On August 5, 2021 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq: RDUS), reported its financial results for the second quarter ended June 30, 2021 and year-to-date (Press release, Radius, AUG 5, 2021, View Source [SID1234585911]). In addition, the Company provided an update on components of the business.

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"Over the past 12 months we have focused on repositioning the business in a comprehensive manner," said Kelly Martin, Radius’ President and CEO. Martin continued, "within this effort three specific goals warrant being highlighted. They are, to become a cash flow positive company, to complete enrollment and execute our three ongoing pivotal trials in a high-quality manner, and opportunistically add assets that have the potential to enhance the Company’s value proposition. While there is still more to do, significant progress on all three of these objectives has been made to date."

Q2 and YTD FINANCIAL HIGHLIGHTS:

Total Net Revenue vs. prior year:
$52 million in Q2, 2021 vs. $50 million in Q2, 2020, +3% year-over-year
$108 million 1H, 2021 vs. $98 million 1H, 2020, +10% year-over-year
TYMLOS Net Revenue:
$52 million in Q2, 2021 vs. $50 million in Q2, 2020, +3% year-over-year
$97 million 1H, 2021 vs. $98 million 1H, 2020, -1% year-over-year
Total Company Adjusted EBITDA:
($6) million in Q2, 2021 vs. ($29) million in Q2, 2020
($11) million 1H, 2021 vs. ($55) million 1H, 2020
FY 2021 guidance: reiterate Adj. EBITDA of $10 million; while reducing TYMLOS revenue from $250 to $240 million
Liquidity position: $100 million of cash, cash equivalents and marketable securities as of 6/30/2021
ABALOPARATIDE COMMERCIAL UPDATE:

Patient growth continues in both quarter-over-quarter and year-over-year periods. Progress on business and market segment includes:

TYMLOS year-over-year new patient growth of 40+% in Q2, 2021 and 18% in 1H, 2021
Top 500 prescribers accounted for ~50% of new patients in Q2, 2021 vs. ~32% in Q1, 2021
In Q2, 2021, ~50% of our top 125 prescribers were orthopedic or spine-focused practices
New patient growth attributable to ortho/spine and bone health prescribers accelerated vs. prior quarter
As a reflection of the timing of new patient adds, we are reducing our FY 2021 TYMLOS net revenue guidance by $10 million. Given new patient growth during Q4, 2020 and 1H, 2021, we anticipate that the net revenue for TYMLOS in 2H, 2021 will be stronger than 1H, 2021.

The Company remains focused on making further progress in the commercial market space by concentrating resources, time, and effort on postmenopausal women with osteoporosis at high risk of fracture, including those with recent fractures.

ELACESTRANT UPDATE:

In partnership with the Menarini Group, progress continues to be made on the elacestrant asset and pivotal trial. The topline readout is still expected to occur in 2H, 2021 as previously communicated.

Importantly, there was a successful outcome in the completion of the bioequivalency study (BE study). This critical path item establishes equivalency between the clinical trial product and commercial product and is an important step forward on the progression of the asset.

Life cycle discussions and planning have been held with Menarini, and will continue to take place, regarding the possible therapeutic applications of elacestrant within the selective estrogen degrader (SERD) space.

RAD011 UPDATE:

As announced in a press release on July 23, Radius plans to initiate a seamless Phase 2/3 pivotal trial for patients with Prader-Willi Syndrome (PWS) in Q4, 2021 or early Q1, 2022. This trial will be global and incorporates feedback from the FDA, the KOL community as well as input from patient advocacy organizations.

Second Quarter 2021 Financial Results

Three Months Ended June 30, 2021

Net Loss
For the three months ended June 30, 2021, Radius reported a net loss of $16.8 million, or $0.35 per share, compared to a net loss of $43.9 million, or $0.95 per share, for the three months ended June 30, 2020.

For the three months ended June 30, 2021, non-GAAP adjusted net loss, was $10.5 million, or $0.22 per share, compared to non-GAAP adjusted net loss of $31.1 million, or $0.67 per share, for the three months ended June 30, 2020.

Revenue
For the three months ended June 30, 2021, TYMLOS net product revenues were $51.8 million compared to $50.1 million for the three months ended June 30, 2020.

Costs and Expenses
For the three months ended June 30, 2021, research and development expense was $27.0 million compared to $44.9 million for the three months ended June 30, 2020, a decrease of $17.9 million, or 40%. This decrease was primarily driven by a decrease of $10.6 million in abaloparatide-TD program cost, a $0.4 million decrease in occupancy and depreciation costs, a $4.6 million decrease in compensation expense, which is comprised of a $1.7 million decrease in compensation expense related to headcount and $2.9 million of billed reimbursable expenses, and a $8.8 million decrease in elacestrant program costs, which is comprised of a $6.6 million increase in gross program expenses offset by $15.4 million of billed reimbursable expenses. These decreases were offset by a $0.8 million increase in abaloparatide-SC program costs, a $3.6 million increase in RAD011 program costs, and a $2.1 million increase in professional fees and other expenses.

For the three months ended June 30, 2021, selling, general and administrative expenses were $32.1 million compared to $38.2 million for the three months ended June 30, 2020, a decrease of $6.1 million, or 16%. This decrease was primarily the result of a $2.6 million decrease in compensation cost, a $3.9 million decrease in professional support costs, and a $0.2 million decrease in occupancy and depreciation costs. These decreases were partially offset by a $0.5 million increase in travel and entertainment costs, and a $0.1 million increase in other operating costs.

Six Months Ended June 30, 2021

Net Loss
For the six months ended June 30, 2021, Radius reported a net loss of $32.6 million, or $0.69 per share, compared to a net loss of $81.5 million, or $1.76 per share, for the six months ended June 30, 2020.

For the six months ended June 30, 2021, non-GAAP adjusted net loss, was $19.0 million, or $0.40 per share, compared to non-GAAP adjusted net loss of $58.6 million, or $1.26 per share, for the six months ended June 30, 2020.

Revenue
For the six months ended June 30, 2021, TYMLOS net product revenues were $97.1 million compared to $98.0 million for the six months ended June 30, 2020.

For the six months ended June 30, 2021, license revenue was $11.0 million. No license revenue was recognized for the six months ended June 30, 2020.

Costs and Expenses
For the six months ended June 30, 2021, research and development expense was $58.4 million compared to $83.9 million for the six months ended June 30, 2020, a decrease of $25.5 million, or 30%. This decrease was primarily driven by a decrease of $14.0 million in abaloparatide-TD program cost, a $0.2 million decrease in RAD140 program costs, a $0.8 million decrease in occupancy and depreciation costs, a $9.3 million decrease in compensation expense, which is comprised of a $2.8 million decrease in compensation expense related to headcount and $6.5 million of billed reimbursable expenses, and a $16.4 million decrease in elacestrant program costs, which is comprised of a $9.6 million increase in gross program expenses offset by $26.0 million of billed reimbursable expenses. These decreases were offset by a $6.1 million increase in abaloparatide-SC program costs, a $3.6 million increase in RAD011 program costs, and a $5.8 million increase in professional fees and other expenses.

For the six months ended June 30, 2021, selling, general and administrative expenses were $66.2 million compared to $74.7 million for the six months ended June 30, 2020, a decrease of $8.4 million, or 11%. This decrease was primarily the result of a $2.0 million decrease in professional support costs, a $6.1 million decrease in compensation cost, and a $0.3 million decrease in other operating costs.

Webcast and Conference Call

In connection with today’s reporting of Second Quarter 2021 Financial Results, Radius will host a conference call and live audio webcast at 8:30 a.m. ET today, August 5, 2021, to review the commercial, research and development, and financial highlights and provide a Company update.

A live audio webcast of the call can be accessed from the Investors section of the Company’s website, www.radiuspharm.com. The full text of the announcement and financial results will also be available on the Company’s website.

A replay of the conference call will be available on August 5 at 11:30 a.m. ET and the audio webcast of the call will be archived on the Company’s website for ninety days. To access the replay, dial (855) 859-2056 or (404) 537-3406 for International, using conference ID number 3659752. The live audio webcast of the call can be accessed from the Investors section of the Company’s website, View Source The full text of the announcement and financial results will also be available on the Company’s website.

On August 5, 2021 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) ("Aeterna" or the "Company"), a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products, reported its financial and operating results for the second quarter ended June 30, 2021 (Press release, AEterna Zentaris, AUG 5, 2021, View Source;id=209178&p=2201538&I=1206939-c7Z3G6f3m8 [SID1234585927]). The Company also provided an update on its pre-clinical and clinical development programs.

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"Our team continues its solid execution to drive forward the development across our clinical and preclinical programs. During the second quarter, we achieved key milestones across multiple fronts including bolstering our management team and scientific expertise, commencing our pivotal Phase 3 DETECT-trial of macimorelin, and advancing our earlier-stage preclinical development pipeline," commented Dr. Klaus Paulini, Chief Executive Officer of Aeterna. "I believe we are well-positioned to propel the Company forward with the goal of creating meaningful shareholder value."

Recent Highlights:

Engaged neuro-immunologist, Michael Levy, MD, PhD, Research Director of the Division of Neuroimmunology & Neuroinfectious Disease at Massachusetts General Hospital as a scientific consultant to support the development of the Company’s targeted, highly specific immunosuppressive therapeutic proteins ("AIM Biologicals") for the potential treatment of neuromyelitis optica spectrum disorder ("NMOSD").
Advanced preclinical development planning for potential orally active COVID-19 (SARS-CoV-2) live-attenuated bacterial vaccine program.
Initiated the preclinical program to qualify macimorelin for clinical development as a potential treatment option for amyotrophic lateral sclerosis (ALS; Lou Gehrig’s disease).
Commenced its pivotal Phase 3 safety and efficacy study AEZS-130-P02 ("the DETECT-trial") evaluating macimorelin for the diagnosis of childhood-onset growth hormone deficiency ("CGHD") in collaboration with Novo Nordisk.
In consultation with The University of Sheffield, UK, selected AEZS-150 as the lead candidate in the Company’s delayed clearance parathyroid hormone fusion polypeptides ("DC-PTH") program.
Appointed Michael Teifel, Ph.D. as Senior Vice President, Non-Clinical Development and Chief Scientific Officer.
Fully settled the previously disclosed class-action lawsuit against it in the U.S. District Court for the District of New Jersey, as approved by the U.S. District Court for the District of New Jersey.
All amounts in this press release are in U.S. dollars unless otherwise noted.

Preclinical and Clinical Programs Update:

Diagnostics Development Pipeline

Macimorelin Diagnostic:Ghrelin agonistin development for diagnostic use in childhood-onset growth hormone deficiency ("CGHD")

Aeterna Zentaris is currently conducting its pivotal Phase 3 safety and efficacy study AEZS-130-P02 ("the DETECT-trial") evaluating macimorelin for the diagnosis of CGHD. Children and adolescents from two to less than 18 years of age with suspected GHD are to be included. The study is expected to include approximately 100 subjects worldwide, with at least 40 subjects in pre-pubertal and 40 subjects in pubertal status. Macimorelin growth hormone stimulation test ("GHST") will be performed twice for repeatability data and two standard GHSTs will be used as controls: arginine (i.v.) and clonidine (p.o.). On April 22, 2021, the U.S. FDA Investigational New Drug Application associated with this clinical trial became active. The first clinical site in the U.S. is now open for patient recruitment.

Next Steps

Conduct and completion of the DETECT-trial.
Therapeutics and Vaccine Development Pipeline

AIM Biologicals:Targeted, highly specific autoimmunity modifying therapeutics for the potential treatment of neuromyelitisoptica spectrum disorder ("NMOSD")

In January 2021, Aeterna Zentaris entered into an exclusive patent license and research agreement with Julius-Maximilians-University of Wuerzburg, Germany for worldwide rights to develop, manufacture and commercialize AIM Biologicals for the potential treatment of NMOSD. The Company is currently conducting in-vitro and in-vivo assessments to identify and characterize an AIM Biologicals-based development candidate for the treatment of NMOSD and to develop a manufacturing process for the selected candidate. Additionally, the Company recently engaged neuro-immunologist, Dr. Michael Levy, who will provide scientific support and advice in the field of inflammatory CNS disorders, autoimmune diseases of the nervous system, and NMOSD.

Next Steps

Conduct further preclinical research to identify and characterize an AIM Biologicals-based development candidate for the treatment of NMOSD.
Manufacturing process development for selected candidate.
Macimorelin Therapeutic:Ghrelin agonist in developmentfor the treatment of ALS (Lou Gehrig’s disease)

In January 2021, the Company entered into a material transfer agreement ("MTA") with Queensland University, Australia, to provide macimorelin for the conduct of preclinical and subsequent clinical studies evaluating macimorelin as a potential therapeutic for the treatment of ALS (Lou Gehrig’s disease). Queensland University researchers have filed for supportive grants and aim to conduct preclinical studies in multiple models to demonstrate the therapeutic reach of macimorelin on disease progression and disease-specific pathology. They also plan to conduct a subsequent investigator initiated clinical trial given positive pre-clinical results.

Macimorelin, a ghrelin agonist, is an orally active small molecule that stimulates the secretion of growth hormone from the pituitary gland. Acting via this mechanism, it is believed that macimorelin may slow the progression of certain neurodegenerative diseases like ALS.

Next Steps

Work with Queensland University to conduct proof-of-concept studies with macimorelin in disease-specific animal models.
Assess alternative formulations.
Formalize preclinical development plan.
Delayed Clearance Parathyroid Hormone ("DC-PTH") Fusion Polypeptides:Potential treatment for primary hypoparathyroidism

In March 2021, Aeterna entered into an exclusive patent and know-how license agreement and research agreement with The University of Sheffield, United Kingdom, for the intellectual property relating to DC-PTH fusion polypeptides with delayed clearance covering the field of all human uses. In consultation with the University, Aeterna has selected AEZS-150 as the lead candidate in its delayed clearance parathyroid hormone fusion polypeptides ("DC-PTH") program. Aeterna will now start the formal preclinical development of AEZS-150 in preparation for a potential IND filing for conducting the first in-human clinical study of AEZS-150. AEZS-150 is being developed with the goal of providing a potential new treatment option of primary hypoparathyroidism in adults.

Next Steps

Work with The University of Sheffield to conduct in depth characterization of development candidate (in-vitro and in-vivo).
Develop manufacturing process.
Oral Coronavirus Vaccine Platform:Potential orally active COVID-19 (SARS-CoV-2) live-attenuated bacterial vaccine

In February 2021, Aeterna entered into an exclusive option agreement with Julius-Maximilians-University to evaluate a preclinical, potential COVID-19 vaccine developed at Julius-Maximilians-University. In March 2021, the Company exercised its option and entered into a license agreement where the Company was granted an exclusive, world-wide, license to certain patent applications and know-how owned by Julius-Maximilians-University to research and develop, manufacture, and sell a potential COVID-19 vaccine.

Julius-Maximilians-University also granted Aeterna an option for the exclusive use of certain patent applications and know-how in an additional, so-far undisclosed field. The Company has six months from the date of the license agreement to exercise that option. Additionally, Aeterna entered into a Research Agreement under which the Company has engaged Julius-Maximilians-University on a fee-for-service basis to conduct supplementary research work and preclinical development studies on the potential vaccine, the results of which will be included within the scope of the license agreement.

Next Steps

Conclude in-vivo immunology experiments with antigen variant vaccine candidates.
Perform challenge experiments in immunized transgenic animals as proof of concept.
Select a development candidate for initiation of the formal preclinical toxicology and safety studies.
Start manufacturing process assessment / development.
Financing and Warrant Exercises

During the period between January 1, 2021 and June 30, 2021, holders have exercised certain of the Company’s outstanding warrants to purchase 35,011,187 of the Company’s common shares for gross proceeds of approximately $20.0 million. The Company had $69.9 million cash and cash equivalents at June 30, 2021 (March 31, 2021 – 73.4 million).

Summary of Second Quarter 2021 Financial Results

Results of operations for the three-month period ended June 30, 2021

For the three-month period ended June 30, 2021, we reported a consolidated net loss of $2.0 million, or $0.02 loss per common share (basic), as compared with a consolidated net loss of $3.5 million, or $0.15 loss per common share (basic) for the three-month period ended June 30, 2020. The $1.5 million improvement in net loss is primarily from an increase in net finance income of $2.1 million and an increase of $0.5 million in total revenues, partially offset by an increase of $1.2 million in total operating expenses.

Revenues

Our total revenue for the three-month period ended June 30, 2021 was $0.60 million as compared with $0.07 million for the same period in 2020, representing an increase of $0.53 million. The 2021 revenue was comprised of $0.5 million in licensing revenue (2020 – $0.02 million), $0.04 million in supply chain revenue (2020 – $0.04 million) and $0.02 million in royalty income (2020 – $0.01 million). The licensing revenue was earned from the recognition of a portion of the deferred 5 million payment from Novo Nordisk received with the amendment to the Novo License agreement in the fourth quarter of 2020.
Operating expenses

Our total operating expenses for the three-month period ended June 30, 2021 was $2.7 million as compared with $1.5 million for the same period in 2020, representing an increase of $1.2 million. This increase arose primarily from a $0.5 million increase in research and development costs and $0.5 million increase in general and administrative expenses and an increase of $0.1 million in selling expenses. This increase in total operating expense is due to the initiation of research and development projects as announced in the first quarter of 2021, and higher public company costs incurred holding our annual shareholders meeting in May 2021, subsequent to our issuance of common shares in the February 2021 financing and the 2021 warrant exercises, in addition to higher auditor fees mostly related to F-3 warrant registration filings.
Net finance (costs) income

Our net finance income for the three-month period ended June 30, 2021 was $0.1 million as compared with net finance costs of ($2.0 million) for the same period in 2020, representing an increase in net finance income of $2.1 million. This is primarily due to the $2.1 million decrease in change in fair value of warrants. Unlike as at June 30, 2020, the Company did not account for its warrants as liabilities as at June 30, 2021. As at June 30, 2020, the fair value of such liabilities was classified as a finance cost in the consolidated statements of loss and comprehensive loss.
Consolidated Financial Statements and Management’s Discussion and Analysis

For reference, the Management’s Discussion and Analysis of Financial Condition and Results of Operations for the second quarter of 2021, as well as the Company’s unaudited consolidated interim financial statements as of June 30, 2021, will be available at www.zentaris.com in the Investors section or at the Company’s profile at www.sedar.com and www.sec.gov.

On August 5, 2021 TransCode Therapeutics, Inc. (Nasdaq: RNAZ), a platform company solving the challenge of RNA delivery in oncology, reported that it has been awarded a Fast-Track Small Business Innovation Research (SBIR) grant from the NIH to support the clinical evaluation of TTX-MC138, TransCode’s lead therapeutic candidate for the treatment of metastatic solid tumors (Press release, TransCode Therapeutics, AUG 5, 2021, View Source [SID1234585944]). The award totals $2.3 million expected over three years.

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"We are honored that the NIH recognizes and supports our mission to overcome the obstacles of RNA delivery in oncology," said Michael Dudley, President and Chief Executive Officer of TransCode. "We believe our TTX delivery platform offers tremendous potential across a range of indications. Specifically, this SBIR grant will help fund our planned first-in-human study of our lead candidate, TTX-MC138, in patients with metastatic breast cancer. We look forward to submitting an eIND application in the first quarter of next year. This trial is designed to evaluate and demonstrate proof-of-concept for the mechanism of action for our novel delivery platform, potentially enabling TransCode to pursue a diverse pipeline of TTX-based therapeutics."

TransCode’s proprietary TTX platform leverages an iron oxide nanoparticle as a novel, image-guided system to safely and efficiently deliver oligonucleotides to their intended RNA target. TTX-MC138 targets MicoRNA-10b, believed to drive metastatic disease. The therapeutic has been validated preclinically in multiple indications and has been shown to induce durable regressions of metastatic disease in murine models of disseminated breast cancer.

On August 5, 2021 Second Genome, a biotechnology company that leverages its proprietary platform sg-4-sight to discover and develop precision therapies and biomarkers from public and proprietary microbiome data, reported they are entering a strategic collaboration with Arena Pharmaceuticals, Inc. (Nasdaq: ARNA) to identify microbiome biomarkers associated with clinical response for their lead program in gastroenterology, etrasimod (Press release, Second Genome, AUG 5, 2021, View Source [SID1234586037]).

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Under the terms of the agreement, Second Genome will utilize its proprietary sg-4-sight discovery engine to identify microbiome biomarkers associated with clinical benefit or adverse effects observed within data from the CULTIVATE clinical trial. Arena’s CULTIVATE trial is a Phase 2/3 clinical trial evaluating the efficacy and safety of etrasimod, a next-generation, once-daily, oral, highly selective sphingosine 1-phosphate (S1P) receptor modulator, in patients with moderately-to-severely active Crohn’s disease. This work will help inform patient stratification and optimize potential treatments for patients in the future.

"The multi-disciplinary capabilities of our sg-4-sight platform and our team’s deep expertise enable Second Genome to collaborate with partners as we continue to advance our own pipeline of precision therapeutics and biomarkers," said Karim Dabbagh, Ph.D., President and Chief Executive Officer of Second Genome. "Arena shares our commitment to unlocking innovative ways to improve human health. We look forward to working with Arena and applying our sg-4-sight platform to discover microbiome signals in Crohn’s disease patients."

"There is substantial evidence that gut microbiome is intricately involved in therapy response in gastrointestinal diseases," said Amit D. Munshi, President and Chief Executive Officer of Arena Pharmaceuticals. "We are pleased to collaborate with Second Genome to discover microbiome biomarkers predictive of clinical response as we continue to progress our CULTIVATE clinical trial."

On August 5, 2021 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results for the quarter ended June 30, 2021 and highlighted recent events (Press release, GlycoMimetics, AUG 5, 2021, View Source [SID1234585802]). Cash and cash equivalents at June 30, 2021 were $118.9 million.

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"There are now six trials underway evaluating our lead clinical candidate, uproleselan, including three registration trials and three ISTs, which we anticipate will provide clinical data flow beginning in 2022. Importantly, recruitment rates in both our Company-sponsored Phase 3 trial and the National Cancer Institute’s Phase 2 portion of the Phase 2/3 trial support our expectation that enrollment in both studies can be completed by the end of this year. The support of clinicians who are enrolling patients in our global studies, and now the new ISTs, has made it possible to broaden the scope of our uproleselan clinical research to address unmet needs in AML and beyond," commented Chief Executive Officer Rachel King.

Operational Highlights

Uproleselan

Enrollment of GlycoMimetics’ pivotal Phase 3 trial in relapsed/refractory AML continued in the U.S., Canada, Australia and Europe at a steady pace throughout the second quarter of 2021. The Company continues to project that enrollment will be completed by year-end 2021.
The pace of enrollment in the National Cancer Institute (NCI)-sponsored Phase 2/3 registration trial, designed to evaluate the use of uproleselan in newly diagnosed older adults with AML who are fit for chemotherapy, continues to support the Company’s expectation that the Phase 2 portion will complete in 2021, and allow for a subsequent interim Event-Free Survival analysis of 262 patients.
During the quarter and shortly after the quarter close, clinicians initiated three ISTs designed to evaluate uproleselan in AML and in bone marrow transplantation for multiple myeloma. These trials are expected to begin producing clinical data in 2022, which the Company believes will support the potential of uproleselan to be used as a foundational treatment in AML to overcome well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment and reduce adverse effects of chemotherapy.
GMI-1359

In April 2021 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) meeting, Duke University clinicians reported biologic activity, as demonstrated by cell mobilization, redistribution of immune subset profiles and changes in other pharmacodynamic markers, observed in the initial two patients treated in the ongoing Phase 1b study in patients with advanced breast cancer with bone metastases. The initial clinical data support the dual functionality of the compound and the potential of GMI-1359 to enhance responses to chemo and immune therapies.
GMI-1687

The Company continued to advance GMI-1687 towards filing of an investigational new drug application (IND), anticipated in the first half of 2022.
Management Transition

Yesterday, the Company announced that its Board of Directors has appointed Harout Semerjian as chief executive officer (CEO), effective August 6, 2021, to succeed Founding CEO Rachel King. Mrs. King, who has served as CEO since the Company’s founding, has decided to retire for personal reasons and will continue her involvement with the Company through her role on the Board of Directors and serving as an advisor during a transition period. Mr. Semerjian, a seasoned executive with strong commercial oncology experience, will lead the Company as it advances its registration trials for uproleselan in AML, accelerates planning for potential commercialization, and continues to build out the Company’s pipeline.
Second Quarter 2021 Financial Results

Cash position: As of June 30, 2021, GlycoMimetics had cash and cash equivalents of $118.9 million as compared to $137.0 million as of December 31, 2020.
R&D Expenses: Research and development expenses increased to $10.2 million for the quarter ended June 30, 2021 as compared to $9.9 million for the quarter ended June 30, 2020. This increase was primarily due to an increase in clinical trial costs in our ongoing global Phase 3 clinical trial of uproleselan in individuals with relapsed/refractory AML.
G&A Expenses: General and administrative expenses were $4.2 million for the second quarter ended June 30, 2021 and 2020.
Shares Outstanding: Shares of common stock outstanding as of June 30, 2021 were 51,539,010.
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 9977599. 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 9977599.

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. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed or refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better-than-expected remission rates and overall survival compared to historical controls, which have been derived from results from third-party clinical trials evaluating standard chemotherapy, as well as lower-than-expected induction-related mortality rates. Treatment in these patient populations was generally well-tolerated, with fewer than expected adverse effects.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4, which are 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 affecting about 900 adolescents a year in the United States. GMI-1359 completed a Phase 1 clinical trial in healthy volunteers, and a Phase 1b clinical study designed to enable investigators to study dose ranging and to generate initial biomarker data around the drug’s activity in breast cancer patients is in progress. In the first two patients evaluated, the study showed evidence of on-target effects, immune-activation and cell mobilization. GMI-1359 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for the treatment of osteosarcoma.

About GMI-1687

Discovered and developed by GlycoMimetics, GMI-1687 is a highly-targeted, highly-potent E-selectin antagonist. It has been shown in preclinical studies to be bioavailable via subcutaneous administration. During 2020, data from oral presentations at major scientific conferences pointed to the potential for a self-administered drug to treat VOC of sickle cell disease. Previously, GlycoMimetics demonstrated in preclinical models that GMI-1687 could be a potentially self-administered drug to be used in treatment of AML. The investigational drug also represents a potential life-cycle extension opportunity for uproleselan.