On December 29, 2017 CEL-SCI Corporation (NYSE American: CVM) reported financial results today for the fiscal year ended September 30, 2017 (Press release, Cel-Sci, DEC 29, 2017, View Source [SID1234522862]). The Company also reported key clinical and corporate developments achieved during and subsequent to fiscal 2017.

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Clinical and Corporate Developments included:

Full enrollment was achieved in CEL-SCI’s pivotal Phase 3 head and neck cancer study. The study’s Independent Data Monitoring Committee (IDMC) completed its most recent review of the data from all 928 patients enrolled in the study and recommended continuing the study as constituted, as there was no evidence of any significant safety questions.
CEL-SCI’s LEAPS vaccine for the treatment of rheumatoid arthritis received a $1.5 million grant from the U.S. National Institutes of Health (NIH). The grant provides funding for CEL-SCI to advance its first LEAPS product candidate, CEL-4000, towards an Investigational New Drug (IND) application. Preclinical data on CEL-4000 were presented at several scientific conferences and published in a scientific journal during fiscal 2017.
In the $50 million arbitration suit brought by CEL-SCI against its former clinical research organization (CRO), the testimony phase of the arbitration has concluded, and all that remains at the trial level are closing statements and post-trial submissions.
CEL-SCI raised approximately $14.7 million gross proceeds during fiscal 2017.
"While we went through a challenging period during fiscal 2017, we are pleased to move forward with the achievement of our biggest milestone to date, completion of enrollment and treatment of all 928 patients in the world’s largest head and neck cancer study," stated CEL-SCI CEO, Geert Kersten. "The study is now fully enrolled. Per the recommendation of the study’s IDMC to continue the study, in accordance with the study protocol, the enrolled patients are being followed for survival outcome. After 298 patient deaths have occurred in the two comparator arms of the study the company will be able to determine if the study’s endpoint is achieved."

CEL-SCI reported a net loss of ($14.36) million in fiscal year 2017 versus a net loss of ($11.51) million in fiscal 2016.

The Company’s audited financial statements contained an audit opinion from its independent registered public accounting firm that included an explanatory paragraph related to the Company’s ability to continue as a going concern.

On December 29, 2017 Progenics Pharmaceuticals, Inc. (NASDAQ:PGNX), an oncology company developing innovative medicines and imaging analytical tools for targeting and treating cancer, reported that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for AZEDRA in patients with malignant, recurrent and/or unresectable pheochromocytoma and paraganglioma, which are rare neuroendocrine tumors (Press release, Progenics Pharmaceuticals, DEC 29, 2017, View Source [SID1234522856]). The FDA granted Progenics’ request for Priority Review and has set an action date of April 30, 2018 under the Prescription Drug User Fee Act (PDUFA).

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"With no FDA-approved therapies for these rare tumors, AZEDRA has the potential to address the high unmet need of patients with malignant pheochromocytoma and paraganglioma," said Mark Baker, Chief Executive Officer of Progenics. "We are pleased that the FDA has accepted our NDA with Priority Review, and look forward to working with the Agency during the review process. At the same time, we will continue to lay the groundwork for our commercial plan and prepare to launch quickly following a potential approval."

The NDA is supported by data from a pivotal phase 2b open-label, multi-center trial that was conducted under a Special Protocol Assessment (SPA) with the FDA. The trial met the primary endpoint evaluating the proportion of pheochromocytoma and paraganglioma patients who achieved a 50% or greater reduction of all antihypertensive medication for at least six months, and showed favorable results from a key secondary endpoint evaluating the proportion of patients with overall tumor response as measured by Response Evaluation Criteria In Solid Tumors (RECIST). AZEDRA was also shown to be safe and generally well tolerated.

About AZEDRA

AZEDRA (iobenguane I 131) is a high-specific-activity radiotherapeutic product candidate in development as a treatment for malignant, recurrent, or unresectable pheochromocytoma and paraganglioma, which are rare neuroendocrine tumors of neural crest origin. AZEDRA is a substrate for norepinephrine reuptake transporter which is highly expressed on the cell surface of neuroendocrine tumors. AZEDRA has been granted Orphan Drug designation, Fast Track status, and Breakthrough Therapy designation in the U.S. Under a SPA agreement with the FDA, a phase 2b pivotal study has been completed in patients with malignant, recurrent, or unresectable pheochromocytoma and paraganglioma. There are currently no FDA-approved therapies for the treatment of this ultra-rare disease.

About Pheochromocytoma and Paraganglioma

Pheochromocytoma and paraganglioma are rare neuroendocrine tumors that arise from cells of the autonomic nervous system. Pheochromocytoma forms in the adrenal medulla, whereas paragangliomas form outside the adrenal gland. Standard treatment options for these tumors include surgery, palliative therapy and symptom management. Pheochromocytoma and paraganglioma tumors frequently secrete high levels of hormones that can lead to life-threatening hypertension, heart failure, and stroke in these patients. Malignant and recurrent pheochromocytoma and paraganglioma may result in unresectable disease with a poor prognosis, representing a significant management challenge with very limited treatment options and no approved anti-tumor therapies.

On December 29, 2017 RXi Pharmaceuticals Corporation (NASDAQ: RXII) a clinical-stage company developing a new class of RNAi-based therapeutics reported that the Company’s President and CEO, Dr. Geert Cauwenbergh, will present at the 10th Annual Biotech Showcase (Press release, RXi Pharmaceuticals, DEC 29, 2017, View Source [SID1234522789]). Taking place during one of the industry’s largest annual healthcare investor conferences, this investor and partnering conference attracts pharmaceutical executives from around the world focused on investment and business development opportunities in the life sciences industry. The conference will be held January 8–10, 2018 at the Hilton San Francisco Union Square, California.

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Date: Monday, January 8, 2018
Time: 9:30am Pacific
Track:Yosemite – C (Ballroom Level)

Dr. Cauwenbergh will present an overview of the Company’s novel self-delivering RNAi (sd-rxRNA) technology and the multiple business development and commercial opportunities available based on this proprietary platform. The presentation will be webcast and available on the "Investors – Events and Presentations" section of the Company’s website, www.rxipharma.com.

About RXi’s self-delivering RNAi (sd-rxRNA) technology platform

sd-rxRNA, RXi’s proprietary self-delivering RNAi platform, is a single chemically modified compound with delivery and therapeutic properties built directly into the compound itself. The compound is asymmetrical with a phosphorothioate backbone and contains chemical modifications that provide for efficient cellular uptake and gene silencing. These compounds are potent, stable and specific, and demonstrated to be safe and active in a clinical setting.

RXi’s novel sd-rxRNA technology differs from natural and most synthetic RNA interference (RNAi) molecules in that they are chemically modified to allow for efficient internalization of the compounds by cells and silencing of the targeted genes. Importantly, unlike other naked siRNA compounds, delivery of sd-rxRNAs are not limited to a specific cell type. For local delivery and ex vivo cell-based therapeutic applications, our compounds do not require delivery vehicles. This is a major advantage, since delivery vehicles can have related toxicity that affects cell viability. sd-rxRNA has demonstrated nearly 100 percent transfection efficiency with high cell viability in numerous cell types.

On December 29, 2017 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for RXDX-106, a novel oral immunomodulatory agent and TAM inhibitor, in patients with solid tumors (Press release, Ignyta, DEC 29, 2017, View Source [SID1234522787]).

Under this IND, the company intends to initiate the TITAN (Targeted Immunomodulatory TAM ANtagonist) study, a first-in-human, open label, multicenter, dose escalation study of RXDX-106 in patients with locally advanced or metastatic solid tumors. TITAN is designed to determine the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary clinical activity of RXDX-106.

"RXDX-106 represents a new class of immuno-oncologic precision medicines that we are excited to advance to the clinic. In preclinical studies, RXDX-106 has demonstrated the potential to elicit and potentiate an immune response to cancer, by targeting the TAM family of receptors in the tumor microenvironment, both as a single agent and in combination with checkpoint inhibitors," said Jonathan Lim, M.D., chairman and CEO of Ignyta. "This agent furthers our commitment to advancing new medicines to help patients in their fight against cancer. We look forward to commencing this Phase 1 study in early 2018."

On December 29, 2017 Medtronic plc (NYSE:MDT), the global leader in medical technology, reported it will participate in the 36th Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2018, in San Francisco (Press release, Medtronic, DEC 29, 2017, View Source;p=RssLanding&cat=news&id=2324263 [SID1234522793]).

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Omar Ishrak, chairman and chief executive officer of Medtronic, will make a formal presentation on the company beginning at 9:00 a.m. PST (11:00 a.m. CST). Shortly following the presentation, Ishrak and Karen Parkhill, executive vice president and chief financial officer, will answer questions on the company.

A live audio webcast of the presentation and Q&A session will be available on January 8, 2018, by clicking on the Investor Events link at View Source An archive of the session will be available on the same webpage later in the day.