On November 9, 2018 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products, reported financial results and recent corporate highlights for the third quarter ended September 30, 2018 (Press release, Fortress Biotech, NOV 9, 2018, View Source [SID1234531206]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "Fortress and our subsidiaries continued to achieve important corporate and clinical milestones in the third quarter of 2018. Mustang Bio expanded its pipeline into gene therapy by securing an exclusive worldwide license for the development of a potentially first-in-class ex vivo lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("X-SCID") from St. Jude Children’s Research Hospital ("St. Jude"). Additionally, Checkpoint Therapeutics announced positive interim safety and efficacy data from its Phase 1/2 clinical trial of CK-101, a third-generation epidermal growth factor receptor ("EGFR") inhibitor being evaluated in advanced non-small cell lung cancer ("NSCLC"). Finally, Cyprium Therapeutics’ product candidate for patients diagnosed with classic Menkes disease, CUTX-101, was granted Fast Track Designation by the U.S. Food and Drug Administration ("FDA"). We plan to continue to acquire and develop compelling and, in some instances, potentially life-saving product candidates, which could lead to maximizing shareholder value."

Financial Results:

· As of September 30, 2018, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit), cash deposits with clearing organizations and restricted cash totaled $136.1 million, compared to $168.3 million as of December 31, 2017, a decrease of $32.2 million year-to-date.
· Net revenue totaled $63.7 million for the third quarter of 2018, compared to $46.9 million for the third quarter of 2017. Total revenue as of September 30, 2018 includes $5.2 million of Fortress revenue, primarily from the sale of Journey Medical Corporation products, and $58.5 million of revenue from National Holdings Corporation1 ("National Holdings"). Total revenue as of September 30, 2017 included $2.5 million of Fortress revenue and $44.4 million of revenue from National Holdings.
· Research and development expenses were $16.1 million for the third quarter of 2018, of which $15.1 million was related to Fortress Companies. This compares to $15.9 million for the third quarter of 2017, of which $14.2 million was related to Fortress Companies. Non-cash, stock-based compensation expenses included in research and development were $1.8 million for the third quarter of 2018, compared to $1.6 million for the third quarter of 2017.
· Research and development expenses from license acquisitions were $3.7 million for the third quarter of 2018, compared to $0.3 million for the third quarter of 2017.
· General and administrative expenses were $12.2 million for the third quarter of 2018, of which $7.4 million was related to Fortress Companies. This compares to $15.1 million for the third quarter of 2017, of which $10.5 million was related to Fortress Companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.3 million for the third quarter of 2018, compared to $2.6 million for the third quarter of 2017.
· National Holdings’ operating expenses totaled $55.2 million for the third quarter of 2018, compared to $47.7 million for the third quarter of 2017.
· Net loss attributable to common stockholders was $16.6 million, or $0.37 per share, for the third quarter of 2018, compared to a net loss attributable to common stockholders of $27.1 million, or $0.67 per share, for the third quarter of 2017. For the first nine months of 2018, net loss attributable to common stockholders was $59.3 million or $1.36 per share, compared to $56.5 million or $1.39 per share in the first nine months of 2017.

On November 9, 2018 VBI Vaccines Inc. (NASDAQ: VBIV) ("VBI"), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported financial results for the third quarter ending September 30, 2018, and provided a corporate update (Press release, VBI Vaccines, NOV 9, 2018, View Source [SID1234531205]).

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"The last few months have seen meaningful progress across our portfolio, marked by achievements in our Sci-B-Vac Phase 3 studies, PROTECT and CONSTANT, and early data from our Phase 1/2a study of VBI-1901 in recurrent glioblastoma (GBM)," said Jeff Baxter, President and CEO. "We have now completed vaccination and enrollment in the PROTECT and CONSTANT studies, respectively, with an exceptionally low drop-out rate, and no safety signals observed or reported to-date. These accomplishments support the clean safety profile of Sci-B-Vac and reaffirm the timeline for top-line data from the PROTECT and CONSTANT trials, expected to read out mid-year 2019 and around the 2019 year-end, respectively."

Mr. Baxter continued, "Additionally, we are encouraged by the early immunologic data we’ve seen from our Phase 1/2a study of VBI-1901 in recurrent glioblastoma (GBM), which has shown evidence of robust boosting of CMV-specific immunity in some subjects in both the low-dose and intermediate-dose cohorts. With the addition of two top neuro-oncology sites, Dana-Farber Cancer Institute and Massachusetts General Hospital, we expect to complete enrollment in the high-dose cohort quickly. Thanks to the dedication and achievements of the entire VBI team, the fundamentals of the company remain strong and on-track as we head into a critical and exciting first half of 2019."

Recent Highlights and Upcoming Milestones

Formation of Three Scientific and Clinical Advisory Boards

● In September, VBI announced the creation of three Scientific and Clinical Advisory Boards (SABs) comprised of leading global experts in immunology and vaccinology. The SABs will work closely with VBI’s management team to develop and advance the Company’s hepatitis B, cytomegalovirus (CMV), and glioblastoma (GBM) vaccine programs.

Appointment of Chief Financial Officer and Head of Business Development

● In August, VBI appointed Christopher McNulty as Chief Financial Officer and Head of Business Development, bringing with him a strong background in licensing transactions, capital markets and financing strategy, and public company corporate finance in the biopharmaceutical and medical device fields.

Sci-B-Vac for Hepatitis B

● The Company’s prophylactic Hepatitis B vaccine, Sci-B-Vac, is currently being evaluated in a global, pivotal Phase 3 clinical program, the results of which are intended to support future regulatory and marketing authorization submissions in the U.S., Europe, and Canada. The program consists of two concurrent Phase 3 studies:
● PROTECT Study: A head-to-head immunogenicity study against Engerix-B.

○ In October, VBI announced completion of vaccination, with the last subject receiving the final vaccination.
○ Patients in the PROTECT study were vaccinated with a low drop-out rate of 4.5%, reinforcing the safety and tolerability of the vaccine.
○ The independent Data and Safety Monitoring Board (DSMB) reviewed all PROTECT safety data available to-date and did not observe or report any safety signals.
○ Top-line data from the PROTECT study are expected mid-year 2019.
● CONSTANT Study: A lot-to-lot consistency study.
○ VBI completed enrollment in the CONSTANT study, with the last subject receiving the first vaccination in November.
○ Top-line data from the CONSTANT study are expected around the 2019 year-end.

VBI-1901 for Glioblastoma (GBM)

● The Phase 1/2a study of VBI-1901 in recurrent GBM is ongoing, with early immunologic data to be presented in a poster discussion at the Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology (SNO) on November 16, 2018.
○ Early data from the low- and intermediate-dose cohorts of VBI-1901 are encouraging, with robust boosting of CMV-specific immunity, directed against multiple antigens, observed in some subjects in both cohorts.
○ In September 2018, VBI announced that the DSMB unanimously recommended continuation of the study without modification after review of all safety data from the fully-enrolled, intermediate-dose patient cohort.
○ Following the DSMB recommendation, VBI initiated patient enrollment in the high-dose cohort of Part A of the study, the dose-escalation phase.
○ VBI expects to complete enrollment in the high-dose study arm later in 2018, at which point a final, pre-specified DSMB review will occur before initiation of enrollment in Part B of the Phase 1/2a study.
○ VBI expects to report more extensive immunologic data and 6-month overall survival and progression-free survival from all dose cohorts in Part A of the Phase 1/2a in the first half of 2019.

VBI-1501 for Congenital Cytomegalovirus (CMV)

● Following the positive safety and immunogenicity data from the Phase 1 randomized, observer-blind, placebo-controlled study evaluating the safety and immunogenicity of VBI-1501 in May 2018, discussions are ongoing with regulatory bodies to determine the design of a Phase 2 dose-ranging study.
● Further details are expected to be announced by the end of 2018.

Third Quarter 2018 Financial Results

○ VBI ended the third quarter of 2018 with $26.0 million in cash and cash equivalents compared with $67.7 million as of December 31, 2017. Net cash used in operations for the nine months ended September 30, 2018 was $38.0 million.

○ Revenue for the third quarter of 2018 was $0.26 million and was primarily attributable to sales of Sci-B-Vac in Israel and in Europe on a named-patient basis. Revenue for the third quarter of 2017 was $0.19 million and was primarily attributable to sales of Sci-B-Vac in Israel.

○ Research and development expenses were $10.5 million for the third quarter of 2018, as compared to $5.2 million for the same period in 2017. The increase was primarily due to the continuation of the Phase 3 program for Sci-B-Vac and the Phase 1/2a clinical study for VBI-1901 in recurrent GBM patients.

○ General and administrative expenses were $3.5 million for the third quarter of 2018 as compared to $2.8 million for the same period in 2017. The increase was primarily due to allocation of expenses from costs of revenues related to the modernization and capacity increases of our manufacturing facility in Rehovot, Israel, human resources expenses, and stock-based compensation expenses.

○ Net loss and net loss per share for the third quarter of 2018 were $15.4 million and $0.24 respectively, compared to a net loss of $9.8 million and a net loss per share of $0.24 for the third quarter of 2017.

On November 9, 2018 Synthorx, Inc., a biotechnology company using a first-of-its-kind Expanded Genetic Alphabet platform technology to discover and develop optimized biologics for cancer and autoimmune disorders, reported company researchers will present results of preclinical studies demonstrating the safety and anti-tumor effects of its lead product candidate THOR-707, a "not-alpha" Synthorin of interleukin-2 (IL-2), for the treatment of solid tumors at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 33rd Annual Meeting (Press release, Synthorx, NOV 9, 2018, View Source [SID1234531204]). Marcos Milla, Ph.D., chief scientific officer of Synthorx, will present the poster on Friday, November 9, from 12:45 – 2:15 p.m. and 6:30 – 8:00 p.m. ET at the Walter E. Washington Convention Center in Washington, D.C.

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The study, titled "THOR-707: An engineered IL-2 for the treatment of solid tumors with superior preclinical efficacy and safety evidence," investigates the application of Synthorx’s Expanded Genetic Alphabet platform technology to engineer THOR-707, a variant of recombinant human IL-2 that is pegylated at one specific site and designed to kill tumor cells without inducing vascular leak syndrome (VLS), a toxicity associated with aldesleukin, an approved recombinant IL-2. Aldesleukin was approved by the U.S. Food and Drug Administration over 25 years ago for the treatment of patients with metastatic renal cell carcinoma and metastatic melanoma. However, widespread use of aldesleukin has been limited by toxicities, which include life-threatening and sometimes fatal VLS, as well as by its short half-life, requiring courses of dosing of three times per day over eight days.

In Synthorx’s preclinical studies, THOR-707 induces molecular markers of T cell activation and the proliferation of peripheral NK and CD8+ effector T cells in vivo. However, dosing of THOR-707 has minimal effect on molecular and clinical markers of VLS, even at high exposures. Analysis of the effect of THOR-707 in syngeneic mouse tumor models showed that THOR-707 induced infiltration of CD8+ T effector cells into tumors and elicited anti-tumor effects alone and in combination with a PD-1 immune checkpoint inhibitor.

On November 9, 2018 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported that it will present at the following upcoming investor conferences (Press release, Ultragenyx Pharmaceutical, NOV 9, 2018, View Source [SID1234531203]):

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Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President, will present at the Credit Suisse Healthcare Conference on Wednesday, November 14, 2018 at 11:30 a.m. MT in Scottsdale, Arizona.
Shalini Sharp, the company’s Chief Financial Officer, will present at the Piper Jaffray Healthcare Conference on Tuesday, November 27, 2018 at 11:30 a.m. ET in New York.
Dr. Kakkis will present at the Evercore HealthConX on Wednesday, November 28, 2018 at 11:00 a.m. ET in Boston.
The live and archived webcast of the company presentations will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

On November 9, 2018 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company developing targeted and immuno-oncology therapeutics, reported a business update and financial results for the third quarter ended September 30, 2018 (Press release, Leap Therapeutics, NOV 9, 2018, View Source [SID1234531197]).

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"Our team has executed extremely well over the past quarter, rapidly enrolling multiple clinical trials and presenting impressive clinical and preclinical data for both of our programs. We are excited about the potential for our DKN-01 antibody in patients with esophagogastric cancer, both in combination with KEYTRUDA (pembrolizumab) and with paclitaxel. We believe that the emerging response and disease control rates, and now the progression-free survival and overall survival data, reflect outcomes that are clinically meaningful to patients with very difficult to treat cancer and are greater than what has previously been demonstrated with approved single agents," commented Christopher K. Mirabelli, Ph.D, President and Chief Executive Officer of Leap Therapeutics. "In addition, we look forward to presenting data from the studies of our TRX518 antibody in combination with KEYTRUDA, OPDIVO (nivolumab) or gemcitabine at the European Society for Molecular Oncology 2018 Immuno-Oncology Congress in December."

Recent Developments

Since the end of the second quarter, the Company has continued to make strong progress with the development of their product candidates.

DKN-01/KEYTRUDA: At the European Society for Molecular Oncology (ESMO) (Free ESMO Whitepaper) 2018 Annual Congress, Leap presented clinical data from a study evaluating DKN-01 in combination with KEYTRUDA (pembrolizumab) in patients with advanced esophagogastric cancer. As of the cut-off date for the poster, DKN-01 and pembrolizumab had a 23.5% overall response rate and 58.8% disease control rate in evaluable gastric or gastroesophageal junction cancer patients who have been heavily pre-treated and not received any prior anti-PD-1/PD-L1 therapy. All four of the responding patients had tumors that were microsatellite stable, which is a subgroup of patients who have historically experienced a less than ten percent response rate to KEYTRUDA monotherapy.

DKN-01/PACLITAXEL: At the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 33rd Annual Meeting, Leap presented an update on a clinical study evaluating DKN-01 in combination with paclitaxel in patients with advanced esophagogastric cancer. The combination of DKN-01 and paclitaxel generated a 46.7% overall response rate, 19.6 weeks median progression free survival, and 61.1 weeks median overall survival in fifteen evaluable patients as a second line therapy. In the benchmark RAINBOW study, paclitaxel monotherapy in second line gastroesophageal junction or gastric cancer patients generated a 16.1% overall response rate, 2.9 months median progression free survival, and 7.4 months median overall survival. Additionally, in our study in the subgroup of twelve evaluable patients with heavily pre-treated esophageal squamous cell carcinoma, the combination of DKN-01 and paclitaxel produced a 33.3% overall response rate, 13.7 weeks median progression free survival, and 31.0 weeks median overall survival.

DKN-01 in HCC: The first patient has been enrolled in Leap’s investigator-initiated study of DKN-01 as a monotherapy and in combination with NEXAVAR (sorafenib) in hepatocellular carcinoma patients with Wnt pathway activation.

TRX518/BAVENCIO: In July, Leap announced a collaboration agreement with Pfizer and Merck KGaA, Darmstadt, Germany to evaluate TRX518 in combination with BAVENCIO (avelumab) and cyclophosphamide chemotherapy. Under the terms of the collaboration, Leap will be conducting a Phase I/II clinical trial in advanced solid tumors including expansion populations in patients with relapsed/refractory ovarian, breast, and prostate cancers.

· TRX518/KEYTRUDA or OPDIVO or GEMCITABINE: During the third quarter, Leap presented initial data from a clinical trial evaluating TRX518 in combination with gemcitabine chemotherapy or in combination with KEYTRUDA (pembrolizumab) or OPDIVO (nivolumab). Three patients treated with TRX518 in combination with anti-PD1 antibodies have experienced clinical benefit. An esophageal squamous cell carcinoma patient treated with TRX518 and KEYTRUDA demonstrated a partial response with a 77% reduction in tumor volume, and an ocular melanoma patient experienced stable disease with a 23% reduction in tumor volume. An urothelial carcinoma patient who had progressed while on KEYTRUDA has had a partial response with TRX518 and OPDIVO with a 39% reduction in tumor volume. We have also fully enrolled the TRX518 and gemcitabine expansion cohort. We plan to present additional data from this trial at the ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress in December 2018.

Selected Third Quarter 2018 Financial Results

Net loss was $6.6 million for the third quarter of 2018, compared to $6.8 million for the same period in 2017.

Research and development expenses were $6.5 million for the third quarter 2018, compared to $6.8 million for the same period in 2017. The decrease of $0.3 million was primarily due to a decrease of $1.3 million in manufacturing costs related to clinical trial material. This decrease was partially offset by an increase of $0.7 million in clinical trial costs, an increase of $0.2 million in payroll and other related costs and an increase of $0.1 million in stock based compensation expense.

General and administrative expenses were $2.1 million for the third quarter 2018, compared to $1.8 million for the same period in 2017. The increase of $0.3 million in general and administrative expenses was primarily due to a $0.2 million increase in stock based compensation expense and an increase of $0.1 million in legal, audit and consulting fees associated with corporate and business development activities.

Cash, cash equivalents and marketable securities totaled $23.2 million at September 30, 2018. Research and development incentive receivables totaled $2.1 million. In October 2018, we received $0.8 million of research and development tax incentive payments from the Commonwealth of Australia. We anticipate that our current cash resources will extend until late in the third quarter of 2019.