On November 6, 2020 Epizyme, (Nasdaq: EPZM), a fully integrated, commercial-stage biopharmaceutical company developing and delivering novel epigenetic therapies, reported third quarter 2020 financial results (Press release, Epizyme, NOV 6, 2020, View Source [SID1234570169]).

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"2020 has been a year of big milestones for Epizyme, and we are executing our TAZVERIK launches well, given the unprecedented challenges presented by the COVID-19 pandemic on FL patients’ access to their physicians and on access to our customers," said Robert Bazemore, president and chief executive officer of Epizyme. "While we acknowledge the impact the COVID-19 dynamics have had on the initial uptake in FL, we are reaching both the ES and FL patient populations expected based on our label, and our view of the value proposition of TAZVERIK is unchanged. We believe that the combination of our commercial and clinical performance, and strong balance sheet, sets us up for a positive future."

TAZVERIK (tazemetostat) Program Updates

Execution of TAZVERIK U.S. Commercialization in Relapsed or Refractory Follicular Lymphoma (FL) and Epithelioid Sarcoma (ES): TAZVERIK generated net product revenue in both ES and FL of $3.4 million in the third quarter, with growth over the second quarter of 2020 of 55% largely driven by the U.S. Food and Drug Administration (FDA) approval of TAZVERIK in FL on June 18, 2020. During the third quarter, the COVID-19 pandemic continued to negatively impact FL patient visits to physicians, new patient starts across all lines of treatment, as well as the ability of Epizyme’s field-based teams to fully access FL prescribers. Epizyme reports that new prescriptions for TAZVERIK in FL have increased month over month and are being written for both EZH2 mutation and wild-type patients; in the academic and community settings; and across multiple treatment lines in relapsed or refractory patients. In addition, payor coverage for ES and FL has been in-line with the TAZVERIK label. Epizyme continues to adapt its commercial strategy to the COVID-19 pandemic to support increased adoption of TAZVERIK in appropriate patients.
Two Lancet Oncology Publications on TAZVERIK Phase 2 Data in ES and FL: In October 2020, The Lancet Oncology published results of the company’s Phase 2 trial cohorts evaluating TAZVERIK for the treatment of ES and relapsed or refractory FL, which supported the accelerated approvals by the FDA for both indications in 2020.
Confirmatory Trials for ES and FL On-Track: Enrollment in the safety run-in portions of Epizyme’s two confirmatory trials of TAZVERIK is on track to be completed in 2020, followed by initiation of the efficacy portions in early 2021. The ES confirmatory trial is evaluating TAZVERIK in combination with doxorubicin compared with doxorubicin plus placebo as a front-line treatment for ES, and the FL confirmatory trial is evaluating TAZVERIK in combination with "R2" (Revlimid plus Rituxan) compared with R2 plus placebo in the second-line treatment setting in patients with FL.
Tazemetostat Expansion Program Updates

Advancing to Efficacy Portion of Combination Trial in Metastatic Castration-Resistant Prostate Cancer (mCRPC): Epizyme completed enrollment in the safety run-in portion of its combination study in mCRPC, and initiation of the efficacy expansion stage is planned for early 2021. Epizyme anticipates reporting safety and efficacy data from the safety run-in portion of the study at a medical meeting in 2021.
Upcoming Presentations at ASH (Free ASH Whitepaper)

Data in FL to be Presented at 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition: Epizyme will present data from its FL development program during multiple poster sessions at ASH (Free ASH Whitepaper), which will be held virtually December 5-8, 2020:
Title: Tazemetostat is Associated with Lower Risk for Safety Outcomes Versus the PI3-Kinases Idelalisib, Duvelisib and Copanlisib, in Patients with Relapsed/Refractory Follicular Lymphoma Who Have Received at Least 2 Prior Systemic Treatments: a Matching-Adjusted Indirect Comparison of Single-Arm Trials
Session Name: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma—Clinical Studies: Poster II
Date: Sunday, December 6, 2020
Title: A Phase 1b/3 Randomized, Double-Blind, 3-Stage Study of Tazemetostat or Placebo Plus Lenalidomide and Rituximab in Patients with Relapsed/Refractory Follicular Lymphoma
Session Name: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma—Clinical Studies: Poster II
Date: Sunday, December 6, 2020
Title: Analyzing Efficacy Outcomes from the Phase 2 Study of Single-Agent Tazemetostat as Third-Line Therapy in Patients with Relapsed or Refractory Follicular Lymphoma to Identify Predictors of Response
Session Name: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma-Clinical Studies: Poster II
Date: Sunday, December 6, 2020
Title: A Phase 2, Open-Label, Multicenter Study of Tazemetostat in Combination with Rituximab for the Treatment of Relapsed or Refractory Follicular Lymphoma
Session Name: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma-Clinical Studies: Poster III
Date: Monday, December 7, 2020
Leadership Update

Organization Strengthened with Strategic Promotions: In October 2020, Matthew Ros was promoted to executive vice president and chief strategy and business officer of Epizyme and Vicki Vakiener was promoted to chief commercial officer. As part of these promotions, Mr. Ros is overseeing execution of Epizyme’s long-term corporate strategy. Ms. Vakiener is overseeing commercial execution of TAZVERIK in ES and FL.
Third Quarter 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $279.9 million as of September 30, 2020, as compared to $322.1 million as of June 30, 2020.
Revenue: Total revenue for the third quarter of 2020 was $3.6 million, comprised of $3.4 million in net sales of TAZVERIK in the U.S. and $0.1 million in collaboration revenue, compared to $2.5 million in the second quarter of 2020, comprised of $2.2 million in net sales of TAZVERIK in the U.S. and $0.2 million in collaboration revenue.
Cost of sales: Cost of sales were $1.6 million for the third quarter of 2020, which reflects the costs of TAZVERIK units sold, amortization of intangible assets and third-party royalties on net product revenue.
Operating Expenses: Total GAAP operating expenses were $57.9 million for the third quarter of 2020, compared to $60.0 million for the second quarter of 2020. Total non-GAAP adjusted operating expenses were $50.2 million for the third quarter of 2020, compared to $50.9 million for the second quarter of 2020.
R&D expenses: GAAP R&D expenses were $25.7 million for the third quarter of 2020, compared to $26.4 million for the second quarter of 2020. Non-GAAP adjusted R&D expenses were $23.5 million for the third quarter of 2020, compared to $23.4 million for the second quarter of 2020.
SG&A expenses: GAAP SG&A expenses were $30.6 million for the third quarter of 2020, compared to $32.7 million for the second quarter of 2020. Non-GAAP adjusted SG&A expenses were $26.2 million for the third quarter of 2020, compared to $27.1 million for the second quarter of 2020.
Net Loss (GAAP): Net loss attributable to common stockholders was $56.1 million, or $0.55 per share, for the third quarter of 2020, compared to $58.5 million, or $0.58 per share, for the second quarter of 2020.
Updated Financial Guidance

Agreement with Pharmakon Advisors to Expand Loan Facility Extends Operating Runway: Epizyme and Pharmakon Advisors, an affiliate of Royalty Pharma, have expanded their original loan agreement established in November 2019, enabling Epizyme to draw down an additional $150 million from the loan facility, subject to customary closing conditions. Based on its current plans and projections, Epizyme believes that its existing cash, cash equivalents and marketable securities combined with the proceeds from the loan facility will fund the company’s operations into at least 2023.
Updated 2020 Operating Expenses: Epizyme updated its guidance for full year non-GAAP adjusted cash operating expenses to between $215 million to $235 million, from the previous $235 million to $255 million. The change is due primarily to a reduction in travel and other expenses and a change in how the company’s manufacturing expenses are recorded with a commercial product, partially offset by an increase in commercial-related expenses to address COVID-19 related challenges. A reconciliation of the non-GAAP financial measures to the most directly comparable GAAP financial measures is presented in the table attached to this press release.
Conference Call Information

Epizyme will host a conference call today, November 6, at 8:00 a.m. ET. To participate in the conference call, please dial (877) 844-6886 (domestic) or (970) 315-0315 (international) and refer to conference ID 9556565. A webcast will be available in the investor section of the company’s website at www.epizyme.com, and will be archived for 60 days following the call.

About Non-GAAP Financial Measures

In addition to financial information prepared in accordance with the U.S. generally accepted accounting principles (GAAP), this press release includes the following non-GAAP financial measures: total non-GAAP adjusted operating expenses on a historical and projected basis, non-GAAP R&D expenses on a historical basis and non-GAAP SG&A expenses on a historical basis. Epizyme derives these non-GAAP financial measures by excluding certain expenses and other items from the respective GAAP financial measure, that is most directly comparable to each non-GAAP financial measure. Specifically, the non-GAAP financial measures exclude stock-based compensation expense, amortization or depreciation of intangibles and milestone payments related to TAZVERIK that are payable under the company’s collaboration agreement with Eisai Pharmaceuticals. The company’s management believes that these non-GAAP financial measures are useful to both management and investors in analyzing its ongoing business and operating performance. Management does not intend the presentation of these non-GAAP financial measures to be considered in isolation or as a substitute for results prepared in accordance with GAAP, but as a complement to provide greater transparency. In addition, these non-GAAP financial measures may differ from similarly named measures used by other companies. A quantitative reconciliation of projected non-GAAP adjusted operating expenses to total operating expenses is not available without unreasonable effort primarily due to the company’s inability to predict with reasonable certainty the amount of future stock-based compensation expense.

About TAZVERIK

TAZVERIK is a methyltransferase inhibitor indicated for the treatment of:

Adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.
Adult patients with relapsed or refractory follicular lymphoma whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least two prior systemic therapies.
Adult patients with relapsed or refractory follicular lymphoma who have no satisfactory alternative treatment options.
These indications are approved under accelerated approval based on overall response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

The most common (≥20%) adverse reactions in patients with epithelioid sarcoma are pain, fatigue, nausea, decreased appetite, vomiting and constipation. The most common (≥20%) adverse reactions in patients with follicular lymphoma are fatigue, upper respiratory tract infection, musculoskeletal pain, nausea and abdominal pain.

View the U.S. Full Prescribing Information here: Epizyme.com

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.

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]).

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"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.

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.

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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.

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]).

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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.