On March 16, 2020 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company, reported financial results and recent corporate highlights for the fourth quarter and full year ended December 31, 2019 (Press release, Fortress Biotech, MAR 16, 2020, View Source [SID1234555604]).

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Fortress achieved multiple key milestones in 2019 and early 2020, including:

·Our five marketed specialty dermatology products generated 2019 net revenues of $34.9 million, representing growth of 49% compared to 2018. Fourth quarter 2019 revenues generated from our five marketed specialty dermatology products were $11.1 million, representing 85% growth over fourth quarter 2018. The products are marketed by our partner company, Journey Medical Corporation.
·We ranked number 10 in Deloitte’s 2019 Technology Fast 500, an annual ranking of the fastest-growing North American companies in the technology, media, telecommunications, life sciences and energy tech sectors, given Fortress’ 8,463% revenue growth due to the increase in Journey Medical’s net product sales from 2015 to 2018.
·We submitted a New Drug Application ("NDA") for IV tramadol to the U.S. Food and Drug Administration ("FDA") in December 2019, which represents our first NDA filing. The FDA accepted the NDA for review and assigned a Prescription Drug User Fee Act ("PDUFA") goal action date of October 10, 2020. Shortly after NDA approval of IV tramadol, and pending our fulfillment of the criteria as defined in the Stock Purchase and Merger Agreement, we plan to close the second stage of the strategic transaction between InvaGen Pharmaceuticals Inc. ("InvaGen") and our partner company Avenue Therapeutics, Inc. ("Avenue"), for an aggregate cash purchase of up to $180 million payable to Avenue shareholders, including $48 million to Fortress directly, as well as future contingent value rights.
·The FDA granted Rare Pediatric Disease Designation to Copper Histidinate, also referred to as CUTX-101, for the treatment of Menkes disease. We intend to begin a rolling NDA submission for CUTX-101 to the FDA in the fourth quarter of 2020.
·Caelum Biosciences, Inc. ("Caelum") executed an agreement with Alexion Pharmaceuticals, Inc. ("Alexion") to advance the development of CAEL-101. Under the terms of the agreement, Alexion purchased a 19.9% minority equity interest in Caelum for $30 million. Additionally, Alexion agreed to make potential payments to Caelum upon the achievement of certain developmental milestones in exchange for which Alexion obtained a contingent exclusive option to acquire the remaining equity in the company.

·We executed license agreements for several product candidates and FDA-approved products, including:
oXimino, a prescription oral antibiotic for acne, with Sun Pharma; the prescription run rate in December 2019 represents a 150% increase over the previous December with just four months of our sales effort;
oMB-108 (oncolytic virus C134) for the treatment of glioblastoma multiforme, with Nationwide Children’s Hospital; and
oAZD7325, now known as BAER-101, a novel α2/3–subtype-selective GABA A positive allosteric modulator ("PAM") for the treatment of select central nervous system ("CNS") disorders, with AstraZeneca.
·We announced confirmation of the registration path for cosibelimab in metastatic cutaneous squamous cell carcinoma ("CSCC"). FDA feedback supports the plan to submit a Biologics License Application ("BLA") based on data from the ongoing Phase 1 clinical trial. Over one-third of enrollment is complete in the cohort of patients with metastatic CSCC. There is potential for cosibelimab to be differentiated both clinically and as a lower-cost alternative to available anti-PD-1/L1 mAbs.
·We announced that the first patient treated with the optimized MB-106 (CD20-targeted, autologous CAR T cell therapy) manufacturing process achieved a complete response at the lowest starting dose in an ongoing Phase 1/2 clinical trial in patients with relapsed or refractory B-cell non-Hodgkin lymphomas.

Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "We have generated significant momentum throughout 2019 and into early 2020. In order to drive our next phase of growth, our world-class business development team continues to identify and acquire high-potential marketed and development-stage assets to further expand our portfolio of product opportunities. Additionally, Fortress and our development partners continue to advance our clinical-stage programs across multiple therapeutic categories. With five commercial products and over 25 programs in development, we aim to continue to meaningfully increase value and decrease overall risk for Fortress shareholders. Looking ahead, we expect 2020 to be a record revenue-generating year and a transformational one for many of the development-stage programs across Fortress and our partner companies. Finally, we look forward to continued acquisitions of marketable dermatology drugs and in-licenses of development-stage drug candidates."

2019 and Recent Corporate Highlights1:

Marketed Dermatology Products

·Our dermatology products are marketed by our partner company, Journey Medical Corporation.
·In 2019, our marketed products generated net revenue of $34.9 million, including $11.1 million generated in the fourth quarter of 2019. This compares to net revenue of $23.4 million in 2018, including $6.0 million generated in the fourth quarter of 2018, representing an increase of 49% year-over-year and an increase of 85% for the fourth quarter-over-quarter.
·We launched Ximino, a prescription oral antibiotic for acne, in the third quarter of 2019; the prescription run rate in December 2019 represents a 150% increase over the previous December. This impact occurred with only four months of our sales effort.
·We expect to launch one to two new prescription drugs in 2020.
·We currently have 41 sales representatives dedicated to the dermatology product portfolio and we expect that number to continue to grow this year.

IV Tramadol

·The stock purchase stage of the strategic transaction between InvaGen and Avenue closed in February 2019. InvaGen acquired approximately 5.8 million shares of Avenue Therapeutics’ common stock at $6.00 per share for total gross consideration of $35.0 million, representing a 33.3% stake in Avenue’s capital stock on a fully-diluted basis. Avenue anticipates that the merger with InvaGen will be completed shortly after the Prescription Drug User Fee Act ("PFUDA") date of October 10, 2020, if IV tramadol is approved by the FDA and pending our fulfillment of the criteria as defined in the Stock Purchase and Merger Agreement, resulting in a potential net distribution to Fortress of approximately $48 million, plus potential future product royalties.
·In June 2019, we announced that our second pivotal Phase 3 clinical trial of IV tramadol achieved the primary endpoint of a statistically significant improvement in Sum of Pain Intensity Difference over 24 hours ("SPID24") compared to placebo in patients with postoperative pain following abdominoplasty surgery. In addition, the trial met all of its key secondary endpoints. The trial also included a standard-of-care IV opioid, IV morphine 4 mg, as an active comparator. In this trial, IV tramadol also demonstrated similar efficacy and safety to that of IV morphine.
·In October 2019, an eAbstract was presented at ANESTHESIOLOGY 2019, the American Society of Anesthesiologists’ annual meeting in Orlando, FL, highlighting the Phase 3 data for IV tramadol in the management of post-surgical pain in patients undergoing bunionectomy, an orthopedic model.
·Also in October 2019, IV tramadol Phase 1 clinical data were published in the peer-reviewed journal Clinical Pharmcology in Drug Development. The paper, titled "Comparing the Pharmacokinetics of 2 Novel Intravenous Tramadol Dosing Regimens to Oral Tramadol: A Randomized 3-Arm Crossover Study," can be accessed here.
·In December 2019, we submitted our NDA to the FDA for IV tramadol for the management of moderate to moderately severe pain in adults in a medically supervised health care setting. The FDA accepted the NDA for review in February 2020 with a PDUFA goal action date of October 10, 2020.
·IV Tramadol is currently in development at our partner company, Avenue Therapeutics, Inc.

CUTX-101

·In January 2020, we announced that the FDA granted Rare Pediatric Disease Designation to CUTX-101 for the treatment of Menkes disease.
·We intend to begin the rolling submission of the NDA for CUTX-101 to the FDA in the fourth quarter of 2020.
·CUTX-101 is currently in development at our partner company, Cyprium Therapeutics, Inc.

CAEL-101

·In January 2019, Caelum signed an agreement with Alexion to advance the development of CAEL-101. Under the terms of the agreement, Alexion purchased a 19.9% minority equity interest in Caelum for $30 million. Additionally, Alexion agreed to make potential payments to Caelum upon the achievement of certain developmental milestones in exchange for which Alexion obtained a contingent exclusive option to acquire the remaining equity in the company.
·In October 2019, the European Commission granted Orphan Drug Designation to CAEL-101 for the treatment of AL amyloidosis. The FDA had previously granted two orphan drug designations to CAEL-101 for the use of CAEL-101 as a therapeutic agent for patients with AL amyloidosis and a radio-imaging agent in amyloidosis.
·Caelum received feedback from the FDA that supports initiating a pivotal Phase 2/3 program, which is expected to begin in the first half of 2020.

MB-107 (Lentiviral Gene Therapy for XSCID)

·In April 2019, the New England Journal of Medicine published data from St. Jude Children’s Research Hospital. The data are from a Phase 1/2 clinical trial of a lentiviral gene therapy, MB-107, for the treatment of newly diagnosed infants under two years old with X-linked severe combined immunodeficiency ("XSCID"), also known as bubble boy disease. Data demonstrate that MB-107 achieved normalization of T-cell numbers in all eight newly diagnosed infants with XSCID to date and disseminated infections resolved completely in all affected infants. Seven of the eight infants treated have developed normal IgM levels to date. Four of those seven infants have discontinued monthly infusions of intravenous immunoglobulin ("IVIG") therapy to date. Three of those four infants who discontinued monthly IVIG infusions have responded to vaccines to date.
·In August 2019, MB-107 was granted Regenerative Medicine Advanced Therapy ("RMAT") designation by the FDA.
·Also in August 2019, we entered into a license agreement with CSL Behring for the Cytegrity stable producer cell line, which will be used to produce the viral vector for the MB-107 lentiviral gene therapy program for the treatment of XSCID.
·Updated Phase 1/2 clinical data for MB-107 were selected for oral and poster presentations at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") Annual Meeting, which was held in December 2019. Data demonstrated that MB-107 preceded by low-dose busulfan conditioning continued to be well tolerated and resulted in development of a functional immune system both in newly diagnosed infants with XSCID, as well as in older patients with XSCID who had received prior hematopoietic stem cell transplantation ("HSCT"). Also, the enhanced transduction procedure demonstrated similar improvements in older patients with XSCID who had received prior HSCT, but with faster time to NK cell reconstitution, as well as faster time to resolution of chronic norovirus infections.
·MB-107 is currently in development at our partner company, Mustang Bio, Inc.

Cosibelimab (formerly CK-301, an anti-PD-L1 antibody)

·In September 2019, positive interim results for cosibelimab were presented at the European Society for Medical Oncology Congress 2019 in Barcelona, Spain. The poster presentation provided updated interim efficacy and safety results from the ongoing multicenter Phase 1 clinical trial of cosibelimab, including expansion cohorts in CSCC and NSCLC. A 50% objective response rate was observed in CSCC, and a 40% objective response rate was observed in NSCLC. Cosibelimab appeared to be safe and well-tolerated with a potentially favorable safety profile as compared to the currently available anti-PD-1 therapies.
·In November 2019, pharmacokinetic and target occupancy modeling data for cosibelimab were presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 34th Annual Meeting. The poster, titled "Semi-mechanistic PK and target-occupancy modeling to support dose justification for anti-PD-L1 clinical candidate CK-301 ("TG-1501") in oncology patients," compares pharmacokinetic and tumor target occupancy data at steady state under various dosing regimens of cosibelimab to those of three marketed anti-PD-L1 monoclonal antibodies: atezolizumab, durvalumab and avelumab. The results demonstrated that cosibelimab dosed at 800 mg and 1200 mg once every two weeks or every three weeks is expected to achieve over 99% PD-L1 target occupancy throughout the dosing interval, which is comparable to atezolizumab and durvalumab and higher than avelumab, at approved doses.
·In January 2020, we announced confirmation of the registration path for cosibelimab in metastatic CSCC. FDA feedback supports the plan to submit a BLA based on data from the ongoing Phase 1 clinical trial. Over one-third of enrollment is complete in the cohort of patients with metastatic CSCC. There is potential for cosibelimab to be differentiated both clinically and as a lower-cost alternative to available anti-PD-1/L1 mAbs.
·Cosibelimab is currently in development at our partner company, Checkpoint Therapeutics, Inc.

CK-101 (third-generation EGFR inhibitor)

·In March 2019, we announced two new patent issuances by the U.S. Patent and Trademark Office and the European Patent Office for CK-101. The patents cover CK-101 in the U.S. and Europe through at least August 2034, not including any potential patent term extensions.
·CK-101 is currently in development at our partner company, Checkpoint Therapeutics, Inc.

MB-102 (CD123-targeted CAR T cell therapy)

·In July 2019, the FDA granted Orphan Drug Designation to MB-102 (CD123-targeted chimeric antigen receptor T ["CAR T"]) cell therapy for the treatment of acute myeloid leukemia ("AML").
·In August 2019, we announced that the FDA approved the Investigational New Drug application to initiate a multicenter Phase 1/2 clinical trial of MB-102 in AML, blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic syndrome.
·MB-102 is currently in development at our partner company, Mustang Bio, Inc.

MB-101 (IL13Rα2-targeted CAR T cell therapy)

·In October 2019, we announced that City of Hope received $4.1 million in grant awards for a clinical trial of MB-101 (IL13Rα2-targeted CAR T cell therapy) in combination with nivolumab (commercial name: Opdivo) and ipilimumab (commercial name: Yervoy) in patients with recurrent malignant glioma. The trial, which is now enrolling patients, is the first human study to combine IL13Rα2-targeted CAR T cell therapy with checkpoint inhibitors, as well as the first to locally deliver CAR T cells with systemic nivolumab combination treatment.
·MB-101 is currently in development at our partner company, Mustang Bio, Inc.

MB-108 (Oncolytic Virus C134)

·In February 2019, we entered into an exclusive worldwide license agreement with Nationwide Children’s Hospital to develop an oncolytic virus (C134), an attenuated herpes simplex virus type 1, for the treatment of glioblastoma multiforme. We intend to combine MB-108 with MB-101 (IL13Rα2-targeted CAR T cell therapy) to potentially enhance efficacy in treating glioblastoma multiforme.
·In May 2019, the FDA granted Orphan Drug Designation to MB-108 for the treatment of malignant glioma, a type of brain cancer with a median survival of less than 18 months.
·In October 2019, we announced that the first patient was dosed in a Phase 1 clinical trial to determine the safety and efficacy of MB-108 in recurrent glioblastoma multiforme.
·MB-108 is currently in development at our partner company, Mustang Bio, Inc.

MB-104 (CS1-targeted CAR T cell therapy)

·In May 2019, we announced that City of Hope began enrolling patients with relapsed or treatment-resistant multiple myeloma in an innovative CS1 CAR T cell therapy (MB-104) trial.
·MB-104 is currently in development at our partner company, Mustang Bio, Inc.

MB-103 (HER2-targeted CAR T cell therapy)

·In August 2019, we announced that the California Institute for Regenerative Medicine ("CIRM") granted $9.3 million to City of Hope to fund an ongoing Phase 1 clinical trial of MB-103 (HER2-targeted CAR T cell therapy) for the treatment of HER2-positive breast cancer with brain metastases.
·MB-103 is currently in development at our partner company, Mustang Bio, Inc.

MB-105 (Prostate Stem Cell Antigen (PSCA)-targeted CAR T cell therapy)

·In September 2019, we announced that City of Hope opened and began to treat its first patients in a Phase 1 clinical trial of MB-105 (prostate stem cell antigen-targeted CAR T cell therapy) for the treatment of prostate cancer.
·MB-105 is currently in development at our partner company, Mustang Bio, Inc.

MB-106 (CD20-targeted CAR T cell therapy)

·Fred Hutchinson Cancer Research Center ("Fred Hutch") presented a poster about the design of the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 (CD20-targeted CAR T cell therapy) for high-risk B-cell non-Hodgkin lymphomas at the 61st ASH (Free ASH Whitepaper) Annual Meeting.
·In February 2020, we announced that the first subject treated with the optimized MB-106 (CD20-targeted, autologous CAR T cell therapy) manufacturing process, developed in collaboration between Mustang Bio and Fred Hutch, achieved a complete response at the lowest starting dose in an ongoing Phase 1/2 clinical trial. The trial is evaluating the safety and efficacy of MB-106 in subjects with relapsed or refractory B-cell non-Hodgkin lymphomas.
·MB-106 is currently in development at our partner company, Mustang Bio, Inc.

BAER-101 (novel α2/3–subtype-selective GABA A positive allosteric modulator ["PAM"])

·In December 2019, we entered into an exclusive worldwide licensing agreement with AstraZeneca for AZD7325, now known as BAER-101, a novel α2/3–subtype-selective GABA A PAM, as well as an agreement with Cincinnati Children’s Hospital Medical Center to advance clinical development in select CNS disorders.
·BAER-101 is currently in development at our partner company, Baergic Bio, Inc.

General Corporate

·In August 2019, we announced the appointment of Kevin L. Lorenz, J.D., to our Board of Directors.
·In November 2019, we announced that Fortress ranked number 10 in Deloitte’s 2019 Technology Fast 500, an annual ranking of the fastest-growing North American companies in the technology, media, telecommunications, life sciences and energy tech sectors.
·In November 2019 and February 2020, we closed on a gross total of approximately $20.4 million in two underwritten public offerings of our 9.375% Series A Cumulative Redeemable Perpetual Preferred Stock.

Financial Results:

·As of December 31, 2019, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit) and restricted cash totaled $153.4 million, compared to $156.0 million as of September 30, 2019, and $99.2 million as of December 31, 2018, a decrease of $2.6 million for the fourth quarter and an increase of $54.2 million for the full year.
·Fortress’ net revenue totaled $36.6 million as of December 31, 2019, which includes $34.9 million in net revenue generated from our marketed dermatology products, which represented growth of 49% year-over-year. This compares to a total of $26.9 million in net revenue as of December 31, 2018.
·Consolidated research and development expenses were $75.2 million for the year ended December 31, 2019, of which $72.6 million was related to partner companies. This compares to $83.3 million for 2018, of which $76.8 million was related to partner companies. Non-cash, stock-based compensation expenses included in research and development were $2.8 million for the year ended December 31, 2019, compared to $5.3 million for 2018.
·Research and development expenses from license acquisitions totaled $6.1 million for the year ended December 31, 2019, compared to $4.1 million for 2018.
·Consolidated general and administrative expenses were $55.6 million for the year ended December 31, 2019, of which $37.3 million was related to partner companies. This compares to $53.4 million for 2018, of which $33.0 million was related to partner companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $10.4 million for the year ended December 31, 2019, compared to $9.7 million for 2018.
·Consolidated net loss attributable to common stockholders was $40.0 million, or $0.73 per share, for the year ended December 31, 2019, compared to a net loss attributable to common stockholders of $84.1 million, or $1.94 per share, for 2018.

On March 16, 2020 BioSpecifics Technologies Corp. (NASDAQ: BSTC), a biopharmaceutical company that originated and continues to develop collagenase-based therapies with a first in class collagenase-based product marketed as XIAFLEX in North America, reported its financial results for the fourth quarter and full year ended December 31, 2019 and provided a corporate update (Press release, BioSpecifics Technologies, MAR 16, 2020, View Source [SID1234555620]).

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"BioSpecifics generated strong growth in 2019. We reported a 16% increase in royalty revenue received from our partner Endo’s sales of XIAFLEX and our fully diluted earnings per share grew by 22%. Looking forward, we have a number of reasons to be enthusiastic about the future growth potential for XIAFLEX. First, the market for XIAFLEX in Dupuytren’s contracture and Peyronie’s disease continues to expand, second the FDA’s decision on Endo’s new BLA for the treatment of cellulite is expected in July 2020 and third Endo has announced the commencement of development programs in two new indications, adhesive capsulitis and plantar fibromatosis," said J. Kevin Buchi, chief executive officer of BioSpecifics. "Operating from a position of sound financial strength, we intend to explore opportunities beyond XIAFLEX."

Fourth Quarter and Full Year 2019 Financial Results

BioSpecifics reported net income of $7.3 million for the fourth quarter ended December 31, 2019, or $1.00 per basic share and $1.00 per share on a fully diluted basis, compared to net income of $6.2 million, or $0.85 per basic share and $0.84 per share on a fully diluted basis, for the same period in 2018. For the full year ended December 31, 2019, the Company reported a net income of $24.5 million, or $3.34 per basic share and $3.33 per share on a fully diluted basis, compared to a net income of $20.1 million, or $2.77 per basic share and $2.73 per share on a fully diluted basis for the same period in 2018.

Total revenue for the fourth quarter ended December 31, 2019 was $11.8 million, compared to $9.9 million for the same period in 2018. For the full year ended December 31, 2019, total revenue was $38.2 million, compared to $33.0 million for the same period in 2018.

As of December 31, 2019, BioSpecifics had cash and cash equivalents and investments of $105.8 million, compared to $82.0 million as of December 31, 2018.

As of December 31, 2019, BioSpecifics had 7,339,578 million shares of common stock outstanding.

Commercial & Pipeline Highlights and Anticipated Upcoming Milestones

BioSpecifics’ Royalty Revenues from the XIAFLEX Commercial Franchise Grew by 16% Year-Over-Year for 2019: XIAFLEX royalty revenue growth was attributable to royalties associated with higher net sales of XIAFLEX by Endo International plc (Endo), in Dupuytren’s contracture and Peyronie’s disease.
Net Sales of XIAFLEX Expected to Continue to Grow in 2020: BioSpecifics’ partner, Endo, expects that XIAFLEX full year revenue growth will be approximately 20 percent in 2020.
Endo’s Biologics License Application (BLA) filing for CCH for Treatment of Cellulite Accepted by U.S. Food and Drug Administration (FDA) in November 2019: On November 19, 2019, the FDA accepted for review the original BLA for CCH for the treatment of cellulite in the buttocks. The Prescription Drug User Fee Act (PDUFA) date for the BLA, has been set for July 6, 2020.
Development in Two New Indications, Adhesive Capsulitis and Plantar Fibromatosis, Announced in 2020: Endo announced in 2020 that it expects to begin development in two new indications, adhesive capsulitis and plantar fibromatosis. Adhesive capsulitis, also known as frozen shoulder, is an inflammation and thickening of the shoulder capsule due to collagen which causes decreased motion in the shoulder. Plantar fibromatosis is a non-malignant thickening of the feet’s deep connective tissue or fascia. There are currently no FDA-approved pharmaceutical therapies available to treat either condition.

On March 16, 2020 Boehringer Ingelheim reported the launch of a patient outcomes database which will provide clinicians with important information when considering optimal treatment for non-small cell lung cancer (NSCLC) patients harboring specific uncommon epidermal growth factor receptor (EGFR) mutations (Press release, Boehringer Ingelheim, MAR 16, 2020, View Source [SID1234555568]). The real-world database includes all available data from such patients treated with afatinib.

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The data from 693 NSCLC patients published in the Journal of Thoracic Oncology (JTO) support the use of afatinib against the most prevalent uncommon EGFR mutations. In patients who had not previously received any form of EGFR tyrosine kinase inhibitor (TKI), treatment with afatinib demonstrated activity against major uncommon mutations (median time-to-treatment-failure (TTF): 10.8 months; 95% CI: 8.1–16.6; overall response rate (ORR): 60.0%), compound mutations (14.7 months; 95% CI: 6.8–18.5; 77.1%), other uncommon mutations (4.5 months; 95% CI: 2.9–9.7; 65.2%). Median duration of response (DoR) was 17.1, 16.6, and 9.0 months, respectively. Outcome in patients with exon 20 insertions was heterogeneous with a TTF of 4.2 months (95% CI: 2.8 – 5.3) with 3% having complete remissions and 5% being on treatment for more than 3 years.i

The pooled analysis was based on global clinical outcome data from 693 patients with NSCLC driven by uncommon EGFR mutations, who received afatinib as part of clinical trials, compassionate use or expanded access programs, phase IIIb trials, non-interventional trials and case series/studies in more than 40 countries. The mutations included: (29% major uncommon EGFR mutations [G719X, L861Q and S768I], 25% T790M, 21% exon 20 insertion, 13% other uncommon EGFR mutation, 12% compound mutation) which had been available via the new searchable database www.uncommonEGFRmutations.com.

This new data supports previous findings from a post-hoc analysis of the LUX-Lung trials, which demonstrated significant clinical activity of afatinib against the most prevalent uncommon mutations.ii Of the EGFR TKI prospective randomized trials to date, only IPASS (gefitinib),iii NEJ002 (gefitinib),iv and LUX-Lung 2, 3 and 6 (afatinib),v, vi included patients with uncommon EGFR mutations, meaning clinical data for other EGFR TKIs against uncommon mutations is lacking. While other TKIs have shown efficacy in a first-line setting in patients with NSCLC and common mutations (Del19, L858R), their clinical activity against uncommon mutations remains largely unknown.

Lead author Dr James Chih-Hsin Yang from the Department of Oncology at the National Taiwan University Hospital said, "The relative scarcity of prospective clinical data concerning the up to 23% of NSCLC EGFR M+ cases that fall into the ‘uncommon mutation’ category can compromise effective treatment decisions. The results from this pooled analysis, alongside the post-hoc LUX-Lung results, should support afatinib as the first-choice treatment for NSCLC patients with uncommon EGFR mutations. Additionally, the uncommon mutation database, which is going to be continuously updated as new data becomes available, will be an invaluable clinical decision-making tool for the treatment of this patient sub-population moving forward."

"The value and the richness of these new combined data establishes afatinib as the meaningful treatment option for patients with uncommon EGFR mutation in addition to its recognized role as first-line therapy for common EGFR mutations," said Dr Victoria Zazulina, Global Head of Oncology, Medicine, at Boehringer Ingelheim. "We hope that this new searchable database will inform clinical decisions and therapeutic choices for patients with various types of uncommon EGFR mutations."

The uncommon mutation data summarized within the paper are available in a searchable online database at www.uncommonEGFRmutations.com. The database can be accessed globally by clinicians and non-clinicians alike and will be regularly updated as new data becomes available.

Boehringer Ingelheim in Oncology
Cancer takes away loved ones, time and untapped potential. At Boehringer Ingelheim we are providing new hope for patients by taking cancer on. We are collaborating with the oncology community to deliver scientific breakthroughs to transform the lives of patients. Our primary focus is in lung and gastrointestinal cancers, with the goal of delivering breakthrough, first-in-class treatments that can help win the fight against cancer. Our commitment to innovation has resulted in pioneering treatments for lung cancer and we are advancing a unique pipeline of cancer cell directed agents, immune oncology therapies and intelligent combination approaches to help combat many cancers.

Boehringer Ingelheim
Improving the health of humans and animals is the goal of the research-driven pharmaceutical company Boehringer Ingelheim. The focus in doing so is on diseases for which no satisfactory treatment option exists to date. The company therefore concentrates on developing innovative therapies that can extend patients’ lives. In animal health, Boehringer Ingelheim stands for advanced prevention.

Family-owned since it was established in 1885, Boehringer Ingelheim is one of the pharmaceutical industry’s top 20 companies. Some 50,000 employees create value through innovation daily for the three business areas human pharmaceuticals, animal health and biopharmaceuticals. In 2018, Boehringer Ingelheim achieved net sales of around 17.5 billion euros. R&D expenditure of almost 3.2 billion euros, corresponded to 18.1 per cent of net sales.

As a family-owned company, Boehringer Ingelheim plans in generations and focuses on long-term success. The company therefore aims at organic growth from its own resources with simultaneous openness to partnerships and strategic alliances in research. In everything it does, Boehringer Ingelheim naturally adopts responsibility towards mankind and the environment.

On March 16, 2020 Helix BioPharma Corp. (TSX: "HBP"), a an immuno-oncology company developing drug candidates for the prevention and treatment of cancer, reported its financial results for the fiscal second quarter ended January 31, 2020 (Press release, Helix BioPharma, MAR 16, 2020, View Source [SID1234555605]).

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OVERVIEW
The Company reported a consolidated net loss and total comprehensive loss, including non-controlling interest of $2,255,000 ($0.02 loss per common share) and $4,466,000 ($0.04 loss per common share), respectively for the three and six-month period ended January 31, 2020. For the three and six-month periods ended January 31, 2019, consolidated net loss and total comprehensive loss including non-controlling interest totalled $1,908,000 ($0.02 loss per common share) and $3,287,000 ($0.03 loss per common share), respectively.

To-date in fiscal 2020, the Company has raised gross proceeds totaling approximately $16,000,000 and as a result, no longer faces a working capital deficiency. In addition, the Company during the fiscal year, to-date has divested a 49% percent stake in its Polish subsidiary and is working on divesting the remaining 51% before the end of the Company’s fiscal 2020 year-end while retaining licensing agreements for future royalties and milestones payments.

The Company has also been in discussions with various capital market firms, both in the U.S. and Canada, with the goal of raising additional capital to further advance the Company’s clinical development programs and to qualify for a NASDAQ listing.

Patient enrollment and screening commenced on the LDOS006 Phase Ib/II clinical study in the U.S. for the treatment of patients with previously treated advanced pancreatic cancer. Two patients have been enrolled to-date. The Phase Ib portion of the study involves three dose escalating cohorts enrolling a total of nine (9) patients. The Phase II portion of the study will enroll an additional eleven (11) patients depending on meeting safety and efficacy criteria. The Company’s other clinical studies for non-small cell lung cancer are in the following stages of development.

LDOS001 clinical study has completed enrollment and the Company is working on finalizing data for reporting while LDOS003 is in the last cohort of the study’s dose escalation phase. Research and development L-DOS47 research and development expenses for the three and six-month periods ended January 31, 2020 totalled $1,332,000 and $2,453,000, respectively (January 31, 2019-$788,000 and $1,649,000, respectively).

L-DOS47 research and development expenditures relate primarily to the Company’s LDOS001 Phase I clinical study in the U.S., the LDOS003 Phase II clinical study in Poland and the Ukraine and the Company’s newly approved LDOS006 Phase Ib/II clinical study in the U.S. The increase in L-DOS47 expenditures in Q2 fiscal 2020 when compared to Q2 fiscal 2019 reflects an increase of approximately $350,000 in L-DOS47 manufacturing activity to produce additional drug substance in addition to increased spend of approximately $295,000 in the Company’s newly launched pancreatic clinical study in the U.S.

The Company commenced enrollment in the new pancreatic clinical study in December 2019. For the six-month period ending Q2 2020 when compared to the six-moth period ended Q2 2019 the increase in spending mainly reflects the increase in cost associated with the commencement of the new pancreatic clinical trial in the U.S. Preclinical V-DOS47 research and development expenses for the three and six-month periods ended January 31, 2020 and 201 totalled $69,000 and $180,000, respectively (January 31, 2019-$102,000 and $232,000, respectively).

The Company’s wholly owned subsidiary in Poland has a grant funding agreement with the Polish National Centre for Research and Development ("PNCRD") for research and development expenditures associated with V-DOS47. In the three and sixmonth period ended January 31, 2020, the Company’s Polish subsidiary received grant funding of $132,000 and $159,000, respectively (January 31, 2019-$87,000 and $222,000, respectively), from the PNCRD. CAR-T research and development expenses for both the three and six-month periods ended January 31, 2020 totalled $nil, respectively (January 31, 2019-$333,000, respectively). The Company’s collaboration with ProMab Biotechnologies Inc. has been impacted by the Coronavirus pandemic and as such certain planned activities have been deferred. Trademark and patent related expenses for the three and six-month periods ended January 31, 2020 totalled $85,000 and $238,000, respectively (January 31, 2019-$43,000 and $68,000, respectively). The Company continues to ensure it adequately protects its intellectual property. Stock based compensation expense for the three and six-month periods ended January 31, 2020 totalled $40,000 and $79,000, respectively (January 31, 2019-$nil and $nil, respectively). The amount represents the expense associated with the vesting of stock options that were granted in May 2019, over their vesting period. Operating, general and administration Operating, general and administration expenses for the three and six-month periods ended January 31, 2020 totalled $654,000 and $1,363,000, respectively ($533,000 and $906,000 respectively for the three and six-month periods ended January 31, 2019). The increase is mainly the result of higher expenses associated with various third-party advisor services such as investor and media relations, legal, business development activities and investment banking services. The Company has been in discussion with various advisory groups as it pursues a listing on a recognized U.S. stock exchange, like the Nasdaq.

LIQUIDITY AND CAPITAL RESOURCES
The Company reported a consolidated net loss and total comprehensive loss including non-controlling interest of $2,255,000 for the three-month period ended January 31, 2020 (January 31, 2019-$1,908,000) and $4,466,000 for the six-month period ended January 31, 2020 (January 31, 2019-$3,287,000). As at January 31, 2020 the Company had working capital of $873,000, shareholders’ equity of $1,088,000 and a deficit of $175,938,000. As at July 31, 2019 the Company had a working capital deficiency of $3,534,000, shareholders’ deficiency of $3,281,000 and a deficit of $171,531,000.

The Company experienced a working capital deficiency for several fiscal quarters, until August 21, 2019 when the Company closed a private placement financing for gross proceeds of $7,000,005 which included a disposition of a 25% stake in the Company’s Polish subsidiary. Subsequent to the August 21, 2019 private placement and as of March 12, 2020, the Company raised an additional $9.000,000. To-date in fiscal 2020 the Company has raised a total of $16,000,000.

As previously disclosed, the Company intends to fully divest its remaining 51.0% interest in its Polish subsidiary to raise additional capital to further fund the Company’s clinical development programs for future royalties and milestone payments.

In addition, the Company has been in discussions with various capital market firms, both in the U.S. and Canada, with the goal of raising additional capital to qualify the Company for a listing on a U.S. stock exchange such as NASDAQ in order to further advance the Company’s clinical development programs.

The Company’s cash reserves of $2,094,000 as at January 31, 2020 in addition to the subsequent private placement the Company closed on March 12, 2020 are insufficient to meet anticipated cash needs for working capital and capital expenditures through the next twelve months, and nor are they sufficient to see planned research and development initiatives through to completion. Though the funds raised have materially assisted the Company in dealing with its working capital deficiency, additional funds are required to advance the Company’s clinical and preclinical programs and deal with working capital requirements To the extent that the Company does not believe it has sufficient liquidity to meet its current obligations, management considers securing additional funds, primarily through the issuance of equity securities of the Company, to be critical for its development needs. 4

The Company’s condensed unaudited interim consolidated statement of net loss and comprehensive loss for the three and six-month periods ending January 31, 2020 and 2019 and the condensed unaudited interim consolidated statement of cash flows for the six-month periods ending January 31, 2020 and 2019 are summarized below:
Consolidated Statements of Net Loss and Comprehensive Loss Consolidated Statements of Cash Flows (thousand $, except for per share data) (thousand $) 2020 2019 2020 2019 2020 2019

The Company’s condensed unaudited interim consolidated financial statements and management’s discussion and analysis will be filed under the Company’s profile on SEDAR at www.sedar.com, as well as on the Company’s website.

On March 16, 2020 Immunic, Inc. (Nasdaq: IMUX), a clinical-stage biopharmaceutical company focused on developing best-in-class, oral therapies for the treatment of chronic inflammatory and autoimmune diseases, reported financial results for the year ended December 31, 2019 and highlighted recent achievements (Press release, Immunic, MAR 16, 2020, View Source [SID1234555621]).

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"In addition to the closing of our stock-for-stock exchange transaction with Vital Therapies, Inc., which provided our Nasdaq listing and a significant capital infusion, 2019 was punctuated by the attainment of other important milestones," stated Daniel Vitt, Ph.D., Chief Executive Officer and President of Immunic. "Most notably, we completed enrollment for our phase 2 EMPhASIS trial of our lead compound, IMU-838, for patients with relapsing-remitting multiple sclerosis (RRMS), roughly nine months ahead of our initial plan. With preclinical data showing a solidly superior profile compared to DHODH inhibitor, teriflunomide, IMU-838 holds promise as an important new, best-in-class oral therapy for RRMS and other underserved immunologic diseases. We anticipate reporting top-line results in the third quarter of this year and believe that positive data could allow us to move quickly into a pivotal phase 3 trial.

"We also reported progress for IMU-838 in moderate-to-severe ulcerative colitis (UC) and primary sclerosing cholangitis (PSC), with a positive phase 2 interim dosing analysis in UC and the initiation of our investigator-sponsored, phase 2 trial in PSC, led by the Mayo Clinic. Given that the interim dosing analysis from the CALDOSE-1 study in patients with UC showed that it was likely not ineffective at the lowest 10 mg dose and that none of the three dose levels revealed unacceptable intolerance, the trial was expanded to 240 patients and top-line data is expected in the fourth quarter of 2021."

Dr. Vitt continued, "Our earlier stage programs have also advanced, with the dosing of healthy volunteers in several dose cohorts in our phase 1 trial of IMU-935, a potentially best-in-class inverse agonist of RORγt. Additionally, we plan to complete the preclinical and manufacturing activities required in order to initiate phase 1 clinical studies of IMU-856, during the first half of this year, having just announced in January 2020 the exercise of our option for the exclusive worldwide license to IMU-856 from Daiichi Sankyo, Co., Ltd. We are highly encouraged by the data we have generated thus far for our programs, most especially for IMU-838, and look forward to additional success in 2020."

Recent Highlights

January 2020: Exercised option from Daiichi Sankyo Co., Ltd. for the exclusive worldwide license to a group of compounds, designated by Immunic as IMU-856, aimed at restoring intestinal barrier function.
December 2019: Presented data, for the first time, on IMU-856, at the Crohn’s and Colitis Foundation IBD Innovate Conference. The presentation highlighted preclinical data indicating that the compound potentially restores intestinal barrier function without impairing the immune system. This represents a new and possibly disruptive approach for the treatment of intestinal diseases.
November 2019: Expanded the Board of Directors to seven members with the appointment of biotechnology executive Barclay "Buck" A. Phillips.
October 2019: Expanded the Board of Directors to six members with the appointment of industry veteran Tamar Howson.
October 2019: Announced early completion of patient enrollment (nine months ahead of initial schedule) for the phase 2 EMPhASIS trial of IMU-838, for the treatment of RRMS.
Upcoming Anticipated Clinical Milestones

Top-line data from the phase 2 EMPhASIS trial in RRMS is expected to be available in the third quarter of 2020.
Completion of the preclinical and manufacturing activities that are necessary for the initiation of phase 1 clinical studies of IMU-856 is expected during the first half of 2020.
The current, single ascending dose trial of IMU-935 is planned to be followed by a phase 1 multiple ascending dose trial in healthy volunteers and a phase 1 trial in patients with mild-to-moderate psoriasis; both are expected to start during the first half of this year.
Top-line data from the phase 2 CALDOSE-1 trial in UC is expected to be available during the fourth quarter of 2021.
Financial and Operating Results

Research and Development (R&D) Expenses were $22.5 million for the year ended December 31, 2019, compared to $9.6 million for the same period during 2018, an increase of $12.9 million. The increase is primarily due to (i) higher external development costs for the company’s IMU-838 program for the phase 2 clinical trial in patients with RRMS and UC and preparation costs related to the phase 2 clinical trial for patients with Crohn’s disease totaling $8.3 million, (ii) an increase of drug supply costs to support clinical trials of IMU-838 totaling $1.5 million, (iii) a contingent payment under the asset purchase agreement with 4SC AG settled in stock valued at $1.5 million, (iv) external R&D costs related to the company’s IMU-856 program of $1.1 million and (v) $0.5 million related to increases across numerous categories.
General and Administrative (G&A) Expenses were $14.5 million for the year ended December 31, 2019, compared to $2.4 million for the same period during 2018, an increase of $12.1 million. The increase is primarily due to (i) one-time costs related to the stock-for-stock exchange transaction completed on April 12, 2019 with Vital Therapies, including $6.4 million of stock-based compensation for executives, key employees and members of the board and $1.7 million of investment banking and legal fees, (ii) $2.6 million related to becoming a public company, including directors and officers liability insurance, audit and legal fees and personnel costs for executives and staff in the U.S. corporate headquarters, (iii) $0.5 million related to travel and (iv) $0.9 million related to increases across numerous categories.
Other Income for the year ended December 31, 2019 was $2.1 million compared to $450,000 for the same period of 2018, an increase of $1.6 million. The increase is primarily attributable to a $0.9 million year-over-year increase in reimbursement of research and development expenses in connection with the option and license agreement with Daiichi Sankyo Co., Ltd., the $0.3 million as a result of the sale of certain of Vital Therapies’ clinical development-related assets and related intellectual property, $0.3 million related to research and development tax incentives for clinical trials in Australia and $0.1 million related to interest income.
Net Loss for year ended December 31, 2019 was approximately $34.9 million, or $4.52 per basic and diluted share, based on 7,722,269 weighted average common shares outstanding, compared to a net loss of approximately $11.5 million, or $13.63 per basic and diluted share, based on 846,953 weighted average common shares outstanding for the year ended December 31, 2018. Substantially all of the company’s operating losses resulted from expenses incurred in connection with its R&D programs and from G&A costs associated with operations.
Cash and Cash Equivalents, as of December 31, 2019, of $29.4 million, is expected to fund the company’s operations into the first quarter of 2021.