On July 31, 2020 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results for the second quarter ended June 30, 2020, and highlighted recent company events (Press release, GlycoMimetics, JUL 31, 2020, View Source [SID1234562624]). Cash and cash equivalents at June 30, 2020 were $149.8 million.

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"The second quarter was quite productive as we noted strong enrollment in our company-sponsored pivotal trial of uproleselan in relapsed/refractory AML. Though we did see a slow down early in the COVID crisis, we were pleased to see enrollment pick back up later in the quarter. This was an important achievement in the face of the COVID-19 pandemic, and we are able to maintain prior guidance that we expect to complete enrollment in the second half of 2021. Also important were new data from a post hoc analysis of the rivipansel Phase 3 study that showed that patients treated with rivipansel within approximately 26 hours of the onset of pain in their crisis experienced statistically significant improvements in the primary efficacy endpoint of time to readiness for discharge compared to placebo. These data provide a foundation, we believe, for us to discuss with the U.S. Food and Drug Administration (FDA) whether there may be a path forward for rivipansel in acute VOC," commented Rachel King, Chief Executive Officer.

Operational Highlights

Uproleselan

●GlycoMimetics’ ongoing pivotal Phase 3 trial in relapsed/refractory AML continued to activate clinical sites and enroll patients in North America, Australia and Europe. While individual sites were affected by COVID-19, overall clinicians continued to enroll patients well this quarter.
●At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2020, held June 22-24, preclinical research for both uproleselan and GMI-1359 was shared in a virtual format. Preclinical data supporting the potential use of uproleselan in the treatment of AML as well as in the setting of stem cell transplantation were presented. Additionally, new information demonstrated the ability of transcriptome profiling to identify those tumor types most likely to benefit from targeted E-selectin inhibition, a key mechanism of GlycoMimetics drug candidates.
●Important new preclinical data on the mechanism of action for uproleselan were published in the April 27, 2020, issue of Nature Communications. The paper outlined how uproleselan, a first-in-class, targeted inhibitor of E-selectin, can reduce chemoresistance in AML through the key mechanism of targeted E-selectin inhibition.
GMI-1687

●An abstract that was accepted for oral presentation at the Foundation for Sickle Cell Disease (FSCDR) meeting and published online in June, disclosed data from a preclinical model of the Company’s E-selectin antagonist, GMI-1687, which is more potent than rivipansel and is being formulated for subcutaneous dosing. The data support development of GMI-1687 as a possible follow-on to rivipansel, which has the potential for subcutaneous self-administration as would be used in an outpatient setting.
Rivipansel

●The FSCDR also published online an abstract including data from a post hoc analysis of the Phase 3 RESET trial of 345 patients (ranging in age from six years to adults, with a mean age of 22 years) who were experiencing acute VOC requiring hospitalization for treatment. The organization selected the abstract for poster presentation at its September meeting. The analysis showed that patients treated with rivipansel early in their acute episode experienced a statistically significant improvement on the primary efficacy endpoint, time to readiness for discharge (p=0.03, median improvement at 56.3 hours compared to placebo).
●Based upon its review of the Phase 3 rivipansel data set, GlycoMimetics is committed to an assessment of what, if any, next steps to take, with a focus on determining if there is a potential path forward for this asset in sickle cell disease.
●GlycoMimetics completed in April a transfer back from Pfizer of the commercial and development rights and licenses for rivipansel, the IND for the clinical development program, and the entire data set for the Phase 3 RESET trial.

Executive Management Team

●The Company announced that veteran regulatory expert Myra Rosario Herrle, PhD, RPh, RAC, joined the executive management team as Vice President, Regulatory Affairs.
Second Quarter 2020 Financial Results:

●Cash position: As of June 30, 2020, GlycoMimetics had cash and cash equivalents of $149.8 million as compared to $158.2 million as of December 31, 2019. During the quarter, the Company sold common stock under its at-the-market facility with Cowen for aggregate net proceeds of $9.6 million.

●R&D Expenses: The Company’s research and development expenses decreased to $9.9 million for the quarter ended June 30, 2020 as compared to $13.1 million for the second quarter of 2019. The Company’s research and development expenses decreased to $22.5 million for the six months ended June 30, 2020 as compared to $24.8 million for the same period in 2019. These decreases were due primarily to a decrease in manufacturing and formulation expenses resulting from lower raw material costs as the validation manufacturing batches were purchased in the prior year. The decreases were offset in part by higher clinical expenses as a result of the increased enrollment in the ongoing global Phase 3 clinical trial of uproleselan in individuals with relapsed/refractory AML and the Phase 2/3 clinical trial being conducted by the National Cancer Institute. Contract research services, consulting and other costs were lower in 2020 as research activities were affected at outside universities and travel by research and development personnel was largely eliminated due to the COVID-19 pandemic.

●G&A Expenses: The Company’s general and administrative expenses increased to $4.2 million for the second quarter ended June 30, 2020 as compared to $3.8 million for the second quarter of 2019. General and administrative expenses for the six months ended June 30, 2020 increased to $8.7 million as compared to $7.1 million in the same period in 2019. Personnel-related expenses increased due to additional general and administrative headcount, annual salary adjustments awarded in the first quarter of 2020 and retention bonuses. Patent, legal fees, consulting and other professional expenses including director and officer’s insurance premiums, increased as compared to 2019. Other general and administrative expenses decreased for both the three and six months ended June 30, 2020, as compared to the same periods in 2019, due to lower travel, meals and conference registration expenses as a result of the travel restrictions due to the COVID-19 pandemic.

●Shares Outstanding: Shares of common stock outstanding as of June 30, 2020 were 46,714,698.
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 or (216) 562-0466 for international participants, with participant code 1677593. Participants are encouraged to connect 15 minutes in advance of the call to ensure that all callers 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, participant code 1677593.

About Uproleselan (GMI-1271)

Discovered and developed by GlycoMimetics, uproleselan and GMI-1687 are investigational, first-in-class, targeted inhibitors 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 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-1687

GMI-1687 is a rationally designed, innovative antagonist of E-selectin that is potentially suitable for subcutaneous (SC) administration. When given by SC injection in preclinical models, GMI-1687 has been observed to have equivalent activity to uproleselan, but at an approximately 1,000-fold lower dose. GlycoMimetics believes that GMI-1687 could be developed as a potential life-cycle expansion to broaden the clinical usefulness of an E-selectin antagonist to conditions, such as sickle cell disease crisis, where outpatient treatment may be preferred or required. GMI-1687 is currently undergoing IND-enabling studies.

About Rivipansel

Rivipansel, a glycomimetic drug candidate that binds to all three members of the selectin family (E-, P- and L-selectin), was GlycoMimetics’ first drug candidate to enter clinical development. After the Phase 3 RESET trial conducted by Pfizer, GlycoMimetics’ former collaborator, did not meet its primary or key secondary efficacy endpoints in 2019, new efficacy data from a post hoc analysis of rivipansel were published in June 2020 in advance of a presentation to occur at the Foundation for Sickle Cell Disease Research Meeting in September 2020. GlycoMimetics is committed to exploring a path forward for the use of rivipansel in treating acute VOC in SCD.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are 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 completed a Phase 1 clinical trial in healthy volunteers. The Duke University Phase 1b clinical study in breast cancer patients is designed to enable investigators to identify an effective

dose of the drug candidate and to generate initial biomarker data around the drug’s activity. 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.

On July 31, 2020 Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmology company, reported that the divestment of its remaining shareholding in VISUfarma, a pan-European ophthalmic specialty pharmaceutical company, has closed as expected and, as per the press release of 10 July 2020, Nicox has received the funds of €5 million (Press release, NicOx, JUL 31, 2020, View Source;utm_medium=rss&utm_campaign=nicox-receives-e5-million-upon-closing-of-visufarma-divestment-2 [SID1234562618]).

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On July 30, 2020 Theralase Technologies Inc. ("Theralase" or the "Company") (TSXV: TLT) (OTCQB: TLTFF), a clinical stage pharmaceutical company focused on the research and development of light activated Photo Dynamic Compounds ("PDC") and their associated drug formulations to safely and effectively destroy various cancers reported to provide an update on its Phase II Non-Muscle Invasive Bladder Cancer ("NMIBC") Clinical Study ("Study II") (Press release, Theralase, JUL 30, 2020, View Source [SID1234568565]).

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The primary purpose of Study II is to discover if the TLD-1433 ("Study Drug") combined with TLC-3200 ("Study Device") (collectively the "Study Treatment") are effective in the destruction of Bacillus Calmette-Guérin ("BCG")-Unresponsive Non-Muscle Invasive Bladder Cancer ("NMIBC").

All Canadian clinical study sites currently remain closed for new patient enrollment and treatment; however, Canadian clinical study sites, who have already enrolled and treated patients, are providing second treatments. The Company is preparing to launch a fifth Canadian clinical study site later in the year.

The Company is preparing to launch a number of US based clinical sites later in the year, subject to the United States economy recovering from COVID-19; however, it is anticipated due to the severity of the COVID-19 pandemic in the United States, that these sites will not be open to commence enrollment and treatment of patients until 2021.

Study II enrolled and treated 12 patients, with the following results:

Patient Status Number of Patients Percentage
First Treatment Provided 12 100.0%
Patients Eligible to Receive Second Treatment 8 66.7%
Second Treatment to be Provided 5 41.7%
Second Treatment Provided 3 25.0%

Response at 90 Days Post Initial Treatment 7 58.3%
Complete Response at 90 Days Post Initial Treatment 3 25.0%

Patients Removed from Study II 4 33.3%
Study II Interim Data Analysis:

Of the 7 patients, who demonstrated a Response to the Study Treatment defined as negative cystoscopy (no evidence of cancer in their bladders) or negative urine cytology (no evidence of the urothelial carcinoma cells in their urine)) at 90 days post initial treatment:

43% achieved a Complete Response ("CR") (negative cystoscopy and negative urine cytology).
43% achieved a Response (negative cystoscopy and positive or suspicious cytology)
14% achieved a Response (suspicious cystoscopy and negative cytology)
The patients who responded to the Study Treatment are currently under medical review to assess the cause of the suspicious cystoscopy by directed bladder biopsy and the cause of the positive or suspicious cytology by repeating the urine cytology analysis. If found to be negative, these patients will be allocated to the CR column. If positive or suspicious again, then Computerized Tomography ("CT") Scan imaging and/or prostatic biopsies will be conducted to rule out Upper Tract Urothelial Cell Carcinoma ("UTUCC"). If UTUCC is proven to exist, then according to FDA’s Bacillus Calmete Guérin ("BCG")-Unresponsive Guidelines to Industry, issued in February 2018, these patients will be classified as CR, as only the bladder was treated by the Study Treatment and not non-addressable areas of the urinary system.

Of the 4 patients that were removed from Study II, the clinical protocol in effect at the time stated in the patient withdrawal criteria that "Patients found to have at Day 90 new T1 high-grade disease, with or without CIS" and "Patients found to have unchanged or worsening CIS at 3 months" will be removed from Study II.

Of the 4 patients removed from the Study, they presented with "unchanged CIS at 3 months".

This was an oversight on behalf of the Company and the clinical protocol has since been updated to remove these two specific patient withdrawal criteria, maintaining the key patient withdrawal criteria of NMIBC progression, which includes "Progression to Muscle Invasive Bladder Cancer ("MIBC")" and "Progression to metastatic disease". These changes, had they been in place, would have led to the retreatment of these 4 patients and an opportunity to achieve CR for these patients at a later medical assessment point.

Study Treatment Optimizations:

Additional optimizations to the clinical study protocol that have been implemented for all future patients to be enrolled and treated in Study II and for the five patients yet to receive their second treatment, include:

Bladder Volume Calculation:

The clinical protocol did not clearly define the bladder volume calculation to be used by the pharmacy and the principal investigator to determine bladder size for administration of the Study Drug and Study Device, respectively. Average bladder volume voided over a 3 day period was used as opposed to a percentage of the maximum bladder volume voided.

Study Device Treatment Time:

The Study Device treatment time was based on detected bladder irradiance, which varied dramatically inside patient bladders due to shape, volume and bladder wall reflection. This led to undertreatment of certain patients with the Study Device by up to 87.9%. This has been modified to now determine Study Device treatment time based solely on the new bladder volumecalculation, resulting in a more consistent Study Device treatment time across patients.

Summary:

The total of these Study II variances (Study Drug Volume, Study Device Volume and Study Device Treatment Time) have led to all 12 patients being undertreated by the Study Treatment from between 30.9% and 154.3%.

About Study II

Study II utilizes the Therapeutic Dose (0.70 mg/cm2) of TLD-1433 and is focused on the enrollment and treatment of approximately 100 BCG-Unresponsive NMIBC patients presenting with Carcinoma In-Situ ("CIS") in approximately 20 clinical study sites located in Canada and the US.

Study II has a:

Primary endpoint of efficacy (defined by CR) at any point in time
Secondary endpoint of duration of CR at 360 days post-initial CR
Tertiary endpoint of safety measured by incidence and severity of Adverse Events ("AEs") grade 4 or higher that do not resolve within 360 days post-initial CR
"For single-arm trials of patients with BCG-unresponsive disease, the FDA defines a CR as at least one of the following:

Negative cystoscopy and negative (including atypical) urine cytology
Positive cystoscopy with biopsy-proven benign or low-grade NMIBC and negative cytology
For intravesical therapies without systemic toxicity, negative cystoscopy with malignant urine cytology, if cancer is found in the upper tract or prostatic urethra and random bladder biopsies are negative.
Intravesical instillation does not deliver the investigational drug to the upper tract or prostatic urethra; therefore, the development of disease in these areas cannot be attributed to a lack of activity of the investigational drug. Thus, sponsors can consider patients with new malignant lesions of the upper tract or prostatic urethra, who have received intravesical therapy to have achieved a CR in the primary analysis; however, sponsors should record these lesions and conduct sensitivity analyses in which these patients are not considered to have achieved a CR." 1

On July 30, 2020 Genenta Science, a clinical-stage biotechnology company pioneering the development of a hematopoietic stem cell gene therapy for cancer (Temferon), reported that they will present the corporate overview at the ACCESS CHINA Forum 2020, Summer Online Partnering Day, hosted by YAFO Capital Life Science (Press release, Genenta Science, JUL 30, 2020, View Source [SID1234568503]).

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Pierluigi Paracchi, Founder, Chairman and CEO of Genenta, will present the immune-gene therapy technology for tumors treatment, ongoing clinical trials, and company’s strategy on July 31, at 11:30am CET

On July 30, 2020 PDL BioPharma, Inc. ("PDL" or the "Company") (Nasdaq: PDLI) reported the signing of a definitive agreement for the sale of 100% of the outstanding stock in its wholly owned subsidiaries Noden Pharma DAC and Noden USA (collectively "Noden") to Stanley Capital ("Stanley Capital") (Press release, PDL BioPharma, JUL 30, 2020, View Source [SID1234565091]). The total value of the transaction will result in payments to PDL of up to $48.25 million in cash.

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"Entering into this transaction for the sale of Noden to Stanley Capital represents another significant step in the execution of our monetization strategy," commented PDL’s President and CEO Dominique Monnet. "We thank Alan Markey and the Noden team for their dedication and service, and we wish Noden and Stanley Capital much success."

Simon Cottle, Partner at Stanley Capital, said: "The acquisition of Noden is the culmination of years of thematic research into the specialty pharmaceuticals sector, which identified the company as a strong buy and build platform. We look forward to working with Noden’s excellent management team and our industry advisors to build the company into a leading pharma platform, providing essential treatments for patients with unique and difficult to treat conditions."

After taking into account the expected adjustments for transaction expenses, indebtedness and working capital, payments to PDL are expected to be approximately $12.0 million in connection with the closing of the transaction. The agreement provides for an additional $33 million to be paid to PDL in twelve equal quarterly installments from January 2021 to October 2023. The agreement also provides PDL with the potential for two additional contingent payments totaling $3.25 million.

Together with their advisors, Torreya and SVB Leerink, PDL’s board of directors and management team evaluated a number of potential transactions to maximize shareholder value for Noden. Torreya was retained by PDL to explore potential strategic transactions and assist in the disposition of Noden, while SVB Leerink has been engaged by PDL to advise on overall liquidation and distribution strategies. PDL believes the transaction with Stanley Capital represents the greatest total value and certainty of completion. The transaction is expected to close in August 2020 and is subject to certain customary closing conditions.

"We also wish to thank Jill Jene, our Vice President Business Development, for her dedication, leadership and highly valued contributions as a key member of the management team and for guiding PDL’s business development efforts for the past two years," added Mr. Monnet. "Jill and her team have been instrumental in the execution of our monetization process. We will enter into a separation agreement with Jill, who will be departing PDL in August 2020. We wish her success in her future endeavors."