On February 10, 2020 CEL-SCI Corporation (NYSE American: CVM) reported financial results for the quarter ended December 31, 2019 (Press release, Cel-Sci, FEB 10, 2020, View Source [SID1234554101]).

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In October 2019, the Independent Data Monitoring Committee (IDMC) for the Company’s pivotal Phase 3 head and neck cancer study of its investigational immunotherapy Multikine* (Leukocyte Interleukin, Injection) performed an official review of the study data and recommended that the trial continue until the appropriate number of events has occurred. The data from all 928 enrolled patients were provided to the IDMC by the clinical research organization (CRO) responsible for data management of this Phase 3 study. The IDMC reviewed "progression free and overall survival and limited demographic and safety data available for the aforementioned protocol."
The Company is now awaiting final study results for the Phase 3 trial, which is the largest study in the world in head and neck cancer. All that remains to be done is to continue to track patient survival until the required number of events have occurred and all of the data have been reviewed and recorded in the study database to allow a determination to be made if the primary endpoint has been met. The primary endpoint of the study is a 10% improvement in overall survival of the Multikine treatment regimen plus standard of care (SOC) vs. SOC alone, the two main comparator arms of the study.
"Our study has now been running for 9 years with the last patients being enrolled in September 2016. We believe that the delay in reaching 298 events is a good sign for the study because patients appear to be living longer than was expected when the study was planned. However, we would be surprised if the study did not end soon. Since the study is well controlled and the SEER data base shows no improvement in the survival of patients treated with standard of care since the study began, it seems illogical that the patients living longer than expected would be patients receiving the standard of care therapies only. We are therefore preparing for commercial scale production of Multikine at our manufacturing facility," stated CEL-SCI CEO, Geert Kersten.

In December 2019, the Company raised gross proceeds of approximately $5.5 million through an underwritten public offering of 606,395 shares of its common stock at a price of $9.07 per share. In January 2020, the underwriters fully exercised the over-allotment option of an additional 90,959 shares of common stock at a price of $9.07 per share bringing the total gross proceeds to approximately $6.325 million.

CEL-SCI reported a net loss of approximately $5.5 million for the quarter ended December 31, 2019 versus net income of approximately $1.2 million for the quarter ended December 31, 2018. The net income decrease was mainly due to the non-cash derivative gain of approximately $0.8 million for the three months ended December 31, 2019 versus a gain on derivative instruments of approximately $5.6 million for the three months ended December 31, 2018. Net interest expense also decreased by approximately $0.2 million for the three months ended December 31, 2019 compared to the three months ended December 31, 2018.

CEL-SCI’s total operating expense increased by approximately $1.6 million for the quarter ended December 31, 2019 versus the quarter ended December 31, 2018. General and administrative expenses increased by approximately $0.9 million compared to the three months ended December 31, 2018. Approximately $0.7 million of the change relates to an increase in the non-cash employee stock compensation expense. Research and development expenses increased by approximately $0.7 million compared to the three months ended December 31, 2018. Major components of this increase include approximately $0.7 million of cost incurred preparing the manufacturing facility for the potential commercial manufacture of Multikine, $0.5 million increase in non-cash employee stock option expense and $0.2 million increase in depreciation expense on the manufacturing facility as a result of adopting the new leasing standard. These increases were offset by a decrease of approximately $0.7 million in expenses related to the Company’s on-going Phase 3 clinical trial.

On February 10, 2020 Gibson Oncology, LLC ("Gibson"), a privately held clinical stage company, obtained world-wide,reported an exclusive commercial rights to a novel series of 56 rationally designed compounds called Azaindenoisoquinolines ("Aza Compounds"), from Purdue University and the National Cancer Institute (Press release, Gibson Oncology, FEB 10, 2020, View Source [SID1234554100]). These Aza Compounds have proven to be dual inhibitors of cMyc and topoisomerase I (TOP1).

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Previous attempts over decades to target cMyc, a major oncogene involved in driving 80% of all tumors, have failed because of the peculiar characteristics of the shallow binding pockets on the cMyc protein, which created the reputation of being an "undruggable" target. Gibson’s novel Aza Compounds were rationally designed to avoid this issue through a novel mechanism of action (MOA) involving the stabilization of the G-quadruplex in the cMyc promoter, resulting in inhibition of transcription of the cMyc gene and thus inhibition of production of the Myc protein. In addition, these Aza Compounds are also potent TOP1 inhibitors, which synergizes with their G-quadruplex stabilizing activity and further enhances their anti-cancer abilities.

Gibson has already signed contracts to produce clinical candidates suitable for an IND.

Dual targeting of cMyc and TOP1 may serve as a novel and highly effective anticancer strategy across a spectrum of cancers. New intellectual property on composition of matter and method of use claims cover such novel compounds to at least 2037.

Randall Riggs, the CEO of Gibson Oncology, LLC, said that "We are excited about the Aza Compounds because scientific literature reports that 80% of all tumors are driven by cMyc. Developing a cMyc inhibitor has been long sought and offers a potential breakthrough therapeutic for many cancers."

On February 10, 2020 Aptorum Group Limited (Nasdaq: APM) ("Aptorum Group"), a biopharmaceutical company focused on the development of novel therapeutics to address global unmet medical needs, reported positive data and development in relation to its repurposed drug candidate, SACT-1, for the treatment of neuroblastoma, a rare type of childhood cancer that develops in infants and young children (Press release, Aptorum, FEB 10, 2020, View Source [SID1234554099]). Subject to completion of current validation studies, Aptorum Group plans to leverage the 505(b)(2) pathway and submit an IND submission with the FDA for SACT-1 in H2 2020.1

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SACT-1 is the first repurposed drug candidate to be developed under the Smart-ACTTM drug discovery platform, which employs a systematic approach to identify, repurpose and develop existing approved drugs against a currently identified universe of 7000+ (and increasing) orphan diseases.2

Through this platform, Aptorum Group intends to accelerate and fast track repurposed drug candidates, which usually have well established human safety and toxicity profiles and data, through the development and clinical phases in order to address the rapidly growing market of orphan diseases. Aptorum Group aims to screen a number of orphan disease areas including, but not limited to, oncology, autoimmune, metabolic and genetic diseases.

Through the Smart-ACTTM platform, Aptorum has successfully identified potential efficacy for and develops SACT-1 for the treatment of neuroblastoma, being an entirely new therapeutic area from its approved indication. In our recent studies, SACT-1 has been shown to be effective against numerous neuroblastoma cell lines, of which 2 are MYCN-amplified cells, which represent the high-risk neuroblastoma patient group. In addition, by using a combination index as a quantitative measure of the extent of drug interaction, Aptorum Group has seen a high and robust synergism between SACT-1 and traditional chemotherapy in vitro, indicating a potential efficacy enhancement/dose reduction of the chemotherapy. In addition, in our recent study, the maximum tolerable dose of SACT-1 in a rodent model was determined to be higher than 400mg/kg. Compared with the MTD of standard chemotherapy such as paclitaxel (20-30mg/kg)3 and cisplatin (6mg/kg) 4, the safety profile of SACT-1 appears to be very impressive.

The reformulation of SACT-1 is a pediatric formulation to better address the needs of neuroblastoma patients who are exclusively children younger than 5. Based on our internal observations of pre-existing information from approved products,5 SACT-1 also exhibits a well-established safety profile: at 150mg/day, the death rate was 0% in prior clinical studies) with no dosage related adverse events.

About neuroblastoma

Neuroblastoma is a rare form of cancer, and classified as an orphan disease, that forms in certain types of nerve tissue and most frequently in the adrenal glands as well as spine, chest, abdomen or neck, predominantly in children, especially for those aged 5 years and below. For the high-risk group, which is close to 20%6 of total new patient population per year, the 5-year survival rate of this condition is around 40-50% as observed by the American Cancer Society7. The current high drug treatment cost for high risk patients can average USD200,000 per regimen (all 6 cycles)8. In addition, most pediatric patients often do not tolerate or survive the relevant chemotherapy stage which, subject to further clinical studies, may be positively addressed by the SACT-1 candidate due to the potential synergistic effects when applied with standard chemotherapy as described above.

On February 10, 2020 TriSalus Life Sciences, a company committed to developing comprehensive solid tumor solutions, reported that Mary Szela, President and CEO, will speak on the panel, "Expanding the Toolkit for Fighting Solid Tumors" at the 2020 BIO CEO & Investor Conference in New York City (Press release, TriSalus Life Sciences, FEB 10, 2020, View Source [SID1234554098]).

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This panel will explore the breadth of the solid tumor-fighting pipelines and those clinical benefits likely to be realized soonest. Experience has shown that to date there has been limited success with the currently approved or experimental therapeutics in impacting solid tumors and that these often have undesirable side effects. While we have witnessed a surge of systemic therapies in recent years (For example, the check point inhibitors), the need for safer and truly effective treatment remains.

However, exciting approaches in other mechanisms of action are also expanding our understanding of solid tumors, the tumor microenvironments, and the opportunities to disrupt tumor growth and help cancer patients. "I am honored to participate in such an important panel discussion of how we can improve outcomes in solid tumors through new approaches such as TriSalus’ tri-pronged approach which combines CAR-T with an immuno-modulation agent and proprietary, intravascular delivery to treat liver metastases and pancreatic cancer," said Mary Szela, President and CEO of TriSalus.

Now in its 22nd year, the BIO CEO & Investor Conference is one of the largest independent investor conferences focused on established and emerging publicly traded and select private biotech companies including two days of productive partnering meetings with institutional and early-stage investors, industry analysts, and senior biotechnology executives, in one location.

Mary has limited availability during the conference. If you are interested in setting up a meeting with Mary, please contact Lisa DeScenza from LaVoieHealthScience at [email protected].

On February 10, 2020 Immunis.AI, a privately held immunogenomics company with a patented liquid biopsy platform, reported that its President and Chief Executive Officer, Mark McDonough, will present at the BIO CEO & Investor Conference being held in New York City from February 10-11, 2020 (Press release, ImmunisAI, FEB 10, 2020, View Source [SID1234554097]).

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Date: Tuesday, February 11th

Time: 10:00 AM

Track: Diagnostics

Venue: Ziegfeld Room, New York Marriott Marquis, 1535 Broadway, New York, NY 10036

To schedule a meeting with Mark McDonough during the conference, please contact [email protected].