary objective of the Phase 1b dose escalation stage is to assess the safety and tolerability of Exicure’s AST-008 drug alone and in combination with pembrolizumab, and to determine a dose for the Phase 2 stage of the study. Patients in the dose escalation stage may have previously been exposed to antibody checkpoint inhibitors, but not as a requirement for inclusion in the trial. In the Phase 2 portion of the study, Exicure will further evaluate AST-008 in combination with pembrolizumab in patients who have previously received but not responded to anti-PD-1 or anti-PD-L1 antibody therapy.

About Exicure’s AST-008 Drug

AST-008 is a toll-like receptor nine (TLR9) agonist oligonucleotide in a proprietary SNA format with immune-stimulatory properties. SNAs are dense, radial arrangements of nucleic acids (DNA) that have high cellular uptake and an enhanced presentation of the DNA for TLR9 agonism. AST-008 is designed to enter into and activate immune cells to elicit an immune response to treat solid tumors in combination with other agents such as checkpoint inhibitors. We observed that AST-008 showed potent antitumor activity as a monotherapy and synergized with anti-PD-1 antibodies in multiple preclinical tumor models. In a successful Phase 1 trial in healthy volunteers, AST-008 activated key immune cells and cytokines predictive for an anti-tumor effect in patients.

On February 25, 2019 Gamida Cell Ltd. (Nasdaq:GMDA), a leading cellular and immune therapeutics company, reported financial results for the fourth quarter and full year ended December 31, 2018, and provided a business update, which highlights the company’s progress advancing its clinical development candidates: NiCord, an investigational advanced cell therapy in Phase 3 clinical development designed to enhance and expand the life-saving benefits of hematopoietic stem cell (bone marrow) transplant, and NAM-NK, an investigational, cell-based cancer immunotherapy in Phase 1 development in patients with non-Hodgkin lymphoma and multiple myeloma (Press release, Gamida Cell, FEB 25, 2019, View Source [SID1234533637]).

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Gamida Cell continues to anticipate completing patient enrollment in the Phase 3 study of NiCord by the end of this year with topline data anticipated in first half of 2020. The international, randomized, multi-center study is designed to evaluate the safety and efficacy of NiCord compared to standard umbilical cord blood for allogeneic bone marrow transplant in approximately 120 patients with no available matched donor. Additionally, the company continues to make progress in its NAM-NK program, and expects to initiate a multi-center Phase 1/2 study of NAM-NK in patients with blood cancer in 2020.

"We are off to a strong start this year and are making meaningful progress across every dimension of our company. The data presented last week at the TCT Annual Meeting demonstrates the potential for NiCord to offer a new cell therapy option for patients in need of a bone marrow transplant," stated Julian Adams, Ph.D., chief executive officer at Gamida Cell. "Additionally, our collaboration with Be The Match BioTherapies and the appointment of Tom Klima as chief commercial officer underscore our commitment to advancing our clinical development plans and beginning commercial readiness activities for the potential approval and launch of NiCord."

Dr. Adams continued, "We are committed to leveraging the transformative potential of our proprietary nicotinamide-, or NAM-based, cell expansion technology, to deliver a multi-product pipeline. Last week, we presented encouraging data from our ongoing Phase 1 study of NAM-NK in patients with non-Hodgkin lymphoma and multiple myeloma, and the emerging clinical profile suggests it has the potential to address some of the limitations of currently available cell therapies. In addition, we have demonstrated the ability to cryopreserve our NK cells in a laboratory setting, and we are working to scale up our manufacturing process to enable the ability to evaluate multiple doses and schedules of NAM-NK therapy in a multi-center, Phase 1/2 clinical study next year."

Recent Company Highlights:

Reported additional immune reconstitution data for NiCord supporting clinical potential for bone marrow transplant: In February, translational data from the completed Phase 1/2 study of NiCord were reported at the 2019 Transplantation & Cellular Therapy (TCT) Meetings of American Society for Blood and Marrow Transplantation and Center for International Blood and Marrow Transplant Research demonstrating that recipients who received NiCord had rapid and robust reconstitution of key immune cells. Successful immune reconstitution is an important factor in the recovery of patients undergoing bone marrow transplant.
Reported initial data from Phase 1/2 study of NiCord in severe aplastic anemia: At the 2019 TCT Annual meeting, data were reported from the ongoing Phase 1/2 study of NiCord in patients with severe aplastic anemia. In the initial cohort of three patients, all successfully underwent a bone marrow transplant consisting of NiCord plus a haploidentical stem cell graft. The rapid cord engraftment, sustained hematopoiesis and accelerated immune recovery in treatment refractory observed in these patients enable the initiation of a second cohort of patients to be treated with NiCord as a stand-alone graft. Patient enrollment in the second cohort is expected to begin in the first half of 2019.
Announced encouraging data from Phase 1 study of NAM-NK in non-Hodgkin Lymphoma and multiple myeloma: During the 2019 TCT Annual Meeting, data reported from the ongoing Phase 1 study of NAM-NK in patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM) demonstrated that NAM-NK was clinically active, with three complete responses observed in patients with NHL and one complete response in a patient with MM. These data, along with safety data showing that NAM-NK was generally well tolerated, support continued clinical development, and Gamida Cell is planning to initiate a multi-center, Phase 1/2 clinical study of NAM-NK in patients with blood cancers in 2020.
Entered into agreement with Editas Medicine to evaluate potential to gene edit NAM-NK cells: In February, Gamida Cell announced an agreement with Editas Medicine, Inc. to evaluate the potential use of Editas Medicine’s CRISPR technology to edit NAM-NK cells. Through this agreement, the companies aim to rapidly discover optimized NAM-NK cells that could be used to improve the treatment of blood cancers and solid tumors.
Formed strategic collaboration with Be The Match BioTherapies: In January 2019, both organizations announced a collaboration to expand the use of bone marrow transplant to treat hematologic malignancies and serious blood disorders. Under the terms of the collaboration agreement, the organizations will explore opportunities to work together across Gamida Cell’s ongoing clinical development program for NiCord, including the ongoing Phase 3 clinical study. Be The Match BioTherapies has an extensive history of involvement in the delivery of cord blood units for transplant and broad access to cord blood banks globally.
Appointed Thomas Klima as Chief Commercial Officer: In January, the company announced the appointment of Thomas Klima as Chief Commercial Officer. Mr. Klima brings nearly 20 years of global experience in the pharmaceutical industry with expertise in cellular therapy, hematology, oncology and transplantation. During his career, he has played key roles in building commercial organizations and leading multiple successful product launches.
Appointed Nurit Benjamini to Board of Directors: In January, the company appointed Nurit Benjamini to Gamida Cell’s board of directors and chair of the board’s audit committee. Ms. Benjamini has served as chief financial officer of TabTale Ltd. since 2013. Previously, she held a number of chief financial officer positions, including at Wix.com Ltd., Sigma Designs Israel Ltd. and Compugen Ltd.
Anticipated 2019-2020 Milestones
Gamida Cell’s anticipated program milestones in 2019-2020 are as follows:

Nicord

Initiate Cohort 2 in the Phase 1/2 study evaluating NiCord as stand-alone graft in severe aplastic anemia in the first half of 2019
Complete enrollment in Phase 3 study of NiCord in patients with hematologic malignancies in the second half of 2019
Report topline data from the Phase 3 study of NiCord in patients with hematologic malignancies in the first half of 2020
Complete BLA filing for NiCord in hematologic malignancies in the second half of 2020, should Phase 3 data be positive
NAM-NK

Complete patient enrollment in the ongoing Phase 1 study in the second half of 2019
Present additional data at a medical meeting in the second half of 2019
Initiate multi-center, Phase 1/2 clinical study in 2020
Fourth Quarter and Full Year 2018 Financial Results:

At December 31, 2018, Gamida Cell had total cash, cash equivalents and available-for-sale securities of $60.7 million, compared to $41.1 million at December 31, 2017.
Research and Development expenses in 2018 were $22.0 million, compared to $15.0 million in 2017. The increase was attributable mainly to a $5.4 million increase in clinical activities relating to the advancement of Gamida Cell’s clinical programs as well as an increase of $1.6 million in other R&D expenses.
General and administrative expenses were $11.6 million in 2018, compared to $4.5 million in 2017. The increase was attributable mainly to a $2.9 million increase in expenses related to expanding the management team and establishing the U.S. headquarters, an increase of $2.0 million in non-cash stock-based compensation expenses, and $2.2 million in professional services expenses incurred during the IPO process, rent and other expenses.
Finance expenses, net, were $19.2 million in 2018, compared to $0.5 million in income, in 2017. The increase was primarily due to non-cash expenses resulting from revaluation of warrants and the revaluation of royalty-bearing grant IIA liability.
Net loss for 2018 was $52.9 million, compared to a net loss of $19.1 million for 2017.
2019 Financial Guidance
Gamida Cell expects cash used for ongoing operating activities in 2019 to range from $35-$40 million, reflecting anticipated expenditures to advance the company’s clinical programs.

Gamida Cell expects that its cash, cash equivalents and available-for-sale securities will support the company’s capital needs through the data readout for the Phase 3 clinical study of NiCord, which is expected in the first half of 2020. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding that may be received or business development activities that may be undertaken.

About NiCord
NiCord, the company’s lead clinical program, is under development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies. NiCord has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration, making it the first bone marrow transplant alternative to receive this designation. It has also received U.S. and EU orphan drug designation. A Phase 3 clinical study evaluating NiCord in patients with leukemia and lymphoma is ongoing in the United States, Europe and Asia.1 NiCord is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.2 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as NiCord. For more information on clinical trials of NiCord, please visit www.clinicaltrials.gov.

About NAM-NK
Gamida Cell applied the capabilities of its NAM-based cell expansion technology to highly functional NK cells to develop NAM-NK, an innate immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. NAM-NK addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. NAM-NK is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.3

NAM-NK and NiCord are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On February 25, 2019 Medpace Holdings, Inc. (Nasdaq: MEDP) ("Medpace") reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Medpace, FEB 25, 2019, View Source [SID1234533638]).

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Fourth Quarter and Full Year 2018 Financial Results under ASC 606

Revenue for the three and twelve months ended December 31, 2018 was $192.1 million and $704.6 million, respectively. Backlog as of December 31, 2018 was $1.1 billion and net new business awards were $231.2 million, representing a net book-to-bill ratio of 1.20x for the fourth quarter of 2018. For the full year 2018, net new business awards were $899.4 million, representing a net book-to-bill ratio of 1.28x. The Company calculates the net book-to-bill ratio by dividing net new business awards by revenue.

For the fourth quarter of 2018 and full year 2018, total direct costs were $131.1 million and $489.1 million, respectively. Adjusted direct costs were $131.9 million and $492.2 million in the fourth quarter of 2018 and full year 2018, respectively. Selling, general and administrative (SG&A) expenses were $20.6 million and Adjusted SG&A expenses were $20.8 million for the fourth quarter of 2018. For the full year 2018, SG&A expenses were $75.7 million and Adjusted SG&A expenses were $75.7 million.

GAAP net income for the fourth quarter of 2018 was $22.8 million, or $0.61 per diluted share, which resulted in a net income margin of 11.8%. GAAP net income for the full year of 2018 was $73.2 million, or $1.97 per diluted share, which resulted in a net income margin of 10.4%. Adjusted EBITDA for the fourth quarter of 2018 was $39.7 million, or 20.7% of revenue. Adjusted EBITDA for the full year of 2018 was $137.8 million, or 19.6% of revenue. Adjusted Net Income was $28.1 million, and Adjusted Net Income per diluted share was $0.76 for the fourth quarter of 2018. Adjusted Net Income was $95.5 million, and Adjusted Net Income per diluted share was $2.59 for the full year of 2018.

Fourth Quarter and Full Year 2018 Financial Results under ASC 605

Net service revenue for the three months ended December 31, 2018 increased 28.6% to $127.9 million, compared to $99.4 million for the comparable prior-year period. Net service revenue for the year ended December 31, 2018 increased 23.7% to $478.1 million, compared to $386.5 million for the year ended December 31, 2017. On a constant currency basis, net service revenue for the fourth quarter of 2018 increased 29.1% compared to the fourth quarter of 2017 and increased 23.4% for the year ended December 31, 2018 compared to the year ended December 31, 2017.

Backlog as of December 31, 2018 grew 19.4% to $626.1 million from $524.4 million as of December 31, 2017. Net new business awards were $146.7 million, representing a net book-to-bill ratio of 1.15x for the fourth quarter of 2018, as compared to $114.7 million for the comparable prior-year period. For the year ended December 31, 2018, net new business awards were $581.0 million, representing a net book-to-bill ratio of 1.22x, compared to $426.1 million for the year ended December 31, 2017.

For the fourth quarter of 2018, Direct service costs, excluding depreciation and amortization, were $67.9 million, compared to $55.6 million in the fourth quarter of 2017. Adjusted Direct service costs were $68.7 million for the fourth quarter 2018, compared to $56.4 million in the fourth quarter of 2017. For the full year 2018, Direct service costs, excluding depreciation and amortization, were $252.3 million, compared to $211.8 million in the full year 2017. Adjusted Direct service costs were $255.4 million for the full year 2018, compared to $215.0 million in the full year 2017.

SG&A expenses were $20.6 million in the fourth quarter of 2018, compared to $16.8 million in the fourth quarter of 2017. Adjusted SG&A expenses were $20.8 million for the fourth quarter 2018 versus $16.4 million in the fourth quarter of 2017. For the full year 2018, SG&A expenses were $75.7 million, compared to $63.4 million for the full year 2017. Adjusted SG&A expenses were $75.7 million for the full year 2018 versus $63.1 million for the full year 2017.

GAAP net income for the fourth quarter of 2018 was $22.5 million, or $0.60 per diluted share, versus GAAP net income of $11.3 million, or $0.30 per diluted share, for the fourth quarter of 2017. This resulted in a net income margin of 17.6% and 11.4% for the fourth quarter of 2018 and 2017, respectively. GAAP net income for full year 2018 was $81.6 million, or $2.20 per diluted share, versus GAAP net income of $39.1 million, or $0.98 per diluted share, for the full year 2017. This resulted in a net income margin of 17.1% and 10.1% for the full year 2018 and 2017, respectively.

Adjusted EBITDA for the fourth quarter of 2018 increased 43.0% to $38.6 million, or 30.2% of net service revenue, compared to $27.0 million, or 27.2% of net service revenue, for the comparable prior-year period. Adjusted EBITDA for the full year 2018 increased 37.0% to $148.0 million, or 31.0% of net service revenue, compared to $108.0 million, or 28.0% of net service revenue, for the prior year. On a constant currency basis, Adjusted EBITDA for the fourth quarter of 2018 increased 38.0% from the fourth quarter of 2017 and increased 36.2% for the full year 2018 compared to the full year 2017.

Adjusted Net Income for the fourth quarter of 2018 increased 88.2% to $27.8 million, compared to $14.8 million for the comparable prior-year period. Adjusted Net Income per diluted share for the fourth quarter of 2018 was $0.75 compared to Adjusted Net Income per diluted share of $0.39 for the comparable prior-year period. Adjusted Net Income for the full year 2018 increased 71.7% to $103.8 million, compared to $60.5 million for the prior year. Adjusted Net Income per diluted share for the full year 2018 was $2.81 compared to Adjusted Net Income per diluted share of $1.52 for the prior year.

A reconciliation of the Company’s non-GAAP financial measures, including EBITDA, Adjusted EBITDA, Adjusted EBITDA margin, Adjusted Direct costs, Adjusted Selling, general and administrative expenses, Adjusted Net Income, and Adjusted Net Income per diluted share to the corresponding GAAP measures is provided below.

Balance Sheet and Liquidity

The Company’s Cash and cash equivalents were $23.3 million at December 31, 2018, and the Company generated $39.4 million in cash flow from operating activities during the fourth quarter of 2018.

Financial Guidance

For full year 2019, the Company is providing guidance under ASC 606. The Company forecasts 2019 revenue in the range of $783.0 million to $807.0 million, representing growth of 11.1% to 14.5% over 2018 revenue of $704.6 million. GAAP net income for full year 2019 is forecasted in the range of $85.2 million to $89.2 million. Additionally, full year 2019 Adjusted EBITDA is expected in the range of $137.0 million to $145.0 million.

Based on forecasted 2019 revenue of $783.0 million to $807.0 million and GAAP net income of $85.2 million to $89.2 million, diluted earnings per share (GAAP) is forecasted in the range of $2.27 to $2.38. Adjusted Net Income for 2019 is forecasted in the range of $97.0 million to $101.0 million, compared to Adjusted Net Income of $95.5 million for 2018. Furthermore, Adjusted Net Income per diluted share for 2019 is expected in the range of $2.58 to $2.69 per share.

Conference Call Details

Medpace will host a conference call at 9:00 a.m. ET, Tuesday, February 26, 2019, to discuss its fourth quarter and full year 2018 results.

To participate in the conference call, dial 800-219-7113 (domestic) or 574-990-1030 (international) using the passcode 5975717.

To access the conference call via webcast, visit the "Investors" section of Medpace’s website at medpace.com. The webcast replay of the call will be available at the same site approximately one hour after the end of the call.

A supplemental slide presentation will also be available at the "Investors" section of Medpace’s website prior to the start of the call.

A recording of the call will be available at 12:00 p.m. ET on Tuesday, February 26, 2019 until 12:00 p.m. ET on Tuesday, March 12, 2018. To hear this recording, dial 855-859-2056 (domestic) or 404-537-3406 (international) using the passcode 5975717.

On February 25, 2019 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported its financial results for the fourth quarter and twelve months ended December 31, 2018 (Press release, VBI Vaccines, FEB 25, 2019, View Source [SID1234533674]).

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"2018 was a foundational year, one that laid critical groundwork as we build towards the transformational milestones expected in 2019," said Jeff Baxter, President and CEO, VBI Vaccines Inc. "Our achievements in 2018 – which included the launch of the collaboration with Brii Biosciences to develop a functional cure for Hepatitis B for up to $129 million plus royalties, the closing of a $42.9 million public offering led by Perceptive Advisors, the positive Phase 1 data readout from our cytomegalovirus (CMV) vaccine candidate, and encouraging early immunogenicity data from the Phase 1/2a of our glioblastoma (GBM) immuno-therapeutic – set the stage for 2019. Heading into this year, VBI is well-positioned to achieve meaningful clinical milestones across all of our lead programs, most notably the top-line data readout from the PROTECT Phase 3 study of Sci-B-Vac expected in four months’ time."

Sci-B-Vac Program Update (Prophylactic Hepatitis B):

In December 2017, the Company initiated enrollment in two pivotal Phase 3 clinical studies – PROTECT and CONSTANT – in a total of approximately 4,500 adults. The studies were designed to assess safety and efficacy of Sci-B-Vac, VBI’s prophylactic hepatitis B (HBV) vaccine that is the only commercially-available trivalent HBV vaccines containing Pre-S1, Pre-S2, and S antigens and adjuvanted with alum.

"Hepatitis B remains a serious public health unmet need, one where enhanced protection through vaccination is vital for long-term control," said Francisco Diaz-Mitoma, M.D., Ph.D., VBI’s Chief Medical Officer. "In the last two decades, Sci-B-Vac has been tested in 22 different clinical trials and has been safely and effectively administered to over 500,000 infants and adults in the commercial setting. We believe the extensive safety and efficacy data we have to-date has largely de-risked the ongoing Phase 3 studies. We look forward to the data readouts later this year, as we work to provide a rapid, potent, and safe hepatitis B vaccine to address this significant unmet infectious disease need."

Upcoming Sci-B-Vac Clinical Data Read-outs:

●Mid-year 2019: PROTECT Phase 3 top-line data

PROTECT is a head-to-head immunogenicity study in approximately 1,600 subjects comparing Sci-B-Vac to Engerix-B.
Primary endpoints include non-inferiority of Sci-B-Vac in adults over age 18, and superiority of Sci-B-Vac in adults over age 45.
Secondary endpoints include non-inferiority of Sci-B-Vac after two vaccinations compared with three vaccinations of Engerix-B, and safety.

●Around year-end 2019: CONSTANT Phase 3 top-line data

CONSTANT is a lot-to-lot consistency study in approximately 2,850 subjects comparing immune responses across three independent, consecutively manufactured lots of Sci-B-Vac.
Secondary endpoints include safety, and efficacy compared with Engerix-B

Other Pipeline Program Updates:

GBM Immuno-therapeutic:

VBI-1901, an immuno-therapy developed using VBI’s proprietary enveloped virus-like particle (eVLP) technology platform, is being assessed in an ongoing phase 1/2a clinical study in recurrent GBM patients. In 2018, VBI completed enrollment in all three dose cohorts in the Part A dose-escalation phase of the study, with six patients enrolled in each cohort. In February 2019, the independent Data and Safety Monitoring Board (DSMB) reviewed all safety data from Part A and unanimously recommended the continuation of the study without modification.

"While Part A of the study was primarily designed to assess safety and tolerability of VBI-1901 to support the identification of the optimal therapeutic dose level for Part B of the study, we are encouraged by the early immunogenicity data we’ve observed to-date," said David Anderson, Ph.D., VBI’s Chief Scientific Officer. "Part B of the study will have narrower enrollment criteria and is primarily designed to assess efficacy signals of VBI-1901 in recurrent GBM patients. With a more homogenous patient cohort in Part B, we look forward to identifying potential initial correlations between immunologic responses and clinical outcomes by year-end 2019."

Upon selection of the optimal dose level in Part A, based on safety and immunogenicity data, the Company expects to initiate enrollment of an additional 10 patients in the subsequent Part B extension phase of the study. Expanded immunologic data and 6-month survival data from all dose cohorts in the Part A dose-escalation phase are expected later in the first half of 2019.

Hepatitis B Immuno-therapeutic:

In December 2018, VBI announced a license and collaboration agreement with Brii Biosciences, for up to $129 million plus royalties, for the development of a functional cure for the treatment of chronic hepatitis B infection using VBI-2601. VBI-2601 is a novel immuno-therapeutic candidate that is uniquely formulated to target B- and T-cell immunity by neutralizing circulation of the hepatitis B virus, blocking hepatitis B infection of hepatocytes through Pre-S1 immunity, and enabling immune-mediated clearance of HBV-infected hepatocytes.

As part of this collaboration, clinical proof of concept studies are expected to be initiated by the end of 2019.

CMV Prophylactic Vaccine:

In May 2018, VBI announced positive top-line data from the Phase 1 study of VBI-1501, the Company’s prophylactic vaccine candidate for CMV. The Phase 1 top-line data showed that VBI-1501 was safe and well-tolerated at all doses and was immunogenic, even at the lower doses tested. In December 2018, VBI announced plans for a formal Phase 2 dose-ranging study to assess safety and immunogenicity of VBI-1501 at higher doses. The highest dose level is expected to be 10-times higher than that tested in the Phase 1 study.

The Company expects to initiate enrollment in the Phase 2 study by the end of 2019, following the requisite toxicology studies.

Financial Results for the Three and Twelve Months Ended December 2018:

○VBI ended the fourth quarter of 2018 with $59.3 million cash and cash equivalents compared with $67.7 million as of December 31, 2017. Net cash used in operating activities for the full year 2018 was $45.5 million, compared to $31.4 million for the same period in 2017. Additionally, the purchase of property and equipment in 2018 was $6.0 million compared with $0.6 million in 2017. This increase was due to the modernization and capacity increase of the Rehovot site, where all clinical and commercial supplies of Sci-B-Vac are manufactured, to enable the supply of commercial quantities of Sci-B-Vac upon marketing authorization approval by the FDA, EMA, and/or Health Canada. As part of this modernization and capacity increase, the site was temporarily shut down as of April 22, 2018. The construction related to the modernization and capacity increase is ongoing, and validation activities are in progress.

○Revenue for the fourth quarter of 2018 was $2.7 million, and was primarily attributable to amounts recognized as part the therapeutic Hepatitis B license and collaboration agreement with Brii Biosciences. Revenue for the fourth quarter of 2017 was $0.2 million and was primarily attributable to sales of Sci-B-Vac in Israel. Product sales in 2018 were $0.6 million compared with $0.5 million in 2017. The change was due to an increase in product sales related to the Sci-B-Vac named-patient program in Europe, offset by a slight decrease in Sci-B-Vac sales in Hong Kong and Israel.

○Cost of Revenue for the full year 2018 was $4.5 million compared with $5.2 million for the full year 2017. This decrease was due to the Rehovot site shut-down, resulting in limited production activity, reduced maintenance and utilities expenses, and allocation of some overhead to general and administrative expenses.

○Research and development expenses for the fourth quarter and full year 2018 were $10.1 million and $38.5 million, respectively. Research and development expenses for the same periods in 2017 were $6.5 million and $20.9 million, respectively. The increase was driven by the initiation and execution of the two Sci-B-Vac Phase 3 trials and the GBM Phase 1/2a clinical trial, which are currently ongoing.

○General and administrative expenses for the fourth quarter and full year 2018 were $9.9 million and $20.8 million, respectively. General and administrative expenses for the same periods in 2017 were $3.4 million and $12.0 million, respectively. The increase was primarily attributable to a $6 million payment made in December 2018 to re-obtain a distribution agreement with a third party who previously held certain distribution rights to certain Asian markets.

○Net loss and net loss per share for the year-end 2018 were $63.6 million and $0.97, respectively, compared to a net loss of $39.0 million and a net loss per share of $0.88 for the year-end 2017

On February 25, 2019 Immunomedics, Inc., (NASDAQ: IMMU) ("Immunomedics" or the "Company"), a leading biopharmaceutical company in the area of antibody-drug conjugates (ADC), reported financial results for the Transition Period ended December 31, 2018 (Press release, Immunomedics, FEB 25, 2019, View Source [SID1234533708]). The Company also announced that Dr. Charles Baum, President and Chief Executive Officer (CEO) of Mirati Therapeutics, Inc. has joined the Immunomedics Board of Directors (Board). In addition, the Board has appointed Behzad Aghazadeh, Executive Chairman, while Scott Canute, current member of the Board, has been appointed to Executive Director. At the same time, Michael Pehl has stepped down from the role of President, CEO and member of the Board, due to personal reasons, and interim Chief Financial Officer (CFO), Usama Malik, has been appointed CFO. Please refer to the Company’s Transition Report on Form 10-K filed today with the SEC for more details on the Company’s financial results.

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"I would like to thank Michael for his leadership and service to Immunomedics over the past fifteen months. We wish him all the best as he returns to Germany. At the same time, we are honored to welcome Chuck to our Board. Chuck has a storied career in drug development culminating in his role as President and CEO at Mirati, while also serving on the Board of Array Biopharma. Chuck will bring a strong scientific, drug development, and executive management perspective that will be invaluable to Immunomedics as we continue to execute and expand on our development priorities," said Behzad Aghazadeh, Executive Chairman of Immunomedics.

"Our primary focus is to ensure a high-quality Biologics License Application (BLA) resubmission and gain FDA approval of sacituzumab govitecan for patients with metastatic triple-negative breast cancer (mTNBC). To that end, we have implemented a dedicated Complete Response Letter (CRL) team, led by our Executive Board member, Scott Canute, a recognized expert in Manufacturing and Quality spanning decades in the biopharmaceutical industry," Behzad further commented.

"We are committed to working closely with the FDA to resolve the CMC issues identified in the CRL. Based on various prior experiences with much more complex manufacturing challenges at Eli Lilly and Genzyme, I am confident that our expert teams will drive a high-quality and timely resubmission of sacituzumab govitecan," said Scott Canute, Executive Director of Immunomedics.

The CRL team is finalizing a response strategy to the issues identified in the CRL in advance of a meeting request with the FDA and anticipates providing additional clarity on the BLA resubmission timelines after such meeting has occurred.

"Sacituzumab govitecan has demonstrated a significant clinical benefit in multiple hard-to-treat cancer settings", commented Dr. Baum. "I am thrilled to join the Board of Immunomedics and leverage my experience to help navigate the near-term resubmission of the BLA for mTNBC, and shape the clinical development strategy in other late-stage indications," he added.

Dr. Baum is President, CEO and Board Member of Mirati Therapeutics since November 2012. Prior to joining Mirati, he was Senior Vice President for Biotherapeutic Clinical Research at Pfizer and served as the Head of Oncology Development and as Chief Medical Officer for Pfizer’s Biotherapeutics and Bioinnovation Center. He was responsible for the development of the oncology portfolio, including Axitinib (Inlyta), Crizotinib (Xalkori) and the approval of sunitinib (Sutent) for the treatment of gastrointestinal stromal tumor (GIST) and renal cell carcinoma. Previously, Dr. Baum was responsible for the development of several oncology compounds at Schering-Plough, including temozolomide (Temodar) which was approved for the treatment of patients with advanced brain tumors. Dr. Baum currently serves on the Board of Array BioPharma.

Mr. Scott Canute has more than 36 years of experience in the biopharmaceutical industry, previously serving as President of Global Manufacturing and Corporate Operations of Genzyme Corporation from 2010 until 2011, where he led a major turnaround in manufacturing in order to ensure on-going supply of life saving products that were in short supply and developed and implemented a comprehensive manufacturing strategy, including a revamped global governance system. Prior to joining Genzyme, Mr. Canute spent 25 years at Eli Lilly and Company, where he served as President, Global Manufacturing Operations from 2004 until 2007. During his tenure at Eli Lilly, Mr. Canute directed all manufacturing and supply chain activities for the company’s global operations, which spanned 24 international manufacturing sites and 80+ contract manufacturing operations.

Recent Company Highlights

·The Company received a CRL from the FDA for the BLA seeking accelerated approval of sacituzumab govitecan for the treatment of patients with mTNBC who have received at least two prior therapies for metastatic disease.

·The Company published in the New England Journal of Medicine updated data with sacituzumab govitecan in patients with mTNBC, showing a favorable benefit:risk profile of the ADC, as well as providing additional and consistent safety information on 420 patients with a variety of epithelial cancers.

· In an oral presentation at the 2019 Genitourinary Cancers Symposium, the Company reported updated results from a Phase 1/2 study of sacituzumab govitecan in patients with previously treated metastatic urothelial cancer, showing an overall response rate of 31 percent and a median duration of response of 12.9 months in 45 relapsed/refractory patients.

·Enrollment into the pivotal TROPHY U-01 study continues to progress well, while the Company prepares for the initiation of the registrational Phase 3 study in hormone receptor-positive metastatic breast cancer, as well as the Trop-2-enriched basket study.

· The Company has strengthened its manufacturing partnerships with Johnson Matthey and BSP to further enhance the capacity and scale of its supply chain for sacituzumab govitecan in preparation for future demand driven by additional indications and broadening of geographic footprint.

Results for the Transition Period Ended December 31, 2018

On December 14, 2018, the Company’s Board of Directors approved a change in the Company’s fiscal year end from June 30 to December 31, effective immediately. The reporting period for the six months ended December 31, 2018 is referred to as the "Transition Period."

The Company had no revenues in the Transition Period due primarily to the discontinued sale of LeukoScan during the quarter ended March 31, 2018 in order for the Company to focus on its ADC business. Revenues in the comparable period ended December 31, 2017 were approximately $1.3 million.

Total costs and expenses were $144.5 million for the Transition Period, compared to $52.3 million for the comparable period ended December 31, 2017, due primarily to a $51.1 million increase in research and development expenses, a $23.3 million increase in general and administrative expenses, and a $18.4 million increase in sales and marketing expenses. Most of these increases were attributable to activities related to preparations for the potential approval and commercial launch of sacituzumab govitecan for patients with at least two prior lines of treatment for metastatic TNBC in the United States, and to expanded clinical development of sacituzumab govitecan into other indications.

The Company recognized a $1.4 million non-cash income for the Transition Period, compared to a $59.6 million non-cash expense for the comparable period ended December 31, 2017, due to the net appreciation in the fair value of outstanding warrants and the exercise of warrants. There were no warrants outstanding as of December 31, 2018.

Interest expense was $20.0 million for the Transition Period, compared to $2.9 million for the comparable period ended December 31, 2017. The increase was due primarily to increased debt balances as a result of the agreement with RPI Finance Trust.

Net loss attributable to stockholders was $157.7 million, or $0.84 per share, for the Transition Period, compared to $121.3 million, or $0.88 per share, for the comparable period ended December 31, 2017.

As of December 31, 2018, the Company had $497.8 million in cash, cash equivalents, and marketable securities, which it believes is adequate to support its clinical development plan for sacituzumab govitecan; further build its clinical and manufacturing infrastructure and fund its operations through 2020.

Conference Call

The Company will host a conference call and live audio webcast today at 5:00 p.m. Eastern Time to discuss financial results for the quarter and six months ended December 31, 2018 and provide a corporate update. To access the conference call, please dial (877) 303-2523 or (253) 237-1755 using the Conference ID 6478246. The conference call will be webcast via the Investors page on the Company’s website at View Source Approximately two hours following the live event, a webcast replay of the conference call will be available on the Company’s website for approximately 30 days.