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Syndax Pharmaceuticals Reports Fourth Quarter 2020 Financial Results and Provides Clinical and Business Update

On March 8, 2021 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, 2020 (Press release, Syndax, MAR 8, 2021, View Source [SID1234576206]). In addition, the Company provided a clinical and business update.

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"We expect 2021 will be a year of immense progress across our two highly promising programs aimed at addressing key areas of unmet need, coupled with a sharp focus on pipeline expansion," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Notably, we plan to present data from the Phase 1 portion of our ongoing AUGMENT-101 trial of SNDX-5613, our selective menin inhibitor, in patients with acute leukemias. We presented promising initial clinical data at the 2020 AACR (Free AACR Whitepaper) Annual Meeting that provided the first clinical evidence that disrupting the interaction between menin and MLL1 can induce rapid responses in difficult to treat acute leukemias. We look forward to sharing updated results which further expand on SNDX-5613’s potential to meaningfully alter the treatment paradigm in genetically-defined acute leukemias."

"Additionally, on the heels of positive data presented at the ASH (Free ASH Whitepaper) Annual Meeting in December from our Phase 1 trial of axatilimab, our anti-CSF-1R monoclonal antibody, in patients with cGVHD, we are pleased to announce that our pivotal Phase 2 AGAVE-101 trial is now underway. Through inhibition of monocyte derived macrophages, axatilimab has demonstrated important clinical benefits in multiple organ systems. We believe axatilimab could represent a meaningful therapeutic option for additional fibrotic diseases where macrophages have been shown to play a significant role, and we are actively exploring additional indications."

Recent Progress and Anticipated Milestones

SNDX-5613

Syndax plans to share data from the Phase 1 AUGMENT-101 trial of SNDX-5613, its highly selective, oral menin inhibitor, in patients with mixed lineage leukemia rearranged (MLL-r) and nucleophosmin (NPM1) mutant acute leukemias late in the first quarter or early in the second quarter of 2021. Additional details regarding the presentation, which will feature the trial’s principal investigator, Eytan M. Stein, M.D. Assistant Attending Physician and Director, Program for Drug Development in Leukemia, Department of Medicine at Memorial Sloan Kettering Cancer Center, will be announced in the coming weeks. The Company remains on track to initiate the Phase 2 portion of the trial in the second quarter of 2021, which could potentially serve as the basis for a regulatory filing.

Axatilimab

At the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December 2020, Syndax reported updated data from its Phase 1 trial of axatilimab, its anti-CSF-1R monoclonal antibody, in patients with chronic graft versus host disease (cGVHD). Data demonstrated deep, durable responses and multiorgan clinical benefit in patients refractory to multiple therapeutic agents.

The Company announced today that the pivotal Phase 2 AGAVE-201 trial, which will evaluate the safety and efficacy of three doses and schedules of axatilimab in patients with cGVHD, began enrolling patients earlier this quarter. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for GVHD, with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score. The Company expects to report topline data in 2023.

Fourth Quarter 2020 Financial Results

As of December 31, 2020, Syndax had cash, cash equivalents and short-term investments of $293.1 million and 51.4 million shares and share equivalents issued and outstanding. This includes 3.6 million pre-funded warrants.

Fourth quarter 2020 research and development expenses increased to $15.5 million from $9.5 million, and for the full year increased to $50.4 million compared to $43.0 million for 2019. The fourth quarter and full year increases were primarily due to increased clinical trial activities, increased CMC activities and professional fees.

General and administrative expenses for the fourth quarter 2020 decreased to $4.7 million from $5.1 million, and, for the year ended December 31, 2020, increased to $22.5 million compared to $16.1 million for the prior year. The fourth quarter decrease is primarily due to decreased pre-commercialization expenses. The increase for the full year was primarily due to increased professional fees and employee related expenses.

For the three months ended December 31, 2020, Syndax reported a net loss attributable to common stockholders of $20.4 million or $0.44 per share compared to $14.0 million or $0.44 per share for the prior year period. For the year ended December 31, 2020, Syndax reported a net loss attributable to common stockholders of $77.8 million or $1.88 per share, compared to $56.0 million or $1.84 per share for the prior year. For the year ended December 31, 2020, this includes a deemed dividend of $3.9 million.

Financial Update and Guidance

In December 2020, Syndax issued 6,250,000 shares of its common stock at $23.00 per share. As a result of the offering, Syndax received gross proceeds of approximately $143.8 million.

For the first quarter of 2021, research and development expenses are expected to be $25 to $30 million, and total operating expenses are expected to be $30 to $35 million. For the full year of 2021, research and development expenses are expected to be $90 to $100 million, and total operating expenses are expected to be $110 to $120 million.

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 8, 2021.

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 followin

CLEAVE THERAPEUTICS LICENSES FIRST-IN-CLASS VCP/P97 INHIBITOR
CB-5339 TO CASI PHARMACEUTICALS FOR GREATER CHINA REGION

On March 8, 2021 Cleave Therapeutics, Inc. ("Cleave"), a clinical-stage biopharmaceutical company focused on valosin-containing protein (VCP)/p97 as a novel target in protein homeostasis, DNA damage response and other cellular stress pathways for therapeutic use in cancer, and CASI Pharmaceuticals, Inc (NASDAQ: CASI), a U.S. biopharmaceutical company with an established clinical development and commercial infrastructure in China, reported they have entered an exclusive licensing agreement for the development and commercialization of CB-5339, a novel VCP/p97 inhibitor, in mainland China, Taiwan, Hong Kong and Macau (Press release, CASI Pharmaceuticals, MAR 8, 2021, View Source [SID1234576205]).

Under the terms of the agreement, Cleave and CASI will develop CB-5339 in both hematological malignancies and solid tumors, with CASI responsible for development and commercialization in China and associated markets. Cleave will receive a $5.5 million upfront payment and is eligible to receive up to $74 million in development and commercial milestone payments plus tiered royalties in the high-single to mid-double-digit range on net sales of CB-5339. In addition to the upfront cash payment, CASI will make a $5.5 million investment in Cleave through a convertible note.

CB-5339, an oral second-generation, small molecule VCP/p97 inhibitor, is being evaluated in a Phase 1 clinical trial in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), while the National Cancer Institute (NCI) is sponsoring and evaluating CB-5339 in a Phase 1 clinical trial of patients with solid tumors and lymphomas.

"Our collaboration with CASI is a strategic step to accelerate the development of CB-5339 globally by initiating trials for additional indications such as multiple myeloma in Greater China," said Amy Burroughs, President and CEO of Cleave Therapeutics. "CASI’s development and commercial capabilities in hematology oncology and long-term commitment as an investor make them an ideal partner at this exciting time in the development of our first-in-class drug candidate."

Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, said: "Cleave’s novel approach to inhibiting VCP/p97 in hematological malignancies such as AML and MDS is supported by extensive preclinical research and early clinical data. CB-5339 represents a promising new agent for selectively targeting VCP/p97 in cancers and is a complementary addition to our growing portfolio of approved and investigational therapies for hematology oncology. We are thrilled to partner with Cleave as CB-5339 advances through clinical development."

Axial Therapeutics Promotes A. Stewart Campbell to Chief Executive Officer

On March 8, 2021 Axial Therapeutics Inc., a clinical-stage biopharmaceutical company focused on leveraging the gut-brain axis for the discovery and development of small molecule therapeutics with an initial focus on autism spectrum disorder (ASD) and Parkinson’s disease (PD), reported the appointment of A. Stewart Campbell, Ph.D. as Chief Executive Officer (CEO) (Press release, Axial Biotherapeutics, MAR 8, 2021, View Source [SID1234576204]). Dr. Campbell will also serve as a member on the Axial Board of Directors.

"Stew is a proven executive leader with extensive experience in R&D innovation. His expertise across the drug development lifecycle, combined with his experience building results-focused teams makes him the right leader to take Axial forward," said Ronald C. Renaud, Axial Board Chair and Chief Executive Officer, Translate BIO. "His focus on bringing new therapies to patients will be critically important as he spearheads Axial’s efforts to advance new gut-targeted small molecule treatments for serious central nervous system (CNS) diseases and conditions."

Dr. Campbell is an accomplished executive with more than 25 years of experience building and leading teams that have strong track records of discovering and developing novel therapeutics, including numerous innovative product candidates that have progressed to the clinic. He is co-inventor on 20 issued patents.

He joined Axial in 2016 and has led the company’s research and development efforts, most recently serving as Senior Vice President, R&D. Prior to joining Axial, Dr. Campbell held various leadership roles at biopharmaceutical and chemical manufacturing companies including Ancora/CordenPharma, Surface Logix and Insmed Inc. He led the acquisition of Ancora by CordenPharma and subsequently served as Head of Product Management and Business Development for Carbohydrates and Lipids at CordenPharma. While at Surface Logix and Insmed, Dr. Campbell played an integral role in the discovery or development of five clinical stage drug candidates, including belumosudil and TAK-607. He holds a Ph.D. in Organic Chemistry from Queen’s University (Canada) and did post-doctoral research at Duke University.

"Axial is well-positioned for future growth with deep expertise in the gut-brain axis and cutting-edge science leading to the discovery of new drug targets and the development of safe and effective first-in-class therapies for CNS diseases and conditions, as well as oncology," said Dr. Campbell. "I look forward to working together with the leadership, the Board, and the dedicated Axial team to expand our pioneering drug discovery approach and advance our product candidates through the clinic."

Dr. Campbell succeeds David H. Donabedian, Ph.D., who served as CEO of Axial since co-founding the company in 2016. "We thank David for his commitment and tireless effort over the past four years, taking Axial from an idea to a company poised to enter Phase 2B in autism and with a robust pipeline of innovative programs in Parkinson’s disease and oncology," said Dr. Campbell.

Axial’s unique focus on targeting the gut as a means to treat CNS diseases and conditions is rooted in the groundbreaking research of its co-founder Dr. Sarkis Mazmanian at the California Institute of Technology establishing the biological link between the gut-brain axis and neurological disease.

The foundation of Axial’s approach is its innovative platform that leverages its proprietary in vivo models and "omics" capabilities as the scientific underpinning for the identification and validation of new disease targets followed by the design and delivery of gut-restricted, small molecule therapeutics. The company’s platform has significant potential for the development of novel therapies that mitigate underlying disease pathology, resulting symptoms, and disease progression in neurological diseases as well as oncology.

Axial’s lead product candidate is AB-2004, an oral compound poised to redefine the treatment of irritability in children with ASD, improving the lives of affected children and their families. In a Phase 1B/2A study, AB-2004 was shown to reduce metabolites implicated in ASD and improve key behaviors including irritability and anxiety. AB-2004 had no drug-related adverse events consistent with the large body of existing human safety data.

The company’s pipeline also includes small molecule, microbiome-inspired therapies in preclinical development for treatment of the underlying disease burden in PD.

Applied DNA to Participate in Upcoming Conferences

On March 8, 2021 Applied DNA Sciences, Inc. (NASDAQ: APDN) ("Applied DNA" or the "Company"), a leader in Polymerase Chain Reaction (PCR)-based DNA manufacturing, reported that it will be participating in the following virtual investor conferences (Press release, Applied DNA Sciences, MAR 8, 2021, View Source [SID1234576203]).

March 9 – 10, 2021 – Dr. James A. Hayward, president and CEO, will present a company overview at the H.C. Wainwright Global Life Sciences Virtual Conference. Dr. Hayward and Beth Jantzen, chief financial officer, will also hold virtual investor meetings. Dr. Hayward’s pre-recorded presentation will be available for viewing beginning March 9 at 7:00 A.M. (ET). The presentation will also be available on Applied DNA’s website (link);
March 15 – 17, 2021 – Dr. Hayward will participate on a virtual panel at Roth Capital Markets’ 33rd Annual Conference titled ‘Therapeutics Against SARS-CoV-2 – Viral variants Will Not Fade Away’. The panel is scheduled for March 15, 2021 at 12:00pm ET and will be available to all Conference attendees, as well as on Applied DNA’s website (link). Management will also hold virtual investor meetings;
March 17 – 19, 2021 – Dr. Hayward will present at the Inaugural Emerging Growth Virtual Conference presented by M Vest LLC and Maxim Group LLC. The presentation will be available to all registered participants of the conference (link) on March 17th – 19th and will be posted on Applied DNA’s website (link) after March 19.

IMV to Announce Fourth Quarter and Fiscal 2020 Financial and Operational Results and Host Conference Call and Webcast on March 17, 2021

On March 8, 2021 IMV Inc. ("IMV" or the "Corporation") (Nasdaq: IMV; TSX: IMV), a clinical-stage biopharmaceutical company pioneering a novel class of immunotherapies, reported that it will hold a conference call and webcast on Wednesday, March 17, 2021 at 8:00 a.m. Eastern Time to discuss the Company’s 2020 fourth quarter and full year financial and operational results (Press release, IMV, MAR 8, 2021, View Source [SID1234576202]).

Financial analysts are invited to join the conference call by dialing (866) 211-3204 (U.S. and Canada) or (647) 689-6600 (international) using the conference ID: 8998652.

Other interested parties will be able to access the live audio webcast at this link: View Source The webcast will be recorded and will then be available on the IMV website for 30 days following the call.