On March 18, 2019 Helix BioPharma Corp. (TSX: HBP) (FRANKFURT: HBP) ("Helix" or the "Company"), a clinical stage immuno-oncology company developing innovative drug candidates for the prevention and treatment of cancer, reported its financial results for its fiscal second quarter ended January 31, 2019 (Press release, Helix BioPharma, MAR 18, 2019, View Source [SID1234536459]).

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FINANCIAL REVIEW

The Company recorded a net loss and total comprehensive loss of $1,908,000 ($0.02 loss per common share) and $2,564,000 ($0.03 loss per common share) for the three-month periods ended January 31, 2019 and 2018, respectively. For the six-month periods ended January 31, 2019 and 2018, respectively, the Company recorded a net loss and total comprehensive loss of $3,287,000 ($0.03 loss per common share) and $4,868,000 ($0.05 loss per common share).

Research and development

Research and development costs for the three and six-month periods ended January 31, 2019 totalled $1,330,000 and $2,344,000, respectively ($1,895,000 and $3,660,000 respectively for the three and six-month periods ended January 31, 2018).

The following table outlines research and development costs expensed and investment tax credits for the Company’s significant research and development projects for the following periods:

L-DOS47 research and development expenses for the three and six-month periods ended January 31, 2019 totalled $788,000 and $1,649,000, respectively ($1,472,000 and $3,010,000 respectively for the three and six-month periods ended January 31, 2018). L-DOS47 research and development expenditures relate primarily to the Company’s LDOS001 Phase I clinical study in the U.S., and preliminary expenditures related to the Company’s LDOS003 Phase II clinical study in Poland, Ukraine and Hungary.

The Company’s LDOS001 clinical study continues to face patient enrolment challenges. An accelerated dosing protocol has been approved to help accelerate the LDOS001 clinical study. The Company continues to be committed to the LDOS001 study and has re-allocated limited resources to improve patient enrollment. Enrolment in the Company’s LDOS002 clinical study was previously halted at the end of stage 1 of a two-stage phase II study as the intensified schedule did not result in improving patient benefits compared to that observed in the Phase I portion of the study. The Company recently advanced some funds to the CRO overseeing the LDOS003 study and most recently announced the dosing of the first patient. The Company is in the late stages of protocol development for a Phase I/II study with L-DOS47 given in combination with doxorubicin, for the treatment of metastatic pancreatic cancer. The Company expects to file an investigational new drug application with the U.S. Food and Drug Administration for a study protocol targeting advanced pancreatic cancer patients sometime in April/May 2019.

The Company’s Polish subsidiary continues to focus its activities on the V-DOS47 pre-clinical program. V-DOS47 research and development expenses for the three and six-month periods ended January 31, 2019 totalled $102,000 and $232,000, respectively ($94,000 and $177,000 respectively for the three and six-month periods ended January 31, 2018). For the three and six-month periods ended January 31, 2019 the Company’s Polish subsidiary received grant funding of $87,000 and $222,000, respectively ($87,000 and $200,000 respectively for the three and six-month periods ended January 31, 2018). Grant funding for the V-DOS4 program is the result of an agreement entered into with the Polish National Centre for Research and Development.

CAR-T research and development expenses for the three and six-month periods ended January 31, 2019 totalled $333,000 and $333,000 respectively ($125,000 and $125,000 respectively for the three and six-month periods ended January 31, 2018). The Company commenced development of novel CAR-T therapeutics and new antibody-based technologies for cell-based therapies. The Company’s CAR-T expenditures relate primarily to collaborative research activities with ProMab Biotechnologies Inc.

Trademark and patent related expenses for the three and six-month periods ended January 31, 2019 totalled $43,000 and $68,000, respectively ($139,000 and $238,000 respectively for the three and six-month periods ended January 31, 2019). The Company continues to ensure it adequately protects its intellectual property.

Operating, general and administration

Operating, general and administration expenses for the three and six-month periods ended January 31, 2019 and 2018 totalled $533,000 and $906,000, respectively ($644,000 and $1,170,000 respectively for the three and six-month periods ended January 31, 2018). The decrease in operating, general and administration expenses mainly reflects companywide cost cutting initiatives.

The Company recorded a net loss and total comprehensive loss of $1,908,000 ($0.02 loss per common share) and $2,564,000 ($0.03 loss per common share) for the three-month periods ended January 31, 2019 and 2018, respectively. For the six-month periods ended January 31, 2019 and 2018, respectively, the Company recorded a net loss and total comprehensive loss of $3,287,000 ($0.03 loss per common share) and $4,868,000 ($0.05 loss per common share), respectively.

As at January 31, 2019 the Company had a working capital deficiency of $1,998,000, shareholders’ deficiency of $1,686,000 and a deficit of $167,292,000. As at July 31, 2018 the Company had a working capital deficiency of $1,901,000, shareholders’ deficiency of $1,527,000 and a deficit of $164,005,000.

The Company continues to work with vendors to manage its cash position while ensuring vendors continue providing services while being paid, albeit over a longer period of time than previously agreed terms. Some vendors have placed the Company on hold (cash in advance) and is impacting the Company’s clinical development program. The Company has raised gross proceeds of approximately $8,518,000 from private placement financings during fiscal 2018 and an additional $3,878,400 during the six-month period ended January 31, 2019. In addition, the Company subsequent to the January 31, 2019 quarter end, announced the closing of a private placement on March 15, 2019 for gross proceeds of $609,450. Nevertheless, the Company’s cash reserves of $306,000 as at January 31, 2019 continue to be insufficient to meet anticipated cash needs for working capital and capital expenditures through the next twelve months, nor are they sufficient to see the current or any planned research and development initiatives through to completion. Though the funds raised have somewhat assisted the Company in dealing with its working capital deficiency and attempts to make vendors current, additional funds are required to advance the various clinical and preclinical programs, pay for the Company’s overhead costs and its past due vendors. 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.

Additional information can be found about the Company’s liquidity and capital resources in the Company’s Management Discussion and Analysis.

The Company’s condensed unaudited interim consolidated statement of net loss and comprehensive loss for the three and six-month periods ending January 31, 2019 and 2018 and the condensed unaudited interim consolidated statement of cash flows for the six-month periods ending January 31, 2019 and 2018 are summarized below:

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 18, 2019 Personalis, Inc., a leader in advanced genomics for precision oncology, reported that they are scheduled to present at ICI-IO Combinations Summit 2019 in Boston on Wednesday, March 20, 2019 at 2:30 PM, EDT (Press release, Personalis, MAR 18, 2019, View Source [SID1234534801]).

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The presentation, entitled "Challenges and Solutions: Enabling multidimensional tumor immunogenomics for advancing biomarker discovery," will introduce Personalis’ new universal cancer immunogenomics platform, ImmunoID NeXT. In addition to an overview, the presentation will also highlight how this platform can be used to overcome the challenges facing immuno-oncology translational and clinical researchers. By deriving new insights through our industry leading NGS analysis platform, ImmunoID NeXT provides solutions to enable the development of safer, more effective precision oncology therapeutics and combinations.

ImmunoID NeXT is the first and only platform to provide comprehensive analysis of both a tumor and its microenvironment from a single sample. The platform can be used to investigate the key tumor- and immune-related areas of cancer biology; consolidating multiple oncology biomarker assays into one. This maximizes the biological information that can be generated from a precious tumor specimen.

The presentation will be delivered by Christelle Johnson, PhD, Senior Field Application Scientist, Cancer Genomics & Immuno-Oncology.

On March 18, 2019 Marker Therapeutics, Inc. (NASDAQ:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported that it will host a business update conference call and webcast on Thursday, March 28th at 5:00 p.m. EDT (Press release, Marker Therapeutics, MAR 18, 2019, View Source [SID1234534757]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Business Update Conference Call and Webcast

Individuals can participate in the conference call by dialing 1-855-238-2333 (domestic) or 1-412-317-5215 (international) and refer to the "Marker Therapeutics, Inc. call." The webcast will be accessible in the Investors section of the Company’s website at www.markertherapeutics.com. The archived webcast will be available for replay on the Marker website approximately two hours after the event.

On March 18, 2019 Scholar Rock Holding Corporation (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the full year ended December 31, 2018 and highlighted progress in 2018 and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAR 18, 2019, View Source [SID1234534501]).

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Key 2018 Accomplishments

Completed initial public offering and raised approximately $86 million in gross proceeds.
Entered into a strategic fibrosis-focused collaboration with Gilead Sciences, Inc. to discover and develop highly specific inhibitors of TGFβ activation ($80 million received upfront and eligible for up to an additional $1,450 million in potential milestone payments).
Transitioned to a clinical-stage company with the initiation of a Phase 1 clinical trial of SRK-015 in healthy volunteers.
Published preclinical data on the therapeutic benefit of inhibiting myostatin activation in mouse models of Spinal Muscular Atrophy (SMA) in peer-reviewed journal Human Molecular Genetics.
Received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) and Orphan Medicinal Product Designation from the European Commission (EC) for SRK-015 for the treatment of SMA.
Presented compelling preclinical data that demonstrate SRK-181-mIgG1 (murine version of SRK-181), a highly specific TGFβ1 inhibitor, can render resistant solid tumors vulnerable to PD1 blockade.
Progressed antibody platform for neuromuscular disorders, cancer immunotherapy, fibrosis, and anemias.
"2018 was a year of remarkable progress with our platform and for establishing a strong foundation as we transitioned to a public company," said Nagesh Mahanthappa, Ph.D, President and CEO of Scholar Rock. "We are focused on advancing our growing portfolio of product candidates, including SRK-015 for the treatment of SMA and SRK-181 to overcome primary resistance to checkpoint blockade therapy, as we continue to pursue our mission of developing innovative therapies to address significant unmet medical needs of patients."

R&D Highlights and Upcoming Milestones

SRK-015 Program:

Presented Interim Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers. In February 2019, Scholar Rock presented positive interim results from the Phase 1 clinical trial of SRK-015, a highly specific inhibitor of myostatin activation, which was observed to be well tolerated with no dose limiting toxicities identified up to the highest evaluated dose of 30 mg/kg. Pharmacodynamic data, measuring serum concentrations of latent myostatin, demonstrated robust and sustained target engagement, providing initial proof-of-mechanism for this unique therapeutic approach. Collectively, the favorable interim safety and tolerability, pharmacodynamic, and pharmacokinetic data supported the advancement of SRK-015 to a Phase 2 proof-of-concept clinical trial in patients with SMA.
Initiating Phase 2 Proof-of-Concept Trial for SRK-015 in SMA in First Quarter 2019. Scholar Rock is initiating a Phase 2 proof-of-concept trial to assess the safety and efficacy of SRK-015. The trial will consist of three non-overlapping cohorts, each evaluating a distinct subpopulation of patients with Type 2 and Type 3 SMA and all patients will receive SRK-015 dosed once every four weeks either as a monotherapy or in conjunction with an approved survival motor neuron (SMN) upregulator therapy. The primary efficacy endpoints will measure motor function through clinically meaningful outcome measures validated in SMA, such as the Hammersmith Functional Motor Scale Expanded (HFMSE) in non-ambulatory SMA and the Revised Hammersmith Scale (RHS) in ambulatory SMA, over a 12-month treatment period. Scholar Rock plans to provide additional details on the Phase 2 trial design at the time of initiating patient dosing in the second quarter of 2019. Interim safety and efficacy results for a subset of patients in each cohort with at least six months of treatment exposure are expected in the first half of 2020.
Plan to Identify Second Indication for SRK-015 in 2020. Scholar Rock continues to see multiple potential opportunities for which SRK-015 could offer clinical benefit and is assessing additional potential clinical settings in which the selective inhibition of the activation of myostatin may offer therapeutic benefit.
SRK-181 Program:

Nominated SRK-181 as Cancer Immunotherapy Product Candidate. In March 2019, Scholar Rock selected SRK-181, a highly specific inhibitor of TGFβ1 activation, as the first product candidate in its TGFβ1 cancer immunotherapy program based on the strength of preclinical data and human translational insights. SRK-181 is being developed for the treatment of tumors resistant to checkpoint blockade therapies (CBTs), such as anti-PD(L)1 antibodies. Scholar Rock plans to initiate a Phase 1 trial in patients with solid tumors in mid-2020.
Additional SRK-181-mIgG1 Preclinical Data to be Presented at Upcoming AACR (Free AACR Whitepaper) Annual Meeting. Scholar Rock will present additional preclinical data from multiple syngeneic mouse models of primary checkpoint resistance at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting being held March 29-April 3, 2019. These studies demonstrate that co-administration of SRK-181-mIgG1 (murine version of SRK-181) with an anti-PD1 antibody permits effector T cell infiltration and expansion into the tumor microenvironment, resulting in tumor regression or control as well as significant survival benefit, while minimizing toxicities traditionally associated with pan-TGFβ inhibitors.
Full Year 2018 Financial Results

For the year ended December 31, 2018, net loss was $49.3 million or $3.15 per share compared to a net loss of $25.0 million or $15.30 per share for the year ended December 31, 2017.

Research and development expense was $36.3 million for the year ended December 31, 2018 compared to $19.9 million for the year ended December 31, 2017. The increase year-over-year in both periods reflect development and manufacturing costs associated with lead product candidate, SRK-015, research costs associated with preclinical studies, as well as increased personnel-related costs to support continued progress with the pipeline.
General and administrative expense was $14.4 million for the year ended December 31, 2018 compared to $5.1 million for the year ended December 31, 2017. The increase year-over-year was primarily attributable to increased headcount and higher professional and consulting fees associated with the IPO and collaboration with Gilead, as well as ongoing business activities and operations as a public company.
As of December 31, 2018, Scholar Rock had cash, cash equivalents, and marketable securities of $175.6 million, compared to $58.0 million as of December 31, 2017.

"We established a strong financial foundation in 2018 with contributions from our successful IPO and our strategic fibrosis collaboration with Gilead," said Rhonda Chicko, Chief Financial Officer of Scholar Rock. "As illustrated by the recent announcement of positive interim Phase 1 results for SRK-015 and the nomination of SRK-181 as the first product candidate in our cancer immunotherapy program, we continue to focus on our robust pipeline of highly specific growth factor modulators across a diverse range of therapeutic areas."

On March 18, 2019 VBI Vaccines Inc. (NASDAQ: VBIV) ("VBI"), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported that Nell Beattie, Chief Business Officer, will present at the Oppenheimer 29th Annual Healthcare Conference on Wednesday, March 20, 2019 at 9:10 AM ET in New York City (Press release, VBI Vaccines, MAR 18, 2019, View Source [SID1234534486]).

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Details of the event are as follows:

Oppenheimer 29th Annual Healthcare Conference

Date: Wednesday, March 20, 2019

Presentation Time: 9:10 – 9:40 AM ET

Webcast: https://www.veracast.com/webcasts/opco/healthcare2019/69204395724.cfm

A live webcast of the presentation and a subsequent replay may be accessed by visiting the Investors page of VBI’s website at: www.vbivaccines.com/investors/events-presentations/. A replay of the webcast will be archived on the company’s website for 90 days following the presentation.