On February 16, 2017 Cellceutix Corporation, (OTCQB: CTIX) ("the Company"), a clinical stage biopharmaceutical company developing innovative therapies with dermatology, oncology, anti-inflammatory, and antibiotic applications, reported a general business update, corporate outlook for on upcoming clinical milestones for its first-in-class drug candidates and select financial results for the second quarter of the fiscal year 2017 ended December 31, 2016 (Press release, CellCeutix, FEB 16, 2017, View Source [SID1234517730]). A comprehensive review of the latest quarter reported is available through Cellceutix’s recently submitted Form 10-Q with the Securities and Exchange Commission at www.sec.gov and on the Cellceutix website.

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Portfolio Development

The second quarter of fiscal 2017 and the start of our third quarter proved very positive for Cellceutix’s three lead compounds, Brilacidin, Prurisol and Kevetrin. The past few months laid the groundwork for some fast-approaching milestones that represent pivotal moments for Cellceutix. As mentioned by management in previous news releases, a primary focus for the 2017 calendar year is completing work necessary to forge partnerships with larger pharmaceutical companies presently engaged with Cellceutix, some under Confidential Disclosure Agreements (CDAs). Should outcomes from ongoing clinical studies yield favorable results, the Company is optimistic that it will be able to capitalize on the data with respect to a partnership. Securing partnerships would generate substantial shareholder value, while also helping to advance the Company’s innovative drug candidates toward market approval, benefiting patients in need of additional safe and effective treatment options.

· Brilacidin—Oral Mucositis (Phase 2 Trial)



· Enrolling 60 patients, the study is evaluating Brilacidin as an oral rinse, 3 times daily for 7 weeks, to prevent and control Oral Mucositis (OM) in patients receiving chemoradiation therapy for head and neck cancer. Even though over 400,000 patients in the U.S. suffer from this condition, presently there are no approved drugs for the treatment of OM in this population, with only limited palliative care options available. Cellceutix’s goal with this trial is to show that Brilacidin has dual functionality in not only shortening the duration and intensity of OM in patients with head and neck cancers, but also preventing the onset of the condition, an accomplishment no other pharmaceutical company has achieved.



· Upcoming Milestone: Interim analysis of the Phase 2 trial is expected in approximately 6 to 8 weeks.


· Brilacidin—Inflammatory Bowel Diseases Ulcerative Proctitis/Proctosigmoiditis (Phase 2a Trial)



· This Proof-of-Concept trial includes enrolling 18 patients divided evenly into three cohorts. Review of safety data from the first 6 patients revealed that Brilacidin, administered for 6 weeks as a retention enema, at 50 mg once daily, appeared well-tolerated, with no measurable systemic absorption detected. Clinically meaningful improvements in symptoms were also demonstrated, as measured by physician assessments and patient reported outcomes, further supported with endoscopic evaluation. Brilacidin may show greater efficacy at higher dosing levels in the trial, which are comprised of 100 mg for the current cohort and 200 mg for the third cohort. Cellceutix believes that a successful trial creates an opportunity to explore Brilacidin for an array of other hard-to-treat Inflammatory Bowel Diseases that current lack effective therapeutics.


· Upcoming Milestone: Cellceutix projects that an interim analysis of the second cohort will be completed in approximately two weeks. The trial is anticipated to be completed in 2Q2017.




Over the next month to two months, Cellceutix expects to have interim data from both Brilacidin trials. This is particularly important for the development of Brilacidin and pursuit of partnerships as the data can provide clinical evidence of the immunomodulatory and anti-inflammatory properties of Brilacidin for indications that represent areas of unmet medical needs.

· Prurisol – Chronic Plaque Psoriasis (Phase 2b Trial)


· Subject recruitment is now ongoing across the United States. The study increases the total daily oral dosing of Prurisol from a previous high of 200 mg, which was shown to be well-tolerated in a successfully completed Phase 2a trial, to include oral Prurisol 300 mg per day, oral Prurisol 400 mg per day, and placebo (3:1:3 randomization). Enrolling approximately 189 patients with moderate to severe chronic plaque psoriasis, treatment duration is 12 weeks (84 days). Primary efficacy is being evaluated using the Psoriasis Area and Severity Index (PASI). The Company believes now is an opportune time to develop an oral treatment for psoriasis given that currently available treatment options are limited, with many not easily administered and often associated with undesirable side effects.


· Kevetrin – Advanced Ovarian Cancer (Phase 2a Trial)


· As disclosed on February 10, 2017, a Phase 2a trial of Kevetrin (CTIX-KEV-201) has commenced. This is an open-label study evaluating the safety, tolerability, and pharmacokinetics of Kevetrin, as well as changes in select biomarkers and objective tumor response when administered to patients with platinum-resistant/refractory ovarian cancer. The clinical trial comprises two different short-term treatment regimens and will enroll an estimated 10 patients. The trial will directly inform how Kevetrin modulates the p53 signaling pathway. Cellceutix is consulting with a world renowned expert on p53 and a member of the National Academy of Sciences who helped design the study, which is intended to provide potential partners key information on the mechanism of action of the novel compound.

"I think it is pretty easy to see why we are thrilled at our prospects with four different mid-stage trials ongoing," said Arthur P. Bertolino, MD, PhD, MBA, President and Chief Medical Officer at Cellceutix. "Each study has a valuable component in demonstrating the robustness and potential of our pipeline and I greatly look forward to what I suspect will be a 2017 with a steady flow of information across all four trials."

"Success in any one of our mid-phase trials could instantly transform the Company overnight," commented Cellceutix Chief Executive Officer Leo Ehrlich. "Having already engaged in productive discussions with large pharmaceutical companies, Cellceutix remains partnership-focused in 2017. Potentially life-saving drugs and tremendous value creation await patients and shareholders alike as we strive to deliver compelling trial results."


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Select Financial Results for the Second Quarter of Fiscal 2017

The current primary potential source of cash available to the Company are equity investments through its $30.0 million equity purchase agreement with Aspire Capital signed in March 2015. The Company has financed its operations to date through the sale of its common stock. The Company had raised approximately $11.0 million from initiation of the equity purchase agreement with Aspire Capital through the three months ended December 31, 2016, leaving approximately $19.0 million available. Additionally, as of December 31, 2016, Cellceutix had approximately $3.9 million in cash on hand.

For the three months ended December 31, 2016, the Company’s net loss was approximately $3.36 million, or $0.03 per share, compared to a net loss of $3.32 million, or $0.03 per share, for the three months ended December 31, 2015.

On February 16, 2017 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the fourth quarter and year ended December 31, 2016 (Press release, Agios Pharmaceuticals, FEB 16, 2017, View Source [SID1234517729]). In addition, Agios highlighted select corporate milestones and data presentations for its preclinical and clinical development programs.

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"Our 2016 accomplishments, including the enasidenib NDA submission with our collaboration partner Celgene and clear proof-of-concept data for our PK deficiency program, demonstrate our ability to transform our scientific discoveries into important precision medicines," said David Schenkein, M.D., chief executive officer at Agios. "In 2017, we are focused on making the transition to a commercial stage company by delivering our lead cancer programs to patients, bringing our first rare genetic disease program into pivotal development and advancing our next research program, focused on MTAP-deleted cancers, into the clinic."

KEY UPCOMING MILESTONES

The company expects to achieve the following key milestones:

IDH Mutant Inhibitors in Hematologic Malignancies

Potential approval of enasidenib in the United States for IDH2m positive relapsed/refractory (R/R) acute myeloid leukemia (AML) in collaboration with Celgene by the end of 2017.
Submit a new drug application (NDA) to the U.S. FDA for ivosidenib (AG-120) for IDH1m positive R/R AML by the end of 2017.
Initiate a global, registration-enabling Phase 3 study (AGILE) combining ivosidenib (AG-120) and VIDAZA in newly diagnosed AML patients with an IDH1 mutation ineligible for intensive chemotherapy in the first half of 2017.
IDH Mutant Inhibitors in Solid Tumors

Complete the dose-escalation phase of the ongoing Phase 1 study of AG-881 in IDHm positive glioma in the first half of 2017.
Rare Genetic Diseases

Finalize design for a global pivotal trial of AG-348 in pyruvate kinase (PK) deficiency in the third quarter of 2017.
Initiate a global pivotal trial of AG-348 in PK deficiency in the first half of 2018.
Cancer Metabolism Research:

Submit an Investigational New Drug (IND) application for the development candidate targeting methylthioadenosine phosphorylase (MTAP)-deleted tumors by the end of 2017. MTAP is a metabolic enzyme that is deleted in approximately 15 percent of all cancers.
ANTICIPATED 2017 DATA PRESENTATIONS

First data from the expansion phase of the ongoing Phase 1 study of ivosidenib (AG-120) in R/R AML in the second half of 2017
First data from the ongoing Phase 1b combination study of enasidenib or ivosidenib (AG-120) with standard-of-care intensive chemotherapy in newly diagnosed AML in the second half of 2017
First data from the cholangiocarcinoma expansion cohort of the ongoing Phase 1 study of ivosidenib (AG-120) in advanced IDH1m positive solid tumors in the first half of 2017
Updated data from the glioma expansion of the ongoing Phase 1 study of ivosidenib (AG-120) in advanced IDH1m positive solid tumors in the second half of 2017
Updated data from AG-348 Phase 2 DRIVE PK study in PK deficiency in both the first and second half of 2017
Updated preclinical data for the program targeting MTAP-deleted tumors at the Keystone Tumor Metabolism Meeting taking place March 5-9, 2017 in Whistler, British Columbia
FOURTH QUARTER 2016 HIGHLIGHTS

Supported Celgene’s submission of an NDA for enasidenib in IDH2m positive R/R AML.
Initiated a global, registration-enabling randomized Phase 3 study (ClarIDHy) for ivosidenib (AG-120) in IDH1m positive advanced cholangiocarcinoma. The FDA also granted ivosidenib Fast Track Designation for the treatment of patients with previously treated, unresectable or metastatic cholangiocarcinoma with an IDH1 mutation.
Completed the dose-escalation phase of the Phase 1 study of AG-881 in IDHm positive hematologic malignancies. The study is now closed for enrollment.
Selected a development candidate targeting MTAP-deleted tumors to enter IND-enabling studies.
FULL YEAR 2016 FINANCIAL RESULTS

Cash, cash equivalents and marketable securities as of December 31, 2016 were $573.6 million, compared to $375.9 million as of December 31, 2015. This increase was driven by cash received under our collaboration agreements with Celgene totaling $258.2 million, which includes a $200 million upfront payment from the May 2016 collaboration agreement, $25 million related to initiation of the enasidenib Phase 3 IDHENTIFY study and $33.2 million of program funding, net proceeds of $162.1 million received from the company’s September 2016 public offering, and $7.9 million from stock award activities. These items were offset by a decrease in cash related to expenditures to fund operating activities and purchases of fixed assets of $230.6 million during the year ended December 31, 2016.

Collaboration revenue was $69.9 million for the year ended December 31, 2016, compared to $59.1 million for the prior year.

Research and development (R&D) expenses were $220.2 million, including $25.4 million of stock-based compensation expense, for the year ended December 31, 2016, compared to $141.8 million, including $17.4 million in stock-based compensation expense, for the year ended December 31, 2015. The increase in R&D expenses was primarily due to increased costs to support advancement of the company’s lead investigational medicines toward later-stage development. Celgene is responsible for all development costs for enasidenib and certain development costs for AG-881 and reimburses the company for development costs incurred for these investigational medicines.

General and administrative (G&A) expenses were $50.7 million, including $16.7 million of stock-based compensation expense, for the year ended December 31, 2016, compared to $36.0 million, including $14.5 million of stock-based compensation expense, for the year ended December 31, 2015. The increase in G&A expense was largely due to increased headcount and other professional expenses to support growing operations.

Net loss for the year ended December 31, 2016 was $198.5 million, compared to a net loss of $117.7 million for the year ended December 31, 2015.

CASH GUIDANCE

Based on its current operating plans, the company expects that its existing cash, cash equivalents and marketable securities as of December 31, 2016, together with anticipated interest income, and anticipated payments from Celgene under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2018.