(Filing, 10-K, Cel-Sci, DEC 11, 2015, View Source [SID:1234508540])

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On December 11, 2015 CytomX Therapeutics, Inc. (Nasdaq:CTMX), a biopharmaceutical company developing investigational Probody therapeutics for the treatment of cancer, reported that it has selected its second clinical candidate from its Probody therapeutic pipeline (Press release, CytomX Therapeutics, DEC 11, 2015, View Source;p=irol-newsArticle&ID=2121979 [SID:1234511333]). The candidate, CX-2009, is a first-in-class Probody drug conjugate targeting CD166. CX-2009 utilizes ImmunoGen, Inc. conjugate technology.

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"CD166 is highly and homogeneously expressed in the majority of patients with a variety of solid tumors," said Michael Kavanaugh, M.D., chief scientific officer. "Despite high expression of CD166 in normal tissues, our Probody technology is designed to concentrate CX-2009 only in tumor tissue. As such, we believe that CX-2009 is uniquely positioned to deliver on the promise of CD166 as a target. We look forward to testing CX-2009 in patients and are on track to file the IND in 2017."

In October, CytomX researchers presented preclinical safety and efficacy data for the CD166 Probody drug conjugate program at the AACR (Free AACR Whitepaper)-NCI-EORTC International Conference on Molecular Targets. The data showed that CytomX’s CD166-directed Probody drug conjugates were efficacious in xenograft tumor models at doses equal to or below the predicted human dose. The therapeutics were also well-tolerated in preclinical studies at a dose predicted to be therapeutically relevant.

Therapeutics developed with CytomX’s Probody platform are designed to be active in the tumor while sparing healthy tissue. By restricting activity to the tumor microenvironment, Probody therapeutics directed against both validated and novel targets have been shown pre-clinically to enable anti-tumor efficacy with a significantly enhanced safety window, relative to traditional antibody-based therapies. CytomX’s preclinical pipeline of wholly-owned and partnered programs includes Probody cancer immunotherapies, Probody drug conjugates and T-cell engaging Probody bispecifics.

On December 11, 2015 CEL-SCI Corporation (NYSE MKT: CVM) ("CEL SCI" or the "Company") reported financial results today for the fiscal year ended September 30, 2015 (Press release, Cel-Sci, DEC 11, 2015, View Source [SID:1234508549]). The Company also reported key clinical and corporate developments achieved during and subsequent to fiscal 2015.

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Clinical and Corporate Developments Included:

CEL-SCI raised net proceeds of approximately $21.15 million during fiscal 2015 and an additional $10.6 million subsequent to the end of the fiscal year, to finance its expanding Phase 3 trial in the treatment of head and neck cancer.

During fiscal year 2015 we added 7 countries to our Phase 3 study and we enrolled another 304 patients in the study. This represented a 115% increase in enrollment over fiscal year 2014. We are continuing this trend in the first quarter fiscal 2016.
We had 2 very important corporate developments that suggest our Phase 3 trial is going well and that we have a strong case in our $50+ million arbitration against the former Clinical Research Organization ("CRO"):

In October 2015 Ergomed, the new CRO running our Phase 3 trial, added another $2 million to its existing $10 million investment in our Phase 3 clinical trial, for a total investment of $12 million. Ergomed will receive its funds back only from a successful drug.
Also in October 2015, we signed a funding agreement with Lake Whillans, a firm that specializes in litigation funding. Pursuant to the agreement, CEL-SCI will receive up to $5.0 million in funding for litigation expenses to support arbitration claims against the former CRO. This case is scheduled for "hearing" (trial) in the spring of 2016. Lake Whillans will get its funds back only from the proceeds derived from the arbitration. CEL-SCI now spends $0 on this arbitration.

CEL-SCI expanded the ongoing Phase 1 study with Multikine in HIV/HPV co-infected men and women by adding a another clinical site and a world renowned key opinion leader in the field. Dr. Joel Palefsky of the University of California, San Francisco (UCSF) joined the study in July 2015 as a Principal Investigator. The study is also being conducted under a Cooperative Research and Development Agreement (CRADA) with the U.S. Navy at the San Diego Naval Medical Center.

CEL-SCI reported an operating loss of ($34.30) million in fiscal year 2015 versus an operating loss of ($27.57) million in fiscal year 2014. Of the 2015 fiscal operating loss, the cash used by the company totaled $23.8 million. The legal expenses for the arbitration were $3.6 million and those expenses will not be repeated in 2016 since the company signed an agreement with the litigation funding firm Lake Whillans to fund any remaining arbitration legal fees. This will allow CEL-SCI to focus its expenditures on the Phase 3 trial.

CEL-SCI’s net loss available to common shareholders for the quarter ended June 30, 2015 was ($4,429,137) or ($0.05) per basic share, versus ($2,444,480) or ($0.04) per basic share during the quarter ended June 30, 2014. The net loss available to common shareholders for the nine months ended June 30, 2015 was ($24,830,691) or ($0.32) per basic share, versus ($21,261,925) or ($0.38) per basic share during the same nine months ended June 30, 2014. The increase in net loss for the three and nine month periods of 2015 as compared to the same periods in 2014 was primarily attributable to the increase in operating loss and the non-cash charge for the loss on a debt extinguishment off-set by the gain reported of the non-cash charge for the change in value of derivative instruments caused by a decrease in the Company’s common stock.

The rise in operating loss was largely attributable to an increase in research and development expenses to $20.95 million in fiscal year 2015 compared to $17.0 million in fiscal year 2014. This expense increased based on the additional activity level of the Phase 3 clinical trial, including more clinical centers joining the study and more patients enrolled.

Geert Kersten, CEL-SCI’s Chief Executive Officer said, "We are rapidly enrolling patients in our Phase 3 trial in head and neck cancer and the trial date for our arbitration is near. Both of these efforts are proceeding well, and we look forward to a positive 2016."

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 appears to have the potential to work with the body’s natural immune system in the fight against tumors.

On December 11, 2015 the U.S. Food and Drug Administration reported that it approved Vistogard (uridine triacetate) for the emergency treatment of adults and children who receive an overdose of the cancer treatment fluorouracil or capecitabine, or who develop certain severe or life-threatening toxicities within four days of receiving these cancer treatments (Press release, , DEC 11, 2015, View Source [SID:1234508548]).

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"Treating cancer requires not only selecting which drug may be most effective and well tolerated, but ensuring the correct dose is given at proper intervals. While rare, unintentional overdose can occur," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "Today’s approval is a first-of-its-kind therapy that can potentially save lives following overdose or life-threatening toxicity from these chemotherapy agents."

Fluorouracil (taken by infusion) and capecitabine (taken orally) are similar types of chemotherapy that have been used for decades to treat several types of cancer, including breast and gastrointestinal cancers. An overdose of fluorouracil or capecitabine is rare, but when it occurs, the effects are serious and can be fatal.

Vistogard, taken orally, blocks cell damage and cell death caused by fluorouracil chemotherapy. Patients should take Vistogard as soon as possible after the overdose (whether or not they have symptoms) or early-onset (within four days) of severe or life-threatening toxicity. The patient’s health care provider will determine when he or she should return to the prescribed chemotherapy after treatment with Vistogard.

The efficacy and safety of Vistogard were studied in 135 adult and pediatric cancer patients who were treated in two separate trials and had either received an overdose of flourouracil or capecitabine, or had early-onset, unusually severe or life-threatening toxicities within 96 hours after receiving flourouracil (not due to an overdose). The studies’ primary measure was survival at 30 days or until chemotherapy could resume if prior to 30 days. Of those who were treated with Vistogard for overdose, 97 percent were still alive at 30 days. Of those treated with Vistogard for early-onset severe or life-threatening toxicity, 89 percent were alive at 30 days. In both studies, 33 percent of patients resumed chemotherapy in less than 30 days.

Vistogard is not recommended for treating non-emergency adverse reactions associated with flourouracil or capecitabine because Vistogard may lessen the efficacy of these drugs. The safety and efficacy of Vistogard initiated more than 96 hours following the end of treatment with flourouracil or capecitabine have not been established.

The most common side effects of treatment with Vistogard were diarrhea, vomiting and nausea.

The FDA granted Vistogard orphan drug designation, which provides financial incentives, like clinical trial tax credits, user fee waivers, and eligibility for market exclusivity to promote rare disease drug development. Vistogard was also granted priority review and fast track designations, which are distinct programs intended to facilitate and expedite the development and review of certain new drugs in light of their potential to benefit patients with serious or life-threatening conditions.

Vistogard is marketed by Wellstat Therapeutics Corporation based in Gaithersburg, Maryland.

On December 11, 2015 the U.S. Food and Drug Administration reported that it approved Alecensa (alectinib) to treat people with advanced (metastatic) ALK-positive non-small cell lung cancer (NSCLC) whose disease has worsened after, or who could not tolerate treatment with, another therapy called Xalkori (crizotinib) (Press release, , DEC 11, 2015, View Source [SID:1234508547]).
Lung cancer is the leading cause of cancer death in the United States, with an estimated 221,200 new diagnoses and 158,040 deaths in 2015, according to the National Cancer Institute. An ALK (anaplastic lymphoma kinase) gene mutation can occur in several different types of cancer cells, including lung cancer cells. ALK gene mutations are present in about 5 percent of patients with NSCLC. In metastatic cancer, the disease spreads to new parts of the body. In ALK-positive NSCLC metastatic patients, the brain is a common place for the disease to spread.

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"Today’s approval provides a new therapy for a group of patients who would have few treatment options once their disease no longer responds to treatment with Xalkori," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "In addition to the primary effect on tumors in the lung, Alecensa clinical trials provide evidence of an effect on tumors that had spread to the brain, which is an important effect for clinicians to understand."

Alecensa is an oral medication that blocks the activity of the ALK protein, which may prevent NSCLC cells from growing and spreading.

The safety and efficacy of Alecensa were studied in two single-arm clinical trials of patients with metastatic ALK-positive NSCLC whose disease was no longer controlled by treatment with Xalkori. Study participants received Alecensa twice daily to measure the drug’s effect on their lung cancer tumors. In the first study, 38 percent of participants experienced a partial shrinkage of their NSCLC tumors, an effect that lasted for an average of 7.5 months. In the second study, 44 percent of participants experienced a partial shrinkage of their NSCLC tumors, lasting for an average of 11.2 months. The trials also examined Alecensa’s effect on individuals’ brain metastases, a common occurrence in this population. Sixty-one percent of participants in the two trials who had measurable brain metastases experienced a complete or partial reduction in their brain tumors, lasting an average of 9.1 months.

The most common side effects of Alecensa are fatigue, constipation, swelling (edema) and muscle pain (myalgia). Alecensa may cause serious side effects, including liver problems, severe or life-threatening inflammation of the lungs, very slow heartbeats and severe muscle problems. Treatment with Alecensa may cause sunburn when patients are exposed to sunlight.

Alecensa was approved using the accelerated approval regulatory pathway, which allows the FDA to approve products for serious or life-threatening diseases based on evidence that the product has an effect on an outcome that is reasonably likely to predict clinical benefit. In the case of Alecensa, the tumor response to treatment, along with the duration of response, provided this evidence. Under the accelerated approval requirements, a confirmatory study is required to verify and describe the clinical benefit of Alecensa.

The FDA granted the Alecensa application breakthrough therapy designation and priority review status. These are distinct programs intended to facilitate and expedite the development and review of certain new drugs in light of their potential to benefit patients with serious or life-threatening conditions. Alecensa also received orphan drug designation, which provides incentives such as tax credits, user fee waivers and eligibility for exclusivity to assist and encourage the development of drugs for rare diseases.