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Nevro to Present at 39th Annual J.P. Morgan Healthcare Conference on Tuesday, January 12, 2021

On December 15, 2020 Nevro Corp. (NYSE: NVRO), a global medical device company that is providing innovative, evidence-based solutions for the treatment of chronic pain, reported that D. Keith Grossman, Nevro’s Chairman, CEO and President, will present at the 39th Annual J.P. Morgan Healthcare Conference on Tuesday, January 12, 2021, at 4:30 pm Eastern Time / 1:30 pm Pacific Time (Press release, Nevro, DEC 15, 2020, View Source [SID1234572890]).

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A live webcast of this event, as well as an archived recording, will be available in the Investors section of Nevro’s website at www.nevro.com.

Internet Posting of Information

Nevro routinely posts information that may be important to investors in the "Investor Relations" section of its website at www.nevro.com. The company encourages investors and potential investors to consult the Nevro website regularly for important information about Nevro.

Protagonist Therapeutics, Inc. Announces Closing of $115 Million Public Offering of Common Stock and Full Exercise of Underwriters’ Option to Purchase Additional Shares

On December 15, 2020 Protagonist Therapeutics, Inc. (Nasdaq: PTGX), a clinical stage biopharmaceutical company, reported the closing of its previously announced underwritten public offering of 5,476,189 shares of its common stock, including 714,285 shares sold pursuant to the underwriters’ exercise in full of their "green shoe" option to purchase additional shares, at a price to the public of $21.00 per share (Press release, Protagonist, DEC 15, 2020, View Source [SID1234572889]). Aggregate gross proceeds to Protagonist from the offering were approximately $115.0 million, before deducting underwriting discounts and commissions and offering expenses.

J.P. Morgan Securities LLC, SVB Leerink LLC, Piper Sandler & Co. and BMO Capital Markets Corp. are acting as joint book-running managers for the offering.

A shelf registration statement relating to the offered shares of common stock was filed with the Securities and Exchange Commission (SEC) on December 10, 2020. A final prospectus supplement and accompanying prospectus relating to the offering have been filed with the SEC and is available on the SEC’s website, located at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus related to the offering may be obtained, when available, from J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204 or by email at [email protected]; from SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132 or by email at [email protected]; from Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924 or by email at [email protected]; or from BMO Capital Markets Corp., Attention: Equity Syndicate Department, 3 Times Square, 25th Floor, New York, NY 10036, by telephone at (800) 414-3627 or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

Rafael Holdings Reports First Quarter Fiscal Year 2021 Results

On December 15, 2020 Rafael Holdings, Inc., (NYSE: RFL), reported revenue of $1.1 million and a loss per share of $0.09 for the first quarter of its 2021 fiscal year, the three months ended October 31, 2020 (Press release, Rafael Holdings, DEC 15, 2020, View Source [SID1234572888]).

Q1 FY 2021 Consolidated Financial Highlights

Revenue of $1.1 million in Q1 FY 2021, generated by Rafael Holdings’ real estate portfolio, decreased from $1.2 million in the year-ago quarter.
Loss per share of $0.09, a decrease from $0.10 in the year ago quarter largely due to a gains on the sale of the Company’s commercial real estate property in Piscataway, New Jersey and other investments which more than offset the increased research and development expense.
On December 7, 2020, the Company acquired the economic rights related to an additional 33.33% membership interest in Altira, a company which owns the rights to a receive a royalty on sales of certain Rafael Pharmaceuticals products. The acquisition added to the 33.33% interest purchased in fiscal 2020 As in the first such agreement, the Company purchased the potential right to receive a 1% royalty on Net Sales on these products.
On December 7, 2020, Rafael Holdings entered into a Securities Purchase Agreement for the sale of 567,437 shares of the Company’s Class B common stock at a price per share of $22.91 (the closing price of Class B common stock on the prior trading day) for an aggregate purchase price of $13 million. In connection with the purchases, each purchaser was granted warrants to purchase 20% of the shares of Class B common stock purchased by such purchaser exercisable at $22.91. A majority of the proceeds received are expected to be used by the Company to exercise an additional portion of the warrant held by the Company’s subsidiary to purchase equity securities of Rafael Pharmaceuticals. The remaining proceeds are expected to be used to fund the operations of Rafael Holdings’ drug development programs including its Barer Institute subsidiary and for general corporate purposes.
Rafael Pharmaceuticals
At October 31, 2020, the Company and its subsidiaries collectively owned securities representing 51% of the outstanding capital stock of Rafael Pharmaceuticals and approximately 37% on a fully diluted basis. Recent developments announced by Rafael Pharmaceuticals include:

The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the Company’s lead compound, CPI-613 (devimistat), for the treatment of both metastatic pancreatic cancer and acute myeloid leukemia (AML).
Rafael Pharmaceuticals announced that it will initiate a Phase 2 clinical trial of CPI-613(devimistat) in combination with hydroxychloroquine in patients with clear cell sarcoma of soft tissue. The Company will begin enrolling patients in partnership with Sara’s Cure and Sarcoma Alliance for Research through Collaboration (SARC).
The FDA has granted Orphan Drug Designation for CPI-613 (devimistat) for the treatment of soft tissue sarcoma. Rafael Pharmaceuticals’ clinical trial will focus on the treatment of relapsed or refractory clear cell sarcoma.
Rafael Pharmaceuticals announced that it had crossed the enrollment of 100 patients in its Phase 3 clinical trial (Armada 2000) for patients with relapsed or refractory acute myeloid leukemia (AML). The multi-center, open-label, randomized pivotal trial is assessing the efficacy and safety of Rafael’s lead compound CPI-613 (devimistat) in combination with high dose cytarabine and mitoxantrone (CHAM) compared to high dose cytarabine and mitoxantrone (HAM) therapy in older patients.
LipoMedix
At October 31, 2020, Rafael Holdings held 68% of the issued and outstanding ordinary shares of LipoMedix, a development-stage Israeli company focused on the development of an innovative, safe and effective cancer therapy based on liposome delivery.

Barer Institute
The Barer Institute has identified and begun to develop new therapeutic compounds, including compounds to regulate cancer metabolism, through internal development and in-licensing. It is working to validate newly discovered biomarkers for resistance and sensitivity within its portfolio compounds and to identify certain novel targetable mechanisms of action.

Remarks by Howard Jonas, Chairman and CEO of Rafael Holdings
"Rafael Holdings’continued to pursue the strategic goal of creating value in the pharmaceutical field including through its investments in Rafael Pharmaceuticals and its wholly owned Barer Institute. In that regard, I am pleased that, among other milestones achieved, Rafael Pharmaceuticals’ lead compound, CPI-613 (devimistat), received U.S. Food and Drug Administration granted Fast Track designation for the treatment of metastatic pancreatic cancer and, as Rafael Pharmaceuticals announced earlier today, for acute myeloid leukemia (AML). The Barer Institute continues to make meaningful progress on its drug development programs."

ESSA Pharma Provides Corporate Update and Reports Financial Results for Fiscal Fourth Quarter and Year Ended September 30, 2020

On December 15, 2020 ESSA Pharma Inc. ("ESSA", or the "Company") (NASDAQ: EPIX), a clinical-stage pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, reported a corporate update and reported financial results for the fiscal year ended September 30, 2020 (Press release, ESSA, DEC 15, 2020, View Source [SID1234572887]). All references to "$" in this release refer to United States dollars, unless otherwise indicated.

"In 2020, ESSA made significant progress towards the development of EPI-7386, our highly-selective, oral, small molecule inhibitor that targets the N-terminal domain of the androgen receptor for the treatment of patients with metastatic castration-resistant prostate cancer (mCRPC)," stated David Parkinson, MD, President and CEO of ESSA. "In July, we initiated a Phase 1 monotherapy dose escalation clinical study with EPI-7386 and the study is progressing as planned. Additionally, with the $48.9 million raised in a public offering in July, we believe we are well-positioned financially to advance the development of EPI-7386 in the current Phase 1 study as well as to initiate one or more combination studies with approved anti-androgen treatments."

Clinical and Corporate Highlights for 2020 Fiscal Year

On September 14, 2020, the Company announced that Fast Track Designation had been granted by the FDA to EPI-7386 for the treatment of mCRPC.
On July 31, 2020, the Company closed a public offering of common shares, led by Jefferies, as sole book-running manager, for gross proceeds of $48.9 million. Certain existing investors participated in the financing along with new investors: Pfizer Inc. (NYSE: PFE), Avidity Partners, CAM Capital, Point72, Ridgeback Capital, Sphera Healthcare, Vivo Capital, and others.
On July 15, 2020, the Company announced that the first patient had been dosed in a Phase 1 clinical trial designed to evaluate the safety and tolerability of EPI-7386 in mCRPC patients who failed standard of care treatments, including second generation anti-androgens. The trial is being conducted at five sites in the United States and Canada and is expected to enroll approximately 18 patients in a standard 3+3 trial design with an approximate 10 additional patients enrolled in the dose expansion cohort.
Throughout the year, at multiple scientific conferences, the Company presented preclinical data characterizing the preclinical profile of EPI-7386 in various prostate cancer preclinical models, including studies evaluating androgen receptor binding, gene expression analyses and the toxicologic profile were presented, in addition to data related to the binding and utility of EPI-7386 against AR-V7 splice-variant driven prostate cancer models.
In October 2019, the Company paid off the balance of its $3.6M debt facility, leaving the Company with no outstanding debt.
Summary Financial Results

Net Loss. ESSA recorded a net loss of $23.4 million ($1.04 loss per common share based on 22,443,893 weighted average common shares outstanding) for the year ended September 30, 2020, compared to a net loss of $12.8 million ($1.51 loss per common share based on 8,433,441 weighted average common shares outstanding) for the year ended September 30, 2019. For the year ended September 30, 2020, this included non-cash share-based payments of $7.5 million compared to $1.1 million for the prior year, recognized for stock options granted and vesting. The net loss for the fourth quarter ended September 30, 2020 was $4.5 million compared to a net loss of $3.3 million for the fourth quarter ended September 30, 2019.
Research and Development ("R&D") expenditures. R&D expenditures for the year ended September 30, 2020 were $12.1 million compared to $6.7 million for the year ended September 30, 2019 and includes non-cash costs related to share-based payments ($1.9M for year ended 2020 compared to $304,786 for year ended 2019). For the fourth quarter ended September 30, 2020, R&D expenditures were $2.2 million (net and gross), as compared to $2.0 million (net and gross) for the fourth quarter ended September 30, 2019. The increase in R&D expenditures for the full year and fourth quarter were primarily related to preclinical work leading to the filing of the IND for EPI-7386 in March 2020, and the increased expenditure on chemistry and manufacturing of drug product, and clinical costs related to the Phase 1 clinical trial of EPI-7386 which commenced with the dosing of the first patient in July 2020.
General and administration ("G&A") expenditures. G&A expenditures for the year ended September 30, 2020 were $11.4 million compared to $5.5 million for the year ended September 30, 2019 and include non-cash costs related to share-based payments of $5.6M for the year ended 2020 compared to $841,921 for the year ended 2019. For the fourth quarter ended September 30, 2020, G&A expenditures were $2.2 million, compared to $1.2 million for the fourth quarter ended September 30, 2019. The increase in the full year and fourth quarter is the result of increased professional fees related to transitioning to be a domestic filer, higher salaries and benefits, as well as the non-cash share-based payments.
Liquidity and Outstanding Share Capital
At September 30, 2020, the Company had available cash reserves and short-term investments of $78,332,100, reflecting the gross proceeds of the July 2020 financing of $48.9 million, less operating expenses in the intervening period.

As of September 30, 2020, the Company had 32,064,411 common shares issued and outstanding.

In addition, as of September 30, 2020 there were 9,272,977 common shares issuable upon the exercise of warrants and broker warrants. This includes 8,863,504 prefunded warrants at an exercise price of $0.0001, and 409,473 warrants at a weighted average exercise price of $39.12. There are 5,309,584 common shares issuable upon the exercise of outstanding stock options at a weighted-average exercise price of $3.42 per common share.

Transition to US GAAP reporting
As noted in prior quarters, ESSA began reporting its results in accordance with US GAAP effective for the fiscal 2020 results. This transition is a result of the fact that the Company is no longer a foreign private issuer ("FPI") as defined under the rules of the United States Securities and Exchange Commission ("SEC"). As a domestic filer, the Company prepares consolidated financial statements in accordance with US GAAP, reports with the SEC on domestic forms, and complies with SEC rules and regulations applicable to domestic issuers.

All prior reporting periods for 2020 have been converted to US GAAP, with the first, second, and third quarter 2020 unaudited condensed consolidated interim financial statements re-filed in accordance with US GAAP.

About EPI-7386
EPI-7386 is an investigational, highly-selective, oral, small molecule inhibitor of the N-terminal domain of the androgen receptor. EPI-7386 is currently being studied in a Phase 1 clinical trial ((NCT04421222) in men with metastatic castration-resistant prostate cancer ("mCRPC") whose tumors have progressed on current standard-of-care therapies. The Phase I clinical trial of EPI-7386 began in calendar Q3 of 2020 following FDA allowance of the IND and Health Canada acceptance. The U.S. FDA has granted Fast Track designation to EPI-7386 for the treatment of adult male patients with mCRPC resistant to standard-of-care treatment. ESSA retains all rights to EPI-7386 worldwide.

Precigen Provides Latest Clinical Developments at Virtual R&D Update Event

On December 15, 2020 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported that held an R&D virtual event to provide an update on the latest progress for its clinical pipeline (Press release, Precigen, DEC 15, 2020, View Source [SID1234572885]).

The event showcased data from several of Precigen’s most advanced clinical programs—PRGN-3005 UltraCAR-T, PRGN-3006 UltraCAR-T and AG019 ActoBioticsTM with presentations and discussions from Precigen executives and key opinion leaders, including Dr. Helen Sabzevari, President and CEO of Precigen, Dr. Pieter Rottiers, CEO of Precigen ActoBio, Dr. Mary L. (Nora) Disis, faculty member at the University of Washington and Fred Hutchinson Cancer Research Center and one of the lead investigators for the PRGN-3005 clinical study, and Dr. Kevan Herold, Professor of Immunobiology and of Medicine (Endocrinology) at Yale School of Medicine and one of the lead investigators for the AG019 clinical study. An archive of the audio recording and presentation will be available in the Press & Events section of the investor relations website at investors.precigen.com/press-and-events.

"When we began the year, we set aggressive goals to deliver clinical data for our most advanced programs by the end of the year. Today’s presentation delivers on several of these goals and represents numerous firsts with respect to new clinical data for Precigen," said Helen Sabzevari, PhD, President and CEO of Precigen. "We presented the first clinical data ever reported for our lead UltraCAR-T programs, including dosing and safety information. In addition, for PRGN-3005 UltraCAR-T in advanced ovarian cancer patients, we were pleased to report encouraging preliminary findings of expansion, persistence and clinical activity. Data presented on PRGN-3006 UltraCAR-T in acute myeloid leukemia showed encouraging expansion and persistence after low dose infusion and the ability for PRGN-3006 to traffic, expand and persist in bone marrow. Finally, we presented data indicating that a single eight-week treatment cycle of oral AG019 induces C-peptide stabilization and antigen-specific immune tolerance. We are incredibly encouraged by the results to date and look forward to providing future updates as these studies progress."

First Clinical Data Reported for PRGN-3005 UltraCAR-T Demonstrating Encouraging Preliminary Results
The first clinical data for patients treated at Dose Level 1 (n=3) and Dose Level 2 (n=3) without prior lymphodepletion from the IP arm was reported, including case studies for three patients. PRGN-3005 UltraCAR-T is currently under clinical evaluation in a Phase 1 clinical study for the treatment of advanced, recurrent platinum resistant ovarian, fallopian tube or primary peritoneal cancer (clinical trial identifier: NCT03907527). Data were also provided for individual target lesion response and change in target tumor burden.

Summary of clinical trial status and key results from the first six patients treated with PRGN-3005 UltraCAR-T:

Enrollment: The Phase 1 trial in the intraperitoneal (IP) arm is enrolling patients in the dose escalation phase.
Dosing: The six patients received PRGN-3005 at one of the following dose levels without lymphodepletion:
Dose Level 1: 3×104 – ≤ 1 x 105 UltraCAR-T cells/kg
Dose Level 2: 1×105 – ≤ 3 x 105 UltraCAR-T cells/k
Prior Lines of Therapy: Patients received between 6 to 9 prior therapies before enrolling in the PRGN-3005 study.
Safety: PRGN-3005 treatment is safe and well-tolerated to date, with no dose-limiting toxicities (DLTs), neurotoxicity or cytokine release syndromes (CRS) reported.
Manufacturing: 100% manufacturing success to date using decentralized, rapid manufacturing process.
Clinical Activity: PRGN-3005 UltraCAR-T cells showed encouraging expansion and persistence after low dose IP infusion without lymphodepletion. Additionally:
PRGN-3005 treatment indicated clinical activity as evidenced by reduction in target lesions with 50% of patients treated (3 of 6) at either Dose Level 1 or Dose Level 2 experiencing regression in target tumor burden; and
33% of patients (2 of 6) achieved Stable Disease (SD) according to RECIST v1.1 criteria at their restaging evaluation.
Expanded Preliminary Data for PRGN-3006 UltraCAR-T, Building on Recent Presentation at the 62nd ASH (Free ASH Whitepaper) Annual Meeting And Exposition
Expanded preliminary data for patients treated at Dose Level 1 (n=3) and Dose Level 2 (n=3) without prior lymphodepletion and Dose Level 1 with lymphodepletion (n=3) were reported for PRGN-3006 UltraCAR-T, currently under clinical evaluation in a Phase 1/1b clinical study for the treatment of patients with relapsed or refractory (r/r) acute myeloid leukemia (AML) or higher-risk myelodysplastic syndromes (MDS) (clinical trial identifier: NCT03927261). These new data build on results presented at the 62nd ASH (Free ASH Whitepaper) Annual Meeting and Exposition. A single patient case study provided additional clinical insights.

Summary of clinical trial status and key results from the first nine patients treated with PRGN-3006 UltraCAR-T:

Enrollment: The Phase 1 trial is enrolling patients in the dose escalation phase of both the lymphodepletion and non-lymphodepletion cohorts.
Dosing: The nine patients received PRGN-3006 at one of the following dose levels:
Dose Level 1: 3×104 – ≤ 1 x 105 UltraCAR-T cells/kg
Dose Level 2: 1×105 – ≤ 3 x 105 UltraCAR-T cells/
Safety: PRGN-3006 treatment is safe and well-tolerated to date, with no DLTs or neurotoxicity. Transient grade 1-3 CRS were reported in two patients.
Manufacturing: 100% manufacturing success to date using decentralized, rapid manufacturing process.
Clinical Activity: PRGN-3006 cells showed encouraging expansion and persistence in peripheral blood after low dose infusion. Additionally:
PRGN-3006 cells showed the ability to traffic, expand and persist in bone marrow; and
PRGN-3006 treatment indicated clinical activity as evidenced by reduction in AML tumor blast levels.
New AG019 ActobioticsTM Data from Phase 1b Monotherapy (up to 12 months follow-up) and Phase 2a Combination Study (up to six months follow-up)
Precigen ActoBio, Inc., a wholly-owned subsidiary of Precigen, announced new data for the Phase 1b monotherapy and Phase 2a combination study of the ongoing Phase 1b/2a clinical study investigating AG019 ActoBiotics for the treatment of early-onset type 1 diabetes (T1D) (clinical trial identifier: NCT03751007, EudraCT 2017-002871-24).

Key clinical results:

Phase 1b oral AG019 monotherapy:

Safety: The AG019 monotherapy treatment was well-tolerated and safe when administered as a single low or high dose and as a repeated low or high daily dose for 8 weeks. There were no AG019 treatment discontinuations due to treatment emergent adverse events (TEAEs).
Clinical Activity: Following a single 8-week treatment cycle of oral AG019, 58% of the adult patients (7 of 12) showed stabilization of C-peptide levels during the first 6 months and slower decline in C-peptide levels at 12 months compared to placebo. Results indicated the potential to preserve insulin production in early onset T1D through its capacity to induce antigen-specific immune modulation.
Potential Differentiation: The ease of treatment due to oral dosing and disease modifying potential differentiates AG019 from competition.
Mechanistic Data: In a mechanistic analysis performed by the Immune Tolerance Network, a leading independent research group, AG019 monotherapy showed the induction of antigen-specific tolerance in conjunction with the reduction of disease-specific T cell responses for adult patients three months after treatment initiation. Specific mechanistic data include:
An increase in antigen preproinsulin (PPI)-specific Type 1 regulatory (Tr1) cells;
An increase of islet-specific (memory) regulatory T-cells expressing inhibitory receptors, which may indicate induction of tissue-specific bystander suppression; and
A significant decrease in antigen (PPI)-specific CD8 T-cells.
Phase 2a AG019 combination therapy:

Safety: The combination of AG019 and teplizumab is safe and well-tolerated to date.
Clinical Activity: Following the treatment with the combination of AG019 and teplizumab, 70% of the adult patients (7 of 10) showed stabilization of C-peptide levels at 6 months post treatment initiation with a trend towards higher C-peptide levels as compared to baseline levels.
Mechanistic Data: Similar to the immunological effects seen in AG019 monotherapy patients, the combination of AG019 and teplizumab showed the induction of antigen-specific tolerance in conjunction with reduction of disease-specific T cell responses for adult patients three months after treatment initiation. The extent of these antigen-specific immune modulatory effects in the combination therapy patients is similar to what was seen in AG019 monotherapy patients indicating that this effect may be attributed to the single 8-week treatment cycle of oral AG019. Specific mechanistic data include:
An increase in PPI- and islet-specific specific Type 1 regulatory (Tr1) cells; and
A significant decrease in antigen (PPI)-specific CD8 T-cells.
"The Phase 1b monotherapy data for AG019 up to six months after treatment and the interim Phase 2a combination data in adult patients show the treatment is safe and well-tolerated and continue to showcase the potential of the ActoBiotics therapeutic platform," said Pieter Rottiers, PhD, CEO of Precigen ActoBio. "The Phase 1b study continues to show higher C-peptide levels at 12 months compared to placebo following only one treatment cycle of oral AG019. The Phase 2a study shows the potential to boost or prolong teplizumab-induced metabolic effects through induction of antigen-specific immune modulation and the opportunity to explore combinations with other systemic inducers in addition to teplizumab."

Precigen: Advancing Medicine with Precision
Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. 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. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

About Precigen ActoBio
Precigen ActoBio is a clinical stage biotechnology company and a wholly-owned subsidiary of Precigen (Nasdaq: PGEN) pioneering a new class of therapeutic agents created on the ActoBiotics platform. The ActoBiotics platform provides a new class of therapeutic agent, a unique delivery platform precisely tailored for specific disease modification, with the potential for superior efficacy and safety via local delivery directly to the relevant tissue. ActoBiotics are targeted, microbe-based, specifically designed agents that express and locally deliver potential disease-modifying therapeutics at disease sites including the intestine, the mouth and the nasopharynx, to treat a range of disorders. Precigen ActoBio has a strong R&D pipeline and an extensive portfolio of candidates advancing toward clinical development across a number of potential indications. Learn more about Precigen ActoBio at www.precigen.com/actobio.

About PRGN-3005 UltraCAR-T
PRGN-3005 UltraCAR-T is a multigenic autologous CAR-T cell treatment utilizing Precigen’s Sleeping Beauty system to simultaneously express a CAR specifically targeting the unshed portion of MUC16, which is highly expressed on ovarian tumors with limited normal tissue expression; membrane bound IL-15 for enhanced in vivo expansion and persistence; and a kill switch to conditionally eliminate CAR-T cells for an improved safety profile. PRGN-3005 is being evaluated in collaboration with the University of Washington and Fred Hutchinson Cancer Research Center in an investigator-initiated open-label, dose escalation Phase 1 study to evaluate the safety and maximal tolerated dose of PRGN-3005 delivered by intraperitoneal infusion (IP) or intravenous infusion (IV) (clinical trial identifier: NCT03907527). 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.

About PRGN-3006 UltraCAR-T
PRGN-3006 UltraCAR-T is a multigenic autologous CAR-T cell treatment utilizing Precigen’s Sleeping Beauty system to simultaneously express a CAR specifically targeting CD33, which is over expressed on acute myeloid leukemia blasts with lesser expression on normal hematopoietic stem cell populations and minimal non-hematopoietic expression; membrane bound IL-15 for enhanced in vivo expansion and persistence; and a kill switch to conditionally eliminate CAR-T cells for improved safety profile. PRGN-3006 is being evaluated in collaboration with the Moffitt Cancer Center in a nonrandomized, investigator–initiated Phase 1/1b dose escalation study to evaluate the safety and maximal tolerated dose of PRGN–3006 UltraCAR-T (clinical trial identifier: NCT03927261). The study population includes patients with relapsed or refractory acute myeloid leukemia or higher risk myelodysplastic syndrome.

About AG019
AG019 is an investigational therapy designed to induce oral immune tolerance to reverse T1D and currently under clinical evaluation for the treatment of early-onset type 1 diabetes (T1D) (clinical trial identifier: NCT03751007, EudraCT 2017-002871-24). The Phase 1b/2a clinical trial is evaluating AG019 as a monotherapy and in combination with teplizumab (PRV-031), which is currently under investigation in the PROTECT Phase 3 study for the treatment of newly diagnosed T1D. Both the Phase 1b portion of the study, testing AG019 monotherapy in patients 12 to 17 years of age and adults 18-40 years of age, and the first cohort of the Phase 2a portion, testing the combination dosing of AG019 plus teplizumab in adults 18-40 years of age, are fully enrolled.

Trademarks
Precigen, Advancing Medicine with Precision, UltraCAR-T and ActoBiotics are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners.