On March 3, 2022 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results and highlights for the fourth quarter and year ended December 31, 2021 (Press release, GlycoMimetics, MAR 3, 2022, View Source [SID1234609446]). Cash and cash equivalents at December 31, 2021 were $90.3 million.

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"During 2021, we focused on completing enrollment of our pivotal trial evaluating uproleselan in AML. Towards the end of the year, within two weeks of each other, GlycoMimetics completed enrollment of our Phase 3 trial and the NCI completed enrollment of the Phase 2 portion of its Phase 2/3 trial. This represents a significant achievement for our entire organization, particularly during a global pandemic that has negatively impacted enrollment rates for numerous clinical studies. The top-line data from these two trials of uproleselan in AML will undoubtedly be transformative for the organization. As the survival data matures, we are actively preparing for a potential regulatory submission and subsequent commercialization of uproleselan," commented Harout Semerjian, Chief Executive Officer.

Operational Highlights

Uproleselan

●GlycoMimetics’ pivotal Phase 3 trial in relapsed/refractory AML completed enrollment of 388 patients across 70 sites in the U.S., Australia and Europe. The Company reiterates its projection of a topline data release after year-end 2022.
●In parallel, the NCI-sponsored Phase 2/3 clinical trial, which is evaluating uproleselan in newly diagnosed older adults with AML who are fit for chemotherapy, completed its Phase 2 enrollment of 267 patients. The Company intends to share topline results from the NCI’s planned interim analysis of Phase 2 data based on event-free survival.
●Efficacy and safety data from a Phase 1/2 clinical study of uproleselan were published online September 16, 2021, in the journal BLOOD. An analysis of minimal residual disease (MRD) showed an MRD negative rate of 69 percent in patients with relapsed/refractory AML, indicating an enhanced depth of response following addition of uproleselan to salvage therapy.
●Investigator-sponsored clinical trials to evaluate expanded indications for uproleselan were initiated at the University of California-Davis, Washington University at St. Louis, MD Anderson Cancer Center and the University of Michigan.
●Apollomics, our exclusive collaborator for development and commercialization of uproleselan in Greater China, received Breakthrough Therapy designation from the China National Medical Products Administration and initiated a Phase 3 registrational study in November 2021.

GMI-1687

●In 2021, the Company advanced an IND-enabling program for GMI-1687, with sickle cell disease acute VOC as the lead indication. The Company reiterates its plan to submit an IND to the FDA in the first half of 2022.

GMI-1359

●At the 63rd Annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December, the Company’s collaborators at MD Anderson highlighted the potential for GMI-1359 in breaking resistance in AML, particularly in patients with FLT-3 ITD mutations. Subject to available funding, the Company plans to evaluate potential indications for further clinical development.

GMI-2093

●At the 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, preclinical data was provided for the first time, as a late-breaking abstract, on the impact of one of the Company’s galectin-3 inhibitors on tumor fibrosis, mononuclear cell infiltration, and antitumor activity in a pancreatic adenocarcinoma model when given in combination with an anti-PD-L1 inhibitor.
●In late 2021, the Company nominated GMI-2093 as a development lead candidate after observing high affinity and selectivity for galectin-3 with oral bioavailability. The role of galectin-3 in cancer, fibrosis and other inflammatory disease is emerging as a therapeutic target, and the Company plans to advance this program through potential strategic partnerships.
Executive Management Team

●The Company expanded its executive management in late 2021 and early 2022:
●Lisa DeLuca, Ph.D., was hired in November as Vice President, Regulatory Affairs
●Bruce Johnson was hired in February 2022 as Senior Vice President and Chief Commercial Officer
●Deepak Tiwari was hired in March 2022 as Vice President, Technical Operations
Fourth Quarter and Year-end 2021 Financial Results:

●Cash position: As of December 31, 2021, GlycoMimetics had cash and cash equivalents of $90.3 million as compared to $137.0 million as of December 31, 2020.

●Revenue: During the years ended December 31, 2021 and 2020, the Company recognized revenue of $1.2 million and $10.2 million, respectively, all of which was the result of payments received under our license and collaboration agreement with Apollomics for the development and commercialization of uproleselan and GMI-1687 in Greater China.

●R&D Expenses: The Company’s research and development expenses increased to $12.9 million for the quarter ended December 31, 2021, as compared to $11.7 million for the fourth quarter of 2020 due to higher development expenses related to manufacturing costs for the uproleselan validation batches and increased costs for IND enabling activities of GMI-1687.

Research and development expenses for the year ended December 31, 2021 increased to $47.5 million as compared to $44.9 million in the prior year. The increase in expenses was due to higher clinical development expenses related to our ongoing global Phase 3 clinical trial of uproleselan in individuals with relapsed/refractory AML, increased manufacturing costs and increased IND enabling activities related to GMI-1687.

●G&A Expenses: The Company’s general and administrative expenses increased to $4.5 million for the quarter ended December 31, 2021, as compared to $4.0 million for the fourth quarter of 2020. General and administrative expenses for the year ended December 31, 2021, increased to $17.1 million as compared to $16.7 million in the prior year. These increases were due to higher recruiting, consulting and legal expenses incurred in 2021 offset by lower personnel-related expenses due to the CEO transition.

●Shares Outstanding: Shares of common stock outstanding as of December 31, 2021 were 52,313,894.
The Company will host a conference call and webcast today at 8:30 a.m. ET. The dial-in number for the conference call is (844) 413-7154 for domestic participants and (216) 562-0466 for international participants, with participant code 4048456. Participants are encouraged to connect 15 minutes in advance of the call to ensure they are able to connect. A webcast replay will be available via the "Investors" tab on the GlycoMimetics website for 30 days following the call. A dial-in phone replay will be available for 24 hours after the close of the call by dialing (855) 859-2056 for domestic participants and (404) 537-3406 for international participants, with participant code 4048456.

About Uproleselan

Discovered and developed by GlycoMimetics, uproleselan is an investigational, first-in-class, targeted inhibitor of E-selectin. Uproleselan (yoo’ pro le’ sel an), currently in a comprehensive Phase 3 development program in AML, has received Breakthrough Therapy designation from the U.S. FDA and from the Chinese National Medical Products Administration for the treatment of adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment.

About GMI-1687

Discovered and developed by GlycoMimetics, GMI-1687 is a highly potent E-selectin antagonist that represents a potential life-cycle extension to uproleselan. GMI-1687 has been shown in animal models to be fully bioavailable following subcutaneous administration. As such, GMI-1687 is being positioned as a potentially self-administered drug to be used in the outpatient setting for the treatment of hematologic malignancies, such as AML and MDS, as well as inflammatory diseases such as acute vaso-occlusive crisis of sickle cell disease.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4 — both adhesion molecules involved in tumor trafficking and metastatic spread. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer, as well as in osteosarcoma, a rare pediatric tumor. GMI-1359 has received Orphan Drug designation and Rare Pediatric Disease designation from the FDA for the treatment of osteosarcoma, a rare cancer affecting about 900 adolescents a year in the United States.

About GMI-2093

GMI-2093 is a galectin-3 inhibitor discovered and being developed by GlycoMimetics. Available evidence indicates that the galectin-3 class may activate a variety of profibrotic factors, promote fibroblast proliferation and transformation, and mediate collagen production. Recent studies have defined key roles for galectin-3 in fibrogenesis in diverse organ systems, including liver, kidney, lung, and heart. Additionally, galectin-3 has been shown in preclinical models to be closely involved in tumor cell transformation, migration, invasion, and metastasis. GMI-2093 is being developed as a therapeutic strategy against tissue fibrosis and the treatment of certain cancers.

On March 3, 2022 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, reported its financial results for the year ended December 31, 2021 and provided a clinical update of its anti-cancer drug candidates currently in development for the treatment of primary and metastatic brain and CNS cancer (Press release, CNS Pharmaceuticals, MAR 3, 2022, View Source [SID1234609445]).

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2021 Key Highlights

May 2021: Commenced patient enrollment in the potentially pivotal study (CNS-201) evaluating efficacy of Berubicin in the treatment of adult recurrent Glioblastoma Multiforme (rGBM), one of the most aggressive types of brain cancer;
June 2021: FDA granted Fast Track Designation to CNS Pharmaceuticals for Berubicin for the treatment of recurrent GBM;
September 2021: Dosed first group of patients with Berubicin in the potentially pivotal study for the treatment of GBM;
December 2021: Received approval from "swissethics," the umbrella organization of the cantonal Ethics Committees (EC) in Switzerland, for the Company’s potentially pivotal study of Berubicin for the treatment of recurrent GBM; and
January 6, 2022: Announced a $11.5M private placement, priced at-the-market under Nasdaq rules.
"Operationally 2021 was a terrific year for the Company as we established the global framework for building and expanding our potentially pivotal clinical study of berubicin for the treatment of GBM. Our priority remains focused on advancing berubicin to bring a meaningful treatment to patients, families and clinicians, who currently have extremely limited and often ineffective treatment options. I am continually impressed by the speed and efficiency with which our small team has executed our Berubicin trial. At the onset of 2022 we completed a $11.5 million private placement which enables us to continue driving the development of berubicin this year towards the milestones ahead of us," commented John Climaco, CEO of CNS Pharmaceuticals. "While staying steadfast in our primary goal of bringing berubicin to GBM patients, we are also continually evaluating platform opportunities for expansion into additional oncology indications to bolster our pursuit of therapies for patients through developing novel treatments for primary and metastatic cancers. I am very pleased with the progress we’ve made over the past year, but am even more so looking forward to what lays ahead for CNS Pharmaceuticals."

Clinical Programs Update

Berubicin – Novel anthracycline

CNS’ lead product candidate, Berubicin, is a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier. Berubicin is currently in development for the treatment of a number of serious brain and CNS oncology indications. In mid-2021 the Company announced it has dosed the first group of patients in its potentially pivotal study evaluating the efficacy of Berubicin in the treatment of adult GBM, one of the most aggressive types of brain cancer. Further patient enrollment, randomization and dosing is currently underway as well as a robust lineup of clinical sites located globally which are advancing toward activation and enrollment.

The FDA granted CNS Pharmaceuticals Fast Track Designation for Berubicin which enables more frequent interactions with the FDA to expedite the development and review process. As previously announced, the Company also received Orphan Drug Designation from the FDA which may provide seven years of marketing exclusivity upon approval of an NDA.

For more information about the potentially pivotal Berubicin trial, visit clinicaltrials.gov and reference identifier NCT04762069.

Upcoming Milestones

Expand potentially pivotal study to evaluate efficacy of Berubicin in the treatment of adult GBM into additional countries;
Interim analysis of the trial when 30-50% of the total expected patients have been on study for 6 months (expected during first half of 2023); and
Complete enrollment in potentially pivotal clinical trial for GBM.
WP1244 Portfolio – Novel class of DNA-binding agents

The Company continues to advance the development of its WP1244 drug technology, which utilizes anthracycline and distamycin-based scaffolds to create small molecule agents and is believed to be 500x more potent than daunorubicin in inhibiting tumor cell proliferation. Preclinical studies of WP1244 demonstrated high uptake in the brain with antitumor activity. The Company’s development work has produced a new mesylate salt of WP1244, now identified as WP1874. The enhanced solubility of this salt may increase its ability to be formulated for use in an IV infusion, while maintaining similar potency and toxicity characteristics. Going forward, WP1874 will be the primary focus in our development efforts of the WP1244 portfolio. CNS Pharmaceuticals is also evaluating the use of WP1244/WP1874 in the treatment of other primary brain and central nervous system cancers, as well as cancers metastatic to the brain including pancreatic, ovarian, and lymphomas.

Upcoming Milestones

File IND in 2023.
Summary of Financial Results for the Full Year 2021

The net loss for the year ended December 31, 2021 was approximately $14.0 million compared to approximately $9.5 million for the comparable period in 2020. The change in net loss is attributable to increased personnel and activity associated with preparing for and commencing the Company’s potentially pivotal clinical trial of Berubicin for recurrent glioblastoma multiforme in 2021. The Company reported research and development expenses of $9.3 million for the year ended December 31, 2021 compared to approximately $5.1 million for the comparable period in 2020. The expenses incurred during the period were related to drug manufacturing and labor related to the preparation for and commencement of the Company’s potentially pivotal Berubicin study. General and administrative expense was approximately $4.7 million for the year ended December 31, 2021 compared to approximately $4.4 million for the comparable period in 2020.

As of December 31, 2021, the Company had cash of approximately $5.0 million and working capital of approximately $5.3 million. In early January 2022, the Company completed an offering of common stock and warrants for gross proceeds of $11.5 million. Our current expectation is that our cash on hand and the proceeds from the offering during January is sufficient to fund our operations into the first quarter of 2023. The timing and costs of clinical trials are difficult to predict and trial plans may change in response to evolving circumstances and as such the foregoing estimates may prove to be inaccurate.

On March 3, 2022 TG Therapeutics, Inc. (NASDAQ: TGTX), reported the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) goal date to June 25, 2022 for the Biologics License Application (BLA) and supplemental New Drug Application (sNDA) for ublituximab in combination with UKONIQ (umbralisib) as a treatment for patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) (Press release, TG Therapeutics, MAR 3, 2022, View Source [SID1234609444]).

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The FDA notified the Company that the updated overall survival analyses which were submitted to the FDA in February 2022, constituted a major amendment to the applications, and therefore the FDA is extending the PDUFA date to provide time for a full review of the submissions.

Michael S. Weiss, Chairman and Chief Executive Officer of TG Therapeutics stated, "As mentioned on our earnings call earlier this week, we believed an extension of the PDUFA date was a likely scenario especially given the proposed timing of the upcoming ODAC meeting. We hope this extension provides the time needed to give proper attention and review to the U2 BLA/sNDA." Mr. Weiss continued, "We continue to believe in the potential of U2 to provide a meaningful treatment option to patients with CLL and SLL."

ABOUT THE ODAC MEETING
In general, the Oncologic Drugs Advisory Committee (ODAC) reviews and evaluates data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of cancer and makes appropriate recommendations to the Commissioner of Food and Drugs. Although the FDA will consider the recommendation of the ODAC Committee, the final decision regarding the approval of a product is made solely by the FDA.

The FDA has notified the Company that potential questions and discussion topics for the ODAC include: the benefit-risk of the U2 combination in the treatment of CLL or SLL, and the benefit-risk of UKONIQ in relapsed/refractory marginal zone lymphoma (MZL) or follicular lymphoma (FL). In addition, as part of the benefit-risk analysis, the overall safety profile of the U2 regimen, including adverse events (serious and Grade 3-4), discontinuations due to adverse events, and dose modifications, is expected to be reviewed. The FDA’s concern giving rise to the ODAC meeting appears to stem from an early analysis of overall survival from the UNITY-CLL trial.

Overall survival was designated as a secondary efficacy outcome in the UNITY-CLL protocol but was not part of the primary analysis in accordance with the study’s statistical analysis plan agreed upon via a Special Protocol Assessment (SPA), and therefore, was not analyzed or included in the BLA/sNDA. Additionally, the study was not powered for overall survival. As part of the ongoing review of the BLA/sNDA, the FDA requested an early analysis of overall survival from the UNITY-CLL trial. As of September 2021, the cut-off date for the overall survival analysis requested by the FDA during their review, there was an imbalance in favor of the control arm (HR: 1.23) though this result was not statistically significant. However, when excluding deaths related to COVID-19, the two arms were approximately balanced (HR: 1.04) with again no statistically significant difference between the treatment groups with regard to overall survival. In February 2022, the Company submitted updated overall survival data with the same September 2021 cut-off date. The Company will continue to evaluate this endpoint over time as more events are available and will continue to analyze how COVID-19 may be impacting the analysis.

The date of the ODAC meeting has not yet been determined, although the FDA has stated that it is targeting holding the ODAC in March or April 2022.

ABOUT UNITY-CLL PHASE 3 TRIAL AND THE BLA/sNDA SUBMISSION
UNITY-CLL is a global, Phase 3, randomized, controlled clinical trial comparing the combination of ublituximab plus UKONIQ (umbralisib), or U2, to an active control arm of obinutuzumab plus chlorambucil in patients with both treatment-naïve and relapsed or refractory chronic lymphocytic leukemia (CLL). The trial randomized patients into four treatment arms: ublituximab single agent, UKONIQ single agent, ublituximab plus UKONIQ, and an active control arm of obinutuzumab plus chlorambucil. A prespecified interim analysis was conducted to assess the contribution of ublituximab and UKONIQ in the U2 combination arm and allowed for the termination of the single agent arms. Accordingly, the UNITY-CLL Phase 3 trial continued enrollment in a 1:1 ratio into the two combination arms: the investigational arm of U2 and the control arm of obinutuzumab plus chlorambucil. Approximately 420 subjects enrolled to the two combination arms and approximately 60% of patients were treatment-naïve and 40% were relapsed or refractory. The primary endpoint for this study was superior progression-free survival (PFS) for the U2 combination compared to the control arm. The trial met its primary endpoint, with U2 significantly prolonging independent review committee (IRC) assessed PFS vs. control (median 31.9 months vs 17.9 months; hazard ratio 0.546 (p<0.0001)) at a median follow-up of 36.7 months, and results were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020. The UNITY-CLL Phase 3 trial is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA).

The BLA/sNDA submissions of U2 to treat CLL were based on the results of the UNITY-CLL trial. The FDA previously granted Fast Track designation to the U2 combination for the treatment of adult patients with CLL and orphan drug designation for ublituximab in combination with UKONIQ for the treatment of CLL.

ABOUT CHRONIC LYMPHOCYTIC LEUKEMIA
Chronic lymphocytic leukemia (CLL) is the most common type of adult leukemia. It is estimated there will be more than 20,000 new cases of CLL diagnosed in the United States in 2020 and approximately 45,000 new cases globally in 2020.1,2 Although signs and symptoms of CLL may disappear for a period of time after initial treatment, the disease is considered incurable and many people will require additional treatment due to the return of malignant cells.

On March 3, 2022 VBL Therapeutics (Nasdaq: VBLT), a late-clinical stage biotechnology company developing first-in-class therapeutics for difficult-to-treat malignant solid tumors and immune or inflammatory indications, reported that management will participate in a fireside chat at the 34th Annual Roth Conference being held on March 13 – 15, 2022 as well as at the 32nd Annual Oppenheimer Healthcare Conference taking place on March 15 – 17, 2022 (Press release, VBL Therapeutics, MAR 3, 2022, View Source [SID1234609443]).

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34th Annual Roth Conference
Format: Fireside Chat
Date: Monday, March 14, 2022
Time: 9:30-9:55 AM (PT)
Fireside Chat Webcast Link
Pre-Conference Webinar Link

32nd Annual Oppenheimer Healthcare Conference
Format: Fireside Chat
Date: Wednesday, March 16, 2022
Time: 8:40-9:10 AM (ET)
Fireside Chat Webcast Link

A link to the webcasts of both fireside chats and the Roth pre-conference webinar will also be available on the Events and Presentations page of the Investor Relations section on the Company’s website at www.vblrx.com.

On March 3, 2022 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated, precision immune tolerance platform, ImmTOR, to develop tolerogenic therapies that selectively mitigate unwanted immune responses, reported that it plans to host a conference call on Thursday, March 10, 2021, at 8:30 a.m. ET to discuss its financial results for the quarter and full-year ended December 31, 2021 and provide a business update (Press release, Selecta Biosciences, MAR 3, 2022, https://selectabio.gcs-web.com/news-releases/news-release-details/selecta-biosciences-host-conference-call-and-webcast-discuss-7 [SID1234609438]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10157869. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the company’s website, www.selectabio.com.