On March 1, 2016 CEL-SCI Corporation (NYSE MKT: CVM) ("CEL SCI" or the "Company") reported that during the month of February it has enrolled 27 patients in its ongoing Phase 3 trial of its investigational immunotherapy Multikine* (Leukocyte Interleukin, Injection) in patients with advanced primary head and neck cancer (Press release, Cel-Sci, MAR 1, 2016, View Source [SID:1234509319]). Total patient enrollment for the trial is now 724 as of February 29, 2016 in the world’s largest Phase 3 study in head and neck cancer.

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"Through clinical centers in 24 countries, we continue to enroll patients at an average rate of about one per day. With enrollment numbers now over 700, we are nearing our full enrollment goal," stated CEL-SCI CEO Geert Kersten.

The current study goal is to enroll 880 patients through approximately 100 clinical centers in over 20 countries.

About the Multikine Phase 3 Study

The Multikine Phase 3 study is enrolling patients with advanced primary squamous cell carcinoma of the head and neck. The objective of the study is to demonstrate a statistically significant improvement in the overall survival of enrolled patients who are treated with the Multikine treatment regimen plus standard of care ("SOC") vs. subjects who are treated with SOC only.

About Multikine

Multikine (Leukocyte Interleukin, Injection) is an investigational immunotherapeutic agent that is being tested in an open-label, randomized, controlled, global pivotal Phase 3 clinical trial as a potential first-line treatment for advanced primary squamous cell carcinoma of the head and neck. Multikine is designed to be a different type of therapy in the fight against cancer: one that is given BEFORE surgery, radiation and chemotherapy because that is when the immune system is thought to be the strongest, one that appears to have the potential to work with the body’s natural immune system in the fight against tumors.

Multikine is also being tested in a Phase 1 study under a Cooperative Research and Development Agreement ("CRADA") with the U.S. Naval Medical Center, San Diego, and at University of California, San Francisco (UCSF), as a potential treatment for peri-anal warts in HIV/HPV co-infected men and women. Dr. Joel Palefsky, a world-renowned scientist and Key Opinion Leader (KOL) in human papilloma virus (HPV) research and the prevention of anal cancer, is the Principal Investigator at UCSF, which was added to the study in July 2015.

CEL-SCI has also entered into two additional co-development agreements for up to $3 million each with Ergomed Clinical Research Limited to further the development of Multikine for cervical dysplasia/neoplasia in women who are co-infected with HIV and HPV and for peri-anal warts in men and women who are co-infected with HIV and HPV.

On March 1, 2016 Provectus Biopharmaceuticals, Inc. (NYSE MKT: PVCT, www.pvct.com), a clinical-stage oncology and dermatology biopharmaceutical company ("Provectus" or "The Company"), reported that it has received a patent from the U.S. Patent and Trademark Office, U.S. Patent No. 9,273,022 (Filing, 8-K, Provectus Pharmaceuticals, MAR 1, 2016, View Source [SID:1234509318]). The patent extends the scope of protection of the manufacturing process conferred initially by U.S. Patent No. 8,530,675, issued in 2013, to include coverage of the use of an alternative raw material in manufacturing the active ingredient (API) in PV-10.

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Provectus believes that this patent, wholly owned by Provectus and conferring coverage to at least 2031, will provide further protection around the proposed commercial process for manufacturing PV-10. Investigational drug product generated using this proprietary technology is being used in all ongoing clinical trials of PV-10, including the pivotal phase 3 trial in melanoma (NCT02288897).

Provectus’ efforts to bring this process development to fruition were supported by Cambrex Charles City, Inc., a subsidiary of Cambrex Corporation (NYSE:CBM, www.cambrex.com), a life sciences company that provides products and services that accelerate and improve the development and commercialization of new and generic therapeutics.

Dr. Eric Wachter, CTO of Provectus, noted, "It is a pleasure to have Cambrex team members as co-inventors on this process patent. Although the scientists and engineers working behind the scenes aren’t always visible to patients or shareholders, these professionals work tirelessly to enable manufacturing the active ingredient in PV-10 on a commercial scale. Chemistry, Manufacturing and Controls (CMC) is a critical part of any investigational new drug (IND) application and subsequent new drug application (NDA), and this aspect of our PV-10 submission is built on a firm foundation, due in no small part to the efforts of the Cambrex team."

Dr. Kurt Kiewel, Director of R&D at Cambrex Charles City, said, "We feel fortunate to bring the depth of our experience in custom development and API manufacturing to support promising investigational products like PV-10. It has been our pleasure to work with the innovative scientists at Provectus to help advance this potential new cancer treatment toward the market."

On March 1, 2016 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported financial results for the three months and year ended December 31, 2015 and provided an overview of key milestones for the company’s lead drug candidates (Filing, Q4/Annual, Lexicon Pharmaceuticals, 2015, MAR 1, 2016, View Source [SID:1234509309]).

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"This has been a transformative year for Lexicon and our two lead drug candidates, telotristat etiprate and sotagliflozin," said Lexicon President and Chief Executive Officer Lonnel Coats. "We entered 2016 well capitalized with more than $500 million in cash and investments and well positioned to progress our first drug, telotristat etiprate, to an NDA filing and, if approved, into the market."

In November 2015, Lexicon announced that it entered into a collaboration and license agreement with Sanofi for the worldwide development and commercialization of sotagliflozin. Under the terms of the agreement, Lexicon received an upfront payment of $300 million and is eligible to receive development, regulatory and sales milestone payments of up to $1.4 billion. Lexicon is also entitled to tiered, escalating royalties on net sales of sotagliflozin.

Pipeline Progress

Telotristat etiprate is the first investigational drug in clinical studies to target tryptophan hydroxylase (TPH), the rate-limiting enzyme involved in the excess serotonin production within metastatic neuroendocrine tumor cells that can lead to carcinoid syndrome, a condition characterized by serious consequences including frequent and debilitating diarrhea, facial flushing, abdominal pain, and heart valve damage.

In December 2015, Lexicon announced top-line results from its second Phase 3 study, TELECAST, which met its primary efficacy endpoint, the percent change from baseline in urinary 5-hydroxyindoleacetic acid (5-HIAA, the main metabolite of serotonin) at week 12, the final week of the double-blind treatment portion of the study (p<0.001 for both the 250 mg and 500 mg dose arms compared to placebo). In addition, despite a lower baseline bowel movement frequency than in the first Phase 3 study, TELESTAR, telotristat etiprate achieved statistically significant reductions in daily bowel movement frequency over the 12 weeks of the study (p=0.004 for the 250 mg dose arm and p<0.001 for the 500 mg dose arm compared to placebo). Telotristat etiprate was well tolerated during the double-blind treatment period, with profiles similar to placebo for both the 250 mg and 500 mg dose arms and no overall differences observed in gastrointestinal disorders or psychiatric disorders, including changes in mood.

Sotagliflozin, which is being developed as a potential treatment for type 1 and type 2 diabetes, is a dual inhibitor of sodium-glucose transporters 1 and 2 (SGLT1 and SGLT2), each of which modulates glucose levels, and is the first investigational medicine to target both of these two proteins.

Under the collaboration with Sanofi, Lexicon will continue to be responsible for clinical development activities relating to type 1 diabetes and Sanofi will be responsible for clinical development activities relating to type 2 diabetes. Lexicon is conducting three Phase 3 clinical trials of sotagliflozin in patients with type 1 diabetes, one of which has already completed enrollment, and expects top-line results from its two pivotal Phase 3 clinical trials to be available in the second half of 2016. Lexicon expects that Phase 3 development of sotagliflozin in patients with type 2 diabetes will be initiated by Sanofi by the end of 2016.

Financial Highlights

Revenues: Lexicon’s revenues for the three months ended December 31, 2015 increased to $127.3 million from $21.5 million for the corresponding period in 2014, primarily due to revenues recognized from the collaboration and license agreement with Sanofi. For the year ended December 31, 2015, revenues increased to $130.0 million from $22.9 million for the corresponding period in 2014.

Research and Development Expenses: Research and development expenses for the three months ended December 31, 2015 increased 52 percent to $30.4 million from $20.0 million for the corresponding period in 2014, primarily due to increases in external clinical and nonclinical research and development costs. For the year ended December 31, 2015, research and development expenses increased seven percent to $95.2 million from $89.3 million for the corresponding period in 2014.

Change in Fair Value of Symphony Icon Purchase Liability: In connection with the acquisition of Symphony Icon, Lexicon made an initial estimate of the fair value of the liability for the associated base and contingent payments. Changes in this liability, based on the development of the programs and the time until such payments are expected to be made, are recorded in Lexicon’s consolidated statements of operations. For the three months ended December 31, 2015 and 2014, the fair value of the Symphony Icon purchase liability increased by $0.8 million and $0.9 million, respectively. The increase in fair value of the Symphony Icon purchase liability was $5.9 million and $1.4 million for the year ended December 31, 2015 and 2014, respectively.

General and Administrative Expenses: General and administrative expenses for the three months ended December 31, 2015 increased 62 percent to $6.4 million from $4.0 million for the corresponding period in 2014, primarily due to increased costs in preparation for commercialization of telotristat etiprate. For the year ended December 31, 2015, general and administrative expenses increased 23 percent to $23.8 million from $19.4 million for the corresponding period in 2014.

Impairment Loss on Buildings: In 2014, Lexicon began to market its buildings and land in The Woodlands, Texas for sale. Lexicon recognized non-cash impairment losses on its buildings of $3.6 million and $13.1 million for the year ended December 31, 2015 and 2014, respectively, as a result of writing down the buildings to the estimated net selling price. In January 2016, Lexicon entered into a purchase and sale agreement, under which Lexicon agreed to sell such buildings and land, subject to the negotiation and execution of a leaseback agreement with respect to a portion of the buildings.

Consolidated Net Income (Loss): Net income for the three months ended December 31, 2015 was $86.8 million, or $0.76 per diluted share, compared to a net loss of $2.9 million, or $0.03 per share, in the corresponding period in 2014. Net loss for the year ended December 31, 2015 was $4.7 million, or $0.05 per share, compared to a net loss of $100.3 million, or $1.31 per share, in the corresponding period in 2014. For the three months and year ended December 31, 2015, net loss included non-cash, stock-based compensation expense of $1.4 million and $6.8 million, respectively. For the three months and year ended December 31, 2014, net loss included non-cash, stock-based compensation expense of $1.5 million and $7.1 million, respectively.

Cash and Investments: As of December 31, 2015, Lexicon had $521.4 million in cash and investments, as compared to $256.4 million as of September 30, 2015 and $339.3 million as of December 31, 2014.

Reverse Stock Split: In May 2015, Lexicon completed a one-for-seven reverse stock split. All references to common shares and per-share data for all periods presented in this release have been adjusted to give effect to this reverse stock split.

On March 1, 2018 FLAG Therapeutics Inc. reported that the U.S. Food and Drug Administration’s Office of Orphan Products Development and the European Medicines Agency (EMA) have both granted Orphan Drug Designation to FLAG-003 for the treatment of glioma (Press release, Flag Therapeutics, MAR 1, 2016, View Source [SID1234525570]). Gliomas (including Glioblastoma) are the most aggressive forms of brain cancer and carries a very poor prognosis for survival and is one of the deadliest forms of cancer. Two and 5-year survival rates are 27% and 10%, with the median progression-free survival (PFS) being only 6.9 months.

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Orphan status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases – those which affect fewer than 200,000 Americans annually. Orphan drug designation entitles FLAG Therapeutics to 7 years marketing exclusivity following product launch in the United States (10 years marketing exclusivity in the EU) and enables the company to apply for research funding, tax credits, a waiver from FDA (PDUFA) user fees, FDA assistance in clinical trial design, and access to the central authorization procedure within the European Union.

FLAG-003 for the treatment of Glioma

FLAG-003 is a small molecule which exerts both cytotoxic and cytostatic activity due to two distinct and well characterized mechanisms of action. It possesses cytotoxic anti-tubulin activity by binding to the colchicine site of tubulin causing microtubule depolymerization. It also possesses anti-angiogenic activity through binding and inhibition of RTK receptor tyrosine kinase (RTKs) activity. The anti-tubulin and anti-angiogenic activities of FLAG-003 have translated into potent antitumor and anti-vascular effects in vivo with significantly better inhibitory activity on GBM tumor growth and vascularization than the currently approved chemotherapy, temozolomide (TMZ).