On March 18, 2019 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol, reported the appointment of Andreas Grauer, M.D., as Chief Medical Officer (Press release, Corcept Therapeutics, MAR 18, 2019, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-appoints-former-amgen-development-executive [SID1234534417]). Dr. Grauer most recently served as Vice President of Global Development at Amgen.

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"We are delighted Andreas will be joining us," said Joseph K. Belanoff, M.D., Corcept’s Chief Executive Officer. "His extensive experience advancing novel compounds through every stage of development and regulatory approval will be invaluable as Corcept’s pipeline progresses."

"There is much to do," said Dr. Belanoff. "Relacorilant is in the midst of its Phase 3 trial as a treatment for patients with hypercortisolism and in Phase 2 as a treatment for patients with advanced ovarian cancer. More indications for relacorilant, including metastatic pancreatic cancer, are under consideration. We expect to select a dose this year of CORT125281 as a treatment for patients with castration-resistant prostate cancer. And CORT118335 will enter the clinic soon as a treatment for antipsychotic induced weight gain and, later this year, non-alcoholic steatohepatitis."

"I am excited to join Corcept at this key moment," said Dr. Grauer. "Cortisol modulation has the potential to improve the lives of many seriously ill patients. I look forward to bringing my experience and expertise to bear to develop truly novel, beneficial medications."

Prior to joining Corcept, Dr. Grauer spent more than ten years at Amgen in a variety of leadership roles, most recently as Vice President of Global Development, where he led or oversaw programs in therapeutic areas including bone, nephrology, and inflammation throughout all phases of development. Dr. Grauer also brings significant experience in regulatory filings across the world that culminated in multiple new drug application and biologic license application approvals. Before Amgen, he held senior executive positions at Procter & Gamble Pharmaceuticals. Dr. Grauer holds an M.D. from the University of Heidelberg Medical School, where he graduated magna cum laude. He is Board Certified in both internal medicine and endocrinology in Germany.

On March 18, 2019 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs, reported financial results for the fourth quarter and year ended December 31, 2018 and provided a corporate update (Press release, Regulus, MAR 18, 2019, View Source [SID1234534442]).

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"2018 was a challenging year for Regulus, but I am encouraged by our program advancements and recent corporate and cost-saving efforts. Importantly, we have nearly completed the transition of the RG-012 program to Sanofi, have positioned the Company to advance our prioritized pipeline, and significantly reduced our operating cash burn. In January, we submitted a comprehensive data package to FDA for RGLS4326 for the treatment of Autosomal Dominant Polycystic Kidney Disease, or ADPKD, and look forward to their feedback, which we hope will define the path forward to resume clinical development," said Jay Hagan, President and Chief Executive Officer of Regulus.

Corporate Updates

Restructuring of Sanofi Collaboration and Transition of RG-012 to Sanofi: In November 2018, Regulus announced that it amended and restructured its Collaboration and License Agreement with Sanofi (the "Amendment"). Under the Amendment, Regulus granted Sanofi a worldwide exclusive license to develop and commercialize its investigational drug targeting miR-21 for all indications, including Alport syndrome. Under the terms of the Amendment, Regulus is eligible to receive approximately $7 million in upfront and material transfer payments. Regulus is also eligible to receive up to $40 million in development milestone payments. In addition, Sanofi will reimburse Regulus for certain out-of-pocket transition activities and assume Regulus’ upstream license royalty obligations. The transition activities for the RG-012 program to Sanofi are nearly complete and upon completion will trigger the remainder of the upfront payment due to Regulus of $2.5 million.

Term Loan Amendments: In January 2019 and March 2019, the Company amended its Term Loan with Oxford Finance to provide for additional periods of interest only for the months of February 2019 and March 2019, respectively. The maturity date of the Term Loan remains unchanged.

Lease Agreement: In February 2019, the Company entered into an amendment of its current lease (the "Lease Amendment") for approximately 59,248 square feet located at 10614 Science Center Drive, San Diego, California 92121. Under the terms of the Lease Amendment, the expiration of the current lease will be accelerated from April 30, 2024 to April 1, 2019. Concurrently with the Lease Amendment, the Company entered into a new lease agreement (the "Lease") for approximately 24,562 square feet at 10628 Science Center Drive, Suite 100, San Diego, California, 92121 which it expects to use as its new principal offices and laboratory for research and development. The commencement date of the Lease is expected to be April 1, 2019. This relocation will reduce the Company’s facility size by approximately 60% and reduce its future contractual lease obligations by approximately 70%.

Financial Results

Cash Position: As of December 31, 2018, Regulus had cash and cash equivalents of $13.9 million.

Research and Development (R&D) Expenses: R&D expenses were $5.3 million and $34.0 million for the quarter and year ended December 31, 2018, respectively, compared to $10.5 million and $53.2 million for the same periods in 2017. The decreases in R&D expenses were primarily attributable to a reduction in external development expenses associated with RG-012 during the negotiation and transfer of the program to Sanofi in the second half of 2018 and a reduction in personnel-related costs subsequent our corporate restructurings.

General and Administrative (G&A) Expenses: G&A expenses were $2.7 million and $12.9 million for the quarter and year ended December 31, 2018, respectively, compared to $3.3 million and $17.0 million for the same periods in 2017. The decreases in G&A expenses were primarily driven by non-recurring severance charges and non-recurring, non-cash stock-based compensation charges recorded during the year ended December 31, 2017 in connection with our May 2017 corporate restructuring.

Revenue: Revenue was less than $0.1 million for the quarters ended December 31, 2018 and 2017, and $0.1 million for the years ended December 31, 2018 and 2017.

Net Loss: Net loss was $8.6 million and $48.7 million for the quarter and year ended December 31, 2018, respectively, compared to a net loss of $14.4 million and $71.9 million for the same periods in 2017. Basic and diluted net loss per share was $0.98 and $5.59 for the quarter and year ended December 31, 2018, respectively, compared to $1.67 and $11.47 for the same periods in 2017.

About Autosomal Dominant Polycystic Kidney Disease (ADPKD)

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading genetic cause of end-stage renal disease. The clinical hallmark of this disease is the development of multiple fluid filled cysts primarily in the kidneys and to a lesser extent in the liver and other organs. Excessive kidney tubule derived cyst cell proliferation, a central pathological feature, fuels the expansion of cysts, ultimately causing end-stage renal disease in approximately 50% of ADPKD patients by age 60. Approximately 1 in 1,000 people bear a mutation in either PKD1 or PKD2 genes worldwide.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of PKD1 and PKD2 in human ADPKD cyst cells, a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD.

On March 18, 2019 Cell Mogrify Ltd (Mogrify), a UK company aiming to transform the future development of cell therapies, reported that it has been awarded $555,000 USD (£420,000 GBP) funding from Innovate UK, the UK’s innovation agency, through the investment accelerator for innovation in precision medicine (Press release, Mogrify, MAR 18, 2019, View Source [SID1234553931]). This funding will support the application of Mogrify’s bioinformatic approach to transition three cell therapy products to preclinical stage, with potential application in wound healing, and oncology immunotherapy.

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Mogrify will utilize its patented systematic big-data approach (Rackham et al., Nature Genetics, 2016) to identify, from next-generation sequencing and gene-regulatory networks, the sets of highly influential and non-redundant transcription factors (in vitro) or small molecules (in vivo), needed to drive the direct conversion of one mature cell type (e.g. fibroblasts) into another (e.g. T cells). The reprogramed cells will then be subjected to a number of functional tests to demonstrate bioequivalence and potential as cell therapies, such as CAR-T for the treatment of cancers.

At present, cells used in cell therapies either need to be extracted and sorted from the patient themselves or a donor, or derived via experimental protocols that can take several years to develop, all of which can delay cell therapies reaching the clinic. Mogrify aims to accelerate this process by using its novel, bioinformatic platform to deliver an efficient, safe and scalable source of cells for the development of multiple personalized regenerative cell therapy products.

Dr. Darrin M. Disley, OBE, CEO, Mogrify, said: "Mogrify’s mission is to place ourselves, and as a result the UK, at the forefront of the next generation of cell therapy companies. By embracing systematic data science approaches built on large-scale transcriptomic, cell regulatory network and epigenetic data we believe better cell therapies can be developed at a lower cost across all therapeutic areas. The funding from Innovate UK will enable Mogrify to expand its portfolio of cell types and extend its reach into new therapeutics areas to address the global cell therapy opportunity, worth an estimated $30 billion USD."

Professor Julian Gough, PhD, Co-founder and CSO, Mogrify, said: "We have already used our bioinformatic-approach to produce chondrocytes and microvascular endothelial cells by transdifferentiation and speed up the protocols for acquiring astrocytes, neurons and chondrocytes from induced pluripotent stem cells. We are already engaging with companies that would like us to support development of autologous and allogeneic T-cell therapies, and are confident that our technology has the potential to provide a platform technology on which any cell for cell therapy can be developed."

Rackham OJL et al. A predictive computational framework for direct reprogramming between human cell types. Nature Genetics. 2016 Mar;48(3):331-5. doi: 10.1038/ng.3487. Epub 2016 Jan 18.

On March 18, 2019, Vaccinex, Inc. presented the corporate presentation (Presentation, Vaccinex, MAR 18, 2019, View Source [SID1234534418]).

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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.