On March 7, 2019 CEL-SCI Corporation (NYSE American: CVM) reported it has been selected by the U.S. National Institutes of Health (NIH) for sponsorship to exhibit and showcase its presentation for its LEAPS technology at the BIO International Convention, to be held June 3-6, 2019 in Philadelphia (Press release, Cel-Sci, MAR 7, 2019, View Source [SID1234534084]).

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LEAPS, which is currently being developed as a therapeutic vaccine for rheumatoid arthritis under a $1.5 million SBIR/STTR grant from the NIH, will be featured at the convention’s Innovation Zone, a turn-key exhibit space dedicated to showcasing select NIH SBIR/STTR awardees.

"We are honored that our LEAPS technology has been selected by the NIH to be showcased at Bio International. Through the NIH’s commitment to assist awardees like CEL-SCI in our path to commercialization, we expect LEAPS will benefit from the premier visibility it will receive from potential development partners and investors at this year’s BIO International Convention," stated CEL-SCI CEO Geert Kersten.

Upon completion of preclinical and Investigational New Drug (IND) enabling studies for the LEAPS-based rheumatoid arthritis vaccine candidate CEL-4000, CEL-SCI intends to file an IND application with the U.S. Food and Drug Administration.

About Bio International Convention

The Convention is the world’s largest biotechnology conference and exhibition. Hosted by BIO, the annual event attracts over 16,000 biotech leaders from 72 countries and covers the wide spectrum of life science innovations and application areas including drug discovery, biomanufacturing, genomics, biofuels, nanotechnology, cell therapy, and more.

About LEAPS

This platform technology has been shown in several animal models to preferentially direct the immune response to a cellular (e.g., T-cell), humoral (antibody) or mixed pathway and has been shown to involve upregulation of T-regulatory (Treg) cells in some animal models. It has the potential to be utilized in diseases for which antigenic epitope sequences have already been identified, such as: a number of infectious diseases, some cancers, autoimmune diseases (e.g., RA), allergic asthma and allergy, and select CNS diseases (e.g., Alzheimer’s).

On March 7, 2019 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the fourth quarter ended December 31, 2018 (Press release, Syndax, MAR 7, 2019, View Source [SID1234534110]). In addition, the Company provided a clinical and business update. As of December 31, 2018, Syndax had $80.9 million in cash, cash equivalents and short-term investments.

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"We expect the coming months to be a very exciting, milestone-rich time for Syndax as we continue to work towards our mission of realizing a future in which cancer patients live longer and better lives than previously possible," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "We remain highly encouraged by the potential for a positive overall survival readout in E2112, our Phase 3 registration trial of entinostat plus exemestane in HR+, HER2- breast cancer, and expect the next interim analysis in the second quarter. As a reminder, any positive overall survival result would allow us to file for full regulatory approval in an indication for which existing therapies have failed to show a survival benefit. In addition, we continue to expect to file an IND in the second quarter for our targeted therapy, SNDX-5613, an inhibitor of the Menin-MLL interaction. Preclinical data from our Menin inhibitor program has provided strong, consistent support for the therapeutic potential of this class in patients with genetically-defined acute leukemias, a disease area lacking effective options."

Syndax also reported that both ENCORE 602, the Phase 1b/2 clinical trial evaluating the combination of entinostat plus Genentech’s PD-(L)1 inhibitor, TECENTRIQ (atezolizumab), in patients with triple negative breast cancer (TNBC), and ENCORE 603, the Phase 1b/2 trial evaluating entinostat in combination with BAVENCIO (avelumab), an anti-PD-(L)1 co-developed and co-commercialized by Merck KGaA Darmstadt, Germany and Pfizer, in patients with ovarian cancer, failed to meet their respective primary endpoints of demonstrating an improvement in progression free survival (PFS).

With results across the entire ENCORE program now in hand, Syndax has decided to defer advancement of the entinostat-PD-1 combination program, including the previously announced ENCORE 607 registration trial in non-small cell lung cancer (NSCLC). Going forward, the Company will primarily focus its resources on advancing entinostat in HR+ breast cancer and SNDX-5613, a Menin inhibitor being developed for genetically-defined population of acute leukemias. Following availability of positive E2112 OS results, the Company will determine whether to advance its entinostat-PD-1 combination programs into one or more registration trials.

Dr. Morrison added, "While we are encouraged by ENCORE 601 entinostat-KEYTRUDA combination data in NSCLC and melanoma which suggests that entinostat has the ability to overcome resistance in PD-1 refractory patients, we believe that it is in the best interest of our stakeholders to prioritize our resources ahead of the E2112 OS readout, at which time we will make a determination on next steps for the I-O combination program. We are highly committed to advancing the balance of our pipeline programs, with an emphasis on our targeted therapy, SNDX-5613, and the E2112 registrational trial for HR+ breast cancer."

Pipeline Updates

Entinostat

The Company continues to anticipate the next interim OS analysis for E2112, its NCI-sponsored, ECOG-ACRIN led Phase 3 registration trial of entinostat, a Class I selective HDAC inhibitor, plus exemestane in advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer, in the second quarter of 2019. Additional interim analyses will be conducted by ECOG-ACRIN approximately every six months until either an OS benefit is observed, or the final target number of events occur. Any positive OS assessment would enable the Company to file for full regulatory approval. The E2112 trial design was informed by the Phase 2b ENCORE 301 trial, the results of which led to entinostat’s Breakthrough Therapy designation in HR+, HER2- breast cancer, in which patients receiving the entinostat/exemestane combination demonstrated a statistically significant OS benefit.

As previously announced, data from the NSCLC and melanoma cohorts of the ENCORE 601 trial will each be featured during oral presentations at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Meeting later this month. Data to be presented will include the Company’s most recent insights into the potential mechanisms that allow entinostat to enhance the benefit of immune checkpoint therapy.

The Phase 1b/2 ENCORE 603 trial, which evaluated entinostat in combination with avelumab in patients with heavily pretreated advanced epithelial ovarian cancer, and the Phase 1b/2 ENCORE 602 trial, which evaluated entinostat in combination with atezolizumab in patients with PD-1 naïve, previously treated TNBC, each failed to meet its respective primary endpoint of a statistically significant improvement in PFS.

Based on the activity observed to date, the Company has decided not to advance the ENCORE 601 cohort of patients with microsatellite stable colorectal cancer (MSS-CRC) to the second stage of the trial.

SNDX-5613

Preclinical data supporting the Company’s Menin-Mixed Lineage Leukemia (MLL) inhibitor program were presented during an oral session at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2018.

The Company continues to expect to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for its Menin inhibitor, SNDX-5613, in the second quarter of 2019, with the initiation of a Phase 1 clinical trial in a defined subset of acute leukemias patients expected to follow.

SNDX-6352

The Company continues to anticipate initial results from the Phase 1 dose escalation trial of SNDX-6352, the Company’s anti-CSF-1R monoclonal antibody, in patients with chronic graft versus host disease (cGVHD) in the second half of the year. The objectives of this trial are to evaluate the safety and preliminary efficacy of SNDX-6352 in cGVHD and to identify a recommended Phase 2 dose and schedule.

The Company continues to anticipate identifying a recommended Phase 2 dose and schedule for SNDX-6352 monotherapy and in combination with IMFINZI (durvalumab), AstraZeneca’s human monoclonal antibody directed against PD-L1, in the second quarter of 2019. The dose selections will be based on the results of the ongoing Phase 1/1b ascending dose trial evaluating the safety of SNDX-6352 alone or in combination with durvalumab.

Fourth Quarter 2018 Financial Results

As of December 31, 2018, Syndax had cash, cash equivalents and short-term investments of $80.9 million and 26.8 million shares issued and outstanding.

License fee revenue decreased to $0.4 million in the fourth quarter 2018 from $1.2 million for the prior year fourth quarter, and for 2018 decreased to $1.5 million compared to $2.1 million for the prior year. The decreases are due to the ratable recognition of a $5.0 million payment from KHK for the achievement of a development milestone in the fourth quarter of 2017.

Fourth quarter 2018 research and development expenses decreased to $15.8 million from $16.6 million, and for the full year increased to $60.1 million compared to $48.2 million for 2017. The fourth quarter decrease was primarily due to expensing a payment of $5.0 million to Allergan to acquire SNDX-5613 in the fourth quarter of 2017, offset by an increase in SNDX-6352 manufacturing expenses. The increase for the full year was primarily due to increased expenses for SNDX-6352 manufacturing, SNDX-5613 program expenses and increased headcount, offset by the payment of $5.0 million to Allergan in 2017.

General and administrative expenses for the fourth quarter 2018 decreased to $3.9 million from $4.1 million, and, for the year ended December 31, 2018 increased to $17.3 million compared to $15.9 million for the prior year. The full year increase was primarily due to increased pre-commercialization expenses.

For the three months ended December 31, 2018, Syndax reported a net loss attributable to common stockholders of $18.8 million or $0.70 per share compared to $19.1 million or $0.80 per share for the prior year period. For the year ended December 31, 2018, Syndax reported a net loss attributable to common stockholders of $74.0 million or $2.92 per share, compared to $60.8 million or $2.90 per share for the prior year.

Financial Guidance

Today the Company provided operating expense guidance for the first quarter and full year 2019. For the first quarter and full year 2019, research and development expenses are expected to be $11 to $13 million and $46 to $50 million, respectively, and total operating expenses are expected to be $15 to $17 million and $60 to $64 million, respectively.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Thursday, March 7, 2019.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 8252397
Domestic Dial-in Number: 855-251-6663
International Dial-in Number: 281-542-4259
Live Webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.

On March 7, 2019 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that the first patient has successfully been dosed in its phase I study of ATOR-1015, its drug candidate in development for tumor-directed immunotherapy (Press release, Alligator Bioscience, MAR 7, 2019, View Source [SID1234538669]). ATOR-1015 is designed with properties that shall enable it to accumulate in the tumor area after an intravenous injection, and selectively exert its effect there.

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The study, being initiated in five different clinics across Sweden and Denmark, is a first-in-human dose-escalation study in up to 53 patients with advanced solid tumor disease. The primary aim of the study is to investigate the safety and tolerability of the drug and to identify the recommended dose for subsequent phase II studies. The results of the study are expected to read out in the second half of 2020.

"I state with satisfaction that ATOR-1015 is the first investigational tumor-localizing bispecific CTLA-4 antibody ever being tested in the clinic. With this, Alligator takes the lead in a very hot area of research. While immune activation through CTLA-4 has shown impressive efficacy in multiple cancers, it is coupled with severe toxicity. We believe that ATOR-1015 will be at least as effective as the approved monospecific CTLA-4 antibody Yervoy, and with less side effects," said Per Norlén, CEO of Alligator. "As the study progresses we look forward to learning more about the potential of this investigational medicine to improve the treatment of multiple cancers".

As previously communicated, Alligator has appointed Theradex Oncology, a global contract research organization with extensive expertise in oncology clinical development, to conduct the phase I study.

For further information, please contact:
Cecilia Hofvander, Director Investor Relations & Communications
Phone +46 46 540 82 06
E-mail: [email protected]

This information is such information as Alligator Bioscience AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 11:45 a.m. CET on March 7, 2019.

About ATOR-1015
ATOR-1015 is a next generation CTLA-4 bispecific antibody developed for tumor-directed immunotherapy with increased capability of regulatory T-cell depletion. It is wholly-owned by Alligator. ATOR-1015 binds to two different immune receptors: the checkpoint receptor CTLA-4 and the co-stimulatory receptor OX40. The immune activation is increased in areas where both target molecules are expressed at high levels, notably in the tumor microenvironment, which is believed to reduce adverse immune reactions.

About Yervoy
The active ingredient in Yervoy is ipilimumab, a protein that helps the body’s immune system to attack and destroy cancer cells. Yervoy is approved for the treatment of advanced melanoma (a type of skin cancer), and for combination treatment of advanced renal cell cancer (RCC) and colorectal cancer (MSIhigh CRC). The global total sales of Yervoy amounted to USD 1.2 billion in 2017. (Source: Fass.se, Cowen Therapeutics Outlook March 2018.)

On March 7, 2019 Quanterix Corporation (NASDAQ:QTRX), a company digitizing biomarker analysis to advance the science of precision health, reported financial results for the three months and twelve months ended Dec. 31, 2018 (Press release, Quanterix, MAR 7, 2019, View Source [SID1234534085]).

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"This past quarter has been highly productive, capping off a strong year of accelerating growth, achieving all our key milestones, and positioning us very well for 2019," said Kevin Hrusovsky, Chief Executive Officer, President and Chairman, Quanterix. "Our Simoa technology is at the forefront of the biomarker revolution, which has gained considerable traction as biomarkers are increasingly being recognized for their promise to transform treatment options by accelerating the development of more effective and safer drugs for all disease categories, and monitoring patient response to treatment therapies to ensure their efficacy and safety. Longer term, we see the promise of Simoa biomarker assays being deployed as health screens to see disease very early in the disease cascade when treatments are most effective. We have been impressed with the acceleration of third-party, peer-reviewed publications and the resulting body of evidence showing the promise for Simoa blood based biomarker assays enabling early ‘pre-symptomatic’ detection of neurological diseases and cancers."

Fourth Quarter 2018 Financial Highlights
Key financial results for the fourth quarter are shown below:

Q4 revenue of $10.9M versus prior year Q4 of $6.6M, an increase of 65%.
Q4 product revenue was $7.5M versus prior year Q4 of $4.1M, an increase of 83%.
Q4 Service and Other revenue totaled $3.4M versus prior year Q4 of $2.2M, an increase of 52%.
Q4 gross margin percent totaled 48.2%, a 450 basis point improvement over the 43.7% in Q4 2017.
YTD 2018 Financial Highlights
Key financial results for 2018 YTD are shown below:

YTD revenue of $37.6M versus prior year $22.9M, an increase of 65%.
YTD product revenue of $23.4M versus prior year $14.1M, an increase of 65%.
YTD Service and Other revenue of $12.1M versus prior year $7.7M, an increase of 58%.
YTD Collaboration revenue of $2.15M versus prior year $1.07M, an increase of 100%.
YTD gross margin percent totaled 47.8%, a 410 basis point improvement over the 43.7% for full year 2017.
Excluding a one-time collaboration revenue item of $1.1M, total YTD revenue growth would have been 60%. See "Non-GAAP Financial Measures" below.

Fourth Quarter and Full Year 2018 Business Highlights

Gained unrestricted rights back for its Simoa technology in IVD markets with the termination of a license agreement with bioMérieux.
Third-party, peer-reviewed publications continued to increase with more than 40 new publications featuring Simoa technology in Q4 alone, and >200 during the full year, bringing the total to over 400.
Acquired Aushon BioSystems and successfully integrated the two companies. The acquisition included a CLIA certified laboratory, expanding services and accelerating entry into pharmaceutical drug trial services, as well as access to novel immunoassay technology to which Simoa sensitivity algorithms are being applied to deliver next generation Simoa capabilities.
Adoption of proprietary Neurofilament light chain (NfL) assay, a biomarker previously measured only in cerebral spinal fluid (CSF), but by using Simoa, researchers have shown it can be measured in blood and correlated to CSF results. This advance is enabling novel applications critical for advancing early detection, treatment and prevention of neurological diseases, including multiple sclerosis (MS); Parkinson’s disease; Alzheimer’s disease; brain cancer; and traumatic brain injuries (TBIs).
Invited by the FDA and presented on progress and the potential future for the NfL biomarker to agency officials.
Launched a test bed, early access program for the SP-X Imaging and Analysis System with a 10-plex Simoa cytokine panel for oncology research and drug development.
Increased employee headcount from 126 at Dec. 31, 2017 to 177 at Dec. 31, 2018 (>40%) and expanded the leadership team with the recent hiring of Jackson Streeter, M.D. to lead corporate development and strategy, formerly chief medical officer and chief executive officer of Banyan Biomarkers; and Mary-Ellen Cortizas to lead the Accelerator Lab, formerly founder and chief operating officer of Claritas Genomics.
Added to the Nasdaq Biotechnology Index (NBI) as part of the annual re-ranking, and attracted additional analyst coverage.
Participated in first EU Powering Precision Health Summit, which was a highly productive meeting of leading researchers, key opinion leaders, and other stakeholders in precision health.
Reinforced the Company’s digital biomarker position through presentations at PULSE The Atlantic Summit on Health Care, The Lake Nona Impact Forum, and MedCity Converge, as well as the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress, where the Company had a record number of posters/presentations mentioning the use of Quanterix’ serum NfL assay for a wide range of clinical studies in MS disease progression and monitoring.
Conference Call
In conjunction with this announcement, Quanterix Corporation will host a conference call on March 7, 2019, at 4:30 p.m., EST to discuss the Company’s financial results and business outlook. To access this call, dial (833) 686-9351 for domestic callers, or (612) 979-9890 for international callers. Please reference the following conference ID: 3288482.

A live webcast will be accessible on the Investors section of Quanterix’ website: View Source The webcast will be available on the Company’s website for one year following completion of the call.

On March 7, 2019 BioTime, Inc. (NYSE American and TASE: BTX), a clinical-stage biotechnology company focused on degenerative diseases, reported that it will report its fourth quarter and full year 2018 financial and operating results on Thursday, March 14th, 2019, following the close of the U.S. financial markets (Press release, BioTime, MAR 7, 2019, View Source;p=RssLanding&cat=news&id=2390475 [SID1234534111]). BioTime management will also host a conference call and webcast on Thursday, March 14th, 2019, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its fourth quarter and full year 2018 financial results and to provide a business update.

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Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and should request the "BioTime Inc. Call". A live webcast of the conference call will be available online in the Investors section of BioTime’s website. A replay of the webcast will be available on BioTime’s website for 30 days and a telephone replay will be available through March 21st, 2019, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and entering conference ID number 1091719.