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PDL BioPharma Reschedules Third Quarter 2020 Financial Results and Business Update

On November 6, 2020 PDL BioPharma, Inc. ("PDL" or "the Company") (Nasdaq: PDLI) reported it has rescheduled the date for announcing third quarter 2020 financial results and holding a conference call to Wednesday, November 11, 2020 (Press release, PDL BioPharma, NOV 6, 2020, View Source [SID1234570168]). PDL will issue its third quarter 2020 financial results news release on November 11 after market close, and PDL’s management will host a conference call and webcast that same day at 4:30 p.m. Eastern time to discuss those financial results and provide an update on its progress in monetizing the Company’s assets. A slide presentation to accompany the call will be available via the webcast link on the PDL website at View Source

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As a result of the approval of the Company’s stockholders on August 19, 2020 to pursue dissolution of the Company, PDL’s basis of accounting transitioned effective September 1, 2020 from the going concern basis of accounting to the liquidation basis of accounting in accordance with U.S. Generally Accepted Accounting Principles. Activities associated with the third quarter earnings release are being rescheduled to November 11 due to the time needed to work through the complexities of switching to the liquidation basis of accounting.

Previously these activities were scheduled for Monday, November 9. Please note that if you pre-registered for the November 9 conference call, as described below, you will receive an updated calendar invitation via email from the conference call vendor with the new date and time. Please accept that invitation and there is no need to pre-register again.

Conference Call Details
We encourage participants to pre-register for the conference call using the following link: View Source Callers who pre-register will be given a conference passcode and unique PIN to gain immediate access to the call and bypass the live operator. Participants may pre-register at any time, including up to and after the call start time.

Those who choose not to pre-register can access the live conference by dialing (833) 685-0901 from the United States or (412) 317-5734 internationally. The conference ID is 10149211. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (877) 344-7529 from the United States, (855) 669-9658 from Canada or (412) 317-0088 internationally. The replay passcode is 10149211.

To access the live and subsequently archived webcast of the conference call, go to "Events & Presentations" on the Company’s website. Please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

Mustang Bio Reports Third Quarter 2020 Financial Results and Recent Corporate Highlights

On November 6, 2020 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported financial results and recent corporate highlights for the third quarter ended September 30, 2020 (Press release, Mustang Bio, NOV 6, 2020, View Source [SID1234570167]).

"Mustang had an eventful third quarter on the regulatory front, as the U.S. Food and Drug Administration ("FDA") granted Rare Pediatric and Orphan Drug Designations to both of our gene therapy product candidates for the treatment of X-linked severe combined immunodeficiency ("XSCID"), MB-107, for newly diagnosed infants, and MB-207, for patients previously treated with hematopoietic stem cell transplantation ("HSCT") and for whom re-treatment is indicated," said Manuel Litchman, M.D., President and Chief Executive Officer of Mustang.

Dr. Litchman continued, "We are also pleased to report progress across our CAR T cell therapy programs during the third quarter and subsequent period. Most importantly, as recently reported, we have observed compelling efficacy without CAR T related toxicity in the first 4 non-Hodgkin lymphoma patients treated with MB-106, a CD20-targeted CAR T cell therapy, following a major revision in the cell manufacturing process at the Fred Hutch Cancer Research Center ("Fred Hutch"). We also dosed the first patient in a Phase 1/2 clinical trial of MB-102, a CD123-targeted CAR T cell therapy, under our own Investigational New Drug ("IND") application for relapsed or refractory blastic plasmacytoid dendritic cell neoplasm, acute myeloid leukemia and high-risk myelodysplastic syndrome. Finally, we are encouraged by the significant response to MB-105, a prostate stem cell antigen ("PSCA")-targeted CAR T therapy, reported by City of Hope in a 73-year-old patient with PSCA-positive metastatic castrate-resistant prostate cancer. This patient, who had failed eight prior therapies, experienced a 94 percent reduction in prostate-specific antigen, with a near complete reduction of measurable soft tissue metastasis by computerized tomography and improvement in bone metastases by magnetic resonance imaging. We look forward to achieving additional milestones in the coming months, as we expect to disclose additional promising clinical data from our MB-106 program at the American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") Annual Meeting next month. In addition, next quarter we expect to enroll the first patient on our pivotal MB-107 lentiviral gene therapy trial for newly diagnosed infants with XSCID and to file an IND for our pivotal MB-207 lentiviral gene therapy trial for previously transplanted patients with XSCID."

Recent Corporate Highlights:

In August 2020, Mustang announced that the FDA granted Rare Pediatric Disease Designation to MB-107, a lentiviral gene therapy for the treatment of XSCID, also known as bubble boy disease, in newly diagnosed infants and to MB-207, a lentiviral gene therapy for the treatment of patients with XSCID who have been previously treated with HSCT and for whom re-treatment is indicated.
In September 2020, the FDA granted Orphan Drug Designation to MB-107 for the treatment of XSCID in newly diagnosed infants and to MB-207 for the treatment of patients with XSCID who have been previously treated with HSCT and for whom re-treatment is indicated.
In October 2020, Mustang announced that the first patient had been dosed in a company-sponsored, open label, multicenter Phase 1/2 clinical trial to evaluate the safety and efficacy of MB-102 (CD123-targeted CAR T cell therapy) in patients with relapsed or refractory blastic plasmacytoid dendritic cell neoplasm (BPDCN), acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (hrMDS).
In October 2020, Mustang licensed LentiBOOST technology from SIRION Biotech GmbH for the development of MB-207.
In October 2020, City of Hope presented initial Phase 1 data on MB-105, a PSCA-targeted CAR T administered systemically to patients with PSCA-positive mCRPC, at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat.
Earlier this month, Mustang announced that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas, were selected for a poster presentation at the 62nd ASH (Free ASH Whitepaper) Annual Meeting. A link to the abstract can be found here.
Financial Results:

As of September 30, 2020, the company’s cash, cash equivalents and restricted cash totaled $76.3 million, compared to $86.4 million as of June 30, 2020, and $62.4 million as of December 31, 2019.
Research and development expenses were $8.0 million for the third quarter of 2020. This compares to $7.3 million for the third quarter of 2019. Non-cash, stock-based compensation expenses included in research and development were $0.3 million for the third quarter of 2020, compared to $0.7 million for the third quarter of 2019.
Research and development expenses from license acquisitions totaled $0.3 million for the third quarter of 2020, compared to $0.7 million for the third quarter of 2019.
General and administrative expenses were $2.2 million for the third quarter of 2020. This compares to $2.0 million for the third quarter of 2019. Non-cash, stock-based compensation expenses included in general and administrative expenses were $0.3 million for the third quarter of 2020, compared to $0.4 million for the third quarter of 2019.
Net loss attributable to common stockholders was $13.0 million, or $0.23 per share, for the third quarter of 2020, compared to a net loss attributable to common stockholders of $10.2 million, or $0.25 per share, for the third quarter of 2019.

Validation of the SRG Rat for Human Tumor Studies Published in PLOS ONE

On November 6, 2020 Hera BioLabs reported that a validation study using the SRG rat engrafted with human tumors (OncoRat) has been published in PLOS ONE (Press release, Hera BioLabs, NOV 6, 2020, View Source [SID1234570164]).

Specifically, the paper validated the use of the OncoRat in human tumor oncology studies. The paper, titled "The SRG rat, a Sprague-Dawley Rag2/Il2rg double-knockout validated for human tumor oncology studies," is a collaboration among researchers working at Hera, the University of Michigan, the University of Kentucky, and others.

The data presented in PLOS One represent years of research that Hera is excited to share with the scientific community.

This research was led by Fallon K. Noto, Ph.D. Executive Director, R&D/In Vivo Services, and many members of the Hera team including Valeriya Steffey, and Bisoye Towobola DMV, and Sam Moody and in collaboration with Goutham Narla, M.D., Ph.D. at the University of Michigan.
Current xenograft models
In vivo oncology models are an essential step in preclinical research for understanding tumor biology and evaluating promising therapeutic agents prior to testing in patients. Patient derived xenograft (PDX) models, primarily performed in mice, have been a huge step forward for oncology research by more closely recapitulating the patient’s tumor architecture and molecular signature throughout the experiment.

To avoid rejection of tumor xenografts, immunodeficient hosts are required. NSG mice in particular have been used in PDX studies, which have knockout mutations of the Prkdc and Il2rg genes, making the mice B, T, and NK cell deficient.

However, immunodeficient mice have limitations for precision medicine and efficacy testing applications for many tumor types. These limitations include:
Low engraftment rates
Slow tumor growth
Small tumor volume
Limited capacity for blood collection for PK/PD analysis
The SRG rat, an improved human xenograft tumor host
The SRG rat was created to add to the oncology toolbox, with improved take rates and growth kinetics for many tumor types while utilizing the rat’s larger size and robust nature to obtain more tissue and blood for important translational analyses.

Through deletion of the Rag2 and Il2rg genes on the Sprague-Dawley rat background, the SRG rat is fully SCID and lacks mature B cells, T cells and NK cells.

In a small pilot study, Hera demonstrated that the SRG rat can establish VCaP tumors, a human prostate cancer cell line, much faster than NSG mice. One advantage of the SRG rat over comparable mouse models is the ability to collect serial blood samples for biomarker analysis, as we have shown with Prostate Specific Antigen (PSA) in VCaP tumor-bearing rats.

In addition, the SRG rat is valuable for establishing banks of PDX samples from patient tissue. In a non-small cell lung cancer (NSCLC) study, the NSCLC-PDX engraftment rates in SRG rat were 78%, whereas mouse models fall between 20-40%. Importantly, the PDX models maintain genomic stability across multiple passages.

The data presented in PLOS One emphasize the value of the SRG rat rat as a model for preclinical oncology studies. The SRG rat is an excellent host for human cancer cell lines, PDX modeling and offers the benefit of serial blood sampling and larger tissue mass for downstream molecular analysis.

The SRG rat as used in oncology (OncoRat) holds promise in personalized medicine, by serving as a patient avatar for guiding genetically based precision therapies!
Learn more about the exciting work at Hera
The OncoRat is just one of the many exciting innovations going on at Hera. These breakthroughs are possible through our proprietary gene editing systems: CasCLOVER and piggyBac.

Advancing on the CRISPR-Cas9 platform, Cas-CLOVER uses a dimeric nuclease system to perform site directed mutagenesis with no detectable off-target effects.

A perfect complement to Cas-CLOVER, piggyBac is our transposase system that can insert genes in a "footprint free" manner, meaning only the gene of interest is inserted into the host genome.

Genprex cancer gene therapy paired with AZ, Merck lung cancer drugs

On November 6, 2020 An experimental gene therapy developed by Texas biotech Genprex reported that it will be paired with AstraZeneca’s Tagrisso and Merck & Co’s Keytruda – both leading their respective drug classes in the treatment of non-small cell lung cancer (NSCLC) (Press release, Genprex, NOV 6, 2020, View Source [SID1234570163]).

The two phase 1/2 trials are zeroing in on NSCLC patients with specific molecular biomarkers, to see if adding Genprex’ Reqorsa (quaratusugene ozeplasmid) – which delivers a gene that suppresses tumour growth – can enhance the activity of the AZ and Merck drugs.

The first trial, called Acclaim-1, will pair EGFR inhibitor Tagrisso (osimertinib) with Reqorsa as a second-line treatment for EGFR-mutated NSCLC patients whose cancer has progressed after first-line Tagrisso treatment.

The Acclaim-2 trial meanwhile will add Reqorsa to PD-1 inhibitor Keytruda (pembrolizumab) in NSCLC patients with PD-L1 expression levels of 1% to 49%, according to the partners. Both studies are due to start in the first half of next year.

Tagrisso is the top-selling EGFR inhibitor, with sales of almost $3.2 billion last year, while Keytruda dominates the market for cancer immunotherapies for NSCLC, accounting for a large chunk of its $11 billion-plus 2019 sales tally.

If positive, the trials would allow Reqorsa to piggyback on that success – assuming it makes it to market. So far, Genprex only has data for the therapy from two phase 1 trials, and part of a phase 2 study showing preliminary evidence of safety as well as efficacy in NSCLC.

Genprex’ therapy takes the form of a copy of the TUSC2 gene in a non-viral lipid nanoparticle formulation, delivered via an intravenous infusion, and was originally developed at the University of Texas’ MD Anderson Cancer Centre in the US.

After infusion, the nanoparticles are taken up by tumour cells, says Genprex, and leads to the expression of the TUSC2 gene into a protein that reprograms them to die.

In preclinical studies, the gene therapy also seems to block mechanisms involved in drug resistance, including TIM3, a bypass pathway that can lead to failure of PD-1-based therapy.

The FDA awarded fast-track status to the Tagrisso/Reqorsa for the treatment of EHGFR-mutated NSCLC earlier this year, as nearly all patients receiving AZ’s drug eventually experience disease progression.

According the company’s data, the median time to progression after first-line Tagrisso is about 18 months.

Austin-based Genprex has been preparing for the Reqorsa clinical trials programme, over the last few months, ramping up its manufacturing capacity for the gene therapy via an agreement with contract manufacturing organisation (CMO) Aldevron.

GLYCOMIMETICS REPORTS HIGHLIGHTS AND FINANCIAL RESULTS FOR THIRD QUARTER 2020

On November 6, 2020 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results for the third quarter ended September 30, 2020, and highlighted recent company events (Press release, GlycoMimetics, NOV 6, 2020, View Source [SID1234570161]). Cash and cash equivalents at September 30, 2020 were $142.9 million.

"During the third quarter, both late-stage trials of uproleselan – the Company-sponsored Phase 3 trial in relapsed/refractory AML patients as well as the NCI’s study in newly-diagnosed AML patients fit for chemotherapy – progressed in the face of the COVID-19 pandemic, thanks in significant part to the dedicated efforts of clinicians, statisticians and sites. We continue to project completion of enrollment in our own Phase 3 trial in the second half of 2021," said Rachel King, Chief Executive Officer. "In sickle cell disease, new post hoc analyses of the rivipansel Phase 3 study provide additional perspective on the potential of our targeted E-selectin product candidates in early vaso-occlusive crisis. We have presented findings at multiple sickle cell congresses, and at the upcoming ASH (Free ASH Whitepaper) meeting, we plan to share key secondary endpoint, subgroup and subset data. We believe these data provide a foundation for our ongoing evaluation of potential opportunities in our pipeline for the treatment of acute vaso-occlusive crisis, or VOC."

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 earlier in the year by the COVID-19 pandemic, in this quarter patient enrollment returned to forecasted rates.
● GlycoMimetics reiterated its guidance that completion of enrollment for its trial was expected in the second half of 2021.
●New preclinical studies support the use of uproleselan with venetoclax and a hypomethylating agent (HMA) in AML:
oAt the upcoming ASH (Free ASH Whitepaper) meeting in December 2020, GlycoMimetics will make an oral presentation of preclinical data from a study in an AML mouse model that shows the potential benefit of a combination therapy of uproleselan with venetoclax and HMA.
oAt the virtual meeting of the Society of Hematologic Oncology (SOHO) in September 2020, GlycoMimetics presented preclinical data showing a statistically significant prolongation of survival in a patient-derived xenograft (PDX) model.
Rivipansel

●At the Foundation for Sickle Cell Disease Research (FSCDR) virtual meeting held in September 2020, GlycoMimetics presented for the first time new efficacy and biomarker data from the post hoc analysis of the Phase 3 RESET trial that showed statistically significant improvements for patients treated early in crisis (within 26.4 hours of onset of pain) in the primary efficacy endpoint of time to readiness for discharge compared to placebo. This primary endpoint analysis demonstrated p=0.03, and median improvement of 56.3 hours compared to placebo.
●At the Annual Scientific Conference on Sickle Cell and Thalassaemia (ASCAT) in October 2020, a GlycoMimetics’ poster highlighted new pediatric and other key secondary endpoint subset/subgroup efficacy and biomarker data from the Phase 3 RESET trial.
●Accepted for oral presentation at the ASH (Free ASH Whitepaper) meeting is an abstract also presenting pediatric and secondary endpoint data from the post hoc analysis of the Phase 3 RESET trial. These data as well as biomarker data show the potential benefits conferred when rivipansel is used to treat patients early in the VOC pain crisis.
●FDA granted GlycoMimetics a Rare Pediatric Disease designation for rivipansel for treatment of sickle cell disease.
●Based upon its review of the emerging Phase 3 rivipansel data set, GlycoMimetics is engaging with the FDA to identify what, if any, next steps to take, with a focus on determining if there is a potential streamlined path forward for this product candidate in sickle cell disease.
GMI-1687

●Building on clinical data for rivipansel disclosed at the FSCDR meeting in September and at ASCAT in October, GlycoMimetics also gave oral presentations at the FSCDR and ASCAT meetings reporting on preclinical data highlighting GMI-1687 in animal models of VOC. The data demonstrated its potential efficacy as a subcutaneously administered treatment for VOC to prevent sickle red blood cell adherence to inflamed vasculature, inhibit vessel occlusion and restore normal blood flow.
●An abstract was accepted for oral presentation at the ASH (Free ASH Whitepaper) meeting in December 2020 on the product candidate’s potential for intravenous and subcutaneous administration to restore blood flow. A mouse model of VOC sickle cell disease will be highlighted.
Third Quarter 2020 Financial Results:

●Cash position: As of September 30, 2020, GlycoMimetics had cash and cash equivalents of $142.9 million as compared to $158.2 million as of December 31, 2019. During the quarter, the Company received a $1 million clinical development milestone from Apollomics pursuant to the Company’s collaboration and license agreement for the development and commercialization of uproleselan and GMI-1687 in Mainland China, Hong Kong, Macau and Taiwan.
●R&D Expenses: The Company’s research and development expenses were $10.7 million for each of the quarters ended September 30, 2020 and 2019. The Company’s research and development expenses decreased to $33.2 million for the nine months ended September 30, 2020 as compared to $35.6 million for the same period in 2019. Manufacturing and formulation expenses decreased in the three and nine months ended September 30, 2020 as compared to the same periods in 2019 as a result of lower raw material costs purchased in 2020. These decreases were offset by higher clinical expenses due to 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 in 2020 as compared to 2019. Contract research services, consulting and other costs were lower in the three and nine months ended September 30, 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 $4.1 million for the third quarter ended September 30, 2020 as compared to $3.4 million for the third quarter of 2019. General and administrative expenses for the nine months ended September 30, 2020 increased to $12.7 million as compared to $10.5 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 the three and nine months ended September 30, 2020, as compared to the same periods in 2019, due to lower travel, meals and conference registration expenses as a result of travel restrictions imposed during the COVID-19 pandemic.
Shares Outstanding: Shares of common stock outstanding as of September 30, 2020 were 47,828,831
The Company will host a conference call and webcast today at 8:30 a.m. ET. The conference call will be broadcast live in listen-only mode on the "Investors" tab of the Company’s website at View Source For those who wish to ask questions, 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 3073766. 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 3073766.

About Uproleselan (GMI-1271)

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 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 Rivipansel

Rivipansel, the Company’s wholly-owned 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 and subsequently presented at the Foundation for Sickle Cell Disease Research Meeting in September 2020. GlycoMimetics is engaging with the FDA to identify what, if any, next steps to take, with a focus on determining if there is a potential streamlined path forward for this asset in sickle cell disease.

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. At the 2018 Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), data presented in a poster about GMI-1687 pointed to the potential for a life-cycle extension for GlycoMimetics’ uproleselan. The investigational drug has also been shown to represent a more highly-potent and subcutaneously bioavailable potential life-cycle extension for rivipansel.