On March 7, 2019 Paige.AI, the leading start-up in computational pathology focused on building artificial intelligence (AI) that will transform the clinical diagnosis and treatment of cancer, reported that it has been granted Breakthrough Device designation by the U.S. Food and Drug Administration (FDA), the first such designation for AI in cancer diagnosis publicly announced by any company (Press release, Paige AI, MAR 7, 2019, View Source [SID1234534083]).

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"Paige.AI is focused on providing artificial intelligence tools to pathologists that will enable them to become faster and more accurate in their diagnosis and treatment recommendations for the care of cancer patients," said Dr. Leo Grady, Chief Executive Officer of Paige.AI. "We are thrilled to receive Breakthrough designation and look forward to collaborating with the FDA to bring our products to market, starting with prostate cancer and expanding from there."

The FDA’s Breakthrough Device designation is granted for technologies that have the potential to provide for more effective diagnosis or treatment for life-threatening or irreversibly debilitating diseases, where timely availability is in the best interest of patients because no approved alternative exists or because the technology offers significant advantages over existing approved alternatives. The Breakthrough Device program was created by the 21st Century Cures Act.

"We are honored to have been granted Breakthrough designation by the FDA, which underscores the groundbreaking nature of our technology as the leading clinical-grade AI in computational pathology to combine vast amounts of high-quality data with unique deep learning architectures in service of delivering better patient care," said Dr. Thomas Fuchs, Co-Founder of Paige.AI.

Paige.AI was launched in early 2018 based on technology developed by Fuchs and his colleagues and a license agreement with Memorial Sloan Kettering Cancer Center (MSK). MSK began digitizing its pathology slides four years ago. Under the license agreement, Paige.AI receives de-identified images of digitized slides – more than one million such slides to date — and is funding the digitization of an additional four million archive slides, which in total will create the largest digital pathology dataset. Paige.AI is working with this de-identified dataset to develop a comprehensive portfolio of AI products across cancer subtypes to serve the needs of pathologists around the world.

On March 7, 2019 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported that it will host a conference call on Thursday, March 14, 2019, at 8:00 a.m. ET to review its full year 2018 financial results and provide an update on the company (Press release, Infinity Pharmaceuticals, MAR 7, 2019, View Source [SID1234534109]).

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A live webcast of the conference call can be accessed in the Investors/Media section of Infinity’s website at www.infi.com. To participate in the conference call, please dial 1-877-316-5293 (domestic) and 1-631-291-4526 (international) five minutes prior to start time. The conference ID number is 3569646. An archived version of the webcast will be available on Infinity’s website for 30 days.

On March 7, 2019 GTx, Inc. (Nasdaq: GTXI) and Oncternal Therapeutics, Inc., a privately held clinical-stage biotechnology company developing potential first-in-class therapeutic candidates for cancers with critical unmet medical need, reported that they have entered into a definitive merger agreement under which the stockholders of Oncternal would become the majority owners of GTx’s outstanding common stock (Press release, GTx, MAR 7, 2019, View Source [SID1234534068]). The proposed merger will create a publicly-traded, clinical-stage oncology company.

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The combined company will be named Oncternal Therapeutics, Inc. and plans to change its ticker symbol on the Nasdaq Capital Market to ONCT upon closing of the transaction.

The combined company will have a strong balance sheet and deep pipeline of promising oncology drug programs advancing in development:

· Oncternal’s lead program, cirmtuzumab, is an investigational, potential first-in-class anti-ROR1 monoclonal antibody. Cirmtuzumab is currently in a Phase 1/ 2 clinical trial in combination with ibrutinib for the treatment of chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL). In addition, an investigator-initiated Phase 1 clinical trial of cirmtuzumab in combination with paclitaxel for women with metastatic breast cancer is being conducted at the University of California San Diego (UC San Diego).

·TK216, an investigational, potential first-in-class small molecule designed to inhibit the biological activity of ETS-family transcription factor oncoproteins, is being evaluated alone and in combination with vincristine in a Phase 1 clinical trial in patients with relapsed or refractory Ewing sarcoma.

·A ROR-1 targeted chimeric antigen receptor T-cell (CAR-T) program is in preclinical development at UC San Diego for hematologic and solid tumors.

·A Selective Androgen Receptor Degrader (SARD) program, an investigational, potential first-in-class preclinical program designed for oral administration to treat castration-resistant prostate cancer in men who are non-responsive to current androgen targeted therapies.

Cash, cash equivalents and short-term investments for the combined company are expected to be approximately $26 million, if the merger closes by the end of the second quarter of 2019. These funds are expected to be sufficient to advance Oncternal’s programs into the second quarter of 2020, including the Phase 2 study of cirmtuzumab and ibrutinib, and will fund the planned SARD preclinical studies to support the submission of an investigational new drug application with the U.S. Food and Drug Administration.

James Breitmeyer, MD, PhD, cofounder, president and CEO of Oncternal and a 30-year veteran of the pharmaceutical industry, will continue as president and CEO of the combined company. David Hale, cofounder of Oncternal and a 35-year veteran of numerous successful private and public biotech companies, will continue as Chairman of the Board of the combined company.

"This merger introduces Oncternal and its promising oncology pipeline to the public market and provides additional capital resources to advance our programs to potential value inflection points," said Dr. Breitmeyer. "In addition to clinical data expected from our cirmtuzumab and TK216 programs later this year and during the first half of 2020, we also plan to have preclinical results that get us ready for clinical testing of our ROR1 CAR-T program. The addition of GTx’s SARD technology strengthens our pipeline and augments our entire oncology franchise, which includes a range of therapeutic approaches for a variety of difficult to treat cancers."

"This transaction with Oncternal reflects the continued commitment of our management team and Board of Directors to deliver value to stockholders and make a difference in patients’ lives," said Robert J. Wills, PhD, Executive Chairman of GTx. "Following a thorough review of strategic alternatives, we have determined that a reverse merger with Oncternal will enable GTx investors to participate in Oncternal’s broader pipeline of oncology opportunities, including product candidates designed to address rare disease indications, and enable the continued development of our first-in-class SARD technology by a company whose leadership has deep experience in developing oncology medicines."

About the Proposed Merger

The merger is structured as a stock-for-stock transaction whereby all of Oncternal’s outstanding shares of common stock and securities convertible into or exercisable for Oncternal’s common stock will be converted into GTx common stock and securities convertible into or exercisable for GTx common stock. Immediately following the closing of the transaction, the former stockholders of Oncternal will hold approximately 75% of the outstanding shares of common stock of the combined company. In addition to retaining an ownership interest representing approximately 25% of the outstanding shares of common stock of the combined company, the GTx stockholders of record as of immediately prior to the effective time of the merger will receive non-transferable contingent value rights ("CVR") entitling the holders to receive in the

aggregate 50% of any net proceeds derived from the grant, sale or transfer of rights to SARD or selective androgen receptor modulator (SARM) technology during the term of the CVR and, if applicable, to receive royalties on the sale of any SARD products by the combined company during the term of the CVR. Under certain circumstances further described in the merger agreement, the exchange ratio of the outstanding shares of common stock of the combined company may be adjusted upward or downward based on cash levels of each of the companies at closing.

Upon closing of the transaction, GTx will be renamed Oncternal Therapeutics, Inc. and will be headquartered in San Diego, California under the leadership of Oncternal’s current management team. Although no GTx employee is expected to remain an employee of the combined company, the merger agreement provides that the Board of Directors of the combined company will be comprised of nine members, including seven designated Oncternal directors as well as Robert J. Wills, PhD and Michael G. Carter, MD, from GTx’s current Board. The combined company is expected to trade on The Nasdaq Capital Market under a new ticker symbol, ONCT. The merger agreement has been unanimously approved by the Board of Directors of each company. The transaction is expected to close in the second quarter of 2019, subject to approvals by stockholders of each company and other customary closing conditions.

Aquilo Partners, L.P. is acting as exclusive financial advisor to GTx on the proposed transaction and Cooley LLP serves as legal counsel to GTx. Piper Jaffray is acting as exclusive financial advisor to Oncternal on the proposed transaction and Latham & Watkins, LLP serves as legal counsel to Oncternal.

Conference Call Information

Dr. Wills and Dr. Breitmeyer will co-host a conference call to discuss the proposed merger on March 7, 2019, at 5:30 A.M. Pacific Time at Oncternal’s headquarters in San Diego.

To access the live conference call, please dial 1.877.407.2991 from the U.S. and Canada or 1.201.389.0925 internationally. A playback of the call will be available from approximately 12:00 P.M. Pacific Time today through May 7, 2019 and may be accessed by dialing 877.660.6853 from the U.S. and Canada or 201.612.7415 internationally, and using conference ID 13688553.

The conference call information will also be available on the Investor section of the GTx website at www.gtxinc.com.

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.