On March 5, 2018 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, 2017 (Press release, Syndax, MAR 5, 2018, View Source [SID1234524398]). In addition, the Company provided a clinical and business update. As of December 31, 2017, Syndax had $133.2 million in cash, cash equivalents and short-term investments.

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"In 2017, we continued to advance our pipeline of potentially transformative best-in-class candidates for the treatment of various cancers. We made significant progress on the clinical development of both our lead product candidate, entinostat, and SNDX-6352, our monoclonal antibody that blocks the colony stimulating factor 1 receptor. We also expanded our pipeline with the addition of a portfolio of preclinical, orally-available small molecule Menin-MLL inhibitors," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "We look forward to several key data readouts in the next six months, including progression free survival data from the Phase 3 E2112 trial of entinostat in combination with exemestane for advanced HR+, HER2- breast cancer. We also anticipate results next quarter from the PD-(L)1 refractory melanoma and NSCLC cohorts of ENCORE 601, as well as initial data and a decision on whether to advance to the second stage of the ENCORE 601 cohort of patients with microsatellite stable colorectal cancer."

Pipeline Updates

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The Phase 3 registration trial of entinostat plus exemestane in advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer, E2112, is 89% enrolled as of the end of February. ECOG-ACRIN Cancer Research Group, the trial sponsor, has notified the Company that the Data Safety Monitoring Committee (DSMC) completed the final progression free survival analysis and the first interim analysis for overall survival in November 2017. The results of this analysis are held confidentially by the ECOG-ACRIN study statistician and the DSMC, and ECOG-ACRIN will release the analysis to the Company upon completion of enrollment. The Company now anticipates enrollment to be complete in the third quarter of 2018.

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In November 2017, the Company presented data at the SITC (Free SITC Whitepaper) Annual Meeting from the melanoma and non-small cell lung cancer (NSCLC) cohorts of ENCORE 601 that support entinostat’s potential to enhance immune checkpoint blockade mediated anti-tumor responses in a broad range of tumors.

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In the second quarter of 2018, the Company expects to present full Phase 2 data from the PD-(L)1 refractory melanoma cohort of ENCORE 601. At that time, the Company also plans to communicate a registration strategy for entinostat in this indication.

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Full Phase 2 data from the PD-(L)1 refractory non-small cell lung cancer (NSCLC) cohort of ENCORE 601 are expected in the second quarter of 2018.

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Enrollment in the first stage of the ENCORE 601 cohort of patients with microsatellite stable colorectal cancer (MSS-CRC) is complete. The Company expects to share initial data, as well as a decision on whether to advance this cohort to the second stage of the trial, in the second quarter of 2018.

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ENCORE 602, the Phase 1b/2 clinical trial evaluating the combination of entinostat plus Genentech’s PD-(L)1 inhibitor atezolizumab (TECENTRIQ) in patients with triple negative breast cancer, is expected to complete enrollment of the Phase 2 portion in the second quarter of 2018. Topline results from this trial are anticipated in the second half of 2018.

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In January, the Company announced a new clinical collaboration with Genentech to evaluate the combination of entinostat and TECENTRIQ in patients with second-line HR+, HER2- metastatic breast cancer.

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ENCORE 603, the Phase 1b/2 clinical trial evaluating entinostat in combination with Pfizer/Merck KGaA’s BAVENCIO in patients with ovarian cancer, continues to enroll patients into the Phase 2 portion and is on track to complete enrollment in the second quarter of 2018. Topline results are expected in the first half of 2019.

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Dosing of patients with solid tumors in the Phase 1 multiple ascending dose (MAD) clinical trial of SNDX-6352 is ongoing. The Company anticipates presenting data from this trial and disclosing a Phase 2 strategy in the second half of 2018.

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In February, the Company entered into a clinical collaboration with AstraZeneca to evaluate the efficacy and safety of SNDX-6352 in combination with durvalumab (IMFINZI), AstraZeneca’s human monoclonal antibody directed against PD-(L)1, in multiple solid tumors. Initial work focusing on establishing the safety of this combination is expected to begin in the second quarter of 2018.

Development of the Company’s portfolio of Menin-Mixed Lineage Leukemia (MLL) inhibitors, in-licensed from Vitae Pharmaceuticals, Inc., a subsidiary of Allergan plc, is ongoing. An abstract describing the preclinical efficacy of these therapies in the treatment of MLL-rearranged acute myeloid leukemia (AML) was accepted for oral presentation at the 2018 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting being held April 14-18 in Chicago. The Company expects to initiate clinical trials in 2019.

Fourth Quarter 2017 Financial Results

As of December 31, 2017, Syndax had cash, cash equivalents and short-term and long-term investments of $133.2 million and 24,390,033 shares issued and outstanding.

License fee revenue increased to $1.2 million in the fourth quarter 2017 from $0.3 million for the comparable period in the prior year. License fee revenue for the year ended December 31, 2017 increased to $2.1 million compared to $1.2 million for the prior year. The increases are due to the ratable recognition of a $5.0 million milestone payment from KHK for the achievement of a development milestone.

Fourth quarter 2017 research and development expenses increased to $16.6 million from $8.5 million for the comparable period in the prior year. Research and development expenses for the year ended December 31, 2017 increased to $48.2 million compared to $31.7 million for the prior year. The increases were primarily due to increased clinical trial activities related to our Phase 1 clinical pharmacology trials, increased enrollment in ENCORE 601, costs related to SNDX-6352 trials, increased activities in ENCORE 602 and ENCORE 603, and CMC activities, increased headcount, legal and consultant expenses, facility costs and travel costs. In 2017 Syndax expensed a nonrefundable upfront payment of $5.0 million to Allergan for the Menin Assets and in 2016 Syndax expensed a nonrefundable upfront payment of $5.0 million related to the UCB license agreement.

General and administrative expenses totaled $4.1 million during the fourth quarter of 2017 and $15.9 million for the year, similar to the $3.0 million and $13.3 million expense level for the respective prior year periods. The increase in general and administrative expenses was primarily due to increases in headcount, increases in pre-commercialization work, professional fees and other costs related to being a public company. These increases were partially offset by decreases in legal expenses.

For the three months ended December 31, 2017, Syndax reported a net loss attributable to common stockholders of $19.1 million or $0.80 per share compared to $10.8 million or $0.59 per share for the comparable prior year period. For the year ended December 31, 2017, Syndax reported a net loss attributable to common stockholders of $60.1 million or $2.90 per share, compared to $47.1 million or $3.22 per share for the prior year period.

Financial Guidance

Today the Company provided operating expense guidance for the first quarter and full year 2018. For the first quarter and full year 2018, research and development expenses are expected to be $18 to $22 million and $67 to $76 million, respectively, and total operating expenses are expected to be $22 to $26 million and $86 to $96 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, Monday, March 5, 2018.

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: 5589398
Domestic Dial-in Number: 1-855-251-6663
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 5, 2018 VistaGen Therapeutics Inc. (NASDAQ: VTGN), a clinical-stage biopharmaceutical company developing new generation medicines for depression and other central nervous system (CNS) disorders, reported that Shawn Singh, Chief Executive Officer, will present at Oppenheimer’s 28th Annual Healthcare Conference in New York City at 8:00 a.m. Eastern Time on Wednesday, March 21, 2018 (Press release, VistaGen Therapeutics, MAR 5, 2018, View Source [SID1234524424]).

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For more information about the conference, or to schedule a one-on-one meeting with VistaGen’s management, please contact your Oppenheimer representative directly, or visit the conference website: View Source

On March 5, 2018 MyBiotics Pharma Ltd., a microbiome therapeutics company, reported that it has entered into a second option agreement with Ferring Holding Ltd. for the validation of MyBiotics’ microbiome-based therapeutics in the field of women’s health (Press release, MyBiotics, MAR 5, 2018, View Source [SID1234577656]). This agreement follows the first agreement between the two parties signed in June 2017, for the validation of MyBiotics’ technology in the treatment of a non-disclosed gastroenterology condition.

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MyBiotics has developed breakthrough and robust production and fermentation techniques for growing a highly stable and diverse bacterial community that can be delivered to the gut efficiently and can reliably restore microbiome equilibrium. The technology is highly potent and suitable for patients with medical indications related to the microbiome or for those who use antibiotics. Preclinical tests have shown that MyBiotics’ products deliver enhanced durability in various gastrointestinal and manufacturing conditions, enable targeted release in different gastrointestinal locations and exhibit robust colonization in the gut, with a stable bacterial population observed for 14 days following treatment.

"Within a few months we have entered into two collaborations with Ferring Holding, which is a true vote of confidence in the potential of our therapeutic approach", stated David Daboush, CEO of MyBiotics Pharma. "Our technology enables us to generate concentrated and effective bacterial raw material that can be introduced into the gastrointestinal tract and restore ecological equilibrium to the gut microbiota. Combining our innovative technology with the extensive drug development and marketing expertise of the Ferring Group has the potential to lead to novel treatments in the fields of gastroenterology and women’s health, offering patients long term relief without significant risks."

"This agreement is further evidence of Ferring’s commitment to seek out novel treatments for important medical conditions. Ferring is very active in the areas of gastroenterology and women’s health, and this partnership with MyBiotics is another indication of our commitment to discover and develop innovative microbiome-based treatments," said Dov Kanner, Director of Ferring Holding. "It is clear by now that the microbiome is of profound importance to a wide variety of pathologies, ranging from digestive disorders through obesity, immune disorders, women’s health and more. Microbiome-based therapies are gaining increasing interest for a variety of microbiome-related indications, and we believe that MyBiotics’ technology will enable the development of such therapies, potentially offering treatment to millions of people around the globe."

Currently available probiotic drugs and dietary supplements have limited clinical impact due to the low survival rates of the delivered bacteria in the gastrointestinal tract. This significantly reduces the ability of probiotic bacteria to impact the microbial diversity of the gut, thus failing to create a healthier community of bacteria.

On March 5, 2018 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that the company is scheduled to present at the Cowen 38th Annual Healthcare Conference in Boston on Monday, March 12, 2018 at 11:20 a.m. ET (Press release, Agios Pharmaceuticals, MAR 5, 2018, http://investor.agios.com/news-releases/news-release-details/agios-present-cowen-38th-annual-healthcare-conference-monday [SID1234524364]).

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A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

On March 5, 2018 Anthera Pharmaceuticals, Inc. (Nasdaq: ANTH) reported a business update and financial results for the fourth quarter and fiscal year ending December 31, 2017(Press release, Anthera, MAR 5, 2018, View Source [SID1234524381]).

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Recent Developments and Business Highlights:

Sollpura (liprotamase) for the treatment of Exocrine Pancreatic Insufficiency ("EPI")

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Phase 3 RESULT study completed patient dosing, top line data expected March 2018

The RESULT study of Sollpura in patients with EPI caused by cystic fibrosis completed dosing for the primary treatment period on February 2, 2018. The study was initiated in May 2017 and enrolled 140 patients in the United States, Europe and Israel. The primary efficacy variable will evaluate the change from baseline in coefficient of fat absorption ("CFA") following 4 weeks of treatment with either Sollpura or Pancreaze. Patients randomized to Sollpura will then be followed for an additional 20-week extension period (total of 24 weeks on study) for longer term assessments of weight, height, BMI, and safety. Top line data will include the major primary and secondary outcome measures based on 4 weeks of comparative treatment and is expected in March.

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RESULT study reported positive interim futility analyses

In December 2017 and January 2018, we reported positive outcomes of two prespecified, sequential, and separately conducted interim futility analyses for the RESULT study after approximately 25% and 50% of patients, respectively, had completed the primary treatment period. Both analyses were conducted by RESULT’s Data Monitoring Committee which is comprised of experts appointed by the Cystic Fibrosis Foundation’s Therapeutics Development Network.

Management Update

On January 1, 2018, we strengthened our executive management team through the appointment of Patrick Murphy as our Senior Vice President, Manufacturing. In this role, Mr. Murphy will oversee the manufacturing and commercial scale-up of Sollpura.

Financing Update

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Private Placement

In October 2017, we completed the first of two closings of a private placement with net proceeds of $2.2 million. In January 2018 the second closing yielded additional net proceeds of $11.1 million.

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Warrant Exercise and Sale of Stock Pursuant to and Equity Purchase Agreement

Subsequent to December 31, 2017, we received aggregate net proceeds of $3.1 million from the issuance of common stock pursuant to warrant exercises and the sale of common stock pursuant to an equity purchase agreement.

NASDAQ Compliance Update

As of February 28, 2018, we met the market capitalization requirement of at least $35 million for ten consecutive trading days for continued listing on the Nasdaq Capital Market. A formal compliance determination is pending from the Nasdaq Stock Market LLC.

Summary of Financial Results:

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Cash Position. We ended the fourth quarter of 2017 with cash and cash equivalents of $2.2 million. Our cash position was subsequently strengthened by additional net proceeds of $11. 1 million from the second closing of the PIPE transaction and $3.1 million from the exercise of warrants and sale of shares pursuant to an equity purchase agreement. For the year ended December 31, 2017, our cash used for operating activities was $36.9 million, compared to $48.9 million for fiscal year 2016. The significant decrease of $12 million in cash used for operating activities was mainly attributable to lower operating expenses as we concentrated our clinical development effort primarily on Sollpura in 2017. In 2016, our clinical development effort included both Sollpura and blisibimod.

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R&D Expense. Research and development expense for the three and twelve months ended December 31, 2017 totaled $7.7 million and $28.6 million, respectively, compared to $10.8 million and $46.5 million for the corresponding periods in 2016. The decrease in 2017 from 2016 was primarily due to lower clinical development expenses as a result of the SOLUTION study in cystic fibrosis patients with EPI and CHABLIS clinical studies in patients with systemic lupus erythematous being substantially completed in 2016. In addition, costs associated with the BRIGHT-SC study, the clinical evaluation of blisibimod in patients with IgA nephropathy, decreased in 2017 compared with 2016 as we completed treatment of the BRIGHT-SC study in August 2017. The change in clinical development activities between the comparative periods resulted in reductions in expenses by $2.9 million and $16.2 million for the three months and year ended December 31, 2017, respectively.

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G&A Expense. General and administrative expense for the three and twelve months ended December 31, 2017 totaled $1.6 million and $7.9 million, respectively, compared to $3.8 million and $11.1 million for the corresponding periods in 2016. The decrease is primarily due to a significant reduction in headcount, which resulted in lower payroll related and stock-based compensation expense by $2.1 million and $3.4 million for the three months and twelve months ended December 31, 2017, respectively.

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Research Award. A research award, granted to us in March 2015 by the Cystic Fibrosis Foundation and recorded as an offset to operating expense, totaled $100,000 for the year ended December 31, 2017, compared to $261,000 in 2016. The amount of the research award recognized represents the value prescribed to the milestones we achieved under the award agreement during the reporting periods. As of March 31, 2017, we had fully recognized the research award.

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Other Income (Expense). For the three and twelve months ended December 31, 2017, we recorded non-operating income (expense) of $(0.4) million and $9.6 million, respectively. For the three and twelve months ended December 31, 2016, non-operating income of $1.6 million and $1.7 million was recorded, respectively. Non-operating income (expense) is comprised primarily of changes in the fair value of warrants issued in connection with our equity offerings in 2016 and 2017, which are accounted for as derivative liabilities, with the change in fair value recorded as part of other income (expense). The number of shares of common stock underlying the warrants issued in September 2016 became fixed in November 2016 and the related fair value was reclassified from liability to stockholders’ equity in 2016. The warrants issued in March 2017 will continue to be accounted for as derivative liability until the warrants are exercised or expired.

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Net Loss Applicable to Common Stockholders. In connection with a registered direct offering of convertible preferred stock and warrants in September 2016, there was an in-the-money conversion feature (beneficial conversion feature, or BCF). The BCF required separate financial statement recognition and was recorded as a discount to the preferred shares. No deemed dividend was recorded for the three months ended December 31, 2017 and $2.1 million in deemed dividend was recorded for the corresponding period in 2016. For the twelve months ended December 31, 2017 and 2016, we recorded a deemed dividend of $2.5 million and $10.9 million, respectively.

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Net Loss Per Basic and Diluted Share. For the three and twelve months ended December 31, 2017, we recorded a net loss of $0.73 and $2.86 per basic and diluted share, respectively, compared to net loss of $2.84 and $12.87 per basic and diluted share, respectively, in the corresponding periods in 2016. The decrease in net loss per basic and diluted share is primarily driven by reduced operating expense in 2017 as compared to 2016.