On August 5, 2019 Precigen, Inc., a wholly-owned subsidiary of Intrexon Corporation (NASDAQ: XON) and a biopharmaceutical company specializing in the development of innovative gene and cellular therapies to improve the lives of patients, reported that the first patient has been dosed with Precigen’s PRGN-3005, a first-in-class investigational therapy using Precigen’s UltraCAR-T therapeutic platform (Press release, Intrexon, AUG 5, 2019, View Source [SID1234538124]). PRGN-3005 UltraCAR-T is an autologous chimeric antigen receptor T (CAR-T) cell therapy manufactured using non-viral gene delivery and is under investigation for the treatment of patients with advanced, recurrent platinum resistant ovarian, fallopian tube or primary peritoneal cancer (clinical trial identifier: NCT03907527).

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PRGN-3005 utilizes Precigen’s transformative UltraCAR-T therapeutic platform, which eliminates ex vivo expansion and reduces manufacturing time to allow for rapid next day administration of UltraCAR-T cells following non-viral gene transfer. PRGN-3005 UltraCAR-T is a multigenic CAR-T cell investigational therapy utilizing Precigen’s advanced non-viral gene delivery system to co-express a chimeric antigen receptor, membrane-bound interleukin‐15 (mbIL15), and a kill switch for better precision and control.

"This is an important milestone in our efforts to develop a new treatment option for patients with ovarian cancer," said Helen Sabzevari, PhD, President of Precigen. "With the first ovarian cancer patient dosed with Precigen’s PRGN-3005 UltraCAR-T investigational therapy, we remain steadfast in our goal of delivering critical new therapies to solid tumor patients with high unmet need."

Conducted in collaboration with the University of Washington and Fred Hutchinson Cancer Research Center, the PRGN-3005 UltraCAR-T clinical study is an open-label, first-in-human Phase 1 dose escalation study to evaluate the safety and maximal tolerated dose of PRGN‐3005 UltraCAR-T delivered by intraperitoneal infusion (IP) or intravenous infusion (IV). The study population includes patients with advanced stage (III/IV) recurrent ovarian, fallopian tube, and primary peritoneal cancer who are platinum-resistant and have progressed after receiving standard-of-care therapies or are not eligible to receive available therapies with known clinical benefit.

"Many women with ovarian, fallopian tube and primary peritoneal cancer have historically poor outcomes," said Mary L. (Nora) Disis, MD, faculty member at the University of Washington and Fred Hutchinson Cancer Research Center and one of the lead investigators for the PRGN-3005 study. "Dosing the first patient with the PRGN-3005 UltraCAR-T represents a potentially significant development for the use of CAR-T cell therapies in solid tumors."

About Ovarian Cancer
Worldwide, nearly 300,000 women are diagnosed with ovarian cancer every year1 with approximately 22,000 of them in the US2. Since early ovarian cancer is often without obvious symptoms, the disease is frequently diagnosed at an advanced stage where cancer has spread to distant parts of the body, such as the liver or lungs2,3. Five-year survival rates depend on stage and type of ovarian cancer with rates decreasing for advanced stage cancers that have spread to distant parts of the body3.

Precigen : Advancing Medicine with Precision
Precigen is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cellular therapies using precision technology to target the most urgent and intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Precigen also follows the science opportunistically in pursuit of promising programs in emerging therapeutics. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. Precigen was founded as a wholly-owned subsidiary of Intrexon Corporation (NASDAQ: XON) and leverages a diverse portfolio of technology platforms to advance human health. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

Precigen’s UltraCAR-T Therapeutic Platform
Precigen’s UltraCAR-T platform has the potential to disrupt the CAR-T treatment landscape by increasing patient access through shortening manufacturing time, decreasing manufacturing-related costs, and improving outcomes using advanced approaches for precise tumor targeting and control of the immune system. The platform brings several key advancements: 1) Non-viral gene transfer using multigenic vectors for expression of multiple effector genes leads to better precision and control of tumor targeting and eliminates the need for virus; 2) Sustained persistence and desired phenotype of infused UltraCAR-T helps address T-cell exhaustion, a common issue with current CAR-T therapies; 3) T-cell control by incorporation of kill switch technology to potentially improve the safety profile; and 4) Rapid manufacturing of UltraCAR-T cells using our proprietary non-viral gene transfer process, which eliminates the need for ex vivo propagation, thus dramatically reducing wait times for patients from weeks to one day after gene transfer.

Safe Harbor Statement
Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to plans, objectives and expectations for the development of our business, including the timing and progress of preclinical and clinical trials and discovery programs. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.

On August 5, 2019 UroGen Pharma Ltd. (Nasdaq:URGN) reported that it will report second quarter 2019 financial results on Friday, August 9, 2019, prior to the open of the market (Press release, UroGen Pharma, AUG 5, 2019, View Source [SID1234538142]). The announcement will be followed by a live audio webcast and conference call on Monday, August 12, 2019 at 8:30AM Eastern Time.

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Audio Webcast

The webcast will be made available on the Investors section of the Company’s website at View Source Following the live audio webcast, a replay will be available on the Company’s website for approximately two weeks.

Dial-In Information

Live (U.S. / Canada): 1 (888) 771-4371
Live (International): 1 (847) 585-4405
Confirmation number: 48773603

On August 5, 2019 Arbutus Biopharma Corporation (Nasdaq: ABUS), an industry-leading Hepatitis B Virus (HBV) therapeutic solutions company, reported its second quarter 2019 financial results and provides a corporate update (Press release, Arbutus Biopharma, AUG 5, 2019, View Source [SID1234538127]).

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"I am excited to join Arbutus at this important inflection point as we look to advance our two lead compounds, AB-506 and AB-729, through Phase1a/1b clinical trials," said William Collier, Arbutus’ President and Chief Executive Officer. "Provided these compounds progress as expected, we anticipate moving into a combination proof-of-concept Phase 2 clinical trial in subjects with chronic hepatitis B in the second half of 2020. We believe a combination regimen that includes several different mechanisms of action will be required to improve upon the existing standard-of-care in HBV."

Recent Clinical Accomplishments and Key Corporate Accomplishments

New President & Chief Executive Officer

William H. Collier was appointed President and Chief Executive Officer of Arbutus and a member of the Board of Directors effective June 24th. Mr. Collier’s appointment filled the vacancy created by the retirement of Mark J. Murray, Ph.D., as Arbutus’ President and Chief Executive Officer, which was effective June 23rd. Mr. Collier has over 30 years of experience as a senior executive in the pharmaceutical industry and previously served as President and General Manager, North America at ViiV Healthcare. At ViiV, he oversaw the industry-leading launches of several new treatments for HIV. Prior to joining ViiV in 2009, Mr. Collier held multiple senior leadership roles at GlaxoSmithKline. Earlier in his career he led the launches of new treatments for herpes and bacterial infections. Mr. Collier received his BSc in Mathematics and Management Sciences from the University of Manchester Institute of Science & Technology, UK, and served on The President’s Advisory Council on HIV/AIDS from 2014 to 2017.
AB-506

In July 2019, Arbutus announced preliminary results from a Phase 1a/1b clinical trial demonstrating that AB-506 is a potent oral capsid inhibitor. These preliminary Phase 1a/1b results support the Company’s confidence in its potential to significantly contribute to the inhibition of HBV replication in a curative combination regimen.

Arbutus expects that safety and efficacy data from this portion of the Phase 1a/1b trial, as well as results from a planned Phase 1 28-day clinical trial in healthy subjects, will be submitted to an appropriate scientific meeting later this year.

Arbutus is planning on dosing additional cohorts and final results of this Phase 1a/1b trial, which are expected in the first half of 2020, will inform next steps toward the combination proof-of-concept Phase 2 clinical trial in subjects with chronic hepatitis B.
AB-729

In July 2019, the Company initiated the healthy subject portion of a single and multiple dose Phase 1a/1b clinical trial for AB-729, a subcutaneously delivered RNAi agent which has been shown in preclinical models to span all HBV transcripts, reduce all viral antigens, including hepatitis B surface antigen (HBsAg) expression, and inhibit HBV replication. In this trial, which will investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AB-729 in healthy subjects and subjects with chronic hepatitis B infection, AB-729 will be dosed monthly.

Preliminary safety and efficacy data from both healthy subjects and several single dose cohorts of subjects with chronic hepatitis B infection are expected in the first quarter of 2020.
Early R&D Programs

Arbutus continues a focused discovery effort on follow-on compounds for its current HBV pipeline, including its HBV RNA destabilizer, AB-452, as well as efforts to identify compounds potentially capable of reawakening HBV patients’ immune response such as PD-L1 blockers and HBV-specific targets such as HBV cccDNA.
ONPATTRO Royalty Entitlement

Arbutus has a royalty entitlement on global net sales of ONPATTRO (Patisiran) for the lipid nanoparticle delivery (LNP) technology licensed by Arbutus to Alnylam Pharmaceuticals, Inc. (Alnylam) for this product. ONPATTRO is an RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis that has been approved by the FDA and the EMA. In July 2019, Arbutus sold this royalty entitlement to OMERS, the defined benefit pension plan for municipal employees based in the Province of Ontario, Canada, effective as of January 1, 2019, for $20 million in gross proceeds before advisory fees. OMERS will retain this royalty entitlement until it has received $30 million in royalties, at which point 100% of this royalty entitlement will revert to Arbutus.

In addition to the royalty entitlement from the Alnylam LNP license agreement, Arbutus is also entitled to a second, lower royalty entitlement on global net sales of ONPATTRO originating from a settlement agreement and subsequent license agreement with Acuitas Therapeutics. The royalty entitlement from Acuitas has been retained by Arbutus and is not part of the royalty entitlement sale to OMERS.
Financial Results

Cash, Cash Equivalents and Investments

Arbutus had cash, cash equivalents and short-term investments totaling $95.3 million as of June 30, 2019, as compared to $124.6 million as of December 31, 2018. The decreased cash balance was due primarily to $34.1 million of cash used in operating activities for the six months ended June 30, 2019, partially offset by $4.7 million of net proceeds from the issuance of shares under its ATM program. In July 2019, the Company received $20 million in gross proceeds from the sale of a portion of its royalty entitlement on net sales of ONPATTRO. The Company believes its cash and investments balance is sufficient to fund operations into the second half of 2020.

Operating Expenses

Research and development expenses were $12.8 million in Q2 2019 compared to $16.3 million in Q2 2018. Research and development expenses in 2019 included costs associated with the Company’s Phase 1a/1b clinical trial for its lead capsid inhibitor (AB-506), pre-clinical studies for its RNAi agent (AB-729), and characterization activities for its HBV RNA Destabilizer (AB-452). The decrease in research and development expenses was due primarily to higher costs in 2018 for AB-452, including drug product manufacturing, and expenses in 2018 associated with the Phase 2 clinical trial for AB-1467, partially offset by increased spending in 2019 for the Phase 1a/1b clinical trial for AB-506 and pre-clinical studies for AB-729. General and administrative expenses were $8.2 million in Q2 2019 compared to $3.8 million in Q2 2018. The increase in general and administrative expenses was due primarily to our former President and Chief Executive Officer’s departure from the Company in June 2019. In accordance with the terms of his legacy employment agreement, he received $2.3 million in cash severance and the Company recognized $2.2 million of non-cash stock-based compensation expense for accelerated vesting of his stock options.

Equity investment loss

As of June 30, 2019, the Company owned approximately 40% of the common equity of Genevant Sciences Ltd. (Genevant), a company launched with Roivant Sciences Ltd. in April 2018. Arbutus recorded a loss of $3.3 million in Q2 2019 for its proportionate share of Genevant’s net loss. In Q2 2018, Arbutus recognized a non-cash gain of $24.9 million in connection with the equity interest received by Arbutus upon Genevant’s formation. Financial results of Genevant are recorded on a one-quarter lag basis.

Net Income (Loss)

Net income (loss) attributable to common shares for Q2 2019 was a net loss of $26.1 million ($0.46 basic and diluted loss per common share) as compared to net income of $0.6 million ($0.01 basic and diluted income per common share) for Q2 2018. Net income (loss) attributable to common shares included $2.8 million of non-cash expense in Q2 2019 and $2.5 million in Q2 2018 for the accrual of coupon on the Company’s convertible preferred shares. Net income in Q2 2018 included a non-cash gain of $24.9 million in connection with the equity interest received by Arbutus upon Genevant’s formation.

Outstanding Shares

The Company had approximately 56.9 million common shares issued and outstanding as of June 30, 2019. In addition, the Company had approximately 8.9 million options outstanding and 1.164 million convertible preferred shares outstanding, which (including the annual 8.75% coupon) will be mandatorily convertible into approximately 23 million common shares on October 18, 2021. Assuming the outstanding options and convertible preferred shares were fully converted, the Company would have had approximately 89 million common shares outstanding as of June 30, 2019.

Conference Call Today

Arbutus will hold a conference call and webcast today, Monday, August 5, 2019 at 8:45 AM Eastern Time to provide a corporate update. You can access a live webcast of the call through the Investors section of Arbutus’ website at www.arbutusbio.com. Alternatively, you can dial (866) 393-1607 or (914) 495-8556 and reference conference ID 2098024.

An archived webcast will be available on the Arbutus website after the event. Alternatively, you may access a replay of the conference call by calling (855) 859-2056 or (404) 537-3406, and reference conference ID 2098024.

On August 5, 2019 Eureka Therapeutics, Inc., a clinical stage biopharmaceutical company developing novel T-cell therapies that harness the evolutionary power of the immune system, reported initiation of Phase 1/2 clinical trial of ET140202 ARTEMIS T-Cell therapy in liver cancer at City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, based in Duarte, California (Press release, Eureka Therapeutics, AUG 5, 2019, View Source [SID1234538143]).

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The Phase 1/2 study is a multicenter, open-label, dose-escalating clinical trial of ET140202 ARTEMIS T-Cell therapy for the treatment of advanced hepatocellular carcinoma (HCC), the predominant type of liver cancer.

Patients with advanced HCC have a very poor prognosis and limited treatment options. The rate of liver cancer diagnosis has more than tripled since 1980, according to the American Cancer Society. About 42,030 new cases are expected, and about 31,780 people are expected to die from the disease in 2019.

ET140202 utilizes Eureka’s proprietary ARTEMIS antibody T-cell receptor (AbTCR) platform engineered with a proprietary human TCR-mimic (TCRm) antibody to target an alpha fetoprotein (AFP)-peptide/HLA-A2 complex on HCC cancer cells. Data presented in September 2018 from Eureka’s ongoing proof-of-concept first-in-human study of ET140202 in China demonstrated a favorable safety profile with no observed cytokine release syndrome or drug-related neurotoxicity.

"The novel T cell platform has the potential to transform T cell therapy into an outpatient procedure," said Yuman Fong, M.D., director of the Center for Surgical Innovation at City of Hope and co-investigator of the ongoing clinical trial. "We, Eureka Therapeutics and others are designing T cell therapies with low toxicity."

The patients who are selected for the clinical trial have metastatic or locally advanced, inoperable liver cancer and have progressed or have not been able to tolerate at least one line of treatment for their disease. The clinical trial tests for safety and proper dosing levels. If the initial study participants react favorably, the trial will move into drug efficacy testing.

"Traditional immunotherapy such as checkpoint inhibitors releases the breaks on the immune system, which allows it to sometimes attack the tumor but can also lead to attacks on other parts of the human body," said Daneng Li, M.D., principal investigator of the ongoing study and a medical oncologist at City of Hope. "This approach engineers immune cells to directly attack a protein that is expressed on liver cancer. We are trying to individualize treatment for patients with advanced liver cancer."

"We are pleased to work with City of Hope on bringing ET140202 therapy to patients with advanced HCC. Our approach has been to use our ARTEMIS T-cell platform with higher target specificity to address the safety and other challenges in the treatment of solid tumors," said Dr. Cheng Liu, Founder and CEO of Eureka Therapeutics. "HCC is a devastating disease, and we are delighted that City of Hope is using their broad expertise to potentially accelerate our efforts and provide additional opportunities to combat this and other diseases."

On August 5, 2019 ArQule, Inc. (Nasdaq:ARQL) reported that Dr. Brian Schwartz, Chief Medical Officer and Head of Research and Development, will participate in the BTIG Biotechnology Conference on Monday, August 12, 2019 at The St. Regis New York in New York City (Press release, ArQule, AUG 5, 2019, View Source [SID1234538128]).

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